The Problem:
Sickle Cell is a hereditary disease that affects as many as 100,000 people in the United States. It is caused by a single mutation in the b-globin gene, causing the malfunction of hemoglobin, the component of the blood responsible for delivering oxygen to internal organs. The abnormal hemoglobin causes cell deformation, or “sickling,” of red blood cells, a disease hallmark. These sickle cells can block blood vessels, causing recurrent pain, and damaging the liver, heart and kidneys, which may lead to premature death.
Since the severe form of SCD can decrease the quality of life and shorten patients’ lives, developing novel therapeutics to treat the disease is a clear unmet medical need. However, the heterogeneity of the SCD manifestations and outcomes makes clinical trials very challenging and the success rate low.
The Solution:
The CP-SCD’s goal is to create a regulatory science strategy and de-risk medical developments to benefit individuals living with this disease. The CP-SCD consortium aims to bring together a wide range of SCD stakeholders, including partners from pharmaceutical industry companies, academic institutions, regulatory agencies and patient-advocacy organizations to accelerate treatments and therapies for SCD.
The consortium collaborates with C-Path’s Rare Disease Cures Accelerator-Data and Analytics Platform, the Quantitative Medicine Program, the Patient-Reported Outcome Consortium, the Regulatory Science group and others to accomplish its mission. We analyze clinical trial data, create mathematical models that predict disease development, identify novel biomarkers and provide regulatory solutions for the pharmaceutical industry that could make clinical trials more rapid, less expensive and increase the success rate, thereby making new therapies available to patients with SCD sooner.
The Impact:
CP-SCD collects, curates and analyzes clinical datasets. To date, CP-SCD has accumulated six datasets of clinical data from over 6,000 patients. Data analysis and disease progression modeling are underway to provide model-informed drug development tools for pharmaceutical companies. That enables them to provide better treatments and improve the quality of life for SCD patients. Together, we save lives.
