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BELLEVUE, Wash., Aug. 12 /PRNewswire/ -- Light Sciences Oncology, Inc. (LSO) today announced the treatment of the first patient in an investigator-sponsored Phase 1 pediatric study of its novel light-activated drug Aptocine(TM) (talaporfin sodium) in neurofibromatosis type 1 (NF-1). Investigators at the Neurofibromatosis/Neuro-Oncology Clinic of Children's Hospital of Philadelphia (CHOP) treated a pediatric patient with NF-1 and plexiform neurofibromas (PN) using Aptocine, which is also in late-stage development for solid tumors. The Neurofibromatosis/Neuro-Oncology Clinic is a program providing care and treatment for children with brain tumors and children with NF-1 and severe and/or life-threatening tumors. Michael Fisher, M.D., at CHOP's Division of Oncology, is the principal investigator of the study.
"A clinical trial in neurofibromatosis is an exciting step forward in the development of Aptocine for patients with a significant unmet medical need outside our primary cancer focus. I am proud and delighted that LSO is now associated with developing a treatment of this condition in children and adults," said LSO's President and CEO, Llew Keltner, M.D., Ph.D.
The goal of the study is to enroll 18-24 patients between 3 and 21 years of age with debilitating, severely disfiguring, life-threatening, or progressive PN tumors that are not surgically resectable, and for which no other standard medical management exists. Complete information on the study is available at ClinicalTrials.gov with the identifier: NCT00716469.
NF-1 is a common, autosomal dominant genetic disorder with an incidence of 1 in 2500 people (> 80,000 in the US); symptoms of NF-1 usually appear at birth and/or during infancy. PN represents one of the most significant complications of NF-1. PN tumors form along peripheral nerves and can become very large. Although PN tumors are not a major cause of mortality in pati
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