Annual American Diabetes Association Conference provides updates on exciting developments in type 1 diabetes research

Annual American Diabetes Association Conference

The American Diabetes Assocation’s 83rd Scientific Sessions were held from June 23- 26th. This annual conference brings together researchers and scientists to both present and learn about the latest in type 1 diabetes research and technological advancements. Many of the presenters are funded by JDRF International, JDRF Canada’s affiliate in the United States.

JDRF-funded researchers presented new study results that will improve outcomes for people with diabetes (T1D). Chief Scientific Officer, Dr. Sarah Linklater, was in attendance, along with many Canadian researchers that JDRF donors generously help to fund through the $100 M Campaign to Accelerate. An impressive program was presented including breakthrough clinical trials and significant research studies that are paving the way to novel treatments and technologies for T1D.

You can view all the oral and poster presentations on the Diabetes Journal website.

Updates in Cure-based Research:
  • An update on Vertex’s clinical trial to test VX-880, a stem cell-derived replacement therapy for diabetes, was presented by Trevor Reichman, M.D., Ph.D. (University of Toronto). Data from 6 participants was presented, and the two with more than a year of follow-up no longer need to administer insulin through injections or pump therapy and exceeded the recommended time-in-range for blood glucose. Vertex’s phase I/II clinical trial of VX-880 was made possible by Doug Melton’s years of JDRF-funded research and a catalytic investment from the T1D Fund in Semma Therapeutics—a biotech company founded by Melton to develop a stem cell-derived islet therapy for T1D—which was acquired by Vertex Pharmaceuticals in 2019.
  • Vertex is also now recruiting patients in Edmonton for their VX-264 therapy. This treatment will use the same cell therapy as VX-880 but encapsulate the cells within a device designed to shield the cells from the body’s immune system, meaning immunosuppression should not be required.
  • In Sernova’s ongoing Phase 1/2 clinical trial of their Cell Pouch System™ – a novel implantable and scalable medical device that forms a natural environment in the body for the housing and long-term survival and function of therapeutic cells – the first five patients to receive the encapsulated donor islet transplants achieved insulin-independence for ongoing periods of six to 38 months. Sernova is a Canadian biotech company headquartered in London, Ontario. 
  • Dr. Harald Stover, CEO of Allarta Life Sciences Inc (Hamilton, Ontario) presented an update on their work using hydrogel microencapsulation for immunoprotective islet replacement therapy. 
  • The clinicians and scientists at University of Alberta presented a 20-year follow up on renal function after islet transplant with whole-body immunosuppression. Highlighting the importance of effective and tailored immunosuppression regimens, and the ultimate goal to reduce or eliminate the need for immunosuppression with transplantation.
  • Dr. Cristina Nostro, whose work is closely tied and complementary to these trials, shared her excitement in stem cell research, recapping the day’s sessions by saying: “In the last 20 years, we’ve learned how to differentiate these cells, and now we’re moving them to the clinic and they’re giving us the results that we want. The future is bright. I’m super excited, and I hope you are too.”
Updates in Disease-modifying Therapies
  • JDRF-funded researchers Halis Akturk, M.D., Martin Thelin, M.D., Ph.D., and Edwin Liu, M.D., presented on the relationship between T1D and other diseases, highlighting how other diseases can be a resource for better understanding and managing T1D.
  • Evaluation of a disease-modifying therapy that may delay or prevent the disease, was presented by Farooq Syed, Ph.D., a JDRF career development awardee.\
Updates in Improving Lives:
  • Researchers and companies at the conference also reported exciting updates to diabetes devices that are now getting smaller, more coordinated, and more automated while improving glucose control and easing the burden of diabetes management. 
  • The team behind the BETTER Project in Quebec led by Drs. Anne-Sophie Brazeau and Remi Rhabasa-Lhoret presented multiple research findings including information on clinical characteristics of LADA (latent autoimmune diabetes of the adult), a comparison between DIY and commercialized automated insulin delivery systems, the use of oral glucose at higher thresholds to prevent mild hypoglycemia, the addition of glucagon to insulin administration to reduce post-meal hypoglycemia, and a comparison of injectable versus intranasal glucagon administration.
  • A randomized clinical trial, presented by JDRF grantee Schafer Boeder, M.D., showed that a medication that helps lower blood sugar (SGLT) plus a glucagon receptor treatment improved blood-sugar control and reduced insulin dose, and there was no diabetic ketoacidosis (DKA)—a risk when just taking an SGLT treatment for T1D.
  • Zucara Therapeutics (a Toronto-based company supported by JDRF) presented the positive results of their Phase 1 trial of ZT-01, a novel therapy to prevent hypoglycemia. Following the success of phase 1, Zucara has recently begun Phase 2 clinical trials to evaluate the efficacy of ZT-01 to prevent nighttime hypoglycemia.
  • Dr. Tricia Tang chaired a session that covered research and initiatives that address the psychosocial aspects of T1D. Dr. Tang also reported in her own talk that through her ongoing trial of the peer support app T1DReachout, she’s learned that individuals require peer support that is choice-based, customizable, and “just in time,” meaning peer support provides an emotional lifeline when people need it most.
Updates in Screening
  • There was a lot of success in T1D screening presented at ADA, including a JDRF-led symposium on the identification and prevention of T1D.  These successes will help inform the CIHR-JDRF Screening Research Consortium, being announced on July 10th. This consortium will investigate the best ways to implement a Canada-wide, universal screening program integrated with the Canadian healthcare system.

To learn more about the sessions, please find the recaps for Day 1, Day 2, and Day 3 of the conference.

CanScreenT1D: Screening Research Consortium in Canada Announced

JDRF Canada, in collaboration with CIHR, is thrilled to announce the recipients of the CIHR-JDRF Type 1 Diabetes Screening Research Consortium. This $12 million grant will develop a single nationally coordinated research network to explore key research questions about the feasibility and acceptability of general population screening for early-stage T1D in Canada. The consortium will build on experiences from other countries with T1D screening programs including the US, UK, Israel, Australia, and multiple European countries. 

