Know-How About New CAR-T Cell Therapies Shifting – Medscape

Chimeric antigen receptor (CAR) T-cell therapies for cancer are still so new and they can be extremely effective, but "it's a therapy that carries a lot of risk," said Lauren Spendley, NP, from the Dana-Farber Cancer Institute and Harvard Medical School in Boston.

At her center, she explained, all patients who have received CAR T-cell therapy are admitted for a minimum of 1week after infusion. A rotating team of advanced practice providers take care of those and other hemo-malignancy patients.

"We realized that we needed a dedicated group to make sure we were able to safely provide quality care for these patients, given the unique toxicity profile" said Spendley, who described her experience as a CAR-T program manager and presented research on associated toxicities from two studies she was involved in (Brain. 2019;142:1334-1348 and JAdv Pract Oncol. 2019;10[suppl3]:11-20).

Typically, the toxicity comes in two waves. "The onset of cytokine release syndrome [CRS] occurs 24 to 48 hours after the cells are infused. It can last about a week. It looks a lot like sepsis," she explained. "Then the neurotoxicity comes on day5 or 6, and can last days to weeks," she said, noting that it usually overlaps CRS.

If patients have severe CRS, "we predict they'll have severe neurotoxicity as well," she added. And patients with a higher burden of disease tend to have higher-grade toxicities. "You can sort of see that coming."

These patients undergo an infection workup, and treatment with antibiotics and tocilizumab usually resolves the infusion reaction. "Having vigilant advanced care at the bedside plays a role in identifying and treating symptoms," Spendley explained. Both CRS and neurotoxicity can be fatal when not treated quickly.

Before CAR T-cell treatment is administered, patients are assessed for comorbidities, performance status, and organ function. "They need to be able to tolerate these toxicities if they become severe," she noted.

But the results of CAR T-cell therapy are nothing short of amazing, she emphasized at the Association of Physician Assistants in Oncology 2019 Annual Symposium in Boston. "That someone suffering so much from their disease can, days to weeks later, be in complete remission is remarkable. It's truly ground-breaking for the lymphoma landscape."

The Centers for Medicare and Medicaid Services recently announced that the costly therapy would be covered for indications approved by the US Food and Drug Administration along with some off-label uses in hospitals that participate in the Risk Evaluation and Mitigation Strategy (REMS) program, as reported by Medscape Medical News.

For children with acute lymphoblastic leukemia, the therapy costs $475,000; for adults with diffuse large B-cell lymphoma, it costs $373,000. Until now, this was out of reach for patients without insurance or other means to foot the bill.

But care teams need to be ready for increased demand. Centers offering the therapy will benefit from a dedicated team, Spendley asserted. "At our center, we can now accommodate a larger volume of patients without missing a beat because we are prepared with the knowledge and experience to know the signs of complications," she said.

The treatment can be highly effective, "making the tumor disappear within days," said Kadee Raser, PA-C, CAR T-cell therapy lead at the University of Michigan Rogel Cancer Center in Ann Arbor.

She described a patient with a tumor on the side of her neck: "Just days after the CAR T-cell therapy, you visibly saw the area shrink dramatically and go away. You don't typically ever see that with standard-therapy radiation."

These patients can end up in the ICU. It's different than your typical transplant or chemo.

However, the toxic effects of the therapy "can get complicated," Raser told Medscape Medical News. These patients "can end up in the ICU. It's different than your typical transplant or chemo."

"We watch these patients very closely," she added.

The Rogel Cancer Center did its first CAR T-cell infusions when the treatment was in its infancy and then developed a dedicated program.

"It helps that our advanced providers were involved from the beginning," said Adrienne Trentacosti, PA-C, outpatient CAR T-cell therapy lead at Rogel. "We have a rapidly growing team of nurse practitioners and physician assistants with CAR-T knowledge who work interchangeably to manage patients."

A physician always performs the preliminary patient consult, Trentacosti said, but once the patient is deemed suitable in a team meeting, advanced practitioners including nurse practitioners and physician assistants set up the therapy and follow the patient through to recovery.

"We're the continuity, the glue, that holds the treatment together, and the daily connection to the patient," she said. That makes it easier to identify any change in the patient. "Assistant practitioners don't rotate; we're always there. Physicians move from inpatient to research to something else."

"We spend more time with the patient than the physicians or nurses are able to," she added.

As the general population ages, oncology will continue to be a growing field for physician assistants. "We're going to see more oncology patients," Trentacosti noted.

Because "the cellular therapy program is fairly new, we're just now hiring dedicated staff, dedicated social workers, and dedicated physician assistants for CAR T-cell therapy," she added. "We're expanding rapidly. I just hired four new people."

