medicaltreatment:gene therapy

“Is curing patients a sustainable business model?” Goldman Sachs analysts ask | Ars Technica
▻https://arstechnica.com/tech-policy/2018/04/curing-disease-not-a-sustainable-business-model-goldman-sachs-analysts-

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One-shot cures for diseases are not great for business—more specifically, they’re bad for longterm profits—Goldman Sachs analysts noted in an April 10 report for biotech clients, first reported by CNBC.

The investment banks’ report, titled “The Genome Revolution,” asks clients the touchy question: “Is curing patients a sustainable business model?” The answer may be “no,” according to follow-up information provided.

Analyst Salveen Richter and colleagues laid it out:

The potential to deliver “one shot cures” is one of the most attractive aspects of gene therapy, genetically engineered cell therapy, and gene editing. However, such treatments offer a very different outlook with regard to recurring revenue versus chronic therapies... While this proposition carries tremendous value for patients and society, it could represent a challenge for genome medicine developers looking for sustained cash flow.

For a real-world example, they pointed to Gilead Sciences, which markets treatments for hepatitis C that have cure rates exceeding 90 percent. In 2015, the company’s hepatitis C treatment sales peaked at $12.5 billion. But as more people were cured and there were fewer infected individuals to spread the disease, sales began to languish. Goldman Sachs analysts estimate that the treatments will bring in less than $4 billion this year.

“[Gilead]’s rapid rise and fall of its hepatitis C franchise highlights one of the dynamics of an effective drug that permanently cures a disease, resulting in a gradual exhaustion of the prevalent pool of patients,” the analysts wrote. The report noted that diseases such as common cancers—where the “incident pool remains stable”—are less risky for business.

China Will Always Be Bad at Bioethics – Foreign Policy
▻http://foreignpolicy.com/2018/04/13/china-will-always-be-bad-at-bioethics

https://foreignpolicymag.files.wordpress.com/2018/04/gettyimages-53309209.jpg

It’s no accident that the Chinese government is leading the world in medical advances — and in dangerous ethical lapses.

(…) the justification of individual sacrifice for the greater good is contrary to any principle-based bioethics framework. When Jesse Gelsinger died at the University of Pennsylvania as the first casualty of gene therapy in 1999, the tragedy halted this type of experimental treatment on humans for several years in the United States, and it still serves as a somber reminder for the medical community. Had the death occurred in China, it would most likely have been either covered up or turned into propaganda depicting Gelsinger as a national martyr.

le titre est affreux et l’article sans doute biaisé, mais aussi plein de bonnes questions et d’infos

#santé #médecine #recherche #éthique #Chine #parti_unique

Goldman asks: ’Is curing patients a sustainable business model?’
►https://www.cnbc.com/2018/04/11/goldman-asks-is-curing-patients-a-sustainable-business-model.html

“Is curing patients a sustainable business model?” analysts ask in an April 10 report entitled “The Genome Revolution.”

“The potential to deliver ’one shot cures’ is one of the most attractive aspects of gene therapy, genetically-engineered cell therapy and gene editing. However, such treatments offer a very different outlook with regard to recurring revenue versus chronic therapies,” analyst Salveen Richter wrote in the note to clients Tuesday. “While this proposition carries tremendous value for patients and society, it could represent a challenge for genome medicine developers looking for sustained cash flow.”

(…) “GILD is a case in point, where the success of its hepatitis C franchise has gradually exhausted the available pool of treatable patients,” the analyst wrote. “In the case of infectious diseases such as hepatitis C, curing existing patients also decreases the number of carriers able to transmit the virus to new patients, thus the incident pool also declines … Where an incident pool remains stable (eg, in cancer) the potential for a cure poses less risk to the sustainability of a franchise.”

#économie #recherche #pharma #biens_publics #merci @archiloque

First Duchenne Patient Dosed in Microdystrophin Gene Therapy!
▻http://community.parentprojectmd.org/m/blogpost?id=1187424%3ABlogPost%3A245554

Our purpose in awarding this grant was to open and accelerate the field of gene therapy – a strategy that seemed impossible as a potential treatment for Duchenne back in the 1990s. If successful, it will provide systemic delivery of a microdystrophin construct that targets both skeletal and cardiac muscle achieved through one IV injection. The FDA has approved the protocol and now we have the first child dosed.

#dystrophie_atrophie_myopathie_Duchenne_traitement_médecine

The doctor responsible for gene therapy’s greatest setback is sounding a new alarm - MIT Technology Review
►https://www.technologyreview.com/s/610141/the-doctor-responsible-for-gene-therapys-greatest-setback-is-sound

https://cdn.technologyreview.com/i/images/wilson-james-lab-shot.jpg?cx=0&cy=0&cw=2858&ch=1607&sw=1200

To attack the disease, researchers replace patients’ damaged copies of a gene called dystrophin by introducing viral particles that carry a correct copy. Reaching the countless muscle cells in a boy’s body requires extremely high doses of these particles—400 trillion or more per pound of body weight.

