Less than a year after a Wisconsin team helped discover a major alternative to human embryonic stem cells, the Madison scientists say more than 800 labs have begun using the approach, suggesting that many stem-cell researchers are starting to move beyond controversial embryonic sources for their work.
Such shifts may reframe the emotionally fraught debate over stem cells--an issue that has ignited passions across the political spectrum. Both presidential candidates have indicated they would lift President George W. Bush's restrictions on research funding, though Sen.Barack Obama has been more adamant than Sen. John McCain.
The biologist doing more than anyone else to stir the debate is University of Wisconsinresearcher James Thomson, who co-discovered human embryonic stem cells a decade ago, in November 1998. Last year Thomson shook the field again when his lab and a Japanese team showed a way of genetically reprogramming adult skin cells to act like stem cells, including the ability to form any of the body's tissues.
Some groups that oppose destroying embryos for research have hailed the new cells, called induced pluripotent stem cells, as a way to eliminate the need for stem cell research based on embryonic material. In a rare extended interview, Thomson said he shares some of their qualms but believes the issue's moral and scientific contours have never been more complex.
October 2008 Archives
Life saving research from Orangelab was published at the Journal of Allergy and Clinical Immunology this month.
They studied 72 individuals with NEMO.
Thirty-six percent of individuals died at a mean age of 6.4 years.
Thirty-two different NEMO mutations were identified.
86% with serious pyogenic infection, 39% with mycobacterial infection, 19% with serious viral infection, and 23% with inflammatory diseases.
Here's the abstract:
Hypomorphic nuclear factor-κB essential modulator mutation database and reconstitution system identifies phenotypic and immunologic diversity
Eric P. Hanson, MDa, Linda Monaco-Shawver, BAb, Laura A. Solt, BSc, Lisa A. Madge, PhDc, Pinaki P. Banerjee, PhDb, Michael J. May, PhDc, Jordan S. Orange, MD, PhDb
Received 30 June 2008; received in revised form 18 August 2008; accepted 20 August 2008. published online 13 October 2008.
Corrected Proof
Background
Human hypomorphic nuclear factor-κB essential modulator (NEMO) mutations cause diverse clinical and immunologic phenotypes, but understanding of their scope and mechanistic links to immune function and genotype is incomplete.
Objective
We created and analyzed a database of hypomorphic NEMO mutations to determine the spectrum of phenotypes and their associated genotypes and sought to establish a standardized NEMO reconstitution system to obtain mechanistic insights.
Methods
Phenotypes of 72 individuals with NEMO mutations were compiled. NEMO L153R and C417R were investigated further in a reconstitution system. TNF-α or Toll-like receptor (TLR)-5 signals were evaluated for nuclear factor-κB activation, programmed cell death, and A20 gene expression.
Results
Thirty-two different mutations were identified; 53% affect the zinc finger domain. Seventy-seven percent were associated with ectodermal dysplasia, 86% with serious pyogenic infection, 39% with mycobacterial infection, 19% with serious viral infection, and 23% with inflammatory diseases. Thirty-six percent of individuals died at a mean age of 6.4 years. CD40, IL-1, TNF-α, TLR, and T-cell receptor signals were impaired in 15 of 16 (94%), 6 of 7 (86%), 9 of 11 (82%), 9 of 14 (64%), and 7 of 18 (39%), respectively. Hypomorphism-reconstituted NEMO-deficient cells demonstrated partial restoration of NEMO functions. Although both L153R and C417R impaired TLR and TNF-α-induced NF-κB activation, L153R also increased TNF-α-induced programmed cell death with decreased A20 expression.
Conclusion
Distinct NEMO hypomorphs define specific disease and genetic characteristics. A reconstitution system can identify attributes of hypomorphisms independent of an individual's genetic background. Apoptosis susceptibility in L153R reconstituted cells defines a specific phenotype of this mutation that likely contributes to the excessive inflammation with which it is clinically associated.
Key words: NEMO, immunodeficiency, genetic database, Jurkat reconstitution, NF-κB activation, A20
Abbreviations used: 7-AAD, 7-Amino actinomycin D, DC, Dendritic cell, EDA, Ectodermal dysplasia and anhidrosis, EMSA, Electrophoretic mobility shift assay, FACS, Fluorescence-activated cell sorting, GFP, Green fluorescent protein, IKK, IκB kinase, NEMO, Nuclear factor-κB essential modulator, NF-κB, Nuclear factor-κB, pNEMO, Parental nuclear factor-κB essential modulator, rNEMO, Wild-type reconstituted NEMO(-), TCR, T-cell receptor
A baby who was grown in order to donate umbilical cord blood to his brother was born last week in Spain, and it was widely reported in Spanish media. They are calling him "baby medicine".
I've seen 400+ media coverage articles about the story.
I also received this report regarding Bishops from Spain from hat seems to be a catholic news site.
I also received this report regarding Bishops from Spain from hat seems to be a catholic news site.
One of the news articles says (Spanish - sorry - lazy to translate - it's Friday):
El Vaticano ya ha lanzado una voz de alarma sobre el avance científico advirtiendo de que -los beneficios fáciles y la arrogancia de sustituir al Creador pueden poner en peligro a la humanidad."
Rapid translation : I'm a danger to humanity.
"cuando tiene como fin el hombre y su bienestar y el progreso de la humanidad - y precisó que - no es el árbol de la ciencia el que mata, sino la desobediencia"
Rapid translation: I'm disobedient.
