Spain’s Balearic Islands vote to outlaw killing bulls at fights – Reuters

Posted on by

MADRID (Reuters) - Spain's Balearic Islands on Monday banned matadors from killing bulls in the ring, becoming one of several Spanish regions to move toward criminalizing the centuries-old blood sport.

The bill adopted in the Balearics parliament prohibits a bull's death in the ring and makes it illegal for the animals to endure physical or psychological damage. Bullfights will be limited to a maximum of 10 minutes, a parliament spokesman said.

Traditionally, every bullfight in Spain involves six of the specially-bred animals pitted against matadors for 20-30 minutes. At that point, matadors try to drive a sword between the bulls' shoulder blades and through the heart.

Under the new rules in the Balearics, the bull will be submitted for a medical checkup after the fight and then returned to the ranch from where it was raised.

The bill was introduced by Spain's center-left Socialist party (PSOE), the far-left Podemos party and other, smaller regional parties. While the ruling conservative People's Party (PP) and market-friendly Ciudadanos voted against it, they lacked the seats to block the bill.

The Balearic Islands are the second Spanish region where bullfighting is currently restricted. The Canary Islands passed a law in 1991 to protect bulls, including a clause against animal abuse in bullfights or local fiestas.

The northeastern region of Catalonia also passed a bill to ban all bullfighting outright in 2010. But the Constitutional court overruled the law last October, calling bullfighting a cultural asset protected under national law.

Held all over Spain, "la fiesta nacional" - as bullfights are known - is deeply embedded in the country's culture. But there is a growing animal rights protest movement calling for a full ban, rallying outside places like Madrid's emblematic Las Ventas bullring during fighting season.

Reporting by Paul Day; editing by Sarah White and Mark Heinrich

Go here to read the rest:

Spain's Balearic Islands vote to outlaw killing bulls at fights - Reuters

Brooklyn WWII Marine’s remains in Gilbert Islands identified as Joseph Carbone – amNY

Posted on by

A fallen Brooklyn soldier will finally be brought home, nearly 74 years after he gave his life for his country.

The U.S. Department of Defenses POW/MIA Accounting Agency said Monday it has identified the remains of Pvt. Joseph C. Carbone who was killed during a battle in the Pacific Theater. Carbone was a Brooklyn native and part of the 2nd Marine Division, which landed against stiff Japanese resistance on the small island of Betio in the Tarawa Atoll of the Gilbert Islands, on Nov. 20, 1943.

He died during the first day of the three-day battle, according to the Department of Defense. Although Allied forces defeated the Axis troops, nearly 1,000 Marines and sailors were killed and more than 2,000 were wounded.

The dead were buried in temporary cemeteries on the island, but their locations and identities werent recorded.

In 2008, the nonprofit group History Flight conducted an extensive research expedition of the island and discovered five burial sites containing the remains of the battles fallen Marines and sailors.

A year later, the Department of Defense ordered the remains to be recovered and identified.

Carbones interment services are pending. A rosette will be placed at the Walls of the Missing at the American Battle Monuments Commission Honolulu site, according to the Department of Defense.

Brooklyn Borough President Eric Adams said he was relieved to hear that one of the boroughs heros would receive their final respects.

This news helps to fill a hole in our collective hearts, one that we may not recognize every day but one that aches for the thousands of Brooklynites who died in World War II, he said in a statement.

Continue reading here:

Brooklyn WWII Marine's remains in Gilbert Islands identified as Joseph Carbone - amNY

Hawaiian Airlines adding three routes between neighbor islands and West Coast – Honolulu Star-Advertiser

Posted on by

Business Breaking| Top News

By Kathryn Mykleseth kmykleseth@staradvertiser.com

Posted July 24, 2017

July 24, 2017

Updated July 24, 2017 4:43pm

CRAIG T. KOJIMA / MAY 1

Hawaiian Airlines will expand its West Coast presence by adding direct routes between Portland and Maui; Oakland and Kauai; and Los Angeles and Kona.

Hawaiian Airlines said today it will add three new daily routes between the neighbor islands and the U.S. West Coast in early 2018.

The airline said it will expand its West Coast presence by adding direct routes between Portland and Maui; Oakland and Kauai; and Los Angeles and Kona. The arrival of A321neo jets enabled the expansion, the airline said.

The introduction of A321neo service to the Western U.S. heralds the dawn of a new era for Hawaiian Airlines and its guests, said Peter Ingram, executive vice president and chief commercial officer at Hawaiian Airlines, in a statement.

The service between Portland and Maui will launch on Jan. 18. The airlines seasonal service between Oakland and Kauai will be offered until Sept. 4. The route will resume on April 11 as a daily A321neo flight. The daily flight between Los Angeles and Kona launches March 11 with widebody aircraft. The A321neo will be introduced to this route in the summer of 2018.

The airlines maiden A321neo flight between West Coast and Hawaii will be Jan. 8 on its existing Oakland-Maui service.

See the original post here:

Hawaiian Airlines adding three routes between neighbor islands and West Coast - Honolulu Star-Advertiser

The abandoned island you can visit just one day a year – BBC News

Posted on by

BBC News
The abandoned island you can visit just one day a year
BBC News
Ahead, on the cusp of the horizon, a whaleback island rose up, caught between surging tides and the setting sun. A little-known, uninhabited isle in the Orkney archipelago, just north of Scotland's mainland, this was Eynhallow: a place of pilgrimage ...

Read more:

The abandoned island you can visit just one day a year - BBC News

Dogged genetics research identifies genes associated with skin disorder – Clemson Newsstand

Posted on by

CLEMSON With patches of exposed skin, large lesions across her face and dull, expressionless eyes, you might think Lorelei, a Shetland sheepdog, has been abused. But that would be far from the truth: Lorelei is loved and well cared-for. She suffers from a painful condition called dermatomyositis, a genetic skin disorder that affects dogs and humans.

Lorelei, a Shetland sheepdog living in France, became the poster dog for dermatomyositis. The disease caused painful lesions on her face, feet, ears and tail when she was a puppy, as seen in this photo.

The discovery, by Leigh Anne Clark, an associate professor of genetics at Clemson University, and her colleagues, could improve the future for dogs with dermatomyositis. The findings could also give scientists clues into the genetic variations of the 10 in 1 million people who have the disease.

The results from our study can be used as a tool for dog breeders to prevent the disease from affecting puppies, while preserving desirable traits and genetic variation within the breed. Using this new resource, even a dog with dermatomysitis can produce healthy puppies with a mate having a compatible genotype, said Clark.

Before their latest study, Clark and her colleagues were aware of several factors that indicated the disease is multifactorial, deriving from a combination of genetic and environmental effects.

Clark is developing a genetic test for breeders that will tell them the risk of a dame and a sire having puppies with dermatomyositis.

In dogs, dermatomyositis is seen almost exclusively in collie and Shetland breeds. A hereditary disorder will only affect certain breeds, whereas a non-genetic disorder should affect all dog breeds at the same frequency, so Clark knew the disease had a genetic basis.

Her team also recognized that the condition is a complex disorder involving several genetic components as opposed to a simple dominant or recessive disorder because of a wide range of characteristics, or phenotypes, that appear in affected dogs. And they knew that dermatomyositis involved genetic changes in the major histocompatibility complex, which functions in immune defense.

Clark also suspected that an environmental component often triggers onset of the disease because many dog owners reported the animals were under stress when the disease first appeared.

The team used genetic analyses from more than 160 dogs around the world, including Lorelei, who lives in France. Then they conducted a genome-wide association study, or GWAS, to compare genetic variants present in dogs that are affected and unaffected to determine which genetic changes are exclusive to affected dogs. GWAS allowed them to identify an association between a genetic variant and the disease phenotype.

The results displayed a very strong correlation between the dermatomyositis phenotype and variants on chromosomes 10 and 31, suggesting that risk variants for dermatomyositis were located on those chromosomes.

I remember when we saw [the results]. We were speechless. We started looking at the genotypes and writing them down, and it was exciting, said Clark.

Due to the complexity of dermatomyositis, Clark suspects that the genetic variants are working in conjunction to produce the disease. This is known as an additive effect, wherein multiple genetic influences combine to produce the disease phenotype.

Human juvenile dermatomyositis and canine dermatomyositis display similar symptoms and clinical expressions; they are both vasculopathies, affecting connective tissues. Clark hopes her research can be applied to identify risk alleles in humans.

Although this study is a breakthrough in understanding the genetic basis of dermatomyositis, Clark believes there is a lot more to learn about the disease. Future research will focus on dogs with moderate-risk genotypes, specifically asking why some moderate-risk dogs express the disease and others do not. Clark and her team also have a grant with the Collie Health Foundation to investigate moderate risk genotypes.

When Clark was growing up in Texas she worked for a Shetland breeder, an experience that fueled her love of dogs, taught her about breeding techniques and introduced her to genetics. She began researching dermatomyositis in 2004 as a postdoctoral fellow at Texas A&M University, but the work hit a dead end. Clark returned to the project several years later at Clemson, following the invention of new genetic techniques. She received funding to investigate the genetic basis of dermatomyositis in 2010.

Clarks work will help breeders accurately identify which dogs to pair for breeding. By understanding the genetic risks, breeders can selectively mate the dogs to reduce the disease in the population.

Eventually, Clark thinks the disease could be bred out of dogs, leaving collies and Shetlands like Lorelei to be models for good behavior and beauty, and not for a genetic disorder.

END

Continued here:

Dogged genetics research identifies genes associated with skin disorder - Clemson Newsstand

GOP sets Senate health care vote buoyed by McCain return – ABC News

Posted on by

Republican leaders are steering the Senate toward a crucial vote on their bill eviscerating much of President Barack Obama's health care law, buoyed by the near theatrical return to the Capitol of the ailing Sen. John McCain.

No stranger to heroic episodes, the Navy pilot who persevered through five years of captivity during the Vietnam War announced through his office that he would be back in Washington for the critical roll call on beginning debate on the legislation. The 80-year-old has been at home in Arizona since he revealed last week that he's undergoing treatment for brain cancer, but a one-sentence statement said he "looks forward" to returning for work on health care and other legislation.

