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A recent example involved treatment with a new cutting-edge drug for brain cancer. This promising treatment for medulloblastoma brain tumors short-circuits tumor growth at a molecular level. The drug, HhAntag, is a signal transduction inhibitor (STI) which has been successful in treating about one-third of brain cancers in adults with non-toxic safe results. HhAntag targets the so-called “hedgehog” pathway and shuts down a signaling mechanism critical to the proliferation of cancer cells.
Unfortunately, the same “hedgehog” pathway regulates bone development and places pediatric patients at risk for bone damage and stunted growth. In a cancer treatment study involving juvenile mice, permanent shortening of the bones and alteration of joint structures occurred within two days of treatment with HhAntag. X-rays revealed that the treatment caused widespread skeletal, cartilage, and joint abnormalities, impaired growth, and weight loss. The damage was found to be irreversible.
Study co-author, Tom Curran, deputy scientific director of the Stokes Research Institute at the Children’s Hospital of Philadelphia, noted that this poses a serious problem for treating children. He believes that the harsh STI consequences observed in young mice would also occur in children. Curran and his team are publishing their findings in the March issue of Cancer Cell.
Medulloblastoma is the most common pediatric central nervous system cancer. Traditional treatments involve surgery, radiation, and chemotherapy. However, these treatments offer a survival rate of only five years in four out of five children. They also cause severe side effects including movement disorders and cognitive impairments. These traditional treatments are also less effective in children under three years and in those children whose disease has spread.
Curran would hope to find a way to deliver these drugs to the brain tumors of young patients without delivering it to their bones. However, because only a few hundred children a year are treated he is doubtful that drug companies will invest in the research.” He noted that they would still use HhAntag to treat terminal patients, as the side effects are certainly worth the risk.
As adoptive parents, I am sure we would want to take the risk of our child having a shortened stature if it saved the child’s life. Since this new treatment seems to work in adults without side effects, one would hope that they could find a way to successfully treat children with HhAntag.
Photo Credit
Attribution license creative commons
Nicolas Boullosa’s photo stream uploaded Feb 28, 2008
