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Icebreaker

My readers know that I have serious differences with the ALS Association (ALSA). However, my promise to deliver the truth (though bathed in hope in the delivery) cuts both ways. When something good happens, no matter who is behind it, I must give kudos to the deserving.

The 2014 social media phenomenon known as the Ice Bucket Challenge marked a seminal moment in the history of public awareness of ALS and in funding for research. Since then, PALS have been demanding that ALSA actually use that money rather than sitting on it. It now appears that ALSA is finally indeed mobilizing a little of that money (about $3M or 2.5%) on two wise and popular targets. This is good news, although there is a slight catch…

ALSA is helping fund a Phase 3 of the Cytokinetics drug tirasemtiv and a Phase 2B of Neuraltus’ drug NP001. Tirasemtiv is a muscular activator, meaning it causes the muscles to react more strongly than normal to a neural input. Tirasemtiv does nothing to halt the death of the motor neurons, but it can let PALS have more independence for longer than without it. NP001 is a highly purified and pH-balanced form of sodium chlorite that reverts the chronic inflammatory attack on the neurons back to a pro-growth state. Some of you might remember our dear departed friends Rob Tison and Ben Harris who experienced remarkable results during the Phase 2A. Now we know why: Based on inflammatory biomarkers discovered in post-hoc analysis, Neuraltus believes it has found a responder subgroup and is restricting the Phase 2B to those patients. I expect very good news from the 2B.

[UPDATE (07-13-2015) From my friend Jenica Lancy at ALSA GoldenWest: Today, The ALS Association announced its support of 58 new research grants totaling $11,621,638 to find treatments and a cure for ALS. The research awards announced today include investigator-initiated grants, drug development contracts, Milton Safenowitz Postdoctoral Fellowships and support of the NEALS/TREAT ALS™ Clinical Trials Network. You can see a full list of the grants here.]

Now for the catch: What ALSA is really doing is funding operations at one of the clinics which promote and direct funding toward ALSA. Both trials will be conducted by that clinic (the excellent Forbes-Norris ALS Clinic in San Francisco).

However, the fact remains that ALSA is supporting two very promising clinical trials. Some of us might wish they would do more, sooner, but they are moving in the right direction. I believe the proper response should be “Thanks! Keep it up!”. Let’s all applaud ALSA and encourage further progress along this path.

4 thoughts on “Icebreaker”

  1. only 2.5%??So where does all the other money go?

    why bother donating?

    Another interesting trial to watch…
    CNS drugmaker teams with CDMO on ALS mAb candidate

    By Fiona Barry +, 15-Jul-2015

    CDMO (contract development and manufacturing organisation) Goodwin Biotechnology is working with CNS biotech Q Therapeutics to produce mAbs for a clinical trial to treat motor neuron disease.

    http://www.biopharma-reporter.com/Downstream-Processing/CNS-drugmaker-teams-with-CDMO-on-ALS-mAb-candidate

    Our Solution: Q-Cells®

    Q-Cells® are the first and only human glial-restricted progenitor (GRP) cell therapeutic

    http://www.qthera.com/live/Technology/Our-Solution.aspx

    1. Indeed, Q Cells is an interesting approach to treatment of ALS. Similar to the Brainstorm product and aimed at reducing the inflammatory attack on the neurons (my pick for being the prime force behind progression).

  2. UPDATE (07-13-2015) From my friend Jenica Lancy at ALSA GoldenWest: Today, The ALS Association announced its support of 58 new research grants totaling $11,621,638 to find treatments and a cure for ALS. The research awards announced today include investigator-initiated grants, drug development contracts, Milton Safenowitz Postdoctoral Fellowships and support of the NEALS/TREAT ALS™ Clinical Trials Network. You can see a full list of the grants here.

    1. very good but patients still need faster access now….all of them…please read.

      Sick kids, desperate parents, and the battle for experimental drugs The complex world of compassionate use drugs and who gets access to them.

      When it comes to compassionate use, educating parents and patients hasn’t been the norm, and it’s been the industry’s biggest mistake. Patients with no time to lose have faced companies offering little support navigating the compassionate-use process — often because the companies themselves don’t know how to handle it. They “get surprised or even nervous” when press releases are posted with clinical trial results and patient requests start pouring in, says Henri Termeer, the former CEO of Cambridge-based Genzyme Corp. During his tenure at Genzyme, Termeer oversaw multiple compassionate-use requests for the company’s rare-disease drugs. “It doesn’t go away because you wish it to go away,” he says. “You have to be part of the solution.”

      Compassionate use is developing into “a major issue for the industry to grapple with,” adds Garabedian, who left Sarepta in April. “With the therapeutics that we see emerging, many of them from the Boston-based companies, you will be able to see signals very early on in a patient or two, and then the entire community is going to say, ‘There is nothing else out there, we want this drug.’ ”
      Indeed, compassionate-use requests to the FDA are exploding.

      There’s growing unrest with the realization that when a company denies a request, patient voices are getting louder. The power of the Internet and especially social media has allowed patients and their families to take their plea to the masses and, in some cases, see their story go viral.

      https://www.bostonglobe.com/magazine/2015/07/15/sick-kids-desperate-parents-and-battle-for-experimental-drugs/Hle3CTwriFfTXoOvQ7TbZP/story.html

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