An effective and safe targeted drug against the FGFR2 mutation after chemotherapy in inoperable or metastatic patients who had no other viable options
The Italian Medicines Agency has recently officially approved the reimbursement for a new treatment, effective in slowing the evolution of the tumor and well tolerated, for patients with locally advanced or metastatic cholangiocarcinoma. It is a rare neoplasm (there are just over 2 thousand cases registered every year in our country) and subtle, because it does not give initial symptoms and is thus often recognized when it is at an advanced stage and more difficult to cure. So much so that the prognosis is still severe for most of the patients.
What is cholangiocarcinoma
Cholangiocarcinoma is a malignant neoplasm that originates from the rapid and uncontrolled proliferation of cholangiocytes, the cells that make up the walls of the bile ducts – explains Giordano Beretta, director of Medical Oncology in time.news and president of the Aiom Foundation (Italian Association of Medical Oncology) – . The bile ducts are the channels that transport bile from the liver to the intestine and, according to the site of tumor onset, intrahepatic (if they develop inside the liver) and extrahepatic (if they arise from the biliary tract) cholangiocarcinomas are distinguished external). A delicate area, where the surgical intervention, which could completely eliminate the tumor mass and allow to aspire to healing, often not practicable: in fact, about 70% of patients discover the tumor when it is late: Unfortunately surgery and chemotherapy they do not guarantee a lasting response, so 5 years after diagnosis less than 20% of patients are alive – adds Beretta -. In recent years, however, we have learned about the different subtypes of cholangiocarcinoma and the different genetic alterations responsible for its growth. As a result, new effective targeted drugs, such as pemigatinib, were also developed.
The new cure
Overall, about half of cholangiocarcinomas have one or more mutations potentially treatable with targeted drugs. In particular, translocations of FGFR2 (fibroblast growth factor receptor 2) are present in about 7% of intrahepatic cholangiocarcinomas – explains Filippo de Braud, Professor of Medical Oncology and director of the Department of Oncology and Hematoncology at the National Cancer Institute from Milan -. Pemigatinib a drug targeted against this alteration and the results of the trials that led to its approval (study FIGHT-202) have shown that, in those patients in whom chemotherapy fails to stop the neoplasm, the new drug alone can keep under I control the tumor in about 37% of cases, on average for 8 months. In practice they have indicated the efficacy and safety of pemigatinib, which now represents the best therapeutic option for Italian patients who have cholangiocarcinoma with FGFR2 fusions or rearrangements – continues de Braud -. The results are important and, in a small percentage of patients, complete responses were even observed, hence the disease disappeared.
Symptoms and centers with many different experts
For 10 years there had been no news for cholangiocarcinoma and there was a lack of valid options for the treatment of this locally advanced or metastatic tumor – says Giovanni Brandi, founder of the Italian Association of Cholangiocarcinoma Patients (APIC) and of the Italian Cholangiocarcinoma Group -. AND’ It is essential that patients be treated in a center with experience, both in the intervention (not simple due to the complexity of the area in which the neoplasm is located) and in the diagnosis and treatment, which require a multidisciplinary team of professionals specialized in this pathology. Precisely because the symptoms are not very specific and the neoplasm is discovered when it is already of considerable size, it is good that the people most at risk pay particular attention to possible alarm bells (in general it is better to go to the doctor in case of unjustified weight loss, jaundice , pain in the right flank radiating posteriorly): gallstones and primary sclerosing cholangitis, a severe chronic inflammatory disease of the liver, increase the odds for intrahepatic forms. Furthermore, the danger of cholangiocarcinoma also increases fatty liver, obesity and excessive alcohol consumption, as well as hepatitis B and C and liver cirrhosis concludes Brandi, who is also director of the Medical Oncology Specialization School at IRCCS Policlinico Sant’Orsola, University of Bologna.
June 22, 2022 (change June 22, 2022 | 19:22)
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