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Category Archives: Stem Cells

2019: The year gene therapy came of age – Eyewitness News

Posted: December 27, 2019 at 3:42 pm

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

Picture: Supplied.

WASHINGTON, United States - In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering -- by editing her genome.

Victoria Gray's recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research -- gene therapy.

"I have hoped for a cure since I was about 11," the 34-year-old told AFP in an email.

"Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency."

Over several weeks, Gray's blood was drawn so doctors could get to the cause of her illness -- stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 -- pronounced "Crisper" -- a new tool informally known as molecular "scissors."

The genetically edited cells were transfused back into Gray's veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

"This is one patient. This is early results. We need to see how it works out in other patients," said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

"But these results are really exciting."

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta-thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

"It's all developing very quickly," said French geneticist Emmanuelle Charpentier, one of Crispr's inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

CURES

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practising the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not -- such as making normal red blood cells, in Victoria's case, or making tumour-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the US and a blood disease in the European Union.

They join several other gene therapies -- bringing the total to eight -- approved in recent years to treat certain cancers and inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

"Twenty-five, 30 years, that's the time it had to take," he told AFP from Paris.

"It took a generation for gene therapy to become a reality. Now, it's only going to go faster."

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a "breakthrough period."

"We have hit an inflection point," said Carrie Wolinetz, NIH's associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million -- meaning patients face gruelling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in the hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion -- and fighting a general infection.

"You cannot do this in a community hospital close to home," said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

**BIOTERRORISM **

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who don't necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal -- and his ex-communication from the scientific community -- when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

"That technology is not safe," said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr "scissors" often cut next to the targeted gene, causing unexpected mutations.

"It's very easy to do if you don't care about the consequences," Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species -- malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesn't believe in the more dystopian scenarios predicted for gene therapy, including American "biohackers" injecting themselves with Crispr technology bought online.

"Not everyone is a biologist or scientist," she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies' crops?

Charpentier thinks that technology generally tends to be used for the better.

"I'm a bacteriologist -- we've been talking about bioterrorism for years," she said. "Nothing has ever happened."

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AgeX Therapeutics Announces Drawdown of Second Tranche of Loan Facility from Juvenescence Ltd. – BioSpace

Posted: December 27, 2019 at 3:42 pm

Juvenescence is pleased to continue its commitment to AgeX through this additional drawdown under the loan facility, commented Gregory Bailey, MD, Chairman of AgeX and CEO of Juvenescence. Juvenescence remains committed to funding the future development plans of AgeX through further advancements under the loan facility or otherwise. Since Juvenescences initial investment in AgeX in June 2018, AgeX has been an important element in the Juvenescence mission and strategy. Juvenescence is also investing its time and personnel to support AgeXs business development initiatives which have impressive potential. We look forward to AgeX announcing its plans for 2020 as it pursues tissue regeneration in Reverse Bioengineering, while advancing the development of BAT and VASC 1, the coupling of HLA-G with PureStem-derived cells for transplant therapies, and exploring partnerships with third parties.

This round of funding will allow us to continue to execute on our strategic plan to provide therapies for certain chronic and degenerative diseases through cellular regeneration and replacement, commented AgeXs founder and CEO Michael D. West, PhD.

As announced in the companys news release on August 14, 2019, AgeX has obtained a $2 million credit facility from Juvenescence to finance AgeXs operations and advance its product development programs.

About AgeX Therapeutics

AgeX Therapeutics, Inc. (NYSE American: AGE) is focused on developing and commercializing innovative therapeutics for human aging. Its PureStem and UniverCyte manufacturing and immunotolerance technologies are designed to work together to generate highly-defined, universal, allogeneic, off-the-shelf pluripotent stem cell-derived young cells of any type for application in a variety of diseases with a high unmet medical need. AgeX has two preclinical cell therapy programs: AGEX-VASC1 (vascular progenitor cells) for tissue ischemia and AGEX-BAT1 (brown fat cells) for Type II diabetes. AgeXs revolutionary longevity platform induced Tissue Regeneration (iTR) aims to unlock cellular immortality and regenerative capacity to reverse age-related changes within tissues. AGEX-iTR1547 is an iTR-based formulation in preclinical development. HyStem is AgeXs delivery technology to stably engraft PureStem cell therapies in the body. AgeX is developing its core product pipeline for use in the clinic to extend human healthspan and is seeking opportunities to establish licensing and collaboration agreements around its broad IP estate and proprietary technology platforms.

