New developments in islet cell therapies for type 1 diabetes

Pancreatic Islet Cells

A significant part of JDRF Canada funding goes towards supporting cure-based research for type 1 diabetes (T1D). One such pathway is via stem cell replacement therapies that replace the beta cells responsible for producing insulin killed in the autoimmune process during T1D. The goal is to implant new beta cells that can one day start producing insulin again in a person with T1D, reducing or even eliminating the need for externally administered insulin, either via pump, pen or multiple daily injections. There are two primary sources of insulin producing cells: deceased donor pancreatic cells and human stem-cell derived islets. 

In late June 2023, the U.S. Food and Drug Administration (FDA) approved CellTrans’s Lantidra™, the first cell therapy to be authorized in the United States, for use in adults unable to approach average blood glucose levels due to current, repeated episodes of severe low blood glucose (hypoglycemia).

This therapy takes deceased donor islets and places them into people with T1D suffering from repeated severe low blood-sugar, called hypoglycemia, events. This therapy, much like an organ transplant, requires the use of immunosuppression drugs. CellTrans has not provided details on commercial availability or insurance coverage at this time, but it has also currently only been approved for use in the United States. 

In other related news, Eli Lilly and Company announced an agreement to acquire Sigilon Therapeutics, a public biopharmaceutical company with whom they had been partnering since 2018 to develop encapsulated islet cell therapies for T1D. These cell therapies include SIG-002, an encapsulated cell therapy that utilizes Afibromer™, a biomaterial designed to protect stem-cell derived insulin-producing cells from the immune system.

Per Lilly, the acquisition is the culmination of years of partnership to develop SIG-002, with Rogerio Vivaldi, M.D., CEO of Sigilon, noting that “Lilly is well-positioned to apply its industry-leading clinical and technical capabilities to harness the full potential of SIG-002 for the benefit of patients and their caregivers.”

Alongside the JDRF T1D Fund, Eli Lilly is also invested in Seraxis, a venture-backed start-up company developing islet replacement cell therapies for T1D. 

How can this help people living with T1D?

 The development of islet cell therapies is paramount to JDRF’s strategy to develop cures for T1D, and this news speaks to a long history of JDRF support for the academic researchers behind these therapies. 

Lantidra, which was developed by CellTrans, is led by José Oberholzer, M.D. He is a JDRFI grantee and a member of the JDRF Cell Replacement Consortium. JDRF has awarded Dr. Oberholzer significant funding since 2006 for his work on stem cell-derived replacement therapies. JDRFI also awarded Daniel Anderson, Ph.D., and Robert Langer, Sc.D., more than $20 million in funding since 2004 to help support the development of Afibromer, the material used in SIG-002.

Lilly’s acquisition of Sigilon is a signal of interest by one of the world’s leading pharmaceutical companies in the development of islet cell therapies—another leading pharmaceutical company working on cures for T1D. This also includes the stem cell-derived islet replacement activities of both Novo Nordisk’s partnership with Aspect Biosystems and Vertex’s positive update at ADA, as well as on the immunology side, Sanofi’s acquisition of Provention Bio. 

The FDA’s approval of Lantidra and Lilly’s agreement to acquire Sigilon represent two steps forward in the path to cures. 

JDRF Canada is currently pursuing multiple therapeutic approaches to cure T1D, and the T1D Fund has made 37 investments in venture backed companies with T1D programs since its launch in 2016, with over 20 active cures programs in development. With the T1D Fund, JDRF will continue to support and pursue these options to help bring these life-changing therapies to more people faster.

New Drug to Prevent Hypoglycemia Enters Clinical Trials

Update: June 15, 2023 

Zucara Therapeutics Inc., today announced additional funding from JDRF International in support of the development of ZT-01, a once-daily therapeutic designed to minimize hypoglycemic episodes in people with type 1 diabetes (T1D). JDRF will support Zucara’s planned Phase 2a study of the effect of ZT-01 On Nocturnal hypoglycemia Events in people with T1D (“ZONE”). Zucara expects to begin clinical trials in Fall 2023.  

ZT-01 has now been approved by both Health Canada and the FDA for the Phase 2 clinical trial.Once the trial sites have been confirmed, JDRF will provide information on how to participate on our clinical trials page found here: https://breakthrought1d.ca/participate-in-research/ 

 Why this news is important: 

  • Having demonstrated proof-of-concept and ZT-01’s ability to significantly increase glucagon release in patients with T1D, the Phase 2 ‘ZONE’ trial will evaluate if ZT-01 can prevent potentially dangerous low blood glucose levels overnight. 
  • The multi-centre trial is slated to begin in Q3 2023, with the incidence of nocturnal hypoglycemic events compared to placebo serving as the primary endpoint.        
  • ZT-01 has the potential to become the first drug available to prevent hypoglycemia in people with T1D.

Low blood glucose, called hypoglycemia, is a real fear for people with type 1 diabetes (T1D). Although there are rescue treatments for it, such as injectable or nasal glucagon (Baqsimi), there are currently no preventive therapies available. Canadian company Zucara Therapeutics, however, may have an answer. It is developing a once-daily therapy, called ZT-01, that restores a person with T1D’s glucagon response so that they can counter-regulate hypoglycemia. 

April 19, 2023 – Update

Zucara Therapeutics announced that it received clearance from the United States’ Food and Drug Administration (FDA) for the Company’s planned Phase 2 ‘ZONE’ clinical trial of ZT-01 for the prevention of night-time hypoglycemia in patients with type 1 diabetes (T1D). 

