Motor Neurone Disease researchers see promising results from the Lighthouse Project – a trial investigating the effectiveness a re-purposed HIV therapy for MND patients.

A world-first trial based at four Melbourne and Sydney sites, known as the Lighthouse Project, has just been completed, with preliminary analysis emerging.

The study focused on participants who had been diagnosed with the sporadic form of amyotrophic lateral sclerosis (ALS). Sporadic ALS has no direct cause and is classified differently to familial MND where the disease is directly inherited genetically from a patient’s parents.

The Lighthouse Project aimed to test whether sporadic motor neurone disease (MND) is caused or triggered by human endogenous retroviruses, and is the first research in the world to use modern combination anti-retroviral therapy in patients with MND.

Specifically, the study aimed to determine the safety and tolerability of an anti-retroviral therapy called ‘Triumeq’ and provide preliminary data on whether it is able to slow down the progression of MND. Triumeq is already used to treat HIV infection safely and effectively, and is produced by GSK.

“The trial is about re-purposing of a medicine from HIV to slow the progression of motor neurone disease,” said Professor Dominic Rowe, a leading MND researcher and neurologist at Macquarie University Hospital.

“Our last patient completed the trial in January this year, and we now doing analysis of the data.

“Initial indications suggest that, for a proportion of participants, the therapy slowed the progression of MND quite significantly. We are hoping that up to around 30 per cent of patients with sporadic MND could benefit from the therapy.

“In fact, preliminary results are so promising that the drug company who produces this therapy is looking to conduct a large international multi-centre trial.”

The Phase 2 study was conducted at Calvary Healthcare Bethlehem Hospital in Melbourne and three sites in Sydney: The Brain and Mind Centre at The University of Sydney, Macquarie University and Westmead Hospital. The study began at Macquarie University in October 2016, having been founded by the Motor Neurone Disease Research Institute of Australia and FightMND.

Principal Investigators Professor Julian Gold and Professor Rowe and his team presented early data analysis from the Lighthouse Project at the International MND Conference in Boston in December 2017.

Globally, The MND research community is highly collaborative. Macquarie University Hospital’s MND Research Group has played a significant role in advancing genetic understanding of the disease and testing the early stages of possible therapies. MQ Health has 70 researchers, and clinical team of 12 based at Macquarie University Hospital. The MQ Health team is internationally recognised team and one of the largest in the world.