Category Archives: Cell Therapy

NATICK, Mass.--(BUSINESS WIRE)--Aleta Biotherapeutics, a privately held immuno-oncology company focused on transforming cellular therapeutics to allow a broad spectrum of cancer indications to be targeted, today announced that Paul Rennert, Ph.D., President and Chief Scientific Officer, will deliver an oral presentation at the 61st ASH Annual Meeting & Exposition, December 7 10, 2019, Orange County Convention Center, Orlando, FL.

The 61st ASH Annual Meeting will be an important opportunity to present mechanistic studies and extensive in vitro and in vivo results from our novel CD19-anti-CD20 bridging protein technology, said Paul Rennert, Ph.D., President and Chief Scientific Officer of Aleta Biotherapeutics. We look forward to advancing the development of this novel therapy based on the data we will be presenting on December 7.

Oral Presentation DetailsTitle: 252 A Novel CD19-Anti-CD20 Bridging Protein Prevents and Reverses CD19-Negative Relapse from CAR19 T Cell Treatment in vivo Session Name: 703. Adoptive Immunotherapy: Mechanisms and New Approaches: MechanismsPresenter: Paul Rennert, Ph.D.Session Date: Saturday, December 7, 2019Session Time: 2:00 PM - 3:30 PMPresentation Time: 3:15 PMLocation: Orange County Convention Center, Valencia A (W415A)

About Aleta BiotherapeuticsAleta Biotherapeutics is an immuno-oncology company focused on transforming cellular therapeutics to allow a broad spectrum of cancer indications to be targeted, including currently intractable solid tumors. The company was founded by Paul Rennert and Roy Lobb, who bring extensive scientific and leadership experience in immunology, oncology and drug development to this new enterprise. Aleta has created a unique portfolio of multi-antigen targeting solutions for cell therapy, designed to address the critical issues of CAR-T persistence, tumor antigen loss leading to patient relapse, and tumor antigen heterogeneity. For more information, visit http://www.aletabio.com.

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Aleta Biotherapeutics Announces Oral Presentation at the 61st American Society of Hematology Annual Meeting (ASH) - Business Wire

There are a number of policy changes that can drive change within the implementation of chimeric antigen receptor T-cell therapy, but further innovation is warranted to improve access, said John Sweetenham, MD, professor in the Department of Internal Medicine at UT Southwestern Medical Center and the Associate Director for Clinical Affairs at UTSWs Harold C. Simmons Comprehensive Cancer Center.

There are a number of policy changes that can drive change within the implementation of chimeric antigen receptor (CAR) T-cell therapy, but further innovation is warranted to improve access, said John Sweetenham, MD, professor in the Department of Internal Medicine at UT Southwestern Medical Center and the Associate Director for Clinical Affairs at UTSWs Harold C. Simmons Comprehensive Cancer Center.

Transcript

What policies have helped or hindered the implementation of CAR T-cell therapy?

So, I think just recently, there are a number of policy changes, for which overall, I think going to prove to be helpful. Probably not helpful enough, but I think they certainly caused some improvements. The first one of those is that CMS has recognized that it will fund up to a certain extent CAR T-cell therapy for the license indications, which at the moment are predominantly in B-cell malignancy, but it also has recognized that it is essential to cover the cost of CAR T for any other indications, which are listed in authoritative compendia of which the National Comprehensive Cancer Network, 1, is probably the most respected and best referenced. So, I think those are positive things. The other positive outcome of the recent change in the national coverage determination is that the amount of reimbursement is increased from 50% of cost to 65% of cost.

So, those things are all good and I think they help us provide this treatment to more of our patients. The problem and the hindrance is that it really still falls a very long way short of the true cost of the treatment. The cells themselves cost somewhere between $370,000 to $450,000, and at the moment with the current reimbursement models, the best that we can do if patients are treated as inpatients is to recover about 80% of that by the time we factor in the new technology, add-on payments as well. So, for most big centers, who are performing inpatient CAR T-cell treatments at the moment, they stand to lose at a minimum probably about $100,000 per patient and that is purely on the price of the CAR T cells. So, that doesn't really factor in what will happen if the patient gets toxicity, needs to be admitted to the [intensive care unit], and so on.

