Foundation Harbors Hope for Many
RedNova News
2005/05/07
Over the last decade, the Ara Parseghian Medical
Research Foundation has greatly advanced research into
the rare disease that not only threatens hundreds of
children but likely holds clues for treating
Alzheimer’s disease, heart disease and stroke.
“The whole field of NP-C (Niemann-Pick Type C)
research has been transformed in the last decade or
so, and obviously the Parseghian foundation has played
a big role in that,” said Dr. Marc Patterson, director
of pediatric neurology at New York’s Columbia
University and a member of the foundation’s scientific
advisory board.
Research got a big boost in July 1997 when National
Institutes of Health researchers announced they had
isolated the NP-C gene, but there is still no proven
treatment for the disease.
But one clinical trial is showing promise, said
Patterson, who is leading the study in the United
States. It involves a drug that slows the progress of
a similar disorder called Gaucher disease. The
Parseghian foundation funded initial testing of the
drug in mice afflicted with NP-C. The clinical trial
began three years ago, but Marcia’s disease was too
advanced for her to qualify for the study.
Patterson described “a range of possibilities” that
scientists hope to pursue. They include using simple
anti-inflammatory drugs such as aspirin, because
inflammation is known to be one of the factors
involved in NP-C. Also being developed are drugs to
prevent “apoptosis” – the natural death of cells – to
salvage cells that have been partially damaged by
NP-C, thereby slowing the neurodegenerative process.
“I think there’s a good chance that we will be able to
identify a drug that ameliorates the course of the
illness,” Patterson said. “This is not likely to yield
a cure for the disease, but we may slow it down or
arrest it.”
A “cocktail” of two or more drugs is more likely to
succeed than a single agent, Patterson said. And
because a treatment might also benefit patients with
Alzheimer’s or another cholesterol disorder, “the
pharmaceutical firms will be prepared to make bigger
investments, because you’re talking about a
potentially bigger market.”
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From: Emil Lazarian | Ararat NewsPress