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Category Archives: Pennsylvania Stem Cells

Knee Stem Cell Therapy and Platelet Procedures Pittsburgh …

Posted: July 2, 2015 at 12:56 am

Rehabilitation and Pain Specialists is the only medical center in Pennsylvania to offer the nations most advanced, non-surgical stem cell and platelet treatments for knee conditions, including injuries to the knee meniscus, anterior cruciate ligament, MCL, knee cartilage and degenerative arthritis. Traditional solutions to these problems include invasive procedures, from arthroscopic knee surgery to total knee joint replacement. With this range of surgeries, patients typically require months of rehabilitation to regain strength, range-of-motion and balance, plus the patient must be prepared to take on the risks associated with surgical trauma and potential for infection. Stem Cell Therapy and Platelet treatments are minimally-invasive regenerative treatments that may help resolve knee pain with a same-day injection procedure. Patients avoid the lengthy periods of downtime and painful rehabilitation that typically follow invasive surgeries.

Stem Cells are in all of us and they are responsible for healing injured bone, ligaments, tendons and tissues. As we get older or injured, we sometimes cannot get enough of these cells into the area in need. The Regenexx Procedures help solve that problem by precisely delivering a high concentration of stem cells into the injured area and aiding your bodys ability to heal naturally. Patients experience very little down time and they typically avoid the long, painful rehabilitation periods that often follow surgery to restore joint strength and mobility. To determine if youre a candidate for these procedures, please complete our Candidate Form below.

Regenexx Procedures were recently featured on The Doctors TV show. The episode featured Dr. Christopher J. Centeno and Dr. Ron Hanson from the Centeno-Schultz Clinic in Colorado, along with a patient who sought stem cell treatment following traditional knee surgery. The 6 minute video provides a nice overview of the Stem Cell procedures that are performed at our offices in Pittsburgh, PA.

Seattle King TV recently featured Regenexx patient Paul Lyon in this news segment. Lyon chose to undergo a Regenexx-SD knee procedure, rather than an invasive and traumatic knee joint replacement surgery.

The Regenexx procedure begins when the doctor numbs the back of the hip (PSIS) and takes a small bone marrow sample through a needle, as well as a blood draw from a vein in the arm. The marrow is rich in Mesenchymal Stem Cells, which are responsible for healing damaged tissues. The stems cells are isolated from the marrow sample and platelets are isolated from the blood. After preparation, these two components will be reinjected directly into the damaged joint using advanced imaging guidance, ensuring the cells are placed in the exact location of need.

If you are suffering from a joint injury, joint pain, a non-healing fracture or a degenerative condition like osteoarthritis, you may be a good candidate for these ground-breaking stem cell and blood platelet treatments. Please complete the Procedure Candidate Form below and we will immediately email you more information.

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Penn vet team points to new colon cancer culprit

Posted: March 16, 2015 at 9:03 pm

IMAGE:Msi2 (stained red) is broadly expressed in intestinal tumors that result from loss of APC. Penn researchers believe activation of Msi2 downstream of APC loss drives metabolic activation of stem... view more

Colon cancer is a heavily studied disease -- and for good reason. It is one of the leading causes of cancer-related deaths worldwide, and its numbers are on the rise, from 500,000 deaths in 1990 to 700,000 in 2010.

This growth comes despite scientists' ever-increasing knowledge of the genetic mutations that initiate and drive this disease. Now, a team of researchers from the University of Pennsylvania has found evidence of a new culprit in the disease, a protein called MSI2.

Their findings provide a new target for potential therapeutic intervention in colorectal cancer and enhance our understanding of the complexities of cancer initiation and progression. Further studies of MSI2 may even help explain how the disease can return after lying dormant for years.

Christopher Lengner, an assistant professor in the Department of Animal Biology in Penn's School of Veterinary Medicine, was the senior author on the work. Collaborators from Penn Vet included co-lead authors Shan Wang and Ning Li as well as Maryam Yousefi, Angela Nakauka-Ddamba and Kimberly Parada. Additional co-authors from Penn included Fan Li, Brian Gregory and Shilpa Rao.

