Category Archives: Molecular Medicine

DetailsCategory: More NewsPublished on Wednesday, 04 August 2021 13:50Hits: 298

HOUSTON, TX, US AI August 04, 2021 I Excel Diagnostics and Nuclear Oncology Center (EDNOC), a premier Diagnostic Imaging and Radioligand Therapy center in Houston, Texas announces approval of its physicians sponsored Investigational New Drug (IND) application for providing Targeted Alpha Therapy (TAT) with 225Ac-PSMA I&T for metastatic Castration-Resistant Prostate Cancer (mCRPC). We are glad to be able to make this game-changing drug available to our patients suffering from mCRPC. Excel Diagnostics and Nuclear Oncology Center has been a pioneer in bringing new targeted Radioligand Therapies such as Lu-177 DOTATATE, and Lu-177 PSMA to serve our patients in the United States. The addition of 225Ac-PSMA I&T is in continuation of our mission to address unmet needs in the field of Nuclear Oncology said Dr. Ebrahim Delpassand, Chairman and Medical Director of EDNOC and Co-Principal Investigator of the trial. PSMA is an established prostate cancer target. Highly encouraging results have been reported by using Targeted Alpha Therapy (TAT) Radiopharmaceuticals in conditions such as neuroendocrine, prostate, or hematological malignancies. These breakthroughs have brought a significant amount of hope to our patients suffering from different types of advanced cancers. said Dr. Rodolfo Nuez, Director of Nuclear Medicine Department at EDNOC and Co-Principal Investigator of the trial. We are pleased to be able to facilitate the availability of 225Ac-PSMA I&T by manufacturing this drug at RadioMedix. We firmly believe that the menu of targeted Radioligand Therapies will only increase in the future. RadioMedix with its wide range of capabilities, from drug discovery to scale-up commercial manufacturing, is ready to meet this challenge said Dr. David Ranganathan, Director of CMC and Regulatory affairs at RadioMedix.

About Excel Diagnostics and Nuclear Oncology Center (EDNOC)

EDNOC is a premier outpatient facility offering a full spectrum of diagnostic imaging, nuclear medicine and Radioligand therapies. In addition to our comprehensive and state-of-the-art imaging services, we have assembled a staff of highly skilled technical and medical professionals to meet and exceed the demanding standards of the industry. EDNOC is fully capable of conducting sponsored clinical research trials in the field of diagnostic and therapeutic nuclear medicine. All our research staff is GCP Trained and CITI certified consisting of highly trained, board-certified investigators, study coordinators, patient recruiters, and support staff. At Excel, you will find areas designed to safely handle and administer radiopharmaceuticals, patient care areas, and physician interpretation resources. The Excel Clinical Research Department (ECRD) is a dedicated department to assist investigators in their clinical trials. Our team is fully trained regarding rules and regulations of conducting human research, FDA requirements, and IRB compliance. We offer our expertise in conducting clinical research to a variety of academic institutions and pharmaceutical companies. ECRD works closely with CROs & IRBs, to support sponsored clinical trials in the field of oncology radiopharmaceuticals. In April of 2020, Excel passed FDA inspection of one of its investigational drugs with no deficiencies or citations. For more information, please visit us at: http://www.exceldiagnostics.com

About RadioMedix

RadioMedix, Inc. is a clinical-stage biotechnology company, based in Houston, Texas, focused on innovative targeted radiopharmaceuticals for diagnosis, monitoring, and therapy of cancer. The company is developing radiopharmaceuticals for PET imaging and therapy (alpha and beta-labeled). RadioMedix has also established contract service facilities for academic and industrial partners including drug discovery and probe development core facility, a small animal molecular imaging facility for pre-clinical evaluation of radiopharmaceuticals, and cGMP and analytical suites for late-stage human clinical trials, and post-approval commercial manufacturing. To learn more, visit http://www.radiomedix.comand LinkedIn. For more information about this press release, please contact:This email address is being protected from spambots. You need JavaScript enabled to view it.">This email address is being protected from spambots. You need JavaScript enabled to view it.

SOURCE: RadioMedix

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Excel Diagnostics and Nuclear Oncology Center Announces FDA Authorization of IND for 225Ac-PSMA I&T Targeted Alpha Therapy for Castration Resistant...

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Foundation Medicine, Inc. today announced that it has received approval from the U.S. Food and Drug Administration (FDA) for FoundationOneLiquid CDx to be used as a companion diagnostic to aid in identifying patients with MET exon 14 skipping (METex14) in metastatic non-small cell lung cancer (NSCLC) for whom treatment with TABRECTA (capmatinib) may be appropriate. TABRECTA is the first therapy approved by the FDA for adult patients with metastatic NSCLC whose tumors have an alteration that leads to METex14. FoundationOne Liquid CDx analyzes the largest genomic region of any FDA-approved comprehensive liquid biopsy test and was approved by the FDA in August 2020 to report genomic alteration results for patients with any solid tumor.

NSCLC accounts for approximately 85% of lung cancer diagnoses,[1] 3 to 4% of which are associated with METex14.[2] Today's approval adds to the number of therapies for which both of Foundation Medicines FDA-approved comprehensive genomic tests are listed as companion diagnostics. FoundationOneCDx, Foundation Medicines tissue test, was approved as a companion diagnostic for TABRECTA in May 2020.

For lung cancer patients with METex14, having the option of a non-invasive liquid biopsy expands access to this first-of-its kind therapy and helps meet a critical patient need, said Brian Alexander, M.D., M.P.H., chief executive officer at Foundation Medicine. This approval, coupled with last years simultaneous therapy and companion diagnostic approval for TABRECTA and our tissue test, FoundationOne CDx, is an important advancement and demonstrates the value of having multiple highly-validated comprehensive genomic testing options for physicians to consider for the individual needs of each patient.

