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Advanced Neurology Role of immunosuppressants in autism
benefits and risks of these drugs in individuals with ASD in Table 1 found that Azathioprine reduced NK cell
and to develop safer and more effective treatments for this cytotoxicity against renal cells in vitro. The authors of the
disorder. In addition to the differences mentioned above, study suggest that this effect may be beneficial in preventing
rapamycin and AZA also have different modes of action rejection of renal transplants. Azathioprine has also
and targets in the body. Rapamycin is an mTOR inhibitor been found to be useful in the treatment of autoimmune
that works by binding to and inhibiting the activity of the diseases in general. Table 1 describes a study in which
mTOR complex, a key regulator of cellular metabolism Azathioprine was used to treat a variety of autoimmune
and growth [144] . In addition to its potential use in treating diseases, including rheumatoid arthritis, systemic lupus
ASD, rapamycin is also used as an immunosuppressant erythematosus, and Crohn’s disease. The study found that
in organ transplant patients and is being studied for its Azathioprine was effective in reducing disease activity and
potential use in treating other neurological disorders, improving quality of life in patients with these diseases. In
such as epilepsy and Alzheimer’s disease. Azathioprine, addition to autoimmune diseases, Azathioprine has also
on the other hand, works by suppressing the immune been used to treat Myasthenia disease, a neuromuscular
system and is used primarily as an immunosuppressant in disorder characterized by weakness and fatigue of
the treatment of autoimmune diseases, such as IBD and voluntary muscles. A study referenced in Table 1 found
rheumatoid arthritis [145] . The drug is thought to work by that Azathioprine was effective in improving muscle
interfering with the DNA synthesis of rapidly dividing strength and reducing disease symptoms in patients
cells, including immune cells, thereby reducing their with Myasthenia disease. Finally, Azathioprine has been
ability to attack and damage healthy tissues. While both used in the treatment of Lupus erythematosus, a chronic
drugs have been studied for their potential use in treating autoimmune disease that affects multiple organs. A study
ASD, it is important to note that there is currently no FDA- referenced in Table 1 found that Azathioprine was effective
approved medication for the treatment of this disorder. In in reducing disease activity and improving quality of
addition, both rapamycin and AZA are associated with life in patients with Lupus erythematosus. In summary,
significant side effects and potential risks, and their use in Azathioprine has been found to be useful in the treatment
the treatment of ASD should be carefully considered on of various diseases by inhibiting NK cells. These diseases
a case-by-case basis. Further research is needed to fully include Behcet’s disease, cellular cytotoxicity in renal
understand the potential benefits and risks of these drugs transplant, autoimmune diseases, Myasthenia disease, and
in individuals with ASD and to develop safer and more Lupus erythematosus.
effective treatments for this disorder.
9. Current challenges facing drugs used in
8. Azathioprine in various treatment by treating autism
inhibiting NK cell
Many research projects are currently underway in the
Azathioprine is an immunosuppressive drug that has been field of neurodevelopmental disorders, specifically
found to be effective in treating a variety of diseases. One autism. However, treating autism is challenging because
such disease is Behcet’s disease, an autoimmune disease there are only two FDA-approved drugs available, which
that affects multiple organ systems. A study referenced are risperidone and aripiprazole. Other drugs with
in Table 1 has shown that Azathioprine is an effective minimal effects are still being tested in clinical trials.
treatment for Behcet’s disease. The study found that Furthermore, researchers are on the quest to find a novel
Azathioprine reduced the severity of oral and genital drug that truly cure the disorder rather than just providing
ulcers, skin lesions, and arthritis in patients with Behcet’s symptomatic relief since these two FDA-approved drugs
disease. Another use of Azathioprine is to reduce cellular have been used for symptomatic relief in autism. Drug
cytotoxicity in renal transplant. A study referenced discovery has been a challenge for researchers due to
the limited pathophysiology and understanding of the
Table 1. Azathioprine in various treatment by inhibiting
natural killer cell disease. Challenges have also been resolved with further
understanding of advances such as fragile X syndrome and
Drug Treatment References TSC. The pharmaceutical and academic communities have
Azathioprine Behcet’s disease [149] emerged as major contributors in the search for treatments
Azathioprine Cellular cytotoxicity in renal transplant [150] for ASD. Unfortunately, the existing pharmacotherapy
Azathioprine Autoimmune disease [151] of ASD still mainly relies on the use of a broad range of
different non-approved agents, including antidepressants,
Azathioprine Myasthenia disease [152] anxiolytics, and antipsychotics, due to a lack of preclinical
Azathioprine Lupus erythematosus [153] and clinical drug design tools and a lack of understanding
Volume 2 Issue 2 (2023) 16 https://doi.org/10.36922/an.391

