Interesting drug trials that I'm watching

The amount of information I'm seeing out there regarding SMA research has just been exploding over the past year. If you have an interest in SMA research--or stem cell research in general, even--read on. I've tried to highlight the most interesting things that I've noticed lately, but surely for every one I see there are a dozen more exciting studies and papers that I am missing.

Last month saw the publication of a German study using lab mice with SMA. The drug is a potential treatment, not a cure, but the researchers seem to think it could be better than CarniVAL, which the babies are currently taking on an investigational basis. The drug is called Saha, and it's actually already on the market for the treatment of cancer, which makes it much easier for us to get our hands on if it's eventually found to be effective in increasing strength in our SMA kids.

Also biotech companies Repligen, deCODE Chemistry, and Invitrogen (and others?) continue to study quinazoline. This drug looks very promising and has "prolonged survival significantly" in SMA lab mice. Quinazoline is a new drug, and so is not currently on the market for any other indications.

Both of these drugs are in the pre-clinical stages of research, which my personal clinical research expert in the next room here tells me means that the research firms involved still have to apply to the FDA to begin their human trials. If their applications are approved, human trials could start in anywhere from two months from now to five years from now. Still, it's nice to know that someone is doing something, right?

My other interest is around U.S. stem cell trials in humans. As everyone knows by now, it took Barack Obama exactly 43 days in office before he reversed the embryonic stem cell research funding ban last year. Like it or hate it, the ban is in fact lifted, and federal dollars can now be used to support some heavy duty stem cell research.

I like it.

SMA is one of the conditions being seriously considered for stem cell treatment here in the U.S. It's been done in other countries, but documentation of these cases is nearly non-existent and what is out there is entirely anecdotal. It's hard for me to wrap my mind around the thought process behind injecting a child with stem cells, but not documenting or publishing the procedure and not having any before/after metrics to even show if the stem cells worked at all whatsoever. I certainly don't judge the families who have chosen to go this route--if you search my archives here, you'll see that if I'd had $50,000 to spare last December, I would have already been to China and back. Twice. Literally. The parent of a child (or children, heh) with SMA is a desperate parent, to be sure.

Here is a basic primer, sort of a Stem Cell Research 101.

So, even though embryonic stem cell therapy on humans has been performed elsewhere, it's not been done in the U.S. or done under the strict regulations of the good old Food & Drug Administration. I bet someone out there has some harsh words regarding the inefficiencies of the FDA, and while I'd love to hear your arguments, that's not really my agenda. Instead, I want to highlight some of the really amazing progress being made here in the U.S. to advance stem cell trials to human subjects. If we're smart, we'll all buy stock in these companies.

California Stem Cell and UC Irvine are planning their human trials for a motor neuron replacement therapy, derived from stem cells, on SMA type 1 patients to occur sometime this year. The Families of SMA have invested in this project over the years, and I can't wait for more information to become available on this trial. For SMA families, if you have heard of Dr. Hans Keirstead, this is the trial in which he is involved.

Geron, a San Francisco based firm, is planning to begin human trials on victims of spinal cord injury in 3Q 2010. According to their product pipeline, Geron is focusing on researching the use of human embryonic stem cells (hESC) in not only spinal cord injuries but also heart disease, cancer, and even diabetes.

This month, Maryland-based Neuralstem announced a human clinical trial at Emory University for ALS patients. The trial involves injecting human embryonic stem cells into the spinal cord, and the first patient has already received treatment. The researchers emphasize that they expect this only to slow the progression of the disease, but that it is not a cure. One notable fact is that it has taken Neuralstem only four months to go from FDA approval to their first injection of stem cells.

What do you think about any of this? Or if you know of something important that I've missed, share that, too.

My next project. Ahem--I'm talking to YOU, PVUSD.

My pregnancy with the twins was a high-risk pregnancy, and I fought to stay pregnant with them for thirty-six weeks and four days. I did this through enduring 11 weeks of bedrest, managing well my gestational diabetes, giving up general possession of my body (sigh), and taking the medications my doctors told me to take.

My twins' diagnosis of SMA was even tougher, but I fight for them by taking them to the best specialists around, in-state and out-of-state. And I fight for them by adhering to a strict germ-free lifestyle and by subjecting them to daily respiratory treatments and yucky-tasting medicine.

My twins needed expensive respiratory equipment last year--which our insurance company deigned to pay for--but before I could fight hard for that, the manufacturer donated it. All of it. Muchas gracias, Respironics.

My twins deserve power wheelchairs in order to move themselves. Studies show that toddlers are developmentally able to manage powerchairs as early as 18 months of age. We applied for their powerchairs at age 24 months, and I fought for eight months to get Lauren's powerchair. Kyle's paperwork was inexplicably put on hold by our insurance company, so the fight for his chair is just beginning.

My twins qualify for an amazing preschool curriculum offered by our state/school district. I met the staff there and toured the facility on Tuesday. The twins will love it! The adults-to-children ratio is 1:5; the classrooms offer challenging yet developmentally appropriate centers and activities; school bus transportation is included for free--including for wheelchair-bound children; there are physical, speech, and occupational therapists for every classroom. There are even a handful of "typical" children in each classroom to act as models for the rest, and those kids pay handsomely for the education and experience.

However.

Sigh.

This special-needs preschool has two playgrounds, and neither of the playgrounds are wheelchair accessible. Yes, the school built especially for special-needs kids, with a playground designed by their physical therapists, excludes my children from participating in that activity, and by design. The playground, where arguably some of the most important preschool social experiences take place, is off limits to my babies due to their physical limitations. Wha...?

You want a fight? I can give you a fight. Are you familiar with the roar of a mother lion?

I fought for months to bring them into this world. I fight the daily fight to keep them happy and healthy. And--oh yes--I shall fight to get them the accommodations they need to board that playground and play with their friends. Reference this info from the University of Colorado and this useful advice from Kompan, a playground equipment manufacturer.

Paradise Valley Unified School District? Get ready. I am a whole-lotta woman to deal with, and you're about to meet me. You have 6 months.

It. Is. On.

Mars and Venus, I'm telling you.

Ways I've noticed that boys and girls are different:

The girl smells the flower.
The boy plucks every petal off and throws it all to the ground.

When the girl is finished eating, she pushes her plate out of the way and asks, "Dessert, please?"
When the boy is finished eating, he flings his cup and plate onto the floor and screams, "Moooooooooom!"

The girl lines up her Disney Princesses in an orderly fashion in front of her and talks to them.
The boy stacks up blocks and knocks them down over and over.

The boy maniacally sprays out messy zerberts.
The girl replies, "Please, don't spit on me."

I swear, I've raised them exactly the same--They're twins. I am with both of them together the same amount (read: all of the) time, treat them the same, I use the same language and vocabulary for both. And somehow, after 2.5 years, the girl seems to be 100% civilized while the boy is still bordering on neanderthalic (unless you're going to tell him one day that I said that, in which case I mean to say "the boy is just hunky dory with me").

What other examples do you have to share?