Phase 2a clinical trial of BrainStorm stem cell product shows ‘clinical benefit’
Nearly all patients who underwent clinical trial showed benefit.
Nearly all patients who underwent stem cell treatment for amyotrophic lateral sclerosis (ALS) developed by Brain- Storm Cell Therapeutics Inc. and tested in a Phase 2a clinical trial at the Hadassah University Medical Center in Jerusalem’s Ein Kerem “showed clinical benefit,” according to the Petah Tikva- and New York-based company.
The firm said Monday the trial was carried out by senior Hadassah neurologist and ALS expert Prof. Dimitrios Karousis, who has been working on stem cells for incurable diseases for years. Known as Lou Gehrig’s disease and usually killing patients within five years of diagnoses, it famously struck British astrophysicist Prof. Stephen Hawking as a young man, who is in his 70’s and the subject of a new biographical film.
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.
The stem cell technology is called NurOwn, which were given to 14 subjects at the hospital. The study achieved its primary endpoint in demonstrating that NurOwn is safe and well-tolerated at doses up to 2 million cells per kg. when administered intrathecally (IT) – introduced into the space inside a sheath, most commonly the arachnoid membrane of the brain or spinal cord – and 48 million cells administered to the muscle (IM).
Of the 12 subjects with three or more months of follow-up, 92 percent experienced an improvement in disease progression for the three-month period after getting the injection, as measured by ALS Functional Rating Score-Revised or forced vital capacity. A total of 50% had an improvement in the slope of the ALSFRS score, and 67% had an improvement in the slope of the percent-predicted FVC.
NurOwn was shown to slow ALS progression in the study, as indicated by an improving slope of both the mean ALSFRS and mean FVC curves after therapy, the company said. For ALSFRS, the stem cells slowed the rate of progression by 45%, from 1.41 points per month during the run-in period to 0.78 points per month for the three months following treatment, and by 57% to 0.60 per month for the six months following treatment. NurOwn had a similarly strong effect on the rate of decline in lung function – the rate of decline in percent- predicted FVC was reduced by 73%, from an average of 2.60% per month during the run-in period to just 0.70% per month for the three months after treatment, and by 67% to 0.86% monthly for the six months after treatment.
“We are gratified to have the final data from this study and are very encouraged by the results,” commented BrainStorm’s CEO Dr. Tony Fiorino. “These results not only extend our earlier phase 1/2 findings regarding the safety of NurOwn, but also continue to provide a consistent and highly promising picture of NurOwn’s efficacy. In particular, I would highlight that we observed not just a highly meaningful slowing of ALS progression on two different parameters, ALSFRS and FVC, but subjects with prolonged stabilization and even improvements in function, and this was achieved with just a single dose of NurOwn. We are excited to proceed to a multidose study to see if these positive results can be amplified and extended by administering repeated doses.”
Karousis, who was the principal investigator of the trial, said, “This is the second study of NurOwn I have completed in ALS patients, and my excitement for these cells as a possible treatment for ALS continues to grow. I am impressed by the consistency of benefit of IT administration we have seen in both studies, and we saw in this study that almost every subject experienced clinical benefit, either on ALSFRS, FVC or both measures. I believe that if future studies demonstrate a similar magnitude of benefit, NurOwn will become an important treatment option for patients suffering from ALS.”
Nearly all patients who underwent clinical trial showed benefit.
Nearly all patients who underwent stem cell treatment for amyotrophic lateral sclerosis (ALS) developed by Brain- Storm Cell Therapeutics Inc. and tested in a Phase 2a clinical trial at the Hadassah University Medical Center in Jerusalem’s Ein Kerem “showed clinical benefit,” according to the Petah Tikva- and New York-based company.
The firm said Monday the trial was carried out by senior Hadassah neurologist and ALS expert Prof. Dimitrios Karousis, who has been working on stem cells for incurable diseases for years. Known as Lou Gehrig’s disease and usually killing patients within five years of diagnoses, it famously struck British astrophysicist Prof. Stephen Hawking as a young man, who is in his 70’s and the subject of a new biographical film.
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.
The stem cell technology is called NurOwn, which were given to 14 subjects at the hospital. The study achieved its primary endpoint in demonstrating that NurOwn is safe and well-tolerated at doses up to 2 million cells per kg. when administered intrathecally (IT) – introduced into the space inside a sheath, most commonly the arachnoid membrane of the brain or spinal cord – and 48 million cells administered to the muscle (IM).
Of the 12 subjects with three or more months of follow-up, 92 percent experienced an improvement in disease progression for the three-month period after getting the injection, as measured by ALS Functional Rating Score-Revised or forced vital capacity. A total of 50% had an improvement in the slope of the ALSFRS score, and 67% had an improvement in the slope of the percent-predicted FVC.
NurOwn was shown to slow ALS progression in the study, as indicated by an improving slope of both the mean ALSFRS and mean FVC curves after therapy, the company said. For ALSFRS, the stem cells slowed the rate of progression by 45%, from 1.41 points per month during the run-in period to 0.78 points per month for the three months following treatment, and by 57% to 0.60 per month for the six months following treatment. NurOwn had a similarly strong effect on the rate of decline in lung function – the rate of decline in percent- predicted FVC was reduced by 73%, from an average of 2.60% per month during the run-in period to just 0.70% per month for the three months after treatment, and by 67% to 0.86% monthly for the six months after treatment.
“We are gratified to have the final data from this study and are very encouraged by the results,” commented BrainStorm’s CEO Dr. Tony Fiorino. “These results not only extend our earlier phase 1/2 findings regarding the safety of NurOwn, but also continue to provide a consistent and highly promising picture of NurOwn’s efficacy. In particular, I would highlight that we observed not just a highly meaningful slowing of ALS progression on two different parameters, ALSFRS and FVC, but subjects with prolonged stabilization and even improvements in function, and this was achieved with just a single dose of NurOwn. We are excited to proceed to a multidose study to see if these positive results can be amplified and extended by administering repeated doses.”
Karousis, who was the principal investigator of the trial, said, “This is the second study of NurOwn I have completed in ALS patients, and my excitement for these cells as a possible treatment for ALS continues to grow. I am impressed by the consistency of benefit of IT administration we have seen in both studies, and we saw in this study that almost every subject experienced clinical benefit, either on ALSFRS, FVC or both measures. I believe that if future studies demonstrate a similar magnitude of benefit, NurOwn will become an important treatment option for patients suffering from ALS.”