Following the Latest Stem Cell Debate in D.C.

News from Parkinson's Action Network (PAN) 9-16-10

A few PD-related news updates

If you haven't read the 9/16/10 Parkinson's Action Network letter for the Senate Hearings on human embryonic stem cell research, here is a direct link to the hearings convened by Senator Tom Harkin.
What you will see is a video of the hearings convened to discuss the science not the legal entanglements or the preliminary injunction.

You can go to PAN to see the status of HR 1362 which is the National MS and Parkinson's Disease Registries Act.  From the same page you will find the link to contact your legistlators:  Help Make Stem Cell Research Legislation a Priority this Fall.  It is not too late to contact your Members of Congress to express your concern.

If you missed some of the Podcasts offered through the Michael J Fox Foundation, it isn't too late to download them.  We missed Dr Andrew Sngleton speaking on What Causes Parkinson'sDisease? so we'll be opening this download.  You can still find the Podcasts listed under News & Events.

Lonnie Ali's recent remarks at a conference sponsored by GSK for PD patients and caregivers made it clear that the support of family and loved ones is so important to getting an early diagnosis. They were married a year after his PD diagnosis but 5 years after his parkinsonian symptoms were dx'd.

What we learn is that everyone does their part.  If you can't be geographically close there is always email and the phone for quick contact.  Just reach out.

Is the Plug Being Pulled on Embryonic Stem Cell Line Distribution?

National Stem Cell Bank NIH Contract Not Renewed - WiCell Plans to Move Forward
How Will This Effect Parkinson's Disease Research?

The hope for the use of stem cells to treat and possibly cure many incurable diseases or life-altering conditions is shared by many patients and families around the world.  Research with human embryonic stem cells (hESC) is still relatively new, having been started at the University of Wisconsin-Madison in 1998 in the lab of James Thomson.  Despite the apparent opening of the field upon the Obama orders to the lift the Bush ban against federal research funds being used for hESC, the new NIH guidelines were not met with overwhelming enthusiasm.  Still restrictive, these guidelines did, however, open the door for researchers. Now, a few months later, it would appear that door is subtly and quietly being closed again.

The National Institutes Of Health (NIH) did not renew the National Stem Cells Bank's $16.1 million four year contract with WiCell Research, a private non-profit at the University of Wisconsin-Madison. The contract, which expired on February 28th, 2010, had subsidized the distribution of Federal approved embryonic stem cells. Since the contract was not renewed, the lack of action and adequate funding caused the bank to regroup.  The closing has led to a doubling of the price for approved stem cells. Costs have risen from $500 a vial to $1000 as there is no longer a subsidy for obtaining those 20 lines. 

Both the general public and medical scientists had thought that stem cell research would be moving much faster after President Obama took office and doubled the number human embryonic stem cell (hESC) lines available.  Instead, just getting the lines distributed to the researchers has become an unexpected hurdle. The former National Stem Cell Bank has now become the Wisconsin International Stem Cell Bank (WISC Bank) but the prices have been doubled in order to meet previously subsidized expenses. It is not clear how this will effect future research.  WiCell has, however, expanded its lines from 20 to 33 and is planning to move forward.

Is there an underlying reason to attempt to bar one door in favor of another to open wider for another area of research?  We don't have that answer; the transition seemed low-key.  But there in recent years there has been an expanding a trend toward not the scientific significance but rather the economic impact of research.  Science, politics and finance do not make a great martini; but they do make the research drink in which currently we find ourselves.

In January NIH expanded its contract with a different cell bank. Coriell Institute for Medical Research is a non-profit institution which established the Human Genetic Cell Repository in 1972. Coriell is known to have the largest bank of human cell lines and has long been associated with the NIH. In 2010 the National Institute of Health provided $27 million to Coriell for banking induced pluripotent stem cells(IPS), for which there is no destruction of embryos, to create a stem cell lab and a human genetic biobank.  Nothing is planned at this point for specifically underwriting embryonic stem cell banking. 

The hope is that the NIH will continue to consider setting up its own public stem cell bank if they are not going to renew with WiCell.  This action would reduce the price and speed the delivery of all lines but especially the new lines approved by the Obama administration.
 

