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A YEAR SINCE OBAMA'S LIFTING OF BAN ON STEM CELL FUNDING, BOSTON TEAM READY TO DISTRIBUTE LINES WORLDWIDE BUT OBSTACLES REMAIN. NEW WEBSITE TO END PUBLIC CONFUSION AND PUSH SCIENCE FORWARD
Researchers at Children's Hospital Boston Aim to Demystify Science and Share Potential for Treatments and Cures on Range of Diseases from Diabetes to Parkinson's
StemCell.ChildrensHospital.org
MARCH 9, 2010 [Boston] - Exactly one year to the day after President Obama's historic decision to reverse an eight-year restriction on federally funded embryonic stem cell research, the Children's Hospital Boston Stem Cell Program today announced the development of a unique, comprehensive web site and released a candid video message from its top researchers on the current state of stem cell research to introduce the new site:stemcell.childrenshospital.org.
Designed as an engaging and accessible resource, the new site seeks to demystify the science, clear up misperceptions and illuminate the public about the power and value of different types of stem cells to create cost effective treatments and potential cures for a range of diseases--including Type I diabetes, Parkinson's, leukemia and other blood cancers, Huntington's, sickle cell anemia, neurological disorders, and more.
Beginning today, visitors to stemcell.childrenshospital.org can access an introductory video, featuring Drs. Leonard Zon and George Daley, along with Stem Cell Task Force leader President of the Kraft group and the New England Patriots, Jonathan Kraft, --who together cite the promise and ongoing challenges in this field and call for new support. Visitors can also sign up to receive updates leading up to the official launch of the website--which will be fully operational on April 26, 2010.
The timing of the April 26th date is also significant in the field of stem cell research, because it marks the 5th anniversary of the release of guidelines from the US National Academy of Sciences (NAS) for the responsible conduct of embryonic stem cell research. Children's was among the first to adopt those guidelines, and in the five years since the guidelines were released, the Stem Cell Program at Children's Hospital has emerged as an internationally recognized leader in stem cell research.
Despite operating under significant constraints over the past eight years, Children's made remarkable progress on multiple fronts:
- Children's created an aggressive business plan, their roadmap for bringing research from the bench to the bedside.
- Children's produced 11 of the first 13 hESC lines approved by the US National Institutes of Health (NIH) for federal funding in December, 2009. Children's is receiving requests for these lines from scientists across the U.S. who are studying diseases from diabetes to Parkinson's.
- Drs. Daley and Zon have been awarded major grants from the NIH for the creation of human models of disease and to study whether stem cells reprogrammed from adult stem cells (iPS) cells are equivalent to those derived from human embryos, one of the field's most pressing questions.
- Dr. Zon's research has already been rapidly translated from the laboratory to the clinic: studies on blood formation in the zebrafish--a model organism pioneered by the Zon lab--led to the discovery of a drug that expands blood stem cells, which is now being tested in patients undergoing umbilical cord blood transplants.
- Children's Researchers were the first to isolate lung stem cells, with implications for treating lung cancer, cystic fibrosis and pulmonary diseases.
- Science Magazine cited Dr. Daley's creation of disease-specific stem cells from patients in its 2008 "Breakthrough of the Year" issue.
"Stem cell research is an incredibly exciting field and this is an extraordinary time to be part of it," said Leonard Zon, MD, Director of Children's Hospital's Stem Cell Program. "In just the five short years since the release of the NAS guidelines, we have seen major break-throughs in the science and in the techniques we can use to investigate potential therapies for a wide range of diseases. It's absolutely essential that we continue that forward momentum."
The Human Face of Stem Cell Research
Now, the stem cell program at Children's Hospital Boston, the principal pediatric teaching hospital for Harvard Medical School, invites the public to become part of the dialogue and the progress. The new web site will clarify misconceptions about stem cell research and provide a broad range of easily accessible information about the history and science of stem cells, current research and issues, a primer on the ethical debates surrounding stem cell research, and much more. Special areas on the site will be devoted to some of the specific diseases mentioned above--and will show individuals how they can help. The newsroom portion will feature the latest breakthroughs in the field, and will also enable visitors to opt in to receive the latest updates via Twitter and Facebook.
