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Australian not-for-profit group wins unprecedented FDA approval for blindness drug

Sunday, July 22nd, 2018

London: An Australian company has won unprecedented US approval for a new drug to treat the second most common preventable cause of blindness in the world.

In the process, Medicines Development for Global Health, based in Melbourne, became the first not-for-profit company in the world to register a medicine with the US Food and Drug Administration.

Mark Sullivan, managing director of Medicines Development for Global Health.

Photo: Supplied

The World Health Organisation has been calling for better treatments for river blindness for more than a decade, but because the medicine would be mostly used by those in poverty-stricken countries, there has been no financial incentive for drug companies to develop new treatments. The current treatment is 20 years old.

River blindness is caused by parasitic worms and spread by black flies. It affects the skin and eyes and is prevalent in sub-Saharan Africa.

In a double-win for Medicines Development for Global Health, it has also won a highly sought after voucher designed to financially reward companies that develop drugs for neglected diseases.

Managing director Mark Sullivan said while FDA approval for the company’s drug, moxidectin, was a “momentous achievement” for any pharma company, it was “a particularly rare and exciting event” for those trying to treat neglected diseases.

The FDA gave its approval for the treatment, which is swallowed as a tablet, in June after the application was submitted in October 2017.

Mr Sullivan established the Melbourne-based not-for-profit company in 2005 with the sole purpose of filling the gap left by the big pharmaceutical companies by developing medicines that were based on need for treatment rather than the patients’ ability to afford them.

Medicines Development for Global Health has been working for five years on the development of the drug and is now planning to develop moxidectin as a new treatment for scabies, a common problem in Indigenous communities.

Priority review voucher

The company has also won a priority review voucher under a scheme set up in 2007 to create a financial incentive to reward drug makers willing to spend the time and money developing treatments for the some of the world’s most neglected diseases. The scheme creates a market for making new drugs that the private market was not filling itself.

Under the scheme, a company that wins a voucher gets a fast track through the FDA for consideration of its  next new drug,  even if it’s a treatment that would have a commercial return. This gives it a head-start over its rivals.

Crucially, a company can also on-sell the voucher to a bigger company willing to pay anywhere between $US100 million and $300 million for the right to almost halve the approval time.

Because Medicines Development for Global Health is a not-for-profit, its proceeds from drug sales and the voucher will be reinvested in the company to develop new drugs & medications for other neglected diseases.

“Our plan is to sell the voucher and use the funds to support further development of moxidectin for other neglected diseases but also to expand our portfolio into other medicines and vaccines,” Mr Sullivan said.

Professor David Ridely from Duke University authored the scheme and said Medicines Development for Global Health was a textbook example of how he envisaged the program would work.

“I’m delighted that the voucher program is playing a role in treating patients with river blindness, and one day eliminating the disease,” he said.

Mr Sullivan said the development of moxidectin could not have been possible without a $US13 million co-investment from the Global Health Investment Fund, which is the social impact investment fund initially put together by the Bill and Melinda Gates Foundation.

”We believe moxidectin may play a pivotal role in eventually eradicating river blindness, and look forward to working with MDGH and others in making this happen,” Curt LaBelle, managing partner at the investment fund, said.

Color-enhanced Scanning Electron Micrograph (SEM) of Onchocerca volvulus, image of a female worm with microfilaria. O. volvulus is a nematode that causes onchocerciasis, or “river blindness,” mostly in Africa. Long-term corneal inflammation, or keratitis, leads to thickening of the corneal stroma which ultimately leads to blindness.

The Food and Drug Administration (FDA) has approved moxidectin for the treatment of onchocerciasis (river blindness) due to Onchocerca volvulus in patients ≥12 years of age.

Moxidectin, a macrocyclic lactone, is an anthelmintic drug that selectively binds to the parasite’s glutamate-gated chloride ion channels. It is active against O. volvulus microfilariae but it does not kill adult O. volvulus parasites. The tropical disease spreads from person to person via black flies that breed near rivers in South and Central America, sub-Saharan Africa and Yemen

RELATED LINKS
 1 in 2 men and 1 in 3 women will develop cancer in their lifetimes. This saddening reality is made worse when it is acknowledged that modern methods of ‘treating’ the disease are often ineffective and only make the symptoms of the disease far worse. In fact, according to one Berkeley doctor, Dr. Hardin B. Jones, chemotherapy doesn’t work 97% of the time.

In the eye-opening video above, Dr. Hardin B. Jones, a former professor of medical physics and physiology at the University of California, Berkeley, discusses how ‘leading edge’ cancer treatment is a sham.

Studied life expectancy of patients for over 25 years

He has personally studied the life expectancy of patients for more than 25 years and has come to the conclusion that chemotherapy does more harm than good. The bone-chilling realization prompted Dr. Jones to speak out against the billion-dollar cancer industry.

“People who refused chemotherapy treatment live on average 12 and a half years longer than people who are undergoing chemotherapy,” said Dr. Jones of his study, which was published in the New York Academy of Science.

“People who accepted chemotherapy die within three years of diagnosis, a large number dies immediately after a few weeks.” 

According to the physician, the only reason doctors prescribe chemotherapy is because they make money from it. Such an accusation doesn’t seem unreasonable, as cancer

Patients rejecting conventional therapy live 4x longer

“Patients with breast cancer who reject conventional therapy live four times longer than those who follow the system. So this is something that you will not hear in the mass media, which will continue  the myth…”

Despite the fact that the United States spends more on healthcare than any other high-income nation in the world, diseases of affluence continue to increase in prevalence, resulting in a shorter life expectancy.

