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Is Annual Eye Exam a Must for Type 1 Diabetes? – WebMD

Posted: April 20, 2017 at 8:43 pm

By Alan Mozes

HealthDay Reporter

WEDNESDAY, April 19, 2017 (HealthDay News) -- People with type 1 diabetes face the risk of developing a disease that can cause blindness, so treatment guidelines have long called for annual eye exams.

But new research suggests this one-size-fits-all advice is costly and ineffective, because people with a low risk may need less-frequent screenings while people at high risk may need to be seen more often.

Diabetic retinopathy can damage the light-sensitive tissue at the back of the eye and trigger full vision loss, the researchers explained. Screening can catch this disease before irreparable damage is done, but not every person with diabetes faces the same risk.

"For example, patients with no or minimal eye changes and good blood sugar levels might not need their next examination for another four years," said study author Dr. David Nathan.

"On the other hand, if the patient already has developing eye disease and their blood sugar control has not been in the recommended range, they might need a repeat examination in as soon as three months," he added.

Nathan is the director of the Diabetes Center and Clinical Research Center at Massachusetts General Hospital, in Boston.

Current guidelines suggest getting an annual eye screening within three to five years of a type 1 diabetes diagnosis. People with type 1 diabetes cannot produce any insulin.

To assess that advice, the investigators focused on type 1 diabetics (aged 13 to 39) who had enrolled in a large, national diabetes trial between 1983 and 1989.

The latest analysis involved 24,000 eye exams conducted over 30 years among roughly 1,400 people with type 1 diabetes.

Retinal photographs were taken every six months until 1993, and then -- in a follow-up study -- once every four years until 2012. The study participants' vision, advanced retinopathy status and general diabetes history were tracked for an average of nearly 29 years.

The researchers determined that those participants who had an average blood sugar level of 6 percent, but no signs of retinopathy, could forgo the annual screening in favor of just one exam every four years. Similar people with mild retinopathy should be screened once every three years, the team concluded.

By contrast, those with severe or moderate retinopathy would do well to get screened every three to six months, respectively, the study authors reported.

People with higher blood sugar levels (8 to 10 percent) might also need to be screened more often, the researchers cautioned.

On average, the new recommendations for people with type 1 diabetes would likely cut the need for eye exams in half over a two-decade period. That would translate into an overall savings of $1 billion, while ensuring that those facing the highest risk got more timely treatment, the researchers said.

The findings were published in the April 20 issue of the New England Journal of Medicine.

Nathan described the results as "definitive." However, he said the jury is still out as to "whether the individualized frequency of eye examinations will be implemented by physicians" and followed by people with type 1 diabetes.

"The risk is that physicians may find it easier to schedule an annual eye examination compared with the new individualized schedule, which may be more difficult for physicians and patients to remember," he acknowledged.

"However, most physicians and ophthalmology offices use computer programs -- including reminder programs -- for scheduling, so we think that this potential barrier should not be a substantial obstacle," Nathan added.

Courtney Cochran, senior manager of media relations for the American Diabetes Association (ADA), noted that the ADA issued updated guidelines for retinopathy screenings in February.

The new recommendations now state that people with type 1 diabetes should start annual screenings within five years of their diabetes diagnosis. But those who remain free of retinopathy for a year or two may "consider" the option of less-frequent exams.

However, the ADA also said that if and when "any level" of retinopathy is detected, annual screenings are a must, while those with progressing retinopathy will require even more frequent screenings.

Dr. Jamie Rosenberg, who wrote an editorial that accompanied the study, suggested the new recommendations reflect "a trend toward reducing unnecessary screening for eye diseases."

"The upside to this new screening protocol would be significant money saved for the health care system, in addition to time saved for both patients and doctors," said Rosenberg, who is an associate professor of clinical ophthalmology and visual sciences at the Albert Einstein College of Medicine, in New York City.

Individualized schedules will make tracking patients more difficult, Rosenberg agreed. But, "this new screening protocol has great potential if adherence to the examination schedule could be assured."

