Beam Therapeutics Announces Collaboration and Exclusive License Agreement with Prime Medicine for Prime Editing Technology – Business Wire

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Beam Therapeutics, a biotechnology company developing precision genetic medicines through base editing, today announced that it has entered into a collaboration and license agreement with a newly-formed company, Prime Medicine, Inc. to research and develop a novel gene editing technology called prime editing, recently developed by one of Beams co-founders, David Liu, Ph.D., and his group at the Broad Institute of Harvard and MIT.

Under the agreement, Beam has the exclusive right to develop prime editing technology for the creation or correction of any single-base transition mutations, as well as for the treatment of sickle cell disease, both of which Beam is already pursuing with its base editing technology. Transition mutations (e.g. A to G, C to T) are the largest single class of disease-associated genetic mutations, and are also potentially treatable with base editing. Beam plans to evaluate prime editing technology for potential use in future programs.

Part of Beams strategy is to continue to access emerging technologies in gene editing and delivery, while finding new ways to create meaningful options for patients. Our collaboration with, and contribution to the formation of, Prime Medicine is a great example of that approach, allowing us to incorporate prime editing into the Beam platform, said John Evans, chief executive officer of Beam. This partnership enables both companies to advance the technology in distinct spaces, with Beam focusing on the kinds of edits that are most similar to our base editing technology.

As part of the collaboration, Beam is providing initial interim leadership to Prime Medicine for the first year of the collaboration, and will have the right to designate a member on Prime Medicines board. The parties will also grant each other non-exclusive licenses to certain CRISPR technology and delivery technology to enable the development of prime editing products.

About Beam Therapeutics

Beam Therapeutics is developing precision genetic medicines through base editing. Founded by leading scientists in CRISPR gene editing, Beam is pursuing therapies for serious diseases using its proprietary base editing technology, which can make precise edits to single base pairs in DNA and RNA. Beam is headquartered in Cambridge, Massachusetts. For additional information, visit http://www.BeamTx.com.

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Beam Therapeutics Announces Collaboration and Exclusive License Agreement with Prime Medicine for Prime Editing Technology - Business Wire

Editas Medicine In The Realm Of Designer Humans – Seeking Alpha

Comparison is the essential valuation activity

Readers familiar with our work may want to skip to the "Comparing Details" section below.

Advances in Genome understanding and prospective adjustments continue at a startling pace. Equity investments provide a means of maintaining perspective and other SA contributors help with the industry development details.

This article rewards investors who choose to direct their investments of TIME and capital to those alternatives with the highest likelihood of successful rates of return among ones compared under identical important measures.

Now, Editas Medicine (EDIT) provides attractive answers to these questions:

These are questions often neither asked nor answered by many investment analysis reports. The commonplace approach is to present those aspects of one investment which may set it apart from others, but fail to make the essential decision-supporting step of comparing alternatives on an equal-measure basis.

Instead of limiting a value-search to industry competitors, our focus is on the alternatives which may address the investor's objectives of his investing mission, not necessarily of a stock candidate's competitive industry concerns.

To that end we turn to an information expert on what is currently of importance to investors - YAHOO Finance - and what also diverts the attention of investors initially interested in the stock of Editas Medicine. From its website traffic records Yahoo lists five other stocks "People also watch." They are NTLA, SGMO, BLUE, ONCE, and CLLS.

With the alternative investment choices in hand, we then turn to folks involved every market day with making bets (usually big bets) about how far their prices are likely to get pushed - both up and down. Those price limits are defined in the choices they make in the way they will protect their capital which must be exposed to market risk as they conduct their every-day market-making activities in large-volume "block trade" transactions.

Price range forecasts are being made in separate hedging deals over time periods defined by the contract lives of the derivative securities involved. Those judgments contain the updated "fundamentals" of interest to all evaluators, collected 24x7 by armies of market-maker (MM) employees world-wide.

Such forecasts are constantly being refined every moment investment markets are operating, and are made part of every market-day's closing records. They provide an historical record (in subsequent market price actions) of how well the "smart money" can make useful forecasts - for specific stocks, ETFs, and indexes.

Present-day markets are driven by major investing organizations commanding multi-billion dollar portfolios with stock contents which can only be adjusted by negotiated volume (block) trades between peers, not by "open auction". Such trades set and move posted prices.

The individual investor typically is merely along for the ride. He/she needs to have a sense of where the negotiators are likely to head, pricewise. Conventional analysis often provides superficial descriptions and little linkage between operating minutia and price forecasts. As an example here is how Yahoo Finance reports on EDIT.

Editas Medicine, Inc. operates as a clinical stage genome editing company. The company focuses on developing transformative genomic medicines to treat a range of serious diseases. It develops a proprietary genome editing platform based on CRISPR technology to target genetically addressable diseases and therapeutic areas. The company develops EDIT-101 for Leber Congenital Amaurosis type 10, a genetic form of vision loss that leads to blindness in childhood. It also develops other therapies for eye diseases, such as Usher Syndrome 2A, which is a form of retinitis pigmentosa that also includes hearing loss; Retinitis Pigmentosa, a progressive form of retinal degeneration; and Herpes Simplex Virus 1 that causes lifelong infections leading to ocular and oral disease.

In addition, the company develops hematopoietic stem cells for treating sickle cell disease and beta thalassemia. It has a research collaboration with Juno Therapeutics, Inc. to develop engineered T cells for cancer; strategic alliance and option agreement with Allergan Pharmaceuticals International Limited to discover, develop, and commercialize new gene editing medicines for a range of ocular disorders; and strategic research collaboration and cross-licensing agreement with BlueRock Therapeutics to combine their respective genome editing and cell therapy technologies to discover, develop, and manufacture engineered cell medicines. The company also has a strategic research collaboration agreement with Editas Medicine, Inc. to explore in vivo delivery of genome editing medicines to treat neurological diseases. The company was formerly known as Gengine, Inc. and changed its name to Editas Medicine, Inc. in November 2013. Editas Medicine, Inc. was founded in 2013 and is headquartered in Cambridge, Massachusetts.

