Taking a step back, to move forward

Progress doesn’t always come in straight lines. Particularly when you are a pioneer in a whole new field of medicine like stem cells where virtually everything you do is being done for the first time, and the therapies you are developing are going to be tested in people for the first time. That’s why everything you do has to be done with extra caution to make sure the best interests of the patients come first. Sometimes that means not rushing ahead, but pausing, while you decide what is the best approach.

SangamoThat’s what Sangamo have done in announcing they are delaying the start of their clinical trial in beta thalassemia – a trial we are funding.

They are taking what amounts to a “time-out” so that they can make a small change in direction, one they – and we – hope will ultimately prove most effective for patients.

Βeta thalassemia is a genetic disease that results in patients producing red blood cells with poorly functioning hemoglobin, the protein that carries oxygen to all our tissues. If not properly managed the condition can be fatal.

The approach Sangamo are taking to cure the problem involves using zinc finger nuclease (ZFN), a kind of molecular scissors, to genetically edit the patient’s own stem cells, correcting the problem and enabling them to produce healthy hemoglobin and healthy red blood cells.

But as they geared up for the clinical trial, one that was approved by the Food and Drug Administration (FDA), they did some preclinical testing and saw that an approach they were using on a similar disease – sickle cell disease (SCD) – appeared to be more efficient and effective at correcting the underlying genetic problem. Both used ZFN to edit the defective gene, but they both had slightly different targets on those genes. The one targeting SCD seemed to have some key advantages, so they have decided to switch to this approach for both conditions.

In a news release Edward Lanphier, Sangamo’s President and CEO, says it wasn’t an easy decision to make, but it is the right decision:

“While our joint decision will result in a delay in the initiation of the beta-thalassemia Phase 1 clinical trial, we believe that the efficiency of the consolidated development path and potential benefit to patients clearly support this decision.”

The next step is for Sangamo to go back to the FDA and file a new Investigational New Drug or IND application for this new approach to beta-thalassemia. They’re hopeful they’ll be able to get that approval, and move ahead with their clinical trial next year.

The good news is that thanks to our new way of funding under CIRM 2.0, Sangamo will have the opportunity to seek CIRM’s support for its work through both our preclinical program, as they make their changes, and then our clinical program if and when they get FDA approval to move ahead. This uninterrupted support is what CIRM 2.0 was set up to achieve, to move promising projects like this to patients as quickly as possible.

For the team at Sangamo it’s obviously disappointing to have to stop and change direction. It’s also disappointing for the patients hoping this would lead to a more effective therapy, even a cure.

But this is not a set back. Rather, it’s a step back. One that allows Sangamo to choose what they believe is a better option, one that will ultimately be much better for patients.

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