First Patient Treated in Pamiparib Trial for Glioblastoma

In a recent press release, the Ivy Brain Tumor Center, a nonprofit translational science initiative, shared that the first patient was treated in a Phase 0 clinical trial. The clinical trial is evaluating pamiparib for patients with recurrent or newly diagnosed glioblastoma. Glioblastoma is typically aggressive. While surgery, radiation, and temozolomide (TMZ) can increase overall survival rate, nearly 40% of patients do not have a response to TMZ, making a new treatment option incredibly important. Thus, if pamiparib is efficacious, it could fill an unmet need in this patient community.


In treating glioblastomas, one difficulty is developing a treatment option which can cross the blood-brain barrier, delivering a more effective treatment straight to the tumor. However, Dr. Nader Sanai, M.D. is confident that this Phase 0 trial will begin the journey to discovering a better and more effective treatment option for patients. This trial is also open to newly diagnosed patients, which is a patient population missing in prior studies.

During the Phase 0 clinical trial, researchers will evaluate pamiparib, an investigational treatment. The therapy inhibits PARP1 and PARP2, both of which help protect cells to survive against damage. Because glioblastomas are able to create their own blood supply, it is important to be able to prevent PARP from keeping the cells healthy and alive. Researchers are currently exploring pamiparib as a singular treatment or in conjunction with other therapies.

Altogether, 18 patients with recurrent glioblastoma and 12 patients with newly diagnosed glioblastoma will enroll in the clinical trial. Prior to surgery to remove the tumor, patients will receive pamiparib for a 4-day period. Following the surgery, researchers will test the tumors. If the drug was able to break through, it shows promise for overcoming the blood-brain barrier issue seen in prior studies.

Beyond glioblastoma, a New Drug Application (NDA) was submitted in China for pamiparib in patients with ovarian cancer.


Glioblastoma is a rare and aggressive astrocytoma, a brain cancer formed in star-shaped astrocyte cells. An estimated 20% of all diagnosed brain tumors are glioblastomas. Normally, glioblastomas form in the cerebrum, or the brain and spinal cord. However, this type of cancer is often extremely malignant. Since the tumors can create their own blood supply, they can feed their growth without additional support. Males are more likely to develop glioblastomas than females. Those who are older are also more likely to develop this cancer than younger individuals. Risk factors include genetic conditions and prior radiation treatment. Symptoms include:

  • Worsening and persistent headaches
  • Blurry vision or other vision changes
  • Nausea and vomiting
  • Difficulty thinking or speaking
  • Seizures
  • Mood swings
  • Appetite loss
  • Muscle weakness
  • Changes in behavior or personality

Learn more about glioblastoma.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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