Mount Sinai researchers awarded grant to find new stem cell therapies for vision recovery

PUBLIC RELEASE DATE:

20-Nov-2014

Contact: Jessica Mikulski jmikulski@nyee.edu 212-979-4274 The Mount Sinai Hospital / Mount Sinai School of Medicine @mountsinainyc

The National Eye Institute (NEI), a division of the National Institutes of Health, has awarded researchers at the Icahn School of Medicine at Mount Sinai a five-year grant totaling $1 million that will support an effort to re-create a patients' ocular stem cells and restore vision in those blinded by corneal disease.

About six million people worldwide have been blinded by burns, trauma, infection, genetic diseases, and chronic inflammation that result in corneal stem cell death and corneal scarring.

There are currently no treatments for related vision loss that are effective over the long term. Corneal stem cell transplantation is an option in the short term, but availability of donor corneas is limited, and patients must take medications that suppress their immune systems for the rest of their lives to prevent rejection of the transplanted tissue.

A newer proposed treatment option is the replacement of corneal stem cells to restore vision. The grant from the NEI will fund Mount Sinai research to re-create a patient's own stem cells and restore vision in those blinded by corneal disease. Technological advances in recent years have enabled researchers to take mature cells, in this case eyelid or oral skin cells, and coax them backward along the development pathways to become stem cells again. These eye-specific stem cells would then be redirected down pathways that become needed replacements for damaged cells in the cornea, in theory restoring vision.

"Our findings will allow the creation of transplantable eye tissue that can restore the ocular surface," said Albert Y. Wu, MD, PhD, Assistant Professor, Department of Ophthalmology at the Icahn School of Medicine at Mount Sinai and principle investigator for the grant-funded effort. "In the future, we will be able to re-create a patient's own corneal stem cells to restore vision after being blind," added Dr. Wu, also Director of the Ophthalmic Plastic and Reconstructive Surgery, Stem Cell and Regenerative Medicine Laboratory in the Department of Ophthalmology and a member of the Black Family Stem Cell Institute at Icahn School of Medicine. "Since the stem cells are their own, patient's will not require immunosuppressive drugs, which would greatly improve their quality of life."

Specifically, the grant will support efforts to discover new stem cell therapies for ocular surface disease and make regenerative medicine a reality for people who have lost their vision. The research team will investigate the most viable stem cell sources, seek to create ocular stem cells from eyelid or oral skin cells, explore the molecular pathways involved in ocular and orbital development, and develop cutting-edge biomaterials to engraft a patient's own stem cells and restore vision.

###

Follow this link:
Mount Sinai researchers awarded grant to find new stem cell therapies for vision recovery

Bubble Boy Disease Cured With Stem Cells

TIME Health medicine Bubble Boy Disease Cured With Stem Cells Alysia Padilla-Vacarro and daughter Evangelina on the day of her gene therapy treatment. Evangelina, now two years old, has had her immune system restored and lives a healthy and normal life. Courtesy of UCLA Researchers have treated more than two dozen patients with a treatment made from their own bone marrow cells

Alysia Padilla-Vaccaro and Christian Vaccaro owe their daughters life to stem cells. Evangelina, now two, is alive today because she saved herself with her own bone marrow cells.

Evangelina, a twin, was born with a severe immune disorder caused by a genetic aberration that makes her vulnerable to any and all bacteria and viruses; even a simple cold could be fatal. But doctors at University of California Los Angeles (UCLA) Broad Stem Cell Research Center gave her a new treatment, using her own stem cells, that has essentially cured her disease. Shes one of 18 children who have been treated with the cutting-edge therapy, and the studys leader, Dr. Donald Kohn, says that the strategy could also be used to treat other gene-based disorders such as sickle cell anemia.

Known to doctors as adenosine deaminase (ADA)-deficient severe combined immunodeficiency (SCID), its better known as bubble boy disease, since children born with the genetic disorder have immune systems so weak that they need to stay in relatively clean and germ-free environments. Until Evangelina and her sister Annabella were 11 months old, We were gowned and masked and did not go outside, says their mother Alysia Padilla-Vaccaro. Our children did not physically see our mouths until then because we were masked all the time. We couldnt take them outside to take a breath of fresh air, because there is fungus in the air, and that could kill her.

Both parents wore masks at work to lower the chances they would be exposed to germs that they might bring back home. And they took showers and changed clothes as soon as they entered the house.

MORE: Gene-Therapy Trial Shows Promise Fighting Bubble Boy Syndrome

SCID is caused by a genetic mutation in the ADA gene, which normally produces the white blood cells that are the front lines of the bodys defense against bacteria and viruses. The Vaccaros decided to treat Annabella in the same way that they cared for Evangelina; They were crawling and playing with each other, and every toy they sucked on, they stuck in each others hands and each others mouth, so we couldnt take one outside to have a grand old time and potentially bring something back that could harm her sister, says Padilla-Vaccaro.

