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What is the significance of “breakthrough drugs”?

One might think this is just “marketing. But that is not necessarily the case. According to Stephen j. Williams,  “breakthrough” status for a drug is helping to push the review process here with the FDA.  In Europe “breakthrough” status seems to facilitate expediting a new drug faster than US.

In a sense, Penicillin and Insulin were in their time breakthrough drugs. Then came oral antibiotics.   Ampicillin was great for ambulatory.With the development of resistance, methicillin was an early “breakthrough”. But it ran in to resistance, and we got to the next round.  We went through many years after WWII with the development of superb synthetic organic chemistry that led the way.

2. Which other Oncology drugs are fulfilling this function?

Kite Pharma just had their CAR-T therapy put on breakthrough status and this therapy will probably be an adjuvant therapy. Some clinicians are starting to put CAR-T and PD1 inhibitor trials together.

3. Why in the context of leukemia, is a breakthrough drug so important?

Amgen’s strategy seems to replace an autologous T-cell strategy, so no purification and re-engineering of a patient’s T-cells would be needed.  Acute leukemias are either acute myelogenous, occurring in adults, and acute lymphocytic, occurring in young adults or in children.  The classification has become more complicated in the last decade, with a relationship between CLL and Hodgkin and non-Hodgkin lymphoma.  The disease CLL can be quite innocuous, and it occurs over age 70 years.  It is usually B-cell type.  There is now breakthrough research occurring with respect to T-cells (thymic derived as differentiated from Bursa of Fabricious, lining the gut mucosa).

All occurs in young adults or in children.  AML is more likely moving up into adulthood.  But ALL – characterized by immature lymphocytes termed lymphoblasts, is very explosisive.  The child normally has a high lymphocyte count compared to an adult in the Ly/Myelo ratio.  Unlike CLL, the count does not reach high levels, but the lymphocytes presenting are immature.  The treatment approved is not only effective in clinical trials, but is also not burdensome in treatment for a very aggressive disease.

What is autologous?

The patient’s blood is removed for treatment, and then retransfused back to the patient.  Actually, only the WBC portion is needed.  What is especially important is that there is no possibility of graft versus host disease, which 100% fatality.