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‘Scienceploitation’: Prof calls for crackdown on unproven stem cell … – MetroNews Canada

July 12th, 2017 4:44 pm

An Edmonton professor is calling for tighter regulation of stem cell therapies, as clinics peddling expensive, unproven and dangerous treatments crop up in Edmonton and across North America.

"This is a big problem, said University of Alberta health-law professor Timothy Caulfield. Were not talking about a handful of clinics, were talking about hundreds and hundreds of clinics all over the world, including in North America.

His research has found many clinics offering the unproven therapies are charging tens of thousands of dollars per treatment, and are not advertising them as experimental.

Clinics are professing to cure conditions like Multiple Sclerosis, ALS, cancer and autism.

They represent them as if they are effective, as if they are relatively routine, and as if they are relatively risk free. And thats just not the case in the vast majority of situations, Caulfield said.

He said some advertised stem cell therapies have led to serious health problems and deaths.

Caulfield and 14 colleagues from around the world call for global regulations in a new paper in Science Translational Medicine.

Unproven stem cell therapies used to only be available through stem cell tourism for North Americans who were willing to travel abroad. But recently, theyve been popping up across North America.

Caulfield said there is one such clinic in Edmonton but did not give its name.

In many jurisdictions, he said, the clinics operate under a legal loophole.

If the treatments are considered medical practices under the domain of professional standards, rather than drugs regulated by organizations like Health Canada or the FDA, they are harder to regulate.

I call it scienceploitation. The idea is that these clinics are using the language of stem cells, an exciting and legitimate field of science, in order to sell products, Caulfield said.

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Medical Tourism in Spotlight as Experts Call for Tighter Regulation – Bioscience Technology

July 12th, 2017 4:44 pm

Countries should unite to tackle unscrupulous advertising of unproven therapies involving stem cells, experts say.

An international group of leading experts has called for tighter regulation of so-called stem cell tourism. This involves patients travelling to other countries, where medical regulations are less strict, for treatment with potentially unsafe therapies.

Hundreds of medical centres around the world are offering therapies that involve transplantation of so-called stem cells -- which they claim have the ability to repair damaged tissues. Clinics are marketing the treatment for a range of conditions, including multiple sclerosis and Parkinson's disease.

Often these therapies are advertised directly to patients with the promise of a cure. But experts say there is often no evidence to show that the treatments will help anyone, or will not cause harm.

Researchers say the practice risks undermining the development of rigorously tested, validated therapies and puts lives at risk.

Writing in the journal Science Translational Medicine, the group has called for coordinated global action to tackle the problem.

They say tighter regulations on advertising stem cell therapies are needed, so that unsupported claims about potential clinical benefits do not go unchallenged.

Global regulatory authorities should agree international standards for the manufacture and testing of cell and tissue-based therapies, they add.

The group -- which includes experts from the University of Edinburgh -- also calls for the World Health Organization to help guide responsible clinical use of cells and tissues, as it does for medicines and medicinal devices.

Their appeal follows the deaths of two children at a clinic in Germany in 2010, which exploited a legal loophole to offer untested treatments. The clinic has since been closed.

Dr. Sarah Chan, a Chancellor's Fellow at the University of Edinburgh, said: "Many patients feel that potential cures are being held back by red tape and lengthy approval processes. Although this can be frustrating, these procedures are there to protect patients from undergoing needless treatments that could put their lives at risk.

"Stem cell therapies hold a lot of promise but we need rigorous clinical trials and regulatory processes to determine whether a proposed treatment is safe, effective and better than existing treatments."

Some types of stem cell transplantation - mainly blood and skin stem cells -- have been approved to treat certain types of cancer and to grow skin grafts for patients with severe burns. These treatments have been rigorously tested in clinical trials.

SOURCE: University of Edinburgh

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The World Health Minute: India’s superbug problem, link found between household products and antibiotic resistance – The News Minute

July 12th, 2017 4:44 pm
The World Health Minute: India's superbug problem, link found between household products and antibiotic resistance
The News Minute
Stem-cell tourism involving patients who travel to developing countries for treatment with unproven and potentially risky therapies should be more tightly regulated, international health experts said. With hundreds of medical centers around the world ...

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The rise of medical tourism is impacting stem cell treatments … – Healthcare Global

July 12th, 2017 4:44 pm

There have been worldwide concerns surrounding stem cell therapies, where many patients are seeking unproven treatments in order to further their ongoing treatments in other countries, increasing the figures surrounding medical tourism. Sought in bone marrow and the umbilical cord, stem cells are utilised in the treatment of immune diseases, degenerative diseases and blood cancers, such as leukaemia, as well as ongoing autoimmune conditions.

However, with a growing rise of patients seeking treatments which have yet to be clinically proven, approximately 15 worldwide experts have announced a need to introduce international regulations to ban the practice for medical teams to offer treatments which are yet to be proven successful in areas such as the treatment of blood cancers, such as leukaemia.

Within the US journal Science Translational Medicine, it states: Approaches for international regulation not only need to develop consistent rules over the commercialisation of medical practices and products but also need to give them teeth by developing cross-border partnerships for compliance. However, this is made further complicated by countries not having a clear directive surrounding current regulations, besides the treatments, which are placed on different clinical frameworks.

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The number of patient deaths in seeking unproven therapies has been on the rise in Australia and Russia, to which the health industry seeks to put a block on such ability for vulnerable patients to undertake, or become involved in unproved treatments, increasing their risks of infection and fatalities.

In the journal, it states: The term 'stem cell' has been used broadly in promises of youth, rejuvenation and good health, as well as in the branding of cosmetics, dietary supplements, and sports products. Such hyperbole carries with it not only an increased risk of exploitation of vulnerable patients and their families desperate for a cure, but also of significant damage to the health of those subjected to these unproven interventions.

Australia adopts some of the highest number of companies advertising medical treatments, without any evidence that such treatments are safe, let alone produce positive results, The Guardian reports. The Therapeutic Goods Administration is therefore seeking to fully close the regulatory loophole which enables the growth of autologous treatments, and the businesses which cater to this practice.

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New NUH study to test stem cells as treatment for liver disease – TODAYonline

July 12th, 2017 4:43 pm

SINGAPORE The use of stem cell treatment to repair liver cirrhosis, or hardening of the liver, will be tested in a clinical trial here involving 46 patients and costing S$2.6 million.

The four-year study, which was launched yesterday, came amid a growing waiting list in Singapore for a liver transplant, which is currently the only cure for patients with end-stage liver cirrhosis.

Conducted by a multi-centre team from several restructured hospitals here, the study is led by the National University Hospital (NUH).

Liver failure is one of the top 20 causes of death in Singapore, but many patients are not suitable for a transplant due to factors such as age and surgical fitness.

Out of every five patients doctors see with end-stage liver disease, only one qualifies for a liver transplant, said Dr Dan Yock Young, principal investigator of the clinical trial and senior consultant at NUHs division of gastroenterology and hepatology.

(A liver transplant) is curative, but it is a complex procedure, and many patients are not suitable for it. For these patients, treatment is limited, but morbidity and mortality rates are high as high as 50 per cent in one year and this is probably worse than many (of the) other terminal illnesses we talk about today, he said.

Animal studies conducted over the last five years have shown that stem cells can reconstruct the micro-environment of a normal liver.

Like how branches are of critical importance in supporting the leaves and fruits of a tree, the endothelial (stem) cells contribute to supporting a nutritious environment for the hepatocyte (liver) cells, Dr Dan explained.

While similar stem-cell studies have been conducted in other centres in Asia, there has been no definitive evidence of the benefits of the treatment for liver patients.

The study will recruit 46 patients aged between 40 and 70 years old, and who are at the terminal stages of chronic liver disease, over three years. It is funded by the National Medical Research Council.

During the clinical trial, patients will be divided into a therapeutic group and a control group.

