Every generation of the Pokmon games can be defined by its starters. These are the three Pokmon that the player gets to choose from at the beginning of the game. They are always of the Fire, Water, and Grass types and are usually strong enough to remain by the players side throughout the game. The original starters Bulbasaur, Charmander, and Squirtle remain some of the most iconic Pokmon after over 20 years.

Leading up to the release of Pokmon Sword and Shield, the fandom was divided (all in good fun this time) among players who planned to start with the gregarious Grass-type Grookey, the weepy Water-type Sobble, and the flamboyant Fire-type Scorbunny. But regardless of which starter everyone chose, the next big question on everyones minds was: What will their evolutions look like? Because starter Pokmon always evolve twice throughout the game from a small, cute pocket monster to a big, scary final evolution. Evolved Pokmon are stronger than their lesser forms, able to learn the most powerful moves and hold their ground against the other legendary monsters that populate their world.

In the real world, just like in Pokmon, evolution refers to the process whereby one species transforms into another species. However, in nature evolution occurs via a process that Charles Darwin called natural selection. In short, new species arise as the result of small inherited genetic variations that convey survival advantages. These variations build up over time until a new species has differentiated from its predecessor. The variations are the result of genetic mutations, which can be caused by a variety of mechanisms such as errors in DNA replication and exposure to mutagenic substances. Suffice to say, the natural process of evolution takes many generations of being exposed to specific natural selection pressures.

In Pokmon, however, the term evolution is used in the broadest sense not to describe the specific process of evolution by natural selection, but rather as a catch-all term for the transformation of one Pokmon species into another. The choice to use the word evolution goes back to the original Japanese versions of the game, which used the Japanese word shinka literally evolution. Yet while Pokmons evolution does not map one-to-one to the scientific concept of evolution, it does map strongly to another biological concept: the life cycle.

In biology, life cycle refers to the series of major biological changes an organism goes through during its lifetime. The butterfly provides an elegant example: It starts life as an egg that then hatches to become a caterpillar. That caterpillar eats and grows until it can grow a chrysalis around itself. Within the chrysalis, it goes through metamorphosis and transforms into a beautiful butterfly. That butterfly grows, mates, and lays eggs, and the cycle begins again. Thats a life cycle.

There are multiple Bug-type Pokmon that go through this exact life cycle. Caterpie, the caterpillar Pokmon, evolves into a glorified chrysalis called Metapod and then soon after evolves again into the butterfly Pokmon Butterfree. Caterpies evolution maps one-to-one to the life cycle of real-world butterflies.

All of this makes a lot of sense once you learn a bit about Pokmons creator, Satoshi Tajiri of Game Freak. In a 1999 interview, Tajiri revealed that he had a deep interest in bug collecting as a child. Tajiri lamented seeing urbanization of his hometown reduce the number and variety of bugs for children to collect, and this directly inspired Pokmons game design.

Tajiri was not asked about the choice to use the word shinka (evolution) specifically, but it seems evident that the word is used in the broadest possible sense. Rather than mapping to the scientific concept of evolution by natural selection, evolution in Pokmon describes the transformation from one species into another species by any mechanism. Even the term species is used in a not-quite-biological way it is applied to the different stages of the life cycle of a single Pokmon. In biology, we consider organisms at different stages of their life cycle to be the same species, but in Pokmon theyre called different species.

We can even note Pokmons representation of evolution-as-life-cycle in the context of the game systems. Pokmon is a Japanese role-playing game (JRPG) and uses a system whereby experience points (XP) are earned by battling Pokmon against each other. Once enough XP is accrued, the Pokmon gains a level, which makes it stronger. Many Pokmon evolve once they hit a certain level in the game. Its almost like the levels actually represent age. If imagined in this hypothetical light, once a Pokmon hits a certain age, it evolves and advances along its life cycle.

While a lot of the Bug-type Pokmon evolve in a way reminiscent of a butterfly, starter Pokmon evolution looks a lot like the life cycle that mammals go through. Think about the human life cycle humans start out as babies (the first stage), then go through a period of rapid growth (adolescence, the second stage), and finally become adults (the third stage). The evolution of the starter Pokmon in each generation follows this same path. In fact, the visual design of the starter Pokmon is often directly inspired by the toddler-adolescent-adult transformation many animals undergo during their natural life cycle in the real world.

While a lot of Pokmon evolve upon hitting a certain level of experience, there are others who only evolve after being exposed to certain items. Take Pikachu, for example. Pikachu is without a doubt the most famous of all Pokmon, but many outsiders may not realize that Pikachu can evolve into a bigger, stronger Pokmon named Raichu. In order to evolve into Raichu, Pikachu needs to be exposed to an item called a Thunder Stone. No matter how much experience and how many levels Pikachu has earned, the Thunder Stone is the key to reaching the next stage of its life cycle. There are many other Pokmon that require an elemental stone to evolve as well, but they all basically follow the same life cycle as Pikachu.

It might be hard to imagine theres a real-world equivalent to this kind of life cycle in nature, but it does indeed exist. To find it, we must look to Tajiris beloved world of insects once again. The closest real-world life cycle that matches the evolution of Pikachu and the other elemental stone Pokmon is the honey bee! Yes, the very same honey bee species that is responsible for making that sweet treat we all love.

Honey bees have three different forms they can take. There are worker bees, who are all infertile females and are responsible for, well, doing all the work such as gathering pollen, taking care of the hive, and so forth. Then there are the drones, all of whom are male, who mainly exist for the purpose of mating. And finally, there are the queen bees. Queen bees are all female just like worker bees, but they are larger and they are fertile. Their job is, essentially, to mate with the drones and lay tons of eggs.

Any fertilized honey bee egg has the potential to become a queen bee. In fact, a worker bee and a queen bee could be genetic clones, but they are very different anatomically, physiologically, and behaviorally. These differences arise because the queen bee is fed large amounts of a special food the worker bees produce called royal jelly. While all honey bee larvae eat small quantities of royal jelly, the queen bee larvae are literally swimming in it. There is evidence that the royal jelly has an effect on the epigenetic state that is, the activation and deactivation state of various genes of the queen bee larvaes genome. These changes prioritize metabolism and maintain fertility while deprioritizing features queen bees dont need, like pollen-collecting baskets. The end result is a completely different type of bee from the workers, all because the larvae were exposed to a large amount of royal jelly.

So perhaps, in the Pokmon world, a Thunder Stone behaves like royal jelly for a Pikachu activating and deactivating genes and leading to a transformation into another, more advanced form. And that may also explain why the elemental stones cause multiple different Pokmon to evolve into their final forms. Perhaps the epigenetic effect works on more than one species.

In a recent interview, Pokmon Sword and Shield director Shigeru Ohmori and producer Junichi Masuda admitted that they werent sure how evolution works biologically in the Pokmon world. Masuda even said that the science of how Pokmon work isnt really set in stone. But even if the scientific details of how it works in the Pokmon world arent clear, its certainly true that a lot about Pokmon evolution is inspired by real-world biological life cycles.

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The Real-World Science That Underlies Pokmon Evolution (and Thunder Stones) - The Escapist

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Two-and-a-half weeks after elite runner Mary Cain aired allegations of forced weight loss and public shaming in a Nike-supported running program, she is still calling for a third-party investigation into the matter.

Her op-ed video I Was the Fastest Girl in America for The New York Times had more than six million views as of Monday afternoon. Cain, who signed up with the now-disbanded Nike Oregon Project in 2013, claimed the programs coach Alberto Salazar publicly shamed her for not being thin enough and bred an eating disorder culture. She exited the program in 2016 after allegedly telling Salazar she has been cutting herself as a form of a self-harm, essentially getting no reaction and phoning her parents to share her problems.

In late September, Salazar was handed a four-year ban from the U.S. Anti-Doping Association for orchestrating and facilitating prohibited doping conduct at Nikes Oregon Project. Nike Inc. executives initially stood by Salazar and then back-peddled days later, cutting ties with the former Olympian. In response to Cains video, Nike challenged Cain in a statement issued earlier this month for not raising her concerns in April when she was looking to rejoin the program. The company also announced plans to launch an immediate investigation to hear from former Oregon Project athletes.

But Cain said Friday, I havent heard much more beyond [that and] they dont seem willing to do a third-party investigation even though I called for it. There really hasnt been much change from their platform and position, so there really hasnt been from mine either.

Should Nike commit to a third-party investigation, Cain said she would happily cooperate. As for whether there will be any legal ramifications against Nike, she said, I dont really know going forward. The truth is a lot of that Im keeping closer to my chest.

A Nike spokesman did not acknowledge requests for comment Monday about the status of the investigation, with specific questions including whether there will be a third-party investigation.

Speaking in broader terms, the recent Fordham grad said she is a big believer in Corporate Social Responsibility in that companies are not people. They are made up of people. The individuals within a company should step up in situations like this and really demand change. If they really believe in a product that they are trying to sell, then they should make sure they are doing it responsibly. And they should make sure that the people they are using for advertising and to sell that product are being treated properly.

She added, In situations like this, a brand should step up and say, Hey, we messed up. Lets do a third-party investigation and make sure that we get this right for the future. Thats the responsible thing to do.

From her perspective, internal investigations inhibit athletes from speaking openly because thats their sponsor and how they make money. Cain said. The company has kind of victim-blamed me in a lot of their statements so that will breed mistrust and a little bit of confusion as to what they plan on doing within an investigation and whether it will be taken seriously.

On a typical day Cain runs between six and 14 miles. Her 30-hour weekly training schedule is comparable to her training at the Oregon Project time-wise, but it is more balanced. She spends 10 hours running with the remaining 20 hours related to recovery. Back then it was 30 hours of heavy training. Now there is more recovery work, physical therapy and things that are less intensive, but that are still time commitments, she said.

Asked if she has signed any new endorsement deals, Cain said, Oh, no, no, this is not why I did this. But after the op-ed broke, various brands sent her running gear. Cain declined to identify any of the companies. She said, The biggest thing that Ive appreciated is that a lot of brands have stepped up and sent me stuff. As a professional runner, for many years Ive been given Nike clothes. Its been kind of cool and fun to try something new and to do something that I havent done in six years train in non-Nike gear.

In a study of Division 1 NCAA athletes, more than one-third of female athletes reported attitudes and symptoms placing them at risk for anorexia nervosa, according to the National Eating Disorders Association. Non-athletes are also suffering from eating disorders more than 10 million American women alone. The issue has been an area of great concern in fashion where models livelihoods are tied to their physiques. Council of Fashion Designers of America first zeroed in on the problem in 2007, by circulating its Health Initiative.

All in all, Cain said she never imagined her story would break past the track world, she said. I just honestly assumed that most people were so ingrained within the system that the reaction would be 50-50 positive and negative. I just genuinely did not expect it to have such an international and national reach. Its really hopeful that it did, because it means theres more opportunity for change. But its almost sad that it did, because it shows how systemic it is and how broadly people can empathize and sympathize with such and experiences. It really does transcend sport.

Allowing that being lean and strong can be desirable for performance, Cain emphasized that taking arbitrary numbers just to be skinny does not create strength and power. And it doesnt make a good athlete. The issue with my story was not the fact that I had a coach who wanted me to lose weight. Sometimes athletes are expected to lose weight over the course of the season. But usually they work with a professional nutritionist. Theres not some arbitrary target thats trying to be reached. Its more go through the process train hard, eat well. Maybe weight would fall off, maybe it wont. Rather than almost targeting a look, she said.

Competitive sports like gymnastics, wrestling, long-distance running, figure skating, diving and dancing may have greater risks of eating disorders, since body size is highly scrutinized among some competitors. With figure skaters, ballerinas and runners, there is very much a picturesque view that the coach will put on the board to say you have to look like that. Genetics dont work like that, Cain said. Societal pressures can really force girls to look one specific way, when in reality theres not one way to be good.

Kara Goucher, Yoder Begley and other world-class runners substantiated Cains claims and pledged their support earlier this month. Goucher offered to share her own experiences via social media. Cain has yet to meet with any of them in the past few weeks, due to geographical challenges. It is really cool to know now that I have this almost second team. Ive obviously made a lot of great connections through this, said Cain, adding she periodically receives texts from supporters to see how she is doing.

While some in the media and via social media have challenged The New York Times for not recognizing Cains struggles when she was profiled for a 2015 magazine piece, Cain defended the new outlet. I was so in the system. They kept the door closed on the reporter [Lindsay Crouse], who I worked with so tightly. I was in college at the time and they made it so difficult for her to get in touch with me. Any time they were going to sit down and talk, a coach or somebody had to be in the room. The process was very difficult. The truth is it was a cult and we really didnt let people in behind the closed doors ever, Cain said. I absolutely dont hold anybody accountable for having not known what was going on. At that time, I wasnt even fully vocal with my parents let alone a reporter I didnt know.

