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Attacking asthma
October 2017
by Ilene Schneider  |  Email the author


MELBOURNE, Australia—Asthma is a chronic, long-term lung condition recognized by the World Health Organization as a disease of major public health importance because of its global prevalence. Patients who suffer from chronic asthma manage the disease through the use of steroid-type drugs, which can have significant side effects and impact on their quality of life. The global market for asthma drugs is expected to reach $25.6 billion by 2024.
Australian stem cell and regenerative medicine company Cynata Therapeutics Ltd. has obtained promising data supporting the efficacy of its proprietary Cymerus mesenchymal stem cells (MSCs) in a second preclinical asthma study. Conducted under the supervision of Associate Prof. Chrishan Samuel and Dr. Simon Royce of the Monash University Lung Biology Network, the study focused on the effects of Cymerus MSCs in combination with or in comparison to the corticosteroid dexamethasone, commonly used to treat exacerbations of asthma in human patients. Using a well-established mouse model of chronic allergic airways disease that closely resembles asthma in humans, the study focused on airway hyperresponsiveness (AHR), a key clinical manifestation of asthma.
Cymerus, a proprietary “off-the-shelf” MSC platform, is said to overcome critical issues in the production of therapeutic mesenchymal stem cells by enabling the economical manufacture of commercial-scale MSCs, independent of multidonor limitations. Cymerus utilizes induced pluripotent stem cells derived from a single blood donation to generate mesenchymoangioblasts, a precursor that is used to manufacture an unlimited number of therapeutic MSCs.
Cymerus has the potential to create a new standard in the development and manufacture of stem cell therapeutics, the company says. The company announced what it says are clear efficacy data for Cynata’s Cymerus MSCs in a second preclinical study in a clinically-relevant model of asthma. The study showed that Cymerus MSCs caused significantly greater reduction of airway hyperresponsiveness compared to corticosteroid treatment. Data from the study suggest that Cymerus MSCs can be administered alone or in combination with corticosteroids to treat the airway hyperresponsiveness associated with asthma. Completion of this study will further advance the path towards clinical trials.
According to initial results from this study, treatment with dexamethasone alone significantly improved AHR, as compared to untreated controls. Treatment with either Cymerus MSCs alone, or Cymerus MSCs in combination with dexamethasone, resulted in a substantially greater suppression of AHR, which was significantly superior to that seen with dexamethasone treatment alone. All of the treatments were administered by the intranasal (IN) route.
A previously-reported study, announced March 2, 2017, found that both intravenous and IN administration of Cymerus MSCs caused statistically significant improvements in the three main features (airway hyperresponsiveness, inflammation and airway remodeling) of asthma in this model. Additionally, IN delivery of Cymerus MSCs completely reversed pathologic collagen deposition, a sign of airway remodeling/fibrosis. Studies by the same group have determined that other types of stem cells did not have similar effects to IN Cymerus MSCs in this model, unless used in combination with other drugs.
In October 2016, Cynata announced initial data from a proof of concept study of Cymerus MSCs in an experimental model of asthma, which was conducted under the supervision of Samuel and Royce. Those initial results demonstrated that Cymerus MSCs have “a dramatic effect on improving airway hyperresponsiveness,” according to the company. The final report of that study demonstrated that Cymerus MSCs also consistently reduced markers of airway inflammation and airway remodeling.
As Samuel said, “It is very striking that Cymerus MSCs, whether alone or in combination with dexamethasone, had a significantly greater effect on decreasing AHR in this model than dexamethasone alone. We are now conducting further analyses on the effects of these cells on other features of the disease process including inflammation and airway remodeling.”
According to Dr. Kilian Kelly, Cynata’s vice president of product development, “These findings raise the possibility that Cymerus MSCs may have superior efficacy to corticosteroids in some asthma patients, in addition to offering a way to treat this condition without the side effects and/or resistance associated with steroid therapy. Furthermore, it was important to show that Cymerus MSCs can be administered in combination with corticosteroids, especially in the context of initial clinical trials, in which most patients are likely to be undergoing corticosteroid treatment at the time of enrollment.”
Code: E101713



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