The Mission, Goal, and Challenges

GABRIELE BARTOLI BRAIN TUMOR RESEARCH LABORATORY

All of the monies being raised by the foundation are being donated to the Bartoli Brain Tumor Research Laboratory at Columbia University Medical Center where Dr. Jeffrey Bruce and his colleagues are working on developing a vaccine that will prevent the growth of brain tumors. Veronica Ronan does not take any salary whatsoever for her efforts to raise money for this most worthy cause.

The Challenge

Few diseases strike more fear in a loved one than the discovery of a brain tumor with its threat to life and neurological well-being. This disease respects no boundaries, often affecting children and adults in the prime of their life, extracting an unforgiving burden on families and friends. Brain tumors are the second leading cause of cancer death in the pediatric population and studies at Columbia have also documented an increase in the elderly.

Despite intensive therapy with conventional treatments including surgery, radiation and chemotherapy, the prognosis remains bleak with virtually no cures and only palliative responses. A 21st century approach is needed to develop a cure and to improve the quality of life for those affected. An unprecedented alliance of basic science and clinical medicine by the best and most respected researchers is being assembled at Columbia to accomplish this lofty goal.

Role and Mission

Why have a Laboratory?

The fundamental goal of the Bartoli Brain Tumor Research Laboratory is to improve the treatment of patients with brain tumors. This research effort provides the structure to support and facilitate interdisciplinary programs of diverse basic laboratory research and clinical investigation of brain tumors through collaborative projects uniting laboratory scientists and clinical investigators. By creating a laboratory under the auspices of an active clinical department in collaboration with diverse interdisciplinary basic laboratory research programs, an environment exists to expedite the incorporation of promising laboratory projects into clinical applications. Additionally, the laboratory provides leadership, education and training in brain tumor research for scientists, clinical investigators, students and health professionals at various levels of training.

Current Site

The Bartoli Brain Tumor Research Laboratory was created in the late 1980's through the vision of its director, Dr. Jeffrey Bruce, and Dr. James McMurtry, Professor Neurosurgery, who raised the initial funds with famed opera singer, Cecilia Bartoli to create the laboratory. Since then the laboratory has grown exponentially to its rightful place as one of the country's premiere laboratories for the practical translational application of promising new treatments for brain tumors. This contribution has been documented by numerous publications, successful competition for NIH and private foundation support as well as recognition with research awards from the American Brain Tumor Association, the National Brain Tumor Foundation, American Cancer Society, American Association of Neurological Surgeons and the Congress of Neurological Surgeons.

The Neurological Institute is the oldest and largest of its kind in the western hemisphere with a long tradition in clinical and bench research on a wide spectrum of neurological disorders. Over 400 patients per year undergo surgery and/or adjuvant treatment for brain tumors at Columbia, making it one of the busiest institutions for brain tumor treatment in the United States, and providing a vast, constant supply of pathological and clinical data for research purposes. The presence of many notable researchers and a strong tradition of cancer research through the Columbia Cancer Center has led to strong collaborative clinician/scientist interactions to investigate important aspects of brain tumors involving basic tumor biology, genetic markers, and novel forms of molecular biologic and genetic therapy.

Current Programs

Novel drug delivery system using high flow microinfusion (convection enhanced drug delivery)

The laboratory is conducting an NIH-sponsored Phase I trial of intratumoral Topotecan for patients with recurrent malignant gliomas. Because of the complex nature of this anti-tumor drug which prevents it from crossing the blood brain barrier without undue systemic toxicity, it must be infused directly into the tumor rather than systemically.

The drug delivery technique involves a positive pressure intratumoral infusion known as "convection enhanced delivery" via a catheter placed in the tumor. Under stereotactic guidance, patients undergo surgical placement of two intratumoral catheters which are then attached to a microinfusion pump. The topotecan is slowly infused over a four-day period, after which the catheters are removed.

Correlative MRI is performed daily to monitor treatment response and delivery parameters. The end result is that much higher levels of drug can be delivered to these brain tumors without systemic side effects. Eight patients have been treated so far, and they are being monitored on a long-term basis for safety, efficacy and quality of life. Additional new anti-tumor drugs are currently being tested in the laboratory for future patient use.

Development of Immunotherapeutic Program

Through interprogrammatic collaboration with investigators in the Cancer Center, plans are made to translate preclinical immunological investigations into clinical trials for malignant glioma. Current studies are focused on characterization of glioma immunological profile, discovery of unique tumor antigens, development of vaccines and evaluation of patient peripheral lymphocytes. Cancer vaccines have the potential to provide an effective and nontoxic approach being highly selective for tumor cells while sparing normal brain cells. Plans are underway to advance the laboratory studies for eventual use in human clinical trials.

Future Plans

With the success of the current laboratory endeavors, the major emphasis will focus on completion of the preliminary studies necessary to permit approval for human use. The laboratory is working towards the goal of establishing an independent center for brain tumor research. Funding from the NIH and industry-sponsored projects along with the generosity of private benefactors will soon make this a reality. Future priorities include expansion of existing laboratory space, recruitment of scientists in cell biology and immunology and improvement of the clinical research infrastructure by recruitment of nursing and data management coordinators.