We investigate childhood malignancies of the nervous system (brain tumors, neuroblastoma), the eye (retinoblastoma) muscle and bone (sarcomas), and blood cells (leukemias). The following highlights some of our accomplishments in the past year.

Neuroblastoma. Neuroblastoma, a tumor of the sympathetic nervous system, is one of the most common solid tumors in infants and children. Drs. Robert Seeger and Shahab Asgharzadeh continue to develop gene based tests that can be used clinically to predict how patients will do. Drs. Yves DeClerck, Leonid Metelitsa, Asgharzadeh, and Seeger have discovered that neuroblastoma cells can “turn on” inflammation in a way that tumor cell growth and spread is promoted.  Collectively, this research will result in patients receiving the most appropriate and effective treatment (“personalized medicine”). 

 Brain Tumors.   Brain tumors are the most frequent solid cancer and also the most common cause of cancer-related death in children. Dr. Asgharzadeh, using microarray “gene chips” discovered genes whose expression (activity) can be used to determine the chance of survival for children with medulloblastoma. Importantly, it appears that young children can be identified who can be cured without the need for radiation therapy, which preserve their ability to learn and function in society.  Dr. Gregory Shackleford has developed a novel model for medulloblastoma through genetic engineering of mice.  This will be very useful for developing new treatments that target genes and pathways responsible for medulloblastoma.  Dr. Leonid Metelitsa has developed a new immunotherapy for brain tumors that relies on oral vaccine and that appears effective in mice.  Dr. Jonathan Finlay continues his innovative clinical treatment, termed “Head Start”, for young children with brain tumors that seeks to cure them without using radiation treatment.

Leukemia.  Acute lymphoblastic leukemia (ALL) is the most frequent type of cancer in children and is seen throughout adulthood as well.  Therapeutic advances over the past 40 years have made ALL a curable disease in 80% of cases, but in the remainder, a small number of leukemia cells become resistant to treatment and grow back as incurable leukemia. Dr. Markus Muschen Muschen is seeking markers to identify these key cells and ways to effectively treat them. Drs. Nora Heisterkamp and John Groffen also are investigating how leukemia cells can resist treatment by using normal cells to create a hospitable and supportive environment for leukemia cells.

Clinical testing of new treatments.  In the area of clinical trials, we test new therapies that are based upon laboratory findings.  We have organized multi-institution consortia that specialize in testing new strategies for childhood neuroblastoma (www.nant.org), brain tumors, and leukemia (www.tacl.us/). Childrens Hospital Los Angeles is the only institution in the US that leads three clinical trials consortia.   

Integration of research teams to generate substantial additional funding for childhood cancer research.  We have used our successful “bench to bedside” research strategy to continue developing teams of laboratory and clinical investigators who work together.  Funding from the T.J. Martell Foundation has provided and continues to provide “venture capital” to establish outstanding groups of investigators.  This strategy has been very successful so that we now have $10 in additional funding for each $1 that the T.J. Martell Foundation has provided.  

Summary.  Many of the advances that we have made and will continue to make are possible because of the consistent support of the T.J. Martell Foundation.  Your support has allowed us to retain and recruit some of the best research scientists and clinical investigators.  We are proud of our accomplishments, but we are equally proud of your continued commitment to us.