Using a simple blood sample, FoundationOne Liquid CDx analyzes over 300 cancer-related genes for genomic alterations. The test is now approved as a companion diagnostic for nine targeted therapies across four cancer types. TABRECTA is the second therapy for which both of Foundation Medicines FDA-approved tests, FoundationOne CDx and FoundationOne Liquid CDx, are listed as companion diagnostics.

Additionally, as a laboratory professional service which has not been reviewed or approved by the FDA, the FoundationOne Liquid CDx report delivers information about the genomic signatures microsatellite instability (MSI) and blood tumor mutational burden (bTMB), as well as single gene alterations, including NTRK fusions, to help inform the use of other therapies including immunotherapies. Also, as a laboratory professional service, the report provides relevant clinical trial information and includes interpretive content developed in accordance with professional guidelines in oncology for patients with any solid tumor.

Foundation Medicines strategic collaboration with Novartis now includes four companion diagnostics for the Novartis portfolio of targeted oncology therapeutics.

About FoundationOne Liquid CDx

FoundationOne Liquid CDx is a qualitative next generation sequencing based in vitro diagnostic test for prescription use only that uses targeted high throughput hybridization-based capture technology to analyze 324 genes utilizing circulating cell-free DNA (cfDNA) isolated from plasma derived from anti-coagulated peripheral whole blood of advanced cancer patients. The test is FDA-approved to report short variants in over 300 genes and is a companion diagnostic to identify patients who may benefit from treatment with specific therapies (listed in Table 1 of the Intended Use) in accordance with the approved therapeutic product labeling. Additional genomic findings may be reported and are not prescriptive or conclusive for labeled use of any specific therapeutic product. Use of the test does not guarantee a patient will be matched to a treatment. A negative result does not rule out the presence of an alteration. Patients who are negative for companion diagnostic mutations should be reflexed to tumor tissue testing and genomic alteration status confirmed using an FDA-approved tumor tissue test, if feasible. For the complete label, including companion diagnostic indications and complete risk information, please visit http://www.F1LCDxLabel.com.

About Foundation Medicine

Foundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patients cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicines molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit http://www.FoundationMedicine.com or follow Foundation Medicine on Twitter (@FoundationATCG).

Foundation Medicine and FoundationOne are registered trademarks of Foundation Medicine, Inc.

TABRECTA is a trademark of Novartis.

Source: Foundation Medicine

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Foundation Medicine Expands Indication for FoundationOneLiquid CDx to be Used as a Companion Diagnostic for TABRECTA (capmatinib) - Business Wire

Its been said that late stage or terminal patients with cancer should have immediate, frank discussions with their oncologists about their prognosis. I understand the practical reasons for such advice, but Im not so sure I agree in other respects. Heres why. No one knows with absolute certainty how our bodies will react to treatment. The statistics dont lie, I understand, but they also allow for a slim margin some might chalk up to miracles and others to giant leaps in modern medicine.

My oncologist didnt tell me the stage of my ovarian cancer when she gave me my diagnosis. In fact, she said it was highly treatable. Notice she didnt term it highly curable. I didnt catch the difference at first. I discovered the metastatic stage 4 diagnosis on my patient portal page right before being admitted to the hospital for my port placement and first round of chemotherapy. It was shocking, scary and stressful. I dont recommend it.

However, in retrospect, I embrace my oncologists can-do attitude from the moment she gave me my diagnosis. She emphasized all the treatment options available to me, regardless of staging. She pointed out that new drugs and treatment protocols were constantly being developed. The longer I stayed alive, the more I had a chance at those new treatments. I decided my job was to actively participate in my treatment. Show up for appointments. Take medications as prescribed. Eat well. Exercise. Communicate about side effects. Pray. And live well.

The sad truth is I couldve done all those things and still succumbed to the disease. Ovarian cancer is the deadliest of the gynecological cancers. Depending on which statistics you believe, my five-year survival rate was somewhere between 19 and 30 percent. Im now in year six. I dont know why Im one of the women who has made it this far. Why am I not platinum resistant? Why did the frontline chemo and surgery result in a period of no evidence of disease (NED) for me and not for other women? Researchers point to molecular makeup of tumors, genetics and other health issues. Maybe those factors played a role. I dont know. So far, Ive survived two recurrences with the third NED period lasting almost two years now.

The point being I couldve had that get-your-affairs-in-order discussion with my doctor in January of 2016. I couldve implemented my bucket list, backed away from writing contracts, outlined my end-of-life wishes and prepared my children for my possible, impending demise. Instead, I signed a four-book contract and started a fulltime career as a fiction writer.

The only step I did take was to prepare a medical directive and a living will. It was the responsible thing to do. We should all do it. Anyone can be struck by tragedy at any moment. The fragility of life is no secret.

Im not trying to ignore grim reality. Nor do I embrace the always-be-positive Im going beat this thing mentality. Nor am I in the Gods got this camp. Yes, I pray and ask my church family to pray for me. I believe in the power of prayer. But I also ask myself why God would decide to answer my prayers and not those of the couple in the pew next to me whose daughter died of metastatic breast cancer a few years ago. Did they not pray hard enough? Part of Gods plan? I refuse to suggest a layperson like myself knows the answer to a question that stumps many learned theologians daily.

Im simply saying make room for miracles and modern medicine. Live every day with all your heart. Make the most of your time, however short or long. Thats good advice for all of us, cancer or not.

For more news on cancer updates, research and education, dont forget tosubscribe to CUREs newsletters here.

