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Category Archives: Cell Therapy

Stem Cell Therapy – Runner’s World

Posted: March 14, 2017 at 2:47 pm


Runner's World
Stem Cell Therapy
Runner's World
While PRP therapy stimulates the healing process of tissue that is already there, stem cells may create new tissue. This is why researchers and physicians think this therapy may help joint injuries caused by worn-out cartilage; in cell cultures, stem ...
$145.8 Million Stem Cell Therapy Market by Type, Therapeutic Application, Cell Source - Global Forecasts to 2021 ...Business Wire (press release)
Has stem cell therapy helped the Boden's poster girl say her first words? Cerebral palsy patient Holly, 11, shows ...Daily Mail
Stem cell therapy for the treatment of Peyronie's disease.UroToday
CanIndia News -Medgadget (blog)
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New Cardiac Stem Cell Therapy passes Phase I/II Trials – Labiotech.eu (blog)

Posted: March 13, 2017 at 8:42 pm

TiGenix announces positiveone-year results forits phase I/II trial of donor-derived cardiac stem cell therapy in acute myocardial infarction (AMI).

The Belgian biotech TiGenixis developing allogeneic stem cell therapies. Now the companyhasannouncedthat its cardiac stem cell therapyAlloCSC-01 reached its primary endpoints in aphase I/IItrial.

In 2015, the companyacquired Coretherapixin a292M deal for its allogeneic cardiac stem cell pipeline, which is being developed for the treatment of AMI.The first-in-human trial was designed to test the safety and feasibility of an intracoronary infusion of donor-derivedexpanded cardiac stem cells (AlloCSCs)in patients with AMI and left ventricular dysfunction.

AlloCSC-01consists of adult allogeneic cardiac stem cells isolated from the heartof donors and expanded in vitro. In vivo studies suggest that these cellshave cardio-reparative potential by activating regenerative pathways and promoting the formation of new hearttissue.

Thecurrent phase II study demonstrated thesafety of these allogeneic stem cells. Initial results also revealed a larger reduction of infarct size in a subgroup of patients.

Myocardial infarction caused by blockade of coronary arteries

TiGenix is well known forChondroCellect, which was the first cell therapyto reach the European market for the repair of knee cartilage.After the companyrecently withdrew its market authorization for this product, due to a lack of reimbursement, the biotech is focusing on another stem cell therapy, Cx601, in addition to AlloCSC-01. Under development for Crohns disease, Cx601 is currently awaitingEMA approval and is in phase III trials in the US.

For a late-stage clinical company, TiGenix has a low market cap of191M. Even so, the company seems to be doing well these days with the progress of Cx601 and AlloCSC-01.

If AlloCSC-01 obtains market approval, it could treat the more than 1.9 millionpeople affected by AMI, a major cause of heart failure. So far, most treatments are palliative or restore myocardial function by angioplasty and insertion of a stent to support the vascular lumen.

Stem cell therapy of the heart is definitely not a new topic, but many trials have been conducted using the patients own stem cells derived from the bone marrow. A recent meta-analysisof such trials has suggested that these therapies are safe, but do not enhance cardiac function. TiGenixs approach using allogeneic heart-derived stem cells may offer a new and promisingopportunity in thefield.

Images via shutterstock.com / Liya Graphics andVeronika Zakharova

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Fate Therapeutics cleared to begin clinical trial assessing NK cell therapy in blood cancer; shares up 9% – Seeking Alpha

Posted: March 13, 2017 at 8:42 pm

Thinly traded micro cap Fate Therapeutics (FATE +9.4%) jumps out the blocks on more than a 6x surge in volume in response to its announcement that the FDA has approved its Investigational New Drug (IND) application for FATE-NK100, an adaptive memory natural killer (NK) cell product. The approval paves the way for a first-in-human clinical trial in patients with advanced acute myeloid leukemia (AML).

The early-stage study, to be conducted at the University of Minnesota, will determine the maximum tolerated dose of a single intravenous administration of FATE-NK100 in an accelerated dose escalation design in four patients.

