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  • Writer's pictureDr. Robert A. Nagourney, MD

For Recurrent Colon Cancer Case: "Right to Try" or "Right to Receive"?

Updated: Oct 25, 2021

Over the past five years I have been treating a 70-year-old gentleman diagnosed in 2010 with lymph node positive, Stage III colon cancer.

After surgery the patient received (adjuvant) chemotherapy but the disease recurred after 2 years.

Chemotherapy {{cta('81d5c984-b7b5-4347-86f9-281162ffa9a3','justifyright')}}again provided benefit but caused significant toxicity.

We then opted to observe the patient off active treatment for 18 months. He had slow progression of disease by CT scan and a rising CEA (tumor marker), but remained entirely asymptomatic.

Functional Profiling Testing

At the third year, the patient underwent a biopsy of an abdominal wall mass that proved to be a suture-line recurrence. This confirmed persistent colon cancer and provided adequate tissue for the EVA-PCD FUNCTIONAL PROFILE.

He was successfully treated with the drugs found active in our laboratory but toxicities again became problematic. Despite his positive response, the fatigue, hair loss and lowered blood counts caused the patient to become discouraged.

I decided to hold chemotherapy and suggested a different approach.

Based on research we had reported in 2005 at the American Association for Cancer Research, I realized that he fit the same profile that we had reported years earlier. In that study we compared the antibody Cetuximab directed against the Epidermal Growth Factor Receptor (EGFr) with Erlotinib, a small molecule that inhibits this same target.

Both compounds, one an antibody and one a tyrosine kinase inhibitor, block the same pathway. What we had observed in the laboratory was that these two classes of drugs were not directly cross resistant and that in some instances they were synergistic, meaning that they could provide real benefit when they were combined together.

I constructed a treatment regimen consisting of an every other week infusion of Cetuximab with oral Erlotinib daily.

By the time we began this new treatment, the patient's CT scan had worsened and his CEA had jumped to 394 (normal 0-5). With treatment he experienced some skin rash and gastrointestinal symptoms so we adjusted the Erlotinib dose and continued. After a few cycles there was a dramatic improvement in the CEA falling from 394 to 25.

More striking, the patient's PET-CT confirmed an objective response with measurable improvement in all areas of disease.

The odd wrinkle in this story is that our carefully constructed treatment that provided such good response did not fall within FDA approved use of Erlotinib.

Erlotinib is simply not approved for colon cancer.

While we can administer Cetuximab (FDA approved for colon cancer), this drug alone did not appear potent enough to provide response. Erlotinib was essential for this treatment to work.

When the patient's prescription for Erlotinib was denied by his insurance company, I was able to obtain a small supply of Erlotinib. I reasoned that it would be appropriate to gift the drug for one or two courses to prove the efficacy. Now several months later with a documented excellent response, we have received yet another insurance denial.

In recent years there has been a great deal of discussion regarding a patients’ “right to try.” Pursuing this line of reasoning, patients with life-threatening diseases have petitioned the FDA for the early release of experimental agents. While there are arguments for and against, these patients are in fact requesting drugs that have not met the FDA’s standard of safety and efficacy.

The current patient's story is quite different. The drug Erlotinib is FDA approved for lung and pancreatic cancer. It has been established to be safe and effective. It can be obtained from any pharmacy and if you have one of the listed diagnoses, it is fully reimbursed.

This patient’s case reflects a deeper problem with the contemporary process of drug approval.

Today, FDA approval requires large and extremely expensive clinical trials that establish the safety and efficacy of a drug in each disease.

As Erlotinib’s safety has been well established, the missing link was a Phase III trial in colon cancer, but it is unlikely that such a trial will ever be conducted. The drug is old with limited patent life remaining. More to the point, it is not likely that a colon trial would be positive in an “unselected” population of patients.

Put simply, it may not be a good investment for the pharmaceutical company to conduct the trial. We had the luxury of gifting the patient enough drug to prove efficacy. With proof in hand we felt confident that insurance coverage would be granted. To our dismay, it has not.

This can be viewed as a “right to receive”.

Under this scenario, patients may find it necessary to pay for one or two cycles of a novel treatment if it is off-label (not approved by the FDA for that specific disease).

After all, insurers and governmental agencies cannot support every novel idea to satisfy someone’s curiosity. However, with proof in hand, objective response established and clinical benefit confirmed, shouldn’t insurers cover treatments that work?

Our laboratory has the unique capacity to select effective cancer therapies. We have the ability to identify concepts that warrant evaluation. This patient's objective response is proof positive that he is benefitting from this treatment.

Shouldn’t patients be able to receive coverage for effective therapies once the efficacy has been proven?

I welcome your thoughts and comments.


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