Cancer is the leading cause of death by disease for children in the United States. Tragically, children with the rare cancer neuroblastoma often succumb to the disease despite aggressive treatment. That is until exciting new research showed that adding an experimental antibody to treatment shows promise to save the lives of children with this rare and aggressive cancer.  About Neuroblastoma Neuroblastoma is cancer that begins in immature nerve cells and primarily affects the brain of babies and children younger than five years old. Each year, about 800 children in the United States are diagnosed with neuroblastoma. More than half of these diagnoses in children are discovered after the cancer spreads and becomes high risk.  Once the cancer has spread, medical professionals must take aggressive measures. Traditionally, treatment starts with high-dose chemotherapy followed by surgery to remove any remaining tumors. Patients then undergo additional chemo, followed by a stem cell transplant to rebuild the immune system, and conclude with radiation.  New Neuroblastoma Treatment in Clinical Trials Recently, doctors have introduced another drug into the regime – Dinutuximab. This drug latches onto proteins on the surface of many neuroblastoma cells. This new treatment hopes to boost the child’s immune response to cancer cells that have survived the treatment. A third of patients treated with Dinutuximab were recurrence-free two years after treatment compared to 46% of those who received standard treatment alone. In this December 2021 study, however, researchers explored how a dose of a similar drug administered earlier might impact treatment outcomes. The team led a clinical trial that found a significant increase in survival rates, with 74% of children recurrence-free two years later. This experimental antibody shares similarities with Dinutuximab, but it was built specifically to limit pain, which was a significant side effect of similar treatments. Children’s Oncology Group will lead the series of trials to test this approach in newly diagnosed children. If approved, chemoimmunotherapy would become the standard therapy and significantly improve chances of curing high-risk neuroblastoma. Improving Brain Cancer Survival This new treatment trial highlights brain cancer treatment’s tremendous leaps and bounds. Families and patients with a once tragic diagnosis can now feel hopeful as new options continue to emerge. The National Foundation for Cancer Research (NFCR) helps accelerate these types of game-changing discoveries through GBM AGILE, a revolutionary clinical trial platform designed for patients battling the deadliest brain cancer, glioblastoma. As a founding member of GBM AGILE, NFCR has taken a leading role in this unprecedented global effort. With help from our generous supporters, rapid progress is happening. New trial sites are opening in the United States and worldwide, and patients are being enrolled and receiving the most promising treatments. Learn more about GBM AGILE or make a donation today to support this impactful initiative. Additional Reads You May Enjoy: Childhood Cancer Treatment by NFCR Partner Receives Fast Track Review and More Good News From the FDA Pediatric Cancer Facts and the Need for Improved Treatment GBM AGILE – Changing the Way We Fight Brain Cancer Stay connected with us! Receive our monthly e-newsletter and blogs featuring stories of inspiration, support resources, cancer prevention tips and more. Sign up here. Related Video:

Oncoheroes Biosciences, a biotech focused on advancing new therapies for childhood cancer, recently announced that the Unites States Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) to volasertib, one of its drugs. Volasertib is a small molecular inhibitor that has been identified as an anti-cancer agent. Receiving ODD and RPDD qualifies Oncoheroes to receive fast-track review, partial tax credits for clinical trial expenditures, and a priority review voucher at the time of marketing approval of volasertib. This is fantastic news in the realm of childhood cancer; however, many individuals are a bit uncertain about what this actually means. To keep the community informed, National Foundation for Cancer Research provides the following simple breakdown. How common is childhood cancer? There are dozens of cancers and numerous subtypes that occur in children aged 0-14 years. Childhood cancers are often different than adult cancers as pediatric cancers are not strongly linked to risk factors related to lifestyle or the environment. In the US, cancer is the leading cause of death by disease in children – taking the life of nearly 1,200 children each year. It is estimated that one in 389 children will be diagnosed with cancer by the age of 15. What is RPDD? RPDD is a voucher system designed to support teams that develop products (such as volasertib) that treat or prevent rare pediatric diseases, including cancer. The voucher system is a type of incentive to encourage the development of these products. Developing a new product is costly and timely – costing millions to billions of dollars and many years to complete. After the lengthy development process, the products then go to the FDA for review. The FDA has 10 months to review each product, further extending the time it takes to move the product to market. The priority review voucher that is awarded with RPDD allows an expedited six-month review instead of the 10-month review. These vouchers can only be awarded by the FDA, demonstrating that the significant potential of volasertib is recognized by government officials. What is ODD? First and foremost, an orphan drug is defined as a drug intended for the treatment, prevention, or diagnosis of a rare disease or condition that affects less than 200,000 people in the United States. The FDA awards Orphan Drug Designation to potential therapies addressing the unmet needs of underserved patients with a rare disease. ODD encourages organizations to invest the time and money into a product that will be used by a comparatively small population. The incentives that accompany this status include a tax credit on expenditures incurred in clinical studies, exemption from filing fees such as the user fee, eligibility for a research grant awarded by the FDA, and seven years of US market exclusivity upon approval. Being granted orphan designation does not modify the standard regulatory requirements to obtain marketing approval. What is volasertib? Volasertib is an enzyme inhibitor, effecting the enzyme known to be involved in disease progression in a number of cancers. This product was originally discovered and developed by Boehringer Ingelheim for the treatment of Acute Myeloid […]