She continued, In any situation like this, theres a tendency to cast blame whether its on the victim, family, friends, the sports world, media anything. But its most important to reflect on the fact that this is systemic and yes, in my case, there were certain bad eggs. But the truth is so much of what I went through so many people do. Its about rewriting the general, societal and cultural pressures that we put on people to make sure this doesnt happen again.

Cultural awareness, as in high school and NCAA coaches discussing the issue with their teams to make people feel comfortable about being more open and talking about their experiences, would be a good starting point, according to Cain. Longer-term, educational programs for coaches and athletes are needed, perhaps along the lines of sexual abuse awareness ones that have proven to be effective, she said. By doing so, athletes would recognize instances of abuse before they escalate to remove themselves or to cause the abuser to be removed sooner.

With an undergrad degree in business and having completed her premed requirements, Cains short-term career plans are to pursue running. On a broader sense, she aims to take on advocacy roles for women in sports with the hope of changing the system. Longer-term, Im not quite sure. In so many ways, I just love athletics. It would be really hard for me to pivot outside of the sports world. But I have so many aspirations in that regard that Im just going to let the next few years play out and take it one step at a time, Cain said.

As for whether Cain has any regrets, she said there are always regrets after situations like this. But she reminds herself that some of the issues that she suffered from at the Oregon Project are systemic throughout running. I cant say with confidence that had I not been in the NCAA or gone to another program that some of these issues wouldnt have still been a problem, she said. Based on the reaction of my piece, its really clear that a lot of girls on so many different levels within the sport go through this. My experience was particularly egregious, based on the nature of it being a professional program. But nonetheless, that does not mean it was a one-off take. Its easy to look back and say, Oh, I should have done something sooner or I shouldnt have listened to them. But I believe that everything happens for a reason. Its incredibly sad to reflect upon what happened. But I cant live my life with regret because then you can never move forward.

Originally posted here:
Mary Cain Still Calling on Nike to Hold Third-Party Probe Into Disbanded Oregon Project - WWD

Recommendation and review posted by Bethany Smith

While it goes without saying that three-time CrossFit Games champion Tia-Clair Toomey and the Icelandic Dottirs have been inspiring CrossFit fans with their brilliant performances for years, there are so many other equally inspiring CrossFit women out therewomen who inspire beyond their competition performances.

There are so many ways I could have gone with this list, but I went the route of 10 women who have exposed their true personalitieswho have opened up vulnerably, expressed insecurities and broken downand have made me laugh and made me cry.

(In no particular order):

This five-time CrossFit Games athlete has moved away from high-level CrossFit competition and has embarked on a path of trying to become pregnant and start a family with her female partner. She has been incredibly brave and open about her struggles to start a family.

On September 10, 2019, Lance-McWherter posted this video on Instagram, where she shared her emotions after another negative pregnancy test.

This time was the absolute worst. It hit me the hardest. I think because I was soooo hopeful this time and I really felt like it would take, she wrote in the post.

As gut-wrenching as her post was, its also a breath of fresh air to watch something so authentic and real.

This 2015 CrossFit Games athlete might just be the most entertaining and witty competitor out there. Her social media posts will make you laugh. Period.

But shes more than just an entertainer. Shes also super honest in her posts about topics most people would shy away from confronting. The best example I can remember was when her partner Meredith Root qualified to the CrossFit Games and she didnt. Most athletes would likely just pretend to be happy for their partner, Parker admitted that, although happy for Root, it was incredibly difficult for her.

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A lot of people asked me this weekend how it was competing against @meredith_root. It was a great experience, however, it was very emotionally challenging. Ill try to explain why, and I will be honest. I am so incredibly competitive in literally everything I do, that I actually annoy myself. And for some reason, I hate when Meredith beats me more than when anyone else does. I get temporarily, insanely and selfishly jealous when she wins, no matter what the competition is. It just doesnt feel right to feel this way towards someone I am dating. That is whats hard about it. Luckily we are open with each other about these feelings. Having said that, I also love to see her succeed, even when its at my expense. Its a frustrating mix of emotions that I really struggle to manage, but being in a relationship with someone who also is competitive has several advantages. I have someone who experiences the similar ups and downs that come with the territory, eats the same food, and understands when I spend all my free time in the gym. And because she is someone I train with, training quality is much higher. Accordingly, our coach only programs 1v1 workouts a couple of times a week so we dont kill each other. Its an extra benefit when that person is someone that gets you (and is a much better cook than you!). It goes without saying that I would not have done as well as I did this weekend without her. She not only supported me through the weekend, but through the whole year. Not to mention, she has, and continues to, help me survive my demanding and time consuming job. Okay, okay, Ill admit it she treats me like a princess. Its also difficult because Im not used to being emotionally invested in someone elses success. When they fail, you feel it. In this moment, she is upset with falling short of her goal, and I felt it like it was me . . . But I still hate that she beat me. Like I said, its a mix of emotions. As hard as a relationship can be at times, it seems to be worth the struggles. Im pretty darn lucky. We are both stronger individually from this weekend and even stronger together. Im already excited for next year. @crossfitgames #regionals2018 #proud

A post shared by Alex Parker (@aaparker1) on May 30, 2018 at 4:54pm PDT

I hate when Meredith beats me more than when anyone else does. I get temporarily, insanely and selfishly jealous when she wins, no matter what the competition is, Parker wrote. Its a frustrating mix of emotions that I really struggle to manage.

Though some might fear admitting this type of emotion to the world would make them less likable, it only made Parker more relatable.

I know I said this wouldnt be performance-based inspiration, per se, but I absolutely cannot leave this seven-time CrossFit Games athlete off the list. After taking a year off competing to give birth to her first child, Saunders appears to be 100 percent back to her former fitness level and recently placed 12th in the CrossFit Open, unofficially qualifying to this summers CrossFit Games.

Through her endearing social media posts, where she takes her baby swimming, its easy to see Saunders is as passionate about being a mother as she is an athlete.

After competing at the CrossFit Games as an individual every single year from 2010 to 2018, Leblanc-Bazinet competed on a team at the Games for the first time last year. After 10 consecutive CrossFit Games appearances, Leblanc-Bazinet is not competing this year.

Moving away from competition can be incredibly hard emotionally for any high level athlete, and what has made Leblanc-Bazinet so inspiring to me this year is how she seems to be redefining what fitness is for her now (its unclear if she has fully retired or is just taking a year off).

Through the months, she has been sharing about how she has embraced more traditional bodybuilding-type training. Its reassuring to know that when you leave something behind, there are many new doors that open and can be as fulfilling as what you left behind.

Michele Letendres story is similar to Leblanc-Bazinets. After competing at the CrossFit Games five times, Letendre stepped away from competition in 2017 and has quickly turned herself into a world-renowned CrossFit coachpossibly the most underrated coach out there. Last year, Letendre coached four CrossFit Games athletes, including Patrick Vellner, Laura Horvath, James Newbury, and Samuel Cournoyer.

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Today I was thinking a lot about the different roles Ive had in my life. This morning I was on my way to the @dekacrossfit and I was going to fix a broken audio wire. I was thinking to myself: man its a good thing I played with electronics at school because otherwise, Id have to get someone else to do this simple job. . . That made me realize that Ive done a lot of different things throughout my life. Ive scooped ice cream, drawn, painted, sculpted Ive worked as a hotel receptionist, Ive lead design projects with school, Ive worked with LEDs, sensors and even a 3D printer Ive sold insuranceIve played waterpolo and competed in CrossFit, then in weightlifting. Ive coached CrossFit in gyms and now in competition. Im newly a gym owner and an owner of @dekacomp . Im only 33 years old. I feel very fortunate to have this much experience at my age and I cant wait to learn more. . I was speaking with my mother not long ago about leadership and gym ownership, she offered her help and it seemed so simple and effective. She said to me, Michele, Ive been doing this for 30 years, and only have I really put things together in the last 5. That got me excited to know that I can only get better and more concise with my message to the world through time and experience I felt a surge of confidence that through my continued exposure to the world and the multiple roles I will take on, i will end up with a solid and meaningful contribution. . . . What have your experiences been? How many roles have you played so far and how do you feel they connect? Lets hear it!

A post shared by Michele Letendre (@mich_letendre) on Oct 30, 2019 at 11:19am PDT

More than anything, though, having met Letendre numerous times, I can say with certainty this woman is genuine as they come.

The older generation in society always like to rag on the younger generation. These days, I often hear adults talking about the poor work ethic of teenagers today, or that they spend too much time on their phones, and definitely that theyre entitled.

Adams, however, is proof there are some seriously hard workers under the age of 20.

Shes only 19 and she has already competed at four CrossFit Gamesthree times in the teenager divisionand she placed sixth last summer in her rookie year in the womens division. And its clear she has only continued to grow in 2019. There couldnt be a better role model for young up-and-comers than this teenager.

I couldnt leave this woman off the list. She has been competing at the CrossFit Games since 2009. In fact, Thorisdottir has only missed one CrossFit Games in the last decade 2013 because of an injury.

And just when we thought she might be starting to fade when she placed 38th in 2015 and 13th in 2016, she came back in 2017 and placed 3rd. Considering Thorisdottir just placed 2nd in the CrossFit Open, its safe to say she isnt fading yet. Her staying power is absolutely inspiring.

This three-time CrossFit Games athlete is proof you can do it all. She has managed to continue to become fitter and fitter each year Huckaby placed 3rd at Games with her Invictus team last summer summer all the while prioritizing the trials and tribulations of motherhood. Theres a sincerity to Huckabys voice on her social media posts that melts your heart.

This list wouldnt be complete without this six-time individual CrossFit Games athlete, who also competed in the Womens 35-39 division at the 2018 CrossFit Games. Like Thorisdottir, Briggs ability to remain competitive year after year is incredible.

But what I love most about her is how she doesnt try to be anything she isnt, and through all her success over the years, she remains humble and down to earth.

What I love most about this four-time individual and two-time team CrossFit Games athlete is her willingness to be courageous and address hard issues. Though Fisher is obviously incredibly lean, she doesnt necessarily have the rippling eight-pack abs many of her competitors possess. And believe it or not, online bullies have brought this to her attention.

On April 20th, Fisher addressed them head on:

How come you exercise so hard and dont have a flat belly? asked the bullies.

I dont have a flat belly because I believe in fueling my body for performance over restricting my calories to look a certain way. Yeah, if I starved myself or cut down my fats and still trained as much as I do I might finally get that 8 pack but reality is my body type wont. I like to eat and I know when I dont eat enough I just dont feel good during training. And a lot of it also comes down to genetics. So Im sorry but I will never be one of the girls with no belly fat at all and Im okay with it, she wrote.

Amen, sister.

Featured image: @mich_letendre on Instagram

Continue reading here:
10 CrossFit Women Who Inspired Us In 2019 - BarBend

Recommendation and review posted by Bethany Smith

Intermountain Primary Children's Hospital, along with University of Utah Health, and Intermountain Precision Genomics are teaming up to launch a pediatric center for personalized medicine that will serve the Intermountain West.

WHY IT MATTERS The center will use precision genomics to discover, address and treat genetic diseases, many of which affect infants and children and can cause life-long disability.

The Center will focus on precision diagnosis, gene therapies and novel therapeutics, and stem cell, immunologic and regenerative medicine.

Precision medicine includes applications across diagnostics, prevention and screening that takes into account individual variabilities in genes, environment, and lifestyle for every individual.

Through its work on precision diagnosis, the Center hopes to provide more targeted care to critically-ill children based on their genetic make-up, where rapid whole genome sequencing can quickly identify genetic causes of hard-to-diagnose diseases.

The initial efforts will be focused on providing answers to critically ill infants in the newborn intensive care unit, and children with severe seizures and heart conditions.

The research into gene therapies and novel therapeutics will help enable children with previously debilitating and fatal genetic diseases, with clinical trials testing gene therapy treatments for Duchenne's Muscular Dystrophy, Adrenoleukodystrophy, and other serious diseases.

The Center is also developing novel therapeutics that target specific diseases and improve health, with a release noting the Center is one of only six hospitals nationwide to provide gene therapy for the common childhood genetic condition spinal muscular atrophy.

Stem cell research uses a child's own cells, or genetically modifies a child's cells and immune system, to fight disease and promote healing, with additional research aimed at developing immunotherapy as a tool to fight pediatric brain tumors.

The organization also noted clinical trials are testing the use of stem cells in repairing diseased hearts and other tissues.

THE LARGER TRENDIntermountain has been busy on this front recently. In June, the health system announced that it is performing a massive clinical DNA study, pairing 500,000 samples drawn from Intermountain Healths patient population and analyzing them with help from deCODE, a subsidiary of Reykjavik-based Amgen.

