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Feature: At stem-cell research's forefront

By STEVE MITCHELL, Medical Correspondent

A good day for Dr. Robert Lanza, medical director of one of the few U.S. embryonic-stem-cell companies, is when the key to the door to his office still works. Or, when the phones have not been disconnected at Advanced Cell Technology, his company, which is focused on developing medical therapies derived from stem cells in the hopes they could one day treat or cure diseases.

ACT's finances have been so low in recent years it often could not distribute paychecks to its 15 or so employees for several weeks in a row.

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Lanza, the vice president of medical and scientific development, is at the forefront of the debate over cloning and embryonic-stem-cell research. During a recent visit to ACT's offices in Worcester, Mass., he told United Press International he is surprised by the rhetoric in the presidential campaign about these technologies portending a revolution in medicine.

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One of the messages to come out of the debate is stem-cell companies are receiving funding from private investors, but Lanza said this has not been enough to propel the field forward.

Due to a dearth of funding, Lanza said his company has been unable to follow up on promising results in animals and carry out experiments that could lead to life-saving therapies for humans. The financial situation is so dire, the company has at times been unable to afford basic office supplies.

"It's being batted around in the presidential debates ... as a future revolution of medicine and all this and we can't even afford to order purified water for experiments," Lanza said. "People have this misconception that the private sector is pouring in all this money ... but we cannot make payroll and have lost employees. That's the reality. People have no clue. This is what happens when you leave it to the private sector."

President George W. Bush and other opponents are wary of the research because it requires the destruction of an embryo -- a ball of cells so tiny it would fit on the head of a pin -- which they consider to be human life. Bush sought a compromise on the potential benefits of the research and the ethical dilemma and limited federal funding to 78 approved stem cell lines, of which only about 20 are available. Yet, some 400,000 embryos languish frozen in storage at fertilization clinics and an untold number are routinely and legally destroyed.

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Scientists and private investors say the administration's policy as well as actions in Congress have had a stifling effect on stem-cell research and private funding in the United States and there also are concerns the research could be made illegal.

The House of Representatives has passed -- and Bush has signaled his support for -- a total ban on cloning, including therapeutic cloning, which involves the creation of new embryonic-stem-cell lines that could be used to replace damaged or diseased tissue.

The Bush administration also has attempted for two years to get the United Nations to pass a worldwide ban on all forms of cloning -- a move Lanza and other scientists worry would block some applications of embryonic stem cells and perhaps hamper the limited research going on in the United States.

Democratic presidential candidate, Sen. John F. Kerry, D-Mass., has made Bush's policy an issue in the campaign by pledging to eliminate the restrictions on federally funded embryonic-stem-cell research if he is elected.

Opponents of the research have dismissed the claims that embryonic-stem-cell research could yield cures as overblown, because so far it has not yielded any disease treatments. Democratic vice-presidential candidate, Sen. John Edwards, D-N.C., was roundly criticized by conservatives for giving patients "false hope" when he said -- after actor and stem-cell-research activist Christopher Reeve died earlier this month -- that if his running mate John Kerry were elected president, the increased federal funding that would be provided for embryonic-stem-cell research would lead to disease cures and enable paralyzed people like Reeve to get up out of their wheelchairs and walk.

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Lanza said Edwards' statement may not be that far off the mark.

Researchers at his company treated a sheep with a form of the crippling disease spina bifida with cells derived from embryonic stem cells. The animal became healthy and able to walk, instead of being paralyzed, as normally would happen with that disorder.

In addition, scientists from University of California, Irvine, presented research last Sunday at the Society for Neurology meeting in San Diego showing that embryonic stem cells repaired damaged spinal cords in rats and enabled them to walk.

Lanza's company could not afford to follow-up on the promising spina bifida results, so it remains unknown how it might have panned out in further experiments. But he noted the initial experiment was done more than three years ago, so if they had the funding to conduct additional research, it could be in the clinic by now and being studied for potential use in humans, he said.

"There are children now in wheelchairs that might not be," Lanza said. "We would have a number of very substantial breakthroughs by now if we had even basic funding."

Whether the therapy used in the sheep spina bifida model would have helped correct Reeve's condition is uncertain, he said, but without the research it will never be known.

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Bush and his supporters have defended the president's stem-cell policy by noting he is the only president to fund the research with federal dollars and he has not barred the private sector from funding it.

The Department of Health and Human Services issued a fact sheet last July, stating the private sector has pumped in $208 million to the research. However, the study the HHS cited was referring to the worldwide amount of money spread among 30 firms -- some of which do not conduct embryonic-stem-cell research. Only seven of the firms conducting this type of research are located in the United States and the study does not indicate how much of the $208 million they accounted for.

Bill Pierce, an HHS spokesman, said the agency does not have a reliable estimate on how much money the private sector has invested in U.S. embryonic-stem-cell research.

"We just don't know exactly how much money it is," Pierce told UPI. "Our policy has no impact on that and it doesn't address that at all," he said.

The White House did not return a phone call from UPI seeking comment.

ACT was in dire straits until just recently, Lanza said, noting the phones had been turned off for a day a couple of weeks ago because the company could not pay its bill.

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"It was really bad," he said. "We were literally not sure if our entry cards would still work when we showed up for work each morning because we were behind on rent," he said.

Some employees left because they had mortgages or children and could not afford to miss paychecks, but none of them wanted to leave, Lanza said.

"Some of them were in tears," he said. "Everybody wanted to hang in because they believe in this."

ACT is not alone in its financial struggles. Other stem-cell companies are encountering some of the same monetary shortcomings. For example, a California company focused on therapeutic cloning recently posted a net loss of $60.6 million for the first six months of 2004.

Lanza said he has had offers to join major biotech companies and double his salary. He also has been pitched offers to leave the stem-cell field for less-controversial and more-stable positions, but he has turned them down because he is convinced of the potential for stem cells to yield therapies and even cures for currently untreatable diseases.

In addition to the financial woes, ACT has had to fend off the federal government and hostile members of Congress who oppose the research they are doing. (An investigation was launched by HHS in 2001 after 30 congressmen accused the company of misusing federal funds for human cloning, a charge that was found to be unsubstantiated.) The company even has had to defend itself from other biotech companies that seek to guard patents they hold and prevent others from using the technology in research.

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"It's like being on the front lines of a battle," Lanza said.

He said he carries on because of the constant barrage of calls he gets from patients suffering from incurable diseases who are desperate for any therapy that might be able to help them.

"They all want to be the first guinea pig," he said. "It's just heart-wrenching. It's almost criminal" that this research is being stymied, he said. These therapies should be moving forward in animal studies and making their way toward experiments in humans, he added.

A recent experience Lanza had with a local police officer is emblematic of the patients who have been contacting him. The cop sought out Lanza because he had heard of ACT's recent study suggesting embryonic stem cells could be transformed into retinal or eye cells. The policeman wanted to inquire about the possibility of treating his 15-year-old son, who was suffering from a rare eye disorder that probably would leave him completely blind within three years.

"I was almost in tears," Lanza said, "but there's not a lot that can be done without the money for research," he said. "These patients don't have time to wait through four more years of Bush's policy."

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Things may be looking up for ACT. The company thinks it might have secured additional investment funding and it was awarded a nearly $2 million grant from the federal government (although this money cannot be used for therapeutic cloning or to derive new human stem-cell lines).

Meanwhile, Lanza had to ask his secretary to make sure ACT had resolved its debt with the local newspaper before placing an ad seeking to hire additional employees whose salaries would be covered by the grant money.

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E-mail sciencemail@upi.com

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