Gene therapy has just begun to set the health-world abuzz with some of its most recent successes. Patients with leukemia and other blood cancers are seeing unprecedented results with these new treatments, sending some into full remission by attacking and killing the highly fatal diseases.
A few years ago, only a handful of leukemia patients with one type of blood cancer were involved with experimental gene therapy, by being given just one single treatment. Today many of those early test-group patients are still living cancer-free. Now more than 120 patients, in 6 test-groups, with a variety of bone marrow or blood cancers are being treated with some amazing results.
Dr. Janice Abkowitz, is the president of the American Society of Hematology and blood diseases chief at Seattle’s University of Washington. Dr. Abkowitz is really excited with the new technology and the results that takes a patient’s cells and engineers them to attack the cancer cells present in the patient’s body.
In one such study, 22 children and 5 adults with acute lymphocytic leukemia had incredible results that sent most into a full remission. Although just a few have relapsed now, early results showed no cancer was found after the treatment. The patients used in the study were subjects that had up to 10 types of chemotherapy, multiple bone marrow transplants and other treatments. Before the gene therapy started on this test-group, the patients were considered gravely ill. Patients used in the trial gene therapy study are usually patients that are in grave dangers of dying because all other conventional methods of treatments have already failed.
The first child patient into the study was 8-year-old Emily Whitehead from Philipsburg, Pennsylvania. Before taking the gene therapy, doctors gave Emily just a few days before her major bodily organs would begin to fail. Today and nearly two years later, Emily shows no signs of cancer.
Currently only one European gene therapy is approved for a rare metabolic disease. However, doctors are highly optimistic that this gene therapy could be the first one approved for cancer on a world-wide scale, and the first gene therapy approved in the United States.
The process in this gene therapy involves the filtering of white blood cells called, T-cells, out of the patient’s blood. Those cells are then altered in a lab with a gene that will target and kill the cancer cells. The cells are then returned to the patients in an infusion over a period of three days. When the treatments are successful, the engineered cells then attack and kill the patient’s cancer cells.
A University of Pennsylvania scientist, Dr. David Porter, led one of these studies. Dr. Porter said that essentially the patient is receiving a living drug of permanently altered cells. Those cells then multiply in the patient’s body forming an army of cells that fight the cancer.
Penn campus in Philadelphia is currently building a research center into gene therapy and planning a clinical trial to begin next year. Several biotech and drug companies have hopped on the bandwagon and started to patent methods of gene therapy that could possibly see approvals of treatments as early as 2016.
Switzerland’s Novartis AG is just one such company that will join in producing gene therapies. President of Novartis Oncology, Hervé Hoppenot, says that they have a sense of doing good things and making history. The Leukemia and Lymphoma Society’s chief scientific officer agrees with Hoppenot, and said that he’s seen some powerful responses to gene therapy that look like some major advancements in the treatment of cancer.
The big advantage to gene therapy over conventional cancer treatments is that there is no donors needed for bone marrow or stem cells and chemotherapy often causes serious side effects. Transplants are often risky for the patients and donors often cannot be found.
The price for treating a single patient with gene therapy is around $25,000, which is much lower than conventional drugs and treatments, and much, much lower than a transplant.
Some of the side effects of gene therapy are temporary and reversible and often mimic a severe flu that can last a few days.
Part of the test group responded with a complete remission after only one gene therapy session, while others went into partial remission and have doctors thinking of a second treatment. A few of the patients did not respond to the sessions, yet some of the patients went in to full remission in just one single month.
The true success of the gene therapy will take years before studies and methods are fully tested and approved. However, if these early trials are already seeing patients in full remission, it means doctors and scientist are on the right path to stalling and killing this horrible disease.
By Brent Matsalla