Not much help, is it?

Instead, here is what the MMs' hedging actions tell today about how high (and low) the stocks might get priced, and how they each behaved subsequent to prior forecasts like the ones now being seen:

Figure 1

The essence of valuation is in comparison, which requires that the compared measures be as close to identical as possible. To that end we place all of our valuations in a carefully defined set of measures, and describe them in as parallel set of comparisons as is possible.

To do so often presents what many readers recognize as text and ideas they have encountered before, as they have in our just-published comparison between Microsoft and Boeing. The use of the heading for this section of the article as an accelerant to reading provides for experienced readers an economy of time and effort, while leaving for the newly-initiated the opportunity for an important introduction.

What is important to us in this analysis is how big a price gain is in prospect, column [E], and how likely is today's RI forecast to produce a profit [H] as a proportion of the [L] sample of such forecasts. That combination result appears in the [I] %payoff which includes loser forecasts as well as the 82% winners. The size of [I] relative to [E] is a measure of [E]'s credibility in [N].

Time required [J] to accomplish the payoff is another important dimension for any investment mission. The retirement, tuition, or health emergency clock won't patiently wait for "long-term-trend" investments to be "sure" (like EK, GM, GE, and others) of their "passive investment" buy&hold strategy results. Compound Annual Gain Rates (CAGR) are the essential measures [K]. Figure 3's rows are ranked by the historical results (of today's RI) statistic.

One additional complication of being time-efficient in an investment strategy is that the score-keeping can't be easily sliced up into uniform time periods. That is not what happens to holdings in an active investment strategy. Gains (and losses) occur in irregular lumps of time, and we need to evaluate likely prospects in the way they may be accumulated.

What is done in proper financial analysis of any capital commitment is to anticipate the RATE of gain or cost in units of change per time of involvement. The most commonly used measure is basis points per day, where a basis point is 1/100th of a percent.

That's a tiny unit, but is what works best. Put together and maintained each day for a year, 19 of them would double your investment. They can be powerful.

In Figure 1 we use the Odds of gain [H] as a weight for the average prior payoffs [I], and take the complement of [H] ( 100 - H ) as a weight for the risk prospect [F]. Put together as [O] + [P] in [Q] we have an odds-weighted net outcome of each row's prior MM RI forecast sample [L]. Then by converting those [Q] nets into bp/day in [R] we have a guide to making investment selection decisions across a broader array of alternatives.

Taking measurements into such precision where the potentials are great for errors in forecasting may only serve to point out the limits of their usefulness. And where the differences between alternatives are great, the efforts involved may not be practical. But the fact remains that investors and investment organizations are determined to risk capital in these uncertain ventures, and progress in some areas of the unknown need guide-rails.

So figure 1 may tell us that if the future is repeated then in this case EDIT is a superior portfolio choice to any of the five alternatives generally being considered by investors consulting Yahoo Finance's data. EDIT's stock superiority lies principally in that in the next 3 months it has better odds of seeing a price +19% higher while encountering interim price drawdowns not significantly worse than any of the alternative stock investments, and do it in approximately the next 8 weeks.

That rate of gain is a CAGR of +201%, which compares to a probable parallel investment in the S&P 500 index ETF (SPY) of some +15%. Out of a population of over 2,700 other coming price range forecasts from the same source an average CAGR of +17% involves roughly equal interim risk exposure of -10% which is typically not only encountered, but also suffered in terms of actual capital loss experience, reducing that forecast population's expected +14% gain to only +3% in the process.

Meanwhile, the best 20 candidate stock investments from that population actually achieved +19% gains, better than the +14% expected, and by being alert to day-by-day price moves accomplished them in the same 8 week average holding period of EDIT, for CAGR rewards at a 342% rate.

Will all these price changes be repeated? Probably not precisely, but at least you have some norms to compare with.

To provide a sense of how EDIT price range forecasts by MMs have been trending recently Figure 2 shows daily expectations for the past 6 months and Figure 3 shows once-a-week excerpts over the past two years.

Figure 2

The small blue picture here shows the distribution of forecast Range Indexes during the past 5 years' 1261 market days. Recording the daily balance between expected upside and downside price changes likely, the current measure of 29 is clearly below its visual average, supporting the 82/100 odds of higher coming prices.

Figure 3

Editas Medicine presents evidences of coming price change prospects which are superior to many relevant equity investment alternatives, and justify its buy as a near-term wealth-building investment.

Disclosure: I/we have no positions in any stocks mentioned, but may initiate a long position in EDIT over the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

Additional disclosure: Peter Way and generations of the Way Family are long-term providers of perspective information, earlier helping professional investors and now individual investors, discriminate between wealth-building opportunities in individual stocks and ETFs. We do not manage money for others outside of the family but do provide pro bono consulting for a limited number of not-for-profit organizations.

We firmly believe investors need to maintain skin in their game by actively initiating commitment choices of capital and time investments in their personal portfolios. So, our information presents for D-I-Y investor guidance what the arguably best-informed professional investors are thinking. Their insights, revealed through their own self-protective hedging actions, tell what they believe is most likely to happen to the prices of specific issues in coming weeks and months. Evidences of how such prior forecasts have worked out are routinely provided in the SA blog of my name.

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Editas Medicine In The Realm Of Designer Humans - Seeking Alpha

Hemanext Announces CE Mark Submission for the HEMANEXT ONE Red Blood Cell Processing System – Benzinga

Regulatory filing is a major step toward commercialization of transfusion-medicine innovation

LEXINGTON, Mass. (PRWEB) November 01, 2019

Hemanext Inc., a privately held medical technology company dedicated to improving patient outcomes through fewer and better red blood cell (RBC) transfusions, today announced CE Mark submission for HEMANEXT ONE, the company's initial product offering that is designed to provide a higher-quality blood product for transfusion patients.

