In the past many of us have wished there was a list of FDA-approved stem cell therapies. Patients and fellow scientists often asked me, but I couldn’t find a list.
As a result, the generic answer was, “the FDA has approved umbilical cord blood therapies for cancer or immune disorders”. Also, “bone marrow transplantation, while not formally approved, is essentially an approved therapy.”
Today we have more information and the goal of this post is to fill you in on where things stand.
What is in this article?
FDA and cell therapies
Other than what I mentioned above, there was nothing else approved that was an actual stem cell therapy. There were, however, cellular therapies approved even if not related to stem cells. In a way these fit into the broader category of regenerative medicine.
Note that you can watch me discuss this post in a new YouTube Video below.
List of diseases treated by stem cells & FDA approved
What about today in 2022? Before we get to the approved products, I made a short list of the diseases treated by stem cells.
Note that only very specific products matched with specific forms of these diseases have the FDA OK. For instance just because damaged cartilage is on the list doesn’t mean anything goes. There’s no approval to treat it with fat stem cells, bone marrow, umbilical cord cells or exosomes.
- Mantle Cell Lymphoma
- Large B-Cell Lymphoma
- B-Cell ALL
- Prostate Cancer
- Some other kinds of cancer
- Facial wrinkles (specific type)
- Receding gums
- Damaged cartilage
- Leber congenital amaurosis (a retinal disorder)
- SMA or spinal muscular atrophy
The list of diseases has been growing. What about the products?
FDA Approved Stem Cell Therapies, key context
We need to turn to the FDA itself for the clearest answer on where things stand now.
Too often stem cell clinics claim that what they offer is “FDA approved”. In actuality at best what some of them sell is not FDA approved, but rather technically compliant with the rules for 361 products. What this means in English is that the products are not regulated as drugs.
So what does the FDA have to say about what they have approved?
In mid-2020, they issued an advisory, “Consumer Alert on Regenerative Medicine Products Including Stem Cells and Exosomes.” In it they wrote:
“Stem cell products are regulated by FDA, and, generally, all stem cell products require FDA approval. Currently, the only stem cell products that are FDA-approved for use in the United States consist of blood-forming stem cells (also known as hematopoietic progenitor cells) that are derived from umbilical cord blood. These products are approved for use in patients with disorders that affect the production of blood (i.e., the “hematopoietic” system) but they are not approved for other uses.”
This fits with the general boilerplate some of us in the stem cell field have used, as noted earlier.
Note that, of course, the US and the FDA are not the only ones in this arena. The Alliance for Regenerative Medicine has what we might call an international list of cleared cell and gene therapies here. They are a great organization.
Cell and gene therapies
The good news today is that the agency has an actual list that is publicly available. Even better news is that there are more approved cell therapies than last time I tried to tackle this topic.
An interesting side note is that the agency for several years now has been combining together cell and gene therapies into one category. This is also reflected in their Regenerative Medicine Advanced Therapy (RMAT) designation program, which contains both types. RMATs still have to go through the trial approval process but get special consideration. You can see a help infographic below that my intern Mina made of the clinical trial approval process.
The formal FDA list of approved drugs made from stem cells is called, appropriately enough, “Approved Cellular and Gene Therapy Products.”
The current list is up to date as of September 1, 2022. I’m going to update this post as the agency updates their list. It’s interesting to speculate on how different this list might be in as short as 5 years given the exciting clinical trials that are ongoing now. In 10 years I predict it’s going to be a much longer list.
List of FDA approved stem cell therapies in 2022
- ALLOCORD (HPC, Cord Blood), SSM Cardinal Glennon Children’s Medical Center. “For use in unrelated donor hematopoietic progenitor cell transplantation procedures in conjunction with an appropriate preparative regimen for hematopoietic and immunologic reconstitution in patients with disorders affecting the hematopoietic system that are inherited, acquired, or result from myeloablative treatment.” This is a cord blood product that can be used in a similar fashion to bone marrow or hematopoietic stem cell transplants.
- BREYANZI. Juno Therapeutics, Inc., a Bristol-Myers Squibb. This is a CAR-T-cell therapy for adults with relapsed or refractory large B-cell lymphoma that has failed to respond to at least 2 other therapies.
- CLEVECORD (HPC Cord Blood), Cleveland Cord Blood Center. Similar to ALLOCORD.
- Ducord, HPC Cord Blood, Duke Med School, Similar to ALLOCORD.
- GINTUIT (Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen), Organogenesis. The FDA says as to uses, “Is an allogeneic cellularized scaffold product indicated for topical (non-submerged) application to a surgically created vascular wound bed in the treatment of mucogingival conditions in adults.” This is hard to understand though. In a nutshell this product treats receding gums, which can be a serious problem. Traditional approaches are painful and don’t always work so this product fills a nice niche. Note that it is an acellular scaffold.
- HEMACORD (HPC, cord blood), New York Blood Center. This and the four HPCs that follow are similar to ALLOCORD.
- HPC, Cord Blood, Clinimmune Labs, University of Colorado Cord Blood Bank.
- HPC, Cord Blood – MD Anderson, MD Anderson.
- HPC, Cord Blood – LifeSouth, LifeSouth Community Blood Centers, Inc.
- HPC, Cord Blood – Bloodworks, Bloodworks.
- IMLYGIC (talimogene laherparepvec), BioVex, subsidiary of Amgen. An oncolytic gene therapy product for treatment of melanoma.
- KYMRIAH (tisagenlecleucel), Novartis. CAR-T cell therapy for B-cell acute lymphoblastic leukemia (B-Cell ALL).
- LAVIV (Azficel-T), Fibrocall Technologies. It is used for treatment of the deep wrinkles some of us get as we age to the sides of our noses.
- LUXTURNA, Spark Therapeutics. A gene therapy for the treatment of Leber congenital amaurosis, an eye disorder that affects the retina and causes vision loss from a young age.
- MACI (Autologous Cultured Chondrocytes on a Porcine Collagen Membrane), Vericel. An autologous therapy (the product uses a processed form of your own cartilage cells) for treatment of damaged cartilage.
- PROVENGE (sipuleucel-T), Dendreon Corp. An immunotherapy against prostate cancer. The FDA calls it, “an autologous cellular immunotherapy designed to stimulate a patient’s own immune system against cancer.” As a prostate cancer survivor myself it really hits home how few options there can be for advanced cases.
- TECARTUS (brexucabtagene autoleucel), Kite Pharma. A CAR-T therapy for treatment of adult patients with relapsed/refractory mantle cell lymphoma.
- YESCARTA (axicabtagene ciloleucel), Kite Pharma. CAR-T for large B-cell lymphoma that hasn’t responded to other therapy.
- ZOLGENSMA (onasemnogene abeparvovec-xioi), AveXis. Also known as Nusinersen, this is drug is related to Spinraza. It was the first therapy approved for SMA or spinal muscular atrophy. As an antisense oligonucleotide therapy, it is a novel approach. You can see a video from the MDA about this above.
List of FDA approved stem cell clinics
What about list of FDA approved stem cell clinics?
Such a list doesn’t exist.
The reason is because no clinics have FDA approval. While some clinics sell stem cells that don’t need formal FDA drug approval, most need that approval and yet don’t have it.
- Consumer Alert on Regenerative Medicine Products Including Stem Cells and Exosomes, July 22, 2022, FDA advisory
- FDA, list of “Approved Cellular and Gene Therapy Products.” Current as of August 2022.