CRISPR lauds easy scale-out of cell therapy – BioProcess Insider – BioProcess Insider

The autologous nature of its gene-edited stem cell candidate CTX001 means scaling manufacturing from clinical to commercial will be relatively easy, says CRISPR Therapeutics.

Codeveloped with Vertex Pharmaceuticals, CRISPR Therapeutics CTX001 is an autologous CRISPR/Cas9 gene-edited hematopoietic stem cell therapy targeting patients suffering from -thalassemia and sickle cell disease.

Production of the candidate involves collecting a patients own blood stem cells via mobilization and apheresis and editing them with CRISPR/Cas9 to increase fetal hemoglobin (HbF) expression. When the edited cells are returned to the patient, they are expected to generate red blood cells that have increased levels of HbF, which may reduce or eliminate patients symptoms.

Image: iStock/zest_marina

The firm recently presented positive Phase I/II data for the autologous candidate and now has an eye on scaling-up production as it progresses through the clinic.

We dont believe we will need to make any major modifications to the manufacturing process that we started with, to take it into commercialization, and thats a huge benefit, Lawrence Klein, CRISPRs COO said at the 20th Annual Needham Virtual Healthcare Conference this week. And thats just by virtue of the fact that our initial manufacturing process led to the type of efficacy that we saw, and we purpose built that process to enable commercial scale.

Therefore, to increase production the firm needs to add more suites: scaling-out, rather then scaling-up.

You can basically just clone those suites, and the more suites you have the more throughput you have, he told delegates. Its not like if youre moving from a 10-liter reactor to a 10,000-liter reactor [when] things change in terms of the biochemistry of the process.

To augment production adding people and facility and instrumentation the firm uses undisclosed contract manufacturing organizations (CMO).

When we started the trial, we had one, said Klein. Weve added CMOs since that point, different facilities and we intend to continue doing that to enable broader scale in different geographies.

The CMOs that weve chosen, theyre scalable. And you can see the major CMOs are making heavy investments in this space of gene and cell therapy. And so, we think well be able to scale that capacity as we move forward into commercialization.

CRISPR Therapeutics is also building a cell therapy manufacturing facility in Framingham, Massachusetts being designed to provide GMP manufacturing in compliance with US Food and Drug Administration (FDA) and European Medicines Agency (EMA) regulations and guidelines.

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NeoProgen, Inc. Receives First Granted Patent for Method to Treat Cardiac Conditions using Neonatal Heart-derived Medicinal Signaling Cells (nMSCs)…

BALTIMORE--(BUSINESS WIRE)-- NeoProgen, Inc., a pre-clinical stage company developing an exosome-based product from human neonatal heart-derived Medicinal Signaling Cells (nMSCs) for tissue repair and regeneration for the treatment of heart failure (HF) and other inflammatory diseases, announced that it has received a patent from the United States Patent Office Patent No.: US 10.967,007 B2 Cardiac Stem Cells for Cardiac Repair. The innovation was developed by Dr. Sunjay Kaushal, a pediatric cardiac surgeon, while he was at the University of Maryland, Baltimore and NeoProgen is the exclusive licensee of the newly issued patent.

The patent claims describe methods for treating cardiac conditions with an exosome-based therapy (conditioned media) derived from neonatal cardiac stem cells. The unique approach that the company has taken to isolating the cells, culturing nMSCs, characterizing them by their secretome and exosome production, and improving cardiac function by administering allogeneic nMSCs to adults and children represents an exciting therapy for heart failure and other inflammatory diseases.

As a Cardiovascular-Thoracic surgeon, my goal is to help patients by performing heart transplantations and heart surgeries, in many cases to newborn babies that need these procedures to correct life-threatening congenital problems. For many years I have had the opportunity to study stem cells that are present in the very young heart to treat chronic diseases seen in adults, having been involved as a Principal Investigator in multiple stem cell trials. Comparative studies analyzing multiple stem cell types support the notion that nMSCs and their secretomes/exosomes derived from neonatal heart tissue covered under this patent are the most regenerative stem cell type discovered, said Dr. Sunjay Kaushal, MD, PhD, Division Head Cardiovascular Thoracic Surgery at Ann & Robert H. Lurie Childrens Hospital of Chicago and founder of NeoProgen. Dr. Kaushal explained, Stem cell ingredients are key to their success in clinical trials. nMSCs are very special cells with a great regenerative potential and prolific abilities. The innate abilities of secretomes/exosomes from these cells set us apart from other stem cell types used in clinical trials. Its my hope that in the future pediatric and adult patients will not only benefit from the surgeries but also by stem cell therapies like this one to improve their outcomes and quality of life.

