An experimental drug has just returned some extraordinary results, meaning we could soon have a way to slow down the onset of Huntington’s disease.
Huntington’s disease is an inherited neurodegenerative condition that causes serious cognitive and movement defects in the human brain. Usually starting in middle age, it can quickly see people lose control of their mental capacity and body movement.
In an effort to help combat its effects, new research using an experimental drug has returned some incredible results, according to Prof Sarah Tabrizi, director of University College London’s Huntington’s Disease Centre, who led a recent trial.
According to The Guardian, the phase one trial included 46 men and women with early onset Huntington’s disease, who were given four spinal injections, one each month, with an increasing dose each time.
As they looked at how the patients responded to the drug, the researchers found a dramatic drop in the amount of harmful protein in the spinal cord. The improvements increased with each larger dose, indicating its benefits in treating the disease.
“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,” Tabrizi said.
“This is probably the most significant moment in the history of Huntington’s since the gene [was isolated].”
Major trial needed
The findings have piqued the interest of other neuroscientists and medical professions as the drug is created from a synthetic strand of DNA, so it just might be adaptable to treat similar brain disorders such as Alzheimer’s and Parkinson’s.
The next major step for Tabrizi and her research team is to conduct another trial, grander in scale and for a longer period of time, as the team was not able to determine whether the patients’ clinical symptoms had improved.
If it is proven to be an effective treatment, someone with the affected gene, or who has yet to develop symptoms of Huntington’s, would receive the spinal injections approximately every three to four months, Tabrizi added.
Despite more needing to be done to test its effectiveness, the pharmaceutical industry has quickly taken an interest, with Swiss company Roche putting down a $45m fee to launch the larger trial.
Earlier this year, a team including researchers from NUI Galway found early promise of a treatment for the disease by targeting an enzyme called histone deacetylase 3. The enzyme is believed to alter important biochemical mechanisms in the brain of Huntington’s patients, accelerating the disease’s progression.