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Alex Trebek died of pancreatic cancer think twice before saying he lost his battle with the disease – MarketWatch

Posted: November 13, 2020 at 4:58 am

Jeopardy host Alex Trebek died Sunday of pancreatic cancer, over a year after he was diagnosed with stage IV cancer. NBC News said he died at 80 after a battle with cancer. CBS News implied it was a battle that he lost.

Trebek himself used similar language to describe his future plans when he was diagnosed with the disease back in March 2019. Im going to fight this, Trebek said. Im going to keep working, and with the love and support of my family and with the help of your prayers also I plan to beat the low survival rate statistics for this disease.

When it comes to discussing this disease, many people use words like battle, fight, survive and beat language that would not be out of place when describing a war zone.

At the time of Trebeks diagnosis, well-wishers took to social media offering messages of support to him that often used this pugnacious language.

Indeed, words like battle and fight often arise when a celebrity or other public figure discloses a cancer diagnosis. That was the case when Sen. John McCain, who died in August 2018, first revealed he had been diagnosed with a primary glioblastoma, the same aggressive form of brain cancer that killed former Vice President Joe Bidens son Beau and Sen. Edward Kennedy, a Democrat from Massachusetts.

At the time of McCains diagnosis, supportive tweets and statements from politicians and other dignitaries referenced this idea of cancer as a war or fight to be won, while also acknowledging McCains stellar career and the service he had given to this country. House Speaker Paul Ryan referred to McCain as a warrior. Others, including former Presidents George H.W. Bush and Barack Obama, referenced McCains service to the country, including his experience as a prisoner of war during the Vietnam War.

When it comes to discussing this disease, many people use words like battle, fight, survive and beat language that would not be out of place when describing a war zone. Of course, in this context it represents a persons stamina and state of mind, and optimism. But without knowing whether the person was comfortable with a word like fight to show their will to go on living and their resilience, as Trebek was, you may want to think twice before using it.

When my grandmother was diagnosed with cancer in December 2013 at the age of 83, I initially figured that she would fight it valiantly. After all, this was a woman who had already beaten cancer twice before to say nothing of how fervently she looked after my family, including my grandfather, who is wheelchair-bound.

But after one round of chemotherapy, my grandmother decided to forego treatment and eventually entered hospice care. When she died about five months later, I struggled with her decision not to continue receiving chemo after that first round. A selfish part of me privately felt like she had given up and let cancer win.

I wasnt alone in how I thought about cancer.

For some cancer patients and their loved ones, these battle metaphors are comforting, said Len Lichtenfeld, deputy chief medical officer for the American Cancer Society. The perspective for a lot of people is that youre entering a battle, and youre trying to deal with a foe you would rather not have to consider, he said. But battling cancer may hit the wrong note for some patients and their loved ones, just like sufferer or victim is rejected by people who have other chronic or aggressive diseases.

Hundreds of thousands of people die from cancer in America every year and many of those who die fought the disease extensively. In 2020, there have been an estimated 1.8 million new cancer cases diagnosed in the U.S. and more than 600,000 Americans died of the disease, according to the American Cancer Society. The five-year survival rate for stage IV pancreatic cancer, which Trebek was diagnosed with, is just 3%.

We still lose too many people to cancer, Lichtenfeld said. If things work out and people do well, are they any more a hero than somebody who did everything they needed to do, but unfortunately the disease was stronger than they were?

We still lose too many people to cancer. If things work out and people do well, are they any more a hero than somebody who did everything they needed to do, but unfortunately the disease was stronger than they were?

The metaphor can be disconcerting even for individuals who go into remission. Krystle McGrady, a social worker who lives in Colorado, was diagnosed with Hodgkin lymphoma at 16. She initially went through six months of chemotherapy followed by radiation treatment. When that was unsuccessful, she had a stem cell transplant. After roughly two years, her cancer went into remission and it was only then, she said, that people began to use these battle-like metaphors around her, something that made her uncomfortable.

It was really uncomfortable for me because it made it seem like I had some sort of choice in the matter, she said. This is what I had to do if I wanted a life. Over a decade later, McGrady now works with cancer patients, and she said she consciously avoids using such terminology particularly when it comes to the word survivor. Treatment isnt necessarily a cure, she said.

The battle language can make having cancer seem like a one-time event that the patient controls, then moves on from. The reality isnt so simple. For a growing number of people diagnosed with cancer, advancements in medicine have made living with it closer to having a chronic condition, Lichtenfeld noted.

Dont miss: Why some CEOs are finally treating mental health days as sick days

The language used when talking about cancer doesnt just have an emotional impact on people it can also influence how they make choices surrounding treatment and medical care.

A 2015 study compared womens reactions to different terminology used when diagnosed with ductal carcinoma in situ (DCIS), a condition thats considered to be the earliest form of breast cancer. Women who were initially told that they had abnormal cells and then later found out they had pre-invasive breast cancer cells were more likely to choose treatment over watchful waiting.

Using a metaphor like journey takes into account patients ability to choose, how treatment may be short- or long-term and how the going may not always be so easy.

As the study points out, research has shown that some over-diagnosis and over-treatment of breast lesions such as DCIS does occur though evidence shows screenings to detect breast cancer also reduce the number of fatalities caused by the disease. Even minor alterations in terminology can change the way we feel or behave in the face of cancer, Anne Moyer, a professor at Stony Brook University, wrote in Psychology Today.

In place of such war-themed language, some have suggested using the journey metaphor or allowing the person with cancer to define their own situation.

The travel-themed metaphor takes into account patients ability to choose, how treatment may be short- or long-term and how the going may not always be so easy. The journey metaphor does not countenance such concepts as winning, losing, and failing, Gary Reisfield and George Wilson from the University of Florida wrote in a 2004 article in the Journal of Clinical Oncology. Rather, there are only different roads to travel, various avenues to be explored, and, always, there are exits.

That said, it is also important to be careful about casting people with cancer as inspiring figures or angels. Putting them on a pedestal can also be discomfiting. Cancer patients are still people they want to share in lifes more mundane experiences. As McGrady points out, she made many good friends and got to have plenty of rewarding life experiences after her diagnosis. Loved ones shouldnt forget to continue talking about the small things in life or be afraid to discuss their own happy life events.

It took me a while, but Ive come to terms with my grandmothers choice. Her cancer carcinoma of unknown primary, a form of cancer in which the location in the body where the disease began isnt known has an average survival time of nine to 12 months, according to data from the American Cancer Society.

Today, I see my grandmothers decision to end treatment as remarkably sagacious. With her prognosis in mind, she ultimately chose to spend her remaining time with my family in comfort, rather than endure uncomfortable treatments that may not have given her much more time in the end. Who knows? Had my family pushed her to fight harder or to try to battle cancer, she might have lost that precious time with us.

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‘SWAT’ Preview: Will Season 4 ‘Do Better’ Exploring Race and Policing? – TVLine

Posted: November 13, 2020 at 4:57 am

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CBS S.W.A.T. is ready to swing back into action, with a two-hour Season 4 premiere airing this Wednesday starting at 9/8c.

