Skip to Main Content

The term “type 1 diabetes” generally conjures up images of insulin. That makes sense, because insulin is the main treatment for this common disease. But it isn’t a cure. A type of cell transplant that comes close to a cure for some people with type 1 diabetes, a technique pioneered and tested in the United States, is now available in many countries but is still deemed an experimental procedure in the U.S., making it almost impossible to get.

That doesn’t make sense to us.

Type 1 diabetes, which affects 1.25 million American children and adults, and more than 20 million people around the world, is a challenging chronic disease caused by the body’s inability to make insulin. Among its most severe forms is brittle diabetes. People with brittle diabetes frequently experience large swings in blood sugar that can quickly move from too high to too low or vice versa. Severely low blood sugar, called hypoglycemia, can cause sudden and unexpected seizures, coma, heart attacks, and even death.


Insulin is made by specialized cells in the pancreas called islet cells. Transplanting these cells from a donated pancreas to an individual with brittle diabetes can restore the recipient’s ability to naturally produce insulin. The procedure has a record of effectiveness. A Phase 3 clinical trial sponsored by the National Institutes of Health, for which one of us (C.R.) was an investigator, showed that such transplants worked in 80% to 90% of the patients treated in eight centers in North America. The procedure virtually eliminated the risk of life-threatening hypoglycemia one and two years after the transplant. As with other types of transplant, the recipient must take anti-rejection drugs.

Individuals with brittle diabetes in Canada, Europe, Asia, and Australia can receive islet cell transplants, much the same way that individuals who need new hearts or livers can receive transplants. Islet cell transplants are even performed in China and Iran, whose doctors came to the U.S. to learn the technique and carried it back home. So why is this successful procedure, which can vastly improve the lives of those living with brittle diabetes, available in the U.S. only after a convoluted process that is often impossible to complete?


If a patient needed a pancreas transplant (which would include islet cells), he or she would be registered on the national organ transplant list and, once a pancreas became available, would receive the transplant, which is generally paid for by insurance.

But for a less-invasive islet cell transplant, an institution that wants to perform the procedure must file an investigational new drug application with the Food and Drug Administration, a very challenging process — and also figure out how to pay for the transplant.

That’s due to the way the FDA interprets the code of federal regulations; specifically the criteria of minimal manipulation of human cells, tissues, and cellular and tissue-based products.

After islet cells are extracted from a donated pancreas, they need to sit in a culture medium at a temperature between 72 and 75 degrees Fahrenheit for two to three days. This gives the transplant team time to perform quality controls on the cells, and also to prepare the recipient for the transplant.

The FDA has interpreted the brief hiatus for islet cells as going beyond minimal manipulation, a concept based on the premise that processing cells does not alter their relevant biological characteristics. The hibernation process for islet cells does not change the cells’ characteristics or increase their number, both of which occur with the manipulation of advanced stem cell therapies, which reasonably need extra scrutiny.

The European Medicines Agency has determined that transplanting pancreatic islet cells should just follow the standard rules of organ transplantation, even though the cells require a brief hibernation period. This recommendation is based in part on its view that transplanting a pancreas, with islet cells intact, is an organ transplant, so there is no reason to treat transplantation of just the islet cells as anything different.

Because of the benefits to the thousands of Americans with brittle diabetes, there is a strong incentive to harmonize the FDA’s approach to islet cell transplantation with the EMA’s approach.

More than a decade ago, the United Kingdom’s National Health Service approved islet cell transplantation for type 1 diabetes — an approval based on an extensive review of the evidence generated by clinical trials conducted in the United States. Our federal dollars supported that research, and this treatment ought to be available to U.S. citizens.

Islet cell transplantation is not a panacea for all forms of type 1 diabetes. And transplantation of any organ, including islet cells, requires the use of anti-rejection drugs that can have a range of adverse side effects. That said, individuals with severe brittle diabetes who are fully informed of the risks and benefits should have the ability to access this lifesaving treatment option.

We fully understand the FDA’s efforts to rein in companies marketing unapproved stem cell products that have little or no evidence to support their use and that may put patients at risk. Yet the FDA should stay equally focused on its commitment to approving evidence-based transformative treatments for devastating diseases and conditions, including brittle diabetes.

