After decades of research to advance cancer progress, an average of 68% of medicines in the oncology pipeline today are likely to be first-in-class, meaning they use a new and unique mechanism for treating a disease. The pipeline is also ripe with innovative therapeutic approaches, like mRNA, with the potential to transform a wide range of cancers — many which have already seen approvals in recent years. For example:
• Immunotherapies, which include monoclonal antibodies and CAR-T, is an approach that works by unleashing the immune system to target and kill cancer cells.
• Gene editing involves manipulation of DNA at particular locations in order to treat a specific cancer.
• Oncolytic viral therapies work by zeroing in on cancer cells, to replicate and cause them to rupture.
• Antibody Drug Conjugates target specific cancer cells with cytotoxic agents without harming normal cells.
Small molecule medicines, which represent the majority of cancer medicines, typically come in the form of a tablet or capsule, are taken by mouth and contain a single chemically synthesized active ingredient. Due to their size, small molecules can more easily reach therapeutic targets inside of cells, cross the blood-brain barrier, and are often available in oral dosage forms which offer greater flexibility and convenience in their administration and ultimately reduce barriers to treatment adherence and factors that can drive health disparities. For cancer specifically, targeted small molecule therapies can act upon specific proteins or genetic material inside cancer cells, causing cancer cells to die. Given cancer begins with genetic changes occurring inside cells, targeted small molecule medicines provide an essential tool in combating the cause of cancer.
Biologics, also referred to as large molecule medicines, in contrast are made by or from living cells, are structurally complex and are generally administered in a doctor’s office or hospital setting via injection or infusion. Due to their larger size, biologics are generally unable to enter cells, but rather are designed to reach therapeutic targets on the surface of cells.
Together, these two types of medicines offer patients and health care providers a wide choice of treatment options that are needed and are indispensable in the treatment arsenal against cancer.
Since 1998, the FDA has approved 111 medicines across the cancers, but there have been a total of 1315 unsuccessful investigational drugs. So-called “failures” are an inherent part of the process because treating human disease is one of the most complex undertakings on the planet. But these projects are not wasted efforts as their findings inform future study and direct research efforts toward new approaches to addressing the cancer causes, growth and progression.
Cancer continues to be a major challenge, but biopharmaceutical companies have over 1,600 potential cancer medicines in development and are dedicated to transforming cancer from a devastating diagnosis to a chronic, manageable condition. This goal drives researchers past the setbacks to discover how to apply the knowledge gained to inform the development of innovative medicines that bring hope to patients and their families and ultimately win the battle against cancer.
Biopharmaceutical innovation and new drug discovery delivers far-reaching benefits to patients, the U.S. health care system and our state and national economies. New cancer medicines discovered and developed by America’s biopharmaceutical research companies are helping patients lead longer, more productive lives, controlling health care costs and stimulating the economy through high-quality jobs and a healthier workforce.
Updated as of December 2023.