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Discovery in Monkeys Could Lead to Treatment for Blindness-Causing Syndrome – Technology Networks

A genetic mutation that leads to a rare, but devastating blindness-causing syndrome has been discovered in monkeys for the first time. The finding offers a promising way to develop gene and cell therapies that could treat the condition in people.

Three rhesus macaques with a mutated gene that's associated with Bardet-Biedl Syndrome have been discovered, according to a study published in the journal Experimental Eye Research. It is the first known naturally occurring nonhuman primate model of the syndrome, which is also called BBS.

BBS leads to vision loss, kidney dysfunction, extra fingers or toes, and other symptoms. It occurs in 1 of 140,000 to 160,000 North American births.

"There is no cure for Bardet-Biedel Syndrome today, but having a naturally occurring animal model for the condition could help us find one in the future," said the paper's corresponding author, Martha Neuringer, Ph.D., a professor of neuroscience at the Oregon National Primate Research Center at Oregon Health & Science University, and a research associate professor of ophthalmology in the OHSU School of Medicine and OHSU Casey Eye Institute.

Broader applications

Rhesus macaques with this disease could help more than just BBS patients. BBS is part of a larger family of diseases called retinitis pigmentosa, all of which affect the retina, or the back part of the eye. A naturally occurring animal model for BBS could help researchers find treatments for a variety of retinitis pigmentosa diseases.

The discovery is timely, as gene therapy is already becoming a reality for some with retinal diseases. In the 1990s, researchers discovered dogs that had a gene mutation linked to a congenital blindness-causing condition called Leber's congenital amaurosis. That animal model played a key role in helping researchers develop what became the first FDA-approved gene therapy for an inherited disease in December 2018. Neuringer's group also hopes to develop a similar therapy for BBS.

Gene mutation

After Neuringer and colleagues discovered two related monkeys without cells that are key to vision, OHSU nonhuman primate genetics experts Betsy Ferguson, Ph.D., and Samuel Peterson, Ph.D., examined the animals' genomes. They quickly found both monkeys had a mutation of the BBS7 gene, one of at least 14 genes associated with BBS.

Because Ferguson leads an effort to genetically sequence 2,000 rhesus macaques at the nonhuman primate research center, they were also able to search the genomes of numerous other monkeys there. As a result, the team found a third monkey with the same mutation. The third rhesus macaque already had serious vision loss when it was identified in 2018 at age three and a half, although it adapted so well among its social group that the vision loss wasn't obvious. Neuringer's team is observing the third monkey over time to better understand how the disease progresses in rhesus macaques.

Neuringer and her colleagues are now using a National Eye Institute grant to breed more animals with the naturally occurring BBS7 mutation. Having more animals with the mutation can help researchers better understand the disease and test potential treatments. The knowledge they gain could enable them to develop gene and cell therapies that could cure BBS and related retinal degenerative diseases.

This article has been republished from the following materials. Note: material may have been edited for length and content. For further information, please contact the cited source.

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Intellia Therapeutics Presents In Vivo and Ex Vivo Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) -…

CAMBRIDGE, Mass., Oct. 24, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ: NTLA), a leading genome editing company focused on the development of curative therapeutics using CRISPR/Cas9 technology is presenting one oral presentation and four poster presentations at the 27th Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) meeting taking place October 22-25, 2019, in Barcelona, Spain.

We are excited to share progress across Intellias in vivo and ex vivo programs at this important scientific venue, said Laura Sepp-Lorenzino, Ph.D., chief scientific officer, Intellia Therapeutics. Our data shows the complexity of the edits we are able to make with CRISPR/Cas9, while achieving important therapeutically relevant results. We are building on the success of our modular platform now having demonstrated consecutive targeted knockout and insertion genome edits in preclinical studies. Additionally, we presented data from our engineered cell therapy program, which continues to demonstrate the use of CRISPR/Cas9 for combined knockout and targeted integration in human T cells.

Intellia Demonstrates Consecutive In Vivo Genome Editing in Alpha-1 Antitrypsin Deficiency Mouse Model

Intellias oral presentation highlights its alpha-1 antitrypsin deficiency (AATD) study showing that consecutive dosing of two distinct lipid nanoparticle (LNP) formulations, in adultmice, achieves two targeted genome editing events, resulting in knocking out the faulty gene and restoring therapeutic levels of normal alpha-1 antitrypsin protein (hAAT). Intellias approach for AATD uses a modular hybrid delivery system combining a non-viral LNP which encapsulates CRISPR/Cas9 with an adeno-associated virus (AAV) carrying donor DNA template. Compared to traditional viral-based delivery of gene editing components, Intellias LNP delivery system can overcome the inherent limitations of immunogenicity to facilitate multiple in vivo gene editing events.

In a mouse model harboring the human PiZ allele, the most severe genetic defect in AATD patients, Intellia first reduced expression of the defective protein using gene knockout. Three weeks following the PiZ allele knockout, Intellia inserted the normal human alpha-1 antitrypsin gene, resulting in stable (throughout 12 weeks of observation), therapeutically relevant circulating protein levels. In the study, a sustained reduction of the circulating PiZ protein levels of >98% was observed for over 15 weeks. This is the first in vivo demonstration of a non-viral delivery platform, enabling a consecutive dosing approach for achieving multiple genome edits in the same tissue of the same animal. Intellias oral presentation, titled In Vivo Gene Knockout Followed by Targeted Gene Insertion Results in Simultaneous Reduced Mutant Protein Levels and Durable Transgene Expression, will be given by Anthony Forget, Ph.D., on October 25, 2019. This presentationwill be available on Intellias website at http://www.intelliatx.com.

