McGill University Health Centre designated provincial centre for islet transplantation for patients with type 1 diabetes

JDRF congratulates the McGill University Health Centre (MUHC) on its recent mandate to provide islet transplantation for persons with T1D in Quebec. This designation by Quebec’s Ministry of Health and Social Services recognizes the clinical and research teams at MUHC for their leadership and dedication in the field of islet transplantation. As one of only a dozen treatment centres in North America able to perform islet transplantation, this designation also means that the MUHC will be able to improve access to their services and treat more individuals.  

JDRF’s Global Research Strategy focuses on funding research that has a high likelihood of accelerating the development and availability of type 1 diabetes (T1D) treatments, as well as improving the lives of people who live with T1D. Cell therapy treatments in particular hold a lot of potential, as they may become a meaningful treatment option leading towards insulin independence.  JDRF is committed to funding research to make cell therapy safer and more accessible for people with T1D. JDRF is proud to have continually supported the development of cell therapies for decades. Over 20 years ago, JDRF funded both the team lead and the pre-clinical trials that lead to the Edmonton Protocol, which revolutionized the procedure for islet transplantation. Today, JDRF supports many projects that research cell therapies, including continuing research into refining islet transplantation. 

Currently, replacing damaged islets is the only treatment that is clinically proven to be able to achieve insulin independence in people with T1D. Even in cases when complete insulin independence is not achieved, islet transplants can be life changing for people with T1D who experience severe hypoglycemia and hypoglycemia unawareness, for example. The treatment is minimally invasive and much lower risk than pancreas transplantation.  

However, barriers to making islet replacement therapy more efficient and accessible remain. The transplant of donor islets requires recipients to take lifelong immunosuppressants, which can have risks and side effects.  There is also a limited supply of donor islets, which limits the amount of people who can receive this treatment. JDRF researcher Dr. Timothy Kieffer, PhD, is one of several JDRF-funded researchers who is currently examining ways to overcome these limitations through gene editing and stem cell technologies. Other factors, such as poor blood supply to the transplanted islets, can also affect the success of islet transplants. JDRF-funded work in Dr. Michael Sefton’s bioengineering lab is exploring solutions to this issue. Recipients may also need additional infusions of islets in the years after the initial treatment to maintain insulin independence, as transplanted islets may not be a lasting solution.  And so, although ongoing research and trials are promising, many hurdles remain before islet replacement therapy is widely accessible to people with T1D. 

Research at the MUHC facility is also exploring ways to overcome these challenges in islet transplantation. The MUHC team is led by Dr. Steven Paraskevas, a transplant surgeon, researcher, and Professor of Surgery at McGill. In addition to his role as Director of the Human Islet Transplantation Laboratory, he is Director of Pancreas and Islet Transplantation at the Royal Victoria Hospital and former President of the Canadian Society of Transplantation. His research focuses on beta cell injury and survival, as well as the way beta cell injury stimulates the immune system. Understanding beta cell injury is crucial in figuring out how to ensure transplanted islets survive longer and better, with less need for additional transfusions. Such research is essential for advancing knowledge to ensure that opportunities for islet replacement therapy increase in the future, as clinical trials are still at relatively early stages.   

The MUHC is also a site for collaboration, with industry partners as well as with other teams of scientists. The MUHC is currently the site of a clinical trial in conjunction with Vertex Pharmaceuticals. Vertex is conducting a clinical trial of VX-880, a new stem-cell derived therapy for T1D, at several sites in North America (of which the MUHC was the first Canadian site, and Toronto is now recruiting participants as well). The VX-880 product uses islets derived from stem cell cultures rather than from donated pancreases. By using stem-cell derived islets, VX-880 could make islet transplantation and insulin independence available to a greater number of people, if Vertex’s trial program goes on to show safety and efficacy. The clinical trial is still ongoing, but early results out of the US trial sites are promising. The patients who have so far received VX-880 have shown improved blood glucose time-in-range with fewer insulin injections.  

The MUHC further supports research by housing the McGill University Health Centre Human Islet Transplant Laboratory, which purifies islet cells from donated pancreases. These cells can then be transplanted into patients as well as being used in research to further our understanding of islet biology and the pathogenesis of T1D. The MUHC’s designation as a provincial centre for islet transplantation will help to ensure that cell therapy treatments will continue to develop.  

The Vertex trial is currently recruiting participants with T1D for their trial in Montreal and in Toronto. To see if you qualify, click here.  

Exciting news: new Health Canada approved clinical trials

Health Canada has approved clinical trials that could lead the way for cell replacement therapy that does not require immune suppression in people with type 1 diabetes.

