Vall d’Hebron Halts Relapses in Childhood Neuroblastoma – Triples Treatment Duration

Rare, but “very aggressive.” That’s how he is childhood neuroblastoma, a type of pediatric cancer which affects approximately 90 children a year in Spain and represents about 10 percent of the oncological cases registered in this population segment. The survival of minors whose illness does not respond to first line of treatment or who present relapses is very low, which makes it essential to develop new therapeutic strategies. From this need arose the Beacon project, aimed at verifying the effectiveness and safety of combining immunotherapy with chemotherapy. The study, which included 65 patients between 1 and 18 years oldcoming from 29 centers in seven European countries, revealed “significant clinical results” to the satisfaction of professionals such as Lucas Moreno, head of the Pediatric Oncology and Hematology Service at the Vall d’Hebron University Hospital and coordinator of the trial.

Those patients who received dinutuximab beta along with chemotherapy in their treatment experienced a tumor reduction in 30.2 percentcompared to 18.2 percent of those who underwent conventional therapy alone, while the progression free survival lasted until 11.1 monthsthat is, triple that recorded by those who did not receive the therapeutic tandem and which was 3.8 months.

Metastasis reduction

One year later, 44 percent of the 43 patients who received combination therapy were still stable diseasecompared to 27 percent of the other group, made up of 22 patients, who initially received only chemotherapy. In fact, there was patients who achieved long-term benefits, although this information is not included in the published results of the study.

For this reason, Moreno assures that immunotherapy not only increases the response, but also prolongs the time until relapse: “Treatments where there is chemotherapy alone, In some cases they manage to stop the diseasebut in the end all patients end up progressing. So, adding immunotherapy makes a greater proportion of patients respondthe tumor becomes smaller, the metastases are reduced and more time passes until the next relapse.

Regarding safety, the data shows that the treatment was well toleratedwith mild side effects such as fever, dizziness or allergic reactions. The issue, as Moreno points out, is “to improve the treatment, but not on the basis of toxicities that involve a very hard burden for patients“. “Many can continue treatment at homewhich prioritizes quality of life”, he adds how to coordinate this study that sought include the entire population in which the first line of treatment had failed or had relapsed, “which ensures that the study population is representative of children with the worst prognosis.”

The specialist also highlights that these findings are influencing clinical practicesince it not only reflects that “adding immunotherapy and chemotherapy is beneficial”, but also that “it has changed how these patients are treated, in both North America and Europe“Chemoimmunotherapy is now offered to all frontline patients who do not respond well enough,” he says.

New combinations

In fact, this year it is scheduled to open in Spain and other European countries. the international clinical trial Beacon2the continuation of the Beacon project that investigates the combination of chemotherapy with dinutuximab beta antibody “in children and adolescents with relapsed or refractory neuroblastoma“. This new work seeks to “optimize combined therapy and explore new combinations” that improve response and patient survival who, according to the data already obtained, could complete the therapy from home.

According to Moreno, the two main objectives are to “expand the data” already obtained and “look for more powerful combinations”since “there are still patients who do not respond or who relapse.” “We are combining it with other targeted therapies or forms of immunotherapy. The trial already began a year ago in the United Kingdom and It is expected to open in Spain during 2026“, he reveals.

Make this therapy accessible as a challenge

“The main challenge is that neuroblastoma is aggressive and many patients end up dying“, continues the doctor. This trial goes to the core of the problem “using drugs in synergy to improve results.” Currently, the key is “to move chemoimmunotherapy to the first line and make it accessible in all countries through regulatory approval“, so that “more patients can benefit from this strategy.

At the same time, the study Beacon-Bio analysis biomarkers and liquid biopsy, which will allow treatments to be personalized and invasive tests to be reduced: “We study the patients’ genomes to see who responds best and we use liquid biopsy to detect relapses early, avoiding painful MRIs and biopsies”. “With a simple blood sample we can have quick and reliable results,” he details.