Senate Majority Leader Mitch McConnell, R-Ky., scheduled the initial health care vote for Tuesday. It seemed unlikely Republicans would bring McCain cross country if they didn't think his vote would make a difference, and his mere presence could make it harder for wavering Republicans to cast a vote against even considering the bill.

Democrats uniformly oppose the effort to tear down Obama's signature legislative achievement. Republicans control the chamber 52-48, meaning they can afford to lose just two Republicans with McCain around and only one in his absence. Vice President Mike Pence would cast a tie-breaking vote.

At least a dozen GOP senators have openly said they oppose or criticized McConnell's legislation, which he's revised as he's hunted Republican support. While it had long seemed headed toward defeat, Republicans Monday began showing glimmers of optimism.

"My mandate from the people of Kentucky is to vote yes, and I certainly intend to do so," McConnell said Monday in what seemed an implicit reminder to his Republican colleagues that they've done the same.

As usual, President Donald Trump was blunter.

"Over and over again, they said, 'Repeal and replace, repeal and replace.' But they can now keep their promise," Trump said of GOP senators in White House remarks.

Senators and aides said talks were continuing that might win over enough Republicans to commence debate. The discussions were covering issues including potentially giving states more leeway to use federal funds to help people losing coverage under Medicaid, the health insurance program for the poor, disabled and nursing home patients.

Should Tuesday's vote fail, it would be an unalloyed embarrassment for a party that finally gained control of the White House, Senate and House in January but still fell flat on its promise to uproot Obamacare. Republicans could try returning to the bill later this year if they somehow round up more support.

Should the initial motion win, that would prompt 20 hours of debate and countless amendments in a battle likely to last all week. Moderate and conservative Republicans would try reshaping the bill in their direction while Democrats would attempt to force GOP senators to cast difficult votes aimed at haunting them in re-election campaigns.

Even then, the measure's ultimate fate still seemed iffy because of GOP divisions.

Obama's law was enacted in 2010 over unanimous Republican opposition. Since then, its expansion of Medicaid and creation of federal insurance marketplaces has produced 20 million fewer uninsured people. It's also provided protections that require insurers to provide robust coverage to all, cap consumers' annual and lifetime expenditures and ensure that people with serious medical conditions pay the same premiums as the healthy.

The law has been unpopular with GOP voters and the party has launched numerous attempts to dismantle the statute. All until this year were mere aspirations because Obama vetoed every major one that reached him.

Ever since 2010, Republicans have been largely united on scuttling the statute but divided over how to replace it.

Those divides sharpened with Trump willing to sign legislation and estimates by the nonpartisan Congressional Budget Office that several GOP bills would cause more than 20 million people to become uninsured by 2026. Polls showing growing popularity for Obama's law and abysmal approval ratings for the GOP effort haven't helped.

The House approved its version of the bill in May after several setbacks. It's similar to the Senate measure McConnell unveiled in June after writing it privately. But he's also revised it in his hunt for GOP votes.

McConnell's bill would abolish much of Obama's law, eliminating its tax penalties on people not buying policies, cutting Medicaid, eliminating its tax boosts on medical companies and providing less generous health care subsidies for consumers.

Moderate Sen. Susan Collins, R-Maine, has remained opposed to beginning debate on any option McConnell has revealed so far. Conservative Sen. Rand Paul, R-Ky., said he would vote no unless leaders agreed to an early vote on simply repealing Obama's statute and giving Congress two years to replace it.

Conservatives were seeking language letting insurers offer bare-bones policies with low premiums, which would be illegal under Obama's law. Moderates from states whose low-income residents rely heavily on Medicaid were resisting the GOP bill's cuts in that program.

Associated Press writers Erica Werner and Andrew Taylor contributed to this report.

Continue reading here:

GOP sets Senate health care vote buoyed by McCain return - ABC News

Senate Health Care Vote: What to Expect – New York Times

Posted on by

The Senate majority leader, Mitch McConnell of Kentucky, says it is time for a vote.

On Tuesday, he will have the Senate vote on a motion to proceed in this case, on whether to take up a health care repeal bill that narrowly passed the House in May.

Nobody expects that bill to become law. Instead, it would essentially serve as the vehicle for the Senates legislation. The House bills text would be swapped out for the Senates preferred language, whatever that ultimately is.

Republicans hold 52 seats in the Senate, and to be successful, they need a majority for the motion to proceed. In a deadlock, Vice President Mike Pence would break the tie in favor of proceeding.

Only days after announcing he has brain cancer, Senator John McCain, Republican of Arizona, plans to return to the Senate on Tuesday. His presence means Mr. McConnell can afford for only two Republicans to vote against the motion. If Mr. McCain had been absent, Mr. McConnell would have been able to lose only a single Republican.

At least one defection is all but certain: Senator Susan Collins of Maine indicated on Monday that she would vote against proceeding in just about every imaginable circumstance.

One big factor is what Mr. McConnell plans to do after the procedural vote.

For example, Senator Rand Paul of Kentucky is an expected no vote if after clearing the procedural hurdle, the Senate turns to a bill by Mr. McConnell to repeal and replace the health law. Mr. Paul detests that bill.

On the other hand, Senators Shelley Moore Capito of West Virginia and Lisa Murkowski of Alaska indicated last week that they would not vote to proceed if Mr. McConnell afterward scheduled a vote on a bill to repeal the health law without providing a replacement.

In addition, a number of other Republican senators have expressed varying qualms, with varying degrees of certitude. They include Mike Lee of Utah, Jerry Moran of Kansas, Rob Portman of Ohio and Dean Heller of Nevada.

Such a vote would start the debate in the Senate on health care. At some point, Mr. McConnell is expected to offer an amendment that would substitute a new measure for the text of the bill that passed the House. But it remains to be seen what that new measure would be. Republicans are trying to pass the bill using special budget rules that limit debate to 20 hours and prevent a Democratic filibuster.

Republicans are not expected to abandon their repeal effort, but its future would appear bleak, at least in the short term.

Well go back to the drawing board, Senator John Thune of South Dakota, a member of the Republican leadership, said on Fox News Sunday. Of voting to repeal and replace the health law, he said, Its not a question of if, its a question of when.

Recent history provides some support for Mr. Thunes optimism. The repeal bill in the House was declared dead before coming back to life and Republicans there ultimately were successful in passing a bill.

View original post here:

Senate Health Care Vote: What to Expect - New York Times

Senate healthcare vote presents critical test for GOP and Trump – Los Angeles Times

Posted on by

With a strong, last-minute push from President Trump, Senate Republicans face a pivotal vote Tuesday in their long bid to repeal or replace the Affordable Care Act.

But the outcome remained in doubt, largely because senators have not even been told which of the various GOP plans will be considered.

Senate Majority Leader Mitch McConnell has kept the process highly secretive as he tries to find a path forward amid stark opposition within his Republican majority, and no backing from Democrats. He has only two Republican votes to spare.

In a sign of the votes importance, Republican Sen. John McCain of Arizona who was diagnosed last week with brain cancer following an operation to remove a blood clot announced late Monday he would return to Washington in time for Tuesdays proceeding.

But many GOP senators remain reluctant to begin formal debate on legislation without knowing where the process will end.

Trump warned senators Monday that Tuesdays planned motion to proceed the first legislative hurdle to passing a Senate bill could be Republicans last chance to undo the Affordable Care Act, also known as Obamacare.

He warned in a tweet that the repercussions will be far greater than any of them understand! hinting of political fallout for senators who vote against the measure.

Any senator who votes against starting debate is telling America that you are fine with the Obamacare nightmare, Trump said during an event at the White House.

For Senate Republicans this is their chance to keep their promise. Over and over again they said repeal and replace, repeal and replace, he said. Theres been enough talk and no action. Now is the time for action.

For Trump, a failure Tuesday could expose the limits of his ability to implement his agenda, even with a GOP-controlled Congress.

But neither admonitions from the White House nor pressure from outside conservative groups seemed strong enough to sway some key centrist Republican senators, who continued to express concerns about how the GOP plan might affect low-income residents in their states, particularly those receiving Medicaid.

The nonpartisan Congressional Budget Office estimated that at least 22 million more Americans would not have coverage under the Republicans repeal and replace plan, and as many as 32 million would join the ranks of the uninsured if Republicans simply repealed the Affordable Care Act.

Among the holdouts is Sen. Shelley Moore Capito of West Virginia, a state with a greater share of its population covered by Medicaid than any other. Trump traveled there Monday evening for a rally at the National Jamboree of the Boy Scouts of America.

Many West Virginians have benefited from our states decision to expand Medicaid, Capito wrote in her weekly newsletter ahead of the visit. I am committed to repealing Obamacare and replacing it with a healthcare system that provides access to affordable care to West Virginians... After meeting with President Trump this week, he assured me we are on the same page.

During his remarks in West Virginia, Trump told his Health and Human Services Secretary Tom Price, "You'd better get Sen. Capito to vote for it."

Otherwise, Trump joked, if Price doesnt produce the needed Senate votes, Youre fired!

Ahead of Tuesdays vote, at least one Republican, Sen. Susan Collins of Maine, remained opposed to opening debate on the bill.

McConnell can afford to lose no more than two Republicans from his 52-seat majority. If needed, Vice President Mike Pence could be called on to cast a tie-breaking vote.

With McCains unexpected return, GOP leaders are hoping they will be able to garner at least 50 votes. Though the Arizona senator has not indicated how he would vote, it seemed unlikely that he would make the trip to vote no. He also expressed a desire to vote on other pressing issues, such as a defense authorization bill and Russia sanctions legislation.

Some Republican senators who initially said they would oppose opening debate on the bill, including Sen. Rand Paul of Kentucky, have since indicated they may be willing to start debate.

Paul and other conservatives support repealing Obamacare but oppose the latest version of the replacement plan, saying it did not go far enough in dismantling the current law.

Among the centrists, the votes of Capito and Sen. Lisa Murkowski (R-Alaska) remained the most closely watched.

Also not saying how he would vote was Sen. Mike Lee of Utah, who had joined Paul in opposing the replacement plan.

McConnell nudged senators forward, promising a free-wheeling floor debate that would allow both GOP centrists and conservatives an opportunity to offer amendments to shape the final product.

