Former Novartis CEO Cell And Gene Therapies Will Drive Innovation – Clinical Leader

By Ed Miseta, Chief Editor, Clinical LeaderFollow Me On Twitter @EdClinical

When Joe Jimenez was promoted to CEO of Novartis in 2010, many in the industry probably thought, Joe who? Jimenez himself chuckles when telling the story, which he did earlier this year at the 2019 Veeva R&D Summit. Jimenez had been at Novartis for less than three years and had a background in the consumer-packaged goods industry. He was working in London when AstraZeneca asked him to sit on the board of the company. In that capacity he learned about the pharmaceutical industry and decided he wanted to be part of it.

When he joined Novartis as division head of Consumer Health, Jimenez took his skillsets from the consumer-packaged goods industry and applied them to pharma. After heading Novartis Consumer Health for a few months, Jimenez was asked to run the pharmaceutical division. That surprised me, he says. I was neither a physician nor a scientist. The board told me they had physicians and scientists. What they wanted was somebody who could look at how the world was changing and position the company to win in that emerging environment."

Joe Jimenez

I said this is nuts, notes Jimenez. We can generate double digit growth if we just start investing in the commercial sales and marketing in China and in parts of Southeast Asia. By doing that, by making those allocation changes, the business took off and then three years later the board made me the CEO.

Partnerships Will Drive Innovation

When Jimenez joined Novartis, the company was launching a lot of products, but many were unsuccessful once they hit the marketplace. He felt the company was not thinking about the commercial potential of company products in the pipeline. To better oversee the income potential of treatments, Jimenez created a group called early commercial development, which he calls the commercial conscience of research.

That was a little nerve wracking, he now says. I was the only individual on the Innovation Management Board with an equal vote who was not an MD or a PhD. This got our scientists to realize that it's not good enough to create a molecule. You have to think about when you're going to launch that molecule and what the competitive landscape is going to look like.

When Jimenez made the move from Big Pharma to small biotech, he also learned about how quickly companies can move. At Novartis, he remembers being in meetings that would last hours simply trying to make a decision that should have taken five minutes. He is now an advisor for several small biotech companies where those long meetings do not take place.

Bureaucracy doesn't necessarily have to be a bad word, he admits. Bureaucracy can certainly mean red tape, but if you have a huge pharmaceutical company without a clear decision making structure, what you are left with is chaos.

One successful strategy that was used at Novartis was in creating a Venture Group, which Jimenez describes as a successful and independent effort. The company put money into a venture fund and told the fund to do its thing. It had access to the resources of Novartis but was not linked to the company.

One thing that makes pharma different from a small biotech is deep resources and the scale and type of people available, says Jimenez. There is an R&D infrastructure and the ability to work on many different projects. In a small biotech we struggle to determine if we are going to focus on one or two projects. A portfolio review at Novartis would discuss 100 different programs. When you are making a small number of bets, you really need to think carefully about those bets. If you make the wrong bet, you will be out of business. A VC firm will help pharma find the good bets by looking for innovation outside their four walls even before those therapies reach the clinical stage."

Jimenez believes we will see more partnering models emerge between pharma and biotech companies, particularly with the explosion of innovation going on in the industry. He believes new treatments being developed in the cell and gene space will drive a lot of the innovation. Those treatments will be assisted by advanced analytics that allow the industry to target disease like never before. That combination will create a new ecosystem of innovation and partnering between Big Pharma and biotech companies.

CAR T Therapy Opens Doors

Shortly after he was promoted to CEO at Novartis, the company was the first to pursue a CAR T therapy. When Novartis invested in the therapy, it still appeared to be science fiction.

We were discussing the concept of taking blood from a cancer patient, genetically engineering the T-cells in that blood to recognize and go after the tumor, and then reinfuse it back into the patient, says Jimenez. This was in 2012, and I remember my oncology people coming into my office with stunned looks on their faces. They had just become aware of Carl June's work at the University of Pennsylvania. His early work was with chronic lymphocytic leukemia, and his CAR T therapy was being used on patients who had no other hope.

