Wednesday, September 11, 2013

Patience is the key to reaching milestones: progress on the road to treating Huntington's disease

Mesenchymal stem cells | Image by Libertas Academica
Getting a potential therapy into a clinical trial is a tremendously complex process involving years, sometimes decades, of research, a lot of money and a huge amount of organization. Just how much work is involved is really not appreciated by most people.
Dr. Vicki Wheelock, Terry Tempkin and their team at UC Davis know very well how much time, effort and energy is needed to move a promising therapy into a clinical trial. They are working on a potential treatment for Huntington’s disease. (We gave them a Disease Team grant last year to support this work.) They are working with Dr. Jan Nolta to use mesenchymal stem cells (adult stem cells usually found in bone marrow or fat) to deliver something called Brain-Derived Neurotrophic Factor (BDNF) to the brains of patients with Huntington’s and hopefully protect the still-healthy brain cells and perhaps restore the health of the brain cells damaged by the disease.

For the past few years they have been doing a series of detailed studies in preparation for a future clinical trial and they just passed a huge milestone; they have been given permission to begin recruiting 40 patients with early stage Huntington’s, patients who may one day be part of that clinical trial.

Terry Tempkin, the Clinical Project Manager, calls this stage a “building block.”
“We have specific aims for the study and for any of these patients to be in the future planned stem cell trial, they will be invited to first enroll in a preliminary study called PRE-CELL Over the next year and a half we’ll be monitoring them carefully to see how their individual disease progresses. By measuring all the changes that take place in their bodies, and their psychological state, we will have a better idea if they are experiencing any unexpected side effects when they undergo the actual therapy. And of course by knowing how their individual disease is progressing we can get a better idea of whether the therapy is slowing down that progression.”
They’ll be using MRI scans and a battery of other tests to study all sorts of things from how patients move and how good their balance is to their thinking skills and their mood and behavior. All the things they measure in this pre-trial will also be measured in the actual trial, so this pre-trial study provides them with important baseline information about each individual patient that will ultimately help them understand if the treatment is safe, and to allow some exploratory measures to determine if the treatment is working.

There’s a good reason for this level of caution. This is the first time this kind of approach has ever been tested in people, and while the team has done a lot of pre-clinical work in animals and in the lab, none of those are the same as testing it in real patients.

Every patient who is interested in being part of PRE-CELL has to first complete the comprehensive informed consent process. This process, which can take an hour or longer, insures that study participants fully understand the goals of the study, as well as the risks and benefits of study participation. Patients who agree to participate will then undergo rigorous screening to ensure they meet the scientific criteria for the study. Those who meet the study criteria will then go through a series of detailed visits every 6 months with the research team. Even when they have agreed to be part of the study, participants are told they can pull out at any time, for any reason.

If all this works, if they are able to recruit enough patients, if they are able to get all the information they need then, and only then, will they be prepared to go forward with the planned stem cell trial. That trial depends on first getting approval from the Food and Drug Administration (FDA) and that approval requires the successful completion of work already underway in the Nolta lab, which will continue during the course of the PRE-CELL study. The team hopes to obtain FDA approval to start the stem cell trial in early 2015.

While that might seem like a long way away Tempkin says they are “thrilled” to be at this stage already, that it’s a sign they are making steady progress.

Science doesn’t always move as quickly as we would like, particularly for people with a disease like Huntington’s where the average patient’s life expectancy after diagnosis is less than 20 years. But the understandable desire for quick results cannot overcome the need for safety. The imperative must always be to first protect the patients. That means moving forward in steady, measured steps. But researchers like Dr. Wheelock and Terry Tempkin know that if the treatment they are pioneering is ultimately effective, it will have been well worth the wait.

Besides this research we are funding a number of other studies focusing on Huntington's. You can read about that work on our Huntington's disease fact sheet.

Kevin McCormack

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