Patient’s Perspective: The Importance of Clinical Trials

Update:  We are deeply saddened to report that Neil passed away on July 29, 2015. It is a privilege to share his story and keep his memory alive.

Neil Schiffman has lived as healthy a life as one can live. An avid cycler and triathlon participant well into his 60s, Neil was visiting Arizona in April 2011 when he began to cough. The cough mimicked the symptoms of exercise-induced asthma, so at first he thought nothing of it. When the cough failed to disappear, he visited his pulmonologist, who suspected bacterial pneumonia. Meanwhile, Neil’s legs and feet began to swell–he went from wearing a size 10 shoe to a size 14. Alarmed, he had an X-ray and a CT scan, which revealed a mass identified as a neoplasm. He was referred to an oncologist, who delivered the bad news in June: Neil had stage IV non-small cell lung cancer (NSCLC).

Fewer than 10% of stage IV patients survive for longer than five years, and the median survival time is less than eight months. But Neil, now 65, is quickly approaching the two-year anniversary of his diagnosis. Perhaps the reason Neil has defied the odds is his tireless pursuit of information about lung cancer, in general, and his own tumor, which put him on a path to volunteer for a clinical trial and receive a more personalized treatment program.

The first thing Neil did after hearing his diagnosis was resume his composure and research the disease that had now changed his life. “I’d always had a bias of [lung cancer] as ‘smoking disease,’ ” says Neil, a never-smoker. He says he’s not a person who gets depressed, but after discovering the short lifespan for stage IV patients, he was definitely below the ‘neutral zone.’

Nevertheless, the research helped him overcome his fear of the unknown. “Knowledge is power,” he says. And after all, somebody has to beat the odds.

Neil scheduled an MRI of the brain and a bone density scan, then underwent a biopsy on his tumor. When the results came back, he discovered his tumor had a mutation in the EGFR gene. Such mutations are present in about 15 percent of NSCLC patients. Having the biopsy was the first step on Neil’s path toward individualized medicine; knowing his EGFR status meant he could predict with a great degree of certainty which drugs would work for him and which ones would not.

Neil saw a clinician at Stanford University on June 30 – his wedding anniversary – less than a month after his diagnosis. By July 14, he had been prescribed an oral drug, Tarceva (erlotinib), which acts to inhibit the EGFR mutation. “The time period between symptoms to diagnosis to the start of treatment seemed like an eternity,” he says. “But I later learned that it was really incredibly quick.”

Tarceva began to have a noticeable effect by the fifth or sixth day. Neil’s cough started to subside, and his legs and feet began to shrink back toward their normal size. On July 29, he was able to go on a bike ride with a friend, an uplifting accomplishment for a former cross-country cyclist.

A major side effect associated with Tarceva is a rash; though uncomfortable, it indicates that the drug is having the desired effect. Neil’s rash was small and completely bearable. The main side effect he had from the drug was elevated energy levels. Already an energetic man, Neil says Tarceva “was like giving speed to a kid with ADHD.”

Unfortunately, Tarceva’s clinical benefits are usually transient. Cancer eventually figures out how the drug works, and devises a way to get around the inhibition. Neil’s cough returned in August 2012, and he realized that the drug was wearing off after more than 13 months of productive use.

Neil was switched off of Tarceva to an intravenous chemotherapy regimen, to which he did not react well. He felt sick, his hemoglobin levels fell, and his energy level dissipated. Realizing that he didn’t want to receive chemotherapy anymore, Neil again did some research, this time into clinical trials. Some of the resources that he used, in addition to information from his doctor, included medical journals, specifically the Journal of Thoracic Oncology and The New England Journal of Medicine, a patient advocacy organization called The Lung Cancer Foundation, and GRACE, a cancer website that puts cancer research into layman’s terms.

A clinical trial is an experiment used to assess and understand the safety, usefulness, risks, and effectiveness of a potential new drug or treatment. All drugs go through scrupulous testing and trials before the FDA considers them for approval and sale to the public.

Neil brought up his discomfort on the chemotherapy regimen with his clinician at Stanford. As it turned out, she was helping conduct an upcoming clinical trial, and Stanford was one of five sites in the United States and Europe where patients would be treated. However, Neil told her that he didn’t want to be a part of the first group in the trial because the first group is always given the lowest dosage of the drug. Neil wanted something that might be able to provide a significant benefit.

Before Neil could sign up for the trial, he had to have another biopsy, to check for another mutation. The results came back positive; Neil had a T790M mutation, a type of EGFR mutation that occurs in about half of patients who have EGFR mutations. Researchers suspect that this mutation causes tumors to develop resistance to Tarceva and render it ineffective. This discovery meant he was a prime match for the clinical trial, which is investigating a drug that targets EGFR T790M.

Neil joined group 11 of the clinical trial, and received an initial dose of 1200 mg of CO-1686, which he tolerated quite well. He did some more research, and discovered that higher doses were being offered. In a somewhat unusual move, he made an appointment with the drug’s manufacturers and gave a presentation explaining why he should be upgraded to a larger dose. Neil was approved, and his dosage was bumped up to 1800 mg of CO-1686 on May 30.

Many cancer patients resist joining clinical trials because they fear the trials will be burdensome and affect their day-to-day lives, but for Neil the process has been non-intrusive and benign. “I can pretty much do what I want, after the first day,” he says.

May 30, the day Neil’s dosage of CO-1686 was increased, was effectively “day 1” of the trial all over again. He spent 12 hours at the Stanford Medical Center being tested and re-tested, to make sure the elevated dosage had no serious effects. After taking the drug, his blood was drawn every 15 minutes for the first hour, and every 30 minutes for the next hour. He also underwent an electrocardiogram (ECG) every hour, to monitor the half-life of the drug in his body. The 12 hours of testing were split up, with a two hour break in the middle. Neil, feeling much better after leaving the chemotherapy behind in favor of CO-1686, spent that time out on the bike paths.

On day 2, Neil returned to life as usual at home. He takes six capsules of the drug in the morning, and six more at night. “[The trial] doesn’t preclude me from doing anything,” Neil says. He’s able to continue his exercise, though sometimes not as strenuously as he would like. The “moment of truth”, as he describes it, comes in six weeks, when he’ll return for a CT scan to get an update on his tumor, and find out if it’s progressing, stable, or shrinking.

Neil believes his continuing battle with lung cancer is possible because of his thorough research and understanding of his options for treatment, and because he had the necessary tests done to discover his genetic mutations. For him, there was never any hesitation before joining the clinical trial. “I’ve always been a proponent of patients being a part of the process,” he says. “Without our participation, [research] would be at a stand-still. Without people participating for Tarceva [trials], I wouldn’t have had that. The patient has a responsibility to himself and every other patient to help investigate treatment options and develop the next best treatment.”

Neil understands why many people are hesitant to join clinical trials. Trials have long been equated with the patient’s last chance at survival, using an unknown, unproven drug. But that stigma has changed over the past five years. Nowadays, Neil says, clinical trials ought to be embraced as an evolutionary process, to personalize medicine in order to find the best available treatment. No one treatment will work for any patient, so clinical trials are critical to identifying treatments that work for the individual patient. By sharing your information through participation in a clinical trial and through a big-data initiative like Cancer Commons, you’re becoming part of the pathway to developing the cure for cancer.

To hear Neil tell more about his story and the importance of clinical trials and data-sharing, follow this link to our YouTube channel.

If you have a story about your experience with a clinical trial that you think would be beneficial for other patients, contact us at