NIH scientist transforming treatment of sickle cell disease

By Partnership for Public Service August 5 at 9:54 AM

Dr. Griffin Rodgers spends most of his waking hours leading the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), but he also manages to carve out time to work on a life-long passion discovering a cure for sickle cell disease.

Long before becoming the director of NIDDK, Rodgers was credited with discovering the first effective therapy for sickle cell disease, an inherited blood disorder that affects more than 90,000 Americans, most of them African-Americans. The disease, which affects millions of people throughout the world, can damage bones, joints and internal organs, cause acute and chronic pain, and often result in premature death.

Prior to his discovery of a drug treatment in the 1990s, the only options for sickle cell patients were blood transfusions for pain and supportive care.

This initial breakthrough has been followed by the recent announcement that Rodgers and a team of National Institutes of Health (NIH) researchers have developed a modified blood stem-cell transplant regimen that is highly effective in reversing sickle cell disease in adults. The findings, based on a clinical trial of 30 patients, represent a potentially transformative treatment.

Dr. Neal Young, chief of NIHs Hematology Branch of the National Heart, Lung and Blood Institute, said Rodgers has been the driving force behind the advanced medical treatments for people with sickle cell disease. His work, said Young, is a very big deal because it will save the lives and alleviate the suffering of thousands of people.

Dr. Thomas Starzl, a physician and researcher who performed the worlds first liver transplant, wholeheartedly concurred.

Griffin Rodgers work on sickle cell disease has been revolutionary, said Starzl. I can only give him rave reviewsfive stars.

Rodgers grew up in New Orleans where he had three high school friends who became debilitated with sickle cell disease. Two of those friends died in their teenage years and the third passed away a few years after high school.

These deaths left a tremendous impression on Rodgers, who pursued a medical career that led him to NIH in 1984 where he began his work on sickle cell disease. Over the years as he made his mark in the laboratory and the clinical setting, Rodgers also progressed through the managerial ranks, heading NIDDKs Molecular and Clinical Hematology Branch starting in 1998, becoming deputy director of NIDDK in 2001 and director of the institute in 2007.

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NIH scientist transforming treatment of sickle cell disease

Lumbar disc pain and knee arthritis 8 months after stem cell treatment by Harry Adelson, N.D. – Video


Lumbar disc pain and knee arthritis 8 months after stem cell treatment by Harry Adelson, N.D.
Steve discusses his outcome eight months after his stem cell treatment by Harry Adelson, N.D. for his low back pain and arthritic knee http://www.docereclinics.com.

By: Harry Adelson, N.D.

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Lumbar disc pain and knee arthritis 8 months after stem cell treatment by Harry Adelson, N.D. - Video

Recent advances in stem cell biology

PUBLIC RELEASE DATE:

31-Jul-2014

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research

Advances in stem cell research will provide enormous opportunities for both biological and future clinical applications. Basically, stem cells could replicate any other cells in the body, offering immense hope of curing Alzheimer's disease, repairing damaged spinal cords, treating kidney, liver and lung diseases and making damaged hearts whole. The potential for profit is staggering. Prof. Jinhui Chen from Indiana University in USA considered that this field of research still faces myriad biological, ethical, legal, political, and financial challenges. The eventual resolution of these conflicts will determine the success of the research and potentially the face of medicine in the future. The relevant study has been published in the Neural Regeneration Research (Vol. 9, No. 7, 2014).

###

Article: " A brief review of recent advances in stem cell biology " by Jinhui Chen1, Libing Zhou2, Su-yue Pan3 (1 Stark Neuroscience Research Institute and Department of Neurological Surgery, Indiana University School of Medicine, Indianapolis, IN, USA; 2 Guangdong-Hongkong-Macau Institute of CNS Regeneration (GHMICR), Jinan University, Guangzhou, Guangdong Province, China; 3 Department of Neurology, Nanfang Hospital, Southern Medical University, Guangzhou, Guangdong Province, China)

Chen JH, Zhou LB, Pan SY. A brief review of recent advances in stem cell biology. Neural Regen Res.2014;9(7):684-687.

Contact: Meng Zhao eic@nrren.org 86-138-049-98773 Neural Regeneration Research http://www.nrronline.org/

AAAS and EurekAlert! are not responsible for the accuracy of news releases posted to EurekAlert! by contributing institutions or for the use of any information through the EurekAlert! system.