Novartis looked at Junes work and decided they had to be involved with it. At the time, no other large pharma companies opted to make an investment. Kite and Juno were two biotechs that emerged in the space and were quickly purchased by Big Pharma.

We were the original pioneers and decided we would do whatever it takes to get those treatments to the patients that needed them, states Jimenez. There were patients who had no other options. We knew this type of therapy had the potential to save thousands of lives. That may not have been our most successful endeavor, but I do think it is the one that has had the biggest impact.

Now that gene editing is a reality, Jimenez believes regenerative medicine holds huge hope for patients. The U.S. population is aging, and Novartis has been investigating how treatments can increase life expectancy and prolong vision, hearing, and muscle as humans age. These are areas where there is significant degeneration.

We can look at muscle as an example, says Jimenez. When humans age they lose a lot of muscle. That makes us frail, which leads to injuries, hip fractures, and eventual death. If we can build muscle among the elderly by finding a way to increase muscle mass, we could potentially extend life. There are currently several small biotech companies looking at muscle, as well as hearing and sight. I believe we may see significant increases in life expectancy in our lifetime.

A Revolution Of Advanced Analytics

Looking towards the future, Jimenez believes advanced analytics and other emerging technologies will revolutionize all elements of the pharma industry, including research, drug development, and commercialization. New technologies can help the entire drug development process work faster, easier, and less expensively.

Jimenez is also a big believer in decentralized trials, which are taking trials to where the patients are instead of to the trial sites that have been used in the past. That model will speed clinical development but also enable patients who would otherwise not be able to participate in a trial. Now anyone can participate even if they dont live near a clinic or medical center.

All of this is happening because of technology, data, and data science, he states. I believe that in the next 10 years we are going to see radical changes in the way that research, development, and commercial are executed. Soon everything will be driven by data.

Today around 40 percent of all investment is going into the area of immuno-oncology. Jimenez believes thats good, since patients will now have new treatment options that they never thought possible. He notes there are around 3,500 immuno-oncology therapies currently being investigated. Still, he worries about what that might mean for more traditional diseases that still have a large unmet medical need. For example, he sees some companies almost completely abandoning respiratory disease, rheumatology, dermatology, GI, and cardiovascular disease. While the news is good for oncology patients, the swing in funding could leave other patients in need.

The patients with respiratory disease deserve new medicines as well, says Jimenez. We have to make sure we prevent what happened in anti-infectives, where there is currently very little activity. We are on the verge of turning several cancers into chronic diseases. That is very exciting news, but we will need companies to pick up the slack in these other disease areas.

Finally, for everyone involved in drug development, Jimenez recommends that you disrupt. Look at what you're working on today and find new ways to radically change the status quo. Data, data science, and advanced analytics are going to be key, he states. The early adopters of technologies that improve efficiency are going to be the ones who win. We are living in an amazing time for healthcare. Our focus is on patients and human health. I just don't think there's a greater mission and purpose.

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Former Novartis CEO Cell And Gene Therapies Will Drive Innovation - Clinical Leader

Global CAR T-Cell Therapy Market 2019 Development Status, Competition Analysis and Application 2024 – News Adopt

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For competitor segment, the report includes globalkey playersofCAR T-Cell Therapyas well as some small players covering:Celgene (Juno Therapeutics), Novartis, Gilead (Kite Pharma), Pfizer, CARsgen Therapeutics, Autolus Therapeutics, Aurora BioPharma, Sorrento Therapeutics, Mustang Bio, Bluebird Bio, Collectis, Allogene Therapeutics, Celyad

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Global CAR T-Cell Therapy Market 2019 Development Status, Competition Analysis and Application 2024 - News Adopt

Early Results Are Positive for Experimental CRISPR Therapies – The Scientist

The first patients in each of two early-stage clinical trials testing CRISPR-based treatments for inherited blood disorders have been symptom free for several months with normalized hemoglobin levels, the companies developing the therapies announced today (November 19). Both participants experienced only treatable, temporary side effects, according to the data.

Last fall, Switzerland-headquartered CRISPR Therapeutics and Vertex Pharmaceuticals in Boston teamed up to launch a trial in Germany testing a CRISPR-based approach to treating -thalassemia. The therapy, known as CTX001, is applied to blood stem cells removed from the patient to cleave the BCL11A gene that represses the production of fetal hemoglobin. These cells are then reinfused to provide a healthy supply this protein, normally only produced in infancy, to overcome problems associated with mutations in the gene encoding the adult version. A few months later, the two companies launched a parallel trial in Nashville, Tennessee, to test CTX001 in sickle cell patients, who also suffer from mutations in the gene for adult hemoglobin.

The data released this week pertains to the first patients treated, one in each of these trials. In both participants, the CRISPRed cells successfully homed to the bone marrow. In the nine months since she received the treatment, the -thalassemia patient has required no blood transfusions, which she had needed regularly for 16 years, and her total hemoglobin levels are near-normal, STATreports. Meanwhile, the sickle-cell patient has not suffered any of the painful and organ-damaging events known as vaso-occlusive crises, where the misshapen blood cells characteristic of the disease stick to and clog small blood vessels, since she received her treatment in July, and her total hemoglobin levels have also normalized, according to STAT.

We are very, very excited, Haydar Frangoul, the treating physician at the Sarah Cannon Research Institute in Nashville, tells NPR. This preliminary data shows for the first time that gene editing has actually helped a patient with sickle cell disease. This is definitely a huge deal.

Both patients experienced only minimal side effects, which the treating physicians attributed to the drug busulfan, used to wipe out the patients mutant bone marrow cells before receiving the infusion of CRISPRed blood stem cells.

These results are remarkable because they represent the first clinical evidence that CRISPR-Cas9 has real curative potential, Jeff Leiden, the president and chief executive officer of Vertex Pharmaceuticals, tells STAT. Vertex and CRISPR Therapeutics say they will now proceed with enrolling a total of 45 patients in each trial.

Jef Akst is managing editor ofThe Scientist. Email her atjakst@the-scientist.com.

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Early Results Are Positive for Experimental CRISPR Therapies - The Scientist

CHMP recommends EMA approval of polatuzumab vedotin for the treatment of adult patients with R/R DLBCL – Lymphoma Hub

On the 14th November 2019, the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) recommended the approval of polatuzumab vedotin, in combination with bendamustine and rituximab (BR), for the treatment of adult patients with relapsed/refractory (R/R) diffuse large B-cell lymphoma (DLBCL) who are unsuitable for a hematopoietic stem cell transplant.1 Polatuzumab vedotin is a first-in-class anti-CD79b antibody-drug conjugate, which kills malignant B cells by delivering anti-mitotic monomethyl auristatin E.2

The combination was approved earlier this year by the U.S. Food and Drug Administration(FDA) for the treatment of patients with DLBCL (read more here).

The CHMPs positive opinion was based on data from the global phase Ib/II study GO29365 (NCT02257567) that evaluated the safety, tolerability, and activity of polatuzumab vedotin in combination with BR or obinutuzumab in R/R follicular lymphoma or DLBCL. The polatuzumab vedotin-BR combination achieved a complete response rate of 40% (n= 16/40) compared with 17.5% (n= 7/40) with BR alone, and a median survival of 12.4 months versus 4.7 months, respectively.3,4

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CHMP recommends EMA approval of polatuzumab vedotin for the treatment of adult patients with R/R DLBCL - Lymphoma Hub

CStone announces first patient dosed in the Phase I bridging registrational study of ivosidenib – PRNewswire

SUZHOU, China, Nov. 19, 2019 /PRNewswire/ -- CStone Pharmaceuticals ("CStone" or the "Company", HKEX: 2616)today announced that the first patient has been dosed in the Phase I bridging registrational study of ivosidenib (TIBSOVO) in China. This stand-alone trial is designed to validate the efficacy, safety, and pharmacokinetics of ivosidenib in patients with IDH1 mutant relapsed or refractory acute myeloid leukemia (R/R AML).

Developed by CStone's partner, Agios Pharmaceuticals (NASDAQ: AGIO), ivosidenib was approved by the U.S. FDA in July 2018 for the treatment of adult patients with R/R AML with a susceptibleIDH1 mutation as detected by an FDA-approved test. In May 2019, CStone submitted a new drug application (NDA) for ivosidenib in Taiwan for the treatment of adult patients with IDH1 mutant R/R AML.

Current standard of care treatment for newly diagnosed AML patients mainly includes intensive induction chemotherapy (IC), followed by consolidation therapy such as allogeneic hematopoietic stem cell transplantation (Allo-HSCT) in order to attain durable remission. Approximately 35% to 40% of those treated patients achieve complete remission, while only about 25% achieve 3 years or longer survival. The majority of AML patients develop acquired resistance to treatment or eventually relapse, leading to R/R AML, which has a very poor prognosis in the absence of standard of care treatment options globally. With the emergence of DNA sequencing technology, the detection of genetic mutations has presented new opportunities and challenges in AML treatment. IDH1 mutations are associated with around 6% to 10% of all AML cases.

Dr. Frank Jiang, Chairman and CEO of CStone, commented: "AML is the most common acute leukemia affecting adults with over 30,000 new cases estimated in China every year. AML is characterized by its rapid progression witha five-year survival rate below 20%. We are faced with the urgent need for clinical development, particularly for IDH1 mutant R/R AML patients, due to the lack of any effective treatment in China. We will rigorously press ahead with the clinical development of ivosidenib to achieve its regulatory approval in China which will allow more AML patients in Greater China to benefit from this precision therapy."

CStone's Chief Medical Officer, Dr. Jason Yang, noted: "Ivosidenib is a potent and highly selective IDH1 inhibitor, and the only targeted therapy currently approved by the U.S. FDA for IDH1 mutant AML. It is very encouraging that we have already initiated two registrational studies of ivosidenib in China, including the global Phase III AGILE study of ivosidenib in combination with azacitidinein adult patients with newly diagnosed IDH1 mutant AML who are not eligible for intensive chemotherapy."

About TIBSOVO (ivosidenib)

TIBSOVO is indicated for the treatment of acute myeloid leukemia (AML) with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation as detected by an FDA-approved test in:

IMPORTANT SAFETY INFORMATION

WARNING: DIFFERENTIATION SYNDROME

Patients treated with TIBSOVO have experienced symptoms of differentiation syndrome, which can be fatal if not treated. Symptoms may include fever, dyspnea, hypoxia, pulmonary infiltrates, pleural or pericardial effusions, rapid weight gain or peripheral edema, hypotension, and hepatic, renal, or multi-organ dysfunction. If differentiation syndrome is suspected, initiate corticosteroid therapy and hemodynamic monitoring until symptom resolution.

WARNINGS AND PRECAUTIONS

Differentiation Syndrome: See Boxed WARNING. In the clinical trial, 25% (7/28) of patients with newly diagnosed AML and 19% (34/179) of patients with relapsed or refractory AML treated with TIBSOVO experienced differentiation syndrome. Differentiation syndrome is associated with rapid proliferation and differentiation of myeloid cells and may be life-threatening or fatal if not treated. Symptoms of differentiation syndrome in patients treated with TIBSOVO included noninfectious leukocytosis, peripheral edema, pyrexia, dyspnea, pleural effusion, hypotension, hypoxia, pulmonary edema, pneumonitis, pericardial effusion, rash, fluid overload, tumor lysis syndrome, and creatinine increased. Of the 7 patients with newly diagnosed AML who experienced differentiation syndrome, 6 (86%) patients recovered. Of the 34 patients with relapsed or refractory AML who experienced differentiation syndrome, 27 (79%) patients recovered after treatment or after dose interruption of TIBSOVO. Differentiation syndrome occurred as early as 1 day and up to 3 months after TIBSOVO initiation and has been observed with or without concomitant leukocytosis.

