Gene therapy has hit a potential speed bump with another cancer scare, according to a news report.
Two letters published in the journal Nature, one by Novartis and one by researchers at Sweden’s Karolinska Institute, suggest that the gene-editing technology known as CRISPR-Cas9 may be able to cause tumors in patients, STAT reported Monday.
The researchers found that applying CRISPR-Cas9 triggers the gene p53 to either cause the cell to repair itself or die. However, in cases when the cells survive, it’s because of p53 dysfunction, a phenomenon that can cause cancers, especially ovarian, colorectal, lung, pancreatic, liver, breast and others. However, the article also reported that some researchers were not alarmed by the findings.
This isn’t the first time that gene therapy researchers have been spooked. Last year, a paper indicated that the technique causes a large number of off-target mutations, but it was retracted in March. Other forms of gene therapy, in particular ex vivo techniques that use viral vectors, have also raised safety concerns about genotoxicity, but experience in clinical trials has not borne out those fears.
Meanwhile, industry interest in gene therapy, including gene editing, has continued unabated. Last month, Cambridge, Massachusetts-based Beam Therapeutics came out of stealth mode with $87 million in Series A funding on hand and a “base editing” technique that allows single-letter gene editing, compared with the less precise “cutting” method used by CRISPR. Also last month, the Food and Drug Administration placed a clinical hold on an application from CRISPR Therapeutics and Vertex Pharmaceuticals to start a clinical trial of their CRISPR gene-editing therapy, CTX001, for sickle cell disease. A second trial, in beta-thalassemia, remains on track to start later this year.
Another potential threat to hurdle to CRISPR-Cas9 is on the legal front, with two academic institutions – the Massachusetts Institute of Technology’s Broad Institute and the University of California Berkeley – vying in court over claims to the technology.
A Deep-dive Into Specialty Pharma
A specialty drug is a class of prescription medications used to treat complex, chronic or rare medical conditions. Although this classification was originally intended to define the treatment of rare, also termed “orphan” diseases, affecting fewer than 200,000 people in the US, more recently, specialty drugs have emerged as the cornerstone of treatment for chronic and complex diseases such as cancer, autoimmune conditions, diabetes, hepatitis C, and HIV/AIDS.
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