Genome complexity: correcting hemophilia B (Introduction)

by David Turell , Friday, September 27, 2024, 19:18 (34 days ago) @ David Turell

A virus-carrying gene:

https://www.sciencedaily.com/releases/2024/09/240925172115.htm

"Adults with hemophilia B saw their number of bleeding episodes drop by an average of 71 percent after a single infusion of gene therapy, according to the results of an international Phase III clinical trial published today in the New England Journal of Medicine...

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"Hemophilia is a genetic disorder that limits the blood's ability to clot and affects around 30,000 people in the United States, mostly males. Left untreated, it can cause spontaneous bleeding, particularly internal bleeding into the joints, which, over time, can cause painful joint damage and mobility issues. Hemophilia B is caused by a lack of clotting factor IX. The gene therapy enables the liver to create factor IX, which allows the blood to clot and protects patients from frequent bleeds.

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"After at least one year of follow-up, participants in the study had an average 71 percent reduction in bleed rate after receiving the gene therapy, compared to the year prior, when they were treated with prophylactic infusions of factor IX, the standard treatment for the disease. More than half of the 45 patients in the study did not have any bleeds after receiving gene therapy.

"Based on the results of this study, the FDA approved the gene therapy (fidanacogene elaparvovec) in April 2024. Cuker was the site lead for the clinical trial at Penn Medicine, which was one of the top-enrolling sites for the study. It represents the second form of gene therapy approved to treat hemophilia B. The first such therapy (etranacogene dezaparvovec-drlb) was approved in November 2022, and Penn Medicine is one of several medical centers in the United States where this treatment is available to patients.

"Gene therapies have very specific guidelines that determine eligibility and require specialized knowledge to carry out patient screening and selection, education about treatment risks and benefits, and post-therapy monitoring. Penn Medicine offers access to numerous clinical trials for gene therapy and expertise in administering FDA-approved gene therapies.

"In the current study, the most common adverse effect was related to an immune system attack on liver cells that were targeted by the gene therapy, which can render the gene therapy ineffective, if not quickly treated. In the study, affected patients were treated with steroids to limit this immune reaction. Patients in the study will continue to be followed for at least five years to monitor potential long-term side effects.

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"'We hear from people born with hemophilia that -- even if their disease is well-managed -- there's this burden that's always in the back of their mind. The frequent infusions, the cost of treatment, the need to plan for infusions when traveling, what happens if they do experience a bleed, and so on, is always there," Cuker said. "Now that we have patients who were treated on this study and are essentially cured of their hemophilia, they're telling us about realizing a new, 'hemophilia-free state of mind.' As a physician, it's amazing to see my patients so happy with their new reality.'"

Comment: Here is a clear example of humans clearing up an error in God's systems.