The HEMANEXT ONE processing system reduces the fuel for oxidative damage prior to and during storage, and converts a unit of conventional, leukoreduced RBC into a transfusion-ready unit of HEMANEXT ONE RBC.

"We have seen a growing body of scientific evidence that oxidative damage to RBCs could have a negative impact on clinical outcomes for many acute and chronic patient populations that require large volumes of transfusions," said Paul M. Ness, MD, Senior Director, Division of Transfusion Medicine and Professor of Pathology, Johns Hopkins University School of Medicine; Editor, Transfusion, the peer-reviewed journal of the American Association of Blood Banks (AABB) (2003-2018); Past President, AABB. "Improving the quality of RBCs could enhance patient care and simultaneously reduce the quantity of RBC units required to treat many diseases and conditions."

Hemanext anticipates receiving CE Mark approval in the first half of 2020, with commercialization initiatives for the HEMANEXT ONE system in Europe commencing in the second half of the year. Hemanext also expects to submit a 510(k) premarket notification to the U.S. Food and Drug Administration (FDA) in 2020.

"Our CE Mark submission is a significant milestone for Hemanext. It is the culmination of years of research and collaborations with the transfusion medicine community in Europe," said Hemanext President and CEO Martin Cannon. "We are grateful for the partnerships that have brought us another step closer to creating a world with fewer and better transfusions."

ABOUT HEMANEXT

Hemanext is a privately held medical technology company dedicated to improving the quality, safety, efficacy and cost of transfusion therapy. The company's research and development efforts center on the study and future commercialization of hypoxically stored red blood cells (RBCs). HEMANEXT ONE, our initial product offering, is designed to improve the quality of life for chronic and high-volume acute transfusion patients while reducing costs.

Visit Hemanext.com to learn more.

Dr. Ness serves as a clinical advisor to Hemanext.

For the original version on PRWeb visit: https://www.prweb.com/releases/hemanext_announces_ce_mark_submission_for_the_hemanext_one_red_blood_cell_processing_system/prweb16693599.htm

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Hemanext Announces CE Mark Submission for the HEMANEXT ONE Red Blood Cell Processing System - Benzinga

Co-Administration Of iRGD Enhances Tumor-Targeted Delivery And Anti-Tu | IJN – Dove Medical Press

Yi Zhong,1,* Tao Su,1,* Qiuxiao Shi,1 Yanru Feng,1 Ze Tao,1 Qiuxia Huang,1 Lan Li,1 Liqiang Hu,1 Shengfu Li,1 Hong Tan,2 Shan Liu,3 Hao Yang1

1Key Lab of Transplant Engineering and Immunology, MOH, West China-Washington Mitochondria and Metabolism Research Center, West China Hospital, Sichuan University, Chengdu 610041, Peoples Republic of China; 2Department of General Surgery, Chengdu Integrated TCM & Western Medicine Hospital (Chengdu First Peoples Hospital), Chengdu 610041, Peoples Republic of China; 3Department of Laboratory Medicine, Sichuan Academy of Medical Sciences & Sichuan Provincial Peoples Hospital, University of Electronic Science and Technology of China, Chengdu 610072, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Shan LiuDepartment of Laboratory Medicine, Sichuan Academy of Medical Sciences & Sichuan Provincial Peoples Hospital, No. 32, West Second Section First Ring Road, Chengdu, Sichuan 610072, Peoples Republic of ChinaTel +86 28 6138 2518Email shanliusyy@163.comHao YangKey Lab of Transplant Engineering and Immunology, MOH, West China Hospital, No. 1, Keyuan 4th Road, Hi-tech Zone, Chengdu 610041, Peoples Republic of ChinaTel +86 28 85164031Email yanghao@scu.edu.cn

Background: Nanoparticles exhibit great promise for improving the solubility and tissue-specific distribution of chemotherapeutic agents; however, the passive and highly variable enhanced permeability and retention (EPR) effects observed in tumors frequently leads to insufficient delivery of nanodrugs into tumors. The tumor-penetrating peptide iRGD can actively enhance tumor-selective delivery of nanoparticles into tumors by binding to integrin and interacting with tissue-penetrating receptor neuropilin-1.Materials and methods: To improve colorectal cancer treatment, in this study, we prepared a paclitaxel (PTX)-loaded PLGA nanoparticle (PLGA-PTX) and evaluated its tumor-targeting and antitumor activity by co-administration with iRGD.Results: Compared to free PTX, encapsulated PTX retained preferential cytotoxicity toward various colorectal cancer cells while effectively sparing healthy cells. PLGA-PTX treatment resulted in cell cycle arrest at the G2/M phase and apoptosis, leading to inhibition of cancer cell migration and invasion. PLGA-PTX combined with iRGD displayed little enhancement of cytotoxicity in vitro. Despite this, iRGD receptors integrin and neuropilin-1 were found to be primarily overexpressed on abundant tumor vessels in mice bearing colorectal tumors. Consequently, co-administration of nanoparticles with iRGD promoted the selective delivery of nanoparticles into tumor tissues in vivo. Additionally, the combined regimen enhanced the antitumor effects compared to those of each individual reagent.Conclusion: Our findings suggest that PLGA nanoparticles combined with the iRGD peptide provide a promising drug delivery strategy for facilitating active drug accumulation into tumors, given that iRGD receptors are overexpressed on tumor vessels. This co-administration system lacking covalent conjugation provides a more convenient means to combine various therapeutic agents with iRGD to achieve personalized nanotherapy.

Keywords: tumor penetrating peptide, nanoparticle, iRGD, paclitaxel, tumor vasculature, colorectal cancer

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

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Severe Vision Loss From Niacin (Vitamin B3) Can Be Reversed – SciTechDaily

In a first-of-its-kind clinical report, retina specialists at the New York Eye and Ear Infirmary of Mount Sinai (NYEE) have shown that severe vision loss from a self-prescribed high dose of over-the-counter niacin is linked to injury of a specific cell type in a patients eye. The experts report that discontinuing the vitamin led to reversal of the condition and have published their findings in the fall issue of Journal of VitreoRetinal Diseases.

