CURE

desperate For a
While governments and pharmaceutical companies have invested billions in order to find a cure for cancer, the dreaded killer still strikes with a vengeance. Yet Dr. Robert Shorr, who has patented close to 200 chemical inventions, isnt giving up, having invested the last decade in a search for that elusive cure. If clinical trials are an indication, cancer sufferers may soon see a breakthough and more than a ray of hope in their frantic race against the deadly invaders
by Barbara Bensoussan photos Meir Haltovsky

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Desperate for a Cure

Its

so preternaturally quiet here, in this vast, landscaped industrial park in Cranbury, New Jersey, that you can almost hear the computers humming inside the handsome brick and plate glass buildings. Were in an area you might call the Silicon Valley of New Jersey: Its not far from Princeton University, and close to the home of the father of the electronic era, Thomas Edison. But were not here for the latest innovations in engineering or physics; its a different kind of breakthrough that has brought us here. Within one of these buildings stands the headquarters of Cornerstone Pharmaceuticals, a pharmaceutical company engaged in clinical trials for a breakthrough new drug that might just might offer new hope for cancer patients. The companys CEO and one of its founders, Dr. Robert Shorr, welcomes us into his simple but roomy office, part of an extensive office suite. Hes a big man, with an even bigger CV: research director at top pharmaceutical companies, entrepreneur, author of some 250 technical articles, abstracts, book chapters and conference proceedings, and originator of close to 200 inventions patented or pending patents worldwide. His work has garnered him such prestigious awards as the NJ Cancer Society Gallo Research Award and the R & D Magazine Innovation Award. But despite the credentials, Rob Shorr is no stereotypical science geek: Hes a Torah scholar who earned smichah from Pirchei Shoshanim, converted his basement into a beis medrash, and is a descendant of the 18th-century Tevuas Shor. His office is decorated by one solitary but striking work of art: a canvas depicting the Mishkan, its flowing, golden keruvim contrasting against a deep indigo background. It was painted by his daughter, an artist living in Silver Spring, Maryland. Looking at him today, its hard to believe there was a time, many years ago, when this distinguished researcher rode a motorcycle with friends, tended bar, and expressed himself through martial arts, weight lifting, and playing electric guitar in a rock and roll band. (I liked the mathematics, the dance aspect of martial arts, he comments. I still have a guitar, but these days I play folk music, not rock.) But you might have predicted the colorful past from his colorful family. His father, Morton Shorr, served in the Navy as a chief petty officer and MP. The elder Shorr was also a boxer, a horseman, and a gun collector who taught all his children to use weapons through the National Rifle Association. But

he was possessed of brains as well as brawn. My father was a famous rocket scientist, Dr. Shorr recounts. He designed propulsion systems. He was also the author of a textbook entitled Solid Rocket Technology. Various jobs took the senior Shorr to locations around the world; some of Rob Shorrs childhood was spent in Brazil, where his mother had been born (as a result, he grew up speaking not only English and Yiddish, but Portuguese as well). We always ended up in places where there were no Jews, or almost no Jews, Dr. Shorr says, with a clear tinge of regret. His father compensated for the paucity of Jewish resources by sharing the riches of his extensive secular knowledge with his three children; he provided his sons with toy microscopes and child-size telescopes, sparking their interest in science from an early age. We learned a lot with my father, Dr.Shorr says. He taught us math, physics, and other sciences. He used to set up and test model rockets in the basement of our house. He was careful, though. Hed figure out how to measure the forces involved and scale things down so they wouldnt do any damage to the house when he set them off. He grins. It was just very loud. For several years the family lived in Colorado, where his father worked for Martin Marietta Materials; it was there that they rode horses and practiced shooting. Dr. Shorrs older brother, recently niftar, followed his father into rocket science, working for NASA and private industry, while their sister became a nurse practitioner. Dr. Shorr admits to being an impatient student as a child. In the early grades, I had difficulty following the teachers directions, he says. Even in college at SUNY Buffalo, which he began at age 16 after an accelerated graduation from high school, his iconoclasm sometimes got him into trouble. I had difficulty accepting what the teachers said as absolute truth, he avows. It seemed to me that the hot topics of the day were masquerading as truth. My father was very into intellectual honesty, he adds. He would get emotional talking about Torah and mitzvos; hed watch the movie Exodus and cry; he really wanted to live a Torah life. But we always lived too far from any sort of real community. The deep family feeling for Yiddishkeit perhaps reflects the imprint of an illustrious ancestor, the 18th-century gaon Rav Ephraim Zalman Shorr (author of Tevuas Shor) who wrote seforim and trained shochtim in the Moldavia/Bessarabia