The Penn researchers teamed with Gerard Minuesa and Michael G. Kharas from Memorial Sloan-Kettering Cancer Center, Zhengquan Yu from China Agricultural University and Yarden Katz from the Broad Institute.

The research will appear in Nature Communications.

Lengner's research has long focused on how stem cells are able to differentiate into a variety of cell types, an ability known as stem cell potency. His lab's work dovetails with cancer research in that it is believed that a population of so-called cancer stem cells is responsible for sustaining cancer in the body once it is established, just as normal stem cells are responsible for continually renewing and sustaining our healthy cells.

In earlier studies, Lengner and Kharas had found that an RNA binding protein called MSI2 played a role in supporting the potency of hematopoietic stem cells. This same protein was also found to be highly active in blood cancers. Yet unlike other well-established genes that, when mutated, result in increased tumor formation, the MSI2 gene itself is not directly mutated in tumors. Rather, the normal, intact gene becomes highly activated as cancer progresses.

When MSI2 is active, the protein promotes cancer not by changing the expression of genes but by altering the ability of RNA to make proteins. Thus, until now, the contribution of MSI2 went undetected by traditional research techniques that are largely aimed at identifying mutations in DNA sequence and alterations in gene expression patterns.

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MD Anderson Names Hwu as Head of Cancer Medicine

Posted: February 26, 2015 at 12:58 am

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Newswise Patrick Hwu, M.D., chair of Melanoma Medical Oncology and Sarcoma Medical Oncology at The University of Texas MD Anderson Cancer Center, has been named division head of Cancer Medicine effective March 4.

Hwus selection came after a competitive national search to fill the position currently being served by Richard Champlin, M.D., on an ad interim basis. Champlin will continue to serve as chair of Stem Cell Transplantation and Cellular Therapy.

Dr. Hwu is an internationally respected physician-scientist who has 25 years of experience in the fields of tumor immunology, targeted therapies and translational studies, said Ethan Dmitrovsky, M.D., provost and executive vice president. Hes a seasoned leader and has successfully chaired two departments and served as co-director of MD Andersons Center for Cancer Immunology Research and its immunotherapy platform. He has also held endowed positions, including the Sheikh Mohamed Bin Zayed Al Nahyan Distinguished University Chair in Cancer Research. Were delighted that he will be leading this vital division, and are thankful for Dr. Champlins skillful leadership during our search for a new division head.

Hwu earned his medical degree from the Medical College of Pennsylvania in Philadelphia and served as a house officer in internal medicine at The Johns Hopkins Hospital. He completed a fellowship in oncology at the National Cancer Institute, where he continued to work for 10 years as a principal investigator leading tumor immunology studies. He joined MD Anderson in 2003 as the first chair of Melanoma Medical Oncology.

Dr. Hwu is an accomplished clinician, researcher and administrator who is well positioned to take the Division of Cancer Medicine already recognized as a global leader to the next level, said Raymond S. Greenberg, M.D., Ph.D., executive vice chancellor for health affairs, The University of Texas System.

An expert in tumor immunology, Hwu has translated multiple concepts from the laboratory to the clinic and helped to launch the field of gene modified T cells, publishing research on the first chimeric antigen receptor (CAR) directed against cancer. Clinical trials using CAR-transduced T cells now are being studied in many types of cancers, and MD Anderson has established an adoptive T cell therapy program, treating more than 80 melanoma patients with T cells to date.

In addition, Hwu has produced novel, ongoing clinical trials based on his teams findings, including a study of combination T cell and dendritic cell therapy and a study of T cells modified with chemokine receptor genes to enhance their migration to the tumor. His most recent preclinical studies have focused on combinations of immune checkpoint blockade and T cell therapy, as well as rational combinations of targeted therapies and immunotherapies. Both of these concepts are being translated to the clinic.