If differentiation syndrome is suspected, initiate dexamethasone 10 mg IV every 12 hours (or an equivalent dose of an alternative oral or IV corticosteroid) and hemodynamic monitoring until improvement. If concomitant noninfectious leukocytosis is observed, initiate treatment with hydroxyurea or leukapheresis, as clinically indicated. Taper corticosteroids and hydroxyurea after resolution of symptoms and administer corticosteroids for a minimum of 3 days. Symptoms of differentiation syndrome may recur with premature discontinuation of corticosteroid and/or hydroxyurea treatment. If severe signs and/or symptoms persist for more than 48 hours after initiation of corticosteroids, interrupt TIBSOVO until signs and symptoms are no longer severe.

QTc Interval Prolongation:Patients treated with TIBSOVOcan develop QT (QTc) prolongation and ventricular arrhythmias. One patient developed ventricular fibrillation attributed to TIBSOVO. Concomitant use of TIBSOVOwith drugs known to prolong the QTc interval (e.g., anti-arrhythmic medicines, fluoroquinolones, triazole anti-fungals, 5-HT3 receptor antagonists) and CYP3A4 inhibitors may increase the risk of QTc interval prolongation. Conduct monitoring of electrocardiograms (ECGs) and electrolytes. In patients with congenital long QTc syndrome, congestive heart failure, or electrolyte abnormalities, or in those who are taking medications known to prolong the QTc interval, more frequent monitoring may be necessary.

Interrupt TIBSOVOif QTc increases to greater than 480 msec and less than 500 msec. Interrupt and reduce TIBSOVOif QTc increases to greater than 500 msec. Permanently discontinue TIBSOVOin patients who develop QTc interval prolongation with signs or symptoms of life-threatening arrhythmia.

Guillain-Barr Syndrome:Guillain-Barr syndrome occurred in <1% (2/258) of patients treated with TIBSOVO in the clinical study. Monitor patients taking TIBSOVO for onset of new signs or symptoms of motor and/or sensory neuropathy such as unilateral or bilateral weakness, sensory alterations, paresthesias, or difficulty breathing. Permanently discontinue TIBSOVO in patients who are diagnosed with Guillain-Barr syndrome.

ADVERSE REACTIONS

DRUG INTERACTIONS

Strong or Moderate CYP3A4 Inhibitors:Reduce TIBSOVOdose with strong CYP3A4 inhibitors. Monitor patients for increased risk of QTc interval prolongation.

Strong CYP3A4 Inducers:Avoid concomitant use with TIBSOVO.

Sensitive CYP3A4 Substrates:Avoid concomitant use with TIBSOVO.

QTc Prolonging Drugs:Avoid concomitant use with TIBSOVO. If co-administration is unavoidable, monitor patients for increased risk of QTc interval prolongation.

LACTATION

Because many drugs are excreted in human milk and because of the potential for adverse reactions in breastfed children, advise women not to breastfeed during treatment with TIBSOVOand for at least 1 month after the last dose.

Please see full Prescribing Information, including Boxed WARNING.

About CStone

CStone Pharmaceuticals (HKEX:2616) is a biopharmaceutical company focused on developing and commercializing innovative immuno-oncology and precision medicines to address the unmet medical needs of cancer patients in China and worldwide. Established in 2015, CStone has assembled a world-class management team with extensive experience in innovative drug development, clinical research, and commercialization. The company has built an oncology-focused pipeline of 15 drug candidates with a strategic emphasis on immuno-oncology combination therapies. Currently, five late-stage candidates are at or near pivotal trials. With an experienced team, a rich pipeline, a robust clinical development-driven business model and substantial funding, CStone's vision is to become globally recognized as a leading Chinese biopharmaceutical company by bringing innovative oncology therapies to cancer patients worldwide.

