Huntington's, for which there is no cure, is caused by the loss of neurons due to a toxic protein made by a tainted gene. The idea behind the new treatment is to stop at least some of that protein's production so that it cannot damage the brain.
The treatment would use a device made by Medtronic that is already implanted in more than 250,000 patients to treat chronic pain and spasticity. The device features a catheter connected to a drug pump that's surgically implanted into the abdomen. The pump pushes drugs through the device and into the fluid around the spinal cord. In the case of the Huntington's RNAi work, the system is adapted to deliver liquids directly into the brain tissue.
"To create pressure, it actively pumps the drug into the brain, and that pressure really moves the drug into the brain and further away than the drugs would otherwise go based on diffusion," says Lothar Krinke, vice president and manager of Medtronic's deep brain stimulation projects.
In a study published earlier this year, the researchers showed that the device can distribute the siRNA to around six cubic centimeters of brain tissue in a rhesus monkey. The results of the study suggest the treatment was safe over 28 days of infusion and showed that the protein product of the Huntington's-type gene in the monkeys was nearly halved, says Krinke.
Medtronic is currently leading the effort to push the device-drug treatment into the clinic. Although the company will not say when it anticipates initiating clinical trials, the work has been funded by CHDI, a nonprofit foundation focused on developing cures for Huntington's.
Alynlam, Huntington's, RNA, RNAi, siRNAView the Original article
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