Administration of sNN0029 (VEGF) using an implanted drug delivery system for the treatment of patients with Amyotrophic Lateral Sclerosis

A drug-delivery system implanted under the skin is being tested to pump a naturally occurring protein directly into the brains of people with amyotrophic lateral sclerosis (ALS), a fatal neurodegenerative disease that kills motor neurons controlling breathing, swallowing, and limb movement. ALS affects 200,000–300,000 people worldwide, and roughly 90% of patients die within five years of diagnosis. The protein, VEGF, shows disturbed expression in the central nervous system of ALS patients and has been shown to promote motor neuron survival in rodent models. The Swedish company NeuroNova AB has already completed a three-month phase I/II safety and tolerability study of this approach. The Wellcome Trust is now funding a phase I/II trial to evaluate the safety and efficacy of higher doses of the drug, called sNN0029. If the trial succeeds, it could provide a new treatment option for a disease that currently has few effective therapies. Even a modest slowing of disease progression would meaningfully extend the time patients can breathe independently, swallow, and move their limbs. The implanted delivery system itself—a pump placed under the skin—could also serve as a platform for delivering other protein-based therapies directly to the brain, potentially benefiting patients with other neurological conditions.