Sickle Cell Anemia Patient Becomes First Person in the US to Have Her Genes Edited With CRISPR – Interesting Engineering

Last week, a woman namedVictoria Gray became the first person in the U.S. to have her cells edited with CRISPR. The41-year-old patient was sufferingfromsickle cell anemia.

RELATED:FIRST HUMAN TRIAL USING CRISPR GENE-EDITING IN US BEGINS

The condition, caused by a genetic mutation that messes with the shape of red blood cells, causes havoc on patients, and to make things even worse, the options for treatment are very limited and ineffective. The only current treatment for sickle cell anemia patients is a donor transplant that works for just 10% of patients, but all that is about to change.

It was clear that analternative, much more effectivesolutionwas desperately needed. After much consideration, doctors believed that editing cells extracted from a patient's own bone marrow could restore effective red blood cell creation, and this is exactly the operation they attempted on Gray.

The doctors used CRISPR to tweak Gray's bone marrow DNA to turn on a specific protein that would allow proper red blood cell generation. The operation makes Gray the first person in the U.S. to undergo a CRISPR editing procedure and the second globally.

The treatment comes from observations made back in the 1940s.In 1941 a pediatrician named Jane Watson noticed that babies with sickle cell didnt have symptoms until 6 months to 1 year of age, Vivien Sheehan, a hematologist at Baylor University told Popular Science.

The pediatrician also discovered that these infants produced fetal hemoglobin for much longer periods than healthy babies.Following Watson's observations, the research since then has indicated that increasing fetal hemoglobin could provide an effective treatment for the disease.

Now, CRISPR may just make that treatment viable. But before we get too excited, it should be noted that the strategy comes with several risks.

In order for the edited cells to be inserted back into the patients bone marrow, other stem cells need to be deactivated. Otherwise, there is the chance the unedited stem cells may continue to produce sickled red blood cells very fast, outpacing the edited cells' production of healthy cells.

Now researchers say they need to follow Gray's progress for at least 15 years to rule out any other potential dangers of the procedure. Still, for those 90% suffering with sickle cell anemia that don't respond well to current treatment, the procedure, if successful, would offer the much-needed lifeline they've been hoping for.

Visit link:
Sickle Cell Anemia Patient Becomes First Person in the US to Have Her Genes Edited With CRISPR - Interesting Engineering

Teen diagnosed with cancer again just months after being told he beat disease – Mirror Online

A teenage boy has been diagnosed with an aggressive cancer again just four months after a doctor told him he had beaten the devastating disease.

There is just a 10 per cent chance that chemotherapy will work this time for 17-year-old Jacob Fradgley, who is "wasting away", his heartbroken mum said.

The teen, from Skegby in Nottinghamshire, originally found out that he had metastatic Ewing sarcoma, a rare form of bone cancer, when he was just 15 in May 2018.

After a "whirlwind" year of treatment, including 19 rounds of chemotherapy and 56 sessions of radiation therapy, Jacob was classed as cancer free in July, his mum, Sammy Fradgley, 34, told NottinghamshireLive.

But they were given the heartbreaking news that his cancer had returned after his mum found a lump on his head just weeks before Christmas.

Jacob was diagnosed with a relapse of metatistic ewing sarcoma, which affects bones and bone tissue.

Only 30 children a year are diagnosed in the UK.

Miss Fradgley has launched a campaign to fulfil some of her son's wishes - including meeting ex-soldier Ant Middleton SAS: Who Dares Wins - after doctors told her that if there was anything he wanted to do he should "do it now".

After his mum found the lump, Jacob was taken to Queen's Medical Centre in Nottingham for a CT scan.

He underwent surgery to remove the tumour in his head a week later.

Doctors then found more tumours in his head, hip and knees.

Miss Fradgley, a senior carer, said: "It took me two weeks to accept it.

"There's not a lot out there to treat a relapse, and we're just getting further away from solutions.

"There's around a 10 per cent chance the chemotherapy will work this time.

"You never think it's going to come back but I've just got to support him now."

Jacob had his final round of radiation therapy in January and his last round of chemo on New Year's Eve.

In the following months he took medication to prevent the cancer returning.

His mum said: "He was able to go to his prom, he was going to cadets and studying Public Services at Vision West Notts College.

"But three weeks ago I was touching his hair, and felt the lump. It all just happened so quick.

"Now its more or less the same thing. It's so aggressive and is known for coming back."

She added: "It has upset him, that he has to go through it all again.

"He has to have six rounds of chemotherapy and the doctors also want to harvest stem cells. If the chemo works, he may be able to go for stem cell treatment."

Jacob's consultant has told her that if there was something he wanted to do he should "do it now".

She has launched a JustGiving campaign to raise enough money so Jacob can create memories with his six-year-old sister Leah.

It has raised more than 2,300 so far.

Miss Fradgley said: "I just don't want to say no. I want to give him everything he wants.

"It's so hard to just watch him wasting away in front of me, but I am trying to do everything I can for him.

Video Unavailable

Click to playTap to play

Play now

"He does still smile, but I am also there when he gets upset.

"His biggest aspiration is to drive a tank, meet Ant Middleton - anything to do with the army.

"His favourite colour is red so I have also started a 'Going Red for Jacob' campaign and I plan to make red bands with 'Team Fradgley' so everyone can support him and donate."

Barbera White, founder of the When You Wish Upon a Star charity, which is supporting the family, said: "I just wanted mum to know if there's anything we can do, we will try.

"I am trying to arrange a wish for him and I am currently working on something that I know he wants.

"Even after 30 years of the charity, I keep in touch with the families I meet, and we all want to help create some memories for Jacob."

Last December, the charity gave Jacob, his mum and his sister a trip to Lapland as he battled cancer.

At the time, he was looking forward to spending Christmas out of hospital and said the Lapland trip was a chance to "be together like a normal family".

