Stem Cell Clinic

4 strategies doctors are using to cure the blind

By Stem Cell Doctors

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

Local clinic treats animals with stem cell therapy

By Uncategorized

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

Great Ormond Street deaths caused by stem cell lab failures, inquest told

By Stem Cell Treatment

Katie Joyce, left, aged four, and Sophie Ryan Palmer, aged 12, were among the four children who died as a result of complications with transplants. Photograph: Steve Parsons/PA

Four children have died after failings in how stem cells used in life-saving operations were frozen at Great Ormond Street hospital, it emerged this week.

The four, who were between one and 12 years old, were among eight children with cancer whose bone marrow transplants did not work as a result of problems with the freezing process.

Britains best-known childrens hospital has admitted that one of them, four-year-old Katie Joyce, might have survived if it had acted more quickly when problems arose.

An inquest into the deaths this week heard that doctors were initially baffled as to why a decade of success using the procedures suddenly came to a halt in summer 2013. Despite extensive investigations, the hospital failed to pinpoint the source of the setbacks in its cryopreservation laboratory, used for freezing stem cells which were kept there for using in bone marrow transplants in children.

The transplanted stem cells were intended to help the childs bone marrow, damaged during chemotherapy, grow again to maximise the chance of recovery.

At the inquest, lawyers for two of the families whose children died accused Great Ormond Street of taking too long to halt the transplants once staff began having concerns.

The hospital has since overhauled its procedures to prevent further incidents and there are calls for the deaths to lead to tighter procedures around how stem cells are stored at hospitals and research centres across the UK.

Concerns were first raised in June 2013 when 12-year-old Sophie Ryan Palmer, who had acute lymphoblastic leukaemia, failed to make progress after her transplant at Great Ormond Street, which involved using a donors stem cells rather than her own.

By October 2013 the hospital had identified that a higher than usual proportion of eight patients who had undergone stem cell transplantation between March and August had suffered setbacks after encountering what doctors call delayed engraftment. It immediately stopped freezing stem cells on site at its base in Bloomsbury, central London, and launched an investigation.

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Great Ormond Street deaths caused by stem cell lab failures, inquest told

Mount Sinai Researchers Awarded $1 Million Grant to Find New Stem Cell Therapies for Vision Recovery

By Stem Cell Medicine

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Newswise NEW YORK November 20, 2014 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 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 patients 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 patients 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, patients 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 patients own stem cells and restore vision.

Other investigators from Mount Sinai include Ihor Lemischka, PhD, Director, Black Family Stem Cell Institute and J. Mario Wolosin, PhD, Professor of Ophthalmology. The research is supported by NEI grant EY023997.

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Mount Sinai Researchers Awarded $1 Million Grant to Find New Stem Cell Therapies for Vision Recovery

Signaling molecule crucial to stem cell reprogramming

By Uncategorized

PUBLIC RELEASE DATE:

20-Nov-2014

Contact: Scott LaFee slafee@ucsd.edu 619-543-5232 University of California - San Diego @UCSanDiego

While investigating a rare genetic disorder, researchers at the University of California, San Diego School of Medicine have discovered that a ubiquitous signaling molecule is crucial to cellular reprogramming, a finding with significant implications for stem cell-based regenerative medicine, wound repair therapies and potential cancer treatments.

The findings are published in the Nov. 20 online issue of Cell Reports.

Karl Willert, PhD, assistant professor in the Department of Cellular and Molecular Medicine, and colleagues were attempting to use induced pluripotent stem cells (iPSC) to create a "disease-in-a-dish" model for focal dermal hypoplasia (FDH), a rare inherited disorder caused by mutations in a gene called PORCN. Study co-authors V. Reid Sutton and Ignatia Van den Veyver at Baylor College of Medicine had published the observation that PORCN mutations underlie FDH in humans in 2007.

