High Risk Neuroblastoma

Investigators: Carlos Morales Betanzos

Collaborators: Francis Eshun, M.D. and David Carpentieri, M.D. (Phoenix Children’s Hospital) and Konstantinos Petritis, Ph.D. (Translational Genomics Research Institute)

Disease Background

Who gets it?
What are the symptoms?
How do we detect it now?
How is it currently treated?
What are the current challenges?
  • Neuroblastoma is a form of cancer that starts in certain types of nerve cells
  • Neuroblastoma is the most common solid tumor in young children (1-10 years old)  and accounts for 7% of all pediatric cancers.
  • Symptoms are pain and abnormal lumps in the abdomen and the back.
  • However its is not usually detected until the lumps are large enough to bother the infant.
  • Surgery
  • High-dose chemotherapy/radiation therapy and stem cell transplant
  • Retinoid therapy
  • Immunotherapy
  • In many cases, more than one type of treatment is needed.
  • Even after a very aggressive treatment High Risk Neuroblastoma has a mortality rate of 30-50%.
  • New therapies to treat the disease are urgently needed
  • Researchers have found important molecular differences between neuroblastoma cells and the normal neuroblasts (early forms of nerve cells) from which they develop.
  • These differences can be explored as possible targets for new treatments.

 


Our Approach

To identify new possible treatments for high risk neuroblastoma, we are exploring a protein called ShcC that is expressed in high risk, but not low risk, neuroblastomas with the idea that by better understanding how this protein functions we can develop treatments that target this disease. To do this, we are collaborating with Dr. Petritis at T-Gen to use mass spectroscopy to identify post-transcriptional modifications of ShcC that may activate the protein and contribute to the pathogenesis of the disease.