CMT1A ENDGAME
Ending the Legacy of CMT
The ENDGAME starts now:
$10 Million Dollars to Treatments and Cures for CMT1A
3 Reasons Why We’re at a Critical Point
CMT1A Facts
We Make Advances Happen
Scientific Roadmap
Two Important Success Stories
Why Your Support is Needed
It’s a Time of New Possibilities
Thank you for joining me on this journey. It is a one-way trip directly to the ENDGAME FOR CMT1A. I, along with other family members, have lived with this disease since our respective childhoods, and now I see my daughter and niece repeating the cycle. It just has to stop, and we are now in a position to make that happen.
I am a volunteer board member for the CMT Research Foundation. I am a father. And I am a staunch advocate and donor that is dedicated to developing treatments and ultimately cures for CMT, particularly CMT1A.
This booklet provides an overview of the fundraising campaign we have developed that will fund the right research, led by the leading experts we have here at the Foundation. I hope that you will consider joining me in our quest to find treatments and cures for CMT1A. I would be pleased to spend whatever time you would like answering your questions and updating you on the campaign. Please feel free to reach out to me if I may be of assistance.
For the first time in more than 45 years, I am encouraged that the science is at a point where treatments and cures are soon to be a reality. I can’t wait for that day.
There is a saying that “hope is not a strategy.” As a businessperson, I can assure you that this is true. However, I like to say that “once you have a strategy, it gives reason for hope.” We have a strategy. It is working. We are closer than ever to finding treatments and cures.
Please join me in the movement to end CMT1A.
Peter deSilva
Board Member & Campaign Chair
Former President – Retail of TD Ameritrade
Fellow, Harvard University Advanced Leadership Initiative
CMT1A: A Debilitating Disease with No Treatments Available
3 Reasons Why We’re at a Critical Point:
- Gene therapies are rapidly coming online, and new genomic testing capabilities are available.
- Pharmaceutical companies are now interested and eager to develop treatments for CMT1A.
- Companies new to CMT, in partnership with the CMT Research Foundation, have real power to catalyze and accelerate progress.
CMT1A Facts
A duplication of one gene causes CMT1A
When the duplication is silenced in mice with CMT1A, symptoms are reversed
Similar gene silencing techniques are already approved for other diseases
There are no treatments for CMT1A
CMT1A is the most common type of CMT, affecting almost 1.5 million people worldwide
Its prevalence makes CMT1A an attractive target for pharmaceutical companies
Why the CMT Research Foundation: We Make Advances Happen
The Research Foundation’s Impact Goes Beyond Simply Making Grants. Our Strategy is Clear:
- Identify key problems or gaps in understanding and target solutions.
- Increase the number of experts working to solve the disease.
- Design projects that increase the likelihood of success in CMT and are poised for commercial viability.
- Share the revenue from funded projects.
- Re-imagine the project or kill the project before money is wasted and lost.
- Commit to accountability and transparency.
The CMT1A Scientific Roadmap
The Science Exists to develop treatments for CMT1A.
The CMT Research Foundation’s Scientific Advisory Board identified five key priorities to end CMT.
Catalyzing Gene Therapies
Bypassing the Blood-Nerve Barrier
Leveraging Adjunct Therapies
Unleashing the Power of Researchers
Accelerating Clinical Trials
Why We Are Taking This Approach:
Two Important Success Stories
Scouting a New Partner to Address a Key Barrier
The Research Foundation’s partnership with DTx Pharma provides striking evidence that our approach works. In 2019 we identified a key barrier in drug development for CMT—delivering therapies to the hard-to-target cells of the peripheral nervous system.
Starting in Academia, Moving to Biotech
World-renowned CMT scientist and clinician Dr. Kleopas Kleopa had investigated the use of viral vector technology to deliver genetic therapies for two types of CMT, but not CMT1A.
Why Your Support is Needed: Imagine the Possibilities
Donors are the catalysts for the early proofs of concept to get us to our ENDGAME.
Donors are the catalysts for the early proofs of concept to get us to our ENDGAME.
- Patient funding of projects, especially those that unlock possible new solutions, are a powerful incentive for pharmaceutical industry partners with deep pockets to make additional direct investments.
- Donor dollars support CMT Research Foundation’s early funding of pre-clinical proof of concept studies that can result in new interest from new researchers.
- CMT Research Foundation shares in the profits from commercial success, then reallocates those dollars to additional projects until all forms of CMT are conquered.
Address
4062 Peachtree Road
Suite A209
Atlanta, GA 30319
Phone Number
404.806.7180