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What is New in Cardiology? A Review of All Major Emerging Technologies for Heart Diseases – Diagnostic and Interventional Cardiology

By daniellenierenberg

This is an overview of some of the biggest cardiology technology advances. These innovations are covered in more detail in the two-volume set titled "Emerging Technologies in Heart Diseases." These innovative technologies mark the midway of a technological revolution in patient care. Here are a list of 10 noteworthy new cardiac technologies:

The emergence of a ventricular assist device (VAD) has revolutionized the care of patients with advanced heart failure. Primarily developed as a bridge to transplantation, the VAD has been shown to prolong life and to improve the quality of life when a donor heart is not found. Older versions required the implantation of a bulky pump and required patients to ambulate with heavy, large external batteries and control units. Yet, several revolutionary improvements in device size, battery reliability, and even wireless charging technologies might make these devices physically unnoticeable in the coming years, and possibly decrease patient susceptibility to infections. In addition, various mechanical modifications and newer modes of operation have limited the rates of hemolysis, thrombosis, and secondary aortic valve insufficiency.

Miniature VAD. Source: Watt et al. Artificial Mechanical Hearts and Ventricular Assist Devices. In: Emerging Technologies for Heart Diseases, Vol. 1 - Treatments for Heart Failure and Valvular Disorders. 2020; Elsevier, Academic Press (AP). Pages 25-40.

Atrial fibrillation (AF or AFib) remains a leading cause of stroke, which in turn may be associated with devastating health consequences and mortality. Yet, oral anticoagulants and left atrial appendage (LAA) occlusion devices may not be appropriate for all patients or may be associated with life-threatening complications. In recent years, novel, device-based technologies for stoke prevention have evolved. Some focused on carotid implants, while newer devices have been designed for continuous embolic filtration at the level of the common aortic pathway. These approaches, which are currently being tested in preclinical studies, might be translated in the near future to treatments available for patients with increased bleeding risks.

Lariat LAA closure device device (SentreHeart Inc, Redwood, Calif.). Source: Goel et al. Percutaneous closure of the left atrial appendage for stroke prevention. In: Emerging Technologies for Heart Diseases, Vol. 2 - Treatments for Myocardial Ischemia and Arrhythmias. 2020; Elsevier, Academic Press (AP). Pages 961-977.

Treatment Options Expand Beyond JAK Inhibition for Patients With Myelofibrosis – Targeted Oncology

By daniellenierenberg

The only curative approach to treating myelofibrosis (MF) at this time is hematopoietic stem cell transplant, but the treatment landscape for this patient population continues to expand, particularly since the identification of the JAK-STAT pathway as a potential target in 2005. Although the introduction of novel agents like JAK inhibitors have been promising for the treatment of patients with MF, there are more agents coming down the pipeline as well that will impact the way physicians treat this population.1

During the National Comprehensive Cancer Network (NCCN) 2020 Virtual Congress: Hematologic Malignancies, Aaron Gerds, MD, MS, assistant professor of medicine (hematology and medical oncology), Cleveland Clinic Taussig Cancer Institute, reviewed the current treatment landscape for patients with MF and whats to come for the treatment of this patient population as clinical trials continue to advance the field.

Treatments of MF focus on the symptoms of the disease. This is a very symptom-forward disease, Gerds explained during his presentation, pointing toward the 4 biggest challenges in treating MF.

Overall, 80% of patients have splenomegaly, 70% experience MF-associated symptoms, 60% to 85% have anemia or cytopenia, and the life expectancy is shortened, with the average time from diagnosis to death being 5 to 6 years in all comers.

Currently, the treatment landscape includes hydroxyurea to control counts, while the JAK inhibitors like ruxolitinib (Jakafi) and fedratinib (Inrebic) are known to control the symptoms and splenomegaly. Patients with lower grades of fibrosis can be treated with interferons as well.

For patients who are anemic, some of the available treatment options include lenalidomide (Revlimid), thalidomide (Thalomid), and danazol, and patients who are moving from MF into an acute leukemia can be treated currently with chemotherapy agents such as azacitidine and decitabine.

A promising advancement in the treatment landscape of MF includes the identification of the JAK-STAT pathway, which is targeted with JAK inhibitors. The first JAK inhibitor to receive approval from the FDA was ruxolitinib, followed by fedratinib. Additional agents from this class of drugs are in development as well, which are showing different clinical benefits than what have been observed with these initial agents.

Targeting the JAK-STAT has really been the centerpiece for the treatment of MF," said Gerds, "and you can see JAK inhibitors are all over the place, from low risk to high risk, from the top to the bottom, it's everywhere. It has really become a cornerstone treatment for MF.

Ruxolitinib received its approval in 2011 on the basis of the COMFORT-1 and COMFORT-2 studies, in which ruxolitinib was compared with placebo or best available therapy (BAT), respectively, in intermediate-2 and high-risk patients with MF. Overall, the agent was able to induce spleen volume reduction of 35% in 41.9% of patients by week 24 compared with 0.7% with placebo (P <.0001), while spleen volume was reduced in 28.5% by week 48 in the COMFORT-2 study versus 0% with BAT (P <.0001).2,3

Although ruxolitinib does not kill MF cells, a survival benefit has also been associated with ruxolitinib. According to a pooled analysis from both the COMFORT-1 and COMFORT-2 studies, the median overall survival (OS) in the ruxolitinib arms was 5.3 years (95% CI, 4.7-not evaluable [NE]) compared with 3.8 years (95% CI, 3.2-4.6) in the control arm (HR, 0.70; 95% CI, 0.54-0.91; P = .0065).

Fedratinib received approval in 2019 based on findings from the open-label phase 2 JAKARTA-2 and the randomized placebo-controlled phase 3 JAKARTA clinical trials. JAKARTA demonstrated that 47% of patients receiving 400 mg fedratinib and 49% receiving 500 mg had spleen volume reduction 35% at week 24, while in JAKARTA-2, this was achieved in 53% of patients with intermediate/high-risk MF who were resistant to prior ruxolitinib treatment and 63% of those who were intolerant to the therapy.4,5

Among other JAK inhibitors coming done the pipeline now for the treatment of MF, the 2 agents that are furthest along include pacritinib and momelotinib, which have also demonstrated interesting activity in clinical trials. However, no 2 JAK inhibitors are alike, Gerds explained during his presentation.

Ruxolitinib is a JAK1/2 inhibitor, while fedratinib targets JAK2 and also hits other targets, including FLT3, which may be the cause of some of the particular off-target effects observed with this agent, like diarrhea and nausea. Pacritinib is a JAK2 inhibitor that has very little sensitivity for JAK1, but it is known to have some off-target effects due to also hitting FLT3 and IRAK1, which may be important in this agent. Momelotinib, on the other hand, is a JAK1/2 inhibitor, but it also has off-target effects in ACVR1, which is suspected to help with anemia in some patients.

We can say that there's room for all these JAK inhibitors in the treatment of MF because they all are a little bit different, and they can be applied to different populations of patients with MF, Gerds explained. For example, momelotinib has a positive effect on patients with anemia.

Momelotinib has been evaluated in 2 large randomized phase 3 studies, the SIMPLIFY 1 and SIMPLIFY 2 clinical trials, in which momelotinib was evaluated in patients who were nave to JAK inhibition (n = 432) and those who were previously treated with ruxolitinib (n = 156), respectively.

Spleen volume reductions of 35% at week 24 were observed in 26.5% receiving momelotinib versus 29% with ruxolitinib (P = .011) in the SIMPLIFY 1 study and in 7% who received momelotinib versus 6% with BAT (P= .90) in the SIMPLIFY 2 study. The total symptom score reduction at week 24 with momelotinib was 28.4% versus 42.2% with ruxolitinib (P = .98) in the SIMPLIFY 1 study and 26% with momelotinib versus 6% with BAT (P = .0006) in the SIMPLIFY 2 study.6,7

The JAK1/2 inhibitor momelotinib appears to have the potential to improve anemia via suppression of hepcidin, Gerds said. Momelotinib has been shown to decrease production of hepcidin and to increase serum iron and erythropoiesis, which leads to transfusion independence and an increase in hemoglobin. This served as the rationale for a phase 2 study of 41 transfusion-dependent patients with MF, in which 41% of patients converted to transfusion independence and 78% of nontransfusion-independent patients achieved 50% decrease in transfusions with momelotinib.8

The phase 3 MOMENTUM (NCT04173494) study has been initiated to evaluate momelotinib at 200 mg daily plus placebo against danazol, which is a therapeutic approach for treating anemia, at 600 mg daily plus placebo. Patients enrolled in the study are randomized 2:1 to either the momelotinib or danazol arm. After spleen progression in the control arm, patients are able to cross over to receive momelotinib. This is a global study being conducted in North America, the European Union, and Asia Pacific. Patients must have received prior JAK inhibitor therapy and have symptomatic disease to be included in the study, as well as have anemia. This trial will be able to validate the value of momelotinib in treating anemic patients with MF.

Pacritinib has been evaluated in 3 key studies, including the phase 2 PAC203 study, and the phase 3 PERSIST-1 and PERSIST-2 studies. PAC203 was a dose-finding study in higher-risk patients with MF who previously received ruxolitinib, while PERSIST-1 included higher-risk JAK inhibitornave patients with any degree of anemia or thrombocytopenia and PERSIST-2 included patients with platelet counts 100,000/mcL, allowing for prior JAK inhibitor treatment as well.

Spleen volume reduction 35% at week 24 occurred in 19% of patients in the pacritinib arm versus 5% with BAT (P =.0003) in PERSIST-1, 18% in the pacritinib arm versus 3% with BAT (P =.001) in PERSIST-2. The spleen volume reduction 35% at week 24 in 18% of patients who received pacritinib in PAC203, and the total symptom scores reduced 50% in 7.4% of patients, which was also observed in PERSIST-2 in 25% of those receiving pacritinib and 14% BAT.9,10

Pacritinib was temporarily placed on a clinical hold due to an increased signal for potential cardiac and bleeding complications, but upon a second look at the data from the PERSIST studies, investigators determined that this was a very high-risk population that are very thrombocytopenic and prone to bleeding events.11

Without the JAK1 inhibition in pacritinib, thrombocytopenia is not as concerning as with other JAK inhibitors, Gerds said. The aim of the ongoing PACIFICA (NCT03165734) study is to potentially fulfill the unmet need for patients with MF who have platelet counts less than 50,000 and who are at risk for thrombocytopenic events.

PACIFICA, a randomized phase 3 study, is now ongoing to determine the efficacy of pacritinib compared with the physicians choice of therapy. The primary end point for the trial is spleen volume reduction at 24 weeks, and secondary end points include total symptom score at 24 weeks, OS, and patient global impression change at 24 weeks. Crossover is not allowed in this study.

In the COMFORT studies, the median time on ruxolitinib was around 3 years, but a real-world analysis demonstrated that the average may be much shorter, Gerds explained. Patients who discontinue treatment with ruxolitinib tend to do poorly, and the median OS is short. New treatments are needed to improve outcomes in this patient population.

Unlike in a disease like chronic myeloid leukemia, in which a single mutation could be targeted with a type of agent that would give significant long-term disease control, there are many other pathways outside of the JAK-STAT pathway that could also be targeted in MF, which is where research is now looking to. Momelotinib and pacritinib remain under evaluation in large randomized trials now, and these agents, as well as luspatercept for anemia, appear most promising in terms of becoming available for the treatment of patients with MF in the near future. However, other agents are coming down the pipeline as well that Gerds noted during his presentation.

