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Better Precision Medicine Outcomes Shown for Leukemia

DNA analysis graphic

(Gerd Altmann, Pixabay)

27 Oct. 2020. Clinical trial results show a strategy that first tests for genomic mutations to guide treatments results in better outcomes for patients with acute myeloid leukemia. Findings from the Beat AML Master Clinical Trial sponsored by the Leukemia and Lymphoma Society, or LLS, in Rye Brook, New York appear in yesterday’s issue of the journal Nature Medicine (paid subscription required).

Acute myeloid leukemia or AML is a cancer of the blood and bone marrow that worsens quickly if left untreated. As the disease develops, bone marrow produces abnormal white blood stem cells called myeloblasts that do not mature into normal functioning white blood cells. The excessive growth of abnormal myeloblasts crowds out healthy white, red, and platelet blood cells, and can spread to other parts of the body. Leukemia and Lymphoma Society says acute myeloid leukemia affects some 21,000 individuals in the U.S. each year, leading to about 10,000 deaths.

Because of the need to start treatment quickly to counteract the rapid spread of AML, most adult patients begin treatments, usually with chemotherapy, as soon as possible after diagnosis. The Beat AML trial is testing a different strategy that first takes a genomic test of the patient’s cancer, returning data on the cell DNA and mutations within seven days. The patients’ physicians then use those data to guide selection of treatments to precisely match the AML’s genomic signature.

The early- and mid-stage clinical trial is enrolling 2,000 participants age 60 and older with AML. The study team is looking primarily at the feasibility of conducting a genomic analysis of patients’ AML within seven days to guide treatment decisions, as well as effects on the clinical response rate of patients to those treatments. Participants in this study receive the genomic test before starting treatment, and then decide to proceed with treatments that match the mutation, or other treatments including standard chemotherapy. Foundation Medicine, a company in Cambridge, Massachusetts providing cancer genomic assessments, performs the tests.

The Nature Medicine paper reports on the nearly 400 patients enrolled in the trial by 30 January 2018. Of the initial 395 participants, 374 or 95 percent were successfully analyzed for genomic properties of their AML within seven days of their diagnosis. Of that sample, 224 participants used the precision medicine strategy from the genomic analysis to guide their treatments, from 11 different therapies. The remaining patients chose the standard chemotherapy — 103 participants, usually a combination of cytarabine and daunorubicin — or opting for pallative care or another clinical trial.

The results show participants selecting the precision medicine treatments guided by genomic testing had lower 30-day mortality and longer overall survivial time than patients selecting standard chemotherapy. Precision medicine participants had a median survival time of 12.8 months compared to 3.9 months for standard chemotherapy recipients.

“The study shows that delaying treatment up to seven days is feasible and safe,” says the paper’s senior author John Byrd, chair of leukemeia research at Ohio State University in an LLS statement, “and that patients who opted for the precision medicine approach experienced a lower early death rate and superior overall survival compared to patients who opted for standard of care.” Byrd adds the study shows that “we can move away from chemotherapy treatment for patients who won’t respond or can’t withstand the harsh effects of the same chemotherapies we’ve been using for 40 years and match them with a treatment better suited for their individual case.”

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Lilly, NIH Halt Covid-19 Antibody Therapy Trial

Stop signs graphic

(kropekk_pl, Pixabay)

27 Oct. 2020. Drug maker Eli Lilly and National Institutes of Health are stopping a clinical trial testing a synthetic antibody to treat hospitalized Covid-19 patients. The trial sponsors say results so far show little clinical value to hospitalized patients from the Lilly synthetic monoclonal antibody therapy bamlanivimab, previously code-named LY-CoV555.

Eli Lilly and Co. in Indianapolis says in a company statement the decision to stop the trial of hospitalized patients does not apply to its other clinical studies of bamlanivimab testing the synthetic antibody among Covid-19 patients: those recently diagnosed with mild to moderate symptoms, non-hospitalized patients, residents and staff in long-term care facilities, and in combination with other drugs.

