New Stem Cell Treatment Found To Cure 'Bubble Baby' Disease

Chuck Bednar for redOrbit.com Your Universe Online

A new stem cell gene therapy developed by researchers at UCLA is set to begin clinical trials early next year after the technique reportedly cured 18 children who were born without working immune systems due to a condition known as ADA-deficient Severe Combined Immunodeficiency (SCID) or Bubble Baby disease.

The treatment was developed by Dr. Donald Kohn, a member of the UCLA Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research, and his colleagues, and according to the university, it is able to identify and correct faulty genes by using the DNA of the youngsters born with this life-threatening condition.

Left untreated, ADA-deficient SCID is often fatal within the first year of a childs life, reports Peter M. Bracke for UCLA. However, after more than three decades of research, Dr. Kohns team managed to develop a gene therapy that can safely restore the immune systems of children with the disease by using their own cells and with no noticeable side effects.

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, Dr. Kohn, who is also a professor of pediatrics and of microbiology, immunology and molecular genetics, said in a recent statement.

Children born with SCID have to be isolated in a controlled environment for their own safety, because without an immune system, they are extremely vulnerable to illnesses and infections that could be deadly. While there are other treatments for ADA-deficient SCID, Dr. Kohn noted that they are not always optimal or feasible for many children. The new technique, however, provides them with a cure, and the chance to live a full healthy life.

SCID is an inherited immunodeficiency that is typically diagnosed about six months after birth, the researchers said, and children with the condition are so vulnerable to infectious diseases that even the common cold could prove fatal to them. This particular form of the condition causes cells to not create ADA, an enzyme essential for the production of the white blood cells which are a vital component of a healthy, normally-functioning immune system.

Approximately 15 percent of all SCID patients are ADA-deficient, according to the university, and these youngsters are typically treated by being injected twice per week with the required enzyme. This is a process that must continue throughout a patients entire life, and even then it doesnt always work to bring their immune systems to optimal levels. Alternately, they could undergo bone marrow transplants from matched siblings, but those matches are rare and the transplanted cells themselves are often rejected by the childs body.

Dr. Kohn and his colleagues tested two therapy regimens on 18 ADA-deficient SCID over the course of two multi-year clinical trials starting in 2009. During the trials, the blood stem cells of the patients were removed from their bone marrow and genetically modified in order to correct the defect. All 18 of the patients were cured.

The technique used a virus delivery system first developed in Dr. Kohns laboratory in the 1990s a technique which inserts the corrected gene that produces the ADA into the blood forming stem cells in the bone marrow. The genetically corrected blood-forming stem cells will then produce the T-cells required to combat infections.

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New Stem Cell Treatment Found To Cure 'Bubble Baby' Disease

Stem cell trial to begin for children suffering from cerebral palsy

Nov. 23, 2014, 3:05 p.m.

It is hoped that a trial due to start next year, and involving about 20 Australian children with cerebral palsy, will show the benefits of using stem cells from their own umbilical cord blood to treat the condition.

About 20 Australian children with cerebral palsy will be infused with their own umbilical cord blood in a trial due to start next year, as physicians warn families against travelling overseas for experimental stem cell treatments.

The long-awaited Australian trial will provide some of the world's first evidence about the safety and effectiveness of using stem cells from umbilical cord blood to repair brain injury that leads to cerebral palsy.

Researchers are waiting on ethics approval for the trial which will provide treatment to families who have chosen to store their child's cord blood at private banks.

In some cases, children with cerebral palsy will be able to receive a sibling's cord blood if this is available.

Cerebral Palsy Alliance head of research Iona Novak said the study, led by the Murdoch Childrens Research Institute, will recruit children from around Australia who have access to privately banked cord blood.

Children aged one to 10 will receive infusions at private blood banks in Melbourne, Sydney and Brisbane, and will be assessed before and after the treatment to check for improvements.

Researchers will be unable to access cord blood from a public bank, which collects blood to treat blood disorders such as leukaemia and cannot be used for untested new therapies.

Associate Professor Novak said the trial was an important first step towards establishing whether stem cells could help repair the brain injury that leads to cerebral palsy, a series of disabilities associated with movement and posture.

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Stem cell trial to begin for children suffering from cerebral palsy

MS treatment first for Inverness mum

Published: 22/11/2014 08:00 - Updated: 21/11/2014 17:04

Written byVal Sweeney

Lucy Clarke hopes pioneering MS treatment will enable her enjoy walks in the park with son, Theo (4) and husband, Dan Jenkins.

A mother with multiple sclerosis is hoping to become the first Scottish patient to undergo cutting edge stem-cell treatment in a Russian clinic.

Lucy Clarke, of Inverness, hopes to raise 40,000 via crowd-funding for the procedure which involves transplanting her own stem cells into her body.

The 38-year-old was first diagnosed with MS, a disease of the central nervous system, nine years ago.

With her condition now worsening, she has opted for treatment unavailable in the UK in a bid to halt the diseases progress and to enjoy family activities such as walking in the park.

