San Diego’s Rady Children’s Hospital recently brought together an all-star cast of speakers for a symposiumon pediatric translational research – laboratory research that could be “translated” into therapies for sick children. This was the ideal place to bring laboratory scientists together with physicians. The close connection to patients was clear, as kids played in the hall outside the conference room, paramedics checked out the posters and doctors used the wall phones to return pager calls and provide consultations.What did they talk about? A little bit of everything that’s hot in pediatric research: brain cancer, leukemia, rare genetic diseases, stem cell therapies and re-wiring the immune response to fight disease.
One of Sanford-Burnham’s newest recruits, stem cell expert Dr. Robert Wechsler-Reya, was there. Dr. Wechsler-Reya hasn’t finished his move to San Diego yet, but he did not want to miss the opportunity to connect with his colleagues in stem cell research and pediatric medicine.
“This is an exciting time for me,” Dr. Wechsler-Reya said as he began his presentation. “I’m in the process of moving my lab and family to San Diego, so it’s fun to get this opportunity to meet new people and see some of the connections I’ll be able to make once we’re officially at Sanford-Burnham.”
Dr. Wechsler-Reya went on to speak about personalized medicine. In cancer research, personalized medicine describes the notion that every tumor is different. If we could simply profile every person’s tumor on a genomic, proteomic and metabolomic level, we’d be better able to develop more appropriate and less toxic therapies.
But, according to Dr. Wechsler-Reya, “If we’re going to develop improved therapies, we need to develop not just personalized medicine, but personalized animal models.”
Personalizing mouse models for a particular tumor type is a major goal of Dr. Wechsler-Reya’s research. His talk centered on his “skip the dish” approach to studying a childhood brain tumor called medulloblastoma. In addition to studying genetically-modified mice that develop medulloblastoma, Dr. Wechsler-Reya’s group also collects human medulloblastoma samples removed during surgery and transfers them straight into mice within a couple of hours. This approach has led to mouse models for several different types of medulloblastoma. Unlike in a dish, here the tumor cells maintain their biochemical properties, allowing researchers to study the role stem cells play in brain development and tumor formation in a more realistic context.
“We’re trying to take information we’ve garnered from studies of human disease and convert it into animal models that will allow us to test hypotheses about the biology and novel approaches to therapy,” Dr. Wechsler-Reya explained.
Sanford-Burnham was also represented at the pediatric symposium by Dr. Robert Rickert and Dr. Carl Ware.