BrainStorm Cell Therapeutics to make scientific presentations at the 30th International Symposium on ALS/MND – BioSpace

BrainStorms Chief Operating and Chief Medical Officer, Ralph Kern MD MHSc, will make a podium presentation at Clinical Trials Session

NEW YORK, Nov. 26, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, announced today that the Company is proud to be a gold sponsor of the 30th International Symposium on ALS/MND.

The symposium will take place December 4 6, 2019, at the Perth Convention and Exhibition Centre in Perth, Australia. The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields.

Ralph Kern MD MHSc, BrainStorms Chief Operating and Chief Medical Officer, will deliver a podium presentation: Modulation of innate immunity by MSC-NTF (NurOwn) cells correlates with ALS clinical outcomes, on December 4, from 11:50 12:10 pm AWST during the opening day Clinical Trials Session. In addition to the podium presentation, the Company will also present Poster 153: MSC-NTF Differentiation Increases the Neurotrophic Effects of MSC Cells: Live Imaging Analysis, that directly demonstrates the neuroprotective effects of NurOwn in a neuronal cell culture model.

Our fully-enrolled phase 3 clinical trial is one of the most advanced clinical programs in ALS, stated Chaim Lebovits, President and CEO of BrainStorm. He added, The International Symposium on ALS/MND is an important venue to update the community on our clinical and scientific efforts towards the advancement of therapies that may address the unmet needs of those living with ALS. BrainStorm Cell Therapeutics is proud to serve as a sponsor of this important annual symposium which underscores our commitment to the international community of ALS and MND patients, their families and their caregivers.

Ralph Kern, MD, stated, It is a privilege to present our innovative biomarker and preclinical research at the International Symposium on ALS/MND. He added, Every year, symposium participants gather together and discuss the opportunities and the challenges that we will face during the upcoming year. Research and medical breakthroughs for the ALS and MND community continue to make significant progress and we look forward to sharing our insights and engaging with colleagues from around the globe. The International Symposium on ALS/MND reminds us how far we have come in investigational therapies and how much more progress is still needed to bring patients a better and more promising future.

About NurOwn

NurOwn (autologous MSC-NTF) cells represent a promising investigational therapeutic approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. BrainStorm has fully enrolled a Phase 3 pivotal trial of autologous MSC-NTF cells for the treatment of amyotrophic lateral sclerosis (ALS). BrainStorm also received U.S. FDA acceptance to initiate a Phase 2 open-label multicenter trial in progressive MS and enrollment began in March 2019.

About BrainStorm Cell Therapeutics Inc.

BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn technology platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled a Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. For more information, visit BrainStorm's website at http://www.brainstorm-cell.com.

The International Symposium on ALS/MND is a unique annual event that brings together leading international researchers and health and social care professionals to present and debate key innovations in their respective fields. The Symposium is planned as two parallel meetings, one on biomedical research and the other on advances in the care and management of people affected by ALS/MND. Joint sessions consider issues of mutual concern, challenging current views and practices.

Safe-Harbor Statements

Statements in this announcement other than historical data and information constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may," "should," "would," "could," "will," "expect," "likely," "believe," "plan," "estimate," "predict," "potential," and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, risks associated with BrainStorm's limited operating history, history of losses; minimal working capital, dependence on its license to Ramot's technology; ability to adequately protect the technology; dependence on key executives and on its scientific consultants; ability to obtain required regulatory approvals; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

BRAINSTORM CONTACTS:

Investors:Uri Yablonka, Chief Business OfficerBrainStorm Cell Therapeutics IncPhone: : +1-201-488-0460Email: uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839Email: sean.leous@icrinc.com

Link:
BrainStorm Cell Therapeutics to make scientific presentations at the 30th International Symposium on ALS/MND - BioSpace

Inside the Beltway: Abortion, immigration among forbidden topics at Thanksgiving table – Washington Times

The warnings are already up in the popular press: Conversations during the Thanksgiving feast can be hazardous if they veer into political territory. But political talk can take place in theory: A data research company has now determined what topics are safe to talk about on the holiday.

With some qualitative analysis and a little common sense, weve created a cheat sheet that will help you blaze a path through Thanksgiving dinner that steers clear of treacherous political pitfalls and dangerous inter-uncle conflicts, reports Ranker.com, a Los Angeles-based media company which uses crowdsourcing to rank public opinion on multiple topics, typically at the rate of 15 million votes a month.

They have determined what political topics are the least and the most likely to set off a Thanksgiving dinner squabble. Their judgment is based on 300,000 votes from 40,000 respondents.

The topics to avoid this year: Abortion, immigration, terrorism and gender equality. The topics which are safe for dinnertime discussion: Ineffective government, health care reform and education.

If there are millennials present, they will be triggered, the organization says, by talk of abortion, police brutality and pollution. Generation X members will be set off by such topics as homelessness, affordable housing and campaign finance reform. Baby boomers will go to battle over terrorism, immigration and the moral decline of the nation.

The organization also has warnings for dinner hosts in certain states. If they live in Florida, their guests will be particularly sensitive about discussions of vaccines. In Indiana, its gender equality while Georgia diners are prone to fight over police brutality. Beware of talking about gun control at dinner tables in both California and Missouri; Texans get feisty over moral decline. New Yorkers get upset over transgender issues.