TVLine spoke with executive producer Aaron Rahsaan Thomas about the powerful, 1992 L.A. riots-themed season opener, which was postponed from last season but is only more relevant now (watch a sneak peek below), as well as the many personal storylines and tests ahead in Season 4.

TVLINE | S.W.A.T. is returning with the Season 3 finale that was postponed by the pandemic, which flashes back to the L.A. riots. Did that episode come out looking a bit different than you originally planned, given every single thing that has happened since?Whats been awesome is how our production team has been really great about being able to plan for this episode in advance. The proactive side of it has been is weve had enough time to really look at how we could still maintain the quality that we were going after in the COVID era. And having looked at the first cut of that episode recently, I can say that weve been able to get pretty much what we wanted. There are considerations regarding, you know, large crowd scenes, and certainly spacing is important. But as far as the material, were pretty much telling the story that we wanted to tell. Were still telling the scope of the story of current day S.W.A.T. dealing with the current political unrest, that tricky dance between the police and the community, and also telling the flashback story back to 1992 of some of the same parallel elements that the police and the community was dealing with at that time. That is all still very much in place.

Donald Dash and Rico E. Anderson play Hondo and Daniel Sr. in flashbacks

TVLINE | No one would have faulted you at all for going with archival news footage of the 1992 riots, because you cant put together a crowd scene the way that you need to. But youre saying you kind of found a way to sell that?Fortunately and unfortunately there are ample examples to pull from when were talking about civil unrest and uprisings in the history of Los Angeles. Theres a lot to pull from both current day and from 1992 that allows us to supplement the look of our episode. So, what weve done, with the help of our director/producer Billy Gierhart, is to in essence maximize our establishing shots based on the stock footage that we were able to get from archives, and then supplement that when we go in to shoot our people on the ground. Normally, if you can sell an audience on the scope of the event from the master shots then you tend to believe and be willing to go with that belief once you go in close. In close, we dont have nearly that many extras that we can or should use, but hopefully your mind is in the space that we actually are in that environment.

TVLINE | Did you slip in any up-to-date references? Like, are Hondo (played by Shemar Moore) and the team talking about George Floyd and Breonna Taylor?Yes and no. The tone of the story certainly shifts a bit, because at the time what we were talking about was how between the Watts riots of 1965 and the Rodney King riots there were 27 years that had passed, and the year 2020 had marked 28 years since Rodney King how maybe were making progress. But the original tone of the story was, Wow, weve learned something new, maybe this time weve gotten it right and then George Floyd happens.

What we tried to do is not necessarily focus on any one modern event or movement; our story is more about the overall legacy of a city, of Los Angeles. The fact is that the things are happening now are not unique and isolated to the year 2020. So, to answer your question: There are references to the current day situations that are happening, and in particular we have a really poignant few places where we reference these modern occurrences. But the overall focus of the story is how the [citys] legacy helps give context to the current day situations more so than the specific current day situation.

TVLINE | Will the premiere also juggle the dangling thread from the Season 3 finales Diablo storyline? Oh, yeah, we very much honor that. Theres something larger and unexpected that happens off of the El Diablo saga from last season. There are some twists and turns to that story that weve yet to play out, and certainly Diablos final lines about the city burning, those will not be in vain.

TVLINE | Are there any personal storylines youre going to hit at the beginning of the season? Like, is Luca (Kenny Johnson) back with the team 100-percent at this point? Also, Deacon (Jay Harrington) confronted his mental health, Tan proposed to Bonnie.All of the above well hit on in the premiere. Moving forward, were definitely going to see Deacon coming off of his arc off of last season and really getting into mental health and starting to counsel a new mentee, based on that idea of him being able to share his wisdom.

Well see definitely Tan (David Lim) and Bonnie (Karissa Lee Staples) examining what you know the next step is for them and Tan having to really consider whether the next step is the best thing or the thing that he wants to do in their relationship.

Luca has always been kind of the heart of our team, a good soldier, and he is going to be facing some leadership opportunities. He will examine whether or not leadership is anything that he wants to do, and why he hasnt considered it before, being a third generation in S.W.A.T. But we have a few twists with Luca, especially early on, where were going to have him working on a larger project that may take him out of the City of Los Angeles. But I dont want to reveal too much just yet with that.

TVLINE | And Street (Alex Russel), he was still dating the commanders daughter, right? Yet theres always something going on with him and Chris (Lina Esco), though we can never quite get a bead on it.Oh, yes, you have the ongoing relationship that Street has with Molly (Laura James), and that provides complications between Street and Hicks himself (Patrick St. Esprit). You know, Streets come a long way since Season 1 weve seen him mature, especially through the last season where he lost his foster brother Nate. But that tends to be, for some people, exactly where you start to backslide. So, Street at the start of the season is in a good spot, but that doesnt necessarily mean things are going to stay that way for him.

Chris, by the way, through the course of the season were going to take her through an emotional kind of whirlwind thats going to test her in a way that she hasnt been tested before. And certainly, her and Streets friendship will be affected by that.

TVLINE | Hondo and Nichelle (Rochelle Aytes) had just broken up.Nichelle went off to work on youth centers, but we really loved the chemistry between the two of them. Nichelle will come back into Hondos life, but that doesnt necessarily mean that theyre going to be romantic. Hondo has some things that he still has to figure out on the personal side before hes really ready to be in a committed relationship again. The two of them, theyre in a unique position in that you have Nichelle who works at a community center and you have Hondo who is a police officer and in the year 2020, they may have different opinions on certain topics. He needs to earn that friendship back and its really going to test Hondo in a way that we dont normally see.

TVLINE | Lastly, I wanted to ask about the statement that you and the writers put out over the summer about wanting to do better in exploring themes of racial injustice and inequality. Have you seen that effort manifest itself yet?What I can tell you is that from the storytelling end of things, we definitely buckled down and really try to make sure that were examining and getting as close to the truth as we can in the stories that we tell. Its a lot of responsibility, with Hondo being our essential character an African-American police officer who respects the job and respects the badge but also understands, having grown up in the community, what the challenges can be, and trying to bridge that gap. Weve had it baked into the DNA of the show from the very beginning from the pilot episode.

Everyone from Shemar Moore to [executive producer] Shawn Ryan to myself to [EP] Andy Dettmann, from the entire writing staff to our entire production and crew, we all have a responsibility to really lean into the show not only as a device for entertainment, but also as a beacon of hope. As an aspirational example of ways that we can improve communication using Hondo as an example of that. So from the opening frame of this season, youre going to see that Hondo is very much confronted by a community that questions his allegiance and he ends up being confronted as well by his police contemporaries who question where his heart is. You have a guy whos caught in the middle and has been since the pilot, but now so more than ever. Like, if you asked Hondo if we were making progress, he wouldve said yes.

