The Fifth of July

Cal received her official diagnosis of late-infantile onset metachromatic leukodystrophy (LI-MLD) on July 5th, 2012.

Pat and I had returned to the hospital to confirm the test results and meet one of the two doctors at CHOP who treated children with leukodystrophy.

The young doctor's name was Amy Waldman.

To be sure, going back to the hospital struck me as a futile exercise.

Cal, who was just 2, was not expected to survive beyond the age of 6. It seemed to me there was nothing to be done but plan for our Make A Wish vacation and "enjoy the time we had left."

My rage at the doctors for having no way to save Cal left me betrayed by the promises from all those children's hospital telethons soft-focus celebrities blathering on about miracles.

When people offered to pray for Cal or advised me to cling to hope, I wanted to scream how they were fools.

Hope and miracles had become a cruel joke in a world where Cal could not be saved.

During our meeting that day five years ago, the meeting that I had not wanted to attend, but the meeting that would change my life: Dr. Waldman made me a promise.

Actually, it was more solemn than that, it was more like a vow.

I had explained to Dr. Waldman that I understood Cal would die, but I had read enough to know that one day MLD might be cured.

And all I wanted now for Dr. Waldman to do was to find me when there was a way to help kids like Cal.

When that day came, I asked Dr. Waldman to find me so that I would "get on a plane to Helsinki or Paris and meet the child the doctors would save from Cal's fate. And if I am really fortunate," I told Dr. Waldman through my tears, "this child will be a little girl with dark eyes and curly hair and she will remind me of my Cal."

Dr. Waldman nodded, she would find me when that day came..

When I asked Dr. Waldman, how long it would take to cure MLD, Dr. Waldman guessed: "we are 20 years away."

In this vision of the future, Cal would be gone, and I would be much older.

It was a dream that would help me fall asleep at night. I would dream of meeting the little girl like Cal and her mother who would not have to suffer as we had.

Over the next months, to cope with our anguish, Pat and I started raising funds for CHOP and Dr. Waldman.

We did not know what to do with the money from the cupcake sales and with the checks our friends and family sent in the mail. Pat and I had talked about a mural project in the hospital's roof garden or even a gift to the palliative care program. It was on the year anniversary of Cal's diagnosis with the publication of Dr. Biffi's paper in the journal Science about a groundbreaking gene therapy, Dr. Waldman made a new suggestion.

It would not take 20 years to defeat MLD, Dr. Biffi's research promised to change what it meant to have MLD a year after Cal's diagnosis. Dr. Waldman assured me that Dr. Biffi's research "was the real thing."

"You know, you need to take the money you are raising," Dr. Waldman said. We had about $20,000 at that point, and "the next time we find a child who is eligible for this trial in Italy, you can help that family to Italy."

Dr. Waldman's idea was absolutely ingenious. We could leverage the money we had to support families and research at the same time. It would take years to raise a million dollars for a gene therapy trial. Getting the children to Milan could be our investment in a medical breakthrough that could prove to be a miracle.

I will confess it was not easy to do this. My first reaction when Dr. Waldman shared this idea was to say:

"Clearly, you have mistaken me for a brave woman."