YCLOAK

A first attempt at gene therapy for a disease that leaves babies unable to move, swallow and, eventually, breathe has extended the tots’ lives, and some began to roll over, sit and stand on their own, researchers reported Wednesday.

Only 15 babies with spinal muscular atrophy received the experimental gene therapy, but researchers in Ohio credited the preliminary and promising results to replacing the infants’ defective gene early – in the first few months of life, before the neuromuscular disease destroyed too many key nerve cells.

“They all should have died by now, said Dr. Jerry Mendell of Nationwide Children’s Hospital, who led the work published by The New England Journal of Medicine. Yet, “those babies are still improving.”

Mendell cautioned that much more study is needed to prove the gene therapy works and is safe. Nor is it clear whether the replacement gene’s effects would wane over time.

Spinal muscular atrophy occurs in about 1 in 10,000 births, and those with the most severe form, called SMA Type 1, rarely reach their second birthday. They can be born looking healthy but rapidly decline. One study found just 8 percent of the most severely affected survived to age 20 months without needing permanent mechanical ventilation to breathe.

There is no cure. The first treatment wasn’t approved until last December _ a drug named Spinraza that requires spinal injections every few months.

The experimental gene therapy approach aims for a one-time fix.

What goes wrong

Spinal muscular atrophy is caused when a mutated gene can’t produce a protein crucial for survival of motor neurons, nerve cells in the spinal cord that control muscles.

Some children carry extra copies of a backup gene that produces small amounts of the vital protein, and thus have much milder forms of the disease.

Gene replacement

Scientists loaded a healthy version of the gene into a virus modified so it couldn’t cause illness. Then 15 babies got a one-time intravenous injection. The virus carried the healthy gene into motor neurons, where it got to work producing the protein those nerve cells require to live.

Three babies received a low dose of the gene therapy, as a first-step safety precaution. The remaining 12 got a high dose.

Results

All of the children are alive, Mendell said, about two years and counting after treatment. All beat the odds of needing permanent machine help to breathe by age 20 months.

But only the high-dose recipients saw better motor control, reaching some developmental milestones usually unthinkable for these patients. Eleven could sit unassisted at least briefly; nine could roll over. Eleven are speaking and able to swallow. Two were able to crawl, stand and then walk, Mendell’s team reported.

Those results are “very striking,” said Dr. Basil Darras, who directs Boston Children’s Hospital’s neuromuscular center and wasn’t involved in the new research.

While the treatment needs testing on far more babies, usually “there are no further developmental gains” after diagnosis, Darras explained. “They stagnate for a while and they go downhill very fast and die.”

The only serious side effect attributed to the gene therapy so far involved possible signs of a liver problem that eased with treatment.

Next steps

AveXis Inc., which is developing the gene therapy and helped fund Wednesday’s study, has opened a second small trial at seven hospitals.

Meanwhile, doctors are prescribing SMA patients the new medication Spinraza, which works by increasing that backup gene’s protein production and, according to a separate New England Journal study, had some benefit in about half of patients. The first year of treatment costs about $750,000, an accompanying editorial noted.

With the drug’s availability, some health groups are urging that SMA be added to the list of diseases that all newborns are screened for, so parents can seek early treatment.
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“Like a monster, it destroys everything.”

That’s how one school girl from a Pacific Island nation described a tsunami.

On Dec. 26, 2004, a magnitude-9.1 earthquake in Indonesia set off a massive tsunami which killed more than 230,000 people across four countries and cost an estimated $10 billion in damage.

Nov. 5 is World Tsunami Awareness Day and at the United Nations Wednesday, disaster risk reduction was high on the agenda.

“What I can tell you is that the tsunami wave cannot be stopped,” said Bulgaria’s U.N. Ambassador Georgi Velikov Panayotov, who with his wife was on vacation in Thailand in 2004 and survived the tsunami. “What we can do is build early warning systems and of course, educating the population about the devastating power of the tsunami wave,” he said.

On March 11, 2011, a magnitude-9 earthquake rocked northeastern Japan triggering a fierce tsunami that also damaged the Fukushima Daiichi nuclear plant, south of Sendai.

It was the most powerful quake ever recorded in Japan, killing more than 15,000 people and causing widespread damage and destruction.

“When the big earthquake hit Japan in 2011, people thought that we were prepared for it,” said Japan’s U.N. Ambassador Koro Bessho. “We had embankments, we had drills, however, we had been counting on something the size of which that hits every 100 years and the earthquake was of the size of possibly every 500 years or thousand years,” he said.

