What has worked?

Familial dysautonomia is a debilitating pediatric disorder characterized by symptoms such as cardiovascular problems, increased sensitivity to pain and temperature, gastrointestinal dysfunction and increased occurrence of pneumonia. If left untreated, most children with the disease […]

Anyone who has seen a commercial for the popular drug Celebrex knows of its effectiveness in osteoarthritis, a disease characterized by bone damage, chronic pain, and decreased range of motion. The drug’s mechanism of action […]

Metformin, a common diabetes drug that has been manufactured at low cost for years, has a long record of safety, effectiveness, and limited side effects in diabetics. In the mid-2000s an interesting discovery was made and […]

Another cancer treatment success story involves the repurposing of all-trans retinoic acid (ATRA), a chemical compound historically used in medications that treat severe acne. Though it does not have anti-cancer properties when used alone, ATRA has been combined […]

Repurposed treatments have also been discovered for sufferers of Fragile-X syndrome, a genetic disorder that leads to cognitive disabilities. Many patients with this disease experience anxiety and mood-related symptoms. Minocycline, a common antibiotic used to treat acne and […]

The anti-fungal compound itraconazole was developed in the 1980s, and suppresses fungal growth by inhibiting membrane function in fungal cells (1). But a 2007 study showed that the drug also has potent anti-cancer properties (2). Further […]

Dose adjustments to existing treatments
The effects of certain drugs can vary greatly based on dosage, making them ideal candidates for repurposing research. Methrotrexate was developed as a chemotherapy drug in the 1950s and has since been administered at […]

Dangerous originally, repurposed safely
One reason for the low success rate of novel drug development is the high percentage of adverse side effects found in late-stage clinical trials. However, failed drugs can be repurposed for different diseases with […]

Repurposing generics for rare diseases
Drug development is a business, and those who synthesize new compounds often do so for large populations to maximize payoff. Unfortunately, this means that very rare diseases are often not the […]

Advancements in treatment have also been made by combining new drugs with repurposed old drugs that can enhance therapeutic effects or specificity. Chloroquine, a drug that has historically been used to treat malaria, has recently been combined with a new […]