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A breakthrough medical technology can save the lives of children with heart defects. Scientists have developed the first-ever heart valve that grows with the child, reducing the need for risky heart surgeries in the future.

Children with congenital heart disease who need valve replacement often undergo multiple surgeries because the valve cannot grow as the child's heart grows. They need multiple heart surgeries to change the heart valve to accommodate the growing size of the heart. However, these surgeries are risky and pose a threat to the child's health.

To solve this problem, a team of scientists from Boston Children's Hospital developed a prosthetic valve that mimics the geometry of the human venous valve. Composed of polymeric leaflets attached to a stainless-steel stent, the valve can be expanded by a minimally invasive balloon catheter procedure, reducing invasive open-heart surgeries.

The doctors can use keyhole surgery to insert a rubber tube attached to a deflated balloon in the valve. They can inflate it depending on the child's heart size.

The valve replacement expanding to accommodate different lengths in implanted sheep. This material relates to a paper that appeared in the Feb. 19, 2020, issue of Science Translational Medicine, published by AAAS. The paper, by S.C. Hofferberth at Boston Children's Hospital in Boston, MA; and colleagues was titled, "A geometrically adaptable heart valve replacement." Image Credit: S.C. Hofferberth et al., Science Translational Medicine (2020)

Multiple heart surgeries

Congenital heart valve disease is life-threatening, and children with this condition may need valve replacement early in their lives. However, children grow, and the artificial heart valve may not be able to accommodate the heart's increasing size.

Many children with this condition face high-risk and multiple open-heart surgeries to remove the valves and replace then with bigger ones.

The scientists used computational modeling to predict how their valve replacement expanded to deal with the stress of blood flow. This material relates to a paper that appeared in the Feb. 19, 2020, issue of Science Translational Medicine, published by AAAS. The paper, by S.C. Hofferberth at Boston Children's Hospital in Boston, MA; and colleagues was titled, "A geometrically adaptable heart valve replacement." Credit: S.C. Hofferberth et al., Science Translational Medicine (2020)

For the first time, the new valve, a biomimetic prosthetic valve, adapts to accommodate growth and structural asymmetries within the heart. In previous heart valve models, they contain three leaflet-like flaps providing a one-way inlet or outlet for blood flow. However, in the new heart valve, it only has two flaps, with a geometry designed to maintain closure, and a one-way flow even when the veins expand in diameter.

"Veins carry approximately 70 percent of our blood volume. The vein dimensions can change dramatically depending on body position, yet the valves must remain functional. We mimicked the geometric profile of the human venous valve to design a bi-leaflet valve of programmed dimensions that is adaptable to growth without loss of one-way flow control," Dr.  Sophie C. Hofferberth, a surgical resident at Brigham and Women's Hospital and lead researcher at Boston Children's Hospital, said.

New artificial heart valve on the way

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The new heart valve has been tested in large animal models, computer simulations, and benchtop studies, demonstrating that it works across a broad range of sizes. What is more, it retains functionality and efficacy when it is expanded through a balloon catheter procedure.

The team tested the prosthetic heart valve in growing young lambs. When implanted on the animals, it exhibited good performance without the blockage of blood flow. In another test in other lambs, the valves stayed functional for ten weeks without causing inflammation or injury to the heart tissues.

Though the study requires human testing and longer-follow up times, there is more work needed to validate the design. If it passes through rigorous testing, it can help more than 1.35 million children across the globe who were born with a congenital heart valve disorder.

The researchers also found that the new prosthetic valve promotes favorable blood flow through the valve, reducing the risk for blood clot formation, which is often observed in existing valve replacement devices. With the invasive heart valve device, there are fewer complications that may endanger the life of the child.

The study was published in the journal Science Translational Medicine.

What is congenital heart valve disease?

The heart pumps blood throughout the body throughout the day, and it contains valves that are responsible for keeping the blood from flowing backward. As a result, the blood flow is controlled, and the oxygenated and non-oxygenated blood will not mix.

A congenital heart valve disease happens if one or more of the valves in the heart do not work well, leading to problems such as regurgitation, stenosis, and atresia. Usually, this occurs when a heart's valves do not develop before birth, causing a defect that keeps the valve from closing completely.

