Ann: Mesoblast Phase 3 CLBP Trial Completes Enrollment, page-196

@sarge17
"intent of my original post was to ascertain what people think about the role of stem cells in chronic conditions such as CLBP. "
"But do these mechanisms and actions trigger an ongoing positive action and benefit in chronic conditions such as lower back pain? I’m not so sure."

Hi Sarge17. Ultimately the result of the phase 3 trial will answer your question, however it wouldn't have got to phase 3 unless phase 2 showed promising results.

The phase 2 results (MPC-06-ID Trial) are available from Mesoblast website, but I will post relevant sections below:
What was minimum efficacy end points?
With respect to primary efficacy endpoints, the FDA has provided guidelines on how to evaluate patient response, utilizing a composite endpoint based on achieving minimally important clinical differences, or MICD, in both pain and function from baseline. Such a composite endpoint for restorative or replacement disc therapies is different than that typically used by pharmacologic agents developed solely for palliative improvement in symptoms, such as analgesics, where short term improvement in mean pain scores between groups is sufficient to support a label for short term pain reduction. The FDA and key opinion leaders, or KOLs, have deemed that for restorative or replacement disc therapies the MICD for pain reduction should be at least a 30% improvement from baseline and for functional improvement at least a 30% improvementor 10 point improvement from baseline using a 100-point functional scale.

MICD'S:
Improvement in Chronic Lower Back Pain.
Improvement in function.
Reduced need for surgical AND non surgical Interventions.
Radiographic measurements.
Composite Endpoint.

RESULTS:
Improvement in CLBP: At both 6 and 12 months, a reduction in pain from baseline of 50% or more, without any additional intervention, was seen in 59.3% of the MPC-06-ID group, 44.8% of the 18 million MPC group, 18.8% of the saline group, and 15.8% of the HA group. (HA=Hyaluronic Acid).
Statistical significance denotes the mathematical likelihood that the results observed are real and not due to chance.

Improvement in function: In line with historical FDA preference for spine fusion and artificial disc replacement marketing application approvals, a responder analysis was performed targeting at least a 15 point improvement in function through 24 months from baseline. At both 6 and 12 months, an improvement in function from baseline of 15 points or more, without any additional intervention, was seen in 50.0% of the MPC-06-ID group. 48.3% of the 18 million MPC group, 31.6% of the HA group, and 17.7% of the saline group

Reduced need for surgical AND non surgical Interventions: MPC-treated patients had a significantly reduced need for additional interventions at the treated disc level, including surgical intervention (spinal fusion, discectomy or artificial disc replacement) or injection (epidural steroid injection, rhizotomy or transforaminal injections), than saline controls. By 12 months, 25% of patients in the saline control group had undergone an additional intervention, compared with 15% of patients in the HA control group, 6.9% of patients in the MPC-06-ID group and only 3.3% of patients who received 6 or 18 million MPCs.

Radiographic measurements: This is an FDA validated measurement that has previously been used in Phase 3 trials of surgical devices for discogenic back pain. At 12 months, MPC-treated patients demonstrated a reduction in radiographically-determined translational movement of the disc, suggesting a treatment effect on disc degeneration, anatomy, and improved disc stability. The 18 million MPC group had a mean translational movement of only 1.3%, the MPC-06-ID group 2.0%, the HA group 2.5%, and the saline group 3.5%

Composite Endpoint: Based on precedent and FDA feedback from our end-of-Phase 2 meeting, we developed a composite endpoint requiring at least a 50% improvement in low back pain, 15 point improvement in ODI and no treatment intervention (surgical or injection) that we believe would be sufficient to meet FDA’s requirements for approval. Utilizing this composite endpoint in a post-hoc analysis of Phase 2 data, separation between treatment and control arms was first seen at 3 months, maximal at 6 months, and sustained for at least 12 months. More specifically, the MPC-06-ID group, the 18 million MPC group, the HA control and the saline control groups had 44.4%, 37.9%, 15.8% and 11.8% of subjects meet the composite endpoint criteria at both 6 and 12 months.
Moreover, the MPC-06-ID group had three times (3x) the proportion of patients achieving treatment success at both 12 and 24 months compared with saline controls.

All the above is now history. FDA confirmed successful results and we entered phase 3.
We are all waiting with baited breath for those phase 3 results to be revealed!

Success for this CLBP treatment is by no means a fait accompli, but with above phase 2 results, I am happy to keep my shares as a LTH. (with associated risks.) After all, this is just 1 of many imminent catalysts as is mentioned regularly in these threads.

Cheers Sarge17. Hope this has helped with your initial querie regards the effectiveness on a chronic condition like lower backpain.

Have a great weekend.
Malown1.