Archive for the ‘Gene Therapy Doctor’ Category

Clinical data from three ongoing trials of Ad-RTS-hIL-12 plus veledimex (referred to as Controlled IL-12) for the treatment of recurrent or progressive glioblastoma multiforme (rGBM) and diffuse intrinsic pontine glioma (DIPG) were presented at the annual (virtual) meeting of the 2020 Society of NeuroOncology (SNO).

The data for the new treatment option, being developed by Ziopharm Oncology, included the first discussion of interim results of a phase II study of Controlled IL-12 in combination with cemiplimab (Libtayo; Regeneron Pharmaceuticals and Sanofi-aventis) for the treatment of rGBM that has recently completed enrollment, and updated interim data from the phase I study of Controlled IL-12 in combination with nivolumab for the treatment of rGBM and data from the first patient enrolled in the ongoing phase I/II study of Controlled IL-12 monotherapy for the treatment of DIPG.

Weve reported data for the first time from the ongoing phase II study of Controlled IL-12 in combination with PD-1 inhibitor cemiplimab, showing activation of the immune system across patients. These data are highly encouraging and underscore the potential of Controlled IL-12 to transform the treatment landscape of recurrent glioblastoma.

Interleukin-12Interleukin 12 or IL-12, a cytokine that is produced by dendritic cells, monocytes, and macrophages, and to a lesser extent by B-cells, induces TH1 differentiation in T-lymphocytes and the subsequent expression of interferon (INF). The cytokine belongs to the family of interleukin-12 which comprises the only heterodimeric cytokines, including IL-12, IL-23, IL-27, and IL-35.

IL-12 also promotes the expansion and survival of activated T-cells and NK cells and modulates the cytotoxic activity of CTLs and NK cells. During the adaptive immune response, IL-12 is known to primes antigen-specific T-cells for high IFN-g production, which drives the differentiation toward the Th-1 pathway. IL-12 can also act as an adjuvant for humoral immunity by enhancing the production of IgG2a and IgG2b antibodies, and it may enhance antibody production by B-cells.

Ziopharms Ad-RTS-hIL-12 plus veledimex is a gene therapy with an Adenoviral vector (Ad) providing the vehicle engineered to express IL-12 under the control of the RheoSwitch Therapeutic System (RTS). Ad-RTS-hIL-12 + veledimex has demonstrated a dose-related increase in tumor IL-12 mRNA and IL-12 protein expression. Discontinuation of veledimex resulted in a return to baseline IL-12 mRNA and protein expression in numerous syngeneic mouse tumor models. Image courtesy Ziopharm Oncology.[1]Although interleukin-12 (hIL-12) has anticancer activity, the systemic application is limited as a result of a toxic inflammatory responses. To solve the problem, scientists at Ziopharm assessed the safety and biological effects of an hIL-12 gene, transcriptionally regulated by an oral activator. [1]

Ziopharms Controlled IL-12 platform turns on the expression of IL-12 on demand, signaling for T-cells to attack and destroy cancerous tissue. As part of the treatment, genes coded to produce IL-12 are delivered to tumor sites. Following this step, patients are given a dose of veledimex to trigger the implanted genes to produce IL-12. This approach allows treating physicians to increase or decrease expression levels of IL-12 or turn it off altogether.[1]

PD-1 inhibitorThe updated data on combining Controlled IL-12 with nivolumab reveal a subset of patients with rGBM that demonstrate very encouraging survival at 16 months. This observation reveals that immune modulation with IL-12 and anti-PD-1 is well tolerated with an apparent survival benefit that will need further confirmation in upcoming more advanced clinical trials, said E. Antonio Chiocca, M.D., Ph.D., study investigator, Chairman of Neurosurgery at Brigham and Womens Hospital, Professor of Neurosurgery at Harvard Medical School, and Surgical Director of the Center for Neuro-oncology at Dana-Farber Cancer Institute.

These survival data in conjunction with previously reported MRIs showing partial responses is consistent with immune-mediated anti-tumor effects, Chiocca added.

CemiplimabThe results from the ongoing Phase II study of Controlled IL-12 in combination with PD-1 inhibitor cemiplimab for the treatment of adult patients with recurrent or progressive glioblastoma multiforme (rGBM), showed an activation of the immune system across patients with rGBM.

The combination trial demonstrated promising initial survival data, with median overall survival not yet reached. The data was comparable to results from a Controlled IL-12 monotherapy study previously presented at the 2020 annual meeting of the American Society of Clinical Oncology (ASCO).

Even with the best available therapies, the median overall survival for patients with rGBM is capped at 12 months. The Controlled IL-12 monotherapy has shown it can bring median overall survival to 16+ months.

Across its Controlled IL-12 studies to date, Ziopharm has identified a total of six partial responses, highlighting the promising potential of this therapy for the treatment of rGBM.

Remote-controlled therapyThe drug itself works like a remote-controlled therapy. The vector is injected directly into the tumor, but the gene of interest, IL-12, is under a transcriptional regulator, meaning that IL-12 is only expressed when the patient takes a drug that activates its transcription. Physicians can remotely control IL-12 expression by telling the patient when to take this pill.

Platform technologyAs we reflect on the growing body of evidence across our efforts utilizing our Controlled IL-12 platform, we are encouraged by the signs of efficacy we are seeing in these very hard-to-treat cancers. Not only are we observing cytokine production, increases in intra-tumoral T-cells, and predictable safety after treatment with Controlled IL-12 as a monotherapy and in combination with PD-1 inhibitors, but we have reported at least one partial response in each rGBM trial we have conducted to date, for a total of six, noted Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm

These MRI data, along with IL-12-driven immune response complement our encouraging survival data and we look forward to future data read-outs in 2021. Further, the initial look at data from the first patient in our phase 1/2 pediatric glioma study supports Controlled IL-12s safety profile and continued development, Cooper added.

Key studies: Controlled IL-12 + cemiplimabControlled IL-12 in combination with PD-1 inhibitor cemiplimab is currently being examined in a phase 2 study for the treatment rGBM (NCT04006119). Preliminary data in the on-demand presentation include:

Key studies: Controlled IL-12 + nivolumabControlled IL-12 in combination with the PD-1 inhibitor nivolumab is currently being examined in a phase I study for the treatment of rGBM. Interim data highlights shared in an oral on-demand presentation include:

As a follow up to our prior readout (ASCO 2020) for this combination which reported partial responses by MRI, the two patients had meaningful improvements in survival with one patient on 20 mg veledimex surviving 17.4 months and the other (10 mg veledimex) surviving 21.0 months (in follow up).

Diffuse Intrinsic Pontine GliomaControlled IL-12 monotherapy is being studied in a phase 1/2 dose-escalation study for the treatment of children with gliomas, including DIPG. Data highlights from the first patient in the study shared in a poster discussion, included:

It is important to note that these trials, including our previously disclosed monotherapy study, now consist of over 125 patients with rGBM. These provide deep learning that is ongoing and is part of the efforts to develop Controlled IL-12 as a potential therapy for brain cancers. We will continue to monitor the data across both the monotherapy and checkpoint inhibitor combination studies in the coming months. We believe there are multiple potential paths to registration for our Controlled IL-12 program, either as a monotherapy therapy or in combination with other agents, Cooper concluded.

Clinical trialsEvaluation of Ad-RTS-hIL-12 + Veledimex in Subjects With Recurrent or Progressive Glioblastoma, a Substudy to ATI001-102 NCT03679754A Study of Ad-RTS-hIL-12 With Veledimex in Subjects With Glioblastoma or Malignant Glioma NCT02026271Study of Ad-RTS-hIL-12 + Veledimex in Combination With Cemiplimab in Subjects With Recurrent or Progressive Glioblastoma NCT04006119A Study of Ad-RTS-hIL-12 With Veledimex in Combination With Nivolumab in Subjects With Glioblastoma; a Substudy to ATI001-102 NCT03636477A Study of Ad-RTS-hIL-12 + Veledimex in Pediatric Subjects With Brain Tumors Including DIPG NCT03330197

Highlights of Prescribing informationCemiplimab (Libtayo; Regeneron Pharmaceuticals and Sanofi-aventis) [Prescribing Information]Nivolumab (Opdivo; Bristol-Myers Squibb) [Prescribing Information]

Meeting presentationsLukas RV, Chiocca EA, OberheimBush NA, Landolfi J, Cavaliere R, Yu J, Kurz SC, Demars N, et al. Phase 2 Trial of Controlled IL-12 in Combination with PD-1 Inhibitor in Adult Subjects with Recurrent Glioblastoma (Abstract #901183) SNO 2020 Annual Meeting; Presented by: Rimas V. Lukas, MD [Presentation]Chiocca EA, Lukas RV, Chen CC, Rao G, Reardon D, Wen P, Bi WL, Peruzzi P, et al. Combination of Controlled Interleukin-12 Gene Therapy with Immune Checkpoint Blockade in Recurrent Glioblastoma: Updated Results of a Multi-Institutional, Open-Label Phase 1 Trial(Abstract #901050) SNO 2020 Annual Meeting; Presented by: Antonio Chiocca, MD.[Presentation]Goldman S, Mueller S, Chi S, Saratsis A, Allen R, Buck J, Demars N, Hadar N, Estupinan T, et al. Phase I/II Study of Controlled IL-12 as Immunotherapy for Diffuse Intrinsic Pontine Glioma (DIPG) (Abstract #901123), presented by Stewart Goldman, M.D., Division Head Hematology-Oncology, Neuro-Oncology & Stem Cell Transplantation at Lurie Childrens Hospital. [Poster]

Reference

[1] Barrett JA, Cai H, Miao J, Khare PD, Gonzalez P, Dalsing-Hernandez J, Sharma G, Chan T, Cooper LJN, Lebel F. Regulated intratumoral expression of IL-12 using a RheoSwitch Therapeutic System (RTS) gene switch as gene therapy for the treatment of glioma. Cancer Gene Ther. 2018 Jun;25(5-6):106-116. doi: 10.1038/s41417-018-0019-0. Epub 2018 May 14. PMID: 29755109; PMCID: PMC6021367.

Featured Image: Doctor report and recommend a method with patient treatment, results on brain x-ray film. Photo courtesy: 2020 Fotolia/Adobe. Used with permission.

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Encouraging Clinical Data for Controlled IL-12 for the Treatment of Glioblastoma and DIPG - OncoZine

PHILADELPHIA--(BUSINESS WIRE)--SwanBio Therapeutics (SwanBio), a gene therapy company advancing AAV-based therapies for the treatment of devastating, genetically defined neurological conditions, today announced the appointments of Patricia Patty Allen, former Chief Financial Officer of Zafgen, Inc.; Danny Bar-Zohar, M.D., Global Head of Development at Merck KGaA; and Alex Hamilton, Ph.D., Partner at Syncona, to the companys Board of Directors. In addition to her role as a non-executive Director, Ms. Allen will serve as the Chair of SwanBios Audit Committee.

SwanBio has been making tremendous progress in the advancement of our AAV-based pipeline of therapeutics for the treatment of neurological diseases, and we are thrilled to expand our Board of Directors with these key appointments, said Tom Anderson, Chief Executive Officer of SwanBio. Patty, Danny and Alex each bring a wealth of knowledge and expertise within their individual focus-areas, and their insights will be invaluable as we advance toward becoming a clinical company. We look forward to benefiting from their experience and collaborating on the important work ahead to deliver new medicines to patients with devastating diseases.

Im pleased to join the SwanBio Board of Directors, along with Danny and Alex, at this pivotal moment in the companys evolution, said Ms. Allen. SwanBio is driven by an exceptional team and founded based on innovative science, with a gene therapy approach that could make a significant difference for a number of people who suffer from genetic neurological diseases. I am excited for the future of this company and the opportunity to help guide them forward.

Ms. Allen is a business finance and operations leader with more than 25 years of experience leading private and public companies through initial public offerings, equity and debt financings, SEC reporting, investor relations, sell-side and buy-side, strategic and long-range planning, FP&A, treasury, risk management and business development. Most recently, she served as Chief Financial Officer of Zafgen, Inc. (now Larimar Therapeutics) from 2013-2020. Prior to Zafgen, Ms. Allen was an independent financial consultant from 2011-2012; served as Vice President of Finance, Treasurer and Principal Financial Officer of Alnylam Pharmaceuticals, Inc from 2004-2011; and as Director of Finance at Alkermes from 1992-2004. Ms. Allen also serves as a Director on the Board of Directors of several biotechnology companies and serves as the Chair of their Audit Committees. Ms. Allen graduated summa cum laude from Bryant College with a B.S. in business administration.

Dr. Bar-Zohar is a certified physician with proven expertise in drug development and a personal commitment to change peoples lives by developing sustainable, transformative healthcare solutions using both traditional and emerging technologies. He was recently appointed Global Head of Development at Merck KGaA in Darmstadt, Germany. Prior to Merck KGaA, Dr. Bar-Zohar was a Partner at Syncona and held various roles at Novartis Pharma AG from 2013-2020, most recently serving as Global Head, Clinical Development and Analytics. Before Novartis, Dr. Bar-Zohar held various clinical and medical affairs roles at Teva Pharmaceuticals Industries from 2006-2012. He obtained his medical doctor degree at the Sackler Faculty of Medicine, Tel-Aviv University and was trained in general surgery at the Tel-Aviv Medical Center.

Dr. Hamilton is an experienced financial leader within the biotech and pharmaceutical industries. Since SwanBios founding, Dr. Hamilton has played a key role in helping shape the companys strategy as a Board Observer. Before joining Syncona, he was a member of the Healthcare Investment Banking team at Jefferies International, where he worked on a range of financings and mergers and acquisitions across the biotechnology, pharmaceutical and healthcare sectors. Dr. Hamilton received his Ph.D. in immunology from the University of Cambridge.

About SwanBio Therapeutics

SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, genetically defined neurological conditions. SwanBio is advancing a pipeline of AAV-based gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications spastic paraplegias, monogenic neuropathies and polygenic neuropathies. SwanBios lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). For more information, visit SwanBioTherapeutics.com.

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SwanBio Therapeutics Expands Board of Directors with Appointments of Proven Industry Leaders - Business Wire

SwanBio has been making tremendous progress in the advancement of our AAV-based pipeline of therapeutics for the treatment of neurological diseases, and we are thrilled to expand our Board of Directors with these key appointments, said Tom Anderson, Chief Executive Officer of SwanBio. Patty, Danny and Alex each bring a wealth of knowledge and expertise within their individual focus-areas, and their insights will be invaluable as we advance toward becoming a clinical company. We look forward to benefiting from their experience and collaborating on the important work ahead to deliver new medicines to patients with devastating diseases.

Im pleased to join the SwanBio Board of Directors, along with Danny and Alex, at this pivotal moment in the companys evolution, said Ms. Allen. SwanBio is driven by an exceptional team and founded based on innovative science, with a gene therapy approach that could make a significant difference for a number of people who suffer from genetic neurological diseases. I am excited for the future of this company and the opportunity to help guide them forward.

Ms. Allen is a business finance and operations leader with more than 25 years of experience leading private and public companies through initial public offerings, equity and debt financings, SEC reporting, investor relations, sell-side and buy-side, strategic and long-range planning, FP&A, treasury, risk management and business development. Most recently, she served as Chief Financial Officer of Zafgen, Inc. (now Larimar Therapeutics) from 2013-2020. Prior to Zafgen, Ms. Allen was an independent financial consultant from 2011-2012; served as Vice President of Finance, Treasurer and Principal Financial Officer of Alnylam Pharmaceuticals, Inc from 2004-2011; and as Director of Finance at Alkermes from 1992-2004. Ms. Allen also serves as a Director on the Board of Directors of several biotechnology companies and serves as the Chair of their Audit Committees. Ms. Allen graduated summa cum laude from Bryant College with a B.S. in business administration.

Dr. Bar-Zohar is a certified physician with proven expertise in drug development and a personal commitment to change peoples lives by developing sustainable, transformative healthcare solutions using both traditional and emerging technologies. He was recently appointed Global Head of Development at Merck KGaA in Darmstadt, Germany. Prior to Merck KGaA, Dr. Bar-Zohar was a Partner at Syncona and held various roles at Novartis Pharma AG from 2013-2020, most recently serving as Global Head, Clinical Development and Analytics. Before Novartis, Dr. Bar-Zohar held various clinical and medical affairs roles at Teva Pharmaceuticals Industries from 2006-2012. He obtained his medical doctor degree at the Sackler Faculty of Medicine, Tel-Aviv University and was trained in general surgery at the Tel-Aviv Medical Center.

