The development of new drugs for rare diseases is hampered by the lack of precise and reliable clinical outcome assessments (COA). Amyotrophic lateral sclerosis (ALS) and other motor neuron diseases (MND) are no exception to this. There are very few disease-modifying treatments available; the most effective one, Riluzole, was approved 30 years ago1 and offers only a modest benefit to patients.
The standard primary endpoints for ALS research are respiratory function, survival, and the ALS Functional Rating Scale - Revised (ALSFRS-R). The lack of sensitivity of these endpoints may result in missing the benefit of certain treatments for patients with ALS, preventing novel treatments from entering the market.
Wearable digital health technologies (DHT) present promising opportunities to close this evidence gap, which is why ActiGraph created the Digital Endpoint Accelerator Research “DEAR” Grant. This grant supports academic research projects that validate digital endpoints in specific clinical populations to lower barriers of adoption for pharmaceutical sponsors and regulators.
As opposed to standard COAs, digital endpoints collected by validated, research-grade wearable DHTs are:
High frequency: Instead of weekly or monthly assessments for the ALSFRS-R, actigraphy provides many data points per second.
Low-burden: No action is required from the study participant beyond wearing a watch. Data is uploaded seamlessly and continuously, opening the possibility of long-term remote monitoring.
Objective: Digital measures have no tester or recall biases.
Patient-centered: Actigraphy captures the performance and behavior of participants during their daily lives, from the comfort of where they live while performing activities that matter to them.
Through the DEAR Grant, ActiGraph is partnering with Cory Holdom, a researcher at the University of Queensland with a background in neurology and developmental biology. Cory Holdom’s main area of research focus has been understanding the impact of MND, primarily through learning about the patient perspective and evaluating novel biomarkers for the disease.
MND is characterized by progressive wasting and paralysis in muscles, which can impact patients’ gait in various ways, including increased risk of mobility problems and falls. Actigraphy can potentially be used to passively monitor these changes and could soon be incorporated into future trial design. With the DEAR Grant, Cory Holdom and his research team will work to validate actigraphy-derived measures of gait that can be applied to study MND progression in future clinical trials.
This research project is one of five that have been selected to receive the DEAR Grant, all of which will progress over the course of 2023. Evidence generated from these studies will reduce friction for the adoption of wearable data as patient-centered outcomes, ultimately improving drug development efficiency.
1In April of this year, the FDA approved QALSODY for amyotrophic lateral sclerosis (ALS) adults who have a mutation in the superoxide dismutase 1 (SOD1) gene. This endotype accounts for about 1% of the ALS case.