Expert Financial Analysis and Reporting

Purpose of This Blog

I received this e-mail communication from a SmithOnStocks subscriber who has a good insight into the Chinese drug market and drug development. This is the first report of any kind that I have seen on patient outcomes in the trial of Neuralstem’s Chinese trial of NSI-566 stem cells in ischemic stroke patients. He wrote as follows:

“Larry,

Hi. I stumbled upon a story about CUR's stroke program in China today.

The story was posted online on Oct 17 2014. It was written in Chinese about the final visit (the 4th visit) of the first patient enrolled in this program.

I translated the abstract of this story and some key points below:

Abstract:

一次性神经干细胞移植手术治疗9个月后，首例参与患者完全恢复独立行走及语言能力，并且持续数月保持改善状态。其余病人也有不同程度的改善。

9 months after one time transplantation of Neuralstem cells, the first patient fully recovered his ability to walk and speak.

And these improvements lasted for months. Other patients expressed various degrees of improvements also.

北京军区总院附属八一脑科医院的医生，按照临床研究的预定方案，本周对参与中风临床研究的第一个病人，进行了神经干细胞移植手术治疗9个月后的第四次回访，

对病人进行了全面检查。结果表明，神经干细胞移植治疗，对这位原来病症较为严重的脑中风偏瘫患者，有很显著的疗效，并且这种效果在三个月前的前次检查初次显示后，

继续保持下来。按各项临床检测的诊断与判别，病人的各种功能恢复，与治疗前相比较，有50%甚至更好的改善。

Doctors from Beijing Bayi hospital conducted the 4th return visit for the first patient this week according to the trial design. The results showed that the transplantation greatly improved the patient who had had a relatively severe stroke greatly improved and the improvements last at least for 3 months. Various assessments, compared to pre-trial baseline, improved 50% or more.

神经干细胞移植的疗效在病人身上的直观表现，是其运动及语言功能障碍的显著改善。在做干细胞移植手术前，该病人需要家属搀扶才能来医院。

经过一次性神经干细胞的移植治疗，病人的肢体运动能力得到显著改善，至今其偏瘫一侧的肢体的功能已经有相当程度的恢复，达到能够完全独立行走、不需要任何人搀扶、协助的水平。

这次以及前一次来医院检查，他都是自己一个人坐公交车来的。

The most obvious relief from the cell transplantation is the patients' improvement in the walking and speaking ability. Before transplantation, this patient has to be carried by his relatives to the hospital.

After one time transplantation, the mobility of the patient’s limbs greatly improved. Though still a little paralyzed in one side of his body, the patient can now walk without ANY assistance.

He himself without company took the bus to the hospital in this visit and in the last one 3 months ago.

对语言功能障碍的恢复，神经干细胞治疗效果更为突出、更加迅速地体现出来：在手术后几天内，病人自己就感觉语言障碍有明显改善（他手术前说话有很严重的障碍，每次想表达一下自己的意思，只能一个字、一个字地“蹦”出来）。一个月回访时，他说话的能力就有相当程度的恢复（他太太当时甚至“抱怨”他话太多了---对于一个患病已达一年之久、因为中风而导致的语言功能障碍而长期“有口难言”的人来说，一旦功能恢复就会常常要打开“话匣子”，是完全可以理解的）。现在该病人的语言能力已基本恢复，可以毫不困难地整段整句地说话了。目前这种语言能力的恢复也继续保持良好。

As to the speaking ability, the cell transplantation worked more prominent and even quicker. Several days after operation, the patient himself felt his ability to speak greatly improved (he had severe speech impairments and could only speak one word at a time to express himself). At the first month visit, his speech ability greatly recovered (His wife even complained he's talking too much. It is understandable for a patient who had had a stroke and could hardly speak for more than one year, though). Now the patient's speaking ability almost fully recovered. And this improvement also preserves well.

后续入组的病人，目前也有不同程度的改善。必须指出的是，中风偏瘫病人的脑组织损伤，常常是非常严重的、有时甚至是不可逆的。因此，以神经干细胞来修复这样的脑组织损伤，是个漫长、也许需要数年之久的长期过程。其最终的治疗效果，也会因为病人的具体情况的不同、脑组织损伤的程度不同、以及病人本身的个体差异，例如年龄、身体状况、是否有其它相关病症、是否积极参与康复治疗等等因素而不同。

Other patients experienced various degrees of improvements as well. We have to point out; brain injuries due to the stroke are generally severe and irreversible. Therefore, it may take a very long period of time, sometimes years, for the patients to recover. The ultimate outcome may vary according to the patient conditions, degrees of injury, patient diversity such as age, general health, other diseases, willingness to participate into the trial, etc.

The remaining report is about the general description of the trial which we all know for a while. So I stop here.

The link of this page is http://www.stroketrial.cn/news/html/?443.html

You may use google translate or other programs if you will."

My Thoughts on This Report

Neuralstem has noted in response to questions that it appears to be seeing encouraging signs in the patients initially treated in the Chinese ischemic stroke trial.  However, this is the very early “dose up” part of the trial. In the US, this would be called a phase 1b trial that treats patients suffering from the disease state as opposed to healthy volunteers. It is extremely early to be expecting or talking about efficacy. The main goal of the first 18 patients treated in this trial is to establish the maximum safe tolerated dose for the following part of the trial which will be equivalent to a phase 2/3 trial in the US.  Investigators are, of course, measuring functional recovery.

Investors generally are skeptical of data coming out of China and Neuralstem is especially sensitive to this issue.  Hence, they are very cautious in talking about early results. Management would not comment to me on this report other than to say that stroke is a huge public health problem in China and one of the main reasons they have established a presence there.

This report is intriguing but I am not urging any action based on this. Still, given the potential importance of this trial, it is something worth watching.