Update: September 19, 2022

  • On September 19, 2022, Zucara Therapeutics reported that the highly positive results from its proof-of-concept Phase 1b trial of ZT-01 has secured continued funding for Phase 2 clinical studies. Please see this link for more information. This is a once-daily therapeutic drug that restores glucagon secretion to prevent hypoglycemia, and if approved would be the first drug of its kind for people with type 1 diabetes. 

What this means:

Zucara has achieved the development milestones required to secure the remaining funds available from its previously announced US$21 million Series A Financing.

Zucara plans to file an investigational new drug (“IND”) application for a Phase 2 trial by year end, with initiation of first patient dosing expected in Q1 2023.

ZT-01 has the potential to become the first therapeutic designed to prevent hypoglycemia in people with type 1 diabetes.

This is a very exciting development and has the potential to change the realities for people with T1D, particularly those who are hypoglycemic unaware. JDRF will continue to monitor trial results and will update as more become available.

***

Update: February 17, 2022
On February 8, 2022 Zucara Therapeutics Inc., a diabetes life sciences company announced that the peer-reviewed journal, Diabetes, Obesity and Metabolism, published a paper reporting positive preclinical data on its diabetes drug ZT-01, which is currently in a Phase 1b clinical trial for the treatment of insulin-induced hypoglycemia in patients with Type 1 diabetes (T1D).

“We are thrilled with the positive results of this preclinical study, whose efficacy portion employed a similar methodology to that of our ongoing Phase 1b clinical trial,” said Richard Liggins, Zucara Therapeutics’ Chief Scientific Officer. “This study demonstrated that ZT-01 significantly increased glucagon secretion in rats with T1D, and reduced the frequency and severity of hypoglycemia, suggesting ZT-01’s potential to reduce hypoglycemia exposure in humans with T1D.”

If approved, this would be the first once daily therapeutic for people with T1D to help reduce the effects of hypoglycemia, one of the most common complications of the disease.

JDRF will provide updates as the trial progresses and further results are known.

***

Previously, JDRF provided funding to move Zucara beyond the “valley of death”—when discovery research is translated into a therapy or technology, but lacks the funding to make it real. In April 2020, Zucara secured the backing of a large venture capital fund to move ZT-01 into clinical trials.

Hypoglycemia is an unintended consequence of insulin therapy, and happens when one takes too much insulin. It causes sweating, fatigue, irritability and, in severe cases, seizures and/or death. It is one of the most feared and acute complications of T1D.

The first clinical trials of ZT-01 began in September, 2020 in Ontario. If these early trials are successful, ZT-01 will be much closer to approval, and to providing much-needed relief to the millions of people with T1D and their families who suffer from the fear and dangers of hypoglycemia.

In November 2020, Zucara also announced new funding to broaden development of ZT-01 for hypoglycemia in people with insulin-requiring type 2 diabetes, indicating that even more people stand to benefit from the new drug.

Meet our new Board Chair – Helena Gottschling

In late April 2023, Helena Gottschling was elected Board Chair for JDRF Canada.

Helena Gottschling is a purpose-driven leader who enjoyed a 37-year career with RBC before retiring in 2022. As Chief Human Resources Officer (CHRO) from 2017-2022, Helena was a member of Group Executive who set the overall strategic direction of RBC, Canada’s biggest bank and one of the largest in the world based on market capitalization. Helena was named one of Canada’s Most Powerful Women in 2021 by the Women’s Executive Network in the C-Suite Executives award category. 

Her distinguished career encompassed senior roles in Retail Banking, Human Resources and National Office, and she has expertise in talent strategy, performance enablement, leadership development, cultural change and enterprise transformation. 

Helena built her reputation as a trusted advisor, a change champion, and a devoted employee advocate by unlocking the potential of employees and strengthening RBC’s position as a top employer of choice on a global scale. She has been responsible for many transformational initiatives impacting 86,000+ employees, including the future of work strategy, COVID-19 response, diversity & inclusion strategy, and reimagining the HR function.

She is also a board member of Plan International Canada.

Helena is married with one son and lives in Kelowna, British Columbia, Canada. Her son lives with type 1 diabetes (T1D).

Helena recently sat down with JDRF Canada to talk about what brought her to the organization and what she hopes to achieve as Board Chair.

JDRF – Tell us a bit about your personal story about your son’s diagnosis.

We moved to Calgary from Vancouver in late spring 2002.  At the time, Matt was 9 years old, and we were all adjusting to our new life in Alberta. We were enjoying our first winter skiing most weekends in the beautiful Rocky Mountains. 

In February of that year, we noticed Matt was experiencing unusual symptoms and behaving in ways that weren’t like him. That’s when he was diagnosed with type 1 diabetes. Nobody in our extended family had T1D so we had a lot to learn along the way.

Matt received excellent care at the Children’s Hospital in Calgary and again in Toronto when we moved there in 2004. Our son never lets diabetes get in the way of his life! He skied, played rep soccer, then rugby at school and university.

And when he was able to access an insulin pump and continuous glucose monitor (CGM) it helped him better manage his diabetes and of course gave him more flexibility and independence. Which is why JDRF advocates and supports patient choice and universal access to these lifesaving devices.

JDRF – What progress have you seen during your time as a board member?

