In a step that could lead to a new diabetes treatment, several Boston-area hospitals have teamed up with the Harvard Stem Cell Institute and biotech Semma Therapeutics to make personalized cell-based therapies and organize clinical trials.
It is one of several initiatives around the country aimed at manufacturing cell-derived treatments, as the hope for such therapies creates a demand for the production of the cells.
In patients with type 1 diabetes, the immune system attacks the pancreatic cells that produce insulin, called beta cells. Doctors have for years transplanted beta cells from dead donors into patients, but the supply is insufficient for the millions of people with type 1 diabetes, and patients’ bodies sometimes reject the donor cells.
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The new program, officially called the Boston Autologous Islet Replacement Program, could provide enough high-quality beta cells needed to treat patients in upcoming clinical trials. And the cells that will be transplanted into these volunteers will be derived from their own blood cells, cutting the risk of rejection.
For the first trial, which won’t start for at least three years, researchers intend to transplant the cells into people who have had their pancreas removed because of pancreatitis or another condition. If the treatment proves effective and safe, doctors could conduct another study with diabetic patients.
As part of the collaboration, clinicians at Brigham and Women’s Hospital and the Joslin Diabetes Center will recruit patients. Experts at the Dana-Farber Cancer Institute will derive the stem cells from the patients’ own blood cells — creating what are called induced pluripotent stem cells — and coax them into becoming beta cells. Doctors at the Brigham will then perform the transplant, with experts there and at Joslin monitoring the patients and their progress.
The program relies on research conducted by Douglas Melton, codirector of the Harvard Stem Cell Institute, who pioneered the idea of converting stem cells into beta cells. Melton is also the scientific founder of Semma.
“We’ve reached a point with the technology where we can legitimately start thinking about cell replacement,” said Dr. Richard Lee, a Brigham cardiologist and researcher at the stem cell institute who helped wrangle the different institutions to form the program.
The Harvard collaboration represents just one cell production effort as researchers and patients eagerly try to move treatments derived from induced pluripotent stem cells, or iPS cells, into clinical trials — and to get them to patients. In addition to offering a patient-specific treatment, iPS cells avoid the ethical issues that come with conducting research or developing therapies with embryonic stem cells.
The National Cell Manufacturing Consortium, for example, on Monday announced its own plan for manufacturing a variety of cells for treatments, including iPS cells. The consortium is an initiative led by the Georgia Research Alliance and Georgia Institute of Technology and includes academic institutions, companies, and government agencies.
Organizers of the Boston program still need to secure more funding if it is going to be successful. The stem cell institute has committed to funding the program for the first year, but Lee and other Harvard officials would not say how much the institute is contributing, or how much they think the program will cost to sustain.
“There’s a need, but there’s a big risk when you set up one of these,” said Dr. Mahendra Rao, the vice president for regenerative medicine at the New York Stem Cell Foundation, who is not involved with the Harvard project.
Maintaining a cell production facility that meets manufacturing standards can alone be very expensive, Rao said. What helps in the Harvard case is that Dana-Farber has a history of producing cell transplant therapies to treat certain cancers, so it meets what are known as “current good manufacturing practices” for these types of therapies.
But developing the treatments for the patients in the trials and the trials themselves will take more funding than the program has so far identified.
“It’s going to be very expensive,” Lee said. “The big challenge is, can the strategy of using an individual’s stem cells come down to a price that’s really feasible?”
The effort to bring iPS cell treatments to patients has been moving forward since the cells were discovered a decade ago, but it hasn’t always been a smooth road.
In 2014, a patient in Japan with macular degeneration became the first person treated with a treatment derived from iPS cells. That study was later put on hold because of problems with the cells meant for a second patient, although Japanese media reported recently that researchers are ready to restart the work.
Also in 2014, Rao resigned as the director of the National Institutes of Health’s Center for Regenerative Medicine because the NIH provided funding to fewer projects than he had hoped. The NIH shuttered the center then, but in September, it created a new Stem Cell Translation Laboratory to promote therapies based on stem cells.
Even with these hurdles, it’s just a matter of time before scientists get the first clinical trial of an iPS cell treatment up and running in the United States. One project that officials say could reach that stage as soon as next year is an effort led by Kapil Bharti at the National Eye Institute, an arm of the NIH, which would use stem cells derived from skin cells to rebuild eye tissue that is damaged in several diseases that cause blindness.