All About Multiple Sclerosis

More MS news articles for October 2002

Taking a Risk for Science

http://news.lycos.com/news/story.asp?section=Technology&storyId=546365&topic=sclerosis

Thursday, October 17, 2002 5:00 a.m. EDT
by Kristen Philipkoski

A revolutionary surgery that involves drilling a hole in the skull and injecting cells could repair some devastating effects of multiple sclerosis.

Specialists in multiple sclerosis say it's a bold effort that could have limited results and carries a high risk. But the surgeon leading the clinical trial to test the procedure -- one of the leading researchers in the field -- thinks the risks are worth taking.

Dr. Timothy Vollmer, chief of neuroimmunology at the Barrow Neurological Institute in Arizona, has completed the trial, which included surgery on five patients. No data will be released until it's published in a peer-reviewed scientific journal, probably in December.

Vollmer will speak at the Shepherd Center, a catastrophic care hospital in Atlanta, at a research conference on Friday.

"This is the most extreme approach (to treating multiple sclerosis)," Stephen Reingold, vice president of research at the Multiple Sclerosis Society, said. "They've done it boldly and cautiously as far as I can tell."

The procedure involves taking Schwann cells -- which make up the vital protective sheath that covers nerve cells called myelin -- from the patients' ankles and injecting them into the brain.

In cases of multiple sclerosis, which affects 1.4 million people in the United States, the immune system attacks and destroys myelin. Without it, nerves can't transmit signals properly.

Schwann cells, which are part of the peripheral nervous system, can regenerate in some cases, while myelin cells in the central nervous system cannot.

The researchers are hoping the Schwann cells will incorporate into the patients' neurons, re-coating them and allowing them to function properly.

Even if the surgery works, however, it will not repair the damage to actual nerve fibers that occurs when myelin is destroyed, Reingold said.

The surgery also might not prevent the faulty immune system from attacking myelin again.

"There's no reason to believe they would not be victim to more nerve damage," Reingold said.

However, researchers hope that since Schwann cells are normally immune to damage from multiple sclerosis, they will continue to be so if incorporated into the patients' brains.

Reingold's final concern was that the surgery can target only one brain lesion at a time, while multiple sclerosis patients often have many lesions.

"Dr. Vollmer knows multiple sclerosis very well, and this is a bold step that not many people would have taken at this point," Reingold said.

The transplantation project grew out of another intrepid project started almost 20 years ago.

Augusto Odone, the father of a boy diagnosed in 1984 with a myelin-attacking disease, was not willing to wait for science to find a treatment for Lorenzo, his son. By then it appeared that X-linked adrenoleukodystrophy, or ALD, would likely kill him.

Instead, Odone, an economist, and his wife, Michaela, a linguist, began teaching themselves everything they could about biochemistry. They wanted to find a cure for their son.

They learned that a hallmark of ALD is an excessive buildup of long-chain fatty acids in the blood, which destroy myelin. After poring over scientific journals, they commissioned a lab to produce a mixture of oils that normalized these levels.

"It's not easy to produce," Odone said. "You have to cut off all the 'bad guys' from the oil through molecular distillation. After six months they sent me a sample."

Odone tested the oil on his sisters-in-law, who carry the ALD gene. Women are carriers but don't manifest symptoms.

"When I meet one of them I say, 'You are my favorite rat,'" he said.

Within 10 days of taking the oil regularly, their long-chain fatty acid levels were normal.

Odone convinced Dr. Hugo Moser, the director of the neurogenetics programs at the Kennedy Krieger Institute at Johns Hopkins University, to get the FDA to approve a clinical trial to test the oil.

"Because ALD is such a serious disease, researchers are motivated to take risks," Moser said. "(Also), the biochemical effect of the oil was so striking."

Although Moser was enthusiastic about the study, other researchers were skeptical and dubbed the treatment snake oil.

"There was so much skepticism from the doctors because I was a layperson -- I was an economist; my wife was a writer," Odone said. "They just could not swallow that we beat them to finding the remedy to a rare disease."

Recently, the Odones' work was validated with the completion of Moser's 10-year clinical trial. It showed that Lorenzo's Oil helped prevent symptoms in 52 out of 68 boys with the gene. Thirty-six other boys participated in the study but did not receive the oil -- 53 percent of them developed the disease.

The results won't be conclusive until the boys reach their 30s, since some boys with the ALD gene develop a later-onset version of the disease in their 20s.

The treatment came too late for Lorenzo, who is now 24. He is unable to walk, move his arms or talk. But he has regained his eyesight and his ability to swallow, and can make small movements with his hands.

Since ALD kills most boys who get it, Odone suspects that the oil halted Lorenzo's degeneration. But Moser believes patients must receive the oil before symptoms appear for it to have any effect.

During the course of their research, the Odones played the roll of matchmaker between extremely competitive researchers who were not keen on sharing their results.

"They hated each other's guts -- there was fantastic competition," Odone said. "Over the years we invited them to various meetings, and we always brought them to nice places and fed them beautifully, and now they are collaborating."

A 1992 movie called Lorenzo's Oil about the Odones' story depicts Augusto Odone gathering various scientists together and cooking Italian meals for them.

It was out of this spirit that they founded The Myelin Project, which funded the myelin transplantation surgery clinical trial.

Some experts say the project is too specific, because myelin replacement alone will not give victims of diseases like multiple sclerosis and ALD back their movement, and there is still no way to repair nerve damage or to prevent further immune system malfunctions.

But Odone has high hopes.

"The second chapter of the story is trying to find a method to bring back Lorenzo -- to restore function in him," he said.
 

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