Author Interviews, Genetic Research, Hematology / 08.12.2020

MedicalResearch.com Interview with: Steven Pipe, MD Professor of Pediatrics and Pathology Laurence A. Boxer Research Professor of Pediatrics and Communicable Diseases Pediatric Medical Director, Hemophilia and Coagulation Disorders Program Director, Special Coagulation Laboratory University of Michigan MedicalResearch.com: What is the background for this study? Response: Hemophilia B is an inherited bleeding disorder where patients are missing clotting factor IX (9), a critical blood clotting protein. Patients with a severe deficiency are at risk for traumatic and spontaneous bleeds – primarily into joints. Repeated bleeding into joints causes more than acute pain and swelling but also leads to inflammatory and degenerative changes in joints that eventually leads to severe debilitating arthritis that can be crippling. To try to prevent this, patients as young as infants are placed on regular infusions of clotting factor IX concentrates. The relatively short half-life of factor IX means patients must infuse on average once to twice a week. These can only be delivered intravenously – parents and then patients themselves have to learn this. Prophylaxis must be continued lifelong to try to prevent bleeding events and protect joint health over the lifespan. This is a tremendous burden on the patient and their caregivers. Even with regular prophylaxis, joint bleeds may still occur and arthropathy may still ensue. This is because the blood levels often reach critically low levels prior to the next infusion. Gene therapy aims to deliver a functional copy of the factor IX gene such that the patient’s own liver will make a continuous supply of factor IX that is delivered to the bloodstream. At steady state with levels close to or within the normal range, patients would no longer be subject to bleeding events and would not require prophylaxis any longer. We hope that such a one-time treatment would produced durable, “functionally curative” levels of factor IX. (more…)
Author Interviews, Cancer Research, COVID -19 Coronavirus, Leukemia / 06.12.2020

MedicalResearch.com Interview with: William Wood, MD Associate Professor of Medicine, Division of Hematology UNC School of Medicine William Wood, MD Associate Professor of Medicine, Division of Hematology UNC School of Medicine MedicalResearch.com: What is the background for this study? What are the main findings? Response: In the earliest days of the COVID-19 pandemic, there were concerns that individuals with cancer, and especially those with hematologic malignancies, could be at higher risk for adverse outcomes following COVID-19 infection than the general population. For this reason the ASH Research Collaborative developed a voluntary data collection registry in which providers or sites caring for patients with hematologic malignancies and COVID-19 infection provided de-identified data via an online data collection platform. We believe that our findings – that 20% of patients with blood cancers who had COVID-19 infection died, including 33% of those who required hospital or ICU level-care – indicate that patients with blood cancers are a medically vulnerable group when it comes to COVID-19. The risk of dying was highest in patients who were older, had more severe infection, opted to forego more intensive treatment, and/or had poorer prognosis before their COVID-19 infection, as determined by their treating clinicians (more…)
Author Interviews, COVID -19 Coronavirus, Hematology / 04.12.2020

MedicalResearch.com Interview with: George Sakoulas, MD Sharp Memorial Hospital and Sharp Rees-Stealy Medical Group Associate Adjunct Professor UC San Diego School of Medicine San Diego MedicalResearch.com: What is the background for this study? Would you briefly explain what is meant by IVIG? Response: IVIG stands for intravenous immunoglobulin. It is a preparation from pooled blood donors isolating antibodies in their blood and highly concentrated for use. It's uses are to either replace immunoglobulins in patients who do not produce enough of them because of some underlying immunodeficiency or, as in the case of COVID, they take advantage of the immune signaling properties of the non-variable part of the antibodies. Many immune cells have receptors that bind the non-variable (also called the Fc-gamma region), and when bound, the activity of these activated immune cells is modulated. IVIG is used in many immunologic complications of infections like Guillan-Barre, Kawasaki's disease, immune mediated encephalitis from mycoplasma, just to name a few. We decided to study IVIG because:
  1. Our anecdotal yet successful use of IVIG in treating ARDS due to influenza and other viruses.
  2. Our very definitive successful use of IVIG in a very sick patient with COVID early in the pandemic.
  3. The weak yet positive retrospective data from China of IVIG in COVID.
  4. The mechanism of IVIG inhibiting neutrophil extracellular trap (or NETS) thought to play an important role in COVID severe disease.
  5. IVIG has been around to 4 decades and many physicians have a level of prescribing comfort.
It is worth clarifying that the benefit here is NOT from neutralizing antibodies against SARS-CoV-2 virus but rather the immunomodulatory effect. It will be very interesting to see if the effect/benefit changes over time as pooled blood donors feeding the IVIG supply do develop more antibodies against SARS-CoV-2, but this is not yet known. (more…)