Rapid translation: I'm disobedient.
"la ciencia - no está capacitada- para establecer principios éticos. -Ella sólo puede aceptarlos en sí y reconocerlos como necesarios para erradicar sus eventuales patologías. La filosofía y la teología sirven en ese momento como ayuda indispensable para evitar que la ciencia proceda en solitario por un camino tortuoso, lleno de imprevistos y de riesgos-
Rapid translation: none
And..
"El nacimiento de una persona humana ha venido acompañada de la destrucción de otras, sus propios hermanos, a los que se les ha privado del derecho fundamental a la vida"
Rapid translation: Too strong. Makes me mad.
Rapid translation: Too strong. Makes me mad.
"Un acto egoísta"
Rapid translation: I'm selfish?
Rapid translation: I'm selfish?
Specially disturbing, they said, is parents choosing a specific embryo because the child's tissues or organs might save the life of a sick sibling.
OOPS.
The Vatican isn't much for trying to save sick children's lives with embryonic stem cell research or PGD because they both mess with embryos and an embryo deserves the same respect as any adult. This is an old argument.
The best response is a perhaps over-used hypothetical scenario:
A building is burning down. Twelve small children are inside screaming for help. The building also contains a freezer storing a dozen or so frozen embryos. Which do you save first?
Allen Goldberg and Laurie Strongin are PGD pioneers and I've been following Allen's blog for years.
Hope for Henry Video from Allen Goldberg on Vimeo.
Hope for Henry Video from Allen Goldberg on Vimeo.
This video shows the important work of the Hope for Henry Foundation, a national organization headquartered in Washington, DC that focuses on improving the quality of life for children facing serious illness and prolonged hospitalization. To help make more smiles possible for kids who need them the most, please make a donation online at
opeforhenry.org/help/donate.html
opeforhenry.org/help/donate.html
Spanish Bishops Condemn Creation of "Savior Sibling," Hand-picked for Optimal Genetic Material
By Kathleen Gilbert
MADRID, Spain, October 22, 2008 (LifeSiteNews.com) - A baby who was grown in order to donate umbilical cord blood to his ailing brother was born last week, an event that was widely reported in Spanish media. The renewed publicity given to the case has sparked criticism from the pro-life community and the Church who, while welcoming the birth of a newborn child, condemned the utilitarian mindset that surrounded the circumstances of his conception and birth.
Baby Javier Mariscal is the first "medicinal baby" to be born in Spain, carried to term in order to donate stem cells that may cure his six-year-old brother Andres, who has severe but non-fatal congenital anaemia. Javier was selected from among six embryos through a technique known as stem-cell selection, which determined that he did not inherit the condition, and could therefore grow stem cells in his umbilical cord that may help Andres. The remaining embryos were either frozen or killed and discarded.
In a statement published last weekend, the Spanish bishops blamed this controversial "savior sibling" method for depriving the remaining embryos of "the fundamental right to life" simply because they would not be able to help Andres. While some hailed Javier's birth as the next step in scientific progress, the bishops warned that it "reduced the dignity of the individual to a simple utilitarian value."
The bishops noted that the secular media has emphasized the good news of the child's birth, and remarked: "Put like this, the news ought to be a source of joy for all. However, the dramatic fact that the sick embryos, and eventually the healthy ones that were not genetically compatible were destroyed has been silenced.
"The Church wants to lend her voice to the voiceless and to those who have been deprived of the fundamental right to life," said the bishops, adding that their statements were not intended to judge, but to "draw attention to the objective ethical principles that protect the dignity of all human beings."
The Spanish pro-life community also reacted with dismay at the case. Manuel Cruz, director of the Life Foundation, told the Times Online, "The method of this birth is degrading for human beings to have been selected like a prize."
The Vatican issued a statement in L'Osservatore Romano following the child's birth, saying it was unjustifiable that parents had "sacrificed other possible children" in order to try curing Andres. About 80% of Spain's population is Catholic.
The case is the first of its kind after the Spanish parliament passed legislation in 2006 allowing the genetic selection of embryos.
The popularity of genetic screening to produce a "designer baby" like Javier is on the rise. However, many bioethicists consider screening for reasons of preference to undermine the intrinsic quality of human life, as it weighs the value of each baby's life according to his or her accidental traits.
Link
Girl in urgent need of bone marrow is saved by the sister born to help her live
By Alison Smith Squire and Nick McDermott
Last updated at 11:58 AM on 20th October 2008
Playing in the autumn leaves, Saskia Graham and her little sister Imogen look like any other happy, healthy youngsters.
But only 18 months ago Saskia was desperately ill with a debilitating blood disorder, in urgent need of a bone marrow transplant to save her life.
Fortunately, there was a ready-made donor - Imogen had been conceived as a 'saviour sibling' through the controversial process in which children are created so their tissue can be used to help a sick brother or sister.
For the girls' parents, however, the decision to use Imogen's bone marrow was still an agonising one. She was only 19 months old and would struggle to survive such an invasive procedure.
To their delight, the transplant worked - curing the blood disorder and creating what will be a lifelong bond between seven-year-old Saskia and Imogen, now three.
Their mother Natalie Barb, 38, said: 'The decision to operate was the most heart-wrenching one we have ever had to make.
'On the one hand, Saskia was growing sicker by the day and desperately needed a bone marrow transplant.
'On the other, the thought of tiny Imogen undergoing surgery was incredibly frightening.'
