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Gene therapy could offer relief from painful diabetic neuropathy symptoms
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Treatment with a plasmid containing two human hepatocyte growth factor isoforms could offer patients with painful diabetic neuropathy significant relief from their symptoms for 3 months, according to research published in Annals of Clinical and Translational Neurology.
In a double-blind, placebo-controlled study completed by 84 patients, two low-dose rounds of the nonviral gene therapy VM202 were safe, well-tolerated and effective and could particularly benefit patients not on gabapentin or pregabalin, according to researchers.
“Those who received the therapy reported more than a 50% reduction in their symptoms and virtually no side effects,” John A. Kessler, MD, of the Davee Department of Neurology, Feinberg School of Medicine, Northwestern University, said in a news release. “Not only did it improve their pain, it also improved their ability to perceive a very, very light touch.”
Kessler and colleagues from other institutions randomized patients to low-dose (n = 31, 8 mg per leg) or high-dose (n = 36, 16 mg per leg) injections of VM202 or placebo (n = 16); divided doses were given at baseline and two weeks.
The researchers primarily looked for change in the mean pain score, based on a 7-day pain diary, with responder analysis, quality-of-life and pain measures, and intra-epidermal nerve fiber density secondary outcomes.
Patients demonstrated the most improvement with 8 mg VM202 per leg, including a reduction in the mean pain score at 3 months (P = .03); pain reduction continued, but lost significance, at 6 and 9 months. More patients experienced at least 50% pain reduction with treatment compared with placebo (48.4% vs. 17.6%).
With VM202, significant improvements were seen in the brief pain inventory for patients with diabetic peripheral neuropathy, along with the questionnaire section of Michigan Neuropathy Screening Instrument. Patients who were not receiving pregabalin or gabapentin experienced the greatest reductions in pain. No significant adverse events were attributable to VM202.
Further investigation is needed to determine if the therapy, containing a naturally occurring growth factor gene that acts to keep nerve cells healthy and functioning, could regenerate damaged nerves and reverse neuropathy.
“Right now there is no medication that can reverse neuropathy,” Kessler said. “Our goal is to develop a treatment. If we can show with more patients that this is a very real phenomenon, then we can show we have not only improved the symptoms of the disease, namely the pain, but we have actually improved function.” – by Allegra Tiver
Disclosure: The researchers report no relevant financial disclosures.
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