WebMD News from HealthDay

SOURCES: David Nathan, M.D., director, Diabetes Center and Clinical Research Center, Massachusetts General Hospital, and professor, medicine, Harvard Medical School, Boston; Jamie Rosenberg, M.D., associate professor, clinical ophthalmology & visual sciences, and associate professor, clinical pediatrics, Albert Einstein College of Medicine, and attending physician, Montefiore Health System, New York City; Courtney Cochran, senior manager, media relations, American Diabetes Association; April 20, 2017, New England Journal of Medicine

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Young minds meet new technology at STEM-focused after-school program – USC News

Posted: April 19, 2017 at 6:44 pm

La Bella Saunders gasped as she steppedtoward the giant fuchsia nucleus floating in front of her. She giggled as she reached out toward two oblong, orange mitochondria and an electric blue ball of protein, then lowered her head to look through a small pore one of dozens of openings in the nucleus to spy the DNAinside.

Saunders, a fifth-grader at Foshay Learning Center near USCs University Park Campus, was exploring human biology using a virtual-reality headset that put her inside a cell.

The experience was part of a Young Scientists Program (YSP) after-school workshop on emerging technologies, held March 22 at the school. YSP is a STEM (science, technology, engineering and math) learning initiative hosted through theJoint Educational Project (JEP) of the USC Dornsife College of Letters, Arts and Sciences.

In addition to exploring virtual and augmented reality, the 25 fourth- and fifth-grade participants experienced 3-D printing and drones many for the first time.

We chose these three fields because they are key elements in todays newly emerging technologies, said JEP STEM Program Manager Dieuwertje DJ Kast, who organized the workshop.

I want to show the YSP students what is possible and introduce them to potential careers that could spring from what theyre learning about today, she said. I also want to help level the playing field by giving them the opportunity to get exposure to this technology now, while theyre young.

Students spent 20 minutes at differentstations learning aboutthe three emerging technologies. YSP teaching assistants were on hand to ensure the youngsters had fun while also understanding the underlying science.

At the 3-D printing station, students designed objects in a virtual space using the 3D Slash computer program. This gave them firsthand experience of how architects and engineers, who work with 3-D concepts, think and design. Thanks to local 3-D printing company Deezmakers, students were able to observe printers in action. They watchedintentlyas their teachers and YSP staff were scanned by the Deezmaker selfie setup, which printed out tiny 3-D models.

At the drone station, students sawprofessional drone pilot Roee Fung fly a selection of four quadcopter drones. They grinned for the camera as a tiny, remote control aircraft snapped aerial drone selfies of them as it hovered overhead.

After watching the aerial drones, 10-year-old Maurice Stepter focused on building a fan-driven drone car of his own using a plastic lunch box, wheels, a propeller and a tiny battery-driven motor. It was the fourth-graders first time attending a YSP event.

When I got here it was like, Whoa, this is cool, and that made me want to stay.

Maurice Stepter

Some teachers were encouraging me to come along today so I did, and when I got here it was like, Whoa, this is cool, and that made me want to stay, he said.

Four stations were dedicated to VRand augmented reality, including the Into the Cell experience, a VR project built by Smart Sparrow Inc. and presented by Jessica Swann and Joe Tamer from Arizona State Universitys Center for Education Through eXploration. Students also got to experience a virtual safari, a roller coaster ride and a dive to the depths of the ocean, thanks to WeLens, which donated 50 cardboard VR headsets for students to take home.

Students learned about erosion by playing with the Deezmakers augmented reality sandbox, which changes the color of sand according to its depth. That allowed YSP staff to explain topographical maps by giving students hands-on experience with creating their own landscapes.

At another station, YSP teaching assistant Michelle Huber, a senior majoring in environmental studies at USC Dornsife, was helping students create holograms by cutting and folding transparencies into a trapezoidal shape and placing them on top of a smartphone inside a black box. As the phone played a video of a blue jellyfish, the images were reflected in the trapezoid, creating a hologram.

Working with YSP helped Huber realize that she wants to pursue a career in teaching. I want to go into education because I have really enjoyed programs like this one that involve teaching young kids, she said.

Saunders was impressed with the YSP workshop.I learned all about cells and how drones can help save people by showing firefighters the places they need to put out fires, she said.

Kast emphasized the importance of YSP workshops in introducing students to STEM and helping them advance on the path that will allow them to turn their career dreams into reality.

