Category Archives: Stem Cell Therapy

Buddy the beagle can walk again
Buddy the beagle wasn #39;t able to walk when he first arrived at the University of Minnesota Veterinary Medical Center. With the help of U of M veterinarians and staff, using stem-cell therapy,...

By: UMN Veterinary Medical Center

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Buddy the beagle can walk again - Video

Roughly 40 million people across the world are blind and, for a long time, most forms of blindness were permanent conditions. The same situation held for degenerative diseases that affect eyesight.

But recently, scientists have made some surprising headway into changing that. New treatments like gene therapy, stem-cell therapy, and even bionic implants are already starting to restore some patients' sight. And these technologies are expected to keep improving in the future.

Here's a look at all the ways scientists have tried and, increasingly, succeeded in curing the blind:

Children's Hospital of Philadelphia, Daniel Burke/AP Photo This undated image released by the Children's Hospital of Philadelphia shows doctors Albert Maguire, left, along with wife Jean Bennett at the University of Pennsylvania. The two are part of two teams of scientists in the United States and Britain that are using gene therapy to dramatically improve vision in four patients with an inherited eye disease that causes blindness in children.

Tweaking genes is one promising route to treat blindness.

In 2011, a group led by Jean Bennett of the University of Pennsylvania used gene therapy to treat some patients with a congenital blindness disorder. The patients in question all hada hereditary disease called Leber congenital amaurosis, and they all had mutations in their RPE65 gene.The patients were each given a non-harmful virus that could sneak a healthy copy of the gene into their eye cells. Six out of 12 showed improvement.

Then, in 2014, researchers led by Robert MacLaren, an ophthalmologist at Oxford,presented some promising early results of a very smallstudy of six patients at various stages of a rare, inherited disease calledchoroideremia. These patients all lacked a protein calledREP1, which leads to progressive vision loss. Doctors took the gene forREP1, put it in a non-harmful virus, and injected that virus into the patients' eyes. All reported some improvement in their sight.

"One patient, who before his treatment could not read any lines on an eye chart with his most affected eye, was able to read three lines with that eye following his treatment,"wrote Susan Young Rojahn at MIT Technology Review.

Commercial treatments are still a ways off, however. Researchers first have to continue to monitor these patients to see what happens to their vision over the long term (and check for side effects).The FDA currently recommends 15 years of safety monitoring before trying to get a specific gene therapy approved.

2) Stem cells

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4 strategies doctors are using to cure the blind

Elite Emage Stem Cell Therapy
Elite Emage Stem Cell Therapy.

By: Elite Emage

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Elite Emage Stem Cell Therapy - Video

AVON LAKE, OH (WOIO) - When Shannon Goulding's bloodhound Butler tore a ligament in his knee his entire personality changed.

"He was sedentary, and he wasn't as active as before," said Goulding.

Dr. Petti a veterinarianat the Avon Lake Animal Clinic told Goulding, who also works at the clinic, suggested that stem cell therapy could help.

"Watching him walk he looked stiff and uncomfortable," said Petti.

The therapy was successful. Goulding said after four weeks after the surgery she could see a change the way Butler moved.

Stem cell therapy helps animals suffering from sore knees and joints by using their own fat cells.

"You take them from the patient, you process them, make them active, and then you re inject them into the parts of the animal that are giving them problems," said Petti.

Petti said Avon Lake Animal Clinic has helped about 15 animals with stem cell therapy and people from all over the country have been calling.

One injection of stem cells can last up to three years, and after that a second injection may be needed.

Stem cell therapy is also an expensive procedure. It ranges from $2,000-2,500, but for Goulding she says seeing Butler run free without pain is worth it.

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Local clinic treats animals with stem cell therapy

Kilian Before After Stemlogix Stem Cell Therapy
dog with arthritis treated with autologous stem cells.

By: mark Greenberg

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Kilian Before & After Stemlogix Stem Cell Therapy - Video

PUBLIC RELEASE DATE:

19-Nov-2014

Contact: Lauren Woods lauren.woods@mountsinai.org 646-634-0869 The Mount Sinai Hospital / Mount Sinai School of Medicine @mountsinainyc

Delivering stem cell factor directly into damaged heart muscle after a heart attack may help repair and regenerate injured tissue, according to a study led by researchers from Icahn School of Medicine at Mount Sinai presented November 18 at the American Heart Association Scientific Sessions 2014 in Chicago, IL.

"Our discoveries offer insight into the power of stem cells to regenerate damaged muscle after a heart attack," says lead study author Kenneth Fish, PhD, Director of the Cardiology Laboratory for Translational Research, Cardiovascular Research Center, Mount Sinai Heart, Icahn School of Medicine at Mount Sinai.

In the study, Mount Sinai researchers administered stem cell factor (SCF) by gene transfer shortly after inducing heart attacks in pre-clinical models directly into damaged heart tissue to test its regenerative repair response. A novel SCF gene transfer delivery system induced the recruitment and expansion of adult c-Kit positive (cKit+) cardiac stem cells to injury sites that reversed heart attack damage. In addition, the gene therapy improved cardiac function, decreased heart muscle cell death, increased regeneration of heart tissue blood vessels, and reduced the formation of heart tissue scarring.

"It is clear that the expression of the stem cell factor gene results in the generation of specific signals to neighboring cells in the damaged heart resulting in improved outcomes at the molecular, cellular, and organ level," says Roger J. Hajjar, MD, senior study author and Director of the Cardiovascular Research Center at Mount Sinai. "Thus, while still in the early stages of investigation, there is evidence that recruiting this small group of stem cells to the heart could be the basis of novel therapies for halting the clinical deterioration in patients with advanced heart failure."

cKit+ cells are a critical cardiac cytokine, or protein receptor, that bond to stem cell factors. They naturally increase after myocardial infarction and through cell proliferation are involved in cardiac repair.

