Supporting MND patients to access innovative treatments

Researchers tested a drug that could help to treat Motor Neurone Disease (MND). Thanks to donors and almost $2 million in MRFF funding, the trial was run at 6 centres.

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New uses for existing drugs

Symptoms and disease courses can vary a lot between different neurological conditions, but scientists, neurologists and researchers often find similarities between the disease mechanisms. When this happens, they often try to repurpose drugs from one disease for other conditions.

Tecfidera is an approved treatment for multiple sclerosis that may also slow down MND by increasing the anti-inflammatory response in the body.

Professor Steve Vucic and Professor Matthew Kiernan led a Phase 2 clinical trial – the TEALS (Tecfidera in Amyotrophic Lateral Sclerosis) study. Both are at the University of Sydney.

‘We used Tecfidera to try and reduce inflammation in the brain and spinal cords of patients diagnosed with ALS,’ Professor Kiernan says.

Amyotrophic lateral sclerosis (ALS) is the most common form of MND in adults. It is also known as Lou Gehrig’s disease in the United States of America.

The researchers were trying to determine if the drug is safe and effective for MND patients.

Testing on a large scale

Dr Bec Sheean is research director at FightMND. The charity raises awareness of the disease, and much needed funds to support research focused on finding treatments. Its vision is to turn the growing body of new knowledge into a cure.

FightMND received $1.96 million from the MRFF in 2017, adding it to funding from other generous donors. The result of this combined investment is the $4.75 million clinical study, with its cutting-edge approach to the disease.

‘We are incredibly grateful to receive this grant and partner with the Federal Government on the TEALS study. A trial like this wouldn’t be possible without investment,’ Dr Sheean points out.

‘This funding has directly improved the access to clinical trials for Australians living with MND.’

The first national clinical trial has reached 109 MND patients, with testing sites in Sydney, Melbourne, Brisbane, Adelaide and Perth.

Professor Vucic and Professor Kiernan recruited the first patients in April 2018. They ran the initial sites at Westmead Hospital, and the Brain and Mind Centre in Sydney.

‘Professor Vucic and Professor Kiernan developed the trial design to test if the drug is effective,’ Dr Sheean says.

According to Dr Sheean, ‘lots of positives’ emerged from the clinical trial activity.

‘This study has established a platform and network of trial sites across Australia. These can be used to continue to improve access to clinical trials for Australians diagnosed with MND,’ she says.

Slowing the silent killer’s progress

MND is a progressive neurological disease. A patient’s average lifespan is 27 months from diagnosis. The cause is unknown.

‘It’s a devastating disease to be diagnosed with’, says Dr Sheean. ‘Clinical trials help to provide hope to patients and their loved ones until an effective treatment is found.’

Our nerve cells control the muscles that allow us to move, speak, swallow, and breathe. With MND, the nerve cells fail to work normally and eventually die. As a result, the muscles weaken and waste.

Professor Vucic explains that Tecfidera can boost or increase the regulatory part of the immune system. While some immune cells can cause harm, others can protect the nerve cells. These may also slow down the disease.

A clearer picture of Tecfidera’s potential will emerge when the researchers analyse their data.

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