Thalassemia Treatment Market With Top Countries Data : Analysis and by Recent Trends and Regional Growth Overview Forecast 2026 – News Description

Transparency Market Research (TMR)has published a new report titled, Thalassemia Treatment Market Global Industry Analysis, Size, Share, Growth, Trends, and Forecast, 20182026.According to the report, theglobal thalassemia treatment marketwas valued at US$ 842.0 Mn in 2017 and is projected to expand at a CAGR of 7.9% from 2018 to 2026. Increase in R&D investment by key players for developing new drugs for treating thalassemia and rise in demand for chelating therapy are anticipated to fuel the growth of the global market from 2018 to 2026. Asia Pacific and Middle East & Africa are expected to dominate the global market owing to increase in prevalence of thalassemia disorder and high adoption of chelation therapy & blood transfusion for treatment by doctors as well as patients. The market in Asia Pacific is projected to expand at the fastest CAGR during the forecast period. Growth of the market in the region is attributed to large base of private clinics and hospitals, rise in number of thalassemia population requiring chelation therapy services after spleen surgery, and surge in adoption of blood transfusion among patients. The thalassemia treatment market in Latin America is likely to expand at a moderate growth rate during the forecast period.

Value Added Features in Thalassemia Treatments to Propel Global Market

The global thalassemia treatment market is projected to be driven by value added features offered by various thalassemia drug manufacturing companies in order to streamline the day to day work flow and increase revenue. The thalassemia treatment provides limited range of features and benefits ranging from patient pain heeling remedies to treatment procedures. For instance, very less number of people go for the much beneficial chelation therapy. These features help physicians and nurses to streamline the chelation therapy required for patients to maintain their daily workflow efficiently and effectively. Key players offering thalassemia treatment are coming up with value added features such as bone marrow transplantation, stem cell regeneration, gene editing methodologies, and effective modality features used for drug manufacturing along with creating a prominent candidate molecule for drugs. These features can reduce the overall operating cost and improve the overall effectiveness and efficiency of treatment practices. Companies are focusing on the development of combined drug therapy in their system to effectively integrate chelating therapy or other treatment procedure at an affordable cost. These value added features save time for physicians and help improve thalassemia patient survival performance.

Chelation Therapy to be Highly Lucrative Segment

Traditionally, blood transfusion based on type of thalassemia treatment was the most commonly used procedure among thalassemia patients. This treatment type was associated with availability of donor and cost of treatment procedure. Moreover, chelation therapy based on thalassemia treatment are priced on perpetual license model and are expensive. Chelation therapy treatment enables patients to practice intensive therapy to treat acute iron overload leading to 90% recovery among thalassemia patients. These chelation therapy based treatments address specific challenges faced during the treatment procedure. The chelation therapy treatment facilitates benefits such as pain relief, and increase in motion of blood flow among patients.

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Asia Pacific Presents Significant Opportunities

North America and Europe accounted for major share of the global thalassemia treatment market in 2017 and are likely to gain market shares by 2026. High rate of immigration from tropical regions, increasing health care budgets by governments, and government initiatives to promote thalassemia treatment technique contributed to the leading share of these regions. Asia Pacific is projected to be the most attractive market for thalassemia treatment, with highest attractiveness index. The market in Asia Pacific is expected to expand at a high CAGR of 9% during the forecast period due to large number of thalassemia patients opting for chelation therapy in developing countries such as India and China. Well-established health care facilities, medical tourism for treatment of thalassemia, and high adoption of blood transfusion safety technique in countries such as Turkey and GCC Countries are likely to drive the market in Middle East & Africa. The market in Latin America is poised to expand at a moderate growth rate during the forecast period.

Trend of R&D among Leading Players to Increase Geographic Presence

The report also provides profiles of leading players operating in the global thalassemia treatment market. bluebird bio, Inc., Acceleron Pharma, Inc., Novartis AG, Celgene Corporation, and Shire plc (Takeda Pharmaceuticals) are the leading players operating in the global market. Companies operating in the thalassemia treatment market aim to increase geographic presence and research & development through strategic acquisitions and collaborations with leading players in respective domains and region. In December 2017, Shire plc committed to pay approximately US$ 1,409.9 Mn to contract vendors for administering and executing clinical trials. Other prominent players operating in the global thalassemia treatment include Incyte Corporation, Kiadis Pharma, Gamida Cell, Celgene Corporation, and Bellicum Pharmaceuticals.

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Thalassemia Treatment Market With Top Countries Data : Analysis and by Recent Trends and Regional Growth Overview Forecast 2026 - News Description

The ART of Having Children – Vision Insights and New Horizons

Im looking for a sperm donor, she said.

So begins Robert Klitzmans new book. If youre imagining that Klitzman is a provocative new writer of edgy cosmopolitan fiction, guess again. In Designing Babies: How Technology Is Changing the Ways We Create Children (2020), the medical doctor explores the many ways in which babies are created today. These ARTs (assisted reproductive technologies) may seem like fiction, but theyre not. Increasing numbers of hopeful parents are investing in ART procedures.

In vitro fertilization (or IVF, the bringing together of sperm and egg outside the body) is the core ART process. Since Louise Browns birth in 1978, IVF has added more than 8 million babies to both traditional and nontraditional families. But many millions more embryos and pregnancies have been unsuccessful. The grief, frustration and financial costs can be overwhelming; even the relationship between potential parents can be ruined by the unfulfilled dream of having an ART child. Meanwhile, leftover embryos are often kept in a frozen limbo awaiting destruction, gestational adoption, or parental retrieval for another round of treatment and possible birth.

So when a friend asked Klitzman, Do you want to be the father of my child? he had plenty to think about. You wouldnt have to do anything other than donate the sperm, she assured him. He writes that although the proposition was tempting, he worried, What if I disagreed with her about how to raise our child? He eventually told her no.

I have often wondered if I made the right decision. I will never know. But the choice helped me understand the predicaments that countless potential parents confront.

We now face crucial moral, social, cultural, psychological, and existential conundrums about how to employ these technologies, he remarks; whether to monitor or control them and, if so, how; and more broadly, where as a species we are or should be heading; and what responsibilities, if any, we have in these realms.

Echoing the advice of most scientists involved in this brave new world of designing babies, he writes, We need now to enhance discussion, awareness and understandings of these issues among patients, their families, clinicians in various fields, professional organizations, policymakers, and the public at large.

Klitzman is a professor of clinical psychiatry and director of the Masters of Bioethics Program at Columbia University. He also cofounded and codirected the Center for Bioethics and is a member of the Research Ethics Advisory Panel of the US Department of Defense and of the New York State Stem Cell Commission. His previous books include, among others, The Ethics Police? (2015) and Am I My Genes? (2012).

Vision contributor Dan Cloer spoke with Klitzman about what he discovered from writing Designing Babies. The conversation began with Chinese biophysics researcher He Jiankuis reasons for proceeding with germ-line editing of human embryos.

DC He Jiankuicreated a set of guidelines called Draft Ethical Principles for Therapeutic Assisted Reproductive Technologies to support his use of CRISPR for editing human embryos last year. How would you evaluate these principles?

RK They sound good, but we need more than nice words. With ethical principles, God is in the details, so to speak. The issue with any ethical principle is how it will be interpreted and applied. And what does one do when different ethical principles conflict? In general, Hes principles are fine, but they are not complete. Most specifically, what is missing for me is that he does not address questions of risk and external review.

