Category Archives: Stem Cell Research

Stem Cell Exosomes Market: Increasing advanced applications of exosomes is expected to drive the market – BioSpace

Posted: December 16, 2020 at 10:57 am

Stem Cell Exosomes Market: Overview

Exosomes possesses the potential to be a carrier for drug delivery owing to their transportation properties. The stem cell exosomes have other properties of high biocompatibility and intrinsic long-term circulation, which are ideal for proteins, nucleic acids, and chemicals. Additionally, new researches showed results of exosomes possessing properties of mediators in intercellular communication and mRNA transcripts, delivering proteins, and many others. They have properties, which make them biocompatible and useful to become agents to provide treatment for various disorders.

The rapidly increasing interest for advanced material to provide disease-based treatment in case of emergency is inducing more research and funding to explore stem cell exosomes. This is a key factor driving growth of the stem cell exosomes market from past few years and is estimated to be the same for next few years as well.

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Stem Cell Exosomes Market: Notable Development

The stem cell exosomes market is identified as highly competitive without dominant players owing to many players operating in the market. Some of the key players in the market include Anjarium Biosciences, Codiak Biosciences, Capricor Therapeutics, Creative Medical Technology Holdings, Evox Therapeutics, Everkine Corporation, Exogenus Therapeutics, ReNeuron, Kimera Labs, and Unicyte AG.

The market is witnessing lucrative investments for adoption of newer and improving technologies. Such investments are on grounds of few acquisitions and mergers, tie ups, and to cater to global population.

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Some of few developments observed in the market:

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Stem Cell Exosomes Market: Growth Factors

The factors impacting on growth of the market include increasing prevalence of cancer and advent of technological advancements in exosomes and its applications. Additionally, increasing advanced applications of exosomes coupled with increasing awareness about presence of improved medical techniques are propelling growth of the global stem cell exosomes market. The 2012 reports by World Health Organization (WHO), the number of patients is expected to increase by 70% in next two decade. Increase in patients may lead to increase in fatality due to cancer, which increase attention toward advanced medications. This factor is likely to boost demand for the exosomes in diagnosis and therapeutics.

However, number of technical difficulties are limiting its adoption globally and hindering growth of the global stem cell exosomes market. The other factors restraining market growth are stringent regulatory frameworks and commercialization of exosomes. Nonetheless, the factors such as increase in research coupled with funding for researches are estimated to open doors of opportunities for growth in coming future.

1st Patients To Get CRISPR Gene-Editing Treatment Continue To Thrive – NPR

Posted: December 16, 2020 at 10:57 am

Victoria Gray (second from left) with children Jamarius Wash, Jadasia Wash and Jaden Wash. Now that the gene-editing treatment has eased Gray's pain, she has been able be more active in her kids' lives and looks forward to the future. "This is really a life-changer for me," she says. Victoria Gray hide caption

The last thing a lot of people want to do these days is get on a plane. But even a pandemic would not stop Victoria Gray. She jumped at the chance to head to the airport this summer.

"It was one of those things I was waiting to get a chance to do," says Gray.

She had never flown before because she was born with sickle cell disease. She feared the altitude change might trigger one of the worst complications of the devastating genetic disease a sudden attack of excruciating pain.

But Gray is the first person in the United States to be successfully treated for a genetic disorder with the help of CRISPR, a revolutionary gene-editing technique that makes it much easier to make very precise changes in DNA.

About a year after getting the treatment, it was working so well that Gray felt comfortable flying for the first time. She went to Washington, D.C., to visit her husband, who has been away for months on deployment with the National Guard.

"It was exciting. I had a window. And I got to look out the window and see the clouds and everything," says Gray, 35, of Forest, Miss.

Gray wore a mask the whole time to protect herself against the coronavirus, kept her distance from other people at the airport, and arrived happily in Washington, D.C., even though she's afraid of heights.

"I didn't hyperventilate like I thought I would," Gray says, laughing as she recounts the adventure in an interview with NPR.

NPR has had exclusive access to follow Gray through her experience since she underwent the landmark treatment on July 2, 2019. Since the last time NPR checked in with Gray in June, she has continued to improve. Researchers have become increasingly confident that the approach is safe, working for her and will continue to work. Moreover, they are becoming far more encouraged that her case is far from a fluke.

At a recent meeting of the American Society for Hematology, researchers reported the latest results from the first 10 patients treated via the technique in a research study, including Gray, two other sickle cell patients and seven patients with a related blood disorder, beta thalassemia. The patients now have been followed for between three and 18 months.

All the patients appear to have responded well. The only side effects have been from the intense chemotherapy they've had to undergo before getting the billions of edited cells infused into their bodies.

The New England Journal of Medicine published online this month the first peer-reviewed research paper from the study, focusing on Gray and the first beta thalassemia patient who was treated.

"I'm very excited to see these results," says Jennifer Doudna of the University of California, Berkeley, who shared the Nobel Prize this year for her role in the development of CRISPR. "Patients appear to be cured of their disease, which is simply remarkable."

Another nine patients have also been treated, according to CRISPR Therapeutics in Cambridge, Mass., and Vertex Pharmaceuticals in Boston, two companies sponsoring the research. Those individuals haven't been followed long enough to report any results, officials say.

But the results from the first 10 patients "represent an important scientific and medical milestone," says Dr. David Altshuler, Vertex's chief scientific officer.

The treatment boosted levels of a protein in the study subjects' blood known as fetal hemoglobin. The scientists believe that protein is compensating for defective adult hemoglobin that their bodies produce because of a genetic defect they were born with. Hemoglobin is necessary for red blood cells to carry oxygen.

Analyses of samples of bone marrow cells from Gray six months after getting the treatment, then again six months later, showed the gene-edited cells had persisted the full year a promising indication that the approach has permanently altered her DNA and could last a lifetime.

"This gives us great confidence that this can be a one-time therapy that can be a cure for life," says Samarth Kulkarni, the CEO of CRISPR Therapeutics.

Gray and the two other sickle cell patients haven't had any complications from their disease since getting the treatment, including any pain attacks or hospitalizations. Gray has also been able to wean off the powerful pain medications she'd needed most of her life.

Prior to the treatment, Gray experienced an average of seven such episodes every year. Similarly, the beta thalassemia patients haven't needed the regular blood transfusions that had been required to keep them alive.

"It is a big deal because we we able to prove that we can edit human cells and we can infuse them safely into patients and it totally changed their life," says Dr. Haydar Frangoul at the Sarah Cannon Research Institute in Nashville. Frangoul is Gray's doctor and is helping run the study.

For the treatment, doctors remove stem cells from the patients' bone marrow and use CRISPR to edit a gene in the cells, activating the production of fetal hemoglobin. That protein is produced by fetuses in the womb but usually shuts off shortly after birth.

The patients then undergo a grueling round of chemotherapy to destroy most of their bone marrow to make room for the gene-edited cells, billions of which are then infused into their bodies.

"It is opening the door for us to show that this therapy can not only be used in sickle cell and thalassemia but potentially can be used in other disorders," Frangoul says.

Doctors have already started trying to use CRISPR to treat cancer and to restore vision to people blinded by a genetic disease. They hope to try it for many other diseases as well, including heart disease and AIDS.

The researchers stress that they will have to follow Gray and many other patients for a lot longer to be sure the treatment is safe and that it keeps working. But they are optimistic it will.

Gray hopes so too.

"It's amazing," she says. "It's better than I could have imagined. I feel like I can do what I want now."

The last year hasn't always been easy for Gray, though. Like millions of other Americans, she has been sheltering at home with three of her children, worrying about keeping them safe and helping them learn from home much of the time.

"I'm trying to do the things I need to do while watch them at the same time to make sure they're doing the things they need to do," Gray says. "It's been a tough task."

But she has been able do other things she never got to do before, such as watch her oldest son's football games and see her daughter cheerleading.

"This is really a life-changer for me," she says. "It's magnificent."

She's now looking forward to going back to school herself, learning to swim, traveling more when the pandemic finally ends, and watching her children grow up without them worrying about their mother dying.

