Expansion and persistence of UCARTCS1A was observed and was found to correlate with clinically meaningful antimyeloma activity and serum cytokine changes in very heavily pretreated patients with multiple myeloma. Also, the CAR T-cell product was noted to be detectable in patients, regardless of donor and batch.

These preliminary data validate CS1 as a target for CAR T-cell products in multiple myeloma and that UCARTCS1A is a promising potential therapy for [those with this disease], Krina K. Patel, MD, MSc, an associate professor of the Department of Lymphoma/Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, said during a presentation on the results.

One of the benefits that comes with utilizing an allogeneic CAR T-cell approach over an autologous approach is that it affords the opportunity for off-the-shelf product availability, according to Patel. Patients are able to avoid a prolonged wait for the CAR T cells to be manufactured; the cells are able to be administered within a couple of weeks, Patel explained. In contrast, it can take 4 to 5 weeks to bring an autologous product to a treatment center.

Scalable manufacturing is another benefit of allogeneic approaches, and this can reduce costs and yield 100 or more doses from 1 batch of donor cells. Also, for allogeneic approaches, T cells are collected from healthy donors; these patients have not been given many steroids, chemotherapy, or have undergone autologous transplant. As such, their T cells will likely be more potent, Patel explained. Lastly, more flexible dosing is an option with allogeneic approaches; this allows for the possibility of redosing and alternate schedules.

UCARTCS1A is the first allogeneic CAR T-cell product developed to target CS1 and SLAMF7, both of which are highly and consistently expressed in multiple myeloma, according to Patel. The product knocks out the TRAC gene to avoid graft-versus-host disease through disruption of T-cell receptor (TCR) assembly. The product also knocks out CS1 to facilitate robust expansion and yield, while avoiding fratricide. Lastly, UCARTCS1A has a RQR8 safety switch, which is a CD20 mimotope that can use rituximab (Rituxan) to kill the cells, if necessary.

Previously, the CAR T-cell product demonstrated durable in vivo efficacy against MM1S tumors. Here, NSG mice were given a 5 x 105 MM1S myeloma cell line, which is known to be pretty aggressive, Patel noted; this was labeled with GFP and was given for 10 days. Subsequently, the mice received the CAR T cells. Investigators observed CAR-positive cells at day 4 and M protein, which is a surrogate marker for multiple myeloma in mice and patients.

We were able to see an early response, as well. However, eventually, the T cells went down, and the myeloma started to go back up, Patel added. Looking at the imaging, mice who [received] CAR T cells obviously did much better and lived longer and there was a dose-dependent response where the mice that got the higher dose did better, with a much longer survival. Investigators were also able to demonstrate that the mice that received the CAR T-cell therapy experienced improvement in lytic lesions over time.

MELANI-01 enrolled patients with confirmed multiple myeloma per International Myeloma Working Group criteria who relapsed following previous therapy for their disease. To be eligible for enrollment, patients needed to have an ECOG performance status of 0 to 2 and acceptable organ function. They could have not previously received an investigational drug or cell/gene therapy targeting CS1.

The key eligibility [for this trial] is similar to most cell therapy trials [that are done in] myeloma. However, for most of those trials, patients are not able to have previously received CAR T cells or BCMA-directed therapies, Patel said. In this trial, [those are not] ineligibility [criteria]. Our patients had really relapsed/refractory [disease.]

After going through screening, patients received lymphodepletion chemotherapy that was comprised of fludarabine at a daily dose of 30 mg/m2 for 3 days followed by cyclophosphamide at a daily dose of 1 g/m2, also for 3 days. The [cyclophosphamide] dose was 2 to 3 times higher than what [has been] used in most other trials, Patel noted.

Patients then received treatment with UCARTCS1A. Patients were started at dose level 1, where they received 1 x 106/kg. One patient went on to dose level 2, which was 3 x 106/kg. Patients underwent their first disease evaluation at day 28.

The primary and secondary objectives of the study included safety and tolerability of UCARTCS1A, as well as determining the maximum-tolerated dose and efficacy of the product. Exploratory end points are examining expression of CS1 on multiple myeloma cells, UCARTCS1A expansion and persistence, and changes in serum biomarkers or immune cell reconstitution.

Patel shared information on 5 patients who received treatment with UCARTCS1A to date; 4 of the patients received dose level 1 (102-101, 102-109, 102-107, and 102-111) and 1 patient (102-113) received dose level 2.

Four of the 5 patients (102-101, 102-109, 102-107, and 102-113) had previously received over 11 lines of therapy and had most had previously received a BCMA-directed therapy. Just to put this into context, most of the autologous CAR T-cell trials that are done have patients who had a median of 5 to 6 prior lines of treatment, Patel noted.

One patient (102-111) had received only 4 prior lines of therapy and was the only patient who had cells expand and responded on dose level 1. However, the patient had very high-risk disease with 90% plasma cells. He had the most myeloma going into the trial, Patel said.

Notably, patient 102-113 who had received dose level 2 and also experienced an expansion of cells at day 7 had received 13 prior lines of therapy, including 2 prior BCMA-targeted CAR T-cell therapies, the last of which was administered just 5 months prior to the study.

Patient 102-111 was 55 years of age, had 4 prior lines of therapy and 90% of bone marrow involvement. He had relapsed within 6 months of every prior line of therapy and he never experienced more than a partial response (PR) to any of his prior treatments, according to Patel. When looking at his peripheral blood at day 28, investigators noted that the CD45+ CAR-positive lymphocytes was almost 72% and a subgroup of CD8+ effector cells that are TCRnegative CAR-positive cells, were about 46%.

[Some might] think that allogenic cells would not last very long, but for this patient, we definitely saw the majority of T cells still there that were CAR positive, Patel said. For him, we were able to get a bone marrow [sample] at month 3, where we could also see CD45+ CAR-positive cells at 60% in the bone marrow of all CD45+ cells. The CD8+ effector [cells] were at 92%.

Moreover, CAR-positive cells were observed in the patients peripheral blood starting at day 14; they peaked at day 21, and then started to decrease. However, some of these cells were still observed at day 80 to 86, according to Patel. The patients white blood cell count was low, while peripheral blood was high, until approximately day 28, before it started decreasing. However, the patients bone marrow remained high, even at day 77, in terms of the vector copy number of the CAR T cells.

This patient experienced grade 2 cytokine release syndrome (CRS) within the first week of cell infusion. The patient also developed hemophagocytic lymphohistiocytosis (HLH), which has previously been observed with other autologous CAR T-cell products in multiple myeloma. Investigators treated the patients with anakinra (Kineret), dexamethasone, etoposide, and the rituximab kill switch. The rationale for triggering the kill switch was because the patient had reactivation of HHV6, which developed into HHV6 encephalitis.

Per the FDA, we were monitoring HHV6 and HHV7 levels, as we do for most of our CAR T-cell therapy trials. We were monitoring this [and when his levels were high enough that we decided to treat], the patient got admitted for antivirals, improved, went home, and then came back with an encephalitis picture. Initially, we treated him dexamethasone and gave the rituximab kill switch thinking that if it was immune effector cell-associated neurotoxicity, we could kill off some of the cells. But in the end, it was HHV6 encephalitis.

Although the patient did improve, and he had double antiviral coverage, he eventually passed away on day 109 from organizing pneumonia in the context of prolonged lymphopenia in the absence of multiple myeloma progression.

At the time, he did not have any myeloma and he had [experienced] this response that he had never had before, a near complete response Patel explained. We looked at his bone marrow, which was minimal residual diseasenegative at the 10-5 level. However, because of the prolonged lymphopenia, he ended up with this infection.

Multiple factors may have contributed to the prolonged lymphopenia, including viral reactivation, concomitant antivirals, and recent prior stem cell transplant, Patel explained.

The other patient with expansion, patient 102-113, was observed to have 25% CD45+ CAR-positive lymphocytes in the peripheral blood at day 9, 77% of which were CD8+ effector cells, according to Patel. Notably, investigators were unable to collect a bone marrow sample from the patient. In the peripheral blood, investigators observed expansion at day 7 and then a peak, and then the vector copy number persisted over the time the blood samples were obtained.

This patient had previously received 14 lines of therapy, including 2 previous BCMA-directed CAR T-cell therapies and associated lymphodepleting regimens, autologous transplant, and venetoclax (Venclexta), as his last line of therapy. The patient did not have any options left and we saw this fantastic response, where the lambda light chains had gone done by almost 90%; his M protein had at least a PR by just day 14.

However, this patient had CRS and HLH, as well. We treated him with etoposide, anakinra, dexamethasone, and the rituximab kill switch and he had improvement in his platelet and his liver function tests, Patel added. The HLH clinically improved for him. However, at day 25, he passed away.

An autopsy revealed G5 hemorrhagic pancreatitis, although he had not exhibited any clinical signs of this condition during his hospital stay. Investigators also found disseminated mucormycosis and pseudomonal pneumonia.

Select serum cytokine changes over time were found to correlate with expansion of the CAR T-cell product. Cytokines were increased much more in the patients who expanded vs those who did not expand at all, Patel noted.

MELANI-01 is currently enrolling patients with protocol modifications, including restarting at dose level -1 (3 x 105). Moreover, lower doses of lymphodepleting chemotherapy are being administered now in an attempt to address lymphopenia and lead to added expansion. The trial will also have additional requirements for monitoring and managing patients with regard to opportunistic infections, as well as CRS and HLH.

Patel KK, Bharathan M, Siegel D, et al. UCARTCS1A, an allogeneic CAR T-cell therapy targeting CS1 in patients with relapsed/refractory multiple myeloma (RRMM): preliminary translational results from a first-in-human phase I trial (MELANI-01). 2021 American Society of Gene and Cell Therapy Annual Meeting; May 11-14, 2021; Virtual. Accessed May 13, 2021. Abstract 118.

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CAR T-Cell Therapy UCARTCS1A Shows Early Activity in Relapsed/Refractory Myeloma - OncLive

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News Release

Tuesday, May 11, 2021

An investigational gene therapy can safely restore the immune systems of infants and children who have a rare, life-threatening inherited immunodeficiency disorder, according to research supported in part by the National Institutes of Health. The researchers found that 48 of 50 children who received the gene therapy retained their replenished immune system function two to three years later and did not require additional treatments for their condition, known as severe combined immunodeficiency due to adenosine deaminase deficiency, or ADA-SCID. The findings were published today inthe New England Journal of Medicine.

ADA-SCID, which is estimated to occur in approximately 1 in 200,000 to 1,000,000 newborns worldwide, is caused by mutations in theADAgene that impair the activity of the adenosine deaminase enzyme needed for healthy immune system function. This impairment leaves children with the condition highly susceptible to severe infections. If untreated, the disease is fatal, usually within the first two years of life.

These findings suggest that this experimental gene therapy could serve as a potential treatment option for infants and older children with ADA-SCID, said Anthony S. Fauci, M.D., director of NIHs National Institute of Allergy and Infectious Diseases (NIAID). Importantly, gene therapy is a one-time procedure that offers patients the hope of developing a completely functional immune system and the chance to live a full, healthy life.

People with ADA-SCID can be treated with enzyme replacement therapy, but this treatment does not fully reconstitute immune function and must be taken for life, usually once or twice weekly. Transplants of blood-forming stem cells, ideally from a genetically matched sibling donor, can provide a more lasting solution. However, most people lack such a donor. Additionally, stem cell transplants carry risks such asgraft-versus-host disease and side effects from chemotherapy medications given to help the donor stem cells establish themselves in the patients bone marrow.

The new research evaluated an experimental lentiviral gene therapy designed to be safer and more effective than previously tested gene-therapy strategies for ADA-SCID. This gene therapy involves inserting a normal copy of theADAgene into the patients own blood-forming stem cells. First, stem cells are collected from the patients bone marrow or peripheral blood. Next, a harmless virus is used as a vector, or carrier, to deliver the normalADAgene to these cells in the laboratory. The genetically corrected stem cells then are infused back into the patient, who has received a low dose of the chemotherapy medication busulfan to help the cells establish themselves in the bone marrow and begin producing new immune cells.

The experimental gene therapy, developed by researchers from the University of California, Los Angeles (UCLA) and Great Ormond Street Hospital (GOSH) in London, uses a modified lentivirus to deliver the ADA gene to cells. Previous gene-therapy approaches for ADA-SCID have relied on a different type of virus called a gamma retrovirus. Some people who have received gamma retroviral gene therapies have later developed leukemia, which scientists suspect is due to the vector causing activation of genes that control cell growth.The lentiviral vector is designed to avoid this outcome and to enhance the effectiveness of gene delivery into cells.