This is a major milestone for NeoProgen, said Bill Niland, the companys CEO who has successfully founded and exited three previous healthcare companies. In addition to expanding our patent portfolio, this patent gives us coverage of our #1 asset: a secretome/exosome product that we expect to get into a phase 1 trial this year for Ischemic Cardiomyopathy, where we have excellent pre-clinical results.

The company has engaged BioPharma Capital to exclusively represent them in actively raising capital and in discussions with potential strategic partners. Dan Ross, Managing Partner of BioPharma Capital added, NeoProgen stands out amongst its peers as harnessing a uniquely potent source for stem cells that, along with this newly issued patent, addresses both of the challenges that have historically beleaguered the stem cell therapy space and continue to do so today: manufacturing/supply and IP. We are pleased to be working with NeoProgen and look forward to doing our small part in helping this promising treatment make its way towards helping patients around the world.

About NeoProgen:

NeoProgen, Inc., is a pre-clinical stage company developing human neonatal heart-derived Medicinal Signaling Cells (nMSCs) and an exosome-based product for tissue repair and regeneration for the treatment of heart failure (HF) and other inflammatory diseases.

About BioPharma Capital:

BioPharma Capital, LLC provides life sciences focused investment banking services, including M&A Advisory, strategic partnering and financing transaction management. We are dedicated to maximizing value for our clients using credible scientific and evidence driven approaches. Our clients are typically pharmaceutical and biotech companies considering partnering, out-licensing, divestitures, fund raising or corporate sales. Our team provides decades of experience in transaction support, healthcare strategy consulting, pricing and market access, forecasting, investment banking and translational research. We understand the science and technology behind the assets we represent and we have vast expertise managing transactions. Securities and Investment Banking Services are offered through Ashland Securities, LLC. Supervised by the Home Office, located at 80 S.W. 8th Street, Suite 2000 Miami, Florida 33130. Phone Number 305-279-3176. Member FINRA SIPC. Please refer to BrokerCheck for more information about Ashland Securities, LLC. BioPharma Capital, LLC and Ashland Securities, LLC are separate and unaffiliated entities.

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NeoProgen, Inc. Receives First Granted Patent for Method to Treat Cardiac Conditions using Neonatal Heart-derived Medicinal Signaling Cells (nMSCs)...

CRISPR gene therapy for sickle cell disease approved by the FDA – BioNews

12 April 2021

A clinical trial for a new gene therapy approach to treat sickle cell disease has been approved to proceed by the US Food and Drug Administration.

Patients with sickle cell disease have a mutation in the beta-haemoglobin gene, causing them to produce misshapen red blood cells that can block blood vessels leading to severe pain, anaemia and potentially life-threatening complications, such as organ damage and strokes.Currently, the only cureis a stem cell transplant from a healthy donor, but in the newly-approved trial, scientists from the University of California will use CRISPR/Cas9 genome editing to replace the faulty gene with a functional version.

'Gene therapy and genome editing allow each patient to serve as their own stem cell donor,' said Professor Donald Kohn, from the Broad Stem Cell Research Centre at the University of California Los Angeles, one of the clinical trial leaders. 'In theory, these approaches should be much safer than a transplant from another person and could become universally available because they eliminate the need to find the needle in a haystack that is a matched stem cell donor.'

In the trial, blood stem cells will be harvested from the patients and grown in the lab. CRISPR/Cas9 will be used to 'cut and replace' a sequence of DNA containing the mutation with a healthy copy. The edited cells will then be returned to the patient's body in the same way they would be if the patient was receiving donor stem cells.