FATE-NK100 is a next-generation immunotherapy based on a subset of NK cells that have shown robust anti-tumor activity in addition to promoting endogenous T cell response while resisting immune checkpoints.

NK cells identify and kill cancer cells by recognizing a range of stress signals expressed on tumor cells. Normal healthy cells are unaffected/unharmed. This is a different mechanism of action compared to T cells which require a specific tumor antigen to elicit an immune response and must be customized for each patient. The company says a significant advantage of NK-based immunotherapy is the avoidance of graft-versus-host disease, a common and potentially serious side-effect of T cell therapies.

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Stem cell therapy for the treatment of Peyronie’s disease. – UroToday

Posted: March 12, 2017 at 6:42 pm

Like other fibrotic diseases, the cause of Peyronie's disease (PD) is still obscure. Since there is now increasing evidence for the role of Mesenchymal Stem Cells (MSCs) as potential treatment to fibrosis, it is crucial to determine their possible efficacy in the treatment of PD. Areas covered: In this review, the authors summarize the emerging data and published studies regarding the use of SCs for the treatment of PD. The authors provide particular focus on the three-first experimental studies for the use of SCs in rat models as well as the sole two studies undertaken in humans. Expert opinion: It seems evident in experimental settings that SCs in general (Adipose Derived SCs in particular) provide a feasible, safe and effective therapy for PD. The potential limits of the rat models used initially have been somewhat overcome with the inception of studies in men. However, further prospective studies are needed in humans to further elucidate the therapeutic potential of stem cell therapy in PD.

Expert opinion on biological therapy. 2017 Feb 28 [Epub]

Athanasios Dellis, Athanasios Papatsoris

a University Department of Urology , Sismanoglio General Hospital , Athens , Greece.

PubMed http://www.ncbi.nlm.nih.gov/pubmed/28274142

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Partnership Formed to Advance Bioelectronic Medicine and Cell Therapy – Pharmaceutical Processing

Posted: March 10, 2017 at 12:41 pm

Northwell Health and United Therapeutics announce strategic partnership.

Northwell Health's Feinstein Institute for Medical Research and United Therapeutics Corporation announced today a strategic partnership focused on the application of bioelectronic medicine and cell therapy to cardiology, hypertension and post-transplant tolerance induction.

"We are truly honored to work with the pioneers of these next generation medical technologies," said Martine Rothblatt, PhD, United Therapeutics' Chairman and Chief Executive Officer. "We expect a great fit with our clinical development pipeline in heart failure, pulmonary disease and transplantation."

"Collaboration is the indispensable factor in successful medical research," said Kevin J. Tracey, MD, President and CEO of the Feinstein Institute. "With great partners, you can accomplish great things for science and for patients. United Therapeutics is such a partner, we share their aims and their values, and we could not be more pleased than to join with them in this effort.

Under the strategic partnership, United Therapeutics will fund Northwell's efforts in four research and development tracks, while United Therapeutics will bring the results into clinical development. The two organizations are working toward the goal of initial regulatory approvals within five years.

Two of the research projects will be conducted by the Feinstein Institute's Center for Bioelectronic Medicine (CBEM). The Feinstein Institute is a worldwide leader for the advancement of scientific knowledge and intellectual property for the rapidly emerging field of bioelectronic medicine.

Bioelectronic medicine represents the convergence of three well-established scientific fields: neuroscience, molecular and cell biology, and bioengineering. The Feinstein Institute team, led by Dr. Tracey, a neurosurgeon who pioneered the field, has been working in this area since 1998, and Northwell Health has already invested $75 million in support of the underlying research. As bioelectronic solutions are successfully identified, tested and refined, CBEM will foster the creation of new companies to bring life-changing solutions to market.

United Therapeutics Corporation is a biotechnology company focused on the development and commercialization of products to address the unmet medical needs of patients with chronic and life-threatening conditions.Northwell Health is New York State's largest health care provider with 21 hospitals and over 550 outpatient facilities.The Feinstein Institute for Medical Research is the research arm of Northwell Health.