JDRF is the leading charitable funder of research into type 1 diabetes in Canada, with a focus on research that will help improve lives today, and lead to disease prevention and cures.

February, 2022 – As the leading charitable funder of type 1 diabetes (T1D) research in Canada, a key focus of JDRF’s strategy is investment in stem cell therapy research for potential T1D cures.

In November, 2021 Health Canada approved clinical trials for ViaCyte, a cell replacement company long supported by JDRF, and CRISPR Therapeutics for a VCTX210, a gene-edited cell replacement therapy for type 1 diabetes (T1D) that doesn’t require immunosuppression.

On February 2, 2022, the companies announced that the first patient was dosed in the phase I study, which will assess the therapy’s safety, tolerability, and immune evasion.

JDRF is very excited about the progress of this clinical trial, and will provide further updates as they become available

On November 16, 2021, ViaCyte, a cell replacement company long supported by JDRF, and CRISPR Therapeutics announced that they will begin clinical trials for a gene-edited cell replacement therapy for type 1 diabetes (T1D) by the end of the year in Canada.

What is cell replacement therapy?

A significant focus of cure-based T1D research is investigating stem cell-based therapy. Researchers look for ways to use stem cells as a renewable source of insulin-producing cells which, when transplanted, would replace the beta cells that are destroyed in a person with T1D, allowing them to produce insulin again. This would lessen or eliminate the amount of external insulin required by someone living with T1D (either by injection, pen, or pump) for months or even decades.

The biggest challenges to stem cell replacement therapy are identifying the appropriate stem cell source (i.e., pancreatic cells, or liver cells) and ensuring that they both function well and will not be rejected by the recipient’s immune system. Much like a transplanted organ – most stem cell replacement therapies require immunosuppressing medications to prevent rejection.

What is different about the ViaCyte and CRISPR therapy?

By the end of the year, Viacyte will start a clinical trial of VCTX210, a gene-edited stem cell replacement therapy for T1D. Combining ViaCyte’s stem cell expertise with CRISPR Therapeutics’ pre-eminent gene-editing platform offers significant potential in the development of a cell replacement therapy that does not require immune suppression.

The phase I clinical trial will begin this year in Canada, and will evaluate the safety, efficacy, and immune evasiveness of the therapy, and will be the first time a gene-edited cell replacement therapy will be tested in people with T1D.

ViaCyte has previously demonstrated that their stem cell-derived beta cells, (which JDRF globally funded the development of), make insulin when transplanted into people with T1D. Now, CRISPR has applied their gene-editing technology to make these cells immune-evasive – meaning that they should not require immunosuppression to prevent rejection – a huge development in making the therapy more universally accessible to people with T1D.

What is JDRF’s role?

JDRF globally has been a long-time and significant supporter of ViaCyte, supporting the company through research funding, including funding 15 years ago (when ViaCyte was called CyThera) that underwrote development of the proprietary line of precursor stem cells used in their treatment.

This clinical trial is one of several potential beta cell replacement cures therapies JDRF is currently funding or supporting.

What does this mean for Canadians with T1D?

JDRF will be closely monitoring the results of the phase 1 clinical trial and will report back on results as they become available.

Exciting news: stem cell-based treatments producing insulin in participants with type 1 diabetes

*Image taken from viacyte.com/pipeline/

A significant focus of JDRF is funding the most promising research that could lead to potential cure therapies for type 1 diabetes (T1D).

ViaCyte, a cell replacement company long supported by JDRF, has three separate stem cell replacement therapy products in development, all with the aim of reducing or eliminating the need for external insulin for people with T1D.

Researchers look for ways to use stem cells as a renewable source of insulin-producing cells which, when transplanted, would replace the beta cells that are destroyed in a person with T1D, allowing them to produce insulin again. This would lessen or eliminate the amount of external insulin required by someone living with T1D (either by injection, pen, or pump) for months or even decades.

The biggest challenges to stem cell replacement therapy are identifying the appropriate stem cell source (i.e., pancreatic cells, or liver cells) and ensuring that they both function well and will not be rejected by the recipient’s immune system. Much like a transplanted organ – most stem cell replacement therapies require immunosuppressing medications to prevent rejection.

Updates on ViaCyte Clinical Trials

PEC-Encap (VC-01™; see middle figure) was ViaCyte’s first product including a stem-cell derived precursor that once implanted in humans has demonstrated that they will mature into beta cells that produce insulin.

