StemCell Therapy

Rheumatoid arthritis (RA) isn't the same as grandma's stiff joints (aka osteoarthritis). It's an autoimmune disease that causes a persons immune system to attack their joints by mistake, causing pain and swellingand it most often shows up between the ages of 30 and 50, per the American College of Rheumatology.

RA isnt super common among all cases of arthritisabout 1.3 million people are affected by it, according to the American College of Rheumatology. But for those who do have it, it can be debilitating. Heres what you need to know.

We dont really know the cause, even to date, Orrin Troum, M.D., a rheumatologist at Providence Saint John's Health Center in Santa Monica, California, tells SELF. What we do know is that it's an autoimmune disease and that there is evidence that it runs in families. According to Dr. Troum, there are some factors that have been linked to RA, including smoking cigarettes, poor dental hygiene, and genetics.

Your genes dont actually cause you to develop rheumatoid arthritis, but they can make you more susceptible to environmental factors, like bacterial or viral infections, that could potentially trigger the disease, according to the Mayo Clinic. In other words, having a genetic predisposition doesn't mean that you'll get RA; rather, it means you inherited genetic variations from your parents that could make it harder for your body to fend off infection (which can then contribute to the development of a disease).

People tend to associate the word arthritis with osteoarthritis, which causes joint inflammation that typically comes from the wear and tear of age. But rheumatoid arthritis is an immune condition that impacts the lining of a person's joints. In some patients, RA can impact the skin (via noduleslumps that form beneath the skinand rashes), cause inflammation in the lungs and whites of the eyes, lead to plaque buildup in the heart and an increased risk of heart attack and stroke, and damage the blood vessels, according to the Arthritis Foundation.

Rheumatoid arthritis starts in a persons joints, but it can be felt all over. Symptoms typically include joint pain, tenderness, swelling, or stiffness (especially in the morning), according to the Arthritis Foundation. People can also experience fatigue, loss of appetite, and a low-grade fever, the organization says.

While you have pain in your joints, your whole body [feels] sick, Marcy OKoon, senior director of consumer health at the Arthritis Foundation, tells SELF. People just feel ill. They have fatigue because they dont sleep well due to the pain. The first symptoms tend to happen in the joints of the hands and feet and then spread to other areas of the body, Reddog Sina, D.O., assistant professor in the department of family and community medicine at Michigan State University, tells SELF.

Depending on how severe their condition is, people with RA can feel fine one day and terrible the next. These flare-ups can be unpredictable and debilitating, the Arthritis Foundation says. Some flare-ups have known triggers, like overexerting yourself and having swollen joints the next day, but others have no known cause.

About 75 percent of patients in the U.S. with RA are women, according to the American College of Rheumatology, and 1 to 3 percent of women may get RA in their lifetime. This is true for several other autoimmune conditions like lupus and thyroid disease, Dr. Troum says, and experts arent sure why it happens. Women with RA tend to feel better during pregnancy, only to have a flare-up afterward, O'Koon says. It may have something to do with hormonal differences, but we just dont know, Dr. Troum says.

Since rheumatoid arthritis can get worse with time, early diagnosis is key, Dr. Troum says. But diagnosis can be difficult. Often, patients arent recognized as having rheumatoid arthritis at first and are treated with over-the-counter medications, he says. It's not uncommon for RA patients to originally be misdiagnosed with osteoarthritis, Dr. Troum says.

Once a person receives a diagnosis, verified through a blood test and sometimes an X-ray, theyre often put on disease-modifying antirheumatic drugs (DMARDS) which can stop the progression of the disease and prevent disability, Dr. Troum says. Typically patients stay on the drugs for life, but they dont have a positive effect on everyone. Its very individually dependent. Some people function incredibly well with medication their entire lives, and others dont, Dr. Sina says. Getting good sleep, eating a healthy diet, and trying to decrease stress may also help people manage their RA, Dr. Troum says.

If youre diagnosed with RA, know this: You can still have a good, active life with proper care. Most of my patients lead normal, healthy, productive lives, Dr. Troum says.

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6 Things You Need to Know About Rheumatoid Arthritis - SELF

The Gorilla NC Fusion Plating System is the latest addition to Paragon 28's robust foot and ankle specific portfolio. The system is made up offour plates, left and right specific, ranging from 28 35.5 mm in length to span the Naviculocuneiform joint. The plates providefive different screw options for compression across multiple joints. The patent-pending NC Plate with PRECISION Guide technology allows for a crossing screw that passes from the medial cuneiform to the lateral aspect of the navicular. A locking screw in the plate allows for fixation between the proximal medial navicular to the intermediate cuneiform. A second locking screw in the navicular helps guard against plantar gapping, while two distal screws in the medial cuneiform have the ability to be placed across one, two or three cuneiforms. All plates are anatomically contoured for optimal fixation and low profile to minimize prominence and avoid soft tissue irritation.

The system also includes templates to facilitate proper plate placement and screw alignment across each joint. The patent-pending NC Fusion PRECISION Guide provides three options for the crossing screw trajectory to account for variations among patient anatomy and can accommodate a 3.5 mm, 4.0 mm, 4.5 mm or 5.5 mm cannulated cross screw. Compression can be achieved by use of a partially threaded cross screw or with external compression using a fully threaded cross screw.

Key Features and Benefits of the System:

Guided Screw Targeting simplifies screw placement and helps to ensure fixation is achieved in highest quality boneAnatomic Plate Contouring cylindrical curvature minimizes the need for interoperative manipulation of plate to conform to anatomyTemplating and Trialing System ensures plates and screws are prepositioned to achieve best fit and intended compressionCompression through and Outside the Plate maximizes distribution of compression across fusion sites

Paragon 28 is grateful for the significant contributions Dr. Byron Hutchinson, DPM, made as the surgeon designer of this system.

Product Page:http://www.paragon28.com/products/ncfusionplatingsystem

CONTACT:Jim Edson, Director of Product Management and Marketing, jedson@paragon28.com

View original content with multimedia:http://www.prnewswire.com/news-releases/paragon-28-launches-plating-and-guided-screw-system-to-address-naviculocuneiform-arthritis-and-flatfoot-gorilla-nc-fusion-plating-system-300501564.html

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Paragon 28 Launches Plating and Guided Screw System to Address Naviculocuneiform Arthritis and Flatfoot- Gorilla ... - PR Newswire (press release)

(Reuters) - The benefits of Johnson & Johnson's experimental rheumatoid arthritis drug sirukumab do not outweigh the risks, an advisory panel to the U.S. Food and Drug Administration concluded on Wednesday.

The panel voted 12-1 that the drug should not be approved, citing safety concerns, including an imbalance in the number of deaths in patients taking sirukumab compared with those taking a placebo. The most common causes of death were major heart problems, infection and malignancies.

"The safety is not there," said Dr. Beth Jonas, interim head of the division of rheumatology at the University of North Carolina School of Medicine.

The FDA is not obliged to act on the recommendation of its advisory panels but typically does so.

J&J originally developed the drug with GlaxoSmithKline Plc. GSK recently said it would return all rights to J&J. GSK held rights to the drug in North, Central and South America.

Sirukumab blocks a cytokine in the body known as interleukin 6 (IL-6) that can contribute to the inflammation associated with rheumatoid arthritis, an autoimmune disorder that affects more than 1.3 million.

