Monthly Archives: December 2020

If it’s to be a merry festive season, consider the child you might conceive – Sydney Morning Herald

Posted: December 12, 2020 at 6:58 pm

Festive season cheer lets face it, alcohol can lead to the conception of children. It can also damage not only one or two but three generations of your offspring. The revolutionary science of epigenetics informs us that men and women, just like mice, rats, fish, chickens, guinea pigs, dogs, ferrets, pigs and non-human primates, are susceptible to the brain damage known as fetal alcohol spectrum disorder (FASD).

Many couples do not know when they have conceived. If they've been drinking, the risks for the fetus are high. Credit:Greg Newington

Professor Kelly Huffman, of the University of California, is among a number of scientists to establish that exposure to alcohol in utero can not only severely harm the brain and body size of the first baby, but through the epigenetic effect, intellectual impairment, anxiety, depression and motor skill problems can be passed on to grandchildren and great grandchildren.

Over the past 20 years in my post-television work in health and education, I have met large numbers of FASD children, their families and carers. I became an ambassador for the organisation NOFASD because I learnt that most Australians about to have a child have little knowledge of FASD

According to one of Australias leading paediatric specialists on FASD, Heidi Webster of the Sunshine Coast Hospital and Health Service, just two binge-drinking sessions anytime during the first trimester is enough to cause FASD.

Visit link:
If it's to be a merry festive season, consider the child you might conceive - Sydney Morning Herald

Posted in Epigenetics | Comments Off on If it’s to be a merry festive season, consider the child you might conceive – Sydney Morning Herald

Embryologist Kim Tremblay Will Explore the Secrets of Liver Regeneration – UMass News and Media Relations

Posted: December 12, 2020 at 6:58 pm

Scientists have known since ancient times, as notable in Greek mythology, that liver tissue has a remarkable ability to regenerate, but embryologist Kim Tremblay, veterinary and animal sciences, says, We still dont know how it does that, its still a mystery even after two thousand years.

She now has a five-year, $1.4 million grant from the NIHs National Institute of Diabetes and Digestive and Kidney Diseases to investigate how the organ can replace itself in an amazingly short time. Unlike any of our other organs, if you cut out two-thirds of your liver, it will grow back in seven days, she says. Whats amazing about that time is that in many organisms that have a liver, it can grow back in seven days.

The liver filters impurities, toxins, alcohol and drugs, for example, from the bloodstream, which kills many cells, so in a sense its not surprising that the organ can quickly respond to injury by making new ones, Tremblay says. There is a reservoir of cells that replace the dead ones. The liver has a huge number of regenerative or homeostatic cues that trigger this, but we have not yet established which cells are responsible for this.

As a developmental biologist, Tremblay will focus on how hepatic cell types emerge during embryonic development. Using this strategy, she hopes to address long-standing disagreement in the field regarding the cells that are involved in regeneration. Some scientists think its one type, others insist it is another, she says, but the problem is that they are often using different regeneration models. What if adult cells are able to respond to distinct regenerative cues in unique ways because of where or how they arose in the embryo?

In the embryonic liver bud, she explains, embryonic precursor cells called hepatoblasts differentiate into two distinct adult cell types hepatocytes and cholangiocytes. Researchers have varying views about which of these might hold the regeneration key. We do know they arise in different developmental pathways in the embryo and they look different in an adult, Tremblay adds.

Her previous experiments in a culture system she developed show that hepatoblasts, the hepatic precursor cells, respond differentlyto cues growth factors and other signals in the embryo. Tremblay believes that a new series of investigations in a mouse liver model using targeted genetic techniques will show that both cell types harbor more than one as-yet-undiscovered variant.

I think we havent yet discovered the differences within those groups, the embryologist says. The grant is designed to explore this heterogeneity in development. Well use RNA transcript analysis from knock-out mice to identify the roles of particular genes, and well also explore ATAC-Seq. ATAC-Seq is a sensitive technique used to look for epigenetic memory, which is where Tremblay hypothesizes that her lab will find the key to how the different cells arise.

So far, the work in our lab has focused on the liver bud around 9.5 days of development, Tremblay says. In the future, we want to study the process later in development but still in the embryo.

By 10.5 days the liver is composed of hepatoblasts in lobes, and a slightly later cells start going down the path toward becoming hepatocytes or cholangiocytes. Thats when well look at the different RNA transcripts, but I think were going to find that the answer has to do more with epigenetics and the different environmental signals the precursors are exposed to. Because developmentally acquired epigenetic traits can be maintained through adulthood, it could provide a mechanism that would explain why some adult liver respond to certain injuries better than others.

Originally posted here:
Embryologist Kim Tremblay Will Explore the Secrets of Liver Regeneration - UMass News and Media Relations

Posted in Epigenetics | Comments Off on Embryologist Kim Tremblay Will Explore the Secrets of Liver Regeneration – UMass News and Media Relations

Epigenetic modifications are the cause of memory loss in Alzheimers disease – News Landed

Posted: December 12, 2020 at 6:58 pm

Dementia is the common sequelae of age-related Alzheimers disease. This would probably occur due to epigenetic failure. As we know, Alzheimers disease is a progressive disorder that causes brain cells to degenerate. It further disrupts cognitive thinking and impairs the behavioral skills of humans.

