Category Archives: Cell Therapy

New Cleveland Clinic research has uncovered key information about cellular interaction between tumor cells and normal tissue, providing better understanding of how therapeutic resistance develops.

In the study of drug resistance, researchers often try to understand the fitness of cells that have specific mutations in the presence of a drug in a laboratory setting, explained Jacob Scott, M.D., D.Phil., radiation oncologist and head of Cleveland Clinics Theory Division in the Lerner Research Institute Department of Translational Hematology and Oncology Research. But the reality is more complex, because tumor cells dont exist in a vacuum; instead, they co-exist in a complex, heterogeneous mixture of other tumor cells and normal tissues an interacting ecology.

Jacob Scott, M.D., D.Phil.

With approximately 90% of cancer deaths attributed to treatment-resistant disease, these cellular interactions, also known as evolutionary games, have high stakes.

In their latest study published July 1 in Science Advances, Dr. Scott and his collaborators used an assay they previously developed to directly measure those interactions in a simplified tumor environment consisting of drug-resistant non-small cell lung cancer cells and drug-sensitive precursor (ancestor) cells.

Cells resistant to the metastatic non-small cell lung cancer treatment gefitinib were derived from existing lung cancer cells by continual treatment with gefitinib over six months, and grown in an in vitro co-culture experiment with their sensitive ancestors. Cellular growth dynamics were assessed with and without gefitinib.

We cultured the two groups of cells together in different starting fractions and we measured how their growth changed depending on how much of each group was mixed together, explained Jeff Maltas, Ph.D., a postdoctoral researcher at Cleveland Clinic and co-lead author on the study.

The researchers found that the fitness of the resistant typeof cellchanges drastically depending on the composition of the mixture. Experiments revealed that the resistant population was outcompeted by the ancestral line at all studied population frequencies in the absence of therapy, pointing to complete competitive exclusion of the resistant populationand a cost of resistance. When gefitinib was added, there was a complete reversal of this effect, and the resistant clone was able to outcompete the sensitive ancestor.

This changing growth dynamics between treatment-resistant and treatment-sensitive cells could not be detected by standard assays available to date and suggests a novel mechanism by which resistant cells persist in the absence of treatment.

This paper highlights the need to understand and directly measure those ecological interactions that come from being around other cell types, Dr. Scott said. Thats something were doing here thats fundamentally different.

Dr. Scott notes that although these latest findings cannot be directly extrapolated to clinical scenarios, they do raise important questions with potentially significant implications on clinical research and trial design. In broader terms, could patients benefit from taking a break from medication to allow the maintenance of a critical treatment-sensitive population of tumor cells? And, could treatment regimens consisting of on-treatment and off-treatment periods allow for better control of cancer cell growth and, in the long term, prolong the lives of patients?

One of the most exciting parts of this paper is the opportunity for clinical advances that it highlights, Dr. Scott said. The design and testing of dynamic treatment regimens that could keep cancer cells in check over the long term may be the next translational application of these important findings.

Cleveland Clinics Theory Division conducts cancer research to develop and make available novel diagnostic tools, targeted therapies and clinical trials for direct use in patient care.

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Cleveland Clinic Researchers Shed New Light on Importance of Cellular Interactions in Targeted Therapy Resistance in Lung Cancer - Cleveland Clinic...

HOUSTON, June 27, 2022 (GLOBE NEWSWIRE) -- Alaunos Therapeutics, Inc. (Alaunos or the Company) (Nasdaq: TCRT), a clinical-stage oncology-focused cell therapy company, today announced that the Company has extended its Cooperative Research and Development Agreement (CRADA) with the National Cancer Institute (NCI), an institute of the National Institutes of Health, using the Alaunos Sleeping Beauty technology through January 2025.

Under the terms of the CRADA, the NCI will work to generate proof of concept utilizing the Companys proprietary non-viral Sleeping Beauty technology for personalized TCR-T cell therapy. In this setting, T-cell receptors (TCRs) that react to the patients tumor will be identified from the patient and used to generate a TCR-T cell therapy. This approach could potentially apply to a wide range of solid tumor cancer patients. Alaunos believes that the non-viral Sleeping Beauty technology could rapidly and cost effectively produce safe and potent TCR-T cell therapies without the complexity of geneediting or viral approaches. Research conducted under the CRADA will be led by Steven A. Rosenberg, M.D., Ph.D., Chief of the Surgery Branch at the NCI's Center for Cancer Research.

We are privileged to extend the productive collaboration with Dr. Rosenberg, a cell therapy pioneer. Dr. Rosenberg and the NCI are working to develop personalized cancer therapies using our novel TCR-T cell platform, commented Kevin S. Boyle, Sr., Chief Executive Officer of Alaunos. Our collaboration reinforces our commitment to improving the lives of cancer patients with solid tumors. We look forward to continuing our collaborating with Dr. Rosenberg and his team to generate proof of concept in this personalized TCR-T approach.

Drew Deniger, Ph.D., Vice President, Research & Development at Alaunos added, Having worked alongside Dr. Rosenberg for many years, I am confident that his team at the NCI will be successful in developing personalized TCR-T therapies using our non-viral Sleeping Beauty technology. As the worlds experts in Sleeping Beauty, we believe that our non-viral means of adding the TCR to T cells is well suited for a personalized approach, with potential to further increase the addressable population for TCR-T therapies.About Alaunos TherapeuticsAlaunos is a clinical-stage oncology-focused cell therapy company, focused on developing T-cell receptor (TCR) therapies based on its proprietary, non-viral Sleeping Beauty gene transfer technology and its TCR library targeting shared tumor-specific hotspot mutations in key oncogenic genes including KRAS, TP53 and EGFR. The Company has clinical and strategic collaborations with The University of Texas MD Anderson Cancer Center and the National Cancer Institute. For more information, please visit http://www.alaunos.com.

Forward-Looking Statements Disclaimer This press release contains forward-looking statements as defined in the Private Securities Litigation Reform Act of 1995, as amended. Forward-looking statements are statements that are not historical facts, and in some cases can be identified by terms such as may, will, could, expects, plans, anticipates, believes or other words or terms of similar meaning. These statements include, but are not limited to, statements regarding the Company's business and strategic plans, the anticipated outcome of preclinical and clinical studies by the Company or its third-party collaborators, the Company's cash runway, and the timing of the Company's research and development programs, including the anticipated dates for filing INDs, enrolling and dosing patients in and the expected timing for announcing preclinical data and results from the Companys clinical trials. Although the management team of Alaunos believes that the expectations reflected in such forward-looking statements are reasonable, investors are cautioned that forward-looking information and statements are subject to various risks and uncertainties, many of which are difficult to predict and generally beyond the control of Alaunos, that could cause actual results and developments to differ materially from those expressed in, or implied or projected by, the forward-looking information and statements. These risks and uncertainties include, among other things, changes in the Companys operating plans that may impact its cash expenditures; the uncertainties inherent in research and development, future clinical data and analysis, including whether any of Alaunos product candidates will advance further in the preclinical research or clinical trial process, including receiving clearance from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies to conduct clinical trials and whether and when, if at all, they will receive final approval from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies and for which indication; the strength and enforceability of Alaunos intellectual property rights; and competition from other pharmaceutical and biotechnology companies as well as risk factors discussed or identified in the public filings with the Securities and Exchange Commission made by Alaunos, including those risks and uncertainties listed in the most recent periodic report filed by Alaunos with the Securities and Exchange Commission. Alaunos is providing this information as of the date of this press release, and Alaunos does not undertake any obligation to update or revise the information contained in this press release whether as a result of new information, future events, or any other reason.

