Monash and The Alfred to run world-first clinical trial that may lead to cure for epilepsy

A pill developed for cancer treatment is offering new hope for epileptics, with a world-first trial to be run by Monash University and The Alfred.

A daily pill discovered in Melbourne more than a decade ago is being hailed as a potential cure for epilepsy.

The drug known by its chemical name sodium selenate was originally developed by researchers at the University of Melbourne as a treatment for cancer.

Now, in a world first, 124 Australians will be recruited for a trial to be run by Monash University and The Alfred to see if it can achieve the same results in treating epilepsy in humans as it has in animal model studies.

Epilepsy affects more than 150,000 Australians and a third struggle to control their condition with available anti-seizure medications.

Danielle Heaven, with her children Callum, 11, and Luci, 8, is in a world-first trial to find a cure for epilepsy. Picture: David Crosling

Monash Neuroscience’s Terry O’Brien, the deputy director of research at Alfred Health and program director of Alfred Brain, describes epilepsy as one of the most common serious disabling brain disorders. He says it is costly to both individuals and their families and to the national health budget, with an estimated annual cost of $12.3bn.

“Despite the cost of the disease, and the enormous amount of research into it, there has not been a single therapy developed to prevent the development of epilepsy, nor to mitigate its severity once established. Until now,” Professor O’Brien said.

Lucy Vivash from Monash Neuroscience will lead the trial and says there is no reason to believe the potentially curative treatment of sodium selenate will not be as effective in human trials.

“Right now the data we have is showing that a proportion of the animals in the preclinical study stopped having seizures altogether, with the remainder having fewer, less severe seizures,” she says.

The drug works by altering the accumulation of a protein in the brain called tau, which has been shown to promote brain-cell damage in neurological degenerative brain disorders such as Alzheimer’s disease and dementia.

“We suspect reducing the amount of the toxic forms of tau can alleviate symptoms of disease and prevent ongoing symptoms. It may be curative,” Dr Vivash says.

The study will recruit people from nine cities including Melbourne with drug-resistant epilepsy who will receive treatment for six months.

Pablo Casillas-Espinosa, who led the preclinical studies, says current therapies target symptoms only.

“So when a patient stops or misses the medication they are as likely to have a seizure as if they had been untreated, and importantly one-third of patients don’t respond to any treatments so far,” Dr Casillas-Espinosa said. “We hope to positively impact the quality of life of people with epilepsy.”

Dr Vivash, who will work with Dr Casillas-Espinosa and Professor O’Brien to run the trial, says the treatment is looking highly promising for all types of epilepsy.

Last week Dr Casillas-Espinosa and his team published the preclinical results in the journal eLife, where they reported that in the animal model sodium selenate showed promise as the first medical disease-modifying therapy for epilepsy.

The researchers have now been awarded a $3m Medical Research Future Fund grant to start the trial in October.

‘This is revolutionary’

A pill a day that could stop her debilitating seizures would be life changing for Melbourne mum Danielle Heaven.

Her odyssey with a rare form of epilepsy has been “epic”, she says. Her seizures started as a baby and continue to affect her life today.

Danielle Heaven (centre) has suffered seizures since she was a baby. Picture: David Crosling

Danielle, 46, has drug-resistant epilepsy and now takes three types of anti-seizure medication to try to control it. They don’t, and the drugs often leave her fatigued and cause memory loss and cognition problems.

It also means she is at increased risk of sudden unexpected death in epilepsy, which is why she underwent neurosurgery last year to have a vagus nerve stimulator implanted.

This is the nerve that sends messages to the brain to calm down.

There is little she hasn’t researched, tried or considered to stop her seizures that only happen in the still of the night, are often invisible and can happen in clusters without warning.

“Even if symptoms are not severe, epilepsy is always there; it affects life decisions, even having a pregnancy as there is that risk you might pass epilepsy on to your children,” she said.

Danielle says there is also fear and stigma around epilepsy.

“Every year or two I have to increase the medication, but the seizures still happen,” she said.

“I didn’t know until a year or two ago when I was participating in some research that I was medically resistant.

“This means once you try two or three medications without success, there is very little chance your seizures will be controlled. So you lose a little bit of hope that you will be seizure free.

“Until now there has been no hope for a cure, nothing in sight. So this is revolutionary.”