New drugs target delay of Huntington's symptoms
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McMaster researchers have discovered a new drug target that may be effective at
preventing the onset of Huntington's disease, working much the same way heart
medications slow the progression of heart disease and reduce heart attacks.
Their landmark research discovered a family of kinase inhibitor drugs - that all target
one enzyme called IKK beta kinase - as effective for Huntington's.
The drug restores a critical chemical change that should occur in the huntingtin protein,
but does not occur in people with Huntington's disease.
"It is the first time anyone has identified drugs that affect how the huntingtin protein
gets modified at one critical site, and through what pathway," said Ray Truant, professor
in the Department of Biochemistry and Biomedical Sciences of the Michael G. DeGroote
School of Medicine at McMaster.
Huntington's disease, which impacts one in 4,000 Canadians, is an inherited disease
that causes certain nerve cells in the brain to waste away. People are born with a
defective gene, but symptoms usually don't appear until middle age. Early symptoms
include depression and cognitive changes, with later symptoms including uncontrolled
movements, clumsiness and balance problems. At some point patients may have
difficulty walking, talking or swallowing. There is no specific treatment for the disease.
Currently kinase inhibitor drugs form a family of successful, new generation drugs that
are coming on the market or have been approved for a wide range of diseases including
stroke, arthritis and cancer.
The McMaster researchers are currently looking at inhibitors that can cross the blood to
brain barrier, before starting preliminary trials. If successful, human clinical trials are
five or more years away.
Truant and Randy Singh Atwal, a PhD graduate, discovered the huntintin protein has an
essential role in chemical stresses relating to human aging and the protein is not
properly modified in response to these stresses during Huntington's Disease.
"This is one explanation as to why it takes until middle age for Huntington's to develop
in most patients, because the role of the huntingtin protein is more critical as a person
ages," said Truant.
The research is supported by the Canadian Institutes of Health Research, the not-for-
profit Cure for Huntington's Disease Initiative Inc., and the Toronto-based Krembil
Family Foundation. Truant is chair of the Huntington Society of Canada's scientific
"These new results are extremely important because they may help to delay the
progression of Huntington's disease," said Dr. Anthony Phillips, scientific director of the
Canadian Institutes of Health Research (CIHR) Institute of Neurosciences, Mental Health
and Addiction. "CIHR is proud to support researchers who devote their time to look into
this genetic brain disorder that has such challenging effects on individuals and their
families in Canada."
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