Research Offers Hope for Better Treatments of Pediatric Brain Tumors and Brain Cancer

Pediatric brain tumors and brain cancer, while rare, are considered to be the deadliest of all childhood cancers. While the incidence rate may be relatively low, with approximately 3,400 new cases diagnosed annually in the United States, the mortality rate of children with these types of cancers is significantly higher than other childhood cancers and diseases. Roughly one-third of these kids will not survive beyond five years.

However, it is encouraging to note that, as a result of advancements in research and treatment methods, there have been noticeable decreases in overall cancer death rates since the early 1990s, with an estimated 25,000 survivors currently living in the U.S. Although “survivorship” for these kids comes with its own lingering effects, such as cognitive damage, physical challenges and social isolation, the research that is taking place today will hopefully, someday help to minimize these effects, and allow these survivors to live longer, healthier lives.

Hope for the children and families who face this dreadful disease comes from the tireless efforts of many non-profit organizations, research foundations, hospitals and other pediatric medical institutions who are dedicated to finding the cause, and ultimately, the cure for this deadly invader of children. A few of the more notable milestones of late include:

• The creation of a tissue bank consortium, a collaborative initiative involving the Children’s Brain Tumor Foundation and a group of researchers at several leading pediatric oncology hospitals nationwide. The bank will enable researchers to obtain samples of brain tumor tissue that can be characterized, analyzed and used to evaluate treatments. The results can then be documented in a database that can be shared with pediatric cancer facilities across the country.

• The publishing of a landmark study of medulloblastoma*, a type of brain tumor typically found in children. The study team found that the number of mutations in pediatric medulloblastoma tumors is five to ten times fewer than in adult medulloblastoma tumors, which suggests that, compared to adult tumors, pediatric tumors may respond better to drugs that target the genes and pathways altered by mutations that drive cancer progression.

Pediatric brain and spinal cord tumors are difficult to diagnose because their signs and symptoms may mimic those of other disorders, and vary according to the exact location of the tumor. Once a diagnosis is made, successful treatment is also difficult because there are so many different kinds of brain tumors and cancers, and their precise causes are unknown.

Research holds the key to quicker, more accurate diagnoses and subsequently, better treatments. While progress is being made on a number of research platforms, it continues to be challenging for several reasons:

• There are many different kinds of children’s brain and spinal cord tumors, which has stymied research as investigators face the challenges of collecting and analyzing tissue, as well as the ethical issues posed in treating children.

• Because the disease is rare and tissue samples of tumors are small, it takes time to test and validate new treatment options, and there is currently not an adequate database for recording and sharing this information.

• Doctors and researchers are dealing with a growing child’s brain and body, so they must first ensure they avoid harming the child.

• Funds for research and treatment options are limited, due to the relatively low rate of incidence, compared to other childhood cancers and diseases.

As any research investigator would attest, much work has been done, much progress has been made, but it is not enough. It is never enough. The efforts will continue, and the unshakable commitment of so many will stand strong until the statistics dwindle from 3,400 to 0.