Sickle Cell Anemia Fact Sheet

Sickle Cell Anemia Fact Sheet

CIRM funds a large number of research projects investigating the basic biology of blood stem cells and uses for those cells in treating diseases, including sickle cell anemia. One CIRM-funded disease team is focusing on a project with the intent of bringing a therapy for the disease to the clinic within four years.

If you want to learn more about CIRM funding decisions or make a comment directly to our board, join us at a public meeting. You can find agendas for upcoming public meetings on our meetings page.

Learn more about stem cell research:
Stem Cell Basics Primer | Stem Cell Videos | What We Fund

Find clinical trials:
CIRM does not track stem cell clinical trials. If you or a family member is interested in participating in a clinical trial, please see the national trial database to find a trial near you: clinicaltrials.gov

The role of stem cells in sickle cell disease

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 Stem Cell Gene Therapy for Sickle Cell Disease $8,834,129
Luisa Iruela-Arispe University of California, Los Angeles Molecular Characterization and Functional Exploration of Hemogenic Endothelium $1,371,477
Steven Artandi Stanford University Self-renewal and senescence in iPS cells derived from patients with a stem cell disease $931,285
Fred Gage The Salk Institute for Biological Studies Development of Induced Pluripotent Stem Cells for Modeling Human Disease $1,737,720
Hanna Mikkola University of California, Los Angeles Improving microenvironments to promote hematopoietic stem cell development from human embryonic stem cells $550,241
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 The Salk Institute for Biological Studies Curing Hematological Diseases $5,979,252
Nicholas Gascoigne Scripps Research Institute Role of Innate Immunity in hematopoeitic stem cell-mediated allograft tolerance $1,705,554
Tippi MacKenzie University of California, San Francisco Maternal and Fetal Immune Responses to In Utero Hematopoietic Stem Cell Transplantation $1,230,869
David Raulet University of California, Berkeley Inactivating NK cell reactivity to facilitate transplantation of stem cell derived tissue $952,896
Judith Shizuru Stanford University Purified allogeneic hematopoietic stem cells as a platform for tolerance induction $1,233,275
Irving Weissman Stanford University Antibody tools to deplete or isolate teratogenic, cardiac, and blood stem cells from hESCs $1,463,881
Ann Zovein University of California, San Francisco Human endothelial reprogramming for hematopoietic stem cell therapy. $2,319,784
Tippi MacKenzie University of California, San Francisco In Utero Embryonic Stem Cell Transplantation to Treat Congenital Anomalies $2,661,742
Inder Verma The Salk Institute for Biological Studies Development of a cell and gene based therapy for hemophilia $2,298,634
Hiromitsu Nakauchi Stanford University Generation of functional cells and organs from iPSCs $5,436,307
Dianne McKay University of California, San Diego Role of intracytoplasmic pattern recognition receptors in HSC engraftment $615,639
Fyodor Urnov Sangamo BioSciences, Inc. A Treatment For Beta-thalassemia via High-Efficiency Targeted Genome Editing of Hematopoietic Stem Cells $6,374,150
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
Donald Kohn University of California, Los Angeles Beta-Globin Gene Correction of Sickle Cell Disease in Hematopoietic Stem Cells $1,652,076
Lili Yang University of California, Los Angeles Differentiation of Human Hematopoietic Stem Cells into iNKT Cells $614,400
Total:
$50,908,649.00

CIRM Blood Disease Videos

Resources

 

© 2013 California Institute for Regenerative Medicine