WHIPPANY, N.J., Oct. 21, 2019 /PRNewswire/ -- Bayer today announced recipients of the inaugural Pulmonary Hypertension Accelerated Bayer (PHAB) Awards, a U.S.-based research grant program created to support clinical research in pulmonary hypertension (PH), with a focus on pulmonary arterial hypertension (PAH) and chronic thromboembolic pulmonary hypertension (CTEPH). The recipients will receive a combined total of $1 million in grants over a two-year period, making the PHAB Awards one of the largest industry-funded grant programs focused on PAH and CTEPH in the U.S. The eight award recipients were formally announced at a ceremony during the American College of Chest Physicians (CHEST) Annual Meeting in New Orleans on Sunday, October 20, 2019.

"Supporting a new generation of researchers is imperative to ensure we continue the progress that has been made during the past decade in pulmonary hypertension and its related conditions," said Aleksandra Vlajnic, M.D., Senior Vice President & Head Medical Affairs Americas at Bayer. "Our hope is that the PHAB Awards program will encourage researchers to think creatively about solving the significant treatment and patient care challenges that remain, knowing Bayer is committed to providing the support needed to help bring those ideas to fruition. We want to congratulate all of the applicants on their winning proposals."

The recipients are:

The PHAB Awards recipients were selected by an independent Grants Review Committee, consisting of the following eminent PH leaders:

"I would like to thank and recognize the Grants Review Committee for their time and commitment, and the PH community in the U.S. for their overwhelming response to the inaugural PHAB Awards," said Sameer Bansilal, M.D., M.S., Medical Director, U.S. Medical Affairs at Bayer. "We look forward to an even greater response next year and encourage eligible applicants to start thinking about submitting their research proposals."

The PHAB Award eligibility, review and category criteria were modeled after the National Institutes of Health (NIH) system; entries were graded on significance, investigator(s), innovation, approach, and environment.

For more information on the PHAB Awards visit: https://www.phab-awards.com/awards/or e-mail PHAB.awards@bayer.com.

Grants were made on the merits of the research, and research must be posted on ClinicalTrials.gov. Every effort should be made to publish or present study outcomes. If the research is not conducted the grant must be returned.

About Pulmonary Arterial Hypertension (PAH)Pulmonary Arterial Hypertension (PAH, WHO Group 1) is defined by elevated pressure in the arteries going from the right side of the heart to the lungs. Typical symptoms of PAH include shortness of breath on exertion, fatigue, weakness, chest pain and syncope. PAH is caused by abnormalities in the walls of the pulmonary arteries.1,2

About Chronic Thromboembolic Pulmonary Hypertension (CTEPH)Chronic Thromboembolic Pulmonary Hypertension (CTEPH, WHO Group 4) is a progressive type of pulmonary hypertension, in which it is believed that thromboembolic occlusion (organized blood clots) of pulmonary vessels gradually lead to an increased blood pressure in the pulmonary arteries, resulting in an overload of the right heart.3,4 CTEPH may evolve after prior episodes of acute pulmonary embolism, but the pathogenesis is not yet completely understood. The standard and potentially curative treatment for CTEPH is pulmonary thromboendarterectomy (PTE), a surgical procedure in which the blood vessels of the lungs are cleared of clot and scar material.5,6 However, a considerable number of patients with CTEPH (20%-40%) are not operable and in up to 35 percent of patients, the disease persists or reoccurs after PTE.7

About BayerBayer is a global enterprise with core competencies in the life science fields of health care and nutrition. Its products and services are designed to benefit people by supporting efforts to overcome the major challenges presented by a growing and aging global population. At the same time, the Group aims to increase its earning power and create value through innovation and growth. Bayer is committed to the principles of sustainable development, and the Bayer brand stands for trust, reliability and quality throughout the world. In fiscal 2018, the Group employed around 117,000 people and had sales of 39.6 billion euros. Capital expenditures amounted to 2.6 billion euros, R&D expenses to 5.2 billion euros. For more information, go to http://www.bayer.us.

Our online press service is just a click away: http://www.bayer.us/en/newsroomFollow us on Facebook: http://www.facebook.com/pharma.bayer Follow us on Twitter: https://twitter.com/Bayerus

Media Contact:David Patti, +1-973-452-6793Bayer, U.S. Product Communications david.patti@bayer.com

Forward-Looking StatementsThis release may contain forward-looking statements based on current assumptions and forecasts made by Bayer management. Various known and unknown risks, uncertainties and other factors could lead to material differences between the actual future results, financial situation, development or performance of the company and the estimates given here. These factors include those discussed in Bayer's public reports which are available on the Bayer website at http://www.bayer.com. The company assumes no liability whatsoever to update these forward-looking statements or to conform them to future events or developments.

References:1Galie et al. 2015 ESC/ERS Guidelines for the diagnosis and treatment of pulmonary hypertension. Eur Heart. 2016;37:67119.2American Lung Association. Pulmonary Hypertension. Accessed November 22, 2017. http://www.lung.org/lung-health-and-diseases/lung-disease-lookup/pulmonary-hypertension.3Piazza G and Goldhaber SZ. Chronic thromboembolic pulmonary hypertension. N Engl J Med. 2011; 364: 351-360.4 Simonneau G et al. Updated Clinical Classification of Pulmonary Hypertension. Journal of the American College of Cardiology. 2013; 62(25):5 D'Armini M. Diagnostic advances and opportunities in chronic thromboembolic pulmonary hypertension. Eur Respir Rev. 2015; 24: 253262.6 Kim et al. Chronic thromboembolic pulmonary hypertension. J Am Coll Cardiol. 2013; 62: D92-9.7 Mathai et al. Quality of life in patients with chronic thromboembolic pulmonary hypertension. Eur Respir J. 2016 Aug; 48(2): 526537.

SOURCE Bayer

http://www.bayer.us

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Bayer Announces Recipients of the Pulmonary Hypertension Accelerated Bayer (PHAB) Awards at CHEST Annual Meeting 2019 - PRNewswire

Cardiac Stem Cells Comments Off on Bayer Announces Recipients of the Pulmonary Hypertension Accelerated Bayer (PHAB) Awards at CHEST Annual Meeting 2019 – PRNewswire | October 22nd, 2019

CALGARY, Alberta, Oct. 21, 2019 (GLOBE NEWSWIRE) -- Hemostemix Inc. (Hemostemix or the Company) (TSX VENTURE: HEM; OTCQB: HMTXF), a biotechnology company developing and commercializing blood-derived stem cell therapies for unmet medical conditions, is pleased to provide a summary of the presentation entitled Autologous Stem Cell Treatment for CLI Patients with No Revascularization Options: An Update of the Hemostemix ACP-01 Trial With 4.5 Year Followup. Lead investigator Dr. York Hsiang, Professor of Vascular Surgery, University of British Columbia gave this update at the 41st Annual Canadian Society for Vascular Surgery Meeting on September 14, 2019.

Dr. Hsiang reported on the blinded results from the long-term follow-up of the first cohort of patients enrolled at two trial sites, Vancouver Coastal Health Research Institute (VCHRI) located in Vancouver, BC, led by principal investigator, Dr. York N. Hsiang, MB, ChB, MHSc, FRCSC and University Health Network, Peter Monk Cardiac Centre located in Toronto, Ontario, led by principal investigator Dr. Thomas Lindsay, MDCM, MSc, FRCSC, FACS.

Following is a summary of the results and conclusion:

In addition, the Companys Data Safety Monitoring Board (DSMB) recently met to review patient safety data in the ongoing Phase II clinical trial for CLI. The DSMB did not find safety concerns with ACP-01 and recommended continuing to enroll patients in the trial. The clinical trial is ongoing at 13 clinical sites in the US and Canada, with several additional sites in the process of being initiated. To date, 46 of the planned 95 patients have been enrolled and treated in the study.

We are very pleased with these blinded long term follow up results, and the recommendation of the DSMB, which are consistent with the findings reported in our two previous published studies of ACP-01 in CLI patients, said Dr. Alan Jacobs, President and Chief Medical Officer of Hemostemix. Patients with critical limb ischemia face a high rate of amputation when revascularization treatment options are exhausted, so seeing this level of improvement, and outcomes maintained for up to 4.5 years after treatment, is extremely encouraging.

ABOUT HEMOSTEMIX INC.

Hemostemix is a publicly traded clinical-stage biotechnology company that develops and commercializes innovative blood-derived cell therapies for medical conditions not adequately addressed by current treatments. It is one of the first clinical-stage biotech companies to test a stem-cell therapy in an international, multicenter, Phase II clinical trial for patients with critical limb ischemia (CLI), a severe form of peripheral artery disease (PAD) caused by reduced blood flow to the legs. The Phase II trial targets a participants diseased tissue with proprietary cells grown from his or her blood that can support the formation of new blood vessels. The Companys intellectual property portfolio includes over 50 patents issued or pending throughout the world. Hemostemix has a manufacturing contract with Aspire Health Science, LLC (Aspire), for the production of ACP-01 and for research and development purposes at Aspires Orlando, Florida, facility. Building towards commercialization, Hemostemix has also licensed the use, sale and import of ACP-01 for certain indications to Aspire in certain jurisdictions. The Company is continuing research and development of its lead product, ACP-01 with other applications, including cardiovascular, neurological and vascular indications.

For more information, please visit http://www.hemostemix.comor email office@hemostemix.com.

Contact:

Kyle Makofka, CEOSuite 2150, 300 5th Avenue S.W.Calgary, Alberta T2P 3C4Phone: (403) 506-3373E-Mail: kmakofka@hemostemix.com

Neither the TSX Venture Exchange nor its Regulation Service Provider (as that term is defined under the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Forward-Looking Statements

This release may contain forward-looking statements. Forward-looking statements are statements that are not historical facts and are generally, but not always, identified by the words expects, plans, anticipates, believes, intends, estimates, projects, potential, and similar expressions, or that events or conditions will, would, may, could, or should occur. Although Hemostemix believes the expectations expressed in such forward-looking statements are based on reasonable assumptions, such statements are not guarantees of future performance and actual results may differ materially from those in forward-looking statements. Forward-looking statements are based on the beliefs, estimates, and opinions of Hemostemix management on the date such statements were made. By their nature forward-looking statements are subject to known and unknown risks, uncertainties, and other factors which may cause actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements. Such factors include, but are not limited to, the Companys stage of development, future clinical trial results, long-term capital requirements and future ability to fund operations, future developments in the Companys markets and the markets in which it expects to compete, risks associated with its strategic alliances and the impact of entering new markets on the Companys operations. Each factor should be considered carefully and readers are cautioned not to place undue reliance on such forward-looking statements. Hemostemix expressly disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events, or otherwise.

