ISSCA Conference at University of Miami Attracts Regenerative Medicine Experts and Physicians from across the Globe – PRUnderground

The International Society for Stem Cell Application (ISSCA), in collaboration with SISDET, held a highly successful three-day medical conference on the University of Miami campus on October 24-26. The conference featured a host of international experts in regenerative medicine and introduced new standards in regenerative medicine protocols to those in attendance. The Miami conference is part of the ISSCAs growing commitment to increasing the awareness and practice of regenerative medicine across the globe in an effort to help alleviate suffering for those diagnosed with degenerative diseases.

Around 200 physicians, scientists, and researchers interested in regenerative medicine traveled to the University of Miami campus for the event. The conference focused on providing attendees with information on todays most successful stem cells treatment protocols and the latest advances in regenerative medicine. Attendees heard from more than 20 expert speakers within the stem cells field, with lecturers from Europe, the US, and Latin America on the conference agenda.

This three-day event included recognized keynote speakers, as well as aspiring young physicians discussing the latest advances in stem cell biology in an informal and collaborative setting, said Benito Novas, Vice President of Public Relations for ISSCA. Our goal with all of our events is to strengthen the cooperative and dynamic spirit in this research area. We would also like to thank the University of Miami for hosting this event, as it was a great honor partnering with such a prestigious university.

ISSCA is a global leader in stem cells research, applications, and education, partnering with major global institutions and locations worldwide to host its independent medical congresses. To learn more about the ISSCA and its all of its past and upcoming events, visit http://www.issca.us

About International Society for Stem Cells Applications

The International Society for Stem Cells Applications (ISSCA) is a multidisciplinary community of scientists and physicians, all of whom aspire to treat diseases and lessen human suffering through advances in science, technology, and the practice of regenerative medicine. Incorporated under the Republic of Korea as a non-profit entity, the ISSCA is focused on promoting excellence and standards in the field of regenerative medicine.

ISSCA bridges the gaps between scientists and practitioners in Regenerative Medicine. Their code of ethics emphasizes principles of morals and ethical conducts.

At ISSCA, their vision is to take a leadership position in promoting excellence and setting standards in the regenerative medicine fields of publication, research, education, training, and certification. ISSCA serves its members through advancements made to the specialty of regenerative medicine. They aim to encourage more physicians to practice regenerativemedicine and make it available to benefit patients both nationally and globally.

For more information, please visit https://www.issca.us/ or send an email to info@stemcellsgroup.com

Read the rest here:
ISSCA Conference at University of Miami Attracts Regenerative Medicine Experts and Physicians from across the Globe - PRUnderground

How maternal Zika infection results in newborn microcephaly – Baylor College of Medicine News

The current study was initiated when a patient presented with a small brain size at birth and severe abnormalities in brain structures at the Baylor Hopkins Center for Mendelian Genomics (CMG), a center directed by Dr. Jim Lupski, professor of pediatrics, molecular and human genetics at Baylor College of Medicine and attending physician at Texas Childrens Hospital, said Dr. Hugo J. Bellen, professor at Baylor, investigator at the Howard Hughes Medical Institute and Jan and Dan Duncan Neurological Research Institute at Texas Childrens Hospital.

This patient and others in a cohort at CMG had not been infected by Zika virus in utero. They had a genetic defect that caused microcephaly. CMG scientists determined that the ANKLE2 gene was associated with the condition. Interestingly, a few years back the Bellen lab had discovered in the fruit fly model that ANKLE2 gene was associated with neurodevelopmental disorders. Knowing that Zika virus infection in utero can cause microcephaly in newborns, the team explored the possibility that Zika virus was mediating its effects in the brain via ANKLE2.

In a subsequent fruit fly study, the researchers demonstrated that overexpression of Zika protein NS4A causes microcephaly in the flies by inhibiting the function of ANKLE2, a cell cycle regulator that acts by suppressing the activity of VRK1 protein.

Since very little is known about the role of ANKLE2 or VRK1 in brain development, Bellen and his colleagues applied a multidisciplinary approach to tease apart the exact mechanism underlying ANKLE2-associated microcephaly.

The team found that fruit fly larvae with mutations in ANKLE2 gene had small brains with dramatically fewer neuroblasts brain cell precursors and could not survive into adulthood. Experimental expression of the normal human version of ANKLE2 gene in mutant larvae restored all the defects, establishing the loss of Ankle2 function as the underlying cause.

To understand why ANKLE2 mutants have fewer neuroblasts and significantly smaller brains, we probed deeper into asymmetric cell divisions, a fundamental process that produces and maintains neuroblasts, also called neural stem cells, in the developing brains of flies and humans, said first author Dr. Nichole Link, postdoctoral associate in the Bellen lab.

Asymmetric cell division is an exquisitely regulated process by which neuroblasts produce two different cell types. One is a copy of the neuroblast and the other is a cell programmed to become a different type of cell, such as a neuron or glia.

Proper asymmetric distribution and division of these cells is crucial to normal brain development, as they need to generate a correct number of neurons, produce diverse neuronal lineages and replenish the pool of neuroblasts for further rounds of division.

When flies had reduced levels of Ankle2, key proteins, such as Par complex proteins and Miranda, were misplaced in the neuroblasts of Ankle2 larvae. Moreover, live imaging analysis of these neuroblasts showed many obvious signs of defective or incomplete cell divisions. These observations indicated that Ankle2 is a critical regulator of asymmetric cell divisions, said Link.

Further analyses revealed more details about how Ankle2 regulates asymmetric neuroblast division. They found that Ankle2 protein interacts with VRK1 kinases, and that Ankle2 mutants alter this interaction in ways that disrupt asymmetric cell division.

Linking our findings to Zika virusassociated microcephaly, we found that expressing Zika virus protein NS4A in flies caused microcephaly by hijacking the Ankle2/VRK1 regulation of asymmetric neuroblast divisions. This offers an explanation to why the severe microcephaly observed in patients with defects in the ANKLE2 and VRK1 genes is strikingly similar to that of infants with in utero Zika virus infection, Link said.

For decades, researchers have been unsuccessful in finding experimental evidence between defects in asymmetric cell divisions and microcephaly in vertebrate models. The current work makes a giant leap in that direction and provides strong evidence that links a single evolutionarily conserved Ankle2/VRK1 pathway as a regulator of asymmetric division of neuroblasts and microcephaly, Bellen said. Moreover, it shows that irrespective of the nature of the initial triggering event, whether it is a Zika virus infection or congenital mutations, the microcephaly converges on the disruption of Ankle2 and VRK1, making them promising drug targets.

Another important takeaway from this work is that studying a rare disorder (which refers to those resulting from rare disease-causing variations in ANKLE2 or VRK1 genes) originally observed in a single patient can lead to valuable mechanistic insights and open up exciting therapeutic possibilities to solve common human genetic disorders and viral infections.