These workshops are extremely valuable because they really show students whats possible, she said, whether that involves careers, experiences or opportunities.

Jasmine Tigolo, Saunders teacher at Foshay, agreed, saying she was delighted to be able to offer YSP to her students.

This is the sort of up-to-date technology they need to know in order to be able to compete in the outside world, she said, so exposing them to something while theyre this young is phenomenal.

More stories about: Community Outreach, Joint Educational Project, Virtual Reality

Experts and peers in their respective fields agree: These Trojans are changing the world.

With the help of USC undergrads, fourth- and fifth-graders take part in a skills-shaping event hosted by the Joint Educational Project.

Students learn to write code, design games and control robots at a computer science workshop.

The Joint Educational Project curriculum brings in a weatherman to talk with youngsters about meteorology.

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3D-printed Patch Can Help Mend a ‘Broken’ Heart – Technology Networks

Posted: April 19, 2017 at 6:43 pm


Technology Networks
3D-printed Patch Can Help Mend a 'Broken' Heart
Technology Networks
In this study, researchers from the University of Minnesota-Twin Cities, University of Wisconsin-Madison, and University of Alabama-Birmingham used laser-based 3D-bioprinting techniques to incorporate stem cells derived from adult human heart cells on ...

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Plandai Biotechnology Sets Aggressive Agenda for 2017 – Yahoo Finance

Posted: April 19, 2017 at 6:42 pm

NEW YORK, NY--(Marketwired - April 19, 2017) - Planda Biotechnology (PLPL) recently presented investors with its outlook for 2017 and a number of milestones that the company plans to accomplish in 2017. Planda's plans are certainly aggressive and representative of the new Chief Operating Officer (COO) -- someone we feel is ideally suited to move the company forward. It didn't take long at all for the new COO, Callum Cottrell-Duffield, to put his stamp on the company's future.

Three weeks ago Planda named Callum Cottrell-Duffield as its new COO, a role that will have him running the day-to-day operations of the company. In the time since being named COO, he has put together a plan to not only get Planda back on track, but to also move it aggressively into the future. With Planda shifting from purely a research and development biotechnology company to a company that is more focused on operations and sales, Callum Cottrell-Duffield is the ideal person to run Planda's day-to-day activity.

After all, he has been there from the beginning where he helped to prepare the company's 8,000-acre tea estate to grow, harvest and produce Planda's signature product, the highly bioavailable Phytofare Catechin Complex. Last year when that signature product was available for mass production and ready to market on a much broader scale, it was Callum Cottrell-Duffield who led the company's sales and marketing team. He has traveled throughout Africa, the United States, Europe, Asia and South America to tell the Planda story, and he has become the face of the company to the industry and to the large buyers who are either current clients or interested in developing a relationship with Planda worldwide.

In the company's latest news release, the COO highlighted several areas where he expects to see improvement and areas where Planda can realize growth. Among those items, Callum Cottrell-Duffield said that he (i) has placed getting the company "current" with its SEC filings at the top of his agenda, (ii) expects to increase production and to continue growing sales with Planda's existing customers as well as gaining traction in the market, which should lead to the addition of new customers all in an effort to make Planda cash flow positive and profitable by the end of 2017, (iii) recently signed a financing agreement that will provide the necessary capital to see the company through until it becomes cash flow positive.

With the appointment of Callum Cottrell-Duffield to COO, the company's CEO, Roger Baylis-Duffield, can now focus his efforts as a scientist on spearheading the company's clinical trials and developing new products for Planda to market. In the company's research and development efforts, the CEO will be busy with a number of major studies this year.

According to the COO, Planda's work in the clinic will include:

Additionally, Planda has granted a research license to Protext Mobility to develop pharmaceutical applications involving Phytofare. Protext will be conducting a human study using Phytofare to regulate glucose levels in Type 2 diabetics as well as, taking over the research to establish a platform for producing non-psychoactive Cannabis. Planda states that the plan for Protext is to produce a Phytofare complex containing bioavailable cannabinoids, cannabinoid acids, and polyphenols that will be formulated into an oral delivery system.

