Following stem cell transplant or treatment with CAR T-cell therapies, patients with hematologic malignancies and coronavirus disease 2019 (COVID-19) tend to have favorable outcomes, especially if they are diagnosed in complete remission (CR) and further out from their cell infusion, according to Miguel-Angel Perales, MD, underscoring that care should not be delayed despite the ongoing pandemic.

Delayed therapy results in patients with relapse or progression of disease who did not receive the intended cellular therapy; [weve seen this happen] in 34% of cases, Perales, chief of the Adult Bone Marrow Transplant Service at Memorial Sloan Kettering Cancer Center (MSKCC), said during a presentation delivered at the 2021 AACR Virtual Meeting on COVID-19 and Cancer.1 Given that we can avoid the risk of nosocomial transmission, I think this clearly indicates that we should be careful about how we manage these patients and not try to delay their care.

In his talk, Perales highlighted registry data detailing the impact of the pandemic on cellular treatment in patients with cancer, outcomes of patients who were infected with the virus and received hematopoietic cell transplantation, and the impact of virus-related delays in care.

Data reported to the ASH Research Collaborative COVID-19 Registry for Hematology, a global reference tool available to the public, showed that as of January 15, 2021, a total of 813 malignant and non-malignant cases of COVID-19 were reported, with just over 500 cases reported in the United States alone.2

When looking at cellular therapies received prior to a diagnosis with the virus, 10 patients had received CAR T-cell therapies (6 recovered, 4 died), 46 patients had undergone allogeneic stem cell transplantation (34 recovered, 7 died, 5 had unknown outcome), and the majority, or 78 patients, had undergone autologous stem cell transplantation (67 recovered, 7 died, 4 had unknown outcome).

An earlier analysis of data collected from this registry showed that among the first 250 patients for whom data were collected, the overall mortality rate was 28% (95% CI, 23%-34%).3 However, in patients with moderate to severe COVID-19 infection, the mortality rate was even higher, at 42% (95% CI, 34%-50%). This is a condition that has significantly impacted our patients with hematologic malignancies, noted Perales.

Another registry, of the Center for International Blood & Marrow Transplant Research (CIBMTR), requires the inclusion of outcomes of patients who have undergone transplantation or received CAR T cells.4 As of January 15, 2021, data for 1258 patients from 195 centers were reported to the registry and showed that 50.08% of patients had undergone allogeneic transplantation and 44.66% had undergone autologous transplantation. Only a small percentage of patients received cell therapy, according to Perales.

The age of patients at the time of infection ranged from less than 20 years to older than 70 years, with the majority of patients between the ages of 60 years and 69 years. When looking at infections by region, 29.35% of cases were reported in the Midwest, 23.44% were reported in the Northeast, and 22.73% were reported in the South. The majority of cases occurred within the first 2 years of their infusion. A total of 614 casesalmost half of all patientshad their infection resolve, while 58 experienced improvement; 187 patients had died.

In a subsequent paper, investigators examined risk factors associated with death from COVID-19 in recipients of allogeneic transplantation based on data from the CIBTR registry.5 Results from the multivariate analysis showed that age greater than 50 years (P = .016), male gender (P = .006), and COVID-19 infection in less than 12 months following transplantation (P = .019) were all significantly associated with increased risk of death.

Interestingly, race and ethnicity were not significant in this series, noted Perales. Similarly, when we look at patients [who have undergone] autologous transplant, the only factor that we saw was the diagnosis of lymphoma versus myeloma. Other factors were not significant.

In another analysis, investigators examined outcomes of patients following transplant who were infected with the virus at MSKCC. Of the first 77 patients diagnosed between March 15, 2020 and May 7, 2020, 37 had undergone autologous transplant, 35 had undergone allogeneic transplant, and 5 had received CAR T-cell therapy.6

The disease distribution was as expected, according to Perales. Thirty-eight percent of patients had plasma cell disease, 23% had acute leukemia, 23% had aggressive non-Hodgkin lymphoma (NHL), 5% had Hodgkin lymphoma, 4% had chronic myeloid leukemia, 4% had myelodysplastic syndrome, and 3% had indolent NHL.

When you look at day [of infection] post infusion, you see there was a significant range, said Perales. In fact, the number of patients were diagnosed with COVID-19 several months or even years after their cell therapy. These are the demographics of 77 patients, but this is representative of the patients that we transplant at our center.

