Diabetes is the most expensive chronic illness in the U.S., with $1 of every $4 in healthcare costs going to care for diabetics. The National Center for Chronic Disease Prevention and Health Promotion (NCCDPHP) estimates that $237 billion is spent directly on diabetes-related medical costs every year, while another $90 billion is lost each year due to reduced productivity associated with diabetes.
The NCCDPHP also estimates that one-third of Americans will develop diabetes at some point during their lifetime. In fact, over 37 million Americans or 11% of the population already have diabetes, representing a sizable market of patients in need of new treatment options.
One of the critical problems with diabetes is that it can create a domino effect in the body, damaging vital organs and causing other systemic problems if the patient’s blood sugar levels are not kept in check. The long-term and serious complications from diabetes, combined with the disease itself, represent a broad array of medical conditions that may require treatment.
Gene Therapy Shows Promise To Treat Diabetes
Among the many treatment options under development for managing diabetes, gene therapy is on the cutting edge. One of these technologies is already showing promise in the early stages. As a result, gene therapy could become a new treatment option for millions of diabetics who currently require insulin replacement therapy, potentially providing long-term effectiveness or even a cure for diabetes.
For example, in one study from 2013 that was later updated in 2017, researchers injected a small DNA sequence into the veins of rats with diabetes, creating cells that produced insulin. Other gene therapy treatments in development for Type 1 diabetes involve reprogramming cells so that they can produce insulin.
One company with a promising treatment in development for both Type 1 and Type 2 diabetes is Genprex, Inc., which is utilizing the reprogramming approach to diabetes gene therapy. The company’s diabetes technologies are the most recent additions to its intellectual property portfolio. They were developed by researchers at the University of Pittsburgh School of Medicine.
GPX-002 Gene Therapy For Type 1 Diabetes
Genprex’s GPX-002 therapy targets Type 1 diabetes and consists of a novel infusion process that utilizes an adeno-associated virus vector to deliver the Pdx1 and MafA genes to the patient’s pancreas. Those genes then reprogram alpha cells in the pancreas into beta-like cells capable of producing insulin.
However, those new beta-like cells are distinct enough from the patient’s actual beta cells that they are believed to evade the immune system. In Type 1 diabetes, the patient’s immune system destroys the beta cells in the pancreas that are needed to produce insulin.
So far, GPX-002 has been tested in both diabetic mouse models and nonhuman primates. The studies in diabetic mouse models indicated that an earlier version of GPX-002 not only restored normal blood glucose levels but did so for an extended period of about four months.
Researchers believe four months of restored blood glucose levels in mice could translate to decades in humans. This early data gives hope that GPX-002 could not only one day be a long-lasting treatment option for millions of diabetics requiring insulin replacement therapy but possibly even a cure.
GPX-003 Gene Therapy For Type 2 Diabetes
GPX-003 is Genprex’s gene therapy for Type 2 diabetes, which works by restoring the patient’s diminished beta cells and boosting their insulin production through the introduction of transcription factors controlled by an insulin promoter. Transcription factors are proteins that bind to specific DNA sequences to regulate the transcription of genetic information from DNA to messenger RNA (mRNA).
GPX-003 is based on the same novel approach as GPX-002.
Gene Editing For Type 1 Diabetes
Yet another potential therapy for Type 1 diabetes involves gene editing, which differs slightly from gene therapy. The goal of gene editing is to reprogram the patient’s DNA, targeting the underlying cause of Type 1 diabetes by removing the bad parts of the patient’s DNA, effectively deleting their diabetes.
ViaCyte, which was acquired by Vertex Pharmaceuticals, and CRISPR Therapeutics have been collaborating on gene editing to create, encapsulate, and implant islet cells into a patient’s body. The created islet cells would be protected from the patient’s immune system, unlike their own islet cells.
Islet cells are found in the pancreas and tasked with producing the hormones insulin and glucagon, which are then secreted into the bloodstream, where they control blood glucose levels.
Gene Therapy For A Rare Genetic Condition In Type 2 Diabetes
Finally, one other novel approach involving gene-related therapy is in development in the U.K. Diabetes U.K. is funding research into the importance of one specific gene for controlling the way the body stores fat and how this gene is linked to Type 2 diabetes.
Researchers at the University of Aberdeen published findings in 2022 about a potential gene therapy for Type 2 diabetes in patients with a rare genetic condition called congenital generalized lipodystrophy. Patients with this condition are born without the gene that tells their bodies where to store fat.
As a result, fat builds up inside vital organs like the liver instead of being stored under the skin, where it is normally stored. The result is typically insulin resistance and Type 2 diabetes.
University of Aberdeen researchers studied mice with lipodystrophy to see what would happen when they gave the missing gene to those mice. They noted that the control group containing mice that didn’t receive the gene therapy saw their weight increase by 25% to 30%.
However, when they gave another group of mice the proposed gene therapy, they observed significantly less weight gain in the mice that received the missing gene. Researchers believe the reduced weight gain was because the gene therapy reversed the mice’s enlarged livers.
They also discovered that their gene therapy increased insulin production in the treated mice when they were given sugar. The mice were also able to use the insulin more effectively, resulting in lower blood glucose levels.
Studies On Novel Treatments For Diabetes Continue
Given the large and growing size of the diabetes treatment market, it’s easy to see why this area of healthcare is a hotbed of innovation. Many studies on novel ways to treat Type 1 and Type 2 diabetes using gene therapy or gene editing continue, with some companies like Genprex, ViaCyte and CRISPR Therapeutics leading the way. Additionally, many universities and other academic institutions are participating in this race for more effective therapies.
While a cure for either type of diabetes has yet to be found, increasing investments in the space are leading to some groundbreaking discoveries that could one day change the lives of diabetics forever.
The author, Michelle Jones, wrote this article on behalf of Quantum Media Group, LLC and their client, Genprex, Inc.