FDA: “Stem cell-cased cures must be supported by sound science”

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In September, the Food and Drug Administration held a public hearing on the NIH campus, wherein stakeholders – researchers, the general public and investors – were called upon to voice their opinions on the current status of stem cell-based therapies in the country.

Last week, the FDA followed this up with an opinion statement, via a paper in the New England Journal of Medicine, highlighting the FDA’s position of these therapies.

In the paper, FDA commissioner Robert Califf, alongside the Center for Biologics Evaluation and Research (CBER) Director Peter Marks and CBER Deputy Director Celia Witten urged caution. In doing so, the FDA warned against “unproven” treatments as well as those with “unsufficient data.”

The FDA claimed,

“To ensure that this emerging field fulfills its promise to patients, we must first understand its risks and benefits and develop therapeutic approaches based on sound science”

before adding,

“Often, these cells (whether derived from autologous or allogeneic sources) are being used in practice on the basis of minimal clinical evidence of safety or efficacy, sometimes with the claim that they constitute revolutionary treatments for various conditions.”

They go as far as calling the lack of data “worrisome,” by adding:

“Published data derived primarily from small, uncontrolled trials plus a few well-controlled, randomized trials have not reliably demonstrated the effectiveness of stem-cell treatments even in some of the most systematically studied conditions, such as heart failure and graft-versus-host disease”

The FDA calls both autologous and allogeneic treatments concerning, albeit for different reasons.

These claims will come as a disappointment to proponents of stem cell-based therapies, particularly patients who, absence other options, were hopeful that these cures would provide the benefit other medications were not able to.

The entire paper, titled “Clarifying stem-cell therapy’s benefits and risks,” can be read here.

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