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Hemostemix Announces Positive Results and Conclusions Reported in Phase II CLI Trial Abstract - Yahoo Finance

Cardiac Stem Cells Comments Off on Hemostemix Announces Positive Results and Conclusions Reported in Phase II CLI Trial Abstract – Yahoo Finance | October 22nd, 2019

In a first, 26-yr-old DMD patient in UP survives with stem cell therapy

Lucknow, Oct 22 (IANS) Duchenne Muscular Dystrophy (DMD) is a deadly genetic disorder, 99.9 per cent people suffering from which, die between the age of 13 to 23 years. However, in a first, a 26-year-old patient from Lucknow has survived DMD by regularly taking stem cells for the last five years.

Children, suffering from DMD, usually die of cardio-respiratory failure. But with the stem cell therapy, this patient has not lost muscle power in last five years and heart and lung muscles and the upper half of the body are working well.

Dr. B.S Rajput, the surgeon who is treating this patient, said, "DMD is a type of muscular dystrophy and being a genetic disorder, it is very difficult to treat. Autologous (from your own body) bone marrow cell transplant or stem cell therapy in such cases was started in Mumbai about 10 years back.

Dr Rajput, who was recently appointed as visiting professor at GSVM Medical College, Kanpur, said he has treated several hundred DMD patients and recently this combination protocol was published in the international Journal of Embryology and stem cell research.

The patient''s father is elated that his son has maintained well with this treatment and now has even started earning by working on computers.

According to Dr Rajput, this disease is endemic in eastern UP, especially Azamgarh, Jaunpur, Ballia and some of the adjoining districts of Bihar, and one out of every 3,500 male child, suffers from the disease.

Yet the disease is not given as much attention as it should be.

Dr Rajput, who is consultant bone cancer and stem cell transplant surgeon from Mumbai, said though patients in Uttar Pradesh and Bihar get financial support from the Chief Minister''s Relief Funds, the treatment of autologous bone marrow cell transplant is not included in the package list of Ayushman Bharat scheme, which deprives many from getting the treatment.

The doctor further informed that efforts are being made to establish the department of regenerative medicine in the medical college, where bone marrow cell transplant and stem cell therapy would be done even for other intractable problems like spinal cord injury, arthritis knee and motor neurone disease.

--IANS

amita/rtp

Disclaimer :- This story has not been edited by Outlook staff and is auto-generated from news agency feeds. Source: IANS

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In a first, 26-yr-old DMD patient in UP survives with stem cell therapy - Outlook India

Spinal Cord Stem Cells Comments Off on In a first, 26-yr-old DMD patient in UP survives with stem cell therapy – Outlook India | October 22nd, 2019

NewsReal LifeLydia Burgess-Gamble has helped a sick woman in her twenties after they were genetically matched

Tuesday, 22nd October 2019, 10:38 am

Lydia Burgess-Gamble felt a lump in her throat when she got the letter saying her stem cells were a match for someone sick.

The 42-year-old had signed up to the register to be a donor almost three years earlier and hadn't given it much thought since.

Ahead of her 40th birthday, she'd wanted to do something altrusistic. Now she had the chance to potentially save someone's life who was battling a blood cancer or blood disorder.

Donating stem cells today is almost as easy as giving blood. "It was a straightforward and painless process and being able to relax and read a book for a few hours was a luxury," she said.

Patients face difficult odds

Every 20 minutes someone in the UK is diagnosed with a blood cancer, such as leukaemia, myeloma or lymphoma. That's more than 30,000 people a year. Worldwide, it's one every 30 seconds.

Patients suffering with these types of cancers can have their bone marrow damaged by the cancer itself, or from chemotherapy and radiation treatments. A stem cell transplant lets the new stem cells take over from the damaged marrow so the body can produce healthy, cancer-free blood cells.

Even though there are over 27 million people on the worldwide register, this isnt enough, according to charity DKMS. At any one time there are around 2,000 people in the UK in need of a transplant.

Matching donors and patients isnt easy because it's determined by tissue type, not blood group. There are thousands of different human leukocyte antigen (HLA) characteristics, in millions of combinations. Doctors look to relatives for a match but two out of three of those in need are unable to find one, and so must rely on the generosity of strangers.

Most donations are day cases at hospital

Lydia, an environmental research scientist from Brighton, became aware of the process involved through a Facebook post. "A friend shared an appeal for a loved one who needed a donor," she said. "I remember watching a documentary about donating bone marrow in the 90s and I hadn't realised it mainly doesn't involve an invasive procedure until I read this post."

The donation usually involves a nonsurgical procedure called peripheral blood stem cell (PBSC) donation for around 90 per cent of all cases, which is the method Lydia used.

With this method, blood is taken from one of the donors arms and a machine extracts the blood stem cells from it. The donors blood is then returned to their body through their other arm. It is an outpatient procedure that usually takes four to six hours.

'I had no side effects, other than I felt a little more tired than usual the next day'

Lydia Burgess-Gamble

This procedure doesn't "deplete" a donor's supply of stem cells, as a donor's stem cells will completely replenish themselves within two to four weeks afterwards.

"I had no side effects, other than I felt a little more tired than usual the next day but within 24 hours I was completely back to normal," said Lydia.

"All I know about my recipient is that it's a woman in her twenties who lives in Turkey. I'd love to make contact one day. I'm not expecting anything but I'm hoping she gets well and we may be able to meet."

The other 10 per cent of donations are made through bone marrow, where donors give cells from the bone marrow in their pelvis. This is under general anaesthetic so that no pain is experienced. The collection itself takes one to two hours and most donors return to their regular activities within a week. Two weeks after donation, your bone marrow will have recovered fully, and the hip bone will have fully healed within six weeks.

Donating: the process

To become a potential blood stem cell donor first check your eligibility on the DKMS website and request a swab kit for your cheek.

Complete the swabs posted to you at home and send them back. Then yourtissue type will be analysed and your details will be added to the UK stem cell registry. Your details can be searched for a genetic match for people all over the world who need a second chance at life.

The odds are you may never be called upon, but if you are, you will have a blood test at your local GP or hospital and will be asked to complete a medical questionnaire and consent form. If you're deemed fit and healthy enough, you'll have a further medical assessment and consultation at a specialist collection centre (where you will later donate your blood stem cells).

It's important to read about the methods used to collect blood stem cells PBSC and bone marrow donation because if youre on the register, you should be willing to donate in either way. The method will be determined by what the doctors believe will be best for the patient. However, you will of course always have the final decision on whether you are happy to proceed.

When a donor is matched with a patient, DKMS will cover the costs (including any travel, meals, or accommodation expenses that may be necessary and lost wages if you are not covered by your employer).

Your blood stem cells will never be stored, they last for around 72 hours and are delivered straight to the person in need by a special courier.

You will be allowed to meet the patient, if they consent, eventually UK guidelines state this can happen two years after the donation (and tules vary by country).Contact through anonymous letters can be established before this time via DKMS.

You will stay on the register until your 61st birthday.

Link:
'I've potentially saved a stranger's life by donating my blood stem cells and it was painless' - inews

Bone Marrow Stem Cells Comments Off on ‘I’ve potentially saved a stranger’s life by donating my blood stem cells and it was painless’ – inews | October 22nd, 2019

Lucknow: Duchenne Muscular Dystrophy (DMD) is a deadly genetic disorder, 99.9 per cent people suffering from which, die between the age of 13 to 23 years. However, in a first, a 26-year-old patient from Lucknow has survived DMD by regularly taking stem cells for the last five years.

Children, suffering from DMD, usually die of cardio-respiratory failure. But with the stem cell therapy, this patient has not lost muscle power in last five years and heart and lung muscles and the upper half of the body are working well.

Dr. B.S Rajput, the surgeon who is treating this patient, said, "DMD is a type of muscular dystrophy and being a genetic disorder, it is very difficult to treat. Autologous (from your own body) bone marrow cell transplant or stem cell therapy in such cases was started in Mumbai about 10 years back.

Dr Rajput, who was recently appointed as visiting professor at GSVM Medical College, Kanpur, said he has treated several hundred DMD patients and recently this combination protocol was published in the international Journal of Embryology and stem cell research.

According to Dr Rajput, this disease is endemic in eastern UP, especially Azamgarh, Jaunpur, Ballia and some of the adjoining districts of Bihar, and one out of every 3,500 male child, suffers from the disease.

Yet the disease is not given as much attention as it should be.

Dr Rajput, who is consultant bone cancer and stem cell transplant surgeon from Mumbai, said though patients in Uttar Pradesh and Bihar get financial support from the Chief Minister's Relief Funds, the treatment of autologous bone marrow cell transplant is not included in the package list of Ayushman Bharat scheme, which deprives many from getting the treatment.

The doctor further informed that efforts are being made to establish the department of regenerative medicine in the medical college, where bone marrow cell transplant and stem cell therapy would be done even for other intractable problems like spinal cord injury, arthritis knee and motor neurone disease.

Original post:
Lucknow: In a first, 26-yr-old DMD patient in UP survives with stem cell therapy - ETHealthworld.com

Bone Marrow Stem Cells Comments Off on Lucknow: In a first, 26-yr-old DMD patient in UP survives with stem cell therapy – ETHealthworld.com | October 22nd, 2019

BOSTON and LONDON, Oct. 22, 2019 (GLOBE NEWSWIRE) -- Orchard Therapeutics (Nasdaq: ORTX), a leading commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies, today announced initial results from a clinical trial with a cryopreserved formulation of OTL-200, a gene therapy in development for the treatment of metachromatic leukodystrophy (MLD) at the San Raffaele-Telethon Institute for Gene Therapy (SR-Tiget) in Milan, Italy. The initial data show that cellular engraftment with OTL-200 using a cryopreserved formulation is similar to that observed using a fresh formulation with the longest patient having 12 months of follow-up since treatment. The data are being featured this week in a poster session at the European Society of Gene & Cell Therapy (ESGCT) Annual Congress in Barcelona, Spain.

MLD is a devastating and rapidly progressing disease with no standard treatment options. In its most severe forms, patients will not survive beyond their first decade of life.

These data compare the initial results of OTL-200 in the first four MLD patients treated using a cryopreserved formulation to a previously presented integrated analysis of 29 patients treated with a fresh formulation that demonstrated meaningful clinical outcomes. Hematopoietic stem cells are collected, purified and transduced in the same way for both formulations. For the cryopreserved formulation, following transduction, the gene-corrected cells are placed in a specific medium that allows them to be stably frozen. After successful testing and release, the cryopreserved cells are shipped to the site of care where they are thawed and administered to patients who have received conditioning.

