Searching for a cure for sickle cell disease

For children and adults with sickle cell disease, usual care involves lowering the number and severity of complications, but it does not get rid of sickle cell disease. With more children and adults looking to City of Hope for a cure to sickle cell disease, we are committed to providing you or your child the best possible care. We provide treatment that goes beyond just managing symptoms. New treatments that we are actively researching involve less toxic transplants from a half-related donor and more effective options for gene therapy. An important part of this new effort is the collaboration across multiple disciplines. Our multidisciplinary team of doctors, nurses and other health professionals has decades of experience in treating inherited blood disorders such as sickle cell disease.

Our expertise in finding a cure for sickle cell disease includes the following research:

Bone Marrow and Stem Cell Transplantation

City of Hope is a world leader in setting standards for stem cell transplantation and improving long-term outcomes in both children and adults. Our Hematologic Malignancies and Stem Cell Transplantation Institute is a leader in groundbreaking and new treatments.         

 

Our expert researchers have explored a cure for sickle cell disease through stem cell transplant using a half-related donor. This is someone whose tissues are only half identical, yet the transplant works as well as with a complete match. This provides you or your child with more donor choices.     

 

In addition, we have new and improved conditioning regimens (including chemotherapy and/or radiation) for patients needing a transplant. We’ve developed an improved nonmyeloablative regimen (a less harsh conditioning regimen) that allows patients who may not be able to do the conventional conditioning, either because of age or presence of complications, to have a successful transplant. We’ve also developed a less toxic way to perform transplants with the usual harsh and more reliable form of conditioning, called myeloablative. Our new myeloablative conditioning regimen lessens the chance of a transplant failing.

At City of Hope, we believe the future of medicine can be found deep within each of us — in our own stem cells. The Center for Gene Therapy, within the Hematologic Malignancies and Stem Cell Transplantation Institute, is dedicated to unlocking that possibility through world-class research and turning that knowledge into real-world treatments and cures as quickly as possible.          

 

Our research efforts include a new gene therapy technology that may have the potential to cure serious genetic diseases such as sickle cell disease. This gene therapy technology may give the same benefits as transplantation, but without the problems that come with using cells from a donor.

 

City of Hope is also researching a treatment approach that would increase the number of stem cells in the blood. In the future, these stem cells could then be genetically corrected in a lab and injected back into the patients as a treatment for sickle cell disease.         

 

As a patient at City of Hope, you or your child will be at the center of leading-edge research that leads to the best and newest treatments. In a given year, City of Hope conducts more than 500 clinical trials, giving many of our patients access to a clinical trial that meets their specific needs.

 

Our multidisciplinary team of doctors, nurses and other health professionals has decades of experience in treating patients with blood disorders. Joining a clinical trial may help you or your child and may also lead to improved care for future patients. To learn more about sickle cell disease clinical trials at City of Hope, contact one of our nurse coordinators: