The news that Dr. Timothy Kieffer’s team at the University of British Columbia, in collaboration with New Jersey based BetaLogics, has found a faster way to create insulin-producing cells is the latest example of how Canada has been a world leader in fighting type 1 diabetes.
It was Canadian Sir Frederick Banting, working with medical student Charles Best, who discovered insulin in 1922 — a breakthrough that has rescued the lives of millions of diabetics around the world. In the late 1990s, a team of researchers and doctors at the University of Alberta developed the Edmonton Protocol, a procedure for implanting pancreatic islets to treat patients with type 1 diabetes mellitus. However, widespread adoption of the protocol has been limited by the shortage of donor tissue — it can take as many as three donated pancreases for each patient. Also, recipients need to take strong immunosuppressive drugs to prevent rejection of the transplanted cells.
What Dr. Kieffer and his collaborators have come up with — a protocol to turn stem cells into reliable, insulin-producing cells in about six weeks, far quicker than the four months it took using previous methods — represents a significant advance. It brings scientists a step closer to being able to produce an unlimited supply of insulin-producing cells to treat this devastating disease that affects more than 2 million Canadians and almost 400 million people worldwide.
According to a UBC media release, the protocol transforms stem cells into insulin-secreting pancreatic cells, called S7 cells, via a cell-culture method. The conversion is completed after the cells are transplanted into a host. Tested on mice, the transplanted cells were successful in rapidly reversing diabetes.
We asked Dr. Kieffer to answer a few questions about the discovery, the results of which have been published in Nature Biotechnology, which you can read here. Here’s what he had to say:
Question: What’s the key advance here – that you can now make insulin producing cells much more quickly or that you can reverse diabetes so effectively?
Answer: The key advance with our work is the development of culture conditions to extend the maturation of the cells well beyond the pancreatic progenitor stage that we and others have previously achieved. The cells have many characteristics of mature insulin producing beta-cells at the time we transplant them, and thus are able reverse diabetes in about one-quarter the time needed with pancreatic progenitor cells, and with only one-quarter of the cell dose.
Question: You developed the protocol for these S7 cells with mice. How far away are you from human trials?
Answer: The protocol for cultivating the cells was developed with humans in mind, not mice. Therefore, human cells and scalable methods for cell manufacturing were used. Testing the cells in mice with diabetes represents an important and necessary step on the path to clinical trials. It will be up to regulatory agencies such as Health Canada to determine what other studies are required before clinical testing can begin. In this regard it is very encouraging that the FDA recently approved the clinical testing in patients with type 1 diabetes of pancreatic precursor cells produced by ViaCyte.
Question: Do you see a day in the next 10 years when this kind of treatment replaces daily insulin injections?
Answer: I am quite enthusiastic for the potential of a stem cell based therapy for diabetes. The clinical path has been proven with islet transplantation — only a few teaspoons of insulin producing cells (cadaveric islets) are infused into the patients and with this, effective glucose control can be re-established. It is only a matter of time before stem cells provide the needed source of cells to replace insulin injections, and I predict this will be within 10 years.
Question: You’re working with BetaLogics Venture of Janssen Research & Development, LLC on the protocol. Do you have plans to commercialize the protocol as a treatment?
Answer: The work by Dr. Alireza Rezania and colleagues at BetaLogics Venture was instrumental in this research; these are the scientists who significantly advanced the differentiation protocol. The involvement of Janssen greatly increases the chances that this stem cell strategy will develop into a product, with the hopes it will not only treat diabetes, but ultimately cure it.
(Note: This research is supported in part by funding from JDRF, the Canadian Institutes of Health Research Regenerative Medicine and Nanomedicine Initiative, and the Stem Cell Network.)