Novel agent PRM-151 works well in reversing fibrosis in the bone marrow, and bromodomain and extraterminal (BET) inhibitors are also under evaluation in some ongoing studies that are heading into phase 3, such as CPI-0610 for the upfront and post-JAK inhibitor setting. BET inhibitors reduce inflammatory cytokine production in MF, and LSD1 inhibitors have been associated with epigenetic reprograming.

Another promising class of drugs coming down the pipeline for the treatment of MF include JAK2type 2 inhibitors, which hit a different target than the known JAK inhibitors. PI3K inhibitors appear to suppress neoplastic clonal hematopoiesis via cell arrest and apoptosis, while SMAC activation, MDM2, and Aurora kinase A can potentially increase apoptosis.

There is some rationale for targeting the mutant CALR trap, which remains on the horizon for the treatment of patients with MF, as well as chimeric antigen receptor (CAR) T-cell therapies and other novel therapeutic approaches.

Beyond that, we are only limited by our creativity and work that is being done by our colleagues in the lab, both basic science and translational labs, Gerds concluded. More therapeutic treatments will be needed in order to delay progression in early disease, and lead to cure without transplant.

References

1. Gerds A. Myeloproliferative neoplasms: emerging treatment options for myelofibrosis. Presented at: NCCN 2020 Virtual Congress: Hematologic Malignancies; October 9-10, 2020.

2. Verstovsek S, Mesa RA, Gotlib J, et al.A double-blind, placebo-controlled trial of ruxolitinib for myelofibrosis.N Engl J Med. 2012;366(9):799-807. doi:10.1056/NEJMoa1110557

3. Harrison C, Kiladjian JJ, Al-Ali HK, et al. JAK inhibition with ruxolitinib versus best available therapy for myelofibrosis.N Engl J Med. 2012;366(9):787-798. doi:10.1056/NEJMoa1110556

4. Pardanani A, Harrison C, Cortes JE, et al. Safety anf efficacy of fedratinib in patients with primary or secondary myelofibrosis: a randomized clinical trial.JAMA Oncol. 2015;1(5):643-51. doi:10.1001/jamaoncol.2015.1590

5. Harrison CN, Schaap N, Vannucchi AM, et al. Janus kinase-2 inhibitor fedratinib in patients with myelofibrosis previously treated with ruxolitinib (JAKARTA-2): a single-arm, open-label, non-randomised, phase 2, multicentre study.Lancet Haematol. 2017;4(7):e317-e324. doi:10.1016/S2352-3026(17)30088-1

6. Mesa RA, Kiladjian JJ, Catalano JV, et al. Mesa R, et al. SIMPLIFY-1: a phase iii randomized trial of momelotinib versus ruxolitinib in janus kinase inhibitor-nave patients with myelofibrosis.J Clin Oncol. 2017;35(34):3844-3850. doi:10.1200/JCO.2017.73.4418

7. Harrison C, Vannucchi AM, Platzbecker U, et al. Momelotinib versus best available therapy in patients with myelofibrosis previously treated with ruxolitinib (SIMPLIFY 2): a randomised, open-label, phase 3 trial.Lancet Haematol. 2018;5:e73-e81. doi:10.1016/S2352-3026(17)30237-5

8. Oh, ST Talpaz M, Gerds AT, et al. ACVR1/JAK1/JAK2 inhibitor momelotinib reverses transfusion dependency and suppresses hepcidin in myelofibrosis phase 2 trial.Blood Adv. 2020 Sep 22;4(18):4282-4291. doi: 10.1182/bloodadvances.2020002662

9.Mesa RA et al. Pacritinib versus best available therapy for the treatment of myelofibrosis irrespective of baseline cytopenias (PERSIST-1): an international, randomised, phase 3 trial.Lancet Haematol. 2017;4:e225-e236. doi: 10.1016/S2352-3026(17)30027-3

10. Mascarenhas J et al. Pacritinib vs best available therapy, including ruxolitinib, in patients with myelofibrosis: a randomized clinical trial.JAMA Oncol. 2018;4:652-659. doi: 10.1001/jamaoncol.2017.5818

11. CTI biopharma announces removal of full clinical hold on pacritinib. News Release. CTI BioPharma Corp. January 5, 2017. Accessed October 11, 2020. https://prn.to/2GT8PuD

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Treatment Options Expand Beyond JAK Inhibition for Patients With Myelofibrosis - Targeted Oncology

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October 14th, 2020

Yes on 14 | Mailbox | independentnews.com – Livermore Independent

By daniellenierenberg

I am a clinical immunologist that happens to also be a stem cell scientist with 45 years of experience. The first CD34 bone marrow transplantation in 1978 was done at Roswell Park using FACS flow cytometry. We watch GvHD take hold to many leukemia patients to these brave patients trying to save their life with no way to treat them, until now with MSC (mesenchymal stem cells).

I watched many patients give their lives to science research for a chance of cures, which we had successes 40 years forward, if you get CML, CLL you have 98% of treatment or cure. CAR T and other treatments etc.

My concerns (are that) the media is presenting a perspective in vacuum of the stem cell world in California. Prop 71 put California in play and pushed embryonic research. The people of California need to protect their investment of $3.3 billion, or the industry leadership will be lost along with the clinical trials supported by CIRM. Please do not underestimate the RPE for blindness. #1 unmet medical need when the Japanese pharma Astellas bought Ocata in 2015 and put it on the shelf setting back embryonic research.

Lets look at say, Mesoblast, a Australian stem cell company and the leader in field with four studies. (They) had a setback recently of their BLA of SR aGvHD for kids under 12 years old (which is a death sentence) using MSC stem cells (approved for treatment in Japan for two years now) on the first stem cells for regenerative medicine to be approved the FDA, on Sept. 30, 2020. Mesoblast has 330 double blind studies for Covid19 treatment.

We will know before Christmas if FDA will approve these cells. MSC will be better than vaccinations, with super antigens stimulating the immune memory cells being develop by many companies and Federal government.

Two points: federal funding for embryonic research is not very well supported, and you cannot put a price tag on the patients who are willing to put their life on the line for hope and a chance.

Stay in the game California - do not be shortsighted.

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Yes on 14 | Mailbox | independentnews.com - Livermore Independent

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October 14th, 2020

Covid-19 Lockdown Impact: Rheumatoid Arthritis Stem Cell Therapy Market Growth and Demand (YEAR), Projected Fact.MR – The Cloud Tribune

By daniellenierenberg

The globalRheumatoid Arthritis Stem Cell Therapymarket study presents an all in all compilation of the historical, current and future outlook of the market as well as the factors responsible for such a growth. With SWOT analysis, the business study highlights the strengths, weaknesses, opportunities and threats of each Rheumatoid Arthritis Stem Cell Therapy market player in a comprehensive way. Further, the Rheumatoid Arthritis Stem Cell Therapy market report emphasizes the adoption pattern of the Rheumatoid Arthritis Stem Cell Therapy across various industries.Request Sample Reporthttps://www.factmr.com/connectus/sample?flag=S&rep_id=1001The Rheumatoid Arthritis Stem Cell Therapy market report highlights the following players:The global market for rheumatoid arthritis stem cell therapy is highly fragmented. Examples of some of the key players operating in the global rheumatoid arthritis stem cell therapy market include Mesoblast Ltd., Roslin Cells, Regeneus Ltd, ReNeuron Group plc, International Stem Cell Corporation, TiGenix and others.

The Rheumatoid Arthritis Stem Cell Therapy market report examines the operating pattern of each player new product launches, partnerships, and acquisitions has been examined in detail.Important regions covered in the Rheumatoid Arthritis Stem Cell Therapy market report include:

North America (U.S., Canada)Latin America (Mexico, Brazil)Western Europe (Germany, Italy, U.K., Spain, France, Nordic countries, BENELUX)Eastern Europe (Russia, Poland, Rest Of Eastern Europe)Asia Pacific Excluding Japan (China, India, Australia & New Zealand)JapanMiddle East and Africa (GCC, S. Africa, Rest Of MEA)

The Rheumatoid Arthritis Stem Cell Therapy market report takes into consideration the following segments by treatment type:

Allogeneic Mesenchymal stem cellsBone marrow TransplantAdipose Tissue Stem Cells

The Rheumatoid Arthritis Stem Cell Therapy market report contain the following distribution channel:

HospitalsAmbulatory Surgical CentersSpecialty ClinicsHave Any Query? Ask our Industry Experts-https://www.factmr.com/connectus/sample?flag=AE&rep_id=1001

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The Rheumatoid Arthritis Stem Cell Therapy market report offers a plethora of insights which include:

Changing consumption pattern among individuals globally.Historical and future progress of the global Rheumatoid Arthritis Stem Cell Therapy market.Region-wise and country-wise segmentation of the Rheumatoid Arthritis Stem Cell Therapy market to understand the revenue, and growth lookout in these areas.Accurate Year-on-Year growth of the global Rheumatoid Arthritis Stem Cell Therapy market.Important trends, including proprietary technologies, ecological conservation, and globalization affecting the global Rheumatoid Arthritis Stem Cell Therapy market.

The Rheumatoid Arthritis Stem Cell Therapy market report answers important questions which include:

Which regulatory authorities have granted approval to the application of Rheumatoid Arthritis Stem Cell Therapy in Health industry?How will the global Rheumatoid Arthritis Stem Cell Therapy market grow over the forecast period?Which end use industry is set to become the leading consumer of Rheumatoid Arthritis Stem Cell Therapy by 2028?What manufacturing techniques are involved in the production of the Rheumatoid Arthritis Stem Cell Therapy?Which regions are the Rheumatoid Arthritis Stem Cell Therapy market players targeting to channelize their production portfolio?Get Full Access of the Report @https://www.factmr.com/report/1001/rheumatoid-arthritis-stem-cell-therapy-market

Pertinent aspects this study on the Rheumatoid Arthritis Stem Cell Therapy market tries to answer exhaustively are:

What is the forecast size (revenue/volumes) of the most lucrative regional market? What is the share of the dominant product/technology segment in the Rheumatoid Arthritis Stem Cell Therapy market? What regions are likely to witness sizable investments in research and development funding? What are Covid 19 implication on Rheumatoid Arthritis Stem Cell Therapy market and learn how businesses can respond, manage and mitigate the risks? Which countries will be the next destination for industry leaders in order to tap new revenue streams? Which new regulations might cause disruption in industry sentiments in near future? Which is the share of the dominant end user? Which region is expected to rise at the most dominant growth rate? Which technologies will have massive impact of new avenues in the Rheumatoid Arthritis Stem Cell Therapy market? Which key end-use industry trends are expected to shape the growth prospects of the Rheumatoid Arthritis Stem Cell Therapy market? What factors will promote new entrants in the Rheumatoid Arthritis Stem Cell Therapy market? What is the degree of fragmentation in the Rheumatoid Arthritis Stem Cell Therapy market, and will it increase in coming years?Why Choose Fact.MR?

Fact.MR follows a multi- disciplinary approach to extract information about various industries. Our analysts perform thorough primary and secondary research to gather data associated with the market. With modern industrial and digitalization tools, we provide avant-garde business ideas to our clients. We address clients living in across parts of the world with our 24/7 service availability.

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Covid-19 Lockdown Impact: Rheumatoid Arthritis Stem Cell Therapy Market Growth and Demand (YEAR), Projected Fact.MR - The Cloud Tribune

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October 14th, 2020

‘Never give up hope and please join the register’ says 11-year-old in need of stem cell donor match – Asian Image

By daniellenierenberg

An appeal has gone out to help an 11-year-old girlwho needs a stem cell transplant from a stranger, to give her a second chance of life.

