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First patient to try FDA-approved drug for amyloidosis is thankful

Dr Benson And Terry Baker

​More than a decade has passed since Terry Baker first noticed signs of amyloidosis. Back in 2008, his toes tingled and felt swollen. He says it felt like he was walking on pins and needles. These symptoms were indicators that he could have the same genetic condition his mother had, so he set an appointment to meet with the doctor she had trusted, Merrill D. Benson, MD, professor of pathology and laboratory medicine with Indiana University School of Medicine. Dr. Benson had been working on developing a treatment for amyloidosis, but it wasn’t ready to be tested in patients just yet.

“It was a huge relief just to know that he was in the process of developing a drug that he thought would help slow or possibly stop progression of the disease,” said Baker, who is 68 years old. “That was very reassuring and I just kind of fell in love with Dr. Benson, as far as his personality and his approach to the whole thing.”

Amyloidosis is considered a rare disease, with fewer than 200,000 patients affected in the U.S. The condition occurs when amyloids, which are proteins, get folded abnormally and build up in a patient’s organs, causing them to begin to shut down. If left untreated, patients can die within a handful of years of showing symptoms. Men tend to develop the disease at a younger age than women. Men are also more likely to have the older-onset form of the disease that’s not hereditary. Other symptoms include:

  • Severe fatigue
  • Shortness of breath
  • Joint pain
  • Sudden weight loss
  • Tingling and numbness in legs and feet

“After seeing these patients and not having any specific therapy for them, other than treating their heart failure, their kidney failure, et cetera, we decided perhaps a specific therapy would be a good thing,” said Dr. Benson.

Dr. Benson started studying the disease in the 1970’s and began developing a treatment for it about 20 years ago. As the doctor was working on the later stages of the treatment, Baker’s condition deteriorated more rapidly. Baker considered getting a liver transplant, which would have made him ineligible for Dr. Benson’s study.

“I actually had three liver transplant options between January and March [2013], but I turned them down and put my faith in Dr. Benson’s drug being available soon,” said Baker, who lives in Findlay, Ohio. “As of April 1, [2013] I was able to make a trip to Indianapolis and actually start the study.”

Baker was the first patient enrolled in this final phase of the FDA trial, which was conducted by the Indiana Clinical and Translational Sciences Institute (CTSI). This part of the study took place between March 2013 and November 2017. For 15 months, patients were given some kind of treatment, but only 2/3 were given the drug itself. The other 1/3 took a placebo, but none of them knew whether they were on the drug. After 15 months, all the patients started using the drug.

Dr Benson And Terry Baker 2

The drug that Dr. Benson developed was later given the scientific name, Inotersen. Now that it’s on the market, the drug is marketed and sold under its brand name, Tegsedi. Tegsedi (Inotersen) cannot cure patients from amyloidosis, but it helps with some symptoms. The drug works by decreasing the production of the protein that creates the amyloid fibers, which build up in different organs. Because of that, patients usually notice improvements in nerve damage, can walk better, and are able to be more self-sufficient in their daily tasks.

 Baker still doesn’t know for sure if he was on the drug during the clinical study, but he is convinced that he was. He says the progression of his symptoms slowed down as soon as he started the weekly injections.

Now that Tegsedi (Inotersen) has been FDA-approved, Baker and other patients can use the drug, which consists of one shot administered on a weekly basis. Even though the study has been over for two years, Dr. Benson’s work isn’t finished.

“The plans are now to test a modification of the drug, which looks as though a smaller dose will be just as effective,” said Dr. Benson.

Dr. Benson hopes that would mean fewer side effects, plus patients wouldn’t need to get shots as often. Baker still has some of his symptoms, but says he feels healthy overall and is convinced he’s better off because he has been on the drug.

“I feel very thankful that the drug became available and I have never doubted for a minute that I made the right decision,” said Baker.

The views expressed in this content represent the perspective and opinions of the author and may or may not represent the position of Indiana University School of Medicine.

Anna Carrera

Research Communications Manager

Anna Carrera is the research communications manager for Indiana University's Precision Health Initiative, IU School of Medicine and the Indiana Clinical and Translational Sciences Institute. She joined the team in June 2019 after working as a TV news rep...