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Recent advances in stem cell biology

Aging Immune System May Get Kick-Start from Discovery of Molecular Defect

Health and Medicine for Seniors

Aging Immune System May Get Kick-Start from Discovery of Molecular Defect

Old stem cells are not just sitting there with damaged DNA ready to develop cancer, as it has long been postulated

"The decline of stem-cell function is a big part of age-related problems. Achieving longer lives relies in part on achieving a better understanding of why stem cells are not able to maintain optimal functioning."

Emmanuelle Passegu, PhD

July 31, 2014 - There's a good reason seniors over 60 are not donor candidates for bone marrow transplantation. The immune system ages and weakens with time, making the elderly prone to life-threatening infection and other maladies, and a UC San Francisco research team now has discovered a reason why.

"We have found the cellular mechanism responsible for the inability of blood-forming cells to maintain blood production over time in an old organism, and have identified molecular defects that could be restored for rejuvenation therapies," said Emmanuelle Passegu, PhD, a professor of medicine and a member of the Eli and Edythe Broad Center of Regeneration Medicine and Stem Cell Research at UCSF.

Passegu, an expert on the stem cells that give rise to the blood and immune system, led a team that published the new findings online July 30, 2014 in the journal Nature.

Blood and immune cells are short-lived, and unlike most tissues, must be constantly replenished. The cells that must keep producing them throughout a lifetime are called "hematopoietic stem cells."

Through cycles of cell division these stem cells preserve their own numbers and generate the daughter cells that give rise to replacement blood and immune cells. But the hematopoietic stem cells falter with age, because they lose the ability to replicate their DNA accurately and efficiently during cell division, Passegu's lab team determined.

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Aging Immune System May Get Kick-Start from Discovery of Molecular Defect

What clinical trials for gene and stem cell therapy are under way in your London laboratory? – Video


What clinical trials for gene and stem cell therapy are under way in your London laboratory?
Robin Ali, BSc, PhD, FMedSci, internationally known for his research in gene and cell-based therapy for the treatment of retinal degeneration, has joined the U-M Department of Ophthalmology...

By: Kellogg Eye Center - Ann Arbor

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What clinical trials for gene and stem cell therapy are under way in your London laboratory? - Video

Early Stem Cell Transplant Vital in ‘Bubble Boy’ Disease …

By Amy Norton HealthDay Reporter

WEDNESDAY, July 30, 2014 (HealthDay News) -- Babies born with so-called "bubble boy" disease can often be cured with a stem cell transplant, regardless of the donor -- but early treatment is critical, a new study finds.

Severe combined immunodeficiency (SCID), as the condition is medically known, actually refers to a group of rare genetic disorders that all but eliminate the immune system. That leaves children at high risk of severe infections.

The term "bubble boy" became popular after a Texas boy with SCID lived in a plastic bubble to ward off infections. The boy, David Vetter, died in 1984 at the age of 12, after an unsuccessful bone marrow transplant -- an attempt to give him a functioning immune system.

Today, children with SCID have a high chance of survival if they receive an early stem cell transplant, researchers report in the July 31 issue of the New England Journal of Medicine.

In the best-case scenario, a child would get stem cells -- the blood-forming cells within bone marrow -- from a sibling who is a perfect match for certain immune-system genes.

But that's not always an option, partly because kids with SCID are often their parents' first child, said Dr. John Cunningham, director of hematopoietic stem cell transplantation at the University of Chicago Comer Children's Hospital. He was not involved in the study.

In those cases, doctors typically turn to a parent -- who is usually a "half" match, but whose stem cells can be purified to improve the odds of success. Sometimes, stem cells from an unrelated, genetically matched donor can be used.

The good news: Regardless of the donor, children with SCID can frequently be cured, according to the new findings. But early detection and treatment is vital.

"These findings show that if you do these transplants early -- before [the age of] 3.5 months, in a child without infection -- the results are really quite comparable to what you have with a matched sibling," said lead researcher Dr. Richard O'Reilly, chief of the pediatric bone marrow transplant service at Memorial Sloan-Kettering Cancer Center in New York City.

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Early Stem Cell Transplant Vital in 'Bubble Boy' Disease ...

Stem cell treatment used by Chris Johnson, hundreds of NFL …

New JetChris Johnson had stem cells from his bone marrow reinjected into his knee to augment Januarysurgery for a torn meniscus. The hope is that it wouldboost healingand perhaps rebuild cartilage. (AP)

Hes 28. He has five 1,000-yard NFL rushing seasons to his name, one 2,000-yarder and a burning desire to prove hes the same speedster hes always been. So when Chris Johnson visited orthopedic surgeon James Andrews in January to fix his ailing left knee, he liked the sound of two intriguing words: Stem cells.