Others who contributed in this study are Hyunglok Chung, Angad Jolly, Marjorie Withers, Burak Tepe, Benjamin R. Arenkiel, Priya S. Shah, Nevan J. Krogan, Hatip Aydin, Bilgen B. Geckinli, Tulay Tos, Sedat Isikay, Beyhan Tuysuz, Ganesh H. Mochida, Ajay X. Thomas, Robin D. Clark and Ghayda M. Mirzaa. They are affiliated to one or more of the institutions: Baylor College of Medicine, Texas Childrens Hospital and the Jan and Dan Duncan Neurological Research Institute in Houston, TX; University of California at Davis and San Francisco; Zeynep Kamil Maternity and Children's Training and Research Hospital, Istanbul, Turkey; Marmara University School of Medicine, Istanbul, Turkey; Dr. Sami Ulus Research and Training Hospital of Women's and Children's Health and Diseases, Ankara, Turkey; Boston Childrens Hospital; Harvard Medical School, Boston, MA; Massachusetts General Hospital, Boston, MA; Loma Linda University Medical Center, Loma Linda, CA; University of Washington, Seattle, WA; and Seattle Children's Research Institute, Seattle, WA.

The study was funded by the National Institutes of Healths F32NS092270, NIH/NINDS R35NS105078, NIH U54NS093793, NIH R24OD022005, NIH/NINDS K08NS092898, Howard Hughes Medical Institute (HHMI), Medical Research Fellowship, Jordans Guardian Angels, a jointly funded NHGRI and NHLBI grant to the Baylor-Hopkins Center for Mendelian Genomics (UM1 HG006542) and the Huffington Foundation.

Read the rest here:
How maternal Zika infection results in newborn microcephaly - Baylor College of Medicine News

Next generation cell and gene therapies: fine tuning the promise – Business Weekly

On 19 November, the UK BioBeat19 summit goes to Stevenage to discuss the potential of cell and gene therapy and how to accelerate these transformational medicines.

Victoria Higgins of GSK and Miranda Weston-Smith from BioBeat spoke to two panellists who gave a sneak peek of their remarks and agree wholeheartedly that the discovery side and clinical side work best when they are teamed up.

Sophie Papa, an oncologist at Guys Cancer at Guys and St Thomas NHS Trust, and Aisha Hasan, a clinical development lead at GSK, both recognise the big challenge ahead for cell therapy researchers: to dial up efficacy and dial down toxicity.

Cell and gene therapies, with their remarkable potential to transform medicine, have seen some important but hard-won milestones: it took 20 years of combined academic and industry research to deliver the first gene therapy approval in 2016 and today there are two CAR-Ts approved for haematological malignancies.

Whilst CAR-Ts recognise proteins expressed on the tumour cell surface, making them ideal for targeting blood cancers, more complicated but with greater potential to address solid tumours are the gene modified TCR-T technologies.

These harness the power of T cells to specifically target and destroy tumours even on the inside of cells. TCR-Ts come with an additional level of complexity, but potentially open the door to a range of untreatable cancer types.

Looking at the TCR opportunity is where Sophie Papa sees the inherent trade-off between risk and benefit as an academic clinician whos now evaluating modified T-cell based therapies in clinical trials.

Sophie urges her peers to take courage. It is important to be brave and tolerant of certain toxicities. Academic clinicians and drug researchers need to work closely together to engage the regulators in early discussion, so that we can move cell therapies earlier in treatment schedules as soon as feasible.

Timing is critical to enable patients to be treated when they are physically fit so they can better tolerate these complex and potentially toxic treatments.

From her perspective, this is not an either/or, but an area where discussion and open dialogue will allow us to make the most of the opportunity. By allowing clinical academics to play a lead role in developing guidelines to manage patient safety, we can address legitimate concerns but not let them stand in the way of clinical development, she says.

Aisha brings the perspective of drug discovery and development and starts by asking what is in the realm of the possible from a design perspective.

She says: A superior T-cell therapy will require engineering approaches that enhance efficacy on one-end while also incorporating switches to minimise toxicity.

For example, in a counter-intuitive way, a T-cell with high-killing capacity actually can create dangerous levels of inflammation in the body, due to the rapid death of cancer cells. But the beauty of drug design opens up options:By building a switch within the engineered T-cells, researchers can inactivate the T-cells and prevent harm to the patient, says Aisha.

But this creative problem solving requires open dialogue between clinicians and pharma. Aisha says: The more we talk about clinical need and toxicity benchmarks, the more sophisticated we can be when developing the next generation of enhanced engineered cell therapies.

Theres no doubt that the challenges of delivering cell and gene therapy span the full spectrum of issues related to medicine development. However, the potential for both curative therapy and commercial opportunity is tremendous.

The scientific, clinical, technical, regulatory and commercial challenges are all surmountable when everyone in the ecosystem work together towards a shared goal, united by an unwavering focus on the patient.

Sophie and Aisha are speaking about the translational journey from science to bedside at the BioBeat19 summit.

The BioBeat19 summit on Accelerating cell and gene therapy, 1-6pm, Tuesday 19 November, GSK Stevenage. Guarantee your place by registering at http://www.biobeat19.org

See the original post here:
Next generation cell and gene therapies: fine tuning the promise - Business Weekly

110-year-olds live so long thanks to ‘super’ immune systems: study – New York Post

If you think daily exercise and a healthy diet were the key to a long life, think again.

Scientists say that the secret to living more than 100 years comes down to a hardy immune system, thanks to an abundance of a particular infection-fighting white blood cell.

In a study coordinated by scientists at Japans RIKEN Center for Integrative Medical Sciences (IMS) and Keio University School of Medicine, researchers discovered that supercentenarians those aged over 110 years have an excess of cytotoxic CD4 T-cells.

These super immune system cells, according to the study published in Proceedings of the National Academy of Sciences (PNAS), are more aggressive and known to kill any damaged cell that crosses its path, such as virus-infected or cancer cells.

We believe that this type of cells, which are relatively uncommon in most individuals, even young, are useful for fighting against established tumors, and could be important for immunosurveillance, said Piero Carninci, deputy director of RIKEN, in a statement. This is exciting as it has given us new insights into how people who live very long lives are able to protect themselves from conditions such as infections and cancer.

Scientists noticed that most of Japans supercentenarians had managed to dodge illness most of their lives, leading them to believe their advanced age might have something to do with their extraordinary immune systems.

To find out, they pulled a total of 41,208 immune cell samples from seven supercentenarians, and 19,994 cells from younger individuals ages 50 to 89. They found that while both groups had about the same number of T-cells altogether, the supercentenarians had an excess of the unique cytotoxic CD4 T-cells.

