Over the years many of us have wished that there was a list of FDA approved stem cell therapies. Patients and fellow scientists often asked me, but there was no list that I could find. Other researchers mentioned being asked too.
As a result, the general answer was, “the FDA has approved umbilical cord blood-based therapies for cancer or immune disorders”. And we’d continue, “but bone marrow transplantation, while not formally approved, is operationally an approved therapy.”
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.
FDA-approved regenerative therapies: the diseases
What about today in 2021? Before we get to the approved products, here is a short summary list of the diseases that can be treated with these approved therapies.
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 there’s approval to treat that kind of condition with fat stem cells, bone marrow cells, umbilical cord cells or exosomes. It isn’t.
- 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
So what are the actual approved products today?
FDA Approved Stem Cell Therapies, some 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 not obviously non-compliant with FDA rules. There’s a big difference between approved and basically tolerated. Some are barely tolerated as they are not subject to FDA action because the agency doesn’t have the resources and they don’t appear to pose a huge risk. Most of the clinics sell both unapproved stem cell “therapies” and scientifically unproven offerings.
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. A wonderful group called the Alliance for Regenerative Medicine has what we might call an international list of cleared cell and gene therapies here.
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.
List of FDA approved stem cell therapies
The formal FDA list is called, appropriately enough, “Approved Cellular and Gene Therapy Products.”
The current list is up to date as of February 5, 2021. 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.
Here is the list, which I’ve annotated to reflect the relevant conditions that the cells are meant to treat.
- 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-derived 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. Similar to ALLOCORD.
- HPC, Cord Blood, Clinimmune Labs, University of Colorado Cord Blood Bank. Similar to ALLOCORD.
- HPC, Cord Blood – MD Anderson Cord Blood Bank, MD Anderson Cord Blood Bank. Similar to ALLOCORD.
- HPC, Cord Blood – LifeSouth, LifeSouth Community Blood Centers, Inc. Similar to ALLOCORD.
- HPC, Cord Blood – Bloodworks, Bloodworks. Similar to ALLOCORD.
- 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 moderate to severe nasolabial fold wrinkles. In English, these are the deep wrinkles some of us get as we age to the sides of our noses.
- LUXTURNA, Spark Therapeutics. This is 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 so hopefully more will come onto the market.
- TECARTUS (brexucabtagene autoleucel), Kite Pharma. A CAR-T therapy for treatment of adult patients with relapsed/refractory mantle cell lymphoma (r/r MCL).
- 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 more widely called 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 above about this exciting development.
- Consumer Alert on Regenerative Medicine Products Including Stem Cells and Exosomes, July 22, 2020, FDA advisory
- FDA, list of “Approved Cellular and Gene Therapy Products.” Current as of February 2021, per the FDA.