How Ilios Therapeutics Is Pioneering a Novel Approach to Treating Neurodegenerative Conditions by Generating Multifunctional Molecules
The human brain, a three-pound universe of tangled neurons and electrical impulses, holds the mysteries of our consciousness, memories, and very sense of self. Yet, this magnificent organ is tragically susceptible to a class of diseases known as neurodegenerative disorders. Alzheimer's, Parkinson's, ALS – these conditions rob individuals of their cognitive abilities, motor functions, and ultimately, their lives.
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The Challenge
Alzheimer's disease, the most common form of neurodegeneration, afflicts an estimated 5.5 million Americans and is projected to balloon to nearly 14 million by 2050. It's a thief of minds, progressively stripping away memories and leaving behind a shell of the person you once knew.
The scientific understanding of Alzheimer's is constantly evolving, but one thing is clear: it's not a singular disease with a single cause. Three processes in the brain are believed to play key roles in neurodegeneration: mis-behavior of different proteins, and chronic stress factors coming from oxidative stress, and neuroinflammation. These three processes are interconnected and require simultaneous modulation.
The traditional approach to drug discovery focused on developing drugs with a single target, like clearing plaques or reducing inflammation. However, these attempts have yielded limited success. The paradigm of one-target, one-drug has fallen short and might not be enough to navigate the complexity of the disease.
Origin Story
Luca Giani, the CEO & Co-founder of Ilios Therapeutics, understands the devastation of neurodegenerative diseases all too well. Witnessing his grandfather succumb to Alzheimer's ignited a personal fire within him. This was further stoked when he was diagnosed with a brain tumor himself and faced the possibility of cognitive decline. Thankfully, surgery proved successful, but the experience left an indelible mark.
"I had a wakeup call through these personal experiences," says Giani. "I decided that I would dedicate the rest of my life to work so that as many people as possible can use their brain for as long as possible."
Giani’s background in venture creation and the biotech industry fueled his entrepreneurial spirit. He saw the need for an innovative approach to neurodegenerative disease treatments by recognizing the multi-factorial complexity of neurodegeneration head-on. This vision gave birth to Ilios Therapeutics.
Ilios’ academic co-founders include renowned professors in the field of Alzheimer’s, and current scientific efforts are advanced by a team of drug discovery experts, with experience in medicinal chemistry, neurobiology, neuropharmacology, and drug development.
Ilios is also fortunate to have been supported by renowned fellowships, such as the Harvard Blavatnik Fellowship in Life Science Entrepreneurship, the Harvard Belfer Fellowship, as well as leading accelerators such as MassBio Drive, Nucleate, and MassChallenge. These resources provide access to cutting-edge research facilities and expertise, which will accelerate Ilios' progress.
Under the Hood
Ilios isn't following the traditional, single-target approach. Instead, they're pioneering a new frontier in drug discovery: multifunctional therapeutics.
Their platform leverages naturally occurring molecules that have shown potential against neurodegenerative processes, but which, by themselves, cannot be turned into drugs because of efficacy of pharmacological limitations. Ilios is the old name of Troy, and the Ilios chemistry platform brings together naturally-occurring components into a single molecule, optimized – like the Trojan horse – to get into the brain and modulate the three key drivers of disease. This "multifunctional molecule" is designed to simultaneously target the three key culprits in Alzheimer's: protein misfolding, oxidative stress, and neuroinflammation.
"We're reverse-engineering the equation," explains Giani. "Instead of screening millions of molecules hoping for a hit, we start with elements that have already shown promise against these processes."
This approach offers several advantages. First, it's more efficient, streamlining the drug discovery process. Second, by targeting multiple factors simultaneously, Ilios aims to achieve therapeutic effects that can be translated to effective medicines for patients. A single drug with a multifunctional activity holds potential compared to a “cocktail” of different medications targeting individual factors, potentially reducing side effects, drug-drug interactions, compliance issues, and a more straight-forward regulatory pathway.
The Early Results: From Promising Molecules to Transformative Treatment
Ilios is in the preclinical stages of development, but the potential is becoming clear: the team has already built the chemistry platform that can rapidly merge naturally-occurring components into single molecules. And several of these proprietary molecules have shown multi-functionality and potential to increase potency through iterative chemistry. In the next twelve months, Giani is most excited about creating lead candidates – molecules that have successfully navigated the initial screening process. These candidates will then undergo further testing and optimization before potentially moving to clinical trials.
The road to a commercially available treatment is long and arduous, but the initial results are encouraging. Ilios' approach has the potential to revolutionize the treatment landscape for ALS, Alzheimer's, and other neurodegenerative diseases. By targeting multiple hallmarks of the disease simultaneously, Ilios' multifunctional drugs could slow or even halt disease progression. This could offer patients a chance to maintain cognitive function and independence for longer, significantly improving their quality of life.
The fight against neurodegenerative diseases is a marathon, not a sprint. But with its innovative approach and a passionate team driven by personal connection, Ilios Therapeutics is offering a beacon of hope in this labyrinthine battle. Their success has the potential to not only change the lives of millions but also pave the way for future advancements in treating other complex diseases. Imagine a world where a new generation of multifunctional drugs tackles a variety of conditions by addressing their underlying causes.
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Call for Alzheimer’s Innovation
With support from the Alzheimer’s Drug Discovery Foundation (ADDF) and Gates Ventures, we’ve launched a new global initiative created to develop a collaborative innovation community alongside leading companies, research teams, and stakeholders with a mission to accelerate progress in prevention, diagnosis, and management of Alzheimer’s disease and related dementias. Learn more and apply for an Alzheimer’s Moonshot Fellowship.
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Published: Jun 13, 2024