In April, the acupuncturist will travel to Moscows Pirogov Center where just 25 patients a year receive Hematopoietic Stem Cell Transplantation (HSCT).

To help fund her one-month stay and post-treatment rehabilitation, her family have set up a campaign website. They are selling Lucys Light candles and her mother is also planning a fundraising skydive to coincide with her 70th birthday.

Any surplus money will be used to set up a charity to support others with MS.

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MS treatment first for Inverness mum

Four children dead at Great Ormond Street after stem cell transplant failure

Patients, aged one to 12, among eight children whose transplants failed Concerns arose in 2013 after operation on fundraiser Sophie Palmer, 12 Hospital says Katie Joyce, 4, could have been saved if quicker action taken Lawyers have also accused hospital of taking too long to stop transplants Doctors 'regret' not stopping sooner but decision seemed right at time Ryan Loughran, 13 months, and Muhanna al-Hayany, 4, also died last year Seventeen months on, investigations are still ongoing into exact cause

By Steph Cockroft for MailOnline

Published: 06:45 EST, 22 November 2014 | Updated: 12:57 EST, 22 November 2014

Four cancer-stricken children died at Great Ormond Street Hospital after a series of failures in stem cell transplants at the world-renowned hospital, an inquest has heard.

The young patients, aged between one and 12, were among eight children whose transplants failed when the stem cell freezing system - used in life-saving operations - inexplicably stopped working.

Four children went on to recover. But well-known charity fundraiser Sophie Ryan Palmer, 12, one-year-old Ryan Loughran, four-year-old Katie Joyce and Muhanna al-Hayany, also four, died between July and October last year.

Katie Joyce (left) and Sophie Ryan (right) were among two of the four young patients who died after a series of failures in stem cell transplants at Great Ormond Street Hospital

The children's hospital has now admitted that Katie might have survived if it had acted more quickly to resolve the problems.

Lawyers for two of the families have also accused Great Ormond Street of taking too long to stop the transplants once concerns arose.

At an inquest into the deaths this week, the court heard that doctors were initially dumbfounded as to why the procedures suddenly started failing after a decade of success, the Guardian reports.

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Four children dead at Great Ormond Street after stem cell transplant failure

Ashley, Athetoid Cerebral Palsy | Stem Cell Treatment Testimonial – Video

Ashley, Athetoid Cerebral Palsy | Stem Cell Treatment Testimonial
Even at 5 years old, Ashley only had the physical capabilities of a 6 month old. Until he was able to receive his first stem cell treatments, Ashley had no w...

By: Beike Biotech

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Ashley, Athetoid Cerebral Palsy | Stem Cell Treatment Testimonial - Video

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

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 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.

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

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

PUBLIC RELEASE DATE:

17-Nov-2014

Contact: Sally Stewart sally.stewart@cshs.org 310-248-6566 Cedars-Sinai Medical Center @cedarssinai

LOS ANGELES (NOV. 17, 2014) - 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.

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

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

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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

UW professor using stem cell research to find treatment for eye conditions

Through stem cell research, two Madison men who suffer from a genetic condition that causes blindness and hearing loss are receiving experimental treatment.

Johnny and Mike Walsh, sons of University of Wisconsin Regent David Walsh who suffer from Usher disease are receiving experimental treatmentfrom David Gamm, a UW professor and expert in retinal and stem biology.

Johnny has had hearing problems from birth and is now legally blind. However, he maintains a positive attitude due to the experimental treatment he is receiving in Madison and does not let his disability get in the way of his career as an attorney at Axley Brynelson LLP.

It is interesting that here I am in Madison getting a diagnosis and then having such a great research institution there in my backyard and its convenient for them to have my family there because you got four kids, three who carry the genes, two who are affected and one who isnt. So you have a perfect control right there, I think thats kind of neat for Dr. [David] Gamm to have and he takes our blood whenever he needs it, Walsh said.

Mike started a project entitled Flight4Sight, where he travels the world to spread awareness and understanding about blindness. His project is on Facebook and a blog, where he takes his followers advice on where to travel next.

Mike said he benefits from the research Gamm, of the Waisman Center,does.

Gammis working closely with Cellular Dynamics International, a Madison-based company which has recently received a $1.2 million grant to continue their research on human stem cells.

CDI specializes in creating human cells, including various types of stem cells. With the National Eye Institutes funding, CDI will carry out this stem cell-based research in the first study of its kind to be performed in the United States.

Eye conditions that include dry age-related macular degeneration affect as many as 11 million Americans who have some form of macular degeneration, according to the CDI statementregarding the grant. The cells being used for this study are CDI developed and manufactured through induced pluripotent stem cells, which will potentially have application in discovering treatments for retinal and eye conditions.

The goal of the study will be to find out how to reprogram stem cells in order to make retina cells to prevent further damage in decaying eyes and eye conditions, Gamm said.

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UW professor using stem cell research to find treatment for eye conditions