Panelists said they were especially reluctant to recommend approval of sirukumab because there are two other drugs on the market in the same class. These are Roche Holding AG's Actemra and Sanofi SA and Regeneron Pharmaceuticals Inc.'s

"If this was a new agent I would probably be a little more enthusiastic," said Dr. Maria Suarez-Almazor, rheumatology section chief at the University of Texas MD Anderson Cancer Center. "There is no reason to think that this new drug will act in a tremendously different way."

The FDA, in briefing documents released on Monday, noted that the trend toward increased overall mortality seemed unique to the sirukumab program.

Reporting by Toni Clarke in Washington; Editing by Jonathan Oatis and Steve Orlofsky

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FDA panel votes against approval of J&J arthritis drug - Reuters

Scientists from IIT Guwahati have synthesised mats made of silk-proteins and bioactive glass fibres that they believe can assist the growth of bone cells and repair worn-out joints in arthritis patients.

The disease most commonly affects joints in the knees, hips, hands, feet, and spine and is marked by the breakdown of joint cartilage and underlying bones. Left untreated, it can cause severe pain, swelling, and eventually limited range of movement.

Current clinical treatment methods are limited by lack of viable tissue substitutes to aid the repair process, Biman B. Mandal from institute said.

Joint disease

To develop a suitable tissue substitute, scientists, including those from the University College London in the U.K., looked into the natural bone-cartilage interface and tried to mimic it synthetically in lab conditions.

Knee osteoarthritis is the most common bone and joint disease in India. However, Mr. Mandal pointed out that the available clinical grafts were expensive.

Enhances healing

We used silk, a natural protein to fabricate electrospun mats to mimic the cartilage portion and bioactive glass to develop a composite material, similar to the natural tissue, said Mr. Mandal.

For the mat, scientists used a kind of silk easily available in northeast India.

Muga [Assam] silk is endowed with properties that enhance the healing process, Mr. Mandal said.

The researchers adopted a green fabrication approach for the developing the silk composite mats electrospinning. It is similar to knitting, except that it utilises electric high-voltage force to draw ultrafine fibres, Mr. Mandal said.

A layer by layer approach was followed, where the bone layer was first formed, on top of which the cartilage layer was developed. The resulting composite mat resembled the architecture of the bone-cartilage interface.

To assist the regenerative process, the mats would be grafted in the defected joint with cells harvested from the patient.

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Silk mats can help treat arthritis - The Hindu

Klotho, in ancient Greek mythology, is one of the Fates controlling the span of human destiny tasked as she was with cutting the string that determined the length of a persons life. Klotho is also a naturally occurring hormone in the body. More than two decades ago, Japanese researchers discovered that this hormone plays a role in aging. People with more klotho in their body, tend to live longer and to retain more of their facultiesthat is to stay sharpwell into old age.

There are many elderly people who walk around and have completely normal, sharp vibrant brain function despite the fact that they are much older and the fact that they have a lot of disease toxins in the brain, Dr. Dena Dubal a neurobiologist at the University of California, San Franciscos Memory and Aging Center told Popular Science.

Dubal is the author on a study released today in the journal Cell Reports that looks at what happens to brain function when you inject klotho protein into mice.

Aging researchers like Dubal arent interested in extending life for its own sakethey dont have a Methuselah complexbut rather because aging is the biggest factor for disease. If they can find a way to slow aging, or at least its effects, they might find a way to allow us to avoid the cognitive declines, and diseases such as stroke, heart disease, and diabetes that tend to come as we flip through more pages on the calendar.

A few years ago, we discovered in collaboration with several people that in human populations those people that had higher levels of klotho had better cognitive functions in normal aging, says Dual.

Which is great if you happen to be genetically blessed with higher levels of klotho, less great if youre not.

Earlier studies have looked at the impacts of klotho on longevity and health, including brain health, but they tended to rely on genetically modified mice. The genes of these mice had been modified to allow them to produce more klotho or less.

Those studies found that mice modified to produce more klotho lived longer, which is promising. But despite recent advances, genetically modifying humans is still an ethical minefield away in the future, especially if the modifications are being made to improve wellbeing as opposed to preventing disease. And, genetically altering embryos might help future populations, but it doesnt do much for those of us already living. So, Dubal wanted to see is if mice injected with klothoacting essentially a hormonal supplementwould show cognitive improvements.

The answer, seems to be yes.

To get the result, Dubal and her colleagues injected three types of mice with a portion of the protein. They injected young mice, aged mice, and mice genetically altered to have brains similar to that which we would see in Alzheimers or Parkinsons patients in humans.

Within hours they showed better cognitive function, says Dubal.

Since you cant exactly administer a mouse an IQ test, they assessed brain power based on the mices ability to navigate a series of water mazes, in an experiment that sounds on par with human a trip to Wisconsins famed waterslide park, The Dells.

They found that mice that had daily injections and were better able to navigate the maze (as measured by the distance traveled to find a hidden platform) than their control group peers. In a classic example of work smarter, not harder, the klotho mice were just much more efficient seekers.

We tested them two weeks later in a different cognitive test and they were still smarter, says Dubal, which suggested that getting the klotho protein into their bodies combined with brain training and stimulation had a long-lasting effect in their brain. Because the half-life of the protein is only seven and a half hours long, any of the protein should have been long out of their system.

There are a few caveats.

First, this was an experiment in micenot in humans. While its incredibly promising, the study results are short term and they werent looking for side effects. The pace at which a promising scientific study is turned into a supplement of dubious efficacy is stunning these days, so please dont subject yourself to some back-alley klotho injection. Between resveratrol and superfoods weve been there before.

At the same time, we know that klotho levels can be affected not only by genetics - but also by stress. If you want to help your body keep its klotho levels at your own peak you might want to try proven stress reduction techniques like exercise and chilling out about your own impending mortality.

Finally, the researchers arent sure how klotho seems to be generating this effect because the protein is too big to pass from the body into the brain. For a long time, weve studied the brain in isolation from the body with the brain mostly telling the body what to do, and the body acting like a series of censors that give the brain useful data. But this study like our growing understanding of the connection between our microbiome and brain health, and exercise and brain health, this study touches on our growing understanding of how the body connects to the brain to help make the brain more resilient.

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An extra dose of this longevity hormone helped make mice smarter - Popular Science

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Monday, August 07, 2017

EDITORIAL

Tom Brady - is his brain immune from injury?

It has been nearly eight years since Malcolm Gladwell wrote his piece in The New Yorker about the long-term tragic impact of football on the brain of those that play the game. It outlines the post-football life, There were men with aching knees and backs and hands, from all those years of playing football. But their real problem was with their heads, the one part of their body that got hit over and over again.

Gladwell's article, Offensive Play How different are dogfighting and football? is one of a growing number of looks at the depressing post-football life of the players - viewed by many as American heroes. He wrote it 2009.

My Life Sucks

Now in 2017, Jim Plunkett, who was a Super Bowl hero back in the day and now 69, told the San Jose Mercury News, My life sucks. Plunkett's body is broken and he is in constant pain. Once a man like Tom Brady - overcoming obstacles and realizing NFL quarterback hero status -- he is now devastated physically and his wife says in the same interview that Plunketts mind is being ravaged by the years of head injuries.

