How we choose a person as a hero is often related to individual interpretation or current societal trends. Many people today claim athletes or entertainers as heroes, while others choose political figures. In general, I don't agree that exploiting a talent for the entertainment of others or seeking public office and financial gain meets that standard. The days when we looked up to people who overcame great challenges or adversity seems to be gone, with a few rare exceptions.
A lot of families who have children affected by serious chronic illness, such as Duchenne, think of research scientists and clinicians involved in helping their loved ones as heroes. I am more inclined to agree with their definition, as researchers and doctors are truly committed to helping others often working long hours without thought of gaining celebrity status, major financial compensation, or long term name recognition. Still, there is a group of people who I believe earn that title because of even more selfless efforts. These individuals may not be acknowledged by others and their efforts may never be recognized as meeting the standard for any special status, yet in my mind they are true heroes. These are people who participate in clinical trials in spite of the odds that their participation will lead to the development of drugs or therapies for people with conditions they share. There are far more drugs that fail in clinical trials then are approved, yet without volunteers participating, no drug would be approved.
This is a new phenomena in the Duchenne community, as clinical trials are not commonplace. Families who have been affected by Duchenne for any length of time are more accustomed reading about progress in basic science and researchers making stronger mice, not men. I am impressed with those who willingly take part in these trials of their own free will knowing full well there is no guarantee of success. I am grateful for the young boys whose parents give their consent and they too are heroes. Yet, as these boys become young men, where they are more capable of understanding the implications of their involvement I am more impressed and humbled by their actions. One of these young men is my son Patrick.
Patrick recently participated in a pharmacokinetics (PK) trial for an antisense oligonucleotide (AON) drug designed to skip exon 51 helping a subset of our sons affected by Duchenne with a certain deletion mutations. My wife Alice and I spent time explaining the possibilities and risks to Patrick before his participation. A PK trial is simply a safety trial, yet as exon skipping is new, many unknowns exist, including possible serious side effects. These trials are done to provide drug companies and regulators safety information prior to starting a wider clinical trial. Plans for a Phase I Trial will begin with ambulatory boys, as there are better outcome measures to gauge efficacy. Patrick is no longer able to walk, so unless there is an extension trial for non-ambulatory young men, it is possible he may be excluded from future participation. The time it takes for completing a trial is against him. Patrick is also 16. The process could take several years and no one can predict how well he will be able to tolerate travel and whether his health will allow him to participate may change. Alice and I both understood this and explained these facts to Patrick. His response was that he still wanted to participate.
Prior to participation, Patrick had to read and understand an "assent form" which explained the process and possible risks. This was in addition to the "consent form" Alice and I read and signed. Because Alice and I explained what would happen, there were no surprises. This was his opportunity to say "no thank you," yet without hesitation Patrick read each page and signed the forms as required.
Not all parents whose sons have Duchenne want them to participate in clinical trials, and not all boys and young men are willing to take part. Those who understand and accept the risks do so for many reasons, yet they are all helping one another in this community. To be 16 years old with Duchenne is hard enough and anyone would understand if the risks of a clinical trial are too great to consider participation.
Maybe they will never wear a cape or a shirt with a big S on their chest, but those who say "yes" are my heroes. I am proud to say my son is one of those heroes willing to help.