A new therapy that could change the face of Muscular Dystrophy treatment has been discovered in Charlotte.

News | 20 days ago

How Atrium Health Medical Research is Helping People with Muscular Dystrophy

Muscular dystrophy is a slow-progressing muscle weakness that leads to loss of mobility and, in some cases, cardiac and respiratory failure. Currently, there are no approved treatments – and no cure. But there is hope. Charlotte-based startup ML Bio Solutions has received fast track designation from the FDA to investigate a potential treatment that originated in Atrium Health’s McColl-Lockwood Laboratory for Muscular Dystrophy Research.

[Photo Courtesy of Piper Warlick Photography] 

The U.S. Food and Drug Administration (FDA) has given “fast track” designation to investigate a potential new treatment option for limb-girdle muscular dystrophy type 2i (LGMD2i). This investigational therapy, initially developed at the McColl-Lockwood Laboratory for Muscular Dystrophy Research at Atrium Health, provides a novel approach that targets the rare disease at its source. Generally diagnosed in young people who are 5 to 20 years old, LGMD2i produces a slow-progressing muscle weakness that leads to loss of mobility and, in some cases, cardiac and respiratory failure. Currently, there are no approved treatments – and no cure.

Fast track designation was granted to Charlotte-based startup ML Bio Solutions, an affiliate of BridgeBio Pharma, Inc., for its BBP-418 program to investigate the treatment of patients with LGMD2i. The FDA grants fast track designation to encourage development and expedite its review process for drugs that may be able to treat serious illnesses and fill unmet medical needs. The partnership between ML Bio Solutions and BridgeBio is dedicated to advancing the development of a potential treatment for LGMD2i.

“LGMD2i is a serious disease with no currently FDA-approved treatment options,” said George McLendon, chief executive officer of ML Bio Solutions, Inc. “I am, personally, deeply gratified to be at the forefront of research built on the work of Atrium Health scientist Dr. Qi Lu. I am also humbled by the patient community and hope that the perseverance of our team will lead to a life changing treatment for these patients and families.” 

"This collaboration and partnership is a perfect example of how innovation, coupled with a commitment to saving and enhancing lives, can change the course of history," said Dr. Rasu Shrestha, executive vice president and chief strategy and transformation officer at Atrium Health. "At Atrium Health, we often extend our mission in new and unexpected ways in order to have the maximum possible impact, including finding novel cures and treatments for disease. We are excited to see that something our teammates are so personally invested in has reached this point in the process.”

Shrestha is also a member of the ML Bio Solutions board of directors. 

The announcement of the FDA action was announced Sept. 15, 2021 – just ahead of the annual observance of limb-girdle muscular dystrophy awareness day on Sept. 30. September is also muscular dystrophy awareness month. 

“Our family has worked closely with Atrium Health for almost 20 years to reach this point,” said ML Bio Solutions Founding Board Chair and President Luther Lockwood. “We are excited about our success at ML Bio Solutions and the other innovations created by Dr. Lu and the team at the lab. Expanding the medical research capabilities in Charlotte is a game changer for our city and our region.”

The success of the McColl-Lockwood Laboratory for Muscular Dystrophy Research and its collaboration with biotech companies and startups, such as BridgeBio and ML Bio Solutions, foreshadows what is envisioned for a new innovation district to be developed to support a second campus of Wake Forest School of Medicine in Charlotte. 

“We want to build an ecosystem of innovation that will advance medical research, impacting the lives of patients both locally and internationally,” said Shrestha. “With our connection to Wake Forest School of Medicine – including the new, second campus coming to Charlotte – this innovation district will become the intersection of education and research, bringing together physicians, students, researchers and businesses. We will build upon the successes of the existing Innovation Quarter in Winston-Salem, in a way that strengthens our regional economy, as well as our learning programs – advancing research into reality in a way that the field of medicine has never seen before.”