Medical treatments of tomorrow are available to the
region’s children today, thanks to the work of the Miami
Children’s Hospital Research Institute, a regional leader
in bringing the newest innovations for the diagnosis,
treatment and cure of pediatric illnesses.
In 2013, the institute was engaged in 225 active research
studies funded through academic affiliations, grants,
industry, the Children’s Oncology Group and the Miami
Children’s Hospital Foundation.
Medical specialties involved with clinical research studies
during the year included adolescent medicine, radiology,
cardiology, neurology and neurosurgery, critical care,
dentistry, urology, psychiatry, gastroenterology, genetics,
orthopedics, pulmonology, pediatric surgery, nursing
and hematology-oncology.
2013 Research Highlights:
Humanitarian Use of Activa
®
Deep Brain Stimulation Therapy
Dystonia is a debilitating progressive disorder in which
sustained muscle contractions cause abnormal body
posture and twisting of limbs in affected children. No
specific treatment is available for the disorder though
deep brain stimulation (DBS), via implantation of a
medical device similar to a pacemaker, has helped
control and in some cases, decrease symptoms. The
Research Institute in 2013 obtained approval from the
FDA for humanitarian use of deep brain stimulation for
the treatment of dystonia and is managing the required
supervised research protocol. The study is led by
Dr. Sanjiv Bhatia of the Division of Neurosurgery and
Dr. Sayed Naqvi of the Division of Neurology.
Exploring Better Treatments for
Pediatric Cardiac Patients
Heart valves that can be implanted via a catheter,
rather than open-heart surgery, show great promise in
supporting the care and improving the lives of patients
with valve disorders, many of whom must undergo
repeated interventions over a lifetime as valves wear out
and require replacement. The Heart Program at Miami
Children’s is participating in a study to assess the safety
and effectiveness of the Edwards SAPIEN™ Transcatheter
Heart Valve in patients with dysfunctional right
ventricular outflow tract (RVOT). Miami Children’s is the
only facility in Florida offering this treatment. The study is
led by Dr. John Rhodes of the Division of Cardiology.
Testing of Compounds to Increase
Knowledge in Cancer Treatment
Can natural herbal remedies used as part of traditional
Chinese therapy help improve the treatment experience
for patients with cancer? A team at the Miami Children’s
Research Institute is doing preclinical research to
assess whether defined extracts from 14 herbs used in
Chinese therapy can support the immune system and
reduce chemotherapy-induced side effects in cancer
patients. Pre-clinical research supports the observations
of clinical studies conducted in 2013 evaluating the
effect of LCS101 in reducing side effects and enhancing
immune support. The LCS101 research is led by
Dr. Steven Melnick, and Cheppail Ramachandran, PhD,
of the Division of Pathology.
Research for a Brighter Tomorrow
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