1.This Act may be cited as the
Mitochondrial Medicine 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 the mitochondria fail to produce enough energy, the cell 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 specifically identified mitochondrial diseases, but the vast majority
have not yet been identified.
(3)Mitochondrial
diseases are a relatively newly diagnosed group of diseases, first recognized
in the late 1960s. Diagnosis is extremely difficult for a number of
reasons.
(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 related disorders, including many common
neurological diseases (such as Parkinson’s, Alzheimer’s, ALS, and autism), and
other diseases associated with aging, diabetes, cancer, and obesity.
(7)Mitochondrial
disease is most commonly the result of genetic mutation, either in the nuclear
DNA or in the mitochondrial DNA. Some mitochondrial diseases also are
attributable to environmental factors, including prescription medications, 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 for mitochondrial disease.
3.Enhancement of
research and treatment activities related to mitochondrial medicine
(a)Mitochondrial
medicine 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 Medicine
(a)There is established within the Office of
the Director of NIH an office to be known as the Office of Mitochondrial
Medicine (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
medicine research plan
(1)The Director shall develop, make publicly available, and
implement a written plan to facilitate research into mitochondrial
medicine.
(2)The
plan required under paragraph (1) shall include the following
objectives:
(A)Improving
coordination of research related to mitochondrial medicine among the national
research institutes and between the National Institutes of Health and outside
researchers.
(B)Providing training
to research scientists and health professionals engaged in research related to
mitochondrial medicine.
(C)Providing training
to health care providers regarding the diagnosis of mitochondrial disease and
dysfunction.
(D)Establishing
scientific review groups with expertise in mitochondrial medicine to oversee
relevant research projects in the National Institutes of Health.
(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;
and
(H)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 medicine, the Director shall annually award—
(1)at least five
grants for integrated, multi-project research programs related to mitochondrial
medicine; and
(2)at least five grants for planning
activities associated with integrated, multi-project research programs related
to mitochondrial medicine.
(d)
(1)The Director may
award grants to institutions or consortiums of institutions to establish
Mitochondrial Medicine Centers of Excellence to promote interdisciplinary
research and training related to mitochondrial medicine.
(2)A grant awarded under paragraph (1) shall be used
to—
(A)conduct basic and clinical research related
to mitochondrial medicine;
(B)facilitate training programs for research
scientists and health professionals seeking to engage in research related to
mitochondrial medicine;
(C)develop and disseminate programs and
materials to provide continuing education to health care professionals
regarding the recognition, diagnosis, and treatment of mitochondrial disease
and dysfunction; and
(D)provide living stipends for research
scientists and health professionals enrolled in mitochondrial research training
programs.
(e)National
registry; biorepository
(1)The Director shall establish a national registry for the
maintenance and sharing for research purposes of medical information collected
from patients with mitochondrial disease or dysfunction.
(2)The
Director shall establish a national biorepository for the maintenance and
sharing for research purposes of tissues and DNA collected from patients with
mitochondrial disease or dysfunction.
(f)In
this section, the term mitochondrial medicine
means medical
treatment related to mitochondrial disease or
dysfunction.
.
(b)Development of
mitochondrial medicine research planThe Director of the Office of Mitochondrial
Medicine shall develop and make publicly available the mitochondrial medicine
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.