When Matt was first diagnosed 20 years ago, I got involved with JDRF through the ‘Ride for a Cure’. It was in Calgary, and I still remember the first year. I think there were two stationary bikes going! It’s incredible how much the event has grown since then and how things have changed!

At that time, we managed his T1D through injections with fast acting and long-acting insulin – testing his blood glucose levels multiple times a day, recording it manually in his workbook and calculating the amount of insulin he would need based on his food intake and exercise.

Since then – and just within the last twenty years – the progress made to help people with T1D manage their diabetes more effectively has been amazing. First with the pump – which helped him stay in range while offering more ‘just in time’ mgmt.

Then came the CGM – as a parent, the CGM gave me some peace of mind.  Especially when our son left home to go to university and then live on his own. Knowing that an ‘alarm’ would alert him when he was too low or high (blood glucose levels) so that he could make adjustments (either with more insulin or eating something with sugar), helped me sleep at night.

And the progress made towards finding a cure is so encouraging – research projects from stem cell research to islet transplantations in countries around the world gives me so much hope as a parent with a son with T1D, and as someone involved with JDRF who is helping us get there.

JDRF – what do you hope to see/achieve as Board Chair?

My ultimate hope of course is that a cure is found for T1D – a world without type 1 diabetes! Until that time, I want to help ensure that JDRF Canada continues to fund research projects towards this goal and continue our advocacy work for those living with T1D through Access for All – ensuring equitable access to insulin, diabetes devices, the Disability Tax Credit and government funding for more research.

Our immediate goal is to complete our $100M Campaign to Accelerate fundraising campaign in celebration of the 100-year anniversary of the discovery of insulin – to date, we’ve raised over $70 million of our goal of $100 million. The support we’ve received has been absolutely inspiring. 

We need to continue to communicate the impact we’ve made to our donors and supporters – dollars to research and most importantly, the outcomes of the research projects we fund. We will also continue to improve our funds to mission, making sure that the funds we raise go to the most promising research both into cures, which accounts for approximately 70% of funding disbursement, and into improving lives for people with T1D today, including our increased focus on mental health.

We also must continue to collaborate with partners along the way – the JDRF affiliates but particularly JDRFI in the United States, all levels of government, academia and other charitable orgs like Diabetes Canada and Brain Canada who support a shared mission and vision.

We need to continue to evolve our culture at JDRF so that employees, volunteers and supporters experience and feel the impact we’re making, because they all contribute through their work each and every day.

And as Chair of the Board, to work with the Senior Leadership team and Board Directors to ensure we deliver on our strategy, our mission, and our promise as an organization to our donors, supporters, staff and all Canadians affected by T1D, as we work towards a world free from type 1 diabetes. 

Why we Walk: The Schaub Family shares their story

Receiving a diagnosis of type 1 diabetes (T1D) for their child can be overwhelming for a family. And it was no different for Tienne and Andreas Schaub, when on April 4th 2022, they learned their daughter Leia had T1D.

To honour Leia’s first year ‘diaversary’ (anniversary of her T1D diagnosis date) the family has put together a team for Vancouver’s Sun Life Walk to Cure Diabetes for JDRF, and Leia has the taken on the role of being the BC Ambassador – something the family considers a privilege.

“Over the past year, we have learned a tremendous amount as a family and have adapted to the new lifestyle. Leia continues to be strong and brave every day. From hiding under the table, crying, and refusing to let us give her an insulin injection, to now doing her own finger pokes, knowing how to read her blood glucose levels and what to do when she is having a low, and even prepping the needles for her shots – all amid starting kindergarten. Pretty amazing for a 5-year-old! She is our hero,” say Tienne and Andreas. 

Like many families, the Schaubs recall noticing out of character behaviour from their daughter in the days leading to Leia’s diagnosis.

“We still vividly remember the day she was diagnosed. For the week leading up to it, we noticed that she was always thirsty and going to the bathroom more than usual. She also seemed to be triggered more easily and very emotional. We started looking up the symptoms and everything pointed to diabetes. We thought “that can’t be, right? No one in our family has type 1 diabetes,” remember Tienne and Andreas. 

“On the Sunday before her diagnosis, she became lethargic and just wanted to lie around all day – not normal for a 4-and-a-half-year-old. So, the next day, we brought her to the doctor and within two hours of getting her blood work, we were called to bring her to emergency right away as her blood glucose was in the forties when it should be between 4-8. Those first few hours seemed like days as we watched her cry and yell as she was afraid to get her finger pokes and had to hold off eating and drinking. Her emotions were out of her control due to her high blood glucose levels, and she was screaming and kicking around in her hospital bed. We have never seen her like that before,” they continue.

There are an estimated close to 300,000 Canadians living with T1D – and this number is increasing at an alarming rate.In fact, Canada now has the fifth highest incidence rate of T1D in children aged 14 years and younger in the world, and researchers still don’t know why.

And while Tienne and Andreas recognized some signs and symptoms of T1D that Leia was experiencing, many parents don’t make the connection to diabetes until it’s a medical emergency. The prevailing medical wisdom used to be that T1D developed quickly, with a sudden onset of symptoms. 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. 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 diabetic ketoacidosis (DKA).

Fortunately for Leia, the family was lucky to have caught her T1D early enough that she did not go into DKA which can be life-threatening and result in a prolonged stay in ICU. They were allowed to go home after a one night stay in the hospital. 

