Breakthrough T1D’s newest mission pillar, Medical Affairs, is bridging the gap between access to and adoption of T1D therapies. The establishment of this program is essential to Project ACT (Accelerate Cell Therapies): Breakthrough T1D’s initiative to accelerate the development of manufactured islet cell replacement therapies that do not require immunosuppression. The goal is to make sure that people with type 1 diabetes (T1D) can get these therapies as soon as they hit the market.
The field is moving quickly: people are becoming insulin-independent in cell therapy clinical trials. We are advancing towards the submission of the first-generation manufactured islet cell therapy that requires immunosuppression, Vertex’s zimislecel (VX-880). We are at a critical moment and need to act now to ensure that healthcare providers (HCPs) are ready to bring manufactured islet cell therapies into clinical settings.
Enter Medical Affairs
This is where Medical Affairs comes in. The team, led by Thomas Danne, M.D., Chief Medical Officer International, is working with the medical community to anticipate obstacles to getting manufactured islet therapies into clinics and find ways to overcome them now.
To accomplish this, Breakthrough T1D recently hosted two cell therapy workshops, convening multidisciplinary, international experts in islet cell transplantation to discuss a clinical roadmap for manufactured islet cell therapies—and how to ensure that clinical teams are in place and prepared to provide these therapies to people with T1D who qualify. By preparing now, we can get this first-generation therapy into the hands of people with T1D as soon as possible after regulatory approval.
The cell therapy workshops were hosted by Breakthrough T1D’s Thomas Danne, M.D., Chief Medical Officer International, and Anastasia Albanese-O’Neill, Ph.D., APRN, CDCES, Vice President of Medical Affairs.
Read on to learn more about the cell therapy workshops, the attendees, and what each accomplished.
Workshop #1: Who may benefit most from manufactured cell replacement therapies
Key Takeaways
- T1D leaders are working on a five-year roadmap for the clinical adoption of manufactured islet cell therapies.
- It will include key criteria and evidence to help clinicians determine who is the best fit for manufactured islet cell therapies.
- The guidelines are prioritizing shared decisions between people with T1D and their care team to optimize outcomes and maximize long-term health.
Attendees
The first of the cell therapy workshops, held in late April, convened transplant surgeons, T1D clinicians and researchers, a member of Vertex’s leadership team, a member of Breakthrough T1D’s Participant Advisory Council to represent people with a lived experience of T1D, and Breakthrough T1D Vice President of Research Esther Latres, Ph.D.
The purpose of this workshop was to start developing a five-year roadmap to help guide the T1D care community to support the adoption of manufactured islet cell therapies in clinical care. The evidence-based recommendations will be vetted by a larger group of clinical experts, diabetes organizations, and people with lived experience with T1D to ensure there is broad agreement. The consensus document will ultimately be published to expand its reach.
This process will summarize the essential evidence that will help HCPs decide who may benefit the most from manufactured islet cell replacement therapies. These decisions will take into account the perspective of people with T1D and differences in age, hypoglycemia unawareness, or kidney health, to name a few. This can help HCPs better understand the benefits versus risks for manufactured cell therapies on a person-by-person basis—making sure that each clinical decision is made jointly to prioritize long-term health.
Workshop #2: Pilot workshop to develop international Centers of Reference for T1D cell therapy
Key Takeaways
- Centers of Reference are expert T1D care centers that are preparing to bring manufactured islet cell therapies to the T1D community.
- This workshop was the first step to understanding what kind of education, training, and resources are needed for Centers of Reference to be effective.
- Attendees discussed how to make sure people T1D who receive manufactured cell therapies at Centers of Reference will have the best clinical experience possible.
Attendees
The second cell therapy workshop, held in early May, convened clinicians from various global medical institutions, including University of Minnesota Medical Center, University of Wisconsin Health Transplant Center, the Penn Rodebaugh Diabetes Center, University of Chicago Medicine, IRCCS Ospedale San Raffaele (Italy), Institute of Transplantation, Newcastle upon Tyne (United Kingdom), and University of Alberta (Edmonton, Alberta, Canada).
Additional attendees from Breakthrough T1D included CEO Aaron Kowalski, Ph.D., Vice President of Research Esther Latres, Ph.D., the Medical Affairs team, a volunteer, and a member of the Participant Advisory Council, who is a person living with T1D.
The objective of this workshop was to take the first step toward creating Centers of Reference for T1D manufactured cell therapies. “The initial purpose is to accelerate readiness of healthcare professionals to deliver manufactured islet cell therapies once they become available,” explained Dr. Danne. “…making such a treatment a success needs teamwork. Accredited Centers of Reference will not only deliver advanced T1D treatments but also serve as a training hub for professionals aspiring to become experts.”
This workshop focused on better understanding what potential Centers of Reference need to be successful. The attendees covered a range of topics: what an ideal T1D care team might look like, the education and training required for experts in T1D manufactured islet cell therapy, and career development for early-stage T1D professionals.
The goal is to prepare expert clinicians—who are already doing islet cell transplants—to bring manufactured islet cell therapies into clinical practice at their institutions and others, once they have regulatory approval. These centers will serve as a benchmark for best practices in T1D manufactured cell therapy, establishing a network of expert teams to make sure that everyone who can benefit from manufactured cell replacement therapy is given the opportunity to consider it.
What the experts are saying
“We need to build consensus and teamwork. When manufactured cell therapies exist, it’s going to take significant coordination between endocrinologists, transplant surgeons, and people with T1D to ensure as many people as possible are benefiting from these therapies.”
Jon Odorico, M.D.
Professor of Surgery and Director of Pancreas and Islet Transplantation at University of Wisconsin Health Transplant Center
“We’re trying to solve access and awareness. There’s a definite gap between primary care endocrine diabetes specialists and transplant specialists…there’s so much more that we have to fill in for [people with T1D].”
Helen Nelson, BSN, RN, CCTC, CPTC
Program Manager, Organ Allocation/Clinical Triage and Pancreas Transplant Program at University of Wisconsin Health Transplant Center
“It’s going to be a significant problem if we have a cure but no one has access to it because no one can deliver it. We must work together—transplant surgeons, endocrinologists, researchers, everyone. It’s like building Cape Canaveral in anticipation of sending rockets into space.”
Peter Senior, MBBS, Ph.D.
Islet Transplant Endocrinologist, Professor in the Department of Medicine, and Director of the Alberta Diabetes Research Institute at the University of Alberta, Canada
“There is amazing excitement around creating cell therapies. People are excited about it. We must ensure that organizations like Breakthrough T1D bridge the gap between research and the T1D population so that there is no difference between an individual’s reality and what therapies are available.”
James Shaw, M.D., Ph.D.
Transplant Endocrinologist and Professor of Regenerative Medicine for Diabetes at the Institute of Transplantation, Newcastle upon Tyne, United Kingdom
This is just the beginning
Manufactured islet cell therapies are coming. We need teamwork to get these therapies into clinics so people with T1D don’t have to wait years to get them. This is why Breakthrough T1D is acting now: when the first manufactured islet cell therapy hits the market, multidisciplinary care teams around the world will be ready. These workshops—the first of many—will help accelerate the safe and effective integration of manufactured islet cell therapies into clinics.
“This way we will ensure that the medical community is ready to deliver manufactured cell therapies once they become more widely available.,” Dr. Danne said. Thanks to the hard work of the Medical Affairs team at Breakthrough T1D, this goal is in sight.
