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Treatment of DMD in patient R. Medical case report No.2

We had honor to accept at EmCell Clinic Mr. R. attended by his parents.

According to the patient's parents, symptoms of the disease started to reveal about 2–3 years ago. Diagnosis was established a year ago. Last year the progress of the disease was more rapid then before. No drug allergy and children infections reported.

After previous treatment at the clinic parents of R. noted the cessation of disease progress and following improvements of the boy's condition:

R. has gained weight and grew up, total mass of his muscles increased. General muscle strength has increased, especially in arms, coordination has improved, gait has become more free, active, and coordinated, waddling is no more observed, walking from the heel has become possible, in August–November he could even run some distance. Before the treatment, R. used to fall several times per day (3–5). After the treatment the quantity of falls reduced to 1–2 per week. There were weeks when he did not fall at all. Movements of the eyes became more concentrated, floating is no more observed, his eyes can now be fixed steadily. Breathing has become easier. Cough is more active. According to the data of instrumental investigations of muscles, the performance of grip test improved: it has increased from 16 to 22 and 23 pounds in two months after the first treatment. Performance in computer testing revealed increase in muscular capacity up to 700% (investigations were conducted in June, July, September, and October). As for his movements and physical activity, the boy had regular training under constant medical control. From the beginning of November, his parents noted some increase of movement difficulties and applied for the next course of cell therapy with the use of fetal stem cell administration that was recommended in 3-4 months after the initial course.

Present condition

General condition is satisfactory. Mental and physical development according to the age.
Hyperlordosis, unsteady gait with tiptoes going down. Skin is of a normal color. Lymphatic nodes are not enlarged at palpation. Heart rate is regular, 86 bpm. Tones of the heart are regular, melody is normal, no murmurs present. On percussion lung fields are clear - normal sound in all lines. On palpation abdomen is soft, painless. Liver edge is at costal arch, normal, spleen is not palpable. According to the patient, stool and urination are regular, normal. Last year the progress of the disease was more rapid than before.

Examination by the neurologist

Patient's condition has substantially improved. He does not fall while walking, he has gained weight and grown by 3 cm, the boy became more active and confident in his movements. Sight is fixed, movements of eye apples are to the full capacity, their convergence is good. At palpation, arms and leg muscles are resilient enough, muscle tone is practically normal, except in the right leg. The same normalization is revealed by passive movement of the extremities. Reflexes are flaccid, in arms D<S, genial – D=S, heel reflex is stronger then before, S=D. Babinsky sign is not observed, plantar symptom is evoked on the left side, on the right side - a "distorted" one (not pathological already, but not normal yet).

The test to sit down on the floor and to get up performs with difficulty, but more surely, "climbs" by his own body only during the last stage of the test. The improvement of the gait: it has become more harmonic, closer to normal, waddling (twisting of the pelvis) has disappeared. Lordosis and hypotrophy of the back muscles are still observed. According to parents' observations, the boy could run some distance in two month after the first treatment (in August - October mostly). By November all motional activities has become more difficult.

Diagnosis: Duchenne muscular dystrophy.

Lab tests (blood count, chemistry, routine urine test) were normal.

Recommendations: continue fetal stem cell therapy

Mr.R. received a course of cell therapy:

1st day of treatment. administration of sample 3038A640 i.v. dropwise, 2 ml. administration of sample 3038AM640 s/c in buttocks, in two depots 0.7 ml each.

2nd day of treatment. administration the sample 3038A640 i.v. dropwise, 1.4 ml. administration of sample 3038AM640 s/c in buttocks, in two depots, 1.0 ml each.

3rd day of treatment. administration of sample 3038IE229 s/c in frontal abdomen, in three depots 0.6 ml each.

4th day of treatment. administration of sample 3038A227 i.v. dropwise, 2.2 ml. administration of sample 3038AM227 s/c in frontal abdomen, in three depots, 1.1 ml each.

5th day of treatment. administration of sample 3038A227 i.v. dropwise, 1.5 ml. administration of sample 3038AM227 s/c in frontal abdomen, in four depots (2 in buttocks, 2 in frontal abdomen) 5.0 ml (total).

The cell suspension samples 3038A640, 3038AM640, 3038IE229, 3038A227 and 3038AM227 are certified as follows:

Bacterial sterility test Negative
Prenatal Diagnostics  
Test with cardiolipid antigen Negative
Ami-HIV1/HIV2 Negative
HBsAg Negative
Embryonic Diagnostics  
Enzygnost Ami-HIV1/HIV2 Negative
HBsAg monoclonal Negative
Anti-HBc monoclonal Negative
Anti-CMV/IgG+IgM Negative
Anti-Rubella Virus/IgG Negative
Varicella/Zoster Negative
Toxoplasmosis/IgG Negative

Second examination by the neurologist

The boy feels well walks without falling down, can run.
In neurological status, he is in cooperative, adequate. He can fix the sight, movements of eye apples are to the full range. Muscle tone of arms and left leg is good, reduced in the right leg. Reflexes are stronger, in arms D=S, knee D=S, heel reflex is stronger, S=D. Plantar reflexes present at both sides. The test of squatting and getting up performs as before the treatment. The boy and his parents note good mood and improvement of physical functions in the process of treatment.

Results of the treatment

Positive changes after first course of treatment described above are quite considerable. The second course of treatment was well tolerated with the good mood and eagerness, despite some moments of childish capriciousness and anxiety. Some immediate improvements of the patient’s condition are noted.

Mr. R. is recommended:

1.    Follow-up by the neurologist in the USA.
2.    Diet rich in proteins (veal, fish, mushrooms, etc.).
3.    Regular training in movement of legs and arms.
4.    Avoid infections.
5.    Reports (by parents and professional by neurologist) once a month.
6.    Repeated course of cell therapy in 8–12 months. The exact timing of the visit is to be arranged according to patient's progress.