Will Cell and Gene Therapy Provide a Diabetes Cure?

Published: October 2023

The global burden of diabetes is undeniable. According to the International Diabetes federation (IDF), more than 530 million individuals worldwide are currently grappling with this metabolic disease characterized by insulin resistance. Shockingly, this number is anticipated to soar beyond 640 million by 2030.1 

Despite available treatments and management strategies, a true cure for diabetes remains elusive. However, amidst this pressing challenge, the approval of first allogeneic cell therapy for Type 1 diabetes in June 2023 has kindled the hope for the fields of cell and gene therapy to provide a cure for diabetes. In this comprehensive article, we embark on a journey to explore the current landscape, ongoing clinical trials, the promise of cell and gene therapy, and the tantalizing possibility of a future where diabetes is not just managed but cured.


The Diabetes Dilemma

Understanding Diabetes

Diabetes is a multifaceted and chronic disease that significantly impacts how the human body regulates its crucial energy source, glucose. This intricate condition manifests in various forms, including type 1, type 2 and gestational diabetes; all contributing to a growing global health concern. Despite their unique characteristics, all these variants share a common thread: an imbalance in blood sugar levels. Type 1 diabetes results from the immune system mistakenly attacking the body's insulin-producing beta cells, leading to a substantial deficiency in insulin, the hormone responsible for regulating glucose levels. It is commonly known as juvenile diabetes. In contrast, type 2 diabetes primarily stems from insulin resistance, where the body's cells become less responsive to insulin signalling, resulting in elevated blood sugar. Gestational diabetes occurs during pregnancy when hormonal changes impair insulin function, posing risks to both the mother and the developing fetus. 

This shared underlying issue of blood sugar dysregulation underscores the critical importance of understanding and effectively managing diabetes, as it affects millions of lives worldwide. Advances in medical research and innovative treatments, including cell and gene therapies, offer hope for more effective management and, potentially, a cure for this complex and challenging condition.

The Quest for a Cure

The quest for a cure for diabetes has been a collective aspiration of researchers, healthcare practitioners, and individuals affected by the condition for many decades. While treatments such as insulin therapy and lifestyle adjustments have significantly improved the management of diabetes, they fall short of providing a definitive cure. A number of drug developers have also developed self administration devices to ease the daily delivery of insulin. The global diabetes drug delivery devices market,   estimated to be worth a staggering $13 billion, reflects the demand for more convenient options for tackling this chronic disease. However, the need for a definitive cure has fueled relentless efforts to explore innovative approaches that could fundamentally transform the way diabetes is treated. Among these approaches, cell and gene therapy have emerged as frontrunners, holding the promise to not only manage the symptoms of this disease, but potentially eliminate its root causes. These therapies offer new hope for individuals living with diabetes, marking a pivotal moment in the ongoing pursuit of a cure that could enhance the quality of life for millions around the world. As research continues to advance, the dream of a cure for diabetes moves closer to becoming a reality, with cell and gene therapy leading the way in the quest to conquer this complex and pervasive condition.


Allogeneic and Autologous Cell Therapy: A Potential Game Changer

Pancreatic Islet Cell Replacement Therapy: Breakthroughs and Clinical Trials

The first breakthrough for cell therapy in diabetes treatment came in July 2023, when FDA approved CellTrans Inc.’s allogeneic cell therapy, Lantidra for the treatment of adults with type 1 diabetes who are unable to approach target glycated hemoglobin because of current repeated episodes of severe hypoglycemia.2 Lantidra cell therapy consists of a single infusion of pancreatic cells from deceased donor into the hepatic portal vein of the patient. Although details on the launch plans are not available yet, the approval has sparked interest in other cell therapies in clinical trials. Researchers worldwide are diligently conducting clinical trials to assess both the safety and efficacy of innovative approaches. 

Vertex Pharmaceuticals is another company with islet cell replacement therapy in clinical trial. Interim results were presented from a phase 1 / 2 clinical trial of Vertex Pharmaceuticals’ VX-880 at the American Diabetes Association 83rd Scientific Sessions in June 2023. VX-880 is stem-cell derived, fully differentiated islet cells that are infused into the hepatic portal vein of Type 1 diabetes patients.

Overcoming the Challenges with Immunosuppression

Both Lantidra and VX-880 are used in conjunction with concomitant immunosuppression to protect the islet cells from immune rejection. The long-term use of immunosuppressants put the patients at risk of severe infection. Companies like Vertex and Sernova have come up with solutions to avoid the use of immunosuppressants. Vertex encapsulated the pancreatic islet cells in an immunoprotective device and initiated clinical trials for this therapy VX-264 in May 2023. Another US based company, Sernova licensed a conformal coating technology from Dr. Alice Tomei at the University of Miami to use with its Cell Pouch device with the aim to eliminate the long term use of immunosuppressants with islet cell replacement.