See the original post:
Teen diagnosed with cancer again just months after being told he beat disease - Mirror Online

Living with cancer: ‘Exercising during my chemotherapy treatment worked wonders’ – inews

NewsReal LifeCathal Morrow, 54, talks about the positive effects running and gym sessions had on him both physically and mentally

Friday, 6th December 2019, 5:33 pm

As part of a new series calledLiving with cancer,iis sharing people's stories about how the disease affects their life and relationships, both practically and emotionally.

Cathal Morrow knew that he could have easily have slumped into a depression after hearing the dreaded C word.

When he began his three month chemotherapy treatment, it knocked him for six. "The first 10 days I lay in bed watching Netflix. I felt rotten, exhausted and sick. I felt like I'd been hit by a bus. I thought oh gosh, I've got three months of this."

Then his youngest son had what he calls a post-traumatic stress disorder (PTSD) episode, and Cathal had to physically restrain him. It made him realise he was stronger than he had realised. And so, from then, he forced himself to turn off the telly and go outside a trip to the shops or a stroll around the park.

"I was knackered afterwards, but it felt good," he said. Having quit smoking when he was diagnosed with T-Cell Lymphoma, a type of blood cancer, he decided to go to the gym. It felt good, so the next day he went for a run, and managed 6 km (3.7 miles), and the next day pounded the pavements again.

Cathal had run a marathon a decade ago, but had stopped running regularly. The 54-year-old soon built up his fitness and took on a half marathon (21 km/13.1 miles) while still having chemo.

Exercise can help general health, and there's been a recent paradigm shift with regards to the role it plays in cancer patients. Last year, leading experts on cancer called for exercise to be prescribed to all cancer patients, alongside traditional treatment, and say not to do so would be harmful.

If the effects of exercise could be encapsulated in a pill, it would be prescribed to every cancer patient worldwide and viewed as a major breakthrough in cancer treatment, Professor Prue Cormie from the Australian Catholic University wrote on The Conversation.

'It stops me from becoming a miserable sod'

Cathal began exercising five times a week, and he says it helped empower him to move out of a more passive patient role, and improved not just his well-being but his attitude, too.

"It would be easy to think, I'm sick, I'm having treatment, so I must wrap myself up in cotton wool. Doing the half-marathon, I was shouting 'Fuck cancer' as I hauled myself up a gruesome hill. It was my way of sticking my finger up to the disease. It worked wonders.

"Running helped me manage my fear of dying. There's no two ways about it, it's terrifying to be told you have cancer. I had a good cry, then I thought right, I'm beating this. Exercise was a way to channel my frustration, at times rage.

"It gave me something to look forward to almost every day, something very immediate to focus on that I could achieve. It made me feel at least partly in control of my health.

"It's also had a positive effect on my two sons. They saw I was fighting cancer, that I refuse to give in. It stops me from becoming a miserable sod, because I dont need to fake positivity."

Rest is 'counterproductive'

Macmillan Cancer Support also say that traditional advice for combating fatigue centering on energy conservation is "counterproductive", since "excessive rest worsens treatment-related loss of physical function".

'Studies have found exercise during treatment can actually change the tumor microenvironment and trigger stronger anti-tumor activity'

Mayo Clinic experts

The charity states: "This can lead to a vicious cycle of accumulating fatigue and deteriorating function. A promising body of evidence indicates that an appropriate balance of physical activity alongside rest during treatment periods helps to control fatigue and maintain physical function."

Experts at the Mayo Clinic say as well as reducing depression and anxiety, exercise can reduce pain. They point out "studies have found that exercise during treatment can actually change the tumor microenvironment and trigger stronger anti-tumor activity in your immune system". And animal studies have found that exercise can lead to tumor reduction in rodents.

Physical activity also helps you manage your weight, which is an important cancer risk factor and has been linked to higher risk of cancer recurrence.

'Balance is key'

Cathal, from Wimbledon, London, says exercise made him feel better prepared physically to be able to handle the treatment. His doctor approved of him getting active, but warned him not to overdo it.

Cathal, who works in PR and as a writer, underwent stem cell treatment last November, and has since gone into remission, although he still struggles with fatigue.

"My doctor is fully supportive of me exercising my way through chemo although she cautioned me not to 'go mental'. I probably did a bit. I just love pushing my body past what I think is possible. It's felt fantastic feeling the fittest I've been in a decade.

"But I think it's also important to listen to you body, balance is key. I aimed to exercise five times a week but if I felt rubbish I'd rest. Same goes for the emotional side. Sometimes you have to succumb to not feeling very positive. Some days I feel too crap to do anything and spend a few days in bed."

Cancer Research says how much and how intense a cancer patient should exercise will depend on the individual, and advises people check with their doctor beforehand. You can read their guidelines about exercising when you have cancer here.

Do you have a personal story? Email claudia.tanner@inews.co.uk

View original post here:
Living with cancer: 'Exercising during my chemotherapy treatment worked wonders' - inews

Takeda to Present Results from the Phase 3 TOURMALINE-AL1 Trial of NINLARO in Patients with Amyloidosis – Business Wire

CAMBRIDGE, Mass. & OSAKA, Japan--(BUSINESS WIRE)--Takeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) today announced that results of the TOURMALINE-AL1 trial will be presented during an oral session at the 61st American Society of Hematology (ASH) annual meeting on Saturday, December 7, 2019 in Orlando, Florida. TOURMALINE-AL1 is a Phase 3, randomized clinical trial evaluating the effect of NINLAROTM (ixazomib) in combination with dexamethasone in patients with relapsed or refractory systemic light-chain (AL) amyloidosis.

The TOURMALINE-AL1 trial did not meet the first of the two primary endpoints of significant improvement in overall hematologic response, as reported in June 2019. Hematologic responses were seen in 53% versus 51% of patients receiving NINLARO plus dexamethasone versus physicians choice (odds ratio 1.10 [95% CI 0.60-2.01], p=0.762) as assessed by an Adjudication Committee (AC). The second primary endpoint of two-year vital organ deterioration or death was not mature at the time of analysis. Other endpoints studied including vital organ progression free survival (PFS), hematologic PFS, time to treatment failure and time to subsequent therapy were numerically higher in the NINLARO plus dexamethasone arm compared to the physicians choice arm. Takeda is committed to making data available to researchers to continue investigation of this disease. NINLARO is not approved as a treatment for AL amyloidosis.

