Huntington's disease - a breakthrough

In December, scientists at University College London announced a major breakthrough in the treatment of Huntington’s disease. Naturally, this led to excitement across the health community and those living with or who have been effected by Huntington’s. So where are we now?

Over the Christmas break, reports were released which highlighted that early human trials indicated that the new medication can stop the progression of the degenerative condition.

Huntington’s disease is a degenerative neurological condition which causes cognitive and physical issues, which only worsen as time goes by. Sufferers can develop difficulty swallowing foods, feeding, dressing and caring for themselves.

The new drug – which is still in experimental stages – is injected into spinal fluid, and lowers levels of toxic proteins in the brain. Patients receive four doses of the drug, which showed to be tolerated and reduced the levels of huntingtin in their bodies.

Professor Sarah Tabrizi, director of the UCL Huntington’s Disease Centre, said: “The results of this trial are of ground-breaking importance for Huntington’s disease patients and families. For the first time a drug has lowered the level of the toxic disease-causing protein in the nervous system, and the drug was safe and well-tolerated. The key now is to move quickly to a larger trial to test whether the drug slows disease progression.”

Swiss company Roche are to lead a phase three trial which will last up to three years, and aims to establish the impact of the drug on symptoms of HD. If successful, the drug will be licensed by the European Medicines Agency, and give hope to thousands of families worldwide.

Exemplar Health Care currently provide specialist support to those living with HD. Nurse-led teams effectively manage the symptoms of HD to encourage an independent, fulfilling life whilst still possible.

Some Exemplar homes bring together NHS specialist consultant neurologists, psychologists, GPs and Huntington’s Disease Association experts at quarterly in-home clinics. Hosting clinics at Exemplar homes avoids changes in routine and the frustration of long hospital waits. Service users benefit from expert consultant care in a familiar environment.

We focus on restoring people’s choices wherever possible, ensuring those living with Huntington’s disease can make decisions about their care and remain independent for as long as possible.