Available Treatments & Ongoing Research
Regenerative medicine is a rapidly evolving field that focuses on the repair, replacement, or regeneration of cells, tissues, or organs to restore or establish normal function. The FDA regulates regenerative medicine products under its Center for Biologics Evaluation and Research (CBER). Here are some FDA-compliant regenerative medicine options available in the USA:
- Stem Cell Therapy
- Hematopoietic Stem Cell Transplantation (HSCT): This is a therapy used for the treatment of certain cancers, such as leukemia and lymphoma, and some blood disorders. It involves the transplantation of blood-forming stem cells.
- Mesenchymal Stem Cells (MSC’s): There are ongoing clinical trials and the FDA has approved the use of MSC’s and other cell therapies for particular blood cancers. We will update this information shortly as new results are announced regularly.
- Tissue-Engineered Products
- MACI (Matrix-Induced Autologous Chondrocyte Implantation): This product is used for the repair of cartilage defects in the knee. It involves using a patient’s own cultured chondrocytes (cartilage cells) which are expanded and then re-implanted into the knee.
- Apligraf and Dermagraft: These are bio-engineered skin substitutes used for the treatment of diabetic foot ulcers and venous leg ulcers. They are composed of living cells and are applied to the wound to promote healing.
- Platelet-Rich Plasma (PRP) Therapy
- PRP therapy involves using a concentration of a patient’s own platelets to accelerate the healing of injured tendons, ligaments, muscles, and joints. While PRP is not specifically approved by the FDA for any particular indication, its use in clinical settings must comply with FDA regulations regarding human cells, tissues, and cellular and tissue-based products (HCT/Ps).
- Landitra
- The FDA has approved Lantidra (donislecel) for the treatment of type 1 diabetes. Lantidra is an allogeneic pancreatic islet cellular therapy, which means it involves the transplantation of islet cells from a donor pancreas into a person with type 1 diabetes. These islet cells are intended to produce insulin, potentially reducing the need for insulin injections.
Lantidra is specifically approved for patients with type 1 diabetes who are unable to approach target glycated hemoglobin (HbA1c) levels because of severe hypoglycemia, despite intensive diabetes management and education. This therapy provides an alternative for those who have not achieved desired glycemic control through conventional treatments.
This approval represents a significant advancement in the treatment options available for individuals with type 1 diabetes, offering hope for improved management of the condition.
Regulations and Compliance
- 21 CFR Part 1271: This regulation outlines the requirements for human cells, tissues, and cellular and tissue-based products (HCT/Ps). Products that fall under these regulations must meet criteria for minimal manipulation and homologous use to be exempt from premarket approval.
- FDA’s Regenerative Medicine Advanced Therapy (RMAT) Designation: This designation is granted to regenerative medicine products that show potential for addressing unmet medical needs for serious or life-threatening conditions. It provides benefits such as increased interaction with the FDA and eligibility for priority review.
The field of regenerative medicine is continuously evolving, with new therapies and products undergoing clinical trials and regulatory review. Patients and healthcare providers should stay informed about the latest advancements and regulatory updates. Please watch this space as we continue to add scientific evidence.
References:
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10536665/
https://www.fda.gov/news-events/press-announcements/fda-approves-first-autologous-cellularized-scaffold-repair-cartilage-defects-knee
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC7589810/
https://www.nature.com/articles/d41573-023-00113-w