"Better health and being able to cure common diseases is the promise of precision medicine, but its not happening fast enough," said Dr. Marc Harrison, president and CEO at Intermountain Healthcare, announcing that initiative. "For too long, the genetic code to better health has been locked. This collaboration with deCODE unlocks that insight so we can rapidly advance well-being not only for ourselves and our families, but for generations to come.

Intermountain's new pediatrics personalized medicine announcement also follows Mount Sinai's just-announced plans to build new precision medicine supercomputer, which will have 15 terabytes of memory, 14 petabytes of raw storage and a peak speed of 220 teraflops per second, to manage massive amounts of genomic data.

ON THE RECORD"Our mission is to leverage the expertise of our scientists, the clinical care of our physicians and care-givers, and the dedication of our community, to discover and develop new cures for children," said Dr. Josh Bonkowsky, Intermountain's medical director of the Primary Children's Center for Personalized Medicine, in a statement. "The work we are doing here and now is transforming pediatric medicine. We will not be done until we have put these diseases out of business."

Nathan Eddy is a healthcare and technology freelancer based in Berlin.Email the writer:nathaneddy@gmail.comTwitter:@dropdeaded209

Healthcare IT News is a publication of HIMSS Media.

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Intermountain to open new center for pediatric precision medicine - Healthcare IT News

Recommendation and review posted by Bethany Smith

The age of artificial intelligence is allowing us to rethink the way that we treat diseases and disabilities. The combination of AI and Big Data, in addition to helping with medical diagnosis, coupled with biological delivery systems, such asgene therapy delivery systemcan significantly alter the way we treat a host of diseases that are, according to modern science, incurable: cancer, autism, some mental illnesses, and rare genetic illnesses. Specifically, combining AI, big data, robotics, gene therapy, and medical research has unleashed a host of possibilities to cure these types of diseases. At the same time, the combined innovation efforts are helping people with disabilities live their lives better.

Heres an overview of some of these advances as we move into the new year.

For years, cancer treatment has been at the forefront of medical research. There have been different attempts to address the various types of the disease. However, with AI and the promise of a gene-editing tool such asthe CRISPR/Cas9, researchers finally have a way to convert cancer cells into non-cancerous cells by deleting genes and re-engineer the cells. The application of this procedure is particularly useful in certain types of blood cancers, where the proliferation of the cancerous cells can spread rapidly.A recent study sponsored by the University of Pennsylvania, the Parker Institute of Cancer Immunotherapy and Tmunity Therapeutics, received promising results trying this new procedure on patients in a clinical trial.Although the results are pending and the path forward is complex, this form of cancer treatment may finally point to a cure.

Autism is a developmental spectrum disorder that affects an increasing number of children in the world. It is a debilitating disorder that inhibits normal child development in children who are on the Autism Spectrum. Currently, theres no cure. The promise of AI has allowed companies to attempt to help those living with Autism to live better lives and to integrate into the world. Robots such as theQTrobot andMiloare teaching children social skills and identifying their emotions. Other robots, such as the InMoov teach children sign language, whileZenoteaches children how to communicate, andKASPARreciprocates mechanical love.The autism glass project from Stanford Universityaims at helping children on the Autism spectrum to live better by providing feedback in social situations, such as greeting people, expressing needs, and resolving conflict to help them adjust to situations in daily life.

Due to Autism being viewed as a developmental disorder rather than a disease, research has not focused heavily on a cure.When Elon Musk unveiled his Neuralink research and white paper, this year, suddenly, there emerged debates around whether Autism can be cured. Musk in his interview on the Artificial Intelligence podcast with Lex Fridman, revealed that Neuralink can potentially treat many brain-related diseases such as autism.

Musk was quoted sayingSo Neuralink I think at first will solve a lot of brain-related diseases. he continued: So could be anything from, like, autism, schizophrenia, memory loss like, everyone experiences memory loss at certain points in age. Parents cant remember their kids names and that kind of thing.

Neuralinks goal is to develop an AI-enabled chip that will be implanted in the brain to gather information, monitor and potentially stimulate the brain to optimize the brains activities. It is not clear whether treating autism as a disease of the brain and not a developmental disorder is the right way to go. It is also not clear that the Neuralink project will be able to make progress in this direction without causing side effects. Nevertheless, the thought that the brains activities can even be augmented by AI unleashes new ways of thinking around finding other ways to help children with ASD, as well as numerous other neural dysfunctions.

In recent years, with the help of AI, most of the advances in mental health treatment have been around therapy (medication, counseling and more) and diagnosis.AI-enabled therapy apps and robots such as BetterHelp, ReGain, Woebot, and Wysa is helping patients around the clock day and night to manage mental health issues outside of a therapists available hours.

This is helping to take the stigma out of mental health issues as well as making therapies more accessible to the general population.

In the area of diagnosis, AI-enabled therapy systems using Big Data such as theLeso Digital Healthwill provide a host of evidence and forecast a probability of diagnosis for the therapist, so that the final diagnosis can be more evidence-based. Delivering Cognitive Behavior Therapy through written forms of communication, the AI system can use the information to measure and improve the treatment plan. It is also allowing therapists to quantify their treatment plans using data, while not losing the qualitative aspect of treatment.

Recently advances in the area of Prosthetic devices have allowed people with disabilities to see the possibilities of improved livelihood in the age of artificial intelligence.Bionic Eye such as the Argus II systemapproved by the FDA allows people with poor vision to see shapes. It will allow people with poor vision to engage in daily activities such as reading large print books and to cross the street.Ossur, a global leader in prostheticsprovides AI-enabled knee devices to enable amputees to walk in a natural way with bionic limbs. Starkey,AI medical device company that developed the Livio AI, has developed a hearing aid that will not only enhance the hearing experience by quieting all the external noise from the environment, it will also track health-related data to enable patients to seek help during emergency situations.

As we advance into the age of Artificial Intelligence, there will be more synergies created between technological innovations in artificial intelligence, big data, robotics, and medical research. The research provides evidence for the application of new possibilities in treatment, diagnosis, and healthcare management. In time, this research will become a crucial part of developing better treatment plans for those living with chronic disabilities and illnesses.

Excerpt from:
How AI is Changing the Way We Treat Diseases and Disabilities - Forbes

Recommendation and review posted by Bethany Smith

(Reuters) - Eleven drugmakers led by Pfizer and Novartis have set aside a combined $2 billion to invest in gene therapy manufacturing since 2018, according to a Reuters analysis, in a drive to better control production of the worlds priciest medicines.

FILE PHOTO: A logo for Pfizer is displayed on a monitor on the floor at the New York Stock Exchange (NYSE) in New York, U.S., July 29, 2019. REUTERS/Brendan McDermid

The full scope of Novartis (NOVN.S) $500 million plan, revealed to Reuters in an interview with the companys gene therapy chief, has not been previously disclosed. It is second only to Pfizer (PFE.N), which has allocated $600 million to build its own gene therapy manufacturing plants, according to filings and interviews with industry executives.

Gene therapies aim to correct certain diseases by replacing the missing or mutated version of a gene found in a patients cells with healthy copies. With the potential to cure devastating illnesses in a single dose, drugmakers say they justify prices well above $1 million per patient.

But the treatments are also extremely complex to make, involving the cultivation of living material, and still pose a risk of serious side effects.

Drugmakers say building their own manufacturing plants is a response to rising costs and delays associated with relying on third-party contract manufacturers, which are also expanding to capitalize on demand.

They say owning their own facilities helps safeguard proprietary production methods and more effectively address any concerns raised by the U.S. Food and Drug Administration (FDA), which is keeping a close eye on manufacturing standards.

Theres so little capacity and capability at contract manufacturers for the novel gene therapy processes being developed by companies, said David Lennon, president of AveXis, Novartiss gene therapy division. We need internal manufacturing capabilities in the long term.

The approach is not without risks.

Bob Smith, senior vice president of Pfizers global gene therapy business, acknowledged drugmakers take a leap of faith when they make big capital investment outlays for treatments before they have been approved or, in some cases, even produced data demonstrating a benefit.

The rewards are potentially great, however.

Gene therapy is one of the hottest areas of drug research and, given the life-changing possibilities, the FDA is helping to speed treatments to market.

It has approved two so far, including Novartiss Zolgensma treatment for a rare muscular disorder priced at $2 million, and expects 40 new gene therapies to reach the U.S. market by 2022.

There are currently several hundred under development by around 30 drugmakers for conditions from hemophilia to Duchenne muscular dystrophy and sickle cell anemia. The proliferation of these treatments is pushing the limits of the industrys existing manufacturing capacity. Developers of gene therapies that need to outsource manufacturing face wait times of about 18 months to get a production slot, company executives told Reuters.

They are also charged fees to reserve space that run into millions of dollars, more than double the cost of a few years ago, according to gene therapy developer RegenxBio.

As a result, companies including bluebird bio (BLUE.O), PTC Therapeutics (PTCT.O) and Krystal Biotech (KRYS.O) are also investing in gene therapy manufacturing, according to a Reuters analysis of public filings and executive interviews.

They follow Biomarin Pharmaceutical Inc (BMRN.O), developer of a gene therapy for hemophilia, which constructed one of the industrys largest manufacturing facilities in 2017.

The FDA is keeping a close eye on standards.

This comes amid the agencys disclosure in August that it is investigating alleged data manipulation by former executives at Novartis AveXis unit.

AveXis had switched its method for measuring Zolgensmas potency in animal studies. When results using the new method didnt meet expectations, the executives allegedly altered the data to cover it up, the FDA and Novartis have said.

One of the former executives, Brian Kaspar, denied wrongdoing in a statement to Reuters. Another, his brother Allan Kaspar, could not be reached for comment.

Novartis and the FDA say human clinical trials, which found Zolgensma effective in treating the most severe form of spinal muscular atrophy in infants, were not affected. Novartis also says its investments in gene therapy production started long before it became aware of the data manipulation allegations.

But the scandal has highlighted the importance of having a consistent manufacturing process for gene therapies, industry executives say.

According to four of them, the FDA has stressed in recent meetings the need for continuity in production processes all the way from the development of a drug to its commercialization.

By bringing production in-house, drugmakers may avoid pitfalls such as the need to switch to a larger facility if contract manufacturers capacity proves limited, executives say.

The FDA is finalizing new guidelines for gene therapy manufacturing, expected at the end of the year.

Manufacturing consistency is always a major concern for the agency, FDA spokeswoman Stephanie Caccomo told Reuters.

Highlighting the pressures on the industry, Sarepta Therapeutics (SRPT.O), which largely outsources manufacturing, delayed a clinical trial of its Duchenne treatment in August, telling investors it wanted to avoid any questions from regulators about consistency in producing its therapy at commercial scale.

Between the trade secrets, the cost schedules and the time lag, it makes a whole lot of sense, if you can do it, to build out your own facilities and more and more gene therapy companies have started to do that, said Krish Krishnan, chief executive of Krystal Biotech Inc.

Krystal, which is developing therapies for rare skin diseases, has built one manufacturing facility and plans to invest more than $50 million in a new one it will start constructing in December.

MeiraGTx (MGTX.O), which focuses on gene therapies for eye conditions, estimates it is currently spending roughly $25 million a year on manufacturing, including process development.

Despite such moves, however, contract manufacturers like Lonza (LONN.S) and Thermo Fisher (TMO.N) are confident their businesses will continue to grow due to the strength of demand.

Thermo Fisher has told investors its Brammer gene therapy manufacturing division, acquired in May, could soon earn $500 million in revenue a year, double its projected 2019 earnings. Lonza CEO Marc Funk is also optimistic.

Demand in gene therapy has increased, he said in an interview. We believe this is going to continue in the coming years.

Reporting by Carl O'Donnell in New York and Tamara Mathias in Bengaluru; Editing by Tomasz Janowski, Michele Gershberg and Mark Potter

Originally posted here:
Pfizer, Novartis lead pharma spending spree on gene therapy production - Reuters

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Gene Therapy Market - Forecasts from 2019 to 2024" report has been added to ResearchAndMarkets.com's offering.

The global gene therapy market is expected to grow at a CAGR of 16.92% reach $8.748 billion by 2024 from $3.425 billion in 2018.

Gene therapy is an experimental technique that involves the introduction of DNA usually containing a functional gene into a patient for the treatment of genetic disorders. Gene therapy works by replacing, repairing or repressing the mutated gene. Therefore, with the right target and approach, it can address the root cause of a severe disease where known genetic mutations lead to deficient or non-functional protein production or other related problems. The gene therapy may be a promising option for genetic diseases such as muscular dystrophy and cystic fibrosis.

The growth in the market may be attributed to the increase in awareness among the people regarding the benefits of gene therapy coupled with the increased funding activities for research and development of gene therapy by various governments. However, the high cost of gene therapy might restrain the growth of the market during the given forecast period. Problems with suppressing the immune responses due to the introduction of new genes may also hamper the market growth during the given time frame.