Currently there are 109 hESC lines awaiting NIH approval for NIH funded research, 237 lines for which there is an intent to submit. 51 hESC lines (3 on hold) are available in the NIH Registry.  The approved lines are eligible for NIH funding grants but these lines are available only through individual institutions. A total of 54 institutions have submitted lines or intend to do so.  The future costs per vial for researchers is unknown although currently Children's Hospital Boston, affiliated with Harvard Medical School which is still shipping lines at a loss.

While there are many big pharma potentials, it is the preliminary and intermediate work in understanding the medical conditions, recognizing the best targets, developing safe and effective processes and procedures that must take place before there is more than profit potential.  And after that the intellectual property rights will have to be determined and the debates will rage.

Waiting for effective treatments are people with Parkinson's and Alzheimer's disease, diabetes, heart disease, spinal cord injury and many more. For these people with incurable conditions has a hope been dashed or simply redirected to another area of scientific research?  Time, once more, may not be on our side.

References:

The Scientist.com

Wi Cell.org

NIH Telethon Network of Genetic Biobanks

Nature.com 

Another Look at Stem Cell Research by Dr Alan Malnak

About the Coriell Institute for Medical Research 

Promises, Promises by Stuart Blackman, The Scientist

You can read more about stem cell basics at the National Institutes of Health website. You can also read the 2006 NIH Stem Cell Report of progress made since 2001.

Parkinson's Disease Research - Part II

Reprogrammed Skin Cells for Parkinson's

There are other methods for developing effective treatments and possibly cures for diseases through the adaptation of non-embryonic stem cells which are induced to have the pluripotent capabilities. There appears to be potential if these cells can live up to their promise.

Induced pluripotent cells
Non-embryonic stem cells induced/stimulated to
become pluripotent (iPS)

Embryonic stem cells
Already pluripotent (ESC) capable of being any cell

hESC
Human embryonic stem cells

hiPSC
Human induced pluripotent cells

Pluripotent
having the capability to be any cell

Gene expression
a measure of which genes the cell uses and how much of the
cell uses those genes

Genetic recombination
a strand of genetic material (DNA or sometimes RNA)
is broken and joined to a different DNA molecule
Botanists have been grafting for centuries but this is nano
micro-scale and complicated

Cre recombinase
Enzyme which catalyzes genetic recombination

Transgene
Genetic material transferredfrom one organism to another;
any DNA sequence introduced into an organism.

Factor-free
without being introduced via a virus factor

As you already know, researchers in Cambridge MA have been able to use skin cells which were induced into pluripotency to become dopamine-producing neuron cells. Unlike previous research, these iPS did not insert cancer producing genes into the DNA because that reprogramming was removed allowing the cells to maintain an ESC-like status.

The problem in the past has been that the reprogrammed cells had the potential to cause cancer, creating an out of the frying pan and into the fire situation. In the new process: the skin cells of a Parkinson's patient are reprogrammed to be iPS cells. And then reprogrammed again so that the potentially cancer-causing program was removed. At this point the human induced pluripotent cells (hiPS) are very similar to human embryonic stem cells (hESC)

In the past researchers were not sure that removing the reprogramming from these human cells would allow the cell to maintain a stable iPS state so that it could multiply. Now they are. This step opens the door...


Links:
http://www.cell.com/abstract/S0092-8674(09)00151-2
http://esciencenews.com/articles/2009/03/05/breakthrough.produces.parkinsons.patient.specific.stem.cells.free.harmful.reprogramming.genes

The Scientists:
Whitehead Institute for Biomedical Research, Cambridge, Mass: Frank Soldner, Dirk Hockemeyer, Caroline Beard, George W Bell, Elizabeth G Cook, Qing Gao, Miasam Mitalippova, Rudolph Jaenisch
Dept of Biology MIT, Cambridge, Mass: Rudolph Jaenisch
Udall Parkinson Disease Research Center of Excellence, Center for Neuroredegeneration Research, McLean Hospital/Harvard Medical School, Cambridge, Mass: Gunnar Hargus, Alexandra Blak, Oliver Cooper, Ole Isacson