Stem Cells for Re-educating the Immune System

Stem cells, often referred to as the body's "master cells," hold the remarkable ability to differentiate into various cell types. In the context of diabetes, stem cell therapy presents immense promise. Researchers are actively exploring the potential of using stem cells to regenerate pancreatic beta cells, which are responsible for producing insulin and are frequently damaged or destroyed in diabetes. This innovative approach offers the potential to restore insulin production and fundamentally transform the way diabetes is managed, offering hope for a future where the disease can be effectively treated if not completely cured.

US based Throne Biotechnologies is evaluating an autologous cell therapy, Stem Cell Educator Therapy, in phase 2/3 clinical trial. This therapy uses the power of CB-SC stem cells derived from human umbilical cord blood to re-educate the patients white blood cells to their pre-diseased states. Another company working with umbilical cord tissue derived stem cells is Sweden based, NextCell. The company is evaluating ProTrans, an allogeneic umbilical cord tissue derived mesenchymal stromal cells, as a therapy to slow the progression of type 1 diabetes. The therapy is infused in a 40 minute session and is in phase 1 / 2 clinical trial in adult patients newly diagnosed with Type 1 diabetes.4 NextCell is also evaluating ProTrans in phase I clinical trials in young patients aged 7-18, who are the most potential candidates for the therapy considering Type 1 diabetes is mainly diagnosed in early teenage years.5 The latest company to begin a clinical trial for stem cell therapy is Creative Medical Technology Holdings, which initiated a phase 1 / 2 clinical trials for its allogeneic therapy, AlloStem, in Type 1 Diabetes in April 2023.

While navigating challenges is an inherent part of this journey, it's heartening to note that there have been notable breakthroughs. Some trial participants have witnessed substantial enhancements in insulin production and blood sugar regulation, instilling hope that stem cell therapies could indeed revolutionize diabetes management and pave the way toward effective treatment, if not an eventual cure for this chronic condition.


Gene Therapy: Rewriting the Genetic Code

Gene Editing and Diabetes

Gene therapy, a revolutionary frontier in medical science, has the potential to rewrite the genetic narrative of diseases, and diabetes is no exception. At its core, diabetes often harbours genetic factors that play a pivotal role in susceptibility and progression. Gene therapy stands as a formidable ally in the quest to address the root causes of diabetes. By precisely targeting and correcting the genetic anomalies responsible for the disease's onset and progression, gene editing technologies, such as CRISPR, offer the promise of transformative treatments. These therapies aim not only to manage symptoms but to fundamentally alter the course of the disease, paving the way for a future where diabetes is not just controlled but potentially cured. As research and clinical trials in gene therapy continue to advance, the prospect of a definitive solution for diabetes is increasingly within reach, igniting hope for millions worldwide grappling with this chronic condition.

The Promise of CRISPR in Diabetes Treatment

The emergence of CRISPR gene editing therapies and technology has ushered in a new era of precision medicine, reshaping the landscape of gene therapy with its extraordinary accuracy and versatility. Within the context of diabetes, where genetic factors play a significant role, CRISPR holds immense promise. Its capacity to meticulously target and modify specific genes not only offers unparalleled precision but also the potential to rectify the genetic mutations underpinning the disease. This groundbreaking capability opens the door to a future where diabetes is not merely managed or ameliorated but definitively cured. As research and innovation in CRISPR technology continue to advance, the tantalizing prospect of a permanent solution for diabetes moves closer to realization, bringing newfound hope to individuals affected by this chronic condition and marking a significant stride toward a healthier future for all. CRISPR Therapeutics in collaboration with ViaCyte is developing multiple gene modified allogeneic stem cell derived beta cell replacement therapy using CRISPR Cas9 technology. Unlike the cell therapies, CRISPR Therapeutics is targeting both Type 1 and Type 2 diabetes with its gene-edited beta cell replacement therapy. Their lead gene edited therapy, VCTX210 has completed phase 1 clinical trial recently.6 Another company, Genprex is using AAV vector to deliver Pdx1 and MafA genes directly to the pancreas.7 Their therapy DIA-001 is currently in preclinical stages.

Gene therapies for diabetes treatment are currently in early clinical development. Clinical trials serve as the linchpin of medical progress, subjecting innovative treatments to the crucible of rigorous examination, where their safety and efficacy are scrutinized and established. By meticulously evaluating the effectiveness and safety of these groundbreaking therapies, clinical trials not only inform medical practice but also offer hope for individuals grappling with diabetes. They represent the final, vital step in the journey to bring revolutionary treatments from the realm of theory to the realm of tangible and life-changing solutions.


