Charcot-Marie-Tooth Association

The CMTA's overarching goal is to find a drug treatment for CMT. While it searches for a treatment, its goal is to provide support and education to the CMT community.

STAR IS BASED ON FIVE CORE BUSINESS PRINCIPLES:
1. Develop a strategy based on knowing the cause of the disease and where to focus.
2. Find the best researchers in the world and ask them to implement projects that support the strategy.
3. Create accountability—Hold those researchers accountable for achieving their goals. We take your money very seriously. Our researchers are not fully paid until they fully deliver.
4. Demand collaboration—We bring the researchers out of their silos to collaborate with each
other. We are now seeing more and more technologies and therapies emanating from many
different fields of study.
5. Encourage partnerships—It costs between $400 million and $1 billion to bring a new drug to
market. Like other nonprofits, the CMTA does not have this kind of money. We work with big pharmaceutical companies that have the money to develop drugs.

We created an advisory board with top-notch researchers. The Scientific Advisory Board has 30 world-class scientists. Because the work of STAR involves translational research—turning science into therapies—we created a Therapy Expert Board (TEB)–a group of experts who tell us how good the science is in terms of turning it into therapies for those with CMT. More recently, we realized we had to get ready for clinical trials so we created the Clinical Expert Board (CEB), a group of experts who are helping us and our partners think about how to design clinical trials.

For CMT1A, which accounts for approximately 90 percent of diagnosed cases, we've done animal studies, performed high-throughput screening, identified hits and worked with a company called Genzyme. Today, we're focused on two families of compounds that are being fine-tuned in the lab. Genzyme, a traditional pharmaceutical company, takes a small molecule approach, which utilizes chemical formulas. In parallel, we are also working with biotech companies, which create biological living proteins, or large molecules. For example, a company came to us with a very interesting approach—RNA interference—which inserts little pieces of DNA into nerves to affect the way the cells create the protein that overexpresses PMP22. We've seen promising results in rat testing. This technology is currently used in two approved drugs on the market.

In the decade since the CMTA’s research efforts were consolidated under the banner of STAR (Strategy to Accelerate Research), the STAR alliance has now expanded to more than 40 pharmaceutical, biotech and service partners. The CMTA has spent more than $18.5 million to finance scores of research projects with 50 of them currently active.