One of two physicians honored at the recent NephCure “Count Down to a Cure” event, Howard Trachtman, M.D., chief of pediatric nephrology at Long Island Jewish Medical Center, offers these insights into the life of a physician/researcher seeking to beat Nephrotic Syndrome and FSGS.

"As you can hear, much as I would have liked it, you are not honoring me for how I play Chopin. Sounds right to me. The question then is why honor me for doing my day job? With all the muttering under my breath during those calls from the hospital during Law and Order reruns, despite my outrage at administrative interference, I would still show up at my office every morning even if they did not pay me. Honoring me for being a pediatric nephrologist is like honoring me for rooting for the Philadelphia Eagles – I can’t help myself. So the question on the table is why me and why now?

I promise you I will be done in about 8 minutes if you are looking at your collective watches. So let’s start and see if we can come up with an answer. You know the kind of work I do. I see your children in clinic and sometimes in the hospital for problems like protein in his or her urine, high blood pressure, swelling, or maybe because your child’s kidneys are not working. You must have thought I was crazy and wondered what kind of problems are these? Children like yours never show up on ER or House. No one you know has these diseases. But trust me – you all know more than the residents when they start an elective in nephrology. Do I have to explain albumin and edema, ACEs and ARBs, podocin and nephrin, cystatin C to a group of parents and friends who have been through what you have experienced? Perhaps the first thing that I may have done is to speak to you in my rushed and impatient way about what was the matter with your child. I hope, I made sense. But, I have been accused of talking too fast and using weird words, gestalt, for example. And, how else can I tell you about the exciting developments in the molecular biology of the podocyte, genetic discoveries about the causes of the nephrotic syndrome and FSGS, insights into the regulation of kidney fibrosis or scarring? As we sit together in those scary examining rooms, I find myself saying things that no nephrologists could have imagined 20 years ago. And remarkably, as we talk, despite the anxiety and fear swirling in your hearts, over time you become my partners as we try to figure out what’s wrong with your child’s kidneys. But everyone does this, so that cannot explain me standing here tonight.

The next thing I do is suggest tests and prescribe medications for your child. Some of this stuff – biopsies, solumedrol, dialysis -- sounds downright awful even to me and I can imagine what they conjure up for you. But here it starts getting tricky. No doubt you may have thought that I had unbelievable tricks up my sleeve and that I would spin up some magic brew to make your child normal again. But the horrible truth is that I am limited in what I have to offer. As smart as we are about molecular biology and genetics, there is a long bridge we have to cross to get to a better place where we can treat your child. There are lots of boys in lots of back rooms trying to come up with the next great new thing. For now, I slug it out with the old workhorses – prednisone, cyclosporine, prograf, lasix, vasotec, cozaar. We’re hard wired to find cause and effect and your natural inclination is think I am brilliant when they actually work and to blame me when they do fail or cause your child to morph into a prednisone intoxicated monster who won’t go to bed and beats up the kids in nursery school. The truth is I deserve no credit either way. You and your child are doing the heavy lifting and I deserve no honor for a higher health rating ala Donovan McNabb. There are many wonderful doctors doing great thing with children with renal disease throughout New York. So this cannot account for my name on the program.

Another big part of my job is to shove protocols at people in my office and say, “Get this through the IRB.” When this is done, I place complicated documents called informed consent forms in front of you and ask for permission to enroll your child in research projects. Because I know that a lot of the treatments I recommend are not the greatest, I feel this intense pressure to come up with a better way of doing things. This is the drive to build a better mouse trap, to find a “cure.” More about that in a moment.