Presenting the first supportive data on OTL-200 using a cryopreserved formulation represents a cross-functional effort involving our clinical, CMC and regulatory teams as we prepare for the upcoming European regulatory submission for MLD followed by a BLA in the U.S., said Mark Rothera, president and chief executive officer of Orchard. If approved, a cryopreserved formulation of OTL-200 would more readily facilitate global commercialization and patient access efforts, which are key elements in our mission to deliver potentially curative therapies to patients suffering from often-deadly rare diseases.

Mr. Rothera continued, With over 40 patients now treated using a cryopreserved formulation across our pipeline of six clinical-stage programs, we are confident our approach is supported by a robust set of evidence.

Study Results At the time of the analysis, four early-onset MLD patients (two late infantile and two early juvenile) have been treated with the cryopreserved formulation of OTL-200. All patients are alive and were followed for a minimum of one month, with the longest follow-up out to 12 months in the first patient treated (median follow-up of 0.38 years). The age at the time of treatment ranged from seven months to 42 months.

The initial results in patients receiving the cryopreserved formulation (n=4) demonstrated the following:

Figure 1. Profiles of VCN in bone marrow CD34+ cells: OTL-200 cryopreserved vs. OTL-200 fresh

https://www.globenewswire.com/NewsRoom/AttachmentNg/83f41457-927b-4b1b-9ac2-9d48ac10353a

Figure 2. ARSA activity profile in peripheral blood: OTL-200 cryopreserved vs. OTL-200 fresh

https://www.globenewswire.com/NewsRoom/AttachmentNg/393ca5f0-98ad-47f8-b723-35c5c6c08d8f

c = cryopreserved; f = fresh; Sbj. = subject

We are pleased that these initial data suggest that using gene-corrected cells that have been cryopreserved has a similar impact on clinical biomarkers for early-onset MLD patients as the OTL-200 fresh formulation, said Dr. Valeria Calbi, a hematologist at San Raffaele Scientific Institute and SR-Tiget and an investigator of the study. The four treated patients showed good levels of engraftment of gene-corrected cells and reconstitution of ARSA activity at multiple time points, as well as encouraging early trends in GMFM scores that we look forward to evaluating with additional follow-up. We believe that these data further support the positive benefit / risk profile of OTL-200 as a therapy with potential lifelong benefit for patients with MLD.

Next Steps for OTL-200 Orchard remains on track to submit a marketing authorization application, or MAA, in Europe for MLD in the first half of 2020, as well as a biologics licensing application, or BLA, in the U.S. approximately one year later.

About MLD and OTL-200Metachromatic leukodystrophy (MLD) is a rare and life-threatening inherited disease of the bodys metabolic system occurring in approximately one in every 100,000 live births. MLD is caused by a mutation in the arylsulfatase-A (ARSA) gene that results in the accumulation of sulfatides in the brain and other areas of the body, including the liver, the gallbladder, kidneys, and/or spleen. Over time, the nervous system is damaged and patients with MLD will experience neurological problems such as motor, behavioral and cognitive regression, severe spasticity and seizures, finding it more and more difficult to move, talk, swallow, eat and see. Currently, there are no effective treatments for MLD. In its late infantile form, mortality at 5 years from onset is estimated at 50% and 44% at 10 years for juvenile patients.1 OTL-200 is an ex vivo, autologous, hematopoietic stem cell-based gene therapy being studied for the treatment of MLD. OTL-200 was acquired from GSK in April 2018 and originated from a pioneering collaboration between GSK and the Hospital San Raffaele and Fondazione Telethon, acting through their joint San Raffaele-Telethon Institute for Gene Therapy in Milan, initiated in 2010.

About OrchardOrchard Therapeutics is a fully integrated commercial-stage biopharmaceutical company dedicated to transforming the lives of patients with serious and life-threatening rare diseases through innovative gene therapies.

Orchards portfolio of ex vivo, autologous, hematopoietic stem cell (HSC) based gene therapies includes Strimvelis, a gammaretroviral vector-based gene therapy and the first such treatment approved by the European Medicines Agency for severe combined immune deficiency due to adenosine deaminase deficiency (ADA-SCID). Additional programs for neurometabolic disorders, primary immune deficiencies and hemoglobinopathies are all based on lentiviral vector-based gene modification of autologous HSCs and include three advanced registrational studies for metachromatic leukodystrophy (MLD), ADA-SCID and Wiskott-Aldrich syndrome (WAS), clinical programs for X-linked chronic granulomatous disease (X-CGD), transfusion-dependent beta-thalassemia (TDT) and mucopolysaccharidosis type I (MPS-I), as well as an extensive preclinical pipeline. Strimvelis, as well as the programs in MLD, WAS and TDT were acquired by Orchard from GSK in April 2018 and originated from a pioneering collaboration between GSK and the San Raffaele Telethon Institute for Gene Therapy in Milan, Italy initiated in 2010.

Orchard currently has offices in the U.K. and the U.S., including London, San Francisco and Boston.

Forward-Looking StatementsThis press release contains certain forward-looking statements which are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements may be identified by words such as anticipates, believes, expects, intends, projects, and future or similar expressions that are intended to identify forward-looking statements. Forward-looking statements include express or implied statements relating to, among other things, Orchards expectations regarding the timing of regulatory submissions for approval of its product candidates, including OTL-200 for the treatment of metachromatic leukodystrophy, the timing of interactions with regulators and regulatory submissions related to ongoing and new clinical trials for its product candidates, the timing of announcement of clinical data for its product candidates, including OTL-200, and the likelihood that such data will be positive and support further clinical development and regulatory approval of its product candidates, and the likelihood of approval of such product candidates by the applicable regulatory authorities. These statements are neither promises nor guarantees and are subject to a variety of risks and uncertainties, many of which are beyond Orchards control, which could cause actual results to differ materially from those contemplated in these forward-looking statements. In particular, the risks and uncertainties include, without limitation: the risk that any one or more of Orchards product candidates, including OTL-200, will not be successfully developed or commercialized, the risk of cessation or delay of any of Orchards ongoing or planned clinical trials, the risk that prior results, such as signals of safety, activity or durability of effect, observed from preclinical studies or clinical trials will not be replicated or will not continue in ongoing or future studies or trials involving Orchards product candidates, the delay of any of Orchards regulatory submissions, the failure to obtain marketing approval from the applicable regulatory authorities for any of Orchards product candidates, the receipt of restricted marketing approvals, and the risk of delays in Orchards ability to commercialize its product candidates, if approved. Given these uncertainties, the reader is advised not to place any undue reliance on such forward-looking statements.

Other risks and uncertainties faced by Orchard include those identified under the heading Risk Factors in Orchards annual report on Form 20-F for the year ended December 31, 2018 as filed with the U.S. Securities and Exchange Commission (SEC) on March 22, 2019, as well as subsequent filings and reports filed with the SEC. The forward-looking statements contained in this press release reflect Orchards views as of the date hereof, and Orchard does not assume and specifically disclaims any obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events or otherwise, except as may be required by law.

1Mahmood et al. Metachromatic Leukodystrophy: A Case of Triplets with the Late Infantile Variant and a Systematic Review of the Literature. Journal of Child Neurology 2010, DOI: http://doi.org/10.1177/0883073809341669

Contacts

InvestorsRenee LeckDirector, Investor Relations+1 862-242-0764Renee.Leck@orchard-tx.com

MediaMolly CameronManager, Corporate Communications+1 978-339-3378media@orchard-tx.com

Figure 1

Profiles of VCN in bone marrow CD34+ cells: OTL-200 cryopreserved vs. OTL-200 fresh

Figure 2

ARSA activity profile in peripheral blood: OTL-200 cryopreserved vs. OTL-200 fresh; c = cryopreserved; f = fresh; Sbj. = subject

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Orchard Therapeutics Presents Data from OTL-200 in Patients with Metachromatic Leukodystrophy Using Cryopreservation - BioSpace

Bone Marrow Stem Cells Comments Off on Orchard Therapeutics Presents Data from OTL-200 in Patients with Metachromatic Leukodystrophy Using Cryopreservation – BioSpace | October 22nd, 2019

The volume of blood needed every day by the NHS, and around the world, is staggering. But what about growing blood in a lab, could we do that? Chris Smith was joined by Cedric Ghevaert from the department of haematology at the University of Cambridge, to find out more about lab grown blood...

Cedric - So platelets are one of the three main blood components. Theyre actually the smallest cell in the body, if you look at a millimetre on a ruler you can line 250 platelets in that millimetre. The interesting thing is that some people argue they're not even a cell, because they don't contain a nucleus, they don't have DNA.

Chris - When I was learning biology at school they said platelets are bits of cells.

Cedric - That's absolutely true. They are fragments of a parent cell that lives in the bone marrow, and called a megakaryocyte, and one megakaryocyte will release about 1000 to 2000 of these little fragments called the platelets.

Chris - Every day?

Cedric - Every day we produce 10 to the 11 platelets

Chris - 100 billion Every day?

Cedric - Thats right.

Chris - And so these cells are just budding off little bits of themselves, which then go circulating around in the bloodstream.

Cedric - That's absolutely right.

Chris - And what are they doing there? What is their role?

Cedric - The main role of the platelets is to monitor your blood vessels, so they contain two things: Their outer layer, which is called the membrane. And on that membrane they have all sorts of little receptors, and these receptors will tell the platelets that the blood vessel has been damaged. When the platelet detects that it does two things, it will attach to the damaged blood vessel, and it will become activated and by that, it will then tell other platelets around, you need to come and help. These platelets will then stick to each other and form a plug to literally block the hole.

Chris - Do they change shape or anything? Do they become, sort of spiky or anything, to become more jagged so they jam in the hole.

Cedric - So they do become spiky, indeed you see that under the microscope when you activate the platelets. They go from a disc to this sort of spider, and that allows them to indeed interact with each other even better. The thing that they also do, is to then pull. They literally pull the wound together to try to stem the blood flow.

Chris - And they're presumably the first responders when you have a wound. They're there first because they're at the scene of the crime already because they're in the blood. And then as more come along, they're recruiting more of their mates from the numbers that come in the blood flow, and what do they provide the initial foundation of a blood thrombus or a clot.

Cedric - That's absolutely right. So once they become activated, inside the platelets there are granules, and those granules contain things that tell the blood, you need to clot. These things are released when the platelets become activated. And that leads to an amplification of the blood clotting that proteins are linked together, they form a polymer, and that polymer is a sort of a mesh that will capture more platelets and really plug the hole.

Chris - So the platelets are pulling more raw materials that are dissolved in the blood into that wound site, and then turning it into this dense mesh work that's gonna be a stable repair.

Cedric - That's absolutely right.

Chris - So they're really critical aren't they?

Cedric - Absolutely.

Chris - We can't do without them. And what's the problem with just growing them in a dish, because we can grow loads of things in dishes these days. We can you know, cells grow in dishes easily. So why can't you just churn out platelets in a dish?