We examined each issue on a case-by-case basis to find the topics that are most likely to cause disagreement, as well as the ones on which people tend to either agree or not care about, Ranker.com explains.

A VERY SPECIALIZED MEAL

While most of us are enjoying turkey and pumpkin pie on Thanksgiving, the staff at one laboratory at Cedars-Sinai Medical Center in Los Angeles will be busy serving a meal to stem cells.

Stem cells do not observe national holidays, says Loren Ornelas-Menendez, manager of the very specialized lab that converts samples of adult skin and blood cells into stem cells which the human body uses to make our cells in the first place.

These special cells help medical scientists learn how diseases develop and how they might be cured. The lab is tending millions of them. Oh, but they have needs.

Stem cells are living creatures that must be hand-fed a special formula each day, monitored for defects and maintained at just the right temperature. And that means the cell lab is staffed every day, 52 weeks a year, the lab notes in a public advisory.

Many people have dogs. We have stem cells, says Ms. Ornelas-Menendez.

Derived from hundreds of healthy donors and patients, the resident induced pluripotent stem cells or iSPCs are keys to potential treatments for diabetes, breast cancer, Alzheimers disease, blindness, Parkinsons disease and Crohns disease, among other conditions. Ten lab technicians monitor the cells through microscopes each day and cull out any cells which have gone awry for one reason or another.

But what do they eat even on Thanksgiving?

While the cells get sorted, a special feeding formula is defrosting in a dozen bottles spread around a lab bench. The formula includes sodium, glucose, vitamins and proteins. Using pipettes, employees squeeze the liquid into food wells inside little compartments that contain the iPSCs. Afterward, they return the cells to their incubators, the lab advises.

Lab director Dhruv Sareen suggests that people consider offering a toast to the stem cells on Thanksgiving.

One day the cells they tend could lead to treatments for diseases that have plagued humankind for centuries, he says. And thats something to be truly thankful for.

THE GIPPERS FAVORITE

Back by popular demand, Inside the Beltway again shares this little known but historic recipe for President Reagans Favorite Macaroni and Cheese enjoyed by Ronald Reagan and his family on Thanksgiving and other holidays. What follows is a step-by-step shared by Mrs. Ronald Reagan, Washington, D.C., Wife of the President in a spiral-bound community cookbook published by the American Cancer Societys Northern Virginia division in 1983. The recipe serves six and is baked at 350 degrees F for 45 minutes.

The directions are from the cookbook reflecting the style, perhaps, of another era:

1/2 pound macaroni, 1 teaspoon butter, 1 egg, beaten; 1 teaspoon salt, 1 teaspoon dry mustard, 3 cups grated cheese, sharp; 1 cup milk.

Boil macaroni in water until tender and drain thoroughly. Stir in butter and egg. Mix mustard and salt with 1 tablespoon hot water and add to milk. Add cheese leaving enough to sprinkle on top. Pour into buttered casserole, add milk, sprinkle with cheese. Bake until custard is set and top is crusty.

Curious about what transpired at a Reagan Thanksgiving? A 1985 Los Angeles Times account noted this:

President and Mrs. Reagan gathered with their family for a quiet Thanksgiving dinner at their fogbound ranch in the Santa Ynez mountains, where the main topic of conversation was the weather. The Reagans did not seem to mind the enforced seclusion as they sat down to a traditional turkey dinner, prepared by Ann Allman, the Reagan familys longtime cook in California. It was an all-American menu that included cornbread dressing, cranberries, string beans, mashed potatoes, salad, pumpkin pie and monkey bread, a family favorite.

POLL DU JOUR

46% of Americans say long standing family tensions are the cause of family fights during holidays.

37% say general politics is the cause; 33% cite the 2020 presidential race.

24% say someones future plans cause the fights; 24% say money.

22% say the behavior of guests; 21% say drinking and alcohol.

18% say holiday cooking is the cause.

Source: A YouGov poll of 1,310 U.S. Adults conducted Sept. 25-26 and released Tuesday.

Have a happy Thanksgiving and thank you for reading Inside the Beltway.

Read the original post:
Inside the Beltway: Abortion, immigration among forbidden topics at Thanksgiving table - Washington Times

Canine Stem Cell Therapy Market Value Projected to Expand by 2026 – The Hilltop Monitor

The current Stem Cells Cryopreservation Equipments market research report has demonstrated all the vital market growth factors and economic fluctuations mentioned owing to the vast attention gained in recent years.

The Stem Cell Cartilage Regeneration market report provides a meticulous picture of the sector by summary of data, production, and method of study originated from various sources.

The high numbers of specialized veterinarians and veterinary clinics in these regions also contribute to the promising growth opportunities held by the canine stem cell therapy market in these regions. The market is growing with technological innovation, competition and M&A activities in the industry are offering specific application products for varied end-users.

The report is a thorough analysis of the Autologous Stem Cell and Non-Stem Cell Based Therapies Market.

Stem cell assay refers to the procedure of measuring the potency of antineoplastic drugs, on the basis of their capability of retarding the growth of human tumor cells. In-depth analysis of numerous components has been studied in the report including supply and demand Figures, cost, price, revenue, gross margins, current geographical zones, technology, demand-supply and consumption. Comprising an in-depth analysis of the various factors boosting and inhibiting the growth of the market, this report is a key to making profitable decisions by investing in the correct segment and sub-segment, which is anticipated to make the most progress in the future.