Most T1D screening studies have focused only on family members, who are at higher risk of T1D than the general population. However, as 90% of people diagnosed with T1D do not have any family history, family-based screening does not identify most people in the population who go on to develop T1D. This new funding opportunity looks to address this gap and will help to determine approaches for identification of Canadians with early-stage T1D who could benefit from education, monitoring and – in the future – therapies that could delay or even prevent the need for insulin therapy.  As well, it will help advance research into potential disease-modifying therapies that could be applied when an individual is identified as high risk and could delay or prevent the need for insulin therapy. 

JDRF is pleased to announce that Dr. Diane Wherrett (Toronto, ON) will lead CanScreenT1D – the Canada-wide T1D Screening Research Consortium team.

Dr. Wherrett is a physician in the Division of Endocrinology, Department of Paediatrics at The Hospital for Sick Children (SickKids) and a professor at the University of Toronto. She is the Canadian Centre Director for T1D TrialNet (an international research network that screens relatives of people with T1D and leads clinical trials of preventative therapies). The new Canadian consortium is made up of over 30 members including academic and clinician researchers, endocrinologists, people with lived experience of T1D, and knowledge users including a diabetes nurse, genetics counsellor, and a Ministry of Health representative. The acceptability of T1D screening in Indigenous communities will be explored, as led by Sasha Delorme of Diabetes Action Canada’s Indigenous Patient Circle and Indigenous people with lived experience of diabetes.

CanScreenT1D team leads are: 

  • Dr. Pranesh Chakraborty, Children’s Hospital of Eastern Ontario (CHEO) 
  • Dr. Robin Hayeems, SickKids 
  • Dr. Monika Kastner, University of Toronto 
  • Dr. Audrey L’Espérance, École Nationale d’administration publique 
  • Dr. Despoina Manousaki, Hôpital Sainte Justine 
  • Dr. Ashish Marwaha, Alberta Children’s Hospital Research Institute 
  • Dr. Jon McGavock, Children’s Hospital Research Institute of Manitoba 
  • Dr. Peter Senior, University of Alberta 
  • Dr. Albert Tsui, University of Alberta 
  • Dr. Bruce Verchere, BC Children’s Hospital Research Institute 
  • Dr. Holly Witteman, Université Laval
  • Conrad Pow, North York General Hospital, Diabetes Action Canada
  • Sasha Delorme, Diabetes Action Canada

CanScreenT1D will study different screening approaches, as well as the effectiveness of education and follow-up of people with early-stage T1D. CanScreenT1D will explore how general population screening for early-stage T1D could be carried out in Canadian health care systems, and conduct pilot studies of approaches to inform future implementation across Canada.  

JDRF will work closely with the Screening Research Consortium to ensure that any opportunities for public participation in research consultation or patient engagement are distributed to our community.  The pilot screening program is estimated to start in Fall of 2024.

The Importance of Screening

General population screening offers the potential to identify people who have early-stage, pre-symptomatic T1D. Canada has one of the fastest growing rates of T1D diagnoses anywhere in the world – and we don’t know why.

“Thanks to our team of researchers and patient partners across Canada, we are creating a pilot screening program to help identify children at risk of type 1 diabetes, aligned with the values and preferences of Canadians. With earlier diagnosis and connections with ongoing research initiatives, we can hopefully prevent serious complications at the time of diagnosis and increase access to treatments that may delay or prevent type 1 diabetes”  – Dr. Wherrett

JDRF-funded research previously discovered that the presence of two or more specific markers indicative of an autoimmune response to the pancreas – called autoantibodies – indicates that a person is almost 100% likely to develop T1D in their lifetime. Screening provides the opportunity to educate those with early-stage disease about the signs and symptoms of T1D and provide supportive follow-up, preventing the life-threatening complication diabetic ketoacidosis (DKA) at diagnosis. With the FDA approval of Tzield, the first ever disease-modifying therapy for T1D, for people with early-stage disease, screening offers the opportunity to delay the onset of T1D diagnosis and further research into more disease-modifying therapies.

The prevailing medical wisdom used to be that T1D developed quickly, with a sudden onset of symptoms including thirst, hunger, increased urination, weight loss, and fatigue. Thanks to advances in screening and a better understanding of the human immune system, we now know that T1D does not develop suddenly but in fact the disease process usually starts long before insulin is required.  

Once the immune system begins to attack the insulin-producing cells in the pancreas, we can detect markers in the blood (autoantibodies) that tell us a person is at increased risk. This is because the disease is otherwise asymptomatic or silent earlier on.

T1D happens in 3 stages:

How to Detect T1D in 3 stages

Because most people do not have a family history of T1D, symptoms and a diagnosis often come out of the blue. In 25-45% of diagnoses in children in Canada, this unexpected diagnosis comes with DKA, a serious and life-threatening complication that can lead to death if not treated promptly. An important part of a screening program will be follow-up monitoring for those who screen positive for T1D autoantibodies, to lower the risk for life-threatening DKA at diagnosis and serious complications, and accelerate the evaluation of disease-modifying therapies that could delay or prevent the disease.  

A key goal of JDRF’s global research strategy is to support research that enables introduction of general population screening to identify high-risk individuals for early detection, reduce DKA at diagnosis, and accelerate the evaluation of disease-modifying therapies that could delay or prevent the disease. 

JDRF has numerous research studies examining the efficacy of potential disease-modifying therapies for T1D. But many of these therapies will work best during stage 1 and 2 T1D, which can only be identified via a screening program. Stopping T1D before it starts is the ultimate goal, and a universal screening program will be essential to prevent new diagnoses of this disease in the future.

Current Screening Options in Canada

Currently, only family members of people with T1D can be screened for T1D risk through the TrialNet research program. TrialNet is an international network of leaders in T1D research and clinical care with centers in the United States and internationally. 