Its not just that there may potentially be benefits to gene editing down the line, but what are the risks for someone today? Ideally, the CRISPR gene-editing tool might be used to intervene when no alternative treatment exists and the patient will otherwise die. In this case, there are good treatments for HIV and methods for avoiding transmission of HIV to a child. Gene editing was not necessary; the risks outweighed the benefits.

Researchers and policy makers around the world struggle to lead human germ-line editing into the future.

The other crucial element in international ethical guidelines is the need for a process of external review. Its not enough for scientists to say, Im going to cure people. I made this invention in my laboratory, and Im going to give it to everyone and save their lives! There must be an outside reviewer to say, Wait a second. What is it? What are the risks? What are you going to tell people about it?

We know that every scientist has conflicts of interest. They want their discovery, intervention or innovation to work. Unfortunately, there have been many cases where scientists, in their eagerness to show that their idea works, overlook risks and problems. For instance, one of Hes principles was Organizations developing genetic cures have a deep moral obligation to serve families of every background. We know now that he had plans to develop an offshore clinic to design babies for wealthy people around the world, who would come to wherever the clinic was located. That certainly shows a conflict of interest. Was he going to provide services for poor people too? Did he feel obliged by that statement to serve everyone?

DCIVF itself is very expensivemaybe $1520,000 for one treatment cycle. Thats a high bar for many right now, even before we start adding embryo gene sequencing and, at some point, gene editing. And there is no money-back guarantee.

RKThese technologies can help us and they can hurt us. They have the potential to make us better by eliminating some diseases, but also to make us more unequal by enhancing the children of people who can pay for it.

I suspect that CRISPR will eventually be introduced at fertility clinics in various countries. Someone will come along and offer somethingIQ, physical traits, other abilitiesand it will cost thousands of dollars.

For technologies that we are using right now, like Preimplantation Genetics Diagnosis (PGD, used to examine IVF embryos for abnormalities), we already face questions of equity and access. Its great that couples who may have inherited genes associated with breast cancer can have embryos examined and screened. But the procedure is expensive and often not covered by insurance. So wealthy people can afford to screen their embryos and maybe remove a disease like breast cancer from their family. Up to now, genetic diseases have been equal-opportunity killers. In the future, I think these conditions may increasingly become diseases of the poor. Obviously a lot of social questions arise when the wealthy can remove a mutation from their gene pool while others cannot.

DCYou say that your views concerning infertility changed while writing Designing Babies. What I saw astonished me, you write. These men and women shed light on the myriad, unforeseen facets and ramifications of these new technologies and dramatically altered my views. How so?

RKI started out in some ways wary of altering the genes of future generations. But I quickly realized that there are times when it might be helpful. PGD can be used to get rid of serious diseases. The key issue is to avoid bad uses and reduce the risks. Rather than saying that designing babies will be either good or bad, the question is when is it good, and when is it bad? It is not monolithic.

DCDo you see any ethical problem in creating IVF embryos that will be tested and rejected or passed on from the clinic to the lab to be used in research? What about surrogate gestation?

RKPersonally, nobut informed consent is important. We have hundreds of thousands of unused embryos in this country. Couples understandably dont want to throw them out, but sometimes they dont want to pay for storage either. But what is the moral status of an embryo? This is also an important question in the abortion and stem-cell debates. My opinion is that if a couple is trying to avoid passing on a gene associated with a serious disease, and some embryos have that gene, then its okay not to use these embryos. These are unfortunate choices that have to be made.

Similarly, I was quite wary of the idea of buying and selling eggs or sperm, or women renting their wombs [as gestational surrogates]. There are real concerns about exploitation of women and horror stories of children abandoned when the prospective parents changed their minds and no longer wanted the child after the surrogate gave birth. But as I looked into the issue through my interviews and learned about the women who were gestational surrogates, I found that many of them were not, in fact, being exploited. They knew what they were doing: Hey, I have two or three kids of my own and if I can make $90,000 just sitting around the house, that could help me pay for my kids college education.

DCSo you began to think differently because you were no longer just at your desk mulling it over academically?

RKExactly right. I wrote this book because the public debate is still focused on whether these technologies are good or bad. Whats missing are the voices of people who are personally involved, invested. Gloria Steinem tells the story of potential exploitation and impoverished women being forced to be surrogates against their will. At least in the US, the data do not support that. Certainly exploitation is bad, and we want to avoid it. But its not clear to me that it is happening. Doing research and finding out what the lived experiences are from the people involved really opened my eyes.

This speaks to the fact that many people dont yet know much about these technologies. Its true of gene editing but also of other forms of ART: buying and selling eggs and sperm, renting wombs. It affects lots of people but goes largely unexamined because there are taboos against talking about sex and reproduction. Men dont like to talk about impotence and low sperm count, feeling it means they are not macho. Women feel great shame about the fact that their eggs arent working or that theyve had to buy someone elses eggs, and that they are therefore not genetically related to their child. Theres secrecy and fear. Parents are afraid that their daughter wont love them as much if she finds out that her mom is not actually her genetic mother. But these secrets have costs. Kids may find these things out later and can become very upset and feel betrayed. Evidence suggests that children should be told from an early age that other people helped bring them into the world, or however a parent might want to communicate that.

I also came to realize how prevalent these issues are. In Denmark, for example, 7 percent of all births occur through some form of ART, and I believe that will soon be the number in the US. This is a big issue that we need to pay more attention to, on both the personal and the policy level. The CDC [Centers for Disease Control] collect some data, but theres more to do.

DCWe dont collect and apply data very well, you say. But there are things potential users of these technologies should knowdarker things that you learned as well.

RKWe do need data, but some issues stand out even now. We know that over 40 percent of twins born through IVF have medical problems. Yet fertility doctors, using IVF, commonly implant multiple embryos to increase the odds of a successful birth; its a selling point for the clinic. If Im an IVF doctor, I can say, Look at how many live births I have. Its good marketing, but not so good for the baby. I did not know this, and many IVF patients dont know it either. Thats why many European countries that provide national health-care coverage for IVF and for neonatal intensive care units have much stricter rules than we do in the US concerning the number of embryos doctors can transfer into the womb.

A lot of data could be readily gotten, but reporting is not mandatory, and many clinics dont want to provide their statistics. Clinics that dont report are actually increasing their business, so theres almost a reverse incentive. Changes in policy could address this problem, but unfortunately the lack of data is not accidental. Many clinics dont want to tell potential customers that there may be problems; they just want to say, Well bring a baby into your life. Some clinics dont want young women they recruit as egg donors to fully understand that the procedures might cause harms, such as Ovarian Hyperstimulation Syndrome, which can have serious symptoms. Collecting and publishing more data might reveal limitations and dangers.

I would say that more information is better for those who are considering using these procedures; but some IVF doctors dont want all the data out there, and they have had sway.

DCYouve called the ART industry in the United States a kind of Wild West in terms of the relative lack of regulation. Is there a business opportunity here to form some sort of watchdog group?

RKThe American Society for Reproductive Medicine (ASRM) and other physician organizations have done a good job of coming up with guidelines in many areas, but I think they can go further. For example, ASRM permits selecting the sex of a baby for family balancing. But what does that phrase really mean? A couple has one girl and now wants a boy? Or they have four girls and now want a boy? The organization needs to be more specific. The question of enforcement also arises. In terms of egg donation, we know that young womens eggs are biologically better. But should a doctor try to get 18- to 21-year-old women to sell their eggs? How risky is this for their own reproductive future? Guidelines say doctors should not recruit such young women, but clinics often do so anyway. Hence, even when there are guidelines, many clinics dont follow them and resist stronger guidelines. Today theres little if any consequence for not adhering to them.

