In 2008, the CMTA’s Board of Directors launched its Strategy to Accelerate Research, or STAR. It was based on two important ideas:
Idea #1: The Genes That Cause CMT Are Known.
We know the causes of many types of CMT. The big breakthrough came in 1991 when PMP22, the gene that causes CMT1A, was discovered. To date, more than 100 different genes that cause CMT have been identified and more are being discovered each year. Once we identify a gene that causes CMT, we can duplicate it in the laboratory. This is the foundation of the STAR strategy. As a famous inventor put it: “A problem well stated is a problem half solved.” This is true of CMT, unlike many other diseases whose causes are either unknown or very complex.
Idea #2: Manage Research According to Sound Business Principles.
STAR is based on five core business principles:
The Core Principles of STAR:
Develop a strategy based on knowing the cause of the disease and where to focus.
Find the best researchers in the world and ask them to implement projects that support the strategy.
Create accountability and hold those researchers accountable for achieving their goals. We take your money very seriously. Our researchers are not fully paid until they fully deliver.
Demand collaboration, bringing 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.
Encourage partnerships – It costs between $400 million and $1 billion to bring a new drug to market. The CMTA does not have this kind of money. We have to work with pharmaceutical companies that have the resources to develop drugs. In the end, they will carry the ball over the line for us.
Our strategy also has five key elements:
Assays – Assays are tests. We recreate CMT in Petri dishes to help test and develop new drugs. We can also use them in high-throughput screens (HTS) to test hundreds of thousands of drugs to see if they improve CMT.
Animal Models – Animal models that replicate the disease and its symptoms are absolutely critical to drug development. When these models do not exist, we create and evaluate them ourselves. If they already exist, we provide preferential access and expert analysis to our partners through our extensive testing infrastructure.
Stem Cells – We take human skin samples and put them through a stem cell process to create neurons (nerve cells) and Schwann cells (which make myelin). This way, we create assays that better represent human biology. We have developed models for CMT1A and CMT Type 2.
Partners – Thanks to the testing tools, models, and expert infrastructure we have developed through STAR, pharmaceutical companies are contacting us in growing numbers to test their drugs and technologies in CMT. Some companies use the traditional “small molecule” approach, while others are leaders in the latest genetic and neurological technologies such as CRISPR, gene therapy, gene silencing and axon and muscle regeneration.
Clinical Trials – We are working in partnership with the INC and our centers of excellence to get ready to conduct clinical trials. This means recruiting large numbers of patients, studying the evolution of CMT in patients and developing outcome measures and biomarkers to measure a drug’s effectiveness quickly and conclusively.
How do we work?
The CMTA’s STAR Advisory Board includes 30 of the top scientists in CMT and related fields. It comprises a Scientific Advisory Board, a Therapy Expert Board and a Clinical Expert Board.
The CMTA STAR Advisory Board
The Scientific Advisory Board (SAB)
Provides scientific input for projects that are ongoing or proposed
John Svaren, PhD, Chair
University of Wisconsin
The Therapy Expert Board (TEB)
Ensures that each research project has translational value
Mark Scheideler, PhD, Chair
HumanFirst Therapeutics LLC
The Clinical Expert Board (CEB)
Provides expert guidance and support to the CMTA’s alliance partners to assure the success of clinical trials
Michael E. Shy, MD, Chair
University of Iowa
The CMTA has teams of experts focused on specific subtypes.
John Svaren, PhD
University of Wisconsin
Maurizio D’Antonio, PhD
San Raffaele Scientific Institute, DIBIT
Steven S. Scherer, MD, PhD
University of Pennsylvania
Robert H. Baloh, MD, PhD
Cedars-Sinai Medical Center
Mario Saporta, MD, PhD
University of Miami
We have come a long way since the inception of STAR in 2008. Over the last 10 years, the CMTA has financed dozens of STAR projects and spent over $10 million on research. We are spending your dollars wisely (our overhead in amongst the very lowest) and in a very focused manner (enabling large investments from the industry) We are spending 10 times more on research than when we initially started. Thanks to the support from all our donors, there is huge momentum and promise.
STAR Research Updates