Archive for April, 2017

Dr. Nabil Ahmed and Dr. Stephen Gottschalk discuss their investigation of a new treatment option for glioblastoma, the most common brain tumor in humans.

Glioblastoma is the most common brain tumor in humans and also one of the most difficult cancers to treat; patients with this type of cancer only survive about one year from time of diagnosis. Researchers at Baylor College of Medicine, Texas Childrens Cancer Center, and the Center for Cell and Gene Therapy at Baylor, Texas Childrens Hospital and Houston Methodist are investigating a new treatment option using modified T cells with anti-tumor properties with the goal of improving outcomes for patients with glioblastoma.

Their research focuses on engineered T cells that target the protein HER 2 expressed in low levels in glioblastoma cells. Results of a Phase 1 study published in the current issue of JAMA Oncology established the safety of these HER 2-specific, chimeric antigen receptor modified T cells (CAR T cells) when infused in to patients in increasing doses and, importantly, results also showed a clinical benefit to patients.

Our inability to effectively treat glioblastoma has been one of the failures of oncology, said Dr. Nabil Ahmed, associate professor of pediatrics at Baylor, Texas Childrens Cancer Center and the Center for Cell and Gene Therapy and first author of the paper. Glioblastoma is resistant to standard therapy, and it is difficult to remove all of the tumor cells through surgery without damaging the brain, so there is an urgent need for new and better treatment. Our work has focused on immune therapy, because it is very targeted and uses tumor-killing mechanisms that the cancers have not shown resistance to in the lab.

CAR T cells are T cells a type of immune cells involved in the defense against tumors that have been programmed to recognize and kill tumor cells carrying one specific antigen, in this case HER2, on the surface of cancer cells through an artificial molecule, the CAR, expressed on their surface.

The study included 17 pediatric and adult patients with HER 2-positive glioblastoma who received up to five escalating doses of the engineered T cells through intravenous infusions. Establishing the safety of the treatment is important, as other immunotherapy treatment approaches for solid tumors have resulted in significant side effects and toxicities for patients, Ahmed said.

First and foremost, the cells were safe. We did not see any life threatening side effects. Along with this we also saw measurable tumor responses, Ahmed said.

Median survival of patients who participated in the trial was 11.1 months post T cell infusion and 24.5 months from diagnosis. Three patients in the trial experienced no disease progression after more than two years of follow up.

With their promising results, Ahmed and his research colleagues, including Dr. Stephen Gottschalk, professor of pediatrics at Baylor, Texas Childrens Cancer Center and the Center for Cell and Gene Therapy and senior author of the paper, turn their focus to the next steps in the research.

In this phase 1 clinical trial we tested a particular modification that renders these cells specific for HER 2 and while the results have been encouraging, we are very interested to further engineer these cells, for example by making the T cells more effective after the infusion and by targeting not only HER2 but other molecules that are expressed on the cell surface of brain tumors, Gottschalk said.

The CAR T cells are produced in the cell manufacturing facility of the Center for Cell and Gene Therapy. Baylor is one of the few academic institutions that has such a facility, Gottschalk noted.

Others who contributed to this study include Vita Brawley, Meenakshi Hegde, Kevin Bielamowicz, Mamta Kalra, Daniel Landi, Catherine, Robertson, Tara Gray, Oumar Diouf, Amanda Wakefield, Alexia Ghazi, Claudia Gerken, Zhongzhen Yi, Aidin Ashoori, Meng-Fen Wu, Hao Liu, Cliona Rooney, Gianpietro Dotti, Andrea Gee, Jack Su, Yvonne Kew, David Baskin, Yi Jonathan Zhang, Pamela New, Bambi riley, Milica Stojakovic, John Hicks, Suzanne Powell, Malcolm Brenner, Helen Heslop, Roberta Grossman, and Winifred Wels, representing Baylor College of Medicine, Texas Childrens Hospital, Houston Methodist Hospital, and the Institute for Tumor Biology and Experimental Therapy in Frankfurt, Germany.

The study received funding from the Alliance for Cancer Gene Therapy, Cancer Prevention and Research Institute of Texas (RP110553), Alexs Lemonade Stand Pediatric Cancer Foundation, Stand Up to Cancer/St. Baldricks Pediatric Dream Team Translational Research Grant (SU2C-AACR-DT1113), the Clinical Research Center at Texas Childrens Hospital, the Dan L Duncan Institute for Clinical and Translational Research at Baylor, and by shared resources through Dan L Duncan Comprehensive Cancer Center Support Grant from the National Cancer Institute (P30CA125123).

Originally posted here:
Clinical trial results show benefit of brain cancer therapy - Baylor College of Medicine News (press release)

NEW YORK (GenomeWeb) Genetic testing for inherited retinal diseases is often not covered routinely by health insurance, yet a molecular diagnosis is required for enrollment in a number of clinical trials for new gene- or mutation-specific treatments. To assess the benefit of genetic testing data and to enable more patients to take advantage of it, the Foundation Fighting Blindness has been conducting a pilot research program, providing gene panel testing to 100 members of its My Retina Tracker patient registry free of charge.

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Inherited Retinal Disease Patients, Therapy Developers May Benefit From New Genetic Testing Program - GenomeWeb

Despite reports that linked Novartis to cutting back on its cell therapy work, the Swiss major said it is going full steam ahead for its CAR-T research as a potential approval for its candidate appears on the horizon.

In August of last year,Endpointsbroke the news thatthe Big Pharma was integratingits once standalone gene and cell therapy unit into the company. Most employees would beredeployed, but around 120 potentially face job losses.

This unit was doing work on the next new cancer class CAR-T, but some speculated that the disbanding of this unit meant that Novartis was looking to take a step back from research in this area.

Talk of its death was, however, greatly exaggerated according the the company. It saidits leading CAR-T candidate, CTL019 (tisagenlecleucel), now has two FDA breakthrough tags in two blood cancers, and was given a priority review for the med by the agency at the end of March.

The company is now in a race with biotech Kite Pharma to be the first to market this new type of cancer therapy that has shown some stellar results in clinical trials (although it also has revealedsome serious side effects from other companies, including Juno Therapeutics).

Speaking to FierceBiotech, Vas Narasimhan, M.D., global head of drug development and CMO at Novartis, said that there was a lot of interpretation last year regarding our reintegrating our cell and gene therapy unit into our R&D infrastructure, but was adamant that this did not create any difference in regards to our commitment in this space.

He said the integration of the unit into Novartis proper was primarily due to its success.

We had incubated the technology which came out of [its 2012 CAR-T deal] with Penn as a dedicated unit, and that was something that made sense: If you had immediately put that into the larger infrastructure of Novartis, it would have got lost, especially in the early stages when it was risky and it was unclear if this would be tractable," he said.

But Narasimhan said that as the unit became scaled, the parallel infrastructure scheme no longer made optimal sense.

To give this technology the best chance of succeeding, and in the most cost-effective way, we decided we should integrate [the unit] into our normal operations, and so CAR-T agents were then no different to an I-O agent, or anything else that we developed at Novartis, other than the fact that the process here is very important for the product," he said. We have the scale to work on these sorts of things quickly. So, the integration has allowed us to work on multiple programs in parallel, where I think the unit was focusing on just one or two programs at a time.

Now integrated, Narasimhan said its full steam ahead for CAR-T, and the immediate focus is to see through the two indications: r/r B-cell acute lymphoblastic leukemia (ALL) in pediatric and young adult patients and r/r diffuse large B-cell lymphoma (DLBCL) for tisagenlecleucel this year, as well as a combined ALL and DLCBL submission in Europe in 2017.

On the R&D side, he sees a three-pillar approach for CAR-T. The first pillar is to continue to be innovative on the manufacturing side of things, which is a tricky and cutting-edge science in itself for CAR-T. We need to continue to invest to optimize our manufacturing platforms, bringing it to more patients as we expand indications, he says.

The second is to continue to work in blood cancers, where results for Novartis and others have been the strongest. He said that they have programs in CAR-T in combination with other meds, as well as research in multiple myeloma, with additional blood cancer programs also slated to run on its CAR-T platform.

And the third pillar, which could turn out to be the toughest for all involved, is the move into solid tumors.

We have a few different constructs now and were moving into the clinic, Narasimhan explains. Our most advanced CAR-T is targeting ovarian cancer as well as a few other solid tumors.

Solid tumor research in the industry using CAR-Ts has not to date, however, seen a translation from the strong data coming out of blood cancers, and a lot of uncertainty remains as to whether, and how, CAR-T can treat these types of cancer.

I asked Narasimhan how confident he and Novartis were in holding up its third pillar.

For solid tumors, we really need to wait for the clinical data to make a judgement and see how tractable it is. We know in the solid tumor microenvironment there is a lot more going on, and the question of course will be combination therapies and what kind of I-O combinations might be required to see deep and durable responses in solid tumors," he said."Its too soon to say what our confidence level is in solid tumors, other than to say that we have targets that we are interested in and weve built those constructs and are taking them into the clinic.

One of the big questions surrounding Novartis CAR-T program is the outcome of its JULIET trial for tisagenlecleucel, a study looking at the meds ability in DLCBL (where rival Kite is also working on). The data are in, but Novartis isnt sharing yet.

Our plan with JULIET is to announce it at an upcoming medical event, and once we get acceptance at a congress, well disclose it. We just want to ensure that we dont undermine our ability to present the data to a scientific congress.

But Narasimhan did say that the breakthrough designation it got this week from the FDA hopefully gives some indication, of how the data have panned out.

Lastly, we spoke about the FDA, which is under pressure from all directions as the new administration looks to cut back on regulations and direct funding (with a proposed increase in user fees), and internally, with some questioning whether it has at times lowered its standards to allow drugs through, such as those from Sarepta and more recently Marathon, and if this signals a sea change for the agency.

Narasimhan defended the FDA, saying it should be strong, but that it is under-resourced for what they have to do. If you look at 21st Century Cures Act and all the new things they have to do, the funding isnt necessarily all there yet for those new requirements.

