Gene therapy center takes big step against Parkinson's disease

Gene therapy center takes big step against Parkinson's disease

In San Sebastia (Euscade) there is the first large gene therapy center not only in Spain, but also in Europe. Gene therapies consist of modifying a cell's genes. Gene therapies for diseases such as Parkinson's and heart failure are already being manufactured at production sites such as the one in San Sebastia, which belongs to the pharmaceutical company Bayer. But treatment will also be sought for other conditions such as multiple system atrophy, Huntington's disease, Pompe disease, or waist muscular dystrophy type 2 I/R9. “It's almost magical,” some experts say.

“Gene therapy is an experimental form of treatment that uses the transfer of genes into a patient's cell to treat a disease. The idea is to modify the genetic information in the patient's cell responsible for the disease,” notes Gustavo Peskin, CEO. From AskBio. AskBio, a pioneering gene therapy company acquired by Bayer in 2020, has founded other companies such as Viralgen (with a manufacturing center in San Sebastian) and TAAV to advance these treatments. All three companies (Viralgen, AskBio, and TAAV) are affiliated with Bayer.

From San Sebastian to the world

Potential treatments for neurodegenerative diseases such as Parkinson's are being manufactured at a center like the one in San Sebastia. “We are in a phase 2 study for Parkinson's patients who have exhausted all available treatments,” explains Peskin. What treatment is being investigated in this trial? “It is a surgical procedure through which gene therapy is introduced into the belly of the brain. It is a neuroprotective treatment: it seeks to stimulate the neurons in the brain that work to protect it, and they are specifically the neurons that are lost in diseases such as Parkinson’s disease,” he adds. .

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This treatment, if proven effective, would treat not only classic Parkinson's disease, but also infantile Parkinson's disease, also called AADC deficiency. Parkinson's disease in children is a disease with a “very low prevalence”: worldwide there are about 200 children diagnosed with this disease. These are children who suffer from tremors and muscle stiffness, who have problems maintaining balance and have great difficulty speaking or walking.

In adults, the incidence of Parkinson's disease is 8 to 18 patients per 100,000 population. It must be said that gene therapy has proven effective in treating this disease.

He explains: “The surgery consists of placing two ceramic cannulas on either side of the brain, through which the gene therapy is infused. The goal is not to touch the blood vessels and to achieve 60% coverage of the putamen. This surgery takes about eight hours.” Bisquin. The goal, he insists, is to stimulate neurons that slow the process of neurodegeneration. “We know there is an inherent complexity to all of this, and we believe it will be reduced over time,” says AskBio's CEO. “Gene therapies for diseases like Parkinson's will be created for patients who have already exhausted other approaches.”

In the company's phase 1 study, 11 patients were treated: five in primary and six in intermediate. “With Parkinson's disease, everyone, whether children or adults, ends up in a neurodegenerative process. Some go faster than others. It is a vicious circle: in order for the patient to control the symptoms, the doses of treatment must be increased and their state of health improves.” “The existence is degrading,” Pesquin points out. In the case of Parkinson's disease, experts focus on the so-called LEDD factor: the gene therapy developed by this pharmaceutical company now seeks not only to prove its effectiveness, but also to reduce the LEDD factor.

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“Our challenge is to manufacture this type of treatment. In the beginning, all the processes are very academic and literal, but we have to understand the process well to do all this on a large scale,” notes Jimmy Vanhoof for his part. , CEO of Viralgen, founded in 2017. This is why it stands out that a gene therapy production plant, such as the one in San Sebastia, which was established in 2021, is the first of its kind on a large scale in Spain and Europe as a whole.

In Spain there are other centers, such as the Clínic de Barcelona General Hospital, that themselves produce CAR-T immunotherapies (a type of cell therapy that is very effective in treating cancer), but their manufacturing is academic and targets specific patients. “The impact that gene and cell therapies have on some people's lives is almost magical,” says Bayer's Ruth Shah.

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