Platelet BioGenesis Receives Contract Worth Up to $56 Million from the Biomedical Advanced Research and Development Authority (BARDA) to Develop Human…

CAMBRIDGE, Mass.--(BUSINESS WIRE)--

Funding will support clinical development of stem cell-derived platelet technology platform

Platelet BioGenesis, Inc. (PBG), the leader in stem cell-derived, on-demand human platelets (PLTs+) and genetically engineered platelet-based therapeutics, announced today it was awarded a contract worth $5 million, with the potential to reach $56 million total with options, by the Biomedical Advanced Research and Development Authority (BARDA), an agency of the US government's Department of Health and Human Services' Office of the Assistant Secretary for Preparedness and Response. PBG will use the funding to develop and establish donor-independent platelets as a medical countermeasure for treating victims of a nuclear or radiological event.

As part of national biodefense preparedness, BARDA has prioritized the development and procurement of therapies for trauma resulting from chemical, biological, radiological and nuclear defense threats, including exposure to high doses of radiation. BARDA, with its extensive experience working with organizations focused on blood-related therapies, has evaluated PBGs technology platform and determined that donor-independent platelets could be a critical medical countermeasure in case of a national threat.

This is a significant milestone for PBG and a highly valuable and timely validation of the groundbreaking work of our scientific founder, Dr. Jonathan Thon, and the research, development and manufacturing teams at PBG, allowing us to use human induced pluripotent stem cells to manufacture platelets on-demand, said Sam Rasty, Ph.D., President and CEO of Platelet BioGenesis. With BARDAs expertise in the development of blood-related therapies, their decision to award us this significant contract will further bolster the companys resources to advance this technology into the clinic. The funding will not only help bring our unique donor-independent platelets to patients as a medical countermeasure but will also expedite the advancement of our broader donor-independent PLTs+ platform.

In a radiological or nuclear emergency, impacted communities will face a significant blood product shortage, explained BARDA Director Rick Bright, Ph.D. We are exploring donor-independent platelet technology to increase surge capacity within the blood industry. Our nation must find innovative ways to make essential blood products available to save lives in any type of mass casualty incident.

PBGs research, development and manufacturing activities under the contract will specifically focus on the development of PLTs+ for the treatment of thrombocytopenia induced by exposure to nuclear radiation. In addition to the funding, BARDA will provide a comprehensive, integrated portfolio approach through mentorship, the facilitation of future partnerships and the enablement of government collaborations with agencies such as the FDA.

About Platelet BioGenesis

Platelet BioGenesis (PBG) has created the only platform that can generate human platelets at scale. The stem cell-derived, on-demand platelets will be the first donor-independent source of platelets to address the chronic shortage worldwide. The company is also developing genetically engineered platelet-based therapeutics, a new treatment modality for cancer and other life-threatening diseases. PBGs platform is patented and cGMP-compliant. The company was spun out of Harvard University and has received venture funding from Qiming Venture Partners USA, Ziff Capital Partners and other investors and obtained grant funding from the Massachusetts Life Sciences Center, the National Institutes of Health and the U.S. Department of Defense. Learn more at plateletbiogenesis.com and follow us on Twitter @plateletbio.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191007005149/en/

Read more:
Platelet BioGenesis Receives Contract Worth Up to $56 Million from the Biomedical Advanced Research and Development Authority (BARDA) to Develop Human...

Researchers identify molecular process that could accelerate recovery from nerve injuries – UCLA Newsroom

Twenty million Americans suffer from peripheral nerve injuries, which can be caused by traumas such as combat wounds and motorcycle crashes as well as medical disorders including diabetes. These injuries can have a devastating impact on quality of life, resulting in loss of sensation, motor function and long-lasting nerve pain. The body is capable of regenerating damaged nerves, but this process is slow and incomplete.

Now, researchers at theEli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLAhave discovered a molecular process that controls the rate at which nerves grow both during embryonic development and recovery from injury throughout life.

The study, led by senior author Samantha Butler and published in the Journal of Neuroscience, used experiments with mice to show that it is possible to accelerate peripheral nerve growth by manipulating this molecular process. The finding could inform the development of therapies that reduce the time it takes for people to recover from nerve injuries.

The human bodys nervous system is comprised of two components: the central nervous system, which includes the brain and spinal cord; and the peripheral nervous system, which encompasses all other nerves in the body. Peripheral nerves extend over long distances to connect limbs, glands and organs to the brain and spinal cord, sending signals that control movement via motor neurons, and relaying information such as pain, touch and temperature via sensory neurons.

Unlike the nerves in the brain and spinal cord, which are protected by the skull and vertebrae, the nerves of the peripheral nervous system have no such protection, leaving them vulnerable to injury. While the body has a mechanism to help peripheral nerves reestablish connections after injury, this process is slow; damaged nerves regrow at an average rate of just one millimeter per day.

The glacial pace of this recovery can take a tremendous toll on peoples lives, as they may have to live with impaired movement and sensation for many months or years.