The results come from three separate Phase 1/2 clinical trials, two conducted in the United States and one in the United Kingdom. The U.S. trials, led by principal investigator Donald Kohn, M.D., of UCLA, enrolled 30 participants with ADA-SCID ranging in age from 4 months to 4 years at UCLA Mattel Childrens Hospital and the NIH Clinical Center in Bethesda, Maryland. The U.K. study, conducted at GOSH and led by principal investigator Claire Booth, M.B.B.S., Ph.D., enrolled 20 participants ranging in age from 4 months to 16 years. Most participants acquired and retained robust immune function following gene therapy 96.7% after two years in the U.S. studies and 95% after three years in the U.K. study and were able to stop enzyme replacement therapy and other medications. Of the two participants for whom gene therapy did not restore lasting immune function, one restarted enzyme replacement therapy and later received a successful stem cell transplant from a donor, and the other restarted enzyme replacement therapy. The lentiviral gene therapy appeared safe overall, although all participants experienced some side effects. Most of these were mild or moderate and attributable to the chemotherapy that the participants received.

Researchers observed similar outcomes in all three trials, although there were some differences between the studies. Stem cells were collected from bone marrow in the U.S. trials and from peripheral blood in the U.K. trial. In one of the U.S. trials, 10 children were treated with genetically corrected stem cells that had been frozen and later thawed. The two other trials used fresh stem cell preparations. In the future, the freezing procedure known as cryopreservation may allow stem cells to be more easily transported and processed at a manufacturing facility far from the patients home and shipped back to a local hospital, reducing the need for patients to travel long distances to specialized medical centers to receive gene therapy. A trial of the cryopreserved treatment is now underway at the Zayed Centre for Research into Rare Diseases in Children in London, in partnership with GOSH.

For more information about the trials described in the New England Journal of Medicine paper, visit ClinicalTrials.gov under identifiers NCT01852071, NCT02999984 and NCT01380990. The investigational lentiviral gene therapy, which is licensed to Orchard Therapeutics, has not been approved for use by any regulatory authority.

The research was funded in part by three NIH Institutes: NIAID; the National Heart, Lung and Blood Institute; and the National Human Genome Research Institute. Additional funding was provided by the California Institute for Regenerative Medicine, the Medical Research Council, the National Institute for Health Research Biomedical Research Centre at Great Ormond Street Hospital for Children National Health Service Foundation Trust and University College London, and Orchard Therapeutics.

NIAID conducts and supports research at NIH, throughout the United States, and worldwide to study the causes of infectious and immune-mediated diseases, and to develop better means of preventing, diagnosing and treating these illnesses. News releases, fact sheets and other NIAID-related materials are available on the NIAID website.

About the National Institutes of Health (NIH):NIH, the nation's medical research agency, includes 27 Institutes and Centers and is a component of the U.S. Department of Health and Human Services. NIH is the primary federal agency conducting and supporting basic, clinical, and translational medical research, and is investigating the causes, treatments, and cures for both common and rare diseases. For more information about NIH and its programs, visit http://www.nih.gov.

NIHTurning Discovery Into Health

DB Kohn, C Boothet al. Autologousex vivolentiviral gene therapy for adenosine deaminase deficiency.New England Journal of MedicineDOI: 10.1056/NEJMoa2027675 (2021).

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Gene therapy restores immune function in children with rare immunodeficiency - National Institutes of Health

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CLEVELAND When 61-year-old Ken Anderson was diagnosed with Multiple Myeloma 3 years ago, he didnt know what to expect.

It kind of hits you. It hits you hard, he said. Its a blood cancer, and its in your bone marrow, and it degenerates your bones is what it does.

The cancer is incurable, but treatable.

You live with it and you have to have many rounds of chemotherapy to keep the myeloma at bay, said Dr. Ted Teknos, the president of University Hospitals Seidman Cancer Center.

With so many unknowns, the dad of 4 girls and grandfather of 2 knew one thing, he was going to fight.

You just have to look to the road ahead, he said.

For the past 3 years, that road has been filled with ups and downs and countless rounds of chemotherapy treatments and even a bone marrow transplant.

They give you your stem cells back and those regenerate and lasted for about 6 months, and then there was a relapse, said Anderson.

Through it all, he remained hopeful for a medical breakthrough. He read about the research and followed up on the results of clinical trials in something called CAR T therapy.

I didn't know how far out that would be. It didn't say how far out it was. It sounded, to me, something like 10 or 20 years.

But it wasnt 20 years, the FDA approved CAR T therapy for Multiple Myeloma patients, and University Hospitals is the first in Ohio to treat patients with it. Anderson, who is from Kirtland, is the first patient in Ohio to receive it.

These treatments, now, are available for those that have run out of options, said Dr. Teknos.

Dr. Teknos compared the treatment to something straight out of a science fiction movie.

In essence, its like a heat-seeking missile for the cells to go find the cancer and eradicate it, he said.

It works by taking a patients own white blood cells, genetically modifying them in a lab and then infusing them back into their body so the patients cells can fight off the cancer cells.

They will engineer them to attack my cancer cells, said Anderson.

Dr. Teknos calls it living therapy.

You're taking living cells out of a patient, you're modifying them, and then you're growing them up in the lab and then re-infusing them back into the patient, he said. It's their own cells that have been modified and fight the cancer.

Dr. Teknos said in clinical trials, about 75% of Multiple Myeloma patients had a response to therapy, and in 1/3 of patients, their cancer went away.

Its really a game changer, said Dr. Teknos. There are patients who literally had weeks to live and then a year and a half later, have no cancer at all.

Andersons cells are currently in the lab. He will receive his infusion next month. He is cautiously optimistic that the next stop on his journey will have him feeling better.

I won't have to be on the chemo anymore, so I'm just back to feeling like myself would be would be really exciting, he said. People who are out there and diagnosed with this, with this disease, know that we are on the cusp of some big things here in the treatment of it, and this is a huge advance.

While Anderson is currently fighting Multiple Myeloma, University Hospitals is also offering a new CAR T cell therapy treatment for patients diagnosed with Diffuse Large B-Cell Lymphoma.

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University Hospitals treats first cancer patient in Ohio with "game changing" CAR T therapy - News 5 Cleveland

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Aplastic Anemia is a medical condition that damages stem cells in a persons bone marrow. These cells are responsible for making red blood cells, white blood cells, and platelets, which are vital to human health. A rare and serious condition, aplastic anemia can develop at any age. It can occur suddenly, or it can come on slowly and worsen over time. It can be mild or severe. Treatment for aplastic anemia might include medications, blood transfusions or a stem cell transplant, also known as a bone marrow transplant. The most common cause of aplastic anemia is from your immune system attacking the stem cells in your bone marrow. Other factors that can injure bone marrow and affect blood cell production include: Radiation and chemotherapy treatments, exposure to toxic chemicals, use of certain drugs, autoimmune disorders, a viral infection and unknown factors.

DelveInsights, Aplastic Anemia Pipeline Insight, 2021 report provides comprehensive insights about 10+ companies and 15+ pipeline drugs in Aplastic Anemia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Some of the Aplastic Anemia Companies are:

Request for free Sample Report: https://www.delveinsight.com/sample-request/aplastic-anemia-pipeline-insight

DelveInsights Aplastic Anemia report covers around 15+ products under different phases of clinical development like

Some of the Aplastic Anemia Therapies are:

Request for free Sample Report: https://www.delveinsight.com/sample-request/aplastic-anemia-pipeline-insight

Current Aplastic Anemia Treatment Scenario and Aplastic Anemia Emerging Therapies:

Request for free Sample Report: https://www.delveinsight.com/sample-request/aplastic-anemia-pipeline-insight

Table of Contents:

Introduction

Executive Summary

Aplastic Anemia: Overview

Pipeline Therapeutics

Comparative Analysis

Therapeutic Assessment

Aplastic Anemia DelveInsights Analytical Perspective

In-depth Commercial Assessment

Aplastic Anemia Collaboration Deals

Late Stage Products (Phase III)

Comparative Analysis

PF-06462700: Pfizer

Drug profiles in the detailed report..

Mid Stage Products (Phase II)

Comparative Analysis

Drug Name: Company Name

Drug profiles in the detailed report..

Early Stage Products (Phase I/II)

Comparative Analysis

Omidubicel: Gamida Cell

Drug profiles in the detailed report..

Pre-clinical and Discovery Stage Products

Comparative Analysis

Hu-PHEC: Hemogenyx

Drug profiles in the detailed report..

Inactive Products

Comparative Analysis

Aplastic Anemia Key Companies

Aplastic Anemia Key Products

Aplastic Anemia- Unmet Needs

Aplastic Anemia- Market Drivers and Barriers

Aplastic Anemia- Future Perspectives and Conclusion

Aplastic Anemia Analyst Views

Aplastic Anemia Key Companies

Appendix

Request for Detailed TOC: https://www.delveinsight.com/sample-request/aplastic-anemia-pipeline-insight

AboutDelveInsight

DelveInsight is a leading Business Consultant and Market Research firm focused exclusively on life sciences. It supports Pharma companies by providing end-to-end comprehensive solutions to improve their performance. Get hassle-free access to all the healthcare and pharma market research reports through our subscription-based platformPharmDelve.

Media ContactCompany Name: DelveInsight Business Research LLPContact Person: Ankit NigamEmail: Send EmailPhone: +19193216187Address:304 S. Jones Blvd #2432 City: AlbanyState: New YorkCountry: United StatesWebsite: https://www.delveinsight.com/report-store/aplastic-anemia-pipeline-insight

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Aplastic Anemia Pipeline: Emerging Therapies and Key pharma players involved by DelveInsight | Pfizer, Regeneron Pharmaceuticals, Regen BioPharma,...

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When he called the Olivia Hospital and Clinic to postpone his physical, he was urged to keep it. Physicals are important, he was reminded.

Keeping that date proved to be a lifesaving decision.

The physical went well, and shortly after he told his daughter that he was as fit as a horse.

But Dr. Jon Kemp, his primary physician who had urged him to keep the date for the physical, noticed a slight abnormality in a standard blood test. He recommended further testing.

On Dec. 20 Kramer was diagnosed with multiple myeloma.

Thanks to the early diagnosis, Kramer, age 62, has the means of keeping this disease at bay. Its a cancer of the plasma cells in bone marrow, and is the second most common blood cancer.

He is about to undergo a stem cell transplant this week as part of his treatment.

He learned that hes not alone on the journey ahead.

At Tuesdays meeting of the Renville County Board of Commissioners, fellow board members came wearing T-shirts proclaiming: In this county, nobody fights alone.

Organizers of the surprise sold 76 of the T-shirts to show support for Kramer and raise funds for the Renville County Walk in the Park campaign. More than 40 T-shirt wearing supporters joined the meeting via Zoom. Staff in the health department sang a song to express their support, and staff members told him they would keep him in their thoughts and prayers.

Thank you, said Kramer. He told the West Central Tribune that he was totally surprised by the display of support.

He has lots of support from family and friends, and its all-important. Kramer farms in eastern Renville County. He has lined up plenty of helping hands while he undergoes the stem cell transplant, which will sideline him for at least six weeks.

He said doctors are confident the stem cell transplant can knock the cancer into remission. They will be harvesting bone marrow cells and freezing a portion of them to make it possible to perform at least two more transplants in future years as well.

The decision to keep the date of that routine physical made all the difference. Absolutely, said Kramer.

Health providers told him that in too many cases, multiple myeloma is not diagnosed until a patient comes in with a broken leg or other bone, and wondering why. The cancer carves holes and weakens bones as it progresses unbeknownst to the person.

Thanks to the early diagnosis, Kramer said they found only pinholes in his bones, having caught the disease in the first of its three stages. He began chemotherapy in early January, and it has proven effective, he added.

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Keeping the physical appointment was critical, the show of support appreciated by Renville County Commissioner - West Central Tribune

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On the day of his Year 10 school prom, as other students excitedly prepared for the big occasion, then 15-year-old Rian Harvey was sat in a ward of Royal Marsden Hospital, awaiting the stem cell transplant that would save his life after a leukaemia relapse.

Despite the hot weather on that day back in July 2015, his hospital room windows had to remain sealed shut, as even the smallest bug bite could have killed him due to his compromised immune system.

Six years on, he finds himself grateful that he relapsed when he did, with five years to build his immunity before the Covid-19 pandemic hit.

Blood cancer patients are one of the most vulnerable groups of people at risk of Covid-19, according to research, being 57 per cent more likely to suffer severe disease compared to other cancer patients.

Recalling his own experience, Rian, now 22, says: Its scary, you look at everything that person has gone through, they had blood cancer and then had a stem cell transplant, they have gone through all the stress of only to be taken by a pandemic that came out of nowhere.

I know the vulnerability that you are in for stem cell transplants, Ive been there myself. Your immune system cant take anything.

Despite the high risk these patients face, charities such as Anthony Nolan, which assist blood cancer patients with finding a stem cell match, were left out of the allocated government budget that was announced in March.