Dr. Hamilton is an experienced financial leader within the biotech and pharmaceutical industries. Since SwanBios founding, Dr. Hamilton has played a key role in helping shape the companys strategy as a Board Observer. Before joining Syncona, he was a member of the Healthcare Investment Banking team at Jefferies International, where he worked on a range of financings and mergers and acquisitions across the biotechnology, pharmaceutical and healthcare sectors. Dr. Hamilton received his Ph.D. in immunology from the University of Cambridge.

About SwanBio Therapeutics

SwanBio Therapeutics is a gene therapy company that aims to bring life-changing treatments to people with devastating, genetically defined neurological conditions. SwanBio is advancing a pipeline of AAV-based gene therapies, designed to be delivered intrathecally, that can address targets within both the central and peripheral nervous systems. This approach has the potential to be applied broadly across three disease classifications spastic paraplegias, monogenic neuropathies and polygenic neuropathies. SwanBios lead program is being advanced toward clinical development for the treatment of adrenomyeloneuropathy (AMN). For more information, visit SwanBioTherapeutics.com.

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SwanBio Therapeutics Expands Board of Directors with Appointments of Proven Industry Leaders - BioSpace

MedicalResearch.com Interview with:

Richard C. Austin, PhDProfessor and Career Investigator of the Heart and Stroke Foundation of OntarioAmgen Canada Research Chair in NephrologyMcMaster University and St. Josephs HealthcareHamilton, Ontario, Canada

MedicalResearch.com: What is the background for this study? What are the main findings?

Response: A previous study published in 2011 by my collaborator, Dr. Michel Chretien at the IRCM, identified a rare mutation in the PCSK9, termed Q152H. Individuals harboring this mutation demonstrated dramatic reductions in their LDL cholesterol levels and had a significantly lower risk of CVD. Furthermore, individuals harboring the Q152H mutation showed increases in longevity with no evidence of other diseases such as liver disease, cancer and chronic kidney disease.This Q152H mutation was unique with only 4 families in Quebec shown to harbor this genetic variant.

In terms of its effect on PCSK9 expression/activity, themutation at Q152H was precisely at the cleavage site in PCSK9 necessary for its activation. As a result, the Q152H mutation fails to be cleaved and activated, thereby blocking its secretion into the circulation. This is why the Q152H mutation is considered a loss-of-function PCSK9 mutant. Given our labs interest in endoplasmic reticulum (ER) stress and ER storage diseases, we began to collaborate with Drs. Chretien and Seidah at the IRCM to investigate whether this Q152H mutant, when overexpressed in liver cells, would cause ER stress and liver cell injury. This was based on the findings that the Q152H mutant does not undergo autocatalytic cleavage and itssubsequent secretion from liver cells.

It is well known in the literature that the accumulation of misfolded or inactive proteins in the ER gives rise to ER stress and cell injury/dysfunction. As a result, we initially showed to our surprise that overexpression of the Q152H mutant in liver cells failed to cause ER stress BUT increased the protein levels of several important ER chaperones, GRP78 and GRP94, known to PROTECT against liver cell injury/dysfunction. As part of our JCI study, we furthered these studies to examine the effect of the Q152H mutant when overexpressed in the livers of mice. This is where we demonstrated that the Q152H mutation showed protection against ER stress-induced liver injury/dysfunction.

MedicalResearch.com: What are the main findings? How is this gene variant related to the PCSK9 inhibitor evolocumab (Repatha)?

1. That the Q152H variant is considered a loss-of-function PCSK9 variant that protects against CVD by lowering LDL cholesterol levels. The reason it is a loss-of-function variant is because the protein fails to be secreted out of liver cells because it is retained in the ER of hepatocytes.

In terms of its relation to evolocumab, there are some major differences. Evolocumab is a fully human monoclonal antibody against PCSK9 that can bind to circulating PCSK9 and block its ability to bind to the LDL receptor on the surface of the liver. As a result, the levels and half life of the LDL receptor increases on the surface of hepatocytes, thereby increasing the uptake of LDL cholesterol. You could say that evolocumab acts like a loss-of-function PCSK9 variant. As indicated above, the Q152H variant blocks the autocatalytic cleavage of PCSK9 and its secretion from the ER of hepatocytes. As a result, the Q152H PCSK9 protein is retained in the ER of hepatocytes and fails to be secreted into the blood.

MedicalResearch.com: What should readers take away from your report?

Response: That mutations in the PCSK9 gene that affect its activation may act to protect against several human diseases and may contribute to increaselongevity and good health. Therefore, gene therapy approaches or new medicines that block the secretion of PCSK9 protein from the liver may have multiple beneficial health effects.

MedicalResearch.com: What recommendations do you have for future research as a result of this study?

Response: We recommend that future research will allow us to determine whether gene therapy to express the Q152H variant or small molecule inhibitors of PCSK9 activation in the liver are novel approaches aimed at protecting against BOTH CVD and liver disease. WE are now in the process of generating a Q152H knockin mouse (using CRISPR/Cas9) to better understand the health benefits of the Q152H variant. This unique mouse model will allow us to perform a number of novel studies aimed at understanding the role of the Q152H variant in tissues/organs that are know to express PCSK9, such as liver, small intestine, kidney and brain.

MedicalResearch.com: Is there anything else you would like to add? Any disclosures?

Response: This study has utilized both clinical and biomedical approaches to better understand how a rare mutation in the PCSK9 gene can have a number of surprisingly protective effects on several different diseases.

Citation:

Paul F. Lebeau, Hanny Wassef, Jae Hyun Byun, Khrystyna Platko, Brandon Ason, Simon Jackson, Joshua Dobroff, Susan Shetterly, William G. Richards, Ali A. Al-Hashimi, Kevin D. Won, Majambu Mbikay, Annik Prat, An Tang, Guillaume Par, Renata Pasqualini, Nabil G. Seidah, Wadih Arap, Michel Chretien, Richard C. Austin.The loss-of-function PCSK9Q152H variant increases ER chaperones GRP78 and GRP94 and protects against liver injury.Journal of Clinical Investigation, 2020; DOI:10.1172/JCI128650

The information on MedicalResearch.com is provided for educational purposes only, and is in no way intended to diagnose, cure, or treat any medical or other condition. Always seek the advice of your physician or other qualified health and ask your doctor any questions you may have regarding a medical condition. In addition to all other limitations and disclaimers in this agreement, service provider and its third party providers disclaim any liability or loss in connection with the content provided on this website.

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Rare Mutation in the PCSK9 Gene Confers Long Healthy Life - MedicalResearch.com

Among the imminent miseries worldwide, the mortalities and morbidities caused by the combination of covid-19 and Diabetes Mellitus are considered to be the most tragic. The pandemic has created a pandemonium and is a byword for misery. Since the beginning of the 21st century, the viruses of Nidovirales order have affected the humanity three memorable times: SARS-CoV in 2002, MERS-CoV in 2012 and SARS-CoV-2 in 2019-2020. Coronaviruses are large, enveloped and positive-stranded RNA viruses. The SARS-CoV-2 consists of an envelope which is made of spikes, membrane and envelope glycoproteins. Inside the envelope, there is the Nucleocapsid that is formed from multiple copies of the N proteins. The Nucleocapsids are perennially attached with te RNA of the virus. The Spike protein is implicated in binding to the ACE2 receptors that are located on the cell surfaces of the respiratory tract of human body. The pinioning of viral envelope and cell membrane of human cell is followed by the release of the viral genome into the target cell.

The clinical symptoms of covid-19 are fever, dry cough, malaise, diarrhea, dyspnea and pneumonia. Lab findings include lymphopenia and bilateral ground glass opacity or consolidation that is observed in Chest CT-scans. Covid-19 can be confirmed by real time RT-PCR and IgM plus IgG antibody tests.

The pharmacists and biotechnologists must track down a veracious antiviral that should eradicate the disease once and for all. Furthermore, the inhabitants of the globe should come forward to voice their apprehensions regarding any possibility of biological warfare in the New Cold War era that has already exhibited and manifested itself since the beginning of the 21st century

As a doctor in the pandemic, I made some observations regarding diabetes mellitus and unintentional childhood injuries at home. Diabetes Mellitus refers to a group of common metabolic disorders sowing as hyperglycemia. The factors contributing are reduced insulin secretion, decreased glucose utilization and increased glucose production. Type 1 DM is a subtype of diabetes mellitus (DM) due to an autoimmune beta-cell destructive process mediated by T-lymphocytes. It can transpire at any age but it frequently manifests itself before 30. It is estimated that between five and ten percent of those developing DM after the age of 30 years have Type 1 Diabetes Mellitus (DM). Another subtype of Diabetes Mellitus is called MaturityOnset Diabetes of the Young (MODY) that is characterized by autosomal dominant inheritance, early onset of hyperglycemia (usually under 25) and impairment in nsulin secretion. MODY is divided into MODY 1, 2, 3, 4, 5, 6 and so on. Other subtypes of DM are Gestational Diabetes and Type 2 Diabetes Mellitus. Type 2 Diabetes Mellitus is characterized by Impaired Insulin Secretion, Insulin Resistance, Excessive Hepatic Glucose Production and Abnormal Fat metabolism.

The acute complications of DM are Diabetic Ketoacidosis and Hyperglycemic Hyperosmolar State (HHS). The micro-vascular complications of DM are Retinopathy, Neuropathy and Nephropathy. The macrovascular complications are Coronary Heart Disease, Peripheral Arterial Disease and Cerebrovascular Disease. Other complications include Diabetic Foots and Ulcers. In the light of all the aforementioned complications of DM, simultaneous COVID-19 disease can lead to dreadful effects. This group of patients needs our sympathy.

Another hallmark of interest is the unintentional injuries of children at home, especially under the age of five. These injuries mostly occur due to burns, drowning, poisoning and fall and are responsible for the mortality of round about seven percent among 875,000 deaths of children each year. According to a report of Centers for Disease Control and Prevention (2001), In the USA, more children between the ages of one and four die annually from unintentional injuries than from all childhood diseases combined. Furthermore, For every childhood death caused by injury, there are approximately 18 hospitalizations, 233 emergency department visits, many more visits to medical facilities, and a much larger number of home-treated injuries. The factors associated with unintentional injuries at home are physical, biological and social environments. During the coronavirus pandemic, there is a dire need to minimize such types of injuries for reducing hospital admissions and stays, which are high-water marks for contamination of COVID-19 in third world countries.

To beat the extremities of agoraphobia and of the purgatory-like scenes around the world, the philanthropists and community of scientists must come forward to ferret out a pragmatic screenplay of active and passive immunity against SARS-CoV-2. Research should be carried out to discover a meaningful vaccine and synthetic immunoglobulins. Though, Pfizer and BioNTech in November 2020 released the details of a vaccine that could prevent more than 90 percent of people from getting covid-19, still much more efforts are required from brains and brawn of humanity. Antivirals like Favipiravir and Remdesivir are already approved for elimination of this menace in some countries but the pharmacists and biotechnologists must track down a veracious antiviral that should eradicate the disease once and for all. Furthermore, the inhabitants of the globe should come forward to voice their apprehensions regarding any possibility of biological warfare in the New Cold War era that has already exhibited and manifested itself since the beginning of the 21st century.

In order to give hope to diabetic patients who also suffer from covid-19, the whole health community should devise plans to find selective TGF- inhibitors. Studies should also be carried out on inhibition of Inflammatory Signalling Pathways such as Nuclear Factor kB (NF-kB) pathway. Similarly, gene therapy by viral vector or by non-viral transduction and stem cells curing techniques should be explored worldwide for the best results. VEGF-A inhibition therapy in retinopathic cases should be provided at nearby primary health care centres by a covid-19-free healthcare professional if possible. Incretin analogues may also be supplied to deserving patients. The depression and anxiety of these patients need to be reduced and for this purpose, psychiatrists and psychologists are advised to play their lead role.

Though Duloxetine is the drug of choice for curtailment of neuropathic pain, the clinicians and policy makers should ponder over the scenario as an organic whole that also includes factors like poverty, environment and trust capital. Vitamin B-12 (Cobalmin) should be proffered free of cost to diabetic patients. Antibiotics that can act against MRSA should be included in the essential drugs list of hospitals during the pandemic.

Policies should be shaped to bring into existence a safe environment within abodes for children during the pandemic and an awareness session must be arranged via media to educate the parents with more emphasis on keeping dangerous objects out of the reach of children.

Another way of bringing new hope and blessings to humanity during the pandemic is via introducing electronic government in every nook and corner of this world. Electronic governance comprises electronic administration, electronic services, electronic democracy and electronic business. The sowing of seeds of biometric voting system in elections and transformation of the revenue system by replacing conventional means should be spearheaded without any delay all over the world during the pandemic. Furthermore, the social security laws and system all over world should be made more labor/employee-friendly so that the poor (excluding rich) victims of unemployment in private sector institutions during this pandemic can satisfy their mouth and stomach. It is also added that States all over the world may initiate debt-free and interest-free currencies for a short period of time to bear the expenses during this pandemic. The humanity can emulate the Bradbury Pound initiative or the Constitutional Monetary System of Lincoln for a period of two or three months. Similarly, single/solitary person tourism should be encouraged to beat the woes of anxiety and depression.

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Living through the second wave - Pakistan Today

NEW YORK Would you buy something you cant see?

Thats the question posed by two New York brothers whove lost much of their vision to a rare degenerative eye disorder and have dedicated their lives and livelihoods to raising money for a cure.

Bradford Manning, 35, and his 30-year-old brother, Bryan, are the founders of the clothing brand Two Blind Brothers. Theyve hit on a strategy thats helped raise more than $700,000 for the cause: selling mystery boxes full of an assortment of their ultra-soft shirts, cozy socks, knit beanies and sunglasses.

The two turn over all profits from the boxes and their other sales to groups like the Foundation Fighting Blindness, funding research on retinal eye ailments like the one theyve suffered from since they were 5, Stargardt disease. Its an inherited form of macular degeneration that causes central vision loss over time.

We just wanted to try and help and raise awareness, and just do something good, said Bryan.

Since 2016, when they left their previous careers Brad worked for an investment firm and Bryan sold software theyve picked up celebrity supporters like Ice-T and entrepreneur Richard Branson. Ellen DeGeneres helped with one of her famous Shutterfly checks for $30,000.

And the sale of their mystery boxes, costing from $30 to $200, is now a social media phenomenon.

Customers have included relatives of the blind, among them parents with vision impaired children; some have posted unboxing videos on TikTok, Facebook and Instagram, with a few opening boxes blindfolded.

The brothers hit on the idea in 2015, when a gene therapy discovered by an underfunded researcher for an unrelated juvenile eye disease was about to hit the market.

It was mind boggling to us, Bryan said. Our whole lives they were like, Oh, a cure is down the line, a cure is down the line. This one isnt for us, but it is happening, and the Foundation Fighting Blindness kicked this off with just a tiny charitable gift to this brilliant researcher.

Soon after, they were separated while shopping at Bloomingdales. When they reconnected, they found that they had purchased the same soft shirt.

It was the feel of it. It felt so soft and comfortable that we both keyed upon it, and then we had this idea, well what if we could take this sense of touch to a different place, make super comfortable clothing and turn over the profits to researchers at work on eye diseases, Bryan said.

With advice from friends in the fashion industry, two blind brothers became Two Blind Brothers.

The casual line of super soft Henleys, hoodies, polos and T-shirts for men and women, along with offerings for kids, are made of sustainable bamboo mixed with cotton and spandex. Theyve incorporated Braille indicating the color of each garment into some of the designs they sell online at Twoblindbrothers.com.

The goods were originally manufactured in Texas, mostly by visually impaired people. But as theyve grown, most of the operation moved to Los Angeles.

Brad was diagnosed at 7 after their mother, a nurse, found a doctor who determined that Stargardt disease was the cause of his declining vision.

The doctor told her to take him home, get him a magnifier and maybe teach him Braille, and good luck, Bryan recalled. But they would not give up on their sons. Today, their condition has slowed, leaving both with peripheral vision.

The brothers include their story in every mystery box, with some special thanks.

When someone shops blind, they prove something remarkable, they write. They prove that genuine trust is real.