AL amyloidosis is a rare condition, for which prognosis and patient outcomes are poor. Current treatments are often retrofitted from therapies used for multiple myeloma, said Angela Dispenzieri, MD, Mayo Clinic, and the trials principal investigator and lead author. For a Phase 3 study that did not meet its primary endpoint, this trial provides interesting information for this community and for future studies. Ongoing research and development to investigate potential treatment options for this underserved patient population is critical.

We look forward to the opportunity to share the data from the TOURMALINE-AL1 trial, said Phil Rowlands, Head of Oncology Clinical Research and Development, Takeda. We are confident that sharing our findings with the community will help encourage conversations around the need for continued research to address the needs that remain in this patient population.

There are serious unmet needs for people living with amyloidosis. AL amyloidosis is a progressive and fatal disease; many patients are diagnosed late, significantly impacting life expectancy. The challenges associated with developing drugs for this disease make continued research and development for treatment critical, said Isabelle Lousada, Founder and CEO of the Amyloidosis Research Consortium. The data from TOURMALINE-AL1 provide valuable insights to researchers as they select endpoints for future amyloidosis studies, and knowledge that will provide context in future drug reviews and approvals, ultimately aiding in providing treatment options for patients.

Primary Results from the Phase 3 TOURMALINE-AL1 Trial of Ixazomib-Dexamethasone Versus Physicians Choice of Therapy in Patients (Pts) with Relapsed/Refractory Primary Systemic AL Amyloidosis (RRAL). Saturday, December 7, 9:30 a.m., Orange County Convention Center, Hall E1.

Key findings, to be presented by Dr. Angela Dispenzieri, include:

About the TOURMALINE-AL1 Trial

TOURMALINE-AL1 (NCT01659658) is an international, randomized, controlled, open-label, multicenter, Phase 3 study, designed to determine whether NINLAROTM (ixazomib) in combination with dexamethasone improves hematologic response, two-year vital organ (heart or kidney) deterioration and mortality rate versus a physician's choice of a chemotherapy regimen in participants diagnosed with relapsed or refractory systemic light chain (AL) amyloidosis. 168 patients were enrolled and randomly selected to receive either NINLARO plus dexamethasone, or physicians choice of the following: dexamethasone plus melphalan; dexamethasone plus cyclophosphamide; dexamethasone plus thalidomide; dexamethasone plus lenalidomide; or dexamethasone alone. The discontinuation of the TOURMALINE-AL1 trial was announced in June 2019. For more information, please visit https://clinicaltrials.gov/ct2/show/NCT01659658.

About AL Amyloidosis

Primary AL amyloidosis is a condition that falls under the umbrella of plasma cell dyscrasias. AL amyloidosis arises from a clonal plasma cell that produces abnormal immunoglobulin light-chain fragments. These misfolded light-chains form insoluble fibrils that aggregate as amyloid deposits in organs and tissues throughout the body, ultimately leading to organ dysfunction and death. The most common organs affected are the kidneys, heart, liver, and autonomic or peripheral nerves.

There are currently no treatments approved for the treatment of AL amyloidosis.

About NINLAROTM (ixazomib) capsules

NINLARO (ixazomib) is an oral proteasome inhibitor which is being studied across the continuum of multiple myeloma treatment settings. NINLARO was first approved by the U.S. Food and Drug Administration (FDA) in November 2015 and is indicated in combination with lenalidomide and dexamethasone for the treatment of patients with multiple myeloma who have received at least one prior therapy. NINLARO is currently approved in more than 60 countries, including the United States, Japan and in the European Union, with more than 10 regulatory filings currently under review. It was the first oral proteasome inhibitor to enter Phase 3 clinical trials and to receive approval.

The comprehensive ixazomib clinical development program, TOURMALINE, includes several ongoing pivotal trials, which together are investigating major multiple myeloma patient populations:

In addition to the TOURMALINE program, ixazomib is being evaluated in multiple therapeutic combinations for various patient populations in investigator initiated studies globally.

NINLARO (ixazomib) capsules: Global Important Safety Information

SPECIAL WARNINGS AND PRECAUTIONSThrombocytopenia has been reported with NINLARO (28 percent vs. 14 percent in the NINLARO and placebo regimens, respectively) with platelet nadirs typically occurring between Days 14-21 of each 28-day cycle and recovery to baseline by the start of the next cycle. It did not result in an increase in hemorrhagic events or platelet transfusions. Monitor platelet counts at least monthly during treatment with NINLARO and consider more frequent monitoring during the first three cycles. Manage with dose modifications and platelet transfusions as per standard medical guidelines.

Gastrointestinal toxicities have been reported in the NINLARO and placebo regimens respectively, such as diarrhea (42 percent vs. 36 percent), constipation (34 percent vs. 25 percent), nausea (26 percent vs. 21 percent), and vomiting (22 percent vs. 11 percent), occasionally requiring use of antiemetic and anti-diarrheal medications, and supportive care.

Peripheral neuropathy was reported with NINLARO (28 percent vs. 21 percent in the NINLARO and placebo regimens, respectively). The most commonly reported reaction was peripheral sensory neuropathy (19 percent and 14 percent in the NINLARO and placebo regimens, respectively). Peripheral motor neuropathy was not commonly reported in either regimen (< 1 percent). Monitor patients for symptoms of peripheral neuropathy and adjust dosing as needed.

Peripheral edema was reported with NINLARO (25 percent vs. 18 percent in the NINLARO and placebo regimens, respectively). Evaluate patients for underlying causes and provide supportive care, as necessary. Adjust the dose of dexamethasone per its prescribing information or the dose of NINLARO for severe symptoms.

