This is fascinating! I wonder if something like this could get the pancreas to begin producing insulin. Type I Diabetes is no joke. It could save tens or hundreds of millions per year, not to mention multiple injections per person per day.
hemophilia a is a disease caused largely by deficiencies in one specific protein. massive technological breakthroughs in recent years have enabled scientists to deliver genes to human cells to consistently, accurately and sustainably produce specific proteins. so treating diseases like hemophilia a has become tractable
however, autoimmune diseases are much more complex and there is no single protein or molecule that causes the diseases. in type 1, people have been trying to discover 1) the "triggering" event and 2) the autoantigens that cause the disease for years, and it looks like there is no "silver bullet". current drug technology can target only one, or sometimes two, disease-causing molecules, so for a disease where many molecules each play a small part, we dont have the answer yet
there are a few promising type 1 companies (semma, viacyte, others) trying to turn stem cells into insulin producing cells, but those cells are often rejected by the bodys immune system. some other companies are increasing the number of "regulatory T cells", basically T cells that inhibit autoimmunity (caladrius, celgene / delinia, parvus, lilly / nektar); these have shown promise but it isnt likely theyd be curative, as they just stop the autoimmune attack but cant regenerate beta cells that are already lost
if youre interested in type 1 research, diatribe is a great website that aggregates the latest science and presents it in a way that is easy for non-scientists, specifically patients and their families, to understand https://diatribe.org/
If not, gene therapy can only be applied pre-emptively, I guess, unless we somehow manage to isolate the mechanism of how measles resets the immune system, and apply it selectively to the part that has learned the autoimmune behaviour[0].
[0] http://www.sciencetimes.com/articles/6168/20150508/measles-w...
the genes involved in autoimmune disease are not as well understood as in some other diseases, and the individual contribution of any one gene is pretty small in autoimmune disease (compared to, say, spinal muscular atrophy). so as you say, a gene therapy approach might not be the right tool for the job.
a lot of current approaches focus on "retraining" immune cells to be "good" rather than "bad" cells. here's a blog post about one of the leading startups in the space which was acquired by celgene, a large biotech company, for almost $800M earlier this year, just 3 months after raising their series a: https://lifescivc.com/2016/09/re-balancing-immunity-via-regu...
Nothing like all this great technology that could save your life that is foiled simply by the type of glue used by the manufacturer.
I often hear it said that problems such as this are caused by cures being less profitable compared to drugs, because drugs are purchased repeatedly, cures only once. But competition is supposed to solve that problem. Is inappropriate regulation the real problem?
And despite all of the amazing technological and medical advances over the past 10-20 years, the best insulin therapy in the world is still comically shitty when compared to a healthy pancreas.
Anyway, it could be way worse and I'm grateful for what is available to us today. Still hope for better days ahead.
"Modified blood stem cells reverse type 1 diabetes in mice"
https://www.diabetes.co.uk/news/2017/nov/modified-blood-stem...
And type 2 got cured by an extreme diet in 86% of patients:
https://sciencealert.com/extreme-diet-reverse-type-2-diabete...
the most common animal model for type 1, the NOD mouse model, is notoriously bad. studies have shown that loud music cures diabetes in these mice. mice raised in one lab get diabetes differently than mice raised in another. the immune systems of these animals are very fickle and almost any environmental perturberance can change an immune response
I realize the mind recoils instinctively at these ideas, but it seems so frustrating and horrific that we have to delay life saving medical advanced due to insufficient experimental subjects.
safety studies are not the largest driver of cost and duration of drug development. failing a later stage human study that assess effectiveness is a much bigger needle mover in terms of cost, as is the arduous and painstaking process of early drug discovery and development. human studies of effectiveness cost $15-500M+ and can take 3-7 years. putting a drug in humans that has a low probability of success is the most value destructive thing you can do in pharma. the other big cost driver is early research in drug discovery and development. understanding the biology and chemisty, developing assays, and screening compounds can cost $15-20M+ and take 5+ years. by comparison, animal safety studies cost maybe $5M and take a year or two
For more context:
There's an important distinction to be drawn between two main types of animal models: models assessing drug "safety", and those assessing "effectiveness". the models of effectiveness are very poor and of questionable value in some cases, although they are often the best we have. animal models of effectiveness are not literally required for FDA approval, although in reality they pretty much are
animal models of safety, however, are required by FDA, and rightly so. they are of much greater value. generally you must do studies in a small species like a mouse as well as a non-human primate. these "toxicology" studies basically entail dosing animals with huge amounts of drug, way more than you'd dose in humans, and then seeing what doses are not toxic. this informs the first dose youd do in humans
the initial human studies are not done to study effectiveness, but to study safety. based on the data from your tox studies in animals, as well as other studies, you gradually dose cohorts of patients with higher doses, watching carefully for safety signals.
This trial was phase I/IIa which I believe means it's combining safety in humans with initial efficacy data. The thinking is, the real treatment is a single dose. If we give participants less than a full size dose we don't learn much from that. If we give them a real dose, we might as well see if it works.
So their endpoints were firstly how is the treatment tolerated, then secondly does it reduce the need to take clotting factors? Hence this good news story.