The Heart of Compassionate Medicine

The British High Court ordered the immediate removal of eleven-month-old Charlie Gard from mechanical ventilation this past July. At the time of his death, he could not hear or open his eyes without assistance, could not breathe on his own, had heart and kidney failure, and suffered chronic seizures.

Charlie was diagnosed with Mitochondrial DNA Depletion Syndrome (MDDS) when he was two months old. MDDS is an extremely rare genetic disorder caused by a mutation in the gene that codes for the RRM2B protein. A dysfunctional form of this protein inevitably leads to muscle weakness, organ failure, and death.

The United States National Library of Medicine has only 15 other recorded cases of MDDS caused by the RRM2B mutation, none of which have been successfully treated. Earlier last year, Dr. Michio Hirano, the head neurologist at the Division of Neuromuscular Disorders at Columbia University Medical Center, set out to change that.

Hirano reached out to the medical team at Great Ormond Street Hospital in London (GOSH), where Charlie was being treated, to discuss his experimental nucleoside treatment. He believed it held a “theoretical possibility” of improving Charlie’s condition. However, an MRI revealed severe infantile epileptic encephalopathy—brain damage so severe that Hirano’s treatment, which would deal mainly with muscle atrophy, would prove futile.

As a result, GOSH began discussing the termination of Charlie’s life support and placing him in palliative care because there was such a limited chance that Hirano’s treatment would improve his quality of life. His doctors believed they were acting in Charlie’s best interest. His parents, however, felt it was their right to provide the treatment for their son.

In the U.K., disagreements between hospitals and parents are taken to the courts, where the court is expected to objectively determine whether or not continuing (or receiving) a treatment is in the child’s best interest. Both the English High Court and the European Court of Human Rights backed GOSH’s stance, and Charlie was taken off mechanical ventilation a week before his first birthday.

While in the U.S., it is ultimately up to the parents of children to decide whether or not they want to seek special permission to test these premature treatments; in the U.K., this decision is left up to the hospitals and courts. In fact, immediately following the European Court of Human Right’s decision, Charlie’s life support was set to be taken away on a date and time set by the hospital, not by his parents.

The long battle between England’s legal system and the hospital against Charlie’s parents raises questions concerning the rights and responsibilities of parents in determining medical treatment for their terminally ill children and the extent to which the government should get involved in these choices.

The type of medical treatment Charlie would have received is also known as compassionate medicine, which is the administration of drugs or treatments not approved by the FDA. It is used as a last resort on patients who have essentially run out of options. But hospitals have reservations about resorting to compassionate medicine because of the extreme risks it runs.

This makes the balance of power between the parents and the hospital an especially difficult one to strike; while a system which gives power to the parents often pressures doctors to perform treatments that they feel are a misdirected use of resources, a system like the one that Charlie’s parents claim condemned their son to death takes away the freedom of a patient to choose his or her medical options. The law essentially makes ethical decisions based on medical conjecture.

In an attempt to use public support to sway his doctors, Charlie’s parents raised over $2,000,000 on their GoFundMe online fundraiser, received support from President Trump and Pope Francis, and obtained an invitation from the New York Presbyterian Hospital and Columbia University Irving Medical Center to evaluate Charlie and carry out the experimental treatment, either in New York or in London.

Despite this popular support, the British government was able to block Charlie from receiving treatment in New York. Though Charlie’s condition at the time made it more than likely that nucleosome transfer wouldn’t make a difference in his life, his parents should have had the right to accept treatments even with a limited chance of success, given a few provisions. One is that parents should understand and accept the fact that compassionate medicine is nearly synonymous with risk. Doctors must take on the task of informing them about all exhaustible options so that the parents can make rational decisions.

Following that, the government should be kept as a third party with limited involvement in order to allow it to focus on streamlining the official drug approval process while also bypassing the socialized medicine argument. Compassionate medicine should not find itself dependent on public funds.

Unfortunately, all of the support Charlie received online also reveals how compassionate medicine’s projected legacy has been pushed and pulled to fit political agendas. Republicans such as House Speaker Paul Ryan are attempting to use Charlie’s stories in order to dismantle the Obama administration’s healthcare system. Ryan recently tweeted, “Health care should be between patients and doctors. Government has no place in the life or death business.” Vice President Mike Pence attributed his death to “all the talk on the left.” Yet note that ultimately, it wasn’t the British government that condemned Charlie to die, but the combined efforts and professional advice of the doctors and the hospitals that treated him.

Using him as a poster boy against government involvement in healthcare purposefully discounts the nuances of his story. The crux of Charlie’s story and the heart of medicine as a whole does not have to do with the morals of public healthcare. It has to do with parents and the law being able to reach a common ground and the definition of a child’s best interest.