The Phase 3 trial fully enrolled around 200 participants across six sites in the USA to test multiple doses of NurOwn and investigate effectiveness. The primary measurements were to examine safety of repeated intrathecal injections of NurOwn and the ability of NurOwn to slow progression of ALS/MND using a scale called the ALSFRS-R. To determine if NurOwn-treated stem cells were providing the intended biological effect, the (CSF) was measured for biomarkers and neurodegenerative/neuroinflammatory factors with the hope that these would be reduced.
The trial design included a criteria used to determine if the trial was a success (or not) which is called a a ‘responder’ analysis. Initially the rate of MND progression of each person is measured using the ALS Functional Rating Scale-Revised (ALSFRS-R) over 6 month to give each person a quantified level of MND progression. If a person in the trial was to be classed as a ‘responder’ following the NurOwn treatment then their rate of ALSFRS-R would need to reduce by a minimum of 1.25 points per month i.e. their rate of physical decline due to MND slows down. It is then possible to count how many people ‘responded’ in the treatment group versus those in the placebo group.
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Unfortunately, at the end of the 28-week study this comparison of responders between NurOwn and placebo was not statistically significant. 34.7% of people were responders in the NurOwn group i.e. met the responder definition described above compared to 27.7% in the placebo group.
Other measures that could indicate a successful treatment (known as Secondary endpoints) included safety, ALSFRS-R change from baseline, the Combined Assessments for Function and Survival (CAFS), slow vital capacity (SVC), and cerebrospinal fluid (CSF) biomarkers. ALSFRS-R mean scores were similar for both NurOwn and placebo groups and there was no difference in CAFS scores between the groups at the end of 28-weeks treatment. SVC was not different between the groups, although results may have been affected by the COVID-19 pandemic as SVC assessments were not possible at this time.
Further, it was reported that that NurOwn treatment resulted in an increase of neurotrophic biomarkers and reduction in neurodegenerative and neuroinflammatory biomarkers when compared to the placebo group, which is aligned with what the trial hoped to achieve. Follow up analysis will investigate whether these biomarker results, in addition to further analysis of the pre-specified subgroup of those with early disease, can reveal any additional information. You can read more about the trial here and the results here.
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Brainstorm has used an Expanded Access Program (EAP) for participants less severely affected by MND, as measured by the ALSFRS-R, who completed the Phase 3 clinical trial. Eligible participants are now able to access a total of 9 doses. Data is continued to be collected in this EAP to better understand the potential benefits of longer term treatment. You can read more about the EAP here.
A correction to the data published in Muscle and Nerve in December 2021 was announced in August 2022. Analysis in the original publication used an incorrect model. This correction had an impact on one of the secondary endpoints, average change from baseline in ALSFRS-R, in the pre-specified subgroup of participants with a baseline score of at least 35. The data showed a statistically significant treatment difference of more than 2 points of those on NurOwn compared to those on placebo for that subgroup. You can read the correction here.
In August 2022, BrainStorm announced they were submitting a Biologics License Application (BLA) to the U.S. Food and Drug Agency (FDA) for the approval of Nurown for the treatment of MND. The FDA declined to consider the approval but have now agreed to hold a Advisory Committee Meeting for NurOwn through the FDA’s File over Protest route. The meeting will be held on the 27th September, with a final decision to made by 8 December 2023.
Source: https://t-tees.com
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