P ancreatic cancer is the diagnosis you don’t want to get. It’s the fourth leading cause of cancer-related deaths, and the American Cancer Society estimates that it will kill over 43,000 Americans this year. The five-year survival rate for those diagnosed with pancreatic cancer is a paltry 7%. Over half of those diagnosed are already in the more advanced stages, where treatment is little more than palliative. But there’s new cause for hope: Rafael Pharmaceuticals, a New Jersey-based company that develops anticancer drugs, has recently received FDA approval to initiate clinical trials of CPI-613, its promising metabolic cancer drug for pancreatic cancer and acute myeloid leukemia (AML).

Mishpacha originally encountered Rafael, then known as Cornerstone Pharmaceuticals, when we interviewed its chief scientific officer, Dr. Robert Shorr, in 2012. Dr. Shorr, a descendant of the Tevuat Shor (Rabbi Ephraim Zalman Shorr), earned his doctorate in biochemistry from the University of London and has since published hundreds of scholarly articles, with 300 patents issued or pending. He served at Enzon Pharma, United Therapeutics, and SmithKline Beecham, before cofounding Cornerstone and coinventing CPI-613.

CPI-613 selectively targets cancer cells and disrupts their metabolic processes, starving the cells and either killing them or preventing their multiplication. In recent trials, CPI-613 demonstrated promising results when used in conjunction with the standard pancreatic cancer treatment, a cocktail of drugs known as FOLFIRINOX. A recent issue of the highly respected, peer-reviewed medical journal, Lancet Oncology, showcased data demonstrating that CPI-613, in combination with modified FOLFIRINOX, seems to be safe, generally well tolerated by patients, and efficacious in treating certain Stage IV metastatic pancreatic cancer patients.

“We believe our clinical trial results may represent a significant medical advancement,” says Dr. Shorr. “Stage IV metastatic pancreatic cancer is a notoriously intractable disease. CPI-613’s results are quite promising.”

CPI-613 has exhibited encouraging results in treating other cancers as well. Rafael has received “orphan drug” status from the FDA for CPI-613 for pancreatic cancer, AML, and myelodysplastic syndromes (MDS). Orphan drug status bestows incentives to companies who devote time and resources to developing treatments for rare diseases that may be overlooked by pharmaceutical research efforts.

The company recently changed its name to Rafael Pharmaceuticals to reflect the religious faith of many of the company’s employees and investors, including Dr. Shorr and Howard Jonas, recently named chairman of the board. Jonas, a renowned businessman and entrepreneur, remarked, “Now that we are beginning pivotal clinical trials for CPI-613, we have changed our name to Rafael to reflect our mission of bringing much-needed healing to those in need. Rafael is the Biblical angel of healing, and we understand that medical breakthroughs and healing are gifts from Hashem.” (Originally featured in Mishpacha, Issue 665)