The site will also give people a chance to experience the human face of stem cell therapy through inspiring videos and interviews with families and patients who have been treated by stem cell transplants or have donated their own cell lines to research in the hope of one day helping to bring about breakthroughs and cures:
- A teenaged sickle cell patient and gifted student who spent his summer helping out by working in Dr. George Daley's stem cell lab.
- An avid cyclist paralyzed in an accident five years ago, whose own tissues have been made into stem cells by Children's researchers, who has organized major support for new studies that could one day benefit patients with spinal cord injuries.
- A family whose decision to conceive a baby daughter using IVF enabled life-saving stem cells from her umbilical cord to save the life of her infant brother, who was born with a fatal genetic defect.
"Over the past year, improvements in governmental policy and breakthroughs in the lab have advanced the field of stem cell research at a more rapid pace the than ever before, but the public dialogue suggests that people want to learn more," said George Daley, MD, PhD, Director of Stem Cell Transplantation at Children's Hospital Boston. "It's critical for people to have a clear understanding of the science, and to appreciate the promise and the limitations of this research. I think this website will help us achieve that."
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Yury gave us a dicount to do the PGD lab setup for NEMO in his Lab.
Jackson Merriman is the son of Doug and Melissa Merriman. He was born NEMO. The only hope for a cure for the immune deficiency is a bone marrow transplant which Jackson will be undergoing in the spring in Denver, Colorado. The family has turned to Children's Organ Transplant Association(COTA) to help raise funds. Not only will there be medical expenses, but travel and living expenses as well. A tax-deductible donation can be given to the family by visiting their website. Thank you for your help.http://cota.donorpages.com/
"I have concluded that we should allow federal funds to be used for research on these existing stem cell lines, where the life-and-death decision has already been made,"
In 2009 Obama said,
"With the executive order I am about to sign, we will bring the change that so many scientists and researchers, doctors and innovators, patients and loved ones have hoped for and fought for these past eight years. We will lift the ban on federal funding for promising embryonic stem cell research."
Therapy status?
"I think that's still a ways off," said Len Zon from Children's.
Glad we didn't wait!
Link to article via NPR with audio.
Nice image:
Image Courtesy The Johns Hopkins University Genetics and Public Policy Center
Erica Henderson, 75, has been allowed to transfer her cells to Paul Hallowes despite being too old under medical guidelines.
Mr Hallowes, 69, had been diagnosed with leukaemia and a high dose chemotherapy had severely impaired his body's ability to produce blood cells.
(Ally Donnelly, NECN: Boston, MA) - A Boston hospital has received a grant to establish the world's first-ever center to study orphan diseases. An orphan disease is any disease or disorder affecting less than 200,000 people in the United States.
Because the disorders affect so few, researchers have a difficult time getting government or private funding. But as one Massachusetts family knows -- research can make the difference...between life and death
Andy Trevino just wants to play air hockey. Being dragged back to this hospital playroom and having a camera stuck in his face is not making things easier.
It's understandable that he's a little shy. The 8-year-old Sudbury, Massachusetts boy has had all the extra attention he needs in his young, tumultuous life.
Paulina, Mother: Two days after he was born, he got a fever and I thought, well, give him some Tylenol or something and that's it.
Unfortunately that would not be it. Andy -- born in Mexico City -- had a blood infection and would stay in the hospital's ICU for more than two weeks. When his parents finally brought their son home, they were not out of the woods.
Andres, Father: First it was a stomach infection then a central nervous system infection also known as meningitis, then he had pneumonia...
His infections were life threatening. Mexican doctors ran every test they could think of... But again and again they came up empty.
Paulina: It hurts a lot to see nurses and people poking at him all the time and watching him crying...
Andres: They were telling us that he had bad luck and we didn't take that for an answer.
The Trevino's had spent 375 days in the hospital in Mexico. Their money was dwindling, but not their hope. They had heard that Boston was the best -- so they came here, to Children's Hospital -- desperate for a medical miracle.
The moment we stepped into the ER, it felt safe.
The Trevino's met with a team of doctors and researchers at the renowned teaching hospital, which immediately went to work. The team soon discovered that Andy had a rare congenital disease. A glitch on one of his genes -- known as Nemo -- wasn't allowing his immune system to battle infection.
Alan Beggs, Children's Hospital: Because they had seen a large number of patients with an immune deficiency, they were able to predict what his defect might be and identify it for his family.