We strongly encourage you to consult with your or your child’s physician for input as you make decisions about screening for T1D risk. Considering various sources of expert guidance and that from one’s own physician is the best way to make personal health choices.  

Exciting updates from Vertex stem cell-based therapy clinical trials

A large area of cure-based T1D research is investigating stem cell-based therapy. The goal of this approach is to use stem cells as a renewable source of insulin-producing cells which, when transplanted, would replace beta cells that are destroyed in a person with T1D, thereby 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.

In February 2021, Vertex announced the launch of a clinical trial for VX-880, a stem cell-derived therapy people with T1D. VX-880 is delivered via infusion into the hepatic portal vein (liver) and requires the use of chronic immunosuppressive therapy to protect the cells from rejection or immune attack.

UPDATE – June 2023

VX-880 Clinical Trial Phase 1/2 Part B:

Six patients have received full doses of VX-880 at staggered times over the past year and a half. Prior to treatment, all patients had undetectable fasting C-peptide (i.e., no self-secreted insulin, or insulin produced by the body), a history of recurrent severe hypoglycemic events in the year prior to treatment and required an average of 34.0 units of insulin per day.

Following treatment, all six patients are self-secreting insulin, improved HbA1c levels, improved time-in-range on continuous glucose monitoring, and reduction or elimination of exogenous insulin use (i.e., externally administered insulin either by pen, pump or multiple daily injection). Patients with greater than 90 days of follow-up also had elimination of severe hypoglycemic events. Two of the six patients are at least 12 months post-treatment and are currently insulin independent with “normal” HbA1c levels (≤6.0%) and time-in-range levels over 95%.

VX-880 has been well tolerated with only mild-moderate adverse events such as: dehydration, diarrhea, hypomagnesemia and rash.

Based on the result of these safety and efficacy data in Part B, the independent data review committee has recommended moving to Part C of the trial, which allows for concurrent dosing of patients at the full target dose of VX-880. Approximately 10 participants will be enrolled in this stage of the trial in Edmonton, Montreal, Toronto, and Vancouver. For more information, please see or Vertex’s website.

VX-264 Clinical Trial Phase 1/2:

This treatment will use the same cell therapy as VX-880, but encapsulate the cells within a device designed to shield the cells from the body’s immune system. Therefore, immunosuppression is not expected to be required. Recruitment is currently ongoing in Edmonton. For more information please see or Vertex’s website.

Vertex partners with Lonza (Switzerland) to build a dedicated manufacturing facility for T1D cell therapies

Vertex and Lonza will partner in the process development and scale-up for the manufacturing of the VX-880 and VX-264 product portfolio and co-invest to build a dedicated new facility in Portsmouth, New Hampshire. Operated by Lonza, the facility will span more than 130,000 square feet and is anticipated to create up to 300 new jobs at peak capacity. Construction is scheduled to begin later this year. For more information, please see the full press release here.

VX-880 Clinical Trial Phase 1/2 Part A:

On October 18, 2021, the company announced that the first trial participant to receive VX-880 now needs 91% less insulin 90 days after receiving an infusion of these stem cells – and at just half the target dose.

The success seen with just half the target dose is exciting as it suggests a lower level of this therapy may still yield positive results.

However, it is important to be cautious at the same time, as this result has thus far only been demonstrated in a single individual.

How is success of this clinical trial measured?

VX-880 is being tested in people with T1D who have severe hypoglycemia and impaired hypoglycemia unawareness. The treatment requires immunosuppression, as the transplanted cells do not have any protection from the immune system. This requirement limits the patient population who can be enrolled in the trial.

The primary goal of this phase 1/2 trial is to assess safety, although efficacy will also be measured. Vertex is evaluating efficacy by measuring a few key metrics. This includes measuring C-peptide levels—a marker that directly indicates insulin production by beta cells. The participant in this study had no detectable C-peptide at all pre-infusion. 90 days after infusion of the VX-880 cells, the participant had both fasting and stimulated C-peptide, which directly indicates the presence of basal and glucose-responsive insulin secretion. In other words, the person was making some of their own insulin.

Treatment with VX-880 also led to a significant reduction in HbA1c, improving from 8.6% to 7.2% without severe hypoglycemic events. Even more impressive is that this lower HbA1c was achieved with a 91% daily reduction in insulin administration.

The study also demonstrated patient safety, as during the first 90 days, the participant did not experience any severe adverse events considered related to VX-880. This is important as immunosuppressive drugs do come with potential side effects.

JDRF’s Role

JDRF’s involvement can first be traced back to 2000, when Douglas Melton, Ph.D. was given a JDRF grant to make insulin-producing beta cells from stem cells—which he did in 2014.

Since then:

  • In 2015 Dr. Melton founded Semma Therapeutics to develop these stem cells into curative therapies for T1D.
  • In 2017, the JDRF T1D Fund made a significant investment in Semma.
  • In 2019, Vertex acquired Semma for almost $1 billion USD.
  • In March 2021, VX-880 received fast-track designation from the US Food and Drug Administration (FDA).

JDRF globally has prioritized stem cell therapy as a potential cure-based therapy and will continue to investigate and fund the most promising research.

What does this mean for Canadians with T1D?

For VX-880 to be broadly accessible to people with T1D, the cell product needs to both work and function without or with minimal immunosuppressive therapies.

The next step is approval to run clinical trials that could eliminate the need for immunosuppressives.

In the meantime, Vertex will continue their clinical trial for people with T1D who have severe hypoglycemia and are currently enrolling in several sites in the United States.

JDRF Canada will continue to monitor results and provide updates as they are made public.

JDRF 2023 Innovation Grants

JDRF provides seed funding for highly innovative research with significant potential to accelerate the most promising type 1 diabetes (T1D) research in both cures and approaches to improve disease management. JDRF Innovation Grants address key challenges in T1D research and have the potential to generate ground-breaking discoveries.

JDRF is thrilled to announce that two Canadian researchers out of the University of British Columbia (UBC) have recently been awarded one-year Innovation Grants for their T1D studies in stem cell derived beta cells. 