The Global Gene Therapy Market - Forecasts from 2019 to 2024 is an exhaustive study that aims to present the key market trends through various chapters focusing on different aspects of the market. The study provides a detailed market overview through the market dynamics sections which detail key market, drivers, restraints, and opportunities in the current market. The report analyzes key opportunity regional markets, and the current technology penetration through lifecycle analysis. The report also analyzes the market through comprehensive market segmentation by Technique, Vector Type, Indication and geography.

Major players in the gene therapy market have been covered along with their relative competitive position and strategies. The report also mentions recent deals and investments of different market players over the last year. The company profiles section details the business overview, financial performance for the past three years, key products and services being offered along with the recent developments of these important players in the gene therapy market.

Key Topics Covered:

1. INTRODUCTION

1.1. Market Overview

1.2. Market Definition

1.3. Scope of the study

1.4. Currency

1.5. Assumptions

1.6. Base, and Forecast Years Timeline

2. Research methodology

2.1. Research Design

2.2. Secondary Sources

3. Executive Summary

4. Market Dynamics

4.1. Market Segmentation

4.2. Market Drivers

4.3. Market Restraints

4.4. Market Opportunities

4.5. Porter's Five Forces Analysis

4.5.1. Bargaining Power of Suppliers

4.5.2. Bargaining Power of Buyers

4.5.3. Threat of New Entrants

4.5.4. Threat of Substitutes

4.5.5. Competitive Rivalry in the Industry

4.6. Life Cycle Analysis - Regional Snapshot

4.7. Market Attractiveness

5. Gene Therapy Market by Technique

5.1. Gene Augmentation

5.2. Gene Inhibition

5.3. Killing of specific cells

6. Gene Therapy Market by Vector Type

6.1. Viral Vectors

6.2. Non-Viral Vectors

7. Gene Therapy Market by Indication

7.1. Genetic Disorders

7.2. Cardiovascular

7.3. Oncology

7.4. Others

8. Gene Therapy Market by Geography

8.1. North America

8.1.1. USA

8.1.2. Canada

8.1.3. Mexico

8.2. South America

8.2.1. Brazil

8.2.2. Argentina

8.2.3. Others

8.3. Europe

8.3.1. UK

8.3.2. Germany

8.3.3. Italy

8.3.4. Spain

8.3.5. Others

8.4. Middle East and Africa

8.4.1. Israel

8.4.2. Saudi Arabia

8.4.3. Others

8.5. Asia Pacific

8.5.1. China

8.5.2. Japan

8.5.3. India

8.5.4. Australia

8.5.5. Others

9. Competitive intelligence

9.1. Competitive Benchmarking and Analysis

9.2. Strategies of Key Players

9.3. Recent Investments and Deals

10. Company Profile

10.1. Novartis

10.2. Amgen

10.3. Bluebird Bio

10.4. Glaxosmithkline

10.5. Shenzhen Sibiono Genetech

10.6. Spark Therapeutics

10.7. AveXis, Inc.

10.8. Chiesi Farmaceutici

10.9. Alnylam

For more information about this report visit https://www.researchandmarkets.com/r/vcck48

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Gene Therapy Market Expected to Grow with a CAGR of 16.92% During the Forecast Period, 2019-2024 - ResearchAndMarkets.com - Business Wire

Recommendation and review posted by Bethany Smith

Vertex Pharmaceuticals is preparing to grow even biggeranother 256,000 square feet bigger, to be exact.

The drugmaker is in advanced talks to lease a building in Innovation Square, a research campus in Bostons Raymond Flynn Marine Industrial Park, The Boston Globe reported.

The target is the entire second phase of the new R&D hub that developer Related Beal is building on the South Boston waterfront. Its close to Vertexs existing 1.1 million-square-feet Fan Pier headquarters and would serve as a research and manufacturing facility for gene and cell therapies, according to the newspaper.

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Vertex scoured the greater Boston region for a new foothold, including sites in Cambridge, Waltham and Watertown, but picked the Innovation Square because its one of the most advanced projects in the neighborhood, on track to open in 2021, Vertex CEO Jeffrey Leiden reportedly said.

Expansion at the Innovation Square comes as the biotech giant diversifies beyond its fundamental cystic fibrosis business and into the burgeoning gene and cell therapy arena.

The question became how are we going to grow those programs if were running out of space at Fan Pier? said Leiden, as quoted by the Globe. The answer is a new building.

Leiden is transitioning to executive chairman, handing the baton to Chief Medical Officer Reshma Kewalramani. But before he moves up, a blueprint for Vertexs future growth has been laid out.

RELATED:The top 10 best-paying places to work in biopharma | 7. Vertex Pharmaceuticals

In June, Vertex put down $245 million upfront to acquire Exonics and its gene editing technology, which uses CRISPR to repair dystrophin, the protein missing in patients with Duchenne muscular dystrophy (DMD). At the same time, it shelled out $175 million upfront to deepen its ties with CRISPR Therapeutics, also for using CRISPR-Cas9 to develop DMD and myotonic dystrophy Type 1 therapies.

The first project coming out of the CRISPR-Vertex partnership has just shown promise. CTX001, a CRISPR-based therapy for severe blood disorders marked by abnormal hemoglobin, helped a beta thalassemia patient live without transfusions for nine months, and a sickle cell patient was free of the painful vaso-occlusive crises after four months, the pair unveiled last week.

Vertex also agreed to pay $950 million to snatch up Semma Therapeutics and its stem cell treatment for Type 1 diabetes.

RELATED:Vertex, CRISPR's gene-editing treatment for blood disorders shows promise in early data

The new building Vertex plans to lease will house 300 to 400 people, including employees from Exonics and Semma, as well as new hires, Leiden said, according to the Globe. Besides the lab and office space at its Fan Pier HQ, Vertex also has a lease for about 100,000 square feet of space in the Marine Industrial Park for certain logistical and laboratory operations and manufacturing equipment, the companys annual securities filing shows.

On the companys third-quarter earnings call in October, Leiden said the company will continue to do deals on early-stage assets, especially bolt-on deals to furtherits gene editing strategy.

Meanwhile, the cystic fibrosis franchise will continue to provideVertexs revenue backbone for some time. Last month, the company wonFDA approval for Trikafta, a triple combo designed to treat cystic fibrosis patients with a mutated delF508 gene, which is found in 90% of the U.S. cystic fibrosis population.

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Vertex plans major Boston expansion to support gene, cell therapy ambitions - FiercePharma

Recommendation and review posted by Bethany Smith

(Reuters) - Blackstone Group Inc (BX.N) said on Monday it will invest $400 million in a joint venture with Swiss drug company Ferring that is working on an experimental gene therapy for bladder cancer, the private equity giants largest ever bet on drug development.

FILE PHOTO: The ticker and trading information for Blackstone Group is displayed at the post where it is traded on the floor of the New York Stock Exchange (NYSE) April 4, 2016. REUTERS/Brendan McDermid

Investing in yet-to-be-approved medicines is a lucrative but also risky proposition for buyout firms, and only few have had the stomach to place such bets. Blackstone made its foray in the sector last year, acquiring Clarus, an investment firm specializing in life sciences.

For its part, Ferring will invest $170 million in the joint venture with Blackstone, dubbed FerGene, bringing its total funding to $570 million, the companies said in a statement.

FerGene is developing a gene therapy for bladder cancer patients with an aggressive form of the disease whose current options include having their bladder removed. The treatment works by entering the walls of the bladder where it releases a gene to trigger the patients own body to make a protein to fight off cancer.

We believe, and Ferring also believes, that this can change the standard of care in bladder cancer, a terrible disease, Nicholas Galakatos, senior managing director of Blackstone Life Sciences, said in an interview.

Oncology is a new area for Ferring, but it is one that we as Blackstone Life Sciences have a lot of experience in

The team assembled by Blackstone has worked at several of the worlds largest cancer drugmakers, including Roche unit Genentech, Merck & Co Inc (MRK.N), and Millennium Pharmaceuticals, now a part of Takeda Pharmaceutical Co Ltd (4502.T).

To minimize its risk, Blackstone invests in the late stages of drug development, when a medicine has already gone through important milestones. Late-stage drug development can also be expensive because of the clinical trials involved, something that Blackstone is seeking to capitalize on by partnering with pharmaceutical firms looking to share the cost burden.

FerGenes therapy, named nadofaragene firadenovec, is currently in the final stage of clinical research, results from which will be presented on Dec. 5 at the Society of Urologic Oncologys annual meeting.

Since it launched its life sciences unit, Blackstone has also formed a new company with Novartis AG (NOVN.S) to study a type of heart drug. Blackstone invested $250 million in that venture.

Reporting by Rebecca Spalding in New York; Editing by Alistair Bell

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Blackstone to invest $400 million in gene therapy venture with Ferring - Reuters

Recommendation and review posted by Bethany Smith

NEW YORK, Nov. 26, 2019 /PRNewswire/ -- Hoth Therapeutics, Inc. (Nasdaq: HOTH) ("HOTH" or the "Company"), a biopharmaceutical company focused on developing new generation therapies for dermatological disorders such as atopic dermatitis, chronic wounds, psoriasis and acne, today announced it has entered into a licensing agreement with North Carolina State University (NC State) to study NC State's Exon Skipping Approach for Treating Allergic Diseases.

This Exon Skipping Approach was developed by Dr. Glenn Cruse, Principal Investigator and Assistant Professor in the Department of Molecular Biomedical Sciences at the NC State College of Veterinary Medicine. During Dr. Cruse's research, a new approach for the technique of antisense oligonucleotide-mediated exon skipping to specifically target and down-regulate IgE receptor expression in mast cells was identified. These findings set a breakthrough for allergic diseases as they are driven by the activation of mast cells and the release of mediators in response to IgE-directed antigens.

Mr. Robb Knie, Chief Executive Officer of Hoth, commented, "This new collaboration will allow us to leverage this invention from the renowned expertise of Dr. Glenn Cruse and his scientific team at North Carolina State University. We look forward to seeing how their work advances and what this might mean for patients suffering from undesirable steroid side effects who need an alternate treatment for asthma and other allergic diseases."

The high-affinity IgEreceptor (FcRI) plays a central role in the initiation ofallergic responses. The research project looks to target novel genes, which are critical for surface IgE receptor expression. The project will utilize splice-switching oligonucleotides (SSOs) to force expression of a truncated isoform of the target genes to reduce expression ofFcRIin mouse asthma models.

Through this collaborative project, NCSU looks to establish the most effective approach for targeting genes that regulate surface expression of FcRI in mast cells that mediate allergic airway inflammation. The study will be administering SSOs for the target genes, to optimize delivery and examine the best therapeutic approach.

About Hoth Therapeutics, Inc.Hoth Therapeutics, Inc. isa clinical-stage biopharmaceutical company focused on developing new generation therapies for dermatological disorders. HOTH's pipeline has the potential to improve the quality of life for patients suffering from indications including atopic dermatitis, chronic wounds, psoriasis, and acne. HOTH has the exclusive worldwide rights to BioLexa, the company's proprietary lead drug candidate topical platform that uniquely combines two FDA approved compounds to fight bacterial infections across multiple indications. HOTH is preparing to launch its clinical trial for the treatment of adolescent subjects, 2-17 years of age, with mild to moderate atopic dermatitis during 2020. To learn more, please visitwww.hoththerapeutics.com.

Forward Looking StatementsThis press release includes "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Forward-looking statements in this press release include, but are not limited to, statements that relate to the advancement and development of the BioLexa Platform, the commencement of clinical trials, the availability of data from clinical trials and other information that is not historical information. When used herein, words such as "anticipate", "being", "will", "plan", "may", "continue", and similar expressions are intended to identify forward-looking statements. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon Hoth's current expectations and various assumptions. Hoth believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. Hoth may not realize its expectations, and its beliefs may not prove correct. Actual results could differ materially from those described or implied by such forward-looking statements as a result of various important factors, including, without limitation, market conditions and the factors described under the caption "Risk Factors" in Hoth's Form 10K for the period endingDecember 31, 2018, and Hoth's other filings made with the Securities and Exchange Commission. Consequently, forward-looking statements should be regarded solely as Hoth's current plans, estimates and beliefs. Investors should not place undue reliance on forward-looking statements. Hoth cannot guarantee future results, events, levels of activity, performance or achievements. Hoth does not undertake and specifically declines any obligation to update, republish, or revise any forward-looking statements to reflect new information, future events or circumstances or to reflect the occurrences of unanticipated events, except as may be required by law.