We ought to have the debate, McConnell said Monday.

The only way well have an opportunity to consider ideas is if senators are allowed to offer and debate them, McConnell said. That means voting to kick off a robust debate.

Senators, though, remained skeptical that their amendments would be included in the final product. Many, including McCain, have called for a return to a traditional legislative process of holding public hearings and debating the bill in committee before pushing it to the floor.

Its highly unusual for senators to vote to proceed on such an important piece of legislation without knowing the preferred plan of GOP leadership.

Technically, the vote Tuesday will be to proceed to the House-passed Obamacare overhaul, the American Health Care Act, which has almost no support in the Senate.

After crossing that initial hurdle, GOP leaders will substitute the House version with the Senate-preferred alternative. But as late as Monday evening, it was unclear which version GOP leadership preferred: a simple repeal or repeal and replace.

Even if McConnell succeeds Tuesday in bringing enough senators to support beginning debate on a bill, final passage remains a challenge.

McConnell appears to be calculating that its better to hold Tuesdays vote and risk an embarrassing defeat in the hopes it will pressure senators to find consensus.

The leader has promised to keep senators in session through the first two weeks of August, cutting into their summer recess, to pass a final bill, meaning Tuesdays vote may not be their last.

Obamacare vs. Trumpcare: A side-by-side comparison of the Affordable Care Act and the GOPs replacement plan

Obamacare 101: A primer on key issues in the debate over repealing and replacing the Affordable Care Act.

"The Tick" cast talks about the humor behind the series and the fan pressure that comes with a cult classic.

"The Tick" cast talks about the humor behind the series and the fan pressure that comes with a cult classic.

Melissa Benoist, David Harewood, Mehcad Brooks, Jeremy Jordan, Chris Wood, Katie McGrath, Odette Annable, plus two EPs:Jessica Queller, Robert Rovner talk "Supergirl" at Comic-Con 2017.

Melissa Benoist, David Harewood, Mehcad Brooks, Jeremy Jordan, Chris Wood, Katie McGrath, Odette Annable, plus two EPs:Jessica Queller, Robert Rovner talk "Supergirl" at Comic-Con 2017.

Lisa.Mascaro@latimes.com

@LisaMascaro

noam.levey@latimes.com

@noamlevey

UPDATES:

6:45 p.m.: This story was updated after Sen. John McCain announced he would return to Washington for the vote.

This story was originally published at 3:40 p.m.

Read the original:

Senate healthcare vote presents critical test for GOP and Trump - Los Angeles Times

Students With Disabilities Are the Overlooked Victims of GOP Health Care Repeal – Mother Jones

Posted on by

Superintendents are concerned cuts to Medicaid will threaten crucial medical services.

Edwin RiosJul. 24, 2017 3:15 PM

FatCamera/GettyImages

On the eastern end of Michigans Upper Peninsula, Rachel Fuerers school district depends on more than $870,000 in Medicaid funds each year. About 5 percent ofits overall annualbudget, the money goestowardprovidingmedical services for more than 1,000 special education students in 19 public schools across 4,000 square miles.

Those dollars, though, will be at risk if Republicans in Washington get their way.

Lastweek, Senate Republicans effortsto repeal and replaceand then just repealObamacare appeared to be dead. But now, at President Trumps urging,senators are again poisedfor avote on some sort of health care overhaul thisweek, though it remains to be seenwhether theyll vote ona partial repeal of the Affordable Care Act (ACA)or a full repeal and replace effort.

What is clear is thatpassing either bill would greatly endanger the future of Medicaid.Therepeal and replace bill, the Better Care Reconciliation Act (BCRA), imposes a cap on Medicaid spending that would sharplyreduce funds in future years. The alternative, a bill introduced by Senate Majority Leader Mitch McConnell (R-Ky.) lastweek that mirrors a 2015 effort to mostly repeal Obamacare, would shrink Medicaid spending over the next decade, especially for people who were newly eligible to receive Medicaid through the ACA.

Though McConnell currently lacks the votes to ensure either bills passage, school officials and advocates are keeping their eyes trainedon theSenate.

Were being told that special education is being spared,says Fuerer,who works as special education director at MichigansEastern Upper Peninsula Intermediate School District, but thats not true at all if there are cuts to Medicaid.

Fuerer joined hundreds of school superintendents from across the country in recentweeks indescending uponWashington to lobbyCongress, meeting with their states representatives to convince them to leave Medicaid alone.Theyre lumping everything together in this repeal and replace, Fuerer says. In doing so, they are trying to fix something thats not broken. Medicaid isnt broken.

ToFuerers surprise, even just the fewHouse members she met with the otherweek didnt fully understand the extent of just how much schools rely on Medicaid dollars.While the programis primarily used to fund health insurance for low-income families,italso doles out billions of dollars each year directly to school districts to fund special education services that are mandated by federal law and to supportservices for poor students.Nowschool superintendents, from both red and blue states, are expressingconcern that the GOPs health care plans would forcedistricts to struggle to provide crucialservices for their most vulnerablestudents.

Intalking about the bill as a repeal of Obamacare, Republican rhetoric can be misleading. BCRAwould fundamentally remake Medicaid, a program that has operated since 1965,slicingspending by$772 billion over the next decade andleaving 15 million morepeople uninsured.It does this by turning Medicaid from an open-ended promise from the government to fund state needsinto a block grant or per capita programstarting in2020 that will limit the amount of money each state receives.

Currently,John Hill, executive director of the National Alliance for Medicaid in Education, estimates thatalmost half the children in this country are on Medicaid. So any cut is going to have an adverse effect on kids, no matter how much theyll assure us its not going to happen,Hill tellsMother Jones.

As mandated by the Individuals with Disabilities EducationAct (IDEA) from 1975, schools must provide a free and appropriate public education to students with disabilities. But Congress has historically fallen short on covering the cost of educating special-needs students under IDEA, so states and local agencies have turned toMedicaid tohelp offset the difference and paythe cost for medical services and equipment. Starting in 1988, schools, like hospitals and insurance providers, have been able to request reimbursements from the Department of Health and Human Services for such expenditures.

In 2015, schools received nearly $4 billion in Medicaid funding, according to the Center on Budget and Policy Priorities, less than a third of whats doled out for special education spending under IDEA.

That $4 billion has been essential to schools covering the costs of speech and physical therapy, behavioral services, and medical equipment, such as wheelchairs and walkers. For low-income students, districts use such funds to pay for vision, hearing, and other healthscreenings, as well asforschool nurses and mental health services. Its also important to note thatschool districts dont just offer services to poor and disabled students during school hours; they act as a connecting point to enrolleligible low-income families in Medicaid and in the Childrens Health Insurance Program and help families find health care providers. In turn, schools get reimbursed for their outreach efforts.

Sherri Smith, thesuperintendent of Lower Dauphin, a suburban Pennsylvaniaschool district of just 4,000 students, was also in D.C. recentlyto meet with representatives. She says the small but targeted $50,000 in Medicaid funding her district receives each yearhelps pay for more medical-related services, such as occupational and physical therapy, along with mental health services to serve a growing need for students in the district. The money also allows the district to pay for additional nurses and personal care aides for students with disabilities and low-income students.

Schools are doing more outside the peripheral of just teaching and educating, Smith says. So actually getting Medicaid dollars for medical services for our students, which they need, allows us the opportunity to do so. Im afraid that without those kinds of dollars, that will go away.

In the broader Medicaid landscape, funding for school districts is just onetiny line item. The National Alliance for Medicaid in Education projects that school districts receive less than 1 percent of all Medicaid reimbursements.

Whileneither the House nor Senate repeal-and-replacebill specifically targets funding for schooldistricts, with lessmoneycoming from Washington, states would be forced to make cuts elsewhere in their budgets, which could impactthe entire community. Districts might be pushedto raise taxes, reduce spending on general education programs, or cutstaff.

If schools opted to diminish services for special education students, they could be at risk of falling out of compliance withfederal law, opening districts up to lawsuits and a potential furtherloss of federal dollars. This is all the more complicated due toa recent Supreme Court ruling that raisesthe standard for what schools mustoffer and expands students rights.

We would have to be stringent on services if Medicaidfunding went away, Smith, of Pennsylvania, says. Depending on what types of services, if that child needs it, and its mandated in their individualized education plan under IDEA, then we would have to provide those services. Wed have to find our money, which means we would have to go back to our taxpayers in Pennsylvania, or we would have to look to cut from our regular programs to make up the difference.

Similarly, for Fuerer in Michigan, the cuts to Medicaid funding in the proposedhealth care billswould mean that her district might need to slashitsoverall budget just so the same level of services can be provided. The reality is, [the loss of Medicaid funding is]probably not cutting a speech provider. Its probably cutting a gen ed teacher, she says. If you weaken the general education services, you are weakening the entire school community.

Sasha Pudelski, assistant director of policy and advocacy for the School Superintendents Association, says that changes to Medicaid could put schools in competition with hospitals and insurance providers for Medicaid dollars. Schools cant compete with other frontline healthcare providers for those dollars, Pudelski says. From our perspective, it could end Medicaid as we know it in schools.

Fuerer attended three meetings with Michigan representatives when she was in Washington this month, at least one of which really surprised her.When she sat downwith freshmanRepublicanRep. Jack Bergman,she says,he had never heard about the Medicaid billing in schools and he listened very intently. He didnt commit to changing his position, she says. He voted for the House bill. But he was interested and asked us for more information. It was better than the other ones.

Fuerer also met with an aide toRepublican Rep. Justin Amash, whovotedyes onthe House bill that includedcuts to Medicaid spending similar to those in the Senate proposals. In themeeting, Fuerer recallsthe staffer saying that Amash believed that responsibility for funding education and healthcare should be left to states. Fuerer says she finds that response a little frustrating.

Chris Kjolhede, co-director of the school-based health program at Bassett Healthcare Network, which runs in-school health clinics across 15 school districts in rural upstate New York,says that though its still tooearly to know the extent of damage on schools in the GOPs legislation, it is terrifying to think about major cuts to Medicaid. He says roughly half of children who work with his program receive care as a result of Medicaidfunding andNew Yorks Child Health Plus program.

