The NurOwn drone

The members of NME see it. I AM ALS sees it. I see it. Ellen Degeneres sees it (see this video). These 29+ people see it (my list of 29 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 to move on this. But why do you say you can’t see it? Please ask yourself today why you perpetuate the NurOwn drone. NurOwn worked in 35% of those treated (since 2016). It’s safe. FDA has approved far less effective drugs for far less brutal diseases. Something is fishy and we need to get to the bottom of it.

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 insurmountable obstacles given the brutality of ALS.  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).

Are you watching the many untested treatments for COVID getting rolled out today? They are being studied as they get rolled out. Are you also against this? Pres. Trump gives half a billion to Johnson & Johnson, who doesn’t even have a COVID clinical trial.

These are just examples I stumbled upon, so there are undoubtedly many more like this.

If you are part of the NurOwn drone — ALS Association, Dagmar Munn, NORD, the Exec VP of Mission Strategy of ALSA 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 look into the 29+ 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 likely works on various patient types too (so stop saying it doesn’t, stop standing in our way, & stop using patient types as a reason to delay rollout!)

If you’ve been in this fight long enough, you’ve seen Brainstorm point to the FDA as its reason for not allowing NurOwn access through Right to Try. Next you saw FDA more or less 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 retracted that and 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 so many 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 see that the fight against ALS needs to match the ferocity of ALS. We need to quickly work together (FDA, ALSA, and Brainstorm), be creative,  plan it out (QUICKLY!), then get ‘er done. ALSA, you can make up for past mistakes but you’ve got to start NOW. Funding should be the least of the hurdles at this point.

Yours respectfully,

Mara

Sources:

Potential class action lawsuit against Brainstorm: https://www.prnewswire.com/news-releases/shareholder-alert-pomerantz-law-firm-investigates-claims-on-behalf-of-investors-of-brainstorm-cell-therapeutics-inc—bcli-301239286.html
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