Math 99% of NFL

In July, a paper in the JAMA found that Chronic Traumatic Encephalopathy, known as CTE, was found in 99% of deceased NFL players' brains those that were donated for scientific research. And, CTE was found in three of the 14 high school players and 48 of the 53 college players whose brains were tested. CTE is directly linked tomemory loss, confusion, impaired judgment, aggression, depression, anxiety, impulse control issues, and suicidal behavior. A slew of former NFL players have committed suicide in the past few years and their brains were found to have CTE.

Beautiful Brady

While so many love the amazing story of Bradys greatest of all-time title and marvel at his longevity built of his dedication to physical performance, luck, genetics, and kale salads, the reality is none of those things can protect his brain from the impact of years of missed blocks or blindside corner blitzes.

The collective failure of those missed blocks and defensive scheming of opponents may not have stopped the Patriots from being the great team in NFL history, but they have taken their toll on jarring the brain of Brady no less than any other player. The reality is that the longer he plays, the more his brain is impacted.

His longevity is now working against his future the statistics prove it. The list of NFL players who were found to have CTE is long and depressingly catastrophic. The Patriots alone can claim Junior Seau, Most Tatupu, and Kevin Turner -- all tested after death and all suffered from CTE. Seau killed himself at age 43, Tatupu was 54 at the time of his death, and Turner died of ALS believed to be linked to CTE at the age of 46.

Deviant Behavior

Gladwell ends the article with a passage from Dogmen and Dogfights, an academic analysis of deviant behavior by Rhonda Evans and Craig Forsyth about those involved with dogfighting.

The authors write:

When one views a staged dog fight between pit bulls for the first time, the most macabre aspect of the event is that the only sounds you hear from these dogs are those of crunching bones and cartilage. The dogs rip and tear at each other; their blood, urine and saliva splatter the sides of the pit and clothes of the handlers. . . . The emotions of the dogs are conspicuous, but not so striking, even to themselves, are the passions of the owners of the dogs. Whether they hug a winner or in the rare case, destroy a dying loser, whether they walk away from the carcass or lay crying over it, their fondness for these fighters is manifest."

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EDITORIAL: 99%, My Life Sucks, Dogfighting, and Brady's Brain - GoLocalProv

This press release was orginally distributed by SBWire

Guildford, UK -- (SBWIRE) -- 08/08/2017 -- When it comes to eye health and vision, the needs of every individual differ. As we are subjected to an increasing girth of information regarding sight correction procedures and vision health, researching choices and options can be overwhelming. With the advancements in technology, ground breaking treatments such as LASIK, LASEK, 3rd Generation ReLEx SMILE and Lens Replacement can transform eyesight overnight thereby reducing the need to rely on optical aids like glasses or contact lenses.

Attending Optegra's informative Open Evening on August 9, 2017 (6 PM) affords all Londoners an ideal opportunity to learn about powerful lens replacement techniques. Ideal for those in the Capital aged 50+ yearning for perfect sight, the event will help provide answers to any and all vision correction concerns and queries.

The North London event will be held at Optegra's state of the art hospital conveniently located at 6 The Technology Park Colindeep Lane, London, NW9 6BX.The evening will be led by Optegra's Consultant Mr. Mark Hulbert along with Optegra's Eye Health Care team. Mr. Hulbert is a specialist in general ophthalmology and has a particular interest in glaucoma and neuro-opthalmology.

The informal structure of the North London event encourages attendees to clarify doubts and discuss any unfounded fears they may have regarding vision correction. The open evening also provides participants an opportunity to learn about past patient experiences and get an understanding about Optegra's clinical outcomes and outstanding success rate standards. Due to the high popularity of the event, prior booking is encouraged to attend the North London Open Evening.

To find out why Optegra is UK's most trusted eye health care provider recommended by GPs & optometrists, its wide range of treatments and money saving offer visit Optegra.com or call 0808 273 6059 Mon-Fri 8am-8pm, Sat 9am-4pm.

For media enquiries contact Tukshad Engineer Head of Digital at Optegra - 0755 499 4155 | tukshad.engineer@optegra.com.

About Optegra Eye Health CareOptegra Eye Health Care is a specialist provider of ophthalmic services in the UK, China, Czech Republic, Poland and Germany. Optegra operates nationwide eye hospitals and clinics in the UK and brings together leading edge research and medical expertise, state-of-the-art surgical equipment and top ophthalmic surgeons renowned for their areas of expertise to offer excellent clinical outcomes in laser eye surgery, lens replacement (Clarivu), cataract removal, AMD, vitreoretinal and oculoplastic procedures all carried out in five-star patient facilities.

Optegra Eye Health Care operates seven dedicated eye hospitals based in Birmingham, Central London, Hampshire, Manchester, North London, Surrey and Yorkshire. For more information please visit Optegra.com.

Contact:Tukshad Engineer, Head of Digital at OptegraCompany: Optegra Eye Health CarePhone: 0755 499 4155Address: The Surrey Research Park, 10 Alan Turing Rd, Guildford GU2 7YF, United KingdomEmail: tukshad.engineer@optegra.comWebsite:http://optegraeyehealthcare.com/

For more information on this press release visit: http://www.sbwire.com/press-releases/have-questions-about-an-eye-condition-or-vision-problems-attend-optegras-informative-open-evening-in-north-london-on-august-9-2017-844882.htm

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Have Questions About an Eye Condition or Vision Problems? Attend Optegra's Informative Open Evening in North ... - Digital Journal

"Any attempt to treat veterans with traumatic brain injuries begins with an accurate diagnosis," says Randall Benson, M.D., CNS Vice President and Medical Director. "Combat injuries are frequently mis-diagnosed resulting in inappropriate and unsuccessful treatment. We can and must do better. Better diagnoses lead to improved outcomes."

Hope After Combat combines diagnostic testing including neurology, neuro-opthalmology and neuropsychology enhanced by CNS' advanced magnetic resonance imaging techniques (MRI), diffusion tensor imaging and susceptibility-weighted imaging. These advanced MRI techniques developed at CNS are significantly more sensitive than traditional MRI imaging and have worked successfully on hundreds of non-combat brain-injury victims.

In the Hope After Combat study, once diagnostic testing is complete a treatment plan is developed and the veteran takes part in an individualized 60-day rehabilitation program based on his or her specific diagnosis. All disciplines continue to provide input and care for the victim and his/her family. The final study is expected to include more than 50 combat victims.

"These veterans and their families are desperate for answers and we're trying to provide them," says John D. Russell, CEO and President of CNS. "The veterans have been real troopers in every sense of the word and we're learning from their individual efforts and progress. But our donors are the ones who've made the study possible. Without them we couldn't bring all these disciplines together for such an important study."

For further information or to participate in or make a contribution to the Hope After Combat study, contact John Russell at 313-228-0930 or visit the CNS website, http://neurologicstudies.com

CNS was founded in 2011 with the objective of advancing scientific research for neurovascular disease. CNS staff are available for interview on brain injuries and brain-related diseases.

View original content:http://www.prnewswire.com/news-releases/military-brain-injuries-are-the-subject-of-center-for-neurological-studies-hope-after-combat-study-300499425.html

SOURCE Center for Neurological Studies

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Military Brain Injuries are the Subject of Center for Neurological Studies "Hope After Combat" Study - PR Newswire (press release)

Mobilizing the forces of the immune system to the fight has changed the game of cancer treatment in recent years.