Read the original here:
How two blind brothers became Two Blind Brothers for a cause - The Columbian

Would you buy something you cant see?Thats the question posed by two New York brothers whove lost much of their vision to a rare degenerative eye disorder and have dedicated their lives and livelihoods to raising money for a cure.Bradford Manning, 35, and his 30-year-old brother, Bryan, are the founders of the clothing brand Two Blind Brothers. Theyve hit on a strategy thats helped raise more than $700,000 for the cause: selling mystery boxes full of an assortment of their ultra-soft shirts, cozy socks, knit beanies and sunglasses.The two turn over all profits from the boxes and their other sales to groups like the Foundation Fighting Blindness, funding research on retinal eye ailments like the one theyve suffered from since they were 5, Stargardt disease. Its an inherited form of macular degeneration that causes central vision loss over time.We just wanted to try and help and raise awareness and just do something good, said Bryan.Since 2016, when they left their previous careers Brad worked for an investment firm and Bryan sold software theyve picked up celebrity supporters like Ice-T and entrepreneur Richard Branson. Ellen DeGeneres helped with one of her famous Shutterfly checks for $30,000. And the sale of their mystery boxes, costing between $30 to $200, is now a social media phenomenon.Customers have included relatives of the blind, among them parents with vision impaired children; some have posted unboxing videos on TikTok, Facebook and Instagram, with a few opening boxes blindfolded. The brothers hit on the idea in 2015, when a gene therapy discovered by an underfunded researcher for an unrelated juvenile eye disease was about to hit the market. It was mind boggling to us, Bryan said. Our whole lives they were like, 'Oh, a cure is down the line, a cure is down the line.' This one isnt for us, but it is happening, and the Foundation Fighting Blindness kicked this off with just a tiny charitable gift to this brilliant researcher.Soon after, they were separated while shopping at Bloomingdales. When they reconnected, they found that they had purchased the same soft shirt.It was the feel of it. It felt so soft and comfortable that we both keyed upon it, and then we had this idea, well what if we could take this sense of touch to a different place, make super comfortable clothing and turn over the profits to researchers at work on eye diseases, Bryan said.With advice from friends in the fashion industry, two blind brothers became Two Blind Brothers.The casual line of super soft Henleys, hoodies, polos and T-shirts for men and women, along with offerings for kids, are made of sustainable bamboo mixed with cotton and spandex. Theyve incorporated Braille indicating the color of each garment into some of the designs they sell online at Twoblindbrothers.com.The goods were originally manufactured in Texas, mostly by visually impaired people. But as theyve grown, most of the operation moved to Los Angeles. Brad was diagnosed at 7 after their mother, a nurse, found a doctor who determined that Stargardt disease was the cause of his declining vision. The doctor told her to take him home, get him a magnifier and maybe teach him Braille, and good luck, Bryan recalled. But they would not give up on their sons. Today, their condition has slowed, leaving both with peripheral vision. The brothers include their story in every mystery box, with some special thanks.When someone shops blind, they prove something remarkable, they write. They prove that genuine trust is real.

Would you buy something you cant see?

Thats the question posed by two New York brothers whove lost much of their vision to a rare degenerative eye disorder and have dedicated their lives and livelihoods to raising money for a cure.

Bradford Manning, 35, and his 30-year-old brother, Bryan, are the founders of the clothing brand Two Blind Brothers. Theyve hit on a strategy thats helped raise more than $700,000 for the cause: selling mystery boxes full of an assortment of their ultra-soft shirts, cozy socks, knit beanies and sunglasses.

The two turn over all profits from the boxes and their other sales to groups like the Foundation Fighting Blindness, funding research on retinal eye ailments like the one theyve suffered from since they were 5, Stargardt disease. Its an inherited form of macular degeneration that causes central vision loss over time.

We just wanted to try and help and raise awareness and just do something good, said Bryan.

Since 2016, when they left their previous careers Brad worked for an investment firm and Bryan sold software theyve picked up celebrity supporters like Ice-T and entrepreneur Richard Branson. Ellen DeGeneres helped with one of her famous Shutterfly checks for $30,000.

And the sale of their mystery boxes, costing between $30 to $200, is now a social media phenomenon.

Customers have included relatives of the blind, among them parents with vision impaired children; some have posted unboxing videos on TikTok, Facebook and Instagram, with a few opening boxes blindfolded.

The brothers hit on the idea in 2015, when a gene therapy discovered by an underfunded researcher for an unrelated juvenile eye disease was about to hit the market.

It was mind boggling to us, Bryan said. Our whole lives they were like, 'Oh, a cure is down the line, a cure is down the line.' This one isnt for us, but it is happening, and the Foundation Fighting Blindness kicked this off with just a tiny charitable gift to this brilliant researcher.

Soon after, they were separated while shopping at Bloomingdales. When they reconnected, they found that they had purchased the same soft shirt.

It was the feel of it. It felt so soft and comfortable that we both keyed upon it, and then we had this idea, well what if we could take this sense of touch to a different place, make super comfortable clothing and turn over the profits to researchers at work on eye diseases, Bryan said.

With advice from friends in the fashion industry, two blind brothers became Two Blind Brothers.

The casual line of super soft Henleys, hoodies, polos and T-shirts for men and women, along with offerings for kids, are made of sustainable bamboo mixed with cotton and spandex. Theyve incorporated Braille indicating the color of each garment into some of the designs they sell online at Twoblindbrothers.com.

The goods were originally manufactured in Texas, mostly by visually impaired people. But as theyve grown, most of the operation moved to Los Angeles.

Brad was diagnosed at 7 after their mother, a nurse, found a doctor who determined that Stargardt disease was the cause of his declining vision.

The doctor told her to take him home, get him a magnifier and maybe teach him Braille, and good luck, Bryan recalled. But they would not give up on their sons. Today, their condition has slowed, leaving both with peripheral vision.

The brothers include their story in every mystery box, with some special thanks.

When someone shops blind, they prove something remarkable, they write. They prove that genuine trust is real.

The rest is here:
How two blind brothers created a one-of-a-kind shopping experience to raise money for a cure - WXII The Triad

As my colleagues and I first described in 2014, we can use CRISPR genome editing to duplicate the most powerful form of gene drive, a ubiquitous natural phenomenon that happens when a genetic change is inherited more frequently than usual. Encode the CRISPR machinery next to a useful edit weve made in the genome, and genome editing will reoccur in every generation, replacing the original with the edited version without limit. In principle, releasing such organisms would gradually alter entire wild populations and associated ecosystems.

Thankfully, driven changes spread slowly and can be readily overwritten, which means that, unlike pandemic viruses, gene drive would be hard to misuse. If developed wisely, safe and controllable ecological editing could help eradicate animal-borne diseases such as malaria, humanely control rodents and insect pests without using poisons, and solve increasingly urgent conservation problems by harnessing tools already present in nature.

Unfortunately, many of these applications are being developed in secret not because my colleagues prefer it that way, but because current scientific incentives still favor secretive research.

That was deeply troublesome even before the pandemic. Today, it sets a precedent that scientists can pursue self-spreading technologies with no required safeguards and negligible public oversight.

Moreover, developing interventions to change our environment is fundamentally different from medical research. If your doctor recommends that you take a new drug, you can always decline. But if we develop interventions to change our environment, and a community decides to use them, everyone there will be affected. We know that actively inviting and responding to concerns and criticism from the public can identify and correct flaws early enough to make a difference so why not here?

A 2016 report by the US National Academies concurred, noting, The best course of action is to ensure that the people who could be affected by a proposed project or policy have an opportunity to have a voice in decisions about it. Experts acting alone will not be able to identify or weigh the true costs and benefits of gene drives.

Its now 2020, and most research still takes place out of public view. Society rewards the first to publish, causing most scientists to keep their experimental plans to themselves lest someone else throw more money and hands at a promising idea and take all the credit. The system endures, even though closeted research is both less efficient and more dangerous: Small teams of similarly trained specialists working in isolation cant reliably predict the consequences of their work, nor invite others to help them improve their designs.

Despite years of lobbying by many of us in the field, funders of gene-drive research have struggled to agree on which safeguards should be required to prevent accidental releases of gene-drive systems into the wild, let alone require transparency and community guidance. Publishers of high-profile journals have similarly declined to act. Crucially, both have cited the absence of a central organizing body to establish standards.

Unlike COVID-19, this is a problem that the World Health Organization can easily solve in this case, by creating a registry for ecological editing research just like they did for CRISPR gene therapy. Doing so would improve our science, our ethics, and the odds that these technologies will one day save millions of lives, not to mention set a better precedent for future research into self-spreading agents.

The question is what should be required to register a project. Based on our work with the communities of Nantucket and Marthas Vineyard on immunizing the local white-footed mice to prevent transmission of Lyme disease which by community preference doesnt involve gene drive or any DNA not found in wild mice weve identified a few key criteria:

Scientists should disclose the nature of the proposed alteration and why its worth considering. People deserve to know enough about the project to suggest changes and share concerns.

Researchers should assess the risk of accidental spread and detail any safeguards that would prevent it from happening if organisms should escape or be deliberately released from a laboratory or field trial site.

Any projects aiming to edit an organism responsible for a real-world problem should require sponsorship by a community interested in pioneering the application who will help to guide the research.

Most important, scientists should be required to register their plans before any technology development begins. Its easier to make changes in response to advice from peers and interested communities before becoming too invested and, in a field where a single mistake could affect millions, its best to invite external advice early on.

An ecological editing registry endorsed by funders, major journals, and scientific societies would enable the high-profile discipline of gene-drive research to serve as a field trial for open and community-responsive technology development, with beneficial effects that could spread far beyond genome editing.

Thats not to say that all research should be open; we certainly dont want to disclose how to make a pandemic, should we be so unlucky as to live in a universe in which its possible. Rather, we should establish transparent, publicly accessible standards to help determine whether, when, and how research that could impact everyone should proceed. Speaking as a scientist who bears a share of moral responsibility for the most prominent self-spreading technology, the best time to do this was several years ago but this year will do nicely.

Kevin Esvelt is an assistant professor at the MIT Media Lab, where he directs the Sculpting Evolution group. An evolutionary engineer, he is an inventor of CRISPR-based gene drive and a founder of the community-guided Mice Against Ticks project to prevent Lyme disease.

Read this article:
The ethical way to alter organisms - The Boston Globe

Introduction

Last week, the Teladoc (TDOC) and Livongo (LVGO) merger was completed. That means that Livongo doesn't trade anymore. If you still had your Livongo shares, you got (or will get if your broker is a bit slower) 0.5920 per share of Livongo in Teladoc shares plus cash of $11.33, paid with a special dividend of $7.09 on October 29th, and the rest when your shares were changed to Teladoc shares.

(Source)

As a former shareholder of Livongo, you may not be completely familiar with how Teladoc is positioned now. If there is a buy-out or a merger, that always generates mixed feelings, or at least it should. If it doesn't, it means that you had a bad stock in your portfolio.

With Potential Multibaggers, my marketplace service here on Seeking Alpha, I try to find multibaggers early on. I picked Livongo as a Potential Multibagger on December 26, 2019. The stock then traded at $24.86 and had a market cap of just $2.5B. The stock returned 462.2%, so it's more than a fivebagger in less than a year. But still, quite a few shareholders, from both companies, didn't feel great about the merger.

I think Teladoc could still be a multibagger at this point for patient investors. I think most investors underestimate that this combined company could represent the future of our healthcare system. In this article, written from a bird's eye perspective, I will try to explain why.

I think a lot of people know that the American healthcare system (and that of most Western countries) is unsustainable. It's too expensive but nobody seems to find a way out to cut costs.

The problem is that our healthcare is one of the last sectors that has not been disrupted by tech yet. The system originates from a time when bigger was better because it was more affordable to have standard procedures. Long ago, there was a family doctor and he knew you and you knew him and you had a personal relationship with that man (female doctors almost didn't exist back then).

After the Second World War, two evolutions emerged that made this system unsustainable: people reached a higher and higher age and the Baby Boomers were born. That put pressure on both ends of the healthcare system and the solutions were more scale and introducing standard procedures, so the productivity of healthcare workers became higher. Specialized care also contributed to more efficiency. And it worked.

But there was a side effect. People don't feel connected to the people that should care for them. They often feel treated like numbers, like patients at best, but mostly not like individual people. I'm not throwing a stone here at doctors, nurses and other healthcare workers. They often share that feeling. They don't have the time to deeply care about people. Their time is limited, they have to reach the quota. A doctor is not paid for listening to you. He's paid per patient that he handles. In other words, the less he or she listens, the more the doctor is incentivized. And that wears out a lot of healthcare workers.

Normally, such a market would correct itself. If you are not properly served in Lowe's (LOW), you go to Home Depot (HD) or the other way around. If one is really not giving enough attention to its clients, the company will go bankrupt eventually. That's where the efficiency of the market plays its role.

But in healthcare, the patients are not the customers. They are the goods, as it were. Customers are the paying party. And who pays for healthcare costs? Exactly, the insurance companies. Their only objective is as little costs as possible and that's why they pay per visit, for example.

But this creates the strange effect that a doctor that treats you very well is only paid once, while a bad doctor, who follows the wrong procedure and has to repair the damage or gives the wrong diagnosis, is rewarded each time he or she treats you and so earns more money than the good doctor.

To fix the healthcare system, a little reparation here and there won't help. We saw in the last decades that tech has entered almost every industry and has disrupted industries completely. Think of how Amazon (AMZN) made Sears obsolete. The same thing should (and probably will) happen in healthcare.

Disruption comes from the Latin verb 'disrumpere', which means to break apart and that is what healthcare needs: breaking things apart to build them up again. The bottom line of every healthcare reform should be to use tech and turn the system upside down. Health should be rewarded and paid for, not sickness.

All insurance is meant for emergency cases, except for healthcare insurance. Doctors are incentivized to do as many consults and tests as possible because someone makes money on that: the doctor himself or the hospital, mostly both. A hospital, for example, makes 10 or 20 times more money if you go to the emergency room than if you use an online platform to talk to a telehealth doctor.

As a patient, if you see a doctor, most of that little time you spend with him or her is dedicated to tests, collecting data. But suppose the doctor already would have all the data when you come in and he or she has already been able to look into your case, your history, and tens of up-to-date data points before you came in, that doctor could have time for that which we all crave when we visit a doctor: talking about what you exactly have and what it means, what we can do to get better, a discussion about what the underlying cause could be, talking about the psychological effects that your condition brings with it, what the best plan of action would be for you, etc. In other words, the doctor could become some kind of health coach, a professional that, with the help of precise data, could prescribe a trajectory to better health, hold you accountable, help you when sticking to the plan is tough etc. A doctor could partly become a real healthcare worker, not just a sickcare worker.

That would mean that you wouldn't have to visit a doctor as often. If you have a chronic condition, you could be monitored 24/7 by sensors, assisted by AI, as Livongo does for diabetes. You are not only monitored but you also get health nudges. That means that you would know exactly what is the right path for you. And that path is much more individualized than most people can imagine.

Hemant Taneja, a venture capitalist of General Catalyst who founded Livongo with Glen Tullman, calls this new space in healthcare 'health assurance'.

(Hemant Taneja, right, together with Livongo founder Glen Tullman, source)

Taneja is convinced that health assurance industry will see several $100B+ companies. I suspect that he was the great driving force behind the Livongo/Teladoc merger, together with Glen Tullman. They realized that Livongo can become so much more combined with Teladoc than on its own.

This is how health assurance is defined:

Health assurance is an emerging category of consumer-centric, data-driven healthcare services that are designed to bend the cost curve of care and help us stay well. Built on the principles of open technology standards, these services employ empathetic user design and responsible AI. This is the future of your health experience.

If you want to know more about this, you could read Taneja's interesting book Unhealthcare.

(Source)

Now that you know the basics of the concept of health assurance, I think you can see the potential for Teladoc and Livongo in this market. There is no company that is as well-positioned as the combination of Teladoc and Livongo. Livongo brings measuring, data collection and especially AI to the table, Teladoc has a worldwide network of telehealth.

Investors who just see what there is now could get scared away from Teladoc. They look at the number of telehealth visits that could slow down after the peak of the coronavirus and they are afraid that Teladoc will just have been a COVID-19-induced fad. But if you look at the future, you see that this company could be in the sweet spot when healthcare will be disrupted over the next decade.

Insurance companies will help them with this. As we have already seen, they just want to pay as little as possible. Livongo saved insurance companies $88 per patient per month. The reason: the monitoring of diabetes patients, the fact that they have a diabetes coach that is always available and the health nudges reduce the medical costs dramatically. $88 per patient per month means more than $1,000 per year per patient. I think you see the potential.

Besides that, the patients also feel freer than before. Their diabetes doesn't control their lives as much. They need less medication and if they need it, the data will indicate it before the attack.

This is just for diabetes. That's already a big market. But Livongo doesn't just focus on diabetes but also on hypertension and obesity. Those are two huge markets as well. The hypertension market is estimated to be $23B in 2026 and the obesity treatment market is estimated to be around $20B in 2026. But suppose you add the weight loss market to this, which is worth about $70B in the US alone, and you can see the potential.