So, I think that what I would say is that the recent policy changes have been definitely a step in the right direction. I think that we still have quite a long way to go, but it undoubtedly will slowly improve the access. The other thing that needs to happen on our end is as oncologists, we need to strengthen the evidence base because I think that's the other thing that's lacking. Then perhaps the final thing I'd say, and I'm definitely in a minority with this opinion, but at some level when Medicarewhen CMS decided to increase the new technology add-on payments, under the new policy change for coverage, it was anticipated that there would be a provision in there that it will be coverage with evidence development so that we would be required to prospectively collect evidence for the efficacy. That requirement was dropped, and I think the reasons for that were good because the feeling is that will accelerate access. On the other hand, I think the downside of that is that it was an opportunity for us to get more of the evidence base that we really need.

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Dr John Sweetenham Outlines the Effect of Current Policies on the Implementation of CAR T Therapy - AJMC.com Managed Markets Network

DetailsCategory: DNA RNA and CellsPublished on Monday, 02 December 2019 15:08Hits: 21

Application Being Evaluated Under Accelerated Assessment

BOSTON, MA, USA and LONDON, UK I December 02, 2019 I Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced that the European Medicines Agency (EMA) has validated the companys Marketing Authorization Application (MAA) for OTL-200, an ex vivo, autologous, hematopoietic stem cell-based gene therapy that has been developed in partnership with the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy, for the treatment of metachromatic leukodystrophy (MLD). Validation of the MAA confirms that the submission is sufficiently complete to begin the formal review process.

We are pleased that OTL-200 for the treatment of MLD is now under review with the EMA, bringing us another step closer to potentially making an approved gene therapy treatment a reality for children and families affected by this devastating and rapidly progressing disease, said Mark Rothera, president and chief executive officer of Orchard Therapeutics. We are committed to working with the EMA as they evaluate our application. Due to the nature of the disease and the urgency to treat those affected by MLD, we have also been working diligently in parallel to make OTL-200, if approved, available to patients in the EU as quickly as possible, while continuing our efforts to expand patient access outside the EU.

Orchard previously announced in November 2019 that the EMA had granted accelerated assessment for OTL-200. Accelerated assessment potentially provides a reduced review timeline from 210 to 150 days once the MAA is filed and validated, not counting clock stops when applicants are requested to provide additional information.

About MLD and OTL-200 Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, the gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged and patients with MLD will experience neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures, finding it more and more difficult to move, talk, swallow, eat and see. Currently, there are no effective treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1 OTL-200 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MLD. OTL-200 was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About Orchard Orchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy initiated in 2010.

Orchard currently has offices in the U.K. and the U.S., including London, San Francisco and Boston.

1 Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature. Journal of Child Neurology 2010, DOI: http://doi.org/10.1177/0883073809341669

SOURCE: Orchard Therapeutics

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Orchard Therapeutics Announces the Filing and Validation of Marketing Authorization Application by European Medicines Agency for OTL-200 for the...

Researchers at the University of Cologne have identified Cav2.3 channels as a new mechanism for the development of Parkinson disease which may be the beginning of a new targeted therapy. The findings were published in Nature Communications.1

Parkinson disease is the second most common neurodegenerative disease in which a specific population of dopamine-producing nerve cells in the mid-brain die off selectively. The resulting lack of dopamine then leads to symptoms such as resting tremors, muscle stiffness, and problems executing voluntary movement. It affects more than 6 million people worldwide and is strongly age-dependent.2

It has previously been found that at the cellular level, disturbances in the calcium-dependent signaling pathways are integral to the development of Parkinson disease. Calcium plays a key role in many cellular signaling pathways, and its concentration is therefore regulated very precisely in the cell.2

Deregulation of the calcium balance causes disturbances of the intracellular signaling cascades, which can lead to cell death. Researchers have now shown that excessive calcium influx through specific ion channels, Cav2.3 channels of the so-called R-type, can contribute significantly to the development of Parkinson disease.1

Researchers were able to prevent the death of dopamine-producing nerve cells by genetically switching off the activity of the Cav2.3 channels. The ion channel Cav2.3 has so far not been associated with Parkinson disease. Further research on dopamine-producing neurons, which have developed from human so-called induced pluripotent stem cells, shows that signaling cascades similar to those that cause Parkinson sensitivity in the animal models are also active in human neurons.1

