Stem Cell Clinic

Emendo Biotherapeutics and Seattle Childrens Research Institute Announce Collaboration to Develop CRISPR-based Therapeutic Strategy for Severe…

By Stem Cell Treatment

Collaboration formed to conduct preliminary research to inform protocol development ahead of gene editing clinical trial

NEW YORK & SEATTLE, December 22, 2021--(BUSINESS WIRE)--Emendo Biotherapeutics, a next-generation CRISPR biotech expanding the reach of gene editing therapeutics, and Seattle Childrens Research Institute today announced a research collaboration to investigate how hematopoietic stem cells (HSCs) extracted from patients with severe congenital neutropenia (SCN) respond to priming treatments ahead of administering a CRISPR-based therapeutic.

ELANE-related SCN, also known as SCN1, is a rare, autosomal dominant disease in which a mutation occurs in one allele of the ELANE gene, thereby preventing HSCs from differentiating into white blood cells, specifically neutrophils, which leaves the patient highly susceptible to recurrent bacterial infections, osteoporosis, developmental delays and abnormalities.

"Patients with SCN often suffer from reduced quality of life due to the lack of improvements in the standard of care," said Dr. David Rawlings, Division Chief of Immunology at Seattle Children's Hospital and Director of the Center for Immunity and Immunotherapies at Seattle Childrens Research Institute. "These children are immunocompromised, and, as a result, we feel a great sense of urgency to ensure were exploring all possible avenues towards a solution."

"Seattle Childrens collaboration with Emendo, utilizing its unique approach to edit only the mutated allele with CRISPR, will enable us to address the unmet needs of SCN at the very core," added Rawlings, who also serves as a professor of pediatrics and adjunct professor in the Department of Immunology at the University of Washington School of Medicine. "Were excited about this opportunity, and look forward to continuing the collaboration beyond this initial study."

Editing the mutated ELANE gene with CRISPR first requires overcoming a technological hurdle: Only the mutated allele must be targeted, while the healthy allele remains intact. Emendo engineered its roster of next-generation CRISPR nucleases to be biologically active and so specific that they can differentiate between two alleles of the same gene. EMD-101, Emendos lead therapeutic candidate for SCN, was specifically engineered to target the mutant ELANE allele.

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HSCs have been widely studied as a treatment for sickle cell anemia and cancer, as well as a potential therapy to treat organ and tissue damage. However, HSCs require initial priming prior to stem cell transplantation, which is typically done by administering G-CSF (granulocyte colony stimulating factor). Yet, the same drug is also a short-term treatment for SCN patients.

To better understand how SCN patients would respond to a priming dose of G-CSF and plerixafor, Emendo will evaluate the mobilization of HSCs excised from a small group of patients with SCN, which would be gene-edited later. Concurrently, Seattle Childrens will evaluate the composition of the HSCs obtained from the same patients. Prior mouse studies conducted by Emendo have shown that human cells edited to excise the disease-causing ELANE allele sufficiently engrafted and replaced existing diseased cells, restoring proper neutrophil differentiation.

"By combining our allele-specific genome editing technology with Seattle Childrens renowned expertise in SCN spearheaded by Dr. Rawlings we are laying the foundation for future clinical trials that could lead to potential therapies to treat the disease," said David Baram, Ph.D., CEO of Emendo. "Our portfolio of engineered nucleases tailored to any gene or allele gives us the unique opportunity to tackle the inherent challenges of SCN. Through this collaboration well be able to provide stronger evidence and further proof points for the capabilities of our technology."

Based on the outcome of the research, a protocol for a clinical trial could be developed with an expected initiation in late 2022, pending regulatory approval. Seattle Children's has certain preferred rights to serve as a clinical trial site.

About Emendo Biotherapeutics

EmendoBio is a next generation CRISPR gene editing company leveraging dual proprietary technology platforms to enable high precision gene editing throughout the genome. EmendoBios novel nuclease discovery platform broadens the targetable range of the genome while its target-specific optimization platform enables highly precise editing, including allele specific editing, while maintaining high efficiencies. The capabilities of the OMNI technology platforms, along with deep expertise in genomic medicine, protein engineering and therapeutic development, provide EmendoBio with a unique advantage when addressing indications within hematology, oncology, ophthalmology and other disease areas. For more information please visit us at http://www.emendobio.com.

About Seattle Childrens

Seattle Childrens mission is to provide hope, care and cures to help every child live the healthiest and most fulfilling life possible. Together, Seattle Childrens Hospital, Research Institute and Foundation deliver superior patient care, identify new discoveries and treatments through pediatric research, and raise funds to create better futures for patients.

Ranked as one of the top childrens hospitals in the country by U.S. News & World Report, Seattle Childrens serves as the pediatric and adolescent academic medical center for Washington, Alaska, Montana and Idaho the largest region of any childrens hospital in the country. As one of the nations top five pediatric research centers, Seattle Childrens Research Institute is internationally recognized for its work in neurosciences, immunology, cancer, infectious disease, injury prevention and much more. Seattle Childrens Foundation works with the Seattle Childrens Guild Association, the largest all-volunteer fundraising network for any hospital in the country, to gather community support and raise funds for uncompensated care and research. Join Seattle Childrens bold initiative It Starts With Yes: The Campaign for Seattle Childrens to transform childrens health for generations to come.

For more information, visit seattlechildrens.org or follow us on Twitter, Facebook, Instagram or on our On the Pulse blog.

View source version on businesswire.com: https://www.businesswire.com/news/home/20211222005552/en/

Contacts

Zuri McClelland HDMZ Emendo.pr@hdmz.com 312-506-5214

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Emendo Biotherapeutics and Seattle Childrens Research Institute Announce Collaboration to Develop CRISPR-based Therapeutic Strategy for Severe...

National Advertising Division Recommends Cryo-Cell Discontinue or Modify Certain Health-Related Claims for Cord Blood Banking and Treatment Services -…

By Stem Cell Treatment

PR Newswire

NEW YORK, Dec. 21, 2021

NEW YORK, Dec. 21, 2021 /PRNewswire/ -- The National Advertising Division (NAD) of BBB National Programs determined that certain advertising claims made by Cryo-Cell International, Inc. reasonably conveyed the unsupported message that families must engage the advertiser's cord blood storage services to ensure access to the advertised cord blood infusion treatments. Therefore, NAD recommended that Cryo-Cell discontinue such claims, or modify its advertising to avoid conveying the message that consumers have exclusive or superior access to advertised cord blood infusion treatments by virtue of choosing Cryo-Cell for storage services.

National Advertising Division (NAD) (PRNewsfoto/National Advertising Division,B)

The advertising at issue was challenged by ViaCord, LLC, a provider of competing cord blood banking services.

The challenged claims, which appeared on the advertiser's website, included express claims stating:

"This partnership will benefit families who store with Cryo-Cell by allowing them to have foremost access to the infusion treatments that are currently only performed at Duke University to treat autism, cerebral palsy, and other neurologic diseases in accordance with the FDA expanded access rights granted to Duke."

"What Sets Us Apart Key Partnerships Patients will have access to investigational therapies for certain conditions through our partnership with Duke University.

"Additionally, families will benefit from access to investigational therapies through the arrival of a new infusion clinic, expected in January 2022."

"Treatment Access" Cryo-Cell "[w]ill offer families expanded treatment options under an FDA-approved IND for therapies using cord blood stem cells."

"New Treatment Possibilities: Receive access to innovative and quality care."

"Access to Innovative Treatments"

"Extended Benefits for Patients access to treatment in clinical trialsExpedited Participationcritically important to provide access to therapy before the child outgrows the number of cells available for treatment."

"NOT ALL CORD BLOOD BANKS ARE CREATED EQUALkey partnerships"

ViaCord also challenged implied claims that:

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Storing cord blood with Cryo-Cell provides consumers exclusive and/or better access to treatments than storing with other cord blood banks.

Such access is because competitors lack "key partnerships" relating to such treatments.

The advertiser offers services relating to the preservation, storage and transportation of cord blood stored on behalf of families for potential future medical use. Expectant parents who choose to bank cord blood with a private bank such as Cryo-Cell register with the bank before the birth of their child. The bank helps facilitate the collection of the blood at the time of birth and the blood is then immediately taken by courier to a facility where it is then processed and stored.

Duke University holds a patent on methods of treating autism with cord blood and has granted Cryo-Cell an exclusive license to practice those treatment methods. Clinical trials of cord blood as treatment for autism and other neurologic disorders are being conducted by Dr. Joanne Kurtzberg at Duke University under the FDA's Expanded Access Protocol (EAP). The cord blood used in these trials has come from numerous cord blood banks, including both ViaCord and Cryo-Cell. Cryo-Cell is planning on opening a clinic in 2022 to provide infusion treatments to patients in accordance with the patent license and the EAP to advance regenerative therapy research and "bring greater access to novel cord blood and cord tissue-based cellular therapies to treat conditions such as autism, cerebral palsy, and other neurological conditions."

Cord blood banking and cord blood-based treatments are distinct services. Families who have stored cord blood at an FDA-compliant bank are free to use that blood for treatments offered by any treatment provider, regardless of where the blood is stored. Cord blood used for treatments is often transferred from the bank to an infusion center, even when within the same facility, where it must be processed according to required protocols.

NAD determined that one reasonable takeaway from the context of the challenged advertising, on an audience of expectant parents under time pressure to make an important health care decision for their family, is that one must engage the advertiser's cord blood storage service to ensure access to the advertised cord blood infusion treatments.

NAD found that the advertiser did not substantiate the message reasonably conveyed that Cryo-Cell storage clients have exclusive access to the treatments. Nor did the Advertiser demonstrate that Cryo-Cell customers have "better" access to the treatments than those families who store cord blood at a different bank in the sense that Cryo-Cell storage customers will be at risk of losing access to the treatments because they store their cord blood elsewhere.

