Trial may bring hope to children with Duchenne muscular dystrophy

Australian researchers are at the forefront of a new clinical trial that may bring hope to children with Duchenne muscular dystrophy (DMD) and their families.

The phase II trial, funded by Antisense Therapeutics, will test a potential new drug for DMD in children who have become wheelchair-bound by the disease. For these patients, there are few existing treatments and the disease usually leads to progressive loss of muscle function over time.

Murdoch Children’s Research Institute (MCRI) is leading the Australian arm of the international study, which also includes The Children's Hospital at Westmead in Sydney and the Queensland Children’s Hospital, as well as hospitals in the UK, Bulgaria and Turkey.

MCRI’s Dr Ian Woodcock, a paediatric neurologist, recently enrolled the first Australian patients into the clinical trial to test the new therapy called ATL1102. The drug, developed by scientists in Melbourne, targets the chronic inflammation which is thought to be responsible for much of the deterioration experienced by patients with DMD. The trial will assess the safety, tolerability and potential efficacy of the drug in boys aged 10-18 years.

Dr Woodcock said, “there is a substantial unmet need for new treatments in Duchenne muscular dystrophy. My colleagues and I have seen encouraging early-stage data with this therapy and are hopeful that it may have a future role to play in the treatment of this challenging disease.”

It is expected that the study will enrol about 12 patients with Duchenne muscular dystrophy in Australia, with recruitment due to complete in the first quarter of 2024.

About 1,000 people have Duchenne muscular dystrophy in Australia. The genetic disease, which almost exclusively affects boys, results in progressive muscle weakness, leaving most patients wheelchair-bound by their teenage years. Life expectancy for these patients is in the mid-twenties.

Results of the ongoing trial are expected to be available in the second half of 2024. If positive, the trial may lead to discussions with the Therapeutic Goods Administration regarding potential marketing approval.

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Dr Ian Woodcock, MCRI neuromuscular disorders researcher

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About

Murdoch Children's Research Institute is the largest child health research institute in Australia committed to making discoveries and developing treatments to improve child and adolescent health in Australia and around the world. They are pioneering new treatments, trialling better vaccines and improving ways of diagnosing and helping sick babies, children and adolescents. It is one of the only research institutes in Australia to offer genetic testing to find answers for families of children with previously undiagnosed conditions.

Funding

The study was funded by Antisense Therapeutics.