BioRestorative Therapies (NASDAQ:BRTX) is a clinical-stage biotech company that is developing cell-based therapies to treat chronic lumbar disc disease and metabolic conditions such as obesity and diabetes. The company is focusing on developing BRTX-100, its lead therapeutic candidate for the treatment of patients with chronic lower back pain caused by degenerative disc disease. According to experts, up to 80% of the population will experience back pain at some point. This technology involves using the patient's own stem cells to regenerate damaged discs in the lower back, potentially reducing pain and improving mobility. 

Previous clinical studies have highlighted the potential impact of the disc's harsh microenvironment on cell viability, which may lead to non-efficacious outcomes or adverse reactions. In order to address this harsh microenvironment, BRTX-100 has been formulated using stem cells that are manufactured using low oxygen to mimic the low oxygen environment found in the disc. BRTX-100 is currently being tested in a phase 2, randomized, double-blinded and controlled study. A total of up to 99 eligible subjects will be enrolled at up to 16 clinical sites in the United States.

Although the data presented is still blinded and at an early stage, it is noteworthy that the Visual Analog Scale (VAS), Oswestry Disability Index (ODI), Roland Morris Disability Questionnaire (RMDQ) and Functional Rating Index (FRI) collected at 26 and 52 weeks after injection indicate a positive trend compared to the baseline. These pain and function questionnaires are used to determine if there have been improvements at weeks 2, 12, 26, 52, and 104 after treatment. In addition to safety, improvements as measured by changes in pain and function play a crucial role in the evaluation of BRTX-100 by the U.S. Food and Drug Administration (FDA) for potential approval of a Biologics License Application (BLA).

“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,” shared Lance Alstodt, Chief Executive Officer of BioRestorative.

BRTX-100 involves the use of autologous hypoxic mesenchymal stem cells and autologous platelet lysate, making it a personalized treatment option. If the company is successful with FDA approval, the candidate could be revolutionary for patients struggling with chronic back pain and become the first orthobiologic approved for disc-related pain. BRTX-100 is unique in its potential to offer patients a less invasive and quicker therapeutic application. BRTX-100 involves a minimally invasive bone marrow and blood harvest procedure, with a collection time of only 20 minutes and a recovery time of approximately one day. 

Other companies competing in the cell-based biologics or chronic pain treatment space include Mesoblast (NASDAQ:MESO), Brainstorm Cell Therapeutic  (NASDAQ:BCLI) and Longeveron (NASDAQ:LGVN).