All patients will receive an injection to stimulate their bone marrow cells as part of the supportive treatment for their liver cirrhosis. However, only patients in the study group will have the stem cells from the bone marrow extracted and deposited directly into their liver for more targeted repair.

Using ones own stem cells will avoid the problem of cell rejection.

The liver tissue will be examined three months later, and an investigation to compare pre- and post-transplant results will be conducted after a year.

Since invasive surgery is not required for stem-cell therapy, the fatality risk is significantly lowered for the patient. However, other risks such as severe bleeding and infections still remain, given the patients weakened condition.

NUH also noted that the stem-cell therapy does not replace liver transplants, and the latter remains the best available treatment for liver cirrhosis.

It is very painful to turn patients away when we cannot offer them a liver transplant, said Dr Dan, adding that this stem cell therapy will serve as an alternative option.

We hope that this is a stepping stone to trials for stem cell candidates, he added.

MORE WAITING FOR A LIVER

The number of people on the waiting list for a liver transplant has been growing in recent years. In June last year, it was reported that there were 54 people on the list, more than double the 24 patients in 2011.

Chronic Hepatitis B remains the primary cause of non-alcoholic fatty liver disease, which refers to a range of liver conditions affecting people who drink little to no alcohol. However, obesity has become a contributing factor to the illness as well.

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Stem Cell Advance Brings Bioengineered Arteries Closer to Reality – Technology Networks

July 12th, 2017 4:43 pm

Stem cell biologists have tried unsuccessfully for years to produce cells that will give rise to functional arteries and give physicians new options to combat cardiovascular disease, the worlds leading cause of death.

But new techniques developed at the Morgridge Institute for Research and the University of WisconsinMadison have produced, for the first time, functional arterial cells at both the quality and scale to be relevant for disease modeling and clinical application.

Reporting in the July 10 issue of the journal Proceedings of the National Academy of Sciences, scientists in the lab of stem cell pioneer James Thomson describe methods for generating and characterizing arterial endothelial cells the cells that initiate artery development that exhibit many of the specific functions required by the body.

Further, these cells contributed both to new artery formation and improved survival rate of mice used in a model for myocardial infarction. Mice treated with this cell line had an 83 percent survival rate, compared to 33 percent for controls.

The cardiovascular diseases that kill people mostly affect the arteries, and no one has been able to make those kinds of cells efficiently before, says Jue Zhang, a Morgridge assistant scientist and lead author. The key finding here is a way to make arterial endothelial cells more functional and clinically useful.

Cardiovascular disease accounts for one in every three deaths each year in the United States, according to the American Heart Association, and claims more lives each year than all forms of cancer combined. The Thomson lab has made arterial engineering one of its top research priorities.

New techniques have produced, for the first time, functional arterial cells at both the quality and scale to be relevant for disease modeling and clinical application.

The challenge is that generic endothelial cells are relatively easy to create, but they lack true arterial properties and thus have little clinical value, Zhang says.

The research team applied two pioneering technologies to the project. First, they used single-cell RNA sequencing to identify the signaling pathways critical for arterial endothelial cell differentiation. They found about 40 genes of optimal relevance. Second, they used CRISPR-Cas9 gene editing technology that allowed them to create reporter cell lines to monitor arterial differentiation in real time.

With this technology, you can test the function of these candidate genes and measure what percentage of cells are generating into our target arterial cells, says Zhang.

The research group developed a protocol around five key growth factors that make the strongest contributions to arterial cell development. They also identified some very common growth factors used in stem cell science, such as insulin, that surprisingly inhibit arterial endothelial cell differentiation.

Our ultimate goal is to apply this improved cell derivation process to the formation of functional arteries that can be used in cardiovascular surgery, says Thomson, director of regenerative biology at Morgridge and a UWMadison professor of cell and regenerative biology. This work provides valuable proof that we can eventually get a reliable source for functional arterial endothelial cells and make arteries that perform and behave like the real thing.

Thomsons team, along with many UWMadison collaborators, is in the first year of a seven-year project supported by the National Institutes of Health on the feasibility of developing artery banks suitable for use in human transplantation.

In many cases with vascular disease, patients lack suitable tissue from their own bodies for use in bypass surgeries. And growing arteries from an individual patients stem cells would be cost prohibitive and take too long to be clinically useful.

The challenge will be not only to produce the arteries, but find ways to ensure they are compatible and not rejected by patients.

Now that we have a method to create these cells, we hope to continue the effort using a more universal donor cell line, says Zhang. The lab will focus on cells banked from a unique population of people who are genetically compatible donors for a majority of the population.

This article has been republished frommaterialsprovided bythe University of Wisconsin-Madison. Note: material may have been edited for length and content. For further information, please contact the cited source.

Reference:

Zhang, J., Chu, L., Hou, Z., Schwartz, M. P., Hacker, T., Vickerman, V., . . . Thomson, J. A. (2017). Functional characterization of human pluripotent stem cell-derived arterial endothelial cells. Proceedings of the National Academy of Sciences, 201702295. doi:10.1073/pnas.1702295114

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Mum shares heartbreaking photograph kissing her son goodbye after he passed away while battling acute myloid … – Metro

July 12th, 2017 4:43 pm

(Picture: Mercury Press)

On 19 June, Daniel Farbace passed away after contracting parainfluenza, while also battling acute myloid leukaemia. He was just 21 months old.

His mum, Ali Farbace, has shared a photograph of herself kissing her son goodbye as a way to celebrate his life.

His last act was to save us, says Ali, 35, explaining that by passing away, Daniel had saved his parents from the impossible decision to turn off their childs life support.

When people say things like sorry for your loss I dont really like that.

He never lost at anything we think he sacrificed himself for us.

Hes made it a lot easier for us. My feeling was that I wanted him to have quality of life, but it was stacking up against him, it wasnt fair.

We just didnt want him to suffer.

If we had had to make the decision to turn the machine off then we would never have forgiven ourselves. I do think he has saved us from that.

Ali and her husband, Dan, spent nine days with Daniels body after he died, staying to make memories with their child at Demelza Childrens Hospice in Sittingborne Kent from 19 June to 26 June.

On the ninth day, Ali and Dan brought Daniels body home to have a funeral.

It would have broken us to go home straight away with nothing, said Ali. When we got to Demelza it was just perfect. To spend that time with Daniel was lovely.

We sang, and talked to him and read to him. We got so much more time. I didnt want to leave him on his own. He was so beautiful.

He had never spent a day apart from me. He had his own bedroom, he was still a person, and it was so nice to see him without the tubes.

Our friends got to come and visit too, and we really got to say goodbye.

The night before his funeral he came home for the night. We were just glad we could all come home together. To spend our last night together was really nice.

Its cr*p, and wed do anything to have him back, but some children dont get to go home.

Despite being on life support for nine days, Daniels death still came as a surprise.

When he was born on 29 September 2015 at 26 weeks, Daniel had to be resuscitated and was on a ventilator for 16 days. He survived that, went home, but had to return to hospital in April 2016 when his parents noticed pin prick marks on his skin.

Doctors initially thought it could just be a viral rash, but soon lumps developed.

He was admitted to Great Ormond Street Hospital in April, where doctors investigated possibilities of Daniel having leukemia and neuroblastoma.

On 19 April Dan and Ali were told Daniel had leukemia, with an official diagnosis on 21 April.

It was unusual because the leukaemia was presenting as something else, said Ali.

They told us it was treatable, they gave him a 68 to 72 per cent chance he would survive.

It was just awful. Everything gets taken out of your hands. We just wanted our baby.

AML is an aggressive, rare cancer that affects the blood. Stem cells found in bone marrow produce too many immature white blood cells which are called blast cells.

The blast cells are unable to properly fight off infection, and if too many are produced the number of oxygen-carrying red blood cells and platelets, which clot the blood, can be reduced.

Symptoms of the illness include pale skin, tiredness, frequent infections, and breathlessness.

