StemCell Therapy

VIDEO Life Lessons: Next generation...

When Audrey Lapidus 10-month old son, Calvin, didnt reach normal milestones like rolling over or crawling, she knew something was wrong.

He was certainly different from our first child, said Lapidus, of Los Angeles. He had a lot of gastrointestinal issues and we were taking him to the doctor quite a bit.

Four specialists saw Calvin and batteries of tests proved inconclusive. Still, Lapidus persisted.

I was pushing for even more testing, and our geneticist at UCLA said, If you can wait one more month, were going to be launching a brand new test called exome sequencing, she said. We were lucky to be in the right place at the right time and get the information we did.

In 2012, Calvin Lapidus became the first patient to undergo exome sequencing at UCLA. He was subsequently diagnosed with a rare genetic condition known as Pitt-Hopkins Syndrome, which is most commonly characterized by developmental delays, possible breathing problems, seizures and gastrointestinal problems.

Though there is no cure for Pitt-Hopkins, finally having a diagnosis allowed Calvin to begin therapy.

The diagnosis gave us a point to move forward from, rather than just existing in that scary no-mans land where we knew nothing, Lapidus said.

Unfortunately, there are a lot of people living in that no-mans land, desperate for any type of answers to their medical conditions, said Dr. Stanley Nelson, professor of human genetics and pathology and laboratory medicine at the David Geffen School of Medicine at UCLA. Many families suffer for years without so much as a name for their condition.

What exome sequencing allows doctors to do is to analyze more than 20,000 genes at once, with one simple blood test.

In the past, genetic testing was done one gene at a time, which is time-consuming and expensive.

Rather than testing one sequential gene after another, exome sequencing saves time, money and effort, said Dr. Julian Martinez-Agosto, a pediatrician and researcher at the Resnick Neuropsychiatric Hospital at UCLA.

The exome consists of all the genomes exons, which are the coding portion of genes. Clinical exome sequencing is a test for identifying disease-causing DNA variants within the 1 percent of the genome which codes for proteins, the exons, or flanks the regions which code for proteins, called splice junctions.

To date, mutations in the protein-coding parts of genes accounts for nearly 85 percent of all mutations known to cause genetic diseases, so surveying just this portion of the genome is an efficient and powerful diagnostic tool. Exome sequencing can help detect rare disorders like spinocerebellar ataxia, which progressively diminishes a persons movements, and suggest the likelihood of more common conditions like autism spectrum disorder and epilepsy.

More than 4,000 adults and children have undergone exome testing at UCLA since 2012. Of difficult to solve cases, more than 30 percent are solved through this process, which is a dramatic improvement over prior technologies. Thus, Nelson and his team support wider use of genome-sequencing techniques and better insurance coverage, which would further benefit patients and resolve diagnostically difficult cases at much younger ages.

Since her sons diagnosis, Lapidus helped found the Pitt-Hopkins Syndrome Research Foundation. Having Calvins diagnosis gave us a roadmap of where to start, where to go and whats realistic as far as therapies and treatments, she said. None of that would have been possible without that test.

Next, experts at UCLA are testing the relative merits of broader whole genome sequencing to analyze all 6 billion bases that make up a persons genome. The team is exploring integration of this DNA sequencing with state-of-the-art RNA or gene expression analysis to improve the diagnostic rate.

The entire human genome was first sequenced in 1990 at a cost of $2.7 billion. Today, doctors can perform the same test at a tiny fraction of that cost, and believe that sequencing whole genomes of individuals could vastly improve disease diagnoses and medical care.

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Life Lessons: Next generation testing - WFMZ Allentown

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A comprehensive genomic analysis of Wilms tumor - the most common kidney cancer in children - found genetic mutations involving a large number of genes that fall into two major categories. These categories involve cellular processes that occur early in kidney development. The study, published in Nature Genetics, offers the possibility that targeting these processes, instead of single genes, may provide new opportunities for treatment of Wilms tumor.

"It is very difficult to therapeutically target over 40 genes that may be mutated in Wilms tumor," said senior author Elizabeth Perlman, MD, from Stanley Manne Children's Research Institute at Ann & Robert H. Lurie Children's Hospital of Chicago. "We discovered that many of these genetic mutations converge into two developmental pathways that lead to cancer. Early development of the kidney starts with rapid proliferation of undifferentiated cells. Within these cells, a signal triggers a switch to undergo differentiation into the normal cells of the kidney. In Wilms tumors, one set of mutations promotes abnormal and continued proliferation of the undifferentiated cells. A second set of mutations impacts the differentiation switch itself. Targeting these two different pathways in future studies might be more efficient than targeting individual gene mutations."

Perlman is the Head of the Department of Pathology and Laboratory Medicine at Lurie Children's and a Professor of Pathology at Northwestern University Feinberg School of Medicine. She is the Arthur C. King Professor of Pathology and Laboratory Medicine.

In the study, Perlman and colleagues in the Children's Oncology Group and the National Cancer Institute initially identified all genetic mutations in 117 Wilms tumor cases. Then they focused on a set of genetic mutations that occurred in more than one case and conducted a targeted analysis of these recurrent mutations in 651 Wilms tumors to validate the results. They found that the most common genes mutated in Wilms tumor were TP53, CTNNB1, DROSHA, WT1 and FAM123B.

In an unexpected finding, Perlman and colleagues also identified underlying germline mutations - or mutations in all the cells of the body - in at least 10 percent of Wilms tumor cases. "Our discovery of germline mutations in so many cases of Wilms tumor means that the children and family members of these patients may be at risk for tumor development," said Perlman.

Explore further: Researchers find new gene mutations for Wilms Tumor

More information: A Children's Oncology Group and TARGET initiative exploring the genetic landscape of Wilms tumor. Nature Genetics (2017). DOI: 10.1038/ng.3940

Journal reference: Nature Genetics

Provided by: Ann & Robert H. Lurie Children's Hospital of Chicago

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Comprehensive genomic analysis offers insights into causes of Wilms tumor development - Medical Xpress

We all have about 24,000 genes. How those genes are structured and interact can determine our current health and our future health.

Modern medicine includes specialists in this field called Clinical Geneticists. In todays TPR Lifeline, Bioscience-Medicine reporter Wendy Rigby talks to Baylor College of Medicines Scott McLean, MD, about his work at the Childrens Hospital of San Antonio.

Rigby: Dr. McLean, what is clinical genetics?

McLean: Clinical genetics is the medical specialty that uses genetic information to improve your genetic health or to understand the basis for a variety of medical conditions.

Those of us who have had children in Texas know that while youre still in the hospital, you get some genetic testing done. What is that called and what are you looking for?

We have newborn screening which is actually a blood test that is given to all babies 24 and 48 hours of age. The blood test involves collecting that blood on a piece of paper, filter paper, and sending that to the Texas State Department of Health Services in Austin where they do a series of tests.

This is the foot prick?

This is where you prick the heel. It seem awfully cruel. Babies cry. Parents dont like it. But its actually a wonderful test because it allows us to screen for over 50 conditions.

Give us some examples. What are some of the genetic conditions we might have heard of?

Well, the initial condition that was screened for in newborn screening in the United States was PKU which stands for Phenylketonuria. This is a condition that results in intellectual disability and seizures. We can change that outcome if we are able to identify the condition early enough and change the diet.

Lets say a child comes in to Childrens Hospital of San Antonio. Doctors are having trouble figuring out whats going on. Are you called in to consult?

Most of our patients that we see in the outpatient clinic are sent to us by consultation from physicians in the community or from nurseries, neonatal intensive care units. They range from situations such as multiple birth defects, to autism, intellectual disability, seizures, encephalopathy, blindness, deafness. Theres a whole gamut of reasons that folks come to see us.

When these children become grownups, does that information that youve learned about them help them out if theyre planning to have their own children in the future?

