Monday, 6 January 2014

Rare combination of myasthenia and ALS treated with mesenchymal stem cell therapy

Mesenchymal Stem Cell
BrainStorm Cell Therapeutics Inc. a developer of adult stem cell technologies for neurodegenerative diseases announced today the upcoming publication of a case report on an individual patient treated with the company’s NurOwn™ cell therapy in "Muscle & Nerve" (John Wiley & Sons).

 The report, which looked at one patient diagnosed with both Myasthenia Gravis (MG) and Amyotrophic Lateral Sclerosis (ALS) showed significant improvements in both cognitive and motor function following initial and repeat treatments with NurOwn.

The paper describes the rare case of a 75-year old man diagnosed with MG and ALS who was treated with NurOwn cells on a compassionate basis.

 One month after receiving both intrathecal and intramuscular injections of the cells, the patient demonstrated significant improvement in cognition, speech, and muscle power. Initial therapy led to improvement in the ALS Functional Rating Scale (ALSFRS-R), respiratory forced vital capacity (FVC) and cognitive function. Following therapy, the patient’s ability to speak improved from dysarthria to being able to deliver a speech clearly to an audience.

At 6 months post-transplantation, due to progression of weakness and deterioration in cognition, a repeat injection of Brainstorm's NurOwn, was performed. Again, improvements in ALSFRS-R and respiratory forced vital capacity (FVC) were observed following second treatment. Treatment with NurOwn appeared safe and well tolerated; the patient did not experience any serious adverse events after either course of treatment.

The results suggest that repeated therapy may maintain and enhance the clinical benefits of NurOwn cell therapy in neurological diseases. 

The authors stated that "the outcome of the second transplantation was even more remarkable."  They noted that laboratory and clinical improvements of the magnitude observed are "highly unusual in the natural course of ALS." They speculated that "repeated transplantation might be needed to maintain and enhance the clinical benefits of stem cell therapies," and that "the improvement in many functional parameters suggest that the motor nerve function may have been more significantly affected by the treatment than MG."

"This report is very promising and this unique case provides insights into the possible effects of repeat treatments of MSC-NTF cell transplantation on ALS. This publication is an important milestone for BrainStorm as this is the first human data on NurOwn to be published in a highly respected scientific journal. While our PI already reported positive and promising saftey and efficacy data from our Phase I/II clinical trial, we look forward to finalizing our ongoing Phase IIa clinical trial data and thus anounce the combined data. Together, these data will include a total of 26 patients with ALS." said Chaim Lebovits, BrainStorm's President.

NurOwn™ is a proprietary, technology that induces autologous bone marrow-derived mesenchymal stem cells (MSCs) to secrete Neurotropic Factors (NTF). The NurOwn treatment approach is to transplant these “MSC-NTF” cells back into the patient at or near the site of damage, in the spine and/or muscles.

References
http://www.brainstorm-cell.com
- Petrou P, Argov Z, Lennon VA, Gotkine M, Kassis I, Vaknin-Dembinsky A, Ben-Hur T, Offen D, Abramsky O, Melamed E, & Karussis D (2013). Rare combination of myasthenia and ALS, responsive to MSC-NTF stem cell therapy. Muscle & nerve PMID: 24327321

1 comment:

  1. ALS victims have begged the FDA chair to allow for compassionate care treatments given that these patients are going to die if nothing is done, and many may die before these myriad trial studies are concluded. If the patients are willing to sign a waiver absolving the clinics from any legal consequences, what's the harm. Furthermore, the compassionate care treatments might provide additional insights for the researchers.

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