(Source: EurekAlert!)

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CMBG Receives US$2.29 Million Grant For Stem Cell Therapy – Asian Scientist Magazine

Posted: March 9, 2017 at 6:43 am

Chinas Cellular Biomedicine Group has received US$2.29 million to support pre-clinical studies of stem cell therapy for knee osteoarthritis.

Asian Scientist Newsroom | March 9, 2017 | Pharma

AsianScientist (Mar. 9, 2017) - Cellular Biomedicine Group Inc., a clinical-stage biopharmaceutical firm engaged in the development of effective immunotherapies for cancer and stem cell therapies for degenerative diseases, has been awarded US$2.29 million by the governing Board of the California Institute for Regenerative Medicine (CIRM) to support pre-clinical studies of AlloJoin, CBMGs Off-the-Shelf allogeneic human adipose-derived mesenchymal stem cells for the treatment of knee osteoarthritis in the United States.

While CBMG recently commenced two Phase I human clinical trials in China using CAR-T to treat relapsed/refractory CD19+ B-cell acute lymphoblastic leukemia (ALL) and refractory diffuse large B-cell lymphoma (DLBCL) as well as an ongoing Phase I trial in China for AlloJoin in knee osteoarthritis, this latest announcement represents CBMGs initial entrance into the United States for its off-the-shelf allogeneic stem cell candidate AlloJoin.

The US$2.29 million was granted under the CIRM 2.0 program, a comprehensive collaborative initiative designed to accelerate the development of stem cell-based treatments for people with unmet medical needs. After the award, CIRM will be a more active partner with its recipients to further increase the likelihood of clinical success and help advance a pre-clinical applicants research along a funding pipeline towards clinical trials.

CBMGs knee osteoarthritis pre-clinical program is considered late-stage, and therefore it meets CIRM 2.0s intent to accelerate support for clinical stage development for identified candidates of stem cell treatments that demonstrate scientific excellence.

We are deeply appreciative to CIRM for their support and validation of the therapeutic potential of our knee osteoarthritis therapy, said Mr. Tony (Bizuo) Liu, Chief Executive Officer of CBMG. The CIRM grant is the first step in bringing our allogeneic human adipose-derived mesenchymal stem cell treatment for knee osteoarthritis (AlloJoin) to the U.S. market.

In order to demonstrate comparability with cell banks previously produced in China for our U.S. IND filing, we are addressing the pre-clinical answers required for the FDA. With the funds provided by CIRM, we will replicate and validate the manufacturing process and control system at the cGMP facility located at Childrens Hospital Los Angeles to support the filing of an IND with the FDA.

CBMG recently announced promising interim three-month safety data from its Phase I clinical trial in China for AlloJoin, its off-the-shelf allogeneic stem cell therapy for knee osteoarthritis. The trial is on schedule to be completed by the third quarter of 2017.

Source: Cellular Biomedicine Group Inc; Photo: Shutterstock. Disclaimer: This article does not necessarily reflect the views of AsianScientist or its staff.

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Stem Cell Therapy Market by Type, Therapeutic Application, Cell Source – Global Forecasts to 2021 – Yahoo Finance

Posted: March 8, 2017 at 6:41 am

NEW YORK, March 7, 2017 /PRNewswire/ -- l stem cell therapy market is estimated to grow at a CAGR of 11.0% during 2016 to 2021 to reach USD 145.8 million by 2021. Growth in the global stem cell therapy market is driven by factors such as the growing awareness of the therapeutic potency of stem cells in effective disease management, development of advanced genome-based cell analysis techniques, increasing public-private investments for development of stem cell therapies, identification of new stem cell lines, and developments in infrastructure related to stem cell banking and processing. In addition, countries such as Japan, South Korea, and China are offering new growth opportunities for players operating in this market. The North American region is expected to command the largest share in the stem cell therapy market in 2016.

