Archive for the ‘Gene Therapy Doctor’ Category

Baby Charlie remains on life support as parents fight doctors for experimental treatment – CBC.ca

He can’t cry, kick his legsor breathe unassisted. His body is growing;his brain is not.

Baby Charlie Gard, threeweeks shy of his first birthday, suffers from a rare degenerative disorder. There is no cure. He’s being kept alive on a ventilator at a renowned children’s hospital in London,where doctors want to take him off life support to allow him to “die with dignity.”

But Charlie’s parents are in a bitter legal fight to try one last experimental treatment in the U.S. that they know won’t say his life but which they say might at least improve it.

“He wakes up, he enjoys his tickles, we lie next to him,”said his mother, Connie Yates.

“If he was suffering, I couldn’t do it.I promise you.”

Charlie has mitochondrial depletion syndrome, which saps his muscles and organs of energy. He has brain damage and cannot move or cry. The disease is considered fatal. (Charlie Gard Facebook)

But whether or not he’s suffering is one of the many disputed facts in this case. Doctors say prolonging his life is causing sufferingand thatno existing treatment can save him.

The Pope, U.S. President Donald Trump and a variety of lawyers, ethicists and doctors have all weighed in on the British baby’s fate. As Charlie lies mute inhospital, an impassioned debate swirls around what rights parents have to control their children’s medical care.

Kaylom Hoppe, 5, leads chants to ‘save Charlie Gard’ outside London’s Court of Justice, which is hearing evidence in the Gard case Thursday. (Susan Ormiston/CBC)

Two previous courts have ruled with the doctors at London’s Great Ormond Hospital. The case is now being heard at Britain’s Supreme Court.Under British law, in the rare case when a dispute arises over a child’s treatment, a hospital can take it to the courts to decide.

Justice Nicholas Francis was expected to rule on the caseThursday morning but delayed the decision until the courtcan hear from the U.S. doctor who would administer the experimental treatment the family is seeking, called nucleoside bypass therapy.

The doctor is expected to testify via videolater this morning.

On Monday, in an emotional, high-spirited hearing before the court, Francisruled the parents had until Wednesday to deliver new evidence of treatment they believe could benefit their son.

“I’m still fighting for the same thing that I’ve been fighting for since November 2016,”said Charlie’s mother.

Even a 10-per-centchance of improvement, at best, is “a good enough chance to take oral medication with no major side effects,” she says.

A health-care facility in the U.S. hasagreed to care for the baby and administer the experimental treatment. It is not curative, but the family’s lawyers argue it could slightly improve the baby’s underlying condition.

Charlie’s father determined to get his baby to the U.S. for an experimental treatment called nucleoside bypass therapy that won’t save his life but that the parents think could improve his condition. (Charlie Gard Facebook )

Born healthy, Charlie’s muscles began to fail around sixmonths. Doctors discovered he had inherited the faulty RRM2B gene thatinhibitscells from making energy and hadmitochondrial depletion syndrome. His current care team says subsequent seizures in January caused irreparable brain damage. His parentsdisagree.

“I’ve yet to see something that tells me my son’s got irreversible structural brain damage,” said Yates.

She told the BBC thatother children with Charlie’s condition are currently on the experimental medication.

“They all have mitochondrial depletion syndrome as well as Charlie, but theirs is caused by a slightly different gene. They’re all getting stronger.”

“This isn’t about the parent’s right to control what happens to their child,” saidPenney Lewis, co-director of the Centre of Medical Law and Ethics at Kings College London.

Protesters stand outside a London court to show support for Charlie and his parents. (Reuters)

“Parents don’t have that right, [but] they do have a responsibility to take care of their child as best they can.

“I think one of the things that seems so heartbreaking is that the parents really believe that he could be saved, that he could have a much improved quality of life, and that doesn’t appear to be what the evidence suggests.”

Yates and Charlie’s father, Chris Gard, have used social media to their advantage to spread the word and to crowdfund enough money on GoFundMefor them to travel to the U.S. for treatment. More than 400,000 people have signed a petition calling on doctors to allow the baby to travel to the U.S. AFacebook page has been chartingthe legal steps in the case, and an American Christian group protesting euthanasia has arrived in London promoting#IamCharlieGard on Twitter.

The world’s most high-profile tweeter, U.S. President Trump, interjected his opinion on Monday:”If we can help little #CharlieGard, as per our friends in the U.K. and the Pope, we would be delighted to do so.”

Then on Wednesday, U.S. Vice-President Mike Pence used the case to make a political point in the U.S. health-care debate. Speaking to Rush Limbaugh on radio, Pence said the baby Gard case is evidence the “single payer”system of medicine doesn’t work.

“The mother and father should be able to choose the lifesaving treatment that’s available … instead of submitting to a government program, which says, ‘No,we’re going to remove the life support from your precious 11-month-old child.'”

But the experimental treatment will not save Charlie’s life, argue many top physicians in the U.K. If it would, doctors at Great Ormond wouldn’t hesitate to administer it.

“I think it is disturbing to watch,” Lewis said in an interview with CBC News. “I think sometimes, one loses sight of a very very unwell child. Because it becomes a kind ofpolitical circus in some sense.”

Britain’s top pediatrician,Neena Modi, wrote in an open letter,”Charlie’s situation is heartbreaking for his parents, and difficult for everyone, including the doctors and nurses looking after him.”

She said the interventions from high-profile figures are “unhelpful.”

But as Charlie lies in his tiny hospital cot, with round-the-clock care, his parents believe all the international attention has “saved his life so far.”

“We’re not strong people, but what is strong is the love for our little boy,”said Chris Gard.”He’s kept us going through all of this.”

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Baby Charlie remains on life support as parents fight doctors for experimental treatment – CBC.ca

Novel cancer treatment wins endorsement of FDA advisers – Washington Post

Food and Drug Administration advisers on Wednesday enthusiastically endorsed a first-of-its-kind cancer treatment that uses patients’ revved-up immune cells to fight the disease, concluding that the therapy’s benefits for desperately ill children far outweigh its potentially dangerous side effects.

The unanimous recommendation from theOncologic Drugs Advisory Committeemeans the treatment could be approved by the FDA by the end of September, forging a new path in the immunotherapy frontier.

8-year-old Ava Christiansen has been battling cancer for half her life. Now a new specialized cancer treatment may be able to keep her in remission. (Whitney Leaming/The Washington Post)

Timothy Cripe, a panel member who is an oncologist with Nationwide Children’s Hospitalin Columbus, Ohio, called the treatment the “most exciting thing I’ve seen in my lifetime.”

Novartis, the drugmaker behind the CAR T-cell therapy, is seeking approval to use it for children and young adults whose leukemia doesn’t respond to traditional treatments a group that numbers 600 or so patientsa year in this country. But the approach also is being tested for a range of diseases from non-Hodgkin lymphoma and multiple myeloma to solid tumors.

If cleared by the FDA, it would be the first gene therapy approved in the United States. But unlike traditional gene therapy, the new treatment doesn’t replace disease-causing genes with healthy ones. Instead, it uses technology to reprogram immune cells called T cells to target and attack malignancies.

When a patient is treated under the Novartis process, T cells are extracted from a patient’s blood, frozen and sent to the company’s plant in Morris Plains, N.J. There, the cells are genetically modified to attack the cancer, expanded in number, refrozen and shipped back to the patient for infusion.

Once inside the body, the cells multiply exponentially and go hunting for the CD19 protein, which appears on a kind of white blood cell that can give rise to diseases, such as leukemia and lymphoma.The turnaround time for manufacturing the therapy, called vein-to-vein time, will be an estimated 22 days, Novartis officials told the committee Wednesday.

From the start of Wednesday’smeeting, committee members made clear that they were not concerned about the treatment’s efficacy, which has been well established 83 percent of patients went into remission in the pivotal Novartis trial. Rather, the panel homed in onhow to best to handle possible shot-term toxicities, as well as long-term safety risks and manufacturing quality.

Most patients in the Novartis study experienced something called cytokine release syndrome, which causes fever and flulike symptoms that can range from mild to extremely severe, said Stephan Grupp, an oncologist at the Children’s Hospital of Philadelphia who led the Novartis trial. Some patients in that study also had neurological problems, including seizures and delirium. But there were no cases of fatal brain swelling, as occurred in another company’s trial, Grupp said.

To try to ensure safety, Novartis is limiting the therapy’s availability to 30 to 35 medical centers where personnel have had extensive training with the treatment. The company also plans to post Novartis employees at hospitals using the therapy and to follow patients for up to 15 years.

During the committee meeting, hundreds of people packed the hearing room at FDA headquarters in Silver Spring, Md., including prominent scientists, such as Carl June of the University of Pennsylvania, who developed the treatment. Though the FDA isn’t required to follow the guidance of its advisory committees, it usually does.

David Maloney, medical director for cellular immunotherapy at Fred Hutchinson Cancer Research Center in Seattle, said he was elated that the field is moving forward. It represents a paradigm shift in treating cancers, said Maloney, who is extensively involved in CAR T-cell research but not in the Novartis product.

One of the big issues in CAR-T cell therapy the cost, which analysts say could be in the hundreds of thousands of dollars wasn’t discussed because that is beyond the FDA’s purview. Novartis hasn’t released pricing information.

During the public comment portion of the hearing, Amy Kappen, whose 5-year-old daughter underwent CAR T-cell therapy in Philadelphia, called for approval. The treatment beat back her daughter’s cancer and brought back the sparkle in her eyes. And while she died three months later, our children deserve this chance, Kappen said.

For other parents, there were happier outcomes. Don McMahon, whose son Connor was treated at Duke Children’s Hospital in North Carolina, said the therapy was far less debilitating than what he endured on standard chemotherapy during two relapses. The boy, an avid hockey player, is doing well now.

Thomas Whitehead, whose daughter was the first pediatric patient to receive the treatment, choked up while telling panel members about Emily’s experience. She got CAR T-cell therapy when she was 6 and close to death from leukemia. The treatment almost killed her, but she recovered and today is cancer free.

“If you want to see what a cure looks like for relapsed ALL [acute lymphoblastic leukemia], shes standing right beside me,” said Whitehead, his voice cracking.

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Novel cancer treatment wins endorsement of FDA advisers – Washington Post

High-tech solutions top the list in the fight against eye disease – Engadget

Cataracts are the single leading cause of blindness worldwide, afflicting roughly 42 percent of the global population, including more than 22 million Americans. The disease, which causes cloudy patches to form on the eye’s normally clear lens, can require surgery if left untreated. That’s why Google’s DeepMind AI division has teamed with the UK’s National Health Service (NHS) and Moorfields Eye Hospital to train a neural network that will help doctors diagnose early stage cataracts.

The neural network is being trained on a million anonymized optical coherence tomography (OCT) scans (think of a sonogram, but using light instead of sound waves) in the hopes it will eventually be able to supplement human doctors’ analyses, increasing both the efficiency and accuracy of individual diagnoses.

“OCT has totally revolutionized the field of ophthalmology. It’s an imaging system for translucent structures that utilizes coherent light,” Dr. Julie Schallhorn, an assistant professor of ophthalmology at UC San Francisco, said. “It was first described in 1998 and it gives near-cell resolution of the cornea, retina and optic nerve.

“The optic nerve is only about 200 microns thick, but you can see every cell in it. It’s given us a much-improved understanding of the pathogenesis of diseases and also their response to treatments.” The new iteration of OCT also measures the phase-shift of refracted light, allowing doctors to resolve images down to the capillary level and observe the internal structures in unprecedented detail.

“We’re great at correcting refractive errors in the eyes so we can give you good vision far away pretty reliably, or up close pretty reliably,” Schallhorn continued. “But the act of shifting focus from distance to near requires different optical powers inside the eye. The way the eye handles this when you’re young is through a process called ‘accommodation.'” There’s a muscle that contracts and changes the shape of the lens to help you focus on close objects. When you get older, even before you typically develop cataracts, the lens will stiffen and reduce the eye’s ability to change its shape.

“The lenses that we have been putting in during cataract surgery are not able to mimic that [shapeshifting] ability, so people have to wind up wearing reading glasses,” Schallhorn said. There’s a lot of work in the field to find solutions for this issue and help restore the eye’s accommodation.

There are two front-runners for that: Accommodating lenses, which use the same ciliary muscle to shift focus, and multifocal lenses, which work just like your parents’ multifocal reading glasses except that they sit directly on the eye itself. The multifocals have been on the market for about a decade, though their design and construction has been refined over that time.

To ensure the lenses that doctors are implanting are just as accurate as the diseased ones they’re removing, surgeons are beginning to use optiwave refractive analysis. Traditionally, doctors relied on measurements taken before the surgery to know how to shape the replacement lenses and combined those with nomograms to estimate how powerful the new lens should be.

The key word there is “estimate.” “They especially have problems in patients who have already had refractive surgery like LASIK,” Schallhorn explained. The ORA system, however, performs a wavefront measurement of the cornea after the cataract has been removed to help surgeons more accurately pick the right replacement lens for the job.

Corneal inlays are also being used. These devices resemble miniature contact lenses but sit in a pocket on the cornea that’s been etched out with a LASIK laser to mimic the process of accommodation and provide a greater depth of focus. They essentially serve the same function as camera apertures. The Kamra lens from AcuFocus and the Raindrop Near Vision Inlay from Revision Optics are the only inlays approved by the FDA for use in the US.

Glaucoma afflicts more than 70 million people annually. This disease causes fluid pressure within the eye to gradually increase, eventually damaging the optic nerve that carries electrical signals from the eye to the brain. Normally, detecting the early stages of glaucoma requires a comprehensive eye exam by a trained medical professional — folks who are often in short supply in rural and underserved communities. However, the Cambridge Consultants’ Viewi headset allows anyone to diagnose the disease — so long as they have a smartphone and 10 minutes to spare.

The Viewi works much like the Daydream View, wherein the phone provides the processing power for a VR headset shell — except, of course, that instead of watching 360 degree YouTube videos, the screen displays the flashing light patterns used to test for glaucoma. The results are reportedly good enough to share with you eye doctor and take only about five minutes per eye. Best of all, the procedure costs only about $25, which makes it ideal for use in developing nations.

And while there is no known cure for glaucoma, a team of researchers from Stanford University may soon have one. Last July, the team managed to partially restore the vision of mice suffering from a glaucoma-like condition.

Normally, when light hits your eye, specialized cells in the retina convert that light into electrical signals. These signals are then transmitted via retinal ganglion cells, whose long appendages run along the optic nerve and spread out to various parts of the brain’s visual-processing bits. But if the optic nerve or the ganglion cells have been damaged through injury or illness, they stay damaged. They won’t just grow back like your olfactory sensory nerve.

However, the Stanford team found that subjecting mice to a few weeks of high-contrast visual stimulation after giving them drugs to reactivate the mTOR pathway, which has been shown to instigate new growth in ganglion cells, resulted in “substantial numbers” of new axons. The results are promising, though the team will need to further boost the rate and scope of axon growth before the technique can be applied to humans.

Researchers from Japan have recently taken this idea of cajoling the retina into healing itself and applied it to age-related macular degeneration cases. AMD primarily affects people aged 60 and over (hence the name). It slowly kills cells in the macula, the part of the eye that processes sharp detail, and causes the central focal point of their field of vision to deteriorate, leaving only the peripheral.

The research team from Kyoto University and the RIKEN Center for Developmental Biology first took a skin sample from a human donor, then converted it into induced pluripotent stem (IPS) cells. These IPS cells are effectively blank slates and can be coerced into redeveloping into any kind of cell you need. By injecting these cells into the back of the patient’s eye, they should regrow into retinal cells.

In March of this year, the team implanted a batch of these cells into a Japanese sexagenarian who suffers from AMD in the hope that the stem cells would take hold and halt, if not begin to reverse, the damage to his macula. The team has not yet been able to measure the efficacy of this treatment but, should it work out, the researchers will look into creating a stem-cell bank where patients could immediately obtain IPS cells for their treatment rather than wait months for donor samples to be converted.

And while there isn’t a reliable treatment for dry-AMD, wherein fatty protein deposits damage the Bruchs membrane, a potent solution for wet-AMD, which involves blood leaking into the eyeball, has been discovered in a most unlikely place: cancer medication. “Genentech started developing a new drug when an ophthalmologist in Florida just decided to inject the commercially available drug into patients eyes,” Schallhorn explained.

“Generally this is not a great idea because sometimes things will go terribly wrong,” she continued, “but this worked super-well. It basically stops and reverses the growth of these blood vessels.” The only problem is that the drugs don’t last, requiring patients to receive injections into their eyeballs every four to eight weeks. Genentech and other pharma companies are working to reformulate the drug — or at least develop a mechanical “reservoir” — so it has to be injected only once or twice a year.

Stem-cell treatments like those used in the Kyoto University trial have already proved potentially effective against a wide range of genomic diseases, so why shouldn’t it work on the rare genetic condition known as choroideremia? This disease is caused by a single faulty gene and primarily affects young men. Similar to AMD, choroideremia causes light-sensitive cells at the back of the eye to slowly wither and die, resulting in partial to complete blindness.

In April of 2016, a team of researchers from Oxford University performed an experimental surgery on a 24-year-old man suffering from the disease. They first injected a small amount of liquid into the back of the eye to lift a section of the retina away from the interior cellular wall. The team then injected functional copies of the gene into that same cavity, replacing the faulty copies and not only halting the process of cellular death but actually restoring a bit of the patient’s vision.

Gene therapy may be “surely the most efficient way of treating a disease,” lead author of the study, Oxford professor Robert MacLaren, told BBC News, but its widespread use is still a number of years away. Until then, good old-fashioned gadgetry will have to suffice. Take the Argus II, for example.

