The month of September is filled with activities recognizing National Sickle Cell Awareness Month in the United States, all aimed at an ultimate cure for sickle cell disease (SCD).
Clinical trials to accelerate development of new gene and cell-based therapies within the next decade have already begun as part of the newly launched National Institutes of Health’s Cure Sickle Cell Initiative.
Other goals of the initiative include engaging academic researchers, the biotech industry, and patients and their caregivers to develop strategies for cures; determining the safest, most effective, and readily adoptable genetic therapies; and moving newly developed genetic therapies into clinical research, according to the NIH’s initiative website.
On Sept. 6, Global Blood Therapeutics hosted its 7th Annual Sickle Cell Disease Therapeutics Conference. Highlighting the latest medical advances and future trends in the treatment of SCD, the conference featured discussions and presentations from leading physicians, patient advocates, and healthcare policy makers.
Topics included the impact of sickle cell on quality of life, the clinical consequences of SCD beyond vaso-occlusive crises, and innovative programs to improve patient access to care.
The pharmaceutical company produces voxelotor, an investigational sickle cell therapy awaiting approval by the U.S. Federal Drug Administration.
The grant, awarded to the Critical Path Institute, will lead to a collaboration of multiple groups that will work together to boost development of disease therapies.
The idea is to work collaboratively on drug development tools and innovative methods and technologies, submit them to regulatory agencies for endorsement, then provide access to all sickle cell researchers.
“We are grateful to the DDCF for this funding, which will allow us to assemble a team of dedicated stakeholders united in identifying the best and most promising treatments for sickle cell disease,” Lynn Hudson, PhD, the chief scientific officer at C-Path, said in a news release. “Sickle cell research is of special interest to the foundation and it’s with a shared commitment and clear objectives that we will collaborate to meet the urgent need for better treatment outcomes.”
Elsewhere, groups such as the Sickle Cell Foundation of Georgia are planning an array of events during Sickle Cell Awareness Month. In Georgia alone, 7,000 people live with the condition.
“We want to raise awareness about the serious health problems associated with sickle cell disease, which is characterized by debilitating pain and life-threatening complications,” said Deborah McGhee McCrary, the foundation’s CEO.
“Awareness month is filled with health fairs, fundraisers, information on testing, and the latest research and treatments for sickle cell,” she added.
Visit the foundation’s website at www.sicklecellga.org for events, or call the foundation at 404-755-1641.
In recognition of Sickle Cell Awareness Month, the office of President Trump issued a message recognizing the disease that affects roughly 100,000 U.S. residents. “My administration is committed to supporting research to develop a cure to SCD that is available to all people, expanding on the achievements of current treatment options,” the late August statement said.
“Additionally, we are working to better train healthcare providers to identify individuals with SCD and improve the quality of care from infancy through adulthood.”
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