Companies Continue to Take Aim at Glioblastoma, a Cancer Highlighted by the Death of Sen. John McCain

John McCain in a suit with a red tie, smiling in front of a podium

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Long before Sen. John McCain was diagnosed with brain cancer, I got the chance to briefly meet the man who developed a reputation as a maverick in the Senate. It was in Raleigh, N.C. about 12 years ago, and he was in town pushing for support of public financing of judicial elections in the Tar Heel State.

I was a political reporter with The Wilson Daily Times (now The Wilson Times) and covered McCain’s push for the plan, something for which he was a proponent. After the presentation, I got to spend a few minutes with the senator. I don’t recall the details of the conversation, only the kindness and character-defining way in which he treated me and all the reporters in the room. Although I no longer write about politics, I (sort of) crossed paths with Sen. McCain last year after he revealed he had been diagnosed with brain cancer. McCain died Saturday after deciding to discontinue treatment for the cancer. He approached the end of his life in the same manner in which he approached me that day in Raleigh – with the strength of character.

When McCain revealed his brain cancer last year, BioSpace pointed to 10 companies that were in pursuit of treatments for glioblastoma, the most common form of brain cancer and one that is highly aggressive. The disease is somewhat rare, impacting only about three out of every 100,000 people in the United States.

Although it’s a rare disease, companies are still chasing after a potential treatment. Citing the National Institutes of Health (NIH), CNN reported there are currently 274 glioblastoma multiforme studies underway or recruiting across the United States.

One early-stage treatment has been receiving some press due, primarily, to one patient who has shown a remarkable response to the treatment. Bob Rulli, a retired airplane engine designer, was diagnosed with glioblastoma multiforme five years ago. He has been a clinical patient of startup Bexion Pharmaceuticals. Rulli has been undergoing treatment with BXQ-350, a formulation of a synthetically produced, human lysosomal protein, Saposin C (sphingolipid activator protein, or SapC), and the phospholipid dioleoylphosphatidylserine (DOPS). Average life expectancy is less than two years. Rulli has been on the Bexion treatment for 19 months. His cancer hasn’t been eliminated, but the tumor has proven to be stable during that time. BXQ-350 is currently in Phase Ia.

Earlier this month AiVita Biomedical initiated its first clinical site for the ROOT OF CANCER Phase II trial in patients with newly diagnosed glioblastoma. Patients will receive autologous dendritic cells loaded with autologous tumor antigens derived from self-renewing tumor cells as an adjunctive therapy following primary surgery plus concurrent chemoradiation, the company said.

In April, China-based CANBridge Life Sciences announced plans to initiate its Phase II/III glioblastoma multiforme trial following the nod from the China Food and Drug Administration. The company’s lead candidate, CAN008 is a “fully human fusion protein that inhibits the CD95 ligand, a member of the tumor necrosis factor (TNF) family,” according to company data. By blocking CD95, CAN008 “restores the immune system’s anti-tumor response and inhibits invasive cell growth,” the company said.

In December 2017, Virginia-based Diffusion Pharmaceuticals initiated a Phase III trial for its lead glioblastoma product, a small molecule trans sodium crocetinate (TSC). Phase II results showed that in an inoperable patient subgroup, the led candidate helped them achieve a “nearly four-fold increase in survival compared with historical controls when TSC was added to their treatment regimen,” the company said. Data showed that 40 percent of the patients were alive at two years vs. 10.4 percent.

In 2016, Celgene acquired marizomib, a novel, brain-penetrant proteasome inhibitor. The company is developing the therapy as a possible treatment for patients with newly diagnosed glioblastoma. The therapy is entering Phase III.

Last year Inovio and Regeneron partnered on a brain cancer study. Inovio will pair two of its investigative products, INO-5401 T cell activating immunotherapy encoding multiple antigens and INO-9012, with Regeneron’s PD-1 inhibitor, REGN2810.

There have also been some setbacks with glioblastoma research.

ZIOPHARM Oncology announced a delay this spring in its planned Phase III glioblastoma treatment, Ad-RTS-hIL-12 plus veledimex. The company is developing a gene therapy product designed to treat patients with recurrent glioblastoma (rGBM) as a monotherapy and in combination with an immune checkpoint inhibitor. In May the company announced it had to delay the trial in order to resolve technical requirements related to Chemistry and Manufacturing Control (CMC). This was the second delay for ZIOPHARM’s study.

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