Charcot Marie Tooth (CMT) Experts Launch National Nonprofit to Fund Only Treatments and Cures
CMT Research Foundation to Speed Dollars to Most Promising Research
Atlanta (September 19, 2018) Two experts in Charcot-Marie-Tooth (CMT), who also have the condition, today launched the CMT Research Foundation (CMTRF) as the only 501(c)(3) not-for-profit organization focused solely on delivering treatments and cures for CMT. Patrick Livney, a former chairman of CMTA and Susan Ruediger, a former CMTA Development Director, have over 20 years of collective experience working with CMT researchers to deliver treatments. By developing partnerships with researchers, pharmaceutical and biotechnology professionals, as well as a national network of CMT patients, they have been instrumental in achieving recent, successful milestones in CMT research.
CMT is a progressive genetic nerve disease and can lead to severe disability or even death. CMT has over 90 known genetic causes and can be passed down in multiple generations. Onset can be at birth or later in life and is characterized by loss of muscles and sensation in the feet, hands, legs and arms. Nearly three million people of every age, race and ethnicity are living with CMT. Currently there is no cure for CMT; and there are not any effective treatments.
"The CMT Research Foundation will play a unique role in the CMT community by limiting overhead and focusing on funding research that will help speed cures not only for various levels of CMT, but also other neurological degenerative diseases including ALS and Alzheimer’s," says Mr. Livney. “Our bold strategy of funding CMT drug delivery is driven by a passion to help patients across the country, many who have more severe cases of the disease than we do. We are committed to stretching every dollar to deliver faster results.”
The CMTRF will support the most promising and effective research aimed at drug delivery by developing strategic alliances with academic and industry stakeholders. With the help of an Advisory Board comprised of academic, medical and biotech experts, the CMTRF will fund translational research for all types of CMT.
“Research is what it takes to develop effective treatments and ultimately a cure for CMT and the only thing standing in the way of that research is funding,” says Ms. Ruediger. “As a patient living with CMT every day, I know firsthand how urgent it is that we eliminate this devastating condition and I won’t stop until there is a cure.”
CMTRF Board of Directors
Joining Mr. Livney and Ms. Ruediger on the CMTRF Board of Directors (still in formation) are:
Paul August, PhD, Vice-President, Biology, ICAGEN, a pharmaceutical company focused on advancing early drug discovery. Previously, he held key scientific and leadership roles for over 18 years with the global pharmaceutical company Sanofi. Dr. August began his Sanofi career in 1998 at the Hoechst-Ariad Genomics Center in Cambridge, Massachusetts. In 2010, he relocated to Tucson, Arizona where he integrated biology-related activities to form the Discovery Biology department at the Sanofi Tucson Innovation Center. In this role, Dr. August has pursued innovative, entrepreneurial, business, and scientific strategies to identify and develop innovative therapeutics, taking the lead on the CMT discovery project. His efforts have focused on building external partnerships with academic investigators, biotechnology companies, and rare disease patient foundations. Dr. August’s research has been particularly focused on developing disease-relevant, human-stem-cell-derived models. Leveraging the high throughput infrastructure and novel chemistry capabilities at the Sanofi Tucson Innovation Center, Dr. August and his team have applied these models and state of the art stem cell technologies as a means to create transformative therapeutics for patients.
Adam Kauffman has been a software engineer for the past 20 years specializing in internet security and managing the development of products for Fortune 500 companies, banks and the federal government. He has been the Engineering Manager at Pindrop since March 2016 and for 9 years before that, was Staff Software Engineer at Damballa. From 1998 to 2006, Mr. Kauffman was a Senior Software Engineer with EarthLink. He is a graduate of the Georgia Institute of Technology.
In his personal time, Mr. Kauffman volunteers in his community. He has served on the executive board of his neighborhood civic association, as a mentor with his alma mater and with his local elementary school. He is an Eagle Scout, who is still active in scouting with his local Pack and kids. He has CMT1A as do his mother and sister. Mr. Kauffman and his wife, Kate, live in Brookhaven, GA with their children, James and Sam.