Dr. Hwu and I worked closely together at the NCI for 13 years. He is one of those rare visionaries when it comes to expanding the frontiers of cancer medicine, said Steven A. Rosenberg, M.D., Ph.D., head of the Tumor Immunology Section and chief of the Surgery Branch at the National Cancer Institutes Center for Cancer Research. He is a brilliant scientist and leader. I congratulate him on this important position and look forward to working with him in his new leadership role at MD Anderson.

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Stemiotics Licenses Modified RNA for Cell Reprogramming

Posted: December 22, 2014 at 11:02 am

San Diego, CA (PRWEB) December 22, 2014

Stemiotics, Inc., a supplier of stem cell generation services, today announced it has licensed key intellectual property pertaining to the application of modified RNA from CELLSCRIPT, LLC of Madison, WI. CELLSCRIPT holds an exclusive license to a portfolio of issued and pending patents based on discoveries made at the University of Pennsylvania covering the use of synthetic messenger RNA (mRNA) containing modified nucleotides to evade antiviral responses in mammalian cells. This breakthrough technology has opened new vistas for the application of mRNA as a gene expression vector in human therapeutics and cell fate manipulation. The license to Stemiotics is for use of modified RNA in the production of human induced pluripotent stem cells (iPSCs) for research applications such as disease modeling and drug discovery.

Stemiotics is already using CELLSCRIPT's ultra-low immunogenicity mRNA to reprogram human skin cells into pluripotent stem cells with the potential to become any cell type in the body. In addition to the incorporation of modified nucleotides, CELLSCRIPT's advanced synthetic mRNA is subject to novel purification techniques that virtually eliminate residual innate immune responses to the mRNA on delivery into human or animal cells in vivo or in culture. Stemiotics is committed to applying clinically relevant, state-of-the-art technology in its iPSC derivation pipeline. The company uses only xeno-free reagents at all steps of the process, from the initial expansion of the donor skin cells to the cryogenic preservation of the artificially-induced pluripotent stem cells. Stemiotics employs the most potent cocktail of cellular reprogramming factors currently available, including engineered transcription factors based on IP which has been exclusively licensed to CELLSCRIPT. This sophisticated technology allows Stemiotics to convert human skin cells into pluripotent stem cells in just over a week in feeder-free conditions and without the need for drug-like small molecule accelerants.

Stemiotics believes that the mRNA-based reprogramming system it has developed is the fastest, most productive and safest approach to converting human skin cells into pluripotent stem cells yet devised. The company offers high-throughput iPSC derivation on a fee-for-service basis with fast turnaround times and at a cost of only $1000 per line, an order of magnitude below prevailing industry norms. The licensing relationship with CELLSCRIPT will further enhance Stemiotics position as an emerging leader in the field of cellular reprogramming, with all its great promise for advancing the understanding of disease, the development of new drugs and, ultimately, for cell-based therapies and regenerative medicine.

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Penn Medicine Researchers Announce Latest Results of Investigational Cellular Therapy CTL019

Posted: December 8, 2014 at 5:56 am

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EMBARGOED FOR RELEASE UNTIL: Abstract #380: 10 a.m. PST, Saturday, Dec. 6 Abstract #1982 and #1983: 5:30 p.m. PST, Saturday, Dec. 6 Abstracts #3087 and #2296: 6:00 p.m. PST, Sunday, Dec. 7

Newswise SAN FRANCISCO The latest results of clinical trials of more than 125 patients testing an investigational personalized cellular therapy known as CTL019 will be presented by a University of Pennsylvania research team at the 56th American Society of Hematology Annual Meeting and Exposition. Highlights of the new trial results will include a response rate of more than 90 percent among pediatric acute lymphoblastic leukemia patients, and results from the first lymphoma trials testing the approach, including a 100 percent response rate among follicular lymphoma patients and 45 percent response rate among those with diffuse large B-cell lymphoma.