Kjolhedes program relies on the federalfunds to provide dental, physical, and mental health services for more than 7,500 kids. He adds that Bassett has advocatedwith elected officials to let them know that the group was concerned about the loss of Medicaid reimbursements in his red district. If the funding goes away, theoretically, I have to figure out how to pay for the services or the services go away, Kjolhede tells Mother Jones. Theres a lot of pressure on those of us trying to make it happen.

Edwin Rios is a reporter at Mother Jones. Reach him at erios@motherjones.com.

Mother Jones is a nonprofit, and stories like this are made possible by readers like you. Donate or subscribe to help fund independent journalism.

Visit link:

Students With Disabilities Are the Overlooked Victims of GOP Health Care Repeal - Mother Jones

Trump to rally in Ohio as residents worry about health care – ABC News

Posted on by

President Donald Trump is scheduled to visit Ohio on Tuesday as the U.S. Senate will vote on whether to open debate on legislation to repeal and replace former President Barack Obama's health care law.

Ohioans have mixed feelings about Republicans' efforts.

Some who rely on government assistance say they're having trouble sleeping at night because they're worried about their medical expenses. Others are hopeful the Republican plan will make things more affordable for them.

Trump, who is scheduled to hold a rally in Youngstown Tuesday night, has been urging Republicans to support an effort to repeal and replace Obama's statute.

Republican Ohio Sen. Rob Portman could be a key swing vote on the GOP legislation and is facing immense pressure from both sides.

On Saturday, Vice President Mike Pence attended the Ohio Republican Party's annual fundraising dinner in Columbus and urged GOP senators to get rid of Obama's health care policies. Then on Monday, Republican Gov. John Kasich, said the Senate should not try to "force a one-sided deal that the American people are clearly against."

Here's a look at some residents' concerns.

FIGHTING FROM A WHEELCHAIR

Darrell Price, 50, was born with cerebral palsy and had his left hip replaced in 2009. He lives in subsidized housing in Cleveland, gets around with a power wheelchair and relies on a Medicaid-funded assistant to wash him, dress him and cook his meals, six hours a day, six days a week.

"If those Medicaid cuts go through, I'm scared to death," said Price, who voted for Hillary Clinton in November. "If I don't have enough funding to go into a nursing home, what happens then? Do I go on the street?"

Price has been jumping on activist conference calls, emailing Portman's office every week and drove two hours to Columbus on Saturday to protest when Pence came to visit.

"Every week, they're saying 'oh, we're going to have a vote in a week,' and then it fails," Price said. "There's no certainty at all. I'll go down fighting if that's what I have to do."

STUCK IN YOUNGSTOWN, HOPEFUL FOR TRUMP

Judy Martin, 72, of McDonald, Ohio, "tosses and turns" in bed at night, wracked with leg pain and worried about her medical bills.

Martin, a factory worker for 51 years, said she burned through her $20,000 in life savings on medical expenses a year after her retirement. She now relies on a monthly $1,500 social security check and Medicare subsidies, but still has to pay roughly $400 a month for medication and supplemental insurance.

"It feels like we're getting punished as we're getting older," Martin said. "I earned my time out there. Here we are, stuck."

Martin, who voted for Trump, "has faith" that Trump will make things more affordable for her, and is excited to hear what Trump has to say about health care when he comes to Youngstown, a 15 minute drive from her home. Though she won't be there in person, she plans to watch his speech online and hear about it from her son who plans to attend.

"I believe that Trump can do it, and that he will take care of the little people," Martin said.

CONSIDERING MOVING OUT OF STATE

Jeni and Kevin Potter's 13-year-old daughter, Erin, has battled leukemia three times and received bone marrow transplants twice. Over the years, their medical bills ran into the millions, mostly paid for by company insurance after the Affordable Care Act removed lifetime caps on insurance payouts.

So when the Potters heard that Congress was trying to repeal the Affordable Care Act, they looked at each other and thought, "What are we going to do?"

"You've got to think out of the box," said Jeni Potter, who voted for Clinton. "Where can we go to get her health care?"

The Potters, who live east of Cleveland, jumped online and scouted houses in Canada. Jeni Potter emailed every single representative in the Massachusetts House, seeing if they would be covered by that state's near-universal health system.

They've relaxed a little in the past month as Republican plans have stalled, but worries still loom large in the back of their minds.

"What does this mean for her?" Jeni Potter asked. "I have no idea. That unknown is terrifying."

Go here to see the original:

Trump to rally in Ohio as residents worry about health care - ABC News

Looking back on ‘healthcare hell’ – The Hill

Posted on by

Bart Stupak didnt seek out the role he played in the fight over ObamaCare more than seven years ago, but he has no regrets over how it all played out.

Stupak, a Democrat who opposes abortion and represented Michigans 1st Congressional District for 18 years, became a thorn in his partys side over demands in the debate on the Affordable Care Act (ACA) against funding for abortion. Now, hes telling his side of the drama during former President Obamas first term that resulted in the passage of the massive healthcare overhaul, just as Republicans struggle in their multi-year push to repeal it.

Stupak eventually voted to pass the final legislation, after securing a promise from Obama that reinforced the governments commitment to the Hyde Amendment.

The book is not about me. The important part is the small group of folks who pushed healthcare over the finish line, Stupak said in a recent interview in his Washington office at law firm Venable, where he now works.

Stupak sees his role in the ObamaCare debate as successful, though it didnt come without sacrifices. Threats were made on his life, and he continues to describe the experience as his personal healthcare hell.

Now, Stupak has a unique perspective watching Republicans wage their own war to dismantle Obamas signature healthcare law.

A months-long push by lawmakers to repeal and replace ObamaCare was left in shambles last week when several Senate Republicans came out against the legislation. The defeat was compounded less than 24 hours later, when certain GOP senators also refused to back a repeal-only plan.

The developments have left Republicans frustrated and with no clear path forward, though Senate Majority Leader Mitch McConnellMitch McConnellMcConnell to pin down colleagues on healthcare Looking back on healthcare hell McCain returning to Senate in time for health vote MORE (R-Ky.) has pledged to hold a vote this week.

The problem, Stupak says, is that lawmakers are focused on politics over policy.

If they want to repeal the Affordable Care Act, every Congress has that right. All I ask is you do it based on policy reasons and not based on political reasons, Stupak said. The policy decisions are no longer debated. Its all politics.

President Trump campaigned on repealing and replacing ObamaCare and has shown growing frustration with GOP lawmakers failure to get the job done. Following the efforts collapse last week, Trump hosted Republican senators at the White House, scolding them over the stalled push and demanding they revive it.

Stupak sees the healthcare fight as indicative of the growing partisanship in Washington, which festered when he was in Congress.

His book lays out a dramatic 2009 scene in which a staffer on the House Energy and Commerce Committee ripped into him privately for voting in favor of a Republican amendment aimed at boosting disclosure of medical costs offered to eventually unsuccessful healthcare legislation.

What are you doing voting with the Republicans? the staffer said, according to Stupaks account.

Stupak says that the divide has gotten worse since, blaming it partially on the increase of money in politics.

Its hard to get to know some of the Republicans. Ive tried, he said. And even some of the Democrats.

The White House, he says, is also a big part of the problem.

Weve got political slogans, but we dont have any policy. Thats my objection with President Trump. I think he means well; he just doesnt know anything, Stupak said.

Stupak announced that he would not seek reelection in 2010, the same year the ACA was passed, a decision he says he made the night Obama was elected in 2008. Stupak, who had contemplated retiring multiple times before, says Obamas win gave him confidence that the Democrats would be able to pass comprehensive healthcare reform, allowing him to retire having fulfilled a major campaign promise he made when he first ran in the early 1990s.

The former police officer maintains he was never going to be a lifer in Congress. I told my wife, Im done. No more. She didnt believe me, but I kept that promise, he said. As things unfolded over those two years, it just reinforced my decisionmaking.

Since he stepped away, he has had more time to devote to law and his family, spending fewer hours on the road trying to span his 600-mile-wide district in Michigan.

Still, Stupak keeps in touch with his old colleagues when he is in Washington. He has served as a fellow at the Harvard Institute of Politics and hopes to keep writing books.

My life is just less complicated, he said.

Stupak has also continued to insert himself into the nexus of healthcare and law, filing Supreme Court briefs supporting Hobby Lobby and Little Sisters of the Poor in objection to the governments application of ObamaCares contraception mandate.

In the six years following his exit, Washington has undergone a tremendous shift the administration of a new, unexpected Republican president has taken over, and the GOP has claimed control of both chambers of Congress.

When asked for his opinion of Trump, Stupak made the unusual move of drawing a parallel between the president and his predecessor, whom he was reluctant to support for president because of his dearth of experience.

I always thought it was good to shake up Washington, but I also believe that experience comes in government. He has no experience, and it shows, Stupak said of Trump.

Read the original:

Looking back on 'healthcare hell' - The Hill

The Conservative Case for Universal Healthcare – The American Conservative

Posted on by

Dont tell anyone, but American conservatives will soon be embracing single-payer healthcare, or some other form of socialized healthcare.

Yes, thats a bold claim given that a GOP-controlled Congress and Presidentare poised to un-socialize a great deal of healthcare, and may even pull it off. But within five years, plenty of Republicans will be loudly supporting or quietly assenting to universal Medicare.

And thats a good thing, because socializing healthcare is the only demonstrably effective way to control costs and cover everyone. It results in a healthier country and it saves a ton of money.

That may seem offensively counterintuitive. Its generally assumed that universal healthcare will by definition cost more.

In fact, in every first-world nation that has socialized medicinewhether it be a heavily regulated multi-insurer system like Germany, single-payer like Canada, or a purely socialized system like the United Kingdom-it costs less. A lot, lot less, in fact: While healthcare eats up nearly 18 percent of U.S. GDP, for other nations, from Australia and Canada to Germany and Japan, the figure hovers around 11 percent. (Its no wonder that smarter capitalists like Charlie Munger of Berkshire Hathaway are bemoaning the drag on U.S. firm competitiveness from high healthcare costs.) Nor are healthcare results in America anything to brag about: lower life expectancy, higher infant mortality and poor scores on a wide range of important public health indicators.