Niacin, also known as vitamin B3, is used for lowering hyperlipidemia or cholesterol and comes in prescription and over-the-counter forms; it can produce a rare toxic reaction called niacin-induced cystoid maculopathy, a form of retinal swelling.

Just because nutritional supplements are available without prescription does not mean they are completely safe to use without supervision. Jessica Lee, MD

People often live by the philosophy that if a little bit is good, more should be better. This study shows how dangerous large doses of a commonly used over-the-counter medication can be, said lead investigator Richard Rosen, MD, Chief of Retina Services at NYEE and the Mount Sinai Health System. People who depend on vision for their livelihood need to realize there could be long-lasting consequences from inadvertent overdosing on this vitamin.

This case serves to remind everyone about the importance of talking to ones physician prior to taking any supplement, or over-the-counter product. Just because nutritional supplements are available without prescription does not mean they are completely safe to use without supervision, explains corresponding author Jessica Lee, MD, Assistant Professor of Ophthalmology at the Icahn School of Medicine at Mount Sinai. No matter how benign a supplement or over-the-counter product may seem, the correct dosage and potential interactions with other medications should be carefully reviewed with a doctor, to avoid preventable unexpected consequences. This case illustrates how dangerous casual self-prescribing of megadoses of vitamins can be.

Sufficient niacin, also called vitamin B3, is important for general good health. However, like many nutrients, too much can be damaging to the human body. To be effective in reducing cholesterol, high doses are needed, which can pose health risks including vision problems, liver damage, glucose intolerance, and gastrointestinal problems.

The team of investigators from NYEE reported on a 61-year-old patient who arrived at the hospital with worsening blurry vision in both eyes that began a month earlier. The initial exam showed that the patient was almost legally blind, with best-corrected visual acuity of 20/150 in the right eye and 20/100 in the left eye. The patient told doctors his medical history included significant hypertension and hyperlipidemia, but initially failed to disclose the extent of his self-prescribing. Subsequently, he admitted to taking an extensive list of supplements, which included three to six grams of niacin daily for several months to reduce his risk of cardiovascular events and was unaware of the risk to his eyesight. He purchased the supplement at a drug store after a doctor told him he had high cholesterol. The standard dosage is one to three grams a day with a maximum dose of six grams, but doctors typically warn against treating the condition with supplements purchased over the counter and prefer to prescribe and monitor an FDA-approved dose of niacin.

While retina specialists have been aware of this unusual reaction to niacin for many years, such a textbook example of extreme toxicity and recovery has never been as well documented by imaging and functional testing. Richard Rosen, MD

NYEE clinicians diagnosed the problem using several state-of-the-art technologies, including fluorescein angiography, optical coherence tomography (OCT), and multifocal electroretinography (MERG), to examine his retina for evidence of cellular damage and monitor his response to therapy. Fluorescein angiography uses fluorescent dye to trace blood flow through retinal arteries and veins, looking for leaks. OCT is an advanced structural imaging technique that reveals the cross-sectional details of the retina cells, layer by layer. MERG measures the electrical signals from different layers of cells in the eyes.

The imaging allowed investigators to diagnose a rare toxic reaction called niacin-induced maculopathy. The high dose of niacin led to cystoid macular edema of the retina, which is fluid in the macula (a small area in the center of the retina that produces detailed and centralized vision) that causes swelling. The technology also allowed investigators to identify the cellular structures responsible for the patients condition. The MERG recorded in this case showed reduced b-waves, which indicated that cells affected by the toxicity were the Muller cells, which span the depth of the retina like support columns.

Discontinuation of the vitamin reversed this effect and restored retinal function and electrical signals. This led investigators to demonstrate, for the first time, that the Muller cells were the target of niacin toxicity, and the cause of niacin maculopathy.

This case illustrates how dangerous casual self-prescribing of megadoses of vitamins can be. Jessica Lee, MD

Ophthalmologists from NYEE explained the situation to the patient and advised him to stop taking the over-the-counter niacin immediately. At his one-week follow-up appointment, his vision had noticeably improved. Two months later, the dysfunction had completely resolved and his vision was back to 20/20. Through the high-tech structural and metabolic imaging, researchers observed that the patients Muller cells had gradually yet dramatically recovered.

While retina specialists have been aware of this unusual reaction to niacin for many years, such a textbook example of extreme toxicity and recovery has never been as well documented by imaging and functional testing, said Dr. Rosen.

In this instance the patient was particularly fortunate that the physicians who saw him were alert to the possible cause and were able to confirm their diagnostic suspicions with appropriate testing. This may not always be the case and other patients may not have such a successful outcome, added Dr. Lee.

Reference: Optical Coherence Tomography, Fluorescein Angiography, and Electroretinography Features of Niacin Maculopathy: New Insight Into Pathogenesis by Jessica G. Lee, MD, Anu Patel, MD, Alessandra Bertolucci, MD and Richard B. Rosen, MD, 1 October 2019, Journal of VitreoRetinal Diseases.DOI: 10.1177/2474126419877567

Niacin, also known as nicotinic acid, is an organic compound and a form of vitamin B3, an essential human nutrient. It has the formula C6H5NO2 and belongs to the group of the pyridinecarboxylic acid.

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Severe Vision Loss From Niacin (Vitamin B3) Can Be Reversed - SciTechDaily

ACS 2019: Outcomes in Renal Cell Carcinoma (RCC) Patients Undergoing Inferior Vena Cava (IVC) Ligation versus Thrombectomy: A Retrospective,…

San Francisco, California (UroToday.com) Gordon Hong noted radical nephrectomy with removal of tumor thrombus is considered standard of care in patients with non-metastatic renal cell carcinoma (RCC). Surgical resection usually consists of nephrectomy and thrombectomy; however, ligation of the IVC is sometimes necessary. This study assessed the outcomes, particularly functional outcomes, in RCC patients undergoing IVC ligation compared to caval thrombectomy.

