|Dr. Mike Lawlor|
Tuesday, January 16, 2018
Guest Post from Dr. Mike Lawlor: Audentes Announces Positive Interim Data from First Dose Cohort of ASPIRO, a Phase 1/2 Clinical Trial of AT132 in Patients With X-Linked Myotubular Myopathy
Audentes Therapeutics has released an interim data update on the ASPIRO gene therapy clinical trial for X-linked myotubular myopathy. In the 4th quarter of 2017, three patients were given a single dose of an adeno-associated virus containing the human myotubularin gene. To quote the press release:
"The early AT132 efficacy data observed in our first dose cohort of patients have exceeded our expectations," stated Dr. Suyash Prasad, Senior Vice President and Chief Medical Officer of Audentes. "At the 12-week timepoint, Patient 1 has improved from a severely compromised baseline to achieve a CHOP-INTEND score and maximal inspiratory pressure that are approaching the ranges normally seen in healthy children. Importantly, Patient 1 has also attained several age-appropriate developmental milestones within this time period, including head-control, rolling over and sitting unassisted. While still early in the trial, we view these initial efficacy data as a promising indicator of the potential for AT132 to bring meaningful benefit to patients and families living with this devastating disease."
On the safety side of things, Audentes reported a total of six adverse events (AEs) reported in ASPIRO, two of which were determined to be serious adverse events (SAEs). Per the press release, “Both SAEs occurred in Patient 3, the first of which was a hospitalization one week post-administration due to pneumonia and was deemed not treatment-related. Patient 3 was also hospitalized at week 7 post-administration due to a gastrointestinal infection and elevated troponin levels, the latter of which was deemed probably treatment-related and is responding to treatment with intravenous steroid administration and supportive care.”
On the pathology side of things, it will be a while before biopsies are evaluated or analyzed, as the trial design incorporated extensive strategies to keep the study pathologists blinded to timepoint, treatment group, and even whether a given sample belongs to a study patient. Hopefully I’ll have the opportunity to share some of the pathology data at the 2019 AANP meeting.
The complete press release can be found at: http://investors.audentestx.com/phoenix.zhtml?c=254280&p=irol-newsArticle&ID=2324833
Friday, December 22, 2017
Best Post of October 2017: Neuropathologist Hannes Vogel featured in New York Times for examining brain of Las Vegas mass murderer
The next in our "Best of the Month" series is from Friday, October 27, 2017:
Prominent neuropathologist Hannes Vogel of Stanford University was featured in the New York Times yesterday as he is examining the brain of Stephen Paddock, who killed 58 concertgoers in Las Vegas this month in a rampage without any clear motive. Here's the link to the NYT article.
|Hannes Vogel, MD|
Monday, December 18, 2017
Thursday, December 14, 2017
Best Post of September 2017 -- Guest Post from Dr. PJ Cimino: Blue discoloration of the gray matter in a patient who received methylene blue for respiratory distress prior to death
The next in our "Best Post of the Month" series is from Monday, September 11, 2017:
Dr. PJ Cimino, whom we profiled when he was a fellow back in November of 2013, is a now faculty member at the University of Washington. I was delighted to receive this email from him today:
"I had an autopsy case with interesting gross pathology findings, which made for some nice clinical images (below). The patient received therapeutic methylene blue in the setting of respiratory distress prior to death. The gross pathology showed striking widespread green-blue gray matter discoloration. I thought these images might be of interest to share with the general neuropatholgy community, and thought your blog might be a good platform to do so, especially since you have posted many good clinical images."
Friday, December 1, 2017
The next in our "Best of the Month" Series is from August 1, 2017:
Monday, November 27, 2017
Best Post of June 2017: Remarkable en bloc dissection of human central and peripheral nervous system accomplished at University of Colorado
The next in our "Best of the Month" Series is from June 7, 2017:
|Shannon Curran, MS with her dissection|
Curran, who is known among students and faculty as a preternaturally efficient prosector, completed the dissection in under 100 hours. Further detailed work is planned on the specimen, including dissection of the extraocular muscles away from the eyeballs while maintaining their connection to the brain. Discussion is underway about loaning the specimen to the Denver Museum of Nature and Science for community health education.
|CNS en bloc dissection with extensive portion of PNS|
|Connection to the eyeballs is maintained, with plans to dissect away extraocular muscles|
|Detail showing maintained connection with digital nerves of the left hand|
Monday, November 6, 2017
|Sclera is at bottom of picture; retinal pigment epithelium is at top right. Between them is choroid with cavernous hemangioma|
Wednesday, November 1, 2017
I am pleased to present a guest post from Dr. Sandro Santagata of Brigham and Women’s Hospital/Dana Farber Cancer Center, who writes:
Our group of collaborators has recently opened a Phase II Trial of BRAF/MEK Inhibitors in Papillary Craniopharyngiomas that is sponsored by the Alliance for Clinical Trials in Oncology.
The eligibility criteria are listed here: https://clinicaltrials.gov/ct2/show/NCT03224767
The trial is based on work published in these two papers:
I am happy to answer any questions that our colleagues may have about this trial. email@example.com