Genetic Map of Aggressive Childhood Brain Tumor Leads to New Drug Discovery
Scientists have created a genetic map of medulloblastoma, an aggressive childhood brain tumour, leading to the discovery of a new drug that significantly extends survival rates. The research, funded by various organisations including the US National Institutes of Health and the Seattle Children's Brain Tumour Endowment, was published in the journal Genome Medicine.
The genetic map, created by collaborative research efforts worldwide, has identified four main subgroups of medulloblastoma: WNT, SHH, Group 3, and Group 4. Each subgroup has distinct genetic features and responds differently to treatments. This understanding has facilitated the development of targeted therapies tailored to these subgroups.
One such therapy is Ixabepilone, a drug typically used for breast cancer. Researchers found that it dramatically reduces tumour size and extends survival rates in preclinical models of medulloblastoma. This is the second drug identified using the genetic map that shows high effectiveness in treating the disease. Long-term, new therapeutics targeting identified proteins and pathways are being explored in collaboration with drug companies. Existing drugs that target specific areas on the genetic map are also being considered for short-term treatment.
The genetic map of medulloblastoma is a significant breakthrough in understanding and treating this aggressive childhood brain tumour. It has led to the discovery of a new drug, Ixabepilone, and opens avenues for further research into targeted therapies. The collaborative efforts of researchers worldwide continue to advance the field, with the ultimate goal of improving outcomes for children with medulloblastoma.
