Category Archives: Stem Cell Medical Center

For veterans, theres no better cause to push than helping other vets – Snoqualmie Valley Record

Two veterans in East King County are just two of many working to help other veterans.

North Bend veteran Jim Curtis doesnt mince words. If he supports something, youll know it.

The same can be said about things hes not a fan of. Hes vocal about his disdain for President Donald Trump and his actions while in office. And politicians, in general, who use veterans as a means for election.

Politicians use us to get elected or re-elected, Curtis said. But after they accomplish that goal, its sayonara until its time for campaigning again.

He channels his ideas through poetry. Mostly, he pours the passion into helping other veterans.

The cause that had his attention on Oct. 24 was this years Mother Brundage Memorial Raffle. He was raising funds for the family of Andrew Yoder, a U.S. Marine Corps combat veteran who was killed when a crane crashed down in Seattle last spring. The raffle would happen a few weeks later, on Nov. 10, the Marine Corps birthday.

Curtis sold tickets for $5 at the North Bend QFC and Mount Si Sports and Fitness. Even as he traveled south to the American Lake VA Medical Center in his red, 2011 Ford Ranger truck, he pushed for others to purchase raffle tickets.

When he approached the check-in desk, after briefly responding to a question prompted by the man at the counter, he popped his own.

Were doing this raffle, he often began his pitch. Curtis would often end his interactions with the words Semper Fi, the Marine Corps motto and short for semper fidelis a Latin phrase that translates to mean always faithful.

Semper Fi, hed say before leaving.

He used similar words each time, having memorized the phrases he echoed to try and convince the listener on the importance of the raffle. And, of course, to shell out some cash to help.

But what could one expect? When it came to serving especially when Curtis did, during the highly-controversial Vietnam War it was other veterans who understood. And it was other veterans who offered their support. Even if that support meant hanging flyers in a VA hospital nearly 55 miles south from Curtiss North Bend home. Or when it meant reaching out to politicians again. And again. And again.

Other causes

Before he began fundraising for Yoders family, Curtis was busy commuting south to Olympia, where he testified before state House and Senate committees. He thought Purple Heart license plates, those given to wounded military or family of those killed in action, should be issued free of payment of any fees or taxes. He had his own Purple Heart, after shrapnel from a mortar blast flew into his back, legs and derriere. This happened in 1969, during his first year in Vietnam, where he was a machine gunner.

Curtis presented the idea to Democrat Paul Graves, at the time house representative for District 5. When Lisa Callan was elected into the seat in November 2018, after defeating Graves by more than 3,000 votes, she approached him on his idea. She had met him previously while doorbelling in the area.

(The change) was the very least of what we could do to say Thank you, Callan said by phone, on Veterans Day. It gives veterans a way to share their story by even having that plate We need to do so much more for those who receive the Purple Heart and their families.

Curtis has other causes now. Hes invited Rep. Callan to visit the VA hospital he visits regularly, to see the condition of the buildings and to hopefully get some kind of change. The facilities are in a degraded state, compared to the state of medical buildings elsewhere, Curtis said. The walls have dark scuff marks, in some spots reaching about a foot in height. And for veterans, who sacrificed their time and had their lives on the line, it just wasnt up to par. Callan hasnt yet paid a visit to the hospital, but said that was in the works.

His other effort entails getting Gregory Pappy Boyingtons name recognized somewhere along the Medal of Honor Memorial Highway. Boyington was a graduate of Lincoln High School in Tacoma and an American combat pilot.He received the honor, the highest and most prestigious, after he spent months shy of two years in a Japanese prison camp.

There are plaques honoring vets there, but no Pappy in sight, Curtis said.

For another veteran, Curtis is simply looking for answers on why stem cell therapy is given to active-duty military at the Walter Reed National Military Medical Center in Bethesda, Maryland, and not an option for a disabled veteran Curtis knows in North Bend. The center serves military beneficiaries in the Washington, D.C. area as well as those from across the country and around the globe, according to its website.

He has contacted Congresswoman Kim Schrier about the issue, but has so far been having back-and-forth conversations with her staff via email, staff who, Curtis said, say shes too busy to leave her office. He figured the doctor he knocked on doors for, who at some point worked at Virginia Mason Medical Center in Issaquah, would be willing to make the drive, considering its not that far a commute, he said. Schrier did not return a request for comment by press deadline.

Gorman

But its not only Curtis helping other veterans. Marine veteran and Redmond resident Mark Gorman has been working to locate safe spaces for veterans without homes to sleep. He started the effort in 2004, when he owned a shop in Kingston. Everyday he spotted an older man fishing. And after that first initial contact, he struck up a friendship with the homeless man.

At one point, he just wasnt there fishing anymore, Gorman said. Three and four weeks went by, and Gorman decided to find the mans hide the safe spot he had carved out for himself outdoors. This guys all alone, no family. He died on his own of malnutrition and his heart gave up.

In the U.S., on a single night in January 2018, there were more than 37,800 veterans experiencing homelessness, according to the U.S. Department of Veteran Affairs. Of that number on that same night, about 23,300 were living unsheltered. Although there was a 5.4 percent decrease from the previous year, in the estimated number derived from the point-in-time count, Gorman said theres still a need.

When he moved to the Eastside in 2013, he realized there was an even bigger need locally. He began to help veterans, who for different, complex reasons, had lost their housing. Some he could help. Others, he said, werent mentally ready for the world.

They camp out in tents, others in trailers, some sleep in cars in church parking lots. Others sleep in Gormans backyard.

Three things in life we do as veterans sacrifice, duty and selflessness, Gorman said of his reasoning for helping. If those three things are not instilled in a boy as a young man, he will never become a real man. A man sacrifices for not only family and self but also for others around him.

Someday

While at the VA that cloudy afternoon in October, Curtis struck up a conversation with a gentleman who was also pushing his own cause to help veterans. He had acquired small American flags from another, and pinned one to Curtiss lapel, on the opposite side of where his Purple Heart medal pin sat. In exchange, Curtis offered a poem he wrote for sergeant first class Nathan Chapman, the first soldier killed during the war in Afghanistan. He was from Puyallup.

Someday well talk about this war, just like those weve had before, was one line he recited in the waiting room. And the spectator, in response, cried. The two shook hands and bonded over the shared experience of serving the country.

Curtis turned to walk away, but not before saying Semper Fi.

After a quick chat in the waiting room of the American Lake VA Medical Center, Jim Curtis, right, is given a small American flag pin by a fellow veteran. Curtis was there drawing attention to his fundraising efforts. Staff photos / Ashley Hiruko

A sign hangs on the bulletin board at the VA medical center for the Ocean Warriors Birthday Party. Curtis hung others, like these, in areas with many eyes to see.