This finding might help explain why so many centenarians will say that drinking booze regularly didnt stop them from reaching 100. Others, though, credit a life without the stress of marriage or children as helping them to outlast their peers.

Amparo Perez, 105, told The Post she doesnt regret never remarrying when her first husband died. No aggravation, she said, [is] the most important thing, not to have aggravation.

Caroline Binns, 101, would agree that husbands were only trouble. She told The Post last year, Id rather be left in peace, not in pieces.

Her friend, 101-year-old Lucille Watson, said dancing and cheesecake inspires her to get out of bed every morning: Lifes pleasures are meant to be enjoyed.

Read more from the original source:
110-year-olds live so long thanks to 'super' immune systems: study - New York Post

Halting the progression of multiple sclerosis by blocking harmful B cells – FierceBiotech

The blood-brain barrier in healthy people is a powerful shield that protects neurons from harmful invaders. But in people with multiple sclerosis (MS), that shield malfunctions, allowing B cells from the immune system to pass into the brain and destroy healthy tissues.

Scientists at the University of Montreal Hospital Research Centre (CRCHUM) have identified a new target that they suggest could be exploited to slow down the flow of B cells into the brains of people with MS. They reported the discovery in the journal Science Translational Medicine.

B cells produce a substance called activated leukocyte cell adhesion molecule (ALCAM) that allows them to migrate into the brain via blood vessels, the researchers found. Blocking ALCAM in mouse models of MS reduced the flow of B cells into the brain and slowed the progression of the disease, they reported.

Like this story? Subscribe to FierceBiotech!

Biopharma is a fast-growing world where big ideas come along every day. Our subscribers rely on FierceBiotech as their must-read source for the latest news, analysis and data in the world of biotech and pharma R&D. Sign up today to get biotech news and updates delivered to your inbox and read on the go.

RELATED: How new gene discoveries could guide precision medicine in multiple sclerosis

B cells are a major culprit in progressive MS, the most severe form of the disease, and there are drugs on the market designed to deplete them, including Roches Ocrevus. Novartis is in phase 3 trials of Arzerra (ofatumumab), a drug that eliminates B cells by binding to the surface protein CD20. Arzerra is approved to treat chronic lymphocytic leukemia, but Novartis has been gunning for a bigger market opportunity. In September, it released new phase 3 data showing that Arzerra reduced MS relapse rates by more than 50% when compared to Sanofis Aubagio.

Meanwhile, several academic teams are looking to genetics as a path to personalized MS treatment strategies. In October, researchers at Johns Hopkins reported that newly discovered variants in the genes C1, CR1 and C1QA are associated with vision loss in progressive MS. They believe further research into these complement genes could lead to the development of new MS therapies.

Blocking ALCAM could also offer a promising strategy for thwarting B cells in MS, said University of Montreal Professor Alexandre Prat, Ph.D., in a statement. "The molecule ALCAM is expressed at higher levels on the B cells of people with multiple sclerosis, he said. By specifically targeting this molecule, we will now be able to explore other therapeutic avenues for the treatment of this disease."

See the article here:
Halting the progression of multiple sclerosis by blocking harmful B cells - FierceBiotech

Editas Medicine Announces Third Quarter 2019 Results and Update – Yahoo Finance

Amended Celgene collaboration to focus on engineered alpha-beta T cell medicines with a $70 million payment to Editas Medicine

Appointed Judith R. Abrams, M.D., as Chief Medical Officer

EDIT-101 (AGN-151587) for LCA10 first patient dosing expected by early 2020

EDIT-301 for hemoglobinopathies in vivo pre-clinical data to be presented at ASH

CAMBRIDGE, Mass., Nov. 12, 2019 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (EDIT), a leading genome editing company, today reported business highlights and financial results for the third quarter of 2019.

"Our momentum in 2019 remains strong in advancing our pipeline of in vivo CRISPR and engineered cell medicines," said Cynthia Collins, Chief Executive Officer of Editas Medicine. We announced this morning an amended agreement with Celgene to further expand and accelerate our oncology pipeline. In hemoglobinopathies, we look forward to presenting in vivo pre-clinical data for EDIT-301 at ASH that supports its potential as a best-in-class medicine. Finally, we eagerly anticipate first patient dosing with EDIT-101 for LCA10 in the coming months.

Recent Achievements and OutlookIn VivoCRISPR Medicines

Engineered Cell Medicines

Corporate

Upcoming Events

Editas Medicine will participate in the following investor events:

Editas Medicine will present pre-clinical data for EDIT-301 to address sickle cell disease and beta-thalassemia in at the 61st American Society of Hematology Annual Meeting & Exposition. Details are as follows:

Abstract Number: 4636Title: EDIT-301: An Experimental Autologous Cell Therapy Comprising Cas12a-RNP Modified mPB-CD34+ Cells for the Potential Treatment of SCDPresenter: Edouard De Dreuzy, Ph.D.Session: 801. Gene Therapy and Transfer: Poster III Time: Monday, December 9, 2019: 6:00 PM-8:00 PMLocation: Hall B, Orange County Convention Center, Orlando, FL

Third Quarter 2019 Financial Results

Cash, cash equivalents, and marketable securities at September 30, 2019, were $332.6 million, compared to $369.0 million at December 31, 2018. The $36.4 million decrease was primarily attributable to operating and capital expenses related to our on-going preclinical and clinical activities, patent costs and license fees, and employee-related costs, partially offset by $42.1 million in proceeds from financing activities.

For the three months ended September 30, 2019, net loss was $32.9 million, or $0.66 per share, compared to $15.2 million, or $0.32 per share, for the same period in 2018.

Conference Call

The Editas Medicine management team will host a conference call and webcast today at 8:00 a.m. ET to provide and discuss a corporate update and financial results for the third quarter of 2019. To access the call, please dial 844-348-3801 (domestic) or 213-358-0955 (international) and provide the passcode 6577216. A live webcast of the call will be available on the Investors & Media section of the Editas Medicine website at http://www.editasmedicine.com and a replay will be available approximately two hours after its completion.

About Editas MedicineAs a leading genome editing company, Editas Medicine is focused on translating the power and potential of the CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a) genome editing systems into a robust pipeline of treatments for people living with serious diseases around the world. Editas Medicine aims to discover, develop, manufacture, and commercialize transformative, durable, precision genomic medicines for a broad class of diseases. For the latest information and scientific presentations, please visit http://www.editasmedicine.com.

About EDIT-101 (AGN-151587)EDIT-101 is a CRISPR-based experimental medicine under investigation for the treatment of Leber congenital amaurosis 10 (LCA10). EDIT-101 is administered via a subretinal injection to reach and deliver the gene editing machinery directly to photoreceptor cells.

