Kristie L. Kahl
The first patient in the United States has been granted access to treatment with an investigational drug through the Right to Try Law of 2017 – legislation that allows patients who are terminally ill to try experimental drugs that have passed preliminary testing in phase 1 of the Food and Drug Administration (FDA) process but remain in clinical trial and are awaiting approval.
In late November 2018, ERC-USA and the University of California, Irvine (UCI), initiated treatment with the company’s investigational compound Gliovac (ERC1671) – which is currently in phase 2 clinical trials in the US – to treat a patient with glioblastoma
, an aggressive form of brain cancer.
According to an ERC-USA press release, the patient resorted to the Right to Try Law
after he failed to qualify for enrollment in an ongoing trial of Gliovac that was being conducted at UCI. Therefore, under the Right to Try laws enacted in California in 2017, the patient requested to receive treatment through ERC-USA. As a result, the company informed the FDA that it would be making the drug available to the patient, to which the agency acknowledged acceptance of such notification in July.
“The patient’s family requested access to the trial through Right to Try laws. (Lead investigator Daniela Bota, M.D., Ph.D.) agreed and sought to implement this access on their behalf,” UCI issued in a statement to CURE
. “It was believed that (Right to Try) offered a more expedited path to treatment, which UCI began after meeting regulatory and compliance requirements of state and federal Right to Try laws.”
According to righttotry.org
, the FDA approval process can take up to 15 years. The Right to Try Law is designed to expand access of these potentially life-saving drugs years before patients would have the option of being treated with them. To be eligible for Right to Try, a patient must meet the following conditions:
- diagnosed with a life-threatening disease or condition;
- have exhausted approved treatment options;
- unable to participate in a clinical trial involving the eligible investigational drug; and
- give written informed consent regarding the risks associated with taking the investigational treatment.
Right-to-try legislation is already a law in 41 states, and when President Donald Trump signed “S.204, the Trickett Wendler, Frank Mongiello, Jordan McLinn and Matthew Bellina Right to Try Act” on May 30, 2018, the legislation now applies federally – so long as a patient meets the qualifications, regardless if their state has adopted Right to Try or not.
With this, comes much controversy surrounding the legislation. Supporters of the bill say it is needed to give terminally ill patients more options, in part because the current FDA program does not help enough people and the agency’s approval process is too lengthy for these individuals to wait.
Those who oppose the bill, however, say that the use of unapproved drugs would bypass the authority of the FDA and undermine the drug development process, as well as patient safety. They also argue that the bill would not guarantee patient access to experimental drugs, because it would not require pharmaceutical companies to provide them.
In addition, opponents have argued that patients already have a route for gaining access to experimental drugs: the FDA’s “compassionate-use” or expanded access program
. The agency has also increased its initiatives with a plan to launch a new program this year – designed to help patients gain access to unapproved therapies by fielding telephone requests from physicians and patients, streamlining the application process, and acting as a middle man between physicians or patients and drug manufacturers.
When queried, the FDA was unable to provide comment at the time of publication due to the partial government shutdown.
Gliovac is an investigational cell-based vaccine based on freshly extracted tumor cells and lysates that stimulate a patient’s immune system to recognize and reject cancer cells. The vaccine contains cancer cells from the patient’s own tumor, as well as allogeneic tumor cells – which are composed of tumor cells isolated from the tumor of one patient, killed, processed and administered to another patient – from three different donor patients, and the lysates (a fluid containing the contents of broken-down cells that compromise its integrity).
The agent is intended to treat patients with grade 4 glioma when all other traditional therapies have failed. It is currently being evaluated as part of combination regimens to treat glioblastoma multiforme and gliosarcoma in randomized, placebo-controlled phase 2 clinical trials.
Interim data have demonstrated Gliovac may be a promising treatment for those who have exhausted other therapies, ERC-USA said in the release, adding that future studies will evaluate the agent to treat patients with newly diagnosed glioma with a predictive marker indicating unresponsiveness to Temodar (temozolomide) chemotherapy
When queried for comment, the company failed to reply at the time of publication.
“We appreciated the opportunity to provide an option for a patient with such advanced disease under California’s Right to Try act, which we believe is an important step forward in ensuring that all patients who may want to use this option would have access to medications that may prolong or improve their lives,” Apostolos Stathopoulos, M.D., Ph.D., president and CEO of ERC Belgium, parent company to ERC-USA, said in the release.