The Harvard Stem Cell Institute (HSCI), three of Harvard’s clinical affiliates, including Dana-Farber Cancer Institute (DFCI), and a biopharmaceutical company have formed a unique collaboration to establish the Boston Autologous Islet Replacement Program
(BAIRT) to accelerate a cure for diabetes.
Recent advancements in stem cell biology have provided an unprecedented opportunity to treat diabetes. HSCI co-director Doug Melton has developed a process to generate virtually unlimited numbers of beta cells – insulin-producing cells found in the pancreas
– from stem cells in the lab. These stem cell-derived beta cells could be used to replace a person’s beta cells to treat diabetes in the clinic.
The newly announced program, designed by HSCI, Brigham and Women’s Hospital (BWH), and the Joslin Diabetes Center (JDC), working closely with Semma Therapeutics (Semma) and Dana-Farber, will work toward translating these stem cell discoveries into treatments
that could ultimately cure diabetic patients.
Generating clinical-grade, pluripotent stem cells suitable for use in patients will take time: The first trial conducted by BAIRT is expected to be at least three to four years away. The initial clinical trial will enroll a very small, very select group
of patients – individuals who have had their pancreas surgically removed because they had intractable pancreatitis or similar conditions and who have not shown signs of islet autoimmunity (the body attacking its own insulin-producing cells). The initial
trial will test both the safety of the stem cell-derived beta cells and make an initial assessment of their efficacy. The program is intended to uniquely leverage the stem cell and beta cell expertise of the participating hospitals, stem cell science
at Harvard, and the Semma Therapeutics team, as well as the clinical expertise and resources of the participating hospitals to bring a novel therapy rapidly to patients.
During the first phase of the program, DFCI, HSCI, and Semma will work together in developing and optimizing the stem cell derivation protocol. BWH and JDC will work together in screening, characterizing, and identifying suitable patients. In the next
phase, the cell production facility at DFCI will derive stem cells from patients and manufacture the beta cell products using Semma’s differentiation protocol. BWH will perform the transplantation procedure, and patients will be followed by both BWH
While establishing the transplantation center and preparing the cells for the first trial may seem far from the mission of Dana-Farber, in fact, DFCI has been for almost 40 years doing blood stem cell transplants – which the public knows as bone marrow
transplants. And to prepare the cells for those procedures, DFCI established the only GMP – Good Manufacturing Practice – cell production program in Boston.
“The Connell O’Reilly Families Cell Manipulation Core Facility (CMCF) at Dana-Farber has supported the bone marrow stem cell transplant programs for adults and children with our partners
at Brigham and Women’s Hospital and Boston Children’s Hospital for more than 20 years,” said Edward J. Benz Jr., MD, President and CEO of Dana-Farber. “With a highly trained staff who have extensive experience
in the process of cells for individual patients, our CMCF has the unique ability to undertake complex cell processing methods and manufacture cells to meet stringent FDA requirements. The ability to create insulin-secreting cells for individual patients
is truly ground-breaking, and we are excited to be part of this innovative program.”
“No one institution anywhere has the expertise and technical abilities to make this kind of clinical trial possible,” said HSCI Co-Director Doug Melton. “But in the unique Harvard biomedical ecosystem, we are able to bring all the necessary expertise
and infrastructure to bear. I cannot begin to express the gratitude I feel to the leaders of Brigham and Women’s, Joslin Diabetes Center, and Dana-Farber for the eagerness with which they have embraced this project.”
The Brigham and Women’s involvement in the project has grown out of its long history as a leader in transplantation research and surgery. “This project builds on our pioneering work in transplantation and exemplifies the Brigham’s commitment to fostering
discovery and innovation in order to translate research advancements into new therapies and treatments for our patients,” said Betsy Nabel, MD, president of Brigham and Women’s Health Care. “We’re excited to partner with these esteemed institutions
to push the boundaries in improving the care and treatment of patients with diabetes.”
Peter Amenta, President of Joslin, said that “one of our highest priorities at Joslin Diabetes Center has been to bring stem cell-based beta cell replacement therapy to people living with diabetes. We are excited to be working with other Boston leaders
in academia and industry to make this a reality. Joslin has a long history of expertise in beta cell research, immunology, and novel diabetes clinical trials. This will synergize nicely with the complementary strengths of our partners – combining
our efforts is a clear and logical step.”
Robert Millman, CEO of Semma Therapeutics, the company founded by Melton to bring his cells to patients, said that the collaboration is in keeping with Semma’s mission. “Semma is committed to advancing cell therapy for the treatment of diabetes,” Millman
said. “This relationship will provide Semma an additional opportunity to work with world leading institutions in developing clinical applications of stem cell-derived beta cells.”