ADDRESSING DISPARITY
ONE LAB ’ S TRASH …
Drug failures yield new hope for neglected diseases .
BY HEATHER BUSCHMAN , PH . D . ’ 08
A NEW DRUG ’ S JOURNEY from laboratory discovery to a pharmacy shelf takes about 14 years and costs upwards of $ 2 billion dollars . If that wasn ’ t staggering enough , consider this : more than 90 percent of drugs in development fail to make it from bench to bedside .
That means for every FDA-approved drug , pharmaceutical companies probably have 10 more — thousands altogether — collecting dust . Most are shelved because they ’ re ineffective at treating the intended disease ; so while proven safe in humans , the best use has yet to been found . That ’ s why UC San Diego researchers are now taking a second ( or third ) look at many of these drugs and repurposing them to treat new diseases .
“ Drug repurposing is a faster , cheaper and safer approach to finding new treatments for disease ,” says James McKerrow , M . D ., Ph . D . ’ 73 , dean of the Skaggs School of Pharmacy and Pharmaceutical Sciences at UC San Diego . With the groundwork already laid , new therapies based on repurposed drugs could benefit patients much sooner than the typical 14 years — saving and improving more lives , and offering an efficient way to combat neglected diseases .
McKerrow recently received a National Institutes of Health award granting access to SAR114137 , a drug the pharmaceutical company Sanofi initially developed to treat chronic pain . McKerrow ’ s team will now test the drug ’ s efficacy in treating Chagas disease , a neglected tropical disease and the leading cause of heart failure in Latin America .
McKerrow has a hunch that SAR114137 could work for Chagas disease because of its similarity to a previous drug that successfully treated infected animals , but couldn ’ t be validated in humans . Since SAR114137 has passed safety studies in humans , it ’ s already many steps ahead of its predecessors .
“ Chagas affects more than 8 million people and is responsible for more than 10,000 deaths worldwide each year . Yet there are currently no FDA-approved therapies ,” McKerrow says . “ We call Chagas a ‘ neglected ’ disease because most pharmaceutical companies aren ’ t interested in developing new therapies for a disease that mainly affects poor communities . This is one area that ’ s especially likely to benefit from drug repurposing .”
DOPAMINE KETAMINE SURAMIN ANTAGONISTS AURANOFIN VERTEPORFIN TAMOXIFEN
OLD DRUG Common anesthetic and pain-killer |
OLD DRUG Treatment for African sleeping sickness |
OLD DRUG Class of drugs used as antipsychotics |
OLD DRUG Intended to treat rheumatoid arthritis |
OLD DRUG Treats abnormal blood vessel formation in the eye |
OLD DRUG Treatment for types of breast cancer |
NEW VISION Treating depression cases unresponsive to conventional anti-depressants |
NEW VISION Could hold great possibility for treating Autism Spectrum Disorder |
NEW VISION Possess tumor-killing activity against glioblastoma , an aggressive brain cancer |
NEW VISION Clinical trials have begun in Bangladesh to treat parasitic infections |
NEW VISION Has been shown to suppress the growth of ocular melanoma |
NEW VISION Could give the immune system a boost against drug-resistant bacteria ( See pg . 14 ) |
David Feifel , M . D ., Ph . D ., professor of psychiatry |
Robert Naviaux , M . D ., Ph . D ., professor of medicine |
Clark Chen , M . D ., Ph . D ., associate professor of neurosurgery |
Sharon Reed , M . D ., professor of pathology and medicine |
Kun-Liang Guan , Ph . D ., professor of pharmacology |
Victor Nizet , M . D ., professor of pediatrics |
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