According to researchers there has been a need for the development of interventional strategies for cardiomyopathy and preventing its progression to heart failure. Heart disease is the number one cause of death in the United States, with cardiomyopathy or an enlarged heart from heart attack or poor blood supply due to clogged arteries being the most common causes of the condition. In addition, cardiomyopathy causes a loss of cardiomyocyte cells that control heartbeat, and changes in heart shape, which lead to the heart's decreased pumping efficiency.

"Our study shows our SCF gene transfer strategy can mobilize a promising adult stem cell type to the damaged region of the heart to improve cardiac pumping function and reduce myocardial infarction sizes resulting in improved cardiac performance and potentially increase long-term survival and improve quality of life in patients at risk of progressing to heart failure," says Dr. Fish.

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Delivering stem cells into heart muscle may enhance cardiac repair and reverse injury

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Newswise UCLA stem cell researchers have pioneered a stem cell gene therapy cure for children born with adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), often called Bubble Baby disease, a life-threatening condition that if left untreated can be fatal within the first year of life.

The groundbreaking treatment was developed by renowned stem cell researcher and UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research member Dr. Donald Kohn, whose breakthrough was developed over three decades of research to create a gene therapy that safely restores immune systems in children with ADA-deficient SCID using the patients own cells with no side effects.

To date, 18 children with SCID have been cured of the disease after receiving the stem cell gene therapy in clinical trials at UCLA and the National Institutes of Health.

All of the children with SCID that I have treated in these stem cell clinical trials would have died in a year or less without this gene therapy, instead they are all thriving with fully functioning immune systems said Kohn, a professor of pediatrics and of microbiology, immunology and molecular genetics in Life Sciences.

To protect children born with SCID they are kept in isolation, in controlled environments because without an immune system they are extremely vulnerable to illness and infection that could be lethal.

Other current options for treating ADA-deficient SCID are not always optimal or feasible for many children, said Kohn. We can now, for the first time, offer these children and their families a cure, and the chance to live a full healthy life.

Defeating ADA-Deficient SCID: A Game-Changing Approach

Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases, and in a child with ADA-deficient SCID even the common cold can prove fatal. The disease causes cells to not create an enzyme called ADA, which is critical for production of the healthy white blood cells that drive a normal, fully-functioning immune system. About 15 percent of all SCID patients are ADA-deficient.

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UCLA Stem Cell Researcher Pioneers Gene Therapy Cure for Children with "Bubble Baby" Disease

Researchers at the Cedars-Sinai Heart Institute have found that injections of cardiac stem cells might help reverse heart damage caused by Duchenne muscular dystrophy, potentially resulting in a longer life expectancy for patients with the chronic muscle-wasting disease.

The study results were presented today at a Breaking Basic Science presentation during the American Heart Association Scientific Sessions in Chicago. After laboratory mice with Duchenne muscular dystrophy were infused with cardiac stem cells, the mice showed steady, marked improvement in heart function and increased exercise capacity.

Duchenne muscular dystrophy, which affects 1 in 3,600 boys, is a neuromuscular disease caused by a shortage of a protein called dystrophin, leading to progressive muscle weakness. Most Duchenne patients lose their ability to walk by age 12. Average life expectancy is about 25. The cause of death often is heart failure because the dystrophin deficiency leads to cardiomyopathy, a weakness of the heart muscle that makes the heart less able to pump blood and maintain a regular rhythm.

"Most research into treatments for Duchenne muscular dystrophy patients has focused on the skeletal muscle aspects of the disease, but more often than not, the cause of death has been the heart failure that affects Duchenne patients," said Eduardo Marbn, MD, PhD, director of the Cedars-Sinai Heart Institute and study leader. "Currently, there is no treatment to address the loss of functional heart muscle in these patients."

During the past five years, the Cedars-Sinai Heart Institute has become a world leader in studying the use of stem cells to regenerate heart muscle in patients who have had heart attacks. In 2009, Marbn and his team completed the world's first procedure in which a patient's own heart tissue was used to grow specialized heart stem cells. The specialized cells were then injected back into the patient's heart in an effort to repair and regrow healthy muscle in a heart that had been injured by a heart attack. Results, published in The Lancet in 2012, showed that one year after receiving the experimental stem cell treatment, heart attack patients demonstrated a significant reduction in the size of the scar left on the heart muscle.

Earlier this year, Heart Institute researchers began a new study, called ALLSTAR, in which heart attack patients are being infused with allogeneic stem cells, which are derived from donor-quality hearts.

Recently, the Heart Institute opened the nation's first Regenerative Medicine Clinic, designed to match heart and vascular disease patients with appropriate stem cell clinical trials being conducted at Cedars-Sinai and other institutions.

"We are committed to thoroughly investigating whether stem cells could repair heart damage caused by Duchenne muscular dystrophy," Marbn said.

In the study, 78 lab mice were injected with cardiac stem cells. Over the next three months, the lab mice demonstrated improved pumping ability and exercise capacity in addition to a reduction in heart inflammation. The researchers also discovered that the stem cells work indirectly, by secreting tiny fat droplets called exosomes. The exosomes, when purified and administered alone, reproduce the key benefits of the cardiac stem cells.

Marbn said the procedure could be ready for testing in human clinical studies as soon as next year. The process to grow cardiac-derived stem cells was developed by Marbn when he was on the faculty of Johns Hopkins University. Johns Hopkins has filed for a patent on that intellectual property and has licensed it to Capricor, a company in which Cedars-Sinai and Marbn have a financial interest. Capricor is providing funds for the ALLSTAR clinical trial at Cedars-Sinai.

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Cardiac stem cell therapy may heal heart damage caused by Duchenne muscular dystrophy