Read the full report: http://www.reportlinker.com/p04759526-summary/view-report.html

Based on the type of therapy, the allogeneic stem cell therapy segment is estimated to command the larger share of the global stem cell therapy market in 2016. This growth can be attributed to the growing availability of allogeneic stem cell therapy products, wider therapeutic applications of allogeneic stem cells, easier production scale-up due to easy availability of sources of stem cells, and growing number of clinical trials of allogeneic stem cell therapies as compared to autologous stem cell therapies.

The stem cell therapy market is niche industry with a growing number of global and local companies involved in the development and commercialization of stem cell therapy products. Osiris Therapeutics, Inc. (U.S.), MEDIPOST Co., Ltd. (South Korea), Anterogen Co., Ltd. (South Korea), and Pharmicell Co., Ltd. (South Korea) were the leading players in the global stem cell therapy market in 2015. New product launches and approvals; expansions; and partnerships and agreements are the major strategies adopted by most of the market players to achieve growth in the stem cell therapy market during 20132016.

Research Coverage This report studies stem cell therapy market based on type of therapy (allogeneic and autologous). These stem cell therapies are used for the treatment of various diseases (including musculoskeletal disorders, wound healing, CVDs, and GI diseases, among others). The report also studies, the factors (such as drivers, restraints, opportunities, and challenges) which affect the market growth in a positive and negative manner. It analyzes opportunities and challenges in the market for stakeholders and provides details of the competitive landscape for market leaders. The report forecasts the revenue of the market segments with respect to four main regions, namely, North America, Europe, Asia-Pacific, and the Rest of the World. The stem cell therapy market report strategically profiles the key players who are involved in the manufacturing and commercialization of stem cell therapy products and comprehensively analyze their market ranking and core competencies. The report tracks and analyzes competitive developments such as new product launches and enhancements; expansions; and partnerships and agreements in the stem cell therapy market.

Reasons to Buy the Report:

From an insight perspective, this research report focuses on various levels of analysismarket share analysis of the top players and company profiles, which together comprise and discuss basic views on the competitive landscape; emerging and high-growth segments of the stem cell therapy market; and high-growth regions and their respective drivers, restraints, challenges, and opportunities.

The report will enrich both established firms as well as new entrants/smaller firms to gauge the pulse of the market, which in turn will help firms in garnering a greater market share. Firms purchasing the report could use any one or a combination of the below-mentioned five strategies (market penetration, product development/innovation, market development, market diversification, and competitive assessment) for strengthening their market shares.

The report provides insights on the following pointers:

- Market Penetration: Comprehensive information on products offered by the top 10 players in the stem cell therapy market. The report analyzes the stem cell therapy market by type, therapeutic application, cell source, and region

- Product Development/Innovation: Detailed insights on research and development activities, developmental product pipeline, and new product launches in the stem cell therapy market

- Market Development: Comprehensive information about the lucrative emerging markets. The report analyzes the markets for various stem cell therapy products across four geographies (North America, Europe, Asia-Pacific, and the Rest of the World)

- Competitive Assessment: Assessment of market shares, strategies, products, distribution networks, and manufacturing capabilities of the leading players in the stem cell therapy market

Read the full report: http://www.reportlinker.com/p04759526-summary/view-report.html

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US Market Report for Orthopedic Cell Therapy 2017 – MedCore – PR Newswire (press release)

Posted: March 8, 2017 at 6:41 am

LONDON, March 7, 2017 /PRNewswire/ -- Description

Download the full report: https://www.reportbuyer.com/product/4759855/

General Report Contents - Market Analyses include: Unit Sales, ASPs, Market Value & Growth Trends - Market Drivers & Limiters for each chapter segment - Competitive Analysis for each chapter segment - Section on recent mergers & acquisitions

There has been much greater interest in bone marrow concentration for use in spinal fusions compared to platelet-rich plasma. The platelet-rich plasma market, on the other hand, is expanding into soft tissue indications. While bone graft substitutes can be soaked in PRP or bone marrow concentrate as an application, the most rapidly growing application, especially for platelet-rich plasma, is for injectables. Such products are directly injected into the site, for example, in a ligament repair or wound operation. Competition over price is the reason the market values for these segments are not growing as quickly as might be expected. Arthrex significantly brought down the ASP for the market when they introduced their ACP platelet concentration system, but prices have now largely stabilized in the PRP segment. Test-tube-based products allow for concentration of smaller volumes; these systems reduce ASP further as customers can shift away from higher ASP disposable bucket products.