People with severe peripheral nerve injuries often lose sensation, which makes them susceptible to further injury, and they lose mobility, which can lead to muscle atrophy, said Butler, who holds the Eleanor I. Leslie Chair in PioneeringBrainResearch in the neurobiology department at theDavid Geffen School of Medicine at UCLA. The process of nerve regrowth can be extremely painful and if muscles have atrophied it requires a lot of physical therapy to regain function. My lab seeks methods to accelerate this healing process.

In a 2010 study in mice, Butler and her colleaguesdiscoveredthey could control the rate at which nerves grow in the spinal cord during embryonic development by manipulating the activity of a gene called LIM domain kinase 1, or Limk1. Limk1 controls the rate of nerve growth by regulating the activity of a protein called cofilin. Cofilin plays a key role in a process known as actin polymerization, or treadmilling, which enables nerves to extend thread-like projections over long distances to form neural networks.

Butlers new paper builds on these findings by showing that Limk1 and cofilin also control the rate of growth of peripheral nerves during both development and regeneration.

We discovered that one of the first things a nerve does after injury is switch on all these early developmental molecules that controlled how it grew in the first place, said Butler, who is a member of the UCLA Broad Stem Cell Research Center. Its somewhat similar to how an adult in crisis might reach out to their childhood friends to renew themselves.

In preclinical tests using mouse models with peripheral nerve injuries, Butlers lab showed that this molecular process can be manipulated to make nerves grow faster. Specifically, they found that mice that were genetically engineered so that the Limk1 gene was removed exhibited a 15% increase in the speed of nerve regrowth following injury.

This is a modest improvement for a mouse but one that could translate into a major improvement for a human because our nerves have so much farther to grow, said Butler, who noted that nerves regrow at the same rate in both mice and humans.

This increased rate of nerve regrowth resulted in faster recovery of both motor and sensory functions as measured by how fast the injured mice regained the ability to walk and the sensation in their paws. This is significant because sensory function can take longer than motor function to recover after a traumatic injury, yet sensory function is critical to quality of life.

As a next step, Butler and her lab are using human stem cell-derived motor neurons to screen for drug candidates that could modify this molecular process and speed nerve regeneration in humans. They are also expanding the scope of their study by examining if adding more cofilin rather than inhibiting Limk1 could be even more effective in speeding up recovery from peripheral nerve injuries.

The experimental treatment model described above was used in preclinical tests only and has not been tested in humans or approved by the Food and Drug Administration as safe and effective for use in humans.

Funding for this study was provided by the National Institutes of Health, the Craig H. Neilsen Foundation, the Merkin Family Foundation and the UCLA Broad Stem Cell Research Center Research Award Program, supported by the Jean Perkins Foundation.

Follow this link:
Researchers identify molecular process that could accelerate recovery from nerve injuries - UCLA Newsroom

American Cancer Society research breakfast highlights Colorado innovations changing the face of cancer care – The Know

On October 1, the American Cancer Society (ACS) Cancer ActionNetwork (CAN) along with presenting sponsors University of Colorado CancerCenter and UCHealth hosted more than a hundred leaders from business,education, government, and research communities to answer an interesting question:What do a highly successful new treatment against leukemia stem cells, a newway to point the immune system at pediatric cancer cells, and new understandingof how Medicare expansion affects cancer outcomes have in common? The answer:All three are born in Colorado. Due in part to new investments in infrastructureand the recruitment of top talent, combined with a climate of collaboration andinnovation, CU Cancer Center researchers are at the forefront of discoveries andinitiatives that are driving a golden age of cancer prevention, research, andcare.

Delivering quick welcome messages were representatives from the offices of Senator Michael Bennet, Senator Cory Gardner, and Representative Jason Crow, along with RJ Ours, Colorado Government Relations Director for ACS CAN. Attendees included John J. Reily, Jr., MD, Dean of the University of Colorado School of Medicine, and Don Elliman, Chancellor of the University of Colorado Anschutz Medical Campus.

Wereworking today to strengthen Colorados network of people collaborating to fightcancer so that you have the new tools and resources to accelerate the pace of discoveryduring this significant period of change, opportunity, and promise to endcancer as a health problem, Ours said, setting the tone.

Leading the mornings program was CU Cancer Center Director,Richard Schulick, MD, who spoke about the burden of cancer, the strategies weuse to attack it, and the activities across the CU Cancer Center consortium todevelop new strategies for cancer prevention, early detection, treatment,education, and access to care.

Im here not only as the cancer center director, but as the sonof two parents who developed cancer during their lifetimes. My passion comesfrom very personal experience, Schulick said. What is our goal? For the peoplein this room, the goal is to eradicate the pain and suffering from cancer.

Despite a 26 percent reduction in the death rate of peoplediagnosed with cancer over the previous 25 years, there remains a long way togo toward Schulicks goal. In the next decade, cancer is set to overtake heartdisease as the leading cause of death in the United States. About 1.7 million Americanswill be diagnosed with dangerous cancer this year, and 600,000 will pass fromthe disease, over 8,000 in Colorado alone. Lifetime cancer risk is about 40percent and the risk of dying from cancer is about 20 percent.

I cant imagine a more pressing problem we need to deal with a society,a research institution, and as a community, Schulick said.

