Drug fuels hope for treating cause of Parkinson’s disease

A drug developed by Melbourne scientists is fuelling hope that the cause of Parkinson’s disease may soon be treated, instead of just the symptoms of the ­crippling degenerative condition.

Doctors have been able to stop and wind back the symptoms of Parkinson’s disease using a new drug developed by Melbourne scientists.

The find fuels hope of treating the cause of the ­crippling degenerative condition, rather than just its symptoms.

Results of a Royal Melbourne Hospital-led trial among 19 patients — announced at the World Congress on Parkinson’s Disease in Canada overnight — show the drug, CuATSM, halted the progression of Parkinson’s disease and significantly reduced the severity of symptoms.

Developed over the past 15 years by scientists at the Florey Institute of Neuroscience and the University of Melbourne, the copper-­delivery capsule could be a significant advance in the treatment of Parkinson’s disease if the result could be replicated in bigger studies, the RMH’s Andrew Evans said.

Royal Melbourne Hospital‘s Dr Andrew Evans. Picture: David Caird

“It would suggest it may well be taking the neurons that are destined to die and turning around the process, giving some symptom benefit as well as slowing the progress of the disease,” Dr Evans said.

“The impact has been that (patients) feel better, but also it gives them hope for the future that they are not going to decline in the same way that most patients will do.”

The same drug has also delayed and reversed the progression of motor neurone disease.

A trial of 32 patients partly funded by the FightMND Foundation found CuATSM slowed the progression of MND by 70 per cent and improved lung function, cognition and motor ability.

In the latest trial, early-stage Parkinson’s patients from Melbourne and Sydney took a pill twice a day for six months, with the biggest benefits occurring in those who took the highest dosage.

While existing treatments can reduce the symptoms of the neurological diseases, finding a drug to target the cause of the conditions remains the holy grail.

But after finding a group of copper compounds that can boost brain cell numbers and improve motor skills of animals with MND and Parkinson’s disease, researchers believe the drug blocks a type of cell death behind the brain diseases.

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Although both have been early Phase 1 trials without a control group, Associate Professor Kevin Barnham, of the Florey Institute, said the mirrored results gave confidence that the drug was acting on the underlying cause of the patients’ illness.

“Having it working in two different indications like this gives us great confidence we are on to something very important here,” Assoc Prof Barnham said.

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