The cancellation of face-to-face fundraising and events, despite the increase in demand for services, have led their gross income to be down by an estimated 5.5m for 2021.

Henny Braund, chief executive of the charity, said people with blood cancer and blood disorders were heavily impacted by the pandemic and everyone who needs treatment and support must be able to access it without delay.

This budget does not address the pressure currently facing cancer services across the UK, he adds.

Stem cell transplants are carried out to treat conditions such as blood cancer. The process involves removing the healthy stem cells of one person and transferring them to another, provided they have a similar or identical special genetic marker called the HLA.

While this match is sometimes present between family members, it is not always the case, leaving patients in the UK reliant on the British Bone Marrow Registry to find a suitable match. The odds of a match are one in 1000.

One of Anthony Nolans primary roles is to encourage more people to put themselves on the registry so patients have an increased chance to find a match. This can be done via a simple cheek swap, which provides sufficient HLA data for the initial matching process.

Will Briant, 24, from London, donated stem cells in 2015 after signing up to be on the registry at university. I think it ultimately is a huge part of who I am now, he says. Its something that I look to in my darker moments and find great inner strength from.

The identities of donors and recipients remain anonymous to one another, but they are allowed to exchange letters after the transplant.

I was incredibly emotional when I got the letter, he adds. He made it clear that not only was I giving him the chance of time for himself, but it was also for all of his family and friends, he told me he had a very big family. Looking back now, at a time where we cant all be with our families, it just highlights just how important and valuable that must have been for him.

Apart from encouraging people to sign up to the registry, the money Anthony Nolan raises go towards funding research, offering support and information to patients and families as well as providing post-transplant-care. They have helped 18,000 people find a match.

Unfortunately, they are part of the 35 per cent of charities who used the furlough scheme offered by the government to curb the loss of income. To ensure their survival, 24 per cent of surveyed charities said they were letting furloughed employees return as volunteers.

Terence Lovell, chief engagement and marketing officer at Anthony Nolan, says: We still desperately need funds to continue our life-saving work through providing stem cells transplants and co-ordinating efforts across the NHS to ensure patients receive the care and support they need.

Despite the circumstances, Rian has decided to make the most of his time in lockdown. He regularly shares his experience fighting cancer on his social media platforms and is currently in the process of writing a book and producing a podcast to further share his message.

The cancer mill is still very much open for business and I am trying to push people, that have not necessarily been through what Ive been through, to be more positive and see the world the way that I do, he says, I wake up in the morning, open my front door, take a deep breath of fresh air and I think this is amazing because five years ago I couldnt even open a window in the hospital.

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How Covid-19 has disrupted efforts to care for blood cancer patients - The Independent

Bone Marrow Stem Cells Comments Off on How Covid-19 has disrupted efforts to care for blood cancer patients – The Independent | May 15th, 2021

Dr Pria Suchak, 31, initially registered with blood cancer charity DKMS last July, when she was inspired by a message on social media.

Every 20 minutes someone in the UK is diagnosed with a blood cancer those that affect the body's bone marrow, blood or lymphatic system - such as leukaemia, myeloma or lymphoma.

Yet, only two per cent of the UK population are registered as potential blood stem cell donors.

Pria said: My friends nephew had leukaemia, so she was using her Facebook page to encourage strangers to sign up him.

"Her nephew is of mixed heritage - half Chinese and half Caucasian. So she was trying to encourage more people for minority ethnic communities to sign up.

"I wanted to help give someone a second chance of life, so I signed up with DKMS, and my husband registered at the same time.

Patients from black, Asian or other minority backgrounds have a 20 per cent chance of finding the best possible blood stem cell match from an unrelated donor, compared to 69 per cent for northern European backgrounds.

Pria ordered a home swab kit in July 2020 and was contacted by DKMS just five months later, informing her that she was a potential match for a stranger in need of a lifesaving blood stem cell transplant.

The mum-of-two said: I received a call from a lady at DKMS. She said I was extremely close to being a match, but there were also eight other people who were identified as possible matches too.

"A few weeks later, I received another call from DKMS saying that I was the best match out of the nine potential donors.

"I didnt expect that. As it was nine of us in total, you never expect you'll be chosen.

Following further tests and a medical examination, a date was set for Pria to donate her blood stem cells by peripheral blood stem cell collection (PBSC).

In the run-up to the procedure, donors are given a drug with the growth factor G-CSF to increase the number of stem cells in the blood.

Pria said: At the time I had so many things going on. We had just gotten past Christmas, both of my children had birthdays in January, and I was about to sit a final GP exam.

"DKMS were excellent and did their best to schedule my G-CSF injections the day after I sat the exam. Of course, they checked that this wouldnt impact the patient.

My actual donation was really nice, especially as there were other donors in the room at the same time donating for other patients.

"We all got on really well and chatted loads. The clinicians told us that we were the chattiest group they had ever had. Ive remained terrific friends with one of my fellow donors.

Because of the minimum two-year anonymity period in the UK, donors can only contact the patient anonymously, by letter or email.

Pria said: I dont know anything about my patient other than she is a woman. She really is a stranger, but I hope my blood stem cells help her to live a long life.

I strongly encourage people in Crawley to register with DKMS. By donating their blood stem cells, not only will you potentially help a stranger in desperate need, but you'll also help their family and friends by giving them more time together.

Crawley has a population of around 114,000 with 14 neighbourhoods, the largest inland town in West Sussex. Yet, just 865 residents have registered with DKMS.

On May 28, DKMS celebrates their day of awareness - World Blood Cancer Day. This May, the charity aims to register 2,000 new registrations (roughly one for every donor in the UK waiting) by the end of May 28.

If you are called upon to donate your blood stem cells it is because you are likely the patients best match.

There are two donation methods. Around 90 per cent of all donations are made through a method called peripheral blood stem cell (PBSC) collection.

This method is very similar to giving blood. It involves being connected to an apheresis machine. Apheresis means 'to separate'.

This machine separates blood being taken from one of the donor's arms, and separates the blood stem cells from it. The donor's blood is then returned to them through their other arm. This is an outpatient procedure that is usually completed in four-to-six hours.

In just ten per cent of cases, donations are made through bone marrow collection. Bone marrow is taken from the pelvic bone under general anaesthetic, and this lasts around an hour.

DKMS need blood stem cell donors from all backgrounds. If you are aged between 17-55 and in good general health, you can support Gareth and the other 2,000 people in need of a lifesaving blood stem cell transplant by registering online at http://www.dkms.org.uk/register-now for your home swab kit.

By registering, you'll join a group of over 840,000 other DKMS lifesavers-in-waiting, ready to make a difference by giving someone a much-needed second chance of life.

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Crawley doctor urges residents to save the life of a stranger - Crawley Observer

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This article was originally published here

PLoS One. 2021 May 13;16(5):e0243014. doi: 10.1371/journal.pone.0243014. eCollection 2021.

ABSTRACT

Multiple sclerosis (MS) is a complex, progressive neuroinflammatory disease associated with autoimmunity. Currently, effective therapeutic strategy was poorly found in MS. Experimental autoimmune encephalomyelitis (EAE) is widely used to study the pathogenesis of MS. Cumulative research have shown that bone marrow mesenchymal stem Cells (BMSCs) transplantation could treat EAE animal models, but the mechanism was divergent. Here, we systematically evaluated whether BMSCs can differentiate into neurons, astrocytes and oligodendrocytes to alleviate the symptoms of EAE mice. We used Immunofluorescence staining to detect MAP-2, GFAP, and MBP to evaluate whether BMSCs can differentiate into neurons, astrocytes and oligodendrocytes. The effect of BMSCs transplantation on inflammatory infiltration and demyelination in EAE mice were detected by Hematoxylin-Eosin (H&E) and Luxol Fast Blue (LFB) staining, respectively. Inflammatory factors expression was detected by ELISA and RT-qPCR, respectively. Our results showed that BMSCs could be induced to differentiate into neuron cells, astrocytes and oligodendrocyte in vivo and in vitro, and BMSCs transplanted in EAE mice were easier to differentiate than normal mice. Moreover, transplanted BMSCs reduced neurological function scores and disease incidence of EAE mice. BMSCs transplantation alleviated the inflammation and demyelination of EAE mice. Finally, we found that BMSCs transplantation down-regulated the levels of pro-inflammatory factors TNF-, IL-1 and IFN-, and up-regulated the levels of anti-inflammatory factors IL-10 and TGF-. In conclusion, this study found that BMSCs could alleviate the inflammatory response and demyelination in EAE mice, which may be achieved by the differentiation of BMSCs into neurons, astrocytes and oligodendrocytes in EAE mice.

PMID:33983943 | DOI:10.1371/journal.pone.0243014

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BMSCs differentiated into neurons, astrocytes and oligodendrocytes alleviated the inflammation and demyelination of EAE mice models - DocWire News

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Multiple myeloma is a type of cancer that originates from white blood cells called plasma cells. Many factors affect the outlook for a person with this disease, including their age, overall health, and kidney function, as well as the stage of cancer at diagnosis.

Multiple myeloma is a cancer of the plasma cells, which are a type of white blood cell. Over time, myeloma cells multiply and accumulate in the bone marrow and solid parts of the bones.

Multiple myeloma can lead to organ damage that affects the kidneys, the bones, and the overall immune system.

In this article, we look at the outlook for people with different stages of multiple myeloma. We also look at the symptoms and treatment of multiple myeloma and what can affect a persons outlook.

The American Cancer Society (ACS) estimates that doctors will diagnose 34,920 new cases of multiple myeloma in 2021 and that there may be 12,410 deaths from the disease.

When a person receives a multiple myeloma diagnosis, the doctor will use the Revised International Staging System (RISS) to determine the stage of the cancer. This staging system is based on:

A person will receive a diagnosis of either stage 1, 2, or 3 multiple myeloma. There is also a stage 0, a slow-growing type of multiple myeloma that is called smoldering myeloma.

However, survival rates are based on summary staging, which the Surveillance, Epidemiology and End Results (SEER) program developed. This staging system groups cancers into:

As multiple myeloma does not spread to the lymph nodes, the regionalized stage is not relevant to this cancer.

The 5-year relative survival rate for multiple myeloma is as follows:

These statistics mean that a person with localized multiple myeloma is 75% as likely as someone without multiple myeloma to live for 5 years after receiving the diagnosis.

People who receive a smoldering myeloma diagnosis can live for years without any treatment. Additionally, beginning treatment early does not appear to affect the outlook.

The stage of multiple myeloma is among the factors that can affect a persons outlook.

Other factors include:

A small 2014 study involving 82 people with an average age of 61 years found that those with damaged kidneys had a median survival rate of 13 months, whereas those without kidney damage lived for an average of 41 months.

Additionally, changes in chromosomes and genetic abnormalities can affect a persons outlook. The specific chromosomal abnormalities that doctors consider high risk affect chromosomes 4, 14, 16, and 17.

The treatment for smoldering myeloma typically consists of watchful waiting, as this stage is slow growing.

Drug therapy for multiple myeloma consists of:

Other treatment options include:

Multiple myeloma can cause:

A doctor may recommend supportive therapies to help manage these side effects. These may include surgery to help support weakened bones and prevent fractures.

Learn more about the treatment options and how to manage the symptoms.

A person should contact a healthcare professional if they notice any symptoms of multiple myeloma.

After receiving a multiple myeloma diagnosis, a person may want to ask the following questions:

Multiple myeloma is a type of cancer that affects the blood. The outlook for people with multiple myeloma depends on the stage of the cancer at the time of diagnosis. It also depends on how well a persons kidneys are functioning and their age and overall health.

However, different treatment options are available. A person should talk with a healthcare professional about which treatment options would best suit them.

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Outlook for multiple myeloma: Figures and factors that affect it - Medical News Today

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Gene therapy has shown promise in recent years for treating a range of diseases, including sickle-cell anemia, hemophilia, various forms of inherited blindness, mesothelioma, and Duchenne muscular dystrophy. A new success story may soon be added to this list, with the publication yesterday of the outcomes of a clinical trial that used gene therapy to cure a rare immune system disorder in infants.

The study, described in the New England Journal of Medicine, was carried out by researchers from UCLA and Great Ormond Street Hospital in London over the course of five years, beginning in 2012.

Adenosine deaminase (ADA) is an enzyme found in a type of white blood cell called lymphocytes, which are primarily active in the brain, GI tract, and thymus gland. Lymphocytes make antibodies and attack infected cells, so theyre pretty crucial to the immune system.

ADAs job is to convert a molecule thats harmful to lymphocytes into a non-harmful version of itself. If ADA cant work its magic, that molecule starts to build up in lymphocytes, becoming toxic and ultimately killing the cellsand leaving the immune system virtually defenseless, highly vulnerable to invaders like viruses and bacteria.