TVLINE | Hes got to wonder now, though.Hes got to wonder now. Hondo definitely has that crisis of at least wondering if the efforts that hes making are actually making a difference, and also wondering if the fight is even worth fighting. Again, considering the legacy, considering how many times this has happened over and over again just in his city, much less the country, these are all questions that were leaning into.Were challenging him this season on multiple layers and rightfully so and were not confining it to one episode. Its not a very special episode and after the premiere well never visit again.

Our entire Season 4, pretty much every episode in some way is hitting on that dilemma and not always from Hondos perspective. Tan is a Chinese American in the age of xenophobia, so he has his own perspective thats going to be very unique from anyone elses. Like I said, were going to put Chris through an emotional ringer, and some of that is based on her own world of working in a very testosterone-driven environment. Well be dealing with white nationalists in the course of this season, and not everyone necessarily has the same perspective on how to deal with them.

Were going to be hitting on a variety of different topics, but as far as our proclamation to try to do better in front of and behind the camera, I feel confident that weve been putting in the effort.

Want more scoop on S.W.A.T., or for any other show? Email InsideLine@tvline.com and your question may be answered via Matts Inside Line.

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The Animal Stem Cell Therapy market to fill the growth bill in the next decade – The Think Curiouser

Posted: November 13, 2020 at 4:56 am

Animal stem cell therapy is a usage of animals stem cell to treat a disease or disorder. The ability of stem cell is to divide and differentiate into a cell with specialized function useful for repairing body tissues damaged by injury or disease. The animal stem cell therapy process involve three steps which include collection of stem cell sample from animals and preparing the sample to concentrate the stem cells. Finally, the therapy includes transferring the stem cells into the injured site for treatment. Animal stem cell therapy increases the expectancy of life in animals with no side effects. It is available for the treatment of arthritis, degenerative joint disorders, tendon, and ligaments injuries in animals. Stem cell therapy is most often used to treat dogs, cats, and horses. But recent developments made it possible to use animal stem cell therapy in tiger, pig, etc. Present animal stem cell therapy is studied in treatments of the inflammatory bowel, kidney, liver, heart and immune-mediated diseases respectively.

Animal Stem Cell Therapy Market: Drivers and Restraints

Increasing prevalence of disease in animals with growing population and to increase the animals quality of life, the companies focus shifting towards animal stem cell therapies. Along with increasing government funding for the protection of animals and fast approvals of FDA contributing towards the rapid growth of the animal stem cell therapy. The research in animal stem cells offers great promise for understanding underlying mechanisms of animal development; it gives great opportunities to treat a broad range of diseases and conditions in animals. Animal stem cell therapy is increasingly recognized as critical translational models of human disease for treatment. All these factors act as drivers for the robust growth of the animal stem cell therapy market.

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Animal Stem Cell Therapy Market: Segmentation

Segmentation based on Applications

Segmentation based on End-user

Animal Stem Cell Therapy Market: Market Overview

Studies in the animal stem cell therapy continue at a breathtaking pace due to increasing demand and treatment cost covered in reimbursements. And animal stem cell therapy is more effective than traditional treatment available in the market which is boosting the companies to increase the spending in the R&D for innovative methods. Because of the novelty and complexity of animal stem cell therapy, FDA encourages individuals, universities and drug companies for further innovations. The future expected with double CAGR during the forecasted period.

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Animal Stem Cell Therapy Market: Region-wise Overview

Regarding geographies, North America is dominating the global animal stem cell therapy market due to the increased incidence rate and awareness about the therapy. U.S represents the largest market share in the North America due to the increasing demand for the therapy. Europe and Asia-Pacific are showing a significant growth rate during the forecasted period due to the growing adoption of the animal stem cell therapy. The animal stem cell therapy market in underdeveloped countries is slow when compared to the developed countries.

Animal Stem Cell Therapy Market: Key Participants

The key participants in the animal stem cell therapy market are Magellan Stem Cells, ANIMAL CELL THERAPIES, Abbott Animal Hospital, VETSTEM BIOPHARMA, Veterinary Hospital and Clinic Frisco, CO, etc. The companies are entering into the collaboration and partnership to keep up the pace of the innovations.

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UB researcher narrows time window for administering specific treatment to infants with Krabbe disease – UB Now: News and views for UB faculty and…

Posted: November 13, 2020 at 4:56 am

A team of UB researchers has published a paper in Nature Communications that is helping to define the best time to give a specific treatment to infants born with Krabbe disease (KD). This treatment has been found to prolong life for these infants for as long as a few years.

The paper was published online in Nature Communication Oct. 23.

Daesung Shin, assistant professor in the Department of Biotechnical and Clinical Laboratory Sciences and the Neuroscience Program, both in the Jacobs School of Medicine and Biomedical Sciences at UB, is the lead investigator. He also conducts research at UBs Hunter James Kelly Research Institute.

KD is an inherited disorder that destroys myelin, the protective coating of nerve cells in the brain and throughout the nervous system. In most cases, signs and symptoms of Krabbe disease develop in babies before 6 months of age, and the disease usually results in death by age 2. When it develops in older children and adults, the course of the disease can vary greatly.

The progressive neurologic disorder is caused by a deficiency of galactosylceramidase (GALC). GALC is an enzyme that breaks down galactosylceramide, an important component of myelin, which ensures the rapid transmission of nerve impulses.

Although there is no cure for KD, hematopoietic stem cell therapy (HSCT), a therapy that makes blood cells, reduces neurologic deterioration and improves developmental advances. These benefits are dependent on the severity of the disease at the time the stem cells are transplanted, and are only beneficial if delivered at a clinically defined pre-symptomatic time point before symptoms appear.

Even though it is widely accepted that early treatment is essential for the most positive outcome, the precise therapeutic window for treatment and what happens during this early time have never been elucidated, Shin says.

To address that issue, his team used mutations to create a novel mouse model of KD.

We engineered an inducible knockout mouse for the GALC gene deletion in specific cells at specific times, which provided us with the opportunity to directly ask when and where GALC enzyme is required for brain development, Shin explains.

We were particularly interested in the role of early developmental GALC function, he says. Our study not only revealed a key developmental process that requires GALC in the perinatal period, but also demonstrated that temporal GALC expression is likely a major contributor to brainstem development.

The researchers found that by increasing GALC levels at or before this newly defined perinatal period they could improve the effectiveness of therapeutic interventions for KD.

For the first time, our work showed the mechanistic evidence to explain why treatment must occur so early, with the defined critical postnatal period at days 4-6 in mice, and demonstrated that temporal GALC expression during this time is a major contributor to brainstem development, Shin says. Augmenting GALC levels at or prior to this newly defined period would likely improve the efficacy of therapeutic interventions for Krabbe patients.

While the time scale between mice and humans is considerably different, the sequence of key events in brain maturation between the two is consistent, he notes. It was estimated that the mouse nervous system at postnatal days 4-6 corresponds to a gestational age of 32 weeks in humans. Therefore, we anticipate that if our result is correct, then in utero treatments at, or prior to, 32 weeks should have better outcomes than conventional postnatal treatment for Krabbe babies.