He also saidthat the science and the technology being put up for review is getting more and more complex, and there is public pressure with right to try and patient group pressures as well on the FDA and these are only increasing, so they have to find the right balance."

I would say, in general, that the FDA does an extraordinary job having worked with them for many years, including on the H1N1 pandemic, and through various cycles of innovation. But, I think they need more time and money to invest in their people, so that they are up to speed on the latest science so that they can make measured decisions.

He said that despitethe agency's outlier cases,generally they continue to be very rigorous, in their reviews.

He also said it was incumbent on both the agency and the industry to do more to speed up R&D and approvals, and be open to new, but scientifically-led, approaches that can help get new drugs to patients more quickly.

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Novartis drug development chief outlines CAR-T research commitment - FierceBiotech

SINGAPORE - It was a bone marrow match that defied the odds of one in 20,000 - not once, but twice.

Just months after his first match fell through when the patient withdrew from treatment, Mr Phil Tan, 27, was again identified as a suitable bone marrow donor for another patient.

His donation saved the life of eight-year-old Ryssa, who was diagnosed with a rare blood disease called Myelodysplastic Syndrome about three years ago. Both met for the first time on Wednesday (April 19). Ryssa received the transplant just before her seventh birthday.

Mr Tan was one of 22 Singaporeans who were honoured by Minister for Home Affairs and Law K. Shanmugam for saving the life of a patient through the donation of their bone marrow.

"We celebrate those who have come forward without expecting a benefit, other than making a huge difference in someone else's life. It is the real spirit of giving," said Mr Shanmugam, who is a patron of the Bone Marrow Donor Programme (BMDP).

Bone marrow or blood stem cell transplant is the best treatment option for patients diagnosed with blood diseases such as leukaemia and lymphoma.

At any one time, there are at least 50 patients waiting to find a matching donor.

Siblings of the patient are the first options for a donation, as they have a one in four chance of DNA compatibility for a transplant.

When that fails, the next option would be a match with a volunteer donor registered in the BMDP.

To date, more than 75,000 volunteers have joined the BMDP register, which records the genetic type of each person.

Since 2015, more than 50 Singaporeans have donated their bone marrow to patients in Singapore and overseas, including in the United States, Britain, Canada and France.

The BMDP, which was set up in 1993, aims to increase the size of the local donor register by another 50,000 by next year.

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8-year-old patient surprises her bone marrow donor at their first ... - The Straits Times

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Brain Organoid Created from Stem Cells | Technology Networks Technology Networks Technology Networks is an internationally recognised publisher that provides access to the latest scientific news, products, research, videos and posters. and more »

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Brain Organoid Created from Stem Cells | Technology Networks - Technology Networks

Salk scientists and collaborators have shed light on a long-standing question about what leads to variation in stem cells by comparing induced pluripotent stem cells (iPSCs) derived from identical twins. Even iPSCs made from the cells of twins, they found, have important differences, suggesting that not all variation between iPSC lines is rooted in genetics, since the twins have identical genes.

Because they can differentiate into almost any cell type in the body, stem cells have the potential to be used to create healthy cells to treat a number of diseases. But stem cells come in two varieties: embryonic stem cells (ESCs), which are isolated from embryos, and iPSCs, which are created in the lab from adult cells that are reprogrammed using mixtures of signaling molecules and are a promising tool for understanding disease and developing new treatments.

Although iPSCs resemble ESCs in most ways, scientists have found that iPSCs often have variations in their epigenetics methyl marks on the DNA that dictate when genes are expressed. These epigenetic markers arent the same between iPSCs and ESCs, or even between different lines of iPSCs. In the past, its been hard to determine what drives these differences.

When we reprogram cells, we see small differences when we compare them to stem cells that come from an embryo. We wanted to understand what types of differences are always there, what is causing them, and what they mean, says Juan Carlos Izpisua Belmonte, a professor in Salks Gene Expression Laboratory and co-senior author, with Kelly Frazer of the University of California, San Diego, on the new paper, which was published in Cell Stem Cell in April 2017. A better understanding of these differences will help researchers refine stem-cell based treatments for disease.

Izpisua Belmonte and Frazer, along with co-first authors of the paper Athanasia Panopoulos, formerly a postdoctoral fellow at Salk and now at the University of Notre Dame, and Erin Smith of UCSD, turned to twins to help sort it out.

Although identical twins have the same genes as each other, their epigenomesthe collection of methyl marks studding their DNAare different by the time they reach adulthood due in part to environmental factors. Reprogramming the skin cells of adult identical twins to their embryonic state eliminated most of these differences, the researchers found when they studied cells from three sets of twins. However, there were still key epigenetic differences between twins in terms of how the iPSCs compared to ESCs.

When the team looked more in depth at the spots of the genome where this variation between methyl marks tended to show up in twins, they found that they often fell near binding sites for a regulatory protein called MYC.

In the past, researchers had found lots of sites with variations in methylation status, but it was hard to figure out which of those sites had variation due to genetics, says Panopoulos. Here, we could focus more specifically on the sites we know have nothing to do with genetics. That new focus, she says, is what allowed them to home in on the MYC binding sites.

The MYC proteinwhich is one of the molecules used to reprogram iPSCs from adult cellslikely plays a role in dictating which sites in the genome are randomly methylated during the reprogramming process, the researchers hypothesized.

The twins enabled us to ask questions we couldnt ask before, says Panopoulos. Youre able to see what happens when you reprogram cells with identical genomes but divergent epigenomes, and figure out what is happening because of genetics, and what is happening due to other mechanisms.

The findings help scientists better understand the processes involved in reprogramming cells and the differences between iPSCs and ESCs, which has implications on future studies aiming to understand the specific causes and consequences of these changes, and the way iPSCs are being used for research and therapeutics.

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Identical twins; not-so-identical stem cells - ScienceBlog.com - ScienceBlog.com (blog)

MADISON, Wis.--(BUSINESS WIRE)--

Cellular Dynamics International (CDI), a FUJIFILM company and a leading developer and manufacturer of induced pluripotent stem cell-derived products, today announced it has signed a distribution agreement with STEMCELL Technologies, a world leader in iPS cell culture media.

This joint agreement with STEMCELL Technologies will make iPSC technology widely available to researchers worldwide, helping advance biological research leading to cellular therapies and drug discovery, said Dr. Bruce Novich, Division President-CNBD for FUJIFILM Holdings America Corporation and Executive Vice President and General Manager for CDI. We believe that STEMCELL Technologies, a leading developer, manufacturer and seller of stem cell related products, is an ideal partner for CDI, because their global sales and distribution infrastructure delivers to an established and an emerging customer base, which translates into faster access to and deeper penetration of CDIs leading edge technologies and products.

Under the terms of the agreement, STEMCELL Technologies will distribute CDIs iCell catalog of products in North America, Europe, and Singapore, with other countries under consideration. CDIs iCell products are differentiated human induced pluripotent stem cell (iPSC)-derived cells, which include cardiomyocytes, hepatocytes, and others, totaling up to 12 cell types.

STEMCELL Technologies is delighted for the opportunity to bring CDIs innovative products to the global research community. STEMCELL and CDI will work together on progressive solutions for the life science tools market. We look forward to a long and productive partnership with the shared goal of improving human health, said Dr. Allen Eaves, President and CEO of STEMCELL Technologies.

About Cellular Dynamics International:

Cellular Dynamics International (CDI), a FUJIFILM company, is a leading developer and supplier of human cells used in drug discovery, toxicity testing, and regenerative medicine applications. Leveraging technology that can be used to create induced pluripotent stem cells (iPSCs) and differentiated tissue-specific cells from any individual, CDI is committed to advancing life science research and transforming the therapeutic development process in order to fundamentally improve human health. The companys inventoried iCell products and donor-specific MyCell Products are available in the quantity, quality, purity, and reproducibility required for drug and cell therapy development. For more information please visit http://www.cellulardynamics.com.

About Fujifilm

FUJIFILM Holdings Corporation, Tokyo, Japan brings continuous innovation and leading-edge products to a broad spectrum of industries, including: healthcare, with medical systems, pharmaceuticals and cosmetics; graphic systems; highly functional materials, such as flat panel display materials; optical devices, such as broadcast and cinema lenses; digital imaging; and document products. These are based on a vast portfolio of chemical, mechanical, optical, electronic, software and production technologies. In the year ended March 31, 2016, the company had global revenues of $22.1 billion, at an exchange rate of 112.54 yen to the dollar. Fujifilm is committed to environmental stewardship and good corporate citizenship. For more information, please visit: http://www.fujifilmholdings.com.

About STEMCELL Technologies:

As Scientists Helping Scientists, STEMCELL Technologies is committed to providing high-quality cell culture media, cell isolation products, accessory tools and educational services for life science research. Driven by science and a passion for quality, STEMCELL provides over 2500 products to more than 90 countries worldwide. To learn more, visit http://www.stemcell.com.

All product and company names herein may be trademarks of their registered owners.

View source version on businesswire.com: http://www.businesswire.com/news/home/20170418005219/en/

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Cellular Dynamics International Signs Distribution Deal with STEMCELL Technologies - Yahoo Finance

My decision to get involved with developing gene therapy for neurological disorders came about because my own daughter, Ornella, 11, was born with a rare genetic condition known as Sanfilippo Syndrome, or mucopolysaccharidosis (MPS-IIIA). One in 70,000 babies is born with this life-limiting condition that results from a genetic mutation of a particular enzyme and leads to toxic build-up of heparan sulphate in brain tissue. Heparan sulphate is necessary for the normal function of the brain, but too much can damage tissue.

Heparan sulphate is necessary for the normal function of the brain, but too much can damage tissue.

At present, there is no cure, treatment is palliative and sufferers die in their early teens. Symptoms range from mild developmental delay and hyperactivity, which leads to sleep disturbance to more profound problems, such as paralysis, usually before the child reaches his or her 10th birthday.

Five years ago, Ornella became the first person in the world to have an experimental new drug treatment that we are developing at Lysogene, the biotech company I founded with Olivier Danos, a leading researcher into neurodegenerative disease. He had already been looking into this area of research and was keen to look at novel approaches.

