Category Archives: Cell Therapy

DUBLIN, Aug. 3, 2022 /PRNewswire/ --The "ROR1-Targeted Therapy: Target Expression Profile, Safety & Efficacy of Drug Modalities, Pipeline Review, and Competitive Landscape Analysis" report has been added to ResearchAndMarkets.com's offering.

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This report evaluates Receptor tyrosine kinase-like Orphan Receptor 1 (ROR1) from an industry perspective for its suitability as a tumor-specific target for cancer therapy based on its expression profile and preclinical and clinical safety and efficacy data of the various drug modalities employed for discovery and development of ROR1-targeted therapy candidates.

The report has identified the players in the field and presents a competitive landscape analysis of stakeholders and a pipeline review based on the specific profiles of drug candidates and companies active in the field. The report includes information about business transaction in the field, such as acquisitions, partnerships & collaborations and licensing deals. Furthermore, the financial background and situation of the key players is described.

Receptor tyrosine kinase-like orphan receptor 1 (ROR1) is a type I transmembrane protein that is physiologically expressed in early embryogenesis and plays a critical role in organogenesis. Expression of ROR1 attenuates rapidly after embryonic development, becoming virtually undetectable on post-partem tissues, with the exception of a few B cell precursors. In contrast, ROR1 is expressed on a variety of cancers, particularly those that are less differentiated, and is associated with early relapse after therapy or metastasis.

Because of its tumor-specific expression and potential functional significance, ROR1 has become of interest as a target for various drug modalities, especially with enhanced effector function. The most advanced molecules is currently being evaluated in potentially registrational phase II/III studies, but the majority of programs are in late preclinical and early clinical development, thus still offering opportunities for improvements.

This report is based information retrieved from proprietary database, clinical trial registries, abstracts, presentations and posters from scientific meetings as well as full publications, from company websites, press releases, SEC filings, investor and R&D presentations.What will you find in the report:

The scientific rationale for ROR1-targeted therapies based on target characteristics and its differential expression profile;

Preclinical safety of ROR1-targeted therapy candidates;

Clinical experience and proof-of-concept with ROR1-targeted drug modalities;

Clinical indications suitable for development of ROR1-targeted therapies and their patient populations;

The competitive landscape of ROR1-targeted drug modalities in development;

Specific profiles of ROR1-targeted drug modalities; and

Profiles of companies active in the development of anti-ROR1 therapy candidates.

Key Topics Covered:

Executive Summary

1 Target Background: Structure & Function

2 Target Antigen Expression Profile2.1 Hematologic Malignancies2.2 Various Solid Tumors2.3 Lung Cancer2.4 Pancreatic Cancer2.5 Colorectal Cancer2.6 Breast Cancer2.7 Ovarian Cancer

3 Preclinical Safety of ROR1-Targeted Therapy Candidates

4 Clinical Experience with ROR1-Targeted Drug Modalities

5 Clinical Indications & Patient Populations5.1 Chronic Lymphocytic Leukemia5.2 Mantle Cell Lymphoma5.3 Diffuse Large B-Cell Lymphoma5.4 Triple Negative Breast Cancer

6 Competitive Landscape & Drug Modalities6.1 ROR1-Targeted Antibody Drug Conjugates6.2 T-Cell EngagingAnti-ROR1 Antibodies6.3 Adoptive Cell Therapy with ROR1-Targeted CAR T-Cells and NK Cells6.4 ROR1 Small Molecule Inhibitor

7 Drug & Cell Therapy Candidate Profiles7.1 Naked Antibody7.2 Antibody-Drug Conjugates7.3 T-Cell Engaging Bispecific Antibodies7.4 CAR T-Cells & NK Cells7.5 Small Molecule Inhibitor

8 Company Profiles

9 References

For more information about this report visit https://www.researchandmarkets.com/r/1xld2f

Media Contact:

Research and MarketsLaura Wood, Senior Managerpress@researchandmarkets.com

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World ROR1-Targeted Therapy Market Research Report 2022 with Profiles of Companies Active in the development of Anti-ROR1 Therapy Candidates - Yahoo...