Bamlanivimab was initially developed by biotechnology company AbCellera Biologics in Vancouver, British Columbia as an immunoglobulin G antibody designed to block the SARS-CoV-2 spike protein that penetrates and infects cells. AbCellera says it screened more than 5 million immune-system cells against a blood sample from one of the first people in the U.S. infected with the novel coronavirus, as it was called at the time.

From this screening, AbCellera says it identified some 500 unique human antibody sequences that respond to the SARS-CoV-2 virus responsible for Covid-19 infections. Lilly and AbCellera partnered with the Vaccine Research Center at National Institute of of Allergy and Infectious Diseases, or NIAID, part of National Institutes of Health, to identify the best-performing candidates.

NIAID is also the co-sponsor with Lilly of the now-halted clinical trial. The study is part of NIH’s Accelerating COVID-19 Therapeutic Interventions and Vaccines, or ACTIV, initiative, a partnership with pharma and biotechnology companies, and other federal agencies, to coordinate and speed Covid-19 therapies and vaccines. The ACTIV-3 trial, in this case, is enrolling 10,000 participants at 51 hospital sites testing bamlanivimab with the antiviral drug remdesivir against a placebo.

The trial uses an adaptive design with a master protocol that allows trial sponsors to add or remove new drugs for testing, as well as adjust the populations tested in the trial as data are collected. The study team planned to first enroll 300 hospitalized Covid-19 patients, then after five days assess any changes in their clinical status using a standard ordinal scale. If the test agent, bamlanivimab in this case, was shown safe and effective, more participants would be recruited. Participants would be monitored for another 90 days, after their release from the hospital.

NIAID says an independent data and safety monitoring board reviewed the ACTIV-3 data after enrolling 326 participants, and recommended that the study enroll no more participants. However, all those enrolled will continue to be monitored for 90 days, as planned.

Earlier in October, that same data and safety monitoring board paused the ACTIV-3 trial when a safety issue emerged, described by NIAID in an institute statement as “a pre-defined boundary for safety was reached at day 5.” NIAID notes, however, “differences in safety outcomes between the groups were not significant in the updated data set and the Oct. 26 decision was driven by lack of clinical benefit for LY-CoV555 in a hospitalized population.”

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Think About Your Real Business Needs

– Contributed content –

Business desktop

(Rawpixel, Pixabay)

27 Oct. 2020. There are a lot of challenges involved in running any kind of business but if there’s one thing that many business owners seem to struggle with more than just about anything else, it’s the fact that they aren’t able to separate themselves from their business. Now, having a lot personally invested in your business is a good thing. It’s always good to be dedicated to your business but that can often become an issue if you reach a point where you’re unable to draw that all-important distinction between what is best for you and what is best for your business.

When it comes to every decision that you make for your business, one of the most important things that you have to consider is whether a decision you make is based on what you want or what your business needs. If you don’t do that, your personal feelings are going to get in the way of you running your business as effectively as possible and will almost certainly cause issues down the line. With that in mind, here are some things that your business needs that might not always be that easy to identify.

The right equipment

One thing that everyone should know if they’re looking to succeed in any business sector is that if you want to get a job done right, you need the right tools. The right equipment can often be the difference between a business with a competitive edge in the modern market and one that ends up getting left in the dust. Look at your business and think really carefully about the tools and equipment that you actually need.

Whether you’re working in manufacturing and you need everything from carbide end mills to safety equipment in the warehouse, to the software needs of a business that’s based on the internet. The industry that you work in and the work you do will always impact the equipment you need but you should never underestimate the importance of having the right tools for the job.

The right people

There are a lot of things in a business that are incredibly important but if there’s one thing that could well be the most important of all it would be your employees. Your employees are the lifeblood of your business. They’re the ones who turn your plans and ideas into something real and tangible. They really can make a business. However, this is only the case if you’re working with the right employees who can help you to push your business forward. Finding the right employees is no easy task and can often take a lot of time and effort but, in the end, it’s always worth it.

