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Photo Algorithms Find Cancerous Melanoma Lesions

Phone photo

(tookapic, Pixabay)

17 Feb. 2021. An engineering team designed artificial intelligence techniques with image analysis to identify early signs of skin cancer in ordinary photographs. Researchers from Massachusetts Institute of Technology and the Wyss Institute for Biologically Inspired Engineering at Harvard University describe their techniques in today’s issue of the journal Science Translational Medicine (paid subscription required).

The team led by Luis Soenksen, a postdoctoral researcher at the Wyss Institute, is seeking simple and reliable ways for general practice physicians to identify melanoma in patients early on, while more treatment options are available. Melanoma is an aggressive type of skin cancer, which while not as common as basal cell and squamous cell skin cancers, is more likely to spread to other parts of the body. If melanoma is caught and treated early, before it spreads or metastasizes, the 5-year survival rate is 99 percent according to American Cancer Society. After the cancer spreads to other parts of the body, however, the 5-year survival rate drops to 27 percent.

Soenksen and colleagues sought to evaluate skin discolorations and growths using computerized techniques from photos taken with consumer-grade cameras, like those found on smartphones, and approached the problem much like trained dermatologists. These specialists use what they call the “ugly ducking” technique, looking for any moles or abnormalities known as suspicious pigmented lesions on a patient’s skin, then focus in on those with particularly problematic characteristics. The team discovered current computerized techniques assess one image at a time, which differs from physicians’ methods.

For their analysis, the researchers built a database of nearly 34,000 images made up of suspicious pigmented lesions and non-cancerous skin images from 133 patients in a hospital in Madrid, Spain, as well as publicly available images. The wide-field photos were taken with consumer-grade cameras and included a variety of backgrounds, such as different color painted walls or furniture fabrics to simulate real-world conditions.

Performing an ugly ducking analysis

The team used these images to write algorithms for a deep convolutional neural network. These algorithms combine image analysis and machine learning to dissect an image by layers for understanding features in the image. Different aspects of each layer discovered and analyzed by the system are translated into data that the algorithm then uses to train its understanding of the problem being solved, with that understanding enhanced and refined as more images and data are encountered.

To perform an ugly-duckling analysis, the researchers trained the neural network to evaluate images with groups of suspicious pigmented lesions and non-cancerous skin growths on patients. The team designed the algorithms to assess the problematic characteristics of a lesion in the context of a patient’s other growths or discolorations, not just individual images on their own.

The researchers validated the techniques by comparing evaluations of 135 photos from 68 patients made by their algorithms with assessments by three board-certified dermatologists. The results show the algorithm’s evaluations matched the dermatologists’ judgments as a group 88 percent of the time, and the physicians’ individual assessments in 86 percent of cases.

“We essentially provide a well-defined mathematical proxy for the deep intuition a dermatologist relies on when determining whether a skin lesion is suspicious enough to warrant closer examination,” says Soenksen in a Wyss Institute statement. “This innovation allows photos of patients’ skin to be quickly analyzed to identify lesions that should be evaluated by a dermatologist, allowing effective screening for melanoma at the population level.”

Along with his research at the Wyss Institute, Soenksen is a venture builder at MIT, where he seeks out new business opportunities from campus research on health care and artificial intelligence. In addition, Susan Conover, a co-author of the paper, is CEO of LuminDx, a Cambridge, Massachusetts company developing a technology with artificial intelligence to evaluate patients’ skin conditions from smartphone photos.

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Turn Your Business Into an International Organism

– Contributed content –

Shipping containers

(A. Kotok)

17 Feb. 2021. If you are looking for the next big step or change for your business, one of the most exciting prospects you might want to consider is to try going international. There are certainly many benefits to taking your business international, not least the fact that you can expect far greater profits and returns, and a more expanded and exciting business in the long run. Of course, there are also risks, and you will need to make sure that you are taking great care as you do this. Let’s take a look at how you can turn your business into an international organism right now.

