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The engineered cells multiply in the body and pursue their course. (Some T cell therapies have so-called “kill switches” meant to deactivate them if they go awry. This particular therapy does not.) Cho said that patients need to understand clearly that once they receive their cells, they will be followed for results.
There was also confusion about the role of two major corporations in the study. Language in the study documents made it seem that Novartis (NYSE: NVS) and Life Technologies (NYSE: LIFE) had financial ties, but June said it wasn’t so. Novartis, for example, only has rights to Penn’s work in a type of cancer therapy called chimeric antigen receptor T cells, or CAR-T. “There’s no CAR here,” said June, referring to the T cells modified with CRISPR-Cas9 that he and his colleagues have been developing.
The committee recommended other consent form changes. Patients need to know that a Phase 1 safety trial is not likely to lead to medical benefits, and they need to know that this is the first time CRISPR-Cas9 would be used in a human therapy. There were also concerns that the trial doesn’t pay enough of the costs, which could limit the trial to people who can afford it. “Will it exclude the poor who can’t pay for testing?” asked Laurie Zoloth, who runs the Center for Bioethics, Science and Society at the Northwestern School of Medicine.
If the Parker-funded program reaches the clinic soon, it would be a remarkable milestone. CRISPR-Cas9 was only described as a potential human therapy a few years ago. Other gene editing technologies have made more headway. Sangamo Biosciences (NASDAQ: SGMO) of Richmond, CA, owns the system known as zinc fingers and has moved a prospective therapy into clinical trials to treat HIV infection, a program June has been involved with. Sangamo also has FDA clearance to start trials in patients with hemophilia B and two types of the rare disease mucopolysaccharidosis.
Yesterday, the French biotech firm Cellectis (NASDAQ: CLLS) announced that its development partner Servier has begun dosing patients with a T cell immunotherapy modified with the gene editing system known as TALENs.