About Leber Congenital AmaurosisLeber congenital amaurosis, or LCA, is a group of inherited retinal degenerative disorders caused by mutations in at least 18 different genes. It is the most common cause of inherited childhood blindness, with an incidence of two to three per 100,000 live births worldwide. Symptoms of LCA appear within the first years of life, resulting in significant vision loss and potentially blindness. The most common form of the disease, LCA10, is a monogenic disorder caused by mutations in the CEP290 gene and is the cause of disease in approximately 2030 percent of all LCA patients.

Story continues

About the Editas Medicine-Allergan AllianceIn March 2017, Editas Medicine and Allergan Pharmaceuticals International Limited (Allergan) entered a strategic alliance and option agreement under which Allergan received exclusive access and the option to license up to five of Editas Medicines genome editing programs for ocular diseases, including EDIT-101 (AGN-151587). Under the terms of the agreement, Allergan is responsible for development and commercialization of optioned products, subject to Editas Medicines option to co-develop and share equally in the profits and losses of two optioned products in the United States. In August 2018, Allergan exercised its option to develop and commercialize EDIT-101 globally for the treatment of LCA10. Additionally, Editas Medicine exercised its option to co-develop and share equally in the profits and losses from EDIT-101 in the United States. Editas Medicine is also eligible to receive development and commercial milestones, as well as royalty payments on a per-program basis. The agreement covers a range of first-in-class ocular programs targeting serious, vision-threatening diseases based on Editas Medicines unparalleled CRISPR genome editing platform, including CRISPR/Cas9 and CRISPR/Cpf1 (also known as Cas12a).

Forward-Looking StatementsThis press release contains forward-looking statements and information within the meaning of The Private Securities Litigation Reform Act of 1995. The words anticipate, believe, continue, could, estimate, expect, intend, may, plan, potential, predict, project, target, should, would, and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Forward-looking statements in this press release include statements regarding the Companys plans with respect to the Brilliance Phase 1/2 clinical trial for EDIT-101 (AGN-151587), including the Companys expectations regarding the timing of dosing a patient by early 2020. The Company may not actually achieve the plans, intentions, or expectations disclosed in these forward-looking statements, and you should not place undue reliance on these forward-looking statements. Actual results or events could differ materially from the plans, intentions and expectations disclosed in these forward-looking statements as a result of various factors, including: uncertainties inherent in the initiation and completion of pre-clinical studies and clinical trials and clinical development of the Companys product candidates; availability and timing of results from pre-clinical studies and clinical trials; whether interim results from a clinical trial will be predictive of the final results of the trial or the results of future trials; expectations for regulatory approvals to conduct trials or to market products and availability of funding sufficient for the Companys foreseeable and unforeseeable operating expenses and capital expenditure requirements. These and other risks are described in greater detail under the caption Risk Factors included in the Companys most recent Quarterly Report on Form 10-Q, which is on file with the Securities and Exchange Commission, and in other filings that the Company may make with the Securities and Exchange Commission in the future. Any forward-looking statements contained in this press release speak only as of the date hereof, and the Company expressly disclaims any obligation to update any forward-looking statements, whether because of new information, future events or otherwise.

Investor ContactMark Mullikin(617) 401-9083mark.mullikin@editasmed.com

Media ContactCristi Barnett(617) 401-0113cristi.barnett@editasmed.com

Visit link:
Editas Medicine Announces Third Quarter 2019 Results and Update - Yahoo Finance

Treatment-Free Remission at Heart of New CML Study – AJMC.com Managed Markets Network

Maggie L. Shaw

Researchers aim to eliminate leukemia stem cells safely.

Its like removing the tree, but leaving the roots that can sprout new shoots, they said in a statement.

Because stem cells are responsible for cell self-renewal and differentiation, Lacorazza and his team set out to better understand the LSC self-renewal process, hoping to pinpoint possible new options to target that activity, prevent relapses, and lead to treatment-free remission. Their results appeared recently in Blood, the official publication of the American Society of Hematology.

Knowing that Krppel-like factor 4 (KLF4) plays an essential part in carcinogenesisalthough it has also been shown to have antitumor activitythe study investigators removed KLF4 to see if it was necessary also for LSCs to survive. The result? Loss of LSC/progenitor cells and increased levels of tyrosine-(Y)-phosphorylation-regulated kinase 2 (DYRK2) protein.

According to the authors, A major change in the absence of KLF4 was an increase in the production of kinase DYRK2, an enzyme involved in protein stability, cell cycle control, and apoptosis.

Stabilizing levels of this protein, then, could be a possible solution. To do this, they inhibited ubiquitin E3 ligase SIAH2 by introducing menadione (vitamin K3), as this has been shown to stimulate cell death in human CML stem/progenitor cells and increase levels of DYRK2. The drawback to this approach is that vitamin K3 can be toxic. To remedy this, the authors suggest SIAH2 inhibitors with lower hematological toxicity, evaluating the safety of that inhibition, and developing alternatives to activating DYRK2 in CML LSCs. In other words, stabilize or increase DYRK2 levels to inhibit LSCs.

Having set out to better their understanding of LSC renewal for new inroads to treatment-free survival and relapse prevention, Lacarozza and colleagues did just that. They identified the DYRK2 checkpoint in LSC/progenitor cell survival and self-renewal, showing there are 2 ways to increase its levels: (1) remove the Klf4 gene or (2) inhibit the ubiquitin ligase SIAH2 pharmacologically.

At present, they continue to search for ways to accomplish this that will not harm patients with CML, who today must take TKIs for life. We envision that targeting the bulk of leukemia with tyrosine kinase inhibitors plus a new drug that targets the stem cells might be a future strategy for patients to reach drug-free remission.

Reference

Park CS, Lewis A, Chen T, et al. KLF4 represses DYRK2 inhibition of self-renewal and survival through c-Myc and p53 in leukemia stem/progenitor cells [published online September 12, 2019]. Blood. doi: 10.1182/blood.2018875922.

Continue reading here:
Treatment-Free Remission at Heart of New CML Study - AJMC.com Managed Markets Network

The female problem: how male bias in medical trials ruined women’s health – The Guardian

From the earliest days of medicine, women have been considered inferior versions of men. In On the Generation of Animals, the Greek philosopher Aristotle characterised a female as a mutilated male, and this belief has persisted in western medical culture.

For much of documented history, women have been excluded from medical and science knowledge production, so essentially weve ended up with a healthcare system, among other things in society, that has been made by men for men, Dr Kate Young, a public health researcher at Monash University in Australia, tells me.

Youngs research has uncovered how doctors fill knowledge gaps with hysteria narratives. This is particularly prevalent when women keep returning to the doctor, stubbornly refusing to be saved.

The historical hysteria discourse was most often endorsed when discussing difficult women, referring to those for whom treatment was not helpful or who held a perception of their disease alternative to their clinician, Young wrote in a research paper published in the journal Feminism & Psychology.

