A Pony for A Cure
On January 27, in Phoenix, Arizona, ten-year-old Andrew gave up his beloved miniature horse, Zig Zag, to raise money for the Juvenile Diabetes Research Foundation (JDRF). He had to think about it hard; they’d been together since he was six. But he swallowed his sorrow and donated his pony to the JDRF’s gala auction because he believed “Zig Zag could help find a cure.”
Private Business Needs Your Help
On January 6, 2007, the JDRF announced that it was giving $2 million to MacroGenics, a private biotechnology company, to fund a phase II/III clinical trial of teplizumab, a compound owned by MacroGenics. It’s part of what the JDRF calls its “Industry Discovery and Development Partnership program.” This program allows the JDRF to partner with (that is, give money to) private, for-profit businesses that have the potential to develop medicines for type 1 diabetes, but are apparently too cash-strapped to do so.
For example, MacroGenic’s compound teplizumab has shown plenty of promise. It’s an antibody engineered to disarm immune T cells once they’re set to destroy islets, so it may actually preserve beta cell function in newly diagnosed patients. MacroGenics acquired the rights to the antibody in 2005, after a major pharmaceutical company dumped it for financial reasons. There are only 3 million people in the U.S. with type 1 diabetes, so spending money to develop teplizumab did not “execute against shareholder value.” And it was going nowhere with MacroGenics too, until the JDRF stepped in.
The Charity’s Charities
The JDRF began helping out private business because it wearied of seeing the fruits of its funding repeatedly stall out in academia. It wants diabetes therapies out in the world where they can do some good. Knowing that big pharma isn’t interested in investments without big paybacks, the JDRF decided to step in as foster parent of some of the treatments owned by smaller companies. It sees itself as a nurturer of compounds that have big promise but a fragile hold on life, orphans that just need a little boost to get started.
The JDRF has partnered with about 14 private companies so far, for a total of about five percent of its research budget. In October 2006, it announced that it would give Sangamo BioSciences Inc., of Richmond, Calif., $3 million for a phase II trial of a drug for diabetic neuropathy. It is paying $4 million for a phase II trial by Transition Therapeutics Inc., of Toronto, of a drug that might regenerate insulin-producing cells, and $3.5 million for a phase III trial by TolerRx Inc., of Cambridge, Mass., of another antibody that might preserve beta cells from autoimmune attack. The JDRF believes that none of these drugs would ever see the light of day without its intervention.
Back in Arizona, Zig Zag was auctioned off for $6000. As it turned out, Zig Zag went to bidders who were so touched by Andrew’s sacrifice that they returned his miniature horse to him. Now Andrew has vowed to use Zig Zag as a therapy pony to cheer hospital patients. Oddly enough, Zig Zag himself has become a cure of sorts.
As for the cure via the JDRF: Helping a private pharmaceutical company to develop a for-profit drug may not have been what Andrew envisioned when he donated his miniature horse. But in the complex world of biopharm, the line between disease charities and for-profit drug companies is no longer clearly drawn. The JDRF considers its money wisely spent on potentially useful treatments that would otherwise wither away. It’s tired of seeing that happen over and over, and it wants to try something new.
Sources: The JDRF
The Wall Street Journal
The Arizona Republic
Oct 25, 2007