Needless to say, with success in these studies, these are all areas where Planda could develop much-needed products that could, in turn, drastically improve the company's bottom line. For a full look into the COO's agenda for 2017, read Planda's latest news release here:

http://finance.yahoo.com/news/plandai-biotechnology-chief-operating-officer-162043100.html

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Puma Biotechnology Inc (PBYI) Plunges 6.94% on April 18 – Equities.com

Posted: April 19, 2017 at 6:42 pm

Market Summary Follow

Puma Biotechnology Inc is a A biopharmaceutical company

PBYI - Market Data & News

PBYI - Stock Valuation Report

Puma Biotechnology Inc (PBYI) had a rough trading day for Tuesday April 18 as shares tumbled 6.94%, or a loss of $-2.75 per share, to close at $36.85. After opening the day at $39.90, shares of Puma Biotechnology Inc traded as high as $40.55 and as low as $35.85. Volume was 2.06 million shares over 12,951 trades, against an average daily volume of 910,555 shares and a total float of 36.95 million.

As a result of the decline, Puma Biotechnology Inc now has a market cap of $1.36 billion. In the last year, shares of Puma Biotechnology Inc have traded between a range of $73.27 and $19.74, and its 50-day SMA is currently $37.71 and 200-day SMA is $42.98.

For a complete fundamental analysis of Puma Biotechnology Inc, check out Equities.coms Stock Valuation Analysis report for PBYI.

Want to invest with the experts? Subscribe to Equities Premium newsletters today! Visit http://www.equitiespremium.com/ to learn more about Guild Investments Market Commentary and Adam Sarhans Find Leading Stocks today.

Puma Biotechnology Inc is a biopharmaceutical company. It is engaged in the acquisition, development and commercialization of products to enhance cancer care.

Puma Biotechnology Inc is based out of Los Angeles, CA and has some 160 employees. Its CEO is Alan H. Auerbach.

Puma Biotechnology Inc is a component of the Russell 2000. The Russell 2000 is one of the leading indices tracking small-cap companies in the United States. It's maintained by Russell Investments, an industry leader in creating and maintaining indices, and consists of the smallest 2000 stocks from the broader Russell 3000 index.

Russell's indices differ from traditional indices like the Dow Jones Industrial Average (DJIA) or S&P 500, whose members are selected by committee, because they base membership entirely on an objective, rules based methodology. The 3,000 largest companies by market cap make up the Russell 3000, with the 2,000 smaller companies making up the Russell 2000. It's a simple approach that gives a broad, unbiased look at the small-cap market as a whole.

To get more information on Puma Biotechnology Inc and to follow the companys latest updates, you can visit the companys profile page here: PBYIs Profile. For more news on the financial markets and emerging growth companies, be sure to visit Equities.coms Newsdesk. Also, dont forget to sign-up for our daily email newsletter to ensure you dont miss out on any of our best stories.

All data provided by QuoteMedia and was accurate as of 4:30PM ET.

DISCLOSURE: The views and opinions expressed in this article are those of the authors, and do not represent the views of equities.com. Readers should not consider statements made by the author as formal recommendations and should consult their financial advisor before making any investment decisions. To read our full disclosure, please go to: http://www.equities.com/disclaimer

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Does the US have the biotechnology and other tools to deal with the next wave of diseases? – Genetic Literacy Project

Posted: April 19, 2017 at 6:42 pm

[Editors note:Tom Frieden served seven and a half years as director of the U.S. Centers for Disease Control and Prevention. He talks about health challenges facing the nation, as well as memorable moments from his tenure.]

What immediate health issues are facing the Trump administration?

Tom Frieden: Zika is not over. It is likely to spread in Latin America and the Caribbean for months and years to come, and we still dont fully understand the range of birth defects it causes. Antibiotic resistance in the U.S. is something that threatens to undermine modern medicineAnd we are always concerned about an influenza epidemic.

How prepared is the CDC to deal with these problems?

Its a big problem that when there is an emerging threat, we are not able to surge or work as rapidly as we should, as a result of a lack of additional funding and legislative authority. When there is an earthquake, the Federal Emergency Management Agency doesnt have to go to Congress and say, Will you give us money for this? But the CDC doesA blind spot anywhere puts any of us at risk.