Intellias Poster Presentations

WT1-Specific TCR Engineered Cell Therapy Studies

Intellia presented new in vitro data showing that CRISPR/Cas9-mediated genome editing for in locus insertion, combined with endogenous T Cell Receptor (TCR) knockout, leads to significant reduction in mispairing of endogenous and transferred TCR chains. This approach is expected to generate transgenic-TCR (tg-TCR) T cell therapies for hematological cancers and solid tumors. Results demonstrate a highly efficient reduction of >98% in endogenous TCR and chains while reaching >70% insertion rates of tg-TCRs without further purification. The poster titled Engineering of Highly Functional and Specific Transgenic T Cell Receptor (TCR) T Cells Using CRISPR-Mediated In Locus Insertion Combined with Endogenous TCR Knockout, was presented on October 24, 2019, by Birgit Schultes, Ph.D.

Researchers also presented in vitro data showing that a library of WT1-specific TCRs were generated, several of which Intellia is currently evaluating as part of its lead engineered cell therapy program targeting Acute Myeloid Leukemia (AML). This presentation, Generation of a Library of WT1-Specific T Cell Receptors (TCR) for TCR Gene Edited T Cell Therapy of Acute Leukemia, was presented on October 23, 2019 by Intellias collaborator, Erica Carnevale, Ph.D., IRCCS Ospedale San Raffaele.

Primary Hyperoxaluria Study

Intellia showed the continued progression of its modular platform capability using CRISPR/Cas9 to knockout either hydroxyacid oxidase 1 (Hao1) or lactate dehydrogenase A (Ldha), leading to a dose-dependent and persistent reduction of urinary oxalate levels in a Primary Hyperoxaluria Type 1 (PH1) mouse model. Data shows Ldha gene disruption also decreased LDH enzyme activity in the liver and did not impair the disposition of lactate in either wild type or renally-impaired mice. These results highlight the potential of editing genes in the glyoxylate detoxification pathway using a non-viral delivery approach as a one-time treatment option for PH1. These data were presented as a poster, titled CRISPR/Cas9-Mediated Gene Knockout to Address Primary Hyperoxaluria, by Sean Burns, M.D., on October 24, 2019.

Off-Target Screening Platform

Intellia demonstrated its approach to assess off-target activity to identify highly specific CRISPR/Cas9 guides. Results from targeted off-target sequencing in edited cells showed that biochemical off-target discovery approaches were the most sensitive and accurate. These data were presented as a poster on October 23, 2019, titled In Silico, Biochemical and Cell-Based Integrative Genomics Identifies Precise CRISPR/Cas9 Targets for Human Therapeutics, by Dan OConnell, Ph.D.

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company focused on developing proprietary, curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements ofIntellia Therapeutics, Inc.(Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an IND application for NTLA-2001 in mid-2020; its plans to generate preclinical and other data necessary to nominate a first engineered cell therapy development candidate for its AML program by the end of 2019; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program and otherin vivoandex vivoprograms such as its AATD program; develop our proprietary LNP-AAV hybrid delivery system to advance our complex genome editing capabilities, such as gene insertion; its presentation of additional data at upcoming scientific conferences regarding CRISPR-mediated, targeted transgene insertion in the liver of NHPs, using F9 as a model gene, via the Companys proprietary LNP-AAV delivery technology, and other preclinical data by the end of 2019; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR and AML programs, in any future studies, including human clinical trials; its ability to develop otherin vivoorex vivocell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; the impact of its collaborations on its development programs, including but not limited to its collaboration withRegeneron Pharmaceuticals, Inc. or Ospedale San Raffaele; statements regarding the timing of regulatory filings regarding its development programs; and the ability to fund operations into the second half of 2021.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain our intellectual property position, including through our arbitration proceedings against Caribou; risks related to Intellias relationship with third parties, including our licensors; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies will not be predictive of future results in connection with future studies; and the risk that Intellias collaborations withNovartisor Regeneron or its otherex vivocollaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with theSecurities and Exchange Commission. All information in this press release is as of the date of the release, andIntellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Media:Jennifer Mound SmoterSenior Vice PresidentExternal Affairs & Communications+1 857-706-1071jenn.smoter@intelliatx.com

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

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Intellia Therapeutics Presents In Vivo and Ex Vivo Data at the 2019 Annual Congress of the European Society of Gene and Cell Therapy (ESGCT) -...

Researchers Used Green Tea as a ‘Remote Control’ to Activate Cell Therapies for Diabetes – D-brief – Discover Magazine

(Credit: Kirasolly/Shutterstock)

Since ancient times,the health benefits of green tea have been the stuff of legend. Now,researchers are turning to the antioxidant-rich leaves for a decidedly modern purpose triggering cell-based therapies.

In a study published Wednesday in Science Translational Medicine, a team of researchers with East China Normal University and First Affiliated Hospital of Shenzhen University found that green tea can work to activate lab-made cells for disease treatment. In short, they used the beverage as a drinkable remote control.