Sometimes the disease is too far advanced, and theres not much we can do, says Dr. Shorr. But Ive had people come in with very short life expectancies who have since gone back to work

tHeRe WAS A tIMe, MANY YeARS Ago, WHeN tHIS DIStINguISHeD ReSeARCHeR RoDe A MotoRCYCle WItH fRIeNDS AND exPReSSeD HIMSelf tHRougH MARtIAl ARtS AND playing

electric guitar

area. The rabbinic mesorah was passed on through quite a few generations; since Dr. Shorrs parents were cousins, the familys yichus flowed from both sides. My mother has family seforim that were brought with them from Europe in the 1860s, he says. Dr. Shorrs first movement toward Torah Judaism came after he had married his wife Pearl and finished his doctorate in biochemistry at the University of London. He was 25 years old, living in Philadelphia in an apartment across from Rabbi Moshe Sommerfeld zl, a Holocaust survivor he describes as a gadol bTorah. They began learning Shulchan Aruch together; he later became connected to Rabbis Levine and Kaplan of the Philadelphia Yeshiva. For a nonconformist, independent thinker like Rob Shorr, the decision to adopt a Torah lifestyle wasnt particularly wrenching: I never cared what people thought about me, he shrugs. Although I was very disappointed when I bumped into the issue of, What hat do I wear? The first time my wife suggested I should get a nice hat, I went to the store and tried one on, and the salesman looked frightened. He told me, You look like someone from the Mafia. He chuckles. Now they tell me, You look like a rabbi. So I guess Ive made progress. Oddly enough, he claims he was never able to grow a beard before he became religious; at age 26, hed never needed to shave more than once a week, and then just to remove some fuzz. But after he stopped shaving completely, during the first sefirah he kept, his beard grew in fully. The Shorrs eventually moved to Highland Park, near Rabbi Jacob Joseph (RJJ) Yeshiva, where they enrolled their sons. Dr. Shorr began learning the daf yomi with Rav Moshe Goldberger, and has since completed three cycles. Along with his son, he also studied for, and received, smichah through the Pirchei Shoshanim institute

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Desperate for a Cure

of Lakewood and Jerusalem. I still want to learn dayanus, he says. Its just not easy to find the time. He attributes his accomplishments, motivation, and continued growth to his wife, Pearl. Pearl has exceeded the role of wife, he says. Shes been a beautiful, loyal friend throughout the years. People occasionally challenge Dr. Shorr with the question, How can you be a scientist with a yarmulke and beard? But as far as hes concerned, theyre simply uninformed. Science is only capable of answering the how of existence, he maintains. It is incapable of answering the why. You shouldnt substitute one for another; science is only a tool.

Chemical Breakthrough Parallel to Dr. Shorrs evolution as a Torah Jew was his impressive evolution as a professional. His doctoral studies in England, he says, focused on the biochemistry of proteins how the nervous system and muscles communicate. From there, he went on to postdoctoral research with recent Nobel Prize winner Dr. Bob Lefkowitz at Duke University, an opportunity he calls a privilege. There, his research was directed at for those of you with pharmacological know-how G-protein receptor-linked drugs. From North Carolina he went to work for the research and development branch of pharmaceutical giant Smith, Kline & French, working on the human genome project, DNA research, and forensics. Then as vice president of Science and Technology for Enzon, Inc., he was responsible for the development and regulatory approval of the drug PegIntron A. Today, that drug has become the standard of care for hepatitis C and melanoma, he states without fanfare. He left to briefly form a company of his own, then sold it and went to work as the director of R & D at United Therapeutics (UTHR), where he was one of the inventors of Pegylated prostacyclin, a drug used in the treatment of pulmonary hypertension. In 2001, through his association with SUNY Stony Brook, Dr. Shorr found himself intrigued by the work of his colleagues, Drs. Bingham and Zachar, on technologies to fight cancer by targeting key enzymes involved in cancer cells metabolism. Together with business partners Bob Rodriguez and Gilbert Polinsky, he launched a start-up firm, Cornerstone Pharmaceuticals, to pursue this innovation. Cornerstone negotiated an arrangement with SUNY Stony Brook to rent out 1,000 square feet of lab space there and gain use of an animal testing facility, and license the technology that forms the basis of Cornerstones main technology platform. The company and the university also collaborate on applying for grants from institutions such as the National Cancer Institute and the National Institutes of Health. (Getting the grants isnt so easy these days, Dr. Shorr sighs. The pendulum swings a lot as to which diseases are hot. For awhile it was HIV; tomorrow it will be something else.) Cornerstone is currently engaged in clinical trials of their lead drug, which theyve labeled CPI-613, and the results so far have been highly encouraging. Dr. Shorr explains the theory behind this

