{"id":2958,"date":"2019-06-13T13:40:45","date_gmt":"2019-06-13T18:40:45","guid":{"rendered":"https:\/\/ibio.org\/?p=2958"},"modified":"2022-02-28T07:42:45","modified_gmt":"2022-02-28T13:42:45","slug":"the-power-of-a-clinical-trial","status":"publish","type":"post","link":"https:\/\/ibio.org\/the-power-of-a-clinical-trial\/","title":{"rendered":"The Power of a Clinical Trial"},"content":{"rendered":"

Successful therapy for their daughter leads a couple to advocate for the next generation of children<\/em><\/p>\n

By Jed Weiner, iBIO News Brief Contributor<\/p>\n

\u201cElizabeth\u00a0was like a sound machine that helped her brother sleep.\u00a0Her labored, nasally breathing was constant. But one night,\u00a0she was so quiet, I got scared. I thought she had stopped breathing,\u201d recalls Christy Clow, mother to Elizabeth and Daniel, now age three, and their big brother, five-year-old Patrick. \u201cBut\u00a0she\u00a0was quiet because her breathing had improved so much, primarily from medication she was getting in a clinical trial.\u201d<\/p>\n

In March 2016, a week after the birth of her twins, Christy and\u00a0her husband, Michael,\u00a0received devastating news: State-mandated genetic screening of newborns revealed that Elizabeth had cystic fibrosis (CF). According to the Cystic Fibrosis Foundation, CF is \u201ca progressive, genetic disease that causes persistent lung infections and limits the ability to breathe over time.\u201d\u00a0Lung\u00a0damage caused by CF eventually shortens\u00a0a\u00a0patient\u2019s life, but medical advances have increased\u00a0average\u00a0life expectancy\u00a0from the mid-20s in the 1980s to the late-40s currently.<\/p>\n

Elizabeth was able to access a new, investigational\u00a0medicine\u00a0because\u00a0Christy and her\u00a0Michael\u00a0chose to enroll their daughter in a clinical trial shortly after her first birthday.\u00a0Elizabeth\u2019s type of genetic mutation was a match with how the drug work\u2019s in a CF patient\u2019s body.<\/p>\n

\u201cBy participating in a clinical trial, we are among the very few who have received a level of hope that just a few years ago was unimaginable,\u201d Christy said\u00a0with deep gratitude in her voice.\u00a0\u201cThe impact of the drug was obvious by the third day!\u201d<\/p>\n

The\u00a0medication\u00a0helps to treat the abnormality in CF cells. Christy and\u00a0Michael\u00a0gave the medicine in soft food twice a day. This was in addition to other treatments that\u00a0Elizabeth\u00a0started\u00a0shortly after her diagnosis, including inhaled medicines and chest\u00a0physical therapy two times a day to loosen mucous that\u00a0could accumulate\u00a0in her\u00a0lungs, enzymes with each meal to help her digest her food, and special\u00a0vitamins.\u00a0Elizabeth\u2019s overall health, including her height and weight,\u00a0was good when she entered the study but\u00a0continued to improve\u00a0over time.<\/p>\n

Christy describes the medical team at Lurie Children\u2019s Hospital in Chicago as \u201camazing.\u201d At every appointment, Elizabeth was seen by a dedicated team of pulmonologists, nutritionists, nurses, social workers, and physical therapists.\u00a0Christy and\u00a0Michael\u00a0say they will be forever grateful to the team.<\/p>\n

\u201cIn\u00a0a clinical trial,\u00a0the patient may benefit from getting a new medication early, but you also have the benefit of additional\u00a0doctor visits, blood draws and\u00a0monitoring,\u201d explained\u00a0Susanna\u00a0A.\u00a0McColley, MD,Associate Chief Research Officer for Clinical Trials at\u00a0Stanley Manne Children\u2019s Research Institute at Lurie Children\u2019s and Professor of Pediatrics at Northwestern University Feinberg School of Medicine, both in Chicago. \u201cYou are\u00a0assessed more frequently than when you\u00a0are\u00a0receiving regular clinical care. You also have the satisfaction that you\u00a0or your child\u2019s participation is broadly going to help others who may be in a similar situation. Knowing that your actions may help your child and help others\u00a0is very meaningful to many people.\u201d<\/p>\n

Altruism\u00a0certainly\u00a0is one of the motivating factors behind\u00a0Michael\u00a0and Christy\u2019s decisions about\u00a0Elizabeth\u2019s care.\u00a0They plan to continue to provide data to a CF registry long after Elizabeth exits the clinical trial later this year.<\/p>\n

Christy says she wants to build on the success of people who advocated for public funding for new research, the Orphan Drug Act. Previously, children often did not receive an accurate CF diagnosis until they\u00a0had severe symptoms,\u00a0such as growth failure or daily cough.\u00a0Implementing\u00a0newborn screening\u00a0throughout the United states\u00a0has made a dramatic difference because treating CF before the onset of\u00a0malnutrition and\u00a0severe lung damage\u00a0leads to\u00a0better health outcomes.<\/p>\n

\u201cWe are\u00a0reaping\u00a0the\u00a0benefits of what\u00a0other have done before, so I\u00a0consider it my obligation to do the same for the next generation of kids,\u201d Christy said. \u201cWe are grateful for their efforts and\u00a0Michael\u00a0and I and our family and friends are advocating\u00a0to\u00a0improve the lives of children with CF and other rare diseases.<\/p>\n

\u201cElizabeth still has a long road ahead of her with lots of uncertainly, but what a gift\u00a0for us\u00a0to know\u00a0that\u00a0she could likely live to see\u00a0the availability of a\u00a0cure\u00a0<\/em>for CF\u00a0while\u00a0her\u00a0lungs\u00a0are\u00a0still healthy enough to live a normal life.\u00a0Advocating for her and other children\u00a0is\u00a0an important mission for us.\u201d<\/p>\n","protected":false},"excerpt":{"rendered":"

Successful therapy for their daughter leads a couple to advocate for the next generation of children By Jed Weiner, iBIO News Brief Contributor \u201cElizabeth\u00a0was like a sound machine that helped her brother sleep.\u00a0Her labored, nasally breathing was constant. But one night,\u00a0she was so quiet, I got scared. I thought she had stopped breathing,\u201d recalls Christy […]<\/p>\n","protected":false},"author":12,"featured_media":2959,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_et_pb_use_builder":"","_et_pb_old_content":"","_et_gb_content_width":"","footnotes":""},"categories":[143,147,471],"tags":[148],"class_list":["post-2958","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-ibio-news","category-patients","category-rd2","tag-patients"],"_links":{"self":[{"href":"https:\/\/ibio.org\/wp-json\/wp\/v2\/posts\/2958","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/ibio.org\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/ibio.org\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/ibio.org\/wp-json\/wp\/v2\/users\/12"}],"replies":[{"embeddable":true,"href":"https:\/\/ibio.org\/wp-json\/wp\/v2\/comments?post=2958"}],"version-history":[{"count":1,"href":"https:\/\/ibio.org\/wp-json\/wp\/v2\/posts\/2958\/revisions"}],"predecessor-version":[{"id":7820,"href":"https:\/\/ibio.org\/wp-json\/wp\/v2\/posts\/2958\/revisions\/7820"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/ibio.org\/wp-json\/wp\/v2\/media\/2959"}],"wp:attachment":[{"href":"https:\/\/ibio.org\/wp-json\/wp\/v2\/media?parent=2958"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/ibio.org\/wp-json\/wp\/v2\/categories?post=2958"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/ibio.org\/wp-json\/wp\/v2\/tags?post=2958"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}