The key to success has been first identifying how cancer cells disarm the T-cell fighters of the immune system and then finding drugs to restore their firepower.

Using drugs called checkpoint inhibitors, patients with incurable cancers like advanced melanoma have shown long-term responses.

Forty percent of melanoma patients will still fail to respond to the treatment, however, which means that cancer cells must have other means ones that are not addressed by checkpoint inhibitors to disable the immune systems weaponry.

To discover what they are, a research team led by Nicholas Restifo at the US National Cancer Institute began with human melanoma cells growing in a dish, and systematically disabled every gene in the melanoma cells using the CRISPR gene-editing technique.

They then tested the ability of the T-cells fighters to recognize each one. It turned out about 100 different genes activated by the cancer were able to prevent the attack by the T-cells.

Of particular interest was a gene called APLNR. While it has been implicated as contributing to some cancers, this was the first evidence that it played a role in disarming T-cells.

If we can truly understand mechanisms of resistance to immunotherapy, we might be able to develop new therapeutics, comments Restifo.

The research is published in Nature.

Link:
The cancer genes that disarm the immune system | Cosmos - Cosmos

After decades with little progress, cancer researchers have made major breakthroughs in the last few years in treatingmelanoma(the most serious type of skin cancer). The key to these advances?Immunotherapy is helping patients live longer.

Cleveland Clinic is a non-profit academic medical center. Advertising on our site helps support our mission. We do not endorse non-Cleveland Clinic products or services.Policy

Chemotherapy which is typically a first-line treatment targets cancers directly. But immunotherapy spurs your immune system to attack cancer cells. This approach can offer new hope if you have melanoma.

Researcher and plastic surgeon Brian Gastman, MD, works with the latest advances in using immunotherapy to treat melanoma. He explains how they work.

One of the newest options is injectinga modified form of the herpes virus directly into the tumor, Dr. Gastman says.

This modified virus is called talimogene laherparepvec, or T-VEC.Once doctors inject it, the virus replicates within the cancer cells and causes them to rupture and die.

Doctors use T-VEC to treat melanoma at the skin level, under the skin or in deep soft tissues, he says.

One of the main reasons we give T-VEC is for whats called in-transit metastases, Dr. Gastman says.

He explains, Lets say someone had a melanoma in their foot. Typically, it would spread from the foot to the lymph nodes and from the lymph nodes all over the body.

But sometimes cancer cells get caught between the original site and the lymph nodes and cause little satellite lesions, he says. Those are in-transit metastases.

If its on the arm or leg, you can inject the arm or leg with chemotherapy and it will kill the cancer, but it will also damage the arm or leg, Dr. Gastman says.

T-VEC offers several advantages over chemotherapy in this situation:

Doctors still use ipilimumab, the first immunotherapy drug used to disrupt the growth and spread of cancer, for high-risk patients, but mostly as a help to other treatments. Its sometimes used alongside or after the initial treatment, Dr. Gastman says.

In this case, patients receive a high dose of ipilimumab after surgery to help prevent the cancer from coming back. Its significant because it is helping patients live longer, he says.

This is the first time that an adjuvant (helper) therapy showed an improvement in survival rates over a placebo, he says.

T-VEC and the new use for ipilimumab are the two major advances the FDA approved in recent years. Theres also a lot of exciting research happening, too, Dr. Gastman says.

Now, another immunotherapy drug called Pembrolizumab is being used a lot, he says. In 2016, there was data showing that 40 percent of stage IV melanoma patients treated only with pembrolizumab were alive in three years. Thats very exciting news.

Theres also ongoing research that finds promise in a treatment that combines ipilimumab with another immunotherapy drug, nivolumab.

The study, which focuses on patients with advanced melanoma who received this combination, reported a two-year overall survival rate of 64 percent. Early resultsshow that using the two drugs together is more effective than using either drug alone.

Dr. Gastman also notes that outcomes are improving as doctors learn more about how to prevent or better manage side effects from these drugs. The side effects, which are sometimes significant, may include:

Were much better now than we were five or six years ago at handling the toxic side effects, he says.

Immunotherapies wont completely replace other treatments like chemotherapy and surgery. But, they offer new and effective options for patients with melanoma. And ongoing research continues to make advances, Dr. Gastman says.

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These New Skin Cancer Treatments Unleash Your Immune System ... - Health Essentials from Cleveland Clinic (blog)

In an Australian-first clinical trial leukaemia and lymphoma patients will have access to potentially life-saving immunotherapy treatment.

Nikola Barac, 6, with his mother Fiona Stamenkovic and brother Milan outside Westmead Institute for Medical Research. Photo: Jessica Hromas

Nikola Barac's immune system has done him no favours. Leukaemia and rare genetic immunodeficiency disorder hyper IgM have left his small body defenceless against life-threatening infections.

"I'd have a heart attack every time he had bathwater in his mouth," his mother Fiona Stamenkovic said.

"I would use boiled water to brush his teeth. We had to be very careful."

When Nikola was four years old, and in spite of his family's constant vigilance, an ultrasound picked up the parasite cryptosporidium in his bile ducts and liver.

"It was just devastating," Ms Stamenkovic said.

"I knew what it meant. I thought his liver was going to fail and he would die."

Nikola received two bone marrow transplants from his younger brother, Milan, to fight the infection. It worked, but another insidious virus called cytomegalovirus had flourished in its place.

"The medication he needed was very toxic. It was horrible giving it to him [knowing it could cause him harm]," Nikola's mother said. This time the treatment didn't work.

In a last-ditch move, Nikola's doctors weaponised the very thing that had failed to protect him in the first place. He was given two rounds of genetically-modified T-cell immunotherapy grown in Westmead Institute for Medical Research (WIMR).

This time he was given the all clear.

"It's incredible. This thing has been a part of our lives for so long and now it's not. It's going to take a while to get used to it," Ms Stamenkovic said.

For the first time in Australia a clinical trial is paving the way for patients with leukaemia and lymphoma to access a potentially-life-saving immunotherapy treatment.

A team of researchers at WIMR and Westmead Hospital has been given the go ahead by the Therapeutics Goods Administration to conduct a phase 1 trial.

Launched on Tuesday, the CARTELL trial will recruit 20 leukaemia and lymphoma patients, children and adults, whose disease has returned despite having bone marrow transplants.

The chance of survival for these patients at five years is roughly 10 per cent.

Immunotherapy has become a promising new frontier in cancer treatment that harnesses the power of the immune system to find and attack cancer cells has yielded remarkable results in clinical trials overseas.

"The results have been incredible, there is no other way to describe it," said Professor David Gottleib, group leader of the Bone Marrow Transplant and Cell Therapies Group at WIMR and senior physician at Westmead Hospital.

"People have been blown away by the amazing response rates in people with leukaemia who had very few other choices," he said of the research field still in its infancy.

A small number of eligible Australian cancer patients have spent an estimated $500,000 - $700,000 to access treatment in US trials, on top of the cost of travel, hospital accommodation costs for themselves and their support people.

Professor David Gottleib at the Westmead Institute of Medical Research. Photo: Supplied

The exorbitant cost and limited trial places means the experimental treatment is beyond their reach for most patients.

"The long term goal is to make CAR T-cells affordable and widely accessible to Australian patients as quickly as we can," Professor Gottleib said.