Arya was diagnosed with a rare blood disorder. But following a diagnosis of aplastic anaemia, a serious condition that occurs when the body stops producing enough new blood cells, she will also be starting immunosuppressant treatment.

This means her immune system isn't working as it should, putting her at a greater risk of infections.

To cure her aplastic anaemia Arya needs a lifesaving stem cell transplant. Blood cancer charity Antony Nolan is searching the worldwide stem cell registers for a donor whose tissue type matches Arya's and who is willing to donate their stem cells to help her live a normal life again.

However, the search for a perfect match is difficult for people like Arya, who is half Indian, with mixed ethnicity so she is sharing her story with Anthony Nolan in order to raise awareness of the need for more people of mixed race to join the stem cell register.

She said, "'They said it would be hard to find a donor for me because of my ethnicity but it isn't impossible. There is hope."

The best possible match for Arya is most likely to have the same background or mix of ethnicities. Currently, people with mixed Asian or other minority backgrounds have a 20% chance of finding a match from an unrelated donor, compared with nearly 70% for people with white, north European heritage.

Arya was diagnosed earlier this year and is receiving treatment at St Mary's Hospital, London.She added, 'When I first became unwell, I remember getting a stomach ache. At first it felt like a stitch but the pain didn't go away so I had more tests.'

These tests revealed something more serious. Arya's mum Brundha recalls: "Arya has always been fit and healthy, but life changed very quickly; all of a sudden we were talking to doctors about aplastic anaemia and Arya has had to stop many of the things she liked doing because her platelets, the tiny blood cells that help your body form clots, were low."

The family were given news of the treatment Arya would need to undergo and the need for a suitable donor.As the search continues, waiting for a match for Arya has inspired the Lloyd family to share their story. Their aim is to raise awareness of the need for more stem cell donors of mixed ethnicities to join the Anthony Nolan register and so increase the likelihood of finding a match for young people like Arya.

Brundha said: 'Because Arya is of mixed race, it was always unlikely we would find a match quickly. We have therefore started this appeal because we don't want to give up hope. It's a waiting game, but there could be someone out there who is a match. We also understand that younger people make better matches, so we would like to do all we can to make this more widely known.'

Aryas Consultant, Professor Josu de la Fuente, who is a Consultant Haematologist and Director of the Paediatric Bone Marrow Transplant Programme at Imperial College Healthcare in London said A well matched donor offers the best opportunity for Arya to establish normal blood production long-term and not to worry about the future.

"I will urge anyone, but particularly those of mixed ethnicity to consider joining the Anthony Nolan register so that no child with blood disorders faces an uncertain future: we can all contribute and be part of the solution.

Arya added: 'What stands out most for me are the bone marrow biopsies and being undergeneral anaesthetic for the first time.'

Rebecca Pritchard leads Anthony Nolan's work to recruit donors aged 1630 to its stem cell register. Rebecca says: 'Despite all she is going through, Arya is standing up to share her story in order to inspire people of mixed background to join the register. There is a potential lifesaver out there who could help her. If you're aged 1630 you can join the Anthony Nolan register online by completing a form and swabbing your cheeks with swabs we'll send in the post.

'Each time we're told a patient is in need of a transplant we'll check whether you're a match for them; if you're found to be a match you could give your stem cells to give hope to families like Arya's.'

Brundha said, 'We were unprepared for this and when it happens you want to know there is a source of donors for your child. That's why we're doing this to highlight the need. Being on the register could have a major impact on someone else's life. It's such an important thing you could do without realising.

"Families would be eternally grateful. You may never be called on, but if you are you could be a lifeline for someone. One person out there could be that person. It's a win-win for everybody.'

Arya added: 'The message I would like people to take away is Never give up hope and please join the register.

To find out more about joining the Anthony Nolan register, or to find out more about how you can support the charity click here

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'Never give up hope and please join the register' says 11-year-old in need of stem cell donor match - Asian Image

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October 14th, 2020

Stem Cell Banking Market to witness an impressive growth during the forecast period 2020 2025 – Express Journal

By daniellenierenberg

In the latest Stem Cell Banking market report, factors that are positively impacting the industry progression as well as the major threats & challenges existing in this domain are expounded. To unveil all the possible opportunities for business expansion, the study scrutinizes the regulatory and macroeconomic framework across the various geographies. It also delves into the competitive dynamics and evaluates how it will evolve during the forecast period. Further, it suggests strategies for dealing with the impact of the COVID-19.

Key highlights from COVID-19 impact analysis:

A gist of the regional landscape:

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Other highlights from the Stem Cell Banking market report:

Market Status:The complete details on Stem Cell Banking Market situation, principal regions, distribution channels, pricing structures are blanketed.

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Strategic Analysis Covered in TOC: - Key Topics Covered

Initially, the document offers an outline of the global market with a complete take a look at key drivers, constraints, challenges, traits and product types sold by using the employer. The file studies the Stem Cell Banking market capacity of key packages with the identity of forecast opportunities. The local evaluation with a focus on specific international locations and area of interest markets is presented. The pinnacle organization profiles with key-word market size and proportion estimation, revenue strategies, products, and other factors are studied.

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Stem Cell Banking Market to witness an impressive growth during the forecast period 2020 2025 - Express Journal

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October 14th, 2020

Clinical trials with stem cells to treat effects of COVID-19 in the lungs advance – Granma English

By daniellenierenberg

Photo composition: Claudia Garca Martnez

Dr. Odalis Mara de la Guardia Pea, an expert immunologist, describes as "encouraging" preliminary findings obtained at the conclusion of the first phase of clinical trials evaluating the use of stem cells in patients facing lung damage caused by COVID-19.

The study, begun during the month of May at the Cuban Institute of Hematology and Immunology (IHI), was undertaken with a view toward eliminating or reducing interstitial inflammatory or fibrotic lung lesions following the infection.

The doctor, also an infectious disease specialist and head of External Services at the IHI, explains that the research will have significant impact "if, as we hope, stem cell therapy produces positive results in these patients with pulmonary alterations post-COVID-19.

"If the treatment is effective, it will be generalized across the entire country, improving the quality of life and respiratory capacity of these patients," she stated with the enthusiasm of someone devoted to the most important mission in the world: saving lives.

THE LUNG, THE "TARGET" ORGAN

De la Guardia Pea commented that, although SARS-COV-2 has a variety of dissimilar effects (cardiovascular, renal, cerebral, vascular, in distal or lower limbs, and others); the "target" organ in the case of COVID-19 is the lung, in which patients experience the most serious impact, both during the disease and once they have recovered, a pattern being studied internationally.

"We have detected cases, specifically in Cuban patients, who have presented this kind of affectation, especially those who have suffered symptoms over a longer period. Among those visited for the study, there were cases of important pulmonary alterations, which is the most frequent, but perhaps not the most serious," the specialist continued.

RECRUITMENT OF VOLUNTEERS

"These recruitment consultations were atypical, as they were done in the field, visiting the homes of recovered patients," the doctor explained, adding that potential volunteers needed to meet several criteria for inclusion in the clinical trials.

Those selected were between 18 and 70 years of age, of both sexes, who had contracted COVID-19 thirty days prior to the trial treatment, testing negative on a PCR at the time of recruitment, and exhibited respiratory symptoms since the beginning of the disease.

Specifically sought for the trials were patients who experienced a more torpid evolution of the disease, those who were hospitalized for more than 20 days, requiring oxygen, assisted ventilation, or the use of some aerosol as treatment, upon reaching serious or critical condition.

"More than 130 homes were visited over almost three months, from May to June; and 141 patients were interviewed, of which about 50 were studied. Twenty patients were included in the trial, which was the determined number," the doctor reported.

PULMONARY SEQUELAE

"During the investigation, several long term effects of COVID-19 were noted, although the most frequent involved the lungs. In some cases, indications of pulmonary fibrosis were detected, a condition that cannot be completely corrected, and can only be treated to increase lung capacity and improve quality of life," the doctor explained.

"The study is still in progress. The first phase has been completed, but there is some time remaining before final evaluation of the patients. What we can say is that, thus far, we are very happy with the results we have observed, they are encouraging," she emphasized.

UNFORGETTABLE STORIES

-Could you recount some stories that particularly impacted you?

-The first day I went out to recruit volunteers, I arrived at the home of a patient who, when she opened the door, exhibited obvious difficulty breathing, evident in plain sight.

We conducted the interview and learned that she experienced this difficulty on a daily basis, five weeks after being diagnosed with COVID-19 and 15 days after a negative PCR test.

This case was significant because we became aware of the lingering effects some patients face, who after having the disease, being discharged and completing all treatment, can have symptoms for a long time.

On another occasion, a patient received us effusively, grateful that he would continue to be treated, that he would receive some follow-up. This attitude was very common in many cases, confirming for us that the patients we visited were still feeling unwell, despite having recovered and been discharged from the hospital.

YOU CAN BE ASYMPTOMATIC OR YOU CAN DIE

"You can be infected and be asymptomatic, or develop the most severe symptoms of the disease and die. This is random, no one understands or can control it," the specialist warns, emphasizing the importance of being fully conscious of taking care of ourselves, since anyone can develop an aggressive case of COVID-19.

"I agree with everything Professor Durn says every day at nine o'clock in the morning, about how measures must be maintained and complied with: the use of facemasks, hand washing, shoe disinfection (with doormats soaked in 0.5% hypochlorite at the entrance to common areas), social distancing, and collective discipline.

"The population must take care; success in containing the pandemic lies in individual responsibility," she concluded.

STEM CELL TREATMENT

-When the patient is included in the study, treatment begins by injecting the granulocyte colony stimulating factor, Ior Leukocim, a product manufactured at Cubas Center for Molecular Immunology, to achieve the mobilization of stem cells from the bone marrow to the bloodstream.

-Subsequently, the patient's blood is extracted and mononuclear cells are separated and concentrated.

-This pool of cells includes hematopoietic and non-hematopoietic stem cells, which have immune-regulatory properties and promote the disappearance of lesions and the reconstitution of lung tissue.

-The cells are infused intravenously.

-The patient is evaluated one month following treatment and again at six months, to determine the clinical efficacy of the stem cell therapy.

Source: Granma interview with Consuelo Macas Abraham, director of the National Institute of Hematology and Immunology.

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October 14th, 2020

This Dublin-based startup raises 30M to develop promising therapies for leukemia – Silicon Canals

By daniellenierenberg

The global blood and bone marrow cancer treatment market was valued at $38.8B (approx 32.8B) in 2018 and is reportedly expected to reach $74.9B (approx 63.4B) by 2027, expanding at a CAGR of 7.7% from 2019 to 2027.

Blood cancer begins in the bone marrow which is the integral source of stem cells, which are later differentiated into different types of blood cells in the human body. Researchers have stated that approximately 1.85 million new cases of blood cancer will be diagnosed by 2040 throughout the globe.

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Europe holds a market share of 30.8% owing to the supportive regulatory framework provided by the European Medical Agency for the development and sale of medication for the treatment of blood cancer.

In the recent development, blood and bone marrow cancer treatment developer Priothera Limited, has raised 30M in its Series A round of funding led by Fountain Healthcare Partners with participation from co-lead investor HealthCap and funds managed by Tekla Capital Management, LLC, as well as EarlyBird Venture Capital.

According to the medtech startup, the raised funds will be used to progress the clinical development of mocravimod a modulator of sphingosine 1 phosphate (S1P) receptors, to enhance the curative potential of allogenic hematopoietic stem cell transplantation (HSCT) for treating AML.

Priothera expects to generate further randomised clinical data in high-risk AML patients with these funds.

