A DIY clinical study? Desperate patients try a therapeutic ‘home brew’

Posted under Blog, Clinical Trials, Companies, Diagnostics, Drug Safety, Funding, Lou Gehrig's disease, Medical Devices, Medical Supply, Neuraltus Pharmaceuticals, NP001, Pharmaceuticals, Startups, Universities, Videos by john

It's not unusual for desperate patients to bombard a biotech company with requests for one of their experimental medications. But in the case of Neuraltus Pharmaceuticals, a small group of ALS patients have gone to the extreme: Making their own "home brew" versions to see if it can help keep them alive.

"We simply don't have time to wait for the results of [clinical trials]. Our life spans are much shorter than the [Food and Drug Administration] approval process," ALS patient Ben Harris tells The Wall Street Journal. 

In this case the object of their desperation is NP001, an experimental drug now in a Phase II study. Investigators theorize that by damping down inflammation in the central nervous system they can slow down the progression of Lou Gehrig's disease. And faced with years of study in the clinic, a group of individuals has been tracking down patents and any other information they can get to design their own version for a home study. They are using a concoction of sodium chlorite, used in municipal water treatment facilities, which they believe is the active ingredient.

Emory University's Jonathan D. Glass, an investigator in the study, says what they are doing is reckless and dangerous. "I feel their pain that they really want it to happen faster, but I don't think you can do it without the medical establishment," he tells the Journal.

- here's the article from The Wall Street Journal

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Biogen Idec bets it can succeed on a risky Phase III ALS gamble

Posted under ALS, Biogen Idec, Blog, Companies, dexpramipexole, Diagnostics, Funding, George Scangos, Lou Gehrig's disease, Medical Devices, Medical Supply, Pharmaceuticals, Pipeline, Startups, Universities, Videos by john

Bloomberg biotech scribe Meg Tirrell has been plumbing Biogen Idec's ($BIIB) late-stage drug for ALS and finds some good reasons to believe that they are on to something big--perhaps even blockbuster-sized.

First, dexpramipexole had to stand up to George Scangos' review process when he took the helm a couple of years ago. Anxious to trim away any low percentage shots at an approval, Scangos was won over by the data and market profile. The drug--in-licensed from Knopp Biosciences--did well in Phase II, demonstrating a significant slowing in disease progression after a brief 12 weeks of treatment at the highest dose of 300 mg daily. A later stage of the trial tracked a significant mortality benefit comparing the high and the low dose. And if the data hold up in the late-stage trial, that could translate into a sizable market of a billion dollars-plus, considering the woeful absence of any effective therapy for patients.

But Scangos had been skeptical for a good reason. A whole slate of prospective treatments has been shot down in the clinical trial process, underscoring how little is known about Lou Gehrig's disease and what needs to be done to slow or stop it. But it's late-stage study--testing 150 mg twice daily--enrolled patients in just a few months. Now late-stage data is due out later this year. A win here would help further burnish Scangos' reputation for turning around the one-time biotech laggard. 

"This is far from a long shot," CEO Scangos told investors at the annual J.P. Morgan confab in San Francisco in January. "We are genuinely hopeful that we will be able to provide meaningful therapy for the thousands, tens of thousands, of ALS patients who right now have very little to help them."

- here's the story from Bloomberg

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Cytokinetics offers upbeat data from mid-stage trial of ALS drug

Posted under ALS, Blog, CK-2017357, Companies, cytokinetics, Diagnostics, Funding, Lou Gehrig's disease, Medical Devices, Medical Supply, Pharmaceuticals, Startups, Universities, Videos by Ryan McBride

After failing to impress investors with previous data on its ALS drug, Cytokinetics' ($CYTK) stock price got a 4% bump this morning after the South San Francisco developer revealed data from a mid-stage trial involving patients with the incurable neurodegenerative disease.

The company said that a part of the Phase II trial for the drug, dubbed CK-2017357, succeeded in "defining the tolerability and pharmacokinetic profile" during two weeks of therapy. The trial, involving 24 patients with amyotrophic lateral sclerosis (ALS), commonly called Lou Gehrig's disease, showed the drug was well-tolerated at daily doses ranging from 125 mg and 375 mg. It reported that 83% of patients on various doses of the drug had at least one adverse event compared with 67% in the placebo group, with dizziness being the most common side effect among patients in the study. Symptoms of the disease decreased in three patients in the trial, Cytokinetics CEO Robert Blum told Bloomberg.

"Even after two weeks, in this study we saw trends toward improvement," Blum told the news service.

Cytokinetics said on Tuesday that it was beginning its third Phase II study of the drug, which is intended to increase sensitivity to calcium in order to activate fast skeletal muscle complex. When the disease robs patients of skeletal muscle strength needed to breath, they die of respiratory failure. The third mid-stage study will test the drug in patients on the ALS drug riluzole, after the previous Phase II studied the experimental treatment in patients who were not taking that drug.

- here's the release
- see Bloomberg's article
- and the report from RTTNews

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