Miracles We Have Seen

by Harley Rotbart

  When Brandon was eighteen months of age, we performed another MRI confirming the tumor was still there. At twenty-three months of age, in August 2003, Brandon developed fever and some bone pain, so we again repeated the MRI to make sure the symptoms weren’t due to tumor progression, but the tumor was still stable. At that point, Brandon’s parents began exploring the options for surgery. They contacted the excellent neurosurgery group at a major West Coast university medical center and arranged for Brandon to be assessed there for possible biopsy and surgery. Their first visit was in late August 2003. An MRI at that time again revealed a stable tumor. Brandon’s parents arranged for him to return to the West Coast university medical center in November 2003 for biopsy and/or surgery.

  When they arrived in November, three months after the most recent unchanged MRI, and two years after the tumor was first diagnosed, Brandon had another MRI to help guide the surgeons for the procedure scheduled for the next day. The results stunned Brandon’s family and their doctors on the West Coast, who had reviewed all the previous scans and were preparing for surgery: the tumor was entirely gone, almost as if it disappeared mid-air on the flight from Atlanta.

  Brandon’s parents considered this a miracle and, certainly, the timing of the tumor’s disappearance was remarkable and very fortuitous—just in the nick of time to spare the child the substantial risks of this type of surgery. But for those of us who had cared for Brandon in Atlanta, the miracles were just starting.

  Brandon’s mom, Kristin, was a corporate attorney for a big law firm in town. She had spent the previous eighteen months in and out of our children’s oncology clinic waiting room, seeing all the kids who, like Brandon, had cancer. All types of cancer, few if any of which would “disappear” on their own. She met parents struggling with dire prognoses for their kids. Many of the kids playing with Brandon in the waiting area already had the severe telltale signs of chemotherapy or radiation or both. Others had undergone surgery or were about to.

  Having a child escape the potential ravages of cancer and its treatment is more than enough to make any parent overjoyed and grateful. But, whereas most parents in that blessed situation would walk out of the oncology clinic with the relief of never having to confront those difficult days again, Kristin walked out of our clinic with a mission. First, she asked if she could help with fundraising for our program, which she did. A year following Brandon’s visit to the West Coast medical center, Kristin took a leave of absence from her law practice to become the Senior Vice President of Community and Business Development at a national childhood cancer nonprofit. She also became involved with a small, local charitable organization called Cure Children’s Cancer (CURE) that helped provide support for families of area kids with the disease. Following her early volunteer days with CURE, Kristin’s efforts became all-consuming. She never returned to her law firm, instead dedicating her life to helping find a cure for kids with cancer.

  Three years after Brandon’s family’s momentous trip to the West Coast medical center, Kristin became the Executive Director of CURE, a position she has now held for nearly ten years. In that time, this small, local organization has become a national force in fundraising for children’s cancer care and research, raising millions of dollars each year. These desperately needed funds support our work with all types of cancer at our institution, but also support children’s cancer programs across the country. CURE also maintains its original goal of supporting families as they go through the difficult journey with their children. The CURE mission statement and vision statement summarize the organization well:

  CURE Childhood Cancer is dedicated to conquering childhood cancer through funding targeted research and through support of patients and their families. CURE Childhood Cancer believes that childhood cancer can be cured in our lifetime.

  Besides having had the privilege of caring for Brandon and other kids with cancer who now benefit by Kristin’s work, I have another, very personal connection to Kristin and CURE. My oldest son and a friend of his started an annual fundraising run in the name of a young man, Sam, who went to the same high school as our son. Sam was a promising athlete who tragically died of metastatic bone cancer. All four of my children have been involved with organizing the run over the past eight years. The funds raised from the run go to the Sam Robb Fund of CURE. This fund, with the help and guidance of Sam’s family and CURE, provides funds to support families going through their kids’ cancer therapy, and also provides full funding for a fellowship in pediatric oncology at Children’s Healthcare of Atlanta and Emory University. These fellows complete their clinical and research training with us and then join the world of children’s cancer-fighting doctors.

  Brandon’s disappearing tumor was a miracle for his family. His mother’s work since that time has been a miracle for all of us treating kids with cancer.

  For more about CURE:

  http://www.curechildhoodcancer.org/#sthash.i786wfd7.dpbs

  For more about the CURE Sam Robb Fund:

  http://www.curechildhoodcancer.org/named-funds/cure-named-funds/the-sam-robb-fund/#sthash

  .TLPbSnBY.dpbs

  Date of event: Summer 1993

  Helping Create Miracles for Babies of the Future

  Richard F. Jacobs, MD

  As a clinical researcher, I test potential new therapies for children that may or may not ultimately be proven to be beneficial. This usually requires randomly assigning some children to a group receiving the experimental therapy and others to a group receiving the existing, standard treatment. If there is no existing therapy, the experimental group is compared with a group receiving a placebo (“sugar pill”). The outcomes of the groups are then studied to determine if the new therapy was safe and effective compared with previous therapies or, if there are no existing therapies, compared to no therapy at all. Before any patient can be included in a research study, they must be thoroughly educated about the study, including all the potential risks. The patient (or parents, in the case of a young child) must then formally agree to participate in the research study. That process is called obtaining informed consent.

