Novel Stem-Cell Therapy for a Type 1 Diabetes Cure Seeks FDA Approval for Clinical Trials
By Adam Brown
Twitter Summary: ViaCyte seeks approval from FDA for first human studies of novel encapsulation stem-cell therapy for #t1D
On July 17, ViaCyte filed applications with the FDA to conduct the first human studies of VC-01, a novel cell replacement therapy for type 1 diabetes. The approach takes pancreatic progenitor (parent) cells and encases them in ViaCyte’s “Encaptra” device (to protect them from immune attack), which is roughly 1 x 3 inches in size. Encaptra is then implanted under the skin, allowing the cells to mature into insulin-producing beta cells. In theory, they will mature to regulate blood glucose in a similar (if not identical) manner to natural pancreatic cells. After implantation, these cells can be easily monitored and readily removed in case of an emergency.
Pending FDA approval of the applications, the early phase 1/2 clinical trial of VC-01 will enroll about 40 type 1 participants over a two-year period. The trial will primarily measure VC-01’s effect on C-peptide, a biomarker for insulin production. If all goes well, ViaCyte hopes to bring this therapy to market by 2020. However, there are several major questions that ViaCyte will need to answer from this early-stage study – Could VC-01 cause tumor growth? How glucose-sensitive will the implanted cells be? How many cells should be implanted for optimal results, and for how long can they be implanted? Will the cells also be able to affect glucagon levels?
While the start of clinical trials are still pending FDA approval, it’s exciting to see a new therapy for type 1 diabetes in the works, as there have been a number of disappointments in the last few years on the cure front. The work of ViaCyte has been supported largely by JDRF, and this makes it the first company projected to bring a cell replacement therapy for type 1 diabetes into human testing. –AJW/AB
*Update: on August 19, ViaCyte announced that the FDA had approved its request to start a phase 1/2 clinical trial for VC-01.