Archive for June, 2021

Imagine being able to reverse blindness, cure multiple sclerosis (MS), or rebuild your heart muscles after a heart attack. For the past few decades, research into stem cells, the building blocks of tissues and organs, has raised the prospect of medical advances of this kind yet it has produced relatively few approved treatments. But that could be about to change, says Robin Ali, professor of human molecular genetics of Kings College London. Just as gene therapy went from being a fantasy with little practical value to becoming a major area of treatment, stem cells are within a few years of reaching the medical mainstream. Whats more, developments in synthetic biology, the process of engineering and re-engineering cells, could make stem cells even more effective.

Stem cells are essentially the bodys raw material: basic cells from which all other cells with particular functions are generated. They are found in various organs and tissues, including the brain, blood, bone marrow and skin. The primary promise of adult stem cells lies in regenerative medicine, says Professor Ali.

Stem cells go through several rounds of division in order to produce specialist cells; a blood stem cell can be used to produce blood cells and skin stem cells can be used to produce skin cells. So in theory you can take adult stem cells from one person and transplant them into another person in order to promote the growth of new cells and tissue.

In practice, however, things have proved more complicated, since the number of stem cells in a persons body is relatively limited and they are hard to access. Scientists were also previously restricted by the fact that adult stem cells could only produce one specific type of cell (so blood stem cells couldnt produce skin cells, for instance).

In their quest for a universal stem cell, some scientists initially focused on stem cells from human embryos, but that remains a controversial method, not only because harvesting stem cells involves destroying the embryo, but also because there is a much higher risk of rejection of embryonic stem cells by the recipients immune system.

The good news is that in 2006 Japanese scientist Shinya Yamanaka of Kyoto University and his team discovered a technique for creating what they call induced pluripotent stem cells (iPSC). The research, for which they won a Nobel Prize in 2012, showed that you can rewind adult stem cells development process so that they became embryo-like stem cells. These cells can then be repurposed into any type of stem cells. So you could turn skin stem cells into iPSCs, which could in turn be turned into blood stem cells.

This major breakthrough has two main benefits. Firstly, because iPSCs are derived from adults, they dont come with the ethical problems associated with embryonic stem cells. Whats more, the risk of the body rejecting the cells is much lower as they come from another adult or are produced by the patient. In recent years scientists have refined this technique to the extent that we now have a recipe for making all types of cells, as well as a growing ability to multiply the number of stem cells, says Professor Ali.

Having the blueprint for manufacturing stem cells isnt quite enough on its own and several barriers remain, admits Professor Ali. For example, we still need to be able to manufacture large numbers of stem cells at a reasonable cost. Ensuring that the stem cells, once they are in the recipient, carry out their function of making new cells and tissue remains a work in progress. Finally, regulators are currently taking a hard line towards the technology, insisting on exhaustive testing and slowing research down.

The good news, Professor Ali believes, is that all these problems are not insurmountable as scientists get better at re-engineering adult cells (a process known as synthetic biology). The costs of manufacturing large numbers of stem cells are falling and this can only speed up as more companies invest in the area. There are also a finite number of different human antigens (the parts of the immune system that lead a body to reject a cell), so it should be possible to produce a bank of iPSC cells for the most popular antigen types.

While the attitude of regulators is harder to predict, Professor Ali is confident that it needs only one major breakthrough for the entire sector to secure a large amount of research from the top drug and biotech firms. Indeed, he believes that effective applications are likely in the next few years in areas where there are already established transplant procedures, such as blood transfusion, cartilage and corneas. The breakthrough may come in ophthalmology (the treatment of eye disorders) as you only need to stimulate the development of a relatively small number of cells to restore someones eyesight.

In addition to helping the body repair its own tissues and organs by creating new cells, adult stem cells can also indirectly aid regeneration by delivering other molecules and proteins to parts of the body where they are needed, says Ralph Kern, president and chief medical officer of biotechnology company BrainStorm Cell Therapeutics.

For example, BrainStorm has developed NurOwn, a cellular technology using peoples own cells to deliver neurotrophic factors (NTFs), proteins that can promote the repair of tissue in the nervous system. NurOwn works by modifying so-called Mesenchymal stem cells (MSCs) from a persons bone marrow. The re-transplanted mesenchymal stem cells can then deliver higher quantities of NTFs and other repair molecules.

At present BrainStorm is using its stem-cell therapy to focus on diseases of the brain and nervous system, such as amyotrophic lateral sclerosis (ALS, also known as Lou Gehrigs disease), MS and Huntingtons disease. The data from a recent final-stage trial suggests that the treatment may be able to halt the progression of ALS in those who have the early stage of the disease. Phase-two trial (the second of three stages of clinical trials) of the technique in MS patients also showed that those who underwent the treatment experienced an improvement in the functioning of their body.

Kern notes that MSCs are a particularly promising area of research. They are considered relatively safe, with few side effects, and can be frozen, which improves efficiency and drastically cuts down the amount of bone marrow that needs to be extracted from each patient.

Because the manufacture of MSC cells has become so efficient, NurOwn can be used to get years of therapy in one blood draw. Whats more, the cells can be reintroduced into patients bodies via a simple lumbar puncture into the spine, which can be done as an outpatient procedure, with no need for an overnight stay in hospital.

Kern emphasises that the rapid progress in our ability to modify cells is opening up new opportunities for using stem cells as a molecular delivery platform. Through taking advantage of the latest advances in the science of cellular therapies, BrainStorm is developing a technique to vary the molecules that its stem cells deliver so they can be more closely targeted to the particular condition being treated. BrainStorm is also trying to use smaller fragments of the modified cells, known as exosomes, in the hope that these can be more easily delivered and absorbed by the body and further improve its ability to avoid immune-system reactions to unrelated donors. One of BrainStorms most interesting projects is to use exosomes to repair the long-term lung damage from Covid-19, a particular problem for those with long Covid-19. Early preclinical trials show that modified exosomes delivered into the lungs of animals led to remarkable improvements in their condition. This included increasing the lungs oxygen capacity, reducing inflammation, and decreasing clotting.

Overall, while Kern admits that you cant say that stem cells are a cure for every condition, there is a lot of evidence that in many specific cases they have the potential to be the best option, with fewer side effects. With Americas Food and Drug Administration recently deciding to approve Biogens Alzheimers drug, Kern thinks that they have become much more open to approving products in diseases that are currently considered untreatable. As a result, he thinks that a significant number of adult stem-cell treatments will be approved within the next five to ten years.

Adult stem cells and synthetic biology arent just useful in treatments, says Dr Mark Kotter, CEO and founder of Bit Bio, a company spun out of Cambridge University. They are also set to revolutionise drug discovery. At present, companies start out by testing large numbers of different drug combinations in animals, before finding one that seems to be most effective. They then start a process of clinical trials with humans to test whether the drug is safe, followed by an analysis to see whether it has any effects.

Not only is this process extremely lengthy, but it is also inefficient, because human and animal biology, while similar in many respects, can differ greatly for many conditions. Many drugs that seem promising in animals end up being rejected when they are used on humans. This leads to a high failure rate. Indeed, when you take the failures into account, it has been estimated that it may cost as much to around $2bn to develop the typical drug.

As a result, pharma companies are now realising that you have to insert the human element at a pre-clinical stage by at least using human tissues, says Kotter. The problem is that until recently such tissues were scarce, since they were only available from biopsies or surgery. However, by using synthetic biology to transform adult stem cells from the skin or other parts of the body into other types of stem cells, researchers can potentially grow their own cells, or even whole tissues, in the laboratory, allowing them to integrate the human element at a much earlier stage.

Kotter has direct experience of this himself. He originally spent several decades studying the brain. However, because he had to rely on animal tissue for much of his research he became frustrated that he was turning into a rat doctor.

And when it came to the brain, the differences between human and rat biology were particularly stark. In fact, some human conditions, such as Alzheimers, dont even naturally appear in rodents, so researchers typically use mice and rats engineered to develop something that looks like Alzheimers. But even this isnt a completely accurate representation of what happens in humans.

As a result of his frustration, Kotter sought a way to create human tissues. It initially took six months. However, his company, Bit Bio, managed to cut costs and greatly accelerate the process. The companys technology now allows it to grow tissues in the laboratory in a matter of days, on an industrial scale. Whats more, the tissues can also be designed not just for particular conditions, such as dementia and Huntingdons disease, but also for particular sub-types of diseases.

Kotter and Bit Bio are currently working with Charles River Laboratories, a global company that has been involved in around 80% of drugs approved by the US Food and Drug Administration over the last three years, to commercialise this product. They have already attracted interest from some of the ten largest drug companies in the world, who believe that it will not only reduce the chances of failure, but also speed up development. Early estimates suggest that the process could double the chance of a successful trial, effectively cutting the cost of each approved drug by around 50% from $2bn to just $1bn. This in turn could increase the number of successful drugs on the market.

Two years ago my colleague Dr Mike Tubbs tipped Fate Therapeutics (Nasdaq: FATE). Since then, the share price has soared by 280%, thanks to growing interest from other drug companies (such as Janssen Biotech and ONO Pharmaceutical) in its cancer treatments involving genetically modified iPSCs.

Fate has no fewer than seven iPSC-derived treatments undergoing trials, with several more in the pre-clinical stage. While it is still losing money, it has over $790m cash on hand, which should be more than enough to support it while it develops its drugs.

As mentioned in the main story, the American-Israeli biotechnology company BrainStorm Cell Therapeutics (Nasdaq: BCLI) is developing treatments that aim to use stem cells as a delivery mechanism for proteins. While the phase-three trial (the final stage of clinical trials) of its proprietary NurOwn system for treatment of Amyotrophic lateral sclerosis (ALS, or Lou Gehrigs disease) did not fully succeed, promising results for those in the early stages of the disease mean that the company is thinking about running a new trial aimed at those patients. It also has an ongoing phase-two trial for those with MS, a phase-one trial in Alzheimers patients, as well as various preclinical programmes aimed at Parkinsons, Huntingtons, autistic spectrum disorder and peripheral nerve injury. Like Fate Therapeutics, BrainStorm is currently unprofitable.

Australian biotechnology company Mesoblast (Nasdaq: MESO) takes mesenchymal stem cells from the patient and modifies them so that they can absorb proteins that promote tissue repair and regeneration. At present Mesoblast is working with larger drug and biotech companies, including Novartis, to develop this technique for conditions ranging from heart disease to Covid-19. Several of these projects are close to being completed.

While the US Food and Drug Administration (FDA) controversially rejected Mesoblasts treatment remestemcel-L for use in children who have suffered from reactions to bone-marrow transplants against the advice of the Food and Drug Administrations own advisory committee the firm is confident that the FDA will eventually change its mind.

One stem-cell company that has already reached profitability is Vericel (Nasdaq: VCEL). Vericels flagship MACI products use adult stem cells taken from the patient to grow replacement cartilage, which can then be re-transplanted into the patient, speeding up their recovery from knee injuries. It has also developed a skin replacement based on skin stem cells.

While earnings remain relatively small, Vericel expects profitability to soar fivefold over the next year alone as the company starts to benefit from economies of scale and runs further trials to expand the range of patients who can benefit.

British micro-cap biotech ReNeuron (Aim: RENE) is developing adult stem-cell treatments for several conditions. It is currently carrying out clinical trials for patients with retinal degeneration and those recovering from the effects of having a stroke. ReNeuron has also developed its own induced pluripotent stem cell (iPSC) platform for research purposes and is seeking collaborations with other drug and biotech companies.

Like other small biotech firms in this area, it is not making any money, so it is an extremely risky investment although the rewards could be huge if any of its treatments show positive results from their clinical trials.

Read more from the original source:
Investing in stem cells, the building blocks of the body - MoneyWeek

Corning HYPERStack 36-layer and 12-layer cell culture vessels

Mesenchymal stem cells (MSCs) are used frequently for cell therapy applications. As multipotent cells, they can differentiate into other lineages such as adipocytes, osteocytes, and chondrocytes. Additionally, they are known to secrete trophic factors that can play important roles in immunoregulation. Although MSCs can be isolated from several different tissue sources, those derived from bone marrow commonly are studied because they are easy to access in quantities large enough for therapeutic dosing (2 106 cells/kg of body weight). Still, that equates to 140 million cells for a 150-pound individual. And the process of expanding MSCs to achieve such quantities can introduce risks for heterogeneity-induced quality failures. Chances of clinical success can improve with a manufacturing process that maintains a homogeneous MSC population after expansion to meet required critical quality attributes (CQAs).

Once cells are scaled up, they need to be cryopreserved for stability and transport. Cryoprotectants such as dimethyl sulfoxide (DMSO) often are added to freezing media to reduce ice formation and increase cell survival after thawing. However, because DMSO can be cytotoxic, its final concentration in a drug product must be minimized. Tools from Corning Life Sciences can help scientists and drug developers meet growing demand for bone-marrowderived MSC therapies.

Figure 1:Expansion of human mesenchymal stem cells (MSCs) in a Corning HYPERStack-36 vessel; MSC densities ranging from 4.4 104 to 5.2 104 cells/cm2 were achieved after five days of culture. Across three studies, total MSC yield averaged 8.7 108 cells per HYPERStack-36 vessel, with >90% average MSC viability.

Mesenchymal Cell Scale-UpMSCs are adherent cells that are sensitive to manufacturing process changes. That sensitivity can complicate scale-up to large quantities. When cultured under suboptimal conditions, MSCs can lose their multipotency. Corning HYPERStack 36-layer cell culture vessels offer a solution. A HYPERStack unit uses proprietary technology to provide a large surface area in a compact footprint. That technology relies on an ultrathin, gas-permeable film to facilitate gas exchange in each layer of the vessel. Each HYPERStack module comprises 12 individual chambers featuring Corning CellBIND surface treatment for optimal cell attachment. One module provides 6,000 cm2 of surface area; three modules can connect to form a HYPERStack 36-layer vessel, totaling 18,000 cm2 of growth surface area.

When human bone-marrowderived MSCs are cultured in a HYPERStack 36-layer vessel, yields of >800 million viable cells can be achieved (Figure 1). Harvested cells show high viability and expression of markers demonstrating MSC multipotency (Figure 2). Such results show that large-scale expansion of MSCs in a HYPERStack vessel generates a homogeneous population of cells that maintain necessary CQAs.

Figure 2: Mesenchymal stem cells (MSCs) recovered from a Corning HYPERStack 36-layer cell culture vessels show >99% expression of CD90, CD105, and CD73 markers while expressing <0.5% of differentiation markers (CD45, CD34, CD11b, CD19, and HLA-DR).

Corning cryopreservation bags remain flexible at ultralow temperatures(e.g., 196 C).

Large-Scale CryopreservationCryopreservation of large quantities of cells has become an important strategy for simplifying cell therapy workflows because it increases product shelf life, allows time for quality testing, and lengthens the period of potential administration. Cryopreservation bags are designed for single-use storage, preservation, and transfer of large volumes of cells. Cornings cryopreservation bags are novel bag-film containers that can remain flexible at ultralow temperatures (196 C) because they are made from a proprietary polyolefinethyl vinyl acetate blend. Corning produces the bags in four sizes covering fill volumes between 20 mL and 190 mL, with demonstrated performance for storage of bone-marrowderived MSCs.

The Corning X-WASH system performs DMSO removal in a closed, sterile format.

DMSO RemovalDMSO can serve as a cryoprotectant for a wide range of cell types. It often accounts for 510% of a freezing solution to reduce ice formation and maintain cell viability. But because of its cytotoxic effects, DMSO must be removed as much as possible from a final cell therapy product. That can be accomplished by centrifugation and buffer exchange.

The Corning X-WASH system can perform those steps in sterile, closed conditions (Figure 3). The X-WASH system also uses highly sensitive infrared sensors and software to transfer process data from the X-WASH control module to a database. That feature supports good manufacturing practice (GMP) data processing, monitoring, and reporting. Ultimately, the X-WASH system is designed to wash, resuspend, and condense cell suspensions without compromising product quality.

Corning has used the X-WASH system to reduce the DMSO concentration from a bone-marrowderived MSC product. About 70 million human MSCs were processed into Corning cryopreservation bags containing 10 mL of a 90% fetal bovine serum (FBS) and 10% DMSO solution. MSCs were thawed into 200 mL of phosphate-buffered saline containing 2% human serum albumin and 5% glucose. Cells were added to X-WASH cartridges for processing, then analyzed for recovery (Figure 4), viability (Figure 5), and multipotency (Figure 6). Ultraperformance liquid chromatography (UPLC) was used to quantify DMSO reduction. UPLC analysis showed that 200-mL dilution followed by a 200-mL wash in an X-WASH system reduced the final DMSO concentration by at least40 fold.