Perhaps this is because mainstream media and the allopathic healthcare system don’t teach about the importance of preventative medicine. Eating a healthy diet, engaging in exercise, thinking positive thoughts, reducing stress, and enjoying the company of others – or habits that bring joy – are all proven to improve longevity and happiness.

Natural alternatives

In addition, potent all natural medicines, such as cannabis oil, are also strictly regulated and illegal in many areas – despite the fact that CBD-rich oil from the marijuana plant is listed as a remedy on the U.S.’ National Cancer Institute’s website.   www.druglinks.info

As it is, there is no money in a healthy population, which is why fast food joints and pharmaceutical industries thrive in America. Hopefully, Dr. Jones’ efforts will inspire people to seek out alternative options if they or someone they know develops the debilitating disease.

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Henry Sapiecha

Video presentation by Jimmy Lin explains how this simple new blood test that can catch cancer early

Friday, January 19th, 2018

A simple new blood test that can catch cancer early | Jimmy Lin

Jimmy Lin is developing technologies to catch cancer months to years before current methods. He shares a breakthrough technique that looks for small signals of cancer’s presence via a simple blood test, detecting the recurrence of some forms of the disease 100 days earlier than traditional methods. It could be a light at the end of the tunnel in a fight where early detection makes all the difference. Check out more TED talks: http://www.ted.com The TED Talks channel features the best talks and performances from the TED Conference, where the world’s leading movers & shakers give the talk of their lives in 18 minutes (or less). Look for talks on Technology, Entertainment and Design — plus science, business, global problems, the arts and much more.

Henry Sapiecha

Killer cancer we know very little about

Sunday, November 12th, 2017

Kristin Washbourne thought she was doing all the right things to cure what she thought was indigestion. Then she was given a two per cent chance of survival

AT 45, worn out from chasing after two small children and sometimes neglecting to take care of herself, Kristin Washbourne didn’t even think to question her GP when he told her the abdominal pain she had been experiencing was probably indigestion.

She accepted the diagnosis, went to the chemist for some antacids, and promised to take better care of herself.

It wasn’t until loading up on yet more packs of the indigestion tablets, that a very insistent young pharmacist warned her that if she’s been taking the treatment for more than 10 days she had to see her doctor about it.

She’d been popping the over-the-counter pills daily for almost a full year.

“Over that year I had been mentioning the pain to my doctor, but also blowing it off a bit and blaming myself,” she told news.com.au.

“I was a bit overweight, not exercising as much as I should, but I sort of kept downplaying it, but after that I asked him to send me off and get tests for ulcers and bacterins that caused ulcers.”

The tests didn’t eventuate, instead Kristin made another promise to take better care of herself.

But a little while later, after a week of extreme fatigue during the summer holidays – barely being able to move for the pain, and feeling like she could only feel OK if she didn’t eat – Kristin began taking the symptoms a bit more seriously and had her husband rush her to the emergency room.

A lick of fake tan had concealed her yellowing skin, and through her tiredness she didn’t notice her eyes had gone a creamy colour.

This picture was taken a couple of days after Kristin Washbourne was diagnosed with pancreatic cancer and given a two per cent shot at survival. She hadn’t even realised her skin and eyes were turning yellow. Picture: Kristin Washbourne

This extreme jaundice, combined with the stomach pains, the fatigue, and some strange bowel movements, all pointed to one thing: pancreatic cancer.

“It was something that I had never even thought of, and looking back now, even when I got the diagnosis, it makes me realise how much I didn’t know,” she said.

Instead of freezing at the word cancer, Kristin said she felt strangely relieved. She thought: “They cure cancer these days, I guess I’ll be fine.”

But what Kristin didn’t know then, and what most Australians aren’t aware of, is that when it comes to pancreatic cancer, there’s no such thing as an easy fix.

It is one of the most aggressive cancers with one of the lowest survival rates.

Of the nine people diagnosed in Australia every day, only one is likely to survive.

When Kristin was diagnosed, just five years ago, she was give a two per cent chance at survival.

On average, when an Australian is diagnosed with the disease, they’ll only have six months to live.

It was the terrifyingly low instances of survival from pancreatic cancer that make Kristin not only lucky to be alive, but also made her journey so difficult.

Even though she was given such a dark prognosis, her treatment was successful and she never felt as sick as she thought she should.

After a gamut of tests and surgery that left her with a scar from her pubic bone to her sternum that made Kristin look “all zipped up”, she cried for 48 hours, didn’t sleep for days, and stayed in hospital for weeks.

Kristin’s daughter Marla has dyed her hair purple to raise awareness for pancreatic cancer. Picture: Kristin Washbourne

Eight rounds of chemo and radiation left her “knocked out” for months, but every step of the way she was just thinking, it could be a lot worse.

As she went through her recovery, Kristin’s understanding of the seriousness of the disease continued to develop, and she realised how lucky she was.

“The pain (from the surgery) was incredible, the recovery from that was just unbearable, but it eventually was over and I felt pretty much OK,” she said.

“In my recovery, I had a really bad feeling of survivor guilt. I lost 30kg, There were months when I was basically bedridden, completely knocked out by steroids, but every step of the way I knew most other people didn’t make it to that stage.”

When Kristin started to feel better, things began to get even more uncertain.

