Sana Biotechnology’s hypoimmune (HIP) donor-derived islets are genetically engineered to avoid immune destruction without the need for immunosuppressants.
Earlier this year, we reported on an update from the first person with type 1 diabetes (T1D) to receive a transplant of these cells, which showed that they were making insulin—without immunosuppression—after four weeks. Then, at the American Diabetes Association (ADA) 85th Scientific Sessions, we heard firsthand from Per-Ola Carlsson, M.D., Ph.D., the Principal Investigator at Uppsala University conducting the study, who provided more exciting data to a packed audience.
Now, these data have just been published in the prestigious New England Journal of Medicine in an article titled “Survival of Transplanted Allogenic Beta Cells with No Immunosuppression.” This study was supported by the Helmsley Charitable Trust, a long-time partner of Breakthrough T1D.
Read on to learn more.
Surviving and thriving
In this first-in-human study, the recipient of Sana’s HIP islets had no detectable insulin production at the time of the transplant (as measured by C-peptide). They received a small dose of islet cells—less than the eventual therapeutic dose—implanted into the forearm to evaluate safety.
C-peptide
C-peptide is an easily measurable biomarker of insulin production and islet function.
After 12 weeks, researchers found that the transplanted islets were successfully avoiding destruction by the immune system—meaning they are surviving AND making insulin!
The recipient had decreased HbA1c levels and stable insulin production between seven days and 12 weeks post-transplant, although, as expected with the small dose of cells used in this pilot study, they still required external insulin therapy. They experienced some non-serious side effects possibly related to the surgical transplant procedure, but not the islet cells themselves. Overall, the recipient is doing well and in good health—an exciting step for the T1D community.
Why this matters
To put it plainly: this is a huge step toward cures for T1D.
Immunosuppression is a major barrier to cell therapies because of the side effects associated with them. This is the first proof-of-concept evidence that we can genetically engineer islets to avoid immune destruction—eliminating the need for chronic, broad immunosuppressants. This means that more people may have the opportunity to benefit.
Importantly, the person in this study received only a fraction of the islet cells that would eventually be used therapeutically. Researchers are just beginning to understand the full potential of engineered HIP islets. Because more islet cells means more insulin production, there’s the possibility of insulin therapy independence if higher doses of HIP islets are implanted—something that will be addressed in future studies.
Thanks to this study, we’ve gained more evidence that forearm muscles may be a possible minimally invasive site for islet transplantation. This transplant site also provides researchers with the advantage of visualizing the islets—and making sure they’re alive—using magnetic resonance imaging (MRI), which isn’t possible with the current transplant site in the hepatic portal vein.
Excitingly, Sana is working to apply this technology to manufactured islets, opening the doors to producing HIP islets at large scale. They expect to begin phase 1 clinical trials for this next-generation HIP islet therapy as early as 2026.
Cell therapies without immunosuppression are a Breakthrough T1D priority
Cell replacement therapies—especially those that do not require immunosuppression—are a priority of Breakthrough T1D’s Cell Therapies Program and the goal of our Project ACT (Accelerate Cell Therapies) initiative.
Project ACT
Scientific progress takes time, resources, collaborations, and effort. To accelerate islet replacement therapies faster than ever, Breakthrough T1D launched Project ACT (Accelerate Cell Therapies) to simultaneously advance research, development, regulatory policies, access, and adoption of manufactured islet therapies that do not require broad immunosuppression.
To accelerate the development of cell therapies that do not require immunosuppression, The T1D Fund: A Breakthrough T1D Venture invested in Sana to help advance their HIP technology platform. Breakthrough T1D continues to support creative and promising approaches that may eliminate the need for immunosuppression with cell replacement therapies, and we are hopeful and excited about what the future may hold.
This wouldn’t have been possible without supporters like you. Thank you for all that you do to drive Breakthrough T1D’s mission, and together we celebrate each step towards a world without T1D.
