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.
Thursday, July 27, 2017