ALS Drug Discovery Platform – To make ALS treatable

Amyotrophic Lateral Sclerosis (ALS) is a serious and intractable disease that causes a gradual loss of motor function while leaving thinking and sensations intact. Research has shown that ALS patients’ motor neurons die, but the underlying cause is still unknown. Studies using ALS animal models have shown that abnormalities in neuromuscular junctions (NMJ) are observed from the early stages of the disease. This suggests that the motor nerves are stripped from the skeletal muscle, resulting in the loss of muscle control. Even though NMJ abnormalities are observed from the very early stages of ALS, patients are not diagnosed until symptoms grow severe.

Understand the pathology of ALS at the NMJ is essential for discovering new treatments. Jiksak Bioengineering focuses on discovering drug targets that will strengthen or promote synaptic growth and maintenance, which we believe will lead to new treatments for ALS.

Using our engineered synaptogenic beads, we recently conducted high throughput screening of FDA-approved compounds and successfully identified targets that promote synapse formation. We offer this technology to our partners through collaboration or contract research for ALS drug discovery.

Images of immunostaining of muscle tissue from ALS model mouse.
At healthy sites, motor axons (green) connect to ACH receptor clusters in muscle (red), generating the NMJ (A3, yellow). In contrast, at pathological sites, the motor nerve terminal is detached from the muscle (A1 and A2).