Approved! Clinical trial of Leucine for DBA… and more

A new study is being conducted on the use of leucine (an amino acid) to treat DBA. It has just been approved.

Click here to read an easy-to-read explanation of the study, or here for the official clinical trial information page.

Adahlia is too young to participate – participants must be 2 years old at least. I have written the researchers, however, to ask them about dosages and to see if they’ll help me.

We are due for a blood check and transfusion this Thursday. If her retic count isn’t still climbing, then I am considering doing my own leucine trial. We spoke with her specialist about trying leucine before steroids at our last appointment. He basically said that since it is not an approved therapy, he cannot supervise it. As far as the hospital is concerned, our only option is steroids at this point. But I am not willing to give her steroids at this time. She does not have any growth problems or medical issues besides the bone marrow failure. There’s no guarantee that steroids would work for her— but it is all but guaranteed that there would be side effects and growth problems from taking them. I am just not willing to do that to her yet.

This particular doctor and I have a wonderful working relationship. He knows I am dedicated to finding a cure for Adahlia. With a smile, he said that I could find out all I need to know about dosage via the published scientific journal articles. He also said that if I start giving her leucine to let him know so that he can monitor her.

In other words, he supports the idea of trying leucine for Adahlia, but he cannot medically advise me (perhaps, legally) or help me administer it.

So I’ve written these researchers, explaining our situation, and saying:

“I was curious about how you settled on 700 mg/m2/dose. Do you consider that to be a high dose, or do you consider it to be a moderate-high dose? Is it considered safe? Why are you not including infants under 2 years? Is it because of concerns about excess leucine in a sensitive developing system? What concerns do you have in particular? What side effects or adverse effects are you monitoring for?

For a 19 lb child (approx 8 kg, which I believe is 0.4 m2), the dosage would be 700/.4, or approx 280 mg each dose, 3 times daily. Is that correct?”

I’m not sure if they’ll respond. If they don’t, or they don’t want to help me, I’ll try emailing some other people, like the nutritionist in London who first discovered that leucine supplementation could resolve generative anemia like DBA. If that doesn’t work, I need to find a pediatric nutrition specialist here in Portland, ideally with expertise in amino acids. I’m all ears for suggestions.

Truly, this study is a big deal. It’s a big deal for patients and their families. It’s a big deal for natural medicine. It’s a big deal for the growth of our understanding of genetic regulation and expression.

There’s a lot we don’t understand about how leucine positively impacts genetic expression, or allows someone’s bone marrow to begin producing RBCs. If the Chinese herbs work, there’s a lot we won’t understand about that, either. But we can create theories, looking back. The most important thing is to find what works, while doing the absolute least harm.

In Adahlia news, she had a very rough couple of days after we gave her the new Chinese herbal formula (with the Gu focus.) I think it hit her especially hard because we make her take it 3 times per day, now, instead of 2. On Weds, after we gave her the first few doses, she looked absolutely awful. Pale, irritable, weak, distressed; tugging at her hair and generally, obviously, unwell. But now she’s doing much better. Eating better, sleeping better, playing better. She’s still fairly pale, and she looks tired. But she wants to participate in life and look at things, and crawl, and play. We will be taking it easy for the next 5 days. I’m nearly certain she’ll need a transfusion this coming Thursday.

I met a friend for lunch recently. She was blown away by the fact that I am forced to experiment on Adahlia. She couldn’t fathom the stress of giving your own infant daughter droppers of herbs and other treatments, not knowing exactly what the results will be, taking such a gamble. After all, I could mess up. I could harm her. What does chlorophyll or Chinese herbs or cherry juice do to a 9 month infant?

There was a point, back in the Fall, when I was very, very stressed by what I was doing. I felt it was all up to me to figure this out, to cure her or harm her or make the decision that could even kill her.

Now, I still accept that full responsibility. Ultimately, her health has always been my responsibility, not the doctors’. But I have realized that I have I have teams of people, from all sorts of different specialities and backgrounds, that I can call upon for advice and assistance. I am not shy about calling them to the front, to see how they wish to play their hand. This is an opportunity for everyone to be their best self.

And there is a presence that has been with us through this ordeal, that has run me into the right person after right person, just when I needed them, or helped me align with my heart just when I was about to be flooded with opinions (sometimes, there are too many well-intended opinions.)

It hasn’t always been palpable. Sometimes, in the exhaustion and turmoil of emotion, its been silent, as though hidden. But it is there. It is always there. I know it, even though it has been mostly out of reach, even though it seems like a lifetime ago, that it reached out and through someone… was it really me?

And perhaps that’s been the hardest thing for me over the past many months. For those that truly know me, have known my connection to Spirit, or God, or what you wish. They have felt it around me, or through me, or with me. Not always, no I am no Saint, but I have been blessed to carry on my lips and through my heart and hands some powerful, transformational gifts.

And in the past year, it is like I have been left. To feel abandoned by God is the worst thing that can happen to one who has met and been infused with God.

But there is an upside to it. It forces one to realize a deeper depth to all the little epiphanies, little awakenings, that seemed so grand, granted earlier. To come to a place where there is nothing, nothing I wouldn’t do or endure to be reconnected with that Awareness of Being. Nothing more important. Nothing more sublime. Only that Oneness and the remarkable sensation of being Awake.

Of hearing clearly. Of sensing cleanly. Of being able to take something slightly tarnished or warped and reflect it through a prism in my own being, and give it back to that person, pure.

Not my own doing. I merely disappear.

Surely it is sacrilegious to say it, but for all Jesus’ understandings, did he not suffer extraordinarily at the end? Was he not mocked? Did he not cry out for having been abandoned? Where was all his awareness, his center, his divinity, his power, at that time? At his greatest trial?

It is part of the path.

You see, it is part of the path.

Awakenings. Blessings. Radiance.

Suffering.

And then a deluge of all the above, again, at a higher, more intense, level.

If you want to know more, if you want to feel more, if you want to experience more, then you will.

We are like fish schooling together to discuss the possibility of the existence of the ocean. (Thank you, Rumi.)

Welcome these gifts from the Beloved.