“The trouble is for me, once I started getting better, it gave me a boost when the oncologist was pretty happy that I was still around so I thought maybe I was here for good,” she said.

“So what I wanted to know was, what happens next? But I couldn’t find any survivors to talk to. No one could tell me what was going to happen, how my life would change, if I would carry on as normal.

“It was a really hard time. I was so desperate to talk to someone to find out what happens next, but the survival rates are even lower than the awareness rates when it comes to this disease.”

Five years on from her diagnosis and fighting fit, Kristin has accepted that she is a survivor, and feels the responsibility to use her experience to help people who have been diagnosed, and those who may be delaying diagnosis like she did for so long.

“The thing about this disease, is there is no reason for it. There’s not like a family history as with breast cancer so you know to get checked, there’s no really obvious or specific symptoms like with bowel cancer or prostate cancer,” she said.

“The only hope now is to know the symptoms, and know if someone’s telling you they’ve got indigestion, if it persists, then go and do something. That’s the only way we’re going to catch it.”

Even when she lost weight and was going through chemo, Kristin’s cancer experience was nothing like what she expected. Picture: Kristin Washbourne

St Vincent’s Hospital pancreatic cancer specialist Dr Lorraine Chantrill says the reason pancreatic cancer doesn’t get the same level of awareness as some others with lower death rates, is the same reason Kristin found it difficult to find answers about her recovery.

“The main reason it doesn’t get that recognition is because people die from it,” she said.

“There are very few people who survive who can go on to campaign for it. The other cancers that have got a lot of visibility are cancers that people survive.”

Dr Chantrill says recognition and awareness around the disease are “getting better”, and it’s rising in line with survival rates.

Having the unenviable badge of one of the worst cancers is getting it noticed as well.

But while recognition is increasing, the symptoms are still vague and hard to pin down.

“It’s a cancer that generally presents in people who are older than 60, it often presents with some vague symptoms like upper abdominal pain, but in people who develop diabetes without being overweight, people who have change in their bowel habits of suddenly lose weight for no reason, we want those people to keep persisting with their doctor and to go and get tests,” she said.

“I think we can start to end the nihilism around pancreas cancer and start actually making a difference and it’s thanks to some really brave people who have participated in research.”

This coming Wednesday is world pancreatic cancer day. The Sydney Opera House will be lit up in purple lights and landmarks across the world will follow.

Kristin’s daughter, Marla, has died her hair purple ahead of the day, and Kristin will wear purple clothing.

Whenever anyone asks them why, they’ll start the conversation Kristin, Dr Chantrill and others affected by pancreatic want Australians to start having.

“If people start talking about it, and people start being aware of it, that’s going to lead to more awareness, more fundraising, more research, and more survivors,” Kristin says.

Leah in hospital after the cancer diagnosis. Picture: 60 Minutes

Mr Debono said the image of Leah in hospital after she had been diagnosed with a brain tumour “haunts me”.

“I never thought I would be thrown into any of this,” he said.

“I was holding onto her till the end.”

He and Leah’s parents are desperately trying to figure out how the medical system could fail the young Aussie, who was told she was cancer-free.

“We watched her take her last breath. You don’t ever want to have to go through something like that, it’s really cruel,” Leah’s father, Lex, told 60 Minutes.

“The GP looked at it, assured her that there was nothing … Some trained professionals may not have done their job properly.”

They are also sharing Leah’s story to spread awareness around melanoma and warn Australians about the risks of this deadly disease.

Henry Sapiecha

Rare flesh-eating cancer shock surprise after visit to dentist

Tuesday, June 20th, 2017

Ceri Jones thought she had an abscess — No- She had a rare flesh-eating cancer

Bravery of Ceri Jones

The Ceri Jones flesh eating disease story

CERI Jones thought it would just be a routine trip to the dentist. www.perfectwhiteteeth.net

The 21-year-old had a lump in her mouth she thought was an abscess, so went to the dentist for a check up. It was only then the pub chef from Wales was horrified to lean her problem was in fact a serious, very rare, form of flesh-eating cancer.

The dentist did X-rays and told her there was nothing there so sent her to hospital for more tests. She then got the devastating diagnosis.

“It was November last year when I was diagnosed with Adenoid Cystic Carcinoma and was referred to Liverpool Women’s Hospital.

“I’d never heard of anything like it, I was so shocked that I actually had it to be honest,” The Mirror reported.

Adenoid Cystic Carcinoma affects the salivary glands of the head and neck. She needed 36 hours on the operating table to remove the tumour. But she also lost her left eye.

But that wasn’t all.

The cancer was at an advanced stage after it had spread so her upper left jaw and upper left facial bones were also replaced with titanium metal and her face needed to be reconstructed.

She also lost her teeth on the left side as she had to have the muscle and skin on her right thigh grafted into her mouth.

Miss Jones told the Daily Post the horrifying detail of the operation.

“I was under sedation for two weeks while they did it and took skin and muscle from my right thigh to replace the left and side palate in my mouth, and they had to connect major arteries to blood vessels in my neck so the palate would keep alive.”

The British health system has paid for her to fly to Florida, in the United States, to undergo specialist radiotherapy for the next few months.

But she has to meet her own costs to cover day-to-day living and other expenses, so her family have launched a GoFundMe page has been set up to help with hopes to raise almost $10,000.

Her mum Sarah Evans said: “I relive this nightmare every day from the day we took Ceri to Liverpool to the day she came home and the morning she went down to theatre for the longest life-changing surgery and the complications she had thereafter.”

