Something seems to be happening with GDNF(and its not about the long delayed NIH trial). Received a Google Alert , stating that MedGenesis, a small biotech in BC, Canada raised 5 million for phase 2 GDNF Trials.The company obtained the rights to Amgen's gdnf protein in 2010, and they are working on delivering this protein to the brain by Convection Enhanced Delivery. This is not gene therapy.
see:

http://news.techfinance.ca/medgenesi...-of-financing/

They are planning to start a phase II trial.
Excerpt:
"“This financing will assist us in the completion of the phase II clinical program for GDNF and allows us to now shift our focus to financing our Phase III program, including the development of pharmaceutical company partnerships,” said Dr. Erich Mohr, Chairman & CEO of MedGenesis Therapeutix. “We are hopeful that this potentially disease-modifying treatment will significantly change the quality of life of patients with Parkinson’s Disease as well as have applications in other neurological indications.”"

See also:
http://www.terrapinn.com/exhibition/...peutix-inc.stm

And an interview with cEO Eric Mohr from 2010
http://biotuesdays.com/2010/04/13/in...th-erich-mohr/

In 2010 the sponsor received a MJFF grant
http://www.michaeljfox.org/research_...s_3.cfm?ID=664

Also see Medgenesis web site
http://www.medgenesis.com/index.htm

" MedGenesis has advanced CED to a potentially disruptive platform technology with a broad range of applications. CED employs a local pressure gradient to infuse fluid (in this case containing the therapeutic GDNF) into the brain, allowing for up to a 1,000-fold increase in the distribution volume relative to conventional diffusion. The MedGenesis proprietary CED system allows for targeting of specific areas in the brain resulting in the accurate distribution of therapeutics.

This integrated approach provides for the potential of a safe, simple, and effective long-term treatment of patients with Parkinson’s disease on an out-patient basis. Patients will continue to see their neurologists monthly, where no more than 1-2 hours will be required to receive the non-invasive infusion that does not even require puncture with a needle."

I emailed the company at the email address listed on their website. I asked for details of the upcoming Phase II gdnf trials. I'll let you know if/when I hear back from them.

Jean

MedGenesis email reply:

At this point, we don’t have a specific date for when our clinical trials will begin in North America. The next clinical study of GDNF is planned to be conducted in the United Kingdom. Once the responsible regulatory and ethical bodies have given approval to move forward with the study, information regarding the study and patient recruitment will be made available online. Importantly, the decision about the inclusion of patients is based on predefined criteria and completely in the hands of the responsible investigators; MedGenesis is not involved in the selection process.

I wonder why they can't make a "nasal-spray" version?

Is this the trial to be run by Stephen Gill & others? Can anyone clarify on this?

It's my understanding that MedGenesis is part of the new Gill trial which I think they are calling Phase II in the UK (when will they begin?) and subsequent phases will be in the US. Now that MedGenesis has secured $5M in funding for the Phase II trial, hopefully the Gill GDNF trial will proceed!

Jean

On Friday the 13th, if you are superstitious, I spoke with Howard Federoff, a leading Neurologist and clinical scientist. He is also Dean of Georgetown Medical School, and as Chair of the NIH recombinant DNA advisory committee, he was instrumental in setting up the 2010 NIH workshop on the use of sham control groups in double blind research designs. (The report from that conference should be published shortly.) It has been 3 years since Dr Federoff first told me about the interests of the Gene Therapy Study Group consortium, an academic group of PD researchers that he leads, in developing GDNF gene therapy. I have been checking in with him on a regular basis to get updates from the top and to offer patient advocate assistance wherever possible (e.g. to help communicate with PWP or FDA), as well as to be a squeaky wheel.

Now after 7 years since Amgen declared GDNF unsafe and ineffective, we may be starting to see real movement. After all the work that many of us did to try to coax Amgen to continue development of GDNF therapy, this is the prize. Although it has been slow, we are fortunate that there are people with the stature and ability of Dr. Federoff and his colleagues to pursue this development, given the limited IP protection (Amgen's original patent runs out this year). Funding the later phases will continue to be a challenge as will the methodology for well controlled trials required by regulation and the "sham wall" (see Katsnelson "Why Fake It" Nature , Aug 10, 2011. http://www.nature.com/news/2011/1108...l/476142a.html and Benderly "Head Games" Miller-McCune, Jan 2012 http://www.miller-mccune.com/health/...thought-38717/ ) that we have observed in the evaluation of past surgically delivered therapies.

Dr Federoff said that the IND (Investigational New Drug) application for the GDNF-gene therapy was complete and would soon be submitted. During the 3 years since this planning began, the researchers have conducted long term toxicology studies on GDNF in primates, and have exhaustively documented all aspectes of the science and research with evidence from preclinical research. Then over the past 6 months they have prepared the IND package. After it is submitted, the FDA has 30 days to put a hold on starting the trial or the trial can proceed without further approval. Given the track record for successful clinical development of new PD therapies, maybe such meticulous effort is necessary to achieve the sucessful outcome we all desire, but as I told him, being a patient with a servious illness takes a lot of patience.

More than a year ago,I had spoken to the NIH neuro-surgeon, Dr Russell Lonser, who will implant the GDNF-AAV virus, who said they already had IRB approval. I dont know what are the other procedures that need to be scheduled other than the informed consent so they could start operating by March if everything goes smoothly, but it seems like it never does. I do not know what the schedule is for subsequent surgeries.

Dr. Federoff also said they already had several people lined up to participate. A number of PWP have been doing a 6 month test of the At-home measurement device developed by the Kinetics Foundation to be used along with clinical neurological examinations and neuro-psychology tests to provide quantitative measures in the evaluation of the outcomes. The 6 months of data will provide a stable baseline assessment of the starting point for patients and will lead into participation in the clinical intervention, inserting the virus in the putamin, for this phase I/II study. Patients will be randomly assigned to one of four groups of 6 patients each to test the safety of each dose level and provide a first look at improvements from the baseline.

Some of us were worried that the announcement on the phase II study of direct infusion of the GDNF molecule might be a signal of a change in priority of the study team, but this appears to not be the case here, if in fact there are no further delays. This other phase II study will be done in Bristol UK, with Dr Steven Gill.

Perry

Perry, thanks for this information. I am one of the PWP testing the Kinetics at home measurement device. I'm 2 months into that trial, and will finish my 6 months test this May.

Jean

The Cure Parkinson's Trust estimates the Gill GDNF trial will start April or May 2012

Woo hoo!

Jean