In this Wild West, cowboys are often doing what they want without too much supervision. There are many wonderful IVF doctors; by no means am I saying they are all bad. But oversight and greater openness to potential limitations are important.

The danger of jumping in right now and attempting to design babies using CRISPR/gene editing is that we really dont know the full effects. Patients should be able to make fully informed decisions.

DCIn the real Wild West, people knew it was wild because they had a model of what civilization looked like. But we dont have a model for reference. Were building from scratch.

RKYes. Part of this is because of ever newer technologies. But just as with implanting multiple embryos, doctors widely usedand sometimes still usetechnologies that turned out to be more harmful and less beneficial than thought. A technique called ICSI [intracytoplasmic sperm injection] took one sperm from a man with a very low sperm count and injected it directly into an egg. Now ICSI is used for two thirds of all patients, without regard to the mans fertility. But the procedure turns out to double the risk of the child having intellectual disabilities. Unfortunately many potential parents dont realize this. It may help get you pregnant, but it can cause problems.

A major challenge of the Wild West is that doctors might be making a lot of money but may not always communicate the risks as well as they should, because they have a conflict of interest: profiting from what theyre doing.

DCWhere do your books Designing Babies and Am I My Genes? overlap?

RKAt one point scientists thought they would discover the cancer gene, the alcoholic gene, the schizophrenia gene. But lo and behold, were finding out that for most common diseases and traits, many genes are involved. Diseases can also be partly genetic, partly environmental. Theres a lot of nuance.

Listing the potential causes of a disease is like describing the possible reasons for a traffic jam: Did a car break down? Was there an accident? Is a bridge closed? Is it icy? Is it rush hour? Or some combination of those?

Several companies market direct-to-consumer genetic testing, pushing the idea that these services will give you important health information and help you get control of your health. Precision medicine can indeed potentially help many people. But our genetics are complicated. Am I My Genes? explored how genetic testing serves almost as a Rorschach, with people interpreting the information in varying ways.

The idea for Designing Babies grew from my work on Genes. When I interviewed people at risk for Huntingtons, I thought I would hear about issues of discrimination or insurance. But what I heard was concerns about their kidswhether to adopt or abort, or to test embryos. I heard a very disturbing observation: If I abort or dont choose an embryo because it has the same mutation that I have, I should have been aborted; am I saying that my life was not worth living? That question still haunts me. It made me want to investigate further the ethical, moral, social and psychological questions that assisted reproductive technologies present us.

The common element and challenge that comes from both books is that we still know relatively little about genes. Its been only 60 years or so since the discovery of the DNA helix and how hereditary information is encoded. The human genome was first sequenced less than 20 years ago. Much of what we have found is not what we expected.

DNA is becoming a second language of sorts because of its most attractive promise: customized, personalized medicine.

DCYou write about individual procreative liberty. Do you have any concern that in the future parents might be pressured to use ART to avoid creating an imperfect child? Could there be a time when national health care includes or compels IVF, and that parents who opt to have children with disease will be ostracized and lose access to social services because they willingly brought a baby into the world that would require more than its share of medical and support resources?

RKPast attempts to improve the genes of people led to horrific results. Nazi Germany sought to remove bad genes by killing people. Hitler got his ideas about eugenics from the United States and our unfortunate history of racism. In the 1920s, we had eugenics fairs and better-baby contests. So we need to be very careful about efforts to improve our genes.

Given our history, however, its hard to predict what the government will do. We know that at one point the government pushed sterilization of the so-called feebleminded. And the question of abortion is obviously very contentious.

The cost of IVF will be an important factor in shaping its possible future. We dont have enough money to provide basic health services to all citizens now. So even if IVF becomes more accessible and less expensive, which I think it will to a certain degree, I dont think it will ever be mandated. People just get pregnant naturally all the time, planned or unplanned. I dont think were going to get to the point where well just not support you unless you used IVF.

DCWhat is the most important question your book helps us explore?

RKWhat kind of society do we want in terms of our ability to design babieswhether choosing or discarding certain embryos; buying and selling human eggs, sperm, and embryos; or altering genes directly?

As with all technologies, ARTs can be used for good or for bad. How do we choose the good over the bad? Do we want to live in a world where people use all of these technologies with few limits? Undoubtedly everyone in America knows someone, whether they realize it or not, who has had infertility problems and has used these technologies. These patients journeys have often been lonely and isolating.

I think the book can help open up the conversation and assist us allas individuals, as friends and family members, and as a society as a wholein figuring out how best to use these technologies for our own good.

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The ART of Having Children - Vision Insights and New Horizons

Previous govts never thought of peoples welfare: CM – Hindustan Times

Chief minister Yogi Adityanath on Saturday said previous governments never thought about peoples welfare, while our government is continuously working on this (aspect).

He made the comment after inaugurating the new academic block of Dr Ram Manohar Lohia Institute of Medical Sciences in Lucknow.

Between 1947 and 2016, the state got 12 medical colleges. Between 2017 and 2019, 15 new medical colleges are coming up, of which seven have started admission. This is the difference in the thought process and efforts. In 70 years, 12 colleges came up. In the last three years, 15 (are being established), the chief minister said.

A proposal for 14 more medical colleges in UP had been sent to the central government, he added.

Without naming any government, the chief minister said, The central government demanded a proposal for life support ambulances but the state never sent it. When we came to power (in 2017), we got the process done in two months. At present, 250 of them (life support ambulances) have served 78,000 patients in the state. In addition, medical mobile units (MMUs) are running in 53 districts.

It is with the prime ministers vision and efforts that the state is getting two AIIMS. An effort is being made to eliminate TB by 2025 across the country, he said.

DOCTORS ASKED TO IMPROVE BEHAVIOUR

Asking doctors to improve their behaviour, Adityanath said, If you misbehave with patients, they will not come to you and your career will be finished.

Institute director Prof AK Tripathi said, We propose to start several new departments -- geriatric medicine, clinical haematology, rheumatology, hospital administration, stem cell and regenerative medicine, international medicine (climate change, tribal medicine) and plastic surgery.

Of these, geriatric medicine and clinical haematology can be started soon, Tripathi added.

The new 14-storey building inaugurated by the CM will house 240 faculty chambers, basement parking (three floors), 48 seminar rooms, four lecture theatres, each one with seats for over 200 people.

INTERNATIONAL MEDICINE DEPT LIKELY

TO ATTRACT STUDENTS

Among the new departments that have been proposed international medicine is likely to attract many students. International medicine is a field of health care, dealing with health across regional or national boundaries.

Practised very little in India, this branch will help treat international patients in a better manner, said Prof AK Tripathi.

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Attending This Year’s North American CF Conference Gave Me Hope – Cystic Fibrosis News Today

Two weeks ago, I watched a livestream of theNorth American Cystic Fibrosis Conference (NACFC) as I popped Trikafta (elexacaftor/tezacaftor/ivacaftor) tablets out of their blister pack. Francis Collins, one of the researchers who discovered the cystic fibrosis transmembrane conductance regulator (CFTR) gene, was singing to an audience of thousands. I teared up when he sang, Dare to dream till the story of CF is history.

It was a poignant moment. A researcher who dedicated his life to cystic fibrosis sang about persisting until we find a cure as I took a medication to slow the progression of my disease.

