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Gene therapy shows promise for severe combined immunodeficiency

Posted: October 9, 2014 at 2:45 pm

Date:

October 8, 2014

Source:

NIH/National Institute of Allergy and Infectious Diseases

Summary:

Gene therapy using a modified delivery system, or vector, can restore the immune systems of children with X-linked severe combined immunodeficiency (SCID-X1), a rare, life-threatening inherited condition that primarily affects boys, researchers have discovered.

Researchers have found that gene therapy using a modified delivery system, or vector, can restore the immune systems of children with X-linked severe combined immunodeficiency (SCID-X1), a rare, life-threatening inherited condition that primarily affects boys. Previous efforts to treat SCID-X1 with gene therapy were initially successful, but approximately one-quarter of the children developed leukemia two to five years after treatment. Results from a study partially funded by the National Institute of Allergy and Infectious Diseases (NIAID), a component of the National Institutes of Health (NIH), suggest that the new vector is equally effective at restoring immunity and may be safer than previous approaches.

In SCID-X1, mutations in a specific gene prevent the development of infection-fighting T cells. The standard therapy for SCID is transplantation of blood-forming stem cells, but some patients lack a suitable donor. In gene therapy, doctors remove stem cells from the patient's bone marrow, use a vector to insert a corrected gene and then return the corrected cells to the patient. Scientists suspect that the vectors used in earlier studies may have activated genes that control cell growth, contributing to leukemia.

In the current study, nine boys with SCID-X1 underwent gene therapy using a vector engineered by the study researchers. Seven boys developed functional T cells at levels comparable to those seen in previous studies and have remained healthy for one to three years after treatment. Analyses of the children's T cells suggest that the new vector causes fewer genomic changes that could be linked to leukemia. Researchers will continue to monitor the boys for leukemia development. Of the two other boys, one died of a pre-existing viral infection shortly after receiving the therapy, and one failed to develop corrected T cells and was given a stem cell transplant from an unrelated donor.

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Cure for Type 1 diabetes imminent after Harvard stem-cell breakthrough

Posted: October 9, 2014 at 2:43 pm

We are now just one pre-clinical step away from the finish line, said Prof Melton.

Asked about his childrens reaction he said: "I think like all kids, they always assumed that if I said I'd do this, I'd do it,

"It was gratifying to know that we can do something that we always thought was possible.

The stem cell-derived beta cells are presently undergoing trials in animal models, including non-human primates, where they are still producing insulin after several months, Prof Melton said.

Type 1 diabetes is an autoimmune condition that causes the pancreas to stop producing insulin - the hormone that regulates blood glucose levels.

If the amount of glucose in the blood is too high it can seriously damage the body's organs over time.

While diabetics can keep their glucose levels under general control by injecting insulin, that does not provide the fine tuning necessary to properly control metabolism, which can lead to devastating complications such as blindness or loss of limbs.

Around 10 per cent of all diabetes is Type 1, but it is the most common type of childhood diabetes. 29,000 youngsters suffer in Britain.

The team at Harvard used embryonic stem cells to produce human insulin-producing cells equivalent in almost every way to normally functioning cells in vast quantities.

Chris Mason, Professor of Regenerative Medicine, University College London, said it was potentially a major medical breakthrough.

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Arthritis of shoulder; results four years after stem cell therapy by Harry Adelson, N.D. – Video

Posted: October 9, 2014 at 2:40 pm


Arthritis of shoulder; results four years after stem cell therapy by Harry Adelson, N.D.
Heavy discusses his outcome four years out from his first bone marrow stem cell treatment for his arthritic shoulders and torn rotator cuffs by Harry Adelson, N.D. http://www.docereclinics.com.

By: Harry Adelson, N.D.

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Stem cell treatments surging into clinic

Posted: October 9, 2014 at 2:40 pm

Michael Scott, a ViaCyte vice president, holds the VC-01 device that holds progenitor cells that will mature to make insulin and other hormones.