Notably, 44% of patients did not have any comorbidities. Investigators also examined the home medications that patients were receiving at the time of their COVID-19 diagnosis. Here, 10 patients were receiving steroids, 18 were receiving immunomodulatory agents, 4 were receiving anticoagulation agents, and 14 were receiving immunosuppressive drugs.

Almost half, or 48%, of patients had mild COVID-19 infection, so they were not admitted to the hospital. Twenty-six percent of patients had moderate infection, and thus, were admitted to the hospital, while 22% had severe infection and were either admitted to the intensive care unit or died.

In that group, the majority of them actually had active malignancy, unlike the other 2 groups where the majority actually were in remission, said Perales. Patients who required high levels of oxygen [were often those who] had active malignancy.

Results from a univariate analysis looking at the predictors of disease severity revealed significant associations between the presence of comorbidities and infiltrates on imaging at the time of diagnosis. Overall, however, we were able to see favorable outcomes with patients after COVID-19 infection, said Perales. Two-thirds of patients actually had a resolution. We did see 14 deaths, which represented 18% of patients. This was 41% of patients who were admitted, but particularly those with an active malignancy.

Among patients who were admitted to the hospital but had a malignancy that was in remission, the mortality rate was 21%. This was due, in part, to the fact that in many cases, patients or their family members decided to forego aggressive medical care.

Additional data revealed that COVID-19 was linked with a drop in lymphocyte populations across the board, added Perales. Notably, lymphopenia with COVID-19 was not found to impair long-term immune reconstitution in patients who had undergone bone marrow transplant.

When looking at survival in patients after infection with COVID-19, overall outcomes were found to be favorable.

Investigators also examined the risk of nosocomial infections in patients who had undergone transplantation or received cellular treatment in light of the pandemic. They looked at a series of 44 cases.

In March 2020, 2 healthcare workers were exposed at MSKCC and 3 patients had documented COVID-19 infection. One patient was receiving treatment in the inpatient setting, but the patient did have frequent visits from family members, according to Perales. So, its unclear when or how the exposure occurred, Perales said. The patient ended up dying.

Two additional patients may have been exposed in the donor room while they were collecting the stem cell from the autologous transplant, added Perales. One patient eventually died from the virus.

Again, its unclear whether these patients were infected in the center or in the community, as COVID-19 was very prevalent at the time, said Perales. Importantly, we have not seen any additional cases of potential or definite COVID-19 nosocomial infection since March 2020 at our center.

When examining the impact of the pandemic on treatment delays, in March 2020, investigators started to prospectively collect data from patients whose transplant or cellular therapy was delayed as a result of the impact of the virus on resources at the hospital, particularly the capability of using intensive care unit beds.1

Results showed that 85 patients delayed treatment; of those patients, 29 have not received their intended cellular treatment. Sixteen were supposed to receive autologous transplant, 12 were supposed to undergo allogeneic transplant, and 1 was supposed to receive CAR T-cell therapy.

Of the 56 patients who eventually proceeded to treatment, 62% received autologous transplant, 67% received allogeneic transplant, and 86% received CAR T-cell therapy. The biggest reason for not proceeding to treatment with autologous transplant and CAR T-cell therapy was because they were deferred due to good disease control. Other reasons included was because of a new comorbidity (12%) or they died from the virus. The most prominent reason for not proceeding to allogeneic transplant during the pandemic was progression of disease (42%).

We conclude that patients who are recipients of allogeneic transplant, and particularly those with acute leukemia, as much as possible should proceed to their indicated therapy and not be delayed, concluded Perales.

Excerpt from:
Perales Examines the Impact of COVID-19 on Recipients of Cellular Therapies for Cancer - OncLive

Image for representation purpose only.

A tech entrepreneur, who is determined to live for 180 years, claims his bizarre strategies will soon be as popular as cell phones. Dave Asprey firmly believes one of the keys to living longer is skipping breakfast. The American lifestyle guru has asserted that he can at least live to the year 2153 by using a variety of techniques, including a cold cryotherapy chamber sitting and intermittent-fasting. The 47-year-old millionaire devised the term 'Biohacking' to detail his methods of turning back the biological clock. In an effort to survive for as long, Dave has spent over $1,000,000 on hacks and techniques to improve the overall functioning of his body. He has parts of his bone marrow removed to re-inject his own stem cells back into his body.

Appearing on ITVs This Morning today, he joined host Holly Willoughby for a virtual conversation. Holly quizzed Dave why he wants to live so long, to which he replied that as a curious person, he feels there is a lot in the world that can be fixed and improved that he thinks he has not done yet. Dave confirmed that some of the things he wants to pioneer are expensive. However, there are other ways that are free of cost like fasting.