Trying to rush through your recruitment process or not really considering it carefully enough is a good way to make sure that you end up with employees who just aren’t the right fit for your business. Carefully vetting every candidate is essential because you don’t want to end up with people who interview really well but just end up letting your business down when it really counts. Working with recruitment agencies might be the best option if you’re looking to find the best possible candidates every time.

A plan

One of the most common mistakes that a lot of new business owners tend to make is that they assume that they can just kind of wing it when it comes to running a business. Anyone who does this is in for a pretty rude awakening sooner rather than later. The reality is that running any kind of business, no matter the size, is an extremely complicated endeavour and if you’re not willing to take the time to plan things out, you’re just going to fall flat on your face. You need to have a plan for just about every aspect of your business, big and small, long and short-term.

Sure, a degree of flexibility is always a good thing but think of your business plan like the scaffolding that you’re building on top of. You don’t need to stick to it obsessively but it gives you a rigid skeleton that allows you to be flexible without your entire business collapsing altogether. A strong business plan can often make the difference between a business that succeeds and a thousand that wallow in obscurity.

A purpose

This might sound a little odd at first but it’s shockingly common for people to start their own business without ever actually asking “why does this need to exist?” You need to be brutally honest with yourself and ask if there is actually a market for your business. What is it offering that no one else is? Are you providing a totally new product? Is your focus on greater levels of customer service than the competitors? Are you simply able to offer the best value on the market? If you can’t identify a reason why customers should absolutely choose your business over any other, then it’s a good idea to head back to the drawing board to figure out what it is that your business has that everyone else lacks.

Now, this is not to say that your emotional investment in your business is always going to be a bad thing. In reality, you are the owner of the business which means that you need to be able to make tough decisions, often to pretty tight deadlines. This means that sometimes going with your gut is the best thing that you can do. The issue is when you start letting your emotions drive every decision you make without being as informed as possible as to what the right choices for your business actually are. The success of your business should always be the priority and one of the worst things that you can do is to let your ego get in the way of things. Being able to put your personal feelings aside and do what’s best for your business is often the key to being able to truly succeed in the modern business world.

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Bayer Gains Gene Therapy Biotech in $4B Deal

AskBio Bioreactor

AskBio Bioreactor (Bayer AG)

26 Oct. 2020. Global drug maker Bayer AG is acquiring the company Asklepios BioPharmaceutical, developer of gene therapies to treat inherited disorders. Under the agreement, Asklepios Bio, or AskBio, could receive as much as $4 billion if all terms of the deal are fulfilled.

AskBio, in Research Triangle Park, North Carolina, designs treatments for genetic diseases, as well as diseases where genetic factors interact with environmental factors that contribute to their severity or frequency. The company’s work is based on pioneering research on adeno-associated viruses by one of its founders Jude Samulski, beginning as a graduate student in the 1970s and 80s. The adeno-associated virus or AAV is a benign and naturally occurring microbe that infects cells, but does not integrate with the cell’s genome or cause disease, other than at most mild reactions. As a result, the virus has become a workhorse for delivering healthy genes to treat inherited diseases.

For gene therapies, AskBio creates engineered AAVs by inserting a healthy human gene into the virus’s genome for delivery to human cells or tissue, as well as removing problematic viral components. In some cases, AskBio’s AAVs can deliver two genes at once. The company further modifies AAVs by designing their capsids, the virus’s protein shell, to carry out tasks such as identifying targets, performing navigation, and controlling dosage for therapeutic delivery. The company says it uses bioinformatics, computer modeling, and crystal structure analytics to design these viral capsids.

AskBio says it maintains a library of engineered capsids that can be assembled to perform specified tasks as needed by therapy designers. The company also maintains a library of components it calls promoters, also designed with bioinformatics, that control gene expression, and provide better targeting of cells and tissue. AskBio’s pipeline today includes gene therapies for the rare inherited disorder Pompe disease, Parkinson’s disease, and congestive heart failure, all in early- or mid-stage clinical trials. Treatments for other neuromuscular, neurological, or metabolic disorders are in preclinical stages.