We have now treated more than 125 patients in our trials of the chimeric antigen receptor (CAR) therapy CTL019, and with each patient, we learn more and more about the potential of this therapy, said the research teams leader, Carl June, MD, the Richard W. Vague Professor in Immunotherapy in the department of Pathology and Laboratory Medicine in Penns Perelman School of Medicine, and director of Translational Research in the Abramson Cancer Center. We are continuing to refine our approach to ensure the best outcomes for patients who may be eligible for this experimental therapy, and we hope our findings will contribute to the emerging field of cellular therapy as a whole.

This personalized cellular therapy approach begins with patients own immune cells, collected through a procedure similar to dialysis. The cells are then engineered in a laboratory and infused back into patients bodies after being trained to hunt and kill their cancer cells. All patients who enroll in the trials have cancers that have progressed despite multiple conventional therapies.

Updated results of a CTL019 trial for children and young adults with relapsed, treatment-resistant acute lymphocytic leukemia who were treated at the Childrens Hospital of Philadelphia (Abstract #380) includes data on 39 patients. The findings, which will be presented by Stephan Grupp, MD, PhD, the Yetta Deitch Novotny Professor of Pediatrics and director of Translational Research in the Center for Childhood Cancer Research at the Children's Hospital of Philadelphia, build on the teams report on 25 pediatric and five adult patients which was published in the New England Journal of Medicine in October.

Thirty six of 39 children (92 percent) achieved a complete response (CR) after receiving an infusion of the modified cells. After a median follow-up of six months, more than two-thirds (70 percent) of children who responded remained in remission and 75 percent were alive, including the first patient to receive the therapy, in the spring of 2012. These results were achieved with only 3 of the patients going on to receive stem cell transplant while in remission.

All pediatric patients who responded to the therapy experienced a cytokine release syndrome (CRS) within a few days after receiving their infusions a key indicator that the engineered cells have begun proliferating and killing tumor cells in the body, but also a known potentially lethal type of toxicity. Patients who experience a CRS typically have varying degrees of flu-like symptoms, with high fevers, nausea, muscle pain, and sometimes, low blood pressure and breathing difficulties. Some patients require treatment with anti-cytokine agents and steroids to manage these symptoms.

The research team will also report the first results of a CTL019 study of patients with relapsed or refractory non-Hodgkin lymphomas (NHL) (Abstract #3087). In patients with follicular lymphoma (FL) or diffuse large B cell lymphoma (DLBCL) who received infusions of CTL019, assessments at three months after treatment revealed that all five FL patients (100 percent) and five out of 11 DLBCL patients (45 percent) responded to the therapy, including complete responses in four patients (80 percent) with FL and four patients (36 percent) with DLBCL. All patients who received infusions developed varying degrees of CRS. The longest complete response durations are ongoing, at 8.8 months for DLBCL and 7.4 months for FL; all other responses continue, as well. The findings will be presented by Jakub Svoboda, MD, an assistant professor of Medicine in the Abramson Cancer Center, on behalf of the Lymphoma Program under the leadership of the studys principal investigator, Stephen J. Schuster, MD, the Robert and Margarita Louis-Dreyfus Associate Professor of Chronic Lymphocytic Leukemia and Lymphoma.

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Cyanide killed hospital employee, official says

Posted: November 29, 2014 at 8:55 pm

PITTSBURGH (AP) A University of Pittsburgh Medical Center employee found dead at work earlier this month was killed by cyanide poisoning, but the medical examiner who made the determination Friday said it's too soon to know whether the death was a suicide, a homicide or an accident.

"There's only so much my office can do in terms of investigating how it got into her hands," Allegheny County Medical Examiner Dr. Karl Williams told the Associated Press. "We have to rely on the police to make that part of the investigation."