Francis didnt just reinforce the solidarity of the researchers, nurse coordinators, social workers, and doctors who work each day to improve our lives. He ignited the room through empathy. His serenade began with a song from Five Feet Apart that emphasizes the perspective of people who face the unknown every day: Im not givin up, even when Im down to my last breath, so dont give up on me.

That is our communitys plea.

This was my first time at NACFC, thanks to the U.S. Adult Cystic Fibrosis Association. My biggest takeaway from the conference is that we are a team. We arent stopping just because we have treatments available to us.

You may be wondering why I was watching the livestream. I realized that in order to conserve energy and optimize the hours between my daily breathing treatments, I couldnt attend every session. Instead, I spent a good amount of time in the exhibitor hall, chatting with people I had only interacted with through email. I was astounded by the incredible science on display and was especially fascinated by the living, breathing pig lungs that were inhaling air through a positive expiratory pressure device.

Despite the emphasis that has been placed on modulator therapies like Trikafta, patients still need anti-infectives. Bacteria wont just disappear from our lungs after decades of colonization. We also need regenerative medicine because many of us have underlying damage that modulators cannot treat. I was concerned that modulators might overshadow the importance of other research areas, but I was pleasantly surprised.

I saw hundreds of research posters on airway clearance devices, novel molecules as anti-infectives, and quorum sensing to eliminate bacterial communication, to name a few. I also met a researcher who knew me as patient EB. James Gurney heard of my case earlier this year when I was treated with the bacteriophage he was presenting at NACFC. The researcher-patient connection came full circle, and we were both amazed.

I stay up to date with research, but the results of a stem cell therapy in Phase 1 patient clinical trials gave me even more hope! I heard trial data on RNA therapy. Organoids are being utilized in theratyping procedures to deliver personalized medicine. Companies Ive never heard of are trying to outdo the success of Trikafta by researching new modulators. I also learned that in multiple labs around the world, gene editing has been successful in fixing the CFTR gene!

Future treatments are fueled by the CF Foundations Path to a Cure. The plan is to provide modulators for everyone, treat comorbidities like infections and cystic fibrosis-related diabetes, optimize medical systems, facilitate mutual learning between doctors and patients, and ultimately find a cure. People are excited to open doors for patients. Medical professionals are starting to recognize our value in facilitating the path toward care partnerships.

I encourage patients and caregivers to watch the NACFC sessions that are available online. More than ever, I encourage people with CF to be active and open to participating in new clinical trials. Be inquisitive, be bold in trying new treatments, and share your health journey with researchers and care teams who need your valuable input.

Without the opportunity to attend NACFC, I wouldnt have known that stem cell trials are happening in the CF community! I hope to attend NACFC again and remain an active part of the research community.

***

Note: Cystic Fibrosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Cystic Fibrosis News Today, or its parent company, BioNews Services, and are intended to spark discussion about issues pertaining to cystic fibrosis.

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Scranton Police Officer Finds a Connection Through Cancer – WNEP Scranton/Wilkes-Barre

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SCRANTON, Pa. -- A Scranton police officer and a teacher in the Riverside School District found themselves connecting over something most people hope to never endure -- a cancer diagnosis.

As a Scranton Police officer and a member of the department's special operations group, Chris Hallock knew he had signed up for a dangerous job, one that might put his life at risk.

"Another interview I did, they asked if being a police officer prepared me for this. And I actually laughed at that question," Hallock said. "There's nothing that can prepare you for this."

"Shots fired, man down." Those were phrases on Hallock's radar. "You have cancer," wasn't one of them.

"You hear about it, and you know we would attend benefits, and I would run 5Ks for people, and you just never imagined it would be you that they'd be telling that you have cancer."

Chris was diagnosed with acute myeloid leukemia. A cop in his mid-30s, who works out for two hours a day, and runs 5Ks, was now having trouble simply walking up the stairs.

"Everyone looks at you like you're the wounded animal now. And, you know, that's a tough pill to swallow," he said.

That feeling is something Maria Voytko, a teacher in the Riverside School District, is all too familiar with.

"I still feel like I have this big C on my forehead, that's like, I have cancer, and everybody's kind of looking at me like, 'Is she OK now?' and it's, unfortunately, never going to go away, so it's something that you just have to deal with."

Maria was diagnosed with the same type of leukemia when she was just a senior in high school. She and Chris connected through a friend of Chris' wife, Angela.

Although they were diagnosed at vastly different stages of their lives, the pair found that cancer ignores circumstances.

"I'll never forget the first time that Maria broke through to me was she told me just ask me how the cancer stares were going. and, you know, I just, I go, 'OK, she gets it,'" Hallock said.

In February of last year, Hallock received a lifesaving stem cell transplant from his sister. He's now in recovery mode, which means countless doctors' appointments and treatments to make sure his immune system regenerates properly.

Even then, as Maria knows, recovery doesn't stop there.

"Remission does not end the chapter of cancer. It is a lifelong sentence, unfortunately."

A sentence that is much easier to live out with people by your side, especially people who understand what you're going through.

"It brought me hope, you know, seeing, Maria. You know, in talking to her and seeing how she beat it."

But the past two years weren't always full of hope and happiness for Hallock.

"People always say you're so strong, and sometimes I struggle with that because people didn't see behind the scenes."

Chris and Angela gave birth to their first son, Giovanni, now 2, one month before the diagnosis. Some might say that's bad timing, but Hallock calls it a blessing in disguise.

"He gave me something to fight for. You know, I knew I needed to be there, and I wanted to be there for him, and I wanted to set a good example for him that you know if times get tough, you just, you know, keep pushing forward. And, you know, I don't know if I would have been as strong as I was if it wasn't for him," Chris said.

For Chris and Maria, it's no longer about surviving life. It's about celebrating it.

"I am so proud of every single year in my life. I'm 41, and I made it here, and I'm going to make 42 and 82, you know, so I'm never going to lie about my age. People think I'm crazy," Maria said.

"It's living in the honor of people who, unfortunately, didn't make it. It's rubbing into cancer's face that, you know what? Yeah, I'm here. I'm here. You gave me your best shot, and I'm here," Chris said.

Now Chris wants to pay it forward and help others the same way Maria helped him giving public speeches, reaching out on social media, even just chatting with other cancer patients he sees at the hospital when he goes in for treatments. Most importantly, he wants to get his message out there

"Tomorrow's a new day with a new beginning, and you just have to remember that even if this is the worst day of your life, tomorrow could be the best day of your life, but if you don't fight to live till tomorrow, you'll never experience that."

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Scranton Police Officer Finds a Connection Through Cancer - WNEP Scranton/Wilkes-Barre

Breaking News: Cardiol Therapeutics Announces Clinical Steering Committee for Phase 2 International Trial in Acute Myocarditis Using CardiolRx(TM) 100…

Cardiol Therapeutics Inc. (TSX: CRDL) (OTCQX: CRTPF), a leader in the production of pharmaceutical cannabidiol (CBD) products and in the development of innovative cannabidiol medicines for heart disease, is pleased to announce the formation of the Clinical Steering Committee (CSC) for a Phase 2 international trial in acute myocarditis using the Companys CardiolRx100 cannabidiol formulation.

The CSC, which comprises key opinion leaders in acute myocarditis from North America and Europe, recently met during the American Heart Associations Scientific Sessions in Philadelphia held November 16th to 18th. The role of the CSC is to advise on the trial design, provide overall supervision of the trial, and ensure that it is being conducted in accordance with the principles of Good Clinical Practice. The CSC has oversight of the protocol, any protocol amendments, and provides advice to the investigators on all aspects of the trial.

