Current Challenges for Assessing Regenerative Medicines
The 21st Century Cures Act includes provisions to accelerate development of innovative treatments. One treatment mentioned in the Act involves using living cells reprogrammed to augment a patient’s immune response to prevent, reduce or even eradicate chronic diseases such as cancer. Referred to as “regenerative medicine advanced therapies” (RMATs) by the U.S. FDA, these living drugs have demonstrated remarkable clinical results so far, and carry the potential to revolutionize medicine (1,2). RMAT development is complicated, however, due to the subtleties of engineering, manufacturing and delivering live cells to patients, which translates to higher costs. These challenges then serve as a clinical barrier. Therefore, uniform standards and best practice guidelines are needed to speed up development and transform the promise of cell therapies into reality.