So when pediatric patients make the transition from pediatric care to adult care, its very common for information and ideas to get lost. And we certainly would hope that people remember that. Sometimes when we have identified a situation in a little baby, I tell the parents that I want them to put a sticky note on the last page of their baby book so that when they are showing the baby book to their childs fiance and they get to the last page, it reminds them you need to go back to see the geneticist because theres this genetic situation that you need to have a nice long chat about so that you can plan your family as carefully as possible.

Right. So the work youre doing today could help someone 30 years in the future.

Well, genetics is a very unique specialty in that regard because when we see a patient were not thinking about their next year of life or their next two years of life or the next month. We do think about that. But this is a lifelong diagnosis and a lifelong situation. So I often joke with my patients that Im going to try to put them on the 90-year plan. What we figure out now about their genetics is going to be helpful for them throughout their entire lifespan, at least up until 90 years. And then after that theyre on their own. But well get them to 90.

So its an exciting time to be in the field.

Very exciting. I think the era of gene therapy which for many people we thought was never going to happen, its very promising because we have new technologies that I think are going to allow for advances in that area.

Dr. Scott McLean with Baylor College of Medicine and the Childrens Hospital of San Antonio, thanks for the information.

Youre quite welcome.

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TPR Lifeline: Clinical Genetics Is A Growing Field - Texas Public Radio

Biology is a scientific branch from which emerged biotechnology, a multidisciplinary subject, which is growing fast in this modern world, having its impact on most of the activities in our day to day life. In simple terms, it is making products using living organisms. It is purely a research-oriented field, where the young minds who are interested in research can flourish. Biotechnology is a diverse field which benefits us in health, environment, industry, research, medical, agriculture and many more areas.

Although biotechnological impact was made in agricultural and industrial fields only in the early periods of the 20th century, it has its roots long back in BCs where beer, wine and bread were produced using single celled organisms via a process known as fermentation. Microorganisms were discovered later, who were the stepping stones to the modern biotechnological field. Karl Ereky, a Hungarian agricultural engineer, coined the term biotechnology. I can be a true biotechnologist only if I mention the two great names James Watson and Thomas Crick who discovered DNA in 1953, after which everyone came to know about the genetic material and how information passes at the molecular level. Later in 1970s, genes were manipulated by scientists, which led to the development of the wide area Genetic Engineering.

Green Biotechnology, Blue Biotechnology, White Biotechnology and Red Biotechnology are the fields which help us know more about the significance of biotechnology in agriculture, marine organisms, industrial products and medical processes. Improved varieties of crops are being produced which can better adapt to the existing environmental conditions. Flavr Savr tomato is a well known example. A better understanding of Blue Biotechnology helps us improve the seafood supply and safety. It is highly impossible to survive in the world without enzymes, the biological catalysts, which are produced through bioprocess technology utilising bioreactors and microorganisms. Thus, biotechnology has proved its importance in the industrial area too. In the medical field, biotechnology gains attention in gene therapy, production of humulin (recombinant insulin), recombinant vaccines etc. Drug designing and drug targeting has also gained too much interest in recent years with the advent of bioinformatics. Nanotechnology is also exploring the chances of developing and utilising nanoparticles for the benefit of mankind.

The future generation interested in this multidisciplinary field should plan a career accordingly from school days itself. It is always beneficial to opt for a biotech programme from the undergraduate level. After completing Masters in the respective branch, the young budding scientist can enter into the research field which leads to a doctoral degree that can be followed by post doctoral research. The job that can be taken up by biotechnologists is of a research scientist, and they can also be absorbed as a research associate, research assistants, laboratory technicians (food, water, environment labs, medical labs etc), teachers/lectures/professors, marketing personnel, business development officer, sales representative etc.

The door to the world of biotechnology is wide open. Lets enter it by contributing our the knowledge we have gained, which can improve and help sustain our life on earth.

Akhila RajanAsst Professor, Dept of Biotechnology, Waljat College of Applied Sciences

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From bio to biotechnology - Muscat Daily

VietNamNet Bridge Agricultural biotechnology is one of the key tools that can improve the yield and quality of agricultural products in a time of climate change, experts said.

Delegates meet at an APEC High-Level Policy Dialogue on Agricultural Biotechnology workshop in Can Tho on August 18-19. Photo: VNS

The experts were attending an APEC High-Level Policy Dialogue on Agricultural Biotechnology (HLPDAB) workshop in Can Tho on August 18-19.

Nguyen Thi Thanh Thuy, chair of the HLPDAB, and director general of the Ministry of Agriculture and Rural Developments Department of Science, Technology and Environment, said that Viet Nam, like many other economies, was facing multiple challenges due to climate change, including shrinking agricultural area and land degradation caused by urbanisation and saline intrusion caused by rising sea levels.

Many localities are suffering severe consequences of extreme weather, such as heavy rains, floods, flash floods, coastal shoreline erosion and salinity intrusion. These are threatening peoples livelihoods and agricultural production, she said.

Viet Nam has identified its response to climate change as one of the key tasks of its entire political system.

Strengthening international dialogue and co-operation in response to climate change is a priority set by the Government. This includes encouraging the use of new innovative technologies to promote sustainable agriculture, in which agricultural biotechnology is one of the key tools.

Speaking on the sidelines of the workshop, Pham Van Toan, associate professor at the Viet Nam Academy of Agricultural Sciences, said climate change, drought and saline intrusion were seriously affecting agricultural production.

With biotechnology, we can create drought-resistant and saline-resistant varieties to help the agricultural sector make products that meet market demand and food security, he said.

Biotechnology can help create new crop varieties that adapt to climate change and reduce greenhouse gas emissions, and especially helps improve the efficient usage of nutrients of crop plants, he said.

Globally, more than 400 genetic modification events (an insertion of a particular transgene) had been approved for food and animal feed, he said, adding that more than 20 events (maize and soybean) had been approved in Viet Nam.

Viet Nams biotechnology industry had begun development 20 years ago, but its achievements remained modest, he said.

Tomorrow, the annual meetings of the Ocean and Fisheries Working Group and the APEC High Level Policy Dialogue on Agricultural Biotechnology will be held.

Source: VNS

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Experts urge use of agricultural biotechnology as response to climate change - VietNamNet Bridge

Puma Biotechnology Inc (PBYI) has ended the week in the black, yielding positive results for the shares at they ticked 1.66%.In taking a look at recent performance, we can see that shares have moved -15.83% over the past 4-weeks, 98.14% over the past half year and 57.11% over the past full year.

Traders are keeping a keen eye on shares of Puma Biotechnology Inc (PBYI). The Average Directional Index or ADX may prove to be an important tool for trading and investing. The ADX is a technical indicator developed by J. Welles Wilder used to determine the strength of a trend. The ADX is often used along with the Plus Directional Indicator (+DI) and Minus Directional Indicator (-DI) to identify the direction of the trend. Presently, the 14-day ADX is resting at 39.85. Generally speaking, an ADX value from 0-25 would indicate an absent or weak trend. A value of 25-50 would indicate a strong trend. A value of 50-75 would signal a very strong trend, and a value of 75-100 would indicate an extremely strong trend.

Some investors may find the Williams Percent Range or Williams %R as a helpful technical indicator. Presently, Puma Biotechnology Inc (PBYI)s Williams Percent Range or 14 day Williams %R is resting at -62.25. Values can range from 0 to -100. A reading between -80 to -100 may be typically viewed as strong oversold territory. A value between 0 to -20 would represent a strong overbought condition. As a momentum indicator, the Williams R% may be used with other technicals to help define a specific trend.

When performing stock analysis, investors and traders may opt to view technical levels. Puma Biotechnology Inc (PBYI) presently has a 14-day Commodity Channel Index (CCI) of -39.59. Investors and traders may use this indicator to help spot price reversals, price extremes, and the strength of a trend. Many investors will use the CCI in conjunction with other indicators when evaluating a trade. The CCI may be used to spot if a stock is entering overbought (+100) and oversold (-100) territory.