Cutaneous reactions occurred in 19 percent of patients in the NINLARO regimen compared to 11 percent of patients in the placebo regimen. The most common type of rash reported in both regimens was maculo-papular and macular rash. Manage rash with supportive care, dose modification or discontinuation.

Hepatotoxicity, drug-induced liver injury, hepatocellular injury, hepatic steatosis, and hepatitis cholestatic have been uncommonly reported with NINLARO. Monitor hepatic enzymes regularly and adjust dose for Grade 3 or 4 symptoms.

Pregnancy- NINLARO can cause fetal harm. Advise male and female patients of reproductive potential to use contraceptive measures during treatment and for an additional 90 days after the final dose of NINLARO. Women of childbearing potential should avoid becoming pregnant while taking NINLARO due to potential hazard to the fetus. Women using hormonal contraceptives should use an additional barrier method of contraception.

Lactation- It is not known whether NINLARO or its metabolites are excreted in human milk. There could be potential adverse events in nursing infants and therefore breastfeeding should be discontinued.

SPECIAL PATIENT POPULATIONSHepatic Impairment: Reduce the NINLARO starting dose to 3 mg in patients with moderate or severe hepatic impairment.

Renal Impairment: Reduce the NINLARO starting dose to 3 mg in patients with severe renal impairment or end-stage renal disease (ESRD) requiring dialysis. NINLARO is not dialyzable and, therefore, can be administered without regard to the timing of dialysis.

DRUG INTERACTIONSCo-administration of strong CYP3A inducers with NINLARO is not recommended.

ADVERSE REACTIONSThe most frequently reported adverse reactions ( 20 percent) in the NINLARO regimen, and greater than in the placebo regimen, were diarrhea (42 percent vs. 36 percent), constipation (34 percent vs. 25 percent), thrombocytopenia (28 percent vs. 14 percent), peripheral neuropathy (28 percent vs. 21 percent), nausea (26 percent vs. 21 percent), peripheral edema (25 percent vs. 18 percent), vomiting (22 percent vs. 11 percent), and back pain (21 percent vs. 16 percent). Serious adverse reactions reported in 2 percent of patients included thrombocytopenia (2 percent) and diarrhea (2 percent). For each adverse reaction, one or more of the three drugs was discontinued in 1percent of patients in the NINLARO regimen.

For European Union Summary of Product Characteristics: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_-Product_Information/human/003844/WC500217620.pdf For US Prescribing Information: https://www.ninlarohcp.com/pdf/prescribing-information.pdf For Canada Product Monograph: http://www.takedacanada.com/ninlaropm

About Takeda Pharmaceutical CompanyTakeda Pharmaceutical Company Limited (TSE:4502/NYSE:TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to bringing Better Health and a Brighter Future to patients by translating science into highly-innovative medicines. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Diseases, Neuroscience and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people's lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries.

For more information, visit https://www.takeda.com

Important Notice

For the purposes of this notice, press release means this document, any oral presentation, any question and answer session and any written or oral material discussed or distributed by Takeda Pharmaceutical Company Limited (Takeda) regarding this release. This press release (including any oral briefing and any question-and-answer in connection with it) is not intended to, and does not constitute, represent or form part of any offer, invitation or solicitation of any offer to purchase, otherwise acquire, subscribe for, exchange, sell or otherwise dispose of, any securities or the solicitation of any vote or approval in any jurisdiction. No shares or other securities are being offered to the public by means of this press release. No offering of securities shall be made in the United States except pursuant to registration under the U.S. Securities Act of 1933, as amended, or an exemption therefrom. This press release is being given (together with any further information which may be provided to the recipient) on the condition that it is for use by the recipient for information purposes only (and not for the evaluation of any investment, acquisition, disposal or any other transaction). Any failure to comply with these restrictions may constitute a violation of applicable securities laws.

The companies in which Takeda directly and indirectly owns investments are separate entities. In this press release, Takeda is sometimes used for convenience where references are made to Takeda and its subsidiaries in general. Likewise, the words we, us and our are also used to refer to subsidiaries in general or to those who work for them. These expressions are also used where no useful purpose is served by identifying the particular company or companies.

Forward-Looking Statements

This press release and any materials distributed in connection with this press release may contain forward-looking statements, beliefs or opinions regarding Takedas future business, future position and results of operations, including estimates, forecasts, targets and plans for Takeda. Without limitation, forward-looking statements often include words such as targets, plans, believes, hopes, continues, expects, aims, intends, ensures, will, may, should, would, could anticipates, estimates, projects or similar expressions or the negative thereof. Forward-looking statements in this document are based on Takedas estimates and assumptions only as of the date hereof. Such forward-looking statements do not represent any guarantee by Takeda or its management of future performance and involve known and unknown risks, uncertainties and other factors, including but not limited to: the economic circumstances surrounding Takedas global business, including general economic conditions in Japan and the United States; competitive pressures and developments; changes to applicable laws and regulations; the success of or failure of product development programs; decisions of regulatory authorities and the timing thereof; fluctuations in interest and currency exchange rates; claims or concerns regarding the safety or efficacy of marketed products or product candidates; the timing and impact of post-merger integration efforts with acquired companies; and the ability to divest assets that are not core to Takedas operations and the timing of any such divestment(s), any of which may cause Takedas actual results, performance, achievements or financial position to be materially different from any future results, performance, achievements or financial position expressed or implied by such forward-looking statements. For more information on these and other factors which may affect Takedas results, performance, achievements, or financial position, see Item 3. Key InformationD. Risk Factors in Takedas most recent Annual Report on Form 20-F and Takedas other reports filed with the U.S. Securities and Exchange Commission, available on Takedas website at: https://www.takeda.com/investors/reports/sec-filings/ or at http://www.sec.gov. Future results, performance, achievements or financial position of Takeda could differ materially from those expressed in or implied by the forward-looking statements. Persons receiving this press release should not rely unduly on any forward-looking statements. Takeda undertakes no obligation to update any of the forward-looking statements contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. Past performance is not an indicator of future results and the results of Takeda in this press release may not be indicative of, and are not an estimate, forecast or projection of Takedas future results.

