ACTC coverage, along with GERN...
Blinded by the Light: Eye-Popping Amount of Positive Media Coverage of ACT’s FDA ApprovalACT’s announcement last Monday, November 22nd that it has received approval to start a Phase I/II Human Clinical Trial using human embryonic stem cells to treat eye disease has been greeted with a cacophony of enthusiastic media coverage from around the world.
From the scientific outlets such as Nature and Scientific American to business wires such as Reuters and AP, to mainstream outlets such as TIME, CNN.com, USA Today and BBC News, the amount of press coverage this small company has received for its announcement has been fully in line with the momentous nature of the news itself.
Nature magazine enthusiastically reports on the approval news in a blog piece, that opened with this passage:
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The second-ever clinical trial of a therapy involving the use of human embryonic stem cells has received the green light from US regulators.
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The piece later appears to raise the possibility of collaboration with Big Pharma with this passage:
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Macular degeneration has been seen as stem cell therapy’s low-hanging fruit because the cells that need replacing are so accessible, both for delivering the treatment and for monitoring its effects. The eye is also an immune-privileged site – meaning that injections won’t elicit an immune response. Last year, the pharmaceutical giant Pfizer agreed to back the work of researchers at University College, London, who are developing a similar therapy.
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The piece also favorably contrasts ACT’s Phase I/II trial with Geron’s, which plans to use embryonic stem cells to treat spinal cord injury:
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Just one other trial for a human embryonic stem cell-based therapy — developed by Geron, a stem cell company based in Menlo Park, California, to treat spinal cord injury — is currently underway. That trial first gained approval in January 2009, but was halted due to safety concerns. It was cleared to restart this summer, and its first patient received the treatment in October.
Lanza says ACT’s macular degeneration trial is likely to be significantly more straightforward. “The advantage, of course, is that we’re talking about a very small number of cells going into a very local area,” he says. Using instruments that can track a single retinal cell in the eye in real time, the researchers will also be able to easily monitor patients’ progress.
“Also, with the eyes there are very objective tests for visual acuity,” he notes, “so we can measure performance gains very objectively.” Tracking improvement after spinal cord injury, on the other hand, is notoriously tricky.
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In a story titled, “New embryonic stem cell experiment, the Washington Post discusses the disease that is the focus of the clinical trial:
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Stargardt’s Macular Dystrophy, which is one of the most common forms of juvenile macular blindness, progressively destroys vision, usually beginning in childhood. It is currently incurable.
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The story also comments on the positive reception ACT’s clinical trial is receiving from stem cell researchers, as well as Geron’s (Nasdaq: GERN) trial using embryonic stem cells to treat spinal cord injury:
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The studies are being welcomed by many stem cell researchers, patients and advocacy groups.
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In its coverage, USA Today includes this remark:
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The firm hopes to gain approval for similar trials to treat macular degeneration, which affects the vision of perhaps 10% of the elderly nationwide.
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As you may know, the first major milestone in this process was cleared in a few short days later: on November 30, ACT submitted an Investigational New Drug Application (IND) to the US Food and Drug Administration (FDA) for the first-ever clinical trial using embryonic stem cells to treat Dry Age-Related Macular Degeneration, a $25-30 billion combined market in the US and Europe alone.
CNN Medical Managing Editor, Miriam Falco, conducted an interview with ACT’s Chairman and Chief Executive Officer, William M. Caldwell IV, and quotes him in her story:
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William Caldwell, CEO of Massachusetts-based Advanced Cell Technology (ACT), tells CNN that 366 days after filing the application, the FDA granted approval for his company to start a clinical trial using cells grown from human embryonic stem cells. The treatment will be for an inherited degenerative eye disease.
“We’re still absorbing the fact that we finally got the approval” says Caldwell. “The real work lies ahead.” Getting the trial going is the next big step, says Caldwell. He hopes the first patients can be enrolled by the first quarter of the next year.
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Ms. Falco continues:
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If this treatment works, Caldwell believes it could be applied to other diseases that lead to blindness, including age-related macular degeneration, which affects millions of Americans and will only increase as baby boomers continue to get older.
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TIME Magazine Senior Reporter Alice Park reports:
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For only the second time, the Food and Drug Administration approved a company’s request to test an embryonic stem cell-based therapy on human patients. Advanced Cell Technology (ACT), based in Marlborough, Mass., will begin testing its retinal cell treatment this year in a dozen patients with Stargardt’s macular dystrophy, an inherited degenerative eye disease that leads to blindness in children.
