WKBW Buffalo recently interviewed ALS patients who are advocating for a bill that would grant access to clinical trial drugs during Phase III and before they go through the long and complicated FDA approval process.
37 year old ALS patient Kristin Goetz reminds us that currently there are no medications to reverse the course of ALS. Kristin has been diagnosed with ALS and told, as are so many other ALS patients, that she may only have between two and five years to live.
Kristin recalls walking out of the doctor’s office after her diagnosis holding a big red folder emblazoned with ALS on the cover and feeling confused. The next few weeks were crowded with thoughts of immediately getting her affairs in order.
Kristin still remembers one incident that had an impact on her. It was a comment made by a woman in response to Kristin saying she wants to buy a house. The woman said rather sternly, I do not think it is wise to buy a house “in your condition.”
This stunned Kristin as it was something she had always wanted to do. She could not see why her “condition” should affect that decision.
At that very moment, Kristin decided that she would not let ALS define or defeat her. She admits that her diagnosis has been difficult to accept, but it has taught her how important it is to remain positive.
Yet the path to find a treatment for Kristin is made more difficult by the fact that she is not eligible for most clinical trials. Kristin explained that most trials require a diagnosis within two years and she is now well past the two-year mark.
Kristin is joined in her advocacy by Michelle Lorenz, the co-founder of Voices for ALS. Michelle agrees that ninety percent of ALS patients are not eligible for most clinical trials.
Michelle gave the analogy of being on the Titanic, knowing the ship is sinking, and that they are facing death. She described patients who are begging and crying for access to drugs and says simply “they’re dying.”
Another ALS patient advocating for the bill has already lost her ability to speak. 37 year-old Mayuri Saxena communicates by way of a computer program that she directs with her eyes.
Mayuri bravely communicated that ALS may have taken her ability to move and to speak, but she will not let it stop her from being an advocate for ALS patients.
About ALS
Amyotrophic lateral sclerosis (ALS) is often called Lou Gehrig’s disease. It is defined by the NIH as a progressive paralysis and fatal disease affecting the nerves (neurons) in the brain and spinal cord. Nerves break down and muscles weaken, affecting voluntary movements such as breathing, walking, and speech.
About the Proposed Congressional Bill
The proposed bill is called the Act For ALS bill. Supporters explain that because their lives have been shortened to such an extent, the FDA approval process is much too long and complicated. Many ALS patients have died waiting for drug approval.
The general feeling among ALS patients is that the bill would at least give them a chance and hope that they do not have now. While they accept the fact that the drugs are not fully approved, they at least would have a slim chance and some hope, rather than the current situation where nothing currently available can prolong their lives.
Readers can help ALS patients by completing the form to contact their representative and ask for action.
