A new clinical trial is now underway at Weill Cornell Medicine, testing intra-arterial chemotherapy for choroid plexus carcinoma. The first patient in the world to undergo this advanced procedure was safely treated by Dr. Mark Souweidane, Dr. Jared Knopman, and Dr. Y. Pierre Gobin in May 2023.
In this trial (Intra-Arterial Chemotherapy for Newly Diagnosed, Residual, or Recurrent Atypical Choroid Plexus Papilloma and Choroid Plexus Carcinoma Prior to Second-Look Surgery), patients receive single doses of the drugs melphalan, carboplatin, and topotecan delivered through one or more arteries in the brain, directly to the site of the tumor. This combination of drugs has already been used successfully to treat retinoblastoma, a devastating tumor that once required the removal of the affected eye. It is hoped that this same combination will be a safe and feasible treatment for patients with newly diagnosed, residual, or recurrent atypical choroid plexus papilloma and choroid plexus carcinoma.
The parents of the first patient agreed to share his story, which was featured at a recent NewYork-Presbyterian event. See clips below:
The primary outcome goal for this Phase I study is establishing the safety of the procedure, and patients will be monitored for a year for any adverse effects. Patients will also be monitored for hoped-for decrease in tumor size and vascularity (since reducing blood flow to a tumor starves it of nutrients and helps prevent growth). Treatment is followed by second-look surgery to resect any remaining tumor.
Intra-arterial chemotherapy (IAC) is an advanced procedure administered by interventional neurosurgeons or neuroradiologists, who specialize in image-guided treatments for brain and spine conditions. By administering chemotherapy drugs directly to a tumor, IAC delivers high concentrations of cancer-fighting therapy without the toxicity of body-wide intravenous drug delivery (more about interventional neurosurgery).
“This is a very promising trial, and I have a sense of guarded optimism about its potential,” says Dr. Souweidane, the principal investigator on the clinical trial. “Choroid plexus carcinoma has excellent survival rates in patients who undergo complete tumor resection, but it is significantly less in those who only have partial resections. If we can reduce tumor volume and vascularity before surgery, we may be able to achieve total resection in more patients.”
Dr. Souweidane, co-director of the Children’s Brain Tumor Project at Weill Cornell Medicine, has dedicated his career to treating children with brain tumors and other neurosurgical conditions, and is internationally renowned for his clinical trial of convection-enhanced delivery of chemotherapy for diffuse intrinsic pontine glioma (DIPG). With this new trial, he now offers new hope to children and families facing the diagnosis of choroid plexus tumors.
For more information, contact Dr. Souweidane at 212-746-2363 or Colleen Sanders at 917-734-8787.
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