For more information, please visit http://www.agexinc.com or connect with the company on Twitter, LinkedIn, Facebook, and YouTube.

Forward-Looking Statements

Certain statements contained in this release are forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Any statements that are not historical fact including, but not limited to statements that contain words such as will, believes, plans, anticipates, expects, estimates should also be considered forward-looking statements. Forward-looking statements involve risks and uncertainties. Actual results may differ materially from the results anticipated in these forward-looking statements and as such should be evaluated together with the many uncertainties that affect the business of AgeX Therapeutics, Inc. and its subsidiaries, particularly those mentioned in the cautionary statements found in more detail in the Risk Factors section of AgeXs Annual Report on Form 10-K and Quarterly Reports on Form 10-Q filed with the Securities and Exchange Commissions (copies of which may be obtained at http://www.sec.gov). Subsequent events and developments may cause these forward-looking statements to change. AgeX specifically disclaims any obligation or intention to update or revise these forward-looking statements as a result of changed events or circumstances that occur after the date of this release, except as required by applicable law.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191226005045/en/

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AgeX Therapeutics Announces Drawdown of Second Tranche of Loan Facility from Juvenescence Ltd. - BioSpace

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Global Allogeneic Stem Cells Market 2020-2024 | Evolving Opportunities with Biosolution Co. Ltd. and Cynata Therapeutics Ltd. | Technavio – Yahoo…

Posted: December 23, 2019 at 9:46 am

Technavio has been monitoring the global allogeneic stem cells market and the market is poised to grow by USD 1.24 billion during 2020-2024 at a CAGR of over 12% during the forecast period. Request Free Sample Pages

This press release features multimedia. View the full release here: https://www.businesswire.com/news/home/20191218005455/en/

Technavio has announced its latest market research report titled global allogeneic stem cells market 2020-2024. (Graphic: Business Wire)

Read the 131-page research report with TOC on "Allogeneic Stem Cells Market Analysis Report by geography (Asia, Europe, North America, and ROW), by application (regenerative therapy and drug discovery and development), and segment forecasts, 2020-2024".

https://www.technavio.com/report/allogeneic-stem-cells-market-industry-analysis

The new product approvals and special drug designations are anticipated to boost the growth of the market. Based on the application, the allogeneic stem cells market has been segmented into regenerative therapy and drug discovery and development. Manufacturers are increasingly emphasizing innovations and improvisation in the development of regenerative therapies. Many of the regenerative therapeutic candidates have obtained approval for clinical trials in the US, Europe, and APAC due to the efficacy of allogeneic stem cell therapeutics. This is encouraging market players to launch new product lines to stimulate the overall product demand for stem or regenerative therapy using allogeneic stem cell therapeutics and provide better options for their customers. Thus, new product approvals are expected to drive market growth during the forecast period.

Buy 1 Technavio report and get the second for 50% off. Buy 2 Technavio reports and get the third for free.

View market snapshot before purchasing

Major Five Allogeneic Stem Cells Market Companies:

Biosolution Co. Ltd.

Biosolution Co. Ltd. is headquartered in South Korea (Republic of Korea) and operates the business under its Unified business segment. The company offers an allogeneic keratinocyte spread medication, Keraheal-Allo, that promotes skin regeneration.

Cynata Therapeutics Ltd.

Cynata Therapeutics Ltd. is engaged in the discovery, development, licensing, manufacturing, marketing, distribution, and sales of innovative therapeutics for the treatment of various diseases. The company provides a mesenchymal stem cell product, Cymerus, which is used to treat graft-versus-host disease.

JCR Pharmaceuticals Co. Ltd.

JCR Pharmaceuticals Co. Ltd. is headquartered in Japan and operates under two business segments, namely Pharmaceuticals, and Medical Devices and Laboratory Equipment. The company offers a regenerative medical product, TEMCELL HS Injection, which uses human mesenchymal stem cells for the treatment of acute graft-versus-host disease.

Lineage Cell Therapeutics Inc.

Lineage Cell Therapeutics Inc. is headquartered in the US and offers products through its Unified business segment. The company provides OpRegen, which is currently being tested in a Phase I/IIa clinical trial. This product is intended for the treatment of dry AMD.