Michelle Moon is a board certified general neurologist in practice for 10 years. Her motivation for serving on the board of CMTRF is deeply personal: in 2016, she lost her 5-year old daughter to a severe and rare form of the disease. Dr. Moon’s story received international attention on CNN and in People Magazine. She was pleased to bring attention to the little known but not-so-rare disease, but her ultimate goal is to find a treatment. She continues to tell Julianna’s CMT story and the story of a grieving family on the blog (juliannayuri.com) and throughout social media.
Dr. Moon brings her clinical expertise and her personal passion to help CMTRF deliver treatments with the goal of preventing other families from suffering the devastating loss of a child to CMT.
Dr. Moon received her medical degree from Nova Southeastern University and completed her neurology residency at the University of Colorado. She lives in Washougal, Washington with her husband, Steve, and son, Alex.
Cleary Simpson has more than 30 years of experience in the media industry. She held senior positions in sales, marketing, business development, and general management at Time Warner working with major brands and advertising agencies. Most recently, she was Managing Director at consulting firm MediaLink, advising early stage digital ventures as well as mature media companies on business strategy, revenue acceleration and organizational restructuring. Before that, Ms Simpson was Senior Vice President for Global Marketing Solutions at Time Warner Inc., managing multi platform partnerships with iconic national brands. Earlier, she worked in leadership roles at several Time Inc. properties including TIME Magazine and Sports Illustrated.
Ms Simpson served on the boards of Do Something.org, and of St. Lukes', a private school in New Canaan, CT. She holds a BA in History and Asian Studies from Connecticut College and was a Fulbright Fellow in Japan.
Her daughter, Anna, has CMT1A. Ms Simpson believes that CMTRF's singular focus on funding research will accelerate the identification and delivery of a cure for CMT.
Scientific Advisors to CMTRF (still in formation):
Richard Shimkets, PhD
President and CEO, Abeome Corporation
Dr. Shimkets serves as the President and CEO of Abeome Corporation. Prior to joining Abeome, he served as Vice President of Drug Discovery and Vice President of Scientific Development for CuraGen Corporation where he developed and managed partnerships with Genentech, Biogen, Bayer, and other biotechnology and pharmaceutical companies. Dr. Shimkets earned his BS from University of Georgia and his Ph.D. from Yale University.
The CMT Research Foundation (CMTRF) is the only organization focused solely on delivering treatments and cures for CMT. Founded by two patients who are driven to expedite drug delivery to people who live with CMT globally, the organization funds research and drug development. The 501(c)(3) federal tax-exempt organization is supported by personal and corporate financial gifts.
Additional Founders Bio Information
Patrick Livney, CMTRF's co-founder and Chairman of the Board of Directors, has CMT type 2 and is one of the world's foremost pioneers in the search for a cure for the disease. He served as the CEO of the Charcot Marie Tooth Association (CMTA) for five years, and prior to that, was the Chairman of the CMTA’s Board of Directors for four years.
Mr. Livney's leadership led to the vision and inception of the CMTA’s STAR Research platform, dedicated to treating CMT through drug development. He gathered the world’s brightest minds in CMT research, created a culture of collaboration, and built an initiative which delivered significant milestones in CMT translational research.
Mr. Livney is currently the CEO of Loan EcoSystem Online. Previously, he was the Senior Managing Director & Head of the Structured Finance Group at Vanderbilt Capital Advisors where he grew assets from $300M to over $20B in four years. Mr. Livney served as the CEO and Chairman of the Vanderbilt Financial LLC. Earlier in his career, he held senior positions in Institutional bond sales for Wall Street firms. Mr. Livney holds a degree in industrial engineering from Roosevelt University in Chicago.
Susan Ruediger, CMTRF's co-founder and CEO, was diagnosed with CMT1A when she was 18 months old and has traced the genetic disease back five generations in her mother’s family. She later started a local CMT support group in Atlanta to generate information about and resources for CMT research.
Recruited by then CMTA Chairman of the Board, Patrick Livney, Ms. Ruediger worked with the not-for-profit as Director of Development for eight years, deeply engaging with the patient and research communities. She led the CMTA's development and marketing strategies and served as its national spokesperson. Ms Ruediger's efforts led to a 380% increase in annual revenue for CMTA. She brings strategic planning, operational, communications, fundraising and non-profit management experience to the CMTRF.