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Stem Cells to Cure Sickle Cell

A new way to cure sickle cell disease for more patients.

Sickle cell is a serious disease that causes pain, anemia, infection, organ damage, and even stroke. It’s the most common inherited blood disorder in the U.S. The good news is bone marrow transplants can be a cure. The bad news is not every patient has a matching donor. Now, researchers are looking at a new way to offer more patients transplants.
Madisyn Travis is like any other nine-year-old, but there’s something that sets Madisyn apart. She has sickle cell, an inherited red blood cell disease.
“It makes me feel bad and sometimes I have to go to the hospital,” Madisyn told Ivanhoe.
“It’s really hard to see her life interrupted,” Denise Travis, Madisyn’s mom, told Ivanhoe.
However, soon Madisyn will get a bone marrow transplant to cure her disease. Her little brother or sister are both matches and one will be the donor.
Madisyn is one of the lucky ones. Only 14 percent of patients have a matching sibling.
“Ten years ago, we’d just tell them, ‘sorry you have no family member. We can’t transplant you,’” Shalini Shenoy, MD, Professor of Pediatrics, Medical Director, Pediatric Stem Cell Transplant Program, Washington University School of Medicine, St. Louis Children's Hospital, told Ivanhoe.
Dr. Shalini Shenoy is studying a new option for patients without related donors. Stem cells from a baby’s umbilical cord can be infused in the arm. They travel to the bone marrow, settle there, and make new cells.
“Now, with this, we can take another 30 percent or 40 percent to transplant,” Dr. Shenoy said.
Madisyn is excited about a cure and even though they annoy her at times, she’ll have her brother and sister to thank for it.
Sickle cell is more common in minorities, occurring in about one in every 500 African Americans and about one in every one-thousand Hispanic Americans.
So far, just three patients have been treated with the umbilical cord blood transplant using unrelated donor cord blood. Ten research centers across the country are participating in Dr. Shenoy’s study.



MEDICAL BREAKTHROUGHS
RESEARCH SUMMARY

TOPIC:  STEM CELLS TO CURE SICKLE CELL
REPORT:  MB #3717

BACKGROUND: Sickle cell disease is a disorder that has an effect on how red blood cells carry oxygen to the body. Normal red blood cells are rounded, but when a person has sickle cell disease, their red blood cells look like a crescent moon. The name “sickle cell” comes from the crescent shape of the cells.  Sickled cells have a hard time moving through the blood vessels. They can get stuck and block blood vessels, which stops the oxygen from getting through. This is a painful disorder that may also cause harm to certain bones, organs, and muscles. Having the disease means a lifelong battle against the health problems it can cause, like pain, anemia, stroke, and infections.  However, people are able to have a good quality of life by learning how to manage the disease.  (Source: www.webmd.com
CAUSES: Sickle cell disease does not just develop; it is inherited from a parent.  However, a child has to inherit two sickle cell genes, one from each parent.  If a child carries the gene from only one parent, then they do not have sickle cell disease, just the sickle cell trait that they could pass on.  (Source: www.webmd.com)   
TREATMENT: Early treatment includes daily antibiotics from two months to five years of age in order to prevent infections.  Managing pain is a big part of treatment for sickle cell disease.  Parents can plan for a painful event ahead of time by creating a pain management plan with the child’s doctor. Some people may need regular blood transfusions to lower the risk of stroke and treat anemia.  (Source: www.webmd.com
NEW TREATMENT: Bone marrow transplants are usually used as a last-line of defense against cancer, especially leukemia.  However, in the past ten years, non-cancerous diseases have gone from making up about 15 percent of all pediatric bone marrow transplants to 35 percent. In the mid-90s, transplants were found to be successful for patients suffering from severe sickle cell disease, but only if their tissue matched a sibling donor.  Transplants from unrelated donors were found to be too toxic, requiring an excessive amount of chemotherapy and radiation.  Researchers tried to use smaller doses of the chemicals, called “reduced intensity,” which resulted in patient’s bodies rejecting the transplants. Dr. Shenoy and colleagues continued testing the “reduced intensity” theory.  Dr. Shenoy’s approach involved using antibodies that target proteins in the immune system and finding the amount of toxic chemicals that wipe out just enough of the body’s immunity to allow the graft to take over.  Her approach was proved successful in treating sickle cell patients and other life-threatening metabolic and bone marrow failure disorders.  About five years ago, it was adopted as the protocol for using transplants from unrelated donors to treat non-cancerous disorders.  However, finding a match is still difficult.  Dr. Shenoy says that you have a better chance at finding a cord match rather than a marrow.  Adding one more immune-suppression drug to the mix, holds promise.  Dr. Shenoy was granted approval to lead a cord-blood transplant study involving 10 research centers across the country.  However, the study has a safety restriction: three patients must have successful transplant before moving on to the next three.  (Source: http://www.stltoday.com/lifestyles)

FOR MORE INFORMATION, PLEASE CONTACT:

Jackie Ferman
Manager, Media Relations
St. Louis Children’s Hospital
(314) 286-0304
Jferman@bjc.org

If this story or any other Ivanhoe story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Marjorie Bekaert Thomas at mthomas@ivanhoe.com.



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