An Uncommon Battle: Living with Chronic Intestinal Pseudo-Obstruction

The 2023 PDA Annual Meeting in New Orleans, April 3-5, was filled with over 300 hundred industry members who came together and reported changes happening in pharma. Monday through Thursday consisted of noteworthy sessions that centered around leaders in the industry, such as scientists and regulators who shared their perspectives on topics regarding the revision of European Union Annex 1: Manufacture of Sterile Medicinal Products, the blooming acceleration of achieving cybersecurity in healthcare and the steps that are actively being pursued in the industry that are pushing the boundaries for new drug development.

Meeting Kelly Baker

The first plenary session on day one of the annual meeting set the tone for the rest of the informative sessions throughout the 2023 PDA Annual Meeting.

After the introductions from Susan Schniepp (Board of Director’s Chair), Glenn Wright (PDA’s President and CEO), Amanda McFarland (Senior Consultant, ValSource, Inc) and Julia Jenkins (Executive Director, EveryLife Foundation for Rare Diseases), Kelly Baker (rare disease representative) took center stage to talk about a rare disorder called chronic intestinal pseudo-obstruction (CIPO).

According to the National Institutes of Health (NIH), CIPO is a lifelong, rare motility disorder found in children that causes abnormalities in the nerves, muscles, or both, preventing food, fluid and air from moving through the stomach and intestine, which cause mechanical obstruction or blockage. In addition, CIPO can occur as a primary condition called idiopathic CIPO or as a secondary condition associated with underlying diseases such as autoimmune disorders, neurological conditions, connective tissue disorders, or certain medications. As it pertains to diagnosing CIPO, the NIH’s studies have shown that it can be difficult, given that it requires subtracting other potential causes of intestinal obstruction and evaluating motility patterns in the gastrointestinal (GI) tract. Diagnostic tests may involve imaging studies (such as X-rays, ultrasound, or computed tomography), manometry (measuring muscle contractions in the intestine) and endoscopy to visualize the intestines directly. Moreover, treatment for CIPO aims to manage symptoms, improve motility and prevent complications. It typically involves a multidisciplinary approach involving gastroenterologists, nutritionists and other specialists. Nonetheless, the Boston Children’s Hospital expounds on the most common treatments of CIPO that assist in controlling symptoms or complications, which include the following:

• Medicine or symptom management: Different medications can be prescribed to regulate intestinal motility, control symptoms like pain and nausea and manage complications such as bacterial overgrowth. These medications usually include oral medicines and epidural anesthesia.
• Nutrition support: Nutritional deficiencies are common in CIPO, and individuals may require specialized diets or nutritional supplements to ensure adequate nutrient intake. Some doctors may recommend parenteral nutrition, which supplies nutrients directly into the veins using a catheter.
• Surgery: In some severe cases, surgery to remove part of the intestines might be necessary, known as surgical decompression. A surgeon may insert a feeding tube directly into the stomach or create an opening in the abdomen through which the intestines can release gas and empty stool, causing pressure to be released from the intestines. In some patients, when pseudo-obstruction is limited to an isolated segment of the bowel, surgical bypass may be considered. However, this procedure is usually the last resort and is not always effective.

After Kelly’s presentation, everyone in the room stood and applauded Kelly for her bravery in speaking about her condition, along with the grit she possesses to live with a lifelong disease that causes frequent abdominal pain.

“That brave young lady who talked about her experience with CIPO is why we are here today and is why we care about science,” said Vinicius Tarantino (Head of Digital Compliance and Protection, GXP-CC) during his session on cybersecurity and risk management at the 2023 PDA Annual Meeting, and a majority of other members at the annual meeting felt that exact sentiment throughout the conference.

[Following the conference, the Managing Editor of the PDA Letter interviewed Kelly regarding her experience with CIPO.]

PDA Letter: Considering the standing ovation you received after your presentation at annual, do you see any urgency or effort in the pharmaceutical industry to find better treatment for CIPO?

Kelly: I appreciate the support from the audience after my presentation. However, I realize that progress in the pharmaceutical industry can take time, especially when it comes to drug development, policy and manufacturing. Currently (and for the past few years), there is not much research or drug/treatment development happening for CIPO.

The main treatment for CIPO is symptom management, which sometimes includes parenteral medications and nutrition. I would like the pharma industry to realize the impact of drug shortages and access concerns for patients like me who rely on these products to live.

PDA Letter: When were you diagnosed with CIPO? And was your diagnosis primary or secondary? Also, what are some personal challenges that you have experienced with this disease?