The family had few options. They could make Andy comfortable with medication, knowing his disease was terminal. They could get a transplant to replace his immune system or they could try and replace Andy's faulty cells with healthy cells from a new donor.
Unfortunately, they couldn't find a match trough the public donor registry for an immune system transplant and to replace Andy's unhealthy cells, they'd need a perfect donor -- like a sibling -- and Andy was an only child.
Andres: She was born March 14, 2004.
Though they knew some people would question their ethics, the Trevino's --- who had always planned on more children -- had Andy's sister -- and savior --- Sofia.
Andres: The first sound I heard when she cried were just the most incredible sound I've ever heard.
Children's doctors were able to take Sofia's bone marrow cells and infuse them into Andy's. Her healthy cells replaced his bad cells and his immune system rebuilt itself.
Andres: To see that he's cured, it's just...incredible.
Andy had what is known as an orphan disease -- a disease that affects less than 200-thousand people in the United States.
Beggs: In fact there may be only a few hundred patients or only a few 10s of patients with any particular disease, so it takes a center that sees a number of these things to bring these cases together.
Alan Beggs runs the new Manton center for orphan disease research. Established with a recent 25 million dollar gift to the hospital, it creates a dream team of researchers to work collaboratively.
The hope is that findings in the lab can translate into treatments in the field. Treatments not only for orphan diseases -- which are historically under funded, but a wide spectrum of conditions from cancer to Parkinson's. Take for example the stem cell researcher who has made significant advances in his own field.
He can create cells that could regenerate muscle for some of my patients or maybe regenerate nerves for some of the neurology patients and so on.
When the Trevino's came to children's in 2001, there was no Manton center, but they say their support will never waiver.
Andres: once they learn about it, they take it to the clinic and once they take it to the clinic they help more children and at the end of the day they save more lives.
More lives....like Andy's. He is now a thriving third grader who loves nothing more than playing with his best friend....his sister.
Please continue to pray for Justin. That he will not find GVHD, and no more pain.
Justin Cilia is an 8 year old boy. Who underwent transplant on June 22nd. He has been sick since he was six weeks old. He has had a few very serious infections. He has been in and out of the hospital on numerous occasions. He was diagnosed in 2002 with an immune deficiency. He started gammaglobulin infusions a.k.a. IVIG in October of 2002. He has received IVIG monthly at CPMC. It was not until about two years ago that Justin's condition continued to get worse. He was diagnosed with auto-immune hemolytic anemia. He was given Rituxan to treat the disease. This was after much reseach with other Dr. The next year he developed a micro-bacteria infection that but him in the hospital for a few months. He had treatment of antibiotics for over 6 months. That is when we decided to contact Dr. Bleesing at Childrens' Hospital. After we met with Dr. Bleesing in June 2006. He did a lot of additional testing on Justin. He also contacted the NIH in Baltimore. That is when Justin was Diagnosed with NEMO. The only cure for NEMO is a Bone Marrow transplant.What place do "saviour siblings" have in paediatric transplantation: establishing the role of pre-implantation genetic diagnosis with HLA typing
Background: Not all children in need of a haematopoietic stem cell transplant have a suitable relative or unrelated donor available. Recently, in vitro fertilization (IVF) with pre-implantation genetic diagnosis (PGD) for human leukocyte antigen (HLA) tissue typing has been used to selectively transfer an IVF embryo in order to produce a child who may provide umbilical cord blood for transplantation to an ill sibling. Such children are sometimes called "saviour siblings".
Objective: To examine the published clinical and epidemiological evidence relevant to the use of this technology, with the aim of clarifying those situations where IVF and PGD for HLA-typing should be discussed with parents of an ill child.
Design: A critical analysis of published literature on: comparative studies of umbilical cord blood versus other sources of stem cells for transplantation; comparative studies of matched unrelated donor versus matched related donor transplantation; and the likelihood of finding an unrelated stem cell donor.
Conclusion: IVF and PGD for HLA-typing is only applicable when transplantation is non-urgent and parents are of reproductive age. Discussions regarding this technology may be appropriate where no suitable related or unrelated donor is available for a child requiring a transplant, or where no suitable related donor is available and transplantation is only likely to be entertained with a matched sibling donor. Discussion may also be considered in the management of any child lacking a matched related donor who requires a non-urgent transplant or may require a transplant in the future.