Co-workers reportedly found Nicole Kotchey, 34, of Ross Township, on the floor near her desk on Nov. 12, and she died about four hours later.

Pittsburgh police major crimes Lt. Daniel Herrmann said police received the medical examiner's findings Friday morning and were continuing to investigate.

UPMC spokeswoman Susan Manko said she was not authorized to comment.

Kotchey's death came a week after another former UPMC medical researcher, Dr. Robert Ferrante, 66, was convicted of first-degree murder in the April 2013 cyanide poisoning death of his wife, Dr. Autumn Klein. Klein, 41, was a neurologist also employed by UPMC. A jury agreed with prosecutors that Ferrante laced his wife's creatine energy drink with the poison, causing her to collapse at their home and die three days later.

Ferrante, a noted researcher into Lou Gehrig's disease, has denied killing his wife and plans to appeal. He acknowledged ordering cyanide for his lab, but said he needed it for research on stem cells that are used to replicate the way neurological cells die as a result of the disease.

Kotchey worked in a hospital lab, but UPMC officials have not commented on the nature of her employment since Williams first raised questions about her death two weeks ago.

Manko has previously said that the health network was cooperating with the police investigation.

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An Eclectic Path to Precision Medicine

Posted: November 25, 2014 at 5:55 pm

Stephen Emerson entered Haverford College with the aim of becoming an astronomer-mathematician. That is, until he met Ariel Loewy, a biology professor on the faculty who encouraged him to change his focus.

He said, All the asteroids and stars are going to be there for the next billion yearslet someone else worry about them, Emerson recalled. Why dont you study whatever science you want but think about applying it to cells and molecules and maybe someday even people?

Emerson spent a summer working in Loewys lab at the small liberal arts college and was hooked, graduating with a double major in chemistry and philosophy. Since then, his career trajectory has continued to take unexpected turns. He spent 13 years as the chief of hematology/oncology at the University of Pennsylvania, where he became a renowned expert in bone marrow stem cell biology, and his labs research in bone marrow stem cell transplantation led to many new medical therapies.

In 2006 he was asked to serve as president of his alma mater. After saying, Huh? but then talking it over, I said I would do it because I owed Haverford my career, Emerson said. I loved the college, and I loved serving as its president. But one wrenching stock market crash [in 2008] while running a small college was enough for me. In all, Emerson was at Haverford just under five years when the call came from Columbia and he returned to medicine full time.

As director of the Herbert Irving Comprehensive Cancer Center at Columbia University Medical Center, he is among a group of Columbia scientists homing in on innovative ways to treat diseases that target a patients genome, sometimes called personalized or precision medicine, which President Lee C. Bollinger has made a University-wide initiative.

And despite his 40 years as a scientist, Emerson remains an advocate of the liberal arts education that changed his life. Honestly, I use the philosophy just as much as I use the chemistry, he said.

Some people call it personalized medicine, which I think is a misnomer because in a sense weve always done personalized medicineits what a good doctor does. Whats new now is we know a lot more about the biology of diseases. And what were starting to realize is that what looked like the same disease among patients can actually be different diseases that just happen to look the same. Many of these illnesses are caused by random mutations in someones DNA within one cell, which can in turn change the function of a key protein, which makes a cell grow abnormally, as a cancer. Since the site of the mutation on the chromosome will be random, and different, for each cancer, every cancer will be fundamentally unique. One patients leukemia will not necessarily be the same as anothers, so the treatment for them should be different depending on the actual cause of the disease in their cells.

Theres a disease called chronic myelogenous leukemia, or CML, which is caused by one genetic change. Its very rare, but if you get this changeand bear in mind, its a single gene change, not twoit turns out that one medicine can control it. In fact, thats how precision medicine started, in a case where one gene was abnormal. In fact, you dont need that gene to work at all; its like the appendix of cells. You can poison this gene and from then on the patients CML cells will behave as if they were normal. The disease is still there, you didnt cure it, but the cells from then on will grow normally. Its amazing.

