Rare Disease Report

Ralph Kern, M.D., Explains NurOwn as Potential ALS Therapy Moves Through the Pipeline

DECEMBER 08, 2017
Mathew Shanley
This weekend at the International Symposium on ALS/MND in Boston, BrainStorm Cell Therapeutics will be presenting data on their stem cell therapy NurOwn which is currently in development for amyotrophic lateral sclerosis (ALS) and a slew of other neurodegenerative diseases.

The company’s Phase 3 clinical trial is expected to enroll approximately 200 patients and will be conducted at 6 leading ALS clinical sites in the U.S. The first patients were enrolled in mid-October at the Massachusetts General Hospital and University of California Irvine Medical Center in California.

The company expects the study to be fully enrolled by this time next year, and with an additional year of research – 2 years from now – it is expected that there will be topline data.

Rare Disease Report spoke with the company’s Chief Operating Officer and Chief Medical Officer Ralph Kern, M.D. about the potential therapy and why the initiation of the Phase 3 program is so exciting for the ALS community.

RDR: What are BrainStorm’s intentions and goals as they pertain to this Phase 3 of NurOwn?

Kern: We’re an innovative biotechnology company that’s focused on developing cell-based therapies for neurological disease. The unmet need in neurological disease is very great, and that’s primarily because existing treatments offer either symptomatic improvement or modestly slow disease. We are looking at a regenerative medicine approach to neurological disease, with the hope that we can go beyond slowing the disease or providing a therapy that is purely symptomatic. At the end of the day, our goal is that it will translate into benefits for patients in terms of function, ultimately hoping to change the course of the disease.

RDR: What is the technology behind the potential therapy? How is it delivered to the patient?

Kern: Our technology platform is derived from cells that come from the patients themselves, and that’s an important starting point. A patient’s own cells are harvested, they’re purified and processed, and we modify the cells to a cell type that can provide benefit to the diseases that we’re trying to treat. So, cells that are returned to the patients are their own cells that are expanded, modified, and then brought back to deliver a basket of biological molecules that we believe are beneficial to the disease. Just to give a timescale, the technology platform has been in development for 10 years, and this is our seventh year in the ALS clinic.

RDR: Why is it justified for the ALS community to be excited about NurOwn?

Kern: We’re excited for a few reasons: first, in the Phase 2 program, the participants in the study who received a single treatment experienced meaningful clinical improvements. The clinical scale used in ALS is called the ALS Functional Rating Scale and essentially it’s a scale that describes all the things that someone does during the day in 12 different domains; there are 4 points per each subscale, leading to a total score of 48. The way that we measure the outcomes in ALS is to evaluate participants in the run-in period and then after treatment, we measure the score again. We compare before and after changes in ALSFRS-R, and we found in Phase 2 that there was, in a group that was declining at a quicker rate at baseline, an improvement in scores. That’s unusual for this group of ALS patients where, historically, the only observed outcomes have been a continued fast rate of decline. This needs to be confirmed in a larger study, and we plan to study the same group in the Phase 3, and we plan to complete this study as quickly as possible.

RDR: What data specifically is being presented at the International Symposium on ALS/MND?

Kern: We are sharing our biomarker data at ALS/MND in Boston; we have done a lot of research and development on understanding exactly who the cells are, what they do, how they work, and the molecules that they make – even down to the genetic level. We’re able to measure the characteristics of the cells before they’re transplanted and also before and after treatment when the cells are administered by lumbar puncture injection into the spinal fluid. We’ve made interesting observations, both in terms of the molecules that these cells make, but also molecules that indicate progression and state of the disease. That’s why we’re here this week; to show some of the biomarker data at the MND/ALS meeting.

RDR: Why are these biomarkers so important to the future of ALS research?

Kern: The reason that this new information is so important is that the biomarkers may, in the future, tell us which ALS patients might have a greater need for our therapy, may predict disease progression or identify which patients may be expected to have a better response to our therapy. Potentially we may be able to track the biomarkers and adjust treatment accordingly.

These are very exciting times for ALS. We’re testing an advanced cell therapy. We have biomarkers that might help inform that. We’re currently conducting a pivotal Phase 3 program. We hope this will lead to a treatment that ALS patients will be able to access and improve their quality of life.


For more information, refer to clinicaltrials.gov (NCT03280056).

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