She was part of a Phase 1/2 clinical trial that took place in 2011 and 2012 to gauge the safety of gene therapy using an adeno-associated viral vector known as AAVrH10 to deliver a genetically modified N-sulfoglucosamine sulfohydrolase enzyme directly into the brain cells. This enzyme effectively mops up the excess heparan sulphate and disposes of it. Animal studies had already shown it was safe and effective. The four children involved ranged in age from just under 3 years old to just under seven years old and they were dosed individually, one after the other, over three month intervals. The vector is injected into areas of the brain that show signs of damage during an operation under general anesthesia

AAVrH10 is a viral vector that is able to move into brain cells more efficiently where it delivers the drug. Other AAV vectors have been investigated by other research teams but with less effective uptake of gene therapy in the brain. All AAV vectors are very safe and stable so they are long-lasting. They also seem to cause no harmful effects in brain tissue.

All AAC vectors are very safe and stable so they are long-lasting.

This is a very elegant solution to a problem that is very localised in MPS-IIIA patients. The vector delivers the drug directly to the neurons where it is needed. It is also a once-only therapy because it fixes the faulty DNA of the enzyme permanently. Sadly, many children with genetic diseases need to have regular therapy, which often requires a hospital visit several times a month. This kind of gene therapy should mean that there is no need to repeat it more than once.

Im glad to say that the trial, which only included human patients with advanced disease, was a success and the results were published in Human Gene Therapy in 2014. As well as being safe, the therapy resulted in reduction in symptoms and a big increase in quality of life. Ornella showed tremendous improvement and started sleeping through the night something that had never happened from the day she was born until she was treated at six years old. We are hopeful that the therapy may also have the ability to extend life for patients with this disease. Certainly, this is what we observed in early animal studies. Potentially, it may even be curative.

Without such promising results, there was no way that I could consider continuing with the research programme. If I had not been convinced of its efficacy and safety, I would have called a halt after the initial trials.

We are now preparing to embark on a larger scale Phase 3 clinical trial with 20 patients in all. This will take place in 2018. If all goes well, we hope to market the drug to patients by 2020. I feel really blessed and proud to be involved in such an important project and to part of such a great team at Lysogene.

Karen Aiach is speaking at the RSM Medical Innovations summit on April 22nd. http://www.rsm.ac.uk

Karen Aiach is CEO and founder of Lysogene, a biotech company developing gene therapy for central nervous system disorders.

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Gene therapy for nervous system disorders - The Hippocratic Post - The Hippocratic Post (blog)

April 19, 2017 by Katherine Unger Baillie The Penn-led research team characterized the underlying mechanism that leads to the blinding Best disease; a loss of the microvilli that support and "feed" photoreceptor cells. The contrast can be seen above, with a normal retina on the right and an affected on on the left. Credit: University of Pennsylvania

Named for Friedrich Best, who characterized the disease in 1905, Best disease, also known as vitelliform macular dystrophy, affects children and young adults and can cause severe declines in central vision as patients age. The disease is one in a group of conditions known as bestrophinopathies, all linked to mutations in the BEST1 gene. This gene is expressed in the retinal pigment epithelium, or RPE, a layer of cells that undergirds and nourishes photoreceptor cells, the rods and cones responsible for vision.

Despite the century of work on bestrophinopathies and the identification of genetic mutations responsible for the conditions, no one had identified the underlying mechanism that led to the vision loss seen in Best disease until now.

Using an animal model of Best disease in combination with biochemical and optical assays, a team of researchers at the University of Pennsylvania has pinpointed a number of abnormalities that give rise to the impairments seen in the disease.

"The genetic cause of the disease has been known for 20 years, but no one had samples of patients at the stage when the disease starts," said Karina E. Guziewicz, research assistant professor of ophthalmology in Penn's School of Veterinary Medicine and lead author on the study. But "we were now able to pinpoint this early stage and find out what factors trigger the development of lesions."

The new information sets the team up for testing a gene therapy to treat the disease, as the researchers will be able to observe whether or not these structural and biochemical abnormalities have been corrected.

"Now that we understand what we're seeing, it allows us to judge the success of a particular therapy," said Gustavo D. Aguirre, professor of medical genetics and ophthalmology at Penn Vet.

Kathleen Boesze-Battaglia, a professor in the Department of Biochemistry in Penn's School of Dental Medicine, also contributed her expertise in lipid biochemistry and spectral analysis of lipid debris to the study, which was published in the journal Progress in Retinal and Eye Research, the top ranked journal in the eye-research field.

"Interestingly, the lipid debris accumulation is similar to cholesterol rich plaque formation, compounded by a complexity of vitamin A metabolism," said Boesze-Battaglia. "Alterations in lipid metabolism likely contribute to the secondary disease pathology in this model."

The main puzzle surrounding Best disease was why, despite the BEST1 gene being mutated in the RPE throughout the retina, vision loss struck the macula and fovea, the central areas of the retina responsible for reading and tasks requiring high-resolution vision, while seeming to spare the rest. Researchers had observed lesions in this area, but it was unknown why they developed.

In this study, the Penn-led team discovered that this predilection of the macula to develop lesions has to do with differences in the supporting structures of rods versus cones.

Rods, which make up more than 90 percent of photoreceptor cells in the retina and are responsible for dim-light vision, have a cluster of supporting structures known as RPE microvilli that cup the cell like stakes holding up a plant. In contrast, cones, the color-sensing photoreceptors that make up 3 to 5 percent of all photoreceptors but are overrepresented in the macula, are engulfed in a sheath of microvilli. In addition, cones are supported by an insoluble matrix.

Examining cross-sections of the fovea-like region in the canine macula of dogs affected with the canine equivalent of Best disease, the researchers found that the microvilli don't form and that the matrix is fragmented. The susceptibility of the macula is due to the fact that cones are the predominant cell type there and rely on the matrix for support and nutrient exchange.

"We were not expecting to find such dramatic structural abnormalities," Guziewicz said. "For a hundred years, this has been thought to be a disease of the RPE, but we have now identified this as a disease of the RPE-photoreceptor interface."

"The RPE provides transport of nutrients to the cones and engulfs the discarded part of cones and rods," said Aguirre. "When you lose the matrix, you lose the connection between those cells and the RPE and that leads to disease."

To determine if the same would be true in humans, the researchers looked at human induced pluripotent stem cell-derived RPE from Best disease patients and found similar signatures: microvilli numbers were decreased in length and density. These experiments were conducted in collaboration with David Gamm's laboratory from the McPherson Eye Research Institute at the University of Wisconsin-Madison.

Looking ahead, the research team would like to continue to probe the biochemical signals that lead to the improper development of the microvilli and matrix and push ahead with developing and testing a gene-therapy approach to treating bestrophinopathies.

"Knowing where the disruptions occur will allow us to develop proper outcome measures for a gene therapy, which is in the works," said Guziewicz.

Explore further: Fighting blindness: Scientists bring a key protein into focus

More information: Karina E. Guziewicz et al, Bestrophinopathy: An RPE-photoreceptor interface disease, Progress in Retinal and Eye Research (2017). DOI: 10.1016/j.preteyeres.2017.01.005

Scientists from the Florida campus of The Scripps Research Institute (TSRI) have discovered how a protein called 24 establishes proper vision. Their research helps explain why mutations in the gene encoding 24 lead ...

Newborns babies can be at risk of congenital blindness, presenting sight defects due to lesions or to genetic mutations in their genome. Among the latter, Leber Congenital Amaurosisor LCAis one of the most widespread ...

Silencing a gene called Nrl in mice prevents the loss of cells from degenerative diseases of the retina, according to a new study. The findings could lead to novel therapies for preventing vision loss from human diseases ...

Vitelliform macular dystrophy, also known as Best disease, is one of a group of vision-robbing conditions called bestrophinopathies that affect children and young adults. Caused by inherited mutations in the BEST1 gene, these ...

In humans, a tiny area in the center of the retina called the fovea is critically important to viewing fine details. Densely packed with cone photoreceptor cells, it is used while reading, driving and gazing at objects of ...

Three years ago, a team from the University of Pennsylvania announced that they had cured X-linked retinitis pigmentosa, a blinding retinal disease, in dogs. Now they've shown that they can cure the canine disease over the ...

Named for Friedrich Best, who characterized the disease in 1905, Best disease, also known as vitelliform macular dystrophy, affects children and young adults and can cause severe declines in central vision as patients age. ...

(HealthDay)Although Zika virus is most well-known for the devastating neurological damage it can cause in the womb, a new study reports that some babies infected with Zika also may have lifelong vision impairment.

A Stanford University research team has created a potentially powerful new way to fix damaged corneasa major source of vision problems and blindness.

The ability to distinguish objects in peripheral vision varies significantly between individuals, finds new research from UCL, Paris Descartes University and Dartmouth College, USA. For example, some people are better at ...

The new chemical composition of the legal high 'poppers' is linked to retinal damage at the back of the eye, finds a small study published online in the British Journal of Ophthalmology.

A light-sensing pigment found in everything from bacteria to vertebrates can be biochemically manipulated to reset itself, an important therapeutic advantage, according to new research out of Case Western Reserve University ...

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Team characterizes the underlying cause of a form of macular degeneration - Medical Xpress

Introduction

Ion channels play a key role in regulating electrical activity in excitable cells, and many additional roles in non-excitable tissues. They are important therapeutic targets in a range of indications including arrhythmia, hypertension, local anaesthesia, pain, stroke, epilepsy, depression, bipolar disorder, COPD, autoimmune disorders and diabetes. Not only are ion channels major drug targets, but they are also important indicators for drug safety. Indeed, many drugs withdrawn from the market due to cardiac related adverse effects have been shown to block the human ether-a-go-go (hERG) ion channel, which delays repolarization of the cardiac action potential and can result in a potentially fatal arrhythmia known as Torsades de Pointes (TdP).