The molecular underpinning showed that the condition changes the gene expression in the prefrontal cortex. This is the place in the brain associated with social and cognitive thinking. Researchers tried to reverse the condition by inhibiting the harmful gene transcription. They have conducted studies on mouse models and found that the drugs made to inhibit such activity, would help to restore normal gene function. Also, this will favor the neuronal function at ages.

- Advertisement -

It is a covalent post-translational modification to histone proteins, that is essential for regulating gene expression. It includes methylation, acetylation, phosphorylation, ubiquitylation, and sumoylation. Histone proteins act to package DNA which wraps around the eight histones, into the chromosome. Above all, the nature of packaging determines whether the genes are either upregulated or suppressed.

Scientists identified H3K4me3, a histone modification, elevated in the prefrontal lobe of AD. Precisely, we can say that modification as histone trimethylation at the amino acid lysine 4. This epigenetic change increases the gene transcription, which leads to the production of more histone-modifying enzymes. And so, while targeting those unnecessary enzymes by the selective drugs, we can possibly have an apt treatment for ADs.

Besides, the researchers found some gene targets, which would get affected by epigenetics. One among is Sgk1, where its level is higher in AD patients. Also, it helps in the opening of ion channels and mediates the action of other hormone releases. Interestingly, the methylation of Sgk1 increases the chance of cellular death in other disease manifestations like Parkinsons, amyotrophic lateral sclerosis. In conclusion, we can administer a specific Sgk1 inhibitor to decrease the level of tau protein, and also treat the AD patients.

Do you want to publish on Apple News, Google News, and more?Join our writing community,improve your writing skills, and be read by hundreds of thousands around the world!

- Advertisement -

Source: MedicalXpress

+ Industry Titan Christopher Nolan Balks at Warner Bros. Same-Day Theaters and Streaming Movie Release Schedule+ Archaeologists How do they know where to dig?

+ A new appthe iGenomicsmakes pocket size DNA analyzer possible+ The first wave of vaccination to begin in the UK from today

Read more here:
Epigenetic modifications are the cause of memory loss in Alzheimers disease - News Landed

Posted in Epigenetics | Comments Off on Epigenetic modifications are the cause of memory loss in Alzheimers disease – News Landed

Healthy lifestyle in obese mothers can improve the baby’s health – Diabetes.co.uk

Posted: December 12, 2020 at 6:58 pm

Obese pregnant women can reduce health-related risks for their unborn child by improving their diet and exercising, researchers have said.

Previous research trials have found high glucose levels in the pregnant mother can trigger changes in the fetus, therefore making the child vulnerable to health conditions in later life.

The most recent study involved more than 550 obese pregnant women. Half of them were asked to improve their diet and start exercising and the other half made no changes to their lifestyle at all.

The researchers looked at DNA patterns among children who were born to mothers who developed gestational diabetes and whether a dietary and physical activity intervention had altered outcomes.

The findings suggested that making lifestyle changes in pregnant did reduce DNA changes in the child which are usually associated with gestational diabetes in the pregnant mother.

Karen Lillycrop, Professor of Epigenetics at the University of Southampton said: These findings suggest that improvements to diet and physical activity can have an impact on the development of their children.

These are very encouraging findings and further studies are now needed to establish whether reducing these epigenetic changes through a healthier lifestyle during pregnancy are accompanied by improved health outcomes for the children in later life.

Professor Lucilla Poston, Tommys Chair of Maternal & Fetal Health and lead investigator of the UPBEAT trial at Kings College London, said: We have known for some time that children of mothers who had gestational diabetes are a greater risk of obesity and poor control of glucose; this new research implies that epigenetic pathways could be involved.

Tommys chief executive Jane Brewin said: Obesity during pregnancy can have lifelong negative impacts on mother and baby so one of the best things mums can do is to improve their health, including their weight, before embarking on a pregnancy.

However, this study shows that mums who are overweight and their babies can still benefit from adopting a healthy diet while pregnant. All mums-to-be need access to healthy eating advice, and those who are overweight should be given non-judgemental practical support and encouragement to eat healthily during pregnancy.

Continue reading here:
Healthy lifestyle in obese mothers can improve the baby's health - Diabetes.co.uk

Posted in Epigenetics | Comments Off on Healthy lifestyle in obese mothers can improve the baby’s health – Diabetes.co.uk

Flavours added to vaping devices can damage the heart: Study – Sify News

Posted: December 12, 2020 at 6:56 pm

New York, Dec 12 (IANS) Researchers have revealed the appealing array of fruit and candy flavours that entice millions of young people to take up vaping are cardiotoxic and disrupt the heart's normal electrical activity.

Mounting studies indicate that the nicotine and other chemicals delivered by vaping, while generally less toxic than conventional cigarettes, can damage the lungs and heart.

"But so far there has been no clear understanding about what happens when the vaporized flavouring molecules in flavoured vaping products, after being inhaled, enter the bloodstream and reach the heart," said study author Sami Noujaim from the University of South Florida in the US.

In the study, published in the American Journal of Physiology-Heart and Circulatory Physiology, the research team reported on a series of experiments assessing the toxicity of vape flavourings in cardiac cells and in young mice.

The flavoured electronic nicotine delivery systems widely popular among teens and young adults are not harm-free.

