How personal loss sparked a coordinated international effort to accelerate ALS research
ALS, or amyotrophic lateral sclerosis, is a devastating, incurable neurodegenerative disease the causes of which are not known. Over time, the disease robs its victims of the ability to control their muscles and most experience a slow, excruciating decline. It is also devastating for the victim’s loved ones who do all they can to alleviate suffering but are, in the end, powerless to stop the disease’s progression. Mike Piscotty, an “IT guy” at Lawrence Livermore National Lab (LLNL), was thrown into this world of pain and grief when his wife, Gretchen, was diagnosed and, ultimately, passed away from ALS. While others may have been incapacitated by this loss, Mike chose to act.
From Grief to Action
Mike is not a doctor nor a medical researcher, but he is persistent and passionate in his efforts to support work toward a cure for ALS. He wanted to understand more about the disease and why it was such an intractable problem. He searched the internet for information, read relevant research papers, and attended conferences of medical researchers to better understand the current state of the research. He soon realized that while there were researchers around the globe working to unravel the mysteries of ALS, there was no strategic, coordinated plan of attack to understand and cure the disease. He also read reports of work at LLNL that used the lab’s supercomputers to model processes related to cancer and traumatic brain injury and wondered if these approaches might apply to ALS.
Mike worked with colleagues at LLNL Foundation to secure funding to inform other LLNL researchers about ALS who then used the lab’s supercomputer to model PDP43, a key protein found in 95% of ALS cases. While this was an important step, Mike realized that curing ALS would require the efforts of researchers from diverse fields and expertise and that those efforts needed to be aligned in order to make significant progress. A colleague at the LLNL Foundation knew of Knowinnovation (KI) and encouraged Mike to reach out.
Building the Roadmap
Mike founded the ALS Cure project (https://www.alscure.org/) and has worked with KI to host three virtual international symposia with the goal of finding a cure for ALS in the shortest time frame possible. Mike urged symposium attendees “to harness the passion and hate that each of us feels for ALS and turn that into innovative thinking and collaboration.” A defining output from the first symposium is the “Roadmap to Cure ALS” that identifies key milestones and multiple research paths that can be pursued to achieve those milestones (see figure). Symposium participants at subsequent gatherings have refined the roadmap as new knowledge emerges. The roadmap has also served as an organizing structure for seed funding for which international teams of symposium participants can apply.
The Power of Global Coordination
An essential element of the symposia is the active participation of researchers from different disciplines and different countries. As Mike points out, “International collaboration is critical. ALS is somewhat rare and there is a limited amount of data and funding available. Countries tend to spend money on diseases that more people are impacted by. This disease is far too complex to be working on just one part of “the elephant.” Leveraging resources around the world gives us hope to achieve a cure.” The symposia are designed to foster conversations between diverse researchers that will lead to innovative approaches to accelerate research that can lead to a cure.
Dr. Jonathan Cooper-Knock, a researcher at Sheffield University in the UK, has participated in all three symposia and served on the ALS Cure Project research council. His participation in the symposia has led to new research collaborations. “The thing that’s most valuable for me are those discussion groups. Every time I’ve been, it’s changed my research. It’s given me ideas and collaborations have come from it. That’s the real game changer.” He adds that the roadmap “crystallizes a set of priorities” and allows researchers to make explicit connections between different elements of the field that may, in turn, lead to changes to the map.
Through the ALS Cure Project, Mike continues to raise funds that will support on-going, collaborative, coordinated research into the ALS disease mechanisms and biomarkers that are essential for developing a cure for ALS. He looks forward to “A day when a person contracting ALS is quickly diagnosed and treated with the ALS cure to stop progression.”