Shin says his team will further identify which cell type needs to be targeted with therapy.

This work will directly impact the design of novel treatment options for KD patients, he says, noting that KD studies are at the basis of research on other, more common neurodegenerative diseases, such as multiple sclerosis and Parkinsons disease. Therefore, the teams work will have implications beyond KD.

Co-authors on the research were Nadav I. Weinstock, MD-PhD student, and Conlan Kreher, former masters student, both of the HJKRI and the Department of Biochemistry in the Jacobs School; Jacob Favret, research technician in the Department of Biotechnical and Clinical Laboratory Sciences; Lawrence Wrabetz and M. Laura Feltri, both co-directors of the HJKRI and members of the departments of Biochemistry and Neurology, as well as the Neuroscience Program.

Duc Nguyen and Ernesto R. Bongarzone of the Department of Anatomy and Cell Biology in the College of Medicine at the University of Illinois at Chicago also participated in the research.

The project was initiated with the support from Empire State development fund for HJKRI, and further developed and finalized by the R01, R56 and R03 grants from National Institutes for Health-National Institute for Neurological Disorders and Stroke awarded to Shin.

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Addenbrooke’s becomes first regional centre to offer CAR-T cell treatment for cancer patients – Cambridge Network

Posted: November 13, 2020 at 4:56 am

The decision to offer the therapy puts Cambridge University Hospitals in the premier league of world class cancer centres.

It also supports progress of the new Cambridge Cancer Research Hospital on the Addenbrookes site, which the Government confirmed in October is included in the second wave of the Hospital Infrastructure Programme (HIP2).

To begin with, the pioneering treatment will be offered to patients with aggressive B-cell lymphomas and acute lymphoblastic leukaemia (ALL) who have relapsed or not responded well to chemotherapy or stem cell treatment.

It is also likely to be offered to cancer patients aged over 70 who are considered to be too high risk to have stem cell transplants.

CAR-T cell therapy can have extremely positive results, but because it can cause unpleasant side effects, it tends to be used when there are few other treatment options available.

CAR-T cell therapy works by re-engineering the patients own immune cells.

Our immune cells, which are called T-cells, circulate around the bloodstream seeking out and destroying any alien intruders, such as infections.

But because cancer cells evolve from our own cells, sometimes our T-cells do not identify them as intruders, and leave them alone.

The new CAR-T cell therapy works by harvesting a patients own T-cells. These are sent to a specialist lab, where they are reprogrammed to express a molecule on their surface, called a Chimeric Antigen Receptor, or CAR, that targets them to the cancer. The reprogrammed cells are grown in a huge numbers over a few weeks and then infused back into the patients body, where they seek out and destroy the cancer. Its a bit like giving immune cells a sat nav.

Until now patients in the east of England needing CAR-T cell therapy have had to make frequent trips to London in preparation for the treatment, which can take another month to administer.

One such patient was Steve Johnson from Bourne who underwent CAR-T cell therapy for relapsed leukaemia at the University College Hospital in London as part of a clinical trial.

Steve explained: "Having the treatment is not pleasant I had a number of fevers and temperature spikes for two weeks after the CAR-T cells were put back in, but I have absolutely no doubt this treatment saved my life and without it I would not be here today."

Steve added: I was lucky for me the trial came at the right time. Having the option to explore and provide revolutionary treatments at places like Addenbrookes and the soon to be built Cambridge Cancer Research Hospital, is vital if we are going to rewrite the story of this devastating illness.

The first patients to be approved for CAR-T cell treatment at Addenbrookes will start having their cells harvested from this week.

Ben Uttenthal, consultant haematologist specialising in CAR-T cell therapy at Addenbrookes, said: This is an exciting new way of treating patients that attacks cancer in a different way from previously available medicines.

"Being able to offer CAR-T cell therapy in our armoury against cancer puts us in the premier league of cancer centres.

It is also a testament to the expertise available across many different specialties in Addenbrookes. Through offering treatments like this, now and in the new Cambridge Cancer Research Hospital, we will be able to benefit cancer patients locally, regionally and nationally.

Initially, the hospital expects to be able to offer CAR-T cell therapy to around 40 patients a year. In the future, treatment is likely to be expanded to include patients with other cancers.

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"Revolutionary" cancer treatment to be offered at Addenbrooke’s Hospital Cambridge – In Your Area

Posted: November 13, 2020 at 4:56 am

By Alya Zayed

Pictured above: Addenbrookes patient Steve Johnson who received the new treatment. Photo credit: Addenbrooke's Hospital.

A revolutionary cancer treatment that could work on patients not responding to chemotherapy will be offered by Addenbrookes Hospital.

The hospital will be the first in East Anglia to offer CAR-T cell treatment, which puts it in the premier league of cancer centres, said one doctor from Cambridge University Hospitals NHS Foundation Trust, which runs the hospital.

The pioneering treatment will be offered to patients with some cancers who have relapsed or not responded well to chemotherapy or stem cell treatment - namely, aggressive B-cell lymphomas and acute lymphoblastic leukaemia (ALL).

It is also likely to be offered to cancer patients aged over 70 who are considered to be too high risk to have stem cell transplants.

The new therapy can have extremely positive results, but is used as a last resort because of its unpleasant side effects, such as high fevers and low blood pressure.

CAR-T cell therapy works by re-engineering the patients own immune cells.

Immune cells, which are called T-cells, circulate around the bloodstream seeking out and destroying any intruders, such as infections.

But because cancer cells evolve from our own cells, sometimes T-cells do not identify them as intruders, and leave them alone.

The new CAR-T cell therapy works by harvesting a patients own T-cells. These are sent to a specialist lab, where they are reprogrammed to express a molecule on their surface, called a Chimeric Antigen Receptor, or CAR, that targets them to the cancer.

The reprogrammed cells are grown in huge numbers over a few weeks and then infused back into the patients body, where they seek out and destroy the cancer.

It can be a bit like giving immune cells a Sat-Nav, explained the hospital.

The first patients to be approved for CAR-T cell treatment at Addenbrookes will start having their cells harvested from this week.

Initially, the hospital expects to be able to offer CAR-T cell therapy to around 40 patients a year.

In the future, treatment is likely to be expanded to include patients with other cancers.

Ben Uttenthal, consultant haematologist specialising in CAR-T cell therapy at Addenbrookes, said: This is an exciting new way of treating patients that attacks cancer in a different way from previously available medicines.

Being able to offer CAR-T cell therapy in our armoury against cancer puts us in the premier league of cancer centres.

It is also a testament to the expertise available across many different specialties in Addenbrookes.

Through offering treatments like this, now and in the new Cambridge Cancer Research Hospital, we will be able to benefit cancer patients locally, regionally and nationally.

Until now, patients in East Anglia have had to travel to London for the treatment.

