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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."
CONTACT:
Eileen Campion
Dera, Roslan + Campion Public Relations
212-966-4600 / Eileen@drcpublicrelations.com
Science was trying to take a shortcut trying not to deal with moral issues. There's no elevator to success, you have to take the stairs! Induced pluripotent cells are cool but why spend all this time checking them out when the real need could be solved by using cells from the source. We need brave scientists who believe and are willing to take a chance. We can hope that in the future ways to obtain compatible cells become easier but in the meantime use what you have and make it happen.
"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
"What we are announcing today is just the beginning," Collins said at a Wednesday briefing. Approval of the lines was "open and shut" he said, because they met the exact paperwork requirements of June guidelines for proper informed consent of embryo donors. About 20 more cell lines will be considered Friday under a separate review process for cell lines that meet the "spirit" of the consent guidelines.
"It's very exciting," said George Daley of Children's Hospital Boston, whose lab submitted 11 of the 13 approved lines. The other two lines belong to the lab of Ali Brivanlou at Rockefeller University in New York. NIH will allow researchers whose 31 grants for using human embryonic stem cells had awaited the announcement, to now proceed with the approved lines, affecting $21 million in research funding. "We have been passionately pursuing stem cell research for more than a decade, and it has been a long fight," Daley says.
"We are just seeing today the beginning of potential flood of stem cell lines," says Dartmouth bioethicist Ronald Green.
Human embryonic stem cells are grown from a few hundred inner cells of a five-to-six-day old embryo. They are unspecialized cells that can grow into every type of body tissue. Researchers such as Daley have proposed using the cells to study embryonic development, screen drugs to treat chronic ailments and perhaps someday grow immune-rejection free replacement organs for patients suffering diseases ranging from cancer to diabetes.
In 2001, the Bush administration limited federal funding of human embryonic stem cell research to already existing cell lines, citing ethical concerns over the destruction of embryos. Actual destruction of embryos to collect the cells is still barred from federal funding by a separate law, but if that takes place using private money, the new guidelines allow federally-backed research on stem cells grown from those embryos. Eventually fewer than two dozen lines were approved for research funding under the Bush rules. NIH spent $88 million last year on the research.
In March, President Obama asked NIH to produce new guidelines for stem cell research, opening funding to more recently-grown cells, if they met ethical standards for informed consent by embryo donors. The approved lines derive from embryos freely donated by fertility patients for research. Daley says his 11 were "low-grade" embryos rejected for fertility treatments, and then donated by couples for research. His team published a Nature Biotechnology study last year describing the collection and growth of the cells.
Richard Doerflinger of the U.S. Council of Catholic Bishops, which opposes research on human embryonic stem cells, called the announcement, "a political event, but the science is all moving in the other direction." He suggests recent progress in so-called "induced" stem cells, grown from skin cells but with many of the tissue-growing potential of embryonic cells, has removed the need for the new cell lines. However, Collins and other scientists suggest that the embryonic stem cells are needed for their own potential and to illuminate advances in induced cell line research.
Collins also says NIH hopes to "review, if not necessarily approve" another 96 cell lines, including those up for review Friday, before NIH grant applications come due in March. Daley estimates that 700 to 800 cell lines have been described in the scientific literature since 2001.
Comment: Does anyone else see the moral/ethical dilemma of creating
and destroying 35 viable human embryos to save 1 human? Even the argument that
the two embryonic lines might lead to cures for disease down the road is on
shaky ground due to the fact that embryonic stem cell research has yet to
produce a single successful treatment. On the other hand, adult stem cells
(like those from the umbilical cord blood) don't require destroying human life
and have 70+ successful treatments to their names.
Each of the other 35 embryos should have been
given a chance to be born, a chance to be given a name, a change to be loved.
Not destroyed as if they were simply lab rats. There are organizations that
would have taken those embryos and attempted to implant them in mothers who
couldn't have children of their own and who would have given them a chance at
life. If that one boy's life was worth creating 36 embryos to save his, then
isn't each of those babys' life worth giving them a chance to have one instead
of being sent off for research.