Developing your base

Before you do anything else, you really need to make sure that you are developing the base of your business into the strongest possible thing it can be. As long as that is something that you can always fall back on and rely upon strongly, you will find that it is much less risky to take your business international, and you will get a lot more out of it in the long run. If you are at all uncertain about your base being strong enough, be sure to first put all of your efforts into making it so before you do anything else.

Trading with success

The whole prospect of trading internationally can be very worrying at first, so if you are not prepared for it you might find that it ends up causing you a lot of problems in the long run. However, as long as you are properly prepared and you know what you are doing, you should find that it is actually simpler than you might have thought. The most important thing is to make sure that you have the right partners on your side, including a customs broker offering fast customs clearance, and to get all of the red tape sorted out early. Do that, and you can trade internationally without too much trouble.

Researching new markets

You should be careful not to simply go into foreign markets without first understanding them at least in part. You need to carry out some research into the places that you are hoping to set up in, otherwise you might find yourself running into some unnecessary faux pas along the way or causing other kinds of problems that are best avoided. The more thorough your research is, the less likely it is that you will have any problems of this kind, so it really is something you are going to want to think about.

Prepare your people

Finally, you need to make sure that you are doing all you can to get your employees fully and properly prepared. If they are not prepared, then you might run into some significant trouble when it comes to actually putting all of this together and making it work. Spend whatever time and resources you need to get your people prepared, and you will find that the whole process is so much easier for you and everyone else involved.

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Novartis, Gates Partner on Sickle Cell Gene Therapy

Gene therapy graphic

(Genome Research Limited, Flickr: https://www.flickr.com/photos/yourgenome/31332923263)

17 Feb. 2021. Global drug maker Novartis is collaborating with the Bill and Melinda Gates Foundation to find affordable gene therapies for sickle cell disease. While the project is funded by the Gates Foundation, dollar amounts and timetables for the partnership were not disclosed, but the foundation’s records show a similar three-year $7.3 million project with Novartis already underway.

Sickle cell disease is a genetic blood disorder affecting hemoglobin, a protein in blood that delivers oxygen to cells in the body. People with sickle cell disease have hemoglobin molecules that cause blood cells to form into an atypical crescent or sickle shape. That abnormal shape causes the blood cells to break down, lose flexibility, and accumulate in tiny capillaries, leading to anemia and periodic painful episodes.

Sickle cell disease is prevalent worldwide, with people in sub-Saharan Africa or of African descent most affected. A study in The Lancet estimates more than 300,000 people are born with sickle cell disease each year. In addition, the disease affects some 70,000 to 80,000 people in the U.S., including about 1 in 500 people of African descent.

While several promising gene therapies are in development to treat sickle cell disease, including clinical trials, they require genetic editing or replacing a patient’s own blood-forming stem cells in the lab, then infusing back into the patient cells with healthy genes to produce healthy hemoglobin. In November 2020, Science & Enterprise reported on one such trial, conducted by Vertex Pharmaceuticals and Crispr Therapeutics testing its therapy to correct genetic defects responsible for sickle cell disease and beta thalassemia, another inherited blood disorder.

Research team devoted to project

These gene therapies require sophisticated lab facilities and highly trained staff to carry out, and very few of these facilities are found in low-resource regions where sickle cell disease is most prevalent. The Novartis/Gates Foundation project aims to discover a gene therapy process that physicians can administer once, with the transfer of healthy genes taking place in vivo, or inside the patient’s body.

“Gene therapies might help end the threat of diseases like sickle cell,” says Trevor Mundel, president of global health at the Gates Foundation in a Novartis statement, “but only if we can make them far more affordable and practical for low-resource settings.” Mundel adds that the collaboration “holds the promise of applying lessons learned to help develop potentially curative options for other debilitating diseases affecting low-income populations, such as HIV.”

The agreement calls for the Novartis Institutes for Biomedical Research, the company’s drug discovery arm, to devote a research team for this project. The Gates Foundation is providing its expertise on health care delivery in low-resource regions, with provisions in the agreement for supporting global access to the results of the initiative.

