MAY 08, 2019 12:49 PM PDT

Drug for Huntington Disease Successfully Lowers Culprit Protein

WRITTEN BY: Nouran Amin

In a study published in the New England Journal of Medicine, a drug for the first time was shown to successfully lower the abnormal protein that causes the debilitating Huntington disease (HD) in patients without any adverse reactions. The culprit protein is mutant huntingtin protein that becomes toxic in the central nervous system of Huntington disease patients.

https://hdsa.org/wp-content/uploads/2019/03/what_is_hd_overview_hd_disease_full.jpg

“HD affects the whole brain, but certain areas are more vulnerable than others. Pictured above are the basal ganglia – a group of nerves cell clusters, called nuclei. These nuclei play a key role in movement and behavior control and are the parts of the brain most prominently affected in early HD.”

Credit: hdsa.org

The study is clinical trial designed to see if the drug halts or slows down progression of Huntington disease. The drug, sponsored by Ionis Pharmaceuticals and Hoffmann-La Roche, was administered monthly to 34 early HD patients via an injection directly into the cerebrospinal fluid (CSF)—which is the location of the protein.

"This is a tremendously exciting and promising result for patients and families affected by this devastating genetic brain disorder," said Dr. Blair Leavitt, neurologist and director of research at the Centre for Huntington Disease at UBC. "For the first time, we have evidence that a treatment can not only decrease levels of the toxic disease-causing protein in patients, but that it is also safe and very well tolerated."

Learn more about Huntington Disease (HD):

Huntington disease (HD) develops adulthood and progresses to causes abnormal involuntary movements, psychiatric symptoms and dementia. It is a fatal a genetic neurological disease caused by a single known mutation. There is currently no effective treatments that have been proven to slow the progression of the disease.

Source: Science Daily

About the Author
  • Nouran earned her BS and MS in Biology at IUPUI and currently shares her love of science by teaching. She enjoys writing on various topics as well including science & medicine, global health, and conservation biology. She hopes through her writing she can make science more engaging and communicable to the general public.
You May Also Like
NOV 25, 2019
Drug Discovery & Development
NOV 25, 2019
Discovery of mechanism behind Alexander disease may lead to enhanced drug development
Researchers have long known that the cause behind Alexander disease is a genetic culprit—mainly a mutation leading to the production of a defective p...
DEC 04, 2019
Cannabis Sciences
DEC 04, 2019
Is Cannabis Helping America Sleep?
Researchers find cannabis is being used as a sleep-aid in Colorado. Many Americans struggle with sleep disturbances -- some estimates put the percentage at...
JAN 06, 2020
Drug Discovery & Development
JAN 06, 2020
Designing Drugs To Fight off C. Diff Infections
A study published by PNAS explains breakthrough research around designing drugs that target C. diff bacterial infections that result in 15,000 deaths in th...
JAN 21, 2020
Cell & Molecular Biology
JAN 21, 2020
Repurposing Existing Drugs to Treat Cancer
Drugs have to be rigorously tested before they can be offered to patients, so it can be much easier to find more than one application for a medication....
FEB 10, 2020
Cancer
FEB 10, 2020
New hope for KRAS-mutant pancreatic cancers
A study published recently in the journal Cancer Research showcases the findings from new research on pancreatic cancer. The study was led by Frank Mc...
FEB 18, 2020
Microbiology
FEB 18, 2020
Newly Found Glycopeptide Antibiotics Kill Bacteria in a New Way
The overuse and misuse of antibiotics and the adaptability of microbes has created a problem that people must solve....
Loading Comments...