The obesity-related healthcare costs are estimated at around $147B annually, so this might mean big savings for healthcare spending.

There will certainly be other companies in this space than just Livongo and Teladoc, companies that will also focus on other domains, but so far, I don't see any competitor that is as advanced as Livongo/Teladoc is.

When I pick a Potential Multibagger, I turn a company inside out and that means that I know Livongo, its founders Glen Tullman and Hemant Taneja and what the company exactly does really well.

I didn't know Teladoc as well before the merger was announced. I had it on my watchlist, but I had not done a really deep dive. When I did, I found a lot that I liked. This statement by Teladoc's CEO Jason Gorevic on the closing of the merger is worth reading word for word if you want to understand the combination of the companies:

Both Teladoc Health and Livongo were founded with the same mission: to create a new kind of healthcare experience, one that empowers people everywhere to live their healthiest life. Today's news (the merger, FGTV) dramatically accelerates our ability to make this a reality for the tens of millions of consumers and healthcare professionals we serve around the world. Together, our team will achieve the full promise of whole-person virtual care, leveraging our combined applied analytics, expert guidance and connected technology to deliver, enable and empower better health outcomes."

There are a few critical phrases here. Let's look at them one by one.

"a new kind of healthcare experience" This is the disruption that I mentioned. Not just fine-tuning the current system, but a completely fresh start.

"one that empowers people everywhere to live their healthiest life".

This is the health assurance that I was talking about. Helping people to live their healthiest life is the reverse of what the current healthcare system thrives on. Empowerment means that people will be able to decide for themselves and take their health into their own hands. The 'everywhere' in this phrase refers to the global footprint Teladoc has.

"whole-person virtual care"

This refers to everything from health assurance to data, treatment plans, health nudges, and specialized diagnosis and surgery. Teladoc/Livongo will be the only one-stop-shop for taking your healthcare into your own hands. Healthcare is one of the few paternalistic sectors left. Paternalistic here means: "We know everything, you don't. Your only function is to give us the money and shut up."

Again, that's not throwing a stone to healthcare workers. I have a really, really deep respect for the people working in healthcare and this pandemic has highlighted even more how crucial they are. Several of my friends work in the sector.

They actually often feel the same frustration as patients. They don't have enough time to really listen, which would help them to diagnose more accurately, they have to do too much administration, they lose precious time gathering simple data and there are no efficient follow-up programs. Most healthcare workers would love to have the time to establish a human connection with each of their patients and listen to every detail that could count. But there is just no time. That's why a lot of people seek help from all kinds of coaches.

This is Teladoc's representation of whole-person care:

(Source)

Let's look into this in a bit more detail.

As you see from the graph, and I think this is really important, the category Wellness and Prevention is included in that care too. It is explained as 'Complete regular screenings and improve nutrition, exercise and well-being'. That means planning your health.

No company flies blindly and every company has a clearly-defined goal for the future often with step-by-step roadmaps. But for health, the advice is often: "Eat well and exercise." That's like saying to a company: "Execute well and make money." In other words, although it's true, it's too general.

A personal note here to illustrate what I mean. For years, I was very tired and I had trouble staying awake after meals or after drinking coffee. The advice I got from my doctors? Eat healthily, do regular exercise.

I found out 5 years ago that I have a milk allergy but I had to find out all about what it meant for my life (dairy-free cooking, avoiding almost all cookies, but also potato chips etc.) on my own. It would have been great to have a specialist that could have coached me there.

The example of my own life is just to illustrate the truth about healthcare that we all intuitively know but that is not acknowledged enough under this system: we are all individuals, with our differences, our unique needs. In stock terms, we are as different as a steel producer stock and a SaaS stock. I'm a man of 6 feet and 5 inches and almost always, I get the same dose of medicine as a woman of barely 5 feet high.

We are on the brink of other breakthroughs in healthcare: stem cell therapy, gene therapy, cheap genome sequencing, CRISPR, and many more. All these trends point in one direction: individualized healthcare. Medicine will not be a mass-produced, mass-prescribed drug anymore. We will evolve to personalized medicine.

Initially, people will be split into different groups based on certain data points (age, weight, condition...) and later it will be really about you, the individual. Your genome, your microbiome, your allergies, your reactions to certain drugs, everything will be known and taken into account for your prescription. Lots of medicines contain milk, for example, as a filling agent. Each time again, I have to say this to a doctor and sometimes there are even no medicines on the market without milk. These will be produced in the individualized healthcare that will come, if only for me. Medicines will be prepared on who you are, not on who the masses are.

But it will be much more than just medicines. Which supplements should you take? What is the perfect exercise regimen for your type of body? How could you build up your condition for that marathon or triathlon you always dreamed of without the risk of an injury because of your specific body composition? What are the best shoes for your feet so they can be 3D-printed? What is the best diet for the specific microbes that you have in your gut? What are the diseases you are genetically susceptible to and what can you change in your lifestyle to prevent them?

Don't get me wrong, I'm not a pie-in-the sky thinker. This will not be for the first years, of course not. And at first, it could be unevenly distributed, as a lot has been throughout healthcare's history. The first individualized programs could come with a hefty price ticket. But probably that will democratize, as a lot of procedures have in healthcare. Or maybe it will democratize from the start but with different degrees of quality, a bit like smartwatches.

The medical know-how of Teladoc, its wide network of doctors and health specialists, combined with Livongo's AI, data gathering and processing capabilities, make that this company is, like no other that I know, prepared for the future of healthcare.

I haven't seen any other AI healthcare platform that even comes close to that of Livongo at this point. With its AI+AI approach (aggregate, interpret + apply, iterate) it has already learned a lot, both from the whole pool of patients as from individual patients. With more patients because of the merger, there will be more data and more data means more insights and new products over time.

And Livongo will double down on its AI and data analysis. Revealera.com is a website that looks at jobs, job openings and it tries to find relevant information from these data.

It showed that of all publicly-traded companies, Livongo had the highest percentage of job openings that require data science and machine learning. 16% of Livongo's jobs openings ask for experience in those fields.

This clearly shows to me that Teladoc/Livongo is skating where the puck will be, not just where it is.

If you look at the combination of the two companies that merged, you can see that they are very complementary. These are Teladoc's key growth strategies and in blue, Livongo could help to accelerate Teladoc's strategy:

(Source)

The companies estimate that there will be $500M in synergies.

(Source)

Now, I know that it's all too common in an acquisition or a merger to overestimate these synergies by a wide margin. But in this case, I think the synergy opportunities are actually very conservative. The companies even acknowledge that in their merger presentation:

There will be a lot of cross-selling, as there is only a 25% overlap in the customers of the companies. Even before the merger officially was closed, Livongo was cross-sold in two deals already by Teladoc.

The first deal was Fresenius Medical Care, a company specialized in working with patients that suffer from CKD (chronic kidney disease). Partnerships and distribution are quintessential in healthcare and in its field, Fresenius is a big player. It provides dialysis for 347,000 kidney patients. The press release of Fresenius explains:

This marks the first time Livongo will use its robust virtual care solutions to specifically support those with CKD and is a significant step forward in Fresenius Medical Care's efforts to provide a more coordinated care experience. With earlier intervention, Fresenius Health Partners also seeks to increase optimal dialysis starts, as well as offer earlier evaluation of transplantation and home dialysis options.

This shows that the possibilities for Livongo to branch out are numerous.

The second deal was with Florida Blue, part of GuideWell Mutual Holding. Together with its merging partner Teladoc, Livongo will offer Florida Blue members with diabetes virtual care, including connected devices, advanced data science, and telehealth.

Being the only one-stop-shop for digital healthcare will provide Teladoc/Livongo with a competitive advantage that others simply don't have at the moment. The companies shared an example about Claire, an imaginary future client. You can see the different stages and situations in which she can be helped by Teladoc after the merger with Livongo:

As you can see, there is not a single moment in the whole process that Claire has to leave the platform. In this way, Teladoc and Livongo show that they are very complimentary. And the data component of Livongo, combined with preventive healthcare, gives Teladoc a lot of flexibility to introduce even more products, each one more and more targeted and eventually personalized.

There are risks to every investment, of course, although I generally believe that too many investors overemphasize risk. What is risk? Risk is not the same as volatility, no matter what some want you to believe. Volatility is risky if you are a short-term investor. If you need the money in 2 years, volatility is a risk. But if you need the money in, let's say, 20 years, why would it matter if a certain stock is up or down 50% this year or the next?

If you read the great book 100 baggers by Chris Mayer, you'll see that ALL (!) of the 100 baggers (stocks that turn your $10K into $1M) saw drops of 50% and more at least once. Most several times, and often they dropped substantially more than 50%.

Risk is the chance that you will lose your money permanently, not volatility. That also means that you should look at risks in their context. All companies make mistakes and if you sell because of a mistake, you'll never find multibaggers. Do I need to remind you of the Amazon Fire phone, the Netflix Quikster failure, the Windows phone, Google Plus and so many more mistakes? Don't let one failed product mislead you. The company as a whole is much more important.

Having said that, what will I keep my eyes on for Teladoc?

First, I want to see that Livongo really has an impact on Teladoc because health assurance is more a part of what Livongo does right now. There is a risk that Teladoc doesn't leverage Livongo's capacities enough.

The second element that I will watch is how the two companies work together when it comes to company culture. Teladoc has a good tracking record when it comes to acquisitions and giving them a place where they feel good inside of the company but this merger with Livongo is on a whole different level. I see some good signs because Teladoc CEO Jason Gorevic and Livongo's founder and executive chairman have already come out together several times and the two seem to share the same vision.

The third and final element of risk that I want to touch on is competition. At this moment, I don't see any competitor that is even close to Teladoc after the merger with Livongo but that can always change fast, of course. On the other hand, this market is so big that there will be several winners. And the size of the market, that's the next topic of this article.

The market cap of the combined company is around $30B at this moment. For Potential Multibagger stocks, I want to see the possibility that the stock could be a tenbagger in the next 10 years. For Teladoc, I think this is still possible, despite its already substantial market cap. The company has everything it needs to start a new era in healthcare as I showed, and it's in a gigantic market. This is the title of recent research:

Global Digital Health Market was Valued at USD 111.4 billion in 2019 and is Expected to Reach USD 510.4 billion by 2025, Observing a CAGR of 29.0% during 2020-2025

Teladoc operates in a TAM (total addressable market) of $510B in 2025 and at this moment it is the only 360 digital health company. That's a great position to be in. For those who wouldn't know: TAM is the yearly total addressable market. The fact that Teladoc projects a CAGR (compound annual growth rate) of 30% to 40% seems conservative to me. If you would add the synergies, it will be at least to the higher end of that margin, in my opinion.

If Teladoc could just bring in 3% of that TAM of 2025, that would already mean $15.3B. If you slap a P/S ratio of 20 on that, you already have a company with a market cap of more than $300B. A P/S of 20 might seem aggressive but for a company growing at more than 30% per year and gross margins which will probably be in the mid-70s, I think it's very reasonable. You can tinker with the numbers but the conclusion to me is always that if Teladoc executes well, it has the potential to become a giant.

I'm not saying that the company will already have 3% of the global digital health market by 2025, mind you. I think revenue of 1% of the TAM, about $5.1B, is possible at that moment, though, and much more growth will be in the pipeline.

There are always a lot of ifs but when I look at Teladoc, I can see the potential to become really big, ten times or more bigger than today.

With a lot of healthcare disruption knocking at the door, such as cheap genome sequencing, CRISPR, personalized medicines and much more, data will become more and more important for healthcare. Livongo's AI will add that to Teladoc.

The combination of Teladoc and Livongo definitely has the first-mover advantage in a very important and big emerging market because it can provide a 360 degrees one-stop-shop for personalized digital healthcare.

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Teladoc Is A Strong Buy: A Radical Healthcare Change Will Come - Seeking Alpha

When Jack Hogan was 2, his parents noticed that he would walk into walls at night. Twice he even fell down stairs, says his mother, Jeanette Hogan.

She would tell him to pick up his toys, "And he wouldn't pick them up. And I'd be like, 'It's right there!' And he wouldn't see it."

Jack was ultimately diagnosed with a form of vision impairment that comes from lacking a gene that's key to how the retina, at the back of the eye, senses light. It gave him night blindness and left him lacking peripheral vision.

Now, at age 13, he has undergone surgery to try to improve his vision by fixing his genes. The procedure is an important medical first, and not just for blindness, says his Massachusetts Eye and Ear surgeon, Dr. Jason Comander.

"This is the first time that an FDA-approved gene therapy was given to a patient for any inherited disease," he says.

The gene therapy, called Luxturna, had been tried on more than two dozen patients in clinical trials, Comander says, and it improved most patients' vision either a little or a lot.

But Jack's surgery, after the FDA's approval last December, marks Luxturna's entry into the medical mainstream including insurance coverage for its very hefty price tag: $425,000 for the eye that was operated on Tuesday, and just as much for the second eye, which is scheduled for next week.

Gene therapy works by packaging the needed genetic correction with a harmless virus that can deliver it into the patient's cells.

"The surgery had a very simple goal, which was to take three drops of liquid with this virus in it and put it underneath Jack's retina," Comander says.

The surgery itself was plenty complex, though it involved removing some gel and membranes in the eye. Then, Comander says, "through a tiny little cannula, the tip of which is the size of a human hair, we inject these three drops of liquid underneath the retina. And inside that drop of liquid are many billions of copies of this virus. And the job of the virus is, it's very good at injecting DNA into cells."

Jack, normally active and athletic, had to lie on his back for six hours after the surgery Tuesday. But by Wednesday morning, he was up to introducing himself to reporters: "Hi, my name is Jack Hogan, and I'm 13 years old, and I live in Fair Haven, New Jersey. I like to play sports, basketball well I can't play baseball..."

It's expected to take up to a month to see what effects the surgery has on his vision. Jack says he can't see a difference yet, but he has high hopes: "I hope it will increase my daylight so I could see at night for longer," he says. "And peripheral vision, so I could play basketball, I could see a whole court."

An estimated 1,000 to 2,000 Americans have the type of genetic blindness that Luxturna treats. Dr. Comander says he has several patients who are good candidates.

"It's every eye doctor's dream to take someone who has bad vision and give them the chance of getting better," he says.

But the significance of Jack's operation is far broader, Comander says. The advent of successful, FDA-approved gene therapy for patients like Jack has boosted gene therapy in general a field that crashed in 1999 after a young patient in a clinical trial died. It has been revived by the many researchers who devoted their work to getting it right, Comander says, and now it holds promise for treating many diseases.

"Different forms of inherited retinal disease are the ones that are near and dear to my heart and are also moving through clinical trials," he says. "On a broader note, people are working on hemophilia and other diseases as well."

The high costs of gene therapy treatments like Luxturna do remain a major concern. And for Jack Hogan, there's one other, much smaller downside: His mother says that better vision will mean that he should be able to do more household chores.

For a far more detailed description of Jack Hogan's treatment, STAT describes it at length here.

See the article here:
Medical Milestone In Boston: 13-Year-Old Gets New Gene ...

More than a century ago, in 1910, President William Howard Taft made what then seemed a bold but reasonable prediction: Within five years, he said, cancer will have been removed from the list of fatal maladies.

So, what happened?

Despite what seem like endless decades of hope, exhaustive research and unyielding effort by the worlds smartest scientists, we still have yet to find a cure or long-term treatments for cancer. But finally, we appear to be edging closer to the finish line, and immunotherapy might prove key.

For decades, the cancer treatment of choice has been chemotherapy. But, while chemotherapy can be incredibly effective at treating cancer, it takes a steep toll on human body. The side-effects of most chemotherapy treatments can be quite severe, and while the end result does often get rid of malignant cells, it also destroys plenty of healthy cells in the process.

Cancer is immensely complicated. Its not just one disease it can actually take over a hundred forms, and attack different parts of our body. Whats worse, what starts as one disease can mutate into something entirely different. Many tumors also contain more than one type of cancer cell.

Another challenge of treating cancer lies in the fact that there are great differences in patients physiologies, lifestyles, attitudes towards treatment, responses to treatment, genetic makeup and even epigenetic factors.

Cancer is as individual as the person who has it, explainsJoyce Ohm, PhD, at the Department of Cancer Genetics and Genomics at Roswell Park Cancer Institute in Buffalo, New York.

Lets say there are identical twin sisters, both with breast cancer. They may have been born with exactly the same genetic mutations, but one responds to therapy and one doesnt. One may live and one may die.

Its exceptionally difficult to find something that will work on everyone. Immunotherapy seeks to resolve this issue by personalizing treatments for each patient.

