According to a report in BioPharma Dive, Brainstorm Therapeutics, a New York biotechnology company, said that while its NurOwn therapy for amyotrophic lateral sclerosis (ALS) appeared to have a positive effect, it did not significantly outperform the trial’s placebo. In the end, the placebo group performed significantly better than expected.

The fact that the NurOwn treatment did not meet the primary endpoint of the trial represents the newest disappointment in ALS research.

Many of the neurodegenerative diseases have baffled the most skilled researchers as well as drug companies. To date, treatment consists of only two medicines and even those have huge limitations.

A Path Forward

Brainstorm’s CEO Chaim Lebovits believes that since NurOwn did perform favorably on its own, a path forward still exists for this therapy.

Mark Bedall definitely agrees. Mark participated in the NurOwn phase 3 trial and contends that NurOwn saved his life.

At his diagnosis, Mark was given three to five years to live but after entering a clinical trial studying NurOwn and its ability to slow the progression of ALS, his mobility has returned to its normal level. He hopes to bring awareness to others about ALS and NurOwn.

About the Trial

The trial, NCT03280056, is a double-blind phase 3 trial where neither the doctors nor the patients, sponsors, or others involved in the trial, know which patients are receiving a placebo or the actual drug.

The stem cells used in the trial are harvested (collected) from patients’ own bone marrow. Then the cells are engineered to secrete molecules that grow neurons intended to replace damaged ALS cells. At that point, they are re-injected into the patient via their own spinal fluid. The goal is to keep the motor neurons viable and for a longer period of time.

Brainstorm had estimated that thirty-five percent of patients treated with NurOwn and fifteen percent of patients treated with a placebo would meet its primary goal. This translates to a 1.25-point monthly improvement using a well-known scale that measures ALS patients’ daily functions.

Brainstorm’s estimate met its mark. It showed that patients treated with NurOwn achieved the anticipated thirty-five percent response rate.

However, the placebo group showed an unexpected twenty-eight percent response rate, causing trial failure.

One reason for the high placebo response was suggested by Ralph Kern, Brainstorm’s CMO. He said that the results could have been influenced by the participants who had severe ALS. This number was higher than other ALS studies and higher than expected in the NurOwn trial. He explained that these patients do not respond well to treatment and are more difficult to evaluate.

About ALS

The onset of ALS begins with weakness in a limb, muscle twitching, or indistinct speech. The upper and lower neurons are affected. Symptoms of this fatal disease eventually worsen to the degree that the patient is unable to speak, move, eat, or breathe independently.

About NurOwn

NurOwn is an autologous (patient’s own cells) cellular therapeutic technology developed by Brainstorm Cell Therapeutics. Brainstorm is a leading developer of adult stem cell therapies. It is based in Israel.

NurOwn uses mesenchymal stem cells (MSCs) isolated from a patient’s bone marrow. MSCs can produce various types of cells, such as bone, cartilage, and fat.

Can ALS be Cured?

There is no cure for ALS, although certain drugs are available that have slightly extended overall survival and provide a better quality of life. The current prognosis from the onset of ALS is two to four years.

However, technology in stem cell research has provided researchers with new tools in their efforts to find a cure for the disorder.

The Importance of Stem Cells

Stem cells do not replace diseased motor neurons. However, transplanted stem cells emit neurotrophic substances that generate a neuroprotective environment and decrease the deterioration of motor neurons.

There are two reasons that researchers are concentrating on stem cells:

• An ample supply of cells is needed for test purposes. A form of stem cells called Induced Pluripotent Stem Cell (IPSCs) bears a resemblance to the ALS affected neurons. Researchers are able to create an enormous amount of IPSCs from just a small skin sample taken from ALS patients. This enables the researchers to conduct tests using identical cells indefinitely.
• Stem cells are able to replace damaged cells of any kind or anywhere in the body. Stem cell transplants protect healthy neurons.

Looking Forward

CEO Lebovits reports that Brainstorm is currently conducting talks with the FDA. Although long-term studies are needed, at this juncture researchers anticipate that stem cell therapy could still have the potential to delay the progression of ALS.