MEDIPOST Co. Ltd.

MEDIPOST Co. Ltd. is headquartered in South Korea (Republic of Korea) and offers products through its Unified business segment. The company provides an allogeneic umbilical cord blood-derived mesenchymal stem cell drug, CARTISTEM, which is used for the treatment of knee cartilage defects.

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Allogeneic Stem Cells Application Outlook (Revenue, USD Million, 2020-2024)

Allogeneic Stem Cells Regional Outlook (Revenue, USD Million, 2020-2024)

Technavios sample reports are free of charge and contain multiple sections of the report, such as the market size and forecast, drivers, challenges, trends, and more. Request a free sample report

Related Reports on Health Care include:

Cancer Stem Cell Therapeutics Market Global Cancer Stem Cell Therapeutics Market by type (allogeneic stem cell transplant and autologous stem cell transplant) and geography (Asia, Europe, North America, and ROW).

About Technavio

Technavio is a leading global technology research and advisory company. Their research and analysis focus on emerging market trends and provides actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions.

With over 500 specialized analysts, Technavios report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavios comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191218005455/en/

Contacts

Technavio ResearchJesse MaidaMedia & Marketing ExecutiveUS: +1 844 364 1100UK: +44 203 893 3200Email: media@technavio.com Website: https://www.technavio.com

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Global Allogeneic Stem Cells Market 2020-2024 | Evolving Opportunities with Biosolution Co. Ltd. and Cynata Therapeutics Ltd. | Technavio - Yahoo...

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Mums plea for stem cell donor to treat her cancer after her ex killed teen son who could have been a match – The Sun

Posted: December 23, 2019 at 9:46 am

A MUM is pleading for a stem cell donor to treat her cancer after her ex killed her teenage son - who may have been her only match.

Tania Morris, 49, was left battered and bruised by former lover Robert Goodwin in a row over fast food last year.

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The monster went on to bludgeon her 19-year-old son Nathan Bates to death while on bail for the attack, before killing himself.

In the latest horror twist, Tania now fears this Christmas could be her last after she was diagnosed with Hodgkin's Lymphoma shortly after burying her only child.

Tania's rare tissue type means there is no one on the entire global register who provides a match.

The pottery worker from Burslem told StokeonTrentLive: "It's heartbreaking. The doctors just keep saying we need a fit and healthy 19-year-old and that breaks my heart as that's how old Nathan was when he was murdered.

"My younger brother could have been a match but he died of a heart attack.

"My other brother Darren was devastated when he was tested and wasn't a match. He wanted so much to help me.

"Dad's only a half match. He's too poorly himself to go ahead but if and when it becomes life or death he could be a last resort. They're worried it would kill both me and him.

"It would really be desperate measures if it comes to that. It would be my last option because it could kill me.

"My mum's not even been tested because she's had a heart bypass.

"We are just hoping that someone comes forward. It's my only chance of beating this."

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Tania was left with horrific injuries in August 2018, when sick Goodwin tried to gauge out her eye before smashing a porcelain doll ornament over her face.

Months later he killed Nathan with a hammer as he slept before killing himself in nearby woodland on October 11 last year.

Tania is still unclear what motivated the murder - that may indirectly lead to her death as well.

The doctors just keep saying we need a fit and healthy 19-year-old and that breaks my heart as that's how old Nathan was when he was murdered.

Goodwin had been due in court the day after Nathans killing to face the assault charge against Tania - but will now never face justice for either offence.

Since her diagnosis in January, Tania has gone through multiple rounds of chemotherapy, but has now been told that the treatment hasn't worked.

She is calling on any young donor to come forward in what she thinks could be her only shot at living.

"I haven't properly grieved for Nathan because of the cancer," she said.

"He just couldn't cope with the thought of going to prison so he killed Nathan, killed himself and if I don't find a donor, he may yet kill me. All the stress he's put our family through also led to my mum's heart attack.

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"He had no reason to do what he did, he just wanted to upset me in the worst possible way.

"He said he loved me to pieces but then he did this."

Anyone who is healthy and aged 16-30 can donate stem cells by signing up with the Anthony Nolan register here.

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Mums plea for stem cell donor to treat her cancer after her ex killed teen son who could have been a match - The Sun

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Developing next-generation biologic pacemakers with stem cells – Health Europa

Posted: December 23, 2019 at 9:46 am

University of Houston associate professor of pharmacology Bradley McConnell is helping to develop new cardiac pacemakers by using stem cells found in fat, converting them to heart cells, and reprogramming those to act as biologic pacemaker cells.

