When it comes to finding a cure for type 1 diabetes, it’s actually been achieved dozens of times — in mice. Curing type 1 diabetes in the more complex system of a human being is a different challenge.
Many of the enthusiastic reports you might see in your newsfeed each year are largely successful “cures” in mice that have yet to be trialed on humans. Most, unfortunately, fail in human trials.
In this article, we’ll look at the top contenders in the research to find a cure for type 1 diabetes, the clinical trials process any potential cure must go through, and the latest cure research that is working in mice.
What would a diabetes cure need to accomplish?
While we often joke about needing a “new pancreas” as people with type 1 diabetes, what’s really failing within our body is our immune system.
In people with type 1 diabetes, your immune system is constantly attacking the beta cells produced by your pancreas that are essential for producing insulin. While your diagnosis may have been years or decades ago, your body still tries every single day to produce insulin.
Unfortunately, your immune system is still attacking and destroying those cells every single day, too.
For a treatment method to fully cure a person with type 1 diabetes, it would need to do at least one of the following:
- Tell our immune system to stop attacking those beta cells
- Provide an alternative source of beta cells that are protected from the immune system
Easier said than done. Let’s take a look at the top (and perhaps only) contender for curing type 1 diabetes in today’s current research.
The main contender for a type 1 diabetes cure
While you may see a variety of news articles each year with researchers claiming they’ve found a successful cure for type 1 diabetes, the majority fail in animal trials and are never heard from again because they don’t pass the early stages of long-term efficacy and safety.
“The majority of it is really lined with hope,” explains Kristina Figueroa, MSPH, who is an expert in type 1 diabetes clinical research and public health, and a passionate patient advocate.
Like most of us, Figueroa says that after her own type 1 diabetes diagnosis at age 6 in 1996, her healthcare team told her a cure was just around the corner. Due to the half dozen cures of type 1 diabetes in mice each year, it can seem that way.
“We’ve made huge advances,” adds Figueroa, “but we’re still nowhere close.”
The researched treatment efforts closest to a successful cure come down to one, maybe two, that have the potential to progress through each critical trial phase.
And hopefully, one day it will become available to all patients with type 1 diabetes.
ViaCyte’s Encaptra cell delivery system
Without a doubt, ViaCyte’s implantable capsule device is closest to paving the way to a successful cure.
How it works
First developed by doctors, ViaCyte’s technology was tested on over 1000 rodents with medically induced type 1 diabetes.
It currently has two clinical trials in progress with two different devices: the Encaptra capsule device which contains pancreatic beta cell precursors (also known as VC-01 or PEC-Encap), and the PEC-Direct works very similarly but is for a more vulnerable portion of the diabetes population.
ViaCyte began working with the University of California, San Diego (UCSD) as its first clinical site.
Update: ViaCyte was acquired by Vertex in 2022, with the goal of accelerating its programs in type 1 diabetes
The PEC-Encap
The PEC-Encap is a fully encapsulated product, and as such, does not require immunosuppression.
Once implanted within the skin, the precursor cells then mature into fully functioning endocrine cells that produce insulin and other hormones like amylin that play a critical role in stabilizing blood sugar levels.
However, the critical detail that makes or breaks ViaCyte’s success is the body’s ability to “vascularize” the product.
“Vascularizing means that blood is flowing through it, allowing the pancreatic progenitor cells to mature into insulin-producing islet cells,” explains Figueroa.
Figueroa adds that the procedure itself is fairly simple, though outpatient, and a short recovery period.
“And then, it takes several months for the product to get vascularized in your system, mature into islet cells, and start producing useful insulin.”
Because the capsule isn’t connected in any way to the existing pancreas, and it’s safe from the patient’s self-destructive immune system, the beta cells can thrive and produce insulin to successfully manage blood sugar levels.