"I want to see them graduate high school and be able to take them to move into dorms in college. And I want to be there for their weddings just everything that the normal people get to do in life. I want to be able to do those things with my kids," she says. "I can look forward now to having grandkids one day being a grandmama."

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1st Patients To Get CRISPR Gene-Editing Treatment Continue To Thrive - NPR

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Cord blood banks sell parents on promising stem cell research, but with no guarantees – The Arizona Republic

Posted: December 9, 2020 at 1:59 am

Stem cell treatment promise sells pregnant parents on cord blood banks Arizona Republic

Moments after Linda Buzans son Luca is born, her OB-GYN goes to work. She carefully cuts the white umbilical cord, then clamps it shut before any blood can escape. Once the cord is cleaned, she carefully inserts a needle with a long tube attached and lets the umbilical cord blood pump into a collection bag.

From there, the sample will travel in a labeled collection box to Tucson, where a laboratory for one of the oldest and biggest private cord blood banking companies nationwide is located. There, at the Cord Blood Registry laboratory, or CBR,baby Lucas umbilical cord blood will be frozen in a metal tank at less than minus 320 degrees, joining almost 900,000 other samples in storage,including that of his older sister, Lola.

Umbilical cord blood is full of stem cells, so it can be transplanted into patients to treat certain types of cancers, diseases and blood disorders. Umbilical cord blood works as an alternative for many patients who cant find bone marrow transplants.

Butthe odds that either Lola or Luca will develop a disease like cancer that would require an umbilical cord blood transplant are slim,about one in 1,000 or one in 2,000, according to University of Arizona umbilical cord blood stem cell researcher David Harris.

Its difficult to reconcile saving for yourself because youre afraid of cancer, Harris said. Do it based on facts, not fear.

Stem cells in umbilical cord blood could have another purpose: regenerative medicine. Current clinical trials show promise for the use of umbilical cord blood to treat a host of conditions such as neurological disorders, orthopedic injuriesand even diabetes. These potential usages are a new draw for parents to bank their childs umbilical cord blood.

The odds of use for these regenerative medicine applications is much, much higher, Harris said.

He estimates the odds of developing a disease that could be treated by umbilical cord blood stem cells is about one in ten.

Butthe science is still developing, which meanscompanies are selling parents ona product they may never be able to use.

In the past two decades, Harris has been involved with studies to treat kids with conditions like cerebral palsy, strokes, traumatic brain injuries and diabetes.

When you now start to talk about being able to treat a knee injury or a heart attack, or a stroke the ability to actually do that and then to see that it works is very exciting, he said. And thats really where the use of cord blood is going.

So far, Harris said hes seen the most success with orthopedic injuries and in treating children that have suffered from strokes.

With strokes, Harris said he observed children go from being completely paralyzed on one side to being fully functioning.

DONATIONS:Donated cord blood saved Sophie Lee's life, but most parents throw it away

Though some initial results show promise, Harris acknowledges that to move forward with any of these treatments, researchers need to demonstrate a good success rate.

The big question when it comes to using cord blood for regenerative medicine is when it will be incorporated into actual medical practice. For safety reasons, FDA approval for new treatments can take years,if not decades.

Currently, the only way to use umbilical cord blood stem cells for FDA-approved regenerative medicine is to qualify for and register in clinical trials to treat specific conditions. Harris has banked his owns sons cord blood on his belief that more and more umbilical cord blood treatments will become commercially available in the future. Buzan has done the same.

Brandon Buzan packages up his newborn son's umbilical cord blood to be shipped to the Cord Blood Registry lab for storage.(Photo: Amanda Morris)

Its not something that you want to say I wish I had done this, because you cant do it later.You have this one shot,"Buzan said."Even its like one percent of saving their life or helpingI mean for your child, youd do anything as a parent.

In Arizona, obstetrics health providers are required by law to educateexpectant parents about the options to publicly donate or privately bank cord blood. YetDr. Jaime Shamonki, the Chief Medical Officer of CBR, estimates that less than 5% of the population chooses to bank cord blood. A small percentage donate the blood, but a much larger percentage simply throws it away.

Kelly Helms, a Scottsdale-based OB-GYN, said the most common reasonher patients give for not banking their childs cord blood is the cost.

Buzan, who is one of her patients, said she got a discount to bank her childrens samples with CBR because of a connection her familyhad with the company. She paid a little over $1,000 for the initial processing and storage fee for both samples, and continues to pay an annual storage fee of about $120 for each one.

To bank one sample of cord blood and cord blood tissue, the initial cost is$2,830, according to CBR, with a $360 annual storage fee after that. To bank just the cord blood, not the tissue, the initial cost is $1,680, with a $180annual storage fee. Both cord blood and cord tissue have different types of stem cells that are thought to potentially repair the body in different ways.

The stem cells and potential treatments for both sources arent fully understood yet, so there are no guarantees that parents or children will actually be able to use the samplesthey pay to store.

Of the nearly 900,000 samples CBR keeps in storage, Shamonki estimates about 600 have been released for customers to use,representing a usage rate of less than 1 percent. According to Shamonki, the low sample release rate is due to FDA regulations, which stipulate that umbilical cord blood can only be used in approved treatments or clinical trials.

If we didnt have the FDA, then I would be able to release like thousands of units, she said. Its really a regulatory problem, its not a utility problem.

To boost usage of the samples, CBR maintains a registry to match eligible customers to clinical trials and has partnered with research institutions to sponsor clinical trials.

Despite FDA regulations, which Shamonki acknowledges are important to protect public health, she said CBR is releasing more and more units every year. Of the samples taken out, about 83% are used for regenerative medicine purposes, according to CBR.

What I know is that in the next fiveyears, next 10 years, there will be so many more opportunities, Shamonki said. So just because you dont have 100 different clinical trials you can sign up for tomorrow doesnt mean that these applications wont exist in fiveor 10 years, and your child might need it.

CBR is one of many companies that market cord blood banking to new parentsand is one of the biggest. Helms said she always recommends her patients to do their research and pick one of the larger, more established cord blood banking companies. Such companies might includeCBR, Cryo-cell, or ViaCord.

I've had patients lose their cord blood, privately banked blood, because they went with a small company and now they closed down, Helms said.

Even with some of the larger companies, the process of cord blood banking doesnt always run smoothly.

Although she couldn't have her daughter's cord blood stored, Chelsea Radford says she paid over $1,000 to ViaCord for processing fees.(Photo: Amanda Morris)

The first time Phoenix-resident Chelsea Radford heard about cord blood banking, she was already pregnant and facingmyriaddecisions that were sometimes overwhelming. She had never heard of it before reading some pamphlets from her gynecologist, but she was quickly sold on the idea of banking her daughters cord blood and tissue with ViaCord.

Radford has a history of Alzheimers disease in her family, and said she was initially interested in what potential treatments cord blood and tissue might offer for the disease. In 2015, one study suggested that human umbilical cord blood cells could have therapeutic benefitsin mice with Alzheimers disease.

In addition to researching studies, Radford said she spent hours looking at different cord blood banking companies and asking representatives questions about the process. Of all the companies, she found ViaCord to be the most responsive and willing to answer her questions in-depth.

Having the communication and the availability that is what sold me on ViaCord. But that really quickly ended there with the sale, she said.

On the day of her daughter Brylees birth in July2018,Radford went to a hospital that ViaCord had assured her was familiar with collecting cord blood. Soon after, she got a call from the company saying her sample couldnt be stored because not enough blood was collected. Shestill owed ViaCord over $1,000 for a lab processing fee.

Radford wanted more information before she would agree to pay, and said she spent monthscalling, leaving the company messages, and getting no response.

After the birth, nobody responds to anything, she said.

The company called her three months later to tell her that if she didnt pay her bill, they would send it to collections.

I got pissed! Radford said. The only thing they seem to care about is the moneythey dont care about is having a conversation with me about why and how this sample didnt turn out the way it shouldve. All they want to talk to me about is the money.

Still, Radford said she refused to pay a dime until she got an explanation. She contacted the doctor who delivered her baby and said she learned the doctor had never done a cord blood collection before and had never been trained on how to do one.

Finally, she spoke with a ViaCord representative, who she saidtold her this sometimes happens, butthe company wasnt responsible for the fact that the doctor who took her sample didnt take enough blood.