A potential cure for T1D is to transplant islet cells (the cells responsible for producing insulin) from recently deceased donors. However, there aren’t enough donors to meet the demand for all people with T1D who could potentially benefit from this treatment. However – there is a potentially limitless supply of insulin secreting cells for transplantation if stem cells could be turned or ‘directed’ into beta cells that secrete insulin in response to glucose. A challenge to this approach is that, thus far, beta cells derived from stem cells do not produce as much insulin as naturally occurring, healthy beta cells.  Therefore, more research is needed to understand how we can derive stem cell-derived beta cells that match or even exceed the properties of naturally occurring beta cells.

The two new innovation grants will attempt to quantify (Dr. Hongshen Ma) and optimize (Dr. Dan Luciani) the insulin-producing capability of stem cell-derived beta cells, to get us closer to a product that can cure T1D.

While Dr. Ma and his team focus on identifying the properties of high achieving stem cell-derived beta cells, Dr. Luciani and his team are examining the role of the mitochondria in the insulin producing capacity of stem cell-derived beta cells.

Dr. Hongshen Ma (University of British Columbia)

Dr. Ma is working to discover why there is limited insulin-secretion capability of stem cell-derived beta cells, by examining the cells at an individual level. 

Recent research has indicated that not all beta cells within an islet are equal. Rather, different beta cells within an islet have different roles, and may produce different amounts of insulin.  One potential explanation for the limited insulin-secretion capability for stem cell-derived beta cells is that there are under and over-producers. It’s been theorized that when studied as a group, all the transplanted stem cells appear to produce little insulin relative to naturally occurring, healthy beta cells, but if looked at individually a subset of these cells are in fact producing much more insulin than the rest. Studying these differences is challenging, but cutting-edge approaches that allow single cell identification can reveal new insights about how islets function — and therefore how we can recreate them for cure therapies. 

To address this challenge, Dr. Ma and his team are developing a new technology to measure the insulin secretion capability of stem cell-derived beta cells at the single cell level. This technology will enable the discovery of which genes and proteins are responsible for the ‘higher insulin achieving’ subtype of stem cells. By further comparing these to cells extracted from donor islets, the researchers will also be able to assess how similar the stem cell-derived beta cells are to those found in a person without T1D. Together, this work will push the boundaries of the current understanding of how islets function so that scientists can develop more effective stem cell-derived therapies for T1D that may one day be available to everyone in need.

Dr. Dan Luciani (University of British Columbia)

Dr. Luciani’s project is rooted in the theory that the development of mature, fully functional, beta cells involves two-way communication between metabolism in mitochondria (essentially the powerhouse of a cell) and the appropriate regulation of specific genes. It is hypothesized that without the proper two-way communication, not all stem cell-derived beta cells mature in a way that allows them to produce insulin in response to glucose. 

Dr. Luciani’s team believes that this maturation process does not get fully activated when beta cells are created from stem cells in the lab, but that their mitochondria can be ‘jump-started’ to trigger a sequence of events that result in formation of beta cells capable of secreting insulin at greater levels, perhaps close to naturally occurring, healthy beta cells. 

To address this challenge, Dr. Luciani and his team will make use of two innovative approaches – first, they will extract fully-functional mitochondria and transplant them into the immature stem cell-derived beta cells. They will also experimentally control the processes by which mitochondria normally fuse with each other or split into smaller units to influence the resulting beta cells. The team will further use specific metabolic molecules, novel drugs, and genetic manipulations to alter the structure and function of the existing mitochondria in immature stem cell-derived beta cells. This work may provide an untapped opportunity to improve the function of beta cells derived from stem cells for transplantation.

Development of a genetic risk score for type 1 diabetes

The causes of type 1 diabetes (T1D) are complex and not fully understood. What is known is that there is a genetic component to developing T1D – but who is at greater risk?

JDRF Canada is pleased to announce a new JDRF grant to support Dr. Despoina Manousaki, pediatric endocrinologist and genetic epidemiologist at Sainte-Justine Hospital in Montreal, that will allow her and her team to explore how the genome of an individual can predict the risk of developing type 1 diabetes (T1D). The more we can understand about the genetic predisposition of T1D, the more effectively we can screen for this risk and develop therapies to halt or delay the progression of the disease.

Dr. Manousaki, a former JDRF postdoctoral fellow, leads a research program focused on the genetics of complex disease in childhood.

How do genetic risk scores for type 1 diabetes work?

Existing genetic risk scores for T1D were largely developed using data from White European populations, which differ substantially from Canada’s diverse population. In her new JDRF-funded project, Dr. Manousaki will develop a trans-ancestral polygenic risk score for T1D, in simpler terms – looking at how different ancestral backgrounds and genetics influence the risk of developing T1D. These newly developed risk scores will be used in research and clinical practice to assess T1D in a more equitable manner. Since the existing polygenic risk scores perform poorly in diverse ancestral populations (as they were developed primarily in White European populations), there is a need to diversify these scores, particularly for T1D-related genes which are known to vary between people of different ancestral backgrounds.

Dr. Manousaki will use machine learning approaches that employ computers and algorithms to examine large European genetic datasets while incorporating genetic information from African, Indian, Latino, South-East Asian and Chinese ancestries. This will create more precise individual risk estimates of developing T1D, an important step for informing T1D screening in a diverse population. By having a better understanding of who might develop T1D, clinical teams can better select candidates across diverse Canadian populations for clinical monitoring as well as T1D prevention trials.

With the recent FDA approval of teplizumab (brand name Tzield), the first ever disease-modifying therapy that can delay the onset of T1D, research like that performed by Dr. Manousaki and her team will ensure that new T1D therapies are tested and applied appropriately in the diverse Canadian T1D community.

Safe and immune cloaked stem cell-derived beta-cells: treatment for type 1 diabetes

A significant part of JDRF Canada’s research strategy is funding the most promising cure-based research for type 1 diabetes (T1D).

