STAR Pathways: Delivering
the First Treatments for CMT
The CMTA is excited to launch STAR Pathways, a fundraising initiative to deliver the first treatments for CMT. For the first time in history, we have reached major milestones with proven process and have identified the sequential steps to necessary for drug delivery. The CMTA has fostered research by some of the best peripheral nerve researchers and clinicians in the world and has engaged the active interest of several pharmaceutical companies. In addition, the CMTA has collaborated with the National Institutes of Health resulting in successful assay screening, and, more importantly, catapulting the CMTA into the research spotlight worldwide.
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There are seven steps to drug discovery and delivery: 1) building a cellular assay, 2) building a laboratory model, 3) screening the assay for potential compounds, 4) testing the laboratory model for effectiveness, safety, 5) dosage and toxicity screens, 6) phase I human trials, and 7) phase II clinical trials in patients.
The CMTA's STAR Initiative has already delivered the first three steps and is currently engaged in the fourth step for CMT 1A, with promising results. In fact, with the current progress of STAR, the CMTA expects to enter phase II clinical trials for CMT 1A within three years. Protocols and partnerships are forged to accomplish the same success for CMT 2A, 2E and X. We are proud of our progress and hopeful that in our lifetime, we will begin to see a world without CMT.
As you know, this research takes significant resources. The CMTA has established a budget specifically designed to fund the completion of CMT 1A phase II clinical trials and the first 5 steps of a second and third disease. This budget was built using competitive bids from current and future partners as well as the expertise of industry leaders. Everyone agrees that in order to achieve the success as outlined above, the CMTA will need to commit $25 million to research in the next 24 months.
For further information about each phase, the expected results and the associated costs, please click here to review the STAR Pathways campaign booklet.
And please, click here and give generously to STAR Pathways. Your gift will help end the legacy of CMT that so many families live with.