More than ever before, stem cell therapies appear poised to transform medicine potentially curing heart disease, diabetes and paralyzing injuries, among other ailments.

But its also clear that such innovations will be very expensive.

How the government, insurers and patients will pay for what could be a flood of these new treatments drew the attention of more than 700 biomedical and health-care executives Tuesday at the 2014 Stem Cell Meeting on the Mesa.

The annual conference, held on La Jolla's Torrey Pines Mesa, will run through Thursday. It brings together the business and academic worlds of cell therapy, including but not limited to stem cell treatments.

In California alone, 131 clinical trials are taking place with stem cells, according to Clinicaltrials.gov, a government website that tracks clinical trials. Patients are being treated for conditions such as blindness from retinal diseases, HIV, leukemia, sickle cell disease, stroke and aging of skin.

The recent proliferation of clinical trials marks great progress toward the ultimate goal of getting new treatments to patients, said stem cell researcher Jeanne Loring, who directs the Center for Regenerative Medicine at The Scripps Research Institute in La Jolla.

Its been a sea change from last year, said Loring, who is working with some colleagues in planning their own stem cell trial to treat Parkinsons disease.

Theyre developing replacement neurons grown from artificial embryonic stem cells called induced pluripotent stem cells. The process begins with cells derived from the skin of patients to be treated.

Home-grown milestone

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Stem cell treatments surging into clinic

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Surgery 4 Pets Seeks Candidates for an Investigational Study of Stem Cells for Dogs with Arthritis

Posted: October 9, 2014 at 2:40 pm

Willamette Valley, Oregon and Southwest Washington (PRWEB) October 09, 2014

Local veterinary surgeon Dr. Scott Gustafson is seeking candidates to participate in an investigational study of donor stem cells for dogs with osteoarthritis. Dr. Gustafson has lectured nationally on stem cell therapy and has performed clinical stem cell therapy for seven years. The ultimate goal of this study is to determine if a single injection of donor stem cells into one or two arthritically affected joints can help reduce pain and inflammation in the treated joints.

Candidates for the current investigational study must be older than nine months, weigh more than five and a half pounds, have osteoarthritis of only one or two leg joints, have had pain or lameness for at least three months, and must not have cancer. Joints that will be included in the study and injected under anesthesia include hips, stifles, shoulders, and elbows. Dogs that may be considered must be in good health and undergo a diagnostic work up before qualifying for the study.

Dr. Gustafson and his team coordinate directly with your veterinarian to provide the most advanced veterinary care available. Surgery 4 Pets mobile surgery provides the highest level of care and surgical expertise to your pet, in the hospital of your primary care veterinarian.

In 2007 Dr. Gustafson was credentialed with Vet-Stem, Inc. in the use of Regenerative Veterinary Medicine for arthritis, ligament and tendon injuries, and joint. To date he has provided about 100 stem cell treatments for dogs.

For information about the study, please contact Michael Stewman at mwstew(at)gmail(dot)com

About Vet-Stem, Inc. Since its formation in 2002, Vet-Stem, Inc. has endeavored to improve the lives of animals through regenerative medicine. As the first company in the United States to provide an adipose-derived stem cell service to veterinarians for their patients, Vet-Stem pioneered the use of regenerative stem cells for horses, dogs, and cats. In 2004 the first horse was treated with Vet-Stem Regenerative Cell Therapy. Ten years later Vet-Stem celebrated its 10,000th animal treated. As animal advocates, veterinarians, veterinary technicians, and cell biologists, the team at Vet-Stem tasks themselves with the responsibility of discovering, refining, and bringing to market innovative medical therapies that utilize the bodys own healing and regenerative cells.