Abstract The scope of the orthopedic cell therapy market in this report covers platelet-rich plasma and concentrated bone marrow aspirate. The platelet and bone marrow concentration market consists of centrifuge equipment that separates and concentrates platelets and cells based on their various densities. The majority of companies participating in this market provide the centrifuges on loan and sell only the disposable components, such as the spin buckets. Typically, one bucket is required per procedure.

Scope: 2013-2023 Download the full report: https://www.reportbuyer.com/product/4759855/

About Reportbuyer Reportbuyer is a leading industry intelligence solution that provides all market research reports from top publishers http://www.reportbuyer.com

For more information: Sarah Smith Research Advisor at Reportbuyer.com Email: query@reportbuyer.com Tel: +44 208 816 85 48 Website: http://www.reportbuyer.com

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/us-market-report-for-orthopedic-cell-therapy-2017---medcore-300419889.html

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Stem Cell Therapy: Modern Solution to Joint Pain Relief – Yahoo Finance

Posted: March 8, 2017 at 6:41 am

PLAINVIEW, N.Y., March 6, 2017 /PRNewswire/ -- Board certified surgeon Dr. Andrew J. Rochman promotes the specialized treatment of Stem Cell Therapy in his private practice in Plainview, NY. The recent wave of positive response from its ability to target a growing number of health conditions has brought increased attention and demand from pain sufferers everywhere- including the Long Island area.

As of the fall of 2016, Dr. Rochman officially opened the Cell Therapy Center of NY (CTCNY) where he focuses on the treatment of osteoarthritis & rheumatoid arthritis, eroded cartilage and joint issues like tennis elbow, jumpers knee and golf elbow. He dedicated his practice to the large population suffering from musculoskeletal damage arising from sports injuries or the wear and tear from aging. "We are always seeking a safer and more effective alternative to surgery to battle physiological symptoms with the hopes of giving patients a pain-free life."

Dr. Rochman underwent extensive training from U.S. Stem Cell, Inc. (Sunrise, FLA) - a center for the development of effective cell technologies recognized for treating a variety of diseases and injuries. U.S. Stem Cell's discoveries include multiple cell therapies in various stages of development that repair damaged tissue due to injury or disease. Chief Science Officer Kristin C. Comella, expert in regenerative medicine with a focus on adipose derived stem cells, pioneered stem cell therapies derived from various sources including cord blood, bone marrow and muscle. "By harnessing the body's own healing potential, we may be able to reverse damaged tissue to normal function.... stem cells have the ability to form many types of tissues like bone, cartilage, muscle and even help to reverse some effects that have been caused by damaged tissue," states Ms. Comella.

Dr. Rochman's treatment center has recently seen an influx of patients from Long Island's large athletic and fitness community. President of the Wildwood Warriors triathlon team, John Graziano is one of the many joint and back pain sufferers from sports injuries seeking this alternative pain treatment. "In the world of triathlon, I train- I race- and I live with pain and lots of it!"

"The potential here is limitless," states Dr. Rochman. "It's actually a simple yet unique form of therapy with the possibilities of doing miraculous things. We found out within the past several years that human beings have stem cells in every tissue of their body and they actually live around the blood vessels." Today's stem cell therapy has been shown to manage and target a wide span of healing possibilities from blood cell diseases to cardiac disorders to autoimmune diseases. "So what we can do now is to extract fat cells from the belly or bone marrow cells and isolate the cells from those damaged tissues... perhaps in the future we can utilize this process to target neurological diseases, heart diseases... and we don't even know where it ends," says Dr. Rochman.