What do you consider unfinished business or a regret?

I hoped that we would be over the finish line on polio [eradication] by now[But] we have further to go.

The GLP aggregated and excerpted this blog/article to reflect the diversity of news, opinion, and analysis. Read full, original post:Is the U.S. Ready for Future Disease Threats?

For more background on the Genetic Literacy Project, read GLP on Wikipedia

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Reminder: San Diego stem cell meeting open to the public tomorrow – The San Diego Union-Tribune

Posted: April 19, 2017 at 6:42 pm

Interested in learning about stem cell science and medicine and what it means to you? A public meeting will be held Thursday from noon to 1 p.m. at the Sanford Consortium for Regenerative Medicine in La Jolla.

Heres more details from my earlier story.

Not only will researchers and a patient advocate with Parkinsons disease discuss the state of stem cell treatments, the public will be able to ask questions.

The Sanford Consortium building is located across the street from the Salk Institute. For those seeking a bite to eat before or after the event, the building also hosts a bistro, the Bella Vista Social Club and Cafe.

The video with this story shows Sandra Dillon describing how she regained stamina and energy after an experimental treatment that blocked cancer stem cells. While not a cure, the treatment reversed what appeared to be an inevitable downward progression.

Dillions doctor, physician-researcher Catriona Jamieson, will be one of the speakers at tomorrows event.

Ill be on site at the event, and will have a report(s) up tomorrow afternoon.

bradley.fikes@sduniontribune.com

(619) 293-1020

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Ex-NFL star Aaron Hernandez dead after hanging self in cell – Medicine Hat News

Posted: April 19, 2017 at 6:42 pm

By The Associated Press on April 19, 2017.

MILFORD, Mass. Massachusetts prison officials say former NFL star Aaron Hernandez has hanged himself in his cell and has been pronounced dead at a hospital. He was 27.

An official with the Massachusetts Department of Corrections says Hernandez was found hanged in his cell just after 3 a.m. Wednesday. Authorities tried to revive the former New England Patriots tight end, and he was pronounced dead at UMass Memorial HealthAlliance Hospital in Leominster at 4:07 a.m.

Prison officials say the Hernandez was in a single cell in a general population housing unit at the Souza Baranowski Correctional Center in Shirley, Massachusetts They say he hanged himself using a bed sheet that he attached to a cell window.

Authorities say Hernandez tried to block the cell door from the inside by jamming the door with various items.

Hernandez, who was serving a life sentence for a 2013 murder, was acquitted Friday in a 2012 double slaying prosecutors said was fueled by his anger over a drink spilled at a nightclub.

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Who Was Henrietta Lacks? 5 Striking Facts About The ‘Mother Of Modern Medicine’ – Huffington Post

Posted: April 19, 2017 at 6:42 pm

Hardly anyone knew of Henrietta Lacks life story prior to 2010.

That year,Rebecca SklootsThe Immortal Life of Henrietta Lackswas released, and went on to become a New York Times best-seller. The biographical book told the story of a black woman born on a tobacco farm in Roanoke, Virginia, in 1920 who revolutionized medical research and saved the lives of millions, without ever knowing it. Now, a new film by the same name starring Oprah Winfrey aims to make her life and impact more widely known.

Who exactly was Henrietta Lacks? And why is she described as the Mother of Medicine? Here are five fascinating facts about Lacks to better understand who she was and how she changed the world forever.

The Washington Post via Getty Images

In 1951, at the age of 31,Lacks visited Baltimores Johns Hopkins Hospital, which served black patients in segregated wards during the Jim Crow era, so doctors could find out what was causing pain in her lower stomach. It turned out there was a cancerous tumor that had grown at a terrifying rate on her cervix.

At the time, cervical cancer was prevalent among women and research samples were taken from those who were diagnosed with it. Richard Telinde, a doctor at Hopkins who led a research study on patients who tested positive, hoped to grow living samples from both normal and infected cells to better understand the cancer. He worked with his colleague Dr. George Gey, the head of tissue culture research at Hopkins, who was relentlessly determined to develop the first line of immortal human cells those that could repeatedly replicate themselves outside of the body without ever dying.

