Gene Therapy Shows Promising Treatment for Blood Cancers


Blood cancers, such as Leukemia, may have found a new enemy in gene therapy. The results are nothing short of remarkable for the patients taking part in the experimental studies.

According to The Mayo Clinic, gene therapy is the process of replacing or altering faulty genes or adding new genes for the purpose of increasing the ability to to fight disease, and even cure some diseases.  This remarkable medical science has already shown potential applications in the treatment of life threatening conditions such as cystic fibrosis, hemophilia, and AIDS.  In the U.S. it is only available through clinical trials, and limited public use in Europe.  Scientists are still researching all the possible applications, as well as the restrictions and parameters, of this futuristic procedure.

The gene therapy being used works by filtering millions of white blood cells, removing them from the body and altering them in the lab. They are returned to the body to become an army locating and attacking the cancer cells. Dr. David Porter, a University of Pennsylvania scientist who held one of the studies, put it this way, “What we are giving essentially is a living drug”. NBC heralds this as one of the biggest advances against blood cancer in recent years.

Patients with a specific type of Leukemia were given the one-time treatment six years ago and some have remained cancer-free since.  One of the most impressive aspects of this and the other half a dozen research studies to date is that many of the participants were very advanced in their disease. So far 120 patients have received the treatment, many have had remarkable successful results.

Many biotech and pharmaceutical companies are developing gene therapy. University of Pennsylvania has patented their method and licensed it to Novartis AG. Bloomberg reports that the Switzerland-based company is building a $20 million research center on the Penn campus in Philadelphia and clinical trials are scheduled for next year. If successful these trials could lead to federal approval as early as 2016.

“There is a sense of making history… a sense of doing something very unique,”

said Hervé Hoppenot, president of Novartis Oncology.

According to their website, The Leukemia and Lymphoma Society donated $68 million to research last year and is hopeful that these new options will help to prolong the lives of those affected. By the end of 2013 nearly 150,000 new cases of leukemia, lymphoma, and myeloma are expected is the US.

Many find success with currently approved treatments. Chemotherapy is the most common. However, bone-marrow and stem-cell transplants are both successful, although risky, when patients are lucky enough to find a matching donor. Gene therapy trials are thus far seen as a last option for cancer patients who are in danger of dying when these other treatment options have failed.

It is an exciting and worthwhile effort to explore gene therapy for treating blood cancer and other serious diseases. The world is holding it’s breath in anticipation of an effective solution that is both safe and long lasting.