GBT Supports the ASH Research Collaborative’s Data Hub Sickle Cell Disease Program
December 02 2021 - 8:00AM
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 DiseaseSickle 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 CollaborativeThe 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 TherapeuticsGlobal 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.
ASH Contact:Andrea Fischer, American Society of
Hematology202-823-4884afischer@hematology.org
GBT Contact:
Steven Immergut (media)650-410-3258simmergut@gbt.com
Courtney
Roberts (investors)650-351-7881croberts@gbt.com
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