The acquisition gives Bayer, headquartered in Berlin, full rights to AskBio’s gene therapy platform, as well as contract management and manufacturing capabilities. In addition, Bayer gains AskBio’s full intellectual property portfolio that AskBio says exceeds 500 patents. AskBio is receiving $2 billion in an initial payment, and the company is eligible for another $2 billion on completion of unspecified milestones, with the vast majority of those achievements expected in the next five years.

Bayer says AskBio will continue to operate as an independent entity on an arm’s-length basis from Bayer’s corporate headquarters. Bayer expects AskBio’s gene therapies to complement stem cell therapies for regenerative medicine gained from its acquisition last year of BlueRock Therapeutics, initially formed as a joint venture of Bayer and Versant Ventures, as we reported in 2016.

“As part of our strategy, we are building new therapeutic platforms including cell and gene therapies,” says Stefan Oelrich, head of Bayer’s pharmaceuticals division, in a statement. “As an emerging leader in the rapidly advancing field of gene therapies, the expertise and portfolio of AskBio supports us in establishing highly innovative treatment options for patients and further strengthens our portfolio.”

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Precision Drug Trial Shows Solid Tumor Shrinkage

Cancer on clipboard

(Nick Youngson,

26 Oct. 2020. Partial results from a clinical trial show a drug to stop specific cancer-causing proteins shrinks advanced tumors in some patients across several tumor types. The findings were reported yesterday at the online Symposium on Molecular Targets and Cancer Therapeutics, sponsored by European Organisation for Research and Treatment of Cancer, or Eortc, American Association for Cancer Research, and National Cancer Institute, part of National Institutes of Health.

Researchers from MD Anderson Cancer Center in Houston, part of the University of Texas system, and colleagues from other institutions, reported on a clinical trial testing the drug code-named PLX8394, designed to block actions that result from mutations in the BRAF gene. The BRAF gene codes for proteins that help transmit chemical signals from outside cells to the cell nucleus, and are part of a larger RAS/MAPK pathway regulating many basic cellular functions.

The BRAF gene is known as an oncogene, which means it can mutate and produce proteins that cause normal cells to become cancerous. BRAF gene mutations are associated with many common and rare solid tumor cancers, such as melanoma on skin, gliomas in the brain, and ovarian cancer, as well as multiple myeloma, a blood-related cancer. Earlier BRAF inhibitors, say the researchers, work for only a limited set of solid tumor cancers and can become resistant to treatment.

“The next generation BRAF inhibitor that we gave to patients in this trial was designed to avoid those problems,” says MD Anderson cancer research and senior author Filip Janku in an Eortc statement. “These results suggest that the combination of drugs we tested is relatively safe and may be effective for some patients.”

The biotechnology company Plexxikon, in Berkeley, California, developed PLX8394 as an oral drug to treat BRAF mutations, but work with more solid tumor cancers and avoid becoming resistant. PLX8394, says Plexxikon, activates the RAS/MAPK pathway to block signals from mutated BRAF proteins, and stop uncontrolled cell division caused by those signals. In June, Plexxikon licensed PLX8394 exclusively to Novellus, a precision cancer drug development company in Jerusalem, Israel, for further research, development, and commercialization. Researchers from Plexxikon and Novellus are among the authors of the conference paper.

The early- and mid-stage clinical trial is testing PLX8394 in patients with advanced solid tumor cancers resulting from BRAF gene mutations at 13 sites in the U.S. The study team aims to enroll 100 participants in the trial, age 12 and up with cancers that so far do not respond to treatment and cannot be surgically removed.

In the first part of the study, researchers are looking primarily for safe dosage levels of PLX8394. The trial’s second part is more complex, with participants divided into two groups: (1) those with glioma tumors and a specific sub-mutation, called V600, as well as no prior treatments targeting BRAF genes, and (2) patients with BRAF mutations other than V600 and who had previous BRAF-targeting treatments. The study team is looking for PLX8394’s safety and dosage levels in patients, but also chemical activity in the body and evidence of anti-tumor response.