And the authors think that the new approach, which they used as part of a successful treatment for diabetes in mice and monkeys, could one day be used to deliver remote controlled, easy-to-follow therapies for diabetes in humans.

In recent years, scientists have worked to refine how cell therapies which have shown promise in treating diseases like cancer are controlled. These therapies often rely on a chemical compound to trigger cells to secrete a specific therapeutic substance. But many of the currently-used triggers, like antibiotics, can have negative side effects or are unsafe for prolonged use.

In search of a bettertrigger, lead author Jianli Yin and her colleagues looked for a compound thatwas natural, nontoxic and perhaps even beneficial to health. Enter green tea.

In addition to itslaundry list of purported health benefits, green tea contains a molecule calledprotocatechuic acid, or PCA. The research team engineered gene switches thatcould be activated by PCA before inserting them in different human and animalcells.

To test out theirnew system, the team implanted the PCA-responsive cells into mice with diabetes,a disease that impairs the bodys ability to produce insulin. After the micewere given concentrated doses of green tea, the researchers observed that therodents insulin levels rose while their blood sugar levels fell.

But the studyauthors also noted that what works in mice doesnt always translate to humans.So, to see if they achieved the same results in our nonhuman primate cousins, thescientists tested their system on macaque monkeys, too.

The findings were indeedsimilar after drinking green tea or receiving an injection of PCA, the monkeysinsulin production returned to normal.

And while there havent yet been clinical studies on similar cell-based therapies in humans, the researchers remain optimistic that their findings could present new possibilities for disease treatment. That is, just so long as you dont mind drinking a little green tea.

Green tea has been an extremely popular beverage for more than 2,000 years, the study said. We suspect that [this system] will be considered safe and help to improve and encourage patient compliance.

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Researchers Used Green Tea as a 'Remote Control' to Activate Cell Therapies for Diabetes - D-brief - Discover Magazine

Cleveland Clinic Predicts Top 10 Medical Innovations for 2020 – HealthLeaders Media

When Cleveland Clinic prognosticators peered into their crystal ball to forecast the top 10 medical innovations for 2020, there must have been a healthy heart beating in the center of the orb. Four of the 10 predictions involve advancements in the field of cardiology.

Many of the innovations involve emerging drug therapies, with the number one innovation related to a new drug to treat osteoporosis, which possesses dual properties to increase bone formation and decrease bone resorption.

As the 2019 Cleveland Clinic Medical Innovation Summit came to a close on Wednesday, organizers released their annual list of the medical innovations that hold the greatest promise to impact healthcare in the coming year. The list of up-and-coming technologies was selected by a panel of Cleveland Clinic physicians and scientists, led by Michael Roizen, MD, emeritus chief wellness officer at Cleveland Clinic.

Healthcare is ever-changing, and we anticipate that these innovations will significantly transform the medical field and improve care for patients at Cleveland Clinic and throughout the world, says Dr. Roizen in a news release issued by the health system.

The top 10 medical innovations for 2020, in order of anticipated importance, are:

1. Romosozumab, a Dual-Acting Osteoporosis Drug

The team hailed a new dual-acting drug, romosozumab, recently approved by the FDA, as the top innovation for 2020. Romosozumab is a monoclonal antibody that binds to and inhibits sclerostin, increases bone formation, and decreases bone resorption, according to an article published on October 20, 2016 in the New England Journal of Medicine. The drug gives "patients with osteoporosis more control in preventing additional fractures," according to the panel. About 10 million Americans have osteoporosis costing patients, their families and the healthcare system $52 billion annually, according to the National Osteoporosis Foundation.

2. Expanded Use of Minimally Invasive Mitral Valve Surgery

Earlier this year the FDA expanded approval of a minimally invasive valve repair device to treat a population of patients who have failed to get symptom relief from other therapies. The team of physicians and scientists say this "provides an important new treatment option," for the condition. Normally, the mitral valve allows blood flow from the hearts left atrium to the left ventricle; however, one in 10 individuals over the age of 75 have a defective valve, which causes the blood to flow backwards or "regurgitate." The device prevents this from occurring.

3. Inaugural Medication for Transthyretin Amyloid Cardiomyopathy

The cardiovascular disorder, ATTR-CM, "is a progressive, underdiagnosed, potentially fatal disease in which amyloid protein fibrils deposit in, and stiffen, the walls of the hearts left ventricle," according to Cleveland Clinic. "A new agent to prevent misfolding of the deposited protein is showing a significantly reduced risk of death," the news release says. "Following fast-track and breakthrough designations in 2017 and 2018, 2019 marked the FDA approval of tafamidis, the first-ever medication for treatment of this increasingly recognized condition."

4. Therapy for Mitigation of Peanut Allergies

A new oral immunotherapy medication to gradually build tolerance to peanut exposure holds the opportunity to lend protection against attack. While not yet approved by the FDA, the drug made progress toward that goal when an advisory committee voted in support of the drug's safety and effectiveness. A full vote is expected in January. The American College of Allergy, Asthma and Immunology (ACAAI) suggests that peanut allergy in children has increased 21% between 2010 and 2017, and that nearly 2.5% of U.S. children may have an allergy to peanuts.

