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GlioblastomaAugust 2017Summary reviewed June 2026

A Cancer Drug's Dangerous Side Effect Can Be Reversed With Breaks and Extra Medication

Researchers tested selinexor, a cancer drug, in 192 patients and discovered why it causes dangerous drops in platelet counts. They found the drug blocks a signal that helps the body make platelets, but giving patients a break from the drug and using platelet-boosting medications can reverse the problem.

What the trial was testing

The trial enrolled 192 patients with glioblastoma. The study was sponsored by Karyopharm Therapeutics Inc and tracked outcomes across the full group of patients who matched the trial's eligibility profile.

It was an early-stage trial — researchers are still confirming safety and getting an early look at how well the treatment works. Trials at this stage are designed to produce evidence regulators and physicians can act on — not just observations to follow up later.

What the results showed

Researchers identified how to reverse the main side effect that limits selinexor dosing in cancer patients.

Blood · 2017 · NCT01607905

These findings — that the study pinpointed why selinexor causes low platelets and found a way to reverse it — were published in the Blood and represent the headline result of the study.

Researchers tracked outcomes across 192 patients enrolled in the trial. The result was consistent enough across the group that the team felt confident reporting it.

What this means for patients

For patients with glioblastoma, this result changes the calculus on what to ask their care team about. Whether it changes day-to-day care depends on factors like disease subtype, prior treatments, and where the patient is in their care journey.

What you can do now

Selinexor is FDA-approved for certain blood cancers but causes low platelet counts as a side effect. This study showed doctors can manage this by scheduling breaks from the drug and using medications that boost platelet production. If you're taking selinexor, talk to your doctor about managing side effects to stay on treatment.

Eligibility for the treatments mentioned above depends on specific test results and clinical history. Bring this summary, the trial name, and your most recent labs or pathology report to your next visit.

Open glioblastoma trials

RecruitingSafety & dosing

A Phase 0/1 Study of BDTX-1535 in Recurrent High-Grade Glioma (rHGG) and Newly Diagnosed Glioblastoma (nGBM) Participants With EGFR Alterations or Fusions

This study will administer the investigational drug, BDTX-1535 to eligible patients with recurrent high-grade glioma (HGG) and newly-diagnosed glioblastoma (nGBM). BDTX-1535 was designed to block a growth signal important to some cancers. BDTX-1535 is being tested in this study to see if it can be given safely to people who have tumors that can be dependent on that growth signal because of changes in a protein called EGFR. These gene changes are called amplifications, mutations, fusions or alterations and are found only in the tumors. The study design includes a Phase 0 component with PK/PD-trigger for participant enrollment into an Expansion Phase 1 component. The primary objective of the Phase 0 component is to evaluate the PK endpoints of BDTX-1535. The primary objective of the Phase 1 component is to establish the safe dose of BDTX-1535 to be used in participants with a specified treatment regimen, three of which include standard of care radiotherapy for nGBM participants.

Chandler, Arizona, United States +1 more
RecruitingInterventional study

Magnetic Resonance Imaging for Improving Knowledge of Brain Tumor Biology in Patients With Resectable Glioblastoma

This clinical trial uses a type of imaging scan called magnetic resonance imaging (MRI) to study brain tumor biology in patients with glioblastoma that can be removed by surgery (resectable). Malignant gliomas are the second leading cause of cancer mortality in people under the age of 35 in the United States. Glioblastoma is a type of malignant glioma with very poor patient prognosis. There are currently only about 3 drugs approved by the Food and Drug Administration (FDA) for the treatment of glioblastoma, one of them being administration of bevacizumab, which is very expensive. It is the most widely used treatment for glioblastoma with dramatic results. However, previous clinical trials have not demonstrated an overall survival benefit across all patient populations with glioblastoma that has returned after treatment (recurrent). The study aims to identify which patients who will benefit from bevacizumab therapy by observing MRI images and corresponding imaging biomarkers.

Los Angeles, California, United States