Physician-Researchers Study New Drugs for Autoimmune Disorders

Last updated: May 08, 2017

Dr. Marc Goldberg likes to think of his work as being one piece of a very large puzzle. Even though there are thousands of pieces, you need every single one to complete the picture.  

In his more than forty years as a rheumatologist, Dr. Goldberg has treated thousands of patients with debilitating autoimmune disorders, which affect as many as one in five Americans. Autoimmune disorders cause the body to recognize its own immune system as foreign and attack healthy cells. When this happens it can affect virtually any system in the body, most commonly the joints (arthritis), bowels (Crohn’s disease), skin (psoriasis), eyes (Sjogren's Syndrome), as well as other major organs like the kidney and lungs (Lupus).

But with every patient he helps, Dr. Goldberg is always thinking about how to impact a larger population. That is why he became involved in clinical research, which tests the safety and efficacy of new drugs for autoimmune disorders in small groups of patients.  

“I love practicing medicine and seeing my patients, but the beauty of clinical research is that we help bring these new drugs from the workbench into the world. We are finding drugs that do not just affect the handful of patients I see here in my office, but hundreds of thousands throughout the world,” explains Dr. Goldberg.

Over the years, Dr. Goldberg has seen that impact first-hand. When he first began practicing medicine in the 1970s his goal was to keep patients with a disease like rheumatoid arthritis—that causes pain, swelling, and joint damage—out of a wheelchair. Today, he aims for those same patients to lead completely normal lives.

The development of new drugs has made that difference. Forty years ago, the only tools in Dr. Goldberg’s arsenal were aspirin, gold shots, and cortisone. Then, in the late 1990s and early 2000s, several breakthroughs occurred. Researchers began to identify some of the inflammatory markers that cause these abnormalities to occur in those with autoimmune disorders.

“In an autoimmune disease, something is triggering the body to have an unusual immune response. We do not know most of the triggers some are probably genetic, environmental, infectious, or hormonal. But by identifying some of these markers that cause the immune system to overreact, we were able to design antibodies to block them,” explains Dr. Goldberg.

When a drug is first developed, researchers test it in what is known as a Phase I clinical trial. If the new therapy looks promising and is deemed safe by the Food and Drug Administration (FDA) and other advisory groups, it moves onto Phase II where it can be studied in a larger sample of patients. 

This is where Dr. Goldberg comes in. As a lead investigator for dozens of clinical trials, he identifies eligible research participants, studies the effects of the potential new drug, and evaluates the safety of the new therapy.    

In the early 2000s, he helped develop two of the most important immunosuppressant drugs to ever come on the market, HUMIRA and ACTEMRA. Immunosuppressant drugs are used to quell the strength of an overzealous immune system.

  • Scientists identified that a marker called tumor necrosis factor (TNF) was involved in systemic inflammation in people with autoimmune disorders. Today, HUMIRA, which blocks TNF and suppresses overactive immune systems, is used to treat six different disorders including rheumatoid arthritis and Crohn’s disease.
  • ACTEMRA works in a similar fashion, inhibiting a protein in the body known as interleukein-6 that is thought to contribute to the inflammation that causes rheumatoid arthritis.

Over the past few years, Dr. Goldberg has also been involved in testing several of the newest medications in the field including:

  • Cosentyx, an immunosuppressant, which helps reduce inflammation and alleviate symptoms of several autoimmune disorders.
  • A promising new class of drugs that block markers known as JAK inhibitors. The first medication of this kind, Xeljanz, has been found to successfully treat and help put patients with diseases like rheumatoid arthritis into remission. Additional JAK inhibitor drugs may be available in the next six months.
  • Expanding the use of Benlysta, a drug that is already approved for patients with Lupus.  

“Originally my job was to make patients comfortable while their disease progressed. Now, we have drugs that can stop the progression of the disease and, in some cases, stop it from developing at all,” says Dr. Goldberg.

“It has been tremendously rewarding to see these drugs we work on come to life and make a real impact on people.” 

References

  1. Interview with Marc Goldberg, MD, rheumatologist, SMG (3/2/17)

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