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Newswise Researchers at UC Davis have found that the drug bortezomib effectively treats chronic graft-versus-host disease (GVHD), a common and debilitating side effect from allogeneic hematopoietic stem cell transplants. The trial showed that bortezomib provides better outcomes than existing treatments and does not impair the immune response against residual cancer cells, or the graft-versus-tumor effect (GVT).

Bortezomib helped a group of patients who desperately needed a treatment, having failed multiple different therapies, said UC Davis hematologist and associate professor Mehrdad Abedi, lead author on the paper. The drug fights chronic graft-versus host disease, and unlike other GVHD therapies such as steroid, cyclosporine or mycophenolate, it treats chronic GVHD without dampening the graft-versus-tumor effect, which can be critically important to help patients avoid relapse. In fact, because bortezomib is an anti-cancer drug, it potentially attacks cancer cells in its own right.

The trial results were published in October in the journal Blood.

Chronic GVHD strikes patients who have received stem cell transplants from donors, commonly called allogeneic transplants. Although the transplants are close matches, they are not identical, and donor cells can attack the recipient, damaging skin, lungs, kidneys and other organs, which can be life threatening.

Developed by Millennium Pharmaceuticals, bortezomib has been used to treat multiple myeloma, leukemia and lymphoma. The drug also has been studied against acute GVHD, making it a promising option against the chronic version of the disease.

The researchers first studied bortezomib in mice, in which the drug delivered excellent results.

The investigation, in collaboration with William Murphy, professor and acting chair of the Department of Dermatology and a co-senior author, found that bortezomib suppresses the donor immune cells that cause GVHD.

We then tested this concept in patients with chronic GVHD in collaboration with members of our bone marrow transplant team, hematologist-oncologists Carol Richman and Joseph Tuscano, Abedi said. Almost all the patients who tolerated and remained on the treatment responded. In some cases, individual responses were quite dramatic. We were able to stop their other immunosuppressive medications and keep the patients under control with just weekly injections of bortezomib.

Originally posted here:
Anti-Cancer Drug Effective Against Common Stem Cell Transplant Complication

Doctors at the University of Minnesotas childrens hospital traded in their white coats for ones embroidered with a new name.

With its $25 million donation, the Minnesota Masonic Charities became the Universitys largest donor, and in honor of the gift, the campuss pediatric hospital was renamed Tuesday as the University of Minnesota Masonic Childrens Hospital.

The donation will primarily go toward finding cures and treatments for childhood diseases, said Eric Neetenbeek, Minnesota Masonic Charities president and CEO.

The gift will specifically enhance patient and family experiences, as well as advance pediatric research on neurobehavioral development, rare and infectious disease, and stem cell therapy.

Dr. Joseph Neglia, the hospitals physician-in-chief, said he hopes the gift will create stronger relationships with pediatrics researchers across the University.

Really, to build new bridges is one big part of what Id like us to do, Neglia said. These gifts are vitally important for the hospital.

With the donation, Neglia said he also hopes to expand the hospitals existing research, like its work on correcting genetic defects in human cells and its pediatric medicine international programs in Kenya and Uganda.

Neetenbeek said the Masons donations, which total $125 million over the last 60 years, have been essential at a time when new health care research struggles to receive competitive funding from larger organizations like the National Institutes of Health.

There arent too many venture capitalists willing to go with untried businesses [and] ideas, he said. The same is true when youre looking at research into health care problems.

In the near future, the Masons will meet physicians at the childrens hospital to discuss which promising research projects to allocate the money toward, Neetenbeek said.

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$25M gift prompts University pediatric hospital name change

Why is there a need for donors of blood stem cells and bone marrow? In the majority of cases, if a person needs a blood stem cell transplant or bone marrow transplant they will need a donor. That donor has to be genetically similar to the patient. Why? Because if the donor cells are not a close match to the patient's cells, the donor cells will see the patient's cells as "foreigners." The donor cells will then tell the patient's immune system to attack the tissues in the patient's body. This can cause a serious complication called graft-versus-host disease. How is a stem cell "match" determined? To be a "match," the donor cells and the patient's cells need to be well-matched or compatible with each other. What follows is a technical explanation of how we go about finding donor cells that match those of the patient.