However, do keep in mind, it’s expected that patients would need to undergo the procedure perhaps every year in order to implant more progenitor cells; this is what ViaCyte is testing in the initial cohort of the ongoing Phase I/II Clinical trial with the smaller, sentinel units.
“At the 2018 ADA, ViaCyte reported two-year histology of VC-01 explants showed regions containing insulin-producing beta cells and glucagon-producing alpha cells, indicating that when vascularization occurs, cells can persist without the need for immunosuppression,” explains Figueroa.
The long-term goal, she adds, is to make the regular addition of new cells a simple non-invasive procedure. And ideally, eventually no need for additional cells at all.
The PEC-Direct
This form of the capsule technology works very similarly to the PEC-Encap but it has a few key differences.
PEC-Direct provides direct vascularization of the graft cells and thus does require chronic immunosuppression; as a consequence, the target population is the high-risk type 1 diabetes patient — more specifically, those patients with hypoglycemia unawareness or those with extreme “glycemic lability,” sometimes referred to as the “brittle diabetic.”
How long until ViaCyte’s cure treatment is widely available?
“It’s important to remember that, even though they’re making incredible progress, they still have a long way to go,” says Figueroa.
Figueroa points out that simply getting to phase 1 of clinical trials took ViaCyte more than 10 years. But they’re making progress.
There are currently two clinical trials in progress: the first is with the PEC-Encap technology. The second is with PEC-Direct. Let’s take a closer look at the phases they must achieve over the next decade:
Phase I: Safety & Tolerability
Cohort 1 of this Phase I/II study was entirely focused on the capsule’s ability to become vascularized within the human body. This phase did not look at whether it managed blood sugars, but simply that the body was able to accept and make use of the capsule by flowing blood through it and picking up the insulin being produced by the cells in the capsule.
“It has to get into the patient’s bloodstream before it’ll start working and producing insulin,” reminds Figueroa.
ViaCyte accomplished this phase in 2018 with a small trial of 4 patients.
Phase II: Efficacy & Side-Effects
As ViaCyte begins Cohort 2 of the Phase I/II study, things get exciting because this is where they determine if the vascularized capsule will effectively produce insulin and manage a patient’s blood sugar levels.
In fact, they’re still recruiting patients for several locations of human trials for the PEC-Direct. Here are the locations:
- San Diego, California
University of California San Diego
Study Coordinator: 844-317-7386 or alphastemcellclinic@ucsd.edu - Baltimore, Maryland
Johns Hopkins University
Study Coordinator: 410-614-0905 or doluka1@jhmi.edu - Minneapolis, Minnesota
University of Minnesota
Study Coordinator: 612-626-4993 or kreel001@umn.edu - Columbus, Ohio
Ohio State University
Study Coordinator: 614-688-6885 or ashley.mintos@osumc.edu - Edmonton, Alberta, Canada
University of Alberta
Study Coordinator: 780-407-1501 or parastoo@islet.ca - Vancouver, British Columbia, Canada
University of British Columbia
Study Coordinator: enrolling by invitation only
To be considered for enrollment, a patient must fall within these categories:
- Men and non-pregnant women of non-childbearing potential
- Diagnosis of T1DM for a minimum of five (5) years
- At least one (1) severe hypoglycemic event in the previous 12 months
- Hypoglycemia unawareness or significant glycemic lability
- Stable diabetic treatment
- Willingness to use a continuous glucose meter
- Acceptable candidate for implantation
If the patient has a history of the following, they would not be considered:
- History of islet cell, kidney, and/or pancreas transplant
- Six (6) or more severe, unexplained hypoglycemic events within six (6) months of enrollment
- Uncontrolled or untreated thyroid disease or adrenal insufficiency
- Diabetic complications such as severe kidney disease or renal dysfunction, proliferative retinopathy, diabetic foot ulcers, amputations attributable to diabetes, and/or severe peripheral neuropathy
- Non-compliance with the patient’s current anti-diabetic regimen
Once they are able to get 55 patients enrolled, vascularized, and able to proven efficacy, it could take another 2 years to compile that data and progress to Phase III of the clinical development plan,” explains Figueroa.
