The previously incurable Huntington’s disease, which gradually impairs a person’s ability to function due to a degeneration of nerve cells, might be in the reach of a treatment, a UK study found.
Huntington’s disease is the most common inherited neurodegenerative brain disorder
in the United States with more than 30,000 sufferers.
The study conducted by the University of Leicester UK, managed to slow the
development of neurodegeneration in fruit flies by targeting a particular enzyme.
Researchers found a causal link between decreased activity of an enzyme, called KMO,
and the reduced number of neurons dying in the fruit flies.
“This breakthrough is important as no drugs currently exist that halt progression, or
delay onset of Huntington’s disease,” said Dr Giorgini, who led the study.
Parallel research in the United States by Dr. Muchowski, PhD, at Gladstone Institute also
found that blocking the same enzyme in mice slowed the onset of Huntington’s disease.
The American researchers have developed a new compound, known as JM6, which
blocks the enzyme and reduces brain inflammation in the mice.
Another study found that targeting the same enzyme with JM6 while treating another
major neurodegenerative condition – Alzheimer’s disease – showed results of
preventing memory loss.
“The discovery has significant implications for two devastating diseases and suggest
that the KMO enzyme is a good protein for us to target with medications in diverse
neurodegenerative disorders,” said Professor Lennart Mucke, of the Gladstone Institute,
where the research took place.
With some luck, phase 1 testing could begin on human patients within the next two
years. The rapid development of testing can be attributed to the fact that JM6 does not
penetrate into the brain, but works by inhibiting KMO in the blood. The blood cells
then send a protective signal to the brain, to stabilize brain-cell function and prevent
“In a world where there is such vacuum of hope about Huntington’s disease, I am
thrilled that someone of Dr. Muchowski’s caliber has suggested the possibility of
imminent clinical trials,” said Charles Sabine, a former NBC correspondent