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New treatments available for Narcolepsy

Our clinic currently monitors around 150 patients with some kind of Central Hypersomnia. All know how hard it was to reach the diagnosis. Often this was after many years of illness and many visits to different specialists, where they were labelled with other illnesses, often depression, epilepsy, psychosis, sleep apnea, etc.

Our first step is usually to withdraw accompanying medication, which occasionally alters the medical chart, and other times simply hides the symptoms and makes a precise diagnosis impossible.

The benefit of doing this, when the correct diagnosis is reached, is that it opens a new, correct path towards controlling symptoms.

Since we have known the physiopathological bases of these illnesses, particularly Narcolepsy, new therapies have been implemented, and currently the scope for controlling symptoms is infinitely better than 15 years ago.

Effective pharmaceuticals against narcolepsy

One of the keys has been the introduction of Sodium Oxybate, a pharmaceutical that has been shown to have an effect on the symptoms of type I Narcolepsy, unknown until its introduction, specifically on cataplexy.

However, the traditional therapy continues to develop with promising new therapies.

Firstly, the appearance of Pitolisant. It is a pharmaceutical that acts on a completely different neurological channel to other medications that we have had until now: the central histaminergic route.

For several decades we have known the importance of this amine in the maintenance of wakefulness and its interaction with other essential arousal centres. The French laboratory Bioprojet has managed to progress all the basic and clinical studies and is prepared for commercialisation in Spain.

Its effect on drowsiness, and what is really interesting, also on cataplexy, has been shown in numerous clinical studies (in which we have participated), all with a very adequate safety profile.

The pharmaceutical is not yet authorised for commercialisation in Spain, but in the meantime, the Ministry of Health allows us to recruit patients into the programme for Compassionate Use of the pharmaceutical at a national level, which our Hospital coordinates with the support of the laboratory that provides us with the pharmaceutical, at zero cost to patients or the institution. It is a genuine opportunity for the many cases in which the available treatments are insufficient. Bioprojet has recently communicated the approval for use with patients under 18 years of age, in the case of them being particularly affected.

We have also begun an experimental study, which will be another great improvement for patients. The American laboratory Jazz Pharmaceuticals has formulated Oxybate, mentioned above, but without sodium, which has been causing us a lot of problems, especially with patients who are hypertensive or have cardiovascular risk.

Some have not been able to benefit from this treatment precisely due to this problem, which is resolved in the new formulation. We are now recruiting patients for this trial, with the advantage of the design of the study leaving no patient unaware of the effect of the treatment. In a few years we will have commercialised it, and everything will be easier.

We also have another trial with Oxybate, with the advantage of a new form of administration (a single dose), and we are analysing newly emerging treatments, such as the intranasal hypocretin and other therapies in the first stages of the illness, if we are able to diagnose them quickly.

In summary, the treatments for central hypersomnia are finally advancing, and we can predict a better symptomatic monitoring of patients each day. We await the day when we can truly cure the illness.

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