The only treatments for SCID are bone marrow transplants from healthy people, ideally a matched sibling; the unaffected cells can then repopulate the immune system of the baby with SCID. But despite being her twin, Annabella wasnt a blood match for her sister, nor were her parents. Padilla-Vaccaro and her husband, Christian, were considering unrelated donors but were concerned about the risk of rejection. We would be trying to fix one problem and getting another, she says.

MORE: Stem Cells Allow Nearly Blind Patients to See

Thats when the doctors at the Childrens Hospital at Orange County, where Evangelina was diagnosed, told her parents about a stem cell trial for SCID babies at UCLA, led by Dr. Donald Kohn. As soon as they said trial, I thought, my kid is dead, says Padilla-Vaccaro of the last resort option. But a dozen children born with other forms of SCIDin which different mutations caused the same weak immune systemswho were successfully treated by Kohn convinced the couple that the therapy was worth trying. Kohn had one spot left in the trial and was willing to hold it for Evangelina until she matured more. Born premature, she was diagnosed at six weeks old and needed more time for what was left of her immune system to catch up to weather the procedure.

View post:
Bubble Boy Disease Cured With Stem Cells

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

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.

Continue reading here:
Delivering stem cells into heart muscle may enhance cardiac repair and reverse injury

Delivery of Stem Cells into Heart Muscle After Heart Attack May Enhance Cardiac Repair and Reverse Injury

Contact Information

Available for logged-in reporters only

Newswise 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 hearts 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.

This study adds to the emerging evidence that a small population of adult stem cells can be recruited to the damaged areas of the heart and improve clinical outcomes, says Dr. Hajjar.

Read more here:
Delivery of Stem Cells into Heart Muscle After Heart Attack May Enhance Cardiac Repair and Reverse Injury

Low back, neck, hip, shoulder, and knee arthritis 7 months after stem cell therapy by Adelson – Video


Low back, neck, hip, shoulder, and knee arthritis 7 months after stem cell therapy by Adelson
Spence describes his outcome from his "full-body make-over" by Harry Adelson, N.D.. Seven months ago, Spence had his own bone marrow stem cells injected into his low back, neck, hips, shoulders,...

By: Harry Adelson, N.D.

Read this article:
Low back, neck, hip, shoulder, and knee arthritis 7 months after stem cell therapy by Adelson - Video

Real-life Ken doll who spent 125K on plastic surgery becomes first in UK to have stem cell hair op

VIEW GALLERY

An air steward who spent 125,000 on plastic surgery to turn himself into a real-life Ken doll has become the first person in the UK to have stem cell hair treatment.

Vain Rodrigo Alves, 30, has undergone 20 cosmetic procedures, including nose jobs, liposuction, six-pack and pec implants, calf shaping and botox fillers.

But he refuses to stop on his quest for perfection and has recently undergone a unique treatment to help cover his emerging bald spots.

The stem cell procedure involved doctors injecting stem cells into his scalp.

It took four hours and now - after just a couple of months - he is seeing the first results.

Rodrigo, who lives in London, said: "I started seeing results after two months, I have lots of baby hair coming through, it's amazing.

"My hair was really thin and my temple was starting to recede but now its coming back in leaps and bounds.

"Seeing my hair begin to go like that was quite shocking. I was considering going to LA to have a transplant until I found these doctors.

"I'm getting so many compliments now.

Read more from the original source:
Real-life Ken doll who spent 125K on plastic surgery becomes first in UK to have stem cell hair op

Establishment of induced pluripotent stem cells from Werner syndrome fibroblasts

19 hours ago

Associate Professor Akira Shimamoto and Professor Hidetoshi Tahara at the Graduate School of Biomedical & Health Science in Hiroshima University, Professor Koutaro Yokote at the Graduate School of Medicine in Chiba University, Visiting Professor Makoto Goto at the Medical Center East in Tokyo Women's Medical University, and collaborators including the staff at the Cancer Chemotherapy Center in the Japanese Foundation for Cancer Research, Tottori University, and Keio University established induced pluripotent stem (iPS) cells from the fibroblasts of Werner Syndrome patients.

These results were published in PLOS ONE in an article entitled "Reprogramming Suppresses Premature Senescence Phenotypes of Werner Syndrome Cells and Maintains Chromosomal Stability over Long-Term Culture."

Werner syndrome is characterized by the premature appearance of features associated with normal aging and cancer predisposition. This syndrome occurs frequently in Japan, affecting 1 in 20,000 to 1 in 40,000 people. The therapeutic methods for this disease are very limited and it is expected that iPS cells can be used for the development of innovative therapies.