'The goal of this form of genome editing therapy is to correct the mutation in enough stem cells so the resulting blood in circulation has corrected red blood cells,' said Dr Mark Walters, from the University of California San Francisco Benioff Children's Hospital, another of the clinical trial leaders.

The study will take place over four years, and include six adults and three adolescents with severe sickle cell disease, testing both safety and efficacy.

The treatment does have risks: the patientswill need to have high dose chemotherapy, to kill allremaining bloodstem cells before the modified stem cells are put back. This is also necessary before receiving donor stem cells and can cause severe side effects as the patient's immune system is temporarily disabled.

A similar trial, using CRISPR/Cas9 to activate bone marrow stem cells to produce an alternative version of haemoglobin, rather than correcting the faulty version, has recently shown promising results in a patient with sickle cell disease (see BioNews 1052).

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CRISPR gene therapy for sickle cell disease approved by the FDA - BioNews

Man’s heart healed by stem cell therapy and love of an old flame – Leeds Live

A Wakefield man has shared the story of how his broken heart was fixed following a chance encounter with an old flame who was looking after him in hospital.

Fitness fanatic Barry Newmans heart was working at just 13% capacity and had him fearing for his life before fate stepped in with a revolutionary medical procedure and an old girlfriend.

At one point Barry was a shell of his former self and a heart transplant looked like the only viable solution.

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But after coming across a new procedure on The One Show he underwent surgery at a private clinic and his heart is now operating at double its former capacity, with one doctor saying he was a "walking miracle".

By chance, one of the people caring for Barry at Leeds General Infirmary was cardiac physiotherapist Nicki Simpson an old flame from their clubbing days in the 1980s.

Their love was quickly reignited and Barry soon popped the question, though it did result in a moment of awkwardness.

When he declared he had something to announce, Nicki feared he had yet more bad news about his health.

But then plasterer Barry smiled and, from behind his back, pulled out a ring with a large diamond in it.

Speaking to The Mirror, Mr Newman said: Nicki had been my cardiac physio since just after I was first diagnosed with my heart condition, so she knew better than anyone what shed be committing to if she said yes.

But within an instant she gave me a massive hug and kiss and said shed love to be my wife.

With Mr Newman's heart failure, the couple feared they might be on borrowed time.

But Barry, 54, would have another lucky break after chancing upon a revolutionary stem cell procedure that has already doubled his hearts capacity to 26% of normal function.

And that means he and Nicki can think about setting a wedding date next year.

Their remarkable love story goes back to their teenage days. Though they eventually drifted apart, an unlikely reunion came after Barry fell ill in late 2013.

After years priding himself on his healthy lifestyle, he began feeling short of breath, to the point of falling to his knees.

He was prescribed antibiotics but the problem persisted. In February 2014 a CT scan showed he had a massive, dilated heart and critical condition cardiomyopathy.

Doctors said it had likely been caused by a virus and his heart was working at a fraction of its usual capacity.

Barry said: I was told the only real solution was a heart transplant.

I was transferred to Leeds General Infirmary for what was basically palliative care to keep me alive as long as possible.

Compared to just a year before, I felt like my body was broken, all my strength was gone and I feared Id not see my son Robert graduate.

Barry had ongoing care from a cardiac nurse and, towards the end of 2014, was told he would also have the support of a cardiac physiotherapist.

He says: I was amazed when that person turned out to be Nicki, an old girlfriend from our clubbing days in the 1980s.

Wed been an item when we were 19. We drifted apart and I hadnt seen her since.

As soon as the pair clapped eyes on one another, their old spark was back even with Barry so terribly unwell.

We hit it off straight away, laughing at all the old memories, he says. In no time we were a couple again, almost three decades later.

Of course she knew this was a rather precarious situation for me as well as us as a couple.

But that didnt bother her one bit.

With professional and emotional support from Nicki, 54, Barry had a defibrillator fitted in his chest in 2016 which would restart his heart if it ever lost rhythm.

He has remained realistic throughout and says: I knew this was just another strategy to keep me alive until I reached crisis point and could be put on the transplant list.