ContactsInvestor Relations Contact:Phone: (646) 756-2997Email:investorrelations@hoththerapeutics.comwww.hoththerapeutics.com

KCSA Strategic CommunicationsValter Pinto / Daniela Guerrero(212) 896-1254 / (212) 682-6300Hoth@kcsa.com

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Hoth Therapeutics and North Carolina State University Enter License Agreement for Gene Therapy - P&T Community

Recommendation and review posted by Bethany Smith

SOUTH SAN FRANCISCO, Calif., Nov. 21, 2019 /PRNewswire/ --CODA Biotherapeutics, Inc., a preclinical-stage biopharmaceutical company developing a chemogenetic gene therapy platform to treat neurological diseases, today announced the appointment of Annahita Keravala, Ph.D., as Senior Vice President, Gene Therapy. In this role, Annahita will lead the gene therapy aspects of CODA's chemogenetic platform. The company also announced the promotions of Orion Keifer, M.D., Ph.D., to Vice President, Discovery and Translational Research, and Steve Dodson, Ph.D., to Vice President, Pharmacology and Early Development, respectively.

Michael Narachi, President and Chief Executive Officer, said, "We are excited to have Annahita, Orion, and Steve in these vital roles as we advance our lead candidates toward the clinic, as well as build our pipeline based on CODA's chemogenetic gene therapy platform. Their combined gene therapy, neuroscience and small molecule expertise, and experience in early stage research and development, will prove invaluable. With this leadership team, we will fulfill our mission of discovering and developing transformative therapies for patients with intractable neurological diseases for whom limited or no treatment options exist."

Annahita brings more than two decades of experience in gene therapy using viral and non-viral vectors. In particular, she has extensive expertise in discovering novel vector technologies and gene therapy drug development for ophthalmic, systemic and inflammatory diseases. Annahita joins CODA from Rocket Pharmaceuticals, where she was Associate Vice President, AAV Platform. At Rocket, she provided strategic, scientific and operational leadership, oversaw all aspects of discovery research, preclinical and assay development, and provided technical insight to the Chemistry, Manufacturing and Controls (CMC) team. This culminated in a successful Investigational New Drug (IND) application filing.

"I am thrilled to be joining CODA at such an exciting time in the Company's development and growth. What attracted me was the opportunity to partner with Mike, Orion, Steve and our talented team to help bring cutting-edge therapeutic options to patients living with some of the most challenging and debilitating neurological conditions and disorders like chronic neuropathic pain and focal epilepsy for which there remains great unmet medical need," said Dr. Keravala.

Prior to her tenure at Rocket Pharma, Annahita held several positions of increasing responsibility at Adverum Biotechnologies (formerly Avalanche Biotechnologies). As Director of Adverum's Novel Vector Technology group, she designed the overall research strategy, led her team to discover and optimize next-generation adeno-associated virus (AAV) vectors, and oversaw process development and preclinical testing to support the company's pipeline. Earlier, Annahita was a Research Scientist at Stanford University School of Medicine. An author of multiple patents, she is also widely published in prestigious scientific journals. Annahita earned a Ph.D. in molecular genetics and biochemistry from the University of Pittsburgh, a M.Sc. in life sciences and biotechnology from the University of Bombay, Bombay, India, and a B.Sc., with Honors in life sciences and biochemistry from St. Xavier's College, Bombay, India. She completed a post-doctoral fellowship in the Department of Genetics at Stanford University School of Medicine.

CODA's Vice President, Discovery and Translational Research, Orion Keifer, M.D., Ph.D., is a neuroscientist with neurosurgical training and hands-on expertise in small and large animal models, focused small molecule, and cell and gene therapies for neurological diseases. Before joining CODA, he worked as consultant translational scientist and surgeon for Above and Beyond focusing on precision medicines for neurodegenerative disorders. Orion earned an M.D. and a Ph.D. in neuroscience from Emory University, and a M.S. in brain and cognitive neurosciences and B.S. degrees in biomedical engineering, applied psychology and applied biology with Highest Honors from Georgia Tech. He completed his post-doctoral training in the Department of Neurosurgery at Emory University.

Steve Dodson, Ph.D., is CODA's Vice President, Pharmacology and Early Development.Prior to joining CODA, Steve served as Senior Director, Drug Discovery and Development at Second Genome, Inc. Previously he held positions of increasing responsibility at NeuroTherapeutics Pharma, Inc., and Renovis, Inc., where his work focused on the discovery and development of small molecule therapeutics to treat pain, central nervous system disorders and inflammation. Steve received his Ph.D. in biological sciences from Stanford University and a B.S. in genetics from University of California, Berkeley.

About CODA BiotherapeuticsCODA Biotherapeutics, Inc., is a preclinical-stage biopharmaceutical company developing an innovative gene therapy platform to treat neurological disorders and diseases. The company is creating the ability to control neurons with its revolutionary chemogenetics-based technology. CODA is located in South San Francisco, CA. For more information, please visit http://www.codabiotherapeutics.com.

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http://www.codabiotherapeutics.com

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CODA Biotherapeutics Deepens Gene Therapy Expertise with Industry Veteran, Annahita Keravala, Ph.D., and Key Promotions - PRNewswire

Recommendation and review posted by Bethany Smith

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Newswise Boston, Mass.Beth Israel Deaconess Medical Center (BIDMC) is among a group of leading hospitals, universities, large pharmaceutical companies, small biotech firms and industry partners working together to create a new center for advanced biological innovation and manufacturing. The new centerwill explore and cultivate innovations in cell and gene therapy, advance biologic innovation and manufacturing, and accelerate developments in immunotherapy, cell therapies, gene editing, and other technologies that carry the promise of lasting impact on human health globally. By fostering collaboration and innovation, this center seeks to speed innovation and broaden the universe of patients that can be served by these emerging therapies.

Our mission at Beth Israel Deaconess Medical Center is to provide extraordinary care supported by world-class research and education, said Peter J. Healy, President of BIDMC. We are happy to be a founding member of this innovative consortium, which will allow us to work collaboratively across the diverse health care ecosystem. Together, we will propel the fields of cell therapy, gene therapy and gene editing forward with the shared goal of transforming how we care for patients right here in Boston and around the world.

Harvard University, Massachusetts Institute of Technology (MIT), Fujifilm Diosynth Biotechnologies (FDB), GE Healthcare Life Sciences, Alexandria Real Estate Equities, Inc., will comprise the Board of Directors, while BIDMC joins other contributing members, including Boston Childrens Hospital, Brigham and Womens Hospital, Dana-Farber Cancer Institute, Massachusetts General Hospital, MilliporeSigma and the Commonwealth of Massachusetts.

The overarching mission of the newly established consortium is to catalyze the development of transformative therapies by shortening the path between research and clinical application. The consortium will harness world-leading expertise to propel forward fast-emerging and promising science, the cost and risks of which are daunting for any single institution to tackle alone. By housing institutions with strengths in each link in the chain of innovation within one facility, the partners believe new innovations in both science and manufacturing will speed the introduction of new therapies to patients.

This new innovative consortium and facility will provide unique resources for our investigators to facilitate translation of research findings to clinical applications in cell-based and other novel therapies, said Gyongyi Szabo, MD, PhD, FAASLD, AGAF, FACP, Chief Academic Officer of BIDMC and Beth Israel Lahey Health. Today, more than 30 funded investigators at BIDMC are already engaged in early stage innovation focused on preclinical engineering of viral vectors, mammalian cells and cell manufacturing. BIDMCs membership in this facility will provide an accelerated path for our investigators to bring these preclinical studies to Phase I and Phase II clinical trials.

BIDMC houses a number of state of the art core facilities and laboratories on the leading edge of biotechnology. TheNon-Coding RNA Precision Diagnostics and Therapeutics Core Facilityis apre-clinical facility at BIDMC and HMS dedicated to non-coding RNA, offering informatics, sequencing, imaging and delivery of therapeutics in vitro. BIDMC is also home to theRandi and Brian Schwartz Family Cancer Immunotherapy and Cell Manipulation Facility,a laboratory that serves as a hub forBIDMCs state of the art program in personalized cell therapy for cancer. This facility is a critical resource in whichBIDMC's physician-scientists produce innovative immune-based treatments, including national leadership of apromising experimental therapeutic vaccine against several forms of blood cancer.

The $50 million center will be an independent non-profit organization located in the greater Boston area and will be named in the new year.

About Beth Israel Deaconess Medical Center Beth Israel Deaconess Medical Center is a patient care, teaching and research affiliate of Harvard Medical School and consistently ranks as a national leader among independent hospitals in National Institutes of Health funding.

For more information, visit http://www.bidmc.org.

BIDMC is part of Beth Israel Lahey Health, a new health care system that brings together academic medical centers and teaching hospitals, community and specialty hospitals, more than 4,000 physicians and 35,000 employees in a shared mission to expand access to great care and advance the science and practice of medicine through groundbreaking research and education.

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BIDMC joins local universities, teaching hospitals and industry partners in creating new central facility for regenerative therapies - Newswise

Recommendation and review posted by Bethany Smith

In the United States, the last Thursday of the month of November is designated as Thanksgiving, a feast day focused on giving thanks for all we have. In light of the Thanksgiving season, here are six things about biopharma that we are thankful for.

Innovation

The biopharma industry is built on innovation. Its often shocking to consider just how far the biopharma industry has come in our lifetimes, although sometimes it seems we take it for granted. There are now cures for infectious diseases like Hepatitis C, effective treatments for HIV that have turned it from a nearly-always fatal disease to a chronic condition, and new gene and cell therapies are beginning to hit the market that can cure previously untreatable diseases. For example, Spark Therapeutics Luxturna was approved by the FDA in 2017 to treat a rare, genetic form of blindness and Novartis/AveXis Zolgensma was approved for spinal muscular atrophy (SMA) in May 2019.

Every day marks new discoveries in the industry and in academia that lead to new approaches to new drugs. CRISPR gene editing promises to lead to new ways to treating hundreds of genetic diseases, and immuno-therapy is leading to new treatments and sometimes cures for tough-to-treat cancers and inflammatory diseases.

Global Oversight

A year ago, in November 2018, a Chinese researcher, He Jianku, announced he had utilized CRISPR-Cas9 gene editing to alter the DNA of embryos for seven couples. He used CRISPR to disable a gene called CCR5. This gene creates a protein that allows HIV to enter a cell. All the men of the seven couples had HIV and the women did not. The gene editings goal wasnt to prevent transmission of HIV, according to He, because the seven mens HIV infections were strongly suppressed by standard HIV drugs.

Because the technique was performed on the genomes of the babiesand at this time at least one set of twins has been bornshould those children have children someday, they will pass the change onto their children. This resulted in a frenzy of condemnation, not just by government regulators, but by the scientific community. The arguments are largely that it is way too early and way too dangerous to be conducting this type of research until ethical and legal guidelines have been developed and the risks understood.

This led to leading scientists and several different international bodies calling for a moratorium on heritable genome editing. The scientists included two of the inventors of CRISPR, Feng Zhang and Emmanuelle Charpentier. The moratorium doesnt call for a permanent ban, but stated, Rather, we call for the establishment of an international framework in which nations, while retaining the right to make their own decisions, voluntarily commit to not approve any use of clinical germline editing unless certain conditions are met.

As grateful as we are for innovation, we are also grateful that the scientific community is aware that innovation can be misused and is taking steps to regulate it.

Vaccines

One of our oldest medical advances, vaccines, is one of the most significant. Here are 14 diseases that vaccines either control or have widely eliminated: polio, tetanus, influenza, hepatitis A and B, rubella, Hib, measles, whooping cough, pneumococcal disease, rotavirus, mumps, chickenpox and diphtheria. It has completely eradicated smallpox.

Although the tried-and-true approach to vaccines is to use a dead or attenuated virus, new approaches are expanding the types of diseases vaccines are being developed for, such as cancer and Alzheimers disease. According to the Pharmaceutical Research and Manufacturers of America (PhRMA), there are 264 vaccines in development to prevent and treat diseases. These include infectious diseases (137), cancer (101), allergies (10), autoimmune disease (8) and Alzheimers disease (4).

There are regular breakthroughs in vaccine development. In May, the FDA approved Sanofis Dengvaxia, a vaccine for all four serotypes of dengue. The disease, a hemorrhagic fever, is endemic in the U.S. territories of Puerto Rico and the U.S. Virgin Islands. The vaccine has a controversial history. It was pulled from the Philippine market in 2017 over safety concerns. However, it has been approved in 10 countries in Latin America and Asia where the disease is endemic. It was approved in Europe in December 2018. And in November 2019, the European Commission approved the worlds first Ebola vaccine. The vaccine is manufactured by Merck & Co. and has a trade name of Ervebo.

We are grateful that these deadly diseases are, one by one, being eliminated thanks for vaccines.

Investors

Funding is the life blood of research and development. Where would the pharmaceutical industry be without those who provide infusions of cash to support that research, as well as other business practices. Funding can come from a number of sources, such as venture capital, the stock market and, of course, the time-honored practice of mergers and acquisitions. M&A has played a big role in industry growth and its expected to continue to do so, particularly in the areas of immuno-oncology and gene therapy. This year alone there have been several mega-acquisitions, including Takedas $60 billion acquisition of Shire; Bristol-Myers Squibbs $74 billion deal for Celgene; and AbbVies $63 billion plan to acquire Allergan.