Dave also speculated that after applying his methods for longer life, people under 40 will be 'happy and highly functional' over a 100-years-old. He stated that he won't be the only one as his wife both is also racing to get to 180 years old. In comparison to others, Dave has set himself up for much less inflammation by controlling what he eats and how he sleeps, besides several other anti-ageing treatments. Explaining the benefits of reintroducing his own stem cells in his body, he said people heal when they are young. With age, the stem cells of the body get exhausted, so he opts for ways which gives him more stem cells.

Dave also follows cryotherapy, which is also known as cold therapy. It involves using low temperatures to treat a variety of tissue lesions. He is also taking cold showers for more than ten years. Dave uses another technique to live a long life known as intermittent fasting. This involves controlling the number of times that one eats meals to create periods of fasting over a certain period. According to Dave, it helps in disease prevention as fasting periods lets his body to 'repair itself' while not digesting food.

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US Man Who Wants to Live for 180 Years Re-injects His Own Stem Cells, Spends Rs 87 Lakh - News18

The reduced osteogenic differentiation of bone marrow mesenchymal stem cells (BMSCs) is the typical characteristics of pediatric aplastic anemia (AA) pathogenesis. Long non-coding RNA MEG3 is reported to promote osteogenic differentiation of BMSCs via inducing BMP4 expression.This study aims to investigate the mechanism of DNMT1/MEG3/BMP4 pathway in osteogenic differentiation of BMSCs in pediatric AA.BMSCs were isolated and purified from bone marrows of pediatric AA patients (n=5) and non-AA patients (n=5). The expression of DNMT1, MEG3, and BMP4 in isolated BMSCs was detected using quantitative real-time PCR and western blot analysis. Osteogenic differentiation was determined using Alizarin red staining. The methylation of MEG3 promoter and the interaction between DNMT1 and MEG3 promoter were detected using methylation-specific PCR and chromatin immunoprecipitation assay, respectively.Lowly expressed MEG3 and BMP4 and highly expressed DNMT1 were observed in BMSCs of pediatric AA patients. The overexpression of MEG3 promoted osteogenic differentiation of BMSCs. Luciferase reporter assay showed that MEG3 overexpression increased transcriptional activity of BMP4. The inhibitor of methylation, 5-azacytidine, suppressed DNMT1 expression and reduced methylation of MEG3 promoter. Overexpression of DNMT1 increased the binding between DNMT1 and MEG3 promoter. The simultaneous overexpression of DNMT1 and MEG3 restored the inhibition of osteogenic differentiation caused by DNMT1 overexpression alone.Our findings indicated that DNMT1 mediated the hypermethylation of MEG3 promoter in BMSCs, and DNMT1/MEG3/BMP4 pathway modulated osteogenic differentiation of BMSCs in pediatric AA.

PubMed

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Hypermethylation-mediated downregulation of long non-coding RNA MEG3 inhibits osteogenic differentiation of bone marrow mesenchymal stem cells and...

The mum of a Stockton toddler says time is running out to find a match for her son who is battling a rare blood cancer.

Little Mason Arrowsmith, one, was diagnosed with Juvenile Myelomonocytic Leukaemia (JMML) on Christmas Eve last year.

The condition is a rare type of slowly developing blood cancer that occurs in young children.

Although it was discovered at an early stage, doctors say Mason needs a stem cell or bone marrow transplant for the best chance of survival.

In a desperate attempted to help, his mum Katie Jordan will donate her own bone marrow later this month, but a full match is urgently needed.

"The hospital has been absolutely amazing, but unfortunately after searching worldwide there is no match for Mason due to his bone marrow being so rare," Katie said.

"We need to get him to transplant as soon as, the hospital is looking at the end of February, using me as his first donor in the hope that this will help him until we find a better match.

"I would give my life for Mason but unfortunately, I can only donate my bone marrow three times in a lifetime and Im not a full match for him.

"Children with JMML live around 12 months after diagnosis, we need to find mason a better match. Twelve months is not long enough for us to have with our boy, we are living the worst possible nightmare."

As Mason does not have a close enough match within his family - the only potential cure is through a stem cell transplant from an unrelated donor.

The family has now launched a 'Masons Mission' to encourage people to support blood cancer charity Anthony Nolan in raising urgent funds to add more donors to the stem cells register.

