A gene-editing technology that made headlines recently for successfully treating a baby with leukemia may one day be used to treat other types of cancers, experts say.
Layla Richard was just 14 weeks old when she was diagnosed with acute lymphoblastic leukemia (ALL), according to the Great Ormond Street Hospital in London, where she was treated. Though doctors had previously tried several treatments, nothing had worked.
The hospital offered Layla's family the chance to try out an experimental treatment that had only been used in mice. The researchers took blood that had been donated, and separated out the white blood cells called T-cells, which normally help to fight infections in the body. Then, they edited the cells' genes, which gave the cells the ability to attack the cancer, and injected the cells into Layla.
In the future, this same technique could be used to treat people with other blood cell cancers, such as chronic lymphocytic leukemia and myeloma, Dr. Waseem Qasim, one of the doctors at the hospital who worked on Layla's case, told Live Science.
And although it will be more difficult to use the treatment for cancers that form solid tumors, researchers are looking at the possibility of using it to treat people with one such cancer, called neuroblastoma, which begins in the nerve cells, according to the American Cancer Society. [5 Amazing Technologies That Are Revolutionizing Biotech]
What made Layla's case special was the use of the designer T-cells, said Dr. Madan Jagasia, of the Outpatient Stem Cell Transplant Program at Vanderbilt-Ingram Cancer Center, who was not involved in treating Layla.
The genetic technique that researchers used to treat Layla is nicknamed TALEN (which stands for transcription activator-like effector nuclease). It involves an enzyme that works like tiny scissors, and cuts genes. In Layla's case, the researchers cut specific sequences out of the DNA of the donated cells, and corrected broken genome sequences.
This gene editing reprogrammed the cells to fight leukemia cells.
It is still unclear how long the treatment will keep Layla's leukemia at bay. "We don't know if this translates into a cure," but the treatment put her in remission long enough for doctors to perform a bone marrow transplant, which can hold cancer at bay for a long time, Jagasia said.
For Layla, the treatment itself took only about 10 minutes — the genetically edited cells were given through an IV line. But after the cells were delivered, she spent several months in isolation to stay protected from infection while her immune system was weak, according to a statement from the hospital.
Once the genetically edited cells had finished their job of killing all of the leukemia cells, Layla received a bone marrow transplant to replace her entire immune system with healthy cells. Now recovering at home, Layla still returns for regular checkups so that doctors can look at her bone marrow cells and blood cell counts, according to the hospital.
The doctors who treated Layla, along with investigators at University College London and biotech company Cellectis, had been working to refine these T-cells. The cells are collected from donors, edited and frozen in doses that can be thawed and provided on demand for patients who need T-cells. Jagasia said that the main advantage of having these "off-the-shelf" (i.e., ready-to-use) treatments is that less time is needed to administer the treatment to the patient.
"It's really a game-changing technology, because you could have these T-cells sitting in a lab everywhere, and you wouldn't have to collect a patient's T-cells," Jagasia told Live Science. "It really changes the cancer therapy field completely."
Though more research is needed, clinical trials funded by Cellectis are currently being planned to test the genetically edited T-cells in larger groups of adults and children who have blood cell cancers. The trials are set to begin in early 2016.