Eventually after the insulin she needed started to kick in, Leia became her normal self again. The family was very grateful to everybody at BC Children’s Hospital for being extremely supportive and helpful in guiding them through those first few challenging days. There were even two clowns who visited the family during their hospital stay that brought a lot of joy to Leia. 

Over the next few days, the family went back to the hospital to learn how to manage type 1 diabetes together. This is a disease that affects an entire family, and the Schaubs along with their extended family approached Leia’s care as a collective.

They quickly came across JDRF and within the first two weeks after Leia’s diagnoses, they received a Bag of Hope, a toolkit with useful resources for children and teens who have been diagnosed with T1D and their caregivers. Along with educational materials, the Bag of Hope includes Rufus, the Bear with Diabetes® — to help show children that they are not alone while learning to take injections and test blood glucose levels.

JDRF also helped to connect Tienne and Andreas with peer support within one week of Leia’s diagnosis, which was extremely helpful to answer all the random questions they had and to talk to another parent who had gone through the same experience. 

Part of the reason why Tienne and Andreas joined the Sun Life Walk to Cure Diabetes is to help educate others who don’t have personal experience with T1D on how much a life and family is changed by a diagnosis, and why funding the research towards cures is so important.

“Leia (or any person with T1D) needs to check her blood glucose level at least 4-5 times a day (before every meal, at bedtime, whenever there are symptoms of a low/high) through finger pokes drawing blood that then gets applied to a glucometer. We did this process for the first few months after diagnosis and often checked her at night as well (midnight and 3am) by doing extra finger pokes to catch a low that might happen overnight and would otherwise go unnoticed. Lots of sleepless nights for us at the beginning and lots of sugar tablets and snacks in the middle of the night for Leia to get her blood glucose back to a safe range,” they explain.

“Now we use a CGM (continuous glucose monitor) which is a sensor that Leia wears on her abdomen 24 hours a day. It allows us to monitor her blood sugar remotely even while she is at school. It will also send us alarms on our phones, so we only need to wake her at night when her low blood glucose alarm goes off.

Still – before each meal, we need to calculate the amount of carbohydrates that Leia will eat and give her insulin shots to match the carbs in her food. That’s 4-5 shots a day and sometimes even more, especially during growth spurts or when she has a cold. There were times where we had to give her 2-3 extra shots at night to correct unexpected high blood glucose levels. It’s a constant balancing act. And there are no breaks, no days off,” they continue.

The family recently did the TrialNet screening to see if there was a genetic component to Leia’s T1D. They were especially worried about their younger son Rudi, as having one child with T1D elevates the risk for siblings to develop the disease as well.

“It’s a big relief to have this screening available as there were several times since Leia’s diagnosis where we were worried or paranoid about Rudi developing T1D and we did finger pokes on him too,” says Andreas.

 TrialNet screens for specific autoantibodies in the blood that make a T1D diagnosis more likely. Fortunately, at the time of screening, all the family’s tests came back negative for these autoantibodies. Research shows that while first-degree family members of T1D are at an elevated risk of developing T1D, like with Leia – around 85-90% of newly diagnosed cases do not have a direct family connection.

It’s been a tumultuous year of learning to adapt to the ‘new normal’ that comes after a T1D diagnosis in a family, but the Schaubs have already made a commitment to giving back and helping the other families who will come after them on this journey with T1D.

Team Leia’s Unicorns has set an ambitious goal of raising $40,000 for type 1 diabetes research, and the family is confident they will reach it.

“We are walking and fundraising with the hope that one day, there will be a cure – not just for Leia – but for every child and every family affected by type 1 diagnosis. JDRF has been with us from day one and has helped us stay positive throughout our journey,” say Tienne and Andreas. They encourage everyone to please support them and the Walk and help to turn type one into type none.

The JDRF-CIHR Partnership to Defeat Diabetes announces 4 new grants in Precision Medicine

Launched in 2017, the JDRF-CIHR Partnership to Defeat Diabetes is a landmark collaboration between the Government of Canada, through the Canadian Institute of Health Research (CIHR) and JDRF Canada for a total combined investment of $30 million to support transformative type 1 diabetes (T1D) research. This Partnership was renewed for a further $30 million through Federal Budget 2021 and through further matched funding from JDRF Canada, now being allocated across three different areas of research: precision medicine, psychosocial health, and screening for T1D risk.

The first of the three funding opportunities has now completed. Complementing previous CIHR and JDRF investments in new T1D research, the CIHR-JDRF Team Grants: Precision Medicine in Type 1 Diabetes was designed to support multi-disciplinary research that would accelerate precision medicine approaches for prediction, prevention, and treatment of T1D. 

The goal of precision medicine is to get the right treatment to the right person at the right time.

Precision medicine approaches for T1D may integrate data about genetic, molecular, and environmental factors to improve T1D diagnosis, care, treatment or prevention. A greater understanding of how a person’s individual characteristics ‒ including genetics, biomarkers and immune and beta cell dysfunction ‒ contribute to T1D risk and progression may lead to more precise therapeutic targets, better characterization of disease risk and how it may progress in an individual, improved opportunities for safe and effective intervention and, ultimately prevention of T1D. 

These grants, worth $3.5 million each, have been awarded to four teams across Canada.

