MIRAMAR, FL, Sept. 19, 2022 (GLOBE NEWSWIRE) -- Stemtech Corporation (Stemtech) (OTCQB: STEK), an innovative nutraceutical company and a pioneer in the field of stem cell nutrition, today announced creation of new stem cell skincare products.

Stemtech Corporation President and Chief Operating Officer, John W. Meyer, said the introduction of the stem cell skincare line is very exciting for us to offer our Independent Business Partners (IBPs) and customers. The product will be introduced at the upcoming Cancun, Mexico Incentive Meeting in December. Meyer continues to say that Stemtechs belief in highly efficacious and quality products expands into a very important area for youthful and healthy skin care. The skincare, along with our stemceutical products of RCM - stemrelease3, StemFlo Advanced and MigraStem, is a winning combination for inner health and outer radiance - fantastic! We all want to feel better, look better. By using Stemtech products, you can!

Fortune Business Insights in August 2021 published that the global skincare market is projected to grow from $100.13 billion in 2021 to $145.82 billion in 2028 at a CAGR of 5.52% in forecast period, 2021-2028. Meyer continues, noting while it may seem that there are many companies already in this space, Stemtech feels confident with our new science being developed, we will have a significant impact on the skincare market using our stem cell scientific knowledge.

Charles S. Arnold, Stemtech Corporation Chairman and CEO stated that the introduction of skincare focuses new Stemtech stem cell technology on skincare for healthier skin, the largest organ of the body, which we recognize as a key to anti-aging. Our excitement at being able to launch new products, complete with samples, for a more youthful and healthy skin will catapult our Field to new growth and revenue opportunities. We know our IBPs will be exuberant with this great new Stemtech Skincare product line.

About Stemtech Corporation

Stemtech Corporation, a leading nutraceutical company with a direct sales distribution model, was founded on April 18, 2018, after acquiring the operations from its predecessor Stemtech International, Inc. which was founded in 2005. From 2010 through 2015, Stemtech International, Inc., was recognized four separate times on the Inc. 5000 Fastest-Growing Companies list. In 2018, the Company underwent an extensive executive reorganization, and continued operations under new leadership. Stemtech specializes in creating products and formulas that are patent protected in the U.S. and in select international markets. The Companys patented formulas help the release, circulation and migration of the bodys adult stem cells from its bone marrow. The Company markets its products under the following brands: RCM System, stemrelease3, Stemflo MigraStem, OraStem (Oral Health Care), and D-Fuze (EMF blocker). Its nutraceutical products are all-natural, plant-based and manufactured under cGMP (Current Good Manufacturing Practices) under the auspices of the Dietary Supplement Health and Education Act (DSHEA). For more information, please visit http://www.stemtech.com.

Forward-Looking Statements

This announcement contains forward-looking statements within the meaning of the safe harbor provisions of the U.S. Private Securities Litigation Reform Act of 1995. Such statements include but are not limited to statements identified by words such as "believes," "expects," "anticipates," "estimates," "intends," "plans," "targets," "projects" and similar expressions. The statements in this release are based upon the current beliefs and expectations of our company's management and are subject to significant risks and uncertainties. Actual results may differ from those set forth in the forward-looking statements. Numerous factors could cause or contribute to such differences, including, but not limited to, results of clinical trials and/or other studies, the challenges inherent in new product development initiatives, the effect of any competitive products, our ability to license and protect our intellectual property, our ability to raise additional capital in the future that is necessary to maintain our business, changes in government policy and/or regulation, potential litigation by or against us, any governmental review of our products or practices, as well as other risks discussed from time to time in our filings with the Securities and Exchange Commission, including, without limitation, our latest 10-Q Report filed onAugust 23, 2022. We undertake no duty to update any forward-looking statement, or any information contained in this press release or in other public disclosures at any time. Finally, the investing public is reminded that the only announcements or information about Stemtech Corporation which are condoned by the Company must emanate from the Company itself and bear our name as its Source.

http://www.Stemtech.com

Investor Relations: Investor Relations: 954-715-6000 ext 1040invrel@stemtech.com

Originally posted here:
STEMTECH CORPORATION ANNOUNCES THE INTRODUCTION OF A NEW - GlobeNewswire

Recommendation and review posted by Bethany Smith

Five people with the autoimmune condition lupus are now in remission after receiving a version of CAR-T therapy, which was originally developed for cancer

By Clare Wilson

Illustration of a CAR-T cell

CHRISTOPH BURGSTEDT/SCIENCE PHOTO LIBRARY

A high-tech cell therapy used to treat cancer has been repurposed as a treatment for lupus, an autoimmune condition that can cause joint, kidney and heart damage.

CAR T-cell therapy has put all five people with lupus treated so far into remission. The participants have been followed up for an average of 8 months, with the first person treated 17 months ago. Thats kind of unheard of, says Chris Wincup at Kings College London, who wasnt involved in the study. This is incredibly exciting.

But it is too soon to know how long the remissions will last, says Georg Schett at the University of Erlangen-Nuremberg in Germany, who was part of the study team.

CAR T-cells were developed to treat blood cancers that arise when B cells, a type of immune cell that normally makes antibodies, start multiplying out of control.

The approach requires taking a sample of immune cells from a persons blood, genetically altering them in the lab so they attack B cells and then infusing them back into the individuals blood. It seems to put 4 out of 10 people with these kinds of cancers into remission.

Lupus, also called systemic lupus erythematosus, is caused by the immune system mistakenly reacting against peoples own DNA. This is driven by B cells making antibodies against DNA released from dying cells.

It is currently treated with medicines that suppress the immune system or, in more severe cases, with drugs that kill B cells. But the treatments cant kill all the B cells, and if the disease flares up badly, some people develop kidney failure and inflammation of their heart and brain.

Schett and his team wondered whether using CAR T-cells to hunt down all the B cells would be more effective. Within three months of receiving the treatment, all five participants were in remission, without needing to take any other medicines to control their symptoms.

The CAR T-cells were barely detectable after one month, and after three and a half months, the volunteers B cells started to return, having been produced by stem cells in bone marrow. These new B cells didnt react against the DNA.

We dont know what normally causes B cells to start reacting against DNA in people with lupus, so it is possible that some kind of trigger may start the process happening again, says Wincup.

The achievement means CAR T-cells may also be useful against other autoimmune diseases that are driven by antibodies, such as multiple sclerosis (MS), in which the immune system attacks nerves, says Schett.

Another radical treatment for MS involves rebooting the immune system by destroying it with chemotherapy. By comparison, CAR T-cells would be less invasive and more tolerable, he says.

But it is too soon to know how effective CAR T-cells will be for autoimmune conditions, says Wincup. This is a small number of patients, so we dont know if this is going to be the result for everyone.

When used in cancer, CAR T-cells are expensive to create for each person, so they may only be used for autoimmune conditions in people with severe disease when no other treatments are available, he says.

Journal reference: Nature Medicine , DOI: 10.1038/s41591-022-02017-5

More on these topics:

Read the original post:
Radical lupus treatment uses CAR T-cell therapy developed for cancer - New Scientist

Recommendation and review posted by Bethany Smith

Esteemed award is an international forum celebrating noteworthy achievements in equine research and individuals who have significantly impacted equine health

The University of Kentucky (UK) Gluck Equine Research Center unveiled the 2022 inductees to the Equine Research Hall of Fame. The winners include Lisa Fortier, DVM, PhD, DACVS; Katrin Hinrichs, DVM, PhD; Jennifer Anne Mumford, DVM; and Stephen M. Reed, DVM.

The scientists were nominated by their fellow peers and past awardees. Nominees may be living or deceased, active in or retired from the field of equine research.

In research, we always stand on the shoulders of those who go before us with great discoveries. This years recipients have made substantial contributions that will ensure an excellent future for equine research, expressed Nancy Cox, UK vice president for land-grant engagement and College of Agriculture, Food and Environment dean, in a university release.1

The success of Kentuckys horse industry is inseparable from the decades of hard work by outstanding equine researchers, added Stuart Brown, chair of the Gluck Equine Research Foundation. Though impossible to measure, it is a unique privilege to recognize the impact made by these four scientists in advancing the health and wellbeing of the horse and, on behalf of the entire equine community, show our appreciation.

Below are the details of each awardee1:

Throughout the past 30 years, Fortier has been renowned for her substantial contributions in equine joint disease, cartilage biology, and regenerative medicine. Her research focuses on early diagnosis and treatment of equine orthopedic injuries to prevent permanent damage to joints and tendons. She is most well-known for her work in regenerative medicine, spearheading the use of biologics such as platelet rich plasma, bone marrow concentrate, and stem cells for use in horses and humans. Additionally, Fortiers lab has been key in strides associated with cartilage damage diagnosis and clinical orthopedic work.

Fortier achieved her bachelors degree and doctor of veterinary medicine degree from Colorado State University. She finished her residency at Cornell, where she also earned a PhD and was a postdoctoral fellow in pharmacology. Currently, she serves as the James Law Professor of Surgery at Cornells College of Veterinary Medicine. She is the editor-in-chief of the Journal of the American Veterinary Medical Association and serves on the Horseracing Integrity and Safety Authority Racetrack Safety Standing Committee.

Hinrichs dedicates her career to research mainly in equine reproductive physiology and assisted reproduction techniques. Her focus has consisted of equine endocrinology, oocyte maturation, fertilization, sperm capacitation, and their application to assisted reproduction techniques.

Her 40 years of research have resulted in various notable basic and applied research accomplishments. The applied achievements include generating the first cloned horse in North America and creating the medical standard for effective intracytoplasmic sperm injection and in vitro culture for equine embryo production. She has mentored over 85 veterinary students, residents, graduate students, and postdoctoral fellows in basic and applied veterinary research. Her laboratories have hosted about 50 visiting scholars worldwide.

Hinrichs achieved her bachelors degree and doctor of veterinary medicine degree from the University of California, Davis. She finished residency training in large animal reproduction at the University of Pennsylvanias New Bolton Center and received a PhD at the University of Pennsylvania.

Mumford is a posthumous inductee who received international respect as among the most prominent researchers of equine infectious diseases, specifically equine viral diseases. Her career at the Animal Health Trust, Newmarket, United Kingdom, began when she was deemed the first head of the newly established equine virology unit. Her work focused on the leading causes of acute infectious respiratory disease in the horse, mainly equine herpesvirus and equine influenza virus, and to a lesser extent,Streptococcus equi.

Mumford impacted several of these realms, including developing enhanced vaccines, diagnostics, and international surveillance. Additionally, she helped create research groups in the related fields of equine genetics and immunology.

Throughout Mumfords over 30 year-career, she helped the Animal Health Trust be recognized as one of the worlds leading centers for the study of the biology, epidemiology, immunology and pathology of diseases.

Reeds nominators deemed his as the last word in equine neurology. He is known as among the most prominent equine neurologists worldwide. His list of 180 peer-reviewed publications feature important contributions to equine medicine, neurology, physiology and pathophysiology. He has shared in his accomplishments as a mentor and role-model for hundreds of aspiring equine practitioners.

Reed received his bachelors degree and doctor of veterinary medicine degree from The Ohio State University. He finished his internship and residency training in large animal medicine at Michigan State University.

The UK Gluck Equine Research Foundation will induct the 4 winners into the UK Equine Research Hall of Fame October 26, 2022 at Kroger Field in Lexington, Kentucky.

Reference

Wiemers H. UK Equine Research Hall of Fame inductees announced. UK College of Agriculture, Food and Environment. News release. September 13, 2022. Accessed September 20, 2022. https://news.ca.uky.edu/article/uk-equine-research-hall-fame-inductees-announced-1

Read this article:
University of Kentucky Equine Research Hall of Fame announces awardees - DVM 360

Recommendation and review posted by Bethany Smith

Background: Male hypogonadism is a clinical and biochemical androgen insufficiency syndrome, becoming more prevalent with age. Exogenous testosterone is first-choice therapy, with several side effects, including negative feedback of the hypothalamic-pituitary-gonadal axis, resulting in suppression of intratesticular testosterone production and spermatogenesis. To preserve these testicular functions while treating male hypogonadism, clomiphene citrate is used as off-label therapy. This systematic review and meta-analysis aimed to evaluate the effectiveness and safety of clomiphene citrate therapy for men with hypogonadism.

Methods: The EMBASE, PubMed, Cochrane databases were searched in May 2021, for effectiveness studies of men with hypogonadism treated with clomiphene citrate. Both intervention and observational studies were included. The Effective Public Health Practice Project Quality Assessment Tool, a validated instrument, was used to assess methodological study quality. The primary outcome measure was the evaluation of serum hormone concentration. Secondary outcomes were symptoms of hypogonadism, metabolic and lipid profile, side effects, safety aspects.

Results: We included 19 studies, comprising four randomized controlled trials and 15 observational studies, resulting in 1642 patients. Seventeen studies were included in the meta-analysis, with a total of 1279 patients. Therapy and follow-up duration varied between one and a half and 52 months. Total testosterone increased with 2.60 (95% CI 1.82-3.38) during clomiphene citrate treatment. An increase was also seen in free testosterone, luteinizing hormone, follicle stimulating hormone, sex hormone-binding globulin and estradiol. Different symptom scoring methods were used in the included studies. The most frequently used instrument was the Androgen Deficiency in Aging Males questionnaire, whose improved during treatment. Reported side effects were only prevalent in less than 10% of the study populations and no serious adverse events were reported.

Conclusion: Clomiphene citrate is an effective therapy for improving both biochemical as well as clinical symptoms of males suffering from hypogonadism. Clomiphene citrate has few reported side effects and good safety aspects.

Keywords: clomiphene citrate; male hypogonadism; testosterone deficiency.

The rest is here:
Clomiphene citrate for men with hypogonadism: a systematic ... - PubMed

Recommendation and review posted by Bethany Smith

What is hypogonadism in females?

Hypogonadism in females describes the inadequate function of the ovaries, with impaired production of germ cells (eggs) and sex hormones (oestrogen and progesterone).

Hypogonadism in females is due to disruption of any section of the hypothalamicpituitaryovarian axis pathway (figure 1). In a correctly functioning hypothalamicpituitaryovarian axis pathway:

Figure 1. The hypothalamicpituitaryovarian axis pathway

Primary ovarian insufficiency and secondary hypogonadism may be congenital or acquired [1,2].

The main mechanism for congenital primary ovarian deficiency remains unknown in the majority of cases. Some cases relate to:

The causes of acquired primary ovarian insufficiency include:

Congenital secondary hypogonadism is gonadotrophin deficiency due to a genetic mutation, such as in Kallmann syndrome.

Acquired secondary hypogonadism can be due to damage to the pituitary/hypothalamus. Causes of acquired secondary hypogonadism can include:

Gonadotropins can be suppressed by:

The clinical features of hypogonadism depend on the age at presentation [3].

Symptoms of low oestrogen levels are rarely present in hypogonadism pre-puberty. The presenting features are absent pubertal development (reduced growth and absence of pubic hair) and primary amenorrhoea (absence of menarche).

After the completion of puberty, the features of hypogonadism include:

The long-term risks of oestrogen deficiency include an increased risk of osteoporosis and cardiovascular disease. The risk is greater with a younger age of onset. In contrast, the risk of breast cancer may be slightly reduced.

Oestrogen has a key role in maintaining skin health. Oestrogen helps maintain skin thickness and collagen levels, skin elasticity, and moisture. It is also thought to play a role in wound healing [4].

Low oestrogen levels are associated with:

Skin changes may also reflect the underlying cause of hypogonadism; for example, hyperpigmentation may be a sign of an autoimmune disease.

If hypogonadism is suspected following a detailed history and examination, the following investigation pathway can be followed.

Treatment of hypogonadism is directed at the underlying pathology where possible, helping the woman become fertile if desired, and preventing the long-term complications of hypoestrogenism (ie, osteoporosis, increased cardiovascular disease, and urogenital atrophy).

As a general rule, women of reproductive age with hypoestrogenism should receive hormone replacement therapy. Specialist input should be sought, as there are potential significant complications of hormone therapy, such as:

Post-menopause, hormone replacement therapy is indicated for significant symptoms.

See the original post:
Hypogonadism in females | DermNet

Recommendation and review posted by Bethany Smith

Aim: Symptomatic late-onset hypogonadism is associated not only with a decline in serum testosterone, but also with a rise in serum estradiol. These endocrine changes negatively affect libido, sexual function, mood, behavior, lean body mass, and bone density. Currently, the most common treatment is exogenous testosterone therapy. This treatment can be associated with skin irritation, gynecomastia, nipple tenderness, testicular atrophy, and decline in sperm counts. In this study we investigated the efficacy of clomiphene citrate in the treatment of hypogonadism with the objectives of raising endogenous serum testosterone (T) and improving the testosterone/estrogen (T/E) ratio.

Methods: Our cohort consisted of 36 Caucasian men with hypogonadism defined as serum testosterone level less than 300 ng/dL. Each patient was treated with a daily dose of 25 mg clomiphene citrate and followed prospectively. Analysis of baseline and follow-up serum levels of testosterone and estradiol levels were performed.

Results: The mean age was 39 years, and the mean pretreatment testosterone and estrogen levels were 247.6 +/- 39.8 ng/dL and 32.3 +/- 10.9, respectively. By the first follow-up visit (4-6 weeks), the mean testosterone level rose to 610.0 +/- 178.6 ng/dL (P < 0.00001). Moreover, the T/E ratio improved from 8.7 to 14.2 (P < 0.001). There were no side effects reported by the patients.

