Updated April 24, 2023:
In mid-April, I officially concluded my one year clinical trial for the next generation Trikafta (the game changing drug that increased my lung function by 14%). This new version reduces the number of pills and more importantly the number of times per day the pills need to be taken. There is also the possibility of improved lung performance and reduced side effects.
During the clinical trial this past year, the study was double blind meaning that the drug company and the patient (me) did not know if the original Trikafta (two pills in the morning and one in the evening) or the new version (two pills in the morning only) was provided. In fact, for the trial, I was taking four pills in the morning and one in the evening with the understanding that "one set" of them was a placebo.
The exciting part is that at the conclusion of the trial, I rolled over to "open label" which officially gives me the new version of the medication until it is approved by the FDA. This is done through a new clinical trial which may last two years. Open label refers to the fact that everyone involved is aware what version of the medication I am on (the new stuff). It's like getting a free Beta version of the newest Tech until it ultimately gets released (sold) to the public.
Being a part of this process is exciting and there are many more medications and treatments being developed that will have an even greater impact on those living with Cystic Fibrosis.
With time and the right resources, a cure is destined to be just around the corner.
Updated: March 1, 2023:
After a 14% increase in lung function since starting the CF Modulator Trikafta almost three and a half years ago, my lung function remains stable and my health is good. Almost a year ago, I started on a clinical trial for the "next generation" Trikafta. The benefits include reducing the dosage to once a day and a potential reduction in side effects, Since the clinical trial is double blind, I am not sure if I am on the new medication or if I remain on the current medication but all is good so far. I was also recently diagnosed with CFRD or Cystic Fibrosis Related Diabetes. It's new to me so it will take a while to figure out how this may impact my health and lifestyle.
For the 90% of CF Adults who have started taking Trikafta, hospitalizations are down and life, in regards to CF, is better and definitely more hopeful.
However, Trikafta is not a cure. It is only the beginning. Your support is still needed. The CF Foundation is in this for the long run, to find treatments for the entire community and to ultimately find a cure. I am confident that with your help, this will happen. Thank you for continuing to be a part of the team.
I consider myself lucky. I am a husband, a stepfather and a full time engineer working for Stellantis (formerly FCA US LLC). My wife and I moved into a new house in the summer of 2020. Our daughter is working toward her masters degree at the University of North Carolina, Chapel Hill and and our son is living in North Carolina as well working for Biotronik. Joelle and I both work full time and I am even getting into the office once or twice a week.
Diagnosed with CF at the age of two, I watched my younger brother struggle and ultimately lose his battle with CF at the age of 21, three years post lung transplant. I do up to six breathing treatments per day, take pills every time I eat and I have been on home IV antibiotics close to a dozen times.
I grew up in Connecticut, attended Lehigh University and studied mechanical engineering. After graduation, I became more involved with the CF Foundation, speaking and fundraising in Michigan and Indiana. I never thought I would be the one to discover the big treatment or cure for CF but I always knew I would be part of the process.
By leading a Great Strides team for over twenty years and participating in six clinical trials, I can say with conviction that I am making things happen. Yet none of these new drugs in the pipeline for CF would exist without hundreds of walk teams and thousands of walkers across the country, like yourself, raising money to support their very existence. Not sure if you are making a difference? I will keep it simple. You are.