Precise Treatment for Pediatric Diabetes: Providing the right care, for the right patient, at the right time, over time 

Dr. Shazhan Amed (University of British Columbia), along with her team Dr. Mark Clements (Children’s Mercy Hospital), Dr. Tricia Tang (UBC), and Dr. Wyeth Wasserman (UBC). 

Dr. Amed and her team will use this grant to fill the information management gap in pediatric T1D care. TrustSphere will be the ‘one trusted place’ for kids with T1D that brings them, their families, and their healthcare providers together to manage their diabetes information and provide the right care to the right patient at the right time. Patients, families, and their healthcare providers will have access to important diabetes information, like blood sugars and insulin doses streaming from glucose sensors and insulin pumps, or recommendations from the diabetes team – all in one digital tool. 

Through collaboration with endocrinologists, mental health experts, and patient partners, TrustSphere will be expanded to include healthy living behaviours, diabetes self-management behaviours, quality of life and mental health. With patients and families, the research team will co-create personalized and tailored care experiences and use advanced methods to analyze data like machine learning to navigate patients and families to the care they need, when they need it.  

The team’s goal is to create a collaborative care experience, improve diabetes self-management, and ultimately improve lives for children and youth with T1D.

EVERYONE: Empowering diverse youth with diabetes through precision medicine 

Dr. Farid Mahmud (The Hospital for Sick Children; SickKids), along with his team Dr. Samantha Anthony (SickKids), Dr. Funmbi Babalola (London Health Sciences Centre), Dr. Andrew Paterson (SickKids), and Dr. Diane Wherrett (SickKids). 

Dr. Mahmud’s overall research focus is diabetes clinical research, relating to the early evaluation and prevention of diabetes-related complications and the impact of the social determinants of health on diabetes outcomes. In this JDRF-CIHR funded grant, he and his team will examine the impact of diversity (genetics, race, sex, gender, income, family support, mental health, etc.) on diabetes management in youth by applying artificial intelligence approaches. The experiences and barriers faced when receiving diabetes care will be gathered through interviews and feedback from youth with T1D and their families.  Armed with this information, the team will develop individualized treatment strategies that consider patient diversity and conduct a clinical trial to determine the feasibility, acceptability, and appropriateness of the program. 

The EVERYONE study will contribute to a greater understanding of the physiologic and social factors underlying diabetes outcomes, and the opportunity to personalize and optimize care to improve lives for children and youth with T1D.

Leveraging biological sex and genetics for beta cell-directed precision medicine in type 1 diabetes 

Dr. Elizabeth Rideout (University of British Columbia), along with her team Dr. James Johnson (UBC), Dr. Dan Luciani (UBC), Dr. Peter Thompson (University of Manitoba), and Dr. Bruce Verchere (UBC). 

Dr. Rideout’s research investigates the effect of biological sex on metabolic genes and pathways. In this JDRF-CIHR grant, her team will apply this work to T1D by examining how beta cell dysfunction differs between biological males and females during the progression of T1D. Rates of T1D are higher in biological males than females, and there is evidence of sex-specific responses to insulin and beta cell functioning in T1D.  Despite this, most T1D studies have used only male animal models and participants, and of those that did use both sexes, most failed to compare data between the sexes.  

Dr. Rideout and her team will examine beta cell function between males and females, which will inform T1D prevention and treatment strategies that account for the impact of biological sex. 

Spatio-temporal dynamics of immune and non-immune islet injury in type 1 diabetes

Dr. Peter Thompson (University of Manitoba), Dr. Herbert Gaisano (University of Toronto), Dr. Guy Rutter (Université de Montréal), Dr. Pere Santamaria (University of Calgary) 

Dr. Thompson’s research focuses on the heterogeneity (individual differences) of beta cells within an islet and how individual cells may respond differently to immune system attacks and stressors. T1D varies greatly between individuals in terms of time to onset, rate of progression, and insulin requirements, but the reasons underlying these differences are unknown. This team grant will investigate the functional “hierarchies” that appear to be present amongst beta cells. Specifically, the team will look at “leader” or “hub” beta cells within an islet that are guiding and coordinating the function of the rest of the cells.  

Through this information, the team will understand the variability in beta cell loss amongst individuals with T1D, which will inform development of disease-modifying therapies and cell therapies for T1D.  

JDRF is very excited to support these cutting-edge research projects that will help further the understanding of T1D’s disease mechanisms and ideally lead to more advanced treatments, therapeutics, patient outcomes and prevention of T1D.

JDRF Canada celebrates International Women’s Day

International Women's Day

Wednesday, March 8 is International Women’s Day (IWD), recognized around the world. IWD asks us to imagine a gender equal world. One that is free from bias, stereotypes, and discrimination. A world that’s diverse, equitable, and inclusive. A world where difference is valued and celebrated. The theme for 2023 is #EmbraceEquity and calls upon us to celebrate women’s achievements, raise awareness about discrimination and take action to drive gender parity globally.

JDRF is proud to celebrate the achievements of the Canadian women researchers who have blazed a trail in the field of type 1 diabetes (T1D) – both making life better, safer and easier for the millions affected by the disease, but also for all the women scientists who came after them.

From Dr. Priscilla White, an early pioneer in diabetes research and treatment, Dr. Dorothy C. Hodgkin who discovered the three-dimensional structure of insulin, Dr. Helen M. Free, who along with her husband created Clinistix, allowing people to check their glucose at home – to the mothers who helped found JDRF, women have played a critical and essential role in advancing our understanding of T1D.