The veteran running back tore the meniscus in that knee in Week 3 of the 2013 seasonhis last with the Titans before being cutbut never missed a game. The injury to the knees natural shock absorber also caused other damage in the joint, and Andrews presented an option that might augment what surgery alone could do. The plan: Take stem cells, the bodys universal building blocks, and deliver them directly to the construction site.

When I tore my meniscus and played the season out, through the wear and tear, I lost a lot of cartilage, says Johnson, who was signed by the Jets to bring explosiveness to their offense. When you put the stem cells in, it might be able to help rebuild that cartilage in your knee. Hopefully, it makes your knee better for even more years.

On the day of his surgery at the Andrews Institute in Gulf Breeze, Fla., Johnson had a small amount of his bone marrow60 milliliters, or the volume of a shot glasssiphoned out of the iliac crest of his pelvis with a long needle pushed through a tiny incision in his skin. Less than an hour later, at the end of the arthroscopic procedure to repair his meniscus, a concentrate of thousands of stem cells from the bone marrow was injected directly into Johnsons knee joint.

Instead of the usual four-to-six-week recovery time from the scope, Johnson stayed off the practice field for the rest of the offseason, giving the stem-cell treatment maximum time to work. At the least, stem cells are a powerful anti-inflammatory. But the hope is they may also play a role in boosting the healing of injured tissues, including stubborn ones like the meniscus, which lacks a robust blood supply, or cartilage, which has long been irreplaceable.

Stem cells are far from mainstreamNFL teams will often not pick up the bill, and the overseas market for treatments not approved in the U.S. makes the whole field seem somewhat taboo.

Johnson is one of hundredsyes, hundredsof NFL players who have invested in the promise of stem cells in the past few years. Peyton Manning reportedly tried a stem-cell treatment in Europe in 2011, his final year with the Colts, to fast-track his recovery from neck surgery. Giants cornerback Prince Amukamara had a slow-healing broken metatarsal treated with stem cells by a foot specialist in North Carolina after his teams Super Bowl XLVI run. One NFL linebacker paid $6,000 a pop for a 1-milliliter vial of donated placenta tissue containing stem cells to be injected into each of his beat-up knees this offseasonbut asked for his name not to be used in this story because he didnt tell his teams medical staff.

Such treatment is more common than you might realize among NFL players (hundreds of players across 32 teams averages to at least six players per team), but its also far from mainstream. Stem cells are still somewhat in the shadowsevidence of their usefulness in treating athletes injuries is so far largely anecdotal, NFL teams often will not pick up the bill for players, and the overseas market for treatments not approved in the U.S. makes the whole field seem somewhat taboo.

Theres a push to change that, though, and Andrews is an important figure at the forefront. His group is currently building a laboratory at its Florida facility specifically dedicated to biologicsthe term refers to substances that are produced in living systems such as humans, animals and microorganisms, rather than manufactured like drugsto be able to offer their star-studded clientele more of these treatments more effectively in the U.S. The agenda includes a research study with retired NFL players on how well stem cells work in treating arthritis of the knee; a trial of a Malaysian technique for regenerating cartilage by using stem cells from the blood after microfracture surgery; and exploring whether torn ACL tissue can be repurposed to help the new ligament graft heal more quickly.

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Stem cell treatment used by Chris Johnson, hundreds of NFL ...

The Adult Stem Cell Technology Center, LLC Launches A Marketing Campaign To License Adult Stem Cell Biotechnologies

Boston, MA (PRWEB) July 31, 2014

Bostons Adult Stem Cell Technology Center, LLC (ASCTC) finds itself flush with innovative adult stem cell biotechnologies. Currently the company holds seven recently issued patents and has three additional patent applications currently under examination by the U.S. Patent and Trademarks Office.

The patented inventions address two of the most vexing problems in adult stem cell biology research and regenerative medicine. Adult stem cells are difficult to identify; and they have been difficult to multiply to sufficient numbers to support regenerative medicine applications.

ASCTC has addressed the identity problem by developing patented biomarkers that are found exclusively on adult stem cells. The biomarkers are based on ASCTCs expertise in defining properties of adult stem cells that are not shared by any other normal cell types in the body. The patented biomarkers also identify some types of cancer stem cells. Therefore, they have applications in both stem cell medicine and cancer medicine.

ASCTCa success in developing procedures for producing adult stem cells in large numbers is due to the companys expertise in adult stem cell growth control. ASCTCs technology uses natural compounds found in the body to instruct adult stem cells to multiply in a controlled manner as during normal body growth.