In addition to innovative, new treatments, Schulick says manygains will come from learning to better use existing treatments. For example, thetraditional workflow of cancer care starts with a primary care physician, whorefers a patient to an oncology specialist, who may send the patient a weeklater for diagnostic imaging, then a week later to consult with a surgeon, who sendsthe patient to a radiologist, etc. all of which results in many appointmentsover the course of weeks or months just to decide on a course of care. Instead,at CU Cancer Center care partners, patients may be seen in multidisciplinaryclinics.

Its a better model for taking care of cancer patients, Schulicksays. They come in the morning, get a physical exam and any needed imaging, seea nutritionist, a pain specialist, etc. Thenall the doctors and specialists meet from noon to one and go over everything,typically with 30 or more people in the room. Everyone is there, they all weighin and argue about the best treatment plan. Then the whole team meets with thepatient, all at the same time. In one day, everything is done, the whole treatmentplan laid out. Patients love this and their families love it even more.

Now a major goal is to expand access to the best cancer care topatients outside the Denver metro area.

Were trying to make the very best care available to every citizenin Colorado and surrounding states, no matter where they live. Its no good ifwe have all the best therapies and clinical trials concentrated only on thiscampus that doesnt do any good for a lot of people who cant get here. So wehave to spread our ability to care for these patients, Schulick says.

The second presentation highlighted this need for additional services to reach Colorados rural and underserved populations.

Cathy Bradley, PhD, CU Cancer Center deputy director, pointed out that the lung cancer survival rate for patients living in the Front Range is 70 percent, while the survival rate for Coloradans living in rural and high-poverty areas is only 55 percent. Likewise, rates of HPV vaccination that can effectively prevent cervical cancer are 45 percent in Colorado as a whole, but only 28 percent in rural areas.

These disparities are wider than they are elsewhere, Bradleysays. Our white population does better than whites nationally, while our Hispanicsdo worse than Hispanics nationally. And the Colorado youth vaping rate is fourtimes the national average.

Bradley also pointed out the benefit in focusing on cancerprevention, pointing out that while $500,000 could help 3,000 people becomenon-smokers, or screen 700 people for lung cancer, or screen 1,200 people forcolon cancer, the same amount of money is only enough to treat 4-8 people withadvanced cancers.

Until recently, one of the worst of these advanced cancer wasacute myeloid leukemia (AML).

AML is an absolute monster of a disease, one of the mostaggressive forms of cancer known to man. Until a couple years ago, they neverwould have invited someone like me to a breakfast like this: I would have beentoo depressing. Thats all changed in the last couple years, says Daniel A. Pollyea, MD, MS, the Robert H. AllenEndowed Chair in Hematology Research and clinical director of Leukemia Servicesat the CU School of Medicine.

Based on CU Cancer Center basic science, Pollyea and colleagueshave built a hematology program specifically focused on targeting leukemia stemcells.

Its population of cells that cant be killed with chemo and thatcauses relapse, Pollyea says. We believed that if we could kill leukemia stemcells, maybe we could even cure the disease.

The treatment that Pollyea was able to offer to Colorado patientsthrough clinical trials in 2015 earned FDA approval in 2018.

Patients here were essentially getting a treatment of the future,kind of time traveling years into the future to get a therapy that wasntavailable then. Thats what so incredible about being a clinician who works inresearch the hope we could deliver a treatment of the future to a patient today,Pollyea says.

Now new trials at CU Cancer Center are refining Pollyeas treatmentand showing that targeting cancer stem cells may have applications for morepeople with AML and perhaps even beyond leukemia.

At other places, it can be like, Weve never done this before sowere not going to do it now. Here at CU, its more like, Weve never donethat before so lets figure out how to make it happen, Pollyea says.

One of these new things we are just figuring out is how to makehappen is engineering a patients own T cells to attack cancer, which is thespecialty of the mornings third speaker, Terry Fry, MD, CU Cancer Centerinvestigator and co-director of the Human Immunology and ImmunotherapyInitiative at Childrens Hospital Colorado.

I was happy with my career at the National Institutes of Health,Fry says. I had developed a good team, and when Lia Gore [of CU Cancer Centerand Childrens Hospital Colorado] called me to take a look, it was sort of a, Oh,okay, Ill take a look. But from my first visit, it was pretty clear that Coloradowas the place I wanted to be to develop the next generation of immunotherapy.

The first generation of immunotherapies was developed more than acentury ago, when a doctor named William Coley noticed that some cancerpatients who developed infections actually had better cancer outcomes in rarecases, an activated immune system would attack tumor tissue. Then radiation andchemotherapy showed more promise, and anti-cancer immunotherapy went on theback burner for many decades. Terry Fry is a pioneer in the generation ofscientists who revived the idea, often despite naysaying by many in theresearch community who thought it would never pan out.

I just heard Jimmy Carter is celebrating his 95thbirthday today. He was one of the first recipients of immunotherapy for braincancer, Fry says.

