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Mary Flowers: Fighting retinoblastoma

When Mary Flowers was four months old, her parents noticed that she wasn’t focusing on her toys.  They worried that their baby couldn’t see. 

Andrea, Mary’s mother, took her child to the pediatrician, bracing herself for the news that her baby was blind.  “Cancer never crossed my mind,” Andrea said. But tests revealed that Mary did indeed have cancer, an eye cancer called retinoblastoma. About 300 children develop this malignancy every year in the United States.

And the place Andrea decided to trust with her precious daughter’s life was St. Jude Children’s Research Hospital.

Andrea explained, “At home, they have a children’s cancer center, but anybody I’ve ever talked to there had never heard of a child with retinoblastoma. And if they haven’t heard of it, then they haven’t treated it.  At St. Jude, I feel Mary is getting the best, most up-to-date care available.”

Because Mary’s cancer is advanced in both eyes, she is undergoing a very intensive treatment regimen that includes 11 courses of chemotherapy. So far, her tumors are responding very well, and our doctors are hoping they won’t have to give Mary radiation -- and that they won’t have to remove her eyes.

Despite aggressive treatments, many children do not respond well. Some eventually require radiation therapy, and many lose their eyes. And there’s another problem:  Radiation therapy has significant side effects on a child this age . . . and increases the risk that Mary could develop other cancers as she grows. 

Our research teams have been working day and night to develop better therapies with better penetration into the eye - - and in recent months, we have seen reason for significant new hope against this eye cancer.

Over the past four years, St. Jude researchers have been investigating a drug called topotecan in the treatment of retinoblastoma. Through our extensive study of topotecan in the treatment of many childhood cancers, we have found this drug to be among the most effective. 

We also have combined topotecan with other anticancer drugs, and when we tested the combination against current drug regimens, our findings suggest that combining topotecan with carboplatin (another very effective drug against retinoblastoma) is the most effective. 

But giving both drugs intravenously is simply too toxic for children with advanced solid tumors. That’s why we’ve developed an alternative: giving topotecan intravenously and administering carboplatin around the eye, as you would with eye drops. This unique treatment will allow us to give the patient the benefit of both drugs while avoiding the toxic effects on other organs.

The implications are dramatic. This emerging retinoblastoma therapy has the potential to save children’s lives, save the eye, and spare these children the toxic side effects of chemotherapy. Our discovery, which was first reported in the November 2, 2006 issue of the scientific journal Nature, is the first example of local delivery of a targeted chemotherapy drug for any childhood cancer.

In the near future, children with newly diagnosed retinoblastoma might not have to suffer as much as Mary has, and their families might not have to confront the uncertain prognosis she faces. And one day, St. Jude will reach its goal of finding cures and saving children.