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These are exciting times in the world of medicine. With every passing day, there is some new innovation which infuses hope in millions of patients. Until now, leukemia was a dreaded disease. But, thanks to gene therapy, even it appears to be treatable.

Recent news about gene therapy curing leukemia in just eight days has brought a smile on the lips of millions of patients suffering from leukemia worldwide. Leukemia accounts for almost 33% of all cancers afflicting children and is responsible for the deaths of 1,340 American children every year.

Now, researchers from Memorial Sloan Kettering Cancer Center in New York have reported in a study published in the journal Science Traditional Medicine that they have

successfully used gene therapy to treat leukemia in just eight days. They were able to do so by making certain genetic modifications in the immune cells of the patients.

All the patients in the study were suffering from acute lymphoblastic leukemia (ALL), an incurable form of blood cancer.

While one of the patients saw the disappearance of cancer cells from his blood within eight days of starting the therapy, four of them were treated within eight weeks. One of the patients died after a relapse, another one died from a blood clot which was unrelated to the therapy. The patients, in whom the gene therapy was successful, have been in remission for between 5 months and 2 years.

What exactly did the researchers do to achieve such marvelous results?

Our immune system contains two types of lymphoblasts, namely the T cells and the B cells. In ALL, the B cells become malignant and start regenerating spontaneously. The researchers taught of ways to control the unchecked growth of these cells. They were able to identify a molecule, called as CD19, which is present only on the surface of B cells. The researchers, extracted T cells from the patients suffering from ALL and genetically modified them so that they would attack all the cells bearing the CD 19 molecule. These modified T cells were re-introduced into the blood stream of the patients, wherein they killed all the cells bearing CD19 molecule. While all the cancerous B cells were killed in this manner, some of the non-cancerous B cells were also killed in the process. In all the five patients who survived, there was no trace of cancerous cells left after this form of gene therapy.

The researchers feel that gene therapy is more effective in patients who use it as the first line of treatment. The two patients who died in this study, the one who suffered a relapse and the one who died due to a blood clot had probably used another form of conventional therapy for treating leukemia before trying the gene therapy.

Continue reading after recommendations

  • “Chimeric Antigen Receptor-Modified T Cells for Acute Lymphoid Leukemia,” by Stephan A. Grupp, et al, published Online First, March 25, 2013 in New England Journal of Medicine, accessed on April 6, 2013
  • “Targeting chronic lymphocytic leukemia cells with a humanized monoclonal antibody specific for CD44,”by Suping Zhang et al, published on March 25, 2013 in PNAS, accessed on April 6, 2013
  • “CD19-Targeted T Cells Rapidly Induce Molecular Remissions in Adults with Chemotherapy-Refractory Acute Lymphoblastic Leukemia,” by Michel Sadelain, et al, published on March 20, 2013 in Science Translation Medicine, accessed on April 6, 2013
  • “Combinatorial antigen recognition with balanced signaling promotes selective tumor eradication by engineered T cells,” by Christopher C Kloss, et al, published in December 16, 2012 issue of Nature Biotechnology, accessed on April 6, 2013.
  • Photo by steadyhealth.com

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