The FDA and the Tumor

This article is from the archive of The New York Sun before the launch of its new website in 2022. The Sun has neither altered nor updated such articles but will seek to correct any errors, mis-categorizations or other problems introduced during transfer.

The New York Sun

Every morning I begin craving my cocktail — a drug cocktail. My life likely depends on it. I suspect Senator Kennedy will develop a similar appreciation for “drug cocktails” since his recent diagnosis with the same form of incurable brain cancer.

It is probably no coincidence that we both chose the Tisch Brain Tumor Center at Duke University for treatment. Duke’s approach defines inspirational, and you need inspiration when conventional medicine tells you there are no options other than to die in 18 months.

Standing in the way of an effective drug cocktail for me and for many other victims of primary brain cancer is the United States Food and Drug Administration.

I was diagnosed with a primary brain tumor in December of 2005, four months after I turned 45. Primary brain tumors originate in the brain and the prognosis usually is death.

My tumor is a glioblastoma multiforme, which is the exact same diagnosis as Senator Kennedy’s. It is the fastest moving and most aggressive kind. Most people with GBMs die in the first six months; those who do not are usually dead by the end of the first year. Only 8% live past two years and fewer than 2% live past 10 years.

Primary brain tumors are sheltered from normal forms of chemotherapy by a biological structure called the blood-brain barrier, which evolved to protect the brain against toxins. Although there are some specific chemotherapies that have been developed to cross that barrier.

The truly insidious thing about primary brain tumors is that the most effective treatments — surgery, chemotherapy, and precision radiation — don’t really work. Once the visible tumor is removed by one or more of those therapies, thousands of microscopic particles remain.

Dr. Peter Dempsey at the Lahey Clinic in Boston, where I receive treatments based on protocols laid out by my doctors at Duke, recently remarked that isolating these cancerous elements for treatment is a little like trying to remove only the vanilla pudding from a mixture of vanilla and chocolate.

Or as Dr. Eric Wong at Beth Israel said to me, “Eliminating a primary brain tumor would be like sprinkling sand on a blanket and then removing all the grains one at a time.”

So it would seem that a systemic solution would be the better bet for a cure. The most common form of such a treatment is a “drug cocktail.” Systemic treatments drove HIV/AIDS from a death sentence to a manageable disease. But the FDA doesn’t approve such therapies for patients with my disease.

In search of a systemic treatment, I have traveled many times between Boston and Durham, where the staff have always worked with multi-agent trials and have pioneered a multi-agent treatment, currently considered the most successful for my particular tumor. Duke is famous for rotating therapies in the first year because they acknowledge the aggressive force of the primary tumors that adapt and overcome standard single-method therapies.

This treatment combines a form of chemotherapy with Avastin, which limits the blood supply to tumor cells. Avastin, though not without risks, is not toxic like chemotherapies. Some people believe it enhances chemotherapies. Using Avastin, I have exceeded the standard of survival, having now lived 30 months since my diagnosis. But my cancer has not been without recurrence.

It returned in October of last year and, following surgery, in January of this year. The new tumor is in an inoperable region of my brain, so I need a stronger drug cocktail, a better systemic treatment. But I can’t get past our prohibitionist FDA, specifically their jurisdiction over clinical trials, the hopeful testing grounds of new therapies.

The FDA monitors so-called clinical trials to prove the usefulness of certain therapies against existing or standard treatments. To get that proof and achieve statistical significance requires thousands of participants, a tough challenge in the case of primary brain tumors, an illness that affects about 6,000 Americans a year.

Over the last 30 months I have kept up with clinical trials and treatments for people living with my disease. Hopeful signs are few. One important clinical trial was just completed by Schering-Plough, makers of Temozolomide, or Temodar, the first chemotherapy agent designed to pass the blood-brain barrier. Since it was the first form of chemo designed for brain tumors, the trial has been unusually humane, conducted without placebos or double-blind studies needed for full statistical evaluation.

The trial compared the use of Temodar to the prior standard treatments — surgery or surgery and radiation — since there is no previously existing standard chemotherapy. It seems to have achieved blessed FDA approval despite the lack of statistical specification.

A main part of the problem with clinical trials for a drug cocktail is that most require patients to abandon already proven treatments in order to get clean statistics. FDA-approved clinical trials are known as single-agent trials because the medical community wants to know for sure that a single drug works.

It follows that the FDA hates drug cocktails precisely because they present statistical chaos. Such treatments make it hard to isolate cures. But to people running out of time, this seems unreasonable, sadistic even.

Besides its methodological puritanism, the FDA also imposes rules that unnecessarily limit the use of promising experimental drugs or treatments. Recently, I was invited to join a clinical trial currently being run by an Israeli company, NovoCure, Ltd., for an electronic device that destabilizes primary brain tumors — a series of electrodes are attached to the skull and powered by a battery carried by the patient.

The electrodes have been proven to disrupt the tumor cells’ rapid growth. The beauty of the NovoCure device is that it can be used as an adjuvant; that is, to complement other treatments such as chemotherapy. Like drug cocktails, it promises systemic therapy.

NovoCure will be giving the new treatment to a group starting at the end of this month, but I might not get into the group because it’s supposed to be a double-blind study. That means in order for me to receive the new drug cocktail, I would have to forego my standard treatments for a month, which would be suicidal.

The average life expectancy of a patient with a recurrent inoperable brain tumor is six months, which is the exact time I have managed to stay alive since the January diagnosis. So to pursue one of my only hopeful options, I would have to gamble with my life because NovoCure wants FDA approval. Health insurance companies fund only FDA-approved treatments.

While FDA rules are great for statisticians, they threaten the lives of the very patients they are intended to help. I understand that we need to be patient for medical science to progress, but the very same medical science tells me there are no single-agent cures on the horizon.

The FDA’s rules for clinical trials don’t engender hope for cancer patients. Instead, they provoke desperation.

Only Congressional action can correct this government-inflicted threat to the lives of its people. A bill recently sponsored by Senator Brownback and Rep. Diane Watson would provide hope to cancer patients everywhere, if approved, and it might supply us with the life-extending drug cocktails now being denied to us by the statistically obsessed FDA.

It is not unreasonable to favor using a series of promising systemic agents and combining them with existing therapies to find a cure.

Mr. Dougherty, who has been the chief executive officer of two publishing companies, lives in the Boston area with his wife. They have three grown sons.

Author’s Note: The Web site that will champion the use of drug cocktails is: global-cures.org/promising_therapies. The most comprehensive book written about drug cocktails is the “Surviving ‘Terminal’ Cancer: Clinical Trials, Drug Cocktails, & Other Treatments Your Oncologist Won’t Tell You About.” It was written by Ben Williams who was diagnosed with a GBM and had most of the tumor surgically removed when he set out to use his research skills to come up with a “drug cocktail” that appears to have worked since he has been tumor free for 12 years.


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