I got progressively worse, to the point where every time I ate anything Id go grey and collapse and would have to be rushed to A&E.

This happened so many times I got to know all the nurses and porters at the hospital by first name.

Then in October 2017, Barry and his son saw a jaw-dropping report about work by the Heart Cells Foundation charity on TVs The One Show that would change the course of his life.

The innovative procedure involved taking bone marrow from the patients pelvis, harvesting stem cells, then injecting them straight into the heart.

With his "heart nearing its end", Barry instantly got his son Robert to contact the charity and its lead clinician, Professor Anthony Mathur.

Barry said: It seemed such a quick and painless procedure."

The foundation treats patients who are at the end-stage of life, and in November 2018 Barry underwent week-long treatment in London.

After a series of injections to stimulate the bone marrow, the procedure was carried out on the last day.

Barry said he was staggered by its apparent simplicity and how a routine op two months later showed how it was already working.

He says: It couldnt have been less painful.

I was booked in for a small operation in January 2019 and the anaesthetist said because of my heart he was hesitant about putting me under. I did a treadmill test to see how strong my heart was and the doctor said I was a walking miracle my results didnt match with my records.

Barry says he has since gone from strength to strength. He adds: While some cardiologists Ive spoken to are still cynical about my stem cell procedure, the facts speak for themselves.

My heart is now at 26% capacity, Im not taking all the various meds I was prescribed before, I can exercise properly, and feel amazing.

Before my procedure one of my worst fears was my amazing dad would have to go to my funeral, something no parent should ever have to do.

Sadly, I lost him in 2020 after a cancer battle, but I was so grateful he didnt have to watch me reach the end, to bury his own son. I also got to see Robert, 25, graduate in aeronautical engineering from Sheffield University, going on to work for a Formula 1 team and Nicki and I are planning our wedding next year.

This miraculous stem cell treatment hasnt just saved my life, its given me and my family a future, which is priceless.

Barry is among nearly 400 Brits who have received the treatment in its early phase and the success rate is 80%.

Patients typically experience improved heart function and a better quality of life with no further use of medication. Now doctors hope it could change the lives of countless more people. The procedure costs up to 10,000 a go and is not currently available on the NHS.

Learn more at heartcellsfoundation.com

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Man's heart healed by stem cell therapy and love of an old flame - Leeds Live

Autologous Stem Cell Based Therapies Market Professional Report 2021 Witness Robust Expansion by 2026 SoccerNurds – SoccerNurds

Global Autologous Stem Cell Based Therapies Market offers a complete analysis which includes market size, share, overview, and growth prospects that are impacting the growth of the Autologous Stem Cell Based Therapies Industry. Global Autologous Stem Cell Based Therapies market report offers consumers to recognize the market challenges and opportunities. This report provides the latest information about the technological developments and market growth prospect based on the regional landscape.

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The report also focuses on the global major leading industry players of the Global Autologous Stem Cell Based Therapies market providing information such as company profiles, product picture, and specification, capacity, production, price, cost, revenue, and contact information.

Major Players Covered in Autologous Stem Cell Based Therapies Market Report are:

Based on product, this report displays the production, revenue, price, market share and growth rate of each type, primarily split into:

Based on the end users/applications, this report focuses on the status and outlook for major applications/end users, consumption (sales), market share and growth rate for each application, including:

Geographically, the detailed analysis of consumption, revenue, market share and growth rate, historic and forecast of the following regions are:

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PART 09: DECISION FRAMEWORK

PART 10: DRIVERS AND CHALLENGES

PART 11: Autologous Stem Cell Based Therapies MARKET TRENDS

PART 12: COMPETITIVE LANDSCAPE

PART 13: COMPETITIVE ANALYSIS

PART 14: APPENDIX

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Mastitis therapy explored | AG | kmaland.com – KMAland

Apart from antibiotics dairy farmers have few tools to treat mastitis. So researchers at Cornell Universitys College of Veterinary Medicine, are exploring compounds secreted by stem cells as a potential therapy.