Frustrated and inundated with other responsibilities that come with caring for a newborn, Radford said she decided to pay the fee so that she could move on.

We paid a company to do nothing for us just to get them to leave us alone and not send a bill to collections that I dont feel like we shouldve had to foot in the first place, she said.

If she had a second child, Radford said she wouldnt choose to cord blood bank again, and doesnt recommend it to other moms.

You can still get stem cell help without using your own banked blood and tissueThis is just a costly option that is not a given that its going to work out 100% in your favor, she said. You could have a newborn and be responsible to pay thousands of dollars for nothing.

ViaCord did not respondto multiple requests for comment.

If parents decide to pay for private banking, Radford said they should be careful about making sure the doctors delivering their children know how to collect the samples. Shesaid blood banking companies should be more responsible in making sure that doctors are trained in blood collection.

While Helms is comfortable doing cord blood collection, she was never formally taught how to do it while studying and training to be an OB-GYN.

It was basically taught by the company, she said. Each kits a little different.

Helms said the procedure is fairly simple, but every once in a while, she comes across a company shes never heard of, and a kit she is unfamiliar with. Sometimes she needs to take on the extra responsibility of making sure she understands the directions for that particular kit.

Each company really should take on the responsibility, she said. I can't surf the Internet and look for every YouTube video on every cord blood banking company, she said.

Another potential complication in banking cord blood orblood tissue is that the blood or tissue can become infected.

Birth is not that clean of a process and ideally when you take that needle and you drain the umbilical cord, youll have cleaned that umbilical cord first and you hope that no bacteria get in to the cord blood unit, but its possible, it does happen on a number of occasions, Shamonki said.

Shamonki says CBR tests for any bacterial contamination before storing the tissue and works with parents who have infected samples to discuss possibilities of being able to use the unit in the future.

A Cord Blood Registry worker processes cord blood for storage in the company's Tucson laboratory.(Photo: Amanda Morris)

Nobody knows when regenerative medicine applications for cord blood will become readily available as FDA-approved mainstreamtreatments. New applications for cord blood are being tested every year and new technologies to expand and utilize stem cells in cord blood are constantly being developed.

We dont really know what the limits are, but there are limits to what (umbilical cord blood stem cells) can do, Harris said.

Because cord blood banking is so new it has only been around since 1989 its unclear how long samples can be stored and remain effective.

According to Harris, cord blood samples can still work after being stored for about two decades.

We recently took one out that was 24, 25 years old, he said.

He speculates that properly stored cord blood samples could probably work throughout a person's lifetime, if not longer.

Buzan is aware that stem cells and cord blood treatments are still a new science with no guarantees, but she also believes in the treatments future potential.

Every month, she and her husband receive email updates from CBR that explain some of the new clinical trials and research discoveries involving cord blood.

To be honest the most exciting thing is the unknown the unknown of what the cord blood could do, what theyre looking into now, thats fascinating, she said. Im just so glad we live in a time that where this is available to use this was not an option for my parents or my grandparents.

Amanda Morris covers all things bioscience, which includeshealth care,technology, new researchand the environment. Send her tips, story ideas, or dog memes at amorris@gannett.com and follow her on Twitter @amandamomorris for the latest bioscience updates.

Independent coverage of bioscience in Arizona is supported by a grant from the Flinn Foundation.

Support local journalism.Subscribe to azcentral.com today.

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Cord blood banks sell parents on promising stem cell research, but with no guarantees - The Arizona Republic

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Procyon Technologies LLC and Novo Nordisk A/S to Collaborate on the Development of a Stem-Cell Based Therapy for Type 1 Diabetes – PRNewswire

Posted: December 9, 2020 at 1:59 am

TUCSON, Ariz., Dec. 8, 2020 /PRNewswire/ --Procyon Technologies LLC today announced that it has entered into an exclusive research collaboration and license agreement with Novo Nordisk A/S to develop an implantable cell encapsulation device to be used in Novo Nordisk's development of a novel therapy for Type 1 diabetes.

The collaboration brings together Procyon Technologies' expertise with development of oxygen enabled implantable cell encapsulation devices and Novo Nordisk's expertise in stem cell-derived insulin-secreting cells.

The partners will work together to further optimize the device and cells for clinical testing and accelerate the path to First Human Dose with the joint vision of delivering a functional cure for people living with Type 1 diabetes.

Under the terms of the agreement, Procyon Technologies, a startup founded to commercialize innovations developed at the University of Arizona College of Medicine Tucson, will receive an upfront license fee and will be eligible for further payments relating to preclinical, clinical and regulatory milestones. In addition, Procyon Technologies will receive tiered sales milestones and royalties on the annual net sales of the products resulting from the collaboration.

Novo Nordisk will be responsible for the development, manufacturing and commercialization of the products resulting from the collaboration for Type 1 diabetes.

The right cells and the right device

Type 1 diabetes is an autoimmune disease in which insulin-producing beta cells in the pancreas are mistakenly destroyed by the body's immune system. For people with Type 1 diabetes, life-long daily administration of insulin to control their blood sugar and constant blood glucose monitoring is the burden of reality.

"If we are able to offer a treatment that safely and effectively replaces the insulin-producing cells that people with Type 1 diabetes have lost, we could essentially offer them a functional cure for their disease," said Jacob Sten Petersen,DMSc, corporate vice president and head of stem cell research and development for Novo Nordisk.

Since 2008, Novo Nordisk has invested in human stem cell technology and worked on generating a protocol for stem cell-derived insulin producing islet-like clusters for the treatment of Type 1 diabetes.

But having the right cells is only half the solution; the cells also need to be protected from the recipient's immune system to avoid rejection, as well as from the autoimmunity of Type 1 diabetes.

For the last two decades, Procyon Technologies co-founder Klearchos Papas, PhD, a professor in the Department of Surgery and director of the Institute for Cellular Transplantation at the University of Arizona College of Medicine Tucson, has been working on a solution to the second part of that challenge.

"As a pancreas transplant surgeon, the idea of replacing beta cell function in a diabetic patient to prevent progression of diabetic complications makes perfect sense," said Michael M.I. Abecassis, MD, MBA, dean of the UArizona College of Medicine Tucson and professor of surgery and immunobiology. "Therefore, the notion of doing this without the need for major surgery and without the need for anti-rejection drugs by leveraging the assets of academia with those of industry represents the next frontier in curing Type 1 diabetes and preventing its complications."

With support from JDRF International and the National Institute of Diabetes and Digestive and Kidney Diseases, and utilizing key University of Arizona facilities and infrastructure (such as the BIO5 Institute device prototyping lab), Dr. Papas and his team developed oxygen enabled implantable immuno-isolation device technology with a focus on safety, practicality, and the maintenance of viability and functionality of encapsulated cells.

"We are delighted and excited to join forces with Novo Nordisk and provide the 'implantable encapsulation device' part of the functional cure for people suffering from Type 1 diabetes. Novo Nordisk is a leader in the development of stem cell-derived insulin producing islet-like clusters, has demonstrated strong commitment, and has the capacity, infrastructure and most importantly, the shared vision and interest in seeking to bring this functional cure to patients," said Dr. Papas.

"The combination of the implantable encapsulation device with islet-like clusters provides a unique opportunity to develop a novel cell therapy for diabetes. This announcement reinforces the value of JDRF in supporting science and technologies that can be further advanced in partnerships," said Esther Latres, PhD, assistant vice president of research at JDRF.

"Dr. Papas' work exemplifies our research mission in the Department of Surgery. The collaboration between our investigators and clinicians allows for the development of innovative, cutting-edge solutions to the clinical problems we treat every day," said Taylor S. Riall, MD, PhD, chair of the UArizona Department of Surgery. "The partnership between Procyon Technologies and Novo Nordisk represents the culmination of years of hard work and will revolutionize the care of people with Type 1 diabetes."

