Children’s Memorial Making Meaningful Progress

Generous donations are hard at work at Children’s Memorial Hospital, a world-famous institution. A dedicated research team, under the direction of Dr. Stewart Goldman, Medical Director of Neuro Oncology, is actively treating children with brain stem gliomas using a novel regimen. It combines the drug Thalidomide with standard radiation and carboplatin chemotherapy in an effort to kill cancer cells and increase glioma survival rates.

According to Dr. Goldman, “More than twenty patients are enrolled in our study in institutions across the country. We have made enough progress using the Thalidomide regimen to be confident in reporting initial results at a meeting of the International Society of Pediatric Neuro Oncology held in London in June of 2002.” One of the goals in making this presentation was to entice other institutions to participate in this study. “Ideally, I would like forty to sixty patients enrolled to increase the validity of our results.”

Two Years Free And Counting
Dr. Goldman and his team have encouraging initial data to report. “The news I would like to focus on with regard to this study is the fact that we have one child who is now more than two years out with no signs of cancer progression. I have to be honest,” he notes, “I do not think we should be on the ten o’clock news yet. It is one child, but we are thankful and this is very hopeful especially when you consider the fact that 96% of children with brain stem gliomas lose their lives within two years of diagnosis. To be progression free at this point is very, very rare. We remain cautious in our optimism, however..”

More Encouraging News
This study has other auspicious reasons to be optimistic. Dr. Goldman reports the development of a follow-up study for children with this devastating disease. The study uses Thalidomide VP 16 randomized to Thalidomide VP 16 and a COX-2 inhibitor. A master at making the complex understandable, Dr. Goldman explains that, “These inhibitors have been shown to be important in inducing ‘apoptosis,’ which is actually ‘program cell death.’ Instead of killing cells by other techniques, program cell death is kind of like telling the cell to shut down and die.”

In addition, Dr. Goldman focuses on one more research area that is innovative and promising, especially where quality of life for patients is concerned. “We are always aware,” he says, “of the need to balance toxicity with potential benefits. The goal is to minimize the toxicity of the treatment to the children.” Given this caveat, Dr. Goldman expresses excitement over a new technique his team is investigating called, “Convexion Enhanced Chemotherapy.”

Dr. Goldman states, “We have taken brain tumor cells and have been able to grow them in the laboratory. Once this is accomplished, it allows us to use the Convexion technique to infuse chemotherapy directly into the tumor. The advantage here is that we are attacking the tumor directly with small amounts of chemotherapy while avoiding toxicity to the whole body.“

“This could represent a new therapeutic option for our young patients with regard to treatment and quality of life” he states. “We are excited about the possibilities here. We need to work further on enhancing techniques and we need specialized expertise. We are hoping to be able to add a physician scientist to the team in the near future to work with us in the lab on refining Convexion methodology.”

In all, the update from Dr. Goldman provided good news on a number of issues, especially the report of the child who has shown no signs of cancer progression for more than two years. Concludes the medical director, “In my heart of hearts, I believe using Thalidomide with the protocol discussed is a step forward. Granted, it is not a home run, but it is definitely progress.”