Investor Relations Contact:Alex LoboStern Investor RelationsAlex.lobo@sternir.com

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Alaunos Therapeutics and the National Cancer Institute Extend Cooperative Research and Development Agreement for Development of Personalized TCR-T...

LONDON, June 30, 2022 (GLOBE NEWSWIRE) -- Achilles Therapeutics plc (NASDAQ: ACHL), a clinical-stage biopharmaceutical company developing precision T cell therapies to treat solid tumors, today announced the appointment of James Taylor as Chief Business Officer and Cassian Yee, MD to its Scientific Advisory Board (SAB), effective July 1, 2022.

James brings over 25 years of value-creating deal experience that includes global platform and asset deals for pharmaceutical and biotechnology companies. Most recently, he was Chief Business Officer at Sosei Heptares, where he completed a major collaboration with Neurocrine Biosciences for a Phase II-ready M4 agonist for schizophrenia, and led deals with Genentech, Takeda, AbbVie, GSK, Verily, Aditum and Biohaven Pharmaceuticals. Prior to this, James was a senior director at GSK where he led deals with GlycoVaxyn, Novartis, Pfizer, Biological E and consulted for Galvani Bioelectronics, the GSK/Google joint venture. He has held executive management roles at AstraZeneca, Summit Therapeutics and Cellzome.

Achilles has pioneered clonal neoantigen-targeted therapy and AI-powered bioinformatics, offering the potential to unlock therapeutic capabilities that I believe will have a tremendous impact for patients, commented James Taylor, incoming Chief Business Officer at Achilles Therapeutics. I look forward to exploring strategic partnerships to maximize the value of precision T cell therapies and platform technologies for the treatment of solid tumors.

The Company has also expanded its Scientific Advisory Board with the addition of Cassian Yee, MD. Dr. Yee is a professor in the department of melanoma medical oncology and the department of immunology at The University of Texas MD Anderson Cancer Center, as well as director of the solid tumor cell therapy program at MD Anderson. He is a highly regarded immuno-oncology leader, and notable pioneer in the field of adoptive cellular therapy.

I am delighted to work closely with the team at Achilles as their precision T cell therapies further advance in the clinic, said Cassian Yee, MD, Director, Department of Solid Tumor Cell Therapy Program, Division of Center for Cancer Immunology Research, MD Anderson Cancer Center. I believe the neoantigen field is poised to make significant advances in the treatment of late-stage solid tumors and Achilles novel platform and modality can be part of that evolution.

About Achilles TherapeuticsAchilles is a clinical-stage biopharmaceutical company developing precision T cell therapies targeting clonal neoantigens: protein markers unique to the individual that are expressed on the surface of every cancer cell. The Company has two ongoing Phase I/IIa trials, the CHIRON trial in patients with advanced non-small cell lung cancer (NSCLC) and the THETIS trial in patients with recurrent or metastatic melanoma. Achilles uses DNA sequencing data from each patient, together with its proprietary AI-Powered PELEUS bioinformatics platform, to identify clonal neoantigens specific to that patient, and then develop precision T cell-based product candidates specifically targeting those clonal neoantigens.

Investors:Achilles TherapeuticsLee M. Stern, VP, IR & External Communicationsl.stern@achillestx.com

LifeSci AdvisorsJohn Mullalyjmullaly@lifesciadvisors.com

Media:Consilium Strategic CommunicationsMary-Jane Elliott, Sukaina Virji, Melissa Gardiner+44 (0) 203 709 5000achillestx@consilium-comms.com

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Achilles Therapeutics Appoints James Taylor as Chief Business Officer and Cassian Yee, MD to Scientific Advisory Board - GlobeNewswire

When you're an oncologist and clinical trialist who finds yourself at the epicenter of a global health crisis, what do you do? Ajai Chari, MD, professor of medicine and the director of clinical research in the multiple myeloma (MM) program at the Icahn School of Medicine at Mount Sinai in New York, NY, saw an opportunity to contribute from the earliest days of the COVID-19 pandemic.

Even as New York City recorded nearly 25% of US deaths from COVID-19 in spring 2020 and the rest of the world watched in horror as the city set up makeshift morgues in refrigerated trucks,1,2 Chari spearheaded an international clinical study on behalf of the International Myeloma Society (IMS) to gather the initial data and perform the f irst large-scale analysis of patients with MM who contracted COVID-19.3

Charis longtime mentor and colleague Sundar Jagannath, MBBS, director of the Center of Excellence for Multiple Myeloma at Mount Sinai, believes his mentees work on this study sums up Chari very well. It was an honor [that Chari was] chosen to lead the IMSs study and it shows that he is highly respected and well thought of in the international myeloma community, Jagannath said. He is devoted to his patients and his trainees and is extraordinarily detail-oriented in everything he does.

This fall, Chari will turn that attention to detail to cochairing the 40th Annual CFS: Chemotherapy Foundation Symposium Innovative Cancer Therapy for Tomorrow. Physicians Education Resource, LLC (PER) is hosting the 3-day event November 9 to 11 as a hybrid meeting featuring live, interactive presentations in New York, NY, along with a virtual option.

When Chari sat down recently with OncologyLive to discuss current trends in therapeutics in MM, he started with a story to illustrate just how quickly the treatment landscape is changing. In 2018, he discussed hospice with a patient with advanced MM refractory to all available therapies and chronic kidney dysfunction that made it difficult to enroll him in nearly all clinical trials. Fortunately, he was able to participate in one study. Four years later he is in a stringent complete remission and in a bit of an existential crisis because he didnt think hed be alive today, Chari said. This case highlights the unprecedented pace of myeloma research in the last few years.

Charis patient joined the phase 2 STORM trial (NCT02336815), which evaluated the combination of oral selinexor (Xpovio) plus low-dose dexamethasone in patients with refractory heavily pretreated MM. The patient saw significant clinical improvements, including in his renal function, enabling him to join other clinical trials. When the FDA approved selinexor for patients with MM in 2019,4 the patient was able to receive the therapy again in combination with other MM agents. After his disease went into remission again, this patient has now received 2 different T-cellredirection therapies when those remissions ended.

Chari contrasts the current regulatory environment with that of even a few years ago. Historically, in multiple myeloma, to receive accelerated approval for a new drug for patients with an unmet need (ie, those who had exhausted all available therapies), you needed a 20% to 30% response rate for 3 to 4 months. Now we have game-changing T-cellredirecting therapies, such as CAR [chimeric antigen receptor] T-cell therapies and bispecific antibodies, he explained. These agents have shown outstanding results, blowing those previous numbers out of the water. T-cellredirection therapies are now giving anywhere from 60% to 100% response rates that are lasting upward of 9 months to we dont even know how long, because theyre so durable.

Even as new MM therapies receive approval, areas of unmet need remain. The definition of unmet need in myeloma is constantly changing as the drugs get better and better, but we have much more work left to do, Chari said.

Current areas of unmet need include as follows:

Although many of these needs will ultimately be tackled by new therapeutics, sometimes these challenges can be addressed by refining standards of care. This is one of Charis particular strengths, said Hearn Jay Cho, MD, PhD, chief medical officer of the Multiple Myeloma Research Foundation and associate professor of medicine at the Icahn School of Medicine at Mount Sinai. He cited a recent phase 2 study (NCT02176213) from Chari and colleagues that examined a regimen combining pomalidomide (Pomalyst), cyclophosphamide, and dexamethasone in patients with relapsed/refractory MM (RRMM).5

Chari and colleagues hypothesized that daily dosing of oral cyclophosphamide would yield good results while meshing well with oral formulations of the other 2 drugs. In 33 evaluable patients, the overall response rate (ORR) was 73%, the median progression-free survival (PFS) was 13.3 months (95% CI, 7.9-not reached [NR]) and the median overall survival (OS) was 57.2 months (95% CI, 42.2-NR). The investigators concluded that adding daily cyclophosphamide to the regimen is a cost-effective oral strategy with encouraging PFS. All-oral treatment options that are well tolerated are especially important for older patients or those who cant travel to receive care frequently, Cho said. This is a great example of Chari applying a creative solution to an unmet need in the myeloma field.

