Encouraging news does not come often. This is why we are pleased to report news of progress, help and hope in the battle against pediatric brain stem gliomas. The welcome news comes from a dedicated research team at the Falk Brain Tumor Center at Children's Memorial Hospital where a new method of treatment has demonstrated positive clinical results.

Dr. Stewart Goldman, Medical Director of Neuro-Oncology, states, "With the support of The Rory David Deutsch Foundation, we have developed and are actively treating children with a novel regime. We are using the medicine, Thalidomide with standard radiation and carboplatin chemotherapy in an attempt to increase the survival rate for children suffering from brain stem gliomas." So far, according to Dr. Goldman and study reports, four of eleven (evaluable) child patients remain progression free. Cancer has not progressed, which is definitely hopeful news, though Dr. Goldman is quick to add cautions and emphasize the need for guarded optimism.

Caution about anything resembling a cure is understandable due to the toughness of the disease and the low survival rate, which is less than one year, for children with these tumors. Dr. Goldman points out that many brilliant medical minds have been trying to unravel this enigma for years, unfortunately with little success. No therapy to date has been able to change the prognosis.

"We've been doing clinical trails," Goldman explains, "and have been aggressive in our attempts to improve results, but our work must also follow treatment standards we have established. Our therapies must respect past outcomes, balance toxicity to potential benefits, minimize the time children are in the clinic and hospital, assure ease of administration and, above all, respect the child undergoing treatment. We want to do everything in our power to cure these children, but with no assurance as to outcome, we don't want the child to remain in the hospital for six months. So it's a balancing act: balancing aggressive treatment while also assuring quality of life."

A Combined One, Two, Three Punch

As Goldman explains, the new therapeutic attack hits cancer on some powerful fronts. First, is treatment with Thaliomide, an antiangiogenic agent that prevents the formation of new blood vessel growth. Antiangiogenic agents are an exciting class of new agents that break ground with traditional therapies by preventing cancer cells from stimulating blood vessel growth and thus getting the oxygen and nutrition they need to grow. "This is great but not enough," adds Goldman. "Once you've got cancer cells against the ropes, you've got to kill them, which is where standard radiation and carboplatin chemotherapy come in. They are classic treatments used to destroy cancer cells."

Another positive punch being added to the fight mix is the fact that, based on the Falk Brain Tumor Center study, five or six other major institutions have expressed interest and have indicated their desire to enroll patients. According to Dr. Goldman, this adds more excitement to the prospect of definitive trial numbers that will lend added credence to the positive results to date. "We'd like to have 40 patients enrolled," he says. He also notes that follow-up studies that are planned at the Falk Center Clinic will further bolster results.

"Meanwhile, we'll take our four of eleven progression free children as a very positive sign. We'll take the good news from this study even though the data is very preliminary and add it to the next, and the next after that. Then we'll need to add one more essential ingredient – a tincture of time, an infusion of waiting until we are sure."