5. Closed-Loop Spinal Cord Stimulation Because current treatment for chronic pain involving spinal cord stimulation has "unsatisfactory outcomes due to subtherapeutic or overstimulation," says the panel, a treatment involving closed-loop stimulation looks promising. The latter treatment "allows for better communication between the device and the spinal cord, providing more optimal stimulation and relief of pain," according to the release. A study, involving researchers from multiple institutions, including one from Cleveland Clinic, is underway to compare the two methods. As reported in Neuromodulation on March 3, 2019, "This study represents the first randomized, double-blind, pivotal study in the field of neuromodulation to measure SC activation in ECAP-controlled closed-loop versus open-loop stimulation and is expected to yield important information regarding differences in safety, efficacy, and neurophysiological properties."

6. Biologics in Orthopaedic Repair

The use of biologics, which include cells, blood components, growth factors and other natural substances, "have the power to replace or harness the body's own power and promote healing," the panel says. Use of biologics in orthopaedic care may enhance the possibility of expedited improved outcomes, speeding the months and years it can take to recover from orthopaedic surgery. An article published January 15 in the Journal of American Academy of Orthopaedic Surgery presents consensus recommendations for use of these substances.

7. Antibiotic Envelope for Cardiac Implantable Device Infection Prevention

According to Cleveland Clinic, an estimated 1.5 million patients around the world who receive an implantable cardiac electronic device every year. There is added protection against infection through the use of antibiotic-embedded envelopes which encase these cardiac devices. Cleveland Clinic researchers participated in a study about this innovation published May 16 by the New England Journal of Medicine.

8. Bempedoic Acid for Cholesterol Lowering in Statin Intolerant Patients

High cholesterol is a major concern for nearly 40 percent of adults in the U.S., according to the Cleveland Clinic release. "Left untreated, the condition could lead to serious health problems like heart attack and stroke." Some individuals experience unacceptable muscle pain with statins, which are typically prescribed to treat this problem. "Bempedoic acid provides an alternative approach to lowering of LDL-cholesterol while avoiding these side effects," the panel says. An article published March 29 in the Journal of the American Heart Association concluded that "bempedoic acid offers a safe and effective oral therapeutic option for lipid lowering in patients who cannot tolerate statins.""

9. PARP Inhibitors for Maintenance Therapy in Ovarian Cancer

Ovarian cancer has a new foe with PARPpoly-ADP ribose polymeraseinhibitors that "block repair of damaged DNA in tumor cells which increases cell death, especially in tumors with deficient repair mechanisms," according to the release. Cleveland Clinic reports PARP therapy is "one of the most recent important advances ovarian cancer treatment. PARP inhibitors have improved progression-free survival and are now being approved for first-line maintenance therapy in advanced stage disease. Several additional large-scale trials are underway with PARP inhibitors set to make great strides in improving outcomes in cancer therapy," the panel reports. As reported by the American Journal of Managed Care, a presentation in March at the National Comprehensive Cancer Network Annual Conference in Orlando, Florida, outlined several key updates for treatment in ovarian cancer based on new studies and approvals forpoly (ADP-ribose)polymerase (PARP) inhibitorsand bevacizumab.

10. Drugs for Heart Failure with Preserved Ejection Fraction

A potential new treatment option is emerging for treatment of heart failure with preserved ejection fraction (HFpEF). The condition is also known as diastolic heart failure, in which the ventricular heart muscles contract normally, but do not relax as they should, the release explains. "With preserved ejection fraction, the heart is unable to properly fill with blood, leaving less available to be pumped out to the body. Currently, recommendations for this treatment are directed at accompanying conditions and mere symptom relief. But SGLT2 inhibitors, a class of medications used in the treatment of type 2 diabetes, is now being explored in HFpEF alluding to a potential new treatment option." An article published online in February by JACC, the Journal of the American College of Cardiology presents the growing case for use of SGLT2i in heart failure.

The annual Medical Innovation Summit is organized by Cleveland Clinic Innovations, the development and commercialization arm of Cleveland Clinic. The Cleveland Clinic Newsroom offers additional information about the annual Top 10 Medical Innovations including descriptions, videos, and year-by-year comparisons.

Mandy Roth is the innovations editor at HealthLeaders.

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Cleveland Clinic Predicts Top 10 Medical Innovations for 2020 - HealthLeaders Media

Here’s why we’re losing the war on cancer, according to this doctor – New York Post

When oncologist and cancer researcher Harvey Preisler was diagnosed with lymphoma in 1998, he wanted one person to oversee his care.

I trust only your judgment, Dr. Preisler told his wife, Dr. Azra Raza, an oncologist.

Despite her hesitations, from that moment on Raza became the point person for her husbands care. The two, who lived in Chicago, pored over MRIs, blood reports and treatment options. Though they shared an unrivaled expertise with years of clinical experience, nothing could prepare them for the horrors that cancer inflicts.

Preisler woke up in buckets of sweat and lived in a constant state of agony thanks to arthritic pain in his joints. He was afflicted with hot and blistering lesions on his tongue, swollen lymph nodes, blood clots, nerve pain, shingles, facial paralysis and, eventually, sepsis and meningitis.

The overwhelming pain rattled Raza, even though she had spent nearly two decades treating and studying bone-marrow preleukemic conditions and acute myeloid leukemia (AML), which has just a 27.4 percent five-year survival rate, according to the National Cancer Institute.

