Biotechnology plays a crucial role in the latest advances against Alzheimer's
- Nearly two decades ago, the first group of compounds to treat Alzheimer's disease were approved, but they do not stop the progression of the neurodegenerative process nor restore the neuronal damage it has caused.
- Despite the efforts and enormous resources dedicated to the field of Alzheimer's disease, there is currently no approved treatment that cures this condition.
- Biotechnology is behind every medication being tested and eventually approved, making its contribution the key factor that has enabled improvements in treatments in recent years.

It is estimated that more than 900,000 people in Spain suffer from dementia, with Alzheimer's being the most common cause, responsible for up to 70% of cases. This is one of the most prevalent diseases among people over the age of 65 (dementia affects one in ten people over 65, and one-third of those over 80). In a context where life expectancy continues to rise and we still do not have a cure for Alzheimer's disease, these figures could increase drastically in the short to medium term.
Biotechnology is behind every single medication that is being tested and eventually approved, making its contribution the key to the improvement in treatments in recent years. It is biotechnology that will enable, through the study and deep understanding of the molecular foundations of the disease, the development of increasingly effective compounds to combat it. For this reason, on September 21st, in celebration of World Alzheimer's Day, we analyze some of the advances being made thanks to the research carried out by some of AseBio's partner companies. Without basic research, translational and clinical research, as well as the chemical synthesis stage of compounds, all encompassed within biotechnology, the invention and development of these molecules would not be possible.
Despite the efforts and enormous resources dedicated to the field of Alzheimer's disease, there is currently no approved treatment that cures this condition. Nearly two decades ago, the first group of compounds to treat Alzheimer's disease were approved. Among them are the so-called "cholinesterase inhibitors" (Donepezil, Rivastigmine, Galantamine, and Tacrine) and "NMDA receptor antagonists" (Memantine). “While it is true that in mild cases, these drugs can improve cognitive function and help patients perform daily activities with some normalcy, unfortunately, they do not stop the progression of the neurodegenerative process or restore the neuronal damage it has caused, and they come with an 85% risk of adverse effects,” note representatives from Laminar Pharmaceuticals, a biopharmaceutical company committed to translating health research.
Laminar Pharma explains that in recent years, drugs have been developed and approved to combat key neuropathological markers of the disease, such as the accumulation of beta-amyloid plaques and tau protein tangles in the brain. This is the case with the monoclonal antibody Aducanumab, approved by the U.S. Food and Drug Administration (FDA) in 2021, the first drug aimed at reducing beta-amyloid plaques. Although its approval was controversial, it marked a milestone in Alzheimer's therapy. Lecanemab, another monoclonal antibody, was approved by the FDA in 2023. It targets beta-amyloid plaques and has been shown to be effective in clinical trials in slowing the progression of the disease. “However, the efficacy of these drugs is still far from what is desired today. Advances in immunotherapy may eventually lead to an effective therapy to combat this devastating disease in the future. Nonetheless, the low efficacy of the compounds approved to date highlights the need to seek new therapies that address the problem from a different perspective and not focus solely on the classical hypotheses,” they conclude.
LAM226 has shown extensive neuroprotective effects against Alzheimer's disease in animal and cellular models
In this context, we find LAM226, a hydroxylated derivative of DHA, which is based on a novel therapy focused on modifying the lipid composition of the cell membrane. This approach, known as membrane lipid therapy or melitherapy, was developed by Laminar Pharmaceuticals. Melitherapy aims to develop compounds that regulate the structure, function, and organization of membrane lipids involved in cellular signaling.
They explain that the structure and composition of the membrane control the translocation of signaling proteins from the cytoplasm to the plasma membrane and vice versa. As a result, the membrane functions as a regulator of the activation or inhibition of numerous signaling pathways relevant to various cellular processes. Attempting to normalize the lipid membrane to induce changes in the different proteins associated with it represents a paradigm shift in drug discovery, which could lead to the development of a new class of therapies with an exceptional balance of safety and efficacy for serious diseases with unmet medical needs.