Mutations in the ADA gene mean the body doesnt make enough of the enzyme to successfully do its job. This deficiency of ADA leads to a condition called severe combined immunodeficiency (SCID). Those suffering from SCID can not only get sick very easily, but conditions that would be neutralized by a normal immune system quickly become deadly for them.

SCID was more commonly known as bubble boy disease after David Vetter, a boy born in Texas in 1971, spent 12 of his 13 years of life enclosed in a plastic bubble to protect him from germs.

About 20 different genetic mutations can cause SCID; ADA-SCID refers to immunodeficiency caused by lack of the ADA enzyme: severe combined immunodeficiency due to adenosine deaminase deficiencya bit of a mouthful. The worst part of ADA-SCID is that it occurs in babies; most are diagnosed with the condition before theyre even six months old, and without treatment they typically dont live past age two.

ADA is rare, estimated to occur in about 1 in 200,000 to 1,000,000 newborns worldwide; both the mothers and the fathers ADA gene must have mutations for the child to end up with this condition.

The first step in the gene therapy treatment was to collect hematopoietic stem cells, which are those that manufacture blood cells, from the patients. The researchers then inserted an intact copy of the ADA gene into the stem cells using an RNA virus called a lentivirus (the most well-known lentivirus is HIV).

The altered cells were re-injected into the patients, where they started producing ADA normally, yielding healthy immune cells.

Out of 50 total patients30 in the US and 20 in the UKwith ADA-SCID, 48 appear to have been rid of their condition thanks to the gene therapy, with no complications reported. The two patients who didnt have success with the therapy went back to traditional treatment methods, and didnt experience any adverse effects as a result of having tried the therapy.

If, or hopefully when, gene therapy becomes the go-to treatment for ADA-SCID, it will be a welcome reprieve from traditional options, which are neither pleasant nor cheap: patients need weekly injections of ADA until a bone marrow transplant can be done, and absent a donor, they must consistently receive injections, take antibiotics, and undergo antibody infusions for life.

If approved in the future, this treatment could be standard for ADA-SCID, and potentially many other genetic conditions, removing the need to find a matched donor for a bone marrow transplant and the toxic side effects often associated with that treatment, said Dr. Claire Booth, co-author of the study and a consultant in pediatric immunology and gene therapy at Londons Great Ormond Street Hospital.

Theres no mention of the cost of the therapy, nor whether this could be a prohibitive factor to making it a viable option. Nonetheless, the study is encouraging not just for its potential to revolutionize treatment of ADA-SCID, but as a harbinger for the promise of gene therapy for a multitude of genetic conditions.

People ask us, is it a cure? Who knows long term, but at least up to three years, these children are doing well, said Dr. Stephen Gottschalk, who was not involved in this study but performed a similar gene therapy on kids with SCID at St. Jude Childrens Research Hospital in Memphis. The immune function seems stable over time so I think it looks very, very encouraging.

Image Credit: liyuanalison from Pixabay

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How One Round of Gene Therapy Fixed 48 Kids' Immune Systems - Singularity Hub

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MILAN, Italy and NEW YORK, May 14, 2021 (GLOBE NEWSWIRE) -- Genenta Science, a clinical-stage biotechnology company pioneering the development of an investigational hematopoietic stem progenitor cell immuno-gene therapy for cancer (Temferon), will present new clinical data from a Phase I/IIa study of Temferon in patients affected by glioblastoma multiforme (GBM) in an oral presentation at the 2021 American Society for Gene and Cell Therapy (ASGCT) Annual Meeting, taking place virtually on May 11-14, 2021.

The data presented at ASGCT are from Genentas ongoing trial of Temferon in patients with GBM. The presentation focuses specifically on patients who have undergone a follow-up surgical procedure for their cancer. In addition to being a treatment option, follow-on surgery provides investigators with an opportunity to understand the impact of therapies at a cellular and molecular level.

The ASGCT presentation shows that genetic markers of Genentas Temferon were detectable in tumor specimens from all four patients with progressive disease who underwent follow-on surgery. Furthermore, the expression of interferon- (IFN) responsive gene signatures in those tumors was increased compared with pre-treatment levels, which suggests that interferon- (IFN-) had been released locally in the tumor by cells derived from Genentas investigational treatment.

Carlo Russo, Chief Medical Officer at Genenta Science, said: These preliminary results provide exciting indications that Temferon acts in the way we anticipated even in the relatively inaccessible setting of glioblastoma multiforme. The data are encouraging and in line with our pre-clinical results, with early evidence that Temferon delivers biological effects that may impact the progression of individual lesions.

One of the four patients had two lesions removed at the second surgery; one was a prior lesion that had not been removed during the first surgery and was stable; the other was a relapsing progressing lesion that had developed at the first surgery site. Compared with the progressing tumor, the stable lesion displayed a higher proportion of T cells and Tie2 Expressing Monocytes (TEMs) within the myeloid infiltrate and had a higher IFN-response signature.

The data presented at ASGCT also supported the initial safety and tolerability profile of Temferon. Concentrations of IFN- in the plasma and cerebrospinal fluid of patients remained low, while IFN- responses were identified in myeloid cells that infiltrate tumors. Temferon-derived differentiated cells also persisted in peripheral blood and bone marrow for up to 18 months at lower levels, indicating the potential durability of the intervention. No dose limiting toxicities have been identified.

Presentation Details:

Title: Changes in the Tumor Microenvironment in Patients with Glioblastoma Multiforme Treated with IFN-a Immune Cell & Gene Therapy (TEM-GBM_001 Study)

Time: Friday May 14, 2021 at 1.30 PM Eastern Time (7.30 PM CET)

Presenting: Carlo Russo, CMO

To access the abstract please visit https://annualmeeting.asgct.org/

About Genenta Science

Genenta (www.genenta.com) is a clinical-stage biotechnology company pioneering the development of a proprietary hematopoietic stem cell gene therapy for the treatment of a variety of cancers. Temferon is based on ex-vivo gene transfer into autologous hematopoietic stem/progenitor cells (HSPCs) to deliver immunomodulatory molecules directly via tumor-infiltrating monocytes/macrophages (Tie2 Expressing Monocytes - TEMs). Temferon, which is under investigation in a Phase I/IIa clinical trial in newly diagnosed glioblastoma multiforme patients, is not restricted to pre-selected tumor antigens nor type and has been designed to reach solid tumors, one of the main unresolved challenges in immuno-oncology. Genenta is based in Milan, Italy, and New York, USA.

About Glioblastoma MultiformeGlioblastoma multiforme (GBM) is a rapidly-growing cancer of the glial cells that support the nerve cells within the brain. The main treatment for GBM is surgery to reduce the bulk of the tumor, which can prolong the lives of patients and to improve quality of life. A second round of surgery is increasingly considered to have significant benefit in prolonging the lives of patients with GBM. Even with treatment, GBM virtually always recurs, typically resulting in death within the first 15 months from diagnosis.

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Genenta Phase I/II Glioblastoma Data at ASGCT Show Temferon Delivered Tumor-Focused Interferon ExpressionData presented at the 2021 American Society...

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Skin is the largest and heaviest organ of the body. It consists of three main layers; the epidermis, dermis, and hypodermis. Skin can either be thin or thick. The main difference is the thickness of the epidermis and dermis, which are the top two layers of skin.

Thin skin covers most of the body and can vary in thinness, with the thinnest skin covering the eyelids. Thick skin is present on the soles of the feet and palms of the hands.

In addition to differing thicknesses, the skin also differs in what is present in the layers. For example, thick skin has no hair follicles or sebaceous glands, whereas thin skin does.

In this article, we look at the differences in appearance, structure, and function of thin and thick skin.

Thin skin covers most of the body, except on the soles of the feet and palms of the hands, and contains fewer cellular layers than thick skin.

The epidermis of thin skin ranges from 0.070.15 millimeters (mm). Thin skin can vary in thickness in different parts of the body and is particularly thin across the eyelids. Thin skin is thickest on the upper back.

Thin skin also contains hair follicles, sweat glands, and sebaceous glands.

Thick skin is present on the soles of the feet and palms of the hands. This is because these areas receive more friction than other areas of the body, and thicker skin helps to protect from potential damage.

The epidermis of thick skin can be up to 1.5 mm. Thick skin does not contain any hair follicles or sebaceous glands. Thick skin also contains no arrector pili muscles, which cause goosebumps.

Thick skin is thicker due to it containing an extra layer in the epidermis, called the stratum lucidum. Thick skin actually has a thinner dermis layer than thin skin, but is still thicker due to the stratum lucidum layer present in the epidermis.

Thick and thin skin appear differently under a microscope. Thin skin contains four layers in the epidermis, while thick skin contains a fifth layer. These layers include:

The stratum basale, also known as the stratum germinativum, is the deepest layer of the epidermis. It is the layer just above the dermis.

This layer continuously produces new skin cells. It also contains melanocytes, which are cells that produce skin pigment and help protect the skin from sun damage.

The stratum spinosum consists of eight to ten layers of cells. People may refer to the stratum spinosum as the prickle cell layer because of the irregular structure of cells, which look like spines or prickles.

The stratum granulosum consists of three to five layers of cells. The stratum granulosum contains granules, which are rich in lipids.

Only thick skin contains the stratum lucidum layer. The stratum lucidum is a thin, transparent layer consisting of two to three layers of cells. It contains a protein called eleidin.

The stratum corneum is the upper layer of the epidermis. It consists of 2030 layers of cells. It contains keratin and horny scales, which make it tougher and able to thicken into calluses.

The stratum corneum contains dead keratinocytes, which produce defensins. Defensins are strings of amino acids that protect the body from infection.

Connecting the dermis and epidermis are structures called dermal papillae. Dermal papillae are more prominent in thick skin than thin skin.

Dermal papillae increase the surface area between the epidermis and dermis, allowing for more oxygen, food, and waste to pass between the layers.

The following table summarizes the key structural differences between thin and thick skin:

Skin in general has many different functions, such as protection, sensation, and thermoregulation. Both thin and thick skin have properties that allow the skin to function correctly.

For example, thin skin contains hair follicles, which are important in producing hair to help regulate temperature and protect from ultraviolet radiation. Hair follicles also provide epithelial stem cells, which help repair wounds.

In addition, thin skin contains sebaceous glands, which produce sebum. Sebum helps to lubricate the skin and protect against infections.

Thin skin also contains eccrine and apocrine sweat glands. Sweat glands help to regulate body temperature by releasing sweat to cool the body, and also help to repair skin damage.

Thick skin provides protection from damage in areas that experience more friction and abrasion, such as the palms of the hands and the soles of the feet. Thick skin also contains eccrine sweat glands to help regulate body temperature.

Skin is a large, complex organ with a wide range of vital roles. Thin skin and thick skin have different structures and functions in the body. The layers they contain provide their thickness and allow them to carry out their roles.

Thin skin is present on most of the body, and helps to protect against infections, regulate temperature, and allows hair to grow. Thick skin covers the palms of the hands and the soles of the feet and protects these areas from extra abrasion and friction.

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Thin skin vs. thick skin: What is the difference? - Medical News Today

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The Medical Skin Care Products Market report by Persistence Market Research throws light on the fact that the healthcare industry is more towards value-based care and continuous improvements based on the feedback. The mainstreaming of this practice is increasing all through. As such, the healthcare providers could make way for customized, lasting, and effective solutions to render utmost care to patients.

Medical skin care products are used for beautifying or to address some other skin care problems. The cosmetic industry is booming and skin care forms a very huge part of this industry. The aesthetic appearance is so important that people spend a lot on skin care products and treatment. People being more technologically aware of the various new skin care products trending in the market. In addition to the aesthetic application, the medical skin care products are also used to address issues such as acne, pimples or scars.

The medical skin care products is primarily driven by the need of natural based active ingredients products which are now trending in the market. Consumers demand medical skin care products which favor health and environment. Moreover, the consumers are updated with the trends so that various companies end up providing such products to satisfy the customers. For instance, a single product face mask has thousands of different variants. This offers consumers different options to select the product depending on the skin type. Moreover, the market players catering to the medical skin care products are offering products with advanced technologies. For instance, Santinov launched the CICABEL mask using stem cell material based on advanced technologies. The stem cells used in the skin care product helps to to protect and activate the cells and promote the proliferation of skin epidermal cells and the anagenesis of skin fibrosis.

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Medical Skin Care Products Market: Segmentation

On the basis of product type the medical skin care products market can be segmented as:

On the basis of application, the medical skin care products market can be segment as:

On the basis of distribution channel, the medical skin care products market can be segment as:

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Medical skin care products are used to address basic skin problems ranging from acne to scars. There are various advancements in the ingredients used to offer skin care products to the consumers. For instance, the use of hyaluronic acid and retinoids is the latest development in the industry. The anti-aging creams are at the forefront as the help treating issues such as wrinkles, scars, acne, and sun damage. Another, product in demand is the probiotic skincare which include lactobacillus and bifidobacterium.