But while Jimmy Carters treatment was meant to remove a kind ofbraking system that kept the immune system from attacking cancer, Fryspecializes in the design and testing of treatments that engineer the bodysimmune system T cells to recognize and attack cancer cells.

Ive been privileged to be part of a field called geneticallymodified T cell therapy, or CAR-T cell therapy. Fifteen or twenty years ago, nobodywould have ever thought that it would be possible to take T cells frompatients, genetically modify them to see proteins on the surface of cancer cells,and then reinfuse them as a drug to target cancer, he says.

Still, major challenges remain for CAR-T therapy. Despite beingable to induce remission in 80 percent of pediatric patients with B-cellleukemia, about 50 percent will relapse within the first year.

I certainly dont feel like were done, Fry says. This is atherapy that is very, very new for us in the field of cancer treatment, and westill need to improve induction rate, durability, and the ability to deliver thetreatment safely.

Another challenge for CAR-T therapy is cost, an issue brought upby the first question delivered to the panel by a parent whose daughter hadbeen treated at UCHealth University of Colorado Hospital.

For a long time, the whole conversation was about therapy success,and now people are starting to talk about the challenge of access, Fry says. Thecost of CAR-T is about three or four-hundred thousand per treatment, so its areal challenge that we need to figure out. One thing being discussed is thatthe standard treatment is chemo and bone marrow transplant, which costs morethan $500K and we pay for that now. If we can do this therapy the right way,theres a possibility it could replace another expensive therapy. Also, a lotof work is being done to bring down the cost of these treatments.

The next question asked about research into the rising rate ofcolorectal cancer diagnosed in adults below age 50.

Right now, in my inbox is data describing Colorado rates ofcolorectal cancer in people under 50, says Cathy Bradley. Its something wereaware of as a problem and is just starting to get attention nationally.

Schulick pointed out that the American Cancer Society is leading apush to lower the recommended age to start colorectal cancer screening from 50to 45. The question is whats the cost and what are the lives saved, Schulicksays, but I think theres enough evidence now to lower the screening age.Another frontier is genetic risk. The idea is that maybe if you have a certainpanel of genes, you have your first colonoscopy at age 20 or 30 or 40. I thinkthe frontiers are being pushed and were learning more about genetics and riskfactors and how to implement screening that saves lives.

Additional questions focused on how federal policies may affectcancer research, including a proposal to increase annual NIH funding by $6 billion,and how Medicare expansion has affected cancer outcomes.

In a study funded by the ACS, we looked at states that expanded Medicareand those that did not, and found that with far more low income women beingscreened, there were fewer late-stage diagnoses, and longer survival, saysBradley. Also, the availability of medications through Medicaid meant that morewere people taking medicines as directed, and more people staying in theworkforce.

These federal policies that affect cancer research and care mayseem abstract, but the ACS closed the morning by offering two ways to getinvolved now: First, ACS Ambassador Martha Cox suggested signing the ACSpetition to increase cancer research funding; second, Cox suggested becominga member of the ACS Cancer Action Network.

Despite significant progress against cancer, there remains muchmore to do. Right now, here in Colorado, we are at an absolute epicenter of researchaimed at the disease. Events like this mornings ACS research breakfast ensurethat everyone in the community of people who care about cancer is aware of thegreat opportunity and also the great responsibility we have to continuepowering this push toward a day when suffering from cancer is no more.

See the original post:
American Cancer Society research breakfast highlights Colorado innovations changing the face of cancer care - The Know

A Therapeutic Counterblow to Traumatic Brain Injury – Technology Networks

A blow to the head or powerful shock wave on the battlefield can cause immediate, significant damage to a person's skull and the tissue beneath it. But the trauma does not stop there. The impact sets off a chemical reaction in the brain that ravages neurons and the networks that supply them with nutrients and oxygen.

It is the secondary effects of traumatic brain injury (TBI), which can lead to long-term cognitive, psychological and motor system damage, that piqued the interest of a team of NJIT biomedical engineers. To counter them, they are developing a therapy, to be injected at the site of the injury, which shows early indications it can protect neurons and stimulate the regrowth of blood vessels in the damaged tissue.

The challenge, researchers say, is that brain cells don't regenerate as well as other tissues, such as bone, which may be an evolutionary strategy for preserving the synaptic connections that retain memories. To date, there is no effective treatment for restoring damaged neurons. The body's protective mechanisms also make it difficult to penetrate the blood-brain barrier, which hampers the delivery of medications.

"Nerve cells respond to trauma by producing excessive amounts of glutamate, a neurotransmitter that under normal conditions facilitates learning and memory, but at toxic levels overexcites cells, causing them to break down. Traumatic brain injury can also result in the activation and recruitment of immune cells, which cause inflammation that can lead to short- and long-term neural deficits by damaging the structure around cells and creating a chronic inflammatory environment," says Biplab Sarkar, a post-doctoral fellow in biomedical engineering and member of the team that presented this work at a recent American Chemical Society conference.

