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

Pope warns against the false sense of compassion in euthanasia

Vatican City

Pope Francis has warned doctors and ethicists on several hot-button social issues, attacking abortion, embryonic stem cell research and euthanasia as playing with life and a sin against God.

In a strongly worded address that marked a departure for a pope who tends to focus more on social justice issues, Francisdenounced what he called the false sense of compassion that was used to promote abortion and those who regarded euthanasia as an act of dignity.

We are living in a time of experimentation with life. But a bad experiment, the pope told members of the Association of Italian Catholic Doctors at the Vatican Saturday.

Francisalso condemned in vitro fertilization, which he said promoted children as a right rather than a gift to welcome, and embryonic stem cell research, which used human beings as guinea pigs to presumably save others.

This is playing with life, he said. Be careful, because this is a sin against the Creator: against God the Creator, who created things this way.

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Francis has spoken out several times against the assisted suicide movement, which he considers to be a symptom of todays throw-away culture that views the sick and elderly as a drain on society.

On Saturday, the pope said it was unlawful to take a life and warned of the dangers posed to the elderly by hidden euthanasia in our culture of waste.

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Pope warns against the false sense of compassion in euthanasia

Rodrigo Alves who has spent 125k on plastic surgery splashes out ANOTHER 5k

Rodrigo Alves, 30, has undergone 20 cosmetic procedures Those include nose jobs, liposuction, six-pack and pec implants Underwent stem cell hair surgery to cover balding patches on head 5,100 procedure saw doctors perform liposuction in his back to extract fat Mixed this with 500ml of his blood and extracted the stem cells The mixture was then injected into his scalp and he loves the results

By Bianca London for MailOnline

Published: 11:14 EST, 17 November 2014 | Updated: 12:20 EST, 17 November 2014

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

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

But that, it seems, is not enough for Mr Alves who recently had his 21st cosmetic treatment - a pioneering form of stem cell surgery that claims to fix baldness.

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Rodrigo Alves who has spent 125k on plastic surgery splashes out ANOTHER 5k

Beyond Batten Disease Foundation and the New York Stem Cell Foundation Chosen as a National Innovator by the Milken …

New York, New York (PRWEB) November 17, 2014

Beyond Batten Disease Foundation (BBDF) and the New York Stem Cell Foundation (NYSCF) have been selected as a national innovator by the Milken Institute and will present their breakthrough findings about juvenile Batten disease at the 6th annual Partnering for Cures, November 16-18 in New York City. The presentation will highlight the collaborative efforts of NYSCF, BBDF and Batten Disease Support and Research Association.

Craig and Charlotte Benson established Beyond Batten Disease Foundation in August 2008 after their then five-year-old daughter, Christiane, was diagnosed with juvenile Batten disease. Together with hundreds of families affected by Batten disease, and many more supporters who share their hope and resolve, they are working tirelessly to create a brighter future for Christiane, and all children with Batten disease.

Watch the Benson Family story:

The Benson Family Story

Beyond Batten Disease and the New York Stem Cell Foundation hope to ramp up funding and partnerships to develop stem cell resources to investigate and explore new treatments and ultimately find a cure for juvenile Batten disease, a fatal illness-affecting children as they convene at the FasterCures, conference. The Washington, D.C.-based center of the Milken Institute will bring together nearly 1,000 medical research leaders, investors and decision-makers to forge the collaborations needed to speed and improve outcomes-driven R&D. NYSCF scientists have created the first iPS cells from a neurological disease and the first ever stem cell disease model from any disease. This discovery was named Time Magazine #1 breakthrough in 2008 because it was the first time anyone has made stem cells from a person with a disease and used them to produce the type of cell that degenerated in that patient. Again, in 2012 Time Magazine recognized the Beyond Batten Disease Foundations creation of a rate genetic disease test as a top ten medical breakthrough.

We know the genetic mutations associated with juvenile Batten disease. This partnership will result in stem cell models of juvenile Batten, giving researchers an unprecedented look at how the disease develops, speeding research towards a cure, said Susan L. Solomon, NYSCF Chief Executive Officer.

Working with NYSCF to generate functional neuronal subtypes from patients and families is a stellar example of one of our key strategies in the fight against juvenile Batten disease: creating resource technology with the potential to transform juvenile Batten disease research and accelerate our timeline to a cure, said Danielle M. Kerkovich, PhD, BBDF Principal Scientist.

Juvenile Batten disease begins in early childhood between the ages of five and ten. Initial symptoms typically begin with progressive vision loss, followed by personality changes, behavioral problems, and slowed learning. These symptoms are followed by a progressive loss of motor functions, eventually resulting in wheelchair use and premature death. Seizures and psychiatric symptoms can develop at any point in the disease.