When Saskia was 11 weeks old she was diagnosed with Diamond Blackfan Anaemia, which is usually fatal before the age of 30. To lead a normal life, she would need a bone marrow transplant from a suitable donor before she turned six, when her condition would worsen.
Doctors told her mother and her father Stuart Graham, a 36-year-old banker, that their best chance was to find a match from a family member.
However, neither the couple, from West London, nor their second child, five-year-old Alice, were suitable.
So they made the controversial decision to create a saviour sibling - a baby genetically selected to be a perfect donor for a sick brother or sister.
Imogen was conceived by IVF using Pre-Implantation Genetic Diagnosis, a screening procedure which ensured she was an ideal match for Saskia.
'We were delighted when Imogen was born. Not only was she a beautiful little sister but she would be a suitable donor for Saskia,' said her mother, a TV producer. 'Her birth gave us the ray of hope we had longed for.' Even then, the decision to use her bone marrow was hard. 'Our fear was that something would go wrong and we would lose not only Saskia but Imogen as well.'
Doctors waited until Imogen was 19 months old and the surgery proved a success.
There has been huge controversy over saviour siblings. But Saskia's mother said: 'We will never regret ensuring that Imogen was a suitable donor. It is just an incredible bonus for us that medical science allowed us to ensure we could also save Saskia's life.'
Saskia is one of many children aged two to 18 treated by the specialist bone marrow transplant centre at St Mary's Hospital in London, part of Imperial College Healthcare NHS Trust, which this week celebrates its 18th anniversary.
Link
From Justin's Mom:
Please continue to pray for Justin. That he will not find GVHD, and no more pain.
Please continue to pray for Justin. That he will not find GVHD, and no more pain.
Who is Justin?
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.
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.Nace en Andalucía el primer bebé libre de una enfermedad genética y compatible con su hermano, gravemente enfermo
La sangre de su cordón umbilical de Javier, el recién nacido, servirá para realizar el trasplante que necesita Andrés para superar una anemia congénita severa
El Hospital Virgen del Rocío de Sevilla ha acogido el nacimiento del primer bebé libre de una enfermedad genética hereditaria y que es compatible al cien por ciento con su hermano, de seis años de edad y enfermo de una grave dolencia, gracias al diagnóstico genético preimplantatorio. Se trata del primer procedimiento de estas características realizado íntegramente en España y va a posibilitar la curación de una persona, cuya única alternativa consiste en un transplante de médula ósea.
Andalucía fue la primera Comunidad Autónoma en incorporar el Diagnóstico Genético Preimplantatorio en la cartera de servicios del Sistema Sanitario Público Andaluz y desde la regulación de este procedimiento han nacido en la sanidad pública andaluza ocho pequeños libre de la enfermedad que les iba a transmitir sus padres. Javier, que se llama el recién nacido, es el noveno y supone un paso más en la técnica puesto que posibilita la curación de su hermano al ser inmunológicamente compatible.
La aprobación en 2006 de la Ley de Reproducción Humana Asistida abría paso a esta nueva opción terapéutica. El Sistema Sanitario Público Andaluz se ha puesto a la vanguardia al conseguir el nacimiento de Javier, el primer bebé de estas características cuyo tratamiento ha sido realizado en España. El alumbramiento de Javier tuvo lugar el pasado día 12 de octubre a las 11.35 horas. El niño ha pesado al nace 3,400 kilos.
El hospital sevillano es referente en Andalucía para la realización del diagnóstico genético preimplantatorio. Esta técnica, que se realiza en su Unidad de Genética y Reproducción Humana Asistida -dirigida por el doctor Guillermo Antiñolo-, consiste en la realización de un análisis genético a preembriones obtenidos por técnicas de fecundación in vitro antes de ser transferidos al útero de la madre, lo que posibilita seleccionar aquellos libres de la carga genética asociada a determinadas enfermedades.
En el caso de Javier, además de haberse evitado la transmisión de la enfermedad de la que son portadores sus padres y que padece su hermano, una anemia congénita severa (beta-Talasemia major), se ha conseguido que los dos hermanos sean perfectamente compatibles puesto que tienen idéntico perfil de histocompatiblidad (HLA), con lo que Javier es el donante idóneo para posibilitar la curación de Andrés mediante trasplante de cordón.
La sangre del cordón umbilical de Javier ha quedado almacenada en el Banco de Cordón Umbilical de Málaga, donde permanecerá hasta que el equipo médico programe el momento del trasplante de progenitores hematopoyéticos. La tasa de éxito de este procedimiento alcanza el 90% por lo que en unos meses, Andrés podrá hacer vida normal, dejando atrás la patología que padece. El pequeño, hasta ahora, no había respondido a ninguno de los tratamientos a los que se había sometido con lo que la donación de la sangre del cordón umbilical de Javier es la única opción terapéutica para el niño.
Diagnóstico genético preimplantatorio
El diagnóstico genético preimplantatorio es la técnica de reproducción asistida que permite evitar la transmisión de enfermedades genéticas hereditarias de padres a hijos. La Unidad de Gestión Clínica de Genética, Reproducción y Medicina Fetal de Hospitales Universitarios Virgen del Rocío es el servicio de referencia en Andalucía para la aplicación de esta técnica, en la que desarrollan su labor genetistas, embriólogos y ginecólogos.