Why does socialized healthcare cost less? Getting rid of private insurers, which suck up a lot money without adding any value, would result in a huge savings, as much as 15percent by one academic estimate published in the American Journal of Public Health. When the government flexing its monopsony muscle as the overwhelmingly largest buyer of medical services, drugs and technology, it would also lower prices-thats what happens in nearly every other country.

So while its a commonly progressive meme to contrast the national expenditure of one F-35 with our inability to afford single-payer healthcareand I hesitate to say this lest word get out to our neocon friendsthere is no need for a tradeoff. If we switched to single payer or another form of socialized medicine, we would actuallyhave more money to spend on even more useless military hardware.

The barrier to universal healthcare is not economic but political. Is profligate spending on health care really a conservative value? And what kind of market incentives are working anywayits an odd kind of market transaction in which the buyer is stopped from negotiating the price, but that is exactly what Medicare Part D statutorily requires: The government is not allowed to haggle the prices of prescription drugs with major pharmaceutical companies, unlike in nearly every other rich country. (Both Hillary Clinton and Donald Trump pledged to end this masochism, but the 45th president has so far done nothing, and U.S. prescription drug prices remain the highest in the world.) Does anyone seriously think medical savings accounts with their obnoxious complexity and added paperwork are the right answer, and not some neoliberal joke?

The objections to socialized healthcare crumble upon impact with the reality. One beloved piece of folklore is that once people are given free healthcare theyll abuse it by going on weird medical joyrides, just because they can, or simply let themselves go because theyll have free doctor visits. I hate to ruin this gloating fantasy of lumpenproletariat irresponsibility,but people need take an honest look at the various health crises in the United States compared to other OECD(Organisation for Economic Cooperation and Development) countries. If readily available healthcare turns people hedonistic yahoos, why does Germany have less lethal drug overdoses than the U.S. Why does Canada have less obesity and type II diabetes? Why does the Netherlands have less teen pregnancy and less HIV? The evidence is appallingly clear: Among first-world countries, the U.S. is a public health disaster zone. We have reached the point where the rationalist santera of economistic incentives in our healthcare policies have nothing to do with people as they actually are.

If socialized medicine couldbe in conformity with conservative principles, what about Republican principles? This may seem a nonstarter given the pious market Calvinism of Paul Ryan and Congressmen like Reps. Scott Perry (R-Pa.) and Mo Brooks (R-Ala.), who seem opposed to the very idea of health insurance of any kind at all. But their fanaticism is surprisingly unpopular in the U.S. According to recent polling, less than 25 percent of Americans approve of the recent GOP healthcare bills. Other polls show even lower numbers. These Republicans arealso profoundly out of step with conservative parties in the rest of the world.

Strange as it may seem to American Right, $600 EpiPens are not the sought-after goal of conservatives in other countries. In Canada, the single-payer healthcare system is such a part of national identity that even hard-right insurgents like Stockwell Day have enthusiastically pledged to maintain it. None of these systems are perfect, and all are subject to constant adjustment, but they do offer a better set of problemsthe most any mature nation can ask forthan what we have in the U.S.

Andvirtually no one looks at our expensive American mess as a model.

I recently spoke with one German policy intellectual, Nico Lange, who runs the New York outpost of the German Christian Democrats main think tank, the Konrad Adenauer Stiftung, to get his thoughts on both American and German healthcare. Is socialized medicine the entering wedge of fascism and/or Stalinism? Are Germans less free than Americans because they all have healthcare (through a heavily regulated multi-payer system), and pay a hell of a lot less (11.3 percent of GDP) for it?

Mr. Lange paused, and took an audible breath; I felt like I had put him in the awkward spot of inviting him over and asking for his honest opinion of the drapes and upholstery. Yes, he said, we are less free but security versus freedom is a classic balance! National healthcare makes for a more stable society, its a basic service that needs to be provided to secure an equal chance for living standards all over the country. Even as Mr. Lange delineated the conservative pedigree of socialized medicine in GermanyYou can certainly argue that Bismarck was a conservative in founding this systemI had a hard time imagining many Democrats, let alone any Republican, making such arguments.

Indeed, the official GOP stance is perhaps best described as Shkrelism than conservatism, after the weasel-faced pharma entrepreneur Martin Shkreli, who infamously jacked up the price of one lifesaving drug and is now being prosecuted for fraud. (Though in fairness, this type of bloodsucking awfulness is quite bipartisan: Heather Bresch, CEO of Mylan corporation, which jacked up the price of EpiPens from $100 to $600, is the daughter of Senator Joe Manchin (D-WV), who defended his daughters choice.)

But GOP healthcare politics are at the moment spectacularly incoherent. Many GOP voters have told opinion polls that they hate Obamacare, but like the Affordable Care Act. And as the GOP healthcare bill continues to be massively unpopular, Donald Trump has lavished praise on Australias healthcare system (socialized, and eating up only 9.4 percent of the GDP there). Even in the GOP, this is where the votes are: Trumps move to the center on questions of social insuranceMedicare, Medicaid, Social Securitywas a big part of his appeal in the primaries. The rising alt-Right, not to hold them up as any moral authority, dont seem to have any problem with universal Medicare either.

It will fall on reform conservatives to convince themselves and others that single-payer or some kind of universal care is perfectly keeping with conservative principles, and, for the reasons outlined above, its really not much of a stretch. Lest this sound outlandish, consider how fully liberals have convinced themselves that the Affordable Care Acta plan hatched at the Heritage Foundation for heavens sake, and first implemented by a Republican governoris the every essence of liberal progressivism.

Trumps candidly favorable view of Australian-style socialized healthcare is less likely a blip than the future of the GOP. Republican governors who actually have to govern, like Brian Sandoval and John Kasich, and media personalities like Joe Scarborough, and the Rock, will be soon talking up single-payer out of both fiscal probity, communitarian decency, and the in-your-face evidence that, ideology aside, this is what works. Even the Harvard Business Review is now giving single-payer favorable coverage. Sean Hannity and his angry brigade may be foaming at the mouth this week about the GOP failure to disembowel Obamacare, but Seans a sufficiently prehensile fellow to grasp at single-payer if it seems opportunejust look at his about-face on WikiLeaks. And though that opportunity has not arisen yet, check again in two years.

The real obstacle may be the Democrats. As Max Fine, last surviving member of John F. Kennedys Medicare task force, recently toldthe Intercept, Single payer is the only real answer and some day I believe the Republicans will leap ahead of the Democrats and lead in its enactment, he speculated, just as did Bismarck in Germany and David Lloyd George and Churchill in the UK. For now, an invigorating civil war is raging within the Democrats with the National Nurses Union, the savvy practitioner-wonks of the Physicians for a National Health Program, and thousands of everyday Americans shouting at their congressional reps at town hall meetings are clamoring for single-payer against the partys donor base of horrified Big Pharma executives and affluent doctors. In a few years there might even be a left-right pincers movement against the neolib/neocon middle, whose unlovable professional-class technocrats are the main source of resistance to single payer.

I dont want to oversell the friction-free smoothness of the GOPs conversion to socialized healthcare. Our funny country will always have a cohort of InfoWars ooga-boogas, embittered anesthesiologists and Hayekian fundies for whom universal healthcare is a totalitarian jackboot. (But, and not to be a jerk, its worth remembering that Hayek himself supported the socialized healthcare of Western Europe in one of his most reasonable passages from the Road to Serfdom.)

So even if there is some banshee GOP resistance at first, universal Medicare will swiftly become about as controversial as our government-run fire departments. Such, after all, was the trajectory of Medicare half a century ago. You read it here first, people: Within five years, the American Right will happily embrace socialized medicine.

Chase Madar is an attorney in New York and the author of The Passion of Bradley Manning: The Story Behind the Wikileaks Whistleblower.

View original post here:

The Conservative Case for Universal Healthcare - The American Conservative

The ‘clock is ticking’ on Republican health-care legislation: Analyst – CNBC

Posted on by

It's coming down to the wire for the Republican health-care bill, with President Donald Trump's push for action on Monday almost sounding like a "Hail Mary," analyst Ipsita Smolinski told CNBC.

Trump on Monday stepped up pressure on Senate Republicans to come up with a plan to overhaul the U.S. health-care system, saying they have "not done their job in ending the Obamacare nightmare."

"If this doesn't get done before the August recess, then really the calendar is running out," said Smolinski, managing director and health-care analyst at Capitol Street.

"The budget resolution ends Oct. 1, so you could come back in September and get this done but the clock is really ticking," she said in an interview with "Closing Bell" on Monday.

Senate Republican leaders are pushing for a vote Tuesday on a motion to proceed with a health-care bill, which would allow it to go up for debate and possible amendments. However, they have not made clear which plan they want to move forward on once they proceed with the proposal.

The Senate GOP's bill to repeal and replace Obamacare stalled out after not garnering enough support for passage. And its plan to simply repeal Obamacare and replace it during a two-year transition period also lacks support.

Smolinski said it looks like changes will have to be made for a plan to gain traction.

"A deal would have to come through in the coming days, whether it's additional Medicaid dollars or something that would get some of those moderate senators who have expanded Medicaid to sign on."

CNBC's Jacob Pramuk contributed to this report.

Excerpt from:

The 'clock is ticking' on Republican health-care legislation: Analyst - CNBC

Stealth BioTherapeutics Initiates Phase 2/3 Study of Elamipretide in Patients With Barth Syndrome – Markets Insider

Posted on by

BOSTON, July 24, 2017 /PRNewswire/ --Stealth BioTherapeutics (Stealth), a clinical-stage biopharmaceutical company developing therapeutics to treat mitochondrial dysfunction, today announced the initiation of TAZPOWER, a Phase 2/3 study evaluating elamipretide in patients with Barth syndrome. Barth syndrome is a rare genetic mitochondrial disease, caused by mutations in the TAZ gene, and characterized by cardiac abnormalities, skeletal muscle weakness, recurrent infections and delayed growth.

"The severe problems experienced by patients with Barth syndrome are caused by misshapen and dysfunctional mitochondria, which reduce the energy production in the affected tissues. The resulting muscle weakness can lead to severe fatigue, heart failure and death," said Stealth Chief Medical Officer Doug Weaver. "In this study, we hope to show that elamipretide may have clinical benefit by improving function in these affected mitochondria."