There have been a number of pharma companies that have braved the stock market with initial public offerings this year, including Beam Therapeutics $100 million IPO, a $115 million IPO from SpringWorks Therapeutics, Bridge Biopharmas $240 million IPO and more. For companies that have not gone public, there have been some big financing rounds reported lately. Recently, Cambridge, Mass.-based eGenesis topped the list with $100 million in a Series B financing round, followed closely by a spate of other companies.

Without these financial infusions of cash, many of the medications we take for granted would not be possible.

Talent

While funding may be the life blood of the industry, its pretty clear that the researchers and other employees are the minds behind the innovations. Weekly, BioSpace notes the comings and goings of some of the top executive talent in a Movers & Shakers column. These executive leaders have shaped the pipelines and research arms of companies and, in some cases, helped shaped the future of the industry. Names like Hal Barron, Jos Baselga, William A. Lee, John C. Reed, Michael Severino, George Yancopoulos, Steffan Land and more have been responsible for guiding and shaping the research conducted in the industry. In addition to those executives, there are thousands of lab-level scientists working day in and day out to understand the mechanisms of action of medications and learning how diseases respond to those therapies.

While the vast majority of the researchers in the industry do not see their names in official company statements, a select few do end up receiving special recognition, such as the elite who win Nobel Prizes in medicine. This last year, a trio of scientists won the Nobel for their work in understanding how oxygen levels affect cellular metabolism and physiological function. The work has helped in the development of new medications to fight anemia, cancer and many other diseases. In 2018, the Nobel was awarded to researchers who cracked the code that led the way to the development of checkpoint inhibitors.

For the millions upon millions of people who rely on medications to live their best lives, we are certainly thankful for the people behind the development of these life-saving and life-enhancing treatments.

Justice Served

New treatments and the stories of those patients who benefit from life-saving and altering medications should receive the bulk of the headlines for the industry. Unfortunately, scandal and crime tends to be among the most memorable and oft-reported stories. Names like Martin Shkreli and Elizabeth Holmes are far more well-known than those previously-mentioned Nobel laureates. And their names are largely known due to the levels of infamy they both rose to in the industry. Shkreli, who is currently serving seven years in federal prison for securities fraud, is mostly known for his unflinching defense of a 5,000% increase on the toxoplasmosis drug, Daraprim, his company owned, as well as the odd behavior he displayed on social media platforms. For Shkreli, his crimes are secondary to his level of infamy. Holmes will soon go to trial for her alleged role in defrauding investors in her now-shuttered company, Theranos. She went from lauded entrepreneur to the subject of podcasts, primetime investigations, books, articles and more. While both Shkreli and Holmes gave the industry something of a black eye, we can all be thankful that these bad players are few and far between and their illegal and unethical behavior is are in the minority of industry leaders. We can be thankful that these bad players ultimately must face justice.

See more here:
6 Things About Biopharma to Be Thankful For - BioSpace

Recommendation and review posted by Bethany Smith

BioMarin Pharmaceuticals is seeking marketing approval in Europe for its investigational gene therapy, valoctocogene roxaparvovec, for the treatment of adults with severe hemophilia A.

The company has submitted a marketing authorization application (MAA) to the European Medicines Agency (EMA) for the experimental gene therapy, formerly known as BMN 270. Administered as a single infusion, the therapy uses adeno-associated virus (AAV) vectors to deliver a functional copy of clotting factor VIII, the protein that is missing in people with hemophilia A.

An ongoing Phase 3 trial, GENEr8-1 (NCT03370913), is investigating the treatments safety and efficacy, and is still recruiting adult patients. Go here for more information on trials locations and here for eligibility criteria.

The EMA had previously given valoctocogene roxaparvovec the designation of priority medicines, or PRIME, in 2017. Now, the potential therapy has been granted accelerated assessment, which may potentially shorten its MAA review process from 210 to 150 days.

Accelerated assessment is given by the EMAs Committee for Medicinal Products for Human Use and Committee for Advanced Therapies to innovative medications that are of major interest to public health. This endorsement is meant to speed up the review process of eligible medications, but does not impact the committees decision to recommend their approval.

BioMarins MAA submission was based on updated three-year data from a Phase 1/2 study (NCT02576795)and on an interim analysis of the ongoing Phase 3 GENEr8-1 trial (NCT03370913), which is still recruiting an anticipated 130 patients from 73 sites around the world to test the dose of 6e13 vg/kg (vector genomes per kilogram). Another Phase 3 trial, the GENEr8-2 (NCT03392974) study, is also ongoing and testing a lower dose (4e13 vg/kg).

Three-year data from the Phase 1/2 trial showed that a single administration of valoctocogene roxaparvovec at the higher dose markedly reduced bleeding episodes and the need for factor VIII infusions in a small group of adults with severe hemophilia A. Specifically, there was a 96% reduction in both the mean ABR (annualized bleed rate) and the mean factor VIII usage over the three years.

The levels of clotting factor VIII remained stable over the course of three years following treatment.

Valoctocogene roxaparvovec was generally well-tolerated by patients. None of the participants developed inhibitors to factor VIII, and none withdrew from the study due to adverse events.

We are grateful to the study participants, who have made this progress possible in the span of approximately four years since the first participant was enrolled in the clinical program, Hank Fuchs, MD, president of BioMarins global research and development, said in a press release.

We are very pleased with the level of engagement we have had with global health authorities, as it aligns with our belief that gene therapy represents the next wave of innovation and potentially could be a meaningful advancement for treating people with severe hemophilia A, Fuchs said.

Valoctocogene roxaparvovec will be the first gene therapy for hemophilia whose MAA will be reviewed by health authorities for potential approval in the E.U. BioMarin is expecting the EMA to start reviewing its application in January 2020 and said it will provide an update at that time.

In the meantime, the company is planning to submit a biologics license application for valoctocogene roxaparvovec to the U.S. Food and Drug Administration (FDA) by the end of the year. The investigational treatment has been given a breakthrough therapy designation by the FDA, as well asorphan drug status from both the FDA and EMA.

Joana is currently completing her PhD in Biomedicine and Clinical Research at Universidade de Lisboa. She also holds a BSc in Biology and an MSc in Evolutionary and Developmental Biology from Universidade de Lisboa. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells cells that make up the lining of blood vessels found in the umbilical cord of newborns.

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Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Tcnico (IST-UL). She worked as a molecular biologist research associate at a Cambridge UK-based biotech company that discovers and develops therapeutic, fully human monoclonal antibodies.

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For Hemophilia A, BioMarin Seeks Approval of Its Gene Therapy in Europe - Hemophilia News Today

Recommendation and review posted by Bethany Smith

Can a reformulation of an old drug on the market for asthma, allergy and mastocytosis protect against liver disease, Alzheimers, and amyotrophic lateral sclerosis?

A team from Massachusetts General Hospital is making a case for the latter two. Having recently found an injection of cromolyn sodium effective in inhibiting amyloid beta (A) aggregation in vitro and in mouse models, researchers set out to investigate whether the same compound can achieve the same in ALS.

Their conclusion, published on Natures open access journal Scientific Reports:

Our results indicate that cromolyn sodium treatment significantly delayed the onset of neurological symptoms, and improved deficits in PaGE performance in both male and female mice, however, there was only an effect on survival in female mice.

While the precise etiology of ALS remains poorly understood, one theory proposes that neuroinflammatory processes are implicated in its initiation and progression.

As cromolyn inhibits mast cell degranulation, the scientists at Mass General hypothesized that it could convert immune cells in the brain, including microglia and astrocytes, from a pro-inflammatory to an anti-inflammatory state as well as reducing the levels of cytokines and chemokines.

After comparing the effects of once-daily injections of cromolyn versus a placebo in wild type mice and mice carrying a genetic mutation for ALS, respectively, the researchers came up empty on the microglia and astrocytes theory. But they did find lower levels of pro-inflammatory cytokines/chemokines in the spinal cord and plasma in addition to observing that the transgenic mice treated with cromolyn sodium had the highest motor neuron counts among the four groups.

Our study supports the notion that inflammation has a significant role in the progression of ALS and therefore exploring anti-inflammatory treatments may be of great value for developing an effective treatment, said Ghazaleh Sadri-Vakili, lead author of the study and director of the NeuroEpigenetics Laboratory at Mass General, in a statement.

Sadri-Vakili has previously noted that the neurology department at Mass General has a plan in place to get the drug provided by AZTherapies into the clinic for ALS.

Unlike the formulations of cromolyn currently available through prescription and over the counter, which are absorbed through lung and nasal inhalation or ingested drops, the version injected into mice in the study can be fully available in the bloodstream and cerebrospinal fluid, according to the biotech.

AZTherapies is conducting a Phase III trial of another formulation of cromolyn, combined with oral ibuprofen, to treat early Alzheimers. It is unclear whether any clinical programs are in place to test cromolyn sodium in primary sclerosing cholangitis after Baylor Scott and White Health researchers reported it decreased biliary proliferation and fibrosis in mice.

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Mass General team presents mouse data to back the case for using reformulated asthma drug to treat ALS - Endpoints News

Recommendation and review posted by Bethany Smith

Japans Asahi Kasei Pharma has stepped up with a deal to buy out Copenhagen-based Veloxis Pharmaceuticals for $1.3 billion, bagging an organ rejection drug in the process.

The deal comes 17 years after Veloxis was founded, and the two big shareholders Novo Holdings and Lundbeckfond Invest have both signed off on the buyout. The biotech makes an immunosuppressive drug called Envarsus XR for kidney transplants.

Veloxis has been a low profile biotech on the continent, but recently saw its share price zoom up. Asahi Kasei is paying DKK 6 cash per share, just a 6% premium over the average trading price tracked from October 14 to November 22. But its a 75% premium over the one-year weighted average. The deal was announced on Monday.

Japanese pharmas have been snapping up pharma products around the world for a number of years now, looking to expand beyond Japan as they look for new and growing markets. And Asahi Kasei President Hideki Kobori says they plan to follow up on the acquisition with some new strategic deals.

The acquisition of Veloxis by Asahi Kasei is a great culmination of more than a decade of research, development, and commercialization of life science innovation, which is now benefitting thousands of patients around the world, said Novo Holdings senior partner Christoffer Sderberg in a prepared statement. We are proud of what the employees of Veloxis have accomplished and are pleased that they will all continue with Asahi Kasei.

Excerpt from:
Veloxis wins $1.3B buyout, and the new owner plans to follow up with new deals - Endpoints News

Recommendation and review posted by Bethany Smith

One of the big themes in R&D over the past few years has been the onslaught of spending on developing new oncology drugs. The FDA has encouraged early approvals, opening the door to smaller trials as an onslaught of investment cash made it possible for small players to go the distance.

Big Pharma, meanwhile, has enjoyed the comfort of better scientific insights and the arrival of some huge new PD-1s on the scene. Next up: A tsunami of combo therapies coming at you from the US, Europe and China the new factor in drug hunting.

For the industry, that means a major new source of revenue from coming therapies. For US patients, that means a much better shot at longer survival and possibly even a cure. As well as bankruptcy.

Wait. What?

Researchers just published a new study in The American Journal of Medicine highlighting that 42% of cancer patients exhaust their savings within 2 years of diagnosis. And 62% of the 9.5 million cancer patients they reviewed were in debt after therapy, with 40% to 85% quitting work due to cancer. After 4 years of therapy, 38.2% were insolvent.

You hear a lot every time a new drug is approved about what drug companies are doing to make their therapies accessible, but the simple fact is that in the US large percentages of patients are being crushed by the price of branded drugs. And while the new drugs being introduced may be more important than ever, the unvarnished truth is that basic pricing strategies are more about maximizing revenue than accommodating patients.

The resulting financial toxicity is enormous.

Lets remember that one of the reasons were seeing all the investment cash pouring in is that Wall Street has embraced a big wave of biotech IPOs. And thats where execs are focused when they price new drugs.

Thats not a wild guess, either.

This is the bottom line researchers totted up after discussing pricing strategies on new drugs for multiple sclerosis with 4 biotech execs, published in Neurology this week.

Participants consistently stated that initial price decisions were dictated by the price of existing competitors in the market. Revenue maximization and corporate growth were drivers of price escalations in the absence of continued market penetration. Lower revenue predictions outside the United States also informed pricing strategies. The growing complexity and clout of drug distribution and supply channels were also cited as contributing factors. Although decisions to raise prices were motivated by the need to attract investment for future innovation, recouping drug-specific research and development costs as a justification was not strongly endorsed as having a significant influence on pricing decisions.

