NeuroScientific Biopharmaceuticals today released the results of two small studies of its StemSmart stem cell therapy in kidney-failure patients, highlighting its safety and clinical potential.
The company says its infusion therapy could become a game-changer in transplant medicine by tackling organ rejection and graft failure where current treatments fall short.
The studies have given the Perth biotech fresh firepower as it pushes a clinical and regulatory program to take on immunosuppressant and anti-inflammatory drugs in a multibillion-dollar global market. The market for kidney-related immunosuppressants alone is estimated to be US$7.2 billion (A$11 billion).
NeuroScientific’s patented StemSmart technology innovatively prepares a specific type of stem cells, mesenchymal stem cells (MSC), for use as an intravenous infusion. MSCs are essentially an “off-the-shelf” universal cell therapy, requiring no matching between donor and recipient.
The company said an historic compassionate case study involved 10 adults facing the gloomy prospect of losing their donated kidney - a condition known as treatment-refractory acute renal rejection. The donated kidneys received StemSmart infusions weekly for four weeks and were then biopsied three and 12 months later.
Eight of the ten patients retained their at-risk kidney following the therapy, with biopsies showing reduced T-cells and inflammatory markers, alongside healthy renal function. Antibodies even reduced or disappeared in patients with high levels of donor-specific antibodies - which usually have poor outcomes. The infusions were well tolerated with no infusion-related side effects.
The second trial involved 12 adults undergoing transplants of kidneys from deceased donors, turning the spotlight onto a common complication known as ischemia-reperfusion injury. This injury typically occurs after blood flow is re-established to the transplanted kidney and potentially triggers inflammation, delayed graft function and, ultimately, rejection.
Patients in this study received StemSmart within 12 hours of blood flow being restored to the donated kidney and again at day seven. The treatment proved safe, with no reported infusion-related toxicity and delivered outstanding results. Ten of the 12 patients required no dialysis at all after surgery, while the other two needed only minimal sessions. Kidney function at three and 12 months was described as excellent.
NeuroScientific Biopharmaceuticals chief scientific advisor Dr Marian Sturm said: “Resolution of acute rejection in the majority of the small group of patients facing loss of their kidney was fantastic. The anti-inflammatory and immunomodulatory properties of StemSmart MSC brought the inflammatory cell invasion of their kidney under control and resulted in a stabilisation of renal function.”
The company was quick to point out that it’s still early days. Both studies involved only small patient groups and were not designed to deliver definitive clinical results. However, together the results make a compelling case for larger trials aimed at preventing graft loss and improving long-term transplant success.
If future trials back up these results, NeuroScientific believes StemSmart could become a powerful add-on, or even an alternative, to today’s standard immunosuppressant drugs. Lifelong immunosuppressants dominate the multibillion-dollar transplant market but often trigger tough side effects and fall short of stopping graft loss over the longer-term.
NeuroScientific is continuing to review historical programs, data and published findings from other small programs since picking up the StemSmart technology through its June acquisition of private Perth-based company Isopogen for $5.1 million.
The deal has opened a range of targets for the therapy. For starters, NeuroScientific has a good lead into the inflammatory Crohn’s disease, which is forecast to be a US$13.8 billion (A$21.2B) market by 2026.
The company also has results to suggest the infusions may offer a last-line treatment for some lung disorders, which is tipped to become a massive US$33 billion (A$51 billion) market globally by 2034. With plenty more historical data still to sift through, the company is now mapping out fresh therapeutic pipelines.
NeuroScientific now has two encouraging studies in hand, a $5.1 million stem cell acquisition under its belt and billion-dollar markets in its sights, as it looks to carve out a bold new path.
If StemSmart continues to deliver, the Perth biotech could soon find itself challenging the dominance of conventional transplant and anti-inflammatory drugs, unlocking a broader pipeline of therapies for a range of diseases and complications where limited or no other options exist.
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By James Pearson