Conclusions: Low dose clomiphene citrate is effective in elevating serum testosterone levels and improving the testosterone/estradiol ratio in men with hypogonadism. This therapy represents an alternative to testosterone therapy by stimulating the endogenous androgen production pathway.

See the original post here:
Clomiphene citrate effects on testosterone/estrogen ratio in male ...

Recommendation and review posted by Bethany Smith

NEW YORK, Sept. 19, 2022 /PRNewswire/ --The male hypogonadism market has been segmented by type (Klinefelter syndrome, Kallmann syndrome, and pituitary disorders) and geography (North America, Europe, Asia, and Rest of World (ROW)). North America will account for 37% of the market's growth during the forecast period. This growth is attributed to factors such as the significant increase in the prevalence of male hypogonadism. Moreover, market growth in this region will be faster than the growth of the market in other regions. The US and Canada are the key countries for the male hypogonadism market in North America.

Themale hypogonadism market size is estimated to grow by USD 684.95 mn from 2021 to 2026. In addition, the growth momentum of the market will accelerate at a CAGR of 5.09% during the forecast period.

Technavio provides a comprehensive report summary describing the market size and forecast along with research methodology. The FREE sample reportis available in PDF format

Company Profiles

The male hypogonadism market report includes information on the product launches, sustainability, and prospects of leading vendors, including AbbVie Inc., Aytu BioPharma Inc., Bayer AG, Bio Techne Corp., Diurnal Group Plc, Eli Lilly and Co., Endo International Plc, Ferring B.V., IBSA Institute Biochimique SA, Lipocine Inc., Merck and Co. Inc., Perrigo Co. Plc, Pfizer Inc., and Teva Pharmaceutical Industries Ltd. The key offerings of some of these vendors are listed below:

Technavio's reports provide key strategic initiatives used by vendors, along with key news and the latest developments. View our FREE PDF Sample Report Now

Competitive Analysis

The competitive scenario provided in the male hypogonadism market report analyzes, evaluates, and positions companies based on various performance indicators. Some of the factors considered for this analysis include the financial performance of companies over the past few years, growth strategies, product innovations, new product launches, investments, growth in market share, etc.

Market Dynamics

Factors such as an increase in the incidence of hypogonadism and the increasing awareness about male hypogonadism and its treatment options will be crucial in driving the growth of the market. However, the loss of patent exclusivities and increasing generic competition will restrict the market growth.

Technavio has identified key trends, drivers, and challenges in the market, which will help vendors improve their strategies to stay ahead of their competitors. View our FREE PDF Sample Report

Related Reports

Sexually Transmitted Diseases (STD) Treatment Market by Service and Geography - Forecast and Analysis 2022-2026: The sexually transmitted diseases (STD) treatment market share is expected to increase by USD 13.48 billion from 2021 to 2026.

Sexual Enhancement Supplements Market by Product and Geography - Forecast and Analysis 2022-2026: The sexual enhancement supplements market share is expected to increase by USD 801.29 million from 2021 to 2026.

Male Hypogonadism Market Scope

Report Coverage

Details

Page number

120

Base year

2021

Forecast period

2022-2026

Growth momentum & CAGR

Accelerate at a CAGR of 5.09%

Market growth 2022-2026

USD 684.95 million

Market structure

Fragmented

YoY growth (%)

3.52

Regional analysis

North America, Europe, Asia, and Rest of World (ROW)

Performing market contribution

North America at 37%

Key consumer countries

US, Canada, Germany, UK, and China

Competitive landscape

Leading companies, competitive strategies, consumer engagement scope

Companies profiled

AbbVie Inc., Aytu BioPharma Inc., Bayer AG, Bio Techne Corp., Diurnal Group Plc, Eli Lilly and Co., Endo International Plc, Ferring B.V., IBSA Institute Biochimique SA, Lipocine Inc., Merck and Co. Inc., Perrigo Co. Plc, Pfizer Inc., and Teva Pharmaceutical Industries Ltd.

Market Dynamics

Parent market analysis, market growth inducers and obstacles, fast-growing and slow-growing segment analysis, COVID-19 impact and future consumer dynamics, and market condition analysis for the forecast period.

Customization purview

If our report has not included the data that you are looking for, you can reach out to our analysts and get segments customized.

Browse Health CareMarket Reports

Table of Contents

1 Executive Summary

2 Market Landscape

3 Market Sizing

4 Five Forces Analysis

5 Market Segmentation by Type

6 Customer Landscape

7 Geographic Landscape

8 Drivers, Challenges, and Trends

9 Vendor Landscape

10 Vendor Analysis

11 Appendix

About Us

Technavio is a leading global technology research and advisory company. Their research and analysis focus on emerging market trends and provide actionable insights to help businesses identify market opportunities and develop effective strategies to optimize their market positions. With over 500 specialized analysts, Technavio's report library consists of more than 17,000 reports and counting, covering 800 technologies, spanning across 50 countries. Their client base consists of enterprises of all sizes, including more than 100 Fortune 500 companies. This growing client base relies on Technavio's comprehensive coverage, extensive research, and actionable market insights to identify opportunities in existing and potential markets and assess their competitive positions within changing market scenarios.

Contact

Technavio Research

Jesse Maida

Media & Marketing Executive

US: +1 844 364 1100

UK: +44 203 893 3200

Email: media@technavio.com

Website: http://www.technavio.com/

View original content to download multimedia:https://www.prnewswire.com/news-releases/male-hypogonadism-market-size-to-grow-by-usd-684-95-mn-abbvie-inc-and-bayer-ag-among-key-vendors---technavio-301626163.html

SOURCE Technavio

Read the original here:
Male Hypogonadism Market Size to Grow by USD 684.95 Mn, AbbVie Inc. and Bayer AG Among Key Vendors - Technavio - Longview News-Journal

Recommendation and review posted by Bethany Smith

With the intensified direct-to-consumer marketing of male sexual medicine treatments, the recent legislative changes in reproductive rights and their unknown long-term effect on assisted reproduction availability for infertile men, and the explosion of telehealth, the practice of male sexual medicine is evolving at a breakneck pace. Specialists in male sexual and reproductive medicine have been tasked with digesting the evolving literature and forming evidence-based treatment guidelines for men with erectile dysfunction, Peyronie disease, infertility, and a host of other conditions. Compared with other areas of urology and medicine in general, male sexual and reproductive medicine has a disappointingly small number of well-designed prospective studies, along with a significant gap in funding for male reproductive health compared with female reproductive health. Several manuscripts published in 2022 started to narrow this gap and provide valuable level 1 evidence supporting (or discounting) key areas within sexual medicine and infertility.

For men with severe male factor infertility and nonobstructive azoospermia, surgical intervention is often indicated to retrieve sperm. Testicular sperm aspiration (TESA) and microdissection testicular sperm extraction (mTESE) are 2 commonly used approaches. A recent study by Jensen et al compared the efficacy of these 2 approaches in one of the few prospective randomized-controlled trials in male infertility.1 In the study, 49 patients were randomly assigned to mTESE with a sperm retrieval rate of 43%, and 51 patients were randomly assigned to TESA with a sperm retrieval rate of 22%. Men with failed TESA then went on to salvage mTESE with a combined sperm retrieval rate of 29%. Participants in the mTESE arm, however, had decreased postoperative testosterone levels, and 24% of participants experienced de novo hypogonadism at 6 months. Prior literature has suggested the testosterone drop is transient and that it will likely recover by 12 months. In summary, the study results showed that mTESE remains the gold standard for treatment of nonobstructive azoospermia, but patients should be counseled on the risk of de novo hypogonadism.

Despite this, mTESE success rates remain modest and are subject to the expertise and skill level of the laboratory and andrologist processing the tissue. Multiple hours can be spent trying to find the few viable sperm hidden among a sea of distractors. A recent study by Lee et al examined the power of artificial intelligence to detect human sperm in semen and mTESE samples using bright-field microscopy for nonobstructive azoospermic (NOA) patients.2 They first trained the program to identify sperm from semen samples of fertile patients. After validating the effectiveness of their algorithm, they retrained it to identify sperm in tissue from NOA patients that had been spiked with large amounts of sperm. When testing it on samples containing 3000 to 6000 sperm among other cell types, they achieved 84.0% positive predictive value and 72.7% sensitivity. Finally, without retraining their algorithm, they tested it on samples containing 10 to 200 sperm, replicating the rare sperm phenomenon seen in patients with NOA. Their model was able to detect 2969 sperm cells out of a total 3517 with an 84.4% PPV and 86.1% sensitivity. The clinical applications of artificial intelligence and machine learning in medicine continue to expand and have made their way to male infertility. Although this is not ready for immediate clinical use, it does highlight the need for further work to harness the power of technology to improve workflow of andrologists and in turn increase the success of infertility care for patients.

There has been a rapid rise in the need for male sexual health and reproductive specialists as the population ages and the number of comorbidities rise, although certain disease processes that fall within this specialty may be able to be addressed by a general urologist. In an analysis of the current educational landscape, Asanad et al call attention to the need for a structured educational curriculum in residency for male infertility.3 In a survey of urology residents, 54 of 72 respondents (75%) reported that male infertility comprises less than 10% of their training. Compared with residents who did not learn from infertility-trained faculty, residents who were exposed to infertility-trained faculty were 14.4 times more likely to feel confident performing infertility procedures (P < .001) and were more likely to feel confident performing fertility procedures after residency (P = .001).3 For trainees, their career depends on what they are exposed to. Smaller subdisciplines within urology may be more difficult to teach uniformly, and perhaps there are ways to improve the exposure to these areas for motivated residents (eg, visiting other programs).

Within male sexual health, one disease process that all urologists should be able to diagnose and initially manage is erectile dysfunction (ED). With studies citing the prevalence of ED as high as 52%, the demand for providers to manage ED remains sky high. Current treatment options include phosphodiesterase type 5 inhibitors (PDE5is), intracavernosal injections, vacuum erection devices, and penile prosthesis. A newcomer to the field is shock wave therapy, which uses controlled energy to induce angiogenesis.

The short-term effectiveness of focused shock wave therapy for patients with moderate ED was investigated in a double-blind, randomized, sham-controlled trial.4 In this study of 70 patients with moderate ED, 35 were randomly assigned to low-intensity shock wave therapy (LiST) and the other 35 were randomly assigned to sham therapy. After a 4 week washout from PDE5i, patients underwent LiST or sham twice weekly for 6 weeks. One month after treatment completion, 59% patients in the LiST group experienced an International Index of Erectile Function (IIEF) score improvement of at least 5 points, compared with 1 patient (2.9%) in the sham group (P < .001). This effect remained present at 3 months post treatment. Thus, the short-term data for LiST are compelling and suggest this may be a viable option in the management of vasculogenic ED for men with mild/moderate ED. Further studies are desperately needed to validate these findings, and urologists have an obligation to provide patients with an honest assessment of the data and only recommend treatments where the risks (including the financial burden) are outweighed by the benefits.

In stark contrast to focused therapy, radial shock wave therapy uses low-pressure radial shock waves to treat ED. In order to characterize its effectiveness, a randomized, double-blind, sham-controlled clinical trial enrolled 80 men with mild to moderate ED.5 Patients were treated weekly with either radial wave therapy or sham therapy for 6 weeks, and the primary outcome measured was change in the IIEF score between baseline and after treatment. Study results showed that there was no significant difference in IIEF scores between groups at 6 weeks or 10 weeks after randomization. Study results displayed the lack of evidence to support the use of radial wave therapy.

Despite the evidence of their ineffectiveness in managing ED, shock wave therapy and particularly radial wave therapy have been heavily marketed directly to consumers in the US. A recent article using a secret-shopper method found troubling marketing and practice trends in the US. The authors noted that patients often are not adequately educated on the different types of treatments and may not know if the administrator is a licensed medical professional.6 With the average cost of treatment ranging from $2600 to $3900 per cycle, clinics offering radial wave therapy have an obvious financial incentive to continue marketing despite the lack of evidence of its effectiveness.

Recent advancements in the field of male sexual health and reproduction present a bright future for the field with new diagnostic and therapeutic options on the horizon. However, it is apparent that demand still outpaces supply for mens health specialty care. Urologists must work diligently to fill this void to not only increase access for patients to receive evidence-based care, but also to prevent men from falling to prey to practices looking to take advantage of this unmet demand and a vulnerable patient population.

References

1. Jensen CFS, Ohl DA, Fode M, et al. Microdissection testicular sperm extraction versus multiple needle-pass percutaneous testicular sperm aspiration in men with nonobstructive azoospermia: a randomized clinical trial. Eur Urol. Published online May 19, 2022. doi:10.1016/j.eururo.2022.04.030

2. Lee R, Witherspoon L, Robinson M, et al. Automated rare sperm identification from low-magnification microscopy images of dissociated microsurgical testicular sperm extraction samples using deep learning. Fertil Steril. 2022;118(1):90-99. doi:10.1016/j.fertnstert.2022.03.011

3. Asanad K, Nusbaum D, Fuchs G, Rodman JCS, Samplaski MK. The impact of male infertility faculty on urology residency training. Andrologia. 2022;54(8):e14457. doi:10.1111/and.14457

4. Kalyvianakis D, Mykoniatis I, Pyrgidis N, et al. The effect of low-intensity shock wave therapy on moderate erectile dysfunction: a double-blind, randomized, sham-controlled clinical trial. J Urol. 2022;208(2):388-395. doi:10.1097/JU.0000000000002684

5. Sandoval-Salinas C, Saffon JP, Martnez JM, Corredor HA, Gallego A. Are radial pressure waves effective for the treatment of moderate or mild to moderate erectile dysfunction? A randomized sham therapy controlled clinical trial. J Sex Med. 2022;19(5):738-744. doi:10.1016/j.jsxm.2022.02.010

6. Weinberger JM, Shahinyan GK, Yang SC, et al. Shock wave therapy for erectile dysfunction: marketing and practice trends in major metropolitan areas in the United States. Urol Pract. 2022;9(3):212-219. doi:10.1097/UPJ.0000000000000299

Read more from the original source:
Male sexual health and reproductive medicine: All that glitters is not gold - Urology Times

Recommendation and review posted by Bethany Smith

DUBLIN--(BUSINESS WIRE)--The "Rare Disease Genetic Testing Market Size, Share & Trends Analysis Report by Disease Type (Neurological, CVDs), by Specialty (Molecular, Biochemical), by Technology (NGS, PCR-based), by End Use, and Segment Forecasts, 2022-2030" report has been added to ResearchAndMarkets.com's offering.

The global rare disease genetic testing market size is expected to reach USD 2.52 billion by 2030, registering a CAGR of 13.94% over the forecast period, according to this report. Effective regulatory plans to combat rare disease is one of the key drivers of the industry. Furthermore, the presence of a substantial number of registries that provide data and relevant information about related diseases has aided in revenue growth over the past years.

Ongoing conferences to raise awareness about rare and ultra-rare conditions are anticipated to boost the adoption of diagnostic kits and services. For instance, Ergomed and PSR Orphan Experts, with their offices in the U.K., Germany, the Netherlands, Poland, and other countries, participate in various activities that are aimed at raising awareness in this area.

Moreover, the Canadian Organization for Rare Disorders (CORD) offers a strong platform to streamline health policy and a healthcare system that is dedicated to the management of patients with disorders. The agency works with clinicians, researchers, governments, and the diagnostic industry to advance R&D, diagnosis, treatment, and service availability for all rare conditions in the country. As per the National Institutes of Health (NIH), around 30 million Americans have been identified with one of 7,000+ known rare diseases. The number of patients undergoing disease testing is expected to increase in the coming years with growing awareness. The U.S. celebrates Rare Disease Day and promotes developments in this area by raising awareness.

In addition, the presence of the Rare Diseases Clinical Research Network (RDCRN), an NIH-funded research network of 23 active consortia or research groups that includes patients, researchers, and clinicians who are focused on the diagnosis & treatment of disorders is anticipated to positively impact the industry.

Around 50% of the children with learning disabilities and approximately 60% of children with congenital conditions do not receive a definitive diagnosis to identify the cause of their disabilities. Furthermore, the lack of awareness among patients and families about diagnosis and genetic testing has further impeded the industry's growth.

North America dominated the industry in 2021 due to the high incidence of rare diseases, a large number of registries, the presence of substantial numbers of R&D facilities in this area, and extensive investments in diagnosis. Asia Pacific is expected to register the fastest CAGR during the forecast years owing to the presence of a substantial number of organizations that are focusing on disease management.

Rare Disease Genetic Testing Market Report Highlights

Market Dynamics

Market Drivers

Market Restraints

Market Opportunities

Market Challenges

Key Topics Covered:

Chapter 1 Research Methodology

Chapter 2 Executive Summary

Chapter 3 Market Variables, Trends, & Scope

Chapter 4 Industry Outlook

Chapter 5 Disease Type Business Analysis

Chapter 6 Technology Business Analysis

Chapter 7 Speciality Business Analysis

Chapter 8 End-Use Business Analysis

Chapter 9 Regional Business Analysis

Chapter 10 Company Profiles

Companies Mentioned

For more information about this report visit https://www.researchandmarkets.com/r/515scb

Read the original here:
Global Rare Disease Genetic Testing Market Report 2022: Ongoing Conferences to Raise Awareness About Rare and Ultra-Rare Conditions to Boost Growth -...

