Stem Cell Clinic

Researchers to educate the public with Stem Cell Awareness Day – News 12 Bronx

For more than 15 years, Doctor Eric Bouhassira and his team have been researching stem cells at Albert Einstein College of Medicine.They say their mission is to use stem cells to create new red blood cells.

According to Dr. Bouhassira, the red blood cells could help many. He says it could help someone with sickle cell disease, who sometimes need a very rare blood type, which could be replaced in this way.

One way they are spreading the message is through Stem Cell Awareness Day. They are aiming to educate the public by hosting the event on Wednesday.

Jo Wiederhorn is the president and CEO of the Associated Medical Schools of New York and is advocating for research. The Associated Medical Schools of New York is a non-profit that represents all of the state medical schools.

The work being done in labs across the state involving stem cells in hopes of treating and curing conditions such as Alzheimer's, Parkinson's and various forms of cancer.

However, researchers say they need a lot of funding because the research is very expensive. They say the federal government no longer supports stem cell research.

Researchers say anything that has advanced the work they have been doing for the past 10 years has come from the state.

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Researchers to educate the public with Stem Cell Awareness Day - News 12 Bronx

To Find Therapies for COPD, Other Disorders, Researcher Awarded $7.5M – COPD News Today

Michael J. Holtzman, MD, has received close to $7.5 million in total funding for research aimed at developing stem cell-based treatments for chronic obstructive pulmonary disease (COPD), asthma, and other disorders.

Holtzmans research atWashington University School of Medicine in St. Louis identified a subset of stem cells cells that are able to grow into other more specialized types of cells that line the airways and help drive mucus production in the lungs.

Stem cells that give rise to mucus cells lining the airway and other sites are part of our immune defense strategy, Holtzman, the director of the Division of Pulmonary and Critical Care Medicine, said in a university press releasewritten by Julia Evangelou Strait.

These cells are activated by common respiratory viruses and other inhaled agents, and prevent airway injury and promote repair.

Once the problem is resolved, the [immune] system should go back to a normal baseline level. But in some people, the stem cell is changed in a way that continues to promote inflammation and mucus production and ultimately compromises airway function even for normal breathing, Holtzman said.

Thus, Holtzmans team is searching fortherapeutic targets to control this stem cell response.

The largest of the grants hes received at $6.6 million is the outstanding investigator award from the National Heart, Lung, and Blood Instituteof theNational Institutes of Health (NIH), given to researchers with proven expertise in innovative research and considered likely to make major advances. The grant will provide seven years of funding for research intended to further characterize these cells and their underlying mechanisms of action.

The award also supports ongoing efforts to identify pharmacological strategies to manipulate these stem cells. One lead compound has shown promise in animal models, preventing airway inflammation and mucus production after a respiratory viral infection.

Pending clearance from the U.S. Food and Drug Administration, clinical trials for this potential therapy are planned in people with COPD, asthma exacerbations, and related upper airway disorders.

Holtzman also received a NIH Small Business Technology Transfer (STTR) of $300,000 to support a startup company he launched in anticipation of the successful development of these treatments.

Besides lung diseases, Holtzman received another $300,000 STT and a $250,000 award from the Siteman Investment Program in support of a stem cell-targeting compound aimed at treating breast cancer.

Your first reaction might be to wonder how in the world such similar compounds could be effective in what seem to be such different tissues, Holtzman said. But airway and breast tissues and other related sites share secretory function and overlap in how this function is controlled.

As a result, he concluded, our compounds can be precisely tailored to address whether the dysregulated stem cell is in airway versus breast tissue, or other sites as well.

Marisa holds an MS in Cellular and Molecular Pathology from the University of Pittsburgh, where she studied novel genetic drivers of ovarian cancer. She specializes in cancer biology, immunology, and genetics. Marisa began working with BioNews in 2018, and has written about science and health for SelfHacked and the Genetics Society of America. She also writes/composes musicals and coaches the University of Pittsburgh fencing club.

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Patrcia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.

