Precision Medicine Program

Signing up for PMP will ensure that you matter in ALS research. This program will enable you to add your information and your data to the battle to find treatments and cures. You should do this because without you participating actively in ALS research we won't have much needed information to defeat ALS.

PMP data is used by ALS TDI and its collaborators toward two general aims

1. identify subtypes of ALS and develop the tools to screen and select targeted treatments for testing against those subtypes
2. understand the potential use of wearable technology as a tool to accelerate all ALS clinical trials

We have IRB approval to enroll 750 people with ALS into the PMP. You can see enrollment stats below, but already scientists at ALS TDI, together with Google scientists, analyzed data donated by hundreds of people with ALS in the PMP. The researchers looked at different types of data – accelerometers or voice recordings – and analyzed them against ALSFRS-R scores, the gold standard for measuring disease progression in ALS.

Early results indicate that most of the subjective ALSFRS-R questionnaire can be replaced by at home measurements, like those used in the PMP. In short, these findings may help make ALS trials faster and less expensive. You enrolling in PMP will add crucial data needed to validate and expand on these crucial finings.

Only by creating an unbiased database of information in true partnership with people living with ALS today do we believe we can identify the subtypes of ALS and work rapidly toward screening potential treatments for each. Ready to be a partner in research? Sign up for PMP! If you have a question about PMP before signing up, email pmp@als.net.

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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.

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Unlike other research studies you may enroll in, in PMP you will get access to the data you provide. This is relatively unique as most research studies done in ALS don’t provide data back to the people enrolled in them. But we feel differently, we feel that people with ALS must be treated as partners in research, not simply the subjects of research.

While it is paramount to note that the Precision Medicine Program is a research study and no experimental medication will be provided, many people in the PMP find that by participating in the program they gain knowledge and tools to track their own disease progression. Some people use these tools to help inform the decisions they are making in collaboration with their medical team about their own treatment and care. Here are a few examples of what you will find in the secure online PMP portal to enter and explore your data:

 

Only by creating an unbiased database of information in true partnership with people living with ALS today do we believe we can identify the subtypes of ALS and work rapidly toward screening potential treatments for each. Ready to be a partner in research? Sign up for PMP! If you have a question about PMP before signing up, email pmp@als.net.

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PMP is designed based on observations made in the program already that signal the opportunity to advance subtype identification by adding data from more and more people with ALS. PMP data may also be used to understand the potential use of wearable technology as a tool to accelerate all ALS clinical trials; something, which is globally accepted by the ALS medical research community as an urgent need. With PMP we aim to collect the following as quickly as possible:

• 10,000 ALSFRS-R scores
• 10,000 speech recordings
• 10,000 exercise datasets using accelerometers
• 10,000 medical and personal history survey responses

Anyone with ALS can add their data to PMP - regardless of where you live, or what stage of the disease you are at. Got an ALS diagnosis – we need you in PMP! However, to help understand the potential power of various measures of disease progression being explored, we will ask some people with ALS that meet specific criteria set by our clinical team to wear accelerometers for example. If you have a question about PMP before signing up, email pmp@als.net. Regardless of where you are at in your disease progression, we need your data. Join PMP now!

Only by creating an unbiased database of information in true partnership with people living with ALS today do we believe we can identify the subtypes of ALS and work rapidly toward screening potential treatments for each. Ready to be a partner in research? Sign up for PMP! If you have a question about PMP before signing up, email pmp@als.net.

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