Dr Anders Haegerstrand
Newron Sweden AB
Administration of VEGF (sNN0029) using an implanted drug delivery system for adose escalation study in patients with Amyotrophic Lateral Sclerosis.
The Wellcome Trust
Motor neurone diseases
Vascular Endothelial Growth Factor (VEGF) has been shown to be of relevance for Amyotrophic Lateral Sclerosis, a devastating neurodegenerative disease with an app. 90 % mortality 5 years after diagnosis. VEGF represents a scientifically unique treatment opportunity for ALS based on data in state of the art rodent models of ALS as well as supportive clinical data describing a disturbed VEGF expression in the CNS of ALS patients. NeuroNova AB has made it possible to initiate clinical testing of VEGF by means of intra-cerebroventricular (icv) delivery of the clinical grade formulation of recombinant human VEGF165 (sNN0029) to patients with ALS. Direct delivery of VEGF to the brain will for the first time reveal the therapeutic potential of a drug that activates anti-apoptotic genes in motor neurons, which are the specific neurons at risk in ALS. NeuroNova is in the process of finalising (Q2 2011) a first in human safety trial investigating 3 different doses. So far no drug related safety problems have been identified. The specific goal of the proposed study is to identify the Maximally Tolerated Dose (MTD) in a step-wise approach, which will allow for a subsequent Phase II/III study to investigate efficacy of the drug.