A Milestone Made Possible in parts Through Dr. Peter W. Schiller’s Basic Research
The Montreal Clinical Research Institute (IRCM) is proud to salute a major advance in the treatment of a rare eye disorder, a success due in large part to groundbreaking work by its own researcher, Dr. Peter W. Schiller, Emeritus Research Professor. Elamipretide — a mitochondria-targeted peptide co-discovered by Dr. Schiller — has shown remarkable results in treating chronic progressive external ophthalmoplegia (CPEO), a rare and currently untreatable condition.
CPEO is the most common form of primary mitochondrial myopathy affecting the eye muscles. Symptoms include drooping eyelids (ptosis), limited eye movement, and misalignment of the eyes (strabismus). Until now, there has been no FDA-approved treatment.
A recent case report [1] describes dramatic symptom improvement in a patient treated with elamipretide through an Expanded Access Program. After only two weeks of therapy, the patient was able to raise his eyebrows, look upward, and move his eyes again. Improvements were also noted in stamina, strength, balance, posture, and overall stability.
The origins of elamipretide, also known as SS-31, trace back to Dr. Schiller’s work at the IRCM. In collaboration with Dr. Hazel Szeto at Weill Cornell Medical College. Dr. Schiller developed mitochondria-targeting peptides based on an opioid peptide analogue he designed in his lab. A key breakthrough occurred when Dr. Schiller created a unique fluorescent version of this peptide for a confocal laser scanning microscopy study, allowing researchers to observe for the first time that it entered cells and concentrated specifically in mitochondria.
This led to the creation of a new class of molecules — Szeto-Schiller (SS) peptides — which localize to the inner mitochondrial membrane, the site of energy production in cells. The discovery that these peptides could act as targeted mitochondrial antioxidants and mitochondria-protecting agents opened up new therapeutic possibilities for a range of mitochondrial diseases.
Importantly, SS-31 was engineered to eliminate opioid activity while retaining mitochondrial targeting, making it suitable for mitochondrial drug development. Its therapeutic potential has since been demonstrated in hundreds of preclinical studies of mitochondrial diseases and is now being explored in advanced clinical trials by Stealth BioTherapeutics.
“We are extremely proud that IRCM research has played a pivotal role in this medical milestone,” said Dr. Jean-François Côté, President and Scientific Director of the IRCM. “Dr. Schiller’s visionary work on peptide design and cellular targeting is a testament to how fundamental research can lead to tangible breakthroughs that improve patients’ lives.”
Elamipretide is currently undergoing phase 3 clinical trials for primary mitochondrial myopathy and age-related macular degeneration.
About Dr Peter W. Schiller
Dr. Schiller's contribution to science is inestimable. The career of this leading scientist spans almost half a century and extends beyond the borders of Quebec and even Canada.
Trained at some of the world's leading institutions, including ETH Zurich and Johns-Hopkins University (USA), this chemist worked in the laboratory of the renowned Dr Christian Anfinsen, winner of the Nobel Prize for Chemistry (1972), at the NIH.
Born in Frauenfeld, Switzerland, Dr. Schiller retains strong ties to the town. He held a fellowship from the Swiss National Foundation for Scientific Research (1972-1973). His expertise spans several continents, from the National Institutes of Health in the USA (1974) to the Japan Society for the Promotion of Science in 1998.
Since 1975, he has been Director of the Chemical and Polypeptide Biology Research Unit at our company. A pioneer in the field of medicinal chemistry and the molecular pharmacology of opioid peptides, his research now focuses on the development of new painkillers whose side effects, such as dependence, tolerance and respiratory depression, are less severe than those of commonly used drugs like morphine. Worthy of mention: in 1991, Dr. Schiller was awarded the Canadian Pacific Chair in Pain, in connection with the work of his IRCM lab.
Together with Professor Hazel Szeto at Cornell Medical College, Dr. Schiller discovered Szeto-Schiller (SS)-peptides, which have major therapeutic potential for the treatment of mitochondrial diseases. IRCM and the Cornell Research Foundation hold several joint patents on these promising compounds, and Stealth BioTherapeutics Inc. is pursuing their clinical development for a variety of rare and common mitochondrial disease indications.
With more than 420 scientific papers and 17 patents to his credit, Dr. Schiller has, among other things, discovered important compounds that are now used as pharmacological tools throughout the world. His research projects were supported by the CIHR for 43 years and by the NIH for 30 years. He is one of only two Quebecers to have won an NIH MERIT Award, which is an eight-year grant.
Source:
[1] Ansari, S. & Koenig, M.K. (2024). Clinical Case Reports, 12:e9591. https://doi.org/10.1002/ccr3.9591
