Advancing Potential ALS Treatments Is Like Accelerating a Glacier
Glaciers are known for their slow movement and the transformative change that they leave in their wake. Most move only a few centimeters a day. Yet they produce lakes, cliffs, moraines, valleys, mountain arêtes and horns, and pronounced landscape striations. Perhaps stem cell research will prove analogous.
BrainStorm Cell Therapeutics is enrolling a Phase 3 study that uses bone-marrow-derived cells extracted from the patient. These cells can differentiate into bone, muscle, fat, and cartilage. Prior to reintroduction, the cells are cultured to produce higher levels of neurotrophic factors — chemicals that protect neurons. The trial is attempting to gather 200 participants who are declining at least one point per month as measured by the ALS Functional Rating Scale (ALSFRS).
Preclinical research into the use of stem cells in ALS was first done in mice in 2006. Based on these mice studies and evidence that stem cell injection was safe, BrainStorm began a clinical program called NurOwn. A Phase 1 safety and tolerability trial demonstrated both. The company then sponsored a Phase 2 trial, and no serious adverse events were observed. It was reported that the treatment slowed disease progression as measured by ALSFRS. Increases in muscle strength and bulk, and also arm and leg circumference, were observed.
BrainStorm then conducted a larger Phase 2 trial. The primary goal was to confirm safety and tolerability, and a secondary objective was disease modification. Again, there were no serious adverse events. More importantly, once the study participants were divided into fast and slow progressors, the fast-progressing subgroup was shown to have an improved rate of decline.
Topline data from the Phase 3 trial is expected in 2020. That means, pending positive results, a stem cell treatment for ALS could be approved in 2021. And there may be more coming. Q Therapeutics has plans to launch a clinical trial this year. Neuralstem has developed a spinal-cord-derived neural stem cell line, currently in clinical-stage testing, for the treatment of ALS and other nervous system diseases and injuries. The Mayo Clinic and Cedars-Sinai also have undertaken research initiatives into the efficacy of stem cell treatment relative to ALS.
That is all exciting and promising. But if an ALS stem cell treatment is commercially available in 2021, it will mark 15 years since it showed potential in the lab. To anyone with ALS, the pace of progress feels glacier-like. However, if the impact on the patient population is equally glacial, the time to market will be quickly forgotten.
It is impossible to predict the gains to be had in modifying disease progression via stem cell therapy. The median benefit as reported in both of BrainStorm’s Phase 2 trials were modest. The optimum amount and frequency of dosing has yet to be established and may vary from person to person. Once this is better understood, the expected benefit will only increase. Still, the stem cell saga had left me mostly nonplussed.
That is, until I saw footage from ABC News. It shares the experience of two patients whose improvements since receiving BrainStorm’s NurOwn have been striking. Both have regained function. One can stand on his own and even walk a bit, after more than two years of being unable to do either. If their progress is maintained and replicated in others, we may be on the cusp of a glacier-led retreat of ALS.
Continuing the metaphor, there is a rare phenomenon called a galloping glacier. About 1 percent of the world’s glaciers exhibit significant bursts of relative speed. One in northern Pakistan “gallops” every 20 years, moving nearly 1,500 times its normal crawl. Scientists are left to speculate why, but most theories include a reduction in friction.
We can be a virtual frictional agent of change. First by rushing to participate in any open stem cell trials. Second, by being vocal. After a Phase 3 trial is complete, the FDA must approve the therapy, insurance must deem it inclusively coverable, and manufacturing and delivery must be scaled up. We must collectively scream that all tasks necessary to afford treatment access be attended to with life-or-death urgency.
A glacier is like a moving mountain. In this case, it just needs to move faster than normal. Jesus said that faith can move mountains. My pastor describes faith as belief plus trust. I believe that WE can. I trust that WE will.
Keep the faith!
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Give me a break. This particular stem cell procedure should have been available in the United States. Big Pharma and the FDA shuffle their feet while people with ALS are dying every day. Compassionate care is available all over the world but here. My husband has had ALS for over 5 years. He did receive 3 stem cell transplants, but not nerown. Just wait, it will be soooooo expensive , it will be impossible to get even when approved. All about money. All people say is get into clinical trials. We've tried at least 20 times to no avail. These companies act like they are Gods. It is so inhumane. I'm sorry to sound so negative but it is so frustrating. And we do think the stem cell treatments did help my husband. But when so many people are affected by this disease, let them make their own choice to try it. This is BS.
Becky you correct to voice your opinion .....the US Government approved the sale of Naive blastomere pluripotent stem cells technology to the Japanese (Astellas) for pennies on the dollar in 2015 and they buried the pre-embryonic technology in favor of induced pluripotent stem cells .....very politics. 4 years later nothing . Your husband needs Embryonic Mesenchymal Signal (Stem)cells -MSC- from pre-embryonic cultures that have an HLA matched for reboot, rescue and repair of the immune system ....Ask the doctors where is it ?
I have to agree!! These trials and "testing" and such should be free or at least Very Little cost to the patients.... Help us help you..if you want to figure out all this....and why would age name a difference??? Everyone is different anyway, outcomes will be different....so Ethats the big thing about the age??? Let's test all this stuff out and If these trials and such are too damn expensive there will be no one or very few that will be able to do these.....Come on... I agree, it's all about the money!