TAZPOWER is a randomized, double-blind, placebo-controlled crossover study that will evaluate the effects of daily elamipretide treatment in a minimum of 12 patients with genetically confirmed Barth syndrome. Patients will be randomized to one of two sequence groups: 12 weeks of single daily subcutaneous injections of elamipretide in Treatment Period 1, followed by 12 weeks of treatment with placebo in Treatment Period 2, with a four-week wash-out period between periods, or vice versa. The primary endpoint is change in distance walked during the six-minute walk test. Secondary endpoints include functional assessments, patient-reported outcomes and safety.

"Our understanding of Barth syndrome and how it manifests has evolved significantly, but current treatment efforts are still limited to the management of symptoms," said Hilary Vernon, M.D., Ph.D., assistant professor of Pediatrics at McKusick-Nathans Institute of Genetic Medicine at Johns Hopkins University and the primary investigator for the study. "The initiation of TAZPOWER represents an important milestone in the potential development of a disease-specific treatment option."

TAZPOWER builds upon Stealth BioTherapeutics's existing rare disease and cardiorenal programs, including three ongoing Phase 2 studies in adults with heart failure (IDDEA-HF, PROGRESS-HF, RESTORE-HF).

"This study underscores our commitment to develop elamipretide for the treatment of rare genetic mitochondrial diseases," said Stealth Chief Executive Officer Reenie McCarthy. "The cardiovascular and skeletal muscle symptoms affecting this population share a common thread with symptoms experienced in diseases commonly associated with aging, such as heart failure, in which mitochondrial dysfunction contributes to the clinical pathology."

For additional information on the TAZPOWER study or elamipretide, please refer to Stealth's website.

About Barth Syndrome Barth syndrome is a rare genetic condition characterized by muscle weakness, cardiac abnormalities, recurrent infections and delayed growth. Barth syndrome occurs almost exclusively in males and is estimated to affect one in 200,000 to 400,000 individuals worldwide at birth. There are currently no FDA-approved therapies for the disease.

About Stealth BioTherapeutics We are a privately held clinical-stage biotechnology company focused on the development of therapeutics for diseases involving mitochondrial dysfunction. We believe there is a strong rationale for our lead product candidate,elamipretide, in indications in these diseases based on encouraging preclinical and early clinical data. We are investigating elamipretide in three primary mitochondrial diseases primary mitochondrial myopathy (PMM), Barth syndrome and Leber's hereditary optic neuropathy (LHON) as well as in heart failure, Fuchs' corneal dystrophy and dry age-related macular degeneration.We received Fast Track designation for elamipretide for the treatment of PMM from the FDA in December 2015. We are developing our second product candidate, SBT-20, for central nervous system disorders.Our mission is to be the leader in mitochondrial medicine. To learn more information about us and our pipeline, visitwww.stealthbt.com.

Contacts Media Relations dna Communications Kate Contreras, 617-520-7088 rel="nofollow">Media@StealthBT.com

Investor Relations Stern IR Beth DelGiacco, 212-362-1200 rel="nofollow">IR@StealthBT.com

View original content with multimedia:http://www.prnewswire.com/news-releases/stealth-biotherapeutics-initiates-phase-23-study-of-elamipretide-in-patients-with-barth-syndrome-300492467.html

SOURCE Stealth BioTherapeutics

View post:

Stealth BioTherapeutics Initiates Phase 2/3 Study of Elamipretide in Patients With Barth Syndrome - Markets Insider

Asia Lung Cancer Summit in Hong Kong Targets Precision Medicine – PR Newswire (press release)

Posted on by

The event was held at Hong Kong Science Park and was co-organised by Hong Kong Cancer Therapy Society, the Hong Kong Association of Community Oncologists, the Hong Kong Society of Clinical Oncology, and supported by Hong Kong Science and Technology Parks Corporation and AstraZeneca. The Scientific Collaboration Partner of the conference is Sanomics Ltd., a start-up in Hong Kong Science Park specialising in blood-based genomics technologies for cancer patients.

Lung cancer is the leading cause of cancer deaths worldwide, claiming more than 1.6 million lives each year-- more than breast, colon and prostate cancer combined. Not only smokers get lung cancer but about 51% of the world's lung cancer cases occur in Asia[1], while it accounts for 21% of cancer deaths in the region[2]. In addition to the current treatment approaches precision medication uses DNA analysis for early detection of cancer and to identify therapies that are tailored for individual patients has become an emerging added solution in the cure of lung cancer.

The well-timed conference drew experts in Asia to discuss and examine the progress of high quality biomarker testing in the region. It also put heavy emphasis on the need for collaboration and why Hong Kong, with its expertise in precision medicine, genome analysis and nano-technology in the field of cancer management, is a natural regional hub for research, development, clinical application and investments in this field.

Genomics biomarkers essential for personalised cancer management

Mrs. Fanny Law, Chairperson of Hong Kong Science and Technology Parks Corporation, in an opening address at the conference, said advances in precision medicine have transformed healthcare and treatment of diseases. Predictive diagnosis and personalised treatment tailored to each person's genetic makeup and the genetic profile of the tumour can enhance efficacy and minimise adverse effects.

"Precision medicine faces many challenges in clinical application and this requires collaboration between all stakeholders," said Mrs. Law. "The therapeutic prospects of precision medicine are enticing, spurring enterprising researches around the world. Today genetic testing is available for over 2,000 clinical conditions and the number of available diagnostic test is increasing exponentially."

Mr. Tony Yung, Chief Executive of Sanomics, said: "Our work on precision medicine effectively supports the decision-making of oncologists based on information about genetic alternations in tumour DNA. Once clinically significant genetic alternations are identified, specific treatments can be aimed at the tumours. These have a much higher chance of achieving desirable clinical outcomes."

Hong Kong has a key role in research and dissemination of knowledge on cancer

"The uniqueness of Sanomics' approach is making use of bodily fluids, such as blood, to screen for genetic alternations when tumour tissues are not available. We call it "liquid biopsy", which is faster, less risky, more convenient and more practical in the clinical setting. We are now building Asia's first hub for liquid biopsy here in Hong Kong, and will lead the region in the use of genomics for personalized management of cancer," said Mr. Yung. "The breakthroughs we achieved not only exemplify the research and development excellence of Hong Kong's biomedical experts but also our strengths to identify and resolve challenges for the Asia healthcare sector."

Hong Kong Science Park has more than 80 biotechnology companies with business scope that spreads across medical devices, research on stem cells, genomics and regenerative medicine, molecular diagnosis, as well as R&D on Chinese and Western medicines. All of these, in their differing ways, contribute to an ecosystem that's highly conducive to research. Importantly, clinical data from Hong Kong is recognised by the China Food and Drug Administration (CFDA) for registration and approval purposes, making it an ideal testing ground for emerging therapies and devices.

"A year ago,the CFDAaccredited two phase 1 clinical trial centres in Hong Kong andaccepted clinical trial data from Hong Kong for registration and approval purposes," said Mrs. Law. "Looking to the future, we must all seize the opportunities arising from the Hong Kong Shenzhen Innovation Technology Park along the border between the two cities and the Guangdong-Hong Kong-Macau Greater Bay Area to enlarge Hong Kong's capacity for biomedical research and development to meet the growing demand for better health care for the ageing population both in Hong Kong and Mainland China."

"Our aspiration is for the Greater Bay Area to be an innovation hub with global impact. And within this area, the Hong Kong-Shenzhen Innovation and Technology Park will be a powerhouse of research where great minds from Mainland China, Hong Kong and the rest of the world meet and work together," continued Mrs Law.

Besides Mrs. Law and Mr. Yung, other keynote speakers at the conference included: Prof. Tony Mok, Chairman, Department of Clinical Oncology at the Chinese University of Hong Kong; Mr. Leon Wang, EVP, International & China President of AstraZeneca (China); and Prof. Yilong Wu, Director of Guangdong Lung Cancer Institute and Vice-president of Guangdong General Hospital (China).

[1] Source: World Cancer Report 2014.Steward, Bernard and Wild, Christopher (eds). (2014)

[2] Source: World Health Organization. Globocan 2012: Estimated Cancer Incidence, Mortality and Prevalence Worldwide in 2012 (2015): http://globocan.iarc.fr/Pages/fact_sheets_cancer.aspx.

About Hong Kong Science and Technology Parks Corporation

Comprising Science Park, InnoCentre and Industrial Estates, Hong Kong Science & Technology Parks Corporation (HKSTP) is a statutory body dedicated to building a vibrant innovation and technology ecosystem to connect stakeholders, nurture technology talents, facilitate collaboration, and catalyse innovations to deliver social and economic benefits to Hong Kong and the region.

Established in May 2001, HKSTP has been driving the development of Hong Kong into a regional hub for innovation and growth in several focused clusters including Electronics, Information & Communications Technology, Green Technology, Biomedical Technology, Materials and Precision Engineering. We enable science and technology companies to nurture ideas, innovate and grow, supported by our R&D facilities, infrastructure, and market-led laboratories and technical centres with professional support services. We also offer value added services and comprehensive incubation programmes for technology start-ups to accelerate their growth.

Technology businesses benefit from our specialised services and infrastructure at Science Park for applied research and product development; enterprises can find creative design support at InnoCentre; while skill-intensive businesses are served by our three industrial estates at Tai Po, Tseung Kwan O and Yuen Long.

More information about HKSTP is available at http://www.hkstp.org.

View original content with multimedia:http://www.prnewswire.com/news-releases/asia-lung-cancer-summit-in-hong-kong-targets-precision-medicine-300493458.html

SOURCE Hong Kong Science and Technology Parks Corporation

http://www.hkstp.org

See the rest here:

Asia Lung Cancer Summit in Hong Kong Targets Precision Medicine - PR Newswire (press release)

Patient-Centered Vs. Lab-Centered ‘Personalized Medicine’ – HuffPost

Posted on by

It is more important to know the patient who has the disease than the disease the patient has. Hippocrates

Personalized medicine was invented 2500 years ago when Hippocrates put the patient at the center of medical care- not the gods or the practitioner or the treatment.