Figure 3:Corning X-WASH system workflow to remove dimethyl sulfoxide (DMSO)

Figure 4:Recovery of human mesenchymal stem cells (MSCs) after washing with a Corning X-WASH system; the bars below represent MSC density, and cell viability levels are represented as dots.

Considerations for Closed Systems and Custom MediaClosed-system cell-culture products help reduce contamination risks during drug development and manufacturing. Thus, they should be considered when planning for cell-culture operations. Ordering multiple components and assembling tubing sets in house can add complexity and time to cell therapy processes. To aid in the development of such processes, Corning offers preassembled closed systems and aseptic-transfer caps that are compatible with many Corning cell culture vessels.

Figure 5:Human MSC multipotency as represented by average marker expression after processing with a Corning X-WASH system (with standard deviation, n = 3)

Corning closed-system solutions arrive at your facility sterile and ready to use. They mitigate contamination risks, reduce the time and expense of sourcing and assembly, and improve overall productivity. Moreover, Cornings extensive library of fully validated filters, connectors, tubing, and clamps enables customized design of a closed-system solution for a specific application.

In cell-based therapies, cultured cells are the final product, requiring different manufacturing processes from those used for conventional biologics production. Major considerations in cell therapy scale-up include culture vessels and media as well as cells themselves. Inadequate attention to culture equipment and raw materials not only can diminish a therapys efficacy, but also can result in regulatory challenges that might delay a candidates progress through development. Because culture media are linked to cell growth and productivity, they rank among the most critical aspects of process development during scale-up.

Figure 6:Dimethyl sulfoxide (DMSO) concentration in final product after 200-mL dilution followed by 200-mL wash in a Corning X-WASH system (data from three independent runs)

Although off-the-shelf media can provide fast and efficient solutions during early stages, they can have trouble meeting specific scale-up conditions later on. Moving from small-scale, small-volume, static cultures into large-scale, large-volume vessels can trigger a host of additional requirements that cannot be addressed easily using an off-the-shelf solution. Customization by a media manufacturer is an attractive solution to concerns associated with large production scales, including media stability, packaging, handling, and storage. Custom media solutions also help to derisk processing. Cornings high-quality custom development and manufacturing services can produce tailored media and reagents to meet cell therapy production needs.

Simplifying Cell Therapy WorkflowsAddressing the growing demand for cell-based therapies requires optimization of scale-up, cryopreservation, and DMSO removal. With some human MSC therapies requiring as many as one billion cells per dose, cell therapy companies need efficient ways to scale up production of homogeneous MSCs that meet CQAs. Additionally, large quantities of MSCs will need to be cryopreserved to simplify cell therapy workflows. Before product administration, DMSO and other reagents used during the manufacturing process will need to be reduced. Corning offers solutions to simplify the complete range of cell therapy workflows.

Hilary Sherman is senior scientist, and Chris Suarez is field applications manager at Corning Life Sciences, 836 North Street, Tewksbury, MA 01876; ScientificSupport@Corning.com; 1-800-492-1110.

CellBIND, HYPERStack, and X-WASH all are registered trademarks of Corning Incorporated.

Read more:
Cell Therapy Workflows Using Corning HYPERStack: MSC Production - BioProcess Insider

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Bone Marrow-Derived Stem Cells (BMSCS) Market Global Briefing and Future Outlook 2020 to 2027 The Courier - The Courier

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Rheumatoid Arthritis Stem Cell Therapy Market Size, Status and Precise Outlook During 2020 to 2026 The Manomet Current - The Manomet Current

Editor's note: Find the latest COVID-19 news and guidance in Medscape's Coronavirus Resource Center.

Patients with blood cancers are particularly vulnerable to COVID-19, and there has been concern that such patients mount poor responses to COVID vaccination.

Perhaps surprising, then, is a new study showing good responses in a subgroup of these patients who underwent intensive treatment with allogeneic hematopoietic stem cell transplant (HCT) or chimeric antigen receptor T-cell (CAR-T) therapy

These patients had relatively good responses to COVID-19 vaccination with the mRNA vaccine, with overall cellular and humoral responses that were near to or over 80%.

"I was actually surprised by the fact patients who underwent allogeneic HCT and are currently treated with immunosuppression medications had a such high response to the vaccine," first author Ron Ram, MD, director of the Bone Marrow Transplantation Unit, Division of Hematology, Tel-Aviv Sourasky Medical Center, Tel Aviv, Israel, told Medscape Medical News.

"In other seasonal vaccines, we usually see much lower responses," he noted. "The problem is that we are not sure how long this response lasts, and this should be further investigated."

The results show that among immunocompromised patients, "the vaccine is safe and efficacious," he concluded.

However, "5% of patients developed transient severe low counts and graft-vs-host disease [GVHD] exacerbation. Therefore, close monitoring of these patients is mandatory.

"COVID-19 is a very dangerous infection to our allogeneic and CAR-T patients, and all patients should be vaccinated as soon as possible," he added.

The study was presented at the European Hematology Association (EHA) 2021 Annual Meeting. It involved 79 eligible patients who received hematopoietic cell transplant (n = 66) and CAR T-cell therapy (n = 14) at the Tel Aviv Sourasky Medical Center.

The patients in the study were being treated for acute myeloid leukemia (46%), myelodysplastic syndromes (9%), acute lymphocytic leukemia (10%), diffuse large B-cell lymphoma (15%); and others.

All patients were vaccinated with the Pfizer/BioNTech BNT162b2 COVID-19 vaccine, which yielded a protection rate of 94.6% in a phase 3 study in healthy patients and is recommended for immunosuppressed patients.

The median age of the patients was 65 years, and the median time from infusion of cells to vaccination was 32 months in the allogeneic HCT group and 9 months in the CAR T-cell therapy group.

Of the allogeneic HCT patients, 62% had active chronic GVHD, and 58% were receiving immune suppressive therapy, mostly calcineurin inhibitors.

In addition, 11% of the patients overall had complete B-cell aplasia.

An evaluation of humoral immune responses to the vaccines at 7 to 14 days after the second vaccine dose, as determined on the basis of serology, showed that 82% of those in the allogeneic HCT group developed immunogenicity. However, the humoral response rate was only 36% in the CAR T-cell group.

When including the results cellular responses, assessed with the ELISpot assay, the tables were nearly turned, with the antibody titer response rate of 46% in the allogeneic HCT group and 79% in the CAR T-cell group.

Combined, the overall antibody responses were 86% of allogeneic HCT patients and 79% of CAR T-cell patients, Ram reported.

A multivariate analysis showed that factors associated with a positive humoral response included increased amount of time from the infusion of cells (P = .032), female sex (P = .028), and a higher number of CD19-positive cells (P = .047).

Age, active GVHD, and the intensity of concomitant immunosuppressive therapy were not predictive of results.

Ram noted that higher numbers of CD19-positive cells and CD4 cells were predictive of positive ELISpot cellular response (P = 0.49 and P = .041, respectively).

Two patients developed SARS-CoV-2 infection after receiving the first dose of the vaccine, although they did not require hospitalization. After fully recovering, both patients received a second dose.

The vaccine was well tolerated among patients in general. Side effects were similar to those observed in the nontransplant population.

Of the patients overall, 5% experienced GVHD exacerbation after each vaccine dose.

A low blood count was observed in about 10% of patients; in 5%, the cytopenia was severe.

Adverse events that were of grade 3 or higher occurred in 4.6% and 7% of the two groups, respectively. Although the adverse events resolved quickly in most cases, one secondary graft rejection occurred; that case is being investigated.

The European Society for Blood and Marrow Transplantation (EBMT) recommends vaccination starting at least 3 months after allogeneic HCT, and Ram said the recommendation "makes sense."

"We did see a nice response in the allogeneic HCT patients 3 months after the transplant," he said.

Exceptions were patients receiving anti-CD19 therapy in the CAR T-cell group and those with B-cell aplasia. "Those patients did not respond well to the vaccine, so this is something to take into consideration," Ram said.

"We certainly need more data about durability of the vaccine and methods in patients who do not have sufficient response to the vaccine."

The EBMT's recommendation on the timing of vaccination is endorsed by the National Comprehensive Cancer Network, which recommends that COVID-19 vaccination be delayed for at least 3 months for patients with allogeneic HCT or those undergoing CAR-T therapy.

In commenting on the study during a press conference, Elizabeth Macintyre, MD, said the new findings were encouraging.

"It's very precious to see consensus recommendations regarding who should be vaccinated and when, and the end result seems to be that it's better to be vaccinated than not," she said.

In a separate talk at the meeting, Evangelos Terpos, MD, PhD, reported finding lower response rates to COVID-19 vaccination among older patients with hematologic malignancies in general, consistent with findings from other studies.

Reporting on responses up to day 50 post vaccination among 48 patients with multiple myeloma (median age, 83 years), 40% of patients did not achieve antibody titers above the level of 30% considered to represent positivity.

Among the 49% who did achieve antibody responses above levels of 50%, representing clinically relevant inhibition, the treatment factors that were associated with the higher response included treatment with lenalidomide. Treatment with daratumumab or anti-BCMA conjugates was associated with very low antibody responses.

He noted other research of 58 older patients with Waldenstrom macroglobulinemia or other low-grade lymphomas showed similarly low responses, particularly among those receiving anti-CD20 treatment, compared with healthy individuals.

"We found that patients with hematological malignancies or solid tumors have lower responses [to the COVID-19 mRNA vaccine], especially those under immunotherapy or targeted therapies, including anti-CD20, anti-CD38, anti-BCMA, Bruton kinase inhibitors, PDL-1 or PD-1 inhibitors," said Terpos, a professor of hematology at the National and Kapodistrian University of Athens School of Medicine, Athens, Greece.

Ram has disclosed no relevant financial relationships. Terpos has relationships with Janssen, Genesis/Celgene, Amgen, Novartis, Sanofi, and Takeda.

European Hematology Association (EHA) 2021 Annual Meeting: Abstract S285. Presented June 11, 2021.

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Good Response to COVID-19 Vaccine After HSCT and CAR T-cell Tx Medscape - Medscape

Data Bridge Market Research has recently added a concise research on theGlobal Stem Cell Manufacturing Market to depict valuable insights related to significant market trends driving the industry. The report features analysis based on key opportunities and challenges confronted by market leaders while highlighting their competitive setting and corporate strategies for the estimated timeline. The development plans, market risks, opportunities and development threats are explained in detail. The CAGR value, technological development, new product launches and Industry competitive structure is elaborated. As per study key players of this market are Thermo Fisher Scientific. Merck Group, Becton, Dickinson and Company. Holostem Advanced Therapies, JCR Pharmaceuticals, Organogenesis Inc and more.

The Global Stem Cell manufacturing Market is expected to gain market growth in the forecast period of 2020 to 2027. Data Bridge Market Research analyses the market to account to USD 18.59 billion by 2027 growing at a CAGR of 6.42% in the above-mentioned forecast period. The growing awareness towards diseases like cancer, hematopoietic disorders and degenerative disorders is going to drive the growth of the stem cell manufacturing market.

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Global Stem Cell Manufacturing Market, By Product (Stem Cell Line, Instruments, Culture Media, Consumables), Application (Research Applications, Clinical Applications, Cell and Tissue Banking), End Users (Hospitals and Surgical Centers, Pharmaceutical and Biotechnology Companies, Clinics, Community Healthcare, Others), Country (U.S., Canada, Mexico, Germany, Italy, U.K., France, Spain, Netherland, Belgium, Switzerland, Turkey, Russia, Rest of Europe, Japan, China, India, South Korea, Australia, Singapore, Malaysia, Thailand, Indonesia, Philippines, Rest of Asia-Pacific, Brazil, Argentina, Rest of South America, South Africa, Saudi Arabia, UAE, Egypt, Israel, Rest of Middle East & Africa) Industry Trends and Forecast to 2027

Healthcare Infrastructure growth Installed base and New Technology Penetration

Stem cell manufacturing market also provides you with detailed market analysis for every country growth in healthcare expenditure for capital equipment, installed base of different kind of products for stem cell manufacturing market, impact of technology using life line curves and changes in healthcare regulatory scenarios and their impact on the stem cell manufacturing market. The data is available for historic period 2010 to 2018.

The Global Stem Cell Manufacturing Market is highly fragmented and the major players have used various strategies such as new product launches, expansions, agreements, joint ventures, partnerships, acquisitions, and others to increase their footprints in this market. The report includes market shares of stem cell manufacturing market for global, Europe, North America, Asia Pacific and South America.

Global Stem Cell Manufacturing Market Scope and Market Size

Stem cell manufacturing market is segmented on the basis of product, application and end users. The growth amongst these segments will help you analyse meagre growth segments in the industries, and provide the users with valuable market overview and market insights to help them in making strategic decisions for identification of core market applications.

Major Market competitors/players:Global Stem Cell manufacturing Market

Some of the major players operating in the stem cell manufacturing market are Thermo Fisher Scientific. Merck Group, Becton, Dickinson and Company. Holostem Advanced Therapies, JCR Pharmaceuticals, Organogenesis Inc, Osiris Therapeutics, Osiris Therapeutics, Vericel Corporation, AbbVie, American CryoStem, AM-Pharma, Anterogen.Co.,Ltd, Astellas Pharma, Bristol-Myers Squibb, Apceth Biopharma, Cellular Dynamics International, Rheacell, Takeda Pharmaceutical, Teva Pharmaceutical Industries Ltd. ViaCyte, VistaGen Therapeutics Inc, Translational Biosciences, GlaxoSmithKline plc, Daiichi Sankyo Company, Limited, among others.

Market Definition:

This market report defines the market trends and forecast the upcoming opportunities and threats of the stem cell manufacturing market in the next 8 years. Stem cell manufacturing is a process of extracting the cells either from bone marrow or peripheral blood cells and culturing the cells in the culture dish containing nutrient media. Stem cells can be isolated from umbilical cord blood, placenta, amniotic sac, amniotic fluid, adipose tissue and menstrual blood. Stem cell manufacturing is used in the cell therapy as well as in gene therapy. Stem cell therapy is under research for many diseases like degenerative diseases and hematopoietic disorders like sickle cell anemia, storage disorders. Now stem cells are also used in making the cell and tissue bank. Some of the cell culture banks are National Institute of Biomedical Innovation, Health and Nutrition and World Federation for Culture Collections.

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Stem Cell manufacturing Market All-Inclusive Research Report (20212027) : Includes Impact of COVID-19 The Manomet Current - The Manomet Current

Dublin, June 18, 2021 (GLOBE NEWSWIRE) -- The "Global Cell Isolation Market By Product (Consumables and Instruments), By Cell Type (Human Cells and Animal Cells), By Source, By Technique, By Application, By End-User, By Region, Competition Forecast & Opportunities, 2026" report has been added to ResearchAndMarkets.com's offering.

The Global Cell Isolation Market was valued at USD7013.71 million in 2020 and is anticipated to reach USD15529.45 million by 2026 by registering a CAGR of 15.25% until 2026.

Cell isolation is a technique of isolating cells for diagnosis and analysis of a particular type of cell. The market growth can be attributed to the rising demand for drugs, vaccines and other related products, as they are manufactured with the assistance of cell isolation technique. Increasing popularity of precision medicines is also working in the favor of the market growth.

The Global Cell Isolation Market has been segmented into product, cell type, source, technique, application, end-user, company and region. Based on technique, the market is further fragmented into centrifugation-based cell isolation, surface-marker based cell isolation and filtration-based cell isolation, amongst which, centrifugation-based cell isolation segment occupied the largest market share in 2020 as it finds extensive applications in various end-user sectors such as academic institutes, research laboratories, etc.

Based on application, the market is further divided into biomolecule isolation, cancer research, stem cell research, in vitro diagnostics and others. Among these, cancer research and stem cell research are projected to be the lucrative segments of the market in the forecast period. Increase in the research activities by biopharma companies and laboratory is the key factor for the growth of the segments.

Based on regional analysis, Asia-Pacific is expected to grow at the highest CAGR during the forecast period. The high CAGR of the region can be attributed to the relaxation in the stringent rules and regulations laid down by the government for drug development. Another factor that can be held responsible for the fastest growth of the region is the availability of competent researchers and personnel who can carry out cell isolation techniques along with a wide genome pool.

The market players are focusing on research and development activities in order to enhance their product portfolios and strengthen their position across the global market. For instance, the major pharmaceutical companies worldwide are making substantial investments in R&D to introduce new drugs in the market.

Such investments are expected to increase the demand for cell isolation products over the coming years. In addition to this, new product developments help vendors to expand their product portfolio and gain maximum share in the sector. For example, Thermo Scientific's Medifuge is a benchtop centrifuge which is having a unique hybrid rotor as well as an interchangeable swing-out buckets and fixed-angle rotors to facilitate rapid & convenient applications on a single platform.