So while the industry likes to talk a lot about the pricing levels needed to back innovation, its just not an accurate reflection of reality.

In just a few weeks, were going to wake up to a new year that will be dominated by drug pricing discussions. Were going to be doing some of this ourselves at JP Morgan.

The industry still has a shot at coming up with some kind of workable reform on drug prices. Barring a market solution, though, you can expect plenty of unworkable and destructive suggestions on drug importation and compulsory licensing and more. And someday, lawmakers will do something about it.

Continued here:
There's one endpoint that the booming biopharma industry has failed at miserably: financial toxicity - Endpoints News

Recommendation and review posted by Bethany Smith

After quietly plowing the arid cancer vaccine field for a decade, a low profile Swiss biotech is throwing itself in the ring as a player to watch in the frenetic race to extend the benefits of immunotherapies to solid tumors.

Co-founded by biotech vet Bernard Mach who was involved in the creation of Biogen and Novimmune and his son Nicolas, MaxiVAX is laser-focused on developing a two-part cancer vaccine that activates the immune system to attack cancer. And it has been awarded a 2.785 million ($3.07 million) grant from the European Commission on top of CHF 5 million ($5.01 million) from a Series B2 round to push through a Phase II study in head and neck cancer in Switzerland and kick off a US study for an undisclosed rare tumor.

Much has been made of cancer vaccines potential to complement immune checkpoint inhibitors, which can be incredibly effective in subsets of patients, sometimes as few as 20%. But the first generation of cancer vaccines, largely made of peptides derived from cancer cells, has failed to induce the immune effects needed to make a difference on their own.

A key issue, MaxiVAX CEO Dimitrios Goundis said, is that peptides carry mutations that vary from patient to patient. Their solution: Isolate the whole tumor cells, containing the complete antigenic repertoire for the immune cells to profile and target, inactivate them, then implant them subcutaneously next to an adjuvant meant to sustain the immune response.

So our therapy basically is a vaccine, which is patient specific, and an immune boosting agent (GM-CSF) which is delivered by encapsulated genetically modified cells, which allow continued delivery of GM-CSF over several days at the site of vaccination, he told Endpoints News. And with that we address we think all the weaknesses that weve identified over the course of the last 20, 30 years when people start to look at vaccines against cancers in a more systematic way.

GM-CSF, or granulocyte-macrophage colony-stimulating factor, is a naturally occurring immune modulator that has a relatively short half life, normally disappearing within a couple hours in the bloodstream. To keep it flowing, MaxiVAX is inserting a gene into hundreds of thousands of cells to produce a steady supply of GM-CSF such as immune cells that can stay energized as they get familiarized with irradiated tumor cells.

The improvement should be clear cut, he said. While checkpoints have extended median survival in advanced and refractory head and neck cancer from three to six months, that still means half of the patients dont live that long. For the ongoing Phase II, for which they are recruiting around 40 patients across six sites, MaxiVAX will be monitoring the number of patients who pass the 6-month survival threshold as the main efficacy measure.

Its a hard endpoint, he said. So were not only looking at whether or not the tumor is reduced in size or disappears, but also what is the effect of that in prolonging the life of the patients.

He expects the final results from the open-label trial to be in around 2021. Before that, MaxiVAXs small team of 10 will also initiate a Phase II for which they have already obtained an IND in the US.

The new funds from private investors MaxiVAX has managed to stay away from venture capitalists in raising over $15 million so far will also go toward building out manufacturing.

Right now the biotech relies on the Geneva Hospital, where Nicolas Mach is head of oncology, to isolate the tumor cells and to generate and fill the capsules with GM-CSF producing cells. Its offices are located in the neighborhood, which also houses AMAL Therapeutics, another next-gen cancer vaccine player recently bought out by Boehringer Ingelheim.

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Another cancer vaccine play out of Geneva shoots for 'hard endpoint' in PhII head and neck cancer test - Endpoints News

Recommendation and review posted by Bethany Smith

At an old Amgen facility tucked just beyond the Rockies. In a warehouse behind a Walmart supercenter in Durham, North Carolina. On a long-time Bristol Myers Squibb site outside Princeton. The tech has emerged, and now the arms race to physically build a generation of gene therapies has begun.

Novartis will spend $500 million scaling its gene therapy manufacturing efforts, Reuters reported today. Thatll put it nearly on par with Pfizer, who committed $600 million for its facilities even before any of its gene therapies have been approved. Together, 11 companies Reuters surveyed will spend $2 billion on gene therapy production.

Additionally, the Boston Globereported today that Vertex had completed its search for a gene therapy research and manufacturing campus in Boston, settling on a 256,000 square-foot center at the Raymond Flynn Marine Industrial Park.

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Make that 2 new sickle cell disease blockbuster hopefuls OK'd by the FDA in just a few days - Endpoints News

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Cancer could be defined as uncontrolled cell growth in the body leading to organ malfunction. If untreated, it can lead to death. Uncontrolled growth of cell is managed by the body in several ways, one of them is by deploying white blood cells to detect and eradicate these cancerous cells. It has been discovered that the immune system could be manipulated to influence cancerous cells to destroy itself.

Access Report Details at: https://www.themarketreports.com/report/global-cancer-gene-therapy-market-by-manufacturers-countries-type-and-application-forecast

Market share of global Cancer Gene Therapy industry is dominate by companies like Adaptimmune, Bluebird bio, Celgene, Shanghai Sunway Biotech, Shenzhen SiBiono GeneTech, SynerGene Therapeutics, Altor BioScience, Amgen, Argenx, BioCancell, GlaxoSmithKline, Merck, OncoGenex Pharmaceuticals, Transgene and others which are profiled in this report as well in terms of Sales, Price, Revenue, Gross Margin and Market Share (2017-2018).

With the help of 15 chapters spread over 100 pages this report describe Cancer Gene Therapy Introduction, product scope, market overview, market opportunities, market risk, and market driving force. Later it provide top manufacturers sales, revenue, and price of Cancer Gene Therapy, in 2017 and 2018 followed by regional and country wise analysis of sales, revenue and market share. Added to above, the important forecasting information by regions, type and application, with sales and revenue from 2019 to 2024 is provided in this research report. At last information about Cancer Gene Therapy sales channel, distributors, traders, dealers, and research findings completes the global Cancer Gene Therapy market research report.

Market Segment by Regions, regional analysis covers:

Market Segment by Type, covers:

Market Segment by Applications, can be divided into

Purchase this premium research report at: https://www.themarketreports.com/report/buy-now/1498456

Table of Contents

1 Market Overview

2 Manufacturers Profiles

3 Global Cancer Gene Therapy Market Competitions, by Manufacturer

4 Global Cancer Gene Therapy Market Analysis by Regions

5 North America Cancer Gene Therapy by Countries

6 Europe Cancer Gene Therapy by Countries

7 Asia-Pacific Cancer Gene Therapy by Countries

8 South America Cancer Gene Therapy by Countries

9 Middle East and Africa Cancer Gene Therapy by Countries

10 Global Cancer Gene Therapy Market Segment by Type

11 Global Cancer Gene Therapy Market Segment by Application

12 Cancer Gene Therapy Market Forecast (2019-2024)

13 Sales Channel, Distributors, Traders and Dealers

14 Research Findings and Conclusion

15 Appendix

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Global Cancer Gene Therapy Market Report with Study of North America, Europe and Asia-Pacific, South America, Middle East and Africa Regions -...

Recommendation and review posted by Bethany Smith

The study of Gene Therapy Market is useful in providing answers to several critical questions that are important for the industry stakeholders such as collaboration solution and service vendors, distributors and partners, end users, etc., besides allowing them in strategizing investments and capitalizing on market opportunities. The report also constructed elementary and necessary Gene Therapy inspection movement, stating through basic analysis by driving wide gatherings with experts holding Gene Therapy key positions inside the business, for example, Presidents, VIPs, executives, and directors. The analysts authoring the report provide a meticulous evaluation of all of the segments included in the report.

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The report involves perceptive data on the main sectors of the global Gene Therapy market, paired with the sub-segments. The aforementioned factors are boosting its adoption and propelling the growth of the global Gene Therapy market. This report studies the global Gene Therapy Market status and forecast, categorizes the global Gene Therapy Market size (value & volume), revenue (Million USD), product price by manufacturers, type, application, and region.

Segmentation Overview:

By Gene Therapy Type (Germline Gene Therapy and Somatic Gene Therapy)

By Type of Vector (Viral Vector and Non-viral Vector)

By Disease Indication (Cardio Vascular Diseases, Cancer, Genetic Disorders, Neuro Disorders, Infectious Diseases, and Others)

By Region (North America, Europe, Asia Pacific, Latin America, Middle East, and Africa)

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GlaxoSmithKline plc, Bluebird Bio, Inc., Adaptimmune Therapeutics plc, Celgene Corporation, Shanghai Sunway Biotech Co. Ltd., Merck KGaA, Transgene SA, and OncoGenex Pharmaceuticals, Inc.

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All other institutes like Government bodies, private firms, ventures will get benefit from Gene Therapy market report A key trend that is expected to predominantly influence the Gene Therapy market in a coming year is an evolution of new applications in future. The Gene Therapy research and development teams in various companies are working towards the further technical development of Gene Therapy in order to cater the next-generation industries.

The Gene Therapy market have widespread application in multiple industries. However, professionals across the globe are intensively working towards the development of Gene Therapy technologies. This will be even more flexible and reliable, along with being cost-effective Gene Therapy industry. This trend is anticipated to drive the demand for Gene Therapy in various other novel industries. Furthermore, Gene Therapy readers will get a clear perspective on the most affecting driving and restraining forces in the Gene Therapy market and its impact on the global market. The report predicts the future outlook for Gene Therapy market that will help the readers in making appropriate decisions on which Gene Therapy market segments to focus in the upcoming years accordingly. In a word, the Gene Therapy report offers a whole consequential study of the parent Gene Therapy market, key tactics followed by leading Gene Therapy industry Players and upcoming segments. Likewise, the former and current Gene Therapy industry forecast analysis in terms of volume and value along with research conclusions is a decisive part of Gene Therapy study. So that Gene Therapy report helps the new aspirants to inspect the forthcoming opportunities in the Gene Therapy market.

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How will Gene Therapy continue to evolve in the next 10 years? - Montana Ledger

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With the help of 15 chapters spread over 100 pages this report describe Gene Therapy for Age-related Macular Degeneration Introduction, product scope, market overview, market opportunities, market risk, and market driving force. Later it provide top manufacturers sales, revenue, and price of Gene Therapy for Age-related Macular Degeneration, in 2017 and 2018 followed by regional and country wise analysis of sales, revenue and market share. Added to above, the important forecasting information by regions, type and application, with sales and revenue from 2018 to 2024 is provided in this research report. At last information about Gene Therapy for Age-related Macular Degeneration sales channel, distributors, traders, dealers, and research findings completes the global Gene Therapy for Age-related Macular Degeneration market research report.

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Market share of global Gene Therapy for Age-related Macular Degeneration industry is dominate by companies like Retrosense Therapeutics, Regenxbio, Agtc and others which are profiled in this report as well in terms of Sales, Price, Revenue, Gross Margin and Market Share (2018-2019).

Market Segment by Regions, regional analysis covers:

Market Segment by Type, covers:

Market Segment by Applications, can be divided into

Purchase this premium research report at: https://www.themarketreports.com/report/buy-now/1497384

Table of Contents

1 Market Overview

2 Manufacturers Profiles

3 Global Gene Therapy for Age-related Macular Degeneration Market Competitions, by Manufacturer

4 Global Gene Therapy for Age-related Macular Degeneration Market Analysis by Regions

5 North America Gene Therapy for Age-related Macular Degeneration by Countries

6 Europe Gene Therapy for Age-related Macular Degeneration by Countries

7 Asia-Pacific Gene Therapy for Age-related Macular Degeneration by Countries

8 South America Gene Therapy for Age-related Macular Degeneration by Countries

9 Middle East and Africa Gene Therapy for Age-related Macular Degeneration by Countries

10 Global Gene Therapy for Age-related Macular Degeneration Market Segment by Type

11 Global Gene Therapy for Age-related Macular Degeneration Market Segment by Application

12 Gene Therapy for Age-related Macular Degeneration Market Forecast (2019-2024)

13 Sales Channel, Distributors, Traders and Dealers

14 Research Findings and Conclusion

15 Appendix

Ask your report related queries at: https://www.themarketreports.com/report/ask-your-query/1497384

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Global Gene Therapy for Age-related Macular Degeneration Market Forecast (2019-2024) Report: By Regions, Type and Application with Sales and Revenue...

Recommendation and review posted by Bethany Smith

No longer just for professional athletes, these are the stem cell and regenerative medicine options DCs need to know about

The health care landscape continues to evolve at a dizzying pace. Insurance deductibles are increasing, and this has placed a financial burden on patients who are required to self-pay for necessary and yet uncovered services.