In addition, some patients receive the drug cobicistat, approved as supplemental treatment for HIV infections that increases the amount of primary HIV drugs in patients. In this trial, the study team is looking to see if cobicistat also increases PLX8394 in cancer patients.

In their conference paper, the researchers say 75 patients are taking part in the trial so far, and report on 45 participants with results. Of those 45 patients, 10 or 22 percent report their tumors responding to the treatments, with shrinkage of at least 30 percent. Three of the 10 patients have gliomas, two have ovarian cancer, and others have melanoma, colorectal, or thyroid cancers. Participants taking cobicistat report two to three times as much PLX8394 exposure, compared to those not taking cobicistat.

All 10 patients have taken part in the trial for at least two years. The most serious adverse affect among some patients is higher than normal levels of bilirubin and liver enzymes in blood samples indicating possible liver damage. However, no skin lesions, reported by patients receiving earlier BRAF inhibitors, were seen in these patients.

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Should We Buy Electric Vehicles?

– Contributed content –

Jaguar EV

Jaguar EV (Ronnie Overgoor, Unsplash)

24 Oct. 2020. Electric vehicles are now easy to see everywhere you go. You might see them on the road, parked in driveways, or at the supermarket. In times gone by, this would have been unthinkable. There was no infrastructure for them, and the technology was never going to compete with gas-motor vehicles.

All of that has changed and relatively quickly. In the course of a decade, the EV market has grown significantly, but not in line with demand. In fact, the demand for EVs has outstripped supply all the way owing to a lack of charging stations and certain reluctance on the part of big brand car manufacturers to go all in.

Now the advantages are clear for everyone to see, and the charging stations are beginning to increase. Big brand car manufacturers are developing a variety of models to suit all styles and tastes, but is it time to ditch your gas-motor in favor of an EV? That’s the question we ask and try to answer in this article.

They’re cheaper but less convenient

If you go to buy an EV, you might be put off by the price; on average, an EV will cost you double that of a gas-powered vehicle. However, it’s clear that the investment pays off in the long term with massive reductions in the overall running costs and virtually no need to service the vehicle.

Unlike a gas-powered vehicle, an EV has no mechanical parts. It has no gearbox, no fly-wheel, no timing belt, and no oil to change. It does have a battery, which is usually sizable and located on the floor. Like all batteries, it eventually dies and will need replacing. This is the only significant expense of running an EV and is not likely to affect you for many years.

Despite this, the EVs are far less convenient. Public access points are limited, and it can be costly to adapt your home for charging them correctly. What’s more, the claim that EVs are zero-emissions may be false in some cases. They are clearly zero-emissions vehicles at the tailpipe since they don’t have one, but unless their electricity is 100% renewable, they won’t be zero emissions.

They perform well with limited range

When people think of an EV, they may be tempted to think of a vehicle that performs less-well than a gas-powered one. Perhaps they get this reputation from the vehicles of the past, the ones in developmental and experimental stages. These days, it might surprise some people that EVs outperform the gas-motors in many cases.

They have no mechanical parts, they are lighter, and they have excellent torque that can be delivered directly to the axle. All this means an EV has excellent acceleration that will race ahead of a gas-powered car quite easily. The other feature of EV technology, exuding the air cooled chiller, is the silence on the road. No engine means no noise, which divides opinion.

But it’s not all roses in the garden. While they are quiet and quick, they do suffer from a lack of range in comparison to traditional vehicles. The majority of EVs on the market will offer a range of between 150-250 miles depending on the model. The range will also be affected by the driving style; lots of accelerating, for instance, will run it down faster.

The range is also an issue depending on how you use your vehicle. Running an EV means you have to think about how far you’re traveling and if there are any charging stations along the way: this can be a deal-breaker for many individuals.

