Research Study Participation

Beginning in 2006, Dr. Benatar began conducting an observational study called The Pre-symptomatic Familial Amyotrophic Lateral Sclerosis (Pre-fALS) Study. Per the linked website, this study is described as follows: "This is a longitudinal study of people who are at risk for developing familial amyotrophic lateral sclerosis (fALS), with the specific goal of improving our understanding of the pre-symptomatic phase of fALS. Participation in the study involves undergoing genetic testing with the option of whether or not to learn genetic test results. Individuals who have undergone genetic testing and can provide documentation of genetic test results may be eligible to participate. Undergoing genetic testing for this study requires that the genetic mutation responsible for ALS in the family be identified."

 

The ClinicalTrials.gov link to this study can be found by clicking here: Pre-fALS.

 

While walking out of Riley's Children's Hospital after receiving his genetic testing results, Tucker dug his phone out of his pocket and called a family connection at an ALS research organization to ask for advice. This connection immediately placed Tucker into contact with Dr. Michael Benatar and his familial research study at the University of Miami (FL).

 

After months on a waiting list, Tucker underwent his initial intake interviews for the study in the Spring of 2018. After being determined eligible, Tucker's made his first visit to the study in September of 2018. To the extent allowable, Tucker will share in detail his visits to those considering participation in the study. As a participant in this study, Tucker returns to Miami, FL roughly every eighteen months and undergoes the same process as described below. Between physical visits, the study schedules quarterly remote check-ins with Tucker to discuss his current neurological health-state.

Below are links to recent work published by Dr. Benatar and others based on the participation of those who volunteer for the Pre-fALS study:

1) Defining pre-symptomatic amyotrophic lateral sclerosis

2) Neurofilaments in pre-symptomatic ALS and the impact of genotype

Tucker has nothing but phenomenal things to say about Dr. Benatar and his staff. While Dr. Benatar's study is one of the few benefactors of funds raised at the annual Olson ALS Foundation fundraiser, Tucker strongly encourages donating to Dr. Benatar's work in the event you are considering donating to ALS research.

The University of Miami (FL) Clinical Research Building
Photo Credit: Google Maps Steet View

Study Vist: Day One

The following morning, a shuttle paid for by the study arrived at the hotel to take Tucker to the University of Miami Clinical Research Building, where he was then escorted to an exam room for various exams.

After the exams, Tucker would be escorted to the cafeteria to briefly rest and enjoy breakfast. After the break, Tucker returned for further exams. 

After concluding a day full of tests, Tucker was sent back to his hotel to rest before returning to the Clinical Research Building the following morning.

Arrival in Miami

As a participant in the study, Tucker and other participants' travel to and from Miami, Florida.

Upon landing at Miami International Airport, Tucker found a ride to the hotel that the study places him at. After lounging around the hotel and the restaurants connected to the hotel, Tucker went to bed early that night knowing he would have a long day ahead of him the following day.

Tucker's breakfast view from the cafeteria of the Miami (FL) Clinical Research Building

ALS Research in 2020 - Update on Clinical Trials, Biomarkers, and Patient Perspective Surveys

Study Vist: Day Two

After waking up the following morning, Tucker made his way to the hotel's breakfast bar to enjoy breakfast before being shuttled back to the study site. Upon arrival, a member of Dr. Benatar's staff escorted Tucker to a separate building where Tucker underwent further testing.

After the morning testing, Tucker was escorted back to the Clinical Research Building to undergo a wide variety of exams. After another day of exams, the study provides Tucker with transportation to the airport for his flight home.

Extracurricular Activities Before/After Study Visit

Due to time and financial constraints, Tucker does not receive many opportunities to travel. This is why Tucker likes to utilize his study visits as a "mini-vacation" by extending his stay in Florida by a couple of days! Of course, the extended Florida stay is not paid for by the study and is instead comes out of Tucker's own wallet!

 

After Tucker's first visit to the study, Tucker rented a car and drove to Orlando, FL to spend time with relatives who live in Orlando.

During Tucker's most recent study visit in January of 2020, Tucker flew to Miami, FL two days early and rented an Airbnb studio apartment that overlooked the ocean! Tucker enjoyed the warm, sunny weather in comparison to the cold Indiana winter back home. Tucker has always enjoyed flying in airplanes and always desired to fly one. While scrolling through AirBnB's advertised activities, Tucker noticed the opportunity to fly a Cessna over Miami Beach and immediately booked the activity. Below are videos and pictures of Tucker's flight!

Upcoming:
The First Pre-Symptomatic ALS Trial

At the annual MNDA Symposium held on December 9th, 2020, the first-ever pre-symptomatic ALS trial was announced for individuals with the SOD1 genetic mutation, such as people like Tucker. This trial was designed in collaboration with Biogen and the research conducted by Dr. Benatar and Joanne Wuu, Sc.M. More specifically, the trial was designed utilizing the research data collected from the longitudinal Pre-fALS study conducted at the University of Miami's Miller School of Medicine. "The trial aims to evaluate whether the investigational SOD1 ASO treatment, Tofersen, can delay the onset or slow the progression of ALS when initiated in pre-symptomatic SOD1 mutation carriers".

As I'm sure most can imagine, Tucker is extremely excited about this trial and what it means for the future of all ALS treatments. As of right now, the trial guidelines have not yet been publicly released. As further information is made publicly available, Tucker will provide an update here on this website.

Thank you for your interest in Tucker's participation in the Pre-fALS study!

Tuck's Time © 2020