FDH is characterized by skin abnormalities such as streaks of very thin skin or different shades, clusters of visible veins and wartlike growths. Many individuals with FDH also suffer from hand and foot abnormalities and distinct facial features. The condition is also known as Goltz syndrome after Robert Goltz, who first described it in the 1960s. Goltz spent the last portion of his career as a professor at UC San Diego School of Medicine. He retired in 2004 and passed away earlier this year.

To their surprise, Willert and colleagues discovered that attempts to reprogram FDH fibroblasts or skin cells with the requisite PORCN mutation into iPSCs failed using standard methods, but succeeded when they added WNT proteins - a family of highly conserved signaling molecules that regulate cell-to-cell interactions during embryogenesis.

"WNT signaling is ubiquitous," said Willert. "Every cell expresses one or more WNT genes and every cell is able to receive WNT signals. Individual cells in a dish can grow and divide without WNT, but in an organism, WNT is critical for cell-cell communication so that cells distinguish themselves from neighbors and thus generate distinct tissues, organs and body parts."

WNT signaling is also critical in limb regeneration (in some organisms) and tissue repair.

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Signaling molecule crucial to stem cell reprogramming

Pluripotent cells created by nuclear transfer can prompt immune reaction, researchers find

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PUBLIC RELEASE DATE:

20-Nov-2014

Contact: Krista Conger kristac@stanford.edu 650-725-5371 Stanford University Medical Center @sumedicine

Mouse cells and tissues created through nuclear transfer can be rejected by the body because of a previously unknown immune response to the cell's mitochondria, according to a study in mice by researchers at the Stanford University School of Medicine and colleagues in Germany, England and at MIT.

The findings reveal a likely, but surmountable, hurdle if such therapies are ever used in humans, the researchers said.

Stem cell therapies hold vast potential for repairing organs and treating disease. The greatest hope rests on the potential of pluripotent stem cells, which can become nearly any kind of cell in the body. One method of creating pluripotent stem cells is called somatic cell nuclear transfer, and involves taking the nucleus of an adult cell and injecting it into an egg cell from which the nucleus has been removed.

The promise of the SCNT method is that the nucleus of a patient's skin cell, for example, could be used to create pluripotent cells that might be able to repair a part of that patient's body. "One attraction of SCNT has always been that the genetic identity of the new pluripotent cell would be the same as the patient's, since the transplanted nucleus carries the patient's DNA," said cardiothoracic surgeon Sonja Schrepfer, MD, PhD, a co-senior author of the study, which will be published online Nov. 20 in Cell Stem Cell.

"The hope has been that this would eliminate the problem of the patient's immune system attacking the pluripotent cells as foreign tissue, which is a problem with most organs and tissues when they are transplanted from one patient to another," added Schrepfer, who is a visiting scholar at Stanford's Cardiovascular Institute. She is also a Heisenberg Professor of the German Research Foundation at the University Heart Center in Hamburg, and at the German Center for Cardiovascular Research.

Possibility of rejection

A dozen years ago, when Irving Weissman, MD, professor of pathology and of developmental biology at Stanford, headed a National Academy of Sciences panel on stem cells, he raised the possibility that the immune system of a patient who received SCNT-derived cells might still react against the cells' mitochondria, which act as the energy factories for the cell and have their own DNA. This reaction could occur because cells created through SCNT contain mitochondria from the egg donor and not from the patient, and therefore could still look like foreign tissue to the recipient's immune system, said Weissman, the other co-senior author of the paper. Weissman is the Virginia and D.K. Ludwig Professor for Clinical Investigation in Cancer Research and the director of the Stanford Institute for Stem Cell Biology and Regenerative Medicine.

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Pluripotent cells created by nuclear transfer can prompt immune reaction, researchers find

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

By Stem Cell Treatment

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.

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

Bubble Boy Disease Cured With Stem Cells

By Stem Cell Doctors

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.

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Bubble Boy Disease Cured With Stem Cells

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