A Phase-II Abatacept Trial to Prevent T1D in At-Risk People Finishes Recruiting
You can read my previous postings on Abatacept here:
https://cureresearch4type1diabetes.blogspot.com/search?q=Abatacept
Abatacept is a treatment that prevents T-cells from becoming activated. Presumably, for type-1 diabetes, it works by blocking the "bad" killer T-cells from activating. This drug is already approved for use in rheumatoid arthritis when other treatments have failed, and is marketed as Orencia.
This is a large (212 person) trial started in 2013, and recruited it's final person in February 2020. Since they plan to collect data from each person for at least a year, this trial is likely to finish in February 2021. The goal of this trial is to see if Abatacept can be given to people before they are diagnosed with T1D, and prevent or delay the onset of the disease. The people enrolled in this study have tested positive for two or more autoantibodies, so they are almost certain to be diagnosed with type-1 diabetes sometime in the next 10 years.
History and Discussion
Abatacept has already been tested on people with T1D in their honeymoon period, and the results were that people treated with Abatacept continued to generate about 50% more of their own insulin, than those not treated. The amount of insulin generated years after diagnosis is pretty small, so the actual difference is half of a tiny number. One way to view these results was that Abatacept delayed the "end of honeymoon" by 9.5 months. Someone who got the drug generated the same amount of insulin 36 months after diagnosis as someone who did not get the drug generated 27 months after diagnosis.
So this result is similar to the recently published Teplizumab results, although the Teplizumab results were a little stronger.
Clinical Trial Registry: https://www.clinicaltrials.gov/ct2/show/NCT01773707
Phase-I Clinical Trial of Substance P Is Over Two Years Overdue
Substance P is a peptide (a part of a protein) which is used by several different organs and for several different purposes. Research done in the early 2000s found that a specific type of neuron (called "TRPV1(+) pancreatic sensory neurons") control islet inflammation and insulin resistance. Removing these neurons from NOD mice prevented diabetes from developing. Injecting NOD mice with Substance P, which affects these neurons, has increased beta cells in mice, and also lowered inflammation. This clinical trial tested this same treatment in people, rather than mice.
The trial started in 2016 and was expected to finish in 2017. The clinical trial record has not been updated since 2016, and I cannot find any published data from this study or the company that sponsored it (Vanilloid Genetics Inc). Twice I've sent email to the researchers running this study, and I have gotten an "on vacation" email back from one of them, so I know they are still at the University where this research was done, but I have not gotten any other reply. I have not found any corporate email info for Vanilloid Genetics.
So therefore, I'm going to remove this study from my list of active studies. If I ever see positive results, then I'll put it back on.
Clinical Trial Registry: https://www.clinicaltrials.gov/ct2/show/NCT02820558
PROCHYMAL® (Adult Stem Cells) for the Treatment of Recently Diagnosed T1D
Way back in 2012, I reported on an unsuccessful Phase-II- study of PROCHYMAL (adult stem cells) by a company called Osiris. However, in March 2020, the clinical trial record for that trial (now completed for 8 years) was updated. The changes made were all small, and I would describe them as "cosmetic". I don't know what this means. If it means anything at all. But it is unusual for the clinical trial record for a trial completed so long ago to be changed.
Clinical Trial Record: https://www.clinicaltrials.gov/ct2/show/NCT00690066
Changing Terminology: At-Risk Instead of Presymptomatic
In the past, I have used the term "presymptomatic" to describe people who have two autoantibodies, but none of the classic signs of type-1 diabetes. TrialNet has published data over the last few years that shows that just about all of these people will have symptoms of T1D within 10 years. Therefore many researchers consider that people with two autoantibodies, but no other symptoms, really do have T1D, it is just that they don't have symptoms, yet. So I used the term presymptomatic to describe the studies being done on these people.
But "presymptomatic" is a mouth full, and it is not a natural sounding word. Also, people who don't inject insulin and don't count carbs don't think of themselves as having T1D at all. So therefore, I'm going to start using the term "at-risk" to refer to these people, and the clinical trials that enroll them. I think it is a more natural English phrase to describe people who are not showing symptoms yet.
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 or JDCA news, views, policies or opinions. 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.
