An Endocrinologist’s Unique Journey Helping with People Living with Type 1 Diabetes

by Dr. Tina Gupta, MD

I went into medicine to help as many patients as possible, and my passion for diabetes care led me to specialize in endocrinology. Over the years, it was amazing to see how my patients living with type 1 diabetes (T1D) benefitted from advancements in the field of diabetes medicine. They were living in a world with rapid-acting insulins, continuous glucose monitors (CGM), insulin pumps, and hybrid closed-loop systems. However, despite access to these therapies and technologies, some of my patients were not able to achieve the clinical outcomes we desired.

I saw the struggles my patients experienced and the burden that T1D posed on their daily lives, and I often thought about what more I could do to help. After a difficult decision, I left clinical practice and joined Vertex Pharmaceuticals, a Boston-based biotech company, whose efforts are aimed at treating serious diseases, such as T1D. You can read more about Vertex’ commitment to understanding life with T1D here.

Vertex Pharmaceuticals and the Legacy of Semma Therapeutics

Vertex began research in the T1D space in 2019 with the acquisition of Semma Therapeutics, but the real story goes back decades earlier. The founder of Semma Therapeutics was Dr. Doug Melton. At the time he founded the company, he was at Harvard University where he had spent many years focused on researching pancreatic islet cells that are implicated in T1D. Motivating this commitment was the fact that, more than 30 years prior, Dr. Melton’s infant son was diagnosed with T1D (his daughter would also be diagnosed years later).

Dr. Melton’s wife was carrying the daily burden of the disease for their family, working nonstop to monitor and manage the relentless demands of T1D for their children. Meanwhile, Dr. Melton worked tirelessly in the lab researching how to differentiate stem cells into islet cells with the hope of replacing the insulin-producing cells that have been destroyed in people with T1D.

 Doug Melton, PhD (third from left) joined the Vertex team in May 2022

Vertex’s Clinical Diabetes Trials: Breakthroughs in Islet Cell Research

After decades of research, Dr. Melton and his team found a way to create islet cells in the lab that respond to glucose to produce insulin, and he knew that bringing this work into clinical trials was the next big challenge. By acquiring Semma, Vertex was able to advance this investigational research into two ongoing clinical trials, both currently enrolling adults living with T1D in the U.S. and globally. You can learn more about these clinical trials and promising data on the Vertex T1D islet cell study here.

Lab-Manufactured Islet Cells Stained for Visibility

During my time seeing patients, I was at ease talking about HbA1c, CGM metrics such as time in range, and treatment options such as insulin pumps. However, when I first began learning about cell therapy and cell encapsulation, it felt like I was reading science fiction. The concept that one could potentially replicate endogenous insulin production for someone living with T1D was interesting. Realizing that this science is currently being researched, and that I have a chance to be part of the effort to potentially bring this to patients if approved has been incredible.

Patient-Centered Drug Development

One unique aspect about Vertex’s drug development process is that it always begins with the individuals living with the disease we seek to treat. For those of us working on the T1D research program, people living with T1D are at the heart of everything we do, and we seek to learn from them in a variety of ways to help inform our work. We start by establishing relationships and building trust, by learning from as many people living with T1D as possible, by engaging with their caregivers and healthcare teams, and by learning from patient advocacy groups. To this end, we have a T1D patient advisory panel that meets throughout the year to provide insights and feedback to our team on a variety of topics so we can better understand the patient experience, their needs, and how we can help support the T1D community.

Vertex’s commitment to patients has been the guiding light that has helped us progress to where we are today. I share that commitment and know it is my responsibility to take what I learned from my patients, mentors, and colleagues and amplify their voices and needs. Vertex truly understands the importance of going beyond research and development to engage in advocacy, awareness, and community support. As we advance our investigational work in T1D, we will continue to look to the patient and healthcare professional community for education, inspiration, and motivation.

A Hopeful Future

No longer getting to care for my patients directly has been difficult. However, as Vertex continues to research treatments for those living with T1D, I remember their stories and hold hope that the work we’re doing today will someday have a positive impact on their future.

Editor’s Note: Vertex Pharmaceuticals was a partner of Taking Control Of Your Diabetes® at the time of publication, but TCOYD® maintains control over editorial content. 

Additional Resources:

Promising Data on Vertex T1D Islet Cell Study, Still Recruiting