One day will will look back on the 19th & 20th
century's holocaust of destroying viable little humans in the same way we look
back on Hitler's holocaust...with sorrow, shame, and a vow: "Never Again".
Response: Thank you for your comment. Yes, many see the moral/ethical dilemma and we did too.
I'm not a doctor and I'm not a priest, I'm the father of a boy who was born with a hole in his immune system. I live in a practical world of choices and I can speak for families like mine that face life threatening conditions and have to make life changing decisions to find a cure. Our solution relied on finding the genetic roots of the disease and this is what gave us options to cure him.
In our case most of the embryonic cells (blastocyst) had a well-defined genetic mutation inside a gene known as NEMO that causes the immune system to fail; a terminal condition, not compatible with life.
When we learned about the procedure that involved in-vitro fertilization (IVF) and pre-implantation genetic diagnosis (PGD) as an option to find a compatible sibling to use umbilical cord stem cells to cure our son we found out that:
a) not many Latinos are registered as stem cell bone marrow donors.
For Andy's treatment we needed compatible cells
to replace the damaged ones and we faced the moral/ethical dilemma
straightforward. We consulted Andy's doctors and 90% of them thought this
procedure could work. We consulted our family and friends, the majority are Catholic,
some Jewish and some Atheist and all of them had a positive response. We consulted Padre Toño, a Catholic priest who
is a friend of the family. He knew about Andy and what he was going through. He gave us a positive response and also
contacted some of his friends at the
I agree that human embryonic cells are worthy of esteem and respect and shall be handled with dignity and even more than all of the other type of cells inside this planet. I don't believe that human embryonic cells are like a person, if they are, why haven't I seen any microscopes inside a church?
I don't agree with the premise that the single celled zygote should be given the same considerations as living persons. I do not view the embryo as a human being, particularly when it's inside a freezer. For me the 36 embryos were a group of cells that as beautiful as they may look (only through the lens of a microscope) are not a baby.
It's a blastocyst in an early stage of embryonic development before implantation (picture via Wikipedia). And for me the miracle happens when the blastocyst attaches to the wall of the uterus and performs implantation when connections between the mother and the embryo occur and the different cells begin to form, including the umbilical cord. At this stage the embryo cannot survive outside the mother. Science still can't explain that moment during implantation and I don't think it will be able to do so during my lifetime, only God knows what happens.
We decided to donate the embryos that carried the NEMO disease for research because we know the researchers and we're hoping that with those cells they will be able to study the disease and find an easier cure. We felt that it was morally justified to derive benefit from embryos through medical research instead of relegating them to medical waste or to donate them to another couple knowing they carried the disease.
Have you ever had a serious infection? How did you feel? Did it hurt, was it uncomfortable? Multiply those feelings by 1,000 which is the number of days that Andy had to be in the hospital in a constant battle against life threatening infections.
This has to do with disease and disability and nothing else. I think that in our case love and hope gave us the answer. Not all of the embryos had NEMO and not all of them were donated to research. We have been blessed again with the arrival of another baby who is beautiful and healthy, she was born April 15 and we know she doesn't have NEMO.
I don't think that we can say "never again" or ignore this type of medical science because it will change things, it will push the human race forward. If medical science offers the option to treat disease and relief suffering, for me it's deplorable and illicit to block its path, especially when the pain and suffering affects children. Hope is part of the human condition, that thing inside us that insists. In this case as in many more the morally licit solution is to be able to swap healthy cells for damaged ones, to restore health and life and to cure an incurable terminal disease like the one that affected my son. Sick and disabled people deserve the same type of scientific research that cured him. Millions of people with chronic illnesses need hope, belief and desire to the ethical progress of biomedicine.
In an unimposing brick building located in the heart of Chicago's Lincoln Park neighborhood, lives a repository of information that could lead to the cures for Cancer, Parkinson's Disease, Diabetes, spinal cord injury and a host of other diseases.