She said she was proud of the “bravery and strength” her daughter had shown.

“She’s an inspiration.”

Henry Sapiecha

Video below on Flesh Eating Cancer

Precision Medicine: What Is Cancer, Really? Scientists overview here.

Monday, May 22nd, 2017

The men and women who are trying to bring down cancer are starting to join forces rather than work alone. Together, they are winning a few of the battles against the world’s fiercest disease. For this unprecedented special report, we visited elite cancer research centers around the country to find out where we are in the war.

I. Precision Medicine: What Is Cancer, Really?

When you visit St. Jude Children’s Research Hospital in Memphis, Tennessee, you expect to feel devastated. It starts in the waiting room. Oh, here we go with the little red wagons, you think, observing the cattle herd of them rounded up by the entrance to the Patient Care Center. Oh, here we go with the crayon drawings of needles. The itch begins at the back of your throat, and you start blinking very fast and mentally researching how much money you could donate without starving. Near a row of arcade games, a preteen curls his face into his mother’s shoulder while she strokes his head. Oh, here we go.

But the more time you spend at St. Jude, the more that feeling is replaced with wonder. In a cruel world you’ve found a free hospital for children, started by a Hollywood entertainer as a shrine to the patron saint of lost causes. There is no other place like this. Corporations that have nothing to do with cancer—nothing to do with medicine, even—have donated vast sums of money just to be a part of it. There’s a Chili’s Care Center. The cafeteria is named for Kay Jewelers.

Scott Newman’s office is in the Brooks Brothers Computational Biology Center, where a team of researchers is applying computer science and mathematics to the question of why cancer happens to children. Like many computer people, Newman is very smart and a little quiet and doesn’t always exactly meet your eyes when he speaks to you. He works on St. Jude’s Genomes for Kids project, which invites newly diagnosed patients to have both their healthy and tumor cells genetically sequenced so researchers can poke around.

“Have you seen a circle plot before?” Newman asks, pulling out a diagram of the genes in a child’s cancer. “If I got a tattoo, it would be one of these.” Around the outside of the circle plot is something that looks like a colorful bar code. Inside, a series of city skylines. Through the center are colored arcs like those nail-and-string art projects students make in high school geometry class. The diagram represents everything that has gone wrong within a child’s cells to cause cancer. It’s beautiful.

A Genetic Disaster: This circular visualization shows real gene mutations found in 3,000 pediatric cancers at St. Jude Children’s Research Hospital. Genes with sequence mutations are labeled in blue; those with structural variations are in red; and those

“These are the genes in this particular tumor that have been hit,” Newman says in a Yorkshire accent that emphasizes the t at the end of the word hit in a quietly violent way. “And that’s just one type of thing that’s going on. Chromosomes get gained or lost in cancer. This one has gained that one, that one, that one, that one,” he taps the page over and over. “And then there are structural rearrangements where little bits of genome get switched around.” He points to the arcs sweeping across the page. “There are no clearly defined rules.”

It’s not like you don’t have cancer and then one day you just do. Cancer—or, really, cancers, because cancer is not a single disease—happens when glitches in genes cause cells to grow out of control until they overtake the body, like a kudzu plant. Genes develop glitches all the time: There are roughly twenty thousand genes in the human body, any of which can get misspelled or chopped up. Bits can be inserted or deleted. Whole copies of genes can appear and disappear, or combine to form mutants. The circle plot Newman has shown me is not even the worst the body can do. He whips out another one, a snarl of lines and blocks and colors. This one would not make a good tattoo.

“As a tumor becomes cancerous and grows, it can accumulate many thousands of genetic mutations. When we do whole genome sequencing, we see all of them,” Newman says. To whittle down the complexity, he applies algorithms that pop out gene mutations most likely to be cancer-related, based on a database of all the mutations researchers have already found. Then, a genome analyst manually determines whether each specific change the algorithm found seems likely to cause problems. Finally, the department brings its list of potentially important changes to a committee of St. Jude’s top scientists to discuss and assign a triage score. The mutations that seem most likely to be important get investigated first.

It took thirteen years and cost $2.7 billion to sequence the first genome, which was completed in 2003. Today, it costs $1,000 and takes less than a week. Over the last two decades, as researchers like Newman have uncovered more and more of the individual genetic malfunctions that cause cancer, teams of researchers have begun to tinker with those mutations, trying to reverse the chaos they cause. (The first big success in precision medicine was Gleevec, a drug that treats leukemias that are positive for a common structural rearrangement called the Philadelphia chromosome. Its launch in 2001 was revolutionary.) Today, there are eleven genes that can be targeted with hyperspecific cancer therapies, and at least thirty more being studied. At Memorial Sloan Kettering Cancer Center in New York City, 30 to 40 percent of incoming patients now qualify for precision medicine studies.

Charles Mullighan,a tall, serious Australian who also works at St. Jude, is perhaps the ideal person to illustrate how difficult it will be to cure cancer using precision medicine. After patients’ cancer cells are sequenced, and the wonky mutations identified, Mullighan’s lab replicates those mutations in mice, then calls St. Jude’s chemical library to track down molecules—some of them approved medicines from all over the world, others compounds that can illuminate the biology of tumors—to see if any might help.

New York: Britta Weigelt and Jorge Reis-Filho use police forensics techniques to repair old tumor samples at Memorial Sloan Kettering so the samples can be genetically profiled.

