For the past two years, Sarah Benoit has been getting around with the help of a walker, waiting for a medicine that’s out of reach.

Benoit, a former congressional aide, has ALS, a fatal neurological disease that gradually causes muscle weakness and paralysis. As it progresses, she struggles to maintain a normal life, but holds out hope that a drug called Radicava — the first new treatment in 22 years — will slow her deterioration.

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  • This world is really cruel, I am dying and these people are laughing and thinking about my money instead of my life, I am just seeing what will happen next, which member of my body will be diagnosed next. Over in Afghanistan, I have 1,00 USD salary which is equal to 6,000 thousand Afgahani, how can I get 145,000 USD to pay for my treatment, shame on you Americans and the company who is producing the Radicava, you people are always talking about Human Rights, so where are my rights and your.

  • My father had ALS (amyotrophic lateral sclerosis) for 3 years His first symptoms were weakness in his hands and losing his balance which led to stumbling and falling. He never lost the ability to swallow or breathe. His one hand, then his legs, were affected first; then his arms. The Rilutek (riluzole) did very little to help him. The medical team did even less. His decline was rapid and devastating. The psychological support from the medical centre was non-existent and if it were not for the sensitive care and attention of his primary physician, he would have died . There has been little if any progress in finding a cure or reliable treatment. So this year his primary physician suggested we started him on Natural Herbal Gardens ALS Herbal mixture which eased his anxiety a bit,We ordered their ALS herbal treatment after reading alot of positive reviews, i am happy to report this ALS herbal treatment reversed my dad condition. His quality of life has greatly improved and every one of his symptoms including difficulty in walking and slurred speech are gone. Their official web site is ww w. naturalherbalgardens. c om He will be 74 soon and can now go about his daily activities

    • yet another unproven remedy offering a complete cure. Do you really think ALS patients are that stupid and/or naive?

  • ““Or if the FDA uses a broader scope for approval, we may still disagree and say we’re not going to pay for it that way. FDA approval is a first step, but only one step.

    “That said, I think our approach to the whole health care system in the U.S. sucks. In a different setting that’s not so profit driven, the rationale would be different and you’d have a different answer” for Radicava, he continued. “But it’s a money-driven system.””

    What a scoundrel. He blames the “money driven system” – but it is precisely his company’s drive for money and profit that he thinks he can decide to reject the labeling criteria that the FDA decided. These insurance companies should NOT have the power to overrule the FDA and its decisions on what medicines are approved and for what patients.

  • Please fact check. Edaravone is a 17 year old product. It’s a repurposed Drug and there were no US r and D costs. The payers are restricting because it failed to work in a larger more ill population with ALS. The failed trials are J16, J17 and J 18. The data is published. The drug is generic in most of the world including Japan. It does not cost $38,000 in Japan. More like 12k for brand and under 5 k for generic. The als Association gets money from the drug company and they refuse to expose this. It’s just like the whole Marathon issue. The drug has been shown to work in less than 7 percent of people with ALS.

    https://www.google.com/amp/s/alsnewstoday.com/2018/01/18/present-all-sides-when-discussing-radicava-als-physicians-urge/%

    https://www.tandfonline.com/toc/iafd20/18/sup1?nav=tocList

    It’s been indicated in Japan for 4 years. Where’s the post marketing data? The company has already made over 2 billion on this product for acute stroke.

    Please go back and get the facts. They are preying in a vulnerable population with ALS. Why is ALSA not doing their job ?

    • Hi Janice,

      Thanks for your note.

      As the story noted, the drug was approved in Japan more than a decade ago for another purpose.

      The trial focused on patients in an earlier stage of disease – there is no evidence one way or the other that it could not help people in a later stage of the disease, which the story also noted. As for the cost, that is the information I have at present.

      As for the ALS Association, the organization has told me about its concerns with the cost of the drug in the U.S. The comments from the executive director, while critical of insurers, also refers to the underlying price that is causing problems for some patients. I have updated the story to note that MT Pharma provides funding.

      I have spoken to association personnel in different parts of the country who were also concerned about the cost, not just insurance denials, and they were helpful directing me toward people who were having difficulty obtaining the drug.

      As to your question about post-marketing data, I do know the FDA approval requires the company to submit a follow up study on all ALS patients in 2022. I will ask about data from Japan.

      Thanks for writing in,
      ed at pharmalot

  • Most ALS patients who take the DP slow down the progression of their disease. As soon as Radicava is stopped the disease accelerates. The DP is cheap and over the counter. http://www.winningthefight.org.
    Further ALS is caused by a bacterial infection that is very difficult to detect. All should be on appropriate antibiotics to cover stages of the bacterial reproduction and it’s location. This has been proven with my daughter.

    • Conclusions
      Mitochondrial dysfunction, glutamate excitotoxicity,
      and oxidative stress have all been implicated in ALS
      pathogenesis, and targeting these mechanisms indi-
      vidually or by a cocktail such as the Deanna Proto-
      col could play a role in future ALS therapies.
      However, many of the preclinical and animal studies
      related to these pathways have not translated into
      successful treatments in patients with ALS. While
      there are anecdotal reports of improvements in
      patients with ALS on the Deanna Protocol, there is
      no convincing objective evidence of benefi t yet.
      Thus, at this time, ALSUntangled does not recom-
      mend the Deanna Protocol to patients with ALS.

      http://informahealthcare.com/doi/pdf/10.3109/21678421.2013.788405

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