A new molecule, vorasidenib, is able to slow down the tumor growth of gliomas, low-grade and slow-growing brain tumors. This was demonstrated by an international study, which saw the participation of the university neuro-oncology of the Molinette hospital of the Città della Salute in Turin, currently directed by Professor Roberta Rudà.
The results of the Indigo study, a phase three trial, the last before the official approval of a new drug, which recruited 331 patients from ten countries, and which could revolutionize clinical practice, were published in the New England Journal of Medicine and previewed in recent days in plenary session at the American Society of Clinical Oncology (Asco) in Chicago. Neuro-oncology of the Molinettes, having participated in the design of the study and recruited patients, appears among the authors of the publication, the only Italian center and among the few European centres.
Vorasibenib, a drug that is taken orally, has shown to be well tolerated and, in addition to having an effect on the tumor, it also appears to be active in reducing epileptic seizures. It will now have to go through the regulatory process and approval from licensing authorities in the US and Europe before it can be prescribed to patients. It will then be able to open up for patients the scenario of “target therapies or precision therapies”, already widely used in oncology but so far of limited use in neuro-oncology.
Cerebral gliomas, it should be remembered, affect about 4-5 people out of 100,000 inhabitants. The so-called “low-grade” ones, represented by astrocytomas and oligodendrogliomas, mainly affect young patients (between 20 and 40 years of age) and are characterized by the presence of the mutation of the Idh1-Idh2 genes, crucial for tumor growth.
Although they are not malignant neoplasms, for these tumors, due to the infiltrative characteristics in the healthy nervous tissue, it is not possible to proceed with a radical surgical removal. Over time, therefore, the residual tumor tends to grow and can evolve towards more aggressive forms.
When the glioma presents the Idh mutation, it produces an abnormal metabolite (oncometabolite), which promotes tumor growth and infiltration and is also implicated in the susceptibility to develop epileptic seizures, a frequent and disabling symptom for these patients. The potential drug vorasidenib is a specific inhibitor (target therapy) of the Idh mutation and its action results in a significant slowing of tumor growth, allowing to postpone radio and chemotherapy, currently considered standard therapies. In fact, these therapies can be burdened by important side effects, which significantly impact the quality of life of these young patients.
The university neuro-oncology center of the Molinette hospital in Turin, which actively participated in the research, has for years represented excellence in the field of diagnosis and therapy of brain tumors, a reference point for patients from all over Italy, in particular diagnosed with glioma .