Precision Medicine Program

The world's first fully implemented effort to identify subgroups of ALS, potential treatments for them using patient data, genomics and iPS cell technology.

Get Prescreened Invest in PMP

 

WHAT IS PRECISION MEDICINE?

Defined by the National Research Council, Precision Medicine refers to the "tailoring of medical treatment to the individual characteristics of each patient. It is the ability to classify individuals into subpopulations that differ in their susceptibility to a particular disease, in the biology and/or prognosis of those diseases they may develop, or in their response to a specific treatment. Preventive or therapeutic interventions can then be concentrated on those who will benefit, sparing expense and side effects for those who will not."

Why this technology now?

Steve Perrin, Ph.D., CEO and CSO
 

The ALS Therapy Development Institute operates the world's first and largest precision medicine program in ALS. The goal of the program is to identify subtypes of ALS and the specific compounds that treat each type to advance the most effective treatments into clinical trial.

WHAT PARTICIPANTS CAN EXPECT

Prescreen
Prescreen
Enrollment
Enrollment

Science

Sample Collection
Sample Collection

We ask you to donate the following:

  • Skin biopsy
  • Blood draw
  • Medical History
Genome Sequencing
Genome Sequencing

We sequence your genome to identify gene mutations that influence ALS.

iPSc Creation
iPSc Creation

We create and bank an induced pluripotent stem cell (iPSc) line from your skin biopsy.

Skin cells are reprogammed into induced pluripotent stem cells, which can be used to produce motor neurons and other cell types affected by ALS. These cells will be used to study mechanisms of ALS progression and search for ALS therapies.

Wellness

Activity Tracking
Activity Tracking

Accelerometers will be provided to you to wear on 6 consecutive days a month help us measure participant's ability to move.

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 you complete an ALSFRS-R form once a month.

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.

Patient Stratification
Patient Stratification

To enable better focused drug discovery and more efficient clinical trial design.

Compound Screening
Compound Screening
Advancement of Promising Treatments to Clinical Trial
Advancement of Promising Treatments to Clinical Trial

PMP PROGRESS (Last updated Sunday, June 26, 2016)

  How many patients are enrolled?

  How much data has been entered in the Wellness Program by Patients?

  How many cell lines have been created?

  How many genome sequences have been completed?

How will this help us go faster?

Fernando Vieira, M.D., director of research operations

What are iPS cells?

Jessie St. Martin, associate scientist

Your support is crucial

Jenny Dwyer, board member