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Pharmacoscopy refers to an ex vivo real-time drug sensitivity profiling platform that has been shown to be of value in the treatment of leukemia (Snijder et al. 2017) (Kornauth et al. 2022) and may help to identify novel treatment opportunities for brain tumors as well (Lee et al. 2022). The rationale for pharmacoscopy-based drug sensitivity testing on real-time patient biopsies or surgery material is multiple: measuring drug response and sensitivity directly in real-time patient material, overcomes the problem of limited molecular biomarkers for established targeted therapeutic options and can identify effective drugs even for non-targeted therapies such as chemotherapy. It can...
Functional precision medicine (FPM) is a relatively new approach to cancer therapy based on direct exposure of patient- isolated tumor cells to clinically approved drugs and integrates ex vivo drug sensitivity testing (DST) and genomic profiling to determine the optimal individualized therapy for cancer patients. In this study, we will enroll relapsed or refractory pediatric cancer patients with tissue available for DST and genomic profiling from the South Florida area, which is 69% Hispanic and 18% Black. Tumor cells collected from tissue taken during routine biopsy or surgery will be tested.
Caregivers of pediatric brain tumor survivors may develop high levels of psychological distress, mostly depression and anxiety, with effects comparable to those of a traumatic event. Several studies suggest that Eye Movement Desensitization and Reprocessing could be a promising treatment in similar clinical populations (i.e. adults with brain tumor), but it has never been used for caregivers of pediatric brain tumor. The aims of the present study will be to test the feasibility of the Eye Movement Desensitization and Reprocessing Integrative Group Treatment Protocol for caregivers of pediatric brain tumor survivors in a clinical setting and to evaluate its effectiveness in...
This prospective registry study will evaluate doses utilized in eye plaque brachytherapy for the treatment of ocular melanoma and their associated outcomes. The goal of this study is to evaluate if lower doses of radiation can maintain high local control rates while minimizing the toxicities related to radiation therapy.
The Fabry Registry is an ongoing, international multi-center, strictly observational program that tracks the routine clinical outcomes for patients with Fabry disease, irrespective of treatment status. No experimental intervention is involved; patients in the Registry undergo clinical assessments and receive care as determined by the patient's treating physician. The primary objectives of the Registry are: - To enhance the understanding of the variability, progression, and natural history of Fabry disease, including heterozygous females with the disease; - To assist the Fabry medical community with the development of recommendations for monitoring patients...
NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll. If you are interested in clinical testing please consider seeing a local genetic counselor or other genetics professional. If you have already had clinical genetic testing and meet eligibility criteria for this study as shown in the Eligibility Section, you may enroll regardless of the results of your clinical genetic testing. While it is well recognized that hereditary factors contribute to the development of a subset of human cancers, the cause for many cancers remains...
The Family Caregiver Survey is a one-time, 30-minute, online survey for people living with and caring for a family member with dementia. The goal of this research is to explore the needs of family caregivers, specifically when it comes to managing swallowing difficulties (dysphagia).
This study aims to assess the diagnostic performance of a new fast MRI sequence named Neuromix compared to routine clinical MRI for brain tumor in pediatric patients
To evaluate the effect of intraoperative sedation dosage on awakening and recovery of consciousness after elective neurosurgical surgery.
The goal of this placebo-controlled double-blind Phase 2 clinical trial is to test in people with early Alzheimer's Disease. The main questions it aims to answer are: - Does treatment with fasudil, a ROCK-inhibitor, lead to significant improvement in working memory (based on computer-based working memory composite scores) compared to placebo in individuals with early Alzheimer's disease (AD) over 12 months? - What is the effect of fasudil treatment for 12 months on other cognitive functions, brain metabolism measured by Fluorodeoxyglucose Positron Emission Tomography (FDG-PET), and other relevant clinical functions and biomarkers in individuals...