Contact: Sue Harman Senior Manager, Clinical Trials Vet-Stem, Inc. 12860 Danielson Court, Suite B Poway, CA 92064 858-748-2004

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Why Didn't They Win? 10 Huge Discoveries Without a Nobel Prize

Posted: October 9, 2014 at 3:57 am

The 2014 Nobel Prize in physiology or medicine has gone to three scientists who discovered brain cells that help us stay orientedour "inner GPS." Announced Monday, the award kicks off the annual salute to human accomplishment that is Nobel week, including Friday's announcement of the Nobel Peace Prize. (See: "Nobel Prize a Reminder of How the Brain Can Surprise Us.")

The ritual of Nobel speculation, particularly about who will win the three science prizes, got the editors at National Geographic thinking: What amazing discoveries haven't won? We asked our Phenomena science bloggers, science editors, and select contributors to pick their favorite advance or invention that was passed over.

Here are their ten picks for discoveries and inventions that haven't won a Nobel, but sorely deserve one.

The World Wide Web

When the National Geographic folks asked what discovery deserves the Nobel Prize but never won, my first instinct was to ask my followers on Twitter. After they gave me a few candidates, I Googled "Velcro" and "dark matter" and "embryonic stem cells" and read about these discoveries.

Then it occurred to me: What could be more deserving of the Nobel Prize than the invention I had so relied on to learn about inventions?

Beginning in the 1960s, researchers in the U.S. federal government created computer communication networks that would evolve into the Internet. But I'd give the Nobel to British computer scientist Tim Berners-Lee, who in 1989 proposed the idea for the World Wide Web and in 1990 created the first website (a page describing the Web).

The Web democratizes information, whether dumb videos of dancing cats or brave tweets from the Arab Spring. And information is power.

Virginia Hughes, Phenomena blog: Only Human

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Plant-based foods fight cancer

Posted: October 9, 2014 at 3:53 am

Pink is the official color of Breast Cancer Awareness Month, but the foods recommended for breast cancer prevention are green, white, red, yellow and brown.

They're all foods of the earth: fruits, vegetables, herbs, spices, nuts, seeds. October is, conveniently, also Vegetarian Awareness Month. This is not a matter of overbooking; health and plant-based food go together.

Behold, your cancer-fighting all-stars:

- Broccoli: A 2011 University of Michigan study found the plant sterols in broccoli reduce breast cancer stem cells.

- Coffee: Java junkies and cold-brew fiends, this one's for you. A 2011 Breast Cancer Research report shows the antioxidants in 2 cups a day protects cells from cancer growth.

- Parsley: This ubiquitous garnish is high in vitamin C and apigenin, another phytonutrient we never knew about before. Apigenin may be cancer's WMD, according to findings by the University of Missouri. Celebrate with some parsley-rich tabbouli.

- Pomegranate: In season now, these sweet-tart beauties can arrest cancer cell growth and destroy existing cancer cells, according to a University of California study. The fine print: You need to consume a lot of pomegranate, about 3 cups a day, for the goodness to kick in.

- Soy: Organic whole soy, the way they eat it in Asia -- tempeh, tofu, edamame and miso -- has received the blessing from the American Institute for Cancer Research. Choose that rather than the GMO soy isolates present in much American processed food.

- Turmeric: Gold dust. Antiseptic, anti-inflammatory and antioxidant, this wonderful warming spice seems to inhibit or erase cancer cell growth, according to a 2011 Cancer Prevention Research study.

- Walnuts: High in omega-3s, the same awesome anti-inflammatory amino acid in salmon. A joint study by the journal Nutrition and Cancer and the American Institute for Cancer Research found walnut consumption reduces breast cancer risk and retards cancer cell growth.

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Siamab Therapeutics Appoints Robert Mashal to Board of Directors

Posted: October 9, 2014 at 3:52 am

SOURCE: Siamab Therapeutics

Receives SBIR Grant Focused on Cancer Stem Cells; Research to Be Conducted in Collaboration With Massachusetts General Hospital

NEWTON, MA--(Marketwired - Oct 8, 2014) - Siamab Therapeutics, Inc., a biotechnology company developing cancer immunotherapies, today announced that Robert Mashal, President & CEO of NKT Therapeutics, has been appointed to the company's Board of Directors. In addition, the company has received a Small Business Innovation Research (SBIR) grant from the National Cancer Institute (NCI) for the development of novel therapeutic agents that target cancer stem cells (CSCs). The research will be conducted in collaboration with Bo Rueda, Ph.D, Director, Vincent Center for Reproductive Biology, Massachusetts General Hospital.