Immunotherapy isnt a new treatment by any means; scientists have been researching it for many years, and theyve invested a lot of time creating the right procedures and improving chances for all cancer patients.

The talk of immunotherapy started way back in 1890s, when William Coley, a physician, started researching how our immune system responds to viral infections. He hypothesized that scientists could jumpstart our natural immune response to cancer by provoking it with a controlled virus infection. But for years, little practical progress was made, and immunotherapy was viewed as having limited potential.

As crude as this method sounds, its basics eventually led scientists to explore how our own immune system responds to cancer and what can be done to target it without damaging other somatic cells. It also works on many types of cancers, even some that do not respond to chemotherapy or radiation.

Although immunotherapy is not yet as widely used assurgery,chemotherapy, orradiation therapy, immunotherapy drugs have been approved to treat many types of cancer. The one the doctor decides to use depends on the type of cancer they are tackling.

The main aim of immunotherapy today is to help activate dormant T-cells and help the immune system better recognize cancerous cells and get rid of them safely. T-cell transfer therapy basically attempts to re-engineer our immune response. Its a complicated process but so far it has shown great success. Other cancer immunotherapy treatments include immune checkpoint inhibitors that block certain chemicals in our body from stopping immune response to cancer cells; the use of cytokines, laboratory-made versions of a type of natural protein that boosts our immune response; lab-made monoclonal antibodies that bind to specific targets on cancer cells; and treatment vaccines.

The key in developing immunotherapies is finding the right cancer targets. Chow Kwan Ting, a researcher from City University of Hong Kong (CityU), who has won the famous Croucher Innovation Award in recognition of her scientific achievements, focuses on the role of plasmacytoid dendritic cells (pDCs) in cancer treatment.

These cells havent been researched thoroughly before, but theyre known to play an important part of immune systems response to viral infections. Dr. Chows studies have shown that pDCs play a role in other types of infections as well, and that they could potentially do more than simply fight off an infection: they might actually play a role in cancer immunity.

Other scientists are advancing a range of promising immunotherapies that researchers hope will lead to breakthrough cures. A team of researchers at the German Cancer Research Center and the Berlin Institute of Health are targeting the metabolic enzyme IL4I1 (Interleukin-4-Induced-1). The survival probability of patients with gliomas, a type of malignant brain tumor, decreased when the enzyme was present in higher concentrations in these tumors.

In 2004, Sophie Lucas, a researcher at the University of Louvain de Duve Institute, began studying the blocking of immune defenses in tumors in order to understand the functioning of cells that are said to be immunosuppressive (they block the bodys immune responses). The goal was to identify and remove them, thus stimulating antibodies to act against the tumor.Last August, Nature Communications published the results of the first tests carried out by Dr. Lucas and her team on a tool using what are called anti-GARP antibodies that prevent the bodys natural immune response from being blocked. It worked on mice; human studies are next.

Of course, more research is needed to turn immunotherapy research into potential cures, but the very fact that scientists keep learning new things about cancer treatment is encouraging. Scientists are looking into liver and breast cancer as they are more prevalent in Hong Kong than other types of cancer.

Immunotherapy can sometimes have similar side-effects as chemotherapy, such as nausea, vomiting and hair loss, but they are usually less severe. It can also be used in combination with radiation therapy or surgery.

Along with gene therapy and tumor DNA sequencing, immunotherapy is providing new options and helping us edge closer to promises made more than a century ago.

Claire Adams is a content creator and external associate of the City University of Hong Kong. Her goal is to promote CityU young scholars research papers that are closely related to the healthcare industry. She wishes to emphasise the importance of the research paper on rare cells and the innovative immunotherapeutic strategy, which truly brings hope to new cancer immunotherapy and vaccine. By promoting this work among the scientific and healthcare community, Claire is hoping to raise awareness of the City University of Hong Kongs contribution to society. Follow her on Twitter@adamsnclaire

See the original post here:
How immunotherapy is revolutionizing cancer care - Genetic Literacy Project

By Maggie Ireland, for The Gazette

October as Breast Cancer Awareness Month shines a spotlight on the battle against breast cancer and the developments in diagnosing and treating the disease that inspire hope for the future.

Breast cancer remains very common. Its still the most common cancer in women, said Dr. Ingrid Lizarraga, a clinical associate professor of surgery and breast surgeon at the University of Iowa Hospitals and Clinics in Iowa City.

But it is no longer the most common cause of death for women it is now the second most common cause of cancer death for women after lung cancer. We know a lot

more about breast cancer than we ever did before. The vast majority of women who have breast cancer will do great.

Admittedly, a breast cancer prognosis largely depends on the type of breast cancer you have and the stage at which it is diagnosed. The good news is that the prognosis has improved considerably over the years.

Mortality rates are decreasing, thanks to improved screenings and improved treatment, said Dr. Rasa Buntinas, an oncologist at PCI in Cedar Rapids. At our cancer center, 80 percent of women are diagnosed at an early stage stage 0 or stage 1 and that means a better chance of survival.

More screening options have become available in recent years.

Mammograms are still what we recommend, Lizarraga said. 3-D mammograms have been around for about five years and can be really helpful for women with dense

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breasts. The recognition that breast density affects how you screen is important. Whole breast ultrasounds can also be useful.

For women with abnormal findings, a breast MRI is an option.

Discussing your personal risk factors with your doctor is the best way to determine what screenings are best for you.

The latest developments in genetic testing can further pinpoint specific risk factors.

Weve identified a set of genes that puts you at high risk for developing breast cancer, and we can test for this gene, Lizarraga said. The trick is to find those at risk who need the genetic testing. If you have concerns, talk to your doctor so they can assess your risk and give you better guidance on a screening regimen.

Today, the widely accepted recommendation is for most women to begin getting an annual mammogram at age 40.

If you have any risk factors at all, start at 40. But the latest you should start is at age 45. And you should screen every year, Lizarraga said. When youre younger, the odds are lower, but the stakes are higher.

In the past, all women were encouraged to do a monthly self-exam, but thats no longer the case.

Self-exams are no longer recommended, Buntinas said. They tended to lead to more testing and biopsies but really didnt improve outcomes. The recommendation now

is breast self-awareness. Be aware of how your breasts look and feel, and report changes to your doctor.

Lizarraga agrees that a woman should trust her instincts if she senses something is wrong.

You know your body better than anyone else, she said. It doesnt just have to be a lump it could be a different appearance of the skin or the nipple. Pay attention to how you feel and how you look, and advocate for yourself if you notice something.

Many developments have been made in treating breast cancer, particularly metastatic breast cancer, Buntinas said.

Were really getting a much better understanding of tumor biology, she said. Specific targets are being identified to better improve treatments and provide more of a personalized medicine approach. In practice, these treatments are really improving survival.

Its an exciting time in the treatment of breast cancer, Lizarraga said, because things pretty much change every day.

If a woman needs a mastectomy, we can give you a result that looks much closer to what your native breast looks like, she said. The attitude used to be that you shouldnt worry about how it looks, because you should be grateful you dont have cancer, but thats not accepted anymore.

Advancements also are being made in addressing the side effects of treatment, like lymphedema, the swelling of a womans arm or hand that can follow the removal of lymph nodes.

Weve gotten better at figuring out more effective techniques that result in less lymphedema, Lizarraga said. Were also now better able to save a womans breasts if she prefers to do that.

The biggest advances in treatment, however, have been in systemic therapies, or the use of drugs to reach cancer cells anywhere in the body, she said.

Almost all women with breast cancer will get some form of systemic treatment pills or therapy.

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Weve become increasingly targeted with treatment, Lizarraga said. We start by figuring out which type of breast cancer you have. We now have genomic tests, so we can test the DNA of the tumor itself to see how likely it is to recur. That actually allows us to figure out whether a woman will benefit from chemotherapy or not.

Thats huge because we can limit the toxic treatment when its not going to be beneficial.

Treatment is more targeted than ever.

We match severity of treatment to the severity of disease, Lizarraga said. Weve figured out how to make things more personal in a lot of cases, weve changed the timing of treatment. In the past, you often came in, had surgery and then began the next form of treatment, like chemotherapy or pills.

Now, we often provide treatment before surgery and then personalize what is needed next.

Lizarraga said women of all ages need to know breast cancer is common but treatable, as long as its caught early.

If you do have a family history, be sure to get evaluated to see if genetic testing is appropriate, she said.

And know that who treats you is important, too.

Get a second opinion make sure the person youre seeing is someone who knows specifically about breast cancer treatment and the latest developments. Advocate for

yourself.

Both doctors agree the prognosis for breast cancer patients is brighter than ever.

The outlook is very positive and hopeful for the future, Buntinas said.

See the article here:
The good news about breast cancer - The Gazette

HARRIS COUNTY, Ga. (WRBL) A Harris County family is celebrating overcoming a major obstacle in treating their 8-year-old-son who has a rare form of Muscular Dystrophy, thanks to a village of supporters and a phone call from the Chief Medical Officer of a major insurance company.

8-year-old Jacob Saalman is like most kids. He loves being outdoors, riding four-wheelers, and playing baseball. This Halloween, he carved pumpkins with his family. Jacob loves his dad, a former Fort Benning Ranger, and his mom. Jacob adores his four-year-old brother, Hudson.

Jacob is very protective of Hudson. Hes a mother hen. Oftentimes I have to tell Jacob to stop parenting, shared Ryan and Brooke Saalman, Jacobs parents.

However, Jacob is not like most kids when it comes to his health. Jacobs handled more medical procedures, biopsies, and tests than many adults. Its heartbreaking. Jacobs mom and dad say hes resilient with a quiet strength.

I would say he is very strong and adaptable. All the things he has to do, all the medical stuff he has been through, pokes and prods and port placement and muscle biopsies. He has been through a lot, said Brooke.

Jacob is very sick. So is his brother Hudson. Both boys have Duchenne Muscular Dystrophy, a rare genetic disease, preventing their bodies from making a protein called Dystrophin that protects our muscles from breaking down with use.

Jacob plays ball now. By 12, he will most likely need a wheelchair. After that, the prognosis remains bleak for the brothers and the parents who love them.

The life expectancy is upper 20s and into the 30s. But you are not walking around or living a normal life. You are most likely on a ventilator and having a parent care of you 24/7, shared Ryan.

Jacobs medical team is convinced an FDA approved home infusion called Exondys 51 will significantly improve Jacobs quality of life by helping him produce partial Dystrophin proteins.

Its not a cure. It slows the rate of the disease, and also its been proven to help kids walk longer, and it is also shown to improve pulmonary function, and thats the issue later on down that causes a lot of problems and even death from this disease, said Brooke.

The Saalmans goal is to stave off the disease for as long as possible. They believe Exondys 51 can help keep Jacob as healthy as possible until a gene therapy is ready.

We still have hope. What gives us the most hope is there are gene therapy trials in phase three. So we are hoping within the next two or three years we will be able to have a gene therapy that will make this a much milder disease, said Brooke.

One of Jacobs physicians, Han C. Phan, MD, with Rare Disease Research LLC in Atlanta, Georgia, shared this statement with News 3:

Jacob has Duchenne Muscular Dystrophy, a slowly progressive condition for which without treatment could limit his life expectancy. Every child with DMD should receive the treatment he deserves and without such could be detrimental to his overall health.

The family says Horizon Blue Cross Blue Shield of New Jersey had twice denied Jacob Exondys 51, despite other major insurance companies approving similar patients treatment. The drug costs 300,000 a year. The company listed several reasons for denying coverage for the medicine in a letter sent to the Saalman family.

Ryan Saalman shared the familes heartbreak and frustration on his Facebook page earlier in the week. The status update was shared several thousand times and sent to News 3.

Monday, the Sallman family spoke with News 3 about their struggle since April to get the medication approved with their insurance company.

Its just frustrating to know there is a child that has this need, and we pay good money for our insurance. You think that if a doctor says this is medically necessary that they will get the medication, and thats not the case. These kids deserve that. Everything they go through. They deserve some help, said Brooke.

News 3 reached out to Horizon Blue Cross Blue Shield of New Jersey on Monday. Tuesday, News 3 worked with The Saalman family and Horizon BCBS of New Jersey to get the necessary medical privacy forms filled out to get information on the insurance companys case. However, before the process was complete, The Saalman family says they got a phone call from the insurance companys Chief Medical Officer. The family was thrilled to learn Exondys 51 has been approved for Jacob.

Today, I think we truly saw the power of community. In this fight against Duchenne Muscular Dystrophy, it takes a village, and I think our village got really big really fast. Our new village gave an 8-year-old boy a loud enough voice that Horizon BCBS has overturned last weeks denial. Jacob will be able to get the medication he needs. The Saalman Family wants to thank the thousands of people that shared our story; we truly believe it is because of all of you that this has happened, said Ryan.

The Saalman family is overjoyed. Time is not on their side as this disease doesnt slow down. With each delay comes another day Duchenne Muscular Dystrophy robs Jacobs muscles of their ability to function. The family hopes the newly approved infusion will be ready for Jacob within a few weeks. The treatment should help slow the diseases progression down and give the family the time they need to wait for a promising gene therapy they pray is on the horizon.

The day before The Saalman family learned the medication had been approved, Jacob hit the winning RBI for the final game of his baseball season in a tie game with two outs. Jacob got the game ball in the final game of what may be his last season of baseball.

And sometimes God shows up in the last game, in the last inning, with two outs and two strikes on the board, when the hope of a win is almost gone and He crushes our expectations. Man, did I ever need that reminder tonight, and maybe you do too, shared Jacobs father on Facebook.

For more information on Jacob and Hudsons battle with Duchenne MD and ways you can help, you can visit their blog: Saalman Strong. The family has been encouraged to establish a GoFundMe page: Jacob and Hudsons fight against Duchenne MD

News 3 will keep you updated on the Saalman family and the journey with their sons Jacob and Hudson.

See more here:
Harris County parents win insurance appeal for 8-year-olds MD treatment - WRBL

In 2009, studies on telomeres and telomerase (its corresponding enzyme) won three American researchers the Nobel Prize in Physiology or Medicine. Since then there has been a flurry of work surrounding this seemingly redundant protein, that has now been touted as the secret to biohacking the ageing process.

We sit down with Functional Medicine Specialist, Miles Price, to figure out why this little protein is so important to our longevity and our health.

Telomeres are proteins called nucleotides which attach to the ends of DNA strandsto protect them from damage every time the cells divide. They play a central role in the cell fate and ageing. Each time the cell divides, some of the telomere sequence gets lost so the telomere cap shrinks. Eventually, when the telomere becomes too short, the cell will either self-destruct through apoptosis or go into senescence. Senescence is an arrested cell state meaning the cell is no longer functioning at100 percent. This contributes to ageing and puts the body at greater risk of developing abnormal cells like cancer.

Getting older is a fact of life. However, the rate of how fast we age is controlled, amongst other things, by our telomere length. These bits of proteins protect our DNA from damage every time the cells divide. Telomere length generally shortens with age however we can slow down the shortening largely with lifestyle interventions. Things which accelerate the shortening include smoking, pollution exposure, lack of physical activity, obesity, stress and inflammation. Skin ageing is also affected by shortened telomeres, however the efficacy for maintaining skin health with telomerase interventions is still being investigated.

Telomere length is greatly influenced by our environment and lifestyle. Through epigenetic influences we can help protect our telomeres by doing the following:

1. Caloric restriction time-restricted eating or fasting up to a minimum of three days(with supervision).2. Evidence suggests that dietary alterations can affect our telomeres. An example ofthis is restricting protein intake.3. Take regular exercise, preferably high intensity intermittent training.4. Increase antioxidants in the diet, typically from plant foods.

A recent gene therapy study on mice consisted of inducing telomerase, the enzyme which slows down the biological clock by extending telomere length, was successful in increasing the lifespan by a further 24 percent. Whilst human trials are still a ways off, what we can do is consider some supplemental interventions together with lifestyle measures discussed earlier. NAD+ (Nicotinamide adenine dinucleotide) is a supplement which is a derivative of Niacin (vitamin B3), and it directly influences the lifespan of the cells and the way the cell ages. This is done by interacting with proteins inside the cell called sirtuins. Sirtuins stimulate various enzymatic processes inside the mitochondria and nucleus which help to protect the DNA and RNA from damage as the cell divides. As we age, our NAD+ levels decline and thus the enzymatic activity of sirtuins declines also, therefore it is becoming increasingly important to consider supplementing with NAD+ as we age.

