PTC596 + Radiotherapy for Brain Tumor

This trial tests the safety of a new drug, PTC596 (Unesbulin), combined with radiotherapy for children newly diagnosed with high-grade brain tumors, such as diffuse intrinsic pontine glioma (DIPG). The main goal is to determine a safe dosage of PTC596 and assess how much of the drug reaches the blood and tumor. Participants will take PTC596 twice a week while initially receiving daily radiotherapy, then continue with the drug alone. Children with newly diagnosed high-grade brain tumors who can swallow pills and have not received previous cancer treatments might be suitable for this trial. As a Phase 1 trial, this research aims to understand how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

Yes, you may need to stop taking certain medications. The trial does not allow participants to take other anti-cancer agents, investigational drugs, enzyme-inducing anticonvulsants, rifampin, medications that prolong the QTc interval, CYP1A2 inhibitors like duloxetine, alosetron, or theophylline, beta-blockers, and therapeutic anticoagulants. Some SSRIs should be used with caution.

Research has shown that PTC596, also known as Unesbulin, is generally well-tolerated. Most patients did not experience major side effects. In one study, only one patient experienced serious side effects, such as low white blood cell counts (neutropenia) and low platelet counts (thrombocytopenia), but these effects were reversible. Overall, safety data from previous studies suggest that PTC596 is safe when used alone or with treatments like radiotherapy.¹²³⁴⁵

Unlike the standard treatments for brain tumors, which often include surgery, chemotherapy, and radiotherapy, PTC596 is unique because it is a small molecule inhibitor that specifically targets cancer cell division. Researchers are excited about PTC596 because it disrupts the activity of a protein called BMI-1, which is involved in tumor growth and survival. This targeted approach could potentially enhance the effectiveness of radiotherapy, making the combined treatment more powerful than existing options. Additionally, PTC596 is administered orally, offering a more convenient option for patients compared to some traditional treatments that require injections or infusions.

Research has shown that PTC596, which participants in this trial may receive, has promising results in early studies. In mice, PTC596 slowed tumor growth, meaning tumors grew more slowly compared to those not given the drug. This treatment targets proteins that cancer cells need to grow and survive. PTC596 has also shown potential against different types of cancer. Additionally, when combined with other treatments, it may enhance their effectiveness. While more research is needed, these early findings suggest PTC596 could help treat high-grade gliomas in children.³⁴⁶⁷⁸