"We look forward to working with Dr. Rueda and his research team, and are honored that the NCI has awarded us this SBIR grant to develop therapies targeting cancer stem cells," said Jeff Behrens, Siamab's CEO. "We are thrilled to have Robert join our Board. His diverse background and extensive experience with emerging companies will be of great value as we continue to grow the company."

Dr. Mashal is President & CEO of NKT Therapeutics.Before joining NKT Therapeutics, Dr. Mashal was President of Alinea Pharmaceuticals.Prior to that, he was a partner at Boston Millenia Partners, a venture capital firm where he focused on investment opportunities in life sciences. He served as a Director of EpiGenesis Pharmaceuticals, Novalar Pharmaceuticals, GlycoFi, Sapphire Therapeutics, CoApt Systems, Protein Forest, and Cardiomems.He was previously a Program Executive with Vertex Pharmaceuticals, where he led cancer drug development strategy and oversaw pre-clinical/clinical development, marketing, regulatory and business activities. He was also a member of the Joint Research Committee for the $800 million Vertex-Novartis collaboration. Prior to that, Dr. Mashal served as a consultant at McKinsey & Company, and a faculty member and Attending Physician at Dana-Farber Cancer Institute, Brigham & Women's Hospital, and Harvard Medical School. Dr. Mashal is a diplomate in both internal medicine and oncology and a graduate of Johns Hopkins University. Dr. Mashal received his M.D. from Johns Hopkins University School of Medicine.

"I am excited to be joining Siamab at such a critical stage in the company's growth and look forward to providing support and guidance to the management team," said Robert Mashal. "Siamab has an impressive technology platform with tremendous potential for developing innovative cancer immunotherapies."

Under the SBIR grant recently awarded, Siamab will collaborate with Dr. Rueda's lab to explore the potential of Siamab's technology to create anti-glycan antibodies that target specific populations of cancer stem cells and explore the relationship between tumor associated carbohydrate antigens ("TACAs") and CSCs.

"Targeting CSCs represents a promising approach to the treatment of cancer," said Dr. Rueda. "I look forward to working with Siamab to explore the potential of their agents to target and inhibit the growth of CSCs."

Cancer stem cells (CSCs) are a subset of tumor cells that possess characteristics associated with normal stem cells. Specifically, they have the ability to self-renew, differentiate and generate the diverse cells that comprise the tumor. CSCs have been identified and isolated in several human cancer types, including breast, brain, colon, head and neck, leukemia, liver, ovarian, pancreas and prostate. These CSCs represent a small percentage of the tumor as a distinct population and cause relapse and metastasis by giving rise to new tumors. While chemotherapy and other conventional cancer therapies may be more effective at killing bulk tumor cells, CSCs are thought to escape and seed new tumor growth due to their quiescent and chemoresistant properties. Therefore, traditional therapies often cannot completely eradicate tumors or prevent cancer recurrence and progression to metastasis. With growing evidence supporting the role of CSCs in tumorigenesis, tumor heterogeneity, resistance to chemotherapeutic and radiation therapies, and the metastatic phenotype, the development of specific therapies that target CSCs holds promise for improving survival and quality of life for cancer patients, especially those with metastatic disease.

