Category Archives: Cell Therapy

Novartis CAR-T cell therapy CTL019 receives FDA Breakthrough Therapy designation for treatment of adult patients with r/r DLBCL.

Novartis announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to CTL019, an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of adult patients with relapsed and refractory (r/r) diffuse large B-cell lymphoma (DLBCL), who have failed two or more prior therapies.

This is the second indication for which CTL019 has received this designation; the first being for the treatment of r/r B-cell acute lymphoblastic leukemia (ALL) in pediatric and young adult patients.

"At Novartis, we are eager to unlock the full potential of CTL019, including the potential to help patients with r/r DLBCL," said Vas Narasimhan, Global Head of Drug Development and Chief Medical Officer, Novartis. "We look forward to working closely with the FDA to help bring this potential new treatment option to patients as soon as possible."

CAR-T cell therapy is different from typical small molecule or biologic therapies currently on the market because it is manufactured for each individual patient. During the treatment process, T cells are drawn from a patient's blood and reprogrammed in the laboratory to create T cells that are genetically coded to hunt the patient's cancer cells and other B-cells expressing a particular antigen.

CTL019 was first developed by the University of Pennsylvania (Penn). In 2012, Novartis and Penn entered into a global collaboration to further research, develop and then commercialize CAR-T cell therapies, including CTL019, for the investigational treatment of cancers. Through the collaboration, Novartis holds the worldwide rights to CARs developed with Penn for all cancer indications.

In March 2017, Novartis announced that the FDA accepted the company's Biologics License Application filing and granted priority review for CTL019 in the treatment of r/r pediatric and young adult patients with B-cell ALL.

The Breakthrough Therapy designation is based on data from the multi-center phase II JULIET study (NCT02445248), which is evaluating the efficacy and safety of CTL019 in adult patients with r/r DLBCL. JULIET is the second global CAR-T trial, following the Novartis ELIANA study (NCT02435849) investigating CTL019 in r/r B-cell ALL. Findings from JULIET are expected to be presented at an upcoming medical congress.

"We are encouraged by the FDA's recognition in the potential of CTL019 for this indication, which follows our promising studies of this therapy for ALL and the FDA filing by Novartis in pediatric and young adult ALL that received priority review," said the Penn team's leader, Carl June, M.D., director of the Center for Cellular Immunotherapies in the Perelman School of Medicine at the University of Pennsylvania. "Work with our collaborators at trial sites across the world is paving a path to bring personalized cell therapies to more patients with these devastating blood cancers."

According to FDA guidelines, treatments that receive Breakthrough Therapy designation are those that treat a serious or life threatening disease or condition and demonstrate a substantial improvement over existing therapies on one or more clinically significant endpoints based on preliminary clinical evidence. The designation also indicates that the agency will expedite the development and review of CTL019 in adults with r/r DLBCL.

This marks the 14thBreakthrough Therapy designation for Novartis since the FDA initiated the program in 2013, underscoring an emphasis to develop innovative treatments in disease areas with significant unmet need.

DLBCL is the most common form of lymphoma and accounts for approximately 30 percent of all non-Hodgkin lymphoma cases1. Ten to 15 percent of DLBCL patients fail to respond to initial therapy or relapse within three months of treatment, and an additional 20 to 25 percent relapse after initial response to therapy2.

Because CTL019 is an investigational therapy, the safety and efficacy profile has not yet been established. Access to investigational therapies is available only through carefully controlled and monitored clinical trials. These trials are designed to better understand the potential benefits and risks of the therapy. Because of the uncertainty of clinical trials, there is no guarantee that CTL019 will ever be commercially available anywhere in the world. ____________________________________________________

References:

1 American Society of Clinical Oncology. Lymphoma - Non-Hodgkin: Subtypes (Dec. 2016 revision).http://www.cancer.net/cancer-types/lymphoma-non-hodgkin/subtypes. Accessed March 2017.

2 Sehn, L. Paramount prognostic factors that guide therapeutic strategies in diffuse large B-cell lymphoma. Hematology, December 2012; 1; 402-409.

(Source: GlobeNewswire)

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Novartis CAR-T Cell Therapy Receives FDA Breakthrough ... - Pharmaceutical Processing

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PolarityTE, Inc. (NASDAQ: COOL) announces the signing of a manufacturing agreement with Cell Therapy and Regenerative Medicine at the University of Utah School of Medicine. CTRM is the established manufacturer of hematopoietic stem cell transplants for renowned institutions of the Salt Lake region such as the Huntsman Cancer Institute and Primary Children's Hospital. In addition, CTRM manufactures a variety of regenerative medicine products, is FACT accredited, and has technical expertise in current Good Tissue Practice (cGTP) and current Good Manufacturing Practice (cGMP).