Checking in on moving averages, the 200-day is at 51.87, the 50-day is 86.05, and the 7-day is sitting at 79.11. Moving averages may be used by investors and traders to shed some light on trading patterns for a specific stock. Moving averages can be used to help smooth information in order to provide a clearer picture of what is going on with the stock. Technical stock analysts may use a combination of different time periods in order to figure out the history of the equity and where it may be headed in the future. MAs can be calculated for any time period, but two very popular time frames are the 50-day and 200-day moving averages.

Shifting gears to the Relative Strength Index, the 14-day RSI is currently sitting at 42.18, the 7-day is 40.31, and the 3-day is currently at 41.06 for Puma Biotechnology Inc (PBYI). The Relative Strength Index (RSI) is a highly popular momentum indicator used for technical analysis. The RSI can help display whether the bulls or the bears are currently strongest in the market. The RSI may be used to help spot points of reversals more accurately. The RSI was developed by J. Welles Wilder. As a general rule, an RSI reading over 70 would signal overbought conditions. A reading under 30 would indicate oversold conditions. As always, the values may need to be adjusted based on the specific stock and market. RSI can also be a valuable tool for trying to spot larger market turns.

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Puma Biotechnology Inc (PBYI): A Look at Recent Performance - FLBC News

A labourer sifts wheat crop in a field. PHOTO: REUTERS

LAHORE:There is a dire need for increasing the agricultural yield to feed an increasing global population, food security experts emphasised on Saturday, highlighting biotechnology and its sub-fields as the key to increasing the productivity per acre.

Remedies derived from biotechnology could allow crop adaptation to biotic and abiotic stresses arising due to massive climate change at the global level, they said, adding technologies like genetic engineering could transform agricultural practices massively to make the sector more productive.

Food Security Policy: PARC finalises draft for cabinet review

Biotechnology is playing an important role in mitigating adverse impacts of climate change on agricultural output by reducing emission of greenhouse gases, said Professor of Biotechnology at FCC University Lahore Dr Kauser Malik.

He said the use of biofuels, for example, would help solve energy supply problems by providing renewable and cheap energy for farmers, apart from reducing the adverse impact of carbon dioxide emissions.

Carbon dioxide emissions are reduced by using traditional and genetically modified crops such as sugarcane, oilseed and rapeseed, he said, adding the need for tillage could also be reduced with the help of genetically modified crops.

We can modify plants through genetic engineering so that they absorb relatively more carbon from the atmosphere and convert it into oxygen. Soil fertility can also be increased by mixing microbes in the soil. In this context, modern environmental biotechnology has gained immense importance in coming up with innovative ways of increasing productivity, Malik said.

Experts claim that rainfall volatility, increasing drought conditions and drastic rises in temperatures all symptoms of climate change affect food production and are also responsible for pest, disease and weed outbreaks in crops.

Fast-paced climate change, experts claim, is not providing crops enough time to adjust to the changing environment, thereby stifling growth. According to an estimate, in 20 to 25 years, the world will get to a point where climatic conditions in most fertile areas will become too extreme for growing crops.

Pakistan Agriculture Research Council: Govt dept fires 500 employees

Malik said under the circumstances, increasing the area under production and improving productivity on the existing farmland were the only two choices to overcome food security challenges and adapt to changing climatic conditions.

Transgenic canola and soybean have been modified to be resistant to specific herbicides. Fungi, bacteria and virus-resistant GM cassava, potatoes, bananas and other crops are also being developed with some having already been commercialised while others are undergoing field trials, he added.

Published in The Express Tribune, August 20th, 2017.

LikeBusiness on Facebook, follow @TribuneBizon Twitter to stay informed and join in the conversation.

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Food security experts shift focus to biotechnology - The Express Tribune

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Ventilin inhaler - Ventolin salbutamol syrup dosage for adults - The Santa Clara

A US study has shown high dose Vitamin C halts the progression of blood cancer in mice by encouraging "faulty" stem cells in the bone marrow to die.

The findings, published in journal Cell, has raised the possibility of new new combination therapies for leukaemia patients carrying a specific gene mutation known as TET2.

"We're excited by the prospect that high-dose vitamin C might become a safe treatment for blood diseases caused by TET2-deficient leukemia stem cells, most likely in combination with other targeted therapies," said Dr Benjamin Neel, director of the Perlmutter Cancer Center.

The TET2 gene carries a protein that produces and matures stem cells, a process beneficial to blood cancer patients.

It's estimated TET2 mutations are found in 10 per cent of patients with acute myeloid leukemia (AML), 30 per cent of those with a form of pre-leukemia called myelodysplastic syndrome, and in nearly 50 per cent of patients with chronic myelomonocytic leukemia.

Previous research had suggested that TET2 could be activated by high-doses of Vitamin C.

"So we had the idea that high-dose Vitamin C be used as a therapy for some forms of Myelodysplastic syndrome and acute myeloid leukemia, particularly those forms who have mutations in this gene called TET2," said Dr Neel.

In the lab, scientists at the Perlmutter Cancer Center in New York added high doses of the Vitamin C to human leukemia cells carrying the TET2 mutations.

"We saw that that stops the growth," said pathologist Dr Iannis Aifantis.

A similar result was produced when tested on genetically engineered mice, according to the study.

It was also found the Vitamin C treatment had an effect on leukemic stem cells that resembled damage to their DNA, says first study author Luisa Cimmino.

"For this reason, we decided to combine Vitamin C with a PARP inhibitor, a drug type known to cause cancer cell death by blocking the repair of DNA damage, and already approved for treating certain patients with ovarian cancer," she said.

The combination had an enhanced effect on leukemia stem cells, further shifting them from self-renewal back toward maturity and cell death.

Scientists are now trying to apply the findings in clinic, with plans underway for a human clinical trial later this year.

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Vitamin C stops blood cancer in mice - NEWS.com.au

Although the traditional chemotherapy drug, cisplatin, is somewhat successful in its own right, researchers have discovered that when combined with a new experimental drug the results are astounding. In a recent University of Michigan study carried out using mice, researchers found that when this combination was used in mice it destroyed a rare form of salivary gland tumor and stopped it from recurring within a 300 day period.

This rare form of cancer in question is called *adenoid cystic carcinoma, or ACC for short. It affects around 3,000 to 4,000 people annually and is most commonly found in the salivary glands. Unfortunately, its one of those cancers that isnt usually detected until its at an advanced stage, is very resistant to therapy, and as of yet has no cure. Normally these type of tumors is treated with surgery and radiation. Chemotherapy is usually avoided as ACC is very slow-growing and chemotherapy is better used on rapidly growing tumors, confirmed Jacques Nor, a UM professor of dentistry, otolaryngology, and biomedical engineering, and principal investigator on the study.

The experimental drug used in the study is called MI-773, and when combined with cisplatin, is very effective at warding off cancer. It does this by preventing the interaction taking place that disarms the vital cancer-fighting protein, p53. As the researchers explain it, by blocking that interaction, ACC cancer cells become sensitized to cisplatin. This drug MI-773 prevents that interaction, so p53 can induce cell death, says Nor. In this study, when researchers activated p53 in mice with salivary gland cancer, the cancer stem cells died.

As part of the study, researchers carried out two different types of experiments in order to fully test how much the ACC tumors were reducing in size as well as their recurrence patterns. The first experiment involved treating tumors in mice with the combination of MI-773 and cisplatin. The results were that the tumors shrank considerably from around the size of an acorn to almost nothing. In the second experiment, researchers removed the acorn sized tumors surgically and followed it up with one months worth of MI-773 treatment.

