Tackling a Rare Disease: Working Toward a Treatment for SMA
Most people have never heard of spinal muscular atrophy (SMA), a rare genetic disease that affects approximately 1 in 6,000 babies born worldwide each year. The early-onset form of the disease is almost always fatal.
Teamwork drives Novartis’ SMA research
Launching drug discovery for a rare disease takes guts. Not only do researchers make bold bets on new science, they often need to join forces with collaborators and push themselves beyond their comfort zones.
Novartis researches SMA’s genetic roots
Spinal muscular atrophy (SMA) offers challenges both exciting and daunting to biomedical researchers: Exciting because of its well understood genetics and daunting because no one has yet succeeded in delivering an approved therapy.
Novartis scientists target resistance threat in malaria
"Novartis is in the fight against malaria for the long term and we are committed to the continued research and development of new therapies to eventually eliminate the disease,” said Joseph Jimenez, CEO of Novartis."
Mutant Protein Spreads Through a Neural Circuit in Huntington’s Disease
The discovery suggests a new therapeutic strategy for the neurodegenerative disease.
Cancer Cocktails Come of Age
Novartis teams test combinations of targeted therapeutic agents to combat drug resistance in tumors.
Cancer Cocktails: LJM716 – Closing A Common Release Valve
The promise of LJM716 lies in its combination with other targeted agents. HER3, its target, isn’t usually the main driver of tumors, but it’s important in the development of resistance following treatment.
Cancer Cocktails: LEE011 – A Popular Combination Partner
LEE011 is designed to inhibit components of a switch that controls the cell cycle. Theoretically, it could be paired with agents that target proteins upstream of the switch to combat the emergence of resistance.
Cancer Cocktails: ABL001 – Hitting One Target Twice
ABL001 is a small molecule designed to inhibit BCR-ABL, the same protein hit by imatinib and nilotinib, which are used to treat chronic myelogenous leukemia. But ABL001 has a different mechanism of action.
Nobel winner shares secret to success
Failure didn’t stop Dr. Richard Roberts. The Nobel laureate failed high-school physics before excelling at the subject and rising to scientific stardom as a genetics and molecular biology pioneer. Dr. Roberts shared his story with local middle and high school students gathered at Novartis Institutes for BioMedical Research (NIBR) in Cambridge, Mass., on May 21.
Why Novartis Scouts for Disruptive Technology
Can drug discovery scientists identify disruptive technology before it takes them by surprise? If a recent collaboration is any indication, the answer is yes.
Robots Speed the Pace of Modern Drug Discovery (Video)
When research scientist Elaine Donohue arrives at the Novartis Screening Group each morning, anyone tagging along with her could be forgiven for thinking they’d just stepped onto the set of a sci-fi movie.
FDA Approves Zykadia (LDK378) in ALK+ Non-Small Cell Lung Cancer
The U.S. Food and Drug Administration (FDA) approved Zykadia (ceritinib, previously known as LDK378) for the treatment of patients with anaplastic lymphoma kinase positive (ALK+) metastatic non-small cell lung cancer (NSCLC) who have progressed on or are intolerant to crizotinib. Born at the Genomics Institute of the Novartis Research Foundation (GNF) and advanced in collaboration with other groups, LDK378 has been on the fast track for approval since it received Breakthrough Therapy designation in March 2013. It first achieved Proof of Concept in May 2012.
A Personal Quest to Prevent Blindness (Video)
A family member’s suffering motivates translational medicine expert Parisa Zamiri to find treatments for ophthalmologic diseases. Her current research focus is dry age-related macular degeneration, a significant cause of blindness in older people worldwide.
A “Swell” discovery on how cells keep in shape
The Cell cover illustration depicts a normal and a swollen cell as artworks in a museum: one small and dwarfed by a dense, wrinkly frame and the other large and smooth. The cover was designed by the illustrator Jorge Colombo using finger painting on an iPad. The GNF scientists admired his work in the New Yorker and contacted him to illustrate their paper in Cell about how cells sense their environment.
Addressing Cognitive Symptoms of Schizophrenia Through a Nicotinic Acetylcholine Receptor Alpha 7 Partial Agonist
NIBR researchers led by Konstanze Hurth (global discovery chemistry) and Dominik Feuerbach (neuroscience) set about looking for something that could help patients suffering from Schizophrenia with daily functioning and social integration.
Collaborating to unravel clues about neuropsychiatric diseases
NIBR teams up with the Broad Institute to lay a foundation for rational drug discovery in autism, schizophrenia, and other brain disorders.
Scientific Renewal Keeps Discoveries Flowing
NIBR scientists embark on mini-sabbaticals to acquire new skills and perspective.
What’s all this talk about rare disease?
The size of the patient population does not drive our research. Do we understand the science? Is there is unmet medical need? Those are the questions the Novartis Institutes for BioMedical Research asks. Surprising for a Pharma Organization? Not really. Here’s why.
Rare Disease - Focus on the patient, follow the science
Sporadic inclusion body myositis (sIBM) is the most common acquired muscle disease of people over 50 and has no approved treatment. Finding therapies for rare disease flows from our desire to help patients underserved by today’s medicines.
Rare Diseases Beyond the Numbers
Novartis Institutes for BioMedical Research created this interactive quiz, hoping that testing people’s knowledge of rare diseases would help them understand the great need for new treatment options for patients with rare diseases.
Toward precision medicine for a rare blood cancer
Novartis has joined forces with lymphoma experts in academia to hack into the genetics of a rare form of blood cancer known as mantle cell lymphoma (MCL). Working with lymphoma experts from Charité – Universitätsmedizin in Berlin and the BC Cancer Research Centre in Vancouver, Canada, Novartis scientists provide evidence of two related but mutually exclusive molecular signaling pathways at play in MCL cell lines.
A rare disease, a challenging clinical trial
Cushing’s is a rare disease that affects only about 10 to 15 people per million. It is difficult to diagnose and can make clinical trials for such a small patient population very challenging. But finding treatments for patients with few options is at the heart of our approach. Learn more about one trial and the scientists and patients who made it work.
Letters To My Younger Self - Renee Bergeron
“I was always planning and very deliberate. I look back now and wish I hadn’t been so deliberate…” Renee Bergeron writes to herself at the age of 38, on her first day back in the office following the birth of her first daughter.