Friday, October 22, 2010

GMP Stem cells

A recent press release by the California Institute of Regenerative Medicine announced the award of $6 million to the lab of Xianmin Zeng, Ph.D., of the Buck Institute for Age Research and The City of Hope, a small biotech research/treatment center in Californina, to generate human stem cells that may one day be suitable for clinical treatment in Parkinson's. What Dr. Zeng's lab has accomplished - and now has funding to try on a large scale - is the ability to grow and differentiate stems cells using defined culture conditions - an important aspect of Good Manufacturing Practice (GMP) that the US Food and Drug Administration (FDA) requires for any compound that will be used for human therapeutics.

What does "defined" mean? It simply means that one knows exactly what compounds are added to the culture media -- the fluid used to grow and nuture cells. Typically, when scientists grow cells for research use, they use undefined conditions where they don't know exactly what the fluid contains. For example, if you are cooking something that requires a banana flavor, you could chose the defined route of just using banana flavoring in a bottle, which is a single chemical called isoamyl acetate. Alternatively, you could choose the undefined route and use a whole banana, which not only contains isoamyl acetate but a whole bunch of unknown chemicals and proteins.

Why does this matter? Replication and Safety.

  • Replication: Because you don't know exactly what is contained in undefined media, it can be difficult to replicate. A classic example is from the early days of heart physiology. Many early advances in the field were made by Sydney Ringer. He created a defined media but used London tapwater as the source. He ran into difficulty trying to replicate his own experiments using distalled water. Turns out London tapwater has trace amounts of calcium which is essential for normal heart function...and he revised the formula for his now famous Ringer's saline solution.

  • Safety: The safety issue centers around the fact that cells in culture often need various proteins and growth factors...not all of them are known. For research, the convenient answer is to use an undefined media, often this mean using an animal derived serum. Another approach has been to use what are called "feeder cells" which also provide the trace amounts of proteins and growth factors to keep stem cells alive. However, both approaches can expose stem cells to nasty foreign biologic material like viruses (both known and unknown) that could be devisatating if subsequently transplanted in to people.

So the advance Dr. Zeng's lab has made in creating GMP-compliant culture conditions is an important step in moving stem cells closer to use in human therapy.

Friday, October 1, 2010

WPC: Final Thoughts

As the 2nd World Parkinson Congress comes to a close, PDF is humbled and honored to include the following closing thoughts from guest blogger and CRLI/WPC reporter, Diane G. Cook.

World Parkinson Congress: A “Tremor Safe” Zone

“Here at the World Parkinson Congress, we’re in a Tremor Safe Zone.” Those words were spoken by Davis Phinney to describe how it feels to be among thousands of others where it’s okay to have PD. No one notices the drag of foot, the slowness of step, the shakiness of hand, the softness of voice. Everyone does it! No self consciousness here!

But there’s something much more significant happening in Glasgow…it’s tangible. You can feel it in the smiles in the corridors, the instant friendships forged over a question posed of a speaker, the hug from a stranger, the expression of interest in a poster, the spontaneous gathering of people after lectures. There is a commonness of purpose and an honoring of each role in the fabric of how we will help ameliorate symptoms while working for a cure. Physicians, scientists, researchers, executives, nurses, therapists, people with Parkinson's, caregivers and the many others working to fight the devastating effects of this disease on many levels are in one conference hall … learning, exchanging views, questioning, and pushing the agenda forward.

One leaves each session in awe of the great minds at work, clear that there is a role for each one of us, stimulated by the learning, comforted by the cross fertilization of ideas, encouraged by the new partnerships and collaboration being forged, inspired by those overcoming limitations, and thankful that you somehow ended up in this special Parkinson’s disease community.

It doesn’t get any better than this! I, for one, am signed on for 2013 in Montreal and will offer my help in any way I can to make the 3rd WPC a significant step forward for the Parkinson’s community.

As I approach my last day, my focus turns to my next challenge: ”How will I translate the power of the Congress and the urgency of Now into my life back home.”


WPC Posters: More CRLI Grads Present

As we chatted about in a previous post, the 2nd World Parkinson Congress includes two poster sessions, one led by researchers about the science and care of Parkinson's and another led by Parkinson's community members who have made efforts in their community to move the cause forward.

We profiled a few of PDF's CRLI graduates yesterday and wanted to update you on some other individuals who have presented their work. Check out Israel's poster on the left and a list of others who presented during the week.

Israel Robledo
Parkinson’s Outreach. Hope For Tomorrow. Help For Today
(Robledo, I)

Israel works to educate people living with Parkinson’s in Midland, TX about clinical trials and, along with his wife Chris, he recently established a local non-profit to cover the cost of Parkinson’s medications and clinical trial participation for his neighbors in need. His poster discusses his first year of work and the lessens he has learned. Israel says that his participation in the WPC is not only an opportunity to talk about his work, but also to learn about the programs of others around the globe. He remarks,

“Sometimes we [people living with Parkinson’s disease] just live in our little shells because we feel that the disease is beyond us. But it’s not. We can help ourselves and others, too.”

