Cancer cure for babies: Engineered T-cells to the rescue

Two babies cured of leukemia with genetically engineered immune cells taken from another person, in technology breakthrough.

MEDICINE & SCIENCE

Immunotherapy: Leukemia cure for babies from edited T-Cells

Two babies have been cured of the leukemia after treatment with genetically engineered immune cells taken from another person.

This is the world's first attempt to use genetically engineered cells to treat cancer.

The treatment began more than a year ago with a cell therapy developed by the French biotechnology (biotech) firm Cellectis.

The details of the case have been published in the medical journal Science Translational Medicine (see here).

Although the two baby girls in the trial of the therapy remained disease-free for as long as 18 months after treatment, long-term monitoring of the effectiveness of the treatment will still be required.

The therapy is now officially known as UCART19 and initial phase I clinical trials are now underway.

The two babies were also given chemotherapy to cure their cancer, so some researchers say we cannot be sure that it was the genetically engineered immune cells that cured the babies. 
 
One of the two babies in the trial developed so-called graft versus host disease (GVHD), an immune reaction against the donated cells in the body of the patient, but steroids and a bone marrow transplant solved the problem.

IMMUNOTHERAPY USING T-CELLS

The new medical technology of immunotherapy is defined as the "treatment of disease by inducing, enhancing, or suppressing an immune response" (see here)

Immunotherapies using engineered T-cells, known as CAR T-cell therapy (see here) have been successfully used to fight leukemia (bone marrow cancer) and blood cancers and are one of the hottest new areas in medical research.

Trials by pharmaceutical companies Novartis and Juno have about 50% success rate in curing cancer.

In CAR T-cell therapy a patient's "T cells are changed in the laboratory so  they will attack cancer cells."

"T cells are taken from a patient's blood [or from a donor's blood]. Then the gene for a special receptor that binds to a certain protein on the patient’s cancer cells is added in the laboratory. The special receptor is called a chimeric antigen receptor (CAR)."

"Large numbers of the CAR T cells are grown in the laboratory and given to the patient by infusion" (source here).

ULTIMATE GOAL: CHEAP OFF-THE-SHELF CANCER THERAPY

The ultimate goal of the research is an off-the-shelf universal therapy, an inexpensive supply of universal immune system cells that could be fed into a patient's bloodstream when needed to fight cancer. 

It is also worth noting that some naturally occuring T cells destroy cancer also, so-called cytotoxic T  lymphocytes (CTL) or killer T cells (see here).

The new therapy in this article, known as UCART19, is different from previous CAR T-cell therapy in that it uses T-cells from healthy donors instead of T-cells from the patient.

This has the potential to make the therapy cheaper and also more suitable for use with young children who commonly suffer from Leukemia and do not have enough T-cells to begin with (see video below).

http://www.reuters.com/article/us-health-cancer-gene-editing-idUSKBN1592IX
https://www.technologyreview.com/s/603502/two-infants-treated-with-universal-immune-cells-have-their-cancer-vanish/
https://www.cancer.gov/publications/dictionaries/cancer-terms?cdrid=771302

https://en.wikipedia.org/wiki/Immunotherapy
https://en.wikipedia.org/wiki/Cytotoxic_T_cell

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