Category Archives: Gene Medicine

A study has found that disabling a gene in the myeloid cells of mice prevents them from developing obesity.

New research has found that inhibiting an immune cell gene in mice prevented them from developing obesity, even when they consumed a diet high in fat.

The studys findings, published in The Journal of Clinical Investigation, may one day help scientists develop therapies that can help people with obesity burn calories more easily.

Obesity is a major health issue, and in the United States, rates of the condition have risen over the past 40 years.

The Centers for Disease Control and Prevention (CDC) report that between 2017 and 2018, 42.4% of people in the country had obesity. Between 1999 and 2000, that figure was 30.5%.

Obesity increases the risk of heart disease, strokes, diabetes, and some types of cancer.

The CDC say that lifestyle changes, including eating a more healthful diet and getting more regular exercise, are key to reducing obesity.

One issue, however, involves obesitys effects on metabolism previous research in mice lead to the suggestion that a person with obesity burns fewer calories than a person who does not have obesity.

Better understanding how and why this might happen, and what scientists and clinicians can do about it, may help with reducing obesity.

In the present study, the researchers inhibited a gene in immune cells in mice. They did this because of an association between obesity and increased inflammation, and immune cells play a key role in controlling inflammation.

The researchers had wanted to find out what part the immune cells play in the metabolic complications of obesity. To their surprise, they found that the cells have a central role in regulating obesity and weight gain.

To study the effects of inhibiting the immune cell gene, the researchers conducted two experiments. In the first, they deleted the gene Asxl2, and in the second, they injected regular mice with nanoparticles that interfered with the function of the gene.

Once the researchers had inhibited this gene in the immune cells, they found that the mice did not develop obesity when fed a high fat diet, and that this was likely due to increased energy expenditure.

Compared with a control group of mice who had obesity but none of the gene inhibition, the mice with the inhibition burned 45% more calories, despite eating high fat diets.

For the studys principal investigator, Prof. Steven L. Teitelbaum, of the Washington University School of Medicine, in St. Louis, MO, Weve developed a proof of concept, here, that you can regulate weight gain by modulating the activity of these inflammatory cells.

It might work in a number of ways, but we believe it may be possible to control obesity and the complications of obesity by better regulating inflammation.

The team is not yet sure why inhibiting the gene in the mices immune cells resulted in them not gaining weight while on a high fat diet. The researchers suspect that the answer may involve encouraging white fat cells to burn fat rather than store it, as brown fat cells do.

While this is only preliminary research, the findings may eventually help people with obesity burn calories at a higher rate, supporting them as they make broader lifestyle changes that involve the diet and exercise.

According to Prof. Teitelbaum, A large percentage of Americans now have fatty livers, and one reason is that their fat depots cannot take up the fat they eat, so it has to go someplace else.

These mice consumed high fat diets, but they didnt get fatty livers. They dont get type 2 diabetes. It seems that limiting the inflammatory effects of their macrophages allows them to burn more fat, which keeps them leaner and healthier.

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Obesity in mice prevented by disabling gene - Medical News Today

ROCKLAND, Mass., May 29, 2020 /PRNewswire/ -- EMD Serono, the biopharmaceutical business of Merck KGaA, Darmstadt, Germany in the US and Canada, today announced that updated data from the ongoing, single-arm Phase II VISION studyevaluating tepotinib* as a single agent in patients with advanced non-small cell lung cancer (NSCLC) with MET exon 14(METex14) skipping alterations were published in The New England Journal of Medicine(NEJM). Results from the primary analysis of data from 99 patients with at least 9 months of follow-up demonstrate consistent response rate and durable anti-tumor activity across lines of treatment in patients assessed by both liquid biopsy (LBx) and tissue biopsy (TBx).Results from the VISION study were also presented at the American Society of Clinical Oncology (ASCO) ASCO20 Virtual Scientific Program on May 29, including data from the primary analysis (Abstract #9556) and including patient-reported outcomes (PROs) of health-related quality of life (HRQoL) (Abstract #9575). Tepotinib is designed to be a highly selective1 oral MET inhibitor that is administered once daily and is designed to inhibit the oncogenic MET receptor signaling caused by MET (gene) alterations.

"METexon 14 skipping is a primary oncogenic driver, but until recently there have been no approved treatment options targeting this genetic alteration in NSCLC," said Paul K. Paik, M.D., primary study investigator, lead author and Clinical Director, Thoracic Oncology Service, Memorial Sloan Kettering Cancer Center. "These new findings highlight the importance of routine next-generation sequencing to identify METex14 skipping alterations and demonstrate tepotinib's durable anti-tumor activity in patients who are typically elderly, and whose cancers are often harder to treat."