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To Find Therapies for COPD, Other Disorders, Researcher Awarded $7.5M - COPD News Today

Drug candidate identified to starve and suffocate breast cancer stem cells – Health Europa

Breast Cancer is one the most common types of cancer in the UK. It is predicted that 1 in 7 women will be affected by the illness in their lifetime.

Research at the University of Salford has uncovered important findings on how to effectively target mitochondria, which normally provide all the necessary energy for driving the proliferation and dissemination of cancer stem cells.

It is well-recognised, by the medical community, that the growth of cancer stem cells (CSCs) is one of the major causes of treatment failure, tumour recurrence and cancer spread, in many different cancer types.

CSCs that are resistant to chemotherapy and radiotherapy, often resulting in tumour recurrence. In research conducted at the Translational Medicine Laboratory at the University of Salford, a candidate drug was identified,Dodecyl-TPP, that was found to be effective when targeting mitochondria within CSCs. Using this approach when treating cancer patients has the scope to reduce the risk of recurrence and spread of cancer.

Professor Michael P. Lisanti, the Chair of Translational Medicine at The University of Salford said: Our pre-clinical research has identified a new drug candidate for targeting mitochondria in CSC.

It was found thatDodecyl-TPPtreatment can potently starve CSCs to death, this being effective in the nano-molar range, blocking their use of oxygen to generate energy in the form of ATP. The findings make a valid case for future clinal trials in this area.

Scientists at the University of Salford also identified five other agents that worked together with Dodecyl-TPP. This includes two FDA-approved drugs (DoxycyclineandNiclosamide) and two nutraceuticals (Vitamin CandBerberine).

The research, led by Professors Michael P. Lisanti and Federica Sotgia, was published in the journalFrontiers in Oncology, a peer reviewed research platform that covers cancer research. This metabolic approach using drug combinations, can potentially improve patient survival by preventing tumour recurrence and metastasis, via the high-efficiency targeting of CSCs.

Around 90% of all cancer patients die as a result of the cancer spreading and tumour recurrence, this being the basis of the research to identify new mitochondria inhibitors.

Mitochondria are the powerhouse of the cell, which drive the production of cellular energy in the form of NAD and ATP. Research has shown thatDodecyl-TPP acts as a mitochondrial inhibitor, starving the CSCs to death. ATP and NAD are high-energy metabolites required for cell survival and propagation.

Dodecyl-TPPcontains a 12-carbon side-chain which is tethered to a TPP (tri-phenyl-phosphonium) moiety. TPP acts as a chemical signal to target the drug to the mitochondria. TPP more efficiently targets mitochondria in cancer stem cells and cancer cells, but is largely excluded from normal cell mitochondria, reducing its potential for toxicity in normal body cells.

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Drug candidate identified to starve and suffocate breast cancer stem cells - Health Europa

Spotlight interview: 15 minutes on medical and scientific writing – PMLiVE

1.WHAT ARE THE KEY MEDICAL AND SCIENTIFIC WRITING ACTIVITIES THAT PHARMA IS CURRENTLY INVESTING IN?

We live in an era of high-science healthcare communications. The drugs and products we work on are becoming ever more complex, and mechanisms of action more difficult to understand. A number of current therapeutic approaches such as gene editing, gene therapy, and stem-cell therapy were not in existence when many healthcare professionals (HCPs) underwent their training, so there is a real role for medical communications professionals to educate HCPs so they can see the relevance of these advances to their clinical practice. In response to this, we have seen the increasing rise to prominence of medical affairs as a central function within pharmaceutical companies, providing a credible means of scientific exchange with HCPs. Pharma is also engaging with a broader range of stakeholders, moving beyond prescribers and HCPs to patients, caregivers, patient advocacy groups, payers, insurers and policy makers. Therefore, it is imperative that teams involved in medical communications create narratives to resonate with each audience, and ensure that their message is heard, and more importantly, has an impact. While advances in science can seem incredible at times, there remains a need to make the story personal to the audience and to create a clear so what? - we cant expect the science to tell the story for us.

