以色列成年干细胞试验-- - ALS患者的希望吗？
特拉维夫（路透社）周一.2012年7月23日，美国东部时间0:32
特拉维夫（路透社）新闻 : BrainStorm正在进行干细胞治疗ALS患者的临床试验表明, 它的成体干细胞治疗耐受性良好，根据一个临时的安全评估,它似乎是安全的,不存在任何不必要的风险 . 此外，通过检测,一些病人的病情呈现稳定迹象.
基于BrainStorm的以色列正在开发NurOwn用于治疗肌萎缩性侧索硬化症（ALS）,又称卢伽雷氏病,一种进行性神经退化疾病，影响大脑和脊髓中的神经细胞 .
“这么快就给出这样一个临床研究疗效数据,实属罕见了. 但在个体基础上 ,我们可不能忽视这么一个事实,那就是在参与试验的病人身上,我们看到了许多改善 ,. 在不同领域的每一个人.” 摩西·纽曼说，他是生物医学研究设计的首席执行官, 生物医学研究设计服务于合同研究组织. 他对路透社记者说,一些病人呼吸得到改善,一些病人肌肉力量得到加强,一些病人说话能力也得到改善.
纽曼说, 每个病人经过九个月观察之后, 最终报告预计在今年年底出来.
BrainStorm总裁哈伊姆·Lebovits说, 初步结果表明，干细胞不仅有可能停止病情恶化，而且甚至可以治愈ALS。
“接下来的试验阶段将证明这一点, 但这些结果也重申我们的信念,对不太严重的病症,如多发性硬化症和帕金森氏,有巨大成功的潜力.”他说.
接受试验的患者用来自他们自己骨髓里的干细胞用NurOwn干细胞技术进行移植治疗.
第I / II期临床试验，旨在评估BrainStorm的治疗的安全性和初步疗效，试验在耶路撒冷的哈达萨医学中心开展。该公司已向以色列卫生部提交了临时安全报告.
“我们这个试验进展到下一半, 这一重要的安全性和耐受性数据是令人欣慰的.” 哈达萨神经内科的迪米特里奥斯说 ,他领导了这个试验. “, 虽然这是1期的临时安全总结报告,但它记录了这项试验的初期终点的结果. 我们可不能忽视一些可能的有前途的对个别病人所观察到的临床疗效,”
尤其有个病人, 耶路撒冷的犹太教教士拉斐尔Shmuelevitz,已经看到他的病情明显得到改善,
他除了患上ALS,还患有称之为重症肌无力神经肌肉疾病,他被看成一个慈悲的病人,用药比其他病人的剂量要高.在他写给BrainStorm的信中, 这是由路透社获得的副本,这个
犹太教祭司说,这治疗把他”从深处升起, 我已经变得像一个新的人。”
他说，在治疗前，他平衡感差，在没有帮助的情况下,他双腿无法行走。
“今天，干细胞治疗后，我能连续平稳地行走,即使没有拐杖的帮助下，我的声音也有巨大的改善，”他说。
BrainStorm正在等待美国食品和药物管理局的批准，开始在马萨诸塞州医学院和马萨诸塞州总医院的大学进行ALS的试验。
“接下来的步骤是探索我们计划协助设计的更多的剂量和第二阶段的安全性和初步疗效研究,” 马萨诸塞州总医院神经内科主席Cudkowicz说.
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Israel adult stem cell trials - hope for ALS patients?
TEL AVIV | Mon Jul 23, 2012 12:32am EDT
TEL AVIV (Reuters) - A clinical trial of ALS patients conducted by BrainStorm Cell Therapeutics shows its adult stem cell therapy is well-tolerated, appears to be safe and does not present undue risk, according to an interim safety review.
Moreover, in some patients signs of stabilization of the disease were detected.
Israel-based BrainStorm is developing NurOwn for the treatment of amyotrophic lateral sclerosis (ALS), also known as Lou Gehrig's Disease, a progressive neurodegenerative disease that affects nerve cells in the brain and spinal cord.
"It's very uncommon to give at such an early point in a clinical study efficacy data, but we cannot ignore the fact on an individual basis we could see improvement in many of the patients involved, each one in different areas," Moshe Neuman, CEO of Biomedical Research Design, which serves as a contract research organization for the trial, said.
In some patients breathing improved, in others it was muscle strength and in others it was speech, he told Reuters.
Neuman said a final report was expected by the end of the year after each patient has been observed for nine months.
BrainStorm President Chaim Lebovits said the preliminary results demonstrate that the stem cells have the potential not only to stop deterioration but perhaps even cure ALS.
"The coming phases in the trial will have to prove this, but these results also reaffirm our belief that we have an enormous potential of being successful with less severe indications such as multiple sclerosis and Parkinson's," he said.
Patients in the trial were transplanted with stem cells derived from their own bone marrow and treated with the NurOwn stem cell technology.
The Phase I/II trial, designed to evaluate the safety and preliminary efficacy of BrainStorm's therapy, is being conducted at Jerusalem's Hadassah Medical Center. The company submitted the interim safety report to Israel's Health Ministry.
"This important safety and tolerability data are reassuring as we progress to the next half of this trial," said Dimitrios Karussis of the Neurology Department at Hadassah, who is leading the trial. "Although this is an interim safety summary report documenting achievement of the study's primary endpoint, we cannot ignore some possible promising indications of clinical efficacy observed in single patients."
In one patient in particular, Rabbi Rafael Shmuelevitz of Jerusalem, significant improvement was seen. Shmuelevitz, who in addition to ALS also has a neuromuscular disease called myasthenia gravis, was received as a compassionate patient and given a higher dose than other patients.
In a letter to BrainStorm, a copy of which was obtained by Reuters, the rabbi said the treatment raised him "up from the depths and I have become like a new man."
Before the treatment he suffered from disequilibrium and could not walk without help from both sides, he said.
"Today, after the stem cell treatment, I am able to walk consistently and with stability even without the help of a cane. In my voice there is also immense improvement," he said.
BrainStorm is awaiting U.S. Food and Drug Administration approval to begin ALS trials at the University of Massachusetts Medical School and Massachusetts General Hospital.
"The next steps are to explore additional dosages and a Phase II safety and preliminary efficacy study that we plan to assist in designing," Merit Cudkowicz, chair of the neurological department at Massachusetts General Hospital, said.
According to the ALS Association, 5,600 people in the United States are diagnosed each year with the disease, which has severely disabled British physicist Stephen Hawking.
The FDA last year granted NurOwn orphan drug designation, which encourages the development of treatments for rare diseases by providing financial incentives.
(Reporting by Tova Cohen; Editing by Maureen Bavdek)
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