Beyond the Diagnosis: Clinical Trial Advocacy & ALS

Hear the inspiring story of Larry Falivena, an ALS patient advocate, who shares his journey through his ALS diagnosis and navigating clinical trials. Larry's diagnosis in 2017 marked a profound shift in his life, prompting him to cherish each moment and advocate for ALS awareness. Despite the grim prognosis, Larry's engagement in clinical trials offers hope for ALS patients. Hear the emotional and logistical hurdles often faced by ALS patients in accessing and engaging with clinical trials and how the industry can improve the patient experience. 

As we reflect on his journey, Larry's insights inspire us to collectively improve patient outcomes by overcoming barriers to trial participation. His story highlights the critical role of patient involvement in research and the need for accessible trials.

Transcript

Jimmy Bechtel: 0:00

Welcome, and thank you for joining the Society for Clinical Research Sites for SCRS Talks. I'm Jimmy Bechtel, the Vice President of Site Engagement with the Society. SCRS Talks allows our partners and those that we work closely with to take a few minutes to address issues of industry concern, share exciting achievements, and to learn more about our community today, we're lucky to have Larry Falivena with us. Larry was a recent patient keynote speaker at our 2023 global site solution summit this past October down in Hollywood, Florida. Larry is currently battling ALS and is here with us to share a little bit more about his journey with his condition as well as his experience in clinical research. Larry, it's great to have you with us. Thanks for taking the time out of your day to again share your story with our audience, but I'd like to start things off and turn it over to you to give us a little bit of an introduction of yourself. Share a little bit about your journey and some of your background.

Larry Falivena: 1:01

Yeah, sure. Thanks, Jimmy. I really appreciated the opportunity to speak at the global site summit. I certainly appreciate what all of you do in the organization. These drug trials, especially for orphan diseases are super important. And in fact, for someone with ALS, like myself, it might be the only opportunity to have a treatment for a disease that up until this point is 100 percent fatal. So, it was really exciting for me to share my story. I was diagnosed in August of 2017 with a genetic form of ALS, even though there hasn't been any history in my family. I'm currently 55, so I was late 40s then thinking everything was going great. I have two boys, great wife, and then suddenly you get this the diagnosis that says you have ALS. There really is no treatment and most people only live two to five years. So it's a wake up call and something that I've tried to share with other people is, you never know what life is going to throw at you. So make sure you enjoy every day. Appreciate every day. Do the best you can every day because you never know what the next day will bring. Currently living in North Carolina. Like I said, I have two teenage boys one in college, one in high school, and I'm no longer working. I spent 20 years or so in the software sales industry. But when I got the diagnosis, I realized that I wanted to do as much as I could with the time that I had left. Thankfully I've had much more time than I expected. And I think a big reason of that is being in the trial and receiving what was an experimental drug has now been approved by the FDA, so really happy to be here and continue to tell my story and let people know about this disease that, again, at this point is still 100 percent fatal, but so much has happened in the last few years, if you think about it, if you remember the ice bucket challenge, and if you ever dumped cold water on your head, you. Which I did before I was diagnosed.

Jimmy Bechtel: 3:14

Absolutely.

Larry Falivena: 3:16

The money that was raised, it was$115 million and that drove so much research, so many new attempts to treat this disease that now we, we've had three treatments in the last six years that are not cures. But they're buying people more time and it's more time for them to experience life and enjoy their loved ones and maybe enough time that we actually do get to a cure. So a lot of really exciting things have happened in the last few years.

Jimmy Bechtel: 3:49

Well, thanks, Larry. And that context and that background is super valuable. And it is cool to continue to see the positive effects of that ice bucket challenge. It is really a case study for when we, as a community of individuals come together. We can make a lot of change with something that, as you said, is is really unfortunately not a positive outcome for really anyone. But Larry, would you mind kind of take us through some of what you have experienced in the last few years of kind of leading up to your engagement in the clinical research enterprise right for right up to the point where you got into your first clinical trial and and take us through your state of mind and where you were headed with your prognosis and some of the things that you might have been experiencing or doing as a result of of your diagnosis.

Larry Falivena: 4:47

Yeah. I mean, again, it was 2017. So back then there was really only one treatment that was developed back in the nineties that was maybe slightly effective. So it was a, daunting diagnosis. I remember the neurologist, almost breaking down giving me that diagnosis. And so, you're faced with this sort of harsh reality and you don't know how much time you have left. And so again, I wanted to spend that time doing as much as I can to make memories with my family, but also to let people know about this disease. It's not incurable. It's maybe just underfunded, right? So the first few years that's what I was focused on. We actually my entire family spent the summer in an RV visiting all 30 major league baseball parks as a campaign to raise awareness and raise money for ALS and research. So that was the first couple of years, but at the same time, as I mentioned in the beginning, really getting into a drug trial was kind of really the only hope, right? And so for anyone who's diagnosed with ALS, that becomes a key factor. How do I get into a study? And I was the same way. I was blessed in, in one way because mine was genetic. It was easy to identify or easier to identify what the cause of the disease was. That's 10% of the ALS population has a gene that we can tie to the other 90%, we, we don't know. The point of that was that I was able to target studies that we're looking at that specific gene. I'm also blessed because my wife happens to be in the industry, and she knew how to navigate the process of finding a study and getting engaged. And actually, initially I screen failed but got in the second time around. So was in the study for Tofersen for six months and now I'm still in the Open Label extension. So, just incredibly thankful that I found a study that was specifically directed at my type of ALS and unlike unfortunately, most of the treatments that fail, this one seems to have worked.

