Neuroblastoma Clinical Trial


What is neuroblastoma?

Neuroblastoma is a cancerous tumor that begins in nerve tissue of infants and very young children. The abnormal cells are often found in the nerve tissue that is present in the unborn baby and later develops into a detectable tumor. Neuroblastoma is rare in children older than 10 years of age, however, it does occur occasionally in adults.

The tumor usually begins in the tissues of the adrenal gland found in the abdomen, but may also begin in nerve tissue in the neck, chest, or spinal cord. The adrenal glands are positioned on top of the kidneys. These glands secrete hormones and other important substances that are required for normal functions in the body.

In the U.S., approximately 700 children are diagnosed with neuroblastoma each year. It is often present at birth, but not detected until the tumor begins to grow and compress the surrounding organs. Most children affected by neuroblastoma have been diagnosed before the age of 5. In rare cases, neuroblastoma can be detected before birth by a fetal ultrasound. It is the most common solid tumor cancer in infants.

Neuroblastoma cancer cells can spread (metastasize) quickly to other areas of the body (for example, lymph nodes, liver, lungs, bones, central nervous system, and bone marrow). Approximately two-thirds of all children diagnosed with neuroblastoma will have some metastatic disease.

Prevention & Risk Assessment

What causes neuroblastoma?

The only risk factor that has been established for neuroblastoma is heredity, although the vast majority of neuroblastomas are not inherited. Recent research indicates that certain genetic variations double the risk of this disease. Also, having this particular variation increases the chance that a child will develop a more aggressive form of the disease.

The average age at diagnosis in genetically linked cases is younger than those cases that are not inherited. Cancer that presents in several different areas of the body at once is a sign that it may be a genetically inherited cancer.

What are the symptoms of neuroblastoma?

The following are the most common symptoms of neuroblastoma. However, each child may experience symptoms differently. The symptoms of neuroblastoma vary greatly depending on size, location, and spread of the tumor. Symptoms may include:

  • Abdominal mass either felt during an examination or seen as swollen abdomen
  • Painless, bluish lumps may be seen under the skin in infants
  • Trouble breathing due to tumors in the chest or abdomen
  • Tumors in the face or head can cause swelling and bruising of the area around the eyes and uncontrolled eye movement or bulging eyes
  • Compression of kidney or bladder by the tumor may cause changes in urination
  • Bone marrow involvement may present as pain, fatigue, limping, paralysis, or weakness
  • Diarrhea may be present; diarrhea is caused by a substance produced by the tumor (vasoactive intestinal peptide or VIP)
  • Fever
  • High blood pressure and increased heart rate may occur depending on location of tumor and the organs the tumor compresses

The symptoms of neuroblastoma may resemble other conditions or medical problems. Always consult your child’s doctor for a diagnosis.

  • Clinical Trial Coordinator
    • Ann Liew, MS, CCRP
    • | 202-476-2021
  • Principal Investigator
    • AeRang Kim, MD, PhD
    • | 202-476-2800