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.

Brian Denger

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Comment by Kathryn R. Wagner on December 30, 2010 at 5:58pm

Brian, I'm just reading your piece on the heroes of clinical trials.  Thank you so much for writing it.  The DMD absolutely needs these young heroes.  I think it is especially difficult for the adolescents and young men who have achieved just the right "balance" and may be concerned about it tipping.  It takes courage.

We are looking forward to one or more of your sons participating in the Revatio for Heart disease in Duchenne Muscular Dystrophy (REVERSE-DMD) trial at Kennedy Krieger.  I"ll get the capes and shirts with the big S ready for them! 

Comment by RAKTIM SINGH on November 11, 2010 at 9:25am
Hat's off to Patrick and family. A big THANKS for all your efforts which will surely make a difference in our lives.

A BIG THANK YOU.
Comment by cheryl cliff on November 10, 2010 at 12:12pm
Ditto!
Comment by jenn on November 10, 2010 at 9:47am
brian i agree, my son austin was in the exon skipping trial in columbus over the summer. it was not fun at all, but he was so brave. at age11 he coped with things i would have thought twice about doing at age 30. like patrick he was aware that this treatment may not arrive in time to help him, but he did it anyway. i truely hope the work and sacrifice of these boys is not in vain

Staff
Comment by Pat Furlong on November 10, 2010 at 12:06am
Brian, there is no doubt in my mind that our sons are the real heroes. Thanks to Patrick as his participation is a great gift to all of us.
Comment by Lori Ware on November 9, 2010 at 11:00pm
Thanks to Patrick...and you and Alice for raising such a fine young man!
Comment by tom evans on November 9, 2010 at 10:37pm
I'm so glad you brought this up.My son Benjamin was in the PTC trial, and went thru 4 biopsies, countless blood draws, flights to and from Atlanta on very small seats, some hotel rooms that considered a grab bar in the bathroom handicap accessible, not to mention the emotional stress that went along with this trial.All the while, he complained very little because he knew the importance of this trial.He is incredible.Once a week he rides therapeutic horses here in Montgomery, Al.I often wonder if any of our star atheletes were told that we were going to bind their feet so their legs couldn't work, put large weights on their arms so they couldn't raise them much above their elbows, and attach weights around their back pulling them forward.Then, we wanted them to let us put them on the back of a horse with a 110lb volunteer college age woman on each side to catch them if they fall, would they have the courage to do this. Essentialy, Benjamin's condition gives him these challenges when he rides, but he not only does it every week, he looks forward to it.Our kids are not recognized by the absolute blessing that they are to this world. I challenge today's leaders to spend a day a month with our kids and others with similar circumstances and tell me they're not stronger than our greatest atheletes. Tom...proud father of Benjamin every day of my life.
Comment by Ian Anthony Griffiths on November 9, 2010 at 10:01pm
Hat's off to Patrick, Brian. Ian :)
Comment by Perlita & Gordy Hains on November 9, 2010 at 9:40pm
I am honored to call you friend, and Patrick is beyond amazing - Tell him we are all so proud
Comment by Jacobs Mommom on November 9, 2010 at 7:49pm
Very well put Brian! You need to include yourself and Alice in that list too!

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