Project ACT series
This article is the last part of a series exploring the different ways that Breakthrough T1D’s Project ACT (Accelerate Cell Therapies) is shaping the future of cell therapies for type 1 diabetes (T1D).
Read last month’s article about the role of Advocacy in cell therapies for T1D.
Cell therapies, which place insulin-producing cells back into the bodies of people with T1D, are making major headway in clinical trials—and people are becoming insulin independent. In past installments of this article series, we’ve explored how we got to this point at every step in the pipeline: from advocating for funding, to conducting research in laboratories, to clinical testing in humans, and to working with regulators and insurers.
But we’re not at the finish line yet. Approval of manufactured cell therapies isn’t enough to make sure that people who want these cells can get them. So, how do we fill this gap?
Medical Affairs is the backbone of clinical adoption
The answer is Medical Affairs—Breakthrough T1D’s newest mission pillar—which aims to support the clinical adoption of manufactured cell therapies. As we are soon approaching the regulatory submission of a first-generation manufactured cell therapy, Medical Affairs initiatives will be especially important in making sure that healthcare professionals (HCPs) have the tools they need to bring these therapies to clinics. By partnering with global experts and HCPs to increase knowledge and clinical competencies, the Medical Affairs team is working to ensure the future delivery of manufactured cell therapies to eligible people with T1D.
Preparation is key
Since manufactured cell therapies for T1D are not yet FDA-approved, we are at a critical moment where preparation is essential. This is why the Medical Affairs team is planning now for the eventual arrival of first-generation manufactured cell therapies. They are partnering with advanced clinical teams to determine best practices to ensure that clinics have the right resources and skillsets. They are anticipating obstacles—and finding solutions—to the adoption of manufactured cell therapies. Then, when these therapies become available, HCPs will be ready and waiting to deliver them to people with T1D.
Assembling expert clinical teams and facilities
For people with T1D to have access to manufactured cell therapies, comprehensive expert clinical care teams need to be at the ready. To make this possible, we are starting at the very beginning: in training programs.
New, updated programs and fellowships must incorporate specialized education around cell therapies (such as optimized immunosuppression regimens and pre- and post-transplant education) for the next generation of HCPs. At a time when there are too few endocrinologists and certified diabetes care and education specialists, there’s no better time than now to encourage trainees to enter the field. We need to ensure that T1D care teams have trained professionals who are up to pace with the newest cell therapy protocols.
In line with these efforts, the Medical Affairs team is also working to recognize expert multidisciplinary Centers of Reference, which are a network of T1D treatment centers that have the personnel, resources, facilities, and skills to deliver manufactured cell therapies to people with T1D with a consistent approach. These centers will act as training hubs and share outcomes to continuously improve and establish best practices as manufactured cell therapies are incorporated into clinics.
Guidelines, statements, workshops, and more
The Medical Affairs team is working with experts in the field on publications that will guide care teams to further support the standardized integration of manufactured cell therapies into clinical practice.
Clinical guidelines
Clinical guidelines are recommendations for clinicians about how to care for people with T1D. They are informed by a systemic review of evidence by experts to assess benefits versus risk of care options.
Consensus statements
Consensus statements are collective opinions of an expert panel representing the multidisciplinary care team and voice of people with T1D. These statements are developed when evidence is still being gathered.
To drive these efforts, the Medical Affairs team hosts workshops, convening international experts to collaborate and discuss ideas. Using established methodologies, these projects ensure that the final document reflects expert consensus. One of the latest workshops assembled islet cell transplantation professionals from around the world to begin developing a consensus on the clinical roadmap of cell replacement therapies—the first step of many toward clinical adoption.
Read more about the most recent Medical Affairs workshops, including one geared toward creating Centers of Reference. See a recent example of clinical guidelines for monitoring early-stage T1D.
Opportunities to learn
Through decades of research, we’ve learned a lot about T1D—and the newest therapies and treatments are as innovative and complex as ever. To keep up with the rapidly changing pace of innovation, the Medical Affairs team has put together opportunities for HCPs learn about cutting-edge advancements.
These HCP resources offer accredited, free, live or on-demand education around the latest T1D research. There is also information on upcoming conferences, published guidelines, and clinical trial opportunities, in addition to an email newsletter with regular updates. Accredited HCP education is essential to ensure that clinicians have the most up-to-date knowledge that they can share with the T1D community—an important step in clinical adoption. Cell therapy resources are currently under development and expected in the next year.
Clinical trial education and awareness
Clinical trials are a key step in ensuring that new and effective T1D treatments and cures, including manufactured cell therapies, can safely make their way to the T1D community. The Medical Affairs team is working to foster a culture of research participation by ensuring that HCPs have access to information on current clinical trials and can help to educate the T1D community on their importance.
The ultimate goal is to increase awareness about clinical trial opportunities and support shared decision-making between people with T1D and their diabetes care team about clinical trial participation. The bottom line? HCPs and the T1D community must be equipped with information to enable them to make informed choices about participating in cell therapy clinical trials.
Medical Affairs help turn groundbreaking science into real care—by supporting doctors, educating communities, and making sure cell therapies for type 1 diabetes reach the people who need them most.”
The bigger picture: Project ACT across the pipeline
In this series, we’ve explored how manufactured cell therapies are moving through every step of the pipeline. We took a deep dive into the challenges that researchers are addressing to optimize manufactured cells. We surveyed the different cell therapies that are in currently clinical testing. We reviewed how advocacy shapes funding, regulatory decisions, and access to cell therapies. Finally, we considered how to ensure the clinical adoption of manufactured cell therapies.
This is where Breakthrough T1D’s Project ACT (Accelerate Cell Therapies) comes in. The ultimate goal is to accelerate manufactured cell therapies that do not require immunosuppression—all the way from the first discovery research in the lab to the adoption of cell therapies in clinics.
Project ACT
Scientific progress takes time, money, and effort. To accelerate islet replacement therapies faster than ever, Breakthrough T1D launched Project ACT (Accelerate Cell Therapies) to simultaneously advance research, development, regulatory policies, access, and adoption of manufactured islet therapies that do not require broad immunosuppression.
At every step of the way, we are moving cell therapies through the pipeline as fast as possible. Breakthrough T1D is preparing for the day when first generation manufactured cell therapies are available, and we are working toward a future with next generations of therapies that do not require immunosuppression. Thanks to concerted efforts by Breakthrough T1D’s Research, Advocacy, and Medical Affairs teams, each day we move closer to manufactured cell therapies becoming a reality for everyone with T1D.