El programa de Diagnostico Genético Preimplantatorio comenzó a funcionar en la comunidad en octubre de 2005, y desde entonces se han iniciado un total de 89 ciclos en 44 parejas. Se ha podido transferir al menos 1 embrión no afecto en 53 ciclos y se han conseguido 13 gestaciones clínicas, siendo la tasa de éxito del programa comparable a la de los mejores centros internacionales.
Desde que en julio de 2006 naciese en Andalucía el primer bebé libre de una enfermedad genética hereditaria en la sanidad pública, se han contabilizado un total de ocho recién nacidos mediante diagnóstico genético preimplantatorio, dos de ellos libres de distrofia muscular de Duchenne, 2 libres de hemofilia, 2 libres de fibrosis quística (parto gemelar) y 2 libres de enfermedad Huntington, a los que se suma el nacimiento de Javier, libre de beta-Talasemia major. Un segundo caso de diagnóstico genético preimplantatorio para que sea compatible con su hermano (DGP-HLA) está pendiente de la aprobación de la Comisión Nacional de Reproducción Humana Asistida.
El programa de Diagnóstico Genético Preimplantatorio, como el del Consejo Genético, se incluyen en el Plan Andaluz de Genética, una herramienta de trabajo que permitirá avanzar en las oportunidades que brinda el genoma humano en la prevención de enfermedades, su diagnóstico precoz e, incluso, el diseño de tratamientos a medida.
ANEXO
¿Qué es el Diagnóstico Genético Preimplantatorio y cómo se realiza?
El Diagnóstico Genético Preimplantatorio es un procedimiento que consiste en realizar un análisis genético a embriones obtenidos por técnicas de fecundación in vitro para transferir al útero únicamente aquellos libres de la enfermedad genética en estudio. En la actualidad constituye una opción reproductiva para familias con alto riesgo de transmitir enfermedades de base genética a sus hijos, siendo una buena alternativa al diagnóstico prenatal.
Además, el Diagnótico Genético Preimplantatorio abre nuevas vías en la prevención de enfermedades genéticas que carecen de tratamiento, al mismo tiempo que ofrece la posibilidad de seleccionar preembriones para que, en determinados casos y bajo el debido control y autorización, puedan servir para tratar la enfermedad del hermano enfermo.
La complejidad de esta técnica exige la colaboración estrecha de especialistas en Genética Médica y Reproducción Asistida. Hay que efectuar una biopsia del preembrión cuando éste tiene de 6 a 8 células. Las células extraídas se procesan para su análisis genético, que ha de ser rápido y preciso, pues los preembriones se deben transferir al útero en un tiempo limitado (dos días desde la biopsia). El análisis genético, mediante FISH y/o estudio molecular, indica qué preembriones pueden ser transferidos, ya que no desarrollarán la enfermedad para la que existe riesgo.
La técnica FISH y el análisis genético molecular
La hibridación in situ fluorescente (FISH) se emplea para prevenir la transmisión de enfermedades hereditarias graves ligadas al cromosoma X (hemofilia, distrofia muscular de Duchenne o síndrome de Alpont) mediante la selección de sexo. Consiste en marcar los cromosomas con sondas de ADN (ácido desoxirribonucleico) fluorescentes específicas para los cromosomas que se están estudiando. A continuación, con el microscopio de fluorescencia, se pueden identificar los cromosomas de interés.
El análisis genético molecular directo o indirecto del gen que produce la enfermedad mediante la reacción en cadena de la polimerasa (PCR) es útil para trastornos hereditarios graves con una mutación conocida (atrofia muscular espinal, fibrosis quística, corea de Huntington).
Consiste en la amplificación de secuencias específicas de un gen, en las que la presencia de una mutación desencadena una enfermedad de base genética. Permite diferenciar qué embriones no tienen un gen mutado y, por tanto, no desarrollarán la enfermedad. Es precisamente el análisis PCR el que permite en el Diagnóstico Genético Preimplantatorio garantizar el HLA idéntico.
¿Qué patologías pueden diagnosticarse?
La sanidad pública andaluza aplica actualmente el Diagnóstico Genético Preimplantatorio a las siguientes patologías: atrofia muscular espinal, distrofia muscular de Duchenne, enfermedad de Huntington, fibrosis quística, hemofilia A y B, síndrome de Alport ligado al cromosoma X y enfermedades hereditarias recesivas ligadas al cromosoma X.
La Comisión Andaluza de Genética y Reproducción es el órgano colegiado adscrito a la Consejería de Salud que vela por el adecuado desarrollo del nuevo derecho, los criterios para su indicación y la información que se proporcione a las parejas candidatas, salvaguardando su intimidad y autonomía. Asimismo, es la responsable de proponer la actualización del listado de enfermedades susceptibles de ser diagnosticadas por Diagnóstico Genético Preimplantatorio. También realiza labores de asesoramiento a los especialistas y, ante la aparición de situaciones con implicaciones éticas, solicitará el correspondiente dictamen de la Comisión Autonómica de Ética e Investigación Sanitarias.
En este órgano colegiado participan representantes de la Administración sanitaria andaluza y de la Comisión Autonómica de Ética e Investigación Sanitarias, así como especialistas en reproducción asistida, embriología humana y genética; colegios de profesionales sanitarios; Agencia de Evaluación de Tecnologías Sanitarias de Andalucía; juristas expertos en la materia, y asociaciones de pacientes y de consumidores y usuarios.
