A mother noticed that her six year-old son’s eyelids were not closing normally and when he walked, he was slightly off balance. A few days later, his parents’ worst nightmare became a reality; they were told that their son has a brain tumor. His diagnosis did not come easy. Various pathologists could not give a definitive answer as to the type of cancer much less its Grade (I-IV). The eventual final diagnosis came from one of the world’s leading authorities on brain tumors. It was an Anaplastic Astrocytoma (Grade III), the most common type of brain tumor in children between the ages of 5 and 9 years old. The child took a series of radiation therapies and chemotherapy agents. He appeared to respond well to some treatments, but it was short-lived.
Finally, after searching and searching, a possible cure was located. There was a drug that had shown early evidence in adult trials to be effective. The drug had appeared to be effective at stopping the activity of a gene that was responsible for driving the growth of certain tumors, including this child’s tumor. There was one major problem. While the drug was the final possibility, it was withheld from him. There were no remaining spaces available for him in the phase I pediatric trial and the pharmaceutical company would not allow the child access to their drug under the FDA’s Expanded Access Compassionate Use program that is available for patients desperately seeking a last chance for survival.
According to the FDA, expanded access – sometimes called “compassionate use” – is the use of an investigational drug outside of a clinical trial to treat a patient with a serious or immediately life-threatening disease or condition who has no comparable or satisfactory alternative treatment options. FDA regulations allow access to investigational drugs for treatment purposes on a case-by-case basis for an patient with similar treatment needs who otherwise do not qualify to participate in a clinical trial. The investigational drug may be effective in the treatment of the condition, or it may not. They also may have unexpected serious side effects – a risk the child’s parents were willing to take.
So, is there a “bad guy” in this child’s story, someone wearing a black hat, that we can point fingers at and add a heap of blame? Not really. The pharmaceutical company stipulated their policy of not permitting expanded access to drugs undergoing clinical trials for compassionate use, regardless of the circumstances of any patient seeking it. Likewise, the FDA does not permit expanded access to Phase I trials. The business and medical reasons for denying the child access to the investigational drug are deemed as rational and prudent. The drug is not yet proven to be safe and non-toxic in children. Its efficacy in curing targeted cancers is still be determined, even if it passes through Phase I testing. And yet, this logic still begs the question: what does anyone, including this child, have to lose? Per this child’s father, “I suppose that I understand why pharmaceutical companies and the FDA will not allow my son access to the trials for statistical integrity, risk, and even business development reasons. Yet, I am here offering my son as our last hope, but also as a test-subject opportunity for this pharmaceutical company to hopefully develop a treatment for this disease with no current cure whatsoever. If my son cannot be saved, maybe he can save other kids in the future. Is that too much to ask?”
Were there any other possibilities for this child, specifically for his type of cancer? The answer is no and the reason is that few, if any treatments reach clinical trials for children’s cancer. Cures for childhood, adolescent, and young adult cancers are basically side bets. They are not the driving force behind drugs such as this one.
Frustration is growing for parents who witness a culture where, generally, promising discoveries not only reach dead ends, but, typically never get consideration for advanced studies. The current system (honors, grants, and tenure), of academia, the NIH, and most foundations, continues to reward basic discovery research, not the ensuing requirements needed to shuttle potential breakthroughs into new diagnostics and drugs.
“Things need to change at both the NIH and foundations that fund research. The funding of just research, while appearing as major steps forward, is not sufficiently helping the advancement of cures for kids like my son. I appreciate the work being accomplished, but when are we going to see some real returns for all the hundreds of millions of dollars spent?” the young boy’s father goes on to ask, “Why do parents have to go begging researchers to help their kids, rather than physicians armed and ready with cures?”
Author: Beth Anne Baber
The Patient Choice Act – Providing Choice Instead of Death by Jonathan Agin A story about proposed new legislation that will enable a parent to choose a new treatment not yet approved for use.
Let him try the drug, he is probably going to die anyway, this is his only chance to live.