#Als stem cell treatment cost trial#
Here, we report results of the BCT-002 phase 3 clinical trial conducted to evaluate whether repeat treatment with MSC-NTF would safely slow the rate of ALS disease progression. MSC-NTF has been shown to favorably modify neuroprotective and neuroinflammatory cerebrospinal fluid (CSF) biomarkers following single intrathecal administration 5 with safety and preliminary efficacy demonstrated in prior studies 5, 6 in ALS participants. Mesenchymal stem cells (MSC) derived from adult bone marrow were induced under proprietary ex vivo conditions to secrete high levels of neurotrophic factors (MSC-NTF). A cell-based therapy that targets multiple disease pathways has the potential to tackle the multifaceted pathogenesis of ALS by supporting survival of diseased neuronal cells, both by secreting neuroprotective factors and by modulating neuroinflammatory and neurodegenerative pathways. There is currently no cure for ALS, and no treatment halts or reverses disease progression. Neuroinflammation is believed to play a prominent role. Given the unmet patient need, the results of this trial warrant further investigation.Īmyotrophic lateral sclerosis (ALS) is an adult neurodegenerative disease, characterized by progressive degeneration and loss of cortical and spinal motor neurons. However, a pre-specified subgroup suggests that MSC-NTF participants with less severe disease may have retained more function compared to placebo.
The study did not reach statistical significance on the primary endpoint. Significant improvements in cerebrospinal biomarkers of neuroinflammation, neurodegeneration, and neurotrophic factor support were observed with MSC-NTF, with placebo unchanged. A pre-specified analysis of participants with baseline ALSFRS-R ≥ 35 (n = 58) showed a clinical response rate at 28 wk of 35% MSC-NTF and 16% placebo (OR = 2.6, P = .29). Thirty-three percent of MSC-NTF and 28% of placebo participants met clinical response criteria at 28 wk (odds ratio = 1.33, P = .45) thus, the primary endpoint was not met. Overall, MSC-NTF treatment was well tolerated there were no safety concerns. A pre-specified analysis leveraged baseline ALSFRS-R of 35 as a subgroup threshold. A change in disease progression post-treatment of ≥1.25 points/mo defines a clinical response.
The primary endpoint evaluated efficacy of MSC-NTF through a responder analysis and safety. Participants received three treatments of MSC-NTF or placebo intrathecally. This randomized, double-blind, placebo-controlled study enrolled ALS participants meeting revised El Escorial criteria, revised ALS Functional Rating Scale (ALSFRS-R) ≥25 (screening) and ≥3 ALSFRS-R points decline prior to randomization. We aimed to evaluate whether mesenchymal stem cells induced to secrete high levels of neurotrophic factors (MSC-NTF), a novel autologous cell-therapy capable of targeting multiple pathways, could safely slow ALS disease progression. Amyotrophic lateral sclerosis (ALS) is a fatal neurodegenerative illness with great unmet patient need.
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