An open letter to everyone who cares about research aimed at type 1 diabetes cures.
I've been blogging on research aimed at curing type 1 diabetes for almost 20 years, but I've never sent out a message like this before. However, for me, the results from a single person clinical trial of Sana Biotechnology's UP421 treatment should motivate a change in how we all think about research to cure T1D.
Until now, I've taken a "diversification of research" point of view. At any time, there are 20 or 30
active research projects aimed at curing T1D, and I supported all of them, based on their results to date. I did not favor one over another based on what I thought would happen in the future. My policy was that we don't know which one will succeed, so we need to support all of them and see which one actually does succeed.
But now, I'm starting to change my point of view. I'm starting to see that one approach is working better; is moving to the front of the competitive pack, and deserves more support and more optimism, than the others.
Sana's UP421 study gave one person a small dose of genetically engineered beta cells. The genetic engineering (using the CRISPR-Cas9 gene editing technology) is designed to render the cells invisible to the immune system in order to avoid the need for any immune suppression, both to prevent foreign organ rejection and to prevent autoimmune attacks. C-peptides were generated and immune suppression was not needed. Six month results were published in the New England Journal of Medicine, and they have released one year results at an investor's conference. Sana is planning on a follow up product, SC451 to start clinical trials in 2026.
Obviously, this result has important limitations: one person, less than one year, a small dose, and a small effect. It used cadaver beta cells and Sana is switching to a different cell source for the future. However, what it did show was invisibility to the immune system, and steady generation of insulin for the first 6 months of data. It did generate less insulin for the next six months, dropping to about half of its initial high point, and that will need to be investigated.
The reason I am excited about this result is that the required scientific breakthrough(s) have already happened. The genetic engineering required to hide the transplanted beta cells appears to be successful in this one person study. Much of the study's results were aimed at confirming this invisibility. The remaining work is much more engineering development than scientific breakthrough. Sana needs to
transplant more cells, they need to be tested for longer, and (especially) in more, different people.
I think that all organizations working to cure type 1 diabetes, should do the following for the next few years, and I think that we, as a community that cares about T1D research should be encouraging them to:
- Establish a focus on genetically engineering beta cells which evade the immune system, as a potential cure for type 1 diabetes.
- Put 5% of their research funds into helping Sana's SC451 product (or successors) progress down the path towards general availability.
- Start advocating with the FDA now to smooth the approval process for this treatment when it is available, and to ensure insurance coverage for this treatment.
- Put an additional 5% (or a little more) into adjacent research, non-Sana projects to use genetic engineering to hide beta cells.
Research is about the unknown, and I'm not saying that Sana is going to cure T1D, but I think they are close enough so that we should focus more on them and their research area (generically engineering beta cells to avoid the immune system). I don't think spending 10% of our research money on this one area is extreme right now.
BreakthroughT1D's Project ACT (Accelerate Cell Therapies), which they started in 2024, is already a step in this direction. It's focus is wider than beta cells genetically engineered to hide from the immune system, but it certainly covers them. In addition, the T1D Fund (BreakthroughT1D's venture capital business fund) is already funding Sana . There is also a Swedish foundation which is focused on funding this research in a university setting. These are all great steps, but the entire T1D community needs to "put a brick on the accelerator" for this.
My thinking here is similar to BreakthroughT1D's (then JDRF's) policy on the Artificial Pancreas. I believe that JDRF's actions there sped up availability of an Artificial Pancreas by several years, and I think we can do the same now, but this time with a cure.
I have often said that the first signal we will get that a cure for type 1 diabetes will occur
is that one person will be cured in a clinical trial. Sana's 2025 result is not quite that, but it is on the path.
None of this means that I'm going to stop following (or even slow down following) other research areas. I continue to believe that limiting ourselves to one one path to a cure is a dangerous form of scientific extremism. So while I am calling on more focus and more support for genetic engineering to hide transplanted beta cells from the body's immune system, I'm not going to exclude other research paths from my blog. I will continue to report on all cure focused research.
Note: There is also another company called Sana which does AI work. This research is being done by Sana Biotechnology.
More Information on Sana Biotechnology's results:
News Coverage: https://www.medscape.com/viewarticle/transplanted-islet-cells-survive-without-immune-suppression-2025a1000kqt
My Blogging: https://cureresearch4type1diabetes.blogspot.com/2025/04/sana-biotechnology-reports-first.html
Publication: https://www.nejm.org/doi/abs/10.1056/NEJMoa2503822
Clinical Trial: https://www.clinicaltrials.gov/study/NCT06239636
Press Release: https://ir.sana.com/news-releases/news-release-details/sana-biotechnology-announces-publication-new-england-journal
Foundation created to support the university side of this research:
About Project ACT:
Joshua Levy
http://cureresearch4type1diabetes.blogspot.com
publicjoshualevy at gmail dot com
All the views expressed here are those of Joshua Levy, and nothing here is official BreakthroughT1D or JDCA news, views, policies or opinions. I sometimes use generative AI ("chatbots") to generate draft blogs, parts of blogs, or drafter alternate wordings for these blogs. I always review every part of every published blog to ensure that it is saying what I want, in the tone that I want, truthfully, and accurately. My kid has type-1 diabetes and has participated in clinical trials, which might be discussed here. I am obese and right on the border of T2D and therefore may be taking drugs for those conditions. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog!
