First Pediatric Patient Treated With Aptocine(TM), A Novel Light-Activated Drug, In Neurofibromatosis Study

Main Category: Neurology / Neuroscience
Also Included In: Cancer / Oncology;  Pediatrics / Children's Health;  Clinical Trials / Drug Trials
Article Date: 13 Aug 2009 - 1:00 PST

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Light Sciences Oncology, Inc. (LSO) 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 patients with NF-1, they can create an extremely negative impact on quality of life by causing pain, disfigurement, and functional impairment. Treatment options for PN are sparse: the only known effective therapy is complete surgical removal; however, this is rarely possible. Although there are other medications being tested for PN, most are not expected to cause tumor shrinkage, but rather to slow or stop tumor growth. In contrast, Aptocine has the potential to shrink the PN tumors and improve the quality of life of patients with this disease.

About Aptocine

Aptocine is a water-soluble drug activated by a small, single-use, disposable drug activator included with the drug. The drug activator contains a tiny array of LEDs at the end of a very narrow (only 1.2 mm wide) flexible coated micro-wire. Administering physicians insert the LED array into a tumor using a biopsy-like procedure and follow with the intravenous injection of Aptocine. When the LEDs activate Aptocine, an energized Aptocine molecule results in the production of singlet oxygen, which can kill target tissues with minimal side effects through vascular closure and apoptosis.

About Light Sciences Oncology

Light Sciences Oncology (LSO) is developing Aptocine for solid tumors as well as other indications. LSO has completed treatment of patients in a Phase 3 trial of Aptocine in hepatocellular carcinoma (HCC) and is currently enrolling patients in a Phase 3 trial for metastatic colorectal cancer (MCRC). LSO is also conducting clinical trials of Aptocine in patients with benign prostatic hyperplasia (BPH), or enlargement of the prostate, as well as in neurofibromatosis, and has clinical or preclinical programs in cardiovascular, ophthalmic, and dermatological diseases.

About the Children's Hospital of Philadelphia and Its Neurofibromatosis Program

The Children's Hospital of Philadelphia was founded in 1855 as the nation's first pediatric hospital. Through its long-standing commitment to providing exceptional patient care, training new generations of pediatric healthcare professionals, and pioneering major research initiatives, Children's Hospital has fostered many discoveries that have benefited children worldwide. Its pediatric research program is among the largest in the country, ranking second in National Institutes of Health funding. In addition, its unique family-centered care and public service programs have brought the 430-bed hospital recognition as a leading advocate for children and adolescents.

Aptocine(TM), Litx(TM), and Light Infusion Therapy(TM) are trademarks of Light Sciences Oncology, Inc.

Source: Light Sciences Oncology, Inc