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Making Room for Miracles And Modern Medicine as a Patient With Stage 4 Cancer - Curetoday.com

The United States is in the midst of a major new upsurge of the COVID-19 pandemic that has already taken the lives of 624,000 people. Over the past month, daily cases have increased 250 percent, driving a rise in hospitalizations and a significant increase in the daily death rate.

The Delta (4th) wave in the United States is already showing it to be on a path to its the worst yet in major hotspots, noted Eric Topol, a professor of molecular medicine at the Scripps Research Institution.

The surge is concentrated in the poorest sections of the country, including Arkansas, Mississippi, Missouri, Florida and Nevada, where vaccination rates are lowest. In Missouri, the tenth-poorest state in the country which has one of the lowest vaccination rates, hospitals are at the highest occupancy at any point of the pandemic. We only get beds available when someone dies, which happens several times a day, Terrence Coulter, the critical-care medical director at CoxHealth, told the Atlantic.

The rise in cases is caused by the Delta variant of COVID-19, which not only spreads more rapidly, but reproduces much more aggressively inside infected people. A study published earlier this month noted that the viral load of people infected with the Delta variant was 1,000 times greater than those infected by the initial variant of the disease.

The surge in sections of the country with the lowest vaccination rates has been accompanied by a troubling growth in so-called breakthrough infections among vaccinated people. According to official figures from the US Centers for Disease Control and Prevention (CDC), 791 fully vaccinated people have died from COVID-19 in the US so far, and 5,000 have been hospitalized.

Three fully vaccinated athletes inside the Olympic village in Tokyo and one staff member tested positive over the weekend, raising the threat of new outbreaks at the worlds premier sporting event. In recent days, three fully vaccinated members of the Texas House delegation in Washington, D.C., tested positive after traveling maskless on a chartered airplane.

Moreover, less than half of the population in the US is fully vaccinated. While the media blames this fact on people who do not want to get the vaccine, the ruling class has been unwilling to organize the type of public education and mass distribution program that is required.

In recent days, it has become clear that the US government expects the upsurge that has already taken place in southern states to be merely the prelude to a new wave of the pandemic.

This is just going to spread through the population, Trumps former FDA director Scott Gottlieb told CNBC on Friday. He pointed to an internal CDC model showing an increasing epidemic, a wave of infection from this Delta variant moving through the population over the next two months.

The assumptions built into those models is no mitigation, no mandates for masks, no closures of businesses, Gottlieb added. I think thats likely to be the norm.

What Gottlieb is describing is the deliberate mass infection of the American population, allowing tens or hundreds of thousands more to die. The Trump-appointed FDA director was not describing some ideal world that would exist if the COVID-19 denier Trump was still in power, but the actual policies of the Biden administration.

On May 13, the CDC, under pressure from the Biden administration, announced that it was no longer recommending that vaccinated people wear masks, triggering the effective abandonment of all social distancing measures by businesses, states and municipalities throughout the country.

The World Socialist Web Site, in a position consistent with the World Health Organization and leading public health experts, opposed the CDCs decision, warning that it would lead to a new resurgence of the pandemic. Less than two months later, these warnings are being confirmed.

Facing the disastrous outcome of the White Houses policies, Bidens CDC Director, Dr. Rochelle Walensky, was asked last week, Is there any consideration, any scenario in which you might want to reverse yourself on reopening schools?

Walensky replied, I remain emphatic that our schools need to open in the fall. They need to open for full, in-person learning. When asked again, CDC is not recommending people who are fully vaccinated wear masks? Walensky responded, We are not.

The Biden administrations open opposition to masking and the end to all social distancing measures is virtually indistinguishable from the policies of former US President Donald Trump, whose disastrous handling of the COVID-19 pandemic was a major factor in Bidens victory.

220,000 Americans dead, Biden said in his opening remarks at the second presidential debate. Anyone is responsible for that many deaths should not remain as president of the United States of America. He added, I will take care of this. I will end this.

But since Inauguration Day, a further 196,000 people have died of COVID-19. That is, almost as many Americans have died under Biden as had died when Biden proclaimed anyone responsible for such mass death had forfeited his right to be president.

The president who pledged to follow the science is rejecting the demands of scientists, discouraging mask-wearing and peddling pseudo-science that children cannot be infected with COVID-19 and that schools are not centers for the transmission of the disease.

Tens of millions of people voted for Biden in the belief that he would take the measures necessary to stop the pandemic. But these promises were empty, because Biden, like Trump, represents the interests of the financial oligarchy that has massively enriched itself as hundreds of thousands have died.

It is urgently necessary to draw the lessons of the year and a half that has elapsed since the start of the pandemic. Under the banner of herd immunity, capitalist governments throughout the world made the calculated decision to sacrifice millions of lives because saving them would have impinged on the profit interests of the financial oligarchy.

With more than four million deaths, the mass of humanity is no closer to eradicating the pandemic than it was in March. Rather, the uncontained spread of the disease has led to the development of ever-more-dangerous variants.

Stopping the pandemic requires a radically different approach. This means the closure of schools and nonessential businesses, with full compensation for all those who lose any wage or small business income. This must be combined with the allocation of vast social resources to ensure that every case of COVID-19 is meticulously tracked and that every infected person is given a safe and comfortable place to quarantinewith full financial compensationuntil they are no longer infectious.

It is a fundamental fact that, despite the unanimous consensus of scientists on the measures necessary to contain COVID-19, there is only one political party in the United States calling for the stopping of nonessential production: the Socialist Equality Party, affiliated with the International Committee of the Fourth International that publishes the World Socialist Web Site. That is because the Socialist Equality Party does not accept the economic prerogatives of the capitalist class.