Moreover, collaborations, mergers & acquisitions and regional expansions are some of the other strategic initiatives taken by major companies for serving the unmet needs of their customers.

Major players operating in the Global Cell Isolation Market include

Years considered for this report:

Objective of the Study:

Key Target Audience:

Report Scope:

Global Cell Isolation Market, By Product:

Global Cell Isolation Market, By Cell Type:

Global Cell Isolation Market, By Source:

Global Cell Isolation Market, By Technique:

Global Cell Isolation Market, By Application:

Global Cell Isolation Market, By End-User:

Global Cell Isolation Market, By Region:

Competitive Landscape

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Global $15.52 Bn Cell Isolation (Human Cells and Animal Cells) - GlobeNewswire

DALLAS June 21, 2021 Jean D. Wilson, M.D., an internationally known endocrinologist whose scientific discoveries led to profound insights into the mechanisms underlying sexual differentiation and led to now widely used treatments for prostate disease, died June 13. He was 88.

Wilson, seen here in 1962, graduated from UTSouthwestern Medical School in 1955 and joined the faculty in 1960, where he began his studies of testosterone.

Wilson, professor emeritus of internal medicine at UTSouthwestern, was largely responsible for current understanding of the mechanisms by which steroid hormones induce male sexual differentiation. He also was instrumental in identifying the scientific underpinnings of a widely prescribed class of drugs known as 5-alpha-reductase inhibitors which include finasteride (Proscar, Propecia) and dutasteride (Avodart) to treat enlarged prostate and balding in men.

Wilsons discovery of 5-alpha-reductase and the identification of dihydrotestosterone as the primary hormone associated with the growth of the prostate transformed our understanding of prostate gland growth and paved the way for new effective treatment of prostate disease, says Daniel K. Podolsky, M.D., president of UTSouthwestern. His findings led to the first medical therapy for benign prostatic hyperplasia, and also provided the basis for understanding of the mechanism underlying the differentiation of male and female genital development. His legacy will be found in the legions of patients who have benefited from the therapy made possible by his discoveries.

Wilson, seen in 1978, was a popular and highly sought-after attending physician on the wards of Parkland Memorial Hospital, valued for his vast expertise in endocrinology and medicine in general.

Jean Wilson was one of the most critical and helpful sources of information concerning the development of two important drugs we were developing at Merck the statins, for control of LDL cholesterol, and Proscar, for treatment of benign prostate enlargement. Wilson was always available to wrestle with problems that often arise in drug development. I needed expert friends in those early days, and probably still do, says P. Roy Vagelos, M.D., former chairman, president, and chief executive officer of Merck & Co. and now chair of the board of Regeneron Pharmaceuticals.

Wilsons research included the study of cholesterol metabolism and steroid hormone action. The UTSouthwestern Medical School graduate and former National Institutes of Health (NIH) researcher earned international prominence for his investigations of testosterone including its formation from cholesterol as well as its metabolism and action. His efforts elucidated disorders resulting from genetic defects that lead to disruption in sex hormone biosynthesis with corresponding alteration in development.

Collaborations at UTSouthwestern with David Russell, Ph.D., professor of molecular genetics, led to the cloning of the 5-alpha-reductase (5AR) gene, development of animal models for 5AR deficiency, and eventually the finding that a 5AR inhibitor blocked prostate growth, which resulted in clinical trials led by Claus Roehrborn, M.D., chair of urology. The human androgen receptor later was cloned in 1989, allowing Wilson and colleagues to identify the receptor as a transcription factor that could regulate both the receptor and 5AR expression in prostate cancer. Other scientists at UTSouthwestern expanded upon his research, identifying androgen involvement in virtually all aspects of prostate development, alternate mechanisms of androgen synthesis, and other forms of androgens related to castrate-resistant prostate cancer.

Among his numerous awards, Wilson received the Kober Medal from the Association of American Physicians (1999); the Fred Conrad Koch Award from The Endocrine Society (1993); Gregory Pincus Award from the Worcester Foundation for Experimental Biology (1992); Henry Dale Medal from the Society for Endocrinology (1991); Amory Prize from the American Academy of Arts and Sciences (1977); and the Eugene Fuller Award from the American Urological Association. He was elected as a member of the American Academy of Arts and Sciences (1982), the National Academy of Sciences (1983), and the National Academy of Medicine (1994) as well as the American Philosophical Society and served as president of the Endocrine Society, the American Society for Clinical Investigation, and the Association of American Physicians.

Wilson, seen in 1992, was elected as a member of the American Academy of Arts and Sciences (1982), the National Academy of Sciences (1983), and the National Academy of Medicine (1994).

Wilson, who had held the Charles Cameron Sprague Distinguished Chair of Biomedical Research, was known as a collaborative colleague and empathetic adviser to students and fellows. His approach with students and trainees was threefold find out what they want to do, encourage them to do it, and develop pathways to fulfill their goals, he said in an interview with The Journal of Clinical Investigation. He also noted that some of the most difficult students to counsel turned out to be late bloomers who really were worth an investment of time and effort.

At UTSouthwestern, he served as the first director of the Medical Scientist Training Program, and it was recently announced that the Physician Scientist Training Program in Internal Medicine would be known as the Jean Wilson Society. The Jean D. Wilson Center for Biomedical Research and The Jean D. Wilson, M.D. Award, which honor excellence in scientific research mentorship, are named in his honor. The center was established with support from Dr. Wilson and his sister, the late Dr. Margaret Sitton, to promote research in endocrinology, developmental biology, and genetics, along with the J.D. and Maggie E. Wilson Distinguished Chair in Biomedical Research. In addition, he served among editors of two landmark medical textbooks Williams Textbook of Endocrinology and Harrisons Principles of Internal Medicine and as editor for The Journal of Clinical Investigation, among other journals. He authored The Memoir of a Fortunate Man, which chronicles his life growing up in the Texas Panhandle through his rise to pioneering academic physician and researcher.

Jean was a popular and highly sought-after attending physician on the wards of Parkland Memorial Hospital, valued for his vast expertise in endocrinology and medicine in general, say Nobel Laureates Joseph Goldstein, M.D, chair of molecular genetics, and Michael Brown, M.D., director of the Erik Jonsson Center for Research in Molecular Genetics and Human Disease. He founded a diabetic foot clinic at Parkland and spent hours each week clipping toenails and treating ulcers on the feet of elderly diabetic patients. After long days on the wards, he would retire to his modest laboratory where he would spend half the night meticulously dissecting rabbit fetuses. Often, when we were just starting our careers, we would sit by his side while he dissected, receiving sage advice about our careers as physician-scientists and life in general. Later, he extended his fatherly role to generations of M.D./Ph.D. students when he became the founding director of our M.D./Ph.D. program.

He had a rich life outside of the Medical Center as well. An avid opera buff, Wilson collected antique gramophones that could play every type of recording that had ever been produced. His extensive collection of 3,500 old 78-rpm operatic recordings included a 1917 disc of Enrico Caruso singing songs of Irving Berlin the only record that Caruso ever recorded in English, they note.

An avid opera buff, Wilson, seen in 2019, collected antique gramophones. His extensive collection of 3,500 old 78-rpm operatic recordings included a 1917 disc of Enrico Caruso singing songs of Irving Berlin the only record that Caruso ever recorded in English.

He took memorable trips to places like the North Pole, Antarctica, the Galapagos Islands, and the Easter Islands. He often incorporated science into his trips, visiting the Kangaroo Island in Australia to study sexual development in wallabies, and to Kenya to biopsy the phallus of the spotted hyena. Fearless in the pursuit of knowledge, he performed a rectal examination on a lion to estimate the size of the prostate, Goldstein and Brown say. A dedicated bird watcher, he traveled the world to many exotic places, hoping to spot that rare bird. But in the end, the rarest of that rare bird was Jean Wilson himself.

Born in Wellington, Texas, in 1932, Wilson obtained an undergraduate degree in chemistry from UT Austin and graduated from UTSouthwestern Medical School in 1955. As a student, he studied the control of urinary acid secretion by adrenal hormones, and as a resident, he investigated cholesterol metabolism. After residency, he spent two years at the NIH, where he studied ethanolamine biosynthesis. He joined the UTSouthwestern faculty in 1960 where he began his studies of testosterone, and worked in 1970 at Cambridge University. In all, he spent 60 years at UTSouthwestern and was named professor emeritus of UTSouthwesterns storied internal medicine department in 2011.

Jean Wilson leaves us with a remarkable legacy a quintessential physician-scientist whose scholarship both inspires and continues to serve as a foundation for new advances, says Podolsky, also professor of internal medicine.

In a career spanning six decades at UTSouthwestern, Dr. Jean Wilsons discoveries included:

Cholesterol metabolism

Dr. Wilson developed methods for quantifying cholesterol synthesis, absorption, degradation, and excretion in lab animals. Together, these analytical methods served as tools for understanding the feedback control of cholesterol synthesis and turnover. In addition, Dr. Wilson demonstrated that plasma cholesterol is synthesized in the intestinal wall and liver, findings that helped researchers define the contributions of diet and endogenous synthesis to cholesterol turnover in humans and other primates.

Male androgens

Concurrently, Dr. Wilson studied the action of male androgens, focusing on testosterone and its metabolite, dihydrotestosterone. Starting with a collaboration with his postdoctoral fellow, Nicholas Bruchovsky, in 1966, the researchers discovered that testosterone is converted inside prostate cells into dihydrotestosterone, a more potent androgen that is responsible for most of male sexual maturation and male sexual function. Dr. Wilson and his colleagues later showed that mutations that impair either the synthesis of testosterone, the conversion of testosterone to dihydrotestosterone, or the function of this metabolites receptor protein are the most common cause of birth defects associated with incomplete development of the male urogenital tract, affecting about four in every 1,000 boys. Cloning these responsible genes eventually allowed researchers to identify asymptomatic carriers of these mutations.

Dihydrotestosterone

Dr. Wilson also discovered that excess dihydrotestosterone is responsible for benign prostatic hyperplasia (BPH), or prostate enlargement, a condition that affects about 210 million men worldwide. Dihydrotestosterone is responsible for prostate growth in all male mammals, but in humans and dogs, prostate growth continues throughout life. Wilson and his colleagues showed that local excess of this potent androgen leads to prostate overgrowth. By curbing its production by inhibiting 5a-reductase, the enzyme that converts testosterone to dihydrotestosterone, they were able to prevent BPH in dog models of this condition. These findings have been developed into multiple 5a-reductase-inhibiting pharmaceuticals to treat this condition in human patients.

Brown, a Regental professor and director of the Erik Jonsson Center for Research in Molecular Genetics and Human Disease, holds The W.A. (Monty) Moncrief Distinguished Chair in Cholesterol and Arteriosclerosis Research, and the Paul J. Thomas Chair in Medicine.

Goldstein, a Regental professor and chair of molecular genetics, holds the Julie and Louis A. Beecherl, Jr. Distinguished Chair in Biomedical Research, and the Paul J. Thomas Chair in Medicine.

Podolsky holds the Philip OBryan Montgomery, Jr., M.D. Distinguished Presidential Chair in Academic Administration, and the Doris and Bryan Wildenthal Distinguished Chair in Medical Science.

Russell holds the Eugene McDermott Distinguished Chair in Molecular Genetics.

About UTSouthwestern Medical Center

UTSouthwestern, one of the premier academic medical centers in the nation, integrates pioneering biomedical research with exceptional clinical care and education. The institutions faculty has received six Nobel Prizes, and includes 24 members of the National Academy of Sciences, 16 members of the National Academy of Medicine, and 13 Howard Hughes Medical Institute Investigators. The full-time faculty of more than 2,800 is responsible for groundbreaking medical advances and is committed to translating science-driven research quickly to new clinical treatments. UTSouthwestern physicians provide care in about 80 specialties to more than 117,000 hospitalized patients, more than 360,000 emergency room cases, and oversee nearly 3 million outpatient visits a year.

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In Memoriam: Jean Wilson, M.D., made scientific discoveries that led to effective prostate treatments, insights into sexual differentiation - UT...

About 5.8 million Americans in 2020 were living with the disease, according to CDC data.2

Estimates show that this number will nearly triple to about 14 million people by the year 2060. Although the disease occurs mostly in older individuals, symptoms sometimes also occur in younger patients.

AD is the sixth-leading cause of death in the United States and the third-leading cause of death for patients older than age 65 years.

The disease is named after physician Alois Alzheimer, MD, who in 1906 discovered changes in the brain of a female patient who had died of mental illness. This patient suffered from language problems, memory loss, and unusual behavior. While examining her brain, Alzheimer found a few abnormal clumps, also known as amyloid plaques, and tangled bundles of fibers called neurofibrillary tangles.

An early symptom of AD is forgetfulness. As the disease advances, the individual may develop more severe memory impairment and can become more debilitated, causing then to struggle with completing everyday tasks. Symptoms are typically noticed by close family members who interact with the individual frequently, and they may become severe to the point that the patients forget relationships and even sometimes the names of loved relatives. Changes in the brains of these patients can also affect their behavior and mood and include aggressiveness, delusions, depression, irritability, more distrust of others, and social withdrawal.

Causes of AD may be related to brain proteins that fail to function normally, causing neurons to not be able to perform their duties. This impairment can be caused by environment, genetics, and lifestyle. The damage starts earlier than the point that the symptoms start to show, and in late stages, the brain shrinks significantly from its normal size.

The proteins involved include beta-amyloid plaques, which when clumped together cause toxic effects on neurons and disrupt the cell-to-cell connection. Meanwhile, Tau proteins help carry essential minerals and nutrients to the brain, and in patients with AD, these proteins change shape and become entangled. This disrupts the nutrient transport system, affecting brain cell function.

Genetics play a role in a patient developing AD, specifically for those with first-degree relatives diagnosed with the disease. Gender also plays a role, with women diagnosed more often than men, though this may also be related to the fact that women tend to live longer.1

One genetic factor known to cause the disease is Apolipoprotein E gene, which with the e4 variation, increases the risk of AD exponentially. Additionally, patients with Down syndrome are more likely to develop AD than others. This is likely related to 3 copies of the chromosome 21, which connect with the creation of beta-amyloid.

Diagnosing AD may include neuropsychological testing and brain imaging, using amyloid positron emission tomography (PET), computer tomography, magnetic resonance imaging, PET scan, or Tau PET imaging.

Medications used to great AD include those that help with memory symptoms and treat cognitive changes. Two major types of the drugs available on the market for these patients include cholinesterase inhibitors and memantine. Cholinesterase inhibitors may help treat agitation and depression and commonly include donepezil (Aricept), galantamine (Razadyne), and rivastigmine (Exelon). Memantine (Namenda) and the combination of memantine and donepezil (Namzaric) combine a cholinesterase inhibitor with memantine to help with medication compliance and help slow the progression of disease symptoms.

On June 8, 2021, the FDA approved aducanumab injection (Aduhelm), an amyloid beta-directed antibody indicated for the treatment of AD. Aduhelm comes in 2 dosages of 170mg/1.7ml and 300mg/3ml solution. The recommended dosage for this drug is 10mg/kg as an intravenous infusion for more than 60 minutes every 4 weeks.

Aduhelm provides new hope for patients diagnosed with AD to have more treatment options available to them after many years of waiting for an FDA approval. The future for research in this area is promising and holds the key to more discoveries, including a cure for AD.

References

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Aduhelm Is A New Option to Treat Alzheimer Disease - Pharmacy Times

Kiwi trans woman weightlifter Laurel Hubbard has reignited debates on transgender participation in sports following the announcement that she would be allowed to compete in the womens category at the Summer Olympics in Tokyo this year.

The landmark decision is the first since the International Olympic Committee modified its guidelines in 2015 to allow trans woman athletes to participate if they showed testosterone levels of less than 10 nanomoles per liter for at least one year prior to competition.

Needless to say, allowing Hubbard to participate in the womens weightlifting category has sparked both praise and criticism, with one side commending its inclusivity, while the other side draws attention to an uneven playing field created, with concerns that trans women may have an advantage over their peers and competitors due to their physical abilities.

Malta is equally divided on the subject, with some activists on one side of the fence and sports professionals on the other.

Laurel Hubbard is the first trans woman to compete in an Olympic Games

When you look at a female-born athlete at a high level, they are competing at around three nanomoles per liter of testosterone which already indicates that the level of testosterone of a female athlete is three times lower than that of a transgender woman that has undergone hormone replacement therapy, renowned strength and conditioning coach Nigel McCarthy told Lovin Malta.

Amongst the benefits of having higher testosterone levels are increased muscle mass, bone density, decreased fat percentage and recovery time.

Apart from the latter, performance does not reflect what happened the previous year but is an accumulation of years of building, he continued. The blood, hormones and cell production in your body are all signaled by previous years of training.

