Two Israeli researchers have discovered how to design molecules that could potentially treat and prevent major diseases related to aging such as Alzheimer’s and Parkinson’s.
Hebrew University of Jerusalem Professors Einav Gross and Shmuel Ben-Sasson identified a group of molecules that enable cells to repair damaged components, making it possible for those tissues to retain proper function. They are now in the process of fine-tuning the structure of a drug candidate that, if successful, could revolutionize medical care by extending people’s life spans and healthy years.
The research team, together with Yissum, the Hebrew University’s technology transfer company, established Vitalunga, a biotech startup that is leading a drug development program aiming to advance this technology to preclinical, and then clinical, studies.
Research team co-leader Ben-Sasson told The Media Line that Vitalunga hopes to finish the preclinical preparations within two years, and then launch a Phase I/II clinical trial that if successful, could gain the drug fast-track approval.
If everything goes well, he estimated that the drug will be available to patients in about five years, but he noted that this also depends on the startup attracting sufficient investments.
“Of course, it depends on how much resources we will be able to recruit,” he said. For us the path forward is clear, and we can accelerate our clinical development plan by raising sufficient funds, he said.
Ben-Sasson added that the ramifications of this discovery are enormous. In essence, Vitalunga hopes to provide a transformative platform technology in two aspects. The first is an oral, mechanism-based treatment for multiple, severe maladies such as Parkinson’s and Alzheimer’s, congestive heart failure, sarcopenia, etc. utilizing a single agent, he said.
The second is a family of agents that in the future could be used for wellness per se – preventive medicine for the general public.
“We have a lot of work to do, but we know exactly what we have to do in order to be in a position to explore this opportunity,” Ben-Sasson said.
Ben-Sasson explained the principles of his and Gross’ discovery. He noted that it is based on a paradigm shift taking place in recent years where many labs in the world realized there is a unified pathogenic mechanism behind the development of multiple aging-associated diseases.
He explained that in every normal cell there is a perpetual erosion of the mitochondria, the powerhouse of the cell, due to its excessive activity. To ensure fresh, fully functional mitochondria in face of this wear and tear, a meticulous quality-control cellular mechanism identifies defective mitochondria and removes them through a process called mitophagy. The removed mitochondria are then replaced by new ones.
Impaired mitophagy is now recognized as a primary pathogenic event underlying diverse aging-associated diseases such as Alzheimer’s and Parkinson’s, congestive heart failure, sarcopenia, and more, that are related to tissues that do not regenerate or multiply such as the neurons, heart, and skeletal muscle, Ben-Sasson said.
Ben-Sasson and Gross initiated a bold and creative drug-design program that resulted in the identification of a novel family of mitophagy activating compounds that are able to rejuvenate cells.
The compounds are user-friendly and can be taken orally, Ben-Sasson said in a press release issued by the Hebrew University.
Vitalunga recently began discussions with several venture capital funds, he said.
Itzik Goldwaser, the CEO of Yissum, said in a press release that Ben-Sasson and Gross’ findings have significant value for the globe’s aging population.
“As Vitalunga advances toward preclinical studies, they’re closer than ever to minimizing the unbearable burden that aging-related diseases, such as Alzheimer’s and Parkinson’s, have on individuals, their families, and our health care systems,” he said.