In one of the biggest advances against leukemia and other blood cancers in many years, doctors are reporting unprecedented success by using gene therapy to transform patients' blood cells into soldiers that seek and destroy cancer.
A few patients with one type of leukemia were given this one- time, experimental therapy several years ago and some remain cancer- free today. Now, at least six research groups have treated more than 120 patients with many types of blood and bone marrow cancers, with stunning results.
"It's really exciting," said Dr.
In one study, all five adults and 19 of 22 children with acute lymphocytic leukemia had a complete remission, meaning no cancer could be found after treatment, although a few have relapsed since then.
These were gravely ill patients out of options. Some had tried multiple bone marrow transplants and up to 10 types of chemotherapy or other treatments.
Cancer was so advanced in 8-year-old
Results on other patients with myeloma, lymphoma and chronic lymphocytic leukemia were to be reported at the hematology group's conference that was to start Saturday in
Doctors say this has the potential to become the first gene therapy approved in
The treatment involves filtering patients' blood to remove millions of white blood cells called T-cells, altering them in the lab to contain a gene that targets cancer, and returning them to the patient in infusions over three days.
"What we are giving essentially is a living drug" - permanently altered cells that multiply in the body into an army to fight the cancer, said Dr.
Several drug and biotech companies are developing these therapies.
The gene therapy must be made individually for each patient, and lab costs now are about
The treatment can cause severe flulike symptoms and other side effects, but these have been reversible and temporary, doctors say.