A collaborative effort between researchers in Italy and the United Kingdom is focusing on a novel approach to combatting high-risk neuroblastoma, a particularly aggressive childhood cancer. The research centers on inhibiting the MYCN gene, a key driver of tumor growth, and utilizes a combination of a mini-protein and an existing antibody therapy to deliver targeted treatment directly to cancer cells. This work represents a significant step in addressing a disease where long-term survival rates remain stubbornly low, particularly for high-risk cases.
Each year in Italy, approximately 130 to 140 children receive a diagnosis of neuroblastoma, according to research efforts focused on the disease. When the neuroblastoma is classified as high-risk – a common scenario – the long-term survival rate falls below 40%, meaning fewer than four out of ten children survive. This grim prognosis fuels the ongoing search for more effective therapies that can directly target the molecular mechanisms driving the cancer’s aggressiveness. The collaborative study, involving Brunel University London and the University of Chieti, aims to address this critical necessitate.
Targeting MYCN: A Modern Therapeutic Strategy
The core of the research lies in combining Omomyc, a mini-protein, with dinutuximab, an antibody already used in first-line treatments for high-risk neuroblastoma. Professor Arturo Sala of Brunel University London explained that the goal is to transform the existing antibody into a more potent delivery system, carrying Omomyc directly into tumor cells. “We are studying the use of a mini-protein called Omomyc delivered through the anti-GD2 dinutuximab antibody, also used in first-line therapies for high-risk neuroblastoma,” Professor Sala stated. “We want to deepen the effects of Omomyc conjugated to dinutuximab.”
Researchers hypothesize a dual benefit from this approach. The dinutuximab antibody is designed to activate the immune system against the tumor by targeting GD2 molecules on the surface of neuroblastoma cells. Simultaneously, Omomyc directly inhibits MYCN within the tumor, reducing its ability to proliferate. This combined attack – stimulating the immune response and directly suppressing tumor growth – offers a potentially more effective treatment strategy.
The VAMOLAA Project: A Multicenter Network
This research from Chieti and London is part of the larger, multicenter project “VAMOLAA” (Valiant Approach against MYCN Oncogene to Leverage Antitumor Activity), currently in its preclinical phase. The project is coordinated by Patrizia Perri, a senior researcher at the Experimental Oncology Laboratory of the Istituto Gaslini in Genoa. VAMOLAA encompasses multiple parallel research lines all converging on the common goal of developing effective strategies to inhibit MYCN in neuroblastoma.
Neuroblastoma is a solid tumor that develops outside the skull and primarily affects young children. Classified as a rare cancer, its prognosis varies significantly. Low-risk forms may even regress spontaneously, but high-risk neuroblastoma remains a major challenge in pediatric oncology. The rarity of the disease also presents challenges for research funding, as it can be less attractive to pharmaceutical industry investment due to the smaller patient population. This underscores the importance of academic research and philanthropic support.
The Chieti-London branch of the VAMOLAA project is supported by the Italian Association for the Fight Against Neuroblastoma (Associazione Italiana per la Lotta al Neuroblastoma) through its Easter campaign, “Cerco un uovo amico” (“I’m looking for a friend egg”). The association, founded in 1993 in Genoa by parents affected by the disease, has been led by Sara Costa since 1996, who tragically lost her son Luca to neuroblastoma. Individuals can support the campaign by visiting the Association’s website or contacting them via email.
The development of new therapies for neuroblastoma, particularly those targeting the MYCN gene, represents a crucial area of research. While still in the preclinical stages, the VAMOLAA project and the collaborative work between researchers in Italy and the UK offer a promising avenue for improving outcomes for children diagnosed with this devastating disease. Further research will be needed to translate these findings into clinical trials and, new treatment options for patients.
The next steps for the VAMOLAA project involve continued preclinical studies to assess the safety and efficacy of the Omomyc-dinutuximab combination. Researchers will be closely monitoring the results to determine whether this approach warrants advancement to clinical trials. The ongoing support of organizations like the Italian Association for the Fight Against Neuroblastoma will be vital in driving this research forward.
Have you or a loved one been affected by neuroblastoma? Share your thoughts and experiences in the comments below.
Disclaimer: This article provides information for general knowledge and informational purposes only, and does not constitute medical advice. It is essential to consult with a qualified healthcare professional for any health concerns or before making any decisions related to your health or treatment.
