• “My gift is my song and this one's for you”

    - Elton John

Posted: December 3rd, 2013

Over the past few months, one of the patients in our Phase II trial to treat ALS has been posting blogs and pictures as she goes through the process of enrollment, surgery and recovery. Her most recent blog can be found here.

It is a touching and fascinating look into the human side of what is one of the most advanced neuroscience projects in the world. As readers, we see all of her fears and her hopes laid bare. We see how she copes with the everyday realities of being an ALS patient, and we get a feel for how difficult it is to recover from an invasive surgery.  

We have now completed the second cohort in the Phase II trial. We are of course focusing on cervical area injections in this trial, and we are increasing the number of injections and the number of cells in each injection over the last trial.  But one only has to read April’s blog to understand how brave and giving the patients have to be to volunteer for this trial.

It surprises people when I say that, and when I mention how grateful we are to our patients and their caregivers. Given the horrible nature of the disease and it’s always fatal consequences, people just assume that these are patients with “nothing to lose.” But that is not true. All of our patients come to us with some quality of life, and some significant amount of “time” left, whether we measure it in months or years.  In the case of our Phase II patients, who are all within two years of the onset of symptoms, this is especially true. And we have designed this trial to guard jealously that quality of life.   

People are also surprised to find out, that the information in these patient blogs is as new to me as it is to them. April has not had (as of her last blog) her official quarterly check up and measurements. Getting a kind of minute by minute, day by day accounting is indeed a gift. We do not know how April’s story will end; the Phase I trial has left us hopeful, but this is still very much a “trial,” not yet a treatment.

 If all goes well, we will transplant the third cohort in January, and the fourth cohort in March. Then, in May that fourth cohort will return for lumbar injections completing the trial. The trial’s data lock will come 6 months after that last surgery; possibly about a year from right now. There may be interim data that is published as the trial moves forward, but it’s hard to know at this point just when that will occur.  Until then April’s song is indeed her gift to all of us.

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