Researchers focus on human testing

The Baltimore Sun

WASHINGTON, D.C. — President Bush‘s veto yesterday of a measure that would have expanded federal funding of research involving human embryonic stem cells promises to prolong years of sparring over whether the work promotes the destruction of life. But in many important ways, ethicists, researchers and even regulators within the Bush administration have moved past the political debate.

 

With state and private funding, the research has moved far enough and fast enough to prompt discussions, writings and the establishment of government committees to prepare for testing the cells in human beings, which is expected in the next five years.

Existing state and federal regulations and recommendations address some of the issues that will arise with human testing, but stem cells present some unique twists, which ethicists, scientists and regulators are now grappling with.

One goal of the preparations is to avoid the political controversy that has so far ensnared the research. A key concern is protecting the safety of the patients who participate in experiments. And, already, there are bitter patent fights over who would control discoveries.

“There’s a recognition across the scientific community that these debates have to occur across the U.S. before the wave of therapies hits our shores or else we’re going to be left with a lot of questions,” said Dr. John O. Agwunobi, assistant secretary for health at the U.S. Department of Health and Human Services.

Agwunobi made it clear that the Bush administration would not support funding for the next wave of research if it involved new lines of embryonic stem cells. But he said the government is preparing to oversee human trials involving all kinds of stem cells, including embryonic ones, to ensure that test subjects are kept safe and any new treatments will work.

In human trials, investigators could implant stem cells into cancer patients who developed brain tumors, children with Tay Sachs disease or adults enduring Lou Gehrig’s disease.

“In all the trials we’ve done, we’ve spent hundreds of hours thinking about” scientific and ethical issues such as which cells to use and which patients to involve, said Dr. Doug Kondziolka. The University of Pittsburgh researcher has finished two trials using mature neural cells on stroke victims and is developing a trial, to begin next year, using stem cells, though not from embryos.

“There’s a lot of tricky stuff happening that has nothing to do with the moral status of the embryo,” said Henry T. Greely, a law professor and director of the Stanford Center for Law and the Biosciences, who has written about the various issues.

One company, Geron Corp. of Menlo Park, Calif., plans to study treating spinal cord injuries using human embryonic stem cells next year.

Last year, the National Academy of Sciences issued guidelines for the proper conduct of research and appropriate handling of cells, and it has just formed a new committee to develop more recommendations.

“There are going to be all of the usual problems associated with doing clinical trials: the consent issues, the safety issues, monitoring problems,” said Jonathan Moreno, a National Academy of Sciences embryonic stem cell committee member who directs the Center for Medical Bioethics at the University of Virginia.

In response to the guidelines, Johns Hopkins and other universities have begun establishing special review panels to make sure that donors gave voluntarily and their confidentiality is protected. The review panels are also to prevent certain projects, such as putting nonhuman cells in human embryos.

The Food and Drug Administration, meanwhile, has convened a committee of experts to help the agency develop standards for making sure stem cell therapies are safe and beneficial before granting approval.

“These days you try to anticipate, especially in this field because of the increased sensitivity and the fact this stuff is really novel,” said Dr. John Gearhart, a stem cell researcher at Johns Hopkins who is serving on the FDA committee.

It was only eight years ago that a University of Wisconsin researcher cultivated the first stem cell, and understanding remains limited enough that regulators and scientists still need to make sure implanting the cells won’t worsen diseases.

Other scientific issues that must be addressed before the cells are tested in human subjects, scientists say, include assuring that the stem cells are produced properly, don’t become contaminated and don’t grow so uncontrollably that they turn cancerous.

Scientists, regulators and ethicists say they are trying to answer these questions now to minimize the potential for problems that might arise when it’s time for clinical trials testing stem cells in human subjects.

“We’re confident that if they’re done right, they will work. And if they work, all of the politics will dissipate,” said Dr. Evan Snyder, who directs the Stem Cell Program at the Burnham Institute for Medical Research in La Jolla, Calif.

The veto by Bush keeps in place the stem cell policy he enumerated in August 2001. The policy allows the federal government to fund research only if it involves stem cell lines derived from embryos gathered before the 2001 announcement.

“This bill would support the taking of innocent human life in the hope of finding medical benefits for others. It crosses a moral boundary that our decent society needs to respect, so I vetoed it,” Bush said yesterday.

As Congress debated the issue this week, a California taxpayer’s group, the Foundation for Taxpayer and Consumer Rights, challenged patent protections on embryonic stem cell discoveries. The challenge seeks to undo three patents governing human embryonic stem cell research controlled by a nonprofit group connected to the University of Wisconsin, Madison.

The filing, with the U.S. Patent and Trademark Office, hints at the sorts of patent, trade secret and other legal issues companies will face as basic stem cell research edges closer to the development of practical treatments a firm could sell to doctors and patients.

The patents held by the Wisconsin Alumni Research Foundation, or WARF, enable the group to charge companies fees for using human embryonic stem cells and obtain annual payments and royalties.

If the WARF patents don’t slow down commercialization of new treatments, a “thicket of patents” obtained by other university technology-transfer groups or companies could, said Kenneth S. Taymor, a corporate lawyer in San Francisco who also works with the Stanford University Program on Stem Cells in Society.

“There are groups all around looking at it,” said Taymor, who co-wrote a recent article that argued some new discoveries may avoid control by existing patents.
————–
Contact the author at: [email protected]