ChinaSCINet Update

[ay2012]

Dr. Young,
I have a question I'd really like your viewpoint on, and it's not meant with any shred of an attempt to antagonize, sincerely. But if the phase III trials show no more and no less of an indication of recovery for the patients (75% of patients recover minimally assisted but non-functional therapeutic stepping, no significant change in sensation, b/b/s or motor function) could you provide your thinking as to the cost-benefit of pushing this through phase III, trying to have doctors agree to do the surgery and cell injection, have patients go through months of rehabilitation, and have insurance companies actually cover this?
I know severe chronic SCI has no other therapies, I know the idea of regaining ANY form of walking is really exciting to all of us and I suspect many of us feel we could "do better" if we just were able to start activating the CPG.
But the other side of me also knows that there is no reason to anticipate any further or stronger recovery until its been demonstrated in the trial. So if recovery levels out at what you've seen so far, could you tell me why you think it's worth the 30 million needed for the trial, the tour-de-force you mentioned for the doctors hesitant to apply the therapy, and the millions (billions?) more dollars and months and months of grueling rehab needed once every SCI starts demanding this therapy, the subsequent rehab, etc.
Again, this question is a sincere one and I'm sure you have some thoughts (better informed than I) on the balance between waiting for the most efficacious therapies and getting SOMETHING (albeit imperfect) to the community. Thanks.

[Wise Young]

Thank you for a very good response!

Of course, but then shouldn't the patient experience scores close to the lesion site? As they are T injurys shouldn't there by now be an increase in motor scores in the trunk, since these would have a short distance to grow. I don't know the amount of locomotor training the patients performed considering the trunk though.

Many of our subjects have improvements in their sensory scores in the thoracic segments. By the way, we don't collect data concerning motor function of the thoracic segments (trunk and abdomenal muscles) because the examination is too hard to do without instrumentation and the ASIA examination omitted these muscles. In order to walk like they do (walk hundreds of meters using a rolling walking frame with minimal assistance with a rope around the knees to assist in the stance phase so that the knees don't buckle), many of our subjects had to make significant improvements in their truncal and postural muscles. So, we suspect that they have improved their trunk motor performance but we have only sensory scores for the trunk. Note that about 25% of the patients in the Kunming study had cervical spinal cord injury and they seemed to have regained as much locomotor function as the thoracic cases.

Wise.

[tumbleweeds]

Dr. Young,
Are there any restrictions or limitations in Phase III for length of post-injury time? I read where 13 years was the longest time of injury in Phase II but was wondering if you would try to find older injuries for Phase III, or does Phase II pretty well confirm age of injury doesn't seem to be an issue?
Thank you.

[fti]

Professor what is the difference between cells from the umbilical cord you use in your clinical trial
and the cell of BIOE of umbilical cord mlpc lien http://www.bioe.com/Product/Product....18&prodNav=pur
I thank you in advance for your answer

[kickingber]

:pray:Dr.Young How happy are the patients who took part in your Kumming trials? I hope they are very,very happy, but what I mean by my question is :Over all. Do the patients seem pleased with the results vesres the amount of work they had to put in to achive those results? (was the squeeze worth the juice?) I'm sure all are glade they did it, I just wonder if it was harder than they expected. How do you feel knowing that you were able to make it possible

[Wise Young]

kickingber,

I met most of the patients mid-way in the trial. At the time, I don't think that many of them were expecting all that much. Many told me that they were getting this or that function back. Most of course knew that they were getting better walking back because they were walking and had worked hard to achieve that recovery. Of course, they were happy about that. I don't know whether they are attributing it to the treatment or to their hard work. I didn't see most of the patients towards the end of the trial.

It was the family members that expressed the strongest positive feelings. Several of them were crying (with happiness). I don't know what the difference of expectation were between the family members and patients. Perhaps some of the patients thought that they were supposed to get such recovery back if they really worked hard at it. It is interesting that a number of them went back home for 6 months and did little or no walking, and lost their ability to walk. However, if they came back to the hospital, they regained the walking only after a relatively short period of practice.

Wise.

[Barrington314mx]

Thanks for posting that Doc. Its pretty uplifting to read. Anyways, have you heard anything about bowel, bladder, and sexual function in these patients? If not, do you plan to see what they are experiencing, if anything, in those areas? Thanks.

[JamesMcM]

Dr. Young when do you expect these results to the publish and release?

[jhope]

[SIZE=14px]" I pointed out that injuries to the lower thoracolumbar cord may not benefit from cord blood cells transplants as much and that we will need neuronal replacement therapies for people with flaccid paralysis of the legs."[/SIZE]

1)Do these therapies exist?

2) Are these trials are c5-c7 chronic sci only?

[Wise Young]

[SIZE=11px]Yesterday, 09:30 PM[/SIZE]

[SIZE=14px]" I pointed out that injuries to the lower thoracolumbar cord may not benefit from cord blood cells transplants as much and that we will need neuronal replacement therapies for people with flaccid paralysis of the legs."[/SIZE]

1)Do these therapies exist?

2) Are these trials are c5-c7 chronic sci only?

(1) Yes, neuronal replacement therapies do exist and the reason why so many scientists are interested in neural stem cell therapy. Neural stem cells should be able to produce neurons to replace neurons that have been lost. NeuralStem Inc. and Stem Cell, Inc. both have neural stem cell lines (from fetal sources) but they assume that the cells will not be immunologically rejected after transplantation and the treatments may require immune-suppression. We have been working on two potential sources of immune-matched neural stem cells. The first is Muse cells from HLA-matched umbilical cord blood. Muse cells are pluripotent cells that have been reported to produce neurons. We have found that umbilical cord blood contains large numbers of such Muse cells. We are trying now to grow these cells. Muse cells would not require genetic manipulation. The second are bone marrow cells. Bone marrow has muse cells and it would be possible to remove bone marrow cells from the patient, extract Muse cells, differentiate them to neurons, and then transplant them into the patient without genetic manipulation. We are doing these studies now in the laboratory.

(2) Which trials are you referring to when you say "these trials"? If you are referring to Phase III trial that we are planning for next year, the trials will be from people from C5 to T11 neurological levels. If you are referring to trials that we hope to start for thoracolumbar injuries, I don't think that we will be ready to start them until 2015 at the earliest and probably not until 2016. There are many problems that still have to be solved before we can take Muse cells to trial.

Wise.