"Altogether, our findings in the cells and mice indicate that vaping does interfere with the normal functioning of the heart and can potentially lead to cardiac rhythm disturbances," Noujaim said.

In mouse cardiac muscle cells (HL-1 cells), the researchers tested the toxicity of three different popular flavours of e-liquid: fruit flavour, cinnamon, and vanilla custard.

All three were toxic to HL-1 cells exposed to e-vapour bubbled into the laboratory dish where the cells were cultured.

Cardiac cells derived from human pluripotent stem cells were exposed to three distinct e-vapours.

The first e-vapour containing the only solvent interfered with the electrical activity and beating rate of cardiac cells in the dish. A second e-vapour with nicotine added to the solvent increased the toxic effects on these cells.

The third e-vapour comprised of nicotine, solvent, and vanilla custard flavouring (the flavour previously identified as most toxic) augmented damage to the spontaneously beating cells even more.

"This experiment told us that the flavouring chemicals added to vaping devices can increase harm beyond what the nicotine alone can do," Noujaim said.

The findings showed that mice exposed to vaping were more prone to an abnormal and dangerous heart rhythm disturbance known as ventricular tachycardia compared to control mice.

"Our research matters because regulation of the vaping industry is a work in progress," Noujaim noted.

--IANS

bu/bg

Read more:
Flavours added to vaping devices can damage the heart: Study - Sify News

Posted in Florida Stem Cells | Comments Off on Flavours added to vaping devices can damage the heart: Study – Sify News

The Chronicle of the Horse – The Chronicle of the Horse

Posted: December 12, 2020 at 6:55 pm

Mattie Worsham couldnt believe her luck the day her trainer suggested she hop aboard Scout. The brown warmblood of unrecorded breeding held superstar status, with the likes of John French, Archie Cox and Liza Boyd competing him at the most prestigious shows.

I can still remember very clearly the first day when I was training with Terry Brown, and he had come into her barn, said Worsham. I can remember the first day where she told me, Go get on Scout. And I was like, Are you sure? Do you really mean Scout? He sort of was and is the big name in the barn.

Worsham was only 13, and she quickly discovered that, despite his talent and training, or perhaps because of it, Scout was no pushbutton pony. I got on him, and I could not make him canter for the life of me, said Worsham. That remained a very big theme in our early years together and sometimes today.

But in 2015, Worshams last junior year, she finally felt like shed reached Scouts level. The pair was consistently champion in the small junior division and had qualified for Devon (Pennsylvania) for the first time.

The week before Devon, Worsham received a call from Brown. Scout had torn a hind suspensory, and theyd discovered osteoarthritis in both front fetlocks. Not only was Devon out of the picture, but it also looked like his performance days might be over for good.

I was so much more devastated at the time to lose a partner who I was so close with, to lose him to retirement and right on the cusp of everything, said Worsham. It was so much harder to think about not being able to ride than going to Devon and things like that.

Worshams parents, Penny and Hugh Worsham, DVM, and Brown helped her see past her sorrow. [They] could really empathize with how hard it was and what I was going through, but [they] also helped me to know that, even at that point, Id already had so many amazing moments with that horse, and he taught me so much, Mattie said. He challenges me, and he inspires me every single day, and he had already [done that]. So, they really helped me see that.

Mattie brought him home to retire at her parents farm near Atlanta. The Worshams didnt have a companion for Scout, so Mattie brought out the wheelbarrow and slept in it next to his stall so he wouldnt spend the night alone.

It was fine except for the fact that at 3 a.m. hed start pulling on my hair, said Mattie. I was within reach right outside the stall. Apparently, he can stretch out and grab me. More than any other horse I ended up sleeping in the stall/outside the stall for this horse.

While the prognosis for his injuries was poor, the Worshams werent ready to give up. Hugh had used Pro-Stride APS, an autologous protein solution made from the horses blood, for an unrelated injury earlier in Scouts career, and hed had good results. Hugh decided to try it again with Scouts fetlocks.

We call it a regenerative product or an ortho-regenerative injection, said Hugh. You harvest 60 ccs of blood off a horse. And theres a centrifuge that we take to the barn, its stall side, and we spin the blood down with these kits that Owl Manor provides.

Commonly used regenerative therapies include stem cells, interleukin-1 receptor antagonist protein (IRAP) and platelet-rich plasma (PRP). The Pro-Stride process produces a concentrated solution of cells, platelets, growth factors and anti-inflammatory proteins from the horses blood, and then the veterinarian injects them back into the point of interest.

Hugh injected Scout a few times, but they didnt put him into a focused rehabilitation program.

He just came home, and he got turned out, and my dad and my mom both worked on him and just never gave up, said Mattie. My dad was the veterinary care, and my mom was the day to day. I was at school at the time for undergraduate.

But then one day in 2018, Hugh saw Scout trotting around.

We can see the field from the deck of our house where we used to live, said Hugh. And Im watching him trotting around like a million bucks with this mini one day. Im thinking, Man, you are way too sound to just be living out here.

Three years after his injury, they pulled Scout out and pointed him toward a coop in the field. With his miniature horse companion Nugget running behind him, Scout jumped it and landed with ease. So the Worshams came up with a plan to get him back into shape, returning to the show ring in September in the adult amateur, 18-35, division.