The Argus II bionic eye from Second Sight has been in circulation since 2013, when the FDA approved its use in treating retinitis pigmentosa. It has since gotten the go-ahead for use with AMD in 2015. The system leverages a wireless implant which sits on the retina and receives image data from an external camera that’s mounted on a pair of glasses. The implant converts that data into an electrical signal which stimulates the remaining retinal cells to generate a visual image.

The Argus isn’t the only implantable eyepiece. French startup Pixium Vision developed a similar system, the IRIS II, back in 2015 and implanted it in a person last November after receiving clearance from the European Union. The company is already in talks with the FDA to bring its IRIS II successor, a miniaturized wireless subretinal photovoltaic implant called PRIMA, to US clinical trials by the end of this year.

Ultimately, the goal is to be able to replace a damaged or diseased eye entirely, if necessary, using a robotic prosthetic. However, there are still a number of technological hurdles that must be overcome before that happens, as Schallhorn explained.

“The big thing that’s holding us back from a fully functional artificial eye is that we need to find a way to interface with the optic nerve and the brain in a way that we transmit signals,” she said. “That’s the same problem we’re facing with prosthetic limbs right now. But there are a lot of smart people in the field working on that, and I’m sure they’ll come up with something soon.”

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High-tech solutions top the list in the fight against eye disease – Engadget

‘Prehab’ therapy helps cancer patients prepare for treatment – KTBS

Receiving a cancer diagnosis is overwhelming, emotional and traumatic for patients. Usually, there’s so much information given to them at one time that it’s hard to remember all the details about their treatment.

Some doctors want to make sure patients are armed and ready for their cancer journey, and they’re doing it through a new type of therapy dubbed ‘prehab.’ It’s a way for patients to learn about lifestyle changes, exercises and signs of a more serious side effect before they start their treatment.

It was just a few months ago when Deanna Miller got the news that there was something suspicious on her mammogram.

“I thought it was just a cyst. I’m sure the biopsies will come back negative,” she recalled.

But it wasn’t.

“When I got the result, that’s when it hit me. It’s cancer!” said Miller.

Doctors diagnosed Miller with stage 3A Breast Cancer and ordered BRCA testing.

“It wasn’t until I had the BRCA testing that it really kicked in because now I have a genetic problem on top of having cancer,” explained Miller.

Knowing that she carried the gene, Miller used this knowledge as a way to help the future health of her family and to fight.

“I never had the opportunity to cry again,” she said.

Miller’s doctor is Dr. Frankie Holmes, an oncologist affiliated withMemorial Hermann Memorial City Medical Center. Dr. Holmes recommended Miller visit occupational therapist Emilia Dewi at TIRR Memorial Hermann Outpatient Rehabilitation at Memorial Hermann Memorial City Medical City.

“Prehab is basically part of a continuing care that starts at diagnosis to the treatment of the disease, whether it be surgery, chemotherapy, radiation,” explained Dewi.

Dewi works primarily with breast cancer patients who often suffer from lymphedema. She measures range of motion and volume in the arms as a baseline.

“I give my patients specific activities and restrictions and exercises post-surgery,” Dewi said. “Patients don’t know what to do post-surgery, and they don’t know what to look for.”

Dewi also explained the warning signs.

“For lymphedema, I tell patients to look for skin wrinkling. We watch for signs of heaviness. Also, if they can’t see their vein anymore,” explained Dewi.

Miller knew how to respond to her symptoms thanks to Dewi.

“The prehab made me aware of the symptoms. They are subtle and they can be confused with the surgery and swelling,” said Miller.

Memorial Hermann also offers prehab for head, neck and throat cancer patients where they can learn swallowing exercises to help them post-surgery. Other patients may receive fitness training to combat the weakness and fatigue of cancer treatment.

As for Miller, she’s still on her journey and doing well. She has a couple more chemo treatments and radiation, and we wish her the best.

Nicole Cross – Evening News Anchor/Health Correspondent

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‘Prehab’ therapy helps cancer patients prepare for treatment – KTBS

Tumor gene testing urged to tell if drug targets your cancer – The … – The Mainichi

In this 2015 family photo, Catherine “Katie” Rosenbaum is seen at a cancer fundraiser by Swim Across America. The Richmond, Va., womans endometrial cancer was successfully treated in a research study that found the immunotherapy Keytruda can target certain tumors that share a particular genetic flaw. (Family photo provided by Katie Rosenbaum via AP)

WASHINGTON (AP) — Colon cancer. Uterine cancer. Pancreatic cancer. Whatever the tumor, the more gene mutations lurking inside, the better chance your immune system has to fight back.

That’s the premise behind the recent approval of a landmark drug, the first cancer therapy ever cleared based on a tumor’s genetics instead of the body part it struck first. Now thousands of patients with worsening cancer despite standard treatment can try this immunotherapy — as long as genetic testing of the tumor shows they’re a candidate.

“It’s like having a lottery ticket,” said Johns Hopkins oncologist Dr. Dung Le, who helped prove the new use for the immunotherapy Keytruda. “We’ve got to figure out how to find these patients, because it’s such a great opportunity for them.”

Today, doctors diagnose tumors by where they originate — breast cancer in the breast, colon cancer in the colon — and use therapies specifically tested for that organ. In contrast, the Food and Drug Administration labeled Keytruda the first “tissue-agnostic” treatment, for adults and children.

The reason: Seemingly unrelated cancers occasionally carry a common genetic flaw called a mismatch repair defect. Despite small studies, FDA found the evidence convincing that for a subset of patients, that flaw can make solid tumors susceptible to immunotherapy doctors otherwise wouldn’t have tried.

“We thought these would be the hardest tumors to treat. But it’s like an Achilles heel,” said Hopkins cancer geneticist Bert Vogelstein.

And last month FDA Commissioner Scott Gottlieb told a Senate subcommittee his agency will simplify drug development for diseases that “all have a similar genetic fingerprint even if they have a slightly different clinical expression.”

It’s too early to know if what’s being dubbed precision immunotherapy will have lasting benefits, but here’s a look at the science.

WHO’S A CANDIDATE?

Hopkins estimates about 4 percent of cancers are mismatch repair-deficient, potentially adding up to 60,000 patients a year. Widely available tests that cost $300 to $600 can tell who’s eligible. The FDA said the flaw is more common in colon, endometrial and gastrointestinal cancers but occasionally occurs in a list of others.

“Say, ‘have I been tested for this?'” is Le’s advice for patients.

MUTATIONS AND MORE MUTATIONS

Most tumors bear 50 or so mutations in various genes, Vogelstein said. Melanomas and lung cancers, spurred by sunlight and tobacco smoke, may have twice as many. But tumors with a mismatch repair defect can harbor 1,500 mutations.

Why? When DNA copies itself, sometimes the strands pair up wrong to leave a typo — a mismatch. Normally the body spell checks and repairs those typos. Without that proofreading, mutations build up, not necessarily the kind that trigger cancer but bystanders in a growing tumor.

THE PLOT THICKENS

Your immune system could be a potent cancer fighter except that too often, tumors shield themselves. Merck’s Keytruda and other so-called checkpoint inhibitors can block one of those shields, allowing immune cells to recognize a tumor as a foreign invader and attack. Until now, those immunotherapies were approved only for a few select cancers — Keytruda hit the market for melanoma in 2014 — and they work incredibly well for some patients but fail in many others. Learning who’s a good candidate is critical for drugs that can cost $150,000 a year and sometimes cause serious side effects.

In 2012, Hopkins doctors testing various immunotherapies found the approach failed in all but one of 20 colon cancer patients. When perplexed oncologists told Vogelstein, “a light bulb went off.”

Sure enough, the one patient who fared well had a mismatch repair defect and a “mind-boggling” number of tumor mutations. The more mutations, the greater the chance that at least one produces a foreign-looking protein that is a beacon for immune cells, Vogelstein explained.

It was time to see if other kinds of cancer might respond, too.

WHAT’S THE DATA?

The strongest study, published in the journal Science, tested 86 such patients with a dozen different cancers, including some who had entered hospice. Half had their tumors at least shrink significantly, and 18 saw their cancer become undetectable.

It’s not clear why the other half didn’t respond. Researchers found a hint, in three patients, that new mutations might form that could resist treatment.

But after two years of Keytruda infusions, 11 of the “complete responders” have stopped the drug and remain cancer-free for a median of eight months and counting.

Catherine “Katie” Rosenbaum, 67, is one of those successes. The retired teacher had her uterus removed when endometrial cancer first struck, but five years later tumors returned, scattered through her pelvis and colon. She tried treatment after treatment until in 2014, her doctor urged the Hopkins study.

Rosenbaum took a train from Richmond, Virginia, to Baltimore for infusions every two weeks and then, after some fatigue and diarrhea side effects, once a month. Then the side effects eased and her tumors started disappearing. A year into the study she was well enough to swim a mile for a Swim Across America cancer fundraiser.

“Nothing else had worked, so I guess we could say it was a last hope,” said Rosenbaum, who now wants other patients to know about the option.

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Tumor gene testing urged to tell if drug targets your cancer – The … – The Mainichi

Charlie Gard: Medical experts weigh in on case of terminally-ill baby – The Independent

Medical experts are seeking out as the case of ill baby Charlie Gard becomes an international incident.

The parents’ hope that their terminally-ill child can be saved through experimental treatment has led to a series of court cases, along with interventions from Donald Trump and the Pope. The case revolves around whether the parents should be allowed to take the child to the US using money they’ve raised a move they hope would offer a chance of treating his illness, but which experts believe could cause more suffering to Charlie and has very little to no chance of success.

But medical experts suggest that the case is far more complex than the often simplistic tabloid coverage and presidential tweets may suggest. The courts have proven there is very little hope of the young boy being saved, they say but there is still some, mostly insignificant, chance that he could be, and could have been in the past.

Poor Charlie has a very rare disease in which the organelle which provides energy source used in daily cell life, called the mitochondrion, has a gene defect. The reason mitochondrial diseases are rare is because they are usually fatal. They show up in babies or young children more often than in later life. If not fatal they are progressive and cause serious neurological illnesses which cannot be cured.

Charlie has one of those most severe of mitochondrial diseases and is untreatable. Medicine is advancing at a wonderful speed but some illnesses are still fatal.

Gene therapy is in its infancy and is a promising field of human endeavour. But there are 6000 inheritable recessive conditions in humans – the prospect of them being cured is some time off.

When a decision to withdraw life support is made for a baby this is not taken lightly and there are often tears in the medical and nursing staff looking after such a baby. But ultimately there is not a cure for many rare diseases. 40% of all rare diseases are in children under age 5 years, and of those most are fatal. The combined burden of such conditions in the UK is such that 1 in 300 people have a rare disease.

Scientists and medical technology companies are making strides to find cures or treatments for many conditions. But alas, in the case of poor little Charlie, there are simply limits to medicine as we know it.

Estimates as to the success of any potential treatment vary but from almost impossible to impossible. But experts agree that there is little help in discussing those chances, because Charlie is now so ill that any benefits from treatment have been undermined.

“The theoretical possibility of benefit from the nucleoside treatment had disappeared after Charlie suffered brain damage from seizures earlier this year,” said Penney Lewis, professor of law and co-director of the Centre of Medical Law and Ethics at King’s College London. “These findings were based on extensive evidence from all of those involved in caring for Charlie, and from a number of independent experts including one chosen by Charlies parents, and the doctor based in the US who is offering to treat Charlie.”

Many experts agree that Charlie Gard should perhaps have been sent to undergo the treatment earlier on his life, at a point when his illness had caused fewer problems. Doing that would at least have allowed for a clearer view of whether the treatment would succeed, and if there was value in prolonging his life.

Charlie Gard should have been allowed to go to the US for experimental treatment back in April (or better January when it was first considered), not because he would have been cured but just because we couldn’t then be confident his life would have been “intolerable”, or not worth living,” said Julian Savulescu, who is the Uehiro Chair in Practical Ethics at the University of Oxford. “The rational strategy was to give a trial of treatment, say 3 months, and agree with the family to withdraw ventilation if there was no improvement. If this had been done we would now have some information on whether there is any prospect of improvement.

This is not a religious or right to life argument, or an argument based on compassion,” Professor Savulesco said. “It’s a secular ethical argument about the extreme complexity of judging someone’s life to be not worth living, or the prospects of having a life worth living not worth taking. The courts have deferred to one group of doctors who are experts in the facts but they are not experts in the ethics.

More than six months have passed since experimental therapy was first considered. We don’t know how bad Charlie’s brain damage is now. Whether experimental therapy is still warranted depends on whether there remains any prospect of any meaningful life. Perhaps the moment has passed.

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Charlie Gard: Medical experts weigh in on case of terminally-ill baby – The Independent

Tumor gene testing urged to tell if drug targets your cancer | KRQE … – KRQE News 13

WASHINGTON (AP) Colon cancer. Uterine cancer. Pancreatic cancer. Whatever the tumor, the more gene mutations lurking inside, the better chance your immune system has to fight back.

Thats the premise behind the recent approval of a landmark drug, the first cancer therapy ever cleared based on a tumors genetics instead of the body part it struck first. Now thousands of patients with worsening cancer despite standard treatment can try this immunotherapy as long as genetic testing of the tumor shows theyre a candidate.

Its like having a lottery ticket, said Johns Hopkins oncologist Dr. Dung Le, who helped prove the new use for the immunotherapy Keytruda. Weve got to figure out how to find these patients, because its such a great opportunity for them.

Today, doctors diagnose tumors by where they originate breast cancer in the breast, colon cancer in the colon and use therapies specifically tested for that organ. In contrast, the Food and Drug Administration labeled Keytruda the first tissue-agnostic treatment, for adults and children.

The reason: Seemingly unrelated cancers occasionally carry a common genetic flaw called a mismatch repair defect. Despite small studies, FDA found the evidence convincing that for a subset of patients, that flaw can make solid tumors susceptible to immunotherapy doctors otherwise wouldnt have tried.

We thought these would be the hardest tumors to treat. But its like an Achilles heel, said Hopkins cancer geneticist Bert Vogelstein.

And last month FDA Commissioner Scott Gottlieb told a Senate subcommittee his agency will simplify drug development for diseases that all have a similar genetic fingerprint even if they have a slightly different clinical expression.

Its too early to know if whats being dubbed precision immunotherapy will have lasting benefits, but heres a look at the science.

WHOS A CANDIDATE?

Hopkins estimates about 4 percent of cancers are mismatch repair-deficient, potentially adding up to 60,000 patients a year. Widely available tests that cost $300 to $600 can tell whos eligible. The FDA said the flaw is more common in colon, endometrial and gastrointestinal cancers but occasionally occurs in a list of others.

Say, have I been tested for this?’ is Les advice for patients.

MUTATIONS AND MORE MUTATIONS

Most tumors bear 50 or so mutations in various genes, Vogelstein said. Melanomas and lung cancers, spurred by sunlight and tobacco smoke, may have twice as many. But tumors with a mismatch repair defect can harbor 1,500 mutations.

Why? When DNA copies itself, sometimes the strands pair up wrong to leave a typo a mismatch. Normally the body spell checks and repairs those typos. Without that proofreading, mutations build up, not necessarily the kind that trigger cancer but bystanders in a growing tumor.

THE PLOT THICKENS

Your immune system could be a potent cancer fighter except that too often, tumors shield themselves. Mercks Keytruda and other so-called checkpoint inhibitors can block one of those shields, allowing immune cells to recognize a tumor as a foreign invader and attack. Until now, those immunotherapies were approved only for a few select cancers Keytruda hit the market for melanoma in 2014 and they work incredibly well for some patients but fail in many others. Learning whos a good candidate is critical for drugs that can cost $150,000 a year and sometimes cause serious side effects.

In 2012, Hopkins doctors testing various immunotherapies found the approach failed in all but one of 20 colon cancer patients. When perplexed oncologists told Vogelstein, a light bulb went off.

Sure enough, the one patient who fared well had a mismatch repair defect and a mind-boggling number of tumor mutations. The more mutations, the greater the chance that at least one produces a foreign-looking protein that is a beacon for immune cells, Vogelstein explained.

It was time to see if other kinds of cancer might respond, too.

WHATS THE DATA?

The strongest study, published in the journal Science, tested 86 such patients with a dozen different cancers, including some who had entered hospice. Half had their tumors at least shrink significantly, and 18 saw their cancer become undetectable.

Its not clear why the other half didnt respond. Researchers found a hint, in three patients, that new mutations might form that could resist treatment.

But after two years of Keytruda infusions, 11 of the complete responders have stopped the drug and remain cancer-free for a median of eight months and counting.

Catherine Katie Rosenbaum, 67, is one of those successes. The retired teacher had her uterus removed when endometrial cancer first struck, but five years later tumors returned, scattered through her pelvis and colon. She tried treatment after treatment until in 2014, her doctor urged the Hopkins study.

Rosenbaum took a train from Richmond, Virginia, to Baltimore for infusions every two weeks and then, after some fatigue and diarrhea side effects, once a month. Then the side effects eased and her tumors started disappearing. A year into the study she was well enough to swim a mile for a Swim Across America cancer fundraiser.

Nothing else had worked, so I guess we could say it was a last hope, said Rosenbaum, who now wants other patients to know about the option.

View original post here:
Tumor gene testing urged to tell if drug targets your cancer | KRQE … – KRQE News 13

Tumor gene testing urged to tell if drug targets your cancer – ABC News

Colon cancer. Uterine cancer. Pancreatic cancer. Whatever the tumor, the more gene mutations lurking inside, the better chance your immune system has to fight back.

That’s the premise behind the recent approval of a landmark drug, the first cancer therapy ever cleared based on a tumor’s genetics instead of the body part it struck first. Now thousands of patients with worsening cancer despite standard treatment can try this immunotherapy as long as genetic testing of the tumor shows they’re a candidate.

