TS-000208 — Ninety percent of cases of amyotrophic lateral sclerosis (ALS) are sporadic and lack a familial association, but the etiology of sporadic ALS remains largely unknown. The laboratory of Dr. Brian Kaspar at Nationwide Children’s Hospital has discovered that overexpression of the HLA-F MHC class I molecule in motor neurons is protective against ALS. Further, they have identified a pharmaceutical composition that increases the expression of HLA-F in motor neurons and would serve as a treatment option for patients with both sporadic and familial ALS.

TS-000149 — ALS, commonly referred to as Lou Gehrig’s disease, is characterized by selective, premature degeneration and death of motor neurons (MNs) in the motor cortex, brain stem and spinal cord. Studies have demonstrated that not only MNs but also non-neuronal cell types including microglia and astrocytes play a significant role in disease onset and progression. Researchers at Nationwide Children’s Hospital have identified a previously undescribed disease mechanism in which astrocytes use killing pathways typically ascribed to the innate immune system, and can use these mechanisms as targets for therapeutic intervention in ALS.