Human Leukocyte Antigens HLA are proteins on the surface of the white blood cells and other body tissues. If they match, the donor stem cells are less likely to trigger a defensive attack by the immune system on the patient's body tissues.

Some people have a family member who is an HLA match, such as a sister or brother. But many people do not have a relative who is a match. That means they must search for a donor. The donor can be someone who is unrelated to them. Or the donor stem cells can come from umbilical cord blood that has been frozen in a cord blood bank.

How can you become a donor?

If you would like to become a blood or marrow transplant (BMT) donor, your HLA typing can be done through a donor registry. The information about your tissue type is stored, along with your name and contact information, in a confidential donor database. If someone who needs a BMT matches your HLA type, you may be called to see if you want to come in for further testing. If testing shows that you are a match, you can donate blood stem cells or bone marrow to the patient in need.

You must be in good health to be a donor. This is true whether you are a family member or an unrelated donor. Age requirements will vary depending on if you are donating stem cells for a family member, or as an unrelated donor.

Why are more minority donors needed? Patients who need a BMT are more likely to match an unrelated donor who has the same ethnic or racial background. However, according to the National Marrow Donor Program, there are not enough potential minority donors in the Be The Match donor registry to meet the needs of minority patients. With thousands of patients in need of BMT, more donors of all ethnic backgrounds are needed. Learn more about the need for minority donors.

What about donating umbilical cord blood?In the past, umbilical cords were simply thrown away after a baby was born. Now we know that umbilical cord blood is a rich source of stem cells. In fact, researchers have found that there is less need for a perfect HLA tissue match when umbilical cord blood is used for a transplant.

Not all hospitals collect umbilical cord blood. If you would like to consider donating your baby's umbilical cord blood, check with your local hospital to see if that is possible. Find out even more about umbilical cord blood donation.

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Becoming a blood stem cell or bone marrow donor ...

An entire functional heart was created using a heart 'shell' and stem cells. This has been done in the rat & pig, with hopes of using a pig's heart 'shell' for people. Livers, kidneys, and pancreas (much harder) are in the works.

They "injected the empty sac with heart cells from newborn rats. Within days, the cells had multiplied to flesh out the heart, which began beating on its own.

"We've taken organs from cadavers, removed all the cells, put cells back in and been able to reanimate what was previously a dead organ," said molecular biologist Doris Taylor, director of the Center for Cardiovascular Repair at the University of Minnesota.

"What that means, we hope, is that one day if you need a new organ, we'll be able to take your cells, transplant them into this framework or scaffold and build you an organ that works for you," she said. Read more at: http://www.thestar.com/sciencetech/Sc...

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New heart built with stem cells - YouTube

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After battling blood cancer for 10 years, Stacy Erholtz has no signs of the disease, thanks to an experimental treatment that used an engineered version of the measles virus.

Now, a year after finishing her treatment, the 50-year-old mother of three is transitioning from patient to advocate, working with the Rochester, Minnesota-based Mayo Clinic to expand the tiny trial that saved her life.

"When I was first diagnosed, there was not a lot of options. We strung together 10 years of life with a disease that is typically done in three to five," said Erholtz, who had tumors on her forehead, color bone, sternum and spine from multiple myeloma before the last-ditch treatment. "I'm encouraged. I want people to join me in remission right now."

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Multiple myeloma is a type of blood cancer that affects the immune system, according to the Mayo Clinic. It can cause kidney-failure.htm" id="ramplink_kidney failure_" target="_blank">kidney failure, bone fractures and repeated infections.

After chemotherapy and stem cell transplants failed, Erholtz was accepted into the measles trial and given the highest possible dose of the engineered virus, which was designed to attack her cancer cells and leave her healthy cells alone, according to her physician, Dr. Stephen Russell.

