Seeking a cure for deadly Lowe syndrome 

LAY LANGUAGE
Lowe syndrome is an uncommon genetic disease that affects the eyes, kidneys, and nervous system. People affected by the disease rarely live beyond the age of 40, with death often caused by kidney failure. Purdue University researchers are testing treatments that could reverse the symptoms.

PROBLEM
Lowe syndrome is an uncommon genetic disease that affects about one in 500,000 people; this amounts to tens of thousands of affected children worldwide. The life span of a person affected by Lowe syndrome rarely extends beyond 40 years, with death often caused by kidney failure.

SOLUTION
Purdue University researchers have developed a patented therapeutic strategy for Lowe syndrome by repurposing rapamycin and statins – drugs currently approved by the U.S. Food and Drug Administration for other conditions. In tests, Purdue researchers observed that patients’ cells started to behave as normal cells when rapamycin and statins were administered at the same dose as their original, FDA-approved uses.

PRIMARY INVESTIGATOR
R. Claudio Aguilar, College of Science

IN THE MEDIA
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INNOVATION DISCLOSURE
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LICENSING CONTACTS
Email: otcip@prf.org

MEDIA CONTACT
Email: Steve Martin // sgmartin@prf.org