The new gene therapy can viably cure hemophilia

The New Gene Therapy is under clinical trial to cure hemophilia

Gene Therapy for Hemophilia

An experimental treatment —Gene therapy, which uses a virus to deliver a healthy copy of a gene to compensate for the one causing excessive bleeding, is on a clinical trial. Gene therapy is an advanced technology, but not all illnesses’ can be put on its trial. Out of a few approved, hemophilia is one such condition that has made it for the clinical trial of gene therapy as its possible cure. Gene Therapy for hemophilia is under clinical trial.

Hemophilia is a condition leading to the body’s inability to form blood clots. It is a genetic disorder which makes people vulnerable and bleeds uncontrollably when injured or even with a minor bruise.

Currently, the available treatments for hemophilia are limited, whose potency last less than a day. In a recent clinical trial, James Addie, a 53-year-old with hemophilia, came forward to volunteer for the experiment.

Addie inherited the bleeding disorder quite early in his life. His elder brother, nephew, and baby grandson are also affected by it.

Hemophilia brought with it profuse bleeding, progressive joint damage, and HIV (due to the use of unfiltered blood) for Addie. It is mainly caused due to missing or defective factor VIII, a clotting protein within the body.

The clinical trial has, however, made the 53 years old optimistic. The trial was invented by BioMarin Pharmaceutical. Its major objective is to effectively cure the disease with just one administration, correcting the genetic anomaly that afflicts people.

“The great prospect of the gene therapy is that we can get people to that place where hemophilia’s not something that they wake up in the morning and think about the first thing,” said Dr. Catherine McGuinn, director of the hemophilia treatment center at Weill Cornell Medical Center. “I think of it as a way to protect people sort of every moment of the day.”

The treatment requires the patient to get infused frequently with a clot forming supplements, known as factor replacement therapy. So far, the curing option has seen positive feedback from the patients. When Addie started the trial, his hemophilia was at 0.4 percent (severe), with the level reaching 10 or 12 percent after a month of treatment.

The treatment Addie is helping test will reach the market in 2020, according to Cowen analyst Phil Nadeau. It has an exorbitant cost of about $1 million per patient, which can constraint its accessibility to all. “Gene therapy is a one-time treatment; the complete amount of the therapy has to be paid upon that treatment,” Nadeau said.

Addie is not the only patient in Bio Marin’s clinical trials that experienced a positive result, however, the durability of Gene therapy for hemophilia potential cure is still questionable.

Daniel is our UK-based freelance Editor. As part of our quest towards credible news, Doctor's Clinic Blog India affiliates with individuals from other parts of the world to provide an in-depth focus on essential topics. Daniel received his degree from the University of Sheffield, and since then, worked to multiple sites as a freelance contributor and editor.

Daniel Martin

Daniel is our UK-based freelance Editor. As part of our quest towards credible news, Doctor's Clinic Blog India affiliates with individuals from other parts of the world to provide an in-depth focus on essential topics. Daniel received his degree from the University of Sheffield, and since then, worked to multiple sites as a freelance contributor and editor.

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Daniel Martin

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