European first for metabolic medicine team

Congratulations to Adult Metabolic Medicine Consultant Dr Reena Sharma and the metabolic medicine research team at Salford Royal who have recruited the first patient in Europe to a phase I/II study for patients with Niemann-Pick disease type C.

The study is evaluating the safety and efficacy of intravenous Trappsol® Cyclo™ in patients with this rare and fatal genetic disease that affects the brain, lung, liver, spleen, and other organs. Dr Sharma is the Coordinating Investigator for the phase I/II trial in Europe. Clinical stage biotechnology company CTD Holdings, Inc, based in the United States, is developing cyclodextrin-based products for the treatment of disease and is aiming to recruit 12 patients to this study in Europe.

Dr Sharma said; “There is currently no treatment available globally for this condition. Those that are available are limited to delaying the progression of neurological symptoms or providing palliative care to relieve, for example, the gastrointestinal symptoms and seizures”.

We need new treatments that help to address progression of neurological involvement and some of the other problems our patients experience relating to the liver, spleen and lungs.  I hope that this study will lead to a new treatment for this devastating condition.

The first patient visit was supported by the National Institute for Health Research (NIHR) Manchester Clinical Research Facility at CMFT, which provides 24-hour cover for intensive and complex research studies.