"But there is still a long way to go before we can regard it as routine."

Trial participants will receive genetically modified donor T-cells fitted with chimeric antigen receptors (CARs) that can identify and kill cancerous leukaemic and lymphoma cells.

Published trials overseas have used a viral system to insert the receptors into the cells, stripping away the dangerous components of a virus. The exhaustive process to ensure the alter virus is safe costs hundreds of thousands of dollars.

But the method used in the Australian trial will bypass the viral delivery system, Professor Gottleib said.

"We essentially take two pieces of DNA and cut and paste the receptor into the DNA," he said.

"It means we'll be able to manufacture the cells quickly and more affordably."

The technique could reduce the cost of commercial T-cell treatment from roughly $300,000 to about $50,000, he said.

But it will need to be rigorously tested.

"We don't know the answer to the efficacy question. That's why we're doing the trials," Professor Gottleib said.

"But how you get the receptor into the cells, one has no reason to think the end results should be different," he said.

The researchers will be on high alert for side effects seen in trials overseas, including the potentially fatal cytokine release syndrome, in which the T-cells become overactivated and release inflammatory chemicals that damage healthy cells.

The treatment can also cause neurotoxicity that damages the nervous system, which can cause confusion, memory loss, convulsions and other cognitive dysfunctions.

"We'll definitely be looking for those sorts of problems," Professor Gottleib said.

The new trial is part of a series of trials conducted by the WIMR researchers, aimed at strengthening immunity to infection in cancer patients receiving chemotherapy and other toxic treatments.

They hope to apply for a second trial using participant's own T-cells to create the CARs among leukaemia and lymphoma patients who have not responded to chemotherapy.

The CARTELL trial is expected to run for 18 months to two years.

Patients who may be interested in participating in the trial should speak to their GP or specialist about their suitability.

The story How an experimental therapy weaponised Nikola's failing immune system first appeared on The Sydney Morning Herald.

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How an experimental therapy weaponised Nikola's failing immune system - Illawarra Mercury

The ACMG Foundation for Genetic and Genomic Medicine announced Aug. 4 that Indian American Madhuri Hegde of Waltham, Mass.-based PerkinElmer Inc. was elected to its board of directors.

"We are delighted that Dr. Hegde has been elected to the ACMG Foundation Board of Directors. She has vast experience in genetic and genomic testing and is a longtime member of the college and supporter of both the college and the foundation," said Dr. Bruce R. Korf, president of the ACMG Foundation, in a statement.

Hegde, who will serve a two-year renewable term, joined PerkinElmer in 2016 as vice president and chief scientific officer of global genetics laboratory services. She is also an adjunct professor of human genetics in Emory Universitys human genetics department.

Previously, Hegde served as the executive director and chief scientific officer at Emory Genetics Laboratory in Atlanta, Ga.; professor of human genetics and pediatrics at Emory University; and assistant professor at Baylor College of Medicines Department of Human Genetics in Houston, Texas.

Additionally, Hegde has served on a number of scientific advisory boards for patient advocacy groups including Parent Project Muscular Dystrophy, Congenital Muscular Dystrophy and the Neuromuscular Disease Foundation.

She earned her doctorate from the University of Auckland in Auckland, New Zealand, and completed her postdoctoral fellowship in molecular genetics at Baylor College of Medicine. She also holds a masters from the University of Mumbai in India.

The foundation, a national nonprofit dedicated to facilitating the integration of genetics and genomics into medical practice, is the supporting educational foundation of the American College of Medical Genetics and Genomics.

Board members are active participants in serving as advocates for the foundation and for advancing its policies and programs.

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Madhuri Hegde Elected to ACMG Foundation for Genetic, Genomic ... - India West

JoAnne Viviano The Columbus Dispatch @JoAnneViviano

In what researchers consider a major scientific leap, a team at Ohio State University has discovered a new way of turning skin cells into any type of cells the body might need, a technology that has limitless potential, from regenerating a wounded limb to repairing a brain after stroke to healing a damaged heart.

The process involves placing a square chip about the size of a fingernail on the skin, adding a droplet containing genetic code, and zapping it with an energy source.

While it hasn't been used in humans yet, the process was used in animals to healbrains after stroke and to generate blood vessels in legs wherethe femoral artery, the limbs major blood supply, had been cut, said Chandan Sen, the director of the Center for Regenerative Medicine and Cell-Based Therapies at Ohio State's Wexner Medical Center.

In leg experiments involving mice, researchers placed the chip on the animals' wounded legs, delivered the appropriate genetic material, and saw blood vessels grown to regenerate limbs within seven to 14 days, Sen said. Legs that otherwise would have turned black and required amputation were pink, and the mice were able to run again.

In brain experiments on mice, the chip was again placed on the leg, different genetic material was dropped on, and neurological cells grew in the area. Three weeks later, scientists detected firing neurons, and the new cells were taken from the leg and inserted into the brain.

The leg-healing process was duplicated in pigs after the Walter Reed National Military Medical Center in Bethesda, Maryland, expressed interest. Sen said the technology could be used to heal troops in the field. One caveat: It must be deployed within 72 hours of a limb being damaged.

Twenty-six Ohio State researchers from the fields of engineering, science and medicine worked together to make the technology a reality.

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The discovery could have countless applications across various medical disciplines, Sen said. He's hopeful other researchers will help stretch the impact of the device.

"There are many smart minds throughout the country and the world that will take this and run," Sen said.

Sen expects that human trials will come soon, after a letter on the research is published Monday in the Nature Nanotechnology journal, a peer-reviewed scientificpublication.The research was led by Sen and L. James Lee, professor of chemical and biomolecular engineering in Ohio States College of Engineering.

Sen said it takes less than a second to deliver the genetic code that spurs the skin cells to switch to something else, then several days for new cells to grow.

The equipment needed can fit in a pocket. And the process can be done anywhere; no lab or hospital is needed.

The black chip, made of silicon, acts as a carrier for the genetic code.

"Its like a syringe thats the chip but then what you load in the syringe is your cargo," Sen explained. "Based on what you intend the cells to be, the cargo will change. So if you want a vasculogenic (blood vessel) cell, the code would be different than if you wanted a neuro cell, and so on and so forth."

The genetic code is synthetically made to mirror code from the patient.

The electric field pulls the genetic material into the skin cells.

Because the research project had a high risk of failure, and because Ohio State wanted to keep it close to the vest, public money was not sought, Sen said. Instead it was funded by university and philanthropic money from Leslie and Abigail Wexner, Ohio States Center for Regenerative Medicine and Cell-Based Therapies, and the universitys Nanoscale Science and Engineering Center.

Approval from the federal Food and Drug Administration is required before Sen, Lee and the research team can try the technique in humans. He expects to get that approval and prove human feasibility within a year. Sen's hopeful that "the floodgates will open" and then thetechnology will be used widely within five years.

The chips are already being manufactured locally due to an assist from the Rev1 Ventures business incubator on the Northwest Side, and the technology has gained interest from Taiwan-based Foxconn Technology Group.

Lee called the concept very simple and said he was surprised by how well it worked.

He had developed similar technology prior to 2011, but it only worked on individual cells and only in processes separate from the body. Since then, he said, many researchers and companies have approached him to come up with a system that worked on tissue in the body.