That’s where the danger could come in, says Wilson. At those doses, he says, his team found that two different viruses caused extreme and sudden immunological effects, including damage to the liver and blood vessels.

[...] The general type of #virus implicated, called an AAV, is widely used and considered safe. More than 2,000 people have been treated with AAVs, which have been used in successful tests of gene-therapy treatments for hemophilia and blindness.

Thanks to those successes, gene therapy has been racing forward, building toward ever more dramatic cures. The latest tests move into unknown territory because they involve much higher doses (which are needed to reach muscles or brain cells) and because the subjects are children.

#thérapie_génique #myopathie

The doctor responsible for gene therapy’s greatest setback is sounding a new alarm - MIT Technology Review
►https://www.technologyreview.com/s/610141/the-doctor-responsible-for-gene-therapys-greatest-setback-is-sound

https://cdn.technologyreview.com/i/images/wilson-james-lab-shot.jpg?cx=0&cy=0&cw=2858&ch=1607&sw=1200

James Wilson of the University of Pennsylvania reported this week that monkeys and pigs given super-high doses of gene therapy died or suffered disturbing behavioral changes.

“What is remarkable is we have not seen it before,” says Wilson. “We were surprised but shouldn’t have been. If you push the dose of anything high enough, you are going to see toxicity.”

The warning comes amidst a scramble by three companies—Sarepta Therapeutics, Pfizer, and Solid Biosciences—to be the first to use the technique to cure muscular dystrophy. That disease strikes young boys, destroys their muscles, and kills them by their 20s.

To attack the disease, researchers replace patients’ damaged copies of a gene called dystrophin by introducing viral particles that carry a correct copy. Reaching the countless muscle cells in a boy’s body requires extremely high doses of these particles—400 trillion or more per pound of body weight.

R. Jude Samulski, who studies viral gene therapy at the University of North Carolina and is involved with Pfizer’s project, says that because Wilson’s viruses were slightly different from those being used in trials, it is not known whether his findings apply to the studies under way.

However, Samulski says the mixture of scientific ambition, technological advances, desperate patients, and potential financial windfalls could be combustible.

“It would be very naïve for our community to assume we won’t have toxic effects,” he says. “People get comfortable, saying, ‘Hey, let’s do it—there is nothing lose.’ Well, it’s out there waiting. But when it’s going to happen, why it’s going to happen—nobody knows.“

#Médecine #Gene_therapy #Thérapie_génique #Néolibéralisme

Boom in human gene editing as 20 CRISPR trials gear up | New Scientist
▻https://www.newscientist.com/article/2133095-boom-in-human-gene-editing-as-20-crispr-trials-gear-up

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The CRISPR genome editing revolution continues to advance at an astounding pace. As many as 20 human trials will be under way soon, mostly in China, New Scientist has learned.

One of these trials will involve the first-ever attempt to edit cells while they are inside the body. The aim is to prevent cervical cancers by using CRISPR to target and destroy the genes of the human papillomavirus (HPV) that cause tumour growth. This study is due to begin in July at the First Affiliated Hospital of Sun Yat-Sen University in China.

Gene therapy, which involves adding extra genes to cells, was first used to cure people in 1990, but it is mainly useful for treating rare genetic disorders. In contrast, gene-editing, which involves altering existing genes inside cells, promises to treat or cure a much wider range of conditions, from HIV infection to high blood cholesterol.

The HPV trial, meanwhile, will break new ground. Instead of editing cells outside the body, a gel containing DNA coding for the CRISPR machinery will be applied to the cervix. The CRISPR machinery should leave the DNA of normal cells untouched, but in cells infected by HPV, it should destroy the viral genes, preventing them from turning cancerous.

“Targeting HPVs seems a sensible approach if they can deliver the genome-editing components to sufficient numbers of cells,” says Robin Lovell-Badge of the Crick Institute in the UK.

#génomique #médecine
“It is tricky to do these experiments in animals as they are not infectable by HPV,” says Bryan Cullen of Duke University Medical Center in North Carolina, whose group also hopes to use gene editing to get rid of HPV. But there is a risk of off-target mutations leading to cancer, he warns.

One Man’s Quest to Hack His Own Genes
▻https://www.technologyreview.com/s/603217/one-mans-quest-to-hack-his-own-genes
▻https://d267cvn3rvuq91.cloudfront.net/i/images/mit-comic2.jpg?cx=0&cy=0&cw=2000&ch=1125&sw=1200

When Brian Hanley set out to test a gene therapy, he started with himself.

#médecine #ADN #recherche #transhumanisme