Performing high throughput screening (HTS) or lead optimization against a target of interest, identification of a compounds target specificity by selectivity profiling, and checking for safety liabilities/risk assessment are all critical steps in the drug discovery process. Ion channel testing has evolved considerably in recent years with third generation automated patch clamping (APC) platforms addressing both voltage and ligand-gated channels at high throughput (HT) and at higher seal resistances. These newer HT APC platforms have allowed fee-for-service providers the possibility to offer cost-effective high quality outsourced ion channel primary screening and selectivity profiling for drug discovery, this is in addition to fluorescent-based assays and lower throughput conventional (manual patch) electrophysiology.

Read:automated patch-clamping trends

Most service providers that offer selectivity profiling have a large collection of stably transfected ion channel cell lines from which they assemble channel panels. Many service providers have also adapted and validated these cell lines for use on HT APC systems, some also sell their cell lines commercially. Providers of outsourced ion channel testing fall into 2 categories: 1) specialty CROs (i.e. providers with offerings limited to ion channel screening or ADME or animal models or clinical studies or any combination of these activities, but NOT the entire value chain); and 2) single-source or integrated CRO (i.e. a one-stop shop offering all activities).

In July 2016, HTStec undertook a market survey on outsourced ion channel testing mainly among research labs in pharma, biotech and academia. The survey was initiated by HTStec as part of its tracking of life science marketplaces and to update their previous outsourced ion channel testing trends report (published May 2013). The main objectives were to comprehensively document current use of and potential interest in outsourcing ion channel primary screening, selectivity profiling and safety liability testing. The survey also investigated access to stably transfected cell lines and future purchasing plans. The aim was to compile a reference document on outsourced ion channel testing, which could be directly compared with HTStecs previous 2013 report. This article contains selected findings from the HTStec market report, Outsourced Ion Channel Testing Trends 2016.

It is intended to provide the reader with a brief insight into recent market trends. It covers only 11 out of the 32 original questions detailed in the full report. The full published report should be consulted to view the entire dataset, details of the breakdown of the responses for each question, its segmentation and the estimates for the future.

Outsourced primary screening of ion channels Only a minority (32%) of survey respondents have outsourced the primary screening of ion channels to date and 18% of survey respondents had no foreseeable interest in outsourcing primary screening over the coming years. Where interested, the platforms for the primary screening of ion channels survey respondents most want to outsource to fee-for-service providers are given in Figure 1. This showed that automated patch-clamp (APC) was the most wanted platform (46%). This was followed by fluorescence-based assays (e.g. FLIPR/Hamamatsu FDSS) (19%), manual patch clamp (16%), multi-electrode array assays (MEA) (12%), and then membrane binding assays (6%). In contrast, the most used platform for primary screening of ion channels undertaken in- house is fluorescence-based assays.

Figure 1. Ion Channel Primary Screening Platform Respondents Want To Outsource At A Fee-For-Service Provider. Outsourced selectivity profiling of ion channelsMost (61%) survey respondents outsourced selectivity profiling of ion channels in 2016 and only a minority (9%) of survey respondents do not anticipate outsourcing over the coming years. The platforms for selectivity profiling of ion channels survey respondents most want to outsource to fee-for-service providers are presented in Figure 2. This showed that the preferred platform for selectivity profiling of ion channels to be accessed at service providers was automated patch-clamp (APC) (47%). This was followed by: manual patch clamp (28%); fluorescence-based assays (e.g. FLIPR/Hamamatsu FDSS) (11%); multi-electrode array assays (MEA) (8%); and membrane binding assays (6%).

Figure 2. Ion Channel Selectivity Profiling Platform Respondents Want To Use/Access At A Fee-For-Service Provider.

The stage in the drug discovery process where survey respondents most want outsourced selectivity profiling is shown in Figure 3. This showed that most (24%) respondents want to outsource selectivity profiling after hits-to-leads (lead optimization). This was followed by either: after secondary/counter screening or after primary screening/HTS (both 15%); after some initial selectivity profiling results generated in- house (14%); no fixed stage, we want to profile lead compounds from other therapeutic areas (12%); and then, when about to start IND enabling (3%).

Figure 3. When Respondent's Want To Outsource Selectivity Profiling.

The preferred way of selecting particular assays for outsourced ion channel selectivity profiling are given in Figure 4. This showed that respondents ranked selection by target as their most preferred way of choosing assays when deciding on ion channel selectivity profiling. This was followed by selection by family, selection by testing platform, and then selection by therapeutic area.

Figure 4. Preferred Way Of Selecting Assays For Ion Channel Selectivity Profiling.

The ion channel panels of most interest when considering outsourced selectivity profiling are presented in Figure 5. This showed that a cardiac channel panel was most wanted for selectivity profiling (26%). This was followed by a cardiovascular channel panel (16%), and then a pain-inflammation channel panel (13%). All others panel had less than 10% interest.

Figure 6. What Motivates Selection Of An Ion Channel Selectivity Profiling Provider.

Outsourced ion channel safety liability testing The aspect of ion channel safety liability testing survey respondents most want to outsource today are given in Figure 7. This showed that cardiac ion channel panel assays - automated patch clamp; hERG IC50 assay - non-GLP; and hERG ion channel assays were the most wanted assays (all with 41%). They were followed by hERG IC50 assay GLP (30%); and then cardiac ion channel panel assays - manual patch clamp and hERG screening assay (both 25%). Least wanted were stem cell-derived human cardiomyocytes assays (field potential, MEA, impedance, IcaICa, L activator assay).

Figure 7. Aspects Of Ion Channel Safety Liability Testing Outsourced. What motivates end-user selection of an ion channel safety liability provider is reported in Figure 8. This showed that cutting-edge gold standard assays were ranked as what most motivates their selection of an ion channel safety liability provider. This is very closely followed by comprehensive nature of the tests and assays offered, and then expertise in predicting cardiac risk and price. Ranked as least influential was Involvement in comprehensive in vitro proarrhythmia (CiPA) working groups.

Figure 8. What Motivates Selection Of An Ion Channel Safety Liability Provider. Spending on ion channel testing servicesHow survey respondents spend on outsourced ion channel testing services is broken down and presented in Figure 9. This showed that the biggest proportion (29%) of survey respondents 2016 outsourced ion channel testing budget was spent on selectivity profiling, this was followed by primary screening (25%); GLP Safety liability testing against hERG (16%); other safety testing (12%); non-GLP Safety liability testing against hERG (11%); and then assays fulfilling CiPA recommendations (7%).

Figure 9. Breakdown Of Current (2016) Outsourced Ion Channel Testing Budget.

The most used fee-for-service providers of ion channel testing services are reported in Figure 10. This showed that Eurofins (33%) was the most used fee-for-service provider of ion channel testing. It was very closely followed by Charles River (30%) and then more distantly by Thermo Fisher Scientific (9%), Aviva Biosciences (4%), SB Drug Discover (4%)y and Wuxi Pharmatech (4%). All other providers (totaling 17%) each had less than 4% share of use. This provider selection should not be confused as a true market share, it is not based on the actual $ value of services purchased, but on which provider survey respondents indicated they have most used over the past 12 months.

Figure 10. Most Used Fee-For-Service Providers Of Ion Channel Testing Services.

The importance (influence on provider selection) of an ion channel testing fee-for-service provider offering specific services is detailed in Figure 11. This showed the survey respondents rated most highly access to selectivity profiling (ion channels only) as the offering an ion channel service provider must provide to be worthy of consideration. This was followed by selectivity profiling (ion channels, GPCRs & kinases); and then assays fulfilling CiPA recommendations and GLP safety liability testing. Rated least wanted (not needed) was med chem around lead series (Figure 10).

Figure 11. Importance Of An Ion Channel Testing Provider Offering The Following Services.

Overall the use of outsourced ion channels testing services, particularly for selectivity profiling, is the preferred option for many drug discovery groups. However, the implementation of the CiPA initiative is expected to change the focus in nonclinical cardiac safety assessment by replacing the early hERG assessment (non-GLP) with an evaluation of compounds against multiple cardiac currents (APC); in silico modeling of cardiac action potentials; and the use of stem cell-derived human cardiomyocyte assays (e.g. MEA), as a multicellular test system that recapitulates the physiological properties of the human heart (e.g. with ECG-like field potentials). CiPA does however not displace GLP hERG or GLP in vivo ECG in a large animal which will still be needed for IND enabling, and are key services available at CROs. It will be interesting to see how outsourced providers of in channel testing services adapt to the changing regulatory requirements and the emergence of new in vitro screening technologies and approaches over the coming years.

DISCLAIMER: HTStec Limited has exercised due care in compiling and preparing these Selected Findings from its Report, which is based on information submitted by individuals in respondent companies. HTStec Limited has NOT verified the accuracy of this information, nor has it established respondents authority to disclose information to HTStec Limited. HTStec Limited expressly disclaims any and all warranties concerning these Selected Findings including any warranties of merchantability and/or fitness for any particular purpose, and warranties of performance, and any warranty that might otherwise arise from course of dealing or usage of trade. No warranty is either expressed or implied with respect to the use of these Selected Findings. Under no circumstances shall HTStec Limited be liable for incidental, special, indirect, direct or consequential damages or loss of profits, interruption of business, or related expenses that may arise from the use of these Selected Findings, including but not limited to those resulting from inaccuracy of the data therein.

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Outsourced Ion Channel Testing Trends - Technology Networks

The Husaini Haematology and Oncology Trust will soon provide facilities of bone marrow transplant and stem cell therapies at its newly established Blood Transfusion Centre and Thalassaemia Centre that was inaugurated by the city director health on Tuesday.

Speaking at the inaugural ceremony, Karachi director health Dr Muhammad Toufique urged the trust officials to share the data of patients undergoing blood transfusions, stem cell therapies and bone marrow transplant.

He said the data would help the Sindh government formulate a plan to establish more such facilities in the future. In addition to the blood screening and storage facilities, the centre is providing blood transfusion service to children suffering from genetic blood disorders as well as diagnostic services related to blood disorders.

This is a state-of-the-art blood transfusion and thalassaemia centre where bone marrow transplant and stem cell therapies would be started very soon, said a renowned haematologist associated with the Husaini Blood Bank, Dr Sarfraz Jaffery, at the inaugural ceremony of the blood transfusion and thalassaemia centre located at Qalandaria Chowk, North Nazimabad.

