Stem cell therapies could be granted accelerated approval by the US Food and Drug Administration (FDA) as part of a new bill passed by the Senate last week.
President Obama is expected to sign the 21st Century Cures Act into law before he leaves office in January. In its current form it would allow stem cell treatments and other regenerative medicines to be approved based on treating symptoms rather than demonstrating clinical benefit. But critics argue that accelerated approval for stem cell therapies could put patients at risk.
'This is a field where we don’t know a lot about the value of these products, and it would be best that we not create such expedited pathways until we have a better understanding of this field,' Dr Michael Carome, director of the Public Citizen's Health Research Group, told STAT.
One concern is that accelerated approval shortcuts the 'gold standard' of randomised clinical trials, leaving patients vulnerable to receiving a stem cell treatment that might later be found to be unsafe. Another concern is that there is already a profusion of clinics in the US offering unproven stem cell treatments to the public, and that more oversight is needed, not less (see BioNews 869).
The FDA itself added its voice to these concerns in a recent paper published in the New England Journal of Medicine, arguing that there are still many uncertainties in the field of stem cell research: '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,' wrote FDA officials.
Others, including some patient groups, see the new provisions as an important step towards giving patients access to new stem cell therapies that show promise for treating serious or life-threatening conditions. Patients with life-threatening conditions say they don't have time to wait for lengthy approval processes.
'I'm just tired of watching my friends die,' Sammy Jo Wilkinson, a multiple sclerosis patient and co-founder of a patients' rights group Patients for Stem Cells, told Wired. 'We're trying to tell everybody the solution is here now. We just need a logical way to bring this to patients sooner rather than later.'
The House of Representatives passed the bill 344 to 77 earlier this month, and the Senate passed it by 94 to 5 last week. President Obama said: 'The Cures Act makes important investments that will save lives. This bill will make a big difference, and I look forward to signing it as soon as it reaches my desk.'
Key provisions include an additional $4.8 billion over 10 years for the National Institutes of Health to fund key programs, with $1.4 billion earmarked for President Obama's Precision Medicine Initiative, $1.8 billion for Vice President Joe Biden's Cancer Moonshot, and $1.6 billion for the BRAIN Initiative. The bill also provides $500 million to the FDA for streamlining the way it approves drugs and medical devices.