A phase 1 study of photodynamic therapy (pdt) with Photofrin® for poor prognosis recurrent/refractory malignant brain tumors

Children’s Wisconsin is currently enrolling patients with relapsed or refractory brain tumors on a Phase I clinical trial, using a photosensitizing agent in combination with tumor resection surgery and intra-operative photodynamic therapy (PDT) in patients with: 

• PNET (primitive neuroectodermal tumor, now known as CNS embryonal tumors)
• Medulloblastoma
• Ependymoma
• High–grade glioma
• Germ cell tumor
• ATRT (atypical teratoid/rhabdoid tumor)

One of our early-phase trial team physicians, Dr. Jeff Knipstein, is the co-principal investigator of this trial. Children’s is currently the only site in the country where this trial is available.

The survival of pediatric patients with recurrent malignant brain tumors is measured in months with few long–term survivors.The methodology utilized by a previous team of PDT researchers has shown impressive survival rates in adults and adolescents with high–grade gliomas with minimal acute or late side effects.We believe the demonstrated efficacy and safety profile of their methods in adults warrants application of this treatment in pediatric patients with recurrent/refractory brain tumors.

Eligible patients will have injection of the photo sensitizing agent, Photofrin^®,followed by surgical resection of the tumor 24 hours later. Activation of residual Photofrin^® in the tumor bed will be performed using an intra–operative laser at a wave length and energy level defined by a previous team of researchers. Patients will receive standard post–operative care and will be closely monitored during the first month for acute toxicities associated with Photofrin^®activation.

Children’s has ranked in 9 specialty areas in the U.S. News & World Report Best Children’s Wisconsins list.