Bone marrow revolution

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Offline Dori

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Bone marrow revolution
« on: June 28, 2010, 03:21:29 PM »

Monday, June 28, 2010

Aurora dad Bob Evanosky is not a perfect match to donate bone marrow to his children, who have a rare, crippling disease.

But a new technique is allowing him to donate bone marrow to two sons in a way that never could have been done before.

The big difference? "You don't die from the new way," Evanosky says.

His three sons have a devastating metabolic disease called metachromatic leukodystrophy, or MLD. Last summer, son John got an experimental outpatient transplant at Duke University.

His quick recovery and exit from the hospital were a far cry from the months his brother Jack had to spend in intensive care after a well-matched transplant the old-fashioned way.

Dad was John's donor even though he's only a half-match. The new cells are making the enzyme the boy's body had lacked, too late to reverse the brain damage that has paralyzed the 8-year-old, but perhaps able to ease some complications.

Evanosky also plans to donate to John's twin Christopher this fall, so this family has new hope.

"If this method is successful," Evanosky says, "it's going to change the way transplants are done."

Bone marrow transplants are undergoing a quiet revolution: No longer just for cancer, they are the focus of research to ease the risks so they can target more people with diseases from sickle cell to deadly metabolic disorders.

The old way: High doses of radiation and chemotherapy wipe out a patient's own bone marrow before someone else's is infused to replace it, hopefully before infection strikes and overwhelms the impaired immune system.

The new way: Rather than destroying the patient's bone marrow, just tamp it down enough to make space for the donated marrow to squeeze in alongside and a sort of twin immune system takes root.

It's what doctors taking a page from mythology call "mixed-cell chimerism" - patient and donor blood and immune cells living together to improve health.

To find the best methods for these less intense transplants, different mixes of low-dose radiation and immune-suppressing drugs are under study at hospitals around the country.

But the ultimate quest is to allow transplants even when donors aren't a good genetic match, says Dr. Suzanne Ildstad of the University of Louisville - whose technique involves an experimental tweaking of donated cells to help them grow better.

"It makes it possible for anyone who has a mom or dad willing to donate marrow to them to have a transplant," says Ildstad, who has families with sickle cell and other childhood genetic illnesses lining up to try.

Separately, several hospitals are testing how to combine kidney transplants with bone marrow transplants from the same donor, in hopes that a hybrid immune system lessens the need for lifelong anti-rejection drugs.

"People are watching with eager expectation," says Dr. Lakshmanan Krishnamurti of Children's Hospital of Pittsburgh, who is helping to plan a multi-hospital study of some of the new methods for hard-to-treat adults with sickle cell disease.

Doctors have long known that a traditional bone marrow transplant can cure young children of sickle cell - if they have a well-matched donor. New marrow produces healthy red blood cells to replace the sickle-shaped ones that can't squeeze through small blood vessels, the cause of the disease's pain, infections and life-threatening organ damage.

But only about 17 percent of children have a suitable donor, usually a healthy sibling. Attempts to transplant adults have failed, their bodies too ravaged from years of the disease. Another hurdle: Certain immune cells in donated marrow sometimes become too aggressive and attack the recipient, called graft-versus-host disease or GVHD.

Enter the new research.

First came a tantalizing success in severely ill adults. Nine of 10 patients who underwent a less intense transplant - using low-dose radiation and two drugs to inhibit problem immune reactions - had their sickle cell apparently eliminated, Dr. John Tisdale and colleagues at the National Institutes of Health reported in December. They developed a hybrid immunity that produces normal red blood cells with no GVHD.

But those people had perfectly matched donor cells provided by healthy siblings. Few patients do.

Back in Louisville, Ildstad gives donated marrow a boost to try to overcome that problem while avoiding GVHD, a risk that worsens with mismatched donors. She removes troublesome immune cells from the donated infusion, leaving concentrated amounts of the blood-producing stem cells patients need plus "facilitating cells" that she discovered seem to help them take root.

In an NIH-funded experiment at Louisville and Duke University, the method so far worked in two children with sickle cell who had well-matched donors and one of four with a half-match.

Dr. Joseph Leventhal of Northwestern University gave an Ildstad-treated stem cell infusion to a handful of kidney transplant recipients who developed hybrid immune systems that seem to be holding nearly a year later. The first three treated are using one anti-rejection drug instead of the usual cocktail, and one soon will attempt full weaning.

Leventhal says they'll do it in patients where it could have potentially the biggest impact: those with unrelated donors.

    * Robert McCoppin of the Daily Herald and Lauran Neergaard of the Associated Press contributed to this report.


Copyright © 2010 Paddock Publications, Inc.

Source: Daily Herald



Offline Sharmin

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Re: Bone marrow revolution
« Reply #1 on: June 28, 2010, 03:26:49 PM »
Thanks for sharing Dore, this sounds great.  It is important to have more than one avenue being explored when searching for a cure.



Offline 7assan

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Re: Bone marrow revolution
« Reply #2 on: June 29, 2010, 06:02:23 PM »
great and that will work with thalassemia ...?


Offline Andy Battaglia

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Re: Bone marrow revolution
« Reply #3 on: June 29, 2010, 09:38:14 PM »
It probably will, since it has been done with mother to child in Italy. The mixed chimerism also appears with cord blood transplants. We are witnessing a revolution in the thinking about BMT. It is becoming much less harsh and more successful.

All we are saying is give thals a chance.


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