I’ve had a number of people ask me about the news stories the last few days which keep saying that there’s a a “cure for HIV” coming. This all goes back to a Maryland company, American Gene Technologies, and a gene therapy they’re working on called AGT-103T. This is an attempt to use a lentiviral-based gene therapy to produce patient-derived CD4 helper T cells that will allow the immune system to recognize and eliminate HIV, while themselves remaining (in theory) immune to infection (through modification of CCR5 and CXCR4). That retroviral attack on this T-cell population is, of course, a hallmark of HIV itself and a fundamental reason the disease is so serious.
None of this is unreasonable; this seems to be a perfectly valid approach to HIV treatment, and I wish AGT good luck in the clinic. But throwing around the word “cure”, before you have treated a single human patient, is flat-out irresponsible. That’s right; this is all publicity for the filing of an IND (the approval needed to begin human trials). And AGT seems to enjoy this approach – here’s the boilerplate language at the bottom of their press release:
American Gene Technologies (AGT) is a gene and cell therapy company with a proprietary gene-delivery platform for rapid development of cell and gene therapies to cure infectious diseases, cancers, and inherited disorders. The Company’s mission is to transform people’s lives through genetic medicines that rid the body of disease. The Company expects to take its patented lead candidate for an HIV cure into the clinic in 2019.
There are a lot of things that can go wrong with gene therapy, and there are a lot of things that we don’t yet understand about its applications. AGT knows this; anyone who works in the field knows this. And that’s why I say that using “cure” for what are actually just interesting ideas in preclinical development is just not right. There are a lot of very sick people out there; AGT and the rest of the biopharma industry are trying to find new medicines to help them. But no one is well served by setting off “Single-dose cure!” headlines before a treatment has gone into a single human being. To AGT’s management and press office, this message: raising hopes like that is not some sort of PR coup – it’s cruelty. You have no idea if you have a cure yet, and you know it. That gets proved in the clinic, and you haven’t even received approval to dose anyone in Phase I, much less anyone who actually has HIV.
It’s a bad look for you, and it’s a bad look for the industry. Sure, we should tell people what we’re trying to accomplish. But that needs to be tempered with the reality of our clinical failure rates. We get humbled all the time in this business; the record is there for anyone to see. So perhaps a little preliminary humility is in order? You can always set off the fireworks after you get some actual results.