Salk Institute Evans Lab Diabetes Research FAQ
Introduction
The lab of Salk Institute Professor Ronald Evans has made a major breakthrough in the treatment options for type 1 diabetes: the development of transplantable human cells that make insulin, controlling blood sugar— without immunosuppressants—in mouse models. The lab has dubbed these cells “HILOs,” short for human islet-like organoids. HILOs are three-dimensional pancreatic cell clusters derived from human stem cells. Read the press release about the discovery here, and see the FAQ below for more information.
Q: In the best-case scenario, how soon could this treatment be available for humans?
A: We expect that it could take 2-5 years to get FDA approval to use of HILOs in people. Typically, clinical trials of treatments designed for humans have four phases, with each phase looking at different aspects of safety and effectiveness, in increasingly larger groups of people. Because study subjects must be followed for certain minimum lengths of time, clinical trials can take a few years even in the best-case scenarios, and are costly to conduct.
Before clinical trials can even begin, preliminary research in the laboratory must indicate that a treatment shows promise. Financial support is critical at this stage to help ensure the best scientific minds are hired to conduct the research as effectively and as thoroughly as possible.
Q: In mice, your HILOs maintained blood sugar for 50 days. Does that mean HILOs have to be administered on a monthly or so basis?
A: No. 50 days was an end point in our mouse studies. In people, we believe that HILOs could last for years, making them game changers for people with type 1 diabetes.
Q: Would this be a treatment people would get in a clinic, or would it be something people could do themselves, the way they inject insulin?
A: For safety, transplanting HILOs would be done in the clinic, similar to the way donor islets are transplanted into patients via injection into a vein.
Q: Since Type 1 diabetes is an autoimmune disease, is it possible to use your immune protection factor to prevent people who may be at risk for Type 1 diabetes from getting it?
A: No, our technology currently works for transplanted cells. However, further research may reveal additional applications that may improve human health.
Q: What do you mean by cure? Others have talked about a “cure’ but often with qualifiers. Ultimately, what does “cure” mean for persons with diabetes and how will their lives change?
A: Patients who receive HILOs would no longer need to monitor blood glucose levels or inject insulin. Additionally, the level of daily effort, inconvenience and physical discomfort would be greatly reduced for these individuals. It is not an understatement to suggest that HILOs would be both life-changing and life-saving.
Q: Will this treatment require any ongoing medications, procedures, etc., for persons who are treated for diabetes?
A: Although we envisage no additional therapies being required, ultimately HILOs need to be tested in humans to ensure their safety and effectiveness. Once a therapy is found in the lab to be promising, it can proceed to clinical trials. Both of these phases of getting a treatment to patients can be lengthy and costly.
Q: What makes this approach/treatment different from other options that purport to be a potentially viable treatment for diabetes?
A: The immune shielding of HILOs will allow them to be transplanted without a containment device and without immune suppressive therapies, which are expensive, often have unpleasant side effects and carry additional health risks, especially when individuals must take them for the duration of their lives.
Q: I’m a physician. How can I get more information about this research?
A: If you are a physician or researcher requiring additional information, please contact Ronald Evans at evans@salk.edu.
Q: How can we support this research or the Evans lab?
A: Charitable donations are vital to accelerating the progress of this research. Please contact India Mittag at imittag@salk.edu or 858-453-4100 x2279.