The company’s second technology, PEC Direct (VC-02™; see top figure), has now been the subject of two papers that report the preliminary results of its clinical study, which involved an international team of clinicians and researchers including several at the University of Alberta and at the University of British Columbia’s Faculty of Medicine and Vancouver Coastal Health (VCH).

The UBC-VCH study is part of larger international clinical trials led by ViaCyte, which is also studying the effectiveness of cell-replacement therapy on participants in sites across Canada, the U.S. and Belgium.

One of the new papers reports on 15 trial participants studied at the Vancouver site. At the start of the UBC-VCH study, each participant had several ViaCyte cell-containing devices implanted just below the skin. Each device, thin as a credit card, contained millions of lab-grown cells that originally came from a single stem cell line. These cells had been trained to mature into insulin-producing beta cells.

Six months after implantation, the cells had not only survived but successfully matured into insulin-producing beta cells, which helped the trial participants to sense blood sugar levels and release insulin when needed.

This study used levels of C-peptide (released into the blood as a byproduct of insulin production) to measure insulin produced by the implanted cells. The researchers found C-peptide levels rose after patients ate a meal—evidence of normal beta cell function. This is significant as external insulin (either received via injection, pump or pen) does not generate C-peptide.

Participants also spent 13 per cent more time in target blood sugar range and some were able to reduce the amount of insulin they injected.

The insulin produced through the implanted cells was not enough for participants to forgo external insulin altogether, but the cells did survive and maintain function a full year post-transplantation. This demonstrates the potential for durability of this kind of cell replacement therapy, and may ideally prevent frequent implantation, which must be done surgically. Importantly, the trial did not reveal any safety concerns.

This research was supported by funding from JDRF Canada, as well as Canada’s Stem Cell Network, Vancouver Coastal Health Research Institute, Canadian Institutes of Health Research, ViaCyte Inc. and California Institute for Regenerative Medicine.

What does this mean for people with T1D?

These studies offer hope and cautious optimism about cell replacement therapy as a possible pathway to a T1D cure.

“Our findings demonstrate the incredible potential of this stem cell-based treatment. With further research, this treatment could one day eliminate dependence on insulin injections and transform the management of Type 1 diabetes,” said the study’s senior author Dr. Timothy Kieffer, professor in UBC faculty of medicine’s departments of surgery and cellular and physiological sciences, who was recently appointed as ViaCyte’s chief scientific officer.  

The next step is researchers need to determine what cells are optimal for transplantation, and the best transplantation site. There also needs to be further study on how long the cells work effectively, remain safe, whether ta greater number of cells is required for long-term insulin production, and whether it is possible to eliminate immunosuppressive therapy.

A third ViaCyte cell replacement product including a gene-edited stem cell source, VCTX210, developed in partnership with CRISPR Technologies, was recently approved for clinical trial by Health Canada. Unlike the PEC-Direct product used in the newly published results, this product would not require immunosuppression – a key next step on the cell replacement roadmap. (See Figure 3)

Read more here: jdrf.ca/exciting-news-about-new-health-canada-approved-clinical-trials/

The ViaCyte clinical trials are one of several potential cell replacement cures therapies JDRF supports globally, as part of its overall research strategy.  Read more about it here: jdrf.ca/research/

Nancy Tout’s Story

When Nancy Tout pursued her passion for science with a PhD in Immunology, she didn’t realize just how much her work and personal life would intersect. Her son Alex was two years old when Nancy and her husband noticed his insatiable thirst and frequent urination. Though Nancy had studied autoimmune diseases and knew the signs and symptoms of type 1 diabetes (T1D), she didn’t suspect her son was living with the condition. But a trip to the doctor revealed his diagnosis, changing their lives forever.

“He was a toddler and therefore couldn’t articulate how he felt, so there was a lot of guesswork and calculation,” Nancy recalls. “I would weigh all his food and always had a calculator on hand to determine a safe range of carbs. I remember him looking up at us when we had to hold him steady to give him the needles. He was such a trooper.”

Shortly after Alex’s diagnosis, the Tout family reached out to JDRF. This was the start of a nearly 20-year volunteer relationship that has flourished thanks to the family’s incredible commitment to JDRF’s mission. This has included involvement in our Walk to Cure Diabetes, lobbying the government for device coverage, and supporting JDRF research.

“We jumped in with both feet and I think we’ve done probably every volunteer gig with JDRF,” Nancy says. “JDRF’s mission to treat, prevent and cure type 1 diabetes through research really resonated with me.”

“A cure just takes one breakthrough in the lab”

Now in his twenties, Alex is living provinces away and managing his diabetes thanks to advances in technology since his diagnosis – many made possible by JDRF-supported research and advocacy efforts, and by Nancy and her family.