The head office of the Husaini Haematology and Oncology Trust is equipped with a diagnostic lab, blood bank having storage capacity of around 3,000 blood bags and blood transfusion centre for thalassaemic patients while its management is also planning to introduce bone marrow transplant and stem cell therapy services at the same facility in the near future.

Felicitating the trust officials, the city director health vowed to support them in their services. He said the government was also striving hard for provision of safe blood to thalassaemic children and other patients.

Dr Toufique hoped that institutions like Hussaini would come forward to support the government in establishing such centres in the province. Talking to journalists, the director health said steps were being taken to control the outbreak of Chikungunya in the city.

He said the health department was in contact with the municipal authorities to start fumigation in various areas of Karachi to eliminate the mosquitoes and prevent people from mosquito-borne diseases, including dengue and Malaria.

The Sindh government was planning to merge the Malaria and Dengue Prevention and Control Cells under one project director, who would be utilizing all the resources to eliminate the mosquitoes that were responsible for the deadly infectious diseases in the province, he added.

I would also urge people to take precautionary measures, prevent themselves and their children from mosquitoes by using repellents, improving sanitation conditions in their residential areas and adopt other preventive measures to protect themselves against the mosquitoes, he advised.

Earlier, speaking at a workshop on thalassaemia management held at the same place, noted haematologists of the country stressed the need for promoting the culture of prevention from diseases in the country.

They called for the implementation of laws regarding thalassaemia screening, saying that both the government and private sector could not treat the increasing number of thalassaemic patients.

Senior haematologist from Lahore, Prof Dr Jovaria Mannan, urged the doctors and researchers to use latest research methods in the field of haematology.

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Husaini trust plans to perform bone marrow transplants, stem cell therapy - The News International

Medical Xpress

Protein primes mouse stem cells to quickly repair injury, study finds ... Science Daily Like drag car racers revving their engines at the starting line, stem cells respond more quickly to injury when they've been previously primed with one dose of a ... Alerting stem cells to hurry up and heal - Medical XpressMedical Xpress all 3 news articles »

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Protein primes mouse stem cells to quickly repair injury, study finds ... - Science Daily

Third in an occasional series on how Harvard researchers are tackling the problematic issues of aging.

If only, wrote an ancient Japanese poet, when one heard that Old Age was coming one could bolt the door.

Science is working on it.

Aging is as much about the physical processes of repair and regeneration and their slow-motion failure as it is the passage of time. And scientists studying stem cell and regenerative biology are making progress understanding those processes, developing treatments for the many diseases whose risks increase as we get older, while at times seeming to draw close to a broader anti-aging breakthrough.

If stem cells offer potential solutions, theyre also part of the problem. Stem cells, which can differentiate into many cell types, are important parts of the bodys repair system, but lose regenerative potency as we age. In addition, their self-renewing ability allows the mutations that affect every cell to accumulate across cellular generations, and some of those mutations lead to disease.

We do think that stem cells are a key player in at least some of the manifestations of age, said Professor of Stem Cell and Regenerative Biology David Scadden, co-director of the Harvard Stem Cell Institute. The hypothesis is that stem cell function deteriorates with age, driving events we know occur with aging, like our limited ability to fully repair or regenerate healthy tissue following injury.

When it comes to aging, certain tissue types seem to lead the charge, according to Professor of Stem Cell and Regenerative Biology Lee Rubin, who directs the Harvard Stem Cell Institutes Therapeutic Screening Center. Particular tissues nerve cells appear to be one somehow signal to others that its time to age. This raises the prospect, Rubin said, that aging might be reversed by treating these key tissue categories, rather than designing individual treatments for the myriad tissue types that make up the body.

The process of aging involves all tissues in your body and, while different things go wrong in each tissue, they go wrong at basically the same rate, Rubin said. We can think of it as a process that is somehow coordinated, or there are fundamental processes in each tissue that play out.

In addition to key tissues, certain chemical pathways like insulin signaling seem to be able to control aging, said Rubin, whose work has received backing from the National Institute of Neurological Disorders and Stroke, as well as private foundations. The insulin signaling pathway is a chemical chain reaction in which the hormone insulin helps the body metabolize glucose. Reducing it has been shown to greatly extend life span in flies and worms, Rubin said. Also, signaling doesnt have to be reduced in all tissues.

If you just reduce it in neurons, the whole fly or worm lives longer, Rubin said. Certain key tissues in those organisms, if you selectively manipulate those tissues, have a positive effect on a number of processes in other tissues.

Because it circulates throughout the body, blood is an obvious place to look for controlling or signaling molecules that prompt or coordinate aging. A key carrier of oxygen and nutrients, blood is also rich with other compounds, some of which appear to play a role in decline linked to age.

Scadden described recent work done separately by Ben Ebert, a professor of medicine working at Harvard-affiliated Brigham and Womens Hospital, and Steve McCarroll, the Dorothy and Milton Flier Associate Professor of Biomedical Science and Genetics, that identified age-related changes in the blood that can increase the risk of diseases we dont typically think of as blood diseases.

Another tantalizing study, published in 2013, used the blood of a young mouse to rejuvenate the organs of an older one. In these parabiotic experiments, conducted by Professor of Stem Cell and Regenerative Biology Richard Lee and Forst Family Professor of Stem Cell and Regenerative Biology Amy Wagers, the circulatory systems of the two mice were joined, allowing the blood of the young to flow through the older ones body. The older mouse showed improvements in muscle tone and heart function. Later, similar experiments done by Rubin also showed improvements in neuronal health and brain functioning.

The young mouses fate depended on the age of the older mouse, Rubin said. If the latter was middle-aged, the young mouse appeared to be fine. If the older mouse was very old, however, the young mouse did worse.

Rubin said the experiments suggest that blood contains both positive and negative factors that influence aging. It may be, he said, that both are always present, but that positive factors outweigh negative in the young and that negative factors increase as we age.

Researchers have identified but not yet confirmed candidate blood factors for the rejuvenating effects. What seems not in doubt is the overall effect of the young blood on the old mouse. Interest is intense enough that a California company, Alkahest, has begun experiments giving Alzheimers patients plasma from young blood in hopes of improving cognition and brain function.

Even if that approach works, Rubin said, there would be practical hurdles to the widespread administration of young peoples blood plasma to older patients. But with an active compound identified, a drug could be made available to restore at least some cognitive function in Alzheimers patients.

In addition to the overall process of aging, researchers at the Harvard Stem Cell Institute, as well as across the University and its affiliated institutions, are investigating an array of diseases whose incidence increases sometimes dramatically with age.

The list includes several of the countrys top causes of death heart disease, stroke, diabetes, and cancer as well as rarer conditions such as the lethal neurodegenerative disorder amyotrophic lateral sclerosis (ALS).

Two decades ago, when stem cell research hit mainstream consciousness, many thought its greatest promise would be in stem cells ability to grow replacement parts: organs and tissues for damage caused by trauma or disease.

The stem cell revolution is still developing, Scadden said, but so far has taken a different form than many expected. The dream of harnessing stem cells to grow replacement hearts, livers, and kidneys remains, but potentially powerful uses have emerged in modeling disease for drug discovery and in targeting treatment for personalized medicine.

We thought stem cells would provide mostly replacement parts. I think thats clearly changed very dramatically. Now we think of them as contributing to our ability to make disease models for drug discovery.

David Scadden

Researchers have taken from the sick easily accessible cells, such as skin or blood, and reprogrammed them into the affected tissue type nerve cells in the case of ALS, which most commonly strikes between 55 and 75, according to the National Institutes of Health (NIH).

These tissues are used as models to study the disease and test interventions. Work on ALS in the lab of Professor of Stem Cell and Regenerative Biology Kevin Eggan has identified a drug approved for epilepsy that might be effective against ALS. This application is now entering clinical trials, in collaboration with Harvard-affiliated Massachusetts General Hospital.

In the end, stem cells might have their greatest impact as a drug-discovery tool, Scadden said.

Much of stem cell medicine is ultimately going to be medicine, he said. Even here, we thought stem cells would provide mostly replacement parts. I think thats clearly changed very dramatically. Now we think of them as contributing to our ability to make disease models for drug discovery.

Also evolving is knowledge of stem cell biology. Our previous understanding was that once embryonic stem cells differentiated into stem cells for muscle, blood, skin, and other tissue, those stem cells remained flexible enough to further develop into an array of different cells within the tissue, whenever needed.

Recent work on blood stem cells, however, indicates that this plasticity within a particular tissue type may be more limited than previously thought, Scadden said. Instead of armies of similarly plastic stem cells, it appears there is diversity within populations, with different stem cells having different capabilities.

If thats the case, Scadden said, problems might arise in part from the loss of some of these stem cell subpopulations, a scenario that could explain individual variation in aging. Getting old may be something like the endgame in chess, he said, when players are down to just a few pieces that dictate their ability to defend and attack.

If were graced and happen to have a queen and couple of bishops, were doing OK, said Scadden, whose work is largely funded through the NIH. But if we are left with pawns, we may lose resilience as we age.

Scaddens lab is using fluorescent tags to mark stem cells in different laboratory animals and then following them to see which ones do what work. It might be possible to boost populations of particularly potent players the queens to fight disease.

Were just at the beginning of this, Scadden said. I think that our sense of stem cells as this highly adaptable cell type may or may not be true. What we observe when we look at a population may not be the case with individuals.

The replacement parts scenario for stem cells hasnt gone away. One example is in the work of Harvard Stem Cell Institute co-director and Xander University Professor Douglas Melton, who has made significant progress growing replacement insulin-producing beta cells for treatment of diabetes.

Another is in Lees research. With support from the NIH, Lee is working to make heart muscle cells that can be used to repair damaged hearts.

Trials in this area have already begun, though with cells not genetically matched to the patient. In France, researchers are placing partially differentiated embryonic stem cells on the outside of the heart as a temporary aid to healing. Another trial, planned by researchers in Seattle, would inject fully differentiated heart muscle cells into a patient after a heart attack as a kind of very localized heart transplant.

