Cells Weekly – July 20, 2014

by Alexey Bersenev on July 20, 2014 · 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. Cells Weekly is posted every Sunday night!

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1. Criticism of “cord blood for autism” trial
Criticism of recently launched autism trial at Duke U is keep coming. SFARI posted a big piece about this trial, citing stem cell scientist Arnold Kriegstein:

“It’s probably premature to run large trials without evidence that they have a therapeutic effect that [we] understand,” cautions Arnold Kriegstein…
“These are not cells that can treat a laundry list of diseases,” he says. Because the stem cells are similar to those that normally give rise to blood cells, he says, it is unlikely that they can repair or replace neurons in the brain. Also, because autism results from errors during development, it is unlikely that the stem cells can reverse those effects…

It was re-posted by Scientific American and went mainstream. Many stem cell researchers agree with Kriegstein, for example Jeanne Loring:

I side with Arnold Kriegstein on this issue, and am glad that he is willing to be so straightforward.

I’m afraid that studies like these are used as rationalization for charlatans.

I don’t see the reason for such “hysteria”. Many cell therapy trials, approved by regulatory agencies, lack of strong scientific background. This one is not an exclusion, but, at least has a rationale. Many human conditions have no good animal models and the way to test it is via approval for first-in-human trial. And what can we learn for such criticism? Do you remember the same kind of criticism for same kind of US-based trial 2 years ago? It’s over and over again. Was this criticism valuable? What did we learn from it?

2. Whistleblower is suing stem cell company
This week, we witnessed unique case in cell therapy history – former employee sues stem cell therapy company:

Rob Williams sued StemCells Inc. on Monday in Alameda County Court, alleging wrongful termination, retaliation and violation of the California False Claims Act.

Apparently, he saw a lot of deficiencies in stem cell product manufacturing, which are not in compliance with GMP rules and put patients safety at risk:

“Shortly after beginning his employment, plaintiff noted poor sterile technique, failure to adhere to current Good Manufacturing Practices in the company’s manufacturing process, and substantial deficiencies in the company’s Manual Aseptic Processing of HuCNS-SC (Human Central Nervous System Stem Cells) cell lines – failure and deficiencies that put patients at risk of infection or death during ongoing clinical trials,” Williams says.

After failed attempts to fix these deficiencies by reporting to upper management, he was fired by company. California Stem Cell Report blog nicely followed this story here, here and here. Company’s representative responded via email to California Stem Cell Report:

“The Company has reviewed the complaint filed by Mr. Williams, a former employee whose employment was terminated for performance deficiencies, and finds no merit to the allegations…”

As far as I know, this is the first scandalous whistleblower-related case in the history of cell therapy.

3. Current state and controversies in cardiac cell therapy
In relation to recent controversy around cardiac stem cells and Anversa’s lab, good critical analysis have been recently written. First, I’d like to highlight excellent blog post from Jalees Rehman – The Road to Bad Science Is Paved with Obedience and Secrecy. He went through history of discoveries and controversies, surrounding cardiac cell therapy:

Considering the size of the Anversa group – consisting of 20, 30 or even more PhD students, postdoctoral fellows and senior scientists – it is puzzling why the discussions among the group members did not already internally challenge their hypotheses and findings, especially in light of the fact that they knew extramural scientists were having difficulties replicating the work.

Another good piece, was published in the last issue of Science and entitled “The elusive heart fix” (unfortunately, behind paywall). This piece is full of “lessons to learn” and great quotes from professionals in the field. Some of my favorite quotes:

Charles Murry: “Everybody has wanted to be the first toregeneratetheheart.”

Cardiologists acknowledge that cell therapy was marked by outsize expec- tations. “We all hype our work,” says Steven Houser, a cardiac muscle biologist at Temple University… “the hype got ahead of the science.”

…scientists publish positive results far more often than negative ones, and that may imply that a novel treatment is more promising than it really is.

… the science is in a state of flux. The idea that bone marrow cells morph into heart muscle has been largely abandoned.

The trial, called BAMI, is “unethical,” Arnesen charges, because, he believes, the therapy it’s testing has already been shown not to help.

I’d highly recommend you to read both – Rehman’s post and Science piece!

4. Somatic in vivo reprogramming makes pacemaker cells
The most interesting scientific paper of the week came up in Science Translational Medicine. Researchers used one transcription factor to reprogram cardiomyocytes in peacemaker cells in pig model of complete heart blockade:

Thus, minimally invasive TBX18 gene transfer creates physiologically relevant pacemaker activity in complete heart block, providing evidence for therapeutic somatic reprogramming in a clinically relevant disease model.

The Scientist nicely covered this paper:

The team monitored the pigs for two weeks after injections and found that the activity of the induced pacemaker cells peaked at day eight and then slowly declined. This was not a surprise because adenovirus-infected cells tend to be cleared from the body, Cingolani explained.

Such short-term reprogramming would be fine for patients requiring a temporary alternative to electronic devices, such as those undergoing treatment for pacemaker-related infections. Indeed, Cingolani said that while developing the technique his team’s aim was “to focus on these high-risk patients.” But for long-term reprogramming, an alternative vector would be necessary.

5. Talk show on experimental stem cell treatment procedures
Australian media outlet SBS recorded talk show about experimental stem cell treatments. I was really enjoying watching it, because there were all kind of people and opinions. Highly recommend you to watch!

6. HIV stem cell chronicle – Berlin Patient – Boston Patients – Australian Patients
Two new cases of “HIV clearance” by bone marrow transplantation were reported this week on 20th International AIDS Conference:

The two patients, both Australian men, became apparently HIV-free after receiving stem cells to treat cancer. They are still on antiretroviral therapy (ART) “as a precaution”, but those drugs alone could not be responsible for bringing the virus to such low levels, says David Cooper, director of the Kirby Institute at the University of New South Wales in Sydney, who led the discovery.

Even though mass media used term “cured”, we all know that it could be too premature. Remember HIV return in “Boston Patients” and “Missiscipi Baby”:

Unfortunately, several months after the ‘Boston’ patients stopped taking ART, the virus returned. An infant born with HIV in Mississippi who received antiretroviral therapy soon after birth, then stopped it for more than three years, was thought to have been cured, but has had the virus rebound, too.

7. The story of creation bioengineered blood vessels
Scientific resource Nautilus posted very nice story about creation of bioartificial organs, taking blood vessels as example:

This year, Niklason’s hollow, milky-white blood vessels are being implanted in patients enrolled in clinical trials in Poland and the United States. The first stage of testing will determine the vessels’ safety. The next stage of trials will demonstrate whether the tubes represent an improvement over existing techniques.

Highly recommended!

8. Fresh reviews:
The Billion Cell Construct: Will Three-Dimensional Printing Get Us There?
Injectable Biomaterials for the Treatment of Myocardial Infarction and Peripheral Artery Disease
Sca-1+ Cardiac Progenitor Cells and Heart-Making: A Critical Synopsis

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