A mother noticed that her six year-old son’s eyelids were not closing 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 had undergone a series of radiation and chemotherapy agents. He appeared to respond well to some treatments, but his tumor’s suppression was always short-lived, with the Anaplastic Astrocytoma always returning back to rapid growth.
Finally, a possible cure may be at hand –a drug that shown early evidence in adult trials to be effective at stopping the activity of a gene responsible for driving the growth of certain tumors including this child’s tumor. There is only one problem. The child was denied access to the drug. There are no remaining spaces available for him in 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 for patients desperately seeking a last chance for survival. While the drug was the final possibility, the drug is being withheld from him.
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 individual patient, or a group of patients 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 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?”
Are 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 science culture where promising discoveries generally not only reach dead ends, but typically never get attempted for advancement. Throughout academia, the NIH, and most foundations, the system of honors, grants and tenure, continues to rewards basic discovery research, not the ensuing requirements needed to shuttle potential breakthroughs into (new diagnostics or) drugs. The child’s father comments, “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? Why do parents still have to go begging for researchers to help their kids rather than physicians with cures?”
Join us 11/27 @ 8pm ET for #treatdiarieschat and meet TNCI.org The Nicholas Conor Institute – a 501(c)(3) not-for-profit medical research organization that concentrates exclusively on cancers common to children, adolescents and young adults. We will be tweeting with Founder and CEO – Beth Anne Baber.