Advanced Cell Technology, Inc. (ACT) announced today that the U.S. Food and Drug Administration (FDA) has green-lit a human trial of embryonic stem cell therapy to treat a rare condition that causes blindness, according to a press release on the Worcester, Mass.-based company’s website.
Stargardt’s Macular Dystrophy (SMD) causes juvenile macular degeneration, a degenerative disease of the retina in young people that can cause blindness and, according to the scientific journal Nature, affects approximately 1 in 10,000 young people in the United States.
This approval lifts the clinical hold placed on the research a year ago, when ACT applied to start human trials with the therapy last November.
The 1983 Orphan Drug Act (ODA) grants special status for products used to treat rare diseases when requested by a sponsor, subject to certain criteria. This status now qualifies ACT for tax credits and ODA marketing incentives, plus access to grants for trials and up to seven years of market exclusivity.
There is currently no treatment for SMD. Raymond Lund, Ph.D., a world expert on vision restoration and retinal cell physiology who works with ACT, said in a statement that the results from animal trials have been “phenomenal,” adding, “If ACT observes even a fraction of that benefit in humans, it will be nothing short of a home run.”
The controversial decision follows a similar approval last month when human trials of embryonic stem cell therapy began on U.S. patients with spinal injuries, sponsored by the Geron Corporation.
ACT’s Chairman and CEO, William M. Caldwell IV, believes this research is “truly a ‘game changer’ for the medical community.”
Meanwhile, the ethical debate over harvesting human embryos for stem cell therapy remains.
Antagonists view the practice as tantamount to murder, because the treatment is derived from human embryos conceived in vitro specifically for this purpose.
Deacon Keith Fournier states: “Even prior to implantation, a human embryo is a unique living human being with the genetic constitution and epigenetic primordial that continues to develop throughout his or her life,” according to the website of Catholic Online, an online community for Catholics.
Stargardt’s Macular Dystrophy (SMD) causes juvenile macular degeneration, a degenerative disease of the retina in young people that can cause blindness and, according to the scientific journal Nature, affects approximately 1 in 10,000 young people in the United States.
This approval lifts the clinical hold placed on the research a year ago, when ACT applied to start human trials with the therapy last November.
The 1983 Orphan Drug Act (ODA) grants special status for products used to treat rare diseases when requested by a sponsor, subject to certain criteria. This status now qualifies ACT for tax credits and ODA marketing incentives, plus access to grants for trials and up to seven years of market exclusivity.
There is currently no treatment for SMD. Raymond Lund, Ph.D., a world expert on vision restoration and retinal cell physiology who works with ACT, said in a statement that the results from animal trials have been “phenomenal,” adding, “If ACT observes even a fraction of that benefit in humans, it will be nothing short of a home run.”
The controversial decision follows a similar approval last month when human trials of embryonic stem cell therapy began on U.S. patients with spinal injuries, sponsored by the Geron Corporation.
ACT’s Chairman and CEO, William M. Caldwell IV, believes this research is “truly a ‘game changer’ for the medical community.”
Meanwhile, the ethical debate over harvesting human embryos for stem cell therapy remains.
Antagonists view the practice as tantamount to murder, because the treatment is derived from human embryos conceived in vitro specifically for this purpose.
Deacon Keith Fournier states: “Even prior to implantation, a human embryo is a unique living human being with the genetic constitution and epigenetic primordial that continues to develop throughout his or her life,” according to the website of Catholic Online, an online community for Catholics.
