Mary Beth Kiser

It’s been said that having faith as small as a mustard seed can move a mountain. Within the rare disease community, rare disease patients and rare disease parents are known to be capable of moving mountains in order to advance awareness, research, and funding for their, or their child’s rare disease.  Once in a while, the rare disease community is gifted with someone who, although neither patient nor parent, is dedicated to moving the mountain for the good of rare patients and rare parents. One such someone is Mary Beth Kiser, President and CEO of Beyond Batten Disease Foundation (BBDF).

In 2022, Mary Beth Kiser, CAE, will celebrate 10 years with Beyond Batten Disease Foundation. Before joining BBDF in 2012, Ms. Kiser was the Chief Operating Officer of Texas Land Title Association. She brought to the foundation skills earned from two decades of professional and volunteer leadership experience, including board and volunteer management, program development and fundraising and business development.  While at the helm of BBDF, she earned the Texas Association of Nonprofits Certificate in Leadership and Management, further bolstering the tools in her toolbox.

Beyond Batten was established in 2008 by parents Charlotte and Craig Benson, after their then 5-year-old daughter Christiane, was diagnosed with juvenile Batten disease. According to BBDF’s website, Batten disease is one of a group of disorders known as neuronal ceroid lipofuscinoses (NCLs). There are many different forms of Batten, differing based on when symptoms first appear. To date, over 400 different errors (mutations) in 13 different genes have been attributed to various forms of Batten, and all affect the nervous system, causing increasing seizures, movement disorders, altered thought and cognitive decline. Children diagnosed with Batten experience vision loss. The CLN3 form of Batten is also known as juvenile Batten, is ultra-rare, inherited, and fatal, regularly leading to a child’s death in the late teens or early 20s.

Mary Beth with Beyond Batten Disease Foundation Principal Scientist Dr. Danielle Kerkovich at a Batten Disease conference.

Mary Beth was introduced to the Bensons by Christiane’s aunt, and in 2012, the Bensons and Ms. Kiser determined the time was right for her to join their mission. Although Mary Beth has no formal medical research training, she and the board of directors collaborated on developing a strategic plan and then setting about putting structure around the plan to stay focused on their mission of eradicating juvenile Batten disease. She brought structure to a volunteer program. They rallied to focus on, in Mary Beth’s words, “How can we make an impact on getting a drug (treatment) into kids?” She describes their work as how rare diseases get addressed. She calls on strength from the children diagnosed with Batten disease and their parents, who provide motivation and resources for their mission.

As Mary Beth describes the frustrating journey of the rare disease family, “You have a crisis (the first onset of symptoms), and a diagnosis and then wonderfully, friends and family rally to help. As the disease progresses, the community support gets redirected to other new crises.” But for the parents, there is no turning away. Theirs is a new altered reality of endless doctor appointments, navigating the world of insurance coverage and juggling full-time jobs and parenting other children. And that’s where Mary Beth and BBDF can be a lifeline for a newly diagnosed child and their family.

Under the BBDF umbrella, the Bensons and Mary Beth give a platform to other families impacted by Batten disease. There are currently about a dozen member families with their own fundraising platforms which support BBDF. Mary Beth has learned so much from these parents, and works to help ensure the funds raised are pooled to have the biggest bang, attracting those “rock star” researchers, as she calls them.

Ms. Kiser recognizes that within a specific rare disease, there may be huge problems that take years to solve, but she knows that the way to take control is by taking action. Ten years after coming to BBDF, Mary Beth reports they are one step closer to getting an approved treatment. Along the way, she was essential to the team’s success in finding a pharmaceutical company partner, writing the protocol, drafting contracts that allow sharing of the research data, and doing regular check-ins with the researchers to ensure optimal use of the funds BBDF provided. A clinical trial for BBDF-101 will commence in January 2022.

Along the journey, Mary Beth says their team is always keeping an eye on determining the end points of experiments, how is data reproduced and validated, how can they drive development of other treatments or utilize compassionate care use of the drug treatment.

Although she admits they don’t have all the answers, Mary Beth believes there are significant advantages to the approach they’ve taken to combat Batten disease. She’s suggested authoring a white paper on starting a rare disease foundation, and hopes to share their model to advance research across diagnoses. She serves as a mentor to other rare disease foundations through the “Rare as One” initiative. As she believes, her biggest value comes from the fact she’s not afraid “to ask the dumb question. What might seem initially like a “dumb question”, may in the long run, be the spark that led to development of a new treatment, because it got the researchers, the business leaders and the patients and families all thinking about the disease differently. And for that fearlessness, Mary Beth Kiser is definitely a Rare Disease Difference Maker®.