First effective drug against primary multiple sclerosis

The company Genentech, belonging to Roche Group, announced on September 27 that the clinical trial, in Phase III, of the experimental drug Ocrelizumab it has been satisfactory.

This medicine succeeds in delaying the progression of primary progressive multiple sclerosis (MSM) by at least 12 weeks, in its initial stages. This subtype of multiple sclerosis (MS), which affects approximately 10-15% of the population with this disease, is a very aggressive pathology. To date there was no cure or treatment, but this multicenter study (at an international level) with Spanish participation, has evidenced the efficacy of this drug that could become the first and only therapeutic option for patients with this disease.

So far there was no treatment for EMM

The study of this drug is called Oratory It has been led by the head of the Clinical Neuroimmunology Service of the Vall d'Hebron Hospital and director of the Multiple Sclerosis Center of Catalonia (Cemcat), Xavier Montalbán. In this study, the efficacy of the drug Ocrelizumab was investigated in 732 patients with primary progressive multiple sclerosis and

the main conclusion is that it manages to stop, at least 12 weeks, the progression of the disability caused by the disease.

Montalbán wanted to celebrate the discovery and declared:

"It is a truly historic moment. It is so to the extent that it is the first time that a drug has been shown to be effective in controlling this type of neurological disease. A window opens towards a better understanding and treatment of multiple sclerosis "

This drug is a monoclonal antibody designed to selectively attack CD20B + cells that is believed the destruction of myelin and nerves, which causes the symptoms of sclerosis, play a key role multiple. By binding to the surface of these proteins, Ocrelizumab helps preserve the most important functions of the immune system.

What is Multiple Sclerosis?

The multiple sclerosis (MS) is a neuroinflammatory disease that affects the central nervous system (CNS), both the brain and the spinal cord. It is not known exactly what causes MS, but this condition damages myelin, a substance that forms the membrane that surrounds nerve fibers (axons), and that facilitates the conduction of electrical impulses between these.

Myelin is destroyed in multiple areas, sometimes leaving scars (sclerosis). These injured areas are also known as demyelination plaques. When the myelinic substance is destroyed, the ability of the nerves to conduct electrical impulses to and from the brain is interrupted, and this fact produces the appearance of symptoms such as:

• Visual disturbances
• Muscular weakness
• Problems with coordination and balance
• Sensations such as numbness, itching, or pricking
• Thinking and memory problems

Multiple sclerosis affects women more than men. Its onset usually occurs between the ages of 20 and 40, although cases have also been reported in children and the elderly. Generally, the disease is mild, but in more severe cases some people lose the ability to write, speak, or walk.

In most cases, this disease progresses in flare-ups, but in primary progressive multiple sclerosis, the disability worsens continuously and slowly over months or years, which is why it is considered a serious form of this pathology.

Phases of the clinical development of a drug

In order for a drug to be put on sale, a process must be followed to evaluate its efficacy and safety, thus avoiding putting the lives of the people who are going to consume it at risk. The development of a new drug is long and difficult, because only two or three out of 10,000 drug substances make it onto the market.

When the drug has been sufficiently evaluated in in vitro models and in animal studies (preclinical phase), research in humans begins, which is called clinical trials. Classically the period of clinical development of a pharmaceutical product is divided into 4 consecutive phases, but they can overlap. These are the phases that are part of the clinical trial:

• Phase I: This phase includes the first studies carried out in humans, the main objective of which is to measure the safety and tolerability of the compound. Given the level of risk involved, the number of volunteers is small and the duration of the phase short.
• Phase ii: The risk in this phase is moderate, and its objective is to provide preliminary information on the efficacy of the product and to establish the dose-response relationship. Hundreds of subjects are needed and this phase can last for several months or years.
• Phase III: This is the phase this medicine is in, and it is necessary to evaluate its efficacy and safety in the usual conditions of use and with respect to the therapeutic alternatives available for the indication studied. For this reason, its use in combination with other drugs is tested for several months or years, during which the degree of incidence of the desired and unwanted effects is analyzed. These are confirmatory therapeutic studies.
• Phase IV: It is carried out after the marketing of the drug to study it again in a clinical context, and to provide more information on its side effects.

Following the positive results in the Phase III clinical trial of Ocrelizumab, European authorization will be requested at the beginning of next year to be able to market this medicine. This usually takes about six months. From then on, each country will decide if it allows the sale in its territory.