Link
(AGI) - Madrid, Oct. 14 - His brother has a rare hereditary disease, beta-thalassaemia major: Javier, 3 kilos and 400 grams at birth, was genetically engineered to cure him. Javier was born on Sunday morning at the Virgen del Rocio hospital in Seville "with the hope of being able to give his brother Andres, the possibility to live", announced the Spanish Health Service, in a note. According to doctors, the blood in his umbilical cord will be used in a bone marrow transplant for his brother, so the boy will be able to start producing healthy red blood cells. "The possibility of healing the boy after the transplant is very high", said the doctors. Until the operation takes place, a date that has not yet been made public, the precious blood will remain in the Umbilical Cord Bank in Malaga. The new born does not have the genetic defect that would also give him the disease that his brother suffers from.
After the green light from the National Commission for Assisted Reproduction, Javier and Andres' parents, from Cadiz, chose to resort to a genetic pre-implant diagnosis; a technique that allows to verify if an embryo is healthy from a genetic point of view, before transferring it to the mother's uterus. For this type of diagnosis, absolutely prohibited in Italy, the embryo obtained by 'in vitro' fertilization' is genetically examined to verify that it does not carry any diseases. Beyond ethical implications, the method allowed the conception of a healthy baby, but also an ideal donor in order to cure the brother through a bone marrow transplant, since he is compatible and able to donate.
NEMO - 72 cases worldwide
From Simon's Mom blog:
More here
From JT's Dad blog:
We're not sure yet if it is bacterial or viral. If it is viral it will take him a lot longer than a child with a working immune system to kick it. Either way, Jacob is stable - not requiring oxygen- and for that we are thankful.
More here.
said Nancy Koehn, a historian at the Harvard Business School referring to the US bank intervention.
Same should apply for stem cell research. The goal is to cure devastating diseases as soon as possible.
Same should apply for stem cell research. The goal is to cure devastating diseases as soon as possible.
Link to article.
Chemical found to simplify production of stem cells
Harvard team hopes technique cuts tumor risk
By Maggie Fox
Reuters / October 13, 2008
WASHINGTON - Researchers trying to find ways to transform human skin cells into stem cells said yesterday that they found a shortcut by adding a chemical to the cells.
The chemical allowed the team at the Harvard Stem Cell Institute to use just two genes to transform skin cells into more powerful induced pluripotent stem cells, or iPS cells.
"This study demonstrates there's a possibility that instead of using genes and viruses to reprogram cells, one can use chemicals," said Dr. Doug Melton, who directed the study published in the journal Nature Biotechnology. That could reduce the risk of tumors developing in the cells.
Melton said Danwei Huangfu, a postdoctoral researcher in his lab, developed the new method.
"The exciting thing about Danwei's work is you can see for the first time that you could sprinkle chemicals on cells and make stem cells," said Melton, a researcher at the Howard Hughes Medical Institute.
Huangfu tried treating the cells first with valproic acid. After she did this, she found it took only two of the four usual genes to reprogram the human skin cells into iPS cells, which resemble embryonic stem cells.
Huangfu said the valproic acid unraveled the chromatin - the physical structure of the chromosomes - making it possible to get in and alter the DNA more easily.
"We may need two types of chemicals, one to loosen the chromatin structure, and one to reprogram. We are looking for that reprogramming chemical, and it should be possible to find it eventually," she said.
Stem cells are the body's master cells, giving rise to all the tissues, organs, and blood. Embryonic stem cells are considered the most versatile kinds of stem cells.
Doctors hope to someday use stem cells to transform medicine. Melton, for instance, wants to find a way to regenerate the pancreatic cells destroyed in type 1 diabetes and perhaps cure that disease.
But embryonic stem cells are difficult to make, requiring the use of an embryo or cloning technology. Many people also object to their use, and several countries, including the United States, limit funding for such experiments.
In the past year, several teams of scientists have reported finding a handful of genes that can transform ordinary skin cells into iPS cells.
To get these genes into the cells, they have had to use retroviruses, which integrate their own genetic material into the cells they infect. This can be dangerous and can cause tumors
Link
http://men.style.com/gq/features/moty/allengoldberg
Nominated by: Laurie Strongin, Washington, DC
"Allen is someone making the world better for seriously ill children. This started when our son, Henry, came into the world in 1995. Henry brought with him a winning smile, a zest for life, and a rare disease, Fanconi Anemia (FA). Allen then became a one-man make-a-wish foundation, arranging visits for Henry with his heroes, including Batman, Cal Ripken, and President Clinton. Henry died on December 11, 2002. Afterwards, Allen and I were determined to make life better for those kids, like Henry, who faced prolonged hospitalization. In 2003, we started the Hope for Henry Foundation (www.hopeforhenry.org). Recognizing the therapeutic effects of smiles and laughter, HFH gives kids undergoing bone marrow transplants and chemotherapy their choice of the latest iPods, DVD players, XM radios, and PSPs. We've given more than 700 pediatric patients these state-of-the-art gifts and are expanding our programs to more hospitals across the country. The message is clear: You are going to get better. This cool gift is yours for the long-term. Allen also blogs about his experience as a father coping with the loss of a child. Strangers who happen upon the blog tell him reading it provides comfort and helps them be a better parent."
Vote for Allen here.A breakthrough, then a surge, in stem cell research
A year after report, research into new type of stem cells continues to grow
Some scientists cite this potential in saying the true moral course is to accelerate research on embryonic cells because it might produce transplant tissue for illnesses like diabetes and Parkinson's disease. But Thomson also said many arguments for expanded federal funding have exaggerated the field's short-term promise. Perfecting such techniques could take many decades, he said. "It's certainly going to happen, but it's going to be hard, and people are not prepared for how hard it's likely to be."