On a foggy October morning, Curtis drives south toward the Tacoma area. He had his flyers in hand, and raffle tickets to sell. They would benefit the family of Andrew Yoder, a Marine Veteran who died in Seattle during the crane collapse this spring. Staff photo/Ashley Hiruko

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For veterans, theres no better cause to push than helping other vets - Snoqualmie Valley Record

Tailored T-cells safe, may be effective in preventing and treating multiple viral infections – News-Medical.net

Tailored T-cells specially designed to combat a half dozen viruses are safe and may be effective in preventing and treating multiple viral infections, according to research led by Children's National Hospital faculty.

Catherine Bollard, M.B.Ch.B., M.D., director of the Center for Cancer and Immunology Research at Children's National and the study's senior author, presented the teams' findings Nov. 8, 2019, during a second-annual symposium jointly held by Children's National and the National Institute of Allergy and Infectious Diseases (NIAID), part of the National Institutes of Health (NIH). Children's National and NIAID formed a research partnership in 2017 to develop and conduct collaborative clinical research studies focused on young children with allergic, immunologic, infectious and inflammatory diseases. Each year, they co-host a symposium to exchange their latest research findings.

According to the NIH, more than 200 forms of primary immune deficiency diseases impact about 500,000 people in the U.S. These rare, genetic diseases so impair the person's immune system that they experience repeated and sometimes rare infections that can be life threatening. After a hematopoietic stem cell transplantation, brand new stem cells can rebuild the person's missing or impaired immune system. However, during the window in which the immune system rebuilds, patients can be vulnerable to a host of viral infections.

Because viral infections can be controlled by T-cells, the body's infection-fighting white blood cells, the Children's National first-in-humans Phase 1 dose escalation trial aimed to determine the safety of T-cells with antiviral activity against a half dozen opportunistic viruses: adenovirus, BK virus, cytomegalovirus (CMV), Epstein-Barr virus (EBV), Human Herpesvirus 6 and human parainfluenza-3 (HPIV3).

Eight patients received the hexa-valent, virus-specific T-cells after their stem cell transplants:

Two additional patients received the T-cell treatments under expanded access for emergency treatment, one for disseminated adenoviremia and the other for HPIV3 pneumonia. While these critically ill patients had partial clinical improvement, they were being treated with steroids which may have dampened their antiviral responses.

These preliminary results show that hexaviral-specific, virus-specific T-cells are safe and may be effective in preventing and treating multiple viral infections. Of note, enzyme-linked immune absorbent spot assays showed evidence of antiviral T-cell activity by three months post infusion in three of four patients who could be evaluated and expansion was detectable in two patients."

Michael Keller, M.D., pediatric immunologist at Children's National and the lead study author

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Tailored T-cells safe, may be effective in preventing and treating multiple viral infections - News-Medical.net

What to Know in Washington: Trump Ally in Impeachment Spotlight – Bloomberg Government

After weeks of Republican complaints that the Democrats impeachment inquiry relied on secondhand information, the centerpiece of this weeks public hearings is testimony from a man with a direct line to President Donald Trump.

The political peril for Trump, who was dealt a series of setbacks last week, will be heightened as the House investigation accelerates with three days of public hearings starting tomorrow.

No witness is more central than Gordon Sondland, the U.S. ambassador to the European Union, a Trump donor and a confederate with Rudy Giuliani in back-channel diplomatic efforts for the president in Ukraine.

Sondland, scheduled to testify Wednesday, has already amended his previous closed-door testimony once because of discrepancies with other witnesses. And now there will be new questions for him to answer about Trumps pressure on the government in Kyiv to launch a probe entangling former Vice President Joe Biden and other political foes of the president.

David Holmes, a member of the embassy staff in Kyiv, came forward last week to tell impeachment investigators that following a phone conversation between Sondland and Trump, the EU envoy told him the president didnt give a s about Ukraine and that the president only cares about the big stuff that benefits him like the Biden investigation that Giuliani, Trumps personal lawyer, was promoting.

Testimony from Tuesday through Thursday will come from a disparate cast of witnesses, some of whom could prove pivotal to the impeachment inquiry, including officials from the State Department, the White House national security teams, and Vice President Mike Pences office. Read more from Ryan Teague Beckwith and Billy House.

Former Adviser SaysAid Was Tied to Probe: A former top White House adviser told House impeachment investigators Ukrainians were advised Sept. 1 that U.S. military aid was being withheld until their president announced an investigation of a company that had hired Bidens son, Hunter. Tim Morrison, a former senior director of European and Russian affairs at the National Security Council, said Sondland told him how he had informed a high-ranking Ukrainian official that release of $400 million in aid was being linked to the investigations, according to a transcript of his closed-door testimony released Saturday.

The House committee also released testimony from Jennifer Williams, an aide to Vice President Mike Pence, who said she found some of the discussion on the July 25 call between the two leaders to be unusual and inappropriate. Read more from Billy House.

Funding Deadline: Lawmakers are facing a deadline of Thursday to pass legislation to continue funding the government, before they depart for a recess through the week of Thanksgiving. House and Senate leadership have expressed optimism that both chambers will pass a measure ahead of the deadline, though no continuing resolution has been introduced as of this morning and the House schedule offered few details of the timing of a vote.

I hope we pass the CR, House Majority Leader Steny Hoyer (D-Md.) said on the floor Friday. I hope its as clean as it can be. The measure will contain some usual anomalies, noncontroversial changes in funding levels or legislative language, Hoyer said.

FDA Pick to Face Questions on Frankenfish, Stem Cells, Vaping: Trumps selection to be the next head of the FDA will face a barrage of questions this week about his views on vaping, oversight of stem-cell therapies and even Frankenfish. Stephen Hahn, chief medical executive of the prestigious MD Anderson Cancer Center at the University of Texas in Houston, will go before the Senate Health, Education, Labor and Pensions Committee Wednesday for his first public hearing since being nominated to become commissioner of the Food and Drug Administration. Alex Ruoff details what to expect.

Risch Warns Erdogan of Sanctions: Senate Foreign Relations Chairman Jim Risch (R-Idaho) warned that if Turkish President Recep Tayyip Erdogan persists in the deployment of a Russian air defense system, he will move ahead with a sanctions bill. It is his choice, and he knows the consequences, Risch said in a statement on Friday evening. Just a day earlier, Risch had said that the Senate should hold off on sanctions over Turkeys invasion of Syria if Erdogan agreed to give up the S-400 anti-aircraft missile system. Read more from John Harney.