As you age your immune system becomes weaker. How this affects you is dependent on many factors, like your diet, exercise levels, sleep patterns, and stress levels, for example. Typically our immune systems become slower to respond, the body heals more slowly from infections, and the ability of the immune system to detect and correct dysfunctional cells also declines. Key immune system organs play a role in this decline. The thymus gland located in the middle of the chest plays a central rolein activating certain white blood cells to identify invaders or rogue cells. It shrinks with age due to free radical damage.

This can be minimised by taking extra zinc and other antioxidants which protect against its shrinkage. Other influences on your immunity include caloric restriction. Research has demonstrated that if you fast for more than three days your immune system is given a real boost, by stimulating autophagy. This is the process whereby the body seeks out and removes old and senescent cells, thereby reversing ageing in that short period of time. Caloric restriction for longer than 24 hours is best supervised by a doctor or functional medicine specialist who can monitor any adverse reactions you may have.

You can test your telomere length by conducting a blood test which isolates our DNA and measures telomere length using the qPCR method. This is the most popular method of assessment. One such test is the ImmniGen Test. ImmuniGen is an at-home telomere test that assesses your immune strength and resilience. By measuring your telomere length (a biomarker of ageing), it can measure how yourimmune system is ageing, known as immunosenescence, as well as key genes that affect ageing and immune health. As telomere length is heavily influenced by our lifestyle, it is recommended to repeat the test at regular intervals to track your ageing progress.

Visit LifeHub to learn more about biohacking your telomeres for a healthier and longer life. LifeHub is currently offering ImmuniGen Testing at HK$1,280, which can be ordered online and delivered straight to your door.

LifeHub is a Modern Wellness Lab in Hong Kong offering the antidote to your busy city lifestyle. As leaders in detox and anti-ageing, we cleanse you of everyday toxins and recharge your body for better performance in work and play.

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Biohack the ageing process by taking care of this little protein in your body - Lifestyle Asia

Yulonda Wilbur remembers precisely the sting of when she was told she had breast cancer.

The news came after a routine physical where her general practitioner did a manual exam, and Wilbur flinched when the doctor touched a tender spot on her left breast. She was already scheduled for her annual mammogram, and her physician added an ultrasound. Wilbur remembers one of the technicians when she had the tests being initially cheerful, and then solemn, and later, the radiologist telling her he didnt like what he was seeing and shed need to undergo a biopsy.

But still, when the call came on Feb. 16, 2019, with news that she had triple-negative breast cancer a cancer that tests negative for estrogen receptors, progesterone receptors and excess HER2 protein and therefore does not respond to normal hormonal therapy medicines or those that target HER2 protein receptors her world metaphorically fell apart.

It was just that moment that changes your life forever, said Wilbur, now 53 and back at work since early this year as a patient care assistant at Lawrence + Memorial Hospital, where she has been employed for two decades.

That journey from her initial physical in December 2018 would include a lumpectomy, followed by chemotherapy, then an additional diagnosis of DCIS, or cancer in her milk ducts, and finally a mastectomy in October 2019. This year has been rough with COVID, but for Wilbur, a mother of four grown children, 2019 was the year she battled breast cancer and shes grateful she didnt wage that war during a pandemic.

While shes worked at L+M for 20 years, shes been a patient care assistant for more than three decades and said working in the medical field doesnt make a breast cancer diagnosis any easier.

What shed learn along the way is that theres a higher prevalence of triple-negative breast cancer in African-American women and that friends and family are essential to get you through.

My advice is dont go it alone. Ask for help. You need support, said Wilbur, who lives in Waterford. And ask questions if you dont understand.

After the diagnosis, when one physician came into the room where Wilbur was waiting and started talking at her, Wilbur reminded her that she hadnt introduced herself and asked her to slow down and explain things more thoroughly.

You need to understand what you are going through, and it is a lot to take in, she said.

The lumpectomy was painful, the chemotherapy made her nauseous and left a distasteful film on her tongue, and she developed neuropathy, anemia and fatigue.

I heard a sermon once, said Wilbur, who is active in her church. And what they said was, When you say why me, what you are really saying is why not someone else? So I just accepted it for the most part, and I wasnt happy about it, but I accepted it.

Shes an advocate of regular checkups and mammograms, of all preventive medical care, and marvels that someone who comes from strong stock, like she does, ended up so ill.

Im one of those people who dont get colds, who dont get sick, but I did, she said. There was no history of breast cancer in her family, and shed test negative for the BRCA gene.

When Wilbur was invited to attend a breast cancer support group at Smilow Cancer Hospital Care Center in Waterford, she initially resisted.

I didnt want to sit around with people who were angry, or be depressed, she said. But it wasnt that way, everybody takes a different path, everyone has their own journey.

Not so long ago, when Wilbur found a lump on her right breast, she panicked, but the diagnosis was a benign cyst.

Its not that I cant, but I dont want to go through it again, she said. Later, I thought maybe I should have had both breasts removed.

Shes back at work at L+M and doing the things she enjoys walking, reading and keeping active in her church.

I have faith, and I prayed a lot when I was going through it," she said. "I asked Jesus to get me through it, and He did."

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'I have faith and I prayed a lot' - News from southeastern Connecticut - theday.com

Postmenopausal women and men with advanced-stage hormone-receptor-positive, HER2-negative breast cancer with a PIK3CA mutation that grew during or after hormonal therapy lived about 8 months longer when treated with the combination of Piqray (chemical name: alpelisib) and Faslodex (chemical name: fulvestrant) compared to Faslodex alone.

These latest results from the SOLAR-1 study were presented on Sept. 19, 2020, at the European Society for Medical Oncology Virtual Congress 2020. Read the abstract of Overall survival (os) results from SOLAR-1, a phase III study of alpelisib (ALP) + fulvestrant (FUL) for hormone receptor-positive (HR+), human epidermal growth factor receptor 2-negative (HER2) advanced breast cancer (ABC).

Advanced-stage breast cancer is breast cancer that has spread to tissue near the breast (locally advanced) or to parts of the body away from the breast, such as the bones or liver (metastatic).

Overall survival is how long a person lives, whether or not the cancer grows.

Piqray is a targeted therapy medicine that inhibits the PI3K pathway. The PI3K pathway helps all cells both healthy and cancer cells get the energy they need. When this pathway is overactivated because of a mutation in the PIK3CA gene, it can allow cancer cells to survive and grow. PI3K inhibitors block this pathway, with the goal of killing cancer cells.

In hormone-receptor-positive breast cancers, a PIK3CA mutation also can make the cancer resistant to hormonal therapy medicines.

About 40% of people diagnosed with hormone-receptor-positive, HER2-negative breast cancer have a PIK3CA mutation.

Piqray is a pill taken by mouth.

Faslodex is a hormonal therapy medicine called an estrogen receptor downregulator. Faslodex sits in the estrogen receptor in breast cells so the cell cant receive estrogens signals to grow and multiply. Faslodex also reduces the number of estrogen receptors and changes the shape of breast cell estrogen receptors so they dont work as well. Faslodex is a liquid that is given once a month as an injection into a muscle.

The SOLAR-1 trial included 572 people diagnosed with advanced-stage hormone-receptor-positive, HER2-negative breast cancer that had previously been treated with hormonal therapy; 341 of the cancers had a PIK3CA mutation. The people in the study were either postmenopausal women or men.

The people were randomly split into two treatment groups:

Median follow-up time was 30.8 months. This means that researchers tracked half the people for a longer time and half the people for a shorter time.

For the 341 people diagnosed with cancer with a PIK3CA mutation, overall survival was:

This difference of about 8 months was not statistically significant, which means that it could have been due to chance and not because of the difference in treatment.

Still, the researchers who did the study said the difference in overall survival was clinically meaningful and supports using Piqray to treat advanced-stage hormone-receptor-positive, HER2-negative breast cancer with a PIK3CA mutation that has grown after hormonal therapy.

Like almost all cancer medicines, Piqray can cause side effects, some of them severe.

The most common side effects of Piqray are:

Piqray also may cause serious side effects, including:

If youre a postmenopausal woman or a man who has been diagnosed with advanced-stage hormone-receptor-positive, HER2-negative breast cancer with a PIK3CA mutation that has grown after treatment with hormonal therapy, the latest results from the SOLAR-1 study are very encouraging.

Earlier results from the SOLAR-1 trial showed that Piqray and Faslodex led to longer progression-free survival compared to Faslodex alone. Progression-free survival is how long a person lives without the cancer growing.

These latest results showing 8 months longer overall survival for people treated with Piqray and Faslodex, while not statistically significant, offer more support for using Piqray to treat this type of breast cancer.

If youre being treated for advanced-stage hormone-receptor-positive, HER2-negative breast cancer with a PIK3CA mutation that has stopped responding to the first hormonal therapy, you and your doctor are likely considering several treatment options, including Piqray. Together, you will weigh the benefits and risks of each treatment and decide on the best treatment plan for your unique situation.

Written by: Jamie DePolo, senior editor

Published on October 16, 2020 at 11:36 AM

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Piqray Improves Overall Survival for Advanced-Stage Hormone-Receptor-Positive, HER2-Negative Breast Cancer With PIK3CA Mutation - Breastcancer.org

The European Parliament voted by a large majority to set up three special committees and a committee of inquiry for 12 months, along with a permanent subcommittee. Of those three special committees, one is the Special Committee on Beating Cancer, which is tasked with looking into different ways and opportunities for the EU to take specificaction, revise legislation and other compelling measures in order to prevent or to fight cancer.

Across the European Union, cancer is the second leading cause of death after cardiovascular disease, claiming the lives of 1.4 million Europeans each year and causing the loss of 2.3 million potential working years due to premature deaths as well as a 70bn loss in productivity.

The burden of cancer brings unimaginable suffering for patients, survivors and caregivers alike, all while stretching available healthcare resources. Cancer is an age-old disease which we are only now beginning to understand, thanks to major breakthroughs over recent decades.

We know that cancer is a complex genetic disease with more than 100 cancer types, involving nearly 1,000 known cancer-related genes, all of which points towards a small probability of ever finding a silver bullet that will treat all cancers. Our best option to beat cancer is to have a comprehensive approach, emphasising the importance of prevention and early detection, while supporting the development of novel therapies and improving follow-up care.

Our best option to beat cancer is to have a comprehensive approach, emphasising the importance of prevention and early detection, while supporting the development of novel therapies and improving follow-up care

Although this plan seems straightforward, the truth is that things are much more complicated. The 33 sitting members of the Parliaments Special Committee on Cancer will act as a bridge between stakeholders, from patients and doctors to industry and research institutes, making sure that all needs are heard and proper funding is sought. It is the Committees job, in partnership with the European Commission, to find all the legal means that will allow the Plan to Beat Cancer to take shape, setting up the political agenda and mapping its main objectives.

Fighting cancer is not an easy endeavour, nor is it cheap. Since 1971, when the US government declared war on cancer, more than $200bn has been spent by Washington alone on research and development of new medical treatments and gaining valuable knowledge on the nature of this disease.

We must grasp from the very beginning the magnitude of overcoming the colossal challenge that lies ahead. One major problem we are dealing with is the huge disparity between EU Member States in terms of cancer fighting programmes. Statistics vary widely from country to country, with more cancer survivors living in the west than in the east.

This is due in part to extensive national screening programmes, better access to specialised treatment and better health coverage. We have to ensure that European citizens enjoy the same level of health care regardless of their nationality. As any doctor will tell you, why treat when you can prevent? Between 30-50 percent of all cancers are preventable.

Prevention must be the cornerstone of our policies. We must set up the EU agenda on promoting a healthier lifestyle, emphasising the need to have a healthy diet, curbing smoking and alcohol consumption, all paired with daily exercise.

Environmental pollution and occupational carcinogens are other substantial risk factors that need to be addressed. When it comes to tumours, early diagnosis is key to improving patient outcomes. We need EU-wide screening programmes for the common types of cancer, improving coverage rates and making sure that patients have easy access to screening centres in all Member States.

We should incentivise research and development for better diagnosis equipment and integrate frontrunner technologies such as liquid biopsies and Artificial Intelligence in the process. Through developing a better understanding of how cancer works, we now have access to more efficient therapies, like personalised medicine or gene therapy.

The 33 sitting members of the Parliaments Special Committee on Cancer will act as a bridge between stakeholders, from patients and doctors to industry and research institutes, making sure that all needs are heard and proper funding is sought

But there is also a growing concern with regards to the affordability of certain treatments. Therefore reducing the cost and access of treatment for cancer patients and ensuring the sustainability of our health systems must be our top priority.

Rare and very rare types of cancer - about 20 percent of all cases - most of them being paediatric cancer, are largely overlooked by industry and research institutes. Encouraging R&D of novel treatment for rare and very rare cancers can be achieved through alternative financing mechanisms, such as milestone prizes.

By the end of our lifetimes, each one of us will have a one-in-two chance of developing a form of cancer and a one-in-five chance of dying from cancer; this is not a warning, but a reality check that most of us will have to face unless we start acting decisively.

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Beating Cancer: Prevention must be 'cornerstone' of EU's new approach - The Parliament Magazine

Dr Humaid Al Shamsi delivers the new injection to patient Joanne Resta Image Credit: Supplied

Abu Dhabi: A brand new subcutaneous injection is set to revolutionise treatment for patients battling breast cancer.

The medicine a combination of the prescription drugs pertuzumab and trastuzumab can be delivered in a simple five-minute injection, instead of the three to five hours needed to deliver the drugs in the form of an infusion.

First time outside US

Called Phesgo, it has been licensed in the UAE and was offered on a trial basis to a patient last week at the Burjeel Cancer Institute. This represented the first time the injection was delivered outside the United States, Dr Humaid Al Shamsi, director of oncology at Burjeels parent company, VPS Healthcare, told Gulf News. The new drugs delivery coincides with Breast Cancer Awareness Month, which is marked every October.

Dr Al Shamsi, who is also president of the Emirates Oncology Society, said the quick and easy form of drug delivery will help improve patients quality of life, in addition to reducing the costs of treatment. This form of treatment should be the standard of care for cancer in the near future, he said.

Phesgo is also the only medicine of its kind available at present, which is why its availability in the UAE will be a game-changer, the doctor added.

Breast cancer type

The medicine can be used for patients who have HER2 positive breast cancer, which affects nearly 25 per cent of breast cancer patients. HER2 itself is a gene that produces HER2 proteins, or receptors. When the gene isnt working properly, it reproduces too many copies, which leads to overexpression of the HER2 protein. This causes uncontrolled breast cell division and the formation of tumors representative of HER2-positive breast cancer.

Dr Al Shamsi said Phesgo can be given in the early stages, as well as for a while in the advanced stages.

Major benefits

Typically, patients receive this combination of drugs for a year, along with chemotherapy for three to four months. Patients will still have to come in for the chemotherapy, but they can be trained to take the Phesgo injections at home. Even if they come in to the hospital to receive the injections, the shortened treatment time frees up cancer chairs, and reduces insurance costs, he explained.

In addition, patients with advanced HER2 positive breast cancer also need to take the medicine for life, so this medicine makes treatment delivery much more convenient.

Insurance approval

Dr Al Shamsi said the prescription medicine is pending insurance approval, but as soon as this is received, at least 50 of his patients would qualify to receive Phesgo. He also explained that the injection has no additional side effects, save some minor discomfort at the inject site, compared to when the injections are given as infusions.

So convenient

Joanne Resta, 42, a homemaker from the Philippines, was the first patient in the UAE to receive Phesgo, and she said it would greatly ease her treatment. I was diagnosed with breast cancer in January 2020, and have already finished the chemotherapy prescribed. I have to now do this pertuzumab-trastuzumab targeted therapy for a year, once every three weeks. Every time I have to come in to the hospital for an infusion, it takes me five to six hours, and I also have to find a sitter with whom I can leave my two children, aged four and five. So this new injection sounds wonderful to me. It is so covenient, Resta said.

Breast cancer is the most commonly detected cancer among women in the UAE, with more than a 1,000 new cases reported every year. It is also the cancer with the second highest rate of mortality in the UAE, after colorectal cancer.

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Watch: New 5-minute injection a game-changer in breast cancer therapy in UAE - Gulf News

As a nurse, Joellyn Koscik knows how important regular health screenings are.

So when the Homer Glen wife and mother turned 40 last year, she followed theAmerican Cancer Societys guidelinesfor breast cancer screening and had her first mammogram.

Never in a million years did she think it would result in a cancer diagnosis.

The Unexpected Diagnosis that Changed Her Life

Kosciks story began in the spring of 2019, when radiologists flagged a possible area of concern in her right breast. Following a diagnosticmammogram,ultrasound and biopsy, her doctor delivered the news:triple negative invasive ductal carcinoma, stage 3. After completing a successful 4-month clinical trial treatment at UChicago Medicine in Hyde Park, Koscik underwent a double mastectomy with reconstruction in June 2020.

