Medical Miracles

Astonishing stories of patients who cheated death or permanent disability, and the physicians who saved their lives.



(page 4 of 8)

Dr. Regina Jakacki can’t stifle a smile when she talks about Leah Koller. Her smile often becomes an out-and-out grin. That’s because of how Leah’s doing. At 12, eight years after she was diagnosed with a glioma — a complicated form of brain tumor — the Waynesburg girl is thriving, thanks to an experimental treatment spearheaded by the Brain Care Institute at Children’s Hospital of Pittsburgh of UPMC.

The team included Dr. Jakacki, director of the neuro-oncology program, and her colleague, pediatric neurosurgery chief Dr. Ian Pollack. The treatment that has been working for Leah is, in essence, a vaccine — not entirely unlike a vaccination against mumps or measles — that attacks certain proteins in the tumor. A team at the University of Pittsburgh and Children’s developed the vaccine, which is the first of its kind in the country.

Because of the tumor’s location, doctors knew that removing it would cause her to be paralyzed on her right side. And it also appeared that Leah’s tumor was spreading throughout the brain, which made surgery an even less viable option. Radiation therapy to the brain can cause severe developmental damage in young patients. So last year, with the Kollers’ blessing, they turned to the experimental vaccine.

The idea of using vaccines came out of a longstanding program that examined how they’d affect malignant brain tumors that weren’t responding to standard therapies like surgery, radiation and chemo, which aren’t curative. Doctors at Children’s suspected that immunotherapy might work better. So they looked at the antigens on the tumor cells and identified several targets — what Dr. Pollack calls “little snippets of protein” that could be used as a vaccine. An early study in adults was encouraging, so Drs. Pollack and Jakacki moved on to children, who tend to have more robust immune systems.

At first, in 2009, even the doctors were skeptical about the vaccine. “I wasn’t a complete believer,” Dr. Jakacki recalls. “I wasn’t even sure people were going to come for it.” For starters, a patient needed to have a certain tissue type to be eligible — a type that is found in only about 40 to 45 percent of the general population. “Initially, it sounded like a pie-in-the-sky kind of thing,” Dr. Jakacki says.

Not much happened at first, and few volunteers came forward. Then enrollment peaked, and progress surfaced in a few patients. One child got much worse during the treatment, and the tumor looked much bigger. However, throughout the next several weeks, the child improved, and an MRI showed that the tumor had shrunk to less than half the size it had been, indicating that the tumor had swelled as the vaccine attacked the tumor cells — but the vaccine was indeed working. Says Dr. Jakacki, “We kind of looked at each other and said, ‘Really?!’”

Leah’s tumor was low-grade and slow-growing yet complicated; it started expanding into the coating of the brain. Leah was put on chemo, but the radiation that could have really tackled the tumor would have also affected her cognition. When she started the vaccine, she had few side effects. But an MRI taken after nine weeks showed the tumor actually seemed worse. They kept going. Then it looked stable. Then, slowly, it shrank — enough that its shrinkage finally became obvious.

Now, Leah’s tumor is about 50 percent smaller — 75 percent, Dr. Jakacki says, if you take into account dramatic shrinkage in the areas of metastasis. Twenty-seven children have tried the treatment, and a dozen remain on it. And Drs. Pollack and Jakacki are looking at ways the vaccine usage might be expanded. That, too, makes Dr. Jakacki grin.

Meanwhile, Leah is starting to flourish.And there’s a new member of the Koller clan these days, not related by blood but rather sweat and tears: Dr. Jakacki, who finds herself an honorary Koller for life.

“She’s just part of our family, whether she wants to be or not,” says Leah’s mother, Raelene. “She’s part of us now.”
— Ted Anthony

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