Meet these women researchers whose work is helping to improve the lives of people affected by T1D today, while getting us closer to cures tomorrow:

Dr. Diane Wherrett – accelerating screening for T1D prevention. Dr. Diane Wherrett is currently a Staff Physician in the Division of Endocrinology, Department of Paediatrics, and a Professor at the University of Toronto at SickKids. She is the Centre Director for the Canadian arm of the TrialNet screening program, a NIH-funded multicentre T1D clinical trial group.

TrialNet offers risk screening for relatives of people living with T1D, and innovative clinical studies testing ways to slow down and prevent disease progression.

Dr. Despoina Manousaki, helping to create genetic risk scores for T1D. Dr Manousaki’s research focuses on the genetics of complex disease in childhood. Her team uses genomics, bioinformatics, and genetic epidemiology methods to better understand the genetic architecture of complex disease, and to apply these findings in translational research.

Her research program focuses on the genetics of type 1 and type 2 diabetes in children and is expanding existing genetic risk scores for T1D calculated from predominantly European-ancestry datasets, to include African, Indian, Latino, and Asian ancestries for a more diverse picture of T1D genetics.

Dr. Megan Levings – helping to find a better understanding of T1D immunology.

Dr. Megan Levings, of the University of British Columbia and BC Children’s Hospital Research Institute is one of Canada’s leading immunologists. She holds two JDRF grants: one examines samples from the ustekinumab (a disease-modifying drug) clinical trial to understand how the drug works in people with T1D. The hope is that ustekinumab (brand name Stelara) can block immune cells soon after the development of diabetes to protect and regenerate any remaining insulin-producing cells. This would ideally lead to the remaining beta cells producing enough insulin so that people with T1D would require less external insulin for a longer period, or not at all.

The other is a CIHR-JDRF grant that is examining the role of regulatory immune cells to apply novel approaches to  the development of cellular therapies to cure the disease.

Dr. Christine Nostro – forging new pathways in stem cell research for T1D.

Islet transplantation could help T1D patients regain control of blood glucose levels, making this an alternative to insulin injections as the only existing treatment. However – donor scarcity, poor islet survival after transplant, the need to optimize the transplant site and for life-long immunosuppressive treatment to prevent transplant rejection mean this treatment is only accessible to a handful of people with T1D.

Dr. Nostro’s team leverages their expertise in stem cell biology, vascular biology, islet transplantation and beta cell biology to address these challenges and are working to develop a safe and effective clinical-grade product for therapy.

The outcome from these studies will accelerate universal stem cell based T1D therapies.

Dr. Tricia Tang – making mental health support for T1D more accessible.

Dr. Tricia Tang is an Associate Professor in the Department of Medicine, Division of Endocrinology at the University of British Columbia Faculty of Medicine. She is also a behavioral scientist and registered clinical psychologist with an expertise in diabetes research in high-risk and medically underserved populations. Over the past 22 years, she has focused on developing, implementing, and evaluating low-cost and sustainable peer support models for improving long-term diabetes-related health outcomes.

Dr. Tang seeks to use digital health strategies and peer support models to close the mental health care gap for people with T1D.

Dr. Tang’s research goal is to design and evaluate a virtual care platform (REACHOUT) for highly trained peer supporters to deliver psychosocial support to adults with T1D living in rural and remote communities of interior British Columbia.
She is also the co-chair of JDRF’s Mental Health + Diabetes steering committee, helping to develop a training program for mental healthcare professionals to better serve their patients with T1D.

Dr. Shazhan Amed – developing new projects that aim to equitably improve outcomes for children with diabetes.

Dr. Shazhan Amed is a pediatric doctor who works at BC Children’s Hospital in Vancouver. Her areas of care are children and youth with diabetes. She is particularly interested in improving the quality of care provided to children with diabetes, as well as preventing childhood obesity and childhood onset type 2 diabetes.

Her team has developed the CAnadian PediAtric diabetes ConsortIum (CAPACIty), a network of 15 childhood diabetes centers from across Canada, with the collaborative goal of improving outcomes for all children with diabetes, particularly those from marginalized or lower-income communities.

To read more about other JDRF-funded researchers: www.breakthrought1d.ca/research/meet-our-researchers/

JDRF Canada – an all-women research department.

Chief Scientific Officer Dr. Sarah Linklater, along with her team Dr. Lara Green, Katie Ryan, and Dr. Anne Marie MacDonald (currently on maternity leave), use their science backgrounds and passion for accelerating type 1 diabetes research – that also may benefit other autoimmune diseases – to help JDRF determine the most promising research to fund. JDRF is so grateful for their efforts that are bringing us closer to our goal of a world free from T1D.

Exciting news: new drug in clinical trials shows slowed type 1 diabetes progression in newly diagnosed children and teens

A new JDRF International-funded study of a drug called verapamil is offering exciting new potential for a path to keeping beta cells healthier for longer in people with type 1 diabetes (T1D).When a person is diagnosed with T1D, they still have some remaining beta cells that continue to insulin for a few months – known as the  “honeymoon” phase. The new trial looked at whether the effects of a hybrid closed loop system (also known as an artificial pancreas system or automated insulin delivery system) and/or verapamil preserved beta cell function – and extends the honeymoon phase – one year after diagnosis in children and teens with T1D.

Verapamil is a drug currently prescribed for conditions like angina or high blood pressure.