The researchers previously investigated the beneficial effects of the bovine mammary stem-cell secretome. The secretome is comprised of all the compounds secreted from laboratory-grown bovine mammary stem cells. Preliminary work showed the secretome had antimicrobial properties. It prevented damage from bacterial toxins and promoted healing through the growth of blood vessels and recruitment of new cells.

Now with help from Elanco, Dr. Gerlinde Van de Walle, a veterinarian and associate professor of microbiology and immunology, and Dr. Daryl Nydam, faculty director of the department of population medicine and diagnostic services, will perform similar experiments with the secretome in actual cows. Theyre treating mastitis-infected cows using different components of the secretome to pinpoint which compounds are responsible for its beneficial effects.

Theyre looking for any antimicrobial effects, differences in milk production, signs of healing, and regeneration of the mammary tissue. They also are watching for changes in the bovine immune system that may help fight the infection and comparing the effects of different types of bovine stem cells.

The long-term goal would be a natural product that could be an adjunct or even a replacement for antibiotics, Van de Walle said.

The Foundation for Food & Agriculture Research awarded a $642,000 research grant to the Cornell researchers. If the treatment is effective and affordable, it has potential for adoption nationwide, resulting in enhanced milk production and farmer profitability, said Sally Rockey, the foundations executive director.

Elanco and the New York Farm Viability Institute contributed matching funds for a $1.4 million total investment. Addressing concerns about the increase in antibiotic-resistant bacteria, Elanco aims to provide farmers alternatives to the use of medically important antibiotics.

The research also has potential applications beyond mastitis, Van de Walle said.

If we find that naturally secreted biomolecules can both replace antibiotics and restore damaged tissue, this work could be expanded to other livestock and other diseases, she said.

Visit vet.cornell.eduand foundationfar.org and nyfvi.org for more information.

At KMA, we attempt to be accurate in our reporting. If you see a typo or mistake in a story, please contact us by emailing kmaradio@kmaland.com.

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Stem Cell Therapy Market Analysis 2021: Size, Share, Sales, Growth, Revenue, Type, Application & Forecast To 2027 SoccerNurds – SoccerNurds

Stem Cell TherapyMarket Size 2021 Industry Share, Strategies, Growth Analysis, Regional Demand, Revenue, Key Players and 2027 Forecast Research Report

The report envelops a few factors that have added to the development of the market lately. It features a couple of the main market drivers and investigations their effect available. Among all factors, the expanding number of organization consolidations and joint efforts decidedly affect market development. With this procurement, the organization will hope to beat its partners and in doing as such, set up a solid presence on the lookout. The report features a couple of the other organization consolidations that have graced the market as of late and measures their effect available.

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Top Key Players in this Market Research Report

Pharmicell Co., Ltd Holostem Terapie Avanzate S.r.l. Osiris Therapeutics, Inc. MEDIPOST Co., Ltd. Nuvasive, Inc. Celgene Corporation Anterogen Co., Ltd. Promethera Biosciences Fibrocell Science, Inc. RTI Surgical, Inc. RTI Surgical, Inc Cytori Therapeutics

By Types

Adult Stem Cells Human Embryonic Induced Pluripotent Stem Cells Very Small Embryonic Like Stem Cells

By Applications

Regenerative Medicine Drug Discovery and Development

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Some Point from Table of Content:

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CAR T-Cell Therapy Enters the Arena in Multiple Myeloma With Ongoing Research Ahead – OncLive

The FDA approval of idecabtagene vicleucel (ide-cel; Abecma) has established a role for CAR T-cell therapy in heavily pretreated, relapsed/refractory multiple myeloma, with the product showing an unprecedented response rate and a generally favorable safety profile, said Saad Z. Usmani, MD, FACP.

However, with additional BCMA-directed constructs, allogeneic CAR T-cell products, and bispecific antibodies in the pipeline, ide-cel could become just 1 of many novel options the field.

BCMA-directed CAR T-cell therapy has good activity in heavily pretreated patients with multiple myeloma, including patients with triple-class refractory disease, said Usmani. Cytokine release syndrome [CRS], neurotoxicity, and low blood counts are [common], but patients [typically] recover from these adverse effects [AEs]. The timing of CRS and neurotoxicity could be different [between products], but we are seeing good activity [overall].