Second Clinical Trial a Precautionary Measure

Although the first round of treatment and mastectomy was successful, Kosciks treatment wasnt over. She was referred toDr. Anne McCall, M.D.,Radiation Oncologist at theUChicago Medicine Comprehensive Cancer Center at Silver Cross Hospitalin New Lenox, who immediately thought Koscik would be a great candidate for a radiation therapy study she was doing.

Joellyn was a perfect candidate for this trial because she had such a great response to her chemotherapy and a very successful reconstruction, said Dr. McCall.

In July of this year, Koscik started her second clinical trial, this one much closer to home atSilver Cross. The trial involved the use of hypofractionated radiation therapy after mastectomy and reconstruction.

Hypofractionationis a form of external beam radiation therapy that emits high-energy X-ray beams carefully aimed at the breast. Conventional radiationis delivered through the same machine, but with hypofractionation, tumors receive a higher dose of radiationper treatment session.

The point of the trial is to see the safety and effectiveness of a higher radiation dose over fewer sessions, which I received, versus a lower dose of radiation at more frequent sessions, which is the standard for radiation therapy, Koscik explains. The hope is that the radiation therapy after mastectomy will reduce my risk of the cancer returning and improve my outcomes.

Care Close to Home Made Treatment Much Easier

Over the course of three weeks in July and August, Koscik received 16 radiation therapy treatments with Dr. McCall, instead of the usual 25. The Monday-Friday sessions lasted only 30 minutes, so living just 15 minutes from theUChicago Medicine Comprehensive Cancer Center at Silver Crossmade getting to and from her treatments a breeze. Fortunately for Koscik, the side-effects were minor; fatigue and redness at the radiation site meant she could continue to work full time as a nurse, her role for the last 20 years.

During her first clinical trial, Koscik took a blood test that uses DNA analysis to identify harmful changes, or mutations, in either one of the two breast cancer susceptibility genes, BRCA1 and BRCA2. With the results of this test, called aBRCA test, Koscik discovered that even without a family history of breast cancer, she is BRCA1-positive (a genetic mutation of the BRCA1 gene), increasing her likelihood of having breast, ovarian and other reproductive organ cancers.

That news solidified her decision to undergo the mastectomy.

Since the trial only recently ended, I have an upcoming appointment with Dr. McCall to see how I did. I do plan on discussing with her the possibility of removing my ovaries and fallopian tubes as a further precautionary measure, says Koscik.

A Supportive Tribe Behind Her

Koscik lights up when talking about the people who never left her side and kept her spirits high through the toughest of days. My husband, Scott and our 11-year old son Jason have been with me through all of this, she beamed. Also, my family, my in-laws, my girlfriends, neighbors and co-workers, they never let me get down on myself and continue to encourage me on this journey.

Koscik also has many compliments on the care she received atSilver Cross Hospital, I would recommend Silver Cross to anyone. Everyone who cared for me, from the doctors and nurses, the radiation team and thelymphedema therapists, they were all so kind and treated me with love and care. During this COVID-19 pandemic, I am just so grateful to have been able to have my treatments, and Im beyond thankful for the great results Ive received so far.

To learn more about the cancer care offered at Silver Cross Hospital, visitwww.silvercross.org/cancer

Silver Cross Hospital

1900 Silver Cross Blvd

New Lenox, IL

60451

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Homer Glen Mom and Nurse Breast Cancer-Free after Radiation Therapy Clinical Trial at UChicago Medicine Comprehensive Cancer Center at Silver Cross -...

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Pittsburgh, PA (KDKA) A West Virginia teen is among the first in the country to get gene therapy treatment for a rare condition.

Kendra Goins has Hurler Syndrome, meaning her body cannot break down certain types of sugar. In addition to damaging her organs, the condition also makes it difficult for her to grow.

Ive spoken with experts all across the country, all the way from the chemist that made this down to the doctor who has used it. This looks like her golden ticket, said Sheryl Goins, Kendras mother.

The goal of the treatment is to force Kendras body to produce the enzymes to break down sugars.

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Teenager among first to receive gene therapy treatment for rare condition - KRDO

A Yes vote authorizes the state to sell $5.5 billion in general obligation bonds primarily for stem cell research and the development of new medical treatments in California. A No vote would mean the state's stem cell research agency will probably shut down by 2023.

In the ramp-up to the 2004 election, a California TV viewer may have come across the popular actor Michael J. Fox urging her to vote Yes on a state proposition. His voice slurred faintly by Parkinsons disease, he still sounded wry, boyish and familiar.

My most important role lately is as an advocate for patients and for finding new cures for diseases, said Fox, eyes level with the camera. Californias Stem Cell Research Initiative 71 will support research to find cures for diseases that affect millions of people, including cancer, diabetes, Alzheimer's and Parkinson's.

Within that 30-second spot, Fox, diagnosed at age 29 with a neurodegenerative disorder that typically does not strike until after 60, used the word "cures" three times.

Proposition 71, which passed with 59% of the vote, authorized the sale of $3 billion in bonds to create an agency that funded stem cell research. The successful campaign grew out of a time, in the early 2000s, when the promise of stem cell and regenerative medicine excited both scientists and the public.

Whether the project has lived up to that promise is a matter of opinion. How voters view the record of the agency may go a long way in their decision whether or not to replenish the fund, which is fast running out of money, with an additional $5.5 billion to be raised with new bonds authorized by Proposition 14, now on the ballot.

President Bush A Demon to Attack

Scientists since the1800s have known about stem cells, which are not yet dedicated to any particular anatomical function and have the potential to become nerve cells, blood cells, skin cells or any other type. They are found in blastocysts, which are human embryos four to five days after fertilization, and in a few areas, such as bone marrow and gonads, in adults.

In the late 1990s, researchers developed ways to steer the development of these cells, and the possibilities for improving medicine seemed endless. If malfunctioning cells were at the root of a particular disease, could new healthy cells tailored to the job fix what was wrong? Scientists and many members of the public were eager to find out.

Anti-abortion groups, however, a key constituency of President George W. Bush, opposed the research, and in 2001 he limited federal funding to a few existing lines of embryonic stem cells, severely curtailing research.

Some in the state of California wanted to get around Bushs restrictions, and Proposition 71 was born.

"(T)hey had this demon they could attack in the campaign the Bush administration," said David Jensen, author of "California's Great Stem Cell Experiment," who also writes the blog California Stem Cell Report. "They could say, 'This is a great opportunity, and the only way we're going to get it done is to do it here in California.'"

The measure created the California Institute for Regenerative Medicine. The stem cell research agency is unique in the U.S.

"No other state has done this kind of level of funding and focus on this kind of thing, said Jensen. It's a really cutting-edge area of science."

A Few Successes

The pace of innovation has been slower than many hoped. As it turned out, grand discoveries were not around the corner, and to date there is no widespread stem cell treatment approved for the public. To date, CIRM has funded more than 64 trials directly and aided in 31 more. Not all have or will result in treatments.

But despite the lack of a marquee cure like one for Alzheimers or Parkinsons, the agency has seen some notable triumphs.

"Probably one of the most spectacular successes they have certainly so far," said Jensen, "is clinical trials that have saved the lives of what they say are 40 children."

Those children were born with severe combined immunodeficiency (SCID), commonly known as "bubble baby syndrome," a rare, generally fatal condition in which a child is born without a working immune system. An FDA-approved gene therapy that grew out of CIRM-funded research can now cure the disease by taking a patients own blood stem cells and modifying them to correct the SCID mutation. The altered cells generate new, healthy blood cells and repair the immune system.

The FDA has also approved two drugs for rare blood cancers that were developed with CIRM funds.

Sandra Dillon, a graphic designer in San Diego, credits one of the drugs with saving her life. She was diagnosed when she was just 28, in 2006. Her doctors told her they would try to manage her symptoms, but that she was going to get progressively sicker.

"Even just the idea of a cure or getting better wasn't even on the table back then," said Dillon, who is featured in ads for the Yes on 14 campaign.

"I remember just praying and begging into the universe, please, someone just look at my disease, please someone help, who is going to look at this thing.

By 2010, Dillon was extremely ill. She connected with a doctor at UC San Diego who received early-stage funding from CIRM and told her she could take part in clinical trials.

"For the first time, there was this moment of, 'Oh, my gosh! There are researchers doing something. And it could help me and I can get access to it.' It was amazing."

The drug received FDA approval in 2019, and today Dillons cancer has retreated to the point where she can live a normal life.

"I love that I am not tethered to a hospital anymore. I can go out on long backpacking trips and hiking and surfing," she said. "I am a completely different person with this drug. And I have a whole future ahead of me."

The original funding raised by Proposition 71 is running out. Proposition 14 would authorize the sale of a new bond to refill the agency piggy bank. Gov. Gavin Newsom, the UC Board of Regents, and scores of patient advocacy groups also support the measure.

Many newspaper editorial boards, however, oppose the proposition, including the San Francisco Chronicle, Mercury News and Los Angeles Times.

Right now the state still owes about $1 billion toward the debt created by Proposition 71. If Proposition 14 passes, the yearly price tag to pay off the new bond would be about $260 million per year for about 30 years.

One of the selling points of the original proposition was the potential for the state to earn big money in royalties from the treatments it helped develop, says Jeff Sheehy, an HIV patient advocate and the only CIRM board member to oppose Proposition 14.

"The promises were made that this would pay for itself. We would be able to pay back the bonds with the money we would get from royalties, etc., etc.

That has not worked out as envisioned: CIRM estimates it has received less than $500,000 in royalties. Early this year, Forty Seven, a company whose therapies were heavily funded by CIRM, sold to Gilead for $4.9 billion. While millions went to various researchers, neither CIRM nor the state of California received anything.

One of the flaws in the original measure is that we [the agency] cannot hold stock in the products that we develop," says Sheehy. "And that's because the California Constitution says that the state of California cannot, as a government entity, hold equity.

Proposition 14 makes it impossible for the state to use profits from its investment on, say, schools or other funding priorities. Instead, any royalties earned must be fed back into programs to make CIRM-funded treatments more affordable.

"What it does is it basically takes all of our returns that we get from this and gives it back to the pharmaceutical and biotech companies," said Sheehy. "It becomes just a blatant giveaway to these companies when we should be requiring access and requiring fair pricing."

Sheehy says he supports medical research, but doesn't like the state going into more debt to pay for it. The greater the state's obligations in bond money, which has to be paid back with interest, the less there is in the general fund, and Sheehy says the state has more pressing needs than stem cell research things like housing, education and transportation.

"The biggest and perhaps the most compelling reason why I feel so strongly that this is not a good idea is that we simply cannot afford it, he said. "If we think this is so important," asks Sheehy, "why don't we just don't pay for [this research] out of the general fund? It would be cheaper.

Opponents of Proposition 14 also point to longstanding complaints of conflicts of interest among the agency board. Most of the $3 billion distributed by the agency has gone to institutions with connections to board members. Critics say the structural conflicts of interest between the board and agency are not addressed in the new measure. Proposition 14 would balloon an already huge board of 29 members to 35.

Funding needs for stem cell research also are not as acute as they were back in 2004. The federal National Institutes of Health now funds some basic stem cell research, spending about $2 billion a year, with $321 million of that going toward human embryonic stem cell research. And private ventures, like nonprofits started by tech billionaires, are pouring more money into biotech.

The problem with assuming that, says Melissa King, executive director of Americans for Cures, the stem cell advocacy group behind the Yes on 14 campaign, is that CIRM fills a neglected funding need.

The NIH does not fund clinical trials at nearly the rate that CIRM can and has been, King said.

She says that's important because of what she calls the "Valley of Death," where promising early-stage research frequently fails to translate into promising treatments that can be tested in clinical-stage research. (What works well in a test tube often does not work well in an organism.) This weeding-out process is costly but necessary. And its where CIRM focused a lot of its effort.

The first- and maybe even second-phase clinical trials, its very difficult to get those funded, King said. It is too much of a risk for business to take on on its own. Venture [capital] isnt going there. Angel [funding] isnt going there.

What voters have to ask themselves, says writer Jensen, is whether stem cell funding is "a high priority for the state of California? Different people make different judgments about that."

CIRM supporters say if Prop. 14 doesn't pass, critical research will stall. Others say federal and private funding will step in and fill the gap.

Absent new funding, the institute expects it will wind down operations leading to a complete sundown in 2023.

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Proposition 14: With Just Handful of Cures, California Stem Cell Agency's Fate Is In Hands of Voters - KQED

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Pittsburgh, PA (KDKA) A West Virginia teen is among the first in the country to get gene therapy treatment for a rare condition.

Kendra Goins has Hurler Syndrome, meaning her body cannot break down certain types of sugar. In addition to damaging her organs, the condition also makes it difficult for her to grow.

Ive spoken with experts all across the country, all the way from the chemist that made this down to the doctor who has used it. This looks like her golden ticket, said Sheryl Goins, Kendras mother.

The goal of the treatment is to force Kendras body to produce the enzymes to break down sugars.

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ALDERSON, W.Va. (WVVA) A Greenbrier County teen is about to become one of the first five patients in history to receive an experimental gene therapy for Hurler's Syndrome.

The Alderson native is in many ways your typical 13-year-old. "I like to help my dad feed cows....and donkeys," said Kendra Goins.

But life hasn't always been easy for Kendra. The extremely rare condition makes it impossible for her body to breakdown certain sugars. In addition to causing damage to her organs, the condition makes it difficult for her body to grow.

But whenever anyone has anything to say about it, her sister, Kiristen is always the first time stand up.

"Me with my big head is always the one to jump in," said Goins, who said she has gotten into quite a few quarrels over the issue at school in defense of her sister.

She worries though that she won't be able to jump in when Kendra heads to California soon for a clinical trial. Kendra is heading into the treatment with her first bone marrow transplant nearing the end of its course.

"I've spoken with experts across the country from the chemists who made the drug to the doctor who has used it. This looks like her golden ticket," said Kendra's mother Sheryl Goins.

Administered through the brain, the goal of the gene therapy is to help her body produce the enzymes she needs to survive.

While the cost of the clinical trial and airfare is covered, the family said they need help with expenses they will incur during their three-month stay.

To learn more about how you can help, visit https://www.gofundme.com/f/kendra039s-gene-therapy?utm_source=facebook&utm_medium=social&utm_campaign=p_cp%20share-sheet&fbclid=IwAR3yzHCexch5a_awjaYy06ijB28zMXJ-72WkfQ-SPEwMQgy5s8x_carlz34

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Regenxbio announces expansion of RGX-121 clinical program

Regenxbio Inc. (RGNX) announced the expansion of its Mucopolysaccharidosis Type II clinical program. The company stated that the expansion will allow it to gain additional understanding of the neurodegenerative aspects of the disease. The expansion will also let Regenxbio in a broader patients population.

The Phase I/II study seeks to examine a single intracisternal administration of RGX-121 in patients under the age of 5 years. All these patients have severe MPS II. The company has already dosed six patients across two dose levels. The main aim of the study is to assess the safety and tolerability of RGX-121 and to evaluate the impact of the drug candidate on biomarkers of I2S enzyme activity, neurocognitive development and other clinical measures.

Regenxbio intends to start expanded enrollment of patients in Cohort 2 immediately. The decision has been taken based on the basis of recommendations made by the Independent Data Monitoring Committee and MPS II treating physicians. Steve Pakola, M.D., Chief Medical Officer of Regenxbio said, Regenxbio is committed to advancing potential gene therapy treatment options for MPS II, as there remains a significant unmet medical need to address the neurological manifestations and prevent or stabilize cognitive decline for patients.

As of September 16, 2020, RGX-121 was found to be well-tolerated in all six patients. No drug-related serious adverse events were reported. The company now plans to dose up to six additional patients with a second dose level of 6.5x1010 genome copies per gram (GC/g) of brain mass. Regenxbio expects to provide further updates from the study by the end of 2020.

Regenxbio also reported that the FDA has cleared its Investigational New Drug Application. The company plans to start the second Phase I/II trial of the drug candidate for treating pediatric patients with severe MPS II ages 5-18 years old. This multicenter, open-label trial will enroll up to six patients and the drug candidate will be administered directly to the cerebrospinal fluid through intracisternal or intracerebroventricular injection.

Regenxbio plans to open a new observational natural history trial for enrollment in the second half of 2020. This trial will study the neurocognitive development and key biomarkers in patients with severe MPS II. The trial will prospectively record the changes in neurodevelopmental issues of cognitive, behavioral and adaptive function over a period of time. It will also evaluate biomarker activity in serum, urine and the CSF. The trial will enroll up to 40 patients between the ages of 1 month and 8 years with genetic diagnosis of severe MPS II. The assessment will be carried out for up to two years.