  It has always amazed me how willing, and even eager, parents are to help us seek new preventions, treatments, and cures for children, not knowing if the research study we are asking them to consider will benefit their own child. In 1993, we were conducting a very important research study, as part of a National Institutes of Health research network, of a medicine to treat babies infected at birth with a dangerous virus called cytomegalovirus or CMV. It was long known that babies who contracted a brain infection with CMV had a high likelihood of permanent and often total deafness. This particular research study required insertion of a deeply placed IV catheter (tube placed in a vein) to give an experimental medicine for six weeks to fight the CMV virus. The medicine was known to be effective in adults with various types of CMV infections, and its side effects were also well known in adults. But the medicine had never been studied in babies over such a long treatment period.

  In this study, babies were to be randomly assigned to either have the deep IV catheter inserted and receive six weeks of treatment with the medicine, or be assigned to a “no treatment” control group, which would receive no IV catheter and no treatment. Because the deep IV catheter carries its own risks, it could not ethically be inserted into babies merely to give a placebo. As a result, unlike many studies where the doctors, patients, and families are all unaware of which patients are receiving an experimental medicine and which patients are not, in this study everyone would know because the “no treatment” patients would not even have the deep IV catheter inserted. This meant explaining to parents, while obtaining informed consent, that their baby would be randomly assigned to either receive the research medication we wanted to evaluate for possible benefit or, because there was no standard treatment available for CMV infection, their baby would receive no treatment at all. Although all babies in the study, whether receiving the
experimental medicine or not, would be given a very comprehensive follow-up, testing, and a detailed assessment of all potential outcomes, parents would immediately know if their baby did or did not receive therapy.

  Obtaining informed consent from parents requires conscientiously providing all information and as complete a description as possible so they can make a truly informed decision regarding what’s best for their child. This great responsibility has always been an emotionally exhausting exercise for me. That is, until I met this particular family who made the process incredibly easy. I was explaining the CMV treatment study to them because their baby had CMV infection of the brain and was at high risk for deafness. Of course, as with all families, they wanted a treatment to help their baby and they asked complete and direct questions. But they started by saying they just wanted to help us answer the important medical questions we were addressing with the study, even if their baby was not one of those randomly assigned to receive the potentially beneficial medicine.

  As it turned out, their son was randomly assigned to receive the experimental treatment and tolerated it well despite some challenges due to the side effect of depressed white blood cell counts, something we knew to expect from this research medication. When enough patients had been enrolled in the study and all the results were analyzed, the study did show that the experi-

  mental treatment was effective in decreasing hearing loss and preserving babies’ hearing over time compared with the no-treatment group.

  This wonderful family’s son retained functional but somewhat decreased hearing, and the family opted to have cochlear implants inserted to bring their son’s hearing up to completely normal levels. Every time I see his family, they hug me and tell me they are so happy to have helped prevent hearing loss for other babies like their son. The study was published in one of the leading pediatrics journals, and the parents even asked me for an autographed copy. While they are convinced the experimental medicine preserved enough hear-ing for their baby to be completely normalized subsequently by the cochlear implants, they are even more convinced of the benefits of today’s research for children in the future. This medicine and another newer medicine based on it have continued to show promise in reducing deafness in CMV-infected babies, and they also appear to possibly reduce other ill effects of the infection in babies, including developmental delay and brain damage.

  I am constantly invigorated by the human spirit of those wanting to help answer questions for the benefit of future patients.

  Date of event:1975

  Making Miracles for Others

  Celia I. Kaye, MD, PhD

  I was providing consultations at a community hospital as a newly trained geneticist, a specialist in diagnosing and treating inherited diseases. Sarah was just a few months old, with severe, ongoing, and long-lasting diarrhea that had required hospitalization and IV fluids (given directly into a vein) for several weeks of her short life. Although we didn’t know what her diagnosis was, we did know she was failing. It was generally agreed that she was too small and fragile to survive, but we were still desperately trying to save her. Our goal was to keep her alive long enough to determine what was wrong with her in hopes that knowledge would lead to her treatment and cure. She was the first child to her young parents, who were terrified they might lose their baby and frustrated by our inability to help her. Since this was before the days we had the capability of giving adequate nutrition intravenously, Sarah was starving to death before our eyes.

