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HIV used in treatment for leukaemia

11th December 2012

Cancer experts at a children's hospital in Philadelphia have successfully used an experimental treatment on a seven-year-old girl with leukaemia.

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The treatment uses a disabled version of the HIV virus to reprogramme the body's immune system to attack cancer cells.

It was tried as a last resort by the desperate parents of Emma Whitehead, who had suffered two relapses are two bouts of chemotherapy for her acute lymphoblastic leukaemia, and whose family was running out of treatment options.

Millions of Emma's T-cells - a type of white blood cell - were removed. New genes were then slotted into them, with the help of the modified HIV virus, programming them to attack B-cells, which are part of a normal immune system but which turn malignant in leukaemia.

The modified T-cells were then reinjected into her bloodstream, where they started multiplying and attacking the cancer.

While the treatment itself nearly killed Emma, because it released a "cytokine storm" of fevers and chills and dangerously low blood pressure, an arthritis medication mitigated the effects on her body.

Emma survived, and emerged from treatment cancer-free. Seven months on, she still has no signs of the disease, and the treatment could well protect her from cancer for some years to come.

Emma, who is her parents' only child, was diagnosed with acute lymphoblastic leukemia at the age of five.

The experimental treatment she received is now being offered on a trial basis at other cancer hospitals, including the National Cancer Institute and the Memorial Sloan-Kettering Cancer Center in New York.

According to Carl June, who heads the University of Pennsylvania research team who pioneered the treatment, the new treatment could eventually replace bone-marrow transplantation, which is currently the last line of treatment for leukaemia and similar diseases.

So far, only around 12 people have received the treatment in Pennsylvania, three of whom had complete remissions, and two of whom had no sign of disease two years later.

So far, only around 12 people have received the treatment in Pennsylvania, three of whom had complete remissions, and two of whom had no sign of disease two years later.

A further four people saw an improvement in their condition, but did not go into full remission, while a child had improved and then suffered a relapse.

Two adults did not respond well to treatment, while another only recently completed treatment, and could not be evaluated.

Overall, despite the mixed picture, researchers are highly optimistic about the potential of the new approach, which was presented at a recent meeting of the American Society of Haematology in Atlanta, Georgia.

According to Johns Hopkins cancer expert Ivan Borrello, the treatment is "a major breakthrough," while bone marrow transplant specialist John Wagner at the University of Minnesota said the results were something that the oncology community had long hoped for.

The research has attracted an investment of US$20 million from Swiss-based drug company Novartis, which is building a research center at the university to bring the treatment to market.

The company thinks the technique has the potential to revolutionise leukaemia treatment and other blood cancers, according to Hervé Hoppenot, president of Novartis Oncology.

However, researchers say the technique is still in the early stages of development, and it is still unclear exactly how it works, and why it can also fail.

 

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