She had less than a year to live with a brain tumor. This treatment saved her life

Five years ago, Eryn Campbell was told she would likely have just six to nine months to live.

The young woman was diagnosed with a glioblastoma, the most aggressive type of brain tumor, in 2015 when she was going to a college for medical assistants in New Jersey. She received the standard of treatment at the time, which included surgery, radiation and Temodar, an oral chemotherapy treatment.

After moving to Cape Coral on Florida’s west coast in 2017, Campbell learned that her cancer had progressed. Doctors referred her to the Sylvester Comprehensive Cancer Center at the University of Miami, where she enrolled in a clinical trial for a targeted cancer treatment for tumors with certain genomic characteristics.

“It was scary to enter a clinical trial because you don’t know what’s going to happen or if it is even going to work. But after the first month, we saw it start to shrink – amazing,” said Campbell. And it continued to shrink.

In the 2 1/2 years on this clinical trial, her tumor has reduced in size by about 70% to 75%. Campbell is still enrolled in the trial and takes two pills a day and returns to Miami about once a month for a visit with her Sylvester neuro-oncologist, Dr. Macarena de la Fuente, and a series of blood work and MRIs to make sure she is tolerating the drug.

Now 24, Campbell describes her quality of life as very good. “I live like a normal person, I work as a waitress, I can travel, and my family and friends are very supportive. I do plan on going back to school, I’m just not sure when.”

Targeted cancer therapies like the one Campbell is undergoing are not only prolonging lives for many patients but they don’t come with the long list of usual and sometimes debilitating side effects that chemotherapy does. Some of these new targeted drugs passed their clinical trials and are now FDA approved and available commercially. Insurance companies typically cover them, too.

As for Campbell, “the main benefit in her case is that she has been doing much, much better than expected. In her particular case, this is prolonging her life,” said de la Fuente, the center’s chief of Neuro-Oncology. “It’s an excellent result – not something we have seen every day, a young patient with an excellent quality of life. This is good.”

Campbell can likely stay on the drug as long as the medicine keeps working for her and she is tolerating it well, de la Fuente said. The sponsor of the clinical trial covers all medical expenses associated with it.

“I don’t want to create false expectations -- this is a very difficult disease to treat, and I always like to be very honest with the patient,” the doctor said. “But this gives us hope for the patients with primary brain tumors. Doing clinical research, we can find there are treatment options for them.”

Primary brain tumors are tumors that start in the brain. But it turns out targeted therapies are showing promise for a number of cancers.

Unlike traditional chemotherapy, a shotgun approach that kills the unwanted cancer cells as well as tissue around it and often comes with side effects including dropping white blood counts, nausea, hair loss and infections, targeted therapies attack the specific mutation in the cancer genes that is driving their growth.

Let’s take a look at a few ways targeted cancer therapies are being used in South Florida.

NICKLAUS CHILDREN’S HOSPITAL

Like at Sylvester and other South Florida hospitals, Nicklaus Children’s Hospital houses a number of ongoing clinical trials for targeted therapies. Among them: Doctors at Nicklaus are taking part in trials for new targeted treatments for the most common pediatric brain tumor, low grade astrocytoma, as well as for plexiform neurofibromas, which can cause pain or disfiguring, and high grade gliomas.

“Doctors found that certain drugs – and more are on the way – can inhibit growth and in many cases cause shrinkage or remission of the cancer,” said Dr. Ziad Khatib, director of Neuro-Oncology at Nicklaus Children’s Hospital. Targeted treatments typically involve taking a pill, with far fewer and less debilitating side effects.

The trials look at the effectiveness of inhibitors – for low grade astrocytoma, it is the MEK inhibitor – which target the mutated pathways that allow uncontrolled growth in the cancer cell, Khatib explained. Two trials that look at the combination of two inhibitors – MEK and BRAF – on low and high grade gliomas when a specific mutation is detected are looking surprisingly effective with fewer side effects, he said.

These treatments require a deep knowledge of the tumor, “like the DNA fingerprint,” the doctor said. “Every tumor that is removed undergoes 500-gene molecular testing so we know how to classify the tumor and to look for targets that will help us find a drug suitable for that treatment.”

Targeted treatments that have been FDA approved can cost thousands of dollars a month, but Nicklaus has found that generally insurance companies will cover them.

“I’m excited about this new era in cancer treatment -- molecular diagnosis and molecular treatment,” Khatib said. We are not as blind as we used to be because we know what we are treating with DNA evidence.”

BAPTIST HEALTH

Dr. Rupesh Kotecha is a radiation oncologist at Miami Cancer Institute, part of Baptist Health South Florida. He specializes in both lung and brain cancers, and there is a good reason for that dual expertise.

Primary brain tumors, or tumors that start in the brain, are rare. Brain metastasis, cancer that started someplace else in the body and spread, is the most common type of cancer in the brain. Annually in the U.S. there are between 170,000 to 400,000 cases, he said.

And most commonly, brain metastasis come from lung cancer.

“Whenever a patient is diagnosed with cancer, and especially in lung cancer in the modern era, we need to know the stage of the cancer and also the molecular profile of the actual tumor, Kotecha said. “For lung cancer patients, knowing the particular mutation is important because we use personalized therapies.”

If doctors can determine what molecular alteration is driving the growth and spread of that patient’s cancer, they can prescribe a targeted drug that will be more effective than the toxic chemotherapy or even immunotherapy because it is personalized to that patient – and it is safer, Kotecha said. Some are oral so they can take a pill rather than have to get an infusion every two or three weeks.

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The new molecular therapies can penetrate into the brain and treat brain metastasis. There is a drug that patients can take for their EGFR-mutated non-small cell lung cancer and this has been shown to have a response rate of 50% to 70% in brain metastasis, he said. Response rates for a more rare mutation have been similar.

“What we have learned is that the targeted therapies work against specific mutations. Those cancers are molecularly different. They have a specific molecular feature that has been altered in the cancer that is their driver for their growth or proliferation,” he said. These drugs can effectively turn that switch off, prolonging lives.

MEMORIAL HEALTHCARE SYSTEM

Targeted therapy research has come a long way in 10 years and the future holds much promise, says Dr. Luis Raez, medical director of Memorial Cancer Institute and a lung cancer expert.

In 2018, Raez was part of the group of researchers who published in the New England Journal of Medicine a discovery of a target agent, now commercialized and called Larotrectinib, that can target 17 tumors at the same time. It was “a revolution in target therapy,” said Dr. Raez. The patients in the study were stage 4, considered incurable, and these agents added an average of four years of a good quality of life to these patients, he said.

An ongoing study at Memorial with 11 patients enrolled focuses on a new drug, now called Loxo-292, that can target lung, colon, thyroid and pancreas cancer, as long as a patient has that genetic marker or aberration, called RET. “I am a lung cancer doctor but thanks to this agent I am treating now thyroid cancer, I’m treating colon cancer, things I never imaged to treat.” The trial started more than a year ago, and only one patient has failed treatment, he said.

What’s more, an upcoming clinical trial will investigate the combination of targeted therapy and precision radiosurgery for this patient population.

Bigger picture: “We try to discover why the tumor arrives. We look for the genetic markers. The location is not enough for us anymore,” Raez said. “Now the race is to find for each gene a therapy for that cancer.”

Discovering all these treatments will take years, he said.

“One by one, we have to keep discovering the drugs for each gene, that’s a lot of work because there are a lot of genes.”

Contact the writer at ndahlbergbiz@gmail.com and follow her on Twitter @ndahlberg.

This story was originally published May 26, 2020 at 6:00 AM.