“It’s like having a lottery ticket,” said Johns Hopkins oncologist Dr. Dung Le, who helped prove the new use for the immunotherapy Keytruda. “We’ve got to figure out how to find these patients, because it’s such a great opportunity for them.”

Today, doctors diagnose tumors by where they originate breast cancer in the breast, colon cancer in the colon and use therapies specifically tested for that organ. In contrast, the Food and Drug Administration labeled Keytruda the first “tissue-agnostic” treatment, for adults and children.

The reason: Seemingly unrelated cancers occasionally carry a common genetic flaw called a mismatch repair defect. Despite small studies, FDA found the evidence convincing that for a subset of patients, that flaw can make solid tumors susceptible to immunotherapy doctors otherwise wouldn’t have tried.

“We thought these would be the hardest tumors to treat. But it’s like an Achilles heel,” said Hopkins cancer geneticist Bert Vogelstein.

And last month FDA Commissioner Scott Gottlieb told a Senate subcommittee his agency will simplify drug development for diseases that “all have a similar genetic fingerprint even if they have a slightly different clinical expression.”

It’s too early to know if what’s being dubbed precision immunotherapy will have lasting benefits, but here’s a look at the science.

WHO’S A CANDIDATE?

Hopkins estimates about 4 percent of cancers are mismatch repair-deficient, potentially adding up to 60,000 patients a year. Widely available tests that cost $300 to $600 can tell who’s eligible. The FDA said the flaw is more common in colon, endometrial and gastrointestinal cancers but occasionally occurs in a list of others.

“Say, ‘have I been tested for this?'” is Le’s advice for patients.

MUTATIONS AND MORE MUTATIONS

Most tumors bear 50 or so mutations in various genes, Vogelstein said. Melanomas and lung cancers, spurred by sunlight and tobacco smoke, may have twice as many. But tumors with a mismatch repair defect can harbor 1,500 mutations.

Why? When DNA copies itself, sometimes the strands pair up wrong to leave a typo a mismatch. Normally the body spell checks and repairs those typos. Without that proofreading, mutations build up, not necessarily the kind that trigger cancer but bystanders in a growing tumor.

THE PLOT THICKENS

Your immune system could be a potent cancer fighter except that too often, tumors shield themselves. Merck’s Keytruda and other so-called checkpoint inhibitors can block one of those shields, allowing immune cells to recognize a tumor as a foreign invader and attack. Until now, those immunotherapies were approved only for a few select cancers Keytruda hit the market for melanoma in 2014 and they work incredibly well for some patients but fail in many others. Learning who’s a good candidate is critical for drugs that can cost $150,000 a year and sometimes cause serious side effects.

In 2012, Hopkins doctors testing various immunotherapies found the approach failed in all but one of 20 colon cancer patients. When perplexed oncologists told Vogelstein, “a light bulb went off.”

Sure enough, the one patient who fared well had a mismatch repair defect and a “mind-boggling” number of tumor mutations. The more mutations, the greater the chance that at least one produces a foreign-looking protein that is a beacon for immune cells, Vogelstein explained.

It was time to see if other kinds of cancer might respond, too.

WHAT’S THE DATA?

The strongest study, published in the journal Science, tested 86 such patients with a dozen different cancers, including some who had entered hospice. Half had their tumors at least shrink significantly, and 18 saw their cancer become undetectable.

It’s not clear why the other half didn’t respond. Researchers found a hint, in three patients, that new mutations might form that could resist treatment.

But after two years of Keytruda infusions, 11 of the “complete responders” have stopped the drug and remain cancer-free for a median of eight months and counting.

Catherine “Katie” Rosenbaum, 67, is one of those successes. The retired teacher had her uterus removed when endometrial cancer first struck, but five years later tumors returned, scattered through her pelvis and colon. She tried treatment after treatment until in 2014, her doctor urged the Hopkins study.

Rosenbaum took a train from Richmond, Virginia, to Baltimore for infusions every two weeks and then, after some fatigue and diarrhea side effects, once a month. Then the side effects eased and her tumors started disappearing. A year into the study she was well enough to swim a mile for a Swim Across America cancer fundraiser.

“Nothing else had worked, so I guess we could say it was a last hope,” said Rosenbaum, who now wants other patients to know about the option.

This Associated Press series was produced in partnership with the Howard Hughes Medical Institute’s Department of Science Education. The AP is solely responsible for all content.

This story is part of Genetic Frontiers, AP’s ongoing exploration of the rapidly growing understanding of DNA and new attempts to manipulate it.

Continued here:
Tumor gene testing urged to tell if drug targets your cancer – ABC News

Cancer treatment is swiftly moving toward individualized molecular and genetic tools that Sparrow Cancer Center’s … – City Pulse

Field notes from a revolution in cancer treatment

Any cancer center, no matter how cutting edge its technology or cheerful its design, is a place where people get bad news.

But theres more good news about cancer treatment than most people think, and the game is changing month by month.

Cancer treatment is swiftly moving toward individualized molecular and genetic tools that Sparrow Cancer Centers director, James Herman, hopes will replace what he calls MOAB (Mother of All Bombs) forms of treatment such as radiation and chemotherapy.

Oncologist Tim McKenna, director of Sparrows breast clinic, has been in practice over 35 years. He said he more optimistic now than he has ever been.

I can see where maybe breast cancer surgeons will be standing on street corners with cardboard signs, McKenna said.

Already, many of McKennas patients never get a mastectomy or lumpectomy. Treatment that combines chemotherapy with monoclonal antibodies that target a patients particular cancer can get rid of a tumor without surgery in many cases.

McKenna said they take a couple of core samples and say, You know what? I guess there isnt any cancer left. Youre done. I predict that in 15 years, 20 years, that will be 90 percent of the cure.

Corrie Bourdon called it a brave new world, amazing and life-saving. As the cancer genetic counselor, a position created a year ago especially for her, Bourdon is Sparrows newest staff member and a herald of that new world.

If you remember the 90s sci-fi movie, Gattaca, its becoming real life, Bourdon said.

Now, when cancer is detected, on cologists ask a whole new set of questions, using a strange new vocabulary. McKenna rattled off a few of the big ones: Is the cancer estrogen receptor positive? Does it overexpress her2/neu? Whats the Oncotype score? Whats the MammaPrint score?

To sample just one spoonful of that jargon stew, MammaPrint is a 70-gene map of the cancer itself, a genetic analysis that helps doctors decide if systemic treatment is warranted, even though they cant prove the cancer might be somewhere else.

It allows us to predict recurrence down the road and take steps now, McKenna said.

Like the genetic engineers of Gattaca, Bourdon tests families for genes that predispose them to cancer. (People sometimes ask Bourdon if she designs babies. She tells them she doesnt.)

If a person carries the mutation, the next step is extra screening or even preventative surgery to keep them from getting the cancer.

The advances are exponentially increasing, she said. Cancer treatment and genetics are converging very, very quickly, if they are not already converged.

Until recently, as Sparrow oncologist Joseph Meunier explained it, many chemotherapy drugs have been designed to treat a particular type of cancer, based on the part of the body affected, such as lung or breast cancer.

But recently, Meunier and his team have been successful doing things they never thought they would do, like treating ovarian cancer with skin cancer drugs.

They wouldnt have thought of trying such a thing five years ago, because no body knew the two forms of cancer had the same genetic mutation in common.

Just the leaps for ward in the last 18 months its been absolutely unbelievable, Meunier said.

A month ago, the FDA approved a chemotherapy type drug for the treatment of a genetic mutation, regardless of the organ of origin.

Thats the first time thats ever happened, Meunier said. I cant even imagine, in the next 10 or 20 years, the way we look at therapy altogether is going to be entirely different.

Genetically tailored treatment is not a silver bullet, though. Bourdon said the environment still plays a huge role in how people get cancer.

Exposures to chemicals, pesticides, or they worked in a factory, Bourdon said. Ive heard a lot about the Dow Chemical Plant in Michigan. Or if someone was in the military, who knows what they were exposed to? But cancers have genes of their own that can be unlocked and, perhaps, manipulated to their distinct disadvantage.

I would not be surprised at all if we actually have a cure for cancer in the next 10, 20 years, or weve at least made such advancements that you just go to your doctor and take a pill to fix your gene and youre cured, Bourdon said.

Gordan Srkalovic, oncologist and director of Sparrows clinical trials program, took a more circumspect view. Srkalovic has been an oncologist for 18 years and did basic oncology research before that. He has been at Sparrow 14 years.

Are we going to conquer cancer? is a loaded question, he said. I dont think we will be able to cure every patient with cancer, at least not in my lifetime.

Its more likely, Srkalovic said, that cancer will be cut down to size, from a deadly, progressive disease to a chronic one that can be treated, comparable to hypertension or high blood pressure, and thats already happening.

The goal at the present is to reduce the burden of disease to the point the patient could have cancer cells, but the cancer is controlled, he said.

Its a more modest prognosis, but still a dramatic leap from cancer outcomes a generation ago.

When I started, you took a Magic Marker and you put an X over where you thought the cancer was, Herman said. Then Id pretend Id know what was going on with the treatment.

The cure rate for cancer has gone from 30 percent to 70 percent since Herman entered the field over 35 years ago.

That means the cancer is gone and they die of something else, he said. You dont hear about that 70 percent. They carry on and live their life. Its a revolution.

So McKenna may end up on a street corner with his cardboard sign after all. It seems perverse to dream of tearing down a building thats brand new, but the Herbert-Herman Cancer Center is a special case.

We love helping patients and curing them, but it would be amazing if we were all out of a job, Bourdon said.

I hope it is the last cancer center, Meunier said. I hope we dont have to build a new one again.

Will we be able to get rid of buildings like this? I hope so, Herman said. Herman is entitled to say that, with his name is on the place and all.

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Cancer treatment is swiftly moving toward individualized molecular and genetic tools that Sparrow Cancer Center’s … – City Pulse

What to Know About Charlie Gard, the Terminally Ill Baby Trump Wants to Help – TIME

President Donald Trump and Pope Francis have voiced their support for the parents of critically ill British baby Charlie Gard. The pair have been engaged in a long legal battle to take their son to the U.S. for treatment for a rare genetic disease.

Who is Charlie Gard, and why is his case so significant? Here’s what you should know.

In September 2016, Charlie Gard, who’s now 10 months old, was diagnosed with a rare genetic condition called mitochondrial depletion syndrome, which causes progressive muscle weakness and brain damage. He cannot move his limbs or eat or breathe without assistance.

His parents, Chris Gard and Connie Yates, are both carriers of the faulty gene, but were unaware of it until Charlie turned three months old. According to a fundraising page they set up, Charlie is only the sixteenth known person in the world with the condition.

Charlie’s parents believe an experimental medication offered in the U.S. may be able to help the child, who is on life support.

“After endlessly researching and speaking to [doctors] all over the world we found hope in a medication that may help him and a [doctor] in America has accepted him in his hospital,” wrote the parents on Charlie’s fundraising page. “It hasn’t been tried on anyone with his gene before . . . but it’s had success with another mitochondrial depletion syndrome called TK2 which is similar.”

Gard and Yates added that they “strongly feel . . . Charlie should get a chance to try these medications” and he has “literally has nothing to lose but potentially a healthier, happier life to gain.”

The pair set up a GoFundMe page to help raise money to send Charlie to the U.S. They raised 1.3 million ($1.68 million) in five months, with donations from 83,563 people.

Charlie’s doctors at London’s Great Ormond Street Hospital for Children, or GOSH, believe there is no cure for his condition, which is terminal.

A statement on the hospital’s website explains that “GOSH explored various treatment options” including nucleoside therapy, the experimental treatment offered by the U.S. hospital. “GOSH concluded that the experimental treatment, which is not designed to be curative, would not improve Charlies quality of life,” the statement says.

After balancing whether the experimental treatment was in Charlie’s best interests or not, the GOSH doctors said they thought it would be best to stop providing life support for Charlie and instead move on to a palliative care regime, allowing him to “die with dignity.”

“One of the factors that influenced this decision was that Charlies brain was shown to be extensively damaged at a cellular level. The clinician in the U.S. who is offering the treatment agrees that the experimental treatment will not reverse the brain damage that has already occurred,” the statement says.

“The entire highly experienced U.K. team, all those who provided second opinions and the consultant instructed by the parents all agreed that further treatment would be futile meaning it would be pointless or of no effective benefit,” it adds.

Because Charlie’s parents disagreed with the doctors’ decision about Charlie’s future treatment, the decision went to the Family Division of Britain’s High Court in London.

The High Court ruled last April “with the heaviest of hearts” that it was in Charlie’s best interests for GOSH to “lawfully withdraw all treatment save for palliative care to permit Charlie to die with dignity.

The judge said his decision not to allow Charlie to go to the U.S. was not related to funding. I dare say that medical science may benefit objectively from the experiment, but experimentation cannot be in Charlies best interests unless there is a prospect of benefit for him,” he said, referring to the trial treatment, The Guardian reported at the time.

On May 2, the couple took their fight to the Court of Appeal, asking the judges not to take away the only remaining hope.” However, on May 25, three Court of Appeal judges upheld the High Court ruling. Britain’s Supreme Court then agreed to review the case, but ruled that Charlie’s life support must be switched off.

Charlie’s parents took the case to the European Court of Human Rights (ECHR). But on June 27, the ECHR ruled that, in agreement with the domestic courts’ ruling, “undergoing experimental treatment with no prospects of success” would offer Gard “no benefit, and continue to cause him significant harm. In a statement acquired by The Guardian, the ECHR declared the decision as “final.”

Our thoughts are with Charlies parents on receipt of this news that we know will be very distressing for them,” said a spokesperson for GOSH. “Todays decision by the European court of human rights marks the end of what has been a very difficult process and our priority is to provide every possible support to Charlies parents as we prepare for the next steps.”

In a Facebook post , Charlie’s parents wrote that they are “utterly heartbroken.” The post added: “We’re not allowed to choose if our son lives and we’re not allowed to choose when or where Charlie dies. We and most importantly Charlie have been massively let down throughout this whole process.”

Following the ECHR’s decision, campaigners gathered at Buckingham Palace to protest the ruling, chanting “save Charlie Gard” and “release Charlie Gard” and holding placards, with one reading, “It’s murder.”

“If we can help little # CharlieGard, as per our friends in the U.K. and the Pope, we would be delighted to do so,” wrote Trump on Twitter Monday morning.

His comment came one day after Pope Francis said in a statement that he was following the case “with affection and sadness,” adding that he was praying that Gard’s parents’ “wish to accompany and treat their child until the end isnt neglected.

Continue reading here:
What to Know About Charlie Gard, the Terminally Ill Baby Trump Wants to Help – TIME

Why the super-rich are ploughing billions into the booming ‘immortality industry’ – Evening Standard

Imagine a world in which youre 90 years old and nowhere near middle-aged. An app on your phone has hacked your DNA code, so you know exactly when to go to the doctor to receive gene therapy to prevent all the diseases you dont yet have. A microchip in your skin sends out a signal if youre at risk of developing a wrinkle so you step out of the sun and hotfoot it to your dermatologist. Every evening you sync your brain-mapping device with The Cloud, so even if you were caught up in a fatal accident youd still be able to cheat death every detail of your life would simply be downloaded to one of the perfect silicon versions youd had made of yourself, ensuring you last until at least your 1,000th birthday.

This may sound like science fiction but it could be your fate provided you can afford it. If current research develops into medicine, in the London of the future the super-rich wont simply be able to buy the best things in life, theyll be able to buy life itself by transforming themselves into a bio-engineered super-race, capable of living, if not forever, then for vastly longer than the current UK life expectancy of 81 years.

The science of turning back the clock has never been more advanced. In Boston, a drug capable of reversing half a lifetime of ageing in mice is about to be tested on humans in a medical trial monitored by Nasa. NMN is a compound found naturally in broccoli which boosts levels of NAD, a protein involved in energy production that depletes as we get older. Professor David Sinclair, who headed up the initial research at Australias University of New South Wales, doses himself with 500mg daily, and claims that he has already become more youthful. According to blood tests analysing the state of the 48-year-olds cells, prior to taking the pills Sinclair was in the same physical shape as a 57-year-old, but now hes 31.4.

Meanwhile, Hollywood stars looking for the elixir of youth might want to keep a close eye on developments at Newcastle University where last February Professor Mark Birch-Machin identified, for the first time, the mitochondrial complex which depletes over time, causing skin to age. Mitochondria are the battery packs that power our cells so if we want to slow down ageing we need to keep them topped up; doing so would be transformative for our appearance. In the future, Birch-Machin believes, well not only be taking pills and applying cosmetics, well have implants in our skin. Implants will tell us the state of it how well our batteries are doing, how many free radicals, and will inform us how we are doing with our lifestyle, he says. You can store it, log it, have that linked to your healthcare package.

Such medical discoveries are being translated into treatment at an unprecedented rate. The day after the results of Birch-Machins study were published in The New York Times, his department was contacted by nine companies hoping to turn his research into revolutionary pharmaceuticals. In 2009, Elizabeth Blackburn, a professor of biology and physiology at the University of California, won a Nobel Prize for her work on telomeres, the protective tips on our chromosomes that break down as we get older, leaving us prone to age-related diseases. Blackburn discovered an enzyme called telomerase that can stop the shortening of telomeres by adding DNA like a plastic tip fixing the end of a fraying shoelace. Today, rich Californians now use telomeres therapy to prolong the life of their pets.

Last year, in Monterey, California, the start-up Ambrosia (founded by Dr Jesse Karmazin, a DC-based physician) began trialling the effect of blood transfusions, pumping blood from teenagers into older patients, following studies thatfound that blood plasma from young mice can rejuvenate old mice, improving their memory, cognition and physical activity.

