Japanese Firm, IU Unite For Research Into Renal Diseases
INDIANAPOLIS -A well-known Japanese company will be seeking therapeutic
discoveries for renal insufficiency from a gene discovered by Indiana
University researchers. The licensing agreement provides exclusive rights
for use of FGF23 to Kirin Brewery Co. Ltd. for development of therapeutic
and diagnostic products.
The gene, isolated in 2000, on the short arm of chromosome 12 is responsible
for autosomal dominant hypophosphataemic rickets (ADHR), a rare disorder
only known to exist in a handful of families worldwide. Only about 30
people in the Untied States are known to suffer from the rare, inherited
form of rickets, which can cause leg deformity, tooth abscesses, bone
pain and fractures. The kidneys of individuals with the FGF23 mutation
cannot retain phosphorus, an important element for maintaining bone integrity.
“Research into this very rare disease may lead to potential therapeutics
for a very common disease,” said Michael J. Econs, M.D., professor
of medicine and of medical and molecular genetics at the IU School of
Medicine. Dr. Econs, along with Kenneth E. White, Ph.D., assistant professor
in the IU Department of Medicine, made the discovery of FGF23 in collaboration
with the German laboratory of Tim M. Strom, M.D.
Researchers at Kirin’s Pharmaceutical Research Laboratories independently
identified FGF23 as a causative factor of tumor-induced osteomalacia,
an adult form of rickets. It exhibits some similar symptoms to ADHR such
as renal phosphate wasting.
“This license makes our patent position strong with our original
invention related to the FGF-23 protein,” said Toshifumi Mikayama,
Ph.D., general manager of Kirin’s Pharmaceutical Research Laboratories.
“FGF-23 will give us important clues to elucidate the mechanisms
of phosphate regulation in the human body and we believe, based on the
discovery, we will be able to bring therapeutic benefits to patients suffering
from kidney or bone diseases.”
Researchers were excited about the identification of FGF23 not only from
the standpoint that it helps a small number of ADHR patients, but also
because it may lead to therapies for individuals with more common diseases
such as moderate renal insufficiency. Due to decreased kidney function,
patients with renal insufficiency retain excessive amount of phosphorus,
which is detrimental to their bones and vessels.
Kirin researchers have developed a test to measure FGF23 concentrations
in the blood. They are developing a commercially available test that can
be used for future research and diagnostic endeavors involving a range
of disorders that potentially involve FGF-23.
Funding for the original research came from the National Institute of
Arthritis and Musculoskeletal and Skin Diseases, part of the National
Institutes of Health. IU’s Advanced Research and Technology Institute
(ARTI) handed the arrangements for granting the license.
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Media Contact: Mary Hardin
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