by Childrens Hospital Boston staff on June 9, 2011
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. Full story »
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.”
Did you hear? This week Children’s launched a brand new website focusing on its Stem Cell Research program and how stem cell research has already impacted the lives of sick children. Thrive featured a first-person account by a family recalling the challenges they faced in trying to help their son. Full story »
The Stem Cell 101 videos page features Len Zon, MD, and George Daley, MD, PhD, the leaders of the Stem Cell Program, talking about what stem cells are, how they’re used in the lab and the work they’re doing to bring real cures to patients’ bedsides using stem cells. Full story »
As a long-time supporter of stem cell research, I’m proud to announce the launch of a new Children’s Hospital Boston Web site that we hope will demystify the science of stem cells and answer some of the public’s questions about them. For the past three and a half years, my wife, Patti, and I have served as co-chairs of Children’s Hospital Boston’s Stem Cell Task Force because we believe that stem cells hold incredible promise for the future of health care. During this time I’ve gotten to know Len Zon and George Daley, the two physician-scientists who head up the hospital’s Stem Cell Research Program, and I believe that the work they are doing will revolutionize health care. Full story »
So where does the science of embryonic stem cells stand after a decade of political wrangling? A lot of exciting basic research is being done with embryonic stem cells, says Len Zon, a stem cell researcher at Children’s Hospital in Boston. But using stem cells for therapy?
“I think that’s still a ways off,” Zon says. “Although there are some studies that the FDA is considering, I think we still have to figure out how to make these cells in a more efficient and effective way, and I think that’s going to take awhile. You have to remember that the stem cell field is only 10 years old at the moment.”
Zon points out that it’s frequently two decades or more before new medical technologies find their way into patients.
by Childrens Hospital Boston staff on December 2, 2009
Stem cell researcher Willy Lensch
By M. William Lensch, PhD, from the Stem Cell Program at Children’s Hospital Boston
At just a little after 12:30 p.m. EST today we reached the end of a very long road. That was when the NIH announced that the first human embryonic stem cell lines (hESC) had been approved for federal funding eligibility under the rules put forth in President Obama’s executive order from earlier this year. A small group of us here in George Daley’s lab were listening in to the NIH press conference over the speaker phone. I couldn’t help but clap my hands and cheer! A lot of us have worked toward this moment for a long time. Full story »
The National Institutes of Health today announced 13 new government-approved embryonic stem cell lines that scientists can get NIH funding to work with. The newly approved lines–11 of which were developed right here at Children’s–were derived from embryos donated by couples undergoing in-vitro fertilization, and all meet the NIH’s strict ethical standards requiring informed consent from donor couples.
With more lines available, and additional approvals expected to come soon, the pace of stem cell research is expected to pick up — and with it, a better understanding of human diseases and better therapies for treating them.
Read researcher Willy Lensch’s first-person post about what this means to the field of stem cell research – and to him personally.
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By Jonathan Kraft, 
