About Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis, or ALS, is a disease of the nerve cells in the brain and spinal cord that control voluntary muscle movement. ALS is an orphan condition in the United States (U.S.) and European Union (EU), and approximately 6,400 people in the U.S. are diagnosed with the condition each year. The incidence of ALS is two per 100,000 people, and it is estimated that more than 20,000 Americans may be living with ALS at any given time. In ALS, nerve cells (neurons) waste away or die, and can no longer send messages to muscles. This eventually leads to muscle weakening, twitching, and an inability to move the arms, legs, and body. The condition slowly gets worse. When the muscles in the chest area stop working, it becomes hard or impossible to breathe.
NSI-566 as a Potential Treatment
NSI-566 is under development as a potential treatment for ALS by providing cells designed to nurture and protect the patients' remaining motor neurons, and possibly repair some motor neurons which have not yet died but which are diseased. Seneca Bio received orphan designation by the FDA for NSI-566 in ALS. NSI-566 was evaluated in Phase 1 and Phase 2 safety studies in 30 patients. The data showed that the intraspinal transplantation of the cells was safe and well tolerated. Subjects from both the Phase 1 and Phase 2 studies continue to be monitored for long-term follow-up evaluations. New data recently developed from the P1/P2 study regarding insights into potential for efficacy.