NAD recommended that the advertiser discontinue the challenged claims or modify its advertising to avoid conveying the message that consumers have exclusive or superior access to the advertised cord blood infusion treatments by virtue of choosing Cryo-Cell for storage services. NAD noted that the advertiser may make this modification by clearly and conspicuously disclosing that using Cryo-Cell's storage services will not impact eligibility for the treatments, or, by modifying its website to separate its claims related to exclusive benefits for Cryo-Cell customers and access to the treatments available to all eligible patients.

Finally, NAD noted that nothing in its decision precludes the advertiser from making truthful and non-misleading claims about any convenience benefit to Cryo-Cell storage customers when accessing cord blood treatments or from advertising the exclusive license it has with Duke University and the treatments that may be available to eligible patients as a result.

In its advertiser statement, Cryo-Cell stated that it "agrees to comply with NAD's recommendations" and that it "will indeed provide additional disclosures stating that access to treatments at the Cryo-Cell Institute for Cellular Therapies, including treatments under Cryo-Cell's exclusive license with Duke University and the FDA's Expanded Access Protocol, will be available to all eligible patients, including those that have their cord blood stored at another cord blood storage provider."

All BBB National Programs case decision summaries can be found in the case decision library. For the full text of NAD, NARB, and CARU decisions, subscribe to the online archive.

About BBB National Programs: BBB National Programs is where businesses turn to enhance consumer trust and consumers are heard. The non-profit organization creates a fairer playing field for businesses and a better experience for consumers through the development and delivery of effective third-party accountability and dispute resolution programs. Embracing its role as an independent organization since the restructuring of the Council of Better Business Bureaus in June 2019, BBB National Programs today oversees more than a dozen leading national industry self-regulation programs, and continues to evolve its work and grow its impact by providing business guidance and fostering best practices in arenas such as advertising, child-directed marketing, and privacy. To learn more, visit bbbprograms.org.

About the National Advertising Division: The National Advertising Division (NAD) of BBB National Programs provides independent self-regulation and dispute resolution services, guiding the truthfulness of advertising across the U.S. NAD reviews national advertising in all media and its decisions set consistent standards for advertising truth and accuracy, delivering meaningful protection to consumers and leveling the playing field for business.

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Seven tech trends to watch in 2022 | The Star – Toronto Star

By Stem Cell Treatment

With COVID resurging and the climate crisis intensifying, 2021 is ending on a precarious note. But there are also reasons to be optimistic (or at least somewhat optimistic). Across Canada, scientists and entrepreneurs are innovating in ways that will help people strike a better work-life balance, live sustainably and stay healthy. Here are seven trends in Canadas thriving innovation economy to watch in the year ahead.

Farm-to-table becomes lab-to-table

With the supply-chain crisis expected to push family grocery bills up by an estimated $966 next year, Canada needs to find new ways to sustainably feed itself. According to Dana McCauley of the Canadian Food Innovation Network, when it comes to food, the really exciting stuff right now is happening in labs.

She points to companies, such as Vancouvers Wamame, which has produced the worlds first plant-based version of Wagyu beef and is leading a consortium to create other meatless meats. Meanwhile, researchers at the University of Guelph have discovered how to make plant-based cheese stretch, which could soon put a tastier and oozier vegan mozzarella on the menu.

Companies are taking advantage of advances in engineering yeast, algae and other microbes to create foods like animal-free milk. But researchers are also working on technologies to grow real meat from cell cultures, which are touted as being cruelty-free and better for the environment. Scientists at McMaster University recently found a way to better control the fat and muscle content of cultured meat, bringing the possibility of lab-grown steak a step closer.

McCauley points out that these techniques can also be used to grow specialized plant products, cutting down food miles: In future, instead of coming from Madagascar, maybe my vanilla will come from Montreal.

Hybrid working becomes a test of corporate culture

When the Omicron wave recedes, executives will again be mulling their return-to-the-office plans. But with 80 per cent of workers not keen on returning to their desks full time, a period of experimentation with various forms of part-office part-remote hybrid working lies ahead. Kyra Jones, head of talent at Communitech, says that successful companies will use this as an inflection point to reimagine how work is done, where work is done and what work looks like.

Toronto-based enterprise software company Sensei Labs is among a growing number of tech companies that have adopted work-from-anywhere policies. Staff are free to log on from the office, at home, in a coffee shop or even further afield. We have a very international team, many of them have family in places like Brazil, or India or Ukraine and we encourage them to go work from there for an extended period, says CEO Jay Goldman. The company also suggests which days each team might go into the office, so staff who want to be in part-time know when theyll be most likely to see their closest colleagues.

But its not just HR policies that are evolving, technology is too. Tools are now emerging designed with long-term remote working in mind. Vizetto, for instance, has created a virtual chalkboard to encourage collaborative meetings. WorkTango, an employee feedback platform, has created quick pulse surveys to make it easier for managers to check how workers are feeling. Regardless of the technology, however, hybrid working will continue to be tricky terrain for managers to navigate. Unsurprisingly, Jones says demand for leadership training courses is surging.

Lithium is the new oil

After production delays in 2021, electric-vehicle manufacturers are hoping to make up lost ground and they are all racing to secure supplies of lithium for the batteries. Amanda Hall, CEO of lithium-extraction firm Summit Nanotech, predicts global production will increase by about a third next year. But suppliers will still struggle to keep up with demand.

The downside is that soaring lithium prices could lead to sticker shock at the dealership if costs are passed on to electric-vehicle buyers. The upside: the focus on lithium is putting pressure on miners to clean up their operations.

The major investors in the world are saying we wont support unsustainable practices anymore, says Hall, who recently won Canadas $1-million Women in Cleantech Challenge for developing an extraction system that generates 90 per cent less waste than traditional chemical methods. Australia, one of the worst polluters, is looking at using electric vehicles and renewable energy in its lithium mines. As Canada considers plans for developing mineral mining in places like northern Ontario, expect environmental safeguards to come under close scrutiny here, too.

Stem cell therapies advance in clinical trials

For two decades, researchers have been experimenting with stem cells to repair damaged tissues and treat diseases. Now, those studies are leaving the lab and moving into clinical trials to see how they perform in real patients.

The long-standing investment in regenerative medicine is going to start to bear fruit but to a limited extent for a limited number of patients, says Michael Sefton, executive director of the University of Torontos Medicine by Design program.

In Vancouver, biotech firm ViaCyte is testing a device that implants pancreatic stem cells into diabetes patients, which should grow into insulin-producing cells and reduce the need for injections. And BlueRock Therapeutics, which has labs in Toronto, has started a trial using stem cells to replace damaged neurons in patients with Parkinsons Disease. If successful, these therapies could transform patients lives.

But Sefton cautions that we are still some way from seeing stem-cell treatments like these in clinical use: I think in 10 years they may be common, at least in large academic centres.

Investment in biotech picks up

After record investment poured into biotech at the start of the pandemic, 2021 was a bumpy year for the sector. Stocks of major pharmaceutical companies languished even as the industry was cranking out billions of doses of life-saving vaccines in record time.

But according to Dan Legault, CEO of Toronto-based Antibe Therapeutics, investment in biotech should pick up again in the coming year.

The pullback was too fast and too strong, he says. Legault puts the investment chill down to a lack of major mergers or acquisitions of late but says that with several promising developments on the horizon there is still huge money looking to invest in the sector.

Self-driving trucks inch closer to the road

While driverless cars may be taking longer than expected, developers of autonomous freight vehicles are stepping on the gas.

Michael Tremblay, CEO of Invest Ottawa, which operates a testing centre for driverless vehicles called AreaX.O, says interest in self-driving delivery vehicles is growing as trucking companies struggle to recruit drivers. Theres a real business need for it, he says.

At facilities like AreaX.O, researchers are now connecting up convoys of small test vehicles under the supervision of single drivers, which is seen as a potential stepping stone to fully autonomous trucks. They are also adapting driverless systems to Canadas harsh climate, including looking at centimetre-accurate GPS to enable vehicles to navigate when snow covers road markings, and testing special coatings to prevent sensors getting iced up.

For now, in Ontario, autonomous vehicles are confined to study tracks and pilot projects, and regulatory changes will be needed before driverless trucks hit our highways. But Tremblay points out that the core technology is available right now. Its a question of having society accept it.

In the meantime, researchers have discovered another uniquely Canadian challenge: geese. They dont behave the way youd expect them to they go right out in front of the vehicle, says Tremblay.

The end of writers block (maybe)

AI assistants can already help organize your life. Soon, they may be able to unlock your artistic side, too.

AI platforms have advanced to the point where they are surprisingly creative Rolling Stone recently deemed a computer-generated rock song to have an anthemic chorus.

According to Olga Vechtomova, a researcher at the University of Waterloo, its now possible to channel that creativity into a kind of electronic muse for artists. Vechtomovas lab has produced an AI model that listens to music and suggests lyrics that fit its rhythm and style. The aim is less to write the song than it is to spark musicians imaginations.

Its power is in its ability to surprise and be unpredictable, says Vechtomova. Ive seen it come up with novel metaphors and turns of phrase that would never occur to me.

There are already several platforms like LyricStudio, which uses AI to generate song lines based on topics and rhymes. Such tools are likely to proliferate in the next year or two.

But will we ever see an AI produce a masterpiece? Vechtomova has her doubts, not least because wed first need to figure out what genius actually is.

Try to set the objective to generate something thats musically brilliant we just cant define that.

David Paterson writes about technology for MaRS. Torstar, the parent company of the Toronto Star, has partnered with MaRS to highlight innovation in Canadian companies.

Disclaimer This content was produced as part of a partnership and therefore it may not meet the standards of impartial or independent journalism.