It had been previously hypothesized that another calcium channel, Cav1.3, plays a central role in the development of Parkinson disease. However, a recently completed clinical trial in which Cav1.3 channels were blocked did not show protection against Parkinson disease.2

This new study provides evidence as to why this clinical trial failed to show protective effects and suggests that selective Cav2.3 inhibitors should be tested as a drug to treat Parkinson disease, the authors concluded.2

Reference

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Channel in Nerve Cell May be Key in Unlocking Parkinson Disease Therapy - Pharmacy Times

Around 2 million Americans are affected by traumatic brain injuries each and every year. Traumatic brain injuries cause inflammation and cell death within the brain and those suffering from a head injury quite often experience lifelong memory loss, and even epilepsy. However, a group of researchers from the University of California has developed a new kind of cell therapy that gives hope to these patients.

In the study, published in Nature Communications earlier this month, the UCI team explains how it successfully transplanted embryonic progenitor cells into the brains of mice with traumatic brain injury. What the researchers discovered is the transplanted neurons moved to the injury where new connections were formed with those brain cells that were injured.

Within just one month, they saw promising results. Not only were the mice showing signs of improved memory, but the cell transplants also stopped the mice from developing epilepsy. This was quite a significant result as more than half the mice that werent treated with new interneurons did develop the disease.

While inhibitory neurons are involved in many aspects of memory, that are highly susceptible to dying after a brain injury. While we cannot stop interneurons from dying, it was exciting to find that we can replace them and rebuild their circuits, said Robert Hunt, Ph.D., lead author of the study and assistant professor of anatomy and neurobiology at UCI School of Medicine.

In 2018, Hunt and his colleagues used a similar approach to restore memory in mice. However, back then the therapy was used to improve the memory of newborn mice born with a genetic disorder. But this new study was a huge leap for the researchers. The idea to regrow neurons that die off after a brain injury is something that neuroscientists have been trying to do for a long time, said Hunt.

To test their observations even further, the team used a drug in which to silence the transplanted neurons. It was exciting to see the animals memory problems come back after we silenced the transplanted cells because it showed that the new neurons really were the reason for the memory improvement, said junior specialist and first author of the study, Bingyao Zhu.

If this same treatment used in mice can be applied to humans, it could make a huge difference in the quality of life for those individuals. Next on the horizon is to create interneurons using human stem cells.

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New Cell Therapy Gives Traumatic Brain Injury Patients Hope - TrendinTech

UK cell and gene therapy firms are worried a shortage of skilled manufacturing staff will slow growth with some concerned Brexit will exacerbate the problem.

The findings come from a new skills survey by the Cell and Gene Therapy Catapult (CGTC) an organisation set up to support the sector by non-departmental government body, Innovate UK.

Of the 41 companies that responded, 98% said they planned to expand their headcount over the next five years. Of these, 83% raised concerns that hiring and retaining skilled staff will be an issue for growth.

Image: iStock/philhol

In addition, some respondents were concerned that Brexit will have a negative impact on recruiting and retaining skilled non-UK EU people according to the report.

More than 1,700 people are employed in bioprocessing roles in the UK cell and gene therapy sector, which is up from the 500 or so working in such positions in 2017. Based on this growth rate the CGTC expects 3,800 people will be working in such roles by 2024.

The survey also revealed 492 people are employed in cell and gene therapy manufacturing roles in the UK. This is expected to increase to 1,456 people up 196% by 2024. Respondents said finding staff with manufacturing skills as a substantial concern.

To address this, the report authors suggested cell and gene therapy firms would need to look beyond the sector.

The lack of talent will highly likely act as a brake to growth, with significant negative consequences on both organic and inward investment.

It is recommended, that companies are supported to deliver on their growth strategies, through the provision of supportive schemes, to both upskill their existing workforce as well as recruiting new talent, from outside of the sector.

The survey did not tally the number of EU nationals working in the UK cell and gene therapy sector or look at the potential impact Brexit would have on sectors ability to recruit.

However, a CGTC spokesperson told us We are confident that the UK cell and gene therapy industry will be able to source the necessary skills, and that the opportunities in research and training will remain attractive.

