Bone marrow transplants have been used to treat about 500 people in the U.S. for sickle cell disease, said Kohn
Doctors at UCLA may have found a way to combat the complicated and deadly disease.
Sickle cell anemia (SCA) is a disease that affects 90,000-100,000 Americans. It is also a disease that heavily afflicts the Black community occurring in one of 500 African-American births. It has been revealed recently to the Sentinel that a huge break through may be on the horizon for those afflicted by SCA thanks to researchers at UCLA’s Eli & Edythe Broad Center of Regenerative Medicine & Stem Cell Research Center (BROAD).
“We’re publishing a paper describing some pre-clinical studies that we’ve been doing over the last three years,” said Dr. Donald Kohn, M.D. professor at UCLA, department of microbiology, immunology, and molecular genetics, the department of pediatrics and BROAD. “[This] would basically be a bone marrow transplant using the patients own bone marrow.”
SCA occurs when Hemoglobin S changes the shape of red blood cells causing them to get stuck easily in the small vessel, breaking into bits and pieces that can interrupt the body’s natural blood flow. The problem decreases the amount of oxygen flowing to the body tissues as well.
Symptoms include fatigue, paleness, rapid heart rate, and shortness of breath. Many sickle cell patients have painful episodes they call “crises” which can last hours or even days. Pain is usually felt in the long bones, back and chest. Patients have developed life-threatening conditions as a result of the disease.
If both parents have SCA, it is proven that their child will have it as well. If one parent has sickle cell anemia, the child will have the SCA trait. A child can only inherit this disease if both parents have this trait. Sickle cell disease is much more common in people of African and Mediterranean descent. It is also seen in people from the Caribbean, the Middle East, South and Central America.
The research was funded by the California Institute For Regenerative Medicines (CIRM), a stem cell initiative funded group. The research, led by Kohn, which is pre-clinical, is intended to support the clinical trial they hope to start within a year.
“Bone marrow transplants have been used to treat about 500 people in the U.S. for sickle cell disease,” said Kohn. “Since the basic problem with sickle cell disease is the red blood cells are abnormal and they’re made in the bone marrow, you can give a patient a new source of bone marrow stem cells that can make normal red cells.”
The transplants that have taken place, according to Kohn, were done with children and their siblings, who happened to be a perfect match. This was done among a small fraction of patients.
UCLA along with Children’s Hospital of Los Angeles and Oakland worked with patients with sickle cell disease. They received 25 bone marrow samples that they used for the studies. The research that they conducted showed some amazing results.
“It showed that if we insert into those cells a normal copy of the hemoglobin gene, then the red cells that they make in the test tube, don’t sickle,” said Kohn. “So we can block the sickling of the red cells that are made by adding a normal copy of the globin gene.
“Instead of giving them someone else’s cells, we need to find a match and they can still have rejection, using their own cells should be a perfect match and not be rejected if our fix to them makes them normal.
“This paper basically shows that at least in the laboratory, the cells that we have gene-modified, seem to function normally and not sickle.”
BROAD has submitted a grant to CIRM for clinical trials. It will have to be reviewed and if approved, they will proceed with doing research with patients in less than a year.
“We don’t know if it’s going to work, like any research, it’s unknown,” said Kohn. “We’ve written the trials very cautiously that we would enroll two patients first and wait six month to evaluate…if it looks promising, then to proceed to the next two and then the next two.
So it’s a total of only six patients that would be enrolled at the most.”
Dr. Kohn stated that the patients that they are looking to work with are young adults, 18 and over. They also are working with specific criteria of who would be eligible for the study.
Though in the early stages of study, there may be some light on the horizon for those afflicted with SCA.