Rather than acknowledge the limitations of medical knowledge, medicine expected women to take control (with their minds) of their disease (in their body) by accepting their illness, making lifestyle changes and conforming to their gendered social roles of wife and mother. Moralising discourses surround those who rebel; they are represented as irrational and irresponsible, the safety net for medicine when it cannot fulfil its claim to control the body.

In her work, Young has shown how endometriosis patients are often viewed by their treating doctors as reproductive bodies with hysterical tendencies. One gynaecologist said to Young: Do mad people get endo or does endo make you mad? Its probably a bit of both. Another said: Theres a lot of psychology, just as much as there is pathology [in gynaecology].

Nobody suggests that endometriosis is not a real disease, or is somehow imagined, but there is a general feeling in medicine that womens reaction to having endometriosis is somehow hysterical, especially when symptoms prevail after treatment has been offered, which is common. And it is not just endometriosis patients treated this way. One male GP said to me: Ive never had a fibromyalgia patient who wasnt batshit crazy.

Historically, Young says, men have made the medical science about women and their bodies, and there is an abundance of research evidence about the ways in which that knowledge has been constructed to reinforce the hysteria discourse and women as reproductive bodies discourse. One of my favourite examples is that in some of the first sketches of skeletons, male anatomy artists intentionally made womens hips look wider and their craniums look much smaller as a way of saying: Here is our evidence that women are reproductive bodies and they need to stay at home and we cant risk making them infertile by making them too educated, look how tiny their heads are. And we see that again and again.

Not only have doctors, scientists and researchers mostly been men, but most of the cells, animals and humans studied in medical science have also been male: most of the advances we have seen in medicine have come from the study of male biology. Dr Janine Austin Clayton, an associate director for womens health research at the United States National Institutes of Health (NIH), told the New York Times that the result is: We literally know less about every aspect of female biology compared to male biology.

Medicine has always seen women first and foremost as reproductive bodies. Our reproductive organs were the greatest source of difference to men and because they were different, they were mysterious and suspicious. But the fallout of this difference is that for a long time medicine assumed it was the only difference. Because women had reproductive organs, they should reproduce, and all else about them was deemed uninteresting.

In the early 20th century, the endocrine system, which produces hormones, was discovered. To medical minds, this represented another difference between men and women, overtaking the uterus as the primary perpetrator of all womens ills. Still, medicine persisted with the belief that all other organs and functions would operate the same in men and women, so there was no need to study women. Conversely, researchers said that the menstrual cycle, and varied release of hormones throughout the cycle in rodents, introduced too many variables into a study, therefore females could not be studied.

Diseases presenting differently in women are often missed or misdiagnosed, and those affecting mainly women remain largely a mystery: understudied, undertreated and frequently misdiagnosed or undiagnosed. This has major knock-on effects for both medical practice and the health of women.

As Young has argued: Medicine defines the female and male bodies as distinct but not equal; analyses of medical texts throughout history reveals the male body to be constructed as superior and the template against which bodies are judged. Any aspect of the female body that differs from the male or that cannot be given a male comparative (exemplified by the uterus) is viewed as evidence of deviation or fault.

Because women can bear children, medical discourse associated women with the body and men with the mind, a binary division that reinforces and is reinforced by the public-private division ... In addition to restricting womens public contribution, such beliefs provide medicine with an explanatory model of disease and illness in women: to deny ones biological destiny is to incite all manner of diseases, as Plato stated when theorising the wandering womb.

We see this in many predominantly female conditions: women with endometriosis are told that delayed childbearing causes the illness, or that pregnancy will cure it; women with breast cancer were once fed this line until advances in research (which only occurred because women campaigned for better knowledge and treatments) proved otherwise.

During the 80s, a group of female scientists in the US formed a society to campaign for better health research in women, now called the Society for Womens Health Research. They teamed up with some US Congress members to draw attention to the discrepancies in medical research and the effect on womens health.

In 1985, a report by the US Public Health Service Task Force on Womens Health warned that the historical lack of research focus on womens health concerns has compromised the quality of health information available to women as well as the health care they receive.

The campaign drew attention to some of the absurdities that resulted from this male bias, which Maya Dusenbery has summarised in her 2018 book Doing Harm: The Truth About How Bad Medicine and Lazy Science Leave Women Dismissed, Misdiagnosed and Sick. She notes that, in the early 60s: Observing that women tended to have lower rates of heart disease until their oestrogen levels dropped after menopause, researchers conducted the first trial to look at whether supplementation with the hormone was an effective preventive treatment. The study enrolled 8,341 men and no women ... And a National Institutes of Health-supported pilot study from Rockefeller University that looked at how obesity affected breast and uterine cancer didnt enrol a single woman.

And thats not all.

The Baltimore Longitudinal Study of Aging, which began in 1958 and purported to explore normal human ageing, didnt enrol any women for the first 20 years it ran. The Physicians Health Study, which had recently concluded that taking a daily aspirin may reduce the risk of heart disease? Conducted in 22,071 men and zero women. The 1982 Multiple Risk Factor Intervention Trial known, aptly enough, as MRFIT which looked at whether dietary change and exercise could help prevent heart disease: just 13,000 men.

The result of this male bias in research extends beyond clinical practice. Of the 10 prescription drugs taken off the market by the US Food and Drug Administration between 1997 and 2000 due to severe adverse effects, eight caused greater health risks in women. A 2018 study found this was a result of serious male biases in basic, preclinical, and clinical research.

The campaign had an effect in the US: in 1993, the FDA and the NIH mandated the inclusion of women in clinical trials. Between the 70s and 90s, these organisations and many other national and international regulators had a policy that ruled out women of so-called childbearing potential from early-stage drug trials.

The reasoning went like this: since women are born with all the eggs they will ever produce, they should be excluded from drug trials in case the drug proves toxic and impedes their ability to reproduce in the future.

The result was that all women were excluded from trials, regardless of their age, gender status, sexual orientation or wish or ability to bear children. Men, on the other hand, constantly reproduce their sperm, meaning they represent a reduced risk. It sounds like a sensible policy, except it treats all women like walking wombs and has introduced a huge bias into the health of the human race.

In their 1994 book Outrageous Practices, Leslie Laurence and Beth Weinhouse wrote: It defies logic for researchers to acknowledge gender difference by claiming womens hormones can affect study results for instance, by affecting drug metabolism but then to ignore these differences, study only men and extrapolate the results to women.

Since the 90s, more women have been included in clinical trials but researchers have not always analysed results by sex and/or gender. And though clinical studies have changed substantially, preclinical studies remained focused on male cell lines and male animals.