The new biologic pacemaker-like cell will be useful as an alternative treatment for conduction system disorders, cardiac repair after a heart attack and to bridge the limitations of the electronic pacemaker.

The work was published in the Journal of Molecular and Cellular Cardiology.

We are reprogramming the cardiac progenitor cell and guiding it to become a conducting cell of the heart to conduct electrical current, said McConnell.

McConnells collaborator, Robert J. Schwartz, Hugh Roy and Lillian Cranz Cullen Distinguished Professor of biology and biochemistry, previously reported work on turning the adipogenic mesenchymal stem cells, that reside in fat cells, into cardiac progenitor cells.

Now those same cardiac progenitor cells are being programmed to keep hearts beating as a sinoatrial node (SAN), part of the electrical cardiac conduction system (CCS).

The SAN is the primary pacemaker of the heart, responsible for generating the electric impulse or beat. Native cardiac pacemaker cells are confined within the SAN, a small structure comprised of just a few thousand specialised pacemaker cells. Failure of the SAN or a block at any point in the CCS results in arrhythmias.

More than 600,000 electronic pacemakers are implanted in patients annually to help control abnormal heart rhythms.

The small mechanical device is placed in the chest or abdomen and uses electrical pulses to prompt the heart to beat normally. In addition to having the device regularly examined by a physician, over time an electronic pacemaker can stop working properly.

McConnell said: Batteries will die. Just look at your smartphone.

This biologic pacemaker is better able to adapt to the body and would not have to be maintained by a physician. It is not a foreign object. It would be able to grow with the body and become much more responsive to what the body is doing.

To convert the cardiac progenitor cells, McConnell infused the cells with a unique cocktail of three transcription factors and a plasma membrane channel protein to reprogram the heart cells in vitro.

In our study, we observed that the SHOX2, HCN2, and TBX5 (SHT5) cocktail of transcription factors and channel protein reprogrammed the cells into pacemaker-like cells. The combination will facilitate the development of cell-based therapies for various cardiac conduction diseases, he reported.

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LncRNA KLF3-AS1 in Human Mesenchymal Stem Cell-Derived Exosomes Ameliorates Pyroptosis of Cardiomyocytes and Myocardial Infarction Through…

Posted: December 23, 2019 at 9:46 am

AIM:

Myocardial infarction(MI) is a severe disease with increased mortality and disability rates, posing heavy economic burden for society. Exosomes were uncovered to mediate intercellular communication after MI. This study aims to explore the effect and mechanism of lncRNA KLF3-AS1 in exosomes secreted by human mesenchymal stem cells (hMSCs) on pyroptosis of cardiomyocytes and MI.

Exosomes from hMSCs were isolated and identified. Exosomes from hMSCs with transfection of KLF3-AS1 for overexpression were injected into MI rat model or incubated with hypoxia cardiomyocytes. Effect of KLF3-AS1 on MI area, cell viability, apoptosis, and pyroptosis was determined. The relationship among miR-138-5p, KLF3-AS1, and Sirt1 was verified by dual-luciferase reporter assay. Normal cardiomyocytes were transfected with miR-138-5p inhibitor or sh-Sirt1 to clarify whether alteration of miR-138-5p or sh-Sirt1 can regulate the effect of KLF3-AS1 on cardiomyocytes.

Exosomes from hMSCs were successfully extracted. Transfection of KLF3-AS1 exosome in rats and incubation with KLF3-AS1 exosome in hypoxia cardiomyocytes both verified that overexpression of KLF3-AS1 in exosomes leads to reduced MI area, decreased cell apoptosis and pyroptosis, and attenuated MI progression. KLF3-AS1 can sponge miR-138-5p to regulate Sirt1 expression. miR-138-5p inhibitor transfection and KLF3-AS1 exosome incubation contribute to attenuated pyroptosis and MI both in vivo and in vitro, while transfection of sh-Sirt1 could reverse the protective effect of exosomal KLF3-AS1 on hypoxia cardiomyocytes.

LncRNA KLF3-AS1 in exosomes secreted from hMSCs by acting as a ceRNA to sponge miR-138-5p can regulate Sirt1 so as to inhibit cell pyroptosis and attenuate MI progression.

