Miesbacher wants millions in funding to help his son

Created: 08/14/2022, 09:00

By: Christian Masengarb

Hope: Marcus Mengual's son suffers from Syngap1 syndrome: Because his brain lacks the substance that calms excited synapses, he is constantly overexcited and inhibited in his development.

There are no drugs for this rare disease.

To change that, Mengual has applied for eight million euros in EU funding to research active substances with 15 European universities.

Chances are he'll get the funds.

© Thomas Plettenberg

Because his son suffers from the rare syngap syndrome, Marcos Mengual from Miesbach is fighting for millions in funding for drug research.

His chances of success are good.

Miesbach – Marcos Mengual from Miesbach has taken a step forward in his fight for treatments for his son's rare Syngap disease: A research consortium initiated by the parental support organization he heads has good chances of EU funding in the millions.

The researchers want to use these to test active ingredients for drugs that help patients suffering from Syngap1 and similar diseases.

We spoke to Mengual about the bid and the chances of success.

More on the subject:

Because his son suffers from a rare disease: Miesbacher starts a fundraiser for research

Miesbacher wants millions in funding to help his son

Mr. Mengual, your research group is in the final round of the EU project Horizon Europe.

What exactly does that mean?

Horizon Europe is an innovation program for various areas, including health.

We are participating in a tender for rare diseases.

The EU is distributing up to eight million euros each to eight research projects.

Around 130 groups applied.

We made it into the last 24.

Now we have to work out our application in much more detail.

At the beginning of next year we will find out whether we will get the money.

24 more candidates, eight get the contract.

So are your chances about a third?

In the first round we had the full number of points according to the EU evaluation criteria.

I was told that we therefore have a very high chance of getting funds.

How does a group of parents come up with the idea of ​​applying for EU funding?

We are actually the only consortium that was set up by patients and not by the research institutes themselves. Last year, a research agency from Munich drew our attention to the call for proposals.

We asked ourselves across Europe who would like to participate with us.

In November we had 15 universities and institutes from all over Europe and a consulting agency together.

It's a very strong group full of well-known researchers doing cutting-edge research in their fields.

They will do time-lapse research for us.

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How difficult was it for your initiative to recruit top researchers for the project?

It was amazingly easy.

Usually a phone call or an email and a short conversation was enough.

Then there were many.

How do you manage that alongside work and family?

It's tedious because you have to do everything in your free time.

After a long day at work, when the children are in bed, you spend another two or three hours on the application.

But it's no use: we have to give up at the beginning of September.

Everything has to be ready by then.

If you are awarded the contract, it should not only benefit Syngap patients.

Our research consortium wants to work on a group of rare diseases in which similar signals in the brain are disrupted for genetic reasons.

If we get the nod, it will help patients with Syngap and three other rare syndromes.

According to your plan, the researchers kill several birds with one stone by testing active ingredients for drugs for several diseases at the same time.

Right.

The project lasts four years.

Ultimately, we hope that a handful of compounds will be discovered that can be tested on patients in clinical trials.

We are also looking for therapies that work without drugs, are testing gene therapy and ways that can get more active ingredients into the brain with the same dose.

So it's a complete package that covers many aspects of drug development.

How do you rate the chances of finding active ingredients?

Of course there is no guarantee.

But we expect helpful insights.

There won't be a finished drug yet, but it's an important step towards it.

In four years your son will be twelve years old.

Will the new medicines be able to cure him?

Lucas won't heal anymore because he missed too many milestones in his development.

But new drugs can improve his life and that of many children.

What if you don't win the bid?

Then the research group is still there.

We have already achieved that Syngap1 has arrived in the research community.

We have contacts and are taken seriously.

We can apply for further funding or approach foundations.

So it will continue.