In severe cases AML can make sufferers extremely vulnerable to life-threatening infections. A bone marrow or stem cell transplant may be necessary alongside chemotherapy or radiotherapy to combat the illness.

After four courses of chemotherapy, Daniel seemed to be getting better. He was even able to go home for his first birthday.

But seven months later, the leukemia had returned, this time bringing a lump in Daniels brain with it.

After numerous chemotherapy treatments to battle theacute myloid leukaemia that had spread to Daniels blood and bone marrow, Daniel contracted parainfluenza.

Unable to fight off the infection, he spent nine days on life support before passing away.

He was on life support for nine days, there were so many times when I thought, he was going to die, said Ali.

Then the day he did die we didnt expect it. We just thought, You little b*gger.

I got to carry him across the corridor to the hospital cold room, which was an honour.

Its quite hard coping with death, he was still our baby.

We shared that picture to show the story goes on after death. People have been so supportive.

Dan said that picture looks like when they used to lay warriors to rest and Daniel was a fighter.

He was always happy and always smiling. I know it sounds crazy, but he never really seemed that unwell. When he was sick he was just quiet.

Now, Ali and Dan want to keep their sons legacy alive by using his story to raise awareness about blood and bone marrow donation.

So far, Daniel has helped to raise over 25,000 for charity.

We just want to get the message out about donating blood and bone marrow transplant now, says Ali.

Sometimes Daniel had to wait 12 hours for transfusions. But he would have died a long time ago if it hadnt have been for donors.

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Blood test holds promise for earlier pancreatic cancer detection – CBS News

July 12th, 2017 4:43 pm

Pancreatic cancer is an especially tough cancer to beat because it usually isn't detected until late in the disease. But a new blood test that uses two protein markers is showing promise for picking it up earlier, researchers report.

The blood test is still in the investigative stages. A new study, published inScience Translational Medicine, finds it works with an 87 percent sensitivity, meaning that's how often it can correctly identify someone with stage 1 or 2 pancreatic cancer. It also had 98 percent specificity, meaning the ability to accurately rule out cancer in a person who doesn't have it.

"What we found is a biomarker panel that's very cheaply, conveniently assayed in the blood and that uses conventional methods used by diagnostic centers around the country. So it could be used to detect pancreatic cancer at stages 1 and 2," study author Kenneth Zaret, director of the Penn Institute for Regenerative Medicine, told CBS News.

The 5-year survival rate for pancreatic cancer patients is only 7 percent, and it's projected to become the second leading cause of cancer death in the United States by 2020.

Zaret worked with Gloria Petersen, from the Mayo Clinic, to identify a pair of biomarkers that physicians could soon use to discover the disease earlier.

Zaret began searching for a way to detect pancreatic cancer earlier after his own mother-in-law passed away from it about 15 years ago.

At the time, scientists were primarily studying late-stage cancer tissues in the lab in order to identify biomarkers for the disease in cells, in the hopes of developing a blood test for pancreatic cancer.

"But I didn't see how that could detect early stage markers," said Zaret, whose expertise is in genetics and stem cell biology, not cancer.

"I came from outside the field and applied genetic reprogramming methods so that's where the novelty is in terms of discovery," he said.

He and colleagues took late stage pancreatic cancer cells and reprogrammed them genetically to a "stem cell-like state" what's called an induced pluripotent stem cell.

"Like an early embryonic stem cell," he said.

Then they let those cells redevelop into differentiated cell types and the cells underwent a progression from early to late stage cancer.

Using those cells, they discovered 107 proteins released from early stage pancreatic cancer cells. From there, they looked for those proteins, which could be found in very low levels in human blood and that would be easy to test for based on current blood lab technologies.

"From those we found a marker called THBS2," said Zaret.

Serum levels of THBS2 were significantly elevated in blood samples from 81 pancreatic cancer patients compared to 80 healthy people who served as the control group.

While the results were promising, "they were not powerful enough to do something statistically with," said Zaret.

So the scientists further improved the test's sensitivity and specificity by combining it with another protein, CA19-9, already being used as a clinical marker. They analyzed samples from 197 cancer patients, 140 healthy people in a control group, and 200 people with pancreatic disorders that weren't linked to cancer.

The combination THBS2 and CA19-9 ratcheted up the test's accuracy to 98 percent. They believe it could serve as a low cost way to screen higher risk people someone with a relative with the disease, or who has diabetes, for example for pancreatic cancer.

"The bottom line is, by looking at two markers and not just the single marker we increased the range of what we could detect in a person with pancreatic cancer," said Zaret.

Combining his background with the expertise of colleagues at the Mayo Clinic made the discovery possible.

"We collaborated with Dr. Gloria Petersen at the Mayo Clinic in Minnesota. She is an expert in obtaining blood samples from pancreatic cancerpatients and controlling for age, sex, and other parameters. So we had a very rigorous study. The extreme care with which we matched patients and controls is part of the strength of our work," he said.

The next step in their work is to use the test in more and more patients, specifically those at higher risk for pancreatic cancer.

"It will let us assess whether our biomarker test will allow us to detect cancer even before it is stage 1," said Zaret "stage negative 1," so to speak.

"Our big goal is to be able to detect pancreatic cancer before it's at the 13 percent survival rate, which is where a stage 1 diagnosis is now."

If that succeeds, doctors would be able to identify more patients earlier, raising hopes of achieving higher survival rates for pancreatic cancer down the road.

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Derma Divine Serum – Plant Stem Cell Skin Repair …

July 12th, 2017 4:43 pm

Stem Cell Skin Treatment

Skin care specialists around the world are turning to non-embryonic stem cells for skincare. The stem cells come from specialized plants that when applied, protect human skin stem cells from damage and deterioration. Derma Divine is a unique topical serum that repairs and protects aging skin at a cellular level. Although you can not avoid getting older, now you can reverse the appearance of aging skin! For wrinkle free, hydrated and youthful looking skin, order online today. Users notice results just after three months of consistent use. Remember, you can never be too old to feel and look young.

The number one cause of aging skin is from sun damage. UV rays and pollution deteriorate skin cells making your skin more prone to damage. Derma Divine restores damaged skin cells creating skin that is wrinkle free, hydrated and youthful. Millions are skin care specialists are turning to stem cell skin treatments instead of injections. Injections can leave scaring and is an unnatural way to repair damage. Stem cell treatments however, repair wrinkled and damaged skin at the source of the problem. This method is much more efficient, natural and effective. Order online today for a confident tomorrow.

Stem cells have the ability to develop into different cell types in the body. For skin treatment, essentially the stem cell turns into a new skin cell. The new cell can then create proteins, carbohydrates and lipids to repair wrinkles and restore firmness and elasticity back into the face. Derma Divine is a light weight serum that is fast absorbent allowing it to penetrate deep into the skin. This allows the active ingredients to repair at a cellular level. Did you know that wrinkles, age spots and dry skin are considered wounds? Stem cells repair skin leaving you with irresistibly young looking skin.

The active ingredients are the most important part of any skin care treatment. Derma Divine Anti Aging Serum is unique to others because it uses stem cell stimulation to restore youthful skin back into the face. We combined botanical extracts into the serum to support and protect new skin growth. Below is a few active ingredients within the serum:

Plant Stem Cell Rebuild damaged skin cells. Reverse appearance of aging skin. Firms, smooths & lifts damaged skin.

Vitamin C Corrects hyper pigmentation (dark spots). Helps fight skin cell damage, protects from sun damage and supports collagen production

We understand that using stem cells may come off as strange to new users. Lots of people are skeptical about new skin treatments on the market and that is okay! It is good to be skeptical in order to avoid scams. That is why we are offering new users a free trial to test the product out for themselves before purchasing. We want you to see the benefits so you are 100% satisfied with our products.

