Recent research progress in the field of central nervous system diseases
November 22, 2017 Source: WuXi PharmaTech
Window._bd_share_config={ "common":{ "bdSnsKey":{ },"bdText":"","bdMini":"2","bdMiniList":false,"bdPic":"","bdStyle":" 0","bdSize":"16"},"share":{ }};with(document)0[(getElementsByTagName('head')[0]||body).appendChild(createElement('script')) .src='http://bdimg.share.baidu.com/static/api/js/share.js?v=89860593.js?cdnversion='+~(-new Date()/36e5)];1. Parkinson's disease stem cell therapy achieves positive clinical results
Recently, International Stem Cell announced that the company's ISC-hpNSC cell therapy has yielded positive results in clinical Phase 1 trials for Parkinson's disease (PD). All of the first patients receiving treatment currently reached the primary end point of the trial in terms of safety.
PD is a neurodegenerative disease that primarily affects motor function in patients. The reason for the PD is due to the death of neurons that produce dopamine in the substantia nigra of the patient's midbrain. There are approximately 10 million PD patients in the world. The current treatment for PD is levodopa (L-DOPA) or other dopamine agonists. These therapies relieve symptoms in the early stages of the disease. However, as the disease develops, the therapeutic effect will gradually decrease, and at the same time, obvious side effects will occur, resulting in involuntary hand and foot exercise.
International Stem Cell uses parthenogenesis to grow pluripotents cells from unfertilized eggs. ISC-hpNSC cells are neural stem cells that are differentiated and highly purified from human parthenogenetic stem cells. In preclinical trials, these cells, after intracranial injection into the brain, increase brain dopamine levels and improve PD symptoms in non-human primate models. Growth factors secreted by ISC-hpNSC transplanted into the brain may help maintain the survival of dopamine neurons, and these cells may differentiate into dopamine neurons to replace the function of dead dopamine cells.
â–²The principle of the production of parthenogenetic stem cells (Source: International Stem Cell)
This clinical phase 1 trial is an open-label, single-center dose escalation trial with no control group. The primary goal is to test the safety and initial efficacy of ISC-hpNSC. Twelve patients with moderate to severe PD received 30 to 70 million neural stem cell transplants and received levodopa. After the transplant, the patient will be followed for 12 months to determine the safety and efficacy of ISC-hpNSC. They will then continue to follow up for 5 years to further monitor the safety of the therapy and its impact on the patient.
The trial data presented this time is the interim data of the first patients who received 30 million ISC-hpNSC cell transplants after 6 months of treatment. The trial data showed that the efficacy of levodopa was significantly enhanced in the treated patients: the effect of levodopa was not obvious, the time for patients showing PD symptoms decreased by 24%; the efficacy of levodopa was significant and the time for no side effects increased. 19%. Emotions in all patients improved 6 months after treatment, and the average emotional improvement was 35% based on the Beck Depression Inventory (BDI). The use of the Parkinson's disease Quality of Life Score-39 (PDQ-93) to test the quality of life of patients showed that the patient's mood, cognitive ability, daily activities and physical inconvenience were improved. Moreover, the patient's impulsive and compulsive disorders decreased by 53%. At the same time, clinical trials did not find any serious adverse events associated with transplanted cells. There is no evidence of the presence of tumors, cysts, inflammation or infection. Furthermore, no human leukocyte antigen antibody against transplanted ISC-hpNSC cells was found.
“We have observed a positive positive effect 6 months after cell transplantation. Although it is difficult to observe statistically significant differences due to the small number of samples, the mid-term results are still very encouraging,†International Stem Dr. Russell Kern, Executive Vice President and Chief Scientific Officer of Cell, said: "To test the safety and tolerability of ISC-hpNSC therapy, the dose of the first group of cell transplantation therapies is lower than the optimal dose from preclinical studies. We anticipate a stronger response as cell doses increase. These clinical trials have laid a solid foundation for our initiation of clinical phase 2 trials for the treatment of PD and traumatic brain injury."
2. Vraylar is approved by the FDA for adult schizophrenia maintenance treatment
Allergan recently announced that the US FDA has approved Vraylar (cariprazine) for a new drug application (sNDA) for adult maintenance treatment of schizophrenia. In the United States, Vraylar is also approved for the acute treatment of schizophrenia and mania, or mixed episodes of bipolar I disorder.
Schizophrenia is a chronic disease that seriously affects the lives of patients. There are approximately 2.4 million adult patients in the United States. Symptoms fall into three broad categories: positive symptoms (illusions, delusions, thinking disorders, and dyskinesias); negative symptoms (such as loss of motivation and social will), and cognitive symptoms (executive function, attention and working memory problems). Without maintenance therapy, 60-70% of people with schizophrenia will relapse within a year. Once a patient with schizophrenia has reached a stage of stabilization or maintenance, a doctor needs to develop a long-term treatment management plan to reduce the risk of recurrence, monitor and reduce side effects, and resolve residual symptoms as much as possible.
Vraylar is a once-a-day oral medication whose efficacy in the maintenance of schizophrenia is based on a 72-week multi-country, double-blind, placebo-controlled, randomized withdrawal clinical study designed to evaluate Vraylar Prevents the recurrence of schizophrenia in adult patients. The study included a 20-week open-label phase in which patients with schizophrenia received 3, 6 or 9 mg of Vraylar daily. During the open-label period, patients who achieved stable criteria were randomized to receive 3, 6 or 9 mg of Vraylar or placebo for 72 weeks until relapse. The primary endpoint was the time to recurrence during a randomized double-blind period.
