PhotoRecent cancer research has focused on using the body's own T-cells to fight the disease, rather than introducing outside elements, like drugs, to attack it.

For the last few years it's been theoretically possible. Now, researchers say they are a step closer to actually doing it. The report of their research is published (PDF) in the Proceedings of the National Academy of Sciences.

Alexander Marson, of the University of California at San Francisco, led a team of researchers that “edited” human genes, replacing components of T-cells with stronger elements that can protect the body against many chronic diseases, including diabetes and cancer.

The scientists say their results may pave the way for a completely new way of fighting – and preventing – these diseases. Instead of introducing drugs into the patient, doctors would extract and edit T-cells, putting them back into the patient's body.

Genome engineering toolkit

“We aimed to overcome long-standing challenges in genetic manipulation of primary T-cells and establish an efficient genome engineering toolkit,” the authors wrote.

T-cells, or T lymphocytes, are a type of white blood cell that plays a key role in the immune system. They are different from other lymphocytes, such as B-cells, because of the presence of a T -cell receptor on the surface of the cell.

In a disease like HIV/AIDS, the patient loses T cells and, therefore, has a weakened immune system. Researchers have been working for a number of years to find ways of genetically engineering T-cells in a patient's blood.

Strong deterrent

The theory was simple: the presence of stronger T-cells would provide a strong deterrent against invasive pathogens that cause potentially lethal disease, along with auto-immune conditions like type 1 diabetes.

As we reported in 2013, private researchers in Cambridge, England made huge strides in the area of T-cell manipulation. Researchers working for Immunocore designed a therapy using the body's T-cells to find cancer cells and destroy them.

Months earlier doctors at the University of Pennsylvania used T-cell therapy to successfully treat leukemia patients. The clinical trial participants, all of whom had advanced cancers, included ten adult patients with chronic lymphocytic leukemia and two children with acute lymphoblastic leukemia.

At the time of the reporting, three of the first ten  patients treated with the protocol remained healthy and in full remission more than 2 years after their treatment.

Marson and his team have taken the process a step further, using Clustered, Regularly Interspaced, Short Palindromic Repeat (CRISPR) gene-editing to cut and replace pieces of DNA within the chromosomes of T-cells in a test tube. Their results convince them that the process can be used effectively to treat patients in the near future.

For example, Marson says the process could lead to developing T-cells that are immune to HIV and injecting them back into the body. While he says there is still a lot of work ahead, gene-editing could become a powerful weapon against cancer and other diseases.


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