But this is the unoptimized cost before manufacturing has been made more economical, and the cost of nursing care for an ALS patient with advanced disease is typically hundreds of thousands of dollars per year, which is usually covered by Medicaid and paid for by taxpayers. Critics also mention the high cost (up to $300k per patient). And given that current ALS "treatments" save 0% of patients, even if Nurown saved a small proportion, that would still be a massive victory. ![]() They also say that it does not work for all patients, which is true, but biomarkers have been identified that accurately predict if a patient will respond to the treatment as expected. The company developing this treatment, Brainstorm Therapeutics, has nonetheless made one last push, rushing to publish the journal paper linked above with the expanded data and additional science.Ĭritics have stated that Nurown has "failed its trial", but that seems to be ignoring all of the incredible documented results and the obvious flaws in the trial design. Because of the poor trial design, it did not meet "statistical significance in primary efficacy endpoint", and the FDA has used this as an excuse to informally reject it last spring, giving a recommendation to the company behind closed doors against filing an application. The parties that have an interest in Nurown not being approved have used this information to claim that the treatment does not work, despite the apparently undeniable results. In other words, this trial was like giving an early-stage cancer treatment to patients that had already progressed to stage 4. Unfortunately, since the trial was too slow for this quickly progressing disease, most of the patients that got the treatment were already too far along in their decline. The data also showed that the probability and magnitude of a patient's response is highly dependent on how early they were able to receive treatment. Or on the other side of the spectrum, for the man listed above that could barely walk then was able to run, his improvement from walking to being able to run was not recorded. For example, if a patient starts with completely paralyzed legs, then improves to being able to move their legs but not able to support themselves to walk, the data would show "no improvement". It's flawed because it often misses changes in symptoms. In ALS research, the primary metric used to evaluate the effectiveness of new treatments is called the Revised ALS Functional Rating Score (ALSFRS-R), which is the result of a questionnaire that asks questions such as "can the patient walk?" and assigns a score of 0-4 for that category (0 for "not at all", 4 for "normal"). After their access was taken away, the patients that received Nurown in the trials have begun declining again, and many have already lost their battle with the disease. However, due to a botched trial design and unfortunate politics surrounding this disease, Nurown might take several more years to reach patients, or might never get to patients at all. And this long list of 30+ additional patients that reported strong measurable responses. A former navy pilot that saw a significant reversal of symptoms, including breathing improvements and being able to stand up from his wheelchair, after not being able for more than two years. A man that could barely walk, received Nurown, and recovered enough to run around the room at the FDA headquarters in front of their director. Patients in the trial and ongoing expanded access program have seen impressive results, including stoppage of the disease and even some symptom reversals. Nurown, a stem cell treatment for ALS, concluded its phase 3 trial and recently published data to a peer-reviewed journal in December that gives strong evidence that this is the first disease-modifying treatment ever developed for the condition. Sorry for posting what might be seen as overly political or incendiary, but I believe that this community should be aware of this information because it could save many of your lives or the lives of your loved ones.
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