Acute myocarditis is characterized by inflammation of the heart muscle (myocardium). The most common cause is viral infection of the heart tissue which is initially responsible for the inflammation. In a significant number of cases, perhaps due to an autoimmune process, the inflammation persists with ongoing myocardial damage and depressed heart function. Although the symptoms are often mild, myocarditis remains an important cause of acute and fulminant heart failure and is the most common cause of sudden cardiac death in people less than 35 years old. In addition, some patients proceed to develop chronic dilated cardiomyopathy which continues to be the leading indication for cardiac transplantation. Symptoms include chest pain, fatigue, shortness of breath, and arrhythmias. Because of the progressive damage to heart cells, heart failure develops (defined as the inability of the heart to pump sufficient blood to meet the needs of the body). The study will use left ventricular ejection fraction (LVEF) as one measure of heart function.

CardiolRx100 is Cardiol Therapeutics pure pharmaceutically (cGMP) produced high concentration cannabidiol formulation that is THC free (<10ppm). Based on the large body of experimental evidence of the anti-inflammatory and cardioprotective properties of cannabidiol in models of cardiovascular disease, Cardiol believes there is an opportunity to develop a potential breakthrough therapy for acute myocarditis that would be eligible for designation as an orphan drug. In the United States, an orphan drug designation is granted for pharmaceuticals being developed to treat medical conditions affecting fewer than 200,000 people. These conditions are referred to as orphan diseases. In the U.S. and the European Union, orphan drugs are eligible for accelerated marketing approvals and companies developing orphan drugs typically receive other incentives, including a prolonged period of market exclusivity that can extend over seven years, during which the drug developer has sole rights to market the drug.

Cardiol has assembled eight highly distinguished thought leaders in cardiology from North America and Europe to oversee and guide our acute myocarditis trial that is being planned at world leading heart institutes, including the Cleveland Clinic, the Mayo Clinic, the Houston Methodist DeBakey Heart and Vascular Center, the University of Ottawa Heart Institute, and Charit University Medicine Berlin, stated David Elsley, President and CEO of Cardiol Therapeutics. The U.S. orphan drug program was successfully utilized to accelerate the first FDA approval of cannabidiol for the treatment of two pediatric epilepsy orphan diseases. We see a similar opportunity with our international trial in acute myocarditis to fast track the development of our CardiolRx formulation for a serious cardiovascular orphan disease for which there is currently no accepted standard of care.

Members of Cardiols Acute Myocarditis CSC include:

Dennis M. McNamara, MD (Chair)

Dr. Dennis McNamara is a Professor of Medicine at the University of Pittsburgh. He is also the Director of the Heart Failure/Transplantation Program at the University of Pittsburgh Medical Center. Dr. McNamara received his undergraduate/graduate education at Yale University, New Haven, Connecticut, and Harvard Medical School, Boston, Massachusetts, respectively. He completed his internship, residency, and cardiology fellowship at Massachusetts General Hospital in Boston. McNamaras current research interests include etiology and pathogenesis of dilated cardiomyopathies; inflammatory syndromes of cardiovascular disease; myocardial recovery in recent onset non-ischemic primary cardiomyopathy; etiology and management of peripartum cardiomyopathy; and genetic modulation of outcomes in cardiovascular disease.

Leslie T. Cooper, Jr., MD (Co-Chair)

Dr. Leslie T. Cooper, Jr., is a general cardiologist and the chair of the Mayo Clinic Enterprise Department of Cardiovascular Medicine, as well as chair of the Department of Cardiovascular Medicine at the Mayo Clinic in Florida. Dr. Coopers clinical interests and research focus on clinical and translational studies of rare and undiagnosed cardiomyopathies, myocarditis, and inflammatory cardiac and vascular diseases, such as giant cell myocarditis, cardiac sarcoidosis, eosinophilic myocarditis, and Takayasus arteritis. He has published over 130 original peer-reviewed papers, as well as contributing to and editing books on myocarditis. In addition to his clinical and research work, Dr. Cooper is a fellow of the American College of Cardiology, the American Heart Association, the European Society of Cardiology Heart Failure Association, the International Society for Heart and Lung Transplantation, and the Society for Vascular Medicine and Biology. He is also the founder and former president of the Myocarditis Foundation and continues to serve on its Board of Directors.

Arvind Bhimaraj, MD

Dr. Arvind Bhimaraj is a specialist in Heart Failure and Transplantation Cardiology and is Assistant Professor of Cardiology, Institute for Academic Medicine, at Houston Methodist and at Weill Cornell Medical College, NYC. He has been Co-Director of the Heart Failure Research Laboratory at Houston Methodist since 2016. His area of focus is anti-fibrotic mechanisms and how to promote recovery of a damaged heart. Dr. Bhimaraj was a Heart Failure Fellow at the Cleveland Clinic from July 2010 to September 2011. Dr. Bhimaraj also specializes in Interventional Cardiology, is board certified in Cardiovascular Disease, and the author of numerous cardiovascular publications.

Matthias Friedrich, MD

Dr. Matthias Friedrich is Full Professor with the Departments of Medicine and Diagnostic Radiology at the McGill University in Montreal and Chief, Cardiovascular Imaging at the McGill University Health Centre. He is also Professor of Medicine at Heidelberg University in Germany. Dr. Friedrich earned his MD at the Friedrich-Alexander-University Erlangen-Nrnberg, Germany. He completed his training as an internist and cardiologist at the Charit University Medicine Center, Humboldt University in Berlin. Dr. Friedrich founded one of the first large Cardiovascular Magnetic Resonance centres in Germany at the Charit University Hospital in Berlin. After his move to Canada, from 2004 to 2011, he was Director of the Stephenson Cardiovascular MR Centre at the Libin Cardiovascular Institute of Alberta and Professor of Medicine within the Departments of Cardiac Sciences and Radiology at the University of Calgary, Canada. From 2011 to 2015, he directed the Philippa and Marvin Carsley Cardiovascular MR Centre at the Montreal Heart Institute and was Michel and Renata Hornstein Chair in Cardiac Imaging at the Universit de Montral.

Peter Liu, MD

Dr. Peter Liu is the Chief Scientific Officer and Vice President, Research, of the University of Ottawa Heart Institute, and Professor of Medicine and Physiology at the University of Toronto and University of Ottawa. He was the former Scientific Director of the Institute of Circulatory and Respiratory Health at the Canadian Institutes of Health Research, the major federal funding agency for health research in Canada. Prior to that role, he was the inaugural Director of the Heart & Stroke/Lewar Centre of Excellence in Cardiovascular Research at University of Toronto. Dr. Liu received his MD from the University of Toronto, and postgraduate training at Harvard University. His laboratory investigates the causes and treatments of heart failure, the role of inflammation, and the identification of novel biomarkers and interventions in cardiovascular disease. Dr. Liu has published over 300 peer-reviewed articles in high impact journals and received numerous awards in recognition of his research and scientific accomplishments.

Wai Hong Wilson Tang, MD

Dr. Wai Hong Wilson Tang is the Advanced Heart Failure and Transplant Cardiology specialist at the Cleveland Clinic in Cleveland, Ohio. Dr. Tang is also the Director of the Cleveland Clinics Center for Clinical Genomics; Research Director, and staff cardiologist in the Section of Heart Failure and Cardiac Transplantation Medicine in the Sydell and Arnold Miller Family Heart & Vascular Institute at the Cleveland Clinic. He attended and graduated from Harvard Medical School in 1996, having over 23 years of diverse experience, especially in Advanced Heart Failure and Transplant Cardiology. Dr. Tang is affiliated with many hospitals including the Cleveland Clinic and cooperates with other doctors and physicians in medical groups including The Cleveland Clinic Foundation.

