Thursday, July 13, 2017

CAR-T Cells

Last year we wrote an extensive analysis of CAR-T cells. In Nature this week there is a piece on the FDA progress on approving them. They note:

External advisers to the US Food and Drug Administration (FDA) have thrown their support behind a therapy that genetically engineers a patient’s own immune cells to target and destroy cancers. In a unanimous vote on 12 July, the panel determined that the benefits of CAR-T therapy outweigh its risks. The vote comes as the agency considers whether to issue its first approval of a CAR-T therapy, for a drug called tisagenlecleucel, manufactured by Novartis of Basel, Switzerland. The FDA is not obligated to follow the recommendations of its advisers, but it often does...Studies have shown that CAR-T therapies can produce lasting remissions in such cases. In one key trial of tisagenlecleucel, which started in 2015, 52 out of 63 participants — 82.5% — experienced overall remissions. The unpublished trial had no control group, so investigators cannot yet say with certainty how much effect the treatment had. But many participants of such trials have remained cancer-free for months or years.

These are most likely the first of many therapeutics that are customized to attack specific individual cancer cells of an individual patient. Simply stated:

1. Immune System is Used to Attack

2. Targets on the cancer cells are identified

3. Chimeric cells are created to use the patients immune system but modified to attack the specific cancer cell

4. The cells are individually customized and then reinserted into the patient.

5. This is a very powerful approach! It may actually be akin to carpet bombing, turning the immune system against itself. There seems to be no way to turn it off.

However, ALL was a deadly disease. One of the first cases I saw was in 1967 when a classmate was sent home from Marine Corps training with a high and uncontrolled fever. It was ALL and he was dead in a couple of weeks. If this approach works it may very well stop this disease and more.