It's said that the philanthropic goals of younger individuals often differ from that of older generations in that they favor a more hands-on approach to their giving. They want to exercise more control over their charitable contributions. This approach was apparent in a recent article about "do-it-yourself drug development" (see: Do It Yourself Drug Development — With Some Help). Below is an excerpt from it:
The effort by two families to buy and develop a drug that holds promise in treating Duchenne muscular dystrophy...is the result of an innovative new model set up to support the burgeoning phenomenon of do-it-yourself drug development. Before the Seckler and Wicka families bought halofuginone, a drug that showed promise in experiments done with Duchenne mice, they set up Dart Therapeutics. Dart is funded by foundations set up by the two families, and run by Eugene Williams, a drug industry veteran with 25 years’ experience, including seven years at Genzyme. Mutual acquaintances led the Secklers and Wickas to Williams in 2010, when the families were discussing how to advance drug development in Duchenne muscular dystrophy. The disease is rare, with only around 20,000 new cases a year, and both families were increasingly frustrated that despite raising many millions to help fund research and experimental drug development programs, promising compounds frequently went nowhere. They wondered if, with expert advice, patients and advocates in some cases might be able to develop drugs themselves. That idea led to the formation of Dart in June, 2010. Williams, who is the chairman, put together a “virtual company” ...comprised of former senior executives and consultants expert in developing drugs and getting them through the FDA approval process. The idea, says Tracy Seckler, whose son Charley has Duchenne ...was to create a vehicle to identify potential compounds, make deals, and develop the drugs....In cases where Dart decides it’s worth it to buy the drug — which is what happened when it took a look at halofuginone — a separate company will be formed. (In the case of halofuginone, the company is called Halo Therapeutics.)
To summarize this complicated narrative, the Seckler and Wicha families have a special interest in Duchenne muscular dystrophy; a son of the Seckler family has the disease. The families established a pharmaceutical company, Dart Therapeutics, that is funded by their family foundations. They then hired a veteran pharmaceutical executive to run Dart with a small staff, The company functions as a virtual organization that hires consultants to assist it in discovering and bringing suitable drugs to market. They then purchased a drug, halofuginone, that shows promise in treating Duchenne mice that will be managed by yet another company, Halo Therapeutics.
Fascinating story. Benefactors from a previous generation with an interest in Duchenne might have channeled their contributions through a charity with a focus on neurologic diseases. But in such a setting, progress toward the development of a new drug would have been slow and tangible results perhaps rare. You might say that drug development is a slow process best left to the professionals. However and in the case of Dart, the families engaged the services of veteran pharma executive to help them pursue their goals.
Here's some more information about orphan drug development from the Wikipedia (see: Orphan drug):
Since the market for any drug with such a limited application scope would, by definition, be small and thus largely unprofitable, government intervention is often required to motivate a manufacturer to address the need for an orphan drug. Critics of free market enterprise often cite this as a failure of free market economic systems. The intervention by government on behalf of orphan drug development can take a variety of forms:
- Tax incentives.
- Enhanced patent protection and marketing rights
- Clinical research financial subsidization.
- Creating a government-run enterprise to engage in research and development
In the case of Dart, federal initiatives to encourage orphan drug development comes in the form of favorable tax treatment for family foundations. Obviously, the creativity of the families behind these foundations played a major role. This is a great model for a more active form of philanthropy in the future.
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