MSC Therapy Well-Tolerated in Lumbar Disc Disease, Blinded Data Show Trends of Pain Improvement
BioRestorative has submitted an IND amendment to compare BRTX-100 to sham injection instead of saline injection.
Francisco Silva
Credit: BioRestorative Therapies
BRTX-100 autologous stem cell therapy has been well-tolerated, according to blinded data from the first few patients with chronic lumbar disc disease (cLDD) treated in a phase 2 clinical trial (NCT04042844).1
“We are thrilled with the progress of our ongoing clinical development programs. With regard to the Phase 2 study investigating the use of BRTX-100 in the treatment of cLDD, we are strongly encouraged by the preliminary data presented at ORS 2024. The preliminary clinical data shows meaningful signals in patients enrolled in the study and, importantly, no notable safety signals,” Lance Alstodt, Chief Executive Officer, BioRestorative, said in a statement.1
The data are from 3 participants that received 40x106 cells of BRTX-100 and 1 that received a saline control (3:1 randomization scheme). All adverse events (AEs) were not serious and were related to expected increases in back pain post-procedure. Two additional patients have been treated in the safety run-in and have not experienced any serious AEs.1
Although the data are blinded, investigators observed reductions in visual analog scale, Oswestry disability index, Roland Morris disability questionnaire, and functional rating index at weeks 26 and 52 from baseline. BioRestorative stated that previous clinical studies have demonstrated non-efficacy or worsening of clinical outcomes after cell therapy due to the harsh microenvironment of the disc and so these data seem to promisingly break from that trend.
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“We want to move away from the saline injection, the FDA required us to do a saline injection, it’s part of what they felt was a true control which it’s really not. You're piercing the disc, you’re injecting a substance that can and will hydrate the disc and will provide temporary relief, there’s a lot of literature showing that,” Francisco Silva, Vice President of Research and Development, BioRestorative Therapies, said during the company’s webcast. He added that BioRestorative has submitted an investigational new drug (IND) amendment to compare BRTX-100 against a sham injection rather than a saline injection.
BRTX-100 consists of hypoxic, cultured mesenchymal stem cells intended to improve blood flow in impacted areas of the body and is delivered via intradiscal injection.The phase 2 trial plans to enroll up to 99 eligible participants at up to 16 clinical sites in the United States in a 2:1 randomization scheme.
An independent data safety monitoring board previously recommended the trial continue past its safety run in without any changes in June 2023 after no dose-limiting toxicities were observed.
“The results of this in-depth safety review by an unbiased team of independent experts provides us with great confidence,” Silva said in a statement at that time.2 “The DSMB, which includes experts in chronic lumbar disc disease, has recommended that the study continue at the present dosage of cells. A DSMB recommendation is a critical step towards confirming the safety of our BRTX-100. We hope that the treatment of the next set of patients will provide further evidence that BRTX-100 is a safe and effective treatment option for patients with chronic lumbar disc disease.”
