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Stem cell researchers use gene therapy to restore immune systems in 'Bubble Boy' disease

By Stem Cell Treatment

ScienceDaily (Sep. 11, 2012) UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years.

In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will be studied in the next phase of the study, which already has enrolled eight of the 10 patients needed.

The study appears Aug. 30 in the advance online issue of the peer-reviewed journal Blood.

"We were very happy that in the human trials we were able to see a benefit in the patients after we modified the protocol," Kohn said. "Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease."

Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases and don't grow well. Chronic diarrhea, ear infections, recurrent pneumonia and profuse oral candidiasis commonly occur in these children. SCID cases occur in about 1 of 100,000 births

Currently, the only treatment for ADA-deficient SCID calls for injecting the patients twice a week with the necessary enzyme, Kohn said, a life-long process that is very expensive and often doesn't return the immune system to optimal levels. These patients also can undergo bone marrow transplants from matched siblings, but matches can be very rare.

About 15 percent of all SCID patients are ADA-deficient. Kohn and his team used a virus delivery system that he had developed in his lab in the 1990s to restore the gene that produces the missing enzyme necessary for a healthy immune system. To date, about 40 children with SCID have received gene therapy in clinical trials around the world, Kohn said.

Two slightly different viral vectors were tested in the study, each modified to deliver healthy ADA genes into the bone marrow cells of the patients so the needed enzyme could be produced and make up for the cells that don't have the gene. Four of the 10 patients in the study remained on their enzyme replacement therapy during the gene therapy study. There were no side effects, but their immune systems were not sufficiently restored, Kohn said.

In the next six patients, the enzyme therapy was stopped and a small dose of chemotherapy was given before starting the gene therapy to deplete the ADA-deficient stem cells in their bone marrow. Of those patients, half had their immune systems restored. The human findings confirmed another study, also published recently in Blood by Kohn and UCLA colleague Dr. Denise Carbonaro-Sarracino, which tested the techniques in parallel, using a mouse model of ADA-deficient SCID.

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Stem cell researchers use gene therapy to restore immune systems in 'Bubble Boy' disease

UCLA stem cell researchers use gene therapy to restore immune systems in 'bubble babies'

By Stem Cell Treatment

Public release date: 11-Sep-2012 [ | E-mail | Share ]

Contact: Kim Irwin kirwin@mednet.ucla.edu 310-435-9457 University of California - Los Angeles Health Sciences

UCLA stem cell researchers have found that a gene therapy regimen can safely restore immune systems to children with so-called "Bubble Boy" disease, a life threatening condition that if left untreated can be fatal within one to two years.

In the 11-year study, researchers were able to test two therapy regimens for 10 children with ADA-deficient severe combined immunodeficiency (SCID). During the study, they refined their approach to include a light dose of chemotherapy to help remove many of the blood stem cells in the bone marrow that are not creating an enzyme called adenosine deaminase (ADA), which is critical for the production and survival of healthy white blood cells, said study senior Dr. Donald Kohn, a professor of pediatrics and of microbiology, immunology, and molecular genetics in Life Sciences and a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA.

The refined gene therapy and chemotherapy regimen proved superior to the other method tested in the study, restoring immune function to three of the six children who received it, Kohn said. Going forward, an even further refined regimen using a different type of virus delivery system will be studied in the next phase of the study, which already has enrolled eight of the 10 patients needed.

The study appears Aug. 30 in the advance online issue of the peer-reviewed journal Blood.

"We were very happy that in the human trials we were able to see a benefit in the patients after we modified the protocol," Kohn said. "Doctors treating ADA-deficient SCID have had too few options for too long, and we hope this will provide them with an efficient and effective treatment for this devastating disease."

Children born with SCID, an inherited immunodeficiency, are generally diagnosed at about six months. They are extremely vulnerable to infectious diseases and don't grow well. Chronic diarrhea, ear infections, recurrent pneumonia and profuse oral candidiasis commonly occur in these children. SCID cases occur in about 1 of 100,000 births

Currently, the only treatment for ADA-deficient SCID calls for injecting the patients twice a week with the necessary enzyme, Kohn said, a life-long process that is very expensive and often doesn't return the immune system to optimal levels. These patients also can undergo bone marrow transplants from matched siblings, but matches can be very rare.

About 15 percent of all SCID patients are ADA-deficient. Kohn and his team used a virus delivery system that he had developed in his lab in the 1990s to restore the gene that produces the missing enzyme necessary for a healthy immune system. To date, about 40 children with SCID have received gene therapy in clinical trials around the world, Kohn said.