We did not observe any recurrence in the mice that were treated with this drug after 300 days (about half of mouse life expectancy), and we observed about 62 percent recurrence in the control group that had only the surgery, said Nor. Its our belief that by combining conventional chemotherapy with MI-773, a drug that kills more cancer stem cells, we can have a more effective surgery or ablation. One slight drawback to the study is that it is based on an observational period of 300 days, whereas nearly half of all ACC tumors recur only after around 10 years. Its still early days for the drug combo in terms of being used on human patients but is still a good place to start nonetheless.

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New Experimental Drug Kills Cancer Cells When Combined With Chemotherapy - TrendinTech

Len Richard (left) and actor Terrance Howard on the set of "Empire" in 2015. View Full Caption

Provided by Len Richard

CHICAGO Len Richard used to fight in movie scenes as a stuntman, but now hes fighting for his life.

Diagnosed with liver disease in 2009, hes been placed on the liver and kidney transplant list to replace his rapidly failing organs. Hes opted to have stem cell replacement therapy in place of the transplant, but his insurance wont cover it, so hes raising $10,000 through YouCaring.

The 44-year-old Englewood native said hes desperate to live a healthy life and doesnt want to risk trying the transplant. Theres the fear that the new organs wont work for long and hell constantly get sick from the anti-rejection medications hell have to take, he said.

Once I understood how stem cell works and how it cures people, it was a no-brainer, Richard said. I rather do that than have someone's organs.

More than 5,000 liver transplants in the United States take place each year, according to the "Stem Cell Therapy for Liver Diseases," a review article published in the Journal of Stem Cell Research and Therapy. About 20,000 people are waiting for a transplant, but only 7,000 procedures are performed each year, and up to 1,500 patients die each year waiting.

Use of stem cells to cure liver diseases has been proved beneficial in most of the conditions, according to the article. Scientific literature reveals the role of stem cells in treatment and cure of various diseases like liver cirrhosis, end stage liver failure, genetic liver disease and also the liver cancer. The stem cells possess the ability to renew and multiply by them or stem cells possess special characteristics of regenerating themselves.

Besides being hospitalized twice in eighth grade for a high fever and an enlarged liver and spleen, Richard has lived a fairly healthy life, he said. He had no major health problems in high school or college.

As an adult, he worked behind the scenes in the control room at Channel 50, before moving to Los Angeles in 1998 for a similar television job. Thats when his life changed.

He was at a gym working out and got invited to train with a group who worked as stuntmen, he said.

A stunt coordinator saw me and said that I looked like Omar and Cuba Gooding, Richard said. He hired me for 'Baby Boy.' Thats how I got into doing stunts.

That job led to other jobs in major films, including "Barbershop" and "Transformers."

Everything was going well for Richard, he said, until 2008 when he tore his rotator cuff during filming for the movie "First Sunday," starring Ice Cube and Katt Williams.

I had surgery, and the person I was seeing at the time noticed that I started losing weight and was going to the bathroom a lot, Richard said.

When he went to the doctor for a routine checkup, he learned that he was diabetic. The doctor reviewed his medical history and asked if he has ever been evaluated for a liver transplant.

In 1987, when he was 13, he mysteriously became ill, he said. He was in the hospital for a high fever and the doctors noticed that his liver and spleen were enlarged, but didnt know why. He was sent home only to return a few days later.

I spent the whole summer in the hospital, Richard said. They did exploratory abdominal surgery and took a sample of all of my organs. They sent it to the CDC and other labs and came back with nothing.

Now that hes on the transplant list, Richard is hoping that hes able to raise enough money to travel to see a doctor in Mexico who was recommended by another patient and have the alternative procedure instead.

I want to avoid the transplant and keep my organs, Richard said.

He said he misses his old life, although he did more recently work on the show "Empire." Hes on disability now, but wants to return to work and the gym.

I was always in the gym, used to go hiking a lot, but now I work out when I feel like it, he said. I have low energy, and its kind of hard right now. I'll ride a bike and try to do air squats, but I was doing crossfit before it became too much for me.

Nobody wants to be sick. Id like to be back in California, moving around. I just get tired of going to doctor, getting poked and having having them tell me I need a transplant. Its mentally draining, and it's scary.

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Former Hollywood Stuntman Raising $10000 For His Stem Cell Therapy - DNAinfo

Researcher Leonard Zon, founder and director of the Stem Cell Program at Boston Children s Hospital, in a lab at the hospital in Boston. AP PHOTO

BOSTON -- In his offices at Boston Children's Hospital, Leonard Zon is busily developing cutting-edge stem cell therapies surrounded by fellow researchers, lab equipment and 300,000 striped, transparent zebrafish.

Zon's lab -- and the zebrafish -- are the results of an initiative begun nearly a decade ago to make Massachusetts one of the country's premier life sciences incubators.

That 2008 initiative, signed by former Democratic Gov. Deval Patrick, committed Massachusetts to spending $1 billion over 10 years to jump-start the life sciences sector -- attracting the best minds, research facilities and the venture capital funding.

By most yardsticks, Patrick's gamble has paid off. Massachusetts, and the greater Boston area in particular, are now seen as a top life sciences hub.

For Zon, and other life sciences leaders, the support has been transformative.

In 2013, the Massachusetts Life Sciences Center, which is charged with distributing the state funds, awarded a $4 million grant to Children's Hospital to help establish the Children's Center for Cell Therapy. Some of the money went toward replacing the original aquaculture facilities at Zon's lab with state-of-the-art systems.

Zon said the changes helped him pursue stem cell therapies -- taking tissues grown from stem cells aimed at thwarting specific diseases and transplanting them into a diseased organ.

"Massachusetts is the best place in the world for biotechnology," he said. "It's been life-changing for us."

Zon's experience isn't unique.

NxStage Medical, Inc., a medical technology company founded in 1998 in Lawrence focused on end-stage renal disease and acute kidney failure, received nearly $1.8 million in tax incentives through the program. In 2013, Woburn-based Bio2 Technologies received $1 million in loan financing, helping it develop bone graft substitute implants.

The state's reputation as a magnet for life sciences also can be seen in the surge of construction in Boston and Cambridge, particularly around the Kendall Square area, where glass-lined office and research buildings have sprouted.

Travis McCready, CEO of the Massachusetts Life Sciences Center, also pointed to the influx of grant money from the National Institutes of Health and funds from world-class academic and research institutions.

"By pretty much any measure we are considered the leading life sciences ecosystem in the U.S., and among the leading ecosystems in the world," McCready said.

McCready said the 2008 initiative helped create a framework for that growth, even as he acknowledged that not every company or research effort that receives funding succeeds.

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$1 billion life sciences plan bears fruit in Massachusetts - Lowell Sun - Lowell Sun

By Sharon N Chonchir, ContributorAugust / September 2017

Irish universities are at the forefront of medical science. Here are some of the recent breakthroughs they have made in understanding human health.

Dr. Philip Dunne. (Photo: QUB)

Improving the prognosis and quality of life for patients with bowel cancer is the aim of researchers at Queens University Belfast.

Patients with bowel cancer are currently offered chemotherapy, which is successful for some. However, it has no effect on fighting the cancer for others, even though those patients still suffer its debilitating side effects.

Scientists at Queens have shown how defining precise gene signatures within bowel cancer can allow for the development of new prognostic markers as well as personalized medical approaches.

Dr. Philip Dunne, senior research fellow at Queens said, Through analysing data generated from tissue samples, we have discovered different subtypes of bowel cancer. This will allow us to identify particular gene signatures that can indicate sensitivity or resistance to specific therapies. Thus, we can tailor treatment to individual patients, maximizing effectiveness while minimizing potential side effects.

Dr. Denise Fitzgerald. (Photo: QUB)

Bowel cancer is the third most common cancer in Ireland, with approximately 2,500 people diagnosed every year. Mortality rates are high and its the second most common cause of cancer death in the country.