A search of the Gates Foundation’s recent awards shows a grant to Novartis Institutes for Biomedical Research in November 2020, “to establish a new team of researchers and leverage the expertise and resources of Novartis to explore, low cost capabilities for the in vivo functional cure of sickle cell and/or durable suppression of HIV in developing countries.” That project runs for 35 months and provides $7,280,864 to Novartis.

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Wearable Sensor Patch Records Multiple Health Measures

Sensor patch

Multi-functional sensor patch (Wang lab, Univ. of California in San Diego)

16 Feb. 2021. An engineering team developed a transparent thin film sensor patch that measures several vital signs for monitoring heart disease and diabetes. The patch is the work of labs in the Center for Wearable Sensors at University of California in San Diego and described in yesterday’s issue of the journal Nature Biomedical Engineering.

Patients with chronic disorders like heart disease and diabetes need to keep track of multiple health indicators, often requiring separate devices in a clinic, with trained staff to take and record measurements. With more use of mobile devices, efforts are underway to further miniaturize medical devices that connect to phones and tablets, and can be used at home. However, most miniaturized and networked devices of this kind are still made to capture and record single vital signs or measures. The UC San Diego researchers sought instead to design a wearable device that measured several functions, relieving the need for multiple devices.

The team led by Joseph Wang, director of the Center for Wearable Sensors, and electrical and bioengineering professor Sheng Xu. Wang’s nanobiolelectronics lab studies nanoscale components and systems, such as sensors, for measuring health indicators, including blood glucose levels needed by people with diabetes. Xu’s research group investigates ways to add more functions to stretchable and wearable electronics for monitoring a person’s health, while keeping down the amount of real estate these devices occupy.

The UC San Diego team designed a set of electronic and chemical sensors built into a thin transparent film worn on the skin. The film is made from a stretchable styrene-ethylene-butylene-styrene or SEBS polymer with electrodes screen-printed on the surface. The patch, worn on the neck over the carotid artery, measures blood-pressure with a set of ultrasound transducers that beam sound waves into the artery. The transducers are powered by piezoelectric electricity generated by stretching motions, with a corresponding sensor to read the ultrasound echoes for measuring dilation and contraction of the artery.

Glucose, lactate, caffeine, and alcohol

The patch also has electrodes for measuring glucose in interstitial fluid that flows between cells. An electrode emits a mild electrical impulse into the skin to produce interstitial fluid on the skin surface. Another set of electrodes measures lactate, caffeine, and alcohol in the wearer’s sweat. Lactate, or lactic acid, is produced by cells while metabolizing food into energy, particularly when exercising. The patch releases a drug on the skin called pilocarpine used to treat dry mouth, but can also induce sweat, for measuring these chemicals. Spacing the sensors and hydrogel insulation limits sensor signal interference.

“Each sensor provides a separate picture of a physical or chemical change,” says Xu in a university statement. “Integrating them all in one wearable patch allows us to stitch those different pictures together to get a more comprehensive overview of what’s going on in our bodies.”

The team tested measurement of each function separately in the lab, validated with conventional techniques, then together to reveal any interference of signals from the different sensors. The researchers also recruited volunteers to test the prototype sensor patch in various real-life scenarios: after eating, drinking, exercise, or fasting. The results show the patch can capture various health indicators, including production of glucose following food digestion.

The prototype sensor patch described in the paper is hard-wired to a receiving system. The researchers are working on a wireless patch, as well as adding more functions, while further miniaturizing the patch itself.

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AI Cell Analysis Start-Up Raises $6M Seed Funds

T lymphocyte

Colorized scanning electron micrograph of a T lymphocyte. (NIAID)

16 Feb. 2021. A new enterprise using artificial intelligence to analyze individual cells for immune-related diagnostics and therapies is raising $6 million in seed funds. Ozette Technologies in Seattle is spun-off from labs at Fred Hutchinson Cancer Research Center and Allen Institute for Artificial Intelligence, both in Seattle.