Dr Richard Siow, who heads up the Age Research department at Kings College London, believes we may be soon reach a significant point in anti-ageing research because of the massive amounts of money allocated by governments and charities worldwide in the hope of making a breakthrough. Indeed, according to a survey by Transparency Market Research, by 2019 the anti-ageing market will be worth 151 billion worldwide. Life expectancy in many countries has already increased from 65-68 all the way through to 70, 80, 85 because people are now surviving heart disease, strokes and cancer, points out Siow, who has been studying anti-ageing compounds found in Indian spices and tea. We are now redefining what ageing means. How can we extend that period of health so were not a burden?

It is in Silicon Valley, however, that the really radical advances seem likely to be made. Freshly minted internet tycoons appear willing to pay any price to prolong their lives and a critical mass of geeks is working furiously towards understanding our biology at an unprecedented rate. Take Dmitry Itskov, the Russian billionaire founder of the life-extension non-profit 2045 Initiative, who is paying scientists to map the human brain so our minds can be decanted into a computer and either downloaded to a robot body or synced with a hologram. Or Joon Yun, a physician and hedge fund manager who insisted at an anti-ageing symposium of the California elite in March that ageing is simply a programming error encoded in our DNA. If something is encoded, you can crack the code, he told an audience which, according to The New Yorker, included multi-billionaire Google co-founder Sergey Brin and Goldie Hawn. Thermodynamically, there should be no reason we cant defer entropy indefinitely. We can end ageing forever.

And then theres PayPal founder (and Donald Trump supporter) Peter Thiel, who has a net worth of 2.1 billion and has reportedly invested in start-up Unity Biotechnology which aims to develop drugs that make many debilitating consequences of ageing as uncommon as polio. Thiel has also offered funding to individual researchers, such as Aubrey de Grey, the Chelsea-born, Cambridge and California-based gerontologist who ploughed the 11 million he inherited from his artist mother, Cordelia, into founding the Strategies for Engineered Negligible Senescence Research Foundation in Mountain View, which promotes the use of rejuvenation biotechnology in anti-ageing research.

Of course, the best known element of the immortality industry is cryogenic freezing. Despite its reputation as the last resort of wealthy cranks, it remains in business; at the Alcor cryonics facility in Arizona, 149 corpses have already been preserved in liquid nitrogen at a temperature of minus 196C since it was founded in 1972. Worldwide there are thousands of people signed up for cryogenics services, including Alcors 28 clients in the UK. The service doesnt come cheap (full-body freezing costs 165,000, while having your head cut off and frozen is around 60,000) but it has some impressive-sounding clients, including de Grey and Dr Anders Sandberg, research fellow at Oxford Universitys Future of Humanity Institute.

Its a gamble but its still much better than being dead, says Sandberg. He envisages a world in which the brain is paramount, so when his is revived it could be transformed into a sort of computer programme containing all of his memories of life on earth. If you actually exist as software you have a lot of options. I do enjoy having a physical body but why have just one when you could have lots of different ones?

Of course, if such experiments do come to fruition, they could have far reaching implications for our society. Already, a rapidly ageing population is placing enormous stress on healthcare and pension systems worldwide. De Grey sees the problem of over-population being cured by a dwindling birth-rate. Buthe says little about the impact this would have on the young.

Then theres the question of whether we will one day be living in a world defined by gaping differences in life expectancy where the haves live for 10 times longer than the have nots. Mortality has been the great equaliser from beggars to kings to emperors, says Dr Jack Kreindler, medical director at the Centre for Health & Human Performance in Harley Street. If people embark on really sophisticated, targeted therapies to repair damage to their cells… I think were definitely entering into them and us territory. As projected in Homo Deus, the best-selling book of Israeli academic Yuval Noah Harari, Kreindler adds, we could witness a schism in humanity where we have some people so bioengineered that only the very, very rich can sustain the amount of maintenance required to look after their enhancements, while others simply cant afford to do anything but be natural.

Nevertheless, the quest to overcome mortality continues apace. Last year, at a TEDx symposium Kreindler convened at the Science Museum, Daisy Robinton, a post-doctoral scientist at Harvard University, put forward the theory that ageing should be considered a disease in itself. She described the excitement in the medical community at the discovery of CRISPR/Cas9, a protein that seems to allow us to target and delete genetic mutations in our DNA. Gene editing provides an opportunity to not only cure genetic disease but also to prevent diseases from ever coming into being, Robinton claimed. To treat our susceptibilities before they ever transform into symptoms.

If this theory became fact, dying of old age might one day seem as outmoded as being felled by one of the mass killers of the past for which we get vaccinated. If gene editing on this scale is possible, Kreindler says we have to ask: Can your cells become immortal, can they live forever?

At the Centre for Health & Human Performance, treatments may still be firmly rooted in the 21st century, focused as they are on helping athletes optimise their fitness and celebritiessuch as David Walliams complete gruelling challenges for Sport Relief. But Kreindler is clearlyin awe of what the latestmedical advances might mean for the future of the human race.

I dont believe this should be only for the very rich, he says. If youre going to do things, dont just do it for the billionaires, do it for the billions.

Continued here:
Why the super-rich are ploughing billions into the booming ‘immortality industry’ – Evening Standard

Worlds Leading Biomarkers Congress | CPD Points …

Conference Series LLC Conferences invites all the participants across the globe to attend 8th International Conference on Biomarkers and Clinical Research during December 05-07, 2016 in Philadelphia, USA which includes prompt Keynote presentations, Oral talks, Poster presentations and Exhibitions.

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Conference Series LLC, the host of this conference is comprised of 3000+ Global Events with over 600+ Conferences, 1200+ Symposiums and 1200+Workshops on diverse Medical, Pharmaceutical, Clinical, Engineering, Science, Technology, Business and Management field is organizing conferences all over the globe.Biomarkers 2016 is the worlds largest multidisciplinarycancer meeting. Biomarkers and cancer conferencesinclude scientific keynote lectures, symposia, workshops, exhibitions with the support fromOncology SocietyandAmerican Oncology Society. Cancer conferences includeEuropean oncology conferences,surgical oncology global cancer conferenceandcancer conferences.

Track 1:Types of Biomarkers

Biomarkeris a characteristic diagnostic tool that is objectively measured and evaluated as an indicator of normalbiological processes, pathogenic processes or pharmacological responses to a therapeutic intervention. Biomarkers can be molecules, or genes, gene products, enzymes, or hormones referred asprotein biomarkers, analytical biomarkers, blood biomarkers, fluorescent biomarkers, circulating biomarkers and molecular biomarkers to quantify the degree of disease condition. Biomarkers are the measures used to perform a clinical assessment in case ofcancer biomarkers. They predict health states in individuals across populations so that appropriate therapeutic intervention can be planned. In the current scenario more than a thousand organizations and universities have contributed to the field of Biomarkers research especially molecular and cancer biomarkers, with its wings spreading across major organizations in USA, UK, Germany and China. The global biomarkers market is expected to grow from $29.3 billion in 2013 to $53.6 billion in 2018, a compound annual growth rate (CAGR) of 12.8%.Different types of biomarkers includeProtein biomarkers, Fluorescent biomarkers,Blood biomarkers, Cancer biomarkers, Analytical biomarkers andMolecular Biomarkers.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

12th Euro Global Summit onCancer Therapy, September 26-28 2016, London, UK; 13th GlobalOncologistsSummit, October 17-19 2016, Dubai, UAE; Global Summit onMelanoma, September 25-26, 2017 Rome, Italy; Multiple Myeloma Conference, October 15-17, 2017 Milan, Italy; Radiology Conference, October 23-25, 2017 Chicago, USA; 6thAnnual Asia-Pacific Prostate Society Conference, September 9 -10 Seoul, Korea; 68thAnnual German Society of Urology Congress, September 28-October 1, 2016 Leipzig, Germany; 72ndAnnual Canadian Urological Association Meeting, June 25-27, 2017 Toronto, Canada; 4th Annual Immuno-Oncology Summit, August 29-September 2, 2016 Boston, USA; 2nd Biomarkers, Diagnostics & Clinical Research Conference, September 19-20 Boston, USA; Biomarkers and Targeted Therapeutics in Sjgrens (BATTS) Conference, September 19-22, 2016 Oklahoma, USA; NCRI Cancer Conference, November 6-9, 2016 Liverpool, UK; 6th Munich Biomarker Conference, November 29-30, 2016 Munchen, Germany; Annual Meeting of American Association of Genitourinary Surgeons, April 27-30, 2017 Florida, USA.

Track 2: Cancer Biomarkers

Cancer biomarkers are used to detect the natural course of a tumour and are used to assess chances of developing cancer. Biomarkers in cancer screening play an important role in cancer detection and risk assessment to reduce cancer deaths. Tumour biomarkers are used to detect cancer development and progression. Uterine cervical cancer, endometrial cancer, trophoblastic neoplasms and ovarian cancer are gynaecologic malignancies for which tumour markers are in clinical use. Effective cancer biomarkers are used to reduce cancer mortality rates by facilitating diagnosis of cancers at early stages. Cancer biomarkers can also be used in diagnosis, risk assessment and recurrence of cancer.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

Oral Cancer Conference, August 18-20 2016, Portland, USA; Surgical Oncology Conference, August 29-31 2016, Sao Paulo, Brazil; Cancer Diagnostics Conference, May 8-10, 2017 Dubai, UAE; 3rd Prostate Cancer Conference, June 26-28, 2017 Baltimore, USA; Lymphoma Conference, July 24-26, 2017 Rome, Italy; 14thUrological Association of Asia Congress, July 20-24, 2016 Suntec, Singapore; 17thAsia-Pacific Prostate Cancer Conference, August 31- September 3, 2016 Melbourne, Australia; ASCO Genitourinary Cancers Symposium, February, 16-18, 2017 Orlando, USA; 2ndInternational Prostate Cancer Symposium, August 6 -7, 2016 Moscow, Russia, 13thMeeting of the EAU Robotic Urology Section, September 14-16, 2016 Milan Italy; 11thAnnual Congress of Russian Association of Oncological Urology, 05-07, 2016 Moscow, Russia; 36thInternational Urology Congress, October 20-23, 2016 Argentina, South America, 27thInternational Prostate Cancer Update, January 24-27, 2017 Colorado, USA.

Track 3:Functional Genomics and Cytogenetic Biomarkers

The branch ofgenomicsthat determines the biological function and complex association of the genes and their products depicts thefunctional genomics. The measurable degree of these parameters through various processes and equipment inclusive of Next generation sequencing, Personalized genome sequencing and mi-RNA sequencing utilizing cellular entities to predict SNP biomarkers, immuno fluorescent biomarkers,oxidative stress biomarkers, si-RNA and mi-RNA will aid in better understanding of the disease outcome. Thecytogeneticbiomarkers are a feasible diagnostic tool to detect DNA and chromatin damage.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

MolecularBiomarkers Conference, September 15-17 2016 Berlin, Germany; Cervical Cancer Conference, September 22-23 2016, Vienna, Austria; Surgical Oncology Conference, October 23-25, 2017 Chicago, USA; 2nd Cervical Cancer Conference, October 29 -31, 2017 Brussels, Belgium; Cancer World Conventaion, November 26-28, 2017 Frankfurt, Germany; 89thAnnual Italian Society of Urology Congress, October 15-18, 2016 Venezia Italy; 62ndAnnual Meeting of Czech Urological Society, October 19-21, 2016 Czech Budejovice, Czech Republic; ESMO 2017 Congress, September 08-12, 2017 Madrid, Spain; International Cancer Education Conference, September 14-16, 2016 Bethesda, USA; 16th Biennial Meeting of the International Gynecologic Cancer Society, October 29-31 Lisbon, Portugal; World Cancer Congress , October 31 – November 3 Paris, France, Malaysia Urology Conference, November 24-28,2016 Kuala Lumpur, Malaysia; Annual AUA Meeting, May 12-16, 2017 Boston, USA.

Track 4: Functional Transcriptomics and Profiling Techniques

The newly emerged discipline in the field of cytogenetic andfunctional genomicsis Molecular imaging biomarkers, aids in better visualization of the cellular function and the follow-up of the molecular process in living organisms without penetrance. Roche Diagnostics, GlaxoSmithKline, Siemens Healthcare, GE Healthcare and Merck & Co are a few of the key players in this market as observed inbiomarkerscongress. The functional genomics and cytogenetic market is estimated to reach 150M$ by 2017.Functional genomicscovers various areas of biomarkers applications like Next gen sequencing, Personalized genome sequencing, Micro RNA sequencing and SNP biomarkers.Cytogenetic biomarkersinclude Immuno flouscent biomarkers, Molecular imaging biomarkers, Oxidative Stress Biomarkers and si-RNA and mi-RNA.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

12th Euro Global Summit onCancer Therapy, September 26-28 2016, London, UK; 13th GlobalOncologistsSummit, October 17-19 2016, Dubai, UAE; Global Summit onMelanoma, September 25-26, 2017 Rome, Italy; Multiple Myeloma Conference, October 15-17, 2017 Milan, Italy; Radiology Conference, October 23-25, 2017 Chicago, USA; 6thAnnual Asia-Pacific Prostate Society Conference, September 9 -10 Seoul, Korea; 68thAnnual German Society of Urology Congress, September 28-October 1, 2016 Leipzig, Germany; 72ndAnnual Canadian Urological Association Meeting, June 25-27, 2017 Toronto, Canada; 4th Annual Immuno-Oncology Summit, August 29-September 2, 2016 Boston, USA; 2nd Biomarkers, Diagnostics & Clinical Research Conference, September 19-20 Boston, USA; Biomarkers and Targeted Therapeutics in Sjgrens (BATTS) Conference, September 19-22, 2016 Oklahoma, USA; NCRI Cancer Conference, November 6-9, 2016 Liverpool, UK; 6th Munich Biomarker Conference, November 29-30, 2016 Munchen, Germany; Annual Meeting of American Association of Genitourinary Surgeons, April 27-30, 2017 Florida, USA.

Track 5:Biomarkers in Clinical Research and Development

TheBiomarkersfinds its valuable application in the field ofClinical researchand development by case study and data management as evident through Biomarker conferences. The Bioethics and intellectual property right establishes the norms and standard of conduct of hypothesis with respect to clinical validation of biomarkers. The incorporation of biomarker inclinical trialsfor various disease conditions will put forth a valid diagnostic and therapeutic approach utilizing even the medical devices to detectclinical biomarkers. Currently this is the booming industry. Most of the reputed organizations like Pfizer, Parexel and Quintiles are into clinical research and development. The companies, hospitals and clinical research organizations are the hot spots for conducting clinical research with its growth rate increasing exponentially by an estimated 75B$ by 2016.In clinical research and development, clinical biomarkers are used in case study anddata management, clinical trials and in medical devices.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

Oral Cancer Conference, August 18-20 2016, Portland, USA; Surgical Oncology Conference, August 29-31 2016, Sao Paulo, Brazil; Cancer Diagnostics Conference, May 8-10, 2017 Dubai, UAE; 3rd Prostate Cancer Conference, June 26-28, 2017 Baltimore, USA; Lymphoma Conference, July 24-26, 2017 Rome, Italy; 14thUrological Association of Asia Congress, July 20-24, 2016 Suntec, Singapore; 17thAsia-Pacific Prostate Cancer Conference, August 31- September 3, 2016 Melbourne, Australia; ASCO Genitourinary Cancers Symposium, February, 16-18, 2017 Orlando, USA; 2ndInternational Prostate Cancer Symposium, August 6 -7, 2016 Moscow, Russia, 13thMeeting of the EAU Robotic Urology Section, September 14-16, 2016 Milan Italy; 11thAnnual Congress of Russian Association of Oncological Urology, 05-07, 2016 Moscow, Russia; 36thInternational Urology Congress, October 20-23, 2016 Argentina, South America, 27thInternational Prostate Cancer Update, January 24-27, 2017 Colorado, USA.

Track 6:Omics Technologies in Biomarkers Discovery and Validation

Biomarkersplay a critical role in disease diagnosis and treatment, especially for the early detection of cancer, to enable screening of asymptomatic populations. Recent omics technologies, such as Transcriptomics,genomicsand proteomics approaches besides Metabolomics are accelerating the rate of biomarker discovery. The incorporation of techniques like microarray data analysis, computational biology, data mining methods, Transcriptomics and profiling techniques are playing a crucial role in the validation of biomarkers. Since theHuman Genome Projectwas completed in April 2003, genome-wide association studies (GWAS) have contributed toward a greater understanding of the genetic basis of complex diseases and advances in high-throughput technologies. This has enabled researchers to rapidly map the genome of vertebrates, invertebrates and pathogens through cost-effective methods. The applications ofBioinformaticstool in biomarker research is the current emerging field promoting better diagnosable parameters. The global omics market was valued at nearly $2.8 billion in 2011, nearly $3.2 billion in 2012, and is forecast to grow to nearly $7.5 billion by 2017 after increasing at a compound annual growth rate (CAGR) of 18.7%. The omics technology segment holds the largest share of ~75% of the biomarker discovery market, primarily due to the increase in adoption ofproteomicsand genomics technologies, globally. There are several approaches in biomarkers discovery and validation likegenomics and proteomicapproaches, Microarray data analysis, Data mining methods and Transcriptomics and profiling techniques by making use of Computational biology and Application of Bioinformatics in biomarker discovery.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

2ndInternational Conference onProstate Cancerand Treatment, August 22-23, 2016, USA, Experts Meeting onGynaecologic Oncology, May 19-21, 2016 , USACancer DiagnosticsConference and Expo, June 13-15, 2016, Italy, 11thAsia PacificOncologistsConference, July 11-13, 2016, Kualalumpur, Malaysia, Global Summit onMelanoma and Carcinoma, July 14-15, 2016, Australia, Controversies inBreast Cancer (CoBRA) October 22-24, 2015, Australia, 16th Biennial Meeting of the InternationalGynaecologic CancerSociety, 29-31stOctober 2016, Lisbon, WSMOS FallOncologyConference, 30thOctober 2015,Uk, Next NRGOncologySemiannual Meeting, Jan 21-24th ,2015, Atlanta, Progress and Controversies inGynaecologic OncologyConference, 16-17 January 2015, Spain; 12thCancer ConferencesEurope September 26-28, 2016 London, UK; 12thOncology ConferencesEurope September 26-28, 2016 London, UK; 12thCancer Science EventsEurope September 26-28, 2016 London, UK;Cancer Global ConferencesMiddle East November 21-23, 2016 Dubai, UAE;Oncology ConferencesNovember 21-23, 2016 Dubai, UAE;Worldwide Cancer EventsNovember 21-23, 2016 Dubai, UAE;Breast Cancer ConferencesOctober 03-05, 2016 London, UK;Womens Health ConferencesOctober 03-05, 2016 London, UK.

