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07/08/2016

Seeking the proper balance of regulation of cellular therapies

The journal Nature is editorializing against something called the REGROW Act, proposed by Senator Mark Kirk as S. 2689.  Looks like it has been referred to Senate committee and is early in the process.

A quick read of the bill is that it would direct the HHS Secretary to establish (through the FDA) a process for conditional approval of certain cellular therapies—which would include certain “adult stem cell” approaches—if they show preliminary evidence of efficacy, before big, long definitive clinical trials have been done.  FDA already has such a process in place for drugs to treat cancer or HIV.  It is called “accelerated approval” and comes with post-approval commitments from the drug company to continue with more definitive studies.  If the drug company does not follow through, the FDA can subsequently revoke the approval.  One criticism of the program is that companies are slow in completing their commitments but FDA rarely holds their feet to the fire.

It looks to me like S. 2689 asks for something similar to accelerated approval for some cellular therapies, even if life-threatening illness is not involved.  The bill would limit the proposed program to “minimally manipulated” cells or “more than minimally manipulated” cells that have not been genetically modified.  The function of the cells in a recipient would have to be the same as in the person from whom the cells were obtained.  That could be the same person, or someone else as a donor.  The cells would also have to be shown unlikely to cause a serious immune reaction in the recipient.

The concern of the editors of Nature folks is that will open the floodgates for untested cell treatments sold to the public by fast-talking salespeople who are taking advantage of the public’s desire for new treatments.  This is a meaningful concern.  Several sources have pointed out that there are a lot of companies that will try to sell you certain unproven stem cell services, but there are very few such cellular treatments that have been shown to be safe and effective in solid clinical trials.

I appreciate the concern, but Nature may be overdoing it.  It has seemed to me that the FDA’s current regulations on these therapies are strict indeed, limiting work on even non-manipulated cells that have only undergone separation from other cell types, without any doctoring.   Plastic surgeons, for example, want to use adult stem cells from fat as part of breast reconstruction, and I have seen in recent years at least one proposal to do a well-designed clinical trial that could not go forward because the doctors mounting the effort did not have a rich company behind them and could not afford to comply with the relevant regulations. 

It is not out of the question that, were S. 2689 to become law, the FDA could not fashion some sort of middle road for cell therapies that pose limited risk.  That said, parsing things out to protect public safety could be challenging, as could the attendant concern to protect the public from profiteers.  But the issues may not be totally black and white.

 

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