A therapeutic implant

The Procyon cell encapsulation device is a small, flat, thin, highly durable, flexible implantable chamber. It mitigates foreign body response, promotes the formation of vascular structures on its surface enabling the rapid diffusion of nutrients to the cells inside and the rapid absorption of insulin (or other therapeutic molecules) secreted by the encapsulated cells while providing a barrier protecting them from attacks by the body's immune system without the need for immunosuppressive drugs. The Procyon technology, designed with practical clinical use as a driver, includes integration of oxygen delivery to the implantable device, which enables tighter packing of cells while maintaining their viability and functionality.

About Procyon Technologies LLC:

Procyon Technologies LLC (https://procyon-technologies.com) was founded in Arizona in 2016. Klearchos Papas, PhD, Allison F. Corkey, MS, Thomas Loudovaris, PhD, and Robert C. Johnson, PhD, are co-founders and worked with Tech Launch Arizona, the University of Arizona commercialization arm, to protect the intellectual property and license the platform technology suitable for the implantation of a variety of therapeutic cells aimed at treating a number of disorders. In addition to being highly respected researchers in the field of diabetes and encapsulation therapy for decades, Dr. Johnson, a part-time research professor in the Department of Surgery at the University of Arizona, has had Type 1 diabetes for nearly 51 years and Dr. Loudovaris is the father of two children with the disease.

Contact:Allison F. Corkey[emailprotected]520-329-1425

SOURCE Procyon Technologies LLC

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Repairing the Brain With Stem Cells? A Conversation With Prof. Jack Price – Being Patient

Posted: December 9, 2020 at 1:59 am

Groundbreaking research in stem cells has propelled scientists understanding of neurodegenerative diseases, including Parksinsons. Could stem cell therapies one day help cure Alzheimers?

Clinical trials of stem cell therapies are now underway to repair the damaged cells of people with Parkinsons disease and age-related macular degeneration. Being Patient spoke with Jack Price, professor of developmental neurobiology at Kings College London and author of the book The Future of Brain Repair, about the potential and challenges of repairing the brain with stem cell therapy.

Being Patient: What is stem cell therapy?

Prof. Jack Price: Its the transplantation of stem cells, either directly into the brain or in a way that gives them access to the brain and influence the brain, to bring about a therapeutic effect.

Being Patient: Are there stem cells in the brain?

Prof. Jack Price: For many years, neuroscientists didnt think there were stem cells in the brain. We now know there are. We know about a population [of stem cells] thats become very important in our understanding of Alzheimers disease and in mood disorders like anxiety and depression. These are stem cells that are found in a part of the brain called the hippocampus.

But by and large, the brain doesnt have stem cells, unlike skin and other tissues in the body. The blood is the classic [example]: Theres a population of stem cells in the bone marrow that regenerates blood all the time.

Being Patient: What makes stem cells so special and why are they a focus of research?

Prof. Jack Price: The definition of stem cells is a population of cells that gives rise to other types of cells. In neural stem cells, precursor cells can make adult brain cells, nerve cells, glial cells, all the different cell types that make up the brain. If you have a disease like Alzheimers or any other neurodegenerative disease, where we know the key pathology is the loss of nerve cells, your brain doesnt normally have the ability to replace those lost brain cells. The idea was [that] if you put stem cells where the loss of brain cells has taken place, maybe those stem cells would replace the lost cells.

Being Patient: What is the potential of stem cell therapy in treating neurodegenerative diseases?

Prof. Jack Price: Theres a piece of absolutely brilliant stem cell science that was done by Shinya Yamanaka in Kyoto in 2006. He showed you could effectively take any cell through a very straightforward genetic manipulation that he discovered, [and] turn them into what we call pluripotent stem cells, which are cells that can make any cell type in the body. They also have an ability that other stem cells generally dont: They can build tissue. If you grow them in a little culture dish, they can start to make little pieces of brain called organoids or cerebroids. This was a groundbreaking technology.

In Parkinsons disease, theres enormous progress and clinical trials are underway now. We know more about the pathology of Parkinsons disease [than in Alzheimers]. The pathology of Alzheimers turns out to be quite complex, and weve had, over the years, quite a few ideas about how it worked. But [turning] those into actual therapies hasnt quite worked as we expected, and we keep having to go back and rethink whats going on in Alzheimers.

The pathology of Parkinsons disease is also difficult. Its not trivial. But at the same time, one thing is clear: a lot of the pathology is associated with the loss of a particular population of nerve cells the midbrain dopaminergic cells. We can start with these pluripotent stem cells and make them make precisely the right type of dopaminergic cell that we know is lost in Parkinsons disease.

This is built on 30 [to] 40 years of research of people trying to find exactly the right cell type to work [with] in Parkinsons disease. They had some early success and fell backwards. But this technology looks much more precise than everything anybodys ever tried before.

In age-related macular degeneration, the disease of the eye where you lose your retinal photoreceptors, there are very clever strategies now where people are using these pluripotent stem cells to make a thing called a retinal pigment epithelium. It lies behind the retina, but its what supports the photoreceptors. It turns out, thats what goes wrong in age-related macular degeneration.

Being Patient: Are there any stem cell therapy approved to treat brain disorders?

Prof. Jack Price: There are no licensed stem cell therapy for any brain disorders anywhere in the world for the simple reason [that] nobody has shown one works. There are a lot of stem cell clinics in the U.S. and somewhat fewer elsewhere who are offering cell therapies that are untested. Theyll put stem cells into you for any disorder youve got. Those cell therapies do not work.

A lot of genuine companies are trying to get these cell therapies to work in clinical trials and falling flat on their face quite often, despite their best efforts. 90% of clinical trials fail, and thats clinical trials of conventional drugs by drug companies that know what theyre doing.

What do you suppose is the chance with a stem cell therapy [that] we dont really understand how it works, [that] we dont quite know how to manufacture it properly, [and that] we dont quite know what cells we really want, of working? The chance is almost zero.

The interview has been edited for length and clarity.

Contact Nicholas Chan at nicholas@beingpatient.com

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How do you donate stem cells? Donating cells can help treat cancer, blindness and other conditions – heres how – The Scotsman

Posted: December 9, 2020 at 1:59 am

HealthA new stem cell treatment could restore eyesight in some people

Friday, 4th December 2020, 3:18 pm

Researchers discovered that the cells of damaged retinas could be repaired by injecting genetically modified stem cells into the eye.

The news comes as comedian Al Murray pushed for stem cell donors to come forward, ahead of a charity gig for blood cancer organisation DKMS.

Heres everything you need to know about the scientific discovery - and how you can donate your own stem cells to save the lives of people with blood cell diseases.

Stem cells are produced by bone marrow, and they have the ability to grow into different types of blood cells such as red and white blood cells and platelets.

A stem cell or bone marrow transplant replaces damaged blood cells with healthy ones and can be used to treat conditions affecting the blood cells, like leukaemia and lymphoma.

The transplant involves destroying the unhealthy blood cells and replacing them with the stem cells removed from the blood or bone marrow.

Often, stem cells are taken from one person - usually a close family member or a match with the same or similar tissue type - and they are transferred to the person that needs them.

How could they be used to treat vision damage?

Researchers in Barcelona recently discovered that modified stem cells could potentially help to cure problems with vision.

They found that the cells of damaged eye retinas send out a rescue signal to attract the stem cells that can repair damage.

Stem cells were genetically engineered to make them more sensitive to those signals.

The modified stem cells were transplanted back into mice and human tissue samples and the researchers found that they flocked to the retina cells in large numbers.

In turn, that kept the tissue of the retina alive and functioning.

The new technique is a breakthrough in stem cell research as it suggests stem cells could help to improve sight, and potentially could cure blindness in the future.

Retinal damage is currently incurable and can cause visual disabilities and blindness, especially in older people.

How can stem cells treat conditions?

Stem cells can already be used to treat a number of conditions where the bone marrow is damaged and unable to produce its own healthy blood cells.

Transplants can be used to treat people suffering from different forms of cancer, with someone elses tem cells replacing the patients blood cells that are damaged or destroyed.

Conditions that stem cell transplants can treat include leukemia and lymphoma, which are cancers affecting white blood cells, myeloma, which affects plasma cells, severe aplastic anaemia (bone marrow failure), and other blood disorders.

A stem cell transplant will usually only be carried out if other treatments have been exhausted, but it could save someones life.