Link:
Takeda to Present Results from the Phase 3 TOURMALINE-AL1 Trial of NINLARO in Patients with Amyloidosis - Business Wire

Stem cells may trigger immune repair to mend hearts – BioNews

2 December 2019

Stem cell therapies may become redundant in repairing cardiac function after a heart attack, suggests a new study in mice.

It showed how stem cell treatments can heal hearts by triggering an immune response which can be achieved by using a chemical instead.

'This work is paradigm-shifting because it demonstrates a mechanism to explain a perplexing phenomenon that has intrigued cardiologists as a result of decades of cardiac stem cell trials,' Dr Jonathan Epstein at the University of Pennsylvania's Perelman School of Medicine in Philadelphia told The Scientist.

Stem cell therapies to repair damaged heart tissue are currently being tested in human clinical trials. In these treatments, human stem cells are injected into the heart and this leads to an improvement in heart function. However, how this works is not fully understood.

One possibility is that the injected stem cells are incorporated into the heart tissue and repair the damage. However, the latest study, published in the journal Nature, suggests that this may not be the case. Instead, the study indicated that the repair is actually a result of triggering the innate immune response.

Researchers injected different types of stem cell or a chemical inducer (zymosan) of the innate immune response into an experimental mouse model of heart disease. They saw improvement in heart function that was similar in all cases, and showed that this repair occurs via activation of macrophage cells of the innate immune system.

'The innate immune response acutely altered cellular activity around the injured area of the heart so that it healed with a more optimised scar and improved contractile properties,' said Dr Jeffery Molkentin at the University of Cincinnati and Cincinnati Children's Hospital Medical Centre, Ohio, who led the study. 'The implications of our study are very straightforward and present important new evidence about an unsettled debate in the field of cardiovascular medicine.'

The work could open up new possibilities for optimising the treatments currently in development, as well as alternative new therapies.

'If there is a chemical off-the-shelf, it would be a much more feasible therapy [than stem cell transplants],'Dr Kory Lavine at Washington University in St Louis, Missouri, told Nature News.

Read more here:
Stem cells may trigger immune repair to mend hearts - BioNews

Omeros Reports Positive Data Across Primary and Secondary Endpoints in Pivotal Trial of Hematopoietic Stem Cell Transplant-Associated Thrombotic…

SEATTLE--(BUSINESS WIRE)--Omeros Corporation (Nasdaq: OMER) today announced positive data from its pivotal clinical trial of the companys novel investigational complement inhibitor narsoplimab in the treatment of hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), a frequently lethal complication of HSCT. These preliminary data were recently provided to FDA as part of the companys ongoing interactions with the Agency on the narsoplimab Biologics License Application (BLA). All safety and efficacy endpoints including the composite primary endpoint and the secondary endpoints are agreed with FDA. The reported data support a strongly positive benefit-risk balance.

The primary efficacy endpoint in this single-arm open-label trial of HSCT-TMA patients is the proportion of patients who achieve a highly rigorous set of response criteria that requires both improvement in HSCT-TMA laboratory markers and improvement in clinical status (organ function and transfusions). Patients who did not fully meet these criteria were considered non-responders. The secondary endpoints include survival rates and change from baseline in HSCT-TMA laboratory markers. Consistent with the pre-specified statistical analysis plan for the trial, the primary and secondary endpoints are assessed for (1) all patients who received at least one dose of narsoplimab and (2) patients who received at least 4 weeks of narsoplimab dosing. Patients enrolled in this trial had a high expected mortality rate. In severe cases of HSCT-TMA, mortality can exceed 90 percent.

Primary Efficacy Endpoint:

Secondary Endpoints:

Safety:

The HSCT-TMA patient population enrolled in this trial had multiple high-risk features that portend a poor outcome. These include persistence of HSCT-TMA despite modification of immunosuppression (which was a criterion for entry into the trial), graft-versus-host disease, significant infections, non-infectious pulmonary complications and neurological findings. Patients in the trial had a high expected death rate, with 93 percent of them having multiple risk factors.

Patient enrollment in the pivotal trial has been completed. The details of the endpoints, including the response criteria agreed with FDA, and the number of patients in the trial remain confidential for competitive business reasons.

Last year the company reported data on 19 HSCT-TMA patients treated with narsoplimab on which FDA granted breakthrough therapy designation. The results reported today are even stronger. The response rate remains equally high at 56 percent, while the 100-day survival has improved from 53 percent to 65 percent.

The response rate in this high-risk population would be expected to be 10 to 15 percent with a 100-day survival rate of less than 20 percent. The response rate and 100-day survival achieved with narsoplimab in this trial demonstrate an unprecedented effect in this condition, said Rafael Duarte M.D., Ph.D., F.R.C.P., Associate Professor, Head of Hematology Department and Hematopoietic Transplantation Program, University Hospital Puerta de Hierro Majadahonda, Madrid, Spain, and Secretary of the European Society for Blood and Marrow Transplantation. The other secondary endpoints are equally impressive. The data are consistent with my personal experience with narsoplimab. Patients with severe forms of HSCT-TMA have a dismal prognosis with no treatment currently available. I expect a treatment with this profile would be widely adopted for use in these patients and even lead to increased physician recognition of the disorder.

Omeros reported the initiation of its rolling BLA in October. Narsoplimab, also referred to as OMS721, is Omeros lead human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2) and has breakthrough therapy designation from FDA for this indication.

The striking results seen in our pivotal trial are tremendously gratifying, said Gregory A. Demopulos, M.D., chairman and chief executive officer of Omeros. Our rolling BLA is underway the nonclinical sections have been submitted and the data from this trial form the efficacy basis of the application. We continue to compile the remaining sections of the BLA and look forward to continued partnership with regulators to make narsoplimab widely available for the treatment of this devastating condition.

Data from this pivotal trial will also support the narsoplimab marketing authorization application for HSCT-TMA in Europe. The data are planned for publication and for presentation at international congresses in the first part of 2020.

