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Welcome to the first of our Ask-a-Scientist Series!
A few weeks ago, we posed a question to our Online Community: if you could ask a scientist anything about T1D research, what would you like to know?
We received many great questions — here, I will tackle a few questions related to autoimmunity and T1D research.
What’s so hard about finding a cure for autoimmune diseases like T1D?
Several Online Community Members asked about the autoimmune process behind T1D and how this relates to the lack of progress on a cure for T1D.
What makes it so difficult to cure an autoimmune disease like Type 1 Diabetes?
Why [is it] so difficult to cure ANY autoimmune disease?
To answer these questions, we need to understand the ‘what’ and the ‘why’ behind autoimmune disorders.
Brief Overview of Autoimmune Diseases
T1D is an autoimmune disorder. The insulin-producing beta cells in the pancreas are attacked by one’s own immune system. Without functional beta cells, your body cannot properly produce six different hormones, including insulin.
Generally, to cure a disease, we need to be able to stop the disease process from happening and to do this, we need to know not only what happens but why it happens. In autoimmune diseases, we understand what happens: the body mistakes its own cells or tissues as something ‘foreign’ — in the same way it would identify a virus, bacteria, or anything else that shouldn’t be there.
There are many autoimmune disorders which share this underlying process, but their damage to the body and symptoms are vastly different, for example:
- In T1D, the body attacks insulin producing cells which results eventually in dangerously high blood glucose levels — and it is eventually fatal if left untreated.
- In rheumatoid arthritis, the body mainly attacks the joints leading to pain, stiffness, and can lead to deformity.
- In systemic lupus erythematosus (SLE or ‘lupus’ for short), the body launches a systemic (or whole body) attack leading to different symptoms depending on where it hits – but it usually has some skin, joint, and organ involvement.
One member shared:
No one else has type 1 diabetes in [my family], but [family members] have many other [autoimmune conditions]: Addison’s Lupus, MS, and thyroid conditions.
Perhaps surprisingly, more than 85% percent of T1D diagnoses are in people with no family history of the disease – and recent a study suggests this number may be as high as 90% of people diagnosed with T1D., but the process behind autoimmunity is thought to be — at least partially — related to genetics (or ‘heritable’). While you may not have anyone else in your family with T1D, chances are you have a relative with a different autoimmune condition.
But it’s not enough that people have a genetic susceptibility to an autoimmune disorder. Something else needs to happen to trigger this process to begin. These triggers might be stressful events, viruses, or any complex combination of these and other environmental causes.
Unfortunately, we don’t have clear answers for what that something else is or in what amount one needs to be exposed to it to definitively trigger an autoimmune response. Because we don’t really know what we’re up against (and some of what we’re up against are normal things in the environment — like viruses and stress), we don’t have advice on how to prevent this process from starting.
We do know that once the autoimmune process begins, it’s nearly impossible to stop.
Instead, the treatment for autoimmune disorders typically involves managing the symptoms of the disorder and — if appropriate — trying to reduce activity in the immune system to prevent further damage. In T1D, treating the symptoms is predominately treating high blood glucose by managing the lack of insulin (caused by the damaged beta cells), but more and more people with T1D being treated with medications like GLP-1 agonists for other affected hormones.
What research is being done now in autoimmune disorders like T1D?
Is anything being done in DNA/Gene research to cure autoimmune disorders (stop the immune system from attacking organs, endocrine systems, the human body?
Yes, work is always being done. There is current work on learning more about how specific immune cells might be able to prevent or delay autoimmune processes in some illnesses, like this work on the role of certain T-cells (special immune cells) in Multiple Sclerosis.
However, this work is in its infancy.
Although we have a lot more information than we used to, autoimmunity appears to be a very complicated mix of genes and environment. In short, the scientific community is still far from having the answers to try to stop the autoimmune process entirely. But we are always making progress and sometimes progress occurs in leaps instead of steps.
While we may not be able to prevent it, we can use what we know from other illnesses and try to suppress the immune system or modify cells in the immune system to slow down the autoimmune attack. In T1D, this is exactly how teplizumab works to delay diagnosis.
Still, there are a lot of unknowns. And, after the autoimmune process begins – particularly after it has done a lot of damage, as is the case with T1D – medicine and science does not have a good way to reverse it.
Bypassing the Autoimmune Problem
Although this was not a member question, you may wonder now does it matter whether we know why autoimmune diseases happen?
We know in T1D the immune system attacks beta cells in the pancreas and creates a lot of problems, so can’t we use that knowledge to find a cure? The answer is yes — but only to a point.
Beta cell transplants and pancreas transplants replace the non-functional cells or organ in T1D and suppress the body’s immune systems to reduce or eliminate the need for insulin. There are a lot of challenges to this approach. First, it’s still experimental and not widely available. Second, because we don’t know the why behind autoimmunity, we can’t stop the autoimmune process from occurring again. For many folks, even if it was available, the risks and costs of transplant (including having to be immunosuppressed and undergo multiple procedures every few years) might outweigh the ‘standard of care’ approach to T1D management.
More recent clinical trials are in the works to improve upon islet cell transplant therapies, like Vertex’s VX-880 clinical trial and the recently announced VX-264 clinical trial — which protects the transplanted cells from the T1D immune attack by protecting them in a small device. This groundbreaking research from Vertex has already led the first recipient of VX-880 to achieve insulin independence within one year of starting the trial.
Scientific Advancements in T1D
Although we may be farther away from a cure than we’d like, there have been many advances even in the past two decades in T1D management aimed at symptom management, improvement of glucose levels, and prevention of complications. Thanks to these advancements, the gap in life expectancy of people with T1D is beginning to close – and many folks with T1D are living longer with fewer complications than in years past.
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