Cancer is what I had been treating for two decades, yet until I shared a bed with a cancer patient, I had no idea how unbearably painful a disease could be, writes Raza in her new book, The First Cell: And the Human Cost of Pursuing Cancer to the Last (Basic Books).

No cutting-edge immune therapies or experimental drug trials would save her husband. Preisler lived with unimaginable pain on-and-off for four years, until he died in 2002 at age 61, leaving behind Raza and their daughter, Sheherzad, who was 8 years old.

Stories like Preislers antidotes to the cheery, ribbon-wearing stories of survival against all odds are woven throughout The First Cell.

Stay positive is the refrain, as if were a sin to voice the intense pain and suffering of cancer patients, writes Raza, now a professor of medicine and the director of the Myelodysplastic Syndrome Center at Columbia University. Why are we so afraid to tell the stories of the majority who die? Why keep promoting the positive anecdote?

These are tough words and this is a tough book but there are solutions embedded, too. The first step, Raza argues: Its time to discard the outmoded way of viewing, researching and talking about cancer.

There is a misconception that we are just around the bend from winning the war on cancer.

A common stat thrown around is that cancer deaths have declined by 20 percent since 1980. But Raza explains how that decrease is not due to improved treatments but mostly to early diagnosis and a decline in smoking.

No one is winning the war on cancer. It is mostly the same rhetoric ... for the past half a century.

Another stat offers that we are managing to cure 68 percent of cancers. Raza points out that most of that cure rate was achieved several decades ago with the surgery-chemoradiation therapies. Recent advances relate primarily to improvement in cancer mortality due to early detection, not meaningful advances in the treatment of metastatic cancers.

Focusing on only the success stories, she says, and portraying the battle as nearly won is engaging in deep denial about what many cancer sufferers are facing on a daily basis.The time has come for us to think about the majority who dont, but who suffer the ghastly toxicities of therapies and end up losing their life savings in the process, she writes.

Take one of Razas patients, Henry W.

He was a handsome, fit and healthy, tennis-loving father of three when he developed spontaneous bruising during a vacation in the Bahamas.

When Henry returned home, tests confirmed the worst: Leukemia had ravaged his body. His only shot at survival would be chemotherapy and a bone marrow transplant. But next steps wouldnt come quick enough: Henry developed a fever and the chemotherapy wore him down into a shell of himself. After a brief respite from the pain and suffering much of which was brought on by the treatment itself he developed sepsis.

Six months after his initial diagnosis, Henry died.

The treatment offered to Harry, as well as the other patients typically, 30 to 40 a week Raza has seen for the past 35 years, has shockingly not evolved much in 50 years ... With minor variations, a protocol of surgery, chemotherapy and radiation the slash-poison-burn approach to treating cancer remains unchanged. It is an embarrassment, she writes. No one is winning the war on cancer. It is mostly hype, the same rhetoric from the same self-important voices for the past half a century.

The sobering reality is this: Ninety-five percent of experimental drugs that hit the market fail; the remaining five might as well have failed, she writes, as 70 percent of them have zero effect on survival, while 30 to 70 percent actually cause harm to the patient.

Of the 72 cancer drugs that hit the market between 2002 and 2014, the average medication prolonged life by a mere 2.5 months.

You could argue that two months is everything to someone facing death. But these two months come at a tremendous cost. Not only might these drugs cause extreme physical suffering (as with Preisler and Henry), but also financial ruin. According to one study cited by Raza, of the 19 million people diagnosed with cancer over 14 years, nearly half lost their life savings.

Tarceva, a drug that, on average, extends the rates of pancreatic cancer survival by 12 days, costs $26,000. An 18-week course of cetuximab for lung cancer costs $80,000.Our goals, Raza writes, are out of whack.

We should not be aiming for weeks of improved survival. Our goals should be higher. The public needs to see how far we have drifted from the original aims, she writes.

First, she says, we need to instate a fundamental change to how research is conducted. Of the 3 million papers published to date on cancer, a good 70 percent are not reproducible. (This also might help explain why so many experimental drugs never make it to market.)

To fix this, Raza writes that we first need to stop studying rats and instead study humans and in doing so accept the complexity of cancer in order to properly treat it.

This failure of progress on the treatment side is systemic, she argues. The vast majority of researchers are studying diseases they never see, in animals who dont get them spontaneously or in test tubes where the cancer must be artificially created ... How can scientists, who demand great precision in everything they do, simply turn their eyes away from such fundamental fallacies?

Her strategy is to redirect research to focus on prevention.

Prevent the appearance of the first cancer cell by finding its earliest footprints, Raza writes. Prevention will be the only compassionate, universally applicable cure.

Were already seeing some positive effects from that approach. Thanks in large part to high-quality screening, theres been a 25 percent decrease in cancer mortality overall with breast-cancer deaths down 39 percent and colorectal cancers down 47 percent for men and 44 percent for women.

Raza sees this as only the tip of the iceberg. She wants to dig deeper and find the so-called first cell the initial seedling of cancer and stop it before it develops.

The concept may not be as sexy as finding the magic bullet that will cure cancer, but Raza believes that treatments and patient outcomes would be most vastly improved by a fundamental alteration of perspective.