Regurgitations happen when the blood backflows because the valve does not close tightly or adequately. The most common cause of blood backflow is a valve prolapse.

Stenosis happens when the flaps become thick, stiff, or fuse, resulting in the inability of the valve to open fully. Stenosis leads to blockage of blood flow. Atresia pertains to a condition when the valve does not have an opening for the blood to pass through.

All these conditions lead to a wide range of heart problems and can endanger the life of the child. Over time, these problems can strain the heart because it works double-time to compensate for the valve defect. It can cause serious problems such as aortic aneurysm, dilated cardiomyopathy, and heart failure.

Source:

National Heart, Lung, and Blood Institute. (2020). Heart Valve Disease. https://www.nhlbi.nih.gov/health-topics/heart-valve-disease.

Journal reference:

Hofferberth, S., Saeed, M., Tomholt, Fernandes, M., Payne, C., Price, K., Marx, G., Esch, J., Brown, J. et al. (2020). A geometrically adaptable heart valve replacement. Science Translational Medicine. https://stm.sciencemag.org/content/12/531/eaay4006?rss=1.

A new study published in February 2020 in the journal JAMA Otolaryngology-Head and Neck Surgery shows that using mainstream media to depict rare diseases in a realistic manner can prompt greater public awareness and support for such conditions.

Rare diseases have a very small presence in this world, affecting about 400 million people around the globe. Despite the very few cases of each kind of disease, they often cause a lot of suffering and require significant treatment and rehabilitation. In other words, they create a heavy burden in financial terms as well as the amount of care they need, both on the patients and the health care system. Yet it is challenging to mobilize funds for research, prevention, and treatment, to raise awareness about the condition, or to get sufficient manpower.

Increasing the level of awareness is an urgent necessity, therefore. One possible way out is to show television shows focusing on these conditions as part of widely seen programs. One is the Netflix series, Stranger Things, which showcases the life of Dustin Henderson, a fictional individual born with the rare disease cleidocranial dysplasia (CCD). Henderson is portrayed by actor Gaten Matarazzo III who himself has this condition.

LOS ANGELES – JUN 28: Cast at the "Stranger Things" Season 3 World Premiere at the Santa Monica High School on June 28, 2019 in Santa Monica, CA. Image Credit: Kathy Hutchins / Shutterstock

CCD is a genetic condition in which the clavicles, or collar bones, and the teeth fail to grow or grow abnormally. These features are emphasized in scenes where Dustin is explaining the condition to his friends and colleagues.

However, symptoms vary between patients, even in the same family. CCD currently affects 1 in a million children the world over, both boys and girls.

The early features of abnormal bone development include a prolonged period of open fontanelles when the bones of the skull remain separate at the front and back of the head for more than the usual length of time. The absence of complete collarbones may make the chest appear narrow, and the shoulders are sloping. In fact, many of these children can make their shoulders touch in front.

Bone density is low, the pelvis is narrow, and the height shorter than expected. Baby teeth are lost early, and the secondary teeth arrive late, which may cause the jaw to be crowded with teeth and the jaw misaligned.

The disease is due to an abnormality of the RUNX2 gene, mostly due to a de novo mutation, that is, one which arises at random in the developing embryo rather than being inherited from the parents. If one of the two copies of this gene is affected, the condition will manifest itself.

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Treatment could include dental replacements, speech therapy, treatment of sinus and ear infections, supplementation with vitamin D and calcium, and protecting the head from trauma until the bones are fused.

The study

The current study uses Google Trends and data from websites related to CCD to examine the link between how the condition is portrayed on television shows and the resulting increase in public interest and awareness of CCD.

Google Trends was used to look at the trends for searches focusing on CCD after the show Stranger Things was released. The researchers did a worldwide search for searches with this keyword, over a 5-year period from 2014 to 2019. Also, they asked several foundations dealing with CCD, such as the Children’s Craniofacial Foundation, about any increase in the number of calls or website hits after each season’s release of Stranger Things. This was compared with the expected traffic.