In addition to breakthrough therapy designation from FDA, narsoplimab has orphan drug designation in both the U.S. and Europe for HSCT-TMA. Narsoplimab also has been awarded breakthrough therapy designation for immunoglobulin A nephropathy (IgAN), and Omeros has Phase 3 programs for narsoplimab ongoing in IgAN and in atypical hemolytic uremic syndrome (aHUS).

Conference Call and Webcast Details

Omeros management will host a webcast and conference call to present data from its pivotal trial of narsoplimab in HSCT-TMA. The call will be held today at 8:30 a.m. Eastern Time; 5:30 a.m. Pacific Time. To access the live conference call via phone, please dial (844) 831-4029 from the United States and Canada or (920) 663-6278 internationally. The participant passcode is 3774209. Please dial in approximately 10 minutes prior to the start of the call. A telephone replay will be available for one week following the call and may be accessed by dialing (855) 859-2056 from the United States and Canada or (404) 537-3406 internationally. The replay passcode is 3774209.

To access the live or subsequently archived webcast and presentation materials on the internet, go to the companys website at http://www.omeros.com and select Events under the Investors section of the website. To access the live webcast, please connect to the website at least 15 minutes prior to the call to allow for any software download that may be necessary.

About Omeros Corporation

Omeros is an innovative biopharmaceutical company committed to discovering, developing and commercializing small-molecule and protein therapeutics for large-market as well as orphan indications targeting complement-mediated diseases, disorders of the central nervous system and immune-related diseases, including cancers. In addition to its commercial product OMIDRIA (phenylephrine and ketorolac intraocular solution) 1%/0.3%, Omeros has multiple Phase 3 and Phase 2 clinical-stage development programs focused on complement-mediated disorders and substance abuse. In addition, the company has a diverse group of preclinical programs including GPR174, a novel target in immuno-oncology that modulates a new cancer immunity axis recently discovered by Omeros. Small-molecule inhibitors of GPR174 are part of Omeros proprietary G protein-coupled receptor (GPCR) platform through which it controls 54 new GPCR drug targets and their corresponding compounds. The company also exclusively possesses a novel antibody-generating platform.

About HSCT-TMA

Hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA) is a significant and often lethal complication of stem cell transplants. This condition is a systemic, multifactorial disorder caused by endothelial cell damage induced by conditioning regimens, immunosuppressant therapies, infection, GvHD, and other factors associated with stem cell transplantation. Endothelial damage, which activates the lectin pathway of complement, plays a central role in the development of HSCT-TMA. The condition occurs in both autologous and allogeneic transplants but is more common in the allogeneic population. In the United States and Europe, approximately 25,000 to 30,000 allogeneic transplants are performed annually. Recent reports in both adult and pediatric allogeneic stem cell transplant populations have found an HSCT-TMA incidence of approximately 40 percent, and high-risk features may be present in up to 80 percent of these patients. In severe cases of HSCT-TMA, mortality can exceed 90 percent and, even in those who survive, long-term renal sequalae are common. There is no approved therapy or standard of care for HSCT-TMA.

About Narsoplimab

Narsoplimab, also known as OMS721, is an investigational human monoclonal antibody targeting mannan-binding lectin-associated serine protease-2 (MASP-2), a novel pro-inflammatory protein target and the effector enzyme of the lectin pathway of complement. Importantly, inhibition of MASP-2 does not appear to interfere with the antibody-dependent classical complement activation pathway, which is a critical component of the acquired immune response to infection. Omeros controls the worldwide rights to MASP-2 and all therapeutics targeting MASP-2.

Phase 3 clinical programs are in progress for narsoplimab in hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA), in immunoglobulin A (IgA) nephropathy, and in atypical hemolytic uremic syndrome (aHUS). The FDA has granted narsoplimab breakthrough therapy designations for HSCT-TMA and for IgA nephropathy; orphan drug status for the prevention (inhibition) of complement-mediated thrombotic microangiopathies, for the treatment of HSCT-TMA and for the treatment of IgA nephropathy; and fast track designation for the treatment of patients with aHUS. The European Medicines Agency has granted orphan drug designation to narsoplimab for treatment in HSCT and for treatment of primary IgA nephropathy.

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934, which are subject to the safe harbor created by those sections for such statements. All statements other than statements of historical fact are forward-looking statements, which are often indicated by terms such as anticipate, believe, could, estimate, expect, goal, intend, likely, look forward to, may, on track, plan, potential, predict, project, prospects, scheduled, should, slated, targeting, will, would and similar expressions and variations thereof. Forward-looking statements, including statements regarding anticipated regulatory submissions, expectations regarding regulatory exclusivities, the timing and results of ongoing or anticipated clinical trials, and the therapeutic application of Omeros investigational product, are based on managements beliefs and assumptions and on information available to management only as of the date of this press release. Omeros actual results could differ materially from those anticipated in these forward-looking statements for many reasons, including, without limitation, availability and timing of data from clinical trials and the results of such trials, unproven preclinical and clinical development activities, regulatory oversight, intellectual property claims, competitive developments, litigation, and the risks, uncertainties and other factors described under the heading Risk Factors in the companys Annual Report on Form 10-K filed with the Securities and Exchange Commission on March 1, 2019. Given these risks, uncertainties and other factors, you should not place undue reliance on these forward-looking statements, and the company assumes no obligation to update these forward-looking statements, whether as a result of any new information, future events or otherwise, except as required by applicable law.

Source: Omeros Corporation

Originally posted here:
Omeros Reports Positive Data Across Primary and Secondary Endpoints in Pivotal Trial of Hematopoietic Stem Cell Transplant-Associated Thrombotic...