We have long been recognised as leaders within the field of oncology, rare disease and specialised medicine and are used to partnering with experts in these rapidly-growing areas. We have correspondingly invested in specialist writers with very strong scientific backgrounds: our writing team comprises PhD's, medical doctors and pharmacists, all of whom have expertise in complex therapy areas, and of course, outstanding communications skills. Also, our OPEN Health values, which focus on themes such as ownership, excellence, collaboration and responsibility, have served to create a strong shared purpose within our teams. When we work together, we challenge each other to expect more from ourselves and our interactions. We routinely collaborate across disciplines within OPEN Health to bring in the right expertise to meet our clients' challenges and enable us to propose solutions that are fresh, purposeful and clinically relevant. Finally, the team demonstrates comfort and expertise with digital communications, which underpins all of our medical communications programmes.

As the medical communications industry grows and matures, the 'sink or swim' approach to training that was once prevalent is rapidly changing. Quite rightly, expectations of medical writers are changing, as they demand more from training and development. To this end, at OPEN Health, we have created a comprehensive training programme to help new medical writers navigate the scientific, editorial and compliance landscape of medical communications. This training is supplemented by strong relationships with more senior members of staff who provide feedback on writers work and who are truly invested in their development. My view is that there is no better place than OPEN Health for a new medical writer to get industry-leading training, outstanding opportunities to develop their skills, and the senior support to accelerate their careers. We're very proud of the fact that many of our senior writers have been with us a long time and have built their careers at OPEN Health.

Our vision is to be the most respected healthcare agency group on the planet. To me, high-quality medical writing is at the heart of this, and fortunately, we're starting from a very strong position. Our size and global reach means that we can draw upon a large team of hugely experienced medical and scientific writers, with expertise in their respective fields - medical affairs content, market access, real-world evidence, publication planning and execution, patient and brand communications, medical education and internal training. We combine this with rigorous internal review, watertight quality control processes, and above all, a shared commitment to, and understanding of, what quality healthcare communications look like.

With the recent consolidation of practices, and the merger with Peloton Advantage, we can benefit from and leverage the vast medical writing experience across the US and UK. Our collaborative approach facilitates cross-fertilisation of ideas, sharing of best practices and a growth mindset with regard to learning and development. We have long recognised the societal shift towards flexible working and have a mature approach to remote working that enables writers to do their best work, in their preferred setting, and at times that work for them. With expanding geographies, industry-leading training, and a strong support network, my vision is for OPEN Health to be recognised as the primary destination for ambitious medical and scientific writers who want to be part of the most respected medical writing team in the industry.

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Spotlight interview: 15 minutes on medical and scientific writing - PMLiVE

Novel Cell Sorting and Separation Markets, 2030 – P&T Community

DUBLIN, Oct. 7, 2019 /PRNewswire/ -- The "Novel Cell Sorting and Separation Market: Focus on Acoustophoresis, Buoyancy, Dielectrophoresis, Magnetophoretics, Microfluidics, Optoelectronics, Traceless Affinity and Other Technologies, 2019-2030" report has been added to ResearchAndMarkets.com's offering.

The Novel Cell Sorting and Separation Market: Focus on Acoustophoresis, Buoyancy, Dielectrophoresis, Magnetophoretics, Microfluidics, Optoelectronics, Traceless Affinity, and Other Technologies, 2019-2030' report features an extensive study of the current landscape and future outlook of the growing market for novel cell sorting and separation technologies (beyond conventional methods). The study presents detailed analyses of cell sorters, cell isolation kits, and affiliated consumables and reagents, that are based on the aforementioned technologies.

Advances in the fields of cell biology and regenerative medicine have led to the development of various cell-based therapies, which, developers claim, possess the potential to treat a variety of clinical conditions. In 2018, it was reported that there were more than 1,000 clinical trials of such therapies, being conducted across the globe by over 900 industry players.

Moreover, the total investment in the aforementioned clinical research efforts was estimated to be around USD 13 billion. Given the recent breakthroughs in clinical testing and the discovery of a variety of diagnostic biomarkers, the isolation of one or multiple cell types from a heterogenous population has not only become simpler but also an integral part of modern clinical R&D. The applications of cell separation technologies are vast, starting from basic research to biological therapy development and manufacturing.

