Our History

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LU was founded in 2012 by a small group of individuals living with lipodystrophy who connected on social media and noted that their experience was not reflected in any accessible public information and growing research in the study of the disease was not accessible to the general public and didn’t directly reflect the true burden of the disease. Recognizing the challenges patients face, Lipodystrophy United (LU) was founded with a clear mission: to serve as a bridge between the patient and research communities.

As is common in rare diseases, they had to learn from scratch how to develop a nonprofit from nuts to bolts, become experts in the disease, build the community and create and execute a plan to improve diagnosis and disease awareness. They secured official 501(c)3 status.

They jumped headfirst into advocacy, driven by the fact that a treatment, a recombinant human leptin analog called metreleptin, was already in development clinical trials had been ongoing for patients with generalized lipodystrophy for many years, and trials for partial lipodystrophy were in the early stages. The existing industry and regulatory involvement provided unique opportunities and additional learning challenges. 

Gaining 501(c)(3) status was crucial for Lipodystrophy United, primarily because it offers several financial and operational benefits that are essential for its growth and sustainability. Here are the key reasons...

1. Enhanced Credibility: 501(c)(3) status adds a significant layer of credibility and legitimacy to LU. It reassures donors, stakeholders, and the public that the foundation is recognized by the IRS as a legitimate entity committed to its mission of advocating for patients. This enhanced trust encourages more significant engagement and support from the community.
2. Attracting Volunteers and Board Members: A recognized 501(c)(3) status can also make the organization more attractive to potential volunteers and board members. People are often more willing to invest their time and expertise in an organization that has an established structure and clear tax-exempt status.
3. Tax-Exempt Status: One of the most significant benefits for a patient advocacy foundation with 501(c)(3) status is exemption from federal income tax on revenue generated from activities aligned with its charitable mission. 
4. Tax-Deductible Donations: Contributions made to a 501(c)(3) patient advocacy foundation are tax-deductible for donors. This tax incentive is crucial as it encourages individuals and corporations to donate more generously and frequently, knowing they can deduct these contributions from their taxable income.
5. Eligibility for Grants: Most funding bodies, including governmental and private foundations, require a 501(c)(3) status for eligibility. This status serves as a validation of Lipodystrophy United’s charitable intentions and its commitment to public benefit, significantly broadening our opportunities to secure essential grant funding.

The Food and Drug Administration (FDA) approves drugs for commercial use, meaning patients can ask their doctors for a prescription. The FDA approval process is lengthy and complex. The pharmaceutical company that owned metreleptin submitted extensive data about the drug's effects on the body, specifically on triglycerides and A1C (glucose) levels.

In December of 2013, several LU board and community members provided testimony at the Advisory Committee reviewing the clinical trial results for metreleptin.  They evaluate the drug's effectiveness and safety, essentially determining if the benefits outweigh the risks. Often, the patient's voice is missing in this process. Individuals treated with metreleptin shared their stories with the expert reviewers. LU's President spoke on behalf of the community about the critical need for treatment options and provided support to make it easier for patients to attend and share their experiences. The advisory ruled the data was strong enough to show benefit over risk for generalized lipodystrophy but found the limited data for people with partial lipodystrophy inconclusive, ultimately recommending further studies. In 2014, the FDA approved metreleptin under the commercial name, Myalept®, for generalized lipodystrophy patients.

Metreleptin has different approval statuses in other countries, including Brazil, Canada, Europe, Israel, Japan, and Saudi Arabia. 

Metreleptin was developed by Amgen in 2000 as a hormone replacement therapy to replace leptin, a protein produced by fat cells that is a hormone acting mainly in the regulation of appetite and fat storage.

Since then, LU has represented the patient's voice and shared the burden of the disease to 6 other pharmaceutical companies who have passed on ownership of metreleptin. Currently, Chiesi pharmaceuticals owns metreleptin and LU is actively collaborating with them on advocacy efforts.