T1D is an autoimmune disease where the body destroys the cells in the pancreas responsible for making insulin. People with T1D must administer external sources of insulin, either through multiple daily injections, pump or pen in order to survive. Many cure-based research studies involve replacing these cells through transplantation, in the hopes that they will start producing insulin again.

Transplantation of donor cells could be a possible cure for type 1 diabetes

Transplantation of donor pancreatic islet tissue is a promising therapy; however, transplant therapy is limited due to shortage of transplantable islets (from deceased donors), limited durability of transplanted cells (cells that stop working or growing after transplantation), and the need for long-term immunosuppression therapy to prevent immune-based rejection of the transplanted cells, similar to organ transplants. Identifying alternative and more universal sources of transplantable beta cells is necessary to make this potential therapy available to larger numbers of people living T1D, and the Nagy lab is dedicated to achieving this.

How does it work?

In a brand-new JDRF funded project, Dr. Andras Nagy, senior investigator at the Lunenfeld–Tanenbaum Research Institute in Toronto, Canada, is testing the functionality of insulin-producing cells created from human stem cells. In collaboration with Dr. Timothy Kieffer (University of British Columbia), Dr. Nagy’s team will cultivate insulin-producing islet cells from an unlimited supply of human stem cells for a potentially unlimited cell therapy.

These cells will incorporate two gene-editing technologies previously developed and patented by the Nagy lab: (1)Dr. Nagy’s FailSafeTM technology is a gene-editing solution that employs an inducible kill-switch to rapidly eliminate dividing, potentially tumour-forming, cells, thereby eliminating the risk of tumours following transplantation of a stem cell-derived cell product. (2) Dr. Nagy and his colleagues have developed an “immunocloaking” strategy for the transplanted cells by modifying specific genes that allow these cells to remain hidden from an immune system attack.

These technologies will offer solutions to the safety concerns of cell therapy and the autoimmune challenge found in T1D, which could allow for stem cell transplants without the need for immunosuppression.

This is an exciting project that harnesses the most cutting-edge methods to develop a safe and effective cell therapy solution that may lead to a cure for T1D.

JDRF Canada will provide updates on this research as it becomes available, and when it moves to the clinical trials stage.

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.

JDRF-CIHR Partnership to Defeat Diabetes Grants Now Announced

The pace of type 1 diabetes (T1D) research is moving faster than ever before. 

In November 2021 to mark November’s National Diabetes Awareness Month, JDRF announced a new investment of $7 million to support four Canadian research teams as part of the JDRF-CIHR Partnership to Defeat Diabetes, which will help to accelerate development of stem cell-based therapies for T1D as well as improve pediatric diabetes research and quality improvement across Canada, and our understanding of variation in human insulin production. 

This is part of the results of the Team Grants in Diabetes Mechanisms and Translational Solutions competition, an investment of $20M in 10 research projects. Diabetes Canada, Kidney Foundation of Canada, and the FRQS have also received funding as part of this competition, as all work together to improve health outcomes for Canadians. 

JDRF is pleased to share the summaries of all four research grants: 

Designing stem cell-derived islets for diabetes therapy

Dr. Timothy Kieffer (University of British Columbia) Nika Shakiba, (University of British Columbia), Dr. Elizabeth Rideout, (University of British Columbia; CIHR Sex and Gender Science), Dr. Corinne Hoesli, (McGill University), Dr. Christopher Moraes (McGill University) 

People with type 1 diabetes lack the islet cells that release the hormone insulin. Scientists at the University of Alberta made breakthrough improvements in transplanting clusters of insulin-producing islet cells. The procedure is quick, and many transplant recipients can reduce or even eliminate insulin injections. Unfortunately, the only current source of islets for transplant is recently deceased donors and only a tiny fraction of those in need can receive the procedure.  

Over the past several years, there have been remarkable breakthroughs in unravelling the process by which islet cells develop naturally in the body. As a result, it is now possible to replicate many steps of this process in the laboratory with cultured stem cells, culminating in insulin-producing cells. Kieffer and his team are aiming to significantly improve upon the manufacturing of the islet cells to obtain more robust insulin delivery, with a focus on generating an optimized process to mass-produce stem cell-derived islet cells that will form the basis for new clinical trials in patients with type 1 diabetes. 

A first-in-human trial of autologous induced pluripotent stem cells (ipsc)-derived islets: Developing a personalized diabetes therapy

Dr. James Shapiro, (University of Alberta), Dr. Timothy Kieffer, (University of British Columbia),
Dr. Gregory Korbutt, (University of Alberta), Dr. Patrick MacDonald, (University of Alberta), Dr. Andrew Pepper, (University of Alberta), Dr. Blaire Anderson, (University of Alberta), Dr. Anna Lam, (University of Alberta), Dr. Peter Senior, (University of Alberta), Dr. Khaled Dajani, (University of Alberta) 

In type 1 Diabetes (T1D, ~10%), the B-cells are destroyed by one’s own immune system. In type 2 Diabetes (T2D, ~90%), the body becomes more resistant to insulin, increasing the demand and eventually leading to B-cell damage. Shapiro and his team will develop a stem cell-based therapy to replace or supplement damaged B-cells in people with all types of diabetes.  

They propose to manufacture new B-like cells from patients’ own blood cells so that they will be accepted by the immune system and no/minimal anti-rejection drugs are needed. In this project, they will conduct a first-in-human trial to implant these cells under the patient’s skin and evaluate their safety and preliminary efficacy. 

Being able to transplant an unlimited supply of self-derived islet cells without immunosuppressants is a novel approach to treat all forms of diabetes.

A deep phenotyping network for understanding human islet variation in health and diabetes 

Dr. Patrick MacDonald, Nominated Principal Investigator: Canada Research Chair; University of Alberta, along with his team: Dr. James D. Johnson, (University of British Columbia) Dr. Jennifer Bruin, (Carleton University) and Dr. Jianguo (Jeff) Xia, (McGill University). 