Recommendation and review posted by Bethany Smith

SOUTH SAN FRANCISCO, Calif. & ST. LOUIS--(BUSINESS WIRE)--Genome Medical, the leading telehealth provider of genetic services and genomics-based care, and Pierian, the global leader in advanced clinical genomics technology and services, announced a collaboration designed to streamline and optimize onsite genomics programs for health care organizations and provider groups. The companies services, when combined with genomic testing capabilities, create an end-to-end patient and clinician experience that elevates standards of care and patient outcomes.

Genome Medical and Pierian are working together to efficiently identify patients who may benefit from genomic testing and an enhanced clinical genomics workflow. The combined solution for clinicians facilitates the ordering of appropriate testing which is then processed in onsite laboratories supported by Pierian.

First, through its RISE Patient Engagement Modules, Genome Medical helps clinicians Reach, Inform, Support and Educate patients. RISE includes a Hereditary Cancer Risk Assessment Module that collects and analyzes family and personal health history to determine if a patient meets genetic testing criteria for hereditary cancer. When criteria is met and testing is ordered, laboratory customers utilize Pierians advanced technology platform to ingest, analyze, interpret and report on genomic insights for more precise care.

For physician-owned or -managed service organizations, Pierian and Genome Medical deliver a streamlined path to in-house, high-quality precision medicine programs that provide recommended and appropriate care to all patients who meet national standards for genomic testing. This can also include virtual post-test genetic counseling from Genome Medicals nationwide team to help explain the test findings and advise on recommended follow-on care, if needed.

We are excited to partner with a like minded innovator, Genome Medical, to combine our leading edge platforms and expertise to enable the clinicians we are privileged to serve, said Mark McDonough, CEO of Pierian. At Pierian, we are passionately committed to catalyzing precision medicine at scale through our tools, our team, our customers, and our partners like Genome Medical. We are united in our belief that all patients deserve access to high quality, affordable, genetic testing.

Genome Medical has pioneered a virtual model of tech-enabled care delivery and assembled an unmatched team of genetic specialists, enabling rapid, efficient access to genetic counseling and related services. The company offers flexible genetic services programs to approximately 100 partners, including health systems, diagnostic testing labs, insurers, and other partners. In addition, its services are a covered, in-network benefit for more than 160 million people in the U.S.

Genome Medical is pleased to be able to partner with Pierian to bring our patient screening and clinical genetic services to provider groups who are looking to improve and expand their genomic testing programs, said Jill Davies, CEO of Genome Medical. This collaboration represents two industry leaders delivering the services and tools that will make in-house genomic testing programs accessible to a wider array of providers and patients.

Pierian partners with academic centers, health systems, physician-owned laboratories and reference laboratories worldwide to establish high-quality clinical genomics programs and a global sharing network. With advanced interpretation technology connected to the most comprehensive knowledge base, Pierians unique, adaptive learning algorithms make intelligent associations between comprehensive datasets and individual patient results. Post analysis and interpretation, clinical reports are easy to generate, which empowers clinicians with genomic insights to fulfill the promise of precision care.

About Genome Medical

Genome Medical, the leading genomic care delivery company, is personalizing health care for all through on-demand access to genetic insights and genomic medicine. We operate as an independent virtual medical practice, powered by a digital health technology platform. By partnering with health systems, providers, health plans, employers, labs and biopharmaceutical companies, we expand the reach and impact of precision medicine. We provide clinical assessments and tools, test recommendations and ordering, and personalized care plans to deliver optimal patient care and improve health outcomes. The company, which is headquartered in South San Francisco, has been honored as The Best Digital Health Company to Work For by Rock Health, Fenwick & West and Goldman Sachs in their Top 50 in Digital Health awards. To learn more, visit genomemedical.com and follow @GenomeMed.

About Pierian

Pierian is a partner in precision medicine, enabling clinicians and medical facilities to advance clinical genomics programs and modernize patient care. We believe in the potential of genomics to transform human health and are working to ensure that communities anywhere can experience the benefits. We curate the worlds genetic knowledge, and our advanced interpretation technology combines this knowledgebase with adaptive learning algorithms that connect diverse sources of information through machine learning. When applied in clinical settings our platform is paired with our enabling services which support workflow design, implementation, validation, interpretation, and reimbursement. For more information, visit http://www.pieriandx.com.

Originally posted here:
Genome Medical and Pierian Announce Collaboration to Optimize Genomic Testing Programs - Business Wire

Recommendation and review posted by Bethany Smith

DNA testing has become increasingly popular over the years, with one of the most prominent companies being DNAfit. DNA testing claims to assess your genetic makeup, ultimately helping users gain insights into their health and fitness that they wouldnt otherwise have. The company can then use this information to tailor a unique workout and diet plan specifically designed for your body.

DNAfit claims to provide some of the most comprehensive DNA testing available. The company also states that it can provide unrivaled insight into users diet, nutrition, fitness, and well-being. However, some of the health claims they make are unsupported by evidence, and theres little scientific evidence for DNA-based personalization. For these reasons, we dont recommend purchasing or using DNAfit kits.

Read on to discover what you need to know about this company and alternatives to consider.

DNAfit is a direct-to-consumer genetic testing company that provides DNA-based insights into diet, fitness, and wellness. The London-based company was founded in 2013 by Avi Lasarow to provide people with a simple way to understand how their genes affect their health and fitness.

The company offers three main products: Diet Fit, Heath Fit, and Circle Premium, with the latter being its most comprehensive option. Each kit provides everything you need to collect a DNA sample from home and send it to their state-of-the-art laboratory for analysis.

Once the results are ready, youll receive a personalized report with actionable insights and recommendations based on your unique genetic makeup.

The primary aim of DNAfit is to provide you with information you can use to improve your health and fitness.

The company offers three different tests, each with a different focus:

We evaluated DNAfit by looking at the companys medical claims and business standards.

Although the company doesnt make any disease claims, some of its health claims are unsupported by solid scientific evidence. For example, personalizing diet and fitness plans according to your genes doesnt correlate with the available evidence.

Likewise, claims that DNA testing results can help you effectively manage stress and sleep are largely unfounded.

In regards to business standards, DNAfit fared well. They have no Food and Drug Administration (FDA) warnings or third-party certifications. Additionally, the company has good privacy standards with SSL encryption and a valid security certificate.

DNAfit is owned by a parent company called Prenetics, which currently doesnt have a Better Business Bureau page. They do, however, have a 3.9-star rating from more than 4,500 reviewers on Trustpilot. Meanwhile, DNAfit has a 3.8-star rating on Trustpilot.

Diet Fit is the most basic of DNAfits offerings. It provides insights into your unique nutrigenetic profile, which is designed to help you understand how your body responds to various types of food and any sensitivities and intolerances you may have. According to the company, youll find out which foods you should eat more or less of to lose weight, maintain weight, or gain muscle with personalized dietary recommendations.

Youll also receive information on building your perfect meal according to how you respond to carbohydrates and fats. You can program the MealPlanner (a personalized meal planning service) with your aim, likes, and dislikes, and it generates a genetically guided recipe plan complete with a shopping list builder.

Health Fit further builds on the diet and nutrient insights from Diet Fit. Youll receive the same insights and have access to the personalized MealPlanner.

Where Health Fit differs is with its fitness response genetic markers. These markers include:

These insights allow you to discover how to optimize your workouts and guide your training choices.

Youll receive information on your stress and sleep profile to help you improve your mental and physical well-being.

Circle Premium is DNAfits most comprehensive offering. In addition to the information provided in Diet Fit and Health Fit, the Circle Premium report is said to assess your genetic risk of certain diseases like dementia, type II diabetes, irritable bowel syndrome, and others.

The aim is to help you better understand your health and take preventive measures like lifestyle changes, check-ups, or more frequent cancer screenings.

It includes over 350 reports covering:

For those thinking of starting a family, the reports provide information on any inherited conditions that could be passed down to future generations.

When you receive your DNAfit report, its important to keep in mind that its only one part of the picture. The results should be used as a guide and not as a certainty.

For example, if your report says you have a higher risk of developing type II diabetes, it doesnt mean youll definitely have the condition in the future. Likewise, a lower risk doesnt necessarily mean symptoms of the condition wont occur.

Remember that your DNA is just one factor in the development of disease. Other important considerations are your lifestyle choices, environment, and more.

The Diet Fit report is easy to understand and isnt too dense in information. It details how your genetics may affect the way you metabolize carbs and fats. It also outlines if you have the required needs for certain vitamins or nutrients.

There are sections for:

The meal planner feature allows you to input your food preferences and generates a genetically tailored meal plan.

The Health Fit report is similar to the Diet Fit report in terms of content and layout. However, it builds on the information with details on how your genes affect fitness levels and provides insights on optimizing your workouts.

It includes:

Theres also information on your stress and sleep profile. The results outline how you cope with stress and your tolerance levels. Youll also find out if youre a warrior or a strategist, which identifies how you process information and perform tasks while under stress.

The Circle Premium report is DNAfits most comprehensive offering. In addition to the information provided in Diet Fit and Health Fit, the Circle Premium report provides the following information:

Youll also discover your ancestry and information on various traits. These include success, behavioral, physical, personality, and gender so you can better understand yourself and your background.

DNAfit claims that they take users privacy very seriously. It was the first company of its kind to become certified by ISO 27001, a globally recognized framework for best practices on information storage and security.

Rather than by name, DNAfit stores your data by ID number and claims that they destroy your samples after use. Your data wont be shared with people outside the company, and DNAfit does not sell your information to third parties.

Here are some other brands that provide DNA testing services. These recommended alternatives have passed Healthlines medical and business standards.

Everlywell provides a convenient way of checking different health issues from allergies to STIs and food sensitivities. However, theres a lack of evidence to support the methods they use for testing food sensitivities. Experts feel that these tests can provide inaccurate and misleading information, so its important to take these results with discretion.

Overall, Everlywell has a solid reputation besides the criticism about their controversial food sensitivity testing. They also use Clinical Laboratory Improvement Amendments (CLIA)-certified laboratories, so youre sure of quality service and results.

Everlywell sells a wide range of at-home testing kits that cover:

Like Everlywell, myLAB Box offers a convenient way to test for STIs and other health conditions from the comfort of your home. In addition, they offer free shipping and results in as little as 2 to 5 days.

They offer sensitivity testing for 96 different foods, and they test for IgA, IgG, and IgG4. This might provide a better picture than relying on IgG alone. But, bear in mind that the test doesnt test for food allergies, as this requires IgE antibody testing.

Other home tests sold by myLAB Box include:

FoodMarble uses a type of breathalyzer that claims to help you figure out which foods cause digestive issues. The device is small and portable, so you that can take it with you on the go.

To use it, you simply breathe into the device after eating, and it tells you if the food is digesting properly. The device measures hydrogen levels in your breath, and together with the app, it provides accurate information.

FoodMarble sells two breathalyzers:

If you still decide that youd like to try out DNAfits at-home DNA test kits, there are a few things to keep in mind before purchasing.

You should first talk to your doctor, who can more accurately assess your medical, health, and family history. They might be able to provide personalized suggestions based on your diet and fitness patterns, especially if youre looking to lose weight. They may also be able to provide some insights by talking through factors like stress, sleep, and your general well-being.

Your doctor may also be able to suggest lab or food sensitivity tests that can provide information regarding food intolerances and your risk of certain diseases like diabetes.

The brand passed our business vetting standards, meaning its a reputable company without any FDA or Federal Trade Commission (FTC) warnings against them. There also havent been any lawsuits filed.

DNAfit does not yet have a Better Business Bureau page, and its products do not appear on Amazon.

However, it has a Trustpilot page that scores DNAfit 3.9 out of 5 stars with more than 2,500 reviews. Although the reviews are good overall, some customers were unimpressed. They claim that the information provided is very basic and vague.

Users also reported problems with accessing the app and poor customer service.

In short, probably not. No scientific evidence supports claims that these tests can help you lose weight or improve your fitness.

A 2018 study found that DNA testing couldnt help guide people to a specific weight loss regimen that was more likely to be successful. Although many companies claim these effects, it seems there is no difference in weight loss between people following diets that allegedly match their genotype compared to those on standard diets.

The services offered by DNAfit are not inexpensive, starting at around $150 for Diet Fit. However, there is little scientific evidence underpinning their value, so its unlikely that DNAfit is worth the money.

That said, many of the reviews on Trustpilot are positive, with some people stating they would happily recommend DNAfit. So, it comes down to personal choice and budget as to whether DNAfit is worth it to you.

DNAfit states that their at-home DNA tests are accurate, but its tricky to find specific figures. However, they claim to regularly spot-check their labs to ensure they test samples correctly and provide results that are 100% accurate.

Currently, the FDA has only approved 23andMe for some of their at-home DNA tests. Overall, the industry is not regulated and has no independent analysis to verify the sellers claims, so you should still use caution when purchasing a test.

However, generally, DNA testing is a safe procedure. Most kits require a cheek swab or saliva sample, so there are essentially no associated risks. But, when samples are self-collected at home, theres an increased risk of contamination and inaccuracies.

DNAfit is a direct-to-consumer DNA testing company that offers several different tests, including Diet Fit, Health Fit, and Circle Premium.

The company has a good reputation, with few complaints from customers. However, the scientific evidence underpinning the value of their tests is lacking, and there are health claims unsupported by evidence.

Overall, its unclear whether DNAfit is worth the money, and we dont recommend purchasing or using the service for these reasons.

Zia Sherrell is a health copywriter and digital health journalist with over a decade of experience covering diverse topics from public health to medical cannabis, nutrition, and biomedical science. Her mission is to empower and educate people by bringing health matters to life with engaging, evidence-based writing.

View original post here:
DNAfit Review: What It Can and Can't Tell You - Healthline

Recommendation and review posted by Bethany Smith

Jen Culton, a 48-year-old mom of four from Omaha, Nebraska, never thought much about genetic mutations. "I had read in magazines that Angelina Jolie had a preventative double mastectomy, and I thought, 'That's really aggressive for someone who doesn't even know if she's going to get breast cancer,'" Culton said. A family diagnosis changed her perspective on her own health and that of her daughters.

In July 2013, Culton's older sister who was 38 years old at the time learned she had breast cancer. A genetic test showed she had a gene mutation called BRCA1 predisposing her to cancer, so her oncologist suggested all of her siblings get tested. That's when Jen learned she had the same mutation.

According to the National Cancer Institute, people who inherit BRCA gene mutations have an increased risk of multiple cancers, particularly breast and ovarian. They also tend to develop these cancers earlier than people who don't carry these mutations.

People who learn they have these genetic mutations can take steps to screen for early signs of cancer, or to prevent the cancers from developing altogether.

Culton ultimately had all of her reproductive organs and her breasts surgically removed, drastically lowering her risk of developing ovarian or breast cancer. Now, she's tasked with potentially guiding her daughters down a similar path.

Culton's two older daughters have already undergone genetic testing. So far, only Sammi Jen's 19-year-old has tested positive for the BRCA1 mutation. "I cried all day, because I felt guilty that I passed this on to her, but Sammi took the news extremely well," Culton said.

Sammi's already entertaining the idea of having the same surgeries her mom did, but it'll probably be a few years before she takes any medical-preventative action.

The National Comprehensive Cancer Network, an organization that develops preventative guidelines for people with a high risk of genetic conditions, currently advises medical providers to begin screening people with a BRCA1 or 2 mutation in their 20s regardless of when they learn about the mutation.

For people with a BRCA1 mutation, guidelines recommend routine breast imaging to detect early signs of breast cancer beginning at age 25. Screening may start sooner for people with a family member who had cancer at a younger age.

"It's very rare for a BRCA1 carrier to have breast cancer younger than age 25," Nicolette Chun, a genetics counselor at the Stanford Cancer Institute, said. "If there is a family history of breast cancer under age 30; we start MRI screening as young as 20."

In place of breast screening, people with BRCA mutations can have mastectomies, or surgical removal of the breasts, at any point. Medical experts usually recommend oophorectomies, or the removal of one or both ovaries, around age 40 or sooner for those who either don't want kids or are done having them.

Culton's 10-year-old daughter, Daisy, has asked about the mutation, but Culton doesn't plan to screen her until she's at least 18. "My husband and I don't want to cause anxiety or put pressure on her, especially because you can't do anything if you find out you have the mutation that young," Culton said.

Experts agree that finding out about a mutation as a child or teen may do more harm than good.

Most major health organizations, including the National Society of Genetic Counselors, advise against testing minors for adult-onset conditions: There's no medical action to take, and learning about a mutation can cause unneeded fear and anxiety for years to come.

"When deciding about genetic testing, we have to consider the effects it's going to have on a person's healthcare, but we also have to think about the person's mental health," Skyler Jesz, a board-certified physician assistant who has worked with Culton and other patients to decide when to perform genetic testing, said. "It's difficult for an adult to have a conversation about an increased risk, let alone a 10-year-old."

While learning about a genetic mutation gave Culton a sense of control over her own health and she feels a sense of responsibility over her daughters' well-being protecting her daughters includes prioritizing their mental health. "I want Daisy to have a childhood," she said. "We can deal with this when she's older."