Some patients with blood cancers for whom all other therapeutic options have been exhausted have one final chance of getting rid of their disease treatment with chimeric antigen-receptor (CAR) T cells.

Described as a "living drug," the treatment involves genetically engineering the patient's own blood cells and reinfusing them back into their system. These CAR T cells then hunt down and destroy cancer cells, and in some cases, they manage to totally eradicate the disease.

About half of patients with leukemia or lymphoma and about a third with myeloma who receive this treatment have a complete remission and achieve a functional "cure."

But not all patients who could benefit from this therapy are able to get it.

Some are spending months on waiting lists, often deteriorating while they wait. These patients have exhausted all other therapeutic options, and many are facing hospice and death.

The scope of this problem was illustrated by a recent survey of the centers that are certified to deliver this complex therapy.

The survey was led by Yi Lin, MD, PhD, associate professor of medicine at the Mayo Clinic, Rochester, Minnesota, and medical director for the cellular therapy program. It was published as an abstract at the recent annual meeting of the American Society of Clinical Oncology, although it was not presented at the meeting.

"We wanted to find out just how widespread this problem is," she said, adding, "There had been nothing in the literature thus far about it."

The team contacted 20 centers across the US and received responses from 17.

The results showed that the median time on the waiting list was 6 months and that only 25% of patients eventually received CAR T-cell therapy.

An additional 25% were able to enter a CAR-T clinical trial.

The remaining 50% of patients either were enrolled in a different type of trial, entered hospice, or died.

For patient selection, all centers reported using a committee of experienced physicians to ensure consistency. They employed different ethical principles for selection. Some centers sought to maximize the total benefit, such as selecting the patients most likely to achieve leukapheresis or a clinical response, while others based their decisions on the time patients spent on waiting list or gave priority to the patients who were the "worst off" with the most limited therapeutic options.

The shortages in CAR T-cell therapies primarily involve the products used for patients with multiple myeloma.

The problem has not, as yet, noticeably spilled over to lymphoma and leukemia treatments, which use a slightly different type of CAR T-cell therapy (it targets CD19, whereas the cell therapies used for myeloma target BCMA).

"We have backlog of myeloma patients who don't have access," said Nina Shah, MD, a hematologist and professor of medicine at the University of California, San Francisco. "We have only four slots for the two myeloma products but about 50 to 60 eligible patients."

Long waiting times for CAR T cells for myeloma have been an issue ever since the first of these products appeared on the market idecabtagene vicleucel (ide-cel; Abecma), developed by Bluebird Bio and Bristol-Myers Squibb (BMS). "As soon as it became available in March 2021, we had people waiting and limits on our access to it," Shah said.

A second CAR T-cell therapy for myeloma, ciltacabtagene autoleucel (cilta-cel, Carvykti), developed by Janssen and Legend Biotech, received approval in February 2022.

While that helped provide centers with a few more slots, it wasn't sufficient to cut the waiting times, and the demand for these myeloma therapies continues to outstrip the capacity to produce CAR-T products in a timely manner.

"For myeloma, the demand is very high, as most patients are not cured from any other existing myeloma therapies, and most patients will make it to fifth-line therapy where the two CAR T-cell products are approved right now," said Krina K. Patel, MD, medical director of the Department of Lymphoma/Myeloma in the Division of Cancer Medicine at the University of Texas MD Anderson Cancer Center, Houston.

"We likely have 10 eligible CAR-T myeloma patients each month at our center," she said, "but were getting two slots per month for the past 8 months, and now are getting four slots a month."

"Our clinic has also experienced the impact of the low number of manufacturing slots offered to each cancer center for some CAR T-cell products," said David Maloney, MD, PhD, medical director, Cellular Immunotherapy and Bezos Family Immunotherapy Clinic, Seattle Cancer Care Alliance, Washington.

He noted that, as with other cancer centers, for multiple myeloma, they are provided a specific number of manufacturing slots for each treatment. "Our providers discuss which patients are most appropriate for available slots for that month," said Maloney.