Anti-Robocall Agreement Reached: House and Senate negotiators on Friday reached an agreement on bipartisan legislation to combat illegal robocalls, and anticipate a vote on the measure this year. The House and Senate passed separate legislation earlier this year with broad bipartisan support, and staff has been working since August to reach an agreement on provisions from the two anti-robocall bills. Read more from Rebecca Kern.

Democrats Subpoena EPA Over Formaldehyde Analysis: Rep. Eddie Bernice Johnson (D-Texas), chairwoman of the House Science, Space, and Technology Committee, sent two subpoenas to the EPA Friday over the agencys alleged refusal to provide answers about removing formaldehyde from its review program outlook. Johnson said in an accompanying letter to EPA Administrator Andrew Wheeler that the committee had suffered through eight months of delayed and insufficient responses. The subpoenas were sent to Wheeler and David Dunlap, deputy assistant administrator for the agencys Office of Research and Development. Read more from Stephen Lee.

Democrats Use Super-PACs to Combat Trump: Wealthy Democratic donors are pouring money into outside groups as part of their effort to defeat Trump in 2020, avoiding contributions to a party apparatus that lost in 2016 and to leading candidates who dont want their help. Outside groups aligned with Democrats have pledged to spend more than $300 million attacking Trump, far more than the $67 million raised by the Democratic National Committee. With little primary opposition, Trump and the Republican Party are already in general election mode, free to spend millions in states hell need to win a second term. The Democratic groups are being fueled by seven-figure checks necessary to advertise in battleground states, blunting Trumps big campaign cash advantage. Read more from Bill Allison.

Buttigieg Wants Public College Free for Some: Pete Buttigieg called for spending $120 billion on the Pell Grant program and making public colleges tuition-free for students eligible for those federal grants as part of his proposal released Monday to improve college affordability. Unlike some of his primary opponents, Buttigieg isnt calling for public colleges to be tuition-free for all students, or for total student-debt cancellation. Hes said families that make over a certain income threshold should pay at least some of the cost of their kids higher education. The plan released Monday focuses on helping lower- and middle-income families. Read more from Tyler Pager.

Louisiana Governors Re-election May Offer Lessons for 2020: John Bel Edwards proved its possible to buck Trumps popularity, winning a second term to remain the only Democratic governor in the Deep South. His victory will give political strategists important insights on what it takes to motivate turnout going into 2020, said University of Pennsylvania political science professor Daniel J. Hopkins. Voters may feel more empowered to back a moderate Democrat, said Hopkins, who explores the nationalization of state politics in his book, The Increasingly United States.

In Louisiana, Trump headlined three rallies to try to drive turnout for Republican Eddie Rispone. Edwards, meanwhile, ran on a record of demonstrated independence from his national party. Read more from Jennifer Kay.

North Carolina Lawmakers Back New Congressional Map: North Carolina lawmakers have approved new congressional boundaries to be used in the 2020 elections. The 13 current U.S. House district lines were redrawn after a state court decided Oct. 28 that the map in place since 2016 couldnt be used, pending further review. The North Carolina Superior Court for Wake County had found that plaintiffs were likely to prevail in a lawsuit claiming the boundaries were illegally drawn to maximize Republican advantage. Read more from Andrew M. Ballard.

Barr Blasts Democrats: Attorney General William Barr fired a broadside against critics of Trump and congressional Democrats in particular while defending the presidents actions. In a fiery speech before the conservative Federalist Society on Friday, Barr said Trumps opponents are using every tool they can to intentionally sabotage his administration. Immediately after President Trump won election, opponents inaugurated what they called the Resistance, Barr said. They essentially see themselves as engaged in a war, to cripple by any means necessary, a duly elected government. Read more from Chris Strohm.

Trump, Kim at Odds Ahead of Deadline: The bonhomie between Trump and North Korean leader Kim Jong Un is nearing a key deadline showing new signs of strain. Trump urged Kim over the weekend to act quickly to get a nuclear deal done, suggesting the two leaders could meet again soon. His comments came hours after North Korea ruled out nuclear talks without a policy change by the U.S. and reported on a military drill observed by Kim himself.

Veteran North Korea nuclear adviser Kim Kye Gwan told Trump that Pyongyang will no longer give him things to boast about, the states official KCNA news agency today quoted him as saying. He added North Korea is no longer interested in talks that the U.S. uses to buy time. Read more from Glen Carey and Jihye Lee.

Lawmaker Speaks Out Against U.S. Defense-Cost Hike: Rep. Grace Meng (D-N.Y.) said Trump is destabilizing the U.S.s relationship with South Korea by demanding the Asian nation pay about $5 billion, or five times the amount of its current one-year deal, to host U.S. troops, Jihye Lee reports. Meng tweeted a letter Friday asking Defense Secretary Mark Esper and Secretary of State Michael Pompeo to devise a better strategy that values the alliance, adding that such a hike would be extorting South Korea. Meng said such a request also puts U.S. national security and economic interests in jeopardy. She asked the U.S. officials to reconsider and engage in good faith negotiations.

Esper Has Great Faith in U.S. Military Justice System: Secretary Esper expressed his faith in the countrys military justice system, after Trump last week pardoned two soldiers convicted in Afghanistan killings, Glen Carey reports. Id say first of all that we have a very effective military justice system, Esper said during a press conference at the Asean Defense Ministers Meeting-Plus in the Thai capital of Bangkok yesterday. U.S. military personnel are trained from day one about the laws of armed conflict and how to conduct themselves during wartime, he said.

China, U.S. Trade Talks Continue: Top negotiators from China and the U.S. talked again this weekend, after signs of concessions from both sides on some of the outstanding issues. Chinas Vice Premier Liu He, the countrys key negotiator in the trade talks with the U.S., spoke with Treasury Secretary Steven Mnuchin and Trade Representative Robert Lighthizer by phone on Saturday morning Beijing time, according to the Chinese Commerce Ministry. They had constructive discussions about each sides core concerns in the phase-one deal, and agreed to stay in close communication, the statement said. The USTR confirmed the call took place. Read more.

Trump to Tour Apples Austin Plant: President Donald Trump is scheduled to tour an Apple manufacturing plant in Austin, Texas, on Wednesday, the White House said yesterday. The president plans to visit the plant along with Apple CEO Tim Cook, according to a tweet by spokesman Judd Deere. The Austin American-Statesman newspaper reported that Trump will travel with Treasury Secretary Steven Mnuchin and other administration officials.

