A Pony for A Cure

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On January 27, in Phoenix, Arizona, ten-year-old Andrew gave up his beloved miniaturehorse, 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 heswallowed his sorrow and donated his pony to the JDRF’s gala auction because hebelieved “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, aprivate biotechnology company, to fund a phase II/III clinical trial of teplizumab, acompound owned by MacroGenics. It’s part of what the JDRF calls its“Industry Discovery and Development Partnership program.” This programallows the JDRF to partner with (that is, give money to) private, for-profitbusinesses 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 todestroy islets, so it may actually preserve beta cell function in newly diagnosedpatients. MacroGenics acquired the rights to the antibody in 2005, after a majorpharmaceutical company dumped it for financial reasons. There are only 3 millionpeople in the U.S. with type 1 diabetes, so spending money to develop teplizumab didnot “execute against shareholder value.” And it was going nowhere withMacroGenics too, until the JDRF stepped in.

The Charity’s Charities

The JDRF began helping out private business because it wearied of seeing the fruits ofits funding repeatedly stall out in academia. It wants diabetes therapies out in theworld where they can do some good. Knowing that big pharma isn’t interested ininvestments without big paybacks, the JDRF decided to step in as foster parent of someof the treatments owned by smaller companies. It sees itself as a nurturer ofcompounds that have big promise but a fragile hold on life, orphans that just need alittle boost to get started.

The JDRF has partnered with about 14 private companies so far, for a total of aboutfive percent of its research budget. In October 2006, it announced that it would giveSangamo BioSciences Inc., of Richmond, Calif., $3 million for a phase II trial of adrug for diabetic neuropathy. It is paying $4 million for a phase II trial byTransition Therapeutics Inc., of Toronto, of a drug that might regenerateinsulin-producing cells, and $3.5 million for a phase III trial by TolerRx Inc., ofCambridge, Mass., of another antibody that might preserve beta cells from autoimmuneattack. The JDRF believes that none of these drugs would ever see the light of daywithout its intervention.

Zig Zag

Back in Arizona, Zig Zag was auctioned off for $6000. As it turned out, Zig Zag wentto bidders who were so touched by Andrew’s sacrifice that they returned hisminiature horse to him. Now Andrew has vowed to use Zig Zag as a therapy pony to cheerhospital 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 afor-profit drug may not have been what Andrew envisioned when he donated his miniaturehorse. But in the complex world of biopharm, the line between disease charities andfor-profit drug companies is no longer clearly drawn. The JDRF considers its moneywisely 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 somethingnew.

Sources: The JDRF
The Wall Street Journal
The Arizona Republic

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