Disease Fact Sheets
All Diseases	Sickle Cell Disease

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 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 suffer major strokes and by 40 years of age almost half of the patients have had central nervous system damage leading to 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 mortality 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 lowers the quality of care they receive for their disease.

Bone marrow transplants are also being used to treat children with the disease – however, bone marrow transplants are extremely risky and require a matched sibling donor. Even under the best conditions there is always the risk of rejection.
The bone marrow is where blood-forming stem cells reside, which form the entire blood system including red blood cells. A bone marrow transplant essentially replaces a person’s defective blood system with a system that produces normal blood cells. Up to 10 percent of patients die after a bone marrow transplant from the actual procedure and another 10 percent die because of rejection issues.

CIRM has funded a $9 million disease team to develop a more effective and safer bone marrow transplant to treat sickle cell disease (here is a summary of that work). The team is led by Dr. Donald Kohn, director of the Human Gene Medicine Program at UCLA, a scientist with the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA and a professor of microbiology and pediatrics.

Kohn and his team intend to develop a treatment whereby they will remove bone marrow from people with sickle cell disease, fix the genetic defect that causes the disease, and then transplant those repaired cells back into the patient. According to Kohn, this approach has the potential to permanently cure the illness with significantly less toxicity than with a bone marrow transplant from another person.

So far, Kohn and his team have shown – in the laboratory – that they can take cells from a patient with sickle cell disease and genetically alter these cells to prevent sickling. The team expects to start talking with the FDA about a future trial by June of 2011. Once the FDA has given the approval for the research to move forward the Kohn lab will work with the other members of the CIRM Disease Team at the University of Southern California and the Childrens Hospitals in Los Angeles and Oakland, to push the trial into the clinic.

CIRM Grants Targeting Sickle Cell Disease

• Stem Cell Gene Therapy for Sickle Cell Disease
• Curing Hematological Diseases

Resources

• NIH: What is Sickle Cell Anemia?
• CDC: Sickle Cell Information
• Find a clinical trial near you: NIH Clinical Trials database
• Sickle Cell Disease Association of America
• Sickle Cell Disease Foundation of California
• American Sickle Cell Anemia Association
• Sickle Cell Association