Blood cancers get new enemy
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. Now at least six research groups have treated more than 120 patients with many types of blood and bone marrow cancers, drawing stunning results.
“It's really exciting,” said Dr. Janis Abkowitz, blood diseases chief at the University of Washington in Seattle and president of the American Society of Hematology. “You can take a cell that belongs to a patient and engineer it to be an attack cell.”
In one study, all five adults and 19 of 22 children with acute lymphocytic leukemia, or ALL, 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 Emily Whitehead of Philipsburg, Pa., that doctors said her major organs would fail within days. She was the first child given the gene therapy and shows no sign of cancer nearly two years later.
Results on other patients with myeloma, lymphoma and chronic lymphocytic leukemia, or CLL, will be reported at the hematology group's conference, which started Saturday in New Orleans.
Doctors say this has the potential to become the first gene therapy approved in the United States and the first for cancer worldwide. Only one gene therapy is approved in Europe, for a rare metabolic disease.
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. David Porter, a University of Pennsylvania scientist who led one study.
Several drug and biotech companies are developing these therapies. Penn has patented its method and licensed it to Switzerland-based Novartis AG. The company is building a research center on the Penn campus in Philadelphia and plans a clinical trial next year that could lead to federal approval of the treatment as soon as 2016.
Talking with the researchers, “there is a sense of making history ... a sense of doing something very unique,” said Hervé Hoppenot, president of Novartis Oncology, the division leading the work.
Lee Greenberger, chief scientific officer of the Leukemia and Lymphoma Society, agreed.
“From our vantage point, this looks like a major advance,” he said. “We are seeing powerful responses ... and time will tell how enduring these remissions turn out to be.”
The group has given $15 million to various researchers testing this approach.
Many patients are successfully treated with chemotherapy or bone marrow or stem cell transplants, but transplants are risky, and donors can't always be found. So far, gene therapy has been tried on people who were in danger of dying because other treatments failed.
The gene therapy must be made individually for each patient, and lab costs are about $25,000 without a profit margin. That is still less than many drugs to treat these diseases and far less than a transplant.
Show commenting policy
TribLive commenting policy
You are solely responsible for your comments and by using TribLive.com you agree to our Terms of Service.
We moderate comments. Our goal is to provide substantive commentary for a general readership. By screening submissions, we provide a space where readers can share intelligent and informed commentary that enhances the quality of our news and information.
While most comments will be posted if they are on-topic and not abusive, moderating decisions are subjective. We will make them as carefully and consistently as we can. Because of the volume of reader comments, we cannot review individual moderation decisions with readers.
We value thoughtful comments representing a range of views that make their point quickly and politely. We make an effort to protect discussions from repeated comments either by the same reader or different readers.
We follow the same standards for taste as the daily newspaper. A few things we won't tolerate: personal attacks, obscenity, vulgarity, profanity (including expletives and letters followed by dashes), commercial promotion, impersonations, incoherence, proselytizing and SHOUTING. Don't include URLs to Web sites.
We do not edit comments. They are either approved or deleted. We reserve the right to edit a comment that is quoted or excerpted in an article. In this case, we may fix spelling and punctuation.
We welcome strong opinions and criticism of our work, but we don't want comments to become bogged down with discussions of our policies and we will moderate accordingly.
We appreciate it when readers and people quoted in articles or blog posts point out errors of fact or emphasis and will investigate all assertions. But these suggestions should be sent via e-mail. To avoid distracting other readers, we won't publish comments that suggest a correction. Instead, corrections will be made in a blog post or in an article.
- Sandusky won’t get his pension back
- Liquor Control Board, Pennsylvania universities target problem drinking
- Poor sales sink multi-state Monopoly Millionaires’ Club lottery
- Philadelphia mother pleads guilty as boy, 2, shoots, kills sister
- Erie man charged with 1990 slaying of Virginia Beach woman
- Pennsylvania Marine who killed family takes own life