"Additionally, juggling patient schedules may be required to address the extended manufacturing time for some products. In some cases, clinical trials may be available in a more timely fashion for appropriate patients, and in some cases, switching to an alternative product is possible," he commented.

The cause of the current bottleneck for myeloma patients is complex. It stems from a shortage of raw materials and supply chain restraints, among other things.

While the biggest impact of shortages has been on patients with multiple myeloma, Patel pointed out that these constraints are also affecting patients with lymphoma at her institution, but to a lesser degree currently.

"This is multifactorial as to why, but most of the issues arise from manufacturing," Patel told Medscape Medical News. "Initially, the FDA limited how many slots each new product could have per month, then there was a viral vector shortage, and then the quality-control process the FDA requires takes longer than the manufacturing of the cells actually do," she explained.

On top of that, "we have about a 5% manufacturing fail rate so far," she added. Such failures occur when the cells taken from a patient cannot be converted into CAR T cells for therapy.

Matthew J. Frigault, MD, from the Center for Cellular Therapies, Mass General Cancer Center, Boston, explained that the growing excitement about the potential for cellular therapy and recent approvals for these products for use in earlier lines of treatment have increased demand for them.

There are also problems regarding supply. The manufacturing and delivery of CAR-T is complicated and takes time to scale up, Frigault pointed out. "Therefore, we are seeing limited access, more so for the BCMA-directed therapies [which are used for myeloma]."

The shortages and delays likely involve two main factors. "For the newer indications, there is a significant backlog of patients who have been waiting for these therapies and have not been able to access them in the clinical trial setting, and manufacturing is extremely complicated and not easily scaled up," he said.

"That being said, manufacturers are trying to increase the number of available manufacturing slots and decrease the time needed to manufacture cells," Frigault commented.

Delays in access to myeloma CAR T-cell therapy are also affecting patient care at the Fox Chase Cancer Center. "We have had about one slot every 2 months for Abecma," noted Henry Fung, MD, chair of the Department of Bone Marrow Transplant and Cellular Therapies. "For Carvykti, there are only 32 certified centers in US, and access is very limited."

Fung explained that they have had to offer alternative treatments to many of their patients. "There are rumors that there's shortage in obtaining raw materials, such as the virus used for transduction, although we have not encountered any problems in other CAR-T products used for lymphomas," he said.

Medscape Medical News reached out to the manufacturers of the CAR-T products. All have reported that they are doing what they feasibly can to ramp up production.

"The complexity of delivering CAR T-cell therapies is unlike any other traditional biologic or small-molecule medicine, using a patient's own cells to start a highly sophisticated and personalized manufacturing process," commented a spokesperson for BMS, which has two CAR T-cell products currently on the market.

"In this nascent field of cell therapy, we continue to evolve every day, addressing supply and manufacturing challenges head on by applying key learnings across our three state-of-the-art cell therapy facilities and two new facilities in progress.

"We have been encouraged by a steady increase in our manufacturing capacity, and we continue efforts to ramp up further to meet the demand for our cell therapies," the BMS spokesperson commented. "We have already seen improvements in the stabilization of vector supply and expect additional improvements in capacity in the second half of 2022."

Novartis said much the same thing. They have a "comprehensive, integrated global CAR-T manufacturing footprint that strengthens the flexibility, resilience, and sustainability of the Novartis manufacturing and supply chain. Together with an improved manufacturing process, we are confident in our ability to meet patient demand with timely delivery," said a Novartis spokesperson.

The spokesperson also pointed out that the company has continuously incorporated process improvements that have significantly increased manufacturing capacity and success rates for patients in need of CAR T cells.

"Data presented at American Society of Hematology annual meeting in 2021 showed the Novartis Morris Plains facility our flagship CAR-T manufacturing site had commercial manufacturing and shipping success rates of 96% and 99%, respectively, between January and August 2021," according to the spokesperson.

Legend and Janssen, the companies behind Carvykti, one of the two approved cell products for myeloma, which launched earlier this year, said that they have continued to activate certified treatment centers in a phased approach that will enable them to expand availability throughout 2022 and beyond.

