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by decreasing the hyperactive firing of damaged neurons, or it could promote “good remodeling” of nerve cells, Sandrock says.
Of course, this all has a long, perilous road to travel to generate proof of the concept in human clinical trials. Only one in 10 drugs that enters clinical trials ever becomes an approved product, and this program sounds riskier than the usual drug that’s following a well-trod path. It’s been a year since the Nature Neuroscience paper, and Biogen isn’t yet in the clinic, although it says it is in discussions with the FDA about putting together an application to start trials. “We’re close to the going to the clinic,” Sandrock says.
Some fundamental things still need to be answered to see if this can become a practical drug. The product is being designed to be delivered intravenously, or through an injection patients can get under the skin, Sandrock says. The animal study called for six injections over an 11-day period, and then saw a lasting benefit for the duration of the study, six months. The company is still discussing what sort of dosing schedule might be required for people, Sandrock says.
Other big drugmakers have their eyes on regenerative medicines. San Diego-based Ceregene recently failed in a mid-stage clinical trial of a gene therapy that was made to stimulate growth of neurons to treat Parkinson’s disease. Swiss drug giant Novartis has tested an antibody that interferes with Nogo, a protein that puts the brakes on natural development of new neurons after a spinal cord injury. Another pharma giant, Johnson & Johnson, is pursuing a competing approach to neublastin for neuropathic pain, although Biogen is thought to be ahead in development, Sandrock says.
Most notably, Menlo Park, CA-based Geron (NASDAQ: GERN) got permission from the FDA in January to start the world’s first clinical trial of an embryonic stem cell therapy for people with spinal cord injuries. Biogen’s treatment may never see this kind of intense media attention because it doesn’t depend on the controversial cells. But the goal behind it—regenerating the damaged nerve tissue to restore the lost function—is similar.
“You may not need to come in to the doctor forever,” Sandrock says. “We got long-lasting restoration of sensory function in preclinical studies. That’s the potential value of a drug that’s disease modifying.”