The findings

The researchers found that search interest surged by over 94% after the third season’s release of Stranger Things. In comparison, an increase of about 11% and almost 13% was seen after season 1 and season 2.

Public interest in CCD was reported to increase by all websites related to this topic. However, quantifiable data were obtained only from the foundation About KidsHealth, which had, on average, 5 to 80 views of the CCD page each week, but in the week succeeding the third season’s release of the show, 10,000 visits.

Implications

The portrayal of the condition on the popular television show has helped spark interest in many individuals. Each season has seen a spurt in weekly searches above the predicted number. Meanwhile, Matarazzo has participated in setting up the new foundation CCD Smiles, which is focused on raising awareness of this condition around the world, helping affected people with their dental work, and helping research.

The importance of harnessing celebrity power is vast reach it can offer via already existing links to the public and the media.

And when public awareness increases, funding goes up, early diagnosis is more likely, treatment becomes more broad-based, and people learn to understand and cope with the disorder. This, in turn, helps those affected by it to develop more coping strategies, feel a greater sense of integration into the broader community, and have a better quality of life.

The media also has a catalytic role in helping hundreds of thousands of people to understand such conditions better and to make sure they are correctly represented. Accurate artistic depiction of such conditions is guided by the Entertainment Industries Council, which develops material to help writers and producers understand the disease better so they can present it correctly.

However, at present, there is no guide material for CCD and many other rare diseases. Therefore, the study concludes, “Although writers need to exercise caution when portraying characters with rare disorders, the inclusion of such characters in the mainstream media may be a beneficial strategy to raise awareness of them.”

Journal reference:

Johnson AL, Torgerson T, Cooper C, Khojasteh J, Vassar M. Public Awareness of Cleidocranial Dysplasia After Season Releases of Stranger Things. JAMA Otolaryngol Head Neck Surg. Published online February 20, 2020. doi:10.1001/jamaoto.2019.4791

Earlier this year, doctors and researchers celebrated the news that the five-year survival rate for pancreatic cancer had crossed into double digits for the first time -; up from just 6% in 2011.

And while researchers at the University of Michigan Rogel Cancer Center and elsewhere continue to pursue scientific insights into the disease and develop new therapeutic approaches, surgeons on the front line of patient care are also working hard to improve outcomes.

Hari Nathan, M.D., Ph.D., an assistant professor of surgery at Michigan Medicine, sat down with the Michigan Health blog to talk about recent advances in surgical care for patients with pancreatic cancer, and some things patients should look for when evaluating where to seek treatment.

Growing evidence that starting chemo before surgery could improve survival

The best approach for treating pancreatic cancer remains physically removing the cancer through surgery, also called resection, in combination with chemotherapy and sometimes radiation therapy, Nathan says.

"It's been known for decades that adding chemotherapy to resection for pancreas cancer improves survival," he says. "It's a very difficult disease to begin with, and the survival rates are still unacceptably low compared to the progress we have made in a variety of other cancers. But rates are improving -; and part of the reason they're improving is the development of more effective chemotherapy regimens over time."

Recently, surgeons at Michigan Medicine have adopted an emerging practice of administering some or all of a patient's chemotherapy before they undergo an operation.

One recent study, for example, found that patients who underwent chemo before surgery -; known as neoadjuvant chemotherapy -; had a two-year survival rate of 63.7% compared to 53.5% in those who received surgery first.

That difference is driven by multiple reasons. Even in the best hands, these operations are challenging and can be fraught with complications. And we know that when we give chemotherapy after an operation, roughly half of patients aren't healthy enough to complete the intended course."

Hari Nathan, M.D., Ph.D., assistant professor of surgery at Michigan Medicine

Giving chemotherapy on the front end -; before a major operation takes a toll on a patient's body -; increases the likelihood they'll complete more of their chemotherapy, which, in turn, is correlated with better outcomes, he notes.

For some patients, earlier administration of chemotherapy could spare them from going through an operation if there are signs it won't be effective.

"A small percentage of patients will develop additional disease during chemotherapy, or have progression of the tumor," Nathan says. "That's a warning sign that their disease is more aggressive and putting them through a major operation may not be helpful to them."