"It's been adapted, so that it learned in the lab how to grow pretty efficiently on myeloma cells, "said Russell, who is in charge of the new trial. "It's lost the ability to cause harm on normal cells."

Original post:
Cancer Survivor Saved by Measles Virus Raises Funds for Expanded Trial

Researchers at the University of Minnesota have developed an animal research model for facioscapulohumeral muscular dystrophy (FSHD) to be used for muscle regeneration research as well as studies of the effectiveness of potential therapies for FSHD.

The research is published in the current edition of the journal Cell Reports.

There is no treatment for FSHD, which is thought by many to be the most common type of muscular dystrophy. FSHD is an unusual genetic disorder because, unlike most genetic diseases, it is not caused by the loss of a functional gene, but rather by the modification of an existing gene, through a genetic mutation. This mutation makes the gene more active so patients with FSHD express a protein, named DUX4, which interferes in an unknown way with muscle maintenance.

"We felt that an animal model would advance progress towards a cure for FSHD for two reasons," said Michael Kyba, Ph.D., lead researcher and associate professor in the Medical School at the University of Minnesota. "First, it would allow us to understand what DUX4 does in muscle to cause muscle loss, and second, it would provide a system in which efficacy of potential therapies could be evaluated before they are tested in humans."

The mouse model designed by Kyba and his team allows the disease-associated DUX4 protein to be produced when mice are treated with doxycycline. The amount of DUX4 can be controlled by varying the dose of doxycycline. Researchers expected the mice to be normal until they were treated with doxycycline, however even when DUX4 was in the "off" state, mice showed profound disease effects, some related to FSHD as well as additional effects not seen in FSHD patients.

"Nothing is black and white in biology," says Kyba. "No gene is truly off, and the off state in this case resulted in enough leaky DUX4 expression to kill the mice."

The team solved this problem by moving the gene to the X chromosome. Because females have two X chromosomes, only one of which is actively used in each cell, the female mice were healthy enough to enable the DUX4 mice to reproduce even though all of their male progeny with the DUX4 gene died. The fact that multiple levels of turning off the DUX4 gene were necessary to allow mice to survive showed that DUX4 is more toxic than researchers expected.

"We learned a lot with this animal model, but perhaps the most important finding was what we observed when we transplanted skeletal muscle stem cells," said Kyba.

The team could isolate muscle stem cells from the male mice before they died and when they transplanted them into muscle-damaged recipient mice, they found that the stem cells were able to regenerate new muscle. But when even low doses of doxycycline were given to the recipients to turn on DUX4 in the skeletal muscle stem cells, muscle regeneration was severely impaired. This suggested that a defect in skeletal muscle regeneration may contribute to muscle loss in FSHD. The finding also provides a very sensitive quantitative readout of DUX4 activity.

"This assay, in which we count new muscle fibers produced by transplanted DUX4-expressing muscle stem cells, will be very useful in testing therapeutics," says Kyba. "Drugs that target DUX4 should allow these transplanted DUX4-expressing muscle stem cells to make more new muscle fibers."

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Researchers find animal model for understudied type of muscular dystrophy

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Minnesota Stem Cell Therapy | Stem Cell Treatments

STEM CELL FREQUENTLY ASKEDQUESTIONS

THE PROMISE OF STEM CELLS

Stem cells can develop into different cell types. They may offer a renewable source of replacement cells to treat diseases, conditions, and disabilities.

Source:http://stemcells.nih.gov/Pages/Default.aspx

The University of Minnesota Stem Cell Institute (SCI), the first such institute in the United States, studies the basic biology of how stem cells work. The SCI faculty works in collaboration with many other areas in the University to lay the foundation for safe and effective treatments using stem cells. The SCI performs research and does not treat patients directly.