The report analyzes the prevalent opportunities for the market's growth and those that companies should capitalize in the near future to strengthen their position in the market.

Common sense to rest Man United seniors for Astana trip: SolskjaerA statement released by the club In February said "everybody looks forward to seeing him wear the red shirt again". Their last outing against Sheffield United ended in 3-3 draw and they surely don't repeat that feat this time.

Global Market portal aims to provide reports like these in order to draw the attention of numerous clients wanting to extrapolate some of the vital details of the Stem Cells Cryopreservation Equipments market on a global scale. The report provides an extensive evaluation of each market segment considering its demand, production, sales revenue, and potential growth. The solid research on the Animal Stem Cell Therapy Market is prepared with the aim to meet the requirements of the customer in terms of the availability of data, analytics, statistics, and an accurate forecast market.The market report also presents the landscape and a corresponding detailed analysis of the major players operating in the market. The ones holding the most lucrative prospects are analyzed, and the factors restraining its trajectory across key segments are also discussed at length. USA, Europe, Japan, China, India, Southeast Asia, South America, South Africa, Others. By kit, the market can be bifurcated into human embryonic stem cell kits and adult stem cell kits.

- Which Trending factors influencing the market shares of the top regions across the globe?

- Who are the key market players and what are their strategies in the global Animal Stem Cell Therapy market? Moreover, studies showing the excellent level of safety granted by allogeneic stem cell therapy are also rising in numbers, leading to increased confidence among pet owners as well as veterinarians regarding canine stem cell therapy.

- What industrial trends, drivers and challenges are manipulating its growth?

- What are the key outcomes of the five forces analysis of the global Animal Stem Cell Therapy market? The clients and other readers can have all the global Stem Cells Cryopreservation Equipments market highlights provided in this very report. It also conducts SWOT analysis to study the strengths and weaknesses of the companies profiled and identify threats and opportunities that these enterprises are forecast to witness over the course of the report's forecast period. The increasing incidence of chronic ailments and the subsequently expanding patient population are the chief drivers of the stem cell assay market in North America. The report has all the vital information regarding supply and demand, market development enhancers, market share, sales distributors, and more advocated in a very formal pattern.

Read the original:
Canine Stem Cell Therapy Market Value Projected to Expand by 2026 - The Hilltop Monitor

Global Stem Cells Market 2019 by Manufacturers, Regions, Type and Application, Forecast to 2025 – Industry News Info

The Global Stem Cells Market report study includes an elaborative summary of the Stem Cells market that provides in-depth knowledge of various different segmentations. Stem Cells Market Research Report presents a detailed analysis based on the thorough research of the overall market, particularly on questions that border on the market size, growth scenario, potential opportunities, operation landscape, trend analysis, and competitive analysis of Stem Cells Market. The information includes the company profile, annual turnover, the types of products and services they provide, income generation, which provide direction to businesses to take important steps. Stem Cells delivers pin point analysis of varying competition dynamics and keeps ahead of Stem Cells competitors such as CCBC, Vcanbio, Boyalife, Beikebiotech.

View Sample Report @https://www.intenseresearch.com/market-analysis/global-stem-cells-market-2019-by-manufacturers-regions.html#request-sample

The main objective of the Stem Cells report is to guide the user to understand the Stem Cells market in terms of its definition, classification, Stem Cells market potential, latest trends, and the challenges that the Stem Cells market is facing. In-depth researches and Stem Cells studies were done while preparing the Stem Cells report. The Stem Cells readers will find this report very beneficial in understanding the Stem Cells market in detailed. The aspects and information are represented in the Stem Cells report using figures, bar-graphs, pie diagrams, and other visual representations. This intensifies the Stem Cells pictorial representation and also helps in getting the Stem Cells industry facts much better.

.This research report consists of the worlds crucial region market share, size (volume), trends including the product profit, price, Value, production, capacity, capability utilization, supply, and demand and industry growth rate.

Geographically this report covers all the major manufacturers from India, China, the USA, the UK, and Japan. The present, past and forecast overview of the Stem Cells market is represented in this report.

The Study is segmented by following Product Type, Umbilical Cord Blood Stem Cell, Embryonic Stem Cell, Adult Stem Cell, Other

Major applications/end-users industry are as follows Diseases Therapy, Healthcare

Stem Cells Market Report Highlights:

1)The report provides a detailed analysis of current and future market trends to identify the investment opportunities2) In-depth company profiles of key players and upcoming prominent players3) Global Stem Cells Market Trends (Drivers, Constraints, Opportunities, Threats, Challenges, Investment Opportunities, and recommendations)4) Strategic recommendations in key business segments based on the market estimations5) To get the research methodologies those are being collected by Stem Cells driving individual organizations.

Research Parameter/ Research Methodology

Primary Research:

The primary sources involve the industry experts from the Global Stem Cells industry including the management organizations, processing organizations, analytics service providers of the industrys value chain. All primary sources were interviewed to gather and authenticate qualitative & quantitative information and determine future prospects.

In the extensive primary research process undertaken for this study, the primary sources industry experts such as CEOs, vice presidents, marketing director, technology & innovation directors, founders and related key executives from various key companies and organizations in the Global Stem Cells in the industry have been interviewed to obtain and verify both qualitative and quantitative aspects of this research study.