This new analysis of data from 99 patients in the fully enrolled Cohort A with at least 9 months of follow-up was published by The New England Journal of Medicine on May 29. Results demonstrate objective response rate (ORR) of 46% (95% CI, 3657) among patients with METex14 skipping alterations identified by either LBx or TBx as assessed by Independent Review Committee (IRC), and 56% (95% CI, 4566) as assessed by investigators. The median duration of response (DOR) was 11.1 months (95% CI, 7.2could not be estimated (NE)) among patients with METex14 skipping alterations identified by either LBx or TBx as assessed by IRC, and 14.0 months (95% CI, 9.718.3) as assessed by investigators.Results were consistent across different lines of treatment and in patients assessed byLBx orTBx. Additional endpoints were progression-free survival (PFS) and overall survival (OS).

Patients with brain metastases at baseline (n=11) benefitted similarly from treatment. In these patients, systemic ORR as assessed by independent review was 55% (95% CI, 2383), with a median DOR of 9.5 months (95% CI, 6.6NE) and a median PFS of 10.9 months (95% CI, 8.0NE).

Results also include the first patient-reported quality-of-life outcomes in patients with NSCLC with METex14 skipping alterations. Quality of life was maintained over time of treatment with tepotinib, with symptoms of dyspnea remaining stable and cough symptoms improving. The first longitudinal on-treatment biomarker data fromLBx samples were also reported, showing high concordance between molecular circulating free DNA response (defined as METex14 depletion) and clinical response based on measurable disease per Response Evaluation Criteria in Solid Tumors (RECIST).

Out of 152 patients evaluable for safety, treatment-related adverse events (TRAEs) of all grades were reported in 135 patients (89%). Grade 3 TRAEs were reported in 38 patients (25%), and 3 patients (2%) experienced Grade 4 TRAEs. One death was considered by the investigator to be treatment-related and occurred in a 79-year-old patient with respiratory failure and dyspnea, secondary to interstitial lung disease. The most common Grade 3 TRAE was peripheral edema, which occurred in 11 patients (7%). Serious TRAEs were reported in 23 patients (15%). Permanent tepotinib discontinuations due to TRAEs were reported in 17 patients (11%), and 50 patients (33%) required a dose reduction due to TRAEs. Peripheral edema was the most common TRAE leading to a dose reduction (25 patients, 16%) or dose interruption (28 patients, 18%); permanent discontinuation was uncommon (7 patients, 5%).

"Designed to have a highly selective mechanism of action, tepotinib has the potential to make a difference in the treatment and lives of people living with non-small cell lung cancer harboringMETex14 skipping alterations," said Luciano Rossetti,Global Head of Research & Development for EMD Serono. "Following on the recent approval of tepotinib in Japan as the first therapy for the treatment of advanced NSCLC harboring MET gene alterations, the publication of these data underscores our commitment to advancing scientific understanding and potential therapeutic options for this challenging cancer."

The ongoing Phase II VISION (NCT02864992) clinical trial is a single-arm, open-label, multi-cohort study investigating the safety and efficacy of tepotinib as a single agent in patients with advanced or metastatic NSCLC with METex14 skipping alterations identified by LBx and/or TBx. The use of both LBx and TBx to identify patients for the VISION study is intended to support improved patient selection and is consistent with the company's focus on patient-centric drug development.

Lung cancer is the most common type of cancer worldwide, with2 million cases diagnosed annually.2Alterations of the MET signaling pathway are found in various cancer types, including 3% to 5% of NSCLC cases, and correlate with aggressive tumor behavior and poor clinical prognosis.3-5 Patients with NSCLC harboring METex14 skipping tend to be older than those with NSCLC harboring other alterations.6In the Phase II VISION study, the patient population is generally characterized as elderly, with a median age of 74.0 years, and as having poor clinical prognosis typical of NSCLC with METex14 skipping alterations.

In March 2020, the Japanese Ministry of Health, Labour and Welfare (MHLW) approved tepotinib for the treatment of patients with unresectable, advanced or recurrent NSCLC with METex14skipping alterations.In September 2019, the US Food and Drug Administration (FDA) granted Breakthrough Therapy Designation for tepotinib in patients with metastatic NSCLC harboring METex14 skipping alterations who progressed following platinum-based cancer therapy.EMD Serono plans to file tepotinib for regulatory review with the FDA in 2020. Tepotinib is also being investigated in the INSIGHT 2 study (NCT03940703) in combination with the tyrosine kinase inhibitor (TKI) osimertinib in epidermal growth factor receptor (EGFR)-mutated, MET amplified, locally advanced or metastatic NSCLC that has acquired resistance to prior EGFR TKI.

Discovered in-house at Merck KGaA, Darmstadt, Germany, tepotinib is an oral MET inhibitor that is designed to inhibit the oncogenic MET receptor signaling caused by MET (gene) alterations.

*Tepotinib is currently under clinical investigation and not yet approved in any markets outside of Japan.

Dr. Paik has provided compensated advisory services to EMD Serono.