"More and more people said, 'This technology can be very, very powerful if you can do tissue,'" he said. "It turns out that it works. It was very surprising."

This version, he said, is a very significant advancement and is "much, much more useful for the medical applications."

jviviano@dispatch

@JoAnneViviano

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Ohio State researchers report breakthrough in cell regeneration - The Columbus Dispatch

San Francisco-based genetic diagnostics company Invitae has acquired Good Start Genetics and CombiMatrix, expanding Invitaes portfolio to include prenatal and pediatric testing. Its part of their long-term plan to make genomic testing routine.

Were building a company for the coming genomic era that includes genetic capabilities through all phases of life, said Invitae CEO Sean George in a phone interview.

Invitae offers a wide range of genomic panels to detect anomalies that could contribute to heart disease, cancer, neurologic disorders and other conditions. In Good Start, Invitae picks up expertise in carrier screening and preimplantation genetic testing. CombiMatrix also provides preimplantation testing, as well as panels to analyze miscarriages and pediatric developmental disorders.

Invitae is issuing 1.65 million shares of stock, paying $18.3 million in cash and assuming $6 million in debt for privately-held Good Start. CombiMatrix shareholders will receive around $27 million in common stock.

Spun off from Genomic Health in 2012, Invitae initially focused on adult inherited diseases and has gradually expanded their portfolio. They now enter a crowded field that includes LabCorp (which acquired Sequenom last year), Illumina, Progenity and others. George believes Invitaes ability to do the hard things will carry them through these market battles.

We are building a technology engine to win the race of scale, said George. We are looking to the OB market and the perinatal space to extend our platforms capabilities. But more importantly, in order to move the world away from the current disease-by-disease, test-by-test market, its managing genetic information for an individual over the course of their life.

Good Start appealed to Invitae for their cost-effective pre-implantation screening and diagnosis. CombiMatrix brings specific expertise in chromosomal microarrays. In addition, the companies could expand Invitaes marketing reach.

The two together have a pretty good commercial presence in the IVF and reproductive medicine sector, said George. Combined, especially with our capabilities, I think its fair to say we are immediately the number one player in the IVF, reproductive medicine segment for genetic information.

These acquisitions add around 150 people to the Invitae payroll, a 20 percent workforce increase. George notes they are always looking around for potential acquisitions but will probably take a breather to focus on moving new products to market. Ultimately, Invitae wants to be the company that mainstreams clinical genomics.

With the broad capabilities we now have at all stages of life, we expect to get traction in this new age of genomic medicine, where all this information can be brought to bear, said George. The first company to have broad capabilities across all of it and to continue to lower the cost basis and deliver that information is likely in position to truly bring genetics into medicine for everybody.

Photo: mediaphotos, Getty Images

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Invitae CEO says the diagnostic company has big plans for genomic medicine - MedCity News

An international team of scientists published a new study last week documenting edits theyd made to viable human embryos carrying a genetic mutation, one associated with a life-threatening heart condition. It is the first study of its kind to take place in the United States.

The researchers were able to remove a problematic mutation in the MYBPC3 gene with a higher success rate than in similar studies. After adjusting their method, 72 percent of the embryos were free of the mutation. The scientists believe they may be able to address other monogenetic diseases using the same technique, CRISPR-Cas9.

But the notion of altering human DNA to eradicate inherited diseases is generating concern, too. These genetic changes would permanently affect the DNA passed through a family line, for one. Other critics raise the possibility of altering embryos to create desired characteristics (though it would be much harder for scientists to target genes associated with humor, creativity or physical traits).

Cardiologist and geneticist Dr. Elizabeth McNally is the director of the Center for Genetic Medicine at Northwestern University. She joins Phil Ponce in discussion.

Related stories:

UIC Launches Stem Cell, Regenerative Medicine Center

June 12: Researchers at UIC will focus on understanding tissue regeneration and spearheading future developments in stem cell biology as a means to repair diseased organs and tissues.

The Science and Ethics of Editing Human Embryos

Feb. 28: Earlier this month, an influential group backs editing the genes in human embryos to eliminate disease. Chicago Tonight guests discuss human gene editing and some of the ethical issues it raises.

Baby with 3 Parents: Genetic Technique Offers Hope, Controversy

Sept. 29, 2016: A baby has been born with the DNA of three parents. We hear about the promise the technique offers for avoiding some birth defects, and the ethical concerns it raises.

Read more:
New Gene Editing Study Raises Possibilities, Questions - Chicago Tonight | WTTW

Genetic engineering has the potential to change the way we live. The science behind the agricultural, medical, and environmental achievements is spectacular, but this excitement is tempered by concern for the unknown effects of tampering with nature. How should we use genetic engineering?

DNA is the root of all inheritance and the key to understanding the basics of all biological inheritance and genetics.

The possibilities of this genetic engineering are endless, and everyone from medicine to industry is scrambling to adopt it and adapt it to their specific needs.

Genetic engineering changes or manipulates genes in order to achieve specific results, and there are many ways to "engineer" genetic material including fixing defective genes, replacing missing genes, copying or cloning genes, or combining genes.

How is genetic engineering used in food production? What political, environmental, and production obstacles could arise in the effort to label genetically engineered foods? What food traits would you like to see genetically engineered?

How could GE help in meeting growing demand for food around the world?

How can GE be used with animals? What are the benefits and risks of using genetic engineering with livestock or with endangered or extinct animals?

How does cloning work? What situations might be viewed as ethical uses of human cloning? Unethical?

What are the potential consequences, positive and negative, of discovery in the genetic engineering field? Who should be involved in determining the ethical limitations of the uses of genetic engineering?

Produced from 2001 through 2004, Iowa Public Television's Explore More online and broadcast series engages students in problems they can relate to, provides compelling content for investigation and gives students opportunities to form their own points of viewon contemporary issues.

Although the full website has been retired, this archive provides links to project videos and related resources. Please contact us if you have questions or comments about Explore More.

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Genetic Engineering | IPTV

Thisarticleoriginally appeared at Ensia and has been republished here with permission.

Which is more disruptive to a plant: genetic engineering or conventional breeding?

It often surprises people to learn that GE commonly causes less disruption to plants than conventional techniques of breeding. But equally profound is the realization that the latest GE techniques, coupled with a rapidly expanding ability to analyze massive amounts of genetic material, allow us to make super-modest changes in crop plant genes that will enable farmers to produce more food with fewer adverse environmental impacts. Such super-modest changes are possible with CRISPR-based genome editing, a powerful set of new genetic tools that is leading a revolution in biology.

My interest in GE crops stems from my desire to provide more effective and sustainable plant disease control for farmers worldwide. Diseases often destroy 10 to 15 percent of potential crop production, resulting in global losses of billions of dollars annually. The risk of disease-related losses provides an incentive to farmers to use disease-control products such as pesticides. One of my strongest areas of expertise is in the use of pesticides for disease control. Pesticides certainly can be useful in farming systems worldwide, but they have significant downsides from a sustainability perspective. Used improperly, they can contaminate foods. They can pose a risk to farm workers. And they must be manufactured, shipped and applied all processes with a measurable environmental footprint. Therefore, I am always seeking to reduce pesticide use by offering farmers more sustainable approaches to disease management.