Secondary Research:

In Secondary research crucial information about the industry value chain, the total pool of key players, and application areas. It also assisted in market segmentation according to industry trends to the bottom-most level, geographical markets and key developments from both market and technology oriented perspectives.

Inquiry for Buying Report: https://www.intenseresearch.com/market-analysis/global-stem-cells-market-2019-by-manufacturers-regions.html#inquiry-for-buying

Thanks for reading this article, you can also get individual chapter wise section or region wise report versions like North America, Europe or Asia. Also, If you have any special requirements, please let us know and we will offer you the report as you want.

Sorry! The Author has not filled his profile.

More here:
Global Stem Cells Market 2019 by Manufacturers, Regions, Type and Application, Forecast to 2025 - Industry News Info

Stem Cell Banking Market Size, Share, Growth, Future Prospects, Competitive Analysis and Forecast To 2026 – Med News Ledger

Stem Cell Banking Market Global Drivers, Restraints, Opportunities, Trends, and Forecasts: 20192026Overview: Stem cells are undifferentiated biological cells that can distinguish into specialized cells, tissue, or an organ. The process of storing these stem cells for the treatment of life-threatening diseases in the future is called stem cell banking. Nearly 500 stem cell banks are functioning globally, and every bank is now striving harder to increase their market share. Stem cell banking has applications in cerebral palsy, thalassemia, leukemia, diabetes, autism, and others. Cerebral palsy holds the major share of nearly quarter of the market share among the various applications. Private stem cell banks are implementing innumerable publicizing strategies to upsurge their product visibility among people.

Key Players: Esperite, Caladrius Biosciences, Via Cord, CBR Systems, Smart Cells, Life Cell, China Cord Blood, Cryo-Cell, StemCyte, Cordvida, ViaCord, Cryoviva, and other predominate & niche players.

Get Sample copy of this Report: http://contrivedatuminsights.com/Home/RequestaSample/2433

The market is driven by factors such as easy method of extraction of stem cells from the samples, increasing birth rate, increased awareness of stem cell therapeutics, and increasing potentials of stem cell treatment. Alongside, the collaboration among the cord blood banks, increasing investments and fundings, and automation of procedures for the banking of stem cells are providing opportunities for the growth of the stem cell banking market. However, intense competition, high operating costs, changes in legal regulations, and high entry barriers are the major factors hampering the market growth.

Market Analysis: The Stem Cell Banking Market is estimated to witness a CAGR of 16.4% during the forecast period 20192026. The market is analyzed based on three segments, namely product types, end-users, and regions.Regional Analysis: The regions covered in the report are North America, Europe, Asia Pacific, and Rest of the World (RoW). North America is set to be the leading region for the stem cell banking market growth followed by Europe. Asia Pacific and RoW are set to be the emerging regions. India, China, and Japan are set to be the most attractive destinations due to the large untapped market.

Product Types Analysis: The stem cell banking market by products is segmented into umbilical cord blood & cord tissue, and adult stem cell banking. The umbilical cord blood & tissue occupies the major share in the market and is also expected to grow at a fast CAGR during the forecast period. The dental stem cell banking and menstrual blood stem cell banking are the latest diversifications in the stem cell banking market. The market is also witnessing various mergers, acquisitions, and collaborations among the top players, which is defining the future of the global stem cell banking market.

Get Special Pricing: http://contrivedatuminsights.com/Home/GetSpecialPricing/2433

Competitive Analysis: These days, stem cell banks exist in most of the developed and developing nations. Around 450 companies are publicizing cord blood banking services internationally, which signifies intense competition in the market. Globally, China Cord Blood Corporation (CCBC) is expected to be the fastest growing stem cell bank worldwide and in the US, it is Amricord. In 2014, the company Amricord achieved 2,200% growth rate from 2011-2013. Future Health Biobank, American Cryostem, Adicyte, Adisave, Celltex, and Vault Stem currently hold around two-thirds of the Mesenchymal Stem Cells (MSC) storage market. However, at present, these companies are still trivial but are swiftly intensifying. Cryostem witnessed its revenue to nearly double in 2016 from $400,000 to almost $800,000.

Benefits: The report provides complete details about the usage and adoption rate of stem cell banking in various regions. With that, key stakeholders can know about the major trends, drivers, investments, vertical players initiatives, government initiatives toward the stem cell therapy and banking adoption in the upcoming years along with the details of commercial devices available in the market. Moreover, the report provides details about the major challenges that are going to impact on the market growth. Additionally, the report gives complete details about the key business opportunities to key stakeholders to expand their business and capture the revenue in the specific verticals to analyse before investing or expanding the business in this market.

For More Information: http://contrivedatuminsights.com/Home/InquiryBeforeBuying/2433

Table of Contents

Global Stem Cell Banking Market Research Report

Chapter 1 Global Stem Cell Banking Market Overview

Chapter 2 Global Economic Impact on Industry

Chapter 3 Global Market Competition by Manufacturers

Chapter 4 Global Production, Revenue (Value) by Region

Chapter 5 Global Supply (Production), Consumption, Export

Chapter 6 Production, Revenue (Value), Price Trend by Type

Chapter 7 Market Analysis by Application

Chapter 8 Manufacturing Cost Analysis

Chapter 9 Industrial Chain, Sourcing Strategy and Downstream Buyers

Chapter 10 Marketing Strategy Analysis, Distributors/Traders

Chapter 11 Market Effect Factors Analysis

Chapter 12 Market Forecast

Any special requirements about this report, please let us know and we can provide custom report.

