From initial success in the lab to promising results in patients, new medicines and therapies have to clear many hurdles before they can be disseminated to the general public. CDX-110, the brain tumor vaccine developed by Duke neurosurgeon John Sampson and manufactured by AVANT Immunotherapies, is well on its way to becoming part of the new treatment regimen available to oncologists.
This year, Pfizer, in conjunction with AVANT, is launching a multisite Phase II/III study to determine whether CDX-110 should become the new standard of care for patients diagnosed with glioblastoma multiforme (GBM) tumors. More than twenty brain tumor centers across the country are participating in the randomized study.
GBM has long been considered an "orphan disease," a designation for conditions that affect fewer than 200,000 people. People diagnosed with orphan diseases often find that therapies to treat their conditions are scarce, owing to the huge financial commitments that underlie research and development. But because CDX-110 targets a mutant protein found in a host of other cancers, the pharmaceutical industry has taken an interest in its development.
"To go to a Phase III trial takes hundreds of millions of dollars these days," says Sampson. "Typically that requires having a huge venture capitalist or big pharma getting involved. In this case, big pharma is getting involved." If the CDX-110 trials go well, he says, Pfizer Inc., one of the world's largest pharmaceutical companies, will conduct the final round of testing before applying for FDA approval to market the drug. In mid-April, Pfizer paid AVANT $40 million and promised a $10 million equity stake for the worldwide rights to the vaccine.
Since the vaccine is only effective in treating tumors with a particular mutation, it won't ever be a cure-all for people diagnosed with GBM. Still, it's more promising than anything else on the market. (Temozolomide, the most recent chemotherapy drug used to target brain tumors, only extends survival rates a couple of months, on average.) Given the slow pace of getting drugs tested and approved for use in the general population, though, the vast majority of people currently diagnosed with GBM will be dead before CDX-110 receives final market approval.