The 'Right to Try Act': A New Hope for Terminally Ill Patients The passage of the federal Trickett Wendler, Frank Mongiello, Jordan McLinn, and Matthew Bellina Right to Try Act of 2017, signed into law by President Trump, was a landmark moment intended to give terminally ill patients more control over their treatment options. The law creates an alternative pathway—separate from the FDA's existing Expanded Access program—allowing patients with life-threatening conditions who have exhausted all approved treatments and are ineligible for clinical trials to access certain unapproved, investigational drugs. Advocates championed the law as a way to liberate patients from bureaucratic red tape, arguing that individuals facing a terminal diagnosis should have the autonomy to try any treatment that their doctor and a drug manufacturer agree upon. This movement is often characterized as directly challenging the established power structure of "Big Pharma" and the stringent, lengthy process of drug development and approval. The law provides liability protection for manufacturers, sponsors, and physicians who provide these experimental treatments, aiming to encourage wider access. Repurposed Drugs and the Search for Cures In the context of the Right to Try Act, attention has surged around drugs already approved for other uses—specifically the antiparasitic medications Ivermectin and Fenbendazole. Scientific Interest in Repurposed Drugs: Repurposing existing, often inexpensive, drugs for new indications like cancer is an active area of research. These drugs have established safety profiles in humans, potentially speeding up their use in patients. Ivermectin's Potential: Laboratory and animal studies have demonstrated that Ivermectin possesses significant anti-cancer properties. Research suggests it may inhibit the proliferation of various tumor cells by inducing apoptosis (programmed cell death) and blocking key cellular pathways involved in cancer growth, such as the PAK1 and WNT-TCF pathways. A Phase II clinical trial investigating Ivermectin in combination with an immunotherapy drug for metastatic triple-negative breast cancer is currently underway, highlighting growing scientific interest. Fenbendazole's Promise: Fenbendazole, primarily used in veterinary medicine, has also shown anti-tumor effects across numerous human cancer cell lines in preclinical studies. Its mechanism is believed to involve disrupting microtubule formation (similar to some chemotherapy drugs), inhibiting glucose uptake in cancer cells (a process known as the Warburg effect), and activating the p53 tumor-suppressor pathway. The Ongoing Debate While the narrative of "Big Pharma exploding" resonates with many patient advocates, the reality of the Right to Try Act's impact is complex. The law gives manufacturers the option, but not the requirement, to provide experimental drugs. Concerns from some medical professionals and organizations center on the lack of FDA oversight in this pathway, the potential for patients to receive treatments with unproven efficacy and unknown side-effect profiles, and the possibility of diverting patients from established clinical trials. The use of Ivermectin and Fenbendazole for cancer, while supported by promising preliminary research, still lacks the robust, large-scale human clinical trial data required by the FDA and mainstream oncology for standard recommendation. The Right to Try Act, however, offers a legal mechanism for terminally ill patients to pursue these and other investigational therapies in consultation with their physicians, empowering a personal health choice when all other options have been exhausted. It represents a fundamental challenge to the traditional drug approval hierarchy, placing the ultimate decision in the hands of the patient and their doctor.
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