About Non-Small Cell Lung Cancer With 2 million cases diagnosed annually, lung cancer (including trachea, bronchus and lung) is the most common type of cancer worldwide and the leading cause of cancer-related death, with 1.7 million mortality cases worldwide.2 Alterations of the MET signaling pathway, including MET exon 14 (METex14) skipping alterations and MET amplifications, occur in 3% to 5% of NSCLC cases.3-5

About TepotinibTepotinib is an oral MET inhibitor that is designed to inhibit the oncogenic MET receptor signaling caused by MET (gene) alterations. Discovered in-house at Merck KGaA, Darmstadt, Germany, it has been designed to have a highly selective mechanism of action,with the potential to improve outcomes in aggressive tumors that have a poor prognosis and harbor these specific alterations.Tepotinibis currently under clinical investigation in NSCLC and not yet approved in any markets outside of Japan.Merck KGaA, Darmstadt, Germany, is actively assessing the potential of investigating tepotinib in combination with novel therapies and in other tumor indications.

References

All Merck KGaA, Darmstadt, Germany press releases are distributed by e-mail at the same time they become available on the EMD Group Website. In case you are a resident of the USA or Canada please go to http://www.emdgroup.com/subscribe to register for your online subscription of this service as our geo-targeting requires new links in the email. You may later change your selection or discontinue this service.

About EMD Serono, Inc.EMD Serono - the biopharmaceutical business of Merck KGaA, Darmstadt,Germany in the U.S. andCanada- is engaged in the discovery, research and development of medicines for patients with difficult to treat diseases. The business is committed to transforming lives by developing and delivering meaningful solutions that help address the therapeutic and support needs of individual patients. Building on a proven legacy and deep expertise in neurology, fertility and endocrinology, EMD Serono is developing potential new oncology and immuno-oncology medicines while continuing to explore potential therapeutic options for diseases such as psoriasis, lupus and MS. Today, the business has approximately 1,500 employees around the country with commercial, clinical and research operations based in the company's home state ofMassachusetts.www.emdserono.com.

About Merck KGaA, Darmstadt, GermanyMerck KGaA, Darmstadt, Germany, a leading science and technology company, operates across healthcare, life science and performance materials. Around 57,000 employees work to make a positive difference to millions of people's lives every day by creating more joyful and sustainable ways to live. From advancing gene editing technologies and discovering unique ways to treat the most challenging diseases to enabling the intelligence of devices the company is everywhere. In 2019, Merck KGaA, Darmstadt, Germany generated sales of 16.2 billion in 66 countries.

The company holds the global rights to the name and trademark "Merck" internationally. The only exceptions are the United States and Canada, where the business sectors of Merck KGaA, Darmstadt, Germany operate as EMD Serono in healthcare, MilliporeSigma in life science, and EMD Performance Materials. Since its founding in 1668, scientific exploration and responsible entrepreneurship have been key to the company's technological and scientific advances. To this day, the founding family remains the majority owner of the publicly listed company.

SOURCE EMD Serono

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New England Journal of Medicine Publishes Primary Analysis of VISION Data for Tepotinib in Advanced NSCLC with METex14 Skipping Alterations -...

When someone has a heart attack, it causes muscle cells in their heart to die, and the heart cannot regenerate these cells. Researchers at the University of Louisville have begun preclinical testing of a new gene therapy that stimulates regeneration of heart muscle cells.

The project, led by Tamer M.A. Mohamed, assistant professor of medicine in the UofL Division of Cardiovascular Medicine and the UofL Institute of Molecular Cardiology, has received a five-year, $3.8 million grant from the National Heart, Lung and Blood Institute.

After a patient suffers a heart attack, the heart loses muscle cells, reducing the hearts ability to pump blood to the rest of the body. Muscle cells in the heart do not regenerate on their own, leaving the heart permanently impaired, Mohamed said. We are developing a transient gene therapy approach to regenerate these muscle cells to heal the heart.

The therapy involves transient overexpression of a combination of four cell-cycle regulating proteins to induce cell division in the heart muscle. The four cell-cycle regulators, cyclin-dependent kinase 1 (CDK1), CDK4, cyclin B1, and cyclin D1, are known collectively as 4F, or four factors. In previous research, the process stimulated cell division in cardiomyocytes, or heart cells, leading to improved heart function.

The new study will determine further the effectiveness and safety of the therapy in animal models as well as in human heart segments using of a biomimetic culture system developed at UofL by Mohamed that keeps slices of human hearts alive for a longer period of time. The system mimics the environment of a living organ through continuous electrical stimulation and oxygenation, maintaining viability and functionality of the heart segments for six days, allowing more extensive testing. The heart culture system is available for use by researchers outside UofL.

In addition to further testing the therapys effectiveness, Mohamed and other investigators will focus on approaches for the process that do not lead to tumor development in other cells.

