Health
Gene therapy treats patients with hemophilia B – Hill
A story that can be understood at a glance
- Hemophilia B occurs when a patient lacks sufficient levels of blood protein factor IX.
- Some current treatments for this condition involve repeated injections of protein.
- However, new studies show that one-off gene therapy may have long-term efficacy in patients.
Hemophilia B Rare symptoms It prevents the patient’s blood from coagulating sufficiently and increases the risk of prolonged bleeding.
However, with the new test results released today, researchers argued that the majority of adults with disabilities could be cured. Within the next 3 years.
This is thanks to FLT180a (verbrinacogene setparvovec). This is a gene therapy that works to normalize levels of factor IX, a blood protein that is inadequate in patients with this condition. The results of a multicenter trial involving 10 patients with severe or moderate hemophilia B are: New England Journal of Medicine..
After receiving different doses of FLT180a for 26 weeks, patients were enrolled in a longer follow-up. After 27 months, all but one patient showed sustained levels of factor IX.
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About 10% of adverse events reported during the study are treatment-related, and about a quarter are glucosose, with or without tacrolimus, a substance used by the body to prevent rejection of transplanted organs. It was due to immunosuppression caused by the use of corticoids.
Questions about FLT180a remain, including how long the benefits last for patients, and it is estimated that they may remain effective for at least 10 years.
Gene therapy is making waves in the medical field because of its sustained efficacy, but current trials are from cancer Glaucoma.
These treatments Introduction of new gene or replacement gene To the patient via a viral vector administered via injection. Despite the seemingly long-term efficacy seen with these therapies, they are often expensive and most must be offered in an inpatient setting. However, for some patients, including FLT180a, they only need one treatment to benefit.
Only a handful of gene therapies have been approved in the United States, including treatment for spinal muscular atrophy (SMA) and advanced lymphoblastic leukemia. Zolgensma, an approved treatment for SMA, is currently costly US $ 2.1 million With a single injection, it is the most expensive drug in the country as of 2021.
Published July 21, 2022
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