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Seven tech trends to watch in 2022 | The Star - Toronto Star

Systemic Mastocytosis Treatments Gain Hope Due To Increasing Novel Treatment Options – PRNewswire

By Stem Cell Treatment

PALM BEACH, Fla., Dec. 21, 2021 /PRNewswire/ -- FinancialNewsMedia.com News Commentary - Systemic mastocytosis is rare disease which affects very few people and it causes due to C-kit mutation which leads to higher number of mast cell production in the body resulting in accumulation of excessive mast cells in the internal body organs such as spleen, liver, bone marrow and small intestine etc. Recently, the World Health Organization (WHO) updated the prognosis, diagnosis and systemic mastocytosis treatment guidelines for the disease which in turn helped to bring uniformity in the approach by healthcare professionals. The manufacturers in the systemic mastocytosis treatment market are focusing on evaluating possible treatment options to cure the disease by investing heavily in the research & development. Various leading manufacturers are focusing on gaining FDA approval to respective drugs for the systemic mastocytosis treatment to enhance their revenue generation. A reportfrom Future Market Insights said:"Increasing awareness about the systemic mastocytosis treatment as well as symptoms of the disease due to extended effort by non-profit organizations, governmental associations and through other platforms expected to drive the growth of the systemic mastocytosis treatment market Increasing approvals and launches of drugs for the systemic mastocytosis treatment expected to drive the growth of the market Increasing spending on research & development by various pharmaceutical companies to develop novel systemic mastocytosis treatment expected to further fuel the growth of market. Increasing early diagnosis rate subsequently followed by increasing treatment seeking rate further expected to drive the growth of the systemic mastocytosis treatment market." Active companies in the markets today include: Hoth Therapeutics, Inc. (NASDAQ:HOTH), Longeveron Inc. (NASDAQ: LGVN), Bristol Myers Squibb (NYSE: BMY), Takeda Pharmaceutical Company Limited (NYSE: TAK), Amgen (NASDAQ: AMGN).

Future Market Insights continued:"The global systemic mastocytosis treatment market is expected to experience significant growth due to increasing novel treatment options. By drug class, systemic mastocytosis treatment market is expected to be dominated by the mast cell stabilizers due to superior efficacy. By indication, systemic mastocytosis treatment market is expected to be dominated by indolent systemic mastocytosis (ISM) due to higher prevalence. By route o administration, systemic mastocytosis treatment market is expected to be dominated by injectables. By distribution channel, systemic mastocytosis treatment market is expected to be dominated by the retail pharmacies due to higher patient footfall. The global systemic mastocytosis treatment market is expected to be dominated by the North America due to comparatively higher prevalence of the disease. Europe systemic mastocytosis treatment market is expected to be second most lucrative market due to higher treatment seeking rate. Latin America expected to show gradual growth in the systemic mastocytosis treatment market due to steadily increasing diagnosis. Asia-Pacific is emerging systemic mastocytosis treatment market due to increasing diagnosis subsequently followed by treatment. Middle East & Africa is the least lucrative systemic mastocytosis treatment market due to least diagnostic rate and lower awareness about the symptoms."

Hoth Therapeutics, Inc. (NASDAQ:HOTH) BREAKING NEWS: Hoth Therapeutics Announces Submission of Orphan Designation Application for HT-KIT to Treat Mastocytosis Hoth Therapeutics, Inc., a patient-focusedclinical-stage biopharmaceutical company, announced it submitted an Orphan Drug Designation Application to the US Food and Drug Administration (FDA) for HT-KIT for the treatment of mastocyctosis. HT-KIT is an antisense oligonucleotide that targets the proto-oncogene cKIT by inducing mRNA frame shifting, resulting in apoptosis of neoplastic mast cells. The KIT signaling pathway is implicated in multiple diseases, including all types of mastocytosis (such as aggressive systemic mastocytosis (ASM), mast cell leukemia (MCL), and systemic mastocytosis with associated hematological neoplasm (SM-AHN)), acute myeloid leukemia, gastrointestinal stromal tumors, and anaphylaxis.

Drugs intended to treat orphan diseases (rare diseases that affect less than 200,000 people in the US)are eligible to apply for Orphan Drug Designation (ODD), which provides multiple benefits to the sponsor during development and after approval. Hoth intends to pursue these benefits as part of the drug development for HT-KIT for treatment of mastocytosis, pending designation of the ODD application.

Benefits of Orphan Drug Designation - Under the Orphan Drug Act, drug companies can apply for ODD, and if granted, the drug will have a status which gives companies exclusive marketing and development rights along with other benefits to recover the costs of researching and developing the drug. A tax credit of 50% of the qualified clinical drug testing costs awarded upon drug approval is also possible. Regulatory streamlining and provide special assistance to companies that develop drugs for rare patient populations. In addition to exclusive rights and cost benefits, the FDA will provide protocol assistance, potential decreased wait-time for drug approval, discounts on registration fees, and eligibility for market exclusivity after approval.

Key benefits of ODD:

Hoth recently announcedthat its novelanti-cancer therapeuticexhibited highly positive results in humanized mast cell neoplasm models, representative in vitro and in vivo models for aggressive, mast cell-derived cancers such as mast cell leukemia and mast cell sarcoma. CONTINUED Read the Hoth Therapeutics full press release by going to: https://ir.hoththerapeutics.com/news-releases

In other news and developments of note in the markets this week:

Amgen (NASDAQ: AMGN) recently announced that the U.S. Food and Drug Administration (FDA) has approved Amgen and AstraZeneca'sTezspire (tezepelumab-ekko) for the add-on maintenance treatment of adult and pediatric patients aged 12 years and older with severe asthma.

Tezspirewas approved following a Priority Review by the FDA and based on results from the PATHFINDER clinical trial program. The application included results from the pivotal NAVIGATOR Phase 3 trial in whichTezspiredemonstrated superiority across every primary and key secondary endpoint in patients with severe asthma, compared to placebo, when added to standard therapy.

Longeveron Inc. (NASDAQ: LGVN), a clinical stage biotechnology company developing cellular therapies for chronic aging-related and certain life-threatening conditions, recently announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for Lomecel-B for the treatment of Hypoplastic Left Heart Syndrome (HLHS), a rare and life-threatening congenital heart defect in infants.

ODD is intended to assist and encourage companies to develop safe and effective therapies for the treatment of rare diseases or conditions. ODD positions Longeveron to be able to potentially leverage a range of financial and regulatory benefits, including government grants for conducting clinical trials, waiver of FDA user fees for the potential submission of a marketing application, and certain tax credits. Receiving ODD may also result in the product receiving seven years market exclusivity upon approval for use in the rare disease or condition for which the product was designated if all of the statutory and regulatory requirements are met.

Bristol Myers Squibb (NYSE: BMY) recently announced thatOrencia(abatacept) was approved by the U.S. Food and Drug Administration (FDA) for the prophylaxis, or prevention, of acute graft versus host disease (aGvHD), in combination with a calcineurin inhibitor (CNI) and methotrexate (MTX), in adults and pediatric patients 2 years of age and older undergoing hematopoietic stem cell transplantation (HSCT) from a matched or 1 allele-mismatched unrelated donor (URD).

"Orenciais the first FDA-approved therapy to prevent acute graft versus host disease following hematopoietic stem cell transplant, a potentially life-threatening complication that can pose a comparatively higher risk to racial and ethnic minority populations in the U.S. due to difficulty finding appropriately matched donors," said Tina Deignan, senior vice president, U.S. Immunology, Bristol Myers Squibb. "With this fourth indication forOrencia,Bristol Myers Squibb draws on its legacy and expertise in both immunology and hematology to deliver an important treatment option for patients in a disease with high unmet need.

Takeda Pharmaceutical Company Limited (NYSE: TAK) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recommended the approval of intravenous (IV) vedolizumab for the treatment of adult patients with moderately to severely active chronic pouchitis, who have undergone proctocolectomy and ileal pouch-anal anastomosis (IPAA) for ulcerative colitis (UC), and have had an inadequate response with or lost response to antibiotic therapy. The CHMP opinion will now be reviewed by the European Commission. If approved, vedolizumab will become the first treatment indicated for active chronic pouchitis across the European Union.

DISCLAIMER: FN Media Group LLC (FNM), which owns and operates Financialnewsmedia.com and MarketNewsUpdates.com, is a third- party publisher and news dissemination service provider, which disseminates electronic information through multiple online media channels. FNM is NOT affiliated in any manner with any company mentioned herein. FNM and its affiliated companies are a news dissemination solutions provider and are NOT a registered broker/dealer/analyst/adviser, holds no investment licenses and may NOT sell, offer to sell or offer to buy any security. FNM's market updates, news alerts and corporate profiles are NOT a solicitation or recommendation to buy, sell or hold securities. The material in this release is intended to be strictly informational and is NEVER to be construed or interpreted as research material. All readers are strongly urged to perform research and due diligence on their own and consult a licensed financial professional before considering any level of investing in stocks. All material included herein is republished content and details which were previously disseminated by the companies mentioned in this release. FNM is not liable for any investment decisions by its readers or subscribers. Investors are cautioned that they may lose all or a portion of their investment when investing in stocks. For current services performed FNM was compensated twenty five hundred dollars for news coverage of current press release issued by: Hoth Therapeutics, Inc. by a non-affiliated third party.

FNM HOLDS NO SHARES OF ANY COMPANY NAMED IN THIS RELEASE.

This release contains "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E the Securities Exchange Act of 1934, as amended and such forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. "Forward-looking statements" describe future expectations, plans, results, or strategies and are generally preceded by words such as "may", "future", "plan" or "planned", "will" or "should", "expected," "anticipates", "draft", "eventually" or "projected". You are cautioned that such statements are subject to a multitude of risks and uncertainties that could cause future circumstances, events, or results to differ materially from those projected in the forward-looking statements, including the risks that actual results may differ materially from those projected in the forward-looking statements as a result of various factors, and other risks identified in a company's annual report on Form 10-K or 10-KSB and other filings made by such company with the Securities and Exchange Commission. You should consider these factors in evaluating the forward-looking statements included herein, and not place undue reliance on such statements. The forward-looking statements in this release are made as of the date hereof and FNM undertakes no obligation to update such statements.

