• Dr. Robert A. Nagourney, MD


Many cancer patients confront hurdles in getting life-saving drugs paid for by their insurers. One such experience has been particularly troubling.

This 64-year-old gentleman with very long-standing hairy cell leukemia had received virtually every known treatment for this disease, from the standard 2CDA (2-chloro-deoxyadenosine), to BRAF-targeted agents, to chemotherapy, combinations, monoclonal antibodies, and antibody-drug conjugates. In fact, the patient had exhausted every known treatment.

Using our laboratory platform (EVA/PCD) we had selected a drug combination 5 years earlier that provided a durable remission with a single cycle that lasted 4 years, the longest in his nearly 20-year history of the disease. When he then progressed we administered the very new antibody-drug conjugate moxetumomab pasudotox (Lumoxiti) which provided a brief response but not without toxicity.

When he again progressed we applied our EVA/PCD platform that now identified the novel BH3 mimetic Venetoclax combined with 2CDA to be highly effective. Venetoclax, approved for the treatment of chronic lymphocytic leukemia and used in acute myelogenous leukemia that arises in myelodysplasia, is not approved in hairy cell leukemia. Nonetheless, the drug was highly active in this patient’s cells.

Mechanistically, the drug turns off the survival signal known as BCL2 and can be so effective that the doses must be adjusted slowly upward to avoid tumor lysis syndrome, whereby the cancers literally explode, leaving the body awash in toxic byproducts

I could not procure Venetoclax for the patient and he was rapidly deteriorating. Fortunately, a small quantity of the drug had been donated and I used the assay results to combine a low dose of 2-CDA with escalating doses of the Venetoclax, based on the clear synergy of the two agents together. As the patient had failed 2CDA many times before, we knew it was only the combination that would work.

Within a short time his hemoglobin improved, then his white count, and then his platelet count. With obvious evidence of response, I wrote the patient a prescription for Venetoclax, requesting that his insurance now pays for this manifestly effective drug.

The insurance company reviewer called to say that they would not pay for the drug. I pointed out that the patient was responding beautifully and that it would be an error to stop the medication. They did not care. They said the drug was not approved for this indication and that he should take a different drug, Ibrutinib; a drug that he was clearly resistant to in my lab analysis

I have long experience in hairy cell leukemia, having originally discovered the use of 2-CDA as the treatment of choice for hairy cell, during my fellowship at the Scripp’s Institute in the 1980’s. I know this disease intimately and am also very familiar with Ibrutinib, a terrific drug for many of my patients but not for this one!

Despite their intransigence, the patient did complete the course of therapy as described using the gifted drug and I am pleased to say he has achieved an unmaintained remission.

We are at a crossroads in medicine. Treatments that work are not reimbursed, while treatments that do not work are reimbursed handsomely.

It is time to re-examine how we select treatments and how we are paying for them or we will all go bankrupt failing to meet our patient’s needs.