Juvenile Batten disease is one disorder in a group of rare, fatal, inherited disorders known as Batten disease. Over 40 different errors (mutations) in the CLN3 segment of DNA (gene) have been attributed to juvenile Batten disease. The pathological hallmark of juvenile Batten is a buildup of lipopigment in the bodys tissues. It is not known why lipopigment accumulates or why brain and eventually, heart cells are selectively damaged. It is, however, clear that we need disease-specific tools that reflect human disease in order to figure this out and to build therapy.

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Beyond Batten Disease Foundation and the New York Stem Cell Foundation Chosen as a National Innovator by the Milken ...

Donor: The German teenager who saved my life: How a global network of stem cell donors found an unlikely saviour for a …

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: 20:35 EST, 17 November 2014

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

This would give her a new, stronger immune system to destroy the cancerous cells in her blood.

No one in her family matched her tissue type, so doctors searched the worldwide donor register and found Nicola Gerber, a student from Mechern, near the French border.

He had recently registered as a stem cell donor, and was the perfect match.

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Donor: The German teenager who saved my life: How a global network of stem cell donors found an unlikely saviour for a ...

Veteran Actor Darius McCrary from Family Matters Receives Stem Cell Procedures with Dr. Raj in Beverly Hills

Beverly Hills, California (PRWEB) November 17, 2014

Veteran television and movie actor Darius McCrary has received a revolutionary stem cell procedure for his painful knee and ankle. The regenerative medicine procedure with stem cells was performed by Dr. Raj, a top orthopedic doctor in Beverly Hills and Los Angeles.

Darius McCrary is well known for his decade long stint on Family Matters as character Eddie Winslow. He won a Best Young Actor Award for this role along with movie roles in both Mississippi Burning and Big Shots. Currently, Darius appears along with Charlie Sheen in the show Anger Management.

While staying in tip top shape for his career, Darius has developed persistent pain in his right knee and ankle. Rather than seek a regular cortisone injection for pain relief or opt for surgery, he desired the ability to repair the joint damage and achieve pain relief. "I couldn't imagine being immobilized because of injury, so I opted for a stem cell procedure."

The procedures were performed by Dr. Raj, who is a prominent Beverly Hills orthopedic doctor with extensive experience in regenerative medicine. The procedure consisted of a combination of platelet rich plasma therapy along with amniotic derived stem cell therapy. Anecdotal studies are showing that the stem cell procedures for extremity joints allow patients to achieve pain relief and often avoid the need for potentially risky surgery.

Dr. Raj has performed over 100 stem cell procedures for patients who have degenerative arthritis or sports injuries. "Patients do extremely well with the procedures. Minimal risk and there's a huge potential upside!"

With an active acting career, Darius McCrary cannot afford to be distracted with chronic pain. "I'm looking forward to getting back in the gym and going hard without this pain," he stated excitedly. The procedure was filmed and can be seen on Dr. Raj's Facebook page.

To discuss stem cell procedures at Beverly Hills Orthopedic Institute and how they can benefit, call (310) 247-0466.

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Veteran Actor Darius McCrary from Family Matters Receives Stem Cell Procedures with Dr. Raj in Beverly Hills

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

Stem Cells Treatment Not Useful In Stroke Patients Finds Indian Study

A large-scale trial conducted in India has shown that stem cell therapy does not work in stroke patientsREUTERS

A study conducted on 120 patients in India has shown that stem cell treatment is not effective in treating paralysis resulting from a stroke.

The research which is thefirst large-scale study conducted in Indiacompared outcomes in those treated with stem cells to others and found no difference, reports Down to Earth.

While 60 patients with some form of disability of limbs caused by a stroke were given conventional treatment, an equal number received bone marrow stem cells in addition. All had experienced a stroke 3-4 weeks before the trial.

"We found that at the end of the first month, patients with stem cells showed more improvement compared to the control group. But at the end of the third month and one year, there was no difference," said Kameshwar Prasad, head, Department of Neurology, All India Institute of Medical Sciences (AIIMS), who led the study.

On an average 280 million bone marrow cells were injected, of which blood forming stem cells were around 2.9 million per patient.

The average age of patients in the study was around 50.

The study, published in the current issue of American journal Stroke, was conducted at AIIMS in New Delhi and four other hospitals covering four cities.

The study comes when many others have been suggesting that stem cells could help treat paralysis in stroke patients. The earlier study was done on a small number of patients as compared to the AIIMs study.

