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.

Celebrating 30 years of the Sun Life Walk Cure to Diabetes for JDRF

Walk 2023 Montreal Ribbon Cutting

Throughout June more than 40 communities gathered across Canada to raise funds for type 1 diabetes research.

The Sun Life Walk to Cure Diabetes for JDRF is the largest fundraising event in Canada that brings together the type 1 diabetes (T1D) community to raise funds for T1D research. A fun-filled family and community celebration, the Walk has raised more than $137 million to date over its 30-year history – making it one of the longest running and most successful fundraising events in Canada.

The collective fundraising goal is $3.1 million that will go towards research into cures for T1D.  This year is the 30th anniversary of the Walk and JDRF is proud to celebrate three decades of progress, commitment, and improving lives for people affected by T1D.

June was once again designated as Walk Month and to date more than $2.7M has already been raised in support of the most promising diabetes research.  Additional Walks are planned for the fall, including Walks in Hamilton, Niagara, Sudbury, Peel and the Walk in Halifax which was delayed due to ongoing wildfires.

The Walk is more than just a fundraiser. It’s a chance for families living with T1D to get together, share their stories and gain support. Many Walks had a tent and activities designated specifically for new families, sponsored by Abbott Diabetes Care and supported by JDRF Volunteers to provide the most up to date information and resources. As well, trained Research volunteers were on-hand at several sites to provide participants with information on the latest updates in T1D research.

Games were set up for the kids and emcees helped keep the energy going with music. Each Walk location provided their own unique offerings to make the day one to remember for the T1D community.

At the Toronto Walk, we were joined by JDRF ambassadors Max Domi and Kaleb Dahlgren, who met with families, took photos and helped to lead the warm-up. It was very moving for many newly-diagnosed families to meet two exceptional athletes with T1D and hear that diabetes doesn’t have to mean giving up on your dreams.

We would like to express our sincere gratitude to all our sponsors. With their support, we were able to offer an incredible day of community spirit with valuable resources for T1D families and innumerable connections made. Across Canada, the Walk was truly a celebration of 30 years of progress in T1D research, with the recognition that there is much work to be done.

Thank you so much to our Walkers, volunteers, donors and our national partners. We could not have done it without you. Together we walked, to one day cure T1D.

Our Sponsors
The Sun Life Walk to Cure Diabetes for JDRF 2023 Sponsors

Local Partners

Booth Sponsors
Farm Girl
Pizza Hut Limited Partnership

Corporate Champions
Alberta Milk
BASF Bryanna Higgins
The BC Dairy Foundation
Blundell Seafoods Ltd.
Capital Ford Lincoln
Capital GMC Buick Cadillac
The Medicine Shoppe Pharmacy Rochdale
Pineapple Therapy
Pure Orthodontics
River City Events
TD Asset Management
Universal Collision Centre
Weigl Enterprises Inc.

Gift In Kind Partners
Alberta Cloth Co. (Edmonton) Ltd.
Elite Auto Centre
GES Canada Inc.
Hotel X Toronto
The Low Carb Company
Pizza Nova
RE/MAX Edmonton Area & Associates
Rockets Candy Company / Ce De Candy Company Ltd.

CGM Use in Pregnancy

Pregnant woman using a Continuous Glucose Monitor

A 2013-2016 JDRF-funded study showed that the use of a continuous glucose monitor (CGM) during pregnancy for people with type 1 diabetes (T1D) results in healthier pregnancies and babies. The CONCEPTT study examined the use of CGMs during pregnancy in over 300 people with T1D. The use of a CGM during and prior to pregnancy resulted in 100 extra minutes per day with blood glucose in a healthy range, as compared to not using a CGM.  

The findings supported the use of CGMs to improve neonatal outcomes (i.e., the health of the newborn). Babies born to people with T1D using a CGM were less likely to have hypoglycemia postpartum, and half as likely to have complications or require intensive care. Publication of the primary results appeared in The Lancet on September 15, 2017: Continuous glucose monitoring in pregnant women with type 1 diabetes (CONCEPTT): a multicentre international randomised controlled trial. 

These findings influenced policy change in the UK, Australia, and the US, and influenced recommendations in Canada to provide all pregnant people with T1D access to CGMs.  

2019: The UK, Australia, and the United States all recommend that CGMs be used to improve HbA1c outcomes in pregnant people with T1D. 

2020: All pregnant people with T1D in the UK are offered funded real-time CGMs to manage diabetes in pregnancy.  

2021: Recommendation in Diabetes Canada guideline: “In pregnant people with type 1 diabetes, real-time CGM should be used to increase time-in-range and reduce the risk of overweight infants, neonatal hypoglycemia and intensive care admissions >24 hours.”  

2022: All Australians living with T1D can now access subsidized CGM devices following the expansion of the CGM Initiative by the Albanese Government. 

These milestones demonstrate the impact that donors to JDRF can have on pushing forward progress that improves the lives of people living with T1D.  

Through JDRF research funding and advocacy, real changes have been made to improve the lives of those living with T1D. A huge thank you to all JDRF supporters for making this happen. 

Follow up studies tracking the children born to the participants involved in the original study will continue with funding from CIHR.  

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.

Changes to Glucagon Availability in Canada

Important Announcement Glucagon availability in Canada
What is Glucagon? 

Glucagon is a hormone that helps the liver release glucose to raise blood glucose levels. It can be administered through injection, auto-injection pen or nasal spray.

Glucagon is generally used when a person with type 1 diabetes (T1D) is unable to swallow liquid or food because of severe hypoglycemia (low blood glucose) symptoms, including extreme drowsiness, unconsciousness or seizure. It is important to have glucagon on hand in case of an emergency and ensure others (especially caregivers of children with T1D) know where it is kept and how to use it.

Changes to Glucagon Availability in Canada 

On July 10, 2023, Novo Nordisk announced that it was discontinuing their glucagon products in Canada, both the GlucaGen vial/vial pack and the GlucaGen Hypo Kits. This comes on the heels of a Health Canada’s announcement in April 2023 that glucagon (Glucagon for injection, rDNA origin) is in a Tier 3 shortage.  In August 2022, Eli Lilly and Company announced the discontinuation of their Glucagon Emergency Kit from all markets, with the last expected date of distribution recently passing in June 2023. Eli Lilly subsequently pulled out of the glucagon field entirely in April 2023, when they divested their intranasal glucagon product Baqsimi to Amphastar Pharmaceuticals. This transaction is set to close in fall 2023.

In response to the Tier 3 shortage, Health Canada has been working with Amphastar to provide “designated drug” status for their generic glucagon injectable product. This status allows drugs approved in other countries (in this case, Amphastar’s glucagon is FDA-approved) to be eligible for “exceptional importation and sale” in Canada during drug shortages.

Novo Nordisk consulted closely with Health Canada during the decision-making process to discontinue sale of glucagon in Canada and are confident that Canadians will not be left without access to glucagon. Based on historical demand, the current supply of Novo Nordisk’s glucagon products in Canada (distributed by Paladin Labs) is expected to last until February 2024. For further information about ordering product, please contact Paladin Labs Inc. Customer Service at 1-866-340-1112. If you have any Medical Information questions, please contact Novo Nordisk Customer Care team at 1-800-465-4334.

Options for Glucagon in Canada 

As of July 2023, the options for glucagon in Canada are as follows: 

It’s important to always have fast-acting sugar sources available with easy access when living with T1D, like glucose or dextrose tablets, full sugar (not diet) sodas, fruit juices or even table sugar.

JDRF Canada will provide any updates on further availability of glucagon products if and when they become available.

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.