Go here to read the rest:
One of my daughters and I have the BRCA1 gene mutation - Insider

Recommendation and review posted by Bethany Smith

Direct-to-consumer home genetic kits allowed startups like 23andMe to offer health and ancestry insights at an affordable cost. Now, similar tech is coming to pets.

Itll help vets, breeders and pet parents to verify parentage and breed, diagnose diseases and plan for future health risks.

Everything we have seen happening in humans, in terms of predictive and personalised medicine and genetics-based diagnostics, has migrated into the pet space, says Sergey Jakimov, founding partner of LongeVC, a European VC fund that focuses on early-stage biotech and longevity. This is super exciting because pets, as living beings, have equalised themselves in importance in terms of how much money and attention is spent on their longevity, and in disease diagnostics and prevention.

Its not the first time human health innovation has come to the animal world US-based Signal Pet, for example, provides artificial intelligence-based radiology but animal genetics could be big business.

Animal genetics market revenue is predicted to exceed $6.4bn by 2027, up from $99m in 2020. Sifted dug into the sector and found the startups to watch and the VCs watching them.

Feragen, an Austria-based pet genetics startup, sees the vet sector as a growth engine for its business. It wants to move from diagnostics, where such tools are common, into disease prevention.

Puppies are more like family members

We want to push the prevention angle. What can we learn from genetics to create a life plan for a dog? says Anja Geretschlger, founder and CEO. Pet parents are becoming more interested in understanding the risk of diseases that might come when the pet is five or six, so they are more prepared when the symptoms show up.

Michael Geretschlger, Anjas husband and collaborator, says preventive health is getting more [attention] as puppies are more like family members. Anja Geretschlger adds that genetic insights are valuable for breeders in the era of designer dogs.

This is because cross-breeding can lead to health complications, such as labradoodles developing skin problems due to different fur structures between labradors and poodles.

Another European player is Germany-based Generatio, which provides genetic testing for animal owners, vets and breeders.

Theres also UK-based AffinityDNA, acquired in May by Australian diagnostics company Genetic Technologies, which provides animal testing for allergies and intolerances, paternity testing and direct-to-consumer (DTC) genetic tests from companies like Embark, Wisdom Panel and BasePaws.

Genetic Technologies portfolio includes General Genetics Corporation and associated brand EasyDNA, which offers UK pet owners breed composition tests, disease susceptibility tests for dogs, and feline and equine offerings.

European VCs are also interested in startups across the pond. Garri Zmudze, a Latvian biotech angel investor and founder of Switzerland-based LongeVC, investedin Basepaws, the American cat genetics company recently acquired by Zoetis, an animal medicines and vaccinations company.

Basepaws plans to expand into the veterinary portfolio of genetic, oral and microbiome screening tools for disease risk, screening 64 feline health markers and over 210 canine health markers.

For some, the pet genetics space is not just a play on the pet market but could inform human health and longevity science. Some diseases are rare in humans but are common in certain breeds of pets, who are useful for studies into genetic disease origins.

There is a tight connection between humans and animals and we can learn from both, says Anja Geretschlger.

Zmudzes investment in Basepaws, for instance, was not a pet consumer market bet at all. Instead it was aligned with his interest in human longevity, given the genetic overlaps between animals and humans in diseases like cancer and some neurodegenerative conditions.

There is a tight connection between humans and animals and we can learn from both

These overlaps are the reason we have animal models in clinical trials, because the metabolic processes are translatable, says Jakimov. There are tonnes of matches.

Matt Kaeberlein, professor of laboratory medicine and pathology at the University of Washington School of Medicine and head of the dog ageing project, a world-leading biological study of ageing in dogs, sits on the LongeVC advisory board, alongside executives from European pharmaceutical giants Roche and Novartis. And Zmudze was also an investor in Insilico Medicine, an AI drug discovery unicorn.

As home to many of the worlds top pharmaceutical companies, Europe could be a major player in longevity research. Switzerland is developing a Longevity Valley initiative, Bristol Myers Squibb and Merck are major investors in cancer immunotherapies and the pharma industry is investing in early stage longevity companies like senescent cells companies, through initiatives like Mercks early stage venture arm.

Pharmaceutical companies live in the future, they live in 10 to 20 year cycles, says Jakimov. They are super focused on the longevity sector.

This article first appeared in our monthly Unleashed pet tech newsletter, a collaboration with Purina Accelerator Lab. All content is editorially independent.Sign upto our newsletter here to keep up to date with the latest goings on in the European pet tech industry.

More here:
Purrsonalised health: The startups and VCs betting on pet genetics - Sifted

Recommendation and review posted by Bethany Smith

The need for understandable and transparent pricing for medical services in the U.S. has increased in urgency with the continuing rise of out-of-pocket healthcare costs.

Americans are delaying healthcare because they are unsure of the cost or they cannot afford it. A recent Willis Tower Watson (WTW) survey of 9,600 U.S. workers showed that 4 in 10 people deferred healthcare in the past year, with 28% delaying or canceling a medical procedure and 17% not filling a prescription.

Recent federal mandates are inching price transparency forward with the goal of improving care while reducing waste and reigning in costs. With lab values as the basis for 70 percent of medical decisions, lab testing transparency for price and appropriate use is essential to lowering costs for patients.

But price transparency goes beyond reducing costs. It will realign how labs, providers, and payers work together to order, approve, and pay for the right care that moves us closer to the Triple Aim of improving the patient experience, improving the health of populations, and reducing costs.

You cant flip a transparency switch

Unfortunately, the lab testing industry cant flip a switch and make transparency universal.

With healthcare affordability as a big concern, Americans are becoming more involved in managing their care, and that includes switching health insurance plans to secure the best benefits and reduce out-of-pocket costs. To help support these consumer decisions, recent federal regulations were enacted.

Even with the government stepping in to improve access to pricing data and reduce unexpected costs, we are still far from having universal lab testing transparency. Achieving full transparency will require: 1) coordinated education, 2) simpler data access, and 3) the strategic implementation of payment integrity programs to eliminate unnecessary lab tests while identifying underutilized tests that improve patient care and outcomes.

Location, location, location

Education about the impact of lab testing location on cost has to move upstream in the care process. Today, that often happens after the lab test has occurred. Patients and their physicians need to understand and discuss which lab testing locations bring the most value and inform the best care.

The lab test location can result in increased costs, over-testing, and unnecessary tests.

Hospital-owned labs are typically paid more than independent labs. Lab tests performed at hospital-owned labs are generally 2.5 to 4.5 times the cost of an independent lab. Avalons analysis of paid claims demonstrates that hospital outpatient labs are paid on average 300%-400% of Medicares independent diagnostic fee schedule. Hospitals frequently argue they need to charge more to support their specialty test innovation and development. That doesnt hold true for routine testing, though. For example, some hospitals will be reimbursed $100 for a routine test, while an independent or non-hospital lab will be reimbursed on average $20 for the same test, performed on the same machine.

Physician office lab testing is more costly and more frequent. When physician offices have their own lab equipment, our analysis of paid claims demonstrates that physician offices are reimbursed on average 120% to 130% of Medicares independent diagnostic fee schedule for those tests. In addition, the frequency of lab testing increases when the laboratory testing is performed in the physicians office. When the elapsed time between tests is less than the time it takes for the body to produce new chemicals in the body (measured by the half-life of the chemical) this is not a clinically useful frequency of testing.

Our analysis of paid claims demonstrates independent labs conduct the most clinically useless test units. The laboratory industry develops the test order menus from which physicians order labs. While developing panels, which represent useful tests commonly ordered together, the labs will add additional tests that are not useful to the physicians diagnostic evaluation. This is known as panel stuffing in the industry. Panel stuffing is a wasteful practice that adds unnecessary tests (those that dont comply with a health insurance plans published policies) within routine lab panels and increases test costs for patients.

Consider that several labs add an experimental subcomponent analysis of Vitamin D to the Vitamin D panel menu. Also, consider that many labs, on the menu for evaluation of thyroid, include seven unique tests when two are important to the most common clinical scenarios. This raises the cost of routine thyroid testing from around $30 to about $137.

Lab tests that lack clinical indications can lead to unneeded sample collection from patients as well as a higher risk of false-positive results and unnecessary costs. We identified that on average there is approximately $2 per member/per month worth of obvious waste in processed claims. This represents the total amount allowed for the testing. Patients, on average, pay 1/3 of the cost at the point of service and payers pay the other 2/3 of that cost. Another way to look at this same data is that for every 1 million members with health benefits, approximately $24MM dollars of useless testing is reimbursed per year, with patients paying $8MM of that out of pocket.

Promoting lab testing at the right locationfor both cost and caremay be harder than it sounds. Physicians who are part of health systems may be pressured and/or incentivized to send patients to the affiliated hospital labs. Plus, payers are often hesitant to educate their members about lower-cost lab testing options because of various provisions in their contracts with hospitals.

Sharing the facts about lab testing locations will require a national education campaignmuch like the campaign conducted for the $0 co-pay generic drugsto motivate patients to insist on having lab testing performed at lower-cost locations and to only pay for tests that benefit patient care.

Right test, right time, right care

Patients, providers, and payers all want the right care. Appropriate lab testing is a critical driver behind this. The demand for lab testing is growing as more providers recognize the importance lab results play in confirming the diagnosis, monitoring patients treatment responses, and monitoring diseases significant to public health (i.e., Covid-19). The high prevalence of chronic and acute diseases, an aging population, and advancements in genetic testing are also fueling this growth. There is an expected 10% compounded annual growth rate through 2029.

Receiving the right care should be as simple as having the right lab test at the right time (in the right location). However, the current healthcare ecosystem includes trends that undermine the journey to this valuable goal. When looking closely at the 13+ billion lab tests performed annually across the U.S., 30% of lab tests are unnecessary, 30% of patients dont receive the tests they need, and 1 in 3 genetic tests are ordered in error.

When you consider that lab testing is the gateway for diagnosis and treatment of many conditions, it transforms each test from being a passive event to a critical data point for proactive value-based care success. With this backdrop, a payment integrity program that includes lab benefit management can serve as a strategic lever to curb these negative testing trends and advance the Triple Aim.

With sound science at the core, payment integrity programs provide input from policies developed by independent clinical boards on what types of tests are not evidence-based and emphasize the appropriate units for routine and genetic testing. This process flags non-adherent tests (from both panel stuffing and inappropriate genetic test orders) and underutilized tests that can inform patient care, especially in cancer care.The ultimate impact is for patients to receive the right tests at the right time to better inform diagnoses and care plans, reduce waste in time and treatments that are not helping patients, and achieve cost alignment that drives the right outcomes.

As the U.S. healthcare industry continues to advance value-based care and population health, lab testing price transparency and payment integrity programs should be a priority.

Photo: champc, Getty Images

Excerpt from:
Lab testing transparency will improve patient care and lower costs - MedCity News

Recommendation and review posted by Bethany Smith

A new tool that uses artificial intelligence (AI) to analyze healthcare records may aid in the early diagnosis of fragile X syndrome, a new study reports.

By incorporating a combination of co-occurring conditions, an AI-assisted pre-screening tool was developed and validated to identify potential cases at least 5 years earlier than the time of clinical diagnosis, the researchers wrote.

The scientists said their AI tool was used successfully to analyze a healthcare database in the U.S. state of Wisconsin. Moving forward, this artificial intelligence-based program could be used across other healthcare databases to better and potentially much sooner identify individuals who may be affected by fragile X.

Our AI-assisted pre-screening approach can facilitate and accelerate the clinical diagnosis of [fragile X syndrome] and decrease the duration of the diagnostic odyssey and degree of stress experienced by patients and their families, the team wrote.

One concern in the move forward is that the AI tool thus far was only used in healthcare systems that were predominately comprised of white patients. More testing and validation is needed in other racial and ethnic patient populations, the researchers said.

The study, Advancing artificial intelligence-assisted pre-screening for fragile X syndrome, was published inBMC Medical Informatics and Decision Making.

Fragile X syndrome can manifest very differently from person to person, which makes diagnosing the genetic disorder a challenge. Studies have shown a marked gap between the estimated prevalence of fragile X and the actual number of people diagnosed suggesting that as many as 70% of people affected by fragile X syndrome have not been properly diagnosed.

To bridge that gap, a team led by scientists at the University of Wisconsin-Madison created an artificial intelligence tool aimed at better diagnosing fragile X syndrome. This tool is applied to data that is routinely collected in electronic healthcare records (EHRs).

Their aim was to identify data in such EHRs that could predict the diagnosis of fragile X, even before the disorder itself is formally diagnosed.

The pre-screening model is not intended to be a replacement for genetic testing, but it can serve as a tool to automatically alert physicians about the presence of multiple [fragile X syndrome]-related phenotypes in the patients medical records, the scientists wrote.

By prompting the physician to further evaluate such individuals and refer them for genetic testing and counseling, our approach could accelerate the diagnostic process and be instrumental in identifying un-diagnosed individuals in the population and addressing their health conditions, the team wrote.

To create the tool, the team used EHR data collected from 1979 to 2018 served by the Marshfield Clinic Health System in Wisconsin.

From the data, the team identified 55 people who had been diagnosed with fragile X syndrome at an age of 10 or older. The scientists also used data from 5,500 people without a fragile X diagnosis, who were similar to the patients in terms of age and sex.

For all of these patients, the researchers extracted data from five years before the formal diagnosis of fragile X syndrome, or the equivalent ages for controls.

All data used in this study are directly collected in a medical setting and are in fact real world data from actual patients, providing further proof of [the AI tools] potential utility in real world clinical applications, the team noted.

With these data in hand, the researchers then trained their artificial intelligence algorithm using a mathematical strategy called random forest. Conceptually, the AI tool uses a set of mathematical rules to look for patterns in the diagnostic codes that could differentiate between people with or without fragile X syndrome.

To test the utility of the trained algorithm, the scientists tested it on data collected from UW Health, a separate healthcare system in Wisconsin.

Our next step, reported here for the first time, was to evaluate the performance of this model in a new unseen dataset, i.e., an external validation study, they wrote.

In this dataset, the team identified data for 52 fragile X cases and 5,200 people without the disorder, matched for sex and age.

To test the tools accuracy, the researchers calculated a statistical measurement called the area under the receiver operating characteristic curve, or AUROC. This is basically a measurement of how well a test can tell the difference between two groups i.e., fragile X or not. AUROC values can range from 0.5 to 1, with higher values suggesting better ability to discriminate.

In the original Marshfield dataset, the AUROC for the AI tool was 0.798. In the UW Health analysis, it was 0.795.

The AUROCs of the predictive models created and evaluated using the Marshfield cases and the UW Health cases were almost identical (0.798 vs. 0.795), representing the high level of reproducibility of results in different health care systems, the scientists wrote.

Our AI-assisted pre-screening tool could significantly improve the diagnostic process and could provide substantial benefits for patients, families and the health care system, they concluded.

A noted limitation of this analysis was that nearly 90% of patients in both healthcare systems were white. The researchers highlighted a need to further validate this model in other populations, especially those of non-European ancestry.

Additional studies on larger populations will provide more precise information on the performance of the model, they wrote.

Read the rest here:
Artificial Intelligence Tool May Help in Early Diagnosis of... - Fragile X News Today

Recommendation and review posted by Bethany Smith

The discovery of a pig carcass on national forest land near Lake Koocanusa has prompted the Montana Department of Livestock, Montana Invasive Species Council, and USDA-APHIS Wildlife Services to hold public meetings about feral swine in Eureka and Libby on Thursday, Sept. 22.

The Libby meeting will be held at 1 p.m. in the Ponderosa Room at Libby City Hall.

While the carcass has not been confirmed as a feral pig, it has features often found in feral swine. The animal could also be a heritage breed of swine that looks similar.

The meetings will cover Montana laws restricting feral swine in the state, identifying signs and damage, and how to report sightings.

The Eureka meeting will be held at 6:30 p.m. at the Timbers Lodge (101 Julian Drive).

In August, the Department of Livestock received a report of a dead pig on Forest Service land west of Lake Koocanusa.

Canvassing of the area has failed to locate an owner of the animal. The investigation is ongoing, including genetic testing, but results wont be available for several weeks and may not be successful due to the decomposed state of the carcass.

Feral swine are an invasive species that pose a serious threat to Montanas agriculture, livestock, wildlife and landscapes.

Officials are asking the public to be on the lookout for feral swine or wild pigs and to report sightings to 406-444-2976.

While feral swine are not yet in Montana, we are aware of their expanding range in Canada and the risk of introduction through natural animal movement, escape of domestic animals, or deliberate release, said Tahnee Szymanski, Assistant State Veterinarian with the Department of Livestock, In coordination with our partners, we are working hard to prevent feral swine introduction.

For more information about feral swine and the Squeal on Pigs campaign go to squealonpigsmt.com.

For more information about these meetings contact the Montana Invasive Species Council at 406-444-0547.

The mission of the Montana Department of Livestock is to control and eradicate animal diseases, prevent the transmission of animal diseases to humans, and to protect the livestock industry from theft and predatory animals.

For more information on the Montana Department of Livestock, visit liv.mt.gov.

Original post:
Feral swine meetings Thursday in Eureka and Libby - The Western News

Recommendation and review posted by Bethany Smith

AGARWAL: How did decide on the treatment path you did?

