When my children were little I would tell them the world had been black and white before they were born and things were only seen in color when they arrived. For a short time they believed me, especially when I showed them black and white family photos or when we watched old movies, but they soon figured out it was another one of my crazy tales used to make a point. As our world grows, whether having children or the result of other major life changes, our perspective changes. Life was much more simple without my children, therefore it was fairly black and white. The whirlwind that followed, adjustments made to our schedules and lifestyle and looking at life through a child's perspective, added color and altered my own view of the world.
My son Matthew was diagnosed with Duchenne thirteen years ago and
Patrick's diagnosis followed. I suppose to continue a theme, this could be described as my "gray period". Like many people facing such devastating news life seemed pretty bleak for a number of months. It took time for my wife and me to pull ourselves together and see life in color again. Many things helped. Focusing on research, meeting families and being involved in the effort to change DMD for all.
Looking back to 1997 and the next several years as I began attending PPMD Annual Conferences, visiting the limited websites available at that time that had any information about DMD and reading any paper I could find about the disorder left me wondering when something, anything might become available that could in someway help our sons. Most of the research I was aware of at that time was focused on being that silver bullet to "cure" DMD. Myoblast transfers, gene transfer therapy and similar targeted therapies were the most commonly discussed strategies. Clinical trials were rarely discussed and most of these therapies seemed to make stronger mice, yet were and still are many years away from being used in human subjects. While most people's attention was on these curative procedures other scientists were looking at the pathology of DMD and considering ways to slow the progression focusing on specific pathways to stop progression or improve strength. Slowly the landscape changed as the discussion turned to building muscle, slowing fibrosis and patching leaky cells.
With the shift in research there seems to be much less emphasis on gene and stem cell therapy, yet these efforts continue while more attention is paid to small molecule and protein drugs that address specific aspects of the disorder. Several years ago I was speaking with John Porter who is a Program Director for Neuromuscular Disease at the NIH about funding research strategies for DMD. His opinion was there was no single area of research in which he was comfortable focusing and said looking at many areas made more sense offering much more potential. This broader focus at the time seemed to encompass may unrelated therapies with nothing standing out as a clear winner. Fortunately that has not been the case. As these scientists have plugged along, several promising drugs are being readied for clinical trial many will be complimentary helping to build and maintain muscle.
What seemed unlikely ten years ago is becoming a greater possibility today. As a community we now agonize over the time it will take for drug X to get to trial or that compound Y has failed in certain outcome measures. As a community and as families our perspective is changing. It is still an emotional roller-coaster, not for the faint of heart, and what we face daily. When Matthew and Patrick were diagnosed I was impatient for the potential of what research might yield. Today our impatience is with what we see as the slowness in the reality of moving research to clinical use. I see this change in hope from someday in the future to someday soon.
We still have no guarantees, but the evidence is much stronger that the therapies under development will do more than make strong mice and flashy headlines. This helps me to remain ever more optimistic and keeps me reevaluating my expectations and increasing hope.
I look forward to the day when I can consider this our black and white time and in that tomorrow the days will be in full color.