KO Pro Bono Client Elpida Treats Ultra-Rare Diseases for Children Worldwide

KO pro bono client Elpida Therapeutics’ mission is to deliver gene therapies to patients with ultra-rare diseases that are non-commercially viable and overlooked by traditional biotech and pharmaceutical companies. By leveraging scientific advancements and gene therapies, Elpida aims to provide treatments for children and families that desperately and urgently need them.

In 2019, Terry and Georgia Pirovolakis’s 15-month-old son Michael was diagnosed with an ultra-rare neurodegenerative disease called Spastic paraplegia 50 (SPG50) caused by a single gene. Without a cure or treatment, Michael would progressively lose his physical and cognitive abilities resulting in cognitive impairment and eventual paralysis.

After the diagnosis, Terry liquidated his family’s life savings and worked tirelessly over three years to raise $4.5 million through fundraising, community support, golf tournaments, a bikeathon, galas, and awareness from media coverage. He worked extensively to secure hundreds of researchers worldwide to develop a prototype gene therapy needed for Michael’s groundbreaking single clinical trial. After three years, four-year old Michael received the treatment in March 2022. The treatment is intended to stop and potentially reverse progression of the disease.

“We think it’s one of the first times someone has taken the strategy of gene therapy and done a single individual clinical trial, so we’re very excited for the results for Michael for this trial, but also what it could mean for other children with rare diseases,” Dr. Jim Dowling, a staff physician in the division of neurology and senior scientist in the Genetics & Genome Biology program at SickKids, who diagnosed Michael and led his clinical trial told CBC News.

The FDA has since approved a Phase III clinical trial for SPG50 gene therapy and Terry recently left his job as an IT manager and made it his mission to tackle ultra-rare disease programs. In May 2023, Terry launched Elpida, a California-based Social Responsible Corporation named for the Greek word for hope. Today, Elpida is serving programs for three diseases – SPG50, Charcot Marie Tooth disease type 4J (CMT4J), Neuronal ceroid lipofuscinosis 7 (CLN7) – and plans to add two more programs into its pipeline. The goal is to have five programs approved and funded over the next few years with each program dosing 8-12 children. Elpida has already enabled children in Connecticut and Spain to receive treatment for SPG50, and the children who have received the treatment have seen improvements in their mobility and cognitive abilities in the months since.

Terry collaborated with the Bespoke Gene Therapy Consortium (BGTC) of the Foundation for the National Institute of Health, officially embracing SPG50 and CMT4J as programs. The BGTC aims to “establishing minimum standards for manufacturing, product analytical testing and pre-clinical testing.”

The KO team, led by partner Brad Schoenfeld, is working with Elpida and its various charities on a pro bono basis to assist with their corporate and contractual legal needs, including negotiating strategic licenses and clinical trial-related agreements. KO attorney Samantha Pjesky and paralegal Lisa McDiarmid and Sara Shelly are also working with Elpida.

To learn more about the great work being conducted by Terry, Elpida and its related charities, and how you can help, please visit https://www.elpidatx.com/.