Charo Explains Breakthrough Genetic Treatment for Leukemia

R. Alta Charo
Thursday, July 27, 2017

For the first time, an FDA panel has recommended that the agency approve a genetic treatment for leukemia. The therapy has been shown to treat a type of leukemia that sometimes doesn't respond to standard therapies like chemotherapy. It uses genetic engineering – removing cells, editing them and then reintroducing them into the patient's body – to transform the living cells into a weapon against cancer. The clinical trial of 55 people had an 85% effectiveness rate, resulting in remission or possibly a long-term cure. In an interview on Wisconsin Public Radio, Consortium collaborator Prof. Alta Charo (University of Wisconsin Law School) notes the treatment is "not a magic bullet yet." She cites some very serious side effects, such as extreme inflammation that can cause extraordinarily high fevers. In addition, it is expensive, potentially costing as much as $500,000. If approved, the therapy is recommended for ages 3-25; however, it could eventually be rolled out to older patients. Charo describes this as an example of the "medicine of the 21st century [which will use] gene editing in order to personalize treatment . . . changing cells so they can become their own kind of biological drug." Charo is a member of the National Working Group for the LawSeqSM project, for which Consortium Chair Prof. Susan M. Wolf is one of the Principal Investigators. Listen to the entire interview here