Almost every drug on the market today followed a similar development path. They were invented in the research lab of a university or a pharmaceutical company, developed by a company, and then sent to market after approval by the FDA. It’s nearly unheard of for a practicing physician to invent a drug, but that is just what Sidney J. Goldfarb, a urologist affiliated with Penn Medicine Princeton Medical Center, has done.
Goldfarb has invented a drug that he believes can save many patients from having to use a catheter to urinate. Inability to urinate is a common condition that can be caused by prostate problems, diabetes, neurological problems, and, temporarily, by anesthesia following surgery. “Some of my patients can’t empty their bladder, and so sometimes they have to catheterize themselves five or six times a day,” he says. “It can get infected and have more problems. If you could void normally, that would be preferable.”
There is an existing drug to help this condition: it’s called bethanechol, and it works by stimulating the muscles in the bladder to contract, helping expel urine. However, Goldfarb says, this drug is not always effective.
Goldfarb did not invent a new drug from whole cloth. Rather, he has found that by raising the dose of bethanechol above the standard amount and administering it together with another drug, Aricept, it was more effective than if given on its own. He believes that combining these two drugs into a single pill could be an effective new drug to treat urinary retention and save thousands of patients across the country from the pain of a catheter.
Urinary retention is a problem that affects thousands every year. By one estimate, 10 percent of men over 70 and a third of men over 80 will develop it in any given five-year period. (It is rare in women.)
Goldfarb has tried his new treatment on 19 patients male patients and four female patients, and says that of this group, 18 of the men and one of the women were able to urinate normally. He wrote his findings in a paper and in 2015 presented it to an international conference for treating underactive bladder. More recently, he showed it to the Temple University Hospital urology department
He says that recommended dosage of bethanechol varies from country to country, and that the dosage being too low is one reason the drug is not effective with most patients. Aricept, a drug used normally to treat Alzheimer’s symptoms, has the side effect of slowing down how fast bethanechol is metabolized, making it stay long enough for the high dose to take effect.
Goldfarb says that when he presented his paper to the other doctors at the urology conference, “jaws dropped.”
“I’m just a doctor, and these people are professors of urology, the chairs of their departments. They do very high level research. Really top-notch research around the world on how the genes of the bladder work and how to put pacemakers in the bladder. It’s very high level science. And I said, ‘hey guys, here’s a drug that we’ve had for 40 years, and you don’t know how to make it work.’”
Because Goldfarb was merely combining drugs that are already considered safe and effective, using one of them for an off-label purpose, he needed only to give his patients informed consent, rather than going through a full clinical trial. If his combination of drugs were to be made into a pill, it would need to get FDA approval and a larger clinical trial — an endeavor far beyond the resources of a local urologist.
That’s why Goldfarb seeks to partner with a pharmaceutical company to develop the drug, which he has patented. He has submitted his small study for publication in a journal and is giving talks in hopes of attracting attention to his new treatment method.
“Thousands of patients need this,” he says.
Dr. Daniel Eun, head of the Temple University Hospital urology department, said Goldfarb’s treatment seemed to be worth further investigation.
Eun said there is currently little that can be done to help patients with chronic urinary retention. “We essentially don’t have a way to properly medicate these patients,” he said. “They are subject to long-term catheterization, or have to catheterize themselves multiple times a day. It’s discouraging to have to go to a patient and tell them, ‘That’s the way you are going to be for the rest of your life.’”
He said that Goldfarb’s treatment showed some promise, but would need to be formally studied.
“This is preliminary work, but I think it deserves some focus,” Eun said. “It would be great to have some funding because I think that to really research that and know if we have something truly effective, it needs to be done on the basis of a clinical trial with properly selected patients to try to minimize bias.”
Eun said Goldfarb’s procedure was an interesting idea. “I hope he finds a drug company that is willing to support this work and further it,” he said.
Goldfarb has been practicing medicine in the Princeton area since 1980. He was born in New York, the son of concentration camp survivors. His father worked in what Goldfarb described as a “sweatshop” and later owned a candy store and a grocery store, sending Goldfarb to study at New York University for his bachelor’s degree and Albert Einstein in the Bronx for his medical degree.
He is married to Naomi Vilko, a psychiatrist, who played a key role in inventing the new treatment. Goldfarb says he often drives his wife around as she gives talks, and he learned a lot about giving presentations from her. “I learned the drug really well, and my wife taught me how to give a talk,” he says. “It helps because you really have to know the drug, know the pharmacology, the interactions, and the contraindications.”
Goldfarb says that he is a “bad salesman” but hopes that he can get a drug manufacturer to take up his idea. If they do so, Goldfarb’s patent will allow him to benefit from his discovery. He says he made sure to get the patent because of an experience he had 22 years ago with another drug.
“I came up with another drug, which I thought was great, and I went to a drug company who I thought could make it, and after persevering for a few years, another company came out with it,” he says. Goldfarb’s idea was to combine a local anesthetic with a different drug that would make it last longer. The result is a way to deal with surgical pain without opioids — a numbing agent that lasts for three days at a time. The drug, which was developed without Goldfarb’s involvement, is called Exparel.
Godlfarb doesn’t believe his idea was stolen, rather, that someone else came up with it independently. But he says it was a learning experience. “If I had known it was a $500 million drug I would have done things differently,” he says.
Exparel showed Goldfarb that keeping an open mind and combining drugs in creative ways can not only help patients, but can be good business as well. And this may not be the last drug to come out of Goldfarb’s office.
“We use other innovative therapies,” Goldfarb says. “If something is not available you have to use your head sometimes and come up with other ideas.”
Sidney J. Goldfarb MD, 419 North Harrison Street, Suite 206, Princeton 08540. 609-921-3008.