I'm part of a small group that focuses specifically on brain tumors. Our multidisciplinary team includes oncologists, a neurologist, neurosurgeons, a radiation oncologist, social workers and a neuropsychologist, along with skilled nurse practitioners. This is the broad spectrum of expertise that The Children's Hospital of Philadelphia brings to treating its patients with brain tumors.
Treating cancer is a team effort at Children's Hospital, and we consider patients and their families a part of the team. Since our young patients are dependent on their parents for medical decision making, I try to give them a sense of some control over their disease, communicating in an age appropriate manner. Kids do much better, even when facing death, when they are part of the treatment plan and "secrets" are not kept from them.
I do both basic science and clinical research that focuses on developmental therapeutics in tumors of the nervous system. In particular I and my colleagues are looking at signal transduction in neuroblastoma and medulloblastoma. We are evaluating the biological effects of neurotrophin receptor expression on the growth properties of these tumors. Neurotrophins are able to signal certain cells to survive, mature or grow. The Trk family of neurotrophin receptors plays a critical role in these diverse behaviors.
We recently completed a clinical trial that targeted these receptors to inhibit signaling in neuroblastoma. The results are promising that this could be a therapeutic target for high risk neuroblastoma. We also are studying the way that Trk receptor molecules located on the surface of medulloblastoma cells regulate tumor growth and development. These studies may help to identify new therapeutic targets in the Trk signaling pathways.
As part of our approach to identifying new treatments for children with brain tumors, we are members of the multi-institutional Pediatric Brain Tumor Consortium (PBTC) and the Developmental Therapeutics Children's Oncology Group. These groups are involved in early phase clinical trials for relapsing and refractory brain tumors, with a particular focus on testing new, targeted treatments in children with brain tumors.
Our Children's Hospital team has partnered with drug development groups, and we are rapidly identifying drugs that show promise in patients with brain tumors who have no other curable option. There is a sense of urgency in our group -- we don't randomly test drugs but work collaboratively with laboratory scientists and drug development companies on rational, biologically-based targets. The best example of this is a drug for medulloblastoma that was recently rapidly brought to a PBTC clinical trial.
There is huge progress in getting promising drugs to kids with cancer. Through our cooperative group consortia, the pace is much more rapid than in past years, and Children's Hospital is a major part of the effort to help bring new agents to children with brain tumors.
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