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For older patients particularly, the prospect of infrequent treatment offers significant advantages over managing complex daily drug regimens.
By Aaron Sull, Jewish Breaking News
A revolutionary gene therapy trial at Rambam Hospital is pioneering the use of genetic editing to treat common diseases that aren’t caused by genetic defects.
The team led by Dr. Oren Caspi is testing a breakthrough treatment for Transthyretin cardiac amyloidosis (ATTR-CA), a condition that affects an estimated 5 percent of heart failure patients.
The disease occurs when proteins don’t fold properly—either due to genetic defects or, more commonly, aging—and accumulate in the heart, causing failure, irregular rhythms, and potentially death.
Unlike traditional gene therapies targeting rare inherited disorders, this CRISPR-Cas9 treatment tackles an age-related problem by permanently editing liver cells to halt production of the disease-causing protein.
The approach requires a one-time intravenous injection, preceded by temporary immunosuppressive therapy to prevent the body from rejecting the treatment’s delivery system.
Once CRISPR reaches liver cells, it permanently removes the defective gene.
For older patients particularly, the prospect of infrequent treatment offers significant advantages over managing complex daily drug regimens.
“When I studied medicine, this disease was considered serious and even terminal,” explains Dr. Caspi, Director of Rambam’s Heart Failure Unit.
“But the existing drug doesn’t stop the deterioration, it only slows it down.”
Beyond Rambam’s heart failure research, the gene-editing approach is expanding rapidly into other areas such as cholesterol management.
Separate trials by companies including CRISPR Therapeutics and Verve—recently acquired by Eli Lilly for up to $1.3 billion—are testing gene-edited treatments for high cholesterol that replicate naturally occurring mutations found in people with low cholesterol levels.
Initial results from these trials showed substantial reductions in “bad” cholesterol and triglycerides.
Although treatments face scrutiny as they advance through regulatory processes, early results suggest a fundamental shift from managing chronic diseases to potentially eliminating their underlying causes with just a single intervention
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