One of the toughest parts of working in the biotechnology industry is the likelihood that you will eventually pour your heart and soul into a project, work long hours, do everything right, advance promising science, raise loads of capital, even see patients helped in clinical trials … yet, ultimately, fail to commercialize a new medicine because of a safety or efficacy issue.
Ninety percent of programs in the clinic do not make it to the pharmacy shelf. The approval of biomedical breakthroughs is a painstakingly slow process of trial and error – of learning from the illuminating failures of your peers.
Because so much disappointment underlies every major scientific discovery, when we do make huge systemic gains over time in saving and extending the lives of children with pediatric cancers, we celebrate the difference made by our collective determination and perseverance.
In the last half-century, biopharmaceutical innovation has dramatically reduced the mortality rate for children and adolescents diagnosed with cancer:
- In 1975, young people under 20 diagnosed with cancer had a 50/50 chance of being alive five years later, according to the National Cancer Institute. The five-year cancer survival rate for children and teens has since grown to 83 percent.
- For the most common childhood cancer, acute lymphoblastic leukemia (ALL), the five-year survival rate for ALL was just 10 percent in the 1960s; treatment breakthroughs have since brought that number up to 88 percent.
- Enormous progress also has been made in treating children and teenagers with non-Hodgkin lymphoma. A five-year survival rate of less than 50 percent in 1970s has since improved to 89 percent.
It’s not that fewer children and adolescents are contracting cancer. Incidences are actually up .6 percent since 1975. However, advances in oncology treatments and greater youth participation in clinical trials have dramatically improved outcomes.
Treatments for childhood cancer often involve surgery, radiation and/or chemotherapy, depending on the stage and type of cancer. Newer treatments in biotechnology, involving targeted therapy drugs and immunotherapy, have become game-changers in saving lives.
The landmark approval of Novartis’ new chimeric antigen receptor T-cell (CAR-T) treatment for ALL on Aug. 30 was the first ever gene therapy approved by the U.S. Food and Drug Administration, and the breakthrough will give new hope to children with ALL who have been unresponsive to other forms of treatment. The therapeutic approach could hold great promise for treating other types of cancer as well.
Other potential oncology breakthroughs are in earlier stages of development. For example, a therapeutic vaccine targeting brain cancer in children is being studied at the University of Florida’s brain tumor immunotherapy program. The work involves extracting genetic material from a patient’s brain tumor and using it to train T-cells to recognize and attack the tumor. This vaccine could represent an important development in the treatment of inoperable brain tumors. Stanford Medical School is in preclinical testing of an antibody that, if approved, could help the immune system target five different types of pediatric brain cancers while leaving healthy cells alone.
As we near the end of Childhood Cancer Awareness Month, we honor the scientists and oncology researchers whose laborious efforts – and, yes, frustrating failures – have led to monumental advances in scientific understanding, saving hundreds of thousands of young lives.