Gene therapy in the 1990s?

Yep. Surprised us as well. According to a 2014 article in Human Gene Therapy, the public perception of gene therapy has improved since the 1990s. Didn’t know there was a baseline in 1990 to compare to, but okay. According to the 2014 data, “a majority of respondents (>90%) accept gene therapy as a treatment for severe illnesses such as Alzheimer disease, but this receptivity decreases for conditions perceived as less severe.” As Dr. Seuss might say, “Oh, the places you’ll go.” Because, 30 years removed from the ‘90s, there are actually approved gene therapy treatments (really good source written for pharmacists), and they ain’t cheap. As of January 2019, there are 3 FDA-approved gene therapy products on the market (Kymriah, Yescarta, Luxturna). Anyway, yes Pat, I’d like to buy a vowel.

Who is “Gene Therapy”?

While the name “Gene” might be trending in the wrong direction (it’s the 2,112nd most popular male name in 2018, down 243 spots from 2017), according to Google, the term “gene therapy” is not. However, “according to a review of recent research studies that measured US public opinion related to gene medicine, the public is not yet aware of gene medicine; is unfamiliar with the terms being used to describe the topic; and, when faced with multiple therapeutic options, is worried about making informed decisions.” Did you know there’s an association focused on gene therapies? (There is always an association.) Introducing the Alliance for Regenerative Medicines or ARM. If you’re at all interested in the topic they seem to have decent publications. If you’re not interested, then here is a very funny video on the topic.

Good question, bad optics

A report released by Goldman Sachs asks a question that the biotech industry has been starting to grapple with: “Is curing patients a sustainable business model?” It’s a fair question, and an easy answer: no. The GS analysts use another GS, Gilead Sciences, as a case study to explain this. A few years ago Gilead came out with their Hepatitis C cure, and US sales of that peaked in 2015 at about $12.5B. This year it’s projected to make less than $4B in the US. So the obvious conclusion is that curing patients with “one-shot” gene therapies makes less money over time than chronic therapies, makes sense. But jeeeeeeez when you take off your impartial industry analyst hat and think about how this kind of report looks to non-industry people… it’s just bad optics.

When blood clotting is a good thing

London researchers have made a rare breakthrough in a rare disease: hemophilia A. In the small-scale clinical study, patients received injections of a missing gene that promotes the production of the factor VIII clotting protein absent in these patients. Results reported in NEJM showed that six of the 7 patients in the high-dose cohort maintained normal levels of factor VIII after a full year. Also amazing, those in the high-dose cohort had an average of 16 bleeding events per year prior to therapy and this was reduced to 1 event per year after therapy. These findings are particularly significant because prior gene therapy studies have shown effectiveness only in hemophilia B sufferers, not hemophilia A, where a different clotting protein is the culprit.

I can’t find genes that fit

Good news, bad news. Companies that are developing cutting-edge gene therapy recently got some good news when the FDA approved the first gene therapy in the US and indicated they will expedite the approval of experimental gene therapies. Great. The bad news? Genes (specifically, modified genes) are in high demand. A recent NY Times article explains that “firms that can (manufacture the genes) are swamped with orders and requests.” Organizations are having to sign-up years in advance to secure manufacturing slots, often before proof-of-concept is complete. Risky. Expensive. Decisions have to be made and one company, Bluebird Bio, has taken the bull by the horns, securing their own manufacturing. We’ll keep you updated, and for an InsightCity approved view of men and skinny jeans go here.
[Editor’s note: two-thirds of InsightCity approved anyway.]

It’s aliiive! And it’s approved!

Last week, the FDA approved Novartis’ Kymriah—a “living drug” that works by making immune cells realize they should get rid of those pesky leukemia cells making a mess of things. We’ve been following this story since June, because it’s really cool for a couple reasons. First, it marks the first time a gene therapy has been approved for use in the US, although more CAR-T treatments are in the pipeline. Second, the treatment is designated for the most prevalent form of childhood cancer in the US—acute lymphoblastic leukemia (ALL). There is a bit of worry that the treatment is prohibitively expensive, which is what sunk the first gene therapy approved in the EU, but maybe competition will help drop prices.

1. Gene therapy for the masses

Here’s a riddle for you. What has four wheels and costs $150K? Hint: it weighs 150 pounds. Give up? It’s the new portable device that automates CAR-T production—we had to Google it, too. The current gene therapy process isn’t what we’d call convenient. It involves: flying the patient or patient’s blood to one of only a dozen research centers globally, a room full of smart people, and dozens of hours just to process one person’s cells. As you can imagine that rings up a hefty price tag—some estimates are around $500K. With the new portable device, Prodigy, the goal is to automate this tedious procedure and take gene therapy to the patient instead of the other way around.

1. Gene therapy for Alzheimer’s disease

While still a long way from the finish line, gene therapy looks promising for blocking the onset of Alzheimer’s disease. Researchers at Imperial College London injected lentivirus, which contains the PGC-1-alpha gene, into the hippocampus and the cortex of mice susceptible to the development of Alzheimer’s disease. Compared to a control group of similarly susceptible mice, the treated group showed little development of amyloid plaques. Not to get too technical here but amyloid plaque is stuff believed to lead to bad things. Like strokes in golf, you want fewer of them. Treated mice also showed no loss of brain cells in the hippocampus and normal performance on memory tasks. All together now… Sci-ence!, Sci-ence!, Sci-ence! Well done, smart people.