News Review From Harvard Medical School — Transplant May Help Adults with Sickle Cell

July 2, 2014

News Review From Harvard Medical School -- Transplant May Help Adults with Sickle Cell

A partial transplant of bone-marrow stem cells may reverse sickle cell disease in adults, a new study finds. People with sickle cell disease have abnormally shaped red blood cells. They get stuck in blood vessels. This causes organ damage, pain and other medical problems. The new study included 30 adults with severe sickle cell disease. Each of them had a brother or sister who was a suitable match for a bone-marrow stem cell transplant. The sibling donor's cells were mixed with some of the patient's own cells. During 3.4 years of follow-up, the partial transplant reversed sickle cell disease in 26 out of 30 people, researchers said. In these patients, the bone marrow began making normal red blood cells. Fifteen people also were able to stop taking drugs to prevent rejection of the transplant. Overall, people were much less likely than before to need hospital treatment for the disease. Use of narcotic drugs for pain also was greatly reduced. The Journal of the American Medical Association published the study. HealthDay News wrote about it July 1.

By Howard LeWine, M.D.Harvard Medical School

What Is the Doctor's Reaction?

In the United States, more than 90,000 people are affected by sickle cell disease. Most of them are African-American. Worldwide, the number is much higher. About 300,000 babies are born with this genetic disease every year.

In sickle cell disease, the red blood cells made in the bone marrow are abnormal. Instead of having a normal round shape, the cells are curved and stiff. This causes the red blood cells to get stuck inside blood vessels before they reach the tissues. The result:

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News Review From Harvard Medical School -- Transplant May Help Adults with Sickle Cell

Marrow transplants can reverse adult sickle cell

CHICAGO (AP) Bone marrow transplants can reverse severe sickle cell disease in adults, a small study by government scientists found, echoing results seen with a similar technique used in children.

The researchers and others say the findings show age need not be a barrier and that the technique may change practice for some adult patients when standard treatment fails.

The transplant worked in 26 of 30 adults, and 15 of them were even able to stop taking drugs that prevent rejection one year later.

"We're very pleased," said Dr. John Tisdale, the study's senior author and a senior investigator at the National Institutes of Health. "This is what we hoped for."

The treatment is a modified version of bone marrow transplants that have worked in kids. Donors are a brother or sister whose stem cell-rich bone marrow is a good match for the patient.

Tisdale said doctors have avoided trying standard transplants in adults with severe sickle cell disease because the treatment is so toxic. Children can often tolerate it because the disease typically hasn't taken as big a toll on their bodies, he said.

The disease is debilitating and often life-shortening; patients die on average in their 40s, Tisdale said. That's one reason why the researchers decided to try the transplants in adults, with hopes that the technique could extend their lives.

The treatment involves using chemotherapy and radiation to destroy bone marrow before replacing it with healthy donor marrow cells. In children, bone marrow is completely wiped out. In the adult study, the researchers only partially destroyed the bone marrow, requiring less donor marrow. That marrow's healthy blood cells outlast sickle cells and eventually replace them.

Sickle cell disease is a genetic condition that damages oxygen-carrying hemoglobin in red blood cells, causing them to form abnormal, sickle shapes that can block blood flow through the veins. It can cause anemia, pain and organ damage. The disease affects about 100,000 Americans, mostly blacks, and millions worldwide.

Results from the adult study, involving patients aged 29 on average, were published Tuesday in the Journal of the American Medical Association. The usual treatment hadn't worked, a drug called hydroxyurea, and they had transplants at an NIH research hospital in Bethesda, Maryland.

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Marrow transplants can reverse adult sickle cell

Studies Test Effectiveness and Safety of Stem Cell Treatment for Urinary Incontinence

Durham, NC (PRWEB) July 01, 2014

Medication and minimally invasive surgery to implant a sling can provide relief for millions of people who suffer from stress urinary incontinence (SUI), but not everyone responds to these therapeutic methods. A new study in the current STEM CELLS Translational Medicine tests the safety and effectiveness of stem cells as an alternative SUI treatment.

SUI results when the pelvic floor muscles, which support the bladder and urethra, weaken to the point that the muscles are not able to prevent urine from flowing when pressure is placed on the abdomen, such as when the person laughs or coughs. It occurs most often in women, due to childbirth and pregnancy.