This is no out-of-touch pipe dream. Its happening right now at Johns Hopkins, where liquid biopsies locate early biomarkers of malignancy in blood, urine or sweat. In these bodily fluids, researchers look for mutations, epigenetic changes, certain cancer-indicating proteins and DNA markers.

One of the researchers there, Dr. Bert Vogelstein, estimates that a focus on early detection could reduce cancer deaths by 75 percent.

And then there are the many technological advancements on the horizon. Machines that scan your body, looking for cancer, in the shower. A smart bra with biosensors that monitor temperature and texture and could potentially suss out early-stage cells. A device you breathe into that can recognize cancer cells at their very initial stages.

These are real-life technologies in various stages of development today, heralding the dawn of a new era in cancer research, Raza writes.

Tackling these issues requires all hands on deck, not a sectioned off lab in an ivory tower with mice models, she writes.

You need geneticists, biomedical engineers, radiologists, oncologists, molecular biologists, nanotechnologists, AI experts, computer scientists and bioinformation wizards all working together on this complex issue.

The public also needs to play a role: demanding that their tax dollars contribute to researchers focusing on early detection.

To change a situation, one has to first lift the blinders and dare to see the situation for what it is, Raza writes. The future is in preventing cancer by identifying the earliest biomarkers of the first cancer cell rather than chasing after the last. I have been saying this since 1984, and I will continue to say it until someone listens.

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Here's why we're losing the war on cancer, according to this doctor - New York Post

Four from Hopkins elected to National Academy of Medicine – The Hub at Johns Hopkins

ByHub staff report

Four Johns Hopkins University faculty members are among 90 new members selected to join the National Academy of Medicine, considered to be among the highest career honors for those in the fields of health and medicine. Colleen Barry, Sharon Gerecht, Elizabeth Jaffee, and Dorry Segev were selected for membership based on their outstanding professional achievement and their commitment to service.

The NAM collaborates with the National Academy of Sciences and the National Academy of Engineering to provide independent and objective analysis to the U.S. government and the international community on critical issues relating to the medical sciences, health care, and public health. New members are elected by current members through a selective process that stipulates that at least one-quarter of the organization is made up of individuals from fields outside the health professions. The newly elected members announced today bring NAM's total membership to more than 2,200 and the number of international members to approximately 180.

"These newly elected members represent the most exceptional scholars and leaders whose remarkable work has advanced science, medicine, and health in the U.S. and around the globe," said NAM President Victor J. Dzau in a message accompanying the announcement of new members. "Their expertise will be vital to addressing today's most pressing health and scientific challenges and informing the future of health and medicine for the benefit of us all. I am honored to welcome these esteemed individuals to the National Academy of Medicine."

More on the new members from Johns Hopkins:

Colleen L. Barry, professor and chair of the Department of Health Policy and Management at the Bloomberg School of Public Health, was selected for her scholarly work on developing and measuring the impact of policies that support mental health and combat addiction. She is co-director of the Bloomberg School's Center for Mental Health and Addiction Policy Research and works to show how governmental policies affect patients' access to health care and social services and the mortality rates for people with mental illness and addiction.

Sharon Gerecht is a professor in the Department of Chemical and Biomolecular Engineering at the Whiting School of Engineering and director of the Institute for NanoBioTechnology. She was recognized for her seminal studies on the interactions between stem cells and their microenvironments. She was also selected for membership for engineering artificial cell microenvironments that are capable of guiding vascular differentiation and the regeneration of tissues.

Elizabeth M. Jaffee, a professor of oncology and deputy director of the Sidney Kimmel Cancer Center, is an international leader in the development of immune based therapies for pancreatic and breast cancers. She was selected for membership to NAM on the strength of her work elucidating the complex interactions between T-cell subsets and cancer and for translating those findings into two generations of vaccine platforms.

Dorry L. Segev, professor of surgery and epidemiology and associate vice chair of the Department of Surgery at the School of Medicine, was selected for his pioneering work developing HIV-to-HIV transplants and kidney exchange. He was recognized for his contributions to the field that included initial research, a congressional bill, studies on implementation, and an examination of the national clinical impact of such transplants. His work has changed the landscape of understanding transplant risk prediction through novel big data approaches.

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Four from Hopkins elected to National Academy of Medicine - The Hub at Johns Hopkins

NASA plans to cut cost of space exploration by commercializing low-earth orbit – Digital Trends

The Artemis 1 Orion Crew Module. NASA

In a presentation at the 2019 International Astronautical Congress in Washington, D.C., NASA Administrator Jim Bridenstine spoke about the agencys plans for the Artemis mission to the moon and then on to Mars. And he laid out how NASA plans for pay for this new era of manned space exploration: By encouraging the commercialization of low-earth orbit in order to cut costs for the agency.

Were not going back to the moon, Bridenstein promised. Were going forward to the moon. The aim for the 2024 mission is for astronauts to stay on the moon for a longer period of time than ever before, and for the mission to be sustainable in a new way by using resources found on the moon itself.

Of course, sending humans off the planet is expensive, especially when trying to take them farther than any man has traveled before. To achieve its aims, NASA intends to double down on partnerships with commercial companies and to encourage even more commercial activities in space.

Currently, NASA has commercial space programs such as the commercial resupply missions for the International Space Station and the upcoming commercial crew program. It wants to expand these programs to include commercial habitation programs too, especially in low-earth orbit where the International Space Station (ISS) resides.