Ozette Technologies aims to provide more precise disease diagnostics with high-powered analytics of individual cells. The company applies artificial intelligence techniques to cytometry, quantitative analysis of cells and cell systems, in this case cells in or interacting with the immune system. Ozette’s technology is based on research in the lab of scientific founder Raphael Gottardo at Fred Hutch that designs statistical and software tools for single-cell analysis, particularly for understanding vaccines and infectious diseases.

Greg Finak, a co-founder of Ozette, is the company’s chief technology officer, and formerly a staff scientist at Fred Hutch. Finak led development of statistical models in Gottardo’s lab for understanding gene expression through RNA sequencing in cells. Gottardo and Finak also produced a statistical package called CytoML for analysis of flow cytometry data with the widely-used commercial statistical software R. CytoML is part of an open-source collection of single-cell analytical packages using R called Cytoverse created in the Gottardo lab.

More detailed and comprehensive analysis

Ozette says its use of artificial intelligence makes possible more detailed profiling of immune-related cells than is possible with most other techniques. The company says its technology can identify and analyze all of cells in a sample, providing a more detailed picture of cellular activity for diagnostics, to identify existing treatments, and eventually for discovery of new therapies. Current methods, says the company, often require manual analytics and do not approach the granularity and comprehensiveness of Ozette’s process.

Another Ozette Technologies founder is Ali Ansary, a Seattle physician and entrepreneur, and formerly entrepreneur in residence at the Allen Institute for AI. The Allen Institute, started by Microsoft founder Paul Allen, incubates start-ups where artificial intelligence is a core technology. The institute partners with venture capital companies to spin-off new AI start-ups, in this case Madrona Venture Group in Seattle.

According to Crunchbase News, Ozette is raising $6 million in seed funds led by Madrona Venture Group, with the Allen Institute and Vulcan Capital taking part in the financing. The company says it also raised another $6 million in grants and other funds since it started up last year. Ansary, now Ozette’s CEO tells Crunchbase News, “We have pioneered this work over the last decade, and where there are huge clinical applications, we are learning from the data that is being generated.”

Madrona Venture Group says in a company blog post that Ozette is the fifth spin-off it supports from the Allen Institute adding, “Both applied AI – AI applied to data and processes – and this intersection of innovation are key themes for our investing now and in the coming years.”

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Grant Funds Brain Disease Tissue Banks

Neurons

(commonfund.nih.gov)

15 Feb. 2021. An advocacy organization for neurodegenerative diseases and medical research foundation are sponsoring formation of a network to collect brain tissue samples. CurePSP in New York and Rainwater Charitable Foundation in Fort Worth, Texas are donating $1.25 million to fund a series of repositories for brain tissue samples for research on degenerative disease from damaging deposits of tau proteins in the brain.

Tau is a naturally occurring protein in the brain, but when tau proteins are misfolded they can accumulate in tangles around neurons, or nerve cells. Those accumulated tau tangles can damage nerve cells, causing taupathies, diseases marked by impaired movement as well as behavioral and cognitive decline. Among the disorders resulting from taupathies are frontotemporal dementia, progressive supranuclear palsy, post-traumatic stress disorder, and chronic traumatic encephalopathy or CTE, associated with brain injuries from military service or contact sports.

Frontotemporal dementia or FTD is a group of diseases caused by loss of nerve cells in the frontal lobes of the brain. In some cases, nerve cell loss is due to damage from tau deposits, leading to deterioration in behavior, personality, and ability to produce or comprehend language. Progressive supranuclear palsy also results from nerve cell damage due to tau deposits, causing involuntary eye movements and blurring of vision, as well as difficulty walking from loss of balance. Both disorders are progressive, meaning they get worse with time.

CurePSP is a group advocating for awareness and research on progressive supranuclear palsy, or PSP, and other neurodegenerative disorders that affect people in middle-age or younger. Rainwater Charitable Foundation supports medical research, including studies of tau-related disorders. The foundation manages the Tau Foundation and awards prizes for research on neurodegenerative diseases, including progressive supranuclear palsy.