Dublin-based Priothera was founded in 2020 by Drs. Florent Gros and Dhaval Patel. Joining the founding team include experienced industry executive, Dr. Christoph Bucher, Dr. Simone Seiter, and CFO Brice Suire.

The company claims to be leading the way in developing orally applied sphingosine 1 phosphate (S1P) receptor modulators for haematological malignancies. S1P receptor modulators have been suggested to largely reduce egress of T cell subsets from lymphatic tissues allowing for dual inhibition of graft-versus-host-disease (GvHD) and enhancing graft-versus-leukemia benefits in patients receiving allogenic stem cell transplant.

Allogenic stem cell transplant is the only potentially curative approach for AML patients but has unacceptably high mortality with current treatments, says Florent Gros, co-founder, and CEO of Priothera.

Florent Gros further adds, We are excited about mocravimod which has a unique mechanism of action and clinical proof of concept demonstrating its ability to improve survival outcomes for this devastating disease.

Acute myeloid leukemia (AML) is an aggressive and highly proliferative form of cancer where the bone marrow generates abnormal myeloblasts (a type of white blood cell). According to the company, AML is the most common form of leukemia in adults and can metastasise quickly if left untreated. This can typically lead to death within a few months of diagnosis.

Priothera has acquired rights to a drug called mocravimod from Japans Kyorin Pharmaceutical for the treatment of acute myeloid leukaemia.

According to the company, Mocravimod has already been extensively tested in multiple immunologic indications and has shown a survival benefit in an early clinical study evaluating acute myeloid leukemia (AML) and acute lymphocytic leukemia (ALL) patients undergoing hematopoietic stem cell transplantation (HSCT).

Priothera is developing mocravimod in AML with the aim of enhancing the curative potential of Hematopoietic Stem Cell Transplantation (HSCT). The company claims that promising early clinical results have revealed that mocravimod has the potential to rebalance the patients immune system by decoupling Graft-versus-Host Disease (GvHD) from Graft-versus-Leukemia (GvL), preventing the first and preserving the latter.

Following the closing of the financing, people who have joined the Board of Directors include Florent Gros (Priotheras co-founder and CEO), Dr. Dhaval Patel (Priotheras co-founder and CSO at UCB), Dr. Manus Rogan (Fountain Healthcare Partners co-founder and MD), Dr. Marten Steen (partner at HealthCap), Dr. Henry Skinner (senior vice president at Tekla Capital Management, LLC) and Lionel Carnot (partner at EarlyBird Venture Capital).

Image credits: Jarun Ontakrai/ShutterStock

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October 14th, 2020

Direct Biologics Granted Expanded Access by FDA for ExoFlo in the Treatment of COVID-19 – PRNewswire

By daniellenierenberg

AUSTIN, Texas, Oct. 13, 2020 /PRNewswire/ --Direct Biologics, LLC, announced today that the FDA has granted expanded access for ExoFlo in the treatment of patients with COVID-19 associated acute respiratory distress syndrome (ARDS).

While Direct Biologics is already enrolling patients in EXIT COVID-19, its national multi-center, Phase II, placebo controlled, randomized clinical trial, the new expanded access protocol will make ExoFlo available to a broader group of patients with severe COVID-19, many of whom would not meet acceptance criteria for EXIT COVID-19, often under conditions of "compassionate use."

Co-Founder and Chief Executive Officer, Mark Adams, states, "The FDA's approval of expanded access for ExoFlo signifies a critical milestone in the development of advanced treatment for COVID-19 associated illnesses including ARDS. We are excited to be able to provide our product to patients with ARDS associated with COVID-19 in critical need of treatment."

"Amid a potential surge in new COVID-19 cases moving into the fall and winter seasons, this approval could not have come at a better time," notes Joe Schmidt, Co-Founder and President. "Our team is working hard to advance our Phase II EXIT COVID-19 trial to offer additional treatment options."

"Approval of our expanded access protocol offers an option for doctors to administer ExoFlo as a treatment to reverse disease progression, extending hope to COVID-19 patients who are not responding favorably to standard of care," states Chief Medical Officer, Vik Sengupta, MD. "We at Direct Biologics are grateful for every opportunity to help these additional patients in need."

Also in attendance was Congressman Michael McCaul who commented, "I am thrilled to hear a local company from Austin, Texas, has been approved for Expanded Access IND by the FDA. Their product, ExoFlo, will help treat COVID-19 patients who are at risk of severe respiratory infection, which often leads to life-threatening circumstances. Direct Biologics, with support of the FDA, will bring high-class treatments and services to Americans who need them the most."

This approval comes on the heels of multiple approvals for single patient Emergency Investigational New Drug (eIND) applications granted in September and October. Emergency INDs are a mechanism by which physicians can obtain rapid approval to administer medication to a single patient through a direct appeal to the FDA.

About ExoFlo ExoFlo is an investigational new drug that has not been approved or licensed by FDA. It is an extracellular vesicle product isolated from human bone marrow mesenchymal stem or stromal cells (MSCs). ExoFlo provides natural bioactive signals that have been shown to modulate inflammation and direct cellular communication.

About Direct Biologics Direct Biologics, LLC, is headquartered in Austin, Texas, with a recently expanded R&D facility located at the University of California, and an Operations and Order Fulfillment Center located in St. Louis, Missouri. Direct Biologics is a market-leading innovator and cGMP manufacturer of regenerative medical products, including a robust line of extracellular vesicle-based biological products. The Company was created to expand the science of regenerative healing by delivering cutting-edge biologic technologies. Direct Biologics' management team holds extensive collective experience in biologics research, development, and commercialization, making the Company a leader in the evolving, next generation segment of the biotherapeutics industry. Direct Biologics is dedicated to pursuing additional clinical applications of its extracellular vesicle biologic products through the FDA's investigational new drug application process. For more information visit http://www.directbiologics.com.

Phone:1-800-791-1021Email:[emailprotected]

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October 14th, 2020

Lymphoma: Higher rates of survival on the horizon – The Straits Times

By daniellenierenberg

To keep you healthy from day to day, your body has its own in-built, germ-fighting network.

This network is also known as the lymphatic system, made up of different vessels and organs in your body, from the lymph nodes to the bone marrow. Lymphoma is the name of the cancer that affects this system.

There are many types of lymphoma but Diffuse Large B-Cell Lymphoma (DLBCL) is the most common aggressive type of non-Hodgkin lymphoma, affecting the B-lymphocytes that produce antibodies which help your body fight infections.

According to the World Health Organizations guidance on classifying tumours, DLBCL accounts for 30 to 40 per cent of newly diagnosed cases of non-Hodgkin lymphoma globally.1

In Singapore, Dr Daryl Tan Chen Lung, who specialises in haematology and practises at Mount Elizabeth Hospitals, estimates that lymphoma is the fifth most common cancer in Singapore.2

Unlike other cancer patients, DLBCL patients hardly have any contributing factors to the development of the condition not even hereditary ones though people with existing HIV infections are more prone to getting DLBCL.

As a result of this, and also because lymphoma is less common than other cancers like breast or lung cancer, regular medical check-ups do not test for its genetic indicators. Instead, patients seek medical help only when they spot early symptoms, says Dr Tan. A growing awareness of lymphoma World Lymphoma Awareness Day takes place on Sept 15 each year and better diagnostics measures have led to much earlier detection of the cancer today, compared to a decade ago.

Dr Tan says that early symptoms of DLBCL include lumps in the neck or groin. Sometimes, other organs like the spleen and bone marrow can be affected. However, some patients show no obvious symptoms beyond abdominal pain, fever and loss of weight. It is thus especially important to seek medical attention should you display any of the common symptoms of lymphoma including swollen lymph nodes in the neck, armpit or groin, a persistent fever, excessive night sweats and unexplained weight loss.

Dr Tan adds that the median age of DLBCL patients in Singapore is 60 to 65, though a very small number of patients in their 20s are diagnosed with a subtype of the cancer known as primary mediastinal B-cell lymphoma.

While DLBCL is an aggressive cancer, the good news is that it is highly treatable in the early stages.

Current therapies for treating first-line DLBCL include chemotherapy, chemo-immunotherapy a combination use of chemotherapy and immunotherapy and stem cell transplant in certain groups of patients. About six in 10 of these patients will respond to their treatment3 and not suffer a relapse within the next five years.

This has a huge impact on younger patients who are then able to resume their daily life and go back to work, for example.

Dr Tan remembers a female patient in her early 20s whose career was just taking off. She was admitted with fever and breathlessness. After performing scans on her, it was discovered that there was fluid surrounding her heart and lungs, and she had a tumour in her chest the size of a rugby ball.

Fortunately, because the cure rate for DLBCL is high, the patients condition has since gone into remission.

A combination of chemo-immunotherapy drugs is commonly recommended as a first-line treatment in DLBCL. Radiation therapy or stem cell transplant can also be included as part of the treatment.

However, about four in 10 patients may not eventually respond to these therapies or suffer from a relapse.3 When this happens, treatment options are limited. Some patients can undergo a stem cell transplant, but about half of them are not eligible.4 This group includes older patients with compromised immune systems.

While affected patients can consider continuing with chemotherapy and chemo-immunotherapy, there are new promising treatment options on the horizon, including targeted therapies such as CAR T-cell therapy. These targeted therapies involve using the patients own immune cells also known as T cells or molecules that bind a chemotherapy agent to an antibody to fight cancerous blood cells.

Beyond that, there are also treatment options known as combination therapy. Previously, patients suffering a relapse have had to rely on existing, first-line treatment, which may or may not be effective in combating the cancer. Combination therapy uses the collective effect of different medications to target different areas of cancerous B-cells and destroy them.

Says Dr Tan: [The medical community is] seeing progress in DLBCL immunotherapy. The current cure rate is about 70 per cent. Hopefully, we can increase the number to 80 per cent or more. But we mustnt rest on our laurels as we hope to focus on the 20 per cent of patients who still dont respond to any treatments.

For more information on lymphoma and treatment options, please speak to your healthcare specialist.

1. Lyon, France. World Health Organization Classification of Tumors of Haematopoietic and Lymphoid Tissues. IARC Press; 2008.2. SingHealth. (n.d). Lymphoma. Retrieved on 30 Sept 2020. (https://www.singhealth.com.sg/patient-care/conditions-treatments/lymphoma)3. Maurer, MJ et al. Event-free survival at 24 months is a robust end point for disease-related outcome in diffuse large B-cell lymphoma treated with immunochemotherapy. J Clin Oncol. 2014; 32: 1066-73.4. Gisselbrecht C, Van Den Neste E. How I manage patients with relapsed/refractory diffuse large B cell lymphoma. Br J Haematol. 2018;182:633643.

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October 14th, 2020

Stem cells are believed to play a key role in hair loss – FLWL News

By daniellenierenberg

Using a brush or comb, in the shower, passing your hand through your hair In a 2015 IFOP survey, three out of four French people (76%) without baldness declared that they lost their hair. In addition, after 65 years, three in ten men suffer from androgenetic alopecia (or baldness). In general, therefore, we often see a loss of density with age, both in men and in women. However, science has long looked into the phenomenon of age-related hair loss, both to understand its origin and to treat it. Focusing on stem cells seems to offer the best hope for a cure, based on numerous studies.

The hair transplant procedure is indeed a sometimes expensive and invasive procedure. As for drugs, finasteride used for androgenetic alopecia in men can induce side effects such as loss of libido and erectile dysfunction while minoxidil can cause hypertrichosis. Today, researchers therefore seem to be looking more at alternative solutions, and in particular by focusing on stem cells.