There is a lot of interest and excitement about the therapy, Raser confirmed, but she warned that the final results are not in yet. "We're a little early in speaking to the durability of the treatment." she said. "We don't know how long it lasts. We are still learning."

Spendley, Raser, and Trentacosti have disclosed no relevant financial relationships.

Association of Physician Assistants in Oncology (APAO) 2019 Annual Symposium.

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Advances Of Chimeric Antigen Receptor T Cell Therapy In Ovarian Cancer | OTT – Dove Medical Press

Wenying Yan,1 Hongmei Hu,2 Biao Tang2

1Department of Gynecology, Wangjiang Hospital, Sichuan University, Chengdu, Sichuan Province, Peoples Republic of China; 2Department of Gynecology, Sichuan Maternal and Child Health Hospital, Chengdu, Sichuan Province, Peoples Republic of China

Correspondence: Biao TangDepartment of Gynecology, Sichuan Maternal and Child Health Hospital, No. 290 Shayan West Second Street, Jinyang Road, Chengdu City, Sichuan Province 610041, Peoples Republic of ChinaTel +86 28 8546 3088Email 286546430@qq.com

Abstract: Ovarian cancer, as a common gynecological tumor, is currently recognized as the most lethal gynecological malignancy. In addition to conventional treatment methods such as surgery, radiotherapy and chemotherapy, adoptive immunotherapy represented by modified immune cells also shows good curative effects and is becoming an important method in the treatment of ovarian cancer. Studies have shown that most cancer cells can avoid the recognition of the immune system, thus limiting the anticancer effect of immunotherapy. Chimeric antigen receptor T (CAR-T) cell technology has emerged and has good targeting, killing, proliferation and persistence. A large number of clinical trials also have shown that this technology has achieved great success in improving the quality of life and prolonging the survival time of patients with malignant hematological tumors. CAR-T cell technology has become a research hotspot for immunotherapy. This article mainly reviews various CAR-T cell treatments and their specific mechanisms in the field of ovarian cancer treatment to provide new ideas for the treatment of ovarian cancer.

Keywords: immunotherapy, T cell, ovarian cancer, chimeric antigen receptor, tumor antigens

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Advances Of Chimeric Antigen Receptor T Cell Therapy In Ovarian Cancer | OTT - Dove Medical Press

The time is now: John Cox assumes CEO post at Flagship-backed Torque, steering cell therapy to clinical trials – Endpoints News

A stealthy Torque Therapeutics is in the midst of raising a Series B round to bankroll an upcoming slate of clinical studies for its cancer cell therapy platform and it will all be overseen by a new CEO.

John Cox, executive chairman since January, is taking over from Bart Henderson as the former chief pursues some other work with Flagship Pioneering.

The appointment marks the first full-time gig for Cox since he flipped Biogens hemophilia-focused spinout Bioverativ to Sanofi for $11.6 billion, earning a payout worth more than $85 million in the process. The little taste he had of cell therapy there, albeit for a distinctly different application, helped convince him that its a modality for the future. He envisions Torque becoming the definitive immuno-oncology cell therapy company, and the time is now to jump in.

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The time is now: John Cox assumes CEO post at Flagship-backed Torque, steering cell therapy to clinical trials - Endpoints News

NIH researchers create new viral vector for improved gene therapy in sickle cell disease – National Institutes of Health

News Release

Wednesday, October 2, 2019

Forward-oriented design might boost treatment effectiveness and broaden use.

Researchers at the National Institutes of Health have developed a new and improved viral vectora virus-based vehicle that delivers therapeutic genesfor use in gene therapy for sickle cell disease. In advanced lab tests using animal models, the new vector was up to 10 times more efficient at incorporating corrective genes into bone marrow stem cells than the conventional vectors currently used, and it had a carrying capacity of up to six times higher, the researchers report.

The development of the vector could make gene therapy for sickle cell disease much more effective and pave the way for wider use of it as a curative approach for the painful, life-threatening blood disorder. Sickle cell disease affects about 100,000 people in the United States and millions worldwide.

Our new vector is an important breakthrough in the field of gene therapy for sickle cell disease, said study senior author John Tisdale, M.D., chief of the Cellular and Molecular Therapeutic Branch at the National Heart, Lung, and Blood Institute (NHLBI). Its the new kid on the block and represents a substantial improvement in our ability to produce high capacity, high efficiency vectors for treating this devastating disorder.

Researchers have used virus-based vehicles for years in gene therapy experiments, where they have been very effective at delivering therapeutic genes to bone marrow stem cells in the lab before returning them to the body. But theres always room for improvement in their design in order to optimize effectiveness, Tisdale noted. He compared the new virus-based vehicle to a new and improved car that is also far easier and cheaper for the factory to produce.