Lees approach will take longer to develop. He wants to exploit the potential of stem cell biology to grow cells that are genetically matched to the patient. Researchers would reprogram cells taken from the patient into heart cells and, as in the Seattle experiment, inject them into damaged parts of the heart. The advantage of Lees approach is that because the cells would be genetically identical to the patient, he or she could avoid antirejection drugs for life.

What were thinking about is longer-term but more ambitious, Lee said. Avoiding immune suppression could change the way we think about things, because it opens the door to many decades of potential benefit.

Change has been a constant in Lees career, and he says theres no reason to think that will slow. Patient populations are older and more complex, disease profiles are changing, and the tools physicians have at their disposal are more powerful and more targeted.

Many of our patients today wouldnt be alive if not for the benefit of research advances, he said. Cardiology has completely changed in the last 25 years. If you think its not going to change even more in the next 25 years, youre probably wrong.

When Lee envisions the full potential of stem cell science, he sees treatments and replacement organs with the power to transform how we develop and grow old.

It may not be there for you and me, but for our children or their children, ultimately, regenerative biology and stem cell biology have that kind of potential, he said. We imagine a world where it doesnt matter what mutations or other things youre born with, because we can give you a good life.

Lees not guessing at future longevity. Hes not even sure extending life span beyond the current record, 122, is possible. Instead, he cites surveys that suggest that most Americans target 90 as their expectation for a long, healthy life.

Thats about a decade more than we get now in America, Lee said. We have work to do.

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Researchers study secrets of aging via stem cells - Harvard Gazette

Quinnipiac University President John Lahey is the recipient of the Stamford-based Alliance for Cancer Gene Therapys first Edward Netter Award for Business and Industry.

Quinnipiac University President John Lahey is the recipient of the Stamford-based Alliance for Cancer Gene Therapys first Edward Netter Award for Business and Industry.

Quinnipiac president to receive new award from Stamford nonprofit

STAMFORD Outgoing Quinnipiac University President John Lahey ranks at the top of the class for his community service, according to the leaders of one of Stamfords major nonprofits.

The Alliance for Cancer Gene Therapy, which supports the development of cell and gene therapies for cancer, will recognize Lahey on Wednesday at its anniversary gala at the Harvard Club in Manhattan with the first Edward Netter Award for Business and Industry.

ACGT officials said Lahey, an ACGT board member since 2004, embodies the qualities valued by the late ACGT co-founder Edward Netter: intellect, creativity, tenacity, curiosity and compassion.

Im deeply honored to receive the first-ever Edward Netter Award for Business and Industry, Lahey said. Edward was a true visionary. He and his wife Barbara have made such an impact in this area of research. Im delighted that Ive been able to be a part of ACGT and help see their vision of successfully treating cancer advance so quickly.

ACGT co-founder and honorary chairman Barbara Netter will present the award to Lahey.

Im so thrilled to honor Dr. John Lahey, Netter said in a statement. He has worked closely with Edward and me over the years to guide ACGT and to fund some of the most innovative and breakthrough cancer research in decades. I know Edward would be extremely proud to know what ACGT has been able to accomplish these years under the stewardship of John Lahey.

Lahey is the eighth president of Quinnipiac. After arriving at the Hamden institution in 1987, Lahey started a planning process that resulted in the growth of student enrollment from 2,000 to nearly 10,000. He also expanded Quinnipiac from a college to a university, which offers more than 100 programs in its nine schools and colleges.

Last week, he announced his intention to retire next year.

Among the other gala speakers, ACGT research fellow Dr. Robert Vonderheide, of the University of Pennsylvania will discuss breakthroughs using immunotherapy for the treatment of solid cancers. Doug Olson will talk about his experience as one of the first three patients treated in the cancer immunotherapy CAR-T clinical trial developed by ACGT research fellow and Scientific Advisory Council member Dr. Carl June.

Since its founding in 2001, ACGT has provided nearly $27 million in funding for cancer cell, gene and immunotherapy research in North America. ACGT officials said the nonprofit has supported the underlying science that has led to the founding of four companies in the final stages of bringing new treatments to patients: Novartis, Ziopharm, Juno Therapeutics and Turnstone Biologics.

pschott@scni.com; 203-964-2236; twitter: @paulschott

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Quinnipiac president to receive new award from Stamford nonprofit - The Advocate

As a practising specialist in internal medicine with 34 years of professional experience at the San Fernando General Hospital, and as a past president of the Council of the Medical Board of Trinidad and Tobago (2004-2010), I am calling on the Medical Association of this country, through its president, to set the record straight with regard to the utterances of the Minister of Health, Terrence Deyalsingh, who continues to insist that the hereditary haemoglobinopathy called beta thalassaemia major, which is what has been reported in the media that the child Shannen Luke suffers from, is not a life-threatening illness in this country.

His statement is patently untrue. The minister has stated in the Parliament that he has obtained five independent professional opinions from doctors in this country and Barbados in support of his position. Further, he has insisted that he has formed his opinion based on the recommendation of the board that advises the Children's Life Fund. I am also, therefore, calling on this board to publicly outline their position regarding the advice given to the minister as it pertains to this specific issue.

Having not been a party to the details of this child's diagnosis, I am guided solely by media reports. All of the reports that I have read concerning the issue of this unfortunate child's illness consistently attest to the fact that she has been diagnosed with beta thalassaemia major.

Beta thalassaemia major is a hereditary haemoglobinopathy that results in variable phenotypes depending on the extent of the patient's inability to synthesise non-alpha globin chains. Based on media reports, including an excerpt of a referring letter from the child's attending haematologist taken together with what the Minister of Health has said in the Parliament, one can reasonably conclude that this child suffers from beta (0) thalassaemia which refers to mutations of the beta-globin locus that result in the absence of production of beta globin. This is to be distinguished from beta thalassaemia intermedia and beta thalassaemia minor.

Only the last of these mutations can truly be considered to be non-life-threatening in the context of the present state of development of the health services in this country.

Beta (0) thalassaemia is a rare illness with a reported annual global incidence of just one in 100,000. Such children usually present with severe anaemia during the first two years of life. These children require repeated and regular transfusions of red cells which, in turn, if not managed properly with commensurate parenteral iron chelating therapy, e.g., deferoxamine (Desferal), administered through a continuous infusion pump device, will lead inevitably to a systemic iron overload with resultant organ failure.

Are infusion pumps routinely and reliably provided by Mr Deyalsingh's ministry today?

Can he say that these patients reliably receive a supply of deferoxamine?

How often are they transfused and how safe are these red cell transfusions? Inadequacy in any one of these aspects of treatment poses a threat to life!

Furthermore, poorly transfused individuals with this disease suffer from obligatory resultant conditions that reduce their life expectancy considerably. On the other hand, regular transfusions in the absence of addressing iron overload similarly lead to a range of pathologic entities, e.g., diabetes, hypothyroidism, hypopituitarism, adrenal insufficiency and most frequently cardiomyopathy.

How on earth then, can the Minister of Health insist that this illness, such as it is reported to be, is not life-threatening?

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Shannen's illnesslife-threatening or not? - Trinidad & Tobago Express

The association between endothelial dysfunction and late onset hypogonadism (LOH) in patients with vasculogenic erectile dysfunction (ED) is not yet well settled. Our objective was to assess the association between LOH and endothelial dysfunction in patients with vasculogenic ED. Throughout 2014-2015 a total of 90 men were enrolled in this cross-sectional observational study. Of them 60 patients with a clinical diagnosis of ED were further subdivided into two equal groups: patients with vasculogenic ED and LOH (A); patients with vasculogenic ED and euogonadal (B). Thirty age-matched men with no ED or hypogonadism were enrolled as control group (C). All patients were subjected to detailed medical and sexual history, total testosterone (TT), calculated free (FT) and bioavailable testosterone (BT), flow cytometric evaluation for endothelial progenitor cells (EPCs) (CD45negative/CD34positive/CD144positive) and endothelial microparticles (EMPs) (CD45negative/CD144positive/annexin V positive). The mean age SD of the three groups A, B and C were 51.311.1, 53.610.6 and 48.35years, respectively, with insignificant age differences (p=0.089). The diagnostic criteria of LOH were adapted according to European male aging study, 2010. The means of TT(ng/mL) were 2.320.21, 6.430.36 and 5.370.30 in groups A, B and C, respectively. There were highly significant differences between group A and groups B and C (p<0.001 for each). The means of EPCs were 0.430.070, 0.220.05 and 0.0320.013 in groups A, B and C, respectively. The means of EMPs were 0.150.029, 0.056 .013 and 0.0140.002 in groups A, B and C, respectively. There were significant differences between group C and groups A and B (p<0.05 for each). This study clearly demonstrated that there is a significant association between LOH and the higher expression of EPCs and EMPs in patients with vasculogenic ED.

Andrology. 2017 May [Epub]

Y A Omar, S E Younis, I Y Ismail, A I El-Sakka

Department of Urology, Suez Canal University, Ismailia, Egypt., Department of Clinical Pathology, Suez Canal University, Ismailia, Egypt.

PubMed http://www.ncbi.nlm.nih.gov/pubmed/28409902

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Testosterone level and endothelial dysfunction in patients with vasculogenic erectile dysfunction. - UroToday

A female specimen of a zebrafish (Danio rerio) breed with fantails

Cardiovascular disease is one of the leading causes of death in the world with approximately 30% of global mortality attributed to it.Cardiovascular disease conditions lead to damage of cardiac muscle cells resulting in defective heart function.

Stem cell therapy, though a relatively young science, is one of the upcoming treatment options for such diseases in the near future. In principle, stem cells from embryos can be made to differentiate into many functional cell types including heart cells, which can be effectively used to replace damaged cells in heart patients. To achieve this, scientists are constantly trying to understand the developmental process by which the heart is formed from various progenitors in a growing embryo. Once we understand this pathway at an organismal level, efforts can be made to use these stem cells for regenerative medicine.

A team of scientists led by Bruno Reversade from Singapore and Ian Scott from the University of Toronto have come together to study heart development in the Zebrafish model.

