Exploring new potential
for old drugs

Given the enormous resources and long timeframes involved in getting a new drug to market, drug repurposing is an appealing alternative. UQ researchers are investigating existing drugs to find out if they have benefits beyond the disease for which they were originally created.

Dr Richard Gordon is testing four drugs with the aim of slowing progression of Parkinson’s disease. One of the drugs being tested has been commonly used since the 1980s to treat high blood pressure. It now shows promise in benefiting patients with the degenerative neurological disease.

If testing in preclinical models is successful, a phase II clinical trial could begin as early as next year. As the project only started this year, this represents rapid progress from the laboratory to patients – at a fraction of the cost associated with developing a new drug.

And 200 years after James Parkinson first discovered the disease, it is fitting that, in 2017, steps are being taken towards better treatment for people with Parkinson’s disease.

As Dr Gordon explains, once the safety and effectiveness of a repurposed drug are confirmed, there are two possible outcomes.

“In some cases, the drug can be prescribed by neurologists as an off-label therapeutic for the management of Parkinson’s disease,” he says.

Dr Gordon says work done by researchers in the United Kingdom and United States this year has, for the first time, found a potential disease-modifying therapy for Parkinson's disease.

"This repurposed diabetes drug, although it has not been proven yet, provides the most compelling evidence so far for any drug that can modify Parkinson's disease," Dr Gordon says of the research, published in The Lancet in August.

An important factor enabling projects, like those being carried out by Dr Gordon, to progress rapidly towards clinical translation is the fact that many repurposed drugs have already passed rigorous clinical testing in humans.

“The repurposed anti-hypertensive drug we are evaluating is considered to be safe and can be taken for an extended period without major adverse side effects,” explains Dr Gordon.

“That makes it very attractive as a drug for people with Parkinson’s disease who could potentially take the drug for a long period of time.”

Dr Gordon believes the benefits of drug repurposing extend far beyond Parkinson’s. Researchers are searching for treatments for conditions including motor neurone disease, Alzheimer’s, cancer and arthritis.

The UQ researcher says there is also immense potential in repositioning ‘rescued drugs’ – those that have already been tested to be safe in humans, but were not effective enough for the disease for which they were originally developed.

Dr Gordon’s research is being funded by the Michael J Fox Foundation and Shake It Up Australia as well as the Cure Parkinson’s Trust UK and the Queensland Government’s Advance Queensland program.

This story is featured in the Summer 2017 edition of UQMedicine Magazine. View the latest edition here. Or to listen, watch, or read more stories from UQ’s Faculty of Medicine visit our content hub, MayneStream.