Amyotrophic Lateral Sclerosis (Lou Gehrig’s disease)
Ceregene is applying its neurotrophic factor and gene delivery technology to develop a novel and effective treatment for amyotrophic lateral sclerosis (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 80 to 85 percent of cases; the remaining cases are inherited, with over 140 mutations thus far linked to the cause of the disease. Finding the cause of ALS has proven challenging, since a variety of factors seem to contribute to this disease. However, the death of motor neurons is a common factor, leading to insidious and progressive loss of all motor function and secondary muscle wasting. While voluntary movement is initially made difficult and then impossible, eventually even involuntary motor functions involving swallowing and breathing become critically impaired. There is no genuinely effective treatment for ALS. Indeed, every person diagnosed with ALS faces a slow, tragic path of degeneration that typically leads to death within three to seven years following diagnosis.
Ceregene's ALS Treatment Approach
Although relatively rare, ALS earns special attention because of the catastrophic consequences it imposes on each person affected, as well as their families and loved ones. We are currently conducting nonclinical research to improve the efficacy and safety of CERE-135 (AAV-insulin-like growth factor). Insulin-like growth factor (IGF) is known to help maintain normal motor function, while also restoring lost function and preventing further degeneration in models of motor disease, such as ALS. Using our gene delivery technology, we are testing the ability of sustained, targeted expression of IGF-1 to provide significant neurotrophic support for the degenerating motor neurons in the spinal cord and the muscles they innervate. Following completion of initial proof of principle efficacy studies, we intend to launch a formal development program to establish adequate safety of CERE-135 to support clinical testing in ALS patients.
Similar to our approach to treating ALS using neurotrophic factors, we are also applying our proprietary gene delivery technology to restore function and prevent disease progression in Parkinson’s disease, Alzheimer’s disease, Huntington’s disease and several ocular diseases that cause blindness.