Sometimes it feels like the stem cell and broader regenerative medicine field face an unending stream of challenges and difficult news, but there is also stem cell good news out there. I mean upbeat developments in the broader regenerative medicine space. That’s the focus of today’s post as the year wraps up with the top 5 encouraging developments.
Duchenne Muscular Dystrophy (deramiocel)
Capricor reported very encouraging data on Duchenne Muscular Dystrophy. The strong data on deramiocel could lead to FDA approval in 2026. I believe that’s reasonably likely. Craig McDonald, my colleague here at UC Davis, is the lead PI. I’m not involved in this trial, but it’s exciting to see it develop.
Stem cells for Parkinson’s
Three trials reported preliminary but encouraging data on stem cells for Parkinson’s. Having three promising products greatly improves the odds of ultimate success here. There are others working in this space as well.
Stem cells for diabetes (Zimislecel)
Vertex’s stem cell diabetes work continues to look exciting with some trial participants being able to stop insulin. It’s still early days, but I’m very hopeful about this work.
Stem cells for vision loss
Ten to fifteen years ago I thought that stem cells for vision loss might be one of the first new generations of FDA-approved (or similarly approved in other countries) stem cell therapies. I was hopeful about pluripotent stem cell-based approaches for macular degeneration. Things slowed on that front due to numerous challenges. I still believe we’re going to ultimately see a stem cell-based therapy for macular degeneration but it’s turning into a marathon.
There’s concern that RPE transplants may not be sufficient for most AMD patients where so many photoreceptors are already gone. The idea of photoreceptor transplants (e.g., made from iPS cells in the lab) is exciting, but very technically challenging.
Still, 2025 brought some good news in the vision space including from a Mass Brigham trial that was quite encouraging on stem cells for vision loss due to corneal damage. The CALEC approach also is promising for eye injury.
Stem cell therapy for SCID
Don Kohn’s work at UCLA continues to cure kids with SCID. The 95% success rate with no serious complications is so impressive.
We’re going to see more combination cell-gene therapy successes like this moving forward.
The approved sickle cell approaches are additional examples. A key issue for combination cell-gene therapy approaches is cost, especially scaling for non-rare diseases.
Finally, you can also see a 2024 post I did on stem cell good news that year.
Hi Sam,
I recently had a 400 million IV Autologus stem cell infusion,done in Japan.
The name of the clinic is Helene, based in tokyo.
Very professional, all work done in in house.
I had the treatment for my damaged lungs (Bornchieaties) from an inhalation injury.
My Ct scans showed good improvement and my symtom severity score dropped.
I can go back and use my own cells for a period of up to 5 years.
Its not a miracle cure but i think with more treatments can certainly improve symtom severity.
Thank you for this amazing info Dr K! I just turned 60. I quit smoking after 40 years, have some ortho issues and have been extremely fatigued for over a year. Have begun exercising, eating more cleanly etc. Im overwhelmed with Stem cell information and clinics outside the US where you can get them. Specifically Im desiring IV infusion of MSCs derived from donated umbilical cords. Have located a clinic in the Caymen Islands thats regulated and sources their cells from the US. Im SO afraid to shell out all that money (25,000K) for possible little or no effects. I know if you’re getting live cells how could you lose right? Im worried about the damage I did to my lungs and living longer for my 9yo twins. May I ask if theres a legit place that you’ve heard of to get them?
@Sam, that’s great on quitting smoking, starting exercising, etc.
Unfortunately, there aren’t any stem cell IV shots that really make sense for trying to heal possible lung damage from smoking, to help fatigue, or for trying to live longer.
Many IV stem cells do appear to end up in the lungs after being infused into the blood, but once they get to the lungs they mostly die or are killed within hours or at most days. They just are not there long enough to do anything positive, especially not in the long run. With all of this mind, $25K is super expensive. Most of the clinics I’ve done research on over the years are designed to maximize profits over all else and that’s part of why there’s this overwhelming amount of supposed stem cell info out there. It’s largely marketing.
I’m not a physician so this isn’t medical advice, but I personally wouldn’t get IV MSCs. There are also going to be risks, some that probably don’t understand yet.
I wish you the best!
Thank you for this blog and all your work over the years. Can’t imagine where this industry would be without Dr. K.
Thanks, EV.
I’m happy you are having successes!
Charyn DuQuette
I really, really hope that the RPE transplants continue to be explored. There are lots of people who have macular degeneration for all kinds of reasons, not just age-related. Plenty of these people don’t have such severe cases and could truly benefit.
Also, I’d be very interested on your thoughts about Dr. Masayo Takahashi using AI and robotic systems for research with iPSC-RPE culture conditions? I will happily put aside all of my negative opinions about AI if that approach helps at all.
What is your opinion on the skin patches being sold as stem cell therapy?
These? https://ipscell.com/2023/02/lifewave-x39-stem-cell-patch-story-has-holes/ I’m skeptical.