‘Catapulting’ forward with MultiStem

The grant—expected to provide up to £2 million ($3 million) in support over the course of three years—will support a Phase 2a clinical study evaluating MultiStem cell therapy on acute respiratory distress syndrome (ARDS) patients. The study will be conducted by Athersys at leading clinical sites in the United Kingdom in conjunction with Cell Therapy Catapult.

“We are pleased to receive this award from Innovate UK and to work with the Cell Therapy Catapult to conduct this important study,” stated Dr. Gil Van Bokkelen, chairman and CEO of Athersys, in a news release announcing the award and collaboration. “Both organizations reflect a commitment to be a driving force in healthcare innovation and to establish the UK as the leading gateway for the regenerative medicine field in Europe.”

“ARDS is a serious condition that is ineffectively treated by current standards of care, resulting in substantial patient and healthcare system impact,” Van Bokkelen added. “We believe that MultiStem cell therapy may provide an important new option for treatment and has the potential to meaningfully improve clinical outcomes for patients that are extremely ill.”

William (B.J.) Lehmann, president and chief operating officer of Athersys, tells DDNews that ARDS “is a serious condition, requiring ICU/ventilator treatment, and occurs from a variety of causes that catalyze a substantial inflammatory response that greatly affects the lungs.”

The Phase 2a clinical study “will explore the safety and efficacy of MultiStem treatment in the ARDS patient population,” Lehmann says. “We have not yet disclosed the details of the study plan, but this will come soon.”

“Based on our prior work, we hypothesize that the treatment will help bring the inflammation under control rapidly and assist in the healing of damaged lung tissue,” he continues. “This should result in better overall recoveries and reduce time on the ventilator and in the ICU setting. Our working in this area has been limited to large animal models of the condition, where we have seen good benefit. Our work in other clinical areas [such as graft-versus-host disease] suggests that we can have an impact in this patient population.”

If the Phase 2a clinical study is successful, “most likely we would proceed with advanced clinical studies, designed to advance us to filing for registration,” Lehmann says.

Keith Thompson, CEO of Cell Therapy Catapult, stated in a news release, “Bringing advanced therapeutic clinical trials to the UK is a key part of our strategy to accelerate the growth of the cell therapy industry to generate health and wealth. We are delighted to collaborate with Athersys on this important trial.”

MultiStem cell therapy may provide an important new option to treatment, according to Thompson, who notes, “Preclinical data suggests that MultiStem cells may have a protective effect by shifting the physiological response from pro-inflammatory to anti-inflammatory. In animal models, MultiStem cells have demonstrated an ability to reduce the severity of pulmonary distress, reduce alveolar edema and return lung endothelial permeability to normal.”

Intravenous MultiStem treatment early on, following the onset of the condition, may ameliorate the initial hyperinflammation and reduce the fibrotic activity that follows, thereby speeding the return to and improving the likelihood of more normal lung function and helping patient recovery, he explains, adding that up to 45 patients will be recruited at six to eight sites for the study.

“As part of our strategy, we are collaborating with a number of companies and institutions, not all grant-funded,” Thompson said. ”We were successful partners in three other projects in the same Innovate UK program this year.”

Furthermore, the UK remains supportive of stem cell therapy.

“The government continues to remain squarely behind regenerative medicine as one of the eight great technologies of the UK,” Thompson said. “The Cell Therapy Catapult aims to develop a portfolio of clinical activities that covers the range of the sector, generating strong clinical data and building supportive structures for routes to market that will drive investment and value into the UK.”

Characterized by widespread inflammation in the lungs, ARDS can be triggered by pneumonia, sepsis or other trauma and represents a major cause of morbidity and mortality in the critical care setting. ARDS has significant implications, as it prolongs intensive care unit and hospital stays and requires convalescence in the hospital and rehabilitation. There are limited interventions and no effective drug treatments for ARDS, making it an area of high unmet clinical need with high treatment costs. The medical need for a safe and effective treatment of ARDS is also significant due to its high mortality rate. Annually, it affects some 33,000 patients in the United Kingdom and 400,000 to 500,000 patients in Europe, the United States and Japan.