Huntington's Disease Drug Trial Shows Halt in Progression

Calvin Saunders
December 13, 2017

'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.

Peter, Sandy and Frank - as well as their partners Annie, Dermot and Hayley - have always promised their children they will not need to worry about Huntington's as there will be a treatment in time for them.

"The case for these is not as clear-cut as for Huntington's disease, they are more complex and less well understood", she told the BBC.

Prof Sarah Tabrizi, the lead researcher and director of the Huntington's Disease Centre at UCL, told the BBC: "I've been seeing patients in clinic for almost 20 years, I've seen many of my patients over that time die".

"It worries me that I am going to change, and people around me are going to be put through what I went through with my father". "I don't want to overstate this too much, but if it works for one, why can't it work for a lot of them?"

Huntington's is an incurable degenerative disease caused by a single gene defect that is passed down through families. This commonly happens between the ages of 30 and 50.

As hoped, IONIS-HTTRx produced significant, dose-dependent lowering of the level of mutant huntingtin - the first time the protein known to cause Huntington's has been lowered in the nervous system of patients.

"Huntington's distant from everyone else is sufficiently energizing", said Hardy, who initially recommended that amyloid proteins assume a focal part in Alzheimer's. "I am very, very excited", he said.

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"The results of this trial are of groundbreaking importance for Huntington's disease patients and families", the chief investigator of the research, Professor Sarah Tabrizi, said.

Each patient received four doses of either IONIS-HTTRx or placebo, given by injection into the spinal fluid to enable it to reach the brain. The drug lowered levels of a toxic, mutated form of a protein that is at the root of this neurodegenerative illness, suggesting a path forward for treatment.

A "GROUND-breaking" drug trial has offered new hope to people with Huntington's disease.

"We wish we could get these therapies to patients faster, but we have to be careful and ensure they are safe", said Dr. Blair Leavitt, a neurologist with the University of British Columbia.

The Phase 1/2a evaluated the first therapy in clinical development meant to target the underlying cause of Huntington's Disease (HD).

Results from the study will be presented at medical conferences in the first half of 2018, and will be submitted for publication in a future issue of a peer-reviewed medical journal. The success in the early-stage clinical trial has prompted Roche to exercise its option to license the product, called IONIS-HTT (Rx). Roche will assume responsibility for further development of the drug.

"Though more work needs to be done, if gene silencing lives up to this promise we could be on the brink of some of the personalized treatments that patients with severe genetic diseases need so badly".

According to a report by BBC, inherited neurodegenerative Huntington's disease progression could now be slowed or even halted by an experimental drug which has proved successful in early patient trials.

Other reports by MaliBehiribAe

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