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  • granicus007 granicus007 May 8, 2013 11:20 AM Flag

    ALS - Orphan drug status for NurOwn

    New Hope For People With Fatal Muscular Disease ALS?

    The Israeli biotechnology company BrainStorm announced successful results in its clinical trial for a drug against ALS (Amyotrophic Lateral Sclerosis) – a fatal neurodegenerative disease also known as Lou Gehrig’s disease.

    The stem cell therapy with the drug NurOwn did not show significant side effects and the treatment has so far proven to be safe, the company said. The U.S. Food and Drug Administration last year granted orphan drug designation to NurOwn.

    Patients in the trial are transplanted with stem cells derived from their own bone marrow and treated with the NurOwn stem cell technology.

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    The initial phase of the study is designed to establish the safety of NurOwn and will later be expanded to assess efficacy.

    However, the patients in the trial have already shown positive results. Among the six first patients, four have shown signs of improvement and the other two have experienced stabilization, meaning their medical condition stopped deteriorating.

    “Even though we are conducting a safety trial, the early clinical follow-up of the patients treated with the stem cells shows indications of beneficial clinical effects, such as an improvement in breathing and swallowing ability as well as in muscular power,” Dimitrios Karussis, head of Israel’s Hadassah Medical Center‘s Multiple Sclerosis unit, who is leading the clinical trial, said in a press release.

    “Patients treated with the Company’s NurOwn therapeutic cells are expected to enjoy a rapid recovery and much enhanced quality of life. BrainStorm‘s therapy has the potential to cure underlying pathology, rather than simply treat symptoms,” the company says.

    ALS tends to affect people over the age of

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    • there is another good news
      Other companies are developing drugs to treat A.L.S. Neuraltus Pharmaceuticals, a privately held company in Palo Alto, Calif., is preparing to enter the final stage of clinical trials for NP001.

      Neuraltus announced in late October that its Phase 2 trial, which involved 136 patients, had failed to show a statistically significant benefit compared with a placebo. But the company said 27 percent of patients receiving the high dose of NP001 had no progression of their disease for six months, two and a half times as many as in the placebo group.

      Cytokinetics, of South San Francisco, Calif., is in midstage testing of a compound, tirasemtiv, which might make muscles respond more forcefully to nerve signals.

      Frustrated ALS Patients Concoct Their Own Drug Wall Street Journal

      Mr. Valor says he knew he wouldn't qualify for the NP001 trial because of his disabilities. Some trials require patients to have had symptoms for 24 months or less and not need mechanical breathing assistance in the hopes that signs of efficacy in a drug will be easier to determine in less-progressed patients.

      Mr. Valor first read about NP001 in a news release. He tracked down published papers that led him to believe the compound was sodium chlorite, a chemical that in various forms is used in municipal water treatment plants. A friend found online the scientists' patent filings. He also consulted an engineer in water treatment to learn more and read environmental reports to get insight into toxicity levels. The chemical is easy to order online and is inexpensive. He estimates he has spent less than $150 total.
      Mixed in distilled water, the sodium chlorite is delivered through Mr. Valor's feeding tube three days a week, one week per month. He says he cautions participants that the chemical isn't as efficacious as NP001 and "that this is only to buy time until NP001 is available to all."

      Jonathan D. Glass, professor of neurology and pathology at Emory University School of Medicine and director of Emory ALS Center, is one of the NP001 site investigators. He said he is concerned that "these people could hurt themselves. Who knows what they are actually making in their kitchen." He says patients need to work with doctors and regulatory officials to find ways to speed up the drug development process. "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 said.

      In the case of Mr. Harris, he enrolled in the Phase 2 NP001 trial. Although no patient knows whether or not he received the drug or a placebo, Mr. Harris says he experienced dramatic improvement in his ability to swallow after his first infusion of NP001. A

    • Spartacus now you are getting wiser as days go by, so tell me
      is this the only company working on ALS, earlier our team had posted few more companies working with FDA so the claim by QCOR is just smoke screen

    • Biologically ACTH derived from porcine of pork, as stem cells from self why shouldn't they work?