How can I donate stem cells?

When its not possible to use someones own stem cells to treat their condition, they need to come from a donor.

However, to improve the chances of the transplant being successful, the donated cells need to have a very similar genetic marker to the patients.

As the number of donors has recently decreased, charities are urgently encouraging healthy people to donate stem cells.

You are able to register to be a donor on the NHS Blood and Transplant website.

The Anthony Nolan charity also takes sign ups, and is specifically looking for younger donors between age 16 and 30.

You will be asked to fill out an application form and will be sent a swab pack so you can be added to the register.

If you ever come up as a match for a patient, you will be contacted by the charity.

Even if you cant join the register, you can donate to Anthony Nolan to help to grow the stem cell register.

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Genetic engineering transformed stem cells into working mini-livers that extended the life of mice with liver disease – The Conversation US

Posted: December 9, 2020 at 1:59 am

Takeaways

Scientists have made progress growing human liver in the lab.

The challenge has been to direct stems cells to grow into a mature, functioning adult organ.

This study shows that stem cells can be programmed, using genetic engineering, to grow from immature cells into mature tissue.

When a tiny lab-grown liver was transplanted into mice with liver disease, it extended the lives of the sick animals.

Imagine if researchers could program stem cells, which have the potential to grow into all cell types in the body, so that they could generate an entire human organ. This would allow scientists to manufacture tissues for testing drugs and reduce the demand for transplant organs by having new ones grown directly from a patients cells.

Im a researcher working in this new field called synthetic biology focused on creating new biological parts and redesigning existing biological systems. In a new paper, my colleagues and I showed progress in one of the key challenges with lab-grown organs figuring out the genes necessary to produce the variety of mature cells needed to construct a functioning liver.

Induced pluripotent stem cells, a subgroup of stem cells, are capable of producing cells that can build entire organs in the human body. But they can do this job only if they receive the right quantity of growth signals at the right time from their environment. If this happens, they eventually give rise to different cell types that can assemble and mature in the form of human organs and tissues.

The tissues researchers generate from pluripotent stem cells can provide a unique source for personalized medicine from transplantation to novel drug discovery.

But unfortunately, synthetic tissues from stem cells are not always suitable for transplant or drug testing because they contain unwanted cells from other tissues, or lack the tissue maturity and a complete network of blood vessels necessary for bringing oxygen and nutrients needed to nurture an organ. That is why having a framework to assess whether these lab-grown cells and tissues are doing their job, and how to make them more like human organs, is critical.

Inspired by this challenge, I was determined to establish a synthetic biology method to read and write, or program, tissue development. I am trying to do this using the genetic language of stem cells, similar to what is used by nature to form human organs.

I am a researcher specializing in synthetic biology and biological engineering at the Pittsburgh Liver Research Center and McGowan Institute for Regenerative Medicine, where the goals are to use engineering approaches to analyze and build novel biological systems and solve human health problems. My lab combines synthetic biology and regenerative medicine in a new field that strives to replace, regrow or repair diseased organs or tissues.

I chose to focus on growing new human livers because this organ is vital for controlling most levels of chemicals like proteins or sugar in the blood. The liver also breaks down harmful chemicals and metabolizes many drugs in our body. But the liver tissue is also vulnerable and can be damaged and destroyed by many diseases, such as hepatitis or fatty liver disease. There is a shortage of donor organs, which limits liver transplantation.

To make synthetic organs and tissues, scientists need to be able to control stem cells so that they can form into different types of cells, such as liver cells and blood vessel cells. The goal is to mature these stem cells into miniorgans, or organoids, containing blood vessels and the correct adult cell types that would be found in a natural organ.

One way to orchestrate maturation of synthetic tissues is to determine the list of genes needed to induce a group of stem cells to grow, mature and evolve into a complete and functioning organ. To derive this list I worked with Patrick Cahan and Samira Kiani to first use computational analysis to identify genes involved in transforming a group of stem cells into a mature functioning liver. Then our team led by two of my students Jeremy Velazquez and Ryan LeGraw used genetic engineering to alter specific genes we had identified and used them to help build and mature human liver tissues from stem cells.

The tissue is grown from a layer of genetically engineered stem cells in a petri dish. The function of genetic programs together with nutrients is to orchestrate formation of liver organoids over the course of 15 to 17 days.

I and my colleagues first compared the active genes in fetal liver organoids we had grown in the lab with those in adult human livers using a computational analysis to get a list of genes needed for driving fetal liver organoids to mature into adult organs.

We then used genetic engineering to tweak genes and the resulting proteins that the stem cells needed to mature further toward an adult liver. In the course of about 17 days we generated tiny several millimeters in width but more mature liver tissues with a range of cells typically found in livers in the third trimester of human pregnancies.

Like a mature human liver, these synthetic livers were able to store, synthesize and metabolize nutrients. Though our lab-grown livers were small, we are hopeful that we can scale them up in the future. While they share many similar features with adult livers, they arent perfect and our team still has work to do. For example, we still need to improve the capacity of the liver tissue to metabolize a variety of drugs. We also need to make it safer and more efficacious for eventual application in humans.

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Our study demonstrates the ability of these lab livers to mature and develop a functional network of blood vessels in just two and a half weeks. We believe this approach can pave the path for the manufacture of other organs with vasculature via genetic programming.

The liver organoids provide several key features of an adult human liver such as production of key blood proteins and regulation of bile a chemical important for digestion of food.

When we implanted the lab-grown liver tissues into mice suffering from liver disease, it increased the life span. We named our organoids designer organoids, as they are generated via a genetic design.

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City of Hope Doctors Present Innovative Therapies to Better Treat Blood Cancers at American Society of Hematology Virtual Conference – BioSpace

Posted: December 9, 2020 at 1:59 am

Dec. 8, 2020 16:00 UTC

DUARTE, Calif.--(BUSINESS WIRE)-- City of Hope doctors participated in research presented at the American Society of Hematology (ASH) virtual meeting, Dec. 5 to 8, that are helping advance the treatment of blood cancers, including one study which demonstrated allogeneic stem cell transplants do have a survival benefit for older adults with myelodysplastic syndromes (MDS) compared with current standard of care.

The study is the largest and most definitive trial to demonstrate the benefits of an allogeneic stem cell transplantation for older adults with MDS, and is just one of numerous studies that City of Hope doctors help lead with the aim of finding more effective treatments of various blood cancers.

This years ASH conference truly showcases City of Hopes leadership in finding more effective treatments for blood cancers, said Stephen J. Forman, M.D., director of City of Hopes Hematologic Malignancies Research Institute. Whether its finding innovative treatments to make it possible for more older adults with cancer to receive stem cell transplants, or pursuing therapies that are more effective with fewer side effects, City of Hope doctors continue to lead innovative research in blood cancers and other hematological malignancies.

City of Hope doctors are leading novel clinical trials for patients with leukemia, lymphoma and other blood cancers.

Multicenter clinical trial led by City of Hope makes stem cell transplant possible for older adults with myelodysplastic syndromes

Allogeneic hematopoietic cell transplantation, or stem cell/bone marrow transplants, for blood cancers that have recurred or are difficult to treat can put the disease into long-term remission and provide a potential cure. The therapy establishes a new, disease-free blood and immune system by transplanting healthy blood stem cells from a donor into a cancer patient after destroying the patients unhealthy bone marrow.

City of Hope and other institutions started this therapy in 1976, primarily for younger patients with blood cancers. The therapy involves using high-dose chemotherapy and/or radiotherapy to make room for a person to receive new stem cells; serious side effects can also occur after transplant. Because of these and other considerations, for many years, older adults with blood cancers have not been considered for transplants.

City of Hope has been leading the way to make transplants possible for more older adults with various cancers.

A new study presented at ASH demonstrates transplants are now a possibility and beneficial for patients with myelodysplastic syndromes (MDS). Approximately 13,000 people in the United States each year are diagnosed with MDS, an umbrella term describing several blood disorders that begin in the bone marrow.