On March 26, 2021, the FDA approved ide-cel as the first BCMA-directed CAR T-cell therapy for patients with relapsed/refractory multiple myeloma after 4 or more prior lines of therapy, including an immunomodulatory agent (IMiD), a proteasome inhibitor (PI), and an anti-CD38 monoclonal antibody.1

The regulatory decision was based on data from the phase 2 KarMMA trial, in which ide-cel elicited an overall response rate (ORR) of 72% and a stringent complete response (sCR) rate of 28% in patients with relapsed/refractory myeloma who had received at least 4 prior treatments.2

Another CAR T-cell product, ciltacabtagene autoleucel (cilta-cel) is currently in the myeloma pipeline, having demonstrated an ORR of 96.9% and an sCR rate of 67.0% in the phase 1b/2 CARTITUDE-1 trial.3 Notably, the median time to onset of CRS was 1 day with ide-cel compared with 7 days with cilta-cel.2,3 In April 2021, Legend Biotech, the developer of cilta-cel, announced that a rolling submission of a biologics license application to support the approval of the drug for the treatment of patients with relapsed/refractory multiple myeloma has been completed.4

In an interview with OncLive during an Institutional Perspectives in Cancer webinar on CAR T-cell therapy, Usmani, chief of Plasma Cell Disorders and director of Clinical Research in Hematologic Malignancies at the Levine Cancer Institute of Atrium Health, discussed the rapidly changing cellular therapy paradigm in relapsed/refractory multiple myeloma.

Usmani: Ide-cel is a BCMA-directed CAR T-cell therapy that is the furthest along in terms of clinical development; we have the most follow-up with that particular construct. The key takeaway from the data [from the KarMMA trial], which were shared during the 2020 ASH Annual Meeting and Exposition, was that the initial signal of efficacy in the dose-escalation study did play out in the subsequent dose-expansion cohort. Longer-term follow-up [data have shown that] patients are getting minimal residual diseasenegative complete responses; some patients who have been off of treatment are showing sustained remissions at 2 years and beyond.

There doesnt appear to be any long-term safety concerns so far with [ide-cel]. The CRS and neurotoxicity that is seen is short term [and occurs] during the early parts of treatment, usually within the first month or so.

Importantly, an analysis [showed that]patients with high-risk cytogenetics [or] extramedullary multiple myeloma tended to have the same disease response with ORRs around 70% or higher.

KarMMA-2 [NCT03601078] has several different cohorts, including patients who are receiving ide-cel in earlier lines of treatment, patients who are receiving ide-cel with prior exposure to BCMA-directed therapy, and patients [who are receiving ide-cel] with high-risk disease, which will serve as a safety signalgenerating cohort.

[The KarMMA-2 trial] led to the development of the KarMMA-4 trial [NCT04196491], which is a frontline study for high-risk patients post induction.

The KarMMA-3 trial [NCT03651128] is another ongoing trial comparing ide-cel with standard triplet regimens in relapsed/refractory multiple myeloma. Some ide-cel studies [within] the Cooperative Group Setting were in the [Blood and Marrow Transplant Clinical Trials Network] looking at ide-cel after autologous stem cell transplant in high-risk patients [with multiple myeloma].

The next endeavor that we are going to have as we are thinking about CARrelated products is how quickly we can give these therapies. Currently, it takes between 4 to 6 weeks from the time that we think about giving a patient CAR T-cell therapy to getting them on a trial, to [them undergoing] apheresis, and finally, getting the product back. Logistically, this can take up to 2 months on clinical trials. It would be ideal to have an off-the-shelf option, which is where the ALLO-715 concept is very important.

If we have an allogeneic product from a single donor, we can make 100 off-the-shelf products ready to go. If I need to give such a product to a patient, I can start lymphodepleting chemotherapy and give them CAR T-cell therapy [shortly thereafter].