Chowdhury: I have been involved in the TITAN study [NCT02489318] as one of the clinical chairs, so I have [vast] experience with apalutamide.1 We know that a significant subgroup in the TITAN study [benefited from therapy] and we know that that subgroup continued to show a benefit. This is a very reasonable treatment. The best evidence [from TITAN] supports apalutamide, and enzalutamide [Xtandi] is supported by the ARCHES study [NCT02677896], and both are very reasonable treatments.2 Certainly in my own practice, we have a lot of experience with apalutamide from TITAN for routine clinical practice. We also use the drug in nonmetastatic [castration-sensitive prostate cancer (CSPC) based on results from the phase 3 SPARTAN trial (NCT01946204)]. Doublets rather than ADT alone have been shown to produce a significant benefit, and sometimes we underestimate that. [This patient] has a poor prognosis and high-volume disease; he isnt someone with a super scan and a PSA level of several hundred ng/mL. Sometimes we can underestimate the disease and the lethality of disease in a man who has many years to live and is young.

Agarwal: How do you determine a treatment based on the available options for a certain patient?

Liaw: [The results of] multiple studies have shown us that moving some of the drugs that we once reserved for castration-resistant disease into the hormone-sensitive metastatic setting gives us a much more augmented long-term disease benefit in terms of disease control and overall survival [OS] benefit. I will talk to a patient who has high-volume disease about 4 options: docetaxel; apalutamide, such as what was done for this particular patient; abiraterone [Zytiga]; and enzalutamide. In someone who might have low-volume disease, sometimes theres a good discussion as to whether we do prostate radiation. In this case, he doesnt have a prostate anymore, so we wouldnt talk about that. But for de novo metastatic low-volume disease, you might think about that.

Often patients question how we choose among these 4 agents to use for systemic therapy, and no one necessarily has the right answer. Many times, it does come down to the patient themselves and their characteristics. In a patient like this who otherwise has an excellent performance status, is young, and has very few comorbidities, hes [a candidate] for all treatment options, vs some patients who might have more issues with steroid use, which would make things like abiraterone a bit harder for us to challenge them with. Patients who have hepatic or renal insufficiency might not be quite as good candidates for chemotherapy. One last thing that sometimes comes into the mix is cost issues with medications. For drugs like abiraterone which are off patent at this point, the price point might be more attractive, vs a drug like enzalutamide or apalutamide that is still branded.

Agarwal: How do you decide among the 4 options to treat prostate cancer?

Lowentritt: In 2019, apalutamide and enzalutamide got their approvals in the metastatic CSPC [mCSPC] space.3,4 Until then, only chemotherapy and abiraterone were available, and only 30% of people received abiraterone, so you still had the vast majority of people getting ADT alone. When I see my patients today and I talk to my partners and colleagues, its important to get to that first level of understanding that we need to intensify their therapy. Plenty of evidence shows that the standard of care [SOC] has long evolved past ADT alone. When Im talking to my patients now with a menu of offerings, I still do counsel them and encourage them. It is very clear that an oral option is often favored, although [in the future,] ways will evolve where theyre not necessarily mutually exclusive.

Abiraterone is still a wonderful medication because the exposure is a little bit longer in this part of the disease state, and patients will be on this for a longer period. Theres a slight concern about chronic steroid use in these patients. I dont know if that weighs heavier than the other concerns about chronic exposure to these medications on other systems in the body, but thats a component of it. Theres also a considerable cost-saving for patients who will be on drugs for a long time, so I definitely consider abiraterone as an excellent option. I have used both other options that are approved, apalutamide and enzalutamide, in this space, and there are differences. I tend to avoid enzalutamide in my elderly patients. My personal experience is there can be some increased level of problems with equilibrium or memory or just word finding for those patients over the long haul. I do think we have to recognize that this will be a longer course of therapy. Thankfully theyre all successful in extending life and having a long course, so I have these discussions [with my patients]. I try to do personal assessments, and there are some drug-drug interactions, although theyre minimal. Thyroid disease is more of a unique marker for apalutamide, but it generally occurs as small incidents and is easily managed. I have these discussions, but it ends up being a personal choice based on a lot of individual patient factors.

Agarwal: How do drug interactions play a role in your clinic? When do you decide to switch an anticoagulant rather than the prostate cancer treatment?

Liaw: In my personal practice, we do run into drug interactions here and there. It usually boils down to what options we have for anticoagulants and for treatment of prostate cancer. If their need for coagulation is a much more urgent issue, like in someone who has a saddle embolism, Im not touching their anticoagulant. Its more of an issue and is my bigger priority. For prostate cancer, if there are other equally suitable options and I can easily make a substitution, Im willing to do so if its a drug interaction issue. I dont come up against these tough decisions daily, but here and there we do have to make a decision to switch away from one drug to another.

Agarwal: When looking at the quality of life, why choose ADT plus apalutamide?

Chowdhury: For quality of life, we look at the studies, personal experiences, and at the patient in front of usand sometimes we underestimate how scary it is for a patient faced with metastatic disease. Patients like this want the best treatment. Sometimes because ADT works well, its an active treatment. Its among the most active treatments we have in solid tumor oncology. We focus on the current outlook rather than thinking about whats coming for the patient in a relatively short period of time. We see that [progression-free survival] on ADT alone is 12 to 18 months, and its shorter in terms of PSA, which obviously drives decision-making anxiety and treatment change. What I discuss with them are the data.

I honestly believe ADT is an elephant in the room. In the [United Kingdom], we underestimate toxicity to the detriment of our patients. Going forward, particularly in patients with lower-volume disease and good responses. Im hoping well see the escalation of therapy, particularly ADT, and get men on single-agent AR [androgen receptor] inhibitors, although that is not [an approved indication].

Something that weve talked about quite a lot in studies such as TITAN, ARCHES, and ARASEN [NCT02799602] is that PSA was blinded to patients and investigators.5 The benefit of PSA psychologically is massive for patients. When patients walk into our clinic room, one of the first, if not the first, question is about PSA. PSA is critical and PSA responsesthe depth, duration, and how that also ties in with the quality of lifeare important. For me, apalutamide and darolutamide [Nubeqa] are both good drugs. I chose apalutamide because of my experience in TITAN.

Agarwal: Why did you choose to use triplet therapy for this patient?

Liaw: In a case like this, we have many of the same options that we spoke about earlier in terms of doublet therapy. One thing to highlight is that this is a patient who has come in with de novo disease, which typically has a worse prognosis vs [a case where] metastatic recurrence has come back on the metachronous end. We want to put our best foot forward, so to speak. He is a 59-year-old patient who is otherwise independent with no medical issues, and he is also highly symptomatic. He has already had a pathologic fracture. His PSA numbers are tremendously elevated. Everything leads to not just high-volume disease, but many aspects of this disease that are high risk as well.

Data that support the use of triplet regimens like ADT, docetaxel, and abirateronea regimen that was championed in the PEACE-1 study [NCT01957436]and the backdrop of understanding the [benefit of] adding an additional layer of intensification to doublet combination therapy have led to success across all the different agents for mCSPC.6 [The PEACE-1] study looked to push that envelope a little bit further by adding on a third agent. We know that prostate cancer has some intrinsic heterogeneity. There will be some early clones that are more inherently insensitive or resistant to ADT, or maybe AR-directed therapy, through mechanisms such as AR variants or alterations. Trying to have something in the chemotherapy category as well as the AR-directed category, investigators hoped to show that a triplet regimen would elevate survival on top of just ADT plus docetaxel alone.

PEACE-1 has an interesting study design; it evolved as the SOC evolved. It was initially designed as a large randomized study to test whether the addition of abiraterone or radiation therapy, or the combination of both, to the SOC of ADT would benefit men with hormone-sensitive prostate cancer. However, [the SOC changed] as data from the CHAARTED trial [NCT00309985] and the STAMPEDE trial [NCT00268476] came out showing the additional benefits of docetaxel.7,8 [PEACE-1] laid out a prespecified statistical plan that provides for primary efficacy analysis of survival with the addition of abiraterone to the docetaxel-treated population. This study was very positive, but more to our point, the triplet regimen focused on the population of patients who got both ADT and docetaxel as part of the SOC. The addition of abiraterone demonstrated significant improvement in OS with a hazard ratio of 0.75 [95% CI, 0.59-0.95; P = .017]. This positive effect on OS was primarily pronounced in the patients with high-volume disease. The addition of abiraterone to hormonal therapy [must be] seriously considered because it is linked to a survival benefit.

Agarwal: As a urologist, how do you choose between doublet and triplet therapy?

Lowentritt: The latest data indicate that only about 3% of [patients] were getting docetaxel for metastatic hormone-sensitive disease. Its hard to call that the SOC, so you would love to see what the results are. My focus now is on intensifying therapy up front for these patients with high-volume disease who can tolerate it. Even in the ARCHES and TITAN trials, you had patients who had received docetaxel and then were put on oral therapy. [Patients enrolled were] usually in the low double digits or high single digits in those trials, but they did well and there were prespecified analyses to look at those that did equally well. [Some] data suggest that patients dont do worse when we combine [treatments].

Now, its about finding those patients whom my medical oncology colleagues will treat and the ones who will tolerate [treatment] well. Patients are [presenting at a] younger age, with better performance status, and they undergo a more intensified treatment up front due to high-volume disease. We are seeing this increased incidence of high-volume de novo disease, likely linked to decreased screening in the United States in the last 5 to 10 years. Regardless, we were seeing that trend even before then, and this disease seems to be getting worse. I do think itll be about finding those patients who fit the criteria and can go through [docetaxel plus oral therapy] together. We do not give cytotoxic chemotherapy in our practice; we give all the other types of therapies. Its about the patients having a good discussion with us and then making sure they have access to the medical oncologist, who can have a discussion with them and give them more details. Thats how I have approached it.

Agarwal: How is the process different if the patient has low-volume disease but is still de novo?

Liaw: PEACE-1 also included patients with low-volume disease, and it also tried to determine what type of [patient] benefited from the triplet regimen. As of the most recent data analysis, [we have no conclusive answers] in terms of OS benefit for these low-volume patients. Further longitudinal follow-up may change that, but right now, not a lot of strong evidence shows that patients with low-volume disease would necessarily benefit from a triplet regimen. Im generally not going to convince patients with low-volume disease to take 3 different therapies at the same time. Some exceptions to that [exist,] because when we define high-volume disease, were looking at the CHAARTED criteria, which are primarily focused on a [particular] number of lesions. It doesnt get at the heart of the matter, which is the overall volume of disease. One tiny, half-millimeter bone lesion still counts as a bone lesion in the CHAARTED trial. If you have a 10-centimeter lymph node thats obstructing kidney function, thats still 1 lesion. CHAARTED doesnt fully accommodate some well-known poor prognostic features of disease, such as a Gleason score or height of PSA [bounce].

There are some gray areas here, [such as if a patient] has low-volume disease and large, obstructive lymph nodes that dont quite meet high-volume disease criteria. If theyre highly symptomatic and they need an immediate response, I think theres still room to make a case for much more aggressive triplet therapy. Does that mean that its necessarily better over a doublet? I dont have those data. I dont know that well have great randomized prospective studies to be able to demonstrate this soon. Most likely, what well have to do is come together as a medical community to understand what the real-world data tell us, to help inform some of these decision-making processes. For patients with low-volume disease, Im still mostly thinking about doublet therapies.

Agarwal: How do you approach genomic profiling?

Chowdhury: [We have] both germline genomic profiling and somatic genomic profiling. From the somatic genomic profiling [perspective], its difficult because many patients [with homologous recombination repair gene alteration] from the PROfound study [NCT02987543] failed the FoundationOne testing.9 A lot of the samples had insufficient DNA extraction, so we need to think about that. When we have a patient with metastatic disease, particularly poor-prognosis metastatic disease, we need to be getting the DNA from those patients to check whether they have somatic mutations or germline mutations. Those are patients who will need options going forward, potentially like a PARP inhibitor.

From the germline side, the [National Comprehensive Cancer Network] guidelines are good. We dont have [enough] geneticist capacity in the [United Kingdom] for every high-risk localizedpatient. One must be a bit more pragmatic and look at particularly high-risk [ancestry] groups, such as Ashkenazi Jews [and others] with a significant history of likely BRCA-related cancers, and [consider] education around that. Its an evolving field. Tests are evolving, and not all genes are equal. BRCA1, BRCA2, and ATM are different genes with different actionability. Not all mutations are equal, so a homozygous deletion is different than a missense. And not all tests are equal. We must be careful about tests that [identify] mutations that just arent actionable. A lot of work [remains] to be done there. Its an interesting area, and you and your group demonstrated with olaparib [Lynparza] that there is actionability that can benefit patients. These are exciting times, but the data are early.

Lowentritt: [My center] was a trial site for PROfound, but I got 0 patients enrolled because all my samples failed. It informed how I then started doing testing, and I am now getting fresher biopsies at diagnosis. Im getting an initial somatic workup because the tissue is fresh. [Genetic testing] is more likely to be successful, and we have more [available samples] so that we can [determine results easier]. If a prostatectomy [has been done], that makes [genetic testing] easier as well. Most patients who were diagnosing with de novo metastatic disease are going to just have a biopsy. Im [receiving tissue samples] upfront with the understanding that many of these [mutations] are currently actionable. My approach now is to test early and test often. We have to recognize that most of the information that we currently know is helpful is available at the beginning. Future discovery will be based on resistance mechanisms and other things that are developing over time, so we can test as patients progress. I have gone to a lot more testing in the de novo setting.

Go here to see the original:
Recap: Recent Advances in the Treatment of Metastatic Castration-Sensitive Prostate Cancer - Cancer Network

Recommendation and review posted by Bethany Smith

Australia has a reputation for living well through our fortuitous supplies of mineral wealth in demand globally.

But in recent years a new breed of entrepreneur has moved the country into the future with developments in the fields of medical and information technology, green tech and biotech.

The companies range from those in the venture capital and development stage to operators listed on the stockmarket that make profits for shareholders.

Just how big the startup sector has become in recent years is highlighted by Paul Naphtali, co-founder and partner of venture fund Rampersand.

Back in 2013 the industry might have been investing $300 million a year, he said.

In 2021 that had jumped to $10 billion in Australia.

Editor of industry newsletter Biotech Daily David Langsam said the sector is moving to a more commercial footing.

Now 15 of the top 20 biotech companies have sales and revenue. They arent necessarily revenue positive but they do have commercial revenue. That wasnt the case 15 years ago, Langsam said.

That growth is reflecting itself on the sharemarkets where the Pharmaceuticals and Biotechnology index has risen 13 times since September 2006, while the All Ordinaries index is up only 1.38 times in the same period.

One new operator is Eugene Labs, which brings genetic testing into the home for a range of conditions and uses.

CEO Kunal Kalro, who co-founded the group with medical scientist Zoe Milgrom, said they are inspired by the belief that great health starts with your genetics.

To that end they provide people with seamless and actionable genomic health care.

Eugene Labs provides people with the means to do a range of genetic testing in the home so they avoid the difficulty and time cost of going to hospital unnecessarily.

One test they offer is called carrier screening.

Its a test that is either done pre-pregnancy or in early pregnancy that helps people understand their risk of having a child with a serious genetic condition, Mr Kalro said.

Once people are tested they can make decisions with the backing of their genetic and health professionals on how to prevent the passing on of genetic disorders, if that is possible.

If that is not possible then management of health and genetic issues can be planned.

There are also cancer and heart health tests available with everything done at home.

If people want to use Eugene Labs services they register on the internet for a saliva collection kit, fill in a detailed health survey online and send in their specimen for processing.

Eugene Labs sends these specimens to the appropriate labs for testing.

We are not a laboratory, Mr Kalro said.

However, It interpretS lab results into understandable language for its users.

Eugene Labs automated processes mean that instead of a clinic seeing 3000 people a year we can see 300,000 a year. That is because people do the testing at home.

The business began in 2019 and is still being funded by investors who want to support its fast, early stage growth.

We could reach profitability in 24 months, but we want to grow harder so we go out and raise more money to fund that, Mr Kalro said.

Another group in the medical field is Imugene, an ASX-listed company, which is developing a range of immunotherapy technologies that allow the body to track down and fight off cancer cells.

The companys most recent breakthrough is a treatment known as oncolytic virus therapy.

This involves a natural virus being genetically modified to enter cancer cells and replicate itself.

The virus kills cancer cells while avoiding damage to healthy cells. It means that the cancer is treated with very limited side effects for the patient undergoing treatment.

This treatment, along with some earlier developments, the company believes, can also help prime peoples immune systems against cancer, which will in turn make it more difficult for cancers to get to first base in the body.

Cancers being tested with the companys treatments include lung, pancreatic, colon and colorectal.

Testing is progressing, but the company is a long way from seeing cashflows.

In the June year just gone it lost $37.8 million, a reflection of the big spends on clinical trials and research necessary to bring new medical technologies to market.

But founder and executive chairman Paul Hopper described the company as in an enviable position financially with a long cash runway.

That means investors are still putting up cash to keep the wheels turning. It raised $95 million last financial year and another $80 million on September 9.

CEO Leslie Chong said the latest funding round will give ourselves an unimpeded runway to progress the numerous clinical trials that we have ongoing.

Eventually that will translate into shareholder value and improved patient outcomes, she said.

Although there are still people prepared to put up cash to fund the group, a shakeout in the biotech sector has hit it hard.

