The fact that childhood cancer is, thankfully, rare belies the fact that it is the leading cause of disease-related death in U.S. children age 1 to 19. The number of people with a direct stake in expanding research into pediatric cancer is quite large, well beyond the small number of children with cancer and their families. Not only are the life-long contributions of children cured of cancer enormous, but understanding cancers of young children could also hold the key to understanding a broad range of adult cancers. The time is ripe to allocate more resources, public and private, to research on pediatric cancer.
In an age of increased understanding of the genetic basis of diseases, one thing is striking about many childhood cancers. They are relatively “quiet” cancers, with very few mutations of the DNA. Young children haven’t lived long enough to acquire the large number of mutations that create the background “noise” associated with years of living. This makes it much easier to pinpoint the relevant genetic abnormalities in a young child’s cancer.
Add to this the growing realization that biology, including how various tumors use common “pathways,” is a major factor in how the cancer responds to treatment. Thus, a mechanism that’s relatively easier to observe in the cancers of young children could help scientists understand cancers in adults, in whom the same mechanism is hidden amid the clutter of mutations acquired over a longer life. …
Fifty years ago, a baby born to President John F. Kennedy and his wife, Jacqueline, died after just 39 hours of life. Born just slightly premature at 37 weeks and weighing only 4lbs, 10½oz, Patrick Bouvier Kennedy developed respiratory distress syndrome, then a frequent cause of death and long-term lung disability in infants born prematurely. At the time, this condition killed about 25,000 children each year.
Today, thanks to National Institutes of Health (NIH)-funded research, we know that babies born prematurely or at very low weights may lack an important protein called surfactant in their lungs. This discovery led to the development here at Boston Children’s Hospital by Mary Ellen Avery, MD, of surfactant replacement therapy, which has revolutionized the care of premature infants. As a direct result of research into this pediatric disease, premature infants born as early as 23 or 24 weeks are viable. The incidence and severity of respiratory distress is extremely reduced: Fewer than 1,000 babies will die from it in the United States this year.
WE WANT YOU! Tell your senators to sponsor the National Pediatric Research Network Act. Here’s how.
Gene therapy, still experimental but beginning to enter the clinic, attempts to utilize advanced molecular methods to treat and even reverse genetic diseases. The field started in earnest about 25 years ago and has had many setbacks along the way to its recent earliest successes.
International collaboration has been critical. Children’s Hospital Boston is one of the founding members of the Transatlantic Gene Therapy Consortium (TAGTC), a new collaboration that seeks to facilitate a more rapid advancement of this technology for treating human diseases. It was initiated shortly after the first trials of gene therapy for X-linked Severe Combined Immunodeficiency (X-SCID) (in both Paris and London) reported leukemia as a serious side effect. The TAGTC was formed to address this setback, developing safer gene therapy reagents, sharing the costs of their development, and then implementing new gene therapy trials for rare diseases across multiple international sites. …