New treatment offers hope to 30,000 leukemia patients

In what is expected to be a major breakthrough in treatment of acute leukemia, a US Food and Drug Administration advisory panel has unanimously endorsed a leukemia treatment that could be the first gene therapy available in the world.

The technology has been developed by taking out patient's own cancer cells and genetically altering them to fight cancer. Doctors say once passed through clinical trials the treatment can benefit over 30,000 acute leukemia patients in India that happen every year.

"It is certainly a major development and can be a breakthrough in treatment. This is a technology where tumor cells are taken out from patient's body and re-engineered to be injected back to fight cancer. Researches to develop this was going on for last three-four years. The development is remarkable," says Dr Vinod Raina, executive director, medical oncology and hematology at Fortis Memorial Research Institute.

However, there are also challenges attached to the discovery. For instance, the technique, a separate treatment for each patient, has to be customized every time it is used. This means once cells are removed at an approved medical center, they have to be frozen, shipped to a pharmaceutical plant for thawing and processing, frozen again and shipped back to the treatment center.

"It is not as easy as it sounds because this treatment is developed through modifying genes. But it is certainly a game changer and will benefit mainly those who do not respond to normal management," says Dr Ratna Puri, Senior consultant, Department of Genetics, Ganga Ram Hospital.

A single dose of the resulting product has brought long remissions, and possibly cures, to scores of patients in studies who were facing death because every other treatment had failed. The FDA panel recommended approving the treatment for B-cell acute lymphoblastic leukemia that has resisted treatment, or relapsed, in children and young adults aged 3 to 25.

According to Dr Puri, there can also be side-effects from the treatment but that has to be assessed

Many researchers and drug companies have been engaged in intense competition for decades to reach the latest milestone. Swiss drug maker Novartis is now seeking approval to use the treatment for patients aged 3 to 25 with a blood cancer called acute lymphoblastic leukemia whose disease has spread or failed to respond to standard treatment.


According to reports, the main evidence that Novartis presented to the FDA came from a study of 63 patients who received the treatment from April 2015 to August 2016. Fifty-two of them, or 82.5%, went into remission — a high rate for such a severe disease. Eleven others died.


"We need long term major clinical trials to assess how long the benefits can be sustained through such therapy," says Dr Raina.


India witnesses 10 lakh new cancer cases every year. Of this, around 30000 are cases of acute leukemia, which is more common among children and young adults. The present line of treatment include drug based therapy and bone marrow transplants.

Source:http://timesofindia.indiatimes.com/home/science/new-treatment-offers-hope-to-30000-leukemia-patients/articleshow/59597979.cms

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