These two events sent the countries of the region into overdrive to review and improve disaster preparedness. In 2015 the Sendai Framework for Disaster Risk Reduction was born. It aims to help create a better understanding of disaster risk and enhance preparedness for an effective response.

Individual countries also assessed their preparedness and planning.

“As a consequence of the 2004 tsunami, Indonesia passed a law on disaster management,” said Willem Rampangilei, head of the Disaster Management Agency of Indonesia, which led to the creation of his agency in 2008. “Our responsibilities include mitigation and preparedness, emergency response, as well as post disaster rehabilitation and reconstruction.”

Indonesia is an archipelago made up of thousands of islands which are disaster-prone. Rampangilei noted that 150 million Indonesians live in danger of earthquakes, 60 million from floods, and four million at risk from tsunamis.

He said his government now has contingency plans for every disaster-prone city which identifies its vulnerabilities, outlines the relief response, and builds overall preparedness.

The Maldives are another Indian Ocean chain of more than one thousand small islands, of which about 188 are inhabited.

“Until the Indian Ocean tsunami … we lived in complacency,” said the Maldives U.N. Ambassador Ali Naseer Mohamed. “We all heard the word ‘tsunami’ probably in our sixth grade geography (class), but never probably paid much attention.”

Banda Aceh, Indonesia, where the quake struck, is about 700 kilometers from the Maldives.

“Three hours after the earthquake hit, the tsunami hit the Maldives and put the entire country under water for a few minutes,” Mohamed said. “About 10 percent of the islands were hit.” The death toll was comparatively low 127 fatalities. But the damage was estimated at about 60 percent of the Maldives Gross Domestic Product and the country’s mainstay  its tourism industry took a nosedive.

Early warning systems

In the aftermath of the 2004 tsunami, the region put in place a Tsunami Warning System. It provides alerts through three regional watch centers in India, Indonesia and Australia, and a network of 26 national tsunami information centers.

In 2012, Banda Aceh again experienced an earthquake, but within eight minutes of it, early warnings were emitted across the region and no casualties were reported.

There are also now tsunami early warning systems in place for the Caribbean and the Northeast Atlantic, Mediterranean and connecting seas.

This week, UNESCO is helping coordinate a drill of the early warning system throughout 15 countries in the northeast Atlantic and Mediterranean. The agency hopes to evaluate local tsunami response plans, increase tsunami awareness and improve regional coordination.

Educating youth

At risk countries are also expanding their education programs, specifically targeting children from an early age so they know how to react in case of a tsunami.

Children are taught to shelter in place till the earthquake passes and then go with their classmates to higher ground away from coastal areas to avoid the walls of water the tsunami triggers.

Japan is sharing its expertise, assisting with joint evacuation drills in schools in 18 countries. It is also hosting next week a summit of high school students from 25 mainly island nations next aimed at improving their understanding on tsunami risks and on life-saving measures during such events.

Building back better

Countries which have experienced earthquakes and tsunamis have also learned that better building codes can save lives and prevent damage.

Chile, which is one of South America’s most earthquake and tsunami-prone nations, has seen the difference tougher building codes and other measures can make.

In February 2010, the country experienced an 8.8-magnitude quake and a devastating tsunami that killed some 525 people. Four years later, an 8.4-magnitude earthquake struck. Fifteen people were killed in the quake, but no one in the tsunami that followed.

“Building codes were strengthened,” said Chilean diplomat Jorge Iglesias Mori of the measures taken between 2010 and 2014. He said the country also put more resources into developing their early warning systems and expanding their disaster education programs and drills, and they joined forces with Japan, sharing knowledge and experiences.
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President Donald Trump’s commission on the opioid crisis called Wednesday for more drug courts, more training for doctors and penalties for insurers that dodge covering addiction treatment.

The panel’s final report stopped short, however, of calling for new dollars to address the worst drug crisis in U.S. history. Instead, the commission asked Congress for “sufficient funds” and suggested giving the White House drug czar’s office the ability to review federal spending on the problem.

“If we are to invest in combating this epidemic, we must invest in only those programs that achieve quantifiable goals and metrics,” the report said. The drug czar’s office “must establish a system of tracking and accountability.”

But adding a new layer of oversight was met with skepticism from addiction treatment advocates. The Office of National Drug Control Policy, known as the drug czar’s office, “is not a watchdog agency,” said Andrew Kessler, a behavioral health consultant in Washington, D.C.

Trump launched the commission seven months ago, tapping his friend and former rival New Jersey Gov. Chris Christie to lead the fight. Since then, it has held five meetings and, in July, issued an interim report urging the president to elevate attention by declaring a national emergency.

