Page 5 - 2021 Spring CMTA Report - Special Research Edition
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HOW DID WE GET FROM THERE TO WHERE WE
ARE TODAY? Board members decided to apply a business
approach to the problem, starting with analyzing the
challenges. The ones they identified are still valid today.
The first is that developing drugs takes a lot of time, in many
cases more than a decade, and a lot of money, often over a
billion dollars. It’s also very risky—more than 90 percent of
the drugs that enter clinical trials fail. CMT is an even riskier
investment for pharmaceutical companies because there
hasn’t yet been a successful clinical trial. And because
CMT is a slowly progressive disease, it takes a long time and
a lot of patients to run a clinical trial. That’s what kept a lot
of potential players on the fence, and that’s what the CMTA
Board decided to address. Gilles Bouchard has led the
CMTA Board since 2016
The core strategy for STAR from day one was figuring out
how to attract partners to work on CMT. The CMTA didn’t
(and doesn’t) have the billions of dollars needed to develop The strategy is working even better than
drugs, so the focus was (and is) on attracting the top players, anticipated, resulting last year in:
top labs and top technologies. By working with researchers • $2.5 million invested in research
from the pharmaceutical industry and academia, the CMTA ($17 million since 2008)
is determined to cross the “Valley of Death” that often lies
between research and successful innovation. • 19 joint preclinical treatment studies with
leading pharmaceutical and biotech companies
Five elements proved particularly important: developing treatments for CMT
• The first one was to enlist key opinion leaders (KOLs) • 32 total alliance partners from top biotech,
to the cause. pharma and gene therapy labs around the world
• The second one was providing a preclinical testing • 50 active research projects and studies and
infrastructure so that companies could test their products eight more projects approved in recent months
quickly, inexpensively and reliably. • More than 30 of the leading CMT scientists and
• The third one was establishing a clinical trial gene therapy experts from around the globe
infrastructure, complete with patients, their natural working with our STAR Advisory Board
histories and biomarkers to measure the effects of a • Research tools for biotech companies to use in
given treatment. testing potential therapies for types CMT1, CMT2,
• Fourth, more and more companies are looking for CMT4 and CMTX
innovation that they can license and take to market. • Investments in the discovery of new genes that
All of the investments the CMTA has made over the cause CMT
years—in the labs and in new developments— Gilles attributes that success to the fact that STAR is
are now starting to draw a lot of interest from supported almost 100 percent by the CMT community.
pharmaceutical companies. That’s why the CMTA had a really good year in 2020,
despite the pandemic.
• The final factor was facilitating access to the CMTA
community so partners can understand how CMT affects One of the reasons the community supports the
patients. CMTA is that its financials are considered best-in-class,
with an overhead of just below 13 percent. Charity
MAKING CMT Navigator gave the CMTA a four-star rating and
ATTRACTIVE TO PARTNERS Guidestar gave it a Platinum Seal of Transparency,
both the highest rankings awarded by the two
organizations. People know that when they donate
to the CMTA the money will be used in the most
efficient and transparent way possible.
CMT
EXPERTS The Board of Directors is also incredibly supportive,
AND KEY OPINION providing some 20 percent of the CMTA’s revenues.
LEADERS Overall, the organization is already seeing bigger
investments—where initially the CMTA might have
CLINICAL PRECLINICAL
TRIAL INFRASTRUCTURE written a five- or six-figure check, the companies
READINESS coming in can write seven- or eight-figure checks.
Just as in the venture capital world, the seed money
ACCESS TO LICENSE that the CMTA put in is now leading to much bigger
COMMUNITY INVENTIONS investments.
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