In the world of cancer treatment, patients have long endured harsh side effects from powerful drugs in exchange for extended survival. However, a growing movement is questioning whether this suffering is necessary. The U.S. Food and Drug Administration (FDA) is urging drugmakers to find the lowest effective dose, even if it means taking more time.
Advances in cancer treatment have allowed millions of people to live for years with incurable cancers. For example, Jill Feldman has survived 15 years with lung cancer thanks to progress in treatment. However, the drug she takes causes joint pain, fatigue, and mouth sores that make eating and drinking painful. Feldman, along with other patients and doctors, advocates for drugmakers to study lower doses early in the research process.
Traditionally, cancer drug development has focused on finding the "maximum tolerated dose," ramping up the dosage in early studies to determine the highest dose patients can handle. While this approach works for chemotherapy, it may not be suitable for newer targeted cancer drugs. These drugs work differently, often targeting specific mutations or boosting the body's immune system.
The FDA's Project Optimus aims to include more patients in early dose-finding trials to gather better data on when lower doses can be effective. The motivation behind this project stems from the demands of patients and advocates for more tolerable cancer drugs.
It is common for patients to skip doses or stop taking drugs due to side effects, leading to problems in treatment. Some dose recommendations have been officially lowered after drug approval, while others are adjusted on a case-by-case basis. A study found that nearly half of patients in late-stage trials of targeted therapy drugs needed to have their doses lowered.
Finding the right dose is challenging due to patient-to-patient variation and the risk of underdosing some patients. However, researchers are working to identify the "sweet spot" where the drug is effective without causing excessive side effects. Studies are being conducted to test lower doses and different dosing strategies for various types of cancer.
While adjusting doses and conducting more head-to-head dosing comparisons may slow down the drug development process, it is believed that it will lead to more effective drugs in the long run. Ensuring that patients can tolerate the drugs and maintain their quality of life is crucial for successful treatment.