Last week, Trump did so, talking in a White House speech about his brother’s alcoholism and declaring the crisis a national public health emergency.

“The president did exactly what I asked him to do,” Christie said Wednesday, addressing reports that a different type of emergency declaration, one overseen by the Federal Emergency Management Agency would have been stronger. Christie said he wanted the Department of Health and Human Services to take the lead, not FEMA.

“It’s now incumbent on Congress to step up and put money in the public health emergency fund,” Christie said. Congress hasn’t replenished the fund for years and it contains just $57,000.

More than 64,000 Americans died from drug overdoses last year, most involving a prescription painkiller or an illicit opioid like heroin.

The panel’s report contained 56 new recommendations and called for streamlining funding to states by using block grants, which would give states more flexibility.

What’s missing is more money, said Dr. Mitchell Rosenthal of Phoenix House, a nonprofit addiction treatment provider. “We need significantly more funding to the states on the front lines of this crisis, otherwise they won’t be able to implement the prevention and treatment programs that can save so many lives,” Rosenthal said.

The commission urged White House support for the Prescription Drug Monitoring Act, which would require states with federal grants to share information on narcotics users in a federal data-sharing hub.

The panel recommended training doctors who prescribe opioids and allowing more emergency responders to administer overdose reversal drugs. It called for establishing drug courts in all 93 federal judicial districts to get more treatment to drug offenders rather than send them to prison.

Alternatives to incarceration are needed, said Lindsey Vuolo of the National Center on Addiction and Substance Abuse and author of a recent strategy guide for states.

“It’s not enough to say addiction is a disease. We have to treat it as one,” Vuolo said.

 
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Plenty of words have been written and spoken about climate change. But residents of Kolkata and other Indian cities are being given the opportunity to get to grips with the issue in a new way: via dance and music.

Ekonama: The Beginning in the End is a contemporary dance work that challenges audiences to consider what humans will have to live for if the environment is ravaged beyond sustainability.

The creators of the hour-long performance hope that the combination of dramatic choreography, folk dance, vivid costumes and music will raise awareness and compel viewers to become climate change activists.

“I find art is extremely impactful in cases where you want the audience to have an emotional response,” Paramita Saha, co-director of Sapphire Creations Dance Company in Kolkata, said in an interview with the Thomson Reuters Foundation.

The performance depicts members of a secluded Indian tribal community going about their lives, reliant on their masked gods to protect them. One day a storm comes and the villagers find themselves caught in the throes of extreme weather brought on by climate change.

The dance depicts a future where hurricanes, droughts, floods and pollution have turned the planet’s last survivors into half-naked creatures scrounging and even killing for food, water and shelter.

Their gods, stripped of their regal costumes, turn out to be themselves only human.

“You could read three textbook chapters on the environment and feel bored, but when the same subject is turned out as an hour-long creative dance you get goosebumps, in awe when it ends,” said 22-year-old Anusaya Mitra about the piece, which premiered in Kolkata in 2016.

End of the journey

Mitra was one of a group of university students who were also studying dance at Sapphire Creations, and who developed an early version of the dance in 2015 as part of a fellowship sponsored by Microsoft.

The fellowship offered 18- to 25-year-olds a chance to experiment with art projects on environmental issues affecting Kolkata.

Mitra’s team created a 15-minute dance work entitled Ekoboom, which was performed in 16 universities and schools in and around Kolkata and seen by around 6,000 young people.

Saha, the co-director of Sapphire Creations, said that Ekoboom’s impact on audiences inspired her company to create the longer version, with a more developed narrative and sophisticated lighting and music.

The costumes worn by the dancers are made from textile off-cuts, to emphasize the need for recycling and reducing waste.

“For the general population a journey of a ‘thing’ ends in its being thrown into the bin or on the road. What happens after is none of their concern or is not even something they would think about normally. Such a journey depicted through any artistic medium… can be extremely thought-provoking for them,” Saha said.

Wake-up call

Mahashweta Bhattacharya, 20, who was part of the fellowship team, said the play was a wake-up call, even for many of the cast.

“We know that each of us is responsible in some small way for the decay of our planet. But we keep ignoring that voice and pushing it to the back of our minds. After seeing Ekonama, we couldn’t ignore our consciences any longer,” Bhattacharya said.

Sudarshan Chakravorty, artistic director of the dance company, worked with Turkish, Singaporean and Canadian choreographers and composers to develop the piece.

“Because climate change is already deeply touching everyone on this planet, Ekonama’s music… shows how art, when about a threat shared by all, can transcend cross-country borders, genres and culture,” Chakravorty said.