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Making advanced therapies takes industrializing personalization – STAT

Posted: December 23, 2019 at 9:46 am

Whats the best way to measure the real rate of progress in personalized cell therapies, gene therapies, and other advanced therapies?

Ive been tracking the ever-growing flow of reports about these therapies in scientific journals and press releases for 15 years, ever since I co-led the passage of Californias $3 billion Stem Cell Research and Cures Act in 2004.

But to truly gauge who will benefit from todays innovations, Ive learned I also need to study the stream of business and technology announcements that runs in parallel. That might seem more mundane but to veterans of advanced therapies, making the science work actually signals success for these gene-, tissue-, and cell-based advanced therapies.

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The reason is simple. My experience working with advanced therapies has taught me, time and again, that true next-generation medicine requires the industrialization of personalization. That sounds like an oxymoron, but it isnt. To create individualized therapeutics in a sustainable way, we need to deliver even if it seems counterintuitive mass customization.

Breakthroughs such as CAR-T cell therapies are inspiring. They are also unsustainably expensive, difficult to manufacture, and complicated to deliver. We can change this by creating a more focused cross-collaborative production and delivery ecosystem.

The Food and Drug Administration anticipates that it will approve 10 to 20 advanced therapies a year beginning in 2025. It also expects to receive up to 200 clinical trial applications for cell and gene therapies per year, starting now. The more than 1,000 advanced therapy clinical trials now underway worldwide could enroll almost 60,000 patients, according to the Alliance for Regenerative Medicine. That pace wont be possible without new systems and networks that reduce cost, simplify manufacturing, and streamline delivery.

I can see some of these on the horizon when I read the biotech and pharma partnerships reported in BioSpace and BioCentury. Of the 100 most recent, almost 10% were dedicated to cell- and gene-therapy companies and organizations. These partnership announcements are typically viewed as opportunities to highlight new business deals or contract wins. But they are also daily snapshots of the infrastructure of an evolving next-generation health care system forming from within. Here are just a few examples from 2019:

Its encouraging to see biopharma manufacturing, logistics, transport, and other partners in the cell- and gene-therapy ecosystem coming together in new ways to ensure the successful and reliable delivery of advanced therapies for individual patients. But much more evolution is needed to provide sustainable patient access to advanced therapies.

We need even more industry collaboration to overhaul and connect existing health care systems, so production and delivery of cell- and gene-based therapies can be more automated and affordable. According to estimates from credible industry colleagues and leaders, end-to-end automation can shave costs by at least 20% to 30%, and at the same time greatly improve predictability and patient safety.

We must also make this new world simpler for health care providers. Doctors and nurses must not only understand how advanced therapies work medically, but be able to order and deliver them safely with a minimum of delay or hassle. As noted in the New Yorker, CAR-T requires bringing a manufacturing lens to medicine. Supporting health care providers means creating true collaboration between digital technology providers, hospitals, logistics providers, biotech and pharma companies, and manufacturing, like the Boston initiative I described earlier.

Standardization is often decried as cookie-cutter medicine. In this space, however, it is the wave of the future.

While patient biology is unique, and each patients cells may produce a one-of-a-kind manufacturing batch, essential parts of the production and delivery process should be as predictable and easy as possible. One key place to start is in-process drug labeling. When patients cells become the raw material for advanced therapies, these labels become more complex and more necessary: When a patient is about to receive a cell therapy infusion, its essential that the name on the bag of genetically re-engineered cells is his or hers. The Standards Coordinating Body, an FDA-funded but independent nonprofit, is now leading an industry-wide labeling initiative for cell and gene therapies.

There are other clear signs that the advanced therapies field gets it when it comes to infrastructure needs, such as the inclusion of digital health and handling of patient data as categories of focus in the federal Cures 2.0 initiative currently circulating in Washington. But much remains to be done.

In centers caring for individuals with cancer and rare diseases, thousands of patients are today receiving advanced therapies that are transforming their lives. We need to make that possible for many, many more by working together to industrialize and personalize in parallel.

Amy DuRoss is the CEO and co-founder of Vineti, a digital technology company that provides next-generation software platforms for advanced therapies. Before that she was managing director for new business creation for GE Ventures, chief business officer at Navigenics, the co-founder and executive director of Proposition 71, Californias $3 billion stem cell research initiative that passed in 2004, and chief of staff at the resulting California Institute for Regenerative Medicine.