The company announced in September that its new Mac Pro computer will be assembled in Texas after it received exclusions from the Trump administration from tariffs on certain parts imported from China. The visit also comes at a time the U.S. and China are close to finalizing the first phase of a highly-anticipated trade deal. Read more from Hailey Waller.

Facebook, Google Donate Heavily to Privacy Advocacy Groups: Few companies have more riding on proposed privacy legislation than Google and Facebook. To try to steer the bill their way, the giant advertising technology companies spend millions of dollars to lobby each year, a fact confirmed by government filings. Not so well-documented is spending to support highly influential think tanks and public interest groups that are helping shape the privacy debate, ostensibly as independent observers.

Bloomberg Law examined seven prominent nonprofit think tanks that work on privacy issues that received a total of $1.5 million over an 18-month period ending Dec. 31, 2018. The groups included such organizations as the Center for Democracy and Technology, the Future of Privacy Forum and the Brookings Institution. The actual total is undoubtedly much higherexact totals for contributions were difficult to pin down. Read more from Daniel R. Stoller.

Scalia Says Big Law is Unwilling to Defend Conservative Views: Major law firms are shying away from defending conservative viewpoints in court, a trend that should trouble the legal profession, Labor Secretary Eugene Scalia told a group of right-leaning lawyers at a Federalist Society event on Friday. It is appropriate, admirable, and necessary for lawyers to take on clients and advance positions that may offend some observers, Scalia said. But some of the countrys biggest law firms appear to be disinclined to protect free speech and free trade in ideas, he argued, calling that evidence of a broad trend of conservative political views being under attack. Read more from Jaclyn Diaz.

To contact the reporters on this story: Zachary Sherwood in Washington at zsherwood@bgov.com; Brandon Lee in Washington at blee@bgov.com

To contact the editors responsible for this story: Giuseppe Macri at gmacri@bgov.com; Loren Duggan at lduggan@bgov.com

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What to Know in Washington: Trump Ally in Impeachment Spotlight - Bloomberg Government

Possible Treatment Found for Contractures in CP, Childhood Paralysis, Early Study Shows – Cerebral Palsy News Today

Scientists at Cincinnati Childrens Hospital Medical Center have found that using the cancer medicine Velcade (bortezomib) in mice prevents the formation of muscle contractures, or tightness of muscles one of the most disabling problems faced by children with cerebral palsy (CP) or brachial plexus injury (NBPI).

This research paves the way for a new and more effective treatment approach for preventing contractures in children born with these conditions, the investigators said.

Their study, Proteasome inhibition preserves longitudinal growth of denervated muscle and prevents neonatal neuromuscular contractures, was published in the journal JCI Insight.

Over time, muscle contractures which develop when normally elastic tissues are replaced by non-stretchy, fiber-like ones disrupt childrens growth in cerebral palsy and NBPI, leading to pain and loss of mobility. Patients may undergo surgery or other treatments to ease the symptoms.

No therapies currently exist that directly tackle the root cause of contractures in childhood paralysis because scientists did not know their biological cause.

But now, a team of researchers at Cincinnati Childrens discovered the culprit to be too much protein destruction in muscles. These scientists found that an approved therapy to treat multiple myeloma andmantle cell lymphoma, called Velcade (marketed by Takeda), can counteract the process and prevent contractures in mice.

In a previous study of a mouse model of NBPI, the team led by Roger Cornwall, MD, a pediatric surgeon at Cincinnati Childrens had found that contractures occurred because paralyzed muscles cannot grow normally in length during early muscle development.

In their new study, the researchers observed that longitudinal muscle growth was actually halted due to a higher than normal breakdown of muscle proteins.

This discovery challenged the long-held view that muscle growth depends mostly on the activity of muscle progenitors, or stem cells.

For decades, neuromuscular contractures have been considered a mechanical problem absent any biological explanation, and only palliative mechanical solutions for them have been available, the researchers said.

Based on the findings, the team tested a new approach to this debilitating and previously unsolved clinical problem. They tested the use of Velcade a chemotherapy known to inhibit protein breakdown in mice models of NBPI shortly after birth.

The treatment proved effective, but required a second medication, [Gly14]-Humanin G, to reduce Velcades known toxic effects.

After four weeks of treatment shortly after birth, our study found that bortezomib [Velcade] significantly reduced shoulder and elbow contractures in a mouse model that mimics these common childhood conditions, Cornwall said in a press release.

Although animal models of CP contractures do not exist, researchers believe the NBPI model can offer valuable insights into the mechanism behind, and therapeutic strategies to prevent contractures in both conditions.

Contractures in CP also are marked by impaired longitudinal muscle growth and appear early after birth, identical to what is seen in the mouse model of NBPI.

Future studies confirming the efficacy of this approach could ultimately render obsolete the destructive surgeries currently required to alleviate contractures in a variety of conditions, Cornwell said.

Given Velcades toxicity, it remains unclear whether it would be deemed safe to test the new therapy in clinical trials.

It also is not known how much older children might benefit from this approach, since the treatments benefits appear to be strongest when given soon after birth.

But the positive results of these early experiments give scientists a starting point from which to work, and could eventually lead to a treatment that could transform childhood paralysis care.

This discovery provides, for the first time, a proof of concept that something we have always considered to be a purely mechanical consequence of limb immobility is actually a biological problem with a medical, rather than physical, solution, Cornwall said.

These findings provide the first strategy to prevent neuromuscular contractures by correcting the underlying deficit in longitudinal muscle growth. Moreover, such an approach may well be harnessed to prevent this same problem resulting from other neuromuscular disorders, the researchers said.

Ana is a molecular biologist with a passion for discovery and communication. As a science writer she looks for connecting the public, in particular patient and healthcare communities, with clear and quality information about the latest medical advances. Ana holds a PhD in Biomedical Sciences from the University of Lisbon, Portugal, where she specialized in genetics, molecular biology, and infectious diseases.

Total Posts: 70

Ana holds a PhD in Immunology from the University of Lisbon and worked as a postdoctoral researcher at Instituto de Medicina Molecular (iMM) in Lisbon, Portugal. She graduated with a BSc in Genetics from the University of Newcastle and received a Masters in Biomolecular Archaeology from the University of Manchester, England. After leaving the lab to pursue a career in Science Communication, she served as the Director of Science Communication at iMM.

