Earlier this month, I discussed how Sangamo Biosciences (Nasdaq: SGMO) created a groundbreaking new HIV treatment – a therapy known as Zinc Finger Nuclease (ZFN).
The therapy acts as molecular “scissors” that cut harmful genes, called CCR5, from a patient’s white blood cells and replace them with modified cells that protect the immune system against infection.
Well, Sangamo just finished phase I of its clinical trial testing a ZFN treatment called SB-728-T. And as I expected, the results are astonishing…
An Unprecedented Leap Over Traditional Treatments
Sangamo revealed three key findings from the trial:
- The study showed a significant suppression of the viral load in patients. Or as Sangamo CEO, Edward Lanphier, told BusinessWeek, “We see a significant anti-viral effect. That’s the big punch line here.”
- Scientists noted “unprecedented improvements” in white blood cell counts. That’s huge, considering existing antiretroviral drugs haven’t been able to restore the immune system. Dr. Ronald Mitsuyasu says, “Improvement and preservation of the immune system is of paramount importance in HIV. And those seen in this study show an improvement over that seen after several years of [taking antiretroviral drugs].”
- One patient who received SB-728-T even had a viral load that decreased to undetectable levels. Meaning that the patient demonstrated complete suppression of the virus.
Each of these results suggests that Sangamo has a promising new treatment option on its hands. But it’s the third result that has scientists really excited.
Because they believe it can be replicated in all patients…
A “Functional Cure” on the Way
According to Sangamo, about 10% of the population – including the patient mentioned above – is born with a CCR5 mutation.
Essentially, this mutation suppresses CCR5 naturally, giving people an innate resistance to most strains of HIV.
As a result, when the trial patient with the mutation received the treatment, twice as many of his cells were modified. And that’s why the virus was no longer detected.
You see, when scientists remove CCR5 using molecular “scissors,” they usually don’t cut it from all of the cells. They remove just enough to help the body build immunity.
But now researchers think that cutting more CCR5 genes is the solution to seeing HIV suppression across the board.
According to one researcher, Dr. Carl June, this “suggests that the next step is to increase the frequency of the modified cells in HIV-infected patients… With the ultimate hope that if we do, we will achieve a ‘functional cure’ and eliminate the need for continued [antiretroviral therapy].”
Bottom line: Sangamo’s treatment threatens to disrupt a drug industry that hit $15.1 billion in sales last year alone. And you can bet Big Pharma giants – especially HIV drug leaders like Gilead (Nasdaq: GILD) and Bristol Myers Squibb (NYSE: BMY) – are already taking notice… so should you.