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Pike River widow ‘on the up’ after stem cell treatment for cancer – Stuff.co.nz

Posted: December 23, 2019 at 9:46 am

Pike River widow Anna Osborne is "on the up" and out of hospital in time for Christmas.

Osborne, whose husband Milton died in the 2010 Pike River mine disaster, had stem-cell treatment for Hodgkinlymphoma in October.

She had been told she only had a month to a year to live without it.

Phil Walter/Getty

Anna Osborne, from the Pike River Family Reference Group, embraces Prime Minister Jacinda Ardern at the mine entrance earlier this year.

Friend and Pike River mother SonyaRockhouse said Osborne's treatment went well, but there was still a long road ahead.

READ MORE:*Pike River mine tunnel entry an important moment for widow*Pike River re-entry team breaks through into mine drift*Pike River widow 'full of nerves' for mine drift re-entry*The road to getting back into Pike River

"I think the treatment is working for now. She just got her bloods done and they were good and they are the most important thing," she said.

Kevin Stent/Stuff

Osborne and Sonya Rockhouse at the announcement the Government would re-enter the Pike River mine.

Osbornewasdiagnosed with Hodgkinlymphoma in 2002 when she was 36.

She had radiation for six weeks and went into remission, but the cancercame back just before the Pike River tragedy in November 2010, when 29 men where killed in a series of explosions at the coal mine. Osborne helped campaign for thelegalisation of medicinal cannabiswhile undergoing chemotherapy in 2015.

Her stem-cells wereharvested and frozenin August. The stem cell transplanttook place in Christchurch in October aftersix days of intensive chemotherapy.

JOANNE CARROLL/Stuff

Anna Osborne, pictured during treatment for Hodgkins lymphoma in 2016.

The treatment had its own risks.

Osborne was in isolation for five weeks but after shereturned home, she hadsome set backs and small complications,Rockhousesaid.

"She was so crook. She lost a lot of weight. She's had two or three trips to hospital since then," she said.

Supplied/Pike River Recovery Agency

Mine worker Bryan Heslip offers a hand to Osborne and Rockhouse after entering the Pike River mine drift during the re-entry operation.

"She's on the up now,but [there is] still a long way to go. She's at home and is getting some colour back in her cheeks, [and is] starting to look like her old self."

Rockhouse said Osborne was focusing on her recovery and hoped to be able to go to the Pike River mine for the next milestone, which was removing the 170m seal expected to take place in January.

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Many stem cell clinics promise unapproved treatments: How to stay safe – NBC News

Posted: November 25, 2019 at 8:45 pm

Google has cracked down on pervasive, predatory ads promoting sketchy medical treatments a move that could affect many of the hundreds of stem cell treatments advertised online.

At the end of October, the internet giant stopped accepting ads for unproven or experimental medical techniques, according to a statement from the company. Google is taking this step after seeing a rise in bad actors offering deceptive and untested treatments, the company said.

Stem cells came up this week during the senate confirmation hearing for the next head of the Food and Drug Administration on Wednesday.

"FDA has taken enforcement action, which it should, against stem cell clinics and manufacturers that might be misleading people," Sen. Lamar Alexander (R-Tenn.) said. "On the other hand, we have diabetes advocates who say regenerative medicine may restore pancreas or put out of business a heart transplant surgeon by restoring a heart. Will you commit to taking seriously the promise of regenerative medicine and stem cell medicines?"

Dr. Stephen Hahn, President Trump's nominee to lead the agency, replied that he would "certainly commit to that."

The number of stem cell clinics in the U.S. doubled every year from 2009 to 2014, according to a report in the journal Regenerative Medicine.

Major renowned medical centers such as the Mayo Clinic are researching stem cell therapies for conditions including arthritis and heart problems.

But as stem cell clinics not affiliated with medical centers proliferate, so have serious concerns. In 2017, the New England Journal of Medicine reported that three women were virtually blinded from unproven "stem cell" treatments that actually involved having fat injected into their eyes.

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That has brought increased scrutiny from the FDA, which in June won a court ruling allowing the agency to regulate treatments at the Florida clinic where the three women were harmed.

