4 ms·

There are lots of people working on regenerative medicine and gene therapy for autoimmune diseases (ive worked with several researchers focused on type 1), but

There are lots of people working on regenerative medicine and gene therapy for autoimmune diseases (ive worked with several researchers focused on type 1), but unfortunately the field isnt there yet. 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/ https://diatribe.org/

baldfat 9y ago
Thankfully artificial pancreas are looking like a good option until we can get to curing type 1. Not only does it save the number of shots like pumps we have today, but it can measure the sugar level and keep the swings down to a minimum. http://www.jdrf.org/research/artificial-pancreas/ http://www.jdrf.org/research/artificial-pancreas/
- aaavl2821 9y ago
  yeah artificial pancreas seems to be the most promising near-term development. the field has suffered from a lack of funding for translational research as VCs have been focused on other diseases, but hopefully that changes. semma (a startup turning stem cells into beta cells) raised $114M last week
  - thingification 9y ago
    Why are they focused on other diseases? Diabetes is hardly a rare disease, nor one with a low impact on its sufferers. So if what you say is true, what is it that's broken about the market for diabetes treatment? 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?
- test6554 9y ago
  They seem good as long as the patient's skin is not allergic to the adhesive used to attach it to the body. I know someone who had an insulin pump that worked perfectly except after a day or two, they would get a rash on the skin and the area would swell up blocking the delivery of insulin. Nothing like all this great technology that could save your life that is foiled simply by the type of glue used by the manufacturer.
  - baldfat 9y ago
    They go under the skin (Most of the plans I have read)
vanderZwan 9y ago
From what I understand, autoimmune diseases are a mix of genetic predisposition and "learned behaviour" by the human immune system, i.e. some stressor making the immune system mislabeling a part of the own body as hostile. Once this is "learned", would removing the genetic predisposition even have an effect? Do we even know how the immune system "remembers" previous illnesses? 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-weaken-immune-system-years.htm http://www.sciencetimes.com/articles/6168/20150508/measles-w...
- aaavl2821 9y ago
  We actually have a pretty decent understanding regarding how the immune system remembers illnesses, although it is above my pay grade. if interested, read up on "adaptive immune system" 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-regulatory-t-cell-potentiation-introducing-delinia/ https://lifescivc.com/2016/09/re-balancing-immunity-via-regu...

baldfat 9y ago

Thankfully artificial pancreas are looking like a good option until we can get to curing type 1. Not only does it save the number of shots like pumps we have today, but it can measure the sugar level and keep the swings down to a minimum. http://www.jdrf.org/research/artificial-pancreas/ http://www.jdrf.org/research/artificial-pancreas/

aaavl2821 9y ago
yeah artificial pancreas seems to be the most promising near-term development. the field has suffered from a lack of funding for translational research as VCs have been focused on other diseases, but hopefully that changes. semma (a startup turning stem cells into beta cells) raised $114M last week
- thingification 9y ago
  Why are they focused on other diseases? Diabetes is hardly a rare disease, nor one with a low impact on its sufferers. So if what you say is true, what is it that's broken about the market for diabetes treatment? 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?
test6554 9y ago
They seem good as long as the patient's skin is not allergic to the adhesive used to attach it to the body. I know someone who had an insulin pump that worked perfectly except after a day or two, they would get a rash on the skin and the area would swell up blocking the delivery of insulin. Nothing like all this great technology that could save your life that is foiled simply by the type of glue used by the manufacturer.
- baldfat 9y ago
  They go under the skin (Most of the plans I have read)

vanderZwan 9y ago

From what I understand, autoimmune diseases are a mix of genetic predisposition and "learned behaviour" by the human immune system, i.e. some stressor making the immune system mislabeling a part of the own body as hostile. Once this is "learned", would removing the genetic predisposition even have an effect? Do we even know how the immune system "remembers" previous illnesses? 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-weaken-immune-system-years.htm http://www.sciencetimes.com/articles/6168/20150508/measles-w...

aaavl2821 9y ago
We actually have a pretty decent understanding regarding how the immune system remembers illnesses, although it is above my pay grade. if interested, read up on "adaptive immune system" 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-regulatory-t-cell-potentiation-introducing-delinia/ https://lifescivc.com/2016/09/re-balancing-immunity-via-regu...