If COVID-19 is to be contained, it will only be through a mass mobilization of the working class to demand urgent measures to stop the pandemic, whatever the cost to the wealth of the financial oligarchy. The ill-gotten gains made by Americas billionaires while hundreds of thousands died must be seized and used to fund the measures necessary to stop the pandemic.

As the disease rips through workplaces, workers will form rank-and-file committees to demand the closure of non-essential production and the enforcement of critical safety guidelines. Teachers must and will resist the efforts to reopen schools for in-person learning under conditions in which the pandemic is still spreading.

The inability of the capitalist system to stop the spread of the COVID-19 pandemic has made clear the incompatibility of capitalism with the social needs of society. The struggle to save human lives in the pandemic is inseparable from the struggle for socialism.

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Urgent action is necessary to stop the US Delta variant surge! - WSWS

We in Massachusetts are living in a bit of a pandemic bubble right now, both literally and figuratively. The high vaccination rate in our state, as well as in neighboring states throughout New England, has contributed to a dramatic drop in the number of COVID-19 infections, deaths, and hospitalizations in our part of the country. The successful reopening of our economy serves as a testament to the value of having a highly-vaccinated and highly-educated population.

Its as if our region of the U.S. is equivalent to an island nation such as New Zealand, where COVID-19 has not made a dent in economic or other activity since the beginning of the pandemic because its prime minister, Jacinda Ardern, sealed off its borders to foreigners from the very beginning of the pandemic. However, New England is not an island. Millions of our own citizens are traveling to other places and returning, and millions of non-residents are coming here to visit this summer.

The Delta variant of COVID-19 is now the dominant strain of the virus both in this country and throughout the world. The troubling aspects of Delta are that it is much more highly-transmissible than the original, it is more lethal, and the vaccines are slightly less-efficacious against it compared to the original strain of the virus for which the vaccines specifically were developed. In Australia (where vaccination rates are very low), the Delta variant has shown itself to be a whole new ballgame, so to speak, in terms of how contagious it is. The virus has been transmitted among people who simply came fleetingly into contact with each other and shared the same airspace in an indoor mall.

It is the most hyper-transmissible, contagious version of the virus weve seen to date, for sure its a superspreader strain if there ever was one, said Eric Topol, a professor of molecular medicine and an executive vice president at the Scripps Research Institution, in a recent interview in Scientific American. The Delta variant is being blamed for the huge increases in infections and deaths throughout the world, particularly in places where vaccination rates are in the low single digits. There also are many areas in the U.S., such as parts of Texas, Missouri, and Arkansas, where vaccination rates are low, that predictably now are seeing large increases in COVID-19 cases caused by Delta.

The Delta variant is concerning enough on its own, but the real problem is this: The more people who become infected with COVID-19, the more likely that the virus will mutate into additional variants, with the possibility that vaccination efficacy could begin to drop significantly if one of these strains develops an ability to evade the vaccines protective effects. It is nothing less than tragic and despicable, really that there are some in public life who are urging Americans NOT to get vaccinated.

That mindset was on display this past weekend at the Republican-dominated CPAC conference, where some clown on a panel who spoke out against the nations vaccination program was actually applauded by those in attendance. There is a strong and vocal minority in this country who strive to create chaos thats what makes them tick. Whether we as a nation can overcome the combination of venality and stupidity that was on display at CPAC this past weekend will determine whether we can beat the pandemic in the short term and whether our democracy and our way of life can survive in the long term

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Sorry Folks But the Pandemic is Far From Over - Lynn Journal

Launching today: eLearning programme to support advanced therapy training in the NHS, UK universities and government bodies

UK, 19th July 2021 The Advanced Therapy Treatment Centre (ATTC) network, London Advanced Therapies (LAT) and the Cell and Gene Therapy Catapult (CGTC), in partnership with Health Education England elearning for healthcare, have developed a new eLearning programme targeted at healthcare and academic professionals to support their learning on both the fundamentals and clinical adoption of advanced therapies.

This series of eLearning sessions is designed to give the learner a core understanding of advanced therapies, how they function in the body, and the steps involved in delivering these medicines. The modules take the learner from the basics of cell and gene therapy, through to a more in-depth look at products currently being delivered through both commissioned treatments and clinical trials.

Advanced therapy medicinal products (ATMPs) are medicines for human use, which use genes, tissues or cells to offer ground-breaking new opportunities for the treatment of disease and injury. These therapies are potentially curative and can offer the promise of treating and altering the course of diseases which cannot be addressed adequately by existing pharmaceuticals, offering a lifeline to some patients who may have exhausted all other treatment options.

Learners will also be introduced to the unique challenges of bringing these pioneering advanced therapy treatments to patients, including the often nuanced logistical and handling requirements that can present unique challenges within usual standard of care.

The sessions, which can be mixed and matched according to the learners needs, comprise the following topics:

For more information and to access the sessions, visit the programme page https://www.e-lfh.org.uk/programmes/advanced-therapy-medicinal-products/

Professor Uta Griesenbach, Professor of Molecular Medicine, Imperial College London and Chair of the pan-UK ATMP Training Group commented:

As more ATMPs enter late-stage clinical trials, we identified a training need for healthcare professionals to ensure fast development of ATMPs and smooth transition into the NHS. This is a fantastic example of collaboration between NHS, academia and industry, coordinated by CGTC to produce valuable training for UK personnel and future-proof the healthcare system. Access to these sessions sets the UK apart from other countries by making high quality, standardised training available to all staff involved in the delivery of these life-changing medicines.

Professor Fiona Thistlethwaite, iMATCH Director, Medical Oncology Consultant and Christie NHIR CRF Director commented:

Providing comprehensive training for staff to understand the complex requirements of these new therapies is essential as we scale up our delivery of ATMPs across multiple patient groups. With input from recognised ATMP experts from the ATTC network and NHS partners across the UK, staff can now use the information provided to support their continued professional development. This tailored online learning approach also allows our staff to build their knowledge in a way that fits around their schedules.