View post:
Stem Cell Banking Market Size, Share, Growth, Future Prospects, Competitive Analysis and Forecast To 2026 - Med News Ledger

Lulu and Nana are the result of a defiant experiment in human gene modification – The Irish Times

Remember these two names, Lulu and Nana. They are twin girls born in China in October 2018, and we already know they are going to be famous.

They are the worlds first genetically-edited humans, and their progress through life will be monitored intensively by medical researchers over the coming years.

Dont doubt their future celebrity. Remember the headlines when Dolly the sheep arrived in 1996, the worlds first mammal cloned directly from an adult cell. Or how about Louise Brown, who made headlines in 1978 as the first baby to be conceived using invitro fertilisation (IVF) techniques.

Although the methods used to bring about these three births are light years apart, all three involve the delivery of an offspring using unorthodox methods.

All three were also controversial in their day. There was no end of condemnation and criticism about playing god and defying the laws of nature for the first two, but now IVF is commonplace for lots of medical reasons and is accepted as a standard medical practice. And news of another species successfully cloned in the lab would not make headlines today.

However, the genetic modification of the twins is a different matter, a genetic change introduced before birth to deliver a permanent alteration of their original genome that will be passed down from generation to generation.

Prof He Jiankui of the Southern University of Science and Technology carried out the genetic change needed to permanently modify the twins genome. He introduced a mutation that gave Lulu and Nana resistance to the Human Immunodeficiency Virus, the virus that causes Aids, and revealed his successful genetic modification one year ago this month.

The backlash was immediate and severe. There was international condemnation that China allowed experimentation on humans. Last January his university sacked him.

The scientific community also criticised the work as having crossed an important red line for genetics research, the reality that we still know too little about how even the smallest genetic change might have unexpected impacts downstream in other parts of the genome.

It was bad enough that a modification had taken place, but the modification was in the germline, the cells that bring about the next generation and the next and the next.

Perhaps the lure of notoriety proved too strong or the desire to be the first, but He Jiankui crossed that line, helped along the way through use of a gene-editing method known as CRISPR-Cas9.

This method emerged over several years, and has become the gene-cutting tool of choice because it allows very tight control over how a gene can be modified, added to or deleted.

When it came into widespread use the scientific community recognised immediately that controls on its application were necessary to prevent its use in human gene modification. This should have prevented He Jiankui from attempting such a daring and defiant experiment but it didnt, and Lulu and Nana were the result.

They were born healthy but now scientists will want to know whether there are unexpected or unwanted effects or other issues that arise. Already researchers have raised doubts about the gene modification that confers resistance to HIV, which means some level of susceptibility to the virus may remain.

The modified gene, known as CCR5, also has other roles in the body, and its modified actions may affect the lifespan of the twins. One large study involving 410,000 subjects showed that people with a similar mutated version of CCR5 were 20 per cent more likely to die before reaching the age of 78.

Despite these misgivings there is no doubt that CRISPR-Cas9 will in the future be used to modify the human genome in the battle against difficult diseases such as cancer and in genetic disorders caused by specific gene mutations. The tool is far too important to avoid its eventual use, or the use of some other similar but as yet unidentified gene-cutting method.

Similarly there is little doubt that online charlatans will offer promised but undeliverable cures using CRISPR-Cas9, as was the case with earlier technologies such as stem cells.

Lulu and Nana, meanwhile, will get on with living their lives, doing what babies do. Their names are pseudonyms in an attempt to conceal them from public view, but it is likely that we may learn their real names in the future.

Lulu and Nanas story came to mind in light of research published last week (November 21st) in the journal Cell about how attempting to create designer babies using other advanced technology could still remain a costly waste of time.

It involves choosing an embryo based on its potential to be tall or smarter than average, but accomplishing this via something like CRISPR-Cas9 is too far beyond our current abilities. Instead the international team of scientists set up a model to simulate one method called pre-implantation genetic testing.

This involves screening the genome for genes that have an association to a given trait, in this studys case intelligence and height, and giving them a score. The team found, however, that at best the top-scoring embryo might be expected to be 2.5cm taller than average and at best 2.5 IQ points above average.

Lurking behind all of this remains the most challenging of issues, the ethics surrounding the use of this technology.

As usual the ethics questions that should have been asked first are the ones obscured by the advance of these promising discoveries.

Continued here:
Lulu and Nana are the result of a defiant experiment in human gene modification - The Irish Times

Types of Adult Stem Cells – Stem Cell Institute

Stem cells reside in adult bone marrow and fat, as well as other tissues and organs of the body including the umbilical cord. These cells have a natural ability to repair damaged tissue, however in people with degenerative diseases they are not released quickly enough to fully repair damaged tissue. In the case of fat stem cells they may not be released at all. The process of actively extracting, concentrating and administering these stem cells has been shown in clinical trials to have beneficial effects in degenerative conditions. Few patients have access to clinical trials. We offer patients and their doctors access to these therapies now. Stem cell treatments are not covered by insurance.

