Q & A with Dr. Matthay

An interview with Dr. Kate Matthay, Chief, Pediatric Hematology/Oncology, UCSF

Current State of Childhood Cancer Research/Treatments/Cures

1) What are the most common types of childhood cancer?

Leukemias/lymphomas followed by brain tumors, neuroblastoma, Wilms’ tumor and sarcomas.

2) How have childhood cancer incidence and survival rates changed over the years?

Survival has significantly increased so that 80% of children with cancer in the U.S. will survive.

3) Have researchers gained any insight into causes of childhood cancer or ways to prevent it from developing?

We have learned that some types have a genetic predisposition, such as mutations in p53, or ALK or RAS. Very few environmental causes have been identified, other than exposure to radiation or toxic chemicals.


4) Have there been (and if so, what are they) any major breakthroughs in terms of research discoveries and/or new treatments in the past year or so?

Many new targeted therapies are under investigation, but the main finding of importance to childhood cancer may be the anti-GD2 antibody for neuroblastoma; and a little longer ago, rituximab for lymphoma and imatinib for Ph1+ leukemias. There have been many new discoveries of genes involved in cancer initiation and progression, but it is too early to say what the impact on cure will be.

5) Personalized medicine seems to be one of the current hot topics in terms of promising research avenues. Are there any other areas of focus that physicians/scientists are excited about?

Yes, we are very interested in identifying specific mutations in the cancer that are “druggable”, such as the ALK mutation in neuroblastoma, or mutations of BRAF in brain tumors. One of the challenges is that there are many different mutations possible even in a single gene, and they may each require a new drug. Many investigators are trying to genetically profile a cancer to personalize the treatment, but we do not yet have enough active drugs for every abnormality.

6) There was recently a scare in which it seemed as though hospitals and clinics would run out of methotrexate, a drug key to the treatment of ALL in children. Given that you see patients; can you convey the impact that these shortages have on children battling the disease?

This has a very great impact, and is multifactorial in cause, partly related to lack of governmental regulation, low revenue for industry, etc. We need to work with doctors and government and industry to prevent this problem.

7) The NCI continues to decrease the amount of funding provided for childhood cancer research. Yet, more children die from childhood cancer in the United States than any other disease. What are researchers to do? How do you compensate for these continued cuts?

Again, support from the Foundation is vital. More publicity is needed!


8) In your expert opinion – do you see us finding a cure for all childhood cancers?

Yes, that is my dream, that no child should die of cancer. We have made great advances, now the challenge is to continue to improve the outcome for the more advanced cancers, decrease the late effects, and also to see that these advances are made available to the other 200,000 children per year in low and middle income countries also afflicted with cancer.