Therapeutic perspectives

A.I.G.S.S. Association



Therapeutic perspectives

Prion diseases are incurable and rapidly fatal neurodegenerative diseases. Very little has been done in the past 30 years. However, something is finally changing. We now expose two drugs for PRNP positive but asymptomatic patients.
Our hope is that they will soon be subjected to further studies and administered in "science and consciousness". In Europe for over 10 years a drug has been administered the active substance is doxycycline, an antibiotic.

Unfortunately, it was a failure, although it has been text tested on patients for many years. Regarding the treatment in question, we wish to make known to those who follow us, the opinion of the greatest researcher currently active in the United States, ERIC MINIKEL.

However, times have changed and although science has long times there is something new, we start from potential drugs for patients who are positive for DNA testing but asymptomatic:

Since prion protein (PrP) is necessary for prion replication but is superfluous for the host, the PrPFRET- enabled high-performance test (PrP-FEHTA) was developed to select compounds that reduce the expression of PrP.
A collection of drugs approved for human use and identified astemizole, which reduced cell surface PrP and inhibited prion replication in neuroblastoma cells, were examined. Astemizole stimulated autophagy, prolonged the survival time of prion-infected mice.

Astemizole is used in humans to treat seasonal allergic rhinitis in a chronic environment. Given the absence of any therapeutic option for patients with CJD, GSS and FFI and the favorable pharmacological characteristics of astemizole, including its ability to cross the blood brain barrier, it can be considered as therapy for prophylactic use in family prion diseases.


A few years ago, a US neurologist together with a staff of researchers identified two substances for the treatment of Creutzfeldt-Jakob disease. It was a tricyclic antidepressant and an antipsychotic drug. We are talking about trimipramine and flufenazine (belonging to the antipsychotic drugs classified as phenothiazines). However, the study, which had given good results in mice, did not receive the approval of the ethics committee of the Langone clinic in New York. Years later, after having passed through a large number of abtracts on potential treatments for prion diseases, we found the experiment, in its entirety. We contacted the neurologist in question, Prof. Thomas Wisniewski, trying to understand if trimipramine could be a good solution in patients who are positive for the PRPN gene but asymptomatic. The answer was that by not having tested it on humans he had no certainties.

However, underlining the fact that on mice it had given positive results, one could try using 50 mg. per day of trimipramine. This old antidepressant sold in the pharmacy with the name of SURMONTIL is indicated above all in the treatment of depression accompanied by insomnia, agitation, anxiety, and in various types of neurosis (anxiety, hysterical, phobic, obsessive). It has a remarkable efficacy against panic attacks and is often used in low doses to treat primary insomnia. We believe that in the absence of valid anti-prion substances, this is a viable way. We will strive for this substance to be further studied and, if possible, administered in "science and consciousness").


The MRC Prion Unit at UCL has conducted long-term research to investigate potential antibody treatments for prion disease and to see if such treatments might work and what side effects or other safety issues might be anticipated.
The immune system recognises foreign proteins and other parts of germs as being alien to the body and this leads to the body producing specific antibodies tailored to fight that infection. However, since prions are formed from one of the body’s own proteins, they are not recognised in the same way by the immune system and lifesaving antibodies are not produced. This is one of the reasons why prions are so lethal. The team at the MRC Prion Unit at UCL have developed a pioneering treatment, PRN100.

PRN100 is an antibody specifically designed to bind tightly to the normal prion protein with the aim of preventing it from combining with the prions and in this way stop a chain reaction and formation of new prions.PRN100 and related antibodies have potent activity in binding to prion protein and preventing prion disease in laboratory models. You should know that our Association will donate at least 85% in donations to the MRC - London.

Professor John Collinge and Dr. Simon Mead are demonstrating that PRN100, when administered in good time (when the patient is still responding) is able to stop CJD disease. We will fight for it to be given to Gerstmann Straussler Scheinker patients as well.


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Italian Association against the disease of GERSTMANN STRAUSSLER SCHEINKER

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