Denver Lough, MD, PhD, Chairman and CEO, stated, "This agreement with CTRM should provide PolarityTE with rapid clinical translation of the promising products we are developing, and takes us one step closer to achieving our goal of clinical application of our launch product, SkinTE. As we prepare for market entry in 2018, our established relationship with CTRM creates a springboard for the anticipated scale-up to address the large burn and chronic wound markets, with the pursuit of our own independent manufacturing facility. Plans are solidifying for a unique solution to both commercial and emergent relief manufacturing of SkinTE and future Polarity products. Our goal is not only to meet demand, but also to be able to deliver promptly around the globe when urgent response is needed."

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PolarityTE (COOL) Signs Manufacturing Agreement with Cell Therapy and Regenerative Medicine - StreetInsider.com

Novartis CAR-T cell therapy CTL019 receives FDA Breakthrough Therapy designation for treatment of adult patients with r/r DLBCL.

Novartis announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to CTL019, an investigational chimeric antigen receptor T cell (CAR-T) therapy, for the treatment of adult patients with relapsed and refractory (r/r) diffuse large B-cell lymphoma (DLBCL), who have failed two or more prior therapies.

This is the second indication for which CTL019 has received this designation; the first being for the treatment of r/r B-cell acute lymphoblastic leukemia (ALL) in pediatric and young adult patients.

"At Novartis, we are eager to unlock the full potential of CTL019, including the potential to help patients with r/r DLBCL," said Vas Narasimhan, Global Head of Drug Development and Chief Medical Officer, Novartis. "We look forward to working closely with the FDA to help bring this potential new treatment option to patients as soon as possible."

CAR-T cell therapy is different from typical small molecule or biologic therapies currently on the market because it is manufactured for each individual patient. During the treatment process, T cells are drawn from a patient's blood and reprogrammed in the laboratory to create T cells that are genetically coded to hunt the patient's cancer cells and other B-cells expressing a particular antigen.

CTL019 was first developed by the University of Pennsylvania (Penn). In 2012, Novartis and Penn entered into a global collaboration to further research, develop and then commercialize CAR-T cell therapies, including CTL019, for the investigational treatment of cancers. Through the collaboration, Novartis holds the worldwide rights to CARs developed with Penn for all cancer indications.

In March 2017, Novartis announced that the FDA accepted the company's Biologics License Application filing and granted priority review for CTL019 in the treatment of r/r pediatric and young adult patients with B-cell ALL.

The Breakthrough Therapy designation is based on data from the multi-center phase II JULIET study (NCT02445248), which is evaluating the efficacy and safety of CTL019 in adult patients with r/r DLBCL. JULIET is the second global CAR-T trial, following the Novartis ELIANA study (NCT02435849) investigating CTL019 in r/r B-cell ALL. Findings from JULIET are expected to be presented at an upcoming medical congress.

"We are encouraged by the FDA's recognition in the potential of CTL019 for this indication, which follows our promising studies of this therapy for ALL and the FDA filing by Novartis in pediatric and young adult ALL that received priority review," said the Penn team's leader, Carl June, M.D., director of the Center for Cellular Immunotherapies in the Perelman School of Medicine at the University of Pennsylvania. "Work with our collaborators at trial sites across the world is paving a path to bring personalized cell therapies to more patients with these devastating blood cancers."

According to FDA guidelines, treatments that receive Breakthrough Therapy designation are those that treat a serious or life threatening disease or condition and demonstrate a substantial improvement over existing therapies on one or more clinically significant endpoints based on preliminary clinical evidence. The designation also indicates that the agency will expedite the development and review of CTL019 in adults with r/r DLBCL.

This marks the 14thBreakthrough Therapy designation for Novartis since the FDA initiated the program in 2013, underscoring an emphasis to develop innovative treatments in disease areas with significant unmet need.

DLBCL is the most common form of lymphoma and accounts for approximately 30 percent of all non-Hodgkin lymphoma cases1. Ten to 15 percent of DLBCL patients fail to respond to initial therapy or relapse within three months of treatment, and an additional 20 to 25 percent relapse after initial response to therapy2.

