The PMP includes participants from nearly every US state and 20+ English speaking countries. This pioneering partnership
has resulted in the creation of the largest, patient-linked database integrating ALS genetics, voice
recordings, lifestyle, demographics and accelerometer data. Participants actively provide valuable data
every month. As of January 2018, PMP participants have provided:
- More than 10,000 voice recordings
- More than 11,000 accelerometer activity data sets
- Approximately 7,000 ALSFRS-R scores
- More than 14,000 survey responses related to drug, supplement use and other health topics
In December 2017, scientists from ALS TDI presented initial findings related to work conducted and data
harvested from the PMP Program. The titles of those posters and presentations are as follows:
- Will Telehealth Revolutionize Clinical Care for ALS Patients?
- Accelerometers as Non-Invasive Tools for the Objective Measurement of Limb Specific Range of Motion and Force in ALS
- Altered Metabolic Phenotype in Cells Derived from Genome Edited Human IPSCs that Express Mutant SOD1
- Assembling a Panel of Isogenic, Genome-Edited Human IPSCs Harboring SOD1 Mutations for Cellular Phenotype Discovery
- Detection of C9orf72 Allele Expansions in a Cohort of 277 ALS Patients and Control Subjects
- Generating A Collection of Induced Pluripotent Stem Cells from Primary Fibroblast Cultures Isolated from ALS Patients
- Impaired Differentiation and Survival of Neural Progenitors Derived from Genome-Edited Human IPS Cells Lacking SOD1
LISTENING TO PEOPLE WITH ALS ASSISTS ALS DRUG DEVELOPMENT IN NEW WAY
Over the course of 2017, scientists at ALS TDI and Google collaborated on analyzing the relationships
between voice recording data and ALSFRS-R scores of PMP participants. The exciting outcome of this research partnership
resulted in the development of an algorithm that accurately generates the ALSFRS-R speech score by
simply analyzing a series of short recordings of a person’s voice. The partners continue to
improve the predictive tool by adding other data from the PMP to broaden the scope of potentially earlier diagnosis.
WEARABLE TECHNOLOGY COULD MAKE ALS DRUG TRIALS EASIER, QUICKER, CHEAPER
Wearable technology is hugely popular in personal healthcare. In diseases like ALS, where a person
is robbed of their ability to move. By finding a new way to detect and track how ALS affects
the persons ability to move, this study provides an important tool for assessing whether a drug
might slow or stope those changes and keep symptoms from getting worse.
For the first time, thanks to the hundreds of people enrolled in the PMP, scientists
at ALS TDI were able to begin to explore this question. They analyzed multi-limb accelerometer
datasets collected at multiple time points from hundreds of ALS patients and compared the data
to the standard outcome measure used in interventional clinical trials (ALSFRS-R).
While additional validation of the model is needed, initial analysis suggests that the use of
accelerometers in an ALS drug trial may reduce the length of a trial and number of patients
required by half. Peer-reviewed publication of this data will be released in 2018.