Adult stem cells can be extracted from most tissues in the body, including the bone marrow, fat, and peripheral blood. They can also be isolated from human umbilical cords and placental tissue. Once the cells have been harvested, they are sent to the lab where they are purified and assessed for quality before being reintroduced back in the patient. Common types of adult stem cells are mesenchymal and hematopoietic stem cells.

Umbilical cord mesenchymal stem cells reside in the *umbilical cords of newborn babies. HUCT-MSC stem cells, like all post-natal cells, are adult stem cells.

The Stem Cell Institute utilizes cord-derived mesenchymal stem cells that are separated from the umbilical cord tissue. For certain indications, these cells are expanded into greater numbers at Medistem laboratory in Panama under very strict, internationally recognized guidelines.

Among many other things, mesenchymal stem cells from the umbilical cord tissue are known to help reduce inflammation, modulate the immune system and secrete factors that may help various tissues throughout the body to regenerate.

The bodys immune system is unable to recognize HUCT mesenchymal stem cells as foreign and therefore they are not rejected. Weve treated hundreds of patients with umbilical cord stem cells and there has never been a single instance rejection (graft vs. host disease). HUCT MSCs also proliferate/differentiate more efficiently than older cells, such as those found in the bone marrow and therefore, they are considered to be more potent.

Through retrospective analysis of our cases, weve identified proteins and genes that allow us to screen several hundred umbilical cord donations to find the ones that we know are most effective. We only use these cells and we call them golden cells.

We go through a very high throughput screening process to find cells that we know have the best anti-inflammatory activity, the best immune modulating capacity, and the best ability to stimulate regeneration.

Human umbilical cord tissue-derived mesenchymal stem cells (MSCs) that were isolated and grown in our laboratory in Panama to create master cell banks are currently being used in the United States.

These cells serve as the starting material for cellular products used in MSC clinical trials for two Duchennes muscular dystrophy patients under US FDAs designation of Investigational New Drug (IND) for single patient compassionate use. (IND 16026 DMD Single Patient)

The bone marrow stem cell is the most studied of the stem cells, since it was first discovered to in the 1960s. Originally used in bone marrow transplant for leukemias and hematopoietic diseases, numerous studies have now expanded experimental use of these cells for conditions such as peripheral vascular disease, diabetes, heart failure, and other degenerative disorders.

At Stem Cell Institute, we use purified autologous (patients own) mesenchymal stem cells from bone marrow in our spinal cord injury protocol along with umbilical cord tissue mesenchymal stem cells.

Fat stem cells are essentially sequestered and are not available to the rest of the body for repair or immune modulation. Fat derived stem cells have been used for successful treatment of companion animals and horses with bone and joint injuries for the last 10 years with positive results.

Experimental studies suggest fat derived stem cells not only can develop into new tissues but also suppress pathological immune responses as seen in autoimmune diseases. In addition to orthopedic conditions, Stem Cell Institute pioneered treating patients with osteoarthritis, rheumatoid arthritis, multiple sclerosis, and other autoimmune diseases using fat derived stem cells. However, we no longer use a patients own stem cells from fat because weve found that mesenchymal stem cells from umbilical cord tissue are superior.

Dr. Riordan published the first scientific article on treating humans (3 multiple sclerosis patients) with adipose-derived stem cells. We have treated many patients with adipose-derived mesenchymal stem cells in Panama but we no longer do so because we have found that umbilical cord-derived MSCs modulate the immune system and control inflammation better. HUCT MSCs also proliferate much more efficiently.

Articles Authored by our Doctors and Scientists about Fat Derived Stem Cells:

*All donated cords are the by-products of normal, healthy births. Each cord is carefully screened for sterility and infectious diseases under International Blood Bank standards.

See the original post here:
Types of Adult Stem Cells - Stem Cell Institute

Adult Leukemia: What You Need to Know – Dana-Farber Cancer Institute

Medically reviewed by Richard M. Stone, MD

More than 60,000 new cases ofadult leukemiaare diagnosed in the U.S. each year. Although it is one of the more common childhood cancers,leukemia occurs more often in older adults.

Leukemia is a cancer of the bodys blood-forming tissues that results in large numbers of abnormal or immature white blood cells. The main types of leukemia are:

AML causes the bone marrow to produce immature white blood cells (called myeloblasts). As a result, patients may have a very high or lowwhite blood cellcount, and lowred blood cellsandplatelets.

CLL is the second most common type of leukemia in adults. It is a type of cancer in which the bone marrow makes too many maturelymphocytes(a type of white blood cell).

ALL is a type of leukemia in which the bone marrow makes too many immaturelymphocytes. Similar to AML, the white blood cells can be high or low and oftentimes the platelets and red blood cells are low. This form of leukemia is more common in children than adults.

CML is usually a slowly progressing disease in which too many mature white blood cells are made in the bone marrow.

People with leukemia may experience:

Because these symptoms can be caused by a variety of other conditions, its important to check with your doctor if they arise.

While studies have shown men to be more atrisk than women, some other risk factors include:

While test procedures vary based on the type of leukemia, the two most common procedures are thecomplete blood count(CBC) test and the bone marrow aspiration biopsy.

