GHP Q2 2019

GHP / Q2 2019 5 NEWS , “Antibody-drug conju- gates are an emerg- ing class of targeted immunotherapies for cancer. This type of therapy, unlike traditional chemo- therapy, is intended to target specific cells,” said Richard Paz- dur, M.D., director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Prod- ucts in the FDA’s Center for Drug Evaluation and Research. “To- day’s approval of Polivy provides an alternative option for patients in whom multiple treatments have not worked.” More than 18,000 people are di- agnosed with DLBCL each year in the U.S. Although it can be cured, about 30 to 40% of pa- tients suffer relapse. This type of cancer grows quickly in the lymph nodes and may affect the bone marrow, spleen, liver or other or- gans. Signs and symptoms of DL- BCL may include swollen lymph nodes, fever, recurring night sweats and weight loss. Polivy is an antibody that is at- tached to a chemotherapy drug. Polivy binds to a specific protein (called CD79b) found only on B cells (a type of white blood cell), then releases the chemotherapy drug into those cells. Efficacy was evaluated in a study of 80 patients with relapsed or refractory DLBCL who were randomized to receive Polivy with BR or BR alone. Ef- ficacy was based on complete response rate and duration of response (DOR), defined as the time the disease stays in remis- sion. At the end of treatment, the complete response rate was 40% with Polivy plus BR compared to 18% with BR alone. Of the 25 patients who achieved a partial or complete response to Polivy plus BR, 16 (64%) had a DOR of at least six months and 12 (48%) had a DOR of at least 12 months. The most common side effects of Polivy plus BR include low levels of white blood cells (neutropenia), platelets (thrombocytopenia) and red blood cells (anemia); nerve damage (peripheral neuropa- thy); fatigue; diarrhea; fever; de- creased appetite; and pneumo- nia. A new study has found a pattern of molecules that ap- pear in the blood before a seizure hap- pens. This discovery may lead to the development of an early warning system, which would en- able people with epilepsy to know when they are at risk of having a seizure. Researchers at FutureNeuro, the SFI Research Centre for Chronic and Rare Neurological Diseases hosted at RCSI (Royal College of Surgeons in Ireland), led the study, which is published in the current edition of the Journal of Clinical Investigation (JCI). FutureNeuro and RCSI research- ers have discovered molecules in the blood that are higher in people with epilepsy before a sei- zure happens. These molecules are fragments of transfer RNAs (tRNAs), a chemical closely re- lated to DNA that performs an important role in building proteins. When cells are stressed, tRNAs are cut into fragments. Higher lev- els of the fragments in the blood could reflect that brain cells are under stress in the build up to a seizure event. Using blood samples from peo- ple with epilepsy, the group found that fragment levels of three tR- NAs “spike” in the blood many hours before a seizure. “People with epilepsy often report that one of the most difficult as- pects of living with the disease is never knowing when a seizure will occur,” said Dr Marion Hogg, Fu- tureNeuro investigator, Honorary New Research Could Help Predict Seizures Before They Happen Research led by FutureNeuro at RCSI could improve the lives of people with epilepsy. Lecturer at RCSI, and the study’s lead author. “We hope that our tRNA research will be a key first step toward de- veloping an early warning sys- tem.” The World Health Organisation estimates that more than 50 mil- lion people worldwide have epi- lepsy, and one third of those do not respond to current treatments. “New technologies to remove the unpredictability of uncontrolled seizures for people with epilepsy are a very real possibility,” said Professor David Henshall, Direc- tor of FutureNeuro and Profes- sor of Molecular Physiology and Neuroscience at RCSI who was a co-author on the paper. “We in FutureNeuro hope to de- velop a test prototype, similar to a blood sugar monitor that can potentially predict when a seizure might occur.” Funders of the research included Science Foundation Ireland (SFI), the European Regional Develop- ment Fund, FutureNeuro industry partners and the European Un- ion’s ‘Seventh Framework’ Pro- gramme FP7 (EpimiRNA). Health care professionals are ad- vised to monitor patients closely for infusion-related reactions, low blood counts and fatal and/ or serious infections. Health care professionals should also monitor patients for tumor lysis syndrome (a complication from many tumor cells being killed off at the same time), liver damage (hepatotoxici- ty) and progressive multifocal leu- koencephalopathy (PML), a fatal or life-threatening infection of the brain. FDA advises health care professionals to tell females of reproductive age to use effective contraception during treatment with Polivy and for three months after the last dose. Women who are pregnant or breastfeeding should not take Polivy because it may cause harm to a developing fetus or newborn baby. Polivy in combination with BR was granted accelerated ap- proval, which enables the FDA to approve drugs for serious condi- tions to fill an unmet medical need based on an endpoint that is rea- sonably likely to predict a clinical benefit to patients. Further clinical trials are required to verify and describe Polivy’s clinical benefit. The FDA granted this applica- tion Breakthrough Therapy and Priority Review designations. Polivy also received Orphan Drug designation, which provides in- centives to assist and encourage the development of drugs for rare diseases. The FDA granted the approval of Polivy to Genentech.