A doctor who gave experimental blood treatments in Atlanta to patients with ALS --- or Lou Gehrig's disease --- is under investigation by the Food and Drug Administration for not seeking the agency's approval and allegedly overselling the promise of the procedure.

Dr. Mitchell Ghen, an alternative medicine specialist from South Carolina, treated 43 patients with the progressive and fatal nerve disease between August and early this month. They traveled to Atlanta from several other states and England, paying $25,000 each.

After the FDA got a search warrant to obtain Ghen's medical records and contacted the Florida blood bank supplying him, he stopped offering the treatments at the Institute of Cellular Medicine in Dunwoody.

Ghen was transfusing umbilical cord blood, which is rich in stem cells. The primordial cells, under the right conditions, can turn into life-sustaining neurons and other cells. The burgeoning field has only recently come under detailed FDA regulations.

One other physician in the country, Dr. Dan Cosgrove of California, was performing the transplants on ALS patients. After treating 13 patients beginning in December, Cosgrove stopped this month after learning about the FDA action against Ghen, he said.

Probe into promises

An FDA spokeswoman confirmed that the agency is investigating Ghen. FDA officials said he should have sought the agency's approval.

Ghen referred a reporter's call to his Houston-based lawyer, Richard Jaffee, who said that Ghen didn't oversell the treatment and didn't need FDA approval.

Jaffee said the probe also focuses on whether Ghen improperly told patients they would be cured.

Few drugs are available for ALS --- or amyotrophic lateral sclerosis --- which gradually paralyzes the muscles, taking away the ability to walk, talk, swallow and breathe. There is no cure, and ALS usually kills within two to five years.

About 30,000 Americans have it, with roughly 5,000 new cases each year.

Researchers in New Jersey and Boston recently found that cord blood transplants on mice with ALS slightly prolonged their life.

Ghen, 53, started offering the treatments at the request of his patients, Jaffee said. People came to Atlanta for weekend visits, during which up to 20 units of cord blood were transfused intravenously through their arms.

Each patient paid $25,000 --- not covered by insurance --- which includes $15,000 for the blood and $10,000 for administration, equipment and other expenses, Jaffee said.

The blood came from Cryobanks International, a private blood bank near Orlando, where people pay to store cord blood for their family's use or donate it for research or transplantation.

Of the 43 patients Ghen treated, some improved, some stabilized and some showed no benefit, Jaffee said. All were adults, except one infant with a pediatric version of the disease.

Blood shipments stop

Two patients died after the treatments --- one from pneumonia and one from ALS, Jaffee said.

The FDA visited Ghen's clinic on Feb. 7, acting on the search warrant, Jaffee said. After the FDA also contacted Cryobanks, it stopped shipping blood to Ghen early this month, said Bruce Mackler, a Washington-based attorney for the blood bank.

Ghen then stopped the treatments.

FDA's regulations regarding cell therapies such as cord blood transplants are evolving. Rules were proposed in 1998 and published in detail in 2001, with some aspects now in effect and others delayed, an FDA spokeswoman explained.
The rules say that therapies in which cells are manipulated or used for purposes other than their natural function must undergo clinical testing and be submitted to the FDA for approval.

Even if Ghen's treatments fall under those rules, Jaffee argues that Ghen is protected by "off-label" drug use, in which the FDA frequently allows physicians to prescribe drugs for purposes other than those on the label.

But the FDA hasn't approved cord blood transplants for any purpose, the spokeswoman said.

The other issue at least partly centers on a letter one of Ghen's assistants read over the phone to an FDA investigator posing as a family member of an ALS patient. The letter told of a patient who dramatically improved after the treatment.

Ghen's Web site offers a somewhat cautionary depiction of the treatment, which Jaffee said has been on the site for months.

Researchers mixed

It reads: "If the results are similar to the animal studies, if and when the procedure if successful in humans, [there] may be a significant delay in the progress of the disease and possible improvement in current disability."
Researchers give the idea of cord blood transplants for ALS mixed reviews.
"In the mice, it has an effect, but not a huge effect," said Lucie Bruijn, science director for the ALS Association in Los Angeles.

Christoper Pazoles, chief operating officer for the ALS Treatment Development Foundation in Boston, said it would be difficult to coax stem cells into enough neurons to reverse the disease. But he said the treatment could help in other ways.

ALS patients say they can't wait for the slow pace of science and government.
Rob Senecal, a 37-year-old computer specialist from New York, came to Atlanta for the treatment in December, two years after being diagnosed with ALS.

His leg muscle strength improved, his breathing stabilized, feeling came back in his right thumb and he was able to sleep better, said his wife, Maryrose Senecal. His speech continued to get worse, she said.

"It's not fair to keep people from trying something that may benefit them," she said.

Fran Hoggatt, of Wichita, Kan., was scheduled to get the treatment from Ghen this month, but it was canceled.

Hoggatt, 58, learned she had ALS in November, after she couldn't touch her tongue to her lip during a diagnostic test. Her speech is now slurred, and she can eat only on one side of her mouth.

She feels her days slipping by.

"I am still very mobile, but this is really slowing me down," Hoggatt said. "I've got a lot of things in life I want to do yet."