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Systemic Mastocytosis Treatments Gain Hope Due To Increasing Novel Treatment Options - PRNewswire

Seven tech trends to watch in 2022 | ThePeterboroughExaminer.com – ThePeterboroughExaminer.com

By Stem Cell Treatment

With COVID resurging and the climate crisis intensifying, 2021 is ending on a precarious note. But there are also reasons to be optimistic (or at least somewhat optimistic). Across Canada, scientists and entrepreneurs are innovating in ways that will help people strike a better work-life balance, live sustainably and stay healthy. Here are seven trends in Canadas thriving innovation economy to watch in the year ahead.

Farm-to-table becomes lab-to-table

With the supply-chain crisis expected to push family grocery bills up by an estimated $966 next year, Canada needs to find new ways to sustainably feed itself. According to Dana McCauley of the Canadian Food Innovation Network, when it comes to food, the really exciting stuff right now is happening in labs.

She points to companies, such as Vancouvers Wamame, which has produced the worlds first plant-based version of Wagyu beef and is leading a consortium to create other meatless meats. Meanwhile, researchers at the University of Guelph have discovered how to make plant-based cheese stretch, which could soon put a tastier and oozier vegan mozzarella on the menu.

Companies are taking advantage of advances in engineering yeast, algae and other microbes to create foods like animal-free milk. But researchers are also working on technologies to grow real meat from cell cultures, which are touted as being cruelty-free and better for the environment. Scientists at McMaster University recently found a way to better control the fat and muscle content of cultured meat, bringing the possibility of lab-grown steak a step closer.

McCauley points out that these techniques can also be used to grow specialized plant products, cutting down food miles: In future, instead of coming from Madagascar, maybe my vanilla will come from Montreal.

Hybrid working becomes a test of corporate culture

When the Omicron wave recedes, executives will again be mulling their return-to-the-office plans. But with 80 per cent of workers not keen on returning to their desks full time, a period of experimentation with various forms of part-office part-remote hybrid working lies ahead. Kyra Jones, head of talent at Communitech, says that successful companies will use this as an inflection point to reimagine how work is done, where work is done and what work looks like.

Toronto-based enterprise software company Sensei Labs is among a growing number of tech companies that have adopted work-from-anywhere policies. Staff are free to log on from the office, at home, in a coffee shop or even further afield. We have a very international team, many of them have family in places like Brazil, or India or Ukraine and we encourage them to go work from there for an extended period, says CEO Jay Goldman. The company also suggests which days each team might go into the office, so staff who want to be in part-time know when theyll be most likely to see their closest colleagues.

But its not just HR policies that are evolving, technology is too. Tools are now emerging designed with long-term remote working in mind. Vizetto, for instance, has created a virtual chalkboard to encourage collaborative meetings. WorkTango, an employee feedback platform, has created quick pulse surveys to make it easier for managers to check how workers are feeling. Regardless of the technology, however, hybrid working will continue to be tricky terrain for managers to navigate. Unsurprisingly, Jones says demand for leadership training courses is surging.

Lithium is the new oil

After production delays in 2021, electric-vehicle manufacturers are hoping to make up lost ground and they are all racing to secure supplies of lithium for the batteries. Amanda Hall, CEO of lithium-extraction firm Summit Nanotech, predicts global production will increase by about a third next year. But suppliers will still struggle to keep up with demand.

The downside is that soaring lithium prices could lead to sticker shock at the dealership if costs are passed on to electric-vehicle buyers. The upside: the focus on lithium is putting pressure on miners to clean up their operations.

The major investors in the world are saying we wont support unsustainable practices anymore, says Hall, who recently won Canadas $1-million Women in Cleantech Challenge for developing an extraction system that generates 90 per cent less waste than traditional chemical methods. Australia, one of the worst polluters, is looking at using electric vehicles and renewable energy in its lithium mines. As Canada considers plans for developing mineral mining in places like northern Ontario, expect environmental safeguards to come under close scrutiny here, too.

Stem cell therapies advance in clinical trials

For two decades, researchers have been experimenting with stem cells to repair damaged tissues and treat diseases. Now, those studies are leaving the lab and moving into clinical trials to see how they perform in real patients.

The long-standing investment in regenerative medicine is going to start to bear fruit but to a limited extent for a limited number of patients, says Michael Sefton, executive director of the University of Torontos Medicine by Design program.

In Vancouver, biotech firm ViaCyte is testing a device that implants pancreatic stem cells into diabetes patients, which should grow into insulin-producing cells and reduce the need for injections. And BlueRock Therapeutics, which has labs in Toronto, has started a trial using stem cells to replace damaged neurons in patients with Parkinsons Disease. If successful, these therapies could transform patients lives.

But Sefton cautions that we are still some way from seeing stem-cell treatments like these in clinical use: I think in 10 years they may be common, at least in large academic centres.

Investment in biotech picks up

After record investment poured into biotech at the start of the pandemic, 2021 was a bumpy year for the sector. Stocks of major pharmaceutical companies languished even as the industry was cranking out billions of doses of life-saving vaccines in record time.

But according to Dan Legault, CEO of Toronto-based Antibe Therapeutics, investment in biotech should pick up again in the coming year.

The pullback was too fast and too strong, he says. Legault puts the investment chill down to a lack of major mergers or acquisitions of late but says that with several promising developments on the horizon there is still huge money looking to invest in the sector.

Self-driving trucks inch closer to the road

While driverless cars may be taking longer than expected, developers of autonomous freight vehicles are stepping on the gas.

Michael Tremblay, CEO of Invest Ottawa, which operates a testing centre for driverless vehicles called AreaX.O, says interest in self-driving delivery vehicles is growing as trucking companies struggle to recruit drivers. Theres a real business need for it, he says.

At facilities like AreaX.O, researchers are now connecting up convoys of small test vehicles under the supervision of single drivers, which is seen as a potential stepping stone to fully autonomous trucks. They are also adapting driverless systems to Canadas harsh climate, including looking at centimetre-accurate GPS to enable vehicles to navigate when snow covers road markings, and testing special coatings to prevent sensors getting iced up.

For now, in Ontario, autonomous vehicles are confined to study tracks and pilot projects, and regulatory changes will be needed before driverless trucks hit our highways. But Tremblay points out that the core technology is available right now. Its a question of having society accept it.

In the meantime, researchers have discovered another uniquely Canadian challenge: geese. They dont behave the way youd expect them to they go right out in front of the vehicle, says Tremblay.

The end of writers block (maybe)

AI assistants can already help organize your life. Soon, they may be able to unlock your artistic side, too.

AI platforms have advanced to the point where they are surprisingly creative Rolling Stone recently deemed a computer-generated rock song to have an anthemic chorus.

According to Olga Vechtomova, a researcher at the University of Waterloo, its now possible to channel that creativity into a kind of electronic muse for artists. Vechtomovas lab has produced an AI model that listens to music and suggests lyrics that fit its rhythm and style. The aim is less to write the song than it is to spark musicians imaginations.

Its power is in its ability to surprise and be unpredictable, says Vechtomova. Ive seen it come up with novel metaphors and turns of phrase that would never occur to me.

There are already several platforms like LyricStudio, which uses AI to generate song lines based on topics and rhymes. Such tools are likely to proliferate in the next year or two.

But will we ever see an AI produce a masterpiece? Vechtomova has her doubts, not least because wed first need to figure out what genius actually is.

Try to set the objective to generate something thats musically brilliant we just cant define that.

David Paterson writes about technology for MaRS. Torstar, the parent company of the Toronto Star, has partnered with MaRS to highlight innovation in Canadian companies.

Disclaimer This content was produced as part of a partnership and therefore it may not meet the standards of impartial or independent journalism.

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EP. 6B: Phenotypic Theranostics in the Future of Precision Medicine – Targeted Oncology

By Gene Therapy Clinics

In September 2021, following the publication of results from the phase 3 VISION trial of lutetium (Lu)-177 PSMA-617 (LuPSMA) in select patients with metastatic castration-resistant prostate cancer (mCRPC),1 the United States Food and Drug Administration (FDA) granted priority review to LuPSMA.2

The FDAs decision comes on the heels of the trials positive results, which are explored in How the VISION trial may change prostate cancer therapy, the fourth article in this Targeted Oncology series, entitled New Precision Medicine Approaches in Advanced Prostate Cancer. However, it also comes after recent advances in genetic testing, biomarkers, nuclear imaging, and combination treatments for prostate cancer. These are discussed, respectively, in The role of imaging and genomic testing in prostate cancer therapy, New horizons in nuclear medicine for prostate cancer, and Expert perspective on the changing treatment spectrum for advanced prostate cancer, also in this series.

As the FDA reviews LuPSMA, experts have questions about this novel radiopharmaceutical and how it might be adopted in the US.

Ahead, Oliver Sartor, MD, medical director at Tulane Cancer Center in New Orleans, Louisiana, co-principal investigator of the VISION trial, and lead author of the published results, considers some of the questions about this novel radiopharmaceutical and how it might be adopted in the US. Dr. Sartor also discusses how the VISION trial fits into new prostate-specific membrane antigen (PSMA) research and explores the future of phenotypic theranostics in precision medicine.

TARGETED ONCOLOGY: What are the key takeaways from the VISION trial?

SARTOR: I think there are a couple. No. 1 is [that] we really wanted to design a trial that would result in regulatory approval in multiple countries, so that was the goal starting out. Of course, we wanted to use the PSMA Lu-177 using the PSMA-617 targeting molecule. That was kind of where we started.

I also felt that having prolongation of survival as an end point was key. To meet it, we chose very difficult-to-treat patients. The patients who enrolled in VISION had already gone through chemotherapy and at least 1 taxane. Many of the patientsabout 40%had actually had 2 lines of a taxane chemotherapy prior to enrolling in the trial. Everyone was also required to have use[d] at least 1 novel hormone, but multiple novel hormones were allowed. Abiraterone and enzalutamide, for instance, would have previously been used. A substantial proportion of them had undergone not just 1 but 2 chemotherapies, and all of them had undergone multiple hormonal treatments. These patients were extremely difficult to treat.

