The Honorable

Lamar Alexander
455 Dirksen Senate Office Building
Washington, DC 20510
Dear Mr. Alexander,
My name is Shirley Walker. I live at 7365 Millsfield Highway, Newbern,
TN. My home telephone number is (731)-627-2448. In June of 2012 my
husband of 53 years passed away after a 28 year struggle with
lymphoma. In August 2013 my only child, Todd, was diagnosed with
ALS (Lou Gehrigs Disease). He is now totally paralyzed and ventilator
dependent but by the grace of God, he still has the ability to speak. As
you know, ALS is a currently a fatal disease, but you have the ability to
change that description.
I am writing to express interest in the approval status of a bio-drug
produced by Genervon Biopharmaceuticals LLS, known as GM604 or
GM6. Because of the drugs safety profile and statistically-significant
efficacy, this drug was granted a fast track designation for the
treatment of ALS in 2013 and granted orphan drug designation by the
FDA in March 2014 for the treatment of ALS. However, it is still
unattainable for ALS patients.
On January 8th, 2015, Genervon announced that it had submitted
results of its single compassionate patient trial (GALS-C) to the FDA of
drug candidate GM6. Last month (February 2015), representatives
from Genervon met with the FDA to request accelerated approval of
GM6 for all ALS patients. They supplemented their case with evidence
of demonstrated safety and statistically-significant efficacy between
treated and placebo groups in biomarker data.
I fully appreciate the vast importance of the FDAs comprehensive and
thorough approval process, and the role of the agency in protecting
public health. But as one who gives care daily for my son with ALS, I
also know the tremendous hardships this devastating disease places
on him, his family and me.
Therefore, I respectfully request and urge that Genervon be given full
and fair consideration as they discuss the need for accelerated
approval of GM6. Waiting for a phase 3 trial will result in certain death
for thousands of ALS patients, including my son. Currently Riluzole is
the only one FDA approved drug for ALS and only increases the life
span of a small number of patients by a few months.

The best hope is to target the FDA via the Senate H.E.L.P. Committee.
Please exert your powerful influence as Committee Chairman by
insisting the FDA be flexible so GM6 can reach patients who need it
now while also collecting additional information from patient
experiences and ongoing non-randomized trials that can lead to full
approval of the drug. The patients would get access to GM6 while
scientists get their data collected over a longer period of time.
It is urgent that the Senators on the H.E.L.P. Committee help change
the way ALS patients are able to access new drugs and treatments that
could change the course of ALS progression and prognosis. That can
start now with Genervon's GM6.
Thank you for your consideration in urging the FDA to allow for
accelerated approval of GM6. Doing so could mean rescue from ALS
for many tens of thousands stricken with this terrible disease as well as
prevent this disease from striking thousands of others.
Sincerely,
Shirley Walker