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GEMMA PETERS, BLOODWISE In the UK, one in 19 people will be diagnosed with blood cancer, but awareness of the symptoms is shockingly low. » p4

DR ALASDAIR RANKIN, BLOODWISE CAR-T is known as the ‘living drug’, that is bringing hope to patients with terminal cancer. » p10

DR ROY SILVERSTEIN, AMH Blood diseases can be the source of so much uncertainty for patients and their caregivers. » p10


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Senior Project Manager: Benedetta Marchesi I E-mail: benedetta.marchesi@mediaplanet.com I Business Development Manager: Roz Boldy I Content and Production Manager: Kate Jarvis Managing Director: Alex Williams I Head of Business Development: Ellie McGregor I Digital Manager: Jenny Hyndman I Designer: Kiane Blackman I Content and Social Editor: Harvey O’Donnell Mediaplanet contact information: Phone: +44 (0) 203 642 0737 E-mail: uk.info@mediaplanet.com All images supplied by Gettyimages, unless otherwise specified


HCT as a curative option for many diseases Multiple malignant and non-malignant diseases can be treated with haematopoietic cell transplantation (HCT). We must work to reduce the cost of HCT and simplify the process so we can cure more people. the donor would be HLA identical with the patient. When an identical sibling is not available, the cell could be taken from unrelated donors that can be found on international registries. However, a new and exciting way to collect stem cells comes from haploidentical donors, i.e. half identical donors. Virtually every patient has a compatible, half identical donor In international registries, more than 25 million healthy individuals have volunteered to serve as stem cell donors, but nowadays, haploidentical allogeneic HCT is becoming more popular because the biological barriers to perform it have been overcome, and v ir t ua l ly ever y patient has a donor. Many diseases, both malignant and non-ma l ig nant have been successfully treated with HCT.

There is experience in less developed countries with lowering costs of transplantation with the aim of making it more available to all patients, regardless of their socioeconomic status. This gives more patients the possibility of cure.” Allogeneic HCT is mainly used in acute leukemias and aplastic anemia, and autologous HCT is routinely performed in multiple myeloma patients all over the world. But many other diseases like lymphomas, autoimmune and benign haematological diseases could be treated with this procedure. How can we make HCT more affordable? HCT was developed in order to save many lives and has been shown to be

2019 NCRI Cancer Conference

a cost-effective therapeutic measure. Since it is not widely available to every patient who could be cured, many modifications have been made to this procedure to make it more affordable. There are exciting developments coming from India and Mexico, describing strategies for performing HCT in developing countries. There are several lessons learned: the use of affordable generic drugs whenever possible, avoiding cryopreservation of stem cells, using reduced-intensity conditioning and an outpatient based HCT. Other measures that could be useful include utilising peripheral blood as a stem cell source; limiting expensive and repetitive diagnostic studies; and avoiding excessive prophylactic blood transfusions. ISH’s mission to cure HCT patients The I nte r n at ion a l S o c ie t y of

Hematology is devoted to promoting the best available therapy to patients all over the world. But, most of all, we are interested in promoting methods that reduce the cost of procedures like HCT, in order to bring this kind of therapy to more patients in the so-called low- and middle-income countries. There is experience in less developed countries with lowering costs of transplantation with the aim of making it more available to all patients, regardless of their socioeconomic status. This gives more patients the possibility of cure.

Read more at healthawareness.co.uk










owadays, there is lot of information regarding the life-saving capacity of haematopoietic cell transplantation (HCT), also known as bone marrow transplantation. Patients worldwide have been treated with the different methods of this procedure that includes the sourcing of cells from bone marrow, peripheral blood and cord blood. The HCT is classified as autologous – in that one’s own stem cells can be removed and used for treatment – and also allogeneic – where stem cells can be provided by a donor. The goal of the former, auto-HCT, is to give the patient chemotherapy and then rescue them with their own previously saved stem cells. Allogeneic HCT, on the other hand, can have different sources of cells, depending on the match of HLA antigens. It can be obtained from a sibling donor, although, ideally

WRITTEN BY: DAVID GÓMEZ-ALMAGUER, MD, FACP Universidad Autonoma De Nuevo León, México Chair Council, International Society of Hematology


3–5 November 2019

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Helping the immune system fight blood cancer Information from Kite - a Gilead company distributed by Mediaplanet




For patients with specific blood cancers, CAR-T therapy harnesses their immune system by re-engineering their own white blood cells.


ur immune system is there to pinpoint cancer cells and kill them. It has the ability to do this all the time. The trouble is, cancer cells can go under the radar of the immune system. “Right now, somewhere in our bodies, a cell is dividing w ith incorrect genetic code,” says Hilary Hutton-Squire, General Manager of Gilead Sciences UK & Ireland; a research-based biopharmaceutical company. “Our immune system is programmed to recognise these mutated cells and destroy them before they cause a problem. Cancer can occur when these genetic mutations either go unrecognised by our immune system, or because our immune system has been overwhelmed by mutated cells in some way.”

White blood cells that are better able to fight cancer Cancer treatment has evolved tremendously over the decades, and more recently, there have been two significant medical developments: targeted therapies, which target specific cancer cell markers; and immunotherapy, which helps the immune system find and destroy cancer cells. And now, thanks to close collaboration between industry and the NHS, a therapy called CAR-T — or chimeric antigen receptor T-cell — is taking treatment a step further by harnessing the immune system to fight cancer through the genetic re-engineering of the patient’s own white blood cells. This therapy is – currently – used to treat a relatively small group of patients with specific aggressive blood

Our immune system is programmed to recognise these mutated cells and destroy them before they cause a problem.” INTERVIEW WITH:

HILARY HUTTON-SQUIRE General Manager UK & Ireland, Gilead Sciences Ltd

cancers who have not responded to two or more previous therapies and have little or no treatment options available to them. “In the case of CAR-T, white blood cells are extracted from the patient and genetically re-engineered, a process that takes a number of weeks,” says Hutton-Squire. “These white blood cells are effectively ‘taught’ how to find the cancer that was evading the patients’ immune system and fight it. The re-engineered cells are injected back into the patient to kill the specific cancer cells.” CAR-T is a one-off therapy, which can benefit certain patients with aggressive blood cancer where previously their prognosis was extremely poor. However, there are side-effects to CAR-T which can be severe and have to be carefully managed by the

treating clinicians. This type of cell therapy approach might be effective in treating a range of different stage cancers in future. “We believe that using immune cells to destroy cancer cells could have a broader application,” says HuttonSquire. “That makes it a core part of cancer research going forward.” WRITTEN BY: TONY GREENWAY

On behalf of

Zinc job bag number: YES/UK/19-08/Cl/1538 Date of preparation: August 2019

“One day, we may have the ride of your life...”


