Trial Information

A Phase I Study of Topotecan by Intracerebral Clysis for the Treatment of Recurrent Primary Malignant Brain Tumors

Clinical efficacy with chemotherapy has been discouraging for malignant brain tumors, mostly
because of side effects and delivery limitations. Because they are locally invasive and
rarely metastasize, malignant gliomas have features that make them uniquely amenable to new
strategies of regional drug delivery. Intracerebral clysis (convection-enhanced delivery) is
a novel drug delivery strategy that uses a microinfusion pump to establish a pressure
gradient in the brain via implanted catheters. The pressure gradient produces convective
forces that distribute a therapeutic agent throughout the tumor and surrounding brain
tissue.

Non-invasive MRI methods of monitoring drug distribution and treatment response have been
developed to maximize the clinical applications and minimize complications associated with
treatment risks.

Study participants will be taken to the operating room to have 2 catheters surgically placed
into their tumor and surrounding tumor bed. These catheters will then be connected to small
infusion pumps which will slowly infuse topotecan continuously over 4-5 days. Patients will
have daily MRI scans while in the hospital. Upon completion of the experimental treatment,
patients will be discharged and will be followed up in the outpatient clinic.