The funny thing about it, when we brought him out of retirement, I havent fallen off in probably a year or two, said Mattie. And after we brought Scout out of retirement, it was like every week for a while. Our very first show back after three years, he bucked me off in the first class. I really wanted to do right by this horse because he was three years older, but so was I. Instead of being 18, I was 21, and I was in a very different place in my life. I felt like I was finally ready to give just everything that I had into doing right by this horse and trying not to let him down ever.

This fall, at 19 years old and five years after his first retirement, Scout traveled with Mattie to Tryon, North Carolina, to compete in the WIHS Adult Hunter Finals. After Scout laid down a second-round score of 86 to win the class and wear the Washington International cooler, Mattie knew this was the curtain call he deserved.

It was so redemptive and cathartic and just unrealI still dont even really believe itthat I could do that with this horse whos been my partner for so long and who I spent every single day with, said Mattie. I live in the barn [in Aiken, South Carolina,] right beside him. I can see his window from my window. It was really a strangely sublime moment, and [I felt] uncontainable gratitude for everything that I have with him. I just knew in that moment, This is the way I want to remember showing him. Being there with my trainer Courtney Goldstein, and my mom was there also, and getting to share that moment with them, thats the way that I want to remember showing this horse who was my junior hunter and then my adult hunter.

The way that he retired the first time was so unexpected and kind of a mess, and it wasnt on our own terms, continued Mattie. So getting to do it on our terms and walking out of the show ring with a win like that, on a horse who is very well known in the show world from his days even before I bought him, it felt really nice for him to get to end on top like that.

And while the win to close out his career was wonderful, just having those extra seasons with Scout is what Mattie will treasure. I had just as much fun when I was winning with him as when he was bucking me off in the ring because I was just so grateful to be showing this horse that I never thought I would show again, Mattie said. It really feels like this unexpected gift, and I wish I could go back and tell my 18-year-old self that this isnt the end. Its going to get better, and this horse is always going to be there for you. Hes never going to let you down.

Go here to see the original:
The Chronicle of the Horse - The Chronicle of the Horse

Posted in South Carolina Stem Cells | Comments Off on The Chronicle of the Horse – The Chronicle of the Horse

Negrin Shines Light on the Orca-T Story in GVHD – OncLive

Posted: December 11, 2020 at 7:59 am

What started out as a journey to better understand regulatory T cells has now led to an intriguing approach with an investigational cell therapy designed to prevent the risk of graft-versus-host disease (GVHD) and to improve relapse-free survival rates in patients undergoing hematopoietic stem cell transplantation (HSCT).

Data of a phase 1/2 trial recently showed that the first-generation precision cell treatment Orca-T compared with a historical control of standard HSCT demonstrated faster neutrophil (median, 12 days vs 14 days; P < .0001) and platelet engraftment (median, 11 days vs 17 days; P < .0001), decreased incidence of grade 2 or higher GVHD at 100 days (10% vs 30%, P = .005) and chronic GVHD at 1 year (3% vs 46%, P = .0002).1,2

The 1-year GVHD-free and GVHD relapse-free survival (GRFS) rates were 75% with the use of Orca-T vs 31% with standard HSCT (P < .0001). The comparator cohort was derived from contemporaneous patients who had been treated at Stanford University with a conventional allograft.

Along with feasibility of the approach, the results also highlight how Orca-T demonstrates potent anti-leukemic activity in patients who have active disease at HSCT, which suggests that the decrease of GVHD does not impact graft-vs-leukemia (GvL).

That is the most exciting part about the Orca-T story; it is the ability to do this with precision, with speed, and to export it to other sites. The results are intriguing, and very supportive, said Robert Negrin, a professor of medicine (blood and marrow transplantation), and chief of the Division of Blood and Marrow Transplantation at Stanford University.

In an interview with OncLive, Negrin, who is senior author on the trial, shared the evolution of Orca-T as a novel approach to HSCT, highlighted his robust experience with using this cell therapy at Stanford University, and how Orca-T is a potential prevention method for GVHD.

OncLive: Please provide some background to this therapeutic approach. What is the mechanism of action? How is it effective in patients undergoing transplant?

Negrin: This whole idea came from mouse studies many, many years ago, where we identified GVHD as being a dysregulated immune reaction that just keeps going, and going, and going. Like you and I, when we react to something, we have a reactionlet's say, influenza. Our body responds, and then we stop reacting and you get better. With GVHD, what we noticed in using a bioluminescent animal model is that the alloreactive T cells just keep going, going, and going and are unrelenting in mice, just like in people. The problem is very similar and affects certain organs in a very similar way.

Therefore, we went about trying to understand the use of so-called regulatory cells. These are cells that everybody has that help control immune reactions. We just applied them in this clinical scenario, first in mice work done by Matthias Edinger, MD, when he was a postdoctoral fellow many years ago [and other researchers]. All of them were very actively involved in these studies, and showed, somewhat surprisingly, that the administration of regulatory T cells could control this dysregulated immune response that we called GVHD.

Probably more surprising was that, at least in the animal models, it also allowed for the benefits of transplant, namely, the graft-vs-tumor effect and better immune recovery. This was in large part because GVHD also impacts the immune repertoire and where the immunity is developed in the recipient.

