4. Don’t shoot the messenger. If you decide you want to take the uphill path and try to get your child into a clinical or safety trial, you need to know most research for extremely rare diseases is parent-driven. This means that possibly before your child was even born, there have been families raising private funds to convince a researcher to work on some unheard of disease, discover the gene responsible for causing it, support the gathering of preclinical data, meet with hundreds of people, and basically do everything necessary to get to the point where you come into the picture.
Most of the charitable organizations trying to help find cures were started by parents of sick children. The goal is always to find a cure, but sometimes we must first develop medical interventions that can slow or halt progression of a degenerative disease in order to buy time for our kids. This is agonizing because we have to wait, and worse than waiting, we have to raise money. When new families ask me what is being done and why it’s taking so long I tell them because we need more money, tons more money — whatever we have is never enough. We need government money, biotech money, and we will always need privately raised funds. It’s unfortunate, but sadly if you want your child to receive some type of experimental treatment and you want it sooner than later, you might need to help raise money.
You absolutely do not have to help. You don’t have to do anything. But if you choose not to help, then often you will be relying on others to determine the fate of your child. If you choose to help and decide to join forces with other families who share your goals, then don’t be shocked or upset when the issue of fundraising comes up, again and again. And remember this is always your choice.
5. Know exactly where the money you raise is going. If you decide to become involved in raising money for a cure, you’ll probably find several places to send your money. This can be tricky, because if you want the money you have raised to go directly to research, or to a specific researcher, then you need to ask the important questions and fact-check. Ask the person who controls the money how and if you can earmark it for a specific purpose. Most organizations have a way to collect money from families who wish to help, but they might not be financially supporting the researcher who is working on the cure for your child’s disease every single year. Or they might not have a way to earmark funds in that way. It’s not always as simple as sending in a check and writing a researcher’s name in the memo — that almost never works unless you have made proper arrangements in advance. If you send money to an organization, there is a good chance that those funds can be used to support the organization and not the research you want. Unless you are the director of a charitable organization, those are questions you might want to ask. Oftentimes you have an option to support the research team directly. This way you have control of the funds and can dictate exactly what they are to be used for. When in doubt, ask a lot of questions and check online because finances are all public record.
6. Securing medical treatment for your child can become a full-time job. Often with no pay and sometimes with a ton of out-of-pocket expenses. There can be lots of travel, uncovered medical bills, and even a loss of work due to surgeries and follow up testing. It is a great deal of work and can go on for months or even years, but sometimes there is no other way to save your child’s life.
7. Research and become an expert on your child’s disease. If you are planning to search the world for something to help your child, then learn more about the disease than any average person should know. Study everything being done that concerns the disease. Become familiar with every known vector, stem cell, and animal model. Study everything, because when you speak to the team who is doing the work, you will need a medical background to understand them. Yes, you should always speak directly to the researchers yourself if humanly possible. Don’t be afraid to become overly involved in the process. I might have been the most annoying parent ever, but my son has had gene therapy twice, so I have no guilt or shame about the extreme measure I had to employ. If you need to start your own foundation to get things done, then do it.
8. Pray for a miracle, but don’t expect one. This road is paved with delay, disappointment and tears. Set high expectations, but don’t fall apart if things take too long, or the cure seems impossible. This requires thick skin, and it can hurt more than anything you thought possible to watch your baby deteriorate while it feels like you are standing helplessly by. Just keep working towards your mission, and hopefully treatments, and one day a cure will come. There is no guarantee. My son had his second gene therapy almost 15 years ago and no new trials have materialized since then. But I’m never going to give up. He’s not cured, but he’s still here and sometimes that’s all we can hope for. I’m still working towards discovering the cure and praying for a miracle.
9. If you are lucky enough to get your child enrolled in a trial, please know exactly what you’re getting into. I can vividly remember one of the doctors saying to me, “you do know you might just be extending his life, right?” It was exactly what I wanted. I never dreamed that my son would be about to celebrate his 19th birthday. He is still profoundly disabled and requires full-time care. He is and will probably always be 100 percent dependent on me for his survival. I knew exactly what I was getting into.
If you have a child who is like my son, please look ahead and be absolutely certain participating in a trial would work for you, before you take drastic measures to medically extend his or her life without really curing them. This can and does happen, because with such outstanding care comes a longer life. These cute little babies and kids will grow into adults, adults that love you and need you for their physical and emotional wellbeing. My son will have a place in my home until he takes his last breath. He’s thriving, smart and happy, and he is the love of my life. This is exactly why I fought to keep him alive. However, because each parent has a different outlook, it is beneficial to bring to light a topic people sometimes overlook or don’t want to think about. So, just know what you’re getting into.
10. Don’t ever give up hope. Sometimes hope is the only thing we have. If you don’t cling to hope, then you won’t keep pushing towards your goal of saving your child. I’m not saying this always works, because I’ve seen too many children lose their battles way too early in life. But if you dream of a cure, just keep hoping and working towards it, and maybe one day it will happen. I know my son will most likely never be cured, but he’s been helped and I hope that my continued efforts to help him will one day result in the cure for Canavan disease.