We also used the PSMA PET scan to choose and exclude patients. We wanted to choose patients who have PSMA PET metastases greater than just in the liver. This wasn't a stringent criterion, but we wanted to make sure that everybody had PSMA positivity. We also excluded patients who had PSMA negativity, lymph nodes greater than 2.5 cm, or visceral lesions of more than 1 cm.[There] were [also] a variety of other inclusion criteria like adequate performance status, adequate bone marrow, etc.

Included patients were randomized to receive a nonchemotherapeutic standard of care [treatment]. This included additional hormones, radiation therapy, bisphosphonates, maybe steroids plus or minus the PSMA lutetium, etc. There was a 2-to-1 randomization. Overall survival (OS) was an end point. Also, after the trial was already designed, there was a radiographic progression-free survival (rPFS) end point added. Patients were intended to be treated with at least 4 cycles for the PSMA lutetium and could receive up to 6 if there was evidence of clinical benefit. That's the basic framework of the trial.

The bottom line is we hit OS, and we hit rPFS. I think the safety profile was good. We also had health-related quality-of-life improvement for the PSMA lutetium. I believe this trial will result in multiple regulatory approvals, which was the goal that we set out to accomplish.

TARGETED ONCOLOGY: Based on findings from the VISION trial, what might we expect from ongoing clinical trials investigating Lu-PSMA-617 earlier in the natural history of prostate cancer?

SARTOR: Patients in the VISION trial had all failed a novel hormone and a taxane-based chemotherapy, so the VISION trial [included]advanced patient[s] with chemotherapy exposure. However, many patients with prostate cancer never receive chemotherapy, so were now starting a trial for patients with [m]CRPC called PSMAfore (NCT04689828), [which] doesn't require patients prior use of chemotherapy. Here, we're taking patients without chemotherapy exposure, but we're requiring that they have at least abiraterone or enzalutamide as a prior treatment. PSMAfore is moving forward with an rPFS end point with a crossover for those who are on the control arm, [so] they would have the opportunity to also receive PSMA lutetium. That trial is already accruing: I've already personally enrolled patients into the trial.

PSMAfore examines the castration-resistant space. We're also moving into the castration-sensitive space. In a phase 3 trial, we're going to be examining metastatic castrate-sensitive prostate cancer. Everybody receives androgen-deprivation therapy (ADT) and a novel hormone. The novel hormone can be [the] doctor's choice: abiraterone, enzalutamide, or apalutamide, all of which are FDA approved. This trial [is] plus or minus the PSMA lutetium. Here again, we're using an rPFS end point. This is going to be a big global trial. It, too, is already accruing patients. We've already consented our first patient here in the United States, and it's accruing in multiple countries around the globe.

We're hopeful that these earlier stage trials with PSMA-617 lutetium are going to result in more regulatory approvals for less heavily pretreated patients than were present in VISION.

There's also another phase 3 trial called the SPLASH trial (NCT04647526) using a PSMA-targeted radiopharmaceutical. Again, [it is] Lu-177, but this time instead of PSMA-617, it is 177 Lu-PSMA-I&T.

The[re] are additional phase 3 trials in the mCRPC nonchemotherapy-pretreated space. These trials are not quite underway to the same degree that PSMAfore is. Nevertheless, I think they can add value as we move forward.

TARGETED ONCOLOGY: If approved, how might Lu-PSMA fit within the current treatment landscape for mCRPC? What challenges do you anticipate for the use or acceptance of this agent?

SARTOR: I think the first label will be in accordance with the VISION-selected patients: mCRPC by conventional imaging and prior treatment with both a novel hormone such as abiraterone or enzalutamide and at least 1 taxane. Everybody would need to be PSMA-positive on the PET scan in accordance with the criteria that we established in VISION, I anticipate. That might not be the case, but I suspect it will.

After approval, the barriers are going to be severalfold. No. 1, there are going to be a lot of patients who do not want chemotherapy and are not treated with chemotherapy. They're going to be frustrated that they can't get this agent because the FDA and other regulatory bodies, I think, are going to require the chemotherapy pre-treatment. That's going to be 1 issue, [and] that's going to be addressed with PSMAfore and others.

No. 2, there are already access issues in the United States for PSMA PET. Not all of the insurance companies have approved it. If a PSMA PET [scan] is required, then somehow all these PSMA PET scans are going to have to be performed. That's a potential holdup.

No. 3, I think that the specialties that are qualified to administer the radiopharmaceuticalseither nuclear medicine or radiation oncologymay be overwhelmed with the demand. I'm worried that not enough centers are going to be ready. Ideally, these patients should be under multidisciplinary care. These are individuals who have multiple potential complications. It's not just pushing an isotope and seeing the patient back in 6 weeks. Multidisciplinary care is optimal. However, getting these patients through multi-d[isciplinary] clinics [to be sent] to those who are qualified to administer the therapy and then ensuring that theyve had chemotherapy and getting them [PSMA] PET scans could all be a hindrance.

There are stumbling blocks that could be apparent, and I think we're going to have to watchall of these as we go forward.

TARGETED ONCOLOGY: Looking beyond Lu-PSMA and the VISION trial, what do you think is most important for clinicians to emphasize in future efforts to treat patients with advanced prostate cancer effectively?

SARTOR: No. 1, we really need to start multidisciplinary care as soon as possible. Everybody can add value to the patient. If a patient is seeing a radiation oncologist, involving a urologist may be of benefit. If somebody is seeing a urologist, a medical oncologist could be helpful. As we move forward, particularly in these complex cases of patients with multiple areas of metastatic disease, coming together as teams can play an important role.

No. 2, we need to be aware of genetics. There are genetically targeted therapies now available. Folks are aware of the PARP inhibitors for homologous recombination repair defects, but things like pembrolizumab are also important. I mention pembrolizumab by name as a PD-1 inhibitor because this is approved in the context of mismatch repair or microsatellite instability (MSI)-high alterations, or even high tumor mutational burdens. Genetic testing is something I think we need to keep in mind, because sometimes the patients can have very robust responses to targeted therapies, provided they have the appropriate genetic milieu.

No. 3, as we move forward, we have to be cognizant of supportive aspects of our care, such as bone health. We have realized that a lot of patients can have pathogenic fractures and pathologic fractures. Mitigating that risk with things like denosumab or zoledronic acid is an important role for our clinicians to play in the management of patients.

TARGETED ONCOLOGY: What are the most exciting or important areas for researchers in this field to focus on?

SARTOR: Im excited about several areas. No. 1 is combination therapies. Currently, PSMA [Lu]-177 is being evaluated in combination with things like PSMA actinium-225. It's being looked at in combination with DNA repair inhibitors such as the PARP inhibitor olaparib, it's being looked at in combination with the PD-1 inhibitors like pembrolizumab, [and] its being evaluated in combination with stereotactic body radiotherapy. As we move forward, combination therapies are important.

Additional isotopes, combinations of isotopes, bispecific antibodies, and novel hormonal targeting agents that are being developed are also exciting, so there's a lot for us to keep aware of as this field marches forward.

TARGETED ONCOLOGY: PSMA-based radiotracers are the latest in a line of biomarkers used in prostate cancer imaging. Do you foresee other biomarkers becoming relevant? What role might PSMA have alongside them?

SARTOR: Combinations of PET imaging may yield very interesting results. For instance, we're having trouble treating emerging neuroendocrine prostate cancer. Often, after previous treatment with agents like abiraterone and enzalutamide, these neuroendocrine phenotypes emerge. The cell surface markers for neuroendocrine phenotypes may be very interesting. I'll mention the bombesin receptor as one. It turns out that these neuroendocrine tumors express receptors beyond just PSMA.

I think PSMA is a fabulous target, by the way, [but] different ways to image PSMA may also be important. There [are] also image-based biomarkers related to the use of immunotherapy. Being able to image things like PD-L1 [may] also [be] quite important.

As we go forward, Im seeing a whole series of newer PET [bio]markers being evaluated, and utility [may be] growing out of even combinations. The Australians today use 18 F-FDG PET in combination with PSMA PET, and by the way, I think that could potentially add value, but it needs to be properly evaluated in the context of prospective trials.

TARGETED ONCOLOGY: What is on the short-term horizon for research in prostate cancer treatment?

SARTOR: I think the short-term horizon in prostate cancer is going to revolve [around] moving these novel radiopharmaceuticals closer to the front of therapy. I've mentioned several trials, including the SPLASH trial, the PSMAfore trial, and the PSMA addition trial, which is for castration-sensitive [prostate cancer], upfront. All of these are going to be actively accruing subjects. I don't think we'll have results in the next 12 to 18 months, but nevertheless, that's going to be the next chain. In addition, we're going to see the rise of these combination therapies initially in phase 1 moving on to phase 2. And then I think we're going to evolve a whole series of novel biomarkers, and these are going to require additional testing, of course, but the field of biomarkers is alive and well. [It is] evolving so, so rapidly right now.

TARGETED ONCOLOGY: As phenotypic theranostics advance, what might be the role of genotypic precision medicine in prostate cancer? Do you think that these 2 areas will grow alongside one another?

SARTOR: I do. When we talk about precision medicine, I think most [but not all] of what weve become accustomed to is related to the genomic alterations that occur in the context of cancer, but phenotypic alterations such as PSMA expression [are] not going to be something you can detect with a gene rearrangement. Its really about protein expression. I think this area also has a bright future. I mentioned very particularly the expression of neuroendocrine markers. I mentioned the bombesin receptor, which is a gastrin-releasing peptide (GRP) receptor. Maybe somatostatin receptors could be important. Maybe other alterations such as DLL3 could be important. These would be called phenotypic biomarkers as opposed to genotypic biomarkers, which would be things like BRCA2 mutations, mismatch repair, rearrangements, etc.

