Researchers from Monash University in Australia are set to conduct a large study into a drug that may one day help halt the progression of multiple sclerosis (MS).
The preclinical study, led by Dr Steven Petratos from the Department of Neuroscience, was given the go-ahead after MS Research Australia announced a $70,000 grant to support it. If successful, clinical trials would follow.
The novel drug could potentially help patients with secondary progressive MS (SPMS), the stage that can follow relapsing remitting MS (RRMS). MS affects more than 23,000 people in Australia, causing symptoms that include muscular spasms and problems with coordination, weakness, balance and functioning of the limbs. In the UK it is estimated that around 100,000 people have MS. NHS guidelines say that around half of those diagnosed with RRMS will develop SPMS within 15-20 years of diagnosis.
The drug, a small molecule called DITPA (Diiodothyropropionic acid), may offer protection to nerve fibres damaged by the disease in the brain, spinal cord and optic nerves, as well as enhancing their repair.
Its effects, and the mechanism that allows it to work, were found serendipitously by Dr Petratos’ team during research into a group of molecules affecting the development of human brain cells called oligodendrocytes, which play an important role in interacting with, supporting and protecting nerve fibres.
MS occurs when the protective covering provided to the nerve fibres by the supporting brain cells is damaged or missing.
DITPA was approved by the US Food and Drug Administration (FDA) for use in clinical trials to treat a rare disorder called Allan-Herndon-Dudley syndrome (AHDS), which severely affects movement, and has been used in larger trials for cardiac problems.
The molecule has the advantage of being able to cross the blood-brain barrier to target affected cells in the brain, a limitation of existing treatment.
Dr Petratos said: “The drug that we’ve identified may have a significant benefit in changing the course of MS progression primarily from the aspect of protection of the central nervous system as well as enhancing repair,” he said.
Dr Petratos said that while a range of existing drugs moderated the disease and treated the inflammation associated with it, they did not address the degenerative aspect.
“This is a potential game-changer for MS patients in the future,” he said.
Dr Petratos’ laboratory will now test for possible side effects of the drug and any toxic outcomes. The drug would need to be tested in the long-term, as repair to nerve fibres would take years, he said. The molecule has been patented and the researchers are negotiating with a potential commercial partner to further develop and test the drug in clinical trials.