As you know, I have been fortunate enough to be involved in the NIH funded FSGS trial to compare cyclosporine with a combination of cellcept and oral pulses of dexamethasone for patients with steroid resistant FSGS. This has been a monumental effort. To date, this study has not yet generated the momentum that most of us anticipated. Maybe you honor me tonight because you think I am a good person to nudge this rock forward and get it rolling down the hill. One thing for sure is that this project will not happen unless we all raise our voices in unison and say that this clinical trial is the best thing for patients with FSGS. I know it is. The movies make us think that a clinical research project will get THE cure we want and generate honors and dinners like this. But I want you to know better. Medicine does not progress in cinematic leaps and bounds most of the time. Instead, it plods along making things a little bit better one small step at a time. After a couple of years, hopefully you can look back and say, “Wow! We have really accomplished something.”

The model for clinical research that I would have you imagine as we ramp up the NIH-funded FSGS trial is to consider leukemia or ALL in children. Prior to 1948, ALL was invariably a fatal disease. When investigators at Dana Faber in Boston gave children a new drug called methotrexate some of the patients survived. Not all, but the change was in the order of magnitude better than anything that had been seen before. Over the last 50 years, pediatric oncologists have slowly but surely improved this 40% to 50% success rate to the point where now over 95% of children with acute leukemia are said to be cured. This is the challenge for us in nephrology and we should all live so long to reach that day for FSGS. The odds are the advances will be very subtle. Nephrologists and their patients will have to work together as we are doing in the FSGS trial. Everyone and no one deserves to be honored for this effort, not just me.

So after all this, maybe you’re thinking you made a mistake with me. I hope not. Let me suggest that the answer will emerge if we put the pieces back together again. Think of it this way. Picture in your minds eye those books with pages full of color. Someone points to one frame and says, “Look at the page, concentrate, and you’ll see a picture of Terrell Owens strutting around the end zone. If you’re lucky, suddenly a picture of number 81 emerges and you say, “Cool.” What I suggest is that you look at the chaos that is my job -- talking to you in the clinic, prescribing medications, monitoring your child’s status, inviting you to enroll in a research trial. At first glance, the picture looks like a mess. But if you look very closely, you will eventually see an organic body composed of many wonderful people working together to treat children with kidney disease and FSGS. I am never alone at my job. The door is open, the phone is always ringing, people are bumping asses and tripping over one another. The best thing I have done is to bring together a bunch of wonderful people to help me help you. There are people at Table X and Y that I have worked with for many years, others who are fairly new to the group. I have older colleagues who have taught me how to be a good doctor even when all I really wanted to do was write another research paper. There are nurses who make me talk slowly and nicely even if there are 4 other people I need to get back to before 5 o’clock. They wait out all my rambling ideas and translate them into something that works for you and your children. There are student volunteers who bring energy and good cheer to the office. There are collaborators who make me see things about clinical trial design or the potential use of a novel medicine that never occurred to me. There are family members who put up with all of my nasty brutish behavior when I come home at night and all I want to do is to eat an administrator for dinner. There are all of you – patients, moms dads, husbands, wives, friends, -- who give purpose and meaning to my life and those people buzzing around my office. And there is NephCure bringing it all together.

So who is at the center of the picture? Imagine another picture. You are familiar with the series of pictures from the Hubble telescope. They start with a photograph of outer space, perhaps the Andromeda galaxy. Then it is magnified and you see the solar system. You magnify it more and you see the earth. Magnify more and you see New York. Magnify again, and you see Schneider Children’s Hospital. One more step and you see a cluttered office on the third floor where I live and work. Where do you finally focus? In the last picture, the most important thing is not the place, but the people who come in and out of SCH365. The focus is not on me but the amazing family of people all around me and the heavy traffic in intelligence, grace, and compassion. I accept this honor for being smart enough to be part of a family of people including each and every one of you committed to negotiating treatments and options, looking back with regret when things don’t work out, celebrating shared victories. Together we come into focus as a family, poised to push forward and develop better ways to make kidney disease and FSGS something that people can at least live with and hopefully overcome at best. Yes, maybe even find a cure.

So thank you very much for this extraordinary honor. I would close with a wish and I am sure that every parent and patient in this room will understand – I hope that you get to the day when you never have to come see me in Mod 2 and all we have to communicate about is the Eagles winning the Super Bowl and our grandchildren."