In terms of geography, medical skin care products market has been divided into five regions including North- America, Asia- Pacific, Middle-East & Africa, Latin America and Europe. North America dominated the global medical skin care products market as international players are acquiring domestic companies to make their hold strong in the U.S. LOral is accelerating its U.S. market by signing a definitive agreement with Valeant Pharmaceuticals International Inc. to acquire CeraVe, AcneFree and Ambi skin-care brands for US$ 1.3 billion. The acquisition is expected LOreal to get hold of the brands in the price-accessible segment. Asia Pacific is expected to be the fastest growing region owing to the increasing disposable income and rising awareness towards the skin care products.

Some of the medical skin care products market participants are Avon Products Inc., Beiersdorf AG, Colgate-Palmolive Company, Kao Corporation, LOral S.A., Procter & Gamble, Shiseido Company, The Estee Lauder Companies Inc., Unilever PLC, Revlon, Clinique Laboratories, llc., Murad, LLC., SkinCeuticals, RMS Beauty, J.R. Watkins and 100% PURE.

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Global To Drive The Substantial Growth Of The Medical Skin Care Products Market KSU | The Sentinel Newspaper - KSU | The Sentinel Newspaper

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City University of Hong Kong (CityU) scientists have created a new microneedle patch to deliver cell therapies but rather than using traditional materials for their needles, they used ice.

The challenge: Cell therapies use living cells to treat medical conditions. Stem cell transplants are a form of cell therapy, as are some types of cancer immunotherapy.

These cells are typically transplanted into the patient via an implant, injection, or surgical graft. Not only can those delivery methods be painful and invasive, they also carry a risk of infection and must be administered by an experienced professional.

That limits the use of cell therapy to people who are willing to subject themselves to the transplantation process and who also have access to professionals capable of administering them.

Ice, ice baby: Microneedle patches are a growing trend in drug delivery. They're usually about the size of a postage stamp and are covered in tiny needles made of biodegradable substances packed with drugs.

Press the patch down on the skin like a band aid, and the needles break off from the back of the patch. They then dissolve into the skin, painlessly delivering the drug.

CityU created the microneedles for its patch out of ice, packed with living cells, coated in a protective medium.

Ice is easier to make and work with than the materials traditionally used for dissolving micropatches, but it melts just as readily. Even better, the icy microneedles can preserve the viability of living cells something other types of patches can't do.

The freezing cold water: Because the microneedles are made of ice, they would have to be transported and stored frozen, which could be a limiting factor in some places.

Additionally, the icy microneedle patch performed well when used to deliver a cell therapy to mice as a proof of concept, but it still needs to be proven safe and effective in humans.

What's next: If CityU's microneedle patch is cleared for use in people, it could have applications even beyond cell therapy.

"This device can also package, store, and deliver DNA, vaccines, and more."

"This device can also package, store, and deliver other types of bioactive therapeutic agents, such as proteins, peptides, mRNA, DNA, and vaccines," lead researcher Xu Chenjie said in a press release.

"I hope this device offers an easy-to-use and effective alternative method for the delivery of therapeutics in clinics."

We'd love to hear from you! If you have a comment about this article or if you have a tip for a future Freethink story, please email us at [emailprotected].

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Icy Microneedle Patch Delivers Cell Therapy, Then Melts - Freethink

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During a virtual Targeted Oncology Case-Based event, Pashna N. Munshi, MD, associate clinical director, Stem Cell Transplant and Cellular Immunotherapy Program, assistant professor of Medicine, Georgetown University School of Medicine at MedStar Georgetown University Hospital, discussed the case of a 48-year-old male patient with acute graft-versus-host-disease (GVHD).

Targeted OncologyTM: What factors contribute to the risk of acute GVHD in a patient like this one?

MUNSHI: A lot of donor-recipient factors and other conditions increase the risk of acute GVHD. [These include] gender matching, human leukocyte antigen disparity, degree of mismatch, and having an older donor. Theres also [blood group] incompatibility and definitely CMV mismatched status. Though now that the FDA has approved letermovir [Prevymis] for patients who are undergoing allogeneic transplant if they have a CMV-positive donor,1 were seeing very little CMV reactivation. That has been a bit of a game changer for the good.

Patients have an increased risk of GVHD if they receive a transplant from a peripheral blood stem cell source versus a bone marrow graft, because the peripheral blood has more T cells in its composition. The myeloablative regimens [are associated with greater risk of GVHD than] reduced-intensity regimens.2

Do you agree with these poll results? Would you start with systemic therapy for this patient?

It can get a little tricky whether you want to give patients systemic steroids or wait and see if something gentler might work. I tend to agree that, at this point, the patient needs to immediately start with systemic steroids, because there are 2 organ systems involved. Once the lower gastrointestinal [GI] tract gets involved, it surely portends a poor prognosis if the grade becomes worse. And they become refractory to steroids very quickly: 50% of these patients will eventually not respond to steroids.

How would you stage this patients GVHD?

There are many criteria for staging GVHD. The criteria that most clinical trials use are the Mount Sinai Acute GVHD International Consortium [MAGIC] criteria.3 They are adapted from the Glucksberg criteria, which are very similar.4

Three organ systems [are involved in] acute GVHD: the skin, the liver, and the GI tract. Skin involvement is graded on the basis of the body surface area involved. Liver involvement is graded on the basis of the total bilirubin level. Upper GI involvement is graded on the basis of anorexia, nausea, and vomiting, and it just comes in stage 0 or stage I, depending on if its persistent or not. To determine lower GI tract involvement, we measure stool volume, especially when patients are admitted to the hospital. But once they go home, we cant do that, so we ask them how many times a day they have diarrhea. Is it watery? Is it muddy? Whats the volume? Is it large or small?

The patient can characterize the stool and tell their doctor how many times per day: 4 times, 5 times, 6 times. This patient is having 4 episodes per day; that puts them in stage I lower GI GVHD. But with a 60% body rash, that puts them in stage III skin GVHD. So really getting up there with skin, but not so much yet for GI. Once each organ [involvement is] staged, theres an aggregate score based on the combination of these organs. Then we come up with the grade.

In this patient, with a stage III rash, stage I upper GI, and stage I lower GI GVHD, they have a total score of a grade 2 acute GVHD. This is still in the mild to moderate zone. Anything above grade 2 is considered very severe GVHD.

Would you recommend that this patient receive systemic steroids?

In the scheme of things, somebody who didnt have symptoms and now is having active symptoms, especially with lower GI tract involvement, definitely needs high-dose steroids to get in there and [stop] the inflammation.

On what would you base a prognosis for this patient?

We can risk stratify these patients on the basis of the stage of organ involvement.5 Broadly, they can be at a standard risk or at a high risk [of poor response to treatment, mortality, and transplant-related mortality]. The patient is at high risk once they have very active GI involvement [or] if they have 2 organs involved. This is one more reason to think about starting these patients early on steroids. Why is this important? Because once a patient has high-risk GVHD, the chance of response to steroids is even lower, and once they dont respond to steroids, there is a higher [risk of] transplant-related mortality. The probability of transplant-related mortality is 44% for patients with high-risk acute GVHD flares, [versus] 22% for patients with low-risk GVHD [P < .001]. These are a few things to think about. Act very swiftly if a patient has 2-organ involvement, especially the lower GI tract.

Can biomarkers guide treatment decisions in this case?

In the field of GVHD, biomarkers are a very exciting advancement. We want a prognostic model of which patients will get GVHD. Can biomarkers in the blood [help] prevent GVHD and improve transplant outcomes?

A large prospective trial was done through the Bone and Marrow Transplant Clinical Trials Network where a set of 6 biomarkers were tested at several time points after the transplant.6 They saw that they could predict when GVHD happened by using these biomarkers. They could see that as the levels of these biomarkers increased, the patients had higher scores of GVHD. Once treatment was started, if specific biomarkers went down it was predictive of response at day 28 [56% vs 17%; odds ratio, 6.32; P = .001] and also predictive of [decreased] transplant-related mortality by day [180 (49% vs 87%; P < .0001)]. If all these biomarkers went up aggressively, overall survival was lower [P < .0001].

The MAGIC Consortium also tried to test biomarkers.7 They looked at 2 biomarkers, REG3Athe regenerating islet-derived 3-alpha, which is specific for the GI tract and ST2. Looking at these 2 biomarkers, they came up with an algorithm of prediction. On the basis of how these biomarkers responded at the time of GVHD and to treatment, they could predict mortality by 6 months. In clinical practice, it is difficult to use this day in and day out. We still use our clinical skills to assess the degree of GVHD. But all patients eventually get treated the same waywith high-dose steroidsdespite biomarkers being elevated or not.

At this point, [biomarker data] may tell us an association rather than a causality. Were not openly using biomarkers to guide our practice, but I think were learning to use them a bit more and knowing that theres something out there that could be used as a predictive tool. It is an exciting development.

Are there alternatives to systemic steroids?

Steroids remain the mainstay. We need to see if we can move to other therapies that are coming down the pipeline.

Data from the REACH1 [NCT02953678] and REACH2 [NCT02913261] trials led to ruxolitinib [Jakafi] approval.8,9 If we can use ruxolitinib in an up-front setting, [maybe we] can use the newly approved rho-kinase or ROCK2 inhibitors as well.10 We want to think about steroid-sparing agents. Maybe biomarkers can guide us in the future for that. But right now, in terms of, Do I start my patient on treatment? or Will they respond to this treatment, I find that [biomarkers are] still not a very useful tool because at the end of the day, the patients all still need to be started on steroids.

The minute you see that your patient is not responding to steroids, very quickly start them on a JAK2 inhibitor.

How do you dose steroids?

This patient received 2 mg/kg of prednisone per day for 14 days. Two mg/kg is a very high dose. The standard is 1 to 2 mg/kg.11 There are data to show that 2 mg isnt any different from 1 mg.12 But a lot of times, if its a very active, severe flare, we will use 2 mg/kg. Im not sure if I would have done 2 mg/kg in this case, but its certainly not out of the realm of treating these patients.

The goals of primary therapy for acute GVHD are to stabilize the organ manifestations, or improve them, and limit long-term treatment toxicity. We want to improve functional capacity and prevent any reduction in quality of life. First-line therapy is always with corticosteroids. Now ruxolitinib is approved for second-line therapy.8 There have been data to show that it can improve overall survival.

How do you taper glucocorticosteroids after achieving initial response?

If the patient is taking 2 mg/kg of steroids, an average 70-kg person, thats over 100 mg of steroids. After 2 weeks, they probably are not getting up from a seated position anymore with all the muscle wasting that can happen.

[As soon as they start to show improvement, it would be safe to start to taper the dose.] Traditionally, [the patient receives the full dose for] at least a week or 10 days. Then it is traditional to decrease the dose 10% every 5 to 7 days, gently coming down, making sure that the patient is not having any flares.

Describe the multidisciplinary teambased approach that you use for acute GVHD.

The incidence of acute GVHD in the patient population is anywhere from 30% to 50%, despite the best [efforts at] prophylaxis. Most patients will get some form of acute GVHDit can go up to even 80%. This [necessitates] a multidisciplinary team approach. If the patient is having diarrhea, theyre having malnourishment. Theres nausea or anorexia, so theyre not eating on top of that. Then theres skin rash, so the risk of infections and cellulitis. Theyre in pain. A dermatologist probably should be involved at some point. A nutrition team is also needed. If theyre on high-dose steroids, physical therapy should be involved up front. So early involvement of a whole team is very important. Thats usually how I treat my patients and usually how centers of excellence continue to treat active patients with GVHD after transplantation.

How do you determine if a patients GVHD is steroid refractory?

The strict definition of steroid refractoriness or resistance is if theres progression of acute GVHD within 3 to 5 days of starting high-dose steroids, or theres failure to improve within 1 week of starting these steroids, or theres incomplete response after more than 28 days of any immunosuppressive treatment.13 So, by and large, in 3 days or a maximum of 7 days, [it will be clear] if the patients GVHD is going to be steroid refractory or not.

Steroid dependence is [defined as when] the patients GVHD initially responded to steroids, but the disease flares when the dose is tapered, so they cannot be taken off the steroids.

Steroid intolerance is when the patient develops [unacceptable toxicity from steroids such as] uncontrolled diabetes or myopathies. Then it becomes hard to keep them on steroids.

What are the treatment options for patients with steroid-refractory GVHD?

Ruxolitinib now has been FDA approved for steroid-refractory acute GVHD, and its a category 1 definition.8,11 Ibrutinib [Imbruvica] has also been approvedits only FDA-approved indication is for chronic GVHD.14 There are many other treatment options [in the National Comprehensive Cancer Network guidelines].11 Oncologists always end up using some combination or other depending on which of these different immune suppression medications they are comfortable using.

