Exciting news was published this week that will give patients suffering from ALS, also known as Lou Gehrig’s disease, something to cheer about. The journal JAMA Neurology reported that a new stem cell treatment was successful in slowing disease progression in a small group of ALS patients in a Phase 2 clinical trial.
This is big news for a fatal, incurable disease that is well known for its progressive, degenerating effects on nerve cells in the brain and spinal cord. We’ve written about ALS a lot in the Stem Cellar, so if you want more background on the disease, read our “Progress to a Cure for ALS” blog.
A patient’s own stem cells can help
The stem cell therapy involves extracting mesenchymal stem cells from the bone marrow of ALS patients. These stem cells are then manipulated in culture into cells that secrete a growth factor called NeuroTrophic Factor (NTF), which helps keep nerve cells in the brain and spinal cord healthy and alive. The NTF-secreting stem cells (called NurOwn cells) are then transplanted back into the same ALS patient (making this an autologous stem cell therapy) by injection into either the spinal fluid or the muscles.
The NurOwn method was developed by BrainStorm Cell Therapeutics, a biotech company based in the US and Israel. Clinical trials to test the safety and efficacy of NurOwn stem cells began in 2011 at the Hadassah Medical Organization (HMO). So far, 26 patients have participated in the trials both in the US and in Israel.
According to the JAMA publication, patients were monitored 3 months before and 6 months after they received stem cell transplants and 6 months after. Twelve of the 26 patients participated in an early stage of the trial (phase 1/2) to test the safety and tolerability of the stem cell therapy. The other 14 patients participated in a later stage (phase 2a), dose-escalating study where their modified stem cells were injected into both their spinal fluid and muscles. Following the treatment, the scientists looked at the safety profile of the transplanted stem cells and for signs of clinical improvement in patients such as their ease of breathing or ability to control their muscle movement.
Stem cell treatment is effective in most ALS patients
Results from the clinical trial showed that a majority of the patients benefitted from the NurOwn stem cell therapy. HMO Principle scientist and senior author on the study, Dr. Dimitrios Karussis, explained:
Dimitrios Karussis (Israel21c)
“The results are very encouraging. Close to 90% of patients who were injected intrathecally through the spinal cord fluid were regarded as responders to the treatment either in terms of their respiratory function or their motor disability. Almost all of the patients injected in this way showed less progression and some even improved in their respiratory functions or their motor functions.”
A PRNewswire press release covering this study called the stem cell therapy the “first-of-its-kind treatment for treating neurodegenerative diseases.”
Not a cure just yet
This stem cell therapy will need to be tested in more patients before the it can be determined truly effective in slowing progression of ALS. And Dr. Karussis was quick to note that the NurOwn stem cell therapy isn’t a cure for ALS, but rather an early-stage therapy that will provide significant benefit to patients by slowing disease progression.
“I am optimistic that within the foreseeable future, we may provide a treatment to ALS patients that can slow down or stop the progression. I believe we are in the early stages of something new and revolutionary with this harvested stem cell infusion therapy. While this is absolutely by no means a cure, it is the first step in a long process in that direction. I see this treatment as being potentially one of the major future tools to treat degenerative diseases of the brain and spinal cord, in general.”
Other stem cell treatments for ALS in the works
A single stem cell therapy that could treat multiple neurodegenerative diseases would be extremely valuable to patients and doctors. However, it’s not clear that the “one ring to rule them all” scenario (couldn’t help making a Lord of the Rings reference) will play out well for all diseases that affect the brain and spinal cord. Luckily, Dr. Karussis and Brainstem Cell Therapeutics are not the only ones pursuing stem cell therapies for ALS.
Clive Svendsen
CIRM is currently funding 21 studies (a total of $56.6 million) that use stem cells to either study ALS or to develop therapies to treat the disease. We wrote about one recent study by Clive Svendsen at Cedars Sinai which is using a combination of gene therapy and brain stem cells to deliver growth factors to protect nerve cells in the brain and spinal cord of ALS patients. Currently, Svendsen and his team are in the latter stages of research and hope to apply for FDA approval to test their therapy in patients in the near future. Svendsen told CIRM, “we will begin recruiting patients the first week we have approval.”