The most profound effect of stem cells in the meantime could be to screen new drugs for safety and to gauge a medication's effectiveness on real human tissue without using patients as guinea pigs.
"It simply means that for the very first time we have access to the human body in the lab," Thomson said. "And for drug screening and drug discovery that's going to make a huge difference. When you use one of those drugs you won't know that human embryonic stem cells or iPS cells were involved. It won't make the front pages at all."
With a day's stubble on his chin and flip-flops on his feet, Thomson does not look like the sort of person who could change the world once, much less twice. He grew up in Oak Park and still keeps a beat-up dart board in his office that dates to his high school days. Thomson said he recently found a pay stub for $14 from his first job--delivering copies of the now-defunct Chicago Daily News.
One of his key skills as a biologist is the patient, constant nurturing of cells that other labs have found difficult to master.
"Things grow for me," Thomson said, gesturing to a set of flourishing staghorn fern plants in his office.
He said he's amazed at how quickly scientists have begun exploring the use of the reprogrammed skin cells he reported on last year. "People are jumping in very rapidly, much more rapidly than they did 10 years ago" after the initial discovery of embryonic stem cells, Thomson said.
In all, 812 labs in dozens of countries have requested the materials needed to reprogram ordinary cells into iPS cells, said Addgene, a Massachusetts-based repository for research supplies. By contrast, a half-dozen or so labs started working with embryonic stem cells in the months after his landmark 1998 paper, Thomson said.
In recent months Thomson has been racing other labs to grow an improved form of iPS cells that potentially could be used in human patients. The original method probably could not be tested in people because it relied on a retrovirus to activate a few genes that reprogram the cells.
The first successful attempt to make iPS cells without such potentially dangerous viruses was published online recently in the journal Science. A group from Massachusetts General Hospital grew the cells using relatively safe viruses that can be cleared from the cells once they do their jobs, though the method was not as efficient as existing techniques.
Already, researchers said, iPS cells have proved easier for individual labs to make than embryonic stem cells, fueling the intense interest.
Before the iPS papers, stem-cell research "was still a select fraternity," said Evan Snyder, director of the stem cells and regenerative medicine program at the California-basedBurnham Institute. "Now it's been kind of opened to the masses."
To use embryonic stem cells, labs must either make their own--which requires the destruction of human embryos--or get them from a lab like Thomson's that already has made its own cell lines. Only cell lines made before August 2001 qualify for federal funding under Bush's research restrictions.
With iPS cells, Snyder said, "you can do a skin biopsy on yourself if you want" and use that tissue as raw material for the stem cells.
Many scientists said the absence of ethical concerns over iPS cells also is a draw. "As soon as you have a cell type that is relatively free of this black cloud, then obviously there's huge pent-up interest," said George Daley, director of the stem cell program at Children's Hospital Boston.
Yet no one knows whether iPS cells can fill the same roles as embryonic stem cells, most researchers said. Some believe iPS cells may work differently depending on the type of tissue they came from--that skin cells might be difficult to transform into blood cells, for example.
That's a major reason why Thomson still refers to his original embryonic stem cells as a "gold standard."
"My belief is that if iPS cells turn out to be completely biologically equivalent [to embryonic stem cells], scientists will just migrate to them because they're easier to deal with," Thomson said. "But that may not be true, and we should simply let the science play itself out."
Taken rom Rhys Dad Blog.
I knew Ward 23 Newcastle was the 'last chance saloon' as it is for all those kids. And was not a dignified death, however where there is life there is hope, and I believed in the Prof and Mario,, but most of all I believed in Rhys.
So we proceeded, I would like to say that we went in nice, quiet and dignified, but all those that know me, know that is not my way... This was WAR and I treated it as such..sand bags out and digging in! I watched and questioned everyone, and nobody that did not get my respect was allowed in the room, only the best was good enough. I was tough on them, tough on me, tough on my loving wife.
It was horrible watching your own son disitergrate in front of your very eyes... The chemo wipes out your immune system. Your immune system is also your throat lining, stomach lining, intestinal lining, all this was ripped apart in order for us to get to day ZERO... Zero immune system, no protection, but allowing his new bone marrow to be introduced.
More here.
The makers of cold and cough medications announced yesterday they are voluntarily warning parents not to give their products to children younger than 4
Link.
THE EMBRYO DILEMMA
Donating embryos for research may be easier said than done
By Shari Roan, Los Angeles Times Staff Writer
October 6, 2008
CHRIS AND TANYA Bailey of Mission Viejo have a 3-year-old and triplet toddlers, all conceived through in vitro fertilization. After the birth of the triplets, they had 13 embryos left over in cryopreservation.
The idea of discarding them made the couple uneasy.
"I thought of them as potential life, but I don't think of them as children," says Chris Bailey. "They are definitely more than sperm and egg."
After much discussion, the couple decided to donate the embryos to research.
"We felt we were so lucky that research had been done and [that it] gave us the opportunity to have children," says Tanya Bailey. "So why not give our embryos to research as well to help somebody else out?"
The decision to donate to research, says Chris Bailey, "was a logical choice."
Even after grappling with the decision, however, many people find that donating to research is easier said than done. People wishing to donate to research must complete detailed paperwork and may even be asked to select the type of medical research for which they want their donation used. Others find they cannot proceed with their donation if they used egg and sperm donors who would not consent to the donation. Still others simply can't find a medical research organization to accept their donation.