What new treatments are in the pipeline?

In terms of BTK inhibitors, I dont think theres anything other than ibrutinib at this time point. There are many JAK inhibitors being studied.15 Baricitinib is another JAK inhibitor thats actively being studied for chronic GVHD, as well as for pulmonary GVHD.16 Then there are other rho-kinase inhibitors, called ROCK2 inhibitors. This is really making waves. Were very excited about this drug because the response rates are very high, about 70%.10 Its a smaller study, but clearly it has antifibrotic pathways. So I think thats going to be used much more in the up-front setting.

Then theres also alpha-1 antitrypsin, which targets the liver and macrophages and has very promising results from trials done at Dana-Farber Cancer Institute and Michigan.17 So I think were going to see very different characteristics of how to approach GVHD.

What data support the use of ruxolitinib in this setting?

The REACH1 study led to the approval of ruxolitinib for steroid-refractory acute GVHD.9,18 In this phase 2 trial, patients with steroid-refractory acute GVHD got ruxolitinib (5 mg twice a day) with or without a calcineurin inhibitor. They were allowed to remain on steroids. The primary end point of this trial was overall response rate [ORR] at day 28. They also looked at response rates at day 56 and day 100, biomarkers, failure-free survival, and durability of these responses. The ORR at day 28 was very high: 54.9%.18 The best ORR, which was at any given time during the treatment, which was as high as 73.2%. The median time to response was 7 days. So this was very quick. The median duration of response was 345 days, with more than 6 months follow-up. Nonrelapse mortality at 6 months was 44.4%. There were deaths from infections, etc, but not related directly to ruxolitinib.

Subsequently there was a phase 3 trial, REACH2.19 They looked at higher doses of ruxolitinib in steroid-refractory acute GVHD. They started off with 10 mg [of ruxolitinib] twice a day. This study had a similar primary end point of ORR at day 28. This was compared with best available therapy. This was done in Europe, so [the comparison was to the] best available therapy used in Europe, like anti-thymocyte globulin, sirolimus [Rapamune], etanercept [Enbrel], photopheresis, or other therapies; all things that we would use in the United States as well. They looked at similar key secondary end points, [including] duration of response at day 56.

The ORR for ruxolitinib was 62% at day 28, compared with the best available therapy arm, which was 39% [odds ratio, 2.64; 95% CI, 1.65 to 4.22; P < .001].19 Durable overall response at day 56 [was higher in the ruxolitinib group than it was in the control group (40% vs 22%, odds ratio, 2.38; 95% CI, 1.43-3.94; P < .001)].19

The lower grade acute GVHD, which was grade 2, had the highest complete response rate with ruxolitinib: 50.9% compared with just 26.4% with best available therapy.19 This is quite remarkable to have a complete response in GVHD so quickly. When you get to higher grades of GVHD, the complete response rate for ruxolitinib is not as impressive; its less than 30%. But its still much higher than the [response rates of] other therapies we would have otherwise treated these patients with in steroid-refractory disease. The key point is to diagnose steroid refractoriness early. Then get ruxolitinib in there to break the cycle and break the progression of organ grade to something higher.

The loss of response wasnt statistically significant. The estimated cumulative incidents for the loss of response at 6 months was 10% in ruxolitinib compared with 39% in the control arm.19 So patients continued to maintain responses, which, again, is what we want to see. We dont want to see flares if they come off steroids.

[Of the 4 organ systems involved in GVHD], the skin responses were the best with ruxolitinib. Lower GI and liver GVHD did have good responses, but the responses were not as remarkable. Ruxolitinib is an ideal drug in this setting, on the basis of the organ responses.

A secondary end point was failure-free survival, basically indicating a time point from randomization to either nonrelapse-related death or any new GVHD. This was not statistically significant because it was not designed to compare ruxolitinib survival outcomes with control therapy. But there were 5.0 months median failure-free survival with ruxolitinib compared with 1.0 month with control [hazard ratio for relapse or progression of hematologic disease, nonrelapse-related death, or addition of new systemic therapy for acute GVHD, 0.46; 95% CI, 0.35-0.60]. That tells you that the responses were maintained, and the treatment was still working.

[Most of the adverse events associated with ruxolitinib] were expected; the bone marrow is recovering so its a bit fragile. [The most common was] thrombocytopenia. You can reduce the dose of ruxolitinib down to 5 mg adjusted accordingly or support patients with transfusions. CMV reactivation was also common. But again, with letermovir, that happens less and less.

References:1. Merck receives FDA approval of Prevymis (letermovir) for prevention of cytomegalovirus (CMV) infection and disease in adult allogeneic stem cell transplant patients. News release. Merck. November 9, 2017. Accessed April 7, 2021. https://bit.ly/3fS6S0Q

2. Scott BL. Long-term follow up of BMT CTN 0901, a randomized phase 3 trial comparing myeloablative (MAC) to reduced intensity conditioning (RIC) prior to hematopoietic cell transplantation (HCT) for acute myeloid leukemia (AML) or myelodysplasia (MDS) (MAvRIC Trial). Biol Blood Marrow Transplant. 2020;26(3):S11. doi:10.1016/j.bbmt.2019.12.07

3. Harris AC, Young R, Devine S, et al. International, multicenter standardization of acute graft-versus-host disease clinical data collection: a report from the Mount Sinai Acute GVHD International Consortium. Biol Blood Marrow Transplant. 2016;22(1):4-10. doi:10.1016/j.bbmt.2015.09.001

4. Martino R, Romero P, Subira M, et al. Comparison of the classic Glucksberg criteria and the IBMTR Severity Index for grading acute graft-versus-host disease following HLA-identical sibling stem cell transplantation. International Bone Marrow

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South Korea and Japan have given the beauty world many gifts cleansing balms, glass-skin serums, inventive masks for making skin gleam. And at this time of unprecedented violence against Asians in this country, the Allure family is more passionate than ever about championing Asian innovation and Asian voices. We will speak out against racism and intolerance and do more than ever to support Asian-owned beauty brands, starting with this special edition of the Allure Beauty Box. Inside are some of the most influential K-beauty and J-beauty products of all time ones that have revolutionized our editors routines and the entire beauty industry. Sulwhasoo First Care Activating Serum is an innovative prep step for glowing skin (one bottle is sold every 10 seconds worldwide) and AHC Essential Real Eye Cream for Face, a unique eye and face cream hybrid, is cherished in South Korea (where one is sold every three seconds.) We hope they become as beloved by you as they are by us! And if you would like to get involved in the movement to Stop Asian Hate, you can also join Allure editors in donating to the Asian American Legal Defense and Education Fund and to GoFundMe's AAPI Community Fund to Stop Asian Hate at gofundme.com/aapi.

- The editors of Allure

You can find all these products and more in this limited-edition box. For just $60 (or $40 exclusively for Allure Beauty Box members), you'll get ten of our favorite J-Beauty and K-Beauty products, worth over $200 in value. Quantities are limited, so order now to take advantage of this exclusive offer before they're sold out.

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Animal cloning has come a long way from Dolly the sheep, but all through that time the ethical and scientific repercussions only continue to pile up.

One thing is getting clearer though: The cloning process has improved by leaps and bounds (though not yet on the levels shown in science-fiction). This has resulted in headways in a number of cloning-related endeavors, from the revival of endangered (and even extinct) species to replicating dead pets.

Still, both critics and proponents are still a bit out of touch with regards to how current cloning tech is being used to address these issues.

There is some truth to the hype that bringing back the woolly mammoth via cloning could be in the not-so-distant future.

On the other hand, many critics question the wisdom of bringing back prehistoric animals to habitats that have long changed from their disappearance. Still, this ignores the possibility of restoring more recently extinct species and how cloning could counteract such drastic environmental changes from their loss.

Another popular argument against cloning is the idea that its novelty and high costs could be redirected to more natural methods of conservation. But while that may be true for a majority of endangered species, it may not be so for those that have been officially declared extinct in the wild.

Cloning could be an important tool in ensuring the genetic diversity that allows populations in captivity to grow (and make reintroduction more feasible).

The idea of cloning animals that retain a set of desirable traits has raised considerable alarm (especially among more conservative groups). However, there are benefits to the practice that cannot be ignored.

Replicating the sharp noses of bomb sniffer (or even disease sniffer) dogs could make a difference in times of need. Likewise, cloning cattle with more consistent yields of milk and meat could have applications for more efficient livestock farming.

Also read: Trained Bees Can Identify COVID-19 Infection Through Sense of Smell Within Seconds!

Now, despite the advances, today's cloning technology is still decades away from producing perfect, one-for-one genetic copies of an original animal. Differences (even large ones) can still be found. There is also the fact that environment, behavior and upbringing could still drastically alter the genetic makeup of a cloned animal (as the study of epigenetics will gradually reveal).

Throwing a bit of caution to the wind will also be important if the increase of cloned designer animals could lead to other adverse effects on the global environment (especially in the feeding of quality livestock). The same applies to the use of clones to restore and reintroduce critically endangered species.

Overall, the bounds of the technology's research has expanded considerably (and so has the conversation). But at the same time, it is important to have a strong sense of moderation regarding its application. It has the potential of causing problems and incurring needless costs, but these should not discourage future research on cloning's potential.

Also read: Snakes Can Store Sperm for up to 5 Years Before Getting Pregnant

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CARLSBAD, Calif.--(BUSINESS WIRE)--Accelerated Biosciences, a regenerative medicine innovator, announced today new data that further demonstrates statistically significant cytolysis with induced pluripotent stem cell (iPSC)-derived natural killer (NK) cells programmed from its ethically sourced human trophoblast stem cells (hTSCs). Pluristyx, a Seattle-based firm supporting drug development, regenerative medicine, and cell and gene therapies, further confirmed Accelerated Biosciences hTSC line offers before-unrealized opportunities in cell-specific therapeutics. Along with this recent data on successful iPSC differentiation, Accelerated Biosciences has already demonstrated efficient differentiation of its pluripotent stem cells with remarkable doubling times and growth characteristics to programmed NK, cartilage, bone, fat, neuron, pancreas, liver, and secretome cells.

This new data validates our findings, explains Yuta Lee, President and Founder of Accelerated Biosciences. We know the properties of our trophoblast stem cells have been long-sought by the medical science community because of the potential to speed and amplify the development of life-saving therapeutics; theyre immune privileged, chromosomally stable (not tumorigenic), pathogen free, pluripotent, easy to scale and manufacturer, and of special interest, they are ethically sourced from the chorionic villi (pre-placental tissue) of non-viable and often life-threatening tubal ectopic pregnancies. Mr. Lees father, Professor Jau-Nan Lee, MB, MD, PhD, an obstetrics and gynecologic physician and researcher in Taiwan, first isolated hTSC in 2003. Mr. Lee created Accelerated Biosciences to elevate the visibility of this pluripotent human trophoblast stem cell platform to those engaged in developing allogeneic cell therapeutics and has been instrumental in the filing and prosecution of intellectual property to protect the companys hTSC platform to date holding 34 patents.

Benjamin Fryer, PhD, Co-founder and CEO of Pluristyx, worked closely with Accelerated Biosciences to prepare much of its key hTSC data. Dr. Fryer, a trophoblast expert who was previously a research scientist at Janssen Research & Development of Johnson & Johnson, now serves on Accelerated Biosciences Scientific Advisory Board. Initially I was skeptical these cells were what they said they were. If we hadnt grown and characterized them in our lab, I might have remained skeptical. These are indeed trophoblast stem cells, explained Dr. Fryer. The potential of these cells is enormous. One of the industrys largest challenges is that its almost impossible to scale primary cells. These cells are scalable. With these cells you can make the amount required for millions of patients and theyre sourced compliant to regulatory requirements. Weve made IPS cells (induced pluripotent stem cells) and NK (natural killer) cells from them, which is the next wave of cells for cell therapies. For therapeutic developers, because these cells are not sourced from a person or viable embryo, these cells deliver the trifecta of legal, ethical, and IP advantages.

As the biotechnology industry works toward developing therapies that target only diseased cells without harming healthy cells and tissues, cell-based therapies draw increasing interest, explains industry expert, Martina Molsbergen, CEO of C14 Consulting, who has partnered with Accelerated Biosciences in a business development role. With all the promise that cell therapies hold, the biotechnology industry also remains concerned that the therapeutics are derived in a socially and ethically responsible manner. Accelerated Biosciences has discovered and is now offering what scientists see as the holy grail of stem cell sources.