"This phased approach was designed to ensure the highest level of predictability and reliability for the patient and the certified treatment centers," the spokesperson said. "We understand the urgency for patients in need of Carvyki and are committed to doing everything we can to accelerate our ability to deliver this important cell therapy in a reliable and timely manner."

With regard to the industry-wide supply shortage of lentivirus, Legend and Jassen say they have put in place multiple processes to address the shortage, "including enhancing our own internal manufacturing capabilities of this essential drug substance, to ensure sufficient and sustained supply."

The supply shortage that myeloma patients are experiencing is all the more poignant and distressing, given the immense potential of CAR T-cell therapy. While not everyone benefits, some patients for whom every other therapy failed and who were facing hospice have had dramatic results.

"Incredibly exciting with unbelievable potential" was how one expert described these new therapies when the first product was about to enter the marketplace. Since then, six CAR T-cell therapies have received regulatory approval for an ever-increasing range of hematologic malignancies.

But these CAR T-cell therapies have their own set of adverse events, which can be serious and even life-threatening. In addition, not all patients become cancer free, although long-term data are impressive.

A study that included one of the longest follow-ups to date was reported at the 2020 annual meeting of the American Society of Clinical Oncology. The researchers reported that remissions lasted over 9 years for patients with relapsed/refractory B-cell lymphoma or chronic lymphocytic leukemia who underwent treatment with Kite's axicaptagene cilleucel (Yescarta). This review included 43 patients and showed an overall remission rate of 76%. Complete remission was achieved for 54% of patients, and partial remission was achieved for 22%.

The results with CAR T-cell therapy in multiple myeloma are not quite as impressive, but even so, the clinical data that supported the approval of Abecma showed that a third of patients, who had previously received a median of six prior therapies, achieved a complete response.

At the time of the Abecma approval, the lead investigator of the study, Nikhil Munshi, MD, of Dana-Farber Cancer Institute, Boston, Massachusetts, commented: "The results of this trial represent a true turning point in the treatment of this disease. In my 30 years of treating myeloma, I have not seen any other therapy as effective in this group of patients."

Roxanne Nelson is a registered nurse and an award-winning medical writer who has written for many major news outlets and is a regular contributor to Medscape.

For more news, follow Medscape on Facebook, Twitter, Instagram, and YouTube.

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Patients Waiting Months for 'Last Chance' CAR T-Cell Therapy - Medscape

This webinar will be hosted live and available on-demand

Tuesday, September 13 20222:30 PM Eastern Time (11:30 AM PT)

The tumor microenvironment can severely diminish adoptive T cell therapy efficacy by expressing ligands that activate T cell inhibitory receptors and by downregulating costimulatory ligand expression. To address these issues, researchers developed fusion proteins (FPs) that combine the ectodomain of one receptor with a different intracellular costimulatory signaling domain. Using in vitro assays, including the Incucyte serial killing over-time (SKO) assay, they evaluated T cell engineering strategies in persistence and survival studies.

In this LabTools webinar brought to you by Sartorius, Shannon Oda will discuss how pairing different extracellular and intracellular domains in FPs confers different attributes to therapeutic T cells.

Topics to be covered

Shannon K. Oda, PhDPrincipal InvestigatorSeattle Childrens Research InstituteAssistant ProfessorDepartment of Pediatrics Division of Hematology/Oncology University of Washington School of Medicine

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Overcoming T Cell Therapy Barriers with Engineered Proteins - The Scientist

Scientists from Cedars-Sinai Medical in Los Angeles are investigating how to grow large batches of a specific type of stem cell.

Their mission has taken them orbital to the International Space Station and it could help unlock a whole host of stem cell therapies to combat deadly diseases.

One of the researchers, Dhruv Sareen, even donated his own stem cells for the experiment, a press statement reveals. If all goes to plan, the scientists hope to eventually grow billions of stem cells in space.

Stem cells can be used to generate nearly any type of cell in the body. Due to their impressive adaptability, they have great potential as a key tool in developing a wide array of treatments for diseases, includingParkinson's and heart disease.