About Siamab Therapeutics, Inc. Siamab Therapeutics, Inc. is a biopharmaceutical company developing novel cancer immunotherapies.Siamab has developed a platform of technologies that enable the rapid discovery and development of therapeutic antibodies that bind to a novel class of carbohydrate antigens present on cancer cells, tumor associated carbohydrate antigens (TACAs). Siamab has developed a patented set of technologies to identify and precisely assay anti-TACA antibodies -- enabling rapid discovery and screening of candidate antibodies as well as characterization of binding epitopes.The company's lead program is in preclinical studies for the treatment of solid tumors. Siamab's corporate headquarters are in Newton, MA and laboratory facilities are in San Diego, CA. Learn more at http://www.siamab.com

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Brain Tumor Stem Cell Lab – Massachusetts General Hospital …

Posted: October 9, 2014 at 3:52 am

Research Projects

Characterizing glioblastoma stem cells

One of the main research focuses of the Brain Tumor Stem Cell Lab (Wakimoto Lab) is to characterize glioblastoma stem cells isolated from patient tumors. We are extensively establishing glioblastoma stem cells that grow in culture and testing their ability to form experimental brain tumors in mice. Our research shows that the capability of glioblastoma stem cells to migrate and invade through the brain varies greatly between patients. We are interested in identifying the molecular mechanisms that control tumor invasiveness. These studies are being conducted in close collaboration with the laboratories of Drs. Martuza, Rabkin, Curry, Cahill, Batchelor and Chi at Massachusetts General Hospital.

Therapeutic development

Glioblastoma stem cells are considered resistant to therapy such as chemotherapeutic drugs, but our research suggests that this is not always the case. Using glioblastoma stem cells as a unique model system that preserves patient-specific disease characteristics, we are evaluating the efficacy of therapeutic agents that include oncolytic herpes simplex virus -1 (in collaboration with the Martuza and Rabkin laboratories) and novel molecular targeting agents (in collaboration with the Batchelor and Chi Laboratories), and trying to discover molecular signatures of cells that help predict effectiveness for such treatment. Working closely with the Shah Laboratory of Radiology at Mass General, we are conducting research that harnesses normal stem cells to deliver therapeutic viruses to glioblastoma in the brain.

Glioblastoma recurrence

Cancer recurrence after surgery, radiation and chemotherapy is a serious issue that makes the management of glioblastoma difficult. We are trying to understand the role glioblastoma stem cells play in the relapse of the tumor. Our approach to this task is to establish glioblastoma stem cells from tumors both before and after therapy, and compare the two stem cells through analysis of their molecular and biological characteristics.

Collaboration

Taking advantage of the rich scientific environment at Mass General, the Brain Tumor Stem Cell Lab (Wakimoto Lab) works closely with the laboratories of Drs. Martuza, Rabkin, Curry, Cahill, Batchelor, Chi, Bernstein, Mohapatra, Iafrate and Shah. We are part of the Molecular Neurosurgery Lab and the Brain Tumor Research Center.

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Illinois (Stem Cell) – what-when-how In Depth …

Posted: October 9, 2014 at 3:51 am

There are a few common causes for this error code including problems with the individual script that may be executed upon request. Some of these are easier to spot and correct than others.

The server you are on runs applications in a very specific way in most cases. The server generally expects files and directories be owned by your specific user cPanel user. If you have made changes to the file ownership on your own through SSH please reset the Owner and Group appropriately.

The server you are on runs applications in a very specific way in most cases. The server generally expects files such as HTML, Images, and other media to have a permission mode of 644. The server also expects the permission mode on directories to be set to 755 in most cases.

(See the Section on Understanding Filesystem Permissions.)

Note: If the permissions are set to 000, please contact our support team using the ticket system. This may be related to an account level suspension as a result of abuse or a violation of our Terms of Service.

In the .htaccess file, there may be rules that are conflicting with each other or that are not allowing an IP address access to the site.

If you would like to check a specific rule in your .htaccess file you can comment that specific line in the .htaccess by adding # to the beginning of the line. You should always make a backup of this file before you start making changes.

For example, if the .htaccess looks like

Order deny,allow allow from all deny from 192.168.1.5 deny from 192.168.1.25

Then try something like this

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