(Press-News.org) Babies who are born with severe combined immunodeficiency (SCID) can be successfully treated with a transplant of blood-forming stem cells, according to experts led by Memorial Sloan Kettering's Richard J. O'Reilly, MD, a world-renowned pioneer in the development of transplant protocols. Their review will be published in the July 30 issue of the New England Journal of Medicine.
SCID is a group of inherited disorders that cause the immune system to severely malfunction. When this breakdown occurs, babies no longer have the ability to fight off routine infections because their natural, built-in defense system has been damaged. If undiagnosed or left untreated, SCID is almost always fatal within the first year of life.
A review of more than 240 patient cases found transplants to be quite effective, especially when performed early in life. Of those children receiving transplants within three and a half months after birth, 94 percent were alive five years later. The best results, not surprisingly, were seen after transplant from "matched sibling" donors. But among patients who didn't have a matched sibling, overall five-year survival rates were quite high — 77 to 93 percent — if they were transplanted in the first three and a half months of life.
"This confirms that transplants for SCID work well in very young children, but it also shows that any child with this disease can be treated with a high likelihood of a cure with a transplant from a parent or unrelated donor, not just a matched brother or sister," explained Dr. O'Reilly, Chair of MSK's Pediatrics Department and Chief of the Pediatric Bone Marrow Transplant Service. "Irrespective of the transplant approach used, if the child is transplanted early — without infection — you will have an extraordinarily good result."
But a critical factor continues to be ensuring that the child has no infection at the time of transplant. "This really illustrates the importance of more widespread screening for SCID so that doctors can intervene immediately," said Dr. O'Reilly. Currently, only 21 states have implemented testing as part of routine newborn screening, including New York, California, and Florida. "By the time symptoms of infection appear, the circumstances for transplant are often less favorable."
MSK has played a pioneering role in advancing stem cell transplantation. The first successful transplant of stem cells from an unrelated donor was performed at MSK in 1973 specifically to treat a child with SCID. In 1981, MSK introduced T cell depletion, a groundbreaking procedure for improving the success of unmatched transplants. Most of the important benchmarks in the development of transplantation and cell therapies have come from the studies of these relatively rare cases involving children who have a lethal genetic immune system disorder, said Dr. O'Reilly.
"Great strides have been made in a relatively short time, allowing children with SCID not only to survive but also to lead normal lives," Dr. O'Reilly added. "Paul Simon had that song [The Boy in the Bubble] where he says, 'These are the days of miracles and wonder.'…. Our research provided a way to cure babies in the bubble. We don't need the bubble anymore."
About Memorial Sloan Kettering Cancer Center
We are the world's oldest and largest private cancer center, home to more than 13, 000 physicians, scientists, nurses, and staff united by a relentless dedication to conquering cancer. As an independent institution, we combine 130 years of research and clinical leadership with the freedom to provide highly individualized, exceptional care to each patient. And our always-evolving educational programs continue to train new leaders in the field, here and around the world. For more information, go to http://www.mskcc.org.
Transplantation shown to be highly effective in treating immune deficiency in children
ELSE PRESS RELEASES FROM THIS DATE:
Journal supplement details progress in African medical education
Medical education in sub-Saharan Africa is being revitalized and expanded through a U.S.-funded effort that is dramatically increasing enrollment, broadening curricula, upgrading Internet access and providing cutting-edge skills labs and other technologies. In the first substantial publication by participants of the $130 million Medical Education Partnership Initiative (MEPI), more than 225 authors detailed progress being made at the African institutions. Their reports are in a supplement being published today by the journal Academic Medicine. Begun in 2010, MEPI is ...
Resistance to key malaria drug spreading at alarming rate in Southeast Asia
WHAT: Resistance to artemisinin, the main drug to treat malaria, is now widespread throughout Southeast Asia, among the Plasmodium falciparum (P. falciparum) parasites that cause the disease and is likely caused by a genetic mutation in the parasites. However, a six-day course of artemisinin-based combination therapy—as opposed to a standard three-day course—has proved highly effective in treating drug-resistant malaria cases, according to findings published today in the New England Journal of Medicine. The research was conducted by an international team of scientists ...