The challenge comes in avoiding development of cancer in other areas of the body, which appears to be a side effect of the process as seen in mice, Mohamed said.

If it is successful, the work will lead to the start of in-human clinical trials.

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UofL receives $3.8M to test new gene therapy for heart attacks - uoflnews.com

IRVINE, Calif. and AMSTERDAM, May 29, 2020 /PRNewswire/ -- Agendia, Inc., a world leader in precision oncology for breast cancer, announced that new data from ongoing clinical research on MammaPrint® and BluePrint® was debuted at the American Society of Clinical Oncology 2020 Virtual Scientific Program (ASCO) today. A total of five posters were presented on Agendia's genomic profiling assays.

The highlighted data below further illustrate the efficacy of Agendia's MammaPrint and BluePrint genomic testing to consistently stratify breast cancers, allowing for a highly personalized regimen throughout a patient's treatment journey. The latest findings from Agendia deliver immediate, actionable information for doctors and patients early in the diagnosis and treatment planning process and build on research that will impact breast cancer treatment and outcomes in the future.

One scientific presentation, entitled "Adding precision to 2018 ASCO/CAP HER2 testing guidelines in breast cancer with genomic profiling," evaluated the concordance between human epidermal growth factor receptor 2 (HER2) status as put forth by the 2018 ASCO/CAP guidelines and Agendia's BluePrint genomic testing. In this real-world diagnostic data set, the 2018 guideline recommendations led to fewer HER2 equivocal tumors overall, confirming the positive impact of the revisions. Of note, BluePrint reclassified 69 percent of HER2-positive tumors and all HER2 equivocal tumors to non-HER2 molecular subtypes, indicating that these tumors may have suboptimal responses to HER2-directed therapy. This study found that molecular classification by BluePrint adds further precision in stratifying HER2-positive patients, offering the potential to predict responsiveness to HER2-targeted therapies.

"In this study, nearly 70 percent of HER2-positive diagnoses were reclassified to non-HER2, based on molecular subtyping. This is interesting and may have the potential at some point to affect treatment decisions and patient outcomes," said Adam Brufsky, MD, PhD, and Professor of Medicine at the University of Pittsburgh School of Medicine. "Data continue to show the value of MammaPrint and BluePrint as diagnostic tools that allow physicians to make more informed decisions to address their patients' disease."

Also at ASCO, Agendia shared updates on the ongoing FLEX trial, the massive real-world clinical data set designed to drive the medical community forward in its approach to precision medicine. In addition to a designated FLEX study poster "The FLEX Real World Data Platform Explores New Gene Expression Profiles and Investigator-Initiated Protocols in Early Stage Breast Cancer" that gave general updates on the registry, Agendia also highlighted FLEX and forward-looking studies, one of which has immediate implications for how a patient's treatment may change based on comprehensive information uncovered by BluePrint.

The FLEX scientific presentation, entitled, "TNBC subtype and clinical estrogen receptor status of genomically basal breast tumors in Caucasian, African American, and Latin American patients," evaluated the distribution of triple-negative breast cancer (TNBC) subtypes in genomically Basal-Type cancers from self-reported patient ethnicities (Caucasian, African American, and Latin American). The data show that Basal-Type tumors are heterogeneous and include all defined TNBC subtypes, independent of ethnicity.

In addition, the study evaluated the association of IHC-determined estrogen receptor status and Basal-Type tumors of each ethnicity. Analyses demonstrated that BluePrint reclassified a subset of estrogen receptor positive (ER+) tumors to molecular Basal-Type and that ER status was not significantly associated with a specific TNBC subtype or ethnicity. This highlights the clinical need to trace basal biology in ER+ patients to refine treatment for basal-like tumors.

"The reclassification of a subset of ER+ tumors identifies an urgent and actionable situation," said Cathy Graham, MD, Assistant Professor of Surgery in the Division of Surgical Oncology at Emory University School of Medicine and Director, Breast Surgery at Emory St. Joseph's Hospital. "From a clinical perspective, when these patients are first diagnosed, they appear to have luminal breast cancer. But, when you are able to look at the underlying mechanism with comprehensive genomic testing, a large subset of these breast cancers is reclassified to Basal-Type, which is high risk. This knowledge allows us to execute a more personalized and precise treatment approach immediately."

Two other forward-looking studies reinforce the future utility of better stratifying patients, and Agendia's ability to provide a more sophisticated platform for discovery in gene signature research.

"The research we are showcasing at this year's ASCO Annual Meeting underscores our commitment to patients and physicians," said William Audeh, MD, Chief Medical Officer at Agendia. "With our growing arsenal of data collected through prospective clinical trials such as FLEX, I-SPY2 and MINDACT, we are able to help patients now while information-gathering for future breast cancer treatment strategy."

