AMPEL BioSolutions Identifies Novel Lupus Treatments and Targets
Virginia researchers and founders of AMPEL BioSolutions, Drs. Amrie Grammer & Peter Lipsky announced the results of a year-long Lupus Drug Repositioning Initiative this week in Boston. The Alliance for Lupus Research (ALR), the Lupus Research Institute (LRI) & AMPEL gathered with scientists at a key meeting of the American College of Rheumatology to launch the Lupus Clinical Investigators Network (LuCIN) of the LRxL-STAT Lupus Drug Repositioning Initiative. LRxL-STAT evaluates existing FDA approved synthetic and natural compounds originally targeted at other diseases for new usage by lupus patients. Lupus is an autoimmune disorder that causes symptoms very similar to other pathologies, allowing drugs to be repositioned. Today, LRxL-STAT is excited to announce the clinical testing of naturally-derived Krill oil treatment, Janssen’s Stelara (originally used for psoriasis), and Kadmon’s KD025 (also originally a psoriasis treatment). In addition, trials of Quinacrine (a well established drug with broad medical applications) and stem cell transplantation are being considered. LuCIN, which is comprised of over 50 academic lupus centers representing all geographic regions of the US, will conduct clinical trials in close consultation with lupus patient groups, with international efforts beginning in early 2015.
A hallmark of Lupus is an over reactive immune system that attacks almost any organ system of the patient’s own body.
Today’s breakthrough announcement comes only a year after the ALR & LRI announced their trailblazing cooperative project to speed up the identification & availability of new treatments for lupus patients. The need is urgent since GSK’s Benlysta is the only treatment approved by the FDA in fifty years and standard-of-care treatments have serious side effects. Drug Repositioning is important, since it can be used to quickly bring treatments to patients whose side effects and mechanism of action are well-characterized. Typical drug development takes about 10-15 years and 1.5 billion dollars. ALR & LRI’s Lupus Drug Repositioning Initiative began with community suggestions for potential treatments in conjunction with a diligent and thorough search of the world’s literature for appropriate drugs by AMPEL BioSolutions. Drs. Grammer & Lipsky performed evidence-based analysis on each potential lupus therapy using the CoLT, or Combined Lupus Treatment, scoring system that incorporates rationale, pre-clinical/clinical experience, drug properties and safety profile. Proof-of-concept trials conducted by LuCIN will be small, focused and biomarker-rich. The first lupus drug repositioning trials will begin by the second quarter of 2015.
Earlier this year, Dr. Lipsky coordinated a scientific study published in Science Translational Medicine (STM) that focused international attention on a novel cellular pathway that could be targeted by pharmaceutical treatment. Dr. Lipsky’s July 23rd article was highlighted on the cover of STM with a photograph of a growling wolf, a long-time symbol of this complex disease.
Drugs are repositioned from their FDA-approved indications into diseases with related pathogenic mechanisms. AMPEL is at the forefront of identification of drug candidates for drug repositioning.
A hallmark of Lupus is an over reactive immune system that attacks almost any organ system of the patient’s own body. Most people have antibodies circulating in their blood that protect them from infection (i.e. after vaccination). Lupus patients have circulating antibodies that attack everything from their kidneys to their brain to their skin. Dr. Lipsky & his Beijing team found that Lupus patients have abnormally low levels of a cellular switch called PTEN, which at normal levels puts harmful, self-attacking components of the immune system to sleep. Lupus patients with low levels of PTEN exhibit a more severe form of the disease as these harmful components are left unrestricted. This discovery is an important step towards control of pathways which lead to self-attack by the immune system.
AMPEL Biosolutions is a proud member of the Virginia biotech community. Following over 20 years of collaboration in Lupus research including time at the NIH, Drs. Amrie Grammer & Peter Lipsky co-founded AMPEL as a local biomedical consulting firm now located in the UVA Research Park. AMPEL is creating partnerships that connect drug companies, doctors, scientists and bioinformaticists to develop individualized treatment regimens for Lupus patients. The principals of AMPEL are deeply involved in the Charlottesville community, most recently co-sponsoring a symposium, “Creativity in the Academic & Corporate Worlds”, last summer at UVA in conjunction with the UVA Chemistry Department, Alpha Chi Sigma & the Graduate Students and Beyond Club at UVA.
- Alliance for Lupus Research (www.lupusresearch.org)
- Lupus Research Institute (www.lupusresearchinstitute.org)
- AMPEL BioSolutions (www.ampelbiosolutions.org)
- LRxL-STAT (www.linkedin.com/in/lrxlstat)
- LuCIN (www.linkedin.com/in/lucinstat)