RGX-121 is being developed for treating Mucopolysaccharidosis Type II. It aims to work by using the AAV9 vector for delivering the human iduronate-2-sulfatase gene which is responsible for encoding the iduronate-2-sulfatase (I2S) enzyme to the central nervous system. RGX-121 has been granted orphan drug product, rare pediatric disease and Fast Track designations by the FDA.

Investment Thesis: Regenxbio had a tumultuous past year and the stock has lost nearly 50 percent of its Pre-Covid valuation. While the latest news is encouraging, it may be a little too early to gauge the companys prospects ahead.

AbbVie Inc. (ABBV) announced that it has taken a voluntary decision so pull Fibristal from the Canadian market. The decision has been taken after the news of certain patients in Europe contracting rare cases of liver injury and requiring transplantation. AbbVie integrated Allergan's business to its core in May this year. The drug belongs to Allergan and the company decided that the recall is the best option in the prevailing circumstances.

Fibristal contains Ulipristal acetate as one of its main ingredients. Earlier this year, the European Medicines Agencys Pharmacovigilance Risk Committee (PRAC) had recommended the cancelation of the marketing authorization of products containing ulipristal acetate used for treating symptoms of uterine fibroids.

Fibristal in approved in Canada for treating moderate-to-severe symptoms of uterine fibroids in adult women of reproductive age. It is also approved as an intermittent treatment of moderate-to-severe signs and symptoms of uterine fibroids in adult women of reproductive age who are not eligible for surgery.

Health Canada is monitoring the effectiveness of the recall. It has advised the patients who take Fibristal to a) stop treatment and contact their doctor to discuss other treatment options; and b) immediately report to their doctor signs and symptoms of liver injury such as nausea, vomiting, stomach ache, severe tiredness, yellowing of the eyes or skin, or dark urine, which could occur after stopping treatment. Health Canada has also issued directives to healthcare professionals to stop prescribing or dispensing Fibristal and to carry out appropriate liver function monitoring.

Investment Thesis: AbbVie is a blue-chip stock with solid foundation. This negative news is not likely to have any material impact on the stock price as the company maintains its robust long-term outlook.

Ionis Pharmaceuticals Inc. (IONS) announced inking a multi-year collaboration with Genuity Science. The deal pertains to the discovery and development of innovative therapeutics across a range of up to 20 diseases. Ionis is looking to boost its presence in key segments with this collaboration and draw synergies.

Genuity Science focuses on high-quality, whole-genome sequence and deep phenotype data. Ionis plans to combine this approach with its proprietary antisense technology to expedite the drug development process. Brett P. Monia, Ph.D., chief executive officer of Ionis said, "The collaboration with Genuity Science gives us the potential to significantly increase our discovery opportunities and better understand the complexities of human diseases based on genetics."

Under the terms of the agreement, Genuity Science will be entitled to receive a combination of upfront payments and development milestones. It will also be entitled to gain product royalties. Genuity Science is a data sourcing, analytics and insights organization. It collaborates with global pharma companies to catalyze precision health services. It offers a wide range of services including high-quality sequencing and disease-specific data sourcing.

Ionis is mainly active in RNA-targeted drug discovery and development segment. The company has developed the first and only approved treatment for all patients, children and adults with spinal muscular atrophy. Its portfolio also contains the worlds first RNA-targeted therapeutic approved for the treatment of polyneuropathy in adults with hereditary transthyretin amyloidosis. The company uses its antisense technology platform to boost its development pipeline. Its drug candidate Pelacarsen is in Phase 3 for cardio-renal indications. Some of its main development partners are Biogen, AstraZeneca and Novartis.

Investment Thesis: Ionis has a well-diversified development pipeline which is expected to keep its stock price buoyant. However, the price has tumbled quite a bit in the recent past, providing an exciting opportunity to build a portfolio with a long-term horizon.

Thanks for reading. At the Total Pharma Tracker, we do more than follow biotech news. Using our IOMachine, our team of analysts work to be ahead of the curve.

That means that when the catalyst comes that will make or break a stock, weve positioned ourselves for success. And we share that positioning and all the analysis behind it with our members.

Disclosure: I/we have no positions in any stocks mentioned, and no plans to initiate any positions within the next 72 hours. I wrote this article myself, and it expresses my own opinions. I am not receiving compensation for it (other than from Seeking Alpha). I have no business relationship with any company whose stock is mentioned in this article.

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Regenxbio RGX-121 Clinical Program, And Other News: The Good, Bad And Ugly Of Biopharma - Seeking Alpha

Dr. Evelyne Bischof speaking at the 2020 China-Israel Summit on Longevity Medicine

Over the past decade, we witnessed unprecedented advances in the field of biogerontology, and the massive convergence of biotechnology, information technology, AI, and medicine. And now we are witnessing the birth of a new field of longevity medicine, which integrates the latest advances in many of these fields of science and technology. My definition longevity medicine is advanced personalized preventative medicine powered by deep biomarkers of aging and longevity.

And, like in the field of AI for drug discovery, women are at the forefront of this revolution and there were precedents when we had to look for a male physician to make a conference panel more diverse.

One of the physician-scientists who stands out in this area is Dr. Evelyne Yehudit Bischof. I first got a note with a request for more information on one of our research papers from Dr. Bischof on December 30th, 2019 while in Shanghai. A request I almost ignored due to the heavy workload but accidentally I looked at her profile which was highly unusual. In brief, Evelyne is a German medical doctor with an MD from Max Planck Institute for Molecular Biology and Genetics, who interned at Columbia University, and Harvard MGH and Beth Israel Medical Deaconess, attending physician at University Hospital Basel in Switzerland, and associate professor at Shanghai University of Medicine and Health Sciences. She fluently spoke six languages including German, Russian, and Mandarin Chinese, which was quite impressive. The second time we met was at Human Longevity Inc, in San Diego when she was interviewing with one of the most influential entrepreneurs and investors in longevity biotechnology, Dr. Wei-Wu He to join HLI as a longevity physician.

Dr. Evelyne Yehudit Bischof

The longevity industry is rapidly emerging and longevity clinics are being set up in various parts of the world. So I decided to ask Eva a few questions to elucidate this new and emerging industry.

Alex: Eva, we know each other for almost a year and you do not fail to impress with your academic publications, public lectures, and clinical work. You are as close to the longevity physician as it can possibly get. Can you tell us a bit more about yourself and about the work that you are doing on the clinical side and on the research side?

Dr. Evelyne Bischof: Thank you, Alex it is an honor to be so generously introduced by a true innovator, scientist and entrepreneur, as well as a longevity KOL and allow me to revert the compliment. I am a rather globally oriented internal medicine specialist, with training and work experience in Germany, USA, Switzerland and China. For almost a decade now, I have been splitting my time between Shanghai and Basel, creating a path that allowed me to conclude my residency and fellowship, develop translational and clinical research niches and collaborators, as well as to engage actively in academic medical education. While my clinical work was mostly based in a university clinic in Basel in internal, intensive and onco-hematologic medicine wards, my scientific pursuits and academic teaching were mostly based in Shanghai, where I went along the track from a junior lecturer to an associate professor in 2016. My research focused primarily, but not exclusively, on oncology and being an internist at core on geroncology and precision medicine in general internal medicine. Geroncology is a crucial field that investigates the very much interlinked pathways of aging and tumorigenesis, leading to the epidemiological observation that age is the number one risk factor to develop cancer for all.

Both Switzerland and China are innovative hubs with strong medical and bioscientific profile, which allowed me to learn from some of the finest experts worldwide. The frequent travels and splitting my life between continents were not always easy, but - coming from a simple background of non-academic farmer and handcraft family Alongside - I will be forever grateful for all the great people I met and worked with, the abundant cultural nuances and differences I was able to learn and appreciate, the stimulating and constructive exchange and so much more in soft and hard skills, on professional and personal level. with the emergence of AI-based solutions in the clinic and with the rise of longevity medicine, my passion and efforts are now focused on these domains, while I continue my clinical practice in the university hospitals, academic lecturing at two medical schools (currently in Shanghai - due to COVID-19-related travel restrictions) and research/public speaking (globally - thanks to COVID-19-related shift to virtual communication).

Alex: Can you tell us about your perspective on the emerging field of longevity medicine starting from your own definition of the field?

Dr. Evelyne Bischof: With pleasure! My personal definition of longevity medicine is clear: it is precision medicine driven by deep aging biomarkers. Surely, the definition is succinct, but extremely deep. Precision medicine is per se an enormously complex and dynamic field, driven by multimodally mined data and their constant re-evaluation, reannotation and reiteration to provide qualitative and quantitative using AI-algorithm outputs applicable for clinical practice. Longevity medicine is a to say the next generation of precision medicine that evaluates the patient within the reference range for the patients ideal age (usually 20-30) and is looking for ways to reduce the gap between the current parameters and the parameters of maximum physical performance for the ideal age. Deep aging clocks as quantifiable, trackable and accurate biomarkers of aging and an indispensable component of longevity medicine. Without being able to actually measure the biological age and its changes due to interventions, longevity medicine cannot be performed. I strongly believe that this field of medicine will revolutionize healthcare and change the mindset of all the doctors, the policy makers, the stakeholders and above all: the patients. Allow me to add that I consider each of us as a patient we all suffer from aging! I also believe that citing Peter Diamandis in the future, if a physician wont be using A.I. in guiding diagnosis and therapy, it'll be a malpractice". This said, I would love to add that we need more passionate physicians in longevity and this can only be achieved with an appropriate educational setting, which will be inaugurated this month by Deep Longevity and collaborators.

Alex: What do you see as the most promising developments in the field of longevity medicine that can truly push the needle and add a few decades if not more to the healthy youthful life of the individual?

Dr. Evelyne Bischof: Besides of deep aging clocks and AgeMetrics, which I truly without cronyism embrace and would encourage all physicians to implement in their daily practice, I see a big potential in gene therapies, in (natural and designed) gerolytics and senolytics, as well as supplements that will show safe efficacy in combating senescence from the molecular to system level. Studies on AKG, rapamycin and metformin are already fueling this hope. Of course, all interventions will require a prior comprehensive precision health assessment and continuous monitoring. For the latter, the wearables and applications will certainly bring us even faster to an extension of a healthy and productive lifespan.

I am encouraged by the fact that there are two major developments, perpetuated by the racing speed of longevity medicine and geroscience. Number one: doctors are shifting from putting a patient on meds to putting a patient on a personalized longevity protocol that becomes a natural, integral, rewarding part of their lives. Number two: society is realizing that it is not important how old one is, but how one shows his/her own age. Remembering this allows one to make sure he or she does not become a slave of the myths about the elderly, but also to be mindful that even at an early chronological age, one might actually experience silent accelerating aging due to modifiable risk factors or pathomechanisms.

Alex: Without promoting Human Longevity Inc or Health Nucleus 100+, can you tell us what an average person with an average income can do to increase their performance and longevity?

Dr. Evelyne Bischof speaking at the 2020 China-Israel Summit on Longevity Medicine

Dr. Evelyne Bischof: This is a very valid question in fact, when it comes to reasonably boosting performance and creating a good base for longevity, one does not necessarily be wealthy. The components of the magic mixture are the well-known pillars of preventative and functional medicine: exercise, nutrition, supplements, moderation. However, longevity physicians are now able to customize the right proportions of each for a specific person, minding the biovariability, comorbidities, chronological age, but also lifestyle and preferences. In an extreme generalization, I would suggest caloric restriction via intermittent fasting to an overall healthy person, with at least an A-Z vitamin and mineral supplement, 15-30min workout at least 3 times a week, moderation in substance use to the minimum, but with permissible enjoyment, if needed (alcohol and cigarettes), a minimum of 6 hours of sleep without interruption, circadian rhythm (regular times) of sleep and food intake, no meals at night (at least 4 hours before night rest) and very importantly cognitive activities (books, foreign languages, crosswords), preferably rewarding ones so that the psychological wellbeing area is also covered. Everyone is able to use stairs as their gym, to not to eat before sleep, to choose water over other drinks, to laugh aloud to oneself and to learn text parts by heart (because decelerating psychological aging and cognitive decline are crucial aspects of healthy longevity). I recall I was always reading the ingredients and how to use? texts on tubes during shower, so as not to waste the time. My first sentence in Russian was actually the instruction of how to use a shampoo.

Dr. Evelyne Bischof speaking at a conference on aging and longevity

Alex: And if someone has nearly unlimited access to capital, what should they do?

Dr. Evelyne Bischof: I believe, as in any other business or property of this particular population, the individuals should seek good investments and insurance in relation to their health and the health of their significant surrounding (family, friends, workers etc.). The investment should involve as precise diagnostics as possible, that harnesses all cutting edge and untapped potential of the human genome, deep quantitative phenotyping, complete -omics and -ioms (e.g. microbiome, epigenomics, metabolomics, proteomics etc.), advanced imaging with radiogenomic algorithms etc. As it is a dynamic field, constantly evolving and implementing new features and/or better ways of interpretation, such diagnostic comprehensive checkups (or part of them) should be repeated regularly. The insurance part does not relate to a contracted policy, but to a complex entity of lifestyle recommendations and interventions lead by an entrusted longevity physician (basically a physician that can list and pronounce the aforementioned terms), who understands and permanently advances in the field, being able to combine human and artificial intelligence and customize an individual approach of prevention and (if needed) therapy for a specific patient. In addition, the leading physician needs to comprehend and implement the personal challenges and preferences of the patient, such as mostly disturbed wake-sleep rhythm, irregular and unhealthy social meals, acute and chronic stress exposure, irritability or fatigue etc., to create a program that will be realistic, allow the patient to remain compliant and engaged based on his/her educated informed decisions. Simply said: knowing 150 GB of a patients data, a physician of trust should be a good lead towards identification, mitigation and elimination of actionable diseases (years and decades ahead) and risk factors that curb the quantity and quality of life.

Alex: I know maybe 3-4 people like you in the world, who have an MD, are actively engaged in biomedical research, and work with some of the high-profile clients who are spoiled with the most cutting-edge medical care provided by the top medical institutions. And all of them are women. Why do we see such gender imbalance in the field?

Dr. Evelyne Bischof in the clinic

Dr. Evelyne Bischof: Again thank you very much for this encouraging statement, this time speaking on behalf of women in medicine, academia and STEM. As you know, one of my side areas of interest is the study of biological sex differences in various diseases, predominantly cancer, and ultimately also on the sex (biological) and gender (socio-cultural) variables influencing pathomechanisms, diagnostic and therapeutic decisions, resulting differing toxicities, follow up strategies and outcomes (recovery, chronification etc.). It was natural to engage in debates and develop curiosity about the gender distribution in academia in general. Recently, with an ad hoc group of collaborators from Europe, USA and China, we demonstrated in a Lancet Oncology paper that female representation at the podium, meaning as keynote speakers and scientific committees at the largest oncological conferences in China. Our data showed that China is much more inclusive, without an intensive active promotion or directives towards gender quotas. As you know, I am a big fan of this country, but this quantitative study once again showed how impressive this country is and perhaps we found one of the contributing factors for the nations booming leading role in biotech and medicine.

Overall however, there are indeed significant differences in various fields, as well as an overall underrepresentation of females in leadership and podium roles. I am happy to see that in longevity science and medicine, we have dedicated females that can unfold their passions and translate them into viable solutions that do impact the public and individual health. As always, the reasons are multifold, but perhaps the most important one is that in longevity, driven women are emerging in an inclusive environment that embraces non-discriminating and non-stigmatized diversion. In different words: the longevity field seems to embrace inclusion at the same (ultrarapid) pace as STEM and medicine are evolving. The sex and gender differences clearly allow to generate creativity and innovation it is a mutually perpetuating process. Last but not least, it is thanks to committed male mentors and collaborators that actually value D&I (diversity and inclusion) intuitively or knowingly (based on evidence that diverse teams outperform the less diverse one by over 35%). Most male KOLs in longevity, like yourself, promote and underline the importance of D&I. On a final note myself, personally, I have always remained at the unconscious side when facing a person I work with. Accountability, motivation and fairness have proven to be non-gender related in my experience as I have faced many challenges being a (previously young) female, permanent foreigner and on top of that blond. The typical situation at a round dinner table in China with 12 male professors usually ended up with us all laughing at my gambei with water being the only discrepancy from the norm.

Dr. Evelyne Bischof speaking at the 2020 China-Israel Summit on Longevity Medicine

Original post:
Women In Longevity Medicine And The Rise Of The Longevity Physician - Forbes