If Mullighan is lucky, one of the compounds he finds will benefit the mice, and he’ll have the opportunity to test it in humans. Then he’ll hope there are no unexpected side effects, and that the cancer won’t develop resistance, which it often does when you futz with genetics. There are about twenty subtypes of the leukemia Mullighan studies, and that leukemia is one of a hundred different subtypes of cancer. This is the kind of precision required in precision cancer treatment—even if Mullighan succeeds in identifying a treatment that works as well as Gleevec, with the help of an entire, well-funded hospital, it still will work for only a tiny proportion of patients.

Cancer is not an ordinary disease. Cancer is the disease—a phenomenon that contains the whole of genetics and biology and human life in a single cell. It will take an army of researchers to defeat it.

Luckily, we’ve got one.

Interlude

“I used to do this job out in L.A.,” says the attendant at the Hertz counter at Houston’s George Bush Intercontinental Airport. “There, everyone is going on vacation. They’re going to the beaach or Disneyland or Hollywood or wherever.

“Because of MD Anderson, I see more cancer patients here. They’re so skinny. When they come through this counter, they’re leaning on someone’s arm. They can’t drive themselves. You think, there is no way this person will survive. And then they’re back in three weeks, and in six months, and a year. I’m sure I miss some, who don’t come through anymore because they’ve died. But the rest? They come back.”

II. Checkpoint Inhibitor Therapy: You Have the Power Within You!

On a bookshelf in Jim Allison’s office at MD Anderson Cancer Center in Houston (and on the floor surrounding it) are so many awards that some still sit in the boxes they came in. The Lasker-DeBakey Clinical Medical Research Award looks like the Winged Victory statue in the Louvre. The Breakthrough Prize in Life Sciences, whose benefactors include Sergey Brin, Anne Wojcicki, and Mark Zuckerberg, came with $3 million.

“I gotta tidy that up sometime,” Allison says.

Allison has just returned to the office from back surgery that fused his L3, L4, and L5 vertebrae, which has slightly diminished his Texas rambunctiousness. Even on painkillers, though, he can explain the work that many of his contemporaries believe will earn him the Nobel Prize: He figured out how to turn the immune system against tumors.

“One day, the miracles won’t be miracles at all. They’ll just be what happens.”

Allison is a basic scientist. He has a Ph.D., rather than an M.D., and works primarily with cells and molecules rather than patients. When T-cells, the most powerful “killer cells” in the immune system, became better understood in the late 1960s, Allison became fascinated with them. He wanted to know how it was possible that a cell roaming around your body knew to kill infected cells but not healthy ones. In the mid-1990s, both Allison’s lab and the lab of Jeffrey Bluestone at the University of Chicago noticed that a molecule called CTLA-4 acted as a brake on T-cells, preventing them from wildly attacking the body’s own cells, as they do in autoimmune diseases.

Allison’s mother died of lymphoma when he was a child and he has since lost two uncles and a brother to the disease. “Every time I found something new about how the immune system works, I would think, I wonder how this works on cancer?” he says. When the scientific world discovered that CTLA-4 was a brake, Allison alone wondered if it might be important in cancer treatment. He launched an experiment to see if blocking CTLA-4 would allow the immune system to attack cancer tumors in mice. Not only did the mice’s tumors disappear, the mice were thereafter immune to cancer of the same type.

Ipilimumab (“ipi” for short) was the name a small drug company called Medarex gave the compound it created to shut off CTLA-4 in humans. Early trials of the drug, designed just to show whether ipi was safe, succeeded so wildly that Bristol Myers Squibb bought Medarex for $2.4 billion. Ipilimumab (now marketed as Yervoy) became the first “checkpoint inhibitor”: It blocks one of the brakes, or checkpoints, the immune system has in place to prevent it from attacking healthy cells. Without the brakes the immune system can suddenly, incredibly, recognize cancer as the enemy.

“You see the picture of that woman over there?” Allison points over at his desk. Past his lumbar-support chair, the desk is covered in papers and awards and knickknacks and frames, including one containing a black card with the words “Never never never give up” printed on it. Finally, the photo reveals itself, on a little piece of blue card stock.

That’s the first patient I met,” Allison says. “She was about twenty-four years old. She had metastatic melanoma. It was in her brain, her lungs, her liver. She had failed everything. She had just graduated from college, just gotten married. They gave her a month.”

The woman, Sharon Belvin, enrolled in a phase-two trial of ipilimumab at Memorial Sloan Kettering, where Allison worked at the time. Today, Belvin is thirty-five, cancer- free, and the mother of two children. When Allison won the Lasker prize, in 2015, the committee flew Belvin to New York City with her husband and her parents to see him receive it. “She picked me up and started squeezing me,” Allison says. “I walked back to my lab and thought, Wow, I cure mice of tumors and all they do is bite me.” He adds, dryly, “Of course, we gave them the tumors in the first place.”

After ipi, Allison could have taken a break and waited for his Nobel, driving his Porsche Boxster with the license plate CTLA-4 around Houston and playing the occasional harmonica gig. (Allison, who grew up in rural Texas, has played since he was a teenager and once performed “Blue Eyes Crying in the Rain” onstage with Willie Nelson.) Instead, his focus has become one of two serious problems with immunotherapy: It only works for some people.

