Sickle Cell Disease Fact Sheet

CIRM funds many projects seeking to better understand sickle cell disease and to translate those discoveries into new therapies.

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Description

More than 80,000 Americans have sickle cell disease and despite decades of research the average life expectancy has dropped from 42 in 1995 to 39 today. It is a disease that largely targets the African-American community and to a lesser degree the Hispanic community.

Sickle cell disease is a genetic disorder that causes red blood cells to assume a sickle shape under stress, clogging blood vessels and producing episodes of excruciating pain, called crises, and leading to progressive organ damage. By twenty years of age about 15 percent of people with sickle cell disease have had major strokes and by 40 almost half of the patients have significant mental dysfunction.

The most common recommendation for people with sickle cell disease is to stay hydrated. The more water a person drinks, the less likely it is that their abnormal blood cells will clog their blood vessels. Another effective treatment is a medication called hydroxyurea, which reduces crises by 50 percent and death by 40 percent, but most adults are not treated. The populations most effected by sickle cell disease also suffer from significant health care disparities, which lower the quality of care they receive for their disease.

Bone marrow transplants are used to treat children with the most severe cases of the disease. In fact one of CIRM’s board members, Bert Lubin, MD, the President and CEO of Children’s Hospital and Research Center Oakland, has been a leader in developing this therapy for kids with sickle cell disease (his bio is here). The replacement bone marrow cells generate an entirely new blood system for the patient. However, bone marrow transplants are extremely risky and require a matched sibling donor and even under the best conditions there is always the risk of rejection.

Research funded by California’s stem cell agency focuses on making bone marrow transplants safer and more effective for treating people with sickle cell disease. In one project, the researchers intend to remove bone marrow from the patient and fix the genetic defect in the blood-forming stem cells. Then those cells can be reintroduced into the patient to create a new, healthy blood system. Because the cells come from the patient this technique avoids the issue of rejection. Other researchers are developing ways of making bone marrow transplants safer.

Disease Team Award

University of California, Los Angeles

This team of researchers plans to remove bone marrow cells from people with sickle cell disease and fix the genetic mutation that causes the disease. The team will then reintroduce the new cells into the patient. Those cells will then generate new, healthy blood cells.

CIRM Grants Targeting Blood Diseases Including Sickle Cell Disease

Researcher name Institution Grant Title Approved funds
Donald Kohn University of California, Los Angeles Beta-Globin Gene Correction of Sickle Cell Disease in Hematopoietic Stem Cells $1,652,076
Paula Cannon University of Southern California Site-specific gene editing in hematopoietic stem cells as an anti-HIV therapy $1,499,400
John Chute University of California, Los Angeles Niche-Focused Research: Discovery & Development of Hematopoietic Regenerative Factors $5,174,715
Tippi MacKenzie University of California, San Francisco In Utero Embryonic Stem Cell Transplantation to Treat Congenital Anomalies $2,661,742
Irving Weissman Stanford University Identification and isolation of transplantable human hematopoietic stem cells from pluripotent cell lines; two steps from primitive hematopoiesis to transplantable definitive cells, and non-toxic conditioning of hosts for hematopoeitic stem cell transplan $1,341,403
Fyodor Urnov Sangamo BioSciences, Inc. A Treatment For Beta-thalassemia via High-Efficiency Targeted Genome Editing of Hematopoietic Stem Cells $3,518,075
Steven Artandi Stanford University Self-renewal and senescence in iPS cells derived from patients with a stem cell disease $931,285
Irving Weissman Stanford University Antibody tools to deplete or isolate teratogenic, cardiac, and blood stem cells from hESCs $1,463,814
Dianne McKay University of California, San Diego Role of intracytoplasmic pattern recognition receptors in HSC engraftment $615,639
Lili Yang University of California, Los Angeles Differentiation of Human Hematopoietic Stem Cells into iNKT Cells $614,400
Hiromitsu Nakauchi Stanford University Generation of functional cells and organs from iPSCs $5,436,308
Andrew Leavitt University of California, San Francisco Small molecule tools and scale-up technologies to expand human umbilical cord blood stem and progenitor cells for clinical and research use $1,416,600
Judith Shizuru Stanford University Purified allogeneic hematopoietic stem cells as a platform for tolerance induction $1,233,275
Ann Zovein University of California, San Francisco Human endothelial reprogramming for hematopoietic stem cell therapy. $2,319,784
David Davidson Bluebird Bio A Phase 1/2, Open Label Study Evaluating the Safety and Efficacy of Gene Therapy in Subjects with β-Thalassemia by Transplantation of Autologous Hematopoietic Stem Cells [REDACTED] $0
Luisa Iruela-Arispe University of California, Los Angeles Molecular Characterization and Functional Exploration of Hemogenic Endothelium $1,371,477
Inder Verma Salk Institute for Biological Studies Development of a cell and gene based therapy for hemophilia $2,298,634
Donald Kohn University of California, Los Angeles Stem Cell Gene Therapy for Sickle Cell Disease $8,833,695
Fred Gage Salk Institute for Biological Studies Development of Induced Pluripotent Stem Cells for Modeling Human Disease $1,737,720
Cornelis Murre University of California, San Diego Generation of long-term cultures of human hematopoietic multipotent progenitors from embryonic stem cells $473,952
Irving Weissman Stanford University Prospective isolation of hESC-derived hematopoietic and cardiomyocyte stem cells $2,471,386
Inder Verma Salk Institute for Biological Studies Curing Hematological Diseases $5,979,252
Hanna Mikkola University of California, Los Angeles Improving microenvironments to promote hematopoietic stem cell development from human embryonic stem cells $550,241
Nicholas Gascoigne Scripps Research Institute Role of Innate Immunity in hematopoeitic stem cell-mediated allograft tolerance $1,705,554
John Zaia City of Hope The Innovation-Alpha Clinic for Cellular Therapies (I-ACT) – A Program for the Development and Delivery of Innovative Cell-based Treatments and Cures for Life-threatening Diseases. $8,000,000
Tippi MacKenzie University of California, San Francisco Maternal and Fetal Immune Responses to In Utero Hematopoietic Stem Cell Transplantation $1,230,869
Hanna Mikkola University of California, Los Angeles A suite of engineered human pluripotent stem cell lines to facilitate the generation of hematopoietic stem cells $1,382,400
David Raulet University of California, Berkeley Inactivating NK cell reactivity to facilitate transplantation of stem cell derived tissue $952,896
Total:
$66,866,592.00

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