Barry Trachtenberg, MD

Dr. Barry H. Trachtenberg is a cardiologist specializing in heart failure and cardiac transplantation. He is also the director of the Michael DeBakey Cardiology Associates Cardio-Oncology program, an evolving field devoted to prevention and management of cardiovascular complications of cancer therapies such as chemotherapy and radiation. His clinical experience includes heart failure and heart transplantation, mechanical support pumps, and cardio-oncology. He has contributed to multiple publications related to advanced heart failure, cardiac transplantation, regenerative therapies, and ventricular assist devices. Dr. Trachtenberg is a member of the American Heart Association, the International Society for Heart and Lung Transplantation, the Heart Failure Society of America, and the International CardiOncology Society of North America.

Carsten Tschpe, MD

Dr. Carsten Tschpe is Professor of Medicine and Cardiology and Vice Director of the Department of Internal Medicine and Cardiology, Charit University Medicine Berlin. He received his doctorate in medicine in 1993 and has over 140 peer-reviewed publications, including overview and book articles, and 120 international original articles. His research interests include inflammatory cardiomyopathy, diabetic cardiopathy, and ischemic cardiopathy. He also includes diastolic dysfunction, endothelial dysfunction, peptide systems, and experimental and clinical studies in cardiology and stem cells in his research studies. For his outstanding research work, Dr. Tschpe was awarded the prestigious Arthur Weber Prize by the German Cardiac Society Cardiovascular Research.

About Cardiol Therapeutics

Cardiol Therapeutics Inc. (TSX: CRDL)(OTCQX: CRTPF) is focused on producing pharmaceutical cannabidiol (CBD) products and developing innovative therapies for heart disease, including acute myocarditis and other causes of heart failure. The Companys lead product, CardiolRx, is designed to be one of the safest and most consistent CBD formulations on the market. CardiolRx is pharmaceutically produced, cGMP certified, and is THC free. The Company plans to commercialize CardiolRx in the billion-dollar market for medicinal cannabinoids in Canada and is also pursuing distribution opportunities in Europe and Latin America.

In heart failure, Cardiol is planning an international clinical study of CardiolRx in acute myocarditis, a condition caused by inflammation in heart tissue, which remains the most common cause of sudden cardiac death in people less than 35 years of age. The Company is also developing proprietary nanotechnology to uniquely deliver pharmaceutical CBD and other anti-inflammatory drugs directly to sites of inflammation in the heart that are associated with heart failure. Heart failure is the leading cause of death and hospitalization in North America with associated healthcare costs in the U.S. alone exceeding $30 billion. For further information about Cardiol Therapeutics, please visitwww.cardiolrx.com.

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Breaking News: Cardiol Therapeutics Announces Clinical Steering Committee for Phase 2 International Trial in Acute Myocarditis Using CardiolRx(TM) 100...

Teen ready to get back on the field after risky tumor surgery – KFOR Oklahoma City

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NORMAN, Okla. (KFOR) - A love of sports runs deep for 14-year-old Xander Brayfield from Norman.

Basketball, soccer, E-sports, and even a friendship with OU Linebacker Kenneth Murray are all things that bring out this teenager's famous grin.

But in quiet moments last May, Xander started feeling a troubling pain he couldn't ignore.

His mother, Kirsten De Beurs recalled, "When it came time to sit down and do his homework, he said my back is hurting so bad I can't take it."

"It was not the best summer," agreed Xander.

Not the best, because of what scans revealed.

Xander had a large tumor, wrapped around his aorta, lodged and growing between his heart and his spine.

"I couldn't listen to the doctors the first week because all I did was cry and I couldn't process the information," said Xander's mother.

Dr. Kisha Beg from Jimmy Everest Cancer Center shakes her head when asked about Xander's treatment for neuroblastoma cancer.

"You get a whole kitchen-sink of treatment," she acknowledged.

She says she's been very impressed by how Xander takes charge of his treatment plan.

"He picked up all the chemotherapy papers, he wanted me to talk to him about all the terms, all the side-effects, and he had GREAT questions," Dr. Beg said.

Chemotherapy did not erase the tumor, and the Brayfields eventually traveled to Michigan to a specialist who collaborated with Jimmy Everest Center doctors and performed an extremely risky operation.

Xander shows how the incision wraps around from his ribs to his shoulder blades.

He explained, "She went through my ribs, and took out the tumor somehow."

This tricky surgery was successful but is just the start of a checklist of needed medical procedures.

Dr. Beg explained, "He has to go through a transplant, stem-cell transplant, radiation, then maintenance."

All this difficult news has been balanced by incredible support from his medical team, his school, and the community.

Xander says he stays on track mentally by "just staying strong and knowing you're going to get back to your normal stuff if you just keep on going."

He added, "I love playing soccer, so as soon as I'm ready for it I'm going to get on the field and practice, get a physical trainer and get back in shape."

Xander is always ready with a smile, ready to reclaim his old life.

If you would like to help kids like Xander fight cancer, consider donating to JECFriends.org.

Kids with Courage is sponsored by Friends of the Jimmy Everest Center.

35.222567-97.439478

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Teen ready to get back on the field after risky tumor surgery - KFOR Oklahoma City

Chemotherapy and constant pills but DWP tells Hull man Kye Eastwood ‘you’re fit for work’ – Hull Daily Mail

A young Hull man whose life was saved by the generous people of Hull after being diagnosed with terminal cancer says he is looking forward to the future.

In 2014, Kye Eastwood, now 28, faced an unimaginable battle after being told there was nothing more UK doctors could do for him in his battle against Hodgkins Lymphoma - but there was hope.

The people of Hull heroically clubbed together and raised 46,000 within a month, paying for ground breaking - and life-saving - stem cell - treatment in Maryland, US.

Now, five years on, Kye and his fianc Chanelle Urquhart, 24, of Kingswood, are looking forward to their lives together - after what Kye has described as one of the most difficult years of his life.

Although the pioneering treatment cleared Kye of cancer, Kye has ongoing health issues. He is still undergoing chemotherapy and he is unable to work.

Despite this, he was told he was not allowed his PIP (Personal Independence Payment) benefits from the Department for Work and Pensions (DWP) after he underwent an eligibility assessment.

He said the report was "all wrong", stating he could do a number of things he could not do, which meant he was deemed fit for work.

He said: The report said I had a healthy complexion - that is completely wrong in the first place because of the vitiligo (pigmentation of the skin) I have.

Chanelle said he also split open a sore on his back trying to lift his arms further above his head during the assessment and it still needs treatment months later.

Although he has since had his benefits reinstated after the report was proved to be wrong, the trauma of living without any income and the strain it put on him for months has taken its toll.

He said: Someone came around in March theyd decided I was poorly enough for them to come to me rather than me go to them. I was on my cycle of chemo at the time and having massive allergic reactions.

Everything in the report was wrong and the woman who came didnt even look at any of the medical evidence and made out that I could move more.

"What the report said was not reflective of what happened in the meeting. We got a letter saying I wasnt entitled and why, and the report shed sent off.

Kye appealed and received another assessment and a complaint was put into the DWP by Chanelle, who sent pictures of Kyes skin and statements from every one of his doctors and specialists who see him in Rotherham.

