People with Tuberous Sclerosis Complex May Have New Hope

Every year, actually every day, the FDA and similar organizations for other countries approve new medicines. Innumerable pharmaceutical companies work tirelessly for years to research, develop, test, distribute, and market these new medications. Fortunately for all of us, they do.

Recently, a new treatment option for tuberous sclerosis complex (TSC) and treatment-resistant epilepsy went through phase 3 clinical trials, which means it may be available on the market some time soon.

Tuberous sclerosis complex is a genetic disorder where noncancerous tumors grow in different parts of the body. Most people with TSC have some sort of abnormal skin condition. Tumors also frequently affect the brain and kidneys, causing seizures or severe problems with the renal system. Less common are tumors growing on the heart and lungs.

Epilepsy is a relatively common neurological disorder that manifests as seizures in those affected. Most people with this disorder can live essentially normal lives with the proper medication. However, certain versions of epilepsy are resistant to treatment. The unpredictability of epilepsy can cause problems in all facets of a person’s life.

The pharmaceutical company Novartis is developing the drug in question, Everolimus. In the study that wrapped up near the end of 2016, more than 350 people, aged two to 65 from 25 countries, were either given a low dose, a high dose, or a placebo.

Low dose recipients saw a modest (29 percent) reduction in the number of seizures. Those who received the high dose saw a significant (40 percent) reduction. All groups, including the control, experienced some degree of side effects.

Authors of the article, published in The Lancet, are hopeful that this new medication can help the estimated one million TSC sufferers as well as the millions who have epilepsy. Let’s hope they are correct and the results are as encouraging once the drug has been approved.

Click here to read an article about the article.

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