FDA and NurOwn
First, before we go any further I want to put a few cards on the table. Although this is ALS Uncensored I try not to swear egregiously. This is not one of those times. Consider grabbing your earmuffs, especially if there are kids or my sisters-in-law close by.
Second, I own some shares of Brainstorm stock. Less than 4000 shares. If the stock runs up maybe I can afford to send my caregiver on a vacation before she keels over.
Third, I was in the NurOwn phase 3 trial.
Fourth, I was also at the first healing als conference in Salt Lake City and I’ve been to Bodyscience in Miami Florida twice for treatment. I’m the guy throwing spaghetti at the wall to see what sticks.
I think that’s pretty good for disclosure. Don’t you? I’m all for transparency so you can ask me anything at Twitter @scottsfight, Facebook is ALS Uncensored. Alright, let’s get to it then! Hmmmmm?
This is common knowledge. ALS has a lot of problems beyond being a shitty and cruel disease affecting 6000 new patients yearly. It’s not rare but it continues to be underfunded and relatively unknown compared to MS, Parkinson’s, Alzheimer’s and of course cancer although it is a close cousin to the first 3. The past 5 years have seen a significant lurch forward and on several fronts but the needle of life expectancy for the newly diagnosed hasn’t moved very much. If at all.
The vast majority of any limited progress has come from the advent and adoption of social media platforms and the rise of patient advocacy groups such as I AM ALS, the needed aggressive pushing and shoving by NO MORE EXCUSES and the persistent voices of Sandy Morris, Brian and Sandra, Steve Gleason and others. As the drum beat grew louder the whispering voices of ALS patients and caregivers have finally started to be heard and now Facebook, twitter, instagram and tik tok are full of helpful and passionate ALS groups and stories. If you take a moment and step back to survey the landscape, much has changed, most of it is very good. Especially the narrative. Unfortunately, the short 2 to 5 year life span of ALS has continued to bulldoze many warriors into its mass grave and keeping any momentum at all is so fucking hard. Ya know? Of course you know. Hmmmmm?
For sure There have also been a few battles won. The Healy platform trials at Massachusetts General Hospital are great and hopefully will bear fruit soon. The recent approval of the new medication Relyvrio/Amylyx is a good addition to the tool box and insurance and pharmaceutical companies need to stop jerking us around, get off their butts and get it to us asap. The red tape between insurance and government is completely unacceptable and embarrassing in a world so wealthy. I’ll never understand the so-called ethics of how we can throw hundreds of billions of dollars and caution to the wind with Covid vaccines but demand impossible perfection for ALS therapies. Actually, I do understand. It’s a numbers game and ALS just doesn’t have the
numbers to command the attention we need. Anyone who says otherwise is full of shit. Hmmmmm.
There are also other bright spots in ALS to be found. Technology and Team Gleason have arguably moved the ALS needle more than anyone when it comes to quality of life. And isn’t that what it’s all about? Hmmmmm? It is For me anyway. there isn’t much point to extending my life if it’s going to suck. I’m the guy here to tell you that my life doesn’t suck. ALS sucks but my life is fantastic! Everyone has problems and I’m not any different than the next guy. Technology has made ALS livable right now! My quality of life is better than most. I want the same opportunities to extend it. That’s all any of us want. Ya know?
I think ALS has a real perception problem and that is why I do what I do. Anyone can look at me and my situation and it’s easy to draw conclusions of suffering, pain and misery. I get that. ALS is tough and cruel but for most it only affects the body. My mind. My soul. Me! That’s all still here and operating just fine thanks! I need people to stop wondering if they would or could live with ALS, like me and others choose too. These are tough questions and choices but they are personal decisions. For no good reason there are a lot of people and barriers in our way. Just stop it! Sheesh!
To be honest, the saddest and most frustrating part of ALS is this right here. Having to beg. Having to plead. Having to get down on my knees and cry to be heard. It pisses me off beyond words and I feel abandoned by the ALS Association today. But here we are. So be it. I have had neurologists act in a similar manner. Watched them shrug and turn away right in front of me. I’m not the only one. It’s disgusting. I’m on my fourth neurologist because I fired the first two before I moved because of blatant apathy. Ya know? I know you know. What a cluster fuck.
