It was 2005, and Beth Anne Baber was seven months pregnant with her third child when she got the kind of news no mother wants to hear. Her second child, Nicholas Conor, had been diagnosed with an inoperable tumor in his chest. He was 15 months old.
Like other parents living the same nightmare, Baber confronted a nerve-wracking dilemma. And as a cancer researcher in a lab at the Salk Institute, Baber was all too familiar with the insidious disease.
“Because both my husband and I are scientists, we really took our scientific background to his treatment,” Baber said. The doctor recommended a bone marrow transplant. Baber and her husband agonized over the fateful decision and eventually refused. They didn’t feel it was warranted.
After enduring multiple rounds of chemotherapy and other treatments over a two-year period, Conor survived. His treatment ended in 2006; he’s nearly six years old and healthy today. But the ordeal changed Baber’s life.
Motivated by her son’s close brush with death, Baber, 44, co-founded the Nicholas Conor Institute, a fledgling pediatric cancer nonprofit that launched here last October. By focusing specifically on children’s cancer, Baber and her colleagues aim to translate research into better treatments.
Developing new drugs and diagnostic tests is often tremendously expensive, and diseases that afflict a small number of patients — like childhood cancers — don’t always offer enough financial return to attract investment. Baber hopes her organization can help find funding and cut the cost of developing new approaches.
Cancer is most common in the elderly, although roughly 11,000 children under age 15 in the United States are diagnosed with it each year, according to the National Cancer Institute. Mortality rates have been cut in half over the last thirty years, but children remain far more vulnerable than adults to the toxic side effects of chemotherapy.
Unfortunately, there are few viable alternatives — as Baber discovered when her son was diagnosed. “I was taken aback when they told us the types of treatments that were available — basically the standard chemotherapy that’s been available for 30 years,” Baber said.
Together with Martin Latterich, a scientist she knew from the Salk Institute, and other local researchers, Baber began to discuss starting a research facility that would specialize in pediatric cancer. “We looked at the possibility of building a standalone institute like Salk or Scripps Research, but the cost was too high,” Baber said. By late 2008, when the idea was hatched, the economy had slumped into a tailspin, and the chances of finding sufficient funding for such an ambitious venture seemed slim.
So Baber and Latterich decided on an “institute without walls” instead — a group of researchers and doctors that collaborate with biotech companies to advance promising tests and treatments.
By collaborating on the same project, academic labs could potentially help biotechs develop new technology faster and at a lower cost than if the company goes it alone. Academics can lend their expertise to overcoming technical challenges; Baber’s organization can also file joint grant applications with biotechs to help them find funding.
“Essentially, we become their human resource department,” Baber said.
At this point the organization relies on volunteer efforts, donations and assistance from supporters, although Baber said they are working on an agreement with a cancer foundation and are pursuing government research grants. They count several Salk and UCSD scientists among their board of directors and are already working with a few biotech firms, including AltheaDX, a small, privately owned San Diego company that produces a genetic test for a class of pediatric cancers that are tough to differentiate.
The company has been working on a new test for three years, said Cyrus Mirsaidi, a company vice president. But it hasn’t yet been able to get it adopted by doctors in general practice. And finding enough samples to design and validate tests is challenging for rare children’s cancers since there are few patients. Mirsaidi says Baber’s group is helping to access samples from doctors and raise awareness among doctors so that the test can become standard.
Assuming she can expand her organization, Baber wants to use genetics to customize treatment for individual children. Genetically, every cancer — like every patient — is unique, and even within a tumor cancer cells can differ from each other.
Depending on which mutations are present in a given cancer, it might exhibit targets that other cancers do not. By testing for these mutations, doctors could improve their ability to differentiate between cancers that respond to various treatments. Baber’s group is working with a Bay Area biotech on genetic tests of cancer cells to determine which treatments are most likely to make an impact.
Genetic testing could also help minimize toxic side effects of chemotherapy drugs. Some patients are more susceptible to side effects of specific drugs than others, depending on their genetic profile. “Eventually, we want to be able to test to determine how patients will metabolize each of these drugs,” Dr. Willert said.
Personalized medicine, or tailoring treatments for individual patients, is an aim across medical research today. But the struggle is very much a personal one for Baber. Her own experience has taught her how harrowing an ordeal cancer in a child can be. It’s her hope, she said, that she can ensure other children with cancer survive to become as healthy as her son is today.
“He’s doing very well,” Baber said. “We’ve been blessed.”
Jonathan Parkinson is a San Diego-based freelance writer. Please contact him directly at firstname.lastname@example.org.