There is currently a blacklog of around 25,000 potential donors due to the impact of the coronavirus pandemic and the charity needs to raise up to an extra 500,000 to add people to the register, from ordering more swab packs to analysing completed swabs in its laboratory.

Any one of the 25,000 people who have applied to join the Anthony Nolan could be a match for Mason or one of the 2,300 patients in the UK, who need a stem cell transplant from a donor each year.

Katie added: "We have set our target at 10,000 which does seem a lot but this would help to cover 250 registrations and kits and allow us to continue our search for my baby."

Henny Braund, Chief Executive of Anthony Nolan said: "Were doing all we can to find a stem cell donor to give Mason a second chance of life.

"A perfect storm of the coronavirus pandemic, and a surge of 40,000 incredible people who have been inspired to join the Anthony Nolan register in the last month by patients, like Mason means that were in urgent need.

"The best thing people can do is support Anthony Nolans work financially. By giving anything, together we can add all potential lifesavers to the register, and give patients like Mason hope."

Anthony Nolan recruits people aged 16-30 to the stem cell register as research has shown younger people are more likely to be chosen to donate.

They also carry out ground-breaking research to save more lives and provide information and support to patients after a stem cell transplant, through its clinical nurse specialists and psychologists, who help guide patients through their recovery.

It costs 40 to recruit each potential donor to the register, so Anthony Nolan relies on financial support.

You can support Masons Mission here.

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Family 'living worst nightmare' as they desperately seek donor for tot with rare blood cancer - Teesside Live

SAN DIEGO, Feb. 04, 2021 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX), a clinical stage pharmaceutical company focused on the discovery, development, and commercialization of novel therapeutics for rare endocrine diseases and endocrine-related tumors, today announced that CRN04894, the company’s lead adrenocorticotropic hormone (ACTH) antagonist for the treatment of diseases associated with excess ACTH such as Cushing’s disease and congenital adrenal hyperplasia (CAH), has advanced into the clinic. Based on encouraging preclinical results, Crinetics has initiated a double-blind, randomized, placebo-controlled Phase 1 study of this orally administered, nonpeptide small molecule drug candidate in healthy volunteers. This study will assess the safety and tolerability of single and multiple doses of CRN04894 and will measure the effect of CRN04894 on suppression of cortisol, cortisol precursors, and adrenal androgens following exogenous ACTH stimulation.

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Crinetics Pharmaceuticals Lead ACTH Antagonist for Congenital Adrenal Hyperplasia and Cushing’s Disease (CRN04894) Advances into Phase 1 Study

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Corvus Pharmaceuticals Initiates Phase 3 Clinical Trial of CPI-006 for Patients with COVID-19

– Pharmacodynamic results confirm localized exoIL-12 pharmacologic activity

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Codiak Reports Additional Positive Phase 1 Results for exoIL-12™ Confirming Local Pharmacology and Dose Selection for Safety and Efficacy Trial...

Orphazyme USInvestor news                                                                                                         No. 02/2021

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Orphazyme to showcase data on arimoclomol in Niemann-Pick disease Type C during the 2021 Annual WORLDSymposium™

MONTREAL, Feb. 04, 2021 (GLOBE NEWSWIRE) -- Theratechnologies Inc. (Theratechnologies) (TSX: TH) (NASDAQ: THTX), a biopharmaceutical company focused on the development and commercialization of innovative therapies, is pleased to announce that the United States Food and Drug Administration (FDA) has granted fast track designation to TH1902 as a single agent for the treatment of patients with sortilin positive recurrent advanced solid tumors that are refractory to standard therapy.

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Theratechnologies’ Lead Peptide Drug Conjugate TH1902 Receives FDA Fast Track Designation for the Treatment of Sortilin-expressing Cancers

SAN DIEGO, Feb. 04, 2021 (GLOBE NEWSWIRE) -- Otonomy, Inc. (Nasdaq: OTIC), a biopharmaceutical company dedicated to the development of innovative therapeutics for neurotology, today announced it will report financial results for the fourth quarter and full year 2020 and provide a corporate update at 4:30 p.m. ET on February 11, 2021.

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Otonomy to Report Fourth Quarter and Full Year 2020 Financial Results and Provide Corporate Update

RESEARCH TRIANGLE PARK, N.C., Feb. 04, 2021 (GLOBE NEWSWIRE) -- G1 Therapeutics, Inc. (Nasdaq: GTHX), a clinical-stage oncology company, today announced that G1’s Chief Executive Officer Jack Bailey will participate in the Guggenheim Healthcare Talks: 2021 Oncology Day conference. The fireside chat will take place on February 11, 2021 at 2:00 PM ET. This meeting is being held virtually, and a live webcast will be accessible on the Events & Presentations page of http://www.g1therapeutics.com.

