This is an important step towards faster, more effective clinical trials and better care for people living with Charcot-Marie-Tooth disease (CMT).
Muscular Dystrophy UK, the leading charity for more than 110,000 children and adults in the UK living with one of over 60 muscle wasting and weakening conditions has funded the new project, TRANSFORM-CMT, led by Professor Mary Reilly MBE, at UCL Queen Square Institute of Neurology.
CMT is one of the most common inherited neurological disorders, affecting approximately 1 in 2,500 people in the UK and worldwide. It affects the peripheral nerves which control muscle movement and relay sensory information, such as touch, back to the brain. It causes progressive muscle weakness and loss of sensation, typically starting in the feet and legs, and later possibly affecting the hands and arms. People can experience difficulty walking, foot deformities and problems with balance and coordination. There is no cure for the rare condition.
Professor Mary Reilly will develop a test using MRI scans to better track and measure small changes in CMT which is crucial to be able to do clinical trials. The project will also collect data from people living with CMT across the UK to build a clearer picture of the genetic types of CMT in the UK, the progression and the impact on patients daily lives. The team will collaborate with centres across the UK to help accurately monitor this.
Having this two-pronged approach will help researchers design and run clinical trials, which can determine if a treatment is working or not with more certainty. Together, transforming how clinical trials are run and speed up the search for new treatments.
Professor Reilly is Professor of Neurology and Head of the Division of Clinical Neurology in UCL Queen Square Institute of Neurology. She’s a clinician scientist in academic clinical neurology and is internationally recognised as a pioneer in inherited peripheral neuropathies with an interest in CMT.
Professor Reilly said: “Charcot-Marie-Tooth disease is by and large a slowly progressive condition, but it is undoubtedly progressive. Many of our measures, such as examining patients or testing muscles, do not measure change quickly enough. They will pick up change over three to five years, but a clinical trial is only one or two years. If we’ve got lots of potential treatments but can’t measure change, then we’ll never know if the treatments work.”
“We’re at a pivotal turning point. We’re already doing two clinical trials and there are multiple drugs in development about to come into human trials. What excites me is that thanks to funding from Muscular Dystrophy UK, this research will give us a way of doing clinical trials in multiple centres in the UK speeding up the development of new treatments.”
Anne Furby, CMT patient, discussing the new project with Professor Reilly at University College London. Credit: Muscular Dystrophy UK
Anne Furby, from Shoreditch, East London, was diagnosed with CMT aged five years old, it’s a condition that also affects other members of her family. The progression of her condition has really increased.
Anne said: “The tests run by my doctor don’t really seem to pick up any decline between appointments. However, I definitely notice a decline in between my appointments.”
“Hearing about this research funded by Muscular Dystrophy UK has given me hope for future generations of people with CMT - that there might be a way to slow down their progression, make living with the condition more comfortable, or maybe even one day stop it completely.”
Dr Kate Adcock, Director of Research and Innovation at Muscular Dystrophy UK said: “Clinical trials are key to knowing if a treatment is working or not. But for conditions that progress slowly, such as CMT, it can be difficult to see the effect a treatment might be having. The project will enable Mary and her team to map people living with the condition and potentially transform clinical trials for Charcot-Marie-Tooth disease.
“At Muscular Dystrophy UK we’re proud to be funding the project at University College London. It’s a key step towards making CMT clinical trials faster, more effective, and more inclusive. By understanding the condition better, and making clinical trials more accurate and accessible, it brings us closer to the day when effective treatments are available to people with CMT.”
Links
- More information or to register your interest to see if you’re eligible to take part.
- Muscular Dystrophy UK
- Professor Mary Reilly’s UCL Profile
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Main image:
Professor Mary Reilly, credit: UCLH
