StemCell Therapy

More than 30 million Americans have diabetes. The vast majority suffer from Type 2 diabetes, which arises when the body doesnt process insulin properly. This causes blood sugar levels to rise and potentially triggers a host of other health problems, like heart disease, kidney disease and loss of vision. The disease has long been characterized as a chronic condition, requiring people to receive regular insulin injections, test their blood sugar levels and take medications.

However, a growing body of evidence suggests that reversing the condition essentially, bringing blood sugar back to a non-diabetic level without meds may be possible through diet and weight loss. In a 2016 study in Barbados, more than half of participants given a low-calorie, low-carb diet, in addition to fibrous fruits and vegetables, were able to reduce their blood sugar to non-diabetic levels. Weight-loss surgery has also been used as a technique to keep diabetes at bay.

But not everybody can control their blood sugar levels without medication, particularly in the diseases later stages. And experts caution that major lifestyle changes involving diet can be difficult for many people to maintain.

Often times, people will go on these very restrictive, low-calorie diets, says Ann Albright, director of the Division of Diabetes Translation at the Centers for Disease Control and Prevention. The evidence is clear that, for a majority of people, those are not sustainable.

Read more:

When Dieting, Should We Be Fasting or Grazing?

The Biggest Factor Behind Obesity May Be One We Don't Want to Hear

Breakfast Might Not Be So Essential After All

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Can You Reverse Type 2 Diabetes with Diet and Weight Loss? - Discover Magazine

Lawton First Christian Church and the Oklahoma Healthy Aging Initiative (OHAI) Southwest Center of Healthy Aging are collaborating to provide the Powerful Tools for Caregivers and Diabetes and Beyond programs.

Both classes will be held at First Christian Church, 701 SW D, beginning the first week of January.

Powerful Tools for Caregivers is a class series designed to help family caregivers take better care of themselves while caring for a family member or friend. In the six weekly classes, caregivers will develop self-care tools to reduce stressors, change negative self-talk, communicate their needs to family members and healthcare or service providers, effectively communicate in challenging situations, deal with difficult feelings, and make tough caregiving decisions. Class participants will have access to a copy of The Caregiver Help Book, developed specifically for the training.

This class will begin at 2 p.m. Jan. 6 and will continue once a week through Feb. 17.

The Diabetes and Beyond Program is a six-week program designed to teach self-management tools to older adults and their caregivers who want to learn how to manage diabetes to improve their overall quality of life. This program is interactive and will teach through small groups, games, activities and support conversations. Lessons include learning about management through meal planning, how diabetes affects the body, how to monitor changes, tips on physical activity and encouraging family involvement.

The diabetes class will begin at 2 p.m. Jan. 8 and will continue once a week through Feb. 12.

Instructors are provided by OHAI, a program of the Donald W. Reynolds Department of Geriatric Medicine at the University of Oklahoma. OHAI serves all individuals who are eligible for its programs without regard to race, national origin, ancestry, color, religion, sex, age or disability, under program guidelines.

To receive additional information or to reserve a spot in the classes, call OHAIs Southwest Center of Healthy Aging at (580) 699-3976.

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Powerful tools for caregivers, diabetes classes on the way - The Lawton Constitution

The significant findings of the DAPA-HF data have been well received in both the scientific and payer communities, but we also need to ensure that patients with diabetes are educated on the signs, symptoms, and risk factors linked with heart failure, said Kiersten Combs, BS, US vice president of Cardiovascular Metabolism at AstraZeneca.

The significant findings of the DAPA-HF data have been well received in both the scientific and payer communities, but we also need to ensure that patients with diabetes are educated on the signs, symptoms, and risk factors linked with heart failure, said Kiersten Combs, BS, US vice president of Cardiovascular Metabolism at AstraZeneca.

Transcript

Can you discuss the importance of the patient-reported outcomes data that was presented at AHA 2019? How do these data contribute to value-based agreements?

Especially in the heart failure patients where these patients are highly symptomatic, the data that we're reporting out, specifically using the Kansas City Cardiomyopathy Questionnaire [KCCQ] score, demonstrates that when patients take FARXIGA, they will actually feel better. So, not only is there an important clinical benefit, but there's also important patient benefit. I believe that will translate into the discussions in the impact that it will have with payers because they're ultimately looking to not only provide better health solutions for their patients, but also lower the cost to serve those patients.

Were hearing more about value-based agreements in the diabetes and cardiovascular area, including agreements directly between manufacturers and large employers or purchasing groups that represent employers. Can you discuss any novel solutions that AstraZeneca is pursuing in this area?

We are extremely proud that we have been thought leaders in this space, not only with the number of value-based agreements we have with payers today, but also that we have these agreements across the breadth of our portfolioso within the cardiovascular metabolic portfolio, but really also across our respiratory and oncology portfolio too. So, when we look at the data, such as what's being presented here, as well as everything else in our portfolio, we are engaging payers across the portfolio seeing what is possible; but I would just add that while the DAPA-HF data has been significant, and well received in the scientific community and in the payer community, I would also say that's not enough. We also need to make sure that we're educating patients on the signs, symptoms, and risk factors associated with heart failure. So, we're also quite proud that we've made an investment in just launching here at AHA 2019, a public service campaign called Diabetes Can Break Your Heart that will do exactly that.

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Kiersten Combs: Educating Patients With Diabetes on Heart Failure Risk Is Key to Effective Care - AJMC.com Managed Markets Network

Abbas Ebadi,1,2 Davide Ausili,3 Ahmed N Albatineh,4 Shahin Salarvand,5 Reza Ghanei Ghashlagh6

1Behavioral Sciences Research Center, Life Style Institute, Baqiyatallah University of Medical Sciences, Tehran, Iran; 2Nursing Faculty, Baqiyatallah University of Medical Sciences, Tehran, Iran; 3Department of Medicine and Surgery, University of Milan-Bicocca, Monza, Italy; 4Department of Community Medicine and Behavioral Sciences, Faculty of Medicine, Kuwait University, Kuwait City, Kuwait; 5Social Determinant of Health Research Center, School of Nursing and Midwifery, Lorestan University of Medical Sciences, Khorramabad, Iran; 6Social Determinants of Health Research Center, Research Institute for Health Development, Kurdistan University of Medical Sciences, Sanandaj, Iran

Correspondence: Reza Ghanei GhashlaghSocial Determinants of Health Research Center, Research Institute for Health Development, Kurdistan University of Medical Sciences, Pasdaran Ave, Sanandaj 6618634683, IranTel +98 9144050284Fax +98 36237511Email Rezaghanei30@yahoo.com

Background: Lifelong self-care is important in particular for patients with diabetes, because preventing diabetes complications can help maintain the quality of life and independence of diabetic patients. Currently, there are 16 self-care tools, the majority of which focus on one part of self-care. Therefore, the aim of this study was to evaluate the psychometric properties of the Farsi Self-Care of Diabetes Inventory (F-SCODI) in Iran.Methods: A cross-sectional study was performed on 400 diabetic patients who were selected by convenience sampling to complete the F-SCODI. In this regard, construct validity was assessed using exploratory factor analysis. Additionally, the internal consistency of the F-SCODI was evaluated by McDonalds omega coefficient and Cronbachs alpha; whereas its stability was assessed by a test re-test approach.Results: In total, four factors were extracted (activity-nutritional behavior, smoking avoidance behavior, illness-related behaviors, and health-promoting behaviors) in the dimension of self-care maintenance, three factors (symptom monitoring, symptom assessment, and symptom recognition) in the dimension of self-care monitoring, two factors (autonomous self-care and consultative self-care) in the self-care management dimension, and two factors (task-specific self-care confidence and persistence self-care) in the dimension of confidence. In this regard, the overall consistencies of the four dimensions were 0.809, 0.767, 0.590, and 0.886, respectively.Conclusion: This study indicated that the Farsi version of SCODI had acceptable internal consistency and reliability as well as content and construct validity. Given the acceptable psychometric properties, this tool can be used in future studies in Iranian patients with diabetes.

Keywords: diabetes, self-care, Self-Care of Diabetes Inventory, factor analysis, Iran

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Psychometric Evaluation of the Farsi Version of the Self-Care of Diabe | DMSO - Dove Medical Press

Mon, Dec 30, 2019 - 5:50 AM

Singapore

DIABETES is one of the fastest growing health challenges of the 21st century. In Singapore, the prevalence of diabetes in adults aged 18 and above has nearly doubled in just 15 years - from 7 per cent in 2004 to 13.7 per cent today (about 606,000 people).

What is perhaps of most concern about the diabetes trend is how unaware people remain of it.

"Diabetes is a silent disease," explained Pedro Goncalves, head of Roche Diabetes Care Region International (APAC, Middle-East, Africa, Russia & LATAM). "You can have it and not know it until it's quite late."

The International Diabetes Federation (IDF) estimates that half of adults with diabetes - about 232 million people worldwide - are undiagnosed, which puts them at high risk of developing serious diabetes-related complications such as blindness, nerve damage, heart disease and limb amputation. Singapore, for example, has the world's highest rate of diabetic kidney failure, despite its proclaimed war on diabetes.

Therefore, companies such as Roche Diabetes Care have begun investing in digital alternatives to help people track their health information.

"It's very important to have information on glucose levels and other markers immediately accessible by doctors and healthcare professionals," said Mr Goncalves. Digital technology that can facilitate quicker, easier consolidation of such information will help diabetes patients get better at staying within healthy ranges, he added.

The advent of digital technology is timely as the cost of diabetes treatment is increasing at double-digit rates throughout Asia. In Singapore alone, over S$1 billion is spent each year on managing diabetes.

Rising trends skew disproportionately towards Type 2 diabetes, driven by growing urbanisation and changing lifestyle habits (higher calorie intake, increasing consumption of processed foods, sedentary lifestyles), said Mr Goncalves. This makes diabetes difficult to detect and manage without regular visits to the doctor.

"It's not about providing only clinical decisions like prescribing, it's really about helping them understand what they do, what they eat, how to balance their lifestyle so they can control the disease better," he said.

Compared to traditional methods, digital technology is able to facilitate not only reactive treatment but also proactive prevention, which many healthcare professionals agree is both more effective and less costly for disease management.

"It's about preventive action and the need to work on the early stage of diabetes, so we avoid this epidemic that is taking place in Asia more than anywhere else in the world," said Mr Goncalves.

One such digital solution is mySugr, a free mobile app for managing diabetes that collects and consolidates relevant therapy data in one place through external devices, integrations, and manual entry. Since its June launch in Singapore, the app has garnered nearly 9,000 users.