Zebrafish, scientifically called Danio rerio, is one of the powerful models for studying various organ functions. Although there are major structural differences between zebrafish and humans, there are strong similarities at the genetic and morphological levels. One of the biggest advantages of using zebrafish is that unlike mice, rats or monkeys, zebrafish embryos are transparent and hence provide a tractable system for visualizing these important developmental processes in situ.

During embryonic development, early heart development requires the activation of one of the important signaling pathways called Nodal or TGF pathway. Depending on the activation levels of Nodal, different cells become different stem cell types. Hence, there has to be a mechanism for fine-tuning of this signaling to produce these activity thresholds. Scientists from these two groups have recently identified the candidates involved in this fine-tuning.

Researchers recently identified a mutation, which leads to zebrafish with no heart at all. This suggests that this mutation somehow alters an early developmental process in heart formation. Interestingly, this gene encodes for a protein called Apelin receptor. So how does the Apelin receptor affect heart development? Scientists revealed that mutation in this receptor caused lower levels of Nodal signaling in mutant embryos as compared to the normal ones, thus failing to induce the formation of cardiac stem cells. When Nodal activity is artificially elevated in embryos that lack the Apelin receptor, they were able to develop hearts further confirming the role of Apelin receptor in this pathway.

A detailed understanding of this molecular cross-talk could help in the derivation of specific cell types from human embryonic stem cells for regenerative medicine, says Bruno Reversade, a human geneticist at the A*STAR Institute of Medical Biology, who co-led the investigation.

Further, this collaborative study showed that the Apelin receptor does not work in cells that produce or receive Nodal signals, suggesting that the Apelin receptor modulates Nodal signaling levels by acting in cells that lie between the cells that release Nodal signals and the cardiac progenitors.

In brief, this receptor functions as a distant regulator for fine-tuning the expression of the Nodal pathway during early stages of heart development ensuring proper cardiac development. One important area of future study is to determine whether modulating the levels of this receptor can prove useful for patients with various heart disorders.

Original article can be found here: https://elifesciences.org/content/5/e13758

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Scientists identify mechanisms of early heart development in Zebrafish - Biotechin.Asia

April 18, 2017 by Gian Galassi

A research team at University of Wisconsin School of Medicine and Public Health has treated its first patient in an innovative clinical trial using stem cells for the treatment of heart failure that develops after a heart attack.

The trial is taking place at University Hospital, one of three sites nationwide currently enrolling participants. The investigational CardiAMP therapy is designed to deliver a high dose of a patient's own bone-marrow cells directly to the point of cardiac injury to potentially stimulate the body's natural healing response.

The patient experience with the trial begins with a cell-potency screening test. Patients who qualify for therapy are scheduled for a bone-marrow aspiration. The bone marrow is then processed on-site and subsequently delivered directly to the damaged regions in a patient's heart in a minimally invasive procedure.

"Patients living with heart failure experience a variety of negative symptoms that can greatly impact their day-to-day life," said UW Health cardiologist Dr. Amish Raval, associate professor of medicine and one of the principal investigators for the trial. "By being at the forefront of research for this debilitating condition, we look forward to studying the potential of this cell therapy to impact a patient's exercise capacity and quality of life."

The primary outcome to be measured is the change in distance during a six-minute walk 12 months after the initial baseline measurement.

Heart failure commonly occurs after a heart attack, when the heart muscle is weakened and cannot pump enough blood to meet the body's needs for blood and oxygen. About 790,000 people in the U.S. have heart attacks each year. The number of adults living with heart failure increased from about 5.7 million (2009-2012) to about 6.5 million (2011-2014), and the number of adults diagnosed with heart failure is expected to dramatically rise by 46 percent by the year 2030, according to the American Heart Association (AHA).

The CardiAMP Heart Failure Trial is a phase III study of up to 260 patients at up to 40 centers nationwide. Phase III trials are conducted to measure effectiveness of the intervention, monitor side effects and gather information for future use of the procedure. Study subjects must be diagnosed with New York Heart Association (NYHA) Class II or III heart failure as a result of a previous heart attack.

Information about eligibility or enrollment in the trial is available at http://www.clinicaltrials.gov, or through a cardiologist.

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A team of biomedical engineering researchers, led by the University of Minnesota, has created a revolutionary 3D-bioprinted patch that can help heal scarred heart tissue after a heart attack. The discovery is a major step ...

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First participant treated in trial of stem-cell therapy for heart failure - Medical Xpress

Biomedical engineering Associate Professor Brenda Ogle (right) and Ph.Dstudent Molly Kupfer, with a mouse heart (Credit: Patrick OLeary, University of Minnesota)

One of the problems with heart attacks (as if there weren't enough already) is that when the heart heals afterwards, it grows scar tissue over the part of the heart that was damaged. That scar tissue never does become beating heart tissue, so it leaves the heart compromised for the rest of the patient's life. There may be hope, however, as scientists from the University of Minnesota have created a new patch that allows the heart to heal more completely.

First of all, yes, this has been done before. We have already seen experimental "heart patches" from places like the University of Tel Aviv, Brown University and MIT, which allow the heart to heal with a minimum of scar tissue growth.

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One of the things that makes this latest patch unique is the fact that it's 3D-bioprinted out of structural proteins native to the heart. It takes the form of a scaffolding-like matrix, which is subsequently seeded with cardiac cells derived from stem cells. The result is a patch of material, similar in structure and material to heart tissue, containing actual functioning heart cells as opposed to inert scar tissue.

In lab tests, one of the patches was placed on the heart of a mouse that had suffered a simulated heart attack. Within just four weeks, the scientists noted a "significant increase in functional capacity." The patch was ultimately absorbed by the body, so no additional surgeries were required to remove it after its job was done.

"We were quite surprised by how well it worked given the complexity of the heart," says associate professor Brenda Ogle, who is leading the research. "We were encouraged to see that the cells had aligned in the scaffold and showed a continuous wave of electrical signal that moved across the patch."

A larger patch is now in the works, which will be tested on a pig heart.

Other institutions involved in the study include the University of Wisconsin-Madison and University of Alabama-Birmingham. A paper on the research was recently published in the journal Circulation Research.

Source: University of Minnesota

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BROOMFIELD, Colo., April 17, 2017 /PRNewswire/ Interventional orthopedics in pain medicine practice was recently published by Elsevier as a chapter in Techniques in Regional Anesthesia and Pain Management. The chapter, authored by Regenexx founder Christopher J. Centeno, MD examines less invasive ways to treat orthopedic pain and injuries through autologous biologics, such as stem cells and platelet rich plasma (PRP), and the shift from surgical orthopedics to interventional orthopedics.

Interventional orthopedics utilizing advanced technologies, such as ultrasound and X-ray guidance, precise percutaneous injections of autologous biologics, and bone marrow concentrate, (BMC) expand nonsurgical options in the field of orthopedics. Citing the dramatic reduction in cardiac surgery rates since the adoption of the specialty interventional cardiology, the authors reveal, We are poised on the brink of the same change in orthopedic care. The authors also state, The field of autologous biologics has the potential to alter the playing field of orthopedic care by allowing percutaneous injections to replace the need for more invasive orthopedic surgeries.

The chapter covers three important tenets in the developing field that will allow Interventional Orthopedics to alter traditional orthopedic care in the future. First is the rapid expansion of injectates (material being injected), such as stem cells and PRP, that can help heal damaged tissue and that can effectively treat musculoskeletal tissues. Second is the precise image-guided placement of those injectates into those damaged tissues. And third is the development of new tools that will advance this regenerative-medicine technology. The chapter also highlights research that supports the use of bone marrow stem cells and the importance of education standards and organization, training, and retraining of physicians to meet these standards.

The full chapter Interventional orthopedics in pain medicine practice can be found online at http://www.sciencedirect.com/science/article/pii/S1084208X16300052.

Christopher J. Centeno, MD, is the CEO of Regenexx and an international expert and specialist in regenerative medicine and the clinical use of mesenchymal stem cells in orthopedics. Dr. Centeno maintains an active research-based practice and has multiple publications listed in the US National Library of Medicine.He has also served as editor-in-chief of a medical research journal dedicated to traumatic injury and is one of the few physicians in the world with extensive experience in the culture expansion of and clinical use of adult stem cells to treat orthopedic injuries.

MEDIA CONTACT Mark Testa 155014@email4pr.com (303) 885-9630

SOURCE Regenexx

Drue has been helping orthopedic companies overcome challenges since starting the firm in 2000. Direct orthopedic industry experience informs Drues perspective on who is best suited to take companies to the next level. A father of four, Drue strives to live a life of integrity and commitment to excellence. Prior to starting TDG, Drue was at Zimmer for 10 years as a Multiple President's Club Achieving Sales Representative before being recruited by Stryker as Branch Manager of the Arizona Branch where he built an award winning team in Reconstruction, Trauma & Spine.

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Regenexx Network Using Regenerative Medicine Technologies in Interventional Orthopedics to Treat Pain - OrthoSpineNews

Ottawa Citizen

Jonathan Pitre has unexpected surgery to control a blood infection Ottawa Citizen Last year, after his first stem-cell transplant, Pitre and his mother were thrilled when doctors discovered new white cells in his bloodstream. But their hopes were crushed when tests showed Pitre's own stem cells had recolonized his bone marrow, and ... and more »

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Jonathan Pitre has unexpected surgery to control a blood infection - Ottawa Citizen

A peculiar kind of cognitive dissonance grips most people who talk about death. On one hand, death is awful: It is the most tragic fate that can befall somebody, murderers are the lowest of the low, and the death of a loved one, even an elderly loved one who has lived a long life, clogs us with sadness.

On the other hand, any intimation that we might wish to, I dont know, abolish death is met with deep suspicion. Everyones time comes eventually, I have been told. Or: Itd be unnatural any other way. Even: But would you really want to live forever?

Yes, actually. Yes I would. I have wanted to live forever for as long as I can remember. My instinctive response when asked why is, well, why not? Life is a self-evident good to me. Justifying that seems absurd dont you like happiness? And love? And experiencing things? Dont you like being alive? Peoples tendency to reply, Well yes, but and trail off, looking vaguely concerned for my mental wellbeing, continues to mystify me.

