By Stephen Minger, Ph.D./Industry Consultant, Scientific Advisor: Cell and gene therapy
The FDA approval of two cell therapies is a major milestone on an exciting journey of discovery. What are some of the important questions still being explored?
At the end of 2017, within months of each other, the first two Chimeric Antigen-Receptor T-cell (CAR T cell) therapy treatments received FDA approval. The approvals marked major milestones for cancer treatments and precision medicine worldwide, bringing the industry closer to realizing the promise of cell and gene therapies, and answered the key question of whether a therapy of this type can get approval. The industry is still at the beginning of this journey, however, while the industry is making incredible progress, there are still more questions than answers today. Here are some key questions that scientists, researchers, and clinicians are exploring to unlock the full potential of cell and gene therapy.
First, let’s clarify what we mean when we talk about CAR T therapies. The two FDA-approved CAR T therapies are examples of autologous treatment: a patient’s own blood cells are extracted, modified to recognize and destroy cancer cells, then returned to the individual patient, now with a new superpower-to find and kill the cancer cells.