Trial Represents First-Ever Phase 0 for Newly Diagnosed Patients with Glioblastoma. The Ivy Brain Tumor Center’s Phase 0 clinical trials program is the largest of its kind in the world and enables personalized care in a fraction of the time and cost associated with traditional drug development.
Shwetal Mehta, PhD, chief operating officer at the Ivy Brain Tumor Center (Image Courtesy of Ivy Brain Tumor Center)
Phoenix, AZ, Dec. 16, 2020 (GLOBE NEWSWIRE) — The Ivy Brain Tumor Center at Barrow Neurological Institute today announced the opening of a Phase 0 clinical trial for patients with newly diagnosed and recurrent glioblastoma (GBM) to evaluate pamiparib, an investigational small molecule PARP inhibitor from BeiGene. The Ivy Center’s novel study approach will assess pamiparib’s ability to penetrate the blood-brain barrier, one of the most significant challenges in the mission to treat brain cancer.
This trial adds to the Ivy Center’s already extensive portfolio of Phase 0 clinical trials for patients with glioblastoma and other aggressive brain tumors but marks the first-ever to enroll newly diagnosed GBM patients, in addition to recurrent GBM patients.
The current standard of care for patients with newly diagnosed glioblastoma consists of maximal surgical resection, followed by radiation and a chemotherapy drug called temozolomide (TMZ) to slow the growth of microscopic tumor cells that remain after surgery.
“The trial itself, marking the first time newly diagnosed glioblastoma patients will be enrolled in a Phase 0 clinical trial, is a major milestone for the brain tumor community and the Ivy Brain Tumor Center,” commented Catherine Ivy, founder and board president of the Ben & Catherine Ivy Foundation. “What we learn from this trial represents yet another pivotal step forward in the work to find a cure for glioblastoma patients.”
Worldwide, it has been 20 years since a new drug approval has provided glioblastoma patients with a survival benefit.
“While temozolomide can extend a GBM patient’s survivability to 15-18 months, it is not curative and shows no benefit for patients with an unmethylated MGMT promoter. Radiation, however, is the only modality that has been proven to work in all patients. If we find that pamiparib is capable of reaching the tumor at sufficient levels, and is subsequently developed and approved in this indication, it will add to our arsenal of radiosensitizer drugs to make radiotherapy for patients with glioblastoma even more effective,” said Shwetal Mehta, Ph.D., chief operating officer and deputy director of the Ivy Brain Tumor Center.
Patients enrolled in this clinical trial will take the study drug for five days leading up to a scheduled operation to remove their tumor. Within days of the operation, the Ivy Center’s team will determine the effects of the investigational treatment on the tumor. Patients with positive results may advance to an expansion phase of the study, which combines therapeutic dosing of pamiparib with fractionated radiotherapy. If pamiparib does not show evidence of sufficient tumor penetration, patients can pivot to another therapy or clinical trial without losing time or receiving ineffective treatment.
“There are two underlying problems with identifying new, effective treatment options for brain cancer. First, the blood-brain barrier prevents most new drugs from ever reaching the tumor. Second, the molecular effects of new drugs are difficult to predict using laboratory animals. Ivy Phase 0 clinical trials address both of these obstacles and accelerate our understanding of treatment in each individual patient,” said Nader Sanai, M.D., director of the Ivy Brain Tumor Center.
“The Ivy Brain Tumor Center’s commitment to their patients, combined with the speed and precision of their clinical trial administration, make them an ideal research partner for this ground-breaking clinical trial,” said Yong (Ben) Ben, M.D., Chief Medical Officer, Immuno-Oncology at BeiGene. “This trial is designed to enable us to understand more about the extent to which pamiparib can cross the blood-brain barrier, and we are hopeful that it could lead to further development, and if approved, bring a much-needed new treatment to the brain tumor community.”
To learn more about this Phase 0 clinical trial, including eligibility criteria, visit: https://clinicaltrials.gov/ct2/show/NCT04614909.
About Ivy Brain Tumor Center
Ivy Brain Tumor Center at the Barrow Neurological Institute in Phoenix, AZ is a non-profit translational research program that employs a bold, early-phase clinical trials strategy to identify new treatments for aggressive brain tumors, including glioblastoma. The Ivy Center’s Phase 0 clinical trials program is the largest of its kind in the world and enables personalized care in a fraction of the time and cost associated with traditional drug development. Unlike conventional clinical trials focusing on single drugs, its accelerated trials program tests therapeutic combinations matched to individual patients. Learn more at IvyBrainTumorCenter.org. Follow the Ivy Brain Tumor Center on Facebook, Instagram, Twitter and LinkedIn.
Pamiparib (BGB-290) is an investigational inhibitor of PARP1 and PARP2 which has demonstrated pharmacological properties such as brain penetration and PARP-DNA complex trapping in preclinical models. Discovered by BeiGene scientists, pamiparib is currently in global clinical development as a monotherapy or in combination with other agents for a variety of solid tumor malignancies. Clinical trials of pamiparib also include a Phase 1b/2 trial (NCT03150862) in combination with radiation therapy and/or temozolomide in patients with first-line or recurrent/refractory glioblastoma. To date, more than 1,200 patients have been enrolled in clinical trials of pamiparib.
A New Drug Application (NDA) for pamiparib for patients with ovarian cancer has been accepted and granted priority review by Center for Drug Evaluation (CDE) of the China National Medical Products Administration (NMPA) for the treatment of patients with deleterious or suspected deleterious germline BRCA-mutated advanced ovarian, fallopian tube, or primary peritoneal cancer who have been treated with two or more lines of chemotherapy.
Ivy Brain Tumor Center