Let’s move it, move it, move it! Across the country, over 3,000 volunteers provide a rapid response courier service free of charge to over 300 NHS hospitals, laboratories and hospices. Not only do they transport red cells, platelets, FFP, pharmaceu-

tical products and faecal transplant material, they also move a variety of samples, including CSF, which often requires urgent analysis if meningitis is suspected. A lt hough t he volunteers are unpaid, they deliver to professional © PROVIDED BY NABB

standards, are GMP trained and comply with MHRA governance and audit requirements. Over five decades of service to the NHS With its roots going back over half a century to the early 60s, the blood bike ‘industry’ has seen the demand for its services more than double in the last six years and, this year, the 34 member groups of NABB (the Nationwide Association of Blood Bikes) are expected to respond to over 100,000 requests for rapid transport from their client hospitals. These groups, which are independently registered charities, provide a service at the county level, but often link up with neighbouring groups to relay samples over long distances to specialist centres for testing. On-scene support Although the majority of consignments are transported between NHS facilities, the volunteers also

support the majority of the HEMS Air Ambulance charities across the UK by ensuring their onboard blood supplies are replenished every 24 hours and any unused product is returned for use in hospital before it exceeds its storage time. One such group is the Freewheelers EVS team, operating in the south west around Bristol, Bath and Taunton. They have recently been called upon to deliver direct to scene in an RTC emergency, when particularly difficult and extended extractions have meant that the onboard supply has been depleted and back up is required to ensure the patient continues to receive that lifesaving transfusion. In 2008, this group was the first to be awarded the prestigious QAVS (the Queen’s Award for Voluntary Ser vice), which has since been awarded to five other blood bike groups across the UK. With around 140 volunteers, made up of 85 advanced riders, 35 controllers and 20+ fund raisers, they responded to


over 6,000 requests for transport last year and travelled the equivalent of seven times round the world with their fleet of 12 bikes. All of this, free of charge! If you would like to volunteer to help, you don’t have to be a biker to contribute. There are a number of different roles from driving, riding, controlling, fund raising and marketing. To find out more, visit the NABB website, or google ‘blood bikes’ and your county name.

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Public awareness is vital to beating blood cancer In the UK, one in 19 people will be diagnosed with blood cancer, but awareness of the symptoms is shockingly low.


ur research has found that only half of adults in the UK can name a single symptom of blood cancer. Blood cancer is the third biggest cancer killer in the UK, with 15,000 people losing their lives to it every year. Unlike many other cancers, it’s not possible to predict whether a person is at a higher risk of developing most types of blood cancer and you can’t currently screen for them. This makes awareness of the signs of blood cancer by both the public and medical professionals even more vital. Derek was – unknowingly – living with blood cancer Every day, we hear stories from


people who have unknowingly lived with blood cancer for months and sometimes even years before being diagnosed. One of them is Derek, a paramedic who became so ill before being diagnosed that he was rushed to hospital in his own ambulance despite having gone to t he GP severa l t i mes t r y i n g to get a d i a g no s i s for s y mptom s t hat cou ld n’t be explained. Derek is not an isolated case. His experience is backed up by the N HS’s ow n data, which shows that people with blood cancer are more likely to have to go to the GP several times before being diagnosed t h a n p e o p l e w it h a ny o t h e r common cancer.

Every day, we hear stories from people who have unknowingly lived with blood cancer for months and sometimes even years before being diagnosed. Symptoms of blood cancer can be easily confused The main challenge faced by not only the public but also GPs is that many of the symptoms of blood cancer are vague. Symptoms can include extreme tiredness, night s weat s, u nex pla i ne d br u i si ng a nd u nex pla i ne d weig ht los s.


This means there is a danger that they could be downplayed as a benign condition or put down to the ageing process, stressful lifestyles or depression, as found by a University of York study of people with lymphoma this year. Early diagnosis of blood cancer is key for saving lives The NHS Long-term Plan has made a commitment to ensure that more cancers are diagnosed earlier. We believe that increasing the number of people that recognise symptoms for blood cancer would be a major step towards better identification and swifter treatment of this terrible disease, and this, in turn, would save lives.

Bloodwise At Bloodwise, we have made raising awareness of the symptoms of blood cancer a priority. To help both the public and GPs, we have developed a simple guide (bloodwise.org. uk/symptoms) on what symptoms should be checked out by a health professional.

Read more at healthawareness.co.uk MEDIAPLANET


Shared decision-making leads to better outcomes – but people need information and support if they are to play an active role in their own care.


elivering personalised care is at the very heart of the NHS’s vision for the future – yet just 33% of people with chronic lymphocytic leukaemia (CLL) are being offered a choice of treatment. That’s according to Leukaemia Care’s latest annual patient survey, which also found just 56% of people living with the blood cancer had access to a clinical nurse specialist (CNS). Peter Wi l l ia m s, D i re c tor of AbbVie’s oncology business unit, says: “All the evidence suggests that when people are engaged in their own care, they make better decisions about managing their health. It helps them to live their life based on what matters to them.” The NHS Long-Term Plan recognises this and has stipulated that everyone diagnosed with cancer should have a personalised care plan by 2021. “What these latest figures tell us,”

says Williams, “is that, while it is a really good ambition, there’s still work to be done to support people to be fully engaged in their own care.” Shifting landscape of supplementing chemotherapy The CLL treatment landscape has changed considerably in recent years, with the traditional bedrock of chemotherapy being supplemented by immunotherapies and more targeted, personalised treatments. And the chronic nature of the cancer means someone may face CLL treatment multiple times, each time requiring a different therapeutic approach. “Each person will have different priorities,” says Williams, explaining that the short- and long-term impact of treatment varies. “Patient choice is about being able to choose a therapy that best suits your overall life goals. It’s about living better with that disease, but