The opioid crisis has left physicians with limited clinical options to treat chronic pain and dysfunction. At the same time, pressure has been placed on health care providers to provide affordable alternatives to invasive procedures that provide limited clinical options with high failure rates. This confluence of supply and demand has resulted in the growth of emerging therapies in the field of stem cell and regenerative medicine. These therapies are bringing hope to patients and new opportunities to health care providers who deliver them.

Regenerative medicine is the process of replacing or regenerating cells and tissues to restore normal function. Initially popularized by professional athletes, these therapies have become mainstream. More than 27 million Americans suffer from osteoarthritis today, and in 2030 25% of U.S. adults will be diagnosed with osteoarthritis. The global regenerative medicine market is predicted to reach more than $100 billion by 2022.

These moderately-invasive regenerative procedures are eclipsing traditional highly-invasive procedures, such as hip and knee implantation, which will have a global market of $35 million over the same period.

There are four primary regenerative medicine options:

Irritant therapies include prolotherapy, ozone and prolozone. Theyincludeadding multipleirritatingsubstances along with numbing agents into degenerated or injured joints, and areas of pain.

These therapies cause inflammation to kick-start regeneration by stimulating the body to send in macrophages, which are cells that ingest and destroy theirritantsolution and trigger the healing response. Irritant therapies are an excellenttreatmentfor all forms of musculoskeletal and joint pain includingchronic neck and back pain, and rotator cuff injuries.

The effect of irritant therapies is analogous to jump-starting the battery in a tractor to get the engine to turn over.

Protease inhibition therapy eliminates the factors causing cartilage degradation, tissue breakdown, inflammation and pain. It cleans and protects joints. It is most commonly used for patients with osteoarthritis (OA) and degenerative disc disease (DDD).

It includes therapies such as alpha-2-macroglobulin (A2M) and interleukin-1 receptor antagonist protein (IRAP). A2M and IRAP are proteins found naturally in our blood. They act as protease inhibitors by binding to and inactivating damaging proteases in the body. Proteases are catabolic enzymes that break larger molecules into smaller units. Proteases trapped in the joints catabolize cartilage and break it down, causing arthritis. A2M is a large protein made in the liver. It blocks activity for all known molecules that cause cartilage breakdown. It works like a Venus flytrap by having a bait-and-trap mechanism on two sides.

Once the proteases are bound on both sides, the molecule initiates a suicide cascade and dies, allowing it to be flushed out of the area by the body.

The binding effect of protease inhibition therapy is analogous to de-weeding a garden and tilling the soil before planting.

A fibronectin-aggrecan complex test (FACT) may be used to determine the presence of FAC, which is a biomarker or indicator of cartilage breakdown caused by proteases. FAC is a unique molecular complex that is specific for painful inflammation of the spine and cartilage.

A small sample of fluid is taken from the joint or disc and sent to a lab for testing. The test looks for the presence of FAC in the fluid sample and determines where you are: FAC+ or FAC-. FAC+ patients are identified as ideal candidates for A2M injections and have a 90% rate of responding to the A2M therapy.

Stem cell therapy is focused on concentrating the workhorses of regeneration and restoration of tissues: stem cells. This results in greater cell signaling and cell recruitment than other regenerative therapies. Stem cells are known as mesenchymal signaling cells. They are considered pluripotent, which means they are undifferentiated and can replicate into various cell and tissue types.

Stem cells are found in bone marrow, the soft spongy tissue found at the center of large bones. Introducing stem cells into an injured area initiates the healing response, repairing damaged tissue by growing new, healthy tissue. The most common stem cell therapies include bone marrow aspirate concentrate (BMA), nanofat and stromal vascular fraction.

Injecting stem cells into an injured area is analogous to planting seeds in a garden.

Growth factor therapies are focused on cell signaling and cell recruitment. Blood is made up of white blood cells, red blood cells, and platelets that are suspended in plasma. Platelets are most widely known for their ability to clot blood. Platelets are also highly rich in growth factors that are proteins that stimulate healing. When an injury occurs, platelets become activated, migrate to the site of injury and release growth factors.

Growth factor therapies are the most popular provider choice for the low-cost regeneration of tissues and include platelet-rich plasma (PRP) and platelet-rich fibrin matrix (PRFM). The therapy includes drawing the patients blood followed by centrifugation to concentrate the platelets and exclude other unwanted blood products.

Another type of growth factor therapy is amniotic fluid growth factor (GF) injection therapy. Amniotic fluid surrounds the fetus during pregnancy and provides protection and nourishment. Human amniotic fluid is sourced from consenting mothers during full-term C-sections. It contains over 200 growth factors, cytokines and proteins. The therapeutic use of amniotic fluid is regulated by the FDA. It must be tested for disease and may not include any viable cells. Amniotic fluid GF therapy has both anti-inflammatory and anti-microbial properties and includes naturally-occurring hyaluronic acid for lubrication. It is most commonly used to promote the repair and reconstruction of soft tissues including cartilage and tendons.

Exosomes are being heralded as the next frontier of growth factor therapies. While they are not cells, exosomes play a vital role in the communication and rejuvenation of all the cells in the body. Exosomes are extracellular vesicles, or small bubbles, released from cells, especially from stem cells. These culture-expanded cell secretions are derived from human placental tissue. They allow for cell-to-cell communication, transporting molecules that are important regulators of intracellular information. Exosomes act as a food source for stem cells and prolong their activity. Exosomes are anti-inflammatory and include more than 300 growth factors, cytokines and proteins.

Patients with Lyme disease, burns, chronic inflammation, autoimmune disease and other chronic degenerative diseases may benefit from including exosomes in their treatment regimen. The application of growth factor therapies is analogous to applying fertilizer to a garden to help the crop grow and flourish.

Moving stem cell and regenerative medicine forward in the treatment algorithm may eliminate the need for other ineffective or potentially harmful therapies. These therapies provide new hope for patients whose only alternatives have been long-term medication, steroid injections, and costly and time-consuming surgery and rehab.

Stem cell and regenerative medicine therapies may only be provided by licensed medical professionals following all appropriate rules and regulations. An understanding of these emerging therapies and the benefits they may provide is essential as the collaboration between doctors of medicine and chiropractic increases and we join forces to combat chronic pain, dysfunction and disease.

MARK SANNA, DC, ACRB LEVEL II, FICC, is a member of the Chiropractic Summit and a board member of the Foundation for Chiropractic Progress. He is the president and CEO of BreakthroughCoaching, and can be reached at mybreakthrough.com or800-723-8423.

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A primer: stem cell and regenerative medicine as 'the' emerging therapy - Chiropractic Economics

Recommendation and review posted by Bethany Smith

OBJECTIVES:

Stem cell therapy is a promising approach in the treatment of acutemyocardial infarction(AMI). Mesenchymal stem cells (MSC) from bone marrow (BM-MSC) and adipose tissue (AT-MSC) are attractive and feasible for preclinical and clinical trials. In this study, we compared the therapeutic potential of BM-MSC and AT-MSC in repairing the hearts of rats with isoproterenol (ISO)-induced AMI.

Forty-two female rats were assigned into two groups; the optimization and the experimental group. The optimization groups were further subdivided into control group and the AMI induced group (using ISO). The experimental group was subdivided into AMI+cell-free media injected in the tail vein, AMI+BM-MSC, and AMI+AT-MSC groups treated with the intravenous injection of their respective cell types. Twenty-eight days after induction, electrocardiogram (ECG) was performed, and heart tissue samples were collected for histological assessment and cells tracing.

MSC therapy repaired cardiac functions shown by the restoration of ST segment, QT and QRS intervals in the ECG when compared to the AMI group. Infarct area was significantly decreased, and cardiac tissue regeneration signs were shown on histopathological examination.

Both MSC sources proved to be equally efficient in the assessed parameters.

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Comparative Study of the Therapeutic Potential of Mesenchymal Stem Cells Derived from Adipose Tissue and Bone Marrow on Acute Myocardial Infarction...

Recommendation and review posted by Bethany Smith

Medically reviewed by Richard M. Stone, MD

More than 60,000 new cases ofadult leukemiaare diagnosed in the U.S. each year. Although it is one of the more common childhood cancers,leukemia occurs more often in older adults.

Leukemia is a cancer of the bodys blood-forming tissues that results in large numbers of abnormal or immature white blood cells. The main types of leukemia are:

AML causes the bone marrow to produce immature white blood cells (called myeloblasts). As a result, patients may have a very high or lowwhite blood cellcount, and lowred blood cellsandplatelets.

CLL is the second most common type of leukemia in adults. It is a type of cancer in which the bone marrow makes too many maturelymphocytes(a type of white blood cell).

ALL is a type of leukemia in which the bone marrow makes too many immaturelymphocytes. Similar to AML, the white blood cells can be high or low and oftentimes the platelets and red blood cells are low. This form of leukemia is more common in children than adults.

CML is usually a slowly progressing disease in which too many mature white blood cells are made in the bone marrow.

People with leukemia may experience:

Because these symptoms can be caused by a variety of other conditions, its important to check with your doctor if they arise.

While studies have shown men to be more atrisk than women, some other risk factors include:

While test procedures vary based on the type of leukemia, the two most common procedures are thecomplete blood count(CBC) test and the bone marrow aspiration biopsy.

CBC is a procedure used to check the redblood cell and platelet counts as well as the number and type of white bloodcells (the red cells carry oxygen, the white cells fight and prevent infection,and platelets control bleeding). A bone marrow aspiration biopsy involvesremoving a sample of bone marrow, including a small piece of bone by insertinga needle into the hipbone. The sample is then examined for abnormal cells.

Treatment for leukemia varies depending on the type and specific diagnosis.

The treatment for acute leukemias may be lengthy up to two years in ALL and is usually done in phases. The first phase, known as remission induction therapy, involves administering several chemotherapy drugs over a several-week period. The goal is to destroy as many cancer cells as possible to achieve a remission (in which cancer cells are undetectable, but small amounts are still present).

The second phase, known aspost-remission or consolidation therapy, seeks to kill leukemia cells thatremain after remission induction therapy. This phase may involve chemotherapyand/or a stem cell transplant.

Additional treatments may also be necessary. ALL patients, for example, may receive special treatment to prevent the disease from recurring in the spinal cord or brain.

The treatment for CML has been revolutionized by the advent of the oral medication imatinib and the second- and third-generation drugs known as tyrosine kinase inhibitors (TKIs). These are oral medications that work to inhibit the function of theBCR-ABLprotein. Many patients take these medications for the rest of their lives. In rare instances, a patient may require a stem cell transplant.

Some patients with CLL are recommended formonitoring and observation. Others,usually those with symptoms or low red cell or platelet counts, requiretreatment. Such treatment may involve intravenous chemotherapy, but often withoral therapy with pills that inhibit the function of a key protein, Brutonstyrosine kinase.

Treatments for leukemia can include:

Drugs that harness the immune system in fighting leukemia have shown considerable promise. Some monoclonal antibodies synthetic versions of immune system proteins are already in use to treat certain forms of leukemia and others are being studies in clinical trials.

Another form of immunotherapy, immune checkpoint inhibitors, which release a pent-up immune system attack on tumor cells, is being tested in several forms of leukemia. Cancer vaccines, which boost the immune systems ability to fight cancer, are being studied for use in leukemia.

CAR T-cell therapy, which uses modified immune system T cells to better target and kill tumor cells, has achieved impressive results in trials involving children and adults up to age 25 with relapsed ALL.

Research into new treatments for adult leukemia is moving along several tracks in addition to immunotherapy.

By tracking the specific abnormal genes within leukemia cells, physicians are increasingly able to tailor treatment to the unique characteristics of the disease in each patient. Targeted drugs such as imatinib and dasatinib, for example, are now used in treating patients with ALL whose leukemia cells have an abnormality known as the Philadelphia chromosome. Targeted agents including IDH or FLT3 inhibitors, which zero in on proteins made from mutated genes, have been approved to treat some patients with AML, while other such inhibitors are being tested in clinical trials.

New tests make it possible to detect ever smaller amounts of leukemia that remain after treatment. Investigators are exploring how these minute levels may influence a patients prognosis and how they might impact treatment.

Researchers are testing whether treatment periods for certain drugs can be safely reduced in some patients. For instance, studies are under way to determine if drugs such as imatinib, which are currently taken for life, can be safely stopped in some patients with CML. Researchers hope to test whether treating patients with CLL with the drug ibrutinib plus other medicine for a fixed amount of time is safe and effective.

Patients may consider treatment through a clinical trial.Dana-Farber currently has more than 30 clinical trials for adult leukemia. A national list of clinical trials is available atclinicaltrials.gov.

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Adult Leukemia: What You Need to Know - Dana-Farber Cancer Institute

Recommendation and review posted by Bethany Smith