Camillo Ricordi, M.D., is professor of surgery and medicine and chief of the Division of Cellular Transplantation at the University of Miami Miller School of Medicine, where he has directed the Diabetes Research Institute and Cell Transplant Center since 1996. Anthony Japour, M.D., is a medical director at ICON plc. The views expressed are those of the authors and not those of the University of Miami or ICON.

  • You state that “a type of cell transplant that comes close to a cure for some people with type 1 diabetes, a technique pioneered and tested in the United States, is now available in many countries but is still deemed an experimental procedure in the U.S., making it almost impossible to get”. Was this technique (the Edmonton Protocol) not pioneered in Canada by Dr. Shapiro?

  • Is there anything we can do I have type one diabetes and yes I have times I don’t feel like I’m worth living because of this type 2 diabetes ruined my childhood and stunt my growth and there’s a chance I can make it go away forever getting an islet transplant and the fda won’t approve of it this is a twisted country we live in they value making money off of our disease rather then help us and it’s nothing I can do but cry every night about this Having diabetes made me not enjoy life and people who don’t have it don’t even bother trying to comfort me or make me feel better you won’t ever no how I feel and it’s nothing anyone can do about it I wanna get rid of it I’m really suicidal rn

    • My son is 13 yrs old diagnosed with T1D age 11. He was in the honeymoon stage for almost 8 month We we’re giving him a small dose of lantus daily but no novalog during the honeymoon stage. He is now completely insulin dependent but is so active with sports we’re constantly having to adjust his dose. Are there any trials that would work for him We live in Boston MA or any diets you can recommend us trying. Thank you

  • Thank you DeWyn you don’t have an idea how much strength I got after read your comment. It mean a lot to 8 years old mom who live with the non stop worries about her son diagnosed with type 1 diabetes at the age 4. Thanks again and sorry about my English it’s my second language.

  • This is all very interesting. I have been Type 1 for over 45yrs. I was told I was “brittle” within the past 5 years. This article, though inspiring, creates much curiosity. About 10-15yrs ago, I also heard about a Canadian Clinical Trial that had Type 1 Diabetics, they were given something by mouth… I believe it was also islet cells… and were reported to be insulin free for 2yrs ( at the time of the report). Curiosities are 1) why, being the most advanced country in medical treatments, is the USA the Farthest behind in actual treatment of patients… WE pay for the studies – through our taxes & donations, the insurance & the prescriptions to keep us alive. 2) can you name some specific anti-rejections drugs…. sometimes the side effect & the Success rates do not equal the chance of trying something that may or may not work.

    For those parents out there, relax. the first few years WILL be frightening at times…. I was in & out of the Hospital several times between diagnosis at age 8 & “settlement” by age 12. Being insulin dependent in today’s world is a far cry different than it was 40 years ago. Don’t let it stop you, or your child from doing ANYTHING…. I’ve white water rafted, Rock climbed & rappelled, I Surf, swim, walk, ski, play sports, was in the Marching band…. just like any other kid. keep the glucose monitor & honey ( Perfect for slipping under the wet suit when surfing) on hand. I eat ice cream ( I substitute other carbs for the luxury). Learn balance. learn patience. LOVE your child. Look into training classes provided by your heath care provider – there’s plenty of information out there. Blessing & I wish you the best! – Oh! yes, the YMCA offers phenomenal Diabetes Summer camps, where your children can be with other Diabetics & make life long friends who know what they are going through!

    • Thank you DeWyn you don’t have an idea how much strength I got after read your comment. It mean a lot to 8 years old mom who live with the non stop worries about her son diagnosed with type 1 diabetes at the age 4. Thanks again and sorry about my English it’s my second language.

    • That’s inspiring. I’m currently going through some depression worrying about my kids growing up without me. I just got my 30th anniversary of type 1. This give me hope.