Stem cells, directly linked to hair lossFor decades, researchers have always focused on keratinocytes, cells that make up the epidermis and integuments (body hair, hair, etc.). However, as part of a study conducted by the University of Calgary (Canada), the team of researchers focused on a small cell group present in hair follicles and in skin stem cells. : fibroblasts. And according to them, these fibroblasts are the main cause of age-related hair loss.

By studying the sparse coat of elderly mice, they noticed that fibroblast stem cells had lost their regenerative function or were malfunctioning. There werent enough of them to regenerate fibroblasts. As a result, the fibroblasts and hair follicles began to miniaturize and were no longer able to produce hair, explains Biernaskie, head of the research team.

Remember that fibroblasts are important because they send messages to keratinocytes to force them to divide, and in so doing, orchestrate the growth cycles of hair follicles allowing the production of new hairs. When the fibroblasts become scarce, the signal then becomes too weak to reach the keratinocytes and maintain the process of capillary growth. For Jeff Biernaskie: if we want to one day succeed in preventing hair loss or re-grow those that are already falling, we must work to preserve the function of these stem cells which are found in the hair follicles.

Against hair loss, but not only!This finding may help guide future research on hair loss more precisely. Scientists at the University of Calgary are particularly hoping to find a way to prevent this degeneration by blocking certain genetic mutations that occur directly in stem cells in fibroblasts.

They also believe that this will have wider implications. Indeed, Wisoo Shin, lead author of the study, points out that similar fibroblasts are found in most of our organs, maintaining their integrity and promoting tissue regeneration. Finding a way to promote self-renewal to produce new functional fibroblasts into old age therefore also offers the hope of being able to treat certain injuries and help the skin to regenerate.

Lamia spent a couple of years interning at an organization that offered medical consultation before joining the editorial team at FLWL News. An enthusiastic fitness freak in the room, she offers the best amounts of insights and craft-based writing style to keep us up to date about the medicine industry, health and science.

Email:lamia@flwl.orgPhone: +1 512-845-8162

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October 14th, 2020

Brain organoids reveal neuronal flaws in syndrome tied to autism – Spectrum

By daniellenierenberg

Electric organoid: Neurons derived from people with 22q11.2 syndrome are hyperexcitable and show calcium-signaling deficits.

Courtesy of Pasca lab

Neurons derived from people with 22q11.2 deletion syndrome, a genetic condition linked to autism, show deficits in electrical activity and calcium signaling, according to a new study1. A single gene appears to be largely responsible for these defects, the study shows.

Up to 20 percent of people who lack part or all of the chromosomal region 22q11.2 have autism. Individuals with the deletion may also have schizophrenia, seizures, heart defects, immune dysfunction or learning problems.

The new findings uncover factors that may contribute to the development of psychiatric conditions associated with 22q11.2 deletion syndrome. They could also help researchers identify new therapeutic targets, says lead author Sergiu Pasca, associate professor of psychiatry and behavioral sciences at Stanford University in California.

The syndrome is relatively common, occurring in up to 1 in 4,000 newborns, Pasca says. But researchers do not fully understand how genes in the 22q11.2 region contribute to autism or other conditions, he adds.

To solve this molecular puzzle, Pasca and his team reprogrammed skin cells from 15 people with the deletion and 15 controls into induced pluripotent stem cells. Using a technique they developed in 2015, they coaxed these cells to turn into neurons, which self-organize in a dish into spherical clusters called organoids. The organoids show some key features of the developing cerebral cortex, a brain region implicated in autism.

The neurons derived from people with 22q11.2 syndrome spontaneously fire four times as frequently as neurons derived from controls, the researchers found. And the electrical activity of the 22q cells does not set off the usual spike in calcium levels, which is crucial for neurons to exchange messages.

In some other syndromes tied to schizophrenia and autism, calcium-channel genes are mutated. But the number of channels and the speed at which they work in 22q neurons is the same as in control neurons. Instead, the 22q cells show an unusually low voltage difference across the cell membrane when they arent firing, causing the signaling defects and hyperexcitability, the researchers found.

The researchers suspected that a gene called DGCR8 might be responsible for the neuronal deficits in the organoids because it lies within 22q11.2 and is linked to abnormal electrical activity in the neurons of mice2. DGCR8 is essential for the synthesis of short RNA fragments, called microRNAs, that regulate gene expression.

Lowering DGCR8s expression levels in control neurons reproduced the abnormalities seen in 22q neurons. In contrast, boosting the genes activity in 22q neurons or treating them with antipsychotic drugs prevented them from being overly excitable and reversed their calcium-signaling defects. The study was published 28 September in Nature Medicine.

Previous studies have analyzed lab-grown neurons derived from people with schizophrenia or autism-related disorders such as Rett and fragile X syndromes. But most used only a few human-derived cell lines, says Guo-li Ming, professor of neuroscience at the University of Pennsylvania in Philadelphia. The new study, Ming says, has a total of 30 human lines thats a huge effort.

By studying brain organoids derived from so many people, the researchers were able to identify the gene that might be involved in the psychiatric conditions associated with 22q11.2 syndrome, says Sally Temple, scientific director of the Neural Stem Cell Institute in Rensselaer, New York. Whenever we have a light shining ahead, saying, This is what you should really be looking at, it means that were making progress, she says.

The study participants with 22q11.2 syndrome vary in their psychiatric diagnoses, and yet all the brain organoids derived from their cells show the same neuronal abnormalities. Thats somewhat surprising, because we know there are a lot of differences in the genetic background of different people, Ming says.

The deletion might conspire with other factors to ultimately determine which psychiatric conditions a person has, Pasca says. It could be that the deletion causes cellular defects, and once there is a stressor such as social stress, disease develops. Its also unclear whether these cellular defects are related to the high prevalence of seizures in people with 22q11.2 syndrome, he says.

The hallmarks of most neuropsychiatric conditions can change over time, says Giuseppe Testa, director of the stem cell epigenetics unit at the European Institute of Oncology in Milan, Italy. Studies that look at a larger number of people with 22q11.2 deletion syndrome or other neurodevelopmental conditions could help to elucidate the relationship between the neuronal defects observed in the lab and the psychiatric manifestations of the conditions, Testa says. The new study, however, is a great resource for understanding how the 22q11.2 deletion contributes to schizophrenia and autism, he says.

Pascas team is trying to pinpoint molecules that could open new therapeutic avenues for 22q11.2 deletion syndrome. The antipsychotics they tested restore the unusual voltage differences in the 22q neurons, but they dont address the core mechanisms responsible for psychiatric conditions linked to the syndrome, Pasca says.

Whats more, antipsychotics have many side effects, and not all individuals respond to them, he says. We need better therapies we need to identify what the key molecular players are and target those.

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October 14th, 2020

Becoming a donor easier than you think – Randfontein Herald

By daniellenierenberg

When thinking about donating bone marrow, most will break out in a cold sweat.

The thought of needles, prodding and poking is enough to put anyone off from becoming a donor but Ndinae Muligwe, Sustainability and Donor Recruitment Coordinator for the South African Bone Marrow Registry (SABMR) explained that it is a less complicated and relatively painless process.

The SABMR was established in 1991 and is a non-profit organisation that conducts searches to find matching bone marrow donors for critically ill children and adults in South Africa who cannot find a match in their own families.

Bone marrow transplants help to treat and even sometimes cure illnesses like leukaemia, Non-Hodgkin lymphoma, bone marrow failure, and some genetic blood and immune-system disorders.

Ndinae explained that the likelihood of a donor finding a match is about one in 100 000. What is more concerning is that there are currently only around 74 000 local donors on the South African Bone Marrow Registry.

Although they do form part of the World Marrow Donor Association that represents about 38 million donors, there are not enough donors for the South African demographic.

Ethnicity plays a role when it comes to who is able to donate, and at the moment the numbers do not match the ethnic groups represented in South Africa. You are more likely to find a match within your own ethnic group.

But how do you become a donor and what is the process involved?

Ndinae said it is as easy as registering on the website. Of course there are some questionnaires to fill in and you will have to meet the criteria and be healthy.

The donating age has recently been lowered from 18 to 16 years of age, and applicants must be between 16 and 45 to register as a potential donor.

If you are eligible you will then be contacted by the SABMR to do a cheek swab free of charge.

Peripheral blood stem cell (PBSC) collection is the most likely way of collecting stem cells. These cells are found in your bone marrow and also the blood stream. A five-day course of growth factor or Granulocyte-Colony Stimulating Factors is given prior to the donation to encourage the stem cells to move from your marrow to your blood.

At the time of donation a needle is placed in one arm. The blood is then passed through a machine that collects the stem cells, and the remaining blood is returned to your body similar to donating blood platelets.

You do not have to pay for anything to make a tissue or blood donation of your bone marrow stem cells, the SABMR covers the cost of testing and collection.

Visitwww.sabmr.co.zafor more information.

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October 10th, 2020

COVID Drug Given to Trump Developed From Aborted Fetus Cells – Quint Fit

By daniellenierenberg

Embryonic stem cell research has been always disputed by the 2020 Republican party. In 2019, Trumps administration paused funding for government scientists to work on studies involving embryonic stem cells, affecting about $31m in research, according to Science Magazine.

Regeneron, on the other hand, doesnt consider these cells fetal tissue because the HEK-293T line of cells has been immortalized and they divide and regenerate themselves in the laboratory.

The investigational drug has been in clinical trials since June. Even though early results from a trial with around 300 non-hospitalised COVID patients showed the drug was safe and could reduce viral levels and improve symptoms, the data is yet to be peer-reviewed.

According to CNN, the treatment is not yet approved for any use from the US FDA. The company, however, is in talks for an emergency approval. Regeneron has also confirmed that it had provided the drug under a compassionate use request for President Trump from the doctors.

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October 10th, 2020

AIVITA Biomedical’s Stem Cell Therapeutic in Vision Loss Published in Investigative Ophthalmology & Vision Science – PRNewswire

By daniellenierenberg

IRVINE, Calif., Oct. 9, 2020 /PRNewswire/ --AIVITA Biomedical, Inc., a private biotechnology company developing personalized vaccines for the treatment of cancer and COVID-19, announced today the publication of the peer-reviewed manuscript, "Retina organoid transplants develop photoreceptors and improve visual function in RCS rats with RPE dysfunction,"in the journal Investigative Ophthalmology & Visual Science. The study, led by researchers at AIVITA Biomedical and the Sue & Bill Gross Stem Cell Research Center of the University of California, Irvine, used 3D-retina organoids generated from human stem cells developed by AIVITA to provide insight into the potential use of transplanted retina organoids as a therapeutic option for blinding diseases.

In the study, transplanted retina organoid sheets were examined to determine if human stem cell-derived photoreceptors coulddevelop, survive and function in vivo without the support of healthy retina pigment epithelium (RPE). Visual function was examined through a variety of tests, including optokinetic testing (OKT), electroretinogram (ERG), and superior colliculus (SC) brain recording. These tests concluded that retina organoid transplantations demonstrated significant improvement in visual function compared to non-surgery and sham surgery controls, supporting the application of AIVITA's stem cell technologies in visual disease therapeutics.

"Leveraging our expertise in stem cell growth and differentiation, I'm excited to see the promise of our technology platform in potential therapeutics for vision loss," said Hans Keirstead, Ph.D., chief executive officer of AIVITA and a contributing author to the paper. "To our knowledge, this study is the first to show that it's possible for photoreceptors derived from stem cells to survive and function after transplantation when a host has a dysfunctional RPE."

This work is supported by funding from the California Institute for Regenerative Medicine (CIRM) and National Institutes of Health (NIH).

