Enzyme Replacement Therapy Success in Treating Hypophosphatasia

There has been a success in enzyme replacement therapy which shows promise for the future. Researchers at the St. Louis Shriners Hospital for Children and Washington University School of Medicine have used ENB-40 to improve bone health, muscle strength, and mobility in subjects with hypophosphatasia. Hypophosphatasia is an inherited disease which causes very low levels of the enzyme alkaline phosphatase. This leads to improper mineralization and weak bones. The researchers used a treatment known as ENB-40 in which they injected a form of purified alkaline phosphatase in children with hypophosphatasia three times a week for six months. Similar studies had been done earlier by others on mice genetically designed to not have the gene which codes for alkaline phosphatase. Levels of alkaline phosphatase increased to normal in the blood as well as parathyroid hormone levels which are responsible for bone mineralization. Bones were visibly healthier on x-rays and muscle strength had increased improving the children's mobility. The study reported that at the beginning the children could barely walk and due to the treatment they were significantly more active like other normal children.

I thought it was interesting that there were such successful results, especially since there haven't been any effective treatments despite our knowledge of the disease for quite some time now. This means great things for not only treating hypophosphatasia but also for enzyme replacement therapy in general.

Source:

http://www.biomedicalblog.com/a-success-for-enzyme-replacement-therapy/40900/