A 2010 study by Annaliese Beery and Irving Zucker reviewed sex bias in research on mammals in 10 biological fields during 2009 and their historical precedents. It found: Male bias was evident in eight disciplines and most prominent in neuroscience, with single-sex studies of male animals outnumbering those of females 5.5 to 1. In the past half-century, male bias in non-human studies has increased while declining in human studies. Studies of both sexes frequently fail to analyse results by sex. Under-representation of females in animal models of disease is also commonplace, and our understanding of female biology is compromised by these deficiencies.

The study also found the justification that researchers gave for excluding female animals that it introduced too much variability in results to be without foundation.

It took until 2014 for the NIH to begin to acknowledge the problem of male bias in preclinical trials, and until 2016 for it to mandate that any research money it granted must include female animals.

These policies and practices have often been framed as paternalistic, designed to protect women against the harmful effects of medical research. But history belies this notion. The practice of brutal experimentation of medical treatments on women throughout history makes medicines unwillingness to include women in scientific studies seem a lot less like magnanimous paternalism. Rather, we are left with the impression that women are not interesting enough for scientific endeavour but good enough for practice.

This is an edited extract from Pain and Prejudice by Gabrielle Jackson, published by Little, Brown (14.99 rrp). To order a copy for 11.24 with free UK p&p, go to guardianbookshop.com or call 020-3176 3837

The rest is here:
The female problem: how male bias in medical trials ruined women's health - The Guardian

Sodium Selenite Improves The Therapeutic Effect Of BMSCs Via Promoting | OTT – Dove Medical Press

Dongmei Yan,1,* Botao Tang,2,* Lixin Yan,3 Lei Zhang,1 Meijuan Miao,1 Xi Chen,4 Guangyi Sui,5 Qi Zhang,1 Daoyuan Liu,1 Hui Wang1

1Department of Blood Transfusion, The Second Affiliated Hospital of Harbin Medical University, Harbin, Peoples Republic of China; 2Department of Cardiology, Heilongjiang Red Cross Hospital, Harbin, Peoples Republic of China; 3Department of Laboratory Medicine, The Second Affiliated Hospital of Harbin Medical University, Harbin, Peoples Republic of China; 4Department of Hematology, The Second Affiliated Hospital of Harbin Medical University, Harbin, Peoples Republic of China; 5Ethics Committee, The Tumor Hospital Affiliated to Harbin Medical University, Harbin, Peoples Republic of China

*These authors contributed equally to this work

Correspondence: Hui WangDepartment of Blood Transfusion, The Second Affiliated Hospital, Harbin Medical University, Xuefu Road No. 246, Nangang District, Harbin, Heilongjiang Province, Peoples Republic of ChinaTel +86-451-86605134Email wanghui@hrbmu.edu.cn

Purpose: Sodium selenite (Na2SeO3) has been known to restore the antioxidant capacity of bone marrow mesenchymal stem cells (BMSCs), reduce the production of reactive oxygen species (ROS) in the cells, and promote cell proliferation and inhibit cell apoptosis. However, it is still not clear whether selenium can mediate the differentiation and inhibit the induced hemagglutination of BMSCs. In this study, we attempted to explore the effect of Na2SeO3 on these aspects of BMSCs.Methods: We evaluated the fate of the MSCs isolated from the bone marrow of mice by studying their differentiation and proliferation after treatment with Na2SeO3. We also simultaneously evaluated the coagulation reaction induced by Na2SeO3-treated BMSCs in vitro.Results: While the mice-derived BMSCs expressed CD44, CD73, CD90, and CD105, they did not express CD45. The morphology of the derived cells was homogeneously elongated. These results showed that the isolated cells are indeed BMSCs. We found that 0.1 M and 1 M of Na2SeO3 promoted the proliferation and apoptosis of BMSCs, respectively. This showed that Na2SeO3 can be toxic and exert certain side effects on the BMSCs. The results of the osteogenic and adipogenic assay showed that 0.1 M Na2SeO3 could significantly promote the osteogenic and adipogenic differentiation of BMSCs by upregulating the lipid factors (LPL and PPRAG) and osteogenic factors, RUNX2, COL1, and BGP, in a concentration-dependent manner. Coagulation experiments in animals (mice and rats) revealed that Na2SeO3 can reduce the coagulation time of BMSCs in a concentration-dependent manner, which is related to the high expression of hematopoietic factors (SDF-1, GM-CSF, IL-7, IL-8, IL-11, and SCF).Conclusion: Na2SeO3 promotes the proliferation and differentiation as well as reduces the coagulation time of BMSCs, and this effect might enhance the therapeutic effect of BMSCs.

Keywords: sodium selenite, BMSCs, proliferation, differentiation, coagulation factors, clotting time

This work is published and licensed by Dove Medical Press Limited. The full terms of this license are available at https://www.dovepress.com/terms.php and incorporate the Creative Commons Attribution - Non Commercial (unported, v3.0) License.By accessing the work you hereby accept the Terms. Non-commercial uses of the work are permitted without any further permission from Dove Medical Press Limited, provided the work is properly attributed. For permission for commercial use of this work, please see paragraphs 4.2 and 5 of our Terms.

More here:
Sodium Selenite Improves The Therapeutic Effect Of BMSCs Via Promoting | OTT - Dove Medical Press

US FDA Grants BeiGene’s BRUKINSA (zanubrutinib) Accelerated Approval to Treat Adult Patients with Mantle Cell Lymphoma Who Received at Least One Prior…

CAMBRIDGE, Mass. and BEIJING, China, Nov. 14, 2019 (GLOBE NEWSWIRE) -- BeiGene Co.,Ltd (NASDAQ: BGNE; HKEX: 06160), a commercial-stage biopharmaceutical company focused on developing and commercializing innovative molecularly-targeted and immuno-oncology drugs for the treatment of cancer, today announced that BRUKINSA (zanubrutinib) has received accelerated approval from the United States Food and Drug Administration (FDA) as a treatment for mantle cell lymphoma (MCL) in adult patients who have received at least one prior therapy.1 BRUKINSA is the first BeiGene-discovered product to be approved, an important milestone toward the companys goal of transforming treatment for cancer patients around the world.

This accelerated approval is based on overall response rate (ORR). Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

We are working to improve outcomes for people with cancer worldwide and this approval brings us closer to realizing our mission of bringing the highest quality therapies to patients globally, said John V. Oyler, Chairman, Co-Founder, and CEO of BeiGene. Todays FDA approval of BRUKINSA, following the previously granted Breakthrough Therapy designation in this indication, validates it as an important treatment option for people with relapsed or refractory MCL. We hope this is the first of many approvals for BRUKINSA as we continue to evaluate its potential in other hematologic cancers.