  Sarah had a range of problems outside of my experience. As a very young and relatively inexperienced “expert,” I was at a loss to figure out what was wrong with her. Doing what others in my position routinely do, I sought help from colleagues elsewhere and found many who, despite not even know-

  ing this patient, gave of their time and energy to help from a distance. After talking to gastroenterology specialists (physicians treating stomach and intestinal disorders) and other genetics experts, we decided to try Sarah on a very new and still unproven type of baby formula that contained only the basic building blocks of nutrients rather than the complex nutrients found in standard baby formulas. We didn’t feel we had a choice—it was clear this baby would die if we continued standard formulas. It was a long and difficult trial, but Sarah ultimately was able to tolerate the new formula, gained weight, and was able to go home with her parents, much to their relief and ours.

  This was only the first step of a long odyssey of trying to figure out what was wrong with Sarah, prompted by the emergence of many new problems as the years passed. Although her intellectual development was normal and she met her childhood milestones, Sarah always had difficulty with feedings and diet, and her growth was very slow. She developed unusual skin changes. In later years, an abnormality in her bone marrow occurred, resulting in not enough mature blood cells circulating in her body, perplexing the blood and bone marrow experts. Despite these many complications, Sarah continued to progress well in other ways, went to school, and ultimately on to college. She was always the “different one,” requiring lots of medical attention, missing school, looking smaller and frailer than her friends. Sarah’s parents never wavered in their love and support for her, despite the ongoing emergence of new medical troubles that always seemed to surprise the experts. Now Sarah is in her thirties, and I have still never learned the cause of her illness, and her future remains uncertain.

  Although close to death when I first saw her, she “hung on” until we were able to find a way to feed her. Against all odds and all the experts’ predictions, Sarah lived. Of course, to her parents and to those of us taking care of her, this was a miracle. But perhaps an even greater miracle is how Sarah has responded to her numerous life-threatening medical developments and ongoing medical challenges.

  Sarah has chosen a career helping children find their own normalcy when faced with chronic medical problems—problems, like hers, that never go away. She has had the energy and the courage to turn her own story into one that can help others, and she is doing this every day. She is determined to make the future brighter for other children who will always be different from their friends and classmates. For those kids, Sarah is now a miracle maker.

  Date of event: 1956

  A Dramatic Cure Leads to Many More

  Michael S. Kappy, MD

  Harley A. Rotbart, MD

  Mike was a sixteen-year-old boy who had experienced a week of increasing numbness and weakness that was gradually ascending up his body from his feet toward his head. There are several very serious conditions that can cause these symptoms, including polio, lead poisoning, and a paralytic neurological condition called Guillain-Barré syndrome (GBS). He was admitted to a large community hospital over fifty years ago and treated emergently with injections of “British anti-Lewisite,” a medicine that was developed back in World War II to treat arsenic poisoning from chemical warfare. The same medicine was effective in other heavy metal poisonings like lead, and lead poisoning was one of the possibilities in this case. But, after many rounds of tests, Mike was ultimately determined to have GBS.

  Mike went on to develop all of the dreaded complications of GBS, including paralysis and failure of the part of his nervous system that controls breathing. Early one morning, he suddenly went into cardiorespiratory arrest—his breathing and heart stopped. After a prolonged and difficult resuscitation, and the recognition that he could not breathe on his own, Mike was placed in an iron lung machine, named such because iron lungs were made of metal and could help a patient to breathe.

  Iron lung machines are now obsolete because we have modern ventilator machines (also caused positive pressure ventilators) that can actively pump air into and out of patients’ lungs when their own breathing fails. But before positive pressure ventilator machines were developed, the only way to move air through the lungs of paralyzed patients was passively, using the iron lung (also called negative pressure ventilator). The iron lung is a body-size metal tube c
onnected to a vacuum pump. The patient is placed inside the tube, which forms a seal around the patient’s body. The machine cycles through suction and non-suction phases, the pace of which can be controlled by the doctors. When the suction part of the cycle was on, the iron lung would expand the patient’s chest cavity, drawing air into the lungs; when the suction was turned off, the chest cavity would return to its resting state, expelling the air. Only the patient’s head extended outside the machine. Mike remembers being told that if he had been an inch taller, he wouldn’t have fit! There were no breathing tubes involved as there are now with ventilator machines that directly deliver air into the lungs. The machine functioned a little like a mouth on a straw, and the body was the straw—when suction was applied, air entered Mike’s lungs, and when suction was released, air was released. Iron lung machines were widely used to treat the most severe forms of polio, in which patients’ breathing muscles were paralyzed, often permanently.

  The doctors treating Mike were desperately hoping that the successful resuscitation and breathing support he was receiving would allow him to recover, since most, but by no means all, patients with GBS slowly regain their muscle function.

  Indeed, Mike gradually regained his ability to move and breathe over a three-week period, and he was then treated with physical therapy for several months, including a three-month stay in a physical medicine rehabilitation hospital. Over the ensuing several months, he regained nearly all of his movement functions. Mike went on to graduate from high school. The path he then chose for his career, after surviving his life-threatening illness and witnessing the heroic efforts his caregivers made to pull him through, is a remarkable example of giving back, and it illustrates how miracles can ensue from “paying it forward.”