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UCLA stem cell researchers use gene therapy to restore immune systems in 'bubble babies'

GMA tweets thanks to docs for allowing stem cell treatment

By Stem Cell Treatment

By: Lira Dalangin-Fernandez, InterAksyon.com September 11, 2012 7:21 PM

InterAksyon.com The online news portal of TV5

MANILA, Philippines - Former President and now Pampanga Representative Gloria Macapagal-Arroyo said Tuesday she was thankful to her doctors at the Veterans Memorial Medical Center for recognizing her right to seek stem cell treatment for her medical condition.

In her Twitter account (@gmarroyo), Arroyo said, "I'm glad my Veterans attending physicians recognize my right to seek alternative treatment and don't consider it in conflict with the conventional treatment they're giving me."

Arroyo on Monday underwent her fourth stem cell intravenous treatment while still attending her weekly therapy at the VMMC.

"Procedure was not only intravenous injection of the stem cell material but also other naturalist treatment therapies," she said in her Twitter account.

"It has to be done under very hygienic conditions. Then I proceeded to my 3x (three times) weekly physical therapy at Veterans," she added.

Earlier, she said her treatment would involve cultured stem cells, which would be "much more modest in price that the one coming from sheep or one's own body."

Stem cell therapy involves the use of fresh cells injected into the body to regenerate cells to reverse aging or treat illnesses.

Arroyo has been complaining of choking sensation because of the "aggressive protrusion" of the titanium plate that was earlier implanted on her in a surgery. Her physician, Dr. Roberto Anastacio, said that the protrusion is slowly blocking her air and food passages.

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GMA tweets thanks to docs for allowing stem cell treatment

New Procedure Saves Dogs Lives

By Stem Cell Clinic

GREEN BAY, WI--A breakthrough in veterinary medicine In Green Bay. Two dogs recover amazingly well after receiving stem cell transplants at Packerland Veterinary Center two months ago. They are the first vet clinic in Wisconsin to perform the procedure.

It's a story that gives hope to pet owners all over the country. Stem cells are taken out of the dog's fatty tissue, harvested, then injected into problem areas leaving the dogs completely healed.

"We couldn't take him on walks. He just laid around a lot," said German Shepherd, DeNiro's owner, Keith Noskowiak.

"We'd hear whimpering overnight. She'd take a few steps and she would sit down," said Luther Kortbein, Shadow's owner, another German Shepherd.

But now the dogs have a whole new life. Until two months ago. DeNiro suffered from severe arthritis.

Shadow. suffered from hip dysplasia. The owners were at their wits end. DeNiro's thought he may even have to put his beloved German Shepherd down.

"We felt we had a decision to make with his quality of life and being in pain we didn't want him to be in pain," said Noskowiak.

Shadow's owner was willing to try anything to cure her.

"Whatever the cost needed to get this done we were willing to do," said Kortbein.

Then Packerland Veterinary Center offered them stem cell therapy. The dog's own stem cells are extracted, then injected back into the bloodstream joints.

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New Procedure Saves Dogs Lives

Stem Cell Therapy Treats Pets

By Stem Cell Treatment

Green Bay -

You hear about people getting stem cell therapy for various ailments. Now it's even available for your pet.

A Northeast Wisconsin veterinary center is the first in the state to offer it. It's not cheap, but we found pet owners feel the results outweigh the cost.

Eleven-year-old DeNiro is about to do something he couldn't do when he was ten. Jump.

Owner Keith Noskowiak couldn't be happier. "That's awesome seeing him be able to do that again."

DeNiro suffered from severe arthritis in his back.

A German shepherd named Shadow had hip dysplasia. She could barely walk, let alone jump as she can today, catching balls in flight.

"She would whimper all night long because she was starting to hurt. It was starting to get more severe," owner Luther Kortbein says.

These two German shepherds were the first dogs to receive stem cell therapy in Wisconsin.

Doctor Maura Mansfield from the Packerland Veterinary Center performed the surgeries in June.

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Stem Cell Therapy Treats Pets

New stem cell treatment for dogs

By Stem Cell Treatment

GREEN BAY - Statistics show Americans spend almost $51 billion on their pets every year.

A new stem cell treatment for dogs is providing relief for pets and their owners, and now it's being done here in the Green Bay area.