Prominent biosciences experts have been drawn to Accelerated Biosciences cell breakthrough. Protein chemist and molecular biologist Igor Fisch, PhD, former President and CEO of Selexis, Geneva, Switzerland, recognizes the impact that Accelerated Biosciences hTSCs will have on human health: Not only are these cells politically correct, but they can also differentiate. Because they are sourced from pre-placenta material, theyre immune privileged, which means that are not seen as foreign by the human body. With these cells, we can create a cell bank a single source for a wide range of patients.

Peter Hudson, FTSE, BSc Hons, PhD, Chief Scientist and a senior advisor to Avipep P/L in Melbourne, Australia, and an adjunct professor at the University of Queensland, led a large oncology consortium to complete the first Phase 1 clinical trial of a novel engineered antibody targeting prostate and ovarian cancer. Hudsons interest in Accelerated Biosciences hTSCs has evolved into a role on its Scientific Advisory Board. Trophoblast stem cells are likely to be the next wave of cancer-targeting therapeutics, explains Dr. Hudson. The ability to ethically source trophoblast stem cells and program them to target only diseased, cancerous cells is very powerful technology.

Why are scientists so interested in stem cell-based therapies?

The human body constantly produces specialized cells from its own stem cells (undifferentiated cells) to renew and repair itself. Current therapies harness this power in autologous cell therapies in which the patients own cells are removed, differentiated into disease-fighting cells, and reinserted.

What makes the human trophoblast stem cell so important to medical science?

The human trophoblast stem cell (hTSC) comes from placental tissue and has special properties that make it extremely desirable to therapeutic developers. The hTSC is such an early stem cell that it has much more capacity for growth than a stem cell taken from an adult, for example. This means that one cell can become millions. The hTSC also carries with it the same immune-privilege that a growing embryo has inside its mother: its not seen as foreign although its genetically different than its mother. Unlike other foreign materials, the hTSC is not rejected by the human body, which means that it can be used with many different patients (allogeneic cell therapy). With these benefits, the scientific community holds a high regard for hTSCs, but it also faces socio-ethical concerns about how those stem cells are typically sourced.

Accelerated Biosciences sidesteps hTSC sourcing concerns in a profoundly elegant way. Dr. Jau-Nan Lee, an OB-GYN in Taiwan, found inspiration in what was considered medical waste. When surgical intervention was necessary to remove an ectopic pregnancy that would otherwise risk the womans life, the non-viable embryo and pre-placental tissue lodged in the fallopian tube was removed, sent to pathology, and discarded. Gaining permission from institutional colleagues and sampling the pre-placental tissue, Dr. Lee isolated hTSC that offered all the benefits of hTSC pluripotency, immune privilege, and scalability without pathogens and without ethical compromises.

About Accelerated Biosciences

Founded in 2013, Accelerated Biosciences is a private company focused on regenerative medicine and built around the hTSC discoveries of obstetrics and gynecology physician and researcher, Professor Jau-Nan Lee, MB, MD, PhD. Accelerated Biosciences holds a large and robust patent portfolio and an encumbrance-free intellectual property (IP) estate. Accelerated Biosciences mission is to leverage its renewable, immune-privileged human cell source to fuel breakthrough cell therapies that effectively target the most challenging diseases of the human body. For more information about Accelerated Biosciences, visit acceleratedbio.com or email mmolsbergen@c14consultinggroup.com.

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Accelerated Biosciences' Immune-Privileged Human Trophoblast Stem Cells (hTSCs) Offer Breakthrough Opportunities in Cancer-Targeting Therapeutics and...

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Factor reveals advances in mRNA, circleRNA, gene editing, cell reprogramming, and iPS cell-derived NK-cell technologies.

Digital presentations will be made available on the ASGCT website on May 11, 2021. For more information on the upcoming American Society of Genetic & Cell Therapy (ASGCT) Annual Meeting, visit https://annualmeeting.asgct.org/

About Factor BioscienceFounded in 2011, Factor Bioscience develops technologies for engineering cells to advance the study and treatment of disease. Factor collaborates with academic and industrial partners to develop therapeutic products based on its mRNA, gene editing, cell reprogramming, and nucleic-acid delivery technologies. Factor Bioscience is privately held and is headquartered in Cambridge, Massachusetts. For more information, visit http://www.factorbio.com.

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Factor Bioscience to Deliver Six Digital Presentations at the American Society of Gene & Cell Therapy (ASGCT) 24th Annual Meeting - PRNewswire

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Small dreams have no power to move hearts, and in a new six-year strategic plan, St. Jude Childrens Research Hospital is thinking very big.

What would it take to drastically increase cure rates for childhood cancer worldwide?

St. Judes answer: $11.5 billion and an additional 1,400 jobs.

To get a rough sense of scale, work it out with a pencil:

Spread over six yearsat $1.916 billion each yearits just under a third of the NCIs annual spend, fourfold this years projected revenues of the American Cancer Society, and more than seventyfold the budget of the World Health Organizations International Agency for Research on Cancer.

Its a broad and ambitious plan that will allow the institution to grow at an almost 8% compound annual growth rate, James Downing, president and CEO of St. Jude, said to The Cancer Letter.

At a global level, we also want to see identifiable increases in cure rates. We are watching those very carefully. Our goal is to move toward cure rates of 60% for diseases like acute lymphoblastic leukemia, Hodgkins lymphoma and Wilms tumor, Downing said. As we look at a global population, survival rates hover around 20%, and wed like to see those go up year by year.

A lot of our efforts are based on implementation science, looking at what works and what doesnt work. Workforce, drug distribution and true advancements in cure rates are what were seeking over the next six years.

The plan, rolled out on April 27, calls for an acceleration of research and treatment globallynot just for pediatric cancer, but also other illnesses, including blood disorders, neurological diseases, and infectious diseases.

Not surprisingly, this amount represents the largest investment the Memphis, Tenn. hospital has made in its nearly 60-year history. The previous strategic plan, the largest expansion in the institutions history, resulted in $7 billion in investments (The Cancer Letter, May 19, 2017).

The multi-phase expansion plan is funded almost entirely by steadily increasing donor contributions generated by ALSAC, the fundraising and awareness organization for St. Jude.

It is an ambitious plan. But were going to have lots of new personnel, new investments, new technology and new partnerships. We have formal partnerships with many U.N. associate agencies and organizations around the world.

Within the past six years, St. Jude has advanced fundamental, clinical, and translational research, Downing said.

Two years ago, we began strategically looking at the most pressing issues in the field of pediatric cancer, Downing said. As we developed the strategic plan over those two years, there were many ideas we critically assessed, and we often said, Its not really best for St. Jude to pursue that.

In the end, we aligned on goals that collectively bring the prospect of remarkable benefits to the field of childhood cancer, and to children with cancer everywhere.

On campus, St. Jude accepted nearly 20% more new cancer patients; increased faculty by 30% and staff by 23%; and embarked on several large-scale construction projects.

The new strategic plan focuses on five areas: fundamental science, childhood cancer, pediatric catastrophic diseases, global impact, and workforce and workplace culture.

Were coming out of a six-year strategic plan in which we increased our number of cancer patients by 20%, with 30% new faculty, 23% more staff, many large-scale construction projects, said Charles Roberts, executive vice president of St. Jude and director of the hospitals Comprehensive Cancer Center. And were now going into a new strategic plan that is 60% larger than our prior plan.

Under the plan, St. Jude will hire nearly 70 new faculty members, plus supporting laboratory staff, to work in basic, translational, and clinical research across 22 departments.

These investigators will have the freedom to pursue the type of conceptually driven research that leads to tomorrows clinical advances.

As we launch a strategic plan, weve identified the most exciting opportunities and challenges at that point in time, Roberts said to The Cancer Letter. However, we fully realize that we dont know whats coming next. New discoveries will be made, and new opportunities will emerge. Via the blue-sky process, weve set aside substantial funds every year to invest in the pursuit of emerging opportunities suggested by faculty and staff.

Part of what brought me here from Boston was the last strategic plan, and its so exciting to be a part of this. But just looking at the numbers, 1,400 new positions, average salary of $90,000. Six hundred and forty of those positions are in research, 266 are in clinical, 100 are in global pediatric medicine, and 394 in support.

Those are the kinds of numbers that you need to make these things real, and I think its exciting for St. Jude and for the field of cancer research, as we bring in all of these new folks.

During the next six years, St. Jude will invest more than $250 million to expand technology and resources available to scientists and clinicians in their search to understand why pediatric catastrophic diseases arise, spread and resist treatments. These investments will include:

St. Jude will invest $3.7 billion during the next six years to expand cancer-focused research and related clinical care. These efforts will center on raising survival rates for the highest-risk cancers and for children with relapsed diseases, while simultaneously improving quality of life for pediatric cancer survivors. The investments will include:

In the U.S., more than 80% of children diagnosed with cancer will be cured. In contrast, 80% of children with cancer live in limited-resource countries, where a mere 20% survive their disease. To address this, St. Jude will more than triple its investment in its international efforts coordinated through St. Jude Global and the St. Jude Global Alliance during the next six years.

This represents an investment of more than $470 million. Global initiatives include:

Under the plan, St. Jude will expand research and treatment programs to advance cures for childhood catastrophic diseases. The $1.1 billion, six-year investment includes work in nonmalignant hematological diseases, such as sickle cell disease; a new laboratory-based research program in infectious diseases that affect children worldwide; and a new research and clinical program to better understand and treat pediatric neurological diseases.

The plan outlines several strategies to encourage teamwork, and internal and external collaboration. These will include:

It is estimated that 87% of funds to sustain and grow St. Jude over the next six years will come from public donations.

Patients at St. Jude do not receive a bill for treatment, travel, housing or fooda model established by ALSAC and St. Jude founder Danny Thomas, who believed in equal access to medical care and driving research advances.

There are an incredible number of donors across the United States who support St. Jude, Downing said. This carries a great responsibility for us to seek the maximum possible impact to improve outcomes for childhood cancer.

Downing and Roberts spoke with Matthew Ong, associate editor of The Cancer Letter.

Matthew Ong: Congratulations on the official launch of St. Judes second six-year strategic plan. Could you briefly walk us through whats in it?

James Downing: It is an exciting time for St. Jude Childrens Research Hospital. Were finishing our prior six-year strategic plan, which started in Fiscal Year 2016. That $7 billion investment in the organization spanned fundamental science, clinical and translational research, clinical operations, and our global efforts. During the course of the plan, we increased faculty by 30% and staff by 23% and accelerated progress against pediatric catastrophic diseases.

About two years ago, we started discussing the next strategic plan. We looked critically at what we had accomplished under the previous plan, the expertise we had assembled, and the major problems in the field of pediatric catastrophic diseases, including cancer, infectious diseases, nonmalignant hematologic diseases and pediatric neurologic diseases. During that period, we involved more than 200 individuals across the institution to develop the new strategic plan.

This plan, at its core, focuses on accelerating progress against pediatric catastrophic diseases on a global scale. It outlines a $11.5 billion investment during the next six years, which includes the addition of 1,400 jobs and $1.9 billion in new capital investments, construction and renovations. Its a broad and ambitious plan that will allow the institution to grow at an almost 8% compound annual growth rate.

The plan has 11 goals, divided among five major areas: fundamental science, pediatric cancer, other childhood catastrophic diseases, global impact, and a focus on people and place. In each of these areas, were recruiting new individuals, investing in new technology, and expanding collaborations across campus, across the United States, and globally.

Ill start with fundamental science. In our last strategic plan, we invested heavily in increasing basic science programs on campus by expanding faculty numbers, technology and institutional data infrastructurein the belief that expanding fundamental science leads to new knowledge that helps advance cures. This is investigator-initiated science, often not related to diseases, but rather to the fundamental questions of biology.

In this new plan, were again investing heavily in expanding fundamental science at St. Jude. Weve committed more than $1 billion to fundamental science. This includes increasing laboratory-based faculty by more than 33% during the next six years, and more than $250 million dedicated to investments in technology.

Our goal is to make sure every dollar is spent wisely and effectively in pursuit of our missionto advance cures and means of prevention for pediatric catastrophic diseases through research and treatment.

The $250 million will fund multiple shared resources, department-based technology centers and new centers of excellence. Some of the faculty are being recruited to the centers of excellence, including those in data-driven discovery, innate immunity and inflammation, leukemia and advanced microscopy. These individuals will also have homes in academic departments.

On the technology front, were investing in shared resources. Well bring online some new ones, as well as some (Center for Modeling Pediatric Diseases and the Center for High-Content Screening) created at the end of the last strategic plan. The newest is focused on spatial transcriptomics. It will allow scientists across campus to look at the expression of genes in tissue context and at the single-cell level.

A new effort in structural biology is to create a $20 million Cryo-Electron Tomography Center. This is the next level of cryo-electron microscopy, which allows the identification of the structure of molecules or molecular machines within the context of cells. Its a developing technology that will feed other investments weve made in structural biology, such as the installation of one of the largest magnets in the world in our NMR facility, our Cryo-Electron Microscopy Center and single-cell analysis capabilities. The plan brings those tools to bear on defining normal biology and disease states.

