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Without PAN, Amy Comstock and our PAN founder Joan Samuelson, we, the PD community, would not have a voice on Capitol Hill


Statement of Amy Comstock Rick Chief Executive Officer Parkinson's
Action Network

Committee on House Small Business Subcommittee on Investigations and
Oversight

February 13, 2008

See:
Ms. Amy Comstock Rick
Chief Executive Officer
Parkinson’s Action Network
Washington, DC

Thank you, Chairman Altmire, and Ranking Member Gohmert for inviting
me to testify on behalf of the Parkinson's Action Network regarding
the Small Business Innovation Research (SBIR) program. As you know, I
am the Chief Executive Officer of the Parkinson's Action Network, also
known by our acronym, PAN.

PAN represents the entire Parkinson's community, including the more
than one million Americans currently fighting Parkinson's disease
(PD), the estimated 60,000 newly diagnosed every year, and their
families, and all the national Parkinson's organizations, including
The Michael J. Fox Foundation for Parkinson's Research, Parkinson's
Disease Foundation, National Parkinson Foundation, Parkinson Alliance,
and American Parkinson Disease Association.

Parkinson's disease is a chronic, progressive neurological disorder
that results from degeneration and premature death of
dopamine-producing brain cells. It is the secondmost common
neurodegenerative disease in the United States. The cause of PD is
unknown, although research points to a combination of genetic and
environmental factors. PD is currently without known cure.

Parkinson's patients experience devastating physical and mental
symptoms such as tremors, debilitating slow movements, postural
instability (balance problems), sleep disturbances, and a variety of
cognitive impairments. Today, treatment options only provide some
symptomatic relief but are in no way neuroprotective; halting or
reversing the progression of the disease. Current state-of-the-art
treatment for people with Parkinson's disease is rooted in levodopa
and its derivatives. Levodopa was approved almost 40 years ago and,
sadly, is still the primary treatment for Parkinson's. Yet, levodopa
and the derivatives only treat the symptoms of the disease and are
only effective in treating symptoms for a limited period of time. We
still have nothing that will actually slow the progression of
Parkinson's or that will ward off ultimate and complete disability.

As Parkinson's progresses, even with treatment, substantial disability
-- including the inability to maintain balance, walk, speak, and move
-- is inevitable and makes assisted living and nursing home care
necessary. Parkinson's disease sufferers are desperately awaiting an
innovative neuroprotective treatment that will relieve their pain and
halt the disease.

Before I begin to discuss the Small Business Innovation Research
(SBIR) program specifically, it is helpful to understand the context
in which PAN views all National Institutes of Health (NIH) programs.
As you may know, NIH is the single largest source of Parkinson's
disease research funding in the world, and the basic discoveries
coming out of NIH are very important, but it is our belief that NIH is
not funding enough research that aims to translate basic scientific
discoveries into therapies for people living with diseases. As I
testified before the House Appropriations Committee in 2006, the
primary focus at NIH is on basic research - research that is not
necessarily geared towards therapeutic outcomes - rather than research
that will advance scientific innovation towards needed therapies.

The drug development process takes many years from beginning to end.
At the beginning you have basic research supported by NIH. At the end,
one hopes, you have a drug, biologic, or treatment, approved by the
FDA, that is available to those afflicted with a particular disease.
Unfortunately, between these two bookends of well-understood areas of
federal oversight, you have a process that is often-times confusing,
inefficient, and not geared toward improving the public health.
Currently, no one in the federal government is responsible for
ensuring that the scientific baton (promising early NIHfunded
research) is passed from basic discovery onto private development,
generally a pharmaceutical or biotechnology company, that will pick up
the project and see it through to the end of the FDA approval process.
This middle part of the process, where promising drugs can be lost and
no one is ensuring that good ideas in the lab are "translated" into
real possibilities for patients, is referred to as the "Valley of Death."

It has been the position of the Parkinson's community for quite some
time that NIH should focus on patient-oriented outcomes by doing more
to combat the "Valley of Death." We have suggested that more of the
NIH extramural grant program should be focused on potential therapies
for particular diseases. Unfortunately, however, due to a lack of
funding and in order to maintain basic research grants, NIH has not
only not focused more on translational research, but has actually cut
these programs. As Dr. Zerhouni said in his Senate Labor, Health and
Human Services, and Education Appropriations Subcommittee testimony on
March 19, 2007, "the impact [of NIH budget cuts] is primarily in our
ability to translate from the laboratory to the clinic to the bedside
into the community what we need to do to prevent diseases."

It is disconcerting for people living with Parkinson's and other
un-treated or undertreated conditions to know that many potential
drugs are languishing in the "Valley of Death" simply because there is
not enough funding to move basic research to product development. This
science is some of the most difficult and costly research needed to
develop therapies and meet the public health need, including
developing pre-human testing, efficacy trials, production design and a
range of other steps needed to determine whether a drug will be safe
and effective. It is also essential for reducing the burden of disease
and disability for millions of Americans.

