BrainStorm Enrolls First Patients in Phase 3 Trial Evaluating NurOwn in ALS
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BrainStorm Cell Therapeutics announced that the first patients have been enrolled in the Phase 3 clinical trial of NurOwn for the treatment of amyotrophic lateral sclerosis (ALS).
NurOwn is a mesenchymal stem cell (MSC) platform created for the treatment of ALS. Because it is made from MSCs, a type of cell that has the ability to transform into other types of cells, NurOwn can transform cells collected from the patients themselves into cells that secrete neuropathic factors (NTFs), which boost nerve tissue growth. NTFs can also help nerve cells survive through their own neuroprotective function. NurOwn can be injected into muscle or into the spinal canal.
The primary objective of the Phase 3 study (NCT03280056) is to determine if NurOwn can improve functional impairment using the ALS Functional Rating Scale-Revised (ALSFRS-R), a tool for monitoring the progression of disability in patients with ALS. BrainStorm anticipates enrolling approximately 200 patients.
A Phase 2 ALS clinical trial (NCT02017912), now complete, evaluated NurOwn’s safety and effectiveness by using ALSFRS-R and slow vital capacity, a measure of breathing capacity. Positive results showed that NurOwn was able to provide meaningful benefits when compared with a placebo. NurOwn was also proven safe and well-tolerated by participants.
The Phase 3 trial will be conducted at six leading ALS clinical sites in the United States, including the University of California Irvine Medical Center (UCI), the Massachusetts General Hospital in Boston, and the California Pacific Medical Center (CPMC) in San Francisco. BrainStorm opened enrollment last August.
“NurOwn is a highly innovative and advanced stem cell therapy now being studied in a Phase 3 trial in ALS. The support of this trial by the California Institute of Regenerative Medicine (CIRM) highlights the importance of addressing ALS unmet need beyond currently available therapies, which slow disease progression but do not maintain or restore function,” Chaim Lebovits, president and chief executive officer of BrainStorm, said in a press release.
“Stem cells are emerging as a potential viable candidate to treat ALS, and we are very excited to be participating in this important Phase 3 trial. The clinical data from NurOwn have been encouraging, demonstrating clear evidence of clinical benefit and supported by analysis of inflammatory markers and neurotrophic factors in treated patients,” added Robert Miller, director of the Forbes Norris ALS Research Center at CPMC.
“NurOwn cells have the important advantage of being autologous, so patients do not need to be immunosuppressed. In addition, the production process is relatively straightforward, and the harvesting and delivery of cells is much less invasive than other stem cell systems that have been tested in the clinic,” Miller said.
The new study is supported by a $16 million grant from the California Institute for Regenerative Medicine (CIRM).
Karen
Their criteria makes me angry, I am 66 so not eligible but would have been a good candidate, but no one cares about us.
Mark Manchester
What is the criteria for phase 3 of this trial?
No White Flags!
Karen
Good question
Lisa
Under 60 and symptomatic for 2 years or less.
Gerald sparks
https://www.change.org/p/brainstorm-pursue-accelerated-approval-of-nurown-treatment-for-als-patients/nftexp/ex16/control/73816758?recruiter=73816758&utm_
Steven
importance of addressing ALS unmet need beyond currently available therapies, which slow disease progression but do not maintain or restore function
Steven
I'm hopeful this will be a durable treatment or cure God bless Pals
Charlie
Results in 3 years' time?
Cliff Hope
When will it be available to Canadian clients?
Cliff Hope
When will stem cell therapy be available to Canadian Clients? Sunnybrook hospital would be a great place to start.
PLEASE, SOON!!!!!!!!!!!
Pam Haas
So are you taking patients that were in phase 2?? Would love for my husband to be able to get into this phase 3. He did very well in phase 2! I've been praying that he can!
Celeste Lamosse
Pam, when you said he did very well in phase 2, what exactly do you mean? How long ago was it? Has he maintained any gains or plateaus?
Linda Milburn
My husband has ALS. And would like for him to be considered. He is 69, found out he had the disease in March.
Karen
I was told that you have to be less than 69years of age. If you find out different please let me know.
Michelle Kafafi
We ALS patients are so desperate for a cure. More and more people are infected with this most horrid disease. We see our lives going to hell day after day. Please open up the studies to all. We don’t want to die. Have some compassion please someone.
Charlie
"Top-line data are expected in 2019"
Strangely, this particularly important piece of information, from Brainstorm Cell Therapeutics's own website, was omitted from the above article.
Lynne Aronson
I’m 61 and would give anything to be a part of this trial. How can I make this happen ?
Lynne Aronson
Thomas Harrison
When will NurOwn be available in Canada and Israel?
Steven
I'm thinking as soon as nurown show multiple treatments are the way to get ALS patients back to normal and then with FDA approval brainstorm will then supply to Canada and Israel.
Karen
Yes probably for those under age 60.
Thomas Harrison
I thought there was a process in place for early release in Canada and Israel.
Lisa
My clinic (UCI) is one of the clinics participating in this clinical trial. I was told you had to be under 60, diagnosed less than 2 years ago and relatively local. They telephone-screened me earlier this month and they also asked if I needed any breathing interventions. Praying that I cleared the first screening and move on to the next!!!
Karen
Best of luck you are truly blessed to have the opportunity
Dr Zahra Afshari
Patient age 34 just diagnosed symptoms onset 8 months ago limb onset. Are you taking new enrollees for phase 3?
jan berkhey
please hurry withe those stemcells
Anthony DeSimone
I have been diagnosed with a form of ALS (motor neuron disease). I am 69 years of age. I am in very good health with the exception of severe spasticity in my lower body.
Are there any programs in New York offering NurOwn?
Frank Campbell
I would like to know if Searchlight will also do the same work that they do with Radicava when NurOwn is blessed by the FDA. We need multiple places to take our love ones too. We live in Mexico and at present am thinking about Irvine, Ca . But if multiple places are available Texas would be closer.
Cindy
Would like to know why the age limitation to 60 and under. I meet every criteria except age. I’m 63.
Cliff Hope
When will it be available in Toronto, Canada
jan berkhey
all the doctors all over the world have taken the oth of hypocrates, that means that they have to treat patients with all of there knowneledge and capability, us patients and doctors haave to realize that. I think it,s murder to hold back the treatments for us. Many will die, because they don,t speed up the treatment.
Cliff Hope
Time is running out for many ALS patients .... when will it be a government priority to fund ALS research and let patients try new therapies for the disease?????
Zdzislaw Stolowski
I'm 54 years old 7 months I have been diagnosed with ALS, I do not go all the way around, can I be treated with a new therapy? I live in Ireland and I am under the care of Dr. Ola Hardiman Chospital Beaumont Dublin
Vilene Strimbu
My best friend was diagnosed at the Cleveland Clinic this week (6/2018) He is 57, he is having difficulty with speech; therefore I am his voice. I am pleading for help, if there is a possibility of enrolling him in a clinical trial,my husband and I will get him there. Please, could someone contact me for guidance.
david cast
True... they don't care, but if it does work, it will take one year for them to get the results and one year is a lifetime for someone with this disease...