What is Regenerative Cell Therapy?

Regenerative cellular therapy—which involves the replacement or regeneration of human cells or tissues to restore or establish normal function in the human body—represents the next generation of groundbreaking treatments that are showing great promise in cardiology, neurology, oncology, orthopedics, ophthalmology, and other areas. Several well-designed clinical trials are now underway to explore the use of regenerative cell therapies to help patients with Alzheimer’s disease, autism, cancer, cerebral palsy, diabetes, several orthopedic conditions, Parkinson’s disease, urologic disease, and wound healing. At the same time, a handful of clinics have caused patient harm or made questionable claims, taking advantage of vulnerable patients and casting a negative light on this emerging science and industry.

What Actions are Needed?

The 21st Century Cures Act contained several provisions to make safe and effective regenerative cellular therapies available to patients, including extending Food and Drug Administration (FDA) expedited programs to include regenerative therapies and providing funds for research through the National Institutes of Health. FDA is also taking many important enforcement actions to address the small number of clinics that are hurting patients.

Several additional actions are needed to help bring both safe and effective therapies to patients, including supporting continued research, developing and driving the adoption of standards, building capacity and a strong workforce in this emerging field, and establishing a national outcomes database to create a community of high-quality researchers, providers, and manufacturers who agree to share data to increase transparency and accountability and advance the science and the field.

Such a national outcomes database could assist in advancing the science; improving the development, manufacturing, and delivery of treatments; monitoring the safety of products; measuring and improving outcomes for patients; informing decision-making regarding coverage and payment; providing another means of real-world evidence development for regulatory decision-making; and helping to inform patients in their decision-making.