The Untreatable Has Become Treatable

“Multiple sclerosis is untreatable, and research into treatments of the disease is the graveyard of many promising careers.”

Or so I was told by a respected neurologist in 1993. I was not to be put off though, and soon afterwards joined a team in Cambridge researching a new treatment for MS.

We were working on an antibody that killed off lymphocytes – one of the white blood cells that fight off illness. We believe these cells are responsible for relapsing multiple sclerosis (MS), the most common form of the disease. People with relapsing MS experience attacks, often lasting weeks, of symptoms such as numbness, weakness, unsteadiness or difficulty with vision. Mostly, young adult women are affected.

Twenty-one years later, the drug we were investigating was licensed, approved by NICE and being given to people in UK and Europe, followed by the United States one year later. Today it’s known as alemtuzumab, and treats many thousands of people around the world. As well as alemtuzumab, a person newly diagnosed with relapsing MS could today be offered up to fourteen different treatments, ranging from injections, to infusions, tablets and even bone marrow transplantation.

We have come a long way, and 2017 has been a particularly exciting year. We had another new treatment emerge for relapsing MS called cladribine – the apogee of convenience. People need to take only a handful of tablets over two weeks in two consecutive years and then – hopefully – will not need any more. Astonishing.

The most exciting news of 2017 is that a drug called ocrelizumab was licensed for a rarer form of MS, called primary progressive. In this type of MS people do not experience attacks that come and go. Instead they feel their mobility, or balance, or vision, very slowly deteriorate, year by year. We are waiting to hear whether NICE judges ocrelizumab to be cost-effective for the NHS. If approved, it will be the first time people with progressive MS have medication available to them.

For my research group, 2017 is a turning point. Like many researchers, we are now investing our energies into ways to repair damage done by the disease. We have known for a few years that stem cells in the brains of people with MS are potentially capable of rebuilding myelin, but for some reason they do not. Luckily a colleague, Robin Franklin, has worked out that we can wake these stem cells up by stimulating a particular receptor in the brain.

The really good news is that we can quickly test his theory because there is an approved skin cancer drug that acts on this receptor: bexarotene. We have started a small trial of bexarotene in fifty people with MS and, if successful, we can repurpose bexarotene as an MS brain-repair treatment.

The MS Society’s Christmas appeal hopes to fund not one but three trials in which licensed drugs are repurposed to promote brain repair or prevent brain damage in MS – this includes our trial of bexarotene. The tantalising prospect is that if just one of these drugs proves to be effective, it could become part of the standard treatment pathway much sooner than if we were starting from scratch. Meaning more effective treatments would be available for MS, faster.

There are 100,000 people living with MS in the UK. I can see a future where all of them can access effective medication, and are offered a package of drug treatments. Some of these might be treatments that target the immune system to prevent more attacks on the brain, and others will encourage resident stem cells to repair the damage that has already been done. This is quite a prospect for a disease that was declared untreatable twenty-four years ago.

Alasdair Coles is the lead investigator of the bexarotene trial, which will be funded by the MS Society’s Christmas appeal. To donate, visit http://ift.tt/2kl8e6U.

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