Tuesday, September 23, 2008

Wise Young

In 1998, a young woman broke her neck in the Goodwill Games. She did her rehab in Mt Sinai . . . we had become good friends.

At her going away party, she was crying and when I asked her why she said that if we found the cure in the USA, she wouldn't be able to get it.

I promised her I would make sure that China would get its own program into place.

I went to China in 1998 and while I was there I met Suzanne Poon, whose son had been injured . . .

In 2004, there were 3 kinds of cells available
embryonic fetal cells
umbilical cord blood cells
bone marrow or peripheral blood stem cells

We didn't have the money to work with bone marrow . . . we decided to work on UCB cells, which has been shown repeatedly to be safe and beneficial in treating neurological conditions.

There are groups around the world working on transplanting UBC into spinal cord injured patients (mentions Mexico . . . says that people are promising cures when they're really just experimenting)

We isolated a cell line that we called NO1.1 . . . when we transplanted them they stayed where we wanted them.

Lithium has been shown to have regenerative effects on neurons. It acts on a single enzyme, which inhibits nuclear factors. The factors control growth and differentiation; lithium applied to stem cells stimulates growth, is the point. We tried it on every kind of stem cell we could think of.

We were concerned that lithium might cause differentiation in cells, but we found that it does not. We transplanted neonatal cells into spinal cords and added lithium and got robust axon growth.

Showing a slide with the cells in bright green migrating across the site of a dark blue injury.

Lithium also boosts growth factors . . .

We've asked the 25 members of the SCI network to let us do clinical trials in chronic spinal cord injury patients . . . these are very well-run hospitals. We propose 5 trials; the first one was an intradural decompression of the cord within a few weeks after injury-- the benefits of this have been very exciting.

They cut into the cord and remove the necrotic tissue . . this has allowed us to understand much better how to operate safely on the cord. Remarkably, the removal of necrotic tissue (macrophages) in itself resulted in functional recovery for many of the patients.

WE've already given a 6-week course of lithium to sci patients to test for tolerance. We've applied for permission to do a cord blood mononuclear cell transplant with 40 patients . . . aiming for a trial with 400 patients. Looking at cord blood cells and then cord blood cells plus lithium.

In order to prepare for the final trial . . . we needed to get the Chinese to start doing follow up. The model had been that sci people in China just left the hospital right after their rehab and never were heard from again. They've now got 100 patients who were treated, re-habbed, and are registered for the trials.

The great advantage of doing trials in China is the number of patients; we can easily get 6,000 patients to randomize. The standards are very high, and we've just spent 4 years teaching them how to conduct rigorous trials. We're preparing to do parallel trials in the USA.

Thousands of Americans have come to me and asked how they can go to China and be part of these clinical trials? This is shameful. This is shameful that Americans are begging to go take part in these trials, and we're committed to conducting them here.

Wow.

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