About AIVITA Biomedical AIVITA Biomedical is a privately held company engaged in the advancement of commercial and clinical-stage programs utilizing curative and regenerative medicines. Founded in 2016 by pioneers in the stem cell industry, AIVITA Biomedical utilizes its expertise in stem cell growth and directed, high-purity differentiation to enable safe, efficient and economical manufacturing systems which support its therapeutic pipeline and commercial line of skin care products. All proceeds from the sale of AIVITA's skin care products support the treatment of people with cancer.

SOURCE AIVITA Biomedical, Inc.

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October 10th, 2020

U of T’s Medicine by Design invests $1 million to advance new ideas in regenerative medicine – News@UofT

By daniellenierenberg

Patients with cystic fibrosis experience recurrent lung infections that eventually destroy their airways, shortening their average life expectancy to 50 years in Canada. Current drug treatments, which target a malfunctioning pathway in cells that causes the infections, are costly and have varying effectiveness.

Now, with funding from Medicine by Design, a researcher at the Hospital for Sick Children (SickKids) is combining stem cells, gene editing and computational modelling to try to hijack an alternative cell pathway in the hopes of restoring lung function in these patients.

If successful, our study will be the first to provide proof-of-concept that this alternative approach to treating cystic fibrosis is effective, saysAmy Wong, a scientist working in developmental and stem cell biology at SickKids who is also an assistant professor in the department of laboratory medicine and pathobiology in the University of Torontos Temerty Faculty of Medicine.

Wongs project is one of seven across U of T and its affiliated hospitals that have been awarded 2020New Ideas AwardsandSeed Fundawards from Medicine by Design. Through a $1 million investment, Medicine by Design is supporting research aimed at advancing new concepts expected to be important to regenerative medicine in the coming years. The funded projects will have potential impacts in diseases and conditions such as vision loss, amyotrophic lateral sclerosis (ALS), intestinal disease in premature babies and more.

Supporting novel strategies and approaches is crucial to moving regenerative medicine into the future, saysMichael Sefton, executive director of Medicine by Designand a University Professor at U of Ts Institute of Biomedical Engineeringand thedepartment of chemical engineering & applied chemistry in the Faculty of Applied Science & Engineering.

Our 2020 New Ideas project portfolio integrates mathematical modelling, physics and computational biology with stem cell biology and biomedical engineering, and strengthens engagement with clinicians who are key to translating our research into patient impact. We are particularly delighted this year to support so many outstanding early-career researchers, who will ensure Toronto remains a global leader in regenerative medicine for years to come.

Wong is one of three investigators to receive a 2020 New Ideas Award, which is valued at $100,000 per year for up to two years. Four additional projects were selected for Seed Fund Awards of $100,000 each for one year to further develop their potential.

Medicine by Design selected the funded projects from among 36 short-listed proposals, which were evaluated and ranked through an external peer review process. Applications were submitted by clinicians and researchers at U of T and its affiliated hospitals from a wide range of disciplines including biochemistry, biomedical engineering, developmental and stem cell biology, immunology, neuroscience and surgery.

Medicine by Design builds on decades of made-in-Canada excellence in regenerative medicine dating back to the discovery of stem cells in the early 1960s by Toronto researchers James Till and Ernest McCulloch. Regenerative medicine uses stem cells to replace diseased tissues and organs, creating therapies in which cells are the biological product. It can also mean triggering stem cells that are already present in the human body to repair damaged tissues or to modulate immune responses. Increasingly, regenerative medicine researchers are using a stem cell lens to identify critical interactions or defects that prepare the ground for disease, paving the way for new approaches to preventing disease before it starts. Medicine by Design is made possible thanks in part to a $114-million grant from theCanada First Research Excellence Fund.

Current cystic fibrosis drug treatments target a genetic mutation that causes epithelial cells, which line the airway and act as a barrier against viruses, to function improperly. The mutation affects the function of an important ion channel in cells, called CFTR, which helps to maintain the right balance of fluid in the airways. Poor function causes mucosal obstructions in the airways and prevents clearance of foreign pathogens, which leads to chronic infections and ultimately destroys airway tissue.

In her project, Wong will explore an alternative ion channel in the epithelial cells to determine if it can be hijacked and used to compensate for the lack of function caused by the mutant CFTR. The research will be conducted using a combination of stem cell-derived lung models, gene editing and computational modelling.

Wongs project builds on decades of cystic fibrosis research at SickKids, where the cystic fibrosis gene was first identified 30 years ago.

To date, more than 2,000 mutations in the cystic fibrosis gene have been identified, says Wong. SickKids scientists and U of T researchers have become the epicentre of incredible cystic fibrosis research to understand how this disease works at the genetic and molecular level.

Wong says that, while the idea of targeting an alternative pathway is not necessarily ground-breaking on its own, its the array of tools now available that makes the idea a potential game changer.

We have access to an incredible resource of primary cells and stem cells from more than 100 individuals with cystic fibrosis harbouring various mutations. Wong says.Our lab has developed human lung models from stem cells that can be used to model lung disease such as cystic fibrosis. And with new advanced tools in single-cell genomics and gene-editing, coupled with key collaborations for computational modelling, we are poised to find new therapeutic targets for cystic fibrosis.

Leo Chou, an assistant professor at the Institute of Biomedical Engineering, andHyun Kate Lee, an assistant professor in the department of biochemistry in the Temerty Faculty of Medicineboth Medicine by Design New Investigators are also leading 2020 New Ideas projects.

Chou, along with co-investigatorsJulie Lefebvre, a scientist at SickKids and U of T assistant professor of molecular genetics, andValerie Wallace, a senior scientist at the Krembil Research Institute, University Health Network and a U of T professor of laboratory medicine and pathobiology and ophthalmology, will focus on cell transplantation in the retina, a process that has demonstrated encouraging pre-clinical results such as partial vision restoration in several animal disease models.

Recent research had demonstrated that this restoration is a result of the transfer of proteins complex molecules required for the structure, function and regulation of the bodys tissues between host tissue and donor cells. But the scope of that transfer process is not well understood. Chous project will develop an imaging approach to detect the transfer of mRNA molecules between host and donor cells. The outcomes from this project will inform the future design of cell transplantation therapies and lead to novel methods to deliver therapeutics. This project could improve therapies for retinal diseases and visual impairments, and inform strategies for other degenerative disorders.

Lee and co-investigatorPenney Gilbert,an associate professor at the Institute of Biomedical Engineering, will look at a common but not well-understood structure called the neuromuscular junction (NMJ), which mediates communication between neurons and muscles throughout the body. Defects in NMJ integrity and function underlie fatal diseases such as ALS. NMJ diseases, which affect more than 500,000 people globally, lack effective treatments. This project will use stem cells derived from reprogrammed skin cells of healthy people to develop NMJs in culture. Through high-resolution imaging, the healthy human NMJs will be studied both on their own and along with NMJs built from ALS patient cells. Through this work, the research team aims to identify genes to target to improve the health of NMJs, which could eventually help prevent or delay NMJ degeneration and even promote regeneration.

Michael Garton, an assistant professor at the Institute of Biomedical Engineering, has received a Seed Fund award to tackle the challenge of translating the genetic tools of synthetic biology an area of research that aims to create or redesign biological components using engineering methods into effective medical therapies against a number of diseases.

But they are difficult to translate into human therapies, Garton says, because the bodys T-cells immune cells that detect and destroy cells containing foreign material will identify these tools as foreign and destroy them.

Instead of switching off the T-cells, Gartons goal is to use computational modelling and high-throughput screening to selectively turn off the bodys foreign antigen display system so the immune system will still respond to foreign invaders when necessary, but allow cells containing synthetic tools to survive. If successful, this approach could enable a new generation of synthetic biology-enhanced cell therapies for a range of diseases.

Medicine by Design funding will help to facilitate the integration of synthetic biology and regenerative medicine and aid the development of cell-based therapies that perform better than nature, says Garton.

Other Seed Fund projects will encompass research in repairing the heart after paediatric cardiac surgery, treating an intestinal emergency in premature babies and creating a database for cell lineage paths.

John Parkinson, a senior scientist at SickKids and a U of T professor of biochemistry and molecular genetics, along with co-investigatorsJason Maynes, Wasser Chair in Anesthesia and Pain Medicine at SickKids and a U of Tassociate professor of anesthesiology and biochemistry, andWilliam Navarre, an associate professor in the department of molecular genetics, will investigate manipulating the microbiome, or community of microorganisms in the gut, to improve cardiac repair in post-operative treatment of a congenital heart disorder. Through a process that will identify prebiotics in breast milk that help enhance the production of molecules that research has shown can aid cardiac repair, the team will organize both observational (how disease alters the microbiome) and interventional (how the microbiome alters the disease) multi-site trials, which will provide the opportunity to immediately translate findings into changes in patient care regimens and improve outcomes.

CliniciansAgostino Pierro, a surgeon at the Division of General and Thoracic Surgery at SickKids and a U of T professor of surgery and physiology, and Philip Sherman, a senior scientist and gastroenterologist at the Division of Gastroenterology, Hepatology and Nutrition at SickKids and U of T professor of dentistry, pediatrics and laboratory medicine and pathobiology, have proposed a novel way of enhancing gut repair for a common intestinal emergency in premature babies, called necrotizing enterocolitis (NEC). A leading cause of death for these infants, NEC causes complications such as blindness, intellectual disability, repeat hospitalizations and gut damage even in those that survive. This project will look at whether intestinal organoids organ-like structures grown in the laboratory from stem cells that mimic some of the functions of native intestines can potentially stimulate repair of the gut and recovery from NEC. The project will define how to best transplant organoids, identify how the organoids protect the intestine from injury and assess if organoid transplantation is a valid new treatment for NEC.

Lincoln Stein, who is head of adaptive oncology at the Ontario Institute for Cancer Research and a professor in the department of molecular genetics at U of T, has received seed funding to build a database called Cytomics Reactome, which will be freely available to Canadian and international researchers. The database will build on recent technologies that open the door to the possibility of deciphering cell lineage paths the series of steps that lead a young, undifferentiated cell into a specialized one at single-cell resolution. To accelerate the path from basic research to clinical application, the database will systematically organize pre-existing knowledge of cell lineage paths into a comprehensive, interactive and easily accessible map that can serve as a framework for interpretation and integration of the latest experimental findings.

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U of T's Medicine by Design invests $1 million to advance new ideas in regenerative medicine - News@UofT

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October 10th, 2020

Discover The Augustinus Bader Wonder Cream Loved By The A List – Grazia

By daniellenierenberg

Two years ago I received an Augustinus Bader cream in a goody bag. I hadnt heard of it at the time, so paid little attention, although it looked nice enough a sleek, big blue bottle with rose gold accents. Shortly after, having just run out of my current moisturiser, I dug it out to give it a go. A few days later, forensically inspecting my skin in the mirror as I am wont to do in my more idle moments, I thought this stuff is actually really great. This was swiftly followed by another thought oh dear when I discovered it costs a whopping 205 a pop (for 50ml, you can get 15ml for 65). But it was too late, I was hooked. And that is how I became a woman who drops 205 on a moisturiser.

I am probably the least glamorous and definitely least well-known member of the Augustinus Bader fan club, which has swiftly reached legendary status in the beauty world and beyond. Kim Kardashian West, Naomi Campbell, Kate Bosworth, Priyanka Chopra and Diane Kruger all love the cult brand. Victoria Beckham invited Professor Bader to sit front row at her AW20 show; she also tapped his talents to collaborate with her on her debut skincare line (from seeing VB up close I can testify that this is a woman who knows good skincare, and the Cell Rejuvenating Power Serum is indeed excellent).