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Possible Treatment Found for Contractures in CP, Childhood Paralysis, Early Study Shows - Cerebral Palsy News Today

Asia’s Top Firms Recognized at the 2019 Frost & Sullivan Asia-Pacific Best Practices Awards – Yahoo Finance

Global Awards

Global Contract Research Organization (CRO) Customer Value Leadership Award

COVANCE INC

Global Halal Glove Company of the Year

KOSSAN RUBBER INDUSTRIES BHD

Global High Performance Liquid Chromatography New Product Innovation Award

SHIMADZU

Global Integrated Drug Development in CRO Industry Competitive Strategy Innovation & Leadership Award

WUXI APPTEC

Asia-Pacific Awards

Asia-Pacific Medical Computing Solutions Company of the Year

ADVANTECH CO., LTD.

Asia-Pacific Home Water Treatment Company of the Year

AMWAY

Asia-Pacific Biotech Entrepreneurial Company of the Year

AUM BIOSCIENCES

Asia-Pacific Inbound Contact Routing Systems Market Share Leadership Award

AVAYA

Asia-Pacific Telecoms Digital Services Platform Growth Excellence Leadership Award

BB TECHNOLOGY CO., LTD

Asia-Pacific Emerging Market Telecom Service Provider of the Year

CHUNGHWA TELECOM

Asia-Pacific Home Water Treatment Customer Service Leadership Award

COWAY

Asia-Pacific Telecoms Tower Company of the Year

EDOTCO GROUP SDN BHD

Asia-Pacific CRO Customer Service Leadership Award

EPS HOLDINGS INC

Asia-Pacific AMI Growth Excellence Leadership Award

ITRON

Asia-Pacific Facilities Management Company of the Year

JLL

Asia-Pacific Logistics Service Provider of the Year

KERRY LOGISTICS NETWORK

Asia-Pacific Road Freight Service Provider of the Year

KERRY LOGISTICS NETWORK

Asia-Pacific Inorganic Disinfectants for Healthcare Industries New Product Innovation Award

LOCAL POWER CO. LTD.

Asia-Pacific SIEM Growth Excellence Leadership Award

LOGRHYTHM

Asia-Pacific Biotech CRO Company of the Year

NOVOTECH (AUSTRALIA) PTY. LTD.

Asia-Pacific Customer Experience System Integrator of the Year

NTT LTD.

Asia-Pacific End-to-End UCC Service Provider of the Year

NTT LTD.

Asia-Pacific Managed Security Service Provider of the Year

NTT LTD.

Asia-Pacific Cloud Contact Center Service Provider of the Year

ORANGE BUSINESS SERVICES

Asia-Pacific Critical Power Infrastructure Vendor of the Year

SCHNEIDER ELECTRIC

Asia-Pacific Secure IOT Service Provider of the Year

SINGTEL

Asia-Pacific Mobile Wallet Provider of the Year

SINGTEL DASH

Asia-Pacific Ultra-low Weight Nitrile Glove Product Leadership Award

SMART GLOVE HOLDINGS

Asia-Pacific Managed UC Service Provider of the Year

TATA COMMUNICATIONS

Asia-Pacific Neurodegenerative Disease Management Technology Innovation Award

TAURX PHARMACEUTICALS LTD

Asia-Pacific Customer Contact Outsourcing Technology Leadership Award

TELEPERFORMANCE INDIA

Asia-Pacific Customer Contact Enabling Technology Leadership Award

TETHERFI

Asia-Pacific Customer Contact Outsourcing Customer Service Leadership Award

TRANSCOM WORLDWIDE PHILIPPINES INC.

Asia-Pacific Critical Infrastructure Solutions Growth Excellence Leadership Award

VERTIV CO.

Asia-Pacific Data Center Power Systems Customer Value Leadership Award

VERTIV CO.

Southeast Asia Awards

Emerging Southeast Asia Smart City Governance Agency of the Year

SMART SELANGOR DELIVERY UNIT

Southeast Asia Contact Center Applications Market Share Leadership Award

AVAYA

Southeast Asia Stem Cell Banking Growth Excellence Leadership Award

MEDEZE

Southeast Asia Unified Communications System Integrator of the Year

NTT LTD.

Southeast Asia Smart Buildings Competitive Strategy Innovation and Leadership Award

QUANTUM AUTOMATION PTE LTD

Southeast Asia Managed Security Service Provider of the Year

TRUSTWAVE, A SINGTEL COMPANY

Country Awards

Australia Customer Contact Outsourcing Service Provider of the Year

STELLAR

Indonesia Payment Gateway Company of the Year

FASPAY

Indonesia Healthcare IT Growth Excellence Leadership Award

HOPE BY PT MEDIA TIGA WARNA

Indonesia Cloud Infrastructure Services Provider of the Year

LINTASARTA

Indonesia Emerging Hospital of the Year

MAYAPADA HEALTHCARE

Indonesia Data Center Services Provider of the Year

PT. SIGMA CIPTA CARAKA (TELKOMSIGMA)

Indonesia IOT Services Provider of the Year

PT TELEKOMUNIKASI INDONESIA TBK

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Asia's Top Firms Recognized at the 2019 Frost & Sullivan Asia-Pacific Best Practices Awards - Yahoo Finance

Takeda, Anderson Cancer Center team up for cell therapy – BSA bureau

Takeda and Anderson Cancer Center announce cooperation to accelerate the development of clinical stage, out-of-the-box CAR NK cell therapy platform

University of Texas MD Anderson Cancer Center(The University of Texas MD Anderson Cancer Center)and Takeda Pharmaceutical Company Limited (Takeda Pharmaceutical hasannounced an exclusive licensing agreement and research agreement to develop cord blood-derived chimeric antigen receptors for "arming" IL-15 for the treatment of B-cell malignancies and other cancers Chimeric Antigen Receptor-directed Natural Killer (CAR NK) cell therapy.

Under the agreement, Takeda will receive access to the CAR NK platform of the Anderson Cancer Center and the exclusive rights to develop and commercialize up to four projects, including a CD19-targeted CAR NK cell therapy and a B-cell maturation. The antigen (BCMA) is the target of CAR NK cell therapy.The Takeda and Anderson Cancer Center will conduct a research collaboration to further develop the above CAR NK project.

Katy Rezvani, MD, Ph.D.,Professor ofStem Cell Transplantation and Cell Therapyat theAnderson Cancer Center,said: "Our vision is to improve existing treatments by developing armored CAR NK, which can be administered in outpatient settings. Out of the box, more patients can get effective, fast and least toxic treatment. Takeda's expertise in hematological malignancies and the commitment to develop next-generation cell therapies make it an ideal partner for our team. Promote CAR NK cell therapy for patients with therapeutic needs."