Cedric - The main challenge with producing platelets in a dish is to do it so efficiently that actually we have a product that can be used, for example by the NHS and cost efficient. So if you look at a bag of platelets which we give for a transfusion, it contains three times ten to the eleven.

Chris - So 300 billion platelets.

Cedric - So where the platelets score as it were, is that we only need to produce one megakaryocyte, to produce a thousand platelets. And we can grow the megakaryocyte from stem cells. So the idea is that we can take stem cells, grow them into a megakaryocyte, and then right at the last stage of production, suddenly you have this massive amplification, a thousand times more platelets than you had megakaryocytes.

Chris - But if it's that easy to just grow these things in a dish, why are we not doing it? What is the what's the problem at the moment.

Cedric - The main problem is that particular last step. When the megakaryocyte is in the bone marrow it gets its cue from its environment. And it will detect the blood flow. It will be talked to by the cells that are around it, and that, it's very difficult to reproduce in the dish. If we produce megakaryocytes in liquid, in a culture dish they can produce 1 to 10 platelets, so we are at least a hundred times below what a megakaryocyte can do.

Chris - So you've got to have some way of recreating that very specialised three dimensional relationship in the bone marrow, where all these cells are in contact in a particularly special arrangement which seems to be the cue to them, to churn out platelets with the efficiency that they do when they're inside the body.

Cedric - And that's exactly the challenge that my group and several other groups across the world are trying to answer. And there are two ways to do this. First we need to tell the megakaryocyte theres a flow. They sense the flow, and that makes them release the platelets. So we put them in a bioreactor where they're exposed to shear, which is basically fluid going along them.

Chris - That's kind of mimicking the blood flowing through the bone marrow. So that would normally be bending and distorting the cells a bit, I presume, and that's what makes them churn off or snap off bits.

Cedric - That's right. So they produce these long digits which we call proplatelets, and these long digits elongate in the bloodstream and then snap off these platelets.

Chris - And when you make them, having mimicked this as best you can, do the platelets that you produce in the dish look like, and critically work like, the ones that are made naturally in the bone marrow?

Cedric - That's the critical thing that we are trying to address at the moment. They are bigger when we produce them in the dish, and they don't seem to quite react like normal platelets. However that doesn't mean that it wont work really well. What we need to do is to test them through a range of assays to really make the statement; these platelets are good, they will monitor your blood vessel, they will last in circulation.

Chris - Is your aim to make platelets bespoke for a patient? Or would you make off the shelf platelets, a bit like we'd currently do with transfusion medicine, where we just make a big bag of platelets collected from a range of donors?

Cedric - So at the moment we can produce platelets from either four donations, from four different donors, or we take them off a special machine where we have one pool of platelets coming from one donor, but we've talked the blood group before, one of the challenges with platelets, is that some people are immunised and they need platelets of a very specific blood type.

Chris - When you say immunisation, you mean that they've made an immune reaction to certain types in the past, so you've got to basically restrict what types you give them?

Cedric - Exactly. The beauty of working with stem cells is that we can edit the DNA somewhat, and because we can edit the DNA we can actually make platelets that don't express blood group, that are universal platelets. The one we produce in the dish can go to anyone. And that's one of the beauties of this technology.

Chris - And are you far away?

Cedric - We are not that far away. We are looking at human clinical trials in the next two to three years.

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Making blood in the lab | Interviews - The Naked Scientists

Bone Marrow Stem Cells Comments Off on Making blood in the lab | Interviews – The Naked Scientists | October 22nd, 2019

Blood stem cells protect themselves against viruses with structures known as 'interferon-induced transmembrane proteins,' seen here in green. These normally useful proteins are problematic for gene therapy treatments, as they work to keep therapeutic lentiviral vectors from infiltrating cells. Scripps Research scientists found a natural compound that lets down this shield, boosting the success rate of gene delivery.Credit: Image courtesy of the Torbett lab at Scripps ResearchLA JOLLA, CA Gene therapy has broadened the treatment possibilities for those with immune system deficiencies and blood-based conditions, such as sickle cell anemia and leukemia. These diseases, which once would require a bone marrow transplant, can now be successfully treated by modifying patients' own blood stem cells to correct the underlying genetic problem.

But today's standard process for administering gene therapy is expensive and time-consuminga result of the many steps required to deliver the healthy genes into the patients' blood stem cells to correct a genetic problem.

In a discovery that appears in the journalBlood, scientists atScripps Researchbelieve they have found a way to sidestep some of the current difficulties, resulting in a more efficient gene delivery method that would save money and improve treatment outcomes.

"If you can repair blood stem cells with a single gene delivery treatment, rather than multiple treatments over the course of many days, you can reduce the clinical time and expense, which removes some of the limitations of this type of approach," says Bruce Torbett, PhD, associate professor in the Department of Immunology and Microbiology, who led the research.

The new finding centers on caraphenol A, a small molecule closely related to resveratrol, which is a natural compound produced by grapes and other plants and found in red wine. Resveratrol is widely known as an antioxidant and anti-inflammatory agent. Similar to resveratrol, caraphenol A is anti-inflammatory, but in this study, it served a different role.

Torbett and his team became interested in the unique chemical properties of resveratrol and similar types of molecules and wondered if they could enable viral vectors, used in gene therapy to deliver genes, to enter blood stem cells more easily. This would be momentous because stem cellsand in particular, self-renewing hemopoietic stem cellshave many barriers of protection against viruses, making them challenging for gene therapy to infiltrate.

Related Article:Solution to 50-Year-Old Mystery Could Lead to Gene Therapy for Common Blood Disorders

"This is why gene therapy of hemopoietic stem cells has been hit-or-miss," Torbett says. "We saw a way to potentially make the treatment process significantly more efficient."

The gene therapy treatment process currently requires isolating a very small population of hemopoietic stem cells from the blood of patients; these young cells can self-renew and give rise to all other types of blood cells. Therapeutic genes are then delivered to these cells via specially engineered viruses, called "lentiviral vectors," which leverage viruses' natural knack for inserting new genetic information into living cells.

However, hemopoietic stem cells are highly resilient to viral attacks. They protect themselves with structures known as interferon-induced transmembrane (IFITM) proteins, which intercept lentiviral vectors. Because of this, it can take many attemptsand a large quantity of expensive gene therapy vectorsto successfully delivery genes into hemopoietic stem cells, Torbett says.

Torbett and his team found that by adding the resveratrol-like compound, caraphenol A, to human hemopoietic stem cells, along with the lentiviral vector mix, the cells let down their natural defenses and allowed vectors to enter more easily. Once the treated stem cells were placed into mice, they divided and produced blood cells containing the new genetic information.

Another key benefit of the approach is time: If gene delivery treatment of blood stem cells can be accomplished in less time, the cells can be re-administered to the patient sooner. This not only makes treatment more convenient for the patient, but it helps to ensure the stem cells don't lose their self-renewing properties, Torbett says. The longer stem cells exist outside of the body and are manipulated, the more likely it is they will lose their ability to self-generate and ultimately correct disease.

Torbett and his team are continuing to study the underlying reasons for stem cells' inherent resistance to genetic modification, with the goal of further improving treatment efficiency and reducing cost. Because many of the diseases treatable with gene therapy affect children, Torbett says he feels a special urgency to advance this discovery from the lab into the clinic.

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When Added to Gene Therapy, Plant-Based Compound May Enable Faster, More Effective Treatments - Lab Manager Magazine

Bone Marrow Stem Cells Comments Off on When Added to Gene Therapy, Plant-Based Compound May Enable Faster, More Effective Treatments – Lab Manager Magazine | October 22nd, 2019

The combination of trilaciclib and chemotherapy was generally welltolerated with promising activity in a phase II trial of patients with metastatic triple-negative breast cancer. However, it did not offer improvements in measures of myelosuppression compared with chemotherapy alone.

Chemotherapy-induced myelosuppression commonly leads to dose reductions that can restrict therapeutic dose intensity, wrote study authors led by Antoinette R. Tan, MD, of the Levine Cancer Institute in Charlotte, North Carolina. Introducing therapy that can protect the immune cells and bone marrow from the cytotoxic effects of chemotherapy has the potential to optimize antitumour activity while minimizing myelotoxicity.

Triliciclib is an inhibitor of cyclin-dependent kinases-4/6; it can enhance antitumor immunity and preserve hematopoietic stem and progenitor cells during chemotherapy. The new phase II study included a total of 142 patients with metastatic triple-negative breast cancer randomized to receive either gemcitabine (Gemzar) plus carboplatin alone (group 1) or 1 of 2 regimens with those agents plus trilaciclib. One regimen included intravenous trilaciclib along with the chemotherapy agents on days 1 and 8 of 21-day cycles (group 2), while the other included gemcitabine and carboplatin on days 2 and 9 along with trilaciclib on days 1, 2, 8, and 9 (group 3). The results of the study werepublishedonline ahead of print on September 28 inLancet Oncology.

The median follow-up for the three groups was 8.4 months, 12.7 months, and 12.9 months, respectively. During the first cycle, the mean duration of severe neutropenia was 0.8 days in the chemotherapy alone group, 1.5 days in group 2, and 1.0 days in group 3; these differences were not significant.

A total of 9 of 34 patients (26%) in group 1, compared with 12 of 33 patients (36%) in group 2 and eight of 35 patients (23%) in group 3 (P= 0.70). There were no differences between the groups with regard to all-cause dose reductions, patients requiring G-CSF, or patients requiring red blood cell transfusion, among other outcomes. The most common treatment-emergent adverse events included anemia, neutropenia, and thrombocytopenia in groups 1 and 2, and in group 3 they included neutropenia, thrombocytopenia, and nausea.

Overall survival outcomes in the 2 trilaciclib groups were significantly better than in the chemotherapy alone group. The combined trilaciclib groups had a median OS of 20.1 months, compared with 12.6 months without trilaciclib.

Though the trilaciclib regimens did not show significant improvements with regard to myelosuppression compared with chemotherapy, the authors highlighted the improvement in anti-tumor activity.

Together with the safety profile reported, the clinically meaningful improvements in overall survival support further studies of trilaciclib in patients with metastatic triple-negative breast cancer, they wrote.

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Can Triliaciclib Improve Chemotherapy Tolerance in Triple-Negative Breast Cancer? - Cancer Network

Bone Marrow Stem Cells Comments Off on Can Triliaciclib Improve Chemotherapy Tolerance in Triple-Negative Breast Cancer? – Cancer Network | October 22nd, 2019

Since MM4MM began with its first climb in 2016, the program has raised over $2.7 million. All the funds raised go directly to the MMRF to accelerate new treatment options for patients with multiple myeloma.

