I have the pleasure to have with me Maria Teresa Pellecchia, Associate Professor from The University of Saleno. Welcome, and thank you very much for your time.
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[00:00:35] Dr. de Pablo-Fernández: So I would like to start with the highlights of research in NSA for the last year or couple of years. One of the main issues is the clinical diagnostic criteria where you were involved as part of the members of the MSA MDS task force.
But what would be the highlights of research over the last couple of years or so?
[00:00:56] Dr. Pellecchia: Yeah, surely the, the new criteria have been very important and we published a few months ago and now we are projecting. Validating this criteria by means of a clinical prospective validation study that will involve many centers with clinical expertise in MSA.
And it will be important not only to validate the clinical features, but also the biomarkers, both MRI biomarkers and not-MRI biomarkers that have been included as supportive features for the new criteria. And we hope that we have great collaboration from many centers in order to perform this study.
Another important I like is the revision of the UPDRS scale that is underway, because we have the need to. have A reliable scale in order to be used for the clinical trials that are coming, there are, many drugs in the pipeline and the problem may be also that we have a not, very, optimal, clinical skill and so we are, planning to review the skill in order to have Both a more comprehensive scale for clinical use, but also an scale to monitor disease progression and to assess if disease progression is modified by the new drugs that are going to be used in clinical trials.
[00:02:31] Dr. de Pablo-Fernández: One of the things you mentioned in the clinical criteria, for example, is that biomarkers, it was felt at the time that the criteria were developed that it was still in progress, that they were not included in the diagnostic criteria, but they are very promising results on several of these biomarkers.
Would you like to expand on that?
[00:02:52] Dr. Pellecchia: Yes. In fact, we have now a new category of prodromal and possible MSA and this category biomarkers,
need To be further explored in order to identify patients in the early prodromal phase, also to be better treated with the new drug that can be demonstrated to be useful for the disease.
So we must show the real, accuracy of these biomarkers in predicting the diagnosis of MSA. And we need the prospective validation Over a great number of patients following that specialized sentence for MSA
[00:03:31] Dr. de Pablo-Fernández: You mentioned the tools that are needed to develop reliable clinical trials in MSA, and to evaluate these drugs with confidence. What is in their horizon, treatment wise in clinical trials?
[00:03:43] Dr. Pellecchia: there are some trials still, already ongoing and other trails that are coming. Fortunately we have, many, many drugs that are being developed. in particular we have some drugs, targeting alpha synuclein. Both organization and both antibodies for alpha synuclein and these are from different companies. And also we have some, drugs, that modify aggregation of alpha synuclein such as ADH 44. that is, currently being, experimented And, we have, other drugs such as anle138b that is expected to modify oligomerization of alpha synuclein And so we have many, many drugs in the pipeline and we are confident that some of these drugs may be effective. Another important, highlights, coming from the group meeting. Is the regional trial that has been conducted in German it was a trial, that was conducted only in German and the results have been submitted.
And, we have some, idea that the results may be positive, that we are waiting for these, paper that treated patients with very high dose of, we know that coins is Quan, is involved in some, patients with familial MSA, but, coins Quan is also reduced in plasma safe from when we see patients not only in Japan, but also in some papers from other So We need to confirm these data also
[00:05:28] Dr. de Pablo-Fernández: Another area that is a hot topic is studies exploring the seeding properties of alpha synuclein and how that can be implemented for the diagnosis of not just multiple system atrophy, but it can be useful as well to distinguish this condition from other alphasyneucleinopathies.
This is a very active area of research. So where do you think things will go in the future?
[00:05:51] Dr. Pellecchia: These kind of biomarkers would deserve improvement and larger application. because they are actually used in a few centers because they require lab expertise that is not very available in the most centers.
Moreover, we need to confirm that this biomarker is really, effective in differential diagnosis and also eventually to monitor disease progression. This is another biomarker that is, included among. Biomarkers for possible prodromal MSA that we aim to study further in the next few years,
[00:06:31] Dr. de Pablo-Fernández: It seems like the task force is quite busy.
[00:06:33] Dr. Pellecchia: Yes. We are very excited for this because we have a lot of projects and we need the other centers and other people interested to collaborate to be more rapid in our advancement.
[00:06:46] Dr. de Pablo-Fernández: Definitely. It's crucial to have an international network to develop things in these sort of rare conditions.
Well exciting times ahead for multiple system atrophy, and I hope that we have good news to share in the next MDS Congress in Copenhagen. Thank you very much, Maria.
[00:07:03] Dr. Pellecchia: Thank you.