Helping more patients become candidates for surgery

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In addition to improving the number of patients who complete their course of chemo, the neoadjuvant approach is also expanding the number of patients who are candidates for surgery, Nathan says.

"What we often find with these newer chemotherapy regimens is that they actually can kill parts of the tumor that will make an operation more viable," he says.

In the past, a patient may have been told that their tumor wasn't resectable because it was wrapped around a vital artery.

"But if we can kill off the part of the tumor that was a barrier to a successful surgery, then it makes it possible to go in and do the operation," Nathan says.

"In a lot of ways, what we're doing in pancreatic cancer now mimics what we've been doing in other cancers for a while," he adds. "The difference is that in colorectal cancer, for example, we've had more effective chemotherapy agents, and so we've been willing to be more surgically aggressive. And now with pancreatic cancer, we're finally getting to that point where we have these more effective chemotherapy regimens."

Where patients get care can make a big difference

There are a number of reasons why receiving care at a nationally ranked hospital or academic medical center, like Michigan Medicine, can also make a big difference for patients, Nathan says.

The first is access to new, otherwise unavailable experimental treatments through clinical trials.

For example, Nathan says, U-M is a site for a trial enrolling patients who are not initially eligible for surgery because their pancreatic cancer involves major blood vessels or other tissue in the abdomen. The trial is to test an investigational medicine that helps chemotherapy to better attack the tumor, with a goal, ultimately, of making the patients candidates for surgery.

Second, larger, high-volume, multidisciplinary centers will have more experience in specifically treating pancreatic cancer (Michigan Medicine was recently designated a National Pancreas Foundation Center of Excellence for Pancreatic Cancer, one of just two in the state.)

"I would argue that medical centers like U-M, by virtue of the breadth and depth of the system as a whole, can provide the highest quality care," Nathan says. "In addition to the quality of our surgeons, our medical oncologists, radiation oncologists, radiologists, geneticists and nutritionists all have expertise in pancreatic cancer. Everybody in our multidisciplinary pancreatic clinic has a large focus of their practice devoted to taking care of patients with pancreas cancer. That's not the case everywhere."

Source:

Michigan Medicine – University of Michigan

Tom Catena, M.D., has been described as ”the world’s most important doctor,” and he is, to more than a million patients.

That’s because the 55-year-old American doctor is the only surgeon for 1.3 million people in the Nuba Mountains of Sudan—a region nearly twice the size of Massachusetts. A Catholic medical missionary in a region torn apart by war, he was just awarded the annual Gerson L’Chaim Prize for “outstanding Christian medical missionary service” from Florida-based nonprofit African Mission Healthcare.

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The international medical missionary prize comes with a $500,000 award—money Catena plans to use to cover some of the running costs of his hospital and to help establish a school to train nurses, midwives and clinical officers (the equivalent of physician assistants in the U.S.).

“The $500,000 goes a long way here,” Catena said via email to FierceHealthcare. “We feel the school is key to just start creating a cadre of trained Nuba health professionals.”

Tom Catena, M.D., with a mother and young child
(African Mission Healthcare)

Catena, who grew up in upstate New York, has served in Africa for more than 20 years. Since 2008, he has practiced in Gidel in the Nuba Mountains, living in the middle of the war-torn and besieged territory, which is fiercely contested by its inhabitants and the former government of Sudan.

“The two things that keep me here are the toughness and resilience of the Nuba people and what I see as my role as a medical missionary,” he said.

“My role is simply to show the love of Christ to others and that can only be done by sticking it out during the times that are most difficult.”

In 2011, when the fighting started in Sudan and the capital began bombing its own people in the southernmost region, all of the other expatriate workers in the country left, he said. It was the missionaries who stayed with the people.

Catena was among those who insisted on staying. A graduate of Duke University Medical School and a former U.S. Navy doctor, Catena has been the medical director at Gidel Mother of Mercy Hospital, which he helped establish.

The 435-bed Catholic hospital is the only major medical facility in the Nuba Mountains, and Catena is on call 24/7, sometimes seeing as many as 350 or more patients in a single day.

At one point subject to bombings by Sudanese fighter jets, the hospital compound now has several foxholes where patients and staff can flee in case of more attacks.