The team's treatment consists of a lab-created mimic of ependymin, a protein shown to protect neurons after injury, attached to a delivery platform -- a strand of short proteins called peptides, contained in a hydrogel -- that was developed by Vivek Kumar, director of NJIT's Biomaterial Drug Development, Discovery and Delivery Laboratory. After injection, the peptides in the hydrogel reassemble at the localized injury site into a nanofibrous scaffold that mimics extracellular matrix, the supporting structure for cells. These soft materials possess mechanical properties similar to brain tissue, which improves their biocompatibility. They promote rapid infiltration by a variety of stem cells which act as precursors for regeneration and may also provide a biomimetic niche to protect them.

Now in preclinical animal trials, rats injected with the hydrogel retained twice as many functioning neurons at the injury site as compared to the control group. They also formed new blood cells in the region.

"The idea is to intervene at the right time and place to minimize or reverse damage. We do this by generating new blood vessels in the area to restore oxygen exchange, which is reduced in patients with a TBI, and by creating an environment in which neurons that have been damaged in the injury are supported and can thrive," Kumar says. "While the exact mechanism of action for these materials is currently under study, their efficacy is becoming apparent. Our results need to be expanded, however, into a better understanding of these mechanisms at the cellular level, as well as their long-term efficacy and the resulting behavioral improvements."

Collaborators James Haorah, an associate professor of biomedical engineering, and his graduate student Xiaotang Ma at NJIT's Center for Injury Biomechanics, Materials and Medicine have shown how a number of TBI-related chemical effects can disrupt and destroy integral brain vasculature in the blood-brain barrier, the brain's protective border, promoting chronic inflammation that can lead to symptoms such as post-traumatic stress disorder and anxiety, among others. Their current work provides insights into the potential neuroprotective and regenerative response guided by the Kumar lab's materials, while future studies will attempt to analyze other mediators of inflammation and blood flow in the brain.

Kumar's delivery mechanism is a customizable, Lego-like strand made of short proteins called peptides, which are composed of amino acids, with a biological agent attached at one end that can survive in the body for weeks and even months, where other biomaterials degrade quickly. Its self-assembling bonds are designed to be stronger than the body's dispersive forces; it forms stable fibers, with no signs of inducing inflammation, that rapidly incorporate into specific tissues and collagen, recruiting native cells to infiltrate. The hydrogel, which is also composed of amino acids, is engineered to trigger different biological responses depending on the payload attached. These platforms can deliver drugs and other small cargo over day-, week- or month-long periods. Kumar's lab has recently published research on applications ranging from therapies to prompt or prevent the creation of new blood vessel networks, to reduce inflammation and to combat microbes.

"The ultimate hope is that that localized delivery of regenerative materials may provide significant benefits for a number of pathologies," he notes.

For example, the team recently developed a class of materials that may be useful against infection. These novel anti-microbial peptides are capable of disrupting dense bacterial colonies and have shown promise against a number of yeasts. Additionally, they promote human cell proliferation and are currently being studied for wound healing. That work was published this summer in the journal ACS Biomaterials Science and Engineering.

Kumar and his lab have created another hydrogel designed to recruit autologous (a person's own) dental pulp stem cells directly to the disinfected cavity after root canal therapy. The tooth would be regenerated in part by prompting growth of the necessary blood vessels to support the new tissue. Yet another peptide-based therapy, armed with antiangiogenic capabilities, targets diabetic retinopathy, an ocular disease affecting more than 90 million people worldwide. People with the disease form immature blood vessels in the retina, obstructing their vision. The hydrogel can be injected directly into the vitreous gel of the eye, where the peptide interacts with the endothelial cells in the aberrant blood vessels, causing them to die.

"Conventional biomaterials used in tissue regeneration suffer from a variety of problems with delivery, retention and biocompatibility, which can lead to rejection by the host," Kumar says. "We're trying to address these issues with a technology designed to be universal in its application, delivering materials that persist in the tissue and promote their biologic effects for long periods of time."

Reference: Sarkar et al. 2019.Membrane-Disrupting Nanofibrous Peptide Hydrogels. ACS Publications. DOI: https://doi.org/10.1021/acsbiomaterials.9b00967.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

Read more:
A Therapeutic Counterblow to Traumatic Brain Injury - Technology Networks

Cancer cell therapy success grows, but hospitals struggle with cost – The San Diego Union-Tribune

A lifesaving new cancer therapy using a patients own immune cells continues to show success, but hospitals are struggling with its immense and under-reimbursed costs.

Doctors with these programs at UC San Diego and City of Hope in Duarte gave that cautionary message Thursday at the annual Cell and Gene Meeting on the Mesa in Carlsbad.

The therapy uses immune cells called T cells, which are genetically engineered to fight the patients cancer. Two therapies are approved for commercial use, and several others are in clinical testing.

While these CAR T cell therapies have risks and often fail, they save many extremely ill patients whose disease has grown resistant to multiple previous rounds of treatment, said Dr. John Zaia, director of its Center for Gene Therapy.

But as these therapies become more accepted, hospitals are now faced with a new drain on revenues that needs a long-term solution, Zaia said.

Its a real problem, Zaia said. The institution loses money on the commercial products that we use.

Medicare reimbursement doesnt cover the hospitals expense for the treatment, he said. In addition, patients are very sick and need to be cared for before and after therapy.

