WASHINGTON and SOUTH SAN FRANCISCO, Calif., Dec. 2, 2021 /PRNewswire/ -- The ASH
Research Collaborative (ASH RC), a non-profit organization
established in 2018 by the American Society of
Hematology (ASH), and Global Blood Therapeutics, Inc.
(GBT) (NASDAQ: GBT) today announced that GBT will provide a grant
of $400,000 to the ASH RC in support
of the ASH RC Data Hub Sickle Cell Disease (SCD) program. With the
Data Hub SCD program, the ASH RC seeks to advance research and
outcomes for people living with SCD by expanding its current data
collection of real-world, research-grade data to include more than
half of people living with SCD in the
United States.
"We have a shared mission with GBT to help transform the
treatment of sickle cell disease," said Martin S. Tallman, M.D., of Memorial Sloan
Kettering Cancer Center, who serves as president of the ASH
Research Collaborative and the American Society of Hematology.
"Working closely with the SCD community, the ASH RC is committed to
efficiently capturing longitudinal real-world data to better
understand the natural history of sickle cell disease and to create
a national repository of high-quality data to accelerate research
and enhance clinical care. We aim to give the SCD community a
meaningful voice in the creation of one of the world's largest
repositories of real-world, research-grade data in SCD. We
appreciate GBT's support to help us work toward improving care and
outcomes for our patients with this rare disease."
The ASH RC fosters collaborative partnerships to advance
progress in hematology and improve the lives of people affected by
blood diseases. The foundation of the ASH RC is its Data Hub, a
multi-stakeholder, real-world data program that facilitates the
exchange of information on hematologic conditions to support
scientific inquiry, discovery, and quality improvement. Nearly 30
U.S. clinical sites providing SCD care are currently participating
in the Data Hub and are submitting HIPAA-compliant data
representing 6,000 individuals living with SCD. The Data Hub is
actively enrolling up to an additional 110 clinical sites, and the
ASH RC believes it is well positioned to capture data on more than
50,000 people in the U.S. living with SCD.
GBT is the first biopharmaceutical company to provide the ASH RC
with financial support through a grant that will help onboard
clinical sites and integrate their data to the Data Hub. These
sites are organized across 19 consortia also enrolled with the ASH
RC SCD Clinical Trials Network. The purpose of the SCD Clinical
Trials Network is to accelerate research that advances treatment
and care of those affected by SCD.
"Comprehensive real-world data can empower people with sickle
cell disease and their healthcare providers to make decisions
regarding their care, while also guiding the development of new
treatments and guidelines that we hope will transform SCD into a
well-managed disease," said Kim
Smith-Whitley, M.D., executive vice president and head of
research and development at GBT. "GBT is proud to support the ASH
RC and its critical mission to collect real-world data to help
address the urgent needs of people with sickle cell disease. There
has never been a more important time to advance research
initiatives with the potential to improve care and address
long-standing gaps in health equity for the SCD community."
The Data Hub can collect a wide variety of data, including
electronic medical record data, clinical and laboratory data,
genomic or molecular correlates, patient-reported outcomes, and
aggregated population data. These data are obtained from inpatient
and outpatient clinical sites, industry or government datasets,
registries, other U.S. or international sources, and directly from
patients. By using state-of-the-art technology to automate data
clinical capture and linkages to other data sources, the Data Hub
is focused on minimizing data capture burden. Patient information
collected in the Data Hub complies with national and local privacy
laws and regulations to protect patient privacy.
To learn more, please visit
www.ashresearchcollaborative.org.
About Sickle Cell Disease
Sickle cell disease affects more than 100,000 people
in the United States,1 an estimated 52,000
people in Europe,2 and millions of people
throughout the world, particularly among those whose ancestors are
from sub-Saharan Africa.3 It also affects people of
Hispanic, South Asian, Southern European and Middle Eastern
ancestry.3 SCD is a lifelong inherited rare blood
disorder that impacts hemoglobin, a protein carried by red blood
cells that delivers oxygen to tissues and organs throughout the
body.4 Due to a genetic mutation, individuals with SCD
form abnormal hemoglobin known as sickle hemoglobin. Through a
process called hemoglobin polymerization, red blood cells become
sickled – deoxygenated, crescent-shaped and
rigid.4-6 The sickling process causes hemolytic
anemia (low hemoglobin due to red blood cell destruction) and
blockages in capillaries and small blood vessels, which impede the
flow of blood and oxygen throughout the body. The diminished oxygen
delivery to tissues and organs can lead to life-threatening
complications, including stroke and irreversible organ
damage.5-8
About the ASH Research Collaborative
The ASH Research
Collaborative (ASH RC) is a non-profit organization established by
the American Society of Hematology (ASH) to improve the lives of
people affected by blood diseases by fostering collaborative
partnerships to accelerate progress in hematology. The foundation
of the ASH RC is its Data Hub and Clinical Trials Network. Through
the ASH RC's state-of-the-art data-sharing platforms and
patient-centric approach to enrollment, design, and execution of
clinical trials for SCD, the ASH RC is making it quicker and more
efficient for companies to develop new treatments to help those
suffering from hematologic conditions, and to maximize the value of
those treatments post-approval. The ASH RC aims to transform
research and practice in malignant and non-malignant hematologic
diseases throughout the world, for the benefit of patients and the
hematology community.
About Global Blood Therapeutics
Global Blood
Therapeutics, Inc. (GBT) is a biopharmaceutical company
dedicated to the discovery, development and delivery of
life-changing treatments that provide hope to underserved patient
communities. Founded in 2011, GBT is delivering on its goal to
transform the treatment and care of sickle cell disease (SCD), a
lifelong, devastating inherited blood disorder. The company has
introduced Oxbryta® (voxelotor) tablets, the first
FDA-approved treatment that directly inhibits sickle hemoglobin
polymerization, the root cause of red blood cell sickling in SCD.
GBT is also advancing its pipeline program in SCD with inclacumab,
a P-selectin inhibitor in Phase 3 development to address pain
crises associated with the disease, and GBT021601 (GBT601), the
company's next-generation hemoglobin S polymerization inhibitor. In
addition, GBT's drug discovery teams are working on new targets to
develop the next wave of potential treatments for SCD. To learn
more, please visit www.gbt.com and follow the company on
Twitter @GBT_news.
References
- Centers for Disease Control and Prevention website. Sickle
Cell Disease
Research. https://www.cdc.gov/ncbddd/hemoglobinopathies/scdc-understanding-sickle-cell-disease.html.
Accessed December 1, 2021.
- European Medicines Agency.
https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3182125.
Accessed June 12, 2020.
- Centers for Disease Control and Prevention website. Sickle
Cell Disease
(SCD). https://www.cdc.gov/ncbddd/sicklecell/data.html.
Accessed June 3, 2019.
- National Heart, Lung, and Blood Institute website.
Sickle Cell
Disease. https://www.nhlbi.nih.gov/health-topics/sickle-cell-disease.
Accessed August 5, 2019.
- Rees DC, et al. Lancet.
2010;376(9757):2018-2031.
- Kato GJ, et al. Nat Rev Dis Primers.
2018;4:18010.
- Kato GJ, et al. J Clin Invest.
2017;127(3):750-760.
- Caboot JB, et al. Paediatr Respir Rev.
2014;15(1):17-23.
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SOURCE American Society of Hematology