Research Initiatives

The University of Rochester Program for Brain Tumors and Spinal Tumors is committed to making available current treatment options as well as new clinical trial opportunities for neuro-oncology patients. Our researchers work side by side with clinicians to interpret the results of clinical trials in order to understand why some therapies perform better than others. In addition, we recognize that future progress in treating these diseases will be made through the ongoing efforts of both basic and clinical researchers.

To improve patient outcomes, new scientific discoveries must be translated into clinical applications. This process is commonly referred to as a “bench-to-bedside” approach, in which basic molecular discoveries are developed into new patient treatments. However, this process actually requires constant input and discussion from both parties – scientists and clinicians – working together. Basic researchers provide physicians with new tools while, in turn, clinicians provide important observations about particular disease characteristics and responses that then further stimulate scientific inquiry.

Like normal organs, tumors are dependent on their blood supply in order to survive and grow. Many scientists believe that blocking the growth of new blood vessels in the tumor (a process referred to as “angiogenesis”) can inhibit the growth of a tumor, essentially through starvation. Our researchers are working to increase the number of targets that could be considered for anti-angiogenesis therapy. Most work in this area has focused on small molecules found in or excreted by the tumor cells themselves. Many of our studies focus on the blood vessel cells (endothelial cells) themselves, which, unlike tumor cells, rarely mutate and so might be less likely to become resistant to therapy and are also more easily reached through the bloodstream. Early efforts have identified a collection of molecules that were found on the endothelial cells in malignant brain tumors at much higher levels than in normal brain blood vessels. Experiments aimed at interfering with the function of some of these molecules have already shown promise in preventing blood vessel growth. Current efforts are aimed at expanding this work and identifying additional targets and specific drugs which can be exploited to interrupt blood vessel and, more importantly, tumor growth.

In a related effort, our researchers are attempting to better understand the changes in the blood-brain barrier (BBB) that are associated with fluid accumulation and brain swelling (edema) in neuro-oncology patients. Normal brain tissue is shielded from the rest of the body by the BBB. This barrier is composed of very tight blood vessels that prevent most substances from entering the brain. Brain tumors have a leaky BBB – this feature can be used to identify tumors on MRI scans. We have identified specific molecules that appear to be associated with the leaky, abnormal vessels while the normal blood vessels with intact BBB produce these molecules at very low levels or not at all. Inhibiting the function of these molecules may help control or prevent disruption of the BBB and limit cerebral edema in brain tumor patients, as well as patients suffering from stroke or traumatic brain injury.

In the not-so-distant future, physicians may be able to examine the molecular “fingerprint” of a patient’s tumor and customize a therapeutic course of action. In order to reach such a goal, researchers need to be able to first study the effects of new drugs on a large number and wide range of tumor specimens. To that end, we maintain a tissue repository (tumor bank) containing a wide range of clinical specimens, which we make available to researchers in order to further their studies. Collections such as this, supported in part through private donation, are critical to a successful, collaborative neuro-oncology research program.

The University of Rochester is home to world-class clinicians and researchers studying these and many additional aspects of neuroscience medicine. Their sustained efforts in these areas underlie our constant goal to improve patient care, outcomes and quality of life.

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