What follows are examples of how minimal GE changes can be applied to make farming more environmentally friendly by protecting crops from disease. They represent just a small sampling of the broad landscape of opportunities for enhancing food security and agricultural sustainability that innovations in molecular biology offer today.

Genetically altering crops the way these examples demonstrate creates no cause for concern for plants or people. Mutations occur naturally every time a plant makes a seed; in fact, they are the very foundation of evolution. All of the food we eat has all kinds of mutations, and eating plants with mutations does not cause mutations in us.

Knocking Out Susceptibility

A striking example of how a tiny genetic change can make a big difference to plant health is the strategy of knocking out a plant gene that microorganisms can benefit from. Invading microorganisms sometimes hijack certain plant molecules to help themselves infect the plant. A gene that produces such a plant molecule is known as a susceptibility gene.

We can use CRISPR-based genome editing to create a targeted mutation in a susceptibility gene. A change of as little as a single nucleotide in the plants genetic material the smallest genetic change possible can confer disease resistance in a way that is absolutely indistinguishable from natural mutations that can happen spontaneously. Yet if the target gene and mutation site are carefully selected, a one-nucleotide mutation may be enough to achieve an important outcome.

There is a substantial body of research showing proof-of-concept that a knockout of a susceptibility gene can increase resistance in plants to a very wide variety of disease-causing microorganisms. An example that caught my attention pertained to powdery mildew of wheat, because fungicides (pesticides that control fungi) are commonly used against this disease. While this particular genetic knockout is not yet commercialized, I personally would rather eat wheat products from varieties that control disease through genetics than from crops treated with fungicides.

The Power of Viral Snippets

Plant viruses are often difficult to control in susceptible crop varieties. Conventional breeding can help make plants resistant to viruses, but sometimes it is not successful.

Early approaches to engineering virus resistance in plants involved inserting a gene from the virus into the plants genetic material. For example, plant-infecting viruses are surrounded by a protective layer of protein, called the coat protein. The gene for the coat protein of a virus called papaya ring spot virus was inserted into papaya. Through a process called RNAi, this empowers the plant to inactivate the virus when it invades. GE papaya has been a spectacular success, in large part saving the Hawaiian papaya industry.

Through time, researchers discovered that even just a very small fragment from one viral gene can stimulate RNAi-based resistance if precisely placed within a specific location in the plants DNA. Even better, they found we can stack resistance genes engineered with extremely modest changes in order to create a plant highly resistant to multiple viruses. This is important because, in the field, crops are often exposed to infection by several viruses.

Does eating this tiny bit of a viral gene sequence concern me? Absolutely not, for many reasons, including:

Tweaking Sentry Molecules

Microorganisms can often overcome plants biochemical defenses by producing molecules called effectors that interfere with those defenses. Plants respond by evolving proteins to recognize and disable these effector molecules. These recognition proteins are called R proteins (R standing for resistance). Their job is to recognize the invading effector molecule and trigger additional defenses. A third interesting approach, then, to help plants resist an invading microorganism is to engineer an R protein so that it recognizes effector molecules other than the one it evolved to detect. We can then use CRISPR to supply a plant with the very small amount of DNA needed to empower it to make this protein.

This approach, like susceptibility knockouts, is quite feasible, based on published research. Commercial implementation will require some willing private- or public-sector entity to do the development work and to face the very substantial and costly challenges of the regulatory process.

Engineered for Sustainability

The three examples here show that extremely modest engineered changes in plant genetics can result in very important benefits. All three examples involve engineered changes that trigger the natural defenses of the plant. No novel defense mechanisms were introduced in these research projects, a fact that may appeal to some consumers. The wise use of the advanced GE methods illustrated here, as well as others described elsewhere, has the potential to increase the sustainability of our food production systems, particularly given the well-established safety of GE crops and their products for consumption.

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When genetic engineering is the environmentally friendly choice - Genetic Literacy Project

A new method of isolating lung stem cells could help speed the development of stem-cell based therapies for lung diseases, including cystic fibrosis, according to a University of North Carolina study.

That method is extracting them with a tube from the mouth to the lung rather than surgery. The team has already used the method in their pulmonary fibrosis research work.

The new study, Derivation of therapeutic lung spheroid cells from minimally invasive transbronchial pulmonary biopsies, was published in the journalRespiratory Research.

Doctors can use stem cells to restore injured lungs, but obtaining and maintaining the cells is challenging.

Not only do they need a lot of lung tissue to extract the stem cells, but the way they have obtained the tissue with surgery is highly invasive. This has led to high death rates among patients who have had the biopsy surgery.

Still, isolating the cells for stem cell-based therapies is a good way to treat many lung diseases.

Until recently,University of North Carolina Health Careresearchers used lung tissue biopsies to obtain stem and support cells that they can cultivate for treatments. They discovered thatlung spheroid cells can help regenerate the lungs of mice with pulmonary fibrosis.

The team is now using a relatively non-invasive procedure, called a transbronchial biopsy, to isolate lung spheroid cells while reducing the risks associated with obtaining them by surgery. In this procedure, a lung pneumologist inserts a thin, lighted tube, or bronchoscope, through a patients nose or mouth to collect several pieces of lung tissue.

This is the first time anyone has generated potentially therapeutic lung stem cells from minimally invasive biopsy specimens, Dr. Jason Lobo, an assistant professor of medicine at the university, said in a press release.

We snip tiny, seed-sized samples of airway tissue using a bronchoscope, said Lobo, a co-lead authoer of the study. This method involves far less risk to the patient than does a standard, chest-penetrating surgical biopsy of lung tissue.

They were able to obtain more than 50 million lung spheroid cells from one small piece of isolated tissue. When they injected the cells into mice, they were delighted to find that the cells ended up in the animals lungs.

These cells are from the lung, and so in a sense theyre happiest, so to speak, living and working in the lung, said the other co-lead author, Dr. Ke Cheng. He is an associate professor in the universitys Departments of Molecular Biomedical Sciences and Biomedical Engineering.

In a second study, published in the journalStem Cells Translational Medicine,researchers highlighted lung spheroid cells potential to treat cystic fibrosis. Injecting the cells in rats with cystic fibrosis significantly reduced their lung inflammation and scarring, compared with control animals.

Also, the treatment was safe and effective, whether the lung spheroid cells were derived from the recipients own lungs or from the lungs of an unrelated strain of rats, Lobo said. In other words, even if the donated stem cells were foreign, they did not provoke a harmful immune reaction in the recipient animals, as transplanted tissue normally does.

Scientists have had discussions with theU.S. Food and Drug Administrationabout clinical trials of lung spheroid cells as a pulmonary fibrosis therapy.

Cells isolated from patients own lung tissue would eliminate the risk of the body rejecting the stem cells. Such treatments require a lot cells, however and doctors might opt to harvest some from healthy volunteers and whole lungs obtained from organ donation networks.

Our vision is that we will eventually set up a universal cell donor bank, Cheng said.

The researchers hope that some day their stem cell therapycan be used in other lung diseases, including cystic fibrosis.

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Researchers Use Simpler, Safer Method to Obtain Stem Cells for Treating Lung Diseases - Cystic Fibrosis News Today

KRAKOW, Poland and RYE BROOK, New York, August 8, 2017 /PRNewswire/ --

Selvita S.A. (WSE: SLV) and The Leukemia & Lymphoma Society (LLS) announced today a partnership to co-fund further preclinical and clinical development of a targeted therapy to treat patients with acute myeloid leukemia (AML). Selvita has discovered and is developing SEL120, a therapy that targets the cyclin-dependent kinase 8 (CDK8) protein, which plays a unique and critical role in gene regulation. Laboratory studies have shown the agent to be effective in certain types of AML cells.

