Orphan drugs find a home
Jan Hermsen | May/June 2012 Issue
They’re called “orphan drugs”—medicines for rare disorders, such as Alexander disease, fatal familial insomnia and ACTH deficiency. They are effective, but the pharmaceutical industry, foreseeing insufficient profits from the small patient populations, mothballs them. Dick van Bekkum, an 85-year-old physician and radiologist, doesn’t like to see a good drug die. As the founder of Cinderella Therapeutics, he wants to test and bring these medicines to the marketplace.
For drug development, Cinderella makes use of its own knowledge and donated capital. Outside help for research is also essential, but van Bekkum has few concerns: “There are enough people who are sympathetic toward the projects and will invest knowledge or money. And they will do so out of emotional considerations or from certain beliefs.”
Drug development costs a great deal of time and a significant amount of money. Cinderella is currently researching a new form of radio-immunotherapy for recurrent brain tumors, along with projects on seven other diseases. If van Bekkum succeeds where big-pharma firms don’t even try, that will be a Cinderella story, indeed.
For more information: www.cinderella-tx.org