Although this trial is still in phase I, in which its safety is evaluated, the positive results that have been obtained invite optimism.
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The Vall d’Hebron Hospital in Barcelona (Spain) has participated in the development of the first effective drug in a subtype of advanced gastrointestinal stromal tumors (GIST), an orphan condition for treatment and with a fatal prognosis of just a year old.
The drug, Avapritinib, has been tested in 56 patients with GIST and reduced the tumor in 55 of them who had a specific cancer mutation, according to the results of the study, published in the journal “The Lancet Oncology”.
The importance of this work lies in the fact that, to date, these patients did not have any type of effective treatment, and their survival was only one year.
The main investigator of the study is Michael Heinrich, from the OHSU Knight Cancer Institute of Portland (USA), while the only participating Spanish researcher has been César Serrano, oncologist at the Vall d’Hebron Hospital and head of the Translational Research Group at Sarcomas del Vall d’Hebron Institute of Oncology (VHIO).
“This represents a further step in the development of precision cancer medicine. Once again we have managed to turn a specific mutation into a therapeutic target that has allowed us to develop an effective treatment, “explained Serrano.
“This new drug has recently been approved by the US Food and Drug Administration (FDA) and is currently under review by the European Medicines Agency (EMA). Its approval by the health authorities is a milestone in oncology, because it will become the first treatment for patients with GIST who present this mutation, ”according to Serrano.
GIST is a type of sarcoma, an infrequent tumor, which determines the difficulty of carrying out clinical studies and finding active drugs.
Up to 85% of patients with GIST have an oncogenic mutation in a receptor tyrosine kinase, which can be in the KIT gene or in PDGFRA, so both became targets for developing new treatments.
The emergence of imatinib in the early 21st century revolutionized treatment as patients with unresectable or metastatic GIST went from uniformly lethal cancer to manageable disease, with long-lasting responses and better overall survival.
“However, patients with the PDGFRA D842V mutation did not benefit at all from either imatinib or subsequent approved tyrosine kinase inhibitors. Avapritinib has been designed to attack this specific resistance mutation in a powerful and selective way, and in view of the results of this assay it is evident that it works, “Serrano explained.
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GIST is a rare tumor, as it is believed to represent only 1 to 3% of all gastrointestinal malignancies.
Patients with this mutation are even more rare, since they barely reach 5% of all GIST tumors, “but despite being few, the development of a drug for them was a necessity, since right now they did not have any alternative and its prognosis was unfavorable, with an overall survival of 15 months, “said Serrano.
The study involved 56 patients with GIST with the PDGFRA D842V mutation, and 55 of them obtained tumor reduction, with almost 90% success in a partial or complete response.
“This is very encouraging when you consider that 96% of the participating patients had metastatic disease, and up to 61%, clinically advanced disease. Until now, experience with other drugs such as imatinib, sunitinib and regorafenib – all type 2 inhibitors – had shown little or no activity in this type of patient, “Serrano concluded.
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