SEL120 is an ATP-competitive and selective inhibitor of CDK8 and a closely related serine kinase, CDK19. CDK8 is a part of a multi-protein complex that regulates gene expression and is distinct from CDK4 or CDK6, which play a role in cell cycle progression. In laboratory experiments, treatment with SEL120 has been shown to result in the death of AML cells especially with elevated phosphorylation of STAT5 and stem cell characteristics, which is significant because AML stem cells are typically resistant to conventional therapies and thereby mediate relapsed disease. Moreover, the status of phosphorylation of STAT5 may provide a useful biomarker for action of the drug. The molecular mechanism of action involves modulation of various oncogenic transcriptional programs that are critical to the survival of AML cells. This unique mechanism, which does not overlap with existing therapies, may allow the development of highly effective combination therapies that may be required to provide long-term control of AML in patients.

SEL120 has shown efficacy in treating AML cells both in vitro (test tube) and in vivo (mouse models). SEL120 has successfully passed a series of non-GLP toxicity studies in mice and monkeys. Selvita initiated Investigational New Drug (IND)-enabling studies for SEL120 in June 2017, a critical step in getting the U.S. Food and Drug Administration's permission to begin in human clinical trials.

Under the terms of the agreement, LLS will provide up to $3.25 million funding over 4 years, through its Therapy Acceleration Program (TAP), in order to help fund further SEL120 IND-enabling studies and a Phase I trial in AML.

"We are very pleased to be partnering with The Leukemia & Lymphoma Society. This partnership constitutes not only a significant validation of the high potential of SEL120 in treatment of AML patients, but also offers a unique opportunity to work with world renowned researchers, specialists in the field of hematological cancers, increasing the chances of bringing a breakthrough treatment to the patients whose treatment options currently remain very limited,"said Krzysztof Brzzka, Ph.D., Chief Scientific Officer at Selvita.

"LLS has developed a comprehensive approach to beat AML, which is one of the leukemias associated with exceptionally high mortality rates. Moreover, very few effective agents are available to control AML, particularly in elderly individuals where the disease commonly occurs," said Lee Greenberger, Ph.D., LLS's Chief Scientific Officer. "LLS is focused on identifying and advancing the most innovative therapeutics to control and/or eradicate blood cancers. LLS's partnership with Selvita understates our commitment to accelerating cures for this deadly disease."

LLS's Therapy Acceleration Program (TAP) funds innovative projects related to therapies, supportive care or diagnostics that have the potential to change the standard of care for patients with blood cancer, especially in areas of high unmet medical need. Further development of the SEL120 project will be co-funded through the Biotechnology Accelerator Division, a strategic initiative to partner directly with biotechnology companies.

About The Leukemia & Lymphoma Society

The Leukemia & Lymphoma Society (LLS) is the world's largest voluntary health agency dedicated to blood cancer. The LLS mission: Cure leukemia, lymphoma, Hodgkin's disease and myeloma, and improve the quality of life of patients and their families. LLS funds lifesaving blood cancer research around the world and provides free information and support services.

Founded in 1949 and headquartered in Rye Brook, New York, LLS has chapters throughout the United States and Canada. Since 1953, LLS has funded over $1 B in blood cancer research through 4000 grants to academic institutions and over 50 therapeutic opportunities through the TAP program.For more information please visit:http://LLS.org/

About Selvita S.A.

Selvita S.A.is a drug discovery company, developing breakthrough therapies in the area of oncology. The company's most advanced program, SEL24, has been licensed to Menarini Group and is currently in Phase I studies in AML.Other projects include SEL120, a first-in-class small molecule inhibitor of CDK8 in preclinical studies, several drug discovery platforms in immuno-oncology, immunometabolism and cancer metabolism, as well as epigenetics, performed independently or in collaboration with global pharma and biotech companies.

Selvita also offers a wide range of integrated drug discovery services, helping biotech and pharma partners discover and develop new drugs.

The company has alliances and partnerships with more than fifty large and medium-sized pharmaceutical and biotechnology companies from USA and Europe, including R&D partnerships with Merck, H3 Biomedicine, Nodthera Therapeutics/Epidarex Capital and Menarini Group.

The company was established in 2007 and currently employs over 400 scientists, among which 30% are PhDs. Selvita is headquartered in Krakow with second research site in Poznan, Poland and international offices located in Cambridge, MA and San Francisco Bay Area, in the US, as well as in Cambridge, UK. Selvita is listed on the Warsaw Stock Exchange (WSE: SLV). More information:http://selvita.com/

SOURCE Selvita S.A.

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Selvita and The Leukemia & Lymphoma Society Announce Partnership to Advance SEL120 Into Phase I for AML Patients - Markets Insider

Eye doctors are fast-tracking efforts to improve stem cell transplants to save the sight of people blinded in Londons acid attack epidemic, the Standard can reveal.

Research at The Royal Free Hospital aims to boost success rates, particularly where cells are taken from deceased donors at present, three-quarters of such transplants fail.

Corneal stem cell transplants have been used for some time on acid victims, including model Katie Piper.

But they are difficult and risky to perform, with doctors hampered by a shortage of donated eyes.

They are most successful when one eye is damaged and cells can be transplanted from the healthy eye. This works in about seven out of 10 cases.

The success rate is about 25 per cent when both eyes are damaged and cells are harvested from the eye of a deceased donor.

Research: consultant Alex Shortt, right, and student Harley Buck at The Royal Free Hospital

Consultant ophthalmic surgeon Alex Shortt, who is carrying out the research at the Royal Frees institute of immunity and transplantation in Hampstead, said the aim was a success rate of 95 per cent. The work is being funded by the Wellcome Trust and Moorfields Eye Charity.

A total of 454 acid attacks were reported to the Metropolitan police last year, almost three times the number in 2014. Mr Shortt said: The last six to 12 months has seen demand for these treatments rise hugely.

He said the challenge was to remove scar tissue without creating a new scar as the eye healed. A sample of healthy corneal stem cells are grown in the laboratory to form a sheet, which is attached to the surface of the damaged eye to enable the cornea to regenerate.

The success rate is about 68 per cent. It fails in three out of 10 people. They regrow a scar and they go blind again, Mr Shortt added.

We are trying to move to the point where we can use donor cells and prevent the body from rejecting them. Then we can treat patients more cheaply, and have a bank of cells ready to go as soon as we see a severe injury.

Next week NHS rationing body NICE is due to decide whether to approve an 80,000 Italian stem cell treatment, Holoclar, that works for seven in 10 patients. However, it is only effective where cells can be taken from the patients undamaged eye.

NICE is likely to require patients to undergo a cheaper treatment first.

Medics are also appealing for more people to donate their corneas after death. NHS Blood and Transplant said more than one in 10 donors place restrictions on the use of their eyes, the most for any organ.

About 70 corneas a week are needed but 50 are donated. Hundreds of corneas are imported from the US and Europe.

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Surgeons focus on stem cell transplants to help save sight of acid attack victims - Evening Standard