The Rory David Deutsch Brain Tumor Research Program is headed by Drs. Stewart Goldman, Maryanne Marymont, Tadanori Tomita, and John Kalapurakal (Northwestern University) at CMH. This multi-institutional research team received FDA approval in November 1999 to begin Thalidomide chemotherapy studies. Currently three of the six participating institutions, CMH, Johns-Hopkins University and Los Angeles Children’s Hospital (USC) are also poised to complete the final Institutional Review Board approvals.

“At this point, our Thalidomide work is 100% for brain stem gliomas, and eventually we'll follow up with a Thalidomide study for other brain tumors as well,” said Dr. Stewart Goldman, the neuro-oncologist and head of the clinical research teams. "Our first patient was enrolled and we'll soon be starting the new Thalidomide therapy treatments with this child.”

After receiving more than $250,000 from the Rory David Deutsch Foundation last April, the The Rory David Deutsch Brain Tumor Research Program team at CMH was able to define the study's parameters and fully develop its two-part strategy involving both the Thalidomide drug therapies and new radiation technologies. With the necessary funds, both studies are now moving ahead on separate, but concurrent schedules.

Dr. Goldman is heartened by the fact that so many new pediatric brain tumor treatment opportunities are emerging as a result of the multi-dimensional and multi-institutional studies being funded in whole or part by the Rory David Deutsch Foundation and Children’s Memorial Hospital Foundation. Dr. Goldman states that he is not aware of any other institutions that are promoting research specifically dedicated to studying brain stem gliomas.

"This is all because of Rory. He was my patient. He cemented our commitment to helping kids with these lethal tumors,” said Dr. Goldman. "The brain stem is like the pentium chip of the brain. You can't operate on them, so we have to study the chemotherapeutic and radiation opportunities out there. This is where our passion, commitment and focus is.”

The second part of the team's research strategy involves studying the benefits of a new radiation technology called the Photon Radiosurgery System (PRS). Often called the "wand,” the PRS is a pencil-sized irradiation tool that can be inserted right into a tumor to send highly controlled radiation doses.

Tremendous support and lobbying from the Rory David Deutsch Foundation helped make the purchase of the cutting-edge PRS (the "wand") possible. The staggering cost notwithstanding, the wand's sophisticated technology may allow surgeons, for the first time, to treat deadly brain tumors more effectively, directly and from the inside. Typically, pediatric oncologists must apply available radiation technology, which is powerful external radiation beams that penetrate through a child's skull and healthy brain tissue as well as the tumor and tumor bed.

Unfortunately, these external radiation treatments can damage healthy tissues, and often are ineffective at treating the fast-growing brain stem gliomas – which claim the lives of 96 percent of children they strike. According to CMH researchers, the powerful new wand therapy may be the newest weapon in the fight to deter and hopefully destroy these cancerous brain tumors.

The prime benefit of the wand is that it directs the radiation from the inside out in a narrow and defined range of tissue.

"This allows us in the operating room, to visualize the tumor, to take this pencil-sized instrument and place it inside the tumor bed, and then apply the radiation,” said Dr. Goldman. "The wand only penetrates tissue in millimeter-sized areas. Through a robotic arm, and using information we're getting from computers and MRIs [magnetic resonance images], we take the wand, map out the tumor area and then treat the tumor.”

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How CMH is Coordinating Efforts

In the first year of the CMH wand study, according to Dr. Maryanne Marymont, director of pediatric radiation oncology at CMH, the team will evaluate how normal tissues tolerate the wand treatments, as well as how well the wand treatments destroy tumor cells.

"For about a year we'll be doing laboratory models and testing the Photon Radiosurgery wand before using it in the patient setting,” said Dr. Marymont. "During the year we'll be assessing the toxicity and effectiveness of varying doses of the highly localized radiation from the wand.”

After year one of the parallel chemotherapy and radiation studies, according to Dr. Goldman, the teams will begin coordinating the studies by joining Thalidomide treatments with the wand technology in patient applications.

"For now we need to catalog the actions of both separately to establish what's safe with the photon arm, what dosages work best. We know the safety advantages established in adults, so now we're establishing the safety in children,” said Goldman. "In year two, with the other scientists and physicians from the other participating institutions, we'll add the chemotherapy agents concurrently.”

To expedite their local and worldwide research efforts, the team's researchers are continuing to search for a full-time scientist who will fulfill a multi-year commitment to the studies. Supported initially by funding from the Rory David Deutsch Brain Tumor Research Program, this scientist will oversee the lab research that the team of neuro-scientists is conducting in the coming years.

This glucose-based drug treatment, called Oral 2-Deoxyglucose, is an agent that interrupts the metabolic or growth pattern in normal cells. A study conducted in India established the treatment's effectiveness in adults. Now researchers are keen to discover its benefits in destroying pediatric brain tumors and brain stem gliomas in particular.

"We have completed a concept proposal that has been scientifically accepted, and now we're looking for a pharmaceutical company to manufacture the compound for us,” said Dr. Goldman.

These and other treatments are also being studied on behalf of the Rory David Deutsch Brain Tumor Research Program, and by the Children’s Memorial Hospital researchers. For instance, with approximately $60,000 from the Rory David Deutsch Foundation, new equipment was purchased for an exciting study being formed and conducted by Dr. John Kalapurakal at Northwestern University. According to Dr. Goldman, the study is looking to find ways to optimize traditional radiation treatments.

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