Beyond Cell and Gene Therapy

Bariatric Surgery and Diabetes

Bariatric surgery, long recognized for its effectiveness in aiding weight loss, has taken on an unforeseen role as a formidable ally in the ongoing fight against diabetes. Remarkably, certain types of bariatric procedures have demonstrated the capacity to bring about substantial improvements in diabetes control, and in some instances, even full remission of the disease. This unexpected connection underscores the intricate relationship between obesity, weight management, and diabetes. Bariatric surgery operates not only as a means to reduce excess body weight but also as a catalyst for physiological changes that positively impact insulin sensitivity and blood sugar regulation. As research continues to delve deeper into these mechanisms, bariatric surgery is evolving from a weight-centric intervention into a multifaceted tool that holds the potential to transform the lives of individuals grappling with diabetes, offering newfound hope and an avenue towards improved health and well-being.

Weight Loss and Lifestyle

Weight management and lifestyle adjustments continue to stand as pivotal pillars in the comprehensive approach to diabetes management, particularly in cases of type 2 diabetes. The intricate interplay between excess body weight, dietary choices, and physical activity levels holds a profound influence on blood sugar regulation. Achieving and sustaining a healthy weight through tailored lifestyle modifications, coupled with dietary alterations and a commitment to regular physical activity, can yield tangible improvements in diabetes control. For many individuals, these changes are transformative, offering the potential not only to mitigate the symptoms but also to achieve remission of the disease. This holistic approach underscores the significance of empowering individuals with the knowledge and tools to actively manage their diabetes, fostering a sense of agency and well-being while holding the promise of a brighter, healthier future for those affected by this chronic condition.

The Artificial Pancreas

The advent of the artificial pancreas represents a remarkable leap in the realm of diabetes care. This bioengineered system ingeniously replicates the intricate functions of the natural pancreas, merging real-time continuous glucose monitoring with automated insulin delivery. The result is a transformative approach that offers a tantalizing glimpse into a future where diabetes is not merely managed but, rather, effectively cured. This innovation promises to liberate individuals with diabetes from the constant vigilance of monitoring and manual insulin administration, fostering greater independence and improving overall quality of life while advancing the dream of a world free from the burdens of this chronic condition. The first artificial pancreas received approval in 2016, with a total of six such systems now available for type 1 diabetes.8

Conclusion: The Pursuit of a Diabetes Cure

In conclusion, the quest for a diabetes cure is a multifaceted journey that encapsulates the resilience of the human spirit, the power of scientific innovation, and the collective commitment to improving lives. Diabetes, a global health challenge of monumental proportions, necessitates a comprehensive approach that encompasses cutting-edge therapies like cell and gene therapy but also emphasizes the significance of lifestyle modifications, weight management, and collaborative efforts among researchers, healthcare professionals, and pharmaceutical companies. As clinical trials progress and the boundaries of medical science expand, the dream of a diabetes cure inches closer to reality. In this relentless pursuit, hope remains the driving force, uniting individuals worldwide in their determination to transform diabetes from a chronic condition to a conquered challenge. 

The landscape of ongoing research in the pursuit of a diabetes cure is marked by resilience, dedication, and unwavering hope. Numerous clinical trials are at the forefront, diligently exploring diverse cell and gene therapy approaches. These trials, conducted by researchers and institutions worldwide, are fueled by the urgent need to revolutionize diabetes care. While challenges loom, such as the imperative for long-term safety data and the rigorous processes of regulatory approvals, the flame of hope burns brighter than ever. It is this hope that propels scientists, healthcare professionals, and individuals living with diabetes forward in their shared quest for a definitive solution. With each clinical trial that progresses, the possibility of a diabetes cure edges closer to reality, offering reassurance and optimism to millions affected by this chronic condition and paving the way for a future where diabetes is a conquered challenge rather than an enduring burden.

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Sources:1 https://idf.org/about-diabetes/diabetes-facts-figures/
Sources:2 https://www.fda.gov/news-events/press-announcements/fda-approves-first-cellular-therapy-treat-patients-type-1-diabetes
Sources:3 https://www.clinicaltrials.gov/study/NCT04011020
Sources:4 https://www.clinicaltrials.gov/study/NCT03973827
Sources:5 https://news.cision.com/nextcell-pharma-ab/r/first-pediatric-patients-treated-with-protrans-in-type-1-diabetes-phase-ii-study,c3688461
Sources:6 https://classic.clinicaltrials.gov/ct2/show/NCT05210530
Sources:7 https://www.genprex.com/news/genprex-announces-groundbreaking-data-from-non-human-primate-study-evaluating-novel-gene-therapy-to-treat-type-1-diabetes-at-16th-annual-international-conference-on-advanced-technologies-tr/
Sources:8 https://www.jdrf.org/blog/2023/05/22/fda-clears-new-artificial-pancreas-system/


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