The ALS Therapy Development Institute has just launched a new initiative within its Precision Medicine Program called, “PMP 2.0.” The goal of PMP 2.0 is to rapidly collect massive amounts of additional data to determine the viability of potential new clinical trial endpoints as well as assist in the identification of subtypes of ALS.
In 2014, the ALS Therapy Development Institute introduced “PMP,” the world’s first precision medicine program in ALS. The goal of the program has been, and will continue to be, to identify subtypes of ALS and discover and advance into clinical trial-specific treatments for each. Two years on, we can share a summary of how the program has unfolded, as well as what we have learned to date.
1. We enrolled 300 people from 12 countries in the program. We have completed full sequencing of more than 270 ALS patient genomes, with more in the queue.
2. We have observed that accelerometer data are more sensitive in their ability to not only detect changes in disease progression but to monitor the spread of disease from an affected to a non-affected limb. These studies may change the way ALS clinical trials are designed and executed.
3. We have observed measurable changes in participants' voice recordings over time as a patient progresses in their disease. We are building statistical models that will allow clinicians to stage disease progression of an ALS patient remotely using voice recordings.
4. This is the only Precision Medicine Program for any disease that is actively making available these data and others, such as genetic variations, with the participants in the study as the data are generated.
5. The Precision Medicine Program cohort is an excellent representation of the genetic diversity described in ALS, as well as likely the much more common sporadic forms of the disease.
PMP 2.0 is an extension of PMP, and is designed based on discoveries that signal the opportunity to advance subtype identification by adding data from more and more people with ALS. We have been approved to enroll 450 participants in PMP 2.0. Both program components will run simultaneously.
The primary objective of PMP 2.0 is to develop a detailed comparison of self-reported ALSFRS-R to monthly voice recordings and monthly accelerometer data. We have learned that non-invasive collection of movement data with accelerometers and repeated voice recordings are more sensitive at detecting disease progression compared to ALSFRS-R. In PMP 2.0, participants will facilitate accurate statistical modeling of the data to assess the ability of these new technologies to improve outcomes in Phase II and Phase III ALS clinical trials. We hope to present the results of these findings to the FDA in 2017.
“We believe that every person with ALS has a role to play in the search for effective treatments,” says Steve Perrin, Ph.D., CEO and CSO of the ALS Therapy Development Institute. “The more people who participate in PMP 2.0 by generating demographic, speech and when possible motion data, the greater advancement we can make in our goal to end ALS. Each PMP participant becomes a partner in research and will have access to their own data in real-time, making this uniquely transparent approach to clinical research.”
PMP 2.0 is open for enrollment. To learn more about the program and to participate, please visit https://www.als.net/pmp2
About the ALS Therapy Development Institute
The ALS Therapy Development Institute and its scientists actively discover and develop treatments for ALS. The Institute is the world’s first and largest nonprofit biotech focused 100 percent on ALS research. Led by people with ALS and their families, the organization understands the urgent need to slow and stop this disease. Based in Cambridge, MA, the Institute has served as one of the leaders in sharing data and information with academic and ALS research organizations, people with ALS and their families. For more information, visit als.net.