The spokesperson suggested overseas scientists continue to view the UK as attractive, adding We continue to see skilled people wanting to work in the UK sector from across the globe.

In addition, the spokesperson also expressed confidence cell and gene therapy developers would continue to see the UK as an attractive development and production base post withdrawal.

The supply chains for these advanced therapies are highly specialised and have been stringently developed in collaboration with medicine regulators to minimise any potential disruption.

There are already a number of advance therapies manufactured in the UK and exported seamlessly to the US for clinical trials and vice versa, we are confident that the movement of these therapies will remain unimpeded.

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Lack of UK cell and gene therapy skills a concern - Bioprocess Insider - BioProcess Insider

Takeda and Anderson Cancer Center announce cooperation to accelerate the development of clinical stage, out-of-the-box CAR NK cell therapy platform

University of Texas MD Anderson Cancer Center(The University of Texas MD Anderson Cancer Center)and Takeda Pharmaceutical Company Limited (Takeda Pharmaceutical hasannounced an exclusive licensing agreement and research agreement to develop cord blood-derived chimeric antigen receptors for "arming" IL-15 for the treatment of B-cell malignancies and other cancers Chimeric Antigen Receptor-directed Natural Killer (CAR NK) cell therapy.

Under the agreement, Takeda will receive access to the CAR NK platform of the Anderson Cancer Center and the exclusive rights to develop and commercialize up to four projects, including a CD19-targeted CAR NK cell therapy and a B-cell maturation. The antigen (BCMA) is the target of CAR NK cell therapy.The Takeda and Anderson Cancer Center will conduct a research collaboration to further develop the above CAR NK project.

Katy Rezvani, MD, Ph.D.,Professor ofStem Cell Transplantation and Cell Therapyat theAnderson Cancer Center,said: "Our vision is to improve existing treatments by developing armored CAR NK, which can be administered in outpatient settings. Out of the box, more patients can get effective, fast and least toxic treatment. Takeda's expertise in hematological malignancies and the commitment to develop next-generation cell therapies make it an ideal partner for our team. Promote CAR NK cell therapy for patients with therapeutic needs."

New ways to deliver out of the box ready to use CAR in outpatient facilitiesallogeneic Anderson Cancer Center platform CAR NK NK cells isolated from umbilical cord blood, the fight against certain cancers expressing CAR targets after processing.CAR NK cells are modified by retroviral vectors to deliver genes and enhance their efficacy against specific tumors.CD19 CAR increases the specificity of these cells against B cell malignancies, while the immune cytokine IL-15 enhances the proliferation and survival of CAR NK cells in vivo.

Existing CAR T cell therapy drugs use patient-generated genetically modified T cells, and the preparation process takes several weeks. In contrast, CAR NK cells are designed to be prepared from non-relative donor sources and stored in an out-of-the-box manner. So that treatment can be implemented faster.

CD19 CAR NK cell therapy is expected to be administered in an outpatient setting.In an ongoing phase 1/2a clinical study, patients with relapsed refractory B-cell malignancies who received CD19 CAR NK cell therapy did not see severe cytokine release syndrome (CRS) observed in existing CAR-T treatments. ) or neurotoxicity.

Anderson Cancer Center to develop CAR NK platform led by Dr. Rezvani, and withadoptive cell therapy platform,Chronic Lymphocytic Leukemia Moon ShotandB-the Cell Lymphoma Moon Shotfurther support of the project were the hospitalMoon Shots Programof In part, the project is a collaborative effort to rapidly develop scientific discoveries into meaningful clinical advances that can save patients' lives.

Takeda: CAR accelerate the development of next-generation multiple platformsAndy Plump Takeda, president of R & D, MD, Ph.D., said: "Anderson Cancer Center CAR NK cell therapy platform represents a potential cure for the drug, which is what we are to CD19 CAR NK Established as a leader in the treatment of drug candidates for cancer in the field. We must work flexibly and purposefully, so we plan to start the pivotal study of CD19 CAR NK in 2021."