Earlier this year, NASA caused some controversy when it announced that the ISS would begin accepting space tourists as early as 2020. Experts worried about the precedent of allowing wealthy individuals to visit a scientific research outpost and about the potential environmental costs of transporting those passengers into space.

Others have raised concerns that NASA may be putting itself in competition with private companies, but Bridenstine doesnt see it that way. NASA wants to be a customer, he explained. One of many in a very robust marketplace in low-earth orbit. The type of work that can be done in low-earth orbit includes bio-medicine projects like the 3D printing of organs using adult stem cells, or the creation of artificial retinas. This work cant be performed on Earth because of the way materials are layered, but it can be done in the microgravity of space.

The hope is that the proliferation of commercial programs will lower costs for NASA: We want those companies to go out and get customers that arent us, to drive down costs, Bridenstine said.

An example of this commercialization in action will be the delivery of the VIPER rover, the lunar rover that should land in 2022. The rover will be built by NASA, but it will be delivered by a private company through the Commercial Lunar Payload Services, or CLPS, program.

Partnerships between NASA and private companies have not always worked out smoothly, however. Bridenstein publicly criticized SpaceX CEO Elon Musk recently for showing off the companys new Starship rocket while its contributions to the commercial crew program are years behind schedule. And similar scheduling issues have beset Boeings crew capsule, the Starliner, which has also been delayed.

Time will tell if the involvement of private companies is the key to a more affordable space program, or whether a focus on profits over exploration will undermine the efforts of NASA and other government space agencies.

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NASA plans to cut cost of space exploration by commercializing low-earth orbit - Digital Trends

BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference – BioSpace

NEW YORK, Oct. 25, 2019 (GLOBE NEWSWIRE) -- BrainStorm Cell Therapeutics Inc. (NASDAQ: BCLI), a leader in the development of innovative autologous cellular therapies for highly debilitating neurodegenerative diseases, today announced that it will be presenting at the Dawson James Securities 5th Annual Small Cap Growth Conference, being held on October 28-29, 2019 at the Wyndham Grand Hotel in Jupiter, Florida.

Preetam Shah, PhD, MBA, Chief Financial Officer is scheduled to present on Tuesday, October 29th at 3:40 p.m. Eastern Time, in Track 2 - Preserve Ballroom B, with one-on-one meetings to be held throughout the conference.

Chaim Lebovits, President and CEO of BrainStorm said, We are pleased to have the opportunity to have Dr. Shah present at the Dawson James Small Cap Growth Conference. Dr. Shah, joined BrainStorm in September 2019, and we look forward to having him present the Companys growth strategy and future to a wide audience of accreditied investors.

About NurOwnNurOwn (autologous MSC-NTF cells) represent a promising investigational approach to targeting disease pathways important in neurodegenerative disorders. MSC-NTF cells are produced from autologous, bone marrow-derived mesenchymal stem cells (MSCs) that have been expanded and differentiated ex vivo. MSCs are converted into MSC-NTF cells by growing them under patented conditions that induce the cells to secrete high levels of neurotrophic factors. Autologous MSC-NTF cells can effectively deliver multiple NTFs and immunomodulatory cytokines directly to the site of damage to elicit a desired biological effect and ultimately slow or stabilize disease progression. NurOwn is currently being evaluated in a Phase 3 ALS randomized placebo-controlled trial and in a Phase 2 open-label multicenter trial in Progressive MS.

About BrainStorm Cell Therapeutics Inc.BrainStorm Cell Therapeutics Inc. is a leading developer of innovative autologous adult stem cell therapeutics for debilitating neurodegenerative diseases. The Company holds the rights to clinical development and commercialization of the NurOwn Cellular Therapeutic Technology Platform used to produce autologous MSC-NTF cells through an exclusive, worldwide licensing agreement. Autologous MSC-NTF cells have received Orphan Drug status designation from the U.S. Food and Drug Administration (U.S. FDA) and the European Medicines Agency (EMA) in ALS. BrainStorm has fully enrolled the Phase 3 pivotal trial in ALS (NCT03280056), investigating repeat-administration of autologous MSC-NTF cells at six sites in the U.S., supported by a grant from the California Institute for Regenerative Medicine (CIRM CLIN2-0989). The pivotal study is intended to support a BLA filing for U.S. FDA approval of autologous MSC-NTF cells in ALS. BrainStorm received U.S. FDA clearance to initiate a Phase 2 open-label multi-center trial of repeat intrathecal dosing of MSC-NTF cells in Progressive Multiple Sclerosis (NCT03799718) in December 2018 and has been enrolling clinical trial participants since March 2019. For more information, visit the company's website.