Despite having to undergo hormone replacement therapy, a body of research indicates that trans women are still at a biological advantage when compared to other female athletes, McCarthy argued.

Not all the advantages are diminished and still a large overwhelming benefit remains from being male years prior when it comes to biological advantages of strength and power, he said.

This is clearly shown by the results of transgender athletes when competing in female categories winning by large distances and not just the mere half a second or a centimeter.

In 2019, transgender athlete Rachel McKinnon set a world record time in sprint cycling with a timing of 11.649 seconds. Her opponent, Dawn Owrick, came in second with a time of 12.063.

Those on the other side of the fence argue that the decision to allow Hubbard to compete in the womens category of the Olympic Games promotes inclusivity and that it is skill, rather than genetics, that determines a champion.

Unfortunately, we think of sports as being fair which in reality is not, it is all a natural genetic lottery you do not choose to be tall, have higher testosterone levels or respond to training as well as others, McCarthy said.

While acknowledging that sports should be accessible to all, the S&C coach also believes in preserving the right of fairness for those who are also competing.

More studies need to be done to test baseline level before and after therapies for transgender athletes, he continued. There also needs to be a better understanding of the biological differences between males and females which determines athletic ability.

Hubbard will be competing in the womens 87kg weightlifting category. Though she has the backing of the New Zealand government and Olympic Committee, the decision on her participation hasnt been welcomed by some of her peers, including Belgian weightlifter Anna Vanbellinghen, who claimed that this particular situation is unfair to the sport and to the athletes.

As things stand, an out-of-proportion and unfair biological athletic advantage is occurring at the expense of born female athletes when competing against transgender athletes, McCarthy continued.

Hubbard had competed in mens weightlifting competitions prior to coming out as transgender in 2013.

The current total weightlifting record for men competing in the 89kg category is 387kg consisting of a combination of clean & jerk and snatch.

Meanwhile, the total female world record for athletes competing in the 87kg category stands at 294kg.

On a personal note, this should really be discussed by female athletes and not by myself or by any outsider not knowledgeable or impacted by the decisions, McCarthy said.

Females are having their hard work and opportunities lost to transgender athletes who evidently have an unfair athletic biological advantage, he ended.

What do you make of this? Let us know below

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'Unfair Biological Athletic Advantage', Maltese S&C Coach Weighs In On Trans Weightlifter Competing At The Olympics - Lovin Malta

Meet Raja, the newest resident of the Point Defiance Zoo & Aquarium. The 2-year-old critically endangered Sumatran tiger was revealed to the public last weekend (June 19-20) and could hold the key to protecting the Sumatran tiger species.

Rajas definitely not in Kansas anymore, but Tacoma welcomes the tiger with open arms and the possibility he brings.

Raja moved from Topeka Zoo & Conservation Center to Point Defiance Zoo in an attempt to boost breeding and ensure the future survival of the big cat species. With roughly 400 remaining, zoos joined the Species Survival Plan for Sumatran tigers to protect the critically endangered species from disappearing entirely because of habitat loss, poaching, lack of prey, and tiger-human conflict. Point Defiance Zoo has become a leader in Sumatran tiger conservation efforts, along with a list of other endangered species.

We are proud to be part of the conservation community working to ensure this species is around for future generations, Dr. Karen Goodrowe Beck, the general curator and Species Survival Plan coordinator for all tiger species at the Association of Zoos & Aquariums, said in a press release.

Rajas valuable genetics are vital to the survival of his species, Dr. Goodrowe Beck said. In the future, Raja will likely father the cubs of the three female Sumatran tigers housed at the zoo: Kali, Kirana, and/or Indah.

Visit Raja in the Asian Forest Sanctuary section of Point Defiance Zoo and stay tuned for news of Sumatran tiger cubs in the future.

Read more here:
Lions, More Tigers, and Bears, Oh My! Meet PDZA's Newest Resident Tiger - southsoundmag.com

Cancer treatments are improving as scientists are finding ways to develop new techniques and treatments. One of which is precision medicine, where they have focused on improving patients health using personalized solutions.

RELATED: Oxfords Genomics Pushing the Boundaries of Personalized Medicine

Precision medicine, in the simplest definition, is the way a patient is treated, diagnosed, or prevent disease by checking his/her genetics, environment, or lifestyle.

This type of treatment is related to pharmacogenomics. Where pharmacogenomics is the study of how a persons gene affects his/her response to a drug, it is used to treat a person through effective and safe medication tailored to their genes.

Precision medicine is now commonly used on patients treated with pancreatic cancer, lung cancer, melanoma/skin cancer, and colon cancer. It is also used to detect and treat HIV and cystic fibrosis.

Slowly, it is also seen in treatments for heart diseases, Alzheimers disease, rheumatoid arthritis, and multiple sclerosis.

In cancer patients, most medical facilities treat every patient the same way. However, studies suggest that not everyone responds to treatments the same way. One persons body may react differently with medicines as compared to another person.

Genetics plays a role in treating tumors, and precision medicine promise to tailor treatments based on a persons genes. It is seeing how a tumor would react to certain treatments that may work for other people.

Precision medicine can be used in the prevention and prediction of disease and management and treatment. Here are some examples of how it is used to treat, prevent, or treat people in a practical setting.

Checking your familys history of diseases and illnesses can somehow determine what you are capable of acquiring. If a family member has a history of cancer, heart diseases, diabetes, high blood pressure, or other chronic diseases, there is a high chance of you getting it.

With this data and information, a doctor can create treatment plans to prevent these from happening to you.

For example, when the doctor finds out that any of your family members had breast cancer, then the chances of you having it is likely. The doctor will then decide for you to have regular mammograms to check for any signs.

Newborns (usually right after theyre conceived) are screened where blood samples are taken. This test will check if they have any pre-existing conditions acquired from their parents, check hearing capabilities or heart defects, among others.

This way, the baby will be treated accordingly if any crucial or life-threatening conditions are seen.

For example, the newborn screening shows Baby Mary has severe combined immunodeficiency (SCID), she will receive a bone marrow transplant immediately to battle her condition. SCID is life-threatening to babies since its responsible for fighting off infections.

Personal trackers such as smartwatches or other mobile devices that check on your health can be lifesavers and be tools for precision medicine.

For example, a person is notified by his smart device that he is experiencing abnormal heart rates even if he has no family history of any heart condition. He then goes to see a doctor because of this and has been diagnosed with atrial fibrillation. This device could have saved his life because that condition can lead to a stroke. Now, he can treat his condition before it worsens.

Genomic sequencing can be used to control and track-out infectious diseases. Similar to whats been used to track COVID-19, this approach shows a DNA of a germ or virus where scientists have the opportunity to learn more about it and find a treatment a cure for it.

An example of this is the COVID-19, where scientists were able to extract samples from those infected with the virus and learn about it and find vaccines and cures for it, which is now slowly happening to us.

As a treatment, tumor profiling is genetic testing of a tumor. It is a way for doctors to choose which kind of treatment they would use for a condition. They would know from this process if cancer will return or would need radiation or chemotherapy.

For example, Jennys breast cancer returned and is diagnosed again. But her tumor profiling reveals she has triple-negative breast cancer. Her approach to this, along with her doctors, is a more aggressive one, including chemotherapy, radiation, and mastectomy.

RELATED: FDA Approves GSKs Checkpoint Inhibitor Jemperli for Endometrial Cancer

As mentioned above, pharmacogenomics studies how a person reacts to a certain treatment based on their genes. Doctors using this treatment can gauge if a certain medicine can be effective or not based on a patients history. They can also determine if the patient will experience any serious side effects.

For example, John needs to undergo Fluorouracil (5-FU), which is a type of chemotherapy. But if John has a low level of an enzyme called dihydropyrimidine dehydrogenase (DPD), which helps metabolize fluorouracil in the body, the doctors would need to check on him using pharmacogenomics. If he has a low dose of fluorouracil, an oncologist will decrease the dosage in the chemotherapy to prevent any serious side effects.

With these examples revealed, some facilities and companies provide precision medicine to improve the living conditions of patients treated with different diseases.

ExactCure is a French start-up that combines artificial intelligence with precision medicine to create flawless software for the use of drugs to be used by patients depending on their kidney status, genotype, gender, or age.

Patients use this service by inputting their data, and ExactCure will give the necessary medications based on the information provided.

Tepthera is a Swiss start-up that focuses on cancer immunotherapy, infectious and auto-immune diseases.

Their focus concerning precision medicine is on identifying T cell antigens for better and personalized therapies and treatment.

Caris Life Sciences is a molecular science company that focuses on precision medicine in oncology. They are working on the development of innovative therapeutics and advance potential treatments for cancer in the clinic.

They develop profiling assays for oncology that scan DNA, RNA and proteins to reveal a molecular blueprint to help physicians determine the best course of treatment for cancer patients.

Precigen is a Maryland-based company that is advancing its UltraCAR-T cell therapy approach to treating cancer.

They are now developing next-generation gene and cell therapies that can change the treatment paradigm in immuno-oncology, autoimmune disorders and infectious diseases.

There are numerous ways to treat diseases and medical conditions with the use of precision medicine. Scientists are continually finding out ways to improve patients lives by using their traits.

Read the original post:
Precision Medicine: Improving Health With Personalized Solutions - BioSpace

COVID appears to be in retreat in the U.S. and other nations that have widespread access to vaccines. But some developing countries with high infection rates have become hotspots for viral variants that may be more transmissible or resistant to vaccinesand these variants can quickly cross national borders. For example, the B.1.167.2 variant (now dubbed Delta) that was first detected in India has spread to more than 70 countries and regions, including the U.S.

Much of the developing world lacks the capacity for viral surveillanceefforts to monitor the spread and evolution of new variants. This process requires expensive genomic-sequencing technology and trained workforces that many nations do not have. Nepal, for instance, has sequenced just 0.01 percent of the more than 600,000 cases reported in the country so far. New variants could undo hard-won progress in curbing the pandemic, according to Alina Chan, a postdoctoral fellow specializing in gene therapy and cell engineering at the Broad Institute of the Massachusetts Institute of Technology and Harvard University. Variants that evolve to be able to reinfect previously infected people are likely to also reduce the efficacy of vaccines, she says.

Scientists and organizations around the world are now working to build capacity to hunt for variants in developing countries. They are mobilizing to deliver funds, training and equipment to where these resources are needed most, with aspirations of creating a lasting viral surveillance infrastructure. COVID is the catalyst, says Jairo Mendez-Rico, a microbiologist and adviser on viral diseases at the Pan American Health Organization (PAHO), headquartered in Washington, D.C. But we also need to survey for other pathogens that for sure will come in the future.

In India, 27 laboratories have now banded together to create the Indian SARS-CoV-2 Genomics Consortium (INSACOG). The group plans to sequence 5 percent of all positive COVID cases in the country (the current rate is only 0.09 percent). Shahid Jameel, a virologist and director of the Trivedi School of Biosciences at Indias Ashoka University, says that bringing existing surveillance capacity under a single umbrella could, in principle, make that a feasible goal. But there are not enough trained field-workers, he says, and the laboratories have acute shortages of chemical reagents needed for genomic analyses.

International experts are now stepping in to help. Recently, a nonprofit volunteer group called INDIA COVID SOS formed to assist with the pandemic response in the country. It aims to scale genomic surveillance across India, as well among neighboring South Asian nations. Aditi Hazra, an epidemiologist at Harvard Medical School, co-leads the groups sequencing team, which meets regularly on video conference calls with the directors of Indias sequencing consortium. She says a key objective is to extend viral surveillance to more people in rural areas, where much of the population lives.

Rural surveillance is a priority in Africa as well. Millions of people on the continent live in remote areas that are also hot spots for disease outbreaks, says Akaninyene Otu, a medical doctor and a senior lecturer at the University of Calabar in Nigeria. Several new partnerships aim to boost sequencing in African countries. Otu highlights the Africa Pathogen Genomics Initiative (Africa PGI), which launched last year with support from international donor organizations and private companies. Most of the sequencing capacity in Africa is concentrated in South Africa, Kenya, Nigeria, Morocco and Egypt. The Africa PGI, which is headed by the Africa Centers for Disease Control and Prevention, is setting out to create a pan-African network of sequencing centers to serve the continents 54 countries.

In Latin American countrieswhich are currently reporting some of the highest COVID infection rates in the worldPAHO is spearheading the COVID-19 Genomic Surveillance Regional Network. Some countries in the region already have fairly strong sequencing capabilities, but the network is leading efforts to build surveillance capacity where it does not exist at all, which is the case throughout much of Central America. In the interim, two large reference labsone in Brazil and one in Chileare sequencing samples sent by other countries at PAHO's expense, Mendez-Rico says.

In addition to building partnerships and networks, scientists are also exploring low-cost sequencing technologies that could be deployed easily in the field. Nearly all of the SARS-CoV-2 cases sequenced so far have relied on large, expensive instruments housed in climate-controlled lab facilities. As an alternative, INDIA COVID SOS is encouraging wider use of a handheld sequencing device made by Oxford Nanopore Technologies in England. The device, called the MinION, can run on a battery pack, processes 96 samples at a time and uses software to generate whole genome sequences that can be stored on a laptop. We're looking for technologies that are cheap, efficient, scalable and portable, and this is an example, Hazra says.

Keith Robison, a computational biologist at Ginkgo Bioworks, a Boston-based biotechnology company, agrees that the MinION is a practical option for developing nationsespecially in rural settings. The portable technology was widely used during the recent Ebola outbreaks in the Democratic Republic of the Congo and other West African countries. You can generate sequences with it from anywhere, he says. The MinION has its drawbacks: the quality of the data is not as good as what the lab-based instruments provide, Robison notes. However, that can also be computationally corrected if you have many copies of the same sequence, he says.

Tue Sparholt Jrgensen, a postdoctoral researcher in microbiology at the Technical University of Denmark, argues that whole-genome sequences may not always be needed. All the important SARS-CoV-2 mutations identified so far, he says, sit on the same stretch of genome encoding the microbes well-known spike protein. Jrgensen says scientists can simply target this piece of the viral geome with an alternative method called Sanger sequencing. This method, which was used as part of the effort that led to the sequencing of the complete human genome back in 2003, is still employed by labs all over the world. Unlike whole-genome methods that sequence millions of genetic fragments simultaneously, the Sanger method sequences one fragment at a time. Sanger can't replace whole-genome sequencing, but you can use it for targeted analyses at a fraction of the cost, Jrgensen says. People have been using it in small labs for decades. Id use it to monitor for known variants, [to] qualify samples for whole genome sequencing and for contact tracing [of infected people] in hospitals.

Jrgensen and his colleagues are now working with health officials in Rwanda on plans to expand Sanger-based COVID surveillance in the country. If a new variant emerges in Rwanda and starts spreading [elsewhere] in Africa, then we want to know about it, he says.

Read this article:
New Coronavirus Variants Are Urgently Being Tracked around the World - Scientific American

Each year, an estimated 1.8 million people in the United States are affected by cancer most commonly cancers of the breast, lung, prostate, and blood cancers such as leukemia. While not everyone overcomes the disease, thanks to science, more people are surviving and for longer than ever before in history.

We asked three people whose lives have been impacted by cancer to share their stories how their lives were changed by the disease, and how they're using that experience to change the future of cancer treatments with the hope that ultimately, in the fight against cancer, science will win. Here's what they had to say.

Photo courtesy of Celine Ryan

In September 2013, Celine Ryan woke up from a colonoscopy to some traumatic news. Her gastroenterologist showed her a picture of the cancerous mass they found during the procedure.

Ryan and her husband, Patrick, had scheduled a colonoscopy after discovering some unusual bleeding, so the suspicion she could have cancer was already there. Neither of them, however, were quite prepared for the results to be positive -- or for the treatment to begin so soon. Just two days after learning the news, Ryan had surgery to remove the tumor, part of her bladder, and 17 cancerous lymph nodes. Chemotherapy and radiation soon followed.

Ryan's treatment was rigorous but in December 2014, she got the devastating news that the cancer, once confined to her colon, had spread to her lungs. Her prognosis, they said, was likely terminal.

But rather than give up hope, Ryan sought support from online research, fellow cancer patients and survivors, and her medical team. When she brought up immunotherapy to her oncologist, he quickly agreed it was the best course of action. Ryan's cancer, like a majority of colon and pancreatic cancers, had been caused by a defect on the gene KRAS, which can result in a very aggressive cancer that is virtually "undruggable." According to the medical literature, the relatively smooth protein structure of the KRAS gene meant that designing inhibitors to bind to surface grooves and treat the cancer has been historically difficult. Through her support systems, Ryan discovered an experimental immunotherapy trial at the National Institutes of Health (NIH) in Bethesda, MD., and called them immediately to see if she was eligible. After months of trying to determine whether she was a suitable candidate for the experimental treatment, Ryan was finally accepted.

