Oral Therapy Approved for Cancers Associated with Rare Genetic Alteration

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Part of the research for the development of the drug – Credits: @Gentileza

Another drug has just been added to the toolbox for treating cancer in what is known as precision medicine, which aims to treat tumors according to genetic biomarkers and is proving to improve response in patients. It is a oral therapy for adults and children with different types of locally advanced solid tumors or with metastases associated with a genetic alteration present in 1% of patients with these cancers, but in up to 90% of cases in some rare diagnoses.

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Yesterday, in the presentation of the results obtained with larotrectinib and disseminated at the last annual congress of the European Society for Medical Oncology (ESMO, for its acronym in English), spokesmen for Bayerthe producing laboratory, pointed out that with the recent approval of the Anmat of this new drug incorporates a therapeutic alternative for patients with pediatric solid tumors, sarcomas, thyroid, lung, gastrointestinal or skin (melanoma), “as long as they have the same genetic modification, which has been described in more of 20 types of cancer.

They estimated that there would be around 100 cancer patients who, each year, would meet these laboratory requirements to indicate the new therapy.

This modification is known as fusion in NTRK gene. In tumors that appear due to this genetic alteration, according to the company, the general response in adult patients was 67% and, in pediatric patients, 87%. The drug is administered through a capsule or a liquid solution, which “does not work on one type of tumor, but on a large number of tumors that express the same genetic abnormality,” he explained. Hector Mirandamedical director of the Pharmaceutical Division of Bayer Southern Cone, on the approved indication for advanced disease in an organ or with metastasis.

The genetic tests indicated for cancer patients to determine certain tumor characteristics that allow better definition of the disease and treatment allow the identification of specific biomarkers for which therapies already exist in the field of precision medicine. In these tests, certain cancers test positive for fusion of neutrophilic receptor tyrosine kinase genes (NTRK, by its name in English).

Those genes –NTRK 1, 2 and 3– can fuse with another gene and cause abnormal proteins to be made (TRK fusion proteins) that alter cell growth so that it becomes uncontrolled and favors the appearance of tumor cells.

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The presentation, in one of the halls of a hotel in downtown Buenos Aires, was also attended by adriana roseoncologist at Garrahan HospitalY Edith Grynszpancholpresident of the Natalí Dafne Flexer Foundation. Each year, according to Rose, some 1,350 pediatric cancers are diagnosed in the country and it is the second leading cause of death in children and adolescents between the ages of one and 15. 80% is treated in the public sector; Garrahan receives 520 of all annual cases.

Adriana Rose, oncologist at Garrahan Hospital, during the presentation of the new oncological therapy

Adriana Rose, oncologist at Garrahan Hospital, during the presentation of the new oncological therapy – Credits: @Gentileza Bayer

“With advances in chemotherapy, radiotherapy, surgery and clinical support, 80% of children can be cured. But there remains a percentage of patients with locally advanced, unresectable, metastatic, or relapsed or refractory tumors. This subgroup needs other types of treatments,” added the pediatric oncologist.

Referring to precision medicine, he pointed out that the advances in this area “generated a revolution in health because it was possible to start doing the genetic and molecular profiling of patients and adopt personalized treatments. In pediatrics, progress has been made in the last 10 years with great difficulty: the patients who have access are very heterogeneous populations, the treatments that are given to them afterwards are also, and, basically, access to these treatments is difficult”.

He stressed that larotrectinib it is the second of three drugs approved for genomic targets, rather than histological, that are being incorporated into cancer treatment. He defined it as “an innovative therapy for solid tumors [con fusión NTRK] both adults and children and that occur in about 20 types of cancer. It is a step to take in the use of medication that is less toxic and with a better quality of life for the patient”.

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