Drugs for autoimmune diseases like multiple sclerosis, type 1 diabetes, and rheumatoid arthritis provide relief from symptoms but don’t address causes. Researchers now hope to change that with an emerging class of vaccines made from DNA that shut down the immune cells that go awry in these diseases. Early results have been so promising that human trials for the first treatment-a multiple sclerosis therapy that made paralyzed mice walk again-will begin early next year.
In MS patients, rogue immune cells attack the nerve covering called the myelin sheath, leading to numbness, weakness, cognitive problems, and eventually paralysis. There’s no existing cure; a leading drug, called beta-interferon, regulates the immune system to reduce the severity of attacks but can carry severe side effects. “Treatments today for these types of autoimmune diseases are basically blunt instruments,” says John Walker, CEO of Bayhill Therapeutics, the Palo Alto, CA, startup developing these DNA vaccines. “We’re interested in taking much more of a rifle-shot approach.”
Two engineered DNA molecules make up the vaccine; both are taken up by specialized “first responder” immune cells, which are thus transformed into MS-fighting machines. One DNA molecule encodes a protein found in the myelin sheath; the first-responders make this protein, which acts as bait for the rogue immune cells. Once this trap is sprung, the second DNA molecule goes to work. It encodes a protein that switches the rogue cells from a destructive mode to a protective mode.
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