Before joining Hawkins Point, I spent 30 years in the Insurance and Financial Services industries. This path surprisingly brought me to a point where I never saw myself: managing IT infrastructure projects at a biotech company. Though my own work has centered around IT operations, my time here has introduced me to some amazing science that I didn’t even know existed.
Sure, insurance and financial products can have a significant change in peoples’ lives, but it pales in comparison to the innovation and miraculous discoveries within the biotech industry. Through the clinical trial process, I’ve seen how lives are changed because of the work being performed by scientists and patient advocates.
In particular, I’ve been amazed at how innovations have had a direct impact on patients with severe genetic diseases—such as sickle cell disease—and T-cell-based immunotherapies. The journeys of hardship and successful breakthroughs shared with patients and their families has been inspiring. I’ve seen the dedicated people in this industry work toward one common goal: the hope for a better life.
The promising world of gene therapy
A major contributor in helping patients with these conditions is gene therapy. To be honest, I had never heard the term “gene therapy” until I did some research before an interview. What I read sounded like something scientists did to create dinosaurs in the Jurassic Park movies. However, I soon discovered that it was not the stuff of sci-fi. It was, in fact, a very real and rapidly evolving technology used to create treatments that are having tangible impacts on peoples’ lives.
For example, genetic diseases, like sickle cell disease, happen when some genes within a person’s body do not function properly. The technology behind gene therapy is designed to alleviate those deficiencies by developing new genes for an individual that provide necessary proteins to combat the cause of the disease.
The hope with this type of transplant is to lessen the effects of the disease and provide a better quality of life for the patient. The science behind this is truly amazing, but what has amazed me the most is learning about the success achieved through stories shared by patients and their families.
Creating life changing treatments for sickle cell patients
Sickle cell is an inherited genetic condition that causes the body to produce sickle-shaped blood cells that clot blood vessels. This results in an array of painful and life-threatening issues, such as severe episodes of pain, strokes and early death. The disease affects around 100,000 people in the US alone. Today, clinical trials on an extraordinarily promising gene therapy are helping treat sickle cell disease and offering patients a new lease on life.
For example, 21-year-old Brandon Williams lives in Chicago with his mother and suffers from sickle cell disease. By the age of 18, he already had four strokes. Brandon had an older sister who suffered from the same disease. She succumbed to sickle cell at the age of 22. His family has suffered greatly, but they have endured. Thanks to experimental gene therapy, the family’s lives have improved immensely. Brandon’s symptoms have vanished. He is pain free and no longer requires blood transfusions. He’s no longer scared of what could happen. He wants to live a normal life and get a job, which wasn’t possible before due to the debilitating effects of the disease.
Another person who has suffered from sickle cell is Carmen Duncan, who lives in Charleston, South Carolina. Due to the effects of sickle cell, the now 20-year-old had to have her spleen taken out when she was just two years young. Carmen has been in and out of hospitals through most of her childhood. At times, her limbs would hurt so much from blood clots that she needed to stay in the hospital for up to two weeks at a time. But that has all changed due to gene therapy. The signs of sickle cell have vanished. Carmen now plans to join the military, a dream that was not previously obtainable due to her condition.
Many other patients and their families have seen similar successes. Though still in clinical trials, these encouraging and life-changing results provide many with what once seemed unobtainable: a normal, pain-free life.
Seeing the lives of people and their families transformed by innovations like gene therapy makes me proud to be part of an the Life Sciences community. Amazing innovations are happening across the industry each day. The work of dedicated scientists and patient advocates is making true progress in achieving their shared goal of offering people real hope for a better life.