BRUKINSA is a BTK inhibitor that was designed to maximize target occupancy and minimize off-target binding. It entered the clinic in 2014 and since that time our broad development program has enrolled more than 1,600 patients globally, said Jane Huang, M.D., Chief Medical Officer, Hematology at BeiGene. Todays accelerated approval is the culmination of many years of effort by the BeiGene team, the dedicated investigators involved in these trials and, most importantly, the patients who participated by enrolling in the clinical trials. We are humbled by the opportunity to develop this therapy and launch it as our first internally discovered and approved cancer treatment.

BTK inhibition is an established mode of treatment for patients with MCL, but many patients treated with previously approved BTK inhibitors do not fully respond to BTK therapy or are forced to discontinue treatment early due to side effects. Today we have a new option for our adult patients who have received one prior systemic or targeted therapy and are living with MCL, an aggressive blood cancer thats often diagnosed at a more advanced stage, said Luhua (Michael) Wang, M.D., Professor, Department of Lymphoma and Myeloma, Division of Cancer Medicine at The University of Texas MD Anderson Cancer Center, and clinical trial investigator.

The approval of BRUKINSA as a second line therapy represents an important advancement for the treatment of mantle cell lymphoma, said Meghan Gutierrez, Chief Executive Officer for the Lymphoma Research Foundation. Expanded treatment options can transform the patient experience and provide hope to people living with a mantle cell diagnosis.

The FDAs approval of BRUKINSA is based on efficacy results from two single-arm clinical trials, with independent review committee (IRC)-assessed ORR per 2014 Lugano Classification as the primary endpoint. Across both trials, BRUKINSA achieved an ORR, which is the sum of complete responses and partial responses, of 84%.

In the multicenter Phase 2 trial of zanubrutinib in patients with relapsed or refractory (R/R) MCL BGB-3111-206 (NCT03206970), the ORR was 84% (95% CI: 74%, 91%), including 59% complete response (FDG-PET scan required) and 24% partial response. In this study, the median duration of response (DOR) was 19.5 months (95%CI: 16.6, NE) and median follow-up time on study was 18.4 months. In the global Phase 1/2 trial BGB-3111-AU-003 (NCT02343120), the ORR was 84% (95% CI: 67%, 95%), including 22% complete response (FDG-PET scan not required) and 62% partial response. In this study, the median DOR was 18.5 months1 (95% CI:12.6, NE) and median follow-up time on study was 18.8 months.

The most common adverse reactions (> 10%) with BRUKINSA were decreased neutrophil count, decreased platelet count, upper respiratory tract infection, decreased white blood cell count, decreased hemoglobin, rash, bruising, diarrhea, cough, musculoskeletal pain, pneumonia, urinary tract infection, blood in the urine (hematuria), fatigue, constipation, and hemorrhage. The most frequent serious adverse reactions were pneumonia (11%) and hemorrhage (5%).

Of the 118 patients with MCL treated with BRUKINSA, eight (7%) patients discontinued treatment due to adverse reactions in the trials. The most frequent adverse reaction leading to treatment discontinuation was pneumonia (3.4%). One (0.8%) patient experienced an adverse reaction leading to dose reduction (hepatitis B).

The recommended dose of BRUKINSA is 320 mg, taken orally 160 mg twice daily or 320 mg once daily with or without food. The dose may be adjusted for adverse reactions, and reduced for patients with severe hepatic impairment and certain drug interactions.1

BRUKINSA is expected to be available to people in the United States in the coming weeks.

myBeiGene Patient Support Program

BeiGene is committed to ensuring that people have access to the medicine and the support needed to have the best possible outcomes and experiences. Coinciding with todays approval, BeiGene is launching myBeiGene in the United States to support patients, caregivers, and healthcare providers with access to BRUKINSA. The myBeiGene program goes beyond financial assistance support to provide patients and caregivers with education about their disease and treatment with BRUKINSA, as well provide practical and emotional support by connecting them to third-party resources that can address their individual unique needs. For more information on myBeiGene, please call 1-833-234-4363 or visit BRUKINSA.com.

About Mantle Cell Lymphoma (MCL)

Lymphoma is a diverse group of cancers that originate from B-, T- or NK- cells. MCL is typically an aggressive form of non-Hodgkins lymphoma (NHL) that arises from B-cells originating in the mantle zone.2 Inthe United States, about 74,200 people will be diagnosed with NHLin 2019,3 with MCL representing about six percent (about 4,452 cases) of all new cases ofNHL.2 MCL usually has a poor prognosis, with a median survival of three to four years,4 and it often diagnosed at a later stage of disease.

About BRUKINSA (zanubrutinib)

BRUKINSA is a small molecule inhibitor of Brutons tyrosine kinase (BTK), discovered by BeiGene scientists, that is currently being evaluated globally in a broad pivotal clinical program as a monotherapy and in combination with other therapies to treat various B-cell malignancies. BRUKINSA was approved by the U.S. FDA to treat adult patients with MCL who have received at least one prior therapy on November 14, 2019.

New Drug Applications (NDAs) in China for relapsed refractory (R/R) MCL and R/R chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL) have been accepted by the China National Medical Products Administration (NMPA) and granted priority review and are pending approval.

BRUKINSA is not approved for use outside the United States.

IMPORTANT SAFETY INFORMATION FOR BRUKINSA (ZANUBRUTINIB)

Warnings and Precautions

Hemorrhage

Fatal and serious hemorrhagic events have occurred in patients with hematological malignancies treated with BRUKINSA monotherapy. Grade 3 or higher bleeding events including intracranial and gastrointestinal hemorrhage, hematuria and hemothorax have been reported in 2% of patients treated with BRUKINSA monotherapy. Bleeding events of any grade, including purpura and petechiae, occurred in 50% of patients treated with BRUKINSA monotherapy.

Bleeding events have occurred in patients with and without concomitant antiplatelet or anticoagulation therapy. Co-administration of BRUKINSA with antiplatelet or anticoagulant medications may further increase the risk of hemorrhage.

Monitor for signs and symptoms of bleeding. Discontinue BRUKINSA if intracranial hemorrhage of any grade occurs. Consider the benefit-risk of withholding BRUKINSA for 3-7 days pre- and post-surgery depending upon the type of surgery and the risk of bleeding.

Infections

Fatal and serious infections (including bacterial, viral, or fungal) and opportunistic infections have occurred in patients with hematological malignancies treated with BRUKINSA monotherapy. Grade 3 or higher infections occurred in 23% of patients treated with BRUKINSA monotherapy. The most common Grade 3 or higher infection was pneumonia. Infections due to hepatitis B virus (HBV) reactivation have occurred.

Consider prophylaxis for herpes simplex virus, pneumocystis jiroveci pneumonia and other infections according to standard of care in patients who are at increased risk for infections. Monitor and evaluate patients for fever or other signs and symptoms of infection and treat appropriately.