Co-led by City of Hopes Ryotaro Nakamura, M.D., director of City of Hopes Center for Stem Cell Transplantation, the study is the largest and first trial to demonstrate the benefits of an allogeneic stem cell transplantation for older adults with MDS as opposed to the standard of care currently provided to these patients. The multicenter trial for patients aged 50 to 75 with serious MDS compared how long transplant patients survived with those who didnt receive a transplant, as well as disease progression and quality of life. The transplant therapy used reduced-intensity conditioning, which delivers less chemotherapy and radiation before transplant and relies more on the anti-tumor effects of the therapy.

Between 2014 and 2018, the study enrolled 384 participants at 34 cancer centers nationwide. It included 260 patients who were able to find a donor for a transplant, as well as 124 patients who did not find a donor for a transplant.

After three years, nearly 48% of MDS patients who found a donor for transplant had survived compared with about 27% of those patients who didnt have a donor for transplant and received current hypomethylating therapy, a type of chemotherapy that is current standard of care for MDS. Leukemia-free survival which is relevant because myelodysplastic syndrome can develop into leukemia was also greater in transplant recipients after three years nearly 36% compared with about 21% for those who did not have a transplant.

There was a large and significant improvement in survival for patients who had a transplant, Nakamura said. The benefit margin in overall survival was over 20% (21.3%) for patients who had a transplant.

In addition, quality of life was the same for both transplant and nontransplant patients. There were no clinically significant differences when taking such measurements as physical and mental competency scores.

This is an extremely exciting study because it provides evidence that stem cell transplant is highly beneficial for older patients with serious MDS and will likely be practice-changing for this group, Nakamura said. Before, many doctors wouldnt even consider a transplant for this group of patients, but our study demonstrates that these patients should be evaluated for a transplant, which could potentially provide a cure for their disease.

The trial is part of Blood and Marrow Transplant Clinical Trials Network, which was established with support from the National Heart, Lung, and Blood Institute and National Cancer Institute, because of a critical need for multi-institutional clinical trials focused directly on improving survival for patients undergoing hematopoietic cell transplantation.

Updated results from a study of a potential new CAR T cell therapy, liso-cel, for relapsed/refractory chronic lymphocytic leukemia

Patients with relapsed or difficult-to-treat chronic lymphocytic leukemia/small lymphocytic leukemia continue to do well 24 months after receiving lisocabtagene maraleucel (liso-cel) chimeric antigen receptor (CAR) T cells, according to Tanya Siddiqi, M.D., director of City of Hopes Chronic Lymphocytic Leukemia (CLL) Program, which is part of the Toni Stephenson Lymphoma Center. She presented these findings during the 2020 ASH annual meeting virtual conference.

Overall, 23 and 22 patients were evaluated for safety and efficacy in this phase 1 trial, respectively. Their median age was 66 and they had received a median of four prior therapies; all patients had received prior ibrutinib, which is one of the standard of care drugs for CLL.

The overall response rate, or patients whose CLL diminished after liso-cel CAR T cell therapy, was 82%, and 45% of patients also had complete responses, or remissions.

After 15 months of treatment, 53% of patients maintained their responses to the therapy, and six patients continued to be in remission. After 18 months, 50% of patients maintained their response, and there were five remissions. All seven patients who completed the 24-month study maintained their response. Median progression-free survival, or the amount of time the cancer did not worsen during and after treatment, was 18 months.

As early as 30 days after receiving liso-cel, about 75% of 20 patients evaluated for the therapys efficacy had undetectable minimal residual disease (MRD, or no detectable traces of cancer) in the blood and 65% had undetectable MRD in the marrow.

These are remarkable results for a group of patients that prior to this CAR T treatment had no good treatment options if they had already progressed on novel targeted therapies like ibrutinib and venetoclax, Siddiqi said. Liso-cel is providing new hope for CLL patients, and the remissions are also long lasting with few serious side effects.

Because of its safety and effectiveness in clinical trials, liso-cel, which targets the CD19 protein on cancer cells, may soon receive approval from the Food and Drug Administration as a commercial therapy for relapsed or refractory B cell lymphoma. City of Hope is also taking part in the phase 2 trial of liso-cel in CLL patients.

Consolidation treatment with brentuximab vedotin/nivolumab after auto stem cell transplant for relapsed/refractory Hodgkin lymphoma patients leads to 18-month progression free-survival

Patients who have Hodgkin lymphoma that has not been cured by initial treatment will usually receive more chemotherapy and an autologous hematopoietic cell transplant. But even after a stem cell transplant, recurrence of the lymphoma is possible.

This multicenter phase 2 clinical trial, led by City of Hope, examined whether treating patients with brentuximab vedotin (BV), an antibody-based treatment that targets delivery of chemotherapy only to Hodgkin lymphoma cells, and nivolumab, which works by blocking the PD-1 immune checkpoint pathway that Hodgkin lymphoma hijacks to evade the immune system, was safe and effective as consolidation to prevent disease recurrence after transplant in patients with high-risk Hodgkin lymphoma.

Alex Herrera, M.D., assistant professor in City of Hope's Department of Hematology & Hematopoietic Cell Transplantation, discussed 19-month progression-free survival for trial participants, as well as overall survival, safety and response rates during ASH.

Fifty-nine patients were enrolled in the trial. Patients received the consolidation treatment starting a median of 54 days after transplant, and received a median of eight cycles of the therapy. The 19-month progression-free survival in patients was 92%, and overall survival in patients was 98%. Only three patients relapsed after receiving BV and nivolumab consolidation after transplant, and one patient passed away due to PCP pneumonia unrelated to the study treatment.

The most common sides effects related to the treatment were peripheral neuropathy (51%), neutropenia (42%), fatigue (37%) and diarrhea (29%).

Using brentuximab vedotin and nivolumab after transplant is a promising approach for preventing relapse of Hodgkin lymphoma after transplant that merits further study, Herrera said.

City of Hope doctors published research on innovative approaches against graft-versus-host-disease

Historically, a bone marrow/stem cell transplant is more likely to be effective if patients have a donor who is a 100% match, or as close to that as possible. Finding that perfect match is more difficult for African Americans, Latinos, Asian Americans and other ethnic groups as bone marrow donor registries are still trying to increase the number of non-white donors.

Transplant doctors are also looking for ways to make the transplant more effective if a perfect match cant be found; donors who are not a 100% or close match are referred to as mismatched unrelated. One major barrier to these transplants being effective is a condition known as graft-versus-host-disease (GVHD). The condition, which is more common in transplants involving mismatched donors, is caused by donated cells that recognize the recipient's cells as foreign and attack them, damaging the skin, eyes, lungs, liver and digestive tract.

In order to help prevent GVHD, therapies can be given to patients after transplant. A prospective clinical trial at City of Hope examined whether using cyclophosphamide after an infusion of stem cells could prevent GVHD.

Thirty-eight patients were enrolled in the trial, which is the first to examine the use of cyclophosphamide in transplants with a mismatched unrelated donor.

With a median follow-up period of 18 months, 87% of patients had survived, and the majority did not relapse or develop severe GVHD.

During the first 100 days post-transplant, acute GVHD incidence was around 50%; most cases were mild to moderate while severe GVHD was only 15%. A year after transplant, 52% of patients had some form of chronic GVHD, but only 3% had moderate or severe chronic GVHD.

The trial also examined toxicities, infections and immune system recovery after the transplant.

Our study showed that patients who received a transplant from a mismatched unrelated donor using post-transplant cyclophosphamide had a comparable outcome to what we see in matched donor transplants with few cases of serious GVHD cases, said Monzr Al Malki, M.D., associate clinical professor of City of Hopes Department of Hematology & Hematopoietic Cell Transplantation and director of unrelated donor BMT and haploidentical transplant programs. Our data support further development of this therapy in transplant patients who would otherwise have no suitable donors and limited treatment options.

City of Hopes Anthony Stein, M.D., also led a pilot trial that examined whether a new treatment approach may reduce the rate of GVHD in patients with acute myelogenous leukemia (AML) who have received an allogeneic hematopoietic cell transplant. Although a transplant can put AML into remission, GVHD remains the main serious complication after transplant, impacting a patients quality of life and increasing health care costs.