"The data allows us to zero in on the areas we can improve on, be it adjusting medicine dosage or food intake, so that I can live as normally as I can," said mySugr user Shane Sim, 31. "It is definitely better than the old school way of using a pen and paper logbook, which is very inconvenient to carry around."

Making diabetes "suck less", as the app's tagline claims, is a helpful motivator for diabetes patients who struggle daily with having to keep track of meals, medications, blood sugar levels and other important data. Said Heng Pei Yan, 33: "I appreciate the constant improvements and modifications made to the app to make blood sugar monitoring fun for users."

"It's a very user-friendly interface," said Eileen Lee, head of communications at Roche Diabetes Care. "It's very gamified, it's very easy to enter your data, so it's not complicated, even for older users."

With the burden of disease management lightened and streamlined by digital solutions, better outcomes can be expected, said Mr Goncalves.

"Patients only see a doctor once per year or twice. It's impossible to manage a disease that is influenced by behaviour if you don't have permanent support," he explained. "We need to find other ways of providing support to people, and that's exactly where digital solutions come in."

For S$210, mySugr also offers a three-month subscription plan for mySugr Coaching, an in-app add-on bundle of special features that includes access to direct communication with diabetes educators for personalised advice between clinic visits.

The growing capabilities of healthcare technology aside, the responsibility to take action remains key to health and disease management.

"Technology is just the bridge to help you do things right," said Mr Goncalves. "But it takes a while for humans to change their behaviour ... and that's always a challenge."

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The war on diabetes goes digital - The Business Times

Poxel and its new diabetes tablet are heading to regulators. Well, in Japan at least.

The French pharma and its Japanese partner Sumitomo Dainippon announced the third straight Phase III success for its new kind of diabetes tablet. In the year-long, open-label TIMES 2 trial, Imeglimin decreased blood sugar levels (HbA1c) as a monotherapy or in combination with one of any of 8 common diabetes drugs. That included a .92% decline when given with DDP-4 inhibitors, one of the most common diabetes meds in Japan.

The results keep Poxelon track for the same 2020 Japanese regulatory submission and 2021 approval they laid out after positive double-blind TIMES 1 results were announced in April. Top-line TIMES 3 results, also positive, were unveiled in November.

The results sent Poxels stock on the European exchange up 9.92% to 9.75 per share.

The TIMES 2 results represent a significant milestone for Imeglimin, with the completion of our robust Phase 3 program in Japan, Poxel CEO Thomas Kuhn said in a statement referring to all three trials. Taken together, these results feature Imeglimins potential to treat type 2 diabetes at multiple stages of the disease.

Poxels path to the rest of the world remains less clear. Nearly two years ago, the French group sold US and European rights to Vivek Ramaswamys Roivant in a deal worth $50 million upfront and up to $600 million in milestones. The deal had been long-sought; although Poxel had a positive Phase IIb in 2014 and dosing levels to go to Phase III, they needed a deeper-pocketed partner willing to take on the heavy financial burden of a global diabetes trial.

Updates, though, have been sparse since they signed that deal in February 2018. The Poxel website still lays out the 2018 plans under the Imeglimin US/EU tab, and the Roivant website refers back to the Poxel one. Clinicaltrials.gov lists only one active trial for the drug a Phase I in Munich for hepatic impaired subjects.

The compound, though, will enter the Japanese regulatory process with a bevy of evidence behind it. Part of a new class of oral chemical agents called glimins, the drug is designed to target all three key organs affected in Type II diabetes: Increasing insulin in the pancreas in a glucose-dependent manner, decreasing excess glucose production in the liver and enhancing insulin sensitivity in muscles.

Collectively, the three trials enrolled 1,100 patients. TIMES 2 was open-label but TIMES 1 and 3 were double-blind. All met primary endpoints.

Poxel should find a ready market, too. An aging population and rising obesity have swelled the number of suspected diabetics in Japan past 10 million as of 2016, according to past estimates from Nikkei.

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Poxel passes 3rd straight Phase III diabetes trial in Japan, but what's going on with US/EU partner Roivant? - Endpoints News

New Delhi [India], Dec 29 (ANI): Nearly 400 people with known diabetes (type-1) below the age of 30 years underwent the comprehensive annual screening for their complication due to their health condition at the annual medical camp- 'Diabetes Onset on Youth' organised with the All India Institute of Medical Sciences (AIIMS) on Saturday.

The participants included children, teenagers, and young adults till 30 years of age from Delhi-NCR for the annual screening process which is a mandatory part of their clinical practices.

Patients were from AIIMS endocrinology department, paediatrics (AIIMS) and Safdarjung Hospital.

"The idea for these camp is that all people with diabetes need to undergo a periodic evaluation and annual evaluation which is more detailed and also includes screening for complications associated with diabetes," (Prof) Dr Nikhil Tandon, head of endocrinology and metabolism department at AIIMS told ANI.

In 2006, ICMR constituted a Registry of People with Diabetes with Young Age at Onset (YDR), to maintain a data- Youth Onset of Diabetes.

"The effort is to try and make it easier for these individuals who already have diabetes. This medical camp at AIIMS is a single-window, single day system and all that patients require for investigation was taken care," Dr Tandon said.

Experts say that Type -1 diabetes occurs usually in the first two decades of life when body's immune system destroys the cell in the pancreas which generates insulin, as a result, these young people do not have insulin production inside their body. So they are compelled to take insulin from outside and dependent on insulin for most of their survival.

On the other hand, Type 2 diabetes occurs mostly in the older age group population. Under this health complication, the initial part of the disease is associated with a condition called- insulin resistance diabetes, though the body produces insulin the body's tissue remains insensitive towards action. Therefore, doctors need to give more & more insulin and hence a lot of time, we need to give medications by mouth is also enough to take care of the process.

In 2014, AIIMS started the annual evaluation of people with known diabetes wherein 200 participants got registered and underwent annual health check for the complications associated with the disease. The project is funded by ICMR.

Investigations like haemoglobin A1c, proteinuria or microalbuminuria in urine, cholesterol, and fats in the blood (lipid profile) and kidney function test were performed on participants.

Citing the latest data published in the International Diabetes Federation (IDF), Dr Tandan said that currently, India has a burden of 100,000 people suffering from known diabetes.

Patients and attendants were sensitised through nukkad nataks, poster making, and quiz competitions. (ANI)

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AIIMS conducts annual screening of about 400 people with Type-1 diabetes - Yahoo India News

Wow, you survived cancer? What's your secret to health care?

As absurd as that sounds, its a question many Americans who get sick are still asking as we ring in the year 2020. Getting health care in this country is still so circuitous it often does feel like a secret a maze deciphered in private that's never quite mastered. The reward for solving it? Perhaps your life; perhaps the loss of your life savings. And thats if youre lucky.

Even with the Affordable Care Act, almost 30 million are without health insurance in the U.S. And if youve perused plans on the ACA marketplace, youll know why. Theyre pricey, and a new year brings fears that insurance premiums are once again rising. (Who knew the inflation rates on a pap smear were that high?!) Meanwhile, 14 Republican-led states are still refusing to expand Medicaid as stipulated in the ACA, even though the federal government would pay for 90 percent of the cost. Why? Something about repeal and replace or socialism. Its hard to keep track.

Even with the Affordable Care Act, almost 30 million are without health insurance in the U.S. And if youve perused plans on the ACA marketplace, youll know why.

I traveled to three states, each with their own unique health care access challenges, for my new MSNBC special "Red, White, and Who? Between Texas, New York and Utah there are major differences in how easy it is to see a doctor without going bankrupt. But every single person I spoke with regardless of job, socioeconomic status or even political affiliation had one identical anxiety: healthcare in one of the most advanced countries in the world is ridiculously, hopelessly complicated.

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Im retired, but I feel like a have a job, Larry Chiuppi told me sitting outside at an RV park in Houston, blocks from one of the top cancer treatment hospitals in the country. Larry has been caring for his wife Nancy Raimondi, who has blood cancer, for over a year. During that time, he himself was diagnosed with prostate cancer. Even with her Medicare and his private health plan under the ACA, navigating the billing systems for the endless hospital visits, specialists and tests each with their own separate charges requires a huge amount of time and vigilance. He tells me they once got a $14,000 bill for a stem cell transplant because someone forgot to link Nancys Medicare. Larry imagined many people wouldve just tried to pay it. And most Americans dont have a retirees free time and Larrys persistence to help them through the bureaucracy, an added burden of getting well.

We also dont all have a mother like Sandra Stein. She and her family live in New York, a state where the uninsured population is less than five percent, and 6.5 million are on Medicaid. I met Sandra on a street corner in upper Manhattan, where activists were flyering for the New York Health Act, a bill that would give every New Yorker state-funded care. Sandra believes in single-payer healthcare because she has experienced the mind-numbing labyrinth that is the private insurance system firsthand.

When her son was nearly three, he developed a rare neurological disease that left him unable to walk or speak. At the time, she and her husband had private insurance, which was relatively good insurance, according to Sandra. But that didnt make things easier. When they first went to the hospital in an ambulance, the doctors there didnt take their insurance even though the hospital did. Her son ultimately stayed in three different hospitals over the course of 15 months.

When we got home it was my job to figure out the pile of bills and the collections threats, she told me. Its been eight years, but Sandras voice cracked like the memory happened yesterday. I couldnt imagine how hard it mustve been to be afraid for your childs life while collections agents breathed down your neck. Sandra says the billing department sought her out even while her son was in the ICU, and that there were so many billing errors that she ultimately asked for an audit.

And yet, Sandra, Larry and Nancy are the lucky ones. They have health insurance, and they have the time and resources to be able to make their way through the bureaucratic hall of mirrors and toward a fighting chance at getting well.

Its this cruel opacity of the private insurance system, on top of the rising monthly costs of just having a plan, that can be the difference between life and death. And it keeps a surprising number of Americans away from the system altogether. Like a rodeo cowboy I met in Texas, whose story youll just have to watch (Im not spoiling it all!). Its also led Americans like Sandra to believe that a massive simplification of our health care system is far overdue.

For many, that simplification comes in the form of cutting out the profit motive and moving toward government-funded insurance, like Medicare for All, which Big Pharmas enemy number one Sen. Bernie Sanders and I hashed out over bagels in a New York City deli.

Ultimately what became clear through my travels is that healthcare in America is often overpriced and even dysfunctional, but its the lack of transparency that can be the most insidious. You pretty much have to be a health care policy expert, or have a loved one who can quit their job to become one, in order to ensure proper help.

Its also strange that in a country that loves the free market as much as we do, we the consumer have no idea how much anything costs when we walk into a hospital. Why would we? Our health is priceless, so we are simply at the mercy of an ineffective system. That is, unless we fight for something different.