For more information about CStone Pharmaceuticals, please visit: http://www.cstonepharma.com.

Forward-looking Statement

The forward-looking statements made in this article relate only to the events or information as of the date on which the statements are made in this article. Except as required by law, we undertake no obligation to update or revise publicly any forward-looking statements, whether as a result of new information, future events or otherwise, after the date on which the statements are made or to reflect the occurrence of unanticipated events. You should read this article completely and with the understanding that our actual future results or performance may be materially different from what we expect. In this article, statements of, or references to, our intentions or those of any of our Directors or our Company are made as of the date of this article. Any of these intentions may alter in light of future development.

SOURCE CStone Pharmaceuticals

http://www.cstonepharma.com

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CStone announces first patient dosed in the Phase I bridging registrational study of ivosidenib - PRNewswire

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.

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

Kate and Flash riding high following injuries recovery – The Scarborough News

Katie Brickman and Flash both recovered from shocking injuries. Photo by Tony Bartholomew.

But vet Katie Brickman, from Welburn, near Malton, refused to give up when doctors told her a hand injury had ended her riding career.

She used the he same determination to bring her horse, Flash, back from an injury most competition horses would never recover from.

Katie, 29, who works at Minster Equine Veterinary Clinic in Ripon, suffered an horrific injury in March 2017 when she fell over while carrying a glass bottle of antibiotics, severing an artery in her left hand.

She was rushed to hospital in Hull, where she underwent two operations, but was told the damage was so serious, she would never be able to properly grip reins again and was advised to retire from riding.

Two months later, Katie suffered another devastating blow when Flash, her competition horse, suffered a serious injury when he ripped a ligament in his stifle joint at the top of his leg while in his field.

Flash was left critically lame by an injury that was so serious, he had to remain on box rest [confined to stable] for six months, and the prognosis was so bleak, he would have to be retired.

Katie and Flash have confounded expectations by making a miracle recovery and returned to competition this year in the world of eventing. Not only have they enjoyed their best-ever year with four wins, they have qualified for Badminton Grassroots Championships at one of the biggest events in the world equine sporting calendar.

Katie, who has been a vet with Minster Equine Veterinary Clinic at Ripon, York and Malton for six years, said: Flash and I have both gone from being told neither of us will never compete again to qualifying for Badminton Grassroots Championships. He has had an amazing season and its been our best ever year.

It goes to show you should never give up and all horses deserve a chance to recover from even the worst kind of injuries. I never gave up hope for either of us, but to reach Badminton is beyond my wildest dreams.

Flash is a very special horse because he had a tough start as I dont think he was treated well before I got him so hes very nervous.

It was really grim news about his injury as he as extremely lame, but I was determined to do everything to help him recover even though everything was stacked against him.

Katie still recovering from the severed artery, which left her hand in a bandage and cast, when Flash, a nine-year-old black Irish Sports Horse, suffered a freak injury in his field.

After six months of box rest, Katie took him for a bone scan at Rainbow Equine Hospital.

While she feared he would never compete in eventing again, she hoped Flash could at least be a pleasure hack.

As an experienced vet, she started giving him laser, shockwave and ultrasound treatment to the injured stifle joint, as well as stem cell therapy.

By August 2018, he was making such good progress, Katie decided to test out his recovery, as well as her own, by jumping and then this year started competing.

In October 2018, she competed at Askham Bryan College in York and won.

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Kate and Flash riding high following injuries recovery - The Scarborough News

I thought I had glandular fever on my gap year, but it turned out to be leukaemia – Telegraph.co.uk

It was meant to be the start of Tom Hunts gap year a fortnight in eastern Europe with five school friends. He had achieved an A* and two As in his A-levels and had an offer to study medicine at the University of Manchester. Before then, he wanted to do a ski season and travel around south-east Asia. But a week into his holiday, at the beginning of September last year, Tom was so ill he couldnt eat, drink or get out of bed. He booked a flight home from Prague.

