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.
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
ALS TDI is proud to share that our collaboration with Google has been featured in
episode two of "The Age of A.I."
a new YouTube Originals documentary series that takes an immersive look at artificial
intelligence and its potential to change the world. Watch the episode on YouTube below.
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.
In-Home Blood Collection & Biomarker Discovery
The use of sensitive biomarkers, like glucose levels for diabetes or cholesterol for heart disease, is crucial
to the diagnosis, management and tracking of a disease. In the field of ALS research, discovering a sensitive
biomarker has been difficult
due to the time, costs and logistical burdens associated with these studies.
To help solve some of these issues ALS TDI has launched the first In-Home Blood Collection program in ALS,
allowing us to collect multiple blood samples over time from the comfort of our PMP participants' homes. In
addition, we are now able to offer whole genome sequencing to participants who are unable to travel to our
labs here in Cambridge.
A participant in this program will be sent a blood collection kit and schedule an appointment with a phlebotomist
from our partner Phlebotek
. After the blood collection, the
phlebotomist will ship the samples back to ALS TDI for processing the next day. We aim to collect blood from each
participant every three months so that we can evaluate potential biomarkers' properties as disease progresses.
The large number of blood samples we collect over time, paired with all the rest of data we collect in the PMP,
gives ALS TDI an exciting and unique opportunity to robustly validate biomarkers for ALS.
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 to screen potential treatments from our compound library in
these ALS-related cells. Our compound library, which we acquired in 2019,
has 30,000 small molecules, which we hope to 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,
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.