List of FDA Approved Stem Cell Therapies & Drugs for 2024

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, I made a comprehensive list, which is the focus of today’s post. I’ve updated the list now in 2024 with the great news that Casgevy and Lyfgenia, gene therapies for sickle cell, are now approved as of late 2023.

What is in this article?

FDA and cell therapies | List of diseases treated by stem cells | Key Context | Cell & Gene Therapies | List of FDA approved therapies 2023 | FDA approved stem cell clinics? | References

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.

  • Melanoma
  • 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?

fda approved stem cell therapies
FDA has regularly warned about unproven stem cells

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.

fda approval graphic 1
Infographic of FDA clinical trial approval process. Image credit Mina Kim.

List of FDA approved cell and gene therapies as of late 2023

List of FDA approved stem cell clinics

What about list of FDA approved stem cell clinics?

Such a list doesn’t exist.

Why?

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.

References

  1. Consumer Alert on Regenerative Medicine Products Including Stem Cells and Exosomes, July 22, 2022, FDA advisory
  2. FDA, list of “Approved Cellular and Gene Therapy Products.” Current as of November 2023.

34 thoughts on “List of FDA Approved Stem Cell Therapies & Drugs for 2024”

  1. John J Pellegrino

    Subscribing doesn’t work, I think someone caused that problem on purpose. So, I put your channel in my Start menu. I am not a doctor, but I follow important subjects like stem cells. The future possibility of curing many human ailments will interfere with Big Pharma’s billions, and in my opinion, a stem cell “industry” will have to contend with very slow acceptance of anything to do with stem cell therapies. So again, I think everyone should learn as much as they can about healthcare so we can be informed patients. I have already lived many decades and I realize I have also gained a bit of cynicism. I do like your channel, thanks for what you are doing to inform us.

  2. Professor Paul
    So what does one do if severe OA already in both shoulders. I am
    An athlete being told both shoulders need replacing. Sat down with stem cell clinic for consult. They want 12 k to treat both shoulders, using my own hematopoietic stem cell from my bone marrow.
    A. Such huge sum $
    B. Read such controversy
    C. Not the best stem cell but accepted in U.S.
    sure appreciate you opinion.

  3. as different capillary’s of blood cancer science is stacking up behind the scenes.
    is the fda ready for approval to allow the science “stem cells b/cancer ” to advance
    t/wards clinical trials, has the fda enrolled mentors to monitor the science.
    thank you..paul….enjoyed the article of great interest atb….michael

  4. Stephane Vandenabeele

    Thanks for this list and bog I discover today.

    Regarding stem cell-based treatment and diseases: what about Multiple Sclerosis?

    To my surprise I recently read a description of HSC (most likely CD34+) transplantation for progressive Multiple Sclerosis. Thanks for any opinion.

  5. Dr. Paul K,
    This web site Bioinformant.com has posted a somewhat biased article “What Stem Cell Clinics Do You Trust?”. I think it is promoting select clinics, and seems to exclude umbilical cord MSCs in their evaluation. Their comment section is structured the same as The Niche.

    I went Googling “Stem Cell Clinics” and seem to observe an exponential growth in the number of clinics advertising on the web, as well as offering Wharton’s jelly umbilical cord derived MSCs. The price for treatment seems to be decreasing as well.

  6. I damaged my cornea five years ago. I am not a cornea transplant candidate. I have many dead stem cells
    Is there any procedure for this?
    My vision in that eye is nearly gone.
    Thank you

  7. Has the FDA approved or is looking in to stem cells treatment for NMO patients? And if they haven’t. Then why not?? Cause there is no specific medication for this frustrating disease. It’s only some sort of chemo.

  8. Is there a stem cell therapy for seronegative rheumatoid arthritis? And if so, where would a person go to inquire about it?

  9. Is there a term for “fda compliant” for section 351 drug. I noticed this term is being used for section 361.

  10. Nicholas Antonelli

    Is there a stem cell treatment that would be applicable to treating paralysis as a result of temporary interruption of blood flow to the muscle of the eye causing double vision in peripheral vision?

    1. Yes, a lot of research. There is hope related to stem cells but only in very specific ways. General shots of stem cells likely won’t help MS. The research combining chemo and stem cells provides real hope but is also very risky and you should talk to your doctor before considering an intervention or enrolling in a trial. This approach is only relevant for certain kinds of MS too.

  11. That is a great question…how was bone marrow transplant approved? I mean what regulatory mechanism was used?

    I know that the FDA had to approve islet transplants, right? with a BLA for Camillo??

    Anyways, thanks for posting interesting things that sometimes we dont think about

    Thomas Ichim

  12. Is umbilical cord (not blood) stem cell therapy approved or recommended? I have failed back surgery (fusion at L3 to S2) and I have National Wellness Centers telling me they can help with the pain.

  13. Regarding the rapid development of unauthorized stem cell clinics, does the FDA acquiesce in their development? Do you think these clinics will exist for a long time?

  14. The FDA continues to cite a short statement referring to Hematopoietic Stem Cells from cord blood. Bone marrow transplant, functionally the same HSCs, are generally considered a transplant (like an organ). Here is the wording the FDA talks about “Stem cells” other than the new list you’ve recently linked:

    “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.”

  15. Is there any information related to stem cell and cerebral palsy. One family I know went to thiland for some helpful care?

    1. Is there a stel cell to be taken orally, they say they acquire the placenta from the deer? If there is one let me know..

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