The study found that newly diagnosed individuals on verapamil were making more insulin one year after diagnosis than those on placebo, with the average C-peptide, which is used to measure insulin production by beta cells, being 30% higher for the verapamil group compared to placebo. HbA1c was 6.6% in the verapamil group versus 6.9% in the placebo group, at one year.

There was no change, however, in the hybrid closed loop system arm. But participants using the hybrid closed-loop system showed improved glucose control and management, which can reduce potential diabetes-related complications.

Verapamil would be a once-a-day oral therapy (pill taken by mouth) and is already being manufactured, making it a cost-effective solution as well.

What does this mean for people with T1D?

Verapamil is not an approved therapy for newly diagnosed people with T1D, and it still must undergo further study to validate these results, ensure safety and efficacy, as well as learn if there are any potential side effects for people with T1D. Additionally, further trials must determine the long-term effects of the drug, if the preserved beta cell function lasts, and for how long.

What comes next?

JDRF International has been funding the study of verapamil for over ten years. The next step is to ascertain longer-term evidence of verapamil’s effectiveness and share this data with the clinical community and other health care leaders.

In the immediate term, JDRF International has a grant for a follow-up study for three years to see if C-peptide benefits persist. They are also funding several clinical trials to validate the results of this study and see if verapamil is effective when used in conjunction with other disease-modifying therapies, such as the recently approved Tzield™ (teplizumab-mzwv).

JDRF Canada will continue to monitor the results of this work, and report on further updates as they become available.

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.

JDRF Canada Youth Advocate Tilly Stimpson selected to represent Canada at the JDRF 2023 Children’s Congress

Tilly Stimpson, JDRF Canada in Washington. Blog Hero Profile Image.

Every two years, more than 160 children living with type 1 diabetes (T1D) gather in Washington, D.C., to meet face-to-face with some of the top decision-makers in the U.S. government. Similar to Kids for a Cure, the youth delegates in JDRF’s Children’s Congress take advantage of this once-in-a-lifetime opportunity to help US Members of Congress understand what life with T1D is like and why research to fund both life-changing and cure-based therapies is so critical. These amazing kids bravely represent the millions of people living with T1D and their families and loved ones.

Tilly Stimpson was diagnosed with T1D at age 2 and a half, in 2010. She has said that ‘Ever since I was diagnosed, raising awareness has been extremely important for me and my family,’ and she has been living that ethos through an extraordinary legacy of volunteering and advocacy with JDRF Canada.

Tilly has attended Kids for a Cure three times, twice in person and once virtually during the early part of the pandemic. She has been a key speaker at the Access for All speakers circuit in Pickering and surrounding areas, and a spokesperson for Access for All at Queens Park (parliament buildings in Toronto for the government of Ontario). She has advocated at the Canadian Institutes of Health Research (CIHR) a key JDRF Canada funding partner for vital cure-based research.

In 2021, to celebrate the 100th anniversary of the discovery of insulin, Tilly wrote 100 letters to seniors in nursing homes and the community to provide company during Covid lockdowns.

She is also an active youth ambassador, has filmed countless videos and participated in several activities with JDRF.

But Tilly is not just a volunteer and advocate, she is a prolific fundraiser too.

Some of her fundraising achievements include, but are not limited to:

Tilly has raised an incredible over $80,000 for diabetes research.

And if that was not enough, Tilly has also participated in two clinical trials, been a stock image photography model for diabetes representation and was nominated in her hometown for her advocacy work for ‘Inspire Women: Day of the Girl’

‘I am absolutely honored to be chosen to represent Canada at the International Children’s Congress.

“Advocacy and educating about T1D are important to me and to be given the opportunity to have a voice alongside international delegates is extremely exciting. I hope I make Canada proud!” says Tilly.

Read more about Tilly on her application here

The JDRF 2023 Children’s Congress takes place between July 9 – 11, 2023, in Washington, D.C.

JDRF Canada once again congratulates Tilly, and we know she will make all Canadians affected by T1D so proud. She already has.

JDRF Canada is pleased to announce the launch of JDRF Your Way

A newly re-tooled third-party fundraising program is now available across Canada: JDRF Your Way.

Support. Participate. Fundraise… Your Way

JDRF’s foundations lie in grassroots efforts and fundraising, and our accomplishments as an organization can be traced back to the power of our incredible community. In 1974, four years after JDRF was founded in the United States, a group of parents determined to find a cure for their children living with type 1 diabetes (T1D) came together and began JDRF Canada, launching the largest funder and advocate for T1D research in Canada.

Although the focus was and remains finding cures for T1D; since then, JDRF has helped fund research that has led to innovation and evolution of treatments that improve the quality of life for people living with T1D, and fulfill our vision of a world without T1D, while supporting our mission of improving lives today and tomorrow by accelerating life-changing breakthroughs to cure, prevent and treat T1D and its complications.

JDRF Your Way harnesses that energy to let supporters fundraise in ways that are meaningful to them. Whether it is holding a yoga class, coordinating a soccer tournament, celebrating a special event, or recognizing a ‘Diaversary’ (the date of diagnosis) – JDRF Your Way provides our community with the tools and resources needed to feel supported and be successful.

Meet Leah, who fundraised her way by organizing her own Ride in support of her daughters living with T1D

“My hope is that the funds raised help move forward research projects so that someday my two daughters, Erica and Tara, and others with T1D can live without the burden of this disease.”

