In the course of his career in biotechnology, Dr. Matvey Lukashev has seen a remarkable evolution of treatment options available for patients with a devastating and once orphan neurologic disease affecting central nervous system, Multiple Sclerosis (MS). He joined Biogen, now a flagship neurology biotech, shortly after the company received FDA approval for its first MS therapy, Avonex®. The severity of unmet medical need that existed in MS at the time was comparable to the current state of Amyotrophic Lateral Sclerosis (ALS).

“Multiple Sclerosis used to be thought of as an orphan disease, just as ALS is sometimes talked about these days,” he says. “There were no effective treatments for it prior to Avonex®. A person diagnosed with MS had few treatment options available to them. Frankly, very little could be done for them at all, and that’s more or less where we’re at with ALS right now.”

However, the grave severity of the two diseases is not the only similarity Dr. Lukashev sees between his past work in MS and his current role as the Senior Director of Translational Research at the ALS Therapy Development Institute (ALS TDI). When he started in the field, MS was a disease that left people with few good options. But today – while there is still no “cure” – there are over a dozen effective therapies that allow those with the condition to live much longer, healthier lives. The advent of the first effective treatment for MS provided a key pointer to biological mechanisms targeting of which was likely to produce therapeutic benefit. As a result, additional effective treatments began to emerge rapidly to produce an array of treatment options available to MS patients now. Dr. Lukashev says he believes that the current state of ALS research brings us close to a similar breakthrough, or series of breakthroughs, that will significantly improve the length and quality of the lives of those diagnosed with ALS.

“The reason I think we may be on the cusp of doing in ALS what was once done for multiple sclerosis,” he says, “is that we have been learning a lot recently about the mechanisms that drive ALS and this rapidly accumulating knowledge is now positioning us for discovery of effective therapies.”

As the Senior of Director of Translational Research at ALS TDI, Dr. Lukashev has built and now leads a team that uses modern tools of genomics, genome editing, stem cell biology and automated phenotypic screening to study mechanisms of ALS and search for novel drug candidates. His team employs two main drug discovery approaches – chemical library screening and rational drug design.

In library screening, the team uses a unique cell-based drug discovery platform to screen a chemical library of 36,000 drug-like compounds that was purchased by the ALS Therapy Development Institute in 2019. The same cell-based platform is also used to evaluate new compounds that are being generated using drug design and synthetic chemistry. The compounds that show promise in a cell-based model of ALS are called “hits,” and the most promising of them get selected for testing in animals, a process that is much slower but more predictive of a drug’s efficacy in a human.

Dr. Lukashev joined ALS TDI in 2013, bringing with him over 14 years of biotech experience. He received his Ph.D. from the Russian Academy of Medical Sciences, and did postdoctoral research at the University of California, San Francisco and Johns Hopkins University. After completing his postdoctoral work, he spent over a decade at Biogen, where he led a research team that supported development of a blockbuster therapy for MS, Tecfidera®. This work earned him the company’s prestigious “Outstanding Scientific Achievement Award.”

After leaving Biogen, Matvey was attracted by the opportunity to join ALS TDI for a fresh start in another challenging disease indication. The new position offered a chance to build a new research function focusing on cell biology and translational research in ALS, a disease that desperately needed effective treatments, much like MS had in his early days with Biogen.

“I liked the mission, the focus and the challenge of building a new translational research and discovery function,” he says of his decision to come to join ALS TDI. “Just before I joined the biotechnology industry MS was in a state almost as desperate as ALS is today due to the shortage of available treatments. Having seen and being a part of the progress achieved in MS drug discovery inspires me to believe that we will soon see similar progress take place in the discovery of effective treatments for ALS.”

To learn more about the research Dr. Lukashev and his team are working on, click here.