Application of a fluorescent green tracer called AMDX-9101 during a routine eye exam can detect toxic transthyretin, which causes symptoms of transthyretin amyloidosis (ATTR), a group of conditions that includes familial amyloid polyneuropathy (FAP). Protein deposits may be detected.
Although it’s too early to know whether this approach will work in clinical practice, the data suggest that this tracer could help doctors treat FAP and other forms of ATTR in the early stages, when treatment is most likely to be effective. It has been suggested that it may be possible to diagnose.
“Developing a simple topical (topically applied) vision test to detect ATTR could transform early diagnosis of ATTR and allow more people to benefit from available treatments.” ” Stella Saraf, Ph.D., CEO and founder of AMDX-9101 developer Amidis, said in a paper. Corporate press release.
The results of the AMDX-9101 study will be presented by Tatiana Millman, M.D., an ophthalmologist who has been involved in the development of the tracer, at the annual meeting of the American Society of Ocular Oncologists and Pathologists in Chicago on October 18. . Millman is a professor of ophthalmology and pathology at Wills Eye Hospital.
ATTR consists of a series of conditions in which transthyretin misfolds and forms toxic deposits called amyloid fibrils that accumulate in tissues and cause damage. The disease most often affects the nerves outside the brain and spinal cord (an inherited form called FAP) and the heart (called ATTR cardiomyopathy).
However, other parts of the body can also be affected, including the eyes. Amyloid fibrils that accumulate in the eye cause a variety of symptoms, from dry eye and blurred vision to vision-threatening complications such as changes in the blood vessels of the eye and glaucoma, and nerve damage due to increased intraocular pressure. Possibly.
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This approach may result in early diagnosis
There are treatments that can slow the progression of FAP and other forms of ATTR, but how effective they are depends on how early the disease is diagnosed. Because ATTR is often underdiagnosed, many patients do not receive treatment until symptoms have progressed.
“I study the ocular symptoms of (systemic) amyloidosis in patients and clearly understand the urgent need for better non-invasive diagnostics for (systemic) amyloidosis disorders.” said Jose Pulido, MD, a member of the association. An ophthalmologist who is a member of Amidis’ scientific advisory board and was involved in the development of AMDX-9101.
Based on the idea that “the eyes are the windows to the body,” Amidis developed AMDX-9101 as a small molecule tracer that can screen for transthyretin amyloid fibers in the conjunctiva, the transparent membrane that protects the eye.
Once the tracer is deposited, the deposit glows green under standard ocular imaging equipment. If the use of tracers can be added to already established workflows in eye care settings, it could be a non-invasive and effective way to detect disease in its early stages, before symptoms are yet evident. .
“I believe AmidiTracer will improve patient outcomes by helping to identify amyloidosis at an earlier stage and personalize treatment plans,” said Will’s Eye, Thomas Jefferson University; said Pulido, who also held a role at the Henry and Corinne Bower Memorial Institute. For translational medicine.
A study by Millman, Pulido and colleagues showed that when AMDX-9101 and transthyretin blocks were mixed in a laboratory dish, a bright green fluorescent signal was generated. This was also observed when researchers added AMDX-9101 to the eyes of deceased FAP patients and to eye samples of people with other forms of inherited ATRR. Tests in pig eyes showed that the tracer could be detected using standard eye imaging equipment.
This finding supported the application of AMDX-9101 to the eye during a simple eye exam as a potential method to diagnose FAP and other forms of ATTR.