Pharmanews: sorafenib repurposed, siponimod approved

In an article published last week by SAGE Publishing, investigators from The Nagourney Cancer Institute and The Albert Einstein Israelite Hospital in São Paulo, Brazil, announced a new approach to identifying effective treatments for patients with advanced metastatic cancers. A drug commonly used to treat kidney and liver cancer was discovered effective for breast cancer, which may open new drug therapies to patients regardless of tumor type. The study reports the use of tridimensional-human-tumor derived organoids that were isolated directly from a patient’s drug resistant breast cancer. The drug identified, sorafenib (Nexavar®), is widely used for the treatment of kidney and liver cancers but has not been found active, used, nor approved for patients with breast cancer. The drug selection technique employed by Dr. Nagourney was developed by scientists in his laboratory in California and named EVA/PCD. It uses fresh biopsies removed directly from patients to study each cancer patient’s biology, to select from amongst dozens of drugs and combinations, those that will work the best. This is but one more example of what is known as repurposing drugs, allowing physicians to outsmart cancers by going around their defenses

Dr. Robert Nagourney, explained the circumstances of the discovery: “The finding of sorafenib activity was unexpected. Of the many targeted drugs that we tested on the cancer cells from a woman who had few options remaining, it was only sorafenib that had this effect, prompting our colleagues in São Paulo to begin therapy immediately.  When the patient with disease that had spread throughout her body had a near complete remission with only two months of oral therapy, a remission that lasted for over a year, the researchers examined possible mechanism for the response and believe that it reflects an entirely new form of cancer cell death known as “ferroptosis”. This iron-mediated process of cancer cell death is uniquely activated by sorafenib. The results could not have been more dramatic as the patient showed compete metabolic response by PET/CT only weeks later. We are witness to a new concept in cancer medicine where drugs can be identified and selected for individual patients in the laboratory regardless of the tumor type. The EVA/PCD testing platform is making it possible for the first time to make rational drug choices for cancer patients based upon each individual’s unique features”.

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The U.S. Food and Drug Administration (FDA) last week approved Mayzent (siponimod) to treat adults with relapsing forms of multiple sclerosis (MS), to include clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease. MS is a chronic, inflammatory, autoimmune disease of the central nervous system that disrupts communications between the brain and other parts of the body. Most people experience their first symptoms of MS between the ages of 20 and 40. It usually starts with a relapsing-remitting course, in which episodes of worsening (relapses) are followed by improvement (remissions). These remissions may not be complete and may leave patients with some degree of residual disability. Least 30% patients with MS experience some degree of persistent disability that gradually worsens over time. In some patients, disability may progress independent of relapses, a process termed secondary progressive multiple sclerosis (SPMS). In the first few years of this process, many patients continue to experience relapses, a phase of the disease called active SPMS. Active SPMS is one of the relapsing forms of MS, and drugs approved for the treatment of relapsing forms of MS can be used to treat active SPMS. Later, many patients with SPMS stop experiencing new relapses, but disability continues to progress, a phase called non-active SPMS

The efficacy of Mayzent was shown in a clinical trial of 1,651 patients that compared Mayzent to placebo in patients with SPMS who had evidence of disability progression in the prior two years and no relapses in the three months prior to enrollment. The primary endpoint of the study was the time to three-month confirmed progression in disability. The fraction of patients with confirmed progression of disability was statistically significantly lower in the Mayzent group than in the placebo group. Mayzent also decreased the number of relapses experienced by these patients. In the subgroup of patients with non-active SPMS, the results were not statistically significant. The most common adverse reactions reported by patients receiving Mayzent in the clinical trials included headache, high blood pressure and altered liver function enzymes. Mayzent may increase the risk of infections, macular edema, transient changes in heart rate, blood pressure and blood liver enzymes values. Women ready for pregnancy should use effective contraception during and for 10 days after stopping the drug due to the potential risk of fetal harm. People using immunosuppressive therapies must consult their physician first because there may be unintended additive immunosuppression with Mayzent. The FDA granted approval of Mayzent to Novartis Pharmaceuticals.

  • Edited by Dr. Gianfrancesco Cormaci, PhD, specialist in Clinical Biochemistry.
Informazioni su Dott. Gianfrancesco Cormaci 1365 Articoli
- Laurea in Medicina e Chirurgia nel 1998 (MD Degree in 1998) - Specialista in Biochimica Clinica nel 2002 (Clinical Biochemistry specialty in 2002) - Dottorato in Neurobiologia nel 2006 (Neurobiology PhD in 2006) - Ha soggiornato negli Stati Uniti, Baltimora (MD) come ricercatore alle dipendenze del National Institute on Drug Abuse (NIDA/NIH) e poi alla Johns Hopkins University, dal 2004 al 2008. - Dal 2009 si occupa di Medicina personalizzata. - Detentore di un brevetto sulla preparazione di prodotti gluten-free a partire da regolare farina di frumento immunologicamente neutralizzata (owner of a patent concerning the production of bakery gluten-free products, starting from regular wheat flour). - Autore di un libro riguardante la salute e l'alimentazione, con approfondimenti su come questa condizioni tutti i sistemi corporei. - Autore di articoli su informazione medica, salute e benessere sui siti web salutesicilia.com e medicomunicare.it