According to Dr. Lisa Diller, an internationally recognized leader in childhood cancer, published pediatric oncologist at the Dana-Farber/Childrens Hospital Cancer Center, and Soupy for Loopy Foundation’s Medical Advisor, mutations in a gene called ALK have been identified and described in approximately 10% of neuroblastoma tumors. These findings have been a major breakthrough in understanding the biologic basis of neuroblastoma. Importantly, ALK gene mutations have been described in a subset of a common form of lung cancer and a type of lymphoma, making ALK a very promising target for drug development and for the pharmaceutical industry. Drugs that inhibit the activity of ALK have been developed by drug companies and have demonstrated encouraging results in patients with ALK-mutated lung cancers and lymphomas. One example of this kind of drug is crizotinib, which was approved by the Food and Drug Administration for treatment of patients with ALK-mutant lung cancers. A trial of crizotinib in pediatric patients has been performed, and the drug has been shown to be well-tolerated, allowing for more extensive study in patients with neuroblastoma.
To facilitate incorporation of targeted inhibitors of ALK kinase into future upfront clinical trials for appropriate patients, testing for ALK mutations in the national setting will be performed in the national Children's Oncology Group. High quality centralized testing will be done. The development of centralized ALK testing is vital for study of the efficacy of introducing pharmaceutical ALK inhibition (crizotinib) into upfront therapy for patients with high-risk neuroblastoma.
Having the funding to do this laboratory testing on all children's neuroblastoma tumors (and knowing which children would be good candidates for receiving ALK-targeted therapies) will be a major advance for the field. Funds from Soupy for Loopy Foundation will be used for the actual genetic testing of the tumors - the reagents needed and the costs of shipping and processing. Results will be used to further understand the frequency of mutations, the type of mutations, and the effectiveness of targeting therapies to ALK.
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