ALLO-715 tried to do that and is being evaluated in a dose-escalation, early-phase study. The important thing [about ALLO-715] is that about 90% of patients were treated within 5 days of enrolling, which is a much shorter timeframe [compared with autologous CAR T-cell therapy]. Moreover, there appears to be clinical activity [with ALLO-715] at the highest [dose] of 320 million [cells]. Six out of 10 patients had a response with 4 out of 10 having a very good partial response or better. [The trial has] short follow-up in a small cohort of patients, but the data are encouraging.

The early approvals will come in the relapsed/refractory setting for patients who have had exposure or refractoriness to PIs, IMIDs, and [monoclonal] antibodies. Most of our early usage once [CAR T-cell therapies] come to the clinic will be for those patients, and part of their usage will be dependent on how much capacity each transplant and cellular therapy center has. Having said that, we are probably going to have a little bit more flexibility in how we can bridge patients to CAR T-cell therapy. Getting patients to CAR T-cell therapy would be ideal, so we will try to do that for most patients. However, bispecific antibodies are also coming down the pike, so those will be good backup options too.

The second most mature dataset we have is with cilta-cel from the CARTITUDE-1 trial, [showing] a very impressive 97% ORR. With [cilta-cel], the time to CRS onset and neurotoxicity events is a bit delayed compared with ide-cel. As we think operationally, if both products [become] available to us, maybe one [product could be] given in the outpatient setting vs monitoring patients in the inpatient setting early on. Every center will look at things in a little bit of a different way based on their resources, but it is a good problem to have more than 2 options for patients.

We also have the bb21217 construct, which has the same construct as ide-cel. However, a PI3K inhibitor is utilized during the CAR T-cell expansion to push it toward more of a memory T-cell phenotype, [which could translate] to long-term remission for patients.

Then we have the LUMMICAR constructs, which are somewhat similar in terms of data and safety to ide-cel with lower-grade CRS. [With the investigational] PRIME-BCMA-101 [product], the CAR T cells do their job and then are out of the system quickly. There too, we are seeing low rates of CRS overall, as well as low [rates of] grade 3 or 4 CRS.

The efficacy and safety, as well as the vein-to-vein time, appear to be the keys [to getting CAR T-cell therapy into the clinic], but it is early. Im curious to see how all of this evolves, especially with ide-cel and cilta-cel as frontrunners.

The advantages in favor of CAR T-cell therapies are that although its more labor intensive early on, they dont require maintenance. Patients do enjoy that quality of life after having recovered from CAR T-cell therapy.

The disadvantages [include] production time and capacity of transplant centers, which play a role in discussing what may be the best option for patients. With the bispecific antibodies coming along, it is highly likely that we could see usage of antibody-drug conjugates and bispecific antibodies in the community setting even before patients come to see us for a CAR T-cell therapy consultation. There are a lot of nuances [to utilizing these products] that we will have to figure out once we have the therapies [available].

[Ultimately], the one-and-done approach that CAR T-cell therapy [provides] is quite unique and many patients will favor that.

Editors Note: This interview took place prior to the March 26, 2021, FDA approval of idecabtagene vicleucel in relapsed/refractory multiple myeloma.

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Leukemia Cutis: Symptoms and Treatment – Healthline

Leukemia cutis can happen when leukemia cells enter your skin. This rare condition causes patches of discolored skin to appear on the body.

In some cases, the appearance of leukemia cutis lesions on the skin is the first sign of leukemia a cancer of the blood and bone marrow.

Along with standard leukemia therapies, this complication can usually be addressed with topical treatments to help heal the damaged skin. If you have leukemia cutis, your outlook will usually depend on your age and the type of leukemia you have.

Leukemia cutis is an uncommon complication, affecting only about 3 percent of people with leukemia. However, it is often a sign that the cancer is at an advanced stage.

With leukemia, malignant leukocytes (white blood cells) are usually only present in the bloodstream. In the case of leukemia cutis, the leukocytes have entered the skin tissue, causing lesions to appear on the outer layer of your skin. The word cutis refers to the skin, or dermis.

Generally, leukemia cutis results in one or more lesions or patches forming on the outer layer of skin. This condition can mean that the leukemia is more advanced and may have spread to your bone marrow and other organs.