They conducted a case-controlled, retrospective study at a high-volume single-center institution. All RCC patients undergoing ligation were matched with thrombectomy patients in a 1:2 ratio based on pre-operative renal function, Charlson Comorbidity Index (CCI), and race. Endpoints were complications, changes in renal function, and mortality.

A total of 20 IVC ligation patients were matched with 40 thrombectomy patients. Median follow-up time was 15.3 months for both groups. There was no statistical difference in major complications (Clavien-Dindo Grade 3a+) between cohorts in their postoperative hospital stay (p=0.345) or at 3 months (p=0.464). Average decline in estimated glomerular filtration rate (eGFR) from baseline to 6-months post-surgery for ligation and thrombectomy was 4.6 mL/min/1.73m2 and 6.0 mL/min/1.73m2, respectively (p=0.767). The difference in the likelihood of lymphedema development (OR 0.15, 95% CI: 0.02-1.03, p=0.054) was not statistically significant, though more ligation patients experienced lymphedema development. Moreover, differences in all-cause mortality (p=0.197) and cancer-specific mortality (p=1.00) were also not statistically significant at all time points.

This study represents one of the largest cohort of RCC patients undergoing IVC ligation to date. Although IVC ligation patients experience more complications, they have similar functional and oncologic outcomes. These data are useful for patient counseling and representative of a single high volume surgeon experience which findings may not be generalizable to others

Presented by:Gordon Hong, BS, Emory University School of Medicine, Atlanta, Georgia

Written by: Stephen B. Williams, MD, Medical Director for High Value Care; Chief of Urology, Associate Professor, Director of Urologic Oncology, Director Urologic Research, The University of Texas Medical Branch at Galveston, TX atthe2019 American College of Surgeons (ACS), #ACSCC19, October 2731 in San Francisco, California

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ACS 2019: Outcomes in Renal Cell Carcinoma (RCC) Patients Undergoing Inferior Vena Cava (IVC) Ligation versus Thrombectomy: A Retrospective,...

In June, FDA announced a patient died from a fecal transplant. Now the doctors are speaking out. – The Daily Briefing

Doctors from the hospital that treated a patient who died after a fecal microbiota transplant published a case study on Wednesday in the New England Journal of Medicine that offers more details on the case as well as another case in which a patient became severely ill from the procedure.

How to eradicate antibiotic overuse

FDA announced in June that it had become aware that patients who undergo FMT can experience severe or life-threatening bacterial infections caused by drug-resistant bacteria. FDA said two patients with weakened immune systems who received FMT developed infections stemming from extended-spectrum-beta-lactamase (ESBL) producing E. coli, which is resistant to antibiotics. FDA reported that one of those patients had died.

FDA said both FMTs involved stool from the same donor. The donor's stool had not been tested for ESBL-producing gram-negative organisms before the providers conducted the transplants, according to FDA.

FDA said the donor's stool underwent lab tests after the two patients experienced adverse reactions, and the tests confirmed the stored stool contained ESBL-producingE. coliidentical to the bacteria found in stool used in the two transplants.

As a result of the developments, FDA issued new safety guidelines for FMTs, saying it would now require FMTs to involve:

FDA did not provide many details on the two cases, which led doctors from Massachusetts General Hospital, where the patients were treated, to issue a report detailing the cases. Elizabeth Hohmann, co-author of the report and associate professor of medicine and infectious diseases at Mass General and Harvard Medical School, said, "We wanted to set the record straight."

According to the report, both patients were involved in clinical trials to see if FMTs could be used as a potential therapy for their conditions. One patient was in a trial to learn whether FMTs could help improve brain function in patients with severe liver disease. The other was participating in a trial to see if FMTs could be used to help immune function in leukemia patients who had undergone chemotherapy and stem cell transplants.

Two and a half weeks after doctors administered the final FMT dose to the liver disease patient, a form of E. coli was found in the patient's bloodstream, the report said. The patient recovered after intravenous antibiotics killed the bacteria.

The leukemia patient also developed the same form of drug-resistant E. coli. However, the patient had taken drugs to suppress his immune system as part of a bone marrow transplant and began to decline faster, the report said. Eight days after his last FMT dose, the patient was placed on a ventilator, and two days later the patient died from a severe bloodstream infection, according to the report.

Upon investigation of the liver patient's infection, doctors discovered that the stool sample used for the FMT contained the drug-resistant organism.

According to Hohmann, the stool donor was "what I call a 'screamingly healthy person.' Only about one in 40 people who think they might be healthy enough to [donate stool] actually turn out to meet all of our criteria. [The donor] had none of the 'risk factors' for carrying these organisms. They could not recall the last time they received antibiotics, had zero medical history, no international travel. Plus, they completed all of the other screening tests."

The doctors had been following FDA protocol testing stool donations for infectious bugs, but were not instructed by FDA to test or destroy older stool samples kept in storage, Hohmann said. The stool sample that sickened the two patients in the report had been stored in a freezer for several months.

"It wasn't obvious to a lot of smart people here," Hohmann said. "We didn't think to go back in time."

Hohmann said the report should serve as "a cautionary and sad tale. It points out some of the important medical issues about immune-compromised [patients] and maybe that changing the microbiome is not always a good idea."

Alexander Khoruts, a professor of medicine and medical director of the Microbiota Therapeutics Program at the University of Minnesota who was not involved in the report, said the report should "set off alarm bells for those who thought that [FMTs were] risk free." He added that Mass General "did the right thing" by sharing details and that he hopes the report will lead doctors to be more cautious.

Stuart Johnson, an associate professor of medicine at Loyola University Stritch School of Medicine, who specializes in the bacterial gut infection Clostridium difficile, said he thinks the report "points out that we don't know everything that's in someone's feces, and I think widespread adoption of this practice is problematic" (Carroll, NBC News, 10/30; Jacobs, New York Times, 10/30; Smith, Medium, 10/30).

