Speakers

Infant-type hemispheric glioma: Are we at the starting point of precision medicine?
1109 14:35-14:45
Interim Meeting of AASPN/303A
Introduction Infant-type hemispheric glioma usually presents as a large intra-axial tumor in infants or young children. Most of these tumors are high-grade gliomas. The treatment strategies include surgical excision, chemotherapy, and radiotherapy. However, limited by patients’ young age and generally large size of the tumors, surgical excision and radiotherapy are risky. In recent years, gene sequencing has identified certain molecular characteristics of this disease, including ALK fusion, NTRK family mutation, etc. A few cases shown in the literature have started to be treated with target therapy. Cases In 2021, the New England Journal of Medicine published a case about a 3-year-old boy with an inoperable infant-type hemispheric glioma. A kinase inhibitor targeting ALK, Lorlatinib, successfully shrank the tumor so that it became invisible on MRI. In our institute, a 10-day-old infant was also diagnosed with infant-type hemispheric glioma. He has also been successfully treated with Lorlatinib for the past 3 years, after two surgical resections and recurrences. A month ago, a 6-day-old newborn again received surgery in our institute for this tumor, and the gene sequencing again showed ALK fusion. Discussion Along with the progression of molecular diagnoses and classification in pediatric brain tumors, precision medicine is gradually showing its power to treat these challenging diseases. Although the case numbers are still very small, we are enthusiastic about this type of target therapy after witnessing its effect in the dramatic shrinkage of infant-type hemispheric glioma, a disease with a typically poor prognosis. More experience and larger-scale clinical trials should be initiated to build a new standard of treatment for infant-type hemispheric glioma. Conclusions Limited cases in the literature and our experience have shown the importance of gene sequencing and miraculous treatment outcomes with target therapy. Perhaps we are at the starting point of long-term control for this challenging disease by using precision medicine.