What is Regenerative Cell Therapy?

Regenerative medicine and cell therapies focus on repairing, replacing, and restoring function to tissues or organs that have been damaged due to disease, trauma, or congenital issues. Many different types of cells are being explored for use in these therapies, including mesenchymal stromal cells (MSCs, which can be derived from bone marrow, cord blood or cord tissue, adipose tissue, placenta or other sources), hematopoietic stem cells, skeletal muscle stem cells, lymphocytes, induced pluripotent stem cells, and many others.

There are thousands of clinical trials underway to explore the use of RMCTs for many conditions, including cancer, cardiovascular disease, diabetes, degenerative joint conditions, neurological conditions, wound healing, and most recently, COVID-19.

What Actions are Needed?

The 21st Century Cures Act contained several provisions to make safe and effective regenerative medicine and cell therapies available to patients, including extending Food and Drug Administration (FDA) expedited programs to include regenerative therapies and providing initial 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 considerably more federal funding for clinical trials, advancing collaborative evidence development efforts including a national outcomes database, improving and sustaining regulatory innovation, supporting scale-up of manufacturing, advancing payment policy, and building a strong workforce.