It's designed to treat problems like hip dysplasia and arthritis.

The procedure can cost thousands of dollars, but pet owners believe it is money well-spent.

"Come on buddy," said Keith Moskowiak, De Niro's owner.

Ten-year-old German Shepherd De Niro is at the veterinarian's office for a routine check up.

Owner Keith Moskowiak says De Niro couldn't have done it just two months ago.

"He wasn't able to get around, you could tell he was in a lot of pain," said Moskowiak.

Five-year-old Shadow has hip dysplasia.

"She was slowing herself down, she truly was. She was reserving herself. She'd run a little bit, then she'd sit," said Luther Kortbein, Shadow's owner.

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New stem cell treatment for dogs

University of Maryland study: Neonatal heart stem cells may help mend kids' broken hearts

By Stem Cell Medical Center

Public release date: 10-Sep-2012 [ | E-mail | Share ]

Contact: Bill Seiler bseiler@umm.edu 410-328-8919 University of Maryland Medical Center

Baltimore, MD September 10, 2012 Researchers at the University of Maryland School of Medicine, who are exploring novel ways to treat serious heart problems in children, have conducted the first direct comparison of the regenerative abilities of neonatal and adult-derived human cardiac stem cells. Among their findings: cardiac stem cells (CSCs) from newborns have a three-fold ability to restore heart function to nearly normal levels compared with adult CSCs. Further, in animal models of heart attack, hearts treated with neonatal stem cells pumped stronger than those given adult cells. The study is published in the September 11, 2012, issue of Circulation.

"The surprising finding is that the cells from neonates are extremely regenerative and perform better than adult stem cells," says the study's senor author, Sunjay Kaushal, M.D., Ph.D., associate professor of surgery at the University of Maryland School of Medicine and director, pediatric cardiac surgery at the University of Maryland Medical Center. "We are extremely excited and hopeful that this new cell-based therapy can play an important role in the treatment of children with congenital heart disease, many of whom don't have other options."

Dr. Kaushal envisions cellular therapy as either a stand-alone therapy for children with heart failure or an adjunct to medical and surgical treatments. While surgery can provide structural relief for some patients with congenital heart disease and medicine can boost heart function up to two percent, he says cellular therapy may improve heart function even more dramatically. "We're looking at this type of therapy to improve heart function in children by 10, 12, or 15 percent. This will be a quantum leap in heart function improvement."

Heart failure in children, as in adults, has been on the rise in the past decade and the prognosis for patients hospitalized with heart failure remains poor. In contrast to adults, Dr. Kaushal says heart failure in children is typically the result of a constellation of problems: reduced cardiac blood flow; weakening and enlargement of the heart; and various congenital malformations. Recent research has shown that several types of cardiac stem cells can help the heart repair itself, essentially reversing the theory that a broken heart cannot be mended.

Stem cells are unspecialized cells that can become tissue- or organ-specific cells with a particular function. In a process called differentiation, cardiac stem cells may develop into rhythmically contracting muscle cells, smooth muscle cells or endothelial cells. Stem cells in the heart may also secrete growth factors conducive to forming heart muscle and keeping the muscle from dying.

To conduct the study, researchers obtained a small amount of heart tissue during normal cardiac surgery from 43 neonates and 13 adults. The cells were expanded in a growth medium yielding millions of cells. The researchers developed a consistent way to isolate and grow neonatal stem cells from as little as 20 milligrams of heart tissue. Adult and neonate stem cell activity was observed both in the laboratory and in animal models. In addition, the animal models were compared to controls that were not given the stem cells.

Dr. Kaushal says it is not clear why the neonatal stem cells performed so well. One explanation hinges on sheer numbers: there are many more stem cells in a baby's heart than in the adult heart. Another explanation: neonate-derived cells release more growth factors that trigger blood vessel development and/or preservation than adult cells.

"This research provides an important link in our quest to understand how stem cells function and how they can best be applied to cure disease and correct medical deficiencies," says E. Albert Reece, M.D., Ph.D., M.B.A., vice president for medical affairs, University of Maryland; the John Z. and Akiko K. Bowers Distinguished Professor; and dean, University of Maryland School of Medicine. "Sometimes simple science is the best science. In this case, a basic, comparative study has revealed in stark terms the powerful regenerative qualities of neonatal cardiac stem cells, heretofore unknown."