Having heard our vision of the need for NIH to refocus some of its
grantmaking, it should be clear why I and the Parkinson's community
are so strongly supportive of the SBIR program. SBIR grants have a
significant role to play in the drug development arena called the
"Valley of Death. The Parkinson's Action Network strongly supports the
entire NIH SBIR program, but there are several important elements of
the program that I will highlight and a few suggestions of areas that
would benefit from improvement. NIH SBIR grants, of course, are
awarded to small companies that conduct biomedical research. In order
to address the NIH SBIR program from a biomedical perspective, it
important to understand in a little more detail, the how these small
biomedical companies function. Generally, these companies have one or
two lead projects for which they are able to raise funds through
private investors. These research projects are investigations into
promising products or therapies that investors have determined to be
worthy of their money. But, like many things in life, there is often a
second tier. The second tier includes research that is also promising,
but which, for one reason or another, is not as appealing to private
investors. The lack of appeal to investors may occur for a number of
reasons - it can be that the science at issue, while worthy, is less
certain and more of a risk so private investors are more leery. Or,
the lack of appeal to private investors may be because of the size of
the potential market. This is a very real issue for a disease like
Parkinson's that, while between one and one and a half million people,
is not a sizeable market that is appealing for potential profit.

Companies may have trouble attracting private investors to support
this second category of projects, which are scientifically valuable
research projects but are less certain in terms of the potential
return on investment. I cannot emphasize to you enough how troubling
it is to a person with Parkinson's or their loved one that there are
potentially hundreds of bright ideas out there for better treatments
for Parkinson's disease that are not being pursued because our system
does not have a process for ensuring that good ideas are not lost. In
fact, in a perfect world there should be a way of ensuring that
promising ideas move through the pipeline as quickly as the science
dictates and the potential benefit to the public health demands. But
this is not the case. There is no guarantee that a promising therapy
for a disease with a very small population, for example, will move
through the pipeline at all. Similarly, there is no guarantee that a
risky idea for a disease that affects a larger population, let's say
Alzheimer's Disease, with a population of about 4.5 million and
growing, will be pursued.

This is where SBIR comes in. The SBIR program supports cutting- edge
research where other sources of research are difficult if not
impossible to obtain. But when you turn that thought around and look
at it from a patient perspective, it is not that this program is about
funding, it is that this program makes possible research for many
diseases that would not otherwise occur. That is invaluable.

Having stated our strong support for the NIH's SBIR program, however,
I do want to offer an important recommendation for the future. As this
committee is well aware, the 2003 SBA ruling regarding SBIR
eligibility based on majority ownership by "individuals" has had a
negative impact on the biomedical research community. It is my
understanding that, since that ruling, applications to the NIH SBIR
program have dropped precipitously. SBIR applications, we have been
told, are down 11.9% in 2005, 14.6% in 2006, and 21% in 2007. And,
given the increase in most applications to NIH, it is fair to assume
that this drop is a direct result of the eligibility ruling.

From a patient perspective it does not seem logical, and is in fact
scary, that we eliminate from eligibility research projects that
otherwise merit funding, because of the financial structure of the
company. And, the reasoning becomes more muddled when one focuses on
the fact that the companies that are being excluded by the SBA rule
are the very ones that are doing work that is good enough, for
whatever reason, to have attracted venture capital money. The very
companies that are doing a good enough job in one area are, because of
that success, barred from federal support for other promising
research. This policy doesn't penalize companies, it penalizes patients.

Let me give you an example of the impact of this ruling. It is a sad
reality that there is a lot we still do not understand about
neurological diseases. There are many investigations into compounds
that show therapeutic promise in pre-clinical and even early clinical
stages in which the research is dropped and no actual therapies are
developed. That is why our community is heavily invested in any
potential therapy that is beginning to show real promise. We have one
now, Spheramine, that is in Phase II clinical trials.

Spheramine, quite simply, injects retinal cells into the brain,
surgically, to provide a continuous source of dopamine. While this
trial is now in Phase II and our community is quite enthusiastic about
its promise, the animal research and Phase I research was funded
through an SBIR grant. We are fearful that keeping the eligibility
ruling as it now stands will keep this kind of promising research from
going forward. In fact, who knows what promising therapies are sitting
now, unfunded and not moving?

I would like to make one final point in support of revisiting the
ruling SBIR ineligibility based on venture capital investment. At NIH,
as with SBIR throughout the government, 2.5 percent of the extramural
grant monies are set aside for this program. By eliminating a large
percentage of private, innovative researchers, we are left with a much
smaller pool of applicants from which NIH can draw when funding these
grants. And, while all applications are peer-reviewed so, presumably,
are all good science, it just seems logical to me that we would want
to do everything we could to invite as many applications as possible
to go into that peer-review process so that we are assured that what
comes out is the best science, with the most promise, that we can fund.

As PAN continues working toward better treatments and cures for
Americans, we respectfully seek the Small Business Committee's support
for a robust SBIR program at NIH. SBIR is an essential program that
provides key funding for patient-oriented research currently
languishing in the "Valley of Death" of the biomedical research
system. We respectfully request that your support include a revision
to not eliminate small companies simply based on their financial
structure.

Thank you again for this opportunity to provide testimony. I look
forward to working with the Committee on this critical issue for the
Parkinson's community, the small business community, and all American
families facing disease and disability.