All of this was very nice in mouse models and was very elegant. We did a lot of studies, published a number of nice papers, and thought this would be a great idea because it sort of solved, or at least addressed, the principal problems after bone marrow transplantationnamely, avoidance of GVHD yet retention of graft-versus-tumor effects and better immunity. A lot of times, people say, "Oh, that sounds good in mice, but, that's too good to be true." And, theyll ask, "Will that all work in people?"

Where did the biggest challenges lie in this approach?

The big challenge came about to try to apply this to patients. We also have one other interesting point that is relevant. If we gave the regulatory T cells first, before the so-called conventional CD4+/CD8+ cells, that allowed for a lower dose of regulatory T cells. This is because a big challenge is the paucity of these cells; you and I don't have that many.

Then, the other big challenge was the technical ability to isolate in cells. What we do in mice is cell sorting, which is a standard technology. But, that was not developed in people because we're bigthere are a lot of cells, and cell sorting is rather slow, and it's very specific. To get enough cells takes a really long time. It's somewhat of a heroic thing to do in people, to get the adequate amount ourselves; of course, we don't really know what this proper cell dose is.

However, what we thought we learned was that the ratio of conventional to regulatory T cells was the key component. Also, if you give the regulatory T cells first, you can get fewer numbers. Those are things you can do in transplant. You can get the cell from the donor, and you can give cells in a certain sequence; all of those things are very doable. It seemed like an attractive thing to do in patients.

Then, the question was: Does it work? There are 3 groups that have really pioneered this work. The first study came from the University of Perugia in Italy. They did this in haploidentical transplantation; you cannot avoid immunosuppression in haploidentical transplants. They were able to show in several nice papers that you could do this strategy, and seemingly, get away with low risk of GVHD, and also low relapse. This is because the other issue is: how do you measure the graft-vs-tumor effect? There is no assay, and we have no test; you have to wait and see who relapses and who doesn't. Therefore, they also showed rather convincingly that you could reduce GVHD risk, yet, there was a very low risk of relapse in their high-risk patient population. Those were very important [data].

Another study from the University of Minnesota did this with umbilical cord blood. They expanded the regulatory T cells from a third cord blood unit, which is somewhat heroicit is another level of complexity to isolate the cells and then expand them. We did this in matched donorseither matched siblings or matched unrelated donors. We published a paper in JCI Insight several years ago showing the initial results, and they look quite favorable.

Therefore, what I think is most exciting about what Orca Bio has done is they are developing technology to isolate the cells more quickly, to be able to do this on a clinical scale, with precision, and with speed. Also, [they are developing the technology] to be able to distribute it to anybody, because the criticism of all these studies is that, "Oh, that's nice. But, this is a single-institution study. Is this really true? Can this be exported? Could this be something that [an organization] other than these [individual] centers are really focused in this area and have developed these technologies could really do? Orca Bio is developing the technology, and improving the technology, because it's still very cumbersome, and exporting the technology so that you could do this, theoretically, at any center.

That's what I think is most exciting about the Orca Bio abstract; it is demonstrating that this can be done. It certainly opens the door to prevention of GVHD. As we move into an era of using cell-based therapeutics, now, this opens up many other possibilities, because you use these regulatory cells and autoimmune disorders and organ transplant tolerance. There are many other cell types that have potential clinical utility, but getting them, and purifying them, is a big challenge. There are many other possibilities that one could think of.

Obviously, more time will be required to follow these patients, but they certainly are supportive of the idea that you can improve overall outcomes using this strategy. That's what we hope to be able to demonstrate further.

Please focus on the scalability of this approach. Through these types of collaborations, how do you see Orca-T potentially moving through the FDA pipeline?

In academia, we don't develop drugs. It's too much, we don't have the resources, we don't have the capability, and we don't have the monitoring capability that is required for multi-institutional studies. Where these commercial partners come in is, they can raise money for interesting concepts, which Orca Bio has done, and they can export this to other centers, and that's critically important.

As we've seen in the CAR T-cell [therapy] world, that can be a quite successful commercial business. Also going through the process of an FDA approvalwhich Orca Bio is moving along in that processand getting the right designations is critically important to commercial entities. In academia, it's important to us, but that's just not our focus.

We don't have the resources around, the people and the expertise to really drive things through that process. We're good at developing the studies and getting FDA approvals, and [investigational new drug applications], but not really [good at] developing drugs as a commercial entity. This collaboration is key to doing this successfully; for example, at Orca Bio, [they have] technology to separate cells more efficiently and effectively. They also have the resources to do a multi-institutional clinical trial, and the expertise to move something through and present it to the FDA. Those are key components.

Could you expand on the study and respective data from this phase 1/2 trial?

Here at Stanford Cancer Institute, we did find in our patients that giving low doses of immunosuppressive medications with a single agent seem to improve the outcomes, and it's remarkable how well these patients have gone through the transplant. It's a little bit hard to appreciate an abstract until you take care of these patients, and many of them just sort of move to the transplant with relatively little challenges. We have not seen greater risks of things like infection [or] disease recurrence; those are obviously things that will be followed.

When we look at the 1-year GVHD relapse-free survival rate, which is an endpoint that most transplant studies would agree is the most important end point, the overall outcomes are much more favorable compared with a historical control group.