The treatment, known as tumor-infiltrating lymphocyte therapy, or TIL, is a testament to how far modern science has evolved. With this therapy, doctors remove a tumor and harvest special immune cells that are found naturally in the tumor. Doctors then grow the cells in a lab over the next several weeks with a protein that promotes rapid TIL growth and once the cells number into the billions, they are infused back into the patient's body to fight the cancer. On April 1, 2015, Ryan had her tumor removed at the NIH. Two months later, she went inpatient for four weeks to have the team "wash out" her immune system with chemotherapy and infuse the cells all 148 billion of them back into her body.

Six weeks after the infusion, Ryan and Patrick went back for a follow-up appointment and the news they got was stunning: Not only had no new tumors developed, but the six existing tumors in her lungs had shrunk significantly. Less than a year after her cell infusion, in April 2016, the doctors told Ryan news that would have been impossible just a decade earlier: Thanks to the cell infusion, Ryan was now considered NED no evaluable disease. Her body was cancer-free.

Ryan is still NED today and continuing annual follow-up appointments at the NIH, experiencing things she never dreamed she'd be able to live to see, such as her children's high school and college graduations. She's also donating her blood and cells to the NIH to help them research other potential cancer treatments. "It was an honor to do so," Ryan said of her experience. "I'm just thrilled, and I hope my experience can help a lot more people."

Photo courtesy of Patrice Lee

Patrice Lee got into scientific research in an unconventional way through the late ocean explorer Jacques Cousteau.

Lee never met Cousteau but her dreams of working with him one day led her to pursue a career in science. Initially, Lee completed an undergraduate degree in marine biology; eventually, her interests changed and she decided to get a dual doctoral degree in physiology and toxicology at Duke University. She now works at Pfizer's R&D site in Boulder, CO (formerly Array BioPharma), leading a group of scientists who determine the safety and efficacy of new oncology drugs.

"Scientists focused on drug discovery and development in the pharmaceutical industry are deeply committed to inventing new therapies to meet unmet needs," Lee says, describing her field of work. "We're driven to achieve new medicines and vaccines as quickly as possible without sacrificing safety."

Among the drugs Lee has helped develop during her career, including cancer therapies, she says around a dozen are currently in development, while nine have received FDA approval an incredible accomplishment as many scientists spend their careers without seeing their drug make it to market. Lee's team is particularly interested in therapies for brain metastases something that Lee says is a largely unmet need in cancer research, and something her team is working on from a variety of angles. "Now that we've had rapid success with mRNA vaccine technology, we hope to explore what the future holds when applying this technology to cancers," Lee says.

But while evaluating potential cancer therapies is a professional passion of Lee's, it's also a mission that's deeply personal. "I'm also a breast cancer survivor," she says. "So I've been on the other side of things and have participated in a clinical trial."

However, seeing how melanoma therapies that she helped develop have affected other real-life cancer patients, she says, has been a highlight of her career. "We had one therapy that was approved for patients with BRAF-mutant metastatic melanoma," Lee recalls. "Our team in Boulder was graced by a visit from a patient that had benefited from these drugs that we developed. It was a very special moment for the entire team."

None of these therapies would be available, Lee says without rigorous science behind it: "Facts come from good science. Facts will drive the development of new drugs, and that's what will help patients."

Photo courtesy of Cynthia Kuk

Cynthia Kuk was just 10 years old when they had a conversation that would change their life forever.

"My mother, who worked as a translator for the government at the time, had been diagnosed with breast cancer, and after her chemotherapy treatments she would get really sick," Kuk, who uses they/them pronouns, recalls. "When I asked my dad why mom was puking so much, he said it was because of the medicine she was taking that would help her get better."

Kuk's response was immediate: "That's so stupid! Why would a medicine make you feel worse instead of better? When I'm older, I want to create medicine that won't make people sick like that."

Nine years later, Kuk traveled from their native Hong Kong to the United States to do exactly that. Kuk enrolled in a small, liberal arts college for their Bachelor's degree, and then four years later started a PhD program in cancer research. Although Kuk's mother was in remission from her cancer at the time, Kuk's goal was the same as it had been as a 10-year-old watching her suffer through chemotherapy: to design a better cancer treatment, and change the landscape of cancer research forever.

Since then, Kuk's mission has changed slightly.

"My mom's cancer relapsed in 2008, and she ended up passing away about five years after that," Kuk says. "After my mom died, I started having this sense of urgency. Cancer research is such that you work for twenty years, and at the end of it you might have a fancy medication that could help people, but I wanted to help people now." With their mother still at the forefront of their mind, Kuk decided to quit their PhD program and enter medical school.

Now, Kuk plans to pursue a career in emergency medicine not only because they are drawn to the excitement of the emergency room, but because the ER is a place where the most marginalized people tend to seek care.

"I have a special interest in the LGBTQ+ population, as I identify as queer and nonbinary," says Kuk. "A lot of people in this community and other marginalized communities access care through the ER and also tend to avoid medical care since there is a history of mistreatment and judgement from healthcare workers. How you carry yourself as a doctor, your compassion, that can make a huge difference in someone's care."

In addition to making a difference in the lives of LGBTQ+ patients, Kuk wants to make a difference in the lives of patients with cancer as well, like their mother had.

"We've diagnosed patients in the Emergency Department with cancer before," Kuk says. "I can't make cancer good news but how you deliver bad news and the compassion you show could make a world of difference to that patient and their family."

During their training, Kuk advocates for patients by delivering compassionate and inclusive care, whether they happen to have cancer or not. In addition to emphasizing their patient's pronouns and chosen names, they ask for inclusive social and sexual histories as well as using gender neutral language. In doing this, they hope to make medicine as a whole more accessible for people who have been historically pushed aside.

"I'm just one person, and I can't force everyone to respect you, if you're marginalized," Kuk says. "But I do want to push for a culture where people appreciate others who are different from them."

More here:
Early-onset Alzheimer's stole the memory of his marriage. Then he proposed again. - Upworthy

By Siddhi Jain

New Delhi Experts are forewarning against the third wave of Covid-19 hitting India as early as September, with many fearing that it could hit children disproportionately. According to Rekha Mittal, Neurologist at Rainbow Hospitals, since many adults would have had the disease or the Covid vaccine, in comparison children would be a susceptible population.

Third wave and more waves can always occur in a pandemic till it burns out or there is herd immunity. So far in our experience, the majority of children who have had Covid infection have either been asymptomatic or had mild symptoms. Therefore we hope the next wave will not be a serious threat to children, Mittal told IANSlife.

The treatment of children who get serious manifestations of Covid requires specialists who are trained in intensive care of children, and the appropriate equipment and support staff. We should definitely plan and be prepared for a crisis situation if it does occur. We should not be complacent.

How can a surge in cases be avoided? The expert suggests not letting our guard down, by maintaining Covid-appropriate behaviour such as social distancing, use of masks, and hand sanitisation measures. We need to avoid crowded places such as malls, markets, gatherings etc. Also, the children will get protected indirectly, if the adults around them receive the Covid vaccine.

Rainbow Hospitals successfully administered the wonder drug Zolgensma, which is also the worlds costliest at Rs 16 crore per shot, to a three-year-old Hyderabad boy this month. The medical marvel Zolgensma is a single dose intravenous injection used in gene therapy is for replacing the defective SMN1 gene through an adenoviral vector.

Asked how critical can it be to administer the expensive injection like this with skill, Mittal says: Administering such an expensive and uncommonly used drug can be a challenge. One will need to have complete knowledge about procuring, handling and administering the medication. Also, one would have to have all facilities to handle the side effects if they occur, such as liver damage, drop in platelet count etc.

Speaking about vaccination for children, the doctor said vaccinations for vaccine preventable diseases should continue on time for childrens as per schedule.

Rainbow hospital provides a safe area where children can be brought in for vaccinations. Trials for Covid vaccination in children above 2 years of age have started in India. As and when Covid vaccine is approved and available for children, Rainbow hospitals will be ready to administer the same to as many children as possible. (IANS)

Continued here:
Pediatric healthcare and the third wave of Covid-19 - India New England

Each year, an estimated 1.8 million people in the United States are affected by cancer most commonly cancers of the breast, lung, prostate, and blood cancers such as leukemia. While not everyone overcomes the disease, thanks to science, more people are surviving and for longer than ever before in history.

We asked three people whose lives have been impacted by cancer to share their stories how their lives were changed by the disease, and how they're using that experience to change the future of cancer treatments with the hope that ultimately, in the fight against cancer, science will win. Here's what they had to say.

Photo courtesy of Celine Ryan

In September 2013, Celine Ryan woke up from a colonoscopy to some traumatic news. Her gastroenterologist showed her a picture of the cancerous mass they found during the procedure.

Ryan and her husband, Patrick, had scheduled a colonoscopy after discovering some unusual bleeding, so the suspicion she could have cancer was already there. Neither of them, however, were quite prepared for the results to be positive -- or for the treatment to begin so soon. Just two days after learning the news, Ryan had surgery to remove the tumor, part of her bladder, and 17 cancerous lymph nodes. Chemotherapy and radiation soon followed.

Ryan's treatment was rigorous but in December 2014, she got the devastating news that the cancer, once confined to her colon, had spread to her lungs. Her prognosis, they said, was likely terminal.

But rather than give up hope, Ryan sought support from online research, fellow cancer patients and survivors, and her medical team. When she brought up immunotherapy to her oncologist, he quickly agreed it was the best course of action. Ryan's cancer, like a majority of colon and pancreatic cancers, had been caused by a defect on the gene KRAS, which can result in a very aggressive cancer that is virtually "undruggable." According to the medical literature, the relatively smooth protein structure of the KRAS gene meant that designing inhibitors to bind to surface grooves and treat the cancer has been historically difficult. Through her support systems, Ryan discovered an experimental immunotherapy trial at the National Institutes of Health (NIH) in Bethesda, MD., and called them immediately to see if she was eligible. After months of trying to determine whether she was a suitable candidate for the experimental treatment, Ryan was finally accepted.

The treatment, known as tumor-infiltrating lymphocyte therapy, or TIL, is a testament to how far modern science has evolved. With this therapy, doctors remove a tumor and harvest special immune cells that are found naturally in the tumor. Doctors then grow the cells in a lab over the next several weeks with a protein that promotes rapid TIL growth and once the cells number into the billions, they are infused back into the patient's body to fight the cancer. On April 1, 2015, Ryan had her tumor removed at the NIH. Two months later, she went inpatient for four weeks to have the team "wash out" her immune system with chemotherapy and infuse the cells all 148 billion of them back into her body.

Six weeks after the infusion, Ryan and Patrick went back for a follow-up appointment and the news they got was stunning: Not only had no new tumors developed, but the six existing tumors in her lungs had shrunk significantly. Less than a year after her cell infusion, in April 2016, the doctors told Ryan news that would have been impossible just a decade earlier: Thanks to the cell infusion, Ryan was now considered NED no evaluable disease. Her body was cancer-free.

Ryan is still NED today and continuing annual follow-up appointments at the NIH, experiencing things she never dreamed she'd be able to live to see, such as her children's high school and college graduations. She's also donating her blood and cells to the NIH to help them research other potential cancer treatments. "It was an honor to do so," Ryan said of her experience. "I'm just thrilled, and I hope my experience can help a lot more people."

Photo courtesy of Patrice Lee

Patrice Lee got into scientific research in an unconventional way through the late ocean explorer Jacques Cousteau.

Lee never met Cousteau but her dreams of working with him one day led her to pursue a career in science. Initially, Lee completed an undergraduate degree in marine biology; eventually, her interests changed and she decided to get a dual doctoral degree in physiology and toxicology at Duke University. She now works at Pfizer's R&D site in Boulder, CO (formerly Array BioPharma), leading a group of scientists who determine the safety and efficacy of new oncology drugs.

"Scientists focused on drug discovery and development in the pharmaceutical industry are deeply committed to inventing new therapies to meet unmet needs," Lee says, describing her field of work. "We're driven to achieve new medicines and vaccines as quickly as possible without sacrificing safety."

Among the drugs Lee has helped develop during her career, including cancer therapies, she says around a dozen are currently in development, while nine have received FDA approval an incredible accomplishment as many scientists spend their careers without seeing their drug make it to market. Lee's team is particularly interested in therapies for brain metastases something that Lee says is a largely unmet need in cancer research, and something her team is working on from a variety of angles. "Now that we've had rapid success with mRNA vaccine technology, we hope to explore what the future holds when applying this technology to cancers," Lee says.

But while evaluating potential cancer therapies is a professional passion of Lee's, it's also a mission that's deeply personal. "I'm also a breast cancer survivor," she says. "So I've been on the other side of things and have participated in a clinical trial."

However, seeing how melanoma therapies that she helped develop have affected other real-life cancer patients, she says, has been a highlight of her career. "We had one therapy that was approved for patients with BRAF-mutant metastatic melanoma," Lee recalls. "Our team in Boulder was graced by a visit from a patient that had benefited from these drugs that we developed. It was a very special moment for the entire team."

None of these therapies would be available, Lee says without rigorous science behind it: "Facts come from good science. Facts will drive the development of new drugs, and that's what will help patients."

Photo courtesy of Cynthia Kuk

Cynthia Kuk was just 10 years old when they had a conversation that would change their life forever.

"My mother, who worked as a translator for the government at the time, had been diagnosed with breast cancer, and after her chemotherapy treatments she would get really sick," Kuk, who uses they/them pronouns, recalls. "When I asked my dad why mom was puking so much, he said it was because of the medicine she was taking that would help her get better."

Kuk's response was immediate: "That's so stupid! Why would a medicine make you feel worse instead of better? When I'm older, I want to create medicine that won't make people sick like that."

Nine years later, Kuk traveled from their native Hong Kong to the United States to do exactly that. Kuk enrolled in a small, liberal arts college for their Bachelor's degree, and then four years later started a PhD program in cancer research. Although Kuk's mother was in remission from her cancer at the time, Kuk's goal was the same as it had been as a 10-year-old watching her suffer through chemotherapy: to design a better cancer treatment, and change the landscape of cancer research forever.

Since then, Kuk's mission has changed slightly.

"My mom's cancer relapsed in 2008, and she ended up passing away about five years after that," Kuk says. "After my mom died, I started having this sense of urgency. Cancer research is such that you work for twenty years, and at the end of it you might have a fancy medication that could help people, but I wanted to help people now." With their mother still at the forefront of their mind, Kuk decided to quit their PhD program and enter medical school.

Now, Kuk plans to pursue a career in emergency medicine not only because they are drawn to the excitement of the emergency room, but because the ER is a place where the most marginalized people tend to seek care.

"I have a special interest in the LGBTQ+ population, as I identify as queer and nonbinary," says Kuk. "A lot of people in this community and other marginalized communities access care through the ER and also tend to avoid medical care since there is a history of mistreatment and judgement from healthcare workers. How you carry yourself as a doctor, your compassion, that can make a huge difference in someone's care."

In addition to making a difference in the lives of LGBTQ+ patients, Kuk wants to make a difference in the lives of patients with cancer as well, like their mother had.

"We've diagnosed patients in the Emergency Department with cancer before," Kuk says. "I can't make cancer good news but how you deliver bad news and the compassion you show could make a world of difference to that patient and their family."

During their training, Kuk advocates for patients by delivering compassionate and inclusive care, whether they happen to have cancer or not. In addition to emphasizing their patient's pronouns and chosen names, they ask for inclusive social and sexual histories as well as using gender neutral language. In doing this, they hope to make medicine as a whole more accessible for people who have been historically pushed aside.

"I'm just one person, and I can't force everyone to respect you, if you're marginalized," Kuk says. "But I do want to push for a culture where people appreciate others who are different from them."

View original post here:
'It's a miracle': Cat that a family thought they cremated turns back up at their home - Upworthy

Each year, an estimated 1.8 million people in the United States are affected by cancer most commonly cancers of the breast, lung, prostate, and blood cancers such as leukemia. While not everyone overcomes the disease, thanks to science, more people are surviving and for longer than ever before in history.

We asked three people whose lives have been impacted by cancer to share their stories how their lives were changed by the disease, and how they're using that experience to change the future of cancer treatments with the hope that ultimately, in the fight against cancer, science will win. Here's what they had to say.

Photo courtesy of Celine Ryan

In September 2013, Celine Ryan woke up from a colonoscopy to some traumatic news. Her gastroenterologist showed her a picture of the cancerous mass they found during the procedure.

