Stem Cell Transplant Stops Sickle Cell in Potential Cure

A stem-cell transplant reversed sickle cell disease in adults, according to a study that offers a potential cure for the debilitating condition.

Half of those who had the transplant, which involved the patient and a sibling, also were able to stop taking immunosuppressant drugs without experiencing rejection or having the donor cells attack their body, research released today in the Journal of the American Medical Association showed. People undergoing stem-cell transplants usually must take immunosuppressants for the rest of their lives.

More than 90,000 people in the U.S. have sickle cell disease, a genetic disorder found mostly in people of African descent, according to the U.S. National Institutes of Health. The condition can cause severe pain, organ damage and stroke. Study author Matthew Hsieh said its too soon to say the researchers have found a cure as patients have been followed only for an average of 3 1/2 years, but he is optimistic.

Theyre sickle-cell free for now, Hsieh, a staff clinician at the National Institute of Diabetes and Digestive and Kidney Diseases and the National Heart, Lung, and Blood Institute in Bethesda, Maryland, said today in a telephone interview. We are cautiously optimistic they are cured.

Children with sickle cell can receive a transplant that combines chemotherapy with stem cells, he said. Adults though are usually considered too sick for that treatment.

For a lot of adults, the only option for them is a partial transplant like ours, he said.

The study included 30 patients ages 16 to 65 years who received a transplant that combined their own stem cells and those of a sibling. All the patients had a sibling who was a full match at the white blood cell level, something that occurs about 20 percent of the time, Hsieh said.

Sickle cell disease was reversed in 26 patients, or 87 percent. Fifteen patients discontinued immunosuppressants one year after their transplant and didnt experience rejection or have the donor cells attack their body, the study showed. Patients were enrolled from July 2004 to October 2013.

The research also found that following transplant, the patients use of narcotics for pain declined as did the rate of hospitalization. Lung function also improved, Hsieh said.

Allison King, who wrote an accompanying editorial, said future studies will need to examine if stem cells from partially matched siblings can be just as beneficial.

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Stem Cell Transplant Stops Sickle Cell in Potential Cure

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NIH trial success suggests a new treatment option for older, sicker patients

Half of patients in a trial have safely stopped immunosuppressant medication following a modified blood stem-cell transplant for severe sickle cell disease, according to a study in the July 1 issue of the Journal of the American Medical Association. The trial was conducted at the National Institutes of Healths Clinical Center in Bethesda, Maryland, by researchers from NIHs National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) and the National Heart, Lung, and Blood Institute.

The transplant done in the study reversed sickle cell disease in nearly all the patients. Despite having both donor stem-cells and their own cells in their blood, the patients stopped the immunosuppressant medication without experiencing rejection or graft-versus-host disease, in which donor cells attack the recipient. Both are common, serious side effects of transplants.

Blood drawn from a sickle cell patient. Source: NIH Molecular and Clinical Hematology Branch

Typically, stem-cell recipients must take immunosuppressants all their lives, said Matthew Hsieh, M.D., lead author on the paper and staff clinician at NIH. That the patients who discontinued this medication were able to do so safely points to the stability of the partial transplant regimen.

In sickle cell disease (SCD) sickle-shaped cells block blood flow. It can cause severe pain, organ damage and stroke. The only cure is a blood stem-cell, or bone marrow, transplant. The partial transplant performed in the study is much less toxic than the standard full transplant, which uses high doses of chemotherapy to kill all of the patients marrow before replacing it with donor marrow. Several patients in the study had less than half of their marrow replaced.

Immunosuppressant medication reduces immune system strength and can cause serious side effects such as infection and joint swelling. In this study, 15 of 30 adults stopped taking the medication under careful supervision one year after transplant and still had not experienced rejection or graft-versus-host disease at a median follow up of 3.4 years.

Side effects caused by immunosuppressants can endanger patients already weakened by years of organ damage from sickle cell disease, said John F. Tisdale, M.D., the papers senior author and a senior investigator at NIH. Not having to permanently rely on this medication, along with use of the relatively less-toxic partial stem-cell transplant, means that even older patients and those with severe sickle cell disease may be able to reverse their condition.

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Autologous stem cell treatment could be the road ahead

The treatment could edge out joint replacement procedures to a large extent.

Hyderabad, June 30:

A team of doctors from a city hospital have harvested stem cells of a person using bone marrow from the pelvis area to replace some dead tissues in the hip. By doing this, they saved the patient from undergoing a hip replacement.

The Apollo Health City team, headed by orthopaedic specialist Paripati Sharat Kumar, diagnosed a 39-year-old women suffering from Avascular Necrosis. Her condition would require undergoing a replacement of hips.

After assessing her condition, the team has decided to go for the autologous stem cell procedure (where donor and the receiver is the same person) to save both the hip joints.

The minimally invasive procedure involved taking bone marrow aspirate from the patients pelvis. Stem cells were harvested from the aspirate through a process that takes about 15 minutes. Stems cells were planted in the area of damage under fluoroscopy control following core decompression, Kumar said in a statement on Monday.

He feels that the autologous stem cell treatment could edge out joint replacement procedures to a large extent in the days to come. The scope of this procedure in orthopaedics and sports medicine is enormous. This could be extended to indications including osteoarthritis of knee, shoulder, hip, elbows, ankle and spine, he said.

(This article was published on June 30, 2014)

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Autologous stem cell treatment could be the road ahead