As you can imagine, the success with treating CML based on its single mutation led people to say, Why dont we do this for all cancers? It turns out there are very few cancers like that; usually its a combination of two or three genes gone wrong, and theyre all very different. But with DNA sequencing becoming more available, and with better computer power to analyze it, we can now perform a complete DNA sequence for any cancer, analyze it with the best computer power and brainpower available, and combine that sequencing information with what we already know about the genes involved. From there we can take a pretty educated guess as to what key gene is causing the problem, and then go to the shelf and try a medicine that might work in a clinical trial. Or put that abnormal gene into the tumor of a little mouse, try 50 different best guesses as to what medicine might work, pick the one thats most effective, and use that for the patient.

Yes. Oncology is the poster child for personalized medicine right now because you can have cancers that look the same under the microscope but are totally different in terms of what genes have been disrupted, which ones cause the cancer. So you have to use a treatment that specifically addresses that cause. But while this revolution has begun in cancer, it will spread to other parts of medicine in ways we cant foresee. Its only starting to be applied to other specialties.

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First Study to Convert Adult Human Cells to Hair-Follicle …

Posted: November 2, 2014 at 4:00 pm

PHILADELPHIA If the content of many a situation comedy, not to mention late-night TV advertisements, is to be believed, theres an epidemic of balding men, and an intense desire to fix their follicular deficiencies.

One potential approach to reversing hair loss uses stem cells to regenerate the missing or dying hair follicles. But it hasnt been possible to generate sufficient number of hair-follicle-generating stem cells until now.

Xiaowei George Xu, MD, PhD, associate professor of Pathology and Laboratory Medicine and Dermatology at the Perelman School of Medicine, University of Pennsylvania, and colleagues published in Nature Communications a method for converting adult cells into epithelial stem cells (EpSCs), the first time anyone has achieved this in either humans or mice.

The epithelial stem cells, when implanted into immunocompromised mice, regenerated the different cell types of human skin and hair follicles, and even produced structurally recognizable hair shaft, raising the possibility that they may eventually enable hair regeneration in people.

Xu and his team, which includes researchers from Penns departments of Dermatology and Biology, as well as the New Jersey Institute of Technology, started with human skin cells called dermal fibroblasts. By adding three genes, they converted those cells into induced pluripotent stem cells (iPSCs), which have the capability to differentiate into any cell types in the body. They then converted the iPS cells into epithelial stem cells, normally found at the bulge of hair follicles.

Starting with procedures other research teams had previously worked out to convert iPSCs into keratinocytes, Xus team demonstrated that by carefully controlling the timing of the growth factors the cells received, they could force the iPSCs to generate large numbers of epithelial stem cells. In the Xu study, the teams protocol succeeded in turning over 25% of the iPSCs into epithelial stem cells in 18 days. Those cells were then purified using the proteins they expressed on their surfaces.

Comparison of the gene expression patterns of the human iPSC-derived epithelial stem cells with epithelial stem cells obtained from human hair follicles showed that the team had succeeded in producing the cells they set out to make in the first place. When they mixed those cells with mouse follicular inductive dermal cells and grafted them onto the skin of immunodeficient mice, they produced functional human epidermis (the outermost layers of skin cells) and follicles structurally similar to human hair follicles.

This is the first time anyone has made scalable amounts of epithelial stem cells that are capable of generating the epithelial component of hair follicles, Xu says. And those cells have many potential applications, he adds, including wound healing, cosmetics, and hair regeneration.

That said, iPSC-derived epithelial stem cells are not yet ready for use in human subjects, Xu adds. First, a hair follicle contains epithelial cells -- a cell type that lines the bodys vessels and cavities as well as a specific kind of adult stem cell called dermal papillae. Xu and his team mixed iPSC-derived EpSCs and mouse dermal cells to generate hair follicles to achieve the growth of the follicles.