Agendia is proud to present these findings at the ASCO 2020 Annual Meeting, which underscore the company's commitment to innovation and discovery through extraordinary, patient-focused research.

About Agendia

Agendia is a precision oncology company committed to improving clinical outcomes and informing the journey for patients with early-stage breast cancer. The company currently offers two commercially available genomic profiling tests, processed through its state-of-the-art facility in Irvine, California. Agendia also provides a next-generation sequencing kit for use by local laboratories outside of the United States.

MammaPrint®, the 70-gene breast cancer recurrence assay, is the first FDA-cleared risk-of-recurrence test backed by peer-reviewed, prospective outcome data and included in both national and international treatment guidelines. BluePrint®, the 80-gene molecular subtyping assay, is a commercially available test that evaluates the underlying biology of a tumor to determine what is driving its growth. Together, MammaPrint and BluePrint provide a comprehensive genomic profile to help physicians make more informed decisions in the pre- and post-operative treatment settings. By developing evidence-based, novel genomic tests, Agendia aims to support the evolving clinical needs of breast cancer patients and their physicians.

Agendia's assays can be ordered on core biopsies or surgical specimens with results provided in as little as 5-7 days to inform pre- and post-operative treatment decisions. For more information on Agendia's assays and ongoing trials, please visit http://www.agendia.com.

About the FLEX Registry

Three years ago, Agendia launched a clinical trial for patients in the US, known as the FLEX Registry. It is a large-scale, prospective, observational breast cancer study that links whole transcriptome profiling, including MammaPrint and BluePrint, with complete clinical data. FLEX generates a comprehensive patient database with the potential to identify new gene associations with prognostic and/or predictive value in breast cancer.

View FLEX Registry details here.

About the I-SPY2 Study

The I-SPY2 trial looks at whether adding experimental agents to standard neoadjuvant medications increases the probability of pathologic complete response (pCR) beyond standard neoadjuvant chemotherapy for each biomarker signature established at trial entry. So far, findings support the use of residual cancer burden (RCB) as a prognostic indicator for three-year outcomes in patients pre-selected as high risk for recurrence, and the importance of MammaPrint in identifying these patients.

I-SPY2 breaks from the traditional randomized trial design, employing an 'adaptive' model that allows multiple treatments (up to six different agents) to be studied in parallel. This master framework also allows new agents to enter and leave the study without having to halt enrollment or resubmit the entire clinical trial protocol for regulatory review.

View the I-SPY2 trial site here.

About the MINDACT Trial

MammaPrint is supported by the highest level of clinical evidence (level 1A) from MINDACT, a landmark independent trial published in the New England Journal of Medicine in 2016.

MINDACT stands for Microarray In Node-Negative and 1-3 node-positive Disease may Avoid ChemoTherapy. It was a phase III, prospective, randomized, clinical study for a breast cancer recurrence test sponsored by the European Organization for Research and Treatment of Cancer (EORTC-10041/BIG3-04).

View the whole trial here.

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Agendia Announces Data Presented at ASCO 2020 Demonstrating Importance of Further Classification of Breast Cancers to Enable Precise Prognosis and...

By TONNY ABET

Dr Misaki Wayengera Dr Misaki Wayengera heads the Ministerial Scientific Advisory Committee on Covid-19 at Ministry of Health that is advising government on safeguarding the country from worst case pandemic scenario. He is in charge of coordinating the work of different experts on the committee.

Dr Misaki is also developing rapid diagnostic test kits for coronavirus, which are expected to be ready in a months time.

He has graduate training across Immunology, filoviruses, vaccinology, clinical microbiology and genetics. Dr Wayengera completed his PhD in 2018. He is member of the African Society for Human Genetics and inaugural chair of the H3Africa Consortiums Education and Coordinated Working Group (ECTWG, 2013-2016).

Dr Misakis research interests focus on Pathogen OMICS aimed at identifying new molecular targets for developing new diagnostics, therapeutics and vaccines.

Dr Misaki also developed testing kits for Ebola RDT, HIV-1 Gene Therapeutic, and next Gen TB Diagnostics. He is a bio-entrepreneur, and founder of Restrizymes Biotherapeutics (U) Ltd. In 2019, he won 1st Prize for the World Health Organisation (WHO) innovation Challenge (Product Development).

Prof Rhoda Wanyenze Prof Rhoda Wanyenze is an epidemiologist who is in charge of Covid-19 case modeling on the National Covid-19 Taskforce. She is a physician, public health consultant, academic and medical administrator, who serves as the Dean of Makerere University School of Public Health.

Born in 1968, Prof Wanyenze attended Nabisunsa Girls S.S. and earned her medical degree from Makerere University in 1993.

She studied Master in Public Health, also at Makerere in 2002, and a PhD from the University of Antwerp, Belgium, in 2010. She received additional training in HIV/Aids programme leadership and management, gender and health, project planning and management, and designing clinical research.

