My husband just enrolled in his fourth clinical trial. It’s part of the CIRM-funded UC Davis HD Pre-Cell trial. So far, the experience of being a part of this historical trial has been wonderful and over the coming months I will be chronicling our journey. I hope that through these chronicles the fears and unknowns of clinical trials will be put to ease. Being a part of a clinical trial in my husband’s and my experience is empowering. It gives us hope of a brighter future for Huntington’s disease.
Huntington’s disease is a genetic, hereditary, neurodegenerative disease. I always say it is a horrific, relentless disease because it is degenerative with no remission and it‘s always fatal. One vicious promise that Huntington’s disease gives to all of us is that our offspring have a 50 percent chance of inheritance.
We see Huntington’s disease repeating generation after generation. The reason why I am such an advocate for regenerative medicine is because I believe that this is what could make our generation be the last generation to live in fear and hopelessness when it comes to our fate with Huntington’s disease. Regenerative medicine gives us a hope for an HD free world like no other form of modern medicine or research.
When you get a positive diagnosis for Huntington’s disease there is nothing that anyone can do for you. No treatments, no therapies, no cure, nothing. I remember at one point they said blueberries may help. BLUEBERRIES! Are you kidding me? And of course there’s no proof they work.
I remember when my husband was first diagnosed with HD, I used to always hope when I opened my computer I would see in huge bold letters “The Cure for Huntington’s disease has been discovered”. I quickly learned that was wishful thinking. One of my dear HD advocate friends said something so wise to me. She said:
“We will never wake up one day and find the cure for HD has been found. We would have heard about the research for a long time. We will battle for funding for the research. We will watch it go through human clinical trial. Then we will eagerly wait on the edge of our seat to see if the FDA approves the treatment. It will take years and we will play an important part in it all.”
At that point, one thing became very clear to me, very fast. We will never have a treatment, therapy or cure for Huntington’s disease if we don’t stand up and support our researchers 100 percent. When I first started advocating for Huntington’s disease, over seven years ago, I didn’t really understand what it meant to bring a brighter future to our HD community. Over the years I have learned some things we have to do in our generation to change the HD world for the future generations.
The FDA’s mission states: Protecting consumers and enhancing public health by maximizing compliance of FDA regulated products and minimizing risk associated with those products. The FDA is there to protect the public. They will never let a drug, device, or therapy like stem cells go to market without first going through human clinical trials.
Clinical trials are important to make sure the potential therapy or treatment is safe and effective. So we will never have a treatment or cure for Huntington’s disease unless it goes through human clinical trial. Humans—that’s us. We have to participate to make our dream of a treatment or cure a reality!
Stem cell agency
My favorite mission is that of CIRM, the California stem cell agency: “To support and advance stem cell research and regenerative medicine under the highest ethical and medical standards for the discovery and development of cures, therapies, diagnostics and research technologies to relieve human suffering from chronic disease and injury.”
In order for the stem cell agency to ever be able to achieve their mission, patient’s and caregivers have to step in and do our part. None of this will work without the patient advocates. This is where we come in. Patients and caregivers are just as important in bringing new treatments and therapies to market as the researchers and industry. Without our participation in the processes we will never see any advancements in medicine. Without us standing up and participating in clinical trials all the work of the researchers will never see the future.
I think that there are so many stigmas and unknowns when it comes to clinical trials. I know the first clinical trial my husband and I participated in we were a little apprehensive. We didn’t know what to expect. I read all the paperwork we were given about the trial but I was still nervous walking up to the hospital on our first screening visit.
The trial my husband just enrolled in was so much easier. My husband and I walked up to the VA hospital, in Sacramento where the UC Davis HD trial is taking place, full of excitement and hope. This is because we no longer have fear when it comes to clinical trials. We also realize that our participation in the trial is extremely important. We want to see a therapy or cure for Huntington’s disease for our family and our HD community. We realize the only way that will ever happen is to play our part in the process.
My husband and I are so excited to be a part of Pre Cell, the First-In-Human trial in the world for a potential therapy for Huntington‘s disease. This is groundbreaking news for everyone in the world that is affected by Huntington‘s disease. The world is watching with excitement of what this historic trial could do for the Huntington’s disease community.
Let me finish this chronicle by saying the Huntington’s community is a strong force. Seeing as how this is a inherited disease we have seen too much pain and suffering in our past generations. We refuse to make that be the case for our generations to come. I feel blessed to be a part of this movement that is happening in Huntington’s disease. But let me tell you that I know that none of this would be happening if it wasn’t for the strong advocates in the HD community standing up and making all of this movement happen. I am proud to be a Huntington’s patient advocate and be a part of all of this groundbreaking change.
There’s more information about the disease and other awards CIRM funds on the Huntington’s disease fact sheet.