Experimental Drug Reduced Toxic Protein that causes Huntington's Disease in Humans.

Huntington's drug trial shows promising results

Huntington's disease is an inherited disorder in which mutant forms of the huntingtin protein damage nerve cells in the brain. The new drug, Ionis-HTTRx, was shown to be safe in humans and lowered levels of mutant huntingtin protein in the nervous systems of trial participants.

 From article, (For the first time, an experimental drug has reduced levels of the toxic protein that causes Huntington's disease in humans.

Huntington's disease is an inherited disorder in which mutant forms of the huntingtin protein damage nerve cells in the brain.
The new drug, Ionis-HTTRx, was shown to be safe in humans and lowered levels of mutant huntingtin protein in the nervous systems of trial participants.

The trial involved 46 patients at early stages of the disease who were enrolled across nine centers in the UK, Germany and Canada. Initial results were announced Monday.
Each patient received four doses of either the new drug or a placebo, injected directly into spinal fluid so it could easily reach the brain. For those receiving the drug, the dose was increased over time, and safety was monitored throughout, according to a statement.
Concentrations of the toxic protein were measured in the patients' spinal fluid before and after the injections.
Among people receiving Ionis-HTTRx, the team saw a significant decline in levels of mutant huntingtin, which causes symptoms of the disease, with higher doses having greater impact. This is the first time the protein has been lowered in patients with the condition.

Huntington's disease is an inherited neurodegenerative disorder in which nerve cells in the brain are damaged, causing uncontrolled movements, changes in behavior and emotions, and altered cognition.

It's hoped this form of drug could be adapted to target proteins in other currently incurable brain disorders, such as Alzheimer's disease.

By changing the DNA sequence in the drug, researchers can target any mRNA, Wild said. In other neurodegenerative diseases, "there is at least one protein you would like to partially reduce," he said. One example is tau protein in Alzheimer's disease. "This is proof of principle you can inject these drugs into spinal fluid; they get into the brain and reduce harmful proteins.")

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