Soon after her first trip to the hospital, the excruciating pain Lacks felt began to worsen as her tumor grew, so she checked herself into Hopkins for immediate treatment through surgery. The doctor who performed the surgery then removed two dime-sized pieces of tissue from Lacks body one from the infected cervix, the other from a healthy part of the organ and had them handed off to Gey. He and his staff used Lacks samples to successfully grow the first line of immortal cells. Lacks eventually died from the cancer, leaving five young children.

However, her cells lived on and soon came to be known as HeLa.

In The Immortal Life of Henrietta Lacks, Skloot writes that while Lacks gave doctors permission to perform a surgical procedure on her, she knew nothing about her cells growing in a laboratory. The hospital had called Lacks husband, David, to tell him about her death and ask if they could do an autopsy on her. Her husband initially denied the request, but visited the hospital later that day to see Lacks body and eventually agreed to sign off on the autopsy because doctors said they wanted to conduct tests that may help their children, and he believed them.

Decades after Lacks death, Rolling Stone published a riveting piece in March 1976that gave a detailed account of what happened to her cells and included comments from her husband. In the piece, he recounted his experience at the hospital after learning of her death and revealed that he had never explicitly been told by doctors or any official about what the samples had been used for:

They said it wouldnt disfigure her none, because it was all down in her womb, to begin with. He nods. They said it was the fastest growing cancer theyd ever known, and they was suppose to tell me about it, to let me know, but I never did hear.

In the same interview, Lacks eldest son,Lawrence, told the reporter: First we heard was about a month ago, a person called us on the phone and asked if wed like to take a blood test. Thats the first time we heard about it.

Helen Lane had quickly become a pseudonym for Henrietta Lacks in print, which Skloot writeswas apparently an intentional move made in an effort to disguise Lacks true identity from the public and the media. According to Skloot, one of Geys colleagues told her Gey himself had created the new name so the media wouldnt discover who Lacks really was. The Minneapolis Star was the first to publish a report on Nov. 2, 1953, that more accurately identified Lacks, only the last name was incorrect: She was recognized as Henrietta Lakes.

Upon the release of the story, journalists dug in and began requesting interviews with Gey and other doctors central to the case, but they all were reluctant to release her real name at the risk of getting into trouble, according to the book.Skloot firmly concludes that had Lacks name been released to the public from the outset, it would have changed her familys life forever.

They would have learned that Henriettas cells were still alive, that theyd been taken, bought, sold and used in research without her knowledge or theirs, she wrote.

HeLa cells have entirely revolutionized medical research. The cell line can be found in labs across the world and has been used in studies that have resulted in countless breakthroughs.

The cells were used to develop the first polio vaccine in 1952 during a time when the disease swept the nation in an outbreakthat left thousands of children paralyzed.

HeLa cells have also traveled to space to help scientists study the impact zero gravity has on human cells; been used to identify abnormalities in chromosomes; helped with research in the mapping of the human genome; and aided in studying the human papillomavirus, commonly known as HPV, which causes the cervical cancer that killed Lacks.

In 2014, chemists and engineers at Penn State University announced that in their study, HeLa cells had been implanted with technology that have potential to cure cancer if they are able to mechanically manipulate cells inside the body.

Both of Lacks daughters have died, including Deborah, who was hugely instrumental in bringing the book to life by working with Skloot and whom Oprah portrays in the film. But her legacy lives on through her three sons, who are now decades old.

And its Lacks eldest son, Lawrence, reportedly the executor of her estate, who is leading the charge for the family to receive compensation from Johns Hopkins Hospital and others. However, in a statement obtained by The Washington Post in February, the institute said it does not own the rights for the HeLa cell line and that they have not profited from the cells.Lawrence plans on continuing to pursue his mission.

Before Deborahs death in 2009, she told Skloot that even though she and her siblings lost their mother, Lacks always knew how to make her presence known.

Deborah believed Henriettas spirit lived on in her cells, controlling the life of anyone who crossed its path, Skloot wrote.Including me.

The Immortal Life of Henrietta Lacks premieres on HBO on Saturday, April 22.

