Human Neural Progenitor Cells Secreting Glial Cell Line-Derived Neurotrophic Factor (CNS10-NPC-GDNF) for the Treatment of Amyotrophic Lateral Sclerosis (IRB no. 42350)
This study focuses on patients who have been diagnosed with amyotrophic lateral sclerosis (ALS), with symptoms present for less than three years. The study is designed to test the investigational use of CNS10-NPC-GDNF cells. Specifically, researchers will evaluate the safety and effectiveness of a drug delivery device, as well as the safety of transplanting cells that have been genetically engineered to produce a growth factor into the spinal cord of ALS patients, to determine if these cells help protect the motor neuron cells of ALS patients. A growth factor is a substance that is required for the stimulation of growth in living cells. The growth factor in the cells used in this study is called glial cell line-derived neurotrophic factor (GDNF).
Financial disclosure: Cedars-Sinai owns the patent application related to the devices being tested in this study. Pablo Avalos, a research scientist in sponsor Clive Svendsen’s laboratory, is named on the patent application related to the device being tested in this study. Therefore, it is possible that he would receive future royalty income under the patent.
Key Inclusion Criteria
- Confirmed diagnosis of ALS
- Duration of symptoms ≤36 months
- Progressive weakness in lower extremities
- At least 18 years old
- Forced vital capacity >60% of predicted normal in supine (lying horizontally, facing up)
Key Exclusion Criteria
- Using invasive ventilatory assistance
- Diagnosis of another active or unstable medical illness that may interfere with study participation
- Individuals with child-bearing capacity who are not willing to practice birth control
Robert Baloh, MD, PhD