NEW YORK – Cure51 hopes to put a dent in cancer mortality by studying the mechanisms of exceptional survival in patients with very aggressive cancers such as those that affect the pancreas and brain.
In those cancers, five-year survival rates can be abysmally low, but research has traditionally focused on how to extend survival for patients who progress quickly or don't respond to treatment, rather than the factors contributing to survival.
The Paris-based company is conducting two clinical studies focusing on cancer survivors. In the Rosalind study, which launched in November 2024, Cure51 is working with Cambridge University Hospitals and seven other UK-based cancer institutes to characterize the underpinnings of very long-term survival in extensive-stage small cell lung cancer, glioblastoma, and metastatic pancreatic ductal adenocarcinoma (PDAC). Researchers will analyze tumor samples from more than 1,000 of the longest survivors with those cancer types for DNA, protein, microbial, and molecular biomarkers using 10x Genomics' Visium HD single-cell resolution, spatial transcriptomic gene expression platform.
Similarly, in a new study launched last week, Cure51 has teamed up with Assistance Publique-Hôpitaux de Paris (AP-HP) to analyze samples from more than 50 pancreatic cancer patients treated at five AP-HP hospitals: La Pitié Salpêtrière, Paul Brousse, Beaujon, Henri Mondor, and Saint-Antoine.
While researchers for years have been trying to glean from cancer patients why some do exceptionally well on treatment, Cure51 CEO and Cofounder Nicolas Wolikow said the company was founded in 2022 and since raised €15 million ($16.3 million) in seed financing to focus exclusively on studying cancer survivors. "Instead of looking at people who are dying, we wanted to share some hope, telling [patients] that it is possible to survive," Wolikow said. Although today it may be exceptional for patients to survive those aggressive cancers, Wolikow believes Cure51's research can make survival a more commonplace occurrence.
Cure51 has the ambitious goal to eradicate cancer within the next 15 years by focusing on the few patients that beat the odds and make it to their five-year anniversary or beyond. Wolikow said he was inspired to start the company after his mother was diagnosed with small cell lung cancer in 2021. "That was a trigger, and I decided to really dedicate all my energy and all my knowledge to try to cure this disease," Wolikow said.
For many cancers such as melanoma, lung, and breast cancer, there have been significant breakthroughs in treatment over the past 20 years to 30 years, and patients have benefited from dramatically improved survival. However, Wolikow pointed out that for other cancers, there have been few or no treatment advances. For example, he noted that in advanced PDAC, patients are still receiving the same standard, first-line FOLFIRINOX chemotherapy they did three decades ago. "The therapeutic landscape did not evolve," he said. "It's the same for glioblastoma and roughly the same for small cell lung cancer."
Wolikow credited hospital partnerships for making Cure51's work possible. Because exceptional survivors are very rare, they are difficult to find. "You can't just navigate on Facebook to find them," Wolikow said. "You need to establish very strong partnerships with hospitals."
Cure51's hospital network includes more than 100 hospitals in 42 countries, and the company is in negotiations with more than 150 more. In addition to identifying exceptional survivors to enroll in the study, hospital partners will collaborate with Cure51 by providing feedback on any signatures or targets identified from the studies. From partner hospitals, researchers have collected tumor samples from 230 survivors and began sequencing them three months ago. Most of those early patients have pancreatic cancer. Although it's too early to draw any conclusions from the data, Wolikow said he has a gut feeling that the driver of very long-term survival in those patients might be related to "an exceptional response to treatment."
Another key to making the project a reality, according to Wolikow, is the availability of advanced sequencing technology. "It was impossible to do it five years ago or 10 years ago because the technology of sequencing the genome was extremely expensive, and we were not able to have single-cell resolution," Wolkow said. Now, thanks to the availability of technologies like 10X Genomics' Visium platform, the company is able to conduct whole-transcriptome analysis at single-cell resolution plus spatial profiling, proteomic, and microbiome analysis for a cost per patient of about €1,000. "It was €100,000 even five years ago," Wolikow said.
Cure51's primary objective is to discover the mechanisms of exceptional survival and potential drug targets that could replicate that effect for every patient. The company aims to build a library of robust, validated drug targets and form partnerships with drug developers to advance therapies against those targets. However, Wolikow said the fruits of its research could include an option to test patients for markers of exceptional survival to inform their care. "The first layer of analysis will be fully in silico," Wolikow said, but if that analysis shows a benefit, the company will progress to early-stage clinical trials for such a test.
Throughout their work, Wolikow and his colleagues at Cure51 have maintained a focus on patients. "Every day we're talking to survivors," with the hope to make survival possible for every cancer patient, Wolikow said. "We want to convince people there's a solution, and we will try to modestly contribute to this."