Evidence suggests that, when people are engaged in their own care, they make better decisions about managing their health.” INTERVIEW WITH:

PETER WILLIAMS Business Unit Director, Oncology, AbbVie

also within the broader considerations of life. “It might be the difference between having the treatment in hospital or at home. It might be about the ability to continue working or not. Others might want to make a choice around the burden of side effects versus the efficacy of the treatment.” More CLL patients need access to a specialist nurse Empowering people to be part of their own care is about the whole system working together to provide everything they need to understand their condition and options, he says. “The CNS plays a critical role in this, so it was disappointing to see only half of the CLL patients in the survey had access to a specialist nurse. That’s a really important area that could be strengthened.” People a l so need to qu ick ly and easily access relevant and

easy-to-understand information about treatment options and disease progression, he added. “Patient organisations are in the best position to provide this information, but the rate of innovation is so fast in oncology and haematology that it can be hard for these organisations to keep up with the pace of change. “That’s where our industry has to partner better with them, so we can provide appropriate information at the right time,” says Williams. WRITTEN BY: AMANDA BARRELL Date of preparation: August 2019 UK-ONC-190026

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Read more at abbvie.co.uk

Patient groups are crucial in building an open dialogue with healthcare professionals Patient groups from around the globe come together in Edinburgh with the clinical research community to share insight on the latest advances in medicine, care and support of chronic lymphocytic leukaemia (CLL) patients.


s treatments for chronic ly mphoc y t ic leu k aem ia improve it is important that patient advocates keep pace and build capacity to aid equitable access to appropriate therapies, care and support. Why bring patient groups/ advocates together? Blood cancers are often poorly understood, and clinical and supportive care can vary dramatically between different countries and types of blood cancer. A diagnosis of chronic lymphocytic leukaemia (CLL) brings complex and varied, long-term psychosocial and physical challenges. Over the last ten years, there have been great advances and changes in how chronic lymphocytic leukaemia (CLL) is treated and managed. Recent breakthroughs are changing the CLL treatment landscape and have necessitated the updating of clinical guidelines, treatment algorithms and sequencing. MEDIAPLANET

The landscape is becoming very complicated and there is much variation of interpretation, access to medicines, care and support. It i s be com i ng i nc rea si ng ly important that patient advocates can keep pace and build capacity in their own country to best support their local patient communities, influence local policy and participate in health technology assessment processes to improve access. Bringing groups together at conferences and in a network across the globe aids with sharing knowledge and experience to advocate for equitable standards of care. Many countries do not have community or patient groups dedicated to supporting CLL patients. Healthcare professionals need open communication with patient groups Patient experiences and preferences are now becoming more complex and are evolving. Real life patient and carers’ experiences of use of

WRITTEN BY: NICK YORK UK CLL patient, Chair of CLL Advocates Network Steering Committee and Patient Advocacy Healthcare Liaison Officer at Leukaemia Care

new treatments, the positive and negative impacts they are having on quality of life, are now being reported in large numbers. Patient groups are ideally placed to collect and share information to evidence patient needs to help inform their support strategies. Bringing the clinical community together with patient groups and advocates empowers both communities to understand challenges together and develop shared initiatives to improve patient experience and outcomes. Patient groups are becoming more and more popular the world over The CLL Advocates Network (CLLAN) was founded in 2014 by representatives from Canada, Czech Republic, France, Ireland, Italy, Portugal, UK and the United States. There are now over 30 groups from different countries forming the network. The network seeks to enhance CLL patient care and improve outcomes

by collaborating with patient groups and advocates supporting CLL. The network seeks to improve access to novel treatments globally and to empower patients to become advocates to support local CLL communities. The network does this by sharing best practices and resources between patient groups through annual meetings and a website. CLL Horizons Edinburgh 2019 19-21 September The CLL Advocates Network (CLLAN) held its first Two CLL Horizons: Learn. Share. Grow Conferences in Belgrade, Serbia in 2016 and Prague, Czech Republic in 2018. This year, they are expanding on the success of these events. The 3rd CLL Horizons Conference 2019 is being held in Edinburgh to bring together 50 patient advocates and patient groups with a focus on supporting CLL patients and their families. Advocates will join with the international clinical research

community to encourage greater collaboration, share best practices and expert knowledge that will help support the needs of patients in different countries. T h i s y e a r ’s C L L H o r i z o n s Conference overlaps with the international Workshop on CLL (iwCLL) in Edinburgh and reports on the value and progress of collaboration between both communities. As well as exploring the latest clin ica l developments and knowledge, conference sessions explore how to involve patient advocates and patient groups in healthcare initiatives.

Read more at healthawareness.co.uk HEALTHAWARENESS.CO.UK



It’s an exciting time for the haemophilia community

After years of inaction, significant breakthroughs have been made in the field of haemophilia treatment, which offer hope to patients. It’s an exciting time for the haemophilia community.


