Last week’s Breakthrough T1D Beta Cell Replacement Consortium enabled 57 researchers the chance to collaborate in ways that help move research forward more quickly and efficiently. This crucial network helps scientists join forces to address gaps in research, complete clinical translation, and solve technical and operational challenges to accelerate life-changing therapies for people living with type 1 diabetes (T1D).
Perhaps the most crucial way that Breakthrough T1D provides resources for beta cell replacement researchers is by creating the consortium itself—a safe place to meet people and talk about successes and failures in the field. Discussing both can save time toward possible cures and donor money by avoiding duplication of ideas that didn’t work.
Qizhi Tang, Ph.D., Professor and Director, Transplantation Research Lab, University of California, San Francisco, says that quite often researchers are working on the same problem, and then at the consortium someone might have a solution to share. Getting the chance to see unpublished data and form collaborations can quickly move the field forward faster. “The consortium is a very nice mechanism to coordinate effort, avoid duplication and create faster synergy in the field.”
Tang and co-workers identified the benefits of co-transplanting human islets with parathyroid gland. This work was partially supported by an Innovative grant from Breakthrough T1D. She says she realized 10 years ago that the major problem in beta cell replacement was graft survival. Recently, the team had some breakthroughs. “We can move the graft survival from 10% to 90% survival,” said Tang. That is major improvement in function.
It’s a new concept that cells can be drugs and they are smart drugs.
Another avenue Tang is exploring is to protect transplanted islets using the recipient’s own immune system, specifically the T cells that regulate immune response. This T cell-based therapy is worth doing for several reasons. First, as Tang points out, the T cells themselves have evolved over billions of years to perform their function. It’s very hard to mimic that function. Second, these cells are long-lived. And third, they have the ability to influence other cells to induce a state of infectious tolerance. “It’s a new concept that cells can be drugs and they are smart drugs,” says Tang. Clearly, thinking outside the box is what Breakthrough T1D’s beta cell consortium is all about.
Another way that Breakthrough T1D helps researchers is by interacting with organizations such as the California Institute for Regenerative Medicine (CIRM). Lila Collins, Ph.D., CIRM Associate Director, Therapeutics, says that CIRM has funded over 1,000 projects at 70 unique institutions in California, making it a center of global stem cell expertise. “We’ve funded the creation of a number of enabling tools for stem cell research, including clinically appropriate and scalable manufacturing processes that we’re really going to need to bring these stem cell therapies to the clinic and to commercialization.”
The institute has funded four T1D clinical trials. One trial has demonstrated the very first production of glucose-responsive insulin in people from a human embryonic stem-cell derived product. As if that exciting news weren’t enough, CIRM is also funding a clinical study to see whether co-transplanting donor human islets with human parathyroid glands will improve the success of those islet transplants. “We think that this really could be a paradigm-shifting trial. It may be able to get us from a situation where we need to transplant islets from multiple donors to a point where we could have a single donor islet transplant. That’s important because it can help access for patients,” said Collins.
CIRM sees the value of interacting with Breakthrough T1D. They are thankful that Breakthrough T1D can help locate T1D representatives to work with the CIRM teams on clinical advisory panels. Collins explains how important it is to develop a trial that works not only for the researchers, but for people with T1D, as well. “Nobody knows what patients need better than patients, and nobody knows what it’s like to participate in a trial like a patient.”
To review the Spring 2019 Breakthrough T1D Beta Cell Replacement Consortium, see here.
