There are only a handful of labs across the country like the one where Carolyn Lutzko, PhD, comes to work.
When Lutzko, director of the Cell Manipulations Laboratory, helps patients get well, it means she or members of her team go into a cleanroom to work with cells to bring therapies to the bedside.
That’s when it starts to look like something out of a science fiction medical thriller.
“When we go in to work with patient samples, we completely cover ourselves with a sterile suit and have to actually maneuver into it without touching anything—the floor, the walls—anything to make sure that we are kept sterile,” Lutzko says.
The lab manipulates cells for researchers working on clinical trials studying blood diseases ranging from sickle cell anemia to severe combined immunodeficiency.
Once researchers have blood or bone marrow stem cells from the patient, they alter the cells by introducing a gene that corrects the deficient, disease-causing gene. Those corrected cells are allowed to proliferate. Once safety testing is complete, a syringe containing the corrected cells is delivered to the patient, where the cells are injected just like a medicine.
“What is really innovative here is that we’re actually working on treatments focused specifically at the DNA or the genetic level,” Lutzko says. “So for a genetic disease, rather than trying to find a drug or something that can mimic what the gene is supposed to do, we are actually giving the patient a healthy copy of that gene. So it’s really a simple form of therapy: Let’s give the patient the DNA that they need.”
In some cases, like a severe combined immunodeficiency trial, that has meant seeing good outcomes—like a patient who spent the vast majority of their life in the hospital being able to go home.
The most fulfilling part of the job, Lutzko says, is working as a team to bring cutting-edge research from the basic science labs at Cincinnati Children’s to the patients.
“It’s a really unique ability and one of the things I’m really proud of,” she says.