BREAKING NEWS : Fort Wayne man loses eye after tear gas canister hits him in face Full Story
BREAKING NEWS : Fort Wayne police arrest nearly 100 people over 2 days of protests that turned violent Full Story
BREAKING NEWS : Protests in Fort Wayne resume for 3rd day, Indiana National Guard on standby Full Story

FDA approves gene therapy for a type of blindness

The US Food and Drug Administration has approved a gene therapy treatment for patients with a rare inherited eye dise...

Posted: Dec 21, 2017 11:47 AM
Updated: Dec 21, 2017 11:47 AM

The US Food and Drug Administration has approved a gene therapy treatment for patients with a rare inherited eye disease.

Voretigene neparvovec, which will be sold as Luxturna, is made by Philadelphia-based Spark Therapeutics Inc.

Luxturna is the first gene therapy treatment for an inherited genetic mutation

The treatment is set to be available in the first part of 2018

The one-time treatment is approved for children and adults with retinal dystrophy due to a mutation of the RPE65 gene, which causes severe visual impairment beginning in infancy. As it progresses, patients experience gradual loss of peripheral and central vision, which can eventually lead to blindness.

There are more than 200 genes that can cause retinal dystrophy. Patients can be tested to determine whether the cause is indeed a mutation of the RPE65 gene and they are thus a potential candidate for this new treatment.

Luxturna is only the third gene therapy approved for use in the United States, preceded by two others this year. However, this is the first to correct an inherited genetic mutation. Some scientists believe that the approval could open the door to other treatments that correct flaws in the inherited genome.

FDA Commissioner Dr. Scott Gottlieb called Tuesday's approval a milestone for gene therapy and noted the potential for treatment of many diseases.

He said the approval "marks another first in the field of gene therapy -- both in how the therapy works and in expanding the use of gene therapy beyond the treatment of cancer to the treatment of vision loss."

Adding a third gene

Luxturna supplies a normal RPE65 gene to retinal cells. It is a liquid that is injected into the eye with a microscopic needle during a surgical procedure. Within the liquid is a gene therapy virus that contains a healthy version of the gene.

Essentially, the drug adds a third version of the gene, which, unlike the mutated genes, is able to code for a protein crucial to vision.

Risks of the treatment include cataracts, elevated eye pressure, retinal tears and holes, and inflammation.

Researchers treated 41 patients between 2007 and 2015 to establish safety and efficacy. Those patients are still being followed. In a separate trial, researchers treated 31 patients and measured changes in their vision based on their ability to complete an obstacle course in low-level light.

The treatment is not permanent, according to William Hauswirth, an ophthalmology professor at the University of Florida College of Medicine who worked on developing it. He said most patients' vision regresses to what it was before therapy after about six years later, but he still considers it successful.

'This is incredible!'

Perhaps the most well-known patient from clinical trials of Luxturna is singer Christian Guardino, who appeared this year on season 12 of "America's Got Talent." The 17-year-old from Patchogue, New York, was eliminated from the show in the semifinals but told his mother, Elizabeth, that the ability to walk unaided onto the stage and see faces in the audience gave him confidence.

In October, he told an FDA advisory committee that the first 12 years of his life were spent in darkness, but he is now able to see things he hadn't seen before, thanks to the treatment.

On Facebook, he called the new approval "the best Christmas gift ever" and said, "this is just incredible!"

When the drug becomes available in early spring, it will become the first approved treatment option for the condition. An estimated 1,000 to 2,000 people in the United States stand to benefit from this option, according to Spark.

The company said cost information and details on timing will be released next month.

Indiana Coronavirus Cases

Data is updated nightly.

Confirmed Cases: 34211

Reported Deaths: 2125
CountyConfirmedDeaths
Marion9761576
Lake3573186
Allen160069
Cass15877
St. Joseph126634
Elkhart126228
Hendricks116671
Hamilton115493
Johnson1097108
Madison58659
Porter53428
Bartholomew50834
Clark49741
LaPorte43023
Howard40428
Tippecanoe3933
Delaware38536
Jackson3821
Shelby37122
Hancock33127
Floyd31839
Boone31635
Morgan27824
Vanderburgh2662
Montgomery23817
White2338
Noble22821
Clinton2271
Decatur22431
Grant21022
Dubois1993
Harrison19422
Henry17211
Greene16924
Vigo1688
Dearborn16821
Monroe16712
Warrick16628
Lawrence15924
Miami1401
Putnam1367
Jennings1304
Kosciusko1271
Orange12622
Scott1193
Franklin1108
Ripley1086
Marshall1021
Carroll932
Daviess8516
Steuben832
Wayne785
Fayette777
Newton7710
Wabash772
LaGrange762
Jasper661
Washington521
Clay511
Jay500
Fulton491
Randolph473
Rush462
Pulaski460
Jefferson451
Whitley413
Starke393
DeKalb371
Sullivan351
Owen341
Brown331
Perry320
Wells310
Benton300
Knox280
Huntington272
Tipton251
Blackford252
Crawford240
Fountain212
Switzerland200
Spencer201
Parke170
Adams171
Posey160
Gibson152
Ohio130
Warren121
Martin110
Vermillion100
Union90
Pike60
Unassigned0167

Ohio Coronavirus Cases

Data is updated nightly.

Confirmed Cases: 35033

Reported Deaths: 2149
CountyConfirmedDeaths
Franklin5773260
Cuyahoga4369226
Marion266028
Hamilton2642148
Lucas2241242
Pickaway205037
Mahoning1428174
Summit1390172
Butler87429
Columbiana74352
Stark72391
Lorain67859
Montgomery65616
Trumbull56948
Belmont42812
Warren36621
Miami35530
Tuscarawas3423
Ashtabula33834
Medina33723
Delaware33113
Portage32357
Lake30612
Wood28746
Geauga28232
Clark2776
Wayne27250
Fairfield2596
Licking24310
Allen21132
Mercer2107
Clermont2065
Richland1993
Erie17114
Darke16721
Madison1527
Washington11819
Crawford1144
Morrow1071
Greene1015
Ottawa9916
Putnam9114
Monroe7812
Auglaize733
Sandusky7311
Ross712
Hocking694
Jefferson672
Hardin600
Huron571
Williams541
Union511
Muskingum500
Hancock501
Clinton431
Shelby413
Wyandot402
Fulton400
Fayette400
Logan380
Coshocton380
Guernsey351
Preble351
Holmes341
Defiance332
Lawrence300
Carroll303
Brown281
Champaign271
Knox251
Highland231
Vinton212
Seneca202
Ashland200
Athens181
Perry181
Henry150
Scioto150
Paulding140
Jackson140
Harrison100
Adams91
Gallia91
Van Wert60
Pike60
Meigs60
Noble60
Morgan50
Unassigned00
Fort Wayne
Clear
61° wxIcon
Hi: 66° Lo: 44°
Feels Like: 61°
Angola
Clear
57° wxIcon
Hi: 62° Lo: 45°
Feels Like: 57°
Huntington
Scattered Clouds
61° wxIcon
Hi: 66° Lo: 44°
Feels Like: 61°
Decatur
Clear
59° wxIcon
Hi: 67° Lo: 44°
Feels Like: 59°
Van Wert
Clear
59° wxIcon
Hi: 67° Lo: 43°
Feels Like: 59°
Sun Continues Monday
WFFT Radar
WFFT Temperatures
WFFT National

Community Events