Networking four brain tissue repositories

Rainwater Charitable Foundation and CurePSP are forming a network of brain tissue repositories in the U.S., at Mount Sinai medical school in New York, Boston University, Mayo Clinic in Jacksonville, Florida, and University of California in San Francisco. These institutions are already collecting samples, but the Rainwater and CurePSP funding aims to enhance their bioinformatics capabilities. The grant also expects to support a more centralized and streamlined process for accessing brain tissue samples. Rainwater is providing $1 million for the network, while CurePSP is adding $250,000.

CurePSP already encourages donations of brain tissue to aid research. “CurePSP has long promoted brain donation through a collaboration with the Mayo Clinic,” says Kristophe Diaz, CurePSP’s vice president for scientific affairs, in a Rainwater statement released on Cision, “and reimburses families for the cost of the procedure. CurePSP now has the opportunity to expand this program.”

“Despite substantial efforts within the neuropathology community,” notes William Seeley, professor of neurology and pathology at UC San Francisco and lead scientist in the network, “there remains an unmet need for centralized, streamlined, and efficient access to high quality and well-characterized tissue samples from patients with frontotemporal dementia spectrum disease.”

Amy Rommel, scientific program director for the Rainwater Charitable Foundation adds, “Once the vision for these four biobanks is fully realized, it will dramatically increase meaningful use of brain tissue in FTD research.”

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Infographic – States Reporting SARS-CoV-2 Variants

Covid-19 variations in states

Click on image for full-size view (Statista)

13 Feb. 2021. There’s both good and bad news about Covid-19 in the U.S. While case counts, hospitalizations, and deaths are declining and the vaccination rate is increasing, more infectious mutations in the SARS-CoV-2 virus responsible for Covid-19 infections are also appearing. The business research company Statista published a chart this week showing in which states cases of those viral variations are reported, based on data compiled as of 9 Feb. by Centers for Disease Control and Prevention.

The most widespread variant so far in the U.S. is B.1.1.7, first discovered in the U.K. B.1.1.7 is more easily transmitted than the original SARS-CoV-2, by 35 to 45 percent, and according to reports from the U.K., also more lethal. So far, according to the CSC, B.1.1.7 cases are reported in 34 of the 50 states and D.C., with the most cases reported in Florida and California.

Another variant, B.1.35 that first became known in South Africa, is reported in three states: Virginia, Maryland, and South Carolina. Cases of a third variant, P.1 first reported in Brazil, are found in Minnesota and Oklahoma. So far, 14 states do not report any of these mutations.

Early data, mainly from lab tests and some clinical trials, show the leading vaccines largely protect against B.1.1.7, but are less effective against B.1.35. Little, if any data, are yet reported for the vaccines protecting against P.1. Because the variants are more easily transmitted, vaccinations are becoming more urgent. And health authorities may need to prepare annual vaccine updates or boosters to protect against SARS-CoV-2, much like seasonal flu vaccines.

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20 Precision Meds Approved by FDA in 2020

Syringe, pills, capsules

(Arek Socha, Pixabay)

12 Feb. 2021. A report by a group advocating for precision medicine notes that the Food and Drug Administration approved 20 new personalized drugs and biologics in 2020. The report by the Personalized Medicine Coalition in Washington, D.C. says precision medicines account for a quarter or more of all new drugs approved by FDA each year in the past six years.

Precision or personalized medicines are prescribed by physicians to meet a patient’s molecular make-up, based on results of diagnostics, and the individual’s medical history. The 20 drugs and biologics approved by FDA in 2020 include 19 new drugs that target or require specific biomarkers, or biologic indicators, for various types of cancer, rare inherited diseases, neurological disorders, pain, and HIV. The agency also approved an immunotherapy for mantle cell lymphoma directed by genetically engineered T-cells, altered to express chimeric antigen receptors, known as CAR T-cells.