Imugene is currently valued at $1.5 billion on the stockmarket, but it was worth $2.5 billion a year ago.

See the rest here:
Australia moves to future in biotech and medical technologies with billions in new investment - The New Daily

Recommendation and review posted by Bethany Smith

Find out who can be a stem cell or bone marrow donor, and how to register.

A stem cell or bone marrow transplant is an important treatment for some people with types of blood cancer such as leukaemia, lymphoma and myeloma.

A transplant allows you to have high doses of chemotherapy and other treatments. The stem cellsare collected from the bloodstream or the bone marrow.Peoplehave a transplant either:

To be a donor you need to have stem cells that match the person you are donating to. To find this out, you have a blood test to look at HLA typing or tissue typing.

Staff in the laboratory look at the surface of your blood cells. They compare them to the surface of the blood cells of the person needing a transplant.

Everyone has their own set of proteins on the surface of their blood cells. The laboratory staff look for proteins called HLA markers and histocompatibility antigens. They check for 10 HLA markers. The result of this test shows how good the HLA match is between you and the person who needs the cells.

Abrother or sisteris most likely to be a match. There is a 1 in 4 chance of your cells matching.This is called a matched related donor (MRD) transplant.Anyone else in the family is unlikely to match. This can be very frustrating for relatives who are keen to help.

Sometimes if your cells are a half (50%) match, you might still be able to donate stem cells or bone marrow to a relative. This is called a haploidentical transplant.

You can't donate stem cells or bone marrow to your relative if you're not a match.

It's sometimes possible to get a match from someoneoutside of the family. This is calleda matched unrelated donor. To find a matched unrelated donor, it'susually necessary to search large numbers of people whose tissue type has been tested. So doctorssearch national and international registers to try to find a match for your relative.

Even if you can't donate to your relative, you might be ableto become a donor for someone else. You can do this by contacting one of the UK registers.

There are different donor registersin the UK.These work with each otherand with international registersto match donors with people who need stem cells. This helps doctors find donors for their patients as quickly as possiblefrom anywhere in the world.

Each registry has specific health criteriaand listmedical conditions that mightpreventyou from donating. Check their websitefor this information. Once registered, the organisation will contactyou if you are a match for someone who needs stem cells or bone marrow.

British Bone Marrow Registry (BBMR)

To register with the BBMR, you mustbe a blood donor. BBMR would like toregister those groups they are particularly short of ontheir register.This includes men between the ages of 17 and 40. And womenaged between 17 and 40 who are from Black, Asian, and minority ethnicities and mixed ethnicity backgrounds.

You have a blood test for tissue typing. Your details are kept on file until you are 60.

Anthony Nolan

You must be aged between 16 and 30 to register with Anthony Nolan. You have a cheek swab to test fortissue typing. Your details are kept on the register until you are 60.

Welsh Bone Marrow Donor Registry

You must be aged between 17 and 30 and your details are kept on the register until you are 60. You have a blood test for tissue typing.

DKMS

To register you must be aged between 17 and 55. You havea cheek swab for tissue typing. Your details stay on the register until your61st birthday.

This page is due for review. We will update this as soon as possible.

See more here:
Who can donate stem cells or bone marrow? - Cancer Research UK

Recommendation and review posted by Bethany Smith

Bone marrow is the spongy tissue inside some of the bones in the body, including the hip and thigh bones. Bone marrow contains immature cells called stem cells.

Many people with blood cancers, such as leukemia and lymphoma, sickle cell anemia, and other life threatening conditions rely on bone marrow or cord blood transplants to survive.

People need healthy bone marrow and blood cells to live. When a condition or disease affects bone marrow so that it can no longer function effectively, a marrow or cord blood transplant could be the best treatment option. For some people, it may be the only option.

This article looks at everything there is to know about bone marrow.

Bone marrow is soft, gelatinous tissue that fills the medullary cavities, or the centers of bones. The two types of bone marrow are red bone marrow, known as myeloid tissue, and yellow bone marrow, known as fatty tissue.

Both types of bone marrow are enriched with blood vessels and capillaries.

Bone marrow makes more than 220 billion new blood cells every day. Most blood cells in the body develop from cells in the bone marrow.

Bone marrow contains two types of stem cells: mesenchymal and hematopoietic.

Red bone marrow consists of a delicate, highly vascular fibrous tissue containing hematopoietic stem cells. These are blood-forming stem cells.

Yellow bone marrow contains mesenchymal stem cells, or marrow stromal cells. These produce fat, cartilage, and bone.

Stem cells are immature cells that can turn into a number of different types of cells.

Hematopoietic stem cells in the bone marrow give rise to two main types of cells: myeloid and lymphoid lineages. These include monocytes, macrophages, neutrophils, basophils, eosinophils, erythrocytes, dendritic cells, and megakaryocytes, or platelets, as well as T cells, B cells, and natural killer (NK) cells.

The different types of hematopoietic stem cells vary in their regenerative capacity and potency. They can be multipotent, oligopotent, or unipotent, depending on how many types of cells they can create.

Pluripotent hematopoietic stem cells have renewal and differentiation properties. They can reproduce another cell identical to themselves, and they can generate one or more subsets of more mature cells.

The process of developing different blood cells from these pluripotent stem cells is known as hematopoiesis. It is these stem cells that are needed in bone marrow transplants.

Stem cells constantly divide and produce new cells. Some new cells remain as stem cells, while others go through a series of maturing stages, as precursor or blast cells, before becoming formed, or mature, blood cells. Stem cells rapidly multiply to make millions of blood cells each day.

Blood cells have a limited life span. This is around 120 days for red blood cells. The body is constantly replacing them. The production of healthy stem cells is vital.

The blood vessels act as a barrier to prevent immature blood cells from leaving bone marrow.

Only mature blood cells contain the membrane proteins required to attach to and pass through the blood vessel endothelium. Hematopoietic stem cells can cross the bone marrow barrier, however. Healthcare professionals may harvest these from peripheral, or circulating, blood.

The blood-forming stem cells in red bone marrow can multiply and mature into three significant types of blood cells, each with its own job:

Once mature, these blood cells move from bone marrow into the bloodstream, where they perform important functions that keep the body alive and healthy.

Mesenchymal stem cells are present in the bone marrow cavity. They can differentiate into a number of stromal lineages, such as:

Red bone marrow produces all red blood cells and platelets and around 6070% of lymphocytes in human adults. Other lymphocytes begin life in red bone marrow and become fully formed in the lymphatic tissues, including the thymus, spleen, and lymph nodes.

Together with the liver and spleen, red bone marrow also plays a role in getting rid of old red blood cells.

Yellow bone marrow mainly acts as a store for fats. It helps provide sustenance and maintain the correct environment for the bone to function. However, under particular conditions such as with severe blood loss or during a fever yellow bone marrow may revert to red bone marrow.

Yellow bone marrow tends to be located in the central cavities of long bones and is generally surrounded by a layer of red bone marrow with long trabeculae (beam-like structures) within a sponge-like reticular framework.

Before birth but toward the end of fetal development, bone marrow first develops in the clavicle. It becomes active about 3 weeks later. Bone marrow takes over from the liver as the major hematopoietic organ at 3236 weeks gestation.

Bone marrow remains red until around the age of 7 years, as the need for new continuous blood formation is high. As the body ages, it gradually replaces the red bone marrow with yellow fat tissue. Adults have an average of about 2.6 kilograms (kg) (5.7 pounds) of bone marrow, about half of which is red.

In adults, the highest concentration of red bone marrow is in the bones of the vertebrae, hips (ilium), breastbone (sternum), ribs, and skull, as well as at the metaphyseal and epiphyseal ends of the long bones of the arm (humerus) and leg (femur and tibia).

All other cancellous, or spongy, bones and central cavities of the long bones are filled with yellow bone marrow.

Most red blood cells, platelets, and most white blood cells form in the red bone marrow. Yellow bone marrow produces fat, cartilage, and bone.

White blood cells survive from a few hours to a few days, platelets for about 10 days, and red blood cells for about 120 days. Bone marrow needs to replace these cells constantly, as each blood cell has a set life expectancy.

Certain conditions may trigger additional production of blood cells. This may happen when the oxygen content of body tissues is low, if there is loss of blood or anemia, or if the number of red blood cells decreases. If these things happen, the kidneys produce and release erythropoietin, which is a hormone that stimulates bone marrow to produce more red blood cells.

Bone marrow also produces and releases more white blood cells in response to infections and more platelets in response to bleeding. If a person experiences serious blood loss, yellow bone marrow can activate and transform into red bone marrow.

Healthy bone marrow is important for a range of systems and activities.

The circulatory system touches every organ and system in the body. It involves a number of different cells with a variety of functions. Red blood cells transport oxygen to cells and tissues, platelets travel in the blood to help clotting after injury, and white blood cells travel to sites of infection or injury.

Hemoglobin is the protein in red blood cells that gives them their color. It collects oxygen in the lungs, transports it in the red blood cells, and releases oxygen to tissues such as the heart, muscles, and brain. Hemoglobin also removes carbon dioxide (CO2), which is a waste product of respiration, and sends it back to the lungs for exhalation.

Iron is an important nutrient for human physiology. It combines with protein to make the hemoglobin in red blood cells and is essential for producing red blood cells (erythropoiesis). The body stores iron in the liver, spleen, and bone marrow. Most of the iron a person needs each day for making hemoglobin comes from the recycling of old red blood cells.

The production of red blood cells is called erythropoiesis. It takes about 7 days for a committed stem cell to mature into a fully functional red blood cell. As red blood cells age, they become less active and more fragile.

White blood cells called macrophages remove aging red cells in a process known as phagocytosis. The contents of these cells are released into the blood. The iron released in this process travels either to bone marrow for the production of new red blood cells or to the liver or other tissues for storage.

Typically, the body replaces around 1% of its total red blood cell count every day. In a healthy person, this means that the body produces around 200 billion red blood cells each day.

Bone marrow produces many types of white blood cells. These are necessary for a healthy immune system. They prevent and fight infections.

The main types of white blood cells, or leukocytes, are as follows.

Lymphocytes are produced in bone marrow. They make natural antibodies to fight infection due to viruses that enter the body through the nose, mouth, or another mucous membrane or through cuts and grazes. Specific cells recognize the presence of invaders (antigens) that enter the body and send a signal to other cells to attack them.

The number of lymphocytes increases in response to these invasions. There are two major types of lymphocytes: B and T lymphocytes.

Monocytes are produced in bone marrow. Mature monocytes have a life expectancy in the blood of only 38 hours, but when they move into the tissues, they mature into larger cells called macrophages.

Macrophages can survive in the tissues for long periods of time, where they engulf and destroy bacteria, some fungi, dead cells, and other material that is foreign to the body.

Granulocytes is the collective name given to three types of white blood cells: neutrophils, eosinophils, and basophils. The development of a granulocyte may take 2 weeks, but this time reduces when there is an increased threat, such as a bacterial infection.

Bone marrow stores a large reserve of mature granulocytes. For every granulocyte circulating in the blood, there may be 50100 cells waiting in the bone marrow to be released into the bloodstream. As a result, half the granulocytes in the bloodstream can be available to actively fight an infection in the body within 7 hours of it detecting one.

Once a granulocyte has left the blood, it does not usually return. A granulocyte may survive in the tissues for up to 45 days, depending on the conditions, but it can only survive for a few hours in circulating blood.

Neutrophils are the most common type of granulocyte. They can attack and destroy bacteria and viruses.

Eosinophils are involved in the fight against many types of parasitic infections and against the larvae of parasitic worms and other organisms. They are also involved in some allergic reactions.

Basophils are the least common of the white blood cells. They respond to various allergens that cause the release of histamines, heparin, and other substances.

Heparin is an anticoagulant. It prevents blood from clotting. Histamines are vasodilators that cause irritation and inflammation. Releasing these substances makes a pathogen more permeable and allows for white blood cells and proteins to enter the tissues to engage the pathogen.

The irritation and inflammation in tissues that allergens affect are parts of the reaction associated with hay fever, some forms of asthma, hives, and, in its most serious form, anaphylactic shock.

Bone marrow produces platelets in a process known as thrombopoiesis. Platelets are necessary for blood to coagulate and for clots to form in order to stop bleeding.

Sudden blood loss triggers platelet activity at the site of an injury or wound. Here, the platelets clump together and combine with other substances to form fibrin. Fibrin has a thread-like structure and forms an external scab or clot.

Platelet deficiency causes the body to bruise and bleed more easily. Blood may not clot well at an open wound, and there may be a higher risk of internal bleeding if the platelet count is very low.

The lymphatic system consists of lymphatic organs such as bone marrow, the tonsils, the thymus, the spleen, and lymph nodes.

All lymphocytes develop in bone marrow from immature cells called stem cells. Lymphocytes that mature in the thymus gland (behind the breastbone) are called T cells. Those that mature in bone marrow or the lymphatic organs are called B cells.

The immune system protects the body from disease. It kills unwanted microorganisms such as bacteria and viruses that may invade the body.

Small glands called lymph nodes are located throughout the body. Once lymphocytes are made in bone marrow, they travel to the lymph nodes. The lymphocytes can then travel between each node through lymphatic channels that meet at large drainage ducts that empty into a blood vessel. Lymphocytes enter the blood through these ducts.

Three major types of lymphocytes play an important part in the immune system: B lymphocytes, T lymphocytes, and NK cells.

These cells originate from hematopoietic stem cells in bone marrow in mammals.

B cells express B cell receptors on their surface. These allow the cell to attach to an antigen on the surface of an invading microbe or another antigenic agent.

For this reason, B cells are known as antigen-presenting cells, as they alert other cells of the immune system to the presence of an invading microbe.

B cells also secrete antibodies that attach to the surface of infection-causing microbes. These antibodies are Y-shaped, and each one is akin to a specialized lock into which a matching antigen key fits. Because of this, each Y-shaped antibody reacts to a different microbe, triggering a larger immune system response to fight infection.

In some circumstances, B cells erroneously identify healthy cells as being antigens that require an immune system response. This is the mechanism behind the development of autoimmune conditions such as multiple sclerosis, scleroderma, and type 1 diabetes.

These cells are so-called because they mature in the thymus, which is a small organ in the upper chest, just behind the sternum. (Some T cells mature in the tonsils.)

There are many different types of T cells, and they perform a range of functions as part of adaptive cell-mediated immunity. T cells help B cells make antibodies against invading bacteria, viruses, or other microbes.

Unlike B cells, some T cells engulf and destroy pathogens directly after binding to the antigen on the surface of the microbe.

NK T cells, not to be confused with NK cells of the innate immune system, bridge the adaptive and innate immune systems. NK T cells recognize antigens presented in a different way from many other antigens, and they can perform the functions of T helper cells and cytotoxic T cells. They can also recognize and eliminate some tumor cells.

These are a type of lymphocyte that directly attack cells that a virus has infected.

A bone marrow transplant is useful for various reasons. For example:

Stem cells mainly occur in four places:

Stem cells for transplantation are obtainable from any of these except the fetus.

Hematopoietic stem cell transplantation (HSCT) involves the intravenous (IV) infusion of stem cells collected from bone marrow, peripheral blood, or umbilical cord blood.

This is useful for reestablishing hematopoietic function in people whose bone marrow or immune system is damaged or defective.

Worldwide, more than 50,000 first HSCT procedures, 28,000 autologous transplantation procedures, and 21,000 allogeneic transplantation procedures take place every year. This is according to a 2015 report by the Worldwide Network for Blood and Marrow Transplantation.

This number continues to increase by over 7% annually. Reductions in organ damage, infection, and severe, acute graft-versus-host disease (GVHD) seem to be contributing to improved outcomes.

In a study of 854 people who survived at least 2 years after autologous HSCT for hematologic malignancy, 68.8% were still alive 10 years after transplantation.

Bone marrow transplants are the leading treatment option for conditions that threaten bone marrows ability to function, such as leukemia.

A transplant can help rebuild the bodys capacity to produce blood cells and bring their numbers to acceptable levels. Conditions that may be treatable with a bone marrow transplant include both cancerous and noncancerous diseases.

Cancerous diseases may or may not specifically involve blood cells, but cancer treatment can destroy the bodys ability to manufacture new blood cells.

A person with cancer usually undergoes chemotherapy before transplantation. This eliminates the compromised marrow.

A healthcare professional then harvests the bone marrow of a matching donor which, in many cases, is a close family member and ready it for transplant.

Types of bone marrow transplant include:

A persons tissue type is defined as the type of HLA they have on the surface of most of the cells in their body. HLA is a protein, or marker, that the body uses to help it determine whether or not the cell belongs to the body.

To check if the tissue type is compatible, doctors assess how many proteins match on the surface of the donors and recipients blood cells. There are millions of different tissue types, but some are more common than others.

Tissue type is inherited, and types pass on from each parent. This means that a relative is more likely to have a matching tissue type.

However, if it is not possible to find a suitable bone marrow donor among family members, healthcare professionals try to find someone with a compatible tissue type on the bone marrow donor register.

Healthcare professionals perform several tests before a bone marrow transplant to identify any potential problems.

These tests include:

In addition, a person needs a complete dental exam before a bone marrow transplant to reduce the risk of infection. Other precautions to lower the risk of infection are also necessary before the transplant.

Bone marrow is obtainable for examination by bone marrow biopsy and bone marrow aspiration.