Tissue engineering offers an attractive method to regenerate sphincter muscle, explained the studys corresponding author, Kirsi Kuismanen, from the department of obstetrics and gynecology at Tampere University Hospital (TUH) in Finland. She and her TUH colleagues teamed up with researchers from the Adult Stem Cell Group of BioMediTech in Tampere and the University of Twente in the Netherlands on the study.

Previously, various different cell sources, such as skeletal muscle-derived stem cells (SkMSCs), mesenchymal stem cells derived from bone marrow (BMSCs) and adipose stem cells (ASCs), have been studied for treating urinary incontinence. The SkMSCs and BMSCs would be a potential alternative for incontinence therapy. However, when compared to ASCs, the major limitation of SkMSCs and BMSCs is the difficulty to obtain these cells in large quantities, Dr. Kuismanen said.

The study involved five SUI patients who either did not want a sling implant or had undergone implants but they proved unsuccessful. They were treated with ASCs combined with bovine collagen gel, which is a bulking agent, and saline.

Prior to the treatment, the ASCs were isolated from subcutaneous fat and expanded for three weeks in a laboratory. The mixture of ASCs and collagen was injected in the patients who were followed for three, six and 12 months after the injections. The primary end point was a cough test to measure the effect of the treatment. Validated questionnaires were used to determine the subjective cure rate.

After six months, one out of five patients displayed a negative cough test with full bladder. At one year, the cough test was negative with three patients; two were satisfied with the results and ended their treatment for SUI. Validated questionnaires showed some subjective improvement in all five patients.

This is the first study describing the use of autologous ASCs in combination with collagen gel for female SUI treatments, Dr. Kuismanen said. Thus far, the treatment with autologous ASCs has proven safe and well tolerated. However, the feasibility and efficacy of the treatment were not optimal so additional research is needed to develop SUI injection therapies.

New treatments are needed for this common condition that affects millions of women, said Anthony Atala, M.D., editor of STEM CELLS Translational Medicine and director of the Wake Forest Institute for Regenerative Medicine. The current study, believed to be the first to evaluate adipose-derived stem cells in combination with collagen, adds to the body of knowledge about the safety and effectiveness of stem cell treatments for stress urinary incontinence.

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Studies Test Effectiveness and Safety of Stem Cell Treatment for Urinary Incontinence

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

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

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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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Animal Cell Therapies – She-Dog’s Stem Cell Treatment Story – Video

Animal Cell Therapies - She-Dog #39;s Stem Cell Treatment Story
She-dog was rescued from the streets and had some pretty severe health issues when she walked in the door. A couple months after her stem cell treatment from...

By: Animal Cell Therapies, Inc.

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Animal Cell Therapies - She-Dog's Stem Cell Treatment Story - Video

Cell scientists slow degeneration in motor neuron mice

TOKYO: Japanese stem cell scientists have succeeded in slowing the deterioration of mice with motor neuron disease, possibly paving the way for eventual human treatment, according to a new paper.

A team of researchers from the Kyoto University and Keio University transplanted specially created cells into mice with amyotrophic lateral sclerosis (ALS), also called Lou Gehrig's, or motor neuron disease.

The progress of the creatures' neurological degeneration was slowed by almost eight per cent, according to the paper, which was published on Thursday in the scholarly journal Stem Cell Reports.

ALS is a disorder of motor neurons -- nerves that control movement -- leading to the loss of the ability to control muscles and their eventual atrophy.

While it frequently has no effect on cognitive function, it progresses to affect most of the muscles in the body, including those used to eat and breathe.

British theoretical physicist Stephen Hawking has been almost completely paralysed by the condition.

In their study, the Japanese team used human "iPS" -- induced pluripotent stem cells, building-block cells akin to those found in embryos, which have the potential to turn into any cell in the body.

From the iPS cells they created special progenitor cells and transplanted them into the lumbar spinal cord of ALS mice.

Animals that had been implanted lived 7.8 per cent longer than the control group without the procedure, the paper said.

"The results demonstrated the efficacy of cell therapy for ALS by the use of human iPSCs (human induced pluripotent stem cells) as cell source," the team said in the paper.