Addenbrookes patient Steve Johnson, from Bourn, south Cambridgeshire, is one of many who went to University College Hospital, London, to take part in a clinical trial.

Steve, whose leukemia had relapsed, said: Having the treatment is not pleasant I had a number of fevers and temperature spikes for two weeks after the CAR-T cells were put back in, but I have absolutely no doubt this treatment saved my life and without it I would not be here today.

I was lucky for me the trial came at the right time. Having the option to explore and provide revolutionary treatments at places like Addenbrookes and the soon to be built Cambridge Cancer Research Hospital, is vital if we are going to rewrite the story of this devastating illness.

The new Cambridge Cancer Research Hospital on the Addenbrookes site was confirmed by the government in October as one of the new hospitals to be built.

See the latest news, information, conversations and much more, all tailored to your neighbourhood, in your InYourArea live feed here.

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Orchard Therapeutics Unveils Details on New HSC Gene Therapy Research Programs as Part of R&D Investor Event Tomorrow at 9:00 a.m. ET – GlobeNewswire

Posted: November 13, 2020 at 4:56 am

First look at preclinical data in frontotemporal dementia with progranulin mutations (GRN-FTD) and new amyotrophic lateral sclerosis (ALS) program

NOD2 mutation revealed as Crohns disease (CD) genetic target, associated with 7-10% of all CD cases in the U.S. and Europe

Deep dive on transduction enhancers and stable cell line technology innovations that support manufacturing for larger indications

BOSTON and LONDON, Nov. 12, 2020 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today previewed details on its investigational hematopoietic stem cell (HSC) gene therapy research programs in GRN-FTD and NOD2-CD in advance of an upcoming virtual R&D investor event. The company also disclosed a new research program in ALS. A live webcast of the presentation will be available in the Investors & Media section of the companys website at http://www.orchard-tx.com starting Friday, November 13, 2020 at 9:00 a.m. ET.

We are excited to draw back the curtain at tomorrows event on our work in larger indications that form an important part of Orchards evolution as a company, including a new program in ALS, in addition to our work in genetic subsets of FTD and Crohns disease, said Bobby Gaspar, M.D., Ph.D., chief executive officer, Orchard Therapeutics. These research programs have been established using a scientific approach that has resulted in more than 160 patients being treated across multiple rare diseases and a recent positive CHMP opinion in the EU for Libmeldy. We believe that HSC gene therapy has the power to transform lives, and we are excited about the possibilities for Orchard and patients with its expanded application.

OTL-204 for GRN-FTD and new ALS research program

The GRN-FTD and ALS programs are based on the same HSC gene therapy approach that has been clinically validated with Libmeldy (OTL-200), Orchards program for metachromatic leukodystrophy, and is under clinical evaluation in the OTL-203 and OTL-201 programs for mucopolysaccharidosis type I and mucopolysaccharidosis type IIIA, respectively. Development work in GRN-FTD and ALS will be undertaken as part of a collaboration with Boston Childrens Hospital (BCH), the University of Padua (UNIPD) and Prof. Alessandra Biffi, chair of the Pediatric Hematology, Oncology and Stem Cell Transplant Division at UNIPD and co-director of the Gene Therapy Program at BCH.

Prof. Biffi commented, The ability of HSC gene therapy to restore healthy microglia function supports the use of this technology for the development of treatments for a variety of diseases with central nervous system involvement. In GRN-FTD, initial in vitro data shows progranulin expression and secretion in culture and uptake indicative of cross-correction. My previous work at BCH researching ALS supports the novel approach of treating this severe neurodegenerative condition by targeting the NOX2 pathway.

OTL-104 for NOD2-CD

Orchards preclinical program in CD targets mutations in the nucleotide-binding oligomerization domain-containing protein 2 (NOD2) gene, which plays a role in immune cell response to bacterial peptides in the gastrointestinal (GI) tract. The companys proposed approach leverages this link, using gene modified HSC-derived cells (monocytes) to replace GI resident macrophages, thus potentially correcting the inflammation and colitis associated with NOD2-CD.

Manufacturing Innovations to Support Work in Larger IndicationsTransduction enhancers (TEs) and stable cell line technology (SCLT)

Orchard has completed a thorough TE screening process and identified and validated several novel TE compounds, which in combination, facilitate lentiviral vector entry into HSCs and have shown a greater than 50% reduction in vector requirements. The enhancers mode of action is expected to be effective in each of Orchards HSC gene therapy programs. An evaluation of enhancer-treated HSC engraftment potential in mice is currently underway.

The company has worked extensively with SCLT, including the technology licensed from GSK for certain programs, to both develop processes to efficiently create SCLs for new vectors and scale up the production of SCLs to clinical grade. Results have delivered consistent levels of high-titer lentiviral production comparable to those seen using conventional methods. Selection of single high-titer clones for new vectors using this method has been achieved within three months. Work at Orchard is ongoing to develop upstream and downstream processes to further improve productivity and scalability.

We have a clear roadmap for Orchards future that prioritizes strategic growth and draws on the many synergies across our scientific, manufacturing and emerging commercial platforms, said Frank Thomas, president and chief operating officer. Over the next 12 months we have an array of exciting commercial, regulatory and clinical milestones that will continue to showcase the breadth and depth of our advanced HSC gene therapy portfolio.

Webcast Information

A live webcast of the presentation New Horizons in Gene Therapy will be available under Events in the Investors & Media section of the companys website at http://www.orchard-tx.com. A replay of the webcast will be archived on the Orchard website following the presentation.

About Orchards Research Collaborations

In connection with its previously disclosed collaboration with Prof. Alessandra Biffi, Orchard has signed agreements with Boston Childrens Hospital and the University of Padua to develop and exclusively license new ex vivo HSC gene therapy programs, patents and technologies for the treatment of neurodegenerative disorders. As part of the collaboration, Orchard has initiated sponsored research agreements and obtained exclusive options to license multiple new preclinical programs, including frontotemporal dementia with progranulin mutations (GRN-FTD), amyotrophic lateral sclerosis (ALS) and other rare and less rare indications.Orchard continues to support Professor Biffis labs in the development of new proprietary technology focused on enhancing the application of gene-modified HSC therapy for CNS disorders.

About Orchard

Orchard Therapeutics is a global gene therapy leader dedicated to transforming the lives of people affected by rare diseases through the development of innovative, potentially curative gene therapies. Our ex vivo autologous gene therapy approach harnesses the power of genetically modified blood stem cells and seeks to correct the underlying cause of disease in a single administration. In 2018, Orchard acquired GSKs rare disease gene therapy portfolio, which originated from a pioneering collaboration between GSK and theSan Raffaele Telethon Institute for Gene Therapy in Milan, Italy. Orchard now has one of the deepest and most advanced gene therapy product candidate pipelines in the industry spanning multiple therapeutic areas where the disease burden on children, families and caregivers is immense and current treatment options are limited or do not exist.

Orchard has its global headquarters in London and U.S. headquarters in Boston. For more information, please visit http://www.orchard-tx.com, and follow us on Twitter and LinkedIn.

