Novel Glioblastoma Trial Begins Immediately

Breakthrough Brain Tumor Clinical Trial Kicks Off a New Paradigm of Glioblastoma Treatment with Seed Funding From PHASE ONE Foundation To Saint John’s Health Center Foundation

SANTA MONICA (April 10, 2018) – Saint John’s Health Center Foundation announced that PHASE ONE Foundation has provided the initial funding for a breakthrough clinical trial for patients with brain cancer. The trial’s principal investigator and author Santosh Kesari, MD, PhD, will conduct the study at Pacific Neuroscience Institute, Saint John’s Cancer Institute and Providence Saint John’s Health Center.

“Glioblastoma is an insidious disease,” says Dr. Kesari, director of neuro-oncology at Pacific Neuroscience Institute and Chair of neurosciences and neurotherapeutics at Saint John’s Cancer Institute. “This is an aggressive and lethal form of brain cancer that carries a very poor prognosis of 14-16 months survival. The tumor generally has a main mass that can be surgically debulked in many cases. However in this cancer type, additional tumor cells extensively invade normal brain tissue and cannot be surgically removed without causing damage.”

Called the Precision Immunotherapy in the Neoadjuvant Setting (PIN) trial, this study provides an alternative immunotherapy option to the current standard of care treatment that patients receive when presenting with a new diagnosis of glioblastoma.

Over the last 30 years, standard of care for newly diagnosed glioblastoma patients has involved surgical removal of the tumor where possible followed by radiation and chemotherapy (temozolomide). While this therapy regimen has afforded limited survival benefit, there is much room for improvement. Expert neurosurgeon, Daniel F. Kelly, MD, PNI director works closely with Dr. Kesari to care for these very sick patients.

“Given the relatively poor outcomes for our glioblastoma patients using current standard of care therapies, it is high time for a fundamentally novel approach. The PIN trial designed by Dr. Kesari is definitely a step in the right direction,” said Dr. Kelly.

The PIN protocol uses immune therapies for difficult-to-treat patients whose immune systems have not yet been compromised by standard radiation and chemotherapy. PHASE ONE Foundation’s investment of $300,000 has allowed the PIN trial to open immediately and begin recruiting patients for the initial phase 1 safety trial.

A Santa Monica-based nonprofit organization, PHASE ONE is dedicated to supporting ground breaking Phase 1 and Phase 2 clinical cancer research, innovative education and treatment programs, and funding forward-thinking leaders in the scientific and medical community.

“When we fund researchers, it’s because they are innovative,” says Stacy Valner, co-founder of the PHASE ONE Foundation and co-chair of the granting committee. “They are thinking differently. They are really trying to tap into areas that haven’t been covered. This is novel and exciting to us because this is a cancer with no cure. Dr. Kesari wants to take these tumors, examine them and biopsy them multiple times. It’s much more cutting edge.”

Phase 1 trials such as the PIN trial offer hope where there is none, says Mieke Neumann, co-chair of the PHASE ONE Foundation granting committee. “Every cure started out as a phase 1 trial. It’s a way to be hopeful and innovative,” she says.

The 3-armed trial will compare standard of care with two immunotherapy drugs that target different immune checkpoint inhibitor pathways to disrupt cancer cell proliferation. Biospecimens including blood and CSF will be collected and analyzed over the course of patient treatment, providing valuable insights into anti-tumor activities of the study drugs and biomarker characteristics of responders.

Current research indicates that temporarily delaying use of standard treatment has little negative effect on overall survival. Patients in the two immunotherapy arms will be monitored for therapeutic efficacy and safety and can remain on study treatment as long as they are benefiting. Mainstream care is always available if needed.

“There are several advantages to initiating the PIN trial here,” said Tiffany Juarez, PhD, program manager for translational research at Saint John’s Cancer Institute. “There is close collaboration between our world-class neurosurgeons and our neuro-oncologist Dr. Kesari. This is essential for managing the logistics of such a trial along with having highly capable and flexible research staff to coordinate efforts. We stay up to date with technological and clinical advances and routinely perform molecular profiling. Over time this will provide a better understanding of the tumor microenvironment and help identify which patients may benefit the most from immunotherapy.”

There are few drugs approved for brain cancer because of hurdles inherent to these kinds of cancer. The difficulty of crossing the blood-brain barrier to get drugs into the brain is one such obstacle, along with specifically targeting infiltrative disease while sparing healthy tissue. “The immunotherapy drugs to be used in the trial have shown a lot of success in other cancer types. This trial can be seen as a platform to test other investigational agents in a setting that gives an early read out of activity,” Dr. Juarez added.

“We are hopeful about this approach which represents a real paradigm shift in the treatment of newly diagnosed glioblastoma patients,” said Dr. Kesari. “The standard treatments using radiation and chemotherapy diminish the patient’s immune system. The benefit of treating the patient before standard therapy should in theory allow the patient to respond to immunotherapy more strongly and fight the cancer more effectively.”

While PHASE ONE Foundation’s support is vital in getting this clinical trial off the ground, more funding is necessary to expand and develop the program.

For more information about opportunities to provide additional resources for this project, contact Mary Byrnes, Director of Development, Saint John’s Cancer Institute at 310-582-7102 or mbyrnes@stjohns.org.