Glial cell-line derived neurotrophic factor (or GDNF) has been a topic of heated discussion in the Parkinson’s community for a long time. Most recently due to the announcement of the results of the Phase II Bristol GDNF clinical trial results, which did not meet the primary end points of the study (Click here to read more about that).
This week at the annual American Association of Neurological Surgeons conference in San Diego, the results of another GDNF clinical trial were presented.
This new study was a Phase I study assessing the safety and tolerability of a gene therapy approach for GDNF in people with Parkinson’s.
In today’s post, we will discuss what gene therapy is, what the new trial results indicate, and what the researchers may be planning to do next for this new clinical trial programme.
Every year members of the American Association of Neurological Surgeons gather together in one spot and compare data/research/clinical notes.
This year the 87th AANS Annual Scientific Meeting was held in spectacular San Diego.
San Diego. Source: AFP
From Saturday 13th April through till Wednesday 17th, clinicians and researchers attended lectures and discussed new data on every aspect of neurological surgery. While I did not (nor planned to) attend the meeting, I was very interested to learn more about one particular presentation.
It involved the announcement of the results of a clinical trial which was focused on a gene therapy approach for Parkinson’s.
The treatment involved GDNF (Click here to read the abstract).
What is GDNF?
In March of 2017, activists all over the world celebrated the 30th anniversary of ACT UP.
ACT UP was an international direct action advocacy group that was set up to help people with Human immunodeficiency virus (HIV) infections and acquired immune deficiency syndrome (AIDS), and more importantly to encourage legislation/policy, medical research and treatment for this devastating.
Some people might say that there have been few advocacy efforts for a medical condition that have had as much impact, or had such a lasting legacy. Others, on the other hand, would suggest that a collective AIDS advocacy effort and a paralysing fear of the deadly condition at the time stimulated most of the action action that followed. Regardless of which opinion is correct, an analysis of the AIDS advocacy makes interesting food for thought.
In today’s post, we will have look at what ACT UP did, what was the result of the overall AIDS advocacy movement, and we will discuss what the Parkinson’s (and neurodegenerative) community could learn from it.
It provides public health information and recommendations based on information collected by the CDC from all of the various state health departments. The material published in each report is provided as a public service and may be reprinted and used without permission.
On the 5th June 1981, the CDC published a MMWR, describing five cases of a rare lung infection (called Pneumocystis carinii pneumonia). This was an extremely rare, but opportunistic infection – it generally occurred in people with very compromised immune systems.
All of the five cases were young (29-36 years of age), and had previously been healthy, homosexual men living in the Los Angeles area. All of the men had additional unusual infections, which suggested to doctors that their immune systems were not working properly.
Two of the men had already died by the time the report is published.
One month later, on the 3rd July (1981), a second MMWR was published. This one mentioned further cases of Pneumocystis carinii pneumonia and cited 26 cases of a rare form of cancer known as Kaposi’s sarcoma, which is caused by a viral infection:
These two MMWRs were the first official reports mentioning what would go on to become known as the AIDS epidemic – a devastating, ongoing global health issue that has thus far claimed 35+ million lives.
It was also the very beginning of what would be an amazing story of patient advocacy. The one that would become the template for many future efforts. Part of that advocacy effort was called “ACT UP”, and in today’s post we will discuss what the movement was and how they achieved some amazing accomplishments.
But before we get to that, let’s start at the beginning:
What exactly is AIDS?