The data are very encouraging, and the overall outcomes look very strong in a reasonable number of patients now. We think it's important for the community to hear about it, and to get it on everybody's radar, and be excited about trying to move this forward as a more standard therapy. This is still a clinical trial, so it's not, it's not part of any standard therapies yet. We are using this quite regularly and have been very encouraged by the ease of which patients go through the transplant. It's still an allogeneic transplant; there still are many challenges there. However, these patients seem to be doing quite well, we're very encouraged, and so we keep going.

How does this approach impact patient outcomes as it relates to quality of life (QoL)?

The hard end points of 1-year relapse-free survival is obviously the most important to patients. However, going through an allogeneic transplant is obviously an incredibly difficult thing. Fortunately, I've only seen it [from] the doctor side, not [as a] patient.

However, I've seen many, many patients, and the quality of their life as they go through this experience is very important to all of us. As we saw these patients go through these studies, we felt like we were capturing something that was really important, and that is the ease [at which] many patients went through this experience, which just seemed different. It's hard to capture that.

It's really important for patients to speak and, and the way patients speak is in different ways. One way is through the QoL measures that they answer. This is [what they find] important, this is what they experiencednot what we say is happening. That's really important to hear that voice too. Those are data we're trying to collect. It's not so easy, because going through a bone marrow transplant is a poor QoL for everybody. But, by just to trying to capture this, [Orca-T seems] better than what we what we thought.

How has this changed the mindset of cell-based approaches in the community?

What has changed is the belief in the concept of cell-based therapies. A lot of these things are somewhat fanciful. It is also important to show that we can translate from an animal model [to a human]. There is a lot of criticism of animal modeling, because people say, "Well, it's nice for animal models, but it doesn't really translate into the clinic." Actually, my view is that because we don't actually follow the animal models, there are many compromises one needs to make. When you translate studies from animals to humans, there are many differences, and it's really important to try to follow them as carefully as you can within the limitations of what is possible. We were very engaged in that and tried to follow as carefully as we could. To me, that is very encouragingthat you can study things in animals that generate new concepts and be able to translate that into a clinical trial.

Obviously, with all of the caveats of an early-phase clinical trial, more time needs to pass, more patients to be treated, and you need to export [the treatment] to other centers. That's a really important point, because there are many things that get lost because, "it's too complicated. It's too expensive. People can't do it." I don't think anybody can do high-speed cell sorting, as a clinical project in a standard or standard cell-processing laboratory. It's above the level of what most processing laboratories can do.

References

Follow this link:
Negrin Shines Light on the Orca-T Story in GVHD - OncLive

Posted in Minnesota Stem Cells | Comments Off on Negrin Shines Light on the Orca-T Story in GVHD – OncLive

CollPlant to Supply rhCollagen to STEMCELL Technologies for Use in a Broad Range of Cell Culture Applications – PRNewswire

Posted: December 10, 2020 at 3:52 pm

REHOVOT, Israel and VANCOUVER, BC, Dec. 10, 2020 /PRNewswire/ -- CollPlant (NASDAQ: CLGN), a regenerative medicine company, and STEMCELL Technologies, Canada's largest privately owned biotechnology company, which develops cell culture media, cell separation systems, instruments, and other reagents for life sciences research, today jointly announced they have entered into aproduct manufacturing and supply agreement. CollPlant will sell its proprietary recombinant human Type I collagen (rhCollagen), the world's first plant-based rhCollagen, to STEMCELL Technologies, which will incorporate CollPlant's product into cell culture media kits.

The recently signed agreement follows the companies' established business relationship, which started in 2014 when STEMCELL began purchasing and incorporating CollPlant's rhCollagen into some of its cell culture expansion and differentiation media kits. To date, hundreds of companies, as well as research and academic institutes, have used these kits for research and development projects. STEMCELL will distribute the kits globally for use in the regenerative medicine research market.

"Incorporation of rhCollagen into STEMCELL's cell culture applications sold to researchers worldwide is designed to help advance the science in a broad range of dynamic fields including stem cells, immunology, cancer, regenerative medicine, and cellular therapy. We are happy to have entered into this agreement with STEMCELL, which, as Canada's largest biotechnology company, is very well positioned to make rhCollagen-containing cell culture kits widely available in the market," stated Yehiel Tal, Chief Executive Officer of CollPlant. "The cell culture market is just one example of the vast potential of our rhCollagen platform technology in life science applications. We continuously evaluate new fields in which CollPlant's products and technologies have the potential to enable breakthroughs that improve patients' lives."

Dr. Sharon Louis, STEMCELL's Senior Vice President of Research and Development noted that "STEMCELL is pleased to utilize CollPlant's animal component free rhCollagen to promote cell attachment in several products that support the culture of diverse human progenitor cell types. The quality and animal component-free composition of CollPlant's rhCollagen is what first brought this product to STEMCELL's attention, and the robust performance rhCollagen provides with a variety of STEMCELL media is what we want to be able to provide to our customers. Upon entering into this agreement, STEMCELL and CollPlant will together provide high-quality reagents that will be used to further our understanding in life sciences and potentiate regenerative medicine research."