Developing a cure for MS is the goal of another team of researchers at Queens. A recent landmark study of theirs has raised hopes of a breakthrough in the treatment of MS and other neurological disorders.

MS affects 2.3 million people worldwide. It involves the body attacking its own myelin sheath the protective layer that surrounds the brain, spinal cord, and optic nerve. These attacks can lead to symptoms such as vision loss, pain, fatigue, and paralysis.

Until now, treatment could limit these attacks but could not reverse the damage already done. This new research shows that a protein made by certain cells within the immune system triggers the brains stem cells to mature into oligodendrocytes that repair myelin.

This opens up new therapeutic potential for myelin regeneration in patients, says Dr. Denise Fitzgerald, senior author of the study.

Professor Noel Caplice. (Photo: Tomas Tyner / UCC)

Scientists at University College Cork may have discovered how to mend broken hearts. In the first trial of its kind in the world, a UCC professor has shown that low dose insulin-like growth factor injected into the heart improves remodelling for heart attack patients.

Professor Noel Caplice, the chair of cardiovascular sciences at UCC, successfully tested the growth factor in a trial of 47 patients, all of whom had experienced serious heart attacks.

Some received the growth factor while others received a placebo. Those who received the growth factor had improved remodelling of their heart muscle in the two months after their heart attack as well as other measures of improved heart performance.

Around a fifth of people who suffer heart attacks have ongoing difficulties because of lasting damage to heart muscle, even after the best current therapies.

We hope that these findings can be replicated in larger trials of many hundreds of subjects in thefuture, Caplice says. A significant minority ofpatients currently remain unwell after heart attacks and we are excited by the possibility that cardiacrepair therapy may help them.

Diabetic kidney disease (DKD) is the single leading cause of end-stage renal disease in the industrialized world, accounting for 40 percent of all new cases in the U.S. and E.U. There are few effective treatments for this condition, but a cell therapy company at the National University ofIreland Galway may have found one.

Dr. Stephen Elliman, the chief scientific officer at Orbsen Therapeutics, has discovered a novel allogeneic stromal cell therapy called ORBCEL-M. This therapy has demonstrated significant improvements in kidney function in pre-clinical models of DKD and clinical trials are now about to begin at locations across Europe, all coordinated from NUIG.

The aim of the research project, called NEPHSTROM, is to establish the safety and efficacy of ORBCEL-M. It hopes to show that important markers of DKD are improved and that the therapy is safe to use in the long term.

For decades, the standard practice for treating stings by the Portuguese man o war jellyfish was to rinse with seawater and apply ice. However, an NUIG study has found these are actually the worst things to do.

Collaborating with jellyfish sting experts from the University of Hawaii, NUIG scientists discovered that the best treatment was to rinse with vinegar,remove tentacles and immerse in 45C (113F) hot water for 45 minutes.

Just last September, unprecedented numbers of Portuguese man o war jellyfish came ashore inIreland. Thankfully we had very few reported stings given the time of year, said Dr. Tom Doyle, lecturer in zoology at NUIG. If this event had occurred during the summer, then we may have had hundreds.

According to Dr. Christie Wilcox from the University of Hawaii, the previous advice could have had fatal results. Because we didnt have solid science to back up medical practices, we ended up with practices that actually worsen stings and even cost lives, she said.

NUIG scientists are now researching the next most venomous snake in Irish waters, the lions mane jellyfish. Its responsible for more bad stings than any other and many victims end up in hospital.

Professor Abhay Pandit. (Photo: Andrew Downes / xposure)

CRAM, the Science Foundation Ireland Centre for Research in Medical Devices based at NUIG, has recently begun to collaborate with the Mayo Clinic on research into blood clots which cause ischemic stroke.

Ischemic strokes occur as a result of an obstruction within a blood vessel supplying blood to the brain. They can be caused by clots that come from the heart, the carotid artery, or other parts of the body. The characteristics of these clots vary widely, which has implications for what therapy is given to patients.

The Mayo Clinic is initiating a nationwide effort in the U.S. to collect samples of clots removed from patients who have suffered strokes in order to analyse them. Through CRAM, NUIG will be establishing a dedicated clot pathology lab to conduct parallel research in Europe.

Professor Abhay Pandit, scientific director of CRAM, commented: Were excited by the opportunity to advance research in this area. We hope this will lead to ground-breaking research and drive significant improvements in outcomes for stroke patients in the future.

Professor Nigel Stevenson. (Photo: TCD)

Scientists at Trinity College Dublin have discovered that a biological molecule known as STAT3 is critical in protecting against infection.

With new viral infections such as Zika and Ebola emerging all the time, the importance of understanding how we can protect our immune systems against viruses has never been greater.

During any viral infection, cells produce interferon to prevent viruses from replicating in our bodies.

Interferon activates other molecules within the cells and when the final molecule is activated, the viralinfection should be cleared.

However some viruses, such as hepatitis C, are not cleared by this response. Immunologists from Trinity, led by assistant professor in immunology Dr. Nigel Stevenson, have discovered that these viruses have evolved to block responses to interferon.

STAT3 may be able to counter this negative effect. Using new molecular techniques, we have revealed that STAT3 is an essential anti-viral component in the signaling pathway, said Dr. Stevenson. Without it, cells cannot even fight the common flu virus. This discovery opens the door to new therapeutic options which we hope will help people to restore their natural immunity against a host of problematic viruses.

Professor Orla Hardiman. (Photo: TCD)

Researchers from Trinity College Dublin have also shown for the first time that motor neuron disease (MND) and schizophrenia share a genetic origin,indicating that the causes of these conditions are biologically linked.

By analysing the genetic profiles of 13,000 MND cases and 30,000 schizophrenia cases, the researchers confirmed that many of the genes associated with these conditions are the same.

Our work has shown us that MND is a much more complex disease than we originally thought, lead investigator Orla Hardiman, professor of neurology in Trinity, said. Combining clinical work and our studies using MRI and EEG, it becomes clear that MND is not just a disorder of individual nerve cells but a disorder of the way these nerve cells talk to one another as part of a larger network.

She and her team now see MND similarly to schizophrenia as a problem of disruptions in connectivity between different regions of the brain. They are looking for drugs to help stabilize these failing brain networks.

This research also shows that the divide between psychiatry and neurology may be a false one. This will have major implications for how we classifydiseases going forward and in turn how we traindoctors in psychiatry and neurology, said Professor Hardiman.

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Irish Universities: Leading the Way in Life & Health Science - Irish America

An international team of medical experts recently published a global call to action in Science Translational Medicine in an effort to curb the unethical, unsubstantiated use of stem-cell based therapies driving medical tourism. Such ill-advised stem cell treatments have led to pediatric deaths in Germany, blindness in the United States, the closure of Italys Stamina Foundation to name a few as well as a variety of untoward effects given their lack of rigorous testing for safety and efficacy.

With high price tags, so-called stem-cell clinics are designing therapies without evidence that serve to do harm, be ineffective, prey on the most vulnerablepotentially curtailing their ultimate treatment choices, and threaten the legitimate work being done that holds great promise for devastating disease.

When greed trumps science, we all lose.

In The Worst 'Healthcare': 'Stem Cell' Clinics Wrought With Red Flags, Insincerity And Blindness, I detail the distressing accounts of three patients who endured irreparable damage to their vision after seeking treatment at the same unnamed stem cell clinic in Broward, Florida. Reported in the New England Journal of Medicine (NEJM), the harrowing experiences of the women aged 72-88 years old resulted in blindness to near blindness from untested stem cell therapies being injected into their eyes while being fleeced $5000 for the procedures. Promised revolutionary therapy, they were left with catastrophic reminders of the unfortunate and unnecessary ordeal.