As a patient founded organization, the MMRF stands together with those who are battling multiple myeloma patients, families, physicians, researchers, and our pharmaceutical partners. This team represents a microcosm of that myeloma community and demonstrates that together, we can collaborate with ever increasing momentum towards a cure, said Paul Giusti, CEO of the Multiple Myeloma Research Foundation. We are thrilled to enter the fifth year of this inspiring program and to have Celgene join us in this effort to raise awareness and critical funds to continue our mission.

The MM4MM team will include four patients living with multiple myeloma:

We are so honored to be a part of yet another hike with the MMRF and Celgene, said Mike Hennessy Jr., president and CEO of MJH Life Sciences, parent company of CURE magazine. This initiative organized by Moving Mountains for Multiple Myeloma not only raises awareness and research funding for multiple myeloma but has brought together the myeloma community to take action and fight for a cure for myeloma patients.

The team will embark on a five-day trek of a lifetime through Patagonia and take on the rewarding and beautiful landscape that includes glaciers, deep valleys and challenging peaks. During this trek, the team will travel through El Chaltn and acclimatize while they experience the mighty range of peaks dominated by Monte Fitz Roy, an 11,020-foot tower with a sheer face of more than 6,000 feet. Next, the team will reach Lago San Martin, where they will traverse the terrain in daily treks, exploring a 10-mile peninsula, climbing to a condor rookery and reaching remote Andean lakes.

Celgene, Cure and the MMRF share an unwavering commitment to improving the lives of patients with multiple myeloma and we are very proud to continue our role in the Moving Mountains for Multiple Myeloma initiative, said Chad Saward, senior director, patient advocacy at Celgene Corp. We are amazed and inspired by all who are participating in this unique awareness program.

To learn more about MM4MM and to donate to multiple myeloma research, click here.

About Moving Mountains for Multiple Myeloma

Moving Mountains for Multiple Myeloma (MM4MM) is a collaboration between CURE Media Group and the Multiple Myeloma Research Foundation (MMRF) to raise awareness and funds for myeloma research. This year, Celgene Corporation and GSK join the effort as sponsors. In addition to Patagonia, the program also led hikes up Mt. Washington and through Iceland in 2019. To date, MM4MM has raised over $2.7 million for myeloma research and included 51 patients with multiple myeloma on 7 climbs. Funds raised go directly to research, supporting the MMRF mission. For more information, visit https://www.themmrf.org/events/.

About Multiple Myeloma

Multiple myeloma (MM) is a cancer of the plasma cell. It is the second most common blood cancer. An estimated 32,110 adults (18,130 men and 13,980 women) in the United States will be diagnosed with MM in 2019 and an estimated 12,960 people are predicted to die from the disease. The five-year survival rate for MM is approximately 50.7%, versus 31% in 1999.

About the Multiple Myeloma Research Foundation

A pioneer in precision medicine, the Multiple Myeloma Research Foundation (MMRF) seeks to find a cure for all multiple myeloma patients by relentlessly pursuing innovations that accelerate the development of precision treatments for cancer. Founded in 1998 by Kathy Giusti, a multiple myeloma patient, and her twin sister Karen Andrews as a 501(c)(3) nonprofit organization, the MMRF has created the business model around cancerfrom data to analytics to the clinic. The MMRF identifies barriers and then finds the solutions to overcome them, bringing in the best partners and aligning incentives in the industry to drive better outcomes for patients. Since its inception, the organization has collected thousands of samples and tissues, opened nearly 100 trials, helped bring 10 FDA-approved therapies to market, and built CoMMpass, the single largest genomic dataset for any cancer. Today, the MMRF is building on its legacy in genomics and is expanding into immune-oncology, as the combination of these two fields will be critical to making precision medicine possible for all patients. The MMRF has raised nearly $500 million and directs nearly 90% of the total funds to research and related programs. To learn more, visit http://www.themmrf.org.

About CURE Media Group

CURE Media Group is the leading resource for cancer updates, research and education. It combines a full suite of media products, including its industry-leading website, CUREtoday.com; innovative video programs, such as CURE Connections; a series of widely attended live events; and CURE magazine, which reaches over 1 million readers, as well as the dynamic website for oncology nurses, OncNursingNews.com, and its companion publication, Oncology Nursing News. CURE Media Group is a brand of MJH Life Sciences, the largest privately held, independent, full-service medical media company in the U.S. dedicated to delivering trusted health care news across multiple channels.

View source version on businesswire.com: https://www.businesswire.com/news/home/20191022006008/en/

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Multiple Myeloma Experts, Patients, Advocates and Caregivers Team Up to Hike Through Patagonia - BioSpace

Skin Stem Cells Comments Off on Multiple Myeloma Experts, Patients, Advocates and Caregivers Team Up to Hike Through Patagonia – BioSpace | October 22nd, 2019

Turmeric has numerous uses when it comes to health benefits. They are being used in Indian households for a long time.These include several health benefits and medicinal uses. Turmeric is one of the most powerful spices. It has a unique taste with a mix of citrusy bitterness. It is also associated with Ayurvedic practices.

Also read:Indian Food: What Are The Uses Of Turmeric In Indian Dishes?

Turmeric also has some benefits to enhance your beauty. Its anti-inflammatory properties help in removing dead skin cells. It can also be used to wash your fash or apply once in a while. There are several benefits you can receive from turmeric. These are some of the imperative ones.

Also read:Basil Benefits: Top Benefits Of Basil For Your Skin

The anti-inflammatory properties that are found in turmeric are used to soothe osteoarthritis and rheumatoid arthritis. These collectively work in your favour. The antioxidant destroys the free radicals in the body that damage the cells. These can help alleviate and relax your mild joint pains. It cannot be used as a substitute for medication.

There is a compound in turmeric that has not been studied as much as the other compounds like curcumin - aromatic turmerone or ar-turmerone. This compound has reportedly been repairing brain stem cells. It also helps in the recovery from neurodegenerative diseases like stroke and Alzheimer's.

A substance in turmeric Lipopolysaccharide has anti-bacterial, anti-fungal, and anti-viral agents. This also helps to stimulate the immune system. Make sure you consume only a teaspoon in warm water.

Also read:Jackfruit: Delicious Recipes To Make With The Diabetic Friendly Fruit

The anti-inflammatory and antioxidant properties of curcumin help to reduce the onset of Type 2 diabetes. It helps to moderate insulin levels and boosts the effect of medications that treat diabetes. But always remember not to use it as a source of medication.

Turmeric increases the production of vital enzymes that detoxify our blood in the liver by breaking down and reducing the toxins. It also helps with the circulation of blood. Overall, it is known to improve liver health.

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Turmeric: Uses and benefits of the spice that you must know - Republic World - Republic World

Skin Stem Cells Comments Off on Turmeric: Uses and benefits of the spice that you must know – Republic World – Republic World | October 22nd, 2019

Exactly when did Halloween get to be so high pressure? Back in the day, cooking up a scary monster or pretty fairy princess costume was all about having fun. But somewhere along the way, all of it got stressful. The hair, the clothing, the Halloween makeup ideasits all expected to look super pro. Its enough to give you flashbacks to how you felt before that super hard biology final the last semester of freshman year. At least, thats how we feel. And thats all on top of trying to figure out a cool costume in the first place.

But rather than spend a bajillion dollars on a costume that someone else will inevitably be wearing, why not let your face be the...

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Seriously Cool Halloween Makeup Ideas That Will Overshadow a Basic Costume - Yahoo Lifestyle

Skin Stem Cells Comments Off on Seriously Cool Halloween Makeup Ideas That Will Overshadow a Basic Costume – Yahoo Lifestyle | October 22nd, 2019

The immune system functions as the bodys defense against pathogens and harmful substances, including cancer cells. They protect the body against potential harm. In cases of cancer, though the body tries to fight the tumor cells, they may become too aggressive for the immune system to eradicate.

Creatine is a popular organic acid that is widely used as a supplement by bodybuilders and athletes. A new study has found that creatine is a potent molecular battery that powers the immune cells to better fight cancer.

Creatine powder - Image Credit: Pavlo Lys/ Shutterstock

In a new study published in the Journal of Experimental Medicine, creatine uptake is vial to the anti-tumor activities of CD8 T cells, also called killer T cells. These immune cells find and destroy infected cells that have been turned into virus-making factories.

The researchers at the University of California Los Angeles Health Services conducted the study on mice showing taking creating supplements can make current immunotherapies more effective.

"Because oral creatine supplements have been broadly utilized by bodybuilders and athletes for the past three decades, existing data suggest they are likely safe when taken at appropriate doses. This could provide a clear and expedient path forward for the use of creatine supplementation to enhance existing cancer immunotherapies, Lili Yang, a member of the Eli and Edythe Broad Center of Regenerative Medicine and Stem Cell Research at UCLA, said.

T cells, to fight cancer cells effectively, need massive energy. The metabolic regulators that control anti-tumor T cell immunity have just begun to be revealed. The researchers observed a rise of the CrT (Slc6a8) gene expression, which works by encoding a surface transporter that controls the uptake of creatine into a cell.

The team demonstrated that creatine uptake deficiency has affected and altered anti-tumor T cell immunity. On the other hand, when they gave creatine supplements to mice, there had been tumor suppression efficacy. Further, the researchers have found that creatine served as a molecular battery, wherein it stored and distributed energy to power the bodys fight against cancer.

"As biologists, we are always asking 'why? We could see that these tumor-battling T cells had increased their capacity to take in creatine, likely for a good reason, so we designed experiments to determine what happens when they can't get it, Yang said.

To validate their theory, they gave non-engineered mice models creatine daily, which is similar to the safe recommended dose for bodybuilders and athletes. The boost of creatine, which was administered through an injection while in some mice, they received it through oral supplementation, gave the two groups of mice the ability to suppress both colon and skin cancer growth.

The findings of the study show that killer T cells need creatine to be able to fight cancer cells. Without creatine, they appear underpowered and cant perform their roles effectively.

In the next series of experiments, the team will try to repeat the tests on special mouse models that contain human tumor grafts and human immune cells. This way, they can determine the proper dose and proper timing of creatine supplementation. Also, they want to see if the same mechanism is true in humans. The team expects the findings could also apply to a variety of cancers, further improving current immunotherapies.

Creatine is a natural compound produced in the body from amino acids, which are the building blocks of protein. Creatine comes from many sources, such as animal proteins and dietary supplements. It has many health benefits, including help muscles, produces energy, improves high-intensity exercise performance, fights neurological diseases, lowers blood sugar levels, and supports muscular function.

The researchers recommend that people should consult a doctor before taking new supplements, such as creatine. Supplements can carry risks of drug interactions with other medicines and other potentially harmful side effects.

