Corner Office: Uprooting the Foundation

"Old-fashioned." "Social entrepreneur." "Venture philanthropist." "Philantrocapitalist."

A lot of phrases—some positive, some negative—are being bandied about these days as those of us who work in the nonprofit medical research sector seek to actualize our missions. Well-meaning commentators needlessly snipe back and forth while espousing their analyses of the pros and cons of either the traditional nonprofit model or those more aligned with a business-oriented approach.

Often, the heart of the matter is lost during this war of words. Medical research foundations, especially those specializing in rare disease research, are focused on ensuring that safe and effective treatments and cures reach those living with the disease. These patients and their families seek answers to their problems now—not 20 years from now, but today.

The steps a foundation takes to achieve its goals are likely to be as individualized as the diseases and disorders each confronts. Rare diseases and disorders, for example, are at a disadvantage when it comes to receiving high levels of federal funding or strong interest from the pharmaceutical industry. With a perception of less public demand, it is all too easy for research into rare diseases to become a low priority. A low priority to everyone, that is, except those who are affected.

At Children's Tumor Foundation, we work to find cures for the approximately one in 3,000 people living with neurofibromatosis (NF), a title that encompasses three distinct genetic disorders (NF1, NF2 and schwannomatosis). NF can cause tumors to grow along various types of nerves and, in addition, can affect the development of non-nervous tissues such as bones and skin. It can cause tumors to grow on or in the body, and it manifests itself differently in every single patient—from blindness in one, to deafness in another, to intense pain or even possibly cancer.