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G1 Therapeutics to Present at the Guggenheim Healthcare Talks: 2021 Oncology Day Virtual Conference

SANTA ANA, Calif., Feb. 04, 2021 (GLOBE NEWSWIRE) -- NKMax America, a biotechnology company harnessing the power of the body's immune system through the development of Natural Killer (NK) cell therapies, announced the appointment of Stephen Chen, MBA to Chief Operating Officer (COO).

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NKMax America Announces Key Leadership Appointment

SOUTH SAN FRANCISCO, Calif., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Global Blood Therapeutics, Inc. (GBT) (NASDAQ: GBT) today announced that an expanded access protocol (EAP) for Oxbryta® (voxelotor) in pediatric patients with sickle cell disease (SCD) has been initiated and is currently enrolling. The EAP is designed to provide access to Oxbryta prior to approval for children ages 4 to 11 years with SCD in the United States who have no alternative treatment options and are ineligible to participate in clinical trials of Oxbryta. GBT enrolled its first patient in the EAP in January 2021.

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GBT Initiates Expanded Access Protocol for Oxbryta® (voxelotor) in Pediatric Patients with Sickle Cell Disease in the United States

SOUTH SAN FRANCISCO, Calif., Feb. 04, 2021 (GLOBE NEWSWIRE) -- CytomX Therapeutics, Inc. (Nasdaq: CTMX), a clinical-stage, oncology-focused biopharmaceutical company pioneering a novel class of investigational antibody therapeutics based on its Probody® technology platform, today announced that Sean McCarthy, D.Phil., president, chief executive officer, and chairman, will participate in a virtual fireside chat at the Guggenheim Healthcare Talks 2021 Oncology Day on February 11th at 3:30 p.m. ET. In addition, management will be available for one-on-one meetings with investors.

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CytomX Therapeutics to Present at Guggenheim Healthcare Talks 2021 Oncology Day

BETHESDA, Md., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Gain Therapeutics, Inc., (“Gain”) a biotechnology company focused on redefining drug discovery by identifying and optimizing allosteric binding sites that have never before been targeted, today announced that it will present three late-breaker abstracts at the 17th annual WORLDSymposium, a research conference dedicated to lysosomal diseases being held virtually February 8-12, 2021. The poster presentations will highlight data supporting Gain’s Gaucher disease, GM1 gangliosidosis and Morquio B programs.

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Gain Therapeutics Announces Three Late-Breaker Presentations at the 17th Annual WORLDSymposium

RP1: Initial data in new indications expected in 2021 in anti-PD1 failed NSCLC, anti-PD1 failed CSCC and CSCC solid organ transplant recipient patients; further updates expected to be provided across all studies

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Replimune Reports Third Fiscal Quarter Financial Results and Provides Corporate Update

Company to Participate in Two Q&A Sessions, Including a Joint Discussion with Zai Lab Company to Participate in Two Q&A Sessions, Including a Joint Discussion with Zai Lab

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Turning Point Therapeutics to Participate in Guggenheim Healthcare Talks 2021 Oncology Days

LEXINGTON, Mass., Feb. 04, 2021 (GLOBE NEWSWIRE) -- Agenus Inc. (NASDAQ: AGEN), an immuno-oncology company with an extensive pipeline of checkpoint antibodies, cell therapies, adjuvants, and vaccines designed to activate immune response to cancers and infections, today announced positive preliminary results from its Phase 1 trial of iNKT cell therapy in patients with moderate to severe symptoms of COVID-19 through its subsidiary, AgenTus Therapeutics.

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Agenus Announces Positive Preliminary Results of iNKT Cell Therapy Trial in COVID-19

Overallotment option on recent public offering partially exercised by underwriter

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Outlook Therapeutics’ Recent Financing Secures Funding to Support ONS-5010 / LYTENAVA™ (bevacizumab-vikg) Through Planned BLA Submission

Milestone Marks Advance for Oral Mucosal Immunotherapy for Peanut Allergies Milestone Marks Advance for Oral Mucosal Immunotherapy for Peanut Allergies

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Intrommune Receives IND Clearance From U.S. Food and Drug Administration for INT301