Stem cells tested to repair damaged hearts

Dr. Aidan R. Raney performs a checkup on heart attack patient Mark Athens, 52, on Tuesday, Dec. 17, at Scripps Green Hospital in La Jolla. Athens received a stem cell treatment to help his heart recover as part of a clinical trial to determine the treatments safety and effectiveness.

A new stem cell treatment may help heart attack patients do something once thought medically impossible regenerate dead heart muscle.

Scripps Health in La Jolla is one of three centers testing the therapy from Capricor, a Los Angeles biotech company. The cardiac stem cells are meant to boost the hearts natural ability to perform minor repairs. If it works, scars should shrink and functional heart muscle should grow.

Capricor gets the cells from donor hearts, grows them into the amount needed for treatment, then sends them to doctors taking part in what is called the Allstar trial. Doctors inject the cells into the coronary artery, where they are expected to migrate to the heart and encourage muscle regrowth.

The trial has successfully completed Phase 1, which mainly evaluates safety. On Dec. 17, Capricor said it had received permission to begin Phase 2, which will examine efficacy in about 300 patients who will get the treatment or a placebo. More information can be found at clinicaltrials.gov under the identifier NCT01458405.

The Allstar trial is funded with a $19.7 million disease team grant from the California Institute for Regenerative Medicine, or CIRM, the states stem cell agency.

This is a highly significant announcement for us at CIRM as its the first time weve funded a therapy into a Phase 2 clinical trial, Chairman Jonathan Thomas said in a Dec. 23 statement.

About 600,000 Americans die of heart disease annually, making it the leading cause of death, according to the Centers for Disease Control and Prevention in Atlanta. Even those surviving may be left permanently impaired, if the heart is severely damaged. These are the patients Capricor seeks to help.

Mark Athens received Capricors treatment on Sept. 25, about a month after having a moderate heart attack. The Encinitas resident was the last treated under Phase 1, said Scripps cardiologist Richard Schatz, who performed the procedure. It will take about six months to know whether the treatment worked, Schatz said.

Unlike many trials, Phase 1 was not placebo-controlled, so Athens knows he got the therapy. He appeared cheerful, smiling and bantering with his examining doctor during a Dec. 17 checkup at Scripps Green Hospital.

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Stem cells tested to repair damaged hearts

Stem cell gene therapy shows promise for treating ‘devastating’ skin diseases

Washington, Dec 27 : Researchers evaluated a patient with a genetic skin disorder known as epidermolysis bullosa (EB) nearly seven years after he had undergone a gene therapy procedure as part of a clinical trial.

They found that a small number of skin stem cells transplanted into the patient's legs were sufficient to restore normal skin function, without causing any adverse side effects.

To evaluate stem cell-based gene therapy as a potential treatment, Senior study author Michele De Luca of the University of Modena and Reggio Emilia, and his colleagues previously launched a phase I/II clinical trial at the University of Modena and recruited an EB patient named Claudio.

The researchers took skin stem cells from Claudio's palm, corrected the genetic defect in these cells, and then transplanted them into Claudio's upper legs.

In the new study, De Luca and his team found that this treatment resulted in long-term restoration of normal skin function. Nearly seven years later, Claudio's upper legs looked normal and did not show signs of blisters, and there was no evidence of tumour development. Remarkably, a small number of transplanted stem cells was sufficient for long-lasting skin regeneration. Even though Claudio's skin had undergone about 80 cycles of renewal during this time period, the transplanted stem cells still retained molecular features of palm skin cells and did not adopt features of leg skin cells.

The study is published in journal Stem Cell Reports.

--ANI (Posted on 27-12-2013)

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Stem cell gene therapy shows promise for treating 'devastating' skin diseases

Gene therapy for human skin disease produces long-term benefits

PUBLIC RELEASE DATE:

26-Dec-2013

Contact: Mary Beth O'Leary moleary@cell.com 617-397-2802 Cell Press

Stem cell-based gene therapy holds promise for the treatment of devastating genetic skin diseases, but the long-term clinical outcomes of this approach have been unclear. In a study online December 26th in the ISSCR's journal Stem Cell Reports, published by Cell Press, researchers evaluated a patient with a genetic skin disorder known as epidermolysis bullosa (EB) nearly seven years after he had undergone a gene therapy procedure as part of a clinical trial. The study revealed that a small number of skin stem cells transplanted into the patient's legs were sufficient to restore normal skin function, without causing any adverse side effects.

"These findings pave the way for the future safe use of epidermal stem cells for combined cell and gene therapy of epidermolysis bullosa and other genetic skin diseases," says senior study author Michele De Luca of the University of Modena and Reggio Emilia.

EB is a painful condition that causes the skin to be very fragile and to blister easily, and it can also cause life-threatening infections. Because there is no cure for the disease, current treatment strategies focus on relieving symptoms. To evaluate stem cell-based gene therapy as a potential treatment, De Luca and his colleagues previously launched a phase I/II clinical trial at the University of Modena and recruited an EB patient named Claudio. The researchers took skin stem cells from Claudio's palm, corrected the genetic defect in these cells, and then transplanted them into Claudio's upper legs.

