2013 ALSRP Integration Panel Chair Vignette

Title: Targeting Drug Discovery

Speaker: Lucie Bruijn, PhD; The ALS Association

ALS is a very difficult and devastating disease. To date we only have one treatment that modestly affects the survival by perhaps 2 to 3 months, Rilutek®, one FDA-approved compound. So what we desperately need are treatments that significantly extend life span and improve quality of life. And I believe because it's such a complex disease we are going to have to take multiple approaches and significant investment to get to a point where we do have treatments and hopefully 1 day a cure for ALS.

Although there are many efforts funded by NIH in ALS research, the Department of Defense funding in the ALS program is very specifically directed towards the piece that most programs don't focus on. It's the translational step bringing good and exciting ideas from the laboratory but taking it down a very structured pathway to developing into a treatment. So this translational area which is risky, challenging-is something we absolutely have to fund and the DoD Program does exactly that.

The integration panel that really works to oversee that the vision of the DoD ALS program is honored includes representation from the NIH, from the Veterans' Affairs and other funding agencies so that whatever we fund we know is being funded uniquely through the program and is complementary to efforts more broadly.

What's very exciting, there are actually two components to the program; one that has later stage studies that are really bringing things much closer to the clinic, and one part of it is some very innovative early discoveries. So from the things that have already come quite far along the development pipeline, we already have one compound, Apocynin, which is looking very promising and is in the process of getting all the required data for FDA approval to bring it into clinical trials. So that really came, would not have happened without the funding from the Department of Defense in this program.

The other is a very interesting finding where atrophic factor seems to be highly expressed and there are ways to by use of a small molecule excessively produce this particular factor. Atrophic factors are very important to help nurture the motor neurons affected in the disease. And we think that these findings through funding through the DoD is going to help move it into potentially clinical trial testing as well. So we really have two compounds that are quite far along the drug development process and will soon be taken to the next step.

Another exciting initiative which was funded by the Department of Defense was to screen for interesting molecules in a very novel laboratory test, really trying to model ALS in a dish in a very clever way, and the funding enabled this investigator to develop that, to actually start bringing novel molecules to the next level. So it's incredibly successful and it certainly is I think what was really needed bringing many, many different ideas along a path that will bring it ultimately into clinical testing.

The Department of Defense ALS program is really a congressionally mandated program and every year ALS patients, their families, and their community come out to Washington, DC and go to the Hill to speak to their-their senators and their congressmen to really advocate for this program. And I think it has given us opportunities to fund in areas that before we haven't had dedicated funding. And I think the ALS DoD Program is-is so instrumental in bringing new ideas from the bench to the bedside. And so we're very grateful to the support.