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Studies show promise for stem cell therapies in treating RA and chronic lower back pain



Mesoblast Ltd., a producer of cell-based therapies based in Melbourne, Australia, recently released the results of two studies that it says show promise for its stem cell products in treating rheumatoid arthritis and chronic lower back pain.

The first study involved a Phase 2 trial in patients with rheumatoid arthritis (RA) resistant to anti-tumor necrosis factor (TNF) agents. The results showed that a single intravenous infusion of the company's allogeneic cell therapy product candidate, MPC-300-IV, was well tolerated and demonstrated a durable improvement in clinical symptoms, physical function and disease activity relative to placebo over this period of follow-up.

Major advances in the treatment of RA using biologic agents have resulted in a $19 billion global market in 2016, the majority of which is due to use of anti-TNF agents. The RA population resistant to anti-TNF agents, which constitutes about one-third of patients treated with anti-TNF agents, is the fastest growing branded market segment within the global RA biologics market, and is set to grow further as multiple anti-TNF biosimilars become available.

The Mesoblast study’s primary objective was to evaluate safety and tolerability of a single intravenous MPC infusion in these biologic refractory RA patients through a 12-week primary endpoint.  Additional objectives were to evaluate clinical efficacy at the 12-week endpoint and to assess the durability of effects and safety profile through the full 52-week study.

The study recruited a total of 48 patients with active RA who were on a stable regimen of methotrexate and had an inadequate prior clinical response to at least one anti-TNF agent. Of the 48 patients, 30 (63 percent) had previously received one to two biologic agents. Patients were randomized to a single intravenous infusion of 1 million MPCs/kg, 2 million MPCs/kg or placebo. 

The study was comprised of a 12-week primary study period and a total study duration of 52 weeks. Key results over nine months were: 

  • A single intravenous infusion of Mesoblast's allogeneic "off-the-shelf" MPCs resulted in durable responses through nine months (39 weeks) 
  • The safety profile over 39 weeks was comparable among the placebo and both MPC treatment groups, with no cell-related serious adverse events
  • Both MPC doses outperformed placebo at week 39 in each of ACR20/50/70 responses, as well as by median ACR-N analysis  
  • The 2 million MPC/kg dose showed the earliest and most sustained treatment benefit

MPCs ease chronic back pain

The second Mesoblast study was a 36-month randomized, placebo-controlled Phase 2 trial of its proprietary allogeneic MPCs in patients with chronic low back pain due to intervertebral disc degeneration.

The results showed that a single intra-discal injection of 6 million MPCs resulted in meaningful improvements in both pain and function that were durable for at least 36 months.  

“The sustained benefits on pain and function over three years seen with a single injection of Mesoblast’s cell therapy have the potential to transform the treatment paradigm for chronic low back pain due to disc degeneration,” said trial investigator Hyun Bae, M.D., professor of surgery and director of education at Cedars Sinai Spine Center and director of the Spine Institute in Los Angeles. “Instead of replacing or fusing the disc, there is mounting compelling evidence that we can use this regenerative medicine to heal the disc. We are fast approaching this inflection point in the treatment of low back pain, which is particularly important in view of the epidemic of opioid abuse."

Mesoblast’s  allogeneic, “off-the-shelf” cell product candidates using mesenchymal lineage adult stem cells target advanced stages of diseases with high, unmet medical needs including cardiovascular conditions, orthopedic disorders, immunologic and inflammatory disorders and oncologic/hematologic conditions.

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