The NurOwn drone

The members of NME see it. I see it. Ellen Degeneres sees it (see this video). These 17 people see it (my list of 17 NurOwn responders). Yet several groups, ALS Association (ALSA) included, and some individuals drone on and on about how we must wait for a long and drawn out formal analysis of phase 3 NurOwn data, the date of which has been pushed back three times since 2018. If you have working eyeballs, you should be able to see what the rest of us see: enough data is in. But why do you say you can’t see it? Please ask yourself today why you perpetuate the NurOwn drone?

And where was your droning when Radicava, which had only one trial on just over 100 individuals in Japan (no U.S. trial!) was approved? You say things like “there’s a cost differential of $70k per year” and “well there are complexities that come with NurOwn (freezing stem cells and cultivating the cells)”. Yes, while these are facts, they should not also be obstacles.  You know that these things can be easily overcome thanks to the millions raised yearly in the name of promising ALS treatments.

And where was your droning when Zolgensma got approved before phase 3, based on data from only 21 patients? Zolgensma’s pricetag is $2.125 million for one treatment for one patient.

And where was your droning when the  antisense oligonucleotide (ASO) for the FUS P525L mutation was rushed out with essentially no trial? Pricetag per patient is $700,000 for the first treatment and around $350 for all treatments after (multiple treatments per year).

These are just examples I stumbled upon in my 20 minutes of research, so there are undoubtedly many more like this.

If you are part of the NurOwn drone — ALS Association, Dagmar Munn, the ALSA medical officer who went on national TV saying NurOwn doesn’t work long term (!!!) and so many others who say there isn’t enough data and who say NurOwn doesn’t work long term — please have a second think about the impact of your actions. Please  consider your droning in context of other drugs approved with less testing and less witnessed effectiveness. Please use your eyeballs and common sense. Please look into the 17 ALS patients who are clear NurOwn responders. And please, for the love of all humanity, stop saying that NurOwn doesn’t work on advanced-stage patients, doesn’t work long term, and doesn’t work on certain patient types.  NurOwn worked on the four advanced-stage patients it was tested on. And if you’re so science-driven (as you purport), WHERE are you getting that NurOwn hasn’t worked on the long term?! It works on the only person who has used it long term, Matt Bellina. It has worked in the follow-up treatments it has been tested on in the responders who’ve gone public. It likely works on various patient types too (so stop saying it doesn’t!)

If you’ve been in this fight long enough, you’ve seen Brainstorm use the FDA as the reason for not allowing NurOwn access through Right to Try. Next you saw FDA tell Brainstorm not to use the FDA as a scapegoat. Then you saw Brainstorm say it needs funding (like support from ALSA) in order to roll out NurOwn. Next you saw ALSA use Brainstorm as the excuse (saying Brainstorm basically did not want to take it to the next level yet). Then ALSA went back to an earlier reason they gave, with this open letter, saying that they simply don’t fund treatments in phase 3 but they do  fund “promising research” (!!!)

Droner, do not perpetuate this clusterfudge that is the NurOwn rollout. Really ask yourself why for two years you’ve been standing in between my amazing husband and the treatment that will likely provide a bridge to his next bridge, which may be a bridge to the cure? Please open your eyes and help roll it out today, at least to those who have been fighting for it for years. Dec. 2020 is too late for so many people I love, and besides, the date could easily pushed back a fourth and fifth time.

Yours respectfully,

Mara

Sources:

https://smanewstoday.com/avxs-101-avexis
http://protomag.com/articles/phasing-out-phase-3
https://www.fda.gov/news-events/press-announcements/fda-approves-innovative-gene-therapy-treat-pediatric-patients-spinal-muscular-atrophy-rare-diseasehttps://www.abigail-alliance.org/2019/06/fda-to-help-some-terminally-ill.html?fbclid=IwAR2g20htpEkw5Wj4eJAwLocYovGBGrdVaGFINze1RYZxFQorvjH24ICZ1FU

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