Availability of Other Information About Orchard

Investors and others should note that Orchard communicates with its investors and the public using the company website (www.orchard-tx.com), the investor relations website (ir.orchard-tx.com), and on social media (Twitter and LinkedIn), including but not limited to investor presentations and investor fact sheets, U.S. Securities and Exchange Commission filings, press releases, public conference calls and webcasts. The information that Orchard posts on these channels and websites could be deemed to be material information. As a result, Orchard encourages investors, the media, and others interested in Orchard to review the information that is posted on these channels, including the investor relations website, on a regular basis. This list of channels may be updated from time to time on Orchards investor relations website and may include additional social media channels. The contents of Orchards website or these channels, or any other website that may be accessed from its website or these channels, shall not be deemed incorporated by reference in any filing under the Securities Act of 1933.

Forward-Looking Statements

This press release contains certain forward-looking statements about Orchards strategy, future plans and prospects, which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, plans, intends, projects, and future or similar expressions that are intended to identify forward-looking statements.Forward-looking statements include express or implied statements relating to, among other things, Orchards business strategy and goals, including with respect to its manufacturing strategy, expected future milestones, and its plans and expectations for the development of its product candidates, including the product candidates referred to in this release, and the therapeutic and commercial potential of its product candidates.These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, these risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including the product candidates referred to in this release, will not be approved, successfully developed or commercialized; the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials of Orchards product candidates will not be repeated or continue in ongoing or future studies or trials involving its product candidates; the risk that the market opportunity for its product candidates may be lower than estimated; and the severity of the impact of the COVID-19 pandemic on Orchards business, including on preclinical and clinical development, its supply chain and commercial programs. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading Risk Factors in Orchards quarterly report on Form 10-Q for the quarter ended September 30, 2020, as filed with the U.S. Securities and Exchange Commission (SEC), as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaChristine HarrisonVice President, Corporate Affairs+1 202-415-0137media@orchard-tx.com

1Knopman DS, Roberts RO. J Mol Neurosci. 2011, Onyike CU, Diehl-Schmid J. Int Rev Psychiatry. 2013 and Riedl L, et al Neuropsychiatr Dis Treat. 20142 Centers for Disease Control and Prevention; European Crohns and Colitis Organisation (ECCO); Ashton, James J et al.Clin Transl Gastroenterol. 2020 Feb

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CAR T-Cell Therapy for Multiple Myeloma: What Community Oncologists Need to Know – OncLive

Posted: November 13, 2020 at 4:56 am

Multiple myeloma is a plasma cell malignancy that accounts for approximately 1% of new cancer diagnoses per year. The development of a combination approach of mainstay therapies including immunomodulatory drugs (IMiDs), proteasome inhibitors (PIs), and antiCD-38 antibodies has greatly improved up-front remission rates and response duration and prolonged overall survival in the past decade. However, for patients who have been heavily exposed to or are triple or penta-refractory to these classes of therapy, survival is under 1 year.1 Therefore, the emergence of cellular-based therapies represents a major opportunity to improve outcomes in the heavily pretreated and refractory myeloma population.

Chimeric antigen receptor (CAR) T-cell therapies approved by the FDA for several relapsed and refractory B-cell lymphomas are presently under investigation in a variety of treatment line settings for multiple myeloma. As more patients in the community become eligible for CAR T-cell therapies, community oncologists will need to be increasingly familiar about the various products, including their immediate and longer-term risks. In addition, it is key to understand the optimal time for referring these patients to an academic institution, as well as how to manage the requisite post CAR T-cell therapy in the community setting.

The major CAR T-cell therapies in trials for multiple myeloma are directed toward B-cell maturation antigen (BCMA), a surface antigen expressed on B cells starting from their period of development in the germinal centers onward and persisting through the time of plasma cell differentiation. BCMA is minimally expressed in normal human tissues but is heavily expressed on myeloma plasma cells. This allows for safe and intentional targeting of neoplastic myeloma cells using CAR T-cell technology.

The first FDA-approved commercial CAR T-cell therapy will likely arrive in the first quarter of 2021. Patients enrolled on the initial BCMA CAR T-cell trials were required to have had 3 or more prior lines of therapy with exposure to a PI, an IMiD, and an anti-CD38 monoclonal antibody.

The earliest trial was the KarMMa-1 study (NCT03361748) of idecabtagene vicleucel (ide-cel, previously bb2121), which demonstrated an overall response rate (ORR) of 73% and a median progression-free survival (PFS) of 8.8 months in all dose levels. Additionally, a median PFS of 12.1 months was observed in the highest dose level of CAR T cells (450 106).2,3 Patients on this trial (n = 128) had a median of 6 prior lines of therapy, and 84% were triple refractory.

A concurrent trial, CARTITUDE-1 (NCT03548207), studying JNJ-4528 (ciltacabtagene autoleucel in China), also targeting BCMA, demonstrated an unprecedented 100% ORR in patients, with 86% achieving stringent complete response (sCR), the deepest response level by International Myeloma Working Group criteria.4 In the most recent evaluation of PFS, 86% of patients on CARTITUDE-1 had not progressed in disease at a median follow-up time of 9 months.5 New data for JNJ-4528 and the other CAR T-cell therapies being investigated will be made available during the 2020 American Society of Hematology Annual Meeting and Exposition in December and should further reinforce the potential for CAR T-cell therapy to deliver deep and durable responses to patients with multiple myeloma who are heavily pretreated.

What Type of Patients Might Benefit the Most From CAR T?

As in the case of BCMA, antigens for CAR T cells are chosen for their unique expression in tumor tissue and low expression in normal, healthy tissue. The complications that occur in patients receiving CAR T-cell therapy will be largely specific to the targeted antigen. Patients must undergo a multistep process to safely and effectively undergo CAR T-cell treatment and should know up front about the process involved.

The initial step involves leukapheresis, or collecting the patients T cells. Once this is completed, the patient must wait 3 to 5 weeks for typical manufacturing of their CAR T cells at a production facility. If required, patients typically can undergo bridging chemotherapy during this waiting period to maintain their disease, particularly if myeloma progression is accelerating. Once the patients CAR T cells are produced, the patient will undergo lymphodepletion (typically with fludarabine/cyclophosphamide) for 3 days before being infused with prepared CAR T cells. Patients must have sufficient health reserve to withstand the potential adverse effects (AEs) associated with CAR T-cell therapy, including cytokine release syndrome (CRS) and immune effector cellassociated neurotoxicity syndrome (ICANS). Therefore, early referral to an academic center is key to getting these patients on therapy at the optimal time.

CRS is the most common acute AE that occurs in patients treated with CAR T-cell therapy. It occurred in nearly 9 out of 10 patients in the KarMMa-1 and CARTITUDE-1 trials, though severe CRS occurred only in 5% to 7% of patients.3,5 CRS is the phenotypic presentation of a supraphysiologic production of cytokines prompted by immune cell activation, including T cells, macrophages, and monocytes (Table 16 ). Preclinical models have shown that macrophages amplify CRS severity and are the source of several of the culprit cytokines of CRS, including interleukin (IL) 6, IL-1, and IFN-. CRS onset occurs variably depending on the CAR construct used.