Studies have shown that Vraylar significantly delayed recurrence time compared with placebo (P = 0.0010). The risk of recurrence in patients receiving Vraylar (29.7%, n = 30/101) was half that of patients receiving placebo (49.5%, n = 49/99), and the safety results were consistent with the results to date.
"The difference in response to treatment in patients with schizophrenia highlights the importance of having additional treatment options," said Dr. David Nicholson, chief development officer at Allergan. "We are pleased that the FDA recognizes Vraylar's maintenance care for adult patients with schizophrenia. Benefits, this approval demonstrates our continued commitment to Vraylar's R&D and our commitment to developing treatment solutions that address the unmet medical needs of people with mental illness."
3. Bill Gates personally donated 100 million US dollars to overcome Alzheimer's disease
Recently, Bill Gates published a long article on his personal blog, and decided to challenge Alzheimer's disease, which has not been published for 15 years.
â–² Bill Gates officially challenged Alzheimer's disease (Source: GatesNotes)
"If you can live to be in your 80s, your chances of getting Alzheimer's disease are close to 50%," Bill Gates said in his blog post: "In the top ten reasons for the death of the United States, this is the only lack of effective. Treatment of the disease. Moreover, it is becoming more and more popular."
Subsequently, Bill Gates mentioned why he should pay attention to Alzheimer's disease. With the continuous development of science and technology, the average life expectancy of ordinary people has been greatly improved, and now the elderly over the age of 80 can be seen everywhere. Although it is gratifying to extend the life expectancy, it is meaningless to live unhealthy life. With age, the probability of developing arthritis, Parkinson's disease, and other diseases increases. Among them, the damage caused by Alzheimer's disease is particularly serious.
"Alzheimer's disease is a bad disease for patients and their loved ones," Bill Gates said. "I know this very well. In my family, many men have Az. Haimo disease. When the disease takes away the mind of the person you love, and you can't do anything about it, it feels bad. You are like a chronic death of an acquaintance."
To this end, Bill Gates personally donated $50 million to the Dementia Discovery Fund. The fund, jointly launched by GlaxoSmithKline, Johnson & Johnson, Eli Lilly, Pfizer, Biogen, and the UK government, will continue to look for new The target of the disease, opening up new new drug pipelines, bringing about a cure for Alzheimer's disease. In addition, according to Reuters, Bill Gates will also donate an additional $50 million to support "non-mainstream" Alzheimer's disease therapy.
Regarding the future, Bill Gates put forward five thoughts: we need to understand how Alzheimer's disease progresses, need to discover and diagnose Alzheimer's disease at an earlier time, need new ways to stop the disease, and need to make people more Convenient participation in clinical trials requires better use of data. He believes that to achieve these five points, "we can greatly reduce the impact of Alzheimer's disease."
4. Pimavanserin is effective in treating dementia-related mental illness
Acadia Pharmaceuticals, which focuses on addressing the medical needs of central nervous system diseases, recently announced at the 10th "Clinical Trials on Alzheimer's Disease (CTAD)" in Boston to evaluate pimavanserin in Az Results of the "-019" clinical phase 2 study of the effects in psychosis in Alzheimer's disease.
Pimavanserin is a selective serotonin receptor inverse agonist (SSIA) that selectively targets the 5-HT2A receptor and reduces its basal activity. In humans, the level of activity of the 5-HT2A receptor is associated with hallucinations. When it is stimulated by psychedelic drugs or other agonists, the central nervous system produces psychedelic phenomena due to excitement. The combination of pimavanserin and 5-HT2A receptors attenuates its activity, thereby reducing the excitability of the central nervous system and reducing the risk of hallucinations or delusions. In 2016, the FDA approved the drug's marketing to treat mental disorders in patients with Parkinson's disease.
The results of this phase 2 clinical trial showed that pimavanserin reached the primary end point of the study, and patients with pimavanserin had a significantly reduced incidence of psychosis compared with placebo. The data presented in the CTAD containing multiple sensitivity and effectiveness analyses supported the main results. The drug shows greater benefits in more severe psychiatric patients. Based on these data, Acadia recently launched a "HARMONY" Phase 3 clinical study using pimavanserin to treat dementia-related psychosis. Dementia-related psychosis includes psychosis in Alzheimer's disease, dementia with Lewy bodies, Parkinson's disease dementia, vascular dementia, and frontotemporal dementia ). There are currently no FDA approved drugs for dementia-related psychosis.
"In the -019 clinical phase 2 study, pimavanserin significantly reduced psychosis in patients with Alzheimer's disease without negatively affecting cognition," Vice-Chancellor, University of Exeter Medical School Professor Clive Ballard, Executive Dean, said: "Pimavanserin has good tolerability characteristics compared to the known side effects of existing antipsychotic drugs. There is currently no significant FDA approved drug for the treatment of dementia-related psychosis. Unmet medical needs. These findings suggest that pimavanserin may be an important new treatment option for this type of patient."
Reference material
[1] International Stem Cell Corporation Announces Positive Interim Clinical Results for Parkinson's Disease Clinical Trial
[2] Allergan Receives FDA Approval for Use of VRAYLAR (cariprazine) in the Maintenance Treatment of Schizophrenia
[3] Bill Gates has a $100M and a 5-point strategy to end 15 years of failure in Alzheimer's R&D
[4] ACADIA Pharmaceuticals Presents Data From the Phase II Study of Pimavanserin in Alzheimer's Disease Psychosis at the Clinical Trials on Alzheimer's Disease (CTAD) 2017 Meeting
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