{"id":378167,"date":"2023-12-10T05:39:19","date_gmt":"2023-12-10T10:39:19","guid":{"rendered":"https:\/\/platohealth.ai\/weekly-reads-fda-oks-2-gene-therapies-for-sickle-cell-new-paolo-macchiarini-series-fujifilm-the-niche\/"},"modified":"2023-12-11T00:30:46","modified_gmt":"2023-12-11T05:30:46","slug":"weekly-reads-fda-oks-2-gene-therapies-for-sickle-cell-new-paolo-macchiarini-series-fujifilm-the-niche","status":"publish","type":"post","link":"https:\/\/platohealth.ai\/weekly-reads-fda-oks-2-gene-therapies-for-sickle-cell-new-paolo-macchiarini-series-fujifilm-the-niche\/","title":{"rendered":"Weekly reads: FDA OKs 2 gene therapies for sickle cell, new Paolo Macchiarini series, Fujifilm – The Niche","gt_translate_keys":[{"key":"rendered","format":"text"}]},"content":{"rendered":"

The big news broke Friday that the FDA approved two new gene therapies for sickle cell disease<\/strong><\/a>. While the approvals were expected and the actual use of these treatments will be complex on several levels, this is a historic development.<\/p>\n

\"Marie
Since Marie Tornyenu received Casgevy in a clinical trial her sickle cell symptoms have gone away<\/strong>. Photo from\u00a0Children\u2019s Hospital Of Philadelphia.<\/figcaption><\/figure>\n

2 FDA-approved gene therapies for sickle cell<\/h2>\n

Friday\u2019s news follows on recent UK approval for one of the same therapies, a gene editing drug called\u00a0Casgevy<\/strong><\/a>\u00a0from Vertex and CRISPR Therapeutics.<\/p>\n

Lyfgenia, from bluebird bio<\/strong><\/a>, 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\u2019s how the FDA describes this combo cell and gene therapy that uses a lentiviral approach:<\/p>\n

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\u201cWith Lyfgenia, the patient\u2019s blood stem cells are genetically modified to produce HbAT87Q<\/sup>, 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.\u201d<\/p>\n<\/blockquote>\n

The key with each approach is getting enough functional hemoglobin protein in the mix.<\/p>\n

There\u2019s 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\u00a0side effects.<\/strong><\/a> 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.<\/p>\n

More data are also needed on some patient populations like young kids if it will ultimately be used to treat them.<\/p>\n

Why this is still very complicated<\/h2>\n

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.<\/p>\n

Gina Kolata<\/strong><\/a> over at NYT also noted obstacles to patient access both in the US and worldwide:<\/p>\n

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\u201cThe obstacles to treatment are myriad: an extremely limited number of medical centers authorized to provide it; the requirement that each patient\u2019s 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.\u201d<\/p>\n<\/blockquote>\n

These therapies will likely cost $2-$3 million per use.<\/p>\n

How will most people in the world with sickle cell be able to access these therapies? The short answer is that they won\u2019t anytime soon. There needs to be a way for great access to both these and other emerging gene and cell therapies.<\/p>\n

Overall, despite this very complicated context, the approval of these gene therapies for sickle cell is great news.<\/p>\n

Paolo Macchiarini under the lens<\/h2>\n

Okay, on to some less upbeat news.<\/p>\n

Paolo Macchiarini<\/strong><\/a> is the subject of intense attention these days after patient deaths, research misconduct investigations, and court convictions. In better times\u00a0he was called the \u2018stem cell surgeon\u2019 in glitzy media pieces celebrating his clinical research.<\/p>\n

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Now, he\u2019s the \u201cbad surgeon\u201d.<\/p>\n

Before we dive into what\u2019s new with him, check out my new video (above) on our YouTube channel fact-checking a NY Post<\/em> article on stem cells and anti-aging that\u2019s got issues. Please subscribe as we head toward 1,000 subscribers.<\/p>\n

Okay, on to Macchiarini.<\/p>\n

\"benita
Paolo Macchiarini floating along with his romantic interest Benita Alexander in better days.<\/figcaption><\/figure>\n

A new documentary on Netflix covers his exploits. The docuseries has sparked a\u00a0Rolling Stone<\/em> piece and many others in the past week that dig into Macchiarini. Here\u2019s the piece. The Dashing Italian Surgeon Who Seduced a Reporter to Mask His Bloody Crimes<\/a>, Rolling Stone.\u00a0<\/em><\/strong><\/p>\n

Okay, so \u201cbloody crimes\u201d is intense but Macchiarini\u2019s action did lead to people\u2019s 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.\u00a0The story of how Macchiarini conning his one-time romantic interest, journalist Benita Alexander<\/strong><\/a>, also came out including in a very tabloid Vanity Fair<\/em> piece.<\/p>\n

I haven\u2019t yet watched this new documentary about Macchiarini. \u00a0It\u2019s got mixed reviews so far. Are you going to watch it?<\/p>\n

This kind of series can be painful to sit through. There\u2019s so much great stem cell stuff going on that it\u2019s 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?<\/p>\n

Of course, not everyone (or anyone?) doing bad stem cell stuff who watches the Macchiarini documentary will say to themselves, \u201cGee, I better stop doing stuff like that too.\u201d Still, media pieces like Bad Batch<\/strong><\/a> about\u00a0Liveyon<\/strong> <\/a>and John Kosolcharoen serve a purpose of educating and warning people.<\/p>\n

Other recommended reads<\/h2>\n
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Related Posts<\/em><\/h3>\n<\/div>\n