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Originally published May 8, 2026
Last updated May 8, 2026
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With some cancers where there aren’t many FDA-approved treatments, thinking innovatively is the only way to treat a patient, says Minh Dang Nguyen, MD, a medical oncologist with USC Norris Comprehensive Cancer Center, part of Keck Medicine of USC. “With a cancer like breast cancer, we have hundreds of drugs that we can use that we know are effective; I wouldn’t veer from the standard of care,” he says. “But for cancers that have really no good treatment options, you have to think outside the box and start looking at other drug options and pathways of care.”
Brain tumors are one area where oncologists need to be creative in their treatment options. There hasn’t been a new approved drug treatment option for glioblastoma in over 20 years, Nguyen says. “So, when you see a brain tumor with a specific type of genetic mutation, you might need to look at another drug that targets that mutation,” he explains.
For example, if a glioblastoma cancer tumor overexpresses the PDL1 protein, doctors can sometimes get creative with using immunotherapy drugs (like pembrolizumab) to target PDL1. This is especially important in lethal cancers with few treatment options, such as glioblastoma.
“Typically, glioblastoma cancer patients only survive for 12 months,” Nguyen says. “If a patient’s glioblastoma happens to express the PDL1 protein, this is a situation where we need to start thinking about using these other drugs that target PDL1 off-label, even though this drug is not quite FDA-approved to treat glioblastoma.”
Most of the time in these situations, you can drive a patient toward a clinical trial. But sometimes, it’s necessary to use other pathways to get a patient covered for an off-label drug. One way Nguyen suggests doing this is through applying for compassionate use with a drug manufacturer, so that a patient can still receive some type of treatment through an off-label use of a particular drug.
While oncologists normally have had freedom to treat patients with pioneering methods, especially in life-threatening cancers, it is becoming more difficult to do so, Nguyen says.
“It’s getting more and more challenging to think outside the box,” he says. “Even as more specialized technology is developed, you still must get these treatments approved by insurance, and sometimes they can be very expensive.”
These requests can even come from the patients themselves, who might have heard about the treatments through their own research, he adds. You might have to petition insurance companies to get more specialized tests like PET scans. “And to get these approved by insurance sometimes requires going to bat for them and doing peer-to-peer reviews,” he explains.
One common way to think creatively in oncology is through clinical trials, Nguyen adds. This is especially important with rare cancers.
“Every cancer type could potentially have a rare form based on the histology of the tumor,” he says. “When you have a rare cancer, you want to try to find a clinical trial that the patient will fit into, but oftentimes there may not be a trial for them or the trial is too far away and they’re not willing to travel.”
For patients in the Los Angeles metro area, luckily there are usually many options for clinical trials. But for smaller, rural areas, where the closest research hospital might be a two- or three-hour drive away, community practitioners might need to get creative with treatments, Nguyen says.
“In these situations, a physician might need to do their own research on the latest science surrounding a cancer and try to mimic treatments from clinical trials on their own,” Nguyen says. “But for doctors practicing in a large metropolitan area, this can be an opportunity to refer a patient to a higher level of care or larger medical practice for more specialized care and access to a full tumor board of multidisciplinary specialists.”
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