The biggest diabetes research meeting in the world is the American Diabetes Assoication (ADA) Science Sessions, which are in June (this year: June 8-12). It is followed by the big European Association for the Study of Diabetes (EASD) Annual Meeting (this year: Oct 1-5). So expect a lot of news over the next few months. We are already seeing press releases from companies publicizing what they will report at these large meetings.
Phase-II Results from Autologous Hematopoietic Stem Cell Transplants by Li at Nanjing UniversityThis is the third group to do a Burt-like cure for type-1 diabetes, and get similar results.
You can read my previous blogging here:
But the basic summary is this: for recently diagnosed type-1 diabetics, this is the closest to a cure there is. People have gone years after this treatment without needing to inject insulin. This result has been reproduced in both Poland and China, after originally being done in Brazil. The down side? Safety. This treatment requires a full immune reboot. Very approximately: They kill off your old immune system, and you regrow a new one, so for a few days (or a few weeks) you are very susceptible to infections and you stay in a special isolation ward in a hospital. As far as I know, no one has died, but the risk is there. Plus, there are long term risks.
From the abstract:
After [treatment], 11 of 13 patients required significantly reduced doses of insulin for adequate glycemic control, accompanied by reduced levels of glycosylated hemoglobin but increased C-peptide concentrations. Three patients achieved exogenous insulin independence for 7-54 months. [Meaning one person was functionally cured for 4 1/2 years.]Abstract: http://www.ncbi.nlm.nih.gov/pubmed/22419704
Clinical Trial Record: http://www.clinicaltrials.gov/ct2/show/NCT01341899
Alefacept Finishes Enrolling a phase-II Clinical TrialThis study is also called "T1DAL", which I'm sure is pronounced "tidal". This trial is being run by the Immune Tolerance Network, and they finished enrolling people in the trial on 30-April-2012.
This drug targets the immune system's T cells, and is already approved for treating "plaque psoriasis" which is an autoimmune disease similar to type-1 diabetes. It has a good safety profile there. The hope is that by giving it to honeymoon type-1 diabetics, beta cells will be preserved.
Why is finishing enrollment important? For two reasons. First, because it is now possible to predict when they will finish collecting data. (This study collects data for 2 years, so they should have it done by May-2014.) Second, because much of the uncertainty that surrounds clinical trials, is involved with recruiting participants. It is often unclear how hard it will be to recruite people, and how long it will take. But at this point, all that uncertainty is behind the researchers. From now on, it is just gather data, then analize data, and then publish data. Researchers have a lot more control over those later stages, then over recruiting people in the first place.
Trial Record: http://www.clinicaltrials.gov/ct2/show/NCT00965458
Company X Gets Orphan Drug Status for Drug Y
I often see headlines like the one above. The most recent was "Andromeda Announces FDA Orphan Drug Designation for DiaPep277". I don't report that as news in this blog, because I don't consider it scientific progress. Orphan Drug is a designation used by the USA and the EU. Companies with such drugs have the legal right to sell them for a longer period of time without competition. The idea is that the drugs target rare conditions, and companies could not profitably develop them, if they only got the benefit of "normal" legal protection, because the market would be too small. So, if the condition is rare enough, then the government says it is an orphan drug, and then the company gets a longer period of time before others can manufacture the drug. Now, type-1 diabetes is a very common disease (over 1 million people in the US alone, by most estimates). So a type-1 diabetes drug would not qualify as orphan. But drugs that only work in the honeymoon phase can get orphan drug status, because only a couple of tens of thousands people are in the honeymoon phase in the US at any time. I'm not sure that was a result the people who originally wrote the law would have approved, but that is how the law has been implemented.
But in any case: this is good news for the company, because it mean bigger revenues (or longer revenues) if the drug is successful. But it does not say anything about the chances of success for the drug. It says more about the disease being treated, than the drug with the status.
Omni Announces Some Phase-I Results for AAT
AAT is an anti-inflammatory drug, which the body makes naturally, and which is already FDA approved for people who have a rare condition where a person don't make enough of it on their own. This treatment is based on the idea that treating inflammation can cure/prevent/treat type-1 diabetes.
There are several trials currently underway with AAT. Omni recently announced some information about the first 12 people treated as part of their trial. This trial is continuing to enroll more people. This is a intermediate report of progress. I'm hoping there will be more information published at ADA or EASD, but this is from their press release. Note that they are reporting on 12 patients (all treated, no untreated comparison group), and that 7 of the patients were in their honeymoon phase, and 5 were not.
The treatment has been very safe and well tolerated by all the study subjects.
Six months following the initial screening four of the seven [honeymoon] patients showed increased C-peptide levels, whereas most Type 1 diabetics progressively lose their ability to produce endogenous insulin, and, therefore, demonstrate progressively decreasing levels of C-peptide.
Three of the seven [honeymoon] patients displayed decreased dependence on insulin during the 3-6 month period following the start of their eight week AAT therapy.My interpretations (opinions) of the results:
- They don't provide any numbers: no c-peptide numbers and no insulin number. I've come to believe this is a bad sign. I hope they publish this data soon.
- For established type-1 diabetics, it looks like AAT had no positive effect at all, in this small sample.
- For honeymoon type-1 diabetics, Omni is trying to be optimistic, and it does look like the treated group did better in some ways than average untreated people. However, the reporting is vague, no numbers are provided, few patients were treated, and things are naturally unsettled in the honeymoon phase, that it is very hard to see the data described so far as a successful result. So I would not get excited about AAT yet.
Press Release: http://www.omnibiopharma.com/_literature_136822/Omni_Bio_Diabetes_Trial_Data_-_May_30,_2012
A Personal NoteSeveral months ago my wife and I started looking for a new house. That takes a lot of time, and actually buying one, even more so. I'm happy to say that we were successful, and are in the middle of moving from one Silicon Valley town to another. That is why I have not had the time to publish any blogs for a while. It now looks like I will move into the new house in mid-June (wooo-hooo!). I hope to return to my normal blogging sometime in late July or August.
I know there are more things going on in the world of research aimed at curing type-1 diabetes than I have included in this blog, and I'm sorry it will take a while to get through the backlog. I'm sure there will be a land side of new information from the upcoming scientific conferences, as well.
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF or JDCA news, views, policies or opinions. My blog contains a more complete non-conflict of interest statement.
Clinical Trials Blog: http://cureresearch4type1diabetes.blogspot.com
Cured in Mice Blog: http://t1dcuredinmice.blogspot.com/