FDA has approved the treatment for Benny & Josh!
Congratulations! Our nearly 3.5-year journey to Save Benny and Josh has crossed another milestone. Late last week we received a letter from the FDA stating, "you have satisfactorily addressed all clinical hold issues…(the FDA has) removed the clinical hold and you may proceed with your study". (See our press release here.)
Benny & Josh
As I’m sure you can imagine we are elated! We are also relieved... & nervous. But we are taking a few well-timed days to savor this accomplishment & enjoy some family time.
OK… ”what's next?” you ask. Well, we now have some more administrative' stuff to deal with such as signing contracts with the hospital and gaining official approval from the hospital board. We have been told that this is standard practice and there is nothing to worry about, but as you all know we’ve heard this before. Please g-d, if all goes well we are looking at the first child being treated in Spring, 2021.
People have said to us, “I don’t know how you do it…” We, (the two of us) didn’t do it. WE, us with YOU, did it!
There are no words to express our thanks to you. Your generosity made this all possible. Your calls & letters of support helped to give us the strength to persevere when all we wanted to do was dig a big hole and jump in. Your acts of kindness have inspired us. And your prayers are being heard - we are sure of it. The next leg of our journey has begun, and we hope you will accompany us on the long road ahead.
People have been asking, what happens next.
Q. What is the actual treatment?
The treatment is a gene therapy that will target the site in the brain that produces the enzyme that Canavan children don’t have. It will be administered via brain surgery.
Q. Will the treatment completely cure the boys?
We won’t know the full effects of the treatment for several months. The range of possible outcomes is anywhere from an improved quality of life to a reversal of the effects of the disease.
Q. Is this a one-time treatment and then they will be “better”?
We have been told that they may need another “dose” (treatment) down the line, but for now, the plan is one surgical dose per child.
Q. Will insurance pay for their medical needs moving forward?
Standard medical needs that are not considered experimental should be covered. There may be some post-surgical rehab that won’t be covered, and any alternative therapy, equipment, or other therapy/rehab may not be covered by insurance.
Q. How can I donate to Benny & Josh’s expenses not relating to the treatment?
We opened a special needs trust which helps pay for alternative treatments, special equipment, and other needs that may arise. This trust is not a 501-C3 charity. You can visit the boy's new Go Fund Me page HERE to donate.
To recap, you, our beloved generous donors have successfully helped us to raise the funds needed for Phase 1 and 2 of the gene therapy medical trial. The charity we began to help fund this treatment, Cure Canavan Fund will continue to raise funds to help treat as many kids with Canavan as possible. So while we have reached our initial goal, we know our work is not over yet. We plan on seeing this trial through so that Gd willing, one day very soon, this treatment will be available for all those in need. Should you wish to make a tax-deductible donation to Cure Canavan Fund you may do so HERE. Beyond your donation dollars, what Benny & Josh really need now are your continued prayers.
Please continue to keep Benny & Josh (full names below) in your prayers and we’ll send more updates as things progress.
Sending love, gratitude, warmth, and blessings for a healthy, happy productive, and easy 2021!
Jennie & Gary, Mikey, Benny, Josh, Evan & baby Racheli.
The boys and their Hebrew names as follows:
Shalom Binyamin & Yehoshua Natan, each ben Shayna Toyba
שלום בנימין בן שיינע טייבע ויהושע נתן בן שיינע טייבע
Cure Canavan Fund is a registered non-profit 501C3. All donations are tax-deductible. EIN# 83-4489648
Smiling Benny & Josh (top). Evan in Fall (left bottom) & Winter (right bottom).
PS. Another congratulation,- the boys have a new baby sister! Rachel Ettel (AKA Racheli, Raquel & Rakeli) born Nov 1, 2020 (2 weeks late) weighing 8lbs. 3 oz. :)