Amyotrophic Lateral Sclerosis (ALS)

Ceregene is applying its neurotrophic factor and gene delivery technology to develop a novel and effective treatment for ALS. This treatment is intended to restore lost motor control and delay further progression in the disease. Roughly 30,000 people in the U.S. suffer with ALS, with about 5,000 new cases reported every year. ALS strikes sporadically in 90 to 95% of cases; the remaining ten percent of cases are hereditary. The cause of ALS has proved challenging, since a variety of factors seem to contribute to this disease. However, the death of motor neurons is a common factor in this disease and cell death may arise from excitotoxicity, accumulation of toxic proteins, and/or abnormal axonal transport. There is no truly effective treatment for these patients and a slow, tragic death typically occurs within three to seven years following diagnosis.

Ceregene’s ALS Treatment Approach

Although relatively rare, ALS earns special attention because of its catastrophic consequences. Patients experience muscular wasting beginning with weakness, progressing to loss of motor function, and nearly always leading to death. However, mental function remains intact in these patients until the very latter stages of this disease. Ceregene is studying the effects of different neurotrophic factors in models of ALS, including insulin-like growth factor-1 (IGF-1) and neurturin (NTN). Both neurotrophic factors are known to help maintain normal motor function, while restoring lost function and preventing further degeneration in models of motor disease, such as ALS. Ceregene has initiated a research and development program, using its gene delivery technology, to identify a product for ALS that will provide significant support of the degenerating motor neurons in the spinal cord with appropriate neurotrophic factors. Following selection of a lead product that has a number of desired characteristics for supporting motor neurons, and following appropriate safety testing, Ceregene hopes to initiate clinical trials in ALS patients

Ceregene’s approach to treating ALS using neurotrophic factors through its proprietary gene delivery technology is also being applied to restore function and prevent disease progression in Parkinson’s disease, Alzheimer’s disease, and several ocular diseases that cause blindness.