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CAR-T Cell Therapy Receives FDA Breakthrough Designation – Pharmaceutical Processing

Posted: April 19, 2017 at 6:41 pm

Novartis CAR-T cell therapy CTL019 receives FDA Breakthrough Therapy designation for treatment of adult patients with r/r DLBCL.

Novartis announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to CTL019, an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of adult patients with relapsed and refractory (r/r) diffuse large B-cell lymphoma (DLBCL), who have failed two or more prior therapies.

This is the second indication for which CTL019 has received this designation; the first being for the treatment of r/r B-cell acute lymphoblastic leukemia (ALL) in pediatric and young adult patients.

"At Novartis, we are eager to unlock the full potential of CTL019, including the potential to help patients with r/r DLBCL," said Vas Narasimhan, Global Head of Drug Development and Chief Medical Officer, Novartis. "We look forward to working closely with the FDA to help bring this potential new treatment option to patients as soon as possible."

CAR-T cell therapy is different from typical small molecule or biologic therapies currently on the market because it is manufactured for each individual patient. During the treatment process, T cells are drawn from a patient's blood and reprogrammed in the laboratory to create T cells that are genetically coded to hunt the patient's cancer cells and other B-cells expressing a particular antigen.

CTL019 was first developed by the University of Pennsylvania (Penn). In 2012, Novartis and Penn entered into a global collaboration to further research, develop and then commercialize CAR-T cell therapies, including CTL019, for the investigational treatment of cancers. Through the collaboration, Novartis holds the worldwide rights to CARs developed with Penn for all cancer indications.

In March 2017, Novartis announced that the FDA accepted the company's Biologics License Application filing and granted priority review for CTL019 in the treatment of r/r pediatric and young adult patients with B-cell ALL.

The Breakthrough Therapy designation is based on data from the multi-center phase II JULIET study (NCT02445248), which is evaluating the efficacy and safety of CTL019 in adult patients with r/r DLBCL. JULIET is the second global CAR-T trial, following the Novartis ELIANA study (NCT02435849) investigating CTL019 in r/r B-cell ALL. Findings from JULIET are expected to be presented at an upcoming medical congress.

"We are encouraged by the FDA's recognition in the potential of CTL019 for this indication, which follows our promising studies of this therapy for ALL and the FDA filing by Novartis in pediatric and young adult ALL that received priority review," said the Penn team's leader, Carl June, M.D., director of the Center for Cellular Immunotherapies in the Perelman School of Medicine at the University of Pennsylvania. "Work with our collaborators at trial sites across the world is paving a path to bring personalized cell therapies to more patients with these devastating blood cancers."

According to FDA guidelines, treatments that receive Breakthrough Therapy designation are those that treat a serious or life threatening disease or condition and demonstrate a substantial improvement over existing therapies on one or more clinically significant endpoints based on preliminary clinical evidence. The designation also indicates that the agency will expedite the development and review of CTL019 in adults with r/r DLBCL.

This marks the 14thBreakthrough Therapy designation for Novartis since the FDA initiated the program in 2013, underscoring an emphasis to develop innovative treatments in disease areas with significant unmet need.

DLBCL is the most common form of lymphoma and accounts for approximately 30 percent of all non-Hodgkin lymphoma cases1. Ten to 15 percent of DLBCL patients fail to respond to initial therapy or relapse within three months of treatment, and an additional 20 to 25 percent relapse after initial response to therapy2.

Because CTL019 is an investigational therapy, the safety and efficacy profile has not yet been established. Access to investigational therapies is available only through carefully controlled and monitored clinical trials. These trials are designed to better understand the potential benefits and risks of the therapy. Because of the uncertainty of clinical trials, there is no guarantee that CTL019 will ever be commercially available anywhere in the world. ____________________________________________________

References:

1 American Society of Clinical Oncology. Lymphoma - Non-Hodgkin: Subtypes (Dec. 2016 revision).http://www.cancer.net/cancer-types/lymphoma-non-hodgkin/subtypes. Accessed March 2017.

2 Sehn, L. Paramount prognostic factors that guide therapeutic strategies in diffuse large B-cell lymphoma. Hematology, December 2012; 1; 402-409.

(Source: GlobeNewswire)

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CAR-T Cell Therapy Receives FDA Breakthrough Designation - Pharmaceutical Processing

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