The coronavirus is grabbing all the headlines. But May is ALS Awareness Month. The other day, Jonathan Greenfield — a Westporter who is battling the disease — traveled to Washington, DC. His wife Iris and children went too.
They participated in a protest. The goal was for the FDA to fast-track a treatment for ALS as quickly as they are gearing up for COVID-19. Jonathan reports:
There are treatments for ALS that we need now. One is Nurown, involving stem cells. Nurown has been in the pipeline for 10 years, and is currently in phase 3 of a trial.
If I can go to my neurologist, have stem cells extracted from my blood and injected into my muscles and private parts for $600 a shot, why can’t I have Nurown — which is basically stem cells harvested from bone marrow — put through a process and then injected into my spinal column?
It’s absurd that the FDA refuses to open pathways for us. Many other countries already have programs similar to Nurown. ALS patients spend lots of money to be treated there.
The idea that we’re the greatest country in the world concerning health is laughable on many levels. But on the development of drugs we’re especially behind. That is because of the FDA.
My life, or any life with ALS, can’t wait. If the past 6 months are any indication, I will be similar to a quadriplegic in another 6. I recently accepted use of a power wheelchair, and my hands can barely type this email on my phone. My speech is seriously diminished. Last week a feeding tube was inserted into my stomach.
I’d much prefer to write about the free weekly Breathe Thru Zoom sessions my foundation Breathe 4 ALS. But I believe people need to be aware of this FDA situation.
The speed at which the FDA is moving towards a COVID vaccine is amazing. Why not for ALS, when proven and safe treatments already exist?
At the protest, I joined individuals who participated in the Nurown trial. Some walked out of wheelchairs. Some got their voices back. Will this work for everyone? Probably not. But it appears to be a good weapon for the arsenal. It’s a treatment, not a cure.
If given to me today I might be able to preserve function, and last longer. Long enough for BIIB078 from Biogen, which targets the reason I have ALS: the familial gene C9orf72. My father passed from ALS in 2015. Currently my brother and I have it. My aunt is one of the oldest living asymptomatic carriers of the gene. Her son is also a carrier and currently asymptomatic.
BIIB078 is currently in cohort 4 of phase 2, a double blind study. I was unfortunately passed over for cohort 4. I believe they’re gunning to make the jump to phase 3, like they did with the sister drug for the ALS gene SOD1. So in short, I have to last longer.
There are other drugs in the pipeline. ALS will soon be treated like HIV. We’ll be on a cocktail of drugs. If ALS were contagious, we’d be there today. And with recent success reversing sickle cell through repairing genes, there is very good reason to believe a cure for my family is coming.
But will I see the day? If the FDA keeps ignoring ALS I will not. Sadly I won’t see my kids off to middle school, high school or college. Even now our usual surf trips to Rockaway have stopped. I’ve gone from the adventurous dad, to the dad who can barely engage.
But my kids have been amazing! I love their help, and hate it at the same time. It was chilly at the protest, and they voiced not one complaint. Not one despite the blocks we walked to get in front of the White House.
They still don’t really understand the rights our democracy affords them concerning protest and freedom of speech. Yet I believe this experience was imprinted in their DNA. They’ll be able to call back to it in years to come.
Meanwhile, if you’d like to help: Please call your senators and representatives!
My mother passed away from ALS in January. It would have been amazing if we had any medication at all that would have helped her.
Jonathan Greenfield is the strongest person I know! Keep fighting!!!! BREATHE!
Thanks for this great update on your trip to DC to protest, Jonathan. ALS deserves a ratcheted up approach to clinical trial testing for Nurown and others as made available, arguably just like many promising cancer innovations that whither in the “valley of death” where promising drugs go to die. The drug development system is not geared to support promising early stage innovations and needs rethinking and fixing. Maybe a conversation with @MonaJhaveri @ SoundAffects.org would be useful at some point to synergize a bit, as the US can clearly get it together when it wants to for contagious pandemics – while other diseases get unwittingly deprioritized and marginalized in the process.
Thanks, Michael. You know that Mona Jhaveri is a Staples grad, right?
You should be able to get the Drug under the Presidents “Right to Try” Bill. I hope!
Nope. Drug makers don’t have to comply. And who’s going to pay? I asked for the price of admission for Biogen’s BIIB078. I was told flat out there is none. The bill sounds great, but has no teeth.
So sorry Jonathan you are going thru this. Happy to help or support such an important mission. Stay strong.
Excellent and informative article — THANK YOU for spreading the message. I will call my congressman and Senator and I will forward this eloquent article to them. Keep up the fight!
Ice Bucket Challenge II Donations:
As some of you know, I was diagnosed with ALS in July of 2019. Early symptoms had started in September of 2018. There are no known bio-markers from which to diagnosis.
In June of this year there will be enrollment at 54 locations, 4 of which I have my name placed on their lists of people interested. This will link you to information about the trial:
It will be the first platform clinical trial for ALS. They will be trying several new meds that are aimed at slowing/stopping/reversing what is currently categorized as a fatal disease. The information I’ve received points towards making ALS a chronic rather than fatal disease within in this decade.
If you have that long.
As mentioned by Jonathan Greenfield, there is a stem cell clinical trial that is now in phase 3, and final results could be presented within the next couple of years or less. I’m very optimistic for a cure, but I pray for the benefit of all ALS sufferers that the answers come sooner than later .
All that said, if you can please chip in some $ to help not just me, but the thousands of others that suffer from this progressive disease. Please go to the link on top.
If you have questions, or just want to touch base, please reach out. In general, they have enough data points to consider me a “slow progressor”. Finally, being slow is a benefit! I am on meds that slow the progression further, but they don’t stop it. I have developed dysarthria, but I’m still understandable. My left leg has “foot drop” and I use a custom carbon fiber AFO for that leg. I can still walk, but I am a bit wobbly. Upper body strength is fine at the moment. I’m still working (from home!).
Pass this on to anyone. Your help is appreciated!!
Still waiting! Be hopeful!!