That means, if required by the FDA upon review of Phase I/II data, Phase III could potentially begin around 2022 if all goes well in phase I/II.
Phase III: Efficacy, Superiority & Monitoring of Adverse Reactions
Phase III in clinical development is when a treatment method or drug is tested on a wider population of patients, for a longer period of time, and, oftentimes on a global scale
“This phase would need anywhere from 300-3,000 patients, and would likely need to be global,” explains Figueroa. “Going global is crucial because patients from one part of the world have different genotypes and phenotypes than patients in another part of the world”
Phase III would ensure the treatment method is effective for a larger part of the type 1 diabetes population, and also monitor the treatment uptake and adverse reactions after longer-term exposure. This phase would persist for 1 to 4 years to further determine safety, efficacy, and long-term stability in multiple sub-populations.
Phase IV: Submit for FDA approval
The phase we’re all hoping for is phase IV — when the treatment has proven to be widely safe and effective, and ready for the rest of us!
In some countries, approval from governing bodies is quick. In the United States, approval from the Federal Drug Administration can take several years.
Ideally, if ViaCyte continues to thrive in each clinical trial phase, we could see the Encaptra delivery system commercially available by the year 2030. Hopefully…sooner!
2nd contender: The BCG Vaccine from Dr. Faustman
The BCG (Bacillus Calmette Guerin) vaccine has existed for nearly 100 years. Originally designed to prevent tuberculosis in areas of the world with higher risks of developing the disease. Dr. Denise Faustman began researching the use of this vaccine in people with type 1 diabetes at the Faustman Lab at Massachusetts General Hospital.
The simple result was that the vaccine was regenerating (or repairing) the pancreas’ ability to produce beta-cells, which in turn successfully produced insulin.
Remarkable success in mice
While it worked remarkably well in mice, it took much longer to see the impact on humans — nearly 3 years after the initial injection.
“This is to be expected,” explains Faustman. “Mice have much shorter life spans and are different in many ways. But like the mice, the lowering of blood sugars to the near normal range continued beyond the 8 years of the clinical trial.”
“We have learned that the NOD mouse model (and we were the first lab to reverse disease in end-stage mice) is not perfect, but fortunately it did correlate for us. We now believe that the reason BCG takes a while to work is that it is permanently resetting the immune system.”
This understanding comes not just from Faustman’s team but also from a global coalition of scientists in research.
In the ongoing Phase III clinical trials with multiple sclerosis, there is a similar lag in autoimmune reversal of 2 years but then continuous benefit beyond 5 years with this simple and safe generic BCG intervention.”
And in 9 humans with type 1 diabetes
She has tested her research on a total of 9 patients with type 1 diabetes. While none are “cured,” most experienced significant reductions in insulin needs.
“We have a fully enrolled Phase IIb clinical trial underway that will read out in as early as 2022,” explains Faustman told DiabetesStrong. “Because of the size of our Phase II trial and BCG’s known safety profile and its generic drug status, we are hoping to pursue approval coming out of Phase II trial.”
Faustman adds that the BCG vaccine has demonstrated a clear and clinically significant response in the primary endpoint for type 1 diabetes trials: a stable and long-term return of blood sugars to the near normal range for over 5 years without hypoglycemia.
- Read more about the clinical trials and potential to participate at Faustman Labs.
Political issues: ADA/JDRF vs. Dr. Faustman
Despite Faustman’s passion and dedication to finding a cure, both the legitimacy of her work and the results have come under great scrutiny by the American Diabetes Association and the JDRF.
“I am still not sure why they chose to issue it, but it was a political not a scientific response to our work, which neither group has funded,” said Faustman.
Fortunately for the type 1 diabetes population, Dr. Faustman continues steadfastly in her work towards a cure despite lack of support from the larger diabetes organizations.
