We got told "we provide the picks and shovels" as most of the "heavy lifting either has been or is done by others" -
FINALLY we get to see what that means:
A fully funded Phase 2 trial in Osteoarthritis, in which others (not us) have spent their (not our) funds on (pre-) clinical research, providing results that highlight the use of MSCs in that particular disease target.
We now step in and provide the "picks and shovels" to NHMRC to do the "heavy lifting".
Osteoarthritis is the last frontier of blockbuster diseases with no treatment
- Most common form of joint disease and the leading cause of disability for people greater than 65 years of age
- OA is a progressive disease strongly correlated with bone marrow edema lesions (BMEL), affecting the entire joint, including synovial inflammation, cartilage loss and bone remodelling
- Blockbuster market – 31m Americans have been diagnosed with OA - ~10% of the total population
- Significant cost – OA currently costs the US economy ~US128+ billion per annum
- Growing crisis – Due to an aging population and high obesity rates the number of OA sufferers in the US is expected to exceed 67m (116% growth) by 2030
- Significant limitations, in efficacy, tolerability and safety of available treatments for patients with moderate – severe OA
- Opioid Epidemic – The US and Australia are experiencing unprecedented opioid addiction and overdoses. The FDA is highly supportive of new, safe and effective, non-opioid treatments for pain
A recent article highlights why NHMRC is willing to do the "heavy lifting" and fund "one of the largest MSC trials ever":
"A proof-of-concept clinical trial conducted in Korea showed promising safety and efficacy results of adipose-derived MSCs to treat OA. Patients showed reduced pain, improved function of the joint and in the high-dose patient cohort, and regeneration of hyaline-like cartilage suggesting the disease-modifying effects of MSCs when injected into the affected joint [70]. Another pilot study by Orozco et al. demonstrated significant improvement in the pain and functional improvement of up to 65% to 78% in chronic OA patients when treated with bone marrow-derived MSCs, as compared with the conventional treatment methods [71]. Cartilage mapping by T2 MRI showed evidence of improvement in the good cartilage quality, i.e., hyaline-like cartilage, and significant decrease in the poor cartilage quality, i.e., fibrocartilage. The same group conducted a pilot clinical trial examining the safety and efficacy of MSC as a novel treatment of intervertebral disc disorder [72]. After a 1-year follow-up, the primary end point of pain and functional improvement was met in approximately 85% of the cases and no adverse event was observed; the water content was significantly improved in the treated disc and patient-reported significant improvement in the quality of life index [72]. Furthermore, the phase I dose-escalation trial to treat severe OA of the knee by using adipose-derived MSCs to treat patients with symptomatic and severe OA of the knee with single-articular injection of autologous adipose-derived MSCs also showed significant improvement in patients after six months of follow-up [73]. These results showed that the treatment was safe and well tolerated by all patients.
Adipose-derived MSCs to treat patients with joint disease also act as a precursor to treat degenerative OA. Osteochondritis dissecans is a joint disorder pertaining to articular cartilage and chondral defects resulting in damage to the articular cartilage and underlying bone. Adipose-derived MSCs have been reported to have disease-modifying effects in a clinical case series published recently [74]. This study showed regeneration of the lost cartilage and significant reduction of pain and improvement in mobility (Figure 4) [74].
These evidence-based clinical outcomes strengthen the model for treatment of OA with MSCs (Figures 4 and 5). The results of these trials provide an exciting and promising long-term relief for OA patients and herald a new paradigm for the treatment of chronic and debilitating OA and other degenerative conditions. Intriguingly, several hundred clinical trials globally have been registered in the past 10 years, but only a handful of results from these trials are published. Therefore, there is a need for more clinical trial data to be released from the completed trials to further support and develop this novel model of treatment."
(20min delay)