Professor Augustinus Bader and Victoria Beckham Getty

The latest addition to the Bader fan club is Emma Corrin. Prepping the new Diana for The Crowns Zoom press junket this week, her makeup artist Florrie White revealed she used The Cream and The Face Oil on the young actress. I use The Cream on everyone; me and my clients, White tells me. It instantly plumps and smooths the skin with three perfect pumps and creates a calm and luxurious base for every make-up look. Everyone always comments on how lovely it feels on their skin.

Today Augustinus Bader is a cult phenomenon but the founder is a man who seems to be more at home in a laboratory than on a red carpet. A leader in stem cell biology, and head of stem cell research at the University of Leipzig, Professor Bader has spent over 30 years focusing on how reawakening these cells can aid the healing process in particular embarking on a mission to help burns victims. In 2008, Bader formulated a groundbreaking wound gel that could help heal third-degree burns without the aid of surgery or skin grafts.

It was this breakthrough that led to the founding of the skincare line. After all, if it could have this effect on serious injuries, imagine what the technology could do for those of us lucky enough to have run-of-the-mill skin niggles? The brand launched with two hero products: The Cream and The Rich Cream which, according to the brand literature, contain patented TFC8 (Trigger Factor Complex) technology, which supports the skins natural processes, leaving all skin types mature, dry, oily, or sensitive looking restored, renewed and regenerated.

I was useless at science at school and all this technical talk is pure gobbledygook to me. What I can tell you is what its done for me. My skin is super sensitive, I have eczema, and have found that Augustinus Bader creams genuinely help to soothe it when it is aggravated. I have found that since using it, my complexion appears clearer, more even and the Holy Grail of beauty dewy (despite drinking gallons of water a day, I have never achieved that before). My mum, who observes me with the scrutiny that only mothers do, confirms that my skin looks great (I am privvy only to this information because she thought it was a result of finally listening to her by stopping smoking and making the hours before midnight count). Is it expensive? Well, yes, theres no way around that. However, in much the same way that I think that an impeccably cut designer jacket earns its value back in cost per wear, I have found that since using The Cream and The Rich Cream my skincare regime has simplified.

Sure, theres an element of hype involved. I still find it thrilling to be In The Know about something. But, for me and my skin, Augustinus Bader lives up to it. Believe me, I wish it wasnt true (I am not snobby with beauty products, and would happily wax lyrical about a 5 wonder find if Id discovered one), but thats the 205 price I have to pay and Id say its worth it.

READ MORE: 'I Just Love The Way A Polished Lipstick Completes A Look': Victoria Beckham's New Posh Lipsticks Explained In Her Own Words

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Discover The Augustinus Bader Wonder Cream Loved By The A List - Grazia

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October 10th, 2020

SGEN: The 3 Top Biotech Stocks That Hedge Fund Managers LOVE – StockNews.com

By daniellenierenberg

The biotech industry has been soaring this year, with the SPDR S&P Biotech ETF (XBI) up 26.6% year to date so far. The race for a COVID vaccine has primarily driven this performance, but what if there was another segment of the biotech industry that top hedge fund managers are all going after right now? While many managers are known to take risks, its certainly worth looking into if there is a consensus between them.

The AlphaClone Alternative Alpha ETF (ALFA) tracks an index of equity securities that hedge funds have significant exposure to. The ETFs top three holdings are all biotech firms working on cancer drugs. While many investors have their attention on biotech and pharmaceutical companies working on a COVID vaccine, big money has been focused on the next big thing in biotech, the future oncology drug boom.

Cancer is the second leading cause of death in the U.S. behind heart disease. Almost everyone knows someone that has been affected by one of the many vicious types of cancer. There are now numerous companies focused on ways to treat and cure the various forms. While COVID is at the forefront, cancer is a long-term play. There is even an ETF that covers the cancer industry, the Loncar Cancer Immunotherapy ETF (CNCR), which is up over 39% over the last six months.

As the oncology drug market is expected to reach $394 billion by 2027, here are the three top cancer stocks based on a consensus of hedge fund managers: Seattle Genetics (SGEN), Fate Therapeutics (FATE), and Blueprint Medicines (BPMC).

Seattle Genetics (SGEN)

SGEN is a biotech firm focused on developing antibody-drug conjugates. Its lead lymphoma drug, Adcetris, has been performing quite well since it launched, and it is the primary growth driver for the company. The drugs label was also expanded, providing more revenue for the company. SGEN has been collaborating with Takeda (OTCMKTS:TKPHF), a Japanese pharmaceutical company, for the global development and commercialization of Adcetris.

In addition to Adcetris, the company has a promising pipeline of drug candidates for its antibody-drug conjugate (ADC) technology. In December, the FDA granted accelerated approval to Padcev to treat patients with metastatic bladder cancer, who were previously treated with a checkpoint inhibitor and platinum-based chemotherapy. This drug was created in collaboration with Astellas Pharma (OTCMKTS:ALPMF), another pharmaceutical company.

In April, the FDA approved Tukysa for the treatment of metastatic HER2-positive breast cancer. Investors should also be happy with the news Merck (MRK) plans to buy a 2.9% stake in SGEN. The companies are co-developing and selling SGENs breast cancer therapy, ladiratuzumab vedotin.

The company is rated a Strong Buy in our POWR Ratings system, with a grade of A in Trade Grade, Buy & Hold Grade, and Peer Grade. Those are three out of the four components that make up the POWR Ratings. The stock is also ranked #2 out of 377 Biotech stocks.

Fate Therapeutics (FATE)

FATE is a clinical-stage biopharmaceutical company engaged in the development of programmed cellular immunotherapies for cancer and immune disorders. The company has been building up its pipeline of immuno-oncology product candidates. These treatments are designed to elicit an immune response in patients with cancer.

The companys progress with FT596 is encouraging. FT596 is cell cancer immunotherapy derived from its iPSC line. The induced pluripotent stem cell (iPSC) platform provides a competitive advantage for the company as iPSC cells are stem cells that can become almost any cell type. They are grown from the same cell instead of a patients donated cells. This means that one engineered cell line can be manufactured for many patients, creating what is known as off the shelf immunotherapy.

If the development of this type of therapy is successful, this would reduce the cost of manufacturing and provide a potential cash cow for the company. FATE has entered into collaborations with other companies for fund and research expertise. It is currently working with Ono Pharmaceutical (OTCMKTS:OPHLY) for two off-the-shelf iPSC-derived CAR T-cell product candidates, and Janssen Biotech develop iPSC-derived CAR NK and CAR T-cell product candidates.

FATE is rated a Strong Buy in our POWR Ratings system. It holds a grade of A in Trade Grade, Buy & Hold Grade, and Peer Grade. It is also ranked #24 out of 377 stocks in the Biotech industry. The stock is up a whopping 145.3% after finishing the day up 6.7%.

Blueprint Medicines (BPMC)

BPMC is a biopharmaceutical company focused on improving patients lives with diseases driven by abnormal kinase activation. The company has developed a small molecule drug pipeline in cancer. The firms lead product, Ayvakit, was approved by the FDA in January to treat metastatic gastrointestinal stromal tumor. The drug generated $5.7 million in the second quarter, so its off to a good start.

The company is also looking to expand its label as it is being studied for advanced and smoldering forms of systemic mastocytosis, a condition where certain immune cells, called mast cells, build up under the skin and, or in the bones, intestines, and other organs. If approved for other labels, that should help drive further growth.

Last month, the FDA approved the companys second drug, Gavreto, for the treatment of RET fusion-positive NSCLC or non-small lung cancer. BPMC worked on the drug with Roche (OTCMKTS:RHHBY). Lung cancer is responsible for more cancer deaths than any other in men and women. If Gavreto can become a standard treatment, it could become a goldmine for the company. The drug can also treat medullary thyroid cancers.

BPMC is rated a Strong Buy in our POWR Ratings system. It has grades of A in Trade Grade, Buy & Hold Grade, and Peer Grade. It is also the #9 ranked stock in the Biotech industry. The stock is up over 27% for the past three months and 8.5% over the past week.

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SGEN shares were unchanged in after-hours trading Friday. Year-to-date, SGEN has gained 75.72%, versus a 9.31% rise in the benchmark S&P 500 index during the same period.

David Cohne has 20 years of experience as an investment analyst and writer. Prior to StockNews, David spent eleven years as a Consultant providing outsourced investment research and content to financial services companies, hedge funds, and online publications. David enjoys researching and writing about stocks and the markets. He takes a fundamental quantitative approach in evaluating stocks for readers. More...

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October 10th, 2020

Gray Hair: What You Need to Know about Causes and Possible Prevention – Coveteur

By daniellenierenberg

Find out what the pros had to say.

You never forget your first. Grey hair, that is. Maybe you pluck it? Heck, its just oneout of sight, out of mind, right? Yeah, we know, you pluck one and five more grow back in its place, and then those five turn into tenyou get the idea. Nothing that a good dye job cant fix. That is, until a pandemic forces you into quarantine with no access to your colorist, and it becomes brutally apparent just how grey your hair actually is these days. This standoff between my stealthy foe and I got me thinking: We know we cant reverse grey hair, but can we slow its progression or delay its initial onset? I called on the pros to find out, but first, some basic hair biology.

A protein called melanin is responsible for the color of our hair, and the cells in the hair follicles that make melanin are called melanocytes. Melanocytes are made by stem cells in the root of the hair, says Dr. Morgan Rabach, board-certified dermatologist, assistant professor at Mount Sinai School of Medicine, and co-founder of LM Medical NYC, and over time the stem cells die out and stop making melanocytes, leaving us with no cells to color the hair.

The age at which we start to go grey varies and depends on many factors. Dr. Caroline Robinson, MD, FAAD, and dermatologist and founder of Tone Dermatology, believes there is likely a genetic tendency at play in most cases of greying hair, but, like many genetic tendencies, there are environmental factors that influence how these changes show up in each of us. Dr. Rabach concurs, adding that grey hair is a combination of genetics and lifestyle.

Premature greying is generally considered less than 20 years old and it is thought to be an inherited predisposition. However, premature greying can also be attributed to certain illnesses and deficiencies. Dr. Robinson notes the importance of annual physicals and doctor visits when it comes to premature greying, as it could be an early sign of metabolic abnormalities in a select population.

Search results for grey hair yield a slew of information centered around the notion that going grey is a result of an accumulation of hydrogen peroxide in the follicle, and that it could be eradicated by a topical pseudocatalase cream. This claim seems to have originated from a segment of research on vitiligo, which then appeared to have been manipulated for headlines. Dr. Robinson weighed in with her thoughts on the claim, saying, There are no double-blinded, controlled clinical studies to support the use of topical pseudocatalase-based creams for loss of pigmentation in hair.

Similarly, some products have appeared on the market claiming to re-pigment the hair, but the consensus among experts is that the claims are unfounded. I havent seen any convincing evidence for products that claim to reverse or prevent greying of the hair, says Holden.

We know all too well the havoc that stress wreaks on our overall health, but the type of stress were referring to here is oxidative stressthe imbalance of free radicals and antioxidants in the body, which leads to cell and tissue damage. In oxidative stress, free radicals damage our cells and tissues when our body doesnt have enough antioxidants to combat them, and in our hair, this oxidative stress can damage the cells that produce melanin, says Kate Holden, consultant trichologist.