Other research in early stages of development
The following studies and treatment methods to cure type 1 diabetes have not moved beyond curing type 1 diabetes in mice. If these attempts at curing type 1 diabetes progress to human trials, and are able to pass phases 1 through 4 of clinical trials, these approaches to curing type 1 diabetes are still a minimum of 20 years away from bringing a commercialized cure to the market.
Vertex (formerly Semma Therapeutics)
Launching from initial research by Doug Melton at Harvard, this method focuses on stem-cell therapy and has achieved a cure in mice.
“Semma’s science is focused on bringing the promise of stem cells to patients through generating billions of stem cell-derived pancreatic beta cells in combination with a state-of-the-art cell delivery technology,” explains their website.
“The pancreas normally differentiates from the endodermal germ layer, and early research in generating pancreatic tissues from stem cells mimicked the step-wise developmental signals observed in model organisms. This work led to the successful generation of stem cell-derived definitive endoderm and pancreatic progenitor tissue. However, the biggest hurdle has remained—differentiating human pancreatic progenitors into fully functional insulin-secreting beta cells in vitro.”
Injections of Collagen & Pancreatic Cells
This research from Purdue and Indiana University is using injections of a solution of collagen and pancreatic cells for a minimally invasive therapy that aims to reverse type 1 diabetes.
In mice with medically induced type 1 diabetes, it has proven to work within 24 hours, maintaining “insulin independence” for at least 90 days.
It’s described as being Trojan-horse-like because the pancreatic cells are ushered in along with a protein the body already makes (collagen) for building muscle, bone, skin, and blood vessels.
The next step is a pilot clinical study on dogs that have naturally occurring type 1 diabetes.
- Read more on type 1 diabetes research at JDRF.org.
- Learn about other clinical trials looking for participants at JDRF.org.
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Suggested next post: What’s the Difference Between Type 1 and Type 2 Diabetes?
John the Greek
My son has diabetes for three years now. I am not sure scientists will find finally a cure. I am very disappointed. The money of diabetes cure with insuline are billions so they don’t want loose this.
Ron Williams
Yes John, I asked a endocrinologist of mine in about 1990 if he thought there would soon be a cure for diabetes type 1 in the near future. His response was “there will be no chance in my lifetime for a cure”. He added… “Unfortunately there is no money in cures”. I’ve had diabetes for 44 1/2 years, most of that time was with no control what so ever, I have had 2 heart attacks and most of the associated problems with uncontrolled diabetes. In the last 8-10 years I’ve done much better controlling it (rather it controlling me) I life isn’t wonderful but I feel blessed that I’m still around andwith a decent quality of life. Looking back… my life today would be more lively and less limited if I had took my diabetes more seriously….. If this is what your son is doing…. Now is the time to change this. Diabetes is one of the more denied illnesses, but denying won’t make it go away.
Lea Ann
Thank you so much for your information. I was diagnosed with Type 1 Diabetes on August 17, 2016 at the age of 55. I have always been very fit and healthy….bike, sail, walk, kayak, etc. They don’t know what happened to trigger this disease. It is Type 1 and not LADA or Type 2. The tests have been done twice by two different Endo’s…one in Florida and one in Texas.
I am still hopeful for a cure. My heart goes out to all that have been fighting this battle for decades. The cure is out there and I look forward to the day it discovered. Like others here, I think the key is to fix the immune system. A re-boot of our system like a computer when it crashes. Thank you again for all your information.
Jack
Lee Ann
Just wondering what prompted you to get tested in 2016, did you have symptoms? I am asking because I am the same age, healthy and my doc notice my C-peptide was slightly low?
