Many years ago we were the first physicians on earth to enter the world of stem cell back treatment by injecting stem cells into low-back discs. We eventually learned what did and didn’t work and licensed our technology to a small publically traded company called BioRestorative Therapies (BRTX). That group spent about 18 months performing extensive preclinical trials using our disc stem cell technology and was just granted permission by the US FDA to begin in phase 2 trials (skipping phase 1). I’d like to share with you some history of how all of that came about as we’re excited here at Regenexx that one of our procedures is entering FDA trials.
The BRTX Press Release
BioRestorative Therapies Receives FDA Clearance to Initiate Phase 2 Clinical Trial for the Treatment of Patients with Degenerative Disc Disease
MELVILLE, N.Y., February 8, 2017 – BioRestorative Therapies, Inc. (“BRT” or the “Company”) (BRTX), a life sciences company focused on stem cell-based therapies, today announced that it has received clearance by the U.S. Food and Drug Administration (FDA) to commence a Phase 2 clinical trial using its lead cell therapy candidate, BRTX-100, to treat chronic lower back pain due to degenerative disc disease related to protruding/bulging discs.
The Phase 2 clinical trial is a 72 patient, randomized, double-blind, controlled, multi-center study designed to evaluate safety and efficacy of a single dose of BRTX-100 in treating chronic lower lumbar disc disease. BRTX-100 will be administered via intradiscal injection into one disc of a subject with chronic lumbar disc disease and whose pain is not responsive to conservative treatment measures (e.g., oral medication, epidural injections and physical therapy). The primary goal of the treatment is to both reduce pain and increase function in these patients.
In January 2017, the Company had submitted an Investigational New Drug Application (IND) to the FDA to obtain clearance to commence this clinical trial using BRTX-100. BRTX-100 is a product formulated using a patient’s own cell population (autologous), which consists of hypoxic (low oxygen) cultured mesenchymal stem cells (MSCs) that are optimized for specific use for a non-surgical, conservative intradiscal procedure that can be performed in a physician’s office.
“We are excited to be able to begin our clinical development of BRTX-100 with this Phase 2 clinical trial,” said Mark Weinreb, President and Chief Executive Officer of BioRestorative Therapies. “This treatment has the potential to positively impact millions of Americans suffering from chronic lumbar disc disease as an alternative to surgery. We believe that this technology can be truly transformative and addresses a large market underserved by current therapies.”
Our Experience with Stem Cell Back Treatment
When we began our stem cell injection work in 2005, it was due to animal research showing that mesenchymal stem cells injected into a degenerated disc could significantly heal that disc. That was exciting enough to get us into regenerative medicine when no other physician in the US even knew what it was. However, when we met with experts who were treating athletic horses at the time, they had no insights in how to deal with low-back discs as horses didn’t get such treatments in 2005. Hence, we began treating both low-back discs and joints in the same way. The low-back-disc patients were almost uniformly a complete failure, while the joint stem cell treatments were working. We then went back to the drawing board on discs and dramatically changed how we culture expanded cells and our technique for implanting them.
In 2007–2008 we had a stem cell back treatment breakthrough. A novel culture method for stem cells and our new procedure that we used to place the cells into the right spot began to show that we could reduce the size of disc bulges on follow-up MRI. By 2013 we had enough experience to license this technology to a new publically traded company called BioRestorative Therapies. The company worked diligently to raise funds and then ultimately began pre-IND work with the FDA in 2014. Their most recent press release is above.
The fact that BRTX got FDA approval to skip phase 1 trials is both a testament to the incredibly detailed preclinical animal work they were able to perform as well as the fact that this autologous cell therapy has been used in humans since 2006. The paper I covered on this blog a few weeks ago allowed the company to show the FDA that the issue with this therapy wasn’t likely to be safety.
The upshot? I’m incredibly proud to have pioneered a stem cell back treatment that is finally beginning phase 2 FDA trials. Congrats to Mark Weinreb and Franciso Silva and the rest of the BRTX team! My colleague Greg Lutz, MD, from H.S.S. has been chosen to lead their clinical research team for these trials, and I have full confidence that Greg will be able to bring “my baby” home to full FDA approval!