The Pictou County Ride for a Cure is an annual fundraising event in Nova Scotia that brings motorcycle and bicycle riders together to create awareness and show their support for type 1 diabetes research.

Meet Vincent, who is looking to raise $100,000 in a series of runs to honour his son’s one-year DiaVersary

“January 5th, 2023, I completed my challenge of running 32 kilometres of the Saint-Charles River Linear Park, as part of a fundraising campaign to mark my son’s one-year anniversary of his diagnosis of type 1 diabetes. In fact, I even ran 35.85 thanks to a course error that made me take a long detour!

Beyond the fundraising, the day allowed us to live an extraordinary family moment. My two boys joined me to run the last kilometre, from the Quebec City Courthouse to Espace 400e. In the evening, we gave Thomas an album celebrating the one-year anniversary of his diagnosis of type I diabetes. The album contains messages of encouragement from family, friends, teachers, and coaches to provide him with the comfort he deserves whenever he is demoralized by the efforts and sacrifices that diabetes requires. He was moved to tears when he started reading the messages, especially his brother’s.”

Meet Anne, a teenager living with T1D who held a fundraiser called The 100 Project to celebrate the 100th anniversary of the discovery of insulin

“The idea behind The 100 Project was to seek $100 donations from 100 Canadian businesses large and small, with a goal of raising a minimum $10,000, to commemorate the centennial of Frederick Banting’s and Charles Best’s discovery of life-saving insulin.

Even when insulin was discovered, Banting and Best didn’t make it complicated. They sold the insulin patents for $1, which is what allowed insulin to be accessible to people. Frederick Banting said, ‘Insulin belongs to the world, not to me’ – they realized insulin is such a life-saving drug that can make such a difference. I would not be here without it.”

Meet Rob, who turned his passion for sports and being on the water into a fundraiser for T1D research

“A few years after starting the Medicine Hat waterski club in 2016, I wanted to organize a 24-hour waterski event and raise money for JDRF, in honour of my daughter’s 2013 T1D diagnosis. After a considerable amount of planning, we decided to split the event over two days and hold it on the longest day of the year, June 21, starting at 5pm on the first day and stopping when we could no longer see, and resuming the following morning at 5am, when the sun came up.

We were able to gather funds and auction items, and had 21 riders involved, rotating turns until the sun went down. The event continued the next day, with slalom skiing, hydro-foiling, surfing, wakeboarding, and even tubing. Once everyone was off the water, we had a band play to finish off the evening. We were able to raise approx. $5500 that went directly to JDRF.”

A newly developed website JDRFyourway.ca offers creative fundraising ideas, a FAQ section and fundraising tools and resources, along with contact information for our JDRF Staff Partner and Support Teams available to help you make your fundraiser a success.

With JDRF Your Way we have made starting a personal fundraiser easier, so you can make an even bigger impact by funding ground-breaking T1D research and providing the necessary supports to those living with T1D to ensure they live healthier, safer, and easier lives today.

Have questions or need more information? Please reach out at yourway@jdrf.ca.

2022 Wrap Up

As we got through the height of the pandemic in 2021 and moved into 2022, we believed that it would be a year of hope. And thanks to the support of our incredible community of donors, volunteers, supporters – we surpassed even our wildest expectations.

We opened the year with one of our biggest undertakings as an organization – our Let’s Make History Again fundraising event in support of our $100 Million Campaign to Accelerate. Five flagpoles and Leaders of History, four cities, 100 Ambassador of History families camping in the cold winter in solidarity. This was spearheaded by a passionate and dedicated JDRF volunteer, Peter Oliver, who himself camped atop a flagpole 30 years before, and whose daughter Vanessa was one of our Leaders in History in April. Sadly, Peter passed away in September, but we carry his spirit and devotion to finding cures for type 1 diabetes with us in everything we do. We are so grateful to our many volunteers, corporate sponsors and staff for taking Peter’s vision and making it reality.  Read more about Peter Oliver’s incredible legacy.

Click here to watch a video of this incredible event

Our community rallied in ways that we could not imagine, and it only continued throughout the year. Thanks to our JDRF supporters, we were able to continue advancing the most promising type 1 diabetes (T1D) research getting us closer to cures, while improving the lives of people affected by T1D today.

Thanks to the generosity of donors, JDRF had another ground-breaking year– dedicating more dollars to research, strengthening our strategic partnerships, continuing to support newly diagnosed families, and sharing the latest research updates with our community.

Some highlights over the past year included:
In T1D research:

In Cell Replacement:

In Disease-Modifying Therapies:

  • Teplizumab was approved by the FDA in the USA. This drug has shown great promise as the first ever disease-modifying therapy for T1D, as it has been shown to significantly delay the onset of T1D.

In Treatments to Improve Lives:

  • JDRF-funded researchers are performing advanced clinical trials on a once-daily therapeutic drug that restores glucagon secretion to prevent hypoglycemia, and if approved would be the first drug of its kind for people with type 1 diabetes.
  • Funding programs devoted to improving mental health care and outcomes for people with T1D, including REACHOUT.

In Screening:

To read more about research updates, please visit www.breakthrought1d.ca/blog to see more stories about the incredible progress that took place over the last year in type 1 diabetes research and development.

It has never been a more exciting time in diabetes research and at JDRF. And together with our JDRF community, we believe that a world free from type 1 diabetes is closer than ever.