Three months later, Kyes benefits were reinstated.

Chanelle said: He was three months without them, which help with his mobility. He was struggling to get to Rotherham, the treatment which ultimately keeps him alive.

She added the complaint has been escalated and is being looked at by an independent case examiner.

A DWP spokesman said: "We have apologised to Mr Eastwood for the confusion over his reapplication for PIP. It was resolved promptly and he is in receipt of all the benefits he is entitled to."

Chanelle said Kye would love nothing more than to go back to work but he needs something flexible that could work around treatment and hospital appointments.

She said: He was always working before the cancer and would love to go back to work and do things but its trying to find something he would be able to do safely, and something that would work around his health problems.

Kye added: I dont know an employer that would want me to be off at least a week every month while I go to my appointments.

During Kye's treatment the couple flew back and forth to Washington for a period of seven months and were able to stay at the amazing centre along with other families of people having treatment there.

During the time between treatments, they were able to explore the country and while staying in San Antonio in 2015 when they had been together for just six months, Kye proposed.

Chanelle said: At the time it happened, we hadnt been together for very long but we didnt know if he was going to survive. We instantly clicked and it was obvious it was going to work out.

Kye said: I knew I was going to do it. Wed known each other for years.

Five years later and they are still going strong - Chanelle even got to be the one to tell Kye he was cancer-free.

She said: He was in America and Id had to stay at home because I couldnt get the time off work.

"Theyd done a scan and the doctor had emailed me and told me he was completely clear. I was in Morrisons and was crying. I was trying to call him but he didnt have any signal.

I got through to him and just said, your cancer has completely gone'. Then he went to the car and told his mum.

Despite the relief at being given the all clear, Kye has faced difficulty during his recovery and still suffers side effects from all of his treatments.

Not long after he was told he was in remission, he started to suffer with a condition called Graft Versus Host Disease (GvHD).

The disease is classed as a medical complication of receiving of transplanted tissue from a different person such as Kyes stem cell treatment.

The white blood cells left in the donated tissue only recognise the receiver as foreign and begin to attack the receivers cells.

This has left Key with patches of dry skin on his body. His skin is thinner and he has vitiligo, which changes the pigment and colour.

Kye said: It started with a bit of itchy skin and I had a dry patch that wouldnt go away and we went for blood tests and they kept coming back really abnormal, my liver levels were through the roof.

Chanelle said: That was quite worrying as its quite dangerous and they were trying all sorts of things.

He now goes to Rotherham every week for treatment for the GvHD, and is on daily chemotherapy tablets.

He says he has tried a lot of different treatments but at the moment, this combination is working - although doctors are looking at other methods.

The GvHD in itself keeps the cancer at bay so doctors want to try and find a healthy balance.

Chanelle said: Hes hooked up to a machine that removes a certain volume of blood cells, which they separate. They give him his red blood cells back and treat the white blood cells with a UV light before putting them back in his body."

As well as GvHD Kye suffers with fatigue, breathlessness, bad sinuses and says he is now going deaf.

He has tried to get back to his fitness level before the cancer, even trying out BMXing, but he tires easily and becomes weak.

Despite all of the stress and heartache throughout this year, the pair are now looking forward to the future.

Kye said: Chanelle wants to finish her degree and get a job and a nice house. Were quite different to a lot of people who get engaged and start planning a wedding straight away. Weve got all the time in the world.

Ill have to be five years in remission before no more check ups, but the scares are always there.

They always will be, Chanelle added.

Kye and Chanelle say a few years down the line, they are still hugely grateful to the people of Hull for helping them to save his life.

Chanelle said: We would like to say thank you to everyone who contributed any money, or shared the story, or got involved in any of the fundraisers.

"They are what made this possible and have given Kye this second chance at life and they are why he is still here.

He was on palliative care and was going to die he probably wouldnt have made it to Christmas.

Kye said: It feels like such a long time ago. Im definitely looking forward to 2020."

Sophie Corcoran is a reporter for Hull Live and the Hull Daily Mail. Her interests include positive news, news about homelessness, court news and breaking news.

You can follow all the latest stories on her Facebook page here , her Twitter page here or on the Hull Live website here.

You can also call her on 01482 315174 or email sophie.corcoran@reachplc.com

Our daily newsletter- To get the latest headlines direct to your email inbox every day,click here.

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Chemotherapy and constant pills but DWP tells Hull man Kye Eastwood 'you're fit for work' - Hull Daily Mail

Redruth woman travels over 3,500 miles to meet the stranger who saved her life – Cornwall Live

Ashop assistantfrom Redruthisappealing for people to become bone marrow donors after meeting the stranger who saved her life.

Carol Timmins was diagnosed with aplastic anaemia in January 2014.

The 58-year-old said: "Being told that I had bone marrow failure came as a great shock. I had no idea what it actually meant but soon realised that I was very, very sick.

"Id previously been diagnosed with Chronic Fatigue Syndrome, so feeling tired and lacking energy had been a way of life for several years. The initial treatment that I received was regular blood transfusions until May when I started an Antilymphocyte Globulin treatment.

"By September it was apparent that the treatment was not working and a bone marrow transplant was my best chance of survival."

Carolhad a bone marrow transplant inDecember 2014from a stranger. Patients and donors must remain anonymous for two years after a transplant takes place.

After this period has passed, if both the patient and donor would like to meet, they can contact blood cancer charity Anthony Nolan, who are then able to put them in contact with each other.

In September 2019, Carol and her husband Paul, travelled from Cornwall to Detroit, USA, to meet her lifesaver.

She added: "Ninety days after my transplant I had decided that it was time to thank my donor so I sent an anonymous letter through Anthony Nolan. Within a few weeks I received a reply. We were both overjoyed at receiving such an important piece of mail and he was very pleased that I was making a good recovery."

From that moment she set her sights on thanking her donor in person.

"I knew that it would be a few years before I could travel but I made it my goal. I had to thank the man who had saved my life," Carol said.

As time passed by, the medication reduced and their correspondence increased. There was a turning point in her life when she was told that she had the option to release her personal information, and to request her donors details.

Carol continued: "I decided not to rush into it, as I needed time to comprehend exactly what that would mean. I had come so far, and to be able to correspond with my donor directly would be quite surreal."

Through letter writing, they contemplated at how their paths had crossed and shared stories about their families. In May 2018 she exchanged personal details with her donor.

She said: "It was a truly amazing day learning the name of the man I had written to for four years - Darin."

Through emails they exchanged family photos and started to make plans for her trip. Their friendship blossomed and finally, in September 2019 they met for the first time.

"The welcome that I received at the airport was something that only dreams are made of. After hugs and tears of joy with Darin and his wife Valerie, I was introduced to a local news channel who were delighted to witness and report on such an emotional and inspiring story," added Carol.

Carol and Paul stayed at Darin and Valeries home in Detroit for eight days. During that time they shared their experiences as a donor and recipient, reminiscing about a very surreal time in their lives.

Carol added: "Darin said that if he was asked to donate again he would do it in a heartbeat.

"I consider myself truly blessed to have found a donor who was a perfect match, but to have the opportunity to thank the man who gave me his bone marrow has meant the world to me.

"Please dont wait until it happens to you or yours, people are dying waiting for their perfect match and you can help them by joining the register. Without Darin I probably wouldnt be here today, so if youre a healthy 16-30 year old (or you know someone who is), Anthony Nolan needs you.

"My heartfelt thanks go to Anthony Nolan and to all of the potential donors on the register, as well as the wonderful team of doctors and nurses who treated me."