The 20 new approvals in 2020 account for 39 percent of all new drugs and biologics okayed by FDA in 2020. Since 2015, at least a quarter of FDA’s new drug approvals each year are precision medicines.

The report says FDA cleared eight new diagnostics in 2020 designed to support precision medicines. The new diagnostics include tests for solid tumors that identify specific biomarkers, independent of tissue or organs where the tumors reside. FDA also cleared a liquid biopsy, a blood test to detect cancer, that identifies cell-free DNA as indicators of cancer, instead of invasive tissue biopsies requiring surgery.

New regulatory guidance documents

In addition, FDA issued seven guidance documents in 2020 related to precision medicine. The guidance provided to developers include the agency’s thinking on manufacturing and control of gene therapies, long-term follow-up after administration of gene therapies, interpreting gene therapies under orphan drug regulations, and testing viral vector-based gene therapies during manufacturing and patient follow-up.

As reported by Science & Enterprise in January 2020, FDA also published a set of guidance documents for three types of disorders: hemophilia, rare diseases, and retinal disorders. Those documents were issued in draft by FDA in July 2018. Looking ahead, in January 2021, FDA issued draft administrative and procedural guidelines for genetic disorders treated with precise gene therapies.

Edward Abrahams, president of Personalized Medicine Coalition, says in an organization statement that the report, “documents FDA’s unwavering commitment to expanding the frontiers of personalized medicine while also demonstrating industry’s commitment to developing innovative and groundbreaking products that serve patients and make health systems more efficient.”

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AI Designs Better Gene Therapy Delivery Viruses

Adeno-associated virus

Adeno-associated virus (Jazzlw, wikimedia Commons)

12 Feb. 2021. An academic-industry team used machine learning to design more effective variations of common benign viruses that deliver gene therapies. Researchers from Dyno Therapeutics, Google Research, and the Wyss Institute at Harvard University describe their techniques in yesterday’s issue of the journal Nature Biotechnology (paid subscription required).

Gene therapies are a promising new type of treatment for a growing number of inherited diseases, where healthy genes replace inherited mutations responsible for disease. Many current gene therapies use adeno-associated virusesbenign and naturally occurring microbes that infect cells, but do not integrate with the cell’s genome or cause disease, other than at most mild reactions.

In their natural state, however, adeno-associated viruses, or AAVs, an imperfect and inefficient delivery vehicles, interrupted in some cases by immune reactions by patients receiving gene therapies. Also, the capsids that make up the outer protein shell of AAVs offer only a few ways of targeting cells and tissue in the body. The researchers in this project sought ways of addressing these limitations in AAVs for gene therapies using machine learning in neural networks, a form of artificial intelligence.

The team led by Harvard and Wyss Institute genetics professor George Church, Google’s Lucy Colwell, and Dyno Therapeutics CEO Eric Kelsic used machine learning to investigate more than 201,000 possible variations in amino acid sequences in natural-state capsid proteins. From these variations, the researchers designed nearly 111,000 engineered candidate capsid shells, with more than 57,000 of these designed capsids surpassing the diversity of capsids in their natural state. Each of the more diverse capsids expressed 12 to 29 mutations in their amino acid sequences.

Algorithms design optimized capsids

Church, who heads Harvard’s synthetic biology platform where the research began says in a Wyss Institute statement, “It shows that neural networks combined with the high-throughput synthetic testing developed in our lab is changing the way we design gene delivery vehicles and protein drugs.”

Dyno Therapeutics is a spin-off company from Church’s lab founded by Kelsic, Church, and co-author Sam Sinai in 2018. The company extends the academic lab’s work in a technology called CapsidMap that uses machine learning algorithms to design optimized AAV capsids. The algorithms find millions of optimal combinations of targeting ability, payload size, immune evasion, and manufacturing capability, then give each variation a unique DNA identifier. The optimized capsids are then assembled to meet specific therapeutic needs, with each design adding to and refining the algorithms’ experience.