Hunter Syndrome Treatment Market Size, Share & Trend Analysis By Treatment, By Region And Segment Forecasts, 2019 – 2026 – P&T Community

NEW YORK, Dec. 4, 2019 /PRNewswire/ --

Hunter Syndrome Treatment Market Size, Share & Trend Analysis By Treatment (Enzyme Replacement Therapy, Hematopoietic Stem Cell Transplant), By Region, And Segment Forecasts, 2019 - 2026

Read the full report: https://www.reportlinker.com/p05830601/?utm_source=PRN

The global hunter syndrome treatment market size is expected to reach a value of USD 1.52 billion by 2026, expanding at a CAGR of 7.1%. High unmet needs, robust pipeline, increasing awareness about this rare disease and growing R&D activities for the development of novel therapies are expected to drive market growth over the forecast period.

Hunter syndrome, also referred as mucopolysaccharidosis type II (MPS II), is a rare genetic disorder caused by the missing or malfunctioning iduronate-2-sulfatase enzyme. According to the data published by the National Institute of Neurological Disorders and Stroke, MPS II syndrome occurs in around 1 in every 100,000 to 150,000 male births.

Presently, there are no approved curative therapies for the treatment of Hunter syndrome.The available treatment options such as enzyme replacement therapy (ERT) and hematopoietic stem cell transplant (HSCT) are focused on providing symptomatic relief and management of complications associated with disease progression.

Shire plc's Elaprase (idursulfase) is the only key drug available for the treatment of Hunter syndrome worldwide, with GC Pharma's Hunterase (idursulfase beta) being approved only in South Korea.

Key players are focused on extensive R&D activities for product development and gaining approval as novel therapies.Launch of such novel therapies in the near future is expected to significantly fuel the Hunter syndrome treatment market growth.

For instance, in May 2018, REGENXBIO Inc. received the U.S. FDA's Fast Track designation for its novel drug candidate RGX-121, indicated for the disease treatment.

Further Key Findings from the Study Suggest: The enzyme replacement therapy segment has acquired the largest share in 2018, owing to the increased adoption of Elaprase and the potential approval of Hunterase worldwide The absence of curative therapies for MPS II creates lucrative opportunities for the key players in the market for developing new therapies The launch of late-stage pipeline therapies is expected to drive the market growth during the forecast period Currently, Shire Plc. is one of the leading players, supported by strong sales of their marketed drug ELAPRASE for the disease treatment Major players in the market are adopting inorganic growth strategies such as partnerships and collaborations for the development and commercialization of novel therapies In 2018, North America held a dominant position in the global market, owing to favorable reimbursement scenario, high awareness regarding rare disorders, and presence of major players Some of the key companies in hunter syndrome treatment market include GC Pharma, Sangamo Therapeutics, Inc.; JCR Pharmaceuticals Co Ltd.; RegenxBio Inc.; and Shire Plc. (Takeda Pharmaceutical Company)

Read the full report: https://www.reportlinker.com/p05830601/?utm_source=PRN

About Reportlinker ReportLinker is an award-winning market research solution. Reportlinker finds and organizes the latest industry data so you get all the market research you need - instantly, in one place.

__________________________ Contact Clare: clare@reportlinker.com US: (339)-368-6001 Intl: +1 339-368-6001

View original content:http://www.prnewswire.com/news-releases/hunter-syndrome-treatment-market-size-share--trend-analysis-by-treatment-by-region-and-segment-forecasts-2019---2026-300968973.html

SOURCE Reportlinker

Here is the original post:
Hunter Syndrome Treatment Market Size, Share & Trend Analysis By Treatment, By Region And Segment Forecasts, 2019 - 2026 - P&T Community

Platelet Rich Plasma and Stem Cell Alopecia Treatment Market is on Course to Expand at a CAGR of XX% Over the Forecast Period 2018 – 2026 – Weekly Spy

Latest Study on the Global Platelet Rich Plasma and Stem Cell Alopecia Treatment Market

The recently published report by Transparency Market Research on the global Platelet Rich Plasma and Stem Cell Alopecia Treatment market offers resourceful insights pertaining to the future prospects of the Platelet Rich Plasma and Stem Cell Alopecia Treatment market. The underlying trends, growth opportunities, impeding factors, and glaring market drivers are thoroughly studied in the presented report.

As per the report, the global Platelet Rich Plasma and Stem Cell Alopecia Treatment market is projected to grow at a CAGR of ~XX% and exceed the value of ~US$ towards the end of 2029. Moreover, an in-depth analysis of the micro and macro-economic factors that are anticipated to influence the trajectory of the Platelet Rich Plasma and Stem Cell Alopecia Treatment market during the forecast period (2019-2029) is included in the report.

Reports are available at discounted rates! Offer expires soon!

Request For Discount On This Report @ https://www.transparencymarketresearch.co/sample/sample.php?flag=D&rep_id=55800

Critical Insights Related to the Platelet Rich Plasma and Stem Cell Alopecia Treatment Market in the Report:

Platelet Rich Plasma and Stem Cell Alopecia Treatment Market Segments

A thorough evaluation of the future prospects of the Platelet Rich Plasma and Stem Cell Alopecia Treatment market across various regions is tracked in the report.

competitive landscape of the industrial workwear market, thereby positioning all the major players according to their geographic presence and recent key developments. The comprehensive industrial workwear market estimates are the result of our in-depth secondary research, primary interviews, and in-house expert panel reviews. These market estimates have been analyzed by taking into account the impact of different political, social, economic, technological, and legal factors along with the current market dynamics affecting the growth of the industrial workwear market. Market share analysis has been included under the competitive landscape to understand the top players contribution to the market. Each company is studied on the basis of basic information, financial highlights, revenue highlights of regional contribution and segment contribution, and product portfolio. Additionally, the company strategy and recent developments if any are also incorporated under each company profile section.

Some of the key players in the industrial workwear market include 3M, VF Corporation, Ansell Ltd., and Honeywell International Ltd., E. I. du Pont de Nemours and Company, Williamson-Dickie Manufacturing Co., Hultafors Group, Lakeland Inc., Aramark, and Fristads Kansas Group. The key players operating in the industrial workwear market adopt numerous strategies to sustain in the market. Product development and acquisition and partnership are some of the main strategies adopted by key players to gain a competitive edge.