Bone marrow harvesting has become a relatively routine procedure. Healthcare professionals generally aspirate it from the posterior iliac crests while the donor is under either regional or general anesthesia.

Healthcare professionals can also take it from the sternum or from the upper tibia in children, as it still contains a substantial amount of red bone marrow.

To do so, they insert a needle into the bone, usually in the hip, and withdraw some bone marrow. They then freeze and store this bone marrow.

National Marrow Donor Program (NMDP) guidelines limit the volume of removable bone marrow to 20 milliliters (ml) per kg of donor weight. A dose of 1 x 103 and 2 x 108 marrow mononuclear cells per kg is necessary to establish engraftment in autologous and allogeneic marrow transplants, respectively.

Complications related to bone marrow harvesting are rare. When they do occur, they typically involve problems related to anesthetics, infection, and bleeding.

Go here to read the rest:
Bone marrow: Function, diseases, transplants, and donation

Recommendation and review posted by Bethany Smith

Home Blog Bone Marrow Stem Cell Dose Matters in Knee Osteoarthritis

If theres one overarching theme in orthobiologics that I have been discussing for almost two decades, its that measuring and delivering higher doses are critical for success. Despite this, 99% of physicians who offer these procedures dont know what dose theyre delivering and use bedside kits that can only achieve low doses. Today well go into our most recent publication that shows that the stem cell dose in bone marrow concentrate is directly tied to clinical outcomes in knee arthritis patients. Lets dig in.

Our study looked at the number of colony-forming mesenchymal stem cells (CFU-fs) in bone marrow concentrate (BMC) in knee arthritis patients and then the clinical outcome of the procedure (1). We found that those patients who had more stem cells in their BMC reported better outcomes. That fits with data published by others on BMC treatments in bone disease and low back degenerative disc disease (2,3).

While this may seem like a mundane finding, its the first of its kind in BMC used for knee osteoarthritis treatment. More importantly, it highlights how important dose is in these treatments and how many BMC treatments being delivered are likely under-dosing patients. Lets dive deeper into that concept.

77 clinic locations offering non-surgical Regenexx solutions for musculoskeletal pain.

77 clinic locations offering non-surgical Regenexx solutions for musculoskeletal pain.

Youre a doctor who just started dipping his toe into the waters of this new field called orthobiologics. You buy a simple bedside kit to produce PRP because its super easy without much commitment. You then at some point add in bone marrow concentrate through the same system with a different kit. Your world is easy and simple, as all your staff needs to know is where to put the kit in the machine and where the On button is located.

However, what you begin to realize after a few years is that all of this simplicity comes at a steep price. For example, you have no idea of the dose of orthobiologic youre delivering. At its most basic, everything else in medicine is tied to a dose, so this seems wrong. In addition, independent research shows that the dose that your simple machine is capable of delivering is low and that higher doses are tied to better outcomes. Hence, at some point, it hits you like one of those clown pies in the face, youve traded simplicity for your staff for poorer patient outcomes.

PRP (Platelet-Rich Plasma) and BMC (Bone Marrow Concentrate) are autologous procedures where the dose of platelets or cells varies widely from patient to patient. This is based on many factors including:

There are other factors that also influence outcomes like where this stuff is injected and how, but today well focus only on how the dose of whats injected can dramatically change how the patient responds.

The machine the doctor buys to produce PRP and BMC matters. The problem is that this decision is often based on a relationship with a sales rep and not the concept of dose as were discussing here. Lets dig in.

Ive blogged a few times on researchers who recently published big and well-done studies, but that used commercial kits that claim to produce PRP, but instead only produce plasma which has fewer than 2 times concentrated platelets (the minimum needed to call the product PRP). These kits are are Arthrex ACP and RegenLab (7). Hence, if you were a doctor who happened to purchase one of these systems and are using this stuff, you think youre delivering PRP, but youre not.

The vast majority of machines produce low-dose PRP at a 3-5X concentration. The good news is that if youre treating young patients this is fine, but as our long-standing research on mesenchymal stem cells in culture and published work on tenocyte healing shows, for older patients this concentration represents a severe under-dose (8). Meaning that if youre middle-aged or older, the higher the dose the better, because your older cells (unlike young ones) will respond to the extra platelets. Given that this is a direct dose-response relationship in these patients, your dose cant be too high in this age group.

High-dose PRP is 7-14X with most older patients needing 10-14X or higher. Few machines can achieve this and all have trade-offs. Take the Arthrex Angel device, which can produce high-dose PRP, but at a price. Rather than producing the more commonly used leukocyte-poor PRP (LP), this machine concentrates white blood cells with platelets, so instead you get bloody and leukocyte-rich PRP (LR). Or other machines that use an off-label double spin technique where the doctor uses the same kit twice. These machines like Emcyte can get to higher concentrations, but as we have seen testing this machine in our lab, the double spin can cause the platelets to clump, distributing them unevenly in the PRP. In addition, no research or FDA clearance is available on using the kit twice, so the reliability of that double spin product is unknown.

Weve never used any of these machines because we can produce any concentration of PRP in the lab that the doctor requires and make it leukocyte poor or rich. We can also produce it from peripheral blood or a bone marrow draw if thats already being done. Whats the downside? This approach takes a bigger commitment from the practice, meaning they have to be all in on orthobiologics.

For BMC, we have seen similar issues with bedside machines. Meaning as we have tested these machines in our lab, their ability to concentrate and get the most stem cells in the smallest volume is limited. The biggest issue is the simple lack of flexibility of the input volume and a higher output volume. What does that mean?

In trying to maximize the number of stem cells in a BMC sample, you first need to be able to increase the volume of high-quality marrow aspirate taken from the patient. That starts with taking a small volume of marrow aspirate from many sites, which maximizes the number of stem cells in the sample (2-4). Regrettably, we still see physicians short-changing patients by taking one large marrow pull from the patient, which dramatically reduces the number of stem cells taken from the patient.

Next, you need the flexibility to increase the marrow aspirate volume based on the age of the patient and the number of areas treated. For example, in an older patient who may have fewer stem cells per ml of BMA, just take more BMA to compensate. This really cant happen with bedside centrifuge kits, as they have a fixed input volume. That means that you only get one option on how much marrow can be processed. Compare that to a flexible lab-based system where you easily increase the volume processed to compensate for the clinical scenario.

Finally, the output volume is critical as well. Meaning, that if you take more BMA to get more stem cells, thats useless if your system gives you a single large volume of BMC to inject. Instead, you need the highest concentration possible from your large volume and that means that the system youre using puts all of those cells in the smallest possible volume. As an example, using a lab-based system, we often take 120 ml of BMA and get that down to 3-5 ml of BMC.

Once you leave the orthobiologic training wheels behind and get a significant number of treated patients completed, whats next? Based on the existing and emerging research, thats making sure that you can deliver the highestPRP and BMC dose possible. That means leaving the bedside kit world and transitioning to a lab. No company on earth has more experience than Regenexx helping providers graduate to a flexible lab platform safely and efficiently with strict SOPs and controls.

The upshot? Dose matters. The research continues to show that the providers who can maximize the dose of platelets and stem cells are likely getting better results than those who have maximized their convenience by using limited bedside kits. Is your practice ready for an upgrade? Is it time to leave the orthobiologic training wheels behind? If so, we got you covered.

_______________________________________________________________

References:

(1) Centeno CJ, Berger DR, Money BT, Dodson E, Urbanek CW, Steinmetz NJ. Percutaneous autologous bone marrow concentrate for knee osteoarthritis: patient-reported outcomes and progenitor cell content. Int Orthop. 2022 Aug 6. doi: 10.1007/s00264-022-05524-9. Epub ahead of print. PMID: 35932306.

(2)Pettine KA, Murphy MB, Suzuki RK, Sand TT. Percutaneous injection of autologous bone marrow concentrate cells significantly reduces lumbar discogenic pain through 12 months. Stem Cells. 2015 Jan;33(1):146-56. doi: 10.1002/stem.1845. PMID: 25187512.

(3) Hernigou P, Beaujean F. Treatment of osteonecrosis with autologous bone marrow grafting. Clin Orthop Relat Res. 2002 Dec;(405):14-23. doi: 10.1097/00003086-200212000-00003. PMID: 12461352.

(4) Batini D, Marusi M, Pavleti Z, Bogdani V, Uzarevi B, Nemet D, Labar B. Relationship between differing volumes of bone marrow aspirates and their cellular composition. Bone Marrow Transplant. 1990 Aug;6(2):103-7. PMID: 2207448.

(5) Muschler GF, Boehm C, Easley K. Aspiration to obtain osteoblast progenitor cells from human bone marrow: the influence of aspiration volume. J Bone Joint Surg Am. 1997 Nov;79(11):1699-709. doi: 10.2106/00004623-199711000-00012. Erratum in: J Bone Joint Surg Am 1998 Feb;80(2):302. PMID: 9384430.

(6) Fennema EM, Renard AJ, Leusink A, van Blitterswijk CA, de Boer J. The effect of bone marrow aspiration strategy on the yield and quality of human mesenchymal stem cells. Acta Orthop. 2009 Oct;80(5):618-21. doi: 10.3109/17453670903278241. PMID: 19916699; PMCID: PMC2823327.

(7) Magalon J, Bausset O, Serratrice N, Giraudo L, Aboudou H, Veran J, Magalon G, Dignat-Georges F, Sabatier F. Characterization and comparison of 5 platelet-rich plasma preparations in a single-donor model. Arthroscopy. 2014 May;30(5):629-38. doi: 10.1016/j.arthro.2014.02.020. PMID: 24725317.

(8) Berger DR, Centeno CJ, Steinmetz NJ. Platelet lysates from aged donors promote human tenocyte proliferation and migration in a concentration-dependent manner. Bone Joint Res. 2019 Feb 2;8(1):32-40. doi: 10.1302/2046-3758.81.BJR-2018-0164.R1. PMID: 30800297; PMCID: PMC6359887.

If you have questions or comments about this blog post, please email us at [emailprotected]

NOTE: This blog post provides general information to help the reader better understand regenerative medicine, musculoskeletal health, and related subjects. All content provided in this blog, website, or any linked materials, including text, graphics, images, patient profiles, outcomes, and information, are not intended and should not be considered or used as a substitute for medical advice, diagnosis, or treatment. Please always consult with a professional and certified healthcare provider to discuss if a treatment is right for you.

Join our free newsletter.

Join the Regenexx Newsletter

See the rest here:
Bone Marrow Stem Cell Dose Matters in Knee Osteoarthritis

Recommendation and review posted by Bethany Smith

Xu, Z. L. et al. Haploidentical hematopoietic stem cell transplantation for paediatric high-risk T-cell acute lymphoblastic leukaemia. Pediatr. Transplant. 20(4), 572580 (2016).

PubMed Google Scholar

Kogon, A. & Hingorani, S. Acute kidney injury in hematopoietic cell transplantation. Semin. Nephrol. 30(6), 615626 (2010).

PubMed PubMed Central Google Scholar

Piana, J. L. et al. Study of kidney function impairment after reduced-intensity conditioning allogeneic hematopoietic stem cell transplantation. A single-center experience. Biol. Blood Marrow Transplant. 15(1), 2129 (2009).

PubMed Google Scholar

Lopes, J. A. et al. Contemporary analysis of the influence of acute kidney injury after reduced intensity conditioning haematopoietic cell transplantation on long-term survival. Bone Marrow Transplant. 42(9), 619626 (2008).

CAS PubMed Google Scholar

Parikh, C. R. & Coca, S. G. Acute renal failure in hematopoietic cell transplantation. Kidney Int. 69(3), 430435 (2006).

CAS PubMed Google Scholar

Abramson, M. et al. Acute kidney injury in the modern era of allogeneic hematopoietic stem cell transplantation. Clin. J. Am. Soc. Nephrol. 16, 13181327 (2021).

CAS PubMed PubMed Central Google Scholar

Matsuoka, D. et al. Impact of acute kidney injury on overall survival in children and young adults undergoing allogeneic hematopoietic stem cell transplantation. Pediatr. Blood Cancer 68(9), e29167 (2021).

CAS PubMed Google Scholar

Kemmner, S., Verbeek, M. & Heemann, U. Renal dysfunction following bone marrow transplantation. J. Nephrol. 30(2), 201209 (2017).

CAS PubMed Google Scholar

Hu, X. et al. A clinical prediction model identifies a subgroup with inferior survival within intermediate risk acute myeloid leukemia. J. Cancer 12(16), 49124923 (2021).

CAS PubMed PubMed Central Google Scholar

Qi, J. et al. HLA-DQB1 mismatch increase risk of severe bleeding independently in recipients of allogeneic stem cell transplant. Ann. Hematol. 100, 23512361 (2021).

CAS PubMed Google Scholar

Hou, Z. L. et al. Pleural effusion-based nomogram to predict outcomes in unselected patients with multiple myeloma: A large single center experience. Ann. Hematol. 100(7), 17891801 (2021).

CAS PubMed Google Scholar

Li, S. et al. Procalcitonin and C-reactive protein predict infection in hematopoietic stem cell transplantation patients. Leuk. Res. 105, 106574 (2021).

CAS PubMed Google Scholar

Glucksberg, H. et al. Clinical manifestations of graft-versus-host disease in human recipients of marrow from HL-A-matched sibling donors. Transplantation 18(4), 295304 (1974).

CAS PubMed Google Scholar

Przepiorka, D. et al. 1994 consensus conference on acute GVHD grading. Bone Marrow Transplant. 15(6), 825828 (1995).

CAS PubMed Google Scholar

Li, M. et al. Unilateral limited laminectomy for debridement to treat localized short-segment lumbosacral spinal tuberculosis: A retrospective case series. Orthop. Surg. 13(4), 11701180 (2021).

PubMed PubMed Central Google Scholar

Cohen, E. P. Renal failure after bone-marrow transplantation. Lancet 357(9249), 67 (2001).

CAS PubMed Google Scholar

Nol, C. et al. Renal failure and bone marrow transplantation. Nephrol. Dial Transplant. 13(10), 24642466 (1998).

PubMed Google Scholar

Kersting, S. et al. Acute renal failure after allogeneic myeloablative stem cell transplantation: Retrospective analysis of incidence, risk factors and survival. Bone Marrow Transplant. 39(6), 359365 (2007).

CAS PubMed Google Scholar

Perazella, M. A. Crystal-induced acute renal failure. Am. J. Med. 106(4), 459465 (1999).

CAS PubMed Google Scholar

Hirano, D. et al. Independent risk factors and long-term outcomes for acute kidney injury in pediatric patients undergoing hematopoietic stem cell transplantation: A retrospective cohort study. BMC Nephrol. 21(1), 373 (2020).

CAS PubMed PubMed Central Google Scholar

Kizilbash, S. J. et al. Acute kidney injury and the risk of mortality in children undergoing hematopoietic stem cell transplantation. Biol. Blood Marrow Transplant. 22(7), 12641270 (2016).

PubMed PubMed Central Google Scholar

Didsbury, M. S., Mackie, F. E. & Kennedy, S. E. A systematic review of acute kidney injury in pediatric allogeneic hematopoietic stem cell recipients. Pediatr. Transplant. 19(5), 460470 (2015).

PubMed Google Scholar

Yu, Z. et al. Risk factors for acute kidney injury in patients undergoing allogeneic hematopoietic stem cell transplantation. Chin. J. Cancer 29(11), 946951 (2010).

CAS PubMed Google Scholar

Li, J. M. et al. Separating graft-versus-leukemia from graft-versus-host disease in allogeneic hematopoietic stem cell transplantation. Immunotherapy 1(4), 599621 (2009).

CAS PubMed Google Scholar

Laughlin, M. J. et al. Outcomes after transplantation of cord blood or bone marrow from unrelated donors in adults with leukemia. N. Engl. J. Med. 351(22), 22652275 (2004).

CAS PubMed Google Scholar

Shaw, P. J. et al. Outcomes of pediatric bone marrow transplantation for leukemia and myelodysplasia using matched sibling, mismatched related, or matched unrelated donors. Blood 116(19), 40074015 (2010).

CAS PubMed PubMed Central Google Scholar

Koh, K. N. et al. Favorable outcomes after allogeneic hematopoietic stem cell transplantation in children with high-risk or advanced acute myeloid leukemia. J. Pediatr. Hematol. Oncol. 33(4), 281288 (2011).

PubMed Google Scholar

Mogul, M. J. Unrelated cord blood transplantation vs matched unrelated donor bone marrow transplantation: The risks and benefits of each choice. Bone Marrow Transplant. 25(Suppl 2), S58-60 (2000).

PubMed Google Scholar

Parody, R. et al. Severe infections after unrelated donor allogeneic hematopoietic stem cell transplantation in adults: Comparison of cord blood transplantation with peripheral blood and bone marrow transplantation. Biol. Blood Marrow Transplant. 12(7), 734748 (2006).

PubMed Google Scholar

Radermacher, J. et al. Pronounced renal vasoconstriction and systemic hypertension in renal transplant patients treated with cyclosporin A versus FK 506. Transpl. Int. 11(1), 310 (1998).

CAS PubMed Google Scholar

Oyen, O. et al. Calcineurin inhibitor-free immunosuppression in renal allograft recipients with thrombotic microangiopathy/hemolytic uremic syndrome. Am. J. Transplant. 6(2), 412418 (2006).