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In June, FDA announced a patient died from a fecal transplant. Now the doctors are speaking out. - The Daily Briefing

‘Any One of Us’ Is More Than an Inspirational Film – Outside

The first time I watched the documentary Any One of Us was about four months before my fianc, Andrew Bernstein, was hit by a van and very nearly killed while riding his bike.

At the time, I was working on a feature story for Bicycling about professional mountain biker Paul Basagoitia, who had sustained a spinal-cord injury in 2015 at Red Bull Rampage, the sports most extreme competition. At the age of 29, Paulwas paralyzed below the waist, and doctors gave him a 5percent chance of walking again. Three and a half years later, Id seen on his Instagram that he was riding an e-mountain bike. When we started talking in February, Paul told me he was releasing a film with Red Bull about his journey. The documentary, called Any One of Us, would premiere in March at the SXSW Film Festival. In February, Andrew and I watched a screener together one Friday night on the couch, my notebook in my lap, our cat curled up in his. By the end of the film, I was crying, rocked not only by Pauls storybut that of the 17 other people in the film who share their honest perspective on what it is like to live with paralysis. A few weeks after that, I went to visit Paul in Reno, Nevada. I filed adraft of my piecein April. I hoped his story would inspire people. I hoped it would remind them to be grateful for what they have, to appreciate how instantaneously it can all go away.

In July, someone driving a cargo van hit Andrew while he was riding his bike home on a country road outside Boulder, Colorado. The driver fled the scene, leaving Andrew in a ditch with over 30 broken bones (including every rib), collapsed lungs, life-threatening internal bleeding from a shattered pelvis, andthough we wouldnt know at firsta spinal-cord injury (SCI). It would paralyze his left leg and disrupt his bowel and bladder functions. In September, after two months of intensive and acute hospitalization, Andrew ended up at Craig Hospital, the same nationally renowned SCI rehab facility in Denver that had treated Paul. We would not come home until October. Any One of Us would debut on HBO two weeks later, on October 29.

The strangeness of the parallel is undeniable. At first I felt sick thinking this could be karmicthat by making Paul revisit the most traumatic moments of his life, by capitalizing (even with good intentions) on his ordeal, I had brought this upon us. A good friend of mine had a more optimistic takethat meeting Paul had prepared us. The most likely reality is neither: This was simply a coincidence. This could happen to youor me.

As the films title belies, thats one of its main messages: a spinal-cord injury couldhappen to anyone. The narrative of Any One of Us follows the fallout of Pauls injury over two years. The first halfwas actually filmed by Paul himself, who hassome videography background and had the remarkable prescience to turn the camera on himself, almost from day one. But first-time director Fernando Villena uses a unique filmmaking technique to powerful effect: introducing what Villena calls a chorusof 17 other people who share their experiences with SCI throughout the documentary. At the moment of Pauls injury, which was recorded not only by Rampages live broadcastbut also on Pauls own GoPro, members of the chorus chimein with their own stories of the incidents that left them in wheelchairs: Caught by a wave while surfing. Botched epidural. BMX crash. Car accident. Knocked over while playing basketball.

Villena and Basagoitiawanted the film to be bigger than the story of one professional athlete. The National Spinal Cord and Injury Statistical Center estimates that 17,700 people in the U.S. suffer new spinal-cord injuries every yearand 288,000 people live with anSCI. The inclusion of the chorus creates the effect of describing a collective SCI experience. The wheelchair-bound individuals speak with clarity and courage about various aspects of the injuryfrom what happened to their sex lives, to the feeling of being in the way or an inconvenience to others, to their defiance at the idea that their value as a human being has somehow changed just because theyre in a wheelchair. I hated my fiance seeing me, says Olympic BMX racer Sam Willoughby.

I wanted to die. I didnt see how I could continue living like that. Or like this, says Steph Aiello, who was injured in a car accident.

Basagoitia himself reveals, Before this injury, I always said I would probably take my life if I was ever paralyzed. Its a sentiment that, even watching the film the first time, hit home. Particularly for active people, the idea of waking up paralyzed is, to borrow Pauls word, a nightmare. Paul had been one of the best professional slopestyle and freeride mountain bikers in the world. At age 17, he entered the inaugural edition of Crankworx, now the worlds premierslopestyle competition, on a lark, riding a borrowed bike. Against the best pros in the world, he won. The next year, he won again. Scenes from his recovery, as he relearns how to do basic things like transfer into a car from his wheelchair and walk haltingly on parallel bars, are juxtaposed with scenes from his career: Paul spinning in the air, doing a 360;Paul spraying himself with champagne on the podium;Paul shirtless, hanging jubilantly out of the drivers seat of a trophy truck he won in Germany(hed sell it the next day: There was no way I was getting that thing back to the U.S., he laughed, when he told me the story in Reno).

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In the hospital, I thought of Paul as I watched my fianc struggle with stairs for the first time after his injury, leaning on a handrail and laboriously placing his left leg, stiff and heavy in its metal brace, on each step. Andrew and I are both cyclists. We rode our bikes five to seven days a week. An elite-level track and road racer, Andrew had always been one of the strongest guys on the group ride. When I was tired, I would sit in the generous draft forged by his six-foot-threeframe, and he would pull me home. In the early days in the ICU, when we knew about his injury but he was too delirious and doped up on pain medications to yet be told, he had still somehow known. One night, after his brother, Eric,and I left the hospital, he texted us, Remember, I used to be a good bike racer.

I wanted to scream.

In Reno, Pauls then girlfriend andnow fiance, Nichole Munk, had told me that she did not cry throughout the three months that Paul was in hospitals. If that sounds unbelievable to you, it wont be once you watch the film, throughout which it becomes apparent that Nichole is an extraordinary human beingand one uncannily equipped to support Paul through his ordeal. Theres a moment in particular in Any One of Us that Paul and Nichole told me got a lot of surprised reactions from people during screenings: After a visit from his father in the hospital, Paul gets emotionaland starts choking up as he talks about it. Instead of cooing over him, Nichole tells him to toughen up.

