MDA’s Development Grants Support Promising Researchers Entering the Neuromuscular Field

Since its founding 75 years ago, MDA has been the nation’s largest nonprofit supporter of neuromuscular disease research. Every year, MDA awards grants to support promising research in the field. Over the years, MDA has supported more than 9,000 scientific investigators, and about 2,500 have received Development Grants. These grants have a specific purpose: nurturing the careers of bright young scientists in the neuromuscular field.

“Supporting the next generation of scientists is key to pushing innovative research forward,” says Brian Lin, PhD, Research Portfolio Director at MDA. “Young scientists bring fresh perspectives and creative ideas that complement the expertise of seasoned researchers, helping us move closer to finding treatments for neuromuscular diseases.”

What are Development Grants?

MDA has been awarding Development Grants since we started funding research. This grant program provides three years of salary support and funding for research supplies for early-stage career scientists. To qualify, applicants must have obtained a doctorate (PhD) or medical degree (MD) and have a minimum of a year and a half of work experience in a laboratory. Development Grant applications are peer-reviewed by MDA’s Research Advisory Committee, an expert panel of independent researchers with significant contributions in neuromuscular disease research, neuromuscular medicine, biotechnology, and therapeutic development.

All Development Grant recipients are paired with senior investigators. The goal is to prepare them for a scientific career path, whether in a laboratory, clinical setting, nonprofit, or government agency.

“Development Grants are a crucial support, giving early-career scientists the resources and opportunities to explore their ideas, collaborate with a global network of researchers, and cultivate the curiosity that will drive their future independent careers,” Dr. Lin says.

Success stories

Many Development Grant recipients have established their own labs or become leaders in their field. Here are just two examples of young scientists who have built successful careers around the work they started in their Development Grant projects.

Dwi U. Kemaladewi, PhD, received an MDA Development Grant in 2013. She is now a Principal Investigator at the FDA.

Dwi U. Kemaladewi, PhD, received a Development Grant in 2018 to study the development of therapeutic genome editing in LAMA2-deficient congenital muscular dystrophy (LAMA2-CMD), a project she started in Dr. Ronald Cohn’s lab at Hospital for Sick Children in Toronto. She credits the Development Grant with making her application for a faculty position at the University of Pittsburgh School of Medicine stand out. She established her independent laboratory and continued her work at that institution, eventually winning additional grants and the NIH Director’s New Innovator Award in 2021.

This year, Dr. Kemaladewi joined the Office of Gene Therapy and Office of Therapeutic Products, Center for Biological Evaluation and Research at the US Food and Drug Administration (FDA) as a Principal Investigator leading a research laboratory on therapeutic genetics and disease modeling in rare diseases. In this role, she also reviews and evaluates the quality and safety of gene therapy products.

“I’m very excited for this once-in-a-lifetime opportunity and grateful to the MDA Development Grant for helping me jumpstart my career,” she says.

Matthew Alexander, PhD, received a Development Grant in 2013 while working as a postdoctoral research fellow in the lab of Dr. Louis Kunkel. His project involved defining a key microRNA pathway that regulated muscles in people with Duchenne muscular dystrophy (DMD).

Matthew Alexander, PhD, received an MDA Development Grant in 2013. He now leads an independent research lab at the University of Alabama at Birmingham.

“Once I received the award, I remember how excited I was to start molecularly and functionally characterizing my DMD microRNA using some of the newest molecular sequencing techniques,” he says. The work led to a significant publication in a scientific journal that helped him establish his independent laboratory at the University of Alabama at Birmingham and Children’s of Alabama. In addition, the data from his Development Grant project eventually led him to other new areas of research, including other muscular dystrophies and spinal cord injury responses.

“I believe it is essential for MDA and other organizations to help the next generation of muscular dystrophy scientists, as many new and innovating lines of research have been the result of the early-stage career support that the MDA Developmental Grant offers,” he says. “I am very grateful for the support MDA showed me early in my career and plan on ‘paying it forward’ by helping both the next generation of muscle investigators and discovering new treatments for those affected by these disorders.”

Meet the new Development Grant recipients

MDA recently awarded Development Grants to these six promising scientists conducting research related to neuromuscular diseases.

Adam Bittel, DPT, PhD

Institution: Children’s National Research Institute, Washington, DC

Development Grant project title: Defining the Protective Effects of Estrogen in FSHD

Research focus: Investigating the mechanisms contributing to less severe disease in females with facioscapulohumeral muscular dystrophy (FSHD) and the potential of using estrogen signaling as a therapeutic strategy.

Why study this? “Clinical and experimental evidence suggests females with FSHD are less severely affected than males. This curious and potentially significant phenomenon may provide critical insight into how this disease develops and/or progresses in skeletal muscles. We are especially excited to continue our investigation into the role of estrogens and estrogen-signaling in this process and to further explore if estrogen-based therapies can be used to mitigate symptoms of FSHD.”

Michele Brischigliaro, PhD

Institution: Miller School of Medicine, University of Miami, Florida

Development Grant project title: Aberrant mt-mRNA Folding as a Mediator of Mitochondrial Encephalomyopathies

Research focus: Gaining a new understanding of mitochondrial disorders by investigating changes in messenger RNA (mRNA) structure.

Why study this? “I am excited to uncover how mRNA structure — a newly discovered layer of mitochondrial gene expression — shapes disease mechanisms. Understanding this hidden regulation is key to revealing how mutations altering mRNA folding contribute to mitochondrial disorders and could pave the way for entirely new treatment approaches.”

Bradley Alan Hamilton, PhD

Institution: Stanford University School of Medicine, California

Development grant project title: Addressing Immunotoxicities to AAV Gene Therapy for DMD and Other Disorders

Research focus: Innovating methods to understand and avoid potentially dangerous immune responses in people administered adeno-associated virus (AAV) based therapies, expanding the population receiving therapeutic benefits.

Why study this? “I believe a world in which no one dies of a genetic disease is possible, and we are moving in that direction one step at a time. My research aims to remove a significant obstacle along the path.”

Felipe de Souza Leite, PhD

Institution: Boston Children’s Hospital and Harvard Medical School, Massachusetts

Development grant project title: Notch Pathway Modulation in Satellite Cells of Two Novel mdx Models

Research focus: Investigating the molecular mechanisms of Notch modulation in DMD and testing a strategy to extend motor function without dystrophin.

Why study this? “I’m thrilled to join a team translating basic science to help patients with Duchenne muscular dystrophy.”

Gianvito Masi, MD

Institution: Yale University, New Haven, Connecticut

Development Grant project title: Investigating Mechanistic Drivers of MuSK Autoimmunity

Research focus: Investigating factors that contribute to the initiation and relapses of muscle-specific tyrosine kinase (MuSK) myasthenia gravis (MG)

Why study this? “MuSK MG is a chronic neuromuscular condition characterized by IgG4, an antibody with intriguing yet still poorly understood properties. Understanding the mechanisms that drive MuSK IgG4 production could inform future therapeutic strategies and have a tangible impact on the care of MuSK MG patients and those with other IgG4-mediated conditions.”

Hichem Tasfaout, PharmD, PhD

Institution: University of Washington, Seattle

Development Grant project title: Development of Next-generation Therapies for Duchenne Muscular Dystrophy

Research focus: Developing a new method to make the dystrophin gene, or any large gene, transportable in an AAV vector for gene therapy.

Why study this? “This project tackles a long-standing challenge faced with AAV vectors. The goal of this award is to optimize further this new approach for efficient and safe applications.”