Eighteen patients between the ages of 18 and 60 enrolled in the trial. Each patient received a novel conditioning regimen of total marrow and lymphoid irradiation, which targets a patients marrow and lymph nodes while reducing radiation to other parts of the body, and cyclophosphamide, a therapy that suppresses the immune system. Tacrolimus was also provided to patients.

Radiation was delivered twice daily on the fourth day before transplant and on the day of transplant without chemotherapy. Cyclophosphamide was given to patients on the third and fourth day after transplant.

There were mild to moderate toxicities. Acute GVHD developed in two patients and only one patient developed the most serious GVHD. Five patients developed mild chronic GVHD. Nearly 60% of patients had not developed GVHD or the condition had not worsened after a year.

After a year, all patients had survived, and 83% had not relapsed. After two years, nearly 86% of patients had survived, and the relapse number remained the same.

The therapeutic approach did not interfere with the transplant process as all patients engrafted, or the donors cells started to produce bone marrow and immune cells.

This is welcome news for AML patients who receive an allogeneic transplant and are concerned about developing GVHD, said Stein, associate director of City of Hope's Gehr Family Center for Leukemia Research. Our study demonstrated that using this new combination of therapies is safe and feasible and does not interfere with the engraftment process.

In addition, after a year, patients in this trial were no longer taking immunosuppressive therapy and had an improved quality of life, Stein said. He added that because many of the patients didnt have GVHD, health care costs after a year were also lower than if patients required treatment for the condition.

City of Hope now plans to start a larger phase 2 trial using this treatment approach.

Bispecific antibodies continue to show promise against blood cancers

Mosunetuzumab is a promising new immunotherapy for the treatment of relapsed/refractory non-Hodgkin lymphoma (NHL) that recently received breakthrough therapy designation from the Food and Drug Administration. The designation is intended to expedite the development and review of drugs for serious or life-threatening diseases.

Elizabeth Budde, M.D., Ph.D., assistant professor in City of Hope's Department of Hematology & Hematopoietic Cell Transplantation, is leading clinical trials that are showing how well mosunetuzumab works against NHL. At this years ASH, one trial discussed is how the therapy is working for patients with follicular lymphoma.

Mosunetuzumab is a bispecific antibody targeting both CD3 (a protein found on the surface on T cells) and CD20 on the surface of B cells. The therapy redirects T cells to engage and eliminate malignant B cells.

Sixty-two patients, ranging in age from 27 to 85 years old, were enrolled in the trial for follicular lymphoma. They received intravenous doses of mosunetuzumab.

Sixty-eight percent of the patients responded to the therapy, and 50% had a complete response, or went into remission. Consistent complete response rates occurred even in patients with double refractory disease and patients who received prior CAR T cell therapy. Median duration of response was approximately 20 months, and media progression free survival was nearly one year.

Side effects were reported in 60 patients with serious adverse effects in 22 patients. The most frequently reported serious side effects were hypophosphatemia, an electrolyte disorder, and neutropenia, a condition caused by low numbers of white blood cells. Fourteen patients experienced cytokine release syndrome, but none required extensive treatment for it.

Neurological side effects included headache, insomnia and dizziness.

Patients in this trial had high response rates and their disease remained in control for a year, Budde said. This is remarkable because many patients were no longer responding to other therapies.

About City of Hope

City of Hope is an independent biomedical research and treatment center for cancer, diabetes and other life-threatening diseases. Founded in 1913, City of Hope is a leader in bone marrow transplantation and immunotherapy such as CAR T cell therapy. City of Hopes translational research and personalized treatment protocols advance care throughout the world. Human synthetic insulin and numerous breakthrough cancer drugs are based on technology developed at the institution. A National Cancer Institute-designated comprehensive cancer center and a founding member of the National Comprehensive Cancer Network, City of Hope has been ranked among the nations Best Hospitals in cancer by U.S. News & World Report for 14 consecutive years. Its main campus is located near Los Angeles, with additional locations throughout Southern California. For more information about City of Hope, follow us on Facebook, Twitter, YouTube or Instagram.

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Better education needed to give patients improved understanding of gene therapies, new review highlights – University of Birmingham

Posted: December 9, 2020 at 1:59 am

Older, male patients with more severe underlying conditions and a greater risk of death tended to be more accepting of new approaches such as stem cell research

A new review of research bringing together patient, carer and public views of cell and gene therapies has highlighted a need for appropriate education to better inform people including how clinical trials work and the risks and benefits of various treatments.

Over the last decade, new cell, gene and tissue-engineered therapies have been developed to treat various cancers, inherited diseases and some chronic conditions. They offer opportunities for the treatment of disease and injury, to restore function, and in some cases offer cures. In response the NHS Advanced Therapies Treatment Centres (ATTCs) were set up to bring together the health service, academia and industry to address the unique and complex challenges of bringing these therapies to patients.

Led by experts from the Centre for Patient Reported Outcome Research (CPROR) at the University of Birmingham and the Midlands and Wales ATTC (MW-ATTC), the review, funded by a MW-ATTC grant from UK Research and Innovation is the first of its kind and the first to consider both patient and public opinions of cell and gene therapies. Examining 35 studies, the majority of which were published between 2015 and 2020, analysis showed that a lack of understanding of the aims of clinical trials and overestimation of the potential benefits of cell and gene therapy were common among both patients and the general public. Patients were generally of the opinion that more information about participating in clinical trials is vital to enable them to make informed assessment of potential risks and benefits.

Older, male patients with more severe underlying conditions and a greater risk of death tended to be more accepting of new approaches such as stem cell research and generally, while views of therapies varied among patients, the provision of adequate information increased acceptance.

Interestingly the review also found that patients considered their clinicians to be the most trustworthy source of information which would suggest that patients would approach and discuss these treatments with their physicians. However, researchers found that this might not always be the case due to a number of reasons including the perception that clinicians do not always approve of cell and gene therapies and may try to discourage them from pursuing treatment and may not have enough knowledge of the field to provide adequate advice.

Lead author Dr Olalekan Lee Aiyegbusi, Co-Deputy Director of the Centre for Patient Reported Outcomes Research (CPROR) said: The findings from this research are intended to inform the patient engagement work of the ATTCs. We hope that by highlighting various issues, efforts will be made to correct misconceptions, and improve the awareness of patients and the public about the potential benefits and risks associated with cell and gene therapies.

It is important that the public and patients are aware of these therapies, understand the issues involved, and can contribute to the ongoing debates. A high level of awareness will also enhance patients ability to make informed decisions about participating in clinical trials and routine administration of cell and gene therapies.

The full paper Patient and public perspectives on cell and gene therapies: a systematic review was published today (Tuesday 8 December 2020) in Nature Communications.

ENDS

For more information please contact Sophie Belcher, Communications Manager, University of Birmingham, on +44 7815607157. Alternatively, contact the Press Office out of hours on +44 (0)7789 921165.

DOI: 10.1038/s41467-020-20096-1.Full paper: http://www.nature.com/ncomms

The University of Birmingham is ranked amongst the worlds top 100 institutions, and its work brings people from across the world to Birmingham, including researchers and teachers and more than 6,500 international students from nearly 150 countries.

About the Midlands and Wales ATTC (MW-ATTC)

The 9M Midlands and Wales Advanced Therapy Treatment Centre (MW-ATTC) is one of three national Innovate UK funded centres whose goal is to accelerate the delivery of advanced therapies.

It is a regional network spanning the Midlands & Wales comprising a large consortium of industry, healthcare and university partners with expertise in advanced therapy manufacturing including academic and commercial partners, logistics companies, specialists in clinical trial delivery and teams focussed on IT logistics solutions and health economics.

The aim of the MW-ATTC is to enable UK advanced therapy companies to reach the clinical market, whilst simultaneously building clinical capacity regionally to deliver these breakthrough therapies to patients.