Sareen's cells arrived aboard a SpaceX resupply mission theSpX-25 dragon cargo mission to the ISS over the weekend."I don't think I would be able to pay whatever it costs now" to travel to space as a tourist, Sareen said. "At least a part of me in cells can go up!"

Several stem cell experiments have been conducted aboard the ISS in the past, as the microgravity conditions aboard the orbital space station provide a wildly different environment in which to investigate new methods and applications. Though most stem cell therapies are still some way off from coming to consumers, space could help overcome one of the main logistical hurdles when it comes to mass production.

In the future, stem cell therapy patients could require billions of cells depending on their treatment. Earth's gravity makes it difficult to grow the vast quantities of stem cells needed for therapies, so that's where the ISS comes in. "With current technology right now, even if the FDA instantly approved any of these therapies, we don't have the capacity to manufacture,"Jeffrey Millman, a biomedical engineering expert at Washington University in St. Louis, explained.

This is because, on Earth, large bioreactors are required to grow stem cells. The cells need to be stirred vigorously, so they don't clump together or fall to the bottom of the tank. However, the stirring itself can damage the cells. In microgravity, there's no force on the cells so that they can grow via a different method.

The Cedars-Sinai team sent up a shoebox-sized container holding pluripotent stem cells for their NASA-funded experiment. It contains pumps and chemical solutions required to keep the stem cells alive for four weeks. The same experiment will be carried out on Earth for comparison. In roughly five weeks, the box in space will be returned to Earth in the same SpaceX capsule it was carried up on. The rideshare mission will allow the scientists to directly evaluate the results in space and on Earth within a short timeframe. This will provide valuable new data that could help unlock a promising new field of medical research.

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Scientists aim to grow billions of stem cells aboard the International Space Station - Interesting Engineering

Newly published results from a Phase I clinical trial suggest that mesenchymal stromal cells (MSCs, a type of cell derived from bone marrow and other bodily tissue) can be used as a safe and potentially effective therapy for the treatment of lupus.

In this Phase I study, six women with refractory lupus (lupus that is resistant to treatment) were treated with MSCs derived from umbilical cord tissue. Three of the participants were Caucasian, two were African American and one was Hispanic.

Overall, five of the six women achieved a clinically meaningful decrease in lupus disease activity scores compared to their baseline level of disease activity, as measured by the Systemic Lupus Erythematosus Disease Activity Index (SLEDAI). By week 24 post-treatment, SLEDAI scores decreased by an average of 5.4 points, from an average of 8.2 at baseline to 2.8 at the end of the trial period.

Additionally, researchers noted encouraging changes in other lupus biomarkers among the participants, and no serious side effects were observed. Based on these promising early results, researchers are moving forward with a Phase II trial, which will include a larger group of study participants and collect data over a longer time period to assess its effectiveness.

This study was funded by the Lupus Foundation of America in partnership with the National Institute of Allergy and Infectious Diseases. MSC therapy is an exciting new frontier in lupus research, and its especially promising for those living with refractory lupus. Learn more about these Phase I and Phase II trials, and the importance of MSC research.

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New Mesenchymal Stromal Cell Therapy Shows Promise for People with Difficult-to-Treat Lupus - Lupus Foundation of America

Acute myeloid leukemia (AML) is a type of blood and bone marrow cancer. It affects the ability of your bone marrow to produce normal-functioning white blood cells (WBCs). The risk of this type of cancer increases with age, and is more common in older adults.

If you have AML, your bone marrow produces an abnormal amount of WBCs that are immature and dont function properly. These abnormal WBCs eventually build up and replace healthy cells.

The early symptoms of AML can be similar to the flu and may include fever, fatigue, and bone pain.

There are a handful of treatments available for AML, all of which carry a risk of short- and long-term side effects. This article will look at at the side effects associated with AML treatment and how to manage them.

AML treatment typically has two phases. The first phase involves destroying active leukemia cells, while the second phase involves keeping AML in remission.

According to the National Cancer Institute (NCI), there are five main treatments available for AML:

When standard treatments for AML arent successful, clinical trials are another treatment option.

