- BrainStorm Cell Therapeutics will test its adult stem cells, derived from patients’ bone marrow tissue, in a phase 2 trial at two Massachusetts sites and one site in Minnesota; the Boston site is now open to recruitment.
- BrainStorm recently announced encouraging interim results from a phase 2a trial of NurOwn cells and an earlier, smaller trial, both of which were conducted outside the U.S.
- So far, the NurOwn cells appear safe and well tolerated when injected into muscle tissue or spinal fluid; and in the phase 2a trial, more participants have stabilized or improved during the after-treatment period than did so during the before-treatment period.
Israeli biotechnology company BrainStorm Cell Therapeutics has enrolled the first participant in a phase 2, U.S.-based trial of adult stem cell transplantation to treat amyotrophic lateral sclerosis (ALS).
The company announced the opening of its first U.S. trial of its NurOwn stem cells in a June 6, 2014, press release. The trial has now opened at Massachusetts General Hospital in Boston and is being overseen by principal investigator neurologist Merit Cudkowicz, who directs the MDA/ALS Center at that institution. (The trial itself, however, has not received MDA funding.)
Additional sites slated to open at the University of Massachusetts Memorial Hospital in Worcester, Mass., overseen by neurologist Robert Brown, who directs the MDA/ALS Center there, and the Mayo Clinic in Rochester, Minn., overseen by neurologist Anthony Windebank.
Encouraging interim results from phase 2a trial in Israel
The opening of the U.S. trial comes on the heels of BrainStorm’s June 2, 2014, announcement of “positive safety and preliminary efficacy [effectiveness]” for its NurOwn cells based on interim results from 10 of 14 ALS patients participating in the company’s phase 2a study, being conducted at Hadassah Medical Center in Jerusalem, and an earlier, completed study that included six patients.
In the two studies, said principal investigator Dimitrios Karussis, no treatment-related serious adverse events were seen.
In the phase 2a study, three months after receiving NurOwn stem cell injections into muscle tissue, spinal fluid, or both, 63 percent of patients showed stabilization or improvement in neurological function as measured by the ALS Functional Rating Scale-Revised, a standard measurement of motor function in ALS. In contrast, in the three months before starting treatment, only 29 percent of participants showed stabilization of function, with 71 percent showing a decline.
The before-and-after treatment difference was considered statistically significant, Karussis said, adding that he was “very encouraged” with the results of these trials, for which the complete analysis will help with the new U.S. study.
About NurOwn cells
BrainStorm’s NurOwn cells come from bone marrow cells taken from each trial participant and converted in the laboratory to stem cells destined to mature into providers of support and protection in the nervous system.
Treatment is via re-injection of the patient’s own cells, which is expected to reduce unwanted responses to them by the immune system.
About the US study
A U.S.-based, phase 2 study of NurOwn cells in 48 adults with ALS has opened at Massachusetts General Hospital in Boston and will eventually open in Worcester, Mass., and Rochester, Minn.
Participants will be randomly assigned to receive either an injection of NurOwn cells into a muscle and into the spinal fluid or a placebo injection into both tissues. The study will last approximately nine months.
The investigators will assess safety (number of patients experiencing adverse events), as well as change in the ALS Functional Rating Scale and in respiratory function.
- be 18-75 years old;
- be a citizen or permanent resident of the United States;
- have a possible, probable or definite diagnosis of ALS;
- have experienced symptom onset within two years prior to study entry;
- have a score of at least 65 percent of normal on a respiratory measurement called slow vital capacity (SVC);
- have been on a stable dose of riluzole or no riluzole for at least 30 days prior to enrollment; and
- meet other study criteria.
Participants must not:
- have received prior stem cell therapy of any kind;
- have a history of an immune system disorder, leukemia, lymphoma or related disorders, or have been treated with whole-body irradiation;
- have a history of cancer within the five years prior to study entry;
- have taken any medication to suppress the immune system within four weeks of the first screening visit;
- have experienced a hip fracture;
- have a severe spinal curvature;
- be using noninvasive or invasive ventilation; or
- be using a feeding tube.
(Note: This is not a complete list of study criteria.)
To participate in the study
For the Boston site (recruiting), contact study coordinator Melissa Arnold at firstname.lastname@example.org. For the Worcester, Mass., site (not yet recruiting), contact study coordinator Colin Quinn at email@example.com. And for the Rochester, Minn., site (not yet recruiting), contact coordinator Delana Weis at (507) 284-2676 or firstname.lastname@example.org.
For more information, see Phase 2 Trial of MSC-NTF Cells in Patients with ALS; or enter NCT02017912 in the search box at ClinicalTrials.gov.