ALS TDI Receives Department of Defense Grant to Accelerate ALS Biomarker Discovery
Posted: April 1, 2022 in Other News
ALS TDI
CAMBRIDGE, MA – The ALS Therapy Development Institute (ALS TDI) has been awarded a $281,000 grant from the United States Department of Defense Congressionally Directed Mandated Research Program for ALS (ALSRP) to support a new study designed to identify biomarkers for ALS. Leveraging PMP data and cutting-edge technologies, this study represents what may be an unprecedented opportunity in the pursuit of ALS biomarkers. Discovery of biomarkers for ALS has always been a key goal of ALS TDI’s Precision Medicine Program (PMP), and this grant from the ALSRP will allow ALS TDI to significantly expand this effort in 2022.
With this funding ALS TDI will perform a study to identify blood-based biomarkers by examining samples from PMP participants who contribute blood samples over a span of one year. This work will be performed in collaboration with SomaLogic™, a leading protein biomarker discovery company, and with our research partner Dr. Michael Brenner, the Michael F. Cronin Professor of Applied Mathematics and Applied Physics and Professor of Physics at Harvard University.
Reliable blood-based biomarkers for ALS would represent an opportunity to better diagnose the disease and more accurately track disease progression – and could potentially serve as a more precise endpoint for human clinical trials. By funding biomarker discovery, the ALSRP grant will advance ALS TDI’s mission by informing our research and accelerating the discovery of effective treatments for everyone with ALS.
A year-long study to expand the PMP’s search for blood-based biomarkers
A key component of the PMP’s search for biomarkers is our first-in-kind in-home blood collection program. Already, in this program’s three years of existence, it has allowed us to gather regular samples from dozens of participants without requiring them to leave their homes. However, sending a mobile phlebotomist to each participant’s home to draw the blood, safely shipping this sample to our lab in Massachusetts, and testing each sample represents a significant expense.
With this grant, we will be able to expand the in-home blood collection program to include more than 100 participants. In this expanded cohort, PMP researchers will conduct a study consisting of four blood draws conducted quarterly over one year. Tracking the levels of different proteins and other biological features in participants’ blood samples over time and comparing them with data about each individual’s disease progression could point the way to potential blood-based biomarkers that relate to and predict disease progression rates.
Accessing new technology to search for protein biomarkers
The ALSRP grant will also enable ALS TDI to utilize new technology to markedly increase the number of potential biomarkers we can analyze in each sample from this study. Each of the quarterly samples will sent to a company called SomaLogic™ in Boulder, CO for protein analysis. SomaLogic™ will analyze these samples using SomaScan™, a unique, proprietary proteomics testing platform that is able to analyze the levels of more than 5000 different proteins in a small sample of blood plasma.
This new, cutting-edge assay represents an opportunity to analyze samples at a greater magnitude, and in more detail, than has been possible up to this point. Previously, even using some of the best commonly available technology, PMP researchers have been limited to elements similar to what might be tested at doctor’s appointment in their search for blood-based biomarkers – things like glucose, calcium, bilirubin, or cholesterol.
The SomaScan™ platform will allow the PMP team to observe the concentrations of thousands of cytokines, growth factors, proteases, protease inhibitors, kinases, structural proteins, hormones, and other proteins in each sample. SomaScan™ has previously been used by researchers to discover promising potential biomarkers in conditions such as Crohn’s Disease, Alzheimer’s Disease, and rheumatoid arthritis.
Working with Harvard researchers to analyze biomarker data
In addition to blood samples, ALS TDI also collects a wealth of data about ALS progression from PMP participants. These include self-reported ALSFRS-r scores, limb movement data gathered with digital accelerometers, and voice recordings. For several years, we have been working with Harvard’s Dr. Michael Brenner and Google artificial intelligence researchers to analyze this data and utilize it to create new tools for track ALS disease progression. Recently, ALS TDI, Dr. Brenner, and Google announced a new product of this collaboration – a machine learning algorithm capable of predicting people living with ALS’ ALSFRS-r voice scores based on these recordings.
By comparing these ML-generated voice scores, accelerometer readings, and ALSFRS-r data with the results of the four rounds of proteomics testing with the SomaScan™ platform, we aim to find evidence of any changes in proteins that may correlate with disease progression – revealing potential biomarkers for further research. With more than 5000 proteins in four samples for 100 people, this study will generate a massive amount of raw data. ALS TDI will work with Dr. Brenner to use their machine learning software and expertise to analyze sample data and search for biomarker “fingerprints” that could predict disease progression.
No previous study in ALS has leveraged these powerful new technologies – the powerful SomaScan™ proteomics platform and Dr. Michael Brenner’s industry-leading machine-learning data analysis expertise – in this way. Combined with the PMP’s long-established data collection methods and our ability to facilitate regular in-home blood collection, this represents what may be an unprecedented opportunity in the search for ALS biomarkers. Additionally, once any positive results from this study have been validated, ALS TDI plans to continue to work with SomaLogic™ to develop commercial testing kits for any discovered biomarkers for use in clinical trials and ALS case management.
