{"id":47349,"date":"2017-10-13T12:26:19","date_gmt":"2017-10-13T12:26:19","guid":{"rendered":"https:\/\/www.biphoo.com\/bipnews\/?p=47349"},"modified":"2017-10-13T12:26:19","modified_gmt":"2017-10-13T12:26:19","slug":"fda-advisers-urge-approval-for-first-gene-therapy-for-inherited-disease","status":"publish","type":"post","link":"https:\/\/www.biphoo.com\/bipnews\/health\/fda-advisers-urge-approval-for-first-gene-therapy-for-inherited-disease.html","title":{"rendered":"FDA advisers urge approval for first gene therapy for inherited disease"},"content":{"rendered":"

FDA advisers urge approval for first gene therapy for inherited disease<\/span><\/strong><\/span><\/p>\n

The company has not provided an estimated cost for the treatment, but it\u2019s likely to be steep. Wall Street analysts are predicting a price of $750,000 to $1 million for both eyes. That could fuel an already heated debate over the rising cost of pharmaceuticals.<\/span><\/p>\n

Children with the dysfunctional gene often are diagnosed at an early age with disorders such as Leber congenital amaurosis or retinitis pigmentosa. They have limited vision that typically gets worse over time, resulting in night blindness and a loss of peripheral and central vision. Almost all end up completely blind.<\/span><\/p>\n

During an all-day committee meeting Thursday, several young people told stories about how the experimental treatment had transformed their lives, allowing them, for the first time, to see stars and their parents\u2019 faces, and to go out at night with friends.<\/span><\/p>\n

A few years ago, \u201cI was at a precipice of losing it all,\u201d Katelyn Corey, a 24-year-old Los Angeles resident, told the panel. She was falling far behind in her college work as her vision deteriorated and her world literally darkened. After getting the gene therapy in 2013, \u201cI was no longer living in a black-and-white film,\u201d she said. \u201cI may not have gained normal vision, but I gained all of my independence.\u201d She graduated from college and earned a master\u2019s degree in epidemiology.<\/span><\/p>\n

Corey and others who spoke Thursday acknowledged that their vision is far from 20-20 and that it isn\u2019t clear that the improvement will be permanent. Still, the effects have lasted for four years or more, researchers say.<\/span><\/p>\n

The committee\u2019s vote of confidence is a major advance for a field that has struggled for decades to overcome devastating setbacks. \u201cOne of the hopes of the Human Genome Project was to use genes to develop medicines,\u201d said Katherine High, president and head of research and development at Spark. \u201cIt has been much more complex than people imagined, but if we can succeed, it means a lot for people with rare inherited diseases.\u201d<\/span><\/p>\n

Hundreds of gene-therapy clinical trials are underway throughout the world, including for hemophilia and Huntington\u2019s disease, according to the Alliance for Regenerative Medicine, an industry group.<\/span><\/p>\n

For retinal diseases alone, there are more than 18 gene-therapy trials underway, according to Stephen Rose, chief research officer at the Foundation Fighting Blindness. The group financed some of the earlier research for the treatment, whose generic name is voretigene neparvovec.<\/span><\/p>\n

The FDA isn\u2019t required to take the recommendations of its advisory committee, but it usually does. Its deadline for deciding on the Spark treatment is mid-January.<\/span><\/p>\n

Spark was founded in 2013 based on research and a substantial investment from Children\u2019s Hospital of Philadelphia (CHOP). In a pivotal trial involving about 30 patients ages 4 to 44, most patients who received the treatment showed improvement in navigating a novel maze \u2014 with obstacles and arrows for directions.<\/span><\/p>\n

The obstacle course was designed to measure patients\u2019 \u201cfunctional vision,\u201d or the ability to handle daily activities in lowlight conditions. The patients didn\u2019t see statistically significant gains in \u201cvisual acuity\u201d \u2014 that is, in reading an eye chart.<\/span><\/p>\n

During the meeting, researchers showed a video of a 6-year-old patient who, before getting the treatment, bumped into objects on the obstacle course and was unable to complete it quickly. At age 10, years after being treated, she was able to move through the course easily in seconds.<\/span><\/p>\n

Christian Guardino, a 17-year-old high school senior from \u00adPatchogue, N.Y., was diagnosed with the disorder when he was less than a year old. \u201cWe literally had to keep all the lights on,\u201d said his mother, Elizabeth. \u201cHe was going completely blind and he couldn\u2019t navigate on his own.\u201d<\/span><\/p>\n

In 2012, Christian, who testified at Thursday\u2019s meeting, got the treatment as part of the trial at CHOP and his vision improved immediately. Now, his mother said, he can play sports, go out with friends at night and read books, albeit ones with large print.<\/span><\/p>\n

Christian describes the change in his life as \u201cincredible.\u201d Recently, he competed on NBC\u2019s \u201cAmerica\u2019s Got Talent,\u201d wowing the judges with his powerful voice. He was especially happy, he said, to be able to see the judges so clearly.<\/span><\/p>\n

Jean Bennett, an ophthalmologist at the University of Pennsylvania who has been involved in the development and testing of the gene therapy, said the treatment allows children to become much more independent. \u201cThey can walk around, play sports,\u201d she said.<\/span><\/p>\n

Source:-\u00a0https:\/\/www.washingtonpost.com\/national\/health-science\/fda-advisers-urge-approval-for-first-gene-therapy-for-inherited-disease\/2017\/10\/12\/d6f4d432-aec2-11e7-9e58-e6288544af98_story.html?utm_term=.e54fef9b32f8<\/span><\/p>\n

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