In addition to CAR NK cell therapy, Takeda and its partners are investigating ways to improve the safety, efficacy, and accessibility of first-generation CAR T cell therapies, includinggamma delta CAR T,induced pluripotent stem cell-derived CAR T,with solid tumors as targets of CAR Tandother next-generation methods.Takeda plans to the end of fiscal year 2020 to promote the five kinds of tumor cell therapy to clinical stage.The platform is being developed through collaboration with partners and the use of Takeda's expertise in transforming cell therapy engines that provide bioengineering, chemistry, manufacturing and control (CMC), clinical and transformation functions in a single location to overcome cell therapy drugs. Numerous manufacturing challenges in development.

Takeda is responsible for the development, manufacture and commercialization of the CAR NK products produced by the agreement.The Anderson Cancer Center will receive upfront payments and be eligible to receive royalties for development and commercialization milestones for each target and for any CAR NK product net sales.

The Anderson Cancer Center and Takeda will continue to conduct research on other targets and the CAR NK platform under the direction of a joint research committee.The Anderson Cancer Center will deploy aninstitutional conflict of interest management and monitoring programfor the study.

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Takeda, Anderson Cancer Center team up for cell therapy - BSA bureau

The University of Texas MD Anderson Cancer Center and Takeda Pharmaceutical Company Limited have entered an exclusive license agreement and research agreement to develop and market chimeric antigen receptor-directed natural killer (CAR NK)-cell therapies.

Under the agreement, Takeda will receive access to MD Andersons treatment platform in order to develop and commercialize the CAR NK-cell therapies for up to 4 programs, according to the announcement made Tuesday.

With their expertise in hematologic malignancies and commitment to developing next-generation cell therapies, Takeda is the ideal collaborator to help our team advance CAR NK-cell therapies to patients in need of treatments, said Katy Rezvani, MD, PhD, a professor of stem cell transplantation and cellular therapy at MD Anderson.

The therapy has a similar strategy to the much-touted CAR T-cell therapy, which shows major promise in many cancers, by collecting certain white blood cells of patients, arming them with targeted surface receptors to battle the subjects particular cancer, and then infusing them back into the patients blood.

However, chemotherapy may leave some patients without sufficient autologous T cells in their blood for treatment with CAR T-cell therapy, while others may not have the time that is required for a laboratory to generate enough T cells, according to the researchers.

CAR NK-cell therapy, developed at MD Anderson, uses natural killer cells from cord blood. The team has said that it allows production of a therapy that doesnt have to be tailored for each and every patientand also obviates the possibility of graft-versus host disease, which is a danger with some T-cell varieties.

The MD Anderson team used a retrovirus to introduce new genes into the NK cells: CD19 is added to increase the CAR NK specificity for B-cell malignancies; interleukin 15 (IL15) is added to prolong the present of the cells in the body; and a CASP9-based suicide gene as a kind of safety measure, which can be activated to trigger apoptosis by small-molecule dimerizers if there is toxicity after infusion.

In announcing the agreement, MD Anderson and Takeda emphasized that the off-the-shelf CAR NK treatment could be administered at outpatient locations.

So far, the treatment has proven safe: An ongoing phase I/2a clinical study in patients with relapsed and refractory B-cell malignancies showed that the CD19 CAR NK-therapy has not been associated with the severe cytokine release syndrome or neurotoxicity observed with existing CAR-T therapies.

Takeda said they plan to initiate a pivotal study of the CD19 CAR NK-cell therapy in 2021.

MD Anderson receives an upfront payment that was unspecified by the parties as part of the deal, as well as tiered royalties on eventual net sales, according to the statement.

Rezvani said the goal is to make therapies that get to patients and ultimately change lives.

Our vision is to improve upon existing treatments by developing armored CAR NKs that could be administered off-the-shelf in an outpatient settingenabling more patients to be treated effectively, quickly, and with minimal toxicities, said Rezvani.

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MD Anderson Partners with Takeda to Develop CAR Natural Killer-Cell Therapy - Cancer Network

A crew of Amgen and Kite vets is publicly throwing their new startup into the cell therapy hat though they are saying little outside of the basics and $57 million in Series A cash.

The Column Group, Vida Ventures, Samsara BioCapital and Nextech Invest are backing A2 Biotherapeutics quest to find new ways of engaging immune cells in selectively attacking cancer. To do so, the biotech is working with two target classes: peptide MHC targets and targets that are irreversibly lost in tumor cells. The former builds on an increasingly popular strategy of locating neoantigens for T cells to home in on, while the latter is inspired by a mechanism used by natural killer cells.