CBC is a procedure used to check the redblood cell and platelet counts as well as the number and type of white bloodcells (the red cells carry oxygen, the white cells fight and prevent infection,and platelets control bleeding). A bone marrow aspiration biopsy involvesremoving a sample of bone marrow, including a small piece of bone by insertinga needle into the hipbone. The sample is then examined for abnormal cells.

Treatment for leukemia varies depending on the type and specific diagnosis.

The treatment for acute leukemias may be lengthy up to two years in ALL and is usually done in phases. The first phase, known as remission induction therapy, involves administering several chemotherapy drugs over a several-week period. The goal is to destroy as many cancer cells as possible to achieve a remission (in which cancer cells are undetectable, but small amounts are still present).

The second phase, known aspost-remission or consolidation therapy, seeks to kill leukemia cells thatremain after remission induction therapy. This phase may involve chemotherapyand/or a stem cell transplant.

Additional treatments may also be necessary. ALL patients, for example, may receive special treatment to prevent the disease from recurring in the spinal cord or brain.

The treatment for CML has been revolutionized by the advent of the oral medication imatinib and the second- and third-generation drugs known as tyrosine kinase inhibitors (TKIs). These are oral medications that work to inhibit the function of theBCR-ABLprotein. Many patients take these medications for the rest of their lives. In rare instances, a patient may require a stem cell transplant.

Some patients with CLL are recommended formonitoring and observation. Others,usually those with symptoms or low red cell or platelet counts, requiretreatment. Such treatment may involve intravenous chemotherapy, but often withoral therapy with pills that inhibit the function of a key protein, Brutonstyrosine kinase.

Treatments for leukemia can include:

Drugs that harness the immune system in fighting leukemia have shown considerable promise. Some monoclonal antibodies synthetic versions of immune system proteins are already in use to treat certain forms of leukemia and others are being studies in clinical trials.

Another form of immunotherapy, immune checkpoint inhibitors, which release a pent-up immune system attack on tumor cells, is being tested in several forms of leukemia. Cancer vaccines, which boost the immune systems ability to fight cancer, are being studied for use in leukemia.

CAR T-cell therapy, which uses modified immune system T cells to better target and kill tumor cells, has achieved impressive results in trials involving children and adults up to age 25 with relapsed ALL.

Research into new treatments for adult leukemia is moving along several tracks in addition to immunotherapy.

By tracking the specific abnormal genes within leukemia cells, physicians are increasingly able to tailor treatment to the unique characteristics of the disease in each patient. Targeted drugs such as imatinib and dasatinib, for example, are now used in treating patients with ALL whose leukemia cells have an abnormality known as the Philadelphia chromosome. Targeted agents including IDH or FLT3 inhibitors, which zero in on proteins made from mutated genes, have been approved to treat some patients with AML, while other such inhibitors are being tested in clinical trials.

New tests make it possible to detect ever smaller amounts of leukemia that remain after treatment. Investigators are exploring how these minute levels may influence a patients prognosis and how they might impact treatment.

Researchers are testing whether treatment periods for certain drugs can be safely reduced in some patients. For instance, studies are under way to determine if drugs such as imatinib, which are currently taken for life, can be safely stopped in some patients with CML. Researchers hope to test whether treating patients with CLL with the drug ibrutinib plus other medicine for a fixed amount of time is safe and effective.

Patients may consider treatment through a clinical trial.Dana-Farber currently has more than 30 clinical trials for adult leukemia. A national list of clinical trials is available atclinicaltrials.gov.

Go here to read the rest:
Adult Leukemia: What You Need to Know - Dana-Farber Cancer Institute

Hatching disease in a dish: The new frontier in drug testing – Maclean’s

Over lunch at the Canadian Centre for Alternatives to Animal Methods (CCAAM), Charu Chandrasekera nonchalantly mentions one of the projects her team is working on. We are just printing some human liver tissue right now, she says.

Chandrasekera launched the CCAAM at the University of Windsor in 2017, with help from the schools vice-president of research and innovation, Michael Siu, and dean of science, Chris Houser. The centre promotes non-animal methods in biomedical research, education, and regulatory (chemical safety) testing. In October 2019, the centre received a million-dollar gift from the Eric S. Margolis Family Foundation, which Chandrasekera says was instrumental in establishing the state-of-the art research laboratory, and in launching a number of important initiatives.

Chandrasekera says the move away from animal testing to human-based research models isnt radical but inevitable. After many years working in biomedical research with mouse models of heart disease and diabetes, It became very obvious that the work I was doing was not translatable [to humans], she says. Nothing was really reproducible; there were so many discrepancies and contradictions, even among the top-notch researchers.

Ninety-five per cent of drugs tested to be safe and effective in animal models fail in human clinical trials, says Chandrasekera. Alzheimers disease99.6 per cent drug failure rate, she says. It has been cured in mice. But we dont even understand the molecular mechanisms of this disease in humans, much less a cure.

RELATED:I am mine: This is what Alzheimers is like at 41

Empirical evidence from across a whole host of biomedical science disciplines shows us that animal models are failing both science and human health, echoes Elisabeth Ormandy, co-founder and executive director of Animals in Science Policy Institute, a registered Canadian charity working to promote better science without animals. Animal models can falsely show that a drug is effective, she says. They can also falsely show no effect, in which case a drug that would have been shown to be effective in humans never gets advanced to human clinical trials.