The study was supported by the NHLBI and the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), both part of the NIH. It was published online today in Nature Communications.

Sickle cell disease is an inherited blood disorder caused by a mutation, or misspelling, in the beta-globin gene (or -globin gene). This mutation causes hemoglobin, the main ingredient of blood cells, to produce sickle-shaped cells that can stick to the walls of blood vessels, causing blockage, pain, anemia, organ damage, and early death. With gene therapy, doctors modify the patients bone marrow hematopoietic (blood-producing) stem cells in the lab by adding a normal copy of the beta-globin gene through the use of a viral vector. They then reinfuse the modified stem cells into the patient to produce normal, disc-shaped red blood cells.

For the past 30 years, researchers have been designing these beta-globin vectors in a reverse structural orientation, meaning the therapeutic genes incorporated into the virus are translated, or read, from right to left by the viral vector-making machinery much like reading an English sentence backwards. The reason for the reverse orientation is the sensitive expression of a key molecular component of the vector called intron 2. This segment is required for high-level beta-globin gene expression but gets clipped out during the normal vector preparation process if it is left in the natural, forward direction. Gene therapy trials using reverse-oriented vectors for sickle cell disease and beta-thalassemia have largely been encouraging, the researchers said, but this complicated gene translation process has made vector preparation and gene-transfer efficiency more difficult.

About 10 years ago, Tisdale and Naoya Uchida, M.D., Ph.D., a staff scientist in his lab, searched for an improved delivery vehicle like designing a better car and decided to undertake a radical redesign of the beta-globin vector. They came up with a unique work-around design that left intron 2 intact and created the new forward-oriented beta-globin vector. In contrast to the old vector, the gene sequence, or message, of the new beta-globin vector is read from left to right like reading a normal sentence making the gene translation approach less complicated, Tisdale explained.

The researchers tested the new vectors in mice and monkeys and compared the results to reverse-oriented vectors. They found that the new vectors could transfer a much higher viral load up to six times more therapeutic beta-globin genes than the conventional vectors and had four to 10 times higher transduction efficiency, a measure of the ability to incorporate corrective genes into repopulating bone marrow cells. The new vectors also showed a capacity for longevity, remaining in place four years after transplantation. Researchers also found that they could be produced in much higher amounts than the conventional vectors, potentially saving time and lowering costs associated with large-scale vector production.

Our lab has been working on improving beta-globin vectors for almost a decadeand finally decided to try something radically differentand it worked, Tisdale said. These findings bring us closer to a curative gene therapy approach for hemoglobin disorders.

The new vector, for which the NIH holds the patent, still needs to undergo clinical testing in humans. Already an estimated 27 people with sickle cell disease have undergone experimental gene therapy using conventional vectors. Through its Cure Sickle Cell Initiative, NIH is working to accelerate the development of these and other new genetic therapies, including gene editing, with the goal of finding a cure for the disease. The initiative is part of NIHs larger multi-pronged approach to reducing the burden of blood disorders. People with sickle cell disease can visit clinicaltrials.gov to find a clinical trial that is actively enrolling.

About the National Heart, Lung, and Blood Institute (NHLBI):NHLBI is the global leader in conducting and supporting research in heart, lung, and blood diseases and sleep disorders that advances scientific knowledge, improves public health, and saves lives. For more information, visitwww.nhlbi.nih.gov.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

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NIH researchers create new viral vector for improved gene therapy in sickle cell disease - National Institutes of Health

TrakCel announces strategic partnership with WuXi AppTec Advanced Therapies for optimization of supply chain for cell and gene therapy treatments -…

Partnership combines global CDMO services with industry leading cellular orchestration platform

Cardiff, UK & Philadelphia, PA, October 1, 2019 TrakCel, the software developer for cell and gene therapy supply chain tracking and orchestration systems and WuXi AppTec Advanced Therapies, a Contract Development and Manufacturing Organization (CDMO), today announce a collaborative agreement designed to accelerate the seamless delivery of advanced therapy treatments to bring an end-to-end solution to mutual customers.

To provide full visibility and control of their collection-to-administration supply chain, cell therapy companies need to ensure consistent product handling and data capture across all supply chain partners. This is increasingly being achieved through the deployment of supply chain management software, such as TrakCels platform. Challenges can arise if software-driven workflows do not account for established processes in place at each supply chain partner. For example, if a contract manufacturer needs to change their processes to fit in with those designed into supply chain management software, this can increase costs and timelines for software adoption. Conversely, rewriting software after it becomes apparent that it will not work for a contract manufacturer also incurs additional expense and can delay software deployment.