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Cell scientists slow degeneration in motor neuron mice

Scientists slow degeneration in motor neurone mice

Friday 27 June 2014 17.27

Japanese stem cell scientists have succeeded in slowing the deterioration of mice with motor neurone disease, possibly paving the way for eventual human treatment.

A team of researchers from the Kyoto University and Keio University transplanted specially created cells into mice with amyotrophic lateral sclerosis (ALS), also called Lou Gehrig's, or motor neurone disease.

The progress of the creatures' neurological degeneration was slowed by almost eight percent, according to the paper, which was published Thursday in the scholarly journal Stem Cell Reports.

ALS is a disorder of motor neurones -- nerves that control movement -- leading to the loss of the ability to control muscles and their eventual atrophy.

While it frequently has no effect on cognitive function, it progresses to affect most of the muscles in the body, including those used to eat and breathe.

British theoretical physicist Stephen Hawking has been almost completely paralysed by the condition.

In their study, the Japanese team used human "iPS" -- induced pluripotent stem cells, building-block cells akin to those found in embryos, which have the potential to turn into any cell in the body.

From the iPS cells they created special progenitor cells and transplanted them into the lumbar spinal cord of ALS mice.

Animals that had been implanted lived 7.8% longer than the control group without the procedure, the paper said.

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Scientists slow degeneration in motor neurone mice

Stem cell transplantation for severe sclerosis associated with improved long-term survival

PUBLIC RELEASE DATE:

24-Jun-2014

Contact: Jacob M. van Laar j.m.vanlaar@umcutrecht.nl The JAMA Network Journals

Among patients with a severe, life-threatening type of sclerosis, treatment with hematopoietic stem cell transplantation (HSCT), compared to intravenous infusion of the chemotherapeutic drug cyclophosphamide, was associated with an increased treatment-related risk of death in the first year, but better long-term survival, according to a study in the June 25 issue of JAMA.

Systemic sclerosis is an autoimmune connective tissue disease characterized by vasculopathy (a disorder of the blood vessels), low-grade inflammation, and fibrosis (development of excess fibrous connective tissue) in skin and internal organs. Previously, small studies have shown that systemic sclerosis is responsive to treatment with autologous HSCT, although it has been unclear whether HSCT improves survival, according to background information in the article. For this study, autologous HSCT involved a multistep process beginning with infusion of high doses of cyclophosphamide and an antibody against immune cells, followed by reinfusion of the patient's own stem cells that had been previously collected from blood and purified.

Jacob M. van Laar, M.D., Ph.D., of the University Medical Center Utrecht, Utrecht, the Netherlands and Dominique Farge M.D., Ph.D, of the Assistance Publique - Hopitaux de Paris, Paris 7 Diderot University, France, and colleagues randomly assigned 156 patients with early diffuse cutaneous (widespread skin involvement) systemic sclerosis to receive HSCT (n = 79) or cyclophosphamide (n = 77; 12 monthly infusions). The phase 3 clinical trial was conducted in 10 countries at 29 centers; patients were recruited from March 2001 to October 2009 and followed up until October 2013.

During a median follow-up of 5.8 years, 53 adverse events occurred: 22 in the HSCT group (19 deaths and 3 irreversible organ failures) and 31 in the control group (23 deaths and 8 irreversible organ failures). Patients treated with HSCT experienced more adverse events (including death) in the first year but had better long-term event-free survival than those treated with cyclophosphamide.

Patients in the HCST group experienced higher mortality in the first year but had better long-term overall survival than those treated with cyclophosphamide. During year 1 there were 11 deaths (13.9 percent, including 8 treatment-related deaths) in the HSCT group vs 7 (9.1 percent, no treatment-related deaths) in the control group. After year 2 of follow-up, there were 12 deaths (15.2 percent) in the HSCT group vs 13 (16.9 percent) in the control group. After 4 years of follow-up, there were 13 deaths (16.5 percent) in the HSCT group vs 20 (26.0 percent) in the control group.

The authors add that HSCT was also more effective than intravenous cyclophosphamide on measures evaluating skin, functional ability, quality of life, and lung function, consistent with previous studies.

"Among patients with early diffuse cutaneous systemic sclerosis, HSCT was associated with increased treatment-related mortality in the first year after treatment. However, HCST conferred a significant long-term event-free survival benefit," the authors conclude.

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Stem cell transplantation for severe sclerosis associated with improved long-term survival