FOR BEST RESULTS PAIR WITH SKINCARE PANAMA

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Stanford’s Final Exams Pose Question About the Ethics of Genetic Engineering – Futurism

July 12th, 2017 4:41 pm

In BriefThe age of gene editing and creation will be upon us in the next few decades, with the first lifeform having already been printed. Stanford University questions the ethics of prospective students by asking a question we should all be thinking about.

When bioengineering students sit down to take their final exams for Stanford University,they are faced with a moral dilemma, as well as a series of grueling technical questions that are designed to sort the intellectual wheat from the less competent chaff:

If you and your future partner are planning to have kids, would you start saving money for college tuition, or for printing the genome of your offspring?

The question is a follow up to At what point will the cost of printing DNA to create a human equal the cost of teaching a student in Stanford? Both questions refer to the very real possibility that it may soon be in the realm of affordability to print off whatever stretch of DNA you so desire, using genetic sequencing and a machine capable of synthesizing the four building blocks of DNA A, C, G, and T into whatever order you desire.

The answer to the time question, by the way, is 19 years, given that the cost of tuition at Stanford remains at $50,000 and the price of genetic printing continues the 200-fold decrease that has occurred over the last 14 years. Precursory work has already been performed; a team lead by Craig Venter created the simplest life form ever known last year.

Stanfords moral question, though, is a little trickier. The question is part of a larger conundrum concerning humans interfering with their own biology; since the technology is developing so quickly, the issue is no longer whether we can or cant,but whether we should or shouldnt. The debate has two prongs: gene editing and life printing.

With the explosion of CRISPR technology many studies are due to start this year the ability to edit our genetic makeup will arrive soon. But how much should we manipulate our own genes? Should the technology be a reparative one, reserved for making sick humans healthy again, or should it be used to augment our current physical restrictions, making us bigger, faster, stronger, and smarter?

The question of printing life is similar in some respects; rather than altering organisms to have the desired genetic characteristics, we could print and culture them instead billions have already been invested. However, there is theadditional issue of playing God by sidestepping the methods of our reproduction that have existed since the beginning of life. Even if the ethical issue of creation was answered adequately, there are the further questions ofwho has the right to design life, what the regulations would be, and the potential restrictions on the technology based on cost; if its too pricey, gene editing could be reserved only for the rich.

It is vital to discuss the ethics of gene editing in order to ensure that the technology is not abused in the future. Stanfords question is praiseworthy because it makes todays students, who will most likely be spearheading the technologys developments, think about the consequences of their work.

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Can Genetic Engineering Put an End to Diamondback Moth Plague … – Growing Produce

July 12th, 2017 4:41 pm

Adult diamondback moth. Photo by Lyle Buss

The diamondback moth (DBM) is a pest of epic proportions to farmers worldwide. Growers of cabbage and collard crops know this all too well. Controlling the notorious DBM has become more difficult in recent times with incidence of pesticide resistance increasing. With that, the search for viable management continues to expand. For the last two years, Cornell scientists have been working with British-based biotech company Oxitec and testing its self-limiting gene capabilities on DBM in a protected environment. The team at Cornell has been waiting for a go-aheadfrom USDA to take the experiment out from behind closed doors and into one if its cabbage fields in Upstate New York. It looks like the wait is over.

USDAs Animal and Plant Health Inspection Service (APHIS) has officially announced the availability of a final environmental assessment and finding of no significant impact and will concurrently issue a permit for the field release of a genetically engineered DBM.

The pest moths are genetically engineered for repressible female lethality and to express red fluorescence as a marker. The purpose of the field release is to assess the feasibility and efficacy of these moths in reducing populations of DBM. Based on the finding of no significant impact, APHIS has determined that an environmental impact statement need not be prepared.

Notice of these actions will be published in theFederal Register.

The Northeast Organic Farming Association of New York (NOFA-NY) has since released a statement denouncing USDAs decision. A portion of the statement reads as follows: NOFA-NY considers the release of a novel genetically engineered organism to be a major activity with potentially significant and heretofore unknown health and environmental effects, said NOFA-NY Policy Advisor Liana Hoodes. It is now up to New York State Department of Environmental Conservation (DEC) to ensure the safety of its citizens before granting the necessary state permit. We call on the NYS DEC to require a full environmental impact statement and public hearings during a complete review under State Environmental Quality Review Act.

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Purple rice developed by Chinese scientists – Agri-Pulse

July 12th, 2017 4:41 pm

CHINA, July 12, 2017 - Purple rice developed by Chinese scientists. A new genetic engineering technique developed by Chinese scientists has been used to develop purple rice packed with antioxidant-boosting pigments called anthocyanins.

A study published in Molecular Plantshows how the scientists created the technique, which is capable of delivering many genes at once, and used it to make rice endosperm seed tissue that provides nutrients to the developing plant embryo. Although anthocyanins are naturally abundant in some black and red rice varieties, they are absent in polished rice grains because the husk, bran, and germ have been removed, leaving only the endosperm.

Previous attempts to engineer anthocyanin production in rice have failed because the underlying biosynthesis pathway is highly complex, and it has been difficult to efficiently transfer many genes into plants. Genetic engineering approaches have previously been used to develop rice enriched in beta-carotene and folate, but not anthocyanins.

We have developed a highly efficient, easy-to-use transgene stacking system called TransGene Stacking II that enables the assembly of a large number of genes in single vectors for plant transformation, says senior study author Yao-Guang Liu of the South China Agricultural University. We envisage that this vector system will have many potential applications in this era of synthetic biology and metabolic engineering. The researchers plan to evaluate the safety of purple endosperm rice as biofortified food and will try to engineer the biosynthesis of anthocyanins in other crops to produce more purple endosperm cereals.

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Genetically engineered salmon is coming to America – The Week Magazine

July 12th, 2017 4:41 pm

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On a hill above the cold waters around Prince Edward Island, technicians painstakingly create fertilized Atlantic salmon eggs that include growth-enhancing DNA from two other fish species. The eggs will be shipped to tanks in the high rainforest of Panama, where they will produce fish that mature far more quickly than normal farmed salmon.

More than 20 years after first seeking approval from the U.S. Food and Drug Administration, AquaBounty Technologies of Maynard, Massachusetts, plans to bring these "AquAdvantage" fish to the U.S. and Canadian markets next year. And in the small town of Albany, Indiana, workers will soon begin converting a land-based aquaculture facility to produce about 1,300 U.S. tons of these salmon annually, in the first U.S. facility to generate GE animals for human consumption.

The company also plans to open a second aquaculture facility at Prince Edward Island if it can rise above its latest round of legal battles and persuade grocery stores and restaurants to snap up the genetically engineered fish. Before the FDA cleared the salmon for consumption in 2015, in its first approval of GE animal protein as human food, it received 1.8 million messages opposing these fish. Perhaps more substantively, many outside researchers remain concerned about AquaBounty's plans.

Safety and nutrition

Aquaculture specialists generally aren't skeptical about whether the fish will be healthy to eat, although that's one issue hinted at in a lawsuit multiple organizations, including Friends of the Earth, have filed against the FDA. Dana Perls, senior food and technology campaigner with Friends of the Earth in Berkeley, California, says the FDA didn't fully examine questions about eating the salmon initially raised by Health Canada, that country's public health department including susceptibility to disease and potential allergic reactions.

"This is a poorly studied, risky, and unlabeled genetically engineered fish," she says, adding that more than 80 U.S. grocery chains have committed not to buy it. However, Health Canada eventually concluded that fillets derived from AquAdvantage salmon "are as safe and nutritious as fillets from current available farmed Atlantic salmon," and approved the fish for consumption in 2016.

"There's no reason to suspect these fish from a food safety perspective," says Cyr Couturier, chair of aquaculture programs at Memorial University's Marine Institute in St. John's, Newfoundland. "They have no unnatural products that humans wouldn't otherwise consume."

