A cancer vaccine being developed by Epitopoietic Research Corp. has become the first drug to be given to a U.S. patient under new “right-to-try” legislation that gives terminally ill patients access to experimental drugs.
ERC has said (PDF) it informed the FDA last June that it intended to make the vaccine—called Gliovac (ERC-1671)—available to a California patient who hadn’t met the eligibility criteria for enrollment in the company’s ongoing phase 2 U.S. study in brain cancer.
The agency acknowledged the request last July, and in November the unidentified patient, who had been diagnosed with recurrent glioblastoma, was treated with the vaccine at the University of California, Irvine, School of Medicine.
The right-to-try law dictates that any compound used in this way must have successfully completed phase 1 safety testing and be under the FDA’s ongoing approval process. The patient only needs a physician and the drugmaker to sign off on the request, although the FDA has to be informed.
Critics say the law will have little impact on medicines access, could expose patients to exploitation by unscrupulous parties and is unnecessary given that the FDA already approves (PDF) 99% of requests to access unapproved treatments under compassionate-use policies.
UC Irvine neuro-oncologist Daniela Bota, M.D., Ph.D., insists however that the right-to-try laws “may be the only alternative for many patients who may not qualify for clinical trials based on a variety of factors, including progression of disease, comorbidities, existing medications, physical limitations and others.”
ERC, meanwhile, has made extensive use of compassionate-use programs outside the U.S., with more than 28 patients with recurrent, terminal gliobastoma treated with ERC-1671 in Belgium, Germany, Colombia, South Africa and Australia by the end of 2018.
The vaccine is based on freshly extracted tumor cells and lysates that, according to ERC, “stimulates the patient’s immune system to recognize and reject cancer cells.”
The Goldwater Institute, which is an advocate for right-to-try, says the law is important because it allows patients to “seek … treatment from the manufacturer without having to first navigate bureaucratic and time-consuming federal red tape.” It notes that in addition to the federal law, 41 states have implemented their own right-to-try laws.
Last year, Israeli biotech BrainStorm backed away from providing an experimental treatment to a patient with neurodegenerative disease amyotrophic lateral sclerosis under right-to-try. In that case, the decision stemmed not from ethical, legal or practical consideration but because the company decided it wouldn’t be possible to fund treatment for all patients who wanted to try it.