Research study is open to participants worldwide to advance understanding and treatments these rare diseases
Today marks the launch of a groundbreaking study by the Raymond A. Wood Foundation in collaboration with the National Organization for Rare Disorders (NORD®), aimed at researching rare hypothalamic-pituitary brain tumors. These tumors can lead to a range of debilitating symptoms including hormonal deficits, visual impairments, psychosocial challenges, cognitive impairment, and hypothalamic dysfunction, resulting in temperature dysregulation, sleep problems, obesity, and hyperphagia.
The Hypothalamic-Pituitary Brain Tumors Patient Registry establishes a global platform for individuals affected by these rare tumors to share vital information. Its primary goal is to create an international resource that will drive future scientific research in this field. Occurring in approximately one out of every million people, hypothalamic-pituitary brain tumors present complex challenges, with treatment typically involving surgical resection with or without radiotherapy. The clinical manifestations vary widely, depending on factors such as tumor location, size, growth pattern, and its relationship to adjacent cerebral structures.
Amy Wood, Co-Founder and Executive Director of the Raymond A. Wood Foundation, emphasized the significance of this initiative, stating, “This global resource will provide data for researchers to advance drug development and treatments, centered around patient needs to help improve care for our loved ones.”
To raise awareness and encourage participation, the Raymond A. Wood Foundation will roll out an extensive launch plan, including a social media campaign, webinars, and events.
“The success of the registry hinges upon community participation. Our goal is to enroll as many patients, parents, or legal guardians as possible,” said Wood.
The Hypothalamic-Pituitary Brain Tumors Patient Registry is a comprehensive natural history study comprising electronic surveys designed to gather information about the patient experience and disease progression. Patients, along with their caregivers or guardians, can input data from anywhere in the world. All information is kept confidential and securely stored in the IAMRARE online portal. The Raymond A. Wood Foundation may share the data with individuals or institutions conducting research or clinical trials, subject to approval by the study’s governing board, which includes scientists, doctors, and patient advocates.
The Raymond A. Wood Foundation and NORD have joined forces in this endeavor, leveraging NORD’s expertise in natural history study platforms as part of their collective mission to address all 7,000 rare diseases.
“NORD is delighted to see the launch of this new important natural history study. This innovative and critical research could not come at a better time for members of the hypothalamic-pituitary brain tumors community. Together with NORD, this new research endeavor can elevate the critical voices and stories of patients, families and members of the rare disease community,” said Aliza Fink, DSc Director of Research Data & Analytics, NORD.
