"When I started in 2001, no one was interested in Muckle-Wells, " recalls UCSF dermatologist Kieron Leslie.
Two months later Fishman hired rheumatologist Wright, who proposed testing Novartis' own antibody in Muckle-Wells.
Regeneron tested its own drug in 47 Muckle-Wells patients, and it helped all of them.
Regeneron's drug is given weekly and could be approved for Muckle-Wells and its milder cousin this year.
This month Novartis begins a follow-up trial in up to 40 Muckle-Wells patients.
Hoffman at UC, San Diego in 2001 pinpointed the gene mutation that causes Muckle-Wells and the inherited overreaction to drafts.
Souther's disorder is the milder cousin of Muckle-Wells syndrome, and the causes of both were unknown until a a few years ago.
In 2003 British researchers tested Amgen's interleukin-blocking rheumatoid arthritis drug, Kineret, in two Muckle-Wells patients who had not been helped by other drugs.
Wright proposed testing the drug in patients with a disease so rare that some of his superiors had never heard of it: Muckle-Wells syndrome.
Novartis' promising Muckle-Wells drug--its antibody against interleukin-1, which it first aimed at arthritis--was one of the first drugs to benefit from Fishman's new testing regimen.
Yancopoulos, Regeneron's chief scientist, says he heard about the possibilities in treating Muckle-Wells and made an "impassioned plea" to Novartis to test Regeneron's drug for that malady.
And though the company earlier had spurned a biotech firm's proposal to jointly study Muckle-Wells, Wright convinced his bosses that the obscure disorder was the perfect test-bed for the new compound.
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