This article is part of the Global Policy Lab: Decoding Cancer.
Genomics, its said, has the potential to turn every cancer into its own unique snowflake, to be tackled not with off-the-shelf treatments but a personalized regimen. That threatens to make the gold standard of drug testing, the randomized clinical trial, look a bit tarnished.
Here are four ways cancer is changing the way we test drugs and treatments, from the perspective of industry, regulators, doctors, researchers and patients.
Out-of-body
In a typical drug trial, the manufacturer sends pills to patients, identical little capsules to be swallowed. Not so for personalized therapies like the prostate cancer treatment currently being tested by Sotio, a mid-sized biotech in the Czech Republic. Instead, patients blood samples are collected in more than 200 sites in more than 20 countries and then sent to Prague, where technicians re-engineer cells to fight their cancer and send them back to the patients.
That makes it “extremely complicated from the logistics point of view,” said Radek Špíšek, Sotios chief executive. In addition to satisfying the usual medicines regulators, the company had to work things out with transport agencies to get the blood on planes. Some countries wanted to put the samples “through X-rays which would kill some cells,” Špíšek said. Once the blood arrives at Sotios site, each treatment for the nearly 1,200 study participants has to be created in a separate room, to avoid mixing them up — a physical space constraint that slows the process down.
Minor additions
Clinical trials typically start recruiting patients at age 18, which means teens dont get much access to experimental treatments. “Physiologically, there is no reason why a 16-year-old couldnt go into that trial,” said Pamela Kearns, the president-elect of the European Society for Paediatric Oncology. There are no legal barriers, either, she said, but rather practical ones: “It now means that youve got that trial not just in an adult unit but also in a pediatric unit.”
Theres a growing movement to bring young patients into trials — driven in part by activist networks of young adults — Kearns said, and a video is making the rounds calling out both scientists and pharma companies for not doing just that. “From personal experience, this is changing in the U.K., but weve got a long ways to go,” said Kearns. “In the rest of Europe, were just beginning to get the message out there.”
Less evidence of more
As we pursue cures for rare cancers, studies are getting smaller; but the breakthroughs can nonetheless be dramatic. Thats convincing drugs regulators that the benefit of a medicine is worth approving, even if developers cant produce the volume of stats theyre accustomed to.
The European Medicines Agencys senior medical officer, Hans-Georg Eichler, cited statins, a treatment for high cholesterol, as an example of traditional testing; in a study with over 1,000 patients, regulators might see 4 percent of patients have a heart problem with the medication, and 6 percent without, and they have to figure out whether that little difference is thanks to the drug. But with rare cancers, the math plays out differently. If a killer cancer starts going into remission in most of the 70 patients receiving an experimental drug in a small trial, “it is in patients best interest to allow a product onto the market based on smaller studies,” Eichler said.
Studies could get even smaller. Rafal Swierzewski, an associate consultant for the European Cancer Patients Coalition, noted the possibility of a clinical trial of one, to see what works for that snowflake cancer. Until recently this possibility existed primarily in the imaginations of academics. But now some companies are giving it a try.
Never-ending trial
Glioblastomas, a type of brain tumor, kill 95 percent of patients within five years. In the coming days, an effort called the GBM AGILE trial will announce the first new therapy it will test with an ambitious new approach. The plan is to create a clinical study that never comes to an end and can incorporate any patient and try out a range of treatments. Instead of “recreating the wheel every time we wanted to create a new drug or a new therapy, were just going to figure out the best way to do it,” and keep repeating, said Brian Alexander, a U.S.-based radiation oncologist behind the effort.
The trials wouldnt necessarily stay random in the traditional sense, Alexander said. And that could benefit the participants. “Say halfway through the trial, we have a therapy that is doing much better than [the placebo or existing standard treatment]. Well we can kind of put our finger on the scale, so to speak,” he said, and send a higher proportion of patients to receive the promising treatment. The GBM AGILE trial will kick off in North America and Australia, Alexander said, but the aim is to expand to Europe and Asia.
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