It's not every parent who gets the opportunity to be involved in research professionally. Nearly 10 years ago my daughter was diagnosed with hydrocephalus, a neurologic condition characterized by abnormal production and absorption of cerebrospinal fluid (CSF) in the brain, sometimes resulting in a buildup of fluid and increased pressure inside the head. Learning that my child was going to live with the pain and limitations of this chronic illness was devastating, and eventually led to my current role as Clinical Coordinator for the Neurodynamics Research Lab at Children's Hospital Boston.
After my daughter's third brain surgery at 14 months, in which she received a ventriculoperitoneal shunt to regulate her CSF flow, my priority became guiding her through the healing process and helping her achieve developmental milestones. But I was often overwhelmed by how much is still unknown about hydrocephalus, and we were faced with the prospect of her shunt failing on any given day, possibly without notice. The fact that this would require future brain surgeries was so illogical and unacceptable that I wanted to help find a better solution.
Getting involved in a research study was the most direct contribution we could make. My daughter's neurosurgeon, Dr. Joseph Madsen, first enrolled us in his lab's Transcranial Doppler Ultrasound protocol, designed to better understand why some people have difficulty processing their CSF and others do not. My younger daughter, who does not have hydrocephalus, was also enrolled as a control. The fact that this study uses ultrasound, a noninvasive technique, was important in our decision to enroll—as was the possibility that our data might help in designing a better shunt or even a shunt alternative.
I was hooked. Contributing to a study that might impact my daughter's health gave me great hope, but I wanted to do more. I had a degree in writing, and my prior career included proposal and grant writing, so volunteering those services was a concrete place to begin.
At weekly lab meetings, I learned about other projects, such as developing "smarter" shunts, testing drugs to treat the headaches caused by hydrocephalus, and possibly hydrocephalus itself, and—most attractive to me as a parent—a protocol for determining whether a patient is shunt-dependent or not. Before long, I transitioned into an official capacity as Clinical Coordinator.
Once a family has been identified and educated about the study and the consenting process is complete, I work with them to coordinate appointment times and locations, further explain the protocols in lay language and sometimes share my personal experience. When I meet parents whose new baby has just been diagnosed, their pain is often palpable, and the opportunity to share my daughter's story and possibly convey some hope is truly an honor.
Each bit of data families contribute is a potentially critical part of developing better tools and, hopefully, preventive measures. But getting patients into a research study can be complicated. Our lab has a bit less of a problem than some, because parents of children with neurologic conditions are highly motivated to help researchers find answers. In fact, they often feel a sense of urgency, which our lab understands well, having three members whose own lives have been touched by hydrocephalus. But even in families with less devastating conditions, coming in for research visits can be burdensome, especially if it requires missing school or work.
Even when families are eager to participate, scheduling time on MRI machines, ultrasound and other necessary equipment can be a challenge. Research sometimes has to take a back seat to clinical needs, leaving less appealing slots for research tests—typically early mornings, nights or weekends. Many parents must wait until the summer months when their child is out of school—just when vacation schedules and increased clinical requests limit available research slots. At this writing, our lab is on the verge of losing two good candidates for studies of CSF flow purely for scheduling reasons.
While some studies may require no more than an extra blood draw, an infusion or stepping on a scale, others can involve time-consuming, claustrophobic and uncomfortable procedures, sometimes entailing a small risk to the patient. Working with parents' sensitivities to their child's comfort zone is an important part of coordinating a study. We need to approach this task delicately, because ultimately a patient or the family shouldn't become involved in something that they're not convinced is right for them. Not all parents' concerns can be mitigated, and the opportunity to collect important data is missed.
In addition, many study procedures can be challenging for children, calling for much advance preparation. Children's researchers studying the genetics of autism, for example, have developed an elaborate protocol, beginning at home, to ameliorate these children's fears about blood draws. A Children's study of dyslexia is among the very first to do functional MRI brain scans in children as young as 4, but requires extra time and game-playing to familiarize them with the scanner and motivate them to hold their bodies still. Sometimes a Child Life Specialist is needed to help children deal with stressful or uncomfortable examinations. All of this is very labor-intensive and requires a big commitment from families, who have to help coax and prep their children.
Keeping families involved long-term is especially challenging. It's the rare study that directly benefits the patients volunteering, and parents can become frustrated with a seeming lack of measurable progress. With a complex disorder like hydrocephalus, it can take numerous studies looking at different parts of the problem to find ways to make it better—and families involved in a single study may not fully appreciate how it helps advance the whole.
Another subtle yet pivotal challenge is getting clinicians to be more aware of research studies, some of them elsewhere in the hospital, so they will refer more patients to them. Earlier this year, I visited the Emergency Department (ED) with my daughter, who was experiencing prolonged, intractable headaches. As it happened, a study in our lab in Neurosurgery was testing a new diagnostic device, and I was certain that the ED team would offer this device and enroll us in the study. In fact, it took a lengthy discussion to convince the clinician to pursue use of the device before ordering a standard CT scan. Had I not been personally involved in the study, this opportunity might have been lost. Such missed opportunities are especially unfortunate for smaller research labs that must work hard to find patient candidates.
As someone who's experienced the difficulties of keeping patients in studies, and also the inconveniences for families already coping with a chronic illness, my hope is to make this process more successful for everyone. It's incredibly gratifying to be involved with families professionally, knowing that the studies we're conducting may one day provide better understanding and better treatments for hydrocephalus. For families who have not considered contributing to clinical research, I urge you to speak to your physician, visit CHB-CONNECT, or read more about a special research unit at Children's and how individual patients are making a difference.