A researcher will do a lot for grant money, the fuel necessary to power a laboratory’s work. Sam Volchenboum, a pediatric oncologist at the University of Chicago Medical Center, took that adage to its follicular extreme last week, volunteering to go bald for funds from the St. Baldrick’s Foundation.
St. Baldrick’s, a California-based organization which raises funds for pediatric cancer research, asks their volunteers and award recipients to shave their heads in solidarity with cancer-stricken children who have lost their hair to chemotherapy. Thursday evening, Volchenboum went under the razor himself, shedding his dark brown hair with the help of Joshua Crosby, a 13-year-old cancer survivor. A small price to pay, Volchenboum said, for a $330,000 award that will help him design faster and more specific diagnostic tools for neuroblastoma.
“It can often take a while and be a little frustrating to get to the diagnosis,” Volchenboum said. “Despite all we know about this disease, even with aggressive treatment – chemo and radiation and surgery – over half of the kids will still die from their disease.”
Despite what its name implies, neuroblastoma is not brain cancer, but rather a cancer of the sympathetic nervous system that connects the spinal cord to organs of the body. Though it’s rare as far as diseases go – with only about 800 new cases a year in the US – it’s nevertheless the most common solid-tumor cancer seen in children and is responsible for about 15% of childhood cancer deaths. But not all neuroblastomas are fatal; in fact, some tumors in infants even regress spontaneously without treatment. That wide variation in prognosis presents a challenge to oncologists, Volchenboum said, who must decide the best course of treatment for a child with neuroblastoma, doing as much as possible to attack the tumor without over-treating with therapies that can be toxic and harmful in an adult, never mind a growing kid.
“We need to be able to sub-stratify the patients to predict outcome better,” Volchenboum said about the goals of his project. “There are probably some patients that will do poorly despite any conventional treatments, so let’s give this patient emerging therapy, let’s try something new. Likewise, there are some patients with apparent high-risk disease who get lots of therapy and are ultimately cured but may not have needed all that therapy.”
Currently, oncologists classify a patient based on their age, if the tumor has spread, and the status of a gene called MYCN – amplification of this gene is “the one of the best predictors of poor outcome,” Volchenboum said. Assembling all this information requires special tests and a biopsy, meaning that it can be several days before doctors have all the information they need to assess the cancer and plan the appropriate treatment. And once that treatment program is established, it’s difficult to monitor how well it’s working other than through coarse, indirect measurements.
Volchenboum’s research project hopes to refine that process via a method called proteomics, measuring the type and number of proteins present in neuroblastoma cells to improve and quicken tumor diagnosis. That goal, Volchenboum explains, will require a blend of traditional cell biology and newer computational methods. In his laboratory, Volchenboum will observe the thousands of proteins produced by tumor cells that do or do not produce many copies of the MYCN gene. Subtle differences between those “protein signatures” can then be analyzed to create a test that will distinguish between the protein levels observed in high-risk and low-risk tumors.
Such a test would probably still require tumor cells at first, meaning a biopsy will still be necessary for diagnosis. But eventually, Volchenboum hopes protein detection (using a machine called a mass spectrometer) will become sensitive enough to measure protein levels in blood, allowing for a rapid proteomic diagnosis. In addition to the work on neuroblastoma tumors in the lab, he is also designing software to speed up this analysis of proteomic data, hopefully some day leading to “real-time” characterization of a patient’s tumor or blood sample. Doctors could then tailor the proper treatment to each patient’s tumor…and even check back on a regular basis with proteomics testing to see if the treatment is working.
“There may be ways to look in the blood for the presence of proteins associated with tumor spread to understand even while on treatment, ‘is this therapy working?’,” Volchenboum said. “Right now we give patients several months of therapy and re-evaluate; would it be possible to check their blood after a month and understand if the therapy you’re giving is affecting the tumor the way you think it is?”
After Thursday’s St. Baldrick’s event, Volchenboum will be initially conducting his research with a much more aerodynamic hairstyle, a change that will also help him when he runs the Minneapolis and Chicago Marathons next month.
“I’m a little bit nervous, if only for vanity reasons,” Volchenboum joked about his new look. “I’ve spent years telling kids their hair always grows back, so I guess I shouldn’t complain.”