Similar transgenic salmon created by a decades-long Fisheries and Oceans Canada research program tested well within normal salmon variations, adds Robert Devlin, engineering research scientist at the agency in North Vancouver, British Columbia. But critics do raise two other main concerns about AquaBounty's quest: the economic sustainability of the land-based approach, and the environmental risk to ecosystems if the fish escape.

Fish on land

AquaBounty will raise its GE fish in land-based recirculating aquaculture systems, known as RAS basically huge aquaria designed to minimize water use, maximize resources and accommodate high stocking densities. "While farming salmon in sea cages is less expensive and less technologically complex than a land-based farm," the company's website points out, "sea cages are susceptible to a number of hazards such as violent storms, predators, harmful algal blooms, jellyfish attacks, fish escapes, and the transmission of pathogens and parasites from wild fish populations."

Given the potential opportunity to achieve greater production control and avoid some of the environmental concerns of sea farms, many RAS projects have launched around the world in the past decade. However, most of these projects are small, and many have failed or are struggling.

The big problem is cost. RAS facilities need much more capital than ocean farms with similar production rates, and they're expensive to operate.

"Land-based systems use a lot of freshwater, even though it's recirculated, and a lot of electricity," notes Couturier. Such systems "operate at an economic disadvantage because much of their cost goes toward creating growing conditions occurring naturally within the ocean," summed up one 2014 report that found producing Atlantic salmon in Nova Scotia would not be economically feasible.

AquaBounty, which is buying its Indiana plant from a collapsed RAS venture, expects to beat these odds mainly because its GE salmon reach market size in about half the time of normal farmed salmon in 1618 months rather than 2836 months, the company says. Ravenous as they are, with their growth hormones continually wired on, the fish still require about a quarter less feed than normal fish. (Although farmed salmon are very efficient at converting food to flesh a pound of feed converts close to a pound of flesh feed remains a major expense.)

The company also says that salmon in its RAS facilities won't need vaccines or antibiotics because it will tightly control conditions. However, "they will have some disease issues of course, as will any animal that's reared in high densities," Couturier predicts.

If AquaBounty can compete on cost, there will be some justification for promoting its product as "the world's most sustainable salmon." In addition to requiring less feed, growing fish in Indiana or Prince Edward Island can slash the high carbon costs of flying fish from Norway or Chile, two leading suppliers of farmed salmon in the U.S.

Still, says Couturier, "I wish them all the best, but I think it will be a small-scale niche for at least a decade."

Losing GE fish

Many aquaculture scientists remain uneasy about the environmental risk to wild ecosystems if transgenic fish slip out of their farms. Although other agencies will presumably be involved in assessing risk as the projects advance, "the FDA has no in-house capacity to evaluate or understand the ecological consequences of transgenics in an aquatic ecosystem," says Conner Bailey, professor emeritus of rural sociology at Auburn University in Alabama. "And once you get anything into an aquatic ecosystem, it's really hard to control."

AquaBounty's protection scheme begins with multiple levels of physical barriers in its RAS facilities. Additionally, the salmon are all female and "triploid" (their DNA is in three rather than two sets of chromosomes) so they can't reproduce. However, scientists say neither of these measures can be 100 percent effective at preventing transgenic fish from escaping, disrupting local ecosystems, and potentially breeding in the wild.

More generally, while AquaBounty is committed to land-based systems, there are concerns that it's also creating far more GE eggs than it needs for its own production. Other industry groups, such as the Atlantic Salmon Federation, worry that other producers AquaBounty sells to might not be so careful, or that other companies around the world might move ahead with similar projects but without the same precautions. And all bets on risk are off if GE fish are raised in the ocean, where fish routinely escape, sometimes in large numbers.

Devlin's group has extensively modeled the results of accidental releases, studying groups of transgenic and non-transgenic fish in "naturalized" aquatic test beds that are exposed to variations in conditions, such as food supply. Transgenic fish often behave quite differently, and the results have varied from peaceful coexistence to one experiment in which fully transgenic fish killed off all their competitors.

"In the multitude of different environments that exist in nature, the uncertainty is too great to make a reliable prediction of what the impact would be," he says.

GE or selective breeding?

Does the fast growth of AquAdvantage salmon justify taking on these unknown risks? Scientists point out that today's selective breeding research programs, built on genomics and other tools of modern biology, also have turbocharged fish development. "Some strains of rainbow trout, which have been selected for fast growth for 150 years, grow incredibly fast compared to wild-type fish," Devlin says. In fact, he says, his lab work across various species suggests that "the absolute fastest growth you can achieve either by domestication or by transgenesis seems to be very similar."

"Today's farmed salmon have had more than 10 generations of selection applied to them, and they are growing at more than double the rate compared to the 1970s," says Bjarne Gjerde, senior scientist at Nofima in Troms, Norway.

Farmed fish also must excel in many traits besides growth, such as disease resistance and food quality, he emphasizes. "Most of the traits we are breeding for are governed by many, many genes with small effects," he says. "That's a real challenge if you just want to take short cuts with genetic engineering."

When and if AquaBounty rises above all its challenges into a groundbreaking success in North America, the firm will send a signal around the world to unleash efforts for commercializing GE fish, observers say. Friends of the Earth's Perls remains hopeful that legal barriers and consumer boycotts will stop AquaBounty in its tracks. If not, "GE salmon could set a precedent to the approval of other GE animals in the pipeline, from fish to chickens, pigs, and cows," she says. "It is critical that we don't approve other GE animals without robust regulations and full environmental reviews to ensure that we're prioritizing human and environmental safety over profit."

"Fish are probably where transgenic animals will emerge, because it's much cheaper to maintain a herd of catfish or salmon than cattle or sheep or pigs," says Bailey.

This story was first published by Ensia, an environmental news magazine from the University of Minnesota.

This article originally appeared at PRI's The World.

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Clinical trial for stem-cell therapy to reverse liver cirrhosis – The Straits Times

July 11th, 2017 9:50 am

SINGAPORE - The use of stem cells to reverse liver cirrhosis - or the hardening of the liver - is being explored in a clinical trial.

Conducted by a multi-centre team led by the National University Hospital (NUH), doctors aim to determine if stem cell therapy can improve liver function.

Previously, liver cirrhosis, caused by various diseases such as chronic hepatitis B and non-alcoholic fatty liver disease, was thought to be irreversible.

A liver transplant provides a definitive cure to end-stage cirrhosis.

However, in Singapore, less than 5 per cent of end-stage liver cirrhosis patients receive a liver transplant.

The number of people on the waiting list for a liver transplant has been increasing over the years, according to statistics from the Ministry of Health.

In 2007, there were nine on the waiting list, compared with 57 last year. There are around 50 waiting for a liver transplant this year.

Also, many patients do not fulfil the eligibility criteria to receive a liver transplant due to other health complications or being above the age limit of 70 years.

The $2.6 million study, which was launched on Tuesday (July 11),is funded by the National Medical Research Counciland 46 patients will be recruited for it. It will run for four years and patients will not need to bear the costs of the stem cell treatment.

Stem cells will be taken from a patient's own bone marrow and will be isolated and injected directly into the patient's liver to initiate the repair.

Similar therapy treatments have been conducted overseas in countries such asEgypt and India, although they have not been fully evaluated for efficacy.

Associate Professor Dan Yock Young, a senior consultant in the division of gastroenterology and hepatology at NUH, said: "We are conducting the study in a systematic and scientific mannerto get definitive evidence of the effects of the treatment."

He also notes that the stem cell therapy is not a substitute for a liver transplant. "This treatment is not intended to pull patients off the waiting list, but provide an option for those who are not eligible for a transplant."