When a person loses hair, they lose both types of cells. Xu explains. We have solved one major problem, the epithelial component of the hair follicle. We need to figure out a way to also make new dermal papillae cells, and no one has figured that part out yet.

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Stem Cell and Xenograft Core: Biomedical Research Core …

Posted: October 27, 2014 at 3:57 pm

Core Description:The Stem Cell and Xenograft Core (SCXC) is a comprehensive resource laboratory that integrates a viable tissue bank of normal human hematopoietic cells and hematopoietic malignancies with a full range of xenograft services.

The SCXC is committed to facilitating and promoting translational research involving viable primary human hematopoietic tissues. Our core offers adult whole bone marrow from healthy donors and umbilical cord blood. Mononuclear and CD34+ cells from normal bone marrow and cord blood are available, and other cell fractions can be provided by arrangement. We maintain a large tissue bank of cells from hematopoietic malignancies including AML, ALL, CML, MDS and MPDs. All samples are fully annotated and frozen as viable cells. We also offer access to an immunomagnetic cell sorter (Miltneyi AutoMacs). Expertise in primary human hematopoietic stem/progenitor and leukemic cell culture and manipulation is available. For consultation or questions, please contact Martin Carroll.

The SCXC offers a wide variety of xenograft services from training to full-service experiments. The Core maintains a large breeding colony of immune-deficient (NSG) mice for users' xenograft studies. We also offer human immune system (CD34-transplanted) NSG mice for a wide variety of studies ranging from gene therapy to HIV. Experimental animals are housed in dedicated BSL-2 animal barrier space equipped for whole body irradiation and all necessary procedures and survival surgeries. Currently established xenograft models include normal human CD34 and leukemia engraftment, human iPS and ES-derived teratomas, human skin grafting, orthotopic human ovarian, hepatic and pancreatic tumor cell injections, renal capsule implantation. We also offer access to a dedicated optical/fluorescence (IVIS Spectrum) imaging system located within the Core's BSL-2 space. For consultation or questions, please contact Gwenn Danet-Desnoyers.

Core Director: Gwenn Danet-Desnoyers, Ph.D. 215-746-0181 gdanet@mail.med.upenn.edu

Technical Director of Xenograft: Xiaochuan Shan, M.D., Ph.D. 215-573-8581 shanx@mail.med.upenn.edu

Martin Carroll, M.D. Medical Director 215-573-5712 carroll2@mail.med.upenn.edu

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Immune Therapy Induces Remission for Many With a Tough-to-Treat Blood Cancer

Posted: October 18, 2014 at 9:53 am

By Amy Norton HealthDay Reporter

WEDNESDAY, Oct. 15, 2014 (HealthDay News) -- An experimental immune-system therapy can often lead to complete remission in leukemia patients who have run out of other options, a new study confirms.

Researchers found that 27 of 30 children and adults with advanced acute lymphoblastic leukemia (ALL) went into full remission after receiving genetically tweaked versions of their own immune system cells.

"Ninety percent of patients who had no options left went into complete remission. That's amazing," said senior researcher Dr. Stephan Grupp, of Children's Hospital of Philadelphia and the University of Pennsylvania.

However, seven patients who went into remission did eventually suffer a relapse, according to the study.

The findings, published Oct. 16 in the New England Journal of Medicine, confirm what smaller studies have suggested: The therapy offers hope to people with ALL that has repeatedly eluded standard treatments.

But while past studies have focused on adults, this study included mostly children.

"It shows the therapy can work just as well in children with ALL, and it's great to see that," said Dr. Michel Sadelain, a researcher at Memorial-Sloan Kettering Cancer Center in New York City who worked on those earlier studies.

But, both Grupp and Sadelain said ongoing studies will have to clarify the therapy's role in treating ALL.

ALL is a cancer of the blood and bone marrow that progresses quickly. It's more common in children than adults, but while children are often cured with chemotherapy, adults have a poorer outlook, Sadelain said.

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