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Gene Therapy Provides Relief for Painful Diabetes Complication

new gene therapy for pdn painful diabetes

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Scientists Discover Potential Treatment for PDN

Twenty to 25 percent of people with diabetes suffer from painful diabetic neuropathy (PDN). This neurological condition causes them to experience pain in their lower limbs and fingertips due to abnormal signals triggered by damaged nerve cells. This can lead to continuous numbness, injuries, chronic foot ulcers and even amputations. While there is no existing treatment, pain relievers and other medications can improve symptoms, often with unwelcome side effects. Now, results of a new clinical study from Northwestern Medicine may offer some relief from PDN.

Scientists at Northwestern Medicine led a phase two, placebo-controlled study to test a new gene therapy called VM202. In the study, neither the subject nor researchers knew which patients received injections of the placebo and which received human hepatocyte growth factor. Growth factor is a protein that keeps nerve cells alive, healthy and functioning. Scientists believe regenerating nerve cells with a growth factor could help reduce pain for people with PDN.

Participants received two doses in a two-week time span and reported their symptoms over the following nine months. Of the 84 participants, those who received low-dose rounds reported more than 50 percent reduction in their symptoms, with virtually no side effects. Those participants also had a greater reduction in pain than those receiving a high dose or placebo.

The Northwestern Medicine team, led by Jack Kessler, MD, the Ken and Ruth Davee Professor of Stem Cell Biology in Neurology, is planning future studies to test the therapy at a phase three level and to investigate if the therapy can regenerate damaged nerves and reverse the neuropathy. The scientists hope this will lead not only to a treatment that reduces the pain of PDN, but one that increases function as well.

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