The Lucas Project: How You Can Help Boys with ALD

 

Lucas’ Story

When Lucas was 3 months old we were notified that he tested positive on the newborn screen panel for Adrenoleukodystrophy, otherwise known as ALD. If you were to Google it, you would likely find a movie called “Lorenzo’s Oil”, or stories of young boys diagnosed with ADHD, who then lose their sight, speech, ability to walk and move, and ultimately die from brain lesions by the age of 7. That was the fate of boys diagnosed with this disease until it was added to the newborn screen. Sadly, many states still aren’t testing for ALD and will continue to lose their sons to this horrific disorder. However, the fate of boys with this diagnosis has been dramatically changed. With early detection, boys are living full, long lives.
 
ALD is a rare genetic disorder in which the ALD protein is missing or malfunctioning in cells. When this happens, it creates elevated fatty acid levels that can potentially cause problems. There are many ways this disease can manifest. One is through adrenal dysfunction, also known as Addison’s Disease, which can easily be managed with medication. Another is problems with the spinal cord. It is possible that over time, a person with ALD will have gait and walking issues and/or could potentially be wheelchair bound, but continue to live normal lives. However, there is one very serious form of this disease called Cerebral ALD (cALD) in which the brain is affected and can ultimately take a person’s life quite quickly. cALD usually occurs in childhood with the average onset of 4-10 years old. It is possible for this to occur later in life, but less likely.

Tragically, many families before us have lost their sons at a very early age to this disease. However, because of these courageous families, there have been so many advances in treatments. Specialists have found that if we can detect brain lesions early, a child can receive interventions to halt the disease in its tracks. This is the very reason it was added to the newborn screen. Monitoring a baby from birth will allow doctors to catch the disease early and intervene before it becomes problematic.

This rare disease was added to the newborn screen panel in California in September 2016. Lucas was born in April of 2017. Had Lucas been born just 8 months earlier, we would never know this information.  Currently, there are only 11 states that have added detection for ALD to the newborn screen. We are very grateful that we live in a state that approved it and that Lucas was born at the right time.

What’s next?

So, what now? Lucas will be monitored with bloodwork every 4-6 months and MRIs every 6 months until he is about 12 years old, and then it will move to every year. If Lucas should develop adrenal dysfunction, he will go on medication. If he should develop a brain lesion, he will need to have a bone marrow or stem cell transplant, or he will have gene therapy. This is not a cure, and it’s risky, but it’s a promising treatment. (Consider becoming a marrow donor: http://bahbad.org/)

Currently, Lucas is doing very well. No adrenal issues to date and he has had 2 MRIs with all clear results. He is just like any other 3-year-old, but we would argue he is more of a superhero, as nothing holds him back.

What can you do?

Lucas is a part of a study on the preventative interventions for ALD. His neurologist, Dr. Van Haren, is a national leader in ALD research and is studying the effects of Vitamin D on the prevention of ALD (among other studies). Lucas currently takes 2,000ius of D per day. Additionally, because of Dr. Van Haren’s advocacy, ALD was added to the newborn screen in California. He played a critical role in allowing us to know about Lucas’ diagnosis. Dr. Van Haren and his team spend countless hours working with ALD patients and researching advancements. We are so grateful that he is leading this and other studies on ALD.

This is where we are focusing our efforts. If you could please help us reach our goal, we would be forever grateful as his research can change the fate of lots of boys. Unfortunately, because ALD is a degenerative disease, time is working against us. The more we are able to fund research, the closer we get to finding a cure. Lucas is approaching the window of his life that could affect him most greatly, and we want to do everything in our power to make every advancement possible. There is nothing more valuable to us and we are forever grateful for your support for our family and our sweet Lucas.

Love, Nic and Alison
#savetheboys 

P.S. We have partnered with Dr. Van Haren and his team at Stanford to create the ALD Family Retreat. The retreat will take place at The Painted Turtle that was co-founded by Lucas’ grandparents, Page and Lou Adler. The camp is designed for children facing life-threatening and chronic illnesses. Over the retreat weekend, the ALD community will come together to enjoy all of the amazing activities at camp and share experiences while also empowering families with knowledge and tools for living with an ALD diagnosis. We are inviting specialists in neurology, endocrinology, transplant, nutrition and alternative medicine to discuss the latest findings, best practices, and what the future holds for our loved ones. 

Learn More

Learn more about Van Haren Lab.

Join the Fight

Make a gift to Dr. Van Haren’s Research. All donations are 100% tax-deductible. 

Please mail checks payable to LPFCH to:
Lucile Packard Foundation
Attn: Development Services
400 Hamilton Avenue, Suite 340
Palo Alto, CA 94301

Please note: Write-in “The Lucas Project” in the memo section on the check. You can also enclose a separate note with this information if there is insufficient space in the memo line of the check.

WHAT IS ALD CONNECT?

ALD Connect is a non-profit organization that brings together patients, families, physicians, scientists, advocates, and industry.

Our mission is to improve health outcomes for patients with X-linked adrenoleukodystrophy (ALD) by empowering patients, raising awareness, and accelerating the translation of scientific advances into better clinical care.

Lisa Ellen Niver

Lisa Ellen Niver, M.A. Education, is a science teacher and an award-winning travel expert who has explored 101 countries and six continents. She sailed the seven seas by cruise ship for seven years and backpacked for three years in Asia. Find her talking travel at KTLA TV and in her We Said Go Travel videos with over one and a quarter million views (1,250,000) on her YouTube channel. She is the founder of We Said Go Travel which is read in 235 countries, named #3 on the top 1000 Travel Blogs and the top female travel blogger 3 times in 2019. She has hosted Facebook Live for USA Today 10best, is verified on Twitter and has over 160,000 followers across social media. Niver is a judge for the Gracies Awards for the Alliance of Women in Media and also ran fifteen travel competitions publishing over 2500 writers and photographers from 75 countries on her own site, We Said Go Travel. From 2017 to 2020 in the Southern California Journalism Awards and National Arts and Entertainment Journalism Awards, she has won three times and been a finalist fourteen times for her broadcast television segments, print and digital articles. Niver won an award for her print magazine article for Hemispheres Magazine for United Airlines in the 2020 Southern California Journalism Awards. She was also a finalist for four other categories including online journalist of the year, digital story for activism journalism with Ms. Magazine, educational reporting for Wharton Magazine and a broadcast lifestyle feature for KTLA TV in Los Angeles.    Niver won a 2019 NAEJ (National Arts and Entertainment Journalism) award for one of her KTLA TV segments and was a finalist for articles published in both Ms. Magazine and Wharton Magazine. In 2018,  she was a finalist for stories in Smithsonian, PopSugar Fitness and the Saturday Evening Post. Niver won a 2017 Southern California Journalism Award for her print story for the Jewish Journal and was a finalist for travel reporting. Niver has written for AARP, American Airways, Delta Sky, En Route (Air Canada), Hemispheres (United Airlines), Jewish Journal, Luxury Magazine, Ms. Magazine, Myanmar Times, National Geographic, POPSUGAR, Robb Report, Saturday Evening Post, Scuba Diver Life, Sierra Club, Ski Utah, Smithsonian, TODAY, Trivago, USA Today 10best, Wharton Magazine and Yahoo. She is writing a book, “Brave Rebel: 50 Scary Challenges Before 50,” about her most recent travels and insights. Look for her underwater SCUBA diving, in her art studio making ceramics or helping people find their next dream trip.  http://lisaniver.com/one-page/

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