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    HICAGO  Doctors are reporting unprecedented success from a new    cell and gene therapy for multiple myeloma, a blood cancer    thats on the rise. Although its early and the study is small     35 people  every patient responded and all but two were in    some level of remission within two months.  
    In a second study of nearly two dozen patients, everyone above    a certain dose responded.  
    Experts at an American Society of Clinical Oncology conference    in Chicago, where the results were announced Monday, say its a    first for multiple myeloma and rare for any cancer treatment to    have such success.  
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    Chemotherapy helps 10 to 30 percent of patients; immune system    drugs, 35 to 40 percent at best, and some gene-targeting drugs,    70 to 80 percent, but you dont get to 100, said Dr. Len    Lichtenfeld, deputy chief medical officer of the American    Cancer Society.  
    These are impressive results, but time will tell if they    last, he said.  
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    Multiple myeloma affects plasma cells, which make antibodies to    fight infection. More than 30,000 cases occur each year in the    United States, and more than 115,000 worldwide. Its the second    fastest growing cancer for men and the third for women, rising    2 to 3 percent per year, according to the National Cancer    Institute. About 60,000 to 70,000 Americans have it now.  
    Nine new drugs have been approved for it since 2000 but theyre    not cures; only about half of U.S. patients live five years    after diagnosis.  
    With cell therapy, I cant say we may get a cure, but at least    we bring hope of that possibility, said Dr. Frank Fan. He is    chief scientific officer of Nanjing Legend Biotech, a Chinese    company that tested the treatment with doctors at Xian    Jiaotong University.  
    The treatment, called CAR-T therapy, involves filtering a    patients blood to remove immune system soldiers called T    cells. These are altered in a lab to contain a gene that    targets cancer and then given back to the patient    intravenously.  
    Doctors call it a living drug a one-time treatment to    permanently alter cells that multiply in the body into an army    to fight cancer. Its shown promise against some leukemias and    lymphomas, but this is a new type being tried for multiple    myeloma, in patients whose cancer worsened despite many other    treatments.  
    In the Chinese study, 19 of 35 patients are long enough past    treatment to judge whether they are in complete remission, and    14 are. The other five had at least a partial remission, with    their cancer greatly diminished. Some are more than a year past    treatment with no sign of disease.  
    Most patients had a group of side effects common with this    treatment, including fever, low blood pressure, and trouble    breathing. Only two cases were severe and all were treatable    and temporary, doctors said.  
    The second study was done in the U.S. by Bluebird Bio and    Celgene, using a cell treatment developed by the National    Cancer Institute. It tested four different dose levels of cells    in a total of 21 patients. Eighteen are long enough from    treatment to judge effectiveness, and all 15 who got an    adequate amount of cells had a response. Four have reached full    remission so far, and some are more than a year past treatment.  
    The results are very remarkable, not just for how many    responded but how well, said Dr. Kenneth Anderson of    Dana-Farber Cancer Institute in Boston.  
    We need to be looking for how long these cells persist and    keep the cancer under control, he said.  
    Dr. Carl June, a University of Pennsylvania researcher who    received the conferences top science award for his early work    on CAR-T therapy, said its very rare to see everyone respond    to a treatment. His lab also had this happen  all 22 children    testing a new version of CAR-T for leukemia responded, his    colleagues reported at the conference.  
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    The first patients we treated in 2010 havent relapsed, June    said.  
    Dr. Michael Sabel of the University of Michigan called the    treatment revolutionary.  
    This is really the epitome of personalized medicine,    extending immune therapy to more types of patients, he said.  
    Legend Biotech plans to continue the study in up to 100 people    in China and plans a study in the U.S. early next year. The    treatment is expected to cost $200,000 to $300,000, and whos    going to pay for that is a big issue, Fan said.  
    The manufacturing process is very expensive and you cant    scale up. Its individualized. You cannot make a batch as is    done with a drug, he said.  
    Nick Leschly, Bluebirds chief executive, said the next phase    of his companys study will test what seems the ideal dose in    20 more people.  
    Marilynn Marchione  
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A first: All respond to CAR-T therapy in a blood cancer study - STAT