Journal reference:

Biase, S., Ma, X., Wang, X., Yu, Wang, Y.C., Smith, D., Zhou, Y., Li, Z., Kim, J., Clarke, N., To, A. and Yang, Li. (2019). Creatine uptake regulates CD8 T cell antitumor immunity. Journal of Experimental Medicine. http://jem.rupress.org/content/early/2019/10/17/jem.20182044

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Creatine powers fight against cancer, improves efficacy of immunotherapies - News-Medical.net

Skin Stem Cells Comments Off on Creatine powers fight against cancer, improves efficacy of immunotherapies – News-Medical.net | October 22nd, 2019

Season 4 ofRiverdalewill feature the series first-ever Halloween episode finally! That means fans get to see Archie, Cheryl and the rest of the gang all dressed up in their finest ~spooky~ gear. The episode doesnt air until October 30, but you can get a sneak peek at the costumes in these Riverdale season 4 Halloween episode photos. Maybe youll find some inspo for your own Halloween costume?

Season 4 Episode 4 of Riverdale is aptly titled, Halloween. You know this series loves its outlandish costumes, so its about time that they devoted an episode to the holiday! The CW released a batch of stills from the episode, and they reveal exactly what Archie, Mad Dog, Cheryl, and Toni dressed up as.

The characters went with a superhero theme. Archie dressed up as his own superhero...

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Archie & Cheryls Costumes On The Halloween Episode Of Riverdale Are So On-Brand - Yahoo Lifestyle

Skin Stem Cells Comments Off on Archie & Cheryls Costumes On The Halloween Episode Of Riverdale Are So On-Brand – Yahoo Lifestyle | October 22nd, 2019

Grant will support researchers from Kings College London and Kings College Hospital to test a personalised treatment approach for Aplastic Anaemia patients who have not responded to available therapies

21 October 2019 - LifeArc, a UK-based medical research charity, and the Aplastic Anaemia Trust (AAT) have jointly awarded a 1.15m research grant to Kings College London and Kings College Hospital to investigate the potential of a novel type of personalised cellular therapy to reverse the ultra-rare condition aplastic anaemia (AA). The results of this research could give new hope to people living with a severe, life-limiting form of this condition.

The grant will fund a clinical trial to investigate the safety and efficacy of using a patients own T-reg cells to restore the blood-making function of the bone marrow. This follows laboratory-based research from the team of scientists where T-reg cells from a patients own blood were collected, selected for activity and multiplied. In a test tube, these cells prevented the immune system from attacking the patients bone marrow stem cells.[i]

Professor Ghulam Mufti, Department of Haematological Medicine at Kings College London and Kings College Hospital, and lead study investigator said: For patients with this ultra-rare disease, were looking for the first time at a personalised medicine approach where their own immune cells could be used to alter their disease. In AA there is a reduction in the number of T-regs and most of the ones that the AA patients do have are non-functional. Weve seen success in the laboratory by selecting and bolstering the number of functional T-reg cells. Now, with funding from LifeArc and the AAT, we can investigate the potential of this approach in treating AA patients who currently have very limited treatment options. AA is an ultra-rare life-threatening illness caused by the bone marrow failing to make enough of all three types of blood cellsred blood cells, white blood cells and platelets. Only around 100150 people in UK are diagnosed per year, affecting all ages but most commonly people between the ages of 10 to 20 years old and those over the age of 60 years.

People with the illness are at greater risk of infections, bleeding, and can experience extreme fatigue, which leaves them unable to carry out simple daily tasks that most people take for granted. Around one in three patients with severe AA fail to respond to existing drug treatments and the other option a bone marrow transplant is reliant on finding a suitable donor, requires life-long treatment with immunosuppression therapy and is unsuccessful in one in three people. Dr Catriona Crombie, LifeArcs Head of Philanthropic Fund explained why the charity had approved the funding: LifeArc set up the Philanthropic fund to support translational research into rare diseases, where there is less interest from commercial organisations. Patients with AA can have limited treatment options; this opportunity with Kings College London, Kings College Hospital and the AAT has the potential to transform the lives of patients living with a severe form of the disease. The trial at Kings College London and Kings College Hospital will run for a duration of three years and aims to recruit nine patients. A blood sample of the patients T-reg cells will be extracted, purified and grown in the lab before being given back to the patient in a higher concentration. As patients with AA are more susceptible to infection, this personalised treatment approach is more likely to avoid the risk of severe infection and inflammation.

Grazina Berry, CEO of the AAT said: AA can severely impact a persons quality of life. Through AATs close work with Kings College London and Kings College Hospital as a specialist centre of clinical care and research in AA, we identified the project with the most potential to directly benefit patients who are currently at a loss for solutions. We are delighted to have partnered with LifeArc and Kings College London and Kings College Hospital to progress this ground-breaking work, which could potentially enable people living with severe AA to once again lead a normal life.

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Patients with ultra-rare bone marrow disease set to benefit from 1.15m grant from LifeArc and The Aplastic Anaemia Trust - PharmiWeb.com

Bone Marrow Stem Cells Comments Off on Patients with ultra-rare bone marrow disease set to benefit from 1.15m grant from LifeArc and The Aplastic Anaemia Trust – PharmiWeb.com | October 21st, 2019

A twice-diagnosed leukaemia patient from Leicester has found a donor after making an emotional plea from his hospital bed.

Cameron Patel was first diagnosed with the illness aged 18 after being rushed to A&E with a dangerously high heart rate.

After months of treatment at Leicester Royal infirmary he was given the all-clear in February and was looking forward to his life getting back to normal.

Despite there being a low chance of Cameron having a relapse in June this year that's exactly what happened.

The leukaemia had returned and this time doctors said he would need a bone marrow transplant.

So the search for the best possible match began.

Due to his dual heritage, the chances of finding a suitable match for him were significantly low so Cameron was told that the search would not be easy.

After celebrating his 20th birthday this month, Cameron from Knighton, Leicester has now revealed that he will be receiving a transplant from his own mother.

"My mum brought me into this world and she's keeping me here," he told LeicestershireLive.

Sarah Patel, Cameron's mum, is only a 50 per cent match for him which is less than a potential match from a stranger would be.

She said: "You support as much as you can but at the end of the day you have to watch him go through it - so to be his donor makes me really happy, even if it is just a half match."

Although every effort was made to find him a match doctors couldn't find a single donor despite looking worldwide.

Now he will have what is called a haploidentical transplant which involves a half-match from a parent.

Sarah said: "Being one of the last resorts made me feel quite worried because even though I'm his mum, a stranger could have been a higher match.

"But he can't keep having chemotherapy forever and we can't wait around too long because he relapsed quite quickly so we know the pattern of his disease now.

"It's been mixed emotions but I'd do anything for him."

In the months leading up to his transplant, which will happen on Thursday 24 October, Cameron and his mother have undergone health checks and and tests to ensure that she is well enough to be his donor.

Any signs of illness or underlying health conditions in Sarah would mean that her stem cells could not be used.

Due to the low percentage match there are risks with the haplo transplant, including graft versus host disease (GvHD) in which the donated bone marrow or stem cells attack the body due to it being foreign.

Cameron will receive conditioning therapy in which chemotherapy will prepare his body for the transplant.

Following the transplant, he will have to remain isolated for 100 days - only his sister and mum will be allowed to see him.

"I've got a few films and games for my recovery period but I can't wait to just get out," he said.

Sarah said that her daughter, Charis has been a "great support for Cameron" and herself.

"They just love each other so much," the 55-year-old said about the siblings.

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The family have coined the day of Cameron's transplant as "day 0" and will count down the days until he can start living a normal life.

"It's been a difficult year or so but I can't really process things - I'm just sort of in this sub-universe where I'm just getting on with it."

"We couldn't do it with out our friends and family and Cameron's friends have been amazing," Sarah said.

Cameron has now moved to Nottingham University Hospital for his transplant, after spending the majority of the last year at Leicester Royal Infirmary.

"To be honest, I've been a bit low about leaving, I miss the nurses and everyone in ward 27 - they're family to me," Cameron said.

"When I relapsed I was in a really bad place and the nurses would come in and watch a film with me or play cards.

"There's doing nurse duties and there's being a friend - I want to thank them for everything."

Throughout his treatment, Cameron has made it his mission to raise awareness of blood disease and how people can help.

"I'm glad that I've been able to get out once in a while to spread the message - it's all good sitting in hospital and doing it from my iPhone but it's nice to go out and see the people you're speaking to," he said.

"Once I'm out of here I want to travel a bit and then carry on spreading awareness to young people," he added.

During his time on the teenage cancer ward Leicester Royal Infirmary, Cameron became friends with Coalville Town's footballer Courtney Wildin, who was also undergoing treatment for leukaemia.

Courtney had a transplant earlier this year and has since stayed in touch with Cameron.

They plan to work together to encourage young people to join the bone marrow donor register.

"He reached out to me while we were both on the ward and we ended up becoming good friends," Courtney said.

"We're going to start off going to colleges and tell people our story."

Without the haploidentical stem cell transplant, Cameron would still be left without a single match.

He wants to help to make sure that there can be a donor for everyone.

He said: "There are a lot of people out there who need a donor - two minutes of your life could save someones entire life."

Link:
Twice-diagnosed leukaemia patient Cameron Patel reveals he has a donor after heartbreaking appeal - Leicestershire Live

Bone Marrow Stem Cells Comments Off on Twice-diagnosed leukaemia patient Cameron Patel reveals he has a donor after heartbreaking appeal – Leicestershire Live | October 21st, 2019

Gene therapy has broadened the treatment possibilities for those with immune system deficiencies and blood-based conditions, such as sickle cell anemia and leukemia. These diseases, which once would require a bone marrow transplant, can now be successfully treated by modifying patients own blood stem cells to correct the underlying genetic problem.

But todays standard process for administering gene therapy is expensive and time-consuminga result of the many steps required to deliver the healthy genes into the patients blood stem cells to correct a genetic problem.

In a discovery that appears in the journal Blood, scientists at Scripps Research believe they have found a way to sidestep some of the current difficulties, resulting in a more efficient gene delivery method that would save money and improve treatment outcomes.

If you can repair blood stem cells with a single gene delivery treatment, rather than multiple treatments over the course of many days, you can reduce the clinical time and expense, which removes some of the limitations of this type of approach, says Bruce Torbett, PhD, associate professor in the Department of Immunology and Microbiology, who led the research.

The new finding centers on caraphenol A, a small molecule closely related to resveratrol, which is a natural compound produced by grapes and other plants and found in red wine. Resveratrol is widely known as an antioxidant and anti-inflammatory agent. Similar to resveratrol, caraphenol A is anti-inflammatory, but in this study, it served a different role.

Torbett and his team became interested in the unique chemical properties of resveratrol and similar types of molecules and wondered if they could enable viral vectors, used in gene therapy to deliver genes, to enter blood stem cells more easily. This would be momentous because stem cellsand in particular, self-renewing hemopoietic stem cellshave many barriers of protection against viruses, making them challenging for gene therapy to infiltrate.