A deserving recipient

The L’Chaim (Hebrew for “to life”) Prize is sponsored by Jewish philanthropists Rabbi Erica and Mark Gerson.

“Dr. Tom Catena has given up everything that we in the U.S. take for granted in order to bring healthcare to more than a million people who, without him, would otherwise not have access to any medical care,” said Mark Gerson, co-founder of African Mission Healthcare, which has been supporting mission hospitals in Africa since 2010, in an announcement about the 2019 award.

The L’Chaim prize, launched in 2016, comes with the world’s largest annual award of its kind dedicated to direct patient care in Africa. Catena will receive the award April 14 at a dinner in New York City.

“For more than a decade, Tom has endured bombings, epidemics, rainy seasons and flooding, loss of power, lack of equipment and staff, and very little connection with the outside world, all because of his dedication to the Nuba people. He exemplifies what it means to ‘walk in all God’s ways and to love Him,’ and we are honored to be his partner in his sacred work,” said Rabbi Erica Gerson, who co-sponsors the prize with her husband.

The $500,000 award will go toward the Nuba 2020 campaign, the goal of which is to raise $7.5 million to keep the only major hospital in the Nuba Mountains fully operational for the next two decades. The money also will help strengthen and expand the hospital and its network of clinics, Catena said.

Mark Gerson commented on Catena’s commitment. “The sheer amount of good he does—as measured in clinic visits, surgeries, deliveries, community clinic patients treated, and children vaccinated—with the amount of resources he has is completely stunning. It is simply incredible to even think about how many lives Tom and the team he has built can save and transform with the money that he is provided,” he said.

A challenging place

Catena, who has also had his face on a 2018 Armenia stamp after he was awarded the second annual Aurora Prize for Awakening Humanity, said he was pleasantly surprised to receive the L’Chaim prize.

“The previous awardees had pretty major accomplishments,” he said. “The prize money will give us a big boost and hopefully help to put us on some solid financial footing. We are completely dependent on individual donors yet are the only major referral hospital for a population of over one million.”

Tom Catena, M.D., with his wife Nasima,
a nurse (African Mission Healthcare)

Catena said it is the people who have kept him in Africa. He recalls a young girl, who was about three or four years old and was being cared for by her elderly grandmother. The girl’s mother was killed in an airstrike by the Sudan Air Force, and the grandmother was struggling to look after her. 

“When the girl came to us, she was skin and bones and unable to walk due to TB of the spine. With TB medicines and good care by our nursing staff, the girl was restored to full health,” he said.

His advice for other doctors? “Working in these remote and challenging settings can be very frustrating yet incredibly rewarding,” he said.

But it’s work he recommends others try. “I’d recommend anyone to do perhaps a short-term mission to see if it’s for them and then something more long term, where they would be able to contribute more.”

The Cancer Prevention and Research Institute of Texas (CPRIT) has awarded new grants totaling $1.8 million to two University of Texas at Dallas scientists for their research related to lung and kidney cancers.

The Individual Investigator Awards are among 55 new grants totaling more than $78 million that the institute announced Feb. 19. To date, CPRIT has awarded $2.49 billion in grants to Texas research institutions and organizations through its academic research, prevention and product development research programs.

With the latest grants to the researchers in the School of Natural Sciences and Mathematics, UT Dallas has received nearly $18.5 million from CPRIT to support cancer studies.

CPRIT continues to be an important source of funding for efforts aimed at the prevention and treatment of cancer. The institute's ongoing support of basic research allows UT Dallas scientists to make important contributions toward the fundamental understanding of disease and the improvement of outcomes for cancer patients."

Dr. Joseph Pancrazio, vice president for research and professor of bioengineering at UT Dallas

Dr. Li Zhang, professor of biological sciences and the Cecil H. and Ida Green Distinguished Chair in Systems Biology Science, received $900,000 for lung cancer research. In previous studies, Zhang and her colleagues discovered that cells of the most common type of lung cancer — non-small cell lung cancer — consume substantially more oxygen than normal cells. The lung cancer cells also outpace their normal counterparts in synthesizing a critical chemical called heme, which helps transport and store oxygen. These elevated levels of oxygen and heme fuel tumor growth and progression.

