California Institute for Regenerative Medicine Awards City of Hope $5.74 Million for Severe Sickle Cell Disease Clinical Trial

The trial features a blood stem cell transplantation procedure developed at City of Hope

DUARTE, Calif.--()--The California Institute for Regenerative Medicine (CIRM) board awarded $5.74 million to City of Hope to fund a phase 1 clinical trial testing a novel blood stem cell transplantation procedure for adult patients with severe sickle cell disease (SCD).

The treatment involves induction of mixed chimerism. This refers to transplanting blood-forming stem cells from a healthy half-matched family member donor into a patient who has received a milder, less toxic chemotherapy treatment that removes some but not all of the patient’s diseased bone marrow stem cells. The donor blood stem cell transplants are depleted of some immune cells called CD4+ T cells prior to transplantation. This approach allows the donor stem cells to engraft and create a healthy supply of non-diseased blood cells without causing graft-versus-host disease, a side effect of bone marrow transplantation.

“The City of Hope transplant program in SCD is one of the largest in the nation,” said Joseph Rosenthal, M.D., chief of the Division of Pediatric Hematology/Oncology at City of Hope, Barron Chair in Pediatrics and the trial’s principal investigator. He noted that 27 transplants for SCD patients have already taken place at City of Hope, which also pioneered the development of a new method for bone marrow stem cell transplants from half-matched donors for patients with nonmalignant, severe life-threatening conditions and continues to be a national leader in providing blood and marrow stem cell transplantation treatment.

“CIRM funding will now allow us to conduct a phase 1 trial in six adult patients with severe SCD,” he added. “City of Hope believes this treatment will improve the quality of life of patients while also reducing the risk of graft-versus-host disease and transplant-related complications. Our hope is that this treatment can be eventually offered to SCD patients as a curative therapy.”

Defu Zeng, a professor of diabetes immunology and hematology/hematopoietic cell transplantation in City of Hope’s Diabetes & Metabolism Research Institute, and the trial’s co-principal investigator, developed this induction of mixed chimerism for the treatment of SCD in preclinical animal models.

SCD refers to a group of inherited blood disorders that cause red blood cells to take on an abnormal, sickle shape. Sickle cells clog blood vessels and block the normal flow of oxygen-carrying blood to the body’s tissues. Patients with SCD have a reduced life expectancy and experience various complications including anemia, stroke, organ damage and bouts of excruciating pain. An estimated 100,000 Americans are affected by SCD, according to the Centers for Disease Control and Prevention.

“The current standard of treatment for SCD is a bone marrow stem cell transplant from a genetically matched donor, usually a close family member,” said Maria T. Millan, M.D., president and CEO of CIRM. “This treatment is typically reserved for children and requires high doses of toxic chemotherapy drugs to remove the patient’s diseased bone marrow. Unfortunately, most patients do not have a genetically matched donor and are unable to benefit from this treatment. The City of Hope trial hopes to address this unmet medical need for adults with severe SCD.”

In addition to this type of transplant for SCD patients, City of Hope has also developed preclinical gene therapy clinical trials and a new approach for gene therapy using non-nuclease technologies.

“City of Hope is at the forefront of developing bolder gene therapy options for patients with severe sickle cell disease,” Rosenthal added. “Our research and clinical trials are creating hope for patients with this devastating disease.”

The Wanek Family Project for Type 1 Diabetes is also funding the research. Zeng’s lab studies have also shown that transplants with half-matched donors are able to cure autoimmune diseases such as type 1 diabetes, systemic lupus and multiple sclerosis in preclinical animal models. If the clinical trial with severe SCD patients shows positive results, City of Hope can extend it to treating autoimmune diseases such as type 1 diabetic patients.

About City of Hope

City of Hope is an independent research and treatment center for cancer, diabetes and other life-threatening diseases. Designated as one of only 49 comprehensive cancer centers, the highest recognition bestowed by the National Cancer Institute, City of Hope is also a founding member of the National Comprehensive Cancer Network, with research and treatment protocols that advance care throughout the world. City of Hope is located in Duarte, California, just northeast of Los Angeles, with community clinics throughout Southern California. It is ranked as one of "America's Best Hospitals" in cancer by U.S. News & World Report. Founded in 1913, City of Hope is a pioneer in the fields of bone marrow transplantation, diabetes and numerous breakthrough cancer drugs based on technology developed at the institution. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.


City of Hope
Letisia Marquez, 626-218-3398

Release Summary

The California Institute for Regenerative Medicine awarded $5.74 million to City of Hope for a phase 1 clinical trial for severe sickle cell disease.


City of Hope
Letisia Marquez, 626-218-3398