About The Precision
Medicine Program

The Precision Medicine Program (PMP) is the most comprehensive and longest running translational research study in ALS. Through PMP, researchers at ALS TDI partner with people with ALS around the world to share and gather data on voice, movement, lifestyle, medical and family histories, genetics, biomarkers, and patient cell biology to better understand the disease.

Only by listening to people with ALS and collecting unbiased data will we develop a better understanding of how to slow or stop ALS. With your participation in PMP, we can accomplish our goals of:

  • Discovering new targeted treatments for ALS.
  • Making clinical trials faster and more efficient.
  • Empowering people with ALS by giving them access to their own data.

Only by working in true partnership with people with ALS can we create a comprehensive database and enhance our knowledge of this disease.

Why Should You Participate?

By participating in ALS TDI's Precision Medicine Program (PMP), you become a partner in ALS research to help find effective treatments for ALS while gaining access to helpful tools regarding your disease progression.

Enrolling in our PMP can help you and your medical team make informed decisions about your own treatment and care. Unlike other research studies, our PMP gives you full access to all the data you provide. You will be able to:

  • View your data in your own secure, easily accessible online portal.
  • Track your disease progression and follow the impact of various interventions on your symptoms and progression.
  • Access regular news and updates through your private, password-protected portal and our exclusive online webinars.
  • As a PMP participant you will know that your data and your experience with ALS are part of the world's most focused and experienced effort to end ALS.

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.

Sign Up For PMP
Access The PMP Portal

Andrea Peet, PMP participant
“I'm grateful to have the opportunity to participate in the PMP. In addition to allowing me to understand and track my disease progression, the PMP makes me feel like I am a true partner in ALS research. It's meaningful for me to know that my data may help ALS TDI to discover potential new treatments for ALS.”
- Andrea Peet, PMP participant
Data Collection Data Collection
Participation and Data Collection
Signing up for our PMP is easy. Anyone with ALS can add their data to PMP—regardless of where you live or what stage of the disease you are at. Click here to provide some basic information to get started. You will be sent an email activation link where you can begin adding the first data points needed in our PMP, including reviewing important HIPAA 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 able to build on this information to help us learn more about the processes that drive and influence each person's ALS.
By signing up for the PMP, you may be asked for data related to:
Personal Background
Personal Background
Complete surveys on topics such as family history, geography, lifestyle, medical history, occupation and your ALS experience.
Speech Tracking
Speech Tracking
You will be able to record a sample phrase each month to assess changes in your speech.
Activity Tracking
Activity Tracking
Participants who meet specific criteria* will be provided with accelerometers on a monthly basis to quantify movement.
Sample Collection
Sample Collection
Participants who meet specific criteria* will be provided with an in-home blood kit or other types of sample collection kits.
ALSFRS-R Tracking
ALSFRS-R Tracking
Complete an online ALSFRS-R** form once a month.
Genetic Testing
Genetic Testing
Participants who meet specific criteria* will be provided with genetic testing information.
*If you meet these criteria, you will be contacted by a member of our Clinical Operations Team.
**The Revised ALS Functional Rating Scale (ALSFRS-R) is a questionnaire-based scale used to measure participant’s ability to perform physical activities in everyday life.

PMP Progress

How many participants have enrolled in PMP?
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 experiments to assess C9orf72 intronic repeat length in DNA from PMP participants have been completed?
530

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 who have been found to have a pathogenic repeat length associated with the C9orf72 gene.

How many blood samples have been collected and analytes assessed within them?

NOTE: The graph above presents two values. The number of samples collected represents the total number of samples of blood collected, we often collect multiple samples of blood from individual patients. The number of biomarker assessments to date represents the number of analytes explored multiplied by the number of blood samples tested for those analytes. Analytes of interest to ALS TDI include proteins such as SOD1 or CD40L, as well as antibodies against TDP43, among others.

Initial PMP Outcomes

Our aim is to enroll 750 people with ALS in our PMP. Our PMP includes participants from nearly every US state and over 40 other countries. This pioneering partnership has resulted in the creation of the largest, patient-linked database integrating voice recordings, movement data, medical histories, family histories, genetics, biomarkers, and patient cell biology.

Participants actively provide valuable data every month. As of July 2019, PMP participants have provided:

  • More than 13,500 voice recordings.
  • More than 15,000 accelerometer activity data sets.
  • More than 10,000 ALSFRS-R scores.
  • More than 21,000 survey responses related to drug, supplement use and other health topics.
Voice Tracking
Once a month, participants are encouraged to record sample phrases to help us interpret changes in speech. To date, we have collected over 13,500 speech recordings from people with ALS.
We collaborate with researchers at Google to leverage their expertise in artificial intelligence to analyze the "big data" collected from our PMP. This research partnership resulted in the development of an algorithm that accurately generates the ALSFRS-R speech score by simply analyzing the series of short recordings of a person's voice. Using this algorithm we have made significant strides towards the development of unbiased, quantitative and sensitive measures of ALS disease progression.
Wearable Technology
Wearable technology is hugely popular in personal healthcare. In diseases like ALS, where a person is robbed of their ability to move, wearable technology could play a significant role in helping to track disease progression and ultimately be used as an unbiased and sensitive measurement tool in ALS clinical trials.
For the first time, thanks to the hundreds of people enrolled in the PMP, scientists at ALS TDI were able to begin to explore the potential of wearable technology in ALS. They analyzed data from multi-limb accelerometer readings collected monthly from hundreds of people with ALS and compared the data to reported ALSFRS-R scores, the standard outcome measure used in interventional clinical trials.
While additional validation of the model is needed, initial analysis suggests that the use of accelerometers in an ALS clinical trials may reduce the length of a trial and number of participants required by half.
Finding The Best Leads To Treat ALS
We are now at a pivotal point in our PMP. Our Translational Research Team has been developing a plan to reprogram human induced pluripotent stem cells, or iPSCs into ALS-related cells in preparation for cell-based drug screening.
Our next goal is to acquire a compound library to screen in these ALS-related cells. Compound libraries consist of tens of thousands, to hundreds of thousands, of small molecules, which we could screen rapidly in these ALS-related cells. We hope to use the data we gather from these experiments to identify which molecules have a positive impact in the cells, making them good starting points. Promising leads will be moved on to the next stage of drug development, involving more tests in other models of ALS to assess toxicity and, in turn, efficacy.
Screening thousands of small molecules in cells represents a significant shift in our scope. It has the potential to identify better quality leads, earlier on in the process, allowing us to pursue great ideas, faster.
As we continue our working partnership with PMP participants, Google scientists, and other collaborators, we will continue to report on findings from analyses of genetics, blood based biomarkers, clinical disease progression metrics, and cell biology experiments to discover therapeutic targets and streamline clinical development processes for ALS.
Steve Perrin, Ph.D., CEO at ALS TDI
“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 at ALS TDI
×
December 3 is
Giving Tuesday
Celebrate #GivingTuesday by helping to
fund vital ALS research at ALS TDI.