Down to the bone

A fusion protein that ferries a healthy version of a bone-related enzyme gone awry has shown early clinical success in treating a rare bone disorder with no known therapy, researchers reported earlier this month at the Endocrine Society's linkurl:annual meeting;http://www.endo-society.org/endo/ in Washington, DC. The drug -- which is essentially a protein-based enzyme delivery mechanism -- could open the door to treatments of other skeletal disorders that have so far been deemed untreatable. Ra

Elie Dolgin
Jun 28, 2009
A fusion protein that ferries a healthy version of a bone-related enzyme gone awry has shown early clinical success in treating a rare bone disorder with no known therapy, researchers reported earlier this month at the Endocrine Society's linkurl:annual meeting;http://www.endo-society.org/endo/ in Washington, DC. The drug -- which is essentially a protein-based enzyme delivery mechanism -- could open the door to treatments of other skeletal disorders that have so far been deemed untreatable.
Radiographic signs of childhood (left)
and adult (right) hypophosphatasia

Image: NIH
"This is probably the most promising approach so far" for metabolic bone disorder therapies, linkurl:Sundeep Khosla,;http://mayoresearch.mayo.edu/staff/khosla_s.cfm an endocrinologist at the Mayo Clinic in Rochester, Minn., who was not affiliated with the work, told __The Scientist__. The new therapy targets a rickets-like disease called linkurl:hypophosphatasia;http://ghr.nlm.nih.gov/condition=hypophosphatasia (HPP), which is caused by a single mutation in the gene coding for an enzyme known as tissue non-specific alkaline phosphatase, or TNSALP....

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