Stem cell products have only been approved for two uses by the agency: for blood cancers and certain immune disorders. That hasnt stopped some providers from claiming their stem cell treatments can "cure" a wide range of conditions, from macular degeneration to multiple sclerosis unapproved treatments that are not only not effective, but could pose serious health risks.

Stem cells are very young cells that can develop into specialized cells, such as blood cells, bone cells and brain cells. According to the FDA, stem cells have the potential to replace, repair, restore and regenerate other cells, and could possibly be used to treat a number of medical conditions.

The only stem cell-based products currently approved by the FDA consist of blood stem cells derived from umbilical cord blood. These products are usually used in patients with blood cancers, such as leukemia and immune disorders. For patients with blood cancers, for example, stem cells are used after chemotherapy to replenish the blood cells that were destroyed during the treatment.

However, other uses are being studied. There are currently more than 650 active clinical trials for stem cell procedures, looking at a myriad of conditions including kidney failure and Crohn's disease.

But the purpose of a clinical trial is to determine if a treatment works or if it's safe.

People should understand that they're trying something that could potentially have benefit, Dr. Peter Marks, FDA director of biologics, said. But we don't know that benefit is really there, which means that they should understand the risk.

We do our best to make sure people are safe when they're getting investigational products, Marks said. When a product is in a clinical trial, the FDA can monitor it to make sure its safe, he added.

For patients considering stem cell treatments, the FDA recommends asking a health care provider if the FDA has reviewed the treatment.

This also applies to stem cell treatments that use a person's own cells. You may be told that because these are your cells, the FDA does not need to review or approve the treatment. That is not true, according to the FDA website.

The FDA has given that road map on how to do it responsibly, said Dr. Shane Shapiro, medical director at the Mayo Clinics Regenerative Medicine Therapeutics Program in Jacksonville, Florida.

Shapiro also recommends going to a provider that has an expertise in the disease or injury that they are offering stem cell products to treat.

I think it is important to point out when we see a patient at Mayo, they are still seeing someone with the expertise of a physician that is a specialist in the disease they need help with, he said.

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Patrick Martin is an associate producer in the NBC News Health & Medical Unit.

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Tendon stem cell discovery could lead to improvements in injury recovery – Drug Target Review

Posted: November 25, 2019 at 8:45 pm

Researchers have discovered the existence of tendon stem cells, which could lead to improvements in treating tendon injuries, avoiding surgery.

New research has revealed the existence of tendon stem cells which could potentially be harnessed to improve tendon recovery after an injury and perhaps even avoid surgery.

The research was led by Chen-Ming Fan at the Carnegie Institution of Science, US.

Once tendons are injured, they rarely fully recover, which can result in limited mobility and require long-term pain management or even surgery. This is due to fibrous scars, which disrupt the tissue structure of the tendon.

This image shows the Patellar tendon 30 days after an injury. The red marks newly discovered tendon stem cells that have self-renewed and are layered over green marked, original tendon cells. During regeneration, some tendon stem cells differentiate to make newly regenerated tendon cells a process during which they transition into a yellow-orange colour. The blue indicates cellular nuclei (credit: Tyler Harvey).

Fan, along with Carnegies Tyler Harvey and Sara Flamenco, revealed all of the cell types present in the Patellar tendon, found below the kneecap, including previously undefined tendon stem cells.

Because tendon injuries rarely heal completely, it was thought that tendon stem cells might not exist, said lead author Harvey. Many searched for them to no avail, but our work defined them for the first time.

The teams research showed that both fibrous scar tissue cells and tendon stem cells originate in the same space the protective cells that surround a tendon. Moreover, these tendon stem cells are part of a competitive system with precursors of fibrous scars, which explains why tendon healing is such a challenge.

It was thought that tendon stem cells might not exist but our work defined them for the first time

The team demonstrated that both tendon stem cells and scar tissue precursor cells are stimulated into action by a protein called platelet-derived growth factor-A. When tendon stem cells are altered so that they do not respond to this growth factor, then only scar tissue and no new tendon cells form after an injury.

Tendon stem cells exist, but they must outcompete the scar tissue precursors in order to prevent the formation of difficult, fibrous scars, Fan explained. Finding a therapeutic way to block the scar-forming cells and enhance the tendon stem cells could be a game-changer when it comes to treating tendon injuries.

The research was published in Nature Cell Biology.

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Tendon stem cell discovery could lead to improvements in injury recovery - Drug Target Review

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