New Treatment for Leukemia Saves Lives, One Step Closer to Market
December 14, 2012 7:19 AM
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Leukemia survivor Emma Whitehead
Drug company Novartis has provided $20 million for a research center
Pennsylvania researchers have found a way to use immune cells in the battle against cancer, and ended up saving a young
leukemia patient's life
A team from the University of Pennsylvania, which was led by Dr. Carl June, created a new cancer treatment that involves the altering of a patient's T-cells. Once modified, the T-cells are placed back into the patient to fight cancer cells.
The treatment works like this: millions of a patient's T-cells, which is a type of white blood cell, are removed and equipped with new genes and a disabled form of the HIV virus. The HIV virus was used because it does a great job of carrying genetic material into T-cells.
The modified T-cells, also called chimeric antigen receptor cells, are reprogrammed to attack a protein called CD-19, which can be found on the surface of most healthy or malignant B-cells. B-cells are a part of the immune system that become malignant in leukemia.
Once a patient
receives this treatment
, they become very ill. That's how you know the T-cells are working, because the chemicals that are released from the T-cells trigger cytokine-release syndrome (aka, severe fevers and chills).
These symtpoms nearly killed 7-year-old Emma Whitehead, who was diagnosed with leukemia when she was only five. After several attempts at chemotherapy, which resulted in relapses, Whitehead was admitted to the Children's Hospital of Philadelphia, where she received June's experimental treatment. It had never been performed on a human before.
The cytokine-release syndrome sent Whitehead's temperature up to 105, and she had to be placed on a ventilator. Just as it seemed there was no hope, June discovered that Whitehead's levels of the cytokine interleukin-6 were ridiculously high, leading to the worsening side effects of the treatment. June used a drug called tocilizumab, which is typically for rheumatoid arthritis, to lower the interleukin-6 levels.
On May 2, after being unconscious for a week, Whitehead woke up on her seventh birthday. She has been cancer-free ever since.
While the method was particularly successful for Whitehead, it has had mixed results with other patients since then. So far, about a dozen patients have had the treatment. Three adults with chronic leukemia are cancer-free, another four adults have improved but do not have full remissions, a child improved and relapsed, and another two adults had no results at all.
June and the research team believe the mixed results may be due to either a flawed batch of T-cells or that not all leukemic cells have the CD-19 protein that the T-cells are designed to attack.
Nevertheless, drug company Novartis has shown interest in the method and offered to pay $20 million for a research center on the University of Pennsylvania's campus to bring the treatment to market. What's more shocking about the investment is that the new T-cell treatment isn't a "one-size fits all" kind of deal; rather, it costs about $20,000 per patient to produce engineered T-cells specific to each individual.
But the treament has changed the life of Whitehead, who is now back in school with her peers and capable of doing the things a healthy child should be able to do.
The New York Times
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12/15/2012 10:33:57 AM
They're exploring similar therapies for lymphoma.
My mom is having a bone marrow transplant in March.
Hopefully she's having an autologous transplant? Not a picnic (I just went through one 6 months ago), but not nearly as tough as an allo transplant. Best wishes to you both.
As for that "$20,000 for creating the T cells", that's not that big an expense compared to existing therapies. One injection of Mozibil (just to mobilize stem cells for collection for transplant) was about $25,000.
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