When Nancy and her husband uprooted from Guelph and moved to Edmonton in 2019, she made one call right away. Within a month, she was touring Dr. James Shapiro’s lab, where he works tirelessly on stem cell research into a cure for T1D.

“A cure just takes one breakthrough in the lab,” Nancy says. “And to have Dr. Shapiro open his doors to me and show me the place where the Edmonton protocol was born was really nice.”

Nancy is most excited about JDRF’s beta cell replacement research, which she believes will lead to a cure in her son’s lifetime. Until that cure is found, she is happy to support research that improves lives today, like the artificial pancreas that greatly reduces the constant burden of checking blood sugar and dosing with insulin. Her son uses the world’s first hybrid closed-loop insulin delivery systems and this helps ease Nancy’s mind as Alex begins a new chapter with a demanding but rewarding career that includes shiftwork.

Her desire to affect change both now and in the future is why she gives to JDRF in several strategic ways. She donates annually to ongoing research initiatives, and she has also made a gift in her Will.

“When we sat down with our financial planner and were asked: ‘What are the most important things to you?’ JDRF was at the top of the list,” Nancy says. “JDRF has changed our lives forever and we want to ensure they can change others’ lives for years to come.”

Nancy believes that making a gift in her Will now as opposed to in her retirement years is a smart way to ensure that everything is taken care of well in advance. With her two children nearly finished their post-secondary education and financially independent, she felt it was time.

She also recognizes that others are not as fortunate as her family. Having in-depth knowledge of autoimmune diseases and access to good health care and technology helped her son navigate living with the disease and avoid the complications that can be all too common with T1D. Nancy and her family want nothing more than for T1D research to make life easier for everyone with T1D today – and lead to an eventual cure.

“Leaving this gift in our Will was a no-brainer for us. We hope that other families supported by JDRF consider the impact they can make by doing the same. The money is going to be well-spent, and that makes me proud and happy to be part of.”

To learn more, please fill out a request for information, or contact:

Leanne Scott
National Manager, Annual Giving
1.877.287.3533
[email protected]
Charitable Number: 111897 6604 RR0001

Manipulating the Microbiome to Prevent Type 1 Diabetes

Type 1 diabetes is an autoimmune disease resulting in a lifelong dependency on insulin treatment and a risk of serious long-term complications. The cause of the disease is unclear, although both genes and factors in the environment seem to be important. As new cases of this burdensome and costly disease are on the rise in almost every country around the world, finding ways to prevent and better treat type 1 diabetes are top research priorities.

In the past decade, research has shown that changes in the gut microbiome – the microbial community that colonizes the intestine – precede the onset of type 1 diabetes in young children. Most of these studies highlight a potential link in the first years of life when the microbiome is highly dynamic and heavily influenced by environmental factors such as mode of birth delivery, diet, infections, and antibiotic use. JDRF and other funders have invested heavily in this area, supporting studies such as the DIABIMMUNE Study, BABYDIET, and TEDDY, which all helped to point researchers towards new therapeutic opportunities. In addition, for many years, JDRF led an international consortium that brought researchers working in this area together on a regular basis to share ideas, compare data, and accelerate progress.

The field is now focused on testing whether manipulation of the microbiome with drugs or diet can delay or even prevent the disease altogether. Other studies are attempting to figure out how changes in the gut microbiome could signal a person is at risk of developing type 1 diabetes.

Dr. Jayne Danska, a professor at the University of Toronto Faculty of Medicine and a senior scientist at The Hospital for Sick Children, is working at the cutting edge of this field. In late 2019, she was awarded a $2 million CAD team grant from JDRF and the Canadian Institutes for Health Research (CIHR) to advance her work studying the role of the gut microbiome in altering risk and progression of type 1 diabetes. She and her team previously showed that deliberate manipulations of the gut microbiome can inhibit autoimmune responses in an experimental model of type 1 diabetes. The new five-year project seeks to understand how environmental changes influence the gut microbiome in infants with genetic risk for type 1 diabetes. The team will attempt to define exactly what risk-associated microbes do inside a developing infant, and how they affect the immune response to either protect against or potentiate the disease. The ultimate objective of Dr. Danska’s work is to identify new therapeutics to prevent type 1 diabetes.

We are facing a moment in time that can forever change the landscape of type 1 diabetes research, with better treatments and cures on the horizon. JDRF is striving to speed up research progress in any way we can. We are constantly scouting for the best ideas, the brightest talent and the innovative project that will bring us the next real breakthrough.

In partnership with Media Planet