Since 2010, when Chari and Jagannath joined Mount Sinai, Chari has served as principal investigator on 36 clinical trials and as a co-investigator on 68 additional trials. Whats more, he has developed 6 investigator-initiated clinical trials with a cumulative $4.3-million budget and worked on virtually every recent FDA approval for MM therapies. Chari offered a snapshot of key therapeutic advances in MM, all of which will be discussed at the CFS meeting.

One of the most stellar achievements of the past decade in myeloma has been the development of CAR T-cell therapies, in which patients own T cells are collected and genetically modified prior to reinfusion. First came idecabtagene vicleucel (ide-cel; Abecma), a B-cell maturation antigen (BCMA)directed CAR T-cell therapy that received FDA approval in March 2021.6

This approval covers adult patients with RRMM who have received 4 or more prior lines of therapy, including an anti-CD38 monoclonal antibody (mAb), a proteasome inhibitor (PI), and an immunomodulatory (IMiD) agent. Approval was based on the phase 2 KarMMa trial (NCT03361748) in which 33% of treated patients had a complete response (CR).7 Investigators confirmed minimal residual disease (MRD)negative status in 33 patients, representing 26% of all 128 patients who were treated and 79% of the 42 patients who had a CR or better. After a median follow-up of 13.3 months, the ORR among all treated patients was 73% (95% CI, 66%-81%).

In February 2022, the FDA approved ciltacabtagene autoleucel (cilta-cel; Carvykti), also a BCMA-directed CAR T-cell therapy, for patients with RRMM after 4 or more prior lines of therapy, including a PI, an IMiD, and an anti-CD38 mAb.8 The decision was based on findings from the phase 1/2 CARTITUDE-1 trial (NCT03548207), an open-label, multicenter study that tested the treatment in 97 patients with RRMM. The ORR was 97.9% (95% CI, 92.7%-99.7%). Among 95 responders, the median duration of response (DOR) was not reached.8,9

Both ide-cel and cilta-cel have been game-changers for our patients in that they are nearly one-and-done therapies. Patients who used to get continuous therapy indefinitely get this one intervention and then enjoy treatment-free intervals that didnt seem possible not long ago, Chari said. Their success has caught us by surprise because usually patients with myeloma have pretty beat-up immune systems with 5 or 6 years of chemotherapy and transplant behind them, so its remarkable that these T cells even work, let alone work this well.

Chari is mindful of the CAR T-cell therapies significant shortcomings, including limited availability from manufacturers, supply chain challenges, and high price tags. There are newer forms of CAR T cells under development that are looking at shorter manufacturing times [due to] an off-the-shelf design, he said.

Bispecific antibodies are another novel therapy on the horizon in MM. Although no such agents have received FDA approval for MM so far, many therapeutics appear promising. In the MM space, there are at least 12 bispecific antibodies undergoing clinical testing, and additional agents are in preclinical development, Chari and colleagues at Mount Sinai wrote in a recent clinical review in Blood Cancer Discovery.10 They said this class of drugs likely will become an important addition to the field, particularly for triple-class refractory, heavily treated disease.

Two of these bispecifics, talquetamab (JNJ64407564) and teclistamab (JNJ-64007957), have been in the spotlight at recent conferences. Chari has been instrumental in studies of both therapies.

Talquetamab is an investigational T-cellredirecting bispecific antibody targeting both GPRC5D, a novel MM target, and CD3, a T-cell receptor. MonumenTAL-1 (NCT03399799), a phase 1 first-in-human dose-escalation study, showed that talquetamab monotherapy administered subcutaneously (sub-Q) evoked high overall responses that deepened over time at 2 different dosing schedules in patients with triple-class and penta-drug refractory disease, according to findings presented at the 2022 American Society of Clinical Oncology Annual Meeting (2022 ASCO).11

With a median follow-up of 13.2 months, the ORR was 70.0% among 30 patients treated with talquetamab at 405 g/kg weekly, including a very good partial response (VGPR) in 56.7% of participants. The ORR was 63.6%, including a VGPR of 56.8%, among 44 patients who received talquetamab at 800 g/kg every other week after a median follow-up of 7.7 months. The median DOR was 10.2 months (95% CI, 3.0-not estimable [NE]) among participants who responded to weekly dosing and 13.0 months (95% CI, 5.3-NE) for the biweekly dosing. The most common nonhematologic adverse events (AEs) were cytokine release syndrome (CRS), skin-related AEs, and dysgeusia, mostly of grade 1 or 2 severity. Grade 3/4 AEs included 1 patient with CRS at the weekly dosing level and 1 with skin-related reactions with biweekly dosing.

Talquetamab monotherapy is being evaluated at both dosing levels in the phase 2 multicohort TALMMY1001-PT3 study (NCT04634552), which seeks to enroll an estimated 320 participants with RRMM after 3 or more prior lines of therapy. Additionally, the agent is undergoing testing in combination with other therapies in phase 1 studies.11

Chari also is an investigator on the multicohort phase 1b TRIMM-2 study (NCT04108195) testing daratumumab with hyaluronidase-fihj (Darzalex Faspro), a sub-Q formulation of daratumumab, plus talquetamab or teclistamab with or without pomalidomide (Pomalyst).12,13

Sub-Q daratumumab is itself a relatively novel form of the anti-CD38 mAb; the FDA initially approved the formulation in May 2020 as monotherapy and in combination for several indications held by the intravenous (IV) form of daratumumab (Darzalex).14 Teclistamab is a bispecific antibody that targets both CD3 expressed on the surface of T cells and BCMA expressed on the surface of myeloma cells.15

During the 2022 European Hematology Association Annual Meeting, investigators presented updated findings for 51 evaluable patients who received talquetamab plus sub-Q daratumumab across dosing levels during TRIMM-2. At a median follow-up of 5.1 months across dosing cohorts, the ORR was 80.4%, including a CR or better in 29.4% and a VGPR or better in 62.7%. Additionally, the ORR was 77.3% among patients (34 of 44) with prior exposure to an anti-CD38 mAb and 72% among participants (18 of 25) with prior BCMA-targeted treatment. The safety profile of the combination was comparable with what has been reported with each agent separately.16

At 2022 ASCO, investigators reported findings for the TRIMM-2 cohort that received teclistamab plus sub-Q daratumumab. In 51 evaluable patients, the ORRs were 75.0% (15 of 20 patients), 74.1% (20 of 27 patients), and 100% (4 of 4 patients) at teclistamab dosing levels of 1.5 mg/kg weekly, 3 mg/kg biweekly, and 3 mg/kg weekly, respectively. All patients received 1800 mg of sub-Q daratumumab with each cycle.17

Teclistamab also demonstrated efficacy as monotherapy in the phase 1/2 MajesTEC-1 trial (NCT03145181; NCT04557098), whose results were simultaneously presented at 2022 ASCO and published in the New England Journal of Medicine (NEJM). The ongoing study involves patients with RRMM who have received at least 3 lines of therapy, including an IMiD, a PI, and an anti-CD38 mAb. Patients were treated with a weekly sub-Q injection of teclistamab dosed at 1.5 mg/kg of body weight after receiving 2 step-up doses.15,18

Among 165 patients who received teclistamab, the ORR was 63.0% (95% CI, 55.2%-70.4%), with 39.4% having a CR or better and 58.8% having a VGPR or better at a median follow-up of 14.1 months. Overall, 26.7% (95% CI, 20.1%34.1%) tested negative for MRD. Among the 65 patients with a CR or better response, the MRD negativity rate was 46.0%. The median DOR was 18.4 months (95% CI, 14.9-NE). The median PFS was 11.3 months (95% CI, 8.8- 17.1). Median OS data were immature.