Precision medicine is going to evolve, I think, on multiple fronts. The beauty of a targeted radiopharmaceutical is that almost anything that you can bind to on the cell surface potentially becomes a target. That means were going to have a vastly expanded series, in my opinion, over the next several decades of targets on cell surfacesnot just for prostate cancer, but for a whole series of different cancers. Right now, we have neuroendocrine cancers of the midgut, the so-called carcinoids, and those neuroendocrine-type cancers that are targeted, but I envision many, many more theranostics going forward, and [LuPSMA] is just a first step.

References

1. Sartor O, de Bono J, Chi KN, et al. Lutetium-177-PSMA-617 for metastatic castration-resistant prostate cancer.N Engl J Med. 2021;385(12):1091-1103. doi:10.1056/NEJMoa2107322

2. FDA grants priority review for investigational targeted radioligand therapy 177Lu-PSMA-617 for patients with metastatic castration-resistant prostate cancer (mCRPC). News release. Novartis. September 28, 2021. Accessed December 9, 2021. https://www.novartis.com/news/fda-grants-priority-review-investigational-targeted-radioligand-therapy-177lu-psma-617-patients-metastatic-castration-resistant-prostate-cancer-mcrpc

Originally posted here:
EP. 6B: Phenotypic Theranostics in the Future of Precision Medicine - Targeted Oncology

MODY: A Rare but Increasingly Common Form of Diabetes – Healthline

By Gene Therapy Clinics

It wasnt until a quarter century after being diagnosed with type 1 diabetes (T1D) that Lori Salsbury in Arkansas realized the condition shed been living with since she was 15 years old might not be what she thought it was.

Though her mom and sister were both initially misdiagnosed with type 2 diabetes (T2D) and later correctly dubbed T1Ds, Lori didnt have a reason at first to be suspicious of her own T1D diagnosis. Not until 2015, when she began seeing more people with diabetes sharing their stories online and realized something was off for her.

Sure, there is a mantra in our community that Your Diabetes May Vary. But for Salsbury, the particulars of her T1D just didnt match what she saw others in the D-Community sharing or what doctors and nurses described as the symptoms most newly diagnosed T1D experience.

At the time of her diagnosis, Salsbury was in her mid-20s and seemed quite healthy. She didnt get nauseous or sick, even a full day after missing an insulin dose. Her insulin dosing needs would change frequently, often sending her into super high glucose levels for weeks until adjusting her insulin or carb ratios; the same would happen on the low end of the scale.

One day, Salsbury heard about a rare, inherited form of diabetes called MODY (maturity onset diabetes of the young), that doesnt require as much insulin, at least initially. That piqued her interest.

She did some online research, and then consulted her endocrinologist and received antibody tests that came back negative. He also ran a C-peptide test that came back at T1D levels, but that was most likely due to her 20+ years of using insulin. A referral to a geneticist led to more bloodwork, and in January 2020 the findings came back showing a genetic mutation, which causes one of the several different known types of MODY.

MODY has the potential of changing how you manage your diabetes, depending on the particular form youre diagnosed with. Some changes could include stopping medications completely or changing from insulin to a different injectable or oral medication, while some MODY forms mandate changes in your diet.

In Salsburys case, the MODY diagnosis brought her some clarity, and finally an explanation of why her diabetes experience seemed so different than others in the T1D community. But she continues insulin therapy.

Since I was originally diagnosed T1D, I am still (labeled that) in my charts so that I wont lose coverage for my insulin pump and CGM that I require to live by, Salsbury said. Most often, if asked I just tell people that I was diagnosed with type 1. Its easier than going through the whole What is MODY? spiel.

The easiest way to think about MODY is that its a subset of diabetes caused by a mutation in one of at least 14 genes in a persons DNA. That mutation impacts the insulin-producing beta cells, which in turn impacts insulin production and glucose regulation.

Since just an estimated 1 to 2 percent of those with diabetes have a genetic mutation leading to MODY, there isnt much discussion about it within the patient community, and most medical professionals dont bring it up unless they are questioned. Yet some advocates and researchers believe the various types of MODY are more common than many think, and that view is becoming more accepted as genetic testing becomes more widely available.

The term MODY was first coined in the 1970s by pioneering researchers who identified what appeared to be a mild form of diabetes in children that didnt necessarily require insulin as was needed for those with the more common juvenile diabetes (before it was later renamed type 1). At that time, MODY was defined as fasting hyperglycemia diagnosed under age 25 which could be treated without insulin for more than two years, and it is inherited, as they found.

While most research existing shows its as rare as 1 to 2 percent of all diabetes cases, more current research now indicates that as many as 6.5 percent of children with antibody-negative diabetes may have a form of MODY.

MODY is passed down genetically from parent to child, making that the common thread for this form of diabetes compared to the other types that are autoimmune, partially genetic, or more lifestyle-based. The typical diagnosis comes before age 25, and its rarely diagnosed in those older than 35 or 40. While children have roughly a 50 percent chance of developing MODY if one of their parents has it, that does not mean mutations cant occur at random and appear in those without a family history of gene mutation.

The gene mutations arent the same for everyone, and they affect different organs in the body, meaning its difficult to diagnose without genetic testing, and it can be more challenging to recognize glucose fluctuations commonly found in those who are newly diagnosed.

Significantly, 80 percent of MODY cases are misdiagnosed as T1D or T2D as the signs are pretty much the same extreme thirst, increased urination, and weight loss. But some forms of MODY do not produce any symptoms. The number of misdiagnoses may be even higher at 95 percent in the United States, according to some researchers.

Dr. Miriam Udler at Massachusetts General Hospital is one of the more well-known names in MODY clinical research. She believes more cases are being diagnosed in recent years as genetic testing has become more available, particularly after COVID-19 led to a telehealth explosion and more at-home testing kits for bloodwork and diagnostic tests normally done in a lab.

Dr. Miriam Udler

It used to be rare and expensive, and that was a barrier to testing and diagnosing MODY correctly, she told DiabetesMine. But now, more providers have access to this and can order the tests to their clinics or patients at home, and insurance is increasingly covering MODY genetic testing.

While MODY is still less common and infrequently discussed in clinics, Udler says it comes down to that particular doctor or patient recognizing something might be different about their diabetes.

That matters a lot, and a correct diagnosis can change management, Udler said. In most common MODY forms, it could mean coming off medication.

For Salsbury, the particular BLK gene mutation she has causes MODY 11, an insulin secretion defect that makes her beta cells less responsive to glucose and leads to less insulin being sent out by the body when its needed. Being overweight is one common feature of this particular gene mutation, according to research.

Once MODY is recognized and diagnosed, it can also be difficult to regulate glucose levels in the same ways that T1D and T2Ds often do, because the symptoms and glucose levels can vary significantly.

As MODY 11 usually presents like T1D and is treated in much the same way, Salsbury has been using insulin since she was diagnosed at age 15 in 1991 and wears an Omnipod tubeless insulin pump and Dexcom CGM, combined into a homemade do-it-yourself (DIY) closed loop system. For her, life with MODY isnt much different from being T1D.

But she knows everyone is not as fortunate on that front and can have many challenges in getting a correct diagnosis and finding a management routine that works for their particular form of MODY.

In New York, Laurie Jones shares her story of being diagnosed at 30 with gestational diabetes late in her first pregnancy through the test often given to pregnant women. She changed her diet and followed it to the letter on exact carb and calorie allowances, and took varying doses of long and short-acting insulins. Though she describes it as intense, all signs of diabetes went away after her first pregnancy.

But within a few years during her second pregnancy, gestational diabetes returned. She began insulin injections right away as well as a strict diet, but Jones found it more difficult than before to regulate high and low blood sugars.

A number of years later, her A1C results were creeping higher and that led to a T2D diagnosis. She took Metformin on the advice of her doctor, but it didnt work to keep her blood sugars in check.

Most adult medicine endos do not push for MODY testing even when the medicine is not working, she explained. Being overweight is usually assumed the reason, therefore even star doctors dont push for MODY testing unless weight is lost.

Her sons diagnosis changed everything. When he was 6 years old, he was diagnosed with eosinophilic esophagitis, and that mandated a diet free of the top allergens. He was about 12 when she took him to an endocrinologist, as he was not growing and low on the weight scale and didnt show any signs of puberty. That endo noticed his blood sugars were elevated and assumed he was in the honeymoon period prior to becoming a fully diagnosed T1D.

Months progressed, and the doctor suggested it was MODY. Genetic testing led to a MODY 2 diagnosis.

We had no idea what that was, and before [the doctor] explained it to us, she noted that most endocrinologists and almost all doctors outside of major medical teaching and research hospitals have not heard of it, the D-Mom said.

After her sons diagnosis, Jones got her own genetic testing and learned she also had MODY 2.

Most controlled by diet, MODY 2 is one of the more common but less intensive forms of MODY that usually doesnt require insulin or other glucose-lowering meds.

That led her to stopping Metformin, and shes been eating healthier and managing her weight for better glucose levels.

MODY 2 is not just about how you produce or use insulin, but mainly when you produce the insulin, she said. We were both told that our pancreas is like a house cooling or heating system that is off-kilter. Basically, our sugar levels have to get much higher than what is considered normal before the pancreas produces insulin. There are also insulin efficiency issues.

Without her sons diagnosis, Jones doesnt think she wouldve ever had the needed genetic testing and would have remained with a T2D diagnosis taking the wrong medications.

Thats likely the story for so many people in our D-Community, she believes.

With a 50 percent chance of being passed on, chances are MODY is not as rare as it is now believed, Salsbury said. If more people knew of it and were tested, we may come to find out that it is the most common or second only to T2D in commonality.

Importantly, a correct MODY diagnosis can highlight other health issues that might potentially arise. For example, a MODY 11 mutation to the BLK gene can increase the chances of developing systemic lupus erethematosus (SLE).

While being correctly diagnosed as MODY may not change your treatment, it can give you other information, Salsbury said. Many forms of MODY also come along with other health issues that the mutation may have caused. Knowing you have MODY can alert your doctors to watch you or check you for other related health conditions.