“Our approach achieves the highest functional diversity of any capsid library thus far,” notes Kelsic. “It unlocks vast areas of functional but previously unreachable sequence space, with many potential applications for generating improved viral vectors, like AAVs with much reduced immunogenicity and much improved target tissue selectivity, and also for highly efficient gene therapies.”

Science & Enterprise reported several times on Dyno Therapeutics, most recently on licensing agreements between the company and drug makers Novartis and Sarepta Therapeutics in May and Roche Group in October 2020. The two deals could bring Dyno Therapeutics as much as $3.5 billion if all terms are met.

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Immune Response Shown in Alzheimer’s Vaccine

Brain activity graphic

(Gordon Johnson, Pixabay)

11 Feb. 2021. Results from a clinical trial show an experimental vaccine produces antibodies for breaking up harmful brain deposits in people with early Alzheimer’s disease. AC Immune SA in Lausanne, Switzerland, developer of the vaccine code-named ACI-35.030, reported today interim findings from the clinical trial, which are not yet peer-reviewed.

AC Immune develops diagnostics, vaccines, and treatments for Alzheimer’s disease and other neurodegenerative disorders. Alzheimer’s disease is a progressive neurodegenerative condition, the most common form of dementia affecting growing numbers of older people worldwide. People with Alzheimer’s disease often have deposits of abnormal substances in spaces between brain cells, known as amyloid-beta proteins, as well as misfolded tangles of proteins inside brain cells known as tau. The company cites data from the World Alzheimer Reports showing an estimated 50 million people worldwide with dementia, a number expected to grow to 152 million by 2050.

AC Immune uses two separate technologies to address misfolded proteins, particularly tau that builds up inside of neurons in the brain, and spreads between cells, and amyloid-beta proteins that accumulate as plaques outside of neurons. One of those technologies, called SupraAntigen, is the basis for treatments that generate antibodies in the immune system for attacking and breaking up tau deposits, particularly before they cause irreversible damage to neurons.

ACI-35.030 is designed to generate antibodies seeking out deposits with phosphorylated tau, or pTau, often found in people with Alzheimer’s disease, or AD.  AC Immune is developing ACI-35.030 in partnership with Janssen Pharmaceuticals, a subsidiary of Johnson & Johnson, which has an exclusive license on the vaccine.

Measuring antibody concentrations with anti-pTau properties

The clinical trial is an early- and mid-stage study, enrolling 32 individuals, age 50 to 75, in the early stages of Alzheimer’s disease or with mild cognitive impairment, at six sites in Finland, the Netherlands, and the U.K. Participants are randomly assigned to receive low, medium, or high doses of ACI-35.030 or a placebo at predetermined intervals over 48 weeks, then followed for another 26 weeks.

The study team is looking primarily at the safety of ACI-35.030, watching for adverse effects including suicidal impulses, and changes in vital signs. But the researchers are also measuring immunoglobulin-G and -M antibody concentrations with anti-pTau properties in the blood of participants.

Early results from the trial, says AC Immune, show ACI-35.030 is safe and well-tolerated with no relevant safety issues observed so far. Blood samples show anti-pTau immunoglobulin-G and -M antibodies in all recipients of ACI-35.030, including recipients of one low or medium dose of ACI-35.030 with anti-pTau immunoglobulin-G antibodies. Moreover, high concentrations of anti-pTau immunoglobulin-G antibodies are found in ACI-35.030 recipients after a single dose.

The published findings do not indicate numbers of participants receiving ACI-35.030, nor any comparisons with placebo recipients. And the trial does not measure effects of ACI-35.030 on tau deposits on neurons in participants.

“As pathological pTau is present as a precursor many years before tau accumulation in the brain is detectable via brain imaging,” notes Andrea Pfeifer, CEO of AC Immune, in a company statement, “such results highlight the significant promise of ACI-35.030 as an early intervention for AD, especially when combined with cutting-edge pTau diagnostics that would enable identification of people at risk of developing tau-driven disease.”

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Disclosure: The author owns shares in Johnson & Johnson.

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