About Health Education England elearning for healthcare

Health Education England elearning for healthcare (HEE elfh) works in partnership with the NHS, third sector and professional bodies to support patient care by developing eLearning resources to educate and train the health and care workforce. The eLearning programmes cover content from anaesthesia to dentistry, end of life care to mental health, and population wellbeing to sepsis. Users can access statutory and mandatory training, obtain certificates and complete eLearning sessions relevant to their role. For more information about elfh visit http://www.e-lfh.org.uk

About the Advanced Therapy Treatment Centre (ATTC) network

The Advanced Therapy Treatment Centre network was set up to address barriers to the clinical adoption of advanced therapies by increasing the capacity and capability of the NHS to efficiently deliver the growing number of these new medicines. The network is coordinated by the Cell and Gene Therapy Catapult and comprises partners in industry, academia and healthcare providers and three regional UK centres: Innovate Manchester Advanced Therapy Centre Hub (iMATCH); the Midlands-Wales Advanced Therapy Treatment Centre (MW-ATTC, comprising Birmingham, Bristol, Cambridge, Cardiff, Leicester, Nottingham and Swansea); the Northern Alliance Advanced Therapies Treatment Centre (NA-ATTC, comprising Edinburgh, Glasgow, Leeds and Newcastle). The network was established through funding from the Industrial Strategy Challenge Fund, and the fund is delivered by UK Research and Innovation. For more information please visit theattcnetwork.co.uk.

About London Advanced Therapies

London Advanced Therapies (LAT) brings together the London scientific community working in the field of Cell and gene based therapies. Funded by research England and led by Kings College London, Imperial College London and University College London, LAT aims to catalyse Londons capabilities and outputs in the area of advanced therapies, through fostering collaborative work, facilitating commercial partnerships and creating a microclimate for innovation. Established in 2019, LAT is rapidly expanding, working with colleagues throughout the UK to establish a nationwide Network of Networks.

About the Cell and Gene Therapy Catapult

The Cell and Gene Therapy Catapult was established as an independent centre of excellence to advance the growth of the UK cell and gene therapy industry, by bridging the gap between scientific research and full-scale commercialisation. With more than 350 employees focusing on cell and gene therapy technologies, it works with partners in academia and industry to ensure these life-changing therapies can be developed for use in health services throughout the world. It offers leading-edge capability, technology and innovation to enable companies to take products into clinical trials and provide clinical, process development, manufacturing, regulatory, health economics and market access expertise. Its aim is to make the UK the most compelling and logical choice for UK and international partners to develop and commercialise these advanced therapies. The Cell and Gene Therapy Catapult works with Innovate UK. For more information please visit ct.catapult.org.uk or visit http://www.gov.uk/innovate-uk.

About the Industrial Strategy Challenge Fund

This project has been funded by the Industrial Strategy Challenge Fund, part of the governments modern Industrial Strategy. The fund is delivered by UK Research and Innovation.

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Launching today: eLearning programme to support advanced therapy training in the NHS, UK universities and government bodies - PharmiWeb.com

DetailsCategory: AntibodiesPublished on Tuesday, 20 July 2021 18:19Hits: 299

Abelacimab achieved a ~80% reduction in venous thromboembolism versus a standard of care comparator in gold standard proof-of-concept efficacy study, indicating its potential in a range of thromboembolic disorders

CAMBRIDGE, MA, USA I July 19, 2021 I Anthos Therapeutics, a clinical-stage biopharma company developing innovative therapies for cardiovascular and metabolic diseases, today announced final results from the Phase 2 ANT-005 study with its novel investigational anticoagulant abelacimab. Published today in the New England Journal of Medicine,1 and simultaneously presented as a late breaker at the International Society of Thrombosis and Haemostasis (ISTH) 2021 Congress, the data showed that a single postoperative dose of abelacimab reduced the rate of venous thromboembolism (VTE) by ~80% compared to enoxaparin (a commonly used low molecular weight heparin), following elective total knee arthroplasty, the gold standard setting for potential new anticoagulants to demonstrate efficacy.

In this parallel group study, 412 participants were randomly assigned to one of three single postoperative intravenous doses of abelacimab (150mg, 75mg or 30mg) in a blinded fashion or open-label standard of care enoxaparin given subcutaneously 40mg once daily for approximately 10 days after surgery. The primary composite efficacy outcome which included deep vein thrombosis detected by venography of the operated leg and documented symptomatic VTE events occurred in4%, 5% and 13% of patients in the 150mg, 75mg and 30mg abelacimab groups respectively, compared with 22% of patients in the enoxaparin group. The 75mg and 150mg abelacimab regimens were both statistically superiorto enoxaparin(p<0.001) while the 30mg dose was non-inferior. Abelacimab was well tolerated with no safety signals and bleeding was insignificant in both study arms.

"The results of this study provide exciting new evidence that inhibition of Factor XI appears an effective way to reduce the risk of pathological thrombosis in this case, with a single post-operative dose of abelacimab. The data highlight the potential of this new approach in other clinical settings where the unmet clinical need is high," observed JeffreyI. Weitz, MD, Professor at McMaster University, Hamilton, Ontario, and one of the study authors.