So far, the beneficiaries of immune checkpoint therapy appear to be those with cancer that develops after repeated genetic mutations—metastatic melanoma, non-small-cell lung cancer, and bladder cancer, for example. These are cancers that often result from bad habits like smoking and sun exposure. But even within these types of cancer, immune checkpoint therapies improve long-term survival in only about 20 to 25 percent of patients. In the rest the treatment fails, and researchers have no idea why.

Lately, Allison considers immune checkpoint therapy a “platform”—a menu of treatments that can be amended and combined to increase the percentage of people for whom it works. A newer drug called Keytruda that acts on a different immune checkpoint, PD-1, knocked former president Jimmy Carter’s metastatic melanoma into remission in 2015. Recent trials that blocked both PD-1 and CTLA-4 in combination improved long-term survival in 60 percent of melanoma patients. Now, doctors are combining checkpoint therapies with precision cancer drugs, or with radiation, or with chemotherapy. Allison refers to this as “one from column A, and one from column B.”

The thing about checkpoint inhibitor therapy that is so exciting—despite the circumscribed group of patients for whom it works, and despite sometimes mortal side effects from the immune system going buck-wild once the brakes come off—is the length of time it can potentially give people. Before therapies that exploited the immune system, response rates were measured in a few extra months of life. Checkpoint inhibitor therapy helps extremely sick people live for years. So what if it doesn’t work for everyone? Every cancer patient you can add to the success pile is essentially cured.

Jennifer Wargo and team remove lymph nodes from a melanoma patient.

Italian neuroscientist intends bringing frozen brains back to life

Friday, April 28th, 2017

London: A neuroscientist claims he will be able to “wake up” people who have been cryogenically frozen within three years, by transferring their brains to donor bodies.

Sergio Canavero, director of the Turin Advanced Neuromodulation Group, has already announced plans to carry out the first human head transplant, an operation which he claims is just 10 months away.

But he is now thinking further ahead, and wants to begin brain transplants within three years.

If the procedures are successful, he believes that frozen brains could be thawed and inserted into a donor, effectively bringing “dead” people back to life.

Hundreds of people who were dying or paralysed have had their bodies or brains cryogenically preserved in the hope that medical science will one day be able to cure their conditions.

Although many experts are sceptical that the brain can be thawed without damage, Professor Canavero said he planned to awaken patients frozen by the Alcor Life Extension Foundation, which is based in Arizona.

“As soon as the first human head transplant has taken place, no later than 2018, we will be able to attempt to reawaken the first frozen head,” he said.

“We are currently planning the world’s first brain transplant, and I consider it realistic that we will be ready in three years at the latest.”

British scientists are sceptical about whether the brain could be fully restored from frozen.

Clive Coen, professor of neuroscience at King’s College London, said the chances of bringing a brain back was “infinitesimal”.

Dr Channa Jayasena, clinical senior lecturer at Imperial College London added: “It is currently not possible to freeze and thaw human tissue without killing many cells contained within it.”

Professor Canavero is working with a Chinese team of doctors led by Dr Ren Xiaoping, of Harbin Medical Centre, who helped perform the first successful hand transplant in the US.

Although Russian computer scientist Valery Spiridonov, who has spinal muscular atrophy, had volunteered to become the first head transplant patient, the team expects the first operation to be with a Chinese donor and patient.

Last year, the team announced a successful head transplant performed on a monkey.

Telegraph, London

Just Hours after this photo was taken, she tragically died

Sunday, March 19th, 2017

Gabrielle Marsh died hours after this photo was taken. She was celebrating her upcoming 20th birthday at home with friends when she suffered a catastrophic brain bleed image www.newcures.info

Gabrielle Marsh died hours after this photo was taken. She was celebrating her upcoming 20th birthday at home with friends when she suffered a catastrophic brain bleed

IT WAS supposed to be a fun night with her friends celebrating her 20th birthday – and when Gabrielle Marsh started to get a headache, no one suspected she would be dead hours later.

Photos of the night show the young Auckland woman raising a toast with her best friends, showing off the platter of food she’d thoughtfully planned and created for the night.

Two hours after those photos were taken Gabby, as she was known, was lying on the floor of her home in agony, her mother Kathryn at her side and an ambulance on its way.

Later that night as Gabby lay hooked up to life support machines Auckland City Hospital staff delivered the heartbreaking news to her family – she had suffered a brain haemorrhage and was unlikely to survive.

The next day a decision was made. Gabby was to be taken off life support – but not until her organs had been donated.

And on Monday March 6, on her 20th birthday, after her family had said their goodbyes, Gabby was taken to surgery.

“The woman at the hospital called me and said it was all done, and the donation was taking place as we speak,” Kathryn Marsh told the NZ Herald.

“Gabby loved doing things for other people, and that was her biggest, most amazing gift.”

Gabby’s organs saved the lives of at least six people; her kidney, pancreas, lungs, liver and heart valves were all successfully donated.

“Of course, more than anything, we would love to have her here, but that’s not to be,” said Kathryn.

“But if anything good can come out of it, if she has helped people, then that’s comforting.”

Gabby was the eldest of three children and is survived by Jacob, 18 and 16-year-old Victoria.

Her death was the second tragedy for her family, her father Shayne died just 17 months ago after a long illness.

“It’s still not really sunken in, it was so sudden,” Kathryn said.

“Shayne was sick for 14 months and we all had time to get used to the idea, but with Gabby it was the complete opposite. It’s left us all a bit shell-shocked.”

Gabby was born and raised in Auckland, attending Mount Albert Grammar School before enrolling at Auckland University.

