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Amyotrophic Lateral Sclerosis (ALS) is a neurodegenerative disease that destroys the motor neurons of the brain and spinal cord. With no definitive therapy, most patients die within 3-5 years of diagnosis. Extensive research is underway including the use of stem cells to regenerate damaged neurons. Although typically an adult disease, childhood cases are reported. Our patient began showing symptoms at age 6 and was formally diagnosed with the rare juvenile form of ALS at age 9. At consult, she displayed marked spasticity with loss of fine motor control. She could not walk or communicate verbally and required nocturnal ventilatory support. She was G-tube dependent for nutrition. Because of her young age, she was ineligible for clinical studies. After an ethics consult, FDA, IRB, and financial approvals, we followed Deda’s procedure (Cytotherapy 2009:11;18-15).
Methods, Intervention, & Analysis
Autologous, bone marrow cells were harvested from iliac crests and collected by Ficoll density gradient. The following day laminectomy of C1 and partial laminectomy of C2 was performed. A suspension of 6 million cells in 300 microliters was prepared. After a 25 gauge needle was advanced to a depth of 9 – 10 mm into the anterior horn of the cervical spinal cord at a 20 degree angle, 500,000 cells/50uL were injected. Six injections were performed on each side of the midline (12 injections). Each injection was 7 mm apart on the mid-rostral caudal line and 2 mm off midline to each other. Two strips of gel foam each infused with 5 million cells/500uL were placed along the anterior quadrant of the spinal cord prior dural closure. An additional 5 million cells/500uL were injected into the subarachnoid space. The remainder of the cells were injected intravenously (1.2 x 10e8 TNC/kg . There were no postoperative complications.
Findings & Interpretation
Peak response was seen at 6 months with 12 month duration: improved phonation, facial movements, swallowing, and upper extremity control. Nocturnal CPAP use was successfully removed. She developed intermittent spasms/clonus with right upper extremity pain. The patient refused additional ventilatory support and with disease progression, died 25 month later of respiratory failure.
Discussion & Implications
Additional studies are needed to evaluate this therapy.