Rare Disease Report


Investigators working on Glioblastoma Moon Shot program have made a key discovery on glioblastoma progression, identifying a key target to help stop it.
The oncolytic adenovirus was safe in terms of toxicity in mice models.
The medulloblastoma subset has a poor prognosis, so investigators have been looking for new treatment options.
The FDA has approved Janssen’s ibrutinib plus obinutuzumab for treatment-naïve patients with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL).
Some of the new subtypes are similar to previously identified ones but show similar prognoses to established subtypes.
The FDA has approved trastuzumab-dttb (Ontruzant) for the treatment of patients with HER2-overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma in combination with cisplatin and capecitabine or 5-fluorouracil.
The FDA has approved cabozantinib (Cabometyx) for the treatment of patients with hepatocellular carcinoma who previously received sorafenib (Nexavar).
Only patients with very serious, life-threatening conditions with a risk/benefit ratio is acceptable for use of the therapy at this time.  
Dipanjan Basu, PhD, is testing non-melanoma drugs, like the investigational therapy omipalisib (GSK2126458) in cultured patient cells.
While the absolute risk of therapy-related blood cancers is low, the treatment is often intensive and linked to substantial morbidity.  
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