    • The Edmonton Protocol pioneered by Dr. Shapiro at the University of Edmonton did not (and does not) involve giving type 1’s “something by mouth”. It involves a transplant of islet cells from cadavers (a minimum of two pancreases are required) which are injected into the portal vein of the liver. These transplants are still being done although the technique has changed somewhat to make the process and the results better. However, the requirements to get one are very stringent and I know of at least two people who were turned down because they didn’t meet the requirements.
      People should keep in mind that anti-rejection drugs come with their own set of risks, one of which is an increased risk of cancer. I am a two time/two types cancer survivor and, having lived through those, I would not consider a transplant, so I have never applied. Some people might think it’s worth the increased risk, but I am not one of them. A diabetes diagnosis is scary enough, a cancer diagnosis is far worse.
      At any rate, I have lived with this disease for 56 years, I’m doing okay and I plan to keep it that way as long as I’m able.


      Study Website:

      THE STUDY IS A SURVEY which takes about 30 minutes to complete and can be taken completely on-line. This study asks you how you would rate the risks and benefits of new treatments that are being developed for T1D. Specifically, we will ask you to weigh the risks and benefits of a treatment called “ISLET CELL TRANSPLANT”. You will choose between a mixture of risks and benefits for an Option 1 and an Option 2. You will choose which option you prefer. Then we will change the combinations and ask you again to choose. We also have questions about yourself, your diabetes, and your quality of life.

      18 years or older and have TYPE 1 DIABETES ( YOU TAKE INSULIN).
      Also, you much have had hypoglycemic (low blood sugar) episodes. We are looking for those with ‘important hypoglycemia events’ where it might be nice to have someone else there to help you. Thanks.

      The process to take the survey is pretty simple:
      1. First email me [email protected]. I am a Research Professor from University of California, San Francisco
      2. I will then send you a CONSENT FORM which describes the study, what you should expect, and your rights for taking the survey,
      3. When you email me back that you are eligible and agree, then I will email you a SURVEY LINK AND A STUDY NUMBER. You will click on the link and enter your study number and then take the survey at home!
      4. You can always call me or email me with any questions before during or after taking the survey. (Telephone: 415-990-1012) (Email: [email protected])

      Thanks so much. Leslie

  • I have been dealing with having brittle diabetes for 35yrs now. My blood sugar drops very low out of nowhere. It also will get dangerously hi. It at one time got to 1600. My body was shutting down. God was holding me. If there is a way to improve or cure brittle diabetes that research has proved has improved quality and health of people that live with this debilitating disease. The FDA should be supporting the approval and use of such treatments.

  • Why does the A.M.A. keep this procedure away from us? I think they stand to loose some high profit procedures.

  • I have been a type 1 diabetic for 43 years, my mom and dad both passed waiting for a cure. I can’t believe we paid for the research and the side in the United kingdom after they came over here to learn about it but it can’t be done here, that’s wrong. Too many of us need it and have lived long enough without it. It’s about helping save people, not big pharma collecting until we die!!!!

    • please Camillo I need help.
      My son was diagnosed with type 1 diabetes last year, and his blood sugar spiked too high and drop too low. I don’t sleep at night. he’s only four years old.
      Any help is highly appreciated.

  • Please find a cure our daughter was diagnosed at age 5 she is now 6 this is the worse disease ever super high super lows so fast when everyone is playing she dropping and keeps asking why why cant they find a cure our hearts are breaking

  • My 44 year old son was diagnosed with type 1 two years ago. Now his daughter who is 7 was just diagnosed 3 months ago with type 1. Praying for a cure and praying for the scientists to find a cure. This is life changing to all of us in our family. Please find a cure soon. My son and Granddaughter are counting on it as our whole family is.

  • I have a dear friend who has had great success after her islet cell transplant. She has gone from huge swings in blood sugar (despite being otherwise healthy and conscientious) to requiring no insulin at all. In fact, she has been completely insulin free for years now. Before her transplant she had several pretty significant scares. Her life has been drastically changed, and likely saved, due to this procedure. The thought of delays due to policy is disheartening. It is my opinion that this option should be open and available for patients who meet the criteria and whose doctors feel it would benefit.

    • I am 41 years old and I’ve had Diabetes since I was 7. I have since also developed Graves Disease, which led to the decision to undergo radio-ablation. I would give anything for a cure for Diabetes. I am in phenomenal shape and follow a strict eating regimen, however the disease is the disease and it’s a tight rope walk that I just can’t seem to stick. I know there’s a cure out there and I just need to find it. The side effects of the anti rejection drugs are almost as debilitating as the disease itself. I need us to do better.

Comments are closed.