Phil Green, a person living with ALS, attended the 2019 ALS MND International Symposium in Perth, Australia as part of their Patient Fellows Program. After reflecting on his time at the Symposium, he answered a few questions we had about his experience.
What are your three biggest takeaways from the International Symposium on ALS/MND?
At the highest level, I was impressed with the number of incredibly brilliant people that had come together to share insights and discuss their efforts to advance the science of ALS. From the outside looking in, it can be very easy to say that we haven't had much progress in the 146 years since Jean-Martin Charcot characterized the disease, but the reality is quite different. These researchers that congregate each year at the International Symposium are making significant progress in better understanding the disease and defining the various mechanisms that are at work. With so much brainpower working on the fundamental science behind the disease, I came away with a confirmation that this is one complex disease (or spectrum of diseases) and confidence that we have the world's best and brightest minds working hard together to solve the mysteries of ALS.
The next takeaway from the week was that we are making significant progress using antisense oligonucleotide (ASO) therapy to treat specific gene mutations. This technology is hyper-targeting upstream targets and for the first time, may actually fix the issue at the source. This still needs to be proven through current and future clinical trials, but it appears to be a significant advancement that may even translate to sporadic ALS sometime in the future. I had the opportunity to spend time with Dr. Richard Smith and Dr. Don Cleveland to better understand the effort that went into developing this treatment.
The most significant takeaway for me was learning about where we are in regards to ALS biomarker research. I had the opportunity to sit with Dr. Martin Turner from Oxford University at the Patient Fellows Dinner. We had the most amazing discussion about his work on ALS biomarkers, and specifically about neurofilament light chain protein (Nfl). Dr. Turner was emphatic that we have the data that supports the use of Nfl as an indicator of efficacy for ALS therapies and interventions. His subsequent presentation the following day at the Symposium provided more visibility to this and other possible ALS biomarker candidates, but Nfl and Nfh (neurofilament heavy) levels seem to be clear evidence that motor neuron death is taking place and the levels present to indicate the rate of progression. Upon learning of this research, I began to strategize on how we can get more data to build a case that can be presented to the FDA to validate Nfl and Nfh to use an endpoint for ALS clinical trials.
What was most impactful for you?
The most impactful thing for me was connecting with the other people living with ALS that were attending from across the globe. It was also impactful for me to connect with the top global researchers and learning about their dedication to this disease, and how long they have invested in their respective areas of focus. I also wanted to make sure that they understood that the patient community appreciates their effort, that their work is important, and hopefully, give them the inspiration to work more urgently and with purpose because of the personal connection we made with each other.
How did you feel when leaving the conference?
I felt an amazing sense of community with all of the people that are in this fight. Participating in the first annual Global Walk to D'Feet MND reinforced this sense of "we are in this together" that I experienced the entire week in Perth. Spending two days with the International Alliance, I got to witness how the sharing of ideas between countries made their ability to serve their respective communities more effectively. I came away with a tremendous amount of hope that we do have the best and brightest working on all aspects of this disease, and we are making significant advances in this fight to end ALS. I came away inspired to help advance biomarker development programs, find more funding for these types of programs, and promote even more collaboration between institutions, organizations, and agencies.
To watch Phil's video testimony that he recorded on the last day of the Symposium please click here.