TrakCel and WuXi AppTec Advanced Therapies will leverage configured manufacturing module in TrakCels software, based around WuXis current standard operating procedures and preferred workflows. The design of this module will be based on the companies long and extensive working relationship. WuXi AppTec Advanced Therapies was the first CDMO to use TrakCels software and its Philadelphia, PA site currently has a greater number of users than any other facility in the supply chain. The resultant pre-built manufacturing module in the TrakCel platform will shorten the time and costs associated with deploying an end-to-end solution to TrakCel and WuXis mutual clients.

Both WuXi AppTec Advanced Therapies and TrakCel will continue to build from their long extensive partnership by exploring opportunities to further enhance their combined service to their mutual customers. As one example, automated population of TrakCels scheduling system with data from WuXis own manufacturing capacity plans will further strengthen the ability of existing clients of both companies to accurately plan patient treatment schedules. By increasing the ability to maximize the utilization of contracted manufacturing resources, this should have a considerable impact on cost of goods to patients and healthcare providers.

As one of the first CDMOs to use TrakCels software and the supply chain partner with more users than any other facility, WuXi AppTec Advanced Therapies is well placed to collaborate with TrakCel to identify a more efficient way of working together. It is the latest development in TrakCels formation of an international partner ecosystem of connected and complementary cell and gene therapy service providers. This partner ecosystem as a result now includes a commercial scale, third party manufacturing company, said Ravi Nalliah, CEO, TrakCel. The TrakCel partnership with WuXi AppTec Advanced Therapies will enable both companies to move towards increased standardization and improved delivery of these innovative treatments to patients around the world.

We are pleased to establish a collaborative agreement with TrakCel to support customers that seek to promote streamlining personalized therapies from specimen connections to manufacturing, said Felix Hsu, Senior Vice President and Global Head of WuXi AppTec Advanced Therapies. With our leading integrated manufacturing and testing services, we believe we can bring even more speed, reliability and security to an already complex manufacturing and supply chain. We are committed to helping our partners deliver more innovative therapies to patients and the market sooner.

About TrakCel

TrakCel is the market leading designer, developer and deliverer of integrated technologies specifically created in 2012 to manage the international autologous and allogeneic cell, gene and immunotherapy supply chain. TrakCel's software platform has been developed in collaboration with, and increasingly adopted by leading companies in the cell, gene and immunotherapy industries. TrakCels solutions deliver real-time control over the entire therapeutic supply chain, from sample collection through manufacturing to treatment delivery. The TrakCel platform accelerates global scale-up and scale-out of cell and gene therapy products, increasing efficiency and decreasing complexity, while maintaining needle-to-needle compliance and traceability.

TrakCel is headquartered in Cardiff, Wales, UK with US offices in California and New Jersey.

Please visit: https://trakcel.com

About WuXi AppTec

WuXi AppTec provides a broad portfolio of R&D and manufacturing services that enable companies in the pharmaceutical, biotech and medical device industries worldwide to advance discoveries and deliver groundbreaking treatments to patients. As an innovation-driven and customer-focused company, WuXi AppTec helps our partners improve the productivity of advancing healthcare products through cost-effective and efficient solutions. With industry-leading capabilities such as R&D and manufacturing for small molecule drugs, cell and gene therapies, and testing for medical devices, WuXi AppTecs open-access platform is enabling more than 3,600 collaborators from over 30 countries to improve the health of those in need and to fulfill our dream that "every drug can be made and every disease can be treated."

Please visit: http://www.wuxiapptec.com

About WuXi Advanced Therapies (WuXi ATU)

The advanced therapies business unit of WuXi AppTec is a Contract Development and Manufacturing Organization (CDMO) that is reducing the complexities of manufacturing by providing integrated platforms that enable cell and gene therapies to be developed, manufactured, and released faster and with greater predictability.

Please visit http://www.advancedtherapies.com.

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Potential Solutions To Current Pricing Models For Cell And Gene Therapies – Life Science Leader Magazine

By Erin Harris, Editor-In-Chief, Cell & Gene Follow Me On Twitter @ErinHarris_1

Move over, religion and politics. Drug prices are one of the new polarizing topics on the minds of anyone paying attention.

