UCLH recruits first participant in Europe for a new trial in muscular dystrophy

UCLH has recruited the first participant in Europe to a global clinical trial for adults with Myotonic Dystrophy Type 1 (DM1)- a genetic condition and type of muscular dystrophy that causes progressive muscle weakness and wasting.

The GALILEO clinical trial aims to learn more about the safety and tolerability of an investigational drug for DM1. This clinical trial is the first time that this investigational study drug has been given to humans.

DM1 is the most common, adult-onset form of muscular dystrophy that affects muscles and many other organs in the body. It is caused by a mutation to a gene called DMPK. DM1 is a multisystemic condition characterised by progressive muscle weakness and wasting and inability to relax contracted muscles, known as myotonia. DM1 can also affect other bodily functions including the heart, lungs, and gastrointestinal system. Currently there is no cure for DM1.

It is hoped the study drug may be able to reverse or halt disease progression, and allow patients to regain independence, improve quality of life and daily functioning, and prolong life expectancy.

Dr Chris Turner, consultant neurologist at UCLH and UCL honorary clinical associate professor, is leading the study at UCLH. The study is being conducted at the NIHR UCLH Clinical Research Facility site located within the National Hospital for Neurology and Neurosurgery. It is supported by the NIHR UCLH Biomedical Research Centre.

Dr. Chris Turner said: “We are very excited to be able to conduct a clinical trial of an advanced investigational therapeutic for people with myotonic dystrophy type 1 (DM1). Such investigational therapeutics aim to provide people living with DM1 the possible option of being able to impact the course of their disease as opposed to symptomatic treatments only. This represents the efforts of years of high quality scientific and clinical work to understand how we can treat the underlying genetic cause of DM1 which affects 10,000 people in the UK.

"I would like to thank all the team at the NIHR UCLH Clinical Research Facility at Queen Square, the UCLH/UCL Joint Research Office and the Muscular Dystrophy clinical team at the National Hospital for Neurology and Neurosurgery for demonstrating great teamwork and tenacity in conducting this clinical trial."

The participant said: “I was very grateful to be given the opportunity to participate in the clinical trial even though I don’t know if I am going to directly benefit from it.”

Approximately 36 subjects will be recruited to the study from around the world. UCLH is one of 11 sites currently supporting the study. For more information, please visit here.

If you are interested in participating in this study at UCLH, please contact Dr Nikoletta Nikolenko at n.nikolenko@nhs.net.