For Californians, at least, donating unused embryos to research has become somewhat easier in the last two years. The launch of the state's stem cell research program and the opening of a dedicated tissue bank at UC San Francisco has opened at least one clear path for donation by providing a place for families to send their embryos where they will be available to researchers.
"As stem cell research moves forward and viable treatments emerge, there will be a greater demand for the use of frozen embryos," says Lois Uttley, director of the MergerWatch Project, a patients' rights organization based in New York City. "That could raise the profile of this issue."
The few states that fund stem cell research are more likely to be able to connect donating families to specific research programs that need embryos. People in other states do not have a clear pathway to donation, says R. Alta Charo, a professor of law and bioethics at the University of Wisconsin at Madison. "Most IVF clinics are not hooked up to a research team and they may not be able to refer couples to a clinic who can do it for them," she says.
Research options
At the UC San Francisco IVF Tissue Bank, which opened in 2005, couples have some choices as to the type of research they will be assisting. In cases where families donate to a specific research program, they often do not have the ability to specify the kind of research they wish to support.
There, however, Level 1 allows donated tissue to be studied but doesn't allow for the creation of stem cell lines. Level 2 allows all Level 1 research and the creation of a stem cell line. Level 3 allows Levels 1 and 2 research and, in addition, allows tissue to be used for stem cell nuclear transfer, which is also known as therapeutic cloning. In this type of research, stem cells are extracted from the embryo and are allowed to grow into a piece of tissue or organ, but never a human being.
Individuals who donate do not incur any costs, but they should be prepared to spend a lot of time on paperwork, says Denise Bernstein, coordinator of the bank. The packet sent to donors from UCSF contains pages of information as well as numerous forms, some of which must be notarized. In addition, the tissue bank conducts a telephone interview with the donors. The process' goal is to fully inform the donor or donors and to gather information scientists may need for their research.
"Some people say, 'You know what? It's easier to discard them,' " Bernstein says. "They have to be motivated to want to do this."
Though the UCSF bank's paperwork may be especially laborious because of the options for stem cell research, most research groups have a detailed consent process, says Dr. David Diaz, an infertility doctor with offices in Fullerton and Fountain Valley.
"The bureaucracy can be overwhelming." In his clinic, says Diaz, fewer than 1% of patients with frozen embryos have donated to research over the last three years.
And a sizable number of potential donors are turned away because of consent questions.
People who used donor eggs or donor sperm to create their embryos must obtain the consent of those donors before embryos can be released to researchers -- something that may be impossible if the gametes (eggs or sperm) were donated long ago, especially if anonymous donors were used. About 15% of all IVF cycles involve donor eggs.
Egg and sperm donors may also be asked to provide some personal health information. Researchers often want to know about the donor's personal and family medical history. Donors may be asked if they can be contacted in the future if the research yields information that could be useful to them or to see if any health changes have occurred since the donation.
Moreover, many research organizations -- including California state-funded research groups -- will not accept frozen embryos in which an egg donor was compensated in any way, which is often the case with anonymous donors.
Protecting gamete donors' rights is critical, says Nanette Elster, director of the Health Law Institute at DePaul UniversityCollege of Law in Chicago.
"Someone may have donated with the idea that he or she is donating to help a woman build a family," she says. "But if that is not what the family is going to use it for, maybe they wouldn't get consent. The donors are individuals with concerns and a stake in the process."
Many uses for embryos
Researchers hope that as the process becomes more familiar, more couples will donate. Evolving research suggests that stem cell researchers may have a need for many embryos.
For example, some studies show that individual stem cell lines have a preference for the way they develop, Charo notes. "Some differentiate more easily into heart tissue. Some seem to differentiate more easily into neurological tissue." Having a broad range of stem cells will help develop lines that can be used more efficiently.
Moreover, some people may have an immune reaction to tissues made with specific stem cells, she says. Thus it may be necessary to have a large variety of stem cell lines to get a variety of immunological matches. Disease-specific stem cell lines are also needed, researchers say. For example, cell lines may be created from an embryo that carries the gene for a specific disease or a high risk of developing that disease.
Link
Although stem cells may one day be derived by other methods, those derived from embryos are, for now, the gold standard in research, says Dr. Marie Csete, chief scientific officer for the California Institute for Regenerative Medicine.
"There is absolutely no need to take every frozen embryo and make a stem cell line," she says. "But the science is changing a lot. We need diverse human stem cell lines to really understand the biology of a stem cell at a baseline."
Cool! A faster way to identify life threatening infections instead of having to wait for 24, 84 or 72 hours for a culture to come back.
New test could help catch serious infections in babies
Blood test may spare infants invasive diagnostic tests and antibiotics
Boston, MA--A simple blood test may help detect serious bacterial infections (SBIs) like urinary tract infections and blood stream infections in young infants who come to the emergency department (ED) with fevers that have no clear cause. Researchers at Children's Hospital Boston, collaborating with investigators at George Washington University, show that a new diagnostic marker called procalcitonin can help identify infants at high risk for SBIs while potentially reducing unnecessary and aggressive testing, medication and hospitalization in low risk infants. The study, published in the October Pediatrics, is the first to examine procalcitonin as a tool for evaluating infant fever in an emergency situation.