To the detriment of patients, Hippocrates brand of patient centered, personalized medicine has been eroded by 150 years of increasingly high tech medicine. Sadly, the laboratory tests, the technical tools, the medical record, the billing system, and the computer are now too often at the center of medical care- all at the expense of the crucial healing that has always come from the doctor/patient relationship.

The modern and misleading use of the term Personalized Medicine began 20 years ago as an outgrowth of the enormous enthusiasm engendered by the human genome project.

The concept was grand. Rather than diagnose and treat people based on very broad and nonspecific features (like shared symptoms or signs), perhaps doctors could use the powerful new genetic tests to determine which specific genes were causative of each individuals disease and then fashion a treatment specific to each persons underlying genetic defect.

There have already been a few dramatic successes of this modern form of personalized medicine, and hopefully with time there will eventually be many more.

But scientifically informed personalized medicine mostly remains a distant hope for the future and has instead become a crude form of marketing hype in the present.

Hospitals, drug companies, doctors, the National Institute of Health, even President Obama have used the misleading term personalized medicine as a branding advertising tool to sell medical services and to gain support for enhanced research funding.

Extravagant promises are made that most certainly cannot possibly be kept. The sell is that magical cures for a whole variety of diseases are just around the corner- especially now that we have the extremely powerful tool of gene editing.

The reality is much more uncertain and difficult. Most diseases have remarkably complex genetic roots involving hundreds of genes, each making tiny and complexly interacting contributions. There are few simple genetic targets; there will be few magical cures. Gene editing will likely be helpful only for the relatively few diseases that have simple causation.

Surely, we should march on with sophisticated research to find them, but mustnt be so dazzled by the potential of the science of medicine that we lose the magic that has always come from its art and humanity.

Nicholas Capozzoli, a neurologist, is the wisest and most humane doctor I know. He will help us recapture the simple things, now so often lost, that have made Hippocratic medicine so effective for so long.

Dr. Capozzoli writes: The once-glorious doctor/patient relationship is being cheapened into a commercial contract- like buying a car or filling out tax forms with an accountant. In my view, the tie between doctor and patient should be a powerful relationship of trust, empathy, and healing.

Personalizing medicine by focusing on the patient is an extension of the ancient and time honored tradition of hospitality. The magic of healing in the doctor-patient relationship begins with the first phone call and continues through every contact.

Your patient should be welcomed like an honored guest, not made to feel like an annoying intruder. Greeting someone in the waiting room and escorting them back to your office seems to be a lost art, but should be as natural as greeting people at the door of your home when they arrive for dinner.

Similarly, apologizing for being late is a simple courtesy that should not be forgotten just because the meeting occurs in a professional setting.

While these gestures may seem small, patients often tell me how surprising and comforting they are. They are much more likely to engage in the intimate conversation that informs medical diagnosis when they feel at home with the doctor.

The onerous demands of protocol-driven medicine and electronic medical records have made the filling out of checklists more important to many doctors than really getting to know the person and understanding the context surrounding his symptoms.

Casual conversation is intrinsic to all human relationships and essential in setting the stage for meaningful history taking. The physician paying close attention to the little details can make an accurate differential diagnosis that usually obviates the need for frequent, expensive, and unnecessary fishing-expeditions of extensive laboratory testing that often turn out to do much more harm than good.

Call me old-fashioned, but referring to patients as healthcare consumers and physicians as healthcare providers degrades their relationship and under-estimates the healing it provides.

I take my own vital signs. Patients are surprised by this and tell me it indicates my interest in them.

I take my notes on a yellow pad and only enter whats required for electronic medical records after the patient has left the office. Patients say that other doctors are so focused on the computer screen, they never once make eye contact.

Ushering people into a metallic 8 x 8 room and asking them to undress before the physician even enters seems to be an awkward and humiliating way to start a relationship. My examining table is in my office where I sit down with them for the first time. My office is folksy and filled with conversation pieces. My home becomes temporarily their home.

Arguments that this sort of personalized medicine does not take into account the current business demands of modern medicine simply do not hold water. The quality of the doctor-patient relationship is not identical with the time spent, but rather the way it is spent.

Patients also tell me of rude staff, long waits in the waiting room, and then long waits in the examining area before the physician comes in. This sabotages the doctor-patient relationship and creates a terrible context for care and cure. Administrative handicaps to care can and must be eliminated with careful selection of personnel, ongoing training, and close attention to effective office or clinic management practices. The patients convenience should take priority over staff convenience.

A strong doctor-patient relationship is essential to good diagnosis, to good treatment decisions, to carrying out the treatment plan, and is the royal road toward patient confidence, comfort, and healing. As physicians, we are allowed into a sacred space and must honor our obligation to personalize and humanize all our contacts with patients.

Thank you Dr. Capozzoli for your precious advice on recapturing old fashioned personalized medicine- how best to form a healing doctor/patient relationship.

Which brings us back to the current spate of fake claims for a scientific personalized medicine. Some researchers and leaders of major cancer centers have made the ridiculous promise that all cancers will be cured in 10-20 years. The advertising pitches made by some medical providers are even more outrageous- they make it sound as if personalized medicine has already arrived at their particular hospital.

There has always been a tendency for medicine to overpromise and under deliver. Often enough the treatments offered are more harmful than the diseases treated.

Thats why the Hippocratic First do no harm is the most important commandment in medicine. The second is: Cure sometimes, treat often, comfort always. We shouldnt neglect the bedside art of medicine as we become overly enamored by its laboratory science.

See the original post here:

Patient-Centered Vs. Lab-Centered 'Personalized Medicine' - HuffPost

Geisinger wins grant for research – Sunbury Daily Item

Posted on by

DANVILLE Geisingers Autism & Developmental Medicine Institute (ADMI) has been awarded a one-year, $150,000 grant by the Simons Foundation Powering Autism Research for Knowledge (SPARK) to help advance research into autism.

ADMI will be a center for recruitment and sample collection for a new genetic study that aims to enroll 50,000 individuals with autism and their families nationwide, making it the most ambitious study of the conditions genetics to date.

The one-year grant to ADMI is renewable for a full, three-year term. SPARK is supported by the Simons Foundations Autism Research Initiative.

Approximately 50 genes have been identified that almost certainly play a role in autism, and researchers estimate that at least an additional 300 are involved. But to identify all the genes at play, many more genetic samples are needed from those with autism and their immediate families.

The national autism research project aims to make important progress possible by pooling together tens of thousands of participants for research.

As autism is a spectrum, researchers need many people with autism to participate in all types of research. Until now, only a small number of individuals and families affected by autism have ever participated in research. SPARK wants to invite the entire autism community to dramatically expand its participation.

The medical and genetic data generated from the program will power important new research that aims to advance the understanding of autism and equally provides meaningful information and resources to participants.

As a SPARK site, Geisingers ADMI joins other renowned national autism centers collaborating on this important project, including, for example, Boston Childrens Hospital, Childrens Hospital of Philadelphia, and the UCLA Center for Autism.

If you, or a family member, are affected by autism and want to participate in this vital national research, you may contact Kate Dent at 570-522-9402 or emailkadent1@geisinger.edu.

Geisingers ADMI is led by nationally known expert Christa Lese Martin, Ph.D., and includes many staff working on the cutting edge of research in developmental disorders. In addition, ADMI has a team of physicians, psychologists, genetic counselors, and speech language pathologists to help fulfill its clinical mission.

ADMI works to integrate the diagnosis of children with autism and other developmental disorders with evidence-based interventions, research and training.

For more information on SPARK, see https://sparkforautism.org

View original post here:

Geisinger wins grant for research - Sunbury Daily Item

New Gene Therapy Produces Hope for a New Era in Cancer … – Healthline

Posted on by

Gene-altering therapy on the cusp of FDA approval may soon be treating children with advanced leukemia.

The Food and Drug Administration (FDA) is on the verge of approving the first-ever gene therapy treatment for use in the United States.

If it goes through as expected, the therapy will be used to treat children and young adults with advanced acute lymphoblastic leukemia (ALL).

The FDA's Oncologic Drugs Advisory Committee earlier this month recommended that the agency approve Novartis experimental chimeric antigen receptor (CAR-T) therapy, CTL019 (tisagenlecleucel).

Its an individualized therapy in which a persons T cells are removed from their blood and reengineered to fight cancer. Then theyre infused back into the patient.

Thats why its called a living drug.

In a recent clinical trial, 83 percent of patients experienced complete remission or complete remission with incomplete blood count recovery within three months.

The FDA is expected to make a decision in September. The agency usually follows its committees recommendations.

Experts are optimistic about the potential of CAR-T.

Dr. Santosh Kesari is a neuro-oncologist and chair of the Department of Translational Neuro-oncology and Neurotherapeutics at the John Wayne Cancer Institute at Providence Saint John's Health Center in California.

Kesari told Healthline that if approved, the therapy will be a revolutionary breakthrough in cancer treatment.

It will be the first application of this type combining gene therapy and immune therapy to modify a patients own cells to go attack cancer cells, he said.

Kesari explained that this application could work in other cancers where theres a specific target.

He pointed to a City of Hope case study involving a 50-year-old man with recurrent multifocal glioblastoma, a type of brain cancer.

The treatment was part of a phase I clinical trial to test the safety of CAR-T therapy when delivered directly to brain tumors. A successful response was sustained for more than seven months, longer than would usually be expected.

So, there is potential for application in solid tumors if we identify the right marker, making sure we manage side effects, said Kesari.

In an email interview with Healthline, Dr. Swati Sikaria, medical oncologist from Torrance Memorial Medical Center in California, explained that the goal is to choose a target as unique to the cancer as possible while avoiding damage to the noncancerous cells of the body.

This initial success paves the way for creating CAR-T cells with targets for other malignancies, she explained.

Whether the success in ALL can be replicated in other types of cancers, Im cautiously optimistic. Well have to see what ongoing and future clinical trials show. The most notable progress has been in multiple myeloma as well as glioblastoma, the type of tumor Senator [John] McCain was recently diagnosed with, said Sikaria.

In a review article published last year, Sikaria wrote that adult B-acute lymphoblastic leukemia (B-ALL) doesnt share the favorable prognosis seen in pediatric patients with the same disease.

