Is FDA slowing down the progress in clinical cell therapy?

by Alexey Bersenev on November 27, 2011 · 2 comments

in cell product, RegenMed digest

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This question is currently highly discussed in professional communities. Professionals think that too strict or/ and unclear regulation system is one of the main hurdles in translation of cell therapy and stem cell research into the clinic.

The FDA is a regulatory agency which approves the use of biological products (including cells) and drugs for medical use in US. Even though the FDA has a set of rules for cell products in place, in some cases there is still misunderstanding and unclear regulation. For example, some cell therapy developers argue that autologous minimally manipulated cells should not be regulated as conventional drugs or regulated by Federal Agency at all. The definition of minimally manipulated remain somewhat fuzzy. The clash between Colorado’s company Regenerexx and FDA on this matter is still continuing. It seems like for cell therapy industry and biotech, things could get even tougher.

Recently, Paul Knoepfler offered a poll on how FDA does its job in regulation of stem cell clinical trials:

A surprising 83% of respondents want the FDA to move faster, and only 17% were happy with the status quo.

In the recent GEN poll “Does the FDA do as much harm as good?”, 49.2% Biotech professionals said “YES“!

However, some professionals think that the FDA does a good job in regulation of cell and stem cell products. Among them I’d notice some scientists and physicians, who are not necessarily involved in development of cell therapies.

Patients, who potentially could benefit from cell therapy, are very frustrated about the length of stem cell clinical trials regulated by the FDA. The vast majority of patients think, that over-regulation of cell therapy trials by the FDA forces them to look for alternatives outside the country. Thus, unintentionally, FDA could fuel the spread of unregulated “stem cell tourism”. This reaction from patients could be explained by desperate quest for cure (they can not wait!) and lack of educational materials.

Most business professionals are frustrated about FDA over-regulation or uncertainty. The famous 20 000 pages Geron’s case is a legend right now. Why did Geron’s case take so long to get a green light? Why is the cell therapy protocols approval process for all other companies and even for academic institutions so long?

The very recent, highly discussed topic is a shift of life sciences venture funds from US to overseas:

National Venture Capital Association released results of a survey that said the FDA – plagued by long delays, unpredictable decisions, poor communication, and excessive caution in new product reviews – has become the No. 1 reason why 40 percent of U.S. venture capitalists have cut their investments in life sciences the past three years, and that the same percentage plan to continue cutting U.S. investment the next three years. Jobs have been lost, and even more will be shipped overseas unless something is done at the FDA, the investors said.

Seems like this could be the case for cell and stem cell therapy. Needless to say how much cell therapy and Regen startups depend on venture capital! Of course, this trend worries business developers very much:

Venture funds are moving overseas to countries where regulations are simpler. Everyone knows that new drugs, stem cell therapies, and medical devices are extremely expensive to research, develop, get approved, and commercialize. Without much-needed venture funds will we see a drop in innovation and progress in medical advancements in the United States (US); are we falling behind?

So, majority of patients, medical professionals and business developers, involved in cell therapy, currently think that FDA is over-regulating and, therefore, holding progress back in the field. How can FDA handle the situation? Does FDA need some reforms? Probably yes, but It’s not that simple. I’d like to cite what Luke Timmerman recently said about FDA:

The FDA is one of the easiest punching bags in American politics. Depending on your point of view, it’s either too hard on business with its unreasonable demands, or too soft on those predatory drug companies seeking to profit off Grandma’s illness regardless of whether the products are safe.

I worry that the FDA could get distracted from its main job of ensuring product safety and effectiveness, if it cares too much about industry’s wishes. And there are plenty of companies willing to cut corners and do the quick-and-dirty thing whenever they can slip it by the FDA, so there has to be a well-resourced, tough watchdog on alert.

It is definitely hard to make happy everyone, but ideally, the FDA should keep some kind of “healthy balance”. Don’t unleash the industry completely, but don’t slow the progress down by over-regulation.

I think, the key is an open dialogue and transparency. It will be good if FDA will always ask for feedback and will seek for professional advice in case of uncertainty. I think, in tough cases the FDA should be always consulted by a panel of independent unbiased external experts.

I think, now FDA appreciates the fact that live cells is a very different from typical pharma- or biotech class of products. A great complexity of this class of products implies that the same regulatory path could not be applied to all of them uniformly. Probably they should be broken down into a few categories with slightly different regulatory requirements. For example, tight FDA regulation of autologous minimally manipulated cells for homologous use as a drugs could be the one extreme. But leave it completely unregulated – yet another extreme. There is no doubt that such category of products as autologous or allogeneic cells extensively manipulated ex vivo (gene modified plus few weeks in culture) for non-homologous use with high disease prevalence, should be be regulated as a drug.

So, because cell and stem cell therapy field currently in rapid development and not very well establish yet, FDA should openly discuss with experts how to regulate it better. How to move the progress fast and safe enough in this evolving field.

Finally, I’d like to point out that recently the FDA did a good job for public education and transparency. For example, educational webcasts about cell therapy products regulation. Few weeks ago, FDA publicly shared an Advisory Committee Meeting, which was discussing the licensing of cord blood products. You can actually watch how FDA discusses the tough issues with the panel of experts. I think, this is a good move!

Please tell us – what do you think?

RegenMed Digest on Stem Cell Assays is sponsored by Regenerative Medicine Jobs. RegenMed is hiring today! Please visit Regenerative Medicine Jobs for recent position openings.

{ 2 comments… read them below or add one }

Barbara Hanson November 29, 2011 at 2:34 am

I think it is nice to see this issue being discussed. There is a balance that must be achieved. The FDA is over regulating, but at the same time, there are unscrupulous companies out there that take advantage whenever they can and put patient’s at risk. The U.S. will also lose jobs and we will fall behind if there isn’t a positive change. The costs involved in getting FDA approval have gotten out of hand. There is a certain mindset it seems of doing business as usual. It’s time to modernize the whole process. Patients will continue to go offshore to get treatment as they are disillusioned by the lack of progress in the U.S.


Leeza Rodriguez August 15, 2012 at 9:10 am

How about a separate category for Adult, Autologous Stem Cell Therapy in the FDA regulations?

There is clearly a difference is risk profile between Adult, Embryonic, and Induced Pluripotent. All of the early phase Clinical Trials point to endpoints of Safety and Efficacy for Autologous Adult Stem Cell Therapy. It’s no secret that Adult Stem Cells have a lower risk profile than ESC and IPS cells. Why not treat Adult as a separate category with lesser layers of regulation? In the same capacity, within Adult, why not make a tiered criteria for Freshly Isolated vs. Culture Expanded. For example:

Class A: Induced Pluripotent (most manipulation= highest risk profile)
Class B: Embryonic Stem Cells
Class C: Adult (lowest risk profile)
1) Freshly Isolated same operative session
2) Culture Expanded

Not only does this granulate Risk Profile for patients, but it gives the Media and the Public a tiered approach to passing judgements. I am really tired of the critics putting all Stem Cell Therapies and Offshore Clinics in the same pot. This is very confusing for the public , and frankly a disservice to patients.

Thanks for starting the dialogue!

Leeza Rodriguez


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