Becca moved on to a checkup by neurologist Packer, who seemed delighted with her reflexes, vision and balance.

"How are the words coming?" he asked.

"Better."

As her parents comfort her, the last stitches from Rebecca's brain surgery in May are removed.
James A. Parcell--The Washington Post

"Can you say, `No ifs, ands or buts'?"

She nailed it, first try.

"You've been practicing," he said in mock accusal.

Busted! Becca smiled and shrugged sheepishly.

"You look wonderful," Packer said. "I think you're doing great." It was amazing, he said, how she had shaken off the meningitis.

He asked if she had any questions -- she didn't -- and then explained that he needed to give her parents a turn. While Becca sat paging through Vogue in the waiting room, Packer and the Lillys talked.

F

or nearly an hour, they weighed ponderables and imponderables: doses, response rates, age limits, side effects. They discussed half a dozen treatments, but focused on the two experimental chemotherapy protocols at NIH: RMP-7 versus phenylacetate.

"I don't want to mislead you," Packer told them. "None of these is a panacea. I don't know what will work."

To a patient or a parent considering such protocols, the fact that jumps out is their frighteningly low success rate against the cancer -- often between zero and 5 percent. Packer tried to explain that judging a Phase 1 trial by its "response rate" could be misleading. Those preliminary studies were designed not to assess the effect on tumors, but to test toxicity and determine the safe dose.

"You were at a hard point when we decided to do gene therapy. Maybe you're at a harder point now."

There were no new brain tumor drugs in Phase 2 testing, let alone phase 3, and the Lillys couldn't wait around.

"How much time do we have?" Maureen Lilly asked.

Another imponderable.

"My gut feeling is, you have to do something as soon as she stabilizes -- if you're going to do anything," Packer said.

Given that her tumor was in the part of the brain that controls speech and vision, Maureen Lilly asked, would it cause her more problems soon?

Y

es, if the tumor kept growing, Packer said. They had bought some time by removing perhaps 80 percent of the tumor by surgery in May. "But sooner or later that runs out." After the last surgery, he noted, they had seen some of what can happen.

"You're really at a hard point," Packer said. "You were at a hard point when we decided to do gene therapy. Maybe you're at a harder point now."

They compared the two main choices.

Phenylacetate was slightly further along in testing -- it was near the end of Phase 1 in children, whereas RMP-7 was just starting. Its side effects were milder -- mainly sleepiness and lethargy. But it would keep Becca hooked to a portable IV pump for 28 days in a row. And it wasn't clear -- even if phenylacetate worked -- that it could do more than stabilize the tumor. At best it would slow the growth, not kill the cancer.

RMP-7, given in a "big bang" monthly dose followed by several weeks off, would likely drop her blood counts low enough to put Becca in the hospital. RMP-7 had never been tried in a child; Becca would be the first. She would get a lower dose than had been tried in adults, to make sure it was safe in children. The Lillys worried that the lower dose might not be sufficient to have maximum effect.

T

he RMP-7/carboplatin combination has the typical toxic side effects of standard chemotherapy, such as nausea, mouth sores and hair loss. On that basis alone, her parents knew, Becca was leaning toward phenylacetate.

Packer made clear that either choice was medically responsible. If he had a therapy available that was clearly superior, he would nudge them toward it. He didn't.

Meanwhile, he would order another brain scan to see how fast the tumor was growing. Packer shook hands with them and left the room. Maureen Lilly stood up and looked at Joe and shrugged.

"Flip a coin?" she said.

The question every parent asks about a Phase 1 trial, said Peter Adamson, a pediatric cancer specialist at NCI, is one he cannot answer: What's the chance of this working for my child?

"Families have to decide ethically what they are willing to go through looking for this unproven cure for their child."

Adamson aims for the fine line between hope and false hope. "There's always excitement about new drugs, but you have to temper that excitement with the knowledge of how difficult the disease of cancer is," he said.

Adamson also tries to reassure parents that in such cases there's no incorrect choice. "Since we don't know which drug is more effective, or even whether either is effective, you don't make the wrong decision by picking Drug A instead of Drug B," he said.

"You only try an experimental therapy if there's no standard treatment available and you have reason to believe the experimental drug might help," Packer said. "Those are the guideposts. You don't want to hurt anyone and you don't make anyone a guinea pig."

When medicine lacks a proven weapon against a tumor, as in Becca's case, quality of life considerations loom larger than ever. Decisions about experimental protocols often turn on questions about side effects, scheduling and lifestyle.

"We're in very uncharted waters," Packer said. "Families have to decide ethically what they are willing to go through looking for this unproven cure for their child." Different families have different priorities.

The Lillys' decision took into account such factors as Becca's fear of losing her hair, her upcoming birthday and the potential disruption of the family's vacation at Ocean City, Md.