Because there are fewer healthy white cells to combat infections caused by other diseases, rashes and sores may be more common among people with leukemia. Low blood platelets from leukemia can cause damage to blood vessels that appear as red spots or lesions on the skin.

These may include:

However, these skin changes are different than those brought on by leukemia cutis.

While the legs are the most common area for leukemia cutis lesions to appear, they can also form on the arms, face, trunk, and scalp. These skin changes can include:

The lesions usually dont hurt. However, with certain types of leukemia particularly acute myeloid leukemia (AML) the lesions may bleed.

A dermatologist may initially diagnose leukemia cutis based on a physical examination of the skin and a review of your medical history. A skin biopsy is needed to confirm the diagnosis.

Leukemia cutis is a sign of leukemia. It wont develop if the body isnt already dealing with this type of blood cancer.

But leukemia isnt just one disease. There are multiple types of leukemia, each one classified by the kind of cell affected by the disease.

You can also have an acute or a chronic form of leukemia. Acute means it comes on suddenly and usually with more severe symptoms. Chronic leukemia develops more slowly and often with milder symptoms.

The types of leukemia that most commonly trigger leukemia cutis are AML and chronic lymphocytic leukemia (CLL).

Scientists arent sure why cancerous leukocytes migrate to skin tissue in some people with leukemia. It may be that the skin is an optimal environment for healthy leukocytes to transform into cancerous cells.

One possible risk factor that has emerged is an abnormality in chromosome 8, which has been found more often in individuals with leukemia cutis than in those without it.

Treating leukemia cutis usually includes treatment for leukemia as the underlying condition.

The standard leukemia treatment is chemotherapy, but other options may be considered depending on your overall health, your age, and the type of leukemia you have.

Other leukemia treatment options include:

For blood cancers, external beam radiation is a typical form of treatment. With this therapy, a focused beam of radiation is delivered outside the body from various angles. The goal is to injure the DNA in cancer cells to stop them from reproducing.

Immunotherapy, a type of biological therapy, uses the bodys own immune system to fight cancer. It is typically given by an injection that either stimulates immune system cells activity or blocks the signals cancer cells send to suppress the immune response.

Immunotherapy may also be given orally, topically, or intravesically (into the bladder).

Stem cell transplantation is more commonly known as a bone marrow transplant. Bone marrow is where blood stem cells develop. Stem cells can become any type of cell.

Through stem cell transplantation, healthy blood stem cells replace stem cells damaged by the cancer or by chemotherapy or radiation therapy. However, not everyone is a good candidate for this treatment.

Only treating the leukemia cutis lesions will not address the underlying disease of leukemia. That means treatments designed to remove or reduce lesions should be done in combination with systemic treatment for leukemia itself.

Treatments for leukemia cutis symptoms can include:

Again, these treatments will only treat the leukemia cutis lesions, but systemic treatment of the leukemia itself will be needed as well.

The length of time leukemia cutis lesions may last depends on many factors, including how well the leukemia itself is responding to treatment. If the leukemia goes into remission, its unlikely more lesions will appear.

With effective treatment, existing lesions could fade. However, other factors, including your age and overall health, can affect how widespread the lesions are and how long they may last.

There are encouraging trends in the treatment of leukemia, but it remains a challenging disease to treat and live with.

For people with AML who dont have leukemia cutis, research suggests that the survival rate at 2 years is about 30 percent. However, the survival rate drops to 6 percent among people with the skin lesions.

A separate study of 1,683 people with AML found that leukemia cutis was associated with a poor prognosis, and that those with AML and leukemia cutis may benefit from more aggressive treatment.

The outlook for people with CLL is better, with about an 83 percent survival rate at 5 years. The presence of leukemia cutis doesnt seem to change that outlook very much, according to a 2019 study.

Leukemia cutis is a rare complication of leukemia. It happens when malignant leukocytes invade the skin and cause lesions on the skins outer surface.

AML and CLL are more often associated with leukemia cutis than other types of leukemia.

While leukemia cutis usually means the leukemia is in an advanced stage, there are treatments for both the cancer and this uncommon side effect that may help extend life and improve its quality.

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Leukemia Cutis: Symptoms and Treatment - Healthline

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