SRPT won't call them out because they might sabotage their chances for a "quick" favorable review. When Geron dropped their stem cell program, there was not one word of outrage from them because they need to get their telomerase drug through the regulatory process.
Their T-cell gene therapy attacks all B lymphocytes, not just the cancerous ones. This would further suppress immunity in a mostly elderly CLL population, whose immune status is already declining.
It was predictable. HIV is known to lurk in nodes in the abdomen. I wonder how hard they searched for suitable donors with natural immunity to HIV, as they did for the German patient, who seems to be cured..
The gutless FDA was so afraid of stem cell treatments it wouldn't allow Geron's phase 1 study to include partial spinal cord injuries, which the Rx was developed for. It insisted on complete spinal cord injuries, because those patients had less to lose if there were safety issues..( the Rx had never worked in animal models of complete spinal cord injuries. Geron showcased movies of its excellent results in rats with partial spinal cord injuries). For the same reason it limited the study to thoracic spine injuries. It refused to allow cervical spine injuries. Geron couldn't adequately enroll the trial because the use of airbags has virtually eliminated thoracic spine injuries, which were mainly caused by steering wheels in crashes. This despite the patients and families begging to be tested, willing to take any risk. After spending an enormous amount of time and money, Geron gave up and dropped its stem cell program. Now there is no treatment for these patients.
Does anyone know whether the RSV vaccine will be given yearly, or if one shot is all that is needed?
The control group gets the standard of care. If the study is properly double blinded, no one knows who gets which treatment. If my mother or father had GBM, I would want them to enter such a study.
Always defrost in the refrigerator. Because it takes so long, even at room temperature, by the time the core is defrosted, the defrosted meat near the periphery can allow salmonella and other bacteria to multiply to the degree where eating it can cause serious illness. The surface meat must be kept at refrigerator temperature while the core defrosts. It will take 24 hours for the turkey to defrost in the refrigerator.
When you do a phase 1 safety study, there is no need to randomize the patients. But you should not release the results as though it was an outcomes study, unless it was a randomize trial. Single center, non-randomized studies are given little respect in medical circles because they are so easily corrupted and misused. Do you understand that?
GBM is occasionally diagnosed "by accident". For example, a very small asymptomatic tumor is picked up when an MRI is done because of a head injury. These patients have their tumors resected, and they are given radiation and/or chemotherapy. They have excellent prognoses. A big referral center like Cedars Sinai would have a number of that type of patient. The obvious question is, with so many GBM patients available there, why didn't they randomize the study?
I had held back because it was a non-randomized single arm study, run at Cedars Sinai by a man who helped develop the drug, and has a financial interest in the drug and in IMUC. Did they cherry pick patients with the best prognoses? Roth said they visited Cedars Sinai, and came away believing that did not happen. Can Roth be trusted? Do they have financial ties to IMUC?
The Yahoo historical prices section will take you back to 1993. Du Pont Pharmaceutical's top scientific guys refused to go along when Bristol Myers bought their company, and formed Incyte. I believe that was in 1991. That's why they are headquartered in Wilmington Delaware, not exactly a Mecca for biotech. . The price dropped when their first drug, a potential best in class HIV drug, had to be canned because of liver toxicity, but they kept going and have produced a potential powerhouse.
I don't think it means much. Vaccines have a limited shelf life, and have to be replaced regularly. This leaves an opening to replace with a better vaccine when one is available.
I've followed since Bristol Myers bought Du Pont Pharmaceuticals, and Du Pont's best scientific guys refused to go along. They had the guts to break off and form Incyte. That's why it's headquartered in Wilmington, Delaware. They took a blow when their first drug, a potential best in class HIV drug, had to jettisoned because of liver toxicity, but persevered, and have produced a future powerhouse. I take my hat off to them.
How do you put someone on ignore? I can't find anything to click on for that. TIA.
I don't understand the disappointment. Four of eight patients with the mutation had responses at 54mg/kg. Now they are going up to 80 mg/kg in the next cohort. I think is a good result. Any opinions?
Thank you for posting this info. It looks better than GLPG's Jak I inhibitor, although the GLPG drug is already in phase 2. The icing on the cake is that INCY owns all rights to 039110, while GLPG has partnered its drug.