She was about to start her third year of a double degree in commerce and law when she died.

“She was a really good sister, she was kind, generous and she was like a second mum to me,” Jacob said.

Her family described her as extremely thoughtful and loving, adventurous, caring, a “rock star academic” and a young woman motivated and driven with a lot of energy.

“She had a killer smile that came easy and often,” her aunt Michelle Cliffe said.

Kathryn said she didn’t know where to begin when asked what was special about her eldest child.

“She just made people feel at ease and she was easy to be around. There was something special about Gabby,” she said.

After Shayne died, Gabby was a “phenomenal help” to Kathryn, stepping up to do her share of cooking, cleaning and helping with her siblings.

“She just got stuff done, she was pragmatic, hard working and so organised,” Michelle said.

The day Gabby died she woke early and went for a walk with Kathryn – something they did most days together.

Then the pair went to Newmarket shopping and Gabby helped her mother choose a new swimsuit for an extended family holiday to Fiji in April.

The family ate lunch together and Gabby went to watch her boyfriend Bradley play softball before returning home to prepare for her party.

She didn’t drink alcohol, but prepared pina colada cocktails for her three best friends, making a rum-free version for herself.

The girls had planned to go out in the city that night; Gabby loved old music so wanted to go dancing at Irish bar Danny Doolans.

Bradley was going to pick them up and drive them to town.

Then, Gabby started to complain about having a headache.

“It was getting worse and worse,” Kathryn said.

“She just wanted to lie down. Her friends left, they told her it was okay, that they would celebrate with her another time and they called Bradley to tell him.”

After the girls left, Gabby started throwing up and became agitated and slurring her words.

Kathryn suspected a severe migraine, and called an ambulance.

As the paramedics arrived – and Bradley – Gabby lost consciousness.

She never woke up.

Doctors have told her family they believe she had a arteriovenous malformation (AVM), a tangle of abnormal blood vessels connecting arteries and veins in the brain.

It is likely she was born with the condition and there was nothing her family could have done to detect or prevent her death.

“She was healthy, she exercised, she didn’t drink,” said Kathryn, shaking her head.

“The specialist said it was like a ticking time bomb,” Jacob added.

The family said the decision to donate Gabby’s organs was easy; they knew it was what she wanted as she specified it on her licence, and she was a generous young woman.

“She had such a bright future in front of her and I would have just loved to see her future unfold,” Kathryn said.

“We said goodbye to her and we knew that she was then going off to theatre – that she was the one giving the gifts on her birthday.

“She’s given life to more than six people on her birthday, that is her legacy.”

Jacob was brimming with pride over his sister’s final gift.

“It’s like she is living on in other people,” he said.

The Marsh family urged people to openly discuss organ donation with loved ones and make their wishes known.

They hoped to one day meet some of the people that Gabby’s organs helped.

The Gabby Marsh Scholarship

Gabby’s university friends have started a Givealittle page to fund a scholarship in her name, with the support of her family.

“Gabby was passionate, fun loving and kind. She smiled easily and often. She was selfless, considerate and generous.

She was someone who impacted everyone she met,” her friends said.

“Gabby changed the lives of so many around her, and we dream for her character and kindness to continue changing the life of others.

“To honour her academic ability, her exceptional character and her future cut tragically short, the Gabby Marsh Scholarship will be established and offered annually to enable a young school leaver demonstrating exceptional character and service to fulfil their dream of studying commerce at the University of Auckland.”

More than $20,000 has been donated so far.

To donate or read more, click here.

Thanks to the generosity of 503 deceased organ donors and their families a record 1,447 Australians were given a second chance at life in 2016. There were an additional 267 living donors, including 44 under the Australian Kidney Exchange Program.

To register on the Australian Organ Donor Register, click here.

www.goodgirlsgo.com

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Henry Sapiecha

Nobel Prize for Medicine Goes to Discovery of Cells’ Garbage Disposal System Article-1 of 2

Monday, October 3rd, 2016

Japanese biologist Yoshinori Ohsumi won the Nobel Prize in medicine image www.newcures.info

STOCKHOLM (AP) — Japanese biologist Yoshinori Ohsumi won the Nobel Prize in medicine on Monday for discoveries on how cells break down and recycle content, a garbage disposal system that scientists hope to harness in the fight against cancer, Alzheimer’s and other diseases.

The Karolinska Institute honored Ohsumi for “brilliant experiments” in the 1990s on autophagy, a phenomenon that literally means “self-eating” and describes how cells gobble up damaged content and provide building blocks for renewal.

Disrupted autophagy (aw-TAH’-fuh-jee) has been linked to several diseases including Parkinson’s, diabetes and cancer, the prize committee said.

“Intense research is now ongoing to develop drugs that can target autophagy in various diseases,” it said in itscitation .

Ohsumi, 71, from Fukuoka, Japan, is a professor at the Tokyo Institute of Technology. In 2012, he won the Kyoto Prize, Japan’s highest private award for global achievement.

Ohsumi said he never thought he would win a Nobel Prize for his work, which he said involved studying yeast in a microscope day after day for decades.

“As a boy, the Nobel Prize was a dream, but after starting my research, it was out of my picture,” he told reporters in Tokyo.

“I don’t feel comfortable competing with many people, and instead I find it more enjoyable doing something nobody else is doing,” Ohsumi added. “In a way, that’s what science is all about, and the joy of finding something inspires me.”

