Olaparib for Pediatric Cancer

This trial aims to determine if olaparib is safe and tolerable for children and teens with solid tumors that have returned or resist treatment. Researchers are testing olaparib, an experimental medication, in different age groups to evaluate its performance. Eligible participants have a solid tumor or primary central nervous system tumor with a specific genetic issue (HRR deficiency) and have exhausted standard treatment options. As a Phase 1 trial, the research focuses on understanding how the treatment works in people, offering participants the opportunity to be among the first to receive this new treatment.

The trial requires that you do not use strong or moderate CYP3A inhibitors or inducers while participating. If you are taking these, you may need to stop. The protocol does not specify other medications, but you should discuss with the trial team.

Research has shown that olaparib has been promising in earlier studies with children. In these studies, most children tolerated the medication well. Some children with specific DNA issues in their tumors even responded positively. However, possible serious side effects should be noted. These can include bone marrow problems, such as Myelodysplastic Syndrome (MDS) or Acute Myeloid Leukemia (AML), which affect blood cell production. Although olaparib is approved for adults with certain cancers, its safety and effectiveness in children remain under investigation. While some evidence suggests children can tolerate it, further research is needed to confirm its safety for them.¹²³⁴⁵

Olaparib is unique because it targets cancer by inhibiting PARP, an enzyme involved in DNA repair. Unlike traditional chemotherapy, which attacks rapidly dividing cells indiscriminately, olaparib specifically targets cancer cells with defective DNA repair mechanisms. This precision reduces damage to healthy cells, potentially leading to fewer side effects. Additionally, olaparib's oral administration offers convenience compared to intravenous options, making it a more appealing choice for pediatric patients and their caregivers. Researchers are excited about this targeted approach as it represents a shift towards more personalized and less toxic cancer therapies.

Research has shown that olaparib might help treat solid tumors in children. In a previous study, olaparib was generally well tolerated and helped shrink tumors in 2 out of 15 patients, indicating possible benefits. Early results suggest the drug can reduce DNA damage in about half of the patients. While olaparib already treats other cancers like ovarian cancer, its success there offers hope for treating children's cancers. This trial will evaluate olaparib in different pediatric age groups, targeting specific gene changes that allow cancer cells to repair themselves, potentially stopping tumor growth.²⁶⁷⁸⁹