In this interview, we speak with Dr. Kuldip Dave from the ALS Association to explore their strategic approach to ALS research funding, the impact of genetic discoveries, and the legacy of the Ice Bucket Challenge. He also discusses the Association’s role in increasing access to clinical trials and ensuring trial findings translate into actionable treatments for people living with ALS.
Moe Alsumidaie: How do you prioritize research funding for ALS clinical trials?
Kuldip Dave: The ALS Association prioritizes funding by focusing on the best research globally. We use a rigorous application review process, starting with letters of intent and followed by a full application, which is evaluated by a peer-review panel. Our criteria include a strong biological rationale for the target, integration of a biomarker program within the trial, and alignment of trial outcomes with the trial phase. For instance, we focus on safety and target engagement in early-stage trials rather than efficacy. This structured approach ensures we fund the most promising and scientifically sound research. By adhering to these criteria, we are able to support research that advances our goal of making ALS a livable disease.
Moe Alsumidaie: How are you adapting to advances in ALS gene identification?
Kuldip Dave: Our strategy is to conduct more, better, and faster trials in ALS. We’ve invested in large genome sequencing initiatives worldwide, such as Project MinE and Answer ALS, which have identified about 40 genes linked to ALS. These initiatives are crucial as they lay the groundwork for targeted therapies. We also fund early-stage clinical trials and enhance trial capacity by supporting 18 trial sites. This ensures that we have the infrastructure to test new therapies as we discover more genetic links. Additionally, we encourage genetic testing for people with ALS and collaborate with myTomorrows to support patients in finding and enrolling in clinical trials, ensuring that genetic discoveries are translated into actionable clinical initiatives. This comprehensive approach is helping reduce the time it takes to go from genetic discoveries to therapeutic development.
Moe Alsumidaie: How did the Ice Bucket Challenge impact ALS research?
Kuldip Dave: The Ice Bucket Challenge (IBC) was transformative for ALS research. IBC brought unprecedented attention to ALS, transforming it from a relatively unknown disease to one that garnered global awareness. It also inspired an influx of donations that allowed us to expand our research initiatives significantly. Before IBC, we were able to award around $4 to $5 million in funding annually. Post-IBC, we quadrupled that to $15 to $18 million. This increased funding enabled us to adopt a comprehensive approach that includes not just clinical trials but also gene sequencing, trial capacity, and patient navigation to trials. The legacy of IBC is its lasting impact on our ability to fund and support a wide range of research projects.
Moe Alsumidaie: What role did you play in the approval of Qalsody?
Kuldip Dave: We played a crucial role in the development and approval of Qalsody. We were the first non-profit to fund antisense oligonucleotide research, which laid the foundation for Qalsody’s development. Our early investment of over $1.3 million in this technology was pivotal in de-risking its development and advancing it to clinical trials. When Qalsody was being evaluated by the FDA in 2023, we conducted an independent review of the data and advocated for its approval, providing testimony at the advisory committee meeting. Post-approval, we continue to address insurance barriers to ensure people with SOD1-ALS have access to Qalsody, demonstrating our commitment from research inception to patient access.
Moe Alsumidaie: How do you ensure trial findings become actionable treatments?
Kuldip Dave: We have several mechanisms to ensure trial findings translate into actionable treatments. We require that data from trials we fund be made publicly available through databases like PRO-ACT and that samples be accessible for further research. This ensures that the data can inform future research even if a trial fails. We also work with sponsors to ensure timely market access post-FDA approval and provide resources to help patients navigate payor systems. Additionally, we monitor real-world data to understand the impact of treatments outside of clinical trials, which allows us to advocate for patient access based on these findings. Our comprehensive approach ensures that research findings are effectively translated into high-quality patient care.
Moe Alsumidaie: What challenges do you face in promoting ALS clinical trials?
Kuldip Dave: One of the main challenges is increasing access to clinical trials. Currently, it takes about one month for one patient to be recruited per site, which is a slow pace for trial recruitment. This means that trials can take a long time to complete. Additionally, the time to diagnose ALS is about 12 to 14 months, which can disqualify patients from trials that require recent symptom onset. To address these challenges, we focus on increasing trial capacity and helping people with ALS find trials they might be eligible for, ensuring that more patients can participate and benefit from potential treatments. By addressing these barriers, we are aiming to accelerate research and transform the experience of being diagnosed and living with ALS—to curing it.
Moe Alsumidaie is Chief Editor of The Clinical Trial Vanguard. Moe holds decades of experience in the clinical trials industry. Moe also serves as Head of Research at CliniBiz and Chief Data Scientist at Annex Clinical Corporation.