The team at Cornerstone. So far, they havent found any cancers that resist CPI-613

breakthrough: Cancer cells, like healthy cells, process sugar and convert it to ATP (energy) molecules, he says. But there are differences in the way healthy cells and cancer cells do this processing. The cancer cells require a lipoate molecule to metabolize sugar; theres a chemical bond, an S-S bond, that needs to open and close. What we did is to make a molecule that looks a lot like it, but instead of performing that function, it activates a different enzyme, which shuts down the whole complex. Hence, it effectively starves the cell. The beauty of this approach is that, unlike most chemotherapy or radiation treatments, it predominantly affects the cancerous cells, not the healthy ones. He types something into his computer, then swivels the monitor around to display images of slides. Some of the slides depict mice with cancer, which were given four injections of CPI-613 over an 11 week period. The tumors disappeared, and the mice lived for another ten months. In their case, the drug was curative, Dr. Shorr declares with pride. He then displays slides showing results from the ongoing human clinical trials, cross sections of bone marrow. The first ones are full of spots, a Swiss-cheese effect created by the ravages of the cancer. The next slides, taken after CPI-613 treatment, show smooth, spotless bone marrow. Weve tested CPI-613 in lab dishes on all sorts of cancers, Dr. Shorr says. We havent found any cancers so far that resist it. Currently were working on a second-generation molecule, which has the advantage of being administered through a pill rather than an injection. The company has applied to receive some government funding for CPI-613 research for AML leukemia, a rare enough form of cancer that it qualifies CPI-613 for status as an orphan drug. Dr. Shorr explains that when a disease affects a very small portion of the population less than 200,000 cases a year then its generally not worth it for large pharmaceutical companies to invest major resources in searching for a cure. To induce private industry to do it anyway, the US government introduced the Orphan Drug Act, which offers grants, marketing exclusivity, and other perks. Its a prestigious thing to get, Dr. Shorr says. In order to qualify for FDA approval, any new drug has to be put through a series of clinical trials. In Phase I, Dr. Shorr explains, you take a number of patients, say 100, who are not being helped by standard care, and give them escalating doses of the new drug. Because the doses are low, you dont expect to see much benefit right off the bat although in our case, we did. Cornerstone is moving into Phase II, with a broader range of subjects in multiple locations, and higher doses of the drug. The company has published some of its positive Phase I results at major medical conferences and research meetings worldwide. Its a measure of CPI-613s promise that many highly respected hospitals have offered to run clinical trials for Cornerstone. We have been fortunate to be working with top-tier institutions and clinicians, and look forward to further expanding our studies, Chaya Reinitz, Cornerstones director of Corporate Development, later told Mishpacha. Physicians have reached out to us to include their patients in our clinical trials. The company is simultaneously working on other pharmaceutical innovations, such as its product called Emulsiphan, an intravenous drug delivery solution that

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Desperate for a Cure

Mrs. Reinitz says can work in tandem with multiple drugs to predominantly target cancer cells. The pressure of heading up what may be a major cancer breakthrough isnt easy, Dr. Shorr avows. Clinical trials are extremely expensive, he explains. Weve already raised over $40 million in capital, but require more. But its the desperation of patients, looking for that last hope, which keeps him at it. Ill often get calls in the middle of the night from patients who are desperate, asking about the drug. But thats okay. Often Ill find I cant fall asleep at night sometimes I just give up on sleeping and just learn straight through the night. His basement has been converted into a beis medrash, so that when insomnia strikes, he can study there without disturbing other family members (an empty can of Red Bull on his desk attests to those sleepless nights). Chaya Reinitz says that Dr. Shorr has a rare devotion. Hes been working on this for ten years, sometimes at great financial sacrifice. But he cant stop; he hears the patients desperate pleas.

