Study: Cancer patients benefit from Phase I drugs

A new study published in the journal Clinical Cancer Research has found that patients with terminal head and neck cancer survived just as well on unapproved, Phase I treatments as FDA-approved drugs.

A team headed by University of Texas M.D. Anderson Cancer Center researcher Dr. Ignacio Garrido-Laguna followed 61 patients over five years who'd participated in an early-stage trial. Fifty-nine of them had previously received an FDA-approved treatment for their cancer. The group compared how well the patients did on the approved treatment without disease progression compared to treatment with Phase I drugs. According to their findings, patients survived an average of 12 weeks on approved treatments versus 10.7 weeks on unapproved drugs. Statistically, the outcomes were comparable. 

Terminal cancer patients have long wanted greater access to experimental drugs. But providing unapproved drugs to patients outside of a clinical trial can be extremely expensive, entails a certain amount of risk, and requires the FDA's support. And developers must keep an eye on the FDA's ever-increasing demands for stringent clinical data.

Critics worry that terminally ill patients desperate for a cure could be taken advantage of by companies who are only just starting to test their drugs in humans. "There are worries about whether people who are vulnerable in this way are able to give informed consent" Dr. Franklin Miller, a bioethicist at the National Institutes of Health, explains to Reuters.

Many cancer patients are inelidgible for later-stage drug trials because of strict inclusion criteria. But those with terminal cancer who've exhausted every available approved drug option may be willing to try something radical in the hopes that an early-stage drug could slow disease progression. Observes Miller, "there really isn't any better option for them. I think this is genuinely what many people want to do."

- read the Reuters story for more