Track 7:Biomarkers of Exposure Response and Susceptibility

Biomarkersof exposure are important in toxicology, because they are an indicator of internal exposure and genetic susceptibility to drug, chemicals or the amount ofchemicalexposure that got accumulated in the body. Significant advances have been made in developing analytical methods that detect and quantify many natural or synthetic toxins or their breakdown products in thebiologicalmatrix. The ability to accurately measure biomarkers of exposure depends upon an adequate understanding of the chemistry and toxicology of the substance under consideration.Epigenetic biomarkersalso quantify the degree of exposure to toxic dynamic and pharmacodynamics parameters inpathologicaland biochemical changes occurring due to exposure to harmful agents, brought to light by toxic dynamics meetings andpharmacodynamicsworkshops. This emerging field of study is gaining importance in industry with an estimate of more than 7,287 personnel conducting study across the globe. While studying the response and susceptibility parameters like toxic dynamic and pharmacodynamic parameters are taken into consideration to measure the internal exposure and genetic susceptibility to drugs and chemicals.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

MolecularBiomarkers Conference, September 15-17 2016 Berlin, Germany; Cervical Cancer Conference, September 22-23 2016, Vienna, Austria; Surgical Oncology Conference, October 23-25, 2017 Chicago, USA; 2nd Cervical Cancer Conference, October 29 -31, 2017 Brussels, Belgium; Cancer World Conventaion, November 26-28, 2017 Frankfurt, Germany; 89thAnnual Italian Society of Urology Congress, October 15-18, 2016 Venezia Italy; 62ndAnnual Meeting of Czech Urological Society, October 19-21, 2016 Czech Budejovice, Czech Republic; ESMO 2017 Congress, September 08-12, 2017 Madrid, Spain; International Cancer Education Conference, September 14-16, 2016 Bethesda, USA; 16th Biennial Meeting of the International Gynecologic Cancer Society, October 29-31 Lisbon, Portugal; World Cancer Congress , October 31 – November 3 Paris, France, Malaysia Urology Conference, November 24-28,2016 Kuala Lumpur, Malaysia; Annual AUA Meeting, May 12-16, 2017 Boston, USA.

Track 8:Biomarkers for Disorders

Biomarkers are the characteristicbiological measurableindictors for the various disorders if occurring inabnormal levels. These are used as quantitative entities for neurological disorders, genetic disorders, metabolic disorders, cardiac disorders and inborn errors. The present era is focusing on the cancer research utilizing biomarkers as indictor of disease conditions. The lungcancer biomarkersand biomarkers for breast cancer are inclusive of genes, enzymes, proteins and cell surface entitles. Registering a compound annual growth rate of 14.60% from 2011 to 2018, the market foroncology biomarkerswas valued at $13.16 billion in 2011 and is expected to be worth $29.78 billion in 2018.Biomarkers are also used in diagnosing and treating various diseases and disorders likeNeurological disorders, Genetic disorders,Metabolic disorders, Cardiac disorders, Inborn errors, Lung cancer and Breast cancer.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

12th Euro Global Summit onCancer Therapy, September 26-28 2016, London, UK; 13th GlobalOncologistsSummit, October 17-19 2016, Dubai, UAE; Global Summit onMelanoma, September 25-26, 2017 Rome, Italy; Multiple Myeloma Conference, October 15-17, 2017 Milan, Italy; Radiology Conference, October 23-25, 2017 Chicago, USA; 6thAnnual Asia-Pacific Prostate Society Conference, September 9 -10 Seoul, Korea; 68thAnnual German Society of Urology Congress, September 28-October 1, 2016 Leipzig, Germany; 72ndAnnual Canadian Urological Association Meeting, June 25-27, 2017 Toronto, Canada; 4th Annual Immuno-Oncology Summit, August 29-September 2, 2016 Boston, USA; 2nd Biomarkers, Diagnostics & Clinical Research Conference, September 19-20 Boston, USA; Biomarkers and Targeted Therapeutics in Sjgrens (BATTS) Conference, September 19-22, 2016 Oklahoma, USA; NCRI Cancer Conference, November 6-9, 2016 Liverpool, UK; 6th Munich Biomarker Conference, November 29-30, 2016 Munchen, Germany; Annual Meeting of American Association of Genitourinary Surgeons, April 27-30, 2017 Florida, USA.

Track 9:Techniques to Maximize Biomarker Identification

Biomarkersare the existing bimolecular and integral indictors of disease condition of biological systems. The techniques used to identify and maximize the expression of biomarkers include RT-PCR genotyping, molecular imaging and dynamics,biochemicalassay and profiling,immunologicaltechniques and chromatographic techniques. A wider approach towards identification of biomarkers lies in theproteomicapproach besides utilizing biosensors as a compatible tool for evaluation of biomarker levels in the biological systems. Most of the companys focus is on generating cost effective durable profiling techniques and equipment to quantify biomarkers within a short span of time. Johnson & Johnson, GlaxoSmithKline Plc., GEHealthcare, Affymetrix Inc., Bio-Rad Laboratories Inc. are a few of the key players in this market. Partnerships, agreements,collaborations, & mergers and acquisitions are the key business strategies adopted by market participants to ensure their growth in the market.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

Oral Cancer Conference, August 18-20 2016, Portland, USA; Surgical Oncology Conference, August 29-31 2016, Sao Paulo, Brazil; Cancer Diagnostics Conference, May 8-10, 2017 Dubai, UAE; 3rd Prostate Cancer Conference, June 26-28, 2017 Baltimore, USA; Lymphoma Conference, July 24-26, 2017 Rome, Italy; 14thUrological Association of Asia Congress, July 20-24, 2016 Suntec, Singapore; 17thAsia-Pacific Prostate Cancer Conference, August 31- September 3, 2016 Melbourne, Australia; ASCO Genitourinary Cancers Symposium, February, 16-18, 2017 Orlando, USA; 2ndInternational Prostate Cancer Symposium, August 6 -7, 2016 Moscow, Russia, 13thMeeting of the EAU Robotic Urology Section, September 14-16, 2016 Milan Italy; 11thAnnual Congress of Russian Association of Oncological Urology, 05-07, 2016 Moscow, Russia; 36thInternational Urology Congress, October 20-23, 2016 Argentina, South America, 27thInternational Prostate Cancer Update, January 24-27, 2017 Colorado, USA.

Track 10:Biomarkers in Nano science

Nano science is the study of structures and materials on the scale of nanometres.Nanotechnologymay be able to create many new materials and devices with a vast range of applications in medicine, electronics, biomaterialsenergy production, and consumer products. Nanotechnology is evolving rapidly with nanoparticles events. An estimated 1 million workers in R&D and production are involved in the field of Nano science and nanomaterial generation. Interaction of biomarkers with nanoparticles aids in identification and validation throughbiologicaland biomedical applications. Current marketholds Nano devices and nanomaterial for identification, quantifying, calibrating and even in surgeries. The US leads the world in investing and in the number ofNanotech Companies. Global consumption ofnanomaterialis expected to grow in unit terms from nearly 225,060 metric tons in 2014 to nearly 584,984 metric tons in 2019, a compound annual growth rate (CAGR) of 21.1% for the period of 2014 to 2019.Nano science is another rapidly growing area where application ofnanotechnologytobiomarkersis used for biological and biomedical applications like Nano devices.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

MolecularBiomarkers Conference, September 15-17 2016 Berlin, Germany; Cervical Cancer Conference, September 22-23 2016, Vienna, Austria; Surgical Oncology Conference, October 23-25, 2017 Chicago, USA; 2nd Cervical Cancer Conference, October 29 -31, 2017 Brussels, Belgium; Cancer World Conventaion, November 26-28, 2017 Frankfurt, Germany; 89thAnnual Italian Society of Urology Congress, October 15-18, 2016 Venezia Italy; 62ndAnnual Meeting of Czech Urological Society, October 19-21, 2016 Czech Budejovice, Czech Republic; ESMO 2017 Congress, September 08-12, 2017 Madrid, Spain; International Cancer Education Conference, September 14-16, 2016 Bethesda, USA; 16th Biennial Meeting of the International Gynecologic Cancer Society, October 29-31 Lisbon, Portugal; World Cancer Congress , October 31 – November 3 Paris, France, Malaysia Urology Conference, November 24-28,2016 Kuala Lumpur, Malaysia; Annual AUA Meeting, May 12-16, 2017 Boston, USA.

Track 11: Biomarkers in Toxicology

Biomarkers are used for detecting kidney toxicity. Kidney toxicity is detected using biomarkers serum creatinine and blood urea nitrogen. Many qualified biomarkers are used to develop products to conquer the kidney toxicity problem. Latest research on biomarkers discovered new approaches to predicting and recognising toxic exposures of macromolecular adducts and their potential consequences.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

12th Euro Global Summit onCancer Therapy, September 26-28 2016, London, UK; 13th GlobalOncologistsSummit, October 17-19 2016, Dubai, UAE; Global Summit onMelanoma, September 25-26, 2017 Rome, Italy; Multiple Myeloma Conference, October 15-17, 2017 Milan, Italy; Radiology Conference, October 23-25, 2017 Chicago, USA; 6thAnnual Asia-Pacific Prostate Society Conference, September 9 -10 Seoul, Korea; 68thAnnual German Society of Urology Congress, September 28-October 1, 2016 Leipzig, Germany; 72ndAnnual Canadian Urological Association Meeting, June 25-27, 2017 Toronto, Canada; 4th Annual Immuno-Oncology Summit, August 29-September 2, 2016 Boston, USA; 2nd Biomarkers, Diagnostics & Clinical Research Conference, September 19-20 Boston, USA; Biomarkers and Targeted Therapeutics in Sjgrens (BATTS) Conference, September 19-22, 2016 Oklahoma, USA; NCRI Cancer Conference, November 6-9, 2016 Liverpool, UK; 6th Munich Biomarker Conference, November 29-30, 2016 Munchen, Germany; Annual Meeting of American Association of Genitourinary Surgeons, April 27-30, 2017 Florida, USA.

Track 12: Biomarkers in Microbial Infections

Biomarkers can be used for microbial infections and can be used for early diagnosis and prognosis of the disease. The diagnostic performance of biomarkers is usually measured in terms of sensitivity.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

Oral Cancer Conference, August 18-20 2016, Portland, USA; Surgical Oncology Conference, August 29-31 2016, Sao Paulo, Brazil; Cancer Diagnostics Conference, May 8-10, 2017 Dubai, UAE; 3rd Prostate Cancer Conference, June 26-28, 2017 Baltimore, USA; Lymphoma Conference, July 24-26, 2017 Rome, Italy; 14thUrological Association of Asia Congress, July 20-24, 2016 Suntec, Singapore; 17thAsia-Pacific Prostate Cancer Conference, August 31- September 3, 2016 Melbourne, Australia; ASCO Genitourinary Cancers Symposium, February, 16-18, 2017 Orlando, USA; 2ndInternational Prostate Cancer Symposium, August 6 -7, 2016 Moscow, Russia, 13thMeeting of the EAU Robotic Urology Section, September 14-16, 2016 Milan Italy; 11thAnnual Congress of Russian Association of Oncological Urology, 05-07, 2016 Moscow, Russia; 36thInternational Urology Congress, October 20-23, 2016 Argentina, South America, 27thInternational Prostate Cancer Update, January 24-27, 2017 Colorado, USA.

Track 13: Biomarkers in Drug Discovery

The role of Biomarkers in drug discovery and development is to understand the pathophysiology of disease. Biomarkers can be a clinical tool for drug discovery and development by confirming the efficacy and safety to the right patient. Biomarkers can be used in understanding the mechanism of drug.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

MolecularBiomarkers Conference, September 15-17 2016 Berlin, Germany; Cervical Cancer Conference, September 22-23 2016, Vienna, Austria; Surgical Oncology Conference, October 23-25, 2017 Chicago, USA; 2nd Cervical Cancer Conference, October 29 -31, 2017 Brussels, Belgium; Cancer World Conventaion, November 26-28, 2017 Frankfurt, Germany; 89thAnnual Italian Society of Urology Congress, October 15-18, 2016 Venezia Italy; 62ndAnnual Meeting of Czech Urological Society, October 19-21, 2016 Czech Budejovice, Czech Republic; ESMO 2017 Congress, September 08-12, 2017 Madrid, Spain; International Cancer Education Conference, September 14-16, 2016 Bethesda, USA; 16th Biennial Meeting of the International Gynecologic Cancer Society, October 29-31 Lisbon, Portugal; World Cancer Congress , October 31 – November 3 Paris, France, Malaysia Urology Conference, November 24-28,2016 Kuala Lumpur, Malaysia; Annual AUA Meeting, May 12-16, 2017 Boston, USA.

Track 14: Personalized Medicine and Data Analysis

Recently there has been enhanced and advanced biomedical technology such as high-throughput molecular imaging and microarrays to monitor SNPs, gene and protein expressions, to provide exhaustive situations for individuals. The biological and medical status from such data sets, which are viewed as biomarkers in a wide sense to help to do identification, association, and prediction studies for phenotypes such as cancer subtypes, prognosis, treatment responsiveness, and adverse reactions for personalized medicine.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

12th Euro Global Summit onCancer Therapy, September 26-28 2016, London, UK; 13th GlobalOncologistsSummit, October 17-19 2016, Dubai, UAE; Global Summit onMelanoma, September 25-26, 2017 Rome, Italy; Multiple Myeloma Conference, October 15-17, 2017 Milan, Italy; Radiology Conference, October 23-25, 2017 Chicago, USA; 6thAnnual Asia-Pacific Prostate Society Conference, September 9 -10 Seoul, Korea; 68thAnnual German Society of Urology Congress, September 28-October 1, 2016 Leipzig, Germany; 72ndAnnual Canadian Urological Association Meeting, June 25-27, 2017 Toronto, Canada; 4th Annual Immuno-Oncology Summit, August 29-September 2, 2016 Boston, USA; 2nd Biomarkers, Diagnostics & Clinical Research Conference, September 19-20 Boston, USA; Biomarkers and Targeted Therapeutics in Sjgrens (BATTS) Conference, September 19-22, 2016 Oklahoma, USA; NCRI Cancer Conference, November 6-9, 2016 Liverpool, UK; 6th Munich Biomarker Conference, November 29-30, 2016 Munchen, Germany; Annual Meeting of American Association of Genitourinary Surgeons, April 27-30, 2017 Florida, USA.

Track 15: Nutritional Biomarkers

A nutritional biomarker can be any biological specimen that is an indicator of nutritional status with respect to intake or metabolism of dietary constituents. It can be a biochemical, functional or clinical index of status of an essential nutrient or other dietary constituent. Nutritional biomarkers may be interpreted more broadly as a biologic consequence of dietary intake or dietary patterns.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

Oral Cancer Conference, August 18-20 2016, Portland, USA; Surgical Oncology Conference, August 29-31 2016, Sao Paulo, Brazil; Cancer Diagnostics Conference, May 8-10, 2017 Dubai, UAE; 3rd Prostate Cancer Conference, June 26-28, 2017 Baltimore, USA; Lymphoma Conference, July 24-26, 2017 Rome, Italy; 14thUrological Association of Asia Congress, July 20-24, 2016 Suntec, Singapore; 17thAsia-Pacific Prostate Cancer Conference, August 31- September 3, 2016 Melbourne, Australia; ASCO Genitourinary Cancers Symposium, February, 16-18, 2017 Orlando, USA; 2ndInternational Prostate Cancer Symposium, August 6 -7, 2016 Moscow, Russia, 13thMeeting of the EAU Robotic Urology Section, September 14-16, 2016 Milan Italy; 11thAnnual Congress of Russian Association of Oncological Urology, 05-07, 2016 Moscow, Russia; 36thInternational Urology Congress, October 20-23, 2016 Argentina, South America, 27thInternational Prostate Cancer Update, January 24-27, 2017 Colorado, USA.

