Stories about: stem cell transplants

There is a cure for sickle cell disease…for some

Maryam Idan (center), a young Iraqi girl with sickle cell disease, was lucky: she could be cured with a stem cell transplant. Leslie Lehmann, MD, wants to make such transplants an option for more sickle cell patients.

I was surprised when chatting recently with Leslie Lehmann, MD, clinical director of the Stem Cell Transplantation Program at Dana-Farber/Children’s Hospital Cancer Center (DF/CHCC). She turned to me and asked, “Did you know there’s been a cure for sickle cell disease for nearly 40 years?”

I had to admit that I didn’t. I’ve always thought of sickle cell—a painful and debilitating disease caused by an inherited mutation that makes red blood cells stiffen into a characteristic sickled shape—as a chronic disease to be managed, not one that could be cured.

I’m not alone in that belief. Lehmann often asks this question when she give talks for medical students, residents and other physicians. Their reaction is puzzlement, then a shaking of heads.

The cure is there, though. It’s a stem cell (aka bone marrow) transplant. The catch is that it’s not available to everyone—but for reasons that Lehmann thinks can be overcome.

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Being PRUDENT about transfusions

What time is the right time to give a transfusion? Doctors at Boston Children's are turning a fresh eye on transfusion guidelines for children. (@alviseni/Flickr)

Cancer. Trauma. Sickle cell disease. Surgery. There are many reasons why a child might need a blood transfusion, but they all share a common theme: the need to replace blood or blood products (e.g., red blood cells, platelets) that have been lost or consumed, or make up for defects that keep the body from producing them in adequate amounts.

And though transfusions can be life saving, they come with risks, such as iron overload, inflammation or disease (a very low risk, thanks to improved screening tests). And blood products are expensive and scarce—another reason to be prudent about transfusions.

“There’s little science behind physicians’ current practices when deciding when to transfuse a patient,” says Jenifer Lightdale, MD, MPH, of Boston Children’s Hospital’s Division of Gastroenterology and Nutrition. “Many doctors use criteria their mentors passed down to them, which their mentors passed down to them, and so on. But ideally, the decision should be based on evidence, not tradition.”

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From fish to people – first drug ID’d in zebrafish crosses a milestone

A recent clinical trial brings the drug FT1050 one step closer to becoming the first drug identified with the help of zebrafish (above) to make it to patients. (Soulkeeper/Wikimedia Commons)

In 2007, working with zebrafish, Leonard Zon and his team in Children’s Stem Cell Program made an unexpected discovery: That a drug originally developed to treat stomach ulcers could boost the production of blood stem cells, by about four-fold.

That drug – FT1050, a chemical variant of a fatty, hormone-like molecule called prostaglandin E2 (PGE2) – recently crossed a major milestone: the successful conclusion of a Phase I clinical trial. Led by Zon’s colleague Corey Cutler, a clinical researcher at Dana-Farber Cancer Institute, the trial aimed to determine the drug’s safety as a way of helping patients who receive umbilical cord blood stem cell transplants recover their immune function more quickly.

The trial brings the FT1050 one step closer to becoming the first drug identified with the help of zebrafish to make it to patients.

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