Dr Wanyenze has 25 years experience in health service delivery, including clinical, research, teaching, programme management, and policy development. Over the past seven years, she has served on boards of six organisations in Uganda.Prof Wanyenze sits on various boards, including that of Baylor-Uganda, and Uganda Aids Commission.

She is also a member of the Ministry of Health HIV Counselling and Testing Committee.

Prof Wilson Muhwezi Prof Wilson Muhwezi is an associate professor of Behavioural Sciences and Mental Health at Makerere University. He is heading the ad hoc committee on psychological interventions.

Prof Muhwezi is in charge of ensuring that putting people in quarantine, separation from family and re-integrating recovered patients is done with minimal effect on their psychological wellbeing.He also advises on how to fight the Covid-19 related anxiety and fears.

Born in 1967, he went to Bugamba Integrated Primary School in Mbarara District. He pursued his O-Level studies at Bugamba and A-Level at Nganwa High School.

In 1989, Muhwezi joined Makerere University to pursue a Bachelors Degree in Social Works and Social Administration. Prof Muhwezi did his Masters in Health Promotion from Bergen University in Norway between 1989 and 1998.He did his PhD in Medical Sciences/Social Medicine in both Karolinska Institute, Sweden, and Makerere University. He is a social scientist.

Prof Denis ByarugabaProf Denis Byarugaba is a microbiologist who heads the National Flu Surveillance Group. He is carrying out more research to understand the type of Covid-19 in the country. Dr Byarugaba has been studying different forms of coronavirus for more than 20 years. He unravels the new findings about the virus and localises Covid-19-related knowledge.

Dr Henry Kajumbula Dr Henry Kajumbura chairs the Infection and Biology committee. He is a microbiologist at Makerere University. He advises on safety measures such as wearing facemasks, hand-sanitising and social distancing.

He has been a clinical pathologist specialising in Medical Microbiology since 1998.

In recognition of his leadership role in the control of antimicrobial resistance in Uganda, Dr Kajumbula was nominated to chair the countrys Antimicrobial Resistance Surveillance Committee, a position he still holds.

As a result, he has coordinated development of the Countrys Nacrobial resistance as well as a national surveillance plan for antimicrobial resistance.

Prof Pontiano Kaleebu Prof Pontiano Kaleebu is a physician, clinical immunologist, HIV/Aids researcher, academic and medical administrator. He is the director of the Uganda Virus Research Institute (UVRI), which is in charge of testing for coronavirus. He advises government on testing for coronavirus and other disease-specific information.

Appointed in July 2010 as the new head of the UVRI, Prof Kaleebu holds a medical degree from Makerere University and a PhD from Imperial College, London.

Born in 1960, Kaleebu attended Jinja Kaloli Primary School in Wakiso District from where he completed and was admitted to St Marys College Kisubi for O-Level. He completed his A-Level from Kampala High School and joined Makerere University to pursue Bachelor of Medicine and Bachelor of Surgery in the mid-1980s.

In 1988, Kaleebu was awarded a scholarship by Royal Postgraduate Medical School, UK, to study immunology. Upon graduation, he pursued post-graduate studies at Imperial College -London where he completed his PhD programme in mid 1990s.

In 1987, Kaleebu was recruited as a medical research officer at UVRI while still pursuing his PhD studies. His service through the years saw him appointed as head of the Immunology Department at UVRI in 1995.While at UVRI, Prof Kaleebu joined the joint Medical Research Council, MRC/UVRI research programme in 1996. In July 2010, he was appointed as director of the UVRI.

As UVRI director, he sits on the senior management of the Ministry of Health. He leads a number of other national and regional networks of scientists and researchers.

Dr Jane Ruth AcengBorn in 1968, Dr Aceng is the Minister of Health, a position she has held since 2016. Her ministry has been at the centre of containing the coronavirus pandemic.

Dr Aceng studied from Shimoni Demonstration School in Kampala for primary and proceeded to Nabisunsa Girls School for both O- and A-Level education.

In 1993, she pursued a Bachelors Degree of Medicine and Bachelor of Surgery at Makerere University. Her career started off at Lira hospital in 1994 as a health officer. She grew through the ranks until she became a consultant paediatrician/ acting director in 2010. She later served as Director General of Health Services for five years until she was appointed Minister of Health. She has served through Ebola outbreaks and nodding disease syndrome.

Dr Joyce Moriku KaducuBorn in 1969 in Moyo District, Dr Joyce Moriku Kaducu, is a paediatrician, academic and politician. She was appointed Minister of State for Primary Healthcare in 2016, replacing Sarah Opendi. In 1984, she joined Laropi Primary School in Moyo District and proceeded to Metu S.S. in Moyo, for O- Level and Sacred Heart S.S. in Gulu District for A-Level education.

