UQ begins first human trial of immunotherapy for type 1 diabetes

UQ begins first human trial of immunotherapy for type 1 diabetes

University of Queensland researchers have dosed 5 participants in the first clinical trial of a potentially revolutionary immunotherapy drug to treat type 1 diabetes.

Professor Ranjeny Thomas AM from UQsThe Frazer Institute led the development of a targeted immunotherapy drug ASITI-201 that balanced the body's immune response to protect insulin-producing pancreatic cells.

“In people with type 1 diabetes, the immune system recognizes pancreatic cells as something it needs to attack, and at the moment the only treatment available is insulin replacement,” Professor Thomas said.

“We took a new approach and developed ASITI-20101 using a protein from the pancreas and vitamin D to calm the immune response.

“This potential treatment harnesses the immune system’s ability to heal and is successful in controlling the disease in mice.”

Lead InvestigatorDr. Aakansha Zala said the drug candidate is intended to preserve as much pancreatic function for as long as possible in people recently diagnosed with type 1 diabetes, reducing the amount of insulin they need to manage it.

“We are looking for people over the age of 18 who have been diagnosed with type 1 diabetes in the last 5 years to take partThe study took place at the Translational Research Institute clinical research facility at Brisbane's Princess Alexandra Hospital," Dr Zala said.

“We will study whether the drug changes the immune system in the way we expect.”

Type 1 diabetes affects more than 120,000 Australians and usually develops in children and young adults.

“Ultimately we need to move to larger trials that include children who have a much faster progression to insulin dependence,” Professor Thomas said.

“We have developed a similar drug to treat rheumatoid arthritis, and this knowledge has supported our development of this drug for type 1 diabetes.”

The trial itself is funded by the Medical Research Fund through the Australian National Biotech Incubator Curator, which helped launch the therapeutics spin-out company from UQ's commercialization company Uniquest.

The drug's preclinical concept was supported by multiple grants from Breakthrough T1D (formerly JDRF) totaling $2.54 million between 2003 and 2015.

The Leona M. and Harry B. Helmsley Charitable Trust subsequently awarded two grants totaling $5.33 million to complete the drug candidate's preclinical development, including safety studies and manufacturing.

Dr. Ben Williams, program officer at Helmsley Charitable Trust, said the organization was proud to support research, which is a step towards improving the lives of people with type 1 diabetes.

“Incorporating drug candidates from the laboratory into clinical testing is always a Herculean effort,” said Dr. Williams.

The Helmsley Charitable Trust is the largest private funder dedicated to transforming the trajectory of type 1 diabetes.

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