Because CTL019 is an investigational therapy, the safety and efficacy profile has not yet been established. Access to investigational therapies is available only through carefully controlled and monitored clinical trials. These trials are designed to better understand the potential benefits and risks of the therapy. Because of the uncertainty of clinical trials, there is no guarantee that CTL019 will ever be commercially available anywhere in the world. ____________________________________________________

References:

1 American Society of Clinical Oncology. Lymphoma - Non-Hodgkin: Subtypes (Dec. 2016 revision).http://www.cancer.net/cancer-types/lymphoma-non-hodgkin/subtypes. Accessed March 2017.

2 Sehn, L. Paramount prognostic factors that guide therapeutic strategies in diffuse large B-cell lymphoma. Hematology, December 2012; 1; 402-409.

(Source: GlobeNewswire)

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CAR-T Cell Therapy Receives FDA Breakthrough Designation - Pharmaceutical Processing

The Husaini Haematology and Oncology Trust will soon provide facilities of bone marrow transplant and stem cell therapies at its newly established Blood Transfusion Centre and Thalassaemia Centre that was inaugurated by the city director health on Tuesday.

Speaking at the inaugural ceremony, Karachi director health Dr Muhammad Toufique urged the trust officials to share the data of patients undergoing blood transfusions, stem cell therapies and bone marrow transplant.

He said the data would help the Sindh government formulate a plan to establish more such facilities in the future. In addition to the blood screening and storage facilities, the centre is providing blood transfusion service to children suffering from genetic blood disorders as well as diagnostic services related to blood disorders.

This is a state-of-the-art blood transfusion and thalassaemia centre where bone marrow transplant and stem cell therapies would be started very soon, said a renowned haematologist associated with the Husaini Blood Bank, Dr Sarfraz Jaffery, at the inaugural ceremony of the blood transfusion and thalassaemia centre located at Qalandaria Chowk, North Nazimabad.

The head office of the Husaini Haematology and Oncology Trust is equipped with a diagnostic lab, blood bank having storage capacity of around 3,000 blood bags and blood transfusion centre for thalassaemic patients while its management is also planning to introduce bone marrow transplant and stem cell therapy services at the same facility in the near future.

Felicitating the trust officials, the city director health vowed to support them in their services. He said the government was also striving hard for provision of safe blood to thalassaemic children and other patients.

Dr Toufique hoped that institutions like Hussaini would come forward to support the government in establishing such centres in the province. Talking to journalists, the director health said steps were being taken to control the outbreak of Chikungunya in the city.

He said the health department was in contact with the municipal authorities to start fumigation in various areas of Karachi to eliminate the mosquitoes and prevent people from mosquito-borne diseases, including dengue and Malaria.

The Sindh government was planning to merge the Malaria and Dengue Prevention and Control Cells under one project director, who would be utilizing all the resources to eliminate the mosquitoes that were responsible for the deadly infectious diseases in the province, he added.

I would also urge people to take precautionary measures, prevent themselves and their children from mosquitoes by using repellents, improving sanitation conditions in their residential areas and adopt other preventive measures to protect themselves against the mosquitoes, he advised.

Earlier, speaking at a workshop on thalassaemia management held at the same place, noted haematologists of the country stressed the need for promoting the culture of prevention from diseases in the country.

They called for the implementation of laws regarding thalassaemia screening, saying that both the government and private sector could not treat the increasing number of thalassaemic patients.

Senior haematologist from Lahore, Prof Dr Jovaria Mannan, urged the doctors and researchers to use latest research methods in the field of haematology.

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Husaini trust plans to perform bone marrow transplants, stem cell therapy - The News International

CAR-T cell therapy is different from typical small molecule or biologic therapies currently on the market because it is manufactured for each individual patient. During the treatment process, T cells are drawn from a patient's blood and reprogrammed in the laboratory to create T cells that are genetically coded to hunt the patient's cancer cells and other B-cells expressing a particular antigen.

CTL019 was first developed by the University of Pennsylvania (Penn). In 2012, Novartis and Penn entered into a global collaboration to further research, develop and then commercialize CAR-T cell therapies, including CTL019, for the investigational treatment of cancers. Through the collaboration, Novartis holds the worldwide rights to CARs developed with Penn for all cancer indications. In March 2017, Novartis announced that the FDA accepted the company's Biologics License Application filing and granted priority review for CTL019 in the treatment of r/r pediatric and young adult patients with B-cell ALL.

