Daniel Offen, head of the neurology laboratory at Tel-Aviv University, Israel, received an MDA grant totaling $359,700 for research into a combined cell and gene therapy approach for ALS (amyotrophic lateral sclerosis, or Lou Gehrig's disease).
Offen's research team will engineer progenitor cells, a type of immature cell that forms muscle, to express various combinations of neurotrophic factors (proteins that support motor neuron health and which previously have been reported to be beneficial in rodent models of ALS). The investigators will inject mixtures of progenitor cells expressing different combinations of the neurotrophic factors into muscles in the SOD1 research mouse model of ALS and then monitor the course of the disease.
Behavioral, physical and biochemical survival indicators will be observed in order to analyze the effects of the stable activity of different growth factors on disease progression, extension of life span and quality of life in ALS. The experiments with the most promising results will then be duplicated using immature human muscle cells.
It's hoped the work will lead to a new cell and gene therapy strategy for treatment of human ALS.
"Past MDA support has enabled us to develop several approaches to isolate and propagate myogenic cells in cultures, some of which will be used in the present investigation," Offen said. "Our current MDA award will enable us to conduct the proposed project and hopefully to contribute to the treatment of ALS patients."
Funding for this MDA grant began August 1, 2010.