or years, treatment for haemophilia has been frustratingly limited, says Brian O'Mahony, President of t he European Haemophilia Consortium (EHC), an international non-profit organisation that represents 46 national patients' organisations of people with rare bleeding disorders. But, scientific breakthroughs over the last five years have made the haemophilia therapy landscape look vastly different, giving patients and healthcare professionals cause for optimism. “In haemophilia, coagulation factors in the blood are either absent or present in lower amounts, which stops the blood from clotting,” explains O'Mahony. “ The most common type of haemophilia is A, where the missing coagulation factor is VIII (8). In haemophilia B, the missing coagulation factor is IX (9). Replacement therapy — i.e., replacing the missing or deficient coagulation factor — has been the standard treatment for years.” New clotting factor therapies last longer in the patient In the mid-1990s, plasma-derived replacement therapy made from


pooled human plasma, and recombinant (or synthetic) clotting factor therapies produced in live cells were introduced. But those were the last big innovations in haemophilia treatment for the best part of 20 years. Thankfully, in 2014, a new range of recombinant concentrates with an extended half-life were licensed ('half-life' refers to the amount of time the body takes to reduce the amount of clotting factor to half in the bloodstream). These last longer in the blood and therefore offer more protection from bleeding. Preventing patients’ antibodies fighting medication There have also been significant d e ve lo p me nt s i n mono c lon a l antibody technology for patients with factor VIII inhibitors (where the body recognises replacement treatment as a foreign protein, produces antibodies to fight it and stops it from working) and factor VIII deficiency. “ T h at 's b e e n a n e x t r e m e l y interesting development,” says O'Mahony. “A Japanese company h a s d e ve l o p e d a m o n o c l o n a l antibody that mimics what factor


BRIAN O’MAHONY President, European Haemophilia Consortium VIII does, with impressive results. Interestingly, this treatment is given subcutaneously, which frees people from constant intravenous injections.” Gene therapy could be a game-changer Then there's gene therapy, which is closer than ever to becoming a reality. “We've been talking about the possibility of gene therapy in haemophilia for 20 years,” says O'Mahony. “It's always been 'on the horizon' but never quite in reach. But, now, gene therapy trials for both factor

VIII and factor IX deficiency are advancing at a pace — and cases have been recorded of patients reaching normal factor levels after a single infusion.” It's still unclear how long the beneficial effects of gene therapy could last, however, and there are drawbacks to overcome. F o r e x a mple , c u r r e nt g e n e therapy models aren't yet suitable for children. Plus, because patients develop antibodies to fight it, the same treatment can't be given again should it stop working. “More research is needed to solve these issues,” says O'Mahony. “But at least we've gone beyond asking: 'Will we ever have gene therapy for haemophilia treatment?' It's just a question of 'when' now. I believe it will probably be licensed within two years. Overall, we've seen more changes in haemophilia therapy in the last five years than in the previous two decades. And I think the changes we'll see in the next five years will make the last five look slow.” Better procurement with involvement of patient groups Unfortunately, the global picture

is that 70% of people with haemophilia have limited or no access to new or current therapies. This must change, O’Mahony insists. Countries — especially developing and emerging countries — need proper procurement systems in place to ensure new and current therapies are available to those who need them at cost-effective prices. They also need to allow clinical experts and patient organisations (rather than just government officials and accountants) to be involved in the procurement process. “One of my major hopes is that some of these new therapies will be priced at a point where they will be more accessible to people,” says O'Mahony. “We need to make sure t hat pressure is put on the pharma companies producing novel and gene t herapies to ma ke t hem affordable. After all, there's no point in them developing a really good new treatment if most patients aren't able to access it.” WRITTEN BY: TONY GREENWAY Read more at healthawareness.co.uk MEDIAPLANET


living life beyond haemophilia

A life-changing vision for today’s haemophilia care INTERVIEW WITH: PHILIP WOOD Head of Haemophilia, Sobi


ae mo ph i l i a i s a blo o d clotting disorder. When a person living with haemophilia bleeds, it takes longer than normal for the bleeding to stop. Those with severe haemophilia can bleed frequently and most of the time for no apparent reason. The consequences of even a minor accident can be serious, so caution is a constant state of mind. B u t P h i l i p Wo o d , H e a d o f Haemophilia at Sobi — a specialised international biopharmaceutical company — insists that the conversation around the treatment of haemophilia must be changed. People living with the condition deserve to live their lives to the full. People with haemophilia deserve to feel safe Philip Wood, believes that, just like anybody else, people with h aemoph i l i a shou ld h ave t he

right to pursue the opportunities they desire. He believes that their treatment should allow them to feel safe, protect them from bleeds, have long-term joint health protection, and generally unburden them from their haemophilia. Haemophilia can make daily activities a constant source of worry In 2018, Sobi conducted a groundbreaking, large-scale, pan-European ethnographic study of 51 people living with haemophilia, which exposed the unique challenges, aspirations and unmet needs, behaviours and perceptions related to life with haemophilia. The research revealed that most people with the condition live a far from normal life. Some face many medical challenges in their everyday lives and often act cautiously. They also make trade-offs on activity levels, fearful that bleeds will cause joint damage as they grow older. But it doesn’t have to be this way. “We truly believe in the potential of the people we are dedicated to

We truly believe in the potential of the people we are dedicated to serving, so we have developed Liberate Life, a vision in partnership with everyone living with haemophilia, their family members and friends, and the professionals who care for them.” ser ving, so we have developed Liberate Life, a vision in partnership with everyone living with haemophilia, their family members and friends, and the professionals who care for them.” The vision celebrates real-life liberation stories of individuals living with haemophilia on their own personal journeys, letting their dreams and drive dictate what is possible.” “By encouraging and motivating the haemophilia community – across the globe – to expect more from life and reach for the many possibilities that advances in care have made possible, we believe that, together, we can challenge the © UNSPLASH / FABRIZIO-VERRECCHIA

status quo of today’s haemophilia care,” says Philip Wood. Personalised protection has the potential to change many lives With today’s therapies, treatment can be personalised to provide the level of protection that a person requires throughout his or her different life stages and endeavours. Treatment can even manage potential clinical situations, such as surgery, that might arise during life. To fully personalise treatment plans by tailoring doses and treatments to an individual’s needs and wishes about how they want to live their life, is a life-affirming and life-changing paradigm shift in haemophilia care. Erik’s haemophilia A often made him feel left out Take Erik, for example, who has severe haemophilia A and, as a child, lived a different life to his friends. While he wanted to do everything that other people did, Erik was often left out if the risks were thought to be too great. “I sometimes felt neglected when I couldn’t participate,” he says. Now, however, Erik has embraced the Liberate Life vision and is trek king Kebnekaise, Sweden’s highest mountain. “It’s important for me to show, not only to myself but to others, that even though I have severe haemophilia A, I can carry out the same challenges as everyone else,” he says. Haemophilia does not have to define who you are Of course, Liberate Life means different things to different people. Not everyone with haemophilia wants to climb a mountain. They might decide to travel, learn a musical instrument, take dance classes, change their careers or aim to go for a walk more often. They can even find liberation by talking about


their haemophilia to others. But whatever they decide to do, people living with the condition shou ld have t he r ight to t ur n their dreams and ambitions into reality — and to live their lives beyond haemophilia. WRITTEN BY: TONY GREENWAY

MEET ERIK Liberator with Haemophilia A

Sobi At Sobi, we are transforming the lives of people affected by rare diseases. We have a wealth of experience in haemophilia and are dedicated to positive change and long-term commitment to transforming the lives of people affected by it. Liberate Life builds on our dedication to opening up opportunities for people living with haemophilia; to celebrate life without compromise.