Abelacimab is a highly selective, fully human monoclonal antibody with novel dual activity against both Factor XI and its activated form, Factor XIa, achieving profound Factor XI suppression for up to 30 days following a single intravenous or subcutaneous dose.1,2

Beyond the compelling efficacy data shown in this study, Anthos' vision in developing abelacimab is to achieve 'hemostasis-sparing' anticoagulation: effective protection from thromboembolic events with a reduced risk of clinically significant bleeding. According to a recently described model of the coagulation cascade,3 Factor XI plays an important role in the development of pathological thrombosis but is hypothesized to play only a minimal role in physiological hemostasis. Factor XI inhibition thus provides a potentially significant opportunity to pharmacologically 'uncouple' the two pathways.

Dan Bloomfield, MD, Chief Medical Officer at Anthos Therapeutics, explained: "All current anticoagulants including direct oral anticoagulants (DOACs) impact physiological hemostasis as well as pathological thrombosis due to their action on the 'common pathway' of the coagulation cascade, thus carrying a well-documented bleeding risk which may be even greater in the real world than in clinical trials.4,5 Fear of bleeding commonly deters prescribers and patients from pursuing optimal anticoagulation6-8soour aim in developing abelacimab is to address this major unmet need." The ongoing Phase 2 ANT-006 study (AZALEA-TIMI 71), investigating long-term once-monthly subcutaneous administration of abelacimab for stroke prevention in patients with atrial fibrillation, is expected to provide insight on the bleeding risk with abelacimab compared to a commonly used DOAC.

Anthos Therapeutics' Chief Executive Officer, John Glasspool, commented: "More than 1 in 4 people worldwide continue to die from thromboembolic events and yet 40-50% of high-risk individuals fail to receive optimal anticoagulation, mainly due to the prevailing fear of bleeding. Factor XI inhibition may provide a paradigm shift towards safer anticoagulation that inspires greater confidence among prescribers and patients." Mr. Glasspool added: "I am proud of the progress we have made to address unmet needs in high-risk cardiovascular and metabolic conditions since we launched two years ago with investment from Blackstone Life Sciences. The efficacy findings announced today represent the first major milestone in our development plans for abelacimab."

ENDS

1. Verhamme P et al. New Engl J Med 2021, in press2. Yi BA et al. ISTH 2021 poster PB00773. Hsu C et al. J Am Coll Cardiol 2021, in press4. Buderi R et al. ISTH 2021 poster PB00475. Fox, KAA et al. BMJ Open 2017; Dec 21;7(12):e017157 6. Hsu JC et al. JAMA Cardiol. 2016; 1: 5562.7. Piccini JP et al. Circulation. 2019; 139:14971506.8. Cohen AT et al. Lancet 2008; 2;371(9610):387-94

ABOUT ANTHOS THERAPEUTICSAnthos Therapeutics is a clinical-stage biopharmaceutical company focused on the development and commercialization of genetically and pharmacologically validated innovative therapies to advance care for people living with cardiovascular and metabolic (CVM) diseases. Anthos Therapeutics aims to combine the agility of a biotech with the rigor of a large pharmaceutical company.Anthos Therapeutics was launched by Blackstone Life Sciences in 2019.

For more information: https://www.anthostherapeutics.com/

About Blackstone Life SciencesBlackstone Life Sciences is an industry-leading private investment platform with capabilities to invest across the life cycle of companies and products within the key life science sectors. By combining scale investments and hands-on operational leadership, Blackstone Life Sciences helps bring to market promising new medicines that improve patients' lives. More information is provided athttps://www.blackstone.com/our-businesses/life-sciences/.

SOURCE: Anthos Therapeutics

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Anthos Therapeutics' novel Factor XI inhibitor abelacimab significantly outperforms standard of care enoxaparin in prospective Phase 2 efficacy...

Louis Fourie

ALTHOUGH the pharmaceutical industry is constantly evolving and new therapeutic approaches are being developed, one aspect has not changed: the use of small, synthetic molecules, which still account for 90 percent of the therapeutics on the pharmaceutical market. Some of the top-selling drugs are small-molecule drugs.

Small-molecule drugs

A small-molecule drug is an organic compound of low molecular weight (less than 900 Daltons or 1,49449-21 grams) and the size of about one nanometre capable of modulating biochemical processes to diagnose, treat or prevent diseases.

Because of their low molecular weight, small-molecule drugs have some definite advantages as therapeutics, because most can be administered orally and can pass through cell membranes to reach intracellular targets. Once inside the cells, they can be designed to engage biological targets, such as proteins, by various modes of action. Their distribution can also be customised, for example, to allow for systemic exposure or to destroy cancer cells. Therefore, many targeted therapies today are small-molecule drugs made from synthetic chemical reactions. However, they are also used in pesticides and in many other roles.

One well-known small-molecule drug is aspirin, which has been with us since 1899. Currently, the world consumes about 40000 tons of aspirin every year for a range of indications, such as cardiovascular health, Alzheimers disease, cancer treatment, pulmonary diseases, and everyday aches and pains. Another classic example is the drug penicillin, which successfully reduced the death rate caused by bacterial-related pneumonia to less than 1 percent during World War II.

Although small-molecule drugs have dominated the pharmaceutical industry since the beginning of modern medicine, it seems as though major developments are now happening in biologics, despite their excessive cost. RNA interference and CRISPR-Cas9, for example, are exciting new gene-editing tools.

But it is not the end of small-molecule medicine. Recent discoveries of small molecules that can modulate protein-protein interactions have created renewed interest in and utilities for small-molecule drugs for many diseases.Furthermore, the ability to design small molecules that can interact with and modulate RNA could create new opportunities for targeting challenging disease pathways.

An anti-ageing drug

Although with us for many years, the rapid advancement of biopharmaceutical research and technology opens up many opportunities for inventive and ingenious approaches to developing small-molecule drugs. We have therefore in recent years seen that significant advancements in structure-based design and imaging, together with automation, artificial intelligence and machine learning, have become important enablers to expedite research and enhance the success rate of small-molecule-led optimisation.

