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    Personalized and costly, but new ALL treatment offers hope

    A new customized therapy for acute lymphoblastic leukemia (ALL) offers high remission rates but at a substantial cost, and only at specialized cancer centers.

    A breakthrough treatment for acute lymphoblastic leukemia (ALL), one of the most common childhood cancers, offers new hope for patients who have not responded to standard therapies.

    The new treatment, developed by Novartis AG (Basel, Switzerland), involves removing cells from affected patients, programming them to attack cancer cells, then returning those cells to the patients. Access to the highly personalized treatment is somewhat limited at this point and very expensive, but oncology experts are still touting the therapy as revolutionary in the field.

    Gwen Nichols, MD, chief medical officer of the Leukemia and Lymphoma Society, says CAR-T cell therapy is a “remarkable breakthrough” for treating children and adults aged younger than 25 years who have relapsed or not responded to standard therapy for B-cell ALL. Nichols notes that the treatment has not been approved by the US Food and Drug Administration (FDA) as a first-line treatment, and can only be used after traditional treatment methods have failed.

    “While the CAR-T cells do not last for all patients who receive this treatment, those patients who have experienced a lasting remission have been able to go on to enjoy their normal lives, returning to school and regular activities within a few months after the single in-hospital treatment,” Nichols says, adding that physicians should remember that the treatment is not widely available at this time. “Doctors should be aware that for now the treatment is only available at major cancer treatment facilities, so patients may need to travel from their homes in order to have access to this treatment.”

    Scourge of ALL

    Acute lymphoblastic leukemia makes up about a quarter of all cancer diagnoses in children aged younger than 15 years and is the most common childhood cancer in the United States. It appears most often between the ages of 3 and 5 years, and affects slightly more boys than girls, according to St. Jude Children’s Research Hospital, Memphis, Tennessee.

    More: Why do so many kids die of cancer so soon after diagnosis?

    Standard treatment options have traditionally been limited and involve a combination of chemotherapy and radiation lasting roughly 2 to 3 years. This regimen works well for about 90% of ALL patients, Nichols says, with younger patients responding better than adolescents and young adults. In those cases where treatment works, the patients are considered cured, although they still may suffer from some long-term or late-term effects from their treatments. Patients who relapse after standard treatment may receive stem cell transplants, but it’s an intensive procedure that requires long hospitalizations with limited exposure to other people.

    “Children undergoing treatment for ALL miss many days of school,” Nichols says.

    The benefit of CAR-T cell therapy, she says, is that the treatment can generally be done with a one-time infusion following some chemotherapy to prepare for the procedure. This is not to say that CAR-T cell therapy is easy, however. Nichols says it can sometimes be followed by an intense reaction called cytokine release syndrome. This involves a high fever, tachycardia, and other serious adverse effects. Nichols says some of these adverse effects can be mitigated and often pass within a few days, but the reaction also can be life threatening. Still, for patients who have exhausted other therapy options, the new treatment offers a lot of hope.

    “This new approach to therapy provides a very promising option for patients who before this had a very poor prognosis,” Nichols says. “Whereas the vast majority of patients respond well to standard therapy, those who do not had very limited options and typically had very poor outcomes. This new therapy is likely to save many hundreds of lives.”

    NEXT: What's the pricetag?

    Rachael Zimlich, RN
    Rachael Zimlich is a freelance writer in Cleveland, Ohio. She writes regularly for Contemporary Pediatrics, Managed Healthcare ...

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