The companys patented method for controlling adult stem cells to multiply without losing their stem cell properties has applications for many different types of adult stem cells. ASCTCs approved patents demonstrate the application of the method for production of human liver stem cells, hair follicle stem cells, and human pancreatic stem cells; but the technology has general application to adult stem cells found in many other types of organs and tissues.

In addition to the main focus on adult stem cell technologies, ASCTCs most recently issued patent applies its cell multiplication methods to produce induced pluripotent stem cells (iPSCs) without transferring exogenous genes. This gene-free single agent method should offer significant value to the many mushrooming companies that supply iPSCs and iPSC production reagents.

As a small start-up, ASCTC is employing a social media marketing strategy. In the past week, the company has launched patent licensing ads on LinkedIn, Vocus, and Facebook, as well increased its advertising references within its recently established Twitter presence.

It would be a shame for these technologies to lie dormant, just because our hands are full with other projects at the moment. James Sherley, director of ASCTC, relates that the companys two main business efforts require only a fraction of its available intellectual property. ASCTC is currently focused on bringing laboratory-scale production of human liver stem cells to manufacturing scales and developing a computer simulation assay for preclinical detection of drug candidates with intolerable toxicity due to adverse effects on adult stem cells.

Sherley adds, We already have a few companies that have expressed interest in licensing. But we could do a lot better at reaching others whose development efforts would benefit from ASCTCs unique technologies. Love to hear from ViaCyte!

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The Adult Stem Cell Technology Center, LLC Launches A Marketing Campaign To License Adult Stem Cell Biotechnologies

Early stem cell transplant may cure "bubble boy" disease

"Bubble boy" David Vetter lived in a protective environment designed by NASA engineers. He died of complications after receiving a bone marrow transplant in 1984, at the age of 12. Baylor College of Medicine Photo Archives

Babies born with so-called "bubble boy" disease can often be cured with a stem cell transplant, regardless of the donor -- but early treatment is critical, a new study finds.

Severe combined immunodeficiency (SCID), as the condition is medically known, actually refers to a group of rare genetic disorders that all but eliminate the immune system. That leaves children at high risk of severe infections.

The term "bubble boy" became popular after a Texas boy with SCID lived in a plastic bubble to ward off infections. The boy, David Vetter, died in 1984 at the age of 12, after an unsuccessful bone marrow transplant -- an attempt to give him a functioning immune system.

15 Photos

Immune disorder forced David Vetter to live in bubble - but breakthroughs from his story now enable similar kids to live free

In the best-case scenario, a child would get stem cells -- the blood-forming cells within bone marrow -- from a sibling who is a perfect match for certain immune-system genes.

But that's not always an option, partly because kids with SCID are often their parents' first child, said Dr. John Cunningham, director of hematopoietic stem cell transplantation at the University of Chicago Comer Children's Hospital. He was not involved in the study.

In those cases, doctors typically turn to a parent -- who is usually a "half" match, but whose stem cells can be purified to improve the odds of success. Sometimes, stem cells from an unrelated, genetically matched donor can be used.

The good news: Regardless of the donor, children with SCID can frequently be cured, according to the new findings. But early detection and treatment is vital.

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Early stem cell transplant may cure "bubble boy" disease

Extracted fat put forward as cure for wobbly knees

Mary Ann Benitez

Thursday, July 31, 2014

Health Innovative Technology chief executive Matthew Fan Chun-yin said his company is banking on the fat graft's potential as a drug in future and acquired ProStemCell last year to expand its bio- bank storage and research capabilities.

The government plans to issue new rules and regulations governing stem cell laboratories, he said.

During a tour of the laboratories of HIT ProStemCell in Kowloon Bay, Fan said scientists discovered stem cells from the extracted fat stimulate the repair of the knee cartilage and relieves pain for osteoarthritic patients.

He said it was unfortunate that liposuction has been tarnished by the death of a dance teacher and studio owner Josephine Lee Ka-ying, 32, who fell comatose and died after a four-hour liposuction treatment at the Regrowth Hair Transplant Centre in Tsim Sha Tsui on June 27 this year.

"You should not do any liposuction in a non-qualified day surgery center," said Fan, a former dietician.

He said ProStemCell, which HIT acquired last year, built a "clean-room" laboratory four years ago which meets the US Food and Drug Administration's standards.

The classification is the same required of companies for drug manufacturing and for hospital operating theaters which do open-heart surgery.

ProStemCell built the laboratory because the administration put out guidelines in 2009 requiring such stringent laboratory guidelines.

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Extracted fat put forward as cure for wobbly knees