Alpha Difluoromethylornithine (DFMO) Starts A Phase-II? Trial in Honeymoon Type-1 Diabetics

This trial started recruiting in April 2015, but I missed it at that time, so I'm blogging about it now.

Alpha Difluoromethylornithine (DFMO) is approved for two quite different issues.  The first is to remove/prevent facial hair in women, while the second is to treat sleeping sickness.  Neither of these are related to type-1 diabetes.   However, this drug was effective in lowering diabetes rates in NOD mice (which are predisposed to an autoimmune diabetes, similar to human type-1 diabetes), and that is what motivated this trial.

The Trial

This trial is recruiting people 12-40 years old, who have been diagnosed with type-1 diabetes in the last 2-8 months.  People will be divided up into 4 different groups, each getting a different dose.  Within each dose group, there will be 6 people who get the treatment and 3 people who get a placebo (and will be a control group).

Primary endpoints are safety related, while other endpoints (such as C-peptide) are targeted at seeing if it works.

The researchers hope to finish gathering data in Dec 2019, so publication should be some time in 2020.  (Although the primary outcome data will be collected in Dec 2018, so there is the possibility of partial results in 2019.)

This trial is being run in three locations:

Riley Hospital for Children: Indianapolis, Indiana, United States, 46202
Contact: Stephanie Woerner, FNP    317-944-2573    sestein@iu.edu 

Women and Children's Hospital of Buffalo: Buffalo, New York, United States, 14222
Contact: Michelle Ecker, RD, CDN, CDE    716-878-7609    mecker@upa.chob.edu 
       
Children's Hospital of Wisconsin: Wauwatosa, Wisconsin, United States, 53226
Contact: Joanna Kramer, CCRC    414-955-8486    jkramer@mcw.edu 


Discussion

Although listed as a Phase-I trial, in some ways it is more like a phase-II study, which is why I've listed it as a Phase-II? study.  The question mark signifies that this drug has never been tested on people with type-1 diabetes before.  I consider it Phase-II because it is the right size (42 people) and the right purpose (testing multiple different doses) to be a Phase-II study.

Clinical Trial Record: https://clinicaltrials.gov/ct2/show/NCT02384889
Results in mice: https://www.ncbi.nlm.nih.gov/pubmed/23846959

Personal Note

I am always surprised how much researching type-1 diabetes teaches me about other subjects.  One part of the story of DFMO is both fascinating and horrifying.  After being developed in the 1990s, it was used intravenously as a treatment for sleeping sickness.  However, in 1995 production was discontinued, because saving lives in Africa was not profitable. Various non-profits lobbied the producer (a big pharma firm) to restart production, but to no avail.  Meanwhile, researchers discovered that it "cured" facial hair in women.  The cream used to do this went into production in 2000, and has been on the market since then.   Under intense pressure from the World Health Organization and non profits, the big pharma company eventually agreed to provide the drug for free to non-profits for distribution to African sleeping sickness patients, starting in 2001.  My best guess is that the lack of drug between about 1995 and 2001 killed between 40,000 and 110,00 people (very roughly).

The key moment in making the drug available may well have been a "60 Minutes" episode.  This is an American weekly news show.  It showed patients dying of sleeping sickness, or enduring painful IV treatments, which was the best non-DFMO drug available, and then followed it up with DFMO's American TV add for removing facial hair.  The juxtaposition highlighted the money-at-the-cost-of-lives reality of the situation.

Joshua Levy
http://cureresearch4type1diabetes.blogspot.com
publicjoshualevy at gmail dot com
All the views expressed here are those of Joshua Levy, and nothing here is official JDRF, JDCA, or Bigfoot Biomedical news, views, policies or opinions.  In my day job, I work in software for Bigfoot Biomedical.  My daughter has type-1 diabetes and participates in clinical trials, which might be discussed here. My blog contains a more complete non-conflict of interest statement. Thanks to everyone who helps with the blog.