My son Pete and I began advocating to have SMA added to the newborn screening panel in Pennsylvania in December of 2017. Pete was 13-years old, type II SMA. He realized the impact that early diagnosis and treatment was having on SMA babies and thought he could make a difference. He has. Here is our story.
December 23, 2016
As I was cooking for Christmas, I received a text from Paula Saxton, a fellow SMA mom and a dear friend. Tears welled in my eyes and I announced excitedly to the family — the FDA had approved Nusinersen (Spinraza) for all types of SMA. After Pete’s diagnosis in 2005, I followed SMA research fervently, even enrolled Pete in a clinical trial across the country in California. But when, year after year, SMA research reported progress but no treatment, I couldn’t allow myself to be hopeful. It was necessary in order to preserve my emotional well-being. Pete was who he was and he was happy. He was confident: he raised his hand in class. He was mischievous: he sang rap music into the microphone he wore to amplify his voice during class when the class was doing quiet work. He was silly: his friends devised a go-cart from Pete’s therapy chair and a dolly and they rode it down a hill, pulled by Pete in his wheelchair. He had solid friends, able-bodied and wheelchair users: they considered him their equal. Now Pete was 12 and there was a treatment!
February 14, 2017
My favorite Valentine’s Day ever! Pete and I went to Children’s Hospital of Philadelphia and met with his awesome team for his Spinraza evaluation appointment. Pete began treatment March 5, 2017.
December 11, 2017
I met the Cure SMA Advocacy representative in Harrisburg for a day of meetings with PA state representatives and senators and/or their staffers. It was my first time advocating for a cause in this manner; I was unsure of what to expect and a bit nervous. We were greeted warmly and enthusiastically all day. I realized that our government employees do indeed consider themselves public servants. And all were empathetic as we talked about life with SMA, and life lost to SMA. Now that there is a treatment for SMA, it should be on the newborn screening panel in Pennsylvania.
December 12, 2017
A few weeks prior, Pete and I had a conversation. “Pete, come to Harrisburg with me. Representative Aaron Bernstine has sponsored an Awareness Resolution on SMA that will be announced on the floor of the House. You could also give testimony in support of screening newborn babies for SMA in Pennsylvania. Most SMA babies who receive early treatment are doing really well, Pete.” He said yes! I decided that, although Pete missed school often due to hospital admissions and doctor’s appointments, the civics lesson this day would be worth it. As Pete and I drove to Harrisburg, he practiced the testimony he would deliver at the Technical Advisory Board meeting that day. “Hi, my name is Peter Henkel. I am thirteen-years-old. I have type II spinal muscular atrophy…I need help getting up and getting dressed in the morning. I also have a 30-minute respiratory treatment every morning…I have had nine spinal surgeries due to scoliosis and kyphosis…I am bigger than my mom now so it is getting harder for her to transfer me…Despite these challenges, it is possible to have a happy life with SMA…”
We began our day in Harrisburg meeting with our representative, Alex Charlton. I knew that Rep. Charlton’s young daughter uses a wheelchair, so I thought he may be supportive. Rep. Charlton met our Cure SMA group in the rotunda. I was impressed with how much time he spent talking to us and was thrilled when he immediately agreed to sponsor a bill in support of newborn screening in Pennsylvania. Thank you, Representative Charlton! After the SMA Awareness Resolution on the floor of the House, we headed over to the Technical Advisory Board meeting. Pete and his friend Jake, who has type III SMA, were prepared to give their testimony but when we arrived, the meeting had already adjourned. The committee had worked through lunch and adjourned early. The boys never gave their testimony.
Pete and Jake Saxton at the Pennsylvania Technical Advisory Board Meeting in December 2017.
January 25, 2018
Dear Representative Charlton, You were so kind to meet us—along with our group from Cure SMA—in the lobby of the Capitol building in Harrisburg on December 12th. My son Peter and I enjoyed talking to you about your own family challenges with a child who uses a wheelchair. I wanted to let you know that we lost Peter unexpectedly on January 3rd. He was on the school bus, just arriving at school, when he had difficulty breathing…I understand you have been moving forward with your support of the newborn screening bill for SMA. Thank you! It is so meaningful to us since many babies and families could benefit from early diagnosis and treatment. Sincerely,
April 26, 2018
I spent a day on Capitol Hill with other Cure SMA members advocating for SMA to be added to the NBS panel and for fiscal support. Our experience was similar to my day in PA. We were greeted politely and enthusiastically and all empathized with our stories of kids struggling with SMA and lives cut short by SMA. Lobbyists can do this work, but when families show up, representatives and senators listen more intently.
May 3, 2018
My husband Tim was frustrated. His work felt endless and he was searching for the meaning in it since Pete’s death. He decided to take a day off from work on the 3rd of each month. Pete was born on the 3rd and died on the 3rd. I have a plan for your May 3rd, I told him. Let’s go to Harrisburg. He said yes!
Tim, Pete’s twin sister Lucy and two of Pete’s best friends, Ben and Nate, and I attended the Technical Advisory Board meeting. The room was full of brilliant people who worked in various aspects of healthcare. Tim and I were invited to contribute to the discussion on adding SMA to the NBS panel. Tim was incensed that there could be opposition due to the cost of adding SMA to the screening panel. I was able to provide details on other states that had already begun screening for SMA and families in PA that had babies who had received early treatment and are asymptomatic. But the most meaningful part of the meeting was when Pete’s twin sister Lucy gave the testimony that Pete never had a chance to give, “Hi, my name is Peter Henkel…I am here today to support screening babies born in Pennsylvania for SMA. Because of the new treatment for SMA, if babies are treated early, they could be much stronger and likely even walk. This would help them avoid many of the challenges I have faced.” They all understood our emphasis on the importance of early diagnosis and treatment. And many of them cried. And I was invited to participate in a sub-committee that was created to gather detailed information on adding SMA to the NBS panel.
Lucy Henkel and Pete’s best friends, Ben and Nate, after the Technical Advisory Board meeting in May 2018.
July 3, 2018
Secretary Azar signed the federal recommendation to the states to add SMA to the NBS panel!
August 17, 2018
Members of Cure SMA’s PA Chapter and I met with a staffer from Senator Pat Toomey’s office. We asked for his support of the Newborn Screening Saves Lives Act, which is up for reauthorization in 2019. We also asked for his support for funding at the highest level possible for federal newborn screening programs at the Centers for Disease Control and Prevention (CDC) and the Health Resources and Services Administration (HRSA). The staffer was attentive during our meeting and emailed us afterward to let us know that we had Senator Toomey’s support.
September 20, 2018
The Technical Advisory Board voted unanimously in favor of adding SMA to the NBS panel in PA. Our work is not yet over, however. The matter now goes to the Governor’s Budget Office. Cure SMA PA Chapter members will write letters asking Governor Wolf and the Secretary of Health for their support. I am optimistic!
Cure SMA has been so helpful and supportive in this advocacy journey. Cure SMA members have, on my behalf, scheduled meetings with senators, congressmen and staffers and provided important statistics and handouts to facilitate those meetings and, in some cases, even attended meetings with me. Do not be intimidated by this process. If you would like to advocate for newborn screening for SMA, be assured that Cure SMA will support you.
Our family is proud to honor Pete through SMA advocacy with Cure SMA.
Thank you to Allyson Henkel for advocating on behalf of the SMA community and for sharing her advocacy story with us!