The result, she says, is billions of public tax dollars being wasted on research using ineffective animal models, and diversion of precious research funding away from other lines of scientific inquiry that might hold greater promise in terms of predicting drug safety, risk, and effectiveness.

Those other promising lines of scientific inquiry, say Ormandy and Chandrasekera, are human biology-based models. We can use human cells and tissues from cadavers, biopsies, and explanted organs [from surgeries], says Chandrasekera. And we can also engineer them. With adult stem cell technology, you can take a small biopsylike two-to-three millimetres from a persons skinto create any cell type in your body, she says. And if that person has a disease, such as Alzheimers, it will still be present in these cells. These cells can then be assembled to form tissue-like structures called organoids, or engineered through 3D-bioprinting to create more complex tissues, all of which can be combined to create what has become known as disease-in-a-dish. At present,Chandrasekera iscreating diabetes-in-a-dish.

Further, those cells and tissues can also be placed onto computer chips the size of thumb drives, where a large number of drugs can be tested to select whats most appropriate for youpersonalized medicine based on your cells, your tissues, your biologynot mouse biology, Chandrasekera explained in her April 2019 TedX Talk. The goal of the scientific community at large is to create a human-on-a-chip to emulate human biology better than animals, she says, which I think will happen over the next decade.

Currently there is no data on the success rates of human biology-based methods, because there are no drugs that have been approved without animal testing, since animal testing was mandated by regulatory guidelines several decades ago, says Chandrasekera.

However, a growing body of scientific data and internationally approved guidelines in chemical safety testing, indicate that alternative methods are equal or superior to animal models in predicting human biology, Chandrasekera says. Even computer simulations are out-predicting animal-derived data.

RELATED:Health care cannot modernize unless health policy changes first

Ifdisease-in-a-dish and toxicity-on-a-chip effortscontinue to advance at a fast pace with a sense of urgencybacked by global scientific, financial, legislative, and ethical mandates, she says, we will come to a point where we can test drugs without relying on animals.

And while Chandrasekera is busy both in the lab and on the global stage promoting her work, she is also focused on enlightening future scientists. Shes working the development of courses and degrees to train the next generation, she says, to think outside the cage.

Here is the original post:
Hatching disease in a dish: The new frontier in drug testing - Maclean's

Bedfordshire Police officer reunited with woman whose life he saved through stem cell donation – Bedford Today

PC Andrew Harris, who is based at the Police Headquarters in Kempston, signed up to the Anthony Nolan stem cell donor register in 2008 when he joined the Metropolitan Police, he has since transferred to Bedfordshire Police.

Beatrice, a mother of two, from Palm Springs, California, was diagnosed with Myelofibrosis at the age of 23. The condition is a rare type of blood cancer which leads to leukaemia.

She has been living with the condition for the majority of her adult life, managing the symptoms with medication and waiting for a donor match, as this is the only known treatment for this condition.

Her ethnic background Beatrice is half European and half Vietnamese - meant that the chance of finding a match was extremely unlikely, and after years of searching in 2008 she gave up on ever finding a donor to focus her time on taking care of her young children.

Over time her health began to deteriorate and she was in desperate need of a stem cell donation to save her life. So in 2015, when she was about to enter into palliative care, her health care provider ran one last check on the donor register and she received the news that there was a perfect genetic match on a donor list a police officer from the United Kingdom.

At the same time the news came through, Beatrices condition had deteriorated and she was very poorly, thankfully, her health improved enough to get through the transplant procedure.

The transplant was successful and after the two year waiting period, Beatrice and Andrew got in touch through Skype. They have stayed in touch over time and met in person in London a month ago.

PC Harris said: When I was contacted and informed about this match I didnt hesitate for a second. I joined the police because I always wanted to help people and this wasnt any different.

The preparation for the procedure was painless and was done by my doctor and a local nurse. I was given injections for a period of five days to release stem cells into my bloodstream.

After that my stem cells were collected through a special machine similar to a dialysis machine, which was filtering them out of my bloodstream. This was done under local anaesthesia and was completely painless.

If felt unreal when I finally got to meet Beatrice over Skype after the two year waiting period. There was a sense of pride from my side and it was an extremely emotional moment for both of us. Since then we have stayed in touch but we only got to meet in person last month.

It was amazing to meet her and her family. Beatrice is such a strong character and to think that only couple of years ago she and her children were preparing to enter palliative care.

"She is now committed to spreading awareness of how important it is to register as a donor. It is not only one life thats been saved; it is also her family and loved ones who get to keep their daughter, mum and friend.

Our force is working with the local charity and we invite them regularly to events, during which you can join the stem cell register. I would like to encourage everyone who is eligible to sign up; you can be someones last hope and save their life.

People aged between 16 and 30 who are in good general health can sign up to the Anthony Nolan register at http://www.anthonynolan.org.

The charity will send you a swab pack in the post, which you should return to the charity. Whenever a patient with blood cancer or a blood disorder needs a lifesaving stem cell transplant, Anthony Nolan searches the register, looking for someone who is a genetic match for that patient.

If you are found to be a match the charity will be in touch, and will ask you to donate if youre still healthy and happy to do so.

See more here:
Bedfordshire Police officer reunited with woman whose life he saved through stem cell donation - Bedford Today