Anthony Nolan is the charity that finds matching donors for people with blood cancer and gives them a second chance of life.

It also carries out ground-breaking research to save more lives and provide information and support to patients after a stem cell transplant, through its clinical nurse specialists and psychologists, who help guide patients through their recovery.

Henny Braund, chief executive of Anthony Nolan said "Its fantastic to see Carol,living her life to the fullest andDarinshould be incredibly proud of the difference he has made to Carol and her family.

"Our amazing stem cell donors are lifesavers and they continue to give patients with blood cancer a second chance of life. Anyone wanting to find out more can visit our website to join the stem cell register or support our work by making a donation, which will help give someone likeCarola second chance of life in the future. Without you there is no cure."

For more information, visit the Anthony Nolan website here.

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Redruth woman travels over 3,500 miles to meet the stranger who saved her life - Cornwall Live

These Scientists May Have Found a Cure for ‘Bubble Boy’ Disease – Smithsonian.com

On the morning of April 25, 2018, in Fort Wayne, Indiana, Omarion Jordan came into the world ten-fingers-and-toes perfect. His mother, Kristin Simpson, brought her dark-haired newborn home to a mostly empty apartment in Kendallville, about 30 miles to the north. Shed just moved in and hadnt had time to decorate. Her son, however, had everything he needed: a nursery full of toys, a crib, a bassinet and a blue octopus blanket.

Still, within his first couple of months, he was plagued by three different infections that required intravenous treatments. Doctors thought he had eczema and cradle cap. They said he was allergic to his mothers milk and told her to stop breastfeeding. Then, not long after he received a round of standard infant vaccinations, his scalp was bleeding and covered with green goop, recalled the first-time mother, who was then in her late teens. She took him to the hospital emergency room, where, again, caregivers seemed puzzled by the babys bizarre symptoms, which didnt make any sense until physicians, finally, ordered the right blood test.

What they learned was that Omarion was born with a rare genetic disorder called X-linked severe combined immunodeficiency (SCID), better known as the bubble boy disease. Caused by a mutated gene on the X chromosome, and almost always limited to males, a baby born with X-linked SCID, or SCID-X1, lacks a working immune system (hence the unusual reaction to vaccination). The bubble boy name is a reference to David Vetter, a Texas child born with SCID-X1 in 1971, who lived in a plastic bubble and ventured out in a NASA-designed suit. He died at 12, but his highly publicized life inspired a 1976 TV movie starring John Travolta.

Today, technological advances in hospitals provide a kind of bubble, protecting SCID-X1 patients with controlled circulation of filtered air. Such safeguards are necessary because a patient exposed to even the most innocuous germs can acquire infections that turn deadly. As soon as Omarion tested positive for the disorder, an ambulance carried him to Cincinnati Childrens Hospital in nearby Ohio and placed him in isolation, where he remained for the next few months. I had no idea what would happen to him, his mother recalled.

Approximately one in 40,000 to 100,000 infants is born with SCID, according to the Centers for Disease Control and Prevention. Only about 20 to 50 new cases of the SCID-X1 mutationwhich accounts for about half of all SCID casesappear in the United States each year. For years, the best treatments for SCID-X1 have been bone marrow or blood stem cell transplantations from a matched sibling donor. But fewer than 20 percent of patients have had this option. And Omarion, an only child, was not among them.

As it happened, medical scientists at St. Jude Childrens Research Hospital in Memphis, Tennessee, were then developing a bold new procedure. The strategy: introduce a normal copy of the faulty gene, designated IL2RG, into a patients own stem cells, which then go on to produce the immune system components needed to fight infection. Simpson enrolled Omarion in the clinical study and Cincinnati Childrens Hospital arranged a private jet to transport her and her son to the research hospital, where they stayed for five months.

St. Jude wasnt the first to try gene therapy for SCID-X1. Nearly 20 years ago, researchers in France reported successfully reconditioning immune systems in SCID-X1 patients using a particular virus to deliver the correct gene to cells. But when a quarter of the patients in that study developed leukemia, because the modified virus also disrupted the functioning of normal genes, the study was halted and scientists interested in gene therapy for the disorder hit the brakes.

At St. Jude, experts led by the late Brian Sorrentino, a hematologist and gene therapy researcher, set out to engineer a virus delivery vehicle that wouldnt have side effects. They started with a modified HIV vector emptied of the virus and its original contents, and filled it with a normal copy of the IL2RG gene. They engineered this vector to include insulators to prevent the vector from disturbing other genes once it integrated into the human genome. The goal was to insert the gene into stem cells that had come from the patients own bone marrow, and those cells would then go on to produce working immune system cells. It was crucial for the viral vector to not deliver the gene to other kinds of cellsand thats what the researchers observed. After gene therapy, for example, brain cells do not have a correct copy of the gene, explained Stephen Gottschalk, who chairs St. Judes Department of Bone Marrow Transplantation and Cellular Therapy.

In the experimental treatment, infants received their re-engineered stem cells just 12 days after some of their bone marrow was obtained. They went through a two-day, low-dose course of chemotherapy, which made room for the engineered cells to grow. Within four months, some of the babies were able to fight infections on their own. All eight of the initial research subjects left the hospital with a healthy immune system. The remarkably positive results made news headlines after being published this past April in the New England Journal of Medicine. Experimental gene therapy frees bubble boy babies from life of isolation, the journal Nature trumpeted.

So far, the children who participated in that study are thriving, and so are several other babies who received the treatmentincluding Omarion. As a physician and a mom, I couldnt ask for anything better, said Ewelina Mamcarz, lead author of the journal article and first-time mother to a toddler nearly the same age as Omarion. The children in the study are now playing outside and attending day care, reaching milestones just like my daughter, Mamcarz says. Theyre no different. Mamcarz, who is from Poland, came to the United States to train as a pediatric hematologist-oncologist and joined St. Jude six years ago.

Other medical centers are pursuing the treatment. The University of California, San Francisco Benioff Childrens Hospital is currently treating infant patients, and Seattle Childrens Hospital is poised to do the same. Moreover, the National Institutes of Health has seen success in applying the gene therapy to older patients, ages 3 to 37. Those participants had previously received bone marrow transplants from partially matched donors, but theyd been living with complications.

In the highly technical world of medicine today, it takes teamwork to achieve a breakthrough, and as many as 150 peoplephysicians, nurses, regulators, researchers, transplant coordinators and othersplayed a role in this one.

Sorrentino died in November 2018, but hed lived long enough to celebrate the trial results. In the early 90s, we thought gene therapy would revolutionize medicine, but it was kind of too early, said Gottschalk, who began his career in Germany. Now, nearly 30 years later, we understand the technology better, and its really starting to have a great impact. We can now develop very precise medicine, with very limited side effects. Gottschalk, who arrived at St. Jude a month before Sorrentinos diagnosis, now oversees the hospitals SCID-X1 research. Its very, very gratifying to be involved, he said.

For now the SCID-X1 gene therapy remains experimental. But with additional trials and continued monitoring of patients, St. Jude hopes that the therapy will earn Food and Drug Administration approval as a treatment within five years.

Simpson, for her part, is already convinced that the therapy can work wonders: Her son doesnt live in a bubble or, for that matter, in a hospital. He can play barefoot in the dirt with other kids, whatever he wants, because his immune system is normal like any other kid, she said. I wish there were better words than thank you.

Excerpt from:
These Scientists May Have Found a Cure for 'Bubble Boy' Disease - Smithsonian.com