Alisa May, Caregiver to Son with ALD and Advocate for Innovation and Access

Alisa May serves as a caregiver to her son Justin, who suffers from adrenoleukodystrophy (ALD), a rare genetic condition that prevents the body from breaking down very long-chain fatty acids in the brain. When these acids accumulate, they destroy the protective sheath of nerve cells responsible for brain function.

Alisa’s father passed away due to complications from ALD at age 56. When Justin was born with the same condition, she prayed for a miracle and became an advocate for scientific innovation so her son would not succumb to the same fate as her father.

After more than 19 years managing his condition at home, it has been a challenge for Justin to manage his adrenal insufficiency on his own during his first year of college. Without working adrenal glands, Justin’s body cannot fight off the disease. As long as Justin is not under any stress, his symptoms are manageable. When stress ensues, it is vital Justin has access to steroid medications to ensure his adrenal glands work properly.

Alisa knows how lucky Justin is to have medication available that keeps him healthy and active but also knows that more treatment options are needed for himself and future patients. If for some reason Justin’s current medication could no longer support his health, they may have nowhere else to turn. That’s why every single day Alisa prays for a cure – or at least another treatment – to be discovered for ALD.

As an advocate for her son and for others with rare diseases, Alisa is concerned about proposals in Congress that could disrupt our innovation ecosystem and slow the development of new treatments and cures. Specifically, she worries government price-setting policies could prevent life-changing therapies from being available to patients entirely or restrict patient access to existing treatments.

For Alisa, knowing researchers and scientists are pursuing new treatments and a cure for ALD gives her hope for a brighter future for Justin, and the last thing she wants to see are misguided policy proposals rob her of that hope.