Headlines about high-priced cell and gene therapies specifically can cause sticker shock for the patient. Exhibit A: Novartis Zolgensma, the spinal muscular atrophy (SMA) gene therapy treatment, priced at $2.125 million, is the worlds most expensive drug to date. But cell and gene therapies are their own animals; the differences between them and traditional biologics are too numerous to count. Their innovative and life-saving properties are unmatched and, make no mistake, these therapies are changing modern medicine. As a result, the often one-time, personalized therapies are expensive and resource-intense propositions. Attend any industry conference that broaches the pricing subject, and youre likely to hear the following: Competition will eventually drive down costs, or its simply too early to tell whats next. Both statements are true, but theyre not very actionable. Therefore, I turned to Eric David, CEO of Aspa Therapeutics; Paul Lammers, M.D., MSc, president, CEO, and director of Triumvira Immunologics; and Janet Lambert, president of the Alliance for Regenerative Medicine (ARM), for their take on current pricing models for cell and gene therapies and potential solutions to drive down costs for the patient.

THE STATE OF CURRENT PRICING MODELS

Prices for cell and gene products represent the value that the therapies bring to patients and to healthcare systems over time, and they help to drive further innovation across the sector. The therapies have the potential to provide a durable or possibly even a curative effect, often with a single administration, and represent a significant improvement in standard of care for many patients, says Lambert. She goes on to say that this method of treating disease by addressing its underlying cause has the potential to provide cost savings to the healthcare system through decreased direct medical costs as well as through improved patient quality of life, increased productivity, improved caregiver quality of life, improved social integration, and other indirect cost savings.

These therapies represent a substantial change in the healthcare reimbursement paradigm, as many treatments for serious conditions currently involve chronic palliative care, providing incremental improvements and/or temporary delays in the progression of disease. Current reimbursement systems are configured toward providing this type of chronic care, and may be unable to cope with the high up-front costs of cell and gene therapies, says Lambert. Coupled with the newness of these technologies and the lack of long-term follow-up data, these therapies can have an undesirably high potential risk profile for payors. Therefore, ensuring patient access to these therapies relies on the development and implementation of payment models that help payers to absorb the costs of these therapies as well as offset the perceived risks.

WHY COSTS WILL COME DOWN EVENTUALLY

At this years BIO conference in Philadelphia, I attended a session titled Gene Therapy 2.0: No Longer Science Fiction. I noticed that when the topic of pricing came up, some panelists at this session became noticeably quiet except for Eric David. In fact, his passion for the topic was so palpable, I decided to contact him after the event to pick his brain.

The challenges to the industry regarding gene therapy pricing are twofold, explains David. First and foremost, the cost to manufacture a gene therapy is significantly more than conventional biologics such as monoclonal antibodies and recombinant proteins. Cost of goods/manufacturing alone for a gene therapy can be between $500,000 and $1 million, and that does not include costs for R&D, the costs to run crucial clinical trials, or the costs to build the commercial infrastructure necessary to provide access to patients. In addition, for the foreseeable future, these therapies will be administered as one-time-only, and they will be administered to very small patient populations sometimes just a few hundred patients worldwide. Companies must be able to recoup their significant investments, or they will not be able to tackle these highly unmet needs.

David went on to explain that current pricing models spread out the payments for a gene therapy over several years, assuming efficacy remains durable. This ensures that payors do not have to bear the entire cost of a gene therapy up front. Over time, costs will come down significantly, as they did for monoclonal antibodies, driven by increased manufacturing capacity, greater commoditization of bioengineering and manufacturing resources, and efficacy-based pricing models, says David.

REIMBURSEMENT MODELS: PAYMENT-OVER-TIME AND PAY-FOR-PERFORMANCE

According to Lambert, two reimbursement models that are currently garnering attention and traction from industry and payors alike are payment-over-time and pay-for-performance. Payment-over-time models, which allow insurers to amortize the cost of therapies over several years, better reflect the value provided by cell and gene therapies, she says. Pay-for-performance models can be combined with amortization models, benchmarking future payments on positive health outcomes for patients, or they can be used stand-alone, providing rebates in cases in which the therapy was not as efficacious as expected. In either case, these models help to shift or share the risk from the payor to the developer, which may make payors more confident.

Other innovative models are already in place to help mitigate the costs of existing expensive therapies. For instance, re-insurance, a process by which risk is shared by multiple insurance companies, has been adopted by insurers in the U.S. to help pay for expensive solid organ and stem cell (bone marrow) transplantation procedures. Risk pools have been used successfully by private insurers in Canada and are the basis for the UKs government-sponsored Cancer Drugs Fund, Lambert explains. The so-called Netflix Model, which has been adopted by two state Medicaid programs [i.e., Louisiana and Washington] to pay for high-cost treatments for Hepatitis C can help normalize costs when demand for a therapy fluctuates significantly from year to year.