Ryan and her husband, Patrick, had scheduled a colonoscopy after discovering some unusual bleeding, so the suspicion she could have cancer was already there. Neither of them, however, were quite prepared for the results to be positive -- or for the treatment to begin so soon. Just two days after learning the news, Ryan had surgery to remove the tumor, part of her bladder, and 17 cancerous lymph nodes. Chemotherapy and radiation soon followed.

Ryan's treatment was rigorous but in December 2014, she got the devastating news that the cancer, once confined to her colon, had spread to her lungs. Her prognosis, they said, was likely terminal.

But rather than give up hope, Ryan sought support from online research, fellow cancer patients and survivors, and her medical team. When she brought up immunotherapy to her oncologist, he quickly agreed it was the best course of action. Ryan's cancer, like a majority of colon and pancreatic cancers, had been caused by a defect on the gene KRAS, which can result in a very aggressive cancer that is virtually "undruggable." According to the medical literature, the relatively smooth protein structure of the KRAS gene meant that designing inhibitors to bind to surface grooves and treat the cancer has been historically difficult. Through her support systems, Ryan discovered an experimental immunotherapy trial at the National Institutes of Health (NIH) in Bethesda, MD., and called them immediately to see if she was eligible. After months of trying to determine whether she was a suitable candidate for the experimental treatment, Ryan was finally accepted.

The treatment, known as tumor-infiltrating lymphocyte therapy, or TIL, is a testament to how far modern science has evolved. With this therapy, doctors remove a tumor and harvest special immune cells that are found naturally in the tumor. Doctors then grow the cells in a lab over the next several weeks with a protein that promotes rapid TIL growth and once the cells number into the billions, they are infused back into the patient's body to fight the cancer. On April 1, 2015, Ryan had her tumor removed at the NIH. Two months later, she went inpatient for four weeks to have the team "wash out" her immune system with chemotherapy and infuse the cells all 148 billion of them back into her body.

Six weeks after the infusion, Ryan and Patrick went back for a follow-up appointment and the news they got was stunning: Not only had no new tumors developed, but the six existing tumors in her lungs had shrunk significantly. Less than a year after her cell infusion, in April 2016, the doctors told Ryan news that would have been impossible just a decade earlier: Thanks to the cell infusion, Ryan was now considered NED no evaluable disease. Her body was cancer-free.

Ryan is still NED today and continuing annual follow-up appointments at the NIH, experiencing things she never dreamed she'd be able to live to see, such as her children's high school and college graduations. She's also donating her blood and cells to the NIH to help them research other potential cancer treatments. "It was an honor to do so," Ryan said of her experience. "I'm just thrilled, and I hope my experience can help a lot more people."

Photo courtesy of Patrice Lee

Patrice Lee got into scientific research in an unconventional way through the late ocean explorer Jacques Cousteau.

Lee never met Cousteau but her dreams of working with him one day led her to pursue a career in science. Initially, Lee completed an undergraduate degree in marine biology; eventually, her interests changed and she decided to get a dual doctoral degree in physiology and toxicology at Duke University. She now works at Pfizer's R&D site in Boulder, CO (formerly Array BioPharma), leading a group of scientists who determine the safety and efficacy of new oncology drugs.

"Scientists focused on drug discovery and development in the pharmaceutical industry are deeply committed to inventing new therapies to meet unmet needs," Lee says, describing her field of work. "We're driven to achieve new medicines and vaccines as quickly as possible without sacrificing safety."

Among the drugs Lee has helped develop during her career, including cancer therapies, she says around a dozen are currently in development, while nine have received FDA approval an incredible accomplishment as many scientists spend their careers without seeing their drug make it to market. Lee's team is particularly interested in therapies for brain metastases something that Lee says is a largely unmet need in cancer research, and something her team is working on from a variety of angles. "Now that we've had rapid success with mRNA vaccine technology, we hope to explore what the future holds when applying this technology to cancers," Lee says.

But while evaluating potential cancer therapies is a professional passion of Lee's, it's also a mission that's deeply personal. "I'm also a breast cancer survivor," she says. "So I've been on the other side of things and have participated in a clinical trial."

However, seeing how melanoma therapies that she helped develop have affected other real-life cancer patients, she says, has been a highlight of her career. "We had one therapy that was approved for patients with BRAF-mutant metastatic melanoma," Lee recalls. "Our team in Boulder was graced by a visit from a patient that had benefited from these drugs that we developed. It was a very special moment for the entire team."

None of these therapies would be available, Lee says without rigorous science behind it: "Facts come from good science. Facts will drive the development of new drugs, and that's what will help patients."

Photo courtesy of Cynthia Kuk

Cynthia Kuk was just 10 years old when they had a conversation that would change their life forever.

"My mother, who worked as a translator for the government at the time, had been diagnosed with breast cancer, and after her chemotherapy treatments she would get really sick," Kuk, who uses they/them pronouns, recalls. "When I asked my dad why mom was puking so much, he said it was because of the medicine she was taking that would help her get better."

Kuk's response was immediate: "That's so stupid! Why would a medicine make you feel worse instead of better? When I'm older, I want to create medicine that won't make people sick like that."

Nine years later, Kuk traveled from their native Hong Kong to the United States to do exactly that. Kuk enrolled in a small, liberal arts college for their Bachelor's degree, and then four years later started a PhD program in cancer research. Although Kuk's mother was in remission from her cancer at the time, Kuk's goal was the same as it had been as a 10-year-old watching her suffer through chemotherapy: to design a better cancer treatment, and change the landscape of cancer research forever.

Since then, Kuk's mission has changed slightly.

"My mom's cancer relapsed in 2008, and she ended up passing away about five years after that," Kuk says. "After my mom died, I started having this sense of urgency. Cancer research is such that you work for twenty years, and at the end of it you might have a fancy medication that could help people, but I wanted to help people now." With their mother still at the forefront of their mind, Kuk decided to quit their PhD program and enter medical school.

Now, Kuk plans to pursue a career in emergency medicine not only because they are drawn to the excitement of the emergency room, but because the ER is a place where the most marginalized people tend to seek care.

"I have a special interest in the LGBTQ+ population, as I identify as queer and nonbinary," says Kuk. "A lot of people in this community and other marginalized communities access care through the ER and also tend to avoid medical care since there is a history of mistreatment and judgement from healthcare workers. How you carry yourself as a doctor, your compassion, that can make a huge difference in someone's care."

In addition to making a difference in the lives of LGBTQ+ patients, Kuk wants to make a difference in the lives of patients with cancer as well, like their mother had.

"We've diagnosed patients in the Emergency Department with cancer before," Kuk says. "I can't make cancer good news but how you deliver bad news and the compassion you show could make a world of difference to that patient and their family."

During their training, Kuk advocates for patients by delivering compassionate and inclusive care, whether they happen to have cancer or not. In addition to emphasizing their patient's pronouns and chosen names, they ask for inclusive social and sexual histories as well as using gender neutral language. In doing this, they hope to make medicine as a whole more accessible for people who have been historically pushed aside.

"I'm just one person, and I can't force everyone to respect you, if you're marginalized," Kuk says. "But I do want to push for a culture where people appreciate others who are different from them."

Read the original:
A wife saved her husband during his heart attack by singing the lyrics to 'Stayin' Alive' - Upworthy

Epilepsy is a common long-term brain condition. It causes seizures, which are bursts of electricity in the brain.

There are four main types of epilepsy: focal, generalized, combination focal and generalized, and unknown. A persons seizure type determines what kind of epilepsy they have.

Different types of seizures affect the brain in different ways. For example, focal seizures affect only one part of the brain, whereas generalized seizures affect the entire brain.

To be categorized as having epilepsy, a person must experience two or more unprovoked seizures. Some people can receive an epilepsy diagnosis if they have had one seizure and a doctor thinks they have a high likelihood of having another.

Read on to learn more about the different types of epilepsy and how to manage them.

Epilepsy is a neurological disorder. Its primary identifying factor is recurrent, unprovoked seizures.

Abnormal electrical activity in the brain causes seizures. This brain activity affects how a person feels, acts, and behaves. Depending on the seizure type and severity, a person may or may not lose consciousness.

Before doctors can diagnose a person with epilepsy, they need to decide if a seizure is provoked or unprovoked.

Many things cause seizures. These include head injuries, toxins, tumors, and infections. Doctors must rule out these potential causes before diagnosing someone with epilepsy.

According to the Centers for Disease Control and Prevention (CDC), there are 3.4 million adults and children with epilepsy in the United States. Although it is common, doctors are still finding out more about this chronic disorder.

There are several types of seizures. A person with epilepsy can experience one or multiple types of seizure.

The three primary seizure types are:

The four different types of epilepsy are defined by the type of seizure a person experiences. They are:

Each type of epilepsy affects the brain differently. This means they have different identifying factors and treatments.

People with this type of epilepsy have generalized seizures. These affect both the left and right sides of the brain. Additionally, these seizures may be either motor, which involve physical movement, or non-motor, which do not.

If someone has a motor seizure, they may experience:

Non-motor seizures are also called absence seizures. Symptoms may include:

Generalized epilepsy usually starts during childhood. However, it can also affect adults.

Learn more about epilepsy in children.

People with focal epilepsy have focal seizures. Unlike generalized seizures, focal seizures only affect one part of the brain. They can start in one area and move to others.

These seizures can begin with an aura, which are minor symptoms signifying the seizures onset. This can feel like an uneasy feeling in the stomach, similar to the feeling of riding a rollercoaster.

As the seizure progresses, a person can experience motor and non-motor symptoms. Some motor symptoms of focal seizures include:

Non-motor symptoms do not affect how someone moves. However, they may cause confusion or changes in emotions. Some non-motor symptoms of focal seizures include:

Learn more about focal seizures.

Someone with combination epilepsy has both generalized seizures and focal seizures. Therefore, they can experience a mixture of the symptoms discussed above.

Combined epilepsy is linked to Dravet syndrome, which is a rare, lifelong form of epilepsy. It is usually caused by a mutation in the SCN1A gene. Because it is often misdiagnosed, people who think they or a family member may have these seizures should contact a doctor.

If doctors do not know where seizures originate, they will diagnose a person with unknown epilepsy.

People with unknown epilepsy can have a combination of motor and non-motor symptoms. Motor seizures often present as tonic-clonic (previously referred to as grand-mal). These seizures can have the following symptoms:

These seizures usually last 13 minutes. If they last more than 5 minutes, call emergency services immediately.

Unknown epilepsy also presents with non-motor symptoms. These can include:

Learn more about tonic-clonic seizures.

Although epilepsy is a seizure disorder, this does not mean that every seizure is a sign of epilepsy.

A person can have provoked seizures, which are seizures due to a cause other than epilepsy. Some examples of things that could induce a seizure are:

If a seizure is solely due to one of these causes, the individual does not have epilepsy.

However, if none of these possibilities prompted the seizure, the person may have epilepsy. To make an epilepsy diagnosis, doctors must first find out if someone has had a seizure. Doctors then determine what type of seizure it was.

Doctors can determine whether a person meets the diagnostic criteria through medical history details, EEG tests, blood tests, and brain imaging tests such as a CT scan or MRI.

There are different types of treatments for epilepsy.

Doctors typically use medication to control and stop epileptic seizures. Some drugs work for only one type of seizure, while others can control various seizure types.

A doctor prescribes medications based on a persons seizure type, medical history, and age. If the medication does not help someones epilepsy, doctors may prescribe a different drug in place of, or combined with, the first medication.

Most people who have epilepsy have a good response to this form of treatment.

Some people have drug-resistant epilepsy. This means they cannot control their epilepsy using the first two medications prescribed. Around 33% of adults and 2025% of children with epilepsy do not respond to their first-line treatment and must consider other options.

A doctor will discuss various treatments a person can try. These may include:

Surgery: This option typically works best for people who have seizures originating from one part of the brain. It involves safely removing the focal point, or the part of the brain where the seizures start.

Dietary changes: Some diets may help control seizures. Recommended diets include the modified Atkins diet, ketogenic diet, and low glycemic diet. These diets should be carried out with support from a registered dietitian.

Vagus nerve stimulation (VNS): This therapy treats people with focal seizures. It works by sending mild electrical pulses through the vagus nerve, which leads to the brain. Over time, it changes how brain cells work.

Other options, like behavioral therapy and CBD oil, may help with treating drug-resistant epilepsy.

Learn more about natural remedies for epilepsy.

People with epilepsy must be consistent with their medication and/or treatment regimen. They should also try to avoid seizure triggers. Because triggers vary from person to person, a person can keep a diary of seizures to record possible triggers.

Children with an epilepsy diagnosis often outgrow it with age. For those whose epilepsy continues into adulthood, or people diagnosed later in life, it is very possible to live a normal life with epilepsy. Two-thirds of adults with epilepsy no longer experience seizures as a result of an effective treatment plan.

Learn more about epilepsy in children.

Anyone who suspects they have had a seizure should seek medical attention. A doctor can determine what caused the seizure, the type of seizure it was, and discuss appropriate next steps.

In many cases, epilepsy can be effectively treated and managed with seizure medication. Receiving an accurate and timely diagnosis is essential.

Epilepsy is a common seizure disorder. There are four main types of epilepsy: focal, generalized, combination focal and generalized, and unknown.

A doctor generally diagnoses someone with epilepsy if they have had two or more unprovoked seizures.

Medication is the most common treatment, and two-thirds of adults with epilepsy live seizure-free because of it. If medication does not work, other treatments are available. These include surgery, brain stimulation, and a modified diet.

People with epilepsy must be consistent with their medication and visit a doctor if their seizures appear to worsen.

Although it is uncommon for epilepsy to go away on its own, proper treatment can control the seizures. It is very possible to live a normal, full life with epilepsy.

See original here:
4 types of epilepsy, their symptoms, and treatments - Medical News Today

Each year, an estimated 1.8 million people in the United States are affected by cancer most commonly cancers of the breast, lung, prostate, and blood cancers such as leukemia. While not everyone overcomes the disease, thanks to science, more people are surviving and for longer than ever before in history.

We asked three people whose lives have been impacted by cancer to share their stories how their lives were changed by the disease, and how they're using that experience to change the future of cancer treatments with the hope that ultimately, in the fight against cancer, science will win. Here's what they had to say.

Photo courtesy of Celine Ryan

In September 2013, Celine Ryan woke up from a colonoscopy to some traumatic news. Her gastroenterologist showed her a picture of the cancerous mass they found during the procedure.

Ryan and her husband, Patrick, had scheduled a colonoscopy after discovering some unusual bleeding, so the suspicion she could have cancer was already there. Neither of them, however, were quite prepared for the results to be positive -- or for the treatment to begin so soon. Just two days after learning the news, Ryan had surgery to remove the tumor, part of her bladder, and 17 cancerous lymph nodes. Chemotherapy and radiation soon followed.

Ryan's treatment was rigorous but in December 2014, she got the devastating news that the cancer, once confined to her colon, had spread to her lungs. Her prognosis, they said, was likely terminal.

But rather than give up hope, Ryan sought support from online research, fellow cancer patients and survivors, and her medical team. When she brought up immunotherapy to her oncologist, he quickly agreed it was the best course of action. Ryan's cancer, like a majority of colon and pancreatic cancers, had been caused by a defect on the gene KRAS, which can result in a very aggressive cancer that is virtually "undruggable." According to the medical literature, the relatively smooth protein structure of the KRAS gene meant that designing inhibitors to bind to surface grooves and treat the cancer has been historically difficult. Through her support systems, Ryan discovered an experimental immunotherapy trial at the National Institutes of Health (NIH) in Bethesda, MD., and called them immediately to see if she was eligible. After months of trying to determine whether she was a suitable candidate for the experimental treatment, Ryan was finally accepted.

The treatment, known as tumor-infiltrating lymphocyte therapy, or TIL, is a testament to how far modern science has evolved. With this therapy, doctors remove a tumor and harvest special immune cells that are found naturally in the tumor. Doctors then grow the cells in a lab over the next several weeks with a protein that promotes rapid TIL growth and once the cells number into the billions, they are infused back into the patient's body to fight the cancer. On April 1, 2015, Ryan had her tumor removed at the NIH. Two months later, she went inpatient for four weeks to have the team "wash out" her immune system with chemotherapy and infuse the cells all 148 billion of them back into her body.

Six weeks after the infusion, Ryan and Patrick went back for a follow-up appointment and the news they got was stunning: Not only had no new tumors developed, but the six existing tumors in her lungs had shrunk significantly. Less than a year after her cell infusion, in April 2016, the doctors told Ryan news that would have been impossible just a decade earlier: Thanks to the cell infusion, Ryan was now considered NED no evaluable disease. Her body was cancer-free.

Ryan is still NED today and continuing annual follow-up appointments at the NIH, experiencing things she never dreamed she'd be able to live to see, such as her children's high school and college graduations. She's also donating her blood and cells to the NIH to help them research other potential cancer treatments. "It was an honor to do so," Ryan said of her experience. "I'm just thrilled, and I hope my experience can help a lot more people."