Biologists describe mechanism promoting multiple DNA mutations
DNA mutations—long known to fuel cancer as well as evolutionary changes in a living organism—had been thought to be rare events that occur randomly throughout the genome. However, recent studies have shown that cancer development frequently involves the formation of multiple mutations that arise simultaneously and in close proximity to each other. These groups of clustered mutations are frequently found in regions where chromosomal rearrangements take place. The discovery, published in the journal Cell Reports, may one day lead to new cancer therapies, according to ...
Classic Lewis Carroll character inspires new ecological model
Inspired by the Red Queen in Lewis Carroll's Through the Looking Glass, collaborators from the University of Illinois and National University of Singapore improved a 35-year-old ecology model to better understand how species evolve over decades to millions of years. The new model, called a mean field model for competition, incorporates the "Red Queen Effect," an evolutionary hypothesis introduced by Lee Van Valen in the 1970s, which suggests that organisms must constantly increase their fitness (or ability to survive and reproduce) in order to compete with other ever-evolving ...
Diverticulitis patients reveal psychological, physical symptoms long after acute attacks
UCLA researchers interviewed people with diverticulitis and confirmed that many suffer psychological and physical symptoms long after their acute illness has passed. For the study, published this week in the peer-reviewed journal Quality of Life Research, a UCLA team led by Dr. Brennan Spiegel interviewed patients in great detail about the symptoms they experience weeks, months or even years after an acute diverticulitis attack. Their striking findings add to growing evidence that, for some patients, diverticulitis goes beyond isolated attacks and can lead to a chronic ...
Finding quantum lines of desire
Groundskeepers and landscapers hate them, but there is no fighting them. Called desire paths, social trails or goat tracks, they are the unofficial shortcuts people create between two locations when the purpose-built path doesn't take them where they want to go. There's a similar concept in classical physics called the "path of least action." If you throw a softball to a friend, the ball traces a parabola through space. It doesn't follow a serpentine path or loop the loop because those paths have higher "actions" than the true path. But what paths do quantum particles, ...
Tidal forces gave moon its shape, according to new analysis
The shape of the moon deviates from a simple sphere in ways that scientists have struggled to explain. A new study by researchers at UC Santa Cruz shows that most of the moon's overall shape can be explained by taking into account tidal effects acting early in the moon's history. The results, published July 30 in Nature, provide insights into the moon's early history, its orbital evolution, and its current orientation in the sky, according to lead author Ian Garrick-Bethell, assistant professor of Earth and planetary sciences at UC Santa Cruz. As the moon cooled and ...
Target growth-driving cells within tumors, not fastest-proliferating cells
BOSTON –– Of the many sub-groups of cells jockeying for supremacy within a cancerous tumor, the most dangerous may not be those that can proliferate the fastest, researchers at Dana-Farber Cancer Institute report in a paper appearing in an advance online publication of the journal Nature. The findings have important implications for the treatment of cancer with precision medicines, the study authors explained: Doctors need to ascertain which cell subgroups are truly driving the tumor's growth and metastasis and select drugs that target the critical genes within those cells. ...
ALMA finds double star with weird and wild planet-forming discs
BOWLING GREEN, O.—From movies to television, obesity is still considered "fair game" for jokes and ridicule. A new study from researchers at Bowling Green State University took a closer look at weight-related humor to see if anti-fat attitudes played into a person's appreciation or distaste for fat humor in the media. "Weight-Related Humor in the Media: Appreciation, Distaste and Anti-Fat Attitudes," by psychology Ph.D. candidate Jacob Burmeister and Dr. Robert Carels, professor of psychology, is featured in the June issue of Psychology of Popular Media Culture. Carels ...
Innovative scientists update old-school pipetting with new-age technology
CAMBRIDGE, Mass. (July 30, 2014) A team of Whitehead Institute researchers is bringing new levels of efficiency and accuracy to one of the most essential albeit tedious tasks of bench science: pipetting. And, in an effort to aid the scientific community at large, the group has established an open source system that enables anyone to benefit from this development free of charge. Dubbed "iPipet," the system converts an iPad or any tablet computer into a "smart bench" that guides the execution of complex pipetting protocols. iPipet users can also share their pipetting designs ...