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Clinical trial for stem-cell therapy to reverse liver cirrhosis - The Straits Times

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Researchers are closer to working capillaries in 3D-printed organs – Engadget

July 11th, 2017 9:50 am

All of our blood vessels are lined with a type of cell called endothelial cells. To form vessels, individual endothelial cells begin to create empty holes in themselves, called vacuoles. They then connect with other endothelial cells that have done the same thing and the linked vacuoles form tubes, which ultimately become capillaries. Here, the researchers took endothelial cells and mixed them with either fibrin -- a protein involved in blood clotting -- or a semi-synthetic material called gelatin methacrylate (GelMA), which can be easily 3D-printed. When mixed with fibrin, the endothelial cells formed tubes fairly easily, but that wasn't the case with the GelMA. However, when the researchers added in another type of cell, a stem cell found in bone marrow, the endothelial cells were then able to form tubes in the GelMA.

"We've confirmed that these cells have the capacity to form capillary-like structures, both in a natural material called fibrin and in a semi-synthetic material called gelatin methacrylate, or GelMA," Gisele Calderon, the lead author of the study, said in a statement, "The GelMA finding is particularly interesting because it is something we can readily 3D print for future tissue-engineering applications."

The benefits of this method over others include cells that can be patient-specific, reducing the risk of immune system complications, and growth environments that are well suited for organ and vasculature growth -- they're reproducible, not likely to induce immune responses and help boost cell growth and vessel development. Along with making 3D-printed organs more viable, this method will also allow for the development of tissue that could make for more effective and efficient drug testing. In a statement, Jordan Miller, whose lab the work was done in, said, "Preclinical human testing of new drugs today is done with flat two-dimensional human tissue cultures. But it is well-known that cells often behave differently in three-dimensional tissues than they do in two-dimensional cultures. There's hope that testing drugs in more realistic three-dimensional cultures will lower overall drug development costs."

You can watch a video of the cells beginning to form tubes here and Calderon explaining her work in the video below.

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Bone marrow transplant last chance – Revelstoke Review

July 11th, 2017 9:50 am

Fear becomes a constant companion when ones child is desperately ill.

Kim Lahti-Scranton knows this all too well as her daughter Jane is fighting for her life at BC Childrens Hospital.

As much as Janes journey is all-consuming, Kim is helping a desperate woman to get young people to donate bone marrow in order to find a better match for her son, Noah, who is in critical need of a bone marrow transplant soon.

The Scranton family is living in Vancouvers Ronald McDonald House and shares a kitchen with 17-year-old Noah Stoltes family.

Noah survived a first round of cancer when he was 10. In April, the family was given the terrifying news that he had relapsed.

His mom thought they were going to find a match within family; theyre 100 per cent Dutch, says Kim, noting family members are usually the best choice, but his three sisters are only a 50 per cent match.

Out of the worldwide pool of donors, there are two people who are a 75 per cent genetic match and are being considered, one of whom lives in Europe. The lower the match, the more chance there is for complications and a less than optimal outcome.

There is a time crunch as Noah needs to have a transplant sometime in September and it can take a couple of months from registration to donation.

One of Kims nephews is an NHL player, who used his connections to help Noahs aunt interest 85 people in becoming bone marrow donors in Edmonton.

Unable to get help from Canadian Blood Services to organize a similar drive in Vancouver because of staffing issues, Kim and Noahs mother, Stacey VanderLee Stolte, went to a Vancouver Canadians game to raise awareness and hopefully encourage people to sign up to be on the registry for stem cell or bone marrow donation.

We were met with a whole range of responses from people who were incredibly receptive, to people who completely ignored us and everything in between, says Kim. We got some interesting comments like no kid would want my bone marrow, Ive done too many drugs, to other people saying no because they thought if they were a match that donating bone marrow would be too painful.

Dr. Lucy Turnham, a clinical associate who oversees the outpatient oncology clinic at BC Childrens Hospital, has performed Janes procedures and says males between the ages of 17 and 35 are the best resource.

Its one of the most wonderful charitable donations you can do, Turnham says, noting many people never receive a call. It has a huge impact on a patient who has no other options.

Turnham explains that donors are educated before they do a swab. If they are told they are a match, they can still say no at any time. They then undergo further testing and maybe counselling at that point, and can still back out an any time.

If they had said yes and the patient begins treatment to get rid of the diseased cells prior to transplant, and then the donor backs out, the patients life is at serious risk, so it is important to know what you are getting into before saying yes, Kim stresses. If the donor says yes to the procedure, they are told beforehand when the treatment would start and told when the last chance to back out would be.

Stem cell donations can be made in two ways: the donor is hooked to a machine with IVs in each arm. Blood is taken from one arm, stem cells are removed, the blood is replaced through the IV in the other arm where the body grows more stem cells.

Or, the donor is anesthetized, a needle is placed into the hip bone from the back and part of the bone marrow is sucked out.

You might be a little bit sore afterwards, but for less than 24 hours, Turnham says, noting women can be donors but not if they have been pregnant. Young bone marrow is more robust and we regenerate marrow and blood all the time.

Becoming a donor is not a speedy process for people living in smaller communities, but would-be donors can receive a swab kit through the mail and return it postage-free.

Marc Plante, a representative with the national office of Canadian Blood Services, says people can go to http://www.blood.ca and proceed to the Stem Cells tab at the top of the page.

If you have an opportunity to save a life and just put up with a couple of days of discomfort, I would do it in a heartbeat, says Kim, who must also face the reality that Jane could one day be in the same dilemma. If most people had the opportunity to save a life, they would do it.

The Scrantons have been living at Ronald McDonald House for several months, while Jane receives treatment.

Shes as good as can be expected; shes very compromised and we need to make sure shes not exposed to anything, says Kim, noting the first, and this, the fourth phase of treatment, are considered to be the toughest. She has nothing to fight off infection.

There is another terrifying aspect to this disease.

As well as living with the fear of potential relapse, some of the chemo drugs being used to treat Jane increase the risk of heart disease and cause secondary cancer, neuro-cognitive issues and behavioural issues.

We dont know if its coming or what it will be, but we dont have a choice, we have to save her life now and deal with the consequences later, Kim says. Youre just kind of waiting for the other shoe to drop, for things to take a turn, its not fun. And while Jane will require a lot of follow-up assessment in the years to come, the tough little hero turns six on Aug. 12.

She has everything she needs; I just want people to sign up for the bone marrow registry or donate blood, Kim says, noting she is grateful for the communitys support throughout the ordeal.

If you cant donate but would like to help Jane celebrate, you can take birthday cards to the Salmar Grand Theatre where manager Daila Duford will make sure they get to their destination.

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Ashli Stempel helped save her brother’s life. She hopes to inspire others. – GazetteNET

July 11th, 2017 9:50 am

A few small scars on Ashli Stempels lower back are the only evidence that a drill burrowed into her hipbone last year at Brigham and Womens Hospital in Boston. The surgery was to harvest the stem cells in her bone marrow to save her older brother Andrew Stempels life.

At age 27, Andrew was diagnosed with cancer of the white blood cells called Hodgkins lymphoma. Donating her bone marrow so that Andrews body could manufacture healthy blood cells, was a small price to pay to give him a shot at survival, Ashli says.

Since the transplant last August after years of treatment and testing Andrew has been cancer-free and Ashli now volunteers periodically in their hometown of Greenfield, where she serves on the Town Council, to spread awareness about this life-saving treatment.

Our bodies are a cure for some cancers, says Ashli Stempel on a recent Saturday as she handsout sign-up forms atGreenfields Energy Park for the Be A Matchnational donor registry. If even one person joins the registry that is awesome.

Its a sunnyday and Stempel, 30,wearing a black and white spaghetti-strap dress stands behind a booth smiling and talking to passersby.

Everybody wants to cure cancer, but I think not everybody understands that we, ourselves, can be the cure for some types of cancers, she says. I can say that I killed cancer and I am pretty excited about that.