About STEMCELL Technologies

STEMCELL Technologies is Canada's largest biotechnology company. Based in Vancouver, STEMCELL supports life sciences research around the world with more than 2,500 specialized reagents, tools, and services. STEMCELL offers high-quality cell culture media, cell separation technologies, instruments, accessory products, and educational resources that are used by scientists advancing the stem cell, immunology, cancer, regenerative medicine, microbiology, and cellular therapy fields.

Find more information at http://www.stemcell.com

About CollPlant Biotechnologies

CollPlant is a regenerative and aesthetic medicine company focused on 3D bioprinting of tissues and organs, and medical aesthetics. Our products are based on our rhCollagen (recombinant human collagen) that is produced with CollPlant's proprietary plant based genetic engineering technology.

Our products address indications for the diverse fields of tissue repair, aesthetics and organ manufacturing, and, we believe, are ushering in a new era in regenerative and aesthetic medicine.

Our flagship rhCollagen BioInk product line is ideal for 3D bioprinting of tissues and organs. In October 2018, we entered into a licensing agreement with United Therapeutics, whereby United Therapeutics is using CollPlant's BioInks in the manufacture of 3D bioprinted lungs for transplant in humans.Recently, the parties announced the expansion of the collaboration with the exercise by United Therapeutics of its option to cover a second lifesaving organ, human kidneys.

Safe Harbor for Forward-Looking Statements

This press release may include forward-looking statements. Forward-looking statements may include, but are not limited to, statements relating to CollPlant's objectives, plans and strategies, as well as statements, other than historical facts, that address activities, events or developments that CollPlant intends, expects, projects, believes or anticipates will or may occur in the future. These statements are often characterized by terminology such as "believes," "hopes," "may," "anticipates," "should," "intends," "plans," "will," "expects," "estimates," "projects," "positioned," "strategy" and similar expressions and are based on assumptions and assessments made in light of management's experience and perception of historical trends, current conditions, expected future developments and other factors believed to be appropriate. Forward-looking statements are not guarantees of future performance and are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Many factors could cause CollPlant's actual activities or results to differ materially from the activities and results anticipated in forward-looking statements, including, but not limited to, the following: the CollPlant's history of significant losses and its need to raise additional capital and its inability to obtain additional capital on acceptable terms, or at all; CollPlant's expectations regarding the timing and cost of commencing clinical trials; regulatory action with respect to rhCollagen-based products, including but not limited to acceptance of an application for marketing authorization, review and approval of such application, and, if approved, the scope of the approved indication and labeling; commercial success and market acceptance of the CollPlant's rhCollagen-based BioInk; CollPlant's ability to establish sales and marketing capabilities or enter into agreements with third parties and its reliance on third-party distributors and resellers; CollPlant's reliance on third parties to conduct some aspects of its product manufacturing; the scope of protection CollPlant is able to establish and maintain for intellectual property rights and the company's ability to operate its business without infringing the intellectual property rights of others; the overall global economic environment; the impact of competition and new technologies; general market, political, and economic conditions in the countries in which the company operates; projected capital expenditures and liquidity; changes in the company's strategy; and litigation and regulatory proceedings. More detailed information about the risks and uncertainties affecting CollPlant is contained under the heading "Risk Factors" included in CollPlant's most recent annual report on Form 20-F, filed with the SEC, and in other filings that CollPlant has made. The forward-looking statements contained in this press release are made as of the date of this press release and reflect CollPlant's current views with respect to future events, and CollPlant does not undertake, and specifically disclaims, any obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise.

Contact atCollPlant:

Eran RotemDeputy CEO & CFOTel: + 972-73-2325600[emailprotected]

Contact at STEMCELL: Luba Metlitskaia Vice President, Business Development & Licensing [emailprotected]

SOURCE CollPlant

See the rest here:
CollPlant to Supply rhCollagen to STEMCELL Technologies for Use in a Broad Range of Cell Culture Applications - PRNewswire

Posted in Cell Medicine | Comments Off on CollPlant to Supply rhCollagen to STEMCELL Technologies for Use in a Broad Range of Cell Culture Applications – PRNewswire

Faze Medicines Launches With $81 Million Series A Financing to Leverage New Biology of Biomolecular Condensates to Treat Disease – Business Wire

Posted: December 10, 2020 at 3:52 pm

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Faze Medicines, a biotechnology company pioneering therapeutics based on the groundbreaking new science of biomolecular condensates, today announced its launch and Series A financing of $81 million. Faze is founded by leading experts in the emerging field of biomolecular condensates with the mission of leveraging this fundamentally new understanding of cell biology to develop therapies to slow, halt or reverse disease pathology. The Series A was led by Third Rock Ventures with Novartis Venture Fund, Eli Lilly and Company, AbbVie Ventures, Invus, Catalio Capital Management, Casdin Capital and Alexandria Venture Investments participating.

Biomolecular condensates are membrane-less clusters of molecules, such as proteins and nucleic acids, that dynamically organize to perform a wide array of cell functions. Research over the past decade, including seminal work by Fazes founders, has found that disturbances in the behavior of condensates play a causative role in myriad human diseases, including amyotrophic lateral sclerosis (ALS) and other neurodegenerative disorders. Faze is now poised to deliver medical breakthroughs based on this fundamentally new understanding of cell biology.