When it comes to novel therapies for MM, the research team at Mount Sinai has been particularly prolific. Weve been involved in almost every single drug thats been FDA approved in the last 10 to 15 years, Chari said.

The list includes belantamab mafodotin-blmf (Blenrep), an antibody-drug conjugate directed against BCMA that obtained FDA accelerated approval in August 2020 for patients with RRMM who have received at least 4 prior lines of therapy.19 The drug was the first approved agent to target BCMA, a cell-surface receptor broadly expressed on MM cells but not on naive and memory B cells. Chari was among the investigators on the pivotal phase 2 DREAMM-2 study (NCT03525678).20

Additionally, Chari was the first author on an article in the NEJM describing the findings of the STORM trial, which paved the way for the FDAs approval of selinexor in combination with dexamethasone for adults with RRMM who have received at least 4 prior therapies and whose disease is refractory to at least 2 PIs, at least 2 IMiDs, and an anti-CD38 mAb. Selinexor inhibits exportin 1, which enables nuclear export of cargo proteins such as tumor suppressor proteins and is overexpressed in MM.4,21

Meanwhile, the further development of sub-Q daratumumab also has been on Charis radar. Daratumumab is a workhorse of the MM treatment landscape. Initially approved in 2015 as monotherapy for double- or triple-refractory MM after at least 3 prior lines of therapy, the IV formulation of daratumumab now carries multiple indications for relapsed and newly diagnosed disease in combination with other agents such as bortezomib (Velcade), carfilzomib (Kyprolis), lenalidomide (Revlimid), and pomalidomide.22

The sub-Q formulation of daratumumab requires shorter administration time3 to 5 minutes compared with 7 hours for the first infusion of IV daratumumab, and with 3 to 4 hours for subsequent cyclesaccording to findings from the COLUMBA trial (NCT03277105). Clinical outcomes with the sub-Q version were noninferior to those with the IV format and there were fewer infusion-related reactions.23

Chari and colleagues demonstrated the efficacy of sub-Q daratumumab in several standard-of-care MM combination regimens in the phase 2 PLEIADES trial (NCT03412565).24 The study findings formed the basis for the FDAs approval of 3 indications for combination regimens incorporating the sub-Q format: with bortezomib, melphalan, and prednisone in patients with newly diagnosed MM who are ineligible for transplant; with lenalidomide/ dexamethasone for RRMM; and with carfilzomib/dexamethasone in RRMM.25

The PLEIADES study looked at various daratumumab combinations in the subcutaneous formulation, Chari said. Key takeaways were that subcutaneous daratumumab has efficacy and pharmacokinetics comparable with what you would expect based on monotherapy and IV combinations of the respective agents.

The plethora of novel therapies that have been introduced to the MM treatment paradigm in recent years has ignited debate about the future role of autologous stem cell transplant (ASCT).

Findings from the phase 3 MAIA study (NCT02252172) in patients with newly diagnosed MM who were not eligible for ASCT demonstrated that the addition of IV daratumumab to lenalidomide plus dexamethasone resulted in superior outcomes compared with a lenalidomide/dexamethasone combination.26

After a median follow-up of 56 months, the median PFS among patients who received daratumumab (n = 368) was not reached (95% CI, 54.8-NR) compared with 34.4 months (95% CI, 29.6-39.2) in participants (n = 369) treated with lenalidomide/dexamethasone alone (HR, 0.53; 95% CI, 0.43-0.66; P < .0001). Median OS was not reached in either group. The median age of participants was 73 years (range, 70-78) in the daratumumab-containing arm and 74 years (range, 70-78) in the control arm.26

Chari said the MAIA findings, published in Lancet Oncology in October 2021, represent a noteworthy advancement for older patients with MM. The problem with older patients in particular is that they dont always get to the umpteenth line of therapy, Chari said. Sometimes you get 1 or 2 shots [at effective treatment].

A median PFS not reached with nearly 5 years of follow-up for those who received daratumumab in MAIA is amazing, considering this is without ASCT in an older MM population, Chari said.

More recently, the MM community has been dissecting findings from the phase 3 DETERMINATION trial (NCT01208662), whose results were simultaneously presented at 2022 ASCO and published in the NEJM.27,28 This trial explored the question of whether ASCT improves outcomes for patients with newly diagnosed MM who are eligible for transplant and receive triple induction therapy with lenalidomide, bortezomib, and dexamethasone (RVd).

Investigators randomized patients (n = 357) to treatment with RVd for 2 to 3 cycles followed by RVd for 4 to 8 cycles or RVd plus melphalan and ASCT (n = 365) followed by RVd for 4 to 5 cycles. All patients were eligible for lenalidomide maintenance therapy until progression. The median age of patients was 57 years (range, 25-66) in the RVd-alone arm and 55 years (range, 30-65) in the RVd plus ASCT arm.

After a median follow-up of 76.0 months, the median PFS was significantly shorter in patients who received RVd alone at 46.2 months (95% CI, 38.1-53.7) than among participants treated with RVd plus ASCT at 67.5 months (95% CI, 58.6-NR). The risk of disease progression or death was 53% higher among those who did not receive ASCT (HR, 1.53; 95% CI, 1.23-1.91; P < .0001). However, 5-year OS, a key secondary end point, was similar between the 2 arms (HR, 1.10; adjusted 95% CI, 0.73-1.65; P = .99).

Chari expects the debate about the clinical utility of ASCT to continue for the foreseeable future. On the one hand, transplant is integral in the improvement of outcomes in patients with myeloma, especially globally, because its a relatively cost-effective standard, he said.

The benefit, however, favors younger patients, according to data from the National Cancer Institutes Surveillance, Epidemiology, and End Results (SEER) Program. If you look at Medicare SEER data, the patients whose survival has improved most are not the frail, older elderly, but the younger patients [who can tolerate transplant], Chari said.

Does transplant still have a role with all these amazing therapies? Chari continued. Many people want OS as a primary end point, but its nearly impossible to do that in large, randomized studies, given the innumerable and also ever-increasing number of salvage therapy options as well as the prohibitively long follow-up time that would be required for a disease where the median OS for some patients with MM is more than 10 years. So, theres this constant back-and-forth about the role of transplant, especially in newly diagnosed patients.

For his part, Chari remains dedicated to exploring ways to improve outcomes for patients with MM through better use of existing regimens and the development of novel therapies. Whats more gratifying than seeing people who are alive and in complete remission for years beyond where they thought they would be? You have patients who are bedridden or bed-bound and within a few months are back to their pre-myeloma quality of life because the therapeutics are so effective, and symptoms of myeloma can be so rapidly reversed. Its really gratifying.

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More Novel Therapies Are on the Horizon in Multiple Myeloma - OncLive

Over the last six decades, hematologists have made momentous strides in research, including the development of new, cutting-edge therapeutic technologies that have paved the way for remarkable advances in the treatment of many serious blood diseases,

NEW YORK (PRWEB) June 30, 2022

Mediaplanet today announces the launch of the print and digital campaign, Blood Health. With the increasing prevalence of people suffering from blood cancers and disorders, its never been more important to educate the public about the human bodys most valuable player and current challenges that patients, providers, and researchers face. This campaign will inspire action and encourage readers to educate themselves by engaging with compelling stories of patients; announcements of scientific and medical breakthroughs; and new therapeutic and diagnostic approaches to blood cancers, sickle cell disease, hemophilia and other blood and bleeding disorders.

