Paul’s Journey with SCID

Cameron’s would like to share an update on one of the families that we have been blessed to meet, Pauline, Kevin, La Dreama and Paul.

Paul was born on November 10, 2017, with a disease called x linked SCID (bubble boy disease). The typical treatment is a bone marrow transplant. His treatment was a trial called gene therapy. There is technically no “cure” for the disease, but his results thus far suggest he does not have x linked SCID anymore. It has been a long road, but things are really looking up.

We were asked by Paul’s grandmother to assist her son’s family back in January 2018, with the purchase of an air purifier that would enable them to bring their newborn son “Paulie” home. We also provided a counter-top sterilizer/dryer that his parents were able to use on his bottles. To help make sure that Paul’s room at home was clean and sterile they needed to wipe everything down (at least 2x a day). We supplied them with Lysol Disinfecting Wipes.

Severe Combined Immunodeficiency (SCID)

Paul’s journey with Severe Combined Immunodeficiency (SCID) started when his mother La Dreama was 13 weeks into her pregnancy, she had her amniotic fluid tested. She knew she was a genetic carrier for x linked SCID, after testing the fluid the Doctors determined Paul would be born with the disease. The Gallagher’s had a lot of time to prepare. Shortly after finding out, accompanied by Pauline Gallagher (Paul’s grandmother) they would have their first ever appointment with Dr. Pai. She is Co-Director, of the Gene Therapy Program at the Dana-Farber’s Jimmy Fund Clinic, and she is largely responsible for how great Paul is doing.

In the first meeting with Dr. Pai, they talked mainly about the normal treatment for this disease, which is a bone marrow transplant. They were given a detailed explanation about how his bone marrow transplant would go. The Gallagher’s knew gene therapy trials were also an option, but the word “trials” scared them, so they really didn’t focus on that option. This was a very somber day in their lives because the details of the road before them for a bone marrow transplant were very sad. Paul would have to endure 10 days of intense chemotherapy. This chemotherapy comes with risks to all the patient’s vital organs and the risks can possibly last forever.

Choosing a Treatment

In the few days after the first appointment, with their collective mind set on a bone marrow transplant; they both started researching the gene therapy option. It all started by asking a Facebook group they are part of which is dedicated to children with SCID, how others feel about gene therapy vs a bone marrow transplant. The gene therapy option came with 3 days of chemotherapy, as opposed to 10, it would still be harsh but not as bad. La Dreama decided to spend a good portion of her pregnancy filling a notebook with comparisons between a bone marrow transplant and gene therapy. As her extensive research progressed, it became increasingly clear that she was leaning towards the gene therapy trials. They chose the gene therapy trials.

Along with Paul’s doctors, the Gallagher’s came up with a birth plan to have Paul in a sterile environment at UMASS Memorial in Worcester, MA. They brought him home to a sterile environment because of the high-powered air purifier and a year’s supply of Lysol wipes donated by Cameron’s Crusaders. Between the air filter and La Dreama’s seemingly endless cleaning they were able to keep Paul from getting sick while waiting for his treatment.

Time absolutely crawled by while they were waiting for treatment. It couldn’t come soon enough. Finally, at 4 months old they checked in overnight to Boston Children’s Hospital so they could harvest Paul’s bone marrow. They extracted stem cells from the bone marrow to reprogram them with the purpose of repairing the faulty gene that causes Paul’s disease (x linked SCID).

Life day/day zero

After waiting a month for them to reprogram the cells the Gallagher’s checked back in for the long haul on April 16, 2018. Upon checking in they knew that Paul would be in the hospital for at least a month. This was the day they’d been waiting seemingly forever to get to. On the 17th and 18th, Paul was given chemotherapy. The 19th was the day to rest his body. On April 20th, 2018 Paul received his genetically modified stem cells. This is called his life day/day zero.

They also knew Paul would experience some type of pain from the chemotherapy. At least a week had gone by since his chemo and he hadn’t had pain, so they let their expectations get a little too high. In the coming days, the pain started. He experienced about a week of pain but eventually it would subside, and his numbers were good enough for them to be discharged.

Paul was discharged on May 20, 2018, the treatment ended up being a huge success.

As of January 2019, Paul was no longer on isolation. His immune system is normal today. He is on no medications, gets sick less than his parents get sick. He is a perfectly normal 17-month-old boy.

We recently had the opportunity to finally meet Paul and his parents, after waiting over a year since we were first asked to assist them. The meeting was at their apt., totally comfortable for all.  We felt like we were visiting family. Cameron’s is happy to know that we were able to help in a small way and make a difference in a child’s life.

Today, April 20th Paul is celebrating the first anniversary of his Life Day….

Written by Kevin Gallagher, La Dreama Wood & Kevin St Jean