Insulin is the primary hormone responsible for controlling blood sugar levels. It is produced by the pancreatic islets of Langerhans, rises after a meal to promote energy storage, and falls during fasting to allow energy mobilization. The levels of insulin in the blood vary tremendously amongst people. Nutrition, age, sex, genetics, and environmental exposures are all important factors likely to impact insulin levels. However, the underlying mechanisms by which these factors affect islet insulin production at the cellular level are not clear.   

This team seeks to understand the variability in human islet function in relation to genetic and environmental impacts on diabetes risk and to identify mechanisms of islet dysfunction in diabetes. To do this they will take advantage of extensive data on the molecular, cellular, and physiological function of islets from human organ donors. They will also produce tools and resources so that other researchers can explore this data to answer their own questions about islet dysfunction in diabetes.  

Building CAPACIty for pediatric diabetes research and quality improvement across Canada  

Dr. Shazhan Amed, Nominated Principal Investigator: B.C Children’s Hospital, along with her team: 

Dr. Meranda Nakhla, (Montreal Children’s Hospital; McGill University), Dr. Julia von Oettingen, (Montreal Children’s Hospital; McGill University) and Dr. Ian Zenlea, (Trillium Health Partners; University of Toronto). 

Although there have been many advances in diabetes care since insulin was discovered 100 years ago, youth with diabetes continue to have a higher risk of other health problems, a lower quality of life, and a shorter life span than their peers without diabetes. This health gap is likely in part due to suboptimal access to and delivery of their diabetes care, which is worse in disadvantaged populations across Canada. This project will develop strategies to address these gaps.   

The CAnadian PediAtric diabetes ConsortIum (CAPACIty) is a network of 15 childhood diabetes centers from across Canada. They are partnering with patients/families and health care professionals to jointly design and develop a Canada-wide childhood diabetes registry and research platform. The registry will enable them to improve diabetes care and health outcomes for Canadian youth through comparison of diabetes care quality and outcomes between Canadian diabetes centers, quality improvement initiatives, patient-informed research initiatives across Canada, and successful advocacy work.   

They anticipate that the CAPACIty registry will not only lead to better health outcomes but also serve as a powerful tool for governments and decision-makers to implement policy decisions that are driven by our data. Lastly, the patient advisory board will ensure better representation of youth with diabetes and their parents among provincial and national associations that advocate for people living with diabetes. 

As we celebrate the centenary of the first successful insulin shot in 2022, a groundbreaking achievement that saved millions of lives, we recognize the need to continue investing in research that will move us beyond insulin treatment towards a cure.  

These new grants are an important step in that direction. 

To read more about all the JDRF-CIHR Partnership to Defeat Diabetes: 

Exciting update in Sernova’s type 1 diabetes (T1D) trial

Principal researcher confirms trial participants with a history of hypoglycemia unawareness are now insulin independent.

Funding cell replacement therapies research is one of JDRF’s most critical undertakings globally, in its efforts to support the most promising cure-based research into type 1 diabetes (T1D).  

On January 10, 2022, Sernova Corp. provided a progress update on its Phase 1/2 T1D clinical trial, a JDRF-funded clinical trial of its cell replacement therapy. The findings were presented by Dr. Piotr Witkowski, the clinical trial’s Principal Investigator at the University of Chicago.

Sernova is a clinical-stage regenerative medicine company and has continued to demonstrate promising results for its Cell Pouch System™. When transplanted with insulin-producing islets, this system has consistently demonstrated in ongoing trials that it can produce insulin in people T1D, and participants maintain more consistent blood sugar levels. Additionally, it has demonstrated ongoing safety and tolerability of the system. In Sernova’s current clinical trial, patients must take immunosuppressive drugs to prevent rejection of the implanted cells.

Sernova’s approach for T1D involves transplanting purified islet cells (the cells in the pancreas that produce insulin and that are destroyed in people with T1D) from organ donors into the Cell Pouch™, an implantable medical device that allows these cells to survive long term and produce insulin. This is a cell replacement therapy that has the potential to be a T1D cure.

The objective of Sernova’s Phase 1/2 clinical trial is to assess the safety, tolerability and efficacy the Cell Pouch™ transplanted with insulin-producing islets in trial participants with T1D who have a history of hypoglycemia unawareness and severe hypoglycemic events.

To be in the study, participants must meet stringent eligibility criteria including, but not limited to, long-standing T1D, recent episodes of hypoglycemic unawareness and an absence of glucose-stimulated C-peptide detectable in their bloodstream (a biomarker that demonstrates the body is making its own insulin).

Hypoglycemia unawareness is a person’s inability to recognize the symptoms of low blood sugar before they become severe or even fatal. It typically occurs when blood glucose levels are below 3.0 mmol/L and is estimated to affect approximately 15% of people with T1D.

Highlights from the study include:

  • Maintained and ongoing safety and tolerability of the Cell Pouch™
  • Islet transplantation to the Cell Pouch™ resulted in the establishment of new, measurable islet function, documented by detectable levels of stimulated C-peptide in the first three participants who completed the course of transplants.
  • A supplemental, single intraportal islet transplant was enough for the first two participants to achieve and maintain sustained ongoing insulin independence and freedom from severe hypoglycemic events for over 21 and 2 months, respectively.
  • The third transplanted participant who recently completed their course of Cell Pouch™ transplants and a supplemental intraportal islet infusion, saw improvements in glucose control, near-normal levels of C-peptide, an absence of severe hypoglycemic events and reductions in daily insulin use.
  • Three additional participants are progressing through the study.

Sernova’s Cell Pouch™ System was created with the goal of one day treating people with T1D and other chronic diseases using stem cell-derived technologies, that will not require immunosuppression. Sernova is partnering with other companies to advance the Cell Pouch™ system to testing using a stem cell-derived source, as well as approaches that will reduce or eliminate the need for immunosuppression.

To date, between our partner organizations JDRF has invested more than $140 million USD in cell-replacement therapy research for T1D. As we celebrate the 100th anniversary in 2022 of the first successful insulin injection, this is another example of Canadian excellence in diabetes research that is accelerating us towards cures.

