American Food and Drug Administration Issues Guidelines for Genetic and Regenerative Medical Therapies

July 15, 2018 – The FDA governs what is considered acceptable medical treatment including new drug therapies for American patients. Many other countries follow the FDA lead. With gene therapy, gene drives, and regenerative medicine moving from laboratories to animal and human clinical trials, this month it issued preliminary guidance on protocols for these novel forms of treatment.

Recently the FDA approved a number of gene therapy products to treat childhood and young adult leukemia, non-Hodgkin lymphoma, and forms of congenital blindness. The guidelines for giving the green light to Novartis, Kite Pharma and Spark Therapeutics, the companies responsible for producing these new genetic treatment protocols are covered in a new document entitled, “Chemistry, Manufacturing, and Control  Information for Human Gene Therapy Investigational New Drug Applications.”

Gene therapies involve the transfer of healthy genetic material into gene sequences to replace bad genes responsible for a particular medical condition. Regenerative medicine involves the therapeutic re-engineering and regrowth of cellular tissue of all types from organs, to bones, to nerves, blood vessels, and the like.

In announcing the new guidelines an FDA spokesperson and health policy expert described how different these new therapies are from traditional drug development and treatment protocols. Whereas the focus in the latter is 80% on clinical issues, when it comes to gene therapies and regenerative medicine the 80% is on manufacturing looking at:

• the description of the manufacturing process,
• process controls,
• cell culture conditions,
• material controls including the identification of the amount of material to be produced for testing,
• cell bank qualification and characterization,
• process validation,
• and finally the end manufacturing process including the quantity of material being produced.

The FDA also has included long-term follow-up guidance for gene therapy and regenerative medicine products. The Agency admits it is still on a learning curve to understand how these new therapies work and what is considered safe in the long term. But in the interest of initial results from laboratory and clinical trials, the goal is to approach these forms of medical treatment with an evolving strategy based on the rapid evolution of the science. So expect to see frequent revisions to FDA treatment protocols as new genetic and regenerative medical therapies are announced by biopharmaceutical companies and academic researchers.