- Obesity Genes Mean Some Folks Must Exercise More for Same Results
- SCOTUS Appears Skeptical of Arguments to Curb Abortion Pill Access
- Sleep Troubles Can Raise Your Blood Pressure: Study
- ADHD Meds Tied to Heart Damage in Young Adult Users
- Could Regular Exercise Cure Your Insomnia? New Research Says Yes
- Black Men Less Likely to Receive Heart Transplants Than White Men or Women
- Could Deep Frying Foods Harm the Brain? Rat Study Suggests It Might
- Human Brains Are Getting Larger With Each Generation
- Animals Catch More Viruses From Us Than We Do From Them
- Young Adults With Migraine May Face Higher Stroke Risk
Gene Therapy Makes Inroads Against a Form of Hemophilia
People with hemophilia B could find their bleeding risk dramatically reduced with just one injection of an experimental gene therapy, a new study reports.
Hemophilia B is a rare and inherited genetic disorder in which people have low levels of the factor IX (FIX) protein, which is needed for forming blood clots.
Patients have to inject themselves regularly with a synthetic version of the FIX protein.
But a new gene therapy called FLT180a led to sustained production of FIX protein in 9 of 10 patients participating in early trials, removing the need for regular replacement injections, researchers reported.
After 26 weeks, five patients still had normal levels of FIX protein, three had levels that had flagged but were still higher than before, and one had an abnormally high level, researchers said.
The therapy works by using a hollowed-out virus to deliver a functional copy of the FIX protein gene to replace the one that isn’t working.
While the treatment was generally well tolerated, all patients experienced some side effects. This included an abnormal blood clot in a patient who received the highest dose of the therapy and later produced the highest levels of FIX protein.
Researchers plan to track the patients for 15 years to judge the long-term safety and durability of the approach.
“Removing the need for hemophilia patients to regularly inject themselves with the missing protein is an important step in improving their quality of life,” said lead researcher Dr. Pratima Chowdary, a hematologist at the Royal Free Hospital in London and University College London (UCL) Cancer Institute.
“The long-term follow-up study will monitor the patients for durability of expression and surveillance for late effects,” she said in a UCL news release.
The findings were published July 21 in the New England Journal of Medicine.
More information
The National Hemophilia Foundation has more about hemophilia B.
SOURCE: University College London, news release, July 20, 2022
Source: HealthDay
Copyright © 2024 HealthDay. All rights reserved.