Girija Muralidhar
The Neurowriters’ Guide To The Peripatetic Pursuit Of Parkinson’s Disease (PD): A Preview
Muralidhar, G (United States); Wittekind, P (United States); Kell, P (United States); Huseman, K (United States); Wheeler, J (United States); Brooks, L (United States); Herman, L (United States); Citron, J (United States); Willocks, P (United States); Ashford, L (United Kingdom); Cummings, R (United States)

Jean Burns
Working And Crossing National And Cultural Boundaries To Spread Parkinson’s Awareness
Capitanio, F (Spain); Burns, JE (United States); Martinez, C (United States)

Kate Kelsall and Valerie Graham
Role Of Patient Support In Shaping Expectations And Decision Making With Deep Brain Stimulation (DBS) Surgery
Klepitskaya, O; Kelsall, K; Graham, VW; McRae, CA (United States)

Jean Burns
Grassroots Effort To Make The PD Tulip The National Symbol For Parkinson’s Awareness In The United States
Burns, JE (United States)

WPC Roving Reporters: Day Three Science

The 2nd World Parkinson Congress continues its three days of sessions discussing the latest news in Parkinson’s science and care. PDF’s reporters have been listening in to a variety of these sessions. We've asked them to report back on what they have seen and heard, sharing the messages that mean the most to them as people living with Parkinson’s.

Jackie Hunt Christensen
Attended: Non-Drug Approaches to Treatments for Parkinson’s Disease

"This session covered how some symptoms of Parkinson’s disease might be improved by exercise; physical therapy; speech therapy; and non-drug, cognitive and psychological therapies.
  • Exercise: It was reported that exercise can improve motor performance and induce changes in the levels of dopamine and glutamate (a neurotransmitter that help us with learning, memory and other cognitive processes) and also affect the immune system function. Aspects of this type of exercise program focus on the intensity, specificity, difficulty and complexity of the task. All of those aspects make the participant use problem-solving skills. In other words, we’d be forced to think about our movements. More research is needed to determine how long these benefits of exercise last.
  • Physical Therapy: This can involve several types of exercises: aerobic, strength training, balance and coordination, and flexibility. Meta-analysis (a comprehensive review of many studies) showed that physical therapy can improve balance, strength, gait (speed as well as stride length) and quality of life. The type of exercise varies for each person, depending on a person’s age, previous experience with that type of exercise and access to any necessary equipment.
  • Speech Therapy: Not everyone with PD who has a speech problem is aware of it. The presenter said that speech problems tend to show up around seven years after onset of PD, and swallowing problems occur around 10 years after onset. Studies are being done on the vocal effects of choral singing (singing with a group). Researchers have compared the Lee Silverman Voice Therapy (“Think Loud!”) with speech amplification. Amplification was shown to increase speech intelligibility by 30%.
  • Non-drug, Cognitive & Psychological Therapies: Depression is very common in people with PD, but not everyone can tolerate antidepressants. Cognitive Behavior Therapy (CBT) is one non-drug method that is being considered for people with PD. CBT is a multi-week program in which patients learn to identify thought processes they undergo when upset or anxious, then work with a therapist to come up with ways to address those things."
Learn More:
Find resources for exercise
Read about complementary therapies

WPC Roving Reporters: Day Two Science

The 2nd World Parkinson Congress continues its three days of sessions discussing the latest news in Parkinson’s science and care. PDF’s reporters have been listening in to a variety of these sessions. They are reporting back what they have seen and heard….and sharing the most important messages they are hearing as people living with Parkinson’s.

Please note that this post was updated on October 5.

Kate Kelsall
Attended: Music as Pleasure and How It Can Empower You
Presenter: Concetta (Connie) Tomaino, D.A., MT-BC, LCAT, Executive Director of the Institute for Music and Neurologic Function in the Bronx, New York

"Some of the points in Connie’s presentation included:

  • We all experience music, but we all feel the beat differently.
  • The importance of singing songs one knows well to cue yourself (she sang “You are my ____”; the audience filled in the blank with SUNSHINE).

  • The prosody (rhythm, stress and the intonation) of singing matches that of the voice.

  • Songs can promote memory retrieval of past events that are associated with certain songs.

  • Music and songs can help with psychological issues such as depression or fatigue.

  • Choose music to move by and choose different music to lull yourself to sleep.

  • People with Parkinson’s have difficulty with articulation and lack of breath support. Singing can help with these issues. She illustrated with the song “Amen," noting she was able to help her patients increase their breath support from three syllables to 19 syllables."

Learn More:
Find resources on music and PD
Watch Ms. Tomaino's video presentation from PDF 2009 educational seminar

Jackie Hunt Christensen

Attended: Non-Motor Symptoms: Sleep, Pain, and Autonomic Dysfunction

"This session gave scientific validation to symptoms that many of us with Parkinson’s disease have been experiencing for years, often without acknowledgment from our physicians. Treatments haven’t been identified for most of these problems, but having them recognized as real phenomena that Parkinson’s disease may cause for some patients is a giant step in the right direction toward better patient care.

  • Sleep issues: It is estimated that 90% of people with PD will experience some sort of “sleep disturbance.” These can include reduced sleep; sleep fragmentation (waking up a lot during the night); Rapid Eye Movement (REM) Sleep Behavior Disorder (acting out our dreams, such as kicking, punching, screaming); or excessive daytime sleepiness. Some of these conditions can be worsened by PD medications.

  • Pain: Two-thirds of PD patients report pain that is directly related to their PD symptoms. This pain can occur in arms, legs, back, shoulders and usually occurs on the side of the body most affected by PD. It may improve after PD drugs are begun. Some people with PD may feel pain in a situation or experience that is painless for those without Parkinson’s. Others may feel extreme pain when people without PD feel only mild discomfort.

  • Autonomic dysfunction: The autonomic nervous system regulates “automatic” body functions. For those of us with Parkinson’s disease, autonomic dysfunction can include bladder problems; constipation; sexual dysfunction – in both women and men, excessive sweating, and sensitivity to cold."

Learn More:
Find resources for nonmotor symptoms in PD
Watch PDF's online seminar on nonmotor symptoms