Over the last six decades, hematologists have made momentous strides in research, including the development of new, cutting-edge therapeutic technologies that have paved the way for remarkable advances in the treatment of many serious blood diseases, writes Jane N. Winter, M.D., president of the American Society of Hematology (ASH) in an introduction article in the publication.

Some of these cutting-edge therapeutic technologies include advancements in CAR T-cell therapy, sickle cell trait testing, and integrated, global master clinical trials for childrens blood cancers. CAR T-cell therapy involves taking a persons immune cells from their blood, reprogramming the immune cells to target the patients blood cancer, and then returning these new guided missiles to the patient as a transfusion, writes Winter. Momentum in research for CAR T-cell therapies for children is growing as well, since it has the potential to cause fewer side effects than chemotherapy, which is one of the key challenges for pediatric blood cancers current standard of care.

This year marked 10 years since pioneer pediatric patient Emily Whitehead was successfully treated for her acute lymphoblastic leukemia (ALL) with CAR T-cell therapy at the Childrens Hospital of Philadelphia. The therapy saved Emilys life, and she is still cancer-free today. Readers can find her story, among many others, within the Blood Health campaign.

The print component of Blood Health is distributed within todays edition of USA Today in New York, Boston, Philadelphia, Chicago, Carolinas, Denver, Dallas, Houston, Kansas City, Nashville, Washington D.C., Baltimore, Pittsburgh, Cleveland, Detroit, Minneapolis, Cincinnati, St. Louis, Los Angeles, and surrounding areas with a circulation of approximately 150,000 copies and an estimated readership of 450,000. The digital component is distributed nationally through a vast social media strategy and across a network of top news sites and partner outlets. To explore the digital version of the campaign, visit: futureofpersonalhealth.com

This campaign was made possible with the support of the American Red Cross, American Society for Clinical Pathology, American Society of Hematology, AstraZeneca, Be the Match, the Emily Whitehead Foundation, Genentech, Jerry Rice, the Leukemia & Lymphoma Society, the MPN Research Foundation, TC BioPharm, and more.

About MediaplanetMediaplanet specializes in the creation of content marketing campaigns covering a variety of industries. We tell meaningful stories that educate our audience and position our clients as solution providers. Our unique ability to pair the right leaders with the right readers, through the right platforms, has made Mediaplanet a global content marketing powerhouse. Our award-winning stories have won the hearts of countless readers while serving as a valuable platform for brands and their missions. Just call us storytellers with a purpose. Please visit http://www.mediaplanet.com for more on who we are and what we do.

Contact:Shannon Ruggiero646-755-7962Shannon.ruggiero@mediaplanet.com

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Smartphones, superglue, electric cars, video chat. When does the wonder of a new technology wear off? When you get so used to its presence that you dont think of it anymore? When something newer and better comes along? When you forget how things were before?

Whatever the answer, the gene-editing technology CRISPR has not reached that point yet. Ten years after Jennifer Doudna and Emmanuelle Charpentier first introduced their discovery of CRISPR, it has remained at the center of ambitious scientific projects and complicated ethical discussions. It continues to create new avenues for exploration and reinvigorate old studies. Biochemists use it, and so do other scientists: entomologists, cardiologists, oncologists, zoologists, botanists.

Cathie Martin, a botanist at the John Innes Centre in Norwich, England, and Charles Xavier, founder of the X-Men superhero team: They both love mutants.

But while Professor X has an affinity for superpowered human mutants, Dr. Martin is partial to the red and juicy type. We always craved mutants, because that allowed us to understand functionality, Dr. Martin said of her research, which focuses on plant genomes in the hopes of finding ways to make foods especially tomatoes in her case healthier, more robust and longer lasting.

When CRISPR-Cas9 came along, one of Dr. Martins colleagues offered to make her a mutant tomato as a gift. She was somewhat skeptical, but, she told him, I would quite like a tomato that produces no chlorogenic acid, a substance thought to have health benefits; tomatoes without it had not been found before. Dr. Martin wanted to remove what she believed was the key gene sequence and see what happened. Soon a tomato without chlorogenic acid was in her lab.

Instead of looking for mutants, it was now possible to create them. Getting those mutants, it was so efficient, and it was so wonderful, because it gave us confirmation of all these hypotheses we had, Dr. Martin said.

Most recently, researchers at Dr. Martins lab used CRISPR to create a tomato plant that can accumulate vitamin D when exposed to sunlight. Just one gram of the leaves contained 60 times the recommended daily value for adults.

Dr. Martin explained that CRISPR could be used across a broad spectrum of food modifications. It could potentially remove allergens from nuts and create plants that use water more efficiently.

I dont claim that what we did with vitamin D will solve any food insecurity problems, Dr. Martin said, but its just a good example. People like to have something that they can hang on to, and this is there. Its not a promise.

Infectious Disease

Christian Happi, a biologist who directs the African Centre of Excellence for Genomics of Infectious Diseases in Nigeria, has spent his career developing methods to detect and contain the spread of infectious diseases that spread to humans from animals. Many of the existing ways to do so are costly and inaccurate.

For instance, in order to perform a polymerase chain reaction, or PCR, test, you need to go extract RNA, have a machine thats $60,000 and hire someone who is specially trained, Dr. Happi said. Its both costly and logistically implausible to take this kind of testing to most remote villages.

Recently, Dr. Happi and his collaborators used CRISPR-Cas13a technology (a close relative of CRISPR-Cas9) to detect diseases in the body by targeting genetic sequences associated with pathogens. They were able to sequence the SARS-CoV-2 virus within a couple of weeks of the pandemic arriving in Nigeria and develop a test that required no on-site equipment or trained technicians just a tube for spit.

If youre talking about the future of pandemic preparedness, thats what youre talking about, Dr. Happi said. Id want my grandmother to use this in her village.

The CRISPR-based diagnostic test functions well in the heat, is quite easy to use and costs one-tenth of a standard PCR test. Still, Dr. Happis lab is continually assessing the accuracy of the technology and trying to persuade leaders in the African public health systems to embrace it.

He called their proposal one that is cheaper, faster, that doesnt require equipment and can be pushed into the remotest corners of the continent. This would allow Africa to occupy what I call its natural space.

Hereditary Illness

In the beginning there was zinc finger nuclease.

That was the gene-editing tool that Gang Bao, a biochemical engineer at Rice University, first used to try to treat sickle cell disease, an inherited disorder marked by misshapen red blood cells. It took Dr. Baos lab more than two years of development, and then zinc finger nuclease would successfully cut the sickle cell sequence only around 10 percent of the time.

Another technique took another two years and was only slightly more effective. And then, in 2013, soon after CRISPR was used to successfully edit genes in living cells, Dr. Baos team changed tack again.

From the beginning to having some initial results, CRISPR took us like a month, Dr. Bao said. The method successfully cut the target sequence around 60 percent of the time. It was easier to make and more effective. It was just amazing, he said.

The next challenge was to determine the side effects of the process. That is, how did CRISPR affect genes that werent being purposefully targeted? After a series of experiments in animals, Dr. Bao was convinced that the method would work for humans. In 2020 the Food and Drug Administration approved a clinical trial, led by Dr. Matthew Porteus and his lab at Stanford University, that is ongoing. And there is also hope that with CRISPRs versatility, it might be used to treat other hereditary diseases. At the same time, other treatments that have not relied on gene editing have had success for sickle cell.

