Special Report: Stem cell play Cynata Therapeutics has just passed another test in its quest to bring a stem-cell based treatment for graft-versus-host-disease (GvHD) to market.

GvHD afflicts bone-marrow recipients when a recipient’s body recognises a transplant has come from someone else.

It’s almost always fatal in cases where the recipient’s immune system is resistant to steroids.

Cynata’s treatment also holds exciting potential to one day be applied to a raft of other conditions including asthma and autoimmune diseases which affect one-in-nine and one-in-20 Australians respectively.

Cynata’s “CYP-001” treatment for steroid-resistant GvHD has just passed all safety and efficacy endpoints for its Phase 1 trial.

Fourteen out of 15 patients showed an improvement in GvHD severity by at least one grade compared to baseline, while signs and symptoms of the disease were completely resolved in eight out of 15 patients.

The company is now looking forward to advancing the treatment to Phase 2.

Devastating disease

“Steroid-resistant GvHD is a devastating disease with a high rate of mortality,” says Cynata’s Vice President of Product Development Kilian Kelly.

“We look forward to advancing our Cymerus MSCs [mesenchymal stem cells] into Phase 2 trials for GvHD and other indications.”

MSCs are adult stem cells that can be isolated from human or animal sources to produce more than one kind of specialist cell.

CYP-001 is the leading product from Cynata’s production of MSCs.

Cynata’s technology is based on induced pluripotent stem cells — the source of MSCs.

The main advantage is they can be grown in almost infinite quantities in the lab and can be derived from anywhere in the body.

Crucially, they don’t require embryos, which means they avoid common religious and ethical questions.

They also avoid the hurdle of requiring a bone marrow donation, which is extremely painful.

Asthma treatment could be next

Inside Cynata’s studies into GvHD is a broader investigation by medical science into methods that we can use to modulate the body’s immune system response.

Conditions like asthma, which affects one-in-nine Australians, and autoimmune disease, which afflicts one-in-20, are on the rise. In these conditions, it’s the body’s overreaction to something that causes the patient distress.

If MSCs can be harnessed to fight off GvHD, treatments to conditions such as asthma and autoimmune conditions could be around the corner.

As for Cynata, next steps are to commence a clinical trial in patients with a condition called critical limb ischaemia (CLI) in 2019.

CLI is a consequence of peripheral arterial disease, which affects at least 10 per cent of the Australian population.

CLI patients are at substantial risk of severe consequences, including limb amputation and mortality, but pre-clinical studies suggest that Cynata’s MSCs may result in a profound benefit in this condition.

 

This special report is brought to you by Cynata Therapeutics.

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