A new report shows that autologous therapy with bone marrow-derived mesenchymal stem cells secreting neurotrophic factors may slow progression of amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig’s Disease).
Henry Louis (“Lou”) Gehrig had a stellar career as first baseman for the New York Yankees. In 17 seasons, through the 1920’s and 1930’s, he hit almost 500 home runs. He seemed indefatigable and powerful, nicknamed “The Iron Horse”. That is, until, in the second half of the 1938 season, his abilities sharply deteriorated. In June 1939, he was diagnosed with amyotrophic lateral sclerosis (ALS). Lou Gehrig died two years later.
There is no cure for ALS. The disease is rare, but fatal. It is characterized by the degeneration of motor neurons, resulting in muscles that atrophy and wither away. Its causes are almost entirely unknown. Treatments can only (and barely) slow the progression of the disease – the solitary FDA-approved medication, Riluzole, extends life expectancy of ALS patients by mere months. New approaches to fight the disease are needed. Among these, autologous cell therapy with bone marrow-derived mesenchymal stem cells (MSCs) or neural stem cells may hold the most promise.
Doctors from the Hadassah-Hebrew University Medical Center in Jerusalem, Israel, now published the encouraging results of proof-of-concept clinical trials performed with bone marrow-derived mesenchymal stem cells producing neurotrophic factors (MSC-NTFs).  These special MSCs had been cultured ex vivo in a specifically formulated growth medium and produce neurotrophic factors that promote survival, development and function of neurons.
In their clinical trials, the doctors measured disease progression through the revised “ALS Functional Rating Scale” (ALS-FRS-R), which assesses muscular function, for three months prior to and six months after cell therapy. They also quantified the patients’ forced vital capacity (FVC), that is, the maximum exhalation air volume. In the initial study, 12 ALS patients were injected either intramuscularly (IM) or intrathetically (IT, into the spine) with autologous bone marrow-derived mesenchymal stem cells that produced HTFs. In a follow-up trial, 14 further patients were given MSC-HTFs both IT and IM.
Most encouragingly, the doctors found that the treatment was well tolerated by the patients. In addition, patients that had been given the MSC-HTFs intrathetically experienced a significant reduction in the negative progression of the FVC and the ALS-FRS-R scores. Overall, the study concluded that 87% of the patients that were treated with IT injection of the mesenchymal stem cells experienced a ≥25% improvement in disease progression.
That does not amount to a cure, of course. But these encouraging results were sufficient to warrant a bigger phase 2 clinical trial, which is currently underway, to evaluate the effects of an MSC-HTF therapy approach in more detail. In the absence of a magic bullet to fight ALS, any increase in patients’ survival time and quality of life bears hope until that cure is finally here.
More research is sorely needed, and HemaCare is excited to contribute to such potentially life-saving efforts with many high quality cell products that are derived from bone marrow, including mesenchymal stem cells. Contact us at (877) 397-3087 or explore all our cell products at hemacare.com.
 Petrou P, Gothelf Y, Argov Z, Gotkine M, Levy YS, Kassis I, et al. Safety and Clinical Effects of Mesenchymal Stem Cells Secreting Neurotrophic Factor Transplantation in Patients With Amyotrophic Lateral Sclerosis: Results of Phase 1/2 and 2a Clinical Trials. JAMA Neurol. 2016 Mar 1;73(3):337-44.