ChinaSCINet Update

[Le Todd]

It's French.

[y8225009]

Dr.Wise, now is June 11th.How is going with the trial in KunMing?

[Jim]

Dr.Wise, now is June 11th.How is going with the trial in KunMing?

Dr Young posted this in another thread this morning-

I am heading to China this week to see if we can kick off the trial in Kunming where the patients have been recruited and the paperwork is more or less completed. I hope that this will move things along.

[fightingtowalkc7]

Thank you Dr. Young, for continuing to give us hope.

[lepups3]

Good luck on your trip and thank you.

[sam i am]

I was thinking about something last night and thought to ask D.R Young about it. My question is: what makes a good trial successful or not? As in, you are currently conducting phase 2 trial on your promising therapy, and so how would you know if you should move on to phase 3 or not. What if only 25% of the people that undergone your therapy only had a 25% functional, sensory,bb, or sexual improvement? Would that be considered successful? If your phase 3 trial brought about these results, would it be provided to the public? Because I think that that would be just fine under the circumstances.

So I was just thinking about the thought process from a researchers point of view.

[KIM]

1% is good enough.

[Wise Young]

I was thinking about something last night and thought to ask D.R Young about it. My question is: what makes a good trial successful or not? As in, you are currently conducting phase 2 trial on your promising therapy, and so how would you know if you should move on to phase 3 or not. What if only 25% of the people that undergone your therapy only had a 25% functional, sensory,bb, or sexual improvement? Would that be considered successful? If your phase 3 trial brought about these results, would it be provided to the public? Because I think that that would be just fine under the circumstances.

So I was just thinking about the thought process from a researchers point of view.

sam,

The decision to go ahead depends on the findings.

1. If the phase 2 treatment shows that the treatment causes complications or is not effective, then you stop or change the procedure and retest in another phase 2 trial.

2. Because phase 2 studies are typically not powered sufficiently (i.e. don't have enough patients to achieve statistical significance), the decision whether to proceed to a phase 3 depends on the results. The results may fall into three categories:
• The therapy has no beneficial effects or even a deleterious effect that cannot be attributed to some problem with dose or delivery. In such a case, one would clearly not proceed.
• The therapy has clear beneficial effects that are statistically significant even at phase 2 levels. In such a case, one would proceed to phase 3 but with fewer patients in the trials because the results should be robust.
• The therapy is safe and there is a trend for benefit. In such a case, one would go ahead with the phase 3, probably with more patients in the trials in case the results occur only in a fraction of the patients or the effects are small.

3. If the phase 3 trial shows that umbilical cord blood cells are significantly beneficial, then all subsequent therapies will be compared against that treatment because it would be the first and best therapy that restores function to chronic spinal cord injury.

Wise.

[Scaper1]

Dr. Young, will there be any preliminary result reports this summer? No pressure.

[Leif]

sam,

The decision to go ahead depends on the findings.

1. If the phase 2 treatment shows that the treatment causes complications or is not effective, then you stop or change the procedure and retest in another phase 2 trial.

2. Because phase 2 studies are typically not powered sufficiently (i.e. don't have enough patients to achieve statistical significance), the decision whether to proceed to a phase 3 depends on the results. The results may fall into three categories:
• The therapy has no beneficial effects or even a deleterious effect that cannot be attributed to some problem with dose or delivery. In such a case, one would clearly not proceed.
• The therapy has clear beneficial effects that are statistically significant even at phase 2 levels. In such a case, one would proceed to phase 3 but with fewer patients in the trials because the results should be robust.
• The therapy is safe and there is a trend for benefit. In such a case, one would go ahead with the phase 3, probably with more patients in the trials in case the results occur only in a fraction of the patients or the effects are small.

3. If the phase 3 trial shows that umbilical cord blood cells are significantly beneficial, then all subsequent therapies will be compared against that treatment because it would be the first and best therapy that restores function to chronic spinal cord injury.

Wise.

Norway, supports these visions, are glad to be a partner in trying to find curative treatments on SCI and are working on visionary, infrastructure for SCI users, with neuroscientists, clinicians, mri interventionists and neurosurgeons - to lift these ideas further and further regardless of conservative obstacles, for SCI possible cures. These are facts.

[Christopher Paddon]

Wouldn't it be great if these trials regrow enough fibres to get complete injured people walking? I mean, the spinal cord injured community could use a break and have some great medical news for a change!

[Leif]

Wouldn't it be great if these trials regrow enough fibres to get complete injured people walking? I mean, the spinal cord injured community could use a break and have some great medical news for a change!

Sure, try fix the change then… What is holding the community back?

[Christopher Paddon]

The community has been told there will never be a cure and you must get on with your lives. Getting on with your life takes a lot of energy and once some sort of life is established the idea doesn't seem to occur to many people - I mean, our doctors should be trusted.

I have met one person in a wheelchair in New Zealand who believes in a cure although Noela Valis, whose husband was paraplegic, has fought tirelessly to get clinical trials started in Christchurch.

[sam i am]

sam,

The decision to go ahead depends on the findings.

1. If the phase 2 treatment shows that the treatment causes complications or is not effective, then you stop or change the procedure and retest in another phase 2 trial.

2. Because phase 2 studies are typically not powered sufficiently (i.e. don't have enough patients to achieve statistical significance), the decision whether to proceed to a phase 3 depends on the results. The results may fall into three categories:
• The therapy has no beneficial effects or even a deleterious effect that cannot be attributed to some problem with dose or delivery. In such a case, one would clearly not proceed.
• The therapy has clear beneficial effects that are statistically significant even at phase 2 levels. In such a case, one would proceed to phase 3 but with fewer patients in the trials because the results should be robust.
• The therapy is safe and there is a trend for benefit. In such a case, one would go ahead with the phase 3, probably with more patients in the trials in case the results occur only in a fraction of the patients or the effects are small.

3. If the phase 3 trial shows that umbilical cord blood cells are significantly beneficial, then all subsequent therapies will be compared against that treatment because it would be the first and best therapy that restores function to chronic spinal cord injury.

Wise.

Thanks for replying Dr. Young.. from your response, it means that proceeding to phase 3 is even bigger news than we expected. Also, from my understanding, your phase 3 can host a big number of patients or a small number of patients depending on your phase 2 results, right?

Thanks again and good luck and I hope that 2013 will have something ready for us.

[Wise Young]

We are holding workshops and consensus conferences to look at new therapies. Whether umbilical cord blood mononuclear cells (UCBMC) and lithium are effective or not, we will need to look for new therapies. If UCBMC+lithium restores function, we need to look at other therapies, such a neural stem cells and see if they are better than UCBMC+lithium. Other possibilities include the olfactory ensheathing glia (OEG) and perhaps combinations of UCBMC and OEG. There is also the possibility of iPS or induced neuronal progenitors (iNP), etc. We may consider combining UCBMC+lithium and nogo antibody, chondroitinase, nogo receptor blocker, cethrin, and other axonal inhibitor blockers.

On May 5-8, 2012, at the Fifth International Spinal Cord Injury Treatment and Trials Symposium (ISCITT5) and International Association of Neurorestorology (IANR) in Xi'an, we will be inviting several companies that have cells that are ready for clinical trials to present to us. So, we are systematically looking for new candidate therapies to compare against UCBMC+lithium to test in 2013.

Wise.