New ways to deliver out of the box ready to use CAR in outpatient facilitiesallogeneic Anderson Cancer Center platform CAR NK NK cells isolated from umbilical cord blood, the fight against certain cancers expressing CAR targets after processing.CAR NK cells are modified by retroviral vectors to deliver genes and enhance their efficacy against specific tumors.CD19 CAR increases the specificity of these cells against B cell malignancies, while the immune cytokine IL-15 enhances the proliferation and survival of CAR NK cells in vivo.

Existing CAR T cell therapy drugs use patient-generated genetically modified T cells, and the preparation process takes several weeks. In contrast, CAR NK cells are designed to be prepared from non-relative donor sources and stored in an out-of-the-box manner. So that treatment can be implemented faster.

CD19 CAR NK cell therapy is expected to be administered in an outpatient setting.In an ongoing phase 1/2a clinical study, patients with relapsed refractory B-cell malignancies who received CD19 CAR NK cell therapy did not see severe cytokine release syndrome (CRS) observed in existing CAR-T treatments. ) or neurotoxicity.

Anderson Cancer Center to develop CAR NK platform led by Dr. Rezvani, and withadoptive cell therapy platform,Chronic Lymphocytic Leukemia Moon ShotandB-the Cell Lymphoma Moon Shotfurther support of the project were the hospitalMoon Shots Programof In part, the project is a collaborative effort to rapidly develop scientific discoveries into meaningful clinical advances that can save patients' lives.

Takeda: CAR accelerate the development of next-generation multiple platformsAndy Plump Takeda, president of R & D, MD, Ph.D., said: "Anderson Cancer Center CAR NK cell therapy platform represents a potential cure for the drug, which is what we are to CD19 CAR NK Established as a leader in the treatment of drug candidates for cancer in the field. We must work flexibly and purposefully, so we plan to start the pivotal study of CD19 CAR NK in 2021."

In addition to CAR NK cell therapy, Takeda and its partners are investigating ways to improve the safety, efficacy, and accessibility of first-generation CAR T cell therapies, includinggamma delta CAR T,induced pluripotent stem cell-derived CAR T,with solid tumors as targets of CAR Tandother next-generation methods.Takeda plans to the end of fiscal year 2020 to promote the five kinds of tumor cell therapy to clinical stage.The platform is being developed through collaboration with partners and the use of Takeda's expertise in transforming cell therapy engines that provide bioengineering, chemistry, manufacturing and control (CMC), clinical and transformation functions in a single location to overcome cell therapy drugs. Numerous manufacturing challenges in development.

Takeda is responsible for the development, manufacture and commercialization of the CAR NK products produced by the agreement.The Anderson Cancer Center will receive upfront payments and be eligible to receive royalties for development and commercialization milestones for each target and for any CAR NK product net sales.

The Anderson Cancer Center and Takeda will continue to conduct research on other targets and the CAR NK platform under the direction of a joint research committee.The Anderson Cancer Center will deploy aninstitutional conflict of interest management and monitoring programfor the study.

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Takeda, Anderson Cancer Center team up for cell therapy - BSA bureau

Takeda and MD Anderson Announce Collaboration to Accelerate the Development of Clinical-Stage, Off-The-Shelf CAR NK-Cell Therapy Platform – BioSpace

Nov. 5, 2019 12:00 UTC

HOUSTON & OSAKA, Japan--(BUSINESS WIRE)-- The University of Texas MD Anderson Cancer Centerand Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) (Takeda) today announced an exclusive license agreement and research agreement to develop cord blood-derived chimeric antigen receptor-directed natural killer (CAR NK)-cell therapies, armored with IL-15, for the treatment of B-cell malignancies and other cancers.

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Under the agreement, Takeda will receive access to MD Andersons CAR NK platform and the exclusive rights to develop and commercialize up to four programs, including a CD19-targeted CAR NK-cell therapy and a B-cell maturation antigen (BCMA)-targeted CAR NK-cell therapy. Takeda and MD Anderson will also conduct a research collaboration to further develop these CAR NK programs.

Our vision is to improve upon existing treatments by developing armored CAR NKs that could be administered off-the-shelf in an outpatient settingenabling more patients to be treated effectively, quickly and with minimal toxicities, said Katy Rezvani, M.D., Ph.D., professor of Stem Cell Transplantation and Cellular Therapy at MD Anderson. With their expertise in hematologic malignancies and commitment to developing next-generation cell therapies, Takeda is the ideal collaborator to help our team advance CAR NK-cell therapies to patients in need of treatments.

A Novel Approach to Delivering Off-the-Shelf CARs in an Outpatient Setting MD Andersons allogeneic CAR NK platform isolates NK cells from umbilical cord blood and engineers them to express CARs against specified cancer targets. CAR NK cells are modified with a retroviral vector to deliver genes and enhance their effectiveness to attack specific tumors. A CD19 CAR increases the cells specificity for B-cell malignancies while the immunocytokine IL-15 enhances the proliferation and survival of the CAR NK cells in the body.

In contrast to current CAR T-cell therapies that utilize a patients own genetically modified T-cells and require a multi-week manufacturing process, CAR NK cells are intended to be manufactured from a non-related donor source and stored for off-the-shelf use, allowing treatment to be delivered more rapidly.

It is anticipated that the CD19 CAR NK-cell therapy could be administered in an outpatient setting. In an ongoing phase 1/2a clinical study treating patients with relapsed and refractory B-cell malignances, the CD19 CAR NK-cell therapy has not been associated with the severe cytokine release syndrome (CRS) or neurotoxicity observed with existing CAR-T therapies.

The development of MD Andersons CAR NK platform is led by Dr. Rezvani and is further supported by the adoptive cell therapy platform, Chronic Lymphocytic Leukemia Moon Shot and B-Cell Lymphoma Moon Shot, all part of the institutions Moon Shots Program, a collaborative effort to rapidly develop scientific discoveries into meaningful clinical advances that save patients lives.

Takeda: Accelerating the Development of Multiple Next-Generation CAR Platforms MD Andersons CAR NK platform represents the curative potential of cell therapies, which is why we are establishing the CD19 CAR NK as our lead cell therapy candidate in oncology, said Andy Plump, M.D., Ph.D., President of Research and Development at Takeda. We need to work swiftly and with purpose, and as such, we intend to initiate a pivotal study of the CD19 CAR NK in 2021.