Theirs is a cautionary taleof the hazards of poor and irresponsible practice at a private stem cell clinic, unfixable medical interventions with no scientific evidence to back claims, sales over substance, marketing hype lacking in meaning and actions taken by seemingly complicit personnel who may have misrepresented their credentials that took advantage of those most in need. See here for a more detailed road map of how to read between the lines of such marketing hype and to understand the timeline of this particular bad outcome.

Heres the deal with stem cells

Stem cells are currently in use for a rather limited scope of disorders. Ones where the data is well-established and been subject to proper design, rigorous testing and clinical trials. They are also being actively studied, in general. The notion that they can cure every type of medical condition is not one based in our present reality or the near term future. The media tends to overstate where we are in stem cell-based therapeutics.

The safety and efficacy of stem cell use when derived from bone marrow or your peripheral blood is well-established, but stem cells are now being increasingly derived from alternate sources like adipose (aka fatty) tissue and put in use for orthopedic to neurological disorders.

Many in the medical community are enthused about their promise and rightfully so as some advancements are already underway. In my recent article Did Gene Therapy Cure Sickle Cell Disease?, I discuss the hopeful work in autologous stem cell transplantation obtained from bone marrow for this and other hemoglobinopathies. This is further explored in this television appearance:Dr. Jamie Wells On Al Jazeera TV Discussing Sickle Cell Anemia.

Facilities offering false hope often based on the most minimal of clinical evidence are popping up all over the country and world without well-controlled clinical trials or having met any regulatory standards. In the cases of autologous use especiallysince they are your own cells, advocates affirm they are safe. These private stem cell clinics are typically patient-funded at nonacademic centers, are not based on preclinical research or sound design and lack investigational new drug application with the FDAbecause, again, they are your own cells despite the fact what the facility mixes them with are unknown agents that have not been tested to confirm safety.(1)

In a perspective written by the U.S. Food and Drug Administration in the NEJM, the FDA maintains: Outside the setting of hematopoietic reconstitution and a few other well-established indications, the assertion that stem cells are intrinsically able to sense the environment into which they are introduced and addresswhatever functions require replacement or repairwhether injured knee cartilage or a neurologic deficitis not based on scientific evidence. The piece goes on to inform about misadventures of their use and the worrisome lack of evidence in particular in circumstances where therapies proved harmful or ineffective when properly studied.

Hence, why this recent global call to action by worldwide leaders in the field

Buoyed by efforts of the scientific community to impact changes in stem cell facilities in Germany and Italy among others, an international consortium of medical experts outline in their latest publication local to transnational considerations that could limit unchecked marketing claims and unfounded science. Appreciating the current climate of expediting lengthy approval processes, the politics of right to try legislation and direct-to-consumer advertising, the authors contend with respect to sham stem cell therapies under-regulation has led to substantial, reverberating harm.

Due to general regulatory resistance, the panel of fifteen urges a more coordinated approach nationally and internationallythat emphasizes engagement, harmonization and enforcement. Highlighting prior success, the group encourages mobilization of international scientific organizations to create global standards and the utilization of traditional and social media type engagement to expand reach to the public as well as positively influence national policies.

With the main goals of eliminating harmful therapies and preserving a patients ability to seek effective treatment, they identify further the risk of not doing anything will worsen the big problems of destabilizing health markets and delegitimizing biomedical efforts that could genuinely benefit society.

Among their requests for proactive efforts is for groups with broad constituencies like the World Health Organization (WHO) to dispense guidelines in conjunction with national authorities for the responsible use of human cells and tissues which they maintain they already dofor medical devices and medicines. They call for cross-border partnerships for compliance.

Of particular note, the article includes a wonderful chart clarifying where inroads can be made locally, regionally and internationally to prevent the all too frequent co-opting of scientific legitimacy. It can be very difficult for patients to suss out what is fact from fiction when powerful advertising runs amok. Words like revolutionary and clinical trials and expert are consistently thrown around to confuse and can be a challenge for many to unpack accurately.

The authors provide in this graphic tangible ways to cut through the nonsense. By outing deceptive tactics used throughout the commercialization of these products and spreading these protective messages, patients can become empowered and the culture of fleecing might shift. Here, they point out a number of ways scientific integrity gets diminished:the use of renting space in academic facilities as a way to appear legitimate by association, suggestby having a patent application this means the product is proven or tested as opposed to just a sign of an initiation of applying, citing preclinical and other findings to rationalize clinical use without efficacy testing etc. (3) I would argue this list is an excellent tool that could be applied well beyond the stem cell industry. Interventions starting withthis listcould certainly support more honesty.

In conclusion

Though rarely a lover of regulation, in general, given the likelihood of overdoing it, creating more problemsand the tendency for many policies to be one-size-fits-all and misguided, in this realm the price people are paying as well as society could be too great to allow the wild west ways of the stem cell industry to continue as is. The authors in this work provide some meaningful measures to compel a more honest arena.

Source(s):

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Fraud Alert! Unproven Stem Cell Use Prompts International Call to Action - American Council on Science and Health

In a new studyin Nature Communications, Stephanopoulos and his colleague Ronit Freeman successfully demonstrated the ability to dynamically control the environment around stem cells, to guide their behavior in new and powerful ways. Credit: Northwestern University

What if one day, we could teach our bodies to self-heal like a lizard's tail, and make severe injury or disease no more threatening than a paper cut?

Or heal tissues by coaxing cells to multiply, repair or replace damaged regions in loved ones whose lives have been ravaged by stroke, Alzheimer's or Parkinson's disease?

Such is the vision, promise and excitement in the burgeoning field of regenerative medicine, now a major ASU initiative to boost 21st-century medical research discoveries.

ASU Biodesign Institute researcher Nick Stephanopoulos is one of several rising stars in regenerative medicine. In 2015, Stephanopoulos, along with Alex Green and Jeremy Mills, were recruited to the Biodesign Institute's Center for Molecular Design and Biomimetics (CMDB), directed by Hao Yan, a world-recognized leader in nanotechnology.

"One of the things that that attracted me most to the ASU and the Biodesign CMDB was Hao's vision to build a group of researchers that use biological molecules and design principles to make new materials that can mimic, and one day surpass, the most complex functions of biology," Stephanopoulos said.

"I have always been fascinated by using biological building blocks like proteins, peptides and DNA to construct self-assembled structures, devices and materials, and the interdisciplinary and highly collaborative team in the CMDB is the ideal place to put this vision into practice."

Yan's research center uses DNA and other basic building blocks to build their nanotechnology structuresonly at a scale 1,000 times smaller than the width of a human hair.

They've already used nanotechnology to build containers to specially deliver drugs to tissues, build robots to navigate a maze or nanowires for electronics.

To build a manufacturing industry at that tiny scale, their bricks and mortar use a colorful assortment of molecular Legos. Just combine the ingredients, and these building blocks can self-assemble in a seemingly infinite number of ways only limited by the laws of chemistry and physicsand the creative imaginations of these budding nano-architects.

Learning from nature

"The goal of the Center for Molecular Design and Biomimetics is to use nature's design rules as an inspiration in advancing biomedical, energy and electronics innovation through self-assembling molecules to create intelligent materials for better component control and for synthesis into higher-order systems," said Yan, who also holds the Milton Glick Chair in Chemistry and Biochemistry.

Prior to joining ASU, Stephanopoulos trained with experts in biological nanomaterials, obtaining his doctorate with the University of California Berkeley's Matthew Francis, and completed postdoctoral studies with Samuel Stupp at Northwestern University. At Northwestern, he was part of a team that developed a new category of quilt-like, self-assembling peptide and peptide-DNA biomaterials for regenerative medicine, with an emphasis in neural tissue engineering.

"We've learned from nature many of the rules behind materials that can self-assemble. Some of the most elegant complex and adaptable examples of self-assembly are found in biological systems," Stephanopoulos said.

Because they are built from the ground-up using molecules found in nature, these materials are also biocompatible and biodegradable, opening up brand-new vistas for regenerative medicine.