In the hollow spaces in a bodys bones, stem cells inside the bone marrow tissue work to create red blood cells, which feed oxygen to the organs, and make white blood cells to fight infections. The bone marrow also produces blood platelets to help form clots but when a cancer of the blood like, leukemia or lymphoma strikes, these life-supporting systems are thrown out of whack, leaving the bodys immune system unable to fight diseases, infection or the cancer.

Chemotherapy and radiation also can kill off bone marrow tissue, leaving patients with more damage to their immune systems, says physician assistant Susanne Smith, donor services clinician at Dana-Farber/Brigham and Womens Hospitals Cancer Center in Boston.

When transplanted into a cancer patients bloodstream, stem cells, a precursor to all the immune system cells in the body, colonize the bones and help fight any remaining cancer, says Smith.

In many cases (a transplant) is the only cure for a leukemia or lymphoma diagnosis chemotherapy can only get a patient so far, says Mary Halet, director of community engagement at the Be The Match Registry, the Minneapolis organization that manages the largest bone marrow registry in the world. But first, a patient must find a tissue match, that is, a donor who has a similar protein marker called the human leukocyte antigen, which is found on most cells in the body.

There are up to 14,000 patients every year who could benefit from a bone marrow transplant, but many of these people will not receive a donation, says Halet. In most cases, the patient will not finda tissue match in his or her own family andmust seek help from a stranger, she says. A patients likelihood of finding a matching bone marrow donor ranges from 66 percent to 97 percentdepending on ethnic background. White patients have a 97 percent chance of finding a match, while black patients only find a match 66 percent of the time.The difference reflects the complexity of the tissues makeup and the number of donors.

Thats why Halets organization promotes recruitment events like the one Ashli Stempel held in Greenfield.

Stempel says she was ecstatic when she found out that she was a match for her brother. She was in her late 20s at the time, a bubbly woman working in communications at Smith College in Northampton, who grew up in a close-knit family.

Her brother, who was working as a retail manager in the Boston area, had discovered a bump on his collarbone.

I woke up one morning and there was a non-painful lump, Andrew Stempel says.

He ignored it for as long as he could before seeing a doctor who diagnosed it as a swollen lymph node caused by Hodgkins lymphoma.

Cancer is a very scary word. I think what you learn going through it is that it is not such a scary word, you can survive, says Ashli Stempel.

The Stempel family had seen that firsthand years earlier when Andrew and Ashlis mother, Deborah, recovered from breast cancer.

Still, that didnt lessen the anxiety for Andrew. As soon as the doctor said the word cancer, he says, his life started to unravel with a battery of experimental drugs, chemotherapy and radiation.

In the begining there was a lot of uncertainty, he says.

Even through his cancer went into remission after a year, doctors did not expect it to remain that way without high doses of chemo or radiation. The plan was to do a bone marrow transplant for long-term survival.

Still, using donated bone marrow meant taking the risk that Andrews body would reject it, which could be fatal.

So, doctors first wanted to try using Andrews own tissue. That would require removing some of his bone marrow, treating it and then injecting it back into his bloodstream.

Within months of the procedure, however, Andrews cancer returned, indicating to doctors that his body wasnt strong enough to fight it on its own.

Ashli was tested via a mouth swab and Andrew was relieved to learn that she was a tissue match.

I was just overwhelmed with happiness, he says.

Ashli went through a month-long screening process to ensure that she was healthy enough to be a donor. People who have infectious diseases like HIV or hepatitis cannot be donors, nor can those with immune systems weakened by autoimmune diseases. Doctors also prefer to use bone marrow from young donors under the age of 44, says Halet. The registry wont accept donors over 60.

When we are young, our immune systems are at their healthiest and the older we get the less robust they are, she says.

It took two years from the time Ashli first learned she was a match for her brother for the transplant to take place.

Not long aftershe woke up from the surgery, Ashli saw the bone marrow that had been taken from her, a two-literjug ofmilky, red liquid. It was whisked away to another partof the hospital where it ended up in a drip bag connected to a vein in Andrews arm.

Doctors saw hisred and white blood cell counts go up immediately after the transplant.

My sisters cells were working, he says. It was amazing.

Even though the transplant was a success, Andrew had to stay in the hospital for a month. Chemotherapy had caused sores in his mouth, he lost his ability to taste food along withhis appetiteand he droppednearly 30 pounds.

It was tough, day to day, but progressively got better, he says.

Since he was essentially receiving a new immune system, like a newborn baby he also had to be shielded from germs, says Ashli.

When his wife, Meghan Stempel, came to visit him, she needed to wear a facemask and gloves. Even when he returned home, he had to be careful. Hetook a year off from his job to recover, spending many afternoons resting on the couch watching TV. After spending months working to building hisstrength back up,he says, most of his weakness has subsided.

I feel a thousand times better, he says.

He is now cancer free and is returning to hisjob as a retail managerat Sherwin Williams this week.

Following her operation, Ashli took off a few weeks from her job in communications at Smith College, but was back on her feet within a couple days. Her hips were sore which meant limping around the house for a short time.

I was in pain, of course, she says. But its a quick recovery.

A few weeks ago Ashli decided to signup for the national bone marrow donor registry through Be A Matchto donate for a second time.

Her name will stay in the system for the foreseeable future. A match could come up or it might never.

Maybe I will be called on to do it again, who knows?

To learn more about becoming a bone marrow donor or to sign up for the registry, go tobethematch.org.

Potential donors can fill out an online form and the registry will mail a mouth swab kit, which can be returned by mail.

If called, a potential donor will undergo a series of blood tests which will evaluate the suitabililty and safety of the match. Though doctors say risks are low for donors, possible complications include infection and bleeding.

Once a donor is cleared, the transplant procedure could occur within a few weeks or a few months, depending on a recommendation from the patients doctor.

The bone marrow transplant is an outpatient procedure for the donor.Recovery time is only a few days anddonors are typically back to their normal routine in two to seven days.

Donors are told their commitment means being willing to devote up to 30 hours spread over four to six weeks to attend appointments and give the donation.

All medical costs for the donation procedure are covered by the National Marrow Donor Program, which operates the Be The Match Registry, or by the cancer patients medical insurance.

Sometime travel is required. Most travel expenses are covered by Be The Match.

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First gene therapy ‘a true living drug’ on the cusp of FDA … – Washington Post

July 11th, 2017 9:49 am

PHILADELPHIA When doctors saw the report on Bill Ludwigs bone-marrow biopsy, they thought it was a mistake and ordered the test repeated. But the results came back the same: His lethal leukemia had been wiped out by an experimental treatment never used in humans.

We were hoping for a little improvement, remembers the 72-year-old retired New Jersey corrections officer, who had battled the disease for a decade. He and his oncologist both broke down when she delivered the good news in 2010. Nobody was hoping for zero cancer.

The pioneering therapy with Ludwig and a few other adults at the University of Pennsylvania hospital paved the way for clinical trials with children. Six-year-old Emily Whitehead, who was near death, became the first pediatric recipient in 2012. Like Ludwig, she remains cancer-free.

Such results are why the treatment is on track to become the first gene therapy approved by the Food and Drug Administration. An FDA advisory committee will decide Wednesday whether to recommend approval of the approach, which uses patients own genetically altered immune cells to fight blood cancers.

If the panel gives the nod, the agency probably will follow suit by the end of September. That would open the latest chapter in immunotherapy a true living drug, says Penn scientist Carl June, who led its development.

The CAR T-cell treatment, manufactured by the drug company Novartis, initially would be available only for the small number of children and young adults whose leukemia doesnt respond to standard care. Those patients typically have a grim prognosis, but in the pivotal trial testing the therapy in almost a dozen countries, 83 percent of patients went into remission. A year later, two-thirds remained so.

And childhood leukemia is just the start for a field that has attracted intense interest in academia and industry. Kite Pharma of Santa Monica, Calif., has applied for FDA approval for aggressive non-Hodgkin lymphoma, and a similar Novartis application is close behind. Researchers also are exploring CAR T-cell therapys use for multiple myeloma and chronic lymphocytic leukemia, the disease that afflicted Ludwig. Theyre also tackling a far more difficult challenge using the therapy for solid tumors in the lungs or brain, for example.