They don’t use gas, but they aren’t zero emissions

One of the big selling points of EVs is the fact they don’t use gas and are sold as zero-emissions vehicles. It is true that they don’t use gas, which is an excellent benefit, both for the planet and for your pocket. However, they are probably not zero emissions, at least not yet.

Not running on gas is a massive advantage of EVs. It’s also a disadvantage as it limits range and access to fuel, but it massively reduces the running costs and the vehicle’s environmental impact. There is no gas to burn, no oil in the car, and fluids like coolant and antifreeze aren’t necessary.

However, there is still an environmental impact from EVs that should not be overlooked or ignored. At present, only around 20% is made up of renewable energy, meaning that these vehicles are still causing harmful emissions from fossil fuels. There are no immediate traces of that as the car itself seems carbon-free, but they are still a net contributor to global warming.

The good news is that companies like Envision Solar are creating more 100% renewable charging stations. The good news is the demand for EVs is outstripping supply, and as the demand grows, the infrastructure will improve.

There’s a limited selection, but they’re good to lease

The market for EVs has been steadily growing since the 2010s, in-line with the environmental movement. A culture of conscious consumerism has developed, and people are beginning to see alternative vehicles’ viability. Despite this, the supply has not kept up, and there is a shortfall and lack of choice within the EV market.

In 2020 and beyond, however, this trend is set to change. More big brand car manufacturers are now developing EV models that compete with gas-motor alternatives in terms of style and performance. There are now EV sports vehicles, SUVs, and pickup trucks. As the range and infrastructure grow, there will be no excuse not to get one.

But buying an EV can be an expensive option. They are twice the price of gas-motors on average, which has led many conscious consumers to lease them instead. Leasing an EV allows you to ditch your gas-motor and go electric without the upfront cost. With an EV lease, you will have an all-electric vehicle for less than you would pay on even a gas-powered finance agreement.

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Infographic – Covid-19 Cases Reach New Highs

Global Covid-19 cases

Click on image for full-size view (Statista)

24 Oct. 2020. New Covid-19 infection cases are surging again in most parts of the world, with numbers accelerating fastest in Europe and the Americas. Those data are collected by World Health Organization and displayed in chart form on Thursday by business research company Statista.

The graph shows the moving seven-day average number of new Covid-19 cases reported by national health authorities since March; the seven-day average smooths out daily fluctuations sometimes influenced by local conditions, such as weekend closures. Cases in Europe — including European Union, Russia, U.K., and Israel — have climbed most markedly since September, with the Americas, notably the U.S. and Brazil, rising almost as quickly.

Covid-19 cases in South-East Asia that covers India, Bangladesh, and Indonesia, are also rising since the summer but not at the same rate as Europe and the Americas. And case counts in the Western Pacific region, which includes China and Japan, remain low.

As of today, WHO reports 41.8 million Covid-19 cases worldwide, with more than 238,000 new cases, and 1.1 million deaths since the pandemic began. The New York Times today reports more than 85,000 new cases in the U.S., from its counts of data from state and local health agencies.

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Phone Video Algorithm Screens for Stroke

Stroke screening test

Kathryn Atkinson, a patient at Houston Methodist Hospital, participates in a smartphone screening test to analyze stroke-like symptoms. (Houston Methodist)

23 Oct. 2020. A medical informatics team designed a computer model from patient videos to help emergency room physicians quickly diagnose stroke. Researchers from Pennsylvania State University in University Park and Houston Methodist Hospital in Texas describe the system in a paper at the International Conference on Medical Image Computing and Computer-Assisted Intervention, a virtual event held earlier this month.

The team led by Penn State bioinformatics professor James Wang is seeking a quick, reliable, and easy-to-use solution for physicians to identify stroke symptoms in emergency room patients. “When a patient experiences symptoms of a stroke, every minute counts,” says Wang in a university statement. “But when it comes to diagnosing a stroke, emergency room physicians have limited options: send the patient for often expensive and time-consuming radioactivity-based scans or call a neurologist, a specialist who may not be immediately available, to perform clinical diagnostic tests.”

