Stories about: Stem cell

From Bahrain to Boston for very early onset IBD care

20150723_GassenIBDVEO-16[1]During a recent visit to Boston Children’s Hospital, three-year-old Gassen Boabed quietly entered the waiting room of the hospital’s Inflammatory Bowel Disease Center.

With Mom and big brother in tow, the tiny toddler, boasting a pretty pink headband and nail polish to match, sat at a child-sized table, picked up crayons and started coloring. She was at ease, and her surroundings were familiar.

For the past year and a half, Gassen, a native of Bahrain, a small island country east of Saudi Arabia, has been receiving treatment at Boston Children’s for a rare and debilitating condition called very early onset (VEO) inflammatory bowel disease (IBD). 

The condition, which affects infants and children under 5, causes severe abdominal pain and bloody diarrhea, and it slows growth. The cause(s) of VEO-IBD remain unknown but likely include a combination of genetic and environmental factors.

“Boston Children’s has been taking very good care of Gassen and us as well,” says Gassen’s mother Manal, with the assistance of an interpreter from the hospital’s Interpreter Services Department. “The way they have been dealing with her case has been excellent.”

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Sickle Cell Awareness Day

It’s World Sickle Cell Awareness Day, so today we bring back the story of Maryam Idan, a young Iraqi girl and sickle cell disease (SCD) survivor. We say “survivor” because last year Maryam had a stem cell transplant—the only cure currently available for SCD, and a complicated and risky one at that. Read on to learn more about Maryam’s story and life.

Maryam and her parents

Born in Baghdad in 2002 without access to advanced medical care, Maryam Idan stood little chance of receiving anything more than the most basic treatment for her sickle cell disease (SCD). By the time Maryam was a baby, her parents, Basim and Janin Al-Zoubaidi, had already lost three of their children to the blood disorder, so they knew the condition’s telltale signs. “When she started showing the same symptoms as her siblings, we immediately thought the worst,” says her father. “We knew there was treatment in America, but we had no idea if we would have the means to get there.”

An inherited disease, SCD causes a child’s red blood cells to malform and become stiff, taking on the shape of a sickle, like the letter C. These abnormally shaped blood cells can stick together and interrupt healthy blood flow throughout the body. This can cause a host of problems that can affect nearly every major organ, but it affects every patient differently. “Some kids don’t have pain or complications until they’re teens, while others suffer strokes and extreme pain and need blood transfusions when they are very young,” says Leslie Lehmann MD, director of the Stem Cell Transplant Program at Dana-Farber/Children’s Hospital Cancer Center (DFCHCC).

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Changing Shape

Maryam and her parents

Born in Baghdad in 2002 without access to advanced medical care, Maryam Idan stood little chance of receiving anything more than the most basic treatment for her sickle cell disease (SCD). By the time Maryam was a baby, her parents, Basim and Janin Al-Zoubaidi, had already lost three of their children to the blood disorder, so they knew the condition’s telltale signs. “When she started showing the same symptoms as her siblings, we immediately thought the worst,” says her father. “We knew there was treatment in America, but we had no idea if we would have the means to get there.”

An inherited disease, SCD causes a child’s red blood cells to malform and become stiff, taking on the shape of a sickle, like the letter C. These abnormally shaped blood cells can stick together and interrupt healthy blood flow throughout the body. This can cause a host of problems that can affect nearly every major organ, but it affects every patient differently. “Some kids don’t have pain or complications until they’re teens, while others suffer strokes and extreme pain and need blood transfusions when they are very young,” says Leslie Lehmann MD, director of the Stem Cell Transplant Program at Dana-Farber/Children’s Hospital Cancer Center (DFCHCC).

Like Maryam, most kids with SCD experience breathlessness, delayed growth, extreme fatigue and pain in their bones from the time they’re young. Kids with SCD also have weaker immune systems, making them susceptible to infections, and they’re at a greater risk for strokes, blindness and fatal iron-overload—another dangerous blood complication. In developed nations, where symptoms can be treated with medication, patients often still only survive until their late 40s. For patients like Maryam in developing countries, even the most basic treatment for these symptoms isn’t usually available, and death at a young age is very common.

The only cure for SCD is a stem cell transplant when the patient is young, similar to a treatment that someone with cancer would undergo. It involves the complete destruction of the immune system (as well as the sickle cells) through chemotherapy, then rebuilding the person’s immune system using healthy stem cells from a matched donor.

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Protecting the fertility of childhood cancer survivors

By Tom Ulrich. A version of this story originally appeared in Vector, Children’s science and innovation blog

While many childhood cancers are readily curable, those cures can come at a cost to future fertility. (Wikimedia Commons)

Thanks to advances in medicine, 75 percent of children currently diagnosed with cancer will live to see adulthood. This is extremely welcome news of course, but with it comes new questions about what adult life holds for survivors of childhood cancers. As science is now discovering, the therapies that are so effective at saving children’s lives can also occasionally lead to problems down the road (called the late effects of cancer treatment.)

Some of the more common concerns surrounding late effects of cancer treatment have to do with its effects on fertility, which can be quite harsh. “There’s a huge segment of the pediatric oncology population that’s at risk for infertility when they grow up,” says Richard Yu, MD, PhD who works on male infertility in Children’s Hospital Boston’s department of Urology.

The problem is hardly gender specific. “It’s as though cancer treatment pushes the ovaries further down the age curve,” says Sara Barton, a fertility specialist at Brigham and Women’s Hospital who collaborates with Lisa Diller, MD clinical director of the Dana-Farber/Children’s Hospital Cancer Center (DF/CHCC). “So while a woman who has survived childhood cancer may be 20 years old, her ovaries act like they’re 35 or 40.”

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