MyTomorrows, a global health technology company, has partnered with the CureLGMD2i Foundation to improve access to clinical trials for individuals affected by Limb-Girdle Muscular Dystrophy (LGMD). This collaboration aims to address the challenges faced by patients, caregivers, and healthcare professionals in navigating the complex landscape of LGMD clinical research and treatment options.
Addressing the Challenges in LGMD Clinical Trials
LGMDs are a group of rare, inherited neuromuscular disorders characterized by progressive muscle weakness and wasting, particularly around the shoulders and hips. With over 30 subtypes, LGMD presents significant challenges in diagnosis, treatment, and clinical trial enrollment. Genetic testing has become crucial for precise diagnosis, enabling more personalized care. However, the rarity of each subtype makes it difficult to find enough patients for clinical trials, hindering drug development.
Kelly Brazzo, founder of CureLGMD2i, emphasized the confusion patients face when trying to identify appropriate clinical trials. "Which trial is available for me? Do I qualify? How do I get enrolled?" she asked, highlighting the need for a reliable and accessible resource. The partnership with myTomorrows seeks to provide a safe space where patients can find accurate information and support.
MyTomorrows' Role in Facilitating Access
MyTomorrows' search engine offers patients and physicians easy access to worldwide LGMD2I/R9 pre-approval treatment options. Expert patient navigators clarify clinical trial terminology and provide ongoing support to help patients make informed treatment decisions. Dennis Akkaya, Chief Commercial Officer at myTomorrows, explained that their service supports patients and their physicians with questions and interest in clinical trials, helping them navigate the complexities of clinicaltrials.gov.
The Current State of LGMD Research
Despite the challenges, there has been significant progress in LGMD research. "Thankfully, several family foundations and private funds have invested in research over the last 20 years, and now we have three clinical trials," Brazzo noted. However, roadblocks remain, including the cost and logistics of traveling to trial sites, as well as concerns about invasive procedures like muscle biopsies.
The clinical pipeline for LGMD includes a Phase 3 trial for a small molecule drug that replaces a sugar molecule affected by the FKRP gene mutation caused by LGMD2I. This trial is fully enrolled, and researchers are hoping for accelerated approval. Additionally, there are gene therapy programs in Phase 2, which represent a significant advancement in treating genetic diseases. However, questions remain about long-term effects and eligibility for multiple therapies.
The Importance of Awareness and Subtyping
Greater awareness and attention from the clinical community are needed to address the complexities of LGMD. With the increasing number of identified subtypes, accurate subtyping is essential for effective treatment. Brazzo highlighted the challenges faced by patients with variants of unknown significance and the need to push for further research to identify treatments for these individuals.
Encouraging patients to get involved with specialists, register in patient registries, and participate in natural history studies is crucial for advancing LGMD research and treatment. The LGMD community is working together to expedite drug development and improve the lives of those affected by this rare disease.