In early March, when President Obama cleared the way for federally funded stem cell research, he also may have launched Chicago as the world's top supplier of valuable stem cell lines. While many laboratories, genetic and fertility centers store stem cell lines, the difference at Reproductive Genetics Institute (RGI), located at 2825 N. Halsted in Chicago, is sheer volume. This relatively small facility houses over 300 stem cell lines, more than the entire cumulative number of all of the other existing stem cell lines worldwide.
'How can this be?' one asks when looking at the building that would be hard pressed to hold 300 people. The answer exists in the heart and philosophy of RGI Founder and Scientific Director, Dr. Yury Verlinsky, who opened RGI in 1990 and has spent the better part of the last six years creating these scientific 'lifelines'. "Our early discoveries of polar body biopsy and recovery are now the standard practice for Preimplantation Genetic Diagnosis (PGD) which is used to help screen out genetic disease in embryos. We continue to be committed to the research and discovery that will allow us to prevent and ultimately cure disease," says Verlinsky. "Stem Cell research is just getting underway and we hope to be able to add to this exciting technology by our existing stem cell lines, some of which represent healthy models, others which contain disease."
Such was Verlinsky's plan six years ago, when, as a privately funded facility, he was allowed to use discarded and diseased embryos that had been abandoned or donated by their owners, to create what he believes will contain some of the answers to the greatest dilemmas posed to science and medicine. Often published and well regarded in his fields of reproductive genetics, cytogenetics and cytology, Verlinsky holds a Ph.D. and has worked as a Laboratory Director for 45 years. And while the accomplishments of the staff at RGI, made up of molecular geneticists, embryologists and physicians have been considerable, they have been quiet in their pursuits and quieter still in heralding their success.
RGI's groundbreaking work in the field helped Denver parents, Lisa and Jack Nash, conceive a healthy child who could donate bone marrow to an older sibling, Molly, who was dying of Fanconi's Anemia. The Molly Nash case sparked a heated controversy, as religious leaders debated the ethics of the procedure. "Ultimately, while they argued, we saved a young girl's life," says Verlinsky with confidence. Since then, PGD has led to the birth of more than 250 healthy children worldwide who have saved their older siblings from effects of deadly or debilitating diseases.
Poised now to face a similar challenge in the newly re-opened stem cell controversy; Verlinsky's lab is hoping to provide a clearinghouse for universities, clinics and institutions worldwide seeking to purchase existing stem cell lines. "We've done the work, the lines are ready to go and we're excited to collaborate within the community to create more milestones," says Verlinsky. "We can provide a running start to the research."
All from an unlikely, yet solid starting block in the lab 'next door'.
Copyright 2009
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Reproductive Genetics Institute was established in 1990 by scientific director and CEO, Dr. Yury Verlinsky. Dr. Verlinsky, an expert in preimplantation genetic diagnosis (PGD), pioneered the polar body biopsy and removal technology that is widely used during PGD today. With centers worldwide, RGI is recognized as a leading genetics institute for the prevention of genetic disease through PGD, whether the disease is caused by a single gene defect such as cystic fibrosis or chromosomal issues, which includes Down syndrome. With ever advancing techniques, RGI's staff of geneticists, genetic counselors, fertility specialists and embryologists are able to counsel families and screen for numerous genetic diseases including certain cancers, early onset Alzheimer's and other conditions that contain an inherited component.
Reproductive Genetics Institute (RGI) does not provide any general, limited or implied warranties regarding the use of stem cell lines or embryos to create donor matches or genes that are disease free. Although RGI believes that research on stem cell lines will lead to cures for diseases, RGI does not warrant or represent that any of its stem cell lines contain specific cures for such diseases. Further, RGI makes no specific claim to being the largest repository of stem cell lines in the world. The information contained in each press release was accurate at the time of issuance, and RGI assumes no responsibility for updating the information to reflect subsequent developments.