It costs up to $1.5 million to manage a CAR T cell patient, he said, citing a study from Oregon Health & Science University.

The success rate with CAR T cells is higher than with other therapies for difficult cancers, Zaia said.

So youre probably getting more value for your dollar if you use CARs, but you still cant afford it. Its got to be solved, because its not a sustainable model.

UC San Diego has similar financial issues with the therapy, said Dr. Dimitrios Tzachanis, an associate clinical professor of medicine at UCSD Blood and Marrow Transplant Program.

It requires strong commitment at the very top of academic centers to say were losing money but we want to be part of this, Tzachanis said. But I dont know how long it will be sustainable.

UCSD administrators are examining CAR T cell costs every month, Tzachanis said.

City of Hope has also seen an unexpected increase in cancer patients who hang on despite being extremely ill, Zaia said. These patients have heard of successes with the therapy and want to give it a try.

Youre talking with patients who are extremely sick about hospice care and end-of-life care, he said. That conversation was curtailed in many cases ... That changes the whole dynamic of patient load into a hospital. Our census in the hospital has been a major problem in the last two years.

City of Hope has been using donor dollars to support the program, Zaia said.

Patients who expect a sure cure are likely to be disappointed, Tzachanis said.

The so-called cure rate is probably in the 40 percent range at this point, he said. So most patients wont respond to the CAR T cells.

More:
Cancer cell therapy success grows, but hospitals struggle with cost - The San Diego Union-Tribune

CAR T-cell Therapy: Reprogramming the Immune System To Treat Cancer – Technology Networks

At the Malaghan Institute for Biomedical Reserach, researchers are developing third generation CAR-T immunotherapy treatments which aim to be more effective with less side effects than previous therapies.

Using patients own modified T-cells, CAR-T therapy has the ability to vanquish cancer cells, vastly improving outcomes of once life threatening diseases.

This year, 2019, Rob hopes to begin clinical trials for CAR-T therapy in New Zealand through the collaboration of scientists from the Malaghan Institute and international researchers. He is optimistic that this next generation treatment will present a new paradigm for treatment of lymphomas and possibly for other cancers in the future.

Dr Robert Weinkove is Clinical Director of the Malaghan Institute, a biomedical research institute in Wellington, New Zealand. After studying medicine at the University of Cambridge and Kings College London, Rob trained in Hematology in London and Germany before moving to New Zealand in 2008 and gaining a doctorate with the University of Otago.

Rob is a key member of the Cancer Immunotherapy Programme to develop and manufacture chimeric antigen receptor (CAR) T-cells for treatment of lymphoma and other blood cancers. He also holds a joint role as a Hematologist at Wellington Blood & Cancer Centre.

Continued here:
CAR T-cell Therapy: Reprogramming the Immune System To Treat Cancer - Technology Networks

Global Personalized Cell Therapy Market Analysis Report 2019 || What are Things You Should Keep in Mind to Grow Your Business? – Sound On Sound Fest

New York City, NY: October 07, 2019 Published via (Wired Release) The global Personalized Cell Therapy market is carefully researched in the report while largely focusing on top key players and their business strategies, geographical development, market segments, competitive landscape, manufacturing, and pricing & cost structures. Each section of the research study is specially prepared to explore key aspects of the global Personalized Cell Therapy market. For instance, the market dynamics section digs deep into the drivers, trends, restraints, and opportunities of the global Personalized Cell Therapy Market. With qualitative and quantitative analysis, we help you with thorough and comprehensive research on the global Personalized Cell Therapy market. We have also focused on SWOT, PESTLE, and Porters Five Forces analyses of the global Personalized Cell Therapy market.

Leading players of the global Personalized Cell Therapy market are analyzed taking into account their market share, latest developments, new product launches, partnerships, mergers/acquisitions, and markets served. We also provide a comprehensive analysis of their product portfolios to explore the products and applications they concentrate on when operating in the global Personalized Cell Therapy market. Furthermore, the report offers two separate market forecasts production side and consumption side of the global Personalized Cell Therapy market. It also provides useful recommendations for new as well as established players of the global Personalized Cell Therapy market.

On the basis of region, the Personalized Cell Therapy report is segmented into North America, Europe, South America, Asia Pacific, and Middle East & Africa.

To Obtain All-Inclusive Information Download Sample Copy of Personalized Cell Therapy Market Report Study 2019-2028 At:https://marketresearch.biz/report/personalized-cell-therapy-market/request-sample

The report analyses below-mentioned aspects in the Personalized Cell Therapy market:

Competitors analysis of Personalized Cell Therapy Market: This section offers information about the competitive landscape among the top players in the Personalized Cell Therapy market. It also contains company profiles, revenue and financial details, product portfolio, and offerings, strategies, mergers & acquisitions, agreements, and partnerships, etc.

