Lessening the Burden of Type 1 Diabetes with Innovative Cell Therapies
By: Manasi Sinha Jaiman, M.D., M.P.H., Vice President, Clinical Development
Imagine an 18-year-old walking into my office. She is looking forward to graduation. Feeling the thrill of receiving that college acceptance letter. Planning gatherings with friends for summer before heading away to school. Then I deliver the diagnosis – Type 1 diabetes (T1D): That patient’s life will never be the same. Now, she’ll need to think about blood glucose constantly. Temporarily, her attention will shift. Her daily life will be focused solely on monitoring, tracking, and administering insulin all day, every day.
She needs to check glucose levels. Take insulin. – Think about the meals planned for today. Count every carbohydrate. Take more insulin. – If there is stress about taking a big exam, that can impact blood glucose so check levels again. – Go to the gym. Carefully track the minutes of exercise. Eat a snack before but time it just right. – Check levels again and again throughout the day. If it’s high, take insulin. If it’s low, eat some carbohydrates. – Plan the night so that she does not go low while she is asleep. –
While there have been transformative advances to improve management of T1D, insulin administration remains the standard of care for patients living with T1D. Even with insulin, patients are impacted by increased risk of developing macro and microvascular complications including cardiovascular disease, nerve damage, kidney damage, retinopathy, skin conditions, and mental health issues. Diabetes is a debilitating disease and the physical, financial, and emotional impacts of T1D along with its continuous care extend beyond the patients to family, friends, and loved ones. The requirement for constant disease management can be a substantial burden for patients with T1D and new approved treatment options are greatly needed to improve quality of life.
As a clinician, I have first-hand experience seeing the significant daily effort required for my patients to control blood glucose. Impacting patient lives motivates our team and drives us to continue to advance our therapy. Currently, there is no widely available and approved treatment that completely dissolves the need for patients to be involved with their day-to-day care. Continuous glucose monitoring and closed-loop technology have decreased some of the burden but still require patients to continue to engage with the devices frequently. What patients need is a functional cure – one that is able to restore the body’s ability to produce insulin and allow precise glycemic control. Transformative cell therapies have the potential to achieve this functional cure.
To that end, gene and cell therapy companies are exploring treatments toward the goal of allowing patients to truly live independently, maintain normoglycemia, and attain insulin independence without the need for constant personal intervention. Islet cell replacement is one promising regenerative medicine-based approach with the potential to achieve the long sought after functional cure for T1D.
In recent years, stem cell-derived islet cell replacement comprising pancreatic endocrine cells delivered to patients either directly or within a retrievable device is emerging as a promising alternative to address the challenges of cadaver islet cell transplants.[i] This therapy is designed to effectively control blood glucose levels, decrease the risk of hypoglycemia, and mitigate short-term and long-term diabetes-related complications. Unlike insulin alone, stem cell-derived islet cell replacement is engineered to enable production of insulin and glucagon (the counter-regulatory hormone) offering the potential for better control of blood glucose balance. Additionally, use of stem cells provides an unconstrained supply to generate pancreatic endocrine cells, opening the door to wider availability of islet cell replacement therapies.
One drawback common to cell therapy approaches is the requirement for immunosuppression to prevent rejection of the cells by the body. Companies are addressing this challenge with engineering of implantable devices to encapsulate the islet cells and gene-engineering the islet cells to make them capable of evading the immune system. These approaches can eliminate the need for immunosuppressive treatments making the use of islet cell replacement therapies easier for patients.
As studies progress, researchers aim to further optimize engraftment and function of cell replacement transplants providing durable physiological responses. Through engagements with patients, I have observed their interest in being part of these new solutions for T1D. For them, experiencing the effect of diabetes and autoimmune conditions on generations of their family members is motivating for their participation in clinical trials. Currently, stem cell-derived islet cell replacement therapies are being advanced from discovery through to clinical trials with the goal of offering patients a path toward insulin independence by enabling more effective glycemic control.
My job as a physician and a mission that translates to my role in leading ViaCyte’s clinical development, is to give patients a path to feel there is hope, management, and improvements coming their way for T1D. I am proud to be part of the team at ViaCyte leading the way in the clinic for stem cell-based islet cell replacement therapies in ongoing Phase 2 trials. Combining technical innovations and engineering approaches, cross-discipline collaboration, scalable manufacturing, and the drive to improve patient quality of life will propel the field forward in advancing optimized T1D cell therapies. These optimized therapies have potential to meet the needs of patients and address existing treatment limitations to lessen the burden of their disease. Though current clinical programs for islet cell replacement therapies are focused on T1D, there is a hope that once proven successful for T1D this approach could be advanced to treat insulin-dependent type 2 diabetes as well.
The field of T1D therapies is active and growing as we are working to alleviate the need for constant management of T1D with a functional cure, changing the paradigm of diabetes treatment completely.
Manasi Jaiman, MD, Vice President of Clinical Development at ViaCyte will share additional insights about the work ViaCyte, Inc. is doing to lessen the burden for patients with T1D during her presentation as part of the World Medical Innovation Forum panel on ‘Diabetes: Grand Challenge’ Thursday, May 20. More information on the event can be found at worldmedicalinnovation.org.
Author Bio: Dr. Manasi Sinha Jaiman is a pediatric endocrinologist with extensive experience in T1D research from industry and academia, including working on the bionic pancreas trials at Massachusetts General Hospital and T1 and T2 drug and device development at Covance, and now leading the effort toward a functional cure in T1D as Vice President of Clinical Development at ViaCyte. Her patient experience has informed not only her drive to find a cure but also her understanding of what that would mean to patients and families.
About ViaCyte: ViaCyte is a privately held clinical-stage regenerative medicine company developing novel pluripotent stem cell-derived cell replacement therapies to address chronic human diseases with an initial focus on type 1 diabetes. The Company is the first to advance human stem cell-derived islet cell replacement therapy into clinical trials for type 1 diabetes to provide a functional cure and reduce the burden of constant disease management for patients. For more information, please visit www.viacyte.com and connect with ViaCyte on Twitter, Facebook, and LinkedIn.
i – de Klerk E and Hebrok M. Stem Cell-Based Clinical Trials for Diabetes Mellitus. Front Endocrinol (Lausanne). 2021; 12: 631463.
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