If you’ve lived with type 1 diabetes (T1D) for more than five minutes, you surely have heard the phrase, “a cure is only five years away!” from well-meaning family, friends and even healthcare providers. It’s a common line that has haunted people with T1D for decades; one filled all at once with hope and heartbreak.
But recently, there’s been a lot of online chatter about curative treatments for T1D, and stories surfacing from people who have actually been functionally cured of T1D in clinical trials.
It’s hard not to let the cynical side of our brains take over when we’re still here, counting carbohydrates and chasing high blood sugar levels day in and day out.
But something is different this time around.
For the first time in well, ever, the “a cure is five years away” feels less cliché and more like a question worth seriously asking.
So, how close are we to a T1D cure, like, for real?
Let’s define what we mean by “cure”
Some level-setting: there is still no definitive cure for type 1 diabetes as we know it today. We all wish there were some magical pill you could simply take one time that would instantly manage your high and low blood sugars for all time. That simply doesn’t exist—yet.
Researchers, advocates and people with T1D are increasingly aligned around what’s called a “functional cure.”
Functional cures are often discussed in chronic disease circles as a cure that doesn’t completely eliminate the underlying condition from someone’s body, but provides long-term protection from immune attack (like in the case of diabetes), essentially allowing someone to be functionally diabetes-free.
That protection from immune attack can take different forms, such as immunosuppressant drugs, encapsulated stem cells or other targeted gene therapies.
Someone with a functional cure for diabetes would produce their own insulin again without daily blood sugar management, they would be protected from high and low blood sugar levels and they wouldn’t face long-term diabetes complications.
Right now in research, it’s the ultimate North Star.
The big three: three paths to a functional cure
While many trials are going on around the globe in research labs and on mice, there are three big clinical trials going on with actual people being functionally cured of T1D.
Eledon and the University of Chicago
Eledon Pharmaceuticals teamed up with the University of Chicago on a clinical trial that has seen amazing results in patients through pancreatic islet cell transplantation.
The transplantation is a minimally invasive procedure where pancreatic islets containing insulin-producing beta cells are isolated from the pancreas of an organ donor. Then using a small catheter, they’re infused into the patient’s liver.
The islet cells lodge in small blood vessels in the liver and release insulin. After the procedure, you remain on immunosuppression therapy, called tegoprubart, that reduces the risk of rejection of an organ after a transplant.
Unlike other immunosuppressants, however, tegoprubart is targeted and doesn’t affect your overall immune system. This could be revolutionary for people with T1D.
So far, 100% of patients in this small clinical trial are insulin-independent after four weeks, and with no signs of rejecting the transplant.
The only problem with this is that the treatment requires donor islet cells along with ongoing tegoprubart.
A few more drawbacks of this trial include the small sample size and its short-term results (so far). Studies find their strength (and clinical trials then succeed) when results are repeatable in a larger population and show lasting clinical change–two things that can take a lot of time.
Vertex Pharmaceuticals
Vertex Pharmaceuticals, located in Boston, Massachusetts, has had early success with its VX-880 clinical trial.
What Vertex is doing is interesting: they’re infusing stem cell-derived beta cells into the liver.
These are lab-grown insulin-producing beta cells made from stem cells, so there is no donor shortage to worry about.
The trial participants have had great results. Those who received zimislecel, a stem cell-derived islet-cell therapy, in a single infusion experienced:
- A1C levels of less than 7%
- Time in Range greater than 70%
- Had a reduction in injected insulin needs by 92%
- 83% of participants required no injected insulin at all by month 12
The only hesitation with this therapy is that it requires lifelong immunosuppression, similar to an organ transplant.
Sana Biotechnology
Sana Biotechnology is looking at the cure a bit differently by engineering gene-edited, hypoimmune stem-cell islets that are invisible to the immune system to avoid attack.
Sana’s goal is to have no islet transplant rejection and also no need for immunosuppressive therapy.
While this study is still early, the single patient is still insulin-independent and showing no signs of islet cell rejection at month 14. Sana is now moving towards broader Phase 1 trials to recruit more participants. In fact, Sana recently announced a partnership with the Mayo Clinic to do just that: scale their trial to reach many, many more people with type 1 diabetes.
The Sana trials are still very early-stage, but with the biggest potential at a true cure with no immunosuppression and no further treatment. We’ll have to wait and see what the long-term safety and efficacy of the procedure look like.
So what does all this mean?
It means, generally speaking, that we are no longer waiting for a single cure.
We are watching multiple companies and research institutions tackle the problem of T1D from many angles: donor islets, stem cell-derived islets, stem cell-derived islets plus an encapsulating device and gene-edited stem cells.
There are no conspiracy theories here. These are real scientists, real researchers and real career-physicians who want to race to a T1D cure as quickly as possible.
Clinical trials must go through three phases that can take anywhere from a few years up to 10 or more years. After Phase 3, a drug’s details and clinical trial results are submitted to regulatory authorities (the FDA in the U.S.) for approval to release the drug to the public.
There can be expedited reviews, called priority review designation, for an expedited approval timeline of six months instead of the standard 10 months. Expedited reviews are saved for the treatment, diagnosis, and prevention of serious conditions, including type 1 diabetes.
All told, we can very seriously be looking at a functional cure for T1D within less than 7 to 10 years.
Scientists have proven that they can solve the “make insulin again” puzzle, and they’re actively working on making sure that these results can stand the test of time—with or without immunosuppression.
Now the next challenge is how to make these therapies replicable, sustainable, safe, and eventually affordable and accessible to everyone.
The question is no longer “will” there ever be a cure for diabetes, but “how soon?”
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