Regards
sandy witbeck
My family carries Lada, a mix of type1 and type2, but I am classified as a 1. I hope you have tried metformin, which was a game changer. It some how stabilized myself and my cousin. I was diagnosed at 52, what a ton of bricks to drop. I had to stop the adventerous travel. Now a big trip is hawaii where a hospital is there just in case. Straun Grant researches late onset diabetes thru the Univ. of Penn. He is so far the only one.
Vinícius
Well i had 25 now and since 5 i was type 1… i controled it without the rights tools… i did check-ups in wrong doctors too… i almost blind in my left eye and my right one are not perfectly… at least i not lost foot, or organs in general… since a year ago when i say to my mom that my eye sight are terrible we find another doctor to make check-ups and we invest more in control… i live in brazil, and the tools goverment release to population is totally useless, shifted to tresiba insulin for long term and lovolin R to meals and peaks (i need a better for this), even better i start buying abbott freestyle sensors and miaomiao to synch up with my smartphone… then i noticed my lifestyle are totally insane and cut off 1/3 to 1/4 of main meals, cut off all not meal foods… and inject more fast acting insulin… my sugar levels are much better now, trying to keep what i have nowadays… i try exercise myself too, but i hate gym, soo i just walk and i will run when doctor take off my limits to not end blinding my eyes hehe… i pay little more than 1/4 of my paycheck (that is more than average population had here in maybe 2 times) just to trate diabetes and my goverment say i not using the rights tools… well when i try using what they leave to us, i almost killing myself… be aware about how to trate it, not just trying. From brazil here, this country sucks a loot… but now in 2019 Bolsonaro has retired some tax and we will get afrezza soon, i will try, my main fear is the cost here… i already are lossing quality in other areas of life to had one… i not able to buy a car or had a house, even with paycheck over the average… 5k is the 10% richests peopels here and i’am here close to this values… this country sucks in soo many ways :/
Christel Oerum
Sounds like you are doing the best you can with what you have available. But painful to know that so much of one’s paycheck goes to keeping us alive
James Boone
Commendable post Ginger!
Type 1 diabetes requires insulin intake otherwise it can adversely impact our body. Kindly provide more information about this trial.
Ginger Vieira
Hi james! We provided TONS of insulin about this ongoing research in the article. Please click the linked text to go directly to the sources. YES, type 1 diabetes requires daily insulin injections or insulin via a pump or pod to stay alive. This cure research is trying to reduce or eliminate the need for those injections by enabling the body to produce insulin again.
Muhammad Maqbool
Thanks for sharing this awesome information about Type-1 D. I’ll be very glad to know where and how can one register for this trial. I guess this will bring great changes. Will be looking for a response to know where and how to register for this trial. Thanks. Regards, Maq.
Edward
Thank you so much Ginger for taking the time to publish such a well written and informative article. I was diagnosed with T1 at 21 after having a bad virus and have had it for exactly 30 years now . I’ve sadly seen many false claims of cures over that time and try not to get my hopes up – however, PEC-Encap seems very interesting to me and I applied for the trail. If I hear anything I’ll be sure to update you on my progress. Thanks again!
Ginger Vieira
Hi Edward!
There is SO much research out there trying to determine the triggers/causes of type 1 diabetes. I’d highly recommend going to PubMed.org and searching to see the many results. That being said, it is well-known that a stressful event (illness, divorce, car accident) can trigger the autoimmune attack. For you it sounds like that food poisoning event overseas was the trigger for yours. (Trigger, not cause). For me, the flu was the trigger of my T1D onset.
I’m sorry this doesn’t answer your question more clearly. There is SO much research on this topic — and TrialNet, for example, tests children of type 1s for autoantibodies. Children with results of autoantibodies forming before signs of diagnosable type 1 diabetes are being enrolled in treatment studies that are, for many, preventing or delaying the further development of type 1. Check-out TrialNet.org for more on that!
Again, there’s so much working on this but there’s nothing we can buy at the check-out counter yet! ;p THANK YOU for reading!
