Wise Q and A

Do the patients need to be able weight bear alone for an hour?

No, just in a standing frame. Probably achievable for most patients.

What's the difference between the kind of rehab they do in China vs. the USA?

This has been the most intense debate we've had . . . in China every patient is pushed to the point of tolerance, which is obviously not the standard. Here we think that 1 hour a day for 3 days a week qualifies as intense. They've landed for the US trial on a standard of 3 hours per day for 3 days a week. All this will be walking, walking, walking. There is no data whatsoever on this subject. In Germany they did 3 hours a day for 5 days a week, which was considered at that time to be very intense.

Q: If you have a settlement, you're golden, if you're in the VA, you're silver, if you're neither, you're screwed . . . what's the plan to get people into these expensive trials?

There will be a lot of competition in the coming years. Money will be very tight. What the sci community can do is share the wealth. I see expensive equipment sitting in peoples' homes, and we should be making maximum use of these devices. There will be studies coming out that will justify insurance payment for these devices. It's hard, because it's a very tough question to answer.

Q: Will there be immunosuppression in your trials?

It won't be necessary, because we're matching blood in 4 out of 6 HLA categories. In our animal studies the cells do not get rejected.

Q: will the lithium cause pain?

Not in our studies. That was one of my primary worries; that lithium would cause pain. we just did an 80 patient study with a randomized placebo control . . . we will have a definitive answer this fall. we don't have any data on transplant plus lithium yet;

Q: Neurotrophins have been tried in the past and have not worked out very well

No, I have not seen that in humans . .
Lithium has been shown to stimulate stem cells in the brain as well as neurotrophins. It's been used for a century in treating the brain and the cord

Q: Will the patients have to live at the centers?

During the observational phase, people will have to be at the center 3 times. The patient doesn't have to come for the whole period. we anticipate a 3 day hospital stay, then they'll go home for 12 days, then come back for an intensive 6 week period of locomotor training. Some of the centers will be able to do more patients, but this has been the source of some very intense discussion, but we have not landed on any definitive answer.

Q: Who is going to pay for this?

We estimated that the total cost for this phase 3 trial would be $32 million. But we got the company to donate the cells, which brings it down to $24 million. Then on August 12 the FDA changed the rules so that we can recover some of the money from the patients; we're doing local fundraising, we're negotiating with the centers to lower the costs. We are committed to not having MONEY be the reason someone gets turned away.

We may do it like college admissions, where everyone who qualifies to be admitted is admitted, and then the potential to pay is factored into the cost for each person.

Q: What would probably be the expense after the trial to a patient?

We're carrying clinical trial insurance, so nothing. Remember that the trial will be randomized, so a quarter of the patients will get nothing, a quarter will get only lithium, a quarter will get only cord blood cells, and a quarter will get both. We have committed to giving all the participants the full treatment, assuming of course that the full treatment is effective.

In closing -- we should do this ourselves. I'm sick of waiting. We should not be waiting for a gift from a sugar daddy, or from the NIH, or from a company. I'm tired of waiting for someone who has a conflict of interest to step up. The people who have no conflict of interest in this -- who have in fact a vested interest in this -- are you.

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