Q: What is Neuroblastoma?

Neuroblastoma is a childhood cancer of the sympathetic nervous system, affecting approximately 650 children in the U.S. every year. It is the second most common solid tumor in infants. Most children are diagnosed by 2.5 years of age. Up to sixty percent of them have high risk disease that has metastasized (spread to other parts of the body) by the time they are diagnosed. Survival is dependent on age and disease stage: children diagnosed before the age of 18 months have a high survival rate, but high risk children diagnosed between 18 months and age 5 have about a 30 percent chance of surviving. For children over age 5, teens, and adults, the prognosis is very poor. Zev was diagnosed at age 3 with stage IV high risk Neuroblastoma.

Q: What is the Treatment for Neuroblastoma?

For children with lower stages of disease and favorable risk factors, surgery and/or some chemotherapy may be sufficient treatment. Children with risk disease typically receive multiple rounds of harsh, high-dose chemotherapies, radiation, surgeries, and retinoid therapy. In addition, many undergo stem cell transplantation, immunotherapy and other painful treatments. Life is disrupted for the entire family--constant visits to the cancer clinic are required and weeks are spent in the hospital. Treatment even in the best of cases can last several years.

Q: Where is Zev being treated and who are his doctors?

Zev is being treated in New York City at Memorial Sloan Kettering Cancer Center (MSKCC). MSKCC treats more children with neuroblastoma than any other hospital in the world. Every day approximately 35-40 children with neuroblastoma are treated in MSKCC?s Pediatric Day Hospital and the in-patient unit.

MSKCC has a world-renowned multidisciplinary team of physicians and scientists solely dedicated to the treatment, control, and cure of neuroblastoma. In contrast, most hospitals do not have specialists in neuroblastoma treatment on staff. Dr. Nai-Kong Cheung is the head of the Neuroblastoma Program and chief researcher. Dr. Brian Kushner, Dr. Kim Kramer and Dr. Shakeel Modak are the team's full time clinicians and researchers. Drs. Michael LaQuaglia (Chief of Pediatric Surgery) and Mark Kayton perform neuroblastoma and ancillary surgeries and practice surgical techniques performed only at MSKCC. Dr. Mark Souwedaine is the pediatric neurosurgeon who operates on tumors that have metastasized to the brain or spine. Radiation therapy is under the direction of Dr. Suzanne Wolden. The neuroblastoma team also includes a large team of highly skilled pediatric nurse practitioners, research nurses specially trained in administrating biologics, and a fulltime social worker.

Q: What progress has the MSKCC Neuroblastoma team made in the treatment of neuroblastoma?

In the 1980s, fewer than 5% of children diagnosed with high risk neuroblastoma survived. Although intensified chemotherapy and radiation treatments improved the chances of killing this vicious cancer, many children suffered severe side effects--including developing other forms of cancer caused by the treatments themselves! In 1987, the MSKCC team began treating children with 3F8, a monoclonal antibody developed by Dr. Cheung. 3F8 can attach to neuroblastoma cells and kill them, and can also train the child?s own immune system to attack and kill neuroblastoma. In the last 2 decades, 3F8 treatment notably improved survival, without lasting side effects.

Q: What innovative projects are on the horizon?

• The 3F8 antibody is presently being redeveloped to allow efficacy in a wider range of children.
• A number of different vaccines are in the process of development, intended to help boost the child?s immune system to better fight Neuroblastoma.
• There is also testing underway to improve the tumor killing activity of the monoclonal antibody 3F8.
• Adoptive cell therapy investigations are also underway.
• Drugs which specifically target the Neuroblastoma stem cells.

Q: Why is the Wolff family trying to raise money to fund anti-cancer therapies?

Funding for cancer research is limited in general. The vast majority of federal funding and private donations for cancer research are directed toward adult cancers. Fortunately, pediatric cancers are uncommon. Unfortunately, cancer funding tends to be distributed based on the number of people who are diagnosed with a particular cancer, rather than the years of potential life lost. For example, prostate cancer, which does not shorten the lifespan of the majority of patients who suffer from the condition, receives far more funding than all pediatric cancers combined.

Similarly, the pharmaceutical industry is generally not interested in pediatric cancers because of the small market size unless the treatment also happens to be effective in adult cancers. Furthermore, liability remains a strong disincentive. As treatments for adult cancers become more specific, these therapies are less likely to be useful in pediatric cancers.