Neuroblastoma treatment: two hopeful drugs

Lucas Moreno, head of the Vall d’Hebrón Pediatric Hematology and Oncology Service.

A new european project in which he participates Vall d’Hebron Hospital seeks to find treatments against him neuroblastomaone of the pediatric tumors more common and aggressive. Lucas Morenohead of the Pediatric Hematology and Oncology Service at the Barcelona hospital, prepares the foundations to start within one year a phase 2 trial with two protagonistsdrugs bevacizumab Y dinutuximab beta (anti-GD2), skeleton base from which new combinations to fight the disease.

This research is part of beacon-projectstarted in 2013 and driven by the University of Birminghamwhich he presented this June at the annual congress of the American Society of Clinical Oncology (ASCO) results of a randomized trial for patients with relapse. Moreno explains in Medical Writing that “22 patients were offered chemotherapy, while the 43 remaining immunotherapy with anti-GD2. Despite being a very small trial, it was shrink the tumor from 17 percent to 35 percent, while the relapse-free survival a year passed from 27 percent to 57 percent with the antibody.

These results have served as the basis for deconstruct the bases next’s trialwhich aims to improve the treatment of patients who follow relapsed. “With this new trial we seek to go one step further with the previous results. Beacon has shown that the immunotherapy treatment is more effective than chemotherapy aloneand now we are looking to test it together with bevacizumab, anti-GD2 and combining both”.

In the world of relapsed neuroblastoma there are very few patients suffering from this disease, and this phase two trial It is, in the words of Moreno, “the largest that has been done in Europe. We generate the data that we can and it is better than what was before, with much more limited information. In this new phase 2 trial we will test new drugs to analyze if we can improve the results”.

Next step: Transfer the results to clinical practice

Another of the main ambitions that this new research hopes to achieve is to transfer the results obtained in Pediatrics and Oncology to clinical practice, so that it can be prescribed to patients. “This in Europe has the complication that, when not be an approved indication, health systems must be convinced that the results are good enough to improve access to drugs. There are already countries where these results are being incorporated, but there is work behind it”, analyzes the Vall d’Hebrón specialist.

Although the drug with immunotherapy is already approved in Europe, it does not have the approval if it is used with chemotherapy. Being a different indication“we must carry out a longest wayuntil it change the indication or that it be allowed to be used with the data we obtain”, explains Moreno.

The new trial will feature a dynamic design in which new drugs will be tested, always maintaining the base of the bevacizumab and of anti-GD2, as Moreno explains: “If some of them don’t work, we eliminate them and try new ones. We are still defining it, although the skeleton from which we will build new combinations is clear. We do not know the final number of branches that we will be generating yet”.

Genomic sequencing to address neuroblastoma

Within the Beacon assay itself there is a Project ambitious of genome sequencing which was presented at ASCO. From blood and tumor samples, researchers try to find out which patients may have a better or worse prognosis against neuroblastoma and which treatment would benefit more than another. after discovering new biomarkers of prognosis such as PHOX2B mRNA and tyrosine hydroxylase, Moreno explains that “patients who have them generate worse results than those who do not. It is the first part of a program for these studies to help us better treat patients in the next trial and in clinical practice.”

Although it may contain statements, data or notes from health institutions or professionals, the information contained in Medical Writing is edited and prepared by journalists. We recommend the reader that any questions related to health be consulted with a health professional.

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