New drug could halt MS in its tracks
The preclinical study marks the culmination of six years of research by Dr Steven Petratos, who was aided by his team at Monash’s Department of Neuroscience. If deemed successful, clinical trials will follow.
Diseases such as MS occur when the protective covering provided to the nerve fibres by the supporting brain cells is damaged or missing. MS affects more than 23,000 Australians, causing symptoms that include muscular spasms and problems with coordination, weakness, balance and functioning of the limbs.
The novel drug, a small molecule called DITPA (Diiodothyropropionic acid), could potentially help patients with secondary progressive MS — the stage that follows relapse and remission, when the disease steadily worsens. Specifically, it may offer protection to nerve fibres damaged by the disease in the brain, spinal cord and optic nerves, as well as enhancing their repair.
The drug’s effects, and the mechanism that allows it to work, were found serendipitously by Dr Petratos’s 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. As explained by Dr Petratos, 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.
“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 moderate MS and treat the inflammation associated with it, they do not address the degenerative aspect. He said the new drug is thus “a potential game changer for MS patients in the future”.
DITPA has already been approved by the US 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. Dr Matthew Miles, CEO of MS Research Australia, said, “Repurposing an existing human drug to protect nerve fibres from degradation is different to the standard approach and we’re excited to see the results.”
Dr Petratos’s lab will now test for possible side effects of the drug and any toxic outcomes. He noted that the drug will need to be tested in the long term, as repair to nerve fibres would take years.
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.
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