The biology of condensates is the kind of science that will rewrite textbooks and, we believe, rewrite medicine, said Cary Pfeffer, M.D., interim chief executive officer of Faze and partner at Third Rock Ventures. Faze is founded by leading experts who have been integral to this field since its very beginnings. Their insights, coupled with the deep expertise of the team we have assembled, will enable us to realize the enormous potential of this new biology.

Cell biology is undergoing a transformation as we come to understand the integral role that biomolecular condensates play within cell processes from DNA repair to intracellular transport, added Rachel Meyers, Ph.D., chief scientific officer of Faze. Faze was founded to translate these exciting discoveries out of the lab and into the clinic, where they could make a real difference in treating diseases that have seen very little therapeutic progress.

The Series A will support Fazes preclinical research in two initial therapeutic focus areas ALS and myotonic dystrophy type 1 (DM1) as well as research to explore condensate biology in other disease areas. In ALS and DM1, a robust body of literature points to a causative role for condensate dysregulation. Leveraging state-of-the-art screening and proteomics techniques, Faze will identify proteins that are key components or regulators of disease-causing condensates, and then employ proprietary assays to discover small molecule drugs targeting these proteins.

Founders and Leadership

Faze is founded by renowned scientific leaders in the field of biomolecular condensates:

Fazes leadership team brings together accomplished biotechnology executives with decades of industry experience and deep scientific, drug discovery and drug development knowledge:

Joining Dr. Pfeffer on the companys inaugural board of directors is:

Faze has additionally established a robust group of expert advisors including those in the areas of drug discovery and clinical development.

About Faze Medicines

Faze Medicines is a biotechnology company harnessing the groundbreaking new science of biomolecular condensates to create medical breakthroughs. Faze was founded by renowned scientific leaders in the field of biomolecular condensates and is supported by a world-class syndicate of investors including Third Rock Ventures, Novartis Venture Fund, Eli Lilly and Company, AbbVie Ventures, Invus, Catalio Capital Management, Casdin Capital and Alexandria Venture Investments. For more information, visit fazemed.com.

Follow this link:
Faze Medicines Launches With $81 Million Series A Financing to Leverage New Biology of Biomolecular Condensates to Treat Disease - Business Wire

Posted in Cell Medicine | Comments Off on Faze Medicines Launches With $81 Million Series A Financing to Leverage New Biology of Biomolecular Condensates to Treat Disease – Business Wire

Editas Medicine Announces Submission of IND Application for EDIT-301 with the FDA – GlobeNewswire

Posted: December 10, 2020 at 3:52 pm

EDIT-301 is in development as a best-in-class, durable medicine for people living with sickle cell disease

CAMBRIDGE, Mass., Dec. 09, 2020 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a leading genome editing company, today announced it submitted an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for the initiation of a Phase 1/2 clinical trial of EDIT-301, an experimental CRISPR/Cas12a gene editing medicine in development for the treatment of sickle cell disease. The Company previously received Rare Pediatric Disease designation from the FDA for EDIT-301.

This IND submission is a key milestone for Editas as we continue to advance several ex vivo cell therapy medicines. This submission brings us one step closer to entering the clinic with our potentially best-in-class, transformative, and durable medicine for people living with sickle cell disease, said Cynthia Collins, Chief Executive Officer, Editas Medicine. This moment is very exciting for the Editas team. We know patients are counting on us, and we look forward to next steps for the clinical development of EDIT-301, including dosing sickle cell disease patients.

Editas Medicine continues to prepare for a Phase 1/2 clinical trial evaluating EDIT-301 for the treatment of sickle cell disease. The Company has identified a lead principal investigator and engaged a Clinical Research Organization (CRO). Clinical trial materials are being manufactured by Editas Medicine.

About Sickle Cell DiseaseSickle cell disease is caused by a mutation in the beta-globin gene that leads to polymerization of the sickle hemoglobin protein (HbS). Fetal hemoglobin (HbF) protects against sickle cell disease by inhibiting HbS polymerization. Individuals with high levels of HbF are protected from sickle cell disease. EDIT-301 is an experimental, autologous cell therapy comprising CD34+ cells genetically modified using a Cas12a ribonucleoprotein (RNP) that targets the HBG1/2 promoter in the beta-globin gene to stimulate HbF production.

About EDIT-301EDIT-301 is an experimental, autologous cell therapy medicine under investigation for the treatment of sickle cell disease. EDIT-301 is comprised of sickle patient CD34+ cells genetically modified using a highly specific and efficient CRISPR/Cas12a (also known as Cpf1) ribonucleoprotein (RNP) to edit the HBG1/2 promoter region in the beta-globin locus. Red blood cells derived from EDIT-301 CD34+ cells demonstrate a sustained increase in fetal hemoglobin (HbF) production, which has the potential to provide a durable treatment benefit for people living with sickle cell disease.

AboutEditas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Companys plans and expectations for EDIT-301. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials and clinical development of the Companys product candidates; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in the Companys most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.

Continued here:
Editas Medicine Announces Submission of IND Application for EDIT-301 with the FDA - GlobeNewswire

Posted in Cell Medicine | Comments Off on Editas Medicine Announces Submission of IND Application for EDIT-301 with the FDA – GlobeNewswire