Photo courtesy of Patrice Lee

Patrice Lee got into scientific research in an unconventional way through the late ocean explorer Jacques Cousteau.

Lee never met Cousteau but her dreams of working with him one day led her to pursue a career in science. Initially, Lee completed an undergraduate degree in marine biology; eventually, her interests changed and she decided to get a dual doctoral degree in physiology and toxicology at Duke University. She now works at Pfizer's R&D site in Boulder, CO (formerly Array BioPharma), leading a group of scientists who determine the safety and efficacy of new oncology drugs.

"Scientists focused on drug discovery and development in the pharmaceutical industry are deeply committed to inventing new therapies to meet unmet needs," Lee says, describing her field of work. "We're driven to achieve new medicines and vaccines as quickly as possible without sacrificing safety."

Among the drugs Lee has helped develop during her career, including cancer therapies, she says around a dozen are currently in development, while nine have received FDA approval an incredible accomplishment as many scientists spend their careers without seeing their drug make it to market. Lee's team is particularly interested in therapies for brain metastases something that Lee says is a largely unmet need in cancer research, and something her team is working on from a variety of angles. "Now that we've had rapid success with mRNA vaccine technology, we hope to explore what the future holds when applying this technology to cancers," Lee says.

But while evaluating potential cancer therapies is a professional passion of Lee's, it's also a mission that's deeply personal. "I'm also a breast cancer survivor," she says. "So I've been on the other side of things and have participated in a clinical trial."

However, seeing how melanoma therapies that she helped develop have affected other real-life cancer patients, she says, has been a highlight of her career. "We had one therapy that was approved for patients with BRAF-mutant metastatic melanoma," Lee recalls. "Our team in Boulder was graced by a visit from a patient that had benefited from these drugs that we developed. It was a very special moment for the entire team."

None of these therapies would be available, Lee says without rigorous science behind it: "Facts come from good science. Facts will drive the development of new drugs, and that's what will help patients."

Photo courtesy of Cynthia Kuk

Cynthia Kuk was just 10 years old when they had a conversation that would change their life forever.

"My mother, who worked as a translator for the government at the time, had been diagnosed with breast cancer, and after her chemotherapy treatments she would get really sick," Kuk, who uses they/them pronouns, recalls. "When I asked my dad why mom was puking so much, he said it was because of the medicine she was taking that would help her get better."

Kuk's response was immediate: "That's so stupid! Why would a medicine make you feel worse instead of better? When I'm older, I want to create medicine that won't make people sick like that."

Nine years later, Kuk traveled from their native Hong Kong to the United States to do exactly that. Kuk enrolled in a small, liberal arts college for their Bachelor's degree, and then four years later started a PhD program in cancer research. Although Kuk's mother was in remission from her cancer at the time, Kuk's goal was the same as it had been as a 10-year-old watching her suffer through chemotherapy: to design a better cancer treatment, and change the landscape of cancer research forever.

Since then, Kuk's mission has changed slightly.

"My mom's cancer relapsed in 2008, and she ended up passing away about five years after that," Kuk says. "After my mom died, I started having this sense of urgency. Cancer research is such that you work for twenty years, and at the end of it you might have a fancy medication that could help people, but I wanted to help people now." With their mother still at the forefront of their mind, Kuk decided to quit their PhD program and enter medical school.

Now, Kuk plans to pursue a career in emergency medicine not only because they are drawn to the excitement of the emergency room, but because the ER is a place where the most marginalized people tend to seek care.

"I have a special interest in the LGBTQ+ population, as I identify as queer and nonbinary," says Kuk. "A lot of people in this community and other marginalized communities access care through the ER and also tend to avoid medical care since there is a history of mistreatment and judgement from healthcare workers. How you carry yourself as a doctor, your compassion, that can make a huge difference in someone's care."

In addition to making a difference in the lives of LGBTQ+ patients, Kuk wants to make a difference in the lives of patients with cancer as well, like their mother had.

"We've diagnosed patients in the Emergency Department with cancer before," Kuk says. "I can't make cancer good news but how you deliver bad news and the compassion you show could make a world of difference to that patient and their family."

During their training, Kuk advocates for patients by delivering compassionate and inclusive care, whether they happen to have cancer or not. In addition to emphasizing their patient's pronouns and chosen names, they ask for inclusive social and sexual histories as well as using gender neutral language. In doing this, they hope to make medicine as a whole more accessible for people who have been historically pushed aside.

"I'm just one person, and I can't force everyone to respect you, if you're marginalized," Kuk says. "But I do want to push for a culture where people appreciate others who are different from them."

Read the rest here:
Confused? This is exactly how the federal government's new Child Tax Credit works. - Upworthy

New Delhi: Experts are forewarning against the third wave of Covid-19 hitting India as early as September, with many fearing that it could hit children disproportionately. According to Rekha Mittal, Neurologist at Rainbow Hospitals, since many adults would have had the disease or the Covid vaccine, in comparison children would be a susceptible population.

Third wave and more waves can always occur in a pandemic till it burns out or there is herd immunity. So far in our experience, the majority of children who have had Covid infection have either been asymptomatic or had mild symptoms. Therefore we hope the next wave will not be a serious threat to children, Mittal told IANSlife.

The treatment of children who get serious manifestations of Covid requires specialists who are trained in intensive care of children, and the appropriate equipment and support staff. We should definitely plan and be prepared for a crisis situation if it does occur. We should not be complacent.

How can a surge in cases be avoided? The expert suggests not letting our guard down, by maintaining Covid-appropriate behaviour such as social distancing, use of masks, and hand sanitisation measures. We need to avoid crowded places such as malls, markets, gatherings etc. Also, the children will get protected indirectly, if the adults around them receive the Covid vaccine.

Rainbow Hospitals successfully administered the wonder drug Zolgensma, which is also the worlds costliest at Rs 16 crore per shot, to a three-year-old Hyderabad boy this month. The medical marvel Zolgensma is a single dose intravenous injection used in gene therapy is for replacing the defective SMN1 gene through an adenoviral vector.

Asked how critical can it be to administer the expensive injection like this with skill, Mittal says: Administering such an expensive and uncommonly used drug can be a challenge. One will need to have complete knowledge about procuring, handling and administering the medication. Also, one would have to have all facilities to handle the side effects if they occur, such as liver damage, drop in platelet count etc.

Speaking about vaccination for children, the doctor said vaccinations for vaccine preventable diseases should continue on time for childrens as per schedule.

Rainbow hospital provides a safe area where children can be brought in for vaccinations. Trials for Covid vaccination in children above 2 years of age have started in India. As and when Covid vaccine is approved and available for children, Rainbow hospitals will be ready to administer the same to as many children as possible.

(IANS)

See the original post:
Pediatric healthcare and third COVID-19 wave - Sambad English

AstraZenecaand MSD'sKoselugo(selumetinib) has been granted conditional approval in the European Union (EU) for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in paediatric patients with neurofibromatosis type 1 (NF1) aged three years and above.

NF1 is a debilitating genetic condition affecting one in 3,000 individuals worldwide.1,2In 30-50% of people with NF1, tumours develop on the nerve sheaths (plexiform neurofibromas) and can cause clinical issues such as disfigurement, motor dysfunction, pain, airway dysfunction, visual impairment and bladder or bowel dysfunction.3-7

The approval by the European Commission was based on positive results from the SPRINT Stratum 1 Phase II trial sponsored by the National Institute of Health's National Cancer Institute (NCI) Cancer Therapy Evaluation Program (CTEP). This trial showedKoselugoreduced the size of inoperable tumours in children, reducing pain and improving quality of life.7,8This is the first approval of a medicine for NF1 PN in the EU and follows the positiverecommendationby the Committee for Medicinal Products for Human Use of the European Medicines Agency in April 2021. Safety and efficacy data from the SPRINT Phase II trial with longer follow up will be provided as one of the conditions of approval.

Brigitte C. Widemann, MD, Principal Investigator of the SPRINT trial and Chief, NCI Pediatric Oncology Branch, said: "For children with neurofibromatosis type 1, plexiform neurofibromas can grow and develop so significantly that, in some cases, it becomes debilitating. In the SPRINT trial, selumetinib shrank NF1-associated PNs in 66% of patients and showed clinically meaningful improvements in PN-related symptoms."

Dave Fredrickson, Executive Vice President, Oncology Business Unit, said: "As the first medicine approved in the EU for patients with neurofibromatosis type 1,Koselugohas the potential to transform the way plexiform neurofibromas are managed and treated. The SPRINT data showed thatKoselugonot only shrank tumours in some children, but also reduced pain and improved their quality of life. This significant milestone was made possible thanks to our research partners, the National Cancer Institute, the Neurofibromatosis Therapeutic Acceleration Program, the Children's Tumor Foundation, the patient community and every child, parent and doctor involved in the clinical trial."

Roy Baynes, Senior Vice President and Head of Global Clinical Development, Chief Medical Officer, MSD Research Laboratories, said: "Before this approval, surgery was the only treatment option for children in the EU with neurofibromatosis type 1 plexiform neurofibromas. This approval marks a significant step forward in addressing the debilitating impact of these tumours."

The SPRINT Stratum 1 Phase II trial showedKoselugodemonstrated an objective response rate (ORR) of 66% (33 of 50 patients, confirmed partial response) in paediatric patients with NF1 PN when treated withKoselugoas twice-daily oral monotherapy.8ORR is defined as the percentage of patients with confirmed complete (disappearance of PN) or partial response (at least 20% reduction in tumour volume).8Results were published inThe New England Journal of Medicine.7

Koselugoisapprovedin the US and several other countries for the treatment of paediatric patients with NF1 and symptomatic, inoperable PN. Further regulatory submissions are underway. Clinical trials ofKoselugoin adult patients with NF1 PN, including an alternative age-appropriate formulation for paediatric patients, are scheduled to begin this year.

NF1NF1 is caused by a spontaneous or inherited mutation in the NF1 gene and is associated with many symptoms, including soft lumps on and under the skin (cutaneous neurofibromas) and skin pigmentation (so-called 'caf au lait' spots). In 30-50% of people, tumours develop on the nerve sheaths.1,3,9,10These PN can cause clinical issues such as pain, motor dysfunction, airway dysfunction, bladder/bowel dysfunction and disfigurement, as well as having the potential to transform into malignant peripheral nerve sheath tumours.4-7,10PN begin developing during early childhood, with varying degrees of severity, and can reduce life expectancy by eight to 15 years.3,6,11,12

SPRINTThe SPRINT Stratum 1 Phase II trial was designed to evaluate the objective response rate and impact on patient-reported and functional outcomes in paediatric patients with NF1-related inoperable PNs treated with selumetinib monotherapy.7This trial sponsored by NCI CTEP was conducted under a Cooperative Research and Development Agreement between NCI and AstraZeneca with additional support from Neurofibromatosis Therapeutic Acceleration Program (NTAP).

KoselugoKoselugo(selumetinib) is an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2).8MEK1/2 proteins are upstream regulators of the extracellular signal-related kinase (ERK) pathway. Both MEK and ERK are critical components of the RAS-regulated RAF-MEK-ERK pathway, which is often activated in different types of cancers.13

Koselugoreceived US FDA Breakthrough Therapy Designation in April 2019, Rare Pediatric Disease Designation in December 2019 and US Orphan Drug Designation in February 2018. Further orphan designations have been granted in the EU, Japan, Russia, Switzerland, South Korea, Taiwan and Australia.

AstraZeneca and MSD strategic oncology collaborationIn July 2017, AstraZeneca and Merck & Co., Inc., Kenilworth, NJ, US, known as MSD outside the US and Canada, announced a global strategic oncology collaboration to co-develop and co-commercialiseLynparza, the world's first PARP inhibitor, andKoselugo(selumetinib), amitogen-activated protein kinase (MEK)inhibitor, for multiple cancer types. Working together, the companies will developLynparzaandKoselugoin combination with other potential new medicines and as monotherapies. Independently, the companies will developLynparzaandKoselugoin combination with their respective PD-L1 and PD-1 medicines.

AstraZeneca in oncologyAstraZeneca is leading a revolution in oncology with the ambition to provide cures for cancer in every form, following the science to understand cancer and all its complexities to discover, develop and deliver life-changing medicines to patients.

The Company's focus is on some of the most challenging cancers. It is through persistent innovation that AstraZeneca has built one of the most diverse portfolios and pipelines in the industry, with the potential to catalyse changes in the practice of medicine and transform the patient experience.

AstraZeneca has the vision to redefine cancer care and, one day, eliminate cancer as a cause of death.

AstraZenecaAstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led biopharmaceutical company that focuses on the discovery, development and commercialisation of prescription medicines in Oncology and BioPharmaceuticals, including Cardiovascular, Renal & Metabolism, and Respiratory & Immunology. Based in Cambridge, UK, AstraZeneca operates in over 100 countries, and its innovative medicines are used by millions of patients worldwide. Please visitastrazeneca.comand follow the Company on Twitter@AstraZeneca.

ContactsFor details on how to contact the Investor Relations Team, please clickhere. For Media contacts, clickhere.

References

1. Cancer.Net. Neurofibromatosis Type 1. Available at:https://www.cancer.net/cancer-types/neurofibromatosis-type 1. Accessed June 2021.

2. National Human Genome Research Institute. About Neurofibromatosis. Available at:https://www.genome.gov/Genetic-Disorders/Neurofibromatosis. Accessed June 2021.

3. Hirbe AC, Gutmann DH. Neurofibromatosis type 1: a multidisciplinary approach to care.Lancet Neurol. 2014;13:834-43. doi: 10.1016/S1474-4422(14)70063-8.

4. Dombi E, Baldwin A, Marcus LJ, et al. Activity of selumetinib in neurofibromatosis type 1-related plexiform neurofibromas.N Engl J Med. 2016;375:2550-2560. doi: 10.1056/NEJMoa1605943.

5. Mayo Clinic. Neurofibromatosis. Available at:https://www.mayoclinic.org/diseases-conditions/neurofibromatosis/symptoms-causes/syc-20350490. Accessed June 2021.

6. NHS. Neurofibromatosis Type 1, Symptoms. Available athttps://www.nhs.uk/conditions/neurofibromatosis-type 1/symptoms. Accessed June 2021.

7. Gross AM, et al. Selumetinib in Children with Inoperable Plexiform Neurofibromas.N Engl J Med. 2020 Apr 9;382(15):1430-1442. doi: 10.1056/NEJMoa1912735.

8. European Medicines Agency.Koselugosummary of product characteristics. Accessed June 2021.

9. Jett K, Friedman JM. Clinical and genetic aspects of neurofibromatosis 1.Genet Med. 2010:12(1):1-11. doi: 10.1097/GIM.0b013e3181bf15e3. PMID: 20027112.

10. Ghalayani P, Saberi Z, Sardari, F. Neurofibromatosis Type I (von Recklinghausen's Disease): A Family Case Report and Literature Review.Dent Res J. 2012;9(4):483-488.

11. Evans DGR, Ingham SL. Reduced Life Expectancy Seen in Hereditary Diseases Which Predispose to Early Onset Tumors.Appl Clin Genet. 2013;6:53-61.

12. NIH National Institute of Neurological Disorders and Stroke. Neurofibromatosis Fact Sheet. Available at:https://www.ninds.nih.gov/disorders/patient-caregiver-education/fact-sheets/neurofibromatosis-fact-sheet. Accessed June 2021.

13.Koselugo(selumetinib) [prescribing information]. Wilmington, DE: AstraZeneca Pharmaceuticals LP; 2020.

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Koselugo approved in the EU for children with neurofibromatosis type 1 and plexiform neurofibromas - PharmiWeb.com

Reparative stem cells have the capability to restore function to damaged tissue by renewing cell growth (shown in green) in cardiac cells destroyed by heart disease.

Approximately 28 million Americans have been diagnosed with heart disease. Traditional medical therapies are not able to fully address the burden of disease, and the shortage of organs for transplantation remains a key barrier more than 117,000 people are on the national transplant list.

This unmet need drives Mayo Clinic researchers to make new discoveries to accelerate regenerative solutions into clinical trials and rapidly provide new hope to patients who can't currently be treated.

Cardiac regeneration is a broad effort that aims to repair irreversibly damaged heart tissue with cutting-edge science, including stem cell and cell-free therapy. Reparative tools have been engineered to restore damaged heart tissue and function using the body's natural ability to regenerate. Working together, patients and providers are finding regenerative solutions that restore, renew and recycle patients' own reparative capacity. Through the vision and generous support of Russ and Kathy Van Cleve, strong efforts are underway to develop discoveries that will have a global impact on ischemic heart disease.

