The big news broke Friday that the FDA approved two new gene therapies for sickle cell disease. While the approvals were expected and the actual use of these treatments will be complex on several levels, this is a historic development.
2 FDA-approved gene therapies for sickle cell
Friday’s news follows on recent UK approval for one of the same therapies, a gene editing drug called Casgevy from Vertex and CRISPR Therapeutics.
Lyfgenia, from bluebird bio, is the other gene therapy now approved in the U.S. It seems that Lyfgenia has gotten less attention than Casgevy. What is Lyfgenia? Here’s how the FDA describes this combo cell and gene therapy that uses a lentiviral approach:
“With Lyfgenia, the patient’s blood stem cells are genetically modified to produce HbAT87Q, a gene-therapy derived hemoglobin that functions similarly to hemoglobin A, which is the normal adult hemoglobin produced in persons not affected by sickle cell disease.”
The key with each approach is getting enough functional hemoglobin protein in the mix.
There’s a lot to learn about these gene therapies for sickle cell disease. For example, with wider use, some additional side effects may become clear. The FDA statement on the approvals already lists some side effects. Note that Lygenia got the most serious type of FDA side effect warning called a black box warning because some blood cancers arose in the patients getting it during trials.
More data are also needed on some patient populations like young kids if it will ultimately be used to treat them.
Why this is still very complicated
These are also intense therapies requiring the use of chemo to make a niche for the transplanted modified cells. This means extensive hospitalization and risk of infection. Chemo can also cause lifelong issues including infertility.
Gina Kolata over at NYT also noted obstacles to patient access both in the US and worldwide:
“The obstacles to treatment are myriad: an extremely limited number of medical centers authorized to provide it; the requirement that each patient’s cells be edited or have a gene added individually; procedures that are so onerous that not everyone can tolerate them; and a multimillion-dollar price tag and potential insurance obstacles.”
These therapies will likely cost $2-$3 million per use.
How will most people in the world with sickle cell be able to access these therapies? The short answer is that they won’t anytime soon. There needs to be a way for great access to both these and other emerging gene and cell therapies.
Overall, despite this very complicated context, the approval of these gene therapies for sickle cell is great news.
Paolo Macchiarini under the lens
Okay, on to some less upbeat news.
Paolo Macchiarini is the subject of intense attention these days after patient deaths, research misconduct investigations, and court convictions. In better times he was called the ‘stem cell surgeon’ in glitzy media pieces celebrating his clinical research.
Now, he’s the “bad surgeon”.
Before we dive into what’s new with him, check out my new video (above) on our YouTube channel fact-checking a NY Post article on stem cells and anti-aging that’s got issues. Please subscribe as we head toward 1,000 subscribers.
Okay, on to Macchiarini.
A new documentary on Netflix covers his exploits. The docuseries has sparked a Rolling Stone piece and many others in the past week that dig into Macchiarini. Here’s the piece. The Dashing Italian Surgeon Who Seduced a Reporter to Mask His Bloody Crimes, Rolling Stone.
Okay, so “bloody crimes” is intense but Macchiarini’s action did lead to people’s unnecessary deaths and suffering. The media has had a real up-and-down relationship with Macchiarini. When he was flying high, some reporters gushed about him. The story of how Macchiarini conning his one-time romantic interest, journalist Benita Alexander, also came out including in a very tabloid Vanity Fair piece.
I haven’t yet watched this new documentary about Macchiarini. It’s got mixed reviews so far. Are you going to watch it?
This kind of series can be painful to sit through. There’s so much great stem cell stuff going on that it’s not fun to see so much bad stem cell news swirling around this one guy. Could it be a wake-up call to others who are selling unproven stem cells?
Of course, not everyone (or anyone?) doing bad stem cell stuff who watches the Macchiarini documentary will say to themselves, “Gee, I better stop doing stuff like that too.” Still, media pieces like Bad Batch about Liveyon and John Kosolcharoen serve a purpose of educating and warning people.
Other recommended reads
- Fujifilm to Invest USD 200 Million to Expand Cell Therapy Development and Manufacturing Capabilities, Fujifilm.
- Getting Accelerated Approval Wrong, In The Pipeline. Is Derek Lowe right that the FDA is becoming too lenient on drug approvals? We haven’t seen that in the cell therapy space but in our arena it does seem like the agency is allowing some unusual and surprising INDs.
- The lucky break behind the first CRISPR treatment, MIT Tech Review.This provides some backdrop for Casgevy approval.
- I couldn’t find a dumb stem cell or other regenerative headline of the week so maybe that’s something positive.
I watched the whole series a couple of evenings ago. Sociopaths are all around us, hard to detect and impossible to cure. As scientists, we are particularly outraged by scientist sociopaths like Macchiarini because they undermine our view of ourselves as being honest and ethical. There was not much said about stem cells, so your readers should know that there were never any stem cells on his plastic transplants.
The only way to deal with a sociopath in one’s life is to run away, but it’s hard to do that when it means you have to leave something precious behind. I guess I’d say that the series is disturbing in part because it doesn’t offer any advice about how to avoid this happening again.