Table 1. ASTCT Criteria for CRS6

For example, CRS occurred at median 1 day after infusion of ide-cel, whereas CRS occurred at median 7 days after infusion of JNJ-4528. Our primary treatment for CRS is tocilizumab (Actemra), a monoclonal antibody directed against IL-6; its use does not diminish efficacy of CAR T cells. Subsequent agents such as the IL-1 receptor antagonist anakinra (Kineret), antiIL-5 monoclonal antibody siltuximab (Sylvant), and corticosteroids may be employed for severe cases of CRS.

The second notable AE associated with CAR T-cell therapy is ICANS, which is defined by the American Society for Transplantation and Cellular Therapy as a disorder characterized by a pathological process involving the central nervous system.Symptoms or signs can be progressive and may include aphasia, altered level of consciousness, impairment of cognitive skills, motor weakness, seizures, and cerebral edema.6 A primarily encephalopathic picture appears when patients develop ICANS, which typically occurrs later than CRS. The primary treatment for ICANS involves corticosteroids such as dexamethasone, initially every 6 hours with a quick taper as symptoms start resolving.

Signs and symptoms of neurotoxicity should be screened at least daily during the acute CAR T-cell treatment period and can be assessed with validated tools such as those from CAR T-cell therapy-associated TOXicity (CARTOX) Working Group or the Immune-Effector Cell-Associated Encephalopathy (ICE) tool.6 Severe neurotoxicity occurred at only a 3% rate in KarMMa-1 and CARTITUDE-1 and can be treated readily if caught early with close monitoring.

Other common AEs such as cytopenia and infection may occur during and after the period of CAR T-cell infusion and expansion. Anemia, thrombocytopenia, or neutropenia may occur for months after the initial treatment period, and clinicians should utilize supportive transfusions and/or granulocyte colonystimulating factor and thrombopoietin mimetics as indicated.

Cytomegalovirus quantitative polymerase chain reaction should be checked monthly. If the patient is persistently neutropenic, opportunistic infections should be considered. Intravenous immunoglobulin should be administered before initial lymphodepletion, but also every month for 6 months after CAR T-cell infusion and during winter months if the patient has a history of recurrent infections. In addition, patients should be vaccinated at 1-year post CAR T-cell therapy; a recommended vaccine schedule is included in Table 2.

Table 2. Recommended Vaccine Schedules After CAR T-Cell Therapy

The multiple myeloma treatment landscape is rapidly evolving, with many exciting new therapies on the forefront for relapsed and refractory multiple myeloma. CAR T-cell therapy is a 1-time treatment that allows patients to achieve deep and durable remissions without the need for the usual continuous cancer-directed treatments. The aforementioned trials and therapies have paved the way for subsequent iterations of cellular therapies. These include nonBCMA-targeted CAR T cells; next-generation CAR T cells with improved costimulatory domains for improved safety, efficacy, or faster production; and allogeneic CAR T cells derived from pluripotent stem cells available off the shelf to patients. In the coming year, as commercially available myeloma CAR T-cell therapies are approved, it will be even more important for community oncologists to better understand these therapies so they can offer them to their patients.

References

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Leading Human Immunology and Infectious Disease Experts to Join UM School of Medicines Institute of Human Virology – Newswise

Posted: November 13, 2020 at 4:56 am

Newswise Baltimore, MD, November 12, 2020 Robert C. Gallo, MD, the Homer & Martha Gudelsky Distinguished Professor in Medicine at the University of Maryland School of Medicine (UMSOM) and Co-Founder & Director of the UMSOMs Institute of Human Virology (IHV), announced today that a team of leading scientists in human immunology, virology and stem cell biology, led by Lishan Su, PhD joined IHV on October 1 with academic appointments in the UMSOM Department of Pharmacology. As part of the Maryland E-Nnovation Initiative Fund (MEIF) to recruit top research faculty and a donation to IHV from the Charles Gordon Estate, Dr. Su has been named the Charles Gordon Smith Endowed Professor for HIV Research. Dr. Su will also head IHVs Division of Virology, Pathogenesis and Cancer.

The team will include a 12-person Laboratory of Viral Pathogenesis and Immunotherapy with two faculty appointments as well as major public and private sector research funding.

Dr. Gallo made the announcement in conjunction with University of Maryland School of Medicine Dean E. Albert Reece, MD, PhD, MBA and Margaret M. McCarthy PhD, James & Carolyn Frenkil Deans Professor, Chair of the Department of Pharmacology.

Dr. Su is one of the most successful active basic researchers in America, said Dr. Gallo, who is also Co-Founder and Chairman of the International Scientific Leadership Board of the Global Virus Network. His research is groundbreaking, and we are so pleased to have him join IHV and lead our Division of Infectious Agents and Cancer, which under his sound leadership, will flourish.

Dr. McCarthy added:Dr. Sus continuing ground-breaking work in HIV and Hepatitis B will be a huge asset to the Department of Pharmacology. I look forward to working with him on advances that could open the door to new therapeutics.

Dr. Su was a faculty member in the Lineberger Comprehensive Cancer Center and Professor in the Department of Microbiology & Immunology at University of North Carolina-Chapel Hill since 1996. He received his BS degree in Microbiology from Shandong University, his PhD degree in Virology from Harvard University, and did his post-doctoral training in Stem Cell Biology & Immunology at Stanford University. He worked as a senior research scientist at SyStemix/Sandoz (Novartis), focusing on HIV-1 pathogenesis and stem cell-based gene therapy in humanized mice and in patients.

I am excited to continue and expand my research programs at the Institute of Human Virology (IHV), said Dr. Su. I have long been impressed by the Baltimore-DC area's research centers with great basic and clinical research programs. IHV, co-founded and directed by Dr. Robert Gallo, is one of the first research institutes in the U.S. to integrate basic science, population studies and clinical trials to understanding and treating human virus-induced diseases. The Department of Pharmacology, headed by Dr. Margaret McCarthy, in the University of Maryland School of Medicine, has been outstanding in developing novel therapeutics including breast cancer drugs. I look forward to working with my new colleagues at IHV and the Department of Pharmacology, and across the University of Maryland School of Medicine, to expand and translate my research programs to treating human inflammatory diseases including virus infection and cancer.

Dr. Su has extensive research experience in human immunology, virology and stem cell biology. Dr. Su made important contributions to several areas of human immunology and infectious diseases, particularly in studying human immuno-pathology of chronic virus infections. His lab at UNC-Chapel Hill published important findings in identifying novel virological and immunological mechanisms of HIV-1 pathogenesis. Furthermore, his lab established humanized mouse models with both human immune and human liver cells that support HCV or HBV infection, human immune responses and human liver fibrosis. In recent years, Dr. Sus group discovered, and focused on, the pDC-interferon axis in the immuno-pathogenesis and therapy of chronic HIV & HBV infections. The group also started investigation of the pDC-IFN axis in tumor microenvironments and in cancer immune therapy.