LAM226 has demonstrated extensive neuroprotective effects against Alzheimer's disease in both animal and cellular models, while regulatory toxicology studies have revealed an excellent safety profile in rodents. Chronic oral administration of LAM226 for four months to 5xFAD mice, a transgenic model of Alzheimer's disease, prevents cognitive decline as well as synaptic and neuronal degeneration. Treatment with LAM226 also improved the behavior of the mice in a radial maze task used to measure cognitive rehabilitation. This neuroprotection is also mediated by the restoration of neuronal proliferation to healthy levels in the hippocampus. "At the molecular level, LAM226 reduces both β-amyloid accumulation and tau protein phosphorylation compared to untreated 5xFAD controls. LAM226 is a potent inhibitor of tau phosphorylation (both in vitro and in vivo), as shown in SH-SY5Y cells differentiated to a neuronal phenotype and in brain samples from 5xFAD transgenic mice with familial Alzheimer's disease," they add.
Additionally, in vitro studies demonstrated LAM226's ability to prevent cell death caused by exposure to neurotoxic stimuli such as the beta-amyloid peptide or NMDA in differentiated neuron cultures. Treatment with LAM226 also promoted the recovery of healthy brain markers and the restoration of synaptic protein expression (synaptophysin and SNAP25) in the hippocampus.
"With these promising results, we are excited and eager to invest more resources in this compound once LAM561, our most advanced compound for glioblastoma treatment, is approved," they celebrate. They are currently completing all the required preclinical experiments in cells and animals to begin human trials. They expect to start Phase 1/2a of this compound by 2025.
ADmit Therapeutics and its early diagnostic test for Alzheimer's disease
ADmit Therapeutics is a company dedicated to the study of Alzheimer's disease and other neurodegenerative processes with a strong social mission. It offers a completely new and disruptive approach to understanding the progression of Alzheimer's disease by combining the analysis of susceptibility factors and biological risk with clinical biomarkers, and molecular analysis through Next Generation Sequencing (NGS) of biomarkers related to the epigenetic regulation of mitochondrial DNA.
The mitochondrion is the epicenter of all energy regulation in the cell, and its involvement as a key element in the development of Alzheimer's disease was first proposed 20 years ago by Professor Russell Swerdlow in the "Mitochondrial Cascade" theory.
This approach allows ADmit Therapeutics to bypass the etiological heterogeneity of Alzheimer's by not focusing on protein biomarkers involved in other neurodegenerative processes similar to Alzheimer's disease. It also enables the prediction of progression to Alzheimer's dementia up to 14 years in advance in patients diagnosed with Mild Cognitive Impairment (MCI), "even in individuals who have tested negative for β-amyloid on a PET scan, with excellent accuracy, sensitivity, and specificity," they assert.
"Starting with a blood sample, ADmit Therapeutics makes predictions in patients with MCI (CDR = 0.5) based on age data, ApoE genotype, and mitochondrial DNA methylation levels via NGS. This data is processed using a classifier model generated by Machine Learning techniques. In this way, ADmit Therapeutics provides a cost-effective, minimally invasive blood test (without the need for PET scan data or cerebrospinal fluid obtained via lumbar puncture)," the company shares.
ADmit Therapeutics offers an extraordinary tool for healthcare systems and pharmaceutical companies in the triage and stratification of patients for treatments and clinical trials. It also changes the social impact of this disease on patients and families, offering the possibility of improving their quality of life with personalized treatment strategies. By providing clarity and direction, it allows for better planning and enhanced well-being for those affected, while also fostering a deeper understanding of the disease by reducing stigma and promoting a culture of empathy and support for patients and their families.
Ángel Luis Jiménez
Communication Director
662 172 126
ajimenez@asebio.com
AseBio brings together more than 300 entities and represents the Spanish biotechnology sector as a whole. Its mission is to lead the transformation of the country, positioning science, innovation and especially biotechnology as an engine of economic growth and social welfare. Its members include companies, associations, foundations, universities, technology and research centers that develop their activities directly or indirectly related to biotechnology in Spain.