Cytopenias

Grade 3 or 4 cytopenias, including neutropenia (27%), thrombocytopenia (10%) and anemia (8%) based on laboratory measurements, were reported in patients treated with BRUKINSA monotherapy.

Monitor complete blood counts during treatment and treat using growth factor or transfusions, as needed.

Second Primary Malignancies

Second primary malignancies, including non-skin carcinoma, have occurred in 9% of patients treated with BRUKINSA monotherapy. The most frequent second primary malignancy was skin cancer (basal cell carcinoma and squamous cell carcinoma of skin), reported in 6% of patients. Advise patients to use sun protection.

Cardiac Arrhythmias

Atrial fibrillation and atrial flutter have occurred in 2% of patients treated with BRUKINSA monotherapy. Patients with cardiac risk factors, hypertension, and acute infections may be at increased risk. Grade 3 or higher events were reported in 0.6% of patients treated with BRUKINSA monotherapy. Monitor signs and symptoms for atrial fibrillation and atrial flutter and manage as appropriate.

Embryo-Fetal Toxicity

Based on findings in animals, BRUKINSA can cause fetal harm when administered to a pregnant woman. Administration of zanubrutinib to pregnant rats during the period of organogenesis caused embryo-fetal toxicity, including malformations at exposures that were 5 times higher than those reported in patients at the recommended dose of 160 mg twice daily. Advise women to avoid becoming pregnant while taking BRUKINSA and for at least 1 week after the last dose. Advise men to avoid fathering a child during treatment and for at least 1 week after the last dose. If this drug is used during pregnancy, or if the patient becomes pregnant while taking this drug, the patient should be apprised of the potential hazard to a fetus.

Adverse Reactions

The most common adverse reactions in > 10% of patients who received BRUKINSA were neutrophil count decreased (53%), platelet count decreased (39%), upper respiratory tract infection (38%), white blood cell count decreased (30%), hemoglobin decreased (29%), rash (25%), bruising (23%), diarrhea (20%), cough (20%), musculoskeletal pain (19%), pneumonia (18%), urinary tract infection (13%), hematuria (12%), fatigue (11%), constipation (11%), and hemorrhage (10%). The most frequent serious adverse reactions were pneumonia (11%) and hemorrhage (5%).

Drug Interactions

CYP3A Inhibitors:When BRUKINSA is co-administered with a strong CYP3A inhibitor, reduce BRUKINSA dose to 80 mg once daily. For coadministration with a moderate CYP3A inhibitor, reduce BRUKINSA dose to 80 mg twice daily.

CYP3A Inducers:Avoid coadministration with moderate or strong CYP3A inducers.

Specific Populations

Hepatic Impairment: The recommended dose of BRUKINSA for patients with severe hepatic impairment is 80 mg orally twice daily.

INDICATION

BRUKINSA is a kinase inhibitor indicated for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy.

This indication is approved under accelerated approval based on overall response rate. Continued approval for this indication may be contingent upon verification and description of clinical benefit in a confirmatory trial.

Please see fullPrescribing Information at beigene.com/PDF/BRUKINSAUSPI.pdf and Patient Information at beigene.com/PDF/BRUKINSAUSPPI.pdf

About the Zanubrutinib Clinical Trial Program

Clinical trials of zanubrutinib include:

About BeiGene

BeiGene is a global, commercial-stage, research-based biotechnology company focused on molecularly-targeted and immuno-oncology cancer therapeutics. With a team of over 3,000 employees in the United States, China, Australia, and Europe; BeiGene is advancing a pipeline consisting of novel oral small molecules and monoclonal antibodies for cancer. BeiGene is also working to create combination solutions aimed to have both a meaningful and lasting impact on cancer patients. In the United States, BeiGene markets and distributes BRUKINSA (zanubrutinib) and in China, the Company markets ABRAXANE (nanoparticle albuminbound paclitaxel), REVLIMID (lenalidomide), and VIDAZA (azacitidine) under a license from Celgene Corporation.5

Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding BeiGenes plans and expectations for the commercialization of BRUKINSA, the potential implications of clinical data for patients, BeiGenes further advancement of, and anticipated clinical development, regulatory milestones and commercialization of BRUKINSA. Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including BeiGene's ability to demonstrate the efficacy and safety of its drug candidates; the clinical results for its drug candidates, which may not support further development or marketing approval; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; BeiGene's ability to achieve commercial success for its marketed products and drug candidates, if approved; BeiGene's ability to obtain and maintain protection of intellectual property for its technology and drugs; BeiGene's reliance on third parties to conduct drug development, manufacturing and other services; BeiGenes limited operating history and BeiGene's ability to obtain additional funding for operations and to complete the development and commercialization of its drug candidates, as well as those risks more fully discussed in the section entitled Risk Factors in BeiGenes most recent quarterly report on Form 10-Q, as well as discussions of potential risks, uncertainties, and other important factors in BeiGene's subsequent filings with the U.S. Securities and Exchange Commission. All information in this press release is as of the date of this press release, and BeiGene undertakes no duty to update such information unless required by law.

Investor Contact

Craig West

+1 857-302-5189

ir@beigene.com

Media Contact

Liza Heapes or Vivian Ni

+1 857-302-5663 or +1 857-302-7596

media@beigene.com

_________________________________________

1 BRUKINSA (zanubrutinib) Prescribing Information. beigene.com/PDF/BRUKINSAUSPI.pdf. BeiGene, Ltd; November 14, 2019.

2 https://www.lls.org/sites/default/files/file_assets/FS4_MCL_Facts_2018-final.pdf

3 https://www.cancer.org/cancer/non-hodgkin-lymphoma/about/key-statistics.html

4 Philip J. Bierman,James O. Armitage, in Goldman'sCecil Medicine(Twenty Fourth Edition), 2012.

5 ABRAXANE, REVLIMID and VIDAZA are registered trademarks of Celgene Corporation.

Photos accompanying this announcement are available at:

https://www.globenewswire.com/NewsRoom/AttachmentNg/f728ceea-0c2b-4042-a937-e6b1638b4323

https://www.globenewswire.com/NewsRoom/AttachmentNg/5e5fe145-2754-4389-b9b9-b23a3731d03e

PDFsaccompanying this announcement are available at:

http://ml.globenewswire.com/Resource/Download/aca04555-626d-4c35-821b-957abaab640c

http://ml.globenewswire.com/Resource/Download/c47ea153-ba25-4dd1-8155-937b64fdf4b7

BRUKINSA Logo

BRUKINSA product logo

BRUKINSA Product Image

BRUKINSA packaging and tablets

View post:
US FDA Grants BeiGene's BRUKINSA (zanubrutinib) Accelerated Approval to Treat Adult Patients with Mantle Cell Lymphoma Who Received at Least One Prior...