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The protocol involves injecting 50,000 to 100,000 RPE cells into the vitreous cavity of the eye. Because this region is protected from the body’s ever-vigilant immune system, the new RPE cells do not have to be immunologically matched to the patients. But since they are made from embryonic stem cells, which can go on to become any of the body’s various tissue types as well as form a type of tumor, the FDA was concerned that some pure stem cells would remain in the transplanted RPE population. Lanza says the company’s scientists have developed an assay that can detect a single such stem cell from about one million RPE cells, a measure that satisfied the agency and may become a standard for future embryonic stem cell based treatments.
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For the moment, however, Lanza says the current study will be focused on establishing the safety of the RPE therapy, with any visual improvement a welcome bonus. “We’re starting out with a safety study in those with advanced disease,” says Lanza. “But hopefully if we get in earlier in the progression of the disease we might see greater impact on visual improvement. What this approval shows is that the readiness of the FDA to work with researchers to move exciting new stem cell based therapies out of the lab and into the clinic.”
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The wire service Reuters reports:
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Stargardt’s affects only 30,000 to 50,000 Americans, according to the American Macular Degeneration Foundation, but ACT has its sights set on a much larger market if this trial is successful — age-related macular degeneration.
This blinding condition affects more than 30 million people worldwide and is the leading cause of blindness in people over 60 in the United States.
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Associated Press covered the news in a feature story, here, which included the following outtakes:
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For only the second time, the U.S. government has approved a test in people of a treatment using embryonic stem cells — this time for a rare disease that causes serious vision loss.
Advanced Cell Technology, a biotechnology company based in Santa Monica., Calif., said the research should begin early next year, following the green light from the U.S. Food and Drug Administration.
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ACT’s experiment will focus on Stargardt disease, which affects only about 30,000 Americans. But the company hopes the same approach will work for similar and more common eye disorders like age-related macular degeneration, which affects millions.
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The BBC conducted an audio interview with Chief Scientific Officer Dr. Robert Lanza. This enjoyable interview is available in online streaming form here.
Rounding out the wire services, Agency France Press (AFP), the third-largest wire service, aimed at European audiences, covers the news with the enthusiasm that has characterized nearly all the coverage:
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Human embryonic stem cells will be tested as a treatment for blindness, a US company announced Monday in the second such clinical trial to examine how the controversial process works in people.
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The process has been tested on rats and mice and has been found to halt the progressive disease without causing tumors or other side effects, said chief scientific officer Bob Lanza at the biotech company Advanced Cell Technology.
“These cells have been really performing quite spectacularly in the animals. If we can see that in the human patients we will hit a home run here,” said Lanza.
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Stargardt’s disease causes blindness by destroying the pigmented layer of the retina, called the retinal pigment epithelium (RPE). After that follows degradation of photoreceptors, which are cells in the retina that detect light.
Patients often experience blurry vision, difficulty seeing in low-light and eventually most lose their ability to see at all. The disease can be inherited by a child when two parents carry the gene mutation that causes it.
“This is a horrific disease that affects children starting at age six, so they are very young and they go blind as teenagers,” said Lanza. “The hope would be to treat patients earlier to prevent this from happening at all.”
The treatment process being tested by ACT worked in animals by creating an abundance of new RPE cells, Lanza explained.
“Using stem cells, we can generate a virtually unlimited supply of healthy RPE cells, which are the first cells to die off in (Stargardt’s) and other forms of macular degeneration,” Lanza said.
The process showed 100 percent improvement in rats’ visual performance and “near-normal function” was also achieved in mice, both without negative side effects, he said.
Researchers expect to see results in real-time due to the use of high resolution instruments that allow them to track the cells in the eye, with an assessment of the progress possible within weeks.
“We should be able to know what’s going on along the way,” said Lanza. “It is quite conceivable we could see improvement within six weeks or so.”
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The Singularity Hub, a popular blog about futuristic technology, science and medical innovations, gushed enthusiastically in an in-depth feature story:
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For only the second time in history, the US FDA has granted approval for clinical trials for a therapy derived from human embryonic stem cells. Advanced Cell Technology (ACT), a Massachusetts based bio-firm,recently announced that they had secured approval for human trials of their retinal pigment epithelial cells to treat Stargardt’s Macular Dystrophy.
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This is just the tip of the iceberg. The press response to this announcement has been nothing short of tremendous.
We invite you to peruse the “ACT In the News” page for links to some of the most in-depth coverage, as well as simply doing a Google News search for “Advanced Cell Technology.” The enthusiastic attention continues, just as ACT continues going about its work. |
