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In the past, inflammatory markers have been observed in the brain, cerebrospinal fluid (CSF) and plasma of patients with Alzheimer's disease (AD), which suggests that inflammation is involved in the pathogenesis of AD and may become a target for treatment.
A recent phase 2 randomized controlled study showed that sargramostim, an anticancer drug used in various types of leukemia to prevent infection, has shown promise in the treatment of AD.
The drug can improve the symptoms of dementia and enhance memory function in patients with mild to moderate AD.
The study was published online on March 24 in Alzheimer's & Dementia: Translational Research and Clinical Interventions.
Yimaitong compiles and organizes, please do not reprint without authorization.
Research Introduction Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (GM-CSF).
Past studies have shown that after receiving high-dose chemotherapy and hematopoietic cell transplantation (HCT), it is supported by GM-CSF The cognitive function of treated leukemia patients at 6 months was significantly better than that of HCT patients who received G-CSF therapy or not.
Sargramostim can stimulate the innate immune system of patients with mild to moderate AD and improve their cognitive function, and sargramostim may improve cognition through a mechanism independent of AD pathology.
Specifically, it may be that GM-CSF has an effect on nerves.
Cells have anti-apoptosis, promote neurogenesis and arteriogenesis, and reduce glial scar formation.
To test this hypothesis, the researchers conducted a randomized, double-blind, placebo-controlled trial.
A total of 40 patients with mild to moderate AD were recruited, and half of the patients received sargramostim (250ug/m2/ Day, 5 days a week, subcutaneous injection for 3 consecutive weeks), half of them received placebo injections.The follow-up time was 45 days and 90 days, and the researchers performed neurological, neuropsychological, blood biomarker and imaging evaluations on the patients.
The main outcome is safety.
Sargramostim is safe, well tolerated, and has no serious adverse reactions or amyloid-related imaging abnormalities.
Main findings: ➤Saggrastim treatment is expected to change the innate immune system markers, and there are no serious drug-related adverse events or amyloid-related imaging abnormalities.
➤At the end of treatment (EOT), compared with the baseline (P=0.
0074) and placebo group (P=0.
0370), the minimum mental state score of the sargramostim group increased, and the treatment effect continued to 45 after EOT Days (P=0.
0272).
Plasma amyloid markers (Aβ40 [AD reduction]) increased by 10% (P=0.
0105); plasma markers of neurodegeneration (total tau protein and UCH-L1) decreased by 24% (P=0.
0174) and 42% (P=0.
0019).
Expert comment As one of the main researchers of the study, Dr.
Potter from the University of Colorado pointed out that the innate immune system is an effective target for the treatment of AD.
This finding is consistent with previous studies in mice, but its clinical The therapeutic effect and curative effect are novel and unexpected.
Regarding the safety of sargramostim and the activation of the innate immune system, Dr.
Potter believes that short-term sargramostim treatment increases the determination of innate immune cells and cytokines in patients with mild to moderate AD, and its safety and tolerance Good sex.
Second, compared with the baseline and placebo groups, the changes in plasma measurements of amyloid, tau and neurodegeneration treated with sargramostim were statistically significant.
Multiple indicators that are significantly related to EOT indicate that sargramostim can regulate the innate immune system, reduce neuropathological responses and improve cognitive function.
Experts also pointed out that the adverse reactions related to sargramostim are mainly skin, gastrointestinal and headache reactions, which are in line with the expectations of the drug and consistent with the drug instructions.
Due to the small sample size of this study and the short treatment period, it is necessary to conduct a long-term treatment trial in the future to test the long-term safety and effectiveness of GM-CSF/sargramostim in the treatment of AD.
Yimaitong compiled from: Cancer Drug Promising for Alzheimer's Disease.
-Medscape – April, 07, 2021.
A recent phase 2 randomized controlled study showed that sargramostim, an anticancer drug used in various types of leukemia to prevent infection, has shown promise in the treatment of AD.
The drug can improve the symptoms of dementia and enhance memory function in patients with mild to moderate AD.
The study was published online on March 24 in Alzheimer's & Dementia: Translational Research and Clinical Interventions.
Yimaitong compiles and organizes, please do not reprint without authorization.
Research Introduction Sargramostim is a recombinant human granulocyte-macrophage colony stimulating factor (GM-CSF).
Past studies have shown that after receiving high-dose chemotherapy and hematopoietic cell transplantation (HCT), it is supported by GM-CSF The cognitive function of treated leukemia patients at 6 months was significantly better than that of HCT patients who received G-CSF therapy or not.
Sargramostim can stimulate the innate immune system of patients with mild to moderate AD and improve their cognitive function, and sargramostim may improve cognition through a mechanism independent of AD pathology.
Specifically, it may be that GM-CSF has an effect on nerves.
Cells have anti-apoptosis, promote neurogenesis and arteriogenesis, and reduce glial scar formation.
To test this hypothesis, the researchers conducted a randomized, double-blind, placebo-controlled trial.
A total of 40 patients with mild to moderate AD were recruited, and half of the patients received sargramostim (250ug/m2/ Day, 5 days a week, subcutaneous injection for 3 consecutive weeks), half of them received placebo injections.The follow-up time was 45 days and 90 days, and the researchers performed neurological, neuropsychological, blood biomarker and imaging evaluations on the patients.
The main outcome is safety.
Sargramostim is safe, well tolerated, and has no serious adverse reactions or amyloid-related imaging abnormalities.
Main findings: ➤Saggrastim treatment is expected to change the innate immune system markers, and there are no serious drug-related adverse events or amyloid-related imaging abnormalities.
➤At the end of treatment (EOT), compared with the baseline (P=0.
0074) and placebo group (P=0.
0370), the minimum mental state score of the sargramostim group increased, and the treatment effect continued to 45 after EOT Days (P=0.
0272).
Plasma amyloid markers (Aβ40 [AD reduction]) increased by 10% (P=0.
0105); plasma markers of neurodegeneration (total tau protein and UCH-L1) decreased by 24% (P=0.
0174) and 42% (P=0.
0019).
Expert comment As one of the main researchers of the study, Dr.
Potter from the University of Colorado pointed out that the innate immune system is an effective target for the treatment of AD.
This finding is consistent with previous studies in mice, but its clinical The therapeutic effect and curative effect are novel and unexpected.
Regarding the safety of sargramostim and the activation of the innate immune system, Dr.
Potter believes that short-term sargramostim treatment increases the determination of innate immune cells and cytokines in patients with mild to moderate AD, and its safety and tolerance Good sex.
Second, compared with the baseline and placebo groups, the changes in plasma measurements of amyloid, tau and neurodegeneration treated with sargramostim were statistically significant.
Multiple indicators that are significantly related to EOT indicate that sargramostim can regulate the innate immune system, reduce neuropathological responses and improve cognitive function.
Experts also pointed out that the adverse reactions related to sargramostim are mainly skin, gastrointestinal and headache reactions, which are in line with the expectations of the drug and consistent with the drug instructions.
Due to the small sample size of this study and the short treatment period, it is necessary to conduct a long-term treatment trial in the future to test the long-term safety and effectiveness of GM-CSF/sargramostim in the treatment of AD.
Yimaitong compiled from: Cancer Drug Promising for Alzheimer's Disease.
-Medscape – April, 07, 2021.