Dr Moriku joined Mbarara University Medical School in 2002, pursuing Bachelor of Medicine and Bachelor of Surgery. Dr Moriku has a PhD in Neuroscience from Gulu University in 2015.

She started working at Lacor Hospital in Gulu. She then worked as medical coordinator for The AIDS Support Organisation (Taso) in Gulu between 2003 and 2005. Dr Moriku later served as a medical director for Mildmay Clinic on Entebbe Road from 2008 to 2009.

Between 2010 and 2015, she was a lecturer in paediatrics at Gulu University and concurrently serves as a consultant pediatrician at Gulu Regional Referral Hospital.

Dr Diana Atwine Born in 1973, Dr Diana Atwine is the permanent secretary at the Ministry of Health, a position she has held since November 2016. She attended Bweranyangi Girls S.S. for O-Level and Mt St Marys Namagunga Girls School for her A-Level.

Dr Atwine pursued her Bachelor of Medicine and Bachelor of Surgery at Mbarara University School of Medicine, specialising in internal medicine. She started working at St Francis Hospital Nsambya, then joined the Uganda Joint Clinical Research Centre.

Dr Atwine moved to State House where she served as the Presidents Private Secretary in charge of medical affairs. In 2009, she was tasked to head the State Houses Medicine and Health Services Delivery Monitoring Unit.

Dr Henry Mwebesa is the Director General of Health Services at the Ministry of Health. He was appointed in February after serving as the Commissioner Health Services and Planning.

Col Dr Henry Kyobe Col Kyobe is the Covid-19 incident commander. He is an army doctor and senior consultant epidemiologist. He is deputised by Dr Atek Kagirita.

Dr Kyobe is responsible for emergency response, including quickly developing incident objectives, managing all incident operations, application of resources as well as responsibility for all persons involved.

Dr Jane NakibukaDr Jane Nakibuka is an intensivist and internal medicine expert at Mulago National Referral Hospital. She is heading the ad hoc committee on Covid-19 case management.

Dr Nakibuuka is behind the type of treatment being given to Covid-19 patients like the hydroxychloroquine, Vitamin C and antibiotics.

Other scientists Prof Noah Kiwanuka, is an Infectious Disease Epidemiologist at Makerere University College of Health Sciences. He is a member of the advisory committee. He harmonises strategies for case identification and infection prevention.

Dr Betty Kivumbi, is a mathematician, who is helping to develop Covid-19 models for Uganda. The models are used to predict how various measures being put or lifted could impact the extent of Covid-19 spread.

Prof Neoline Nakasujja, is a specialist in psychiatry and the Chair for the Department of Psychiatry where she serves as a senior lecturer at the Makerere University College of Health Sciences.

Prof Pauline Byakikais a Ugandan specialist physician and epidemiologist. She serves as an Associate Professor of Medicine at Makerere University College of Health Sciences.

Dr William Worodria, is a physician trained in pulmonology and clinical research. He is a member of the MSAAC. He has a Master of Medicine (Internal Medicine) from Makerere University, Kampala and a Doctorate from University of Antwerp, Belgium.

Dr Ethel Nakakawa, is a medical Microbiologist, College of Health Sciences, Makerere University. She holds a Masters in Medical and Diagnostic Virology.

Dr Arnold Kiwereza, is an anesthesiologist at Mulago National Referral Hospital who is also member of MSAAC.

Dr James Elima, is the director of Gulu Regional Referral Hospital.

Dr Byarugaba Baterana, is the executive director of Mulago National Referral Hospital.

Dr Moses Muwanga, is the director of Entebbe Grade B hospital.

Dr Susan Nabadda Ndidde, is the head of Central Public Health Laboratory that is also testing for coronavirus.

Prof Freddie Ssengooba Prof Freddie Ssengooba is a health policy expert heading the ad hoc committee on policy implication for Covid-19. He is in charge of assessing the public disposure and advises on the best way to put in place policies such as lockdown and when it should be lifted.

Prof Ssengooba is the director of the SPEED Project. SPEED refers to Support for Policy Engagements for Evidence-based Decisions for universal health coverage in Uganda.

Prof Ssengooba is also a lecturer in the Department of Health Policy, Planning and Management at the School of Public Health, College of Health Sciences, Makerere University.

He is a seasoned researcher in health systems and policy. In the last 12 years, Prof Ssengooba has led a programme of research on the organisational reforms like decentralisation of health services; autonomy and efficiency of hospitals; performance-based contracting and its impacts on health system in general and on the workforce in particular.

His current research engagements focus on the effectiveness of donor aid for HIV/Aids on the health system and researching the translation of evidence into policies and programmes.

He has provided technical services to World Health Organisation, World Bank, ministries of health, Uganda Aids Commission and multi-lateral and bilateral agencies.

Dr Monica Musenero Dr Monica Musenero is an epidemiologist and a veterinarian. She is the presidential adviser on epidemics.