Stephanopoulos' tool kit includes using proteins, peptides, lipids and nucleic acids like DNA that have a rich biological lexicon of self-assembly.

"DNA possesses great potential for the construction of self-assembled biomaterials due to its highly programmable nature; any two strands of DNA can be coaxed to assemble to make nanoscale constructs and devices with exquisite precision and complexity," Stephanopoulos said.

Proof all in the design

During his time at Northwestern, Stephanopoulos worked on a number of projects and developed proof-of-concept technologies for spinal cord injury, bone regeneration and nanomaterials to guide stem cell differentiation.

Now, more recently, in a new study in Nature Communications, Stephanopoulos and his colleague Ronit Freeman in the Stupp laboratory successfully demonstrated the ability to dynamically control the environment around stem cells, to guide their behavior in new and powerful ways.

In the new technology, materials are first chemically decorated with different strands of DNA, each with a unique code for a different signal to cells.

To activate signals within the cells, soluble molecules containing complementary DNA strands are coupled to short protein fragments, called peptides, and added to the material to create DNA double helices displaying the signal.

By adding a few drops of the DNA-peptide mixture, the material effectively gives a green light to stem cells to reproduce and generate more cells. In order to dynamically tune the signal presentation, the surface is exposed to a soluble single-stranded DNA molecule designed to "grab" the signal-containing strand of the duplex and form a new DNA double helix, displacing the old signal from the surface.

This new duplex can then be washed away, turning the signal "off." To turn the signal back on, all that is needed is to now introduce a new copy of single-stranded DNA bearing a signal that will reattach to the material's surface.

One of the findings of this work is the possibility of using the synthetic material to signal neural stem cells to proliferate, then at a specific time selected by the scientist, trigger their differentiation into neurons for a while, before returning the stem cells to a proliferative state on demand.

One potential use of the new technology to manipulate cells could help cure a patient with neurodegenerative conditions like Parkinson's disease.

The patient's own skin cells could be converted to stem cells using existing techniques. The new technology could help expand the newly converted stem cells back in the laband then direct their growth into specific dopamine-producing neurons before transplantation back to the patient.

"People would love to have cell therapies that utilize stem cells derived from their own bodies to regenerate tissue," Stupp said. "In principle, this will eventually be possible, but one needs procedures that are effective at expanding and differentiating cells in order to do so. Our technology does that."

In the future, it might be possible to perform this process entirely within the body. The stem cells would be implanted in the clinic, encapsulated in the type of material described in the new work, and injected into a particular spot. Then the soluble peptide-DNA molecules would be given to the patient to bind to the material and manipulate the proliferation and differentiation of transplanted cells.

Scaling the barriers

One of the future challenges in this area will be to develop materials that can respond better to external stimuli and reconfigure their physical or chemical properties accordingly.

"Biological systems are complex, and treating injury or disease will in many cases necessitate a material that can mimic the complex spatiotemporal dynamics of the tissues they are used to treat," Stephanopoulos said.

It is likely that hybrid systems that combine multiple chemical elements will be necessary; some components may provide structure, others biological signaling and yet others a switchable element to imbue dynamic ability to the material.

A second challenge, and opportunity, for regenerative medicine lies in creating nanostructures that can organize material across multiple length scales. Biological systems themselves are hierarchically organized: from molecules to cells to tissues, and up to entire organisms.

Consider that for all of us, life starts simple, with just a single cell. By the time we reach adulthood, every adult human body is its own universe of cells, with recent estimates of 37 trillion or so. The human brain alone has 100 billion cells or about the same number of cells as stars in the Milky Way galaxy.

But over the course of a life, or by disease, whole constellations of cells are lost due to the ravages of time or the genetic blueprints going awry.

Collaborative DNA

To overcome these obstacles, much more research funding and recruitment of additional talent to ASU will be needed to build the necessary regenerative medicine workforce.

Last year, Stephanopoulos' research received a boost with funding from the U.S. Air Force's Young Investigator Research Program (YIP).

"The Air Force Office of Scientific Research YIP award will facilitate Nick's research agenda in this direction, and is a significant recognition of his creativity and track record at the early stage of his careers," Yan said.

They'll need this and more to meet the ultimate challenge in the development of self-assembled biomaterials and translation to clinical applications.

Buoyed by the funding, during the next research steps, Stephanopoulos wants to further expand horizons with collaborations from other ASU colleagues to take his research team's efforts one step closer to the clinic.

"ASU and the Biodesign Institute also offer world-class researchers in engineering, physics and biology for collaborations, not to mention close ties with the Mayo Clinic or a number of Phoenix-area institutes so we can translate our materials to medically relevant applications," Stephanopoulos said.

There is growing recognition that regenerative medicine in the Valley could be a win-win for the area, in delivering new cures to patients and building, person by person, a brand-new medicinal manufacturing industry.

Explore further: New technology to manipulate cells could help treat Parkinson's, arthritis, other diseases

More information: Ronit Freeman et al. Instructing cells with programmable peptide DNA hybrids, Nature Communications (2017). DOI: 10.1038/ncomms15982

Original post:
Bio-inspired materials give boost to regenerative medicine - Medical Xpress

A darling British biotech that is working on the development of the worlds leading gene therapy for cancer has posted reduced losses and a leap in sales.

Oxford BioMedica signed a deal worth up to $100m (77m) with Swiss drugs giant Novartis last month to supply cell material for its potential blockbuster treatment, known as CTL019, for a type of leukaemia.

The living drug was recommended for approval by US regulators in June, with a final determination expected this autumn.

It would be the first gene therapy for cancer and if approved is expected to generate a spike in revenues for Oxford BioMedica, with the potential for further tie-ups on other cancer treatments.

Oxford BioMedica is also trialling its lentiviral vectors, which help manipulate genes, in potential treatments for Parkinsons Disease and an eye condition.

In half-year results today, Oxford BioMedica pared back losses to 2.2m, compared to 6.9m in the red the previous year. Revenues jumped 26pc to 15.7m.

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Gene therapy darling Oxford BioMedica pares losses - Telegraph.co.uk

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GAINESVILLE, Florida Imagine only being able to see the things in front of you in soft focus, and just in black and white.

For people with the genetic eye condition achromatopsia, those are just some of the side effects.

Each time Tara Cataldo prepares to leave her house, she has to make sure her face is completely shielded from the sun.

"I need to have very dark, very tinted sunglasses to feel comfortable outside, to see really well," said Tara.

Tara has achromatopsia, a genetic condition that makes her eyes incredibly sensitive to light.She is also very nearsighted; even while wearing glasses or contacts, she can only see clearly at a very short distance.

"I cannot drive a car so I rely on public transportation and my bike to get around," she said.

"There are currently no approved or no effective treatments for achromatopsia," said Dr. Christine Kay, a surgical ophthalmologist at the University of Florida.

But she's working to change that.

She is one of a handful of experts testing a gene therapy.

"For achromatopsia the cells we have to target are cone cells responsible for decreased vision and color vision. and those are cells at the very bottom layer of the retina," explained Dr. Kay.

Using a tiny cannula, doctors deliver a normal copy of one of two mutated genes, the CNGA-3 or CNGB-3 gene, directly into the eye to restore vision.

Tara`s myopia is so severe that her risk of retinal detachment from any retinal surgery is high, which rules her out for the current trial.

In the meantime, Tara says she's learned to adapt to achromatopsia and live without limitations.

"And I hope, ya know, all young achromats, ya know, learn the same thing."

Dr Kay says if the gene could eventually be delivered to the surface of the retina, additional patients, like Tara, could be treated. AGTC, the biotech company that developed the therapies, and several U.S. universities have successfully tested this therapy in dogs and sheep.

