CLOSINGS: View Closings

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.

Cases: 726600

Reported Deaths: 13379
CountyCasesDeaths
Marion992781737
Lake53359964
Allen40387675
St. Joseph35449550
Hamilton35440408
Elkhart28376439
Tippecanoe22336217
Vanderburgh22271396
Porter18637306
Johnson17876377
Hendricks17154314
Clark12919191
Madison12574339
Vigo12426245
Monroe11845169
LaPorte11773210
Delaware10615185
Howard9859215
Kosciusko9366117
Hancock8239140
Bartholomew8042155
Warrick7766155
Floyd7645177
Grant7019174
Wayne7018199
Boone6669101
Morgan6547139
Dubois6148117
Marshall6000111
Dearborn578577
Cass5780105
Henry5681102
Noble558283
Jackson500472
Shelby489496
Lawrence4492120
Gibson434391
Harrison434271
Clinton426853
DeKalb425284
Montgomery423388
Whitley394439
Huntington387480
Steuben382957
Miami380266
Knox371890
Jasper362747
Putnam358560
Wabash353178
Adams340654
Ripley338970
Jefferson328881
White312854
Daviess295899
Wells290481
Decatur283592
Fayette278662
Greene276685
Posey270933
LaGrange264770
Scott264753
Clay259245
Randolph239781
Washington239532
Spencer231331
Jennings229248
Starke214452
Fountain211946
Sullivan211042
Owen197056
Fulton194640
Jay191830
Carroll188120
Perry182637
Orange182454
Rush172925
Vermillion168343
Franklin167735
Tipton162345
Parke145916
Blackford134532
Pike133034
Pulaski116045
Newton107034
Brown101541
Crawford99114
Benton98414
Martin87815
Warren81415
Switzerland7848
Union70710
Ohio56211
Unassigned0413

Ohio Coronavirus Cases

Data is updated nightly.

Cases: 1080121

Reported Deaths: 19344
CountyCasesDeaths
Franklin1254981392
Cuyahoga1115202107
Hamilton798171200
Montgomery512711012
Summit46997933
Lucas41982782
Butler38273580
Stark32190907
Lorain24910480
Warren24249297
Mahoning21474586
Lake20586368
Clermont19727238
Delaware18496131
Licking16396210
Fairfield16144199
Trumbull15996466
Medina15233262
Greene15027244
Clark13960297
Wood13056188
Portage12806201
Allen11609231
Richland11308198
Miami10659215
Muskingum8795132
Wayne8777210
Columbiana8756229
Pickaway8547121
Marion8511135
Tuscarawas8467243
Erie7859154
Hancock6900126
Ross6835152
Ashtabula6777169
Geauga6665148
Scioto6398101
Belmont5856167
Union570047
Lawrence5542102
Jefferson5511151
Huron5422119
Darke5344122
Sandusky5330120
Seneca5268121
Athens518258
Washington5148109
Auglaize488484
Mercer480185
Shelby468593
Knox4479110
Madison435461
Putnam4268100
Ashland421289
Fulton420969
Defiance418997
Crawford3965106
Brown392957
Logan381176
Preble379098
Clinton370561
Ottawa366279
Highland353861
Williams338075
Champaign330458
Guernsey315453
Jackson312051
Perry294550
Morrow283839
Fayette281449
Hardin270364
Henry268166
Coshocton264258
Holmes2590101
Van Wert242863
Pike237433
Adams237052
Gallia235048
Wyandot230654
Hocking215162
Carroll191247
Paulding172440
Meigs144639
Noble133337
Monroe131942
Morgan108323
Harrison107537
Vinton82415
Unassigned02
Fort Wayne
Cloudy
39° wxIcon
Hi: 59° Lo: 36°
Feels Like: 39°
Angola
Cloudy
39° wxIcon
Hi: 56° Lo: 37°
Feels Like: 36°
Huntington
Clear
43° wxIcon
Hi: 59° Lo: 35°
Feels Like: 43°
Decatur
Cloudy
39° wxIcon
Hi: 60° Lo: 37°
Feels Like: 39°
Van Wert
Cloudy
45° wxIcon
Hi: 60° Lo: 37°
Feels Like: 40°
We'll start Friday off dry, but the chance of rain returns. Scattered showers will be possible by late morning until the evening.
WFFT Radar
WFFT Temperatures
WFFT National

Community Events