Wang’s lab studies technologies for gaining knowledge more systematically from images, particularly artificial intelligence tools such as machine learning, and statistical modeling. For this task, the researchers aim to emulate standard clinical checklists — Cincinnati Pre-hospital Stroke Scale, and Face Arm Speech Test — used by neurologists to diagnose stroke, but to put these analytical tools in the hands of emergency room physicians, who may not to able to call in a neurologist to conduct these tests.

The Penn State researchers enlisted colleagues at Houston Methodist Hospital, who asked patients with stroke symptoms to participate. The team built a collection of videos from 80 Houston Methodist patients with suspected stroke symptoms, captured on iPhones, where participants completed a speech test. The researchers then used the videos to train machine-learning algorithms, combined with image analysis and natural language processing, for inspecting visual and audio evidence to find signs of stroke, such as facial motion weakness, slurred speech, and other speech disorders.

“The acquisition of facial data in natural settings makes our work robust and useful for real-world clinical use,” says Penn State information sciences professor and team member Sharon Huang, “and ultimately empowers our method for remote diagnosis of stroke and self-assessment.”

The researchers tested their algorithm analyzing smartphone videos with emergency room patients performing actual speech tests for stroke. The results show the computer model matches results of the speech tests, confirmed by CT scans, 79 percent of the time. In addition, the algorithm shows a true-positive sensitivity of 93 percent. And the computer model returned results in about four minutes.

John Volpi, a vascular neurologist at Houston Methodist and co-author of the paper, says emergency room physicians often use a binary method for diagnosing stroke, focusing more on patients with obvious symptoms, which may overlook stroke patients with less severe symptoms. “If we can improve diagnostics at the front end, then we can better expose the right patients to the right risks and not miss patients who would potentially benefit,” notes Volpi, who adds, “We have great therapeutics, medicines, and procedures for strokes, but we have very primitive and, frankly, inaccurate diagnostics.”

Penn State and Houston Methodist filed a provisional patent for the algorithm.

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Start-Up Developing Engineered B-Cell Therapies

Human B-cell

Human B-cell (NIAID, Flickr)

22 Oct. 2020. A new enterprise, spun off from Seattle Children’s Research Institute, is creating therapeutics for diseases from engineered B-cells in the immune system. Be Biopharma, in Cambridge, Massachusetts, is also raising $52 million in its first venture funding round.

Be Biopharma is commercializing work by immunologists Richard James and David Rawlings at Seattle Children’s Research Institute, who both study B-cells, white blood cells in the immune system that produce antibodies. James and Rawlings are founders of Be Biopharma, along with entrepreneur and venture investor David Steinberg. The company aims to take advantage of the natural ability of B-cells to produce proteins, while refining that process to generate working therapeutics for a range of diseases.

James’s lab at Seattle Children’s studies the biology and engineering of B-cells. The lab focuses on antibody-producing B-cells because of their ability to indefinitely produce proteins, and in quantities similar to many industrial cell lines. The lab’s researchers developed techniques for genetically engineering B-cells, which they say was previously a difficult process. James and colleagues are investigating the optimal conditions for transforming engineered B-cells into long-term antibody producers, and determining the impact of natural processes for cell recycling on B-cells, as well as effects of engineered B-cells on host immune systems.

“B cells play a key role in combating diseases by catalyzing humoral immunity, the arm of the immune system that manufactures large quantities of proteins to neutralize disease-causing pathogens and manipulate immune cell behavior,” says Rawlings in a Be Biopharma statement released through BusinessWire. “Our ambition is to advance the field by building a new class of engineered B cell medicines that will provide direct control over the power of humoral immunity and help transform the prognosis for patients who currently have limited treatment options.”