One of these innovative small-molecule drugs that could delay ageing is being tested by the US Militarys Special Operations Command (Socom), the organisation that controls the USs Special Operations forces. The anti-ageing pill comprises a human performance small molecule in the form of a nutraceutical with the aim of improving performance characteristics such as endurance and faster recovery from injuries, which typically declines with age.

Socom is working with the private biotech laboratory Metro International Biotech (MetroBiotech) on the development of an anti-ageing pill based on a human performance small molecule.

MetroBiotech is an offshoot of David Sinclairs Harvard University Medical School laboratory. The first-in-class small molecule on which they are working is Nicotinamide Mononucleotide (NMN), which MetroBiotech describes on their website as an enhancer that leverages the nicotinamide adenine dinucleotide (NAD+) cycle that is critically important to the function of all living cells and also to treat rare mitochondrial diseases and other medical conditions. These rare mitochondrial diseases often have serious effects on skeletal and cardiac muscle, as well as the central nervous system.

Increased NAD+ levels have been shown to induce mitochondrial biogenesis and enhance natural pathways (for example, sirtuins a family of dormant proteins found in all living beings) that are key to improving mitochondrial health. Mitochondria are cell parts (organelles) that produce energy for the cell in the form of a chemical called adenosine triphosphate (ATP). Cells need ATP to function properly, and NAD+ is a cofactor required for the enzymatic processes that generate energy within the cell through the continuous production of ATP inside the mitochondria.

Research has demonstrated the broad therapeutic potential of increasing NAD+ levels to preserve health and normal metabolism. It increases mitochondrial health and longevity, rejuvenates stem cells and provides neuroprotection. In general, it improves the health of most organs such as the brain (neurodegeneration), heart (inflammation, cardio protection), liver fatty acid oxidation, gluconeogenesis or regeneration), pancreas (insulin secretion), and skeletal muscles (insulin sensitivity, fatty acid oxidation) and white adipose tissue or white fat (lipogenesis).

Ageing and certain diseases, such as mitochondrial dysfunction, inflammation and other associated diseases, cause a decline in the NAD+ levels in humans, with serious consequences with regard to energy, performance and endurance. Treating people with NAD+ could thus slow the degenerative effects of ageing, prevent the onset of injury and thus allow people, according to MetroBiotech, to lead longer and healthier lives.

Several studies have been published in respected journals indicating how supplementation with a NAD precursor delays motor neuron degeneration, decreases markers of neuroinflammation in the spinal cord, improves blood flow, heart pathology, and musculoskeletal endurance, slows Alzheimers, improves energy metabolism, insulin sensitivity and plasma lipid profile, reverses retinal degeneration, mitochondrial biogenesis in skeletal muscle, and prevents noise-induced hearing loss and spiral ganglion neuron degeneration after noise exposure.

Since the small-molecule drug is a nutraceutical, a dietary supplement or food containing health-giving additive with medicinal benefit, it is not regulated by the US Food and Drug Administration and is therefore exempt from the rigorous standards regulating prescription drugs. It seems that MetroBiotech is following the nutraceutical route and not that of a prescription drug, because ageing is not yet itself a diagnosable disease to be treated by a prescription. Still, Socom have finished pre-clinical safety and dosing studies, and will soon start performance testing of the anti-ageing pill in clinical trials.

The thinking behind the anti-ageing pill is to address the cause of mitochondrial diseases and cure them all at once instead of the current repetitious and often futile one-by-one approach. According to MetroBiotech, its technology also supports key organ functions and could slow neurodegeneration, decrease inflammation in the body, increase cardio protection and improve sleep. NAD+ can apparently also reduce the functional effects of ageing on the human body, such as speed and reaction time.

A pipedream or a breakthrough?

For as long as humans have existed, they have been on a quest for a magic substance that would extend life or even bestow immortality. Over the years, the medieval alchemists and many others pursued the elixir of life. The slowing or prevention of ageing has therefore long been the Holy Grail of medicine, but has largely evaded us until now, except for a few studies in their infancy, such as Israeli oxygen therapy to increase telomere length and decrease the number of senescent cells (geriatric cells that can no longer divide).

The anti-ageing pill of MetroBiotech that slows down ageing and keeps you young may therefore sound like a sci-fi story. We will thus have to wait for the clinical trials to prove that NAD+ can indeed do what MetroBiotech is claiming it can do. However, the US military has spent serious money on this nutraceutical about R41 million since 2018. It must thus indeed be a promising drug that could become a game-changer in slowing the effects of ageing and preventing the onset of injuries. Despite being a controversial figure and often criticised, Sinclair may indeed be on the verge of a public health breakthrough that we have never seen before.

See you at our 150-year party!

Professor Louis CH Fourie is a technology strategist.

*The views expressed here are not necessarily those of IOL or of title sites.

BUSINESS REPORT ONLINE

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Tech news: Soon you will be able to stay young forever By Louis Fourie Jul 19 - IOL

Variations on a common tablet design, which can be distinguished by both colour and shape. Photo by Ragesoss (CC BY-SA 2.0)

Insulin costs around $6,000 annually for an insured individual in the U.S., a consequence of the countrys for-profit healthcare system. To get around this aspect of the government not enacting a fair and equitable healthcare system, alternative ideas are being considered. In relation to insulin, one such idea is the collective ownership by diabetics of the therapy.

It has been more than 40 years since drug development has changed from systems (heart, liver, spleen) to molecular medicine (targeted therapies). However, the pharmaceutical business model has not significantly shifted.