Its a jarring interactionand one that flies in the face of our preconceptions of how women are supposed to be caretakers: soft and endlessly nurturing. And yet, a few minutes later, Nicholewho started dating Paul while she was cheerleading for the University of Nevada, Renois dancing in his wheelchair, laughing, an infectious smile on her face. And hes laughing, too. I have to be positive so he can be happy, she says in a voice-over. I have to be the rock. Munk does not embody the performative sympathy we think someone in that position might want; she is the constant source of no-bullshit straight talk, relentless positivity, and unshakable love that Paul Basagoitia actually needs. A year later, as the two of them argue over whether he should try fetal-stem-cell therapy, Nichole tells him, Youre not a very pleasant person to be around right now. They offer a real, unvarnished look at what it takes to be a partner and caregiver during this kind of life-altering ordeal.

Andrews injury has become the sun around which we orbit. It has its own gravitational pull, which overpowers that of the minor planets our lives used to revolve around: our bikes, our careers, our friends, our families. After July 20, life became a sleep-deprived blur of commuting up and down a traffic-choked interstate, days and nights spent in hospitals, and, once I went back to work, struggling to reintegrate into a new job Id just started three weeks before his crash. It was, plainly put, exhausting. I could sense, on a few occasions, that when friends and family dropped in on us, they thought I could be more dotingnicer, more attentive, fussing over him more. But as Andrew has gotten more self-sufficienthe spends most of his day in a wheelchairbut can walk short distances on forearm crutcheshe doesnt need someone to hover over himor shield him from the hard emotional work of making the best of this shitty situation. He needs someone to scrape the snow off his car early in the morning so he can get to PT, to carry the bags back in from the grocery store, and to call him out or make him smile or do both at the same time so he can self-arrest during the inevitable slides into darkness. He doesnt need a nurse. He needs a partner.

Since Andrews injury, I have thought of Nichole Munk often. In my mind, shes an example of what it means to be the support someone needs, not just the support everyone expects you to be. We should all hope to have and be the kind of partner she is.

At its core, this movie is an instructional. But for whom? The genre of disability stories too easily falls into the trap of inspiration porn for an able-bodied audience. Butfrom the first clips Paul took in the hospital, Any One of Us didnt begin as a documentary for the rest of us. In a way, it often feels like a conversation between people living with an SCI. We are allowed in to listen, watch, and learn.

When Basagoitia started filming himself, he told me, he didnt know what he was going to do with the footage. He just had the sense that his experience might one day help others going through the same thing. As early as the day he got his staples out from his back surgery, a couple of weeks after his crash, he said into the camera, I just hope this can be a guide for someone else. One of the most challenging aspects of a spinal-cord injury is uncertainty. The delicate bundle of nerve fibers that carries messages from your brain to the rest of your body, and which alsoregulates sensory, motor, and autonomic function, is highly susceptible to damageand heals slowly once injured, if at all. Mostrecovery takes place within 18 months to two years, but the neurological recovery of every person is different, and it is impossible to know what youll regain.

When Paul began collaborating with Villena and Red Bull, the intentions for the film became twofoldto be a field manual for people living with an SCIand to raise awareness about their experience. Awareness leads to interest, which leads to funding for a curewhich doesnt currently exist for spinal-cord injuries, though some experimental therapies have shown promise. Until now, awareness has been critically lacking, Basagoitia told me in Reno. Since the late actor Christopher Reeve, who suffered an SCI while horseback riding, passed in 2004, the popular media has largely overlooked it. Any One of Us was just one of two feature-length documentaries I could find online about the topic. (The other was Coming to My Senses, released in 2018.)

Basagoitia holds nothing back in educating viewers about the reality of an SCI. One of the rawest scenes of the film shows Paul nude in front of the hospital toilet in the middle of the night, inserting a catheter to urinate. It takes him over seven minutes. There is footage of Nichole helping him bathe. Its a searingly honest look into what people with an SCI endure on a daily basis. But it must also be said that both Paul and Andrew were relatively fortunate; both were paralyzed below the waist, with incomplete injuriestheir cords were not totally severed. Those with complete injuries face a life of paralysis with little hope of significant recovery. At Craig Hospital, Andrew and I also interacted with quadriplegic patients, some of whom were learning to control powered wheelchairs using their breath. In one scene, after he arrives at Craig, Paul tells the camera somberly, Theres not one person here at the hospital who isnt in a wheelchair.In fact, theres a lot of people who are in worse situations.

As the film follows Paul through his recovery, the viewer comes to relate with the experience of finding triumph in the smallest victoriesa feeling Andrew and I have come to know well. Hewould text me to tell me a new muscle strand had started twitchingor that he had stood for a full 30 seconds on his own. But there are big wins, too. Villena selected an impressive group to represent the chorus: author Annette Ross, who wrote a memoir after her injury; Chelsie Hill, who started a wheelchair dance team; Jesse Billauer, who became the adaptive world champion ofsurfing in 2015. As for Paul, he makes his way back onto the bike about a year after his injury. His first ride is captured on film, and his pure joy is palpable.

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These moments, which are paired with a stirring but not overly melodramatic soundtrack, are emotional, but its more than voyeuristic inspiration. Its a shared sense of triumph thats earned after bearing witness to the hardships of this injury. And for anyone living with an SCI, the scenes of those in the chorus walking, playing basketball, dancing, and riding a bike againare a powerful promise that your best days can still lie before you.

Perhaps that is one of the greatest accomplishments of this film, that it manages to speak to both audiences. Andrew and I know this because weve seen the film twice. The first time, we were deeply affected by the experience and determination of this community. We learned that, with resolve, life in a wheelchair could in fact be richer and more beautiful than life before it. We saw that its a defect, a bug coded into our human nature, that we never know how to truly appreciate what we have until we lose it.

