Repurposing an existing drug to repair damage caused by MS

Repurposing an existing drug to repair damage caused by MS

20 February, 2020
  • Repair of myelin, or remyelination, is believed to be an important step in helping prevent and reverse MS symptoms, and is also believed to be important in treating progressive forms of MS for which there are very limited treatment options.
  • MS Research Australia funded researcher, Dr Steven Petratos, is working to repurpose a treatment used in clinical trials for another neurological condition (Allen-Herndon-Dudley Syndrome), for people with MS.
  • The treatment, called DITPA, has shown promising results in repairing myelin in laboratory models of MS, and could be beneficial for people with progressive forms of MS.

MS results from the loss of myelin, the insulating sheath around nerve fibres, in the brain and spinal cord. The body has limited abilities to repair myelin (a process called remyelination), and myelin repair is often incomplete in people with MS. Currently, all the available therapies target the immune system, and there are no treatment options capable of repairing the damage. Repairing myelin is thought to be very important as it may reverse some symptoms of MS and could provide protection against further damage to the nerves.

MS Research Australia funded researcher, Dr Steven Petratos from Monash University VIC, and his team are currently working to repurpose an existing drug to encourage the body’s repair mechanisms to remyelinate the nerves in the brain and spinal cord. The drug, called DITPA, mimics the action of a thyroid hormone and has been used in clinical trials to treat a rare disorder called Allan-Herndon-Dudley syndrome (AHDS), which severely affects movement.

Dr Petratos’ work has shown that this drug has the ability to encourage precursor cells within the brain to become myelin producing cells, and these then might enhance the ability of the body to repair areas of damaged myelin in the brain and spinal cord. He has shown that this has promising effects on laboratory models of MS and it may reverse some symptoms of MS.

Additionally, DITPA has the advantage of being able to cross the blood-brain barrier to directly target affected areas in the brain. This is one of the biggest barriers for any potential treatment of MS. If successful, this study may have important implications for people living with MS due to its potential to reverse some of the damage caused by the disease, as well as provide protection from further damage. It may be beneficial for people with both relapsing and progressive forms of MS. This is exciting because while MS affects over 25,600 people in Australia, treatment options are mostly limited to people with relapsing remitting MS (RRMS) and unfortunately there are currently very few options for those with progressive forms of the disease.

DITPA is currently in the pre-clinical stages of research, with Dr Petratos and his team hoping to move this potential treatment option into clinical trials in a few years. He has recently been awarded a patent on the drug and is also in discussions with a major pharmaceutical company to develop the drug for trials in people with MS. While this is still in early stages of research, if successful it may have a big impact on MS.

Dr Petratos was recently awarded a MS Research Australia and Trish MS Research Foundation Project Grant to support this work.

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