Track 16: Current Research Concepts in Biomarkers

Current Research Concepts in Biomarkers include research in glucose disorders, Biomarkers in disease and health, technologies in biomarker discovery, translational biomarker research and the use of biomarkers in pre-clinical and clinical studies.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

MolecularBiomarkers Conference, September 15-17 2016 Berlin, Germany; Cervical Cancer Conference, September 22-23 2016, Vienna, Austria; Surgical Oncology Conference, October 23-25, 2017 Chicago, USA; 2nd Cervical Cancer Conference, October 29 -31, 2017 Brussels, Belgium; Cancer World Conventaion, November 26-28, 2017 Frankfurt, Germany; 89thAnnual Italian Society of Urology Congress, October 15-18, 2016 Venezia Italy; 62ndAnnual Meeting of Czech Urological Society, October 19-21, 2016 Czech Budejovice, Czech Republic; ESMO 2017 Congress, September 08-12, 2017 Madrid, Spain; International Cancer Education Conference, September 14-16, 2016 Bethesda, USA; 16th Biennial Meeting of the International Gynecologic Cancer Society, October 29-31 Lisbon, Portugal; World Cancer Congress , October 31 – November 3 Paris, France, Malaysia Urology Conference, November 24-28,2016 Kuala Lumpur, Malaysia; Annual AUA Meeting, May 12-16, 2017 Boston, USA

Track 17: Oncologists: Biomarkers

An oncologist is a doctor who specializes in treating people with cancer. The oncologists research into the causes, prevention, detection, and treatment of cancer is going on in many medical centres throughout the world.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

12th Euro Global Summit onCancer Therapy, September 26-28 2016, London, UK; 13th GlobalOncologistsSummit, October 17-19 2016, Dubai, UAE; Global Summit onMelanoma, September 25-26, 2017 Rome, Italy; Multiple Myeloma Conference, October 15-17, 2017 Milan, Italy; Radiology Conference, October 23-25, 2017 Chicago, USA; 6thAnnual Asia-Pacific Prostate Society Conference, September 9 -10 Seoul, Korea; 68thAnnual German Society of Urology Congress, September 28-October 1, 2016 Leipzig, Germany; 72ndAnnual Canadian Urological Association Meeting, June 25-27, 2017 Toronto, Canada; 4th Annual Immuno-Oncology Summit, August 29-September 2, 2016 Boston, USA; 2nd Biomarkers, Diagnostics & Clinical Research Conference, September 19-20 Boston, USA; Biomarkers and Targeted Therapeutics in Sjgrens (BATTS) Conference, September 19-22, 2016 Oklahoma, USA; NCRI Cancer Conference, November 6-9, 2016 Liverpool, UK; 6th Munich Biomarker Conference, November 29-30, 2016 Munchen, Germany; Annual Meeting of American Association of Genitourinary Surgeons, April 27-30, 2017 Florida, USA.

Track 18: Biomarkers in Market

With the emerging importance to quantify and validate various disease conditions, many organizations, companies, and universities have stepped forward to contribute to the field of biomarkers discovery and quantification for better prognosis of disease conditions. The Biomarkers is the second leading industry after clinical research and development. The Biomarkers in pharmaceutical industry, biomarkers in oncology & other diseases has attained utmost recognition due to global spread of cancer and other diseases. The Biomarkers validation and regulatory affairs and diagnostic biomarker are booming industry with an estimate of more than 270 companies involved across the globe in 2016.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

MolecularBiomarkers Conference, September 15-17 2016 Berlin, Germany; Cervical Cancer Conference, September 22-23 2016, Vienna, Austria; Surgical Oncology Conference, October 23-25, 2017 Chicago, USA; 2nd Cervical Cancer Conference, October 29 -31, 2017 Brussels, Belgium; Cancer World Conventaion, November 26-28, 2017 Frankfurt, Germany; 89thAnnual Italian Society of Urology Congress, October 15-18, 2016 Venezia Italy; 62ndAnnual Meeting of Czech Urological Society, October 19-21, 2016 Czech Budejovice, Czech Republic; ESMO 2017 Congress, September 08-12, 2017 Madrid, Spain; International Cancer Education Conference, September 14-16, 2016 Bethesda, USA; 16th Biennial Meeting of the International Gynecologic Cancer Society, October 29-31 Lisbon, Portugal; World Cancer Congress , October 31 – November 3 Paris, France, Malaysia Urology Conference, November 24-28,2016 Kuala Lumpur, Malaysia; Annual AUA Meeting, May 12-16, 2017 Boston, USA.

Track 19: Biomarkers Case Reports

Biomarkers case reports play a crucial role in moving new treatments to patients who need those most, securing data so regulatory approvals can be obtained and new drugs can move into widespread clinical practice.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

12th Euro Global Summit onCancer Therapy, September 26-28 2016, London, UK; 13th GlobalOncologistsSummit, October 17-19 2016, Dubai, UAE; Global Summit onMelanoma, September 25-26, 2017 Rome, Italy; Multiple Myeloma Conference, October 15-17, 2017 Milan, Italy; Radiology Conference, October 23-25, 2017 Chicago, USA; 6thAnnual Asia-Pacific Prostate Society Conference, September 9 -10 Seoul, Korea; 68thAnnual German Society of Urology Congress, September 28-October 1, 2016 Leipzig, Germany; 72ndAnnual Canadian Urological Association Meeting, June 25-27, 2017 Toronto, Canada; 4th Annual Immuno-Oncology Summit, August 29-September 2, 2016 Boston, USA; 2nd Biomarkers, Diagnostics & Clinical Research Conference, September 19-20 Boston, USA; Biomarkers and Targeted Therapeutics in Sjgrens (BATTS) Conference, September 19-22, 2016 Oklahoma, USA; NCRI Cancer Conference, November 6-9, 2016 Liverpool, UK; 6th Munich Biomarker Conference, November 29-30, 2016 Munchen, Germany; Annual Meeting of American Association of Genitourinary Surgeons, April 27-30, 2017 Florida, USA.

Track 20: Biomarkers: Entrepreneur Investments Meet

A key ingredient in successful entrepreneurship is self-knowledge. Biomarkers-2016 aims to bring together all existing and budding bio entrepreneurs to share experiences and present new innovations and challenges in cancer community. Each year, over a million companies are started in the world with about 510 of them classified as high technology companies. Turning ideas into business ventures is tricky and the opportunity-recognition step is critical in new venture creation. This gestalt in the entrepreneur’s perception of the relationship between the invention and final product is refined into a business model that describes how the venture will make money or provide an appropriate return to the potential investors. Cancer science is complex and rapidly changing and requires a specialized knowledge to understand the value of the innovation and its competitive position in the industry. This three day community-wide conference will be a highly interactive forum that will bring experts in areas ranging from Biomarkers to signalling pathways to novel therapeutic approaches to the scientific hub. In addition to our outstanding speakers, we will also showcase short talks and poster presentations from submitted abstracts .The speakers will discuss state-of-the-art treatments, current guidelines, clinical challenges, and review recent trial data and emerging therapeutic approaches with the potential to impact clinical practice. This session will include combined efforts of World-renowned speakers, the most recent techniques, developments, and the newest updates in Biomarkers.

Related Biomarkers Conferences | Cancer Conferences | Biomarkers Meetings

Oral Cancer Conference, August 18-20 2016, Portland, USA; Surgical Oncology Conference, August 29-31 2016, Sao Paulo, Brazil; Cancer Diagnostics Conference, May 8-10, 2017 Dubai, UAE; 3rd Prostate Cancer Conference, June 26-28, 2017 Baltimore, USA; Lymphoma Conference, July 24-26, 2017 Rome, Italy; 14thUrological Association of Asia Congress, July 20-24, 2016 Suntec, Singapore; 17thAsia-Pacific Prostate Cancer Conference, August 31- September 3, 2016 Melbourne, Australia; ASCO Genitourinary Cancers Symposium, February, 16-18, 2017 Orlando, USA; 2ndInternational Prostate Cancer Symposium, August 6 -7, 2016 Moscow, Russia, 13thMeeting of the EAU Robotic Urology Section, September 14-16, 2016 Milan Italy; 11thAnnual Congress of Russian Association of Oncological Urology, 05-07, 2016 Moscow, Russia; 36thInternational Urology Congress, October 20-23, 2016 Argentina, South America, 27thInternational Prostate Cancer Update, January 24-27, 2017 Colorado, USA.

OMICS International hosted the 6thInternational Conference on Biomarkers & Clinical Research (Biomarkers 2015) during August 31September 02 at Toronto Airport Marriott Hotel, Toronto, Canada. The scientific meeting has laid path for the designing and development of research methodologies with the theme impact of Lab to industry as bio-signatures to therapeutic discovery.

Biomarkers 2015 was fortunate to acquire support from association and societies – Clinical Research Association of Canada (CRAC), Hypertension Canada, International Society for Cellular Therapy (ISCT), The Egyptian Biophysical Society and media partners -Biomarkers Profile Corporation, Gate2Biotech, The Technology Networks, Council of European Bio-Region, Oncology Education and Edinburgh Science Triangle.

The highlights of the meeting were the eponymous lectures, delivered byDr. Claude Prigent, University of Rennes, France, Dr. Trevor G Marshall, Autoimmunity Research Foundation, USA, Dr. Alain Moreau, Sainte-Justine University Hospital, Canada, Dr. Sergey Suchkov, I. M. Sechenov First Moscow State Medical University, Russia, Dr. Alexander M Buko, Human Metabolome Technologies, USA, Dr. Chee Gee See, Proteome Sciences, UK, Dr. Biswendu B Goswami, FDA Center for Food Safety and Applied Nutrition, USA.

Biomarkers 2015 held pre-conference workshop on August 1, 2015 in Mumbai University, India under the supervision of Prof. K. P. Mishra, Founder President of Society of Radiation Research, India. The workshop gathered 650+ participants inclusive of students, faculty, societies and industrial personnel.

The conference held 2 workshops under the supervision of Prof. Sergey Suchkov, I. M. Sechenov First Moscow State Medical University, Russia; Dr. Trevor G Marshall, Autoimmunity Research Foundation, USA and their team from Czech Republic and Prof. Youhe Gao, Beijing Normal University, China.

Biomarkers-2014

The5thInternational Conference on Biomarkers & Clinical Research, the Biomarker-2014, was held during April 15-17, 2014 at Oxford, UK.

Biomarkers-2014 has taken up the scientific thoughts towards proving the importance of accurate diagnostics to be prevital towards the curing efficacy. The scientific meeting has laid path for the designing and development of research methodologies with the theme impact of Diagnostic significance of the therapeutic bio-clinical molecule.

The conference was greeted by the welcome message from Presidents desk at the European Association for Predictive, Preventive and Personalised Medicine (EPMA), Brussels, EU. The support was extended through the PPPM workshop being conducted with the PPPM representatives from Russia, USA, Czech Republic and Saudi Arabia. The conference has gathered support from Everest Biotech, EuroScienceCon, Biomarkers Profile Corporation, ArrayMold, BioNews, Edinburgh Science Triangle, Biowebspin, The Technology Networks, European Biotechnology Thematic Network Association, Visiongain and Current Partnering as the media partners. In addition SCIENION has participated at the conference as Exhibitor at this conference.

The program highlights of the meeting were the eponymous lectures, delivered byDr. Sergey Suchkovfrom I.M.Sechenov First Moscow State Medical University, Russia;Dr.Pavel Vodickafrom Institute of Experimental Medicine, Czech Republic;Dr.Ondrej Topolcan from Charles University in Prague, Czech Republic;Dr. Claudio Nicolinifrom University of Genova, Italy andDr. Claude Prigentfrom University of Rennes, France.

Biomarkers-2013

OMICS Grouporganized 4thInternational Conference on Biomarkers & Clinical Research, during July 15-17, 2013 at Philadelphia, USA under the theme of Impact of Biomarkers Development in Health Diagnostics and Clinical Research.

The conference was initiated with a series of invited lectures delivered by Dr. Jizu Yi from BD Diagnostics, USA; Dr. Yaping Tian from PLA General Hospital, China; Dr. Leticia Cano from Biomarker Profile Corporation, USA and Dr. Lawrence Greenfield from Affymetrix, USA.

Biomarkers-2012

The3rd International Conference on Biomarkers & Clinical Research, organized by theOMICS Groupwas held onJuly 2-4, 2012 at Embassy Suites Las Vegas, USA under the theme of “Commercialization of Biomarkers”. There were about 200 delegates representing 25 countries from different corners of the world who made this conference a big success in the field ofBiomarkers and Clinical Research.

The conference was initiated with a series of invited lectures delivered by both Honorable Guests and members of the Keynote forum. The list includesDr. Josip Blonder, Frederick National Laboratory for Cancer Research (NIH), USA;Dr. Marcel M. Daadi, Stanford University, USA;Dr. Ting-Chao Chou, Memorial Sloan-Kettering Cancer Center, USA;Dr. Jacob Kagan, National Cancer Institute, NIH, USA;Dr. Michael Sullivan, Worldwide Clinical Trials-Drug Development Solutions, USA;Dr. Hitoshi Sohma, Sapporo Medical University Center for Medical Education, Japan andDr. Da Zhi Liu, University of California at Devis, USA.All the above mentioned Honourable Guests and Keynote speakers gave their energetic and fruitful contributions atBiomarkers-2012. All accepted abstracts have been indexed in OMICS Group Journal of Molecular Biomarkers & Diagnosisas a special issue.

Biomarkers-2011

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Worlds Leading Biomarkers Congress | CPD Points …

Breast Cancer Risk Factors: Genetics

About 5% to 10% of breast cancers are thought to be hereditary, caused by abnormal genes passed from parent to child.

Genes are particles in cells, contained in chromosomes, and made of DNA (deoxyribonucleic acid). DNA contains the instructions for building proteins. And proteins control the structure and function of all the cells that make up your body.

Think of your genes as an instruction manual for cell growth and function. Abnormalities in the DNA are like typographical errors. They may provide the wrong set of instructions, leading to faulty cell growth or function. In any one person, if there is an error in a gene, that same mistake will appear in all the cells that contain the same gene. This is like having an instruction manual in which all the copies have the same typographical error.

Most inherited cases of breast cancer are associated with two abnormal genes: BRCA1 (BReast CAncer gene one) and BRCA2 (BReast CAncer gene two).

Everyone has BRCA1 and BRCA2 genes. The function of the BRCA genes is to repair cell damage and keep breast, ovarian, and other cells growing normally. But when these genes contain abnormalities or mutations that are passed from generation to generation, the genes don’t function normally and breast, ovarian, and other cancer risk increases. Abnormal BRCA1 and BRCA2 genes may account for up to 10% of all breast cancers, or 1 out of every 10 cases.

Having an abnormal BRCA1 or BRCA2 gene doesn’t mean you will be diagnosed with breast cancer. Researchers are learning that other mutations in pieces of chromosomes — called SNPs (single nucleotide polymorphisms) — may be linked to higher breast cancer risk in women with an abnormal BRCA1 gene as well as women who didn’t inherit an abnormal breast cancer gene.

Women who are diagnosed with breast cancer and have an abnormal BRCA1 or BRCA2 gene often have a family history of breast cancer, ovarian cancer, and other cancers. Still, most people who develop breast cancer did not inherit an abnormal breast cancer gene and have no family history of the disease.

You are substantially more likely to have an abnormal breast cancer gene if:

If one family member has an abnormal breast cancer gene, it does not mean that all family members will have it.

The average woman in the United States has about a 1 in 8, or about 12%, risk of developing breast cancer in her lifetime. Women who have an abnormal BRCA1 or BRCA2 gene (or both) can have up to an 80% risk of being diagnosed with breast cancer during their lifetimes. Breast cancers associated with an abnormal BRCA1 or BRCA2 gene tend to develop in younger women and occur more often in both breasts than cancers in women without these abnormal genes.

Women with an abnormal BRCA1 or BRCA2 gene also have an increased risk of developing ovarian, colon, and pancreatic cancers, as well as melanoma.

Men who have an abnormal BRCA2 gene have a higher risk of breast cancer than men who don’t — about 8% by the time they’re 80 years old. This is about 80 times greater than average.

Men with an abnormal BRCA1 gene have a slightly higher risk of prostate cancer. Men with an abnormal BRCA2 gene are 7 times more likely than men without the abnormal gene to develop prostate cancer. Other cancer risks, such as cancer of the skin or digestive tract, also may be slightly higher in men with abnormal BRCA1 or BRCA2 genes.

Changes in other genes are also associated with breast cancer. These abnormal genes are much less common and don’t seem to increase risk as much as abnormal BRCA1 and BRCA2 genes, which are considered rare. Still, because these genetic mutations are rarer, they haven’t been studied as much as the BRCA genes.

In 2015, an abnormal version of the SEC23B gene also was linked to Cowden syndrome. The SEC23B gene also helps regulate cell growth. Because this discovery is so new, there is not a clinical test available for an abnormal SEC23B gene.

Inheriting two abnormal copies of the BRCA2, BRIP1, MRE11A, NBN, PALB2, RAD50, or RAD51C genes causes the disease Fanconi anema, which suppresses bone marrow function and leads to extremely low levels of red blood cells, white blood cells, and platelets. People with Fanconi anemia also have a higher risk of several other types of cancer, including kidney cancer and brain cancer.

There are genetic tests available to determine if someone has an abnormal BRCA1 or BRCA2 gene. A genetic counselor also may order testing for an abnormal ATM, CDH1, CHEK2, MRE11A, NBN, PALB2, PTEN, RAD50, RAD51C, or TP53 gene, individually or as part of a larger gene panel that includes BRCA1 and BRCA2 if it’s determined from your personal or family history that these tests are an option. Right now, there is not a clinical test for an abnormal SEC23B gene.

For more information, visit the Breastcancer.org Genetic Testing pages.

If you know you have an abnormal gene linked to breast cancer, there are lifestyle choices you can make to keep your risk as low it can be:

These are just a few steps you can take. Review the links on the left side of this page for more options.

Along with these lifestyle choices, there are other risk-reduction options for women at high risk because of abnormal genetics.

Hormonal therapy medicines: Two SERMs (selective estrogen receptor modulators) and two aromatase inhibitors have been shown to reduce the risk of developing hormone-receptor-positive breast cancer in women at high risk.

Hormonal therapy medicines do not reduce the risk of hormone-receptor-negative breast cancer.