Red, White, and Who premieres on MSNBC on Dec. 29 at 9 p.m. E.T.

Francesca Fiorentini is journalist and comedian. She is the host of "Red, White and Who?" on MSNBC and Newsbroke on AJ+. Follow her @franifio.

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Health care in America is dysfunctional but its lack of transparency is downright dangerous - NBC News

No matter how packed with all that makes the game greata golf year might be and Id contend that theres never been a year that wasnt it wont stand out in history unless it has one extra special moment. And 2019 had that.

Golfs broad strokes rarely cut as wide a swath through culture as the Big 3 team sports, but Tiger Woods victory at the Masters was voted The Associated Presss top sports story of the year. Its importance may come to be considered greater than his 1997 Masters victory, or his U.S. Open wins at Pebble Beach in 2000 and Torrey Pines in 2008. And as a comeback from adversity, Woods first major victory in 11 years at least rivals and arguably surpasses not only Ben Hogans 1950 U.S. Open, but ANY sports comeback ever. It was extra special.

But so much else happened in 2019. What to make of the rest?

Well, lets start with Woods. Its easy to forget that a lot of things had to go just right on Sunday for Tiger to win at Augusta. And that as much as the place is considered his sweet spot, he hadnt won there since 2005. For me, the singular moment that meant more going forward was the Zozo Championship in November. Not because it became Woods record-tying 82nd career. And despite the tournament being a limited field, off-season event over a short golf course in a faraway land.

Was there any doubt? For our No. 1 Storyline of the Year, we look back on Tiger Woods' 15th major title at the Masters Tournament.

It was the WAY that Tiger played with a return to an ease and smoothness in his action that not only recalled much earlier days, but which promises repeatability and consistency. As well as on the right occasions dominance.

Next on the hit parade the education of Rory McIlroy. The four-time major winner added important elements to his game namely better putting and overall ball control to set the foundation for another sustained run of greatness in his 30s.

But it was also a year of searching. McIlroy came into 2019 reflective and open to new ideas. He said meditation, juggling and several self-help books had led him to decide that he would no longer allow my score to define who I am as a person. His consistency improved and he impressively won The Players in March. But McIlroy also had several flattish Sundays with chances to win, and the Masters where he continues to chase the career Grand Slam didnt go so well.

Prior to the U.S. Open, McIlroy roared to a seven-shot win in Canada. But he tied for ninth at Pebble Beach. Expectations were again high at Portrush, a short car ride from his boyhood home and where he had shot the course record of 61 at age 16. He opened The Open with a nervous 79 and missed the cut. The next week he got boat-raced by winner Brooks Koepka in a final Sunday pairing at the WGC-FedEx in Memphis.

It was again time to reassess.

After winning the FedExCup at East Lake, this time outplaying Koepka in the last group in what he would later call the highlight of his year, McIlroy revealed having committed to a harder and more self-aware competitive edge.

I think one of the biggest things is sometimes Ive tried to treat Sundays the same as a Thursday or Friday, and theyre not, said McIlroy, who would go on to win WGC-HSBC in Shanghai in November for his fourth victory of the year. Ive gone into them maybe a little too relaxed, but its not the same, and its about trying to get yourself in the right mindset. I guess thats the ultimate compliment I can give Brooks is that I wanted to be a little bit more like him.

McIlroy on Koepka rivalry: Feels good to take down No. 1

Speaking of Big Game Brooks, his ruthless march through the major championships since 2017 has been undervalued. In the last 30 years, only Woods, McIlroy and Nick Faldo have had such prolonged periods of excellence in the biggest events.

This year, Koepka showed true dominance in building a seven-stroke lead through three rounds in his victory at the PGA at Bethpage. That he bookended that performance with seconds at the Masters and at the U.S. Open got short shrift. And after he finished fourth at Portrush, when his putter uncharacteristically failed him (and he was being bothered by a torn patella tendon in his left knee that required stem cell treatment and from which he is still recovering), too many acted as though his reign had ended.

That impression was strengthened when McIlroy was chosen as PGA Tour Player of the Year by a vote of his peers. In the last couple of years, Koepka has used relatively small slights for fuel. But going into 2020 and turning 30 in May, he will be on a mission to strengthen his hold on world No. 1 and outdo McIlroy in the process. Koepka betrayed some saltiness in October by pointing out that, Ive been out here for what, five years. Rory hasnt won a major since Ive been on the PGA Tour. So I dont view it as a rivalry.

Sounds like a rivalry.

Although Jon Rahm, who enters 2020 at No. 3 in the world, is expected to intrude.

The 25-year-old Spaniard earns the description beast in the same way as team sport athletes who appear physically overwhelming. Along with his nine combined victories on the PGA and European tours, Rahm has also validated his combination of power and touch with a relentless consistency in his first 89 official worldwide professional starts, Rahm has 44 top-10s, only one less than Woods in his first 89. As he continues to mature and he got married just this month expect a calmer, more controlled Rahm to be even more dangerous.

In the womens game, Jin Young Ko was by far the best player of the year, winning two majors and two other events in only her second season on the LPGA tour. In a gracious acceptance speech for year-end honors at the tours awards banquet, the 24-year-old South Koreans accented, but precise English reflected the same discipline and exactitude that is so evident in her game. The current Rolex No. 1 knows thats been a precarious perch over the last decade in womens golf, and she seems determined to change the cycle. This is not the end, she told the gathering, but only the beginning.

Ok, thats the highest profile stuff. But there was also a pervasive theme that permeated 2019. In so many ways, it was an extraordinarily feel-good year.

Usually in these end-of-the-year assessments, what sticks with me most and reinforces my generally tragic sense of competitive golf are the deeply wounding, self-induced losses brought on by late implosions. You know, Phil Mickelson at Winged Foot, Adam Scott at Royal Lytham & St. Annes, and Jordan Spieth at the 2016 Masters with plenty of other examples to stuff into the hurt locker. But as I remember 2019, only two players caused such sadness, Francesco Molinari at the Masters and Lizette Salas at the Womens British Open. Molinari, the seemingly unflappable ball-striking machine led by two strokes on the 12th tee Sunday at the Masters before mishitting an 8-iron into Raes Creek, opening the door for Woods. Salas, who played the best golf of her life with a closing 65 at Woburn, missed a 5-foot birdie putt on the 72nd hole, and then watched Hinako Shibuno win it with a 20-footer.

Instead of a bevy of heartbreak, we got a full complement of Capra-esque moments.

Winning putt: Pettersen clinches the Solheim Cup for Europe

Suzann Pettersen, after making an 8-footer on the final green in the last match that spelled the difference between winning and losing the Solheim Cup, announced her retirement at age 38. One of the great walk-offs ever in professional sports. Pettersen said she reached the decision spontaneously with the thought, This is it. This is the peak.

Shane Lowry, as an underachiever scarred by a Sunday failure at the 2016 U.S. Open, shouldering the immense mental load before thousands of home fans in a land that hadnt held the Open Championship since 1951, and winning by six. The panorama on Portrushs 72nd hole, with fansrunning up the fairway behind Lowry, some waving Irish flags in the rain amid a constant roar, was one of pure cathartic release.

Shibuno winning the Womens British Open at Woburn in her first professional tournament outside Japan. A babe in the woods at 20, she was bolstered by innocence and a constant, infectious smile, even as she four-putted early in the final round. Shibunocaught fire and closed with a 31 on the final nine, her final putt rammed in with a blissful freedom, to become the second Japanese player to win a major championship.

The scene at the inaugural Augusta National Womans Amateur, where the image of women striding the hallowed grounds was a transformative moment for the game. The impressive brand of head-to-head power golf played by winner Jennifer Kupcho and runner-up Maria Fassi was the icing on the cake.

The effervescent Helen Alfreddson winning the second U.S. Senior Womens Open at Pine Needles, the most joyous, about time and appreciated championship in golf. Love of the game is never more palpable than among too-long-ignored 50-and-over LPGA veterans, and Alfreddsons passion and exuberance spoke for them all.

Cameron Champ won the Safeway Open in October while dedicating his play to his gravely ill African-American grandfather, Mack, who started him in the game. The 24-year-old bombers calm as he garnered his second victory was reminiscent of Ben Crenshaws march to the 1995 Masters after being a pallbearer at the funeral of his teacher, Harvey Penick, earlier that week.

In the most exciting finish of the year, Matthew Wolff he of the fascinatingly powerful swing and unofficial leader of the games latest youth movement in only his third pro start, won the 3M Championship with an eagle on the 72nd hole to beat Bryson DeChambeau, who had also eagled the last, by one.

Finally and excuse my darkness Koepka and Rahm saving big victories after blowing huge Sunday leads. For some reason, nothing makes me happier (or more accurately relieved) than seeing a player who has gone from the zone to full meltdown, and then reverse what suddenly looks like his or her inevitable and awful fate in the nick of time. Koepka dug to the very bottom of his deep reservoir of poise to do it at Bethpage after four straight bogeys on the final nine had him lose all but one of his seven-stroke lead. Rahm had a five shot lead with 10 to play at the DP World in Dubai, but it was all gone thanks especially to a couple of knuckleheaded three-putts from inside 25 feet when he reached the 72nd hole. Hell remember that birdie with a smile and a shudder for the rest of his life.

Adding additional poignancy to our main theme, it was also the year of journeymen each capable, but with a history of struggle at the highest level seizing the day.

There is a fine line between success and slump in professional golf. It took only one swing to send Brendon Todd over that line and years to make it back.

Brendan Todd ran away with this category, returning from nearly four years in the wilderness that included a stretch of missing 37 of 41 cuts, to win back-to-back at Bermuda and Mayakoba, and then nearly won again at the RSM. The 34-year-old, who won the Byron Nelson in 2014, came down with a nightmare dose of the swing yips (the lose-it-way-right strain) that by late-2018 had him on the verge of giving up pro golf and opening a pizza franchise. Instead, Todd got some help from swing coach and former player Bradley Hughes and pulled off one of the great turnarounds in golf history.

And consider this roll call of others who went through storybook lost-and-found cycles to convert a week of magic into first victories that take them into 2020 with transformed lives: Max Homa (Wells Fargo Championship), JT Poston (Wyndham Championship), Nate Lashley (Rocket Mortgage), Lanto Griffin (Houston Open), Tyler Duncan (RSM Classic), Adam Long (Desert Classic). Inspirations all.

And at the risk of belaboring the feel-good point, it seemed that just about every level of pro golf ended the year on a happy note.

