New drugs and therapies are approved for general use in the U.S. after being tested first on a select few, in what are called clinical trials.
Every new drug or therapy for every kind of illness from depression to the flu needs to go through these lengthy trials to ensure effectiveness and safety.
But for those with serious illnesses and no other options, taking part in a trial has added urgency, as one family from Louisiana found out this year.
For Casey and Stacy Henagan, the results of a brain scan on their baby daughter Haven were hard to take. "It was like, 'what do you mean it's not good news, because here's our child, she looks perfectly normal - how can you say there's a tumor the size of a small tangerine in the back of our one-year-old's head?' It was tough."
The diagnosis was malignant brain cancer. Haven underwent immediate surgery to remove the tumor. But the cancer would almost certainly return without more treatment.
The Henagan's surgeon told them about a clinical trial at St. Jude's Children's Hospital in Memphis.
At St. Jude's, Dr. Richard Heideman explains that the new combination of chemotherapy and radiation being tested holds promise, but there are no guarantees and many risks.
"One of the unfortunate side effects is that it can cause some decreased hearing and it's quite variable," Heideman explains. "Some people have mild hearing loss. Some people have severe hearing loss requiring hearing aids."
He told the Henagans, "I can virtually guarantee there will be some degree of hearing loss, hopefully not badÂ… We'll be checking her hearing after every cycle of treatment."
The Henagans are willing to take the risk, even though they don't know whether the treatment will work. As Casey Henagan puts it, "What is her life expectancy? Will we have her past five, past ten? Some of those questions were answered by 'we don't know because this is such a new procedure'."
"I know that nothing's guaranteed. We just have to try it," Stacy Henagan says. "To us in our minds it's the best thing that we can do for her besides prayer, and with God in control we know she's going to be okay."
The success or failure of Haven's treatment may not be known for years. From a scientific standpoint, she is simply a statistic in a trial. But for her family and her doctors, she is a precious one-year-old.
"This isn't experimentation," says Dr. Richard Heideman. "This isn't like treating a guinea pig. If we do nothing the child will surely die of her disease in several months time, and if we do something we have the chance to make this child a long-term survivor."
While he long-term odds are against her, there is hope for Haven in the unknown, and hope that her tiny role in this trial will be a bigger step to help others.
"If this can help some children in the future then we're going to be very excited even if our child does not make it," says Casey. "If they take this and better it and the percentages gradually get stronger for the child being healthy, then it will definitely be worth it for us."
Haven is doing well so far with her chemotherapy. Depending on her progress, she will receive a new radiation treatment as part of her therapy.
How can you find out about clinical trials?
Ask your doctor if he or she knows about any studies being done. For cancer trials, the best-known U.S. cancer treatment centers are a good information resource. The National Institutes of Health provides information about clinical trials nationwide. You can get information on specific trials at the Clinical Trials Database at the National Institutes of Health site.
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