Sunday, January 4, 2009

Best News of 2008 in Clinical Trials to Cure Type-1 Diabetes

Here is my list of Best News of 2008 in Clinical Trials to Cure Type-1 Diabetes. I've based this list on these simple rules:
  1. The best news is a cure, or a measurable improvement in BG, A1C, or insulin used.
  2. The bigger the drop (in BG, A1C, or insulin used) and the more people helped, the better.
  3. Results for non-honeymoon diabetics are better than the same results for honeymooners.
  4. Results from later phase trials are better than results from earlier phase trials.
So with that in mind, here are five "best news" and a few special mentions. Note that I have not parked one "best" another "second best" etc. They are all really good news in different ways:

The "Big 5"

LCT finishes a phase-I trial, and shows some results
This news had it all: shows improvements for type-1 diabetics, works for people who have had diabetes for a long time, and marks the end of a clinical trial, with data we can see. Insulin usage dropped 24%, and almost everyone's A1C number went down. One person was cured (used no insulin at all) for a period of months.
Hope for next year: The start of a bigger trial and to see longer term data from this trial. Publication in a peer reviewed journal would be nice, too!

More data here: http://cureresearch4type1diabetes.blogspot.com/search/label/LCT
and here: http://joshualevy.pbwiki.com/DiabetesCureReadyForHumanTrials#LivingCellTechnologiesDiabecell

Burt Publishes 3+ year follow up data for their phase-I trials
This is the most amazing results seen yet: more than half of the patients where completely and permanently cured (low BG, A1C, and no more insulin needed). Some for over 3 years! Safety concerns linger, and it was honeymoon only.
Hope for next year: Start a phase-II trial for this, or better understand the safety issues.

More data is here: http://cureresearch4type1diabetes.blogspot.com/search/label/Burt
and here: http://joshualevy.pbwiki.com/DiabetesCureReadyForHumanTrials#UniversityofSaoPauloBurt

Diamyd starts two large phase-III trials
These guys are the closest to mass marketing a type-1 cure for some people. Their treatment shows improvements (lower BG, A1C and insulin usage) for many people, and might cure some people. It has only been tested on honeymooners, but once it is approved for general use, anyone will be able to try it. Their phase-II trails suggested that Diamyd use doubled the amount of surviving insulin producing beta cells. This means they require less insulin, and are very likely to have far fewer complicatoins in later life.
Hope for next year: See some intermediate results from this phase-III trial, or test it on non-honeymooners, or both!

More data is here: http://joshualevy.pbwiki.com/DiabetesCureReadyForHumanTrials#DiamydDiamydTorGAD65
and here: http://diamyd.blogspot.com/

ToleRx starts a phase-III trial
These guys are also making steady progress on a treatment that may cure some people and improve the health of many more. It has been shown to lower BG, A1C, and insulin requirements, but has only been tested on honeymooners. People treated in phase-II trails required significantly less insulin even after 18 months, compared with untreated people. (Although they did not have a big milestone this year, MicroGenics is testing a similar CD3 based cure, and is only a half step behind ToleRx.)
Hope for next year: Maybe finish this phase-III trial, or test it on non-honeymooners, or both!

More data is here: http://cureresearch4type1diabetes.blogspot.com/search/label/tolerx
and here: http://joshualevy.pbwiki.com/DiabetesCureReadyForHumanTrials#TolerrxTRX4orChAglyCD3

Gitelman and Osiris both started phase-II trails.
These are different immunology based cures, which are both at very similar point in their development. They are both being tested on honeymoon diabetics. Gitelman's research is based on previous phase-I research aimed a curing type-1 diabetes. The Osiris treatment has been tested in the past on several other immunological diseases, but this is the first time it has been tested as a cure for type-1 diabetes.
Hope for next year: probably nothing, but in 2010 good results from both!

More data is here: http://cureresearch4type1diabetes.blogspot.com/search/label/Gitelman http://cureresearch4type1diabetes.blogspot.com/search/label/Osiris
and here: http://joshualevy.pbwiki.com/DiabetesCureReadyForHumanTrials#NIAID/ITNGitelmanThymoglobulin/ATG http://joshualevy.pbwiki.com/DiabetesCureReadyForHumanTrials#OsirisTherapeuticsPROCHYMAL

More Generally

While the above paragraphs describe some specific research milestones of 2008, there are also more general good news out there. For example, I've been covering human trials aimed at curing type-1 diabetes for several years now, and every year I do it, there are more and more trials to keep track of. This year, for example, is the first year that there has been more than 1 phase-III clinical trial active. There were three of them at the end of the year. Will they all pan out? Probably not, but more trials means a bigger chance of one success. And we only need one success.

I'm also very intrigued by the new findings this year, about the importance of inflammation as a possible causative factor in type-1 diabetes. Although none of this research has yet led to clinical trials, it is clearly something new and different. As it is only a year old as a research direction, there is still lots of time for it to grow into something useful. Watch these guys:
http://joshualevy.pbwiki.com/DiabetesCureReadyForHumanTrials#HarvardKoulmandaandStromAAT





3 comments:

DiamydBlog said...

Hi Joshua,
I think beta cell regeneration treatments will be a large part of adapting honeymoon treatments to non-honeymooners.

I also put big hopes in the Diamyd prevention trials. This is different from the Phase III trials and may prove to actually prevent or delay the outbreak of T1D in high risk patients. If they start in mid 2009, intermedient results should be ready by 2011, at the same time the Phase III trials are ready.

I don't agree with people saying you can't "vaccinate" all high risk patients. You can, one you have a good enough safety profile.

By the way, there will be no intermedient results from the two Phase III trials. End results are expected in Q3 2011.

Good job keeping track of all this research!

Joshua Levy said...

I agree that some form of regeneration will turn every honeymoon cure into a cure for everyone.

As for the prevention trials, if those pan out, it will be great for the people who get type-1 in the future, but my interest is for the people who already have it, and prevention trials will not help those people at all.

I agree with you that people who say you can't vaccinate all high risk people are completely wrong. We vaccinate just about everyone for many different things. Vaccinations are the only way to completely remove a disease from the population (like smallpox, and like we are close to for polio). And Diamyd's treatment is perfectly made for vaccinations, if the prevention trials work. Once it is approved for use, it will be easy to vaccinate the brothers, sisters, and children of type-1 diabetics. There are 1000s of such people each year in the US. Based on that large population, well be able to run large safety and effectiveness tests, and decide who else should be vaccinated. Getting people to sign up will be easy, because it will already be approved, and because seeing a close relative with type-1 will encourage people to try the vaccine.

Anonymous said...

Joshua-

This September I was dosed in Tolerx's Phase III trial. As of November, I no longer have needed to take insulin. Now, it is a double blind study and I cannot say for certain I received the drug, but I think I have good reason to believe I did. Having been diagnosed as type-1 for nearly three months before being administered the drug, I can see how difficult and time consuming it can be to properly manage your glucose. It has an affect on every aspect of your life. I am so grateful for having the chance to be a part of this study. I hope other people who receive treatment share similar successes. And I hope this drug helps many many more.

Thanks. I will certainly continue to follow your research.