Precision Medicine Program

The Precision Medicine Program (PMP) at the ALS Therapy Development Institute is the world's premier partnership between scientists and people with ALS to identify treatments and cures. This pioneering partnership between scientists and people with ALS has produced outcomes that could potentially lead to clinical trials being conducted faster and easier. Thus identifying new leads on potential therapeutic targets in subtypes of ALS disease. Read more below and get prescreened for enrollment today!

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Why should you do this?

Because we need treatments for ALS already! Your ALS experience is unique and for the first time, researchers acknowledge that ALS is a complex disease with many different subtypes. Only by listening to people with ALS, collecting unbiased data about your past and your progression can we classify the subtypes of ALS and screen and target treatments more precisely. Only with your participation in PMP can we accomplish this goal. Learn More

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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PMP PROGRESS

(Last updated Wednesday, May 23, 2018)
750
0
503

  How much data has been entered in the PMP?

 

  How many cell lines have been created?

 

  How many genome sequences have been completed?

 

  How many C9orf72 repeat expansion characterization experiments have been done?

121

NOTE: In order to determine the repeat length of C9orf72 in any given individual enrolled in the PMP, scientists have to conduct several experiments. The number above is the total number of experiments done to understand the repeat lengths in the current PMP cohort. This number does not represent the number of individuals in the PMP that have been found to have a pathogenic repeat length associated with the C9orf72 gene.

 

  How many blood samples have been collected for biomarker research?

312

 

I want to sign up for PMP

Initial PMP Outcome

Scientists at ALS TDI, together with outside collaborators, have analyzed a massive amount of data. Initial analysis has led to discoveries that may accelerate all ALS clinical trials. Similarly, ALS TDI team members have already conducted significant iPSc based phenotyping experiments and are expanding that effort to prepare for high throughput, high content cell-based drug screening on prioritized drug targets. These are important initial outcomes, but the program has not yet achieved its goals and the partnership between patients and scientists must not only continue but expand.

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.

What do you get?

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What do you get?

You will know that your data, your experience with ALS, and your battle is part of the most focused and experience effort to end ALS in the world. The experience you share and the data you provide will be viewed, listened to and analyzed by the ALS researchers. and actually be used. You will be provided with your own get access to a secure online portal where all of the data you provide is located and easily accessable. lives as part of the Precision Medicine Program. You will be able to explore your own progression rates and follow how various interventions impact your progression rates. You will be able to access regular updates by checking your private password protected portal and through webinars.

“Each PMP participant becomes a partner in research and will have access to their own data in real time, making this a uniquely transparent approach to clinical research.”
Steve Perrin, Ph.D.
CEO / CSO

 

I want to sign up for PMP

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:

PMP Login

 

Tracker Activity

 

ALS FRS

 

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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How does it work?

Signing up for PMP is as easy. Click here or on any Sign Up button on this page. You will be sent an email activation link which will get you going on building the first data points needed in the PMP, including reviewing important HIPPA compliance and informed consent forms as well as answering basic medical questions about you and your experience with ALS so far. From there, you will be enabled to add thousands of pieces of information to the battle to identify subtypes of ALS against which more precise treatments can be screened. See the graph below for more information about PMP works for you and researchers 100% focused on ending ALS at the ALS Therapy Development Institute.

I want to sign up for PMP

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!

Impact of Ice Bucket Challenge on ALS Research (June 9, 2015)

Precision Medicine Program Data & Update (February 9, 2016)

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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WHAT PARTICIPANTS CAN EXPECT

 

Prescreen

Enroll

Enroll online to get started in the Precision Medicine Program.

Speech Tracking

Speech Tracking

You will be able to record a sample phrase once a month to help us interpret changes in speech.

ALSFRS-R Tracking

ALSFRS-R Tracking

You will be asked to complete an ALSFRS-R form once a month online.

The Revised ALS Functional Rating Scale is a questionnaire-based scale used to measure participant's ability to perform physical activities in everyday life. Data can be useful in pre/clinical trial.

Surveys

You will be asked to complete surveys on topics such as family history, geography, lifestyle, medical history, occupation and your ALS experience.

Activity Tracking

Activity Tracking

Not everyone enrolled in PMP is required to do movements (compared to the first phase of PMP when these were required by all). Instead, our team will identify participants whose profiles meet criteria and they will be provided with accelerometers. If you meet these criteria, you will be contacted by a member of our team.

Sample Collection

Sample Collection

Not everyone enrolled in PMP is required to donate samples (compared to the first phase of PMP when these were required by all, and have since been used to generate hundreds of genomes and cell lines). Instead, our team will identify participants whose profiles meet specific criteria and they will be provided with an in-home blood sample collection kit. If you meet these criteria, you will be contacted by a member of our team.

 

I want to sign up for PMP

Want to fund an end to ALS?

The ALS Therapy Development Institute is a 501c3 nonprofit organization. We are the world's largest independent ALS research organization and have advanced ALS research in revolutionary ways for the past 15 years. We breakdown walls and solve the problems inherent in the drug development process. We are the most highly rated ALS nonprofit in the world according to CharityNavigator.com. Most important to you though is how we spend the money donated to us. Fact is, we spend a greater percentage of every dollar donated on ALS research than any other nonprofit in the world. So if you want to ensure that your philanthropic donation actually goes immediately to ALS research, there is only one choice – making that gift to the ALS Therapy Development Institute.

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