Autoimmune disorders are diseases in which the body mistakenly attacks itself, causing diseases such as diabetes mellitus or multiple sclerosis for example. Scientists strive to combat these diseases by finding therapies that treat or neutralize the immune response against self cells. However, a barrier that these scientists often face is that neutralization doesn’t always just apply to the part of the immune system that is attacking the wrong cells but often attacks the cells that are working properly which leaves the body vulnerable to a plethora of sicknesses.
A team in Utah is attempting to separate the effects of different therapies on cells of the immune system that cause damage while keeping the normally functioning cells in tact. Their study, which was conducted on mice, seeks to target a surface protein PD-1 which plays a key role in regulating the immune system.
Both B and T cells express this regulatory protein; however, in those with autoimmune disorders this protein become ineffective. The team focused on creating a molecule that would deplete the store of this protein in the B and T cells in order to avoid long-term damage done by this malfunctioning cells.
The molecule that they sought to create was composed of three parts: an anti PD-1 antibody, an albumin-binding domain protein, and the Pseudomonas exotoxin. The three components work together to bind the cells expressing PD-1 and killing them, while maintaining the ability to continue to circulate through the blood stream to find other damaging cells.
The molecule was shown to delay the onset of type 1 diabetes in mice from the usually 19 weeks to 29 weeks. However, the most significant results were in mice with multiple sclerosis who showed stopped paralysis progression and even in some cases regained the ability to walk again.
Although these results are very optimistic, the study was only done on a limited number of a mice and has not been conducted in humans. This therapy might be beneficial if a similar PD-1 protein is found in humans.
Although scientists do not exactly understand how the body attacks the beta cells in patients with type I diabetes, scientists have also tried therapies that help to protect these beta cells by manipulating the patients stem cells. These stem cells have the ability to become multiple different types of cells and may be coaxed into becoming beta cells in the lab which would allow for regenerative therapy if scientists are able to implement them back into the body successfully. The regenerated beta cell would have the ability to “hide from the immune system” by adding molecules that the body already recognizes. Labs are now trying to screen for genes that produce these protective properties, however a specific genes that were identified were obvious choices for a successful therapy.