Dr. Bao and his lab are still trying to determine all the secondary and tertiary effects of using CRISPR. But Dr. Bao is optimistic that a safe and effective gene-editing treatment for sickle cell will be available soon. How soon? I think another three to five years, he said, smiling.

Cardiology

It is hard to change someones heart. And thats not just because we are often stubborn and stuck in our ways. The heart generates new cells at a much slower rate than many other organs. Treatments that are effective in other parts of the human anatomy are much more challenging with the heart.

It is also hard to know what is in someones heart. Even when you sequence an entire genome, there are often a number of segments that remain mysterious to scientists and doctors (called variants of uncertain significance). A patient might have a heart condition, but theres no way to tie it definitively back to their genes. You are stuck, said Dr. Joseph Wu, director of the Stanford Cardiovascular Institute. So traditionally we would just wait and tell the patient we dont know whats going on.

But over the past couple of years, Dr. Wu has been using CRISPR to see what kind of effects the presence and absence of these befuddling sequences have on heart cells, simulated in his lab with induced pluripotent stem cells generated from the blood. By cutting out particular genes and observing the effects, Dr. Wu and his collaborators have been able to draw links between the DNA of individual patients and heart disease.

It will be a long time before these diseases can be treated with CRISPR, but diagnosis is a first step. I think this is going to have a big impact in terms of personalized medicine, said Dr. Wu, who mentioned that he found at least three variants of uncertain significance when he got his own genome sequenced. What do these variants mean for me?

Sorghum is used in bread, alcohol and cereal all over the world. But it hasnt been commercially engineered to the same degree as wheat or corn, and, when processed, it often isnt as tasty.

Karen Massel, a biotechnologist at the University of Queensland in Australia, saw quite a bit of room for improvement when she first started studying the plant in 2015. And because millions of people eat sorghum worldwide, if you make a small change you can have a huge impact, she said.

She and her colleagues have used CRISPR to try to make sorghum frost tolerant, to make it heat tolerant, to lengthen its growth period, to change its root structure we use gene editing across the board, she said.

Not only could this lead to more delicious and healthier cereal, but it could also make the plants more resistant to the changing climate, she said. But it is still no small task to accurately edit the genomes of crops with CRISPR.

Half the genes that we knock out, we just have no idea what they do, Dr. Massel said. The second we try to get in there and play God, we realize were a bit out of our depth. But, using CRISPR combined with more traditional breeding techniques, Dr. Massel is optimistic, despite being a self-described pessimist. And she hopes that further advances will lead to commercializing gene-edited foods, making them more accessible and more acceptable.

In 2012, a 6-year-old girl was suffering from acute lymphoblastic leukemia. Chemotherapy had been unsuccessful, and the case was too advanced for a bone-marrow transplant. There didnt seem to be any other options, and the girls physicians told her parents to go back home.

Instead, they went to the Childrens Hospital of Philadelphia, where doctors used an experimental treatment called chimeric antigen receptor (CAR) T-cell therapy to turn the girls white blood cells against the cancer. Ten years later, the girl is cancer free.

Since then, Dr. Carl June, a medical professor at the University of Pennsylvania who helped develop CAR T-cell therapy, and his collaborators, including Dr. Ed Stadtmauer, a hematologist-oncologist at Penn Medicine, have been working to improve it. That includes using CRISPR, which is the simplest and most accurate tool to edit T-cells outside the body. Dr. Stadtmauer, who specializes in dealing with various types of blood and lymph system cancers, said that the last decade or so has just seen a revolution of treatment of these diseases; its been rewarding and exciting.

Over the past couple of years, Dr. Stadtmauer helped run a clinical trial in which T-cells that underwent significant CRISPR editing were inserted into patients with treatment-resistant cancers. The results were promising.

Nine months into the trial the edited T-cells had not been rejected by the patients immune systems and were still present in the blood. The real benefit is that scientists now know that CRISPR-aided treatments are possible.

Even though its really sort of science fiction-y biochemistry and science, the reality is that the field has moved tremendously, Dr. Stadtmauer said. He added that he was less excited by the science than how useful CRISPR had become. Every day I see maybe 15 patients who need me, he said. Thats what motivates me.

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The Many Uses of CRISPR: Scientists Tell All - The New York Times

The global Mobile Cardiac Telemetry Systems Market research report gives a comprehensive analysis of market size, market trends, and market growth prospects. This report also provides extensive information on the technology expenditure for the forecast period, which gives a unique view of the global Mobile Cardiac Telemetry Systems Market across multiple segments. The global Mobile Cardiac Telemetry Systems market report also helps consumers recognize market opportunities and challenges. This report includes the most current Mobile Cardiac Telemetry Systems market forecast research over the expected period. The global Mobile Cardiac Telemetry Systems market report provides extensive information on technological developments and market growth prospects based on regional landscapes. The Mobile Cardiac Telemetry Systems Market Report is also designed using advanced methodologies and includes a detailed analysis of the Mobile Cardiac Telemetry Systems marketplaces sales and providers.

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Leading players of Mobile Cardiac Telemetry Systems Market including:

BioTelemetry Inc., Biotricity Inc., ScottCare, Welch Allyn, Medicomp Inc., Applied Cardiac Systems Inc., Preventice Solutions, iRhythm Technologies, Inc., Telerhythmics LLC

In addition to this, the report by Adroit Market Research has been designed through the complete surveys, primary research interviews, as well as observations, and secondary research. Moreover, the Mobile Cardiac Telemetry Systems market report introduced the market through several factors such as classifications, definitions, market overview, product specifications, cost structures, manufacturing processes, raw materials, and applications. Moreover, the study offers a complete analysis of the market size, segmentation, and market share. Additionally, the Mobile Cardiac Telemetry Systems report contains market dynamics such as market restraints, growth drivers, opportunities, service providers, stakeholders, investors, key market players, profile assessment, and challenges of the global market.

Further to this, the Mobile Cardiac Telemetry Systems market report by Adroit Market Research holistically touches upon well-orchestrated data sources and insightful factors about multiple manufacturers and market honchos working extensively in the Mobile Cardiac Telemetry Systems market. This report also entails supply chain nuances, financial data analysis, products & services records, core developments, as well as elaborate description on acquisitions & mergers, current & future growth probabilities trends, as well as advances, inclusive of technological sophistication, that carefully craft market players footprint in the global Mobile Cardiac Telemetry Systems market.

The market segmentation information in the research report is a combination of primary and secondary research methods. The report includes both a quantitative as well as qualitative analysis of target market evaluations over the forecasted period to show the economic potential of the global target market. The global Mobile Cardiac Telemetry Systems industry is covered in this report, which includes current and prospective market trends. This will help to determine the potential market investment for the Mobile Cardiac Telemetry Systems industry. The research report also includes an industry analysis and forecasts for the registered forecast period. In addition, the Mobile Cardiac Telemetry Systems Market Study provides comprehensive data about the opportunities, key driver, and restraining factor with the contact analysis.

Mobile Cardiac Telemetry Systems market Segmentation by Type:

NA

Mobile Cardiac Telemetry Systems market Segmentation by Application:

By Application (Lead-Based, Patch-Based) End Use (Hospitals, Cardiac Centers, Others)

The Mobile Cardiac Telemetry Systems Market Reports Key Highlights:

The Mobile Cardiac Telemetry Systems global market report provides a comprehensive qualitative and quantitative analysis that will provide insight into the industry. This Mobile Cardiac Telemetry Systems market insight includes data from influential participants such as marketers, business experts and investors. The Mobile Cardiac Telemetry Systems market reports objective is to provide a comprehensive perspective from all stakeholders for young marketers and entrepreneurs. Trends and drivers are discussed in the Mobile Cardiac Telemetry Systems Market Report The global Mobile Cardiac Telemetry Systems market report provides an overview of the global competitive environment. It provides information about the market, its share and revenue. The Mobile Cardiac Telemetry Systems Market Report identifies the major growth regions, with Asia Pacific leading during the forecast period.