This is why gene therapy of hemopoietic stem cells has been hit-or-miss, Torbett says. We saw a way to potentially make the treatment process significantly more efficient.

The gene therapy treatment process currently requires isolating a very small population of hemopoietic stem cells from the blood of patients; these young cells can self-renew and give rise to all other types of blood cells. Therapeutic genes are then delivered to these cells via specially engineered viruses, called lentiviral vectors, which leverage viruses natural knack for inserting new genetic information into living cells.

However, hemopoietic stem cells are highly resilient to viral attacks. They protect themselves with structures known as interferon-induced transmembrane (IFITM) proteins, which intercept lentiviral vectors. Because of this, it can take many attemptsand a large quantity of expensive gene therapy vectorsto successfully delivery genes into hemopoietic stem cells, Torbett says.

Torbett and his team found that by adding the resveratrol-like compound, caraphenol A, to human hemopoietic stem cells, along with the lentiviral vector mix, the cells let down their natural defenses and allowed vectors to enter more easily. Once the treated stem cells were placed into mice, they divided and produced blood cells containing the new genetic information.

Another key benefit of the approach is time: If gene delivery treatment of blood stem cells can be accomplished in less time, the cells can be re-administered to the patient sooner. This not only makes treatment more convenient for the patient, but it helps to ensure the stem cells dont lose their self-renewing properties, Torbett says. The longer stem cells exist outside of the body and are manipulated, the more likely it is they will lose their ability to self-generate and ultimately correct disease.

Torbett and his team are continuing to study the underlying reasons for stem cells inherent resistance to genetic modification, with the goal of further improving treatment efficiency and reducing cost. Because many of the diseases treatable with gene therapy affect children, Torbett says he feels a special urgency to advance this discovery from the lab into the clinic.

Authors of Resveratrol trimer enhances gene delivery to hematopoietic stem cells by reducing antiviral restriction at endosomes, include Stosh Ozog, Nina D. Timberlake, Kip Hermann, Olivia Garijo, Kevin G. Haworth, Guoli Shi, Christopher M. Glinkerman, Lauren E. Schefter, Saritha DSouza, Elizabeth Simpson, Gabriella Sghia-Hughes, Raymond R. Carillo, Dale L. Boger, Hans-Peter Kiem, Igor Slukvin, Byoung Y. Ryu, Brian P. Sorrentino, Jennifer E. Adair, Scott A. Snyder, Alex A. Compton and Bruce E. Torbett.

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This transcript has been edited for clarity.

Eric J. Topol, MD: Hello. I'm Eric Topol, editor-in-chief for Medscape. I'm thrilled to speak with Hala Durrah today, who I got to know about through a remarkable essay she wrote for Health Affairs back in March. Hala, welcome to our program.

Hala H. Durrah, MTA: Thank you for having me.

Topol: It's a real pleasure and this an important and critical topic. Your essay was "My Child Is Sick; Don't Call Her a 'Consumer.'"[1] There is so much to learn here for Medscape folks. Can you tell us a little bit about your background and your family?

Durrah: Sure. I am a patient/family engagement consultant by profession, which basically means that I work with healthcare organizations and systems around the concept of patient/family engagement, spanning anywhere from patient-centered measurements to quality improvement, and so on. I came to the work, though, not as part of a plan, but by fate and destiny. My firstborn, whose name is Ayah, was born with a very rare liver disease which we knew would require liver transplantation. She is 16 years old now, but as you can imagine, we've been on quite a journey in the healthcare system. In addition, I have four other children and my husband is an adult medical hospitalist at a major academic center on the East Coast, so healthcare is a big piece of our lives.

I realized a few years into the journey with my daughter that I wanted to become more engaged in improving healthcare and partnering with our care teamthe physicians, nurses, pharmacists, and the larger group of specialists that she was seeing on a regular basis. I had the opportunity to volunteer at a local hospital that was looking for more patient/family involvement in some of their quality improvement work, and I decided that this was definitely something I wanted to pursue further, so I did. But my daughter's healthcare journey continues and I continue to see the good, the not so good, and everything in between.

In addition, I think I have a unique perspective because my husband is a physician and works in the hospital setting. I understand and can empathize with the real challenges facing physicians that are constantly placing pressure on them and demanding their time. I am very cognizant of that, so in my work I really try to balance the voice of the patient or caregiver, and the voice of the physician or nurse, because I can definitely see all of the different perspectives. At the end of the day, I think we all really want these meaningful relationships where we communicate and empathize with one another, and at the same time, we all want the best care for each other. That is my goal and the work I've been doing for the past several years.

Topol: That is terrific. You have a panoramic view of the healthcare landscapethat's for sure.

Topol: You wrote about your daughter; it is a miracle story in so many respects. Her name, Ayah, means "sign of God"is that right?

Durrah: Yes. We named her that before I knew she was going to be born with this illness, and it's pretty remarkable that she fit that name and the meaning of her name since she was born. Her first liver transplant, unfortunately, failed within the first 24 hours. As you all know, a liver does not wait, so we were preparing to say goodbye to her when she was about 5 years old.

Subsequently, by miracle, she got another liver 2 days later. It was from the same hospital where she was at, which was even more amazing because her surgeons were prepared to travel anywhere to get another liver. She was placed number one on the nation live listing after that first transplant failed, and it's remarkable that she got another one.

Topol: After she had these two liver transplants at age 5, she got Burkitt lymphoma.

Durrah: Unfortunately, because of her therapies, and also being Epstein-Barr viruspositive, she developed stage III Burkitt lymphoma 2 years after the liver transplant, which was quite devastating, to say the least.

Topol: It required another type of transplant, a bone marrow transplant.

Durrah: Yes. Unfortunately, she had several months of a pretty intense chemotherapy regimen that ultimately failed, and we had to move to an autologous bone marrow transplant. She was not able to have donor cells because she was already an organ transplant recipient. She sat one day in the hospital with a catheter, and they took her stem cells and then gave them back to her about a month later after some more intense chemotherapy.

In all our experiences, I don't believe that physicians and nurses ever defined my daughter as a consumer.

Obviously, she has been through quite a bit. A lot goes along with being a patient of liver disease or a patient who survived cancer with all of the chemotherapy, so while her health is stable, things are constantly moving in the background that we're watching. I tell people that I sometimes look at it like a radar screen. You kind of see the little red dots bleeping and some of them get brighter and some of them get dimmer. A lot of people are monitoring those lights, but I'm the chief monitor of all of those lights and the coordinator of making sure we stay on top of them.

Topol: You are quite an advocate and it is just an amazing story. I want to get into this message that you have about using the term "consumer," because I have hated that term for years. Why do we use this term when we're talking about patients in the health world? You wrote, "I share our story because I am becoming increasingly troubled by a trend in healthcaretoward thinking of patients as 'consumers' but not actually engaging communities in healthcare improvement and innovation." Tell us a bit about this objection of this term, because I think if anybody has a right to object to the use of that term, it would be you.

Durrah: The term definitely does not resonate with me, and I share your strong feelings against the utilization of this term. Overall, I think it continues to underline and push the business imperative of healthcare versus the humanity imperative in which healthcare was initially built upon. I reject in some ways that this notion of consumerism will improve healthcare because healthcare did not start as a business imperative, nor was the framework built to support such a term.

In all of our experiences, I don't believe that physicians and nurses ever defined my daughter as a consumer. I believe [the term] distances us from one another and does not amplify or support that relationship-based care that we all seek to have and to improve. I also don't think "consumer" empowers us, although I understand why some believe it may, because the framework of healthcare is not set up in such a way where "consumer" denotes choice. A lot of healthcare is dictated to the patient or to the caregivers by their insurance companies or their lack thereof, or the communities in which they live, or by lots of different other parties that are involved in it. I'm not quite sure where the choice comes in, especially because no one chooses to be sick. A consumer has choices, but we don't have that choice.

It does not make sense to use that term. I understand where people come from wanting to empower individuals by changing the terminology [to a less] "passive" role of the patient, but I'm not quite sure that changing the terminology we use makes us equals, gives us any more choices, or improves the care we will receive.

Topol: Words are important. This term is hackneyed, it's pervasive, and I think you made the most eloquent case in the history of the medical literature because your intersection with the healthcare world is extensive.

Durrah: Oh my gosh. I'm humbled by that. I'm not sure if I deserve all that, but I appreciate it.

Topol: You have a master's degree from George Washington and you worked in tourism, and you made some interesting parallels between a consumer of tourism versus a patient. Can you talk to us about that?

Durrah: Sure. My master's is in tourism and administration, with a focus in meeting and event management, and here I am in healthcare advocacy now. But I found that it's been very useful because you do see in healthcare this shift of trying to create a tourism-type experience for healthcare. And generally speaking, tourism is a choice; we all choose to travel or to use a certain hotel or rental car company because we've had experiences. Most of the time, you come back from a vacation with great memories, and when you share those memories with others, they may want to have the same experiences as you.

But I don't believe that we can really equate healthcare to a vacation. I'm not quite sure who would utilize that same experience as a vacation. Things that healthcare has that no other industry has are the trauma, pain, and anguish that go into being either a patient or the caregiver of someone. I think physicians and nurses have a lot of emotions in the healthcare experience that you could never find in tourism. And tourism tries to have a constant feedback loop with their customers or consumers; healthcare does not. Yes, we tout these wonderful surveys that are supposed to increase patient satisfaction, but were any of them co-designed with the communities the health systems serve? The answer is no. Do they allow you to really respond in such a way that will provide meaningful data using stories of your experiences in the system? No. So again, trying to equate the healthcare experience with the lessons learned from tourism is a mismatch.

Topol: Right. Even that term "medical tourism" is about a different concept.

There are many parts of your daughter's story that were really touching and deep, but one of the things that stood out was how her liver doctor would hold your hand, and how that was not about consumerism but, as you say, a choice driven by humanity and compassion. This, I think, speaks to the stark dramatic differences between the terms.

Topol: Another term I'm interested in asking you about is "providers." It's another term I don't care for. Should that term be used?

Durrah: It's interesting, because I believe that in the article I do reference "care providers" at one point. Initially when I started in this work, I always [used the specific terms of] "physicians," "nurses," "pharmacists," and so on. All of a sudden that term "provider" got thrown into the mix, and I adapted it because it seemed that everyone was using it to cover all the members of the care team that might interact or touch a patient. I'm guilty of using that term and I got a lot of response after my article came out, particularly from physicians, about how the term "provider" was not liked. I believe the term came about with this whole business imperative/push in healthcare. Insurance companies use that term and healthcare systems have now adopted that term as well, and it probably is in play because of this whole consumerism push as well.

