Sight-saving research halted by stem cell ruling

ARVO members investigate hESC therapies for treating diseases such as age-related macular degeneration (AMD), diabetic retinopathy, glaucoma and corneal disease, along with studies related to eye tissue transplantation, regeneration and engineering. ARVO's policy supports technological developments and policies that encourage all facets of stem cell research, including research utilizing hESCs.

"A generation of researchers may turn away from pluripotent stem cell research in general as a result of this ruling," says one ARVO member. This researcher uses stem cell lines that were in use for many years prior to President Obama's Executive Order and were approved for use earlier this year under retroactive 2009 guidelines governing the use of hESCs. But because of the District Court injunction, a key grant for research on human retinal development has been stopped. He adds: "Left to those who continue to claim that adult stem cells hold all the same properties as pluripotent stem cells (a vocal minority), the stem cell field could regress substantially. In addition, promising research in other areas related to embryonic stem cell research may never reach its full potential."

A scientist working on translational AMD studies stated, "This ruling will disrupt or halt important stem cell research programs, and it represents a major setback to the hopes of hundreds of thousands of patients who stand to benefit from the outcomes of this work . . . These pioneering trials will require much additional research to refine stem cell therapies and to improve their safety and efficacy before they can become routinely available to patients on a large scale. It is critical that the National Institutes of Health (NIH) support the basic and translational studies that are essential to achieve this goal and to move the field forward. The decision will not only impact ongoing studies, but will also send a very negative message to scientists and physicians, particularly young researchers, who have dedicated their careers to pursue this promising new area of science. We can only hope that the ruling will be reversed promptly."

Much progress in vision science with hESCs is occurring outside the United States. ARVO members work under liberal stem cell policies in Australia, Belgium, China, India, Israel, Japan, South Korea, Sweden and the United Kingdom. Vision scientists in the United Kingdom aim to have a hESC therapy for AMD available for clinic use by 2011.

ARVO members working under restrictive stem cell policies include those in the United States, Austria, Germany, Ireland, Italy, Norway and Poland. Barred from federal funding, United States researchers expect to use private funds for a hESC trial for juvenile macular degeneration (Stargardt's disease). However, there simply is not enough private funding to cover all embryonic stem cell research. An irreparable loss to biomedical research will result from NIH being forced to stop peer review of 50 grants, halt final review of 12 grants worth $15-20 million and stop renewal of 22 grants worth $54 million. Overall, this puts the United States further behind the curve in solving major health problems that could bring relief to millions of suffering patients and their families.

Judge Lamberth claimed his ruling "would not seriously harm hESC researchers because the injunction would simply preserve the status quo and would not interfere with their ability to obtain private funding for their research." This prediction may be internally consistent with his legal ruling, but affected researchers have found that, contrary to the judge's expectation, the ruling immediately disrupted research. As one vision scientist put it, extramural scientists overnight were forced to separate any infrastructure, equipment, consumables or personnel funded by Federal agencies from those funded by private sources, completely obstructing all research activities. Worse, all intramural hESC projects at NIH have been terminated. As ARVO Board members have emphasized, "Restarting these research programs after political issues are resolved will be extremely difficult, and in some cases, likely will be impossible. Human embryonic stem cells are being maintained in cultures in labs around the world. Precious cell cultures at NIH and elsewhere could be lost if ongoing work is halted until political decisions are resolved."

ARVO stands behind the statement made by NIH Director Francis Collins, MD, PhD, "The injunction threatens to stop progress in one of the most encouraging areas of biomedical research, just as scientists are gaining momentum — and squander the investment we have already made. The possibility of using these cells to replace those that have been damaged by disease or injury is one of the most breathtaking advances we can envision. Human embryonic stem cells also represent a powerful new approach to the early stages of screening for new drugs, and may hold the secrets to creating entirely new, targeted clinical therapies. We must move forward — without delay — to sustain this field of research that provides so much hope for thousands of patients and their families."

### About ARVO

ARVO is the world's largest international association of eye and vision scientists. Our members include more than 12,600 scientists from over 80 countries. ARVO encourages and assists research, training, publication and knowledge-sharing in vision and ophthalmology. www.arvo.org

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