Sponsored by


Read more at liberatelife.eu HEALTHAWARENESS.CO.UK



Improving care for people with haemophilia globally The World Federation of Hemophilia has been improving care for people with bleeding disorders around the world for the past 50 years. Treatment, care and support for people with bleeding disorders has improved dramatically over this time. Despite improvements, access to quality care continues to be a challenge for many patients around the world. We continue to work to overcome these challenges and look optimistically to the future as we enter a new era of treatments. How has the landscape of haemophilia changed over the last 20 years in terms of diagnosis and treatment? Identification and diagnosis of patients with all bleeding disorders has improved dramatically over the past 20 years. In 1999, approximately 75,000 people with haemophilia (PWH) were identified. Today, this number has more than doubled, with almost 200,000 PWH identified. Although these are impressive gains, this number represents only 40% of the expected number of PWH, demonstrating the work that still needs to be done. A long w it h t he i nc rea se i n number of PWH identified, the global

treatment of PWH has also improved over the past 20 years. Consumption of FVIII, the main treatment for haemophilia, has increased globally over time. However, these improvements are not equally distributed around the world. Wh i le c on s u mp t ion a mon g upper-income countries has more than doubled, now exceeding 7 IU/ capita, countries in the low-income group are trailing far behind, with an average of 0.105 IU/capita. If you consider the latest recommendation of the Council of Europe that 4 IU/capita should be the minimum in any given country, you realise how dramatic the situation is for patients living in low and middle income countries.

The disproportionate distribution of treatment globally is best demonstrated in Figure 1, which shows that 20% of all PWH (those living in high-income countries), use 70% of the available treatment worldwide, leaving only 30% of the worldwide supply of treatment for the majority of PWH (those living in lower-income countries). What are the key challenges in diagnosing haemophilia? Identification and diagnosis have reached a satisfactory level in North America, Western Europe, Japan and Australasia, but that is far from being the case in other parts of the world. We know, based on data from the WFH Annual Global Survey, where

Figure 1

GLOBAL ACCESS TO CARE Population distribution

Estimated FVIII use

20% of the world uses 70% of available FVIII

Data Source: WFH Annual Global Survey 2017


WRITTEN BY: ALAIN WEILL President, World Federation of Hemophilia

countries report their number of identified patients, and in the known incidence of haemophilia (1 in 5,000 live male births), the number of identified patients represents only 40% of the expected patients globally. If we break this down based on income level of the country, a mere 6% of patients in the lowest-income countries have been identified, while close to 100% of those in high-income countries have been identified. The two main reasons for the low levels of diagnosis are lack of education and laboratory testing capabilities. The WFH is working hard to increase the knowledge of haemophilia and the ability to make correct diagnoses, by providing training around the world. How accessible are treatments to patients? Are current treatments available to everyone? There is an imbalance in the distribution of clotting factor consumption between geographical regions of the world. Access and affordability of care is a real challenge for haemophilia patients in most parts of the world. Too often, haemophilia is totally absent from ministry of health budgets. Also, the cost of treatment is so high that low- and middle-income countries can hardly afford purchasing and procuring treatments for patients. The World F e d e r at ion of He m o ph i l i a h a s d e ve lo p e d a Hu m a n i t a r i a n A i d p r o g r a m . Thanks to predictable and sustainable donations from some pharma-

ceutical companies, we have been able to help patients living in poor countries perform orthopaedic surgeries and initiate prophylaxis to young children. What does the future hold for haemophilia? What kind of game-changer could or should be on the horizon for haemophilia? Scientists and researchers have made fantastic progress in terms of product quality and safety and, very soon, gene therapy is expected to be licensed and accessible to some patients. Today, a person with haemophilia can enjoy a quality of life very similar to the average population, can get access to education and can have a similar professional and family life if properly diagnosed and taken care of in his very early age. The future could be bright if: a. All governments and especially those from low- and medium-income countries include haemophilia into their health priorities, invest in education, identification and diagnosis b. Pharmaceutical companies significantly increase their production of clotting factors and gene therapy is being proposed to a maximum number of patients at reasonably affordable cost to all. Read more at healthawareness.co.uk



Bringing biotechnology to Italy - an essential step to providing treatments for rare diseases The importance of the biotech industry to the growth and competitiveness of the European pharmaceutical sector is fundamental. Today, the majority of innovative medicines – as well as many diagnostic products – are developed or manufactured using biotechnology.


lobally, biotech medicines represent 20% of commercialised drugs, 40% of authorised drugs and 50% of drugs under development. These medicines are particularly needed for patients with rare diseases, where the principles of large pharma are rarely relevant. Italy’s biotech landscape suffers from limited investment from the mainstream sectors, due to inadequate funding and challenges in the transfer of technologies. With 110 biotech drugs available for treating a range of disorders, Italy’s biotech industry nevertheless lags behind in Europe; in a Biotechnolog y Innovation Scorecard, Italy scored 28.7/100, placing it 38th of the 54 countries studied. However, the situation is starting to change with coordinated efforts in research, finance and organisation. In recent years, the biotech industry in Italy has developed due to excellent academic and industrial research as well as the value of the technology and products developed. • Italian biotech companies totalled 571 in 2017 • Italian biotech turnover is over $12.2 billion (increasing by 12% between 2014 and 2016) MEDIAPLANET