However, conventional cell sorting techniques, including adherence-based sorting, membrane filtration-based sorting, and fluorescence- and magnetic-based sorting, are limited by exorbitant operational costs, time-consuming procedures, and the need for complex biochemical labels. As a result, the use of such techniques has, so far, been restricted in the more niche and emerging application areas.

Amongst other elements, the report features:

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/ss4o5a

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Novel Cell Sorting and Separation Markets, 2030 - P&T Community

AVROBIO Announces First Patient Dosed in Phase 1/2 Trial of Gene Therapy for Cystinosis – BioSpace

Oct. 8, 2019 11:00 UTC

CAMBRIDGE, Mass.--(BUSINESS WIRE)-- AVROBIO, Inc. (NASDAQ: AVRO) (the Company) today announced that the first patient has been dosed in the Companys AVR-RD-04 investigational gene therapy program for cystinosis, a devastating lysosomal storage disease, in an ongoing Phase 1/2 clinical trial sponsored by academic collaborators at the University of California San Diego. The gene therapy is derived from the patients own hematopoietic stem cells, which are genetically modified to produce functional cystinosin, a crucial protein that patients with cystinosis lack.

The trial will enroll up to six patients with cystinosis, a rare inherited disease caused by a defect in the gene that encodes for cystinosin. The cystinosin protein enables transport of the amino acid cystine out of lysosomes. When it is absent, cystine accumulates and crystalizes, causing progressive damage to the kidneys, liver, muscles, eyes and other organs and tissues. Cystinosis affects both children and adults; they face shortened life spans and often painful symptoms, including muscle wasting, difficulty breathing, blindness and kidney failure.

Cystinosis is a debilitating and progressive disease, and new treatment options are sorely needed. The current standard of care does not avert deterioration; at best, it can attenuate symptoms. Thats why gene therapy is particularly exciting: It has the potential to change the course of disease -- and the lives of patients -- by addressing the underlying cause of cystinosis, said Birgitte Volck, MD, PhD, President of Research and Development at AVROBIO. We believe we can engineer patients own stem cells so they sustainably produce the functional protein that is needed to prevent a toxic buildup of cystine and halt progression of the disease. We are so pleased that this investigational gene therapy is now in the clinic in collaboration with Dr. Stephanie Cherqui at UC San Diego.

The single-arm trial will enroll four adults and a potential follow-on cohort of two adults or adolescents at least 14 years of age who are currently being treated with cysteamine, the standard of care for cystinosis. If started at an early age and taken on a strict dosing schedule, cysteamine can delay kidney failure. However, the treatment regimen is highly burdensome, with side effects that can be severe and unpleasant, and many patients find it difficult to adhere to this treatment regimen. Even if compliance is high, cysteamine therapy cannot prevent kidney failure or avert other complications.

For people with cystinosis, there are no healthy days. They must take dozens of pills a day, around the clock, just to stay alive. It is a relentless disease and we urgently need new treatments, said Nancy J. Stack, President of the Cystinosis Research Foundation, which supported development of the gene therapy with more than $5.4 million in grants to Dr. Cherquis lab at UC San Diego. We believe that we are now an important step closer to the potential cure that our community has been working toward for many years.

The trials primary endpoints are safety and tolerability, assessed for up to two years after treatment, as well as efficacy, as assessed by cystine levels in white blood cells. Secondary endpoints to assess efficacy include changes in cystine levels in the blood, intestinal mucosa and skin and cystine crystal counts in the eye and skin. Efficacy will also be evaluated through clinical tests of kidney function, vision, muscle strength, pulmonary function and neurological and psychometric function, as well as through assessments of participants quality of life after treatment. The trial is funded by grants to UC San Diego from the California Institute for Regenerative Medicine (CIRM) as well as the Cystinosis Research Foundation.

This investigational gene therapy starts with the patients own stem cells, which are genetically modified so that their daughter cells can produce and deliver functional cystinosin to cells throughout the body. With this approach we aim to prevent the abnormal accumulation of cystine that causes so many devastating complications, said Stephanie Cherqui, PhD, an Associate Professor of Pediatrics at UC San Diego School of Medicine, and consultant to AVROBIO. We have been working toward this trial for years and we are grateful for all the support that brought us to this moment.

