SOUTH SAN FRANCISCO, Calif., Sept. 15, 2022 (GLOBE NEWSWIRE) — Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) introduced its assist for the Sickle Cell Disease Treatment Centers Act of 2022, which was launched at present and, if handed in its present type, would authorize for appropriations of $535 million in annual funding to assist sickle cell illness (SCD) remedy, analysis and schooling. The new laws is sponsored by Congresswoman Barbara Lee of California, Congressman Danny Davis of Illinois, Senator Chris Van Hollen of Maryland and Senator Cory Booker of New Jersey. The invoice would create a nationwide community of SCD remedy facilities by which healthcare suppliers and community-based organizations associate to optimize care supply, together with psychological assist, whereas implementing instruments that enhance schooling, coping and illness understanding by sufferers and households. The invoice focuses on affected person care, with knowledge assortment and analysis as further optimistic advantages.
“Sickle cell disease has long suffered from a lack of attention, investment and innovation, resulting in a lack of access to high-quality health care for those living with this devastating condition. Since our founding, GBT has been committed to partnering with the community to address and overcome these health inequities. The introduction of the Sickle Cell Disease Treatment Centers Act of 2022 marks a significant step toward equitable access for the sickle cell community by potentially creating the infrastructure of coordinated care that is so desperately needed, in addition to increasing education and broader community support,” mentioned Ted W. Love, M.D., president and CEO of GBT. “We commend Congresswoman Lee, Congressman Davis, Senator Van Hollen and Senator Booker for their dedication and efforts to bring this legislation forward, which we wholeheartedly support.”
If enacted, the invoice will set up a nationwide community of SCD remedy facilities based mostly on a hub-and-spoke mannequin related by way of nationwide/regional coordinating facilities, together with funding for at the very least 128 remedy facilities and 100 community-based organizations or nonprofit entities. The nationwide/regional coordinating facilities will facilitate knowledge assortment, monitoring and distribution, function a repository of finest observe tips, facilitate publication of analysis and academic supplies, in addition to promote public consciousness campaigns and different outreach supplies that broaden group assist.
Approximately 100,000 Americans at the moment reside with SCD.1 Despite the want for constant and coordinated remedy, few individuals with SCD have entry to multidisciplinary care groups.2 The life expectancy of individuals with SCD is 2 to a few many years shorter than the remainder of the inhabitants due, at the very least partly, to restricted entry to specialised care.3
The Council for Sickle Cell Disease Health Equity, an advisory council convened by GBT to assist drive a unified nationwide advocacy agenda, labored with the sponsors to develop the Sickle Cell Disease Treatment Centers Act of 2022.
“The key elements of the Sickle Cell Disease Treatment Centers Act of 2022 are based on a vision developed by a diverse group of stakeholders representing individuals living with sickle cell disease, caregivers, advocates, community-based organizations, physicians, and payers. The introduction and passing of this act have the potential to dramatically improve the lives of individuals living with sickle cell disease across the United States,” mentioned Biree Andemariam, M.D., director of the New England Sickle Cell Institute at the University of Connecticut and the chairperson of The Council for Sickle Cell Disease Health Equity. “The establishment and funding of more than 100 treatment centers would ensure far greater access to quality expert care for those living with sickle cell disease around the country, filling a longstanding inequity and largely ignored gap in the current healthcare landscape for the sickle cell disease community.”
About Sickle Cell Disease
It is estimated that greater than 100,000 individuals in the United States,1 52,000 individuals in Europe,4 as much as 100,000 individuals in Brazil,5 and tens of millions of individuals all through the world have sickle cell illness (SCD).1 SCD happens notably amongst these whose ancestors are from sub-Saharan Africa, although it additionally happens in individuals of Hispanic, South Asian, Southern European and Middle Eastern ancestry.1 SCD is a lifelong inherited blood dysfunction that impacts hemoglobin, a protein carried by pink blood cells that delivers oxygen to tissues and organs all through the physique.6 Due to a genetic mutation, people with SCD type irregular hemoglobin often known as sickle hemoglobin. When sickle hemoglobin turns into deoxygenated, it polymerizes to type rods, which deforms the pink blood cells into sickled – crescent-shaped, inflexible – cells.6–8 The recurrent sickling course of causes destruction of the pink blood cells, hemolysis and anemia (low hemoglobin attributable to pink blood cell destruction), which drives vascular irritation contributing to blockages in capillaries and small blood vessels (vaso-occlusion) that impede the circulation of blood and oxygen supply all through the physique. Episodes of painful vascular occlusions are generally known as vaso-occlusive crises (VOCs). The diminished oxygen supply to tissues and organs can result in life-threatening issues, together with stroke and irreversible organ injury.7–11 Complications of SCD start in early childhood and might embody neurocognitive impairment, acute chest syndrome, and silent and overt stroke, amongst different severe points.12 Early intervention and remedy of SCD have proven potential to switch the course of this illness, scale back signs and occasions, forestall long-term organ injury, and prolong life expectancy.7
About Global Blood Therapeutics
Global Blood Therapeutics, Inc. (GBT) is a biopharmaceutical firm devoted to the discovery, improvement and supply of life-changing therapies that present hope to underserved affected person communities, beginning with sickle cell illness (SCD). Founded in 2011, GBT is delivering on its aim to remodel the remedy and care of SCD, a lifelong, devastating inherited blood dysfunction. The firm has launched Oxbryta® (voxelotor), the first FDA-approved medication that immediately inhibits sickle hemoglobin (HbS) polymerization, the root explanation for pink blood cell sickling in SCD. GBT can also be advancing its pipeline program in SCD with inclacumab, a P-selectin inhibitor in Phase 3 improvement to handle ache crises related to the illness, and GBT021601 (GBT601), the firm’s subsequent era HbS polymerization inhibitor. In addition, GBT’s drug discovery groups are engaged on new targets to develop the subsequent wave of potential therapies for SCD. To study extra, please go to www.gbt.com and observe the firm on Twitter @GBT_news.
References
- Centers for Disease Control and Prevention. Sickle Cell Disease Data and Statistics (SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html. Accessed June 7, 2022.
- Grosse SD, et al. Pediatrics. Pediatrics. 2009;123(1):407–412.
- Lee L, et al. Public Health Rep. 2019;134(6):599–607.
- European Medicines Agency. https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125. Accessed June 12, 2022.
- Ministério da Saúde (Brasil), Protocolo Clínico e Diretrizes Terapêuticas da Doença Falciforme, Feb. 19, 2018
- National Heart, Lung, and Blood Institute. Sickle Cell Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease. Accessed February 23, 2022.
- Kato GJ, et al. Nat Rev Dis Primers. 2018;4:18010.
- Rees DC, et al. Lancet. 2010;376(9757):2018-2031.
- Kato GJ, et al. J Clin Invest. 2017;127(3):750-760.
- Caboot JB, et al. Paediatr Respir Rev. 2014;15(1):17-23.
- Nader E, et al. Front Immunol. 2020 Mar 13;11:454.
- Kanter J, et al. Blood Rev. 2013 Nov;27(6):279-87.
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