A STRATEGY TO BRING DOWN COSTS: MANUFACTURING IMPROVEMENTS

Dr. Lammers, of Triumvira Immunologics, says one strategy that could reduce the costs of these therapies involves fully automating T-cell therapy production. There is a strong movement in the T-cell manufacturing world to move toward the use of fully automated systems, (i.e., you put the patients leukapharesis material in, and 10-14 days later, the finished CAR-T or TAC-T cells are ready to be re-infused into the patient), says Dr. Lammers. Then, you insert a new cartridge, and the system is ready to process the next patients materials. These systems are small about the size of a small microwave and can be stacked, so the actual footprint needed would be far smaller than the current process used. Dr. Lammers and David note that increased manufacturing capacity also will drive down costs.

Eric DavidCEO, Aspa Therapeutics

INVOLVING THE PATIENT IN THE PROCESS

We all know that, from a patients perspective, it can be hard to swallow the published price of a therapy, But payors understand the why behind high costs of gene therapy as well as how few patients will be treated, and most patients and their families will never have to pay the whole price of a therapy, says David. In addition, the tremendous innovation occurring across scientific, regulatory, and commercial models is enabling investment in these areas of profound unmet medical need. Without this innovation, which is stimulated in part by the markets acceptance of the current pricing models, there would remain absolutely no investment in these diseases, as had been the case for decades previously.

Simply put, cell and gene therapies are expensive to develop, manufacture, and commercialize, and they will continue to be for the foreseeable future. As industry and payors continue to pursue innovative financing models, it is important for policymakers to identify and reduce the legal and regulatory barriers to the adoption of these programs, particularly for public payors.

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Potential Solutions To Current Pricing Models For Cell And Gene Therapies - Life Science Leader Magazine

Astellas invests $13m in cell and gene therapy innovation incubators – Cleanroom Technology

3-Oct-2019

Japanese Astellas $12.5m will make it a Founding Sponsor of LabCentral's new incubator with a core lab space for process development studies and a non-GMP pilot plant

An established LabCentral facility

Astellas Pharma has announced it will invest nearly US$13 million into two innovation incubators operated by LabCentral, a US-based laboratory facility for next-gen biotech startups.

Astellas will invest $12.5m to become the only pharmaceutical/biotechnology company among five Founding Sponsors of a new incubator, which will feature a core lab space where companies can easily conduct process development studies and a non-GMP pilot plant, being developed by LabCentral at 238 Main Street in Cambridge, MA.

This initiative, combined with the more than $1bn Astellas has previously committed to driving innovation in Massachusetts, contributes to the Boston-area life science community's ongoing efforts to accelerate the discovery and development of potential breakthrough therapies in areas of significant unmet need.

"Astellas has a long-standing commitment to the Boston-area life sciences ecosystem, where world-class talent are dedicated to turning innovative science into value for patients," said Kenji Yasukawa, President and CEO of Astellas. "Our presence in the greater Boston area comprises over 200 professionals across several locations driving innovation in regenerative medicine, immuno-oncology, mitochondrial function, genetic regulation and beyond. Accelerating early-stage scientific innovation in areas such as cell and gene therapy is a strategic focus for Astellas, and is superbly aligned with the mission of LabCentral to serve as a launching-pad for cutting-edge biotech and life sciences start-ups."

The investment, announced today in a ceremony with the Massachusetts Life Sciences Center and elected officials including Lieutenant Governor Karyn Polito, provides support to start-up companies and entrepreneurial founders seeking to create scientific innovation in areas of unmet need such as cell and gene therapy. The new incubator is expected to be operational in 2021.

Astellas also announced it will invest at least $450,000 over three years to become a Gold Sponsor of LabCentral's existing incubator located at 700 Main Street in Cambridge.

By supporting these incubators, Astellas can select, support and access innovation from leading start-ups creating healthcare solutions in its areas of focus.

Since 2010, Astellas has invested more than $800 million in, and committed nearly $500 million more to, Massachusetts-based innovation through the acquisitions of Ocata Therapeutics, Inc., Mitobridge, Inc. and Potenza Therapeutics, Inc., as well as the construction of a state-of-the-art headquarters for the Astellas Institute for Regenerative Medicine (AIRM) in Westborough, MA. The new facility, expected to open in 2020, will enable AIRM to accelerate research and development in the field of regenerative medicine and cell therapy.

Astellas sais that the impact of this agreement on its financial results in the fiscal year ending March 31, 2020 will be limited.

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Astellas invests $13m in cell and gene therapy innovation incubators - Cleanroom Technology

RNA Therapies, Immunotherapies and Cancer Tests Showcased in… – Labiotech.eu

Biotech companies around the world gathered in Barcelona, Spain, to present breakthroughs in cancer diagnostics and treatments such as cell therapies and cancer vaccines. Heres our take on the most interesting offerings from the European crop.

