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Welcome to Connecting ALS. On today's show we discuss expanding access to clinical trials.
Connecting ALS is a weekly podcast produced by The ALS Association in partnership with CitizenRacecar. We aim to discuss research and technology developments, highlight advocacy efforts, and share the personal stories woven through the community.
This transcript was exported on Jan 20, 2023 - view latest version here.
Dr. Merit Cudkowicz:
Right now, I'll just say it's maybe one person per month per site that is enrolled in a trial, and that is way too slow. It should be by 10.
Jeremy Holden:
Hello, everyone, and welcome to Connecting ALS. I am your host, Jeremy Holden. In order to make ALS a livable disease and ultimately find cures, we need to increase the number of clinical trials and make it easier for more people to participate in those trials. Increases in clinical trial capacity will allow for potential new therapies to be tested faster and will improve trial accessibility for people with ALS currently in underserved areas. In order to meet this urgent need for more shots on goal, as they say, the ALS Association has established a Clinical Trial Capacity Grant Program. These Clinical Trial Capacity Awards will support efforts to increase the number and diversity of people living with ALS who have the opportunity to participate in clinical trials and efforts to improve the speed and efficiency of clinical trial conduct at both established and emerging clinical trial sites across the United States.
Many people living with ALS want to participate in clinical trials, but are currently either unaware of opportunities or unable to engage in them due to lack of accessibility to clinical trial sites within their local areas. These awards will help eliminate barriers, especially those impacting currently underserved populations to trial participations. Similarly, many ALS clinics and drug development companies wish to pursue clinical development in ALS. These awards should help establish infrastructure, allowing them to conduct faster and more efficient trials in ALS. To get a deeper understanding of the challenges we need to overcome to meet this urgent need, I recently talked to Dr. Merit Cudkowicz, Director of the Sean M. Healey & AMG Center for ALS, Chief of Neurology at Mass General, and Director and the Julieanne Dorn Professor of Neurology at Harvard Medical School. Dr. Cudkowicz, thanks so much for being with us this week on Connecting ALS.
Dr. Merit Cudkowicz:
I'm happy to be here. Thank you.
Jeremy Holden:
Yeah. We're excited about this topic and it's something we're going to be talking about. Throughout the year, I know you've worked clearly closely with Dr. Kuldip Dave and the team over the ALS Association on the RFA for clinical trial expansion. And as you were going through that, what did you see? What were some of the major impediments to increasing the number of clinical trials in the ALS space?
Dr. Merit Cudkowicz:
First, I'm so happy that the ALS Association is supporting this initiative to improve access to trial. So needed. There's a couple impediments. I'd say one is actually physician time and availability. So there's a shortage in general of neurologists and also ALS specialists. And they want to see patients within two weeks at the minimum, and there's a lot of care issues. They don't have the time for the clinical trials. So part of it is the need to train more trialists to free up their time and also to rethink how you do it. Maybe work more with nurse practitioners and other people that can be available every day to enroll people in trials.
Jeremy Holden:
You mentioned the issue of enrolling people in trials. So what can be done there in terms of expanding the pool of people who are able to participate in clinical trials?
Dr. Merit Cudkowicz:
I think we need to make sure that people with ALS know about the trials. That's step one is knowledge, so that they know where they are, and that there are centers near them that they can go to for access to trials. We also have to simplify the trials, so that there's this concept now in the trial literature about decentralized trials where you're really bringing the trial more to the person. So can you do things remotely with tele trials? Can you do things in the home and make the burden less? Because people want to be in trials. I like to use the example of pediatric oncology where every child with cancer is enrolled in a trial. We should have the same in ALS, and everybody with ALS has an option for a trial.
Jeremy Holden:
How much of that comes to the issue of time to diagnosis and really getting a diagnosis quicker in the process earlier in progression and making people more eligible in terms of how far along they are in disease progression?
Dr. Merit Cudkowicz:
That is such an important point. I'd say one is I think we need to have trials for everybody, for early in the illness, later in the illness, even pre-illness like prevention. I think they're hampered by a long delay in diagnosis. So the average person with ALS sees three physicians before they get to a neurologist and before they get to a diagnosis, and that can take up to a year. And so that's a year of time when they're not getting intervention. And we really think in all neurodegenerative diseases, ALS, Alzheimer's, Parkinson's, that earlier is going to be more likely to have a larger effect. So we also need to change that paradigm of getting people to the right doctor faster. I know there's a lot of initiatives that ALS Association and other groups are trying to fund to accelerate diagnosis.
Jeremy Holden:
You talked about bringing clinical trials to patients. To the participants in a clinical trial, it's not feasible really to have a clinical trial site on every block in the country. But while we're working to expand the number of sites that are conducting clinical trials, what have we learned in the past couple years during the pandemic when it was very difficult for people to maybe come together to go see their doctor? To participate in clinical trials in a clinic, what are some of the learnings that we can take going forward to engage in more remote type trials?