Personalized Cell Therapy Market Leading Players:

Cytori Therapeutics Inc, Bellicum Pharmaceuticals Inc, Saneron CCEL Therapeutics Inc, MolMed S.p.A., Vericel Corporation, Oxford Nanopore Technologies, Cell Medica, MediGene AG, TxCell

Personalized Cell Therapy Market Segmentation:

Global personalized cell therapy market segmentation, by technique:

Platelet TransfusionsBone Marrow TransplantationPacked Red Cell TransfusionsOrgan TransplantationGlobal personalized cell therapy market segmentation, by therapeutic area:

Cardiovascular DiseasesNeurological DisordersInflammatory DiseasesDiabetesCancer

*Production volume analysis of Personalized Cell Therapy Market: This part includes import/export data, production volume, price (ASP) etc.

Some Key aspects cover in Personalized Cell Therapy Market Report:

Overview of the Personalized Cell Therapy market share, supply chain analysis

Competitive landscape of key players in Personalized Cell Therapy market

Forecast for global Personalized Cell Therapy market up to 2028

Personalized Cell Therapy Market Overview and success factors

Have Any Query Or Specific Requirement? Ask Our Industry Expertshttps://marketresearch.biz/report/personalized-cell-therapy-market/#inquiry

In conclusion, the global Personalized Cell Therapy market report provides a systematic and descriptive analysis of the Personalized Cell Therapy market, supported by historical and current information of key players and vendors, and all the above-mentioned factors and potential developments in future to aid in gaining crucial insights regarding volume, revenue, and others, which could aid clients in business-related needs.

* Inclusion of Import/export data, production volume are subjected to the scope of the market study

Questions Answered in Global Personalized Cell Therapy Market Report 2019:

What will the be the Personalized Cell Therapy market size in 2028?

What will be the growth rate?

What are the major Personalized Cell Therapy market trends?

What is urging Personalized Cell Therapy market?

Who are the outstanding vendors in the world Personalized Cell Therapy market?

What are the challenges to Personalized Cell Therapy market growth?

What are market trends striking the growth of the Personalized Cell Therapy industry?

Share Your QuestionsHere For More Details On this Report or Customizations As Per Your Need:https://marketresearch.biz/report/personalized-cell-therapy-market/#request-for-customization

Contact Us:

Mr. Benni Johnson ([emailprotected])

MarketResearch.Biz (Powered By Prudour Pvt. Ltd.)

420 Lexington Avenue, Suite 300

New York City, NY 10170,

United States

Website:https://marketresearch.biz

This content has been distributed via WiredRelease press release distribution service. For press release service inquiries, please reach us at[emailprotected]

See the original post here:
Global Personalized Cell Therapy Market Analysis Report 2019 || What are Things You Should Keep in Mind to Grow Your Business? - Sound On Sound Fest

Cell therapy safe for liver patients, trial shows – Mirage News

Liver disease patients could one day benefit from a new cell therapy that has just completed its first clinical trial.

Researchers who tested the potential treatment in patients with liver cirrhosis where long term damage produces scarring found the therapy had no significant adverse effects.

Now the team, based at the Universitys MRC Centre for Regenerative Medicine, is to gauge the effectiveness of the treatment which is based on white blood cells called macrophages, that are key to normal liver repair.

The next stage of the trial will measure whether the therapy helps the liver to reduce scarring and stimulate regeneration. The results should be known within the next two years.

At present the only successful treatment for end-stage liver cirrhosis which claims around 14,000 lives in the UK each year (British Liver Trust) is an organ transplant. The safety trial is a vital step forward in finding an alternative therapy.

During the trial scientists took cells from the blood of nine patients with the disease and turned them into macrophages, in the Scottish National Blood Transfusion Services (SNBTS) cell therapy facility.

The new cells were then re-injected into the patient with the hope of repairing the damaged organ from within.

Causes of liver cirrhosis include infections such as hepatitis C, obesity, alcohol excess and some genetic and immune conditions.

Liver cirrhosis is a major healthcare issue in the UK and is one of the top five killers. The results from this first safety trial are encouraging and we can now progress to testing how effective it is in a larger group of people. If this was found to be effective it would offer a new way to tackle this important condition.

The research which was published in the journal Nature Medicine, received funding from the Medical Research Council and was conducted in partnership with the SNBTS and the Cell and Gene Therapy Catapult.

View original post here:
Cell therapy safe for liver patients, trial shows - Mirage News

New Automation Solution to Accelerate Cell and Gene Therapy Manufacturing Announced – Labmate Online

Terumo BCTs latest cell therapy technology, the Finia Fill and Finish System, is a first-of-its-kind device developed to help bring reproducibility and control to cell therapy manufacturing to get therapies to more patients who need them.

Finia is a fully automated, modular, functionally closed system that creates the final formulation of cell and gene therapies and divides them into user-defined doses for patients. The technology automates a process that is currently manual and labour- intensive with the added risk of error.

Terumo BCT technologies have been automating the collection and processing of blood and cells for decades, said Antoinette Gawin, President and Chief Executive Officer, Terumo BCT. Now, were translating this knowledge to support technology breakthroughs in cell and gene therapies. Finia helps cell therapy developers and researchers simplify processes while increasing accuracy, consistency, reliability and reproducibility - key to securing regulatory approval.