More research needs to be done, before stem cells are used in therapy as in India, many private clinics are openly offering stem cell treatment for various diseases.

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Stem Cells Treatment Not Useful In Stroke Patients Finds Indian Study

Reprogramming cells, long term

Harvard Stem Cell Institute (HSCI) researchers, representing five Harvard departments and affiliated institutions as well as the Massachusetts Institute of Technology (MIT), have demonstrated that adult cells, reprogrammed into another cell type in a living animal, can remain functional over a long period.

The work by Joe Zhou, an associate professor in Harvard's Department of Stem Cell and Regenerative Biology, and his collaborators is an important advance in the effort to develop cell-based therapies for tissue repair, and specifically in the effort to develop improved treatment for diabetes.

The researchers used a combination of genes to change pancreatic exocrine cells -- one of the main forms of cells in the pancreas -- in adult mice that have diabetes into insulin-producing beta cells that appeared to cure about a third of the mice of the metabolic disease, and improved insulin production in most of the other mice.

A report on the work was published today in the journal Nature Biotechnology.

The new findings are a major advance in work by HSCI co-director Doug Melton and Zhou, who in 2008 reported having converted exocrine cells into functional beta cells in mice. At that time, however, it was not known how long, and how well, the repurposed cells would function.

"The efficiency of reprogramming has always been an issue," Zhou said. "Until now, the new cells have either dropped dramatically in number or disappeared completely," he said, noting that since his work with Melton in 2008 there have been reports published in other programing systems that question whether the reprogrammed cells could be stable enough ultimately to be useful.

"What we have demonstrated is that yes, the reprogrammed cells can be useful, and for that to happen you have to create a niche environment in which the cells can survive," Zhou continued. "We have improved the reprogramming efficiency to a point where one can create a large enough number of the new cells that the new cells create their own niche environment."

Zhou said that the researchers studied the mice for up to about 13 months, approximately half their normal life span, and found that "the cells are still there, and fairly robust. These are diabetic animals, and we were able to, I wouldn't use the word 'cure' because that's a very freighted word for me to use, but they became highly glycemic animals -- though not every animal became normal. That may be because to completely control the glucose level of the animal, you not only need beta cells, you need about a quarter of a million functional beta cells. If you are short of this number, even if the beta cells are perfectly normal," they can't completely control blood sugar levels, Zhou said.

When discussing the implications of the study for the field of cellular reprogramming, Zhou cautioned that the pancreas has a particularly simple cellular organization and structure, and thus findings in the pancreas might not necessarily apply to other organs.

Diabetes is a metabolic disease that is seen in two basic forms.

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Reprogramming cells, long term

Pope Says Euthanasia Is 'Sin Against God;' Blasts Abortion, Stem-Cell Research

November 16, 2014|8:19 am

Pope Francis prays at the Austro-Hungarian cemetery of Fogliano in Redipuglia, Italy, September 13, 2014. Pope Francis marked the centenary of World War I at the Redipuglia Military Sacrarium with a mass.

Euthanasia does not reflect dignity but is, in fact, a sin against God and creation and a "false sense of compassion," Pope Francis said Saturday, denouncing the right-to-die movement. The pontiff also rejected abortion, in vitro fertilization and embryonic stem cell research.

"We're are living in a time of experimentation with life. But a bad experiment (we're) playing with life," he said, addressing about 4,000 doctors from the Association of Italian Catholic Doctors in the Vatican, according to Catholic News Agency.

"Be careful, because this is a sin against the Creator: against God the Creator," the pope said, adding that the assisted suicide movement can be attributed to a "throw-away culture" that sees the sick and elderly as a burden on society.

It's like telling God, "'At the end of life I do it, like I want.' It's a sin against God. Think well about this."

Pope's remarks come about a fortnight after California woman Brittany Maynard, who had terminal brain cancer, decided to end her own life.

Francis blasted the notion that abortion is good for women or euthanasia is "an act of dignity," or "a scientific breakthrough to 'produce' a child (who is) considered a right instead of accepted as a gift." He also denounced "(the) use of human life as laboratory mice supposedly to save others."

The pope went on to say that medical science appears to have diminished "the ability to 'take care' of the person, especially when they are suffering, fragile and defenseless."

He encouraged medical professionals to take "courageous and against-the-grain" decisions in line with church teaching on the dignity of life. "Your mission as doctors puts you in daily contact with so many forms of suffering," he said.

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Pope Says Euthanasia Is 'Sin Against God;' Blasts Abortion, Stem-Cell Research