IMAGE BY CAROL N. IBE AND EUGENE O. MAJOR, COURTESY OF NIHA woman in California recently made headlines when she grew superfluous bones in her eye socket after receiving a botched cosmetic procedure that involved injecting fat stem cells from her abdomen into her face. Hers is just one of the horror stories that have resulted from the use of unregulated, unapproved stem cell therapies in the U.S.
In July 2012, after several companies were found to be treating patients with unapproved stem cell therapies, the U.S. District Court in Washington affirmed the right of the FDA to regulate therapies made from a patient’s own stem cells. This was a good decision that will protect patients, strengthen efforts to develop new regenerative medicines and help to ensure the safety and therapeutic value of approved products.
A rising call to introduce appropriate oversight and regulation of stem cell therapy comes as no real surprise—the number of authorized cell therapy clinical trials has increased steadily in the U.S. over the past 10 years, from 12 trials initiated in 2001 to more than 50 trials initiated in 2011. While certain aspects of the US regulatory process are considered burdensome, it is imperative that stem cell therapies be tested rigorously and to the highest standards. The fact that such therapies are derived from cells found in the body does not diminish this responsibility.
As we have seen, clinical trials often identify unexpected and unanticipated safety issues. Rigorous clinical trials can also show that some investigational products are not as efficacious as they need to be to balance their risks. For example, only one in four biologic treatments have ultimately proven to be both safe and effective, according to a BioMedtracker study. Marketing any therapy, including those for serious health problems, that has not been proven to be both safe and effective in rigorous clinical trials puts patients at risk and ultimately hurts all research organizations working to develop new therapies. Within this framework, we also need a pathway for the US Food and Drug Administration to rapidly respond to post-marketing findings related to approved products. In this way, the FDA can remain positioned to balance the benefits of bringing new therapies to market quickly against emerging risks and have in place a plan to take post-approval action when necessary.
Some recent changes in regulatory guidelines have included welcome and much needed reforms. For example, the Federal Drug and Safety Innovation Act (FDASIA), which President Obama signed in July 2012 to speed up drug development, provides important support in two areas for companies, such as Aastrom, that are working to develop regenerative therapies. First, FDASIA has granted more therapies “Accelerated Approval” status, which reduces the number of regulatory steps necessary before a therapy can be submitted to the FDA. Second, it also created a “Breakthrough Therapies” category to expedite the review of innovative therapies intended for serious medical conditions. A streamlined approval process can help to reduce the cost and complexity of clinical trials, and will potentially make it possible to advance promising new therapies more efficiently in years to come. With these important changes in place, we should continue to take steps to clarify and expedite the regulatory process for regenerative medicines and introduce refinements that take into account the unique and complex production requirements and molecular mechanics of these therapies.
To bring the best thinking to this process, we should also position clinical investigators to work more closely with industry and regulators to streamline regulatory procedures. The insights and recommendations of clinicians are key to the success of many clinical research programs in regenerative medicine, and we should encourage and expand their participation in this process.
Currently, Aastrom’s cell therapy ixmyelocel-T is being evaluated in a Phase 3 clinical trial for critical limb ischemia and in a Phase 2 clinical trial for ischemic dilated cardiomyopathy. These trials reflect the approach to developing regenerative medicines that balance both public health and business considerations. We are working hard to maintain the highest standards for our clinical trials, engage the most knowledgeable and experienced investigators, and give our cell therapy product the best chance of clinical, regulatory, and commercial success. We are also committed to presenting and publishing our data and other research findings in peer-reviewed settings for researchers, clinicians, patients and others involved in developing and using new treatments for patients with serious cardiovascular diseases.
There is already substantial evidence to indicate that regenerative medicine has the potential to improve medical outcomes, enhance quality of life, and reduce overall healthcare costs for many patients. But none of these goals can be achieved without first ensuring that cell therapies are both safe and effective for patients. A rigorous clinical trial system and regulatory oversight are key to protecting patients, improving care, and bringing much-needed therapies to patients and the physicians that care for them.
Dan Orlando is interim CEO of Aastrom Biosciences, a company that develops stem cell treatments for critical cardiovascular diseases.