Israel’s BrainStorm Offers Encouraging Phase 2 Data on Stem-Cell Therapy NurOwn to Treat ALS
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Israel’s BrainStorm Cell Therapeutics has announced positive top-line Phase 2 data on NurOwn, a customized stem-cell therapy technology to treat amyotrophic lateral sclerosis (ALS).
BrainStorm has been developing and commercializing NurOwn in partnership with Ramot, the business engagement unit of Tel Aviv University. NurOwn enables the production of growth factors that support the survival of neurons, a critical step in treating neurodegenerative diseases.
In July 2016, BrainStorm announced positive results from its phase 2 randomized, placebo-controlled study of NurOwn in ALS patients, and presented the results at the 27th International Symposium on ALS/MND in Dublin, Ireland. That study mainly showed that NurOwn was safe and well tolerated. It also achieved multiple efficacy endpoints when given to 48 ALS patients, including the ability to halt or reverse disease progression over the course of a six-month follow-up.
Subsequently, BrainStorm received U.S. Food and Drug Administration (FDA) permission to start a Phase 3 trial. This will be a randomized, double-blind, placebo-controlled multi-dose trial conducted at multiple sites in the United States and Israel. Patient enrollment is expected to begin later this year.
“Following our end of Phase 2 meeting with the FDA, we are making final preparations to begin a Phase 3 clinical trial, which, when successful, will position us to bring to ALS patients and their families a much-needed and innovative ALS disease modifying therapy,” BrainStorm President and CEO Chaim Lebovits said in a press release.
The company plans to use cryopreservation to enable repeated doses of autologous NurOwn from a single bone marrow aspirate.
The FDA has already accepted key elements of the Phase 3 program to support a Biologic License Application (BLA) for NurOwn, which is a necessary step in commercializing the product.
Separately, BrainStorm announced plans to partner with City of Hope’s Center for Biomedicine and Genetics in the Los Angeles area to produce clinical supplies needed for its Phase 3 study. That study also involves Israel’s Tel Aviv Sourasky Medical Center.
Kim Persaud
Please I will like to part of the phase 3 treatment. Please let me know how I can make this happen.
Thank you.
José Lopes, PhD
Dear Kim, thank you for your comment. I would recommend that you talk with your clinician about the possibility of taking part in the phase 3 study. He/she will certainly be able to help you more efficiently than we are.
Karen
When you say to talk to your clinician are you referring to in the USA, can a person from USA be considered for treatment in Israel under compassionate use?
José Lopes, PhD
Dear Karen, the original information from BrainStorm states that the phase 3 trial will be a multi-center study, performed in both US and Israel. Thank you.
Jose otavio
quero participar da fase 3 de pesquisas
como devo proceder.
jose figueira
[email protected]
José Lopes, PhD
Dear José,
The phase 3 trial will be done in Israel and the US. We are a news service and we don't have more information beyond what you read in the article and what BrainStorm provides. I would suggest you discuss that possibility with your clinician. Thank you for your message.
david joki
What is the procedure to get on a trial volunteer list? Do we need to have a treating physician contact someone from this agency? Thanks.
José Lopes, PhD
Dear David, yes, I would suggest you to ask a physician to help you with that process. Thank you.
Steve Heyworth
ALS patients with a ALSRS less than or equal to 35 should be aloud to take drugs/treatment that have gone though a phase 2 clinical trial. We are running out of time! Endaravone is another good example.
Debbie Wilson
Yes we are running out of time!!!! My family has the FALS.....what can we do to expedite this????
Ed Mueller MD
I agree. The phase 2 trial shows excellent tolerability with no to minimal side effects. Looking at the efficacy in the phase 2 study showed excellent efficacy with some patients even improving. I think it is unethical to deny patients with a fatal disease a treatment that has been shown to be well tolerated and could significantly improve their quality of life and longevity.
Bev
Can a Canadian participate in the US study?
José Lopes, PhD
Dear Bev, thank you for your question. The information we have is all in the article and in my reply to Karen. I would recommend discussing the possibility of participating in the clinical trial with a clinician. Thank you!
Bev
Karen is US citizen. Would a Canadian citizen be accepted in the US study. If not, will there be a trial in Canada soon. I will refer to my clinician when I know the answer.
José Lopes, PhD
Dear Bev, we are a news service and we usually don't have more information beyond what companies provide in their press releases, their website or the clinical trials pages. This trial will be performed in the US and Israel, and we don't have any indication of a similar study in Canada. Regarding acceptance of non-US citizens, that would depend on the eligibility criteria, which are not available yet. Thank you.
Bev
Thank you for clearing that up and reporting the good news! Hopefully phase 3 won't take too long.
José Lopes, PhD
You're welcome!
Becky
Right to try. Shouldn't even be a question about this with the FDA. Their bureaucracy is allowing folks to die.
diana custer
I so agree this is ridiculous the right to try we r dying anyway
Cynthia Dixon
I agree...although we all realize trials are necessary, this beyond horrible disease is deadly enough that it should be given extra speed with patients accessing it. I feel once it's into phase 3, it should no longer be a placebo trial. They've established it's safe by then. We are so close to a treatment. Please, help push this through.