Cells are categorized as stem cells when they have the ability, as they divide and reproduce, to generate cells of several different types. The Stem Cell Institute works with many different types of stem cells. Stem cells can be pluripotent (able to generate any type of cell found in the body), multipotent (able to generate a limited number of different cell types), or oligopotent (able to generate two or more cell types within a specific tissue).

Induced pluripotent stem cells (iPSCs) are created by taking adult cells and reprogramming them to express genes that are active in stem cells until they again have the potential to develop into different types of cells. In this way, iPSCs made from adult skin cells could be used not only to repair the skin, but to repair a damaged heart muscle or liver. This technology is extensively in use at the University of Minnesota and is thought to be a highly promising option for many different patient therapies. An important potential advantage of using a patients own cells for treatment is that bodies do not reject their own cells. This reduces the risk and increases the possible effectiveness of using these cells.

Adult stem cells, found in the blood, bone marrow, muscle, and organs (for example, the brain, liver, and skin), are multipotent and part of the bodys system to maintain and repair itself. Their ability to generate different cell types is usually limited to the type of tissue in which they are found.

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Stem Cell FAQs - MED - Stem Cell Institute, University of ...

The Stem Cell Institute awarded four one-year grants for collaborative projects thatbringresearchers together across University departments.

James Dutton, PhD Anannya Banga PhDGenetics, Cell Biology and Development collaborating withMelanie Graham, MPH, PhDSurgery

Karen Echeverri, PhDGenetics, Cell Biology and Developmentcollaborating withMark Masino, PhDNeuroscience

Susan Keirstead, PhDIntegrative Biology & Physiology collaborating withRita Perlingeiro, PhDCardiology

Walter Low, PhDNeurosurgery collaborating withPerry Hackett, PhDGenetics, Cell Biology and Development

Read about Stem Cell Institute research fordiabetes.

Dr. Ann Parrawarded CTSI and Wings of Life grants for her work.

University of Wisconsin faculty and lawyer Alta Charowrites about the problems with medical "tourism" for unproven stem cell therapies. You may also want to read the "Patient Advisoryon Medical Tourism" put out by a group of reputable scientific organizations.

Learn about the vision for the University of Minnesota's Stem Cell Institute. DirectorJakub Tolar, MD, PhD, addressed the faculty, staff, and students of the SCI in January 2013.

Patients worldwideareeager to tap into the regenerative promise of stem cell therapies, but caution is needed. Legitimate researchcarefullyfocuses on patient safety and treatment effectiveness. It takes time and science to prove that a treatment really works.Read how scientists in Italy are fighting to protect patients from unproven and predatory treatment methodsin this Nature article "Taking a stand against pseudoscience."

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Home - MED - Stem Cell Institute, University of Minnesota

The mighty stem cell. Perhaps no other scientific frontier is as exciting and full of potential, and researchers from the University of Minnesota are on the forefront of understanding just what might be possible.

Our Stem Cell Institute, founded in 1999, was the first to be established in the United States. Our vision has always been to use stem cell biology to change the practice of medicine through discovery, education, and translation.

We explore the science of stem cell biology with the purposes of responding to the medical needs of today and educating the researchers of tomorrow. In doing so, were expanding the international understanding of whats possible using stem cells, and where the future of stem cell technology may take us.

Our pioneering research with stem cells has already led to new treatments and therapies for blood cancers, kidney diseases, and conditions that benefit from the regeneration of tissue.

Were also developing ways to use stem cells to repair heart muscle damaged by heart attacks, to repair the brain stem, and to fight diabetes through islet cell transplantation. Recently, Lillehei Heart Institute researchers have made incredible progress in combating muscular dystrophy using stem cells programmed to regenerate muscle.

Other recent highlights include the treatment of a rare skin disease using bone marrow and cord blood grafts, new technology for de-cellularizing and re-cellularizing organs, potential use of Natural Killer cells to treat cancer and were progressing in how we enable muscle differentiation from embryonic stem cells

The impact of stem cell research promises to reach far and wide. In fact, weve only just begun to unlock the possibilities.

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Stem cells | Health Sciences - University of Minnesota