CAS PubMed Google Scholar

Karimzadeh, I. et al. The pattern of cyclosporine nephrotoxicity and urinary kidney injury molecule 1 in allogenic hematopoietic stem cell transplant patients. Exp. Clin. Transplant. 19(6), 553562 (2021).

PubMed Google Scholar

Molema, G. et al. Renal microvascular endothelial cell responses in sepsis-induced acute kidney injury. Nat. Rev. Nephrol. 18, 95112 (2022).

CAS PubMed Google Scholar

Schrier, R. W. & Wang, W. Acute renal failure and sepsis. N. Engl. J. Med. 351(2), 159169 (2004).

CAS PubMed Google Scholar

Raghavan, R. & Shawar, S. Mechanisms of drug-induced interstitial nephritis. Adv. Chronic Kidney Dis. 24(2), 6471 (2017).

PubMed Google Scholar

Liu, H. et al. A multicenter, retrospective study of acute kidney injury in adult patients with nonmyeloablative hematopoietic SCT. Bone Marrow Transplant. 45(1), 153158 (2010).

CAS PubMed Google Scholar

Ciurea, S. et al. The European Society for Blood and Marrow Transplantation (EBMT) consensus recommendations for donor selection in haploidentical hematopoietic cell transplantation. Bone Marrow Transplant. 55(1), 1224 (2020).

CAS PubMed Google Scholar

Kagoya, Y. et al. Pretransplant predictors and posttransplant sequels of acute kidney injury after allogeneic stem cell transplantation. Biol. Blood Marrow Transplant. 17(3), 394400 (2011).

PubMed Google Scholar

View original post here:
Predicting the risk of acute kidney injury after hematopoietic stem cell transplantation: development of a new predictive nomogram | Scientific...

Recommendation and review posted by Bethany Smith

Recently, an impressive development in embryology was reported by the Israeli Weizmann Institute of Science. Using only stem cells, without the presence of sperm, eggs, or even a womb, researchers successfully created functioning mouse embryos, complete with beating hearts, blood circulation, brain tissue and rudimentary digestive systems. Carolyn Johnson in The Washington Post described the discovery as a fascinating, potentially fraught realm of science that could one day be used to create replacement organs for humans.

For the more than 100,000 people currently waiting for a life-saving organ donation, that kind of breakthrough would indeed seem like a miracle. However, since scientists are still years away from creating human organs in a lab for the purpose of transplant, the technology raises serious ethical questions, none of which should be taken lightly.

One of these questions is, in fact, an old one. Do the promises of embryonic stem cell research justify it? While some stem cells can be harvested from a variety of non-embryonic sources such as bone marrow, others are harvested from so-called unused embryos that have been donated to science. The lives of these tiny, undeveloped human beings are taken in the process.

For context, the research conducted by the Weizmann Institute uses embryonic stem cells. Though, for the time being, this implies only embryonic stem cells harvested from mice, the move to human research would involve the harvesting of stem cells from human embryos and involve tissue derived from already living human beings.

The Christian stance on when life begins is the same as the science. Human life begins at conception, and every single human life is worthy of protection. If we would not take the life of a born child in our research for a cure for some medical condition, neither the anonymity of an embryo nor the confines of a laboratory justify doing the same thing in the process of embryonic stem cell research.

Science is a process of trial and error, but we should never employ trial and error with the lives of thousands of human beings, in particular human beings who cannot consent to our actions. A rule of thumb is this. If you wouldnt try an experiment on an adult or small child, dont do it to human embryos at any stage.

The breakthrough at the Weizmann Institute, however, takes this old debate a step further. On one hand, lead researcher Dr. Jacob Hanna was quick to clarify that the goal is not to make complete, living organisms of mice or any other species. We are really facing difficulties making organs, he said, and in order to make stem cells become organs, we need to learn how the embryo does that.

Given the history of science, including the last chapter involving breathless promises of what embryonic stem cell research would bring, the grandiose predictions of scientists should be taken with at least a grain of salt. The process of growing organs for mice, for example, involved the creation of entire embryos. Should the technology be perfected in mice, what ethical or legal limits are there to prevent the creation of synthetic human embryos for the purpose of harvesting their organs?

Our first concern should be what these embryos would be created for. The answer is, inevitably, science, devoid of any consideration for human purpose, relationships, worth, or dignity as equal members of the human species. All societies that treat people as a means of scientific advancement, instead of infinitely valuable ends in-and-of themselves, have a track record of perpetrating atrocities.

A second concern is what these embryos would be deprived of. Though not all do, every human should enter the world with the love and commitment of their biological mom and dad. The very design of human development suggests this, and societies have long recognized that those born without these relationships have had something priceless taken from them. Creating children from cloning or stem cells intentionally makes them orphans, ripping them from the vital context of parental relationship. It is a grave injustice.

Bringing children into the world as a product of pure science without the possibility of relationship with their biological parents or relatives is enough an ethical consideration to oppose such research, but we should also consider the implications of recklessly creating humans for future experimentation and of dismantling them to see how their components work.

Science is, in many ways, blind to what should be ethical bright lines. Creating organs for transplant in order to save lives is a worthy goal. But such work should only proceed in an ethical manner, one which does not require the death of other distinct, valuable, human beings. Unfortunately, such ideas have not shaped the society we live in today.

Continued here:
Creating Organs Cannot Be at the Expense of Human Embryos - BreakPoint.org

Recommendation and review posted by Bethany Smith

Exclusive:

Brave Aubrey Austin, four, donated her own stem cells and saved her baby brother Carey's life on the day he turned one, after he was diagnosed with a rare type of blood cancer aged just eight months

Image: Supplied via Lucy Laing)

A brave little girl saved the life of her baby brother on his first birthday.

Carey Austin was diagnosed with a rare type of blood cancer when he was just eight months old.

His only hope of survival was a stem-cell transplant.

Against all odds, his sister Aubrey, four, was a perfect match.

Surgeons operated on Careys first birthday and six months later he is cancer-free thanks to his big sister.

Image:

Their mum Naomi said: She absolutely adores Carey and when we explained to her about the transplant she wanted to do everything she could to save him.

Shes only four years old, yet she was only thinking of how she could help him. We felt so guilty putting her through an operation too, but it was Careys only chance of survival.

"She was so brave about it. She knew that her blood was going to save him.

During a two-hour procedure at Great Ormond Street Hospital, London, surgeons took out Aubreys stem cells and they were put into Careys body via a drip.

Naomi said: The fact that the transplant took place on Careys birthday was so significant that she was giving him a second chance at life on that special day.

The doctors and nurses said they had never seen anyone have a stem cell transplant on their birthday before.

Aubrey was very groggy and woozy when she came around from the operation, and she had puncture wounds on her back from where the stem cells had been taken out.

But she was still smiling through it all. She was so brave. She never complained about being in pain and she was just pleased to see how her little brother was afterwards.

Image:

Image:

When the brother and sister saw each other for the first time after the operation, there was not a dry eye in the room.

Naomi said: It was so sweet when they were reunited.

We took Aubrey to see Carey and she gave him a cuddle. They were thrilled to see each other again.

After a two-day hospital stay for Aubrey and seven weeks for Carey, the family were able to settle back into life back home in Brighton, East Sussex.

Carey is now in remission, with no signs of the cancer cells in his body.

But his parents have been warned that the disease is so aggressive that until March next year there is a 40% chance of it returning. After that, the likelihood falls to just 5%.

Naomi added: Two other children lost their lives on the cancer ward while we were there, so we know how lucky Carey has been.

He and Aubrey have always been close but now their bond is stronger than ever.

"Shes a superstar and he couldnt have wanted anything more from a big sister. Hes doing so well now. He loves playing with his cars and hes just learning to walk too.

Aubrey is with him all the time she just adores him. She knows that she has saved his life and she loves being a big sister to him. They play cars together and hes learning to walk, so she stands with him encouraging him to take his steps.

Image:

Carey fell ill last November but Naomi, a paediatric audiologist, and her husband Simon, a CPS lawyer, both 43, thought it was bronchitis because his sister had recently had the same thing.

A GP agreed but two days later he was rushed to hospital by ambulance with breathing difficulties.

Doctors at Great Ormond Street diagnosed juvenile myelomonocytic leukaemia, or JMML, which cannot be treated with chemotherapy. There are only 1.2 cases per million children in the UK each year.

Naomi said: I was hysterical. I kept trying to tell them that it wasnt cancer, it was bronchilitis. I couldnt accept what was happening.

Because parents are not suitable donors, Aubreys bone marrow was tested, a process that involves drawing a sample out using a needle.

Naomi said: There is only a 25% chance of any sibling being a match, so even with Aubrey we knew that the odds werent in our favour.

"If she hadnt been a match then we would have had to wait until doctors found an anonymous donor, but that may not have happened in time for Carey.

When the results came back to say that she was a perfect match for him, we couldnt believe it. We had been praying that she would save him, so to get the news that she was a match for him was just incredible.

When we heard I couldnt stop crying, it was so emotional. To think that Carey was going to have a chance of survival thanks to his big sister was the answer to our prayers.

The mum added: We did feel guilty about putting her through the procedure, but when we spoke to her about it, all she wanted to do was help. We were so proud of her.

The transplant was made even more special as it took place on March 15, which was Careys first birthday, giving the family a double celebration.

They are keen to raise awareness of the cancer symptoms and the charity Childhood Cancer and Leukaemia Group, which has helped them throughout their ordeal.

Naomi said: Having a child with cancer is one of the worst things that can happen to you. We didnt realise that it was leukaemia so we are thankful that it was spotted in time.

We received amazing support throughout from the hospital and from the CCLG.

We feel so lucky that Carey has come through it and it feels like a miracle to have him with us now.

Geoff Shenton, a childrens cancer specialist at Newcastle Upon Tyne Hospitals NHS Foundation, said: In a very small proportion of cases JMML can disappear on its own, but this is rare.

Most children will need a bone-marrow or stem-cell transplant. There is still a significant chance that the disease can relapse. There may be a possibility of a second transplant if this happens, but despite our best efforts, children still die from JMML.

For more information and support visit cclg.org.uk

Visit link:
Girl, four, saves baby brother's life by donating her stem cells on his 1st birthday - The Mirror

Recommendation and review posted by Bethany Smith

Fast-track status is granted for frontotemporal dementia, next to the existing designation for traumatic spinal cord injury

GELEEN, Netherlands, Sept. 8, 2022 /PRNewswire/ -- The European Union has grantedstem cell biotech Neuroplastan orphan medicinal product designation for the applicability of its stem cell technology platform to frontotemporal dementia (FTD), following a positive opinion from The European Medicines Agency (EMA). With the existing orphan disease designation (ODD) for traumatic spinal cord injury (TSCI), Neuro-Cells is now approved for a fast-track development pathway with market exclusivity for both a trauma-induced and a chronic degenerative central nervous system disorder. This marks an important milestone in the development roadmap of Neuroplast's Neuro-Cells platform, as a stepping stone to other chronic neurodegenerative diseases such as Alzheimer's, ALS and Parkinson's Disease. The potential width in therapeutic applicability of the Neuroplast technology gives perspective to millions of people suffering from neurodegenerative diseases that currently have no outlook on effective treatment.

One technology addresses underlying mechanisms of multiple acute and chronic neurological disorders

Several conditions of the central nervous system, even when they seem unrelated at first and may have distinctive causes, have similar underlying disease mechanisms in common. These include unprogrammed cell death boosted by inflammation. Neuro-Cells, an autologous, bone-marrow derived Advanced Therapy Medicinal Product, addresses that disease mechanism by moderating inflammation of damaged cells in the central nervous system, to limit further impairment. The treatment objective in acute disorders is to limit impact of sudden injury, where the treatment objective in chronic disorders is to limit progression of the disease.

Neuroplast is already running a fast-track development pathway for traumatic spinal cord injury (TSCI), with a Phase II clinical trial in progress. This designation for frontotemporal dementia illustrates the broader applicability of the same technology for acute as well as chronic neurodegenerative disorders, paving the way to explore further applicability to conditions such as ALS, Alzheimer's disease, traumatic brain injury, subarachnoid stroke and Parkinson's Disease.

Orphan disease designation for FTD awarded based on pre-clinical evidence

Orphan disease designations are restricted to products for rare conditions for which there are no satisfactory methods of treatment authorized. It allows for a faster market authorization pathway and ten-year market exclusivity.

Frontotemporal dementia (FTD) is a degenerative condition in the brain that affect approximately 3.8 people in 10,000 persons in the EU. Typical survival rate lies between three and fourteen years from symptom onset, dependent on the FTD variant at play.

For this approval, the European Union followed the positive opinion from the EMA after the EMA followed positive recommendations from the Committee for Orphan Medicinal Products (COMP). COMP partly based their conclusions on the availability of pre-clinical evidence in mice, that showed decrease in neuroinflammation markers and rescue of cognitive and social behavioral deficits. Examples include reduction of anxiety, depressive-like behavior and abnormal social behavior.

Neuroplast CEO Johannes de Munter states:

"This designation for frontotemporal dementia is an important milestone in expanding the Neuro-Cells development to a wider range of therapeutic areas. Using the same technology platform for traumatic spinal cord injury and frontotemporal dementia, illustrates an unusual range of acute and chronic neurological disorders that could potentially benefit from this."

Neuroplast is open to discuss investor opportunities to effectuate the clinical pathways to a wider scope of neurological conditions.

About Frontotemporal dementia

Frontotemporal dementia (FTD) is a degenerative condition in the brain that is characterized by behavioral and language impairments. Depending on the variant, patients experience changes in personality, emotion, speech or motor functions. Patients may first become indifferent or careless and have difficulty understanding sentences. While the condition progresses, patients may become language impaired, lack initiative and lose executive functions. The typical survival rate lies between three and fourteen years from symptom onset, dependent on the FTD variant at play.

FTD affects approximately 3.8 people in 10,000 persons in the EU, for whom there are no effective treatments available. Patients typically receive antipsychotics to limit behavioral symptoms.

About Neuro-Cells

Neuro-Cells is a transformative treatment under GMP. It contains non-substantially manipulated bone marrow-derived hematopoietic and mesenchymal stem cells, manufactured from a patient's own bone marrow (donor and receiver are the same person). Inflammatory inducing components and pathogens are removed during this process.

About Neuroplast

Neuroplast is a Dutch stem cell technology company focusing on fast-track development programs using autologous cell products for treatment of neurodegenerative diseases, with the aim of giving back perspective to people who suffer from those conditions.

The company was founded in August 2014 by physician Johannes de Munter and neurologist Erik Wolters. Current funders are Lumana Invest, Brightlands Venture Partners, LIOF and the Netherlands Enterprise Agency. Neuroplast is located at Brightlands Chemelot Campus in The Netherlands.

For more information, please visite http://www.neuroplast.com

About Lumana Invest

Investment company Lumana was established by entrepreneurs and unique due to not having a predetermined investment horizon. The Lumana founders showcase strong commitment to their portfolio companies by actively supporting management in strategic decision making.

About Brightlands Venture Partners

Brightlands Venture Partners (BVP) is the fund manager of Chemelot Ventures and is a so-called ecosystem investor. BVP invests in companies benefiting from and contributing to the Brightlands campuses in the south of The Netherlands. Other funds under management are BVP Fund IV, Brightlands Agrifood Fund and Limburg Ventures. The funds of BVP focus on sustainability and health; together the funds have made over 40 investments.

About LIOF

LIOF is the regional development agency for Limburg and supports innovative entrepreneurs with advice, network and financing. Together with entrepreneurs and partners, LIOF is working towards a smarter, more sustainable and healthier Limburg by focusing on the transitions of energy, circularity, health and digitalization.

About The Netherlands Enterprise Agency

The Netherlands Enterprise Agency operates under the auspices of the Dutch Ministry of Economic Affairs and Climate Policy. It facilitates entrepreneurship, improves collaborations, strengthens positions and helps realize national and international ambitions with funding, networking, know-how and compliance with laws and regulations.

Forward looking statements

All statements other than statements of historical facts, including the statements about the clinical and therapeutic potential and future clinical milestones of Neuro-Cells, the indications we intend to pursue and our possible clinical or other business strategies, and the timing of these events, are forward-looking statements. Forward-looking statements can be identified by terms such as "believes", "expects", "plans", "potential", "would" or similar expressions and the negative of those terms. These forward-looking statements are based on our management's current beliefs and assumptions about future events and on information currently available to management. Neuroplast B.V. does not make any representation or warranty, express or implied, as to the improper use of this article, accuracy, completeness or updated status of above-mentioned statements. Therefore, in no case whatsoever will Neuroplast B.V. be legally liable or liable to anyone for any decision made or action taken in conjunction with the information and/or statements in this press release or for any related damages.

In case of any further questions, please contact:

Neuroplast

Johannes de Munter, CEOT: +31 (0)85 076 1000E: [emailprotected]

LifeSpring LifeSciences Communication, Amsterdam

Leon MelensT: +31 6 538 16 427E: [emailprotected]

Logo: https://mma.prnewswire.com/media/1666795/Neuroplast_Logo.jpg

SOURCE Neuroplast

Link:
Neuroplast receives second orphan medicinal product designation for Neuro-Cells, paving the way for application to both chronic and trauma-induced...

Recommendation and review posted by Bethany Smith