Im so pleased to welcome Dr. Su to our faculty. His work advances the mission of the School of Medicine, which is to provide important new knowledge in the area of immunology and chronic disease to discover new approaches for treatments, said Dean Reece, who is also University Executive Vice President for Medical Affairs and the John Z. and Akiko K. Bowers Distinguished Professor. Dr. Sus stellar research capabilities will provide vital opportunities for collaboration across our Institutes and Departments.

About the Institute of Human Virology

Formed in 1996 as a partnership between the State of Maryland, the City of Baltimore, the University System of Maryland and the University of Maryland Medical System, IHV is an institute of the University of Maryland School of Medicine and is home to some of the most globally-recognized and world-renowned experts in all of virology. The IHV combines the disciplines of basic research, epidemiology and clinical research in a concerted effort to speed the discovery of diagnostics and therapeutics for a wide variety of chronic and deadly viral and immune disorders - most notably, HIV the virus that causes AIDS. For more information,www.ihv.organd follow us on Twitter @IHVmaryland.

About the University of Maryland School of Medicine

The University of Maryland School of Medicine was chartered in 1807 and is the first public medical school in the United States and continues today as an innovative leader in accelerating innovation and discovery in medicine. The School of Medicine is the founding school of the University of Maryland and is an integral part of the 11-campus University System of Maryland. Located on the University of Marylands Baltimore campus, the School of Medicine works closely with the University of Maryland Medical Center to provide a research-intensive, academic and clinically based education. With 43 academic departments, centers and institutes and a faculty of more than 3,000 physicians and research scientists plus more than $400 million in extramural funding, the School is regarded as one of the leading biomedical research institutions in the U.S. with top-tier faculty and programs in cancer, brain science, surgery and transplantation, trauma and emergency medicine, vaccine development and human genomics, among other centers of excellence. The School is not only concerned with the health of the citizens of Maryland and the nation, but also has a global vision, with research and treatment facilities in more than 30 countries around the world. For more information, visitwww.medschool.umaryland.edu.

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Angiocrine Bioscience Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to AB-205 (Universal E-CEL Cell Therapy) to…

Posted: November 13, 2020 at 4:55 am

Angiocrine Bioscience Announces FDA Regenerative Medicine Advanced Therapy (RMAT) Designation Granted to AB-205

About Regenerative Medicine Advanced Therapy (RMAT) DesignationEstablished under the 21st Century Cures Act, the RMAT designation was established to facilitate development and expedite review of cell therapies and regenerative medicines intended to treat serious or life-threatening diseases or conditions. Advantages include the benefits of the FDA's Fast Track and Breakthrough Therapy Designation programs, such as early interactions with the FDA to discuss potential surrogate or intermediate endpoints to support accelerated approval.

About HDT-AHCT High-dose therapy and autologous hematopoietic cell transplantation (HDT-AHCT) is considered a standard-of-care therapy for patients with aggressive systemic Hodgkin lymphoma (HL) and non-Hodgkin lymphoma (NHL).Although efficacious and considered a potential cure, HDT-AHCT is associated with severe regimen-related toxicities (SRRT) that increase patient morbidity and risk for mortality, especially in the aging population. Effective prevention of SRRT may lead to more patients being eligible for a potential cure through HDT and stem cell transplantation.

About SRRT Consequences of Diffuse Injury to the Organ Vascular NichesThe human body is capable of renewing, healing and restoring organs.For example, the human oral-GI tract renews its lining every 3 to 7 days. Both the organ renewal and healing processes are dependent on organ stem cell vascular niches made up of stem cells, endothelial cells (cells that line blood vessels) and supportive cells.When tissues are injured, the vascular niche endothelial cells direct the stem cells, via angiocrine factor expression, to repair and restore the damaged tissue. This restorative capacity is most active during childhood and youth but starts to diminish with increasing age.HDT provided to eradicate cancer cells also cause diffuse, collateral damage to vascular niches of multiple healthy organs. In particular, the organs with the highest cell turnover (ones with most active vascular niches) are severely affected.Specifically, the oral-GI tract, dependent on constant renewal of its mucosal lining, starts to break down upon vascular niche injury.The mucosal breakdown can cause severe nausea, vomiting and diarrhea. In addition, the bacteria in the gut may escape into the circulation, resulting in patients becoming ill with endotoxemia, bacteremia or potentially lethal sepsis.HDT-related vascular niche damage can also occur in other organs resulting in severe or life-threatening complications involving the lung, heart, kidney, or the liver.Collectively, these complications are known as severe regimen-related toxicities or SRRT.SRRT can occur as frequently as 50% in lymphoma HDT-AHCT patients, with increased rate and severity in older patients.

About AB205AB-205 is a first-in-class engineered cell therapy consisting of proprietary 'universal' E-CEL (human engineered cord endothelial) cells.The AB-205 cells are intravenously administered after the completion of HDT on the same day as when the patient's own (autologous) blood stem cells are infused. AB-205 acts promptly to repair injured vascular niches of organs damaged by HDT.By repairing the vascular niches, AB-205 restores the natural process of tissue renewal, vital for organs such as oral-GI tract and the bone marrow. Successful and prompt organ restoration can prevent or reduce SRRT, an outcome that is beneficial to quality of life and cost reductive to the healthcare system.

About CIRMThe California Institute for Regenerative Medicine (CIRM) was established in November, 2004 with the passage of Proposition 71, the California Stem Cell Research and Cures Act. The statewide ballot measure provided $3 billion in funding for California universities and research institutions.With over 300 active stem cell programs in their portfolio, CIRM is the world's largest institution dedicated to stem cell research. For more information, visit http://www.cirm.ca.gov.

About Angiocrine Bioscience Inc.Angiocrine Bioscience is a clinical-stage biotechnology company developing a new and unique approach to treating serious medical conditions associated with the loss of the natural healing and regenerative capacity of the body.Based on its novel and proprietary E-CEL platform, Angiocrine is developing multiple therapies to address unmet medical needs in hematologic, musculoskeletal, gastrointestinal, soft-tissue, and degenerative/aging-related diseases.A Phase 3 registration trial is being planned for the intravenous formulation of AB-205 for the prevention of severe complications in lymphoma patients undergoing curative HDT-AHCT.This AB-205 indication is covered by the Orphan Drug Designation recently granted by the US FDA.In addition, Angiocrine is conducting clinical trials of local AB-205 injections for the treatment of: (1) rotator cuff tear in conjunction with arthroscopic repair; and, (2) non-healing perianal fistulas in post-radiation cancer patients.

For additional information, please contact:

Angiocrine Bioscience, Inc.John R. Jaskowiak(877) 784-8496[emailprotected]

SOURCE Angiocrine Bioscience, Inc.

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