Jimmy Bechtel: 7:16

That's incredible, Larry and the experience that I want to talk to you about next is, I guess, finding and getting into that clinical trial. And I know, like you had mentioned, you were in a little bit of a advantageous, we'll call it, or unique situation in that your wife was a member of the industry, right? She knew about clinical research and about clinical trials. So can you maybe share a little bit about that process of kind of finding and and maybe initiating kind of getting into that clinical trial and what that looked like? But also, as part of that, I'd love to hear your perspective of I would, we'll call it the lay person's perspective had you not had someone who could talk the talk and walk the walk on your behalf or with you a little bit as well.

Larry Falivena: 8:01

Sure. Yeah. I mean, it's a daunting process, right? You try to find a trial and you wind up going to that.Gov website. Which contains a lot of information, but maybe isn't the easiest thing to navigate. And I, I think for a lot of people they rely on their doctor to point out to them a drug trial that might be helpful. Interestingly enough, There have been several tools created by non profits that support ALS research and advocacy. They've created tools to make that process easier, because it is a daunting process and it's not the easiest thing to navigate. So there have actually been third party tools to try and help make that happen. Then you get into trying to navigate the informed consent and particularly with this disease. If you are not near a major metropolitan area with an ALS clinic or a research hospital travel can certainly be a barrier. At the very least, very difficult. Again, I'm blessed that, I'm still pretty mobile. So, I was able to deal with making the trip from Raleigh, North Carolina to Johns Hopkins in Baltimore. But there's a lot of obstacles.

Jimmy Bechtel: 9:23

Thanks, Larry. And it does paint a challenging picture for anyone who wasn't in the same situation or similar situation to you where they had someone to help them navigate the industry, they were close to a center to be able to participate in those clinical trials that were being conducted, et cetera. So again It does paint a grim picture for someone who might not even know clinical research exists and they're subject to standard of care or they are in a rural area or, far beyond greater than three hours outside of a major metropolitan area that might be, might have a center conducting these clinical trials.

Larry Falivena: 10:04

Right. And again, talking about being in a rural area, you might be dealing with a neurologist who hasn't read anything about ALS since he was in medical school and it's no offense to the neurologist, but it's a rare disease, right? So it's one of the things I think we're trying to fix is to not let your zip code determine your outcome of this disease. And I know for your entire industry that's an issue, right? How do we diversify trials? How do we reach people where travel is an obstacle. So I know it's something that the entire industry is trying to solve.

Jimmy Bechtel: 10:48

It is absolutely and that's where I was going to head next to Larry was, I was going to ask you to share a little bit about your clinical trial experience. Talk about the maybe share some of the good and the bad and the ugly. And then also if you had that magic wand, if you could give advice to our partners in the industry that are listening on ways to make this better for you as a patient, what would those ways be?

Larry Falivena: 11:16

Sure, honestly, and I know this is outside the realm of your particular industry, but it actually goes all the way back to the design of the trial itself. And when the trial is being designed and I realize we have to stick to the science and, we have to get the information that's needed, but incorporating needs of the patient into the trial design itself. Certainly if we can do decentralized trials that's a huge thing. Again, particularly with people with A.L.S. where traveling is an obstacle. The team obviously that took care of me, the coordinators, the nurses, et cetera, are phenomenal. If we think about our medical industry in general, there's a, a shortage of staff, right? So you've got people who are juggling a lot of things and doing the best they can, but there's only so much capacity. And that, in my mind, limits the number of trials that can happen. And again, I know that's something that your industry is working on. The entire medical field is working on. Travel, I've already talked about I mentioned I have two kids. Well, when I started this Obviously they were younger and you have to find someone to watch them. It's time away from them. For anyone who's traveled, it's not the most pleasant experience, even under the best of circumstances. But I, again, am incredibly grateful to have found a trial that was not only specific to my ALS, but also seems to have worked. So, it's a trade off, right? Again, for someone with this disease. You don't have a lot of choices, and so that may mean you have to jump through a lot of hoops and in my particular case, this drug is administered through a lumbar puncture, so it wasn't even like, you could just send me a bottle of pills and I would take it at home, so, there's a lot of factors involved. Hopefully, that answered your question.

Jimmy Bechtel: 13:23

Yeah, it does. And what I got from that, Larry, what I take away, what the message that I would give back to our industry partners, succinctly that are listening is that we need to enable our. Potential patient populations here. Patients like you that are in need of these life prolonging and hopefully soon life saving medications to enable them to be able to be seen or treated or brought into these clinical trials, regardless of their geographic limitations. The other aspect that I heard you say really centers around, as well, awareness. We need to raise awareness that these opportunities are potentially available to patients that are subject to these diseases. And there's a lot of different ways that we can potentially do that. Probably that's not your job to solve, right? You're the patient. That's our job in the industry to solve. But if I was to kind of put a pin in two different takeaways from this, those would be the ones that I would like our listeners to hone in on for sure.

Larry Falivena: 14:30

Yeah. That's great.

Jimmy Bechtel: 14:33

I can't tell you how much I appreciate having this conversation with you, learning more about you and learning more about what you're going through and hearing from the mouth of the patient, the voice of the patient, some things that we can do to make this process easier. Again, I thank you for sharing your story and sharing your insights and your thoughts with us today.

Larry Falivena: 14:54

Well, again, thank you for the opportunity and thank you for the work that you all do. These things wouldn't happen if it wasn't for the folks in your industry and running these trials. So thank you.

Jimmy Bechtel: 15:07

For everyone listening, make sure that you register for upcoming summits being held throughout the year by visiting the summit page on our website, myscrs.org. While you're on our website, be sure to check out other SCRS publications made for the community in the publication section of that same website. We appreciate your participation in today's program and look forward to having you join us. For more great content coming out very soon. Thanks again for listening.