So what is my point? Well if you listen at all to my previous podcasts you know I try to always have a relevantly lucid story and a point. You also know that I’m on the side of pALS and quality of life. So here it is.
NurOwn works. NurOwn works somehow, someway, for some. The testimonies out there are from real people who have responded differently in what appears to be a very broad spectrum of results. I’ve met some of them and I believe I am someone who has also benefited from being in the phase 3 NurOwn trial. I had a great experience even though Covid tried to derail me. I received my first injection in January 2020 at the California Pacific Medical center in San Francisco, second dose in early May and third dose late June. During that time my ALSFRS scores stabilized. My breathing jumped 10 points and my walking strengthened to the point where I was constantly forgetting my walking cane. If you want to know greater detail you can see my interview on YouTube with Mike Henson. While my progression has continued, I just returned from a fun filled long weekend in Tahoe. Not too shabby for a guy pushing into a fourth year with ALS. Ya know? Hmmmmm?
Now, I will absolutely concede that I may have been in the placebo group. ALS is too heterogeneous for me to be matter of fact but this is not important. What is important is that the
FDA allows Brainstorm to present before an advisory committee and everyone else gets the opportunity to have an open, vibrant and transparent discussion about NurOwn, in public. Under the bright lights. Simple. That’s all that is being asked for. Ya know?
I want NurOwn to work. I fucking need it to work damn it! I pray to God every day. But I’m aware that even if it does work, it might not work for me and I’m okay with that, well, mostly okay. The heads of neurology for Harvard Medical and Massachusetts General Hospital think it works. Brainstorm thinks it works well enough to drag their reputations and millions of dollars into the debate. Dr. Brown thinks it works. Many others, way smarter and more qualified and articulate than me have reviewed the data and seem to think NurOwn shouldn’t be left to die and unceremoniously thrown on the trash heap of decades of ALS failure and neglect. Hmmmmm? What do you think? Don’t you want to hear what everyone has to say? Hmmmmm?
I have said before, I had a front row seat to how stage 4 colon cancer is treated. My wife, Jennifer, was just racked with cancer. When they looked us in the eye and told us she had 2 to 3 years to live they knew they were being generous. However, they still threw every medicine and trick at her they could find to try saving her life but it didn’t work. Well, actually that is not entirely true. Her tumors shrank and her liver metastasis shrank quite a bit but it wasn’t enough. She died 55 weeks after diagnosis. Hmmmmm.
But you know what also happened? It worked for the other guy! Yup. Craig and Carlos and others are still very much alive today. Living, working, enjoying full lives with family because the same aggressive treatments were tried and worked for them, and that’s what matters. The same opportunities and privileges given to other diseases must be applied for ALS! The same flexibility and creative effort that was afforded to HIV/AIDS 35 years ago Must happen for ALS now! Right now! Today! And MUST MEANS MUST! Ya know? Well if you didn’t know before you sure do now! Hmmmmm? ALS is tired of hearing about tomorrow. Tired of all the turning away. Damn! Damn! Damn it!
So here is the ask. Because there is always an ask. Please go to I AM ALS DOT ORG , and sign the petition asking the FDA to hold the Advisory Committee hearing for NurOwn, now. Then ask one person to do the same. Easy peasy Mac and cheesy. Will you do that? Please! Ya know, everything has its rubicon and this is the ALS rubicon. The point of no return. If NurOwn can’t get a full hearing. If the FDA can’t stand and take the red face test and explain WHY the data isn’t good enough, even for partial approval, then it’s over for tens of thousands of Americans and hundreds of thousands worldwide. Another decade lost for ALS. This is a fact. Everything else in the pipeline is on a similar trajectory and there are no magic bullets. NurOwn is a tool we desperately need and our current tool box is a few rolls of old duct tape. We need the foot in the doorway the FDA stated we could have in its guidance.Hmmmmm.
So that’s my soapbox speech. My cry for help. Maybe it helps and maybe not. We need one million signatures before December first and not one less. No matter what happens, it doesn’t change anything for me. I have a wonderful life and I’m grateful that I get to see another holiday
with my family and my beautiful girlfriend. I wish you the same and more and may God bless us all!
Besides, it’s only a mild case of ALS.