Like large swathes of secular ethics, I suspect that this hesitancy is, in some sense, a hangover from Christianity. Christians, of course, might reasonably shun the idea of earthly immortality, but the basic impulse underlying Christianitys doctrine of life and death that one must endure an imperfect and pious life on Earth before rejoicing in the eternity of the empyrean is the same one that motivates me. I just have less faith that death brings anything other than an ineffable and everlasting nothingness.

Immortality is no longer, however, as niche an aspiration as it was even five, ten years ago. Tad Friend recently published a (highly recommended) piece in The New Yorker that documents the recent anti-aging buzz that has overcome Silicon Valley. Iconoclastic tech entrepreneur and venture capitalist Peter Thiel, ever ahead of the zeitgeist, wrote in 2009 that he stand[s] against the ideology of the inevitability of the death of every individual.

Since then, a steadily growing number of futurists have become interested in abolishing aging in one form or another. Donald Trump considered appointing Jim ONeill, a man who considers aging a disease to be overcome, to head the FDA, before, disappointingly, settling on the more establishment, Big Pharma-friendly Scott Gottlieb. Cryonics (freezing ones corpse in the hope that future technology may breathe life into it anew), once dismissed as mere science fiction, has slowly but surely gained popularity among Silicon Valleys elite. Futurist and AI researcher Eliezer Yudkowsky, a man unafraid of polemical positions (he once argued on utilitarian grounds that a single person being tortured for fifty years was preferable to a sufficiently large number of people getting dust specks in their eyes), wrote in a post on the website Less Wrong that If you dont sign up your kids for cryonics then you are a lousy parent. Thinking about cryonics reminds me of an H.P. Lovecraft line from the fictional text The Necronomicon, an esoteric book filled with secrets so vast in their cosmic implications that readers are sent insane merely by reading it. One of the few lines that Lovecraft reveals from the book goes like so: That is not dead which can eternal lie,/And with strange aeons even death may die. Strange aeons indeed, but perhaps ones not so far away.

I find this exhilarating. The world especially outside of Silicon Valley is starved of the kind of grand projects that can inspire a nation. Something like the space race would be nigh-unthinkable today (just ask Newt Gingrich). Even political projects like the New Deal or the Great Society, whatever you think of their outcomes, had an idealistic flavor to them that neither side of mainstream politics except, arguably, parts of Trumpism and Sanders-esque social democracy is really willing to embrace anymore. The prospect of seizing a truly fundamental part of human destiny the inevitability of death and forging it into a shape that befits our will is intoxicating in its grandiosity.

I think that one day the idea that death was so readily embraced, and that there was resistance against a project to eliminate it, will be incomprehensible to people. Life, and as much life as possible, will simply be taken for granted as a wonderful thing. Perhaps thats naive of me.

Tell you what, if Im still wrong in a thousand years, Ill write an apology column.

Contact Sam Wolfe at swolfe2 at stanford.edu.

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The technologist's stone - The Stanford Daily

Most children who identify as transgender are faking it, says gender clinic psychiatrist

Boys in tutus are not transgender

Should schools and parents rush to indulge the fantasies of children who claim they identify with the gender opposite their sex in particular, starting them on hormone treatments?

Absolutely not, according to psychiatrist Stephen Stathis, who runs a statewide gender service at Australias Lady Cilento Childrens Hospital.

Stathis told The Courier Mail that gender confusion in some girls he has treated seemed to stem from sexual abuse:

The girls say, If only I had been a male I wouldnt have been abused, Dr Stathis said.

Only a minority of the expected 180 children to visit his clinic this year will be diagnosed with gender dysphoria many are trying out being transgender and theyll drop it by the time they reach puberty, according to Stathis:

The psychiatrist has also seen transgender children so desperate to start puberty blockers then progress to irreversible hormone treatment they harm themselves.

Ive seen genital mutilation, some who try to cut off their penis, he said.

The thought of touching their genitals is so abhorrent they dont wash them and get infections.

A quick glance at the psychiatrists name on Twitter shows many people trying to assail him as transphobic or a quack.

Stathis told Brisbane Times earlier this year that his clinic traffic started to climb significantly about three years ago, when it was still his personal project and not yet funded at the childrens hospital:

To the point where in November last year, I had a two-year waiting list for me to see young people, Dr Stathis said.

And then the waiting list continued. The numbers were growing by about 25 per cent per quarter.

He criticized the intersection of cultural normswith the new interest in transgender identity, saying it drives kids who are not transgender to think they are:

Dr Stathis said girls could do stereotypical boys things and were called a tomboy.

But boys, you put on a pink tutu and youre off to see the doctor, he said.

Many of these young people were just gender variant, they didnt identify as the opposite gender, they just like to do things that the opposite gender likes to do, he said.

A little boy would say to me, Dr Steve, I like playing with dolls and I like fairies and I like dressing up, but Im still a boy. Thats not gender dysphoria.

And other kids simply see being transgender as a lifestyle choice, not something innate, he says.

Dont tell Lady Gaga.

Readthe stories.

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IVme is opening a new clinic this month in Marina City. View Full Caption

DNAinfo/Ted Cox; Provided

RIVER NORTH IV therapy: it's not just for hangovers anymore says the owner of a new facility opening in Marina City.

IVmeplans toopen a clinic by the end of the month at 346 N. State St., co-founder and medical director Dr. Jack Dybissaid.

The 2,600-square-foot clinic coming to the landmarkcorncob towers will follow a facility IVme opened three years ago in Old Town.Though thatclinic billed itself on hydration therapy and vitamin supplements that help with hangovers, Dybis said the new one at Marina City will offer a whole lot more.

"It's kind of grown up," he said.

New treatments include Botox,hormone therapy billed as a way to help with weight loss and other things, and other blood work that can help patients "have a greater say in their own health," Dybis said. Hydration therapies run from $89 to $199 per the clinic's website.

"Some people go to work out," Dybissaid. "Some people are now using IV as part of their health routine."

Treatments are by appointment and usually last between 45 minutes and an hour, Dybis said. He plans to keep the clinic open from 9 a.m. to 6 p.m. daily to start, but may expand the clinic's hours later.

IVme first opened as "Revive" in 2012 but later changed its name and moved to Old Town. The Marina City clinic will be the company's second. Dybis said he's already searching for a third location.

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IV Clinic Coming to Marina City: It's Not Just For Hangovers Anymore - DNAinfo

Photo courtesy of the University of Minnesota

MINNEAPOLIS/ST. PAUL A team of biomedical engineering researchers, led by the University of Minnesota, has created a revolutionary 3D-bioprinted patch that can help heal scarred heart tissue after a heart attack. The discovery is a major step forward in treating patients with tissue damage after a heart attack.

The research study was published Apr. 14 inCirculation Research, a journal published by the American Heart Association. Researchers have filed a patent on the discovery.

According to the American Heart Association, heart disease is the No. 1 cause of death in the U.S. killing more than 360,000 people a year. During a heart attack, a person loses blood flow to the heart muscle and that causes cells to die. Our bodies cant replace those heart muscle cells so the body forms scar tissue in that area of the heart, which puts the person at risk for compromised heart function and future heart failure.

In this study, researchers from the University of Minnesota-Twin Cities, University of Wisconsin-Madison, and University of Alabama-Birmingham used laser-based 3D-bioprinting techniques to incorporate stem cells derived from adult human heart cells on a matrix that began to grow and beat synchronously in a dish in the lab.

Watch a video of the cells beating on the patch.

Video credit:College of Science and Engineering, UMN

When the cell patch was placed on a mouse following a simulated heart attack, the researchers saw significant increase in functional capacity after just four weeks. Since the patch was made from cells and structural proteins native to the heart, it became part of the heart and absorbed into the body, requiring no further surgeries.

Related Article:3D-Printed Guide Helps Regrow Complex Nerves After Injury

This is a significant step forward in treating the No. 1 cause of death in the U.S., said Brenda Ogle, an associate professor of biomedical engineering at the University of Minnesota. We feel that we could scale this up to repair hearts of larger animals and possibly even humans within the next several years.

A team of biomedical engineering researchers has created a revolutionary 3D-bioprinted patch that can help heal scarred heart tissue after a heart attack. Two of the researchers involved are biomedical engineering associate professor Brenda Ogle (right) and PhD student Molly Kupfer (left).Photo credit: Patrick OLeary, University of MinnesotaOgle said that this research is different from previous research in that the patch is modeled after a digital, three-dimensional scan of the structural proteins of native heart tissue. The digital model is made into a physical structure by 3D printing with proteins native to the heart and further integrating cardiac cell types derived from stem cells. Only with 3D printing of this type can we achieve one micron resolution needed to mimic structures of native heart tissue.

We were quite surprised by how well it worked given the complexity of the heart, Ogle said. We were encouraged to see that the cells had aligned in the scaffold and showed a continuous wave of electrical signal that moved across the patch.

Ogle said they are already beginning the next step to develop a larger patch that they would test on a pig heart, which is similar in size to a human heart.

The research was funded by the National Science Foundation, National Institutes of Health, University of Minnesota Lillehei Heart Institute, and University of Minnesota Institute for Engineering in Medicine.

In addition to Ogle, other biomedical engineering researchers who were part of the team include Molly E. Kupfer, Jangwook P. Jung, Libang Yang, Patrick Zhang, and Brian T. Freeman from the University of Minnesota; Paul J. Campagnola, Yong Da Sie, Quyen Tran, and Visar Ajeti from the University of Wisconsin-Madison; and Jianyi Zhang, Ling Gao, and Vladimir G. Fast from the University of Alabama,

To read the full research paper entitled Myocardial Tissue Engineering With Cells Derived from Human Induced-Pluripotent Stem Cells and a Native-Like, High-Resolution, 3-Dimensionally Printed Scaffold, visit theCirculation Researchwebsite.

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3D-Printed Patch Can Help Mend a 'Broken' Heart - Lab Manager | News (press release) (blog)