The reference centre for rare or early onset dementias at the Pitié-Salpêtrière hospital (responsible Pr Bruno DUBOIS) www.cref-demrare.fr devotes part of its activity to progressive supranuclear paralysis. Dr. Isabelle LE BER, a neurologist and researcher at the Brain Institute, coordinates the national network of expert centres (centres of reference and competence) on PSP. These networks bring together a large number of cases of PPH, providing both more data to advance PPH research, as well as harmonizing diagnostic practices and providing more capacity to improve management at the national level. The expertise developed within the reference centre and competence centres can thus benefit as many patients as possible.
The treatments of progressive supranuclear paralysis proposed today are said to be symptomatic because they allow to decrease or even to suppress the clinical signs but do not stop the progress of the disease.
The average patient survival to date is 6 to 8 years. Deaths occur either by false routes due to progressive difficulty swallowing or by trauma from a fall. Poor prognosis factors include the patient’s age, frequent falls early in the illness, and swallowing disorders.
However, multidisciplinary care can improve certain symptoms and provide aids for daily living. This management is tailored to the clinical presentation and situation of each patient. Kinesiotherapy and speech therapy are important for managing motor and speech disorders, cognitive functions and, if necessary, swallowing. Counselling is offered to support the patient and those around him. Social care is also important in order to provide, where necessary, human or financial support. Patients and their families are also accompanied by patient associations (http://www.pspfrance.org)
At the Paris Brain Institute
A study of transcranial stimulation stimulating certain neural networks affected by PSP showed significant effects on cognitive abilities, including language, which was greatly reduced in some patients. This “double-blind versus placebo” study, conducted by the FRONTLAB team of the Institut du Cerveau, the Institut de la Mémoire and the Centre de Référence National (Department of Neurology, Pitié-Salpêtrière) showed a significant improvement in the language skills of the treated patients. They find the words and their meanings more easily, which leads to the conclusion that
of this therapy for patients. Trials using iterative stimulation sessions in a large group of patients to validate its therapeutic efficacy in PSP are underway.
In late 2019, ANSM (Agence Nationale de Sécurité du Médicament et des produits de santé) authorized the launch of a phase 2a clinical trial with the drug candidate AZP2006 in the indication of Progressive Supranuclear Paralysis (PSP).
This treatment is based on a small molecule, developed by ALZPROTECT, that decreases the phosphorylation of the tau protein – the mechanism that causes the pathological form of the protein. This therapeutic trial is currently in progress at the Hospital of La Pitié Salpêtrière in Paris (AP-HP), under the direction of Professor Jean-Christophe CORVOL and at the CHU in Lille, in the service of Professor DEFEBVRE. The objectives of this clinical trial are to verify the tolerability of the product in patients, to strengthen the pharmacokinetic data of the product after 3 months of treatment, but also to evaluate the impact of the treatment on markers of the disease (more than 20 markers). Phase 2a should be completed in 2021 before first results are published in late 2021, early 2022.
“The start of this phase 2a study is an opportunity for patients with PSP, a rare disease for which no treatment is currently able to halt progression,” says Pr. Jean-Christophe CORVOL, Professor in Neuropharmacology, Head of the Centre de Recherche Clinique en Neurosciences at the Pitié-Salpêtrière and co-leader of the research team at the Institut du Cerveau.