Dr. Merit Cudkowicz:
We learned a ton. It accelerated everything people were talking about. We learned that we can do consent. We can get really informed consent virtually. And we used to think we need everybody in-person, but you really can do it over a screen and with a lot of time. It actually can be better. They say you're less rushed. You can do assessments in a home. We had to pivot during COVID to do breathing assessments, which are important for care as well as trials in the home because we weren't actually allowed to do them in the hospital. They were thought to be a risk for spreading COVID. So we adapted other machines and we've been able to show that it's pretty similar. A person doing it themselves in a home with their spouse over video coaching is just as good as at the hospital. Other ways to assess the illness like using digital technology. Now, what we have to prove is that those are as good as the gold standards of what we're doing in the clinic, and I think that's all happening now.
Jeremy Holden:
You talk about the gold standards of what we do in the clinic. You've been doing this for a number of years. What have you seen in your time in the clinical trial space in terms of it growing, the ability to participate, the number of trials? What have you seen, and what gives you hope that we are in fact moving in the right direction in terms of growing this even more?
Dr. Merit Cudkowicz:
The biggest change is just the number of trials. It used to be that maybe we had one trial. And now, we have a spreadsheet that we have to bring to the clinic. We have about 14 different trials, and those are drug trials, and then another 10 on biomarkers. So that's exciting. But it can also be a little overwhelming to know which one might be for a particular person, the best one for them. But that's a good problem to have. I've also seen more people going into the ALS field, which is also exciting. I'll just say in the '90s when I was going into this field, I was actually told there was no career in it. Now, it's a great career and lots of young medical students want to go into ALS. And there's so many companies working in this space that are looking for ALS expertise. So it's changed completely in a very passive way.
Jeremy Holden:
Yeah. That's exciting to hear and it's something that we've heard from a lot of folks that have come by and stopped by the Connecting ALS studio, as we say. A big conversation that has been happening in a lot of spaces in recent years has been around diversity, equity, and inclusion. How is that impacting the efforts to expand clinical trials? What can be done to make clinical trials more inclusive?
Dr. Merit Cudkowicz:
I start by saying it's a big problem with ALS trials, and then really all neurology trials. There's efforts being led by a couple foundations, including Tambourine and the ALS Association's working with them to try to understand what the barriers are, and then pilot different initiatives. And one of the barriers is that trials, again, enroll people usually Monday through Friday. And so unless you have a very nice boss who lets you take time off, that doesn't work for most people. So there's been some initiatives again about this bringing the trials to the home or having late evening or weekend options, covering the cost for people for lost wages, for leaving work to be in a trial. So those are some of the things being piloted. There's also, again, back to the education part of the knowledge, getting the word out about what's there, and that's not consistently done throughout the country.
Jeremy Holden:
We're going to have the great fortune to talk to some of the recipients of the grants that we're talking about today in the coming weeks and months. But before I let you go back to the critical work that you're doing, I want to ask, what will success look like, say, 5 years from now, 10 years from now? What does success look like as we look at expanding access to clinical trials?
Dr. Merit Cudkowicz:
I think success is going to look like the trials and ALS are enrolling much faster than they are right now. I'd like to see a triple or quadruple in speed. Right now, I'll just say it's maybe one person per month per site that is enrolled in a trial, and that is way too slow. It should be by 10. So we want to see the enrollment rate go up. I think we'd want to hear from people with ALS that it's not hard to enroll in a trial, that they have the information from their physician and they have the opportunity. Because right now, we do hear from people, "Oh, my doctor never mentioned that. Oh, there's no trial near me."
Jeremy Holden:
Yeah.
Dr. Merit Cudkowicz:
I'd like to not hear that anymore. Those would be two good metrics.
Jeremy Holden:
Yeah. A lot of tools are in development to make it easier for folks to find clinical trials, something we'll be talking about down the road. Dr. Cudkowicz, thanks so much for your time today.
Dr. Merit Cudkowicz:
Thank you, Jeremy.
Jeremy Holden:
I want to thank my guest this week, Dr. Merit Cudkowicz. Now, in the coming months, we are going to be exploring some of the exciting projects funded through the ALS Association's Trial Capacity Awards. So make sure you subscribe to Connecting ALS, so you don't miss an episode talking about some of the work being done around the world to make it easier for more people to participate in clinical trials. If you like this episode, share it with a friend. And while you're at it, please rate and review Connecting ALS wherever you listen to podcasts. It is a great way for us to connect with more listeners. Our production partner for this series is CitizenRacecar, post-production by Alex Brouwer, production management by Gabriela Montequin, supervised by David Hoffman. That's going to do it for this week. Thanks for tuning in. We'll connect with you again soon.
ConnectingALS_011923_Ready2_YouTube (Completed 01/20/23)
Transcript by Rev.com
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