Wise, help please.

[Wise Young]

Originally Posted by keeping on

Wise, thank you as usual; we're waiting to be tested ourselves and are eagerl and willing participants to find a cure. Remeber sitting ina chair and waiting for soemthing big to happen can be torturous. Please understand our emotions and expectamcy for some breakthru. I know you understand this; but those of us that experience everyday thoughly understand this.

Thanks,
T.J.

T.J.,

It would be so good if we had many clinical trials and not just one or two. The more the better.

Wise.

[keeping on]

I have not spoken to Kessler; just read reviews. What improvements is he looking at? When will his clinical trials begin? Thanks in advance.

T.J.

[medstudent]

Question to Dr. Young-

What dollar amount would be necessary to push spinal cord research and trials to the point at which a truly effective therapy (for complete chronic injuries) can be developed?

[Schmeky]

Originally Posted by Young

"Years away" seems about right. I agree with Dr. Kessler. We are years but, in my opinion, not decades away from therapies that restore function.

This is not personal, and know you're working diligently, but this sounds very familiar to me.

[lakboy]

well if it is not decades away then nine years is too long also !!

[lakboy]

half of this community wont be alive to see the cure then.

[Wise Young]

Originally Posted by medstudent

Question to Dr. Young-

What dollar amount would be necessary to push spinal cord research and trials to the point at which a truly effective therapy (for complete chronic injuries) can be developed?

It currently costs a pharmaceutical company an average of over $1 billion to move one therapy from laboratory to market. The entire investment of this nation into spinal cord injury research is less than $100 million per year.

Let me give you two concrete examples. First, Acorda Therapeutics took 12 years to get Fampridine through phase 1, 2, and 3 trials plus manufacturing and regulatory approval and onto the market for multiple sclerosis about $468 million, including failed attempts at getting approval for spinal cord injury. This is considered to be very efficient. Second, our phase 3 clinical trial for umbilical cord blood cells transplants plus lithium will cost approximately $32 million. We will be doing parallel trials in China for about $6 million. We are not counting all the preclinical work that went into developing this treatment nor all the volunteer work that is being done to raise the money, organize and train the networks, and the voluntary contributions of the doctors. If we are successful in getting cord blood and lithium treatment of spinal cord injury approved, it will be a record of efficiency and low cost.

Wise.

[Wise Young]

Originally Posted by Schmeky

This is not personal, and know you're working diligently, but this sounds very familiar to me.

Schmeky,

I have gone through many therapy battles for spinal cord injury, perhaps as much or more than anybody else in the field. I have helped bring two treatments through the gauntlet from laboratory to clinic.
• Methylprednisolone. It took us about 11 years (from 1979 to 1990) to get methylprednisolone from our first discovery of its effects in the laboratory to completion of phase 3 trials for acute spinal cord injury. It took another 4 years before a majority of doctors were using the drug around the world.
• Fampridine. It took 12 years (from 1997 to 2009) and $468 million to get regulatory approval for Fampridine treatment of multiple sclerosis and we failed to do so for spinal cord injury. Actually, the preclinical work for Fampridine was originally done in the 1980's so the "bench-to-bedside" took over 20 years.

I have spent the last six years and travelled nearly 2 million miles to develop and build chinascinet and more recently scinetusa to do clinical trials of promising therapies for chronic spinal cord injury. We have completed three trials (CN100, CN101, CN102A) and are starting our fourth clinical trial (CN102B) testing umbilical cord blood mononuclear cells and lithium. We will begin CN103 and US103 next year. I can't even begin to count the thousands of meetings, presentations, phone conferences, and fundraisers that this effort has required. I am very grateful to Rutgers and my family for allowing me to be away so much. Many hundreds of people have volunteered their efforts to make these trials come into being. We will know by 2012 whether or not umbilical cord blood and lithium works.

I am seeing the light at the end of the tunnel for the umbilical cord blood and iithium trials. However, we need dozens of trials to test all the promising therapies. That is why I have spent so much time setting up networks that have the capability of doing multiple trials each year. A lot of people have asked me why I don't just focus my effort on just one or two hospitals. The reason is of course because I believe that we need a network to cast many throws of the dice, not just one. Even though there is a limited probability that each throw will yield the jackpot, we increase our probability of winning by throwing the dice many times. More important, if we are successful once, both funding and the likelihood of success increases.

A network should be able to do several trials per year year, testing the most promising therapies as quickly and efficiently as possible. ChinaSCINet, for example, has access to an almost unlimited number of patients. Our main obstacle is funding. I believe that funding should be less of a problem once we have had our first success and show that we can do clinical trials faster and more efficiently than anybody else. When that happens, many companies will be flocking to us to test their therapies. Now, if you think that the above will happen in the next year or two, I don't think so. On the other hand, i think that there is a strong likelihood that it will happen within a decade.

Wise.

[swh2007]

Five years ago, the Juvenile Diabetes Research Foundation was funding 5 clinical trials. Right now, it is funding 43 clinical trials. It has the same fundraising challenges as every other research organization. It seems like there is real hope that sci research could experience similiar progress. But fundraising is a hard deal right now and it has to be approached with single minded effort that seems to be lacking in the sci community. Maybe it's because we don't have a national, well organized, dominant sci fundraising organization. Just some obervations. They may be wrong.

[Rollin Rick]

Originally Posted by lakboy

well if it is not decades away then nine years is too long also !!

Agreed!! I cannot take this kind of news anymore.... Better get used to your chair... Or the next couple of chairs should I say?