At the PGA Tours finale at East Lake, McIlroy spread much joy in Ponte Vedra, with one fell swoop validating the wisdom of the Tours more compressed and earlier finishing schedule, getting the new staggered start scoring system at the Tour Championship off on the right foot, and winning in the final group in another showdown with Koepka.

The LPGAs season ended on a high note with Sei Young Kim making a 22-foot birdie putt on the last hole to win the richest first-place prize ever in the womens game $1.5 million at the CME Group Tour Championship. A new format had been questioned for seeming to put sheer money over an equitable reward for season-long performance, but Kims stature as a top player and the cliffhanger nature of her victory over Charley Hull made for a satisfying result.

The PGA Tour Champions season ended with a bang when Jeff Maggert holed out from 123 yards for eagle to win the Charles Schwab Cup Championship in sudden-death.

And at the last big event of the year, the Presidents Cup, Woods was fittingly triumphant as both captain and player. And, as he has done more with age, a strong display of emotion spread the joy.

So finally, did something happen that set the tone for all this happiness? Was there a beginning?

Amy Bockerstette, a 20-year-old golfer with Down syndrome, got to play the iconic 16th hole at the Waste Management Phoenix Open and made par with Gary Woodland watching.

To say there wasnt would be to underestimate the impact of Amy Bockerstette, a 20-year-old collegiate golfer and Special Olympics athlete with Down syndrome, who in January played the 16th hole with Gary Woodland at the pro-am of the Waste Management Phoenix Open. Ill admit it, tears fill my eyes each time I watch the 2-minute and 50-second video, which has reached double-digit million views.

Seeing the way Bockerstette, clearly thrilled to meet her playing partners, Woodland and Matt Kuchar, reveled so genuinely as the center of attention on golfs iconic stadium hole, and then stepped up, assertively telling herself, I got this, is irresistible. She hit a good tee shot, followed with a deft bunker shot, and then, again repeating her mantra out loud, drilled the 10-footer for par with Nicklausian poise.

One guess at the phrase Woodland told himself before pulling off the shot of the year a perfectly clipped 60-degree wedge off the 17th green at Pebble Beach that carried and spun to within 4 feet and a crucial par.

Said Woodland of Bockertette: Theres nobody that Ive seen be in the moment as much as she is.

In a particularly feel-good year, it might have been the most extra special moment of all.

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All the feels: From start to finish, a year of emotional stories - Golf Channel

For decades, the DNA of living organisms such as corn and salmon has been modified, but Crispr, invented in 2012, made gene editing more widely accessible. Image: YinYang/IStock.com via AFP Relaxnews

In the summer, a mother in Nashville with a seemingly incurable genetic disorder finally found an end to her suffering by editing her genome.

Victoria Grays recovery from sickle cell disease, which had caused her painful seizures, came in a year of breakthroughs in one of the hottest areas of medical research gene therapy.

I have hoped for a cure since I was about 11, the 34-year-old told AFP in an email.

Since I received the new cells, I have been able to enjoy more time with my family without worrying about pain or an out-of-the-blue emergency.

Over several weeks, Grays blood was drawn so doctors could get to the cause of her illness stem cells from her bone marrow that were making deformed red blood cells.

The stem cells were sent to a Scottish laboratory, where their DNA was modified using Crispr/Cas9 pronounced Crisper a new tool informally known as molecular scissors.

The genetically edited cells were transfused back into Grays veins and bone marrow. A month later, she was producing normal blood cells.

Medics warn that caution is necessary but, theoretically, she has been cured.

This is one patient. This is early results. We need to see how it works out in other patients, said her doctor, Haydar Frangoul, at the Sarah Cannon Research Institute in Nashville.

But these results are really exciting.

In Germany, a 19-year-old woman was treated with a similar method for a different blood disease, beta thalassemia. She had previously needed 16 blood transfusions per year.

Nine months later, she is completely free of that burden.

For decades, the DNA of living organisms such as corn and salmon has been modified.

But Crispr, invented in 2012, made gene editing more widely accessible. It is much simpler than preceding technology, cheaper and easy to use in small labs.

The technique has given new impetus to the perennial debate over the wisdom of humanity manipulating life itself.

Its all developing very quickly, said French geneticist Emmanuelle Charpentier, one of Crisprs inventors and the cofounder of Crispr Therapeutics, the biotech company conducting the clinical trials involving Gray and the German patient.

Cures

Crispr is the latest breakthrough in a year of great strides in gene therapy, a medical adventure started three decades ago, when the first TV telethons were raising money for children with muscular dystrophy.

Scientists practicing the technique insert a normal gene into cells containing a defective gene.

It does the work the original could not such as making normal red blood cells, in Victorias case, or making tumor-killing super white blood cells for a cancer patient.

Crispr goes even further: instead of adding a gene, the tool edits the genome itself.

After decades of research and clinical trials on a genetic fix to genetic disorders, 2019 saw a historic milestone: approval to bring to market the first gene therapies for a neuromuscular disease in the United States and a blood disease in the European Union.

They join several other gene therapies bringing the total to eight approved in recent years to treat certain cancers and an inherited blindness.

Serge Braun, the scientific director of the French Muscular Dystrophy Association, sees 2019 as a turning point that will lead to a medical revolution.

Twenty-five, 30 years, thats the time it had to take, he told AFP from Paris.

It took a generation for gene therapy to become a reality. Now, its only going to go faster.

Just outside Washington, at the National Institutes of Health (NIH), researchers are also celebrating a breakthrough period.

We have hit an inflection point, said Carrie Wolinetz, NIHs associate director for science policy.

These therapies are exorbitantly expensive, however, costing up to $2 million meaning patients face grueling negotiations with their insurance companies.

They also involve a complex regimen of procedures that are only available in wealthy countries.

Gray spent months in hospital getting blood drawn, undergoing chemotherapy, having edited stem cells reintroduced via transfusion and fighting a general infection.

You cannot do this in a community hospital close to home, said her doctor.

However, the number of approved gene therapies will increase to about 40 by 2022, according to MIT researchers.

They will mostly target cancers and diseases that affect muscles, the eyes and the nervous system.

Bioterrorism

Another problem with Crispr is that its relative simplicity has triggered the imaginations of rogue practitioners who dont necessarily share the medical ethics of Western medicine.

Last year in China, scientist He Jiankui triggered an international scandal and his excommunication from the scientific community when he used Crispr to create what he called the first gene-edited humans.

The biophysicist said he had altered the DNA of human embryos that became twin girls Lulu and Nana.

His goal was to create a mutation that would prevent the girls from contracting HIV, even though there was no specific reason to put them through the process.

That technology is not safe, said Kiran Musunuru, a genetics professor at the University of Pennsylvania, explaining that the Crispr scissors often cut next to the targeted gene, causing unexpected mutations.

Its very easy to do if you dont care about the consequences, Musunuru added.

Despite the ethical pitfalls, restraint seems mainly to have prevailed so far.

The community is keeping a close eye on Russia, where biologist Denis Rebrikov has said he wants to use Crispr to help deaf parents have children without the disability.

There is also the temptation to genetically edit entire animal species malaria-causing mosquitoes in Burkina Faso or mice hosting ticks that carry Lyme disease in the US.

The researchers in charge of those projects are advancing carefully, however, fully aware of the unpredictability of chain reactions on the ecosystem.

Charpentier doesnt believe in the more dystopian scenarios predicted for gene therapy, including American biohackers injecting themselves with Crispr technology bought online.

Not everyone is a biologist or scientist, she said.

And the possibility of military hijacking to create soldier-killing viruses or bacteria that would ravage enemies crops?

Charpentier thinks that technology generally tends to be used for the better.

Im a bacteriologist weve been talking about bioterrorism for years, she said. Nothing has ever happened.IB/JB

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2019: The year gene therapy came of age - INQUIRER.net

Global Gene Therapy MarketResearch Report 2020-2029 is a vast research database spread across various pages with numerous tables, charts, and figures in it, which provides a complete data on the Gene Therapy market including key components such as main players, size, SWOT analysis, business situation, and best patterns in the market. This analysis report contains different expectations identified with income, generation, CAGR, consumption, cost, and other generous elements. Further, the report determines the opportunities, its restraints as well as analysis of the technical barriers, other issues, and cost-effectiveness affecting the market during the forecast period from 2020 to 2029. It features historical & visionary cost, an overview with growth analysis, demand and supply data. Market trends by application global market based on technology, product type, application, and various processes are analyzed in Gene Therapy industry report.

The Top Players Functioning in the Gene Therapy market are Novartis, Kite Pharma Inc, GlaxoSmithKline PLC, Spark Therapeutics Inc, Bluebird bio Inc, Genethon, Transgene SA, Applied Genetic Technologies Corporation, Oxford BioMedica PLC, NewLink Genetics Corp., Amgen Inc.

To obtain all-inclusive information on forecast analysis of global Gene Therapy Market, request a Free PDF brochure here:https://marketresearch.biz/report/gene-therapy-market/request-sample

Gathering information about Gene Therapy Industry and its Forecast to 2029 is the main objective of this report. Predicting the strong future growth of the Gene Therapy Market in all its geographical and product segments has been the oriented goal of our market analysis report. The Gene Therapy market research gathers data about the customers, marketing strategy, competitors. The Gene Therapy The manufacturing industry is becoming increasingly dynamic and innovative, with more private players enrolling in the industry.

Identifying The Basic Business Drivers, Challenges, And Tactics Adopted:

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Various challenges overlooking the business and the numerous strategies employed by the industry players for successful marketing of the product have also been included.

Market Segmentation Based on vector type, gene type, application, and region:

By Vector: Viral vector Retroviruses Lentiviruses Adenoviruses Adeno Associated Virus Herpes Simplex Virus Poxvirus Vaccinia Virus Non-viral vector Naked/Plasmid Vectors Gene Gun Electroporation Lipofection By Gene Therapy: Antigen Cytokine Tumor Suppressor Suicide Deficiency Growth factors Receptors Other By Application: Oncological Disorders Rare Diseases Cardiovascular Diseases Neurological Disorders Infectious disease Other Diseases

Furthermore, Gene Therapy industry report covers chapters such as regions by product/application where each region and its countries are categorized and explained in brief covering: North America, Europe, South America, Asia Pacific, and the Middle East and Africa.