More frequent screening: If you’re at high risk because of an abnormal breast cancer gene, you and your doctor will develop a screening plan tailored to your unique situation. You may start being screened when you’re younger than 40. In addition to the recommended screening guidelines for women at average risk, a screening plan for a woman at high risk may include:

Women with an abnormal breast cancer gene need to be screened twice a year because they have a much higher risk of cancer developing in the time between yearly screenings. For example, the Memorial Sloan-Kettering Cancer Center in New York, NY recommends that women with an abnormal BRCA1 or BRCA2 gene have both a digital mammogram and an MRI scan each year, about 6 months apart (for example, a mammogram in December and an MRI in June).

A breast ultrasound is another powerful tool that can help detect breast cancer in women with an abnormal breast cancer gene. This test does not take the place of digital mammography and MRI scanning.

Talk to your doctor, radiologist, and genetic counselor about developing a specialized program for early detection that addresses your breast cancer risk, meets your individual needs, and gives you peace of mind.

Protective surgery: Removing the healthy breasts and ovaries — called prophylactic surgery (“prophylactic” means “protective”) — are very aggressive, irreversible risk-reduction options that some women with an abnormal BRCA1 or BRCA2 gene choose.

Prophylactic breast surgery may be able to reduce a woman’s risk of developing breast cancer by as much as 97%. The surgery removes nearly all of the breast tissue, so there are very few breast cells left behind that could develop into a cancer.

Women with an abnormal BRCA1 or BRCA2 gene may reduce their risk of breast cancer by about 50% by having prophylactic ovary and fallopian tube removal (salpingo-oophorectomy) before menopause. Removing the ovaries lowers the risk of breast cancer because the ovaries are the main source of estrogen in a premenopausal womans body. Removing the ovaries doesnt reduce the risk of breast cancer in postmenopausal women because fat and muscle tissue are the main producers of estrogen in these women. Prophylactic removal of both ovaries and fallopian tubes reduces the risk of ovarian cancer in women at any age, before or after menopause.

Research also has shown that women with an abnormal BRCA1 or BRCA2 gene who have prophylactic ovary removal have better survival if they eventually are diagnosed with breast or ovarian cancer.

The benefit of prophylactic surgeries is usually counted one year at a time. Thats why the younger you are at the time of surgery, the larger the potential benefit, and the older you are, the lower the benefit. Also, as you get older youre more likely to develop other medical conditions that affect how long you live, such as diabetes and heart disease.

Of course, each woman’s situation is unique. Talk to your doctor about your personal level of risk and how best to manage it.

It’s important to remember that no procedure — not even removing both healthy breasts and ovaries at a young age — totally eliminates the risk of cancer. There is still a small risk that cancer can develop in the areas where the breasts used to be. Close follow-up is necessary, even after prophylactic surgery.

Prophylactic surgery decisions require a great deal of thought, patience, and discussion with your doctors, genetic counselor, and family over time — together with a tremendous amount of courage. Take the time you need to consider these options and make decisions that feel comfortable to you.

For more information, visit the Breastcancer.org Prophylactic Mastectomy and Prophylactic Ovary Removal pages.

Think Pink, Live Green: A Step-by-Step Guide to Reducing Your Risk of Breast Cancer teaches you the biology of breast development and how modern life affects breast cancer risk. Order a free booklet by mail or download the PDF of the booklet to learn 31 risk-reducing steps you can take today.

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Breast Cancer Risk Factors: Genetics

Local Doctor Leads Study Of Gene Therapy Treatment For …

February 18, 2016 5:55 PM By Dr. Maria Simbra

PITTSBURGH (KDKA) When part of the brain is no longer working properly, would it be possible to put something in to boost function?

Neurosurgeon Dr. Mark Richardson is trying to find out.

What were trying to do with this study is to replace an enzyme thats lost as cells degenerate in Parkinsons disease, Dr. Richardson said. The enzyme helps the brain make dopamine.

The brain chemical dopamine is important to keeping movements smooth. The problem in Parkinsons disease is the lack of dopamine because of worn out brain cells, and you end up with shaking, stiffness, and slowness of movement.

People can take medicine for Parkinsons disease, but there can be symptom fluctuations and at higher doses, side effects.

Typically in Parkinsons disease, these symptoms kind of go up and down like this, and they can be masked very well by medication, but unfortunately what tends to happen for all of these patients is progression to more of a roller coaster ride of ups and downs during the day, Dr. Richardson said.

Dr. Richardson is leading part of a study, first funded by the Michael J. Fox Foundation and now by a biotherapy company, to see whether inserting a gene into a specific part of the brain will be the on switch for more dopamine production.

The idea of brain surgery for a chronic disease is very different than continuing to take medication, Dr. Richardson said.

The gene is delivered into the brain through the skull by a thin tube and carried by a virus

The idea of a virus probably sounds very scary to some people. But, this virus cannot reproduce, Dr. Richardson said. It can insert itself into a cell, and it can only do one thing there. It can release the gene to allow this enzyme to be made.

Dr. Richardson and the lead investigator in San Francisco are looking for 20 patients to participate. They will be followed for three years, and their need for medication will be evaluated and compared before and after.

To qualify you have to be between 40 and 70 and on certain medicines for Parkinsons disease for at least three years with increasing fluctuations in movement.

Dr. Richardson hopes gene therapy leads to smoother days and fewer symptoms.

If we can show this is a small group of patients, the trial will be expanded, Dr. Richardson said. With a little bit of luck, within the next decade, we will see a gene therapy accepted as a proven and viable treatment option.

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Dr. Maria Simbra is a multi award-winning medical journalist, who brings a unique set of skills to her position as medical reporter on KDKA-TV. A member of the KDKA news team since May 2002, this physician and formally trained journalism professi…

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Local Doctor Leads Study Of Gene Therapy Treatment For …

Dr Rajiv Desai Blog Archive GENE THERAPY

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GENE THERAPY:

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Caveat:

Medicine is an ever-changing science. As new research and clinical experience broaden our knowledge, changes in treatment and drug therapy are required. I have checked with sources believed to be reliable in their efforts to provide information that is complete and generally in accord with the standards accepted at the time of publishing this article. However, in view of the possibility of human error or changes in medical sciences, I do not assure that the information contained herein is in every respect accurate or complete, and I disclaim all responsibility for any errors or omissions or for the results obtained from use of the information contained in this work. Readers are encouraged to confirm the information contained herein with other sources. I have taken some information from articles that were published few years ago. The facts and conclusions presented may have since changed and may no longer be accurate. Questions about personal health should always be referred to a physician or other health care professional.

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Prologue:

BLASPHEMY! some cried when the concept of gene therapy first surfaced. For them tinkering with the genetic constitution of human beings was equivalent to playing God, and this they perceived as being sacrilegious! On the other side was the scientific community, abuzz with excitement at the prospect of being able to wipe certain genetic disorders in humans entirely from the human gene pool. Although the term gene therapy was first introduced during the 1980s, the controversy about the rationality of this line of treatment still rages on. In the center of the debate lie the gene therapy pros and cons that derive opinions from religious, ethical and undoubtedly, political domains. The concept of genes as carriers of phenotypic information was introduced in the early 19th century by Gregor Mendel, who later demonstrated the properties of genetic inheritance in peas. Over the next 100 years, many significant discoveries lead to the conclusions that genes encode proteins and reside on chromosomes, which are composed of DNA. These findings culminated in the central dogma of molecular biology, that proteins are translated from RNA, which is transcribed from DNA. James Watson was quoted as saying we used to think that our fate was in our stars, but now we know, in large measures, our fate is in our genes. Genes, the functional unit of heredity, are specific sequences bases that encode instructions to make proteins. Although genes get a lot of attentions, it is the proteins that perform most life functions. When genes are altered, encoded proteins are unable to carry out their normal functions, resulting in genetic disorders. Gene therapy is a novel therapeutic branch of modern medicine. Its emergence is a direct consequence of the revolution heralded by the introduction of recombinant DNA methodology in the 1970s. Gene therapy is still highly experimental, but has the potential to become an important treatment regimen. In principle, it allows the transfer of genetic information into patient tissues and organs. Consequently, diseased genes can be eliminated or their normal functions rescued. Furthermore, the procedure allows the addition of new functions to cells, such as the production of immune system mediator proteins that help to combat cancer and other diseases. Most scientists believe the potential for gene therapy is the most exciting application of DNA science, yet undertaken.

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Note:

Please read my other articles Stem cell therapy and human cloning, Cell death and Genetically modified before reading this article.

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The rapid pace of technological advances has profound implications for medical applications far beyond their traditional roles to prevent, treat, and cure disease. Cloning, genetic engineering, gene therapy, human-computer interfaces, nanotechnology, and designer drugs have the potential to modify inherited predispositions to disease, select desired characteristics in embryos, augment normal human performance, replace failing tissues, and substantially prolong life span. As gene therapy is uprising in the field of medicine, scientists believe that after 20 years, this will be the last cure of every genetic disease. Genes may ultimately be used as medicine and given as simple intravenous injection of gene transfer vehicle that will seek our target cells for stable, site-specific chromosomal integration and subsequent gene expression. And now that a draft of the human genome map is complete, research is focusing on the function of each gene and the role of the faulty gene play in disease. Gene therapy will ultimately play Copernican part and will change our lives forever.

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Gene therapy, the experimental therapy as on today:

Gene therapy is an experimental technique that uses genes to treat or prevent diseases. Genes are specific sequences of bases that encode instructions on how to make proteins. When genes are altered so that the encoded proteins are unable to carry out their normal functions, genetic disorders can result. Gene therapy is used for correcting defective genes responsible for disease development. Researchers may use one of several approaches for correcting faulty genes. Although gene therapy is a promising treatment which helps successfully treat and prevent various diseases including inherited disorders, some types of cancer, and certain viral infections, it is still at experimental stage. Gene therapy is presently only being tested for the treatment of diseases that have no other cures. Currently, the only way for you to receive gene therapy is to participate in a clinical trial. Clinical trials are research studies that help doctors determine whether a gene therapy approach is safe for people. They also help doctors understand the effects of gene therapy on the body. Your specific procedure will depend on the disease you have and the type of gene therapy being used.

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Introduction to gene therapy:

Gene therapy is a clinical strategy involving gene transfer with therapeutic purposes. It is based on the concept that an exogenous gene (transgene) is able to modify the biology and phenotype of target cells, tissues and organs. Initially designed to definitely correct monogenic disorders, such as cystic fibrosis, severe combined immunodeficiency or muscular dystrophy, gene therapy has evolved into a promising therapeutic modality for a diverse array of diseases. Targets are expanding and currently include not only genetic, but also many acquired diseases, such as cancer, tissue degeneration or infectious diseases. Depending on the duration planned for the treatment, type and location of target cells, and whether they undergo division or are quiescent, different vectors may be used, involving nonviral methods, non-integrating viral vectors or integrating viral vectors. The first gene therapy clinical trial was carried out in 1989, in patients with advanced melanoma, using tumor-infiltrating lymphocytes modified by retroviral transduction. In the early nineties, a clinical trial with children with severe combined immunodeficiency (SCID) was also performed, by retrovirus transfer of adenosine deaminase gene to lymphocytes isolated from these patients. Since then, more than 5,000 patients have been treated in more than 1,000 clinical protocols all over the world. Despite the initial enthusiasm, however, the efficacy of gene therapy in clinical trials has not been as high as expected; a situation further complicated by ethical and safety concerns. Further studies are being developed to solve these limitations.

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Historical development of gene therapy:

Chronology of development of gene therapy technology:

1970s, 1980s and earlier:

In 1972 Friedmann and Roblin authored a paper in Science titled Gene therapy for human genetic disease? Rogers (1970) was cited for proposing that exogenous good DNA be used to replace the defective DNA in those who suffer from genetic defects. However, these authors concluded that it was premature to begin gene therapy studies in humans because of lack of basic knowledge of genetic regulation and of genetic diseases, and for ethical reasons. They did, however, propose that studies in cell cultures and in animal models aimed at development of gene therapies be undertaken. Such studiesas well as abortive gene therapy studies in humanshad already begun as of 1972. In the 1970s and 1980s, researchers applied such technologies as recombinant DNA and development of viral vectors for transfer of genes to cells and animals to the study and development of gene therapies.

1990s:

The first approved gene therapy case in the United States took place on 14 September 1990, at the National Institute of Health, under the direction of Professor William French Anderson. It was performed on a four year old girl named Ashanti DeSilva. It was a treatment for a genetic defect that left her with ADA-SCID, a severe immune system deficiency. The effects were only temporary, but successful. New gene therapy approach repairs errors in messenger RNA derived from defective genes. This technique has the potential to treat the blood disorder thalassaemia, cystic fibrosis, and some cancers. Researchers at Case Western Reserve University and Copernicus Therapeutics are able to create tiny liposomes 25 nanometers across that can carry therapeutic DNA through pores in the nuclear membrane. Sickle-cell disease is successfully treated in mice. The mice which have essentially the same defect that causes sickle cell disease in humans through the use a viral vector, were made to express the production of fetal hemoglobin (HbF), which normally ceases to be produced by an individual shortly after birth. In humans, the use of hydroxyurea to stimulate the production of HbF has long been shown to temporarily alleviate the symptoms of sickle cell disease. The researchers demonstrated this method of gene therapy to be a more permanent means to increase the production of the therapeutic HbF. In 1992 Doctor Claudio Bordignon working at the Vita-Salute San Raffaele University, Milan, Italy performed the first procedure of gene therapy using hematopoietic stem cells as vectors to deliver genes intended to correct hereditary diseases. In 2002 this work led to the publication of the first successful gene therapy treatment for adenosine deaminase-deficiency (SCID). The success of a multi-center trial for treating children with SCID (severe combined immune deficiency or bubble boy disease) held from 2000 and 2002 was questioned when two of the ten children treated at the trials Paris center developed a leukemia-like condition. Clinical trials were halted temporarily in 2002, but resumed after regulatory review of the protocol in the United States, the United Kingdom, France, Italy, and Germany. In 1993 Andrew Gobea was born with severe combined immunodeficiency (SCID). Genetic screening before birth showed that he had SCID. Blood was removed from Andrews placenta and umbilical cord immediately after birth, containing stem cells. The allele that codes for ADA was obtained and was inserted into a retrovirus. Retroviruses and stem cells were mixed, after which the viruses entered and inserted the gene into the stem cells chromosomes. Stem cells containing the working ADA gene were injected into Andrews blood system via a vein. Injections of the ADA enzyme were also given weekly. For four years T cells (white blood cells), produced by stem cells, made ADA enzymes using the ADA gene. After four years more treatment was needed. The 1999 death of Jesse Gelsinger in a gene therapy clinical trial resulted in a significant setback to gene therapy research in the United States. Jesse Gelsinger had ornithine transcarbamylase deficiency. In a clinical trial at the University of Pennsylvania, he was injected with an adenoviral vector carrying a corrected gene to test the safety of use of this procedure. He suffered a massive immune response triggered by the use of the viral vector, and died four days later. As a result, the U.S. FDA suspended several clinical trials pending the re-evaluation of ethical and procedural practices in the field.

2003:

In 2003 a University of California, Los Angeles research team inserted genes into the brain using liposomes coated in a polymer called polyethylene glycol. The transfer of genes into the brain is a significant achievement because viral vectors are too big to get across the bloodbrain barrier. This method has potential for treating Parkinsons disease. RNA interference or gene silencing may be a new way to treat Huntingtons disease. Short pieces of double-stranded RNA (short, interfering RNAs or siRNAs) are used by cells to degrade RNA of a particular sequence. If a siRNA is designed to match the RNA copied from a faulty gene, then the abnormal protein product of that gene will not be produced.

2006:

In March 2006 an international group of scientists announced the successful use of gene therapy to treat two adult patients for X-linked chronic granulomatous disease, a disease which affects myeloid cells and which gives a defective immune system. The study, published in Nature Medicine, is believed to be the first to show that gene therapy can cure diseases of the myeloid system. In May 2006 a team of scientists led by Dr. Luigi Naldini and Dr. Brian Brown from the San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) in Milan, Italy reported a breakthrough for gene therapy in which they developed a way to prevent the immune system from rejecting a newly delivered gene. Similar to organ transplantation, gene therapy has been plagued by the problem of immune rejection. So far, delivery of the normal gene has been difficult because the immune system recognizes the new gene as foreign and rejects the cells carrying it. To overcome this problem, the HSR-TIGET group utilized a newly uncovered network of genes regulated by molecules known as microRNAs. Dr. Naldinis group reasoned that they could use this natural function of microRNA to selectively turn off the identity of their therapeutic gene in cells of the immune system and prevent the gene from being found and destroyed. The researchers injected mice with the gene containing an immune-cell microRNA target sequence, and the mice did not reject the gene, as previously occurred when vectors without the microRNA target sequence were used. This work will have important implications for the treatment of hemophilia and other genetic diseases by gene therapy. In August 2006, scientists at the National Institutes of Health (Bethesda, Maryland) successfully treated metastatic melanoma in two patients using killer T cells genetically retargeted to attack the cancer cells. This study constitutes one of the first demonstrations that gene therapy can be effective in treating cancer. In November 2006 Preston Nix from the University of Pennsylvania School of Medicine reported on VRX496, a gene-based immunotherapy for the treatment of human immunodeficiency virus (HIV) that uses a lentiviral vector for delivery of an antisense gene against the HIV envelope. In the Phase I trial enrolling five subjects with chronic HIV infection who had failed to respond to at least two antiretroviral regimens, a single intravenous infusion of autologous CD4 T cells genetically modified with VRX496 was safe and well tolerated. All patients had stable or decreased viral load; four of the five patients had stable or increased CD4 T cell counts. In addition, all five patients had stable or increased immune response to HIV antigens and other pathogens. This was the first evaluation of a lentiviral vector administered in U.S. Food and Drug Administration-approved human clinical trials for any disease. Data from an ongoing Phase I/II clinical trial were presented at CROI 2009.