A2 Biotherapeutics has potent, highly selective binders that we combine into molecular constructs to integrate multiple signals and potentially provide a large therapeutic window, said CSO Alexander Kamb, who co-founded the biotech after a stint as SVP of research at Amgen. They could be antibody and T-cell receptor fragments, according to its statement.

Scott Foraker, his former colleague at the Big Biotech, recently signed on as president and CEO.

Working out of Agoura Hills, California, their team of 40 expects to usher the first product candidate into the clinic next year. Its four programs in development span cancer testis antigen for solid tumors; targets lost antigens for solid tumors; neoantigens for head and neck cancer; and neoantigens for pancreatic, colorectal and lung cancer.

It also plans on constructing a manufacturing facility arguably the most important core competency that differentiates one cell therapy player from another in 2020 with the mindset of ultimately producing its own commercial supply. The site would support an autologous approach, though A2 Bio didnt specify what kind of cells it would use as the source.

Their lead in technical ops is Michelle Kreke, who was credited for leading chemistry, manufacturing and controls for the pioneering CAR-T Yescarta.

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New cell therapy player launches with $57M from marquee investors to go after 'definitive' tumor targets - Endpoints News

News

This year, researchers from the University of Pennsylvania launched the first-ever clinical trial to genetically edit the immune cells of cancer patients using the technology.

Penn Perelman Center for Advanced Medicine | Courtesy photo

Researchers from the Abramson Cancer Center at the University of Pennsylvania are the first in the United States to attempt to genetically edit a cancer patients immune cells in the lab using CRISPR-Cas9 technology. Penn launched the first-ever U.S. clinical trial for this research earlier this year with support from the Parker Institute for Cancer Immunotherapy(PICI) andTmunity Therapeutics. On Wednesday, researchers confirmed they have successfully infused three participants in the trial thus far two with multiple myeloma and one with sarcoma and have observed the edited cells expand and bind to their tumor target with no serious side effects.

This trial is primarily concerned with three questions: Can we edit T cells in this specific way? Are the resulting T cells functional? And, are these cells safe to infuse into a patient? This early data suggests that the answer to all three questions may be yes, said Edward Stadtmauer, the studys principal investigator and the section chief of hematologic malignancies at Penn.

Its still too early, however, to answer the more pressing question the trial has posed: Can these genetically modified cells destroy cancer cells? But researchers believe the feasibility and safety theyve demonstrated so far provides optimism that the approach may be applicable across multiple areas of gene therapy research.

These early findings are the first step as we determine if this new breakthrough technology can help rewrite how we treat patients with cancer and perhaps other deadly diseases, said Sean Parker, the billionaire entrepreneur and founder and chairman of PICI. CRISPR editing could be the next generation of T-cell therapy, and we are proud to be a part of the first human trial in the United States.

It has taken Penn more than two years to get the appropriate institutional and federal regulatory approvals, and to recruit optimal participants for the trial. Patients had to be screened ahead of time to make sure their tumors were a match for the approach. Participants who met the requirements received other therapy as needed while they waited for their cells to be manufactured. Once that process was complete, all three received the gene-edited cells in a single infusion after a short course of chemotherapy.

Analysis of blood samples revealed that the CRISPR-edited T cells expanded and survived in all three participants. While none of the patients cancer cells have yet responded to the therapy, researchers say there were no treatment-related serious adverse events.

CRISPR has been more popularly known for its ethically questionable potential to alter human DNA, but the studys senior author and immunotherapy pioneer Carl June says his team is squarely focused on moving the field of gene therapy forward.

Our use of CRISPR editing is geared toward improving the effectiveness of gene therapies, not editing a patients DNA, June said. We leaned heavily on our experience as pioneers of the earliest trials for modified T-cell therapies and gene therapies, as well as the strength of Penns research infrastructure, to make this study a reality.

Previous human trials using CRISPR technology have predominantly been conducted in China until several trials launched in the U.S. this year. The technology is currently being tested by researchers in Massachusetts to potentially treat genetic blood disorders like sickle cell disease and certain forms of inherited blindness. Penns team aims to test the approach on a total of 18 patients by the end of the trial.

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Here Are the Early Results From the First US CRISPR Trial for Cancer - Philadelphia magazine