Dr. Andrew J. Rochman is a native New Yorker and a board-certified surgeon. He is a graduate of Colgate University and received his formal medical training from Nordestana University. He is an active member of the American Medical Association, the Medical Society of the State of New York, Nassau County Medical Society and the American College of Phlebology. Dr. Rochman manages several practices in specialized studies such as advanced vein therapy and gallbladder surgery. His current certification is with U.S. Stem Cell, Inc. specializing in cardiovascular treatment through stem cell technology. The Cell Therapy Center of NY is located at 700 Old Country Road, Suite 205 Plainview, NY. For more information, visit: http://www.celltherapycenterny.com or call 516-280-1333.

Media contact: Lennard Gettz 148804@email4pr.com 631-553-8748

To view the original version on PR Newswire, visit:http://www.prnewswire.com/news-releases/stem-cell-therapy-modern-solution-to-joint-pain-relief-300417654.html

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A Cure for Cancer? How CAR-T Therapy is Revolutionizing Oncology – Labiotech.eu (blog)

Posted: March 8, 2017 at 6:41 am

CAR-T has been hailed as a cure for cancer, butwhat really is this miraculous technology and what can we actually expect when it reaches the market?

The field of immuno-oncology is booming with billions of euros in investment. The ability to rewire our own immune system to fight cancer has certainly created huge expectations. After the success of the first checkpoint inhibitorson the market, many are turning their attention to CAR-T therapy,the next big cancer therapy to hit the market.

But is CAR-T therapy really a cure for cancer, as many seem to believe? Can the technology meet such high expectations? Are the recent deaths in clinical trials a sign that CAR-T is not ready for commercialization? What will the future bring?

To answer some of the most burning questions, Italked with some of the leaders in this field to draw an overview of the current state of the field.

CAR therapy is at the same time cell therapy, gene therapy, and immunotherapy.It represents a radical departure from all forms of medicine in existence until now, Michel Sadelain, co-founder of Juno Therapeutics, told The Scientist.

A CAR-T therapy consists in the infusion of engineered T-cells that express a Chimeric Antigen Receptor (CAR) on their cell membrane. This receptor counts with an externaltarget-binding domain designed to recognize a specific tumor antigen and an internalactivation domain responsible for activating the T-cell when the CAR-T binds its target. Second and third generation CAR-Ts have additional costimulatory domains that further enhance the immune response.

The most common procedure for CAR-T therapy consists in the extraction of T-cells from the own patient, which are then genetically modified and expanded in vitro. Finally, they are reinfused into the patient ready to fight the tumor.

Checkpoint inhibitors have already been incredibly successful. The drugs inhibitthe mechanism that tumor cells use to inhibit T-cell activity and overcome the immune system. CAR-T goes one step further and engineers the T-cell itself to enhance the immune response against a specific tumor antigen.

CAR-T clinical trials have shown huge remission rates, of up to 94% in severe forms of cancer, which is particularly impressive considering most of the trials recruit patients that have not responded to all other available treatments for their form of cancer.These preliminary results have fed the expectations of patients and investors alike, but its important to remember that the therapy also has flaws.

Im just trying to be realistic, CAR-T is not the miracle cure for cancer, Andr Choulika, CEO of Cellectis,told us.CAR-T therapy has in fact been linked tosevere side-effects, such as neurotoxicity and cytokine release syndrome.The recent report of several deaths in CAR-T trials made many realize that the technology might not be as perfect as many had originally expected.

These results mostly come from the most studied application of CAR-T, which targets the CD19 antigen found in B-cells.The companies leading the development of CAR-T all target the CD19 antigen to treat B-cell malignancies, which include several forms of lymphoma and leukemia.

The initial furor and excitement of CAR-T hasled to extensive and rapid clinical development in the CD19 target space, saysDavid Gilham, R&D VP at Celyad. Research is busy catching up at the moment, in particular concerning toxicity. The lack of good preclinical models hampers this work, but with clinical samples available, ongoing investigations are now closer to identifying the underlying mechanisms and further refining the approach.