On Their Own Were not the only company that receives calls from patients begging for an experimental drug. Dr.

Shorr sighs. Any company involved in clinical trials experiences this. He explains that when people receive a cancer diagnosis, they generally begin seeking care through a community oncologist or at a local hospital or cancer center. Different institutions provide various levels of care, and differ in how many approaches theyre willing or able to try; success will also depend on the patients individual reaction to treatment. When the more well-established approaches dont work, many patients find themselves on their own to see if a drug in clinical trials can work for them. Most of them go to the Internet, Dr. Shorr says. The government has a site where the trials are listed: www. clinicaltrials.gov. The site lets you know which trials are completed and no longer recruiting, and which are in progress and still recruiting. Often the explanations of the trials are very heavy on the jargon, so then people will turn to advocacy groups like the American Cancer Society to help them understand what its all about. By the time patients approach Cornerstone to participate in their trials, theyve usually exhausted all other forms of treatment. They call me in a very emotional state,

highly distressed, at all hours of the day and night, Dr. Shorr says. If I can help get them into one of our trials, I will. If not, Ill search the databases and scientific literature to help them connect to an alternative clinical trial, or to a doctor who may be able to offer something new. They find him not only through the Internet, but through doctor recommendations, magazine articles, and word of mouth. He says he often develops a bond with the people and their spouses who seek his help. Sometimes the disease is too far advanced, and theres not much we can do, he says. Its very hard when we lose them. But in some cases, it does work out well; Ive had people come in with very short life expectancies who have since gone back to work.

Take Your Vitamins

Dr. Shorr developed an interest in vitamin supplements when a chassidic rebbe recommended that he might be interested in the work of an alternative medicine practitioner in Boro Park. This practitioner provided him with some supplements, and to his surprise, he felt healthier after trying them. This led him to developing a sideline hobby in vitamin supplements; he has invented some of his own and brought them to market. The ground we farm gets depleted of nutrients, which means even our produce may be lacking them, he says. You can identify where plants originated, what terrain theyre from, by analyzing their vitamin and mineral content. He discovered he has some vitamin deficiencies and allergies, and now uses some vitamin supplements himself; hes also become careful about what he eats. Today, most people are low on selenium, calcium,

phosphorus, and vitamin D, he states. But isnt a balanced diet enough? Arent many vitamin claims a lot of hype? There is a lot of hype out there, Dr. Shorr admits. But theres truth too. One of these days, holistic medicine and clinical medicine will meet in the middle. Unfortunately, when it comes to alternative medicine, theres more data out there than real information. By this he means that while there are lots of products being advertised, there is less useful information available about how to use them, when to use them, and how to evaluate if an alternative medicine practitioner is a qualified professional or not. He also acknowledges that different peoples bodies react differently to the same products. It has to be that way, to allow for a person to make his own bechirah, he says. For example, smoking will cause cancer in many people, but not in everyone. The statistics say one thing. That doesnt mean miracles never happen. While still consumed 24/6 by CPI-613 research and development, Dr. Shorr nevertheless continues to envision new projects. He says he has ideas to help or cure conditions such as inflammation, chronic pain, and diabetes. He also has ideas how hed like to help the Jewish community, when G-d willing his new drugs and delivery systems are approved by the FDA, and the company goes public. Id like to fund projects that have to do with ahavas Yisrael, he says. And Id like to help fund limudei kodesh the real thing. The situation in day schools and yeshivos for struggling families is horrible; there were times when I was making $150,000 a year before taxes, and paying $100,000 in tuitions; I mortgaged my house more than once to pay tuition. My wife and I have been very rich and very poor, he admits with a wry smile. We have experienced both the blessing of giving and receiving. But given the promise of CPI-613, the future looks bright for Rob Shorr and his associates. Better yet, it looks bright for those thousands of patients, desperate for life, waiting for a miracle.

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