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Magenta Therapeutics Announces Commencement of First Phase 2 Clinical Trial of MGTA-145 for Stem Cell Mobilization, Oral Presentation of MGTA-145…

Posted: December 9, 2020 at 1:59 am

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Magenta Therapeutics (NASDAQ: MGTA), a clinical-stage biotechnology company developing novel medicines to bring the curative power of stem cell transplant to more patients, today announced final clinical results from its earlier completed Phase 1 clinical trial as well as development updates for its MGTA-145 stem cell mobilization therapy, including commencement of enrollment in a Phase 2 clinical trial in multiple myeloma, and its plans for a Phase 2 clinical trial in allogeneic stem cell transplant for patients with acute myeloid leukemia (AML), acute lymphocytic leukemia (ALL) and myelodysplastic syndrome (MDS). The company also previously announced a clinical collaboration with bluebird bio to evaluate MGTA-145 for mobilizing and collecting stem cells in adults and adolescents with sickle cell disease (SCD). Additional preclinical results were also presented at the 62nd American Society of Hematology (ASH) Annual Meeting and Exposition, taking place virtually from December 5-8, 2020, on the Magenta conditioning platform, including MGTA-117 program, which is a targeted antibody-drug conjugate (ADC) to prepare patients for stem cell transplant.

MGTA-145 Advancement to Phase 2 Development in Blood Cancers

The company announced that enrollment has started and is ongoing in a Phase 2 clinical trial of MGTA-145, used in combination with plerixafor, to mobilize and collect stem cells for autologous stem cell transplantation of multiple myeloma patients at Stanford University. Magenta expects that this trial will provide patient-level data on stem cell mobilization and collection, characteristics of the mobilized graft and engraftment in patients with multiple myeloma.

Additionally, through a collaboration with the National Marrow Donor Program/Be The Match, a global leader in facilitating allogeneic hematopoietic stem cell transplantation, Magenta plans to initiate a Phase 2 clinical trial in early 2021 using MGTA-145 to mobilize and collect stem cells from allogeneic donors for transplant in patients with AML, ALL and MDS. Allogeneic stem cell transplant provides a potentially curative therapeutic option for patients with these diseases. This clinical trial will evaluate stem cell mobilization, collection, cell quality, engraftment and the potential for reduced Graft-versus-Host Disease (GvHD), which is of particular importance in the allogeneic transplant setting.

MGTA-145 in Sickle Cell Disease

Magenta Therapeutics recently announced an exclusive clinical collaboration with bluebird bio to evaluate the utility of MGTA-145, in combination with plerixafor, for the mobilization and collection of stem cells in adults and adolescents with SCD.

The data from this clinical trial could provide proof-of-concept for MGTA-145, in combination with plerixafor, as the preferred mobilization regimen for patients with SCD. bluebird bios experience with plerixafor as a mobilization agent in SCD aligns with Magentas combination therapy approach, utilizing MGTA-145 plus plerixafor with potential for safe, rapid and reliable mobilization of sufficient quantities of high-quality stem cells to improve outcomes associated with stem cell transplantation.

MGTA-145 Presentations at ASH

Magenta presented final clinical data from its MGTA-145 stem cell mobilization Phase 1 clinical trial in healthy volunteers at the ASH Annual Meeting. All primary and secondary endpoints were met in the study completed earlier this year.

The results demonstrate that a single dose of MGTA-145, in combination with plerixafor, rapidly and reliably mobilized high numbers of stem cells in a single day without the need for G-CSF for potential use in diseases that can benefit from autologous and/or allogeneic stem cell transplantation. The additional data also offer further confirmation that MGTA-145, in combination with plerixafor, was well tolerated and provides a rapid and reliable method to obtain large numbers of hematopoietic stem cells. Transplant of these cells in preclinical models resulted in enhanced, durable engraftment, in addition to highly immunosuppressive properties, leading to reduced GvHD.

Results from this study provide a robust dataset and proof of concept that MGTA-145, in combination with plerixafor, provides rapid and robust mobilization of stem cells and that these cells have better engraftment potential, are able to be gene modified and engraft and reduce GvHD in preclinical models compared to cells mobilized with other available agents. The data reinforce the availability of compelling opportunities for development in both the autologous and allogeneic transplant settings, said John Davis Jr., M.D., M.P.H., M.S., Head of Research & Development and Chief Medical Officer, Magenta Therapeutics.

The data were presented by Steven M. Devine, MD, Chief Medical Officer of the National Marrow Donor Program/Be The Match and Associate Scientific Director of the CIBMTR (Center for International Blood and Marrow Transplant Research).

Conditioning Program (MGTA-117 and CD45-ADC) Presentations at ASH

Magenta also provided updates on its conditioning platform at the ASH Annual Meeting, including MGTA-117 and CD45-ADC programs. Preclinical data from a study of MGTA-117 demonstrate that it is an effective, potent conditioning agent for transplant with anti-leukemic activity, significantly decreasing tumor burdens, leading to delayed tumor growth and increased median survival rates in animal models of AML. Ongoing GLP toxicology and GMP manufacturing progress continue to be supportive of advancing MGTA-117 towards an IND filing in AML and MDS.

Additionally, preclinical data from a study of Magentas CD45-ADC, a CD45-targeted conditioning agent designed to remove the cells that cause autoimmune diseases to enable curative immune reset, demonstrated the ability to achieve successful outcomes as a single agent in the most challenging disease model through fully mismatched allogeneic hematopoietic stem cell transplant, where only radiation or combinations of toxic chemotherapies are available, potentially providing patients the option of a reduced toxicity conditioning regimen. The company continues to evaluate this program preclinically.

About MGTA-145

MGTA-145 is being developed in combination with plerixafor to harness complementary chemokine mechanisms to mobilize hematopoietic stem cells for collection and transplantation. This new combination has the potential to be the preferred mobilization regimen for rapid and reliable mobilization and collection of hematopoietic stem cells to improve outcomes in autologous and allogeneic stem cell transplantation, which can rebuild a healthy immune system for patients with blood cancers, genetic diseases and autoimmune disorders.

MGTA-145 has the potential to replace the current standard of care for patients and allogeneic donors who currently rely on the use of granulocyte-colony stimulating factor (G-CSF) alone or in combination with plerixafor, which can take up to five days or longer to mobilize sufficient numbers of stem cells, often resulting in significant bone pain and other side effects.

About Magenta Therapeutics

Magenta Therapeutics is a clinical-stage biotechnology company developing medicines to bring the curative power of immune system reset through stem cell transplant to more patients with blood cancer, genetic diseases and autoimmune diseases. Magenta is combining leadership in stem cell biology and biotherapeutics development with clinical and regulatory expertise, a unique business model and broad networks in the stem cell transplant world to revolutionize immune reset for more patients.

Magenta is based in Cambridge, Mass. For more information, please visit http://www.magentatx.com.

Follow Magenta on Twitter: @magentatx.

Forward-Looking Statement

This press release may contain forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995 and other federal securities laws. The use of words such as may, will, could, should, expects, intends, plans, anticipates, believes, estimates, predicts, projects, seeks, endeavor, potential, continue or the negative of such words or other similar expressions can be used to identify forward-looking statements. The express or implied forward-looking statements included in this press release are only predictions and are subject to a number of risks, uncertainties and assumptions, including, without limitation risks set forth under the caption Risk Factors in Magentas Annual Report on Form 10-K filed on March 3, 2020, as updated by Magentas most recent Quarterly Report on Form 10-Q and its other filings with the Securities and Exchange Commission. In light of these risks, uncertainties and assumptions, the forward-looking events and circumstances discussed in this press release may not occur and actual results could differ materially and adversely from those anticipated or implied in the forward-looking statements. You should not rely upon forward-looking statements as predictions of future events. Although Magenta believes that the expectations reflected in the forward-looking statements are reasonable, it cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur. Moreover, except as required by law, neither Magenta nor any other person assumes responsibility for the accuracy and completeness of the forward-looking statements included in this press release. Any forward-looking statement included in this press release speaks only as of the date on which it was made. We undertake no obligation to publicly update or revise any forward-looking statement, whether as a result of new information, future events or otherwise, except as required by law.

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Magenta Therapeutics Announces Commencement of First Phase 2 Clinical Trial of MGTA-145 for Stem Cell Mobilization, Oral Presentation of MGTA-145...

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Magenta Therapeutics Announces Commencement of First Phase 2 Clinical Trial of MGTA-145 for Stem Cell Mobilization, Oral Presentation of MGTA-145…

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