Project ALS and Columbia University Unite in $6.3M Push for ‘Meaningful’ Treatments

Project ALS and Columbia University Unite in $6.3M Push for ‘Meaningful’ Treatments

Project ALS and Columbia University are launching a new platform with a goal of discovering more useful treatments for amyotrophic lateral sclerosis (ALS) patients, and pushing the most promising candidates to clinical trials.

Core, as it is called (officially, the Project ALS Therapeutics Core at Columbia), is a three-year, $6.3 million initiative that hopes to promote “better trials” and to find “the first meaningful therapies for ALS,” the two partner institutions announced in a press release.

“It’s time for drugs that work,” said Valerie Estess, director of research at Project ALS.

“For twenty years, Project ALS researchers have built tools that allow us to screen potential drugs more efficiently. The Core is the first attempt to use all the validated tools under one roof, at Columbia,” she said. “This is our best shot to make a big difference right now.”  

The initiative is the first joining an academic institution and a nonprofit organization and will span the full process of ALS therapy development, from discovery to preclinical evaluation and clinical trials in patients. The seven-unit platform will test in-house therapeutic candidates and team up with pharmaceutical companies to test potential treatments. 

Most promising and core-validated candidates will be evaluated in clinical trials, with the help of the Eleanor and Lou Gehrig ALS Center, a clinic dedicated to motor neuron diseases at Columbia’s Department of Neurology.

The Core brings together world-leading researchers and physicians “to set a new standard for ALS therapy development,” the release said. It builds upon recent discoveries in ALS genetics and biology, and makes use of novel technologies, including stem cell techniques, to identify and evaluate potential therapies. It also integrates seven research groups: clinical research, lipidomics (the large-scale study of lipids, or fatty acids), gene therapy, antibody development, in vitro (lab dish) screening, in vivo (in a living body) evaluation, and in vivo electrophysiology.

It all “begins and ends with ALS patients,” the two institutions said, noting that patient blood samples will be used to screen for treatments, look for novel biomarkers, and perform genetic studies.

“For the first time, ALS patients can directly participate in research that will move us toward therapies that actually work,” said Neil Shneider, MD, PhD, and the center’s director. “The Core provides an immensely exciting opportunity to capitalize on decades of ALS advances and translate them into meaningful treatments now.”

Core scientific directors, all from Columbia University, are Serge Przedborski, MD, PhD; Hynek Wichterle, PhD; and Shneider.

6 comments

  1. Randy says:

    Meaningful? All these years what have you been doing? Another 10 years of waiting and the disease is over 100 years old ! Amazing how HIV came out in the 80’s and now there’s a cure. ALS has only 2 mediocre medicines.All the tremendous cures for cancer that have come out,everyday on TV the research companies are coming up with new ways to cure cancer,not 1 ALS commercial or advancements !

  2. Randy says:

    meaningful?
    amazing how there’s only 2 mediocre drugs for ALS when the disease has been around for well over 100 years.
    HIV came out in the 80’s and there’s a cure for that. On tv you see all the tremendous advancements with different cancer treatments. Not ONE commercial from a research company saying that they lead the way for a cure for ALS.

  3. Ronnie Cooley says:

    Ronnie c from Ms. all I ever hear is how much money was donated to a organization. Or at the end of the story. (study suggest) that’s all good. But when are the ones suffering from als going to get results. Doctors have known about this disease for a long time. And a lot of people have died because of it and are still dying. When are we going to get help? I hope to God there is no greed involved. God bless the ones who find a cure. PS. Don’t forget we are still suffering.

  4. Dave Reckonin says:

    “…and translate them into meaningful treatments now.”
    We hear this talk about ‘speeding things up’ quite a lot; but Phases 1/2/3 clinical trials happen at the same old pace. Nothing changes up a gear.
    I’d like to see this particular project put its money where its mouth is. Well, it’s other people’s money, of course, but you know what I mean.

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