• Investments in R&D amounted to $2 billion between 2014 and 2016 • There are about 3,790 employees in biotech research. Biotechnology is among the sectors utilising the higher level of innovation in Italy. In particular, smaller companies are driving innovation, addressing the challenges posed by limited funding and technology transfer through building skilled scientific and commercial teams, developing intellectual property and entering into partnerships with likeminded organisations. A network with room for improvement Furthermore, technology transfer infrastructure in Italy is still very underdeveloped, and commercialising technologies from institutions can be a difficult process, unless adapted to the particular needs of rare disease therapies. Moreover, with few exceptions, the local biotech community is still poorly networked; scientific groups tend to work in isolation rather than integrating with the international biotech community. However, new events, awards

Biotechnology is among the sectors utilising the higher level of innovation in Italy.” and accelerator programmes, like BioInItaly, Meet in Italy for Life Sciences and Bioupper, are taking off. Banks, venture capitalists and even the Government are also now more willing to invest in Italy’s biotech industry, due to the evidence that the innovative approach of small companies is paying off. Supporting those who need it most Infrastructure, funding and collaboration are persistent challenges for the biotech industry. Despite this, in the least economically advantaged parts of the country, biotech companies are focusing on the provision of therapies that are otherwise inaccessible. “Pharma philosophy should be about creating a balance between social engagement and financial necessity. For example, it may be difficult to get medication to a patient in a particular country, but a pharmaceutical company should deliver in such a situation. Even if the cost of delivery is more than the cost of the

WRITTEN BY:: ALBERT FARRUGIA BioVIIIx drugs, it is the responsibility of such large organisations to ensure that the patient is taken care of,” says Dr Rosiello, founder of biotech and pharmaceutical company, BioVIIIx, who specialise in bleeding disorders and rare diseases, especially haemophilia. There has been marked commitment from one firm in Naples to haemophilia and related bleeding disorders to deliver products overlooked by the mainstream providers. Steady growth has also stimulated the formation of a new division based on the development of orphan medicines for rare diseases. Rare disease research and development needs a strong internal core of commercial and scientific expertise committed to innovation. An ethos of openness to the establishment of relationships with a number of innovative, local biotech companies and international startups can enable the generation of strong intellectual property addressing substantial unmet medical need in the areas of oncology and medical information technology. Dr Rosiello says: “In order to protec t pat ients, doc tors, colleagues, suppliers and stakeholders in general, it is essential to keep to a set of principles that govern every day of our lives, whatever our role

in the company, and irrespective of the mandatory requirements of the governing bodies.” BIOVIIIx operates under the philosophy of “having a multiplicity of arrows in our quiver, in order to maximise the opportunity of hitting our target!” BIOVIIIx’s success has been recognised through the recent award for Excellence in Innovation & Leadership in Pharma and Biotech Year in 2018 by the LeFonti awards, which is one of the world’s leading ceremonies of business awards and personal honours support services. Overall, BIOVIIIx’s experience serves as a useful case study of the Italian biotech industry, and a possible microcosm for its future success.

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Conquer blood diseases WRITTEN BY: DR ROY SILVERSTEIN President, American Society of Hematology

The future of blood cancer treatment is now WRITTEN BY: DR ALASDAIR RANKIN Director of Policy, Research and Support, Bloodwise

Known as the ‘living drug’ that brings hope to patients with terminal cancer, CAR-T therapy is the most exciting development in cancer treatment in decades.


he development of CAR-T began in the late 1970s, but it is only now, almost 40 years later, that it is becoming available in treatment. The t herapy t a kes it s na me from white blood cells, called T cells, which are harvested from the patient and reprogrammed to recognise specific cancer cells. Once the modified cells are put back into the patient, they hunt down and kill the cancer cells. NHS patients first treated with CAR-T in 2019 This year saw the first children and adults with blood cancer treated with CAR-T on the NHS in England. Among them was Mike Simpson, from Durham, who was diagnosed with an aggressive blood cancer in 2015. Despite two gruelling bouts of chemotherapy, the cancer returned and, by the end of last year, he was told he had little time left. But after starting treatment with this revolutionary new therapy in February, Mr Simpson is already back home, telling the media that he is looking forward to the future with hope. Early availability to CAR-T therapy is limited Sad ly, C A R-T doesn’t work for everyone who receives it. People can experience serious and potentially fatal side effects, which is one reason why this treatment is currently only available at hospitals with the expertise and facilities to manage them. Mr Simpson became unwell a few days after the infusion and spent almost a week in intensive care.


Happily, this is an issue that is being addressed by ongoing research. Although it is already making an impact, CAR-T is still very new, which means that, currently, only a few hundred patients will benefit in the next two or three years. CAR-T licensed to treat a small number of cancers There are more than 100 types of blood cancer, but CAR-T is currently licensed to treat just three types: childhood acute lymphoblastic leukaemia, diffuse large B-cell lymphoma and primary mediastinal large B-cell lymphoma. And CAR-T is only available for the minority of people who don’t respond well to existing proven treatments. However, with some 20 clinical trials and research studies for promising new CAR-T therapies in the UK alone, some of which are funded by Bloodwise, it is likely that we are only beginning to see the benefits that CAR-T therapy can bring to a growing range of blood cancers, and perhaps to some other types of cancer. Mr Simpson is enjoying spending time with his young grandchildren and is even considering a return to work. “I’m incredibly grateful for being given the opportunity to have this therapy, as I know it’s a costly, one-time treatment. I describe it as my L’Oreal treatment… because I’m worth it,” he joked.

