Matt Bellina - A Consumer's Perspective: An Increased Sense of Urgency

Posted May 12, 2017

FY2017 ALSRP Consumer Vignette
Interview: Matt Bellina, ALS Therapy Development Institute

Matt Bellina: I was a Prowler Pilot. I trained in Pensacola and then Meridian. And then up to Whidbey Island. After that I did some anti-terror operations in East Africa and then administrative work when I got sick.

I had no genetic disposition, no family history of ALS. Military Veterans are approximately twice as likely to get ALS. Since 2001, ALS has killed more Veterans than the wars in Iraq and Afghanistan combined. It's costing the U.S. taxpayer about $450 million a year and we�re putting out $7.5 million in research. It's pretty unbalanced. The average ALS patient only lives about two to five years after diagnosis. So the current drug development pipeline of the FDA, it takes about 15 years to get a drug into patients. So you know obviously that needs to change.

It's a very difficult disease. It's very variable. I�m lucky in that you know I can still get up and walk around a bit, still eat, still talk, still somewhat feed myself. But every day is a new challenge. What's hard for me is, you know, I have a three and a five-year-old boy. And all they want to do is play and wrestle and, you know, it's hard because I feel like I�m letting them down every day.

Caitlin Bellina: There's nothing that can stop it, nothing that can reverse it. There's just one drug called Riluzole which can slow it; for like three to six month maybe it�ll extend your life span. And that's pretty frustrating considering, you know, it's called Lou Gehrig's disease because it was named after the great Yankee, Lou Gehrig, who died over 75 years ago. And in the 75-plus years there's one drug for a terminal illness. It's--it's a little mind-boggling.

Matt: Early on I contacted ALS-TDI. They you know were quick to get me involved. They nominated me to be on the CDMRP panel as a Peer Reviewer. That is really when it took off. I got involved. And we haven't looked back. The ALSRP is a very promising program because it's looking after therapeutic ideas for the aggressive development of a treatment, which, you know, in addition to saving my life, could ultimately save the taxpayer a lot of money. I was blown away by the scientists working on this; they�re trying to get us where we need to go.

Back in 2014, we reviewed an application for a compound called Copper ATSM initiated by Dr. Joseph Beckman. It's a copper that�and I didn�t even believe it when I saw it�but it stopped disease completely in the SOD1 mice. The award was granted and the data looks incredible.

Dr. Pierre Drapeau with his study on Pimozide is collecting final data now at this point from the clinical trial. Pimozide is a calcium channel inhibitor, so that drug in particular could be something that could slow down the disease. And that's in human trials right now. And that already being an FDA-approved drug, it means that we can be very close to putting that into patients in the clinics.

We have in our brains something called an efflux pump. Unfortunately the efflux pump binds very, very well to the one drug that is approved for ALS, and that's Riluzole. The longer you have ALS, the faster your body gets rid of Riluzole. So Dr. Pasinelli was able to look into the prospect of developing an efflux pump inhibitor that would actually increase the saturation and effectiveness of Riluzole.

Dr. Svendsen is looking at using GDNF to treat early upper neuron degeneration and hopefully we�ll see something being presented at the FDA pretty soon on that.

Given the limited amount of funding, it's actually pretty shocking that these accomplishments have occurred. Really, it just goes to show what we can achieve with the effort that we�ve put forward. This program is essential.

One of the other things I�ve been involved in is the Right to Try Movement, and that is basically kind of like a patient bill of rights, the rights of terminally ill people. What I find is that people with ALS are willing to accept much greater risk in trying treatments than--than the FDA historically allowed. And that should not be the case. The people making the decisions should be the people that are affected by it--the patients. So we�ve worked pretty hard on that.

Caitlin: When Matt gets excited about some of the therapies that are coming out or some of the research, if he's excited about it, I know he's researched it. So I�ll get excited about it. But I�m also--I�m a little less enthusiastic as he is. I like to call myself cautiously optimistic. You know it's been--it's been so long since anything has happened that I want to believe it but at the same time I don�t want to get my hopes up and then sit in his lap and cry for an hour again when it doesn�t work.

Matt: The developments we�ve seen have led people to believe that hey, we can--we can effectively treat this disease and with that has come that increased sense of urgency. I think now that people believe it can be done, they want it done now.

Caitlin: It's a scary thing. But we�ve taken it as an opportunity to really find out what matters to us and not get so caught up in the small stuff, and use it as an opportunity just to, you know, make our little part of the world a better place.

Matt: The ALSRP has really given me a lot of hope because I�ve learned that we know a lot more about what's going on than we did even last year. Every year I go, I�m amazed at how much the body of knowledge has grown. It's exponential, so we�re going to get there; I guarantee it.