By Pat Anson, PNN Editor

An Australian regenerative medicine company has released positive results from a Phase III randomized trial showing that a single injection of its proprietary stem cell product can provide long-term relief for people with chronic lower back pain caused by degenerative disc disease.

Mesoblast Limited said the results are so promising it plans to meet with the U.S. Food and Drug Administration to discuss ways to accelerate approval of the drug as a treatment that reduces the use of opioid pain medication.

The company’s stem cell product -- remestemcel-L -- has been under development for several years. It uses mesenchymal precursor cells taken from the bone marrow of healthy donors to reduce inflammation by inhibiting the production of pro-inflammatory cytokines by white blood cells.

Sixty percent of the patients in the clinical trial who were injected with remestemcel-L reported minimal or no pain after 12 months. After 24 months, 54 percent reported little or no pain, with the greatest pain reduction in patients in the early stages of degenerative disc disease. Many patients also significantly reduced their use of opioids during the study period.

“The durable pain reduction for at least two years from a single administration indicates that rexlemestrocel-L has the potential to change the treatment paradigm for chronic low back pain due to inflammatory disc disease, a condition that affects as many as seven million patients across the United States and Europe, and to prevent or reduce opioid use and dependence,” Dr. Silviu Itescu, CEO of Mesoblast, said in a statement.

Over 400 patients were enrolled in the Phase III trial, which was conducted at 48 sites around the world, mostly in the United States. Although Mesoblast told physicians and patients not to change any medications during the trial, after 24 months there was a 40% reduction in opioid use in patients injected with rexlemestrocel-L. Those who were given a placebo saline injection increased their daily opioid consumption.

In a previous study of patients with chronic lower back pain who did not respond to conventional treatment, a single injection of remestemcel-L also reduced pain for at least two years.

The FDA has prioritized the development of new pain treatments that reduce the use of opioids. Although the agency has taken a dim view of some stem cell therapies as “unproven and potentially dangerous,” Mesoblast believes the FDA will be more open-minded about its rexlemestrocel-L treatment. Last year the agency approved an investigational new drug application for rexlemestrocel-L as a therapy for COVID-19.

“We now have two studies that show significant pain reduction and we’re fully prepared to have a discussion with FDA on a path forward,” said Mesoblast Chief Medical Officer Dr. Fred Grossman. “We’re going to get into discussions to see if there’s an accelerated path. Or, if we do need to do another study, we now have a very defined patient population where we see significant pain reduction.”