Groundbreaking FDA Approval for This Cancer Gene Therapy

Little Emily Whitehead is looking attentively at a butterfly that has landed on her finger. She coos at it and lets it rest on the tip of her thumb peacefully. In 2012, six-year-old Emily might not have had the chance to enjoy this refreshing and carefree moment. But now, she is living cancer-free, according to The Washington Post.

Emily, like too many other children and adults, had lethal leukemia.
But unlike most children in her position, Emily became the first child to receive a new trial of gene therapy that offers considerable hope and promise to cancer patients. While once Emily was not responding to more traditional forms of therapy and was near death, she now lives without the deadly cancer.

The University of Pennsylvania hospital has made its own footprint with clinical trials for adults and children. It has had several successful trials with gene therapy, first with adults, and now with children. In 2010, they did an experimental treatment never-before-human-tested on a patient named Bill Ludwig who was overjoyed when his own bone marrow biopsy came back as cancer free.

 Cart t-cell treatment is now the first approved gene therapy by the Food and Drug Administration.

This level of immunotherapy could save thousands of lives. The drug is is manufactured by Novartis and goes by the name Kymriah. The therapy works by genetically altering a patient’s immune cells to fight blood cancers.

Given it’s approval, CAR T-cell treatment will be available for a limited number of children and adults who have been unresponsive to standard care. Usually, advancements like this are not immediately available to children because therapies are often developed with adults in mind first. But this particular gene therapy will be available for those patients who are 25 and younger.

Childhood leukemia is just one of the areas that gene therapy has promised to help save lives in. Kite Pharma of Santa Monica is also hopefully awaiting news for FDA approval for treatments for several aggressive types of non-Hodgkin lymphoma.

Examples of non-Hodgkin lymphoma include Mantle cell lymphoma, which is a rare b-cell non Hodgkin lymphoma that mostly affects men over the age of 60. Another non-Hodgkin lymphoma that could be helped by new treatments is Marginal lymphoma which is slow growing and mostly affects those over the age of 65.

While FDA approval on new treatments like this is super exciting, one might caution against the expensive costs of these treatments as well as the potential life threatening side effects.

The cost of treatment is expected to be as much as $500,000 for just one infusion.

Though the Novartis is brainstorming ways to make it more affordable for patients. Also, since the treatment operates by changing patient’s immune cells, the supercharged immune system can cause side effects such as a raised fever and other symptoms mirroring the flu, that can leave the patient in intensive care.

Read more about this new therapy in NPR here.

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