This news may be of interest to pregnant women. Researchers from Duke University Medical Center claim that a latest development in cord blood transplants for hereditary metabolic disorders could possibly be curative for some babies who are treated while still inside the womb.
Metabolic disorders are also known as lysosomal storage disorders. These disorders include numerous dozen rare, inherited genetic diseases marked by the deficiency of specific enzymes which the body requires in order to break down and get rid of metabolic byproducts of energy production. If left untreated, it could result in bone, brain, and central nervous system problems and early death.
Joanne Kurtzberg, MD, professor of pediatrics and pathology and director of the Pediatric Blood and Marrow Transplant Program at Duke University Medical Center stated that the novel approach seems to make use of a small, pick number of therapeutic stem cells which have been treated to accelerate and improve the process of engraftment.
Kurtzberg is known to be examining properly the method in a pilot test which is open to newly pregnant mothers. Apparently, these expecting mothers are at possibility for having children with lethal metabolic disorders.
For the past decade or so, physicians are believed to have conducted experiments with cord blood transplants after birth as a method to treat these diseases. Furthermore, in many cases, treatments appear to have been successful. Kurtzberg was of the opinion that the timing of the transplant seems to be crucial.
“The idea is to give the baby cord blood stem cells from a healthy donor that have the potential to provide healthy genes that can replace the ones that aren’t working properly in the baby’s own cells,†elucidates Kurtzberg.
She stated that generally quicker the treatment, greater the possibility that the donor cells may work. As a result transplants before the baby is born appear to be ideal.
Supposedly, the fetal transplant itself is comparatively simple. After a physician diagnoses the occurrence of one of the genetic diseases and the parents approve to treatment, donor cells are noted to be injected directly into the baby’s abdomen at approximately 12 to 14 weeks into the pregnancy.
Subsequently, there seems to be an extensive period where the transplant needs to ‘take,’ or engraft. At birth, the baby may possibly be tested in order to check if donor cells are present and if they’re already working to fix the malfunctioning genes. If not, the baby may be entitled for a conventional transplant with chemotherapy within the first few weeks of life.
Kurtzberg was of the opinion that the test appears to be open to pregnant women who are at risk of having a baby with Krabbe disease, metachromatic leukodystrophy (MLD), Pelizaeus-Maerzbacher Disease (PMD), Tay-Sachs disease, or Sandoff disease. Donor cells are believed to be manufactured by Aldagen, Inc., a biopharmaceutical company in Durham and a partner in the research.