治疗重症肌无力的新希望

BHT-3034治疗重症肌无力的IND获FDA批准

据THE MEDICAL NEWS网站报道，Bayhill制药公司基于其专有的BHT-DNA?平台以开发针对自身免疫性疾病和免疫介导性疾病的新型治疗方法，近日宣布向美国FDA提交的BHT-3034新药申请（IND）成功获批，BHT-3034是一种用于免疫治疗自体免疫性神经肌肉接头疾病重症肌无力患者疾病改善的DNA质粒疫苗。该份IND的提交是基于有希望的临床前疗效研究结果，即疾病严重程度明显减轻和抗乙酰胆碱受体抗体减少，以及GLP毒理学研究没有证据表明其有毒性和免疫原性。该IND还包括一项I / II期剂量递增临床研究，其给予40例重症肌无力患者肌肉注射BHT-3034。

BHT-3034是利用Bayhill公司专有的BHT-DNA?平台进行研发进入临床试验的第3个化合物。

该公司创办人兼董事、斯坦福大学免疫学跨学科项目主任Lawrence Steinman医师表示，“我们为通过这个机会为重症肌无力患者带来一种新的缓解疾病方法，感到非常兴奋。BHT-3034有通过一种高度特异的方式显着降低自体免疫反应的潜能，从而证明其临床益处。”

（生物谷Bioon.com）

BHT-3034 - Program Overview
About Myasthenia Gravis (MG)
MG is an autoimmune disease thought to interrupt the transmission of nerve impulses to muscles at the neuromuscular junction, or the location where nerve cells connect with the muscles they control. In MG an autoimmune response attacks acetylcholine receptors (AChR), which blocks nerve impulses and muscle stimulation via the neurotransmitter acetylcholine. As a result of this self attack, MG patients experience chronic fatigue, weakness, double vision and in some cases respiratory distress.
The Myasthenia Gravis Foundation of America estimates that up to 60,000 people in the United States may have MG. Approximately 12%-16% of those afflicted experience potentially fatal myasthenic crisis due to progressive weakness of the respiratory muscles, which is exacerbated by physical activity.
Current Treatments for MG
No disease-modifying therapies are currently approved for MG. Existing treatment options have limited efficacy, are often associated with significant side effects and only address the symptoms of MG. Therapies include anticholinesterase drugs, such as neostigmine and pyridostimine, which address chronic fatigue and weakness; and off-label use of chemotherapeutic agents and broad-based immunosuppressants such as corticosteroids. Although thymectomy, or surgical removal of the thymus gland, is performed in more severe cases it may produce remission.
About BHT-3034
BHT-3034 is a plasmid encoding the AChR designed to induce tolerance and improve the clinical outcomes in patients with MG. This product candidate has already shown positive results in an experimental autoimmune MG model (EAMG). An IND was filed for BHT-3034 with the FDA in January 2011.
Preclinical data
The efficacy of an animal-model equivalent of BHT-3034 (rBHT-3034) was demonstrated in rodents using the EAMG model. Either rBHT-3034 or saline was administered intramuscularly once per week and data was collected on several parameters of EAMG severity in already established disease. rBHT-3034 showed favorable results versus saline injections as follows:
Statistically significant reduction in overall mean disease severity
based on disease scores at end of treatment vs. baseline
Increased percentage of animals with no disease
Reduced activity of AChR-specific T cells

咨询了美国的药剂师，虽然已经获得FDA临床试验的批准，但是离开正式上市还有一段比较长的时间，尽管这样，这个机会还是为重症肌无力症患者带来了新的希望。

一个既没有副作用又有很好疗效的治疗方法即将面世，多么令人高兴的消息，我们拭目以待！

天山雪莲