Following Ekonama’s international premiere at the Seattle International Dance Festival in June, the U.N. Framework Convention on Climate Change newsroom also has publicized the piece, noting the contribution of art and culture in raising awareness about climate change.

“Science, economics and politics will be crucial (to building climate resilience) but so will new thinking and new ways of expressing the challenges and opportunities to both leaders and the public — something arts and culture can do in fresh and fundamental ways,” Nick Nuttall, director of communications for the UNFCCC, said at a seminar earlier this year.

Now the Sapphire Creations troupe is busy practicing for performances scheduled for Mumbai’s Contemporary Dance Season in December, and the Uday Shankar Dance Festival in Jaipur, Rajasthan, in February.

Moved to act

Performers say they have already seen those affected by the performance taking action on environmental issues.

“Ekonama made an environmental activist out of my mother. She now goes up to people at our apartment block and asks them not to throw trash everywhere, especially (one-use) plastic stuff,” said Bhattacharya, who is pursuing a master’s degree in sociology at Delhi’s Jawaharlal Nehru University.

Saha points out that India, a country heavily vulnerable to climate disasters, needs greater public awareness of the need to act — but also policies to put that will into action.

“Performances like ours (will) help to open the climate conversation more in the mainstream,” she predicted.

 
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A recent report by the World Health Organization and the World Bank says climate change and pollution are damaging the health of millions every year. Add to that the increasing impact of water scarcity, and the future is troubling for places like Jordan. VOA’s Kevin Enochs reports.
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The smog-prone northern Chinese region of Beijing-Tianjin-Hebei will set up a joint environmental protection agency in an effort to coordinate the region’s war on pollution, the official China Securities Journal reported on Wednesday.

The new agency, part of wider efforts to improve cross-regional environmental governance, will be in place by the end of the year, the paper said, citing Ministry of Environmental Protection officials.

The region, also known as Jing-Jin-Ji, was home to eight of China’s 10 smoggiest cities in September and is involved in a winter campaign that will slash industrial output and restrict traffic in a bid to meet air quality targets.

Creating unified environmental standards across the region was a key element of a regional economic integration plan launched by President Xi Jinping in 2014.

According to academic studies, around a third of the smog drifting across the capital, Beijing, originates in neighboring Hebei, China’s biggest steel-producing region and also a major producer of cement.

Regulators have already promised to establish a unified system of environmental governance that will create cross-regional emission standards and prevent non-compliant firms in Beijing from shifting operations to neighbouring Hebei.

They have also vowed to implement coordinated emergency response plans during heavy smog outbreaks.

 
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California Governor Jerry Brown is continuing his international fight against climate change with an 11-day trip to Europe starting Saturday that includes stops at the Vatican and a U.N. conference in Germany.

Brown is a chief adversary to Republican President Donald Trump in the battle over U.S. climate policy, promising to help the country reach its emissions reductions targets even as Trump withdraws from an international climate accord. He’s been named the special adviser for states and regions at the U.N. Climate Change Conference in Bonn, Germany.

“While the White House declares war on climate science and retreats from the Paris Agreement, California is doing the opposite and taking action,” Brown said in a statement announcing the trip. “We are joining with our partners from every part of the world to do what needs to be done to prevent irreversible climate change.”

The nonprofit California State Protocol Foundation, which accepts donations from private businesses, pays for Brown’s international travel. Travel for Brown’s staff members will be partially covered by money from the nonprofit Climate Registry and the Climate Action Reserve, a program that deals with carbon offset projects, spokesman Evan Westrup said.

Summit next year

Brown’s November trip follows visits to China and Russia earlier this year to promote international collaboration on climate change. Next year, he plans to host a summit in San Francisco.

He will give a speech Saturday to the Vatican Pontifical Academy of Sciences symposium. During the week, Brown will address European Parliament leaders and the state parliament in Baden-Wurttemberg Germany, meet with representatives from national scientific academies and serve on several panels at the U.N. conference.

Governors Kate Brown of Oregon, Jay Inslee of Washington and Terry McAuliffe of Virginia, all Democrats, will join him on a panel about states’ roles in fighting climate change. California Senate leader Kevin de Leon, also a Democrat, is scheduled to speak Friday at a Vatican workshop on climate.

The trip ends November 14.
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The head of the Environmental Protection Agency said Thursday he intends to replace the outside experts that advise him on science and public health issues with new board members holding more diverse views.