When I picked him up from the airport, he looked dead, says Andrea, 51, his mother. He was really grey and smelt of bacteria.

Toms symptoms loss of appetite, fatigue, dizzy spells had all the hallmarks of glandular fever. But he also had a large, swollen haematoma on his knee, which had appeared after hours spent with his legs crossed on a train.

When I woke, it was killing me, just here, says Tom, 19, pointing at his thigh above his left knee. I thought it was a dead leg, but there was a massive lump and it was painful to walk.

Andrea thought Tom would get better with rest but within a week, having returned to the family home, he was hospitalised. The GP told Andrea: This lad needs tests and monitoring,because hes at risk of infection.

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I thought I had glandular fever on my gap year, but it turned out to be leukaemia - Telegraph.co.uk

Global Joint Pain Injections Market Growth Rate, Production Volume and Future Opportunities From 2019-2028 – Trade Examiner

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For veterans, theres no better cause to push than helping other vets – Snoqualmie Valley Record

Two veterans in East King County are just two of many working to help other veterans.

North Bend veteran Jim Curtis doesnt mince words. If he supports something, youll know it.

The same can be said about things hes not a fan of. Hes vocal about his disdain for President Donald Trump and his actions while in office. And politicians, in general, who use veterans as a means for election.

Politicians use us to get elected or re-elected, Curtis said. But after they accomplish that goal, its sayonara until its time for campaigning again.

He channels his ideas through poetry. Mostly, he pours the passion into helping other veterans.

The cause that had his attention on Oct. 24 was this years Mother Brundage Memorial Raffle. He was raising funds for the family of Andrew Yoder, a U.S. Marine Corps combat veteran who was killed when a crane crashed down in Seattle last spring. The raffle would happen a few weeks later, on Nov. 10, the Marine Corps birthday.

Curtis sold tickets for $5 at the North Bend QFC and Mount Si Sports and Fitness. Even as he traveled south to the American Lake VA Medical Center in his red, 2011 Ford Ranger truck, he pushed for others to purchase raffle tickets.

When he approached the check-in desk, after briefly responding to a question prompted by the man at the counter, he popped his own.

Were doing this raffle, he often began his pitch. Curtis would often end his interactions with the words Semper Fi, the Marine Corps motto and short for semper fidelis a Latin phrase that translates to mean always faithful.

Semper Fi, hed say before leaving.

He used similar words each time, having memorized the phrases he echoed to try and convince the listener on the importance of the raffle. And, of course, to shell out some cash to help.

But what could one expect? When it came to serving especially when Curtis did, during the highly-controversial Vietnam War it was other veterans who understood. And it was other veterans who offered their support. Even if that support meant hanging flyers in a VA hospital nearly 55 miles south from Curtiss North Bend home. Or when it meant reaching out to politicians again. And again. And again.

Other causes

Before he began fundraising for Yoders family, Curtis was busy commuting south to Olympia, where he testified before state House and Senate committees. He thought Purple Heart license plates, those given to wounded military or family of those killed in action, should be issued free of payment of any fees or taxes. He had his own Purple Heart, after shrapnel from a mortar blast flew into his back, legs and derriere. This happened in 1969, during his first year in Vietnam, where he was a machine gunner.

Curtis presented the idea to Democrat Paul Graves, at the time house representative for District 5. When Lisa Callan was elected into the seat in November 2018, after defeating Graves by more than 3,000 votes, she approached him on his idea. She had met him previously while doorbelling in the area.

(The change) was the very least of what we could do to say Thank you, Callan said by phone, on Veterans Day. It gives veterans a way to share their story by even having that plate We need to do so much more for those who receive the Purple Heart and their families.

Curtis has other causes now. Hes invited Rep. Callan to visit the VA hospital he visits regularly, to see the condition of the buildings and to hopefully get some kind of change. The facilities are in a degraded state, compared to the state of medical buildings elsewhere, Curtis said. The walls have dark scuff marks, in some spots reaching about a foot in height. And for veterans, who sacrificed their time and had their lives on the line, it just wasnt up to par. Callan hasnt yet paid a visit to the hospital, but said that was in the works.