The company says its engineered B-cells can overcome some of the drawbacks of current cell and gene therapies, notably the inability to provide multiple doses, unpredictable chemical reactions in some individuals, and the need for chemotherapy regimens prior to cell therapies that can be dangerous to patients. Be Biopharma plans to apply its technology to range of diseases, such as cancer, autoimmune disorders, and infectious diseases.

Be Biopharma was formed by venture capital company Longwood Fund in Boston that specializes in life science and health care enterprises. “B cells can be engineered to express a wide variety of proteins, have the potential to generate durable responses, and can be dose-titrated and administered multiple times without the need for toxic preconditioning,” notes Steinberg, the company’s CEO. “Moreover, the varied functions of B cells suggest that B cell medicines can address a range of conditions including autoimmune diseases, cancer, and monogenic disorders, as well as enhance the immune response to infectious pathogens.”

Be Biopharma is raising $52 million in its first venture funding round, led by Atlas Venture and RA Capital Management. Joining the round are Alta Partners, Longwood Fund, and Takeda Ventures.

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Roche Gains Covid-19 Oral Drug Candidate

Pills and capsules

(stevepb, Pixabay)

22 Oct. 2020. Global drug maker Roche Group is acquiring an experimental antiviral therapy for Covid-19 infections taken as a oral drug, rather than an infusion. The deal provides Atea Pharmaceuticals Inc. in Boston, developer of the therapy code-named AT-527, $350 million in an initial payment, with more payments later as unspecified milestones are achieved.

Atea Pharmaceuticals develops antiviral prodrugs, precursor compounds that metabolize in the body to become active drug ingredients. The company says it has a library of some 1,000 prodrug candidates made of nucleic acid components that act against specific RNA polymerases, enzymes that help transcribe DNA to RNA and promote replication of the virus in the body. Atea says it refines these candidates into working therapies by enhancing their antiviral potency, selectivity, and tolerability as oral drugs.

The company says it’s advancing AT-527 as an oral drug to treat patients with Covid-19 infections. Atea first designed AT-527 as a treatment for hepatitis-C infections, where the company says early and mid-stage clinical trials demonstrate its safety. When its potential to treat coronavirus infections was discovered, Atea began testing AT-527 as a Covid-19 therapy. The company says its hepatitis-C studies show AT-527 is safe for patients for up to 12 weeks with a daily dose of 550 milligrams. The results also show the drug is absorbed quickly by patients and has a long shelf life.

Atea’s founder and CEO Jean-Pierre Sommadossi says in a statement, “AT-527 is expected to be ideally suited to combat Covid-19 as it inhibits viral replication by interfering with viral RNA polymerase, a key component in the replication machinery of RNA viruses. Importantly, the manufacturing process for our small molecule direct-acting antiviral allows us to produce AT-527 quickly and at scale.”

AT-527 is now being tested in a mid-stage clinical trial with hospitalized patients having moderate Covid-19 infections. The 190 trial participants are older, age 45 to 80, and have additional risk factors such as obesity, hypertension, or diabetes. Participants are randomly assigned to receive one or two tablets of AT-527, each 550 milligrams, or a placebo, on the first day, followed by one tablet a day for another four days. The study team is looking primarily for changes in participants’ respiratory capacity and blood oxygen levels, as well as signs of adverse effects from the drug. Researchers are also tracking time needed for patients to recover, or if they encounter respiratory failure or death.

Roche plans to evaluate AT-527’s potential among non-hospitalized Covid-19 patients as well. “By joining forces with Atea,” says Roche Pharmaceuticals’ CEO Bill Anderson, “we hope to offer an additional treatment option for hospitalized and non-hospitalized Covid-19 patients, and to ease the burden on hospitals during a global pandemic.” A late-stage trial is planned for the first quarter of 2021 testing AT-527 among non-hospitalized Covid-19 patients.

The agreement calls for Atea and Roche to collaborate on clinical trials and manufacturing of AT-527. Atea will be responsible for distribution of AT-527 in the U.S., although it has the option to call on Roche’s Genentech subsidiary if needed. Roche is responsible for distribution outside the U.S.

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