Stem cells really are giving hope where none existed before.
05 Mar 2009
Stem cell research helps children suffering from rare Niemann-Pick Type C disease
Niemann-pick-type-cNiemann-Pick Disease is one of a group of lysosome storage diseases that affect metabolism and that are caused by genetic mutations. There are three most commonly recognized forms, they are Niemann-Pick Types A, B and C.
According to the National Niemann-Pick Disease Foundation Web site. Niemann-Pick Type C disease is a genetic disease that affects metabolism of cholesterol and other lipids. Excessive amounts of cholesterol accumulate in the liver and spleen and other lipids accumulate in the brain.
Type C Niemann-Pick Disease has about 500 cases diagnosed worldwide. It is believe, however, that the number of people affected by NPC is higher, but diagnostic difficulties do not allow an accurate assessment of the occurrence rate.
There are approximately 1,200 cases of NPA and NPB world wide with the majority being Type B or an intermediate form.
There is no cure or treatment for the disease, and it is always fatal, but doctors and researchers are working to find drugs that slow the process, said Dr. Alan Moy, CEO of Cellular Engineering Technologies and director of the John Paul II Stem Cell Research Institute in Iowa City.
Peyton Hadley, 11, and his sister, Kayla, 8, have both been diagnosed with Niemann-Pick Type C. The Hadley family came from Oregon to Iowa City in December to work with Moy. Peyton and Kayla Hadley both had fat removed at Mercy Hospital in Iowa City and stem cells were isolated and grown at Cellular Engineering Technologies at the University of Iowa Oakdale campus.
Researchers are hopeful that the stem cells derived from fat would be used to screen drugs that may control dreadful Niemann-Pick Type C disease along with other rare diseases.
05 Mar 2009
Stem cell research helps children suffering from rare Niemann-Pick Type C disease
Niemann-pick-type-cNiemann-Pick Disease is one of a group of lysosome storage diseases that affect metabolism and that are caused by genetic mutations. There are three most commonly recognized forms, they are Niemann-Pick Types A, B and C.
According to the National Niemann-Pick Disease Foundation Web site. Niemann-Pick Type C disease is a genetic disease that affects metabolism of cholesterol and other lipids. Excessive amounts of cholesterol accumulate in the liver and spleen and other lipids accumulate in the brain.
Type C Niemann-Pick Disease has about 500 cases diagnosed worldwide. It is believe, however, that the number of people affected by NPC is higher, but diagnostic difficulties do not allow an accurate assessment of the occurrence rate.
There are approximately 1,200 cases of NPA and NPB world wide with the majority being Type B or an intermediate form.
There is no cure or treatment for the disease, and it is always fatal, but doctors and researchers are working to find drugs that slow the process, said Dr. Alan Moy, CEO of Cellular Engineering Technologies and director of the John Paul II Stem Cell Research Institute in Iowa City.
Peyton Hadley, 11, and his sister, Kayla, 8, have both been diagnosed with Niemann-Pick Type C. The Hadley family came from Oregon to Iowa City in December to work with Moy. Peyton and Kayla Hadley both had fat removed at Mercy Hospital in Iowa City and stem cells were isolated and grown at Cellular Engineering Technologies at the University of Iowa Oakdale campus.
Researchers are hopeful that the stem cells derived from fat would be used to screen drugs that may control dreadful Niemann-Pick Type C disease along with other rare diseases.