His other effort entails getting Gregory Pappy Boyingtons name recognized somewhere along the Medal of Honor Memorial Highway. Boyington was a graduate of Lincoln High School in Tacoma and an American combat pilot.He received the honor, the highest and most prestigious, after he spent months shy of two years in a Japanese prison camp.

There are plaques honoring vets there, but no Pappy in sight, Curtis said.

For another veteran, Curtis is simply looking for answers on why stem cell therapy is given to active-duty military at the Walter Reed National Military Medical Center in Bethesda, Maryland, and not an option for a disabled veteran Curtis knows in North Bend. The center serves military beneficiaries in the Washington, D.C. area as well as those from across the country and around the globe, according to its website.

He has contacted Congresswoman Kim Schrier about the issue, but has so far been having back-and-forth conversations with her staff via email, staff who, Curtis said, say shes too busy to leave her office. He figured the doctor he knocked on doors for, who at some point worked at Virginia Mason Medical Center in Issaquah, would be willing to make the drive, considering its not that far a commute, he said. Schrier did not return a request for comment by press deadline.

Gorman

But its not only Curtis helping other veterans. Marine veteran and Redmond resident Mark Gorman has been working to locate safe spaces for veterans without homes to sleep. He started the effort in 2004, when he owned a shop in Kingston. Everyday he spotted an older man fishing. And after that first initial contact, he struck up a friendship with the homeless man.

At one point, he just wasnt there fishing anymore, Gorman said. Three and four weeks went by, and Gorman decided to find the mans hide the safe spot he had carved out for himself outdoors. This guys all alone, no family. He died on his own of malnutrition and his heart gave up.

In the U.S., on a single night in January 2018, there were more than 37,800 veterans experiencing homelessness, according to the U.S. Department of Veteran Affairs. Of that number on that same night, about 23,300 were living unsheltered. Although there was a 5.4 percent decrease from the previous year, in the estimated number derived from the point-in-time count, Gorman said theres still a need.

When he moved to the Eastside in 2013, he realized there was an even bigger need locally. He began to help veterans, who for different, complex reasons, had lost their housing. Some he could help. Others, he said, werent mentally ready for the world.

They camp out in tents, others in trailers, some sleep in cars in church parking lots. Others sleep in Gormans backyard.

Three things in life we do as veterans sacrifice, duty and selflessness, Gorman said of his reasoning for helping. If those three things are not instilled in a boy as a young man, he will never become a real man. A man sacrifices for not only family and self but also for others around him.

Someday

While at the VA that cloudy afternoon in October, Curtis struck up a conversation with a gentleman who was also pushing his own cause to help veterans. He had acquired small American flags from another, and pinned one to Curtiss lapel, on the opposite side of where his Purple Heart medal pin sat. In exchange, Curtis offered a poem he wrote for sergeant first class Nathan Chapman, the first soldier killed during the war in Afghanistan. He was from Puyallup.

Someday well talk about this war, just like those weve had before, was one line he recited in the waiting room. And the spectator, in response, cried. The two shook hands and bonded over the shared experience of serving the country.

Curtis turned to walk away, but not before saying Semper Fi.

After a quick chat in the waiting room of the American Lake VA Medical Center, Jim Curtis, right, is given a small American flag pin by a fellow veteran. Curtis was there drawing attention to his fundraising efforts. Staff photos / Ashley Hiruko

A sign hangs on the bulletin board at the VA medical center for the Ocean Warriors Birthday Party. Curtis hung others, like these, in areas with many eyes to see.

On a foggy October morning, Curtis drives south toward the Tacoma area. He had his flyers in hand, and raffle tickets to sell. They would benefit the family of Andrew Yoder, a Marine Veteran who died in Seattle during the crane collapse this spring. Staff photo/Ashley Hiruko

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For veterans, theres no better cause to push than helping other vets - Snoqualmie Valley Record