In the new study, De Luca and his team found that this treatment resulted in long-term restoration of normal skin function. Nearly seven years later, Claudio's upper legs looked normal and did not show signs of blisters, and there was no evidence of tumor development. Remarkably, a small number of transplanted stem cells was sufficient for long-lasting skin regeneration.

Even though Claudio's skin had undergone about 80 cycles of renewal during this time period, the transplanted stem cells still retained molecular features of palm skin cells and did not adopt features of leg skin cells. "This finding suggests that adult stem cells primarily regenerate the tissue in which they normally reside, with little plasticity to regenerate other tissues," De Luca says. "This calls into question the supposed plasticity of adult stem cells and highlights the need to carefully chose the right type of stem cell for therapeutic tissue regeneration."

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Stem Cell Reports, De Rosa et al.: "LONG-TERM STABILITY AND SAFETY OF TRANSGENIC CULTURED EPIDERMAL STEM CELLS IN GENE THERAPY OF JUNCTIONAL EPIDERMOLYSIS BULLOSA."

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Gene therapy for human skin disease produces long-term benefits

Day 2 Doing exercise Katrina has to keep healthy to do stem cell treatment that will save her life. – Video

Day 2 Doing exercise Katrina has to keep healthy to do stem cell treatment that will save her life.
Katrina Brown was a British army nurse who has served in Iraq and Afghanistan. Whilst in Iraq she was exposed to Depleted Uranium and this has caused her to ...

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Day 2 Doing exercise Katrina has to keep healthy to do stem cell treatment that will save her life. - Video

BCRO (Stem) Cell Transplants as Treatment For Incurable and …

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MEDISTEM PURCHASED BY INTREXON

By Bradley J. Fikes U-T 12:01 a.m.Dec. 24, 2013

Medistem, a San Diego-based developer of stem cell treatments, has been purchased for about $26 million by biotech Intrexon Corp. of Germantown, Md.

Intrexon will use its genetic engineering technology to increase the potency of Medistems stem cells, said Alan Lewis, Medistems chief executive. The sale, announced Friday, is expected to be completed by the first quarter.

Medistem says its stem cells can form new blood vessels, reduce inflammation and could treat diseases such as diabetes and cardiovascular disease.

These adult stem cells are unusually versatile and can be used in unrelated patients, making them a universal donor kind of stem cell, Lewis said. Derived from the uterine lining, they are called endometrial regenerative cells.

Medistem is a small company, with just a handful of employees. Lewis said he and some others from the company will be taken on by Intrexon, although exact details have yet to be specified.

In January 2012, Medistem began a clinical trial of its cells in Russia in patients with congestive heart failure. The company has done preclinical studies in the United States for a diabetes treatment. Intrexon is developing synthetic biology technology for use in health, food and environmental applications, similar to the goals of genomics pioneer Craig Venter, Lewis said.

Medistems cells will extend Intrexons reach in health care, giving Intrexon the ability to make clinical-grade stem cells, said Samuel Broder, senior vice president of Intrexons health division, in a statement. The division is located in San Diego.

Medistem developed its stem cells as a source of transplantable tissues. Broder said Intrexons technology could also be used to engineer the stem cells to produce protein and RNA-based drugs.

In another announcement last week, Intrexon said it had hired Gregory Frost, CEO of San Diego-based Halozyme, to head its health sector.

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MEDISTEM PURCHASED BY INTREXON

‘I will walk.’ Kim Dennie’s journey of hope brings her closer to goal

Niagara This Week - St. Catharines

THOROLD -- Following a month-long stay in China, Kim Dennie has more faith now than she ever did that her wheelchair will be a part of her past.

On Jan. 13, Dennie left her home in Allanburg on a journey to China for stem cell therapy treatments not available in Canada. After an assessment, Dennie was told the words she has so longed to hear since her life was changed nearly four years ago.

"They told me that my condition is not that bad and I should be able to walk again," Dennie told This Week. "With the right mindset and persistence and continued therapy, I will walk."

In July 2003 Dennie, and her two children, 13-year-old Brittany and 12-year-old Jamie, were returning from a trip to Sudbury. The car she was riding in was broadsided by an oncoming vehicle. The car slammed into the passenger side door where she was sitting. Dennie broke her back, neck, shoulder and pelvis and hasn't been able to walk since.

The initial diagnosis was complete spinal chord injury, leaving her paralyzed from the waist down.

Three months later she was wiggling the toes on her left foot and the diagnosis was changed from complete to an incomplete spinal cord injury. Since that day, Dennie has vowed to herself that she will walk away from the wheelchair she has come to rely on.

She heard about the treatment from a friend she met in therapy and immediately knew it was something worth trying.

Dennie had four rounds of stem cell injection into her spinal chord and another which ran through an IV in her hand. Following the first treatment she saw no changes, but after the second her spasms decreased, her neurological burning pain decreased and she started to regain movement in her right leg.

"I've regained quite a bit of mobility," a beaming Dennie said. "I can sit on the edge of my bed with my leg hanging over it and can lift it back up. I couldn't do any of that before I left."

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'I will walk.' Kim Dennie's journey of hope brings her closer to goal