Another effort is a Center of Excellence in Advanced Microscopy. Over the last six years, weve become one of the leading centers in the application of advanced microscopy to fundamental biology. This has been led by investigators in our Developmental Neurobiology, Cell and Molecular Biology, and Immunology departments.

Were positioned to build the next generation of microscopes to explore biology in ways never dreamed. With new faculty recruitments and collaborations with commercial companies and other institutions, we seek to develop the next generation of microscopes, and apply that to normal biology and to pediatric catastrophic diseases.

Another area were investing in heavily is data science. Over the last six years, and even before that, we expanded data sciences across campus. This initially started with the Pediatric Cancer Genome Project in 2010. Since then, we recruited many data scientists, and coalesced them into our Computational Biology, Biostatistics, and Epidemiology and Cancer Control departments, and into shared resources that provide bioinformatics support.

But over the last several years, weve seen the explosion of data, from structural biology to microscopy.

As we look to the future and the capabilities weve amassed, were poised to significantly increase our investment in data science and become a leading institution in the application of data science to biologic discovery. This is a $40 million investment with 30 full-time employees.

We have a task force led by faculty members to develop the roadmap for how were going to move forward. As data is accumulated and we look across those disparate data types, we can gain knowledge through true data scienceexploring that data and advancing our understanding of biology.

The last area in fundamental research is our graduate school. During the last strategic plan, we developed the St. Jude Graduate School of Biomedical Sciences, which offers a PhD and two masters programs.

Were going to expand that over the coming six years by increasing the number of students in the Biomedical Sciences PhD, the Master of Science in Global Child Health and the Master of Science in Clinical Investigations programs. We will also create a new masters program in data science. That will bring a new population of students to campus, which will further enhance our scientific enterprise.

Pediatric cancer is our next area of focus. This has always been our institutions major focus. This area encompasses $3.7 billion of the operating dollars we will spend over the next six years. Although weve invested heavily in this effort in the past, were going to expand it significantly.

Were going to focus on pediatric cancers where the least progress has been madecancers that are incurable and relapsed diseaseand gain insights into how we can change the outlook for those cancers.

The first area of investment is new faculty10 laboratory-based individuals who will expand our research efforts in understanding the biology of cancer. Some of those faculty will go into the Center of Excellence in Leukemia, but others will focus on solid tumors, brain tumors, or on biologic aspects that cut across cancer types.

Our second area for expansion will focus on assessing new therapeutic approaches for the highest-risk cancers. We need to access and evaluate more new therapies in a rigorous manner to identify those which should be moved forward into frontline clinical trials. Pediatric cancer encompasses many different types of cancer.

To run clinical trials, you need a sufficient number of patients to be able to answer questions in a reasonable time frame. We need a way to identify the best new agents to move into clinical trials.

Our investment in preclinical testing will help us set up that infrastructure. Much of it was established in the last strategic plan, but it must be expanded so that we have the best pediatric cancer models and can assess single and combination therapies to see which are worth moving forward into clinical trials.

On the clinical trial front, we want to expand our infrastructure to run those clinical trialsnot only on our campus, but in collaborations across the United States and around the globe. To make progress in some of these high-risk pediatric cancers, we need many patients. For many of the high-risk cancers, there are not a sufficient number of patients in the United States to conduct the trials. We, therefore, need to set up global collaborative networks that can address these high-risk cancers.

So, were investing in our ability to access drugs through commercial sources, to rigorously assess these in preclinical models and to establish the global infrastructure to run these clinical trials with an associated translational science infrastructure second to none.

Our third emphasis under the cancer focus is cancer immunotherapy. We began our work in cancer immunotherapy decades ago. We developed the chimeric antigen receptor, or CAR, against CD-19. That is the basis for the FDA-approved therapy that is being used right now on a variety of different fronts. Over the last several years, weve also invested heavily in expanding our cancer immunotherapy efforts, primarily focused on CAR-modified T cells.

As part of this new strategic plan, we are creating a new program, the Translational Immunology and Immunotherapy Initiative. It will facilitate cross-departmental efforts focused on cancer immunotherapy and will explore the fundamental biology of chimeric antigen receptor approaches to cancer immunotherapy.

What makes an ideal antigen that can be attacked by a chimeric antigen receptor? How does one manipulate CAR T cells so that they undergo exhaustion and stop killing the tumor? How do we change that? And what are the features of the microenvironment that decrease the killing potency of CAR T cells? These will require significant investments, including additional faculty.

Another emphasis will be looking at long-term toxicities of the therapies we use to treat children with cancer. As we cure more and more pediatric cancers, we must continually look at the toxicities from therapy and figure out how to reduce those without sacrificing the ability to be cured. Part of that is precision medicine, and so we are continuing to invest in our genomic and pharmacogenomic efforts and our proton therapy center.

Part of reducing toxicities comes from learning from long-term survivors. So, we will continue to invest in St. Jude LIFE, our long-term, follow-up study. We will expand that to some of the newer pediatric cancer therapies, including immunotherapy and targeted agents. We will assess long-term complications from these therapeutic approaches and try to define which patients will be susceptible to these toxicities.

MO: I have to mention the obvious: $11.5 billion is quite the budget. Your new strategic plan is work that, one could argue, might be on par or exceeds the coordination and budget required to realize multiple projects, say, at the World Health Organization or even at some U.S. federal agencies. What did it take for you and your team at St. Jude to get to this point?

JD: There are an incredible number of donors across the United States who support St. Jude. Our goal is to make sure every dollar is spent wisely and effectively in pursuit of our missionto advance cures and means of prevention for pediatric catastrophic diseases through research and treatment. This carries a great responsibility for us to seek the maximum possible impact to improve outcomes for childhood cancer.

We have the ideal team at St. Jude to execute this. Our leadership meets multiple times each week. Two years ago, we began strategically looking at the most pressing issues in the field of pediatric cancer. We discussed which areas represented the greatest opportunities for St. Jude to contribute. We talked to many experts inside and outside of the institutionaround the globeand made hard decisions as we went forward.

Strategic planning is deeply engrained at St. Jude as a rigorous process that is part of our scientific culture. We knew it was going to take two years to develop this plan. We dont hire consultants; we do it all ourselves. Faculty across the institution participated in the development of priorities and goals for this strategic plan via structured meetings.

As we developed the strategic plan over those two years, there were many ideas we critically assessed, and we often said, Its not really best for St. Jude to pursue that. In the end, we aligned on goals that collectively bring the prospect of remarkable benefits to the field of childhood cancer, and to children with cancer everywhere.

Every child who comes on this campus is part of our mission. We provide them with the best care possible. We do that in the context of research studies, so that were learning from every single patient. That means were not only helping children today; were also advancing cures for children tomorrow.

Weve rolled out the new strategic plan across campus during the last month, and the excitement is palpable. Our commitment continues long after the strategic plans launch.

We have routine strategic planning retreats, where we assess the goals for the year, evaluate progress against the prior years goals, perform talent assessments and proactively seek out emerging opportunities. Every employee on campus will develop yearly goals that cascade down from the goals of this plan.

As we develop this roadmap, we know there are going to be new ideas. Charlie can tell you about a process incorporated into the strategic plan that allows us to not only move forward on this roadmap, but also add initiatives as new ideas emerge.

Charles Roberts: Its a process we began with the last strategic plan, called our blue-sky process. As we launch a strategic plan, weve identified the most exciting opportunities and challenges at that point in time.

However, we fully realize that we dont know whats coming next. New discoveries will be made, and new opportunities will emerge. Via the blue-sky process, weve set aside substantial funds every year to invest in the pursuit of emerging opportunities suggested by faculty and staff.

Ideas that have emerged from the blue-sky process have been phenomenal. Our engagement with World Health Organization (WHO)a collaboration to raise childhood cancer survival rates internationallyis one example.

The Center for Modeling Pediatric Diseases is another example. This center makes iPS cells that come from patients so that we can investigate mechanisms that underlie cancer predisposition.

In another blue-sky project, were looking at DNA methylation to characterize pediatric solid tumors with the goal of identifying new therapeutic opportunities. Some of our immunotherapy initiatives also came out of the blue-sky process. Were looking forward to growing our blue-sky endeavors as we go forward.

Were coming out of a six-year strategic plan in which we increased our number of cancer patients by 20%, with 30% new faculty, 23% more staff, many large-scale construction projects. And were now going into a new strategic plan that is 60% larger than our prior plan.

The other central part of our strategic planning process focuses on the importance of collaboration. We have systematically incorporated a focus upon collaboration into our entire strategic planning and execution process. Our strategic planning efforts began by bringing together the intellectual resources of faculty and staff at St. Jude. This yielded projects that have interactions between many investigators on campus.

We recognize, however, that were still a relatively small institution, and theres a lot of expertise outside. We asked: How can we engage top scientists to tackle problems related to cancer and other catastrophic illnesses of childhood?

In pursuit of this, during our last strategic plan, we created the St. Jude Research Collaboratives, in which we fund investigators from multiple institutions who collaborate with investigators at St. Jude.

Initially, we were planning to fund two or three Collaboratives. However, they were remarkably successful, and top scientists eagerly joined.

Consequently, weve grown the program to five St. Jude Research Collaboratives already. These teams are each funded at an average of $8 million over 5 years, so each investigator is getting R01-level funding, or a little bit better. This has been a phenomenal success.

In the new strategic plan, were going to grow the program to a steady state of 11 funded collaboratives, representing close to a $90 million investment. So far, three of the Collaboratives are directly focused on childhood cancer. A fourth is a basic science-focused project relevant to childhood cancer. Were excited about the growth of this collaboration-focused program.

Lastly, Id like to address global collaboration. If you look across the globe, in low- and middle-income countries, the cure rates for childhood cancer are less than 20%.

This is a problem we know we can solve. Weve proven in the United States we can drive the cure rate to 80%. How can we help the rest of the world?

Because of the resources brought to us by our donors, we are able to think about these things, and so were now collaborating around the globe to drive cure rates forward for childhood cancer worldwide.

JD: As an example of new ideas and how rapidly we can act on them, Id like to tell you about a new blue-sky proposal that came up at the end of the last strategic plan. This idea was precipitated at a faculty retreat. One of our senior investigators was presenting, and during a coffee break, someone said, Well, what if you did this? That emerged into a blue-sky proposal, Seeing the Invisible in Protein Kinases. This was work from Dr. Babis Kalodimos, our Structural Biology department chair. He had a Science paper that came out several months ago, where he used the high-field NMR spectroscopy to look at the structure of the ABL kinase. He was able to identify transient conformational states that help to explain how resistant mutants work.

This gave us new insights into transient states that exist in molecules that can only be seen under high-field NMR, not with other structural biology approaches.

Based on that, we started thinking, Well, what if you did this on all kinases? What if you just did it against tyrosine kinases, serine kinases, receptor tyrosine kinases? What new rules would emerge from this? What would it tell us about families of kinases? What would it tell us about mechanisms of inhibition to kinase inhibitors? What might it tell us about new approaches to developing drugs against protein tyrosine kinases?

And since kinases are a major focus for targeted therapy, there was great excitement about pursuing these studies. Dr. Kalodimos developed the proposal and brought it forward; however, it was clear that this effort would be beyond the scope of our blue-sky process.

Blue-sky initiatives are usually somewhere in the $12 million range, and this was north of $30 million. Yet, in the end after thorough internal and external reviews, the project will move forward as part of the new strategic plan..

This is an approach that will give us fundamental knowledge and can have a profound impact on our understanding of a major class of targets for next-generation therapy.

MO: If I recall accurately, St. Jude has a network of partnerships with a few dozen countries worldwide. Does this plan call for an expansion of efforts within each of those countries? And how many of them?

JD: When I took over in 2014, we had what we called the International Outreach Program, which was 24 programs in 17 countries. During the programs 25-year history, we had made great progress. We were making significant impact and changing the outlook for children with cancer in those 17 countries. But we were affecting only about 3% of children with cancer across the globe, and the International Outreach Program was not scalable.

So, at the beginning of the last strategic plan, we recruited Dr. Carlos Rodriguez-Galindo. He developed a vision that after assessing, we decided to move forward on. This new effort encompasses the Department of Pediatric Global Medicine, St. Jude Global and the St. Jude Global Alliance.

These are all integrated. We developed a model that we think is scalable around the world, and we think this model ultimately can affect children with cancer everywhere.

The idea is that first we must train a workforce to treat children with cancer around the globe. We cant train the workforce ourselves, but we can train the trainers, who will then train the workforce.

More:
St. Jude's $11.5B, six-year plan aims to improve global outcomes for children with cancer and catastrophic diseases - The Cancer Letter

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