A blow-by-blow look at how Colorado doctors and researchers are taking the fight to cancer right now.
—Illustration by C.J. Burton
One More Last Chance
Jeff and Linda Lambertson say they should’ve noticed that their two-and-a-half-year-old daughter Katie was lethargic and sleeping restlessly. Maybe they could’ve noticed she was a little pale and bruising easily. But hindsight, as they say, is always clearer. Instead, the Danville, California–based parents of three daughters didn’t know anything was wrong until Katie threw up after dinner one night. “Honestly, we took her to the pediatrician the next morning,” Jeff says, “because we had a nice weekend coming up that we were looking forward to.” He laughs and says in a soft, loving tone, “If only we’d known what was coming.”
The family’s pediatrician immediately latched onto the little girl’s pallor and bevy of contusions and ordered a blood test. When the results came back the following morning, the pediatrician sent the Lambertsons to a nearby hospital where doctors told them Katie had cancer and suggested an immediate medevac transfer to Children’s Hospital Oakland. A normal white blood cell count for a child is between 5,000 and 10,000 per microliter; Katie’s was 750,000. The Lambertsons’ tow-headed toddler had full-blown acute lymphocytic leukemia (ALL). At Children’s Hospital Oakland, Katie had to get blood transfusions just so doctors could do necessary procedures. “They told us we might lose her in the first 24 hours,” Jeff says.
Katie made it through the night, but the Lambertsons’ bad-news-laden journey had just begun. Doctors explained that Katie had a rare form of the cancer called T-cell ALL, a more aggressive, less-well-understood subtype that affects only about 15 percent of ALL patients. She would need chemo, and quickly.
An initial regimen of chemo usually puts pediatric patients into a temporary state of remission. But not Katie. After 30 days, her parents moved her to Lucile Packard Children’s Hospital at Stanford, where doctors were standing by to tackle Katie’s unruly form of the disease. After more chemo, doctors got the tot into a tentative medical remission and then recommended a bone marrow transplant as the only way to save Katie’s life. Her siblings were not matches, and although a donor was found, the match was not perfect. The Lambertsons and Katie’s doctors decided it would have to be good enough: Katie couldn’t wait. Five months after her diagnosis, Katie underwent total body irradiation to kill her immune system and then went through the transplant. It failed 100 days later. “We were at the end,” Jeff says. “She couldn’t get another transplant and there was nothing left doctors there could do.”
The Lambertsons decided that wasn’t acceptable. Even as they were hoping the transplant would work, they had been researching for the worst-case scenario. They found a clinical trial for T-cell ALL patients for a drug called forodesine, manufactured by BioCryst Pharmaceuticals. But there were obstacles: The trial had not yet been fully opened to pediatric cases; Katie had to be very sick, but not too sick; and few hospitals would even participate in what many considered experimentation on kids. “We moved heaven and earth to get her into the trial with BioCryst,” Jeff says, “but until the trial organizers suggested Dr. Lia Gore at Children’s Hospital Colorado’s Experimental Therapeutics Program, we didn’t know how we were going to get Katie the only drug we thought might work.”
Like almost every family that shows up to Gore’s clinic, the Lambertsons had been told their child was going to die. And like almost every parent Gore sees, Jeff and Linda needed to be absolutely sure they had exhausted every possibility to save their child. Gore founded the Experimental Therapeutics Program in 2003 to be the place families like the Lambertsons could come to. “It’s a privilege to take a family through this journey,” says Gore, whose vision it was to create a center where new treatments for kids with cancer could be tested and developed, “and how we do that is the ultimate challenge for me. Every day something comes up that wasn’t anticipated, so it’s really about how we move forward with each family and how we learn along the way to make it better for the next family.”
Ten months after her diagnosis and a few months after her transplant failed, Katie found herself on a clinical trial for forodesine under the care of Gore. There was little to no data on the drug in kids, and there was scant information on dosages, but Gore worked with BioCryst to get it right for Katie, the youngest patient ever to be given the drug.
That was nine years ago. After just two weeks on forodesine, Katie achieved full remission. After six months, she went off the drug completely—and has never relapsed. Today, Katie is a healthy 12-year-old who gets A’s and B’s in school, likes volleyball, and is learning to play golf. She has zero memory of the pain she experienced as a toddler, but she remembers Gore. Jeff and Linda remember Gore, too. “Dr. Gore is second to none,” Jeff says. “She and her team are with you 24 hours a day, and they made sure everything was always ready to go for Katie.”
All these years later, the Lambertsons are still hyperaware their daughter’s recovery was unlikely—that she is an outlier. Katie may be an anomaly in the grand pantheon of cancer statistics, but she is much like many of Gore’s other patients: kids who weren’t given a chance by anyone else and who, with the doctor’s help, beat astronomical odds. “There are a lot of kids whom we still can’t cure,” Gore says, “but childhood cancer is infinitely more curable now than 10 or 15 years ago. And that’s only because there are people like the Lambertsons who are willing to take a chance on experimental therapy.”
The Long Run
Surviving Childhood Cancer
In the ’60s, defeating pediatric leukemia was nearly unheard of—in fact, only five percent survived the onslaught of cells. Today, however, the cure rate frequently tips 90 percent. That upswing is one of modern medicine’s biggest success stories. But as pediatric cancer survival rates have continued to climb, a different kind of medicine has become necessary. “Kids weren’t living long enough back in the day for us to learn about the long-term side effects of cancer treatment,” explains Dr. Brian Greffe, medical director of the HOPE Survivorship Program at Children’s Hospital Colorado. “Now we know there are things that require follow-up care.”
Chemotherapy, radiation, surgery, and other newer treatments for childhood cancer can cause a host of long-term side effects: infertility, bone tissue death, heart and lung complications, cognitive impairment, endocrine issues, kidney damage, and secondary tumors. The problem is only about 20 percent of pediatric cancer survivors who are now adults get care to prevent, diagnose, or address problems that can arise years after treatment stops. Why? “We’re still educating doctors to look for symptoms and talk with patients about these issues,” Greffe says. “Plus, many patients have anxiety about going back to the doctor, or they simply don’t have a general practitioner once they get into their 20s or 30s.”
Greffe explains that even if a patient hasn’t had any complications, it’s a good idea to set up an appointment at the HOPE clinic—or at the adult-specific TACTIC clinic, an extension of HOPE located at University of Colorado Hospital. “The book of side effects is hundreds of pages long,” Greffe says, “but that means we know what to look for and we can often help before something goes wrong.”