30-Year-Old Antibiotic Offers Hope for Patients With Huntington's Disease
June 30, 2000 -- There is currently no treatment to slow disease progression
in patients with Huntington's disease, the devastating hereditary brain
disorder that killed folk-singer Woody Guthrie and afflicts some 30,000
Victims' mental and physical abilities degenerate, leading to dementia, and
they lose control of their movements and eventually die. But animal studies
from Harvard University's Brigham and Women's Hospital offer hope that
sufferers will soon have a weapon to help them fight the disease -- one that
has been around for the last 30 years.
Mice bred as models for Huntington's lived 14% longer when treated with the
common antibiotic minocycline, the July issue of the journal Nature
Medicine reports. Harvard researchers say they will soon begin human trials
of the antibiotic in patients with Huntington's disease.
"If the human safety trials go well, we should be able to recommend this
for Huntington's patients within the year," study author Robert M.
Friedlander, MD, tells WebMD. "Our studies showed that there were more
minocycline side effects in the Huntington's mice compared to normal mice. This
could pose a problem, but I don't think it will translate to humans."
If the antibiotic appears safe and effective in patients with Huntington's,
Friedlander suggests it might be taken daily by those who know they will
develop the disease, beginning perhaps five to 10 years before symptoms appear.
Children who have a parent with Huntington's have a 50% chance of also
developing the disease, and symptoms generally first develop between the ages
of 35 and 45. A genetic test has been available since 1993.
"This is all hypothesis, because we have no human data at this
point," Friedlander says. "What is real is that today there is no drug
treatment for Huntington's. This is a very good candidate for a treatment, not
Friedlander and other experts say the antibiotic could be the first in a
series of advances bringing the management of Huntington's out of the Dark
Ages. They envision a drug cocktail approach similar to that now being used to
treat AIDS patients, in which different agents target different mechanisms of
this complex disease.
"I think the importance of this study is not necessarily that this drug
will be useful in humans, although it may turn out to be, but it shows that
drug compounds can potentially be helpful," says Christopher Ross, MD, PhD,
who was not involved with the study.
"This drug by itself may not have a huge impact, but if you can get two
or three drugs acting in different ways, the cocktail might be quite
effective," says Ross, professor of psychiatry and neuroscience at Johns
Hopkins University and chair of the Medical and Scientific Advisory Committee
of the Huntington's Disease Society of America (HDSA).