Researchers note the same, including Dr. Toni Pollin, a genetic researcher and counselor who in 2016 co-founded the Monogenic Diabetes Research and Advocacy Project (MDRAP) at the University of Maryland School of Medicine. The MDRAP effort promotes the correct diagnosis of MODY and also helps raise money for that effort. She co-founded MDRAP with a patient advocate whod been diagnosed with a form of MODY.

While improving MODY diagnosis will certainly improve the clinical care for patients, it will also have broader implications, researchers wrote in this 2015-published Undiagnosed MODY: Time for Action manuscript. Screening and genetic testing for MODY among patients with diabetes will provide a model for identifying and diagnosing highly penetrant forms of other otherwise common complex diseases [through] the power of genetics and genomics for improving patient care and public health.

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MODY: A Rare but Increasingly Common Form of Diabetes - Healthline

Eggschain, Pioneering Chain of Custody Solution for Fertility Treatments and Other Biospecimens, Officially First Healthcare Biotech on Bitcoin…

By Stem Cell Clinics

AUSTIN, Texas--(BUSINESS WIRE)--Eggschain, the pioneering authority in blockchain-based digital chain of custody tracking of blood, genome, tissues, organs, DNA, RNA, sperm, eggs, embryos, and other biospecimens, is making history again.

On November 24th, 2021, Eggschain founder and CEO Wei Escala made history by storing her eggs on the blockchain, doing so through the platform she created. Working with Dr. Hugh Taylor, Chair of Obstetrics, Gynecology and Reproductive Sciences at Yale School of Medicine, his team of physicians and nurses, as well as the Eggschain team, Wei underwent egg retrieval on the 24th of November. She entered her data through the Eggschain platform and broadcasted to the blockchain, once Yale School of Medicine verified the accuracy of the data on the same day through the platform.

This is an exciting milestone for the field of fertility preservation, said Dr. Taylor. A reliable and confidential tracking system will allow us to assure the safety and quality of frozen reproductive tissue. Eggschain enables greater transparency and security for patients and health professionals.

Earlier this month, Eggschain successfully generated a bitcoin cryptographic block hash, becoming the first biotech healthcare company on the mainnet. The first patented supply chain system that combines blockchain and genetics to bring secure, transparent, and professional grade information and data to individuals undergoing IVF. Eggschain was developed on Stacks, a collection of independent entities, developers, and community members working to build a user-owned internet on Bitcoin.

Eggschain will mark these dual milestones with an NFT drop that celebrates the arrival of Babies on the Blockchain. The first NFT, Into the Light, shows the meeting of sperm and egg as a light projection on a dark sky, an evocative illustration of the art and science of IVF and the potential for future life. Eggschain will open a whitelist with 2,000 spots in early 2022, with approximately 2,500 to 10,000 NFTs to be released.

Fertility treatment is emotionally and physically demanding, often creating stress and a sense of isolation along the way, said Ms. Escala. By sharing my Eggschain experience, I hope to begin to build a sense of community that help others undergoing fertility treatments feel less alone and more empowered. By delivering medical grade information in a secure and universally trackable format, Eggschain provides transparency and peace of mind, holding the potential to forever change that dynamic for the better.

For further information about Eggschain, please visit https://eggschain.com/.

About Eggschain, INC.

Eggschain is a healthcare technology company and thought leader in the fertility, health tech, family-building and cryogenic preservation industries. Patent-granted and cloud-based, Eggschain is the first blockchain-integrated inventory management and chain of custody technology for tracking biospecimens, including sperm, eggs, embryos, genome, stem cell, tissues and organs, and other genetic material. In partnership with several of the worlds leading experts in reproductive biology, endocrinology and high complexity labs, Eggschain delivers medical grade information and data to individuals undergoing IVF in a secure, transparent and universally trackable format and also provides the means for clinics to monitor their inventory, either personal, such as oocytes and sperm, or lab-related, including equipment inventory and maintenance scheduling. At its core, Eggschain strives to enable better decision-making, preserving the hope of life and helping to advance humanity.

About Stacks

Secure smart contracts and apps for Bitcoin. The Stacks ecosystem is a collection of independent entities, developers, and community members working to build a user-owned internet on Bitcoin. The Stacks 2.0 blockchain extends the design of Bitcoin to enable secure apps and predictable Clarity smart contracts without modifying Bitcoin itself, opening innovation on the network for the first time. The Stacks cryptocurrency (STX) is used as fuel for networking activity and contract execution and can be locked by STX holders via Stacking to earn Bitcoin (BTC) rewards for supporting blockchain consensus. Stacks cryptocurrency was distributed to the general public through the first-ever SEC qualified token offering in U.S. history.

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Eggschain, Pioneering Chain of Custody Solution for Fertility Treatments and Other Biospecimens, Officially First Healthcare Biotech on Bitcoin...

Cancer Stem Cell Therapy Market opportunities, threats, main drivers, and key restraints and COVID-19 analysis to 2026 | Advanced Cell Diagnostics,…

By Stem Cell Clinic

Cancer Stem Cell Therapy Market

The report named Global Cancer Stem Cell Therapy Marketby Worldwide Market Reports offers a profound comprehension of the development and working of the global market on a worldwide just as a local premise. This qualitative report is the gathering of all the extensive information relevant to the market elements over the previous years alongside a few estimates. First and foremost, the market report incorporates the key market players including producers, organizations, associations, providers, etc. This will help the purchasers to understand the methodologies and activities taken up by these players to set a solid foot and spotlight on battle rivalry inside the worldwide Cancer Stem Cell Therapy market.

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The Cancer Stem Cell Therapy Market Report is an accurate and thorough cut-out study aimed at key drivers, market strategies, and the huge growth of the major players. Globally, Cancer Stem Cell Therapy Industry also provides in-depth studies of dynamics, segmentation, revenue, and stock forecasting, enabling you to make superior business decisions. The report provides compelling statistics on the market size of major manufacturers and is a vital source of advice and guidance for companies and individuals operating in the Cancer Stem Cell Therapy industry.

Competitive Section:

Major Key Players in the Cancer Stem Cell Therapy Market:

AVIVA BioSciences, AdnaGen, Advanced Cell Diagnostics, Silicon Biosystems

NOTE: Consumer behavior has changed within all sectors of society amid the COVID-19 pandemic. Industries on the other hand will have to restructure their strategies in order to adjust to the changing market requirements. This report offers you an analysis of the COVID-19 impact on the Cancer Stem Cell Therapy market and will help you in strategizing your business as per the new industry norms.

COVID-19 Impact

The report covers the Impact of Coronavirus COVID-19: Since the COVID-19 virus outbreak in December 2019, the disease has spread to almost every country around the globe with the World Health Organization declaring it a public health emergency. The global impacts of the coronavirus disease 2019 (COVID-19) are already starting to be felt, and will significantly affect the Cancer Stem Cell Therapy market in 2021. The outbreak of COVID-19 has brought effects on many aspects, like flight cancellations; travel bans, and quarantines; restaurants closed; all indoor/outdoor events restricted; over forty countries state of emergency declared; massive slowing of the supply chain; stock market volatility; falling business confidence, growing panic among the population, and uncertainty about future.

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Regional Analysis:

The report provides information about the market area, which is further subdivided into sub-regions and countries. In addition to market share in each country and subregion, this chapter of this report also provides information on profit opportunities. This chapter of the report mentions the share and market growth rate of each region, country, and sub-region in the estimated time period.

North America (USA, Canada) Europe (Germany, France, UK, Italy, Russia, Spain, Netherlands, Switzerland, Belgium) Asia Pacific (China, Japan, Korea, India, Australia, Indonesia, Thailand, Philippines, Vietnam) The Middle East and Africa (Turkey, Saudi Arabia, UAE, South Africa, Israel, Egypt, Nigeria) Latin America (Brazil, Mexico, Argentina, Colombia, Chile, Peru).

By Type

Autologous Stem Cell Transplants, Allogeneic Stem Cell Transplants, Syngeneic Stem Cell Transplants, Others

By Application

Hospital, Clinic, Medical Research Institution, Others

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Some of the crucial questions answered in the professional intelligence study on the Cancer Stem Cell Therapy market include:

Which key regions are likely to have the largest share of the Cancer Stem Cell Therapy market? What are the potential obstacles for new players looking to enter the market? What changes has consumer buying behavior observed during the Covid-19 pandemic? Which end consumer industries are likely to drive the demand in the Cancer Stem Cell Therapy market during the forecast period? Which countries are among the main consumers or manufacturers of the Cancer Stem Cell Therapy market? What are the threats and opportunities for stakeholders and market players? Which regions offer lucrative investment opportunities for industry players in the Cancer Stem Cell Therapy Market? What is the type of competition in the market? Which large established companies have the largest share of the Cancer Stem Cell Therapy market? What strategies are these key players pursuing to maintain their dominant position in the Cancer Stem Cell Therapy market?

Please contact us if you would like more information about the report. If you have any special requirements and would like customization, please let us know. We will then offer the report as you wish.

Key Indicators Analysed:

Market Players & Competitor Analysis: The report covers the key players of the industry including Company Profile, Product Specifications, Production Capacity/Sales, Revenue, Price and Gross Margin 2016-2021 & Sales by Product Types.

Global and Regional Market Analysis: The report includes Global & Regional market status and outlook 2022-2026. Further, the report provides breakdown details about each region & countries covered in the report. Identifying its production, consumption, import & export, sales volume & revenue forecast.

Market Analysis by Product Type: The report covers the majority of Product Types in the Cancer Stem Cell Therapy Industry, including its product specifications by each key player, volume, sales by Volume, and Value (M USD).

Market Analysis by Application Type: Based on the Cancer Stem Cell Therapy Industry and its applications, the market is further sub-segmented into several major Applications of its industry. It provides you with the market size, CAGR & forecast by each industry application.

