24-Hour Crisis Hotline: (877)SAFEGBC or (877)723-3422 Mental Health & Substance Abuse Issues

6502 Nursery Drive, Suite 100
Victoria, TX 77904
Fax: (361)578-5500

Medical Disorders
Basic InformationLookupsLatest News
Sticking With Meds Lowers Lupus Patients' Diabetes RiskU.S. Coronavirus Cases Reach 34: CDCAHA News: Research Opens New Avenues to Reduce Foot, Toe AmputationsYour Best Bet Against Heart Attack, Stroke? Lower Blood PressureLung Diseases on the Rise WorldwideNew China Coronavirus Cases Decline, 2 Passengers From Affected Cruise Ship DieAHA News: What Women Need to Know About Breast Cancer and Heart DiseaseU.S. Scientists Take Key Step Towards Towards Coronavirus VaccineQuarantine Ends on Cruise Ship in Japan as Coronavirus Cases Near 75,000AHA News: Race and Gender May Tip the Scales on Traditional Stroke Risk FactorsMeasles Complications Can Affect Every Organ: StudyBabies' Exposure to Household Cleaning Products Tied to Later Asthma RiskCoronavirus: Are U.S. Hospitals Prepared?14 Americans From Cruise Ship Hit By Coronavirus Test Positive for InfectionHot Chocolate Could Help Ease Painful Clogged Leg VesselsAntiviral Drug, Plasma Transfusions Show Promise in Treating CoronavirusHow to Dispel Your Child's Fears About the New CoronavirusCholesterol Drugs Might Help Curb 'High-Risk' Prostate CancersCoronavirus Spreads Most Easily When Patients Are Sickest: CDCWill Brushing and Flossing Protect You Against Stroke?Young Black Adults More Prone to Stroke, but Don't Know ItAHA News: Stroke Rates Down for Mexican Americans, Up for White AdultsCoronavirus Cases, Deaths Rise Sharply, While 2 New Cases Reported in U.S.Scientists Spot Antibody That Might Help Diagnose, Treat Autoimmune DisordersCoronavirus in America: Keep Your Panic in CheckCoronavirus Spread Slows, But Death Toll Jumps to 1,113Growing Up in U.S. 'Stroke Belt' Bad for the Brain Later in LifeShingles Vaccine Bonus: Reduced Risk of Stroke?Air Pollution Made in One State Can Cause Deaths in OthersWere You Born in an H1N1 Flu Year or an H3N2? It MattersStricter Clean Air Laws Could Save Thousands of Lives a Year: StudyCoronavirus Fears Have U.S. Pharmacies Running Out of Face MasksCoronavirus Death Toll Tops 1,000, While 13th U.S. Case ConfirmedMeds May Not Prevent Migraines in KidsHigh Testosterone Levels Have Different Health Impact for Men and WomenCoronavirus Cases Top 40,000, While Deaths Hit 908With Macular Degeneration, 1 Missed Visit to Eye Doc Can Mean Vision LossHundreds Suspected, 12 Confirmed: How CDC Identified U.S. Coronavirus CasesFor Patients on Blood Thinners, GI Bleeding May Signal Colon Cancer: StudyStudy Finds 'No Clear Rationale' for 45% of Antibiotic PrescriptionsThere's a Virus Spreading in U.S. That's Killed 10,000: The FluSome U.S. Workers Are Bringing Toxins Home to Their KidsAHA News: Expert Heart Advice for Rare Genetic Muscle Disorder9/11 Study Shows PTSD Tied to Earlier DeathWorkers With Cluster Headaches Take Twice as Many Sick DaysMore Americans to Be Evacuated From China; 12th Coronavirus Case ReportedYoung-Onset Parkinson's May Start in the Womb, New Research SuggestsHealthy Habits Can Slide After Starting Heart MedicationsWide Variations Found in 'Normal' Resting Heart RateLab Discovery Offers Promise for Treating Multiple Sclerosis
Questions and AnswersLinks
Related Topics


Gene Therapy May Be Long-Term Cure for Type of Hemophilia

HealthDay News
by By Dennis Thompson
HealthDay Reporter
Updated: Jan 2nd 2020

new article illustration

THURSDAY, Jan. 2, 2020 (HealthDay News) -- A new gene therapy appears to serve as a functional cure for the most common type of hemophilia, early clinical trial results indicate.

Patients who received the one-time intravenous therapy continue to have a more than 90% decrease in bleeding events two to three years after their initial treatment, researchers reported Jan. 1 in the New England Journal of Medicine.

The therapy fixes a broken gene in liver cells that causes production of flawed factor VIII, a protein that plays a key role in blood clotting.

People with this genetic mutation have hemophilia A, the most common type of this bleeding disorder. Hemophilia A accounts for 8 out of 10 cases of hemophilia, researchers said.

Hemophilia A patients must inject themselves with factor VIII every other day to prevent bleeding, said lead researcher John Pasi, a professor at Barts and The London School of Medicine and Dentistry in England.

"There's been a massive reduction in bleeding in the patients, and none of them any longer need to regularly treat themselves with factor VIII to prevent bleeding," Pasi said of participants in the phase 1/phase 2 clinical trial. "That huge treatment burden of having to give yourself an intravenous injection every other day has gone away."

The therapy uses a virus to carry the DNA sequence for a functional factor VIII gene into liver cells, he said.

"It infects liver cells and transfers into those cells the factor VIII gene," Pasi said. "Liver cells make factor VIII and then secrete it, and it passes into the circulation."

Seven initial participants in the study have a 96% decrease in bleeding events three years out, researchers report. Another six who joined later had a 92% decrease in bleeding by the end of year two.

"At three years, the patients who were treated at a higher dose were expressing functional levels of factor VIII -- somewhat lower than they were at their peak, but they're still at really good levels that are hugely effective in protecting the patients against bleeding," Pasi said.

A phase 3 trial involving more than 130 patients is underway, he said, and therapy manufacturer BioMarin Pharmaceutical has begun the application process with the U.S. Food and Drug Administration.

The most notable side effect is changes in liver enzymes with some patients, but those go away after treatment with steroids, Pasi said.

The treatment will not initially be available for every hemophilia A patient.

Because it involves infecting the liver with a virus, patients with hepatitis or HIV were kept out of the clinical trial, Pasi said. The treatment is only for adults now.

The biggest roadblock for some patients will be whether their bodies have developed an immune reaction to factor VIII, Pasi said.

About a third of patients with severe hemophilia develop antibodies to factor VIII, he said. Half of those cases resolve on their own, and the rest might need to be treated with immune tolerance protocols to get rid of the antibody.

Meg Bradbury, research director at the Hemophilia Federation of America, said the results are "extremely encouraging." But she noted that work needs to continue to find a cure for everyone with a bleeding disorder.

"This therapy and others like it may not be a one-size-fits-all for everyone," Bradbury said. "We still need to think about who is eligible for this particular therapy, who it will be effective for, and how we can help everyone in our community."

The therapy also is not likely to be cheap. Given that it will be a one-time treatment, its pricing will be different from that of a maintenance drug taken regularly, BioMarin representatives said.

They noted that the annual cost of therapy for a hemophilia A patient in the United States is around $500,000.

More information

The Hemophilia Federation of America has more about hemophilia.