Dr. Merit Cudkowicz, chief of Neurology at Massachusetts General Hospital, where the ALS Multidisciplinary Clinic in the Healey Center for ALS is associated with MDA, is dedicated to performing academic-led clinical trials in amyotrophic lateral sclerosis (ALS). Her internationally renowned research has led to advanced treatments for people with living with ALS.
What role has MDA played in advocacy and research that has directly impacted your line of work?
Dr. Cudkowicz: The first grant I received to work in ALS was from MDA. I immediately became part of this amazing research family where I met many other researchers and mentors. Since that time, I had the privilege to be part of the MDA medical review board for many years. This was the most positive and helpful review board I ever participated on. The goal was to fund the best research and to help researchers develop the best studies. I met colleagues and forged many new collaborations.
What is the one clinical experience that reminds you of why you chose to work with patients with neuromuscular disease?
Dr. Cudkowicz: I love all my patients and families. It is an honor for me to be part of their lives, to care for them, and to work together to understand ALS and to find effective treatments. At the conclusion of the first clinical trial I ever ran, when I called one of my patients who was in the study to share the study results, she asked me not to be sad about the results and most importantly not to give up until we find the cures. She thanked us for trying. I never forgot her words.
How have you been inspired by the patients and/or families you serve?
Dr. Cudkowicz: I am inspired by each of my patients and their families. ALS is an awful disease and it is hard on families. I am inspired by how people adapt and don’t let it define them, how they volunteer to be part of research, the way they care about their friends and families. I have met so many beautiful people who remind me each day of the power of family, friendship, and hope. I also have learned so much from my patients and families, and we have laughed and cried a lot together. I am both inspired and motivated to fight until we have figured this illness out and have truly effective treatments. I feel that we are on the cusp of that turning point in ALS. We wouldn’t be here without all the people with ALS who have been part of the research, nor without MDA and other foundations that support that research.
How has MDA directly impacted your investigative research project/s?
Dr. Cudkowicz: MDA started my career by supporting me as a new research fellow fresh out of neurology training. Since that time, MDA has supported many of my research projects. I met many colleagues and amazing mentors through MDA review and other meetings. In addition, several of the amazing researchers at the Healey Center for ALS at Mass General received their first training grants from the MDA. Our clinic has long been supported by the MDA — this helps us provide excellent multidisciplinary care to our patients and families.
How will collaboration get us closer to finding a breakthrough?
Dr. Cudkowicz: We are only going to solve these disorders through collaboration. We need to learn from every patient, every study. New ideas come from people working and talking together. I firmly believe in collaboration. We started the Northeast ALS Consortium in 1995 to build an ALS community that works together to find cures; this group brought scientists, clinicians, people and families with ALS, and all healthcare providers who care for people with ALS together to solve ALS. This is the model we also built at the Healey Center for ALS and the department of Neurology at Mass General to tackle all neurological disorders.
Why is the multidisciplinary approach that MDA applies to all 150+ Care Centers (with 48 specialized MDA ALS centers) important for ALS patient care?
Dr. Cudkowicz: ALS is a complex disease with many symptoms. We know we can improve the lives of people and families with ALS through multidisciplinary care. We could not offer this without MDA’s support of the Care Centers. It is crucial, and we are grateful for that support.
What is on the horizon for a cure for ALS and what will it take to get there?
Dr. Cudkowicz: The Healey Center for ALS at Mass General with the Northeast ALS Consortium is about to launch the first Platform Trial for ALS. This is an innovative approach to greatly accelerate therapy development. It is the right time for this in ALS — in fact it is imperative that we bring this to ALS. The science has exploded with many new targets and ideas for therapies. The pipeline is big and we can no longer test one drug at a time. We can learn from success in oncology with Platform Trials and bring this to ALS and other neuromuscular disorders. We are very excited about this; it will broaden access, accelerate therapy development, and cut time and costs. This will take collaboration between pharma, clinicians, foundations, and people with ALS. All of this exists in ALS and is ready to take off.