Cancer One, War Less Than One

The FDA and the institutional review boards, rather than being impediments to patients gaining access to potentially life-saving drugs, must become a partner with patients. Regulatory impediments before parents must be eased.
01/20/2012 05:47 pm ET Updated Mar 21, 2012

President Nixon declared a war on cancer on Dec. 23, 1971. The war on cancer is arguably being lost on a daily basis. My daughter Alexis Agin, just two weeks shy of her fifth birthday, became yet another name etched on the proverbial wall of names who laid down their life in this war. Sure, some battles may have been won, but the war rages on, worse than ever, all these years after the initial battle cry. Unfortunately, some in the medical profession are estimating that cancer deaths may in fact double in less than 20 years. Does this sound like we are winning? During Alexis' long battle, we saw behind the curtain of Oz that is the manner in which we fight this disease. I became convinced that we are losing the war because we are not properly equipping our soldiers.

First, there is the way the government manages the care and access to treatment of those diagnosed. Imagine being sat down in a small, cramped dark room next to a nursing station. Your 27-month-old daughter sitting right across the hall. The previous night was a sleepless one on a narrow bench next to your daughter's metal hospital crib. You are told that your child has cancer and that she has six months, maybe a year to live. Sent back to your child's room with no other information, you know nothing about the world of pediatric cancer. You have no idea about the limitations on access to drugs that may help your child. The red tape, paperwork, institutional review boards (IRB), and the Federal Drug Administration. Although these are all theoretically established to protect the patient, it becomes patently obvious that they hinder your child's chances of survival in many respects. Patients given terminal diagnoses are often not allowed to try the most novel therapies under the guise that the government is trying to protect the patient from harm. Yet, these individuals have no hope, no possibility of beating the odds and are told they are going to die. Instead, these soldiers are left to lose their personal battles.

Then you have the cancer hospitals. When it became obvious that Alexis' tumor was progressing in the wrong direction, we scoured the Internet, spoke with her doctors, overturned as many rocks as we could, and ultimately decided that the most promising treatment was located at Memorial Sloane Kettering (MSK) in NYC. This was the only location where this therapy was offered. Each week we traveled from Washington, D.C. to New York City. Each week, Alexis obtained an infusion of a drug called Temsirolimus. She also received an oral chemotherapy with the infusion, which we were able to give her at home. Despite the hardships on Alexis, our jobs, our family, I often wondered why we had to travel each and every week for a drug already approved by the FDA. The reason we had to travel and could not administer this treatment regimen here in Washington, D.C. was simple; the FDA and the IRB of MSK. This trial regimen was only approved at MSK. Interestingly, Temsirolimus was being administered at Children's National Medical Center in Washington, D.C. as part of another trial. Thus, there was plenty of experience with this drug locally. Consequently, if you wanted your child to participate in this trial, you traveled on a weekly basis regardless of hardship, despite the readily available nature of the drugs. For us, we were lucky that we lived within reasonable proximity to MSK. For parents unable to do so, despite the potential promising nature of the treatment, their child was unable to receive the drugs in this combination.

It is this set of obstacles that tie parents' hands and keep this war un-winnable. It is for this reason that participation in clinical trials is declining. Several publications in the spring and summer of 2011 highlighted this fact. The red tape and impediments placed before patients hinders the ability to recruit and fill slots. It makes for a difficult system for the average parent and patient to navigate. Physicians do not have the time to educate parents on this system. With respect to Phase I clinical trials, there are rotating enrollments. Thus, timing is everything. If a slot is not open for your child at the time they are eligible, they may ultimately lose the chance to be placed on that trial.

Finally, for pediatric cancer patients, the fact that a new pediatric-specific cancer drug has not been approved in the last 20-plus years is indicative of the mentality of how we approach pediatric and childhood cancer in this country. There are little to no incentives for pharmaceutical companies to develop new pediatric drugs. The Orphan Drug Act, Public Law 97-414, as amended, may hopefully change this equation. It has yet to be seen whether this system of incentivizing drug manufacturers to release drugs targeting orphan diseases, such as the one that claimed Alexis, will have any impact. Pediatric and childhood cancer is not a profit center for the large pharmaceutical companies. Bald children do not sell or generate profits.

Regulation is necessary in order to maintain control and standards. Regulation does protect those who require protection. That being said, those who are given a death sentence should be afforded all opportunities and access to any treatment that is feasible. And if that means taking a calculated risk, then so be it.

To sum it up, I firmly believe we need to shift the paradigm and try a different approach, lest we continue to let children die. The FDA and the institutional review boards, rather than being impediments to patients gaining access to potentially life-saving drugs, must become a partner with patients. Regulatory impediments before parents must be eased. This paradigm is shifting slightly. Thanks to so many parents who are on the front lines, change has already happened, unfortunately not fast enough. As long as the government remains embroiled in partisan fighting with an eye on issues that do not surround life and death, the war on cancer will continue to claim more victims. This simply is unacceptable.