Celebrating Leonard Thompson Day

January 23rd marks the 100th Anniversary of the first successful insulin injection

JDRF’s primary goal is to cure type 1 diabetes (T1D) through research – as quickly as possible. To support people with T1D while cure research is advancing, we also fund ground-breaking research that seeks to make life with T1D easier, safer and healthier. With the discovery of insulin 100 years ago, JDRF is committed to funding the most promising research to move us beyond this treatment and toward cures.


Before insulin treatment, a diagnosis of type 1 diabetes meant inevitable death. T1D destroys the beta cells in the pancreas that make insulin, without which, the body cannot maintain healthy blood sugar levels.

In 1921, Frederick Banting and Charles Best discovered that the pancreas produced the insulin hormone under the directorship of John Macleod at the University of Toronto. With the help of James Collip, they purified a synthetic version of insulin and produced the first real treatment for diabetes. To this day, it remains one of the most important scientific breakthroughs in the medical field, often called ‘Canada’s gift to the world’.

January 11, 1922

On this date, fourteen-year-old Leonard Thompson, a teenage boy who was dying from T1D complications, became the first person to receive an insulin injection. But instead of lowering his blood sugar, it caused an allergic reaction. Leonard’s doctors went back to the lab, where they worked nearly around the clock to improve the formulation. 

January 23, 1922

Almost two weeks later, they returned to Leonard’s bed with a new syringe of insulin. With this version, Leonard’s symptoms began to disappear, and he regained his health. He would live another 13 years. The discovery of insulin, along with Leonard’s willingness to try this new treatment meant the death sentence that was T1D could be lifted, and people around the world could use insulin to manage their diabetes.

The importance of clinical trials

Research is how insulin was discovered and it is how we will find the next generation of therapies for T1D, and eventually a cure.

And clinical trials are an essential component of medical research and development. While Leonard’s receiving the first insulin to human injection was not known as a “clinical trial,” at the time, his participation allowed his doctors to rework and refine the formula that would make it a successful treatment for diabetes for over 100 years. In modern terms, Leonard was the first human participant in a clinical trial to test the impact of insulin on T1D.

This demonstrates the far-reaching impact of clinical trials. These studies can help bring new, better treatments to market that will impact the lives of millions. Thanks to Leonard Thompson, the updated version of insulin became a true breakthrough in diabetes care.

Funding cell replacement therapies research is one of JDRF’s most critical undertakings globally, and between our partner organizations we have invested more $140 million USD to date.

JDRF funds stem cell derived beta cell replacement trials that are ongoing right now, like those at ViaCyte, Vertex and Sernova and from here we may witness the research that leads to a T1D cure.

Participating in clinical trials

JDRF is funding many clinical trials, and one of the biggest challenges is finding volunteers to take part in studies – and it’s often because they simply didn’t know they could. It is also important to have a diverse group of participants who represent the Canadian T1D community, so that potential therapies and treatments can be tested for efficacy against the larger group who will be using them. When someone participates in a clinical trial, they help all people living with T1D, by enabling research towards better health outcomes and cures.

Choosing to participate in a clinical trial is a very personal decision. Early access to promising new treatment can be an enormous benefit. Others have found that by participating in a clinical trial, they learned more about their health or T1D management. And it can be motivating to know you are contributing to helping accelerate research that can improve the lives of people living not only with T1D, but other chronic conditions as well.

To learn more about Canadian clinical trials, and to find studies near you:

One person can make a difference – watch Tilla’s story of participating in clinical trials after her T1D diagnosis

On January 11, 1922, Leonard Thompson took a leap of faith. And when that first injection did not work, he took another step forward and tried again. From the bravery of one teenage boy, came millions of people whose lives have been saved by insulin. And every year across the globe, people living with T1D celebrate their ‘insulin anniversary’ while waiting for the breakthrough that will move us beyond insulin and towards a cure.

Every person who participates in a T1D clinical trial study helps us get closer to moving therapies from the lab to the market. We recognize too their courage and are so grateful for their help in accelerating the research that will one day mean a world free from diabetes.

New CDC Study: Children Who Have Recovered from COVID-19 May Be at Increased Risk of Diabetes

JDRF is committed to providing timely information about the latest research updates relevant to the diabetes community. Early January 2022 researchers from the Centers for Disease Control and Prevention (CDC) in the United States reported that children who have recovered from COVID-19 may be at an increased risk of being diagnosed with diabetes.

The CDC’s analysis was published in the organization’s January 7, 2022, edition of Morbidity and Mortality Weekly Report.

The New York Times reported the results January 7, 2022, (a subscription may be required to view full article).

According to The New York Times article, “…The CDC study is among the first to examine large insurance claim databases in the United States to estimate the prevalence of new diabetes diagnoses in children under age 18 who had COVID or were known to be infected with the coronavirus.”

It is important to note that the study—which does not differentiate between type 1 diabetes (T1D), type 2 diabetes (T2D) or any other form of diabetes—only highlights an association born out in the data examined.

The study does not identify how COVID-19 could cause (or provide evidence that it does cause) an increased risk of diabetes in children.

“As a research organization, JDRF will continue to analyze and update our community about these and other data related to COVID-19,” said Sanjoy Dutta, Ph.D., Vice President, Research, at JDRF International. “Other data globally have not shown an association between COVID-19 and diabetes onset, and we are in discussions with clinical leaders to further assess the evidence. In the meantime, we urge the public to be alert to signs of T1D and take steps such as vaccination to protect you and your loved ones.”

How was the study conducted?

The CDC study is based on a summary of findings from two U.S. medical claims databases, IQVIA and HealthVerity.

The examined data from the two databases focused on approximately 500,000 children under the age of 18 years who had a COVID-19 diagnosis between March 1, 2020, and June 28, 2021.

Diabetes incidence in COVID-19 positive individuals was 166 percent higher in the IQVIA cohort and 31 percent higher in the HealthVerity cohort (both compared to COVID-19 negative individuals).