Ron
Can someone share with me a research study into why the immune system may attack it’s on beta cells? The triggers I can say for myself getting this disease of type one I feel stemmed from getting sick after eating bad food in another country. Ever since then my body started to experiment many ailments until I was diagnosed type one 8 years later. You would think that with the knowledge of genes we could some day find the one that could turn this negative function of destroying our own cells off at the immune system.
Is there a study revolving around gene therapy’s in this way? Or is it even possible. Thank you and for everyone out there stay positive.
Philip Stein
Hi there, what about curing or putting type 2 in remission? Are we there yet, or even close? Phil
Christel Oerum
Ginger recently wrote an article on Type 2 diabetes remission. You can find the article HERE
Vincent Fitzpatrick
Hi,
My daughter Kate which is 15 has had Type 1 since she was 7. She is really struggling with the disease at the moment. Not physically as she is very fit and really makes sure her diet is very controlled. She has been an absolute hero in the quiet way she deals with this horrible disease. But in the last year we have noticed how disappointed Kate has become that a cure has not been found.
We / she would love to take part in any clinical trials of this or other cures or treatments.
Many thanks,
Vincent and Paula Fitzpatrick.
Christel Oerum
I’m sorry to hear that the lack of a cure is impacting her so much. Hopefully, researchers will get a breakthrough but until then participating in trials is a great idea. I’m not sure Kate can participate until she is a little older, but it’s worth looking into. To find clinical trials near you try doing a google search on “diabetes clinical trials near me”
Norma
Hi Ginger,
Thank you so much for this informative article! My son is 15 and has had type 1 since he was about to turn 5, it’s been extremely hard for him and still is very challenging for all of us since apparently he just does not want to take care of it. He’s always refused to accept it and doesn’t want to deal with it anymore. He is also very disappointed that there’s no cure yet. I always encourage him that a cure will be found and in the meantime he should cooperate to manage this (wretched) disease better.
We noticed though that the illness mostly attacks Irish, English, Scandinavian descendants, in our case, nobody else in our family has it, but his dad and grandparents are Irish descendants.
We live in Canada and we are also looking for any new treatment or trials.
Jo F
Thank you Ginger…hope you’re well. Happy 2019!!! Appreciate the information. I might have missed it but when I think of a cure I think of “what is causing the immune system to go haywire”. I see a vaccine as a treatment, along with an implantable (where the immune system could still be going haywire). I’d love to see more info and/or research into what in our environment (EMF? Leaky gut? denatured food), biology, microbiome….etc is causing our “inflammation” to get to such a level that the immune system wants to kick in and find the culprit (but is not). Or, just too complex to turn around since so many things are involved and hormones are so tricky and gotta be hard to “research”. Hearing more and more from the functional medicine consortium…..it seems if we can get a handle on those things…that would also be a great breakthrough. 🙂
Ginger Vieira
Jo! As a fellow type 1, I hear ya loud and clear on the definition of a “cure” being treating the immune system versus implanted technology that produces insulin. It’s definitely up for debate, and many folks feel like you do on the subject. That being said, ViaCyte’s treatment method is being labeled a cure in terms of creating a closed-loop self-running method of achieving non-diabetic blood sugar levels.
Your quest for more research on those other topics…there’s a lot out there! Have you ever spent time on PubMed searching through studies? You could spend hours, weeks, months in there! Let us know what you find 😉 it’s endless!
Ezinne
I live in Nigeria and I follow your blog closely. Can someone in Nigeria participate in this trial?
Many patients here die within a few years because of high cost of insulin vis a vis ,low income .
I know a few people who meet the criteria above.
Can they sign up?
Ginger Vieira
Hi Ezinne,
I’m afraid not — the legalities and the logistics of including participants from other countries would make that impossible. It is heartbreaking that the cost of insulin today leads to a death within a few years in so many countries — and it shouldn’t be that way. Hopefully the fight that is brewing in many countries all over the world to lower the price of insulin makes headway soon.