In addition to CAR NK-cell therapies, Takeda and its partners are investigating multiple approaches to improving the safety, efficacy and accessibility of first-generation CAR T-cell therapies including gamma delta CAR Ts, induced pluripotent stem cell-derived CAR Ts, CAR Ts targeting solid tumors, and other next-generation approaches. Takeda plans to advance five oncology cell therapies to the clinic by the end of FY20.1 These platforms are being developed both with partners and by applying the expertise of Takedas translational cell therapy engine which provides bioengineering, chemistry, manufacturing and control (CMC), clinical and translational capabilities in a single footprint to overcome many of the manufacturing challenges experienced in cell therapy development.

Takeda is responsible for the development, manufacturing and commercialization of CAR NK products resulting under the agreement. MD Anderson will receive an upfront payment and is eligible to receive development and commercial milestones for each target as well as tiered royalties on net sales of any such CAR NK product.

MD Anderson and Takeda will continue research for the additional targets and CAR NK platform under the direction of a joint research committee. MD Anderson will implement an Institutional Conflict of Interest Management and Monitoring Plan for this research.

About MD Anderson The University of Texas MD Anderson Cancer Center in Houston ranks as one of the world's most respected centers focused on cancer patient care, research, education and prevention. The institutions sole mission is to end cancer for patients and their families around the world. MD Anderson is one of only 50 comprehensive cancer centers designated by the National Cancer Institute (NCI). MD Anderson is ranked No.1 for cancer care in U.S. News & World Reports Best Hospitals survey. It has ranked as one of the nations top two hospitals for cancer care since the survey began in 1990, and has ranked first 15 times in the last 18 years. MD Anderson receives a cancer center support grant from the NCI of the National Institutes of Health (P30 CA016672).

About Takeda Pharmaceutical Company Limited Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Gastroenterology (GI), Rare Diseases and Neuroscience. We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions. For more information, visit https://www.takeda.com

Forward-Looking Statements This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takedas future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. In particular, this press release contains forecasts and management estimates related to the financial and operational performance of Takeda, including statements regarding forecasts for Revenue, Operating profit, Adjusted EBITDA, Profit before income taxes, Net profit attributable to owners of Takeda, Basic earnings per share, Amortization and impairment and other income/expense, Underlying Revenue, Underlying Core Earnings margin, Underlying Core EPS and Net Debt. Without limitation, forward looking statements often include the words such as targets, plans, believes, hopes, continues, expects, aims, intends, will, may, should, would, could anticipates, estimates, projects or words or terms of similar substance or the negative thereof. Any forward-looking statements in this document are based on the current assumptions and beliefs of Takeda in light of the information currently available to it. Such forward-looking statements do not represent any guarantee by Takeda or its management of future performance and involve known and unknown risks, uncertainties and other factors, including but not limited to: the economic circumstances surrounding Takedas business, including general economic conditions in Japan, the United States and worldwide; competitive pressures and developments; applicable laws and regulations; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; changes in exchange rates; claims or concerns regarding the safety or efficacy of marketed products or products candidates; and post-merger integration with acquired companies, any of which may cause Takedas actual results, performance, achievements or financial position to be materially different from any future results, performance, achievements or financial position expressed or implied by such forward-looking statements. For more information on these and other factors which may affect Takedas results, performance, achievements, or financial position, see Item 3. Key InformationD. Risk Factors in Takedas Registration Statement on Form 20-F filed with the U.S. Securities and Exchange Commission, available on Takedas website at: https://www.takeda.com/investors/reports/sec-filings/ or at http://www.sec.gov. Neither Takeda nor its management gives any assurances that the expectations expressed in these forward-looking statements will turn out to be correct, and actual results, performance or achievements could materially differ from expectations. Persons receiving this press release should not place undue reliance on forward looking statements. Takeda undertakes no obligation to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make. Past performance is not an indicator of future results and the results of Takeda in this press release may not be indicative of, and are not an estimate, forecast or projection of Takedas future results.

1 Takedas 2020 fiscal year begins April 1, 2020 and ends March 31, 2021.

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Takeda and MD Anderson Announce Collaboration to Accelerate the Development of Clinical-Stage, Off-The-Shelf CAR NK-Cell Therapy Platform - BioSpace

Diabetics make insulin for first time with stem cell-derived therapy – The San Diego Union-Tribune

Stem cell therapies are becoming increasingly versatile. Two presentations at this falls Cell and Gene Meeting on the Mesa in Carlsbad illustrated how the field is maturing.

San Diegos ViaCyte is developing two versions of an experimental diabetes treatment, using insulin-producing cells grown from embryonic stem cells. The cells are encapsulated in a device thats implanted below the skin.

One version, called PEC-Direct, shows intermittent signs of producing therapeutic levels of insulin, the first time this has been seen, said Paul Laikind, ViaCytes CEO.

This was seen indirectly through the presence of a byproduct of insulin production. However, production isnt reliable enough to substitute for injected insulin, Laikind said. ViaCyte is now working to improve reliability.

PEC-Direct is meant for the most severe cases of type 1, or insulin-dependent, diabetes. It allows blood vessels to grow into the device to contact the cells. To prevent an immune reaction, patients get immunosuppressive drugs.

The other product, PEC-Encap, shields the cells from direct contact. Immune-suppressing drugs arent required. However, it didnt work well in early testing, because scar tissue built up around the implant.

ViaCyte has worked on that problem with partner W. L. Gore & Associates, makers of Gore-Tex. The partner makes the implant material, and has been collaborating with ViaCyte to make it less likely to produce scarring. Early signs are that the newly formulated material is meeting that goal.

Another company at the meeting, Cleveland-based Athersys, reported on its multi-purpose cell product, MultiStem. Its in late-stage or Phase 3 testing for stroke, caused by blood clots. Its also in testing for heart attacks, and being considered for brain trauma.

MultiStem is a proprietary cell that can be given off the shelf to different patients. Its obtained from adult bone marrow. Gil Van Bokkelen, Athersys chairman and CEO, said MultiStem doesnt provoke an immune reaction.

The cells secrete various proteins and other substances that reduce inflammation and promote healing, Van Bokkelen said. They dont persist, and eventually disappear from the body.

This healing potential got the attention of doctors at The University of Texas Health Science Center at Houston and the Department of Defense, he said.

Athersys is now planning a midstage, or Phase 2, trial of MultiStem in trauma patients. The goal is to temporarily reduce inflammation, which goes to excess in trauma.

If you can tip the scales in favor of a more constructive response, stop the bad stuff, that hyper-inflammatory tidal wave, you can really alter the trajectory of how the patient rebounds, he said.

While these cells naturally exist in the body, Van Bokkelen said there arent enough of them to take care of major injuries.

Its all about kinetics, he said. In a major trauma, youre talking about minutes or hours. Youre not talking about weeks. And so the cells simply cannot respond fast enough to be able to really have the impact that we see that we can have.

