HealthVorasidenib: A new drug gives hope to people with the cancer that...

    Vorasidenib: A new drug gives hope to people with the cancer that killed Severiano Ballesteros | Health & Wellness

    In 2017, after suffering an epileptic seizure, Alberto (not his real name) received horrible news. That year, at Jimenez Diaz in Madrid, he was diagnosed with a strange brain tumor that his doctors did not know how to deal with. Undecided, “they decided not to give me chemo or radio and see what would happen,” he says. “Afterward,” he recalls, “I started having more and more frequent seizures and they decided to operate on me again.” That operation was performed by another surgeon, who offered to remove the entire tumor. After the operation, he lost the mobility of the right side of his body and had to start a tough rehabilitation.

    “I remember feeling lost seeing that the doctors didn’t know which way to go. That made me look for another surgeon on my own and I ended up finding Dr. Sepulveda”, he says. Juan Manuel Sepulveda, coordinator of the Neuro-oncology Unit of the Hospital Universitario 12 de Octubre in Madrid, was surprised to hear that he had not received radiotherapy or chemotherapy, but he told him that, on this occasion, luck had been on his side. he. Sepulveda was at the time recruiting patients for the Indigo trial, designed to test a new drug in patients who had received no treatment other than surgery.

    Tumors like Alberto’s are known as low-grade gliomas, and it was a glioma of this type that the well-known golfer Severiano Ballesteros suffered. These tumors are characterized by having a mutation in the IDH 1 and 2 genes. This genetic alteration, found thanks to projects for the massive sequencing of the genomes of dozens of types of cancer launched in 2008, changes the activity of two essential enzymes in the function of the organism, which continue to do their homework, but begin to generate a toxic metabolite that damages DNA. Over time, the damage accumulates and mutations that fuel cancer growth proliferate.

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    Surgery and deterioration

    For decades, people with this condition have undergone surgery to remove the disease and then receive chemotherapy and radiation therapy to control it. These tumors are not cured with surgery and usually come back, although the return can be delayed up to five years. With chemotherapy and radiotherapy it was possible to prolong life, between 10 and 20 years, with good conditions. Later, the damage from radiotherapy begins to manifest itself and memory problems appear, intellectual performance decreases or it is difficult to walk quickly. Normally, in 12 or 14 years, patients cannot lead a normal and independent life.

    The discovery of IDH mutations allowed the development of drugs aimed at inhibiting the action of this damaged enzyme that poisons the brain. According to Sepulveda, vorasidenib, a drug with a special ability to reach the brain, began to be used, as almost always happens at first with experimental drugs, in people with advanced disease, “diffuse gliomas that had already been treated with chemotherapy and radiotherapy, in some cases on several occasions”. “But only 30 to 40% of the patients responded,” he recalls.

    Those figures made us think that the drug was useless, but later they considered that perhaps they had used it too late, when the modification in the expression of the genes and the evolution of the tumor clones had gotten out of control and the inhibition of an enzyme had already it was useless. “So we decided to go at the beginning,” says Sepulveda. “We did a study for patients with grade 2 glioma who had undergone surgery, but had not received chemo or radiation,” he says. The results of that work have just been presented at the annual meeting of the American Society for Medical Oncology, in Chicago, and have been published in the journal New England Journal of Medicine.

    “We have moved the counter back 27 months to when these people have to receive more aggressive treatments”

    Juan Manuel Sepulveda, coordinator of the Neuro-oncology Unit at Hospital Universitario 12 de Octubre in Madrid

    The Indigo study, which included 331 patients from around the world, showed that the drug, developed by the pharmaceutical company Servier, increased the time in which the disease did not progress after surgery, from 11.1 months when receiving placebo to 27. .7 months with vorasidenib. Two and a half years after the start of the study, the disease had progressed in 28% of the participants, compared with 54% of those who received placebo. The study’s lead author, Ingo Mellinghoff, of the Memorial Sloan Kettering Cancer Center in New York, said in a presentation to the media, that “the results offer an opportunity to change the treatments of this type of glioma with a new targeted therapy ”.

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    “For now, we can say that we have delayed the counter 27 months until the moment when these people have to receive more aggressive treatments with more long-term sequelae,” says Sepulveda, who believes that “there will be a group of very long survivors , because they are people who are very sensitive to these drugs”. “There is a patient who started taking it three years ago, the tumor has shrunk and cannot be seen, and we don’t know how long it can stay that way,” he exemplifies.

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    “This opens the door to personalized medicine for these patients. This disease is rare, it is a very rare type of tumor, and these results give hope for a disease in which there was little research,” says Cristobal Belda, now director of the Carlos III Health Institute, and formerly an oncologist specializing in brain tumors. . At the time, he treated Ballesteros. “It’s an exceptional advance,” he says.

    Six years after his diagnosis and after horrible periods, in which he needed a large amount of medication against epilepsy and could not even go outside or go to rehabilitation, Alberto lives with hope. “Now we are lowering the medication and I continue to notice improvement because he had lost a lot of physical capacity. I couldn’t even get in front of the computer, because I would have an epileptic fit”, explains Alberto, who previously worked as a computer engineer. “Now I can leave the house and walk, which will seem silly to you, but for me it is incredible, and I am back in intensive rehabilitation. I am very happy, ”he sums up.

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    Source: EL PAIS

    This post is posted by Awutar staff members. Awutar is a global multimedia website. Our Email: [email protected]


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