Safe-Harbor Statements Statements in this announcement other than historical data and information, including statements regarding future clinical trial enrollment and data, constitute "forward-looking statements" and involve risks and uncertainties that could cause BrainStorm Cell Therapeutics Inc.'s actual results to differ materially from those stated or implied by such forward-looking statements. Terms and phrases such as "may", "should", "would", "could", "will", "expect", "likely", "believe", "plan", "estimate", "predict", "potential", and similar terms and phrases are intended to identify these forward-looking statements. The potential risks and uncertainties include, without limitation, BrainStorms need to raise additional capital, BrainStorms ability to continue as a going concern, regulatory approval of BrainStorms NurOwn treatment candidate, the success of BrainStorms product development programs and research, regulatory and personnel issues, development of a global market for our services, the ability to secure and maintain research institutions to conduct our clinical trials, the ability to generate significant revenue, the ability of BrainStorms NurOwn treatment candidate to achieve broad acceptance as a treatment option for ALS or other neurodegenerative diseases, BrainStorms ability to manufacture and commercialize the NurOwn treatment candidate, obtaining patents that provide meaningful protection, competition and market developments, BrainStorms ability to protect our intellectual property from infringement by third parties, heath reform legislation, demand for our services, currency exchange rates and product liability claims and litigation,; and other factors detailed in BrainStorm's annual report on Form 10-K and quarterly reports on Form 10-Q available at http://www.sec.gov. These factors should be considered carefully, and readers should not place undue reliance on BrainStorm's forward-looking statements. The forward-looking statements contained in this press release are based on the beliefs, expectations and opinions of management as of the date of this press release. We do not assume any obligation to update forward-looking statements to reflect actual results or assumptions if circumstances or management's beliefs, expectations or opinions should change, unless otherwise required by law. Although we believe that the expectations reflected in the forward-looking statements are reasonable, we cannot guarantee future results, levels of activity, performance or achievements.

CONTACTS

Corporate:Uri YablonkaChief Business OfficerBrainStorm Cell Therapeutics Inc.Phone: 646-666-3188uri@brainstorm-cell.com

Media:Sean LeousWestwicke/ICR PRPhone: +1.646.677.1839sean.leous@icrinc.com

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BrainStorm Cell Therapeutics to Present at the Dawson James Securities 5th Annual Small Cap Growth Conference - BioSpace

CAR T-cell therapy in Ireland – Independent.ie

As with all new drugs, cost is a barrier to application.

Professor Owen Smith, consultant paediatric haematologist at Our Lady's Children's Hospital, Crumlin, says the hospital is in close discussions with Novartis to bring the drug to Irish children with refractory relapsed leukaemia. Smith estimates they will be treating four to seven young patients a year.

However, at the end of September, the National Centre for Pharmacoeconomics recommended that Kymriah not be considered for reimbursement by the HSE in Ireland unless cost-effectiveness can be improved relative to existing treatments.

Smith says the HSE is already de facto funding CAR T-cell therapy. Two Irish children this year received the therapy in England, under the HSE Treatment Abroad Scheme.

Both children are now what Smith calls MRD negative: "They have no evidence that they have leukaemia," he says. "We now want to have these therapies done here as it's easier for the family unit.

"These treatments cost about 320,000 and the hospital can't carry the cost - it will have to be provided by the HSE. If these children are deemed eligible for CAR T and it is the standard of care for such patients in the UK and across the EU - do we just send these patients off on the Treatment Abroad Scheme? The money is still coming out of the public purse but we're losing the expertise to perform the therapies here. We will be saying to the HSE they have to fund this.

"Where we are with CAR T-cell therapy is not where we are going to be in five years' time," adds Professor Smith, who is the national clinical lead for children and adolescent/young adults (AYA) with cancer at the National Cancer Control Programme.

He says that an agreement has been reached across 15 EU countries that treatment will begin next year for 15,000 high-risk ALL patients aged from one to 44.

"It is pointless giving these patients chemo. It just makes them sicker. The Government needs to come to terms with the fact. CAR T is a new therapy but it's going to get bigger; it is going to be rolled out not just for leukaemia but also for other cancers such as ovarian cancer."

Martin Pule and Claire Roddie's clinical trials are not yet available to Irish patients.

Indo Review

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CAR T-cell therapy in Ireland - Independent.ie

EXCLUSIVE: Brian Cage Talks His Back Injury From Rebellion and How Stem-Cell Therapy Greatly Helped – LordsofPain.net

During yesterdays IMPACT media call, I had the pleasure of speaking with heavyweight champion Brian Cage, where I asked the former Lucha Underground star about the trip he took to Columbia for Stem-Cell therapy to rehab his back from an injury sustained at the Rebellion pay per view last April. Cage recalled the incident as one of the most painful in his career, but explains that the treatment was greatly beneficial, and highly recommends it to those in the industry. He also reveals how the guy who got him involved is a huge fan of the business, which has led to a wave of wrestlers traveling to Columbia to heal their bodies.

The injury was awful. One of the most painful moments of my career. I literally thought my career might be over. It kept me off TV and being able to defend my title for quite some time. I was doing everything under the sun to try and recover from that, including going to Columbia and getting the stem-cells, and I do believe it did have a substantial benefit to it. The guy that got me involved in that is a huge fan of wrestling. Thats why if you havent noticed theres been a handful of wrestlers going down there lately. Ive already been semi-educated and into stem-cells as well. Just the healing nature and what the benefits are and its a shame that you cant get the actual umbilical stem-cells here in the states.

The champ would then state that he will most-likely be going back for future injuries and maintenance.

YesIt helped me get past this. Its beneficial, and more likely be doing it again in the future. For maintenance or any other injury that may occur.

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EXCLUSIVE: Brian Cage Talks His Back Injury From Rebellion and How Stem-Cell Therapy Greatly Helped - LordsofPain.net