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Huntington's drug can slow the progression of the disease
June 21 2018, 06:42 | Rex Rios
Huntington’s gradually causes problems in the brain
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. Therefore, the main aim of the UCLstudy was determining whether the experimental drug - given to the majority of the 46 men and women from the UK, Germany and Canada with early stages Huntington's who took part - is safe.
Researchers working in other fields of degenerative disease are giddy with excitement about what the study means for future therapies with lab-made genetic material.
Huntington's disease normally appears in adulthood and causes involuntary movements, psychiatric symptoms and dementia. But the drug manufacturer Roche is seeking to bring the drug to market and has paid $45 million for development rights.
"The key now is to move quickly to a larger trial to test whether the drug slows disease progression", Tabrizi said.
The onset of the disease usually begins between the ages of 30 and 50 and continually worsens over a period of 10 to 25 years.
The drug known as IONIS-HTTRx is injected directly into the spinal fluid of the patient, this allows the drug to be sent straight to the brain.
Although the trial was too small, and not long enough, to show whether patients' clinical symptoms improved, it showed the drug was safe, well tolerated by patients and crucially reduced the levels of huntingtin in the brain.
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".
Current treatments only help with symptoms, rather than slowing the disease's progression.
The genetic mutation that causes Huntington's is heritable, meaning a child with a carrier parent has a 50/50 chance of developing the disease themselves. Around a quarter were given a placebo.
It is a synthetic single strand of DNA customised in order to latch the Huntington messenger molecule.
Researchers found the drug decreased the concentration of the harmful protein in the spinal fluid in proportion with the strength of the dose, indicating the drug was effective. "This is presumably the most noteworthy crossroads in the historical backdrop of Huntington's since the quality [was isolated]".
The company behind the drug, Ionis Pharmaceuticals, has licensed it to drugs giant Roche for $45m (£33m) and academics speculated that it could herald similar techniques to treat other neurological diseases, like Alzheimer's.
Even though the research is in its infancy, she said she's optimistic. "You can target [any protein]".
"We were pleasantly surprised by the results", Dr. Edward Wild, a senior investigator on the UCL drug trial, told DW.
"Alzheimer's and Huntington's are at opposite ends of the dementia spectrum - so if this holds true for these types, then I believe it is highly likely it will hold true for all the major age-related dementias", Cooper said, in the paper published in the Proceedings of the National Academy of Sciences.