Researchers at The University of Queensland 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 UQ’s
Frazer Institute has led the development of a targeted
immunotherapy drug, ASITI-201, which has been designed to
rebalance the body’s immune response to protect
insulin-producing pancreatic cells.
“In
people with Type 1 diabetes, the immune system starts to
recognise pancreatic cells as something it needs to attack,
and right now the only available treatment is insulin
replacement,” Professor Thomas said.
“We’ve
taken a new approach and developed ASITI-201 using a protein
from the pancreas, along with vitamin D to calm the immune
response.
“This potential treatment uses the immune
system’s ability to heal and has been successful in
controlling the disease in mice.”
Lead investigator
Dr Aakansha Zala said the drug candidate aims to preserve as
much pancreatic function for as long as possible in people
recently diagnosed with Type 1 diabetes, to reduce the
amount of insulin they need to administer.
“We are
looking for people over the age of 18 who have been
diagnosed with Type 1 diabetes within the last 5 years, to
participate in the trial at the Translational Research
Institute’s Clinical Research Facility based at
Brisbane’s Princess Alexandra Hospital,” Dr Zala
said.
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“We will be looking at whether the drug
changes the immune system in the way that we
anticipate.”
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 which include children, who
have a much faster progression to insulin dependence,”
Professor Thomas said.
“We’ve been developing a
similar drug with the potential to treat Rheumatoid
Arthritis, and that knowledge has supported our development
of this drug for Type 1 diabetes.”
The trial itself
is being funded by the Medical Research Future Fund via
Australia’s national biotech incubator CUREator which
helped form spin-out company Liperate Therapeutics – which
was established by UQ’s commercialisation company
UniQuest.
The drug’s preclinical proof of concept
was supported by multiple grants from Breakthrough T1D
(formerly JDRF) totalling $2.54 million between
2003-2015.
The Leona M. and Harry B. Helmsley
Charitable Trust subsequently awarded two grants totalling
$5.33 million to complete the preclinical development of the
drug candidate, including safety studies and
manufacturing.
Dr Ben Williams, Program Officer at
Helmsley Charitable Trust, said the organisation is proud to
have supported the research which is a step towards
improving the lives of people with Type 1
diabetes.
“Bringing drug candidates from the
laboratory into clinical testing is always a herculean
effort,” Dr Williams said.
The Helmsley Charitable
Trust is the largest private funder dedicated to
transforming the trajectory of Type 1
diabetes.