I did prefer saying "physicians" or "nurses" or "pharmacists" and that we're all part of the "care team." I prefer that terminology, just as I prefer to be called a "partner" in my care versus a "consumer" or a "customer." I think that the term does not fit the role that physicians and nurses play. They are not just a provider. I look at them as part of my team and I'm on their team, so we're all working together. I think "provider" also sounds a little bit like a hierarchy. It does not suggest that notion of that partnership and just reinforces a distance. Consumerism is kind of defined by this transactional relationship, so you would use that term to reinforce that. I see how it has developed and how it's being pushed, and I think it's all related to the business of medicine versus the actual human experience and the humanity of medicine.

We so largely lost the 'care' in 'healthcare.

Topol: These terms got adopted when healthcare was transformed to a business, and now we're relooking at this.

Your work and eloquence and what you continue to do as an advocate in patient care will help us get back on track. It's just one of many things we need to do.

Topol: You also brought up the term "lean principles." It would be interesting for you to just touch on this as well.

Durrah: I've noticed in my work in healthcare thus far that health systems are adopting a lot of frameworks. One of these is becoming "lean" or utilizing "lean principles," which are driven by efficiency and cost savings.

Typically, those who advocate for lean principles within health systems say, "This is going to benefit everyone: physicians, nurses, patients and families, and the communities we serve." But if you really dig down deep, I don't think it has trickled down at all to any cost savings for patients or families. I don't think it's improved quality for communities which the healthcare system serves. And I certainly don't see physicians and nurses not being more burned out or more extended. They get affected by lean principles, where staffing is cut in order for that cost savings to occur. They are being told to do 150 things versus 100 things a day.

When I first learned about "lean" and read about it, I thought it sounded great. But then when I got deeper into this work, I thought, "Wait a minutewho is this benefiting other than the bottom line of healthcare systems?" It's really interesting how healthcare tends to cherry-pick principles or terminology from other industries with the goal of trying to improve healthcare, when it's really not doing that. It's simply putting a Band-Aid on one problem and creating a new one.

Topol: We so largely lost the "care" in "healthcare." You referred to the "care team." You undoubtedly experienced that with your daughter, and we want to bring it back. We should be more precise about language and avoid business terms, and talk about clinicians as doctors, nurses, pharmacists, physical therapists, or whoever. We should be more specific than using this "provider" term which obviously could be a relative or a friend.

Topol: What response did you receive from the essay you wrote in Health Affairs? It clearly resonated, and when I posted it on Twitter, there was quite a bit of response.

Durrah: I got an incredible response from the article, and I appreciate you tweeting and retweeting it because a lot of people saw that and reached out to me and started retweeting as well. A number of people sent me personal emails and messages, and I really was humbled by the response. I was quite nervous to write the article I did because in my work, there is kind of a fine line that you can only push the boundary so far, and then if you push them too far you get a lot of pushback. I sometimes feel like I'm this one little patient advocate voice among all these other voices. The article really resonated with physicians in particular, and they really understood the story which I shared about my daughter's liver doctor.

When we found out that she was diagnosed with cancer, she asked to sit in the meeting with the oncologists. She didn't have to do that, but she wanted to. I said, "Of course you can join us." She sat next to me and held my hand the entire meeting. Every time they said something distressing, she would just squeeze my hand, but she didn't utter a word. I think that encapsulates the relationship that patients and caregivers have with their physician or nurse. It's such a special bond, and the term "consumer" could never define that particular bond; she would never look at this as a transactional relationship. Would "consumer" teach her to do that? No. As you said before, it is the humanity and compassion of medicine, and that is why most everyone who comes into the profession really wants to help humanity and give that empathy and compassion.

We have a lot to learn, and it's going to take many voices to speak up and push back against consumerism because some pretty large organizations and groups are trying to push consumerism and continue this business imperative of healthcare that is motivated by bottom lines. I think we're going to get there. Maybe we're already there. We're at a tipping point. There is so much dissatisfaction, and so many parts of the system are broken. To say that consumerism will give you more choices, shorter wait times, and maybe even price transparency, even though you still have to deal with your insurance provider or lack thereof, makes me giggle because I don't think that is going to be the solution.

The only solution I see is that we have to partner with one another and co-create solutions to improve healthcare as teamsequal voices at the tableand begin this pushback. If we don't, I fear what is ahead because I've already had a glimpse of it. I'm nervous because as my daughter slowly moves into adulthood, and she is almost there, these were the things I hoped we would have conquered before she got to that point. A lot of this is pushing us away from one another and distancing ourselves from one another, and that is not going to change healthcare.

Topol: You said it so well. I've been eager to meet you since I read your work months ago, and I wanted the whole Medscape audience to get to know you and your story. Most of them are not on Twitter and most don't read Health Affairs. Your story is so darn important, and you convey it in a powerful way.

I'm so glad to know that your daughter is okay, having gone through 16 years of rough times, especially in her earlier years. I just want you to carry on. You are an important voice. You may qualify yourself as only one, but it's a powerful one, and very few people have had an experience like yours. You're in a rarified group and can transmit the emotion and the sense of caring. Let's get these words right, and let's zap "consumer" from this story of future healthcare.

Thanks so much for joining us today on Medscape, and we will look forward to following you and learning more from you in the future.

Durrah: Thank you. I look forward to partnering with your audience on improving healthcare.

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Healthcare Terms Are Out of Touch, Says Mom Advocate - Medscape

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Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Fanconi Hypoplastic Anemia Pipeline Insight Market Research Report 2019: by Trends, Development, Types,...

Bone Marrow Stem Cells Comments Off on Fanconi Anemia (FA) Fanconi Hypoplastic Anemia Fanconi Hypoplastic Anemia Pipeline Insight Market Research Report 2019: by Trends, Development, Types,… | October 21st, 2019

CALGARY, Alberta, Oct. 21, 2019 (GLOBE NEWSWIRE) -- Hemostemix Inc. (Hemostemix or the Company) (TSX VENTURE: HEM; OTCQB: HMTXF), a biotechnology company developing and commercializing blood-derived stem cell therapies for unmet medical conditions, is pleased to provide a summary of the presentation entitled Autologous Stem Cell Treatment for CLI Patients with No Revascularization Options: An Update of the Hemostemix ACP-01 Trial With 4.5 Year Followup. Lead investigator Dr. York Hsiang, Professor of Vascular Surgery, University of British Columbia gave this update at the 41st Annual Canadian Society for Vascular Surgery Meeting on September 14, 2019.

Dr. Hsiang reported on the blinded results from the long-term follow-up of the first cohort of patients enrolled at two trial sites, Vancouver Coastal Health Research Institute (VCHRI) located in Vancouver, BC, led by principal investigator, Dr. York N. Hsiang, MB, ChB, MHSc, FRCSC and University Health Network, Peter Monk Cardiac Centre located in Toronto, Ontario, led by principal investigator Dr. Thomas Lindsay, MDCM, MSc, FRCSC, FACS.

Following is a summary of the results and conclusion:

In addition, the Companys Data Safety Monitoring Board (DSMB) recently met to review patient safety data in the ongoing Phase II clinical trial for CLI. The DSMB did not find safety concerns with ACP-01 and recommended continuing to enroll patients in the trial. The clinical trial is ongoing at 13 clinical sites in the US and Canada, with several additional sites in the process of being initiated. To date, 46 of the planned 95 patients have been enrolled and treated in the study.

We are very pleased with these blinded long term follow up results, and the recommendation of the DSMB, which are consistent with the findings reported in our two previous published studies of ACP-01 in CLI patients, said Dr. Alan Jacobs, President and Chief Medical Officer of Hemostemix. Patients with critical limb ischemia face a high rate of amputation when revascularization treatment options are exhausted, so seeing this level of improvement, and outcomes maintained for up to 4.5 years after treatment, is extremely encouraging.

ABOUT HEMOSTEMIX INC.

Hemostemix is a publicly traded clinical-stage biotechnology company that develops and commercializes innovative blood-derived cell therapies for medical conditions not adequately addressed by current treatments. It is one of the first clinical-stage biotech companies to test a stem-cell therapy in an international, multicenter, Phase II clinical trial for patients with critical limb ischemia (CLI), a severe form of peripheral artery disease (PAD) caused by reduced blood flow to the legs. The Phase II trial targets a participants diseased tissue with proprietary cells grown from his or her blood that can support the formation of new blood vessels. The Companys intellectual property portfolio includes over 50 patents issued or pending throughout the world. Hemostemix has a manufacturing contract with Aspire Health Science, LLC (Aspire), for the production of ACP-01 and for research and development purposes at Aspires Orlando, Florida, facility. Building towards commercialization, Hemostemix has also licensed the use, sale and import of ACP-01 for certain indications to Aspire in certain jurisdictions. The Company is continuing research and development of its lead product, ACP-01 with other applications, including cardiovascular, neurological and vascular indications.

For more information, please visit http://www.hemostemix.comor email office@hemostemix.com.

Contact:

Kyle Makofka, CEOSuite 2150, 300 5th Avenue S.W.Calgary, Alberta T2P 3C4Phone: (403) 506-3373E-Mail: kmakofka@hemostemix.com

Neither the TSX Venture Exchange nor its Regulation Service Provider (as that term is defined under the policies of the TSX Venture Exchange) accepts responsibility for the adequacy or accuracy of this release.

Forward-Looking Statements

This release may contain forward-looking statements. Forward-looking statements are statements that are not historical facts and are generally, but not always, identified by the words expects, plans, anticipates, believes, intends, estimates, projects, potential, and similar expressions, or that events or conditions will, would, may, could, or should occur. Although Hemostemix believes the expectations expressed in such forward-looking statements are based on reasonable assumptions, such statements are not guarantees of future performance and actual results may differ materially from those in forward-looking statements. Forward-looking statements are based on the beliefs, estimates, and opinions of Hemostemix management on the date such statements were made. By their nature forward-looking statements are subject to known and unknown risks, uncertainties, and other factors which may cause actual results, events or developments to be materially different from any future results, events or developments expressed or implied by such forward-looking statements. Such factors include, but are not limited to, the Companys stage of development, future clinical trial results, long-term capital requirements and future ability to fund operations, future developments in the Companys markets and the markets in which it expects to compete, risks associated with its strategic alliances and the impact of entering new markets on the Companys operations. Each factor should be considered carefully and readers are cautioned not to place undue reliance on such forward-looking statements. Hemostemix expressly disclaims any intention or obligation to update or revise any forward-looking statements whether as a result of new information, future events, or otherwise.

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Hemostemix Announces Positive Results and Conclusions Reported in Phase II CLI Trial Abstract - GlobeNewswire

Cardiac Stem Cells Comments Off on Hemostemix Announces Positive Results and Conclusions Reported in Phase II CLI Trial Abstract – GlobeNewswire | October 21st, 2019