Amyloidosis is a rare disease which can cause organ failure. It is the result of an abnormal buildup of amyloid proteins in the blood. These proteins are then deposited within the body. They stiffen tissues, organs, muscles, and nerves. Patients, in extreme cases, need chemotherapy and organ transplantation.
ATTR amyloidosis is just one form of the condition which leads to elevated levels of the TTR protein.
Unfortunately, there has yet to be a cure for amyloidosis. Although treatments do allow patients to live a better life. Nonetheless, researchers are continuously looking for new options.
The latest research study, led by Dr. Julian Gillmore and published in the New England Journal of Medicine, demonstrated that CRISPR gene-editing technology may be able to provide ATTR amyloidosis patients substantial relief.
Dr. Gillmore concentrated on studying ATTR amyloidosis. This form of the condition is often associated with heart failure, as the nerves of the heart are typically affected.
The condition places a burden on patients, caregivers, and children to who the condition could be passed in the future.
This study aimed to evaluate whether CRISPR could improve outcomes. It involved 6 patients who were diagnosed with ATTR amyloidosis. 3 of the individuals were given one infusion of NTLA-2001, a low dose of CRISPR. The last 3 patients were given a higher dose of the same treatment.
The hope was that this therapy would lower the levels of TTR in the bloodstream.
This is exactly what they found. At the one-month mark, TTR was lowered significantly. Those with the higher dose of the treatment decreased TTR levels by 87% on average. The low dose group experienced a decrease of 52% on average.
These results are particularly meaningful because they demonstrate that one single infusion can improve patient outcomes dramatically.
Although there is much more research to be done on this therapy in amyloidosis, the results so far are very promising.
You can read more about this study and this investigative therapy for amyloidosis here.