According to a story from Chronicle Live, children struggling with the rare childhood cancer neuroblastoma and their families celebrated the news that the National Institute for Health and Care Excellence (NICE) has released a resolution in which the organization recommended that the medication dinutuximab beta be approved for coverage on the National Health Service (NHS) for patients with high-risk neuroblastoma. The decisions means that these patients will have access to an all new treatment option that will improve their chances of long-term survival or even safe their lives.
About Neuroblastoma
Neuroblastoma is a form of cancer that develops in nerve tissue and most commonly appears in young children under age five. Causes of this type of cancer are unclear. A small percentage of cases appear to be the result of inherited genetic mutations. Possible risk factors could appear due to events during pregnancy, such as usage of medications, hair dye, other birth complications, and use of hormone and fertility drugs; however, the subject requires far more research. The tumor usually begins at one of the adrenal glands, but can originate in other areas, such as the back, neck, chest and abdomen. Symptoms include a lump in the affected area (which may be blueish and usually painless) and bone pain. Other symptoms depend on the location of the tumor; for example, a tumor in the chest could cause breathing issues. Five year survival depends on the stage of the cancer, but stands at 68 percent for tumors detected after the first year of life. To learn more about neuroblastoma, click here.
Getting Approved
The decision comes after a draft proposal released last month from NICE in which they appeared to be prepared to reject the drug for coverage, primarily because of its great cost. This prompted a backlash from patient advocacy organizations such as the Bradley Lowery Foundation. Bradley Lowery was a neuroblastoma patient who died about a year ago; however, treatment with dinutuximab beta was able to buy him precious months of time with his family that he would not have had otherwise. It is likely that negotiations between these organizations and NHS officials played a major role in causing NICE to change its mind.
A mother from the North Shields area, named Kelly, also says that treatment with the drug saved the life of her daughter Hope Feeney. After participation in a clinical trial testing the drug, her neuroblastoma stabilized. Now, patients in the UK will have access to this innovative treatment option.