The excitement among doctors and researchers is palpable. Were saving patients who three or four years ago we were at our wits end trying to keep alive, said Stephen Schuster, the Penn oncologist who is leading a Novartis lymphoma study. Both the study and a Kite trial have shown that the treatment can put about one-third of adults with advanced disease those who have exhausted all options into remission.

Yet along with the enthusiasm come pressing questions about safety, cost and the complexity of the procedure.

It involves extracting white blood cells called T cells the foot soldiers of the immune system from a patients blood, freezing and sending them to Novartiss sprawling manufacturing plant in Morris Plains, N.J. There, a crippled HIV fragment is used to genetically modify the T cells so they can find and attack the cancer. The cells then are refrozen and sent back to be infused into the patient.

Once inside the persons body, the T-cell army multiplies astronomically.

Novartis hasnt disclosed the price for its therapy, but analysts are predicting $300,000 to $600,000 for a one-time infusion. Brad Loncar, whose index fund focuses on cancer immunotherapy treatment, hopes the cost doesnt prompt a backlash. CAR-T is not the EpiPen, he said. This is truly pushing the envelope and at the cutting edge of science.

The biggest concerns, however, center on safety. The revved-up immune system becomes a potent cancer-fighting agent but also a dangerous threat to the patient. Serious side effects abound, raising concerns about broad use.

Treating patients safely is the heart of the rollout, said Stephan Grupp of the Childrens Hospital of Philadephia, who as director of its Cancer Immunotherapy Program led early pediatric studies as well as Novartiss global trial. The efficacy takes care of itself, but safety takes a lot of attention.

One of the most common side effects is called cytokine release syndrome, which causes high fever and flulike symptoms that in some cases can be so dangerous that the patient ends up in intensive care. The other major worry is neurotoxicity, which can result in temporary confusion or potentially fatal brain swelling. Juno Therapeutics, a biotech firm in Seattle, had to shut down one of its CAR T-cell programs because five patients died of brain swelling. Novartis has not seen brain swelling in its trials, company officials said.

To try to ensure patient safety, Novartis isnt planning a typical product rollout, with a drug pushed as widely and aggressively as possible. The company instead will designate 30to 35 medical centers to administer the treatment. Many of them took part in the clinical trial, and all have gotten extensive training by Grupp and others.

Grupp said he and his staff learned about the side effects of CAR T-cell therapy and what to do about them through terrifying experience that began five years ago with Emily Whitehead.

The young girl, who had relapsed twice on conventional treatments for acute lymphoblastic leukemia, was in grave condition. Grupp suggested to her parents that she become the first child to get the experimental therapy.

I said, Surely, this has been tried on kids somewhere else in the world, recalled her father, Thomas Whitehead of Philipsburg, Pa. But Steve said, Nope, some adults got it, but that was a different kind of leukemia.

After getting the therapy, Emilys fever soared, her blood pressure plummeted, and she ended up in a coma and on a ventilator for two weeks in the hospitals intensive care unit. Convinced his patient would not survive another day, a frantic Grupp got rushed lab results that suggested a surge of interleukin 6 was causing her immune system to relentlessly hammer her body. Doctors decided to give Emily an immunosuppressant drug called tocilizumab.

She was dramatically better within hours. She woke up the next day, her 7th birthday. Tests showed her cancer was gone.

The approval of CAR T-cell therapy would represent the second big immunotherapy advance in less than a decade. In 2011, the FDA cleared the first agent in a new class of drugs called checkpoint inhibitors. It has approved four more since then.

There are big differences between the two approaches. The checkpoint inhibitors are targeted at solid tumors, such as advanced melanoma, lung and bladder cancer, while CAR-T cell therapy has been aimed at blood disorders. And although checkpoint inhibitors are off the shelf, with every patient getting the same drug, the other is customized to an individual. Many immunotherapy experts think the greatest progress against cancer will occur when researchers figure out how to combine the approaches.

For the Penn team, the CAR T-cell story goes back decades, starting at the then-National Naval Medical Center in Bethesda, where June and a postdoc fellow named Bruce Levine worked on new HIV treatments. In the process, they figured out a way to turbocharge T cells to make them more powerful and plentiful.

The pair moved to Philadelphia in 1999 and dove into cancer research. Two years later, Junes wife died of ovarian cancer, something he has credited as spurring him to work even harder in the field. In the years that followed, researchers across the country, including at Memorial Sloan Kettering Cancer Center in New York and Fred Hutchinson Cancer Research Center in Seattle racked up an array of tantalizing discoveries involving T cells.

Fast-forward to 2010, when Ludwig, who lives in Bridgeton, N.J. became Penns first patient to receive CAR T-cell therapy. Two other men got the treatment not long after. One is still in remission; the other relapsed and died.

But after those three patients, the Penn researchers ran out of money for more treatments. To try to raise interest and funding, they decided to publish the results of their work. The article that appeared in the New England Journal of Medicine in August 2011 created a firestorm, June said one that brought them new resources. David Porter, a Penn oncologist working with June, was on vacation in western Maryland and had to stop at a Kohls to buy a dress shirt for the immediate TV interviews.

The pediatric trial opened the following spring with Whitehead. Six months later, Penn licensed its technology to Novartis in exchange for financial support, which included a new cell-manufacturing facility on campus.

With FDA approval seeming imminent, the researchers who were so instrumental in the therapys development and testing are almost giddy. Grupp is especially pleased that the advance will be available first to children. Usually everything is developed first for adults, he noted recently, and children are an afterthought.

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Gene therapy in hemophilia advances with big drops in patient bleeding rates – STAT

July 11th, 2017 9:49 am

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Gene therapy in hemophilia advances with big drops in patient bleeding rates - STAT

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Pioneering gene therapy patients stay on track, boosting Spark’s … – Endpoints News

July 11th, 2017 9:49 am

After rattling investors with early signs of an immune reaction in a couple of cases as well as an unexpected infusion for a suspected knee bleed, Spark Therapeutics $ONCE says that its early Phase I/II study for its hemophilia B gene therapy is staying on track, slashing the rate of annual infusions and the bleed rate among the 10 patients on the pioneering therapy SPK-9001.

The annual infusion rate has new dropped 99%, down to a mean of 1 compared to 67.5 ahead of treatment. Five of the 10 are now past the one-year mark since their treatment, with no bleeding issues. The group of 10 posted an average bleed rate of 0.4 compared to 11.1 ahead of once-and-done therapy.

Singling out the first patient, whos now past the 18-month mark, researchers say hes had zero bleeds with no Factor IX infusions. Both cases of elevated liver enzymes indicating an immune response to the delivery vector were resolved with steroids and neither have had bleeds or the need for infusions.

Spark has now accumulated close to 10 years of patient responses to its therapy, an important first step in laying out the potential for gene therapy to end hemophilia.

The latest update arrived at a scientific conference in Berlin marked by the dramatic showdown between Shire and its rival Roche, which fielded more newly contested boasts about its would-be hemophilia blockbuster emicizumab. Alnylam and Sanofi also stepped up with a promising look at the latest Phase II data on their RNAi approach underscoring some significant gains in the field for a variety of new approaches to the rare blood disorder.

We continue to be encouraged by the SPK-9001 clinical trial results observed to date, with all participants having discontinued routine infusions of factor IX concentrates, said Katherine A. High, M.D., president and chief scientific officer at Spark Therapeutics. The growing body of data showing a sustained response is a promising sign for this investigational hemophilia B gene therapy program.

Full-text daily reports for those who discover, develop, and market drugs. Join 17,000+ biopharma pros who read Endpoints News by email every day.

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Pioneering gene therapy patients stay on track, boosting Spark's ... - Endpoints News

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