Read more at healthawareness.co.uk

From life-long blood disorders, like sickle cell disease and haemophilia, to sudden, life-altering blood cancer diagnoses like leukemia or multiple myeloma, blood diseases can be the source of so much uncertainty for patients and their caregivers.


ortunately, there is much to be hopeful for in the understanding and treatment of these disorders. The American Society of Hematology (ASH) is working with scientists, research institutions, pharmaceutical companies and policymakers to accelerate scientific discovery, drug development and deployment of new therapies to conquer blood diseases. Harnessing our immune system to defeat cancer In 2017, a revolutionary therapy made the leap from the research bench to the bedside when the US Food and Drug Administration (FDA) approved a groundbreaking CAR T-cell therapy to treat certain leukemia patients. In a process that once would have sounded like science fiction, this therapy involves taking a person’s immune cells from their bone marrow, reprogramming them to target their cancer, and returning them to the patient. CAR T-cell therapy is a prime example of precision medicine’s potential to revolutionise the care we deliver to our patients. CAR T-cell therapy has also been approved for

Almost every day, clinical trials are reporting further success in treating patients with other types of blood cancers.” some patients with another blood cancer called lymphoma. Almost every day, clinical trials are reporting further success in treating patients with other types of blood cancers, such as multiple myeloma, using CAR T-cells. ASH is committed to improving the safety, effectiveness, and availability of revolutionary cancer therapies, and we are actively promoting research to hasten their delivery. Sickle cell disease: from the first discovered to the first conquered molecular disease Sick le cel l disease (SCD) is an inherited blood disorder that affects nearly 100,000 Americans, primarily of African, Mediterranean, and Middle Eastern descent. SCD causes red blood cells to become rigid and sickle-shaped, leading to reduced oxygen flow to almost every organ, causing crises of severe pain, stroke, organ damage, and even death. © JOCHEN SANDS

While treatment options are currently limited for those with SCD, several research teams from around the globe are using the latest advancements in precision medicine to make cures in SCD possible in the near future. This includes calling on gene therapies and genome editing techniques to correct the genes responsible for this disease. While it’s still too early to deliver many of these therapies and cures to people, scientists are hard at work making them a reality. Today, tomorrow, and beyond In the 1960s, the first successful trials in chemotherapy were reported in people with leukemia. Today, with scientific breakthroughs in precision medicine, haematologists are mapping the frontiers of medicine, and ASH is honored to play a key role in fostering this groundbreaking work. Every day, our 17,000+ members are hard at work to improve the lives of millions of patients around the world. Get involved Perhaps you or a loved one has been affected by a blood disorder, and you would like to support new research that will contribute to novel treatments for the disease. By becoming an advocate for haematology, you can help increase public awareness about blood disorders and support state and federal funding for research, which is critically needed to make this exciting science count for patients. To learn more about blood health, the importance of funding biomedical research, and how you can get involved, please visit hematology.org/patients

If you would like to donate to our efforts to conquer blood diseases, please visit

hematology.org/foundation Read more at healthawareness.co.uk MEDIAPLANET


Removing the toxicity from bone marrow transplant conditioning

A new method of non-toxic bone marrow transplant conditioning promises to transform the treatment of haematological cancers and autoimmune diseases.


lood cancers affect more than 1.1 million people annually in the United States alone. More than 150,000 new cases are expected to be diagnosed this year. Unfortunately, some blood cancers, such as acute myeloid leukemia (AML), are extremely difficult to treat. In fact, first-round treatments fail in about 70% of all AML cases, and a bone marrow/haematopoietic stem cell transplant (BMT) is the only remaining option. Yet many people can’t tolerate the BMT conditioning process, which carries a high level of toxicity and danger, and are therefore not able to obtain a BMT. However, newly developed antibodies, currently in pre-clinical trials, could offer a solution — and new hope for blood cancer patients. Traditional BMT involves a toxic procedure For nearly half a century, bone marrow transplants have been demonstrated to successfully treat blood cancers and prolong life. “For many people with leukemia, lymphoma or multiple myeloma, who have failed front line therapy, a BMT is their only viable option,” says Vladislav Sandler, Co-founder and CEO of Hemogenyx, a Brooklyn-based biotechnology company listed on the London Stock Exchange (LSE:HEMO). MEDIAPLANET

Nevertheless, cautions Sandler: “It’s a very difficult procedure that often fails.” That’s because preparing the patient for the procedure involves first administering maximally tolerated doses of a cocktail of chemotherapeutic agents with or without radiation. These preparative regimes are toxic and can have severe side effects including radiation damage to the heart or lungs, thyroid problems, fertility problems, bone damage, and development of other cancers years later. It is also difficult to eliminate cancer cells that have possibly already developed resistance to drugs during the initial treatment. “In one sense, you could say that conditioning nearly kills the patient before the BMT tries to save them,” says Sandler. Indeed, between 2-4% of people who undergo a BMT die during the conditioning stage. ‘A walking skeleton’ Sandler knows about the horrors of BMT conditioning first-hand. When Sandler’s father was 63 years old, he was diagnosed with lymphoma. His doctors started him on an aggressive round of chemotherapy combined with the first generation of immunotherapy and radiation. Almost immediately, he relapsed. That’s when he began the preparation

Many people can’t tolerate the BMT conditioning process, which carries a high level of toxicity and danger.” INTERVIEW WITH:

VLADISLAV SANDLER PH.D. Co-founder and CEO, HemoGenyx LLC treatment for a BMT. “He was tortured by the first chemo. Then he was tortured again,” says Sandler. “He was like a walking skeleton.” Unfortunately, the conditioning failed to kill the lymphoma. The transplant ultimately failed, and just seven months after his initial diagnosis, Sandler’s father passed away. A ‘transformative’ approach Having witnessed his father suffer in his final year of life, Sandler was

inspired to found Hemogenyx, which is developing an antibody, codenamed CDX, that has the potential to completely reinvent BMT conditioning as well as the treatment of relapsed/refractory AML, increasing the chances of patient survival. CDX is a bi-specific antibody that works by instructing the patient’s own T-cells to rid the body of unwanted cells, eliminating the need for chemotherapy or radiation and their toxic side effects. “People won’t lose their hair, their bowels won’t be destroyed, and young women won’t have to freeze their eggs over fertility concerns,” says Sandler. “The difference, potentially, is absolutely transformative.” Moreover, this approach could make BMTs available to a range of previously ineligible patients, such as those considered too old or weak to contend with the toxicity of conditioning. Potential for autoimmune diseases I n ad d it ion t o t r a n s for m i n g the treatment of blood cancers, Hemogenyx’s antibodies could expand the use of BMT treatments for patients suffering from severe autoimmune diseases such as lupus, multiple sclerosis and possibly others. Evidence points to the efficacy of BMT as a treatment for these diseases,