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Benchmarking: It includes functional benchmarking, process benchmarking, and competitive benchmarking

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Technological Intelligence: It helps you to investigate future technology roadmaps, choose the right technologies, and determine feasible technology options

The following years taken into consideration in this research to forecast the global Gene Therapy market size are as follows:

Base Year: 2019 | Estimated Year: 2020 | Forecast Year: 2020 to 2029

TOC of Gene Therapy Market Report Includes:

1. Industry Overview of Gene Therapy

2. Industry Chain Analysis of Gene Therapy

3. Manufacturing Technology of Gene Therapy

4. Major Manufacturers Analysis of Gene Therapy

5. Global Productions, Revenue, and Price Analysis of Gene Therapy by Regions, Creators, Types and Applications

6. Global and Foremost Regions Capacity, Production, Revenue and Growth Rate of Gene Therapy

7. Consumption Value, Consumption Volumes, Import, Export and Trade Price Study of Gene Therapy by Regions

8. Gross Margin Examination of Gene Therapy

9. Marketing Traders or Distributor Examination of Gene Therapy

10. Global Impacts on Gene Therapy Industry

11. Development Trend Analysis of Gene Therapy

12. Contact information of Gene Therapy

13. New Project Investment Feasibility Analysis of Gene Therapy

14. Conclusion of the Global Gene Therapy Industry 2020 Market Research Report

CONTINUE

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Gene Therapy Market 2020: New Innovative Solutions to Boost Global Growth with New Technology, Busin - PharmiWeb.com

For biotech, 2019 ended like the penultimate episode of a prestige TV show. We got answers to some weighty questions, but mostly, the year left a breadcrumb trail to some major reveals.

The IPO window stayed open, helping scores of companies go public. Futuristic therapies proved their worth in clinical trials, pointing to a new era in medicine. And the markets ended the year on a high, buoyed by a Food and Drug Administration that seems ever more flexible when it comes to approving new drugs.

Now, with 2020, well get the more important answers. Sure, theres a lot of public biotech companies now, but what if thats a bad thing? Yes, cell and gene therapies look transformational, but what if they never make any money? And since when is everyone so confident they understand whats going on inside the FDA?

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Here are three trends to watch in biotech in 2020, a year that looks to be laden with opportunities and stumbling blocks for the drug industry.

While every biotech startup is undoubtedly special in the eyes of the venture capitalists quoted in its press releases, 2020 could be a year marked by fatigue for the outside public.

More than 140 biotech companies have gone public since 2017, according to the analysts at Evercore ISI, and now theres upward of 500 of them trading on the Nasdaq. Keeping tabs on them all is essentially impossible, and its become fairly commonplace for biotech types to see the name of a given company for the first time by reading about its implosion.

Thats arguably a good problem to have in societal terms. More biotech companies means more efforts to treat human disease. But it could be problematic for the herd. Drug development remains an expensive proposition, and the majority of the biotech companies that went public in the past three years have negligible or nonexistent revenue. That means theyre going to have to go back to the market with follow-on offerings, and they may not like what they find.

According to Cowens biotech thermometer, a regular update on Wall Street sentiment, investors are increasingly selective when it comes to equity offerings, spooked by slumping IPO returns and a glut of supply. If that trend continues into 2020, some of those 500-plus biotech companies might need to look for other means of keeping the doors open, including mergers that thin the flock.

Much of the conversation around cell and gene therapies has focused on how much they cost, and understandably so. Two million dollars is, objectively, a lot of dollars. But the anxiety in biotech circles is a bit different: Is anyone going to make money on these things?

Take, for instance, CAR-T cancer therapy. For some patients, a single dose erases any trace of aggressive, otherwise untreatable cancer. For every patient, a single dose costs about $400,000. That sounds like a lot, but churning out a genetically engineered immune cell is hardly akin to widget manufacture. CAR-T companies dont disclose their underlying costs, but these therapies are understood to be low-margin products.

Theyre also considered commercial disappointments. The first two approved CAR-Ts, Kymriah and Yescarta, have underperformed analyst expectations to date. And that has stoked concern that a coming wave of gene therapies could face similar commercial difficulties.

Like CAR-T, gene therapy is costly to make, can be administered only at certain sites, and has made headlines for its six- or seven-figure list prices. Biotech companies and their investors have staked billions of dollars on the idea that such one-time treatments can become lucrative products. If that assumption is incorrect and the industry cant figure out how to make money in therapy, there could be a painful knock-on effect for biotech.

Handily, theres a one-company test case to follow in 2020. Novartis (NVS) sells a CAR-T in the form of Kymriah and a gene therapy called Zolgensma. Furthermore, thanks to a recent $9.7 billion acquisition, it will likely soon sell an RNAi treatment for high cholesterol. Each endeavor is a bet that futuristic science can turn into money-making medicines. By the end of the year, well have a decent idea of whether its a wise one.

Remember 2015, when the FDA would approve or reject a drug, and people would form an opinion and move on? That all changed the following year when the agency approved eteplirsen, now called Exondys 51, which is a treatment for Duchenne muscular dystrophy from a company called Sarepta Therapeutics (SRPT).

Without relitigating the whole ordeal, its fair to say Sareptas case relied on scant, debatable evidence from a small trial. To some, the FDAs decision to approve eteplirsen anyway was a sign of forward-thinking regulation that put patients first. To others, it was a dereliction of duty that threatened to erode decades of pharmaceutical jurisprudence. And to a great many, it was reason to get on the internet and be churlish, conspiratorial, and even threatening.

On Twitter, the fight over eteplirsen has never really ended, just taken on different forms, like a biotech analog to Gamergate. Earlier this year, the debate over a heart drug made by Amarin (AMRN) quickly metastasized into eteplirsen redux, with name-calling, accusations of bad faith, and armchair psychoanalysis of FDA staff. There were smaller but similar fights over Axovant Sciences, Clovis Oncology (CLVS), and nearly every biotech company with a sizable short interest.

Its at least somewhat understandable why eteplirsen marked such a shift in biotech discourse. Where FDA past decisions seemed to come down from Mount Sinai with little in the way of transparency, the messy eteplirsen process made public internal infighting and clashing personalities at the agency. The FDAs top drug evaluator even considered Sareptas balance sheet while evaluating the drug, a departure from the agencys hands-off approach to the business of biopharma and evidence that approval decisions can be about more than benefits and risks.

Theres no evidence that the FDA was fundamentally changed by a single decision, as organizations that employ 17,000 people rarely are. But that peek behind the curtain was enough to give credence to seemingly any biotech bull case online. Where the FDA once appeared monolithic, now there were heroes and villains within, actors whose imagined biases could support any conspiracy theory. Formerly anonymous public servants became the topic of vicious debate among strangers with alphanumeric Twitter handles and pictures of dogs as online avatars. One even got called a cuck.

With all that as a backdrop, next year, Biogen (BIIB) is going to ask the FDA to approve aducanumab, a treatment for Alzheimers disease. The supporting data are confusing, drawn from a pair of terminated trials with divergent results. The agencys decision will have major implications for the drug industry, the health care system, and the more than 5 million Americans with Alzheimers.

And, on the fractious little planet that is biotech Twitter, aducanumab presents an opportunity to play out the eteplirsen debate on the grandest scale yet, with more kremlinology, more circular logic, and more vitriol. Be nice to one another out there.

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3 trends in biotech to watch in 2020 - STAT

2019 was an eventful year in the BioHealth Capital Region (BHCR), to say the least, and BioBuzz has been there to document it every step of the way.

Whether it was the retirement of the MedImmune brand, Viela Bios $150M IPO, the $1.2B acquisition of Paragon Bioservices, Kite Pharmas new manufacturing site in Frederick County, Maryland, an HIV cure emerging in Maryland or our continued coverage of the regions expanding cell and gene therapy cluster-its been a truly remarkable year within the BHCR.

In many cases, BioBuzzs most popular featured stories mirror many of the most important trends impacting the industry, both regionally and nationally.

Before we reveal our top 10 most viewed BioBuzz articles of 2020, wed like to take a moment to thank our sponsors and readers for an amazing year. Wed also like to thank all of the BHCR thought leaders, industry executives, entrepreneurs, investors and others that have trusted us to cover these stories by being so open and generous with their time and being actively engaged in our regional community.

Now, to the top ten

To succeed in thisfield, this new breed of biotechnology companies needs to understand andreimagine almost everything in their operationfrom supply chain and logisticsto workflow, manufacturing, quality, safety, and labor. Each of thesecomponents impacts facility design and engineering. Its such a new industrythat many processes havent even been developed, making it all that morechallenging for companies to navigate their growth.

To dig deeper intothis topic, we reached out to CRBs process architect, Grace Linton, RA, AIA, LEED APBD+C, who has been involved in designing commercial manufacturing facilitiesfor multiple cell and gene therapy companies.

704 QO offersready-to-occupy lab/office spaces for companies of all sizes. What really makesthis new building stand out isnt just the fully customizable individual labunits, but the suite of amenities that are tailored to the specific needs ofemerging life science companies in the BioHealth Capital Region and theemployees that work for them.

Frederick, Marylandcontinues to be a hotbed for biotech and a destination of choice for many newstartups. The last few years have seen one-time startups like RoosterBio, Inc., BioFactura, Inc. and Akonni Biosystems thrive and growwithin the Frederick life science support ecosystem.

These milestonesrepresent just a few recent examples of Frederick startup success. Now, a newgroup of Frederick startups are poised to spur the next generation of greatbiotech success stories. Lets take a look at a few of the hottest startupsready to take a big leap forward.

Paragon BioServices, the home-grown Maryland biotechcompany that started in a Baltimore incubator 30 years ago, has grown to morethan 400 employees and is two weeks away from beginning production in a brandnew 150,000 square foot state-of-the-art, world-class GMP manufacturingfacility for gene therapies.

However, before a single new production run will take place, Catalent, a world-leading Contract Development and Manufacturing Organization (CDMO) with $2.4B annual sales are set to make Paragon its latest acquisition to expand its gene-therapy manufacturing capabilities with a $1.2B all-cash deal.

These CEOs and theirteams have been inventing everything from novel vaccines and therapeutics togroundbreaking medical devices and equipment. Weve been following the progressof these companies and their founders, many since the beginning, and are readyto spotlight the ones that are on the rise.

Kite Pharma, a California-based biopharmaceutical company that developsinnovative cancer immunotherapies, announced plans today to open a newbiologics manufacturing facility in Frederick County that will produceinnovative cell therapies for people with cancer. A Gilead company, Kite willopen the new facility on a 20-acre site in the Urbana Corporate Center, withplans to create a significant number of job opportunities.

The BioHealth Capital Region (BHCR) is rapidly becoming an industry hub for companies that are developing next-generation therapeutics that focus on personalized and regenerative medicine for the treatment and potential cure of unmet medical needs. Autolus, Inc., a CAR T-cell therapy company, and REGENXBIO, a gene therapy company focused therapies for rare diseases, are two examples of emerging leaders in the personalized medicine field making their imprint on the region.

A host of other BHCR companies are focused onregenerative medicine and are developing a range of cutting edge stem celltherapies.

