As first reported last week, researchers at Leonard Wolfson Experimental Neurology Centre in London have tested an experimental drug that safely reduced the toxic proteins in the brain that cause Huntington’s.
Though more research needs to be done, this finding has doctors feeling optimistic that the progression of the disease could be slowed or even stopped in some patients.
“It’s fascinating,” said Alana Cozier, a neurohospitalist with Wellmont Medical Associates. “It has the potential to be life-altering for people who have Huntington’s and people at risk, also.”
What is Huntington’s disease?
Cozier describes Huntington’s as a “progressive, inherited neurodegenerative disorder.” In simpler terms, this means the disease is passed down from parent to child, and it causes nerve cells in the brain to degenerate and die over time. Other neurodegenerative disorders include Alzheimer’s and Parkinson’s.
In a person with Huntington’s, the degeneration of nerve cells causes a number of symptoms, including difficulty thinking, behavioral problems, a decline in motor skills, mood changes and chorea, or abnormal involuntary movements.
Cozier said there are five to 10 cases of Huntington’s per 100,000 people worldwide. About 30,000 people in the U.S. are known to have the disease and about 150,000 are at risk, meaning one of their parents has the disorder.
How is the disease currently being treated?
Because no cure exists for the disease itself, doctors are able only to treat the symptoms, Cozier said. The most commonly prescribed drug for Huntington’s patients is tetrabenazine, which treats the involuntary movements.
Cozier added that counseling is often recommended for people with the disease due to the poor prognosis.
“The prognosis is invariably grim,” Cozier said, “which is why diagnosis early on is very important to help patients get counseling, symptomatic treatment and also genetic counseling.”
What is the new drug?
The experimental drug was tested on 40 people via a lumbar puncture, or spinal tap. After injecting the drug into each person’s spinal fluid, researchers then took samples of the fluid and measured the amount of Huntington’s disease-causing proteins that were present.
Researchers found that after administering the drug, the amount of toxic proteins in a patient’s spinal fluid had decreased. Patients who received the highest dose of the drug experienced the greatest decrease in toxic proteins, Cozier said.
What are the next steps?
Cozier said the research “has the potential to be quite groundbreaking” because no drug has ever been developed to target a neurodegenerative disease.
She added that further clinical development is needed to determine whether the drug can actually cure the disease or lessen its severity.
“That’s essentially what they are hoping to achieve, but the clinical trial is still in its infancy,” Cozier said. “They’ve found that it’s safe to be administered. The next step is to determine whether lowering the (proteins) in this manner actually has any benefit.”