Your apt analogy drives home the point. And your last line - another analogy from the Master Teacher illustrating the power of active faith. Love your column.
The 'Master Teacher' doesn't seem to be doing much for pALS.
NurOwn seem to be working hard and doing their best for pALS.
The Master Teacher's dad created the universe and everything in it. That includes ALS.
Now...why did he do that ?
'My pastor describes faith as belief plus trust."
I believe I trust NurOwn to be the more likely to produce good outcomes for pALS.
The 'Master Teacher's' dad is said to BE love.
God loves us so much, says the Dusty Old Book.
Is ALS the way he shows his love for us?
(Asking for a friend.)
I was diagnosed in early 2017 Symptom onset Oct 2016. Became aware of Neurown and met every criteria except age. The cut off was 60. I was 62 at the time. Now I’ve had the disease too long to qualify for NurOwn, even if the age factor were modified. Still I am hopeful it will be available in time for me. If they fast tracked Radicava they can fast track stem cell therapy. We patients need to advocate for ourselves. Thank you for your column.
Rick, thank you for this excellent summary of the progress of BrainStorm's development of NurOwn to date. I was diagnosed in March, 2019 with symptom onset in early fall, 2017. At 73, I am well over the age cut off for Phase 3, but reading your article helps to motivate me to do all I can to "stay upright and walking" until NurOwn or a similar treatment becomes available. With this disease, sparks of hope are so rare; thanks for offering us one today.
Yes I am on radicava now but just like you too old and too far along since diagnosed. I’m still doing good , just hoping nurown is fast tracked!
"If they fast tracked Radicava they can fast track stem cell therapy."
Well, actually, no.
Thing was, Radicava was already approved for patient usage - for strokes; in Korea and Japan.That was why it could be fast-tracked.
That won't happen with stem cell therapy for pALS
Anyways, Radicava is mostly useless to the majority of pALS. (Terrific for the share price though).
Whenever I hear the words 'fast-track' I think the words 'BS.'
You should also check another patient that received Nurown called Mark, he went from a wheelchair to running around after three treatments:
Yes indeed, we have three neurown patients who recorded amaizing improvments so far... and I can’t wait for the fourth!!
There are plenty of the phase III trial and there were some that reported improvement after the poo hase II trial ( with a single dose) such as Boby or Michael Cimbura...
Dr. David Greene
Awesome Pepe, Thank you for sharing this here, would inspire other to go for stem cell treatment which is real game-changer.
BLOG: The Backbone of Neurons
supports growth cone formation
A stabilized cytoskeleton is a condition for building growth cones. They are compounds that can stabilize the backbone of the growing axon. In fact, in vitro treatment with paclitaxel (a chemotherapy against cancer), or epothilone B (another class of potential cancer drugs) improve growth cone formation in adult spinal cord neurons. This category of drugs offers a very interesting target to promote regeneration.
And this is true for two additional reasons. First, they seem to help transport the signal that neurons are damaged and that they need to regrow. Second, recent experiments show that they diminish the formation of a scar and limit the number of inhibitory molecules.
What does that mean?
My heart goes out to everyone who have or cares for ALS patient. My husband was diagnosed with ALS 3years ago, He had gone through several bouts of depression, the first sign of ALS appeared in the hand and arm as he experienced difficulty with simple tasks such as buttoning a shirt, writing, or turning a key in a lock later affected one of the legs, trouble breathing, slurred and nasal speech, difficulty chewing or swallowing. He also experienced awkwardness when walking or running and I noticed that he was tripping or stumbling more often. He had gone through several bouts of depression, over 6 hospitalizations in just two years. Coping with ALS wasn’t easy. He had attempted suicide and all treatment had done little for his improvement. When I became fragmented in my thinking, I started to have my worst problems. We've finally benefited from some real dedicated support group of people with natural treatment and a small number of friends and family that knew and loved us. Dr vihaan treatment has turned his life around, he is now symptom free. I don’t want people to feel that suicide is their only option, know you are loved, you are valuable, you are worth more than your darkness. Reach out and get the support you need : start your natural treatment, Try to understand the blackness, lethargy, hopelessness, and loneliness they’re going through. Be there for them when they come through the other side.
Crystal, who is Dr.Vihaan you mentioned in your comments? What is his contact information?
My mother was diagnosed with Als dec 3 2019. She is 64 , we are looking for anything that will help
brad. check out teamstevensnation movement. his bless. stay strong.
Dr. David Greene
Brad, I am sorry to hear about your mother, and I will advise you to select a provider with a successful background and the one who has approval, because many are just running businesses.
The ALS foundation is a bunch Of BS! All the money they have gotten where has it gone?
Someone needs to be held accountable. And the FDA is as always dragging it's feet ALS patients need help now not five years from now.
Very disappointed about age limit of 60 or under for clinical trial phase 3 at UCI.
I may have to go out of the country--not happy. I want help now, not 5 years from now. I may not be here.
R3 Stem Cell
Mary, We understand your concern, and i would advise you to consider a personal visit to a clinic.