1.This Act may be cited as the
Brittany Wilkinson Mitochondrial
Disease Research and Treatment Enhancement Act
.
2.
(a)Congress finds the following:
(1)Mitochondrial disease results when there is
a defect that reduces the ability of the mitochondria in a cell to produce
energy. As mitochondria fail to produce enough energy, the cells will cease to
function properly and will eventually die. Organ systems will begin to fail,
and the life of the individual is compromised or ended.
(2)There are more
than 40 mitochondrial diseases.
(3)Mitochondrial
diseases are a relatively newly diagnosed group of diseases, first recognized
in the late 1960s. Diagnosis of these diseases is extremely difficult.
(4)Mitochondrial
diseases can present themselves at any age, with associated mortality rates
that vary depending upon the particular disease. The most severe diseases
result in progressive loss of neurological and liver function, and death within
several years.
(5)According to the
National Institute of Environmental Health Sciences, half of those affected by
mitochondrial disease are children, who show symptoms before age five and
approximately 80 percent of whom will not survive beyond the age of 20.
(6)Mitochondrial dysfunction is also
associated with numerous other disorders, including many neurological diseases
(such as Parkinson’s, Alzheimer’s, ALS, and autism), and other diseases
associated with aging, diabetes, and cancer.
(7)Mitochondrial
diseases are most commonly the result of genetic mutation, either in the
nuclear DNA or in the mitochondrial DNA. Some mitochondrial diseases have been
attributable to environmental factors that interfere with mitochondrial
function.
(8)Researchers
estimate that one in 4,000 children will develop a mitochondrial disease
related to an inherited mutation by the age of 10 years, and that 1,000–2,000
children are born each year in the United States who will develop mitochondrial
disease in their lifetimes. However, studies of umbilical cord blood samples
show that one in 200 children are born with both normal and mutant
mitochondrial DNA, and the number of children with these mutations who actually
develop a disease is unknown.
(9)There are no cures
for any of the specifically identified mitochondrial diseases, nor is there a
specific treatment for any of these diseases.
(10)Improving our
basic understanding of mitochondrial function and dysfunction has potential
application to numerous areas of biomedical research. The National Institutes
of Health has taken an increased interest in mitochondrial disease and
dysfunction and has sponsored a number of activities in recent years aimed at
advancing mitochondrial medicine, including incorporating research into
functional variation in mitochondria in the Transformative Research Grants
Initiative.
(b)It
is the purpose of this Act to promote an enhanced research effort aimed at
improved understanding of mitochondrial disease and dysfunction and the
development of treatments and cures for mitochondrial disease.
3.Enhancement of
research and treatment activities related to mitochondrial disease
(a)Mitochondrial
disease research enhancementPart A of title IV of the Public Health
Service Act (42 U.S.C. 281 et seq.) is amended—
(1)by redesignating
section 404H as section 404I; and
(2)inserting after
section 404G the following new section:
404H.Office of
Mitochondrial Disease
(a)There is established within the Office of
the Director of NIH at the Division of Program Coordination, Planning and
Strategic Initiatives, an office to be known as the Office of Mitochondrial
Disease (in this section referred to as the Office
), which shall
be headed by a Director (in this section referred to as the
Director
), appointed by the Director of NIH.
(b)Mitochondrial
disease research plan
(1)The Director shall develop, make publicly available, and
implement a written plan to facilitate and coordinate research into
mitochondrial disease.
(2)The
plan required under paragraph (1) shall include the following
objectives:
(A)Improving
coordination of research related to mitochondrial disease among the national
research institutes and between the National Institutes of Health and outside
researchers.
(B)Providing training
to research scientists and clinical researchers engaged in research related to
mitochondrial disease.
(C)Conducting
programs to provide information and continuing education to health care
providers regarding the diagnosis of mitochondrial disease.
(D)Ensuring relevant
scientific review groups contain individuals with expertise in mitochondrial
disease.
(3)In
developing the plan under paragraph (1), the Director shall consult
with—
(A)the Director of
the National Cancer Institute;
(B)the Director of the National Institute of
Child Health and Human Development;
(C)the Director of the National Institute of
Environmental Health Sciences;
(D)the Director of the National Heart, Lung,
and Blood Institute;
(E)the Director of the National Institute of
Neurological Disorders and Stroke;
(F)the Director of the National Institute of
Diabetes and Digestive and Kidney Diseases;
(G)the Director of the National Eye
Institute;
(H)the Director of
the National Institute of Mental Health;
(I)the Director of
the National Institute of Arthritis and Muscoloskeletal and Skin
Diseases;
(J)the Director of
the National Human Genome Research Institute; and
(K)the heads of such
other institutes and offices as the Director considers appropriate.
(4)The
Director shall update the plan required under paragraph (1) on a biennial
basis.
(c)In addition to any
grants otherwise awarded by the National Institutes of Health for research in
mitochondrial disease, the Director may award competitive, peer-reviewed
grants—
(1)for integrated,
multi-project research programs related to mitochondrial disease; and
(2)for planning activities associated with
integrated, multi-project research programs related to mitochondrial
disease.
(d)
(1)The Director may
award grants to institutions or consortiums of institutions to establish
Mitochondrial Disease Centers of Excellence to promote interdisciplinary
research and training related to mitochondrial disease.
(2)A grant awarded under paragraph (1) may be used
to—
(A)conduct basic and clinical research related
to mitochondrial disease;
(B)facilitate training programs for research
scientists and health professionals seeking to engage in research related to
mitochondrial disease; and
(C)develop and disseminate programs and
materials to provide continuing education to health care professionals
regarding the recognition, diagnosis, and treatment of mitochondrial
disease.
(e)National
registry; biorepository
(1)The Director of the Centers for Disease Control and
Prevention shall establish a national registry for the maintenance and sharing
for research purposes of medical information collected from patients with
mitochondrial disease.
(2)The
Director of the Centers for Disease Control and Prevention shall establish a
national biorepository for the maintenance and sharing for research purposes of
tissues and DNA collected from patients with mitochondrial disease.
(f)In
this section, the term mitochondrial disease
means mitochondrial
diseases, mutations, dysfunctions and functions.
(g)Authorization
of appropriationsThere is authorized to be appropriated, such
sums as may be necessary to carry out this
section.
.
(b)Development of
mitochondrial disease research planThe Director of the Office of Mitochondrial
Disease shall develop and make publicly available the mitochondrial disease
research plan required under section 404H(b)(1) of the Public Health Service
Act, as added by subsection (a) of this section, not later than 180 days after
the date of the enactment of this Act.