PISCES II is a single arm, open-label study in patients living with disability resulting from ischaemic stroke. Eight centres across the UK’s NHS hospital service were involved in recruiting and treating the 21 patients in the study.
The study’s primary endpoint was for two patients to reach a minimum two-point improvement in the grasping and lifting test, sub-test number 2, of the Action Research Arm Test (“ARAT”), at three months post-treatment. Three of the 21 patients achieved this at three, six or twelve months respectively after treatment and were within a group of four responders who also showed clinically relevant improvements on the total ARAT score of arm motor performance. Although the ARAT sub-test number 2 study endpoint was not met as some responses came later than the three-month target, the result is nonetheless highly encouraging.
Strongly positive results were also seen in the other endpoints of the study, with seven patients (33%) showing a clinically relevant improvement on the Modified Rankin Scale (a measure of disability and dependence) and eight patients (38%) showing a clinically relevant improvement on the Barthel Index (a measure of performance in activities of daily living). In total, 15 out of 21 patients had a clinically significant response on at least one efficacy measure. Improvements in the ARAT scores, Modified Rankin Scale and Barthel Index were all sustained throughout the follow up period.
Patients in the PISCES II study were monitored prior to treatment to ensure that their disability was stable and showing no spontaneous improvement. A dose of 20 million CTX cells was administered to the patients between two and thirteen months (median seven months) after the stroke via direct injection adjacent to the area of the brain with the stroke damage. Patients were monitored for changes in arm function as well as a range of measures to assess disability and activities of daily living. At the time of reporting, all 21 patients in the study had completed three-month follow-up, with ten patients followed for six months and three for twelve months. Further data will continue to be collected until all patients have reached 12 months post treatment.
The PISCES II study also demonstrated that the CTX treatment was well tolerated. The most common adverse events were transitory and related to the surgical procedure, such as headache and nausea. Safety and efficacy data from the study will be presented at forthcoming stroke and rehabilitation medical conferences.
As a result of the positive data from the PISCES II study, we intend to apply to the US and European regulatory authorities to commence a randomised, placebo-controlled, pivotal Phase III clinical trial in patients who are living with disability post-stroke.
The PISCES II study was part-funded by a regenerative medicine and cell therapy development grant from Innovate UK.
Further information regarding our clinical trials, including relevant contact details, may be found on clinicaltrials.gov.