Two of Marylands gene therapy companies are amongst the Top 10 players in the CAR T-celltherapy development field, and two of the four current FDA approved genetherapies, Yescarta and ZOLGENSMA, are also tied to Maryland companies. Thiscommercial success will secure more capital for these companies to fuel theirpipelines and allow the region to further advance new technologies that canimpact even more patients.

These five gene therapy companies in Maryland are creating innovativesolutions to combat disease and cancers and bring new hope to patients in direneed.

there is a whole ecosystem of innovative biotech andlife science technology companies behind them that are developing the tools,technologies, and infrastructure that is propelling the industry forward.

We have identified five companies that stand out fortheir unique technologies and solutions that are responsible for driving progressin the cell and gene therapy field and helping their partners bring theirproducts through the clinic and to the patients that need them.

It would be a fittingconclusion for an HIV cure to emerge from the state where the virus was firstlinked to AIDS and where the first human diagnostic was developed.

Multiple Maryland companies and research institutions are on the leading-age of HIV research and development, making the state a hotbed of potential next-generation HIV therapies and, possibly, the source of a cure for this devastating global health issue. Some of the most promising cure candidates are coming out of Marylands thriving cell and gene therapy cluster.

What will 2020 bring for the BioHealth Capital Region? Whatever is in store for the year to come, be sure that BioBuzz will be there to cover the important issues and bring you all of the top stories of the new decade!

Over the past 8 years, Chris has grown BioBuzz into a respected brand that is recognized for its community building, networking events and news stories about the local biotech industry. In addition, he runs a Recruiting and Marketing Agency that helps companies attract top talent through a blended model that combines employer branding and marketing services together with a high powered recruiting solution.

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2019s Top 10 Most Read Biotech News Stories from the BioHealth Capital Region - BioBuzz

DetailsCategory: DNA RNA and CellsPublished on Thursday, 26 December 2019 15:57Hits: 971

NEW YORK, NY, USA I December 26, 2019 I Prevail Therapeutics Inc. (Nasdaq: PRVL) (Prevail or the Company), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, today announced that the U.S. Food and Drug Administration (FDA) has notified Prevail that the Companys Investigational New Drug (IND) application for PR001 for the treatment of neuronopathic Gaucher disease (nGD) patients is now active and that Prevail may proceed with initiating its proposed clinical trial. As previously reported, Prevails IND for PR001 for the treatment of nGD had been put on clinical hold by the FDA, and this clinical hold has now been removed.

The Companys planned Phase 1/2 clinical trial for nGD patients will commence at a dose higher than originally proposed. Prevail submitted nonclinical data in which no PR001-related safety events or adverse findings were observed, supporting the initiation of the Phase 1/2 clinical trial at this higher dose.

Prevail is activating a Phase 1/2 clinical trial for Type 2 Gaucher disease patients and expects to initiate patient dosing during the first half of 2020. Type 2 Gaucher disease is the more severe form of nGD, which presents in infancy and involves rapidly progressing neurodegeneration leading to death in infancy or early childhood. The Company also plans to initiate a Phase 1/2 clinical trial for Type 3 Gaucher disease patients in the second half of 2020, under the same nGD IND. Type 3 Gaucher disease is a form of nGD that typically presents in childhood and involves multiple neurological manifestations.

We are pleased to now have an active IND for PR001 for the nGD indication and look forward to initiating a Phase 1/2 clinical trial in the first half of 2020, said Asa Abeliovich, M.D., Ph.D., Founder and Chief Executive Officer of Prevail. Patients with nGD have the most severe form of Gaucher disease and a significant unmet need for therapies to treat their neurological manifestations. We believe PR001 has tremendous potential to help patients suffering from this devastating disease.

Prevail is also developing PR001 for Parkinsons disease patients with a GBA1 mutation (PD-GBA). The Company has an active IND for PR001 for the treatment of PD-GBA and the PROPEL Phase 1/2 clinical trial for PD-GBA patients is now recruiting.

About Neuronopathic Gaucher DiseaseGaucher disease is a lysosomal storage disorder caused by mutations in the glucocerebrosidase gene GBA1, leading to multi-organ pathology. Patients with severe mutations in the GBA1 gene can present with neuronopathic Gaucher disease, also termed Type 2 or Type 3 Gaucher disease. Type 2 Gaucher disease presents in infancy and involves rapidly progressive neurodegeneration leading to death in infancy or early childhood. Type 3 Gaucher disease typically presents in childhood and can involve neurological manifestations such as gaze and motor abnormalities and seizures. There are no therapies approved by the FDA for the treatment of neuronopathic Gaucher disease.

About Prevail TherapeuticsPrevail is a gene therapy company leveraging breakthroughs in human genetics with the goal of developing and commercializing disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases. The company is developing PR001 for patients with Parkinsons disease with a GBA1 mutation (PD-GBA) and neuronopathic Gaucher disease; PR006 for patients with frontotemporal dementia with GRN mutation (FTD-GRN); and PR004 for patients with certain synucleinopathies.

Prevail was founded by Dr. Asa Abeliovich in 2017, through a collaborative effort with The Silverstein Foundation for Parkinsons with GBA and OrbiMed, and is headquartered in New York, NY.

SOURCE: Prevail Therapeutics

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For all the flak the pharmaceutical industry has taken for its exorbitant pricing practices, theres no getting around the fact that its been a pretty stunning decade for medical progress.

Multiple new categories of medicines have moved from dreams and lab benches into the market and peoples lives, and investors who came along for the ride often reaped extraordinary profits. The Nasdaq Biotech Index is up 360 per cent over the last 10 years to the S&P 500s 190 per cent. And thats without mentioning the hundreds of billions of dollars in takeovers that rewarded shareholders with windfalls.

As 2020 approaches, its worth highlighting how far weve come in the past 10 years in developing new therapies and approaches to treating disease, even as politicians grapple with how to rein in health-care costs without breaking an ecosystem that incentivises the search for new discoveries.

Here are some of the decades biggest medical breakthroughs:

Cell therapies

First approved in the US two years ago, these treatments still sound like science fiction. Drugmakers harvest immune cells from patients, engineer them to hunt tumours, grow them by the millions into a living drug, and reinfuse them. Yescarta from Gilead Siences Inc. and Novartis AGs Kymriah the two treatments approved so far can put patients with deadly blood cancers into remission in some cases. At the beginning of the decade, academics were just beginning early patient tests.

Its still early days for the technology, and some issues are holding these drugs back. There are significant side effects, and the bespoke manufacturing process is expensive and time-consuming. That has contributed to a bruising price tag: Both of the approved medicines cost over $350,000 (Dh1.28 million) for a single treatment. And for now, cell therapy is mostly limited to very sick patients who have exhausted all other alternatives.

Luckily, more options are on their way. Some drugmakers are focused on different types of blood cancers. Others hope to mitigate side effects or create treatments that can be grown from donor cells to reduce expenses and speed up treatment. In the longer run, companies are targeting trickier solid tumours. Scientists wouldnt be looking so far into the future without this decades extraordinary progress.

Gene therapies

Researchers have spent years trying to figure out how to replace faulty DNA to cure genetic diseases, potentially with as little as one treatment. Scientific slip-ups and safety issues derailed a wave of initial excitement about these therapies starting in the 1990s; the first two such treatments to be approved in Europe turned out to be commercial flops.

This decade, the technology has come of age. Luxturna, a treatment developed by Spark Therapeutics Inc. for a rare eye disease, became the first gene therapy to get US approval in late 2017. Then in May came the approval of Novartis AGs Zolgensma for a deadly muscle-wasting disease. The drugs have the potential to stave off blindness and death or significant disability with a single dose, and, unsurprisingly, Big Pharma has given them a substantial financial endorsement. Roche Holding AG paid $4.7 billion to acquire Spark this year, while Novartis spent $8.7 billion in 2018 to buy Zolgensma developer Avexis Inc.

Dozens of additional therapies are in development for a variety of other conditions and should hit the market in the next few years. They offer the tantalising potential not just to cure diseases, but to replace years of wildly expensive alternative treatment. If drugmakers can resist the temptation to squeeze out every ounce of value by doing things like charging $2.1 million for Zolgensma, theres potential for these treatments to save both lives and money.

RNA revolution

The above treatments modify DNA; this group uses the bodys messaging system to turn a patients cells into a drug factory or interrupt a harmful process. Two scientists won a Nobel Prize in 2006 for discoveries related to RNA interference (RNAi), one approach to making this type of drug, showing its potential to treat difficult diseases. That prompted an enormous amount of hype and investment, but a series of clinical failures and safety issues led large drugmakers to give up on the approach. Sticking with it into this decade paid off.

Alnylam Inc. has been working since 2002 to figure out the thorny problems plaguing this class of treatments. It brought two RNAi drugs for rare diseases to the market in the past two years and has more on the way. The technology is also moving from small markets to larger ones: Novartis just paid $9.7 billion to acquire Medicines Co. for its Alnylam-developed drug that can substantially lower cholesterol with two annual treatments.

Ionis Pharmaceuticals Inc. and Biogen Inc. collaborated on Spinraza, a so-called antisense drug that became the first effective treatment for a deadly rare disease. It was approved in late 2016 and had one of the most impressive drug launches of the decade. And Moderna Therapeutics rode a wave of promising messenger RNA-based medicines to the most lucrative biotechnology IPO of all time in 2018. From pharma abandonment to multiple approvals and blockbuster sales potential in under 10 years. Not bad!

Cancer immunotherapy

Scientists had been working on ways to unleash the human immune system on cancers well before the 2010s without much luck. Checkpoint inhibitors drugs that release the brakes on the bodys defence mechanisms have since produced outstanding results in a variety of cancers and are the decades most lucrative turnaround story.

Merck got a hold of Keytruda via its 2009 acquisition of Schering-Plough, but it was far from the focus of that deal. Once Bristol-Myers Squibb & Co. produced promising results for its similar drug, Opdivo, Merck started a smart development plan that has turned Keytruda into the worlds most valuable cancer medicine. Its now available to treat more than 10 types of the disease, and has five direct competitors in the US alone. Analysts expect the category to exceed $25 billion in sales next year.

If anything, the drugs may have been too successful. Copycat efforts are pulling money that could fund more innovative research. There are thousands of trials underway attempting to extend the reach of these medicines by combining them with other drugs. Some are based more on wishful thinking than firm scientific footing. Still, the ability to shrink some previously intractable tumours is a considerable advance. If drugmakers finally figure out the right combinations and competition creates pricing pressure that boosts access, these medicines will do even more in the years to come.