Barcelona is the home of Gaudi and Barcelona Football Club. Over the last few days, it was also the home of the European Society for Medical Oncology (ESMO) Congress 2019.

While big pharma drew attention at the conference, especially with the drug Lynparza, European biotechs brought advanced technology to the table. This included cell therapies, RNA therapy, cancer vaccines, and diagnostic tests that could help to detect cancer before it spreads.

Tests to catch cancer early

A phase III study carried out at the Hpital Europen Georges-Pompidou in Paris, France, found that just 64% of colorectal cancer patients with the tumors DNA in their blood survived after two years. In contrast, 82% of patients lacking this tumor DNA in their blood survived. The tumor DNA could, therefore, be a useful biomarker for tailoring treatments to patients with this condition.

When patients have surgery for early-stage colorectal cancer, doubts remain as to whether the disease has been completely eradicated, stated Alberto Bardelli, Professor at the University of Turin, Italy, and an expert in the field who was not directly involved in the study. However, the results have shown we can now use a blood test to say whether the patient is clear or not.

The Dutch biotech SkylineDx reported that its diagnostic test saved 41% of skin cancer patients in a pilot study from unnecessary surgery. This test analyzes genes in skin tumor biopsies and is able to predict whether or not the tumor will spread to the lymph nodes. This means that physicians could soon check whether patients need surgery to remove the lymph nodes, instead of just removing them as a precaution like they do currently.

A blood test presented by the Swiss company Novigenix was able to detect colon cancer in 83% of patients with the condition in a trial with 282 participants. Novigenix test works by detecting mRNA in immune cells that is produced in response to cancer in the body. This makes it a potentially useful test for detecting the cancer early, and is less unpleasant for the patient than colonoscopies, the standard diagnostic technique.

Epigenetics, RNA and immunotherapies

Results from an ongoing phase I trial of a cell therapy developed by the UK company Adaptimmune were also on show. Twelve patients with the rare cancer synovial sarcoma have received the treatment so far, and 11 of them had their tumors partially shrink. This makes it a promising treatment for synovial sarcoma, which has few options at present.

Adaptimmunes technology, called SPEAR T-cell therapy, consists of modifying the patients immune T-cells to target cancer antigens. Unlike CAR-T immunotherapies, which are approved to treat forms of blood cancer, Adaptimmunes immunotherapy is able to treat solid tumors by targeting a cancer antigen called MAGE-A4.

The London-based company MiNA Therapeutics reported final phase I data of its RNA therapy in patients with advanced liver cancer. On its own, the therapy was not so impressive out of 35 patients, the therapy shrank the tumor of one patient and stabilized the cancer in 15 other patients. However, it showed potential for making the tumor more vulnerable to sorafenib, a standard liver cancer drug. Of five patients given sorafenib after the RNA therapy, three were tumor-free for more than a year.

MiNA Therapeutics therapy consists of RNA molecules that increase the production of the protein C/EBP- in cancer cells via a process called RNA activation. This is believed to increase the ability of other therapies such as antibody drugs to destroy liver cancer. The company is now planning to test the treatment at a higher dose to establish the best dose for a phase II trial.

In the wake of a high-profile phase III failure for its lead cancer treatment, the French biotech Transgene presented phase I results for a therapeutic vaccine for head and neck cancer. According to the results, the vaccine shrank the tumors in three out of six patients given the highest dose, and showed a good safety profile.

Transgenes vaccine is designed to alert the immune system to infections by a type of human papilloma virus that can cause cancers such as cervical and head and neck cancer. The company now plans to proceed with the phase II stage of the trial, which will enroll 40 patients.

The Spanish biotech Oryzon Genomics presented interim phase II results for its epigenetic drug combined with chemotherapy in lung cancer patients. Of the eight treated patients so far, six have stopped the growth of the cancer. Oryzons drug works by blocking an epigenetic protein called LSD1, which controls the expression of key genes in the cell.

We must be prudent as this phase II trial is still in its early stages, however, the level of responses is really promising, particularly as some of them are long-lasting, Roger Bullock, Oryzons CMO, stated.

Other positive phase I results were reported by the Finnish company Faron Pharmaceuticals, which is developing an immunotherapy for colorectal cancer that targets immune cells called macrophages. The German company 4SC also presented its results regarding a type of epigenetic drug called an HDAC inhibitor.

Small molecule drugs breaking tumor resistance

A radio-sensitizing drug developed by the Swiss company Debiopharm showed promising phase II results in its conference presentation. Eighteen months after being treated with Debiopharms drug in combination with chemo and radiotherapy, 21% more patients had their cancer stopped in its tracks compared with the traditional therapies alone.

