Israel's BrainStorm says stem cell drug benefits most patients in ALS trial

C

Claire

New member
Israel's BrainStorm says stem cell drug benefits most patients in ALS trial
Published January 05, 2015
Reuters


TEL AVIV – Israel's BrainStorm Cell Therapeutics said final results from a clinical trial of its adult stem cell treatment in amyotrophic lateral sclerosis (ALS) were positive, with most patients showing a slowing in the disease's progression.

According to the ALS Association, 5,600 people in the United States are diagnosed each year with the neurodegenerative disease, which has severely disabled British physicist Stephen Hawking.

A single dose of the stem cell treatment called NurOwn was administered in a mid-stage phase 2a trial in 14 patients with ALS, also known as Lou Gehrig's Disease, at Hadassah Medical Center in Jerusalem.

"Nearly all subjects in this study experienced clinical benefit from treatment with NurOwn," the company said on Monday.

Of the 12 patients with three or more months of follow-up, 92 percent experienced an improvement in disease progression. NurOwn slowed the progression of ALS using two different parameters and had a strong effect on the rate of decline in lung function, BrainStorm said.

Shares in the company, which is also developing stem cell treatments for multiple sclerosis and Parkinson's disease, have nearly doubled in the past two sessions on Nasdaq in anticipation of the trial's results. The stock has nearly tripled in the past year, closing at $7.50 on Friday.

In October the U.S. Food and Drug Administration designated NurOwn as a "fast-track" product to treat ALS.

"We observed not just a highly meaningful slowing of ALS progression on two different parameters ... but subjects with prolonged stabilization and even improvements in function, and this was achieved with just a single dose of NurOwn," said BrainStorm Chief Executive Tony Fiorino.

BrainStorm, which is also conducting clinical trials at three sites in the United States, plans to move to a study in the next few months to see if the results can be amplified with repeated doses.
 
K

kenschatz

New member
Barbara,
The BrainStorm/Nurown seems much less invasive, and uses the patients own stem cells. They are recruiting I think - double blind placebo. I live in AZ, where they passed a "right to try" proposition also. Sure would like to figure out a way to try the BrainStorm.
Thanks
 
B

barbara

Pioneer Founding member
Petitioning BrainStorm Cell Therapeutics, Ltd.

https://t.co/SNZImLPw2f

Petitioning BrainStorm Cell Therapeutics, Ltd.

This petition will be delivered to:
BrainStorm Cell Therapeutics, Ltd.
Grant expanded access (compassionate care) of NurOwn autologous adult stem cell therapy to treat Cheryl Sweeney and other late-stage ALS patients.

Elise Lamont
Saint Paul, MN
Cheryl Sweeney, 62 years old of Duluth, Minnesota, was diagnosed with Amyotrophic Lateral Sclerosis (ALS) in 2011 at the Mayo Clinic in Rochester, Minnesota. ALS, more commonly known as Lou Gehrig’s disease, is a type of motor neuron disease in which the body attacks motor neurons, or the nerve cells found in the brain, brain stem and spinal cord resulting in the loss of control of muscle movement. Patients progressively worsen losing the ability to walk, move their arms, eat food or swallow, speak or breathe on their own, eventually resulting in death.

Cheryl is a devoted mother and wife, a first-rate paralegal and compassionate leader in her church and community. She serves as a second mother to many of the neighborhood children. Prior to her first symptoms, Cheryl was an active outdoors enthusiast and loved camping, hiking, kayaking, and climbing. Recently, Cheryl has been placed on a ventilator. She is otherwise healthy and is in full possession of her mental faculties. Cheryl has educated others about ALS in her church and community. Her tenacity for life and her love for her family have garnered support that stretches beyond her small neighborhood to reach the halls of the United States Congress, Minnesota State Senate and House of Representatives. Her wit, compassion and vivacious spirit bring a smile to everyone who is fortunate enough to meet her.

There is no cure for ALS. Treatment focuses on symptom management. Most ALS patients live on average 2 to 5 years after diagnosis. However there is still hope for Cheryl and the nearly 5,000 U.S. citizens who are diagnosed with this incurable disease each year and their families. A new stem cell therapy developed by BrainStorm Cell Therapeutics, Ltd., NurOwn, uses stem cells from the patient’s own body to treat, slow and reverse the symptoms of ALS. In October 2014, the U.S. Federal Food and Drug Administration (FDA) announced they were fast tracking NurOwn based on the results from Phase I/IIa clinical trials in Israel and its success in treating six compassionate care cases in Italy and Israel. Two U.S. sites are now in the process of recruiting 32 patients for double-blinded, Phase 2 clinical trials: Massachusetts General Hospital (Boston, MA) and the Mayo Clinic (Rochester, MN) and a Phase 3,

Cheryl does not meet the qualification criteria to participate in the clinical trials, however she hopes to gain access to this potentially life-saving treatment through compassionate care, also known as emergency expanded access outside of clinical trials. Previously, BrainStorm Cell Therapeutics, Ltd. has granted access to NurOwn stem cell transplantation under expanded access, including Rabbi Rafael Shmuelevitz, an 81-year old patient and prominent leader from Jerusalem who was diagnosed at the Mayo Clinic (Rochester, MN) with ALS and myasthenia gravis and was treated successfully in Israel.

Like Cheryl, these patients were designated as advanced ALS. However, unlike these patients, she does not suffer from other diseases (co-morbidities). We are asking BrainStorm Cell Therapeutics, Ltd. to grant expanded access of NurOwn to Cheryl and others as they generously did for those patients in Israel.

Group-Level Expanded Access Program a Win-Win: Specifically we are asking BrainStorm Cell Therapeutics, Ltd., to consider offering a Group-Level Expanded Access Program (EAP) for late-stage ALS patients in the United States. We acknowledge that a major concern for pharmaceutical and therapeutics companies and the treating hospitals with respect to offering expanded access to patients is recuperation of costs. Working with a third party non-profit organization such as the ALS Emergency Treatment Fund, we believe will help resolve this issue by opening up grant and other funding opportunities as well as an option for patient cost-sharing in order to provide potentially life-saving treatment for those in end-stage ALS. Moreover there is much to be gained from studying stem-cell therapy in late-stage ALS patients. Limited data suggests that late-stage ALS patients could derive benefit from NurOwn therapy (Petrou et al., 2014). Clinical trials focus on ALS patients in the earlier stages of their disease therefore unless we include late-stage ALS patients in the investigations we will not know whether or not stem cell therapies will be efficacious throughout the course of the disease. Another interesting research question that could be investigated is whether or not there is a difference in treatment response between those with known ALS genetic markers, those suspected to have inherited a genetic link and those with sporadic ALS.

Sign the Petition, Save Cheryl and Other ALS Patients: We hope that BrainStorm Cell Therapeutics, Ltd. will consider expanded access of NurOwn for Cheryl and other late-stage ALS patients. Cheryl meets the FDA criteria for compassionate use and has the full support of her doctor, family, friends, church, state representatives and community. Without treatment now, Cheryl will die. By signing this petition in support of Cheryl, we all will take the stand that no person is beyond the effort to save.

LETTER TO
BrainStorm Cell Therapeutics, Ltd.
Grant expanded access (compassionate care) of NurOwn autologous adult stem cell therapy to treat Cheryl Sweeney and other late-stage ALS patients.
 
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