There are several companies competingto be the first tomarket. Competition is tight and the results of a clinical trial can change everything.Juno Therapeutics, whichused to lead the race has just announced the termination of its lead CAR-T program after a total of 5 patients died of cerebral edema caused by the therapy. The company had created huge expectations, and itmarket cap reached over 2.5Bn only 3 years after its inception.

In terms of clinical development, Novartis seems to be leading now. In November, the company recently presented results from a Phase II trial with its CAR-T candidate CTL019 for B-cell acute lymphoblastic leukemia (ALL), which achieved remission in 82% of patients after 3 months. The company is preparing to submit applications to the FDA and EMA this year, despite 50% of patients in the trial were reported to present severe side-effects.

Kite Pharmajustreportedresults from a Phase II clinical trial with its lead candidate, axicabtageneciloleucel, previously KTE-C19. The therapy managed to keep 36% of patients with lymphoma clear of the disease after 6months, which made Kites shares jump by 16%. However, this ongoing trial has already reported three deaths, two of them linked to the CAR-T therapy.Hoping to get ahead of Novartis, Kite Pharmastarted regulatory submissions with the FDAin December.

Despitesevere side-effects and several deaths in clinical trials, some argue that CAR-T therapy is worth the risk when a patient has not responded to any other available treatments. But others are already developing improved versions of CAR-T that are safer for the patients.

Cellectis originally developed UCART19, now licensed to Servier and Pfizer. Its a CAR-T therapy with a switch control system that only activates the engineered cells when rapamycin is present. The therapy is in Phase I and has already saved two babies with aggressive forms of leukemia. Bellicum Pharmaceuticals, in the US, is developing a similar technologycalled GoCAR-T that requiresrimiducid for CAR-T cell activation.

For its part, Celyad is developing a version of CAR-T that makes T-cells express Natural Killer Receptors (NKRs).Its disruptive because NKR-2 binds to 8 different ligands that are expressed on above 80% of solid and hematological malignancies, said Christian Homsy, CEO and co-founder of Celyad.Weve started one of the largest and broadesttrials in the sector, targeting 7 indications.

Targeting solid tumors is a big challenge in the field of immuno-oncology. Low T-cell infiltration and an immunosuppressive environment prevent the immune system from effectively attacking solid tumors. Bluebird bio and Celgene are developing their own CAR-T technology for solid tumors from the US.

Others think that the way to go for CAR-T is combination therapies. We are excited about combining checkpoint inhibitors such as PD-1 inhibitors and anti-CTLA4 drugs with CAR-T cells, said Zelig Eshhar, pioneer of the CAR-T technology at the NIH, for The Scientist.

Most approaches to CAR-T engineer the patients own cells, but this process requires several weeks and is expensive. Some estimates for the therapys price go over 500,000. A possible solution is allogeneic CAR-T therapy, i.e. sourced from a healthy donor and ready to go when the patient needs it.

Cellectis and Celyad are developing their ownallogeneic CAR-T each, and theres friction between them both. In this area, Celyad is still in preclinical phase, while Cellectis UCART123 recently received FDA approval to start clinical trials, making it the first study for allogeneic CAR-T in humans.

The technology promises a faster and cheaper approach to CAR-T, but developing an off-the-shelf therapyis a scientifically challenging avenue according to Celyads CEOChristian Homsy.

I doubt that allogeneic CAR-T can be a real off-the-shelf therapy, he commented. There are still some significant scientific challengeswith regards to immunology, as well as manufacturing, transportation, traceability and banking solutions necessary to reach the scale needed for widespread patient treatment.

As you can appreciate, theres plenty going on in the CAR-T space. The technology is not perfect yet, but it offers hope for patients that had none before. Whoever makes it first to the market will open the way for better alternatives to come in the next years.

Images from CI Photos /Shutterstock; Kite Pharma;Klebanoff et al. (2014) Nature Reviews Clinical Oncology 11, 685686;Bellicum Pharmaceuticals

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