All cancer treatments carry a risk of side effects. With AML treatment, there are many possible side effects.

The range and severity of your side effects will depend on the type of treatment. In addition, if you receive chemotherapy, side effects will depend on the type of chemotherapy drug you receive.

Other factors that can affect your side effects include your age, sex, and overall health.

With that said, side effects of AML treatment can be intense, according to a 2019 survey of 1,182 AML survivors.

The survey results indicated that:

The authors of the study concluded that side effects of treatment for AML constitute a substantial burden for survivors.

Short-term side effects occur at the start of treatment. Theyre temporary, and usually only last for the duration of the therapy itself.

According to the 2019 survey cited above, the most common and severe short-term side effects of AML treatment included hair loss, experienced by 78% of participants, and fatigue, experienced by 33% of participants.

Other possible short-term side effects of AML treatment include:

Side effects of AML treatment can continue or develop after the treatment is finished. Some possible long-term side effects of AML treatment include:

Its not clear how many AML survivors experience long-term and/or late effects of treatment. In addition, the risks may be greater for certain groups.

For example, a 2021 study of 1,168 adolescent survivors of AML found that 26% experienced endocrine problems later in life, including hypothyroidism, diabetes, and fertility issues.

Cardiovascular problems, such as hypertension and heart diseases, were also common among young adult survivors, affecting around 19%. About 7% of adolescent survivors experienced long-term respiratory issues.

Among adult survivors of AML, weakness and fatigue are common long-term symptoms. A 2018 study followed up with adult AML survivors at 1, 2, and 3 years. The authors reported that after 3 years, 23% still experienced fatigue.

In addition, among older adults with AML, many didnt fully recover their physical strength and functioning after 3 years.

AML is an aggressive cancer that requires intensive treatment. The side effects can be uncomfortable and are likely to temporarily impact your quality of life.

In the past several years, there have been substantial improvements in treatment outcomes. According to the NCI, between 60% and 70% of adults with AML go into remission after appropriate treatment.

In addition, over 25% of adults with AML can expect to survive for 3 or more years after their initial diagnosis.

Treatments for AML include chemotherapy, radiation, and stem cell transplants. Targeted therapies and other drug therapies are also available.

All of these treatments can lead to side effects, some of which can persist in the long term. Some common side effects include hair loss, fatigue, and fertility issues.

Your doctor can help you understand what to expect with treatment, including potential side effects. Remember that you have options.

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AML Treatment Side Effects: What to Expect, How to Manage - Healthline

Treatment uses modified white blood cells to target cancer cells that are resistant to standard therapies.

The McGill University Health Centre (MUHC) Cancer Care mission is pleased to announce that the Ministry of Health and Social Services (MSSS) has officially designated the MUHC as a provider of the Immune Effector Cell Therapy CAR-T Program for adult patients. Patients with specific types of leukemia or lymphoma that are resistant to standard therapies will now be able to benefit from this innovative treatment, which uses genetically modified white blood cells to attack and destroy cancer cells. The MUHC has been added to the list of centres designated by the MSSS to administer the therapy, which is also available at the Hpital Maisonneuve-Rosemont, the Centre hospitalier universitaire Sainte-Justine of the CIUSSS de lEst-de-lle-de-Montral and the Centre hospitalier universitaire de Qubec Universit Laval.

We are delighted to be able to extend the services of this innovative therapy and allow more patients to benefit from this leading-edge technology in Quebec. Each year, about 20 patients will be admitted to the MUHC and treated by a dedicated and qualified team, says Dr. Pierre Gfeller, President and Executive Director of the MUHC.

The CAR-T therapy is a customized treatment made from T-cells, a type of white blood cell that plays a crucial role in the proper functioning of the immune system. These cells are taken from the patient's blood and then modified in the laboratory to carry a new gene that allows them to target cancer cells. The modified lymphocytes thus created are then administered to the patient in a single intravenous dose.

At the MUHC, the treatment is available to the following patients who have not responded to standard therapies: those over the age of 18 with diffuse large B-cell lymphoma (DLBCL) and those aged 18-25 with B-cell acute lymphoblastic leukemia (b-ALL), who have not responded to standard therapies. The indications for this treatment will no doubt change with time.