Market Trends: Market key trends include Increased Competition and Continuous Innovations.

Opportunities and Drivers: Identifying the Growing Demands and New Technology

Porters Five Force Analysis: The report will provide the state of competition in the industry depends on five basic forces: the threat of new entrants, bargaining power of suppliers, bargaining power of buyers, threat of substitute products or services, and existing industry rivalry.

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Cancer Stem Cell Therapy Market opportunities, threats, main drivers, and key restraints and COVID-19 analysis to 2026 | Advanced Cell Diagnostics,...

Stem cells: Sources, types, and uses – Medical News Today

By Embryonic Stem Cells

Cells in the body have specific purposes, but stem cells are cells that do not yet have a specific role and can become almost any cell that is required.

Stem cells are undifferentiated cells that can turn into specific cells, as the body needs them.

Scientists and doctors are interested in stem cells as they help to explain how some functions of the body work, and how they sometimes go wrong.

Stem cells also show promise for treating some diseases that currently have no cure.

Stem cells originate from two main sources: adult body tissues and embryos. Scientists are also working on ways to develop stem cells from other cells, using genetic reprogramming techniques.

A persons body contains stem cells throughout their life. The body can use these stem cells whenever it needs them.

Also called tissue-specific or somatic stem cells, adult stem cells exist throughout the body from the time an embryo develops.

The cells are in a non-specific state, but they are more specialized than embryonic stem cells. They remain in this state until the body needs them for a specific purpose, say, as skin or muscle cells.

Day-to-day living means the body is constantly renewing its tissues. In some parts of the body, such as the gut and bone marrow, stem cells regularly divide to produce new body tissues for maintenance and repair.

Stem cells are present inside different types of tissue. Scientists have found stem cells in tissues, including:

However, stem cells can be difficult to find. They can stay non-dividing and non-specific for years until the body summons them to repair or grow new tissue.

Adult stem cells can divide or self-renew indefinitely. This means they can generate various cell types from the originating organ or even regenerate the original organ, entirely.

This division and regeneration are how a skin wound heals, or how an organ such as the liver, for example, can repair itself after damage.

In the past, scientists believed adult stem cells could only differentiate based on their tissue of origin. However, some evidence now suggests that they can differentiate to become other cell types, as well.

From the very earliest stage of pregnancy, after the sperm fertilizes the egg, an embryo forms.

Around 35 days after a sperm fertilizes an egg, the embryo takes the form of a blastocyst or ball of cells.

The blastocyst contains stem cells and will later implant in the womb. Embryonic stem cells come from a blastocyst that is 45 days old.

When scientists take stem cells from embryos, these are usually extra embryos that result from in vitro fertilization (IVF).

In IVF clinics, the doctors fertilize several eggs in a test tube, to ensure that at least one survives. They will then implant a limited number of eggs to start a pregnancy.

When a sperm fertilizes an egg, these cells combine to form a single cell called a zygote.

This single-celled zygote then starts to divide, forming 2, 4, 8, 16 cells, and so on. Now it is an embryo.

Soon, and before the embryo implants in the uterus, this mass of around 150200 cells is the blastocyst. The blastocyst consists of two parts:

The inner cell mass is where embryonic stem cells are found. Scientists call these totipotent cells. The term totipotent refer to the fact that they have total potential to develop into any cell in the body.

With the right stimulation, the cells can become blood cells, skin cells, and all the other cell types that a body needs.

In early pregnancy, the blastocyst stage continues for about 5 days before the embryo implants in the uterus, or womb. At this stage, stem cells begin to differentiate.

Embryonic stem cells can differentiate into more cell types than adult stem cells.

MSCs come from the connective tissue or stroma that surrounds the bodys organs and other tissues.

Scientists have used MSCs to create new body tissues, such as bone, cartilage, and fat cells. They may one day play a role in solving a wide range of health problems.

Scientists create these in a lab, using skin cells and other tissue-specific cells. These cells behave in a similar way to embryonic stem cells, so they could be useful for developing a range of therapies.

However, more research and development is necessary.

To grow stem cells, scientists first extract samples from adult tissue or an embryo. They then place these cells in a controlled culture where they will divide and reproduce but not specialize further.

Stem cells that are dividing and reproducing in a controlled culture are called a stem-cell line.

Researchers manage and share stem-cell lines for different purposes. They can stimulate the stem cells to specialize in a particular way. This process is known as directed differentiation.

Until now, it has been easier to grow large numbers of embryonic stem cells than adult stem cells. However, scientists are making progress with both cell types.

Researchers categorize stem cells, according to their potential to differentiate into other types of cells.

Embryonic stem cells are the most potent, as their job is to become every type of cell in the body.

The full classification includes:

Totipotent: These stem cells can differentiate into all possible cell types. The first few cells that appear as the zygote starts to divide are totipotent.

Pluripotent: These cells can turn into almost any cell. Cells from the early embryo are pluripotent.

Multipotent: These cells can differentiate into a closely related family of cells. Adult hematopoietic stem cells, for example, can become red and white blood cells or platelets.

Oligopotent: These can differentiate into a few different cell types. Adult lymphoid or myeloid stem cells can do this.

Unipotent: These can only produce cells of one kind, which is their own type. However, they are still stem cells because they can renew themselves. Examples include adult muscle stem cells.

Embryonic stem cells are considered pluripotent instead of totipotent because they cannot become part of the extra-embryonic membranes or the placenta.

Stem cells themselves do not serve any single purpose but are important for several reasons.

First, with the right stimulation, many stem cells can take on the role of any type of cell, and they can regenerate damaged tissue, under the right conditions.

This potential could save lives or repair wounds and tissue damage in people after an illness or injury. Scientists see many possible uses for stem cells.

Tissue regeneration is probably the most important use of stem cells.

Until now, a person who needed a new kidney, for example, had to wait for a donor and then undergo a transplant.

There is a shortage of donor organs but, by instructing stem cells to differentiate in a certain way, scientists could use them to grow a specific tissue type or organ.

As an example, doctors have already used stem cells from just beneath the skins surface to make new skin tissue. They can then repair a severe burn or another injury by grafting this tissue onto the damaged skin, and new skin will grow back.

In 2013, a team of researchers from Massachusetts General Hospital reported in PNAS Early Edition that they had created blood vessels in laboratory mice, using human stem cells.

Within 2 weeks of implanting the stem cells, networks of blood-perfused vessels had formed. The quality of these new blood vessels was as good as the nearby natural ones.

The authors hoped that this type of technique could eventually help to treat people with cardiovascular and vascular diseases.

Doctors may one day be able to use replacement cells and tissues to treat brain diseases, such as Parkinsons and Alzheimers.

In Parkinsons, for example, damage to brain cells leads to uncontrolled muscle movements. Scientists could use stem cells to replenish the damaged brain tissue. This could bring back the specialized brain cells that stop the uncontrolled muscle movements.

Researchers have already tried differentiating embryonic stem cells into these types of cells, so treatments are promising.

Scientists hope one day to be able to develop healthy heart cells in a laboratory that they can transplant into people with heart disease.

These new cells could repair heart damage by repopulating the heart with healthy tissue.

Similarly, people with type I diabetes could receive pancreatic cells to replace the insulin-producing cells that their own immune systems have lost or destroyed.

The only current therapy is a pancreatic transplant, and very few pancreases are available for transplant.

Doctors now routinely use adult hematopoietic stem cells to treat diseases, such as leukemia, sickle cell anemia, and other immunodeficiency problems.

Hematopoietic stem cells occur in blood and bone marrow and can produce all blood cell types, including red blood cells that carry oxygen and white blood cells that fight disease.

People can donate stem cells to help a loved one, or possibly for their own use in the future.

Donations can come from the following sources:

Bone marrow: These cells are taken under a general anesthetic, usually from the hip or pelvic bone. Technicians then isolate the stem cells from the bone marrow for storage or donation.

Peripheral stem cells: A person receives several injections that cause their bone marrow to release stem cells into the blood. Next, blood is removed from the body, a machine separates out the stem cells, and doctors return the blood to the body.

Umbilical cord blood: Stem cells can be harvested from the umbilical cord after delivery, with no harm to the baby. Some people donate the cord blood, and others store it.

This harvesting of stem cells can be expensive, but the advantages for future needs include:

Stem cells are useful not only as potential therapies but also for research purposes.

For example, scientists have found that switching a particular gene on or off can cause it to differentiate. Knowing this is helping them to investigate which genes and mutations cause which effects.

Armed with this knowledge, they may be able to discover what causes a wide range of illnesses and conditions, some of which do not yet have a cure.

Abnormal cell division and differentiation are responsible for conditions that include cancer and congenital disabilities that stem from birth. Knowing what causes the cells to divide in the wrong way could lead to a cure.

Stem cells can also help in the development of new drugs. Instead of testing drugs on human volunteers, scientists can assess how a drug affects normal, healthy tissue by testing it on tissue grown from stem cells.

Watch the video to find out more about stem cells.

There has been some controversy about stem cell research. This mainly relates to work on embryonic stem cells.

The argument against using embryonic stem cells is that it destroys a human blastocyst, and the fertilized egg cannot develop into a person.

Nowadays, researchers are looking for ways to create or use stem cells that do not involve embryos.

Stem cell research often involves inserting human cells into animals, such as mice or rats. Some people argue that this could create an organism that is part human.

In some countries, it is illegal to produce embryonic stem cell lines. In the United States, scientists can create or work with embryonic stem cell lines, but it is illegal to use federal funds to research stem cell lines that were created after August 2001.

Some people are already offering stem-cells therapies for a range of purposes, such as anti-aging treatments.

However, most of these uses do not have approval from the U.S. Food and Drug Administration (FDA). Some of them may be illegal, and some can be dangerous.

Anyone who is considering stem-cell treatment should check with the provider or with the FDA that the product has approval, and that it was made in a way that meets with FDA standards for safety and effectiveness.

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Stem cells: Sources, types, and uses - Medical News Today

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