The researchers used a novel procalcitonin test, recently approved by the FDA, in 234 feverish babies under 3 months of age, of whom 18 percent had definite or possible SBIs confirmed by independent clinical criteria. The results showed that procalcitonin not only detected all cases of SBIs in feverous infants but proved sensitive enough to establish a threshold value that would identify infants at low risk for serious infections. Indeed, its overall performance as a single clinical marker of infection approached that of current strategies that involve a variety of laboratory tests and clinical evaluations.
In the United States, infant fever accounts for a vast majority of pediatric visits to the ED, of which up to 20 percent of cases have no identifiable cause of infection. While most turn out to be minor and self-limiting illnesses, a proportion of infants have SBIs such as bacteremia, meningitis, pneumonia or urinary tract infections. The risk is most significant in infants under 3 months of age.
"About 12 percent of those whom we consider 'well appearing' end up having serious infections when we do an evaluation," said Richard Bachur, MD, acting chief of emergency medicine at Children's.
Because clinicians cannot reliably determine which children with fever have more serious infections, many babies end up undergoing extensive evaluations. Routine evaluation of infants less than 3 months of age includes blood tests, urine tests, and often a lumbar puncture for spinal fluid, followed by treatment in the hospital with antibiotics.
Prompted by the inefficiency of current fever management in young infants, Bachur and colleagues have sought a rapid diagnostic test that will determine which children have serious infections at the first visit to the ED. "We hope to identify those infants that are at very low risk of serious infection and tailor their evaluation so as to minimize invasive testing and exposure to unnecessary antibiotics," said Bachur.
The high sensitivity of the new procalcitonin test has allowed Bachur and colleagues to establish realistic cut-off values to help guide clinicians in identifying children who are at low risk for SBIs.
The researchers are now looking to do a multi-center study to evaluate the use of procalcitonin on a larger scale. If it proves to be valuable, Bachur hopes it will become a standard tool for the evaluation of young infants with fever.
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The study was supported by the Frederick H. Lovejoy, Jr, MD Resident Research Fund and the American Academy of Pediatrics Resident Research Grant. The biomarker assay, procalcitonin (PCT), is available to clinicians and manufactured by Brahms Diagnostica.
Children's Hospital Boston is home to the world's largest research enterprise based at a pediatric medical center, where its discoveries have benefited both children and adults since 1869. More than 500 scientists, including eight members of the National Academy of Sciences, 11 members of the Institute of Medicine and 12 members of the Howard Hughes Medical Institute comprise Children's research community. Founded as a 20-bed hospital for children, Children's Hospital Boston today is a 397-bed comprehensive center for pediatric and adolescent health care grounded in the values of excellence in patient care and sensitivity to the complex needs and diversity of children and families. Children's also is the primary pediatric teaching affiliate of Harvard Medical School. For more information about the hospital and its research visit:www.childrenshospital.org/newsroom.
What a coincidence! Check out the last name of reporter who writes this story about PGD is "Goodchild".
British couple have entire family using embryo screening
By Sophie Goodchild
A couple have become one of the first in Britain to conceive their entire family through a genetic screening technique. oanne and Adam Henry feared they would never be parents after doctors discovered Joanne had a gene defect. Some of her chromosomes were in the wrong order, causing severe abnormalities in their first child Jasmine. hey were devastated when Jasmine died within minutes of her birth.But experts at Guy's and St Thomas' Hospital in London used a pioneering embryo-testing technique to help conceive their healthy children Amy, five, and Harrison, who is just a few weeks old. Pre-implantation genetic diagnosis is used to assess the health and viability of embryos. Doctors give patients fertility treatment then take a cell from the resulting embryos to test if they are 'normal'. The healthy embryo is implanted back into the mother's womb. Mr and Mrs Henry, of Welwyn Garden City, Hertfordshire, are among only a handful of couples in Britain to have had more than one child using this method. Mr Henry, 38, said his children would not be here without the help of Guy's. We thought we would have to come to terms with not having children but now we have a life with two healthy children in it,' said the company director. Amy was the only one of seven eggs produced by Mrs Henry, 35, which was of good enough quality to implant. This treatment is amazing because without it our children would not be here,' Mr Henry added. 'It was horrible to have a loss like [Jasmine] so when Amy arrived it felt like a real sense of achievement. We even have a photograph of her as an embryo.' About one in 500 people are 'silent' carriers of the same chromosome disorder as Mrs Henry. The condition only becomes obvious when they conceive children. It results in a late miscarriage or a child born with severe disabilities.
Under new laws, doctors can now make more use of PGD to create ' disease free' embryos.
This includes screening embryos to eliminate genes which can increase the chance of developing cancer as well as diseases such as cystic fibrosis and muscular dystrophy. Mr and Mrs Henry were treated by Alison Lashwood at Guy's. Four couples at their fertility unit have become parents to more than one child over the past decade, thanks to PGD.
A fertility nurse told the Evening Standard: 'We only use this procedure if we know the child is at risk of abnormalities.
'It was only because Mrs Henry's first baby was stillborn that we found out she was a carrier of a chromosome rearrangement.
'It is not a solution because a child could still be a carrier, but it spares parents the heartache of repeated miscarriage.'
Some experts have raised concerns about the procedure because doctors have to discard the 'abnormal' embryos.
But Mr Henry said: 'The whole process was very gruelling and we didn't know if we could go through it all again - we had been through so much already, especially with losing Jasmine.
'I'm annoyed people saying, 'Is this ethically correct?' No one is trying to clone a child. We are just two normal people who lost a baby.'
