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Doctors Corner
By Martha NanceDoctor's corner
As another year comes to a close, and a new century and new millennium begin, it is
time to pause and reflect on what we have accomplished so far for the HD families of
Minnesota and what remains to be done. What remains to be done is simple: find the
real answers to HD, either a treatment that stops the disease before it starts, or
treatments to suppress or slow down the cell damage that takes place in the brain.
What we have accomplished in Minnesota over the last two decades, we hope, is to
create a clinic that is second to none in providing care for individuals with HD and their
families. Our HD Clinic at Hennepin County Medical Center (HCMC) is considered to be
a model that other centers are attempting to emulate in other locations. We believe
that the needs of HD families are best met by a team of health care providers. In
our center, the team includes two physicians, a nurse practitioner, a nueropsychologist, a
psychologist, a social worker, a genetic counselor, a speech pathologist, a physical
therapist, an occupational therapist, a nutrition specialist, a research nurse, and a
chaplain. These team members work together to meet the needs of not only the person
who has HD, but also the family members, some of whom are caregivers and some of whom are
at-risk for developing HD themselves. As the leader of the team since 1991, I seem
to have a growing reputation around the country as an expert in the care of people with
HD. I helped to edit the new edition of the Physician's Guide to HD, which was
published by HDSA this year, and I am working on a manuscript about juvenile HD (maybe
before next year's convention?). HDSA has recognized me with two awards in the last two
years for my efforts in providing care for HD families, and I have spoken not only at the
US HD convention, but at the Canadian HD convention and will soon speak at the Australian
HD convention about care and about research hopes for the future. Finally, I have
been invited to write a chapter about the management of HD for an HD textbook edited by a
wonderful physician and researcher, Professor Peter Harper of the University of
Wales.
We are working to improve your access to clinical research. Our
clinic is a member of the Huntington Study Group (HSG), a consortium of clinical centers
around the country (and world) interested in trying new therapies for HD, and I have
recently been elected to the Executive Committee of the HSG. We participate in an
ongoing effort by the HSG to collect information about people with HD (all those forms I
fill out when people come to clinic!). Shortly after the first of the year, we will
begin enrolling subjects in a short trial of a drug called Riluzole, which is used to
prolong the course of ALS, or Lou Gehrig's disease. We want to see if it is safe to
use this drug in people with HD. A European group is planning a much longer, 3-year
trial to see if this expensive drug is effective in slowing HD. The US study, which
we are participating in, is called RID-HD. We are also involved in a study I wrote
about in this newsletter earlier this year, called PHAROS, in which people who are at-risk
for developing HD are examined every year or so to see whether very early neurologic
findings can be detected even before the full-blown disease is present. If you think
you might be a candidate for one of these studies, please contact my Research Coordinator,
Dawn Radtke, at 612-347-2943.
Although we do not do any laboratory research at HCMC, it is obviously in the
laboratory that our hopes lie for a cure for HD. Laboratory research has exploded in
many different directions since the discovery of the HD gene less than 7 years ago.
You should be aware that several University of Minnesota researchers are studying
HD, most notably Dr. Walter Low and Dr. Janet Dubinsky (whose current research is funded
in part by HDSA!), and that Dr. Harry Orr, a world leader in research in hereditary ataxia
(a group of disorders similar to HD) heads the HDSA committee which oversees the HDSA
Coalition for the Cure. So, I think that you should consider Minnesota to be a
leader in laboratory research too!
Several scientific meetings take place in the fall-the Society for the
Neurosciences meeting, the American Society of Human Genetics, and the American
Neurological Association. The Huntington Study Group, and a group I chair called the
US HD Genetic Testing Group also have their annual meetings in the fall. As best as
I can tell after all those long plane rides in the middle of the night, there were not any
laboratory breakthroughs this fall that directly translate into treatments for patients.
HOWEVER, the EXCITING thing is that several researchers are putting the HD mice to
use to screen many drugs and potential drugs for their effectiveness in preventing,
delaying the onset, or slowing the course of, HD. Having the mice available to use
for initial therapeutic research studies is critical in speeding up the process of drug
development for HD. This approach seems to be fruitful already, as it is steering
researchers toward some drugs and away from some others they might otherwise have
considered candidates to use in people. The other fascinating new technology that
researchers are beginning to apply to HD is that of DNA microchips. The idea behind
this technology is food for another whole article, but the net result is that it may be
possible to screen thousands of genes quickly and efficiently to see which genes are
"turned on" and which are turned off at different times in the course of HD.
Knowing which genes become more active than they usually are as HD develops, even
before there are symptoms, will allow researchers to work even more quickly to develop
"designer drugs" that block the functions of those genes, sort of like doing
chemotherapy for cancer, where doctors use several drugs that block the different steps
that lead to cell division.
I think the new millennium will be a very exciting one in HD research,
certainly a better millennium than the last. Until the answers come, do
as one of my patients said to me, and "Celebrate life!" Happy 2000 to
all of you!
Doctor Nance
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