Cells Weekly – May 12, 2013

by Alexey Bersenev on May 12, 2013 · 0 comments

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Cells Weekly! is a digest of the most interesting news and events in stem cell research, cell therapy and regenerative medicine. We pick and post it every Sunday. Follow us!

1. Circulating factor able to “rejuvenate the heart”
For a decade, Harvard’s investigator Amy Wagers was looking for factors circulating in the blood and able to rejuvenate aged organs and tissues. Her studies, methodologically based on conjunction of circulation young and old animals, finally brought some results. For the first time, Wagers and Richard Lee’s labs were able to identify a factor, circulating in blood of young animals – Growth Differentiation Factor 11 (GDF-11), which reversed age-related heart hypertrophy.

Obviously, this is just one factor from many. Taking in account rapidly increasing aged population, this study can provide a platform for future identification of other “rejuvenating” factors and have significant impact in regenerative medicine.

Also watch Cell video abstract.

2. Japan proposed a rapid track for stem cell-based therapies regulation
Nature reported on new proposal for regulation of stem cell therapies in Japan:

A retooling of Japan’s drug authorization framework, on its way to becoming law, could produce the world’s fastest approval process specifically designed for regenerative medicine.

This proposal reminds me the previous attempt to implement fast track approval for cell therapies in South Korea.

Rather than using phased clinical trials, companies will have to demonstrate efficacy in pilot studies of as few as ten patients in one study, if the change is dramatic enough, or a few hundred when improvement is more marginal.

With the bar for regenerative therapies dramatically lowered by requiring only limited safety and efficacy data—and essentially doing away with the need for high-powered phase 3 trials—the amendments’ architects say it will be possible to get a stem cell treatment to the market in just three years, rather than the typical six or more.

We will watch how this proposal will be accepted and implemented in Japan.

3. More concerns about non-compliant stem cell clinics in US
Professor Leigh Turner continues to fight with non-compliant stem cell clinics in US. This week he posted an analysis of Precision StemCell clinic:

On December 13, 2012, concerned about the safety of the increasing number of individuals with ALS receiving adipose-derived stem cells at Precision StemCell, I contacted the FDA’s Center for Biologics Evaluation and Research and urged FDA officials to investigate the clinic. I took that step because it appeared to me that Dr. Williams was violating federal regulations, putting stem cell recipients at risk of harm, and charging for interventions that had not first been tested for safety and therapeutic efficacy. Dr. Williams’ administration of stem cells to a minor also played a role in my decision to send a letter to the FDA.

Will FDA investigate this case. More likely YES. Has FDA a muscle to capture and inspect all clinics like that in US? More likely NO.

4. More on Italian stem cell therapy regulation scandal
We’re following the recent scandalous case of deregulation of stem cell therapy in Italy. This topic is highly discussed all over online professional communities for the last 2-3 weeks. This week, physician-scientist and popular blogger David Gorski posted here and here about Italian case:

One point that the scientists didn’t really nail, and that’s the issue of informed consent. One notes that Vannoni’s stem cell quackery has no evidence for it published in the peer-reviewed biomedical literature nor any compelling clinical trial results. Consequently, if claims are being made for this treatment it is impossible to give informed consent because there is no evidence upon which to base even a rough estimate of the chances for success are weighed against the risks of the treatment. Even worse, we don’t even know that these are really stem cells. Seriously. As the scientists point out, there is no transparency, and if there’s an area of clinical research where transparency is essential, it’s stem cell research.

His post generated nearly 200 comments from both blogs! I’d highly recommend you to read his post and comments here and here.

5. iPS cell conventional wisdom
Paul Knoepfler polled readers few times on his blog about roadblock issues in iPS cell field. This week, he summarized the results of 4 polls in one post:

The overall gestalt is that iPS cells are unlikely to be immunogenic so that’s really not a big concern today, but people do remain concerned, first of all, about epigenetic issues such as epigenetic blemishes in iPS cells including memories ,and secondly most people are still convinced that iPS cells have a handful of mutations.

Very interesting and importnat information!

6. Skin into the heart – more on cellular alchemy
Professor Jalees Rehman in his recent post discusses the conversion of fibroblasts into functional heart cells:

Where does this whole body of work leave us? One major finding seems to be fairly solid. Fibroblasts can be converted into beating heart cells. The efficiency of conversion and the quality of the generated heart cells – from mouse or human fibroblasts – still needs to be optimized. Even though the idea of cellular alchemy sounds fascinating, there are many additional obstacles that need to be overcome before such therapies could ever be tested in humans. The method to introduce these genes into the fibroblasts used viruses which permanently integrate into the DNA of the fibroblast and could cause genetic anomalies in the fibroblasts. It is unlikely that such viruses could be used in patients.

7. Presentations from webinar on stem cell clinical trials
A month ago CIRM was held a webinar: Clinical Trials: Moving Stem Cell based Therapies to the Clinic. Presentation were recorded and this week posted on CIRM YouTube channel:
John Hyde of FDA – First-in-Human Trials of Cellular Therapies
Eduardo Marbán – Lessons learned from Phase 1 trial of heart-derived stem cells as we move into Phase 2
Jonathan Glass – Lumbar Intraspinal Injection of Neural Stem Cells in Patients with ALS

My favorite quote from Glass:

Why stem cells is a magic? Because we don’t know if it’s working or not!

Have a wonderful week, folks!

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