There was no significant difference in diabetes incidence between the age sub-groups or sexes.

Additionally, there is no information in the examined data about other health conditions that could also be associated with a diabetes diagnosis (such as body mass index, blood pressure, etc.). It also does not account for race/ethnicity differences, or comparison with other cohorts that may have differential healthcare access (including less-than-adequate healthcare access).

A similar study has not been conducted in Canada with a Canadian cohort.

Prevention is still the best medicine. Vaccines and T1D

Getting vaccinated is everyone’s best line of defense against severe illness and hospitalization resulting from COVID-19.

All data show the approved COVID-19 vaccines are safe and effective for people with T1D. Vaccines were tested in people with diabetes (9.4% of Moderna, 8.4% of Pfizer-BioNTech, and 0.5% of J&J trial participants had diabetes) and no adverse effects were reported in these populations.

Currently, JDRF does not have information on the number of people with T1D in these trials, nor about T1D-specific adverse events reported yet in scientific literature, but there is no evidence now to indicate that these vaccines are unsafe for people with T1D.

Starting in early winter 2021, JDRF Canada successfully advocated that those with T1D be categorized as high priority to receive COVID-19 vaccines, based on data that shows adults who contract COVID-19 and have diabetes experience a three to four times higher risk of severe illness and hospitalization than people without diabetes.

Since late 2021, Pfizer-BioNTech has been approved for children aged 5 – 11 years old.

No vaccines yet are approved for children ages 6 months to 4 years, but Pfizer is conducting clinical trials for kids in this age group.

What to do if you have COVID-19 and T1D

If a person with T1D is infected with COVID-19, it can increase the risk for hyperglycemia (high blood glucose levels) and diabetic ketoacidosis (DKA).

DKA can have symptoms that feel flu-like, as does COVID-19. It is essential to continually check blood glucose levels and seek immediate medical attention if any symptoms of DKA are experienced. 

For more information on managing T1D while ill with COVID-19:

Know the Signs of T1D

According to The New York Times article about the CDC’s data analysis, “Many of the children in the study were only diagnosed (with diabetes) after having an episode of diabetic ketoacidosis (DKA), a (potentially) life-threatening complication that occurs when the body doesn’t have enough insulin to allow blood sugar into cells for use for energy.”

As members of the T1D community know, DKA can be terrifying and is frequently what leads to diagnosis.

To help prevent DKA at diagnosis, it is important to know of the signs of T1D, and see your primary healthcare providers if any of the following are noted:

Frequent urination

Increased thirst

Dry mouth

Itchy or dry skin

Increased appetite

Unexplained weight loss

Yeast infections

Learn more about the signs and symptoms of T1D:

If you are or a loved one are newly diagnosed with T1D, JDRF Canada has resources and support to help you adapt to your new normal with this disease. Please visit for more information, or connect to our Community Engagement team through our support services:

JDRF will continue to monitor research updates about any connection from COVID-19 to T1D and provide more information as it becomes available.

First-in-Human T1D Vaccine Trial for the Coxsackie B Virus Reports Positive Results

What is the Coxsackie B virus and how does it relate to diabetes?

JDRF is committed to funding the most promising research into cures for diabetes, and better understanding the mechanisms of the disease. One of the most significant challenges of preventing or curing type 1 diabetes (T1D) is that we don’t yet know its causes.

It has been theorized that a viral infection may be partly responsible for triggering T1D. What this means is that a common virus can set off the autoimmune response in the body that results in the beta cells responsible for producing insulin being killed off. This is one piece of the overall T1D puzzle, with certain biomarkers (that can be screened) making a person more likely to have this autoimmune response from a virus. But if we can prevent the virus from taking hold in the first place, we may be able to bring down the number of new diagnoses.

The main virus suspected as a potential cause of T1D is coxsackie B—a common infection that in most circumstances is asymptomatic or has mild symptoms. In rare cases, it may lead to viral meningitis, a heart or brain infection, and hand, foot, and mouth disease.

A potential vaccine

Presently, there are no vaccines against coxsackie B. That said, a year ago, the company Provention Bio started a clinical trial to test a vaccine that they developed against the coxsackie infection.

This trial was the first-in-human study of a coxsackie B vaccine, known as PRV-101, and positive interim results are out. Not only was the vaccine well tolerated by the trial participants (low incidence of side effects), but it induced high concentrations of anti-coxsackie B antibodies – meaning that should someone be exposed to the virus, they would have the means to fight off the infection before it could take hold and prevent triggering the autoimmune response that might cause T1D.

What is JDRF’s role in this vaccine development?

Since the late 1970s JDRF globally has been funding research that seeks to understand the viruses that may lead to the development of T1D. This research has demonstrated that several common viruses can attack islet cells and induce symptoms resembling diabetes.

In 1994, JDRF funded a postdoctoral fellowship for Heikki Hyöty, M.D., Ph.D., working in the lab of Michael Knip, M.D., Ph.D. The project title: “The Role of Coxsackie B and Other Enteroviruses in the Pathogenesis of Insulin-Dependent Diabetes Mellitus.” He went on to show that enteroviruses (of which coxsackie B is a member)—are the main culprit for the development of T1D.

Since this discovery, JDRF has funded Dr. Hyöty with more than 10+ grants, and Dr. Knip with more than 20 grants since 1997.

Hyöty and Knip co-founded Vactech in 2001, which developed PRV-01, and licensed it to Provention Bio in 2017.

In 2017, Provention Bio received an investment from the JDRF T1D Fund to advance the coxsackie B vaccine into clinical trials, and these trials began in December 2020.

What does this mean for people with T1D?

The results released from this study were preliminary; the final results will come in 2022. If the final results remain promising, the next step will be the development of the vaccine from clinic to population, ideally reducing the incidence of T1D diagnoses in children.

This is very exciting news that could prove to be an incredibly useful tool for preventing T1D from ever taking hold with a simple vaccine.