Dr. Shimamoto and his collaborators analyzed patient-derived iPS cells and found that telomeric abnormalities in the fibroblasts of these patients, which were caused by the lack of WRN helicase encoded by the gene responsible for Werner syndrome, were recovered in the iPS cells generated from these patients. Furthermore, Dr. Shimamoto found that the expression levels of aging-related genes, including those encoding cell cycle inhibitors and inflammatory cytokines, in the patient-derived iPS cells were the same as those in normal iPS cells, even though the expression levels of these genes in the fibroblasts of the patients were higher than those in normal fibroblasts.

Dr. Shimamoto said, "So far, the use of patient cells was restricted to blood or dermal cells in basic research. The iPS cells that we have established will provide an opportunity for drug discovery for the treatment of Werner syndrome and also help with better understanding of the mechanism of this disease. In addition, the mutated WRN gene in patient-derived iPS cells can be corrected by genome editing. This advantage will be help in the development of new gene and cell therapies for Werner syndrome."

Explore further: Scientists find that SCNT derived cells and IPS cells are similar

Journal reference: PLoS ONE

Provided by Hiroshima University

A team led by New York Stem Cell Foundation (NYSCF) Research Institute scientists conducted a study comparing induced pluripotent stem (iPS) cells and embryonic stem cells created using somatic cell nuclear transfer (SCNT). ...

More here:
Establishment of induced pluripotent stem cells from Werner syndrome fibroblasts

Donor: The German teenager who saved my life

Sue Walters only chance of survival from leukaemiawas a stem cell transplant No one in her family matched her tissue type Doctors searched the worldwide donor register They found Nicola Gerber, a student from Mechern, near the French border

By Chloe Lambert for the Daily Mail

Published: 20:21 EST, 17 November 2014 | Updated: 04:28 EST, 18 November 2014

838 shares

19

View comments

When Sue Walters was diagnosed with leukaemia, she hoped that the best of medical science would be used to cure it.

What she could never have anticipated was that her life would be saved by an 18-year-old boy from a remote German village.

Sues only chance of survival was a stem cell transplant previously known as a bone marrow transplant.

What Nicola has done is amazing it really is a gift of life. If I hadnt had the transplant, it was unlikely Id have lived beyond three months,' said Sue Walters of her donor Nicola Gerber

Excerpt from:
Donor: The German teenager who saved my life

NeoStem (NBS) Stock Plummets Today on Disappointing Cardiac Stem-Cell Therapy Data

NEW YORK (TheStreet) -- Shares ofNeoStem (NBS) plummeted 25.52% to $5.05 in late morning trading Tuesdayafter the biotech company announced poor results from a trial of its proprietary cardiac stem-cell therapy NBS10.

NBS10, which used to be called AMR-001, missed two primary endpoints in the study to test the therapy's efficacy.The stem-cell therapy comesfrom a patient's own bone marrow and is injected into patients after a heart attack. The stem cells are then supposed to help blood flow and build cardiac muscle.

NeoStem's trial used non-invasive imaging to monitor blood flow through the heart six months after a one dose of NBS10 or a placebo. The study showed no difference between NBS and placebo, NeoStem said.

Must Read:NeoStem's Stem Cell Therapy Fails Mid-Stage Heart Attack Study

Jim Cramer and Stephanie Link reveal their investment tactics while giving advanced notice before every trade.

Access the tool that DOMINATES the Russell 2000 and the S&P 500.

Jim Cramer's protg, David Peltier, uncovers low dollar stocks with extraordinary upside potential that are flying under Wall Street's radar.

Link:
NeoStem (NBS) Stock Plummets Today on Disappointing Cardiac Stem-Cell Therapy Data

UCLA Doctors Hail Potential Cure For Bubble Baby Syndrome

CBS Los Angeles (con't)

Affordable Care Act Updates: CBSLA.com/ACA

Health News & Information: CBSLA.com/Health

WESTWOOD (CBSLA.com) Doctors say a groundbreaking stem cell therapy treatment out of UCLA may have cured Bubble Baby syndrome once and for all.

KNX 1070s Brian Ping reports Dr. Donald Kohn has perfected a gene therapy that has now cured 18 children born without an immune system, known as ADA-deficient severe combined immunodeficiency (SCID).

Only weeks after giving birth to fraternal twins in 2012, Alysia and Christian Padilla-Vaccaro found out their daughter Evangelinas immune system was so deficient that she could have no exposure to the outside world.

After enrolling their daughter in Dr. Donald Kohns revolutionary stem cell gene therapy treatment which was nearly three decades in the making doctors extracted stem cells from the bone marrow in Evangelinas hip, then used a modified mouse virus to correct her faulty gene before replacing the marrow.

You hear the words mouse virus and you want to run the other way, said mom Alysia. But they modify it so that its teaching it to do something that they want it to do, which is put something in there that was missing.

Evangelinas new immune system developed without side effects and she is now living a healthy normal life.

Her mother Alysia said while the process was difficult for any mom to go through, it was all worth it.

Read more:
UCLA Doctors Hail Potential Cure For Bubble Baby Syndrome