Monthly Archives: September 2013

When Will Gene Therapy Come to the United States?

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Susan Young for MIT Technology Review 2013-09-30 14:11:37 UTC

Though many gene therapies have been tested in patients around the world in hopes of curing hereditary diseases, few governments have approved their sale, and none has been approved in the United States. That could change in coming years as several therapies enter advanced trials.

A big step forward already came in November 2012, when the European Medicines Agency gave the Dutch biotech startup UniQure permission to sell its treatment. That approval came as a relief to many in the field, who had been waiting for a break in the clouds hanging over the technology since failed and fatal trials in the 1990s. You see a resurgence in terms of investors, and in truth, a number of problems have been solved, says Katherine High, a medical researcher at Childrens Hospital of Philadelphia, who is overseeing a late-stage clinical trial for a different gene therapy.

Still, experts say it is likely to be a few years before a treatment is approved in the U.S. With its European approval in hand, UniQure may have a good chance of also getting the first U.S. approval, but the company says it has not yet submitted an application to the FDA.

Like most gene therapies, UniQures treatment uses a modified virus to deliver a working copy of a gene to patients who lack a healthy version. In this case, the gene is needed for the body to break down fats; without it, patients can develop painful and even fatal inflammation of the pancreas. UniQure uses a modified version of a virus that most of us already carry. The choice of virus used to deliver a gene therapy depends in part on where the treatment needs to go in the body and whether the viruses are intended to replicate themselves. Some viruses, for instance, are designed to spread throughout the body to kill cancer cells.

There are several groups that could be the first to develop a U.S.-approved gene therapy (see table). Highs team is one; they are enrolling patients in a late-stage trial of a treatment for a disorder that causes blindness at an early age. The patients in this trial have previously been given the gene therapy in one eye, and now the other will be tested.

In the experimental treatment, doctors inject a virus-based particle just behind a patients retina. The treatment improved some patients vision to the point that they were no longer legally blind. Some patients have been stable for nearly six years. The trial is scheduled to end in April 2015.

Another possibility comes from Massachusetts-based Bluebird Bio, which has published results from patients who have seemingly been cured of a genetic blood disease. The company is about to start testing its approach in a hereditary neurological disorder that is often fatal in young boys.

SEE ALSO: Gene Therapy Combats Hereditary Blood Disease

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When Will Gene Therapy Come to the United States?

When Will Gene Therapy Come to the U.S.?

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Several gene therapies are or will soon be in late-stage human trials. One of them could be the first to get FDA approval for sale in the U.S.

Though many gene therapies have been tested in patients around the world in hopes of curing hereditary diseases, few governments have approved their sale, and none has been approved in the United States. That could change in coming years as several therapies enter advanced trials.

A big step forward already came in November 2012, when the European Medicines Agency gave the Dutch biotech startup UniQure permission to sell its treatment. That approval came as a relief to many in the field, who had been waiting for a break in the clouds hanging over the technology since failed and fatal trials in the 1990s. You see a resurgence in terms of investors, and in truth, a number of problems have been solved, says Katherine High, a medical researcher at Childrens Hospital of Philadelphia, who is overseeing a late-stage clinical trial for a different gene therapy.

Still, experts say it is likely to be a few years before a treatment is approved in the U.S. With its European approval in hand, UniQure may have good chance of also getting the first U.S. approval, but the company says it has not yet submitted an application to the FDA.

Like most gene therapies, UniQures treatment uses a modified virus to deliver a working copy of a gene to patients who lack a healthy version. In this case, the gene is needed for the body to break down fats; without it, patients can develop painful and even fatal inflammation of the pancreas. UniQure uses a modified version of a virus that most of us already carry. The choice of virus used to deliver a gene therapy depends in part on where the treatment needs to go in the body and whether the viruses are intended to replicate themselves. Some viruses, for instance, are designed to spread throughout the body to kill cancer cells.

There are several groups that could be the first to develop a U.S.-approved gene therapy (see table). Highs team is one; they are enrolling patients in a late-stage trial of a treatment for a disorder that causes blindness at an early age. The patients in this trial have previously been given the gene therapy in one eye, and now the other will be tested.

In the experimental treatment, doctors inject a virus-borne gene just behind a patients retina. The treatment improved some patients vision to the point that they were no longer legally blind. Some patients have been stable for nearly six years. The trial is scheduled to end in April 2015.

Another possibility comes from Massachusetts-based Bluebird Bio, which has published results from patients who have seemingly been cured of a genetic blood disease (see Gene Therapy Combats Hereditary Blood Disease). The company is about to start testing its approach in a hereditary neurological disorder that is often fatal in young boys.

In a different form, gene therapy could also become an option for cancer treatment. At a meeting this summer, Amgen announced that it had met its goals for an advanced test of a gene therapy for melanoma that has spread from the skin to other parts of the body. The Amgen treatment, which was engineered from a virus that normally causes cold sores, takes a two-pronged approach to fighting cancer. The virus selectively infects cancer cells, where it replicates until the cell bursts. While growing inside the cell, the virus also produces a protein that rouses the immune system. When the cell explodes, immune cells are attracted to the tumor site to fight the disease.