Conquering hepatitis C

From a combined economic and public-health standpoint, a new group of highly effective hepatitis C medicines may outstrip just about anything else on this list so far. Cure rates for earlier treatments werent especially high; they took some time to work and had nasty side effects. The approval of Gileads Sovaldi in 2013, followed in time by successor drugs such as AbbVie Inc.s Mavyret, have made hepatitis C pretty easily curable in a matter of weeks. For Gilead, getting to market rapidly with its drug proved enormously profitable; it raked in over $40 billion in revenue in just three years.

Hepatitis C causes liver damage over time that can lead to transplants or cancer. The existence of a rapid cure is a significant long-term boon even if the initial pricing on the drugs made them, in some cases, prohibitively expensive. Sovaldi notoriously cost $1,000 per pill at launch and over $80,000 for a course of treatment. The good new is, treatments have become a lot more affordable, which should allow this class of drugs to have a broad and lasting positive health impact.

Hepatitis C is one of the relatively few markets where the drug-pricing system has worked well. As competing medicines hit the market, the effective cost of these treatments plummeted. That, in turn, made the drugs more accessible to state Medicaid programmes and prison systems, which operate on tight budgets and care for populations with higher rates of hepatitis C infection. Louisiana has pioneered the use of a Netflix model, under which the state paid an upfront fee for unlimited access to the drug. Its an arrangement that will help cure thousands of patients, and other states are likely to follow its lead.

Many of the medicines highlighted in this column have list prices in the six figures, a trend thats helped drive up Americas drug spending by more than $100 billion since 2009. Building on this decades medical advances is going to lead to even more effective medicines that will likely come with steeper prices. Id like to hope that policymakers will come up with a solution that better balances the need to reward innovation with the need to keep medicines accessible. That would really be a breakthrough.

Max Nisen is a Bloomberg Opinion columnist covering biotech, pharma and health care.

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The medical breakthroughs of the past decade | Op-eds - Gulf News

A Belgian researcher has made important steps in developing a new technique that would recruit the bodys own defences against the presence of cancer cells.

Ultimately, the technique would be much less damaging than conventional techniques such as radio- and chemotherapy.

Sophie Lucas is a researcher at the Duve Institute, linked with the Catholic University of Louvain-la-Neuve (UCL). Her work concerns a type of cell known as Regulatory T lymphocytes, or Tregs cells that are part of the lymph system which makes up a large part of the bodys immune system.

Some reactions of the immune system can cause auto-immune conditions, where the bodys defences attack healthy tissues, causing numerous conditions including Type 1 diabetes, lupus, psoriasis and coeliac disease. Even simple hay fever is a sort of auto-immune condition, where the body has a violent reaction to something which is not in itself dangerous, i.e. tree or grass pollen.

The body has equipped itself with Treg cells to suppress excessive immune reactions, but these cause a problem in the case of cancer. In cancer patients, they may favour tumour progression by suppressing immune responses against tumour cells: Tregs inhibit anti-tumour T cells, Lucas writes on the Institutes website. Our group studies how human Tregs inhibit anti-tumour responses.

Cancer itself can be seen as a consequence of the inhibitory action of Tregs. When cancer cells begin to grow in the body, the immune system attacks them as foreign bodies. But as the cancer grows large enough to be detectable, the immune system is not able to keep up with its growth, and this is one probably result of Treg action: the Tregs have stopped the immune system from working full-out.

The idea is to somehow harness the power of Tregs to turn them against not the bodys own immune response, but the presence of the cancerous cells themselves. The Tregs and the bodys normal immune system would then work together against the invader, instead of being in opposition to each other.

The Institutes work is firstly to study how Tregs work, to allow researchers to develop ways of making them work as desired. But while Tregs can be difficult to differentiate from ordinary white blood cells, the team has found a work-around: We circumvented this problem by deriving clones of human Treg cells. Clones are pure populations of cells that can be kept in culture for long periods of time, and provide very stable material to perform repeated experiments and derive robust results. We used our human Treg clones to identify functional features present in Tregs, but not in other types of lymphocytes, that could therefore mediate the Treg immune suppressive functions, Lucas writes.

So far, the team has found that Tregs produce a protein called TGF-, which has an important role in immune system suppression. They regulate TGF- by using another protein attached to their surface, known as GARP. Now, if the exact roles of the two proteins separately and apart can be decoded, the possibility of ordering Tregs to stand down when the immune system is fighting cancer cells but not other malign cells, the battle against cancer could enter a new era.In the long term, the objective is to try to manipulate immune responses to make them more effective, and to permit the patient to reject their own tumour cells.

Alan HopeThe Brussels Times

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Belgian researcher aims to turn body's own defences against cancer - The Brussels Times

Acts of kindness can keep us healthy by releasing chemicals in the pleasure center of our brains, such as serotonin and oxytocin, says Dr. Sara Whatley-Dustin, family medicine physician at Dignity Health. Anxiety lowers our immune system and therefore makes us more susceptible to illness.

Mother knows best

Talia Segal Fidler, nutritionist at The Lodge at Woodloch, a destination spa resort in Pennsylvania, suggests taking a page from Mother Natures recipe box to beat a winter cold or virus. If you eat with the seasons, you will be giving your body exactly what it needs, she says. This includes winter greens like chard, kale, cabbage, and collards for fiber, antioxidants, phytonutrients, vitamins, and minerals. Also, root veggies like beets, sweet potatoes, and turnips provide immune system-loving minerals and Vitamin A. And, citrus fruits from warmer climes in the winter months are Vitamin C-rich and hydrating, help your body absorb other nutrients, and are anti-bacterial, too.

Cover up

New York City-based Kemi Adewumi, founder and CEO of Go Galavant, a group trip travel platform for travelers who hate group travel, just returned from an eight-month travel stint and swears by covering your face holes on flights, long train rides, and long bus rides. Yes, youll look like patient zero, but youll avoid actually being patient zero, she says.

She started out with scarves, and now uses the mouth masks, like the kind that doctors use during surgery (you can find the masks on Amazon or in some drug stores). You can also buy fun-looking reusable ones, says Adewumi, which, of course, require washing after use.

Keep hydrated

Your nose, throat, and lungs use a special part of the immune system called IgA to fight infections on moist surfaces, says Dr. Jacob Teitelbaum, board-certified internal medicine physician and author of Real Cause, Real Cure. You can think of it as the bodys Navy. Just like the Navy, it works very poorly on dry surfaces. So, drinking plenty of water (not sodas, as the sugar in one can of soda suppresses immune function by 30 percent) or hot tea (which loosens the secretions so they can be coughed out) can be very helpful in preventing and fighting respiratory infections.

Strike a pose

Liz Zabel, co-manager of the Emerson Spa at Emerson Resort & Spa in the Catskills (named for Ralph Waldo Emerson), is a certified yoga instructor who has practiced for more than a decade. Zabel endorses easy, quick yoga poses to ward off winter travel bugs. To change the blood flow and redirect pranic energy, lay on the floor for a few moments with your legs lifted over your head and against a wall, says Zabel. This simple pose, Legs Up the Wall, will help relieve stress and spark the bodys inner ability to heal itself. The pose is especially helpful because it does not add any stress to the body, it calms the nervous system and helps regulate blood flow.

Just breathe

Breathing, including yawning, can also help the immune system bully germs. If you find yourself holding back any yawns (or sighs) throughout the day, says Zabel, try letting them go. Yawning and sighing can help to cool your brain down from clotting and is a natural way for your body to wake itself up. It also improves the circulation to your brain by carrying more oxygen through the bloodstream and moving more carbon dioxide out.

Rest up

One of the most powerful ways to immune suppress an animal is to sleep deprive it, says Dr. Teitelbaum. Humans are no different. The average nights sleep in the United States until light bulbs were invented 140 years ago was nine hours a night. We are now down to six and three-quarter hours. This is a contributing factor in the increasing immune dysfunction being seen today.

Dr. Rand McClain, chief medical officer at Santa Monica, Calif.-based LCR Health, also emphasizes sleep. The most important immune supporting tool that I see most in the Western world eschew is adequate sleep, says Dr. McClain. He recommends seven to nine hours a night and routinely at the same time period, such as 11 p.m. to 7 a.m. nightly, rather than at varying times. So, if not always, then before traveling awhile on the road, pay attention to getting adequate sleep, he says.

Laurie Wilson can be reached at laurieheather@yahoo.com.

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How to stay healthy while traveling this winter - The Boston Globe

Scientists piece together the inflammation mechanism in IgG4-related disease, an autoimmune condition with no current cure, revealing possible therapeutic targets

IgG4-related disease is an autoimmune disorder affecting millions and has no established cure. Previous research indicates that T cells, a major component of the immune system, and the immunoglobulin IgG4 itself are key causative factors, but the mechanism of action of these components is unclear. Now, Scientists from Tokyo University of Science have meticulously explored this pathway in their experiments, and their research brings to light new targets for therapy.

Autoimmune diseases are a medical conundrum. In people with these conditions, the immune system of the body, the designated defense system, starts attacking the cells or organs of its own body, mistaking the self-cells for invading disease-causing cells. Often, the cause for this spontaneous dysfunction is not clear, and hence, treatment of these diseases presents a major and ongoing challenge.

One recently discovered autoimmune disease is the IgG4-related disease (or IgG4-RD), which involves the infiltration of plasma cells that are specific to the immunoglobulin (antibody) IgG4 into the body tissue, resulting in irreversible tissue damage in multiple organs. In most patients with IgG4-RD, the blood levels of IgG4 also tend to be higher than those in healthy individuals. Previous studies show that T cellswhich are white blood cells charged with duties of the immune responseplay a key role in the disease mechanism. In particular, special T cells called cytotoxic T lymphocytes, or CTLs, were found in abundance from the inflamed or affected pancreas of patients, along with IgG4. But what was the exact role of CTLs?

In a new study published in International Immunology, a team of scientists from Tokyo University of Science decided to find the answer to this question. Prof. Masato Kubo, a member of this team, states that their aim was twofold. We planned to explore how IgG4 Abs contributes to the CTL-mediated pancreas tissue damage in IgG4-RD, and also to evaluate the pathogenic function of human IgG4 Abs using the mouse model that we have established. The latter is especially important, as IgG4 is not naturally present in mice, meaning that there is a severe lack of adequate animal models to explore this disease.

With these aims, they selected mice that have been genetically programmed to express a protein called ovalbumin (the major protein in egg white) in their pancreas. Then, they injected IgG4 that specifically targets ovalbumin into the mice. Their assumption was that IgG4 would target the pancreas and bring about IgG-4-RD-like symptoms. However, what they found was surprising. No inflammation or any other symptom typical of IgG4-RD appeared. This convinced the researchers that IgG4 alone was not the causative factor of IgG4-RD.

