When it comes to curing cancer, one scientist gives God all the credit as he moves one step closer to slaying the resilient killer.
Rabbi Dr. Robert Shorr, 55, the CEO of Cornerstone Pharmaceuticals, is overseeing the Phase I/II clinical trials of his newly developed cancer combatant drug, CPI-613, produced in conjunction with researchers and technology at Stony Brook University’s Long Island High Technology Incubator. Aiming to target pancreatic cancer and a wide array of other diseases, doctors are testing CPI-613 both alone and in conjunction with gemcitabine, an already standard chemotherapy treatment for pancreatic cancer — the disease that recently killed actor Patrick Swayze after a 20-month battle.
Meanwhile, as Shorr dives headlong into cancer research, the Orthodox biochemist remains a practicing rabbi, teaching students for free through the Partners in Torah over-the-phone learning service.
“I try to infuse in my professional life not [just] learning the Torah, but living it, and that’s a sanctification of Hashem’s name,” said Shorr, who finds no conflicts between matters of God and science.
For Shorr, the biggest obstacle in developing cancer drugs is the fact that no two cancer cells are the same. “Even within a single patient not all the cancer cells are going to be the same,” he said.
But Shorr’s new drug works to destroy a resource that every cancer cell needs for survival — adenosine triphosphate (ATP), the energy-transferring molecule that cells produce through glucose conversion.
“Without ATP, cells can’t do anything and they eventually die,” Shorr said. “What our drug does is turn off the ability of cancer cells to make ATP — a catastrophic shut-down of ATP synthesis.”
Shorr says his drug is able to shut down ATP synthesis in the cancer cells without doing so in healthy cells, because it mimics a vitamin co-factor that cancer cells — and only cancer cells — actually require for ATP production.
“This technology is particularly compelling because it takes a unique and new approach to combating the disease which is quite different from what many others have done,” said Joe Scaduto, assistant director of business development of the Center for Biotechnology at Stony Brook, where Shorr worked for several years and is now a financial supporter for emergent companies like Cornerstone. “[Shorr] transcends that business and science component of what it means to bring one of these drugs to market.”
Gemcitabine, the second drug, is more of a classic anti-cancer agent and turns off nucleic acid synthesis to prevent the rapid cell division among cancer cells.
The FDA has currently granted CPI-613 “Orphan Drug Status,” a status grouping that falls under the Orphan Drug Designation Act, which provides financial incentives for pharmaceutical companies to develop drugs that serve patient populations under 200,000.
In normal clinical trials for non-cancer drugs, tests occur in three phases: toxicity tests on healthy volunteers, efficacy within a small patient pool and then larger, expanded trials, Shorr explained. But in cancer drug trials, Phases I and II must be combined because cancer drugs are too toxic to test out on everyday volunteers. Only patients in life-threatening situations who have tried all other options qualify for such Phase I/II cancer drug trials.
“The last thing in the world you want to do is take a healthy volunteer and give them something toxic,” Shorr said. “These are patients who have pretty much failed everything else.”
Life expectancy for patients who enter Shorr’s trial at Eastchester Center for Cancer Care in the Bronx is typically three months to a year, and their bodies are physically exhausted, unresponsive and resistant to other treatments. Patients who volunteer for the combined CPI-613 and gemcitabine trials, however, are not required to have failed all other treatments because the gemcitabine is already FDA-approved for cancer treatment.
The combined trial at Eastchester currently has two patients with a few more people joining soon, while the CP-613 solo trial has one patient and two more to come shortly, according to Avi Kamelhar, the executive director at the Center. Other local trial facilities are spread throughout the country, he added.
“These patients don’t have any other hope and this is giving them something,” Kamelhar said.
In both the combined and solo trials, the patients started on a low dosage of CPI, and doctors have been able to gradually escalate the dose, achieving some visible benefit at low toxicity levels, according to Shorr.
“In studying mice that have cancer we have seen an effective dose that’s a thousand times lower than a toxic dose,” he said. “For most cancer agents the difference between an affective dose and a toxic dose are only a few percent.”
Shorr is equally excited about achievements with human patients.
“The results are encouraging enough for us to want to expand the clinical trials,” he said. “If the drug is well tolerated and the patient has resumed their life and regular activities, those are the most important considerations and everything else is considered secondary. Survival is the most important of all.”
Kamelhar and the rest of the Eastchester Center staff express great faith in Shorr’s project and hope to see continued positive results.
“[Shorr] is motivated by wanting to help and he does not attribute his success to his own credit,” Kamelhar said. “He’s a man of faith and he attributes everything to God. He wants to help people — that’s what his motivation is. We’re really excited to be part of that.”
Realistically, Shorr explained, a successful cancer drug takes about three to five years to complete the clinical trial process. He hopes that CPI-613 will fall into this category. And if all goes as planned, he hopes one day to be able to drastically improve patients’ lives.
“We’re not in this for the incremental improvement, though any improvement is welcome. We’re in it for the big improvement. We’d like to make cancer a manageable disease, prolong life and improve its quality.”
“With three out of four Americans expected to experience cancer in their lifetimes,” Shorr continued, “I’d say the matter is pretty urgent.”