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University of Maryland study: Neonatal heart stem cells may help mend kids' broken hearts

State stem cell research funding agency awards $37.3 million to aid UC Irvine efforts

By Stem Cell Clinic

Public release date: 6-Sep-2012 [ | E-mail | Share ]

Contact: Tom Vasich tmvasich@uci.edu 949-824-6455 University of California - Irvine

Irvine, Calif., Sept. 6, 2012 Efforts to begin human clinical trials using stem cells to treat Alzheimer's disease and retinitis pigmentosa received a $37.3 million boost from the California Institute for Regenerative Medicine during its most recent round of funding on Sept. 5.

UC Irvine scientists will be part of two research teams garnering CIRM Disease Team Therapy Development Awards, which are designed to accelerate collaborative translational research leading to human clinical trials. In one, Dr. Henry Klassen, an associate professor of ophthalmology in UC Irvine's Sue & Bill Gross Stem Cell Research Center, and his collaborators at UC Santa Barbara and Cedars-Sinai Medical Center, received $17.3 million to cultivate therapeutically potent retinal progenitor stem cells to treat the blinding effects of retinitis pigmentosa.

In the other, StemCells, Inc. in Newark, Calif., received $20 million and will collaborate with Frank LaFerla and Mathew Blurton-Jones neurobiologists with the stem cell research center and the Institute for Memory Impairments and Neurological Disorders (UCI MIND) to advance research using the company's proprietary purified human neural stem cells to improve memory in people with Alzheimer's disease.

"CIRM's support for UC Irvine's efforts to advance stem cell-based treatments for a variety of diseases is extremely gratifying," said Peter Donovan, director of the Sue & Bill Gross Stem Cell Research Center. "Henry's work on retinitis pigmentosa and Frank and Mathew's on Alzheimer's disease hold great promise, and we are delighted that they have the support to see their work move toward the clinic."

Klassen's objective is to introduce stem cells that rescue and reactivate damaged and dying photoreceptor rods and cones, thus reversing the course of RP even at relatively advanced stages. The current CIRM funding will allow Klassen and his collaborators to grow these cells under conditions ensuring that pharmaceutical standards are met. The resulting cells will be tested in animals for safety and to make certain that they are therapeutically potent. Then the team will seek FDA approval for the use of these cells in early clinical trials, in which a small number of patients with severe RP will be injected with cells in their worse-seeing eye and followed clinically for a specified period of time to determine the safety and effectiveness of the treatment.

"We believe it's possible to rejuvenate a clinically significant number of cones in the degenerating retina," said Klassen, whose work also has received long-standing support from the Discovery Eye Foundation. "Our methods have been validated, and I'm optimistic that stem cell-based treatments can help restore fading vision in people with eye diseases."

The CIRM award will further LaFerla and Blurton-Jones's efforts with StemCells, Inc. to understand how human neural stem cells can treat Alzheimer's disease, the leading cause of dementia in the U.S. Earlier this year, the researchers reported findings showing that neural stem cells restored memory and enhanced synaptic function in two animal models relevant to Alzheimer's disease, possibly by providing growth factors that protect neurons from degeneration. With these studies establishing proof of concept, the team intends to conduct further animal studies necessary to seek FDA approval to start testing this therapeutic approach in human patients.

"Our goal is to research ways to make memories last a lifetime, and we're excited to investigate the potential efficacy of stem cells for Alzheimer's disease," said LaFerla, the UCI MIND director and Chancellor's Professor and chair of neurobiology & behavior.

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State stem cell research funding agency awards $37.3 million to aid UC Irvine efforts

Could this stem cell cure for wrinkles end the endless hunt for the perfect skin cream?

By Stem Cell Clinic

British firm is trialling new method by injecting patient's own stem cells to restore skin's youthful elasticity

By Tamara Cohen

PUBLISHED: 10:40 EST, 9 September 2012 | UPDATED: 02:12 EST, 10 September 2012

Scientists will begin clinical trials in 12 months, using stem cells harvested from a blood sample from the patients

Scientists are working on a new weapon in the war against wrinkles.

There are not many things women have not tried in the quest for a youthful complexion from lotions and potions to Botox and cosmetic surgery.

But a British firm is trialling a new method which involves injecting the patients own stem cells to restore skins youthful elasticity.

Researchers believe they will spur the growth of new skin cells, called fibroblasts, which make the elastic ingredient collagen which is produced in large quantities when we are young, but declines as we age.

The company Pharmacells, based in Glasgow, plan to begin clinical trials in 12 months, using stem cells harvested from a blood sample from the patients.