The MUHC has assembled a dedicated and professional team to manage all aspects of this program, as the medical director of the Immune Effector Cell Therapy Program Dr. Michael Sebag, attests. This groundbreaking therapy has been made possible through exceptional teamwork for the patients who need it. We are committed to providing our patients with unparalleled service and we are pleased that these years of hard work have come to a result, he says.

This is a real opportunity for our patients who often have no other treatment options, says Christine Bouchard, clinical-administrative coordinator of the MUHC Cancer Care Mission.

We are pleased to open the MUHC CAR-T Program to the public and to become one of the designated centres in Quebec to offer this innovative form of immune therapy. The program would not have been possible without the outstanding teamwork and support of the MUHC, adds Dr. Gizelle Popradi, director of the MUHC Stem Cell Transplant Program.

The treatment tisagenlecleucel, known under the trade name KYMRIAHMDand developed by the company Novartis received approval from Health Canada in September 2018 and subsequently received a positive opinion from the National Institute of Excellence in Health and Social Services (INESSS) in January 2019. It is the first personalized T-cell therapy approved in Canada. YESCARTA received its approval from Health Canada in February 2019 and became reimbursed in Quebec as of November 2020. ABECMA received its approval from Health Canada in May 2021, for treatment of multiple myeloma, but remains under qualifying notice and is not yet approved for use in Quebec or Canada.

At the MUHC, the clinical team is trained to offer this new category of therapy and is ready to welcome its first patient, says Chantal Souligny, recently retired director of nursing and project sponsor. This is a new chapter for the MUHC Cancer Care mission.

Media contact

Evelyne DufresneInformation OfficerMcGill University Health Centreevelyne.dufresne@muhc.mcgill.ca

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MUHC offers immunocellular therapy for patients with leukemia or lymphoma - McGill University Health Centre

In a filing to the Securities and Exchange Commission (SEC), Legend Biotech announced it had informed the Food and Drug Administration (FDA) that it has terminated a phase 1 clinical trial investigating the efficacy of its novel CAR-T cell therapy LB1901 in adults with relapsed or refractory T-cell lymphoma, a rare type of blood cancer.

The manufacturer noted the decision was based on a lack of benefit associated with a similar CAR-T cell product with the same protein as LB1901 that was being analyzed in a different study. Legend Biotech also mentioned that the decision was made to cancel the trial to focus on other drug candidates it is developing.

In February of this year, the FDA placed a clinical hold on the trial after the manufacturer informed the agency that low CD4-positive T-cell counts were observed in the peripheral blood of the only patient to have been treated with the investigational therapy.

Previous findings published in the journal Leukemia & Lymphoma have shown that low CD4-positive T-cell counts have been associated with poor prognosis in patients with newly diagnosed multiple myeloma, which is another rare blood cancer that affects plasma cells.

The decision to terminate the trials comes several weeks after Legend reported that the FDA had lifted the hold on the trial.

For more news on cancer updates, research and education, dont forget tosubscribe to CUREs newsletters here.

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Early-Phase Trial Assessing Novel CAR-T Cell Therapy in a Type of Blood Cancer Terminated - Curetoday.com

TAIPEI (Taiwan News) A 10-year-old girl suffering from leukemia is the first child in Taiwan to receive CAR T-cell therapy and to have fully recovered from the cancer as a result.

The girl, identified as Tingting (), was diagnosed with childhood B-cell acute lymphoblastic lymphoma four years ago. After undergoing first-line therapy, she still relapsed.

In the past, such a patient would have to wait for a stem cell transplant to save their lives. However, with the assistance of doctors at National Taiwan University Hospital (NTUH), she became the first CD19-targeted chimeric antigen receptor-engineered (CD19 CAR) T-cell recipient in Taiwan and has fully recovered, with no residual cancer cells detected in her body.

