Ilaris Marks New Drug Development Approach for Novartis
Illaris, approved by the U.S. Food and Drug Administration on June 18, may never have seen the light of day if Dr. Tim Wright, head of translational medicine at Novartis, hadn’t decided to “revive a drug that had been languishing in the company's labs,” Kerry Capell reported for BusinessWeek.
Novartis, a Swiss pharmaceutical company, had previously tested the drug for rheumatoid arthritis, but had come up with disappointing results. Known as canakinumab, the drug works by blocking an immune system protein known as interleukin-1, according to BusinessWeek.
Scientists may have never touched the drug again, but Wright stepped in and, along with his boss, Dr. Trevor Mundel, pushed for new trials. They wanted to try canakinumab on Muckle-Wells, an illness so rare that it “afflicts just a few thousand patients worldwide.”
It worked. “Within five hours of a single injection, the patient's symptoms had visibly improved,” Capell wrote. “Within a day they were gone, and by the end of the week the disease was barely detectable in the blood.”
The FDA has approved the drug for two forms of cryopyrin-associated periodic syndrome, or CAPS—Muckle-Wells and familial cold auto-inflammatory syndrome. It is the first treatment approved for patients as young as four who suffer from the diseases. Symptoms can include conjunctivitis, fatigue, joint pain, headaches, rash and fever, reports Reuters, and long-term consequences can be serious or even fatal.
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Although scientists once believed that “master” genes controlled the growth of cells that cause these conditions, the new study found that hundreds of genes interact, and that the variations within this genetic network explain the different ways in which people develop diseases. Scientists believed the study would be a key step in gaining a better understanding of cancer, multiple sclerosis and other autoimmune conditions in which the body’s immune system mistakenly turns against the body’s own tissue.