The pros agree that oxidative stress plays a key role in the loss of hair pigmentation. From recent research we know that oxidative stress, the same type of stress that our skin faces in response to UV rays and pollution, can be an important factor in the loss of hair pigmentation, says Dr. Robinson. While oxidative stress occurs naturally in our bodies, environmental factors can increase its effects, such as alcohol, smoking, sugar and processed foods, cortisol levels, etc.

In addition, a recent study conducted by a group of Harvard researchers looked at the impact of stress (like, say, the kind you feel during a worldwide pandemic) on pigment-producing cells and found that the hyper-activation of the sympathetic nerves caused the depletion of melanocyte stem cells. Trichologist Leata A. Williams explains, When we are under stress, our bodies signal the fight-or-flight response, and it is the nerves that send the response to our hair follicles, causing the hair to grey.

Just think of how many world leaders have gone grey while in officethats the sympathetic nervous system depleting their hair follicles of melanocytes. And although most of us will never feel the stress of running a country, were still susceptible to the same greying effect from our everyday lives.

Unfortunately for us, the pros agree that there is no real evidence to suggest that we can turn back the clock and re-pigment or slow the greying of hair in any tangible way. What we can do, however, is engage in healthy lifestyle behaviors that promote our overall health, and increase our intake of antioxidants. Increasing antioxidant intake through foods in our diet (leafy veggies, fruit) has not directly been shown to impact greying hair, but it can help to decrease overall oxidative stress levels in the body, says Dr. Robinson, which is something she discusses with her patients.

Dr. Rabach agrees and adds that she believes that a stress-free and healthy lifestyle makes your whole body healthier, and this would be reflected in the youthfulness of your hair. While we may not be able to reverse or halt grey hairs from popping up, what is encouraging is Dr. Rabachs belief that through good hair health, nutritious diet, and stress management, we might be able to delay their initial appearance. Increasing your antioxidant intake, whether it be from your diet, a multivitamin, or even a product formulated for hair health, will be beneficial in reducing oxidative stress and may help delay the initial onset of grey hair, says Dr. Rabach, who also encourages the use of hair products with antioxidants.

According to Dr. Robinson, there are some reports that Platelet Rich Plasma (PRP) therapy an in-office procedure that involves scalp injection of a processed form of ones own bloodcan promote hair re-pigmentation or slow greying because of its ability to deliver growth factors to the the hair follicle. While it sounds promising, more data needs to be gathered to determine its efficacy as a hair treatment.

Bottom line: Dont waste your money on products that claim to turn back the clock on grey hair, and focus instead on increasing your antioxidant intake and mitigating the effects of oxidative stress. Here are a couple products to get you started.

$17

Dr. Rabach promotes the use of antioxidant-rich hair products to help protect against the effects of oxidative stress. Look for one that is formulated with powerful superfruits like goji, acai, and pomegranate, and includes biotin for the added hair-nurturing benefits.

Buy

$65

This ingestible powder is chock-full of antioxidant-rich superfoods, adaptogens, and a probiotic blend to help your body resist stress, support detoxification, and support immunity. Add a teaspoon to water or a cold beverage of your choice, and reap the benefits of this all-natural, whole-food blend and help fend off the effects of oxidative stress.

Buy

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Gray Hair: What You Need to Know about Causes and Possible Prevention - Coveteur

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October 9th, 2020

Proposition 14: With Just Handful of Cures, California Stem Cell Agency’s Fate Is In Hands of Voters – KQED

By daniellenierenberg

A Yes vote authorizes the state to sell $5.5 billion in general obligation bonds primarily for stem cell research and the development of new medical treatments in California. A No vote would mean the state's stem cell research agency will probably shut down by 2023.

In the ramp-up to the 2004 election, a California TV viewer may have come across the popular actor Michael J. Fox urging her to vote Yes on a state proposition. His voice slurred faintly by Parkinsons disease, he still sounded wry, boyish and familiar.

My most important role lately is as an advocate for patients and for finding new cures for diseases, said Fox, eyes level with the camera. Californias Stem Cell Research Initiative 71 will support research to find cures for diseases that affect millions of people, including cancer, diabetes, Alzheimer's and Parkinson's.

Within that 30-second spot, Fox, diagnosed at age 29 with a neurodegenerative disorder that typically does not strike until after 60, used the word "cures" three times.

Proposition 71, which passed with 59% of the vote, authorized the sale of $3 billion in bonds to create an agency that funded stem cell research. The successful campaign grew out of a time, in the early 2000s, when the promise of stem cell and regenerative medicine excited both scientists and the public.

Whether the project has lived up to that promise is a matter of opinion. How voters view the record of the agency may go a long way in their decision whether or not to replenish the fund, which is fast running out of money, with an additional $5.5 billion to be raised with new bonds authorized by Proposition 14, now on the ballot.

President Bush A Demon to Attack

Scientists since the1800s have known about stem cells, which are not yet dedicated to any particular anatomical function and have the potential to become nerve cells, blood cells, skin cells or any other type. They are found in blastocysts, which are human embryos four to five days after fertilization, and in a few areas, such as bone marrow and gonads, in adults.

In the late 1990s, researchers developed ways to steer the development of these cells, and the possibilities for improving medicine seemed endless. If malfunctioning cells were at the root of a particular disease, could new healthy cells tailored to the job fix what was wrong? Scientists and many members of the public were eager to find out.

Anti-abortion groups, however, a key constituency of President George W. Bush, opposed the research, and in 2001 he limited federal funding to a few existing lines of embryonic stem cells, severely curtailing research.

Some in the state of California wanted to get around Bushs restrictions, and Proposition 71 was born.

"(T)hey had this demon they could attack in the campaign the Bush administration," said David Jensen, author of "California's Great Stem Cell Experiment," who also writes the blog California Stem Cell Report. "They could say, 'This is a great opportunity, and the only way we're going to get it done is to do it here in California.'"

The measure created the California Institute for Regenerative Medicine. The stem cell research agency is unique in the U.S.

"No other state has done this kind of level of funding and focus on this kind of thing, said Jensen. It's a really cutting-edge area of science."

A Few Successes

The pace of innovation has been slower than many hoped. As it turned out, grand discoveries were not around the corner, and to date there is no widespread stem cell treatment approved for the public. To date, CIRM has funded more than 64 trials directly and aided in 31 more. Not all have or will result in treatments.

But despite the lack of a marquee cure like one for Alzheimers or Parkinsons, the agency has seen some notable triumphs.

"Probably one of the most spectacular successes they have certainly so far," said Jensen, "is clinical trials that have saved the lives of what they say are 40 children."

Those children were born with severe combined immunodeficiency (SCID), commonly known as "bubble baby syndrome," a rare, generally fatal condition in which a child is born without a working immune system. An FDA-approved gene therapy that grew out of CIRM-funded research can now cure the disease by taking a patients own blood stem cells and modifying them to correct the SCID mutation. The altered cells generate new, healthy blood cells and repair the immune system.

The FDA has also approved two drugs for rare blood cancers that were developed with CIRM funds.

Sandra Dillon, a graphic designer in San Diego, credits one of the drugs with saving her life. She was diagnosed when she was just 28, in 2006. Her doctors told her they would try to manage her symptoms, but that she was going to get progressively sicker.

"Even just the idea of a cure or getting better wasn't even on the table back then," said Dillon, who is featured in ads for the Yes on 14 campaign.

"I remember just praying and begging into the universe, please, someone just look at my disease, please someone help, who is going to look at this thing.

By 2010, Dillon was extremely ill. She connected with a doctor at UC San Diego who received early-stage funding from CIRM and told her she could take part in clinical trials.

"For the first time, there was this moment of, 'Oh, my gosh! There are researchers doing something. And it could help me and I can get access to it.' It was amazing."

The drug received FDA approval in 2019, and today Dillons cancer has retreated to the point where she can live a normal life.

"I love that I am not tethered to a hospital anymore. I can go out on long backpacking trips and hiking and surfing," she said. "I am a completely different person with this drug. And I have a whole future ahead of me."

The original funding raised by Proposition 71 is running out. Proposition 14 would authorize the sale of a new bond to refill the agency piggy bank. Gov. Gavin Newsom, the UC Board of Regents, and scores of patient advocacy groups also support the measure.

Many newspaper editorial boards, however, oppose the proposition, including the San Francisco Chronicle, Mercury News and Los Angeles Times.

Right now the state still owes about $1 billion toward the debt created by Proposition 71. If Proposition 14 passes, the yearly price tag to pay off the new bond would be about $260 million per year for about 30 years.

One of the selling points of the original proposition was the potential for the state to earn big money in royalties from the treatments it helped develop, says Jeff Sheehy, an HIV patient advocate and the only CIRM board member to oppose Proposition 14.

"The promises were made that this would pay for itself. We would be able to pay back the bonds with the money we would get from royalties, etc., etc.

That has not worked out as envisioned: CIRM estimates it has received less than $500,000 in royalties. Early this year, Forty Seven, a company whose therapies were heavily funded by CIRM, sold to Gilead for $4.9 billion. While millions went to various researchers, neither CIRM nor the state of California received anything.

One of the flaws in the original measure is that we [the agency] cannot hold stock in the products that we develop," says Sheehy. "And that's because the California Constitution says that the state of California cannot, as a government entity, hold equity.

Proposition 14 makes it impossible for the state to use profits from its investment on, say, schools or other funding priorities. Instead, any royalties earned must be fed back into programs to make CIRM-funded treatments more affordable.

"What it does is it basically takes all of our returns that we get from this and gives it back to the pharmaceutical and biotech companies," said Sheehy. "It becomes just a blatant giveaway to these companies when we should be requiring access and requiring fair pricing."

Sheehy says he supports medical research, but doesn't like the state going into more debt to pay for it. The greater the state's obligations in bond money, which has to be paid back with interest, the less there is in the general fund, and Sheehy says the state has more pressing needs than stem cell research things like housing, education and transportation.

"The biggest and perhaps the most compelling reason why I feel so strongly that this is not a good idea is that we simply cannot afford it, he said. "If we think this is so important," asks Sheehy, "why don't we just don't pay for [this research] out of the general fund? It would be cheaper.

Opponents of Proposition 14 also point to longstanding complaints of conflicts of interest among the agency board. Most of the $3 billion distributed by the agency has gone to institutions with connections to board members. Critics say the structural conflicts of interest between the board and agency are not addressed in the new measure. Proposition 14 would balloon an already huge board of 29 members to 35.

Funding needs for stem cell research also are not as acute as they were back in 2004. The federal National Institutes of Health now funds some basic stem cell research, spending about $2 billion a year, with $321 million of that going toward human embryonic stem cell research. And private ventures, like nonprofits started by tech billionaires, are pouring more money into biotech.

The problem with assuming that, says Melissa King, executive director of Americans for Cures, the stem cell advocacy group behind the Yes on 14 campaign, is that CIRM fills a neglected funding need.

The NIH does not fund clinical trials at nearly the rate that CIRM can and has been, King said.

She says that's important because of what she calls the "Valley of Death," where promising early-stage research frequently fails to translate into promising treatments that can be tested in clinical-stage research. (What works well in a test tube often does not work well in an organism.) This weeding-out process is costly but necessary. And its where CIRM focused a lot of its effort.

The first- and maybe even second-phase clinical trials, its very difficult to get those funded, King said. It is too much of a risk for business to take on on its own. Venture [capital] isnt going there. Angel [funding] isnt going there.

What voters have to ask themselves, says writer Jensen, is whether stem cell funding is "a high priority for the state of California? Different people make different judgments about that."

CIRM supporters say if Prop. 14 doesn't pass, critical research will stall. Others say federal and private funding will step in and fill the gap.

Absent new funding, the institute expects it will wind down operations leading to a complete sundown in 2023.

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