Less than 50 percent of patients experience long-term survival, and for those adults over age 60, long-term survival is only 10 percent. At time of relapse, five-year prognosis is a dismal 7 percent. Novel and less toxic agents are urgently needed, she continued.

Sikaria called CAR-T cells a highly promising new agent, even in patients who are heavily pretreated.

Dr. Samantha Jaglowski is a hematologist at The Ohio State University Comprehensive Cancer Center. She specializes in stem cell transplants for patients with lymphoma and chronic lymphocytic leukemia.

While she hopes this truly is a breakthrough, she told Healthline shes hesitant to declare victory too soon.

It certainly appears promising. I sincerely hope it meets the expectations being put forth, said Jaglowski.

Its an exciting thing to be involved with. There are already many studies in the pipeline for many other kinds of cancers, she said.

There are two main concerns about CAR-T.

The first is the potential for serious side effects.

One of these is a life-threatening reaction called cytokine release syndrome (CRS).

Sikaria said this reaction is common and can occur within hours of the CAR-T cell infusion. But it can be effectively treated.

The drug also commonly causes reversible neurologic symptoms and a drop in blood counts, which can lead to infection. We have to remember that patients receiving CAR-T cells are battling a disease almost certain to be fatal otherwise, so I do think the risks are outweighed by benefits, she explained.

Jaglowski agreed that CRS is usually manageable. She noted that there havent been enough patients yet to evaluate long-term effects.

This will not be a first-line therapy until theres more data behind it. Patients will have had to have a couple of lines of therapy before becoming eligible for this. Im a lymphoma physician. In lymphoma trials, they require failure of previous lines of therapy. Its for people who are further on in the disease course and who have fewer options, said Jaglowski.

The second major concern is the potential cost. Novartis hasnt put a price tag on it, but industry analysts project that it could hit $500,000 per infusion.

Hopefully, well see competition in the market from other companies CAR-T cells, as a number are in development. Some help from Washington is also needed to bring the cost down, said Sikaria.

Kesari compared the therapy with other treatments.

Some drugs we [already] use, especially biologics, cost anywhere from $5,000 to $20,000 a month and involve repeated treatments in a year. Some cost several hundred thousand dollars a year. This is a one-time or few-time treatment. Its not like youre getting treatment for years. It puts all the costs upfront. If you get cured, whos to argue that its not worth it? he said.

Sikaria said that unless theres any new information concerning treatment-related deaths between now and October, the FDA should approve it.

CAR-T cells are exciting and have high response rates, but we need to see how long-lasting these results are as we continue to shoot for a cure. For now, bone marrow transplant is still an important part of the management of a patient with ALL, said Sikaria.

Kesari believes the FDA will approve CAR-T due to an unmet need and lack of options for a disease where patients uniformly end up dying.

This technology of how to modify genes started in the 80s and 90s. Building on that, we learned about the immune system and how to modify it to kill cancers. Its taken so long to prove value out to the point where were getting a drug approval. But its been going on for decades, he continued.

I commend the people that did this and to make it work in humans thats amazing, said Kesari.

Originally posted here:

New Gene Therapy Produces Hope for a New Era in Cancer ... - Healthline

4D Molecular Therapeutics and Foundation Fighting Blindness … – Business Wire (press release)

Posted on by

EMERYVILLE, Calif. & COLUMBIA, Md.--(BUSINESS WIRE)--4D Molecular Therapeutics (4DMT), a leader in adeno-associated virus (AAV) gene therapy vector discovery and product development, and the Foundation Fighting Blindness (FFB), the worlds largest non-governmental source of research funding for inherited retinal degenerations (IRD) and dry age-related macular degeneration (AMD), today announced a partnership to develop intravitreal gene therapeutics for patients with these blinding conditions using 4DMT-proprietary AAV vectors. Under the terms of the agreement, 4DMT will provide access to its vector technology, development expertise and manufacturing capabilities while FFB will identify potential academic and business collaborators, provide drug development expertise and fund approved projects to develop transformative gene therapy products. 4DMT retains all patent and commercial rights to its 4DMT proprietary AAV vector variants. FFB and 4DMT will jointly review and approve all programs initiated within this collaboration.

New vector technologies are critical to the successful use of gene therapies for IRDs in order to improve targeting to affected cells within the retina, and to maximize efficacy and safety. Vectors that can be delivered intravitreally would simplify the procedures used for treatment and reduce the costs of administration.

Affecting approximately 200,000 patients in the US, inherited retinal diseases (IRDs) are a major cause of adult and childhood blindness. Mutations in more than 260 genes are known to cause these rare, orphan conditions for which there are currently no approved therapies. Gene therapy holds tremendous promise for the treatment of these conditions by introducing genes to the retina that may be able to replace the lost or dysfunctional genes, correct underlying mutations, or deliver therapeutically-active genes that can prevent cell loss and degeneration.

We are very impressed with 4Ds vector evolution approach, the companys product pipeline and manufacturing expertise. The potential is great for developing a number of gene therapeutics that could treat those affected by retinitis pigmentosa and allied conditions using a simple intravitreal injection approach, said Patricia Zilliox PhD, Chief Drug Development Officer at the FFB.

We are extremely excited by this collaboration with FFB, a globally-recognized leader in the effort to cure blindness due to inherited retinal degenerations. FFB has tremendous expertise identifying the best retinal research as well as an outstanding network of funded investigators and companies with whom we hope to collaborate to develop a portfolio of products that will benefit those affected with retinal degenerative diseases, said David Kirn, MD, co-Founder and CEO of 4DMT.

About Foundation Fighting Blindness (FFB)

Since FFBwas established in 1971 it has raised more than $700 million toward its mission of preventing, treating and curing blindness caused by inherited retinal diseases. In excess of 10 million Americans, and millions more worldwide, experience vision loss due to retinal degenerations. Through its support of focused and innovative science, and by teaming with industry, FFB drives the research that has and will continue to provide treatments and cures for people affected by retinitis pigmentosa, macular degeneration, Usher syndrome and other inherited retinal diseases.

About 4D Molecular Therapeutics (4DMT)

4DMT is focused on the discovery and development of targeted and proprietary AAV gene therapy vectors and therapeutic products for use in patients with severe genetic diseases with high unmet medical need. Our robust discovery platform, termedTherapeutic Vector Evolution, empowers us to create customized gene delivery vehicles to deliver genes to any tissue or organ in the body, by optimal clinical routes of administration with resistance to pre-existing antibodies. These proprietary and targeted products allow us to treat both rare genetic diseases and complex large market diseases. 4DMT is creating a diverse and deep product pipeline through its own internal 4D products, as well as partnered programs. 4DMT partners include: Pfizer (PFE), Roche (SIX: ROG; OTCQX: RHHBY), uniQure (QURE), AGTC, Benitec, Cystic Fibrosis Foundation and Choroideremia Research Foundation.

About 4DMTs Therapeutic Vector Evolution

Current clinical stage gene therapy products are generally based on one of 10 AAV vectors that are wild-type or primitive vectors, meaning they were found in nature as laboratory contaminants or as monkey infections. These first-generation AAV vectors, while generally safe and well-tolerated in patients, do not have optimized delivery properties and often require aggressive and/or invasive dosing to attempt the desired transduction of target cells. 4DMT is advancing the field of AAV vector technology by deploying principles of evolution and natural selection to create vectors that efficiently and selectively target the desired cells within the diseased human organ via clinically optimal routes of administration with resistance to pre-existing antibodies in the population. Our Therapeutic Vector Evolutionplatform deploys approximately 100 million unique AAV variants from proprietary 4DMT AAV libraries with extensive diversity. 4DMT then applies proprietary methods to identify lead vectors that are highly optimized for a specific target cell and organ, route of therapeutic administration, and capacity to evade pre-existing antibodies in patients. The result is a customized, novel, and proprietary pharmaceutical-grade vector uniquely designed for therapeutic gene delivery and efficacy in humans.

Visit link:

4D Molecular Therapeutics and Foundation Fighting Blindness ... - Business Wire (press release)

Orchard’s gene therapy for rare ‘bubble baby’ disease gets FDA fast … – Labiotech.eu (blog)

Posted on by

Orchard Therapeutics has received rare pediatric disease designation for OTL-101, agene therapy for the treatment of the rare disease ADA-SCID.

Orchard Therapeutics,launched just a year ago in London, is developing what could be a new version of GSKsStrimvelis, the second gene therapy to reach the European market and theonly after uniQures Glybera was withdrawn from the market after being used only by one patient.

Orchards gene therapy, OTL-101, consists of a procedure in which hematopoietic stem cells from the patient are harvested, genetically engineered outside of the body and reinfused. It is intended as a cure foradenosine deaminase severe combined immunodeficiency(ADA-SCID), a rare disease affecting only 350 births per year. Without treatment, children die before their second birthday. In the past, children like David Vetter lived in sterile chambers to protect them, giving it the name of bubble babydisease.

David Vetter, with ADA-SCID, lived until age 12 thanks to sterilized suits and rooms

OTL-101 has been tested in 40 patients so far, all of which have survived since the start of a Phase I/IItrial last December. And despite the low numbers of patients, Orchard seems determined to compete with GSK in the ADA-SCID market. Currently, the only option for children in the US with this disease is enzyme replacement therapy, which still leaves them at high risk of life-threatening infections.

GSK got EU approval for Strimvelis in May 2016, and has since treated only one patient. The price tag, 594,000refundable if the therapy does not work could account for the slow adoption. It remains to see whether the number of patients will rise or if Strimvelis is doomed to a similar fate to that of Glybera.

Strimvelis is currently available to children across the EU but is only performed in the Ospedale San Raffaele in Milan. GSK has mentioned plans to file for FDA approval but it might wait a while given regulations are quite different between Europe and the US for the approval of gene therapies. In fact, no gene therapies are currently approved in the US.Spark Therapeutics might bring the first one this year, against LCA, a form of genetic blindness.

Image via ustas7777777 / Shutterstock; NASA Johnson Space Center / Creative Commons

See the original post here:

Orchard's gene therapy for rare 'bubble baby' disease gets FDA fast ... - Labiotech.eu (blog)