Science

Autologous Cellular Therapy

Autologous cellular therapy has recently emerged as a credible and practical treatment option for cancer and other highly debilitating diseases.

BrainStorm is focused on developing clinical-stage autologous cellular therapy as a potentially transformative approach to treating neurodegenerative diseases.

BrainStorm has developed a targeted, innovative, proprietary and validated autologous cellular technology platform (NurOwn®) for the treatment of neurodegenerative diseases.

MSC-NTF Cells

Autologous MSC-NTF cells represent a promising therapeutic candidate by targeting disease pathways important in neurodegenerative disorders.

Autologous MSC-NTF cells are produced from the patient’s own bone marrow-derived MSCs that have been differentiated in culture. 

A patient’s own MSCs are harvested and differentiated to secrete high levels of NTFs using a proprietary technology. 
The differentiated MSCs, known as MSC-NTF cells, are then harvested and prepared for injection into the patient. The MSC-NTF cells are not genetically modified.

Autologous MSC-NTF cells represent an innovative cellular therapy approach that can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression.
BEFORE DIFFERENTIATION: MSCs
After Differentiation: MSC-NTF Cells

Source: BrainStorm, Data on file. Detection of GDNF expression in MSC and MSC-NTF cells by immunofluorescence.

NTF Levels before and after differentation
Autologous MSC-NTF cells secrete a unique profile of bioactive molecules, including NTFs, microRNA and cytokines
Play Video
Following intrathecal administration, the autologous MSC-NTF cells may activate neuroprotective and immunomodulatory pathways
Play Video

MSC-NTF Cell Production

BrainStorm has pioneered production of autologous MSC-NTF cells. We have developed proprietary methods to engineer, produce, and purify autologous MSC-NTF cells at a scale and quality necessary to bring MSC-NTF therapeutics to patients with debilitating neurodegenerative diseases.

Each treatment consists of a ready-for-injection syringe containing 100-125 x 106 freshly-harvested autologous MSC-NTF cells in a volume of 4 mL. The MSC-NTF cells are autologous and therefore unlikely to induce an adverse immune response.

BrainStorm has entered into agreements with Dana-Farber Cancer Institute (Dana-Farber) in Boston, Massachusetts and the City of Hope National Medical Center in Duarte, California to provide clean room facilities for production of autologous MSC-NTF cells.

PATENTS

BrainStorm has a robust intellectual property portfolio.

We hold rights to clinical development and commercialization of the NurOwn® technology platform through an exclusive, worldwide licensing agreement.

RECENT BRAINSTORM PUBLICATIONS AND PRESENTATIONS

December, 2018

A Systematic Review of Enrichment Strategies for Current Clinical Trials in ALS

Presented at the International Symposium on ALS/MND
October, 2018

MicroRNA Changes in the NurOwn® Phase 2 ALS Randomized Clinical Trial: Relationship to Neuroprotection and Innate Immunity

Presented at the Annual NEALS Conference
April, 2018

Modulation of CSF miRNAs in ALS Phase 2 Study Participants Treated With MSC-NTF Cells (NurOwn®)

Presented at the Annual AAN Meeting
April, 2018

NurOwn® Phase 2 ALS Trial: ALSFRS-R Improvement is Reflected in Subscale Domains

Presented at the Annual AAN Meeting
November, 2017

miRNA profiling of NurOwn®: mesenchymal stem cells secreting neurotrophic factors

Stem Cell Research & Therapy
January, 2016

Safety and clinical effects of mesenchymal stem cells secreting neurotrophic factor transplantation in patients with amyotrophic lateral sclerosis

Published in JAMA Neurology
BrainStorm’s MSC-NTF cell therapy is investigational and not FDA approved.