In a test in patients with late-stage melanoma, 26 percent of patients whose cancer had spread saw a partial or complete tumor response for at least six months. In 11 percent of patients, the cancer completely disappeared, which suggests that the therapy spreads throughout the body, targeting tumors that werent initially injected. Overall survival rates for cancer patients in the trials are expected to be reported in the first half of 2014.

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When Will Gene Therapy Come to the U.S.?

Mark Howitt – Metal Master Kingdom: March of Freedom Interview (08-06-2013) – Video

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Mark Howitt - Metal Master Kingdom: March of Freedom Interview (08-06-2013)
Interview with Mark Howitt by Ingrid Newton from Metal Master Kingdom about the March of Freedom. This exclusive Interview took place in Toronto on August 6,...

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American Freedom Radio Producer Needs Your Donations! Vinny Eastwood 24Sep2013 – Video

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BLACKLIST # 31 – Fifth Freedom «» Let’s Play Splinter Cell Blacklist | Full-HD – Video

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Story County Freedom Flight begins with send-off ceremony

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The second Story County Freedom Flight is Tuesday, but it began Sunday at Ames City Auditorium with a send-off ceremony honoring the veterans who will be on the flight.

This trip has been on my bucket list, Gary Evans, a Vietnam veteran from Nevada, said. Im just excited to be going.

This years participants are 31 World War II veterans, 50 Korean War veterans and 69 Vietnam veterans, including two women. All of the veterans on the flight either live in Story County now or did when they enlisted.

This means a lot because weve been there, done that, and served our country with pride, Eldon Boswell, of Nevada, said. Boswell, who served during the Korean War, said he wanted to see the monuments with this group, and that there was a sense of brotherhood in seeing them together.

On Sunday, the veterans gathered first at Iowa State University for a motorcade tour through the campus and downtown Ames. The veterans rode on buses with a motorcycle escort and were accompanied by area law enforcement officials and firefighters. The motorcade was greeted when it arrived at City Auditorium by a large crowd of friends and family members.

Highlights of the ceremony included a medley of service branch songs, a performance of God Bless America and a special presentation of an honorary high school diploma to veteran Donald Phipps.

Phipps was a member of the 2012 Freedom Flight, and received his honorary diploma through the Iowa Department of Veterans Affairs Operation Recognition program. The program recognizes veterans who didnt complete their high school diplomas because of service during World War II, the Vietnam War or the Korean War. Phipps, a World War II veteran, received his honorary diploma from Boone High School.

He was then also presented with a letter of honorary acceptance to ISU, signed by ISU President Steven Leath.

Also presented at the ceremony were a Gold Star wreath and POW/MIA flag.

The Gold Star wreath, which represents soldiers who lost their lives in service, will be taken on Tuesdays trip and placed on the Tomb of the Unknowns at Arlington National Cemetery, Tony Nussbaum, Sundays master of ceremonies, said.

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Americans' freedom story revealed in 50 documents

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Sunday September 29, 2013

Americans freedom story revealed in 50 documents

Proclamation from Harpers Ferry abolitionist among pieces highlighted

The Associated Press

In this Tuesday, Sept. 24, 2013 photo, Erin Paulson stabilizes a 1900th century document at the The Historical Society of Pennsylvania in Philadelphia. The society's new web site charts the evolution of American freedom, a story of struggle and conflict through 50 carefully selected historic documents, from an early draft of the Articles of Confederation to a suffragette's letter to her parents to a 1970's tract on gay rights. The organization hopes its interactive "Preserving American Freedom" web site will gain favor with educators.

PHILADELPHIA -- The Historical Society of Pennsylvania holds more than 21 million documents and items. It was Rachel Moloshok's job to sort, sift and select just 50.

What was she looking for? No less than a history of how Americans have defined freedom and how they've fought for it.

After two years of effort, the fruits of her labor can be seen on a new interactive website that charts the evolution of what many Americans take for granted. It's a 350-year-old tale of struggle and conflict told through original documents both famous and obscure, from an early draft of the Articles of Confederation to a suffragette's letter home, from a signed copy of the Emancipation Proclamation to a 1970s speech arguing against the classification of homosexuality as a mental disorder.

The documents are a reminder that Americans' quest for freedom didn't end with the British surrender at Yorktown, or the ratification of the Constitution and Bill of Rights. Rather, it was just beginning, with succeeding generations claiming freedom for themselves and fighting for it, nurturing it and protecting it.

"The way I define this project is not necessarily tracing the history of American freedom, but essentially the history of how Americans have defined or have envisioned and struggled for freedom," said Moloshok, project director of the Preserving American Freedom initiative. "At some point, everybody who has struggled for their own freedom kind of turns around and says, 'Yeah, but we didn't mean you.' It's this great, very complicated history of struggle."

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