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First US 'Right to Try' patient dies, but ERC reports some positive Gliovac data


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First US 'Right to Try' patient dies, but ERC reports some positive Gliovac data

The first U.S. patient to receive an experimental treatment for recurrent glioblastoma under the federal Right to Try Act has died, according to a top executive at Epitopoietic Research Corp. But another patient with the aggressive brain cancer who also received ERC's investigational immunotherapy Gliovac has shown early signs of possible remission.

However, Joseph Elliot, managing director for ERC-USA, cautioned that it was still too early to know the durability of that possible remission since that patient has only been treated for about six months with Gliovac.

The product, also known as ERC1671, is an experimental immunotherapy based on freshly extracted tumor cells and lysates and is intended to stimulate the patient's immune system to recognize and reject cancer cells.

SNL ImagePresident Donald Trump signing the Right to Try Act, May 30, 2018.
Source: AP Photo

The patient with recurrent glioblastoma who died had been treated under Right to Try, or RTT, with Gliovac starting in late November 2018.

Daniela Bota, medical director at the University of California, Irvine School of Medicine's Health Comprehensive Brain Tumor Program, oversaw the treatment.

That patient was "very unhealthy" when the therapy began, Elliot said. The company has reported the death to the FDA.

About 75% to 85% of patients with glioblastoma die within 15 to 18 months of diagnosis, according to the U.S. National Institutes of Health. Without treatment, patients with recurrent glioblastoma often die within three months of being diagnosed.

ERC is not yet disclosing the date the patient died or how long the person had been treated with Gliovac.

RTT program

The company has been testing Gliovac in an ongoing placebo-controlled phase 2 clinical trial in patients with recurrent glioblastoma, the most aggressive and deadly form of brain cancer.

Bota is the principal investigator.

But ERC has also been providing Gliovac to a small number of patients who meet the criteria under the Right to Try Act, which permits critically ill people to sidestep the U.S. Food and Drug Administration in accessing experimental treatments.

ERC's RTT program is pay-to-participate, meaning patients or their representatives are required to cover all or part of the costs.

ERC is not offering the product under the FDA's expanded access program, also known as compassionate use, Elliot told S&P Global Market Intelligence. The company's RTT policy emphasizes that "ERC believes that participating in clinical trials is the best way for patients to access medicines prior to approval."

The company also said it requires ethics approval and oversight for all RTT cases, whether mandated by the jurisdiction or not. The federal RTT law does not require oversight of an institutional review board, an independent body that ensures ethical and regulatory requirements are met for research involving humans.

President Donald Trump has insisted the Right to Try Act is a "miracle" that has saved "many lives" — a claim not backed up by evidence. Critics of the law have raised concerns that unscrupulous practitioners could market unproven products, including stem cell treatments, to desperately ill patients, which could harm them.

"The hype around Right to Try as providing miracles for users, danger of misunderstanding around the known efficacy and safety of these treatments and vulnerability of the participants points out the need for careful oversight of those offering treatments via Right to Try," Jeremy Snyder, a professor and bioethicist at Simon Fraser University, told S&P Global Market Intelligence.

Snyder said ERC should be applauded for its commitment to involve IRB oversight in its program and for reporting the death of a patient receiving its treatment to the FDA ahead of the agency issuing its overdue annual RTT reporting requirements.

But he said it remains concerning other companies may choose not to follow ERC's lead in requiring IRB oversight.

Other patients

Bota is now using Gliovac in a second patient with recurrent glioblastoma under the federal RTT law. That patient started treatment in early summer, Elliot said.

ERC does not have many details on that second UC-Irvine patient because of confidentiality requirements, but Elliot said the company is aware the person has continued to improve because patients must meet a certain level of immune function to be able to progress on Gliovac.

Many patients with recurrent glioblastoma have previously received treatment with chemotherapy and radiation, so the function of their immune cells is reduced, Elliot said.

If that function is too low, it would be futile for patients to use Gliovac because their immune system would not respond, he said.

A third patient who is being treated under RTT at the John Theurer Cancer Center in Hackensack, N.J. has shown signs that his recurrent glioblastoma may be in remission — a status not often observed with the disease, Elliot said.

That patient is one of five being treated with Gliovac under RTT at the John Theurer Cancer Center by neuro-oncologist Samuel Singer, Elliot said.

Those patients started the therapy in late spring, he said. A sixth patient is expected to also soon be treated with Gliovac under RTT, he added.

While all five of Singer's patients remain alive, Elliot again cautioned they have received the therapy for a relatively short period.

"In order to make it meaningful, we need to see how these patients do over 12 to 24 months," he said. "And if there is a real benefit, that's when it will become overwhelmingly obvious."

ERC plans to publish data on the RTT patients within the next 12 months.

Phase 2 trial

Meanwhile, this past weekend, Bota reported preliminary results of the phase 2 trial at the annual meeting of the Society for Neuro-Oncology in Phoenix. The early data demonstrated a median survival of 50% greater in patients treated with Gliovac versus those in the control group, 11.7 months compared with 6.5 months, Elliot said.

Elliot acknowledged the phase 2 Gliovac trial consists of a small population of fewer than 20 patients.

However, ERC plans to soon start enrolling patients in the trial at a second clinical site at Dana-Farber Cancer Institute in Boston, he said.

Elliot said ERC plans to seek FDA fast-track designation, an expedited review process for medicines intended to treat serious and life-threatening conditions.

The company anticipates completing the phase 2 trial in 12 to 18 months, he said.