2007:

On 1 May 2007 Moorfields Eye Hospital and University College Londons Institute of Ophthalmology announced the worlds first gene therapy trial for inherited retinal disease. The first operation was carried out on a 23 year-old British male, Robert Johnson, in early 2007. Lebers congenital amaurosis is an inherited blinding disease caused by mutations in the RPE65 gene. The results of a small clinical trial in children were published in New England Journal of Medicine in April 2008. They researched the safety of the subretinal delivery of recombinant adeno-associated virus (AAV) carrying RPE65 gene, and found it yielded positive results, with patients having modest increase in vision, and, perhaps more importantly, no apparent side-effects.

2008:

In May 2008, two more groups, one at the University of Florida and another at the University of Pennsylvania, reported positive results in independent clinical trials using gene therapy to treat Lebers congenital amaurosis. In all three clinical trials, patients recovered functional vision without apparent side-effects. These studies, which used adeno-associated virus, have spawned a number of new studies investigating gene therapy for human retinal disease.

2009:

In September 2009, the journal Nature reported that researchers at the University of Washington and University of Florida were able to give trichromatic vision to squirrel monkeys using gene therapy, a hopeful precursor to a treatment for color blindness in humans. In November 2009, the journal Science reported that researchers succeeded at halting a fatal genetic disorder called adrenoleukodystrophy in two children using a lentivirus vector to deliver a functioning version of ABCD1, the gene that is mutated in the disorder.

2010:

A paper by Komromy et al. published in April 2010, deals with gene therapy for a form of achromatopsia in dogs. Achromatopsia, or complete color blindness, is presented as an ideal model to develop gene therapy directed to cone photoreceptors. Cone function and day vision have been restored for at least 33 months in two young dogs with achromatopsia. However, the therapy was less efficient for older dogs. In September 2010, it was announced that an 18 year old male patient in France with beta-thalassemia major had been successfully treated with gene therapy. Beta-thalassemia major is an inherited blood disease in which beta haemoglobin is missing and patients are dependent on regular lifelong blood transfusions. A team directed by Dr. Phillipe Leboulch (of the University of Paris, Bluebird Bio and Harvard Medical School) used a lentiviral vector to transduce the human -globin gene into purified blood and marrow cells obtained from the patient in June 2007. The patients haemoglobin levels were stable at 9 to 10 g/dL, about a third of the hemoglobin contained the form introduced by the viral vector and blood transfusions had not been needed. Further clinical trials were planned. Bone marrow transplants are the only cure for thalassemia but 75% of patients are unable to find a matching bone marrow donor.

2011:

In 2007 and 2008, a man being treated by Gero Htter was cured of HIV by repeated Hematopoietic stem cell transplantation with double-delta-32 mutation which disables the CCR5 receptor; this cure was not completely accepted by the medical community until 2011. This cure required complete ablation of existing bone marrow which is very debilitating. In August 2011, two of three subjects of a pilot study were confirmed to have been cured from chronic lymphocytic leukemia (CLL). The study carried out by the researchers at the University of Pennsylvania used genetically modified T cells to attack cells that expressed the CD19 protein to fight the disease. In 2013, the researchers announced that 26 of 59 patients had achieved complete remission and the original patient had remained tumor-free. Human HGF plasmid DNA therapy of cardiomyocytes is being examined as a potential treatment for coronary artery disease as well as treatment for the damage that occurs to the heart after myocardial infarction.

2012:

The FDA approves clinical trials of the use of gene therapy on thalassemia major patients in the US. Researchers at Memorial Sloan Kettering Cancer Center in New York begin to recruit 10 participants for the study in July 2012. The study is expected to end in 2014. In July 2012, the European Medicines Agency recommended approval of a gene therapy treatment for the first time in either Europe or the United States. The treatment, called Alipogene tiparvovec (Glybera), compensates for lipoprotein lipase deficiency (LPLD), which can cause severe pancreatitis. People with LPLD cannot break down fat, and must manage their disease with a restricted diet. However, dietary management is difficult, and a high proportion of patients suffer life-threatening pancreatitis. The recommendation was endorsed by the European Commission in November 2012 and commercial rollout is expected in late 2013. In December 2012, it was reported that 10 of 13 patients with multiple myeloma were in remission or very close to it three months after being injected with a treatment involving genetically engineered T cells to target proteins NY-ESO-1 and LAGE-1 which exist only on cancerous myeloma cells.

2013:

In March 2013, Researchers at the Memorial Sloan-Kettering Cancer Center in New York, reported that three of five subjects who had acute lymphocytic leukemia (ALL) had been in remission for five months to two years after being treated with genetically modified T cells which attacked cells with CD19 genes on their surface, i.e. all B-cells, cancerous or not. The researchers believed that the patients immune systems would make normal T-cells and B-cells after a couple of months however they were given bone marrow to make sure. One patient had relapsed and died and one had died of a blood clot unrelated to the disease. Following encouraging Phase 1 trials, in April 2013, researchers in the UK and the US announced they were starting Phase 2 clinical trials (called CUPID2 and SERCA-LVAD) on 250 patients at several hospitals in the US and Europe to use gene therapy to combat heart disease. These trials were designed to increase the levels of SERCA2a protein in the heart muscles and improve the function of these muscles. The FDA granted this a Breakthrough Therapy Designation which would speed up the trial and approval process in the USA. In July 2013 the Italian San Raffaele Telethon Institute for Gene Therapy (HSR-TIGET) reported that six children with two severe hereditary diseases had been treated with a partially deactivated lentivirus to replace a faulty gene and after 732 months the results were promising. Three of the children had metachromatic leukodystrophy which causes children to lose cognitive and motor skills. The other children had Wiskott-Aldrich syndrome which leaves them to open to infection, autoimmune diseases and cancer due to a faulty immune system. In October 2013, the Great Ormond Street Hospital, London reported that two children born with adenosine deaminase severe combined immunodeficiency disease (ADA-SCID) had been treated with genetically engineered stem cells 18 months previously and their immune systems were showing signs of full recovery. Another three children treated since then were also making good progress. ADA-SCID children have no functioning immune system and are sometimes known as bubble children. In October 2013, Amit Nathswani of the Royal Free London NHS Foundation Trust in London reported that they had treated six people with haemophilia in early 2011 using genetically engineered adeno-associated virus. Over two years later all six were still producing blood plasma clotting factor.

2014:

In January 2014, researchers at the University of Oxford reported that six people suffering from choroideremia had been treated with a genetically engineered adeno-associated virus with a copy of a gene REP1. Over a six month to two year period all had improved their sight. Choroideremia is an inherited genetic eye disease for which in the past there has been no treatment and patients eventually go blind. In March 2014 researchers at the University of Pennsylvania reported that 12 patients with HIV had been treated since 2009 in a trial with a genetically engineered virus with a rare mutation known to protect against HIV (CCR5 deficiency). Results were promising.

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The three main issues for the coming decade will be public perceptions, scale-up and manufacturing, and commercial considerations. Focusing on single-gene applications, which tend to be rarer diseases, will produce successful results sooner than the current focus on the commoner, yet more complex, cancer and heart diseases.

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What is Gene?

A gene is an important unit of hereditary information. It provides the code for living organisms traits, characteristics, function, and physical development. Each person has around 25,000 genes that are located on 46 chromosomes. Gene is a segment of DNA found on chromosome that codes for a particular protein. It acts as a blue print for making enzymes and other proteins for every biochemical reaction and structure of body.

What is allele?

Alleles are two or more alternative forms of a gene that can occupy a specific locus (location) on a chromosome.

What is DNA?

Deoxyribonucleic acid (DNA) is a nucleic acid that contains the genetic information used in the development and function of all known living organisms. The main role of DNA is the long-term storage of information. DNA is often compared to a set of blueprints or a recipe or code, since it contains the instructions needed to construct other components of cells, such as proteins. The DNA segments that carry this genetic information are called genes.

What are Chromosomes?

A chromosome is a singular piece of DNA, which contains many genes. Chromosomes also contain DNA-bound proteins, which serve to package the DNA and control its functions. Chromosomes are found inside the nucleus of cells.

What are Proteins?

Proteins are large organic compounds made of amino acids. They are involved in many processes within cells. Proteins act as building blocks, or function as enzymes and are important in communication among cells.

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What are plasmids?

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Plasmid is any extrachromosomal heritable determinant. Plasmids are fragments of double-stranded DNA that can replicate independently of chromosomal DNA, and usually carry genes. Although they can be found in Bacteria, Archaea and Eukaryotes, they play the most significant biological role in bacteria where they can be passed from one bacterium to another by horizontal gene transfer, usually providing a context-dependent selective advantage, such as antibiotic resistance.

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In the center of every cell in your body is a region called the nucleus. The nucleus contains your DNA which is the genetic code you inherited from each of your parents. The DNA is ribbon-like in structure, but normally exists in a condensed form called chromosomes. You have 46 chromosomes (23 from each parent), which are in turn comprised of thousands of genes. These genes encode instructions on how to make proteins. Proteins make up the majority of a cells structure and perform most life functions. Genes tell cells how to work, control our growth and development, and determine what we look like and how our bodies work. They also play a role in the repair of damaged cells and tissues. Each person has more than 25,000 genes, which are made up of DNA. You have 2 copies of every gene, 1 inherited from your mother and 1 from your father.

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DNA or deoxyribonucleic acid is the very long molecule that encodes the genetic information. A gene is a stretch of DNA required to make a functional product such as part or all of a protein. People have about 25,000 genes. During gene therapy, DNA that codes for specific genes is delivered to individual cells in the body.

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The Human Genome:

The human genome is the entire genetic code that resides in every cell in your body (with the exception of red blood cells). The genome is divided into 23 chromosome pairs. During reproduction, two copies of the chromosomes (one from each parent) are passed onto the offspring. While most chromosomes are identical for males and females, the exceptions are the sex chromosomes (known as the X and Y chromosomes). Each chromosome contains thousands of individual genes. These genes can be further divided into sequences called exons and introns, which are in turn made up of even shorter sequences called codons. And finally, the codons are made up of base pairs, combinations of four bases: adenine, cytosine, thymine, and guanine. Or A, C, T, and G for short. The human genome is vast, containing an estimated 3.2 billion base pairs. To put that in perspective, if the genome was a book, it would be hundreds of thousands of pages long. Thats enough room for a dozen copies of the entire Encyclopaedia Britannica, and all of it fits inside a microscopic cell.

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Our genes help make us unique. Inherited from our parents, they go far in determining our physical traits like eye color and the color and texture of our hair. They also determine things like whether babies will be male or female, the amount of oxygen blood can carry, and the likelihood of getting certain diseases. Scientists believe that every human has about 25,000 genes per cell. A mutation, or change, in any one of these genes can result in a disease, physical disability, or shortened life span. These mutations can be passed from one generation to another, inherited just like a mothers curly hair or a fathers brown eyes. Mutations also can occur spontaneously in some cases, without having been passed on by a parent. With gene therapy, the treatment or elimination of inherited diseases or physical conditions due to these mutations could become a reality. Gene therapy involves the manipulation of genes to fight or prevent diseases. Put simply, it introduces a good gene into a person who has a disease caused by a bad gene. Variations on genes are known as alleles. Because of changes in the genetic code caused by mutations, there are often more than one type of gene in the gene pool. For example, there is a specific gene to determine a persons blood type. Therefore, a person with blood type A will have a different version of that gene than a person with blood type B. Some genes work in tandem with each other.

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Genes to protein:

Chromosomes contain long chains of DNA built with repeating subunits known as nucleotides. That means a single gene is a finite stretch of DNA with a specific sequence of nucleotides. Those nucleotides act as a blueprint for a specific protein, which gets assembled in a cell using a multistep process.

1. The first step, known as transcription, begins when a DNA molecule unzips and serves as a template to create a single strand of complementary messenger RNA.

2. The messenger RNA then travels out of the nucleus and into the cytoplasm, where it attaches to a structure called the ribosome.

3. There, the genetic code stored in the messenger RNA, which itself reflects the code in the DNA, determines a precise sequence of amino acids. This step is known as translation, and it results in a long chain of amino acids a protein.

Proteins are the workhorses of cells. They help build the physical infrastructure, but they also control and regulate important metabolic pathways. If a gene malfunctions if, say, its sequence of nucleotides gets scrambled then its corresponding protein wont be made or wont be made correctly. Biologists call this a mutation, and mutations can lead to all sorts of problems, such as cancer and phenylketonuria. Gene therapy tries to restore or replace a defective gene, bringing back a cells ability to make a missing protein.

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Length measurements of DNA/RNA:

The following abbreviations are commonly used to describe the length of a DNA/RNA molecule:

bp = base pair(s) one bp corresponds to approximately 3.4 (340 pm) of length along the strand, or to roughly 618 or 643 daltons for DNA and RNA respectively.

kb (= kbp) = kilo base pairs = 1,000 bp

Mb = mega base pairs = 1,000,000 bp

Gb = giga base pairs = 1,000,000,000 bp.

For case of single-stranded DNA/RNA units of nucleotides are used, abbreviated nt (or knt, Mnt, Gnt), as they are not paired.

Note:

Please do not confuse these terms with computer data units.

kb in molecular biology is kilobase pairs = 1000 base pairs

kb in computer data is kilobytes = 1000 bytes

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Read this article:
Dr Rajiv Desai Blog Archive GENE THERAPY

Gene Therapy | Doctor | Patient

In the 1990s there was great hope that this novel approach may provide an answer to many hitherto incurable diseases. The basic idea is to correct defective genes responsible for disease development. This can be achieved in a number of ways:

When a normal gene is inserted into the genome, a carrier molecule (a vector) is used. This will deliver the new gene to the target cells. The most commonly used vectors are viruses. The most commonly used viruses are:

These viruses are altered to carry normal human DNA. The patient’s target cells are infected with the vector, which deposits its genetic load including the gene to be replaced . The target cell is then able to produce a functioning protein. More recently, success has been seen by combining a tumour-specific adenovirus vector and several single therapy genes. Targeting gene-virotherapy has killed tumour cells with minimal damage to normal cells in mice.[1][2] There are also nonviral insertion options. The simplest method is direct introduction of new DNA into the target tissues. This is limited by the type of tissue and the amount of DNA required. An artificial lipid sphere with an aqueous core is created – a liposome – which can both carry the therapeutic DNA and pass it through the target cells membrane. The therapeutic DNA can also bind chemically to molecules that will attach to target cell receptor sites. These are then taken into the cell’s interior. This tends to be less effective than the other methods.

Human gene therapy is still largely in the experimental phase. There have been few big breakthroughs since the first trial started in 1990. There has also been at least one death attributed to therapy and two cases of leukaemia developing post-therapy. There are also technical problems involved:

In a bid to alleviate disease at the earliest possible stage, in utero fetal gene therapy has also been tried.[6] Prenatal screening for severe genetic disease such as Crigler-Najjar syndrome, Pompe’s disease and haemophilia B has been tested in mouse models. There have been issues with the development of liver tumours, insufficient target cells are reached and the therapy is not toxic enough to target cells. There are attempts underway to manufacture antitumour vaccines.In this technique Epstein-Barr virus vectors mediate gene transfer into human B lymphocytes.[7] Other areas of research include:

A recent trial, approved by the American Food and Drug Administration, is for the treatment of Parkinson’s disease. This is a phase 1 clinical trial with 11 patients already enrolled. They are aiming to produce the neuroprotective and restorative subthalamic glutamic decarboxylase. There have been no adverse events reported to date.[13]

Read more here:
Gene Therapy | Doctor | Patient

Gene Therapy | Doctor | Patient.co.uk

In the 1990s there was great hope that this novel approach may provide an answer to many hitherto incurable diseases. The basic idea is to correct defective genes responsible for disease development. This can be achieved in a number of ways:

When a normal gene is inserted into the genome, a carrier molecule (a vector) is used. This will deliver the new gene to the target cells. The most commonly used vectors are viruses. The most commonly used viruses are:

These viruses are altered to carry normal human DNA. The patient’s target cells are infected with the vector, which deposits its genetic load including the gene to be replaced . The target cell is then able to produce a functioning protein. More recently, success has been seen by combining a tumour-specific adenovirus vector and several single therapy genes. Targeting gene-virotherapy has killed tumour cells with minimal damage to normal cells in mice.[1][2] There are also nonviral insertion options. The simplest method is direct introduction of new DNA into the target tissues. This is limited by the type of tissue and the amount of DNA required. An artificial lipid sphere with an aqueous core is created – a liposome – which can both carry the therapeutic DNA and pass it through the target cells membrane. The therapeutic DNA can also bind chemically to molecules that will attach to target cell receptor sites. These are then taken into the cell’s interior. This tends to be less effective than the other methods.

Human gene therapy is still largely in the experimental phase. There have been few big breakthroughs since the first trial started in 1990. There has also been at least one death attributed to therapy and two cases of leukaemia developing post-therapy. There are also technical problems involved:

In a bid to alleviate disease at the earliest possible stage, in utero fetal gene therapy has also been tried.[6] Prenatal screening for severe genetic disease such as Crigler-Najjar syndrome, Pompe’s disease and haemophilia B has been tested in mouse models. There have been issues with the development of liver tumours, insufficient target cells are reached and the therapy is not toxic enough to target cells. There are attempts underway to manufacture antitumour vaccines.In this technique Epstein-Barr virus vectors mediate gene transfer into human B lymphocytes.[7] Other areas of research include:

A recent trial, approved by the American Food and Drug Administration, is for the treatment of Parkinson’s disease. This is a phase 1 clinical trial with 11 patients already enrolled. They are aiming to produce the neuroprotective and restorative subthalamic glutamic decarboxylase. There have been no adverse events reported to date.[13]

Read more here:
Gene Therapy | Doctor | Patient.co.uk

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