Stories about: hematopoietic stem cell transplants

Souped-up fish facility boosts drug discovery and testing

closeup of zebrafish-20150526_ZebraFishCeremony-60The care and feeding of more than 250,000 zebrafish just got better, thanks to a $4 million grant from the Massachusetts Life Sciences Center to upgrade Boston Children’s Hospital’s Karp Aquatics Facility. Aside from the fish, patients with cancer, blood diseases and more stand to benefit.

From a new crop of Boston-Children’s-patented spawning tanks to a robotic feeding system, the upgrade will help raise the large numbers of the striped tropical fish needed to rapidly identify and screen potential new therapeutics. It’s all part of the Children’s Center for Cell Therapy, established in 2013. We put on shoe covers and took a look behind the scenes. (Photos: Katherine Cohen)

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A double-shot of good news for SCID: Promising transplant and gene therapy data

Hematopoietic hierarchy aging blood cell hematopoietic stem cell blood disorder Derrick Rossi
Blood-forming hematopoietic stem cells (top) give rise to all blood and immune cell types. In children with SCID, the steps leading to immune cells are broken.

In the world of fatal congenital immunodeficiency diseases, good news is always welcome, because most patients die before their first birthday if not treated. Babies with severe combined immunodeficiency disease, aka SCID or the “bubble boy disease,” now have more hope for survival thanks to two pieces of good news.

Transplants are looking up

First came a July paper in the New England Journal of Medicine (NEJM) by the Primary Immune Deficiency Treatment Consortium. This North American collaborative analyzed a decade’s worth of outcomes of hematopoietic stem cell transplant (HSCT), currently the only standard treatment option for SCID that has a chance of providing a permanent cure.

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Can blood cells be rebooted into blood stem cells?

Hematopoietic hierarchy blood development stem cells
The classic hematopoietic hierarchy. What if we could turn those arrows around?

Think, for a moment, of a cell as a computer, with its genome as its software, working to give cells particular functions. One set of genetic programs turns a cell into a heart cell, another set creates a neuron, still another a lymphocyte and so on.

The job of controlling which programs get booted up, and when, falls in part to transcription factors—genes that act like molecular switches to turn other genes on and off.

Derrick Rossi, PhD, spends a lot of his time thinking about transcription factors. A stem cell and blood development researcher in Boston Children’s Hospital’s Program in Cellular and Molecular Medicine, Rossi believes that transcription factors hold the power to achieve one of the most sought-after goals in regenerative medicine: producing, from other cell types, transplantable hematopoietic stem cells (HSCs).

“There are about 50,000 HSC transplants every year,” Rossi explains, noting that the success of a transplant is highly dependent on the number of cells a patient receives from her donor. “But HSCs only comprise about one in every 20,000 cells in the bone marrow.

“If we could generate autologous HSCs from a patient’s other cells,” he continues, “it could be transformative for transplant medicine and for our ability to model diseases of blood development.”

As they reported April 24 in Cell, Rossi and his collaborators have taken a significant step toward that goal: Using a cocktail of eight transcription factors, they reprogrammed mature mouse blood cells into what they have dubbed induced HSCs (iHSCs).

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New research on blood stem cells takes root

Word cloud of words associated with hematopoietic stem cells and blood development.
The demand for hematopoietic stem cell transplants is rising. But how can we get more cells? (Text from Bryder D, Rossi DJ and Weissman IL. Am J Pathol 2006; 169(2): 338–346.)
You need a lot of hematopoietic stem cells to carry out a hematopoietic stem cell transplant, or HSCT. But getting enough blood stem cells can be quite a challenge.

There are many HSCs in the bone marrow, but getting them out in sufficient numbers is laborious—and for the donor, can be a painful process. Small numbers of HSCs circulate within the blood stream, but not nearly enough. And while umbilical cord blood from newborn babies may present a relatively rare but promising source for HSCs, a single cord generally contains fewer cells than are necessary.

And here’s the rub: The demand for HSCs is only going to increase. Once a last resort treatment for aggressive blood cancers, HSCTs are being used for a growing list of conditions, including some solid tumor cancers, non-malignant blood disorders and even a number of metabolic disorders.

So how do we get more blood stem cells? Several laboratories at Boston Children’s Hospital and Dana-Farber/Boston Children’s Cancer and Blood Disorders Center are approaching that question from different directions. But all are converging on the same end result: making more HSCs available for patients needing HSCTs.

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Blood stem cell transplants for metabolic disorders of the brain?

Bone marrow being extracted for a hematopoietic stem cell transplant
A patient’s bone marrow is extracted for a hematopoietic stem cell transplant, or HSCT. Once just a last-resort treatment for cancer, HSCTs are now used for a growing list of conditions, including certain metabolic disorders affecting the brain. (US Navy/Wikimedia Commons)

The history of hematopoietic stem cell transplant (HSCT) starts with severe cancers of the blood or immune system, like relapsed leukemias or lymphomas. Today, HSCTs are no longer solely the treatment of last resort for cancer but is used to treat a growing list of pediatric and adult conditions.

Most of these are cancers and blood disorders, but in recent years, a new frontier has opened up for HSCT: treatment of metabolic diseases, in particular, ones that affect the function of the brain.

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