Category Archives: Treatments and Therapies

Treatments Approved For Rare Diseases

Ten new treatments for rare diseases have been approved in Europe.  Among those approved are two hybrid medicines:  Aripiprazole which treats schizophrenia and manic episodes in Bipolar 1 disorder, and idebenone which treats visual impairments in patients suffering from Leber’s hereditary optic neuropathy.

Other drugs that were improved include docetaxel for breast cancer, non-small cell lung cancer, prostate cancer, and head and neck cancer; duloxetine for major depressive disorder, and generalized anxiety disorder; and pregabalin for epilepsy as well as generalized anxiety disorder.

The Committee for Medicinal Products for Human Use requested further data be collected and submitted on other drugs and therapies that were presented to the CHMP but that were not authorized.

Hope For Those Suffering From RRMS

According to a new study published in the 25 June 2015 edition of the “Journal of Neurology, Neurosurgery and Psychiatry,” patients suffering from relapsing-remitting Multiple Sclerosis benefit from the long-term fingolimod therapy by Novartis.

Fingolimod is an immunomodulating drug and has been determined to be an effective therapy for RRMS.  The study was extensive, lasting up to 4.5 years, and the findings were that there was a 50 percent reduction in annualized relapse rate and a reduced MRI disease activity among patients treated with fingolimod.

It is estimated that globally 2.3 million people suffer from MS, with 400,000 in the U.S. alone.  Relapsing-remitting MS is the most common form of the disease.

Epilepsy Genetic Initiative Kicks Off

The Epilepsy Genetic Initiative has established a project in partnership with the Citizens United for Research in Epilepsy (CURE) and the National Institute of Neurological Disorders and Stroke, that will research the causes of epilepsy which affect three million Americans.  The project is being sponsored by the John and Barbara Vogelstein Foundation.

The project hopes to create a centralized database with the genetic data of patients diagnosed with epilepsy.  The data will be used to assist patients, clinicians, and researchers in advancing how epilepsy is treated as well as identifying the genes that are associated with epilepsy.

Currently, the cause of epilepsy in nearly 70 percent of individuals with epilepsy is unknown.  What’s more one in three patients do not respond to traditional treatments for epilepsy.

CURE Launches Epilepsy Genetics Initiative, First Foray Into Personalized Medicine

A New Drug To Treat LAM

A new drug has been approved by the U.S. Food and Drug Adminstration to treat lymphangioleiomyomatosis.

Lymphangioleiomyomatosis (LAM) is a rare, progressive lung disease that blocks normal airflow in the lungs, and interferes with oxygen being delivered to the rest of the body.  It affects 5 women in a million according to the FDA.

Originally, Rapamune was approved to help prevent organ rejection for patients aged 13 and older who underwent a kidney transplant.

Clinical trials showed that the drug was effective in treating LAM, and that discontinuation of use of the drug in patients with LAM resulted in a decline in lung function that was similar to patients in the placebo group.

More information can be found on the Food and Drug Administration website.