Ed Mueller MD
Agree! The FDA is allowing people to die when we have a phase 2 study showing excellent tolerability and remarkable efficacy with some patients even improving. They should at a minimum allow people to sign a waiver to get the treatment.
Steve Cliff
Are there any plans for a U.K. Trial ?
José Lopes, PhD
Dear Steve, the information we have is all in the article and in my reply to Karen. Thank you!
Nim
We would like to know about this trial as well, my husband has just been diaagnosed last week with als so I really somehow something has to happen
Emma Getliffe
Hi I would also like to be considered for the Phase 3 trial. I will talk to my specialist here in France 4th May and see what he says.
John Tallis
Amazing what you can find..
I hope all the research comes through soon for us. Had lots of dogs over last 40yrs and wish I could get out and about walking. A son doing the London Marathon on Sunday and got 3k for MND
Regina Parker
Can you tell me what the criteria is to be included in the phase 3 trial? i would like to take part but gather I will need to get my neurologist to make the application.
José Lopes, PhD
Dear Regina, the information on eligibility criteria is not available yet. Thank you.
Azzar
Can you please tell me when the phase 3 trials will be completed? I just turned 33 and still holding onto hope I can survive this. Thanks.
José Lopes, PhD
Dear Azzar, we don't have that information yet. We will definitely report the phase 3 trial results once they are published. Thank you.
Chris Gargaro
My wife would like to be considered a a participant for the study. She was recently diagnosed ALS earlier this month. How does she get on the list of candidates?
José Lopes, PhD
Dear Chris, I would suggest discussing that possibility with your wife's clinician. We are a news service, so I am sure he/she would be far better-qualified to provide you with that information. Thank you.
HappyERdoc
IMHO, everyone interested should contact Brainstorm and their congressperson! There is power in numbers!
http://www.brainstorm-cell.com/patients-caregivers/patient-access-to-nurown/
"Brainstorm is exploring the possible commencement of a program to make NurOwn® available to a limited number of patients with Amyotrophic Lateral Sclerosis (ALS, also known as motor neuron disease) through a regulatory pathway designated for an orphan drug with a good safety profile and strong efficacy signals. This program would potentially provide patients, who today face an unmet need, with access to this investigational treatment before it receives final marketing approval from the regulatory authorities."
Edward J. Mueller M.D.
Edward J. Mueller MD
What has happened with this initiative. I printed out the notice from the Brainstorm website in June, but have seen no further information on this. I think it is unethical to deny this therapy to patients when the efficacy shown in the phase 2 trial has already been presented at a major medical meeting and disseminated via a webinar. Time is critical in this disease.
Cynthia Dixon
I totally agree. We are intelligent people here, but enough is enough. Unfortunately, this entire journey has made me feel less competent with the medical system. This is such a horrible disease. How can we make a difference?
Michelle Duboff
Both my dad and his brother have been diagnosed with hereditary ALS, if there's any way for them to participate in any trials we would greatly appreciate it.
Edward J. Mueller M.D.
see my comment above.
Steve Cliff
This is the only real treat of hope that I've seen in the three years since I've been diagnosed. I can't get through to Brainstorm. I'm guessing they are inundated with calls of interest.
John LeVan
My husband has been diagnosed with ALS in April of this year and it is moving rather fast started in his right arm and now is over his entire body. It has affected his speech and the breathing. I know that if we have to wait for the stem cell research to be completed he will not be here. What has he got to lose by being a candidate for a stem cell program. It is a fast down hill spiral. We are US citizen but are willing to do anything, at this rate he will not be around long. Please consider him...
Dan and Cindy Wilkinson
My wife has been diagnosed with early onset Als. we have tried Liberation procedure, desferral and other chelation therapy with some improvement. We are presently trying a ketogenic diet. Recently her symptoms have worsened and we would do whatever it takes to be involved in the stem cell study including paying for all expenses.
Ed Mueller MD
One of the problems with the phase 3 study is it is a double blind placebo controlled study. Plus the patients cannot take Radicava, a drug that can slow progression by up to 30%. Thus patients may get placebo and cannot take Radicava so they may not get any therapy for a year. That is why the FDA needs to approve NurOwn as an orphan drug based upon the phase 2 efficacy, NOW!!!
Debbie Wilson
I agree with you Ed!!! What can we do to expedite this???
Edward J. Mueller M.D.
I am so frustrated knowing there is a treatment out there (NurOwn stem cell therapy) that has been shown to be efficacious in halting disease progression in a significant number of patients and patients whose symptoms have even improved, but we are unable to access this therapy. I don't understand why all neurologists that treat ALS patients don't contact the FDA in the US and the Ministry of Health in Israel to petition them to grant the therapy orphan drug status in the US and grant a hospital exemption to the therapy in Israel. Many patients diagnosed with ALS today do not have the time to wait for the results of a phase III study to be completed. At least allow patients to sign a waiver releasing Brainstorm from any liability should efficacy not be achieved so these patients can receive the NurOwn therapy.