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rGBM Phase 2 Clinical Trial Results
June 29, 2023

Trial Results Offer Hope for Improved rGBM Survival Outcomes

by Guest Author

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Expert neuro-oncologist Santosh Kesari, MD, PhD, Director of Neuro-oncology at Pacific Neuroscience Institute and Chair and Professor of the Department of Translational Neurosciences at Saint John’s Cancer Institute, published the results from a phase 2 clinical trial for recurrent glioblastoma (rGBM), offering hope for improved survival outcomes and pushing novel glioblastoma treatment one step closer to FDA approval.

Importance of the Study

Despite considerable efforts over the past 4 decades, outcomes for glioblastoma patients remain poor with no standard of care available for rGBM (recurrent glioblastoma). Approved therapies have shown median survival of only 6–9 months, a 1-year survival rate of 0%–10%, and 12-month PFS (progression-free survival) rate of 2%–10%.

In addition, treatment of rGBM is constrained by the blood-brain barrier, its aggressive and infiltrative nature, and the presence of an immunosuppressive tumor microenvironment. An immunosuppressive tumor microenvironment refers to the conditions and factors within a tumor that suppress the immune response, allowing the tumor to evade immune surveillance and continue growing.

In a phase 2 clinical trial, investigators tested the efficacy of MDNA55 as a treatment for GBM. MDNA55 is an immunotoxin that targets a receptor associated with GBM disease progression. The study demonstrated a meaningful benefit after a single MDNA55 treatment, resulting in ~50% increase in median survival and almost 100% increase in 12-month PFS when compared to approved therapies. This finding presents a promising treatment option for rGBM patients who otherwise rapidly succumb to this disease. Read the full study published by Oxford University Press on behalf of the Society for Neuro-Oncology.

Transforming biomedical research and medicine into promising new treatments used clinically starts here at Pacific Neuroscience Institute and Saint John’s Cancer Institute.

Santosh Kesari, MD, Director of Neuro-oncology, Pacific Neuroscience Institute

What is recurrent glioblastoma?

Brain tumor graphic

Recurrent glioblastoma (rGBM) is the reappearance or progression of glioblastoma (GBM), a type of malignant brain tumor, after initial treatment. GBM is the most aggressive and common type of primary brain tumor in adults. Despite advancements in treatment, including surgery, radiation therapy, and chemotherapy, GBM recurs in most cases. Resection is not widely adopted nor regarded as effective since most patients (~70%–75%) are not candidates for repeat gross total resection at recurrence. Treatment options are very limited with no universally held standard of care, resulting in a large unmet need for this patient population.

Adapted from the June 2023 issue of Neuro-oncology.

Pacific Brain Tumor Center

At PNI’s Pacific Brain Tumor Center, we incorporate leading-edge technology such as MRI fiber tractography, functional MRI , surgical navigation, and awake craniotomy protocols, with proven surgical experience, to make glioma surgery safer, less invasive, and more effective. We have a large experience in the comprehensive personalized care of all types of gliomas including low-grade astrocytomas, oligodendrogliomas, anaplastic astrocytomas, and glioblastomas.

Our doctors and specialist have years of experience treating gliomas and other brain tumors that are complex in their diagnosis and treatment and warrant a team approach of experienced specialists to achieve optimal outcomes. From diagnosis to treatment, as well as education and support, we are here for you. 

Dr. Kesari treats patients at Pacific Neuroscience Institute® (PNI). Find out more about Dr. Kesari’s approach to GBM treatment at PNI.

More information:
Santa Monica | Neuro-oncology 310-829-8265 | Neurosurgery 310-582-7450
Torrance | 424-212-5361
Burbank | 818-847-6049


About Dr. Santosh Kesari

Santosh Kesari, MD, PhD

Neuro-oncologist Dr. Santosh Kesari, MD, PhD, is a board-certified neurologist and is currently Chair and Professor, Department of Translational Neurosciences, Saint John’s Cancer Institute. He is the regional medical director of Providence Southern California’s Research Clinical Institute. Dr. Kesari is also Director of Neuro-oncology at Pacific Neuroscience Institute, Providence Saint John’s Health Center, and Providence Little Company of Mary Medical Center Torrance, and leads the Pacific Neuroscience Research Center at Pacific Neuroscience Institute.

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Last updated: June 30th, 2023