The second time we watched the film, two weeks before it aired on HBO and three months after our own SCI journey began, we were searching for the common threads between our story and those of others who had been through it before. This timewe were hunting for clues that would show us the way forward. We cried when we saw them over and over.

Paul Basagoitia wanted to be a guide for how to beat a spinal-cord injury. With Any One of Us, he ended up creating a guide for how to live. This film is more than an inspirational story. It is a testimony. It is a map through hell. It is a gift of hope.

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'Any One of Us' Is More Than an Inspirational Film - Outside

Parents of Thalassemia-affected siblings seek Kochiites assistance – The New Indian Express

By Express News Service

KOCHI:Every parent prays for the well-being of their children. While the prayers of many are answered, there are some who are not so lucky. Yet, they do not lose hope and fight for their childrens lives.Mubarak and Nisha are one such couple which is fighting for the lives of not one but all three of their children.

Faizy, our six-year-old son, was diagnosed with Thalassemia Major, a blood disorder, when he was three-months-old, said Nisha. The couple had been struggling to come to terms with the news when they were dealt another blow.

We neither knew nor expected that Faiha, our five-year-old daughter, would also be diagnosed with the same disorder. She was diagnosed with Thalassemia Major when she was just nine-months-old, she said.For the past six years, the children have been undergoing blood transfusions every 14 days. Our lives revolve around the transfusion dates and the struggle to find the money for the procedures, she said. Her husband, Mubarak, is a driver. He drives a waste collection truck. However, the regular hospital visits mean he has to skip work on many occasions, Nisha said.

Tragedy struck the family again when their third child, a boy, was also diagnosed with the same disorder when he turned one.

The doctors said only a stem cell transplant could save my kids. We got ourselves tested, but I was ruled out and my husbands stem cell was a partial match, she said.She said the doctors have warned against performing surgery with a partially-matched stem cell as it has its dangers. So, we are seeking donors. Everyday is painful for my children. Faizy alone has undergone 100 transfusions till date, she said.

Ramiz Rehman of Smilemakers, Cusat, said time was limited for Faizy. We need a donor as soon as possible. Else, the family will have to use the fathers stem cell. The procedure has to be done in January, said Ramiz.

How to helpTo help the children, Cusats Smilemakers, in association with the Department of Sociology and the NSS unit of St Teresas College as well as Datri (Blood stem cell donors registry), are conducting a blood stem cell donor registration drive on Tuesday at the college.Aby Sam, the state head of Datri, said one registration drive each were held in Changanassery and Mattancherry.

The drive at Mattancherry saw the highest participation with over 3,600 people registering as donors, he said. People believe that blood is checked to identify donors and compatibility. It is not true. A cheek swab is done for the purpose, said Aby.We only examine and register people aged between 18 and 50 years as donors, said Aby, who was the first person in the state to register as a stem cell donor.

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Parents of Thalassemia-affected siblings seek Kochiites assistance - The New Indian Express

Induced Pluripotent Stem Cell Market is expected to witness a strong CAGR of 7.0% from 2018 to 2026 – Zebvo

The healthcare industry has been focusing on excessive research and development in the last couple of decades to ensure that the need to address issues related to the availability of drugs and treatments for certain chronic diseases is effectively met.

Healthcare researchers and scientists at the Li Ka Shing Faculty of Medicine of the Hong Kong University have successfully demonstrated the utilization of human induced pluripotent stem cells or hiPSCs from the skin cells of the patient for testing therapeutic drugs.

The success of this research suggests that scientists have crossed one more hurdle towards using stem cells in precision medicine for the treatment of patients suffering from sporadic hereditary diseases. iPSCs are the new generation approach towards the prevention and treatment of diseases that takes into account patients on an individual basis considering their genetic makeup, lifestyle, and environment. Along with the capacity to transform into different body cell types and same genetic composition of the donors, hiPSCs have surfaced as a promising cell source to screen and test drugs.

In the present research, hiPSC was synthesized from patients suffering from a rare form of hereditary cardiomyopathy owing to the mutations in Lamin A/C related cardiomyopathy in their distinct families. The affected individuals suffer from sudden death, stroke, and heart failure at a very young age. As on date, there is no exact treatment available for this condition. This team in Hong Kong tested a drug named PTC124 to suppress specific genetic mutations in other genetic diseases into the iPSC transformed heart muscle cells. While this technology is being considered as a breakthrough in clinical stem cell research, the team at Hong Kong University is collaborating with drug companies regarding its clinical application.

The unique properties of iPS cells provides extensive potential to several biopharmaceutical applications. iPSCs are also used in toxicology testing, high throughput, disease modeling, and target identification.

This type of stem cell has the potential to transform drug discovery by offering physiologically relevant cells for tool discovery, compound identification, and target validation. A new report by Persistence Market Research (PMR) states that the globalinduced pluripotent stem or iPS cell marketis expected to witness a strong CAGR of 7.0% from 2018 to 2026. In 2017, the market was worth US$ 1,254.0 Mn and is expected to reach US$ 2,299.5 Mn by the end of the forecast period in 2026.

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Customization to be the Key Focus of Market Players

Due to the evolving needs of the research community, the demand for specialized cell lines have increased to a certain point where most vendors offering these products cannot depend solely on sales from catalog products.

The quality of the products and lead time can determine the choices while requesting custom solutions at the same time. Companies usually focus on establishing a strong distribution network for enabling products to reach customers from the manufacturing units in a short time period.

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Entry of Multiple Small Players to be Witnessed in the Coming Years

Several leading players have their presence in the global market; however, many specialized products and services are provided by small and regional vendors. By targeting their marketing strategies towards research institutes and small biotechnology companies, these new players have swiftly established their presence in the market.

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Induced Pluripotent Stem Cell Market is expected to witness a strong CAGR of 7.0% from 2018 to 2026 - Zebvo