In relation to insulin specifically, the most commonly used forms of insulin is estimated to cost ten time more in the U.S. than in any other high-income country. The Big Three pharmaceutical companies that produce 90 percent of the insulin in the U.S. are Eli Lilly, Novo Nordisk, and Sanofi Aventis.

The situation for many diabetics is precarious. For example: You dont know if you will have enough of a freaking liquid that your whole life depends on. You dont know if you have enough life. Thats what being not sure if you can afford your insulin means said Marina Tsaplina in a Business Insider article.

Yet there are alternative models, provided that the pharmaceutical world is prepared to innovate. This requires the need to avoid duplication and internal conflicts across multi-billion-dollar organizations discourage initiative and create an unsupportable level of overhead.

Increasing innovation will require a complete restructuring of the industry and other technology-driven industries have undergone multiple generations of change. All industries and business models follow the law of diminishing returns, and many industries have come and gone through history. In fact, the pharma industry itself sprouted out from the terminal decline of the chemicals and dye industry as it was slowly commoditized.

A new breakthrough with insulin treatment could be one funded by diabetics who believe in it and stand to benefit the most from it.

The idea is for a new type of organization, and such an organization has been formed. This is a DAO (Decentralized Autonomous Organization) that exists on a blockchain. Such an organization is decentralized and therefore does not have a central point of failure.

VitaDAO is the first organization to tackle biomedical research. Thorough the use a token, the process it provides patients with a way to get governance over the intellectual property in the medicines they need. With this DAO, those working on new therapies and anyone who provides valuable work or resources can be financially empowered through tokens, becoming an active participant.

The idea is that by decentralizing intellectual property ownership the investment and capital injections can happen at an earlier stage and new open commercialization models can incentivize their development.

The VitaDAO model enables fundraising through alternative routes. Here, the public can get mobilized towards a new approach to medicine, thats not designed to keep them buying drugs to just manage the symptoms of the chronic diseases that are associated with age, but rather prevent cellular degeneration, which is the major risk factor for most chronic diseases. Cell and gene therapies, regenerative medicine approaches have the potential to completely change the way the world provides medical care.

The aim is for the experimental architecture to solve some of the problems presented, and in the long run perhaps all of them by creating entirely new, open, intellectual property business models.

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Disrupting the pharma development process with blockchain - Digital Journal

CAMBRIDGE, Mass. & NEW YORK--(BUSINESS WIRE)--Oncology practices will now be able to order and track Foundation Medicine, Inc.s comprehensive genomic profiling (CGP) tests for their patients without leaving Flatiron Healths OncoEMR platform, the two companies announced today.

This integration, the first of a series planned by Flatiron, will support more efficient clinical decision making by allowing electronic ordering, order tracking and receipt of Foundation Medicines CGP test results all within the OncoEMR platform. Almost all oncology practices use an electronic medical record (EMR) system to input, view and manage the full patient medical record in a single location, replacing a traditional paper chart with a digital one.1 EMR platforms also support clinical teams by enabling them to more efficiently order and track tests, view results, communicate treatment plans to patients and enable the completion of charting, documentation, and billing.

With the number of targeted treatments growing exponentially, the opportunity for cancer care transformation has never been greater. Clinicians increasingly rely on genomic insights to guide clinical decision-making, and Foundation Medicine is committed to implementing new solutions that enable widespread access to CGP, said Kathleen Kaa, Interim Chief Commercial Officer at Foundation Medicine. The integration of Foundation Medicine tests into OncoEMR, and other leading EMR systems to follow, is just one way were improving our offerings to fuel precision medicine for cancer patients. The integrations will create efficiencies for oncology healthcare teams to deliver precision treatment plans based on individual genomic insights to their patients.

"We are excited to welcome Foundation Medicine in the first of our planned CGP integrations with OncoEMR, said James Hamrick, MD, MPH, Vice President, Clinical Oncology at Flatiron Health. This kind of integration marks an important milestone in advancing precision medicine, helping oncologists have access to the information they need to select therapies.

The two companies are planning similar integrations with other CGP platforms and EMRs, respectively, in the oncology space, with the goal of helping every patient to realize the benefit of precision cancer care. These workflow-streamlining integrations are being designed by clinical and product experts in partnership with oncology practices.

About Foundation Medicine

Foundation Medicine is a molecular information company dedicated to a transformation in cancer care in which treatment is informed by a deep understanding of the genomic changes that contribute to each patient's unique cancer. The company offers a full suite of comprehensive genomic profiling assays to identify the molecular alterations in a patients cancer and match them with relevant targeted therapies, immunotherapies and clinical trials. Foundation Medicines molecular information platform aims to improve day-to-day care for patients by serving the needs of clinicians, academic researchers and drug developers to help advance the science of molecular medicine in cancer. For more information, please visit http://www.FoundationMedicine.com or follow Foundation Medicine on Twitter (@FoundationATCG).

Foundation Medicine is a registered trademark of Foundation Medicine, Inc.

About Flatiron Health

Flatiron Health is a healthtech company dedicated to helping cancer centers thrive and deliver better care for patients today and tomorrow. Through clinical and data science, we translate patient experiences into real-world evidence to improve treatment, inform policy, and advance research. Cancer is smart. Together, we can be smarter. Flatiron.com @FlatironHealth

OncoEMR is a registered trademark of Flatiron Health.

1 2019 Genentech Oncology Trend Report. 11th ed. San Francisco, CA: Genentech; 2019: 16. Available at: https:/www.genentech-forum.com/content/dam/gene/genentech-forum/pdfs/genentech-oncology-trend-report-2019.pdf. Accessed June 22, 2021.

Source: Foundation Medicine

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Foundation Medicine and Flatiron Health Announce First-of-its-Kind Integration of Genomic Profiling Into OncoEMR - Business Wire