Mayo Clinic researchers are leading efforts in translating new knowledge into applicable therapeutics through a multidisciplinary community of practice. As technology evolves, it offers the potential to regenerate cardiac tissue from noncardiac sources and ultimately provide personalized products and services to people with cardiovascular disease.

The overarching vision for the cardiac regeneration program at Mayo Clinic is to develop new therapies to cure ischemic heart disease. Mayo researchers are developing products for clinical testing that span the disease spectrum, including the following areas:

More information about cardiac regenerative medicine research at Mayo Clinic is on the Van Cleve Cardiac Regenerative Medicine Program website.

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Key global participants in the Autologous Stem Cell Based Therapies market include:Med cell Europe US STEM CELL, INC. Tigenix Mesoblast Pluristem Therapeutics Inc Brainstorm Cell Therapeutics Regeneus

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Segmentation on the Basis of Application:Neurodegenerative Disorders Autoimmune Diseases Cardiovascular Diseases

Market Segments by TypeEmbryonic Stem Cell Resident Cardiac Stem Cells Umbilical Cord Blood Stem Cells

Table of Content1 Report Overview1.1 Product Definition and Scope1.2 PEST (Political, Economic, Social and Technological) Analysis of Autologous Stem Cell Based Therapies Market2 Market Trends and Competitive Landscape3 Segmentation of Autologous Stem Cell Based Therapies Market by Types4 Segmentation of Autologous Stem Cell Based Therapies Market by End-Users5 Market Analysis by Major Regions6 Product Commodity of Autologous Stem Cell Based Therapies Market in Major Countries7 North America Autologous Stem Cell Based Therapies Landscape Analysis8 Europe Autologous Stem Cell Based Therapies Landscape Analysis9 Asia Pacific Autologous Stem Cell Based Therapies Landscape Analysis10 Latin America, Middle East & Africa Autologous Stem Cell Based Therapies Landscape Analysis 11 Major Players Profile

This market study also includes a geographical analysis of the world market, which includes North America, Europe, Asia Pacific, the Middle East, and Africa, as well as several other important regions that dominate the world market. The Market study highlights some of the most important resources that can assist in achieving high profits in the firm. This Autologous Stem Cell Based Therapies market report also identifies market opportunities, which will aid stakeholders in making investments in the competitive landscape and a few product launches by industry players at the regional, global, and company levels. As numerous successful ways are offered in the study, it becomes possible to expand your firm. By referring to this one-of-a-kind market study, one can achieve business stability. With the help of this Market Research Study, you may achieve crucial positions in the whole market. It does a thorough market analysis for the forecast period of 2021-2027.

Autologous Stem Cell Based Therapies Market Intended Audience: Autologous Stem Cell Based Therapies manufacturers Autologous Stem Cell Based Therapies traders, distributors, and suppliers Autologous Stem Cell Based Therapies industry associations Product managers, Autologous Stem Cell Based Therapies industry administrator, C-level executives of the industries Market Research and consulting firms

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Autologous Stem Cell Based Therapies Market to Eyewitness Huge Growth by 2027 with Covid-19 Impact The Manomet Current - The Manomet Current

The Autologous Stem Cell Based Therapies Market is expected to grow at a CAGR of 8.98% and is poised to reach US$XX Billion by 2027 as compared to US$XX Billion in 2020. The factors leading to this extraordinary growth is attributed to various market dynamics discussed in the report. Our experts have examined the market from a 360 degree perspective thereby producing a report which is definitely going to impact your business decisions.

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The market research report by DECISIVE MARKETS INSIGHTS looks at several important elements that affect the global industrys development. This research includes a concise explanation of all the variables impacting these market participants growth, as well as information about their organizations, business models, marketing strategies, operational activities, technological integrations, and more. The top competitors in the global industry are included in this market analysis. In addition, the study includes mergers and acquisitions that have improved the product portfolios of various companies. The research provides an in-depth examination of several customer journeys that are relevant to the Autologous Stem Cell Based Therapies market and its sectors. It provides a variety of client perspectives on the products and services.

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Key Companies Operating in this Market

Regeneus, Mesoblast, Pluristem Therapeutics Inc, US STEM CELL, INC., Brainstorm Cell Therapeutics, Tigenix, Med cell Europe

Key Highlights of the Autologous Stem Cell Based Therapies Market Report

Market Segments and other perspective have been studied across 360 degree perspective Both Supply and Demand side mapping has been done to understand the market scenario We have used data triangulation to derive the market numbers Our data and analysis have been verified through C-level Executives while conducting primary interviews Porters Five Forces Analysis, Swot, Analysis, PEST Analysis, Value Chain Analysis and Market Attractiveness would be an added advantage in the report Market Size is Provided from 2019 to 2027; whereas CAGR is Provided from 2020 to 2027 Historical Year: 2019; Base Year: 2020; Forecast Years: 2020 2027

Market Segmentation and Scope of the Global Autologous Stem Cell Based Therapies Market

Market by TypeEmbryonic Stem CellResident Cardiac Stem CellsUmbilical Cord Blood Stem Cells

Market by ApplicationNeurodegenerative DisordersAutoimmune DiseasesCardiovascular Diseases

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What are the greatest investment options for expanding into new product and service categories? What value propositions should companies aim for when investing in new research & innovation? Which legislation will assist stakeholders to improve their supply chain network the most? Which regions could see consumption in particular segments mature shortly? What are some of the greatest vendor cost management tactics that some well-established players have found to be successful?

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Autologous Stem Cell Based Therapies Market 2021 Industry Statistics, Applications, Forecast 2026, and Key Player Analysis- Regeneus, Mesoblast,...

The treatment of patients with newly diagnosed multiple myeloma is an evolving area, said Pianko. The drug combinations we have now are highly effective, but many of the options coming [down the pike] could allow us to provide deeper responses for patients. Data supporting the use of quadruplet regimens for [this patient population] are also coming.

Future trials will help us [determine] which of the triplet backbones will be the best partner for a CD38-based quadruplet regimen. However, before quadruplets can really be considered a true new standard of care, more data are required, added Pianko, a clinical assistant professor at the University of Michigan Health.

During the 2021 Institutional Perspectives in Cancer webinar on multiple myeloma, faculty from the University of Michigan Health zeroed in on therapeutic updates in the frontline and relapsed settings and how more novel approaches, including CAR T-cell therapy, are shifting best practices in the paradigm. Pianko, who chaired the event, noted that the webinar had a key role in helping to establish and connect the academic institution with local referring community providers to discuss cutting edge developments in myeloma treatment.

In an interview with OncLive, Pianko reflected on the abovementioned topicsspecifically how he approaches patients with newly diagnosed multiple myeloma, and differentiating options based on transplant eligibility, as well as which emerging immune-based therapies he is most intrigued by.

Pianko: My approach to the treatment of [patients with] newly diagnosed multiple myeloma incorporates a very patient-centric [tactic]. I look at multiple factors specific to the patient, which can help to guide treatment decisions, [including] age, other medical conditions, cardiovascular risk, pre-existing neuropathy, and transplant eligibility. These factors play into how we select treatment.

Recent data from several trials have allowed for multiple choices in the frontline setting that would be appropriate for both transplant-eligible and -ineligible patients with multiple myeloma. Largely, tailoring therapy to a specific patient is becoming more possible with much of the data we have in newly diagnosed multiple myeloma.

For the transplant-eligible population, our current practice is to generally use triplet regimens, [such as] bortezomib [Velcade], lenalidomide [Revlimid], and dexamethasone [VRd] or carfilzomib [Kyprolis], lenalidomide, and dexamethasone [KRd]. The patients age, cytogenetic risk, and pre-existing neuropathy can help us to choose [between these triplet regimens].

The ENDURANCE trial [NCT01863550] was a large, randomized, phase 3 study that compared VRd with KRd and showed that VRd was not superior to KRd. The study highlighted that there is a known difference in the adverse effect [AE] profiles of these [triplets]. The patients getting VRd had a high incidence of treatment discontinuation [because of] treatment-related AEs, including peripheral neuropathy, which is associated with bortezomib. In the KRd combination, high incidences of cardiac, pulmonary, and renal toxicities [were observed].

Largely, there [doesnt] seem to be a difference in terms of progression-free survival [PFS] between the 2 groups, but we did see a difference in the AE profiles. The basis for choosing one [triplet] over the other can be guided by the expected AEs and driven by the [individual] patient.

In my practice, I tend to favor KRd in young patients with newly diagnosed multiple myeloma without significant medical comorbidities and independent of cytogenetic risk. [This is] because of the peripheral neuropathy bortezomib [can cause] that can be permanent. For many patients who have a life expectancy of potentially at least 1 decade, the cumulative quality-of-life burden of daily pain from peripheral neuropathy is a significant issue to consider.

My discussion with my patients often discusses the risk of peripheral neuropathy and cardiopulmonary AEs from carfilzomib. Ultimately, after discussing [these risks] with the patient, we together choose which [treatment] is the most appropriate way forward.

In the transplant-ineligible patient population, there are younger patients who have medical comorbidities that might preclude a transplant, and we have our older patients. Generally, [transplant ineligibility] is in the range of 75 to 80 years old. That is when we could classify someone as being potentially transplant ineligible but [we need to consider] geriatric and frailty assessments that can help guide us.

For patients who are intermediate-fit or frail, we might consider a doublet regimen, such as lenalidomide plus dexamethasone [Rd]. The inclusion of daratumumab to this doublet [based on] the MAIA trial [NCT02252172] showed us that [Rd plus daratumumab] is a viable approach for patients with newly diagnosed, transplant-ineligible multiple myeloma. The toxicity profile of daratumumab pairs well [with Rd] in this [patient population] to be [considered] a potential standard of care.

Other regimens include the VRd-lite regimen, which uses a modified dose and schedule for bortezomib and lenalidomide. That is another option for our transplant-ineligible patients.

A study was recently published looking at a modified schedule of lenalidomide/dexamethasone where patients would get [the doublet] for 9 cycles and then were able to de-escalate [treatment] and drop the dexamethasone. It seems like this approach is another viable option for our transplant-ineligible patients, particularly those with intermediate-fit or frail disease.

The take-home message is that there are multiple options that we can choose from in both the transplant-eligible and -ineligible patient populations.

The role of daratumumab in the frontline setting for multiple myeloma is an evolving one. Based on the MAIA trial, Rd plus daratumumab is now an FDA-approved regimen for patients with newly diagnosed, transplant-ineligible multiple myeloma. We have seen promising early data from the GRIFFIN study [NCT02874742], which looked at the quadruplet combination of daratumumab plus VRd vs VRd alone.

We saw [from the GRIFFIN trial] that the addition of daratumumab to this regimen was another viable approach. We saw superior depth of response and higher rates of stringent complete responses in up to 60% of patients [treated with] daratumumab plus VRd vs about 20% for the triplet regimen. We also saw a higher degree of minimal residual disease [MRD]negative disease with the quadruplet [compared with the triplet]. The safety profile [with the quadruplet] was acceptable, and namely, the stem cell collection did not seem to be compromised by the inclusion of daratumumab in the up-front setting. Patients were able to successfully collect stem cells without any compromise in the quadruplet arm.

There is a large, international, phase 3 trial called the Perseus trial [NCT03710603], which is going to further evaluate the quadruplet vs triplet combinations that were evaluated in the GRIFFIN trial. [The results of the Perseus trial] should provide further information as to whether [daratumumab plus VRd] could be a potential standard [treatment for patients with] newly diagnosed multiple myeloma.

The KRd triplet [is also] a potential backbone for daratumumab-based therapy and there have also been some exciting studies looking at that [quadruplet]. At the University of Alabama at Birmingham, the phase 2 MASTER trial [NCT03224507] was led by Luciano J. Costa, MD, and looked at daratumumab plus KRd as a potential approach in newly diagnosed multiple myeloma.

There was also a recently reported small study out of Memorial Sloan Kettering Cancer Center called the MANHATTAN trial, which looked at weekly KRd plus daratumumab. In about 41 patients enrolled in the non-randomized trial, the overall response rate was 100%, and 39 patients had a very good partial response or better. Also, these patients had an exceedingly high rate of MRD-negative disease. This is a promising potential option, but randomized data need to be evaluated [because] this was a small, non-randomized study.

Speaking to that, C. Ola Landgren, MD, of the University of Miami Miller School of Medicine, is leading a multicenter, phase 3 trial called ADVANCE [NCT04268498] thatis looking at VRd vs KRd vs daratumumab plus KRd. It is a 3-arm, randomized trial that is likely to provide further data on the feasibility and efficacy of using [these triplets and quadruplets] in the up-front setting.

[Determining] the standard of care for patients with newly diagnosed multiple myeloma has become complicated these days. There are several trials in progress that can help answer this question soon. However, currently, daratumumab is a promising potential partner for each of our backbone triplets. From my perspective, phase 3 data are required to cement a quadruplet option as a standard of care for newly diagnosed multiple myeloma.

I anticipate that these upcoming trials in progress will show us whether a quadruplet regimen is the way to go for newly diagnosed disease, but those data are not available yet.

The management of toxicities from multi-drug regimens in newly diagnosed myeloma is an important consideration. When looking at combinations that include daratumumab and immunomodulatory drugs, such as lenalidomide, then cytopenias, low blood counts, and low neutrophil counts can certainly be an issue early on in treatment. Dose modifications of lenalidomide and potential use of growth factor support can be helpful in maintaining the blood counts to a sufficient level so that we can continue to give patients the highest doses [of the medications] to achieve deep remissions.

The peripheral neuropathy [associated with] bortezomib requires careful management. Much of the published data from these duties included twice-weekly subcutaneous bortezomib on days 1, 4, 8, and 11 at 1.3 mg/m2. In the community [setting] and in our practice, we use a modified schedule of once-weekly bortezomib that has a much lower rate of peripheral neuropathy.

Much progress [is being done regarding] the treatment of patients with relapsed/refractory multiple myeloma. [We are starting to] use immunotherapeutic drugs to try to harness the power of the immune system to attack myeloma. Myeloma has been such an exciting area of practice with all these new agents that have been coming out for patients with relapsed disease.

There are several drugs in this setting that are exciting and could potentially be considered as options for the newly diagnosed patient population. However, we are still several years away from [those drugs being introduced to the armamentarium].

Data presented during the 2020 ASH Annual Meeting showed that a host of bispecific antibodies targeting BCMA [are being investigated]. BCMA is a receptor protein on the surface of myeloma cells that seems to be universally expressed on myeloma plasma cells. [BCMA] is a good target for immune-based treatments and the advantage of bispecific antibodies is that they are off-the-shelf agents. They clearly offer a way to provide rapid responses [to patients].

Looking ahead, the current challenges of cytokine release syndrome [CRS] and dealing with the toxicities of [cellular therapies] are important to figure out, especially if there is any relationship between CRS and tumor burden. Bringing these agents into the frontline setting with patients who have a much larger tumor burden is going to be something to carefully consider in future trials. Yet, these drugs are not far enough along to be considered for frontline treatment, but they [yield] potent effects and can be administered off-the-shelf to patients.

It will be fascinating to see how the bispecific antibodies are incorporated into frontline treatment, as well as the early-relapse setting, but we are several years away from that. We have yet to see among many competitors in the bispecific antibody race which one will become available first and which will be the best. Will we have multiple options in this category? All these questions have yet to be answered.

CAR T-cell therapy is another platform that has been very exciting. With the recent FDA approval of idecabtagene vicleucel [Abecma], we have another option for heavily relapsed patients. It will be interesting to see if we can use CAR T-cell therapy in earlier lines of therapy and what effects [that would yield].

CAR T-cell therapy does require a fair amount of preparation, planning, manufacturing time, and lead time, so the logistical considerations for administering CAR T-cell therapy are significant. Likely, if [this treatment] is going to be considered in the frontline setting, patients will receive other agents prior as preparation for CAR T-cell therapy.

There is a fascinating future for immunotherapy in myeloma. The years to come will show us whether [bispecific antibodies and CAR T-cell therapies] have a role for the treatment of newly diagnosed disease.

The other thing to consider is treatment with checkpoint inhibitors has had a checkered history in myeloma. PD-1 inhibitors were shown to be toxic in combination with lenalidomide or pomalidomide [Pomalyst]. The checkpoint blockade approach, which has been successful in many other tumor types, has not had a future in myeloma; however, other novel immune checkpoints are being considered in the relapsed setting with TIGIT and LAG3 [inhibitors]. These are agents are being evaluated in ongoing trials.

There are other potential approaches we can use to modulate the immune system for the treatment of patients with multiple myeloma, but it takes many years to do these studies and establish safety and [efficacy]. It will be some time before we have other immune therapies applied to the frontline setting.

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Quadruplets, Immune-Based Regimens Slated to Expand the Frontline Myeloma Paradigm - OncLive