Table of Content:

Chapter 1. Research ObjectiveChapter 2. Executive SummaryChapter 3. Strategic AnalysisChapter 4. Mobile Cardiac Telemetry Systems Market DynamicsChapter 5. Segmentation & StatisticsChapter 6. Market Use case studiesChapter 7. KOL RecommendationsChapter 8. Investment LandscapeChapter 9. Competitive IntelligenceChapter 10. Company ProfilesChapter 11. AppendixContinued

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Stem Cell Therapy Market Overview by Advance Technology, Future Outlook 2028 Anterogen Co., Ltd., MEDIPOST Co., Ltd., Osiris Therapeutics, Inc. and...

New Jersey, United States This Cell Therapy And Tissue Engineering Market research examines the state and future prospects of the Cell Therapy And Tissue Engineering market from the perspectives of competitors, regions, products, and end Applications/industries. The Worldwide Cell Therapy And Tissue Engineering market is segmented by product and Application/end industries in this analysis, which also analyses the different players in the global and key regions.

The analysis for the Cell Therapy And Tissue Engineering market is included in this report in its entirety. The in-depth secondary research, primary interviews, and internal expert reviews went into the Cell Therapy And Tissue Engineering reports market estimates. These market estimates were taken into account by researching the effects of different social, political, and economic aspects, as well as the present market dynamics, on the growth of the Cell Therapy And Tissue Engineering market.

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Key Players Mentioned in the Cell Therapy And Tissue Engineering Market Research Report:

BioCardia Inc., Betalin Therapeutics Ltd., MEDIPOST Co. Ltd., MaxCyte Inc., BioReliance Corporation

The Porters Five Forces analysis, which explains the five forces: customers bargaining power, distributors bargaining power, the threat of substitute products, and degree of competition in the Cell Therapy And Tissue Engineering Market, is included in the report along with the market overview, which includes the market dynamics. It describes the different players who make up the market ecosystem, including system integrators, middlemen, and end-users. The competitive environment of the Cell Therapy And Tissue Engineering marketis another major topic of the report. For enhanced decision-making, the research also provides in-depth details regarding the COVID-19 scenario and its influence on the market.

Cell Therapy And Tissue EngineeringMarket Segmentation:

Cell Therapy and Tissue Engineering Market, By Type

Cell Therapy Tissue Engineering Others

Cell Therapy and Tissue Engineering Market, By Application

Cell Defects Genetic Diseases Tissue Malignancy Neurodegenerative Disease Others

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Cell Therapy And Tissue Engineering Market Report Scope

Key questions answered in the report:

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4. What are the drivers and restraints of the Cell Therapy And Tissue Engineering market?

5. Which regional market will show the highest growth?

6. What will be the CAGR and size of the Cell Therapy And Tissue Engineering market throughout the forecast period?

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Cell Therapy And Tissue Engineering Market Size, Scope and Forecast | BioCardia Inc., Betalin Therapeutics Ltd., MEDIPOST Co. Ltd., MaxCyte Inc.,...

Leukapheresis is a laboratory procedure to separate white blood cells from a sample of blood which can occur with chronic lymphocytic leukemia (CLL). Leukapheresis is a good treatment option for patients who have very high white blood cell count or to obtain white blood cells for later transplant to treat decrease in white blood cells during chemotherapy. It also helps fightingprostate cancerand other form of cancers.

Earlier, leukapheresis was designed for palliative treatment for leukemia with only limited efficacy. But later as this technique was used for treatment modality for hyperleukocytic acute myeloid leukemia, and now it is also used to collect granulocytes to transfuse into neutropenic patients with bacterial or fungal infections, the growth of adoption of this technique is expected to increase.

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How Leukapheresis is important for CAR or Adaptive Cell Therapy Manufacturing?

Chimeric antigen receptor(CAR) T cell therapymay be offered to people when their cancer comes back. Leukapheresis is the first step in this treatment. Research is going on for this treatment. Researchers want to collect T cells from people who may become eligible for a CAR T-cell study in the future. Leukapheresis is an essential step for CAR therapy or other adaptive cellular therapy products. The procedure is required to develop a streamlined process where patients have to undergo apheresis for development of a CAR cell or other adoptive cell therapy product. Once patient meets the eligibility conditions for one of the NCI CAR clinical cell therapy trials, they will undergo leukapheresis estimated by recipient weight and target cell harvest dose in Department of Transfusion Medicine (DTM). Therefore, need for leukapheresis in CAR therapy is a major factor for the boost in its adoption.

Prevalence of Leukemia Is projected to Boost the Treatment

Cases of leukemia have been increasing significantly. According to American Cancer Society, in 2020, there were 60,530 new cases of all kinds of leukemia which witnessed 23,100 deaths. Acute myeloid leukemia being the most prevalent accounted for 19,940 cases in US. The cancer burden in Europe is estimated to have risen to 2.7 million new cases and 1.3 million deaths in Europe as per the Joint Research Centre in European Union. Therefore, it is expected that the rising cases of leukemia and cancer may pave a way of growth for leukapheresis.

Side Effects of Leukapheresis may restrain the Growth

Like any other medical treatment, leukapheresis also have some risks and side effects which may decline its growth. Hypocalcemia, anemia, local or systematic infections may occur among the patients. Hypocalcemia is an irregular drop of calcium from the body when white blood cells are extracted. The lack of calcium in the body may cause numbness, muscle spasms, etc. Anaemia can also occur due to low count of red blood cells or platelets which may happen during the process. Although, in most of the cases, the effect of anaemia is mild. Local or systematic infections, redness and bruising at the vein puncture may also happen. All these side effects can cause the patients to decline the treatment if it is not very important.

Alternative Technologies may Cause Hindrance in Market Boost

As per the research article by pnas.org, clinical leukapheresis can concentrate mononuclear cells from almost the entire blood volume, but such large numbers and cell concentrations are incompatible with such technology. Therefore, an ultra-high throughput microfluidic chip, CTC-iChip rapidly sorts through an entire leukapheresis product of over 6 billion nucleated cells, increasing the CTC isolation capacity by two orders of magnitude. CTC based liquid biopsies provide cancer diagnostics, treatment selection, and response monitoring. Therefore, these factors may cause restrain to the leukapheresis market during the forecast period.

Competitive Landscape

Prominent players in the market include Caltag Medsystems Limited (UK), BioIVT (US), Asahi Kasei Medical Co. Ltd. (Japan), Terumo BCT, Inc. (US), MEDICA S.p.A (Italy), StemExpress, LLC (US), AllCells, LLC (US), Charles River Laboratories (US), Lonza Group AG (Switzerland), Macopharma SA (France), PuriBlood Medical Co. Ltd. (Taiwan), Miltenyi Biotec (Germany), ZenBio (US), Discovery Life Sciences (US), Intelligent Tissue Group (US), Haemonetics Corporation (US), etc.

AllCells, LLC, a leading provider of prmary cells supporting biomedical research announced in September 2020 about the addition of Cyropreserved Leukppaks to their extensive product portfolio. The primary cells are of high quality due to on-site facilities. These cells are cryopreserved using GE Healthcares GMP-compliant VIA freeze quad controlled rate freezer which is capable of consistently high post recovery and viability. Cryopreserved cells are flexible and thus, experiments on them can be done as per the schedule without waiting on delivery of fresh cells.