At a press conference held by the university on Thursday (July 14), Chou Hsien-tang (), a hematologist oncologist at NTUH, said that Tingting had been diagnosed with the cancer when she was 6 years old, reported ETtoday. After receiving a three-year chemotherapy regimen for high-risk groups, the remaining cancer cells were undetectable for a period of time.

However, the cancer recurred, and even with the use of new standard drugs, the cancer cells could not be removed. After discussion with parents, doctors decided to attempt CART T-cell therapy.

Chou explained that the treatment principle relies on high-tech genetic engineering. First, T-cells are isolated from the patient's body, and are genetically modified by adding a gene for a receptor called chimeric antigen receptor (CAR), which enables the T-cells to attach to a specific cancer cell antigen.

The cancer cells from childhood B-cell acute lymphoblastic lymphoma contain an antigen called CD19. Therefore, in this patient's case, the CART T-cell technique was used to design T-cells to attach to the CD19 antigen.

Chou compared it to a precise "immunization army" that can accurately and continuously destroy cancer cells. The advantage is that a one-time injection can generate these results, said Chou, as was the case with Tingting.

In April of this year, NTUH became the first medical center in Taiwan to provide formal clinical use of CD19-targeted CAR T-cell therapy. Tingting was the first patient in Taiwan to receive the treatment and experience a full recovery.

Currently, no cancer cells have been detected in her system, but follow-up examinations will continue to monitor her status. NTUH expressed the hope that it will be able use this therapy in the future to improve the quality of cancer treatment and prevent recurrences.

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Taiwan reports 1st child with cancer cured by CAR T-cell therapy - Taiwan News

Chimeric antigen receptor T-cell therapy weaponizes our natural immune cells to make them more effective cancer killers. It extracts a small number of white blood cells, called T cells, from a patients blood, then genetically modifies them to kill cancer. Those cells are then infused back into the patient, where they replicate into a vast army of cancer killers. The entire process takes about two weeks, as opposed to other processes that can take months.

CAR T-cell immunotherapy has had significant success in battling blood cancers, but it has been less effective against solid tumors. Lees new finding could enhance its ability to target these tumors that now resist CAR T-cell treatment.

His new work identifies specific targets on glioblastoma and DIPG cells that the immunotherapy could exploit. When tested in mice, the approach was very effective the tumors shrank or disappeared, and the weaponized immune cells proved long-lasting. The approach also avoided side effects that have previously hampered its use in people.

Particularly notable was the approachs effectiveness againstDIPG, a cancer that threads itself through the brain, making it extremely hard to treat. Lees immunotherapy approach shrank thetumors and improved survival among lab mice. The scientists note that the cancers invasive nature likely will continue to pose a challenge, especially when the cancers tentacles extend into vital areas of the brain, but the researchers were encouraged by the promising results.

Many solid tumors share the same vulnerabilities Lees team identified in glioblastoma andDIPG. That suggests the results could have wider applications for solid tumors.

We have already begun to see if we can use this same therapy to treat other tumors, like melanoma, breast cancer and the pediatric muscle tumor rhabdomyosarcoma, in the lab, said Lee, a pediatric oncologistat UVA Childrens and UVA Cancer Center. My team in the lab is working incredibly hard to understand how we can bring new and safer CAR T-cell therapies to the citizens of Virginia and beyond. Our findings in brain tumors represent a huge leap forward in doing just that, but there is much more work to be done.

While the new discovery has shown exciting promise, significantly more research will be needed before it can be translated into better treatments forpatients. This type of cutting-edge researchis an urgent mission of UVA Cancer Center, which is one of only 52 cancer centers in the country to be designated as a Comprehensive Cancer Center by the National Cancer Institute.The designationrecognizes elite cancer centers with the most outstanding cancer research and treatment programs in the nation.

UVA Cancer Center is the only Comprehensive Cancer Center in Virginia.

Reference:Cobb DA, Rossi J de, Liu L, An E, Lee DW. Targeting of the alphav beta3 integrin complex by CAR-T cells leads to rapid regression of diffuse intrinsic pontine glioma and glioblastoma. J Immunother Cancer. 2022;10(2):e003816. doi:10.1136/jitc-2021-003816

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