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The Stem Cellar 
What about a person who has seven adult children? Could replacement protein or other cells possibly help cure or improve the patient. My mother has bulgar ALS and is 75. She is a patient of Dr. Weiss in Seattle at t UW Medical center.
My grandmother passed away from ALS. It was really sad to see her go like that. It got to the point where she would chew food for flavor and then just spit it out into tissue. She died because that made her thirsty, and then she couldn’t swallow her water, and then she aspirated. It’s so sad. It’s really exciting for me to follow the advancements in treating this horrible disease! Thank you for sharing, these things always make my day better.
Good evening Sir :I am Hemendra Vidya Saagar from Tamilnadu India. My father affected ALS disease now he unable to walk and too breathing difficulty. Due to underwent for treatment for Hospital now he associated with trachestomy. Swallowing trouble also there. Now he capable of ryles tube feeding undergoing..
My wife is suffering from ALS bulbar ince last thirteen years. She is not able to walk, speak and any kind of movement. She takes semi solidfood and drinking liquid with great difficulty
Hello, I was wondering if you had any stem cell trials coming up in ALS patients.? This disease as been in our family for decades and now my brother was diagnosed in July 2016.. Any kind of trials would be greatful.. Thank you for your time.
Hello, why isn’t this already on FDAs desk? Also, University Medical Center in Jackson Ms would be a great place to start a trial as well. This should be expedited by FDA!
I totally agree.
where will the next trials be for ALS patients. My husband was diagnosed with the disease and is rapidly declining. He is bedridden, unable to turn over without help and has trouble swallowing. Please send me details on the trials being conducted for stem cell treatment.
My husband was diagnosed with ALS about a month ago. He is interested in finding out more about the Stem Cell treatments. Any help would be appreciated.
Hi Sharon. I’m sorry to hear about your husband’s diagnosis. Our agency is funding a clinical trial testing a stem cell-based treatment in ALS patients. You can read about the trial in our blog. If you are interested in learning more about the trial or contacting the trial sponsor you can find information here.
ALS is a very important
Be patient is required
My daughter in law has ALS, her brain power is very clear in that she is Normal very alert and precise in
thinking. Only problem is that she cannot walk or move on her own, lost all limbs power, wastage of flesh,
muscles and nerve power in her wrists and hands, impossible to even hold or lift a small spoon in her
hands ir brush her teeth. She was hale and healthy till 2013, but in 2014, this disease began to manifest
itself slowly but steadily till she could not use any of her limbs or hands. She use to walk with a walker
steadily, able to sit on a chair go to the loo with the walker, eat her own breakfast but declined since then.
she is in a wheelchair, has to be lifted up bodily by someone daily. Since she is bright and clever as her brain functions normally, what is the reason for the decline of her limbs? Marie was working in Toronto,
Canada, when this sudden attack occured which destroyed her entire career and brought severe problems to the family not only financially but stressful for her and shock to the entire family to see her in
such a physical helpless state.
URGENT PLEASE TREAT THIS “ALS” DISEASE WHICH IS RUINING THE LIVES OF HEALTHY PERSONS YOUNG AND OLD ALL AROUND HE WORLD”. GIVE THIS ALS TOP PRIORITY AS IT IS SORROWFUL TO SEE LOVED ONES SUFFER AND STAND BY HELPESSLY. IT IS LIKE CANCER
EATING AWAY THE LIVES OF YOUNG AND OLD RIGHT IN FRONT OF THE EYES OF THE FAMILY MEMBERS WHO ARE HELPLESS TO HELP THEM.
KINDLY TREAT THIS AS URGENT WHICH NEEDS RESEARCH TO STUDY THE CAUSE AND CURE OF THIS DREADFUL DISEASE.
THANK YOU.
I’m in the same boat I was diagnosed 1-17 and I’ve also degenerated in the last 10 months . I’m still able to use a walker but my hands and arms are getting weaker by the week. I have trouble eating ,holding utinsels and swallowing. I would love to do a trial but have tried to get in dozens never got a response. Starting to think these research trials are bull since they always ask for money that none of have anymore because of the disease.