About AVR-RD-04

AVR-RD-04 is a lentiviral-based gene therapy designed to potentially halt the progression of cystinosis with a single dose of the patients own hematopoietic stem cells. The stem cells are genetically modified so they can produce functional cystinosin with the aim of substantially reducing levels of cystine in cells throughout the patients body. Before the infusion of the cells, patients undergo personalized conditioning with busulfan to enable the cells to permanently engraft. The Phase 1/2 clinical trial is being conducted under the name CTNS-RD-04 by AVROBIOs academic collaborators at the University of California, San Diego.

About Cystinosis

Cystinosis is a rare, inherited lysosomal storage disorder characterized by the accumulation of cystine in all the cells of the body, resulting in serious and potentially fatal damage to multiple organs and tissues and the shortening of patients life spans. The kidneys and eyes are especially vulnerable; more than 90% of untreated patients require a kidney transplant before age 20. An estimated 1 in 170,000 people are diagnosed with cystinosis.

About AVROBIO, Inc.

AVROBIO, Inc. is a leading, Phase 2 gene therapy company focused on the development of its investigational gene therapy, AVR-RD-01, in Fabry disease, as well as additional gene therapy programs in other lysosomal storage disorders including Gaucher disease, cystinosis and Pompe disease. The Companys plato platform includes a proprietary vector system, automated cell manufacturing solution and a personalized conditioning regimen deploying state-of-the-art precision dosing. AVROBIO is headquartered in Cambridge, MA and has offices in Toronto, ON. For additional information, visit http://www.avrobio.com.

Forward-Looking Statements

This press release contains forward-looking statements, including statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by words such as aims, anticipates, believes, could, estimates, expects, forecasts, goal, intends, may, plans, possible, potential, seeks, will and variations of these words or similar expressions that are intended to identify forward-looking statements. These forward-looking statements include, without limitation, statements regarding the therapeutic potential of our product candidates, the design, commencement, enrollment and timing of ongoing or planned clinical trials, including the ongoing Phase 1/2 trial of the Companys AVR-RD-04 investigational gene therapy, the anticipated benefits of our gene therapy platform, the expected safety profile of our product candidates, timing and likelihood of success of our current or future product candidates, and the market opportunity for our product candidates. Any such statements in this press release that are not statements of historical fact may be deemed to be forward-looking statements. Results in preclinical or early stage clinical trials may not be indicative of results from later stage or larger scale clinical trials and do not ensure regulatory approval. You should not place undue reliance on these statements, or the scientific data presented.

Any forward-looking statements in this press release are based on AVROBIOs current expectations, estimates and projections about our industry as well as managements current beliefs and expectations of future events only as of today and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to, the risk that any one or more of AVROBIOs product candidates will not be successfully developed or commercialized, the risk of cessation or delay of any ongoing or planned clinical trials of AVROBIO or our collaborators, the risk that AVROBIO may not realize the intended benefits of our gene therapy platform, including the features of our plato platform, the risk that our product candidates or procedures in connection with the administration thereof will not have the safety or efficacy profile that we anticipate, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical or clinical trials, will not be replicated or will not continue in ongoing or future studies or trials involving AVROBIOs product candidates, the risk that we will be unable to obtain and maintain regulatory approval for our product candidates, the risk that the size and growth potential of the market for our product candidates will not materialize as expected, risks associated with our dependence on third-party suppliers and manufacturers, risks regarding the accuracy of our estimates of expenses and future revenue, risks relating to our capital requirements and needs for additional financing, and risks relating to our ability to obtain and maintain intellectual property protection for our product candidates. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause AVROBIOs actual results to differ materially and adversely from those contained in the forward-looking statements, see the section entitled Risk Factors in AVROBIOs most recent Quarterly Report on Form 10-Q, as well as discussions of potential risks, uncertainties and other important factors in AVROBIOs subsequent filings with the Securities and Exchange Commission. AVROBIO explicitly disclaims any obligation to update any forward-looking statements except to the extent required by law.

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AVROBIO Announces First Patient Dosed in Phase 1/2 Trial of Gene Therapy for Cystinosis - BioSpace