The industrial workwear market is segmented as follows:

Industrial Workwear Market

By Product Type

By End-use Industry

By Fit Type

By Distribution Channel

By Geography

Request Sample Report @ https://www.transparencymarketresearch.co/sample/sample.php?flag=B&rep_id=55800

Important queries related to the Platelet Rich Plasma and Stem Cell Alopecia Treatment market addressed in the report:

Why Choose TMR?

RequestTOC For ThisReport @ https://www.transparencymarketresearch.co/sample/sample.php?flag=T&rep_id=55800

Original post:
Platelet Rich Plasma and Stem Cell Alopecia Treatment Market is on Course to Expand at a CAGR of XX% Over the Forecast Period 2018 - 2026 - Weekly Spy

miR-194 Inhibits the Proliferation of SW620 Colon Cancer Stem Cells Th | CMAR – Dove Medical Press

Bo Sun,1,* Yan-Tian Fang,1,* Dan-Juan Jin,2 Zong-You Chen,3 Zhen-Yang Li,3 Xiao-Dong Gu,3 Jian-Bin Xiang3

1Department of Gastric Cancer, Fudan University Shanghai Cancer Center, Shanghai, Peoples Republic of China; 2Department of General Surgery, Songjiang District Central Hospital, Shanghai, Peoples Republic of China; 3Department of General Surgery, Huashan Hospital, Fudan University, Shanghai, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Xiao-Dong Gu; Jian-Bin XiangDepartment of General Surgery, Huashan Hospital, Fudan University, 12 Wulumuqi Middle Road, Shanghai 200040, Peoples Republic of ChinaTel +86-21-5288 7044; +86-21-5288 7040Fax +86-21-62495490Email gxdgxd737@163.com; xjbzhw131@sohu.com

Purpose: Colorectal cancer (CRC) stem cells are tumorigenic, capable of self-renewal, and resistant to therapy. Although the expression pattern and functions of micro RNA (miR)-194 in CRC cells have been widely investigated, little is known about its role in CRC stem cells. Therefore, the aim of this study was to investigate the potential role of miR-194 in CRC stem cells.Materials and methods: CRC stem cells were isolated from the SW620 colon cancer cell line using microbeads. The expression levels of miR-194 and slingshot 2 (SSH2) in CRC stem cells were detected by RT-PCR and Western blot. A luciferase reporter assay was performed to confirm that miR-194 directly targets SSH2. Proliferation of CRC stem cells was examined by colony formation and MTT assays. Apoptosis in CRC stem cells was detected by cell cycle and apoptosis assays. The role of miR-194 in tumor growth was determined in vivo.Results: Cells positive for CD44 and CD133 accounted for approximately 88.7% of the isolated population after microbead isolation. We reveal for the first time that miR-194 expression is decreased in CRC stem cells. Specifically, miR-194 is involved in inhibiting the proliferation of CRC stem cells and promoting CRC stem cell apoptosis by directly targeting SSH2. Furthermore, overexpression of miR-194 resulted in blocking the G1/S transition, the induction of cellular apoptotic process, thereby suppressing the malignant behaviors of CRC stem cells.Conclusion: This study represents a novel characterization of miR-194 function in CRC stem cells, which may aid in the development of promising therapeutic strategies targeting CRC.

Keywords: colorectal cancer, miR-194, SSH2, proliferation, stem cell

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

Visit link:
miR-194 Inhibits the Proliferation of SW620 Colon Cancer Stem Cells Th | CMAR - Dove Medical Press

Stem Cell Therapy Market Growth opportunities, Trends, Industry Analysis, and Forecast to 2020 – Kentucky Reports

Persistence Market Research(PMR), in its recent market report, suggests that the Stem Cell Therapy Market report is set to exceed US$ xx Mn/Bn. The report finds that the Stem Cell Therapy Market registered ~US$ xx Mn/Bn in 2018 and is spectated to grow at a healthy CAGR over the foreseeable period 2020.

The Stem Cell Therapy Market research focuses on the market structure and various factors (positive and negative) affecting the growth of the market. The study encloses a precise evaluation of the Stem Cell Therapy Market, including growth rate, current scenario, and volume inflation prospects, on the basis of DROT and Porters Five Forces analyses. In addition, the Stem Cell Therapy Market study provides reliable and authentic projections regarding the technical jargon.

ThisPress Release will help you to understand the Volume, growth with Impacting Trends. Click To get SAMPLE PDF (Including Full TOC, Table & Figures) athttps://www.persistencemarketresearch.co/samples/3253

The Stem Cell Therapy Market study answers critical questions including:

The content of the Stem Cell Therapy Market report includes the following insights:

Get Access To TOC Covering 200+ Topics athttps://www.persistencemarketresearch.co/toc/3253

All the players running in the global Stem Cell Therapy Market are elaborated thoroughly in the Stem Cell Therapy Market report on the basis of R&D developments, distribution channels, industrial penetration, manufacturing processes, and revenue. In addition, the report examines, legal policies, and comparative analysis between the leading and emerging Stem Cell Therapy Market players.

Some of the major companies operating in the global stem cell therapy market are Mesoblast Ltd., Celgene Corporation, Aastrom Biosciences, Inc. and StemCells, Inc.

Key points covered in the report

Access Research Methodology Prepared By Experts athttps://www.persistencemarketresearch.co/methodology/3253

Why choose PMR?

About us:

Persistence Market Research (PMR) is a third-platform research firm. Our research model is a unique collaboration of data analytics and market research methodology to help businesses achieve optimal performance.

To support companies in overcoming complex business challenges, we follow a multi-disciplinary approach. At PMR, we unite various data streams from multi-dimensional sources. By deploying real-time data collection, big data, and customer experience analytics, we deliver business intelligence for organizations of all sizes.

Contact us:Persistence Market Research305 Broadway, 7th FloorNew York City, NY 10007United StatesPh.no. +1-646-568-7751

Continued here:
Stem Cell Therapy Market Growth opportunities, Trends, Industry Analysis, and Forecast to 2020 - Kentucky Reports