Technology Gives Young Hemophiliac His Life Back

For two years now, Brock Spacek has been reconstituting his own freeze-dried vials of Helixate® FS, a recombinant factor VIII product manufactured by CSL Behring and used to treat hemophilia, every other day. He is a little too squeamish yet to insert the needle into the surgically implanted port in his chest so he has his mother do it for him. You can’t blame him, though, if he is not ready to take on that scary step. Brock Spacek is seven years old.

When not mixing drugs, Brock plays soccer and baseball and recites multiplication tables into the twelves for his beaming mother. “He pretty much does everything other kids his age do,” Katherine Spacek told HCAR this week, “except football. I don’t think we’ll ever be quite confident enough to let him play a sport where bleeding is an expected part of every game.”

Like many volatile medications, Helixate® cannot be shipped in its liquid state. It would lose potency within hours. Brock’s parents receive their monthly supply in a freeze-dried powder. Until recently, the process of reconstituting it immediately before injection used to involve a dozen steps with glass vials, filters and a series of needles. At any step along the way it was possible to make a mistake and spoil a batch. Plus, there was always the danger of accidental needle sticks during one step or another, not a welcome event for a hemophiliac.

In early 2006, however, the Spacek family discovered a new technology, Mix2Vial™, which is manufactured by Medimop Medical Projects Ltd., a subsidiary of West Pharmaceutical Services. The plastic transfer system reduces the Helixate® reconstitution process to four steps, which Brock not only mastered in one lesson but also recently taught to his three year old sister.

Instead of needles, the Mix2Vial system employs a built-in filter and plastic spikes to puncture plastic vials and measure an exact amount of water or saline solution into a pre-measured amount of powder. Switching to it, Mrs. Spacek said, has transformed Brock’s life. Larger doses are possible with the Mix2Vial system and only need to be administered every other day.

According to Graham Reynolds, Vice President of Reconstitution and Transfer Systems for West Pharmaceutical Services, Mix2Vial is one of several delivery systems his company has developed and provides to pharmaceutical manufacturers and distributors. Selling directly to home care providers is a market channel he is interested in exploring.

At the beginning, Katherine Spacek and her husband were told how easy the new procedure would be, and they knew Brock was a bright kid, but they were surprised when he learned to do it by himself at age 5. Katherine said Brock’s math wizardry surprised her too. Neither it nor hemophilia run in either her or her husband’s families.

“There were two times we nearly lost him in the first year,” she related, the emotion backing up in her voice as she revived memories of two near-fatal episodes from 2000. “Fear,” she finally managed to whisper without succumbing to another sob. “Fear and helplessness are what you feel when you watch your infant son lying in a hospital bed, in a pool of his own blood that the doctors and nurses cannot control. The beeping of the blood pressure monitor forces you to keep one eye on its readings as they steadily shrink to numbers you thought were too small to sustain life.”

The unexpected diagnosis arose when Brock’s blood did not clot after his circumcision at age two days. For months afterward, the Spaceks and Brock’s doctors tried to find the right medication at the right dosage. Drugs available even as recently as six years ago were less reliable than the one Brock uses today. At seven months, internal bleeding nearly took him a second time, Katherine reluctantly related.

Today, Brock is a typical second-grade boy, exhibiting a normal makeup of snakes and snails and puppy dog tails, plus a little Helixate® FS. The only differences between him and his classmates are both apparent only at home. “He looks at me kind of wistfully,” Katherine smiles with a mother’s compassion, “and says, ‘Mommy, I wish my blood would just start to clot.’ And then he goes into the kitchen and mixes vials of potent drugs.”