Debiopharms drug blocks the action of proteins called Inhibitor of Apoptosis Proteins in the tumor cells. These proteins stop the tumors from committing suicide when exposed to cancer treatments. By blocking these proteins, the drug is therefore designed to make the tumor less resistant.

Other companies presenting phase II updates included the Norwegian biotech BerGenBio, which is developing a small molecule drug targeting a protein promoting drug resistance called AXL receptor tyrosine kinase. Positive results also came from the French company Noxxon Pharma, which is testing a small molecule drug for colorectal cancer, and the UK biotech Bicycle Therapeutics, which is developing drugs carrying a toxic payload to treat solid tumors.

Both cancer treatments and cancer diagnostic tests were showcased at ESMO, showing similar potential for tackling the complex disease. While new cancer treatment options continue to appear, diagnostic technology is also proving its worth in telling physicians which patients might benefit the most from this increased range of treatments. It will be fascinating to see how these technologies have advanced in the next ESMO conference, so watch this space!

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Brooks Koepka Reveals He Had Stem Cell Treatment on Knee Injury in PGA Offseason – Bleacher Report

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Brooks Koepka tied for the most wins on the PGA Tour in 2019 despite having a knee injury that required stem cell surgery during the offseason.

Per the Associated Press (h/t ESPN.com), the four-time major champion revealed he had treatment on his left patella one day after the Tour Championship ended on Aug. 25.

Koepka noted his knee started giving him problems in March and he needed three to four weeks of rehabilitation after having the procedure.

The 29-year-old won the PGA Championship and PGA of America Player of the Year award for the second consecutive year. His three tournament victories tied Rory McIlroy for most on the circuit, and he finished in the top four in all four major championships.

In between those performances, Koepka did have some struggles as the season wore on. The Florida native finished 24th or worse in five of his final nine tournaments, dropping from first in the standings when the FedEx Cup Playoffs began to a tie for third at the end of the TOUR Championship.

Koepka will begin the 2019-20 PGA Tour season at theShriners Hospitals for Children Open starting Thursday in Las Vegas.

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Michael Schumacher health latest: What is stem cell therapy and how will it help Schumi? – Express.co.uk

Formula One legend Michael Schumacher suffered life-changing head injuries in a skiing accident in 2013 and has since been receiving care at his home in Switzerland. It is believed the record-breaking racing driver cannot walk or stand and may have trouble communicating according to former Ferrari manager Jean Todt. So far Mr Schumachers treatment has been kept private, and nothing has been confirmed officially, however now the racing legend is under the care of world-renowned cardiac surgeon Philippe Menasch.

Mr Menasch is described as a pioneer in cell surgery at his hospital, Georges-Pompidou, in Paris.

According to the NHS: Stem cells are special cells produced by bone marrow (a spongy tissue found in the centre of some bones) that can turn into different types of blood cells.

The cells have been used since the 1980s to grow skin grafts for patients who have suffered life-threatening burns.

Read More:Michael Schumacher health update: Where is Michael Schumacher now?

They are also used in cancer treatments for cancers of the blood, and most recently have been used in repairing damage to the cornea - surface of the eye.

A lot about stem cells is still being discovered, with clinical trials taking place for illnesses and conditions like MS and macular degeneration, heart disease and spinal cord injuries.

The special cells are also being used in neurodegenerative diseases like Parkinsons and Alzheimers and traumatic brain injuries like Mr Schumachers.

In an interview online, Mr Menasch explained stem cell treatment for cardiac conditions is only touching the surface.

Read More:Michael Schumacher could be able to cry and move his thumbs'

He said: Nobody really knows how stem cells are working.

They do not permanently transplant into the myocardium, the muscular tissue of the heart after a couple of days or weeks they just disappear.

Mr Schumacher received stem cell therapy in September in Paris, however, not much information has surfaced about the procedure.

MrMenasch spoke to Italian newspaper La Repubblica and said: There was an explosion in the attention our department received but the situation has already normalised.

Local media dubbed the procedure experimental however MrMenasch denied this saying: everyone is looking for me but I have not used experimental cures.

I do not perform miracles. My team and I are not doing an experiment, an abominable term that is not in line with a serious medical view.

Michael Schumacher is motor racings most successful driver, with a record 91 Grand Prix wins.

He won his first titles with Benetton in 1994 and 1995 followed by five in a row with Ferrari between 2000-2004.

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Michael Schumacher health latest: What is stem cell therapy and how will it help Schumi? - Express.co.uk