World’s-First Gene Therapy Developed for Type 1 Diabetes

Gene therapy for diabetes gets a funding boost from the Australian government. Shutterstock

The Australian government has given a funding boost to an Australian biomedical research organisation, the Garvan Institute of Medical Research, to conduct clinical trials for the world’s first gene therapy for type 1 diabetes.

The therapy was also developed thanks to the government’s ten-year $600 million (US$385 million) research accelerator program, targeted at addressing chronic and complex diseases.

This will be the first time that genetically engineered pancreatic cells will be transplanted into humans to treat type 1 diabetes.

Instead of using drugs or surgery, gene therapy is an experimental technique that uses genes to treat or prevent disease. It alters a person’s genetic makeup by carrying genetically engineered DNA molecules into a patient.

Lead researcher at Garvan Institute Prof. Shane Grey said that the clinical trial is the beginning of a long but exciting journey.

“We’ve worked on this research for 20 years and to see it now reach a stage of translating to people is amazing,” Grey said in a release on Oct. 31.

To engineer the cells, the research team identified that a key protein involved in inflammation and autoimmune disorders known as A20 could be used for controlling the immune system.

They found that the protein could be used in the genetic engineering of islet cells to produce insulin.

“A20 is like a thermostat for the immune system; it can turn it down to a simmer or ramp it up to be more aggressive,” Grey said.

“For type 1 diabetes, we can use it as a handbrake on the immune system to stop the damage to pancreatic islet cells.”

Grey and his team showed that genetically engineered cells in mouse models had 80-100 percent survival of the islet grafts, without the need for heavy immunosuppression drugs to keep the immune system from attacking the body.

“The genetically engineered cells seem to re-educate the immune system to accept the transplant as self. It’s a really amazing discovery—that the transplant can tweak the whole immune system,” he said.

A Novel Technique With Unknown Risks

There has been an acceleration in gene therapies since the COVID-19 outbreak, with recent research looking at using gene therapy to treat head and neck cancer, sickle cell anaemia, rare neurological disorders, and congenital blindness as future therapies.

However, despite the potential benefits of the therapy, there are significant risk factors that go hand in hand with gene therapy.

Osteopathic physician Joseph Mercola said that while gene therapy can lessen symptoms of diseases, it is an experimental technique that changes an individual’s genetic coding, turning a person into “a viral protein factory that has no off-switch.”

According to MedlinePlus.gov: “Gene therapy is a promising treatment option for a number of diseases, [but] the technique remains risky and is still under study to make sure that it will be safe and effective. Gene therapy is currently being tested only for diseases that have no other cures.”