but typically these patients aren’t candidates for BMTs because the chemotherapy and radiation that are currently part of the conditioning stage are too dangerous and the risk/ reward profile of the treatment is unfavourable. Hemogenyx recently created a first generation, humanised mouse-based model of human lupus, which the company is using to better understand the disease. Sandler says two new treatment approaches have been identified. However, he adds, Hemogenyx’s research is “still in the early stages.” In the meantime, Hemogenyx continues to race toward full development of its CDX antibodies, with a view toward expanding the use of bone marrow transplants in the treatment of blood diseases and other disorders. “It’s a very exciting time in science and research,” says Sandler. “Every day we’re opening up new opportunities for the future.” WRITTEN BY : LOWELL GOODMAN

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Sepsis: The biggest killer WRITTEN BY: you’ve never heard of DR RON DANIELS BEM

CEO, UK Sepsis Trust

Sepsis affects more than 250,000 people in the UK every year, claiming 52,000 of those lives. But how much do people know about sepsis, or about its long-lasting consequences?

Giving clinicians the confidence to act – in under 3 hours WRITTEN BY: DR HELEN V BENNETT Market Development Manager, Momentum Bioscience LTD

Introducing a new class of product, in which all microorganisms in a whole blood sample can be captured and tested, allowing ultrasensitive detection without hours of culturing.


e te c t ion of p at ho g e n s causing bloodstream infection (BSI) is critical to improving patient survival from sepsis and impacting antimicrobial stewardship. C l i n ic ia n s t reat i ng pat ient s suspected of sepsis simply do not get reliable information fast enough and remain reliant on culture-based methods. The ideal is a universal, rapid, more reliable result that allows clinicians to have more confidence in their initial empirical therapy, add or adjust antimicrobials earlier and monitor the patient’s progress. Is it sepsis or not? Our recently automated ETGA® test, Magnitor, was piloted in studies at two different US sites during 2018. From 264 patients, 231 had negative blood cultures after 16-20 hours and gave valid Magnitor results for each of two blood bottles per patient. When comparing Magnitor to blood culture for individual bottles, the NPV was 98.6%. The further identification aspect of the test correctly identified five of the six ETGA positives, providing no result for one patient. This was excellent performance that generated data suppor ting t he efficacy of t he Magnitor test. Viewed as the precursor to a direct-from-blood test for positive detec t ion, t he resu lt s showed prom i se b ot h i n deter m i n i ng viability and further identification. Saving time saves lives Our new direct-from-blood test has been designed to capture microorganisms from 5mL of whole blood and concentrate them into a 0.1mL volume. MagRapid can universally identify the presence or absence of a viable bacterial or fungal BSI,


followed by the categorisation of the causative organisms as Gram positive, Gram negative or yeast (Candida) – or combination thereof. All in less than three hours. With further delineation being developed, this rapid molecular identification enables earlier change of therapy; a switch from IV to oral, de-escalation or a switch to antifungals. It could also enable faster focus of antibiotics in unknown BSI cases.


epsis is the body’s overreaction to an infection where, instead of fighting an infection, the immune system attacks the body’s own organs and tissues. Sepsis can occur as a result of any infection, from a small cut or insect bite to a chest infection or UTI. It’s more common than heart attacks and kills more people than bowel, breast and prostate cancer and road accidents combined. Current practice for diagnosis and treatment could be costing the UK economy up to £15.6 billion annually. Despite these staggering figures, awareness of the condition is still far too low and fewer still know about the lasting after-effects of sepsis, including post-sepsis syndrome. What is post-sepsis syndrome? Sepsis is a life-threatening condition that can take a good deal of time for your body and mind to recover from. Some sepsis survivors experience a variety of physical, psychological and emotional problems while recovering. This is known as PostSepsis Syndrome (PSS) and usually lasts between six and 18 months,

somet imes longer. Post-sepsis syndrome is very common in sepsis survivors. Symptoms of PSS There are a variety of physical symptoms of PSS, the most common of which are: • Lethargy/excessive tiredness • Poor mobility/muscle weakness • Breathlessness/chest pains • Joint and muscle pains • Insomnia • Hair loss • Repeated infections from the original site or a new infection There are also a number of psychological or emotional symptoms of PSS, including: • Anxiety/fear of sepsis recurring • Depression • Flashbacks • Nightmares • Insomnia (due to stress or anxiety) • Post-traumatic stress disorder • Poor concentration • Short-term memory loss

• Mood swings Recovering from sepsis takes longer than you might think There is no specific treatment for PSS, but most people will get better with time. In the meantime, it’s a case of managing the individual problems and looking after yourself while you are recovering. Some survivors find that their immune system is not as effective in the year following their sepsis. As a result, they get one infection after another, whether it’s coughs and colds, repeated water infections or a recurring wound infection. Awareness of sepsis has increased greatly over the last few years, but awareness of the long-lasting after-effects are still known by too few. For sepsis survivors, the median time taken to return to work is six months, and so the next step is to make people aware of the fact that recovery after sepsis can take time, and to raise awareness of post-sepsis syndrome. Read more at healthawareness.co.uk

Revolutionising sepsis diagnostics MagRapid is in a prototype format and will be used to test clinical samples in a multi-site study commencing in January 2020. Sites in both the US and UK will generate data to investigate performance characteristics in clinical situations, supporting further development of the MagRapid test. The output from this test has the capability of enabling other tests to be performed sooner and can be used in downstream applications such as Antimicrobial Susceptibility Testing and Next Generation Sequencing. MagRapid could have a positive impact on antibiotic stewardship, patient management and hospital care costs. MagRapid is designed to run on already established clinical laboratory instruments and Momentum is seeking partners for joint-commercialisation.

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Blood Health - Q3 - Aug 2019