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Diabetics make insulin for first time with stem cell-derived therapy - The San Diego Union-Tribune

An artist and a transplant researcher discuss the heart – Harvard Gazette

Doris A. Taylors so-called replacement ghost heart suggests something otherworldly, but the eerie-looking form is far from an apparition. Its an innovative approach to organ transplantation that has inspired many in the medical community and at least one artist.

The Texas researchers process piggybacks on natures sophisticated design. Together she and a team of researchers strip cells off human and animal cadaver hearts with a soapy solution, leaving ghostly white protein shells that retain the form of the organ. They inject them with a patients blood or bone-marrow stem cells, and the ghost hearts act as scaffolding on which the newly introduced cells can slowly transform into a beating muscle.

What we said was, Wouldnt it be really cool if we could wash the sick cells out and put the healthy cells back in? said Taylor, director of Regenerative Medicine Research and director of the Center for Cell and Organ Biotechnology at the Texas Heart Institute, during a recent talk at the Radcliffe Institute for Advanced Study.

The hope is that one day these regenerated hearts will resolve the most challenging issues transplant patients currently face: the lack of a permanent artificial replacement, concerns about rejection, and the shortage of viable donor hearts.

Taylors efforts are driving what could become a revolution in organ transplants, and they have sparked the creativity of transdisciplinary artist Dario Robleto, whose latest work, on view at the Johnson-Kulukundis Family Gallery in Radcliffes Byerly Hall, recreates in images and sounds the original pulse wave of the heart first captured in visual form by scientists in the 1900s. Robleto and Taylor, longtime friends and Texas residents, explored those connections during Mondays Radcliffe discussion, which was moderated by Jennifer Roberts, Elizabeth Cary Agassiz Professor of the Humanities.

Robletos exhibit, Unknown and Solitary Seas, touches on the overlap between the medical mysteries and workings of the vascular pump, and the metaphor for the heart as the emotional center of the soul. It includes a video installation that features recreated sounds of a beating heart from the 19th century, reconstructed images of how the earliest pulse waves first appeared on the page, and a series of heart waveform sculptures in brass-plated stainless steel.

Roberts said that with his work, Robleto acknowledges the pulse waves promise, their profundity, their scientific value, but he also reclaims some of their ambiguity and asks us to wonder whether we can or should accept that these waveforms have escaped the realms of art, culture, and emotional communication.

Taylor similarly views her work as a blend of the scientific and the human. It transcends complicated, complex science, she says, in that her ghost hearts require a kind of passion, commitment, care, attention, and nurturing similar to whats required by a small child. Its really about building hearts at the emotional, mental, spiritual, and physical level that I think is going to get them to work, she said.

For Robleto, big ideas, like the creation of a new human heart, require multiple perspectives.

The artist called Taylors work one of the most fascinating and definitely one of the most emotional things Ive ever seen. As an object, he added, the ghost heart is stunningly beautiful but it also raises questions about the self, identity, emotion, the notions of form and where memory is truly held, questions he thinks artists can help address. He cited two of the nations earliest heart transplants, after which the patients wives asked their husbands, who had received donor hearts, if they still loved them.

Taylors work, Robleto said, is right at the edge of identity and materiality and so when the day comes when someone says the first ghost heart transplant I think we will have a similar moment where perhaps we will be forced to re-evaluate what we ask from our heart metaphor.

Dario Robletos Unknown and Solitary Seas is on view in Byerly Halls Johnson-Kulukundis Family Gallery through Jan. 18, 2020.

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An artist and a transplant researcher discuss the heart - Harvard Gazette

Impact Of ELN Risk Stratification, Induction Chemotherapy Regimens And | CMAR – Dove Medical Press

Shanglong Feng,1,* Li Zhou,2,* Xinhui Zhang,1,* Baolin Tang,2 Xiaoyu Zhu,2 Huilan Liu,2 Zimin Sun,2 Changcheng Zheng1,2

1Department of Hematology, Anhui Provincial Hospital, Anhui Medical University, Hefei, Peoples Republic of China; 2Department of Hematology, The First Affiliated Hospital of USTC, Division of Life Sciences and Medicine, University of Science and Technology of China, Hefei, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Changcheng ZhengDepartment of Hematology, Anhui Provincial Hospital, Anhui Medical University, Hefei 230001, Peoples Republic of ChinaTel/fax +86-551-62284476Email zhengchch1123@ustc.edu.cn

Background: Hyperleukocytic acute myeloid leukemia (AML) (initial white blood cell count100 109/L) is a clinical emergency often accompanied by leukostasis syndrome, tumor lysis syndrome (TLS), and disseminated intravascular coagulation (DIC), with a poor clinical prognosis. The aim of this study retrospectively analyzed the clinical features of hyperleukocytic AML, focusing on high-risk factors affecting prognosis, the selection of initial induction therapy, and the impact of hematopoietic stem cell transplantation (HSCT) on prognosis.Patients and methods: A total of 558 AML patients at our center from January 2013 to December 2017 were diagnosed, and 52 (9.32%) patients presented with hyperleukocytosis were retrospectively reviewed.Results: The 3-year overall survival (OS) rate in the 1539 years old and 4060 years old group was 58.8% and 25.4%, respectively; the longest survival time in patients aged >60 years was only 8 months, and the 8-month OS rate was 8.3% (p=0.002). The 3-year OS rate of the patients in the favorable risk group, intermediate risk group and high risk group, according to the 2017 ELN risk stratification, was 50%, 28.0%, and 29.5%, respectively (p=0.374). The 3-year OS rate of patients carrying CEBPA or NPM1 mutation and those with FLT3-ITD or MLL mutation was 37.5% and 30.0%, respectively (p=0.63). The 3-year OS rate of patients employing an induction regimen of a standard IA regimen was 58.4%, and of those employing a non-standard IA regimen was 22.2% (p=0.065). The 3-year OS rate of the transplantation patients reached 73.8%, while the 9-month OS rate of patients without transplantation was 11.4% (p<0.001).Conclusion: This study suggest that hyperleukocytosis is an independent risk factor for AML patients, regardless of the risk stratification based on cytogenetic or molecular abnormalities. Age is the main factor influencing the prognosis of hyperleukocytic AML. The use of a standard IA regimen and HSCT can significantly improve the patients prognosis.

Keywords: acute myeloid leukemia, hyperleukocytosis, ELN risk stratification, induction chemotherapy, hematopoietic stem cell transplantation

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Impact Of ELN Risk Stratification, Induction Chemotherapy Regimens And | CMAR - Dove Medical Press