HIV/AIDS vs. THE CALIFORNIA STEM CELL AGENCY: Disease-a-week Challenge # 13

So little attention is paid to HIV/AIDS nowadays, one might be forgiven for thinking it cured. But is it?

To date, only one (1) person has been cured of HIV-AIDS.

One million Americans have HIV/AIDS today—as do more than thirty million world citizens—many of them children, who caught the disease from their mothers’ breast milk.

They are not cured.

It might be said that HIV/AIDS is controllable now, for those with the resources to pay for it.

A therapy called ART (Anti-Retroviral Therapy) can  keep HIV patients alive. As long as the 30-40 pills are taken every day, in the right order and at the right time, HIV (Human Immunodeficiency Virus) is survivable.

But without the pills, (and they are not cheap) HIV advances to the death sentence of AIDS (Acquired Immunodeficiency Syndrome).   HIV can be lived with; AIDS cannot.

The disease is only managed, not cured. Even the remedy itself carries major risks. People on long-term ART may face cancer, heart and lung disease, osteoporosis, and other life-threatening maladies.

And the one cured person, Timothy Ray Brown, better known as the “Berlin patient”?

Residing in Berlin, Germany, Tim Brown had both leukemia and HIV, which latter he was managing by the ART pills. But to save him from dying of leukemia, Brown was given a bone marrow transplant—from a person immune to HIV.

Apparently, as many as one percent of  Northern Europeans are blessed with a mutation that prevents HIV from entering their cells and infecting them. (This might be an immunity passed down from survivors of Europe’s Black Plague.)

Tim Brown overcame both leukemia and HIV-AIDS.

But his experience will not work for everyone. A bone marrow transplant operation is painful, dangerous, and depends on finding a suitable “match” for the donation.

But might there be a way to make a person immune to HIV?

Enter the California stem cell agency. Begun by the citizen’s initiative, Proposition 71, the California Institute for Regenerative Medicine (CIRM) gives grants to fight  all forms of “incurable” disease.


Two teams of California stem cell researchers were selected to take on the challenge of HIV-AIDS: they would try to make changes in the stem cells of the blood, so what cured the Berlin patient might be done again across the world.

Team One is Calimmune, Inc., a company begun by David Baltimore, Nobel Prize winner and former board member of the California stem cell board of directors. That team is led by Ronald Mitsuyasu, MD, of the University of California at  Los Angeles, and Jacob P. Lalezari, MD, of Quest Clinical Research in San Francisco.

Team Two is comprised of John Zaia, David DiGiusto, Amrita Krishnan,  and Paula Cannon from the Beckman Research Institute of City of Hope, and the University of Southern California in partnership with Sangamo Biosciences, Inc..

These teams face a battlefield of microscopic complexity: the gene chain of our lives.

Complicated? Wow! Hearing them talk is like reading a bowl of alphabet soup.

Look at the following “word”: CCR5.

CCR5 is a gene, part of our DNA structure, the blueprint of our lives. But CCR5 is not always our friend. It  sometimes lets HIV into our bodies, like a door for assassins, who may enter and kill.

But what if the gene had a change, or a mutation—delta 32 (^32)—added to it?  The door now locks.  The HIV virus has no way to enter.

CCR5—bad, CCR5^32—good.

Tim Brown was given a bone marrow transplant from a person who had the CCR5^32 injected into his body; Tim Brown was cured.

Would it be possible to put delta 32 into the gene structure and permanently lock the CCR5 “door”?  Or could that door be removed altogether, by snipping out CCR5 from the DNA chain?

That is the hope of scientists funded by the California stem cell agency.

Let’s meet someone from that battlefield:  Dr. Paula Cannon from the University of Southern California (USC). Dr. Cannon is the president of the Keck School of Medicine Faculty Council.

When I asked Dr. Cannon why she had chosen her career, she seemed a little  embarrassed, saying she had chosen biology “almost at random” in college. She had done well, earning a degree in microbiology, but after graduation she “did a 180-degree career spin” and went into the music business in Liverpool, home of the Beatles. She did not like it, she said, but it shaped her future.

HIV-AIDS was ravaging the entertainment industry; people were dying with no hope from this modern day plague.

Time passed;  another 180-degree career spin, and Paula Cannon was back in the science business again, but this time with a goal—to fight the “thief of lives” as a medical researcher.

As Dr. Cannon can talk science in “people-talk”, I asked for answers to three key questions:

  1. The CIRM grant involves something called a zinc finger nuclease (ZFN); what is that, and why is it important?

“A zinc finger nuclease is a sort of genetic scissors. It can snip out bad stuff in the body’s DNA chain. We hope to program ZFNs to snip out CCR5, so it won’t let the AIDS virus in again.”

  1. Same question for ribonucleic acid (RNA)—what is it, why does it matter?

“If DNA is like a thousand-page blueprint for a house, RNA might be considered one page—maybe for the upstairs bathroom. RNA may bring very specific and permanent changes in the body’s structure, by transporting the ZFN where you want it to go.”

  1. How important was the California stem cell program to you as a scientist?

“The CIRM grant made it possible for me to fight against AIDS within a stem cell team structure; without CIRM, this would not have happened.”—Paula Cannon, personal communication.

California is challenging the thief of lives: this virus called AIDS.

The battle is not won.

But it is surely joined.

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