Stories about: Stem cell

Bursting the SCID-X1 bubble

The Caceres family

Agustin Caceres’s baptism was the only time his family was allowed close to him. But even then, anyone who came in contact needed to wear masks, gloves and gowns. After the ceremony, Agustin went back into isolation, along with his mother Marcela, who stays with him and only comes out for meals.

Agustin spent much of his life separated from the world because he has a form of X-linked Severe Com­bined Immunodeficiency—or SCID-X1—better known as “bubble boy disease.” It affects only boys, leaving their bone marrow unable to make T-lymphocytes, the white blood cells that fight germs and infection.

To keep Agustin safe, his father, Alberto, and his four-year-old brother, Jeremias, live in a separate bedroom. At one point Jeremias even left his nursery school to make sure he couldn’t bring home any infections his baby brother might catch.

Unfortunately, this is not the Caceres’s first experience with SCID-X1; their first son was also born with the disease and died from it before he was even 5 months old. So when Jeremias was born healthy, the family banked stem cells from his umbilical cord blood to benefit future treatment, should they have another son born with the disease. At first doctors were hopeful Jeremia’s stem cells could be used to treat Agustin, but the boys’ tissues weren’t compatible. Without interven­tion, Agustin was likely to die from a bacterial or viral infection before his first birthday.

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Children's researcher honored by TIME Magazine

In September 2010, the journal Cell Stem Cell released important findings by Children’s Hospital Boston researcher Derrick Rossi, PhD. Rossi is an investigator at the Immune Disease Institute/Program in Cellular and Molecular Medicine at Children’s, Assistant Professor in the Department of Pathology at Harvard Medical School, and a member of the Principal Faculty of the Harvard Stem Cell Institute.

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Injunction halts federally funded stem cell research

A federal district judge made a surprising ruling yesterday, blocking federal funding for stem cell research, overturning policies established by the Obama administration in 2009.

Under President Bush, federal dollars could be used to fund research on a few existing stem cell lines, but couldn’t be used to develop or study new lines. The Obama administration opened government funding for research on new lines, until Chief Judge Royce Lamberth’s controversial ruling yesterday.

Andres Trevino was devastated when he heard about Lamberth’s injunction. Trevino, a Mexico City native, first came to America in 1999 to seek treatment for his son, Andy, who was sick with a rare and often fatal genetic mutation called NEMO. Thanks to Children’s doctors and medical knowledge and procedures attributed to stem cell research, Andy’s life was saved. (See a video, below, of Andres talking about yesterday’s injunction.)

Trevino and his wife were so grateful that they donated embryos with NEMO mutation to Children’s to create stem cell lines that could be used to study the disease. But under Lamberth’s injunction, even with the Trevinos’ blessing, federal dollars cannot be used in any capacity to further research using their donated stem cell line. According to Lamberth, current policies on federal funding for stem cell research violate a 1996 law preventing federal money from being used to finance research in which an embryo is destroyed.

The ruling is under review by many in the legal and scientific communities—if and how long it will stand remains uncertain—but for the time being stem cell research has been set back even beyond the limitations created by the Bush administration.

“This ruling means an immediate disruption of dozens of labs doing this work since the Obama administration made its order,” George Daley, MD, PhD, director of Children’s Hospital Boston’s Stem Cell Transplantation Program said to the New York Times. “Our lab will have to return to the old mode of keeping human embryonic stem cell research separate from everything else, which means slower progress.”

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New hope for gene therapy

Imagine that instead of drugs or surgery, you could use genes to treat or even  prevent diseases. That’s the promise of gene therapy, an experimental technique where a gene is inserted into a patient’s cells to replace a non-functioning or disease carrying gene.

genetherapy2While gene therapy could potentially cure a variety of diseases, the development of gene therapy strategies against SCID-X1, better known as “bubble boy disease,” has been stalled since 2005, when five boys in Europe with SCID-X1, developed leukemia during clinical trials. Now, a new international trial, sponsored in the United States by Children’s Hospital Boston’s David Williams, MD, director of Translational Research, hopes to revive this potentially life-saving treatment.

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