Next, to check if it was the CTLs that were perhaps the villain of the story, the scientists injected both IgG4 specific against ovalbumin as well as CTLs. Now, the pancreas of the mice showed tissue damage and inflammation. Thus, it was established that the presence of CTLs and IgG4 was necessary for pancreatic inflammation.

When they probed further, they found that another variation of T cells, known as T follicular helper or TFH cells, which develop from the natural T cells of the mice, produce self-reactive antibodies like IgG4, which induce inflammation in combination with CTLs.

Once the puzzle was pieced together, the scientists now had the opportunity to zero in on the target step for intervention; after all, if one of these steps is disrupted, the inflammation can be prevented. After much deliberation, they propose that Janus kinase, or JAK, can be a suitable target. JAK is a key component of the JAK-STAT cellular signaling pathway, and this pathway is an integral step in the conversion of natural T cells of the mice to TFH cells. If this JAK is inhibited, this conversion will not take place, meaning that even the presence of CTLs will not be able to induce inflammation.

Prof. Kubo also suggests a broader outlook, not limited to the therapeutic option explored in the study. He states, based on our findings, the therapeutic targets for IgG4-related diseases can be the reduction of TFH cell responses and the auto-antigen specific CTL responses. These can also provide the fundamental basis for developing new therapeutic applications.

These proposed therapeutic targets need further exploration, but once developed, they have the potential to improve the lives of millions of patients with IgG4-RD worldwide.

###

Reference

Journal:

International Immunology

About The Tokyo University of Science

Tokyo University of Science (TUS) is a well-known and respected university, and the largest science-specialized private research university in Japan, with four campuses in central Tokyo and its suburbs and in Hokkaido. Established in 1881, the university has continually contributed to Japans development in science through inculcating the love for science in researchers, technicians, and educators.

With a mission of Creating science and technology for the harmonious development of nature, human beings, and society, TUS has undertaken a wide range of research from basic to applied science. TUS has embraced a multidisciplinary approach to research and undertaken intensive study in some of todays most vital fields. TUS is a meritocracy where the best in science is recognized and nurtured. It is the only private university in Japan that has produced a Nobel Prize winner and the only private university in Asia to produce Nobel Prize winners within the natural sciences field.Website: https://www.tus.ac.jp/en/mediarelations/

About Professor Masato Kubo from Tokyo University of Science

Dr Masato Kubo is a Professor at the Tokyo University of Science. A respected and senior researcher in his field, he has more than 226 publications to his credit. He is also the corresponding author of this study. His research interests include Immunology and Allergology. He is the team leader at the Laboratory for Cytokine Regulation, RIKEN Center for Integrative Medical Sciences.

Funding information

This study was supported by grants from JSPS KAKENHI (grant no. 19H03491), Japan Agency for Medical Research and Development (AMED), AMED-CREST, and Toppan Printing CO., LTD.

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Solving the puzzle of IgG4-related disease, the elusive autoimmune disorder - QS WOW News

CLEVELAND, Ohio Immunotherapy, which boosts a patients immune system to destroy cancer cells, is widely seen as the future of cancer care.

This kind of therapy uses substances made by the body, or in a laboratory, to improve or restore the immune system. It can slow the growth of cancer cells, help the immune system do a better job of destroying tumors and slow the spread of cancer to other parts of the body.

There are several kinds of immunotherapy, including CAR-T cell therapy and immune checkpoint inhibitors. In CAR-T, white blood cells called T cells are genetically modified to activate the immune system to recognize and destroy certain cancers.

Immune checkpoint inhibitors are drugs that block proteins made by some immune system cells. When these proteins are blocked, T cells are better able to kill cancer cells.

Cancer vaccines, which are used to prevent and treat cancer, also are a kind of immunotherapy.

Have you or a family member experienced immunotherapy treatment for cancer? Was the treatment effective or ineffective? We want to hear your story.

Please write a short email, no more than 500 words, about your experiences with immunotherapy. Include your full name, age, city and daytime phone number. Your name, age and city will be published with your comment, but your phone number will be kept private.

Comments must be received by Friday, Jan. 10.

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When it came to biomedical breakthroughs, this past decade represented a lot more hype than substance. We were promised a new era of "precision medicine," where every patient would receive highly personalized treatments to target specifics fault in their genes. But in recent months, reports surfaced that these experimental therapies were failing most of the time.

Vas Bailey, a biotech investor with Artis Ventures, acknowledges that it might have seemed like a disappointing decade, barring a few important breakthroughs in the fields of gene editing and therapeutics. But Bailey thinks that there's a lot more to the story.

In recent years, he notes, scientists have focused on the "tools and building blocks" that will drive the breakthroughs in next decade.

"With HIV, we made progresses in life expectancy but didn't cure it; with gene-editing, we achieved one approval that could lay the foundation for others; with AI we learned how to develop it, but haven't yet applied it; and with the microbiome, we have learned more about the relationships (between organisms) but we still haven't designed next generation drugs."

So with the help of a team of biotech experts, here's our list of the most important advancements in biomedicine, many of are likely to propel great advancements going forward.

CAR-T sounds like the stuff of science fiction: Take blood from a cancer patient, re-engineer the blood cells to target and fight cancer cells, and re-infuse the cells in the human body.

But it's real, with the U.S. Food and Drug Administration granting approval in 2017 to the first two drugs that use this approach: Novartis's Kymriah for acute lymphoblastic leukemia (ALL) and Gilead Sciences' Yescarta for certain types of non-Hodgkins lymphoma.

But this therapeutic approach has faced roadblocks, including price -- the drugs cost hundreds of thousands of dollars -- and the logistical complexity of making a treatment out of the patient's own T cells. It can take up to a month to manufacture the cells while the patient's health might be getting progressively worse from the cancer. But this decade also saw progress in companies making off-the-shelf T cells, which are made from healthy donor cells and used for multiple patients.

The next ten years could see advances in using these donor cells -- in particular, reducing the likelihood that patients' immune systems will attack them -- by applying technologies and tools like CRISPR/Cas9.

And that brings us to...

Jennifer Doudna, inventor of the revolutionary gene-editing tool CRISPR photographed in the Li Ka Shing Center on the Campus of the University of California, Berkeley.

Nick Otto | The Washington Post | Getty Images

CRISPR has been hailed as one of the most important breakthroughs of all time. It's essentially a pair of molecular scissors, or a technique to make precise edits in DNA. It was discovered by scientists while exploring the immune system of bacteria.

Scientists have subsequently learned that there are risks to making these modifications. There are also ethical considerations, which were brought to the fore this decade when a scientist in China called He Jiankui reported that he had used the technology to create the first human babies with CRISPR-edited genes.

But this decade ended on a positive note. NPR reported that the first patient in the United States with a genetic disease -- a blood disorder called sickle cell anemia -- was treated with the CRISPR technique. The director of the National Institutes of Health Dr. Francis Collins, told NPR it could give patients a "chance for a new life."

After decades of research into better treatments for Hepatitis C, a debilitating liver disease that could affect as many as 4 million Americans, the U.S. Food and Drug Administration approved a new treatment in 2017 that can reverse the disease in as little as 8 weeks.

The drug from AbbVie followed approvals for similar drugs from fellow drug makers Gilead and Merck, providing a welcome alternative to the prior regimen of shots and pills that didn't always work and came with many side effects. Gilead, which saw its first drug for Hepatitis C approved in 2013, provided a cure in 12 weeks for about 90 percent of patients.

The drugs are extremely expensive, and led to a big spike in revenues for Gilead, but new entrants will provide more competition, potentially leading to lower prices.

A man checks in with a program manager after driving nearly three hours to get to his appointment at Open Arms Healthcare Center on Wednesday, January 23, 2019, in Jackson, MS. Open Arms provides PrEP, or Pre-exposure prophylaxis, which is "a way for people who do not have HIV but who are at substantial risk of getting it to prevent HIV infection by taking a pill every day," according to the CDC.

Jahi Chikwendiu | The Washington Post | Getty Images

This decade, scientists did not cure HIV, the virus that causes AIDS, but they did make some major breakthroughs.

In 2012, the Food and Drug Administration approved Truvada, the first drug to reduce the risk of HIV infection in uninfected individuals who might be at high risk. The drug, which can be taken daily, is used for pre-exposure prophylaxis (PrEP) to lower chances of becoming infected with HIV if exposed.

Unlike some of the other breakthroughs on this list, Truvada has become more pervasive and widespread throughout the decade as costs have come down. The Trump Administration recently unveiled a plan to make these drugs free for people who don't have insurance coverage, and virtual medicine companies like Nurx and Plushcare have sprung up to prescribe PrEP online without a lab visit.

A young boy is handed a certificate confirming he is Ebola-free, outside the Ebola treatment centre in Beni, eastern Democratic Republic of the Congo.

Sally Hayden | SOPA Images | LightRocket | Getty Images

In November of 2019, European regulators finally approved an immunization against Ebola. That decision means that Merck can market its vaccine and distribute it beyond Africa. It has also been approved by regulators in the U.S.

Several vaccines are in development to prevent the outbreak of the fever, which causes such symptoms as diarrhea and bleeding, but Merck's is the only one that was tested during a real outbreak from 2014 to 2016. Ebola has killed more than 2,000 people in the Congo since the middle of this year alone.

Merck has said that it expects to start manufacturing licensed doses of the vaccine in the third quarter of 2020, and that it is working closely with the U.S. government, public health organizations and other groups.

These are just five of the breakthroughs from the past decade, which also saw developments in new "biologic" drugs, which contain or are produced from living organisms, advancements in prosthetics, and in a heartwarming twist, a new medicine for a single patient.

But as Bailey reminds, the best might still be to come. In the past ten years, scientists also made great strides to map out all the cells in the human bodyand to understand contributors to disease encoded in DNA. Teams of researchers also sought to create reference databases for the collections of microbes living in our bodies, and computer scientists teamed up with biologists to extract meaning from huge volumes of medical information, including X-ray imaging and pathology slides.

With all this in mind, Bailey thinks we could see in the next ten years regulatory approvals for the first blood test to screen for cancer at the earliest stages, new therapies that take advantage of our understanding of RNA (the intermediary between DNA and the proteins it instructs the body to make), drugs based on our ever-growing knowledge of the human microbiome, a cure for HIV, and novel approaches to ending multi-drug resistance.

What we've built in recent years are "enabling systems," said Bailey, "and we will benefit from it in the next decade."

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These biomedical breakthroughs of the decade saved lives and reduced suffering - CNBC