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)