Cerebral Palsy (CP) Pediatric Research and Clinical Trials

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Collaborating to build robotic clothing for children with CP

Countless scientific epiphanies never leave the bench – unless there’s the kind of serendipitous encounter that set Boston Children’s Hospital psychologist Gene Goldfield on a path he never expected to follow.

One in eight babies are born prematurely, putting them at greater risk for cerebral palsy, an inability to fully control their muscles. Goldfield saw these children being wheeled around the hospital, and was convinced that they did not have to be wheelchair-bound.

Cerebral palsy hand actuatorDuring early infancy, he knew, the developing brain naturally undergoes a rewiring of its circuits, including those that control the muscles. Could some type of early intervention encourage more typical motor development by replacing damaged circuits with more functional connections?

At Boston Children’s Innovators’ Forum, Goldfield discussed his envisioned solution: the use of programmable robots to promote new brain connectivity and improve mobility.

Read the full story in Boston Children's Vector blog.

Groundbreaking hypothermia treatment may have implications for CP

Research at Boston Children's Hospital is leading to many clinical innovations in potentially preventing cerebral palsy in infants. One of the leading causes of cerebral palsy may be a lack of oxygen at birth, or hypoxic-ischemic encephalopathy (HIE).  Premature infants are particularly at risk to experience hypoxic-ischemic episodes. 

Since 2007, Anne Hansen, MD, MPH, medical director of the NICU, and Janet Soul, MD, CM, associate director of the Neonatal Neurology Program, have been offering newborn patients a new hypothermia treatment that may prevent or minimize the long-term consequences of brain injuries caused by HIE, including cerebral palsy.  The hypothermia treatment involves placing an infant onto a cooling blanket, which quickly brings down the body temperature. The cooling likely reduces brain damage, by lowering the overall energy needed for the brain to function and by slowing down the enzyme pathways in the brain that lead to death of the brain cells. Boston Children's is the first and only hospital in Massachusetts to offer this protocol.

Clinical Innovation: Tabletop therapy technology

tabletop therapy for cerebral palsy

The problem

By the time some patients get to the Cerebral Palsy Program at Boston Children's Hospital, they're already uncomfortable, anxious and in no mood for the frustrating and sometimes painful stretching and dexterity exercises involved in their therapy.

Innovative solution

Developer Paolo Bonato, PhD, director of the Motion Analysis Laboratory at Spaulding Rehabilitation Hospital (SRH), led a multidisciplinary team from SRH and the Harvard School of Engineering and Applied Sciences, collaborating with Donna L. Nimec, MD, and Brian Snyder, MD, PhD, both from Boston Children's Orthopedic Center, to develop a system that would be fun for children to use, but also deliver legitimate therapeutic value. The result is like a giant iPad, programmed with custom-developed games and activities that encourage the principal elements of cerebral palsy therapy. (Patients stretch by reaching for the keys of a virtual piano, and they coordinate both of their hands by capturing animated butterflies.) Games can be designed around specific goals, such as range-of-motion or increased visual skills.

Implications

When patients at Boston Children's Cerebral Palsy Program tested the tabletop, their response was overwhelmingly positive. The project is still in development, but the team envisions a system in which hospitals maintain progress records for each patient who uses the system. Patients and their families would be able to track their progress and plan therapy goals, giving kids something positive to look forward to at therapy time.

Other cerebral palsy research underway

Simon Manning, MD and colleagues in Boston Children's Department of Neurology and Neurobiology Program are investigating drug treatments for reducing the neurological conditions caused by HIE, including cerebral palsy.

•   When given within 48 hours of the hypoxic-ischemic episode, one drug called topirimate, reduced neuromotor
    abnormalities in animal models. 
•   Another drug, memantine, normally used to treat Alzheimer's, reduces the white-matter injury after a
    hypoxic-ischemic episode.  While this drug has only been tested in animals models, it holds promise to reduce
    brain damage in premature infants, including cerebral palsy.

Others are investigating how to prevent or reduce periventricular leukomalacia (PVL), which refers to damage and softening of the white matter, the inner part of the brain that transmits information between the nerve cells and the spinal cord, as well as from one part of the brain to another.   Infants who experience PVL are at a higher risk of developing cerebral palsy.

•   Neurologist Paul Rosenberg, MD, PhD has shown that Vitamin K may boost newborns antioxidant mechanisms and
    reduce effects of PVL.  He is also testing other enzymes that may prove protective.
•   Joseph Volpe, MD, neurologist-in-chief emeritus at Boston Children's, has been trying to discern what factors lead
    to PVL through monitoring infants, as well as laboratory and animal studies.  Once Volpe and his team figures out
    the cause, they can begin tackling how to stop it.
•   Neurologist Adre du Plessis, MD, believes PVL's root cause comes from impaired regulation of blood flow in the
    brain.  He's been monitoring premature babies at length searching for predictors of PVL and disturbances in blood
    flow.  Predicting which babies are most at risk will help refine neonatal care, minimize brain damage and provide
    grounds for testing the drugs mentioned above. 

While these treatments and studies do not offer a cure for children who already have the condition, this continued research may reduce the future number of cases of cerebral palsy and other neo-natal neurological conditions.

Clinical trials for cerebral palsy

Boston Children’s is known for pioneering some of the most effective diagnostic tools, therapies and preventive approaches in pediatric orthopedics, neurology, orthopedic surgery and neurosurgery. A significant part of our success comes from our commitment to research—and to advancing the frontiers of mental health care by conducting clinical trials.

Boston Children’s coordinates hundreds of clinical trials at any given time. Clinical trials are studies that may involve:

•   evaluating the effectiveness of a new drug therapy
•   testing a new diagnostic procedure or device
•   examining a new treatment method for a particular condition
•   taking a closer look at the causes and progression of specific diseases 

Boston Children’s is involved in several multi-site clinical trials and studies with promise for diagnosing and treating neuromuscular disorders. While children must meet strict criteria in order to be eligible for a clinical trial, your child may be eligible to take part in a study. Before considering this option, you should be sure to:

•   consult with your child’s treating physician and treatment team
•   gather as much information as possible about the specific course of action outlined in the trial
•   do your own research about the latest breakthroughs relating to your child’s condition 

Taking part in a clinical trial at Children’s is entirely voluntary. Our team will be sure to fully address any questions you may have, and you may remove your child from the medical study at any time. 

•   Search current and upcoming clinical trials at Boston Children’s
•   Search the National Institutes of Health’s list of clinical trials taking place around the world

We are grateful to have been ranked #1 on U.S. News & World Report's list of the best children's hospitals in the nation for the third year in a row, an honor we could not have achieved without the patients and families who inspire us to do our very best for them. Thanks to you, Boston Children's is a place where we can write the greatest children's stories ever told.”
- Sandra L. Fenwick, President and CEO

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