They believe the procedure could be commercially available in just three years, potentially revolutionising the market for anti-ageing treatments.

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Could this stem cell cure for wrinkles end the endless hunt for the perfect skin cream?

Researchers Reveal a Chemotherapy-Resistant Cancer Stem Cell as the "Achilles' Heel" of Cancer

By Uncategorized

Newswise Scientists at Mount Sinai School of Medicine have discovered a subpopulation of cells that display cancer stem cell properties and resistance to chemotherapy, and participate in tumor progression. This breakthrough could lead to the development of new tests for early cancer diagnosis, prognostic tests, and innovative therapeutic strategies, as reported in Cancer Cell.

Resistance to chemotherapy is a frequent and devastating phenomenon that occurs in cancer patients during certain treatments. Unfortunately, tumors that initially respond to chemotherapy eventually become resistant to it, contributing to tumor progression and death. The study reveals that these new cancer stem cells, which have not been differentiated into more specific cell types, are capable of multiplying despite being exposed to chemotherapy, while differentiated cells die.

Led by Carlos Cordon-Cardo, MD, PhD, Chair of Pathology, and Josep Domingo-Domenech, MD, PhD, Assistant Professor of Pathology at Mount Sinai, the research team generated cellular models of drug resistance by treating prostate tumor cell lines with increasing doses of the common chemotherapy drugs, including docetaxel. They identified a cell population expressing markers of embryonic development. In addition, these cells displayed cancer stem cell functions, including the capacity to initiate tumor cell growth. Next, the team evaluated human tissue samples of prostate cancer and found that patients with more aggressive or metastatic tumors had more of these cancer stem cells.

This is the first time these so-called cancer stem cells of prostate have been identified as the basis for drug resistance and tumor progression, indicating that they are cancers Achilles Heel, said Dr. Cordon-Cardo. These findings are the culmination of more than six years of innovative research, which has led to the successful unveiling of cancer characteristics that are critical to understanding how the disease works and progresses.

The study also defines a new therapeutic strategy for patients with prostate cancer, consisting of a combination of standard chemotherapy and two pharmacological agents that inhibit key signaling pathways associated with embryonic development and cell differentiation. Results showed that chemotherapy eliminated differentiated tumor cells, whereas the signaling pathway inhibitors selectively depleted the cancer stem cell population. Some of these inhibitors are already in clinical trials, and some are FDA-approved.

By targeting these newly identified cancer stem cells, we are attacking the foundation of tumor growth, rather than treating the symptoms of it, said Dr. Domingo-Domenech. The novel discovery of this cell population could lead to the development of new tests for early cancer diagnosis, prognostic tests, and innovative therapeutic strategies.

Ongoing studies suggest that this new cell type exist in other tumor types such as breast cancer, colon cancer, bladder cancer and lung cancer. Dr. Cordon-Cardos team is studying these disease areas to determine the presence and impact of these cancer cells.

About The Mount Sinai Medical Center The Mount Sinai Medical Center encompasses both The Mount Sinai Hospital and Mount Sinai School of Medicine. Established in 1968, Mount Sinai School of Medicine is one of the leading medical schools in the United States. The Medical School is noted for innovation in education, biomedical research, clinical care delivery, and local and global community service. It has more than 3,400 faculty in 32 departments and 14 research institutes, and ranks among the top 20 medical schools both in National Institutes of Health (NIH) funding and by US News and World Report.

The Mount Sinai Hospital, founded in 1852, is a 1,171-bed tertiary- and quaternary-care teaching facility and one of the nations oldest, largest and most-respected voluntary hospitals. In 2011, US News and World Report ranked The Mount Sinai Hospital 16th on its elite Honor Roll of the nations top hospitals based on reputation, safety, and other patient-care factors. Mount Sinai is one of 12 integrated academic medical centers whose medical school ranks among the top 20 in NIH funding and US News and World Report and whose hospital is on the US News and World Report Honor Roll. Nearly 60,000 people were treated at Mount Sinai as inpatients last year, and approximately 560,000 outpatient visits took place. For more information, visit http://www.mountsinai.org/.

Find Mount Sinai on: Facebook: http://www.facebook.com/mountsinainyc Twitter: @mountsinainyc YouTube: http://www.youtube.com/mountsinainy

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Researchers Reveal a Chemotherapy-Resistant Cancer Stem Cell as the "Achilles' Heel" of Cancer

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