Frequently Asked Questions
Why is the current standard of care (exogenous insulin, pumps, glucose sensors, etc.) insufficient for patients with T1D?
There have been great technological advances in the last several years that help many diabetics improve glycemic control. However, there is a subset of patients diagnosed with brittle T1D. These worst case T1D patients no longer feel when their blood sugars drop to life threatening low levels (hypoglycemic unawareness). An islet cell transplant can not only prevent patients with brittle T1D from going dangerously low, but in most cases will also allow them to live a life without insulin injections. Furthermore, many “standard of care” technologies rely on patient compliance and can be a financial burden for the patients or caregivers. An islet transplant will provide the patient with more autonomy and reduce medical costs over their lifetime.
What is allogeneic islet transplantation and why is it a better alternative than the standard of care?
In an allogeneic islet transplant, islets are taken from the pancreas of a deceased organ donor. The islets are purified, processed, and transplanted to a patient with brittle T1D. Once transplanted, the beta cells in the islets begin to make and release insulin. For most patients, islet transplantation eliminates hypoglycemic unawareness and provides a “functional cure” so that they no longer require daily injections of insulin. Furthermore, improved glycemic control achieved after an islet transplant prevents secondary complications associated with the microvasculature, which include cardiovascular disease, neuropathy, nephropathy, and retinopathy.
What can be done to expand access to allogeneic islet transplantation — an experimental procedure that can serve as a sustainable cure for T1D? What barriers need to be addressed?
The Food and Drug Administration (FDA) has determined that the islet purification process is beyond the criteria in the code of federal regulations regarding minimal manipulation. Because of this, islets are regulated as a biologic drug. For an islet cell transplant to be considered a standard care option, it is necessary to receive FDA approval of the biologic drug “islet cells.” A biologic license application (BLA) is an enormous task with a high level of scrutiny. Once a BLA for islet cells is approved, insurance coverage, medical procedural and billing codes are also necessary to remove barriers to access which will make the procedure more affordable for diabetic patients. These steps are costly. In the meantime, the CDP supports patients who need an islet cell transplant through restricted grants to medical centers that are approved by the FDA to perform the procedure.
Aside from islet transplantation, what existing and/or emerging research or technologies are most promising to serve as an artificial pancreas?
The CDP supports important research investigations to improve upon allogeneic islet transplantation. As with any transplant, anti-rejection (immunosuppressive) drugs must be taken, which can have a range of adverse side effects. To eliminate the need for these medications, the CDP supports work to encapsulate islets to protect them from the host’s immune system. The CDP also supports work which utilizes the blood of T1D patients to learn new information about the innate and fibrotic responses to biomaterials in order to establish draft protocols for clinical trials/biocompatibility testing and implantation of capsules in patients with T1D.
What are the major barriers to advancing these technologies from the research bench to the clinic?
Funding is a major barrier to support the costly and complex research. These experiments are expensive. Grant funding is often slow and delayed. The CDP can provide timely seed grants to our investigators that can help bridge delays from larger funding agencies.
How does the CDP help advance ongoing diabetes research, including international collaborations, grants, etc.?
The CDP advances ongoing diabetes research through collaborations and grant funding. We facilitate communication and sharing of experimental results among collaborating teams. We also add nimbleness to the research by providing funding for critical experiments that may not be covered by other grants.
What type of work has been funded by the CDP to date and what is the current status?
The CDP supports islet cell transplant clinical trials, encapsulation technologies and research to solve the problem of an unlimited insulin producing cell source.
Does CDP offer resources to patients and families impacted by T1D?
Yes, the CDP supports the follow-up care of patients who have received an islet cell transplant. Since the procedure is considered experimental, medical costs are often not covered by insurance companies. The CDP provides scientific updates to educate patients and families regarding the research and work towards a functional cure. The CDP holds video conferences, yearly events and lab tours with our scientists and researchers for anyone who would like to learn more.
How can individuals help with CDP’s mission?
The CDP welcomes you to join us in many ways to raise money. We have our running team the “Cellmates on the Run” that participates in the New York and Chicago Marathons and the Chicago half marathon. If you would like to run on your own and raise money, you can do that as well. We also have our 101 to Cure Type One challenge in May and a baseball tournament in August. Please check out our fundraising page for more information or reach out to Jill or Patsy with any questions.