In announcing the changes, EPA Administrator Scott Pruitt suggested many previously appointed to the panels were potentially biased because they had received federal research grants. The 22 boards advise EPA on a wide range of issues, including drinking water standards and pesticide safety.

“Whatever science comes out of EPA shouldn’t be political science,” said Pruitt, a Republican lawyer who previously served as the attorney general of Oklahoma. “From this day forward, EPA advisory committee members will be financially independent from the agency.”

Pruitt has expressed skepticism about the consensus of climate scientists that man-made carbon emissions are the primary cause of global warming. He also overruled experts that had recommended pulling a top-selling pesticide from the market after peer-reviewed studies showed it damaged children’s brains.

Pruitt said he will name new leadership and members to three key EPA advisory boards soon — the Science Advisory Board, Clean Air Scientific Advisory Committee, and the Board of Scientific Counselors.

It was not clear from the EPA’s media release if all current board members serving out their appointed terms were immediately dismissed. EPA’s press office did not respond to messages seeking clarification on Tuesday.

As part of his directive, Pruitt said he will bar appointees who currently in receipt of EPA grants or who is in a position to benefit such grants. He exempted people who work at state, local or tribal agencies, saying he wants to introduce more “geographic diversity” to the panels.

The five-page policy Pruitt issued Tuesday makes no mention of other potential conflicts of interest, such as accepting research funding from corporate interests regulated by EPA.

Tuesday announcement comes after Pruitt in May said he would not reappoint nine of the 18 members of the Board of Scientific Counselors to serve a second three-year term, as had been customary.

Current board chairwoman Deborah Swackhamer said the members were already required to follow rules intended to prevent conflicts of interests.

“It obviously stacks the deck against scientists who do not represent corporate special interests,” said Swackhamer, a retired professor who taught environmental health sciences at the University of Minnesota. “It speaks volumes that people funded by special interests are OK to be advisers, but not those who have received federal grants.”

Senate Environment Committee Chairman John Barrasso, a Wyoming Republican who shares Pruitt’s skepticism of mainstream climate science, cheered the move. He said EPA’s science boards would now better reflect the views of rural states like his own.

But environmentalists worried that Pruitt will now select board members with financial ties to the fossil fuel and chemical industries.

“The Trump EPA’s continued attack on science will likely be one of the most lasting and damaging legacies of this administration,” said Sen. Tom Udall of New Mexico, the ranking Democrat on the appropriations subcommittee that approves EPA’s funding. “Pruitt is purging expert scientists from his science boards — and replacing them with mouthpieces for big polluters.”
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What killed off the Neanderthals? It’s a big debate, and now a study says that no matter what the answer, they were doomed anyway.

Our close evolutionary cousins enjoyed a long run in Europe and Asia, but they disappeared about 40,000 years ago after modern humans showed up from Africa.

The search for an explanation has produced many theories including climate change, epidemics, or inability to compete with the modern humans, who may have had some mental or cultural edge.

The new study isn’t intended to argue against those factors, but just to show that they’re not needed to explain the extinction, says Oren Kolodny of Stanford University.

He and colleague Marcus Feldman present their approach in a paper released Tuesday by the journal Nature Communications.   

They based their conclusion on a computer simulation that represented small bands of Neanderthals and modern humans in Europe and Asia. These local populations were randomly chosen to go extinct, and then be replaced by another randomly chosen population, with no regard for whether it represented the same species.

Neither species was assumed to have any inherent advantage, but there was one crucial difference: Unlike the Neanderthals, the modern humans were supplemented by reinforcements coming in from Africa. It wasn’t a huge wave, but rather “a tiny, tiny trickle of small bands,” Kolodny said.

Still, that was enough to tip the balance against the Neanderthals. They generally went extinct when the simulation was run more than a million times under a variety of assumptions.

If survival was a game of chance, “it was rigged by the fact that there’s recurring migration,” Kolodny said. “The game was doomed to end with the Neanderthals losing.”

Kolodny said the evidence that such migrations actually occurred is suggestive rather than conclusive. Such migrations would not be expected to leave much of an archaeological trace, he said.

Experts in human origins said the paper could help scientists pin down the various factors that led to the Neanderthals’ demise. It fits in with other recent attempts to explain the extinction without assuming behavioral differences between Neanderthals and our ancestors, said Wil Roebroeks of the University of Leiden in the Netherlands. The notion of such differences is largely disproven, he said.

Katerina Harvati of the University of Tuebingen in Germany said while the new work could be useful in solving the extinction mystery, it doesn’t address the question of why modern humans dispersed from Africa into Europe and Asia. It’s important to figure out what was behind that, she said in an email.
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