The production and delivery of cell-based therapies is a complex and logistically challenging process, said Delara Motlagh, Vice President, Cell Therapy Technologies, Terumo BCT. Developers and manufacturers appreciate support in determining when to automate, which processes to automate and how to integrate automation into their existing process.

Finia combines, mixes and divides the final product into predetermined volumes, all under controlled, refrigerated temperature. It automatically removes air before it seals the final product bags, simplifying the downstream process. Finia also works with Terumo BCT's Cell Processing Application software in the customers' network to facilitate compliance to current good manufacturing practices (cGMP), including electronic recordkeeping, control of process workflow, user credentialing and permission, and guidance of operators through the process with an intuitive user interface module (UIM).

With Finia, we are expanding our portfolio of automation technologies aimed at this growing industry, said Motlagh. We are bringing multiphase solutions for cell and gene therapies helping to scale manufacturing and drive toward commercialisation to reach larger patient numbers.

More information online: https://ilmt.co/PL/6VaE

More here:
New Automation Solution to Accelerate Cell and Gene Therapy Manufacturing Announced - Labmate Online

Arcellx Raises $85 Million in a Series B Financing to Advance its Intelligent Cell Therapy Platform – GlobeNewswire

GAITHERSBURG, Md., Oct. 03, 2019 (GLOBE NEWSWIRE) -- Arcellx, a privately-held biopharmaceutical company, today announced that it has raised $85 million in an oversubscribed Series B financing. Proceeds will be used to advance the Companys ARC-T + sparX programs, including clinical development of a bivalent BCMA-targeted cell therapy in multiple myeloma, and a CD123-targeted therapy in acute myeloid leukemia. The Series B will also fund earlier stage ARC-T + sparX programs for patients with solid tumors and diseases outside oncology.

Participants in the Series B include both existing and new investors to Arcellx. New investors Aju IB and Quan Capital co-led the round, followed by Mirae Asset Venture Investment, Mirae Asset Capital, LG Technology Ventures, JVC Investment Partners, and certain funds managed by Clough Capital Partners, L.P. Existing investors Novo Holdings, S.R. One Limited, NEA and Takeda Ventures also participated in the financing.

Concurrent with the financing, Hugo Beekman, Partner at Aju IB, and Lewis (Rusty) Williams, M.D., Ph.D., Venture Partner at Quan Capital, have joined the Arcellx board of directors.

The financial and strategic support from our investors allows Arcellx to accelerate development of a robust pipeline of ARC-T + sparX programs for patients in need, commented David Hilbert, Ph.D., President and Chief Executive Officer of Arcellx. As impressive as conventional CART therapies have been, their safety and efficacy profiles are challenged by severe toxicities, high rates of relapse, and challenging target selection in the solid tumor setting. The ARC-T + sparX platform addresses these concerns by placing ARC-T cells under the control of one or more sparX proteins that uniquely determine how the ARC-T cells recognize tumor, and the speed with which ARC-T cells kill tumor. In the coming months we will begin clinical testing of our lead BCMA-targeted therapy in multiple myeloma.

Rusty Williams, M.D., Ph.D., commented, Arcellx hasreached a positive inflectionin its novel platform and pipeline with the potential to improve efficacy and safety. We are excited to support the company as it advances new cell therapies with the potential to deliver better outcomes for patients.

Hugo Beekman, Partner at Aju IB, commented, Arcellx has invented a differentiated cell therapy platform with ARC-T + sparX that allows simultaneous and sequential targeting of multiple tumor antigens. The ability of sparX proteins to reprogram the specificity of ARC-T cells has the potential to address the high incidence of tumor relapse, as well as the inherent diversity of tumor antigens expressed within solid tumors. The features of this platform, along with scalable and efficient manufacturing processes, are intended to facilitate the Companys development of new therapies in oncology, and more broadly, in autoimmune disease and the transplant setting.

About ARC-T + sparX Technology

Arcellx has pioneered a proprietary sparX + ARC-T platform in which a Soluble Protein Antigen-Receptor X-linker (sparX) simultaneously binds one or more tumor antigens and engages a universal receptor expressed on the Antigen-Receptor Complex T cells (ARC-T). The formation of a sparX + ARC-T + tumor cell complex results in tumor killing. This therapeutic platform is designed to enhance safety and efficacy while accelerating development by broadening patient accessibility and increasing efficiency of manufacturing relative to existing cell therapies.

About Arcellx, Inc.

Arcellx is a privately held biopharmaceutical company located in Montgomery County, Maryland. The Arcellx team is devoted to providing patients with superior immune cell therapies through scientific innovation, accelerated development, and responsible patient care. Although our initial clinical focus is cancer therapy, we are committed to extending our Antigen-Receptor Complex T cell (ARC-T) therapies across a broad spectrum of human disease.

Contact:Solebury TroutZara Lockshin (media)Tel: +1 646-378-2960Email: zlockshin@troutgroup.com

Alan Lada (investors)Tel: +1 646-378-2927Email: alada@troutgroup.com

Originally posted here:
Arcellx Raises $85 Million in a Series B Financing to Advance its Intelligent Cell Therapy Platform - GlobeNewswire