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On November 20, 2008, members of the CMTA Board
of Directors met with the principle investigators
working on the three projects of the STAR initiative and
scientists at the NIH Chemical Genomics Center in
Rockville, Maryland, where the high-throughput
screening will take place once the stable cell line
currently being engineered by Ueli Suter and his
colleagues at ETH Zurich is ready, perhaps as early
as July, 2009.
The meeting, held to review progress thus far and to
allow the investigators to coordinate their plans, lasted
several hours. In addition to reviewing how the
Strategy to Accelerate Research had evolved from
recommendations made at the 2006 NIH Peripheral
Neuropathy Conference (chaired by Dr. Steven
Scherer), and meetings with the
Myelin Repair Foundation and NIH scientists, each
investigator presented a report on his work. (Click here to read the details of
these presentations in the scientists' progress report.)
Overall, it was incredibly encouraging to hear of the
progress being made, especially as it relates to
speeding up the search for therapeutic agents for
CMT1A and other forms of CMT. Rather than
completing work on one project before beginning the
next, the scientists are working simultaneously to
develop the cell line for use in screening the
compounds in the NIH library, to create a laboratory
model for further testing of the compounds identified
that reduce the overexpression of PMP22, and to make
sure that the reporter/promoter constructs identified in
these projects are also usable in clinical trials
involving humans.
Dr. Christopher Austin, Senior Advisor to the Director
for Translational Research at the NIH, put this in
perspective when he used a football analogy to
describe the process of developing an investigational
new drug (IND). Instead of starting at the 5-yard line,
he said STAR will get the ball on the 30-yard line at the
completion of the high-throughput screening (HTS).
While that leaves 70 yards to go, the infrastructure the
CMTA has already put in place will help cover the
remaining distance.
Specifically, Dr. Austin said, the CMTA has already
taken appropriate measures to avoid failure. This
includes maintaining control over the process of
developing "lead compounds" by independently
funding both the initial projects of the STAR initiative
and the ascorbic acid clinical trial, which has provided
the framework for evaluating them. Once the viability of
these compounds has been established, funding for
clinical trials may be available from other sources,
including the NIH and the pharmacological industry.
The meeting concluded with an opportunity to tour the
HTS facility, which houses an impressive array of
robotic equipment, and it was impossible not to come
away with a feeling of confidence in the process after
seeing how the screening will actually be performed.
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