NEW TECHNOLOGY: A new clinical trial is underway testing gene therapy for achromatopsia. The genes are responsible for releasing proteins essential for the function of all the cells. Researchers remove the virus from the host DNA so it does not have the capacity to make the patient sick, and then insert the gene of interest into the DNA. Surgically done by a vitro retinal surgeon, the gene is then directly delivered to the retinal tissue. Researchers try to avoid directly touching the retina, to avoid detaching it and keep the surgery less evasive. The CNGA3 or CNGB3 gene would help restore the patient`s vision. This can only be done on certain patients; those with extremely severe achromatopsia are not able to participate in this trial because of the risk of retinal rupturing.

If this story has impacted your life or prompted you or someone you know to seek or change treatments, please let us know by contacting Jim Mertens at jim.mertens@wqad.com or Marjorie Bekaert Thomas at mthomas@ivanhoe.com.

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YOUR HEALTH Gene therapy for better eyesight - WQAD.com

Investment Thesis

With an undervalued stock, a critically-acclaimed drug leading in its space and a barrage of financial and expertise-based backing, Sangamo Therapeutics (NASDAQ:SGMO) is the ideal vehicle for exposure to a market with high growth potential and a hedge against the antiquated methods (CRISPR/Cas9) of treating hemophilia. The same method has also been found to work to a high degree of accuracy on other genetic disorders.

Sangamo Therapeutics' accomplishments to date have helped to draw integrity to its operations and raise its profile against common competitors in the space (QURE, GSK, ONCE, BMRN, BLUE, BIVV (a pure-play hemophilia company). Principally, Sangamo has secured an exclusivity agreement (and subsequent vote of confidence) from Pfizer (NYSE:PFE) (also once a competitor) regarding the development and commercialization of gene therapy programs for hemophilia A (SB-525), which affects 85% of hemophilia sufferers (Nelson Pediatrics), bagging $70 million upfront and a potential $475 million in milestones and royalties (Reuters). (The Zinc Finger Protein Nuclease technology method adopted is targeted at diseases which are caused by genetic defects, as their removal is known to respond best to gene editing targeting, in particular, tauopathies, thalassemias, hemophilia, and HIV/AIDS).

The company's strong balance sheet was further bolstered by a raise of circa $78.1 million through public offering at a price of $7.25 per share, with a revised analyst share price target estimates from Jefferies LLC of $17 per share (jefferies.com), as a "financial investment hedge against emerging gene-editing technology of CRISPR," the CRISPR/Cas9 system, has seen less successful testing, coupled with scientific criticism for its reported high incidence of nonspecific DNA cleavage, mosaicism (failure to replicate mutant alleles in only some of the cells) and inversely, and overproduction of mutations in a given organism.

SGMO's latest 10-Q (SEC.gov):

In the context of gene therapy, Sangamo ($707.16 million market cap) (capitaliq.com) was the only firm with innovative gene therapy drugs to advance in two clinical trials (namely the SB-728-T program in the Sangamo ZFP Therapeutic Pipeline targeting HIV/AIDS), and the first to conduct genome editing studies in clinical trials, as covered in the Financial Times. The stock's performance has been volatile, and investors were disinclined to back the drug lottery in gene therapy as biotech became heavily saturated in players, regulations and setbacks, stunting the prospect of any tangible advancement.

Sangamo has jumped on the "radar" to become a buy. The FDA has authorized Fast Track designation, the scientific backing is legitimate (and has received industry-wide appraisal), the longer-term targets look plausible and Sangamo's management and consultants are capable of executing given their credentials and two decades of development.

By all measures of valuation, Sangamo is undervalued, and price-action enthusiasts will recognize the surge in trading volumes and the breaking of the 50-day moving average. Early riders would have capitalized on the short-term price depression triggered by the publicizing of the mucopolysaccharidosis Type I (MPS I) and MPS II delay, an overreaction from the market that posed a great value opportunity at the beginning of this month, whose benefits were felt just last week when the stock surged on the impressive FIH results.

Gene therapy (and emicizumab) renders recombinant factor VIII/IX proteins redundant. This a slice of the industry that is represented by $8-10 billion annually (with 90%+ profit margins) shared by Shire (NASDAQ:SHPG), Bayer (OTCPK:BAYRY), Novo Nordisk (NYSE:NVO), Pfizer and Bioverativ (NASDAQ:BIVV), an overvalued stock whose current operations consist entirely of the moribund method of treating hemophilia - a potential sell. It recently acquired True North and thus entering into cold agglutinin disease market, a result of severe hemolytic anemia.

Sangamo now has the backing and leeway to make its drugs commercially viable, magnetizing further interest - 48 institutions have increased their respective positions in SGMO, with Wasatch and BlackRock leading the pack. The smart money and medical experts are backing Sangamo.

Bold tickers indicate potential buys should this thesis play out as predicted.

This is not investment advice, you are advised to carry out your own due diligence.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Sangamo Therapeutics: The Breakthrough In Gene Therapy - Seeking Alpha

MOSCOW, August 17. /TASS/. A Russian gene therapy drug for individuals infected with HIV called Dinavir is undergoing pre-clinical trials, and the drug has already proved its efficiency on cells. The pre-clinical tests on animal models, clinical trials and the registration procedure may take up to 10 years, senior research fellow at the Epidemiology Central Research Institute of Rospotrebnadzor (the Federal Service on Surveillance for Customers Rights Protection and Human Well-Being) Dina Glazkova told TASS.

"This is not about the next year, but rather in five years, at the earliest. It takes up to 10 years on the average," she said.

Glazkova reiterated that the registration is made after the clinical trials. "Again, the clinical trials are costly, and the drug production is costly as well," the scientist added.

Dinavir proved to be safe while tested on cells, in vitro. A Phase II pre-clinical trial will utilize animal models to test the efficiency and safety of treatment. A Phase I clinical trial will be carried out on humans to test safety of the therapy and will take up not less than a year.

"Phase II takes up two to three years, and it is unclear how much will be required from us. Phase I is about safety, and it takes a few patients: five, maybe ten. Phase II is when we have to prove that the drug works in these five to ten [patients] and that it had a positive effect on them. Phase III is when we enroll a lot of patients [in the trial] to show that the five were cured not by accident and that it [the gene therapy] really works," Glazkova explained.

The gene therapy for HIV treatment is being developed by a group of researches at the Epidemiology Central Research Institute of Rospotrebnadzor.

Original post:
Scientists foresee Russian gene therapy for HIV cure may be registered in 5-10 years - TASS

Whether youre diabetic, youre caring for a person in one of the various stages of the disease or you simply want to maintain a healthy lifestyle Santa Ynez Valley Cottage Hospital (SYVCH) in Solvang is offering free nutrition and diabetes education classes.

Beginning Sept. 1, the community hospital, located at 2050 Viborg Road, is hosting the classes to encourage healthy eating habits and diabetes management for all ages.

Led by clinical dietitian Stacey Bailey, the support classes will be held in the hospitals conference room (enter through the Physicians Clinic). No RSVP is required and participants can bring their own lunch to eat during class.

People have so many concerns about food types, preparation, diets and supplements, said Bailey, M.S., R.D. and C.D.E. Please join me as I share some tips during these informal, free classes.

Bailey, who joined SYVCH as clinical dietitian in August 2011, provides inpatient and outpatient dietary management and counseling services. She also assists in the formulation of meal and menu planning specifically for the hospital.

SYVCH reports it treated 72,000 patients through its 24-hour emergency departments and helped deliver 2,400 newborns. One-on-one nutrition sessions with Bailey are available with a doctors referral.

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We are delighted to be able to offer these free, educational and fun classes taught by our popular clinical dietitian Stacey Bailey," said Wende Cappetta, SYVCHs vice president. We encourage everyone to take this opportunity to learn how to achieve improved health and wellness with better food choices.

For more information, contact Bailey directly at 805-694-2351.

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Free nutrition and diabetes classes offered | Local News ... - Lompoc Record