After over a decade of preclinical research and development, a new gene therapy treatment for Sickle Cell Anemia (SCA) is reversing disease symptoms in two adults and showing early potential for transportability to resource-challenged parts of the world where SCA is most common. Preliminary data from a pilot Phase 1-2 clinical trial testing the gene-addition therapy were presented Dec. 3 at the American Society of Hematology’s (ASH) annual meeting in San
Researchers report in Nature Communications they figured out why air sacs in the lungs clog up with a thick substance called surfactant in a brutal disease called Pulmonary Alveolar Proteinosis (PAP), and they show taking cholesterol-busting pills called statins can effectively treat the disease. That’s good news for people with PAP because at present the current standard treatment is something called a whole lung lavage. Essentially, it involves flushing patient
CHOP Oncologists Update Analysis in Pivotal Global Trial of CAR T-Cell Treatment for Relapsed or Refractory ALL Updated results from a global clinical trial of the CAR T-cell therapy, tisagenlecleucel, a landmark personalized treatment for a high-risk form of acute lymphoblastic leukemia (ALL), reveal that children and young adults continued to show high rates of durable, complete remission of their disease. Most side effects were short-lived and reversible, according to
An antibody therapy against a key inflammatory molecule involved in migraines reduces the number of headaches that chronic migraine patients experience per month in a phase III trial. A new study of fremanezumab, an immunotherapy that counteracts one of the molecules released during migraine, was found successful in reducing the number of days that chronic migraine sufferers experienced headaches. The results of the phase III clinical trial were published November
Findings from a phase III clinical trial point to a more effective initial treatment for patients with ALK-positive non-small cell lung cancer (NSCLC). Compared to the current standard of care crizotinib (Xalkori), the newer ALK inhibitor alectinib (Alecensa) halted cancer growth for a median of 15 months longer and caused fewer severe side effects. The study was featured in a press briefing today and presented at the 2017 American Society
Medicortex Finland Oy, a biotechnology company developing a diagnostic kit for brain injury detection based on a medical breakthrough biomarker, recently completed analyzing the results from the first clinical trial. Medicortex is working towards the validation of a Traumatic Brain Injury (TBI) biomarker and incorporating it into a quick and reliable diagnostic kit that can be easily used by the first responders and healthcare professionals, and by people with no
An Ontario-led international clinical trial with 1,206 men with localized prostate cancer shows that compressing radiation treatments into four weeks from eight delivers similar outcomes. The findings, published online today in the Journal of Clinical Oncology, provide a new standard of care worldwide, which the participating centres have already adopted, says co-principal investigator Charles Catton, radiation oncologist, Princess Margaret Cancer Centre, University Health Network. Dr. Catton is also a Professor,
New clinical trial offers combination therapy to directly kill cancer cells and teach the immune system to keep them from coming back. Fewer than half of women diagnosed with ovarian cancer live for five years or more. Sarah Adams, MD, hopes her new clinical trial will change this outcome. Adams recently opened a clinical trial at The University of New Mexico Comprehensive Cancer Center to test a new approach to
Imagine conquering childhood cancer, only to find out that years down the road your heart may fail. Unfortunately, many children who have battled cancer face this reality. While often lifesaving, the effects of chemotherapy treatment (drugs that kill cancer cells) can take a toll on the developing body of a child, potentially resulting in life-threatening late side effects like cardiac damage. “You go through terrible chemotherapy, achieve remission, have a
Building on earlier clinical trials, UCLA researchers have confirmed that the “breakthrough” drug palbociclib when used in combination with the traditional hormonal therapy letrozole delays progression of advanced breast cancer significantly and without the harsh side effects seen in some women prescribed letrozole alone. The study, published online in the New England Journal of Medicine, is the phase 3 study following phase 1 and phase 2 clinical trials that led
The results of numerous high-impact clinical trials that could affect kidney-related medical care will be presented at ASN Kidney Week 2016, November 15–20 at McCormick Place in Chicago, IL. • In the Liraglutide Effect and Action in Diabetes: Evaluation of cardiovascular outcome Results (LEADER) trial, 9340 patients with type 2 diabetes and high cardiovascular risk were randomized liraglutide (a long-acting glucagon-like peptide-1 analog) or placebo. Over a median follow-up of
Genetic profiling of cancer tumors provides new avenues for treatment of the disease, according to a study conducted by Sanford Health and recognized by the American Society of Clinical Oncology. In 2014, Sanford developed and launched the Genetic Exploration of the Molecular Basis of Malignancy in Adults, or GEMMA, to determine if evaluating genetic information could help customize treatment options for adult patients whose cancer had progressed after the first
A phase 1 clinical trial testing a novel combination therapy developed by scientists at VCU Massey Cancer Center slowed the growth of cancer in the majority of trial participants, which were patients with advanced solid tumors. Approximately 61 percent of these patients experienced some degree of tumor growth delay, with multiple partial responses and one complete response. A phase 2 study testing the same combination of the drugs sorafenib and
The lack of participation in clinical research may be the Achilles’ heel of today’s cancer community. According to a new survey of more than 1,500 consumers and nearly 600 physicians conducted on behalf of Memorial Sloan Kettering Cancer Center (MSK), only 35 percent of Americans indicated that they were “likely” to enroll in a clinical trial. Other studies have shown that only 4 percent of cancer patients enroll in clinical
