Rebecca Lilly's brain tumor stubbornly persists.
James A. Parcell--The Washington Post

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For Rebecca, there is no clear choice for medical treatment. Instead she and her family must choose from a variety of experimental options

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Rebecca Lilly Takes the Next Step

R

ebecca Lilly didn't get what the big deal was. Why did she have to go to a church banquet when she felt so crummy?

She was on the 13th floor of the National Institutes of Health Clinical Center in Bethesda, and she was throwing up. Her dad had brought her in for a blood test because the next morning she was due to start a new round of treatment for her brain tumor.

All day she had felt headachy and blah. The thought of another needlestick didn't help. When she got sick to her stomach they put her to bed with an intravenous line in her arm, and now she just wanted to sleep.

So why was Dad insisting they drive to Silver Spring for dinner if she couldn't even keep lunch down? Besides, didn't they already hold that CYO thing?

"SURPRISE!" As Becca pushed open the door to the St. Bernadette's parish hall, more than 100 people erupted into a standing ovation. Her 4-year-old sister Sarah made a beeline for her and delivered the first hug. The disc jockey dished up "Only Sixteen," as Becca, wide-eyed and smiling, waded into the jubilant crowd.

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inutes later, she was out on the dance floor, boogieing with the rest.

"She looks a lot better now," said her dad, Joe Lilly, looking a little relieved himself.

Becca Lilly, of Takoma Park, who turned 16 on July 18, has lived with brain cancer for five years. She has undergone open-brain surgery four times -- twice with follow-up radiation. Each time, the tumor has grown back. Becca has a high-grade malignant glioma, one of the most relentless and hard-to-treat tumors. She has beaten steep odds already; of the children diagnosed with that type of tumor, only one in five is alive three years later.

Rebecca Lilly blows out the candles at her surprise 16th birthday party.
James A. Parcell--The Washington Post

Last November, Becca became the first child to undergo gene therapy for a brain tumor. In a nine-hour operation at Children's National Medical Center, surgeons removed her tumor and then injected into her brain a harmless virus carrying a gene designed to trick any remaining tumor cells into self-destructing. Gene therapy did not work a miracle for her. A brain scan in May showed that the tumor had returned.

On May 28, surgeons at Children's operated again on Becca, knowing that this time they could not remove the whole tumor without causing unacceptable brain damage. The hope was to "debulk" the tumor, relieving pressure on her brain and buying time for an experimental therapy.

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hen Becca and her parents, Joe and Maureen Lilly, faced an agonizing question: What next? Recurrent brain cancers -- tumors that grow back despite treatment -- often do so with malignant abandon. The Lillys and their doctors had to decide soon what else -- if anything -- to try.

Always before, there had been a clear medical choice, a therapy that stood out from the others, offering at least a reasonable chance of fending off the tumor for a while. This time was different. The phrase "reasonable chance" seemed misplaced, or cruel.

"This is a desperate field," said neurologist Roger Packer, director of the brain tumor program at Children's.

When a child has a life-threatening illness and her doctors have run out of proven options, how do parents choose?

On the Internet, at the library and in detailed discussions at Children's and NIH, the Lillys investigated numerous possible experimental therapies for Becca.

At Packer's suggestion, they visited NIH in June to discuss two unproven treatments being tested in studies, or protocols, that Becca seemed eligible for. One combined a proven anti-cancer drug -- carboplatin -- with a new substance called RMP-7 designed to help it get inside the tumor. The other was phenylacetate, a natural "maturing agent" that in very high concentrations had shown some ability to disable tumor cells by making them mature too fast to multiply.

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oth treatments were in the earliest stage of testing in children. One had been tried in 17 children; the other in none.

"It basically comes down to: They just don't know," Joe Lilly said after the NIH meeting. "Crapshoot" was a word both Joe and Maureen Lilly would use over the next few weeks.

Each year, in laboratories at NIH and around the country, researchers supported by the National Cancer Institute check about 10,000 chemical compounds for possible anti-cancer activity. Only about a half dozen show enough promise to merit further testing in humans -- a Phase 1 clinical trial.

Rebecca follows the lead of her sister Anne Marie, left, dancing the Macarena at her 16th birthday party.
James A. Parcell--The Washington Post

Phase 1 trials are designed to observe a promising drug in human patients for the first time and determine the safe dose. Phase 2 trials measure effectiveness by testing that dose on several dozen patients with the same diagnosis. A drug that shows good results in Phase 2 moves on to a much larger Phase 3 trial, where it is tested against existing treatment in hundreds or even thousands of patients, to see if it can match or outperform standard therapy.

Unfortunately, the only experimental drugs for Becca's tumor were in Phase 1, their effectiveness still unknown.

What made the Lillys' choice all the more frustrating was that Becca was feeling quite well, better than at any time since her May surgery. She had bounced back impressively from an operation that not only wore her out but triggered several seizures and temporarily disturbed her balance, memory and ability to use language. At first, Becca had trouble remembering her sister's name. Her conversation was halting and she mixed up common words.

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ut two weeks out from surgery, she attended a family wedding in Maine. With the help of massive intravenous antibiotics, she fought off an attack of spinal meningitis from an infection in her incision.

By early July, Becca was once again active and steady on her feet. She regained command of her conversation. She was more "with it," more expressive, more like herself. She couldn't wait to complete the antibiotic course and get to the pool or the beach.

"Look at me," she said one day in the clinic waiting room at NIH. "I'm so disgusting! I've got like no tan."