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Novartis in October 2020 announced the authorization from Japans Ministry of Health, labour and Welfare for a site for manufacturing of commercial CAR-T cell therapy for patients in Japan. The commercial is the first to get approval in Asia. The companys aim is to being treatment closer to patients. Kymriah is the first ever FDA approved CAR-T cell therapy, and the first ever CAR-T to be approved in two distinct indications. Kymriah is currently approved for the treatment of pediatric and young adults of acute lymhoblastic leukemia.

Haemonetics Corporation, Boston based company have made an agreement to sell its Fajardo, Puerto Rico manufacturing operations to filter GVS. The company will retain all intellectual property rights to its proprietary blood filters which are currently manufactured at their Fajardo facility. The company has offered exclusive rights to manufacture and supply the blood filters. Thus, Fajardo facility will become GVS employees.

Regional Outlook

The major contributors towards the growth of leukapheresis include, increasing incidences of leukemia, growing demand for leukapheresis procedures, research applications, growing demand for leukopaks for research applications, etc.

North America is expected to hold the dominating share owing to rise in geriatric population, increasing cases of leukemia. According to Leukemia and Lymphoma Society, approximately every 3 minutes, one person is diagnosed with leukemia in US and in 9 minutes someone dies. According to American Cancer Society, 60,530 new cases of all kinds of leukemia were diagnosed in 2020 which witnessed 23,100 deaths. Acute myeloid leukemia is the most prevalent and accounted for 19,940 cases in US. North Ameirca is one of the leading countries to record deaths due to cancer and leukemia. Furthermore, research and development and new technological advancements are another factor for the market growth.

European region is also expected to have a significant growth during the forecast period 2021-2031. The acute promyelocytic leukemia which is a rare disease also accounted for 2.5 per million person in Italy, 1.39 per million in Spain, 0.54 per million in Great Britain, 0.4 per million in France according to frontiers.org. Growing AML cases are also the concerning topic, it is expected that the cases will increase by 17% by 2027, therefore increasing the growth of its treatment options. All these factors offer a contribution towards the growth of increasing leukapheresis market.

In Asia, China and Pakistan account for 36.2% and 43.5% in AML cases. Increasing medical infrastructure, emerging technologies, increasing trials and investments due to rising geriatric population, and cases of leukemia is paving the way for market growth. As per Globocan, over 20,000 new cases of childhood blood cancer are detected in India of which almost 15,000 cases are of leukemia. China is expected to witness increase over 14,000 cases of acute lymphocytic leukemia by 2029, therefore, rising incidences are projected to grow the adoption of leukapheresis.

Segmentation

Based on Type

Based on Application

Based on End user

Based on Region

Key Questions Answered

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Leukapheresis Market Dynamic Demand and Drives with Forecast to 2031 - Digital Journal

Researchers from Rice University and the University of Texas MD Anderson Cancer Center have discovered potential new drugs that work in concert with other drugs to deliver a deadly one-two punch to leukemia.

The potential drugs are still years away from being tested in cancer patients, but arecently published studyin the journalLeukemiahighlights their promise and the innovative methods that led to their discovery.

In previous studies, the research groups of Rice biochemistNatasha Kirienkoand MD Anderson physician-scientistMarina Konoplevascreened some 45,000 small-molecule compounds to find a few that targeted mitochondria. In the new study, they chose eight of the most promising compounds, identified between five and 30 closely related analogs for each and conducted tens of thousands of tests to systematically determine how toxic each analog was toleukemiacells, both when administered individually or in combination with existing chemotherapy drugs likedoxorubicin.

One of the big challenges was to establish optimal conditions and doses for testing on both cancer cells and healthy cells, said study lead authorSvetlana Panina, a researcher at the University of Texas at Austin who conducted the research during her postdoctoral studies at Rice. The results from our previously published cytotoxicity assay were helpful, but very little is known about these small-molecule compounds. None of them had been thoroughly described in other studies, and we had to essentially start from scratch to determine how much to use, what they do in cells, everything. All the doses and treatment conditions had to be adjusted by multiple preliminary experiments.

In prior work, Kirienkos lab had shown the eight compounds targeted energy-producing machinery inside cells called mitochondria. Dozens to thousands of mitochondria are at work every minute in every living cell, and like all machines, they wear out with use. The eight compounds inducemitophagy, the housekeeping routine cells use to decommission and recycle mitochondria that are past their prime.

During times of extreme stress, cells can temporarily forgo mitophagy to get an emergency energy boost. Cancer is notorious for hijacking these sorts of programs to fuel pathological growth. For example, previous research has shown leukemia cells have far more damaged mitochondria than healthy cells and are also more sensitive to mitochondrial damage than healthy cells.

Kirienko and Konopleva reasoned that mitophagy-inducing drugs might weaken leukemia cells and make them more susceptible to chemotherapy.

We hypothesized that if they activate mitophagy, they may be particularly toxic to leukemia cells, said Kirienko, the corresponding author of the new study. And indeed, we found that six of the eight small-molecule compounds were deadly to leukemia cells. We then wanted to study them more in depth. So we looked at closely related molecules, and we looked at combinations.

When two or more drugs are given in combination, researchers can also administer them individually and compare the effectiveness of each regimen.

There is a number called synergy coefficient that quantifies interactions between drugs, Kirienko said. If the coefficient is negative, the drugs are antagonistic and work against one another. Zero means no effect, and positive numbers indicate positive interactions. Anything above 10 is considered synergistic.

For example, one currently prescribed drug combination for leukemia doxorubicin and cytarabine has a synergy coefficient of 13, Kirienko said. The teams experiments showed several mitophagy-inducing compounds were significantly more synergistic with doxorubicin. The most synergistic, a compound called PS127B, had a coefficient of 29.

The point of synergy is that there are concentrations, or dosages, where a single drug doesn't kill, Kirienko said. There is no death of healthy cells or cancer cells. But administering those same concentrations in combination can kill a considerable amount of cancer cells and still not affect healthy cells.

The team started by testing the toxicity of its mitophagy-inducing compounds and combinations against acute myeloid leukemia (AML) cells, the most commonly diagnosed form of the disease. They then tested the six most effective AML-killing compounds against other forms of leukemia and found five were also effective at killing acute lymphoblastic leukemia (ALL) cells and chronic myelogenous leukemia (CML) cells. Control studies found all the mitophagy-inducing drugs caused far less harm to healthy cells.

In their final experiments, the researchers tested one of the most effective mitochondria-targeting compounds, PS127E, using a cutting-edge technique called apatient-derived xenograft(PDX) model. In PDX, also referred to as a mouse clinical trial, mice are implanted with cancer cells from a leukemia patient. Once the cells grow, the mouse is exposed to a drug or combination of drugs as a closer-than-cells test of the treatments effect. PDX tests on one compound, PS127E, showed it was effective at killing AML cells in mice.

Although this is very promising, were still some distance from having a new treatment we can use in the clinic, Kirienko said. We still have a lot to discover. For example, we need to better understand how the drugs work in cells. We need to refine the dose we think would be best, and perhaps most importantly, we need to test on a wide variety of AML cancers. AML has a lot of variations, and we need to know which patients are most likely to benefit from this treatment and which are not. Only after weve done that work, which may take a few years, would we be able to start testing in humans.

Reference:Panina SB, Pei J, Baran N, et al. Novel mitochondria-targeting compounds selectively kill human leukemia cells. Leukemia. 2022:1-13. doi: 10.1038/s41375-022-01614-0

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