She was among Ugandan experts who led the fight against Ebola outbreak in Sierra Leone in 2016 and DR Congo in 2018.Dr Musenero is a public health professional who has distinguished herself as a trailblazer in implementing strategic responses to medical emergencies.

Her service spans Ugandas Ministry of Health, local governments, higher education institutions, field epidemiology training programmes and engagements in various sub-Saharan countries.

She has engaged with multinational and multi-cultural leaders in both managerial and frontline contexts. Dr Museneros leadership versatility extends to initiatives that seek to build a critical mass of thought leaders to contribute to the countrys development agenda.

Dr Julius Lutwama Dr Julius Lutwama is a virologist leading the testing for coronavirus at UVRI. He has long-term experience in infectious disease diagnosis.

Dr Lutwama trained as an Entomologist, obtaining his PhD in 1991 and received further specialised training in molecular virology and entomology at the Centers for Diseases Control, Fort Collins, Colorado, US.

He is the acting deputy director of the Institute and he is the head of the Department of Arbovirology, Emerging and Re-Emerging Infectious Diseases at UVRI.

He also heads the WHO Collaborating National Influenza Centre and the Highly Infectious Diseases Diagnostic Laboratory at UVRI.

Dr Lutwama is Honorary Associate Professor in the Department of Medical Microbiology at the Makerere University College of Health Sciences.

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Gifted brains, hands in the Covid-19 fight - Daily Monitor

By Jonny Lupsha, News Writer

According to the Fierce Biotech article, the mice who underwent the new gene therapy were injected with a gene that makes the protein follistatin, which in turn blocks a protein called myostatin. Myostatin regulates muscle growth. The therapy caused a significant buildup of muscle mass in the mice while also preventing obesity, the article said. The mice didnt just build muscle; they also nearly doubled their strength without exercising any more than they usually did. Despite being fed a high-fat diet, they had fewer metabolic issues and stronger hearts than did animals that did not receive the follistatin gene.

Scientists have been developing gene therapy for many years. It can change our bodies in many ways, and has potential serving as a treatment for cancer and muscular dystrophy.

The procedure that the mice underwent encapsulates what gene therapy isalthough scientists generally focus on people.

I define [gene therapy] as the addition of genes to humans for medical purposes, said Dr. David Sadava, Adjunct Professor of Cancer Cell Biology at the City of Hope Medical Center.

Dr. Sadava said gene therapy is based on four assumptions. First, whoever is doing the gene therapy has to know the gene thats involved in whichever problem needs to be treated. Second, they must have a normal, healthy copy of that gene available in the lab. Third, they must know where and when the gene is normally expressed. Finally, they have to be fairly certain what will happen when the gene is expressed normally.

Additionally, gene therapy must do several things in order to be considered successful.

First, gene therapy must get the gene into the appropriate cells, Dr. Sadava said. Second, gene therapy must get the gene expressed in those cells. Third, we have to get the gene integrated into the genome of the target cells so itll be there permanently. And fourth, you better not have any bad side effects to gene therapy, like any therapy in medicine.

According to Dr. Sadava, one kind of gene therapy is referred to as gene augmentation, and it comes into play when the functional product of a gene has been lost and no longer gets produced normally. By injecting a gene into someone, healthy copies of a protein product will be made and function restored.

We could hypothetically think of muscular dystrophy as a good target for gene therapy, he said. We know that muscles lack the protein dystrophinits an organizing proteinso well put in the good gene for good dystrophin.

Another kind of gene therapy is called target cell killing. Dr. Sadava said it uses a gene that either produces a poison that kills certain types of cells or it stimulates the immune system to do so. Target cell killing can be applied to cancer.

A gene is put into cancer cells that allows them to produce a protein that will make a toxic drug from a harmless chemical, Dr. Sadava said. So the idea is we inject a harmless chemical into the body, it goes all over the body and when it enters a tumor cell, its converted into a poison by the gene product of the gene that weve put in for gene therapy. So we might put in a gene that will cause a protein to be made that attracts killer T cells so the tumor will stick up its hand and say Come kill me now.'

Gene therapy is an exciting field in science and medicine with a lot of potential for humans. For now, it may seem like its just helping some overweight mice get a confidence boost, but the practical applications should shore up within our lifetime.

Dr. David Sadava contributed to this article. Dr. Sadava is Adjunct Professor of Cancer Cell Biology at the City of Hope Medical Center in Duarte, CA, and the Pritzker Family Foundation Professor of Biology, Emeritus, at The Claremont Colleges. Professor Sadava graduated from Carleton University with a B.S. with first-class honors in biology and chemistry. He earned a Ph.D. in Biology from the University of California, San Diego.

The rest is here:
Lab Mice Shed Fat and Build Muscle with Gene Therapy - The Great Courses Daily News