ISTO’s NuQu Technology: The “Magic Bullet” for Cartilage Regeneration?
Company CEO spoke with ODT about the future of the firm’s technology and more.
“We’re all struggling to find the magic bullet,” said George Muschler, M.D., an orthopedic surgeon at the Cleveland Clinic. He was speaking at the Clinic’s Medical Innovation Summit 2012 held October last year, on the topic of cartilage regeneration.
Vague laws and very young research make the field of tissue regeneration difficult to navigate, but dynamic. St. Louis, Mo.-based orthobiologics company ISTO Technologies Inc. has been working since 2002 to perfect cartilage regeneration, which is particularly problematic. Finding stem cells (a term that Muschler pointed out is much too broadly defined) that can regenerate cartilage reliably without fusing with and turning into hard bone down the line has proved very difficult. Recent research has shown that children younger than one year of age have much more of a cell population capable of regenerating cartilage than anyone else.
“It regenerates cartilage that not only looks like cartilage two years from now, but years from now does not turn into bone,” said Muschler. “It keeps regenerating itself.”
ISTO’s CEO Mitch Seyedin, Ph.D., spoke with Orthopedic Design & Technology
about his company’s award-winning cartilage regeneration technology, and how it has been changing lives. NuQu, the company’s cell-based technology platform for cartilage regeneration, is a minimally invasive surgical (MIS) treatment for lumbar and discogenic back pain. A Phase II trial for the product began in the fourth quarter of 2012; though currently only in trial for spinal applications, Seyedin said he hopes to see it applied to knees and shoulders as well.
“Cartilage regeneration is the holy grail of orthopedics,” explained Seyedin. “Cartilage covers all joints, and from [ISTO’s] perspective this includes spinal discs. As we get older, our cartilage doesn’t repair itself. We like stem cells but we want cells that we don’t have to worry about turning into [hard] bone.”
When it comes to cartilage tissue donation, allogeneic solutions (tissues from donors rather than the patient) are preferred over autologous technology (tissue from the patient) for several reasons, not the least of which is the extreme benefit of juvenile tissue. According to Seyedin, cells that are younger than one year old have at least a 100-fold difference in repair and regeneration capacity. Other benefits of allogeneic tissue include: The need for only one surgery instead of two; the availability of an off-the-shelf product; economies of scale benefits; and the creation of a platform technology—such as ISTO’s—that can potentially be leveraged into additional applications.
“Laying down of cartilage tissue is almost like contact lenses,” said Seyedin. “We can set it in fluid, it has a two month shelf life, we can send it out, [physicians] can clean the defect and glue tissue to the defect.”
NuQu is aimed at the early treatment of moderate to severe back pain. It targets patients that have not responded to conservative therapy, and ISTO considers it an alternative to a surgical approach along the continuum of care. Since it is minimally invasive, it can be performed as an outpatient procedure.
“It’s done through a percutaneous application with a protein application,” explained Seyedin. “For spinal applications, we try for MIS with 2D (two-dimensional) fluoroscopy. This is for patients that haven’t responded to conservative therapy for six months to two or three years.”
NuQu’s Phase I trial had 15 enrolled patients, and all subjects have 24 months of follow-up visits. According to ISTO, the primary outcome has been safety. There have been no “serious and unexpected adverse events.” The secondary outcome has been preliminary efficacy, or “statistically significant” improvement observed in disability, pain, and general health. Based on the outcomes at 12 months follow-up, authors of an analysis of the trial results conclude that no serious and unexpected safety concerns have emerged, and that the treatment results “are promising and warrant further investigation in a prospective, randomized, double-blinded, placebo-controlled study design.”
“Given the limited treatment options available today for patients suffering from chronic low back pain, NuQu potentially represents a unique and innovative technology that could eliminate a number of surgical procedures and improve quality of life for a sizable patient population,” said Domagoj Coric, M.D., lead clinical investigator for the project and a practicing neurosurgeon at Carolina Neurosurgery and Spine Associates and chief of neurosurgery at Carolinas Medical Center, both in Charlotte, N.C.
The Phase II trial is randomized against saline as a placebo.