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YOUR HEALTH: When a life-saving transplant becomes deadly

SEATTLE, Washington – Transplants save lives.  But can also cause serious health problems after the surgery is considered a success. Though stem cell transplant...

SEATTLE, Washington – Transplants save lives.  But can also cause serious health problems after the surgery is considered a success.

Though stem cell transplants can be the best hope for a cure from diseases like leukemia, too often rejection, or Graft versus Host disease (GVHD), follows.

And that can be deadlier than the original disease.

BACKGROUND:  Graft versus Host disease, or GVHD, is a condition that may occur after an allogenic (bone and marrow) transplant. The donated bone marrow or peripheral blood stem cells think the body is foreign, and the donated cells will begin to attack the body.   It can develop in the skin, liver, eyes, or gastrointestinal tract and symptoms may appear within weeks after the transplant.   (Source: https://my.clevelandclinic.org/health/articles/graft-vs-host-disease-an-overview-in-bone-marrow-transplant)

The Ferguson family chronicled their son Noah's battle against leukemia, then his bout with GVHD.

The stories are in a book, a thick book.

"He had already gone through chemo and had gone through all of this, and by far, our hardest battle was his graft versus host disease," recalled Noah's mother, Kari Ferguson.

Doctors wanted Noah to get a small amount of GVHD from his stem cell transplant to eliminate remaining cancer cells, but the disease invaded his liver and gut.

He was in and out of the hospital with complications for more than six months.

"He truly has some long term digestive issues that could come back and they could cause problems down the road."

Childhood cancer researcher Dr. Leslie Kean has worked her entire career to find something to prevent GVHD.  She found that therapies that eliminate bad T-cells suppress good ones too.

Until now.

The immunosuppressant rapamycin and an experimental antibody called KY-1005 work together to block pathways and prevent GVHD in animal tests.

"Rapamycin is partially effective by itself.  KY-1005 is partially effective by itself.   But when we put them together is where we really saw really amazing activity," explained Dr. Kean, the associate director of the Ben Towne Center for Childhood Cancer Research at the Seattle Children's Research Institute

Noah's family is excited about the trial's potential.   Maybe someday, Noah will help, when he grows up to be a doctor.

"I've been through so much with all the doctors' help," he said.   "I want to help some other kids, too."

The English biotherapeutic company Kymab is running a phase one trial of the antibody combination on people with psoriasis right now.   Dr. Kean hopes to start designing a GVHD study for KY-1005 and rapamycin within the next year.

SIGNS/SYMPTOMS:   Early treatment can make a big difference in a person`s long-term recovery and health.   Not all people experience warning signs of GVHD, but many experience some of them, and some symptoms may be temporary while others might develop into long-term problems.   Signs of acute GVHD include but are not limited to skin blisters or a rash that looks like sun burn, nausea or loss of appetite, feeling full or bloated, having belly pain or diarrhea and blood in the stool, jaundice, dark urine, or swelling in the legs and belly.   Chronic GVHD may show signs such as nail changes, unusual hair loss or thinning, itchy skin, muscle pain and cramps, dry eyes and blurred vision, trouble opening the mouth, sores and irritations in the mouth, cough and shortness of breath or trouble breathing, irritation or rash in the genital area, and painful intercourse.   (Source: https://bethematch.org/patients-and-families/life-after-transplant/physical-health-and-recovery/gvhd-signs-and-symptoms/)

If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens at jim.mertens@wqad.com and Marjorie Bekaert Thomas at mthomas@ivanhoe.com.