Stopping Parkinson’s disease with a brain injection. It sounds like science fiction, but that’s exactly what a clinical trial using gene therapy for patients with moderate disease is currently investigating, with excellent results, and which has just begun Phase II .
The basis on which this research is being developed is based on the fact that “when biopsies were taken from people who died from this advanced disease, they found that GDNF (a human glial cell line-derived neurotrophic factor transgene) had disappeared in 75% of the dopamine-producing neurons. This is where the thesis comes from that GDNF as a growth factor plays an important role in the reproduction of these dopaminergic neurons ,” Gustavo Pesquin, CEO of AskBio, a subsidiary company owned by Bayer, explains to A TU SALUD.
The treatment, ” highly complex but minimally invasive “—just a cannula in the brain—seeks to place functioning copies of GDNF in the hope that this will help, like a “fertilizer,” to proliferate these dopaminergic neurons. ” There needs to be at least 30% of these neurons remaining before they need to be fertilized ,” Pesquin points out, in order to restore their function.
Having just begun this new large-scale placebo-controlled phase, the preliminary data are more than encouraging: ” In phase 1b, with six patients in the moderate stage who had been living with the disease for seven years, the results presented showed that after a year and a half of treatment, their numbers were the same as those in the mild stages at the start of the study . In other words, the disease has rewound seven years .” Furthermore, ” the big question was how long the effect would last, and what we saw in the 36-month reading is that it does persist, that the disease does not follow the same curve as at the start. We have very high hopes ,” acknowledges the CEO of AskBio.
The treatment seeks to restore the function of dopamine-producing neurons.
The trial, which has been in development for 36 months, has 87 randomized patients and they expect to have results by the end of 2027 , beginning of 2028. And, if everything goes according to plan, it could be approved by 2029. “It is so transformative that there is a possibility that the American Food Agency (FDA), with extraordinary results, the therapy deserves accelerated approval. If that happens, we must also be prepared in manufacturing, which is what we do at Viralgen (the largest gene therapy plant in Europe, located in San Sebastián and which will be in charge of producing the therapy worldwide) to have the validated commercial-grade product, and we are doing all the work in parallel,” explains Pesquin.
This doesn’t seem far-fetched, given that the results obtained so far have been so positive that, based on preliminary clinical data, the FDA granted it Regenerative Medicine Advanced Therapy (RMAT) designation for its potential to slow disease progression and improve motor outcomes in patients with moderate Parkinson’s disease.
Author: Eva, Sainz Corada
Source: msn.com
Date: April 2025.
Note: The Nutrigenomics Institute is not responsible for the opinions expressed in this article.
PHOTO FROM PIXABAY.