Nobel committee secretary Thomas Perlmann said Ohsumi seemed surprised when he was informed he had won theNobel Prize.

“The first thing he said was ‘ahhh.’ He was very, very pleased,” Perlmann said.

Nobel judges often award discoveries made decades ago, to make sure they have stood the test of time.

Though scientists have known that autophagy exists for more than 50 years, its fundamental significance was only recognized after Ohsumi’s “paradigm-shifting research” on yeast in the 1990s, the committee said.

“Thanks to Ohsumi and others following in his footsteps, we now know that autophagy controls important physiological functions where cellular components need to be degraded and recycled,” it said.

The term autophagy was coined in 1963 by Belgian scientist Christian de Duve, who shared the 1974 Nobel Prize in medicine for discoveries on cell structure and organization.

But before Ohsumi’s research, scientists “didn’t know what it did, they didn’t know how it was controlled and they didn’t know what it was relevant for,” said David Rubinsztein, deputy director of the Institute for Medical Research at the University of Cambridge.

Now “we know that autophagy is important for a host of important mammalian functions.” For example, it protects against starvation in the period when a newborn animal hasn’t yet started breastfeeding, by providing energy, he said.

It also removes proteins that clump together abnormally in brain cells, which is important in conditions like Huntington’s and Parkinson’s diseases and some forms of dementia. If autophagy didn’t do that job, “the diseases would appear more early and be more aggressive,” he said.

Animal studies suggest that boosting autophagy can ease and delay such diseases, said Rubinsztein, whose lab is pursuing that approach for therapy.

“As time goes on, people are finding connections with more and more diseases” and normal cellular operations, he said.

In 1993 Ohsumi published his “seminal discovery” of 15 genes crucial to autophagy, and cloned several of those genes in yeast and mammalian cells in subsequent studies, the Nobel committee said.

“He actually unraveled which are the components which actually perform this whole process,” said Rune Toftgard, chairman of the Nobel Assembly. “Having those components at hand were also important tools to … do functional experiments to understand how important it was for different types of processes in the body.”

In Tokyo, Ohsumi said many details of autophagy are yet to be understood and that he hoped younger scientists would join him in looking for the answers.

“There is no finish line for science. When I find an answer to one question, another question comes up. I have never thought I have solved all the questions,” he said. “So I have to keep asking questions to yeast.”

It was the 107th award in the medicine category since the first Nobel Prizes were handed out in 1905.

Last year’s prize was shared by three scientists who developed treatments for malaria and other tropical diseases.

The announcements continue with physics on Tuesday, chemistry on Wednesday and the Nobel Peace Prize on Friday. The economics and literature awards will be announced next week.

Each prize is worth 8 million kronor ($930,000). The awards will be handed out at prize ceremonies in Stockholm and Oslo on Dec. 10, the anniversary of prize founder Alfred Nobel’s death in 1896.

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Henry Sapiecha

Japanese Scientist Wins Nobel Prize in showing How Human Cells Cannibalize Worn Out Parts

Monday, October 3rd, 2016

yoshinori_osumi_nobel-prize-recipient image www.newcures.info

Even the best-man made machines eventually break down. And the human body, made up of millions of tiny machine-like cells, is no different. Over the years, cells gradually wear from the grueling work of keeping you alive. To restore themselves, they devour their own broken parts. This morning, cell biologist Yoshinori Ohsumi was awarded the Nobel Prize in Physiology or Medicine for identifying the genes and underlying mechanisms that keep our cells in tip-top shape.

The cellular process known as “autophagy” (Greek for “self-eating”) has been known since the 1960s. As far as biological processes go, it’s one of the most important ones. Without being able to tear apart old, broken-down cells for parts, we would age much faster and be more vulnerable to diseases like cancer caused by error-riddled cells running amok.

In the 1950s, scientists discovered that cells of plants and animals are packed with tiny structures called organelles, which are responsible for cellular functions such as generating energy. Researchers noticed, however, that one of these organelles also contained bits and pieces of proteins and structures from the cell itself, “like a garbage dump,” write Gina Kolata and Sewell Chan for the New York Times. This trash pile, dubbed the “lysosome,” cannibalizes worn out parts of the cell for the raw materials to build anew, according to the Nobel Assembly at Stockholm’s Karolinska Institutet.

Before Ohsumi’s work, however, cellular biologists didn’t have a firm understanding of the inner workings of this process. Scientists knew that cells built little sacs around worn-out proteins and organelles for transport to the lysosome. But beyond this basic process, the cellular recycling remained a mystery, Ariana Eunjung Cha and Anna Fifield report for The Washington Post. By studying the inner workings of small, simple yeast cells, Ohsumi was able to identify the genes that make autophagy possible, how cells determine which parts need replacing and what happens when things go wrong.

“Looking into bodily processes, I found that we have an ongoing renewal process without which living organisms can’t survive,” Ohsumi tells the Japanese broadcaster NHK. “This recycling process did not receive as much attention as it deserved, but I discovered that we should be paying more attention to this autophagy process.”

Ohsumi’s discoveries shed new light on some of the most important processes our cells use to stay healthy. By understanding how autophagy works, scientists hope to better understand the role it plays in aging and disease. Yet despite his accomplishments, Ohsumi remains humble, calling himself “just a basic researcher in yeast,” in an interview with the Canadian newspaper TThe Globe and Mail last year after he received the Canada Gairdner International Award. Perhaps—but some yeast researchers clearly rise to the top more than others.

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Henry Sapiecha