“I want the country that eliminated polio and mapped the human genome to lead a new era of medicine – one that delivers the right treatment at the right time. In some patients with cystic fibrosis, this approach has reversed a disease once thought unstoppable. Tonight, I’m launching a new Precision Medicine Initiative to bring us closer to curing diseases like cancer and diabetes – and to give all of us access to the personalized information we need to keep ourselves and our families healthier.” – President Barack Obama, State of the Union address to Congress, 2015.
During the 2015 State of the Union address to Congress and the nation, President Barack Obama outlined a number of ideas and programs, including some related to medical research and the advancement of treatments and cures. One of the most central themes the President discussed related to medicine was the emerging field of precision medicine. Precision medicine is a field which aims to leverage genomic data together with an individual’s own medical history and disease symptoms and progression to more rapidly identify potential therapies.
“We applaud the President for recognizing the opportunity of precision medicine during the State of the Union this year, and see it as a call to action to continue to accelerate the first precision medicine program for ALS. At the Institute, we have long believed that precision medicine is essential to the future advancement of treatments and cures for ALS,” said Steve Perrin, Ph.D., CEO and CSO of the ALS Therapy Development Institute.
The ALS Therapy Development Institute began planning its precision medicine initiative in 2013 and announced a call for volunteers in August 2014. The program is unique in that it includes efforts to identify subtypes of ALS against which potential treatments can be screened in a high-throughput manner using the latest technology available. The Institute’s Precision Medicine Program will integrate a patient’s genetic code obtained by a full sequence of their genome, a patient’s phenotypic data characterized by monthly self-reporting questionnaires, unbiased accelerometer data, voice recordings, and the creation of an induced pluripotent stem cell line (iPS) which will be used for drug screening in the research lab. The Institute is providing all participants with access to the data generated using a secure online portal. Participants will be able to track their disease progress by viewing changes in accelerometer, speech and other self-reported data, see the results of their genome sequencing in relation to the currently known 30+ ALS-associated genes, and view images of their own skin and iPS cell lines created at the Institute.
When the Institute first launched the program, it was expected to enroll up to two dozen people. However, with funding from the social media phenomenon the Ice Bucket Challenge, the Institute was able to expand enrollment to hundreds. It will enroll 100 participants by the end of March 2015.
“As a person enrolled in the Precision Medicine Program at the ALS Therapy Development Institute, I am humbled to know that a little piece of me may be able to speed up finding a treatment for ALS. I want to spend my life with my family and continue doing all the things that I love. To have the President talk about precision medicine in the State of the Union was amazing, and I hope it draws more support to this effort,” said Jay Smith (pictured here at the time of enrollment) who was diagnosed with ALS in early 2014 and founded the Every 90 Minutes Foundation which supports the work of the ALS Therapy Development Institute.
“I am so proud to see precision medicine being applied to ALS, and to have that being done here at the ALS Therapy Development Institute. The President’s support for this type of innovative medical research is so important to me, and families like mine, who have faced ALS directly in our homes, in our loved ones. Thank you, President Obama, for making precision medicine a priority,” said Jenny Gore-Dwyer, board member of the ALS Therapy Development Institute who attended the State of the Union address at the Capitol Building in Washington, D.C. Dwyer’s husband, Patrick Dwyer was diagnosed with ALS in 2005, ending his journey with the disease in 2013. She plans to enroll in the Institute’s precision medicine program as a healthy volunteer later this year.
Earlier in the day, before the State of the Union address, a delegation of ALS community leaders, including Dwyer and Stephen Gleason, met with HHS Secretary Sylvia Mathews Burwell and Acting Director of the National Institutes of Neurodegeneration and Stoke (NINDS), Walter Koroshetz, M.D. (pictured together here). Gleason, a former NFL player with the New Orleans Saints, was diagnosed with ALS in 2012. He founded Team Gleason, which has been actively involved in raising awareness of the need and use of speech generating devices for people who lose their ability to communicate due to chronic diseases like ALS.
For more information:
• Enrollment information for the precision medicine program at the ALS Therapy Development Institute
• CNN Impact Your World segment on Anthony Carbajal enrolling in precision medicine program
• Jose Cofino, PALS, on what enrolling in the precision medicine program means to him
• Kristin, Dustin and Deb Quinn, a family with a genetic form of ALS, discusses their enrollment
• University of South Carolina economics professor and PALS Stephen Finger discusses his enrollment
Media Contact: Mari Cody, ALS.net/ALS Therapy Development Institute, 617-441-7220, mcody@als.net
All precision medicine enrollment inquires to pmp@als.net.