With the new CPRIT grant, Zhang will use advanced imaging techniques in animal models to investigate whether drugs that target heme synthesis and uptake can be a successful strategy for suppressing lung tumors and improving the effectiveness of chemotherapy, radiotherapy and immunotherapy.

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Zhang previously received a CPRIT grant of $900,000 in 2015.

Dr. Jie Zheng, professor of chemistry and biochemistry and the Cecil H. and Ida Green Professor in Systems Biology Science, also received $900,000 for his research, which is aimed at improving the accuracy of computerized tomography (CT)- and fluorescence-guided kidney cancer surgery.

With more kidney cancers being diagnosed in the early stage, partial kidney removal is becoming an increasingly important treatment, in particular for those patients who have poor kidney function or cancer in both kidneys. In current clinical settings, CT is used first to noninvasively localize and stage kidney cancers, followed by fluorescence imaging of normal renal tissue to guide surgery. However, due to the limitations of current contrast agents, no significant improvement in reducing positive margin rates in kidney cancer surgery has been achieved, Zheng said.

Zheng's project will focus on developing a single material, based on gold nanoparticles, that can achieve high contrast in both CT and fluorescence imaging of kidney cancers. His approach takes advantage of the unique physiological microenvironment associated with kidney cancer in a way that allows the tumor margins to be more accurately differentiated during surgical removal. His nanoparticles also have the potential to effectively and selectively deliver anti-cancer drugs to tumors that cannot be treated surgically.

Zheng received three previous CPRIT grants in 2011, 2014 and 2016 totaling nearly $2.4 million.

Source:

University of Texas at Dallas

Emory Healthcare in Atlanta now has the nation’s first 5G-enabled healthcare lab.

The health system is collaborating with Verizon to develop and test 5G Ultra Wideband-enabled medical use cases at its Emory Healthcare Innovation Hub.

It comes on the heels of the U.S. Department of Veterans Affairs announcement earlier this month that it would launch the first 5G-enabled hospital. The VA’s Palo Alto Health Care System, which is an affiliate of Stanford University School of Medicine, also worked with Verizon to bring 5G technology online.

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Emory’s healthcare hub will test how 5G could enhance augmented and virtual reality (AR/VR) applications for medical training, enable telemedicine and remote patient monitoring and provide point-of-care diagnostic and imaging systems from the ambulance to the ER. 

With 5G, doctors should be able to perform tasks like creating holographic 3D anatomical renderings that can be studied from every angle and even projected onto the body in the operating room to help guide surgery, said Tami Erwin, CEO of Verizon Business Group.

The 5G network’s larger bandwidth, faster speeds, and ultra-low latency have the potential to help redefine patient care with real-time data analytics, giving researchers the ability to explore solutions such as connected ambulances, remote physical therapy, and next-generation medical imaging, according to Verizon.

Speed to data is critical to the digital evolution of health,” Scott Boden, MD, vice president for business innovation for Emory Healthcare, said in a statement.

“The healthcare industry, driven by value-based care and increased consumerization, is set for a paradigm shift that will put a much greater focus on connectivity and access to data,” Boden said. 

The Emory Healthcare Innovation Hub was set up in 2018 to improve patient care and provider experience by using cutting-edge health technology. The hub came about from a partnership between Emory Healthcare and Sharecare to use a demand-driven innovation approached developed with 11ITEN Innovations Partners to identify technology improvements with a focus on the end-user while having an impact on cost, quality, and patient outcomes.

The innovation hub works with nine strategic partners to focus on precision medicine, genetics, trauma/emergency medicine, orthopedics, obesity, and rural access to care through telehealth.

As part of the collaboration, Verizon will offer network and security services, project management, professional consulting services, and managed infrastructure and sit on the Emory Hub Executive Advisory Board.

Verizon operates five 5G Labs in the U.S. and one 5G Lab in London. The Emory Healthcare Innovation Lab is the first 5G lab Verizon has set up on-premises for a customer, and it will be part of an ongoing initiative to co-develop 5G-related use cases.