Every year about 3,000 American families are given the devastating news that their child has acute lymphocytic leukemia (ALL), the most common childhood cancer. Thankfully, highly effective treatment exists. In a miracle of modern medicine, what was once a fatal disease has been transformed into one where 90 percent of children are cured.
Leaps and bounds
How did this happen? Until recently, it was not because of new drugs. Between the 1980s and early 2000s, not a single new chemotherapy was introduced into standard treatment. Instead, through collaboration and clinical trials, we learned how to use existing chemotherapies better, how to identify which children needed more intense therapy and how to deliver that therapy safely.
More work to do
Our fight is not over — 10 percent of children with ALL still die from their disease. We must redouble our efforts to cure these children. A new era of targeted therapy offers hope of doing just that. The FDA recently approved a new technique for fighting ALL in which a patient’s own immune cells are genetically transformed to recognize and seek out the patient’s leukemia cells; This is the first gene therapy ever to be approved. This is just one of a number of new “immunotherapies” that have shown extremely encouraging preliminary results. As we learn more about the genetic mutations that cause leukemia, new drugs that target specific mutations are being developed and tested.
At the same time, these exciting technologies cannot blind us to the basics. ALL treatment includes a several-year period in which oral chemotherapy is given daily at home. We know that children who receive less than 95 percent of their prescribed oral chemotherapy doses are three times as likely to relapse. Helping families navigate the practical barriers, financial or otherwise, that prevent them from consistently administering their child’s therapy may have just as large of an impact as targeted therapies.
Now is not the time for complacency. Curing every child with ALL is within our grasp, but requires accelerated efforts on the part of researchers, charities and government. With these efforts will come the day when no parent needs to go through the nightmare of losing a child to this disease.