Hi Herb. The clinical trials CIRM is funding for ALS at Cedars-Sinai and BrainStorm Cell Therapeutics do not charge patients money to participate. You can learn more about these trials and their contact information on clinicaltrials.gov:
https://clinicaltrials.gov/ct2/show/NCT02943850
https://clinicaltrials.gov/ct2/show/NCT03280056
what state are you located in ?
Hi Mike. CIRM is located in California. The clinical trial for ALS mentioned in this blog is being conducted at Cedars-Sinai medical hospital in Los Angeles, California. You can find the contact information for this trial here: https://clinicaltrials.gov/ct2/show/NCT02943850
Any idea on when FDA will approve this,. I do not have the wealth needed for this treatment without help from insurance
Hi Angela. I don’t know the answer to your question unfortunately. Brainstorm is testing their stem cell treatment in Phase 3 clinical trials right now (one of these trials CIRM is funding and you can read more about it here: https://www.cirm.ca.gov/clinical-trial/phase-3-randomized-placebo-controlled-multicenter-study-evaluate-efficacy-safety). If the trial shows that this candidate treatment is safe and effective in ALS patients, then the FDA will decide whether it will be approved. You can keep track of Brainstorm’s progress on their website: http://www.brainstorm-cell.com.
This is amazing! My father passed away just maybe 6 months after being diognosed with ALS in 2006. Seeing that there is progress being made makes my heart so happy. It is a terrible thing to watch a loved one go through.
I am interested in the stem cell research. It runs very high on my dads side. In my lifetime, I have lost my grandmother and aunt. My dad was just diagnosed this week. He is interested in this & anything we can do to find out why it’s so high in our family. Please contact me back with what we need to do.
Dear Jennifer, thank you for your email. I’m sorry to hear about your dad’s recent diagnosis. That must have come as quite a shock to the family. The clinical trial we are funding is being run by a company called Brainstorm. Here is a link to the website that has all the information you need to determine if your dad is eligible to be part of this trial and also who to contact if he would like to apply.
https://clinicaltrials.gov/ct2/show/NCT03280056
I hope that helps.
Yours truly
Kevin
Hi, what is happening with the application to be able to provide this treatment for a fee out of Canada and Israel? Last year brainstorm announced that they where aiming to have this in place soon, but its gone very quiret. Can you help with an update on this please?
Many thanks
Helle
Dear Helle, thanks for your comment but I’m afraid we are only involved in the California portion of this clinical trial. The research taking place in Canada and Israel is not something we have any connection with – even though it’s the same company and same therapy. You would have to contact Brainstorm directly to find out about that.
I hope that helps.
Yours
Kevin
My husband was diagnosed with ALS about a month ago. He is interested in finding out more about the Stem Cell treatments. Any help would be appreciated.
Dear Kredi, I’m so sorry to hear about your husband’s diagnosis. I can only imagine how distressing it was to hear that he has ALS. Here is a link to the page on our website that talks about the use of stem cells for ALS and also has information about two clinical trials we are funding targeting the disease. I hope this is of some help.
https://www.cirm.ca.gov/our-progress/disease-information/amyotrophic-lateral-sclerosis-als-fact-sheet
kevin
Please share any locations near Portland Oregon that will treat or offer clinical trials for my Sister Diagnosed last week with ALS (It has been progressing for 2 years)
https://alsfight.blogspot.com/
als for my Sister Diagnosed last week with ALS (It has been progressing for 2 years)
My younger cousin got diagnosed with ALS last month. Previously, he was having trouble with his kidneys, and were on donor receiving list. Is there any doctor around NJ area who can enroll him into trial study?
I am so sorry to hear about your cousin. I can only imagine how devastating it must be to get that kind of diagnosis. I’m afraid I don’t know of any clinical trials in the NJ area.The only clinical trial I know of that might help is this one: https://clinicaltrials.gov/ct2/show/NCT03268603
If you are looking for a support group the ALS Association is a great place to start. They have information on doctors in your area and also other patients who can offer guidance and advice.
I hope that help.
Yours truly
Kevin