The Breakthrough Therapy designation is based on data from the multi-center Phase II JULIET study (NCT02445248), which is evaluating the efficacy and safety of CTL019 in adult patients with r/r DLBCL. JULIET is the second global CAR-T trial, following the Novartis ELIANA study (NCT02435849) investigating CTL019 in r/r B-cell ALL. Findings from JULIET are expected to be presented at an upcoming medical congress.

"We are encouraged by the FDA's recognition in the potential of CTL019 for this indication, which follows our promising studies of this therapy for ALL and the FDA filing by Novartis in pediatric and young adult ALL that received priority review," said the Penn team's leader, Carl June, MD, director of the Center for Cellular Immunotherapies in the Perelman School of Medicine at the University of Pennsylvania. "Work with our collaborators at trial sites across the world is paving a path to bring personalized cell therapies to more patients with these devastating blood cancers."

According to FDA guidelines, treatments that receive Breakthrough Therapy designation are those that treat a serious or life threatening disease or condition and demonstrate a substantial improvement over existing therapies on one or more clinically significant endpoints based on preliminary clinical evidence. The designation also indicates that the agency will expedite the development and review of CTL019 in adults with r/r DLBCL. This marks the 14th Breakthrough Therapy designation for Novartis since the FDA initiated the program in 2013, underscoring an emphasis to develop innovative treatments in disease areas with significant unmet need.

DLBCL is the most common form of lymphoma and accounts for approximately 30 percent of all non-Hodgkin lymphoma cases1. Ten to 15 percent of DLBCL patients fail to respond to initial therapy or relapse within three months of treatment, and an additional 20 to 25 percent relapse after initial response to therapy2.

Because CTL019 is an investigational therapy, the safety and efficacy profile has not yet been established. Access to investigational therapies is available only through carefully controlled and monitored clinical trials. These trials are designed to better understand the potential benefits and risks of the therapy. Because of the uncertainty of clinical trials, there is no guarantee that CTL019 will ever be commercially available anywhere in the world.

DisclaimerThe foregoing release contains forward-looking statements that can be identified by words such as "Breakthrough Therapy designation," "investigational," "potential," "expected," "eager," "look forward," "priority review," "upcoming," "encouraged," "promising," "yet," "will," or similar terms, or by express or implied discussions regarding potential marketing approvals for CTL019, or regarding potential future revenues from CTL019. You should not place undue reliance on these statements. Such forward-looking statements are based on the current beliefs and expectations of management regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that CTL019 will be submitted or approved for sale in any market, or at any particular time. Nor can there be any guarantee that CTL019 will receive regulatory approval or be commercially successful in the future. In particular, management's expectations regarding CTL019 could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; the company's ability to obtain or maintain proprietary intellectual property protection; general economic and industry conditions; global trends toward health care cost containment, including ongoing pricing and reimbursement pressures; safety, quality or manufacturing issues, and other risks and factors referred to in Novartis AG's current Form20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.

About NovartisLocated in East Hanover, NJ Novartis Pharmaceuticals Corporation is an affiliate of Novartis which provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic and biosimilar pharmaceuticals and eye care. Novartis has leading positions globally in each of these areas. In 2016, the Group achieved net sales of USD 48.5 billion, while R&D throughout the Group amounted to approximately USD 9.0 billion. Novartis Group companies employ approximately 118,000 full-time-equivalent associates. Novartis products are sold in approximately 155 countries around the world. For more information, please visit http://www.novartis.com.

Novartis is on Twitter. Sign up to follow @Novartis at http://twitter.com/novartis and @NovartisCancer at http://twitter.com/novartiscancer For Novartis multimedia content, please visit http://www.novartis.com/news/media-library For questions about the site or required registration, please contact media.relations@novartis.com

References

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SOURCE Novartis

http://www.novartis.com

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Novartis CAR-T cell therapy CTL019 receives FDA Breakthrough ... - PR Newswire (press release)

A research team at University of Wisconsin School of Medicine and Public Health has treated its first patient in an innovative clinical trial using stem cells for the treatment of heart failure that develops after a heart attack.

The trial is taking place at University Hospital, one of three sites nationwide currently enrolling participants. The investigational CardiAMP therapy is designed to deliver a high dose of a patients own bone-marrow cells directly to the point of cardiac injury to potentially stimulate the bodys natural healing response.

The patient experience with the trial begins with a cell-potency screening test. Patients who qualify for therapy are scheduled for a bone-marrow aspiration. The bone marrow is then processed on-site and subsequently delivered directly to the damaged regions in a patients heart in a minimally invasive procedure.

Patients living with heart failure experience a variety of negative symptoms that can greatly impact their day-to-day life, said UW Health cardiologist Dr. Amish Raval, associate professor of medicine and one of the principal investigators for the trial. By being at the forefront of research for this debilitating condition, we look forward to studying the potential of this cell therapy to impact a patients exercise capacity and quality of life.

The primary outcome to be measured is the change in distance during a six-minute walk 12 months after the initial baseline measurement.

Heart failure commonly occurs after a heart attack, when the heart muscle is weakened and cannot pump enough blood to meet the body's needs for blood and oxygen. About 790,000 people in the U.S. have heart attacks each year. The number of adults living with heart failure increased from about 5.7 million (2009-2012) to about 6.5 million (2011-2014), and the number of adults diagnosed with heart failure is expected to dramatically rise by 46 percent by the year 2030, according to the American Heart Association (AHA).

The CardiAMP Heart Failure Trial is a phase III study of up to 260 patients at up to 40 centers nationwide. Phase III trials are conducted to measure effectiveness of the intervention, monitor side effects and gather information for future use of the procedure. Study subjects must be diagnosed with New York Heart Association (NYHA) Class II or III heart failure as a result of a previous heart attack.

Information about eligibility or enrollment in the trial is available at http://www.clinicaltrials.gov, or through a cardiologist.

The trial is funded by Biocardia, Inc., which developed the potential therapy.

Date Published: 04/17/2017

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UW Treats First Participant in Trial of Stem-Cell Therapy for Heart Failure - University of Wisconsin-Madison

DUBLIN--(BUSINESS WIRE)--

Research and Markets has announced the addition of Jain PharmaBiotech's new report "Cell Therapy - Technologies, Markets and Companies" to their offering.

This report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

The cell-based markets was analyzed for 2016, and projected to 2026. The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.

The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 305 of these are profiled in part II of the report along with tabulation of 291 alliances. Of these companies, 170 are involved in stem cells. Profiles of 72 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 64 Tables and 22 Figures. The bibliography contains 1,200 selected references, which are cited in the text.

Key Topics Covered:

Part I: Technologies, Ethics & Regulations

0. Executive Summary

1. Introduction to Cell Therapy

2. Cell Therapy Technologies

3. Stem Cells

4. Clinical Applications of Cell Therapy

5. Cell Therapy for Cardiovascular Disorders

6. Cell Therapy for Cancer

7. Cell Therapy for Neurological Disorders

8. Ethical, Legal and Political Aspects of Cell therapy

9. Safety and Regulatory Aspects of Cell Therapy

10. Markets and Future Prospects for Cell Therapy

11. Companies Involved in Cell Therapy

12. Academic Institutions

13. References

For more information about this report visit http://www.researchandmarkets.com/research/dw396t/cell_therapy

View source version on businesswire.com: http://www.businesswire.com/news/home/20170417005383/en/

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Cell Therapy Report (2017-2026) - Markets & Future Prospects for ... - Yahoo Finance

DUBLIN--(BUSINESS WIRE)--Research and Markets has announced the addition of Jain PharmaBiotech's new report "Cell Therapy - Technologies, Markets and Companies" to their offering.

This report describes and evaluates cell therapy technologies and methods, which have already started to play an important role in the practice of medicine. Hematopoietic stem cell transplantation is replacing the old fashioned bone marrow transplants. Role of cells in drug discovery is also described. Cell therapy is bound to become a part of medical practice.

Stem cells are discussed in detail in one chapter. Some light is thrown on the current controversy of embryonic sources of stem cells and comparison with adult sources. Other sources of stem cells such as the placenta, cord blood and fat removed by liposuction are also discussed. Stem cells can also be genetically modified prior to transplantation.

The cell-based markets was analyzed for 2016, and projected to 2026. The markets are analyzed according to therapeutic categories, technologies and geographical areas. The largest expansion will be in diseases of the central nervous system, cancer and cardiovascular disorders. Skin and soft tissue repair as well as diabetes mellitus will be other major markets.

The number of companies involved in cell therapy has increased remarkably during the past few years. More than 500 companies have been identified to be involved in cell therapy and 305 of these are profiled in part II of the report along with tabulation of 291 alliances. Of these companies, 170 are involved in stem cells. Profiles of 72 academic institutions in the US involved in cell therapy are also included in part II along with their commercial collaborations. The text is supplemented with 64 Tables and 22 Figures. The bibliography contains 1,200 selected references, which are cited in the text.

Key Topics Covered:

Part I: Technologies, Ethics & Regulations

0. Executive Summary

1. Introduction to Cell Therapy

2. Cell Therapy Technologies

3. Stem Cells

4. Clinical Applications of Cell Therapy

5. Cell Therapy for Cardiovascular Disorders

6. Cell Therapy for Cancer

7. Cell Therapy for Neurological Disorders

8. Ethical, Legal and Political Aspects of Cell therapy

9. Safety and Regulatory Aspects of Cell Therapy

10. Markets and Future Prospects for Cell Therapy

11. Companies Involved in Cell Therapy

12. Academic Institutions

13. References

For more information about this report visit http://www.researchandmarkets.com/research/dw396t/cell_therapy

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Cell Therapy Report (2017-2026) - Markets & Future Prospects for Cell Therapy - Research and Markets - Business Wire (press release)

NAVI MUMBAI: A forum group in Navi Mumbai conducted a question and answer session at Vashi to clear that there is no proof of stem cell therapy cure for autism. Around 100 parents of autistic children were part of the session held at Sunshine Autistic School in Vashi organised by the Forum for Autism group on Saturday. Guest speaker Dr Tatyana Dias, a PhD in neurobiology from the University of Edenburgh, UK, said, "All traditional therapies like occupational therapy, speech Therapy and special education are evidence-based which means they have been proved to be effective through immense research and practice. Whereas stem cell therapy is in research stage, its effectiveness is strongly doubted, even its practice is banned in many countries and if practised, it is done in labs and under strict regulations. In India at present there is no particular body or law to regulate stem cell research." Babita Raja, secretary, Forum for Autism, said, "Parents run from pillar to post for their children's treatment. We are hoping that this awareness programme would help them in deciding what they want to try."

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'No proof that stem cell therapy can cure autism' - Times of India

In the world of cancer medicine, immunotherapy has taken over with a vice-like grip, offering far-reaching potential for nearly every tumor type known to man. Most prominent in the marketplace have been the immune checkpoint inhibitors, with almost every one of the big five PD-1/PD-L1 antibodies (nivolumab, pembrolizumab, atezolizumab, avelumab, durvalumab) gaining some high-profile drug approval in the last 5 years, to say nothing of the landmark approval of Yervoy in 2011 to kick all of this fervor off.

But before immune checkpoint inhibitors were approved, we had cell-based immunotherapy, notably with the introduction (and subsequent challenges) of Provenge. Cell-based immunotherapy actually goes all the way back to the late 1800s, with bacterial infection being used as a vector to stimulate an immune response in cancer patients.

Now we've gotten more sophisticated. Three companies, Kite Pharma (NASDAQ:KITE), Juno Therapeutics (NASDAQ:JUNO), and Novartis (NYSE:NVS) have been frontrunners in the race to bring so-called CAR-T cell therapy to market for hematologic malignancies.

A primer

Source for image: cancer.gov

The intricate details of CAR-T cell manufacture are too complex to manage in a short publication like this one. But it can be simplified in broad terms down to a few steps:

Some of the more curious among us might be asking...why go to all this trouble? We can train the body's immune system to recognize specific targets. Heck, we've been doing it for decades now with Herceptin and Rituxan. What's wrong with the body's natural defense?

The answer is that CAR-T cells present a few extra advantages to ramp up the immune response: the CAR itself - The name of the technique gives this away. "Chimeric" isn't just a cool word (which it certainly is); it signifies that we've done something special to the receptor in question. In the current line of techniques, we've fused the antigen recognition portion of an antibody to the part of the T cell receptor that tells the cell to grow and divide.

This differs from the normal method the body has to detect a foreign antigen and develop T cells against it:

Source: Srivastava, et al.

You may not recognize the names of the molecules in this figure, but you should be able to see that on the left side, there is careful coordination of a large number of molecules that is required to activate a T cell.

CARs short circuit the whole process, allowing for direct activation of the T cells by tumor cells. This MHC-independent T cell activation is the linchpin of the whole process, bypassing a number of tumor cell defenses and allowing us to develop a special subset of T cells that specifically look for and eliminate any cells in the body that express the antigen we're looking for. In the current case, this is CD19, which is a marker of B cells, hence why all of these latest studies are looking at diseases like B-cell leukemia and diffuse large B-cell lymphoma.

Bioengineered T cells have an end in sight, with several techs being reviewed at the FDA

Since the seminal publication by Maude, et al in 2014 showing incredible response rates in a small cohort of children with relapsed/refractory acute lymphoblastic leukemia (ALL), the world has been watching and waiting for the emergence of CAR-T cell therapy and its revolutionary potential.

No rides are ever smooth in biotech, it seems. For a while, the three big players- KITE, JUNO, NVS- were chasing three different patient populations.

NVS had CTL019, which was being studied in pediatric patients with ALL.

JUNO had JCAR015 for adult patients with ALL.

KITE decided to chase a different beast first, focusing on patients with diffuse large B-cell lymphoma (DLBCL), an aggressive form of non-Hodgkin lymphoma.

In my mind, this presented three distinct patient classes that could allow all three technologies to be marketed simultaneously. In the United States, ALL in kids and adults is not generally managed by the same hematologists; pediatric doctors handle children, specifically.

Unfortunately, fate was not kind to JUNO, who had to suspend their ROCKET trial in adults due to life-threatening toxicity risk. I wrote about this episode last year, and even though the clinical hold was lifted, JUNO eventually terminated development of its JCAR015 platform in March 2017, choosing instead to focus on JCAR017 for DLBCL.

KITE and NVS, in contrast, have achieved significant progress in moving CAR-T cells to the clinic. Both axicabtagene ciloleucel and CTL019 are now being reviewed by federal regulators, and it is likely we'll see responses by the end of 2017.

Given results like those we've seen with the ZUMA and ELIANA (the former I covered in my digest series, 3 Things You Should Learn Today in Biotech), it seems like CAR-T cell therapy presents an enormously promising treatment strategy for these intractable B cell malignancies. Aside from the risk of cytokine storm (an active area of research), these CAR-T platforms are not associated with an outsized risk of severe toxicity, either. I am going to be very surprised if these two techs do not get the nod from the FDA.

Approval of just one of these methods has the promise to usher in a new era for immunotherapy

It is difficult to overstate how reticent the FDA can be to accept a new therapeutic strategy into the fold. They are definitely conservative, and I say this is a very GOOD thing. The history of cancer medicine is peppered with charlatans who have generated excitement and clamor for new, promising cancer therapies.

The FDA needs to be the voice of reason and consider everything, from manufacturing to efficacy to every bit of safety they can uncover. As such, many are frustrated with the speed at which they move.

But the data on CAR-T cells are too compelling to ignore. I think this is going to prompt the FDA to get more familiar with cell-based immunotherapy in general and develop a different tolerance for risk of these approaches.

This represents a major, major inroad for other forms of cell therapy, including JUNO's JCAR015 and the other KITE/NVS platforms for CAR-T cell therapy. We could potentially see approvals for CAR-T cells emerge quickly in other hematologic malignancy settings.

But it also could signal an increasing tolerance for other approaches. And this is the biggest implication for those looking for diamonds in the rough with the stock market. Lots of small up-and-comers are exploring cell-based immunotherapy in various forms. To name just a few:

It's time to get ready for a wild ride in immunotherapy

To be clear, pointing out these companies does not mean I'm suggesting you buy, buy, buy. There are still risks associated with all these nascent technologies, and many will not pan out. Hematologic malignancies have had a long history of achieving groundbreaking therapeutics results that do not translate to solid tumors, so CAR-T cell therapy for, say, pancreatic cancer sounds tantalizing, as this is a huge unmet need. But pancreatic cancer chews through "promising" technologies like nothing else. The graveyard is long and grim there.

Still, my thesis here is that the likely approval of CAR-T cells in heme malignancies is going to give the FDA more experience with "